Page last updated: 2024-11-08

melphalan

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Description

Melphalan: An alkylating nitrogen mustard that is used as an antineoplastic in the form of the levo isomer - MELPHALAN, the racemic mixture - MERPHALAN, and the dextro isomer - MEDPHALAN; toxic to bone marrow, but little vesicant action; potential carcinogen. [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

melphalan : A phenylalanine derivative comprising L-phenylalanine having [bis(2-chloroethyl)amino group at the 4-position on the phenyl ring. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID460612
CHEMBL ID852
CHEBI ID28876
SCHEMBL ID5872
MeSH IDM0013330

Synonyms (187)

Synonym
phenylalanine mustard
alanine nitrogen mustard
sk 15673
nsc-8806
BIDD:GT0044
smr000058720
MLS002153368
AB00053282-07
AB00053282-08
BRD-K87827419-001-02-8
DIVK1C_000653
KBIO1_000653
4-[bis(2-chloroethyl)amino]-l-phenylalanine
melphalan, powder
SPECTRUM_000397
PRESTWICK_1006
BSPBIO_002407
alkeran
levofalan
melfalan
nci-c04853
l-sarcolysin
cb 3025
(2s)-2-amino-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoic acid
l-pam
sk-15673
alanine, 3-[p-[bis(2-chloroethyl)amino]phenyl]-, l-
l-sarcolysine
l-sarkolysin
nsc8806
l-phenylalanine mustard
IDI1_000653
SPECTRUM5_001601
SMP2_000174
NCGC00090757-01
melphalanum [inn-latin]
l-3-(para-(bis(2-chloroethyl)amino)phenyl)alanine
hsdb 3234
nsc 241286
levofolan
ccris 374
rcra waste no. u150
l-phenylalanine, 4-(bis(2-chloroethyl)amino)-
brn 2816456
alanine, 3-(p-(bis(2-chloroethyl)amino)phenyl)-, l-
melfalano [inn-spanish]
levopholan
p-l-sarcolysine
rcra waste number u150
einecs 205-726-3
C07122
148-82-3
melphalan
p-n,n-bis(2-chloroethyl)amino-l-phenylalanine
p-bis(beta-chloroethyl)aminophenylalanine
3-(p-(bis(2-chloroethyl)amino)phenyl)-l-alanine
4-(bis(2-chloroethyl)amino)-l-phenylalanine
DB01042
l-3-(p-(bis(2-chloroethyl)amino)phenyl)alanine
3-p-(di(2-chloroethyl)amino)-phenyl-l-alanine
p-l-sarcolysin
p-n-bis(2-chloroethyl)amino-l-phenylalanine
l-phenylalanine, 4-[bis(2-chloroethyl)amino]-
phenylalanine nitrogen mustard
p-di-(2-chloroethyl)amino-l-phenylalanine
nsc-241286
D00369
melphalan (jp17/usp/inn)
alkeran (tn)
NCGC00090757-02
KBIO3_001627
KBIOGR_001284
KBIO2_000877
KBIO2_003445
KBIO2_006013
KBIOSS_000877
SPECTRUM3_000684
SPECTRUM4_000882
SPBIO_000287
SPECTRUM2_000074
NINDS_000653
SPECTRUM1500382
NCGC00090757-03
mustard, phenylalanine
HMS2090B09
HMS2091B16
cb-3025 ,
chebi:28876 ,
CHEMBL852 ,
sarcolycin, l-
HMS502A15
MLS001333666
melphalanum
melfalano
8057-25-8
1217854-43-7
(s)-2-amino-3-(4-(bis(2-chloroethyl)amino)phenyl)propanoic acid
2-amino-3-{4-[bis-(2-chloro-ethyl)-amino]-phenyl}-propionic acid(melphalan)
2-amino-3-{4-[bis-(2-chloro-ethyl)-amino]-phenyl}-propionic acid(l-pam)
2-amino-3-{4-[bis-(2-chloro-ethyl)-amino]-phenyl}-propionic acid (melphalan)
(s)-2-amino-3-{4-[bis-(2-chloro-ethyl)-amino]-phenyl}-propionic acid
bdbm50025837
2-amino-3-[4-bis(2-chloroethyl)amino]phenylpropanoic acid
2-amino-3-{4-[bis-(2-chloro-ethyl)-amino]-phenyl}-propionic acid
(2s)-2-azanyl-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoic acid
A808810
NCGC00090757-04
(2s)-2-azaniumyl-3-[4-[bis(2-chloroethyl)amino]phenyl]propanoate
3-(p-(bis(2-chloroethyl)amino)phenyl)alanine
AY33600000 ,
niosh/ay3360000
alanine, 3-(p-(bis(2-chloroethyl)amino)phenyl)-
NCGC00260071-01
tox21_202522
4-[bis(2-chloroethyl)-amino]-l-phenylalanine
4-[bis(2-chloroethyl)amino]-(l)-phenylalanine
nsc757098
pharmakon1600-01500382
nsc-757098
dtxsid6020804 ,
dtxcid00804
cas-148-82-3
tox21_111010
4-[bis-(2-chloroethyl)amino]-l-phenylalanine
(2s)-2-amino-3-(4-[bis(2-chloroethyl)amino]phenyl)propanoic acid
HMS2235D21
CCG-39704
3025 c.b.
4-14-00-01689 (beilstein handbook reference)
melphalan [usan:usp:inn:ban:jan]
q41or9510p ,
unii-q41or9510p
NCGC00090757-05
melphalan [iarc]
melphalan [jan]
melphalan [orange book]
melphalan [ep monograph]
melphalan [inn]
melphalan [mart.]
melphalan [mi]
melphalan [vandf]
melphalan [usp monograph]
melphalan [hsdb]
melphalan [usan]
melphalan [who-dd]
EPITOPE ID:141802
AKOS015895374
S8266
DL-442
HY-17575
CS-3120
l-phenylalanine mustard (l-pam)
gtpl7620
phelinun
SCHEMBL5872
tox21_111010_1
NCGC00090757-06
4-[bis(2-chloroethyl)-amino]-l-phenyl-alanine
W-108096
AB00053282_09
mfcd00057717
(2s)-2-amino-3-{4-[bis(2-chloroethyl)amino]phenyl}propanoic acid
SR-05000001667-1
sr-05000001667
SBI-0052787.P003
Q2298283
AS-13314
EN300-7479343
l01aa03
para-di(2-chloroethyl)amino-l-phenylalanine
3(p-(bis(2-chloroethyl)amino)phenyl)-l-alanine
melphalan (usp monograph)
melphalan (mart.)
melphalan (usan:usp:inn:ban:jan)
l-sarcolysin phenylalanine mustard
p-di(chloroethyl)amino-l-phenylalanine
melphalan (iarc)
melphalanum (inn-latin)
melphalan (ep monograph)
wr-19813
l-phenylalanine,4-(bis(2-chloroethyl)amino)-
para-di(2-chloroethyl)aminophenylalanine
melphalan usp, 2 mg
p-di-(2-chloroethyl)amino-l-phenylalnine
p-n-bis(2-chloroethyl)amino)phenyl)-l-alanine
melfalano (inn-spanish)
4-bis(2-chloroethyl)amino-l-phenylalanine

Research Excerpts

Overview

Melphalan is an alkylating agent used as part of conditioning prior to pediatric hematopoietic cell transplantation (HCT) It covalently binds to nucleophilic sites in the DNA and effective in the treatment.

ExcerptReferenceRelevance
"Melphalan is an alkylating agent used as part of conditioning prior to pediatric hematopoietic cell transplantation (HCT). "( Population Pharmacokinetics of Melphalan for Pediatric Patients Undergoing Hematopoietic Cell Transplantation.
Apsel Winger, B; Chan, D; Dvorak, CC; Gobburu, JVS; Li, S; Long-Boyle, J; Lu, Y, 2022
)
2.45
"Melphalan is a bifunctional alkylating agent that covalently binds to nucleophilic sites in the DNA and effective in the treatment, but unfortunately has limited therapeutic benefit."( Newly Synthesized Melphalan Analogs Induce DNA Damage and Mitotic Catastrophe in Hematological Malignant Cancer Cells.
Gaipl, US; Ionov, M; Krzeczyński, P; Lubgan, D; Marczak, A; Poczta, A; Rogalska, A, 2022
)
1.78
"Melphalan (Mel) is an antineoplastic widely used in cancer and other diseases. "( Solid-State Formation of a Potential Melphalan Delivery Nanosystem Based on β-Cyclodextrin and Silver Nanoparticles.
Donoso-González, O; Kogan, MJ; Lang, E; Noyong, M; Sierpe, R; Simon, U; Yutronic, N, 2023
)
2.63
"Melphalan (L-PAM) is an ageless drug."( Spotlight on Melphalan Flufenamide: An Up-and-Coming Therapy for the Treatment of Myeloma.
Al-Janazreh, H; Canale, FA; Cutrona, G; D'Arrigo, G; Gentile, M; Martino, EA; Martino, M; Mendicino, F; Morabito, F; Morabito, L; Neri, A; Todoerti, K; Tripepi, G; Vigna, E, 2021
)
1.71
"Melphalan is a bifunctional alkylating agent that elicits its cytotoxic activity by rapidly forming an initial DNA monoadduct, which then produces an inter-strand crosslink. "( A systematic review of inter-individual differences in the DNA repair processes involved in melphalan monoadduct repair in relation to treatment outcomes.
Burns, KE; Helsby, NA; van Kan, M, 2021
)
2.28
"Melphalan is an efficient chemotherapeutic agent that is currently used to treat retinoblastoma (Rb); however, the inherent risk of immunogenicity and the hazardous integration of this drug in healthy cells is inevitable. "( Co-delivery of miR-181a and melphalan by lipid nanoparticles for treatment of seeded retinoblastoma.
Chain, JL; Derbali, RM; Hamel, P; Hardy, P; Superstein, R; Tabatabaei, SN; Yang, C, 2019
)
2.25
"Melphalan is a widely used cytotoxic agent in cancer treatments. "( Analysis of novel melphalan hydrolysis products formed under isolated lung perfusion conditions using liquid chromatography/tandem mass spectrometry.
Boschmans, J; de Bruijn, E; Lemière, F; Van Schil, P, 2013
)
2.17
"Melphalan is an efficacious alternative agent in patients undergoing CRS/HIPEC for aggressive and recurrent peritoneal surface malignancies. "( Melphalan: a promising agent in patients undergoing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy.
Gushchin, V; Jimenez, W; Nieroda, C; Sardi, A; Shankar, S; Sittig, M, 2014
)
3.29
"Melphalan is a highly effective alkylating agent which causes many types of DNA lesions, including DNA base alkylation damage that is repaired by base excision repair (BER)."( Functional analysis of the involvement of apurinic/apyrimidinic endonuclease 1 in the resistance to melphalan in multiple myeloma.
Du, J; Li, M; Li, Z; Wang, D; Wang, G; Xie, J; Yang, S; Zeng, L; Zhang, L; Zhou, L, 2014
)
1.34
"Melphalan is an effective chemotherapeutic agent. "( Alternative treatment for retinoblastoma: Intra-arterial chemotherapy with melphalan.
Aviño, J; Barranco, H; De Freytas, A; Harto-Castaño, M; Martinez-Costa, R, 2015
)
2.09
"Melphalan is an alkylating agent frequently used in an intravenous formulation to treat hematologic malignancies and solid tumors in both adults and children. "( Stability of Melphalan in 0.9% Sodium Chloride Solutions Prepared in Polyvinyl Chloride Bags for Intravenous Injection.
Desmaris, RP; Mercier, L; Paci, A, 2015
)
2.23
"Melphalan is a frequently used chemotherapeutical agent for the treatment of myeloma, breast cancer, ovarian cancer and sarcoma of soft tissue. "( Covalent adducts of melphalan with free amino acids and a model peptide studied by liquid chromatography/tandem mass spectrometry.
Dewaele, D; Lemière, F; Sobott, F, 2016
)
2.2
"Melphalan is a bifunctional alkylating agent that covalently binds to the nucleophilic sites present in DNA. "( Separation and identification of trinucleotide-melphalan adducts from enzymatically digested DNA using HPLC-ESI-MS.
Linscheid, M; Mohamed, D, 2008
)
2.05
"Melphalan is a chemically unstable antineoplastic drug which in the current commercial formulation (Alkeran(®) for Injection) has some limitations with regard to solubility, stability and biocompatibility."( Preclinical comparison of intravenous melphalan pharmacokinetics administered in formulations containing either (SBE)7 m-β-cyclodextrin or a co-solvent system.
Charman, SA; Katneni, K; Koltun, M; McIntosh, MP; Morizzi, J; Shackleford, DM, 2010
)
1.35
"Melphalan is an important cytotoxic drug. "( [The variance of melphalan doses related to kilogram of body weight and the consequences].
Vokurka, S, 2010
)
2.14
"Melphalan is a bifunctional alkylator that creates inter- and intra-strand DNA cross-links."( Melphalan as a treatment for BRCA-related ovarian carcinoma: can you teach an old drug new tricks?
Arseneau, J; Foulkes, WD; Kushner, YB; Osher, DJ, 2011
)
2.53
"Melphalan (MEL) is a chemotherapeutic agent used in breast cancer therapy; however, MEL's side effects limit its clinical applications. "( Resveratrol chemosensitizes breast cancer cells to melphalan by cell cycle arrest.
Casanova, F; da Costa, DC; da Silva, JL; Fialho, E; Quarti, J; Ramos, CA, 2012
)
2.07
"Melphalan is a chemotherapeutic agent that shows increased pharmacological activity with heat."( Hyperthermia modifies pharmacokinetics and tissue distribution of intraperitoneal melphalan in a rat model.
Glehen, O; Mohamed, F; Stuart, OA; Sugarbaker, PH, 2004
)
1.27
"Melphalan is a chemotherapeutic drug that exerts its cytotoxic effect mainly through the formation of DNA adducts. "( Immunohistochemical detection of melphalan-DNA adducts in colon cancer cells in vitro and human colorectal liver tumours in vivo.
Koevoets, C; Kuppen, PJ; Mulder, GJ; Rothbarth, J; Tilby, MJ; Tollenaar, RA; van de Velde, CJ, 2004
)
2.05
"Melphalan is an alkylating substance used as a therapeutic agent; its mutagenicity is related to its ability to produce monoadducts and to form DNA cross-links. "( Melphalan-induced DNA damage in p53(+/-) and wild type mice analysed by the comet assay.
Cinelli, S; Cordelli, E; Lascialfari, A; Pacchierotti, F; Ranaldi, R, 2004
)
3.21
"Melphalan is an alkylating agent, which is commonly used as an antineoplastic drug. "( Melphalan reduces the severity of experimental colitis in mice by blocking tumor necrosis factor-alpha signaling pathway.
Alioshkin, V; Pukhalsky, A; Sabelnikov, A; Shmarina, G, 2007
)
3.23
"As melphalan is a phenylalanine derivative, the pharmacokinetic variability may be determined by genetic polymorphisms in the L-type amino acid transporters LAT1 (SLC7A5) and LAT2 (SLC7A8)."( Genetic polymorphisms in the amino acid transporters LAT1 and LAT2 in relation to the pharmacokinetics and side effects of melphalan.
Brockmöller, J; Heider, U; Hohloch, K; Kaiser, R; Kühne, A; Muhlke, S; Niere, W; Overbeck, T; Schirmer, M; Sezer, O; Trümper, L, 2007
)
1.06
"Melphalan is an alkylating agent approved for the treatment of multiple myeloma and ovarian cancer. "( Melphalan and its role in the management of patients with multiple myeloma.
Avonto, I; Boccadoro, M; Bringhen, S; Falco, P; Gay, F; Morabito, F; Palumbo, A, 2007
)
3.23
"Melphalan is a particularly well suited agent for use with autologous bone marrow rescue and produces response in chemo-resistant tumours."( High dose melphalan with autologous marrow rescue in cancer treatment.
Dady, PJ; Dewar, JM; Forgeson, GV, 1984
)
1.39
"Melphalan (Alkeran) is an alkylating agent commonly used in the treatment of multiple myeloma and other neoplasia. "( Selection and characterization of Chinese hamster ovary cell mutants resistant to melphalan (L-phenylalanine mustard).
Elliott, EM; Ling, V, 1981
)
1.93
"Melphalan is an anti-neoplastic agent formulated for parenteral use as a sterile, non-pyrogenic, freeze-dried powder."( New injectable melphalan formulations utilizing (SBE)(7m)-beta-CD or HP-beta-CD.
Ma, DQ; Rajewski, RA; Stella, VJ, 1999
)
1.38
"Melphalan is a bifunctional alkylating agent that covalently binds with intracellular nucleophilic sites. "( Analysis of melphalan adducts of 2'-deoxynucleotides in calf thymus DNA hydrolysates by capillary high-performance liquid chromatography-electrospray tandem mass spectrometry.
Berneman, Z; Deforce, D; Esmans, EL; Hoes, I; Lemière, F; Van Bockstaele, D; Van den Eeckhout, EG; Van Dongen, W; Vanhoutte, K, 1999
)
2.13
"Melphalan is an effective drug in the treatment of myeloma, but is potentially toxic to progenitor cells."( The effects of prior induction therapy with melphalan on subsequent peripheral blood progenitor cell transplantation for myeloma.
Ahsan, G; Kazmi, MA; Schey, SA, 2001
)
1.29
"Melphalan is a more potent leukemogen than cyclophosphamide or radiotherapy."( Risk of leukemia after chemotherapy and radiation treatment for breast cancer.
Bernstein, L; Boice, JD; Curtis, RE; Flannery, JT; Greenberg, RS; Hoover, RN; Moloney, WC; Schwartz, AG; Stovall, M; Weyer, P, 1992
)
1
"Melphalan (MEL) is an aromatic alkylating agent which is useful for the treatment of a number of human cancers, including myeloma and ovarian cancer. "( Selective enhancement of antitumor activity of N-acetyl melphalan upon conjugation to monoclonal antibodies.
McKenzie, IF; Pietersz, GA; Smyth, MJ, 1987
)
1.96

Effects

Melphalan has been widely used for the treatment of several types of cancers despite its gonadotoxic effects. Melphalan resistance has been considered one of the major obstacles to improve outcomes in multiple myeloma (MM) therapy.

ExcerptReferenceRelevance
"Melphalan has a steep dose-response curve, but the use of high doses results in unacceptable myelosuppression. "( A phase I trial of amifostine (WR-2721) and melphalan in children with refractory cancer.
Adamson, PC; Balis, FM; Belasco, JE; Berg, SL; Blaney, SM; Craig, C; Lange, B; Poplack, DG, 1995
)
2
"CE-melphalan at 2 mg/mL has 24-hour stability at RT and can be used for extended infusion times or may be compounded ahead of time."( Stability of Captisol-enabled versus propylene glycol-based melphalan at room temperature and after refrigeration.
Bashir, Q; Champlin, RE; Ciurea, SO; Gulbis, AM; Kawedia, JD; Liu, X; Qazilbash, MH; Ramchandran, S; Titus, M, 2022
)
1.48
"Melphalan has been widely used for the treatment of several types of cancers despite its gonadotoxic effects."( Melphalan induced germ cell toxicity and dose-dependent effects of β-aminoisobutyric acid in experimental rat model: Role of oxidative stress, inflammation and apoptosis.
Jena, G; Kumar, V; Panghal, A, 2023
)
3.07
"Melphalan resistance has been considered one of the major obstacles to improve outcomes in multiple myeloma (MM) therapy; unfortunately, the mechanistic details of this resistance remain unclear. "( Functional analysis of the involvement of apurinic/apyrimidinic endonuclease 1 in the resistance to melphalan in multiple myeloma.
Du, J; Li, M; Li, Z; Wang, D; Wang, G; Xie, J; Yang, S; Zeng, L; Zhang, L; Zhou, L, 2014
)
2.06
"Melphalan has the strongest, and carboplatin the weakest association with reduction in ERG response amplitudes; but for the most part, these changes are minimal and likely clinically insignificant. "( Electroretinogram monitoring of dose-dependent toxicity after ophthalmic artery chemosurgery in retinoblastoma eyes: six year review.
Abramson, DH; Brodie, SE; Dunkel, IJ; Francis, JH; Gobin, YP; Marr, BP; Riedel, ER, 2014
)
1.85
"Melphalan (L-PAM) has been an integral part of multiple myeloma (MM) treatment as a conditioning regimen before stem cell transplant (SCT). "( The glutathione synthesis inhibitor buthionine sulfoximine synergistically enhanced melphalan activity against preclinical models of multiple myeloma.
Kang, MH; Reynolds, CP; Singh, H; Tagde, A, 2014
)
2.07
"Melphalan has been used in the treatment of various hematologic malignancies for almost 60 years. "( In vitro and in vivo activity of melflufen (J1)in lymphoma.
Delforoush, M; Enblad, G; Gullbo, J; Larsson, R; Strese, S; Wickström, M, 2016
)
1.88
"Melphalan has been a mainstay of multiple myeloma (MM) therapy for many years. "( Bendamustine overcomes resistance to melphalan in myeloma cell lines by inducing cell death through mitotic catastrophe.
Ciavarella, S; Cives, M; Dammacco, F; De Matteo, M; Rizzo, FM; Silvestris, F, 2013
)
2.11
"Melphalan has a steep dose-response curve, but the use of high doses results in unacceptable myelosuppression. "( A phase I trial of amifostine (WR-2721) and melphalan in children with refractory cancer.
Adamson, PC; Balis, FM; Belasco, JE; Berg, SL; Blaney, SM; Craig, C; Lange, B; Poplack, DG, 1995
)
2
"Melphalan has brought the first improvement in the therapy of multiple myeloma at the beginning of the sixties. "( [Treatment of multiple myeloma--melphalan monotherapy after bone marrow transplantation].
Adam, Z; Hájek, R; Král, Z; Krejcí, M; Vásová, I; Vorlícek, J, 1996
)
2.02
"Melphalan has brought the first improvement in the therapy of multiple myeloma at the beginning of the sixties. "( Evolution of multiple myeloma treatment from melphalan monotherapy to bone marrow transplantation.
Adam, Z; Hájek, R; Král, Z; Vásová, I, 1996
)
2
"Melphalan has rarely been used as a single agent for conditioning prior to allogeneic marrow transplantation. "( Melphalan alone prior to allogeneic bone marrow transplantation from HLA-identical sibling donors for hematologic malignancies: alloengraftment with potential preservation of fertility in women.
Horton, C; Mehta, J; Powles, R; Singhal, S; Swansbury, GJ; Treleaven, J, 1996
)
3.18
"Melphalan at 30 mg/m2 has little activity among patients with metastatic colorectal carcinoma."( Phase II trial of intravenous melphalan for metastatic colorectal carcinoma. A Southwest Oncology Group study.
Baker, LH; Coltman, CA; Constanzi, JJ; Goodman, P; Knight, WA; Macdonald, JS; Taylor, SA, 1990
)
1.29
"Melphalan has been reported to be actively transported into tumor cells by two amino acid carrier systems. "( Facilitated transport of melphalan at the rat blood-brain barrier by the large neutral amino acid carrier system.
Greig, NH; Momma, S; Rapoport, SI; Smith, QR; Sweeney, DJ, 1987
)
2.02
"Melphalan has been evaluated against a series of seven childhood rhabdomyosarcomas, each derived from a different patient and maintained in vivo as xenografts in immune-deprived mice. "( Melphalan: a potential new agent in the treatment of childhood rhabdomyosarcoma.
Cook, RL; Houghton, JA; Houghton, PJ; Lutz, PJ, 1985
)
3.15

Actions

Melphalan did not increase the doubling of turbidity that idarubicin hydrochloride shows upon simple dilution. Melphalan showed lower cytotoxicity at short exposure times and high drug concentrations. chlorambucil exhibited higher cytot toxicity at longer exposure times.

ExcerptReferenceRelevance
"Melphalan did not increase the doubling of turbidity that idarubicin hydrochloride shows upon simple dilution."( Physical compatibility of melphalan with selected drugs during simulated Y-site administration.
Martinez, JF; Trissel, LA, 1993
)
1.31
"Melphalan can rarely cause interstitial pneumonitis and fibrosis. "( Melphalan-associated pulmonary toxicity following high-dose therapy with autologous hematopoietic stem cell transplantation.
Akasheh, MS; Freytes, CO; Vesole, DH, 2000
)
3.19
"Melphalan showed lower cytotoxicity at short exposure times and high drug concentrations, while chlorambucil exhibited higher cytotoxicity at longer exposure times."( Concentration and time-dependent inter-relationships for cytotoxicities of nitrogen mustard drugs against lymphoblasts in-vitro.
Ehrsson, H; Ringborg, U; Wallin, I, 1988
)
1

Treatment

Melphalan has been applied intra-arterially by catheterisation of the ophthalmic artery or intravitreally, aiming to reduce systemic side effects of intravenous drug therapy. Melphalan pretreatment or cotreatment with dl1520 enhanced inhibition of proliferation and increased apoptosis by up to 25%. In melphalan-treated cells one can detect both 10kb and 20kb DNA intermediates, indicating that in such cells the gaps present in a replicon are not f.

ExcerptReferenceRelevance
"Melphalan treatment significantly altered all the above-mentioned parameters and the high dose (100 mg/kg) of BAIBA restored melphalan-induced toxicity in a significant manner by exerting antioxidant, anti-inflammatory and antiapoptotic effects."( Melphalan induced germ cell toxicity and dose-dependent effects of β-aminoisobutyric acid in experimental rat model: Role of oxidative stress, inflammation and apoptosis.
Jena, G; Kumar, V; Panghal, A, 2023
)
3.07
"Melphalan treatment of hiPSC-CMs induced oxidative stress, caused Ca"( Melphalan induces cardiotoxicity through oxidative stress in cardiomyocytes derived from human induced pluripotent stem cells.
Castellino, SM; Du, Y; Fischbach, P; Fu, H; Li, D; Liu, R; Mandawat, A; Maxwell, JT; Rampoldi, A; Sun, F; Wu, R; Xu, C, 2020
)
3.44
"Melphalan, as a treatment for retinoblastoma, has been applied intra-arterially by catheterisation of the ophthalmic artery or intravitreally, aiming to reduce systemic side effects of intravenous drug therapy. "( Investigating short-term toxicity of melphalan in a model of an isolated and superfused bovine retina.
Aisenbrey, S; Bartz-Schmidt, KU; Hagemann, U; Hofmann, K; Januschowski, K; Krupp, C; Mueller, S; Schnichels, S; Spitzer, MS, 2016
)
2.15
"Melphalan pretreatment or cotreatment with dl1520 enhanced inhibition of proliferation by dl1520 by up to 60% and increased apoptosis by up to 25%."( Synergistic cytotoxicity against human tumor cell lines by oncolytic adenovirus dl1520 (ONYX-015) and melphalan.
Ferguson, PJ; Figueredo, R; Koropatnick, J; Sykelyk, A,
)
1.07
"Melphalan-treated CCRF-CEM leukaemia cells were analysed by the trapped-in-agarose DNA immunostaining (TARDIS) method using fluorescein immunofluorescence and Hoechst dye-DNA fluorescence."( Quantification of DNA adducts in individual cells by immunofluorescence: effects of variation in DNA conformation.
Frank, AJ; Tilby, MJ, 2003
)
1.04
"In melphalan-treated cells one can detect both 10 kb and 20 kb DNA intermediates, indicating that in such cells the gaps present in a replicon are not filled at the same time which allows the detection of a molecule which is formed by the joining of two 10 kb DNA intermediates."( Two discrete DNA replication intermediates are formed in melphalan-treated cells.
Lönn, S; Lönn, U, 1983
)
1.03
"For melphalan, neither pretreatment agent produced any change in tumor response."( Effect of misonidazole or metronidazole pretreatment on the response of the RIF-1 mouse sarcoma to melphalan, cyclophosphamide, chlorambucil and CCNU.
Twentyman, P; Workman, P, 1982
)
0.96
"The melphalan treated patients showed a significantly higher overall objective response frequency (59%), according to Myeloma Task Force criteria, when compared to those treated with BCNU (40%) or CCNU (42%)."( Comparison of oral melphalan, CCNU, and BCNU with and without vincristine and prednisone in the treatment of multiple myeloma. Cancer and Leukemia Group B experience.
Brunner, K; Cooper, MR; Cornwell, GG; Cuttner, J; Glowienka, LP; Haurani, FI; Henderson, E; Kochwa, S; Kyle, RA; McIntyre, OR; Pajak, TF; Rafla, S; Silver, RT, 1982
)
1.07
"Melphalan treatment alone improved the median survival to 31 days for animals with localized myeloma and 34 days in animals with disseminated disease."( Radiopharmaceutical therapy of 5T33 murine myeloma by sequential treatment with samarium-153 ethylenediaminetetramethylene phosphonate, melphalan, and bone marrow transplantation.
Berger, JD; Claringbold, PG; Glancy, RJ; Manning, LS; O'Donoghue, HL; Turner, JH, 1993
)
1.21
"Melphalan treatment should be avoided in patients who are candidates for high-dose chemotherapy."( Successful mobilization of peripheral blood stem cells in heavily pretreated myeloma patients with G-CSF alone.
Gutensohn, K; Hassan, HT; Hummel, K; Kröger, N; Krüger, W; Lölliger, C; Renges, H; Zander, AR; Zeller, W, 1998
)
1.02
"When melphalan treatment was used to produce tumor-bearing mice with an intact delayed hypersensitivity response but devoid of a significant antibody response, the drug-treated animals were found to have high levels of ADCC effector cells in situ."( Immunologic factors influencing the intra-tumor localization of ADCC effector cells.
Haskill, S; Parthenais, E, 1978
)
0.71
"Melphalan treatment resulted in arrest of cells in late S- and G2-phases in a population of unsynchronized cells."( Interphase cell death as related to the cell cycle of melphalan-treated human myeloma cells.
Fernberg, JO; Lewensohn, R; Skog, S, 1991
)
1.25
"In melphalan-treated patients where LCLs were established serially, the melphalan Do increased after further melphalan treatment in vivo and decreased when no further treatment was given."( Melphalan-resistant lymphoblastoid cell lines established from patients with ovarian cancer treated with cross-linking agents.
Daunter, B; Khoo, SK; Maynard, K; Musk, P; Parsons, PG, 1985
)
2.23
"Melphalan treatment of the cells produced an increased secretion of IgG and IgM, whereas adriamycin did not."( Melphalan treatment of human peripheral T cells promotes Ig production by B cells in vitro.
Baral, E; Blomgren, H; Rotstein, S; Virving, L; von Stedingk, LV; Wasserman, J, 1985
)
2.43
"Treatment with melphalan-based therapy significantly induced the expression of PD-1 on CD8"( Anti-PD-1 checkpoint blockade improves the efficacy of a melphalan-based therapy in experimental melanoma.
Johansson, J; Kiffin, R; Martner, A; Olofsson Bagge, R, 2021
)
1.22
"Treatment with melphalan is currently standard of care for younger and fit patients when followed by hematopoietic stem cell transplantation (HSCT), and in transplant ineligible patients when used in combination regimens."( Polymorphism in ANRIL is associated with relapse in patients with multiple myeloma after autologous stem cell transplant.
Cho, YK; Hofmeister, CC; Lamprecht, M; Li, J; Phelps, MA; Pichiorri, F; Poi, MJ; Sborov, DW; VanGundy, Z, 2017
)
0.79
"Treatment with melphalan plus the proteasome inhibitor MG132 reduced global protein degradation for MM cells to roughly 60% of that seen without drugs, but the reduction was approximately three times greater for CCL-156 cells."( Evidence that aberrant protein metabolism contributes to chemoresistance in multiple myeloma cells.
Burgis, NE; Lewis, BS; Sipes, RK; Xia, X, 2012
)
0.72
"Treatment with melphalan resulted in 2 (20%) complete responses and 1 (10%) partial response (response rate, 30%; 95% CI 8%, 65%)."( Melphalan for the treatment of patients with recurrent epithelial ovarian cancer.
Davis-Perry, S; Hernandez, E; Houck, KL; Shank, R, 2003
)
2.1
"Treatment with melphalan (6 mg) and prednisolone (35 mg) for 4 days every 6 weeks decreased the fever and alleviated his symptoms."( Primary amyloidosis with pulmonary involvement which presented exudative pleural effusion and high fever.
Fujimoto, N; Ito, M; Kosaka, H; Masuoka, H; Nakano, T; Ota, S, 2003
)
0.66
"Treatment with melphalan and prednisolone was initiated without beneficial response."( [Granulocyte-colony stimulating factor-producing myeloma with clinical manifestations mimicking chronic neutrophilic leukemia].
Kusaba, N; Mishima, K; Ohkubo, F; Okamura, T; Sata, M; Shimamastu, K; Yoshida, H, 2004
)
0.66
"Treatment with melphalan, a classic DNA-damaging agent, led to the induction of the DNA damage checkpoint and growth arrest in the G2 phase of the cell cycle."( Cross-talk between DNA damage and cell survival checkpoints during G2 and mitosis: pharmacologic implications.
Bozko, P; Larsen, AK; Sabisz, M; Skladanowski, A, 2005
)
0.67
"Treatment with melphalan and dexamethasone allowed stabilization during more than six months."( [Original case report of amyloidosis with pleural involvement: the role of echocardiography in the diagnosis].
Billotet, C; Roubille, C; Roubille, F, 2006
)
0.67
"Treatment with melphalan was as effective as ovariectomy, but the combination of melphalan with ovariectomy was no more effective than either treatment alone."( Enhanced cytostatic effectiveness of aniline mustard against 7,12-dimethylbenz[a]anthracene-induced rat mammary tumors during regression in response to ovariectomy.
Benckhuysen, C; Ter Hart, HG; Van Dijk, PJ,
)
0.47
"Treatment with melphalan or cyclophosphamide resulted in a decrease in the serum IgE level and in the level of Bence Jones protein in the urine."( Immunoglobulin E (IgE) multiple myeloma: a case report and review of the literature.
Asakawa, H; Endo, T; Kikuchi, K; Munakata, J; Nomura, T; Okumura, H; Otake, M, 1981
)
0.6
"Treatment with melphalan, paclitaxel, perfosfamide or tamoxifen failed to enrich for mismatch repair-deficient cells, and no change in sensitivity to these agents was detected in the clonogenic assays."( The effect of different chemotherapeutic agents on the enrichment of DNA mismatch repair-deficient tumour cells.
Aebi, S; Fink, D; Haas, M; Howell, SB; Kim, HK; Nebel, S; Norris, PS, 1998
)
0.64
"Treatment with melphalan and prednisolone was effective against the myeloma as well as the scleredema and acanthosis nigricans."( [Scleredema, acanthosis nigricans and IgA/Kappa multiple myeloma].
Bernardo, A; Farinha, F; Massa, A; Ribeiro, P; Valente, L; Velho, GC, 1997
)
0.64
"Treatment with melphalan and prednisone results in an objective response in 50%-60% of patients."( Management of patients with multiple myeloma: emphasizing the role of high-dose therapy.
Kyle, RA, 2001
)
0.65
"Treatment with melphalan and prednisone resulted in great improvement of cutaneous lesions and paraproteinemia remission."( Necrobiotic xanthogranuloma with lambda paraproteinemia: case report of successful treatment with melphalan and prednisone.
Criado, PR; Lopes, LF; Pegas, JR; Ramos, CF; Tebcherani, AJ; Valente, NY; Vasconcellos, C, 2002
)
0.88
"The treatment with melphalan was discontinued and the spread of the KS was arrested by irradiation and bleomycin."( Multiple myeloma associated with Kaposi sarcoma.
Djaldetti, M; Gafter, U; Kende, L; Lask, D; Mandel, EM; Weiss, S, 1977
)
0.58
"Treatment with melphalan and steroids resulted in a three year remission."( [Alpha-chain disease presenting as malabsorption syndrome with exudative enteropathy (author's transl)].
Havemann, K; Kalbfleisch, H; Martini, GA; Menge, H; Riecken, EO; Roth, S; Sodomann, CP, 1976
)
0.6
"Treatment with melphalan was associated with histologic evidence of reversible gastrointestinal toxicity, reversible myelosuppression, and histologic evidence of acute renal tubular necrosis, with no differences being observed between mice that had been pretreated with BSO and those that had been pretreated with vehicle."( Melphalan-induced toxicity in nude mice following pretreatment with buthionine sulfoximine.
Bigner, DD; Friedman, HS; Griffith, OW; McMahon, DP; Postels, DG; Skapek, SX; VanDellen, AF, 1991
)
2.06
"Treatment with melphalan had no effect on already induced suppressor T cells as shown by incubation of spleen cells with melphalan (0.15-5 micrograms/1 X 10(7) cells) after incubation with ConA."( Effect of melphalan in vitro on induction of murine suppressor T cells by ConA.
Ben-Efraim, S; Ophir, R, 1985
)
1.01
"Treatment with melphalan and prednisolone resulted in remission of both myeloma and myopathy."( Amyloid myopathy and myeloma: response to treatment.
Sheehan-Dare, RA; Simmons, AV, 1987
)
0.61
"Treatment with melphalan, cyclophosphamide, and prednisone was associated with long-term survival (median, 540 days)."( Prognostic factors for multiple myeloma in the dog.
Hurvitz, AI; Leifer, CE; MacEwen, EG; Matus, RE, 1986
)
0.61

Toxicity

Intravitreous injection of melphalan may result in toxic effects on the anterior segment of the eye, in addition to retinal abnormalities. This appears to be more common in the meridian of the injection where the drug concentration is highest. The pharmacokinetics and clinical efficacy of ILI from various centres are summarised.

ExcerptReferenceRelevance
" Mild or moderate limb toxicity is usual, but severe toxic reactions in the limb sometimes occur."( Isolated limb perfusion for melanoma: effectiveness and toxicity of cisplatin compared with that of melphalan and other drugs.
Gianoutsos, MP; Thompson, JF,
)
0.35
" One of the peptides, L-propyl-m-sarcolysyl-L-p-fluorophenylalanine (PSF), is highly toxic to melanoma cells."( Cytotoxicity and DNA cross-linking induced by peptide conjugated m-L-sarcolysin in human melanoma cells.
Hansson, J; Lewensohn, R; Ringborg, U,
)
0.13
" In a previous investigation we found that Peptichemio was less toxic to human lymphoblasts than m-L-sarcolysin."( Increased toxicity and DNA cross-linking by peptide bound m-L-sarcolysin (Peptichemio) as compared to melphalan and m-L-sarcolysin in human melanoma cell lines.
Ehrsson, H; Hansson, J; Lewensohn, R; Ringborg, U,
)
0.35
"4-fold more toxic toward bright cells."( Classification of antineoplastic treatments by their differential toxicity toward putative oxygenated and hypoxic tumor subpopulations in vivo in the FSaIIC murine fibrosarcoma.
al-Achi, A; Herman, TS; Holden, SA; Teicher, BA, 1990
)
0.28
" Our studies with one such agent, the vasodilator hydralazine, have clearly demonstrated that it can increase the tumor cytotoxicity of drugs which are known to be more toxic under hypoxic conditions."( Potentiation of the tumor cytotoxicity of melphalan by vasodilating drugs.
Acker, B; Chaplin, DJ; Olive, PL, 1989
)
0.54
" When the dose of methyl-CCNU was further increased to 40 mg kg-1 toxic death occurred, which was, however, significantly reduced by 'priming' with the low dose given."( Priming with low doses of methyl-CCNU reduce the toxicity of high doses of methyl-CCNU and melphalan, and increase the lifespan of mice implanted with Lewis lung carcinoma.
Livnat, I; Perk, K; Zimber, A, 1988
)
0.5
"05) more toxic than melphalan."( In vitro comparative studies of the myelotoxicity and antitumor activity of 6-[bis-(2-chloroethyl)-amino]-6-deoxy-D-glucose versus melphalan utilizing the CFU-C and HTSCA assays.
Dufour, M; Isabel, G; Lazarus, P; Panasci, LC, 1986
)
0.8
" While almost all patients experienced toxic reactions during the therapy, only 3%--4% of recipients of melphalan (L-PAM; P) and 4%--5% of recipients of L-PAM + 5-FU(F)(PF) failed to complete 2 years of therapy because of toxicity."( Acute toxicity during adjuvant chemotherapy for breast cancer: the National Surgical Adjuvant Breast and Bowel Project (NSABP) experience from 1717 patients receiving single and multiple agents.
Fisher, B; Foster, R; Glass, A; Lerner, H; Margolese, R; Plotkin, D; Redmond, C; Shibata, H; Wieand, HS; Wolmark, N; Wolter, J,
)
0.35
" These results suggest the existence of a novel zinc-inducible mechanism which protects cells against the toxic effects of alkylating agents."( Zinc-induced resistance to alkylating agent toxicity.
Enger, MD; Griffith, JK; Hildebrand, CE; Tobey, RA, 1982
)
0.26
"To determine if a lower perfusion flow rate would reduce leakage and consequently toxic effects."( Systemic leakage and side effects of tumor necrosis factor alpha administered via isolated limb perfusion can be manipulated by flow rate adjustment.
Abu-Abid, S; Aderka, D; Bar-On, J; Gutman, M; Halpern, P; Kudlik, N; Lev, D; Rudich, V; Setton, A; Sorkin, P, 1995
)
0.29
" Consequently, the systemic hemodynamic, metabolic, and hematologic toxic effects are virtually abolished."( Systemic leakage and side effects of tumor necrosis factor alpha administered via isolated limb perfusion can be manipulated by flow rate adjustment.
Abu-Abid, S; Aderka, D; Bar-On, J; Gutman, M; Halpern, P; Kudlik, N; Lev, D; Rudich, V; Setton, A; Sorkin, P, 1995
)
0.29
" Interestingly, these 2 conjugates, differing only in melphalan position, showed completely different cytotoxicity in terms of IC50 values, Pep 1 being 24 times more toxic to all cells; but the 2 were equally specific to melanoma cells."( Receptor-mediated cytotoxicity of alpha-MSH fragments containing melphalan in a human melanoma cell line.
Botyánszki, J; Ghanem, G; Libert, A; Loir, B; Medzihradszky, K; Morandini, R; Süli-Vargha, H, 1994
)
0.77
" Age over 60 years, female sex and more severe acute regional toxic reactions were correlated with an increased incidence of systemic side-effects."( Systemic toxicity after isolated limb perfusion with melphalan for melanoma.
Eggermont, AM; Klaase, JM; Kroon, BB; Nieweg, OE; Sonneveld, EJ; van Dongen, JA; van Geel, BN; Vrouenraets, BC, 1996
)
0.54
" In summary, the DexaBEAM/G-CSF/CBV strategy appears to be safe and effective for salvage treatment in patients with poor risk malignant lymphomas."( Peripheral blood progenitor cell mobilization with Dexa-Beam/G-CSF, ether lipid purging, and autologous transplantation after high-dose CBV treatment: a safe and effective regimen in patients with poor risk malignant lymphomas.
Berdel, WE; Hoppe, B; Knauf, WU; Koenigsmann, MP; Notter, M; Oberberg, D; Reufi, B; Thiel, E, 1996
)
0.29
" Within the NHL group, 21 patients were in 2nd or subsequent complete remission (CR) at transplant, 34 had sensitive disease and 11 resistant disease; 46 patients were transplanted in 1st CR due to the presence of > or = 2 adverse prognostic features at diagnosis or to a slow CR."( BEAM chemotherapy followed by autologous stem cell support in lymphoma patients: analysis of efficacy, toxicity and prognostic factors.
Caballero, MD; Corral, M; del Cañizo, MC; García-Sanz, R; Gonzalez, M; Heras, I; Jean-Paul, E; León, A; Moraleda, JM; Rifon, J; Rocha, E; Rubio, V; San Miguel, JF; Vázquez, L; Vidriales, B, 1997
)
0.3
" Toxicity information for IFN had been collected using patient-completed diaries so the actual duration of each adverse event could be determined."( Quality-adjusted time without symptoms or toxicity analysis of interferon maintenance in multiple myeloma.
Browman, G; Cole, B; James, K; Johnston, D; Li, T; Pater, J; Sugano, D; Zee, B, 1998
)
0.3
"The optimal toxic reaction of the normal tissues in perfused limbs after isolated limb perfusion (ILP) is unknown."( Relation between limb toxicity and treatment outcomes after isolated limb perfusion for recurrent melanoma.
Eggermont, AM; Hart, GA; Klaase, JM; Kroon, BB; Nieweg, OE; van Geel, BN; Vrouenraets, BC, 1999
)
0.3
"These data suggest that, in our cohort of patients, the combination of total-body irradiation and melphalan is safe and associated with good antileukemia activity, making ABMT an appealing alternative for postremission therapy in children with acute myeloid leukemia in first CR."( Total-body irradiation and melphalan is a safe and effective conditioning regimen for autologous bone marrow transplantation in children with acute myeloid leukemia in first remission. The Italian Association for Pediatric Hematology and Oncology-Bone Mar
Bonetti, F; Cesaro, S; De Stefano, P; Fagioli, F; Giorgiani, G; Lanino, E; Locatelli, F; Messina, C; Montagna, D; Pession, A; Prete, A; Rondelli, R; Santoro, N; Zecca, M, 1999
)
0.82
" Toxic mortality prior to day + 100 was 29% in the L-PAM group and 9% in the non-L-PAM group of patients."( Allograft with unrelated donor accentuates the gastrointestinal toxicity associated with high-dose melphalan and total body irradiation preparative for bone marrow transplantation in children.
Hovi, L; Saarinen-Pihkala, U; Taskinen, M; Vettenranta, K, 2000
)
0.52
" The results of this study demonstrate that the use of CD34(+) cells is safe and has no adverse effects either with respect to haematological, immune reconstitution or to infections after HDT."( High-dose therapy in patients with Hodgkin's disease: the use of selected CD34(+) cells is as safe as unmanipulated peripheral blood progenitor cells.
Blystad, AK; Delabie, J; Holte, H; Kvalheim, G; Kvaløy, S; Smeland, E, 2001
)
0.31
"O6-Benzylguanine (BG) inactivates O6-alkylguanine-DNA alkyltransferase (AGT), resulting in an increase in the sensitivity of cells to the toxic effects of O6-alkylating agents."( Effect of O6-benzylguanine on nitrogen mustard-induced toxicity, apoptosis, and mutagenicity in Chinese hamster ovary cells.
Cai, Y; Chung, AB; Dolan, ME; Ludeman, SM; Wilson, LR, 2001
)
0.31
" BEAM allogeneic transplantation with early reduction in immunosuppression is safe (no treatment-related deaths) and effective in advanced Hodgkin's disease where autografts have failed."( BEAM allogeneic transplantation for patients with Hodgkin's disease who relapse after autologous transplantation is safe and effective.
Cooney, JP; Parthasarathy, M; Stiff, PJ; Toor, AA, 2003
)
0.32
" While BEAC and BEAM appears to have equal antitumour efficacy in patients with NHL, BEAM seems to be more toxic to the gastrointestinal tract."( BEAC or BEAM for high-dose therapy in patients with non-Hodgkin's lymphoma? A single centre analysis on toxicity and efficacy.
Jantunen, E; Kuittinen, T; Nousiainen, T, 2003
)
0.32
" To determine a safe and tolerable dose for efficacy studies the acute toxicity following intravenous injection in the tail vein was monitored using a 14-day schedule with up to four doses."( Antitumor efficacy and acute toxicity of the novel dipeptide melphalanyl-p-L-fluorophenylalanine ethyl ester (J1) in vivo.
Bashir-Hassan, S; De La Torre, M; Ehrsson, H; Gullbo, J; Larsson, R; Lewensohn, R; Lindhagen, E; Luthman, K; Nygren, P; Tullberg, M, 2004
)
0.56
" These results suggest that CD34 selection of reduced-intensity PBSC allografts may cause adverse effects upon specific antimicrobial immunity which can lead to fatal infections, particularly in high-risk patients."( Reduced-intensity conditioning followed by allografting of CD34-selected stem cells and < or =10(6)/kg T cells may have an adverse effect on transplant-related mortality.
Hartwig, UF; Herr, W; Huber, C; Kolbe, K; Kreiter, S; Meyer, RG; Schneider, PM; Ullmann, AJ; Wehler, T; Winkelmann, N, 2005
)
0.33
"5 million adverse drug reaction (ADR) reports for 8620 drugs/biologics that are listed for 1191 Coding Symbols for Thesaurus of Adverse Reaction (COSTAR) terms of adverse effects."( Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
Benz, RD; Contrera, JF; Kruhlak, NL; Matthews, EJ; Weaver, JL, 2004
)
0.32
" We conclude that MEL200 is a safe and efficacious treatment in selected elderly myeloma patients."( High-dose melphalan (200 mg/m2) supported by autologous stem cell transplantation is safe and effective in elderly (>or=65 years) myeloma patients: comparison with younger patients treated on the same protocol.
Jantunen, E; Kuittinen, T; Lehtonen, P; Mahlamäki, E; Nousiainen, T; Penttilä, K, 2006
)
0.74
" When irradiated cells residing in G2 phase are exposed to very low doses of cisplatin at a toxic dose of 5%."( Inhibition of homologous recombination repair with Pentoxifylline targets G2 cells generated by radiotherapy and induces major enhancements of the toxicity of cisplatin and melphalan given after irradiation.
Bohm, L, 2006
)
0.53
" Specific grade 3/4 haematological (3%) or cardiac adverse events occurred infrequently."( Efficacy and safety of melphalan, arsenic trioxide and ascorbic acid combination therapy in patients with relapsed or refractory multiple myeloma: a prospective, multicentre, phase II, single-arm study.
Berenson, JR; Bessudo, A; Boccia, R; Bozdech, M; Ferretti, D; Flam, M; Jilani, S; Louie, R; Lutzky, J; Moss, R; Patel, R; Russell, K; Siegel, D; Stadtmauer, E; Steis, R; Swift, RA; Talisman Pomeroy, J; Volk, J; Wong, SF; Yeh, HS, 2006
)
0.64
" These cases demonstrate that it is feasible and safe to perform HSCT in pediatric patients with low nGFR using melphalan- and thiotepa-based preparative therapy."( Autologous hematopoietic stem cell transplant with melphalan and thiotepa is safe and feasible in pediatric patients with low normalized glomerular filtration rate.
Gross, TG; Grovas, A; Klopfenstein, K; Rosselet, R; Termuhlen, AM, 2006
)
0.8
" HDT and autologous transplant is safe and feasible in elderly myeloma patients."( Autologous stem cell transplantation is safe and feasible in elderly patients with multiple myeloma.
Alousi, AM; Champlin, RE; Couriel, DR; De Lima, M; Flosser, T; Giralt, SA; Hosing, C; Kebriaei, P; Mendoza, F; Popat, U; Qazilbash, MH; Qureshi, SR; Saliba, RM; Wang, M; Weber, DM, 2007
)
0.34
" Genetic variation in 4F2hc, LAT1, and LAT2 does not appear to be a major cause of inter-individual variability in pharmacokinetics and of adverse reactions to melphalan."( Genetic polymorphisms in the amino acid transporters LAT1 and LAT2 in relation to the pharmacokinetics and side effects of melphalan.
Brockmöller, J; Heider, U; Hohloch, K; Kaiser, R; Kühne, A; Muhlke, S; Niere, W; Overbeck, T; Schirmer, M; Sezer, O; Trümper, L, 2007
)
0.74
" In addition, we studied glutathione S-transferase GSTM1, GSTT1, and GSTP1 polymorphisms in relation to adverse events."( Population pharmacokinetics of melphalan and glutathione S-transferase polymorphisms in relation to side effects.
Brockmöller, J; Heider, U; Hohloch, K; Kaiser, R; Kühne, A; Meineke, I; Muhlke, S; Niere, W; Overbeck, T; Sezer, O; Trümper, L, 2008
)
0.63
" The most common grade 3/4 hematologic adverse events (AEs) were neutropenia (31%/0%), thrombocytopenia (25%/2%), and anemia (13%/0%)."( Safety and efficacy of bortezomib and melphalan combination in patients with relapsed or refractory multiple myeloma: updated results of a phase 1/2 study after longer follow-up.
Berenson, JR; Hilger, J; Lee, SP; Mapes, R; Morrison, B; Nassir, Y; Swift, R; Vescio, RA; Wilson, J; Yang, HH; Yellin, O, 2008
)
0.62
"ILI is a safe alternative to the more invasive and laborious ILP technique to treat melanoma confined to a limb."( Factors predictive of acute regional toxicity after isolated limb infusion with melphalan and actinomycin D in melanoma patients.
Kam, PC; Kroon, HM; Moncrieff, M; Thompson, JF, 2009
)
0.58
" In conclusion, bortezomib-based regimens are safe and effective and should be considered as appropriate treatment options for MM patients with any degree of RI."( Safety and efficacy of bortezomib-based regimens for multiple myeloma patients with renal impairment: a retrospective study of Italian Myeloma Network GIMEMA.
Baldini, L; Boccadoro, M; Bringhen, S; Callea, V; Casulli, AF; Catalano, L; Cavo, M; Ciolli, S; Di Raimondo, F; Galimberti, S; Gentile, M; Mannina, D; Mele, G; Morabito, F; Musto, P; Offidani, M; Palmieri, S; Palumbo, A; Petrucci, MT; Pinotti, G; Piro, E; Tosi, P, 2010
)
0.36
" On the other hand, most of the compounds afforded clear evidence of being far less toxic towards human HGF gingival fibroblasts, HPC pulp cells and HPLF periodontal ligament fibroblasts which are non-malignant cells."( Cytotoxic 2-benzylidene-6-(nitrobenzylidene)cyclohexanones which display substantially greater toxicity for neoplasms than non-malignant cells.
Chu, Q; Das, U; Dimmock, JR; Doroudi, A; Inci Gul, H; Kawase, M; Pati, HN; Sakagami, H; Stables, JP, 2010
)
0.36
" Thalidomide, in combination with MP, is associated with adverse events (AEs) including peripheral neuropathy and venous thromboembolism."( Consensus guidelines for the optimal management of adverse events in newly diagnosed, transplant-ineligible patients receiving melphalan and prednisone in combination with thalidomide (MPT) for the treatment of multiple myeloma.
Bladé, J; Davies, F; Delforge, M; Facon, T; Garcia Sanz, R; Kropff, M; Leal da Costa, F; Moreau, P; Morgan, G; Palumbo, A; Schey, S, 2010
)
0.57
" The pharmacokinetics of melphalan and the clinical efficacy and adverse effects of ILI from various centres are summarised."( Isolated limb infusion with melphalan and actinomycin D in melanoma patients: factors predictive of acute regional toxicity.
Kam, PC; Thompson, JF, 2010
)
0.96
" Nonhematologic grade 3/4 adverse events were reported in 35% of once-weekly patients and 51% of twice-weekly patients (P = ."( Efficacy and safety of once-weekly bortezomib in multiple myeloma patients.
Benevolo, G; Boccadoro, M; Bringhen, S; Callea, V; Cangialosi, C; Cavalli, M; Cavo, M; De Rosa, L; Evangelista, A; Falcone, AP; Gaidano, G; Gentili, S; Genuardi, M; Grasso, M; Guglielmelli, T; Larocca, A; Levi, A; Liberati, AM; Musto, P; Nozzoli, C; Palumbo, A; Patriarca, F; Ria, R; Rizzo, V; Rossi, D, 2010
)
0.36
" However, numerous adverse effects limited their use."( Optimization of the N-lost drugs melphalan and bendamustine: synthesis and cytotoxicity of a new set of dendrimer-drug conjugates as tumor therapeutic agents.
Gust, R; Scheffler, H; Scutaru, AM; Wenzel, M; Wolber, G, 2010
)
0.64
"Dose reduction and shortening of duration of perfusion in isolated limb perfusion with TNF-α and Melphalan (TM-ILP) are associated with less systemic toxicity and seem to be safe and effective on short-term."( TNF dose reduction and shortening of duration of isolated limb perfusion for locally advanced soft tissue sarcoma of the extremities is safe and effective in terms of long-term patient outcome.
Bastiaannet, E; Hoekstra, HJ; Hoven-Gondrie, ML; Suurmeijer, AJ; van Ginkel, RJ, 2011
)
0.59
"Dose reduction and shorter duration of TM-ILP seem to be safe and effective regarding long-term patient outcome, as 5-year local control rates and (limb)-survival are not compromised."( TNF dose reduction and shortening of duration of isolated limb perfusion for locally advanced soft tissue sarcoma of the extremities is safe and effective in terms of long-term patient outcome.
Bastiaannet, E; Hoekstra, HJ; Hoven-Gondrie, ML; Suurmeijer, AJ; van Ginkel, RJ, 2011
)
0.37
" In conclusion, the new BeEAM regimen is safe and effective for heavily pretreated lymphoma patients."( BeEAM (bendamustine, etoposide, cytarabine, melphalan) before autologous stem cell transplantation is safe and effective for resistant/relapsed lymphoma patients.
Caballero, MD; Capria, S; Cuberli, F; Falcioni, S; Ferrara, F; Galieni, P; Gaudio, F; Gherlinzoni, F; Giardini, C; Gobbi, M; Isidori, A; Malerba, L; Meloni, G; Ocio, EM; Rocchi, M; Santoro, A; Sarina, B; Specchia, G; Stefani, PM; Visani, G, 2011
)
0.63
" In both arms, greater rates of severe hematologic adverse events were associated with RI (eGFR < 50 mL/min), however, therapy discontinuation rates were unaffected."( Safety and efficacy of bortezomib-melphalan-prednisone-thalidomide followed by bortezomib-thalidomide maintenance (VMPT-VT) versus bortezomib-melphalan-prednisone (VMP) in untreated multiple myeloma patients with renal impairment.
Baldini, L; Benevolo, G; Boccadoro, M; Bringhen, S; Cascavilla, N; Cavo, M; Di Raimondo, F; Gentile, M; Grasso, M; Guglielmelli, T; Majolino, I; Marasca, R; Mazzone, C; Montefusco, V; Morabito, F; Musolino, C; Musto, P; Nozzoli, C; Offidani, M; Palumbo, A; Patriarca, F; Petrucci, MT; Ria, R; Rossi, D; Vincelli, I, 2011
)
0.65
"This technique is potentially safe and effective at a low cost and may play a promising role, especially in the treatment of recurrent and/or resistant vitreous disease in retinoblastoma, as an alternative to enucleation and/or external beam radiotherapy."( Profiling safety of intravitreal injections for retinoblastoma using an anti-reflux procedure and sterilisation of the needle track.
Balmer, A; Beck-Popovic, M; Gaillard, MC; Moulin, AP; Munier, FL; Soliman, S, 2012
)
0.38
" Unfortunately, recent clinical reports associate adverse vascular toxicities with SSIOAC using melphalan, the most commonly used chemotherapeutic."( Intra-ophthalmic artery chemotherapy triggers vascular toxicity through endothelial cell inflammation and leukostasis.
Haik, BG; Jackson, JS; Johnson, D; Mandrell, TD; Soderland, C; Steinle, JJ; Stewart, CF; Thompson, KE; Toutounchian, J; Wang, F; Williams, JS; Wilson, MW; Yates, CR; Zhang, Q, 2012
)
0.6
" Palifermin has permitted safe dose escalation of melphalan up to 180 mg/m(2) in patients with RI."( Melphalan 180 mg/m2 can be safely administered as conditioning regimen before an autologous stem cell transplantation (ASCT) in multiple myeloma patients with creatinine clearance 60 mL/min/1.73 m2 or lower with use of palifermin for cytoprotection: resul
Abidi, MH; Abrams, J; Agarwal, R; Al-Kadhimi, Z; Ayash, L; Cronin, S; Deol, A; Lum, L; Ratanatharathorn, V; Uberti, J; Ventimiglia, M; Zonder, J, 2012
)
2.07
" In an attempt to design an efficacious and safe prehematopoietic stem cell transplantation conditioning regimen, we investigated the cytotoxicity of the combination of busulfan (B), melphalan (M), and gemcitabine (G) in lymphoma cell lines in the absence or presence of drugs that induce epigenetic changes."( Epigenetic modifiers enhance the synergistic cytotoxicity of combined nucleoside analog-DNA alkylating agents in lymphoma cell lines.
Andersson, BS; Champlin, RE; Li, Y; Murray, D; Nieto, Y; Valdez, BC; Wang, G, 2012
)
0.57
" (4) No fever, septicemia and other systemic toxic effects occurred."( [Analysis on the safety of ophthalmic artery cannulation for intra-arterial chemotherapy in 42 patients with intraocular stage retinoblastoma].
Li, YS; Liu, HY; Liu, QL; Mao, GS; Miao, LX; Sun, YF; Wang, J; Wang, XL; Wang, YF; Yang, XJ; Yuan, HL; Zhao, F, 2012
)
0.38
"Ophthalmic artery cannulation for IAC is a safe and effective method in treating intraocular stage retinoblastoma."( [Analysis on the safety of ophthalmic artery cannulation for intra-arterial chemotherapy in 42 patients with intraocular stage retinoblastoma].
Li, YS; Liu, HY; Liu, QL; Mao, GS; Miao, LX; Sun, YF; Wang, J; Wang, XL; Wang, YF; Yang, XJ; Yuan, HL; Zhao, F, 2012
)
0.38
" We conclude that FBM-A is an effective and safe conditioning regimen for adults up to age 69 with hematologic malignancies who have low-, intermediate-, or high-risk scores according to the DRI."( Reduced toxicity conditioning and allogeneic stem cell transplantation in adults using fludarabine, carmustine, melphalan, and antithymocyte globulin: outcomes depend on disease risk index but not age, comorbidity score, donor type, or human leukocyte ant
Adams, RH; Betcher, JA; Dueck, AC; Fauble, VD; Khera, N; Klein, JL; Leis, JF; Noel, P; Reeder, CB; Slack, JL; Sproat, LO, 2013
)
0.6
"Ten studies with original IViT ocular side effect data were included in this systematic review."( Ocular side effects following intravitreal injection therapy for retinoblastoma: a systematic review.
Mohney, BG; Smith, BD; Smith, SJ, 2014
)
0.4
" We conclude that with appropriate patient selection and a risk-adapted treatment approach, HDM/SCT is safe and effective in patients with AL amyloidosis and cardiac involvement."( Safety and efficacy of high-dose melphalan and auto-SCT in patients with AL amyloidosis and cardiac involvement.
Berk, JL; Doros, G; Girnius, S; Meier-Ewert, HK; Quillen, K; Ruberg, FL; Sanchorawala, V; Seldin, DC; Sloan, JM, 2014
)
0.68
" In an effort to minimize these adverse effects, we conducted bone marrow transplantation (BMT) from unrelated volunteer donors using a conditioning regimen without BU or TBI."( Fludarabine, cytarabine, granulocyte colony-stimulating factor and melphalan (FALG with L-PAM) as a reduced toxicity conditioning regimen in children with acute leukemia.
Kato, K; Matsumoto, K; Matsuyama, T; Yoshida, N, 2014
)
0.64
"This conditioning regimen of FLAG combined with L-PAM (which did not contain BU and TBI) was associated with good outcomes and minimal late adverse effects in children with acute leukemia who have undergone allogeneic BMT from unrelated volunteer donors."( Fludarabine, cytarabine, granulocyte colony-stimulating factor and melphalan (FALG with L-PAM) as a reduced toxicity conditioning regimen in children with acute leukemia.
Kato, K; Matsumoto, K; Matsuyama, T; Yoshida, N, 2014
)
0.64
"In this retrospective study, a chemotherapy protocol using dexamethasone, melphalan, actinomycin D, and cytosine arabinoside (DMAC) was evaluated for efficacy and adverse event profile as a first line rescue protocol in 86 client-owned dogs previously treated with a CHOP-based protocol."( The efficacy and adverse event profile of dexamethasone, melphalan, actinomycin D, and cytosine arabinoside (DMAC) chemotherapy in relapsed canine lymphoma.
Monteith, G; Parsons-Doherty, M; Poirier, VJ, 2014
)
0.88
"0-graded systemic adverse events."( Efficacy and toxicity of second-course ophthalmic artery chemosurgery for retinoblastoma.
Abramson, DH; Brodie, SE; Dunkel, IJ; Francis, JH; Gobin, YP; Marr, BP; Tendler, I, 2015
)
0.42
" The incidence of grade 3 mucositis and stomatitis was low (10% and 5%, respectively) with no grade 4 mucositis or stomatitis reported (graded according to National Cancer Institute Common Terminology Criteria for Adverse Events)."( A Phase IIb, Multicenter, Open-Label, Safety, and Efficacy Study of High-Dose, Propylene Glycol-Free Melphalan Hydrochloride for Injection (EVOMELA) for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Transplantation.
Aljitawi, OS; Allen, LF; Arce-Lara, C; Bhat, G; Callander, N; Hari, P; Nath, R; Stockerl-Goldstein, K, 2015
)
0.63
"While no toxic effects for a concentration of 80 μg/ml were observed, both b- and a-waves were significantly reduced after application of 160 (b-wave 43."( Investigating short-term toxicity of melphalan in a model of an isolated and superfused bovine retina.
Aisenbrey, S; Bartz-Schmidt, KU; Hagemann, U; Hofmann, K; Januschowski, K; Krupp, C; Mueller, S; Schnichels, S; Spitzer, MS, 2016
)
0.71
"Epiretinal or intraretinal concentrations of 80-μg/ml melphalan do not cause toxic effects in this in vitro model."( Investigating short-term toxicity of melphalan in a model of an isolated and superfused bovine retina.
Aisenbrey, S; Bartz-Schmidt, KU; Hagemann, U; Hofmann, K; Januschowski, K; Krupp, C; Mueller, S; Schnichels, S; Spitzer, MS, 2016
)
0.96
" Although this technique can save eyes otherwise destined for enucleation, ocular salvage may be accompanied by local toxic effects."( Anterior Ocular Toxicity of Intravitreous Melphalan for Retinoblastoma.
Abramson, DH; Brodie, SE; Francis, JH; Marr, BP, 2015
)
0.68
" We report a series of 5 patients from this cohort who developed anterior segment toxic effects."( Anterior Ocular Toxicity of Intravitreous Melphalan for Retinoblastoma.
Abramson, DH; Brodie, SE; Francis, JH; Marr, BP, 2015
)
0.68
"Intravitreous injection of melphalan may result in toxic effects on the anterior segment of the eye, in addition to retinal abnormalities, and appears to be more common in the meridian of the injection where the drug concentration is highest."( Anterior Ocular Toxicity of Intravitreous Melphalan for Retinoblastoma.
Abramson, DH; Brodie, SE; Francis, JH; Marr, BP, 2015
)
0.98
"The purpose of this study is to evaluate the fluctuations of coagulation parameters during cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (HIPEC) and confirm beyond doubt that epidural anaesthesia is safe with this type of operations."( Lack of significant intraoperative coagulopathy in patients undergoing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (HIPEC) indicates that epidural anaesthesia is a safe option.
Alevizos, L; Daskalou, T; Eleftheriadis, S; Iatrou, C; Korakianitis, O; Mavroudis, C; Stamou, K; Tentes, AA; Vogiatzaki, T, 2015
)
0.42
"Our results support the belief that epidural analgesia is a safe option in cytoreductive surgery and HIPEC despite certain intraoperative fluctuations in coagulation parameters."( Lack of significant intraoperative coagulopathy in patients undergoing cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (HIPEC) indicates that epidural anaesthesia is a safe option.
Alevizos, L; Daskalou, T; Eleftheriadis, S; Iatrou, C; Korakianitis, O; Mavroudis, C; Stamou, K; Tentes, AA; Vogiatzaki, T, 2015
)
0.42
" We, therefore, investigated the cellular toxic effects of melphalan, topotecan and carboplatin on the RPE in a cell culture model."( Toxic effects of melphalan, topotecan and carboplatin on retinal pigment epithelial cells.
Aisenbrey, S; Hagemann, U; Januschowski, K; Schnichels, S; Schrader, M; Süsskind, D, 2016
)
1.02
"Morphological monitoring and toxicity assays indicate a direct toxic effect of melphalan and the other two cytostatic drugs on ARPE19 cells."( Toxic effects of melphalan, topotecan and carboplatin on retinal pigment epithelial cells.
Aisenbrey, S; Hagemann, U; Januschowski, K; Schnichels, S; Schrader, M; Süsskind, D, 2016
)
1
" We conclude that OP administration of BEAM conditioning is safe and may offer significant advantages, including decreased length of hospitalization, reduced costs, decreased risks for severe toxicities and infectious complications, and likely improvement in patient satisfaction and quality of life."( Outpatient administration of BEAM conditioning prior to autologous stem cell transplantation for lymphoma is safe, feasible, and cost-effective.
Baran, A; Barr, PM; Becker, MW; Friedberg, JW; Liesveld, JL; Milner, LA; Phillips, GL; Reid, RM; Wedow, L, 2016
)
0.43
" The adverse events during TAS were generally tolerable, but 39 (10."( Efficacy and toxicity of the combination chemotherapy of thalidomide, alkylating agent, and steroid for relapsed/refractory myeloma patients: a report from the Korean Multiple Myeloma Working Party (KMMWP) retrospective study.
Choi, YS; Eom, HS; Han, JJ; Kang, HJ; Kim, HJ; Kim, K; Kim, MK; Kim, SH; Kwon, J; Lee, JH; Lee, JJ; Lee, JO; Lee, WS; Min, CK; Moon, JH; Yoon, DH; Yoon, SS, 2017
)
0.46
" Peri- and post-procedural adverse events (AE) were registered."( Percutaneous Isolated Hepatic Perfusion as a Treatment for Isolated Hepatic Metastases of Uveal Melanoma: Patient Outcome and Safety in a Multi-centre Study.
Brüning, R; Engelke, C; Gebauer, B; Koch, SA; Lotz, G; Radeleff, B; Scholtz, JE; Vogel, A; Vogl, TJ; Wacker, F; Willinek, W; Zeile, M, 2017
)
0.46
" The common adverse events (AEs) were in line with the well-known toxicity profiles associated with VMP."( A prospective, open-label, multicenter, observational study to evaluate the efficacy and safety of bortezomib-melphalan-prednisone as initial treatment for autologous stem cell transplantation-ineligible patients with multiple myeloma.
Bae, SB; Bae, SH; Cho, DY; Choi, CW; Do, YR; Hyun, MS; Jeong, SH; Jo, DY; Joo, YD; Kim, BS; Kim, H; Kim, HG; Kim, HJ; Kim, JA; Kim, JS; Kim, K; Kim, KH; Kim, MK; Kim, SH; Kim, SJ; Kim, SY; Kim, YS; Kwak, JY; Lee, HS; Lee, JH; Lee, JJ; Lee, JO; Lee, MH; Lee, SM; Lee, WS; Lim, SN; Min, CK; Moon, JH; Mun, YC; Nam, SH; Park, JS; Park, KW; Park, MR; Park, SK; Shin, HJ; Song, MK; Yi, HG; Yoon, SS, 2017
)
0.67
"HAIC offers a safe and effective salvage treatment strategy in heavily pretreated patients with LMBC and no further treatment options."( Hepatic arterial infusion chemotherapy for extensive liver metastases of breast cancer: efficacy, safety and prognostic parameters.
Bogner, S; Peis, MW; Reinboldt, MP; Schuler, M; Tewes, M; Theysohn, JM; Welt, A, 2017
)
0.46
"The ILI technique proved safe and effective in elderly patients."( Safety and Efficacy of Isolated Limb Infusion Chemotherapy for Advanced Locoregional Melanoma in Elderly Patients: An Australian Multicenter Study.
Barbour, A; Coventry, BJ; Giles, MH; Henderson, MA; Kroon, HM; Paddle, P; Serpell, J; Smithers, BM; Speakman, D; Thompson, JF; Wall, M, 2017
)
0.46
"1 and Common Terminology Criteria for Adverse Events (CTCAE) v4."( Percutaneous hepatic perfusion with melphalan in uveal melanoma: A safe and effective treatment modality in an orphan disease.
Choi, J; Gangi, A; Gupta, S; Hardman, D; Karydis, I; Ottensmeier, C; Pearce, N; Sileno, S; Stedman, B; Takhar, A; Thomas, K; Wheater, MJ; Wilson, I; Zager, JS, 2018
)
0.76
" A less toxic regimen might improve the outcome of patients with lymphoma after transplantation."( High-dose Bendamustine-EAM followed by autologous stem cell rescue results in long-term remission rates in lymphoma patients, without renal toxicity.
Boehm, A; Keil, F; Koller, E; Menschel, E; Moestl, M; Noesslinger, T; Panny, M; Simanek, R, 2018
)
0.48
" Adverse events (AEs) and toxicity were evaluated."( Percutaneous hepatic perfusion (chemosaturation) with melphalan in patients with intrahepatic cholangiocarcinoma: European multicentre study on safety, short-term effects and survival.
Brüning, R; Ferrucci, PF; Kirstein, MM; Manns, MP; Marquardt, S; Prevoo, W; Radeleff, B; Trillaud, H; Tselikas, L; Vicente, E; Vogel, A; Wacker, FK; Wiggermann, P; Zeile, M, 2019
)
0.76
"PHP is a standardised and safe procedure that provides promising response rates and survival data in patients with iCCA, especially in non-metastatic disease."( Percutaneous hepatic perfusion (chemosaturation) with melphalan in patients with intrahepatic cholangiocarcinoma: European multicentre study on safety, short-term effects and survival.
Brüning, R; Ferrucci, PF; Kirstein, MM; Manns, MP; Marquardt, S; Prevoo, W; Radeleff, B; Trillaud, H; Tselikas, L; Vicente, E; Vogel, A; Wacker, FK; Wiggermann, P; Zeile, M, 2019
)
0.76
" • PHP is a standardised and safe procedure that provides promising response rates and survival data in patients with intrahepatic cholangiocarcinoma (iCCA), especially in non-metastatic disease."( Percutaneous hepatic perfusion (chemosaturation) with melphalan in patients with intrahepatic cholangiocarcinoma: European multicentre study on safety, short-term effects and survival.
Brüning, R; Ferrucci, PF; Kirstein, MM; Manns, MP; Marquardt, S; Prevoo, W; Radeleff, B; Trillaud, H; Tselikas, L; Vicente, E; Vogel, A; Wacker, FK; Wiggermann, P; Zeile, M, 2019
)
0.76
" Although considered bioequivalent, there remains limited literature directly evaluating the adverse events between the two agents."( Evaluating the adverse effects of melphalan formulations.
Glotzbecker, B; Laubach, J; McDonnell, AM; Ni, J; Soiffer, R; Xiang, E, 2019
)
0.79
" These data may assist in better understanding the anticipated adverse effects of a high-dose melphalan conditioning therapy."( Evaluating the adverse effects of melphalan formulations.
Glotzbecker, B; Laubach, J; McDonnell, AM; Ni, J; Soiffer, R; Xiang, E, 2019
)
1.01
"Isolated limb perfusion (ILP) is a safe and well-established treatment for in-transit metastases of melanoma."( Response and Toxicity of Repeated Isolated Limb Perfusion (re-ILP) for Patients With In-Transit Metastases of Malignant Melanoma.
Belgrano, V; Katsarelias, D; Mattsson, J; Nilsson, JA; Olofsson Bagge, R; Pettersson, J, 2019
)
0.51
"UL-ILI is safe to perform and effective, resulting in low limb toxicity."( Evaluation of the efficacy and toxicity of upper extremity isolated limb infusion chemotherapy for melanoma: An Australian multi-center study.
Barbour, A; Coventry, BJ; Henderson, MA; Kroon, HM; Serpell, J; Smithers, BM; Thompson, JF, 2019
)
0.51
" Current treatments have many adverse effects."( Low retinal toxicity of intravitreal carboplatin associated with good retinal tumour control in transgenic murine retinoblastoma.
Cassoux, N; Doz, F; Fréneaux, P; Leboucher, S; Lemaître, S; Poyer, F; Thomas, CD, 2020
)
0.56
"Patients with primary HLH transplanted 2009-2016 after treosulfan- or melphalan-based conditioning regimens were analyzed in a retrospective multicenter study for survival, engraftment, chimerism, and adverse events."( Risk factors for mixed chimerism in children with hemophagocytic lymphohistiocytosis after reduced toxicity conditioning.
Albert, MH; Bader, P; Beier, R; Burkhardt, B; Chada, M; Ehl, S; Greil, J; Gruhn, B; Janka, G; Kühl, JS; Lang, P; Lehmberg, K; Meisel, R; Müller, I; Ozga, AK; Schlegel, PG; Schulz, A; Seidel, M; Speckmann, C; Sykora, KW; Wawer, A; Wössmann, W; Wustrau, K, 2020
)
0.79
"ILI is safe and effective to treat melanoma in-transit metastases."( Factors predicting toxicity and response following isolated limb infusion for melanoma: An international multi-centre study.
Barbour, A; Beasley, GM; Coventry, BJ; Daou, H; Delman, KA; Farley, CR; Farma, JM; Farrow, NE; Henderson, MA; Kenyon-Smith, TJ; Kroon, HM; Lowe, MC; Miura, JT; Mosca, PJ; Mullen, D; Potdar, A; Serpell, J; Smithers, BM; Speakman, D; Sun, J; Teras, J; Thompson, JF; Tyler, DS; Zager, JS, 2020
)
0.56
" The experimental programme was safe and active in a multicentre setting, with only two episodes of grade 4 non-haematological toxicity (hepatotoxicity and mucositis), and no cases of systemic fungal infections; two patients died of toxicity (bacterial infections)."( A dose-dense short-term therapy for human immunodeficiency virus/acquired immunodeficiency syndrome patients with high-risk Burkitt lymphoma or high-grade B-cell lymphoma: safety and efficacy results of the "CARMEN" phase II trial.
Allione, B; Calimeri, T; Cattaneo, C; Donadoni, G; Facchetti, F; Ferrari, D; Ferreri, AJM; Foppoli, M; Fumagalli, L; Lleshi, A; Pecciarini, L; Ponzoni, M; Re, A; Rigacci, L; Rossi, G; Sassone, M; Spina, M; Verga, L, 2021
)
0.62
"Current melphalan-based regimens for intravitreal chemotherapy for retinoblastoma vitreous seeds are effective but toxic to the retina."( Intravitreal HDAC Inhibitor Belinostat Effectively Eradicates Vitreous Seeds Without Retinal Toxicity In Vivo in a Rabbit Retinoblastoma Model.
Bogan, CM; Boyd, KL; Bridges, TM; Calcutt, MW; Chen, SC; Daniels, AB; Friedman, DL; Kaczmarek, JV; Lindsley, CW; Liu, Q; Liu, X; Pierce, JM; Richmond, A; Tao, YK, 2021
)
1.06
"To define a safe treatment dose of ipilimumab (IPI) and nivolumab (NIVO) when applied in combination with percutaneous hepatic perfusion with melphalan (M-PHP) in metastatic uveal melanoma (mUM) patients (NCT04283890), primary objective was defining a safe treatment dose of IPI/NIVO plus M-PHP."( Combining Melphalan Percutaneous Hepatic Perfusion with Ipilimumab Plus Nivolumab in Advanced Uveal Melanoma: First Safety and Efficacy Data from the Phase Ib Part of the Chopin Trial.
Blank, CU; Burgmans, MC; Jonker-Bos, MA; Kapiteijn, E; Lutjeboer, J; Martini, CH; Roozen, CFM; Speetjens, FM; Sporrel-Blokland, M; Tijl, FGJ; Tong, TML; van der Meer, RW; van Erkel, AR; van Persijn van Meerten, EL; van Rijswijk, CSP; Zoethout, RWM, 2023
)
1.51
" Grade III/IV adverse events (AE) were observed in 2/3 patients in cohort 1 and in 3/4 patients in cohort 2, including Systemic Inflammatory Response Syndrome (SIRS), febrile neutropenia and cholecystitis."( Combining Melphalan Percutaneous Hepatic Perfusion with Ipilimumab Plus Nivolumab in Advanced Uveal Melanoma: First Safety and Efficacy Data from the Phase Ib Part of the Chopin Trial.
Blank, CU; Burgmans, MC; Jonker-Bos, MA; Kapiteijn, E; Lutjeboer, J; Martini, CH; Roozen, CFM; Speetjens, FM; Sporrel-Blokland, M; Tijl, FGJ; Tong, TML; van der Meer, RW; van Erkel, AR; van Persijn van Meerten, EL; van Rijswijk, CSP; Zoethout, RWM, 2023
)
1.31
"Combining M-PHP with IPI/NIVO was safe in this small cohort of patients with mUM at a dose of IPI 1 mg/kg and NIVO 3 mg/kg."( Combining Melphalan Percutaneous Hepatic Perfusion with Ipilimumab Plus Nivolumab in Advanced Uveal Melanoma: First Safety and Efficacy Data from the Phase Ib Part of the Chopin Trial.
Blank, CU; Burgmans, MC; Jonker-Bos, MA; Kapiteijn, E; Lutjeboer, J; Martini, CH; Roozen, CFM; Speetjens, FM; Sporrel-Blokland, M; Tijl, FGJ; Tong, TML; van der Meer, RW; van Erkel, AR; van Persijn van Meerten, EL; van Rijswijk, CSP; Zoethout, RWM, 2023
)
1.31
" The most common adverse effects were febrile neutropenia (98%), mucositis (72%) and colitis (60%)."( Single-center retrospective study assessing the efficacy and safety of BeEAM (bendamustine, etoposide, cytarabine, melphalan) as conditioning regimen for autologous hematopoietic stem cell transplantation.
Boudreault, JS; Feng, X; Plante, MÉ,
)
0.34
"For adult patients with MM undergoing aHCT, the outpatient setting is safe and reduces the total length of hospital stay and thus overall transplant costs."( Effectiveness, Safety, and Cost Implications of Outpatient Autologous Hematopoietic Stem Cell Transplant for Multiple Myeloma.
Edwards, K; Hashmi, H; Maldonado, A; Marini, J; Neppalli, A; Weeda, E, 2023
)
0.91

Pharmacokinetics

Melphalan may provide a pharmacokinetic and clinical advantage in gastrointestinal cancer patients who cannot be made cancer-free with cytoreductive surgery. Concentrations of melphalan in tumor nodules on the peritoneal surface were approximately ten times higher than in plasma.

ExcerptReferenceRelevance
"9 mg) followed by a second dose on day 2, calculated on the basis of pharmacokinetic data to achieve a target area under the concentration-time curve (AUC)."( Pharmacokinetically guided dosing for intravenous melphalan: a pilot study in patients with advanced ovarian adenocarcinoma.
Ardiet, C; Chauvin, F; Clavel, M; Guastalla, JP; Ploin, DY; Rebattu, P; Tranchand, B, 1992
)
0.54
" The pharmacokinetic advantages of ILP can be quantified by the ratio of AUCa/AUCs, median value 37."( The pharmacokinetic advantages of isolated limb perfusion with melphalan for malignant melanoma.
Blackie, R; Burnside, G; Byrne, DS; Hughes, J; Kerr, DJ; MacKie, RM; McKay, AJ; Scott, RN, 1992
)
0.52
" bolus pharmacokinetic data."( Pharmacokinetics of very high-dose oral melphalan in cancer patients.
Alberts, DS; Asbury, RF; Boros, L; Goodman, TL; Hickox, DE; Peng, YM; Penn, TE, 1990
)
0.55
" In this patient, melphalan pharmacokinetics was different from that of patients without important renal dysfunction for the area under the concentration curve (1,324 mg."( [Pharmacokinetics of high-dose melphalan (200 mg/m2) in a case of total renal insufficiency].
Ardiet, C; Biron, P; Mercatello, A; Philip, T; Tranchand, B, 1988
)
0.89
" administration were determined in 17 patients with various malignancies for the purpose of assessing interpatient and intrapatient pharmacokinetic variability."( Plasma pharmacokinetics of high-dose oral melphalan in patients treated with trialkylator chemotherapy and autologous bone marrow reinfusion.
Beschorner, JC; Bitran, JD; Choi, KE; Fullem, LJ; Golick, JA; Ratain, MJ; Williams, SF, 1989
)
0.54
" No correlation was found between GFR and the terminal plasma half-life time."( Pharmacokinetics of oral melphalan in relation to renal function in multiple myeloma patients.
Ehrsson, H; Eksborg, S; Mellstedt, H; Osterborg, A; Wallin, I, 1989
)
0.58
" When L-PAM was given 24 h before DDP, the elimination half-life (t 1/2 beta), plasma clearance (Clp), and volume of distribution (Vd beta) of L-PAM were, respectively: 46."( Lack of effect of cisplatin on i.v. L-PAM plasma pharmacokinetics in ovarian cancer patients.
Cavalli, F; D'Incalci, M; Sessa, C; Willems, Y; Zucchetti, M, 1988
)
0.27
"The pharmacokinetic parameters of the alkylating agent melphalan were determined in 15 children and 11 adults with advanced malignant solid tumors."( Pharmacokinetics of high-dose intravenous melphalan in children and adults with forced diuresis. Report in 26 cases.
Ardiet, C; Biron, P; Philip, T; Rebattu, P; Tranchand, B, 1986
)
0.78
" The elimination half-life (t1/2 less than 80 min) allows autologous bone marrow transplantation 24 h after the drug administration."( Pharmacokinetics of high-dose melphalan in children and adults.
Gouyette, A; Hartmann, O; Pico, JL, 1986
)
0.56
" The apparent plasma elimination half-life (t1/2) was 17."( The effect of systemic hyperthermia on melphalan pharmacokinetics in mice.
Bleehen, NM; Donaldson, J; Honess, DJ; Workman, P, 1985
)
0.54
" The initial loss with a half-life (t1/2) of approximately 5 to 10 min is interpreted as rapid uptake of melphalan by the tissue of the perfused extremity."( Pharmacokinetics of melphalan in clinical isolation perfusion of the extremities.
Briele, HA; Das Gupta, TK; Djuric, M; Jung, DT; Mortell, T; Patel, MK, 1985
)
0.81
" No significant difference was seen in pharmacokinetic parameters between patients undergoing and not undergoing forced diuresis."( High dose melphalan in children with advanced malignant disease. A pharmacokinetic study.
Baurain, R; Cornu, G; de Selys, A; Ninane, J; Trouet, A, 1985
)
0.67
" Such a pharmacokinetic effect could account for part of the potentiation of MEL and CY action observed in tumours with large single doses of MISO."( The effect of radiosensitizers on the pharmacokinetics of melphalan and cyclophosphamide in the mouse.
Hinchliffe, M; McNally, NJ; Stratford, MR, 1983
)
0.51
"A pharmacokinetic study of high dose intravenous melphalan, 180 mg/m2, was performed in eight patients."( Pharmacokinetics of high dose melphalan.
Hersh, MR; Knight, WA; Kuhn, JG; Ludden, TM, 1983
)
0.81
"0166 min-1, Tmax = 59."( The pharmacokinetics of melphalan in patients with multiple myeloma: an intravenous/oral study using a conventional dose regimen.
Hamilton, P; Lennard, A; Rawlins, MD; Woodhouse, KW, 1983
)
0.57
" Mass spectrometry confirmation of each drug from patient sample separations is presented to provide unambiguous identification for valid pharmacokinetic parameter determination."( HPLC, MS, and pharmacokinetics of melphalan, bisantrene and 13-cis retinoic acid.
Alberts, DS; Davis, TP; Goodman, GE; Peng, YM, 1982
)
0.54
" The results are discussed in relation to the pharmacokinetic availability of drugs in the plasma compartment of patients treated by different therapuetic regimens."( Pharmacokinetic approach to in vitro testing of ovarian cancer cell sensitivity.
Balconi, G; D'Incalci, M; Erba, E; Garattini, S; Morasca, L; Ottolenghi, L; Salmona, A, 1980
)
0.26
" On a pharmacokinetic basis, chlorambucil's greater in vitro stability, its more rapid and predictable systemic availability after oral dosing, and its extremely low urinary excretion make it a more predictable alkylating agent for clinical use than melphalan, especially for patients with reduced renal function."( Comparative pharmacokinetics of chlorambucil and melphalan in man.
Alberts, DS; Chang, SY; Chen, HS; Evans, TL; Larcom, BJ, 1980
)
0.7
"The development of time-dependent pharmacodynamic models in cancer chemotherapy has been extremely limited."( Time-dependent pharmacodynamic models in cancer chemotherapy: population pharmacodynamic model for glutathione depletion following modulation by buthionine sulfoximine (BSO) in a Phase I trial of melphalan and BSO.
Brennan, J; Gallo, JM; Halbherr, T; Hamilton, TC; Laub, PB; O'Dwyer, PJ; Ozols, RF, 1995
)
0.48
" Pharmacokinetic sampling was performed so as to describe the clearance of melphalan in this population and in an attempt to carry out pharmacodynamic modeling for toxicity and response."( Phase II trial and pharmacokinetic assessment of intravenous melphalan in patients with advanced prostate cancer.
Ambinder, RM; Egorin, MJ; Ellis, PG; Jodrell, DI; Kreis, W; Smith, DC; Trump, DL; Zuhowski, EG, 1993
)
0.76
" For all patients, AUC, CIT, Vdss, t1/2 beta and MRT were significantly correlated with creatinine clearance; the different pharmacokinetic parameters calculated showed great interindividual variations."( Pharmacokinetics of high-dose melphalan in adults: influence of renal function.
Harousseau, JL; Kergueris, MF; Larousse, C; Milpied, N; Moreau, P,
)
0.42
" The present study showed that previous carboplatin administrations induced wide variations of melphalan pharmacokinetic parameters between the two administrations."( [Effect of carboplatin on the pharmacokinetics of melphalan administered intravenously].
Ardiet, C; Biron, P; Bouffet, E; Brunat-Mentigny, M; Nasri, F; Philip, I; Tranchand, B, 1994
)
0.76
" Pharmacokinetic parameters (mean values of steady-state volume of distribution 14."( A pilot study of 220 mg/m2 melphalan followed by autologous stem cell transplantation in patients with advanced haematological malignancies: pharmacokinetics and toxicity.
Bataille, R; Bulabois, CE; Harousseau, JL; Kergueris, MF; Larousse, C; Le Tortorec, S; Mahé, B; Milpied, N; Moreau, P; Rapp, MJ, 1996
)
0.59
" This method was used to determine the pharmacokinetic parameters of melphalan following high-dose (140 mg/m2) intravenous administration in patients with advanced malignancies undergoing peripheral blood hematopoietic progenitor-cell transplantation."( High-performance liquid chromatographic assay for melphalan in human plasma. Application to pharmacokinetic studies.
Astre, C; Bressolle, F; Grosse, PY; Joulia, JM; Martel, P; Pinguet, F, 1996
)
0.78
" Plasma levels of melphalan declined in a biexponential fashion with a mean terminal half-life of 83 minutes (range 52-168 minutes)."( Pharmacokinetics of high-dose intravenous melphalan in patients undergoing peripheral blood hematopoietic progenitor-cell transplantation.
Astre, C; Bressolle, F; Canal, P; Culine, S; Fabbro, M; Martel, P; Petit, I; Pinguet, F,
)
0.73
"The optimal dosing schedule for melphalan therapy of recurrent malignant melanoma in isolated limb perfusions has been examined using a physiological pharmacokinetic model with data from isolated rat hindlimb perfusions (IRHP)."( Melphalan dosing regimens for management of recurrent melanoma by isolated limb perfusion: application of a physiological pharmacokinetic model based on melphalan distribution in the isolated perfused rat hindlimb.
Roberts, MS; Smithers, BM; Wu, ZY, 1997
)
2.02
" The time course of melphalan concentrations in perfusate and tissues during a 60-min melphalan perfusion and 30-min drug-free washout in the single-pass perfused rat hindlimb was examined using a physiologically based pharmacokinetic model."( Tissue and perfusate pharmacokinetics of melphalan in isolated perfused rat hindlimb.
Roberts, MS; Smithers, BM; Wu, ZY, 1997
)
0.89
" Three patients with severe renal impairment first received a low test dose of MEL (16 mg/m2) for pharmacokinetic studies."( Safety of autotransplants with high-dose melphalan in renal failure: a pharmacokinetic and toxicity study.
Alberts, DS; Barlogie, B; Bracy, D; Dorr, RT; Jagannath, S; Johnson, C; Meyers, R; Roe, DJ; Tricot, G; Vesole, DH, 1996
)
0.56
" The temperature of the perfusate plasma and tissue, pH, administration method, and flow rate were modified and compared with regard to their influence on pharmacokinetic parameters."( Pharmacokinetics of melphalan in isolated limb perfusion.
Goehl, J; Hohenberger, W; Loos, U; Norda, A; Sastry, M, 1999
)
0.63
"The objectives of the present study were to determine the following: (a) the maximum tolerated dose (MTD) of melphalan using a 24-h continuous infusion; (b) the clinical toxicity; and (c) the pharmacokinetic characteristics of melphalan at each dose level."( A phase I and pharmacokinetic study of melphalan using a 24-hour continuous infusion in patients with advanced malignancies.
Astre, C; Bressolle, F; Chevillard, C; Culine, S; Fabbro, M; Pinguet, F; Serre, MP, 2000
)
0.79
"Pharmacokinetic and pharmacodynamic data were collected in 17 women entered in a phase II trial of sequential HDP, and high-dose melphalan and cyclophosphamide/thiotepa/carboplatin."( The pharmacokinetics and pharmacodynamics of high-dose paclitaxel monotherapy (825 mg/m2 continuous infusion over 24 h) with hematopoietic support in women with metastatic breast cancer.
Balmaceda, CM; Egorin, MJ; Hesdorffer, CS; Huang, M; Kaufman, E; Papadopoulos, KP; Troxel, AB; Vahdat, LT, 2001
)
0.52
" The degree of neurotoxicity subsequent to HDP was associated with the degree of baseline neuropathy but was not predictable from pharmacokinetic parameters."( The pharmacokinetics and pharmacodynamics of high-dose paclitaxel monotherapy (825 mg/m2 continuous infusion over 24 h) with hematopoietic support in women with metastatic breast cancer.
Balmaceda, CM; Egorin, MJ; Hesdorffer, CS; Huang, M; Kaufman, E; Papadopoulos, KP; Troxel, AB; Vahdat, LT, 2001
)
0.31
" We report pharmacokinetic data from 12 patients treated by ILI for tumours of the limb in Brisbane."( Pharmacokinetics and pharmacodynamics of melphalan in isolated limb infusion for recurrent localized limb malignancy.
Anissimov, YG; Roberts, MS; Siebert, GA; Smithers, BM; Thompson, JF; Wu, ZY, 2001
)
0.58
"By studying the pharmacokinetic data of melphalan in the circuit and in the systemic circulation, we were able to validate the 99mTc procedure used during clinical perfusion."( Melphalan monitoring during hyperthermic perfusion of isolated limb for melanoma: pharmacokinetic study and 99mTc-albumin microcolloid technique.
Buffa, E; Cattel, L; Ceruti, M; Cesana, P; De Simone, M; Dosio, F; Novello, S,
)
1.84
"In order to control busulfan pharmacokinetic variability and toxicity, a specific monitoring protocol was instituted in our bone marrow transplant BMT paediatric patients including a test dose, daily Bayesian forecasting of busulfan plasma levels, and Bayesian individualization of busulfan dosage regimens."( Improved clinical outcome of paediatric bone marrow recipients using a test dose and Bayesian pharmacokinetic individualization of busulfan dosage regimens.
Aulagner, G; Bertrand, Y; Bleyzac, N; Dai, Q; Galambrun, C; Janoly, A; Jelliffe, RW; Magron, P; Maire, P; Martin, P; Souillet, G, 2001
)
0.31
"166Ho-DOTMP has physical and pharmacokinetic characteristics compatible with high-dose myeloablative treatment of multiple myeloma."( High-dose 166Ho-DOTMP in myeloablative treatment of multiple myeloma: pharmacokinetics, biodistribution, and absorbed dose estimation.
Bensinger, W; Durack, LD; Eary, JF; Fritzberg, A; Rajendran, JG; Vernon, C, 2002
)
0.31
" The population pharmacokinetic analysis was performed using NONMEM through the graphical interface Visual-NM."( Population pharmacokinetics of melphalan, infused over a 24-hour period, in patients with advanced malignancies.
Astre, C; Bressolle, F; Culine, S; Fabbro, M; Mougenot, P; Pinguet, F; Poujol, S, 2004
)
0.61
"The population pharmacokinetic approach developed in this study should allow dosage to be individualized in order to decrease toxicity while maintaining good efficacy."( Population pharmacokinetics of melphalan, infused over a 24-hour period, in patients with advanced malignancies.
Astre, C; Bressolle, F; Culine, S; Fabbro, M; Mougenot, P; Pinguet, F; Poujol, S, 2004
)
0.61
" The pharmacokinetic study showed that pelvic SFP was associated with a leakage rate higher than femoral SFP (38% vs 28%)."( Hypoxic antiblastic stop-flow perfusion: clinical outcome and pharmacokinetic findings.
Bertolo, S; Casara, D; Darisi, T; Foletto, M; Lise, M; Minante, M; Miotto, D; Mocellin, S; Nitti, D; Ori, C; Rossi, CR, 2004
)
0.32
"3-18 years) and determine whether any clinical factors affect the pharmacokinetic parameters Additionally, to examine whether a test melphalan dose can predict the pharmacokinetics of a full dose, when there are 5 intervening days of carboplatin therapy."( Melphalan pharmacokinetics in children with malignant disease: influence of body weight, renal function, carboplatin therapy and total body irradiation.
Earl, JW; Montgomery, K; Nath, CE; Shaw, PJ, 2005
)
1.98
" Pharmacodynamic correlations were noted between the docetaxel area under the curve and peripheral neuropathy or stomatitis."( Phase I and pharmacokinetic study of docetaxel combined with melphalan and carboplatin, with autologous hematopoietic progenitor cell support, in patients with advanced refractory malignancies.
Barón, AE; Bearman, SI; Cagnoni, PJ; Gustafson, D; Jones, RB; Long, M; Matthes, S; McSweeney, PA; Nieto, Y; Shpall, EJ, 2005
)
0.57
" The use of heated intraoperative intraperitoneal melphalan may provide a pharmacokinetic and clinical advantage in this group of gastrointestinal cancer patients who cannot be made cancer-free with cytoreductive surgery."( Pharmacokinetic and phase II study of heated intraoperative intraperitoneal melphalan.
Stuart, OA; Sugarbaker, PH, 2007
)
0.82
"In the present work, we report on the results of our pilot study of hyperthermic isolated hepatic perfusion (IHP) with melphalan alone for patients with unresectable metastatic liver tumors refractory to conventional treatments, with particular regard to the correlation between pharmacokinetic findings and hepatic toxicity."( Correlation between melphalan pharmacokinetics and hepatic toxicity following hyperthermic isolated liver perfusion for unresectable metastatic disease.
Casara, D; Corazzina, S; Da Pian, P; Forlin, M; Innocente, F; Lise, M; Mocellin, S; Nitti, D; Ori, C; Pilati, P; Rossi, CR; Ujka, F, 2007
)
0.87
"To develop a population pharmacokinetic model for melphalan in children with malignant diseases and to evaluate limited sampling strategies for melphalan."( Population pharmacokinetics of melphalan in paediatric blood or marrow transplant recipients.
Earl, JW; Montgomery, K; Nath, CE; Shaw, PJ, 2007
)
0.88
" Population pharmacokinetic modelling was performed with the NONMEM software."( Population pharmacokinetics of melphalan in paediatric blood or marrow transplant recipients.
Earl, JW; Montgomery, K; Nath, CE; Shaw, PJ, 2007
)
0.63
"A population pharmacokinetic model for melphalan has been developed and validated and may now be used in conjunction with pharmacodynamic data to develop safe and effective dosing guidelines in children with malignant diseases."( Population pharmacokinetics of melphalan in paediatric blood or marrow transplant recipients.
Earl, JW; Montgomery, K; Nath, CE; Shaw, PJ, 2007
)
0.89
" As melphalan is a phenylalanine derivative, the pharmacokinetic variability may be determined by genetic polymorphisms in the L-type amino acid transporters LAT1 (SLC7A5) and LAT2 (SLC7A8)."( Genetic polymorphisms in the amino acid transporters LAT1 and LAT2 in relation to the pharmacokinetics and side effects of melphalan.
Brockmöller, J; Heider, U; Hohloch, K; Kaiser, R; Kühne, A; Muhlke, S; Niere, W; Overbeck, T; Schirmer, M; Sezer, O; Trümper, L, 2007
)
1.1
" A high interindividual pharmacokinetic variability was observed in 84 patients."( Population pharmacokinetics of melphalan and glutathione S-transferase polymorphisms in relation to side effects.
Brockmöller, J; Heider, U; Hohloch, K; Kaiser, R; Kühne, A; Meineke, I; Muhlke, S; Niere, W; Overbeck, T; Sezer, O; Trümper, L, 2008
)
0.63
" We present the results of pharmacokinetic analysis of a phase I and extension trial."( Pharmacokinetics of isolated lung perfusion with melphalan for resectable pulmonary metastases, a phase I and extension trial.
De Bruijn, E; Grootenboers, MJ; Hendriks, JM; Knibbe, CA; Schramel, FM; Stockman, B; van Boven, WJ; van Putte, B; Van Schil, PE; Vermorken, JB, 2007
)
0.59
" pharmacokinetic data on 670 drugs representing, to our knowledge, the largest publicly available set of human clinical pharmacokinetic data."( Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
Lombardo, F; Obach, RS; Waters, NJ, 2008
)
0.35
"Population pharmacokinetic modelling (using NONMEM) was performed with total and unbound concentration-time data from 100 patients (36-73 years) who had received a median 192 mg m(-2) melphalan dose."( Population pharmacokinetics of melphalan in patients with multiple myeloma undergoing high dose therapy.
Duffull, SB; Earl, J; Gurney, H; Hegarty, G; Joshua, D; Kerridge, I; Kwan, YL; McLachlan, AJ; Nath, CE; Presgrave, P; Shaw, PJ; Tiley, C; Trotman, J; Zeng, L, 2010
)
0.84
" Pharmacokinetic studies were fitted to a linear, 2-compartment model in which dose reduction led to incomplete saturation of CD52 binding sites and greater antibody clearance."( Impact of in vivo alemtuzumab dose before reduced intensity conditioning and HLA-identical sibling stem cell transplantation: pharmacokinetics, GVHD, and immune reconstitution.
Bloor, A; Chakraverty, R; Clark, A; Clark, R; Collin, M; Crawley, C; Fielding, A; Hale, G; Johnson, P; Kottaridis, P; Mackinnon, S; Milligan, D; Morris, E; Orti, G; Parker, A; Peggs, K; Pettengell, R; Pettitt, A; Roughton, M; Shen, J; Snowden, J; Thomson, K, 2010
)
0.36
" Pharmacokinetic parameters, including half-life, volume of distribution, clearance and extent of renal elimination of melphalan were essentially unchanged between the two formulations."( Preclinical comparison of intravenous melphalan pharmacokinetics administered in formulations containing either (SBE)7 m-β-cyclodextrin or a co-solvent system.
Charman, SA; Katneni, K; Koltun, M; McIntosh, MP; Morizzi, J; Shackleford, DM, 2010
)
0.84
" Standard pharmacokinetic parameters were calculated and nonparametric testing was applied to assess the differences between aprepitant and placebo treatment."( The NK₁ receptor antagonist aprepitant does not alter the pharmacokinetics of high-dose melphalan chemotherapy in patients with multiple myeloma.
Burhenne, J; Egerer, G; Eisenlohr, K; Gronkowski, M; Mikus, G; Riedel, KD, 2010
)
0.58
" In addition, AUC and terminal elimination half-life were not changed by aprepitant."( The NK₁ receptor antagonist aprepitant does not alter the pharmacokinetics of high-dose melphalan chemotherapy in patients with multiple myeloma.
Burhenne, J; Egerer, G; Eisenlohr, K; Gronkowski, M; Mikus, G; Riedel, KD, 2010
)
0.58
"To evaluate the ability of a physiology-based pharmacokinetic (PBPK) model to predict the systemic drug exposure of high- and low-dose etoposide in children from a model developed with adult data."( Physiologically based pharmacokinetic modelling of high- and low-dose etoposide: from adults to children.
Boos, J; Hempel, G; Kersting, G; Lippert, J; Willmann, S; Würthwein, G, 2012
)
0.38
" This approach could be useful for planning pharmacokinetic studies in children."( Physiologically based pharmacokinetic modelling of high- and low-dose etoposide: from adults to children.
Boos, J; Hempel, G; Kersting, G; Lippert, J; Willmann, S; Würthwein, G, 2012
)
0.38
" Pharmacokinetic and biodistribution studies of the optimized formulation in comparison to the pure drug suspension were done using γ-scintigraphy on female Balb/c mice."( Anticancer efficacy, tissue distribution and blood pharmacokinetics of surface modified nanocarrier containing melphalan.
Bali, V; Pathak, K; Rajpoot, P, 2012
)
0.59
" In patients, a large variability in pharmacokinetic parameters was observed and it was explained mainly by body weight (P<0."( Pharmacokinetic analysis of melphalan after superselective ophthalmic artery infusion in preclinical models and retinoblastoma patients.
Abramson, DH; Asprea, M; Bramuglia, GF; Buitrago, E; Ceciliano, A; Chantada, GL; Fandino, A; Requejo, F; Schaiquevich, P; Taich, P; Torbidoni, A, 2012
)
0.67
"Single-center, prospective, clinical pharmacokinetic study."( Clinical pharmacokinetics of intra-arterial melphalan and topotecan combination in patients with retinoblastoma.
Buitrago, E; Ceciliano, A; Chantada, GL; Fandino, A; Lucena, E; Romero, L; Sampor, C; Schaiquevich, P; Taich, P; Villasante, F, 2014
)
0.66
" Plasma samples were obtained for pharmacokinetic studies, and a population approach via nonlinear mixed effects modeling was used."( Clinical pharmacokinetics of intra-arterial melphalan and topotecan combination in patients with retinoblastoma.
Buitrago, E; Ceciliano, A; Chantada, GL; Fandino, A; Lucena, E; Romero, L; Sampor, C; Schaiquevich, P; Taich, P; Villasante, F, 2014
)
0.66
"Melphalan and topotecan pharmacokinetic parameters and efficacy and safety parameters."( Clinical pharmacokinetics of intra-arterial melphalan and topotecan combination in patients with retinoblastoma.
Buitrago, E; Ceciliano, A; Chantada, GL; Fandino, A; Lucena, E; Romero, L; Sampor, C; Schaiquevich, P; Taich, P; Villasante, F, 2014
)
2.11
" Concomitant topotecan administration did not influence melphalan pharmacokinetic parameters."( Clinical pharmacokinetics of intra-arterial melphalan and topotecan combination in patients with retinoblastoma.
Buitrago, E; Ceciliano, A; Chantada, GL; Fandino, A; Lucena, E; Romero, L; Sampor, C; Schaiquevich, P; Taich, P; Villasante, F, 2014
)
0.91
"Relationships between pharmacokinetic (PK) parameters of etoposide and toxicity survivals were reported in cancer patients treated at standard doses."( Etoposide pharmacokinetics impact the outcomes of lymphoma patients treated with BEAM regimen and ASCT: a multicenter study of the LYmphoma Study Association (LYSA).
Bachy, E; Casasnovas, O; Freyer, G; Guitton, J; Hénin, E; Ribrag, V; Salles, G; Sebban, C; Tilly, H; Tod, M; You, B, 2015
)
0.42
" Melphalan was quantitated in the biological samples using a validated high-performance liquid-chromatography technique and pharmacokinetic parameters were calculated by means of compartmental models."( Pharmacokinetics of Melphalan After Intravitreal Injection in a Rabbit Model.
Asprea, M; Buitrago, E; Chantada, G; Schaiquevich, P; Williams, G; Winter, U, 2016
)
1.67
" Also pharmacokinetic advantage of melphalan over systemic administration of the drug was shown."( [Melphalan pharmacokinetics during isolated limb regional perfusion in patients with skin melanoma and soft tissue sarcoma].
Gafton, GI; Gafton, IG; Kireeva, GS; Petrov, VG; Semiletova, YV; Senchik, KY; Zinoviev, GV, 2015
)
1.6
"We performed an experimental study of busulfan and melphalan pharmacodynamic and pharmacokinetics in iron overloaded mice."( Iron Overload Exacerbates Busulfan-Melphalan Toxicity Through a Pharmacodynamic Interaction in Mice.
Bouligand, J; Daudigeos-Dubus, E; Deroussent, A; Kessari, R; Mercier, L; Munier, F; Opolon, P; Orear, C; Paci, A; Richard, C; Simonnard, N; Tou, B; Valteau-Couanet, D; Vassal, G, 2016
)
0.96
" This clearance alteration was exacerbated in iron overloaded mice demonstrating a pharmacokinetic interaction."( Iron Overload Exacerbates Busulfan-Melphalan Toxicity Through a Pharmacodynamic Interaction in Mice.
Bouligand, J; Daudigeos-Dubus, E; Deroussent, A; Kessari, R; Mercier, L; Munier, F; Opolon, P; Orear, C; Paci, A; Richard, C; Simonnard, N; Tou, B; Valteau-Couanet, D; Vassal, G, 2016
)
0.71
"Iron overload increases melphalan and busulfan-melphalan toxicity through a pharmacodynamic interaction and reveals a pharmacokinetic drug interaction between busulfan and melphalan."( Iron Overload Exacerbates Busulfan-Melphalan Toxicity Through a Pharmacodynamic Interaction in Mice.
Bouligand, J; Daudigeos-Dubus, E; Deroussent, A; Kessari, R; Mercier, L; Munier, F; Opolon, P; Orear, C; Paci, A; Richard, C; Simonnard, N; Tou, B; Valteau-Couanet, D; Vassal, G, 2016
)
1.02
" Pharmacokinetic dosing of busulfan (Bu) is frequently done for myeloablative conditioning, but evidence for its use is limited in RIC transplants."( Fludarabine-Busulfan Reduced-Intensity Conditioning in Comparison with Fludarabine-Melphalan Is Associated with Increased Relapse Risk In Spite of Pharmacokinetic Dosing.
Al-Kali, A; Alkhateeb, HB; Damlaj, M; Gangat, N; Gastineau, DA; Hashmi, S; Hefazi, M; Hogan, WJ; Litzow, MR; Partain, DK; Patnaik, MM; Wolf, RC, 2016
)
0.66
" From a population pharmacokinetic model, we identified that fat-free mass, hematocrit, and creatinine clearance were significant covariates, as reported previously."( Associations of High-Dose Melphalan Pharmacokinetics and Outcomes in the Setting of a Randomized Cryotherapy Trial.
Benson, DM; Cho, YK; Devine, SM; Efebera, YA; Gao, Y; Hade, EM; Hofmeister, CC; Lamprecht, M; Li, J; Phelps, MA; Poi, M; Rosko, AE; Sborov, DW; Tackett, K; Wang, J; Williams, N, 2017
)
0.76
" In addition, Cmax was associated with a progression-free survival advantage."( Bortezomib pharmacokinetics in tumor response and peripheral neuropathy in multiple myeloma patients receiving bortezomib-containing therapy.
Choi, K; Han, S; Hong, T; Lee, J; Lee, SE; Min, CK; Park, GJ; Yim, DS, 2017
)
0.46
"A pharmacokinetic study was performed in 5 melanoma patients treated with surgical IPP and five with percutaneous IPP."( Surgical versus percutaneous isolated pelvic perfusion (IPP) for advanced melanoma: comparison in terms of melphalan pharmacokinetic pelvic bio-availability.
Clementi, M; Fiorentini, G; Giordano, AV; Guadagni, S; Marsili, L; Masedu, F; Palumbo, G; Valenti, M, 2017
)
0.67
"The purpose of this study was to develop a population pharmacokinetic model and optimal sampling strategy."( Population Pharmacokinetics and Optimal Sampling Strategy for Model-Based Precision Dosing of Melphalan in Patients Undergoing Hematopoietic Stem Cell Transplantation.
Anaissie, EJ; Chandra, S; Dong, M; Fukuda, T; McConnell, S; Mehta, PA; Mizuno, K; Vinks, AA, 2018
)
0.7
"The developed pharmacokinetic model-based sparse sampling strategy promises to achieve the target area under the curve as part of precision dosing."( Population Pharmacokinetics and Optimal Sampling Strategy for Model-Based Precision Dosing of Melphalan in Patients Undergoing Hematopoietic Stem Cell Transplantation.
Anaissie, EJ; Chandra, S; Dong, M; Fukuda, T; McConnell, S; Mehta, PA; Mizuno, K; Vinks, AA, 2018
)
0.7
" A population pharmacokinetic model was built for carboplatin to evaluate various dosing formulas."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.76
"Allometric weight was the only significant, independent covariate for the pharmacokinetic parameters of carboplatin, etoposide and melphalan."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.96
"GFR did not appear to influence the pharmacokinetics of carboplatin after adjusting pharmacokinetic parameters for weight."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.76
" Population pharmacokinetic analyses were conducted to determine the pharmacokinetics of intravenous daratumumab in combination therapy versus monotherapy, evaluate the effect of patient- and disease-related covariates on drug disposition, and examine the relationships between daratumumab exposure and efficacy/safety outcomes."( Pharmacokinetics and Exposure-Response Analyses of Daratumumab in Combination Therapy Regimens for Patients with Multiple Myeloma.
Belch, A; Capra, M; Clemens, PL; Dimopoulos, MA; Gomez, D; Ho, PJ; Iida, S; Jansson, R; Leiba, M; Medvedova, E; Min, CK; Schecter, J; Sonneveld, P; Sun, YN; Xu, XS; Zhang, L, 2018
)
0.48
" We hypothesized either one or a combination of factors: (1) frailty (assessed by IMWG frailty score), (2) generic melphalan pharmacokinetic area under the curve (AUC) assessed by high-performance liquid chromatography, and (3) pharmacogenetics of glutathione S-transferase (GSTP1) assessed by Sanger sequencing, to be associated with toxicity and survival outcomes post auto-HCT."( Impact of frailty, melphalan pharmacokinetics, and pharmacogenetics on outcomes post autologous hematopoietic cell transplantation for multiple myeloma.
Jain, A; Kasudhan, KS; Khadwal, A; Lad, DP; Malhotra, P; Malhotra, S; Nampoothiri, RV; Patil, AN; Prakash, G; Varma, N; Varma, S; Verma Attri, S, 2019
)
1.05
" Our aim was to establish a method of pharmacokinetic (PK) testing for real-time melphalan dose adjustments."( Development of a method for clinical pharmacokinetic testing to allow for targeted Melphalan dosing in multiple myeloma patients undergoing autologous transplant.
Calip, GS; Galvin, JP; Hofmeister, CC; Johnson, JJ; Mahmud, N; Patel, P; Rondelli, D; Sweiss, K; Vemu, B; Wenzler, E, 2020
)
1.01
" We performed a single-center, prospective pharmacokinetic study of 37 pediatric patients undergoing HCT from March 2015 to 2019."( Population Pharmacokinetics of Melphalan for Pediatric Patients Undergoing Hematopoietic Cell Transplantation.
Apsel Winger, B; Chan, D; Dvorak, CC; Gobburu, JVS; Li, S; Long-Boyle, J; Lu, Y, 2022
)
1.01
"There exists considerably large interpatient variability in pharmacokinetic exposure of high dose melphalan in multiple myeloma patients with hematopoietic stem-cell transplantation."( Evaluating the Impacts of CYP3A4*1B and CYP3A5*3 Variations on Pharmacokinetic Behavior and Clinical Outcomes in Multiple Myeloma Patients With Autologous Stem Cell Transplant.
Cho, YK; Hofmeister, CC; Li, J; Phelps, MA; Poi, MJ; Sborov, DW,
)
0.35

Compound-Compound Interactions

The study aimed to determine the maximum tolerated dose (MTD) of lenalidomide in combination with melphalan and dexamethasone (M-dex) The aim was to assess the efficacy and tolerability of this therapy for patients with de novo AL amyloidosis.

ExcerptReferenceRelevance
" Cis-dichlorodiammineplatinum (II) may be effectively combined with sarcolysin."( [Antitumor action of cis-dichlorodiammineplatinum(II) and the effectiveness of a combination with sarcolysine].
Konovalova, AL; Presnov, MA; Romanova, LF; Sof'ina, ZP; Stetsenko, AI, 1978
)
0.26
" The effect of the immunopotentiator when used in combination with alkylating agents was greater than that seen when it was used with the antimetabolite 5-fluorouracil."( The effect of Corynebacterium parvum in combination with 5-fluorouracil, L-phenylalanine mustard, or methotrexate on the inhibition of tumor growth.
Fisher, B; Rubin, H; Saffer, E; Wolmark, N, 1976
)
0.26
" BSO demonstrated in vivo antitumor activity in B16 melanoma-bearing mice prolonging survival by 29% and in combination with 3,4-DHBA resulted in a slight (48% versus 38%) increase in life span as compared to 3,4-DHBA alone."( Melanoma cytotoxicity of buthionine sulfoximine (BSO) alone and in combination with 3,4-dihydroxybenzylamine and melphalan.
FitzGerald, GB; Prezioso, JA; Wick, MM, 1992
)
0.49
" The cytotoxic drug combination of methotrexate and melphalan, or nifedipine alone (0."( The effect of nifedipine alone or combined with cytotoxic chemotherapy on the mouse NC carcinoma in-vitro and in-vivo.
Bennett, A; Chambers, E; Gaffen, JD; Stamford, IF; Tavares, IA, 1991
)
0.53
"This preliminary analysis of a phase II study suggests that high-dose rTNF alpha can be administered with acceptable toxicity by ILP with dopamine and hyperhydration."( High-dose recombinant tumor necrosis factor alpha in combination with interferon gamma and melphalan in isolation perfusion of the limbs for melanoma and sarcoma.
Delmotte, JJ; Ewalenko, P; Lejeune, FJ; Lienard, D; Renard, N, 1992
)
0.5
"We reported previously that treatment of mice bearing MOPC-315 plasmacytoma with the drugs L-PAM (phenylalanine mustard) or 5-FU (5-fluorouracil), in combination with low doses of THF-gamma 2, was more effective in increasing their survival time than treatment with the drug alone."( Therapeutic effectiveness against MOPC-315 plasmacytoma of low or high doses of the synthetic thymic hormone THF-gamma 2 in combination with an "immunomodulating" or a "non-immunomodulating" drug.
Ben-Efraim, S; Burstein, Y; Harshemesh, H; Ophir, R; Pecht, M; Trainin, N, 1991
)
0.28
" In addition, the influence on the therapeutic efficacy of the combination with diazoxide, causing a mild, reversible diabetes, and with insulin was investigated."( Antineoplastic efficacy of melphalan and N-(2-chloroethyl)-N-nitrosocarbamoyl-omega-lysine, in combination with diazoxide or insulin in autochthonous mammary carcinoma of the Sprague-Dawley rat.
Berger, MR; Fink, M; Klenner, T; Schmähl, D; Zelezny, O, 1990
)
0.58
" There was little evidence that either form of treatment was superior in prolonging survival, for in one study the patients treated with a drug combination lived significantly longer than those treated with MP, while in another the reverse was true, and there was no difference in the survival of the two groups in the remaining 11 studies."( Melphalan/prednisone versus drug combinations for plasma cell myeloma.
Bergsagel, DE, 1989
)
1.72
" This reversal was inhibited when menogarol was combined with melphalan."( Synergistic combination of menogarol and melphalan and other two drug combinations.
Adams, EG; Bhuyan, BK; Crampton, SL; Johnson, M, 1985
)
0.78
" New methods of administration are being studied giving interferon alfa-2b as a single agent or in combination with cisplatin by the intraperitoneal route to patients with relapsing ovarian carcinomas limited to the peritoneal cavity."( Overview of preclinical and clinical studies of interferon alfa-2b in combination with cytotoxic drugs.
Welander, CE, 1987
)
0.27
" SHDAAs, including doxorubicin (DOX), cyclophosphamide (CTX), carmustine (BCNU) and melphalan (L-PAM) were then combined with L-BSO in mice bearing P388, MOPC-315 or colon 38 tumors."( Lack of enhanced antitumor efficacy for L-buthionine sulfoximine in combination with carmustine, cyclophosphamide, doxorubicin or melphalan in mice.
Dorr, RT; Soble, MJ,
)
0.56
" Vitamin A induces cytotoxic effects: in combination with melphalan (MELPH), as can be deduced from the resulted synergism on induction of SCEs, the produced cell division delay and the suppressed mitotic index; in combination with caffeine (CAF), producing synergism on induction of SCEs and suppressing the mitotic index; and in combination with MELPH and CAF, producing cell-cycle delays and reducing the mitotic index."( Induction of sister-chromatid exchanges and cell-cycle delays in human lymphocytes by vitamin A alone or in combination with melphalan and caffeine.
Dozi-Vassiliades, J; Granitsas, A; Mourelatos, D; Myrtsiotis, A, 1985
)
0.72
" When ibuprofen was given in combination with a cytotoxic drug, the primary cytoreductive effect was that of the cytotoxic agent."( Response of human adenocarcinoma to chemotherapy: as sole agents and in combination with sodium ibuprofen.
Braly, PS; DiSaia, PJ; Stratton, JA, 1984
)
0.27
" In this study we determined the activity and toxicity of melphalan in combination with another GST-activity inhibitor, sulfasalazine, an agent used to treat ulcerative colitis."( Activity of melphalan in combination with the glutathione transferase inhibitor sulfasalazine.
Awasthi, S; Awasthi, YC; Fields, L; Gupta, V; Jacobs, S; Jani, JP; Levitt, M; Singh, SV; Sreevardhan, M; Xu, BH, 1995
)
0.91
" We have used clonogenic cell survival assays and DNA flow cytometry to examine the effect of paclitaxel combined with melphalan, thiotepa, or cisplatin on the survival and cell-cycle parameters of human lung A549 and breast MCF-7 adenocarcinoma cells."( Sequence dependence of paclitaxel (Taxol) combined with cisplatin or alkylators in human cancer cells.
Cook, JA; Fisher, J; Liebmann, JE; Teague, D, 1994
)
0.5
"In vitro and clinical studies have shown antineoplastic effects of hyperthermia alone and in combination with other treatment modalities."( Total body hyperthermia in combination with etoposide and melphalan in a child with acute myelomonocytic leukemia.
Dehnen, H; Göbel, U; Jürgens, H; Wessalowski, R; Willnow, U, 1994
)
0.53
"Three hundred thirty-five previously untreated patients with multiple myeloma in clinical stages II and III entered a randomized trial comparing intermittent oral melphalan and prednisone (MP) therapy (n = 171) with MP in combination with natural (leukocyte-derived) alpha-interferon (MP/IFN) (n = 164)."( Natural interferon-alpha in combination with melphalan/prednisone versus melphalan/prednisone in the treatment of multiple myeloma stages II and III: a randomized study from the Myeloma Group of Central Sweden.
Björeman, M; Björkholm, M; Brenning, G; Carlson, K; Celsing, F; Gahrton, G; Grimfors, G; Gyllenhammar, H; Hast, R; Osterborg, A, 1993
)
0.74
"The purpose of the study was to determine the maximum tolerated dose of continuous infusion of high-dose VP-16 in combination with high-dose melphalan (HDM) for conditioning before autologous bone marrow transplantation (ABMT)."( Phase I study of high-dose continuous intravenous infusion of VP-16 in combination with high-dose melphalan followed by autologous bone marrow transplantation in children with stage IV neuroblastoma.
Benhamou, E; Bonnay, M; Couanet, D; Hartmann, O; Plantaz, D; Pondarré, C; Valteau-Couanet, D; Vassal, G, 1996
)
0.71
"To determine the efficacy of isolated limb perfusion (ILP) with tumor necrosis factor-alpha (TNF) in combination with interferon-gamma (IFN) and melphalan as induction therapy to render tumors resectable and avoid amputation in patients with nonresectable extremity soft tissue sarcomas (STS)."( Isolated limb perfusion with high-dose tumor necrosis factor-alpha in combination with interferon-gamma and melphalan for nonresectable extremity soft tissue sarcomas: a multicenter trial.
Eggermont, AM; Hoekstra, HJ; Kroon, BB; Lejeune, FJ; Liénard, D; Schraffordt Koops, H; van Geel, AN, 1996
)
0.71
" We report the use of high-dose carboplatin, with the dose based on glomerular filtration rate (GFR), combined with melphalan followed by autologous stem cell rescue in children with advanced stage or chemoresistant solid tumours."( Melphalan combined with a carboplatin dose based on glomerular filtration rate followed by autologous stem cell rescue for children with solid tumours.
Pinkerton, CR; Shaw, PJ; Yaniv, I, 1996
)
1.95
" We determined the cytotoxicity of BSO (dose range 0-1000 microM) alone and in combination with L-PAM (dose range 0-0 microM) in a panel of 18 human neuroblastoma cell lines."( Buthionine sulphoximine alone and in combination with melphalan (L-PAM) is highly cytotoxic for human neuroblastoma cell lines.
Anderson, CP; Chan, W; Forman, HJ; Lui, RM; Park, CK; Reynolds, CP; Tian, L; Tsai, J, 1997
)
0.55
"The most promising developments in the field of isolated limb perfusion have centred around the use of the recombinant cytokine tumour necrosis factor-alpha (rTNF-alpha) in combination with melphalan."( Lack of antitumour activity of human recombinant tumour necrosis factor-alpha, alone or in combination with melphalan in a nude mouse human melanoma xenograft system.
Altermatt, HJ; Althaus, U; Furrer, M; Lejeune, FJ; Liénard, D; Ris, HB; Rüegg, C, 1997
)
0.7
"Treatment with (131)I-MIBG in combination with myeloablative chemotherapy and hematopoietic stem-cell rescue is feasible with acceptable toxicity."( Pilot study of iodine-131-metaiodobenzylguanidine in combination with myeloablative chemotherapy and autologous stem-cell support for the treatment of neuroblastoma.
Braun, T; Ferrara, JL; Hubers, D; Hutchinson, RJ; Levine, JE; Matthay, KK; Shapiro, B; Shulkin, BL; Sisson, JC; Spalding, S; Yanik, GA, 2002
)
0.31
" The majority of patients received total body irradiation (TBI) combined with either melphalan (56."( Analysis of outcome following allogeneic haemopoietic stem cell transplantation for myeloma using myeloablative conditioning--evidence for a superior outcome using melphalan combined with total body irradiation.
Cavenagh, J; Cook, G; Hunter, A; Hunter, HM; Littlewood, T; Mahendra, P; Marks David, I; Pagliuca, A; Peggs, K; Potter, M; Powles, R; Rahemtulla, A; Russell, NH; Towlson, K; Williams, CD, 2005
)
0.75
"The purpose of this study was to define the maximal tolerated dose (MTD), extramedullary toxicities, and pharmacokinetics of docetaxel combined with high-dose melphalan and carboplatin with autologous hematopoietic progenitor cell support."( Phase I and pharmacokinetic study of docetaxel combined with melphalan and carboplatin, with autologous hematopoietic progenitor cell support, in patients with advanced refractory malignancies.
Barón, AE; Bearman, SI; Cagnoni, PJ; Gustafson, D; Jones, RB; Long, M; Matthes, S; McSweeney, PA; Nieto, Y; Shpall, EJ, 2005
)
0.77
"To evaluate the potential benefit of histamine combined with melphalan in the isolated limb perfusion (ILP) as an alternative to TNF-alfa and melphalan combination, for the treatment of irresectable soft tissue sarcomas of the limbs in Brown Norway (BN) rats."( Histamine combined with melphalan in isolated limb perfusion for the treatment of locally advanced soft tissue sarcomas: preclinical studies in rats.
Brunstein, F; Eggermont, AM; Ferreira, LM; Santos, ID; Ten Hagen, TL; van Tiel, ST,
)
0.68
"Histamine combined with melphalan had a promising effect in the ILP warranting future studies to better explore the mechanism of action as well as its potential use in organ perfusion."( Histamine combined with melphalan in isolated limb perfusion for the treatment of locally advanced soft tissue sarcomas: preclinical studies in rats.
Brunstein, F; Eggermont, AM; Ferreira, LM; Santos, ID; Ten Hagen, TL; van Tiel, ST,
)
0.75
" After 9 months, the disease progressed and several chemotherapy agents, including three courses of rituximab combined with etoposide, sobuzoxane or methotrexate, only resulted in a stable disease response."( Rituximab combined with a small dose of melphalan for a refractory follicular lymphoma patient.
Chinzei, T; Kawano, S; Kondo, S; Kumagai, S; Okamura, A; Okumachi, Y; Saigo, K; Takenokuchi, M, 2006
)
0.6
" This effect was less pronounced when FK866 was used in combination with another alkylating agent, melphalan."( Chemopotentiating effects of a novel NAD biosynthesis inhibitor, FK866, in combination with antineoplastic agents.
Azzam, K; Hasmann, M; Nüssler, V; Pelka-Fleischer, R; Pogrebniak, A; Schemainda, I, 2006
)
0.55
"The purpose of this trial was to define the maximum tolerated duration (MTD), dose-limiting toxicity (DLT), regimen-related toxicities (RRT), and pharmacokinetics of gemcitabine infused at a fixed dose rate (FDR) of 10 mg/m2/min, combined with docetaxel/melphalan/carboplatin, using autologous stem cell transplantation (ASCT)."( Phase I and pharmacokinetic study of gemcitabine administered at fixed-dose rate, combined with docetaxel/melphalan/carboplatin, with autologous hematopoietic progenitor-cell support, in patients with advanced refractory tumors.
Aldaz, A; Aramendía, JM; Aristu, J; Centeno, C; Hernández, M; Moreno, M; Nieto, Y; Pérez-Calvo, J; Rifón, J; Sayar, O; Viteri, S; Viúdez, A; Zafra, A; Zufia, L, 2007
)
0.73
"Treatment of tumor-bearing mice with mouse (m)TNF-alpha, targeted to tumor vasculature by the anti-ED-B fibronectin domain antibody L19(scFv) and combined with melphalan, induces a therapeutic immune response."( Therapy-induced antitumor vaccination by targeting tumor necrosis factor alpha to tumor vessels in combination with melphalan.
Accolla, RS; Balza, E; Borsi, L; Carnemolla, B; Castellani, P; De Lerma Barbaro, A; Fossati, S; Mortara, L; Sassi, F; Tosi, G, 2007
)
0.75
" Considered as a semi-malignant disease, CA is often a contraindication for HTx; however, depending on the type of CA, there are excellent treatment regimes that can be combined with HTx."( Treatment options for severe cardiac amyloidosis: heart transplantation combined with chemotherapy and stem cell transplantation for patients with AL-amyloidosis and heart and liver transplantation for patients with ATTR-amyloidosis.
Dengler, T; Goldschmidt, H; Karck, M; Koch, A; Kristen, A; Sack, FU; Schnabel, PA, 2008
)
0.35
"Cardiac amyloidosis is a potentially curative disease after HTx when combined with either chemotherapy and SCT or LiverTx depending on the type of the amyloidosis."( Treatment options for severe cardiac amyloidosis: heart transplantation combined with chemotherapy and stem cell transplantation for patients with AL-amyloidosis and heart and liver transplantation for patients with ATTR-amyloidosis.
Dengler, T; Goldschmidt, H; Karck, M; Koch, A; Kristen, A; Sack, FU; Schnabel, PA, 2008
)
0.35
"To determine the maximum-tolerated radiation-absorbed dose (RAD) to critical organs delivered by yttrium-90 ((90)Y) ibritumomab tiuxetan in combination with high-dose carmustine, etoposide, cytarabine, and melphalan (BEAM) chemotherapy with autologous transplantation."( Yttrium-90 ibritumomab tiuxetan doses calculated to deliver up to 15 Gy to critical organs may be safely combined with high-dose BEAM and autologous transplantation in relapsed or refractory B-cell non-Hodgkin's lymphoma.
Erwin, W; Evens, AM; Gordon, LI; Inwards, DJ; Mehta, J; Micallef, I; Molina, A; Patton, D; Rademaker, AW; Singhal, S; Spies, S; Tallman, MS; Weitner, BB; White, CA; Williams, SF; Winter, JN; Wiseman, G; Zimmer, M, 2009
)
0.54
"Dose-escalated (90)Y ibritumomab tiuxetan may be safely combined with high-dose BEAM with autologous transplantation and has the potential to be more effective than standard-dose radioimmunotherapy."( Yttrium-90 ibritumomab tiuxetan doses calculated to deliver up to 15 Gy to critical organs may be safely combined with high-dose BEAM and autologous transplantation in relapsed or refractory B-cell non-Hodgkin's lymphoma.
Erwin, W; Evens, AM; Gordon, LI; Inwards, DJ; Mehta, J; Micallef, I; Molina, A; Patton, D; Rademaker, AW; Singhal, S; Spies, S; Tallman, MS; Weitner, BB; White, CA; Williams, SF; Winter, JN; Wiseman, G; Zimmer, M, 2009
)
0.35
" A phase 1 dose escalation study to evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of systemic ADH-1 in combination with melphalan via isolated limb infusion in patients with in-transit extremity melanoma was performed."( A phase 1 study of systemic ADH-1 in combination with melphalan via isolated limb infusion in patients with locally advanced in-transit malignant melanoma.
Augustine, CK; Beasley, GM; McMahon, N; Norris, R; Peters, WP; Peterson, B; Ross, MI; Sanders, G; Selim, MA; Tyler, DS, 2009
)
0.8
"Systemic ADH-1 at a dose of 4000 mg on Days 1 and 8 in combination with melphalan via isolated limb infusion is a well-tolerated, novel targeted therapy approach to regionally advanced melanoma."( A phase 1 study of systemic ADH-1 in combination with melphalan via isolated limb infusion in patients with locally advanced in-transit malignant melanoma.
Augustine, CK; Beasley, GM; McMahon, N; Norris, R; Peters, WP; Peterson, B; Ross, MI; Sanders, G; Selim, MA; Tyler, DS, 2009
)
0.83
" The results of five phase III trials assessing thalidomide in combination with melphalan and prednisone (MPT) have demonstrated significantly improved response rates compared with melphalan and prednisone (MP) alone."( Consensus guidelines for the optimal management of adverse events in newly diagnosed, transplant-ineligible patients receiving melphalan and prednisone in combination with thalidomide (MPT) for the treatment of multiple myeloma.
Bladé, J; Davies, F; Delforge, M; Facon, T; Garcia Sanz, R; Kropff, M; Leal da Costa, F; Moreau, P; Morgan, G; Palumbo, A; Schey, S, 2010
)
0.79
" This phase 1/2 dose-escalation study aimed to determine the maximum tolerated dose (MTD) of lenalidomide in combination with melphalan and dexamethasone (M-dex), and assess the efficacy and tolerability of this therapy for patients with de novo AL amyloidosis."( Lenalidomide in combination with melphalan and dexamethasone in patients with newly diagnosed AL amyloidosis: a multicenter phase 1/2 dose-escalation study.
Alakl, M; Benboubker, L; Bridoux, F; Fermand, JP; Harousseau, JL; Hermine, O; Jaccard, A; Leblond, V; Leleu, X; Moreau, P; Planche, L; Roussel, M; Royer, B; Salles, G, 2010
)
0.85
" In a preclinical animal model, systemic ADH-1 given with regional melphalan demonstrated synergistic antitumor activity, and in a phase I trial with M-ILI it had minimal toxicity."( Prospective multicenter phase II trial of systemic ADH-1 in combination with melphalan via isolated limb infusion in patients with advanced extremity melanoma.
Augustine, CK; Beasley, GM; Grobmyer, SR; Hochwald, SN; Peterson, B; Riboh, JC; Ross, MI; Royal, R; Tyler, DS; Zager, JS, 2011
)
0.83
" In this study, by applying the US-MB strategy for melanoma chemotherapy, we evaluated the antitumor effect of melphalan combined with US-MB on a melanoma cell line (C32) in vitro and in vivo."( Synergistic inhibition of malignant melanoma proliferation by melphalan combined with ultrasound and microbubbles.
Endo, H; Feril, LB; Matsuo, M; Nakayama, J; Ogawa, K; Tachibana, K; Yamaguchi, K, 2011
)
0.82
"This study was designed to evaluate the safety and efficacy of a conventional dose of yttrium-90 ((90)Y) ibritumomab tiuxetan combined with the etoposide rabinoside acytarabine melphalan (BEAM) regimen before autologous stem cell transplantation (ASCT) in chemosensitive relapsed or refractory low-grade B-cell lymphomas."( (90)Y ibritumomab tiuxetan (Zevalin) combined with BEAM (Z -BEAM) conditioning regimen plus autologous stem cell transplantation in relapsed or refractory low-grade CD20-positive B-cell lymphoma. A GELA phase II prospective study.
Bologna, S; Bosly, A; Bouabdallah, K; Brière, J; Coiffier, B; Decaudin, D; Delarue, R; Ghesquières, H; Gisselbrecht, C; Huynh, A; Le Gouill, S; Morschhauser, F; Mounier, N; Ribrag, V; Tilly, H, 2011
)
0.56
"A bioanalytical assay for the new poly(ADP-ribose) polymerase-1 inhibitor olaparib in combination with melphalan was developed and validated."( Liquid chromatography-tandem mass spectrometric assay for the PARP-1 inhibitor olaparib in combination with the nitrogen mustard melphalan in human plasma.
Beijnen, JH; den Hartigh, J; Martens, I; Schellens, JH; Sparidans, RW; Valkenburg-van Iersel, LB, 2011
)
0.79
" In preclinical and clinical studies, panobinostat showed good anti-myeloma activity in combination with several agents."( Phase II study of melphalan, thalidomide and prednisone combined with oral panobinostat in patients with relapsed/refractory multiple myeloma.
Alesiani, F; Ballanti, S; Boccadoro, M; Caraffa, P; Catarini, M; Cavallo, F; Corvatta, L; Gentili, S; Leoni, P; Liberati, AM; Offidani, M; Palumbo, A; Polloni, C; Pulini, S, 2012
)
0.71
"Panobinostat is a histone deacetylase inhibitor that has shown synergistic preclinical anti-myeloma activity when combined with other agents, recently exhibiting synergy with the alkylating agent melphalan (Sanchez et al."( A phase 1/2 study of oral panobinostat combined with melphalan for patients with relapsed or refractory multiple myeloma.
Berenson, JR; Boccia, RV; Dressler, K; Ghazal, HH; Harb, WA; Hilger, JD; Jamshed, S; Kingsley, EC; Matous, J; Nassir, Y; Noga, SJ; Swift, RA; Vescio, R; Yellin, O, 2014
)
0.84
"L19-tumor necrosis factor alpha (L19mTNF-α; L), a fusion protein consisting of mouse TNFα and the human antibody fragment L19 directed to the extra domain-B (ED-B) of fibronectin, is able to selectively target tumor vasculature and to exert a long-lasting therapeutic activity in combination with melphalan (M) in syngeneic mouse tumor models."( Schedule-dependent therapeutic efficacy of L19mTNF-α and melphalan combined with gemcitabine.
Balza, E; Borsi, L; Carnemolla, B; Castellani, P; Mortara, L; Orecchia, P, 2013
)
0.81
"To improve isolated hepatic perfusion (IHP), we performed a phase I dose-escalation study to determine the optimal oxaliplatin dose in combination with a fixed melphalan dose."( Isolated hepatic perfusion with oxaliplatin combined with 100 mg melphalan in patients with metastases confined to the liver: A phase I study.
de Leede, EM; den Hartigh, J; Gelderblom, H; Hartgrink, HH; Kuppen, PJ; Nortier, JW; Tijl, FG; Tollenaar, RA; Vahrmeijer, AL; van de Velde, CJ; van Iersel, LB, 2014
)
0.84
"Between June 2007 and July 2008, 11 patients, comprising of 8 colorectal cancer and 3 uveal melanoma patients and all with isolated liver metastases, were treated with a one hour IHP with escalating doses of oxaliplatin combined with 100 mg melphalan."( Isolated hepatic perfusion with oxaliplatin combined with 100 mg melphalan in patients with metastases confined to the liver: A phase I study.
de Leede, EM; den Hartigh, J; Gelderblom, H; Hartgrink, HH; Kuppen, PJ; Nortier, JW; Tijl, FG; Tollenaar, RA; Vahrmeijer, AL; van de Velde, CJ; van Iersel, LB, 2014
)
0.82
"The purpose of this study is to compare the efficacy and safety of Gefitinib versus VMP in combination with three-dimensional conformal radiotherapy (3D-CRT) for multiple brain metastases from non-small cell lung cancer (NSCLC)."( Comparison of Gefitinib versus VMP in the combination with radiotherapy for multiple brain metastases from non-small cell lung cancer.
Chen, S; Hao, Y; Li, B; Li, L; Liu, C; Ning, F; Wang, F; Yu, Z, 2015
)
0.42
"To evaluate BSO-mediated glutathione (GSH) depletion in combination with L-PAM for children with recurrent or refractory high-risk neuroblastoma (NB) as a means to enhance alkylator sensitivity."( Pilot study of intravenous melphalan combined with continuous infusion L-S,R-buthionine sulfoximine for children with recurrent neuroblastoma.
Anderson, CP; Bailey, HH; Groshen, S; Hasenauer, B; Maris, JM; Matthay, KK; Neglia, JP; Perentesis, JP; Reynolds, CP; Seeger, RC; Villablanca, JG, 2015
)
0.71
"3 (dose level 2) mg/m(2) in combination with melphalan 8 mg/m(2) on days 1-4 and dexamethasone 20 mg on days 1, 2, 4, 5, 8, 9, 11, and 12."( Phase 1 study of bortezomib in combination with melphalan and dexamethasone in Japanese patients with relapsed AL amyloidosis.
Abe, M; Ando, Y; Fuchida, S; Hata, H; Imai, H; Ishida, T; Miyamoto, T; Sawamura, M; Shimazaki, C; Suzuki, K; Takamatsu, H; Yamada, M, 2016
)
0.95
"The aim of this study was to determine the efficacy of 252Californium neutron intracavitary brachytherapy using a two-channel Y applicator combined with external beam radiotherapy for the treatment of endometrial cancer."( Clinical assessment of 252Californium neutron intracavitary brachytherapy using a two-channel Y applicator combined with external beam radiotherapy for endometrial cancer.
Chen, S; Lei, X; Tang, C; Xiong, YL; Xu, WJ; Zhao, KW; Zhou, Q, 2016
)
0.43
"252Californium neutron intracavitary brachytherapy using a two-channel applicator combined with external beam radiotherapy was effective for treating endometrial cancer and the incidence of serious late complications related to this combination was within an acceptable range."( Clinical assessment of 252Californium neutron intracavitary brachytherapy using a two-channel Y applicator combined with external beam radiotherapy for endometrial cancer.
Chen, S; Lei, X; Tang, C; Xiong, YL; Xu, WJ; Zhao, KW; Zhou, Q, 2016
)
0.43
"For treatment of metastatic lung lesions there was used the method of isolated chemoperfusion in combination with metastasectomy."( [Immediate results of isolated chemoperfusion of the lung with melphalan and cisplatin in combination with metastasectomy in treatment for resectable metastatic lung lesions].
Barchuk, AS; Kalinin, PS; Levchenko, EV; Mamontov, OY; Mishchenko, AV; Senchik, KY, 2015
)
0.66
"To evaluate the efficacy and adverse effects of low dose thalidomide (TD) combined with modified VCMP (vincristine+cyclophosphamide+melphalan+prednisone) (TD+mVCMP) and VAD (vincristine+doxorubicin+dexamethsone) (TD+VAD) regimens for treating aged patients with MM."( [Efficacy Comparison of Low dose Thalidomide Combined with Modified VCMP and VAD regimens for Treatment of Aged MM Patients].
Liu, HB; Wang, W, 2016
)
0.64
"Low dose TD combined with modified VCMP regimen for treatment of newly diagnosed aged patients with MM is safe and effective, which may be used as the first line treatment regimen for population in aged MM patients."( [Efficacy Comparison of Low dose Thalidomide Combined with Modified VCMP and VAD regimens for Treatment of Aged MM Patients].
Liu, HB; Wang, W, 2016
)
0.43
" Patients undergoing hematopoietic stem cell transplantation (HSCT) are exposed to various types of drugs, and understanding how these drugs interact is of the utmost importance."( Development of an assay for cellular efflux of pharmaceutically active agents and its relevance to understanding drug interactions.
Andersson, BS; Hassan, M; Valdez, BC, 2017
)
0.46
"0 μM and at 75 nM in combination with cisplatin or melphalan."( Initial testing (stage 1) of M6620 (formerly VX-970), a novel ATR inhibitor, alone and combined with cisplatin and melphalan, by the Pediatric Preclinical Testing Program.
Houghton, PJ; Kang, M; Kurmashev, D; Kurmasheva, RT; Reynolds, CP; Smith, MA; Wu, J, 2018
)
0.94
"For treatment of metastatic lung lesions there was used the method isolated chemoperfusion of the lung in combination with metastasectomy."( [Long-term results of isolated chemoperfusion of the lung with melphalan and cisplatin in combination with metastasectomy in treatment for resectable metastatic lung lesion].
Barchuk, AS; Gorokhov, LV; Kalinin, PS; Lemekhov, VG; Levchenko, EV; Mamontov, OY; Mikhnin, AE; Mishchenko, AV; Senchik, KY; Tin, V; Ven, C,
)
0.37
" To investigate the effects of these compounds on bortezomib's anti-proliferative potency and its intracellular accumulation and potency to inhibit the chymotrypsin-like proteasomal subunit, seven myeloma cell lines were investigated after exposure to bortezomib alone or either combined with adriamycin plus dexamethasone (PAD regimen) or melphalan plus prednisolone (VMP regimen), respectively."( Elucidating the beneficial effects of melphalan, adriamycin, and corticoids in combination with bortezomib against multiple myeloma in vitro.
Burhenne, J; Schäfer, J; Theile, D; Weiss, J, 2019
)
0.96
" The VEO alone and in combination with D-α-tocopheryl polyethylene glycol 1000 succinate (TPGS) has significantly increased the MLN and SVN loading."( Vitamin E Oil Incorporated Liposomal Melphalan and Simvastatin: Approach to Obtain Improved Physicochemical Characteristics of Hydrolysable Melphalan and Anticancer Activity in Combination with Simvastatin Against Multiple Myeloma.
Eswara, BRM; Manjappa, AS; Nagadeepthi, N; Sambamoorthy, U; Sanapala, AK, 2021
)
0.89
" Here, we retrospectively analyze CINV data of 100 patients who received either SEAM (semustine, etoposide, cytarabine, melphalan) or MEL140-200 (high-dose melphalan) before ASCT, evaluate the efficacy and safety of multiple-day administration of fosaprepitant combined with tropisetron and olanzapine (FTO), and compare the results to those of patients who received a standard regimen of aprepitant, tropisetron, and dexamethasone (ATD)."( Multiple-day administration of fosaprepitant combined with tropisetron and olanzapine improves the prevention of nausea and vomiting in patients receiving chemotherapy prior to autologous hematopoietic stem cell transplant: a retrospective study.
Cao, J; Chen, D; Du, X; Hu, Y; Jiang, L; Li, S; Liu, X; Lu, Y; Pei, R; Tang, S; Wang, T; Ye, P; Zhang, P, 2022
)
0.93
" Studies on the interactions of HDACi with PARP inhibitors in hematologic cancers are limited, especially when combined with chemotherapeutic agents."( HDAC inhibitors suppress protein poly(ADP-ribosyl)ation and DNA repair protein levels and phosphorylation status in hematologic cancer cells: implications for their use in combination with PARP inhibitors and chemotherapeutic drugs.
Andersson, BS; Murray, D; Nieto, Y; Valdez, BC; Yuan, B, 2022
)
0.72

Bioavailability

The objectives of the present study were to circumvent the moisture-associated instability, enhance bioavailability and achieve enhanced passive targeting of melphalan to the ovaries. Cimetidine pretreatment reduced the bioavailability of oralmelphalan by approximately 30% (P less than 0.1)

ExcerptReferenceRelevance
" Some instances of failure of tumor response to oral melphalan may be due to inadequate bioavailability rather than inherent tumor resistance."( Oral melphalan kinetics.
Alberts, DS; Chang, SY; Chen, HS; Evans, TL; Moon, TE, 1979
)
1.02
" The bioavailability showed large interindividual variations, and was not significantly affected by the dose given."( Oral melphalan pharmacokinetics--relation to dose in patients with multiple myeloma.
Ehrsson, H; Eksborg, S; Lindfors, A; Mellstedt, H; Osterborg, A, 1989
)
0.79
" The oral bioavailability of BSO, based on plasma BSO levels, was extremely low."( Pharmacokinetics of buthionine sulfoximine (NSC 326231) and its effect on melphalan-induced toxicity in mice.
Grieshaber, CK; Liao, JT; Page, JG; Smith, AC; Wientjes, MG, 1989
)
0.51
"Previous studies have demonstrated that the bioavailability of melphalan and chlorambucil may be reduced under non-fasting conditions, and that the gastrointestinal and cellular absorption of melphalan is an active process, while that of chlorambucil is passive."( The effect of dietary amino acids on the gastrointestinal absorption of melphalan and chlorambucil.
Adair, CG; McElnay, JC, 1987
)
0.74
" Flurbiprofen did not seem to alter the bioavailability of the chemotherapeutic agents."( Increased survival of cancer-bearing mice treated with inhibitors of prostaglandin synthesis alone or with chemotherapy.
Bennett, A; Berstock, DA; Carroll, MA, 1982
)
0.26
"This study deals with the design of a new macrofilaricidal drug derived from melphalan and having a lymphotropism to avoid the hepatic first pass effect and enhance bioavailability after oral administration."( Study of lymphotropic targeting and macrofilaricidal activity of a melphalan prodrug on the Molinema dessetae model.
Deverre, JR; el Kihel, L; Gayral, P; Letourneux, Y; Loiseau, PM, 1994
)
0.75
" Although intracellular events involved in the pharmacologic action of these compounds have been extensively studied, the role of plasma membrane transporters in nucleoside-derived drug bioavailability and action in leukemia cells has not been comprehensively addressed."( Fludarabine uptake mechanisms in B-cell chronic lymphocytic leukemia.
Bellosillo, B; Casado, FJ; Colomer, D; Gil, J; Molina-Arcas, M; Montserrat, E; Pastor-Anglada, M, 2003
)
0.32
" Human oral bioavailability is an important pharmacokinetic property, which is directly related to the amount of drug available in the systemic circulation to exert pharmacological and therapeutic effects."( Hologram QSAR model for the prediction of human oral bioavailability.
Andricopulo, AD; Moda, TL; Montanari, CA, 2007
)
0.34
" These "in combo" PAMPA data were used to predict the human absolute bioavailability of the ampholytes."( The permeation of amphoteric drugs through artificial membranes--an in combo absorption model based on paracellular and transmembrane permeability.
Avdeef, A; Sun, N; Tam, KY; Tsinman, O, 2010
)
0.36
"Defibrotide is a novel orally bioavailable polydisperse oligonucleotide with anti-thrombotic and anti-adhesive effects."( Melphalan, prednisone, thalidomide and defibrotide in relapsed/refractory multiple myeloma: results of a multicenter phase I/II trial.
Anderson, K; Benevolo, G; Boccadoro, M; Bringhen, S; Cavallo, F; Gaidano, G; Gay, F; Genuardi, M; Iacobelli, M; Kotwica, K; Larocca, A; Magarotto, V; Masini, L; Mitsiades, C; Palumbo, A; Richardson, P; Rossi, D; Rus, C, 2010
)
1.8
" INCB16562 was developed as a novel, selective, and orally bioavailable small-molecule inhibitor of JAK1 and JAK2 markedly selective over JAK3."( INCB16562, a JAK1/2 selective inhibitor, is efficacious against multiple myeloma cells and reverses the protective effects of cytokine and stromal cell support.
Arvanitis, A; Caulder, E; Combs, AP; Favata, M; Fridman, JS; Kelley, JA; Li, J; Newton, R; Rogers, JD; Scherle, PA; Solomon, KA; Sparks, RB; Thomas, B; Vaddi, K; Wen, X, 2010
)
0.36
" CYT387 is a novel, orally bioavailable JAK1/2 inhibitor, which has recently been described."( The novel JAK inhibitor CYT387 suppresses multiple signalling pathways, prevents proliferation and induces apoptosis in phenotypically diverse myeloma cells.
Burns, CJ; Khong, T; Monaghan, KA; Spencer, A, 2011
)
0.37
"The objectives of the present study were to circumvent the moisture-associated instability, enhance bioavailability and achieve enhanced passive targeting of melphalan to the ovaries."( Anticancer efficacy, tissue distribution and blood pharmacokinetics of surface modified nanocarrier containing melphalan.
Bali, V; Pathak, K; Rajpoot, P, 2012
)
0.79
" Here, we analyzed the effects of the novel, orally bioavailable HSP90 inhibitor NVP-HSP990 on MM cell proliferation and survival."( The novel, orally bioavailable HSP90 inhibitor NVP-HSP990 induces cell cycle arrest and apoptosis in multiple myeloma cells and acts synergistically with melphalan by increased cleavage of caspases.
Gao, Z; Heider, U; Jensen, MR; Kaiser, M; Lamottke, B; Mieth, M; Nikolova, Z; Sezer, O; Sterz, J; von Metzler, I, 2012
)
0.58
"The ATP-binding cassette transporter P-glycoprotein (P-gp) is known to limit both brain penetration and oral bioavailability of many chemotherapy drugs."( A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
Ambudkar, SV; Brimacombe, KR; Chen, L; Gottesman, MM; Guha, R; Hall, MD; Klumpp-Thomas, C; Lee, OW; Lee, TD; Lusvarghi, S; Robey, RW; Shen, M; Tebase, BG, 2019
)
0.51
" The therapeutic effects of drug molecules are majorly dependent on the bioavailability and, in essence, on the solubility of the used drug molecules."( Exploration on the drug solubility enhancement in aqueous medium with the help of endo-functionalized molecular tubes: a computational approach.
Paul, R; Paul, S, 2021
)
0.62

Dosage Studied

Melphalan should be dosed on a weight basis, and treatment groups should be stratified by weight in randomized clinical studies. There was statistically significant greater myelosuppression, stomatitis, and diarrhea in the very high dosage DTIC and melphalan (Regimen A) compared with the other two regimens. A risk-adapted approach to melphAlan dosing with PBSCT is an effective treatment in patients with primary AL amyloidosis.

ExcerptRelevanceReference
" Because of rapid progression of the amyloidosis and further infections, cytotoxic drug therapy was stopped, corticosteroid dosage was decreased, and supplementary immunoglobulin therapy was instituted."( Amyloidosis associated with dermatomyositis and features of multiple myeloma. The progression of amyloidosis associated with corticosteroid and cytotoxic drug therapy.
Dawkins, RL; Zilko, PJ, 1975
)
0.25
" This combination appears to be safe, provided the field of radiation is not so large that is may add significantly to the myelosuppressive effect of chemotherapy and the dosage of concomitantly administered radiopotentiating agent(s) is reduced."( Feasibility of integration of modalities in melanomas and sarcomas.
Berger, JL; Friedman, M; Holyoke, ED; Karakousis, CP; Lopez, R; Takita, H, 1979
)
0.26
" Leukocyte and platelet counts must be performed every three weeks, and the dosage of melphalan adjusted accordingly."( Treatment of scleromyxedema with melphalan.
Harris, RB; Kyle, RA; Perry, HO; Winkelmann, RK, 1979
)
0.76
" Most patients are put on a fixed oral dosage regimen."( Oral melphalan kinetics.
Alberts, DS; Chang, SY; Chen, HS; Evans, TL; Moon, TE, 1979
)
0.77
"[14C]melphalan ([14C]L-PAM) was rapidly absorbed from the gut of dogs after oral dosing and reached a maximum concentration in the serum by 30 minutes."( Pharmacokinetics of the absorption, distribution, and elimination of melphalan in the dog.
Brown, RK; Duncan, G; Furner, RL, 1977
)
1.01
" The body weight was the same in the two groups, indicating that the patient's weight is of minor importance for the dosage of melphalan."( Factors responsible for bone marrow toxicity after treatment of myeloma patients with different alkylating agents.
Lewensohn, R; Ringborg, U, 1978
)
0.46
" Addition of ethacrynic acid (3 micrograms ml-1) to treatment of the cell lines with melphalan or with cisplatin did not alter the dose-response curves to these agents."( A study of ethacrynic acid as a potential modifier of melphalan and cisplatin sensitivity in human lung cancer parental and drug-resistant cell lines.
Rhodes, T; Twentyman, PR, 1992
)
0.76
" just prior to the injection of melphalan (10 mg/kg), cyclophosphamide (150 mg/kg) or BCNU (50 mg/kg), the greatest tumor growth delays were obtained with dosage levels between 4 g and 12 g of the perfluorochemical perfluorooctyl bromide/kg."( A new concentrated perfluorochemical emulsion and carbogen breathing as an adjuvant to treatment with antitumor alkylating agents.
Ara, G; Ha, CS; Herman, TS; Holden, SA; Northey, D; Teicher, BA, 1992
)
0.57
") was administered just prior to an alkylating agent, the combination treatment produced significantly more tumor cell killing across the dosage range of each alkylating agent tested compared with the alkylating agent alone."( Modulation of alkylating agents by etanidazole and Fluosol-DA/carbogen in the FSaIIC fibrosarcoma and EMT6 mammary carcinoma.
Bubley, G; Coleman, CN; Eder, JP; Frei, E; Herman, TS; Holden, SA; Tanaka, J; Teicher, BA, 1991
)
0.28
" 15% of previously untreated myeloma patients achieve a clinical response to IFN alpha alone with a possible dose-response relationship."( Treatment of multiple myeloma with interferon alpha: the Scandinavian experience.
Björeman, M; Björkholm, M; Brenning, G; Gahrton, G; Grimfors, G; Gyllenhammar, H; Hast, R; Juliusson, G; Mellstedt, H; Osterborg, A, 1991
)
0.28
" In vivo, coadministration of doxorubicin with verapamil increased animal survival when either continuous infusion or bolus dosing regimens were used."( Drug resistance-reversal strategies: comparison of experimental data with model predictions.
Michelson, S; Slate, D, 1991
)
0.28
" When treatment response was related to the dosage of melphalan given by mg/kg of body weight, the numbers of responding and non-responding patients were similar in the group of patients without dose reduction as well as in that with dose reduction."( Oral dosage of melphalan and response to treatment in multiple myeloma.
Fernberg, JO; Johansson, B; Lewensohn, R; Mellstedt, H, 1990
)
0.88
" Melphalan was given at a dosage greater than or equal to 140 mg/m2 followed 24 h later by autologous marrow rescue."( High dose melphalan and autologous marrow rescue in advanced epithelial ovarian carcinomas: a retrospective analysis of 35 patients treated in France.
Bergerat, JP; Dufour, P; Guastalla, JP; Herve, P; Legros, M; Maraninchi, D; Oberling, F; Philip, T; Plagne, R; Viens, P, 1990
)
1.59
" On the other hand, results from isolated limb perfusion for satellitosis and in-transit metastasis suggest distinct dose-response correlations with tumoricidal properties of appropriate antineoplastic agents."( [Systemically administered regional tumor therapy. Regional hemi-body chemotherapy of metastatic malignant melanoma--an experimental therapy concept].
Aigner, KR; Link, KH; Müller, H; Voigt, H; Walther, H, 1989
)
0.28
"A melphalan-resistant human rhabdomyosarcoma xenograft, TE-671 MR, was established in athymic mice by serial melphalan treatment of the parent xenograft, TE-671, at the 10% lethal dosage (LD10); significant resistance was evident after ten passages of the tumor."( Establishment of a melphalan-resistant rhabdomyosarcoma xenograft with cross-resistance to vincristine and enhanced sensitivity following buthionine sulfoximine-mediated glutathione depletion.
Bigner, DD; Bigner, SH; Colvin, OM; Elion, GB; Friedman, HS; Griffith, OW; Horton, JK; Lilley, E; Rosenberg, MC, 1989
)
1.33
" There was statistically significant greater myelosuppression, stomatitis, and diarrhea in the very high dosage DTIC and melphalan (Regimen A) compared with the other two regimens."( High-dose, double alkylating agent chemotherapy with DTIC, melphalan, or ifosfamide and marrow rescue for metastatic malignant melanoma.
Carr, T; Chadwick, G; Craig, P; Jones, R; Lind, M; Morgenstern, G; Thatcher, D, 1989
)
0.73
" A dose-response effect of melphalan against the drug-sensitive plasmacytomas was present in vivo."( Glutathione and glutathione S-transferases in a human plasma cell line resistant to melphalan.
Ahmad, H; Awasthi, YC; Gupta, V; Medh, RD; Singh, SV, 1989
)
0.8
" This complication was no longer seen after adjustment of the dosage and dose distribution of doxorubicin, but the morbidity after perfusion with doxorubicin remained considerable."( Results of regional isolation perfusion with cytostatics in patients with soft tissue tumors of the extremities.
Albus-Lutter, CE; Benckhuijsen, C; Klaase, JM; Kroon, BB; van Geel, AN; Wieberdink, J, 1989
)
0.28
"A previously untreated 31-yr old female with Ph-positive chronic myeloid leukaemia (CML) received busulphan and melphalan at high dosage followed by an autograft of peripheral blood stem cells collected 4 weeks earlier."( Complete remission after autografting for chronic myeloid leukaemia.
Apperley, JF; Brito-Babapulle, F; Dowding, C; Goldman, JM; Guo, AP; Rassool, F, 1987
)
0.48
" Prior to each ABMT patients received massive chemotherapy with melphalan at a dosage of 140 mg/m2."( Repeated courses of high dose melphalan and unpurged autologous bone marrow transplantation in children with acute non-lymphoblastic leukemia in first complete remission.
Blaise, D; Demeocq, F; Gaspard, MH; Gastaut, JA; Lepeu, G; Maraninchi, D; Michel, G; Novakovitch, G; Perrimond, H; Stoppa, AM, 1988
)
0.8
" Throughout the melphalan dosage range examined there is approximately 1 log greater tumor cell kill observed with the addition of Fluosol-DA and carbogen breathing compared to the drug treatment alone."( Approaches to defining the mechanism of enhancement by Fluosol-DA 20% with carbogen of melphalan antitumor activity.
Holden, SA; Jacobs, JL; Teicher, BA, 1987
)
0.84
" In dogs, melphalan should be dosed on a weight basis, and treatment groups should be stratified by weight in randomized clinical studies, particularly when the weight range of treated subjects is great."( Unexpected toxicity associated with use of body surface area for dosing melphalan in the dog.
Allen, SL; Dewhirst, MW; Gillette, EL; Heidner, GL; Macy, DW; McGee, ML; Page, RL; Sim, DA; Thrall, DE, 1988
)
0.91
" Further studies should evaluate different dosing intervals to take advantage of the slower rate of GSH replenishment observed in normal tissues compared to solid tumor cells (Colon 38) in vivo."( Lack of enhanced antitumor efficacy for L-buthionine sulfoximine in combination with carmustine, cyclophosphamide, doxorubicin or melphalan in mice.
Dorr, RT; Soble, MJ,
)
0.34
" The drug sensitivity of myeloma cells (MY-CFUc) compared with normal haemopoietic cells (GM-CFUc) can be measured using dose-response curves in individual patients."( A simple method for culturing myeloma cells from human bone marrow aspirates and peripheral blood in vitro.
Ayliffe, MJ; Bell, JB; Lakhani, A; McElwain, TJ; Millar, BC; Selby, PJ, 1988
)
0.27
"119 dosage required to reduce the viable L1210 cell fraction by 50% (TCD50)."( Multiple transport pathways for L1210 cells: uptake of PTT.119, a bifunctional alkylator with carrier amino acids.
Bekesi, JG; Chin, SE; Holland, JF; Scanlon, KJ; Yagi, MJ, 1988
)
0.27
" These results demonstrate the existence of a dose-response effect of high-dose melphalan regimens in relapsed acute leukemias, without marked increases in nonhematological toxicity with these doses."( High-dose melphalan with or without marrow transplantation: a study of dose-effect in patients with refractory and/or relapsed acute leukemias.
Beaujean, F; Gastaut, JA; Hartmann, O; Hayat, M; Kamioner, D; Maraninchi, D; Mascret, B; Novakovitch, G; Pico, JL; Sebahoun, G, 1986
)
0.9
" In experimental models, a steep dose-response curve for colorectal cancer has been demonstrated using various agents."( Phase II trial of high-dose melphalan and autologous bone marrow transplantation for metastatic colon carcinoma.
Daly, MB; Johnson, DB; Knight, WA; Leff, RS; Messerschmidt, GL; Mosley, KR; Ruxer, RL; Thompson, JM, 1986
)
0.57
" All two-drug combinations required attenuation to one-half the LD10 dosage for each."( Combination chemotherapy with a new folate analog: activity of 10-ethyl-10-deaza-aminopterin compared to methotrexate with 5-fluorouracil and alkylating agents against advanced metastatic disease in murine tumor models.
DeGraw, JI; Otter, GM; Schmid, FA; Sirotnak, FM,
)
0.13
"Since the extent of host toxicity of cytostatics is considerably affected by dosing time, a chronopharmacologic approach was undertaken for optimizing the therapeutic index of the alkylating agent, peptichemio (PTC)."( Circadian time dependence of murine tolerance for the alkylating agent peptichemio.
Bailleul, F; Horvath, C; Lemaigre, G; Levi, F; Mathe, G; Mechkouri, M; Reinberg, A; Roulon, A, 1987
)
0.27
" The resulting variation in total melphalan dosage gave rise to varying drug concentrations in the perfusate as the extracorporeal system was operated with a fixed volume of priming fluid."( Pharmacokinetics of melphalan in isolated perfusion of the limbs.
Benckhuijsen, C; Hart, AA; Noordhoek, J; Varossieau, FJ; Wieberdink, J, 1986
)
0.87
" A dose-response relationship was apparent in 605 women receiving melphalan and suggested in 333 women receiving cyclophosphamide."( Melphalan may be a more potent leukemogen than cyclophosphamide.
Bennett, JM; Boice, JD; Dembo, AJ; Gershenson, DM; Greene, MH; Harris, EL; Malkasian, GD; Melton, LJ; Moloney, WC, 1986
)
1.95
" Melphalan dosage was calculated per ml of blood and applied at 20 to 40 micrograms/ml."( A simple and accurate new method for cytostatics dosimetry in isolation perfusion of the limbs based on exchangeable blood volume determination.
Ghanem, GE; Lejeune, FJ, 1987
)
1.18
" Different dosing regimens of BSO were found to potentiate L-PAM toxicity in a manner that depended upon the degree of GSH depletion."( Chemosensitization of L-phenylalanine mustard by the thiol-modulating agent buthionine sulfoximine.
Ahmad, S; Greene, K; Kramer, RA; Vistica, DT, 1987
)
0.27
" Despite an average increase of melphalan dosage of 18% above the maximum for iliac perfusions recommended in the literature, no increase in toxic tissue reactions was observed after hyperthermic iliac perfusions."( Dosimetry of cytostatics in hyperthermic regional isolated perfusion.
Oldhoff, J; Schraffordt Koops, H; van Os, J, 1985
)
0.55
" Dose-response studies failed to demonstrate cross-resistance to the tripeptide by L-PAM resistant cells."( PTT.119, p-F-Phe-m-bis-(2-chloroethyl)amino-L-Phe-Met ethoxy HCl, a new chemotherapeutic agent active against drug-resistant tumor cell lines.
Bekesi, JG; Chin, SE; Holland, JF; Scanlon, KJ; Yagi, MJ, 1985
)
0.27
" The steep dose-response relationships in other lines support the rationale for high-dose therapy either by intraperitoneal or systemic administration of drugs."( Pharmacologic reversal of drug resistance in ovarian cancer.
Ozols, RF, 1985
)
0.27
" In patients receiving melphalan RFS was not significantly affected by either the occurrence of amenorrhoea or the dosage of melphalan received."( Controlled trial of adjuvant chemotherapy with melphalan for breast cancer.
Bulbrook, RD; Bush, H; Chaudary, M; Crowther, D; Fentiman, IS; George, WD; Hayward, JL; Howat, JM; Howell, A; Knight, RK; Rubens, RD; Sellwood, RA, 1983
)
0.83
" Bleomycin's and melphalan's reduced systemic availability after IP dosing suggests that their dose could be increased safely by a factor of two over their standard IV doses."( The disposition of intraperitoneal bleomycin, melphalan, and vinblastine in cancer patients.
Alberts, DS; Chang, SY; Chen, HS; Peng, YM, 1980
)
0.86
" A similar dose-response curve was obtained with melphalan for each of the four xenografts, despite previous treatment with an alkylating agent in two of the patients from whom the xenografts originated."( Cell survival in four ovarian carcinoma xenografts following in vitro exposure to melphalan, cisplatin and cis-diammine-1,1-cyclobutane dicarboxylate platinum II (CBDCA,JM8).
Jones, AC; Steel, GG; Wilson, PA, 1984
)
0.75
"One hundred and fifty-six patients with extremity melanomas of known level or thickness who were perfused prophylactically with l-phenylalanine mustard (1-PAM) between January 1974 and December 1978 were studied retrospectively to determine the effect of variation of drug dosage and temperature on regional toxicity and disease control."( Effect of variation of drug dosage on disease control and regional toxicity in prophylactic perfusion for Stage I extremity melanoma.
Ames, FC; Boddie, AW; McBride, CM; Mikhail, RA; Zimmerman, SO, 1984
)
0.27
" However, a chronic low dosing schedule of MISO did not affect the plasma half-life of either cytotoxic drug, although a significant potentiation of each drug in combination with a chronic MISO dose has been obtained in some tumours."( The effect of radiosensitizers on the pharmacokinetics of melphalan and cyclophosphamide in the mouse.
Hinchliffe, M; McNally, NJ; Stratford, MR, 1983
)
0.51
" Cytostatics are dosed per liter of perfused extremity."( [Technic of isolated perfusion of the extremities. Experience with 171 cases].
Aigner, K; Schwemmle, K, 1983
)
0.27
"The pharmacokinetics of melphalan have been studied after intravenous and oral dosing (10 mg) in 6 patients with multiple myeloma."( The pharmacokinetics of melphalan in patients with multiple myeloma: an intravenous/oral study using a conventional dose regimen.
Hamilton, P; Lennard, A; Rawlins, MD; Woodhouse, KW, 1983
)
0.88
"The optimal single dosage of melphalan in isolation perfusion of the limbs for malignant melanoma was assessed."( Dosimetry in isolation perfusion of the limbs by assessment of perfused tissue volume and grading of toxic tissue reactions.
Benckhuysen, C; Braat, RP; Olthuis, GA; van Slooten, EA; Wieberdink, J, 1982
)
0.56
"Twenty-three patients with Stage III, Stage IV, or recurrent epithelial ovarian cancer were treated with a combination of melphalan and levamisole to determine a tolerable dosage schedule, possible adverse effects, and a general estimate of response rate and duration."( Chemotherapy of advanced ovarian epithelial carcinoma with melphalan and levamisole: a pilot study of the Gynecologic Oncology Group.
Di Saia, PJ; Gusdon, JP; Heise, ER; Herbst, GA; Homesley, HD; Lovelace, JV; Muss, HB; Richards, F; Spurr, CL, 1981
)
0.71
" There were no differences in response to therapy or in survival between the patients treated with melphalan followed by 5-fluorouracil and then by methotrexate in high dosage and the patients treated with the same agents in combination."( Combination v. sequential therapy with melphalan, 5-fluorouracil and methotrexate for advanced ovarian cancer.
Boyes, DA; Dodds, DJ; Gerulath, A; Kirk, ME; Klaassen, DJ; Levitt, M; Miller, AB; Pearson, JG; Wall, C, 1980
)
0.75
" To obtain dose-response curves and quantitative comparisons of different treatments, an agar-colony assay was used to measure survival of cells from excised tumours."( Response of two mouse tumours to hyperthermia with CCNU or melphalan.
Joiner, MC; Steel, GG; Stephens, TC, 1982
)
0.51
" Regrowth delay has been used as the assay, and by producing dose-response curves the effect has been classified as additive or interactive."( Chemosensitization of mouse tumors by misonidazole.
Denekamp, J; Randhawa, VS; Stewart, FA,
)
0.13
" Dose-response studied indicated that melphalan concentrations of 2 X 10(-8) M had no effect, while concentrations of 8 X 10(-7) M were totally toxic, after 72-hr exposures to the drug."( Decreased immunoglobulin production by a human lymphoid cell line following melphalan treatment.
Atchley, CE; Griffin, GD; Novelli, GD; Owen, BA; Solomon, A, 1982
)
0.76
" The differences in drug exposure and in cellular chemosensitivity between chambers and tumors suggest caution in the interpretation of drug testing using this system, but the log-linear nature of the dose-response curves is an important feature which may be useful in the eventual development of optimal chemosensitivity testing systems."( Use of the agar diffusion chamber for the exposure of human tumor cells to drugs.
Selby, PJ; Steel, GG, 1982
)
0.26
" Combination chemotherapy of tumor bearing mice with thiamine and mechlorethamine increased the mechlorethamine dosage required for a 50 to 60 percent increase in survival time but did not improve survival over that obtained with mechlorethamine alone."( Thiamine protection of murine L1210 leukemia cells against mechlorethamine cytotoxicity and its relation to the choline uptake system.
Naujokaitis, SA, 1981
)
0.26
" While earlier results suggested that adjuvant chemotherapy is especially effective in premenopausal women, newer studies and analyses indicate that appropriate dosage and consistent administration of chemotherapy are of decisive importance."( [Results of, and indications for adjuvant chemotherapy in breast cancer].
Brunner, KW; Goldhirsch, A; Joss, R; Sonntag, RW; Tschopp, L, 1981
)
0.26
" The extent of melphalan cross-linking was dependent on both drug dosage and exposure time."( L-phenylalanine mustard (melphalan) uptake and cross-linking in the RPMI 6410 human lymphoblastoid cell line.
Belch, A; Brox, LW; Gowans, B, 1980
)
0.92
" Dose-response curves were obtained and the surviving fraction at drug levels estimated to be achieved in man was used as a measure of in vitro drug sensitivity."( In vitro chemosensitivity tests on xenografted human melanomas.
Bateman, AE; Selby, PJ; Steel, GG; Towse, GD, 1980
)
0.26
" Time-dependent pharmacodynamic models are seen as a powerful means to design dosing regimens and to provide a mathematical platform for mechanistic based models."( Time-dependent pharmacodynamic models in cancer chemotherapy: population pharmacodynamic model for glutathione depletion following modulation by buthionine sulfoximine (BSO) in a Phase I trial of melphalan and BSO.
Brennan, J; Gallo, JM; Halbherr, T; Hamilton, TC; Laub, PB; O'Dwyer, PJ; Ozols, RF, 1995
)
0.48
"Melphalan has a steep dose-response curve, but the use of high doses results in unacceptable myelosuppression."( A phase I trial of amifostine (WR-2721) and melphalan in children with refractory cancer.
Adamson, PC; Balis, FM; Belasco, JE; Berg, SL; Blaney, SM; Craig, C; Lange, B; Poplack, DG, 1995
)
2
" The A2058 line showed the lowest level of synergism with hyperthermia, and displayed a marked plateau at 10% of controls in the dose-response for survival, yet no melphalan-resistant subpopulation could be isolated."( Melphalan uptake, hyperthermic synergism and drug resistance in a human cell culture model for the isolated limb perfusion of melanoma.
Addison, RS; Clark, J; Grabs, AJ; Parsons, PG; Roberts, MS; Smithers, BM, 1994
)
1.93
" The responses of the cell lines to TNF were in the rank order of 939 cells > 987 > 284 > C8161 > 852 > A2058 approximately 0, and all displayed shallow dose-response curves; no significant thermal enhancement of TNF cytotoxicity was apparent with this heat dose."( Tetramodality treatment of human melanoma in vitro.
Auzenne, E; Feig, B; Klostergaard, J; Ross, MI; Tomasovic, SP, 1995
)
0.29
"Melphalan's lack of efficacy at the doses administered does not disprove the steep chemotherapy dose-response relationship postulated for many solid tumors."( Phase II trial of intravenous melphalan in advanced colorectal carcinoma.
Abbruzzese, JL; Moore, DF; Pazdur, R, 1994
)
2.02
"In order to perform melphalan dosage adjustment, the linearity of melphalan kinetics was studied, in the case of previous carboplatin administration."( [Effect of carboplatin on the pharmacokinetics of melphalan administered intravenously].
Ardiet, C; Biron, P; Bouffet, E; Brunat-Mentigny, M; Nasri, F; Philip, I; Tranchand, B, 1994
)
0.87
" Melphalan dosage was 30 mg/m2 every three weeks."( Phase II study of intravenous melphalan (NSC-8806) in the treatment of patients with advanced squamous carcinoma of the head and neck.
Blough, R; Grinblatt, D; Kies, MS; Runge-Morris, M; Taylor, S; Watkins, A, 1994
)
1.49
" The first series of 12 patients received a total dosage TNF-alpha of 2-4 mg, and the second series of 10 cases received an escalating dosage of TNF-alpha (1."( Treatment of in-transit metastases from cutaneous melanoma by isolation perfusion with tumour necrosis factor-alpha (TNF-alpha), melphalan and interferon-gamma (IFN-gamma). Dose-finding experience at the National Cancer Institute of Milan.
Belli, F; Inglese, MG; Manzi, R; Persiani, L; Santinami, M; Santoro, N; Vaglini, M, 1994
)
0.49
" Nevertheless, we found a dose-response effect in these patients with relapsed and resistant Hodgkin's disease."( Dose intensification with autologous bone-marrow transplantation in relapsed and resistant Hodgkin's disease: results of a BNLI randomised trial.
Chopra, R; Goldstone, AH; Hancock, B; Hudson, GV; Linch, DC; McMillan, A; Milligan, D; Moir, D; Winfield, D, 1993
)
0.29
" Maximal GSH depletion (40% of baseline values, absolute values 200 to 250 ng/10(6) PBLs) was noted after the sixth BSO dose; extended BSO dosing schedules beyond six total BSO doses did not further deplete GSH."( Phase I clinical trial of intravenous L-buthionine sulfoximine and melphalan: an attempt at modulation of glutathione.
Alberti, D; Arzoomanian, RZ; Bailey, HH; Mulcahy, RT; Pomplun, M; Spriggs, DR; Tombes, MB; Tutsch, KD; Wilding, G, 1994
)
0.52
" We conclude that, in multidrug therapies, the continuation of corticosteroid at conventional dosage beyond the first course does not improve response rate or survival time in multiple myeloma."( Corticosteroid is not beneficial in multiple-drug combination chemotherapy for multiple myeloma. Finnish Leukaemia Group.
Ala-Harja, K; Almqvist, A; Elonen, E; Hallman, H; Hänninen, A; Ilvonen, M; Isomaa, B; Järvenpää, E; Jouppila, J; Palva, IP, 1993
)
0.29
"To complete research in this type of coagulation and cancer, a multicentric randomized clinical trial was performed, including 303 patients with small cell lung cancer (SCLC), treated by the addition of aspirin at 1 g/day (a dosage at which aspirin is considered to be a platelet aggregation inhibitor) to combined chemotherapy."( No effect of an antiaggregant treatment with aspirin in small cell lung cancer treated with CCAVP16 chemotherapy. Results from a randomized clinical trial of 303 patients. The "Petites Cellules" Group.
Chastang, C; Fabre, C; Lebeau, B; Massin, F; Muir, JF; Vincent, J, 1993
)
0.29
" Melphalan given at the 90% lethal dosage produced severe gastrointestinal mucositis and mortality (13 of 23 treated mice)."( Development of a model of melphalan-induced gastrointestinal toxicity in mice.
Bigner, DD; Castellino, S; Cattley, RC; Dewhirst, M; Elion, GB; Friedman, HS; Griffith, OW; Kurtzberg, J; Scott, P, 1993
)
1.5
" Under schedule B, HDM was followed by IL-3 alone at the same dosage from day 1 to day 3, IL-3 and G-CSF (idem) from day 4 to day 7 and G-CSF alone from day 8 until completion of apheresis."( Mobilization of peripheral blood stem cells with chemotherapy and cytokines in multiple myeloma.
Becker, M; Donatini, B; Eisenmann, JC; Hénon, PR; Sklenar, I; Sovalat, H, 1995
)
0.29
" We measured plasma concentrations of R- and S-BSO by high-performance liquid chromatography (HPLC) in 22 patients throughout the dosing period."( Phase I trial of buthionine sulfoximine in combination with melphalan in patients with cancer.
Bookman, MA; Brennan, J; Comis, RL; Gallo, JM; Halbherr, T; Hamilton, TC; Hoffman, J; Kilpatrick, D; LaCreta, FP; O'Dwyer, PJ; Ozols, RF; Young, RC, 1996
)
0.54
" Using this assay in the present study, dose-response relationships of cytotoxicity, PFC response and histology in the spleen were evaluated in rats receiving alkylating agents."( Dose-response relationships of cytotoxicity, PFC response and histology in the spleen in rats treated with alkylating agents.
Doi, T; Nagai, H; Suzuki, T; Tsukuda, R, 1996
)
0.29
"The optimal dosing schedule for melphalan therapy of recurrent malignant melanoma in isolated limb perfusions has been examined using a physiological pharmacokinetic model with data from isolated rat hindlimb perfusions (IRHP)."( Melphalan dosing regimens for management of recurrent melanoma by isolated limb perfusion: application of a physiological pharmacokinetic model based on melphalan distribution in the isolated perfused rat hindlimb.
Roberts, MS; Smithers, BM; Wu, ZY, 1997
)
2.02
" IFN alpha, at 500 IU/ml, a noncytotoxic dosage significantly increased the cytotoxicity of Mel to K562/Mel."( [Mechanisms of resistance to melphalan in leukemia cell line and reversal by interferon alpha].
Qian, Q; Yan, W; Yang, CZ, 1996
)
0.59
" Combinations of haemopoietic growth factors and their dose-modifications should be investigated to improve PBPC collection, to allow a dosage reduction of the mobilization chemotherapy."( Factors influencing collection of peripheral blood progenitor cells following high-dose cyclophosphamide and granulocyte colony-stimulating factor in patients with multiple myeloma.
Goldschmidt, H; Haas, R; Hegenbart, U; Hohaus, S; Wallmeier, M, 1997
)
0.3
"The availability, mean transit time and normalised variance (CV2) obtained for labelled red blood cells, sucrose and water were similar before and after melphalan dosing and with the two methods of calculation but varied between the patients."( Relative dispersion of intravascular transit times during isolated human limb perfusions for recurrent melanoma.
Egerton, WS; Rivory, LP; Roberts, MS; Smithers, BM; Weiss, M; Wu, ZY, 1997
)
0.5
" The EMT-6/PRK5 beta 1E tumor was markedly resistant to a dosage range of cyclophosphamide, cisplatin, melphalan and thiotepa compared with the EMT-6/Parent tumor."( Transforming growth factor-beta 1 overexpression produces drug resistance in vivo: reversal by decorin.
Ara, G; Herbst, RS; Ikebe, M; Keyes, SR; Teicher, BA,
)
0.35
" The possibility of increasing ILI response rates by using other drugs and drug combinations and by multiple fractionated dosing is being investigated."( Isolated limb infusion with cytotoxic agents: a simple alternative to isolated limb perfusion.
Harman, CR; Kam, PC; Thompson, JF; Waugh, RC,
)
0.13
" Because alkylating agents generally have steep dose-response curves, mitomycin C (MMC) and melphalan (L-PAM) entered phase I/II studies on IHP."( Delivery of anticancer drugs via isolated hepatic perfusion: a promising strategy in the treatment of irresectable liver metastases?
Kuppen, PJ; Vahrmeijer, AL; Van De Velde, CJ; Van Der Eb, MM; Van Dierendonck, JH,
)
0.35
"The dose-response relationships for DNA fragmentation (assessed by pulsed-field gel electrophoresis, PFGE) and for viability (evaluated by measuring the reduction of MTT dye which can be accomplished by viable cells only) were investigated in order to discriminate between genotoxicity and cytotoxicity in the pathogenesis of DNA double-strand breaks (DSB)."( Discrimination between genotoxicity and cytotoxicity in the induction of DNA double-strand breaks in cells treated with etoposide, melphalan, cisplatin, potassium cyanide, Triton X-100, and gamma-irradiation.
Hoffmann, HD; Hormes, P; Lutz, WK; Vamvakas, S; Vock, EH, 1998
)
0.5
"Among the drugs used in conditioning regimens for stem cell transplantation, high-dose melphalan (HDM) plays an important role for both its strong myeloablative effect and for its favourable dose-response ratio."( Paroxysmal atrial fibrillation after high-dose melphalan in five patients autotransplanted with blood progenitor cells.
Brunori, M; Centanni, M; Corvatta, L; Ferretti, GF; Leoni, P; Montanari, M; Offidani, M; Olivieri, A, 1998
)
0.78
"High dosage melfalan chemotherapy with subsequent autologous blood stem cell transplantation in suitably selected patients with multiple myeloma greatly increases the probability that complete remission will be achieved and it prolongs the mean survival period as compared with classical chemotherapy."( [Autologous transplantation of peripheral hematopoietic cells in a patient with multiple myeloma and renal insufficiency].
Adam, Z; Doubek, M; Hájek, R; Krejcí, M; Mayer, J; Vorlícek, J, 1997
)
0.3
"In an attempt to improve tumor response and survival among patients with colorectal cancer metastases confined to the liver, we developed an experimental (rats and pigs) and clinical isolated hepatic perfusion (IHP) technique to exploit maximally the steep dose-response relation of may anticancer drugs."( Phase I/II studies of isolated hepatic perfusion with mitomycin C or melphalan in patients with colorectal cancer hepatic metastases.
Marinelli, A; Vahrmeijer, AL; van de Velde, CJ, 1998
)
0.53
" TNF dosage was 20 microgram for mice and 200 microgram for rats."( Synergism of tumor necrosis factor-alpha and melphalan in systemic and regional administration: animal study.
Gutman, M; Klausner, JM; Lev-Chelouche, D; Merimsky, O; Sofer, D, 1997
)
0.56
"Melphalan (MEL) is probably the most effective chemotherapeutic agent in multiple myeloma (MM) with a clear dose-response effect."( Safety of autotransplants with high-dose melphalan in renal failure: a pharmacokinetic and toxicity study.
Alberts, DS; Barlogie, B; Bracy, D; Dorr, RT; Jagannath, S; Johnson, C; Meyers, R; Roe, DJ; Tricot, G; Vesole, DH, 1996
)
2
" With all other chemicals, the dose-response curves for DNA fragmentation and viability were mirror images."( Investigation of the induction of DNA double-strand breaks by methylenediphenyl-4-4'-diisocyanate in cultured human lung epithelial cells.
Gahlmann, R; Lutz, WK; Vamvakas, S; Vock, EH, 1998
)
0.3
"The time-dependent dose-response relationships for the induction of DNA double-strand breaks (DSB) assessed by pulsed-field gel electrophoresis (PFGE) and for viability (evaluated by the MTT cytotoxicity test) were investigated in order to discriminate between genotoxic and cytotoxic mechanisms of DNA fragmentation."( Discrimination between genotoxicity and cytotoxicity for the induction of DNA double-strand breaks in cells treated with aldehydes and diepoxides.
Ilinskaya, O; Lutz, WK; Vamvakas, S; Vock, EH, 1999
)
0.3
" Epidoxorubicin (70 mg/m2) was added in patients who previously received a suboptimal dosage of antracycline."( Stop-flow in mediastinum and thorax for resistant lymphoma.
Abate, G; Aigner, KR; Amicucci, G; Benita, C; Capannolo, B; D'Alessandro, V; Filippo, R; Gianfranco, A; Giuseppe, A; Guadagni, S; Luca, M; Marsili, L; Pozone, T; Roland, AK; Russo, F; Stefano, G; Tullio, P; Valfredo, D,
)
0.13
" There are several strategies for increasing dose intensity, one being a protracted daily dosing strategy without major dose reduction for toxicity."( Cytotoxic chemotherapy regimens that increase dose per cycle (dose intensity) by extending daily dosing from 5 consecutive days to 28 consecutive days and beyond.
Albella, B; Bueren, JA; Keyes, KA; LoRusso, PM; Parchment, RE, 2000
)
0.31
"A 14C study of chemobiokinetics of sarcolysin and its peptides of glutaminic acid, dosage and routes of administration was conducted in intact rats and those bearing Walker's carcinoma."( [Chemobiokinetics of sarcolysin and its peptides with glutaminic acid].
Filov, VA; Kon'kov, SA; Korsakov, MV; Krasnov, VP; Mironiuk, TA; Reztsova, VV; Zhdanova, EA, 2000
)
0.31
" Melphalan was administered at a dosage of 150-220 mg/m(2) (median 180)."( High-dose melphalan with autologous hematopoietic stem cell transplantation for acute myeloid leukemia: results of a retrospective analysis of the Italian Pediatric Group for Bone Marrow Transplantation.
Bagnulo, S; Caniggia, M; Cesaro, S; Lanino, E; Locatelli, F; Meloni, G; Messina, C; Pession, A; Pillon, M; Proglia, A, 2001
)
1.62
"In order to control busulfan pharmacokinetic variability and toxicity, a specific monitoring protocol was instituted in our bone marrow transplant BMT paediatric patients including a test dose, daily Bayesian forecasting of busulfan plasma levels, and Bayesian individualization of busulfan dosage regimens."( Improved clinical outcome of paediatric bone marrow recipients using a test dose and Bayesian pharmacokinetic individualization of busulfan dosage regimens.
Aulagner, G; Bertrand, Y; Bleyzac, N; Dai, Q; Galambrun, C; Janoly, A; Jelliffe, RW; Magron, P; Maire, P; Martin, P; Souillet, G, 2001
)
0.31
" Despite prompt initiation of antimycotic therapy the outcome was fatal; dosage of conventional and liposomal amphotericin B was limited due to treatment-related toxicities."( A novel type of metastatically spreading subcutaneous aspergillosis without epidermal lesions following allogeneic stem cell transplantation.
Bethe, U; Cornely, OA; Pels, H; Ritzkowsky, A; Seibold, M; Soehngen, D; Toepelt, K, 2001
)
0.31
" Further detailed pharmacokinetic studies of irinotecan in patients receiving concomitant therapy with enzyme-inducing anticonvulsants are required so that rational dosing recommendations can be provided for this patient population."( Influence of phenytoin on the disposition of irinotecan: a case report.
Berg, S; Bernstein, M; Blaney, SM; Cherrick, I; Kuttesch, N; Murry, DJ; Salama, V, 2002
)
0.31
" We discuss such a risk-adapted approach to melphalan dosing in detail and conclude with a brief overview of current research using SCT to treat patients with AL."( Autologous stem cell transplantation for primary systemic amyloidosis.
Comenzo, RL; Gertz, MA, 2002
)
0.58
" A dosage schedule based on body surface area should be used especially in young children to reduce the age-dependent difference in kinetics."( A phase II trial of liposomal busulphan as an intravenous myeloablative agent prior to stem cell transplantation: 500 mg/m(2) as a optimal total dose for conditioning.
Aschan, J; Eber, S; Gungor, T; Hassan, M; Hassan, Z; Hentschke, P; Ljungman, P; Nilsson, C; Ringdén, O; Seger, R; Winiarski, J, 2002
)
0.31
" The future research in HILP with TNFalpha is directed in increasing tumor sensitivity for TNF with lowering the dosage without decreasing tumor response."( Hyperthermic isolated limb perfusion in the management of extremity sarcoma.
Hoekstra, HJ; van Ginkel, RJ, 2003
)
0.32
"Hyperthermic isolated limb perfusion (HILP) with melphalan as treatment for locally recurrent or in-transit malignant melanoma is frequently performed but the principle for calculating drug dosage remains poorly understood."( Marked variability of melphalan plasma drug levels during regional hyperthermic isolated limb perfusion.
Abdul-Wahab, O; Aloia, T; Cheng, TY; Colvin, M; Fedrau, R; Friedman, H; Grubbs, E; Hung, CF; Leu, SY; Petros, W; Pruitt, S; Tyler, D, 2003
)
0.89
"The population pharmacokinetic approach developed in this study should allow dosage to be individualized in order to decrease toxicity while maintaining good efficacy."( Population pharmacokinetics of melphalan, infused over a 24-hour period, in patients with advanced malignancies.
Astre, C; Bressolle, F; Culine, S; Fabbro, M; Mougenot, P; Pinguet, F; Poujol, S, 2004
)
0.61
"0% tended to have more proteinuria, more organs involved, lower serum albumin, more diuretic use, and dosage adjustment during mobilization."( Excessive fluid accumulation during stem cell mobilization: a novel prognostic factor of first-year survival after stem cell transplantation in AL amyloidosis patients.
Cha, SS; Dispenzieri, A; Gertz, MA; Lacy, MQ; Leung, N; Leung, TR, 2005
)
0.33
"The current study was performed to evaluate the efficacy and toxicity of a chemotherapy treatment using melphalan administered over a 24-h period with individual adapted dosing in advanced ovarian cancer."( Phase II study of melphalan as a single-agent infused over a 24-hour period with individual adapted dosing in patients with recurrent epithelial ovarian cancer.
Bressolle, F; Culine, S; Fabbro, M; Mougenot, P; Pinguet, F; Pouessel, D, 2006
)
0.88
" There was no dose-response relationship for doses 30 Gy or higher, even for larger tumors."( Prognostic factors in solitary plasmacytoma of the bone: a multicenter Rare Cancer Network study.
Belkacémi, Y; Bolla, M; Castelain, B; Knobel, D; Landmann, C; Oner, FD; Ozsahin, M; Poortmans, P; Tsang, RW; Zouhair, A, 2006
)
0.33
"03), with a positive trend also with regard to the dosage of the antiblastic drug employed (P<0."( Hyperthermic antiblastic perfusion in the treatment of locoregional spreading limb melanoma.
Anzà, M; Botti, C; Callopoli, A; Cavaliere, F; Di Filippo, F; Di Filippo, S; Frezza, F; Garinei, R; Giannarelli, D; Maialetti, R; Perri, P; Psaila, A; Sega, F; Viticci, C, 2003
)
0.32
"6 mg, we conclude that 1 mg is the best dosage to be applied during HAP."( Hyperthermic antiblastic perfusion with TNFalpha and melphalan in stage III limb melanoma patients: A phase I - II SITILO study.
Anzà, M; Botti, C; Cavaliere, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Garinei, R; Laurenzi, L; Perri, P; Principi, F; Rossi, CR, 2003
)
0.57
" However, lower TNF dosage produces a very strong effect that is to increase the drug penetration into the tumour, presumably by decreasing the intratumoural hypertension resulting in better tumour uptake."( Recombinant human tumor necrosis factor: an efficient agent for cancer treatment.
Lejeune, FJ; Rüegg, C, 2006
)
0.33
" Data about BNP dosage for cardiovascular monitoring of patients with ALA on renal replacement therapy are lacking."( Role of B-type natriuretic peptide in cardiovascular state monitoring in a hemodialysis patient with primary amyloidosis.
Cantelli, S; Catizone, L; Fabbian, F; Molino, C; Russo, G; Russo, M; Sartori, S; Stabellini, N, 2006
)
0.33
" The BSA dosing resulted in a wide range of melphalan doses given (2."( Oral mucositis in myeloma patients undergoing melphalan-based autologous stem cell transplantation: incidence, risk factors and a severity predictive model.
Anaissie, EJ; Barlogie, B; Dong, L; Fassas, A; Grazziutti, ML; Kiwan, E; Klaus, H; Krishna, SG; Miceli, MH; Syed, N; van Rhee, F, 2006
)
0.85
" Our findings suggest that drug dosage personalization based on the measurement of drug distribution volumes might minimize hepatic toxicity."( Correlation between melphalan pharmacokinetics and hepatic toxicity following hyperthermic isolated liver perfusion for unresectable metastatic disease.
Casara, D; Corazzina, S; Da Pian, P; Forlin, M; Innocente, F; Lise, M; Mocellin, S; Nitti, D; Ori, C; Pilati, P; Rossi, CR; Ujka, F, 2007
)
0.66
" The TNFalpha was empirically employed at a dosage of 3-4 mg, that is ten times the systemic maximum tolerated dose (MTD)."( Hyperthermic isolation limb perfusion with TNFalpha in the treatment of in-transit melanoma metastasis.
Anzà, M; Botti, C; Cavaliere, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Garinei, R; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M,
)
0.13
" Forty patients were treated with a TNFalpha dosage > 1 mg and 73 with 1 mg."( Hyperthermic isolation limb perfusion with TNFalpha in the treatment of in-transit melanoma metastasis.
Anzà, M; Botti, C; Cavaliere, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Garinei, R; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M,
)
0.13
" We tried to correlate the typed tumor response (CR or not CR) and the TNFalpha dosage < or = 1 mg or > 1 mg, but no statistically significant difference was found between the two groups."( Hyperthermic isolation limb perfusion with TNFalpha in the treatment of in-transit melanoma metastasis.
Anzà, M; Botti, C; Cavaliere, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Garinei, R; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M,
)
0.13
"A population pharmacokinetic model for melphalan has been developed and validated and may now be used in conjunction with pharmacodynamic data to develop safe and effective dosing guidelines in children with malignant diseases."( Population pharmacokinetics of melphalan in paediatric blood or marrow transplant recipients.
Earl, JW; Montgomery, K; Nath, CE; Shaw, PJ, 2007
)
0.89
" Cytotoxicity of deferoxamine for neuroblastoma cell lines measured by the DIMSCAN assay achieved dose-response curves similar to data obtained by manual trypan blue counts or colony formation in soft agar but with a wider dynamic range."( A fluorescence microplate cytotoxicity assay with a 4-log dynamic range that identifies synergistic drug combinations.
Frgala, T; Kalous, O; Proffitt, RT; Reynolds, CP, 2007
)
0.34
" BSA- or age-based dosing is used with therapeutic drug monitoring (TDM) to reduce this variability."( Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring.
Doz, F; Espérou, H; Galambrun, C; Gentet, JC; Mechinaud, F; Michel, G; Neven, B; Nguyen, L; Puozzo, C; Valteau-Couanet, D; Vassal, G; Zouabi, H, 2008
)
0.35
"A new intravenous (IV) dosing of busulfan (Bu) based on body weight, designed to improve AUC targeting without TDM and dose-adjustment, was prospectively evaluated."( Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring.
Doz, F; Espérou, H; Galambrun, C; Gentet, JC; Mechinaud, F; Michel, G; Neven, B; Nguyen, L; Puozzo, C; Valteau-Couanet, D; Vassal, G; Zouabi, H, 2008
)
0.35
" This new dosing enabled to achieve a mean exposure comparable to that in adults."( Prospective validation of a novel IV busulfan fixed dosing for paediatric patients to improve therapeutic AUC targeting without drug monitoring.
Doz, F; Espérou, H; Galambrun, C; Gentet, JC; Mechinaud, F; Michel, G; Neven, B; Nguyen, L; Puozzo, C; Valteau-Couanet, D; Vassal, G; Zouabi, H, 2008
)
0.35
" The dose-response relationships for melphalan and irinotecan in individual samples showed great variability."( Heterogeneous activity of cytotoxic drugs in patient samples of peritoneal carcinomatosis.
Glimelius, B; Graf, W; Grundmark, B; Larsson, R; Mahteme, H; Nygren, P; Påhlman, L; Tholander, B; von Heideman, A, 2008
)
0.62
" These data may aid the design of studies to clarify optimal dosing and leukapheresis with pegfilgrastim."( A phase 2 pilot study of pegfilgrastim and filgrastim for mobilizing peripheral blood progenitor cells in patients with non-Hodgkin's lymphoma receiving chemotherapy.
Baker, N; Barker, P; Boogaerts, M; Canizo, CD; Johnsen, HE; Mesters, R; Russell, N; Schmitz, N; Schubert, J; Skacel, T, 2008
)
0.35
" Pharmacokinetic assays of mycophenolic acid (MPA) showed a therapeutic area under the curve (AUC) at the dosage of 3 g daily, although a large inter- and intraindividual variations of MPA plasma levels were found."( Reduced-intensity conditioning allogeneic transplantation from unrelated donors: evaluation of mycophenolate mofetil plus cyclosporin A as graft-versus-host disease prophylaxis.
Caballero, D; Calvo, MV; de la Cámara, R; de Oteiza, JP; Heras, I; Martino, R; Pérez-Simón, JA; Rebollo, N; San Miguel, JF; Sierra, J; Valcarcel, D, 2008
)
0.35
" The goal of dosing 200 mg per day was achieved in just 17 of 31 patients, and the median tolerated thalidomide dose was 100 mg per day."( Thalidomide maintenance following high-dose melphalan with autologous stem cell support in myeloma.
Callander, NS; Chang, JE; Gangnon, RE; Juckett, MB; Kahl, BS; Longo, WL; Mitchell, TL, 2008
)
0.61
" Weight-based dosing has been suggested to better predict toxicity of the conditioning regimen."( Effect of the dose per body weight of conditioning chemotherapy on severity of mucositis and risk of relapse after autologous haematopoietic stem cell transplantation in relapsed diffuse large B cell lymphoma.
Ansell, SM; Costa, LJ; Inwards, DJ; Johnston, PB; Litzow, MR; Micallef, IN; Porrata, LF, 2008
)
0.35
" The optimal target area under the curve (AUC) and dosing schedule of intravenous busulfan in children undergoing hematopoietic stem cell transplantation (HSCT) remain unclear, however."( Association between busulfan exposure and outcome in children receiving intravenous busulfan before hematologic stem cell transplantation.
Bartelink, IH; Belitser, SV; Bierings, M; Boelens, JJ; Bredius, RG; Egberts, AC; Egeler, M; Knibbe, CA; Lankester, AC; Suttorp, MM; Zwaveling, J, 2009
)
0.35
" Further work is indicated to determine optimal dosing regimens, maximal tolerated dosage, and subsequent visual function in these patients."( Persistence of retinal function after selective ophthalmic artery chemotherapy infusion for retinoblastoma.
Abramson, DH; Brodie, SE; Dunkel, IJ; Kim, JW; Pierre Gobin, Y, 2009
)
0.35
"This study aims to determine what effect correcting melphalan dosing for ideal body weight (IBW) has on toxicity and response in isolated limb infusion (ILI) in patients with advanced extremity melanoma."( Optimizing melphalan pharmacokinetics in regional melanoma therapy: does correcting for ideal body weight alter regional response or toxicity?
Augustine, CK; Beasley, GM; Cheng, TY; McMahon, N; Padussis, JC; Petros, W; Sanders, G; Spasojevic, I; Tyler, DS; Zipfel, P, 2009
)
0.99
" Body surface area-based dosing in the FLU/MEL regimen led to a wide range of MEL doses administered per kilogram body weight (2."( Characteristics and risk factors of oral mucositis after allogeneic stem cell transplantation with FLU/MEL conditioning regimen in context with BU/CY2.
Karas, M; Koza, V; Steinerova, K; Vokurka, S, 2009
)
0.35
"In isolated limb perfusion (ILP) with tumor necrosis factor-alpha (TNFalpha) and interferon (IFN)-gamma, pioneered by Lienard and Lejenne in 1988, TNFalpha was empirically employed at a dosage (3-4 mg) ten times higher than the systemic maximum tolerable dose (MTD)."( Prognostic factors influencing tumor response, locoregional control and survival, in melanoma patients with multiple limb in-transit metastases treated with TNFalpha-based isolated limb perfusion.
Anzà, M; Armenti, A; Botti, C; Cavaliere, F; Corrias, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Ferraresi, V; Garinei, R; Giacomini, P; Ginebri, A; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M; Sofra, C; Sperduti, I,
)
0.13
" Forty patients were treated with a TNFalpha dosage of >1 mg and 73 with 1 mg."( Prognostic factors influencing tumor response, locoregional control and survival, in melanoma patients with multiple limb in-transit metastases treated with TNFalpha-based isolated limb perfusion.
Anzà, M; Armenti, A; Botti, C; Cavaliere, F; Corrias, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Ferraresi, V; Garinei, R; Giacomini, P; Ginebri, A; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M; Sofra, C; Sperduti, I,
)
0.13
" The TNFalpha dosage of 1 mg was as effective as 3-4 mg, with lower toxicity and cost."( Prognostic factors influencing tumor response, locoregional control and survival, in melanoma patients with multiple limb in-transit metastases treated with TNFalpha-based isolated limb perfusion.
Anzà, M; Armenti, A; Botti, C; Cavaliere, F; Corrias, F; Di Angelo, P; Di Filippo, F; Di Filippo, S; Ferraresi, V; Garinei, R; Giacomini, P; Ginebri, A; Pasqualoni, R; Perri, P; Rossi, CR; Santinami, M; Sofra, C; Sperduti, I,
)
0.13
" We administered estradiol, in several dosing regimens, to male, female and ovariectomized nude rats in a survival study."( Estrogen increases survival in an orthotopic model of glioblastoma.
Barone, TA; Gorski, JW; Greenberg, SJ; Plunkett, RJ, 2009
)
0.35
"Even after complete surgical resection of pulmonary metastases, many patients develop recurrent disease in the thorax despite the use of systemic chemotherapy, dosage of which is limited because of systemic toxicity."( Locoregional therapy.
Friedel, G; Furrer, M; Van Schil, PE, 2010
)
0.36
"Because of the less graft-facilitating effect by bone marrow (BM), we need to assess a dosage of conditioning more accurately particularly in combination with reduced-intensity conditioning."( A prospective dose-finding trial using a modified continual reassessment method for optimization of fludarabine plus melphalan conditioning for marrow transplantation from unrelated donors in patients with hematopoietic malignancies.
Atsuta, Y; Imahashi, N; Ito, T; Kato, T; Miyamura, K; Morishita, Y; Murata, M; Naoe, T; Nishiwaki, S; Ohashi, H; Sawa, M; Suzuki, R; Terakura, S; Yasuda, T, 2011
)
0.58
"Isolated limb infusion (ILI) with melphalan (M-ILI) dosing corrected for ideal body weight (IBW) is a well-tolerated treatment for patients with in-transit melanoma with a 29% complete response rate."( Prospective multicenter phase II trial of systemic ADH-1 in combination with melphalan via isolated limb infusion in patients with advanced extremity melanoma.
Augustine, CK; Beasley, GM; Grobmyer, SR; Hochwald, SN; Peterson, B; Riboh, JC; Ross, MI; Royal, R; Tyler, DS; Zager, JS, 2011
)
0.88
" The empirical practice of body surface area-based (BSA) dosing (mg/m2) of melphalan has been critically analyzed in several observations."( [The variance of melphalan doses related to kilogram of body weight and the consequences].
Vokurka, S, 2010
)
0.93
"Obesity has implications for chemotherapy dosing and selection of patients for therapy."( Effect of obesity on outcomes after autologous hematopoietic stem cell transplantation for multiple myeloma.
Ballen, K; Bashey, A; Freytes, C; Gibson, J; Hari, P; Heitjan, DF; Holmberg, L; Kamble, R; Kyle, RA; Lazarus, HM; Maharaj, D; Maiolino, A; McCarthy, PL; Pérez, WS; Roy, V; Stadtmauer, EA; Vesole, D; Vogl, DT; Wang, T; Weisdorf, D, 2011
)
0.37
" Lenalidomide was dosed 10 mg on days 1 to 21 of a 28-day schedule for a total of 24 cycles."( Lenalidomide maintenance after nonmyeloablative allogeneic stem cell transplantation in multiple myeloma is not feasible: results of the HOVON 76 Trial.
Bruijnen, CP; Cornelisse, PB; Cornelissen, JJ; Emmelot, M; Huisman, C; Huls, G; Janssen, JJ; Kersten, MJ; Kneppers, E; Lokhorst, HM; Meijer, E; Minnema, MC; Mutis, T; Sonneveld, P; van der Holt, B; Zweegman, S, 2011
)
0.37
"The PBPK model matched the pharmacokinetics in different dosing regimens in adults."( Physiologically based pharmacokinetic modelling of high- and low-dose etoposide: from adults to children.
Boos, J; Hempel, G; Kersting, G; Lippert, J; Willmann, S; Würthwein, G, 2012
)
0.38
" A risk-adapted approach to melphalan dosing with PBSCT is an effective treatment in patients with primary AL amyloidosis."( [Three patients with primary AL amyloidosis treated by high-dose melphalan with autologous peripheral blood stem cell transplantation].
Kanashima, H; Mukai, S; Nakao, T; Ogawa, Y; Teshima, H; Yamane, T, 2012
)
0.91
" The UE appears relatively resistant to toxic effects of melphalan-based ILI as currently performed, which suggests a potential for further optimization of drug dosing for UE ILI."( A multi-institution experience comparing the clinical and physiologic differences between upper extremity and lower extremity melphalan-based isolated limb infusion.
Beasley, GM; Dewhirst, MW; Lidsky, M; Masoud, M; Miller, M; Mosca, PJ; Sharma, K; Turley, RS; Tyler, DS; Wong, J; Zager, JS, 2012
)
0.83
" The primary objective was to determine the safest and best tolerated maintenance dosing (MD) of bortezomib (B)."( Phase I trial of bortezomib during maintenance phase after high dose melphalan and autologous stem cell transplantation in patients with multiple myeloma.
Abidi, MH; Abrams, J; Al-Kadhimi, Z; Ayash, L; Deol, A; Gul, Z; Lum, L; Mellon-Reppen, S; Ratanatharathorn, V; Uberti, J; Ventimiglia, M; Zonder, J, 2012
)
0.61
" By the late 1960s, extended dosing with melphalan and prednisone tripled survival from diagnosis and became the standard of care for newly diagnosed MM."( Evolving therapeutic paradigms for multiple myeloma: back to the future.
Cherry, BM; Korde, N; Kwok, M; Landgren, O; Roschewski, M, 2013
)
0.66
" Palifermin has permitted safe dose escalation of melphalan up to 280 mg/m(2), thus reaching the cumulative dosage of melphalan administered in tandem ASCT."( A phase I dose-escalation trial of high-dose melphalan with palifermin for cytoprotection followed by autologous stem cell transplantation for patients with multiple myeloma with normal renal function.
Abidi, MH; Abrams, J; Agarwal, R; Al-Kadhimi, Z; Ayash, L; Cronin, S; Deol, A; Lum, L; Ratanatharathorn, V; Tageja, N; Uberti, J; Ventimiglia, M; Zonder, J, 2013
)
0.9
" Meanwhile, the median dosage of MEL in our procedure was 103 mg/m(2) (range 68∼180), less than the recommended dose, and patients were maintained on miscellaneous therapies."( [Clinical analysis of autologous stem cell transplantation for nine cases of cardiac amyloidosis].
Abe, Y; Iizuka, H; Kusaka, S; Nakagawa, Y; Nishiyama, S; Ogura, M; Sekine, R; Suzuki, K, 2012
)
0.38
"The average concentration of melphalan required for inducing significant apoptosis was 61 μM, or about 3-fold less than the theoretical concentration of 192 μM, achieved in the hepatic artery during CS-PHP dosing with melphalan."( Evaluation of melphalan, oxaliplatin, and paclitaxel in colon, liver, and gastric cancer cell lines in a short-term exposure model of chemosaturation therapy by percutaneous hepatic perfusion.
Johnston, DS; Sheets, TP; Uzgare, RP, 2013
)
1.04
" As a RIC regimen for CBT, 140 mg/m(2) melphalan with fludarabine and anti-lymphocyte globulin or anti-thymocyte globulin may be feasible, but further dosage optimization should be performed in controlled clinical trials."( Feasibility of reduced-intensity conditioning followed by unrelated cord blood transplantation for primary hemophagocytic lymphohistiocytosis: a nationwide retrospective analysis in Japan.
Atsuta, Y; Goto, H; Inagaki, J; Inoue, M; Ishii, E; Kato, K; Kawa, K; Koike, K; Ohga, S; Okada, K; Sawada, A; Suzuki, N; Suzuki, R; Yabe, H; Yasutomo, K, 2013
)
0.66
" Further studies to examine intra-arterial chemotherapy's pharmacokinetics and dose-response relations are warranted in order to minimize the necessary exposure to chemotherapy."( Intra-arterial chemotherapy for group C retinoblastoma with adjacent high-flow infantile hemangioma.
Orbach, DB; Shah, AS; Trief, D; Vanderveen, DK; Yonekawa, Y,
)
0.13
" Panobinostat + melphalan appears to have tolerability issues in a dosing regimen capable of producing a response."( A phase 1/2 study of oral panobinostat combined with melphalan for patients with relapsed or refractory multiple myeloma.
Berenson, JR; Boccia, RV; Dressler, K; Ghazal, HH; Harb, WA; Hilger, JD; Jamshed, S; Kingsley, EC; Matous, J; Nassir, Y; Noga, SJ; Swift, RA; Vescio, R; Yellin, O, 2014
)
1
"Significant ocular complications following IViT for retinoblastoma are uncommon, and this risk may be reduced further by the use of careful injection technique and standard dosing regimens."( Ocular side effects following intravitreal injection therapy for retinoblastoma: a systematic review.
Mohney, BG; Smith, BD; Smith, SJ, 2014
)
0.4
"To evaluate whether blood-based genotoxicity endpoints can provide temporal and dose-response data within the low-dose carcinogenic range that could contribute to carcinogenic mode of action (MoA) assessments, we evaluated the sensitivity of flow cytometry-based micronucleus and Pig-a gene mutation assays at and below tumorigenic dose rate 50 (TD50) levels."( Pig-a gene mutation and micronucleated reticulocyte induction in rats exposed to tumorigenic doses of the leukemogenic agents chlorambucil, thiotepa, melphalan, and 1,3-propane sultone.
Avlasevich, SL; Bemis, JC; Cottom, J; Dertinger, SD; Macgregor, JT; Mereness, J; Phonethepswath, S; Torous, DK, 2014
)
0.6
" This article reviews the current published literature on the dosing of pharmacologic agents used for HCT preparative regimens with specific focus on the obese patient population."( Conditioning chemotherapy dose adjustment in obese patients: a review and position statement by the American Society for Blood and Marrow Transplantation practice guideline committee.
Bubalo, J; Carpenter, PA; Leather, HL; Majhail, N; Marks, DI; Perales, MA; Pidala, J; Savani, BN; Shaughnessy, P; Wingard, J, 2014
)
0.4
" Limited data exist on the comparison of the two dosing schedules."( Comparison of 1-day vs 2-day dosing of high-dose melphalan followed by autologous hematopoietic cell transplantation in patients with multiple myeloma.
Alsina, M; Bookout, R; Kim, J; Nishihori, T; Parmar, SR; Perkins, J; Shapiro, JF; Tombleson, R; Tomblyn, M; Yue, B, 2014
)
0.66
" The first step of the study was to assess the dose-response of Melphalan 21 days after engraftment."( Comparative analysis of multiple myeloma treatment by CD138 antigen targeting with bismuth-213 and Melphalan chemotherapy.
Bruchertseifer, F; Chérel, M; Davodeau, F; Faivre-Chauvet, A; Gaschet, J; Gouard, S; Kraeber-Bodéré, F; Matous, E; Maurel, C; Morgenstern, A; Pallardy, A, 2014
)
0.86
"Substantial efficacy has been demonstrated with bortezomib-melphalan-prednisone in phase III studies in transplant-ineligible myeloma patients using various twice-weekly and once-weekly bortezomib dosing schedules."( Bortezomib cumulative dose, efficacy, and tolerability with three different bortezomib-melphalan-prednisone regimens in previously untreated myeloma patients ineligible for high-dose therapy.
Boccadoro, M; Bringhen, S; Desai, A; Di Raimondo, F; Esseltine, DL; García-Sanz, R; Lahuerta, JJ; Larocca, A; Londhe, A; Mateos, MV; Oriol, A; Palumbo, A; Richardson, PG; San Miguel, JF; van de Velde, H, 2014
)
0.87
" We present a retrospective analysis of 73 consecutive patients aged over 65 years treated for aggressive or relapsed lymphoma by HDT with carmustine, etoposide, cytarabine and melphalan (BEAM) at full dosage followed by ASCT."( High-dose chemotherapy with carmustine, etoposide, cytarabine and melphalan followed by autologous stem cell transplant is an effective treatment for elderly patients with poor-prognosis lymphoma.
Barriere, J; Borchiellini, D; Boscagli, A; Coso, D; Garnier, G; Gastaud, L; Gutnecht, J; Martin, N; Naman, H; Petit, E; Peyrade, F; Re, D; Rossignol, B; Saudes, L; Thyss, A, 2015
)
0.85
" In the phase 1 portion of the study, 24 patients received CMP at carfilzomib dosing levels of 20 mg/m(2), 27 mg/m(2), 36 mg/m(2), and 45 mg/m(2)."( Phase 1/2 study of carfilzomib plus melphalan and prednisone in patients aged over 65 years with newly diagnosed multiple myeloma.
Attal, M; Avet-Loiseau, H; Benboubker, L; Caillot, D; Chaleteix, C; Chiffoleau, A; Facon, T; Fortin, J; Hulin, C; Kolb, B; Leleu, X; Mary, JY; Moreau, P; Planche, L; Roussel, M; Tiab, M; Touzeau, C, 2015
)
0.69
" Busulfan dosing targeted 4000 μM-minute/day (days -8 to -5)."( Vorinostat Combined with High-Dose Gemcitabine, Busulfan, and Melphalan with Autologous Stem Cell Transplantation in Patients with Refractory Lymphomas.
Ahmed, S; Alousi, A; Anderlini, P; Andersson, BS; Bashir, Q; Champlin, R; Dabaja, B; Fanale, M; Gulbis, A; Hagemeister, F; Hosing, C; Jones, RB; Liu, Y; Nieto, Y; Oki, Y; Pinnix, C; Popat, U; Qazilbash, M; Shah, N; Shpall, EJ; Thall, PF; Valdez, BC, 2015
)
0.66
"Despite advances in cross-sectional imaging, chemotherapeutic dosing for isolated limb infusion (ILI) in melanoma is currently calculated through cumbersome and potentially imprecise manual measurements."( Computed Tomography-Based Limb Volume Measurements for Isolated Limb Infusion in Melanoma.
Bashir, MR; Beasley, GM; Bhatti, L; Brys, AK; Jaffe, TA; Mosca, PJ; Nath, NS; Salama, AK; Tyler, DS, 2016
)
0.43
"CT-based limb volume measurement is feasible for chemotherapy dosing in patients undergoing ILI for melanoma and has predictive value with respect to clinical response and toxicity."( Computed Tomography-Based Limb Volume Measurements for Isolated Limb Infusion in Melanoma.
Bashir, MR; Beasley, GM; Bhatti, L; Brys, AK; Jaffe, TA; Mosca, PJ; Nath, NS; Salama, AK; Tyler, DS, 2016
)
0.43
" Precision dosing trials are warranted."( Alemtuzumab levels impact acute GVHD, mixed chimerism, and lymphocyte recovery following alemtuzumab, fludarabine, and melphalan RIC HCT.
Davies, SM; Filipovich, AH; Jodele, S; Lane, A; Marsh, RA; Mehta, PA; Neumeier, L, 2016
)
0.64
" This analysis of the pivotal phase 3 FIRST trial examined the impact of renally adapted dosing of lenalidomide and dexamethasone on outcomes of patients with different degrees of renal impairment."( Impact of renal impairment on outcomes with lenalidomide and dexamethasone treatment in the FIRST trial, a randomized, open-label phase 3 trial in transplant-ineligible patients with multiple myeloma.
Belhadj, K; Bensinger, W; Chen, G; Cheung, MC; Derigs, HG; Dib, M; Dimopoulos, MA; Eom, H; Ervin-Haynes, A; Facon, T; Gamberi, B; Hall, R; Jaccard, A; Jardel, H; Karlin, L; Kolb, B; Lenain, P; Leupin, N; Liu, T; Marek, J; Rigaudeau, S; Roussel, M; Schots, R; Tosikyan, A; Van der Jagt, R, 2016
)
0.43
" Melphalan and tumor necrosis factor-alpha are used at a dosage that depends on the volume of the limb."( Calculating regional tissue volume for hyperthermic isolated limb perfusion: Four methods compared.
Bodanza, V; Bui, F; Campana, LG; Cecchin, D; Frigo, AC; Gregianin, M; Negri, A; Rastrelli, M; Rossi, CR; Zucchetta, P, 2016
)
1.34
" Pharmacokinetic dosing of busulfan (Bu) is frequently done for myeloablative conditioning, but evidence for its use is limited in RIC transplants."( Fludarabine-Busulfan Reduced-Intensity Conditioning in Comparison with Fludarabine-Melphalan Is Associated with Increased Relapse Risk In Spite of Pharmacokinetic Dosing.
Al-Kali, A; Alkhateeb, HB; Damlaj, M; Gangat, N; Gastineau, DA; Hashmi, S; Hefazi, M; Hogan, WJ; Litzow, MR; Partain, DK; Patnaik, MM; Wolf, RC, 2016
)
0.66
" We retrospectively analyzed the outcome of 79 adult patients who underwent auto-HSCT for lymphoma using this regimen in two centers, with 1- and 2-day dosing of MEL, respectively."( Retrospective evaluation of the MEAM regimen as a conditioning regimen before autologous peripheral blood stem cell transplantation for lymphoma in two centers with different dosing schedules of melphalan.
Ashizawa, M; Fujiwara, S; Hatano, K; Ito, S; Kako, S; Kanda, Y; Kawasaki, Y; Mashima, K; Minakata, D; Muroi, K; Nakano, H; Oh, I; Ohmine, K; Okazuka, K; Sato, K; Sugimoto, M; Suzuki, T; Umino, K; Yamamoto, C; Yamasaki, R, 2016
)
0.62
" This prompted us to explore the concept of less intense drug dosing with shorter intervals between courses with the aim of preventing inter-course relapse."( Dose-dense and less dose-intense Total Therapy 5 for gene expression profiling-defined high-risk multiple myeloma.
Alapat, D; Avery, D; Bailey, C; Barlogie, B; Crowley, J; Epstein, J; Heuck, CJ; Hoering, A; Jethava, Y; Khan, R; Mitchell, A; Morgan, G; Petty, N; Sawyer, J; Schinke, C; Smith, R; Stein, C; Steward, D; Thanendrarajan, S; Tian, E; van Rhee, F; Waheed, S; Yaccoby, S; Zangari, M, 2016
)
0.43
"Stem cell transplantation (SCT), an effective therapy for amyloid light chain (AL) amyloidosis patients, is associated with low treatment-related mortality (TRM) with appropriate patient selection and risk-adapted dosing of melphalan (RA-SCT)."( Long-term event-free and overall survival after risk-adapted melphalan and SCT for systemic light chain amyloidosis.
Bello, C; Chou, JF; Comenzo, RL; Devlin, SM; Giralt, S; Hassoun, H; Landau, H; Landry, C; Smith, M, 2017
)
0.88
" We previously suggested an optimal day 0 targeted range of alemtuzumab, but there are no pediatric data regarding the pharmacokinetics (PK) of subcutaneous alemtuzumab to guide precision dosing trials."( Pretransplant Absolute Lymphocyte Counts Impact the Pharmacokinetics of Alemtuzumab.
Chandra, S; Emoto, C; Fukuda, T; Khandelwal, P; Marsh, RA; Mehta, PA; Neumeier, L; Teusink-Cross, A; Vinks, AA, 2017
)
0.46
" These findings contribute to ongoing efforts to personalize melphalan dosing in transplant patients."( Associations of High-Dose Melphalan Pharmacokinetics and Outcomes in the Setting of a Randomized Cryotherapy Trial.
Benson, DM; Cho, YK; Devine, SM; Efebera, YA; Gao, Y; Hade, EM; Hofmeister, CC; Lamprecht, M; Li, J; Phelps, MA; Poi, M; Rosko, AE; Sborov, DW; Tackett, K; Wang, J; Williams, N, 2017
)
1
"High-dose chemotherapies to treat multiple myeloma (MM) can be life-threatening due to toxicities to normal cells and there is a need to target only tumor cells and/or lower standard drug dosage without losing efficacy."( Multiple Myeloma Tumor Cells are Selectively Killed by Pharmacologically-dosed Ascorbic Acid.
Allamargot, C; Coleman, KL; Frech, I; Nessler, R; Tricot, G; Xia, J; Xu, H; Zhan, F; Zhang, X, 2017
)
0.46
" On day 1 of cycle 1, seven whole-blood samples were collected for 3 h after dosing completion to obtain the maximum plasma concentration and area under the time-concentration curve during 3 h postdose (AUC0-3) in each patient."( Bortezomib pharmacokinetics in tumor response and peripheral neuropathy in multiple myeloma patients receiving bortezomib-containing therapy.
Choi, K; Han, S; Hong, T; Lee, J; Lee, SE; Min, CK; Park, GJ; Yim, DS, 2017
)
0.46
" This study demonstrated that a fast analysis for the purpose of quantifying a chemically unstable drug, such as melphalan, is feasible and important for the development of commercial dosage forms."( Rapid and simple flow injection analysis tandem mass spectrometric method for the quantification of melphalan in a lipid-based drug delivery system.
Badea, I; El-Aneed, A; Michel, D; Mohammed-Saeid, W, 2017
)
0.88
" The current dosing strategy based on body surface area results in a high incidence of oral mucositis and gastrointestinal and liver toxicity."( Population Pharmacokinetics and Optimal Sampling Strategy for Model-Based Precision Dosing of Melphalan in Patients Undergoing Hematopoietic Stem Cell Transplantation.
Anaissie, EJ; Chandra, S; Dong, M; Fukuda, T; McConnell, S; Mehta, PA; Mizuno, K; Vinks, AA, 2018
)
0.7
"Limited guidance exists for dosing melphalan for autologous stem cell transplantation (ASCT) in the obese patient population, because the current literature reports conflicting clinical outcomes between obese and nonobese patients."( Impact of Dose-Adjusted Melphalan in Obese Patients Undergoing Autologous Stem Cell Transplantation.
Arp, C; DeFrates, S; Shultes, KC; Stockerl-Goldstein, K; Trinkaus, K, 2018
)
1.06
" This information can inform delivery location and dosing strategies on a patient-specific basis."( Estimation of intra-arterial chemotherapy distribution to the retina in pediatric retinoblastoma patients using quantitative digital subtraction angiography.
Alexander, M; Amans, MR; Cooke, DL; Damato, B; Darflinger, R; Dowd, CF; Halbach, VV; Hetts, SW; Higashida, RT; Kao, A; Kondapavulur, S; Matthay, KK, 2018
)
0.48
" A daily melphalan dosing schedule reportedly is well tolerated and associated with favorable outcome."( Comparison of two melphalan protocols and evaluation of outcome and prognostic factors in multiple myeloma in dogs.
Chon, E; Fernández, R, 2018
)
1.23
"Positive results support the use of either dosing regimen for the treatment of dogs with MM, and renal disease and NLR were negative prognostic factors."( Comparison of two melphalan protocols and evaluation of outcome and prognostic factors in multiple myeloma in dogs.
Chon, E; Fernández, R, 2018
)
0.81
" We note that induction therapy may deprive some patients of the opportunity to proceed to SCT but is likely needed if the marrow plasmacytosis is > 10%, that risk-adapted melphalan dosing continues to be relevant, and that post-SCT consolidation improves the complete response rate as well as long-term overall survival."( High-dose melphalan and stem cell transplantation in systemic AL amyloidosis in the era of novel anti-plasma cell therapy: a comprehensive review.
Comenzo, RL; Varga, C, 2019
)
1.11
"Carboplatin dosage is calculated by using the estimated glomerular filtration rate (GFR) to achieve a target plasma area under the plasma concentration-time curve (AUC)."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.76
" A population pharmacokinetic model was built for carboplatin to evaluate various dosing formulas."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.76
" This model-based approach validates the use of weight-based dosing as an appropriate alternative for carboplatin in children with either mild renal impairment or normal renal function."( Population pharmacokinetics of carboplatin, etoposide and melphalan in children: a re-evaluation of paediatric dosing formulas for carboplatin in patients with normal or mild impairment of renal function.
Boddy, AV; Duong, JK; Errington, J; Ladenstein, R; Nath, CE; Shaw, PJ; Veal, GJ, 2019
)
0.76
" Using various dosing schedules, the majority of patients (684/694) received daratumumab at a dose of 16 mg/kg."( Pharmacokinetics and Exposure-Response Analyses of Daratumumab in Combination Therapy Regimens for Patients with Multiple Myeloma.
Belch, A; Capra, M; Clemens, PL; Dimopoulos, MA; Gomez, D; Ho, PJ; Iida, S; Jansson, R; Leiba, M; Medvedova, E; Min, CK; Schecter, J; Sonneveld, P; Sun, YN; Xu, XS; Zhang, L, 2018
)
0.48
"These data support the recommended 16 mg/kg dose of daratumumab and the respective dosing schedules in the POLLUX and CASTOR pivotal studies."( Pharmacokinetics and Exposure-Response Analyses of Daratumumab in Combination Therapy Regimens for Patients with Multiple Myeloma.
Belch, A; Capra, M; Clemens, PL; Dimopoulos, MA; Gomez, D; Ho, PJ; Iida, S; Jansson, R; Leiba, M; Medvedova, E; Min, CK; Schecter, J; Sonneveld, P; Sun, YN; Xu, XS; Zhang, L, 2018
)
0.48
" Mean dosage given showed significant difference between the groups."( Ocular toxicity of intravitreal melphalan for retinoblastoma in Chinese patients.
Meng, F; Qian, J; Ren, H; Xue, K; Zhang, R, 2019
)
0.8
"5 mg, whereby a strict protocol with age-appropriate dosage was not used until 2015."( Histopathology of retinoblastoma eyes enucleated after intra-arterial chemotherapy.
Bechrakis, NE; Biewald, EM; Bornfeld, N; Göricke, S; Ketteler, P; Kiefer, T; Metz, KA; Radbruch, A; Schlüter, S; Sirin, S, 2020
)
0.56
" The phase III VISTA trial established the bortezomib dosing schedule for VMP."( Comparative Efficacy of Bortezomib, Melphalan, and Prednisone (VMP) With or Without Daratumumab Versus VMP Alone in the Treatment of Newly Diagnosed Multiple Myeloma: Propensity Score Matching of ALCYONE and VISTA Phase III Studies.
Cavo, M; Deraedt, W; Dimopoulos, MA; He, J; Jakubowiak, A; Kampfenkel, T; Lam, A; Mateos, MV; Qi, M; San-Miguel, J, 2020
)
0.83
"Here we outline the methods to establish novel melphalan dosing using PK testing in MM patients undergoing ASCT to target a desired melphalan AUC."( Development of a method for clinical pharmacokinetic testing to allow for targeted Melphalan dosing in multiple myeloma patients undergoing autologous transplant.
Calip, GS; Galvin, JP; Hofmeister, CC; Johnson, JJ; Mahmud, N; Patel, P; Rondelli, D; Sweiss, K; Vemu, B; Wenzler, E, 2020
)
1.04
" One patient experienced renal dysfunction during the first HDCT, which was alleviated by sufficient hydration and diuretics and resulted in the reduction of melphalan dosage for the second HDCT."( Tandem high-dose chemotherapy with autologous stem cell rescue for stage M high-risk neuroblastoma: Experience using melphalan/etoposide/carboplatin and busulfan/melphalan regimens.
Arakawa, Y; Hiwatari, M; Hogetsu, K; Kato, S; Koh, K; Kubota, Y; Takita, J; Watanabe, K, 2020
)
0.96
" To more thoroughly investigate the dose-response relationships, benchmark dose (BMD) analyses were performed with PROAST software."( Pig-a gene mutation assay study design: critical assessment of 3- versus 28-day repeat-dose treatment schedules.
Dertinger, SD; Elhajouji, A; Hove, TT; Martus, H; O'Connell, O, 2020
)
0.56
"A validated PS-MS/MS assay for melphalan in patients undergoing HSCT is described that facilitates pharmacokinetic-guided individualized precision dosing with immediate bedside dose adjustments to improve outcomes by balancing toxicity and efficacy of melphalan."( Paperspray Ionization Mass Spectrometry as a Tool for Predicting Real-Time Optimized Dosing of the Chemotherapeutic Drug Melphalan.
Mehta, PA; Mizuno, K; Setchell, KDR; Sharat, C; Vinks, AA; Zhao, J, 2021
)
1.12
" This model can be used to define the therapeutic window of melphalan, and subsequently to develop individualized dosing regimens aiming for that therapeutic window in all patients."( Population Pharmacokinetics of Melphalan in a Large Cohort of Autologous and Allogeneic Hematopoietic Cell Transplantation Recipients: Towards Individualized Dosing Regimens.
Admiraal, R; Alperovich, A; Boelens, JJ; Carlow, D; Cruz Sitner, N; Dahi, P; Giralt, SA; Lin, A; Proli, A; Ruiz, J; Schofield, R; Scordo, M; Shah, GL; Tamari, R, 2022
)
1.25
"To improve the therapeutic properties of the antitumor agent Sarcolysin, we have previously developed and characterized a dosage form representing its ester conjugate with decanol embedded in ultra-small phospholipid nanoparticles less than 30 nm in size ("Sarcolysin-NP")."( [The effect of lipid derivative of anti-tumor drug sarcolysin embedded in phospholipid nanoparticles in the experiments in vivo].
Khudoklinova, YY; Kostryukova, LV; Sanzhakov, MA; Tereshkina, YA; Tikhonova, EG; Torkhovskaya, TI, 2021
)
0.62
" Results suggest the empiric weight-based dosing (mg/kg) used in children <12 kg or 2 years of age may result in subtherapeutic exposure."( Population Pharmacokinetics of Melphalan for Pediatric Patients Undergoing Hematopoietic Cell Transplantation.
Apsel Winger, B; Chan, D; Dvorak, CC; Gobburu, JVS; Li, S; Long-Boyle, J; Lu, Y, 2022
)
1.01
" Measured GFR (mGFR) may better predict drug dosing to mitigate toxicity and increase the chances of successful engraftment."( Relationship of iothalamate clearance and NRM in patients receiving fludarabine and melphalan reduced-intensity conditioning.
Alkhateeb, HB; Barreto, EF; Bartoo, GT; Hogan, WJ; Kutzke, JL; Leung, N; Litzow, MR; Mangaonkar, AA; Mara, KC; Merten, JA; Pawlenty, AG; Shah, MV, 2022
)
0.95
" We found that the traditional fixed weight-based dosing of propylene glycol-free (captisol-enabled) melphalan in BEAM results in a wide variation in exposure as estimated by both models."( Evaluation of Melphalan Exposure in Lymphoma Patients Undergoing BEAM and Autologous Hematopoietic Cell Transplantation.
Admiraal, R; Boelens, JJ; Carlow, D; Cho, C; Dahi, PB; DeRespiris, L; Devlin, SM; Flynn, JR; Giralt, SA; Lahoud, OB; Lin, A; Perales, MA; Ruiz, JD; Sauter, CS; Scordo, M; Shah, GL, 2022
)
1.3
" Various dosing schedules and modifications, timing of administration, and novel drugs-based combination regimens are discussed."( High dose (conditioning) regimens used prior to autologous stem cell transplantation in multiple myeloma.
Al Hadidi, S; Ali, MO, 2022
)
0.72
" So, time of exposure as well as the amount of exposure (total dosage administered) is critical in determining the magnitude of the damage in germ cell risk assessment."( Juvenile exposure and adult risk assessment with single versus repeated exposure of melphalan in the germ cells of male SD rat: Deciphering the molecular mechanisms.
Jena, G; Panghal, A; Sahu, C; Singla, S, 2022
)
0.95
" Future studies will aim to modulate melphalan exposure by PK-targeted dosing and characterize patient taste preferences to personalize diets for improved nutritional intake."( A prospective study of dysgeusia and related symptoms in patients with multiple myeloma after autologous hematopoietic cell transplantation.
Adintori, PA; Barasch, A; Buchan, ML; Carino, CA; Carlow, DC; Chung, DJ; Devlin, SM; Giralt, SA; Klang, MG; Knezevic, A; Lahoud, OB; Landau, HJ; Lin, AP; Maloy, MA; Mastrogiacomo, B; Nguyen, LK; Peled, JU; Preston, EV; Rodriguez, NT; Schofield, RC; Scordo, M; Shah, GL; Sitner, NC; Slingerland, AE; Slingerland, JB; Stein-Thoeringer, CK; van den Brink, MRM, 2022
)
0.99
" A protocol with repeated 10-day cyclical dosing of melphalan has been used at our institution but has not been described in the literature."( Cyclical 10-day dosing of melphalan for canine multiple myeloma.
Hume, KR; O'Connor, KS; Sylvester, SR; Teddy, L, 2023
)
1.46
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (5)

RoleDescription
antineoplastic agentA substance that inhibits or prevents the proliferation of neoplasms.
carcinogenic agentA role played by a chemical compound which is known to induce a process of carcinogenesis by corrupting normal cellular pathways, leading to the acquistion of tumoral capabilities.
alkylating agentHighly reactive chemical that introduces alkyl radicals into biologically active molecules and thereby prevents their proper functioning. It could be used as an antineoplastic agent, but it might be very toxic, with carcinogenic, mutagenic, teratogenic, and immunosuppressant actions. It could also be used as a component of poison gases.
immunosuppressive agentAn agent that suppresses immune function by one of several mechanisms of action. Classical cytotoxic immunosuppressants act by inhibiting DNA synthesis. Others may act through activation of T-cells or by inhibiting the activation of helper cells. In addition, an immunosuppressive agent is a role played by a compound which is exhibited by a capability to diminish the extent and/or voracity of an immune response.
drug allergenAny drug which causes the onset of an allergic reaction.
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (4)

ClassDescription
organochlorine compoundAn organochlorine compound is a compound containing at least one carbon-chlorine bond.
nitrogen mustardCompounds having two beta-haloalkyl groups bound to a nitrogen atom, as in (X-CH2-CH2)2NR.
L-phenylalanine derivativeA proteinogenic amino acid derivative resulting from reaction of L-phenylalanine at the amino group or the carboxy group, or from the replacement of any hydrogen of L-phenylalanine by a heteroatom.
non-proteinogenic L-alpha-amino acidAny L-alpha-amino acid which is not a member of the group of 23 proteinogenic amino acids.
[compound class information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Protein Targets (87)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, TYROSYL-DNA PHOSPHODIESTERASEHomo sapiens (human)Potency56.23410.004023.8416100.0000AID485290
glp-1 receptor, partialHomo sapiens (human)Potency5.62340.01846.806014.1254AID624417
thioredoxin reductaseRattus norvegicus (Norway rat)Potency79.43280.100020.879379.4328AID588453
15-lipoxygenase, partialHomo sapiens (human)Potency31.62280.012610.691788.5700AID887
phosphopantetheinyl transferaseBacillus subtilisPotency5.62340.141337.9142100.0000AID1490
RAR-related orphan receptor gammaMus musculus (house mouse)Potency11.23450.006038.004119,952.5996AID1159521; AID1159523
Fumarate hydrataseHomo sapiens (human)Potency16.72590.00308.794948.0869AID1347053
USP1 protein, partialHomo sapiens (human)Potency89.12510.031637.5844354.8130AID504865
GLS proteinHomo sapiens (human)Potency22.38720.35487.935539.8107AID624170
TDP1 proteinHomo sapiens (human)Potency0.38800.000811.382244.6684AID686978; AID686979
GLI family zinc finger 3Homo sapiens (human)Potency26.58940.000714.592883.7951AID1259392
AR proteinHomo sapiens (human)Potency26.22650.000221.22318,912.5098AID1259243; AID1259247; AID588516; AID743035; AID743036; AID743042; AID743053; AID743054; AID743063
thioredoxin glutathione reductaseSchistosoma mansoniPotency14.12540.100022.9075100.0000AID485364
Smad3Homo sapiens (human)Potency10.00000.00527.809829.0929AID588855
caspase 7, apoptosis-related cysteine proteaseHomo sapiens (human)Potency31.40160.013326.981070.7614AID1346978
aldehyde dehydrogenase 1 family, member A1Homo sapiens (human)Potency2.81840.011212.4002100.0000AID1030
hypoxia-inducible factor 1, alpha subunit (basic helix-loop-helix transcription factor)Homo sapiens (human)Potency50.11870.00137.762544.6684AID2120
estrogen receptor 2 (ER beta)Homo sapiens (human)Potency32.95400.000657.913322,387.1992AID1259377
nuclear receptor subfamily 1, group I, member 3Homo sapiens (human)Potency9.12860.001022.650876.6163AID1224838; AID1224893
progesterone receptorHomo sapiens (human)Potency13.33320.000417.946075.1148AID1346784
cytochrome P450 family 3 subfamily A polypeptide 4Homo sapiens (human)Potency13.24590.01237.983543.2770AID1346984; AID1645841
EWS/FLI fusion proteinHomo sapiens (human)Potency3.58540.001310.157742.8575AID1259252; AID1259253; AID1259255; AID1259256
retinoic acid nuclear receptor alpha variant 1Homo sapiens (human)Potency17.94650.003041.611522,387.1992AID1159552; AID1159553; AID1159555
retinoid X nuclear receptor alphaHomo sapiens (human)Potency19.40200.000817.505159.3239AID1159527; AID1159531; AID588544
estrogen-related nuclear receptor alphaHomo sapiens (human)Potency14.07300.001530.607315,848.9004AID1224841; AID1224842; AID1224848; AID1224849; AID1259401; AID1259403
farnesoid X nuclear receptorHomo sapiens (human)Potency27.82980.375827.485161.6524AID588526; AID588527
pregnane X nuclear receptorHomo sapiens (human)Potency31.98790.005428.02631,258.9301AID1346982; AID1346985; AID720659
estrogen nuclear receptor alphaHomo sapiens (human)Potency19.89400.000229.305416,493.5996AID1259244; AID1259248; AID588513; AID588514; AID743069; AID743075; AID743079; AID743080; AID743091
GVesicular stomatitis virusPotency38.90180.01238.964839.8107AID1645842
cytochrome P450 2D6Homo sapiens (human)Potency19.49710.00108.379861.1304AID1645840
polyproteinZika virusPotency16.72590.00308.794948.0869AID1347053
glucocerebrosidaseHomo sapiens (human)Potency39.81070.01268.156944.6684AID2101
peroxisome proliferator-activated receptor deltaHomo sapiens (human)Potency32.32540.001024.504861.6448AID588534; AID588535
peroxisome proliferator activated receptor gammaHomo sapiens (human)Potency30.79810.001019.414170.9645AID588536; AID588537; AID743140; AID743191
vitamin D (1,25- dihydroxyvitamin D3) receptorHomo sapiens (human)Potency44.66840.023723.228263.5986AID588543
caspase-3Homo sapiens (human)Potency31.40160.013326.981070.7614AID1346978
euchromatic histone-lysine N-methyltransferase 2Homo sapiens (human)Potency5.22760.035520.977089.1251AID504332
aryl hydrocarbon receptorHomo sapiens (human)Potency33.22270.000723.06741,258.9301AID743085; AID743122
cytochrome P450, family 19, subfamily A, polypeptide 1, isoform CRA_aHomo sapiens (human)Potency33.22270.001723.839378.1014AID743083
Histone H2A.xCricetulus griseus (Chinese hamster)Potency34.57640.039147.5451146.8240AID1224845; AID1224896
Caspase-7Cricetulus griseus (Chinese hamster)Potency33.49150.006723.496068.5896AID1346980
potassium voltage-gated channel subfamily H member 2 isoform dHomo sapiens (human)Potency15.84890.01789.637444.6684AID588834
caspase-3Cricetulus griseus (Chinese hamster)Potency33.49150.006723.496068.5896AID1346980
thyroid hormone receptor beta isoform 2Rattus norvegicus (Norway rat)Potency6.92070.000323.4451159.6830AID743065; AID743067
importin subunit beta-1 isoform 1Homo sapiens (human)Potency125.89205.804836.130665.1308AID540263
snurportin-1Homo sapiens (human)Potency125.89205.804836.130665.1308AID540263
nuclear factor erythroid 2-related factor 2 isoform 1Homo sapiens (human)Potency54.48820.000627.21521,122.0200AID651741; AID720636
urokinase-type plasminogen activator precursorMus musculus (house mouse)Potency2.51190.15855.287912.5893AID540303
plasminogen precursorMus musculus (house mouse)Potency2.51190.15855.287912.5893AID540303
urokinase plasminogen activator surface receptor precursorMus musculus (house mouse)Potency2.51190.15855.287912.5893AID540303
gemininHomo sapiens (human)Potency9.82850.004611.374133.4983AID624296; AID624297
peripheral myelin protein 22Rattus norvegicus (Norway rat)Potency9.53330.005612.367736.1254AID624032; AID624044
survival motor neuron protein isoform dHomo sapiens (human)Potency14.12540.125912.234435.4813AID1458
cytochrome P450 3A4 isoform 1Homo sapiens (human)Potency3.16230.031610.279239.8107AID884; AID885
Gamma-aminobutyric acid receptor subunit piRattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Voltage-dependent calcium channel gamma-2 subunitMus musculus (house mouse)Potency18.41460.001557.789015,848.9004AID1259244
Interferon betaHomo sapiens (human)Potency38.90180.00339.158239.8107AID1645842
HLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)Potency38.90180.01238.964839.8107AID1645842
Cellular tumor antigen p53Homo sapiens (human)Potency25.07880.002319.595674.0614AID651631; AID651743; AID720552
Gamma-aminobutyric acid receptor subunit beta-1Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit deltaRattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-2Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Glutamate receptor 2Rattus norvegicus (Norway rat)Potency18.41460.001551.739315,848.9004AID1259244
Gamma-aminobutyric acid receptor subunit alpha-5Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-3Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-1Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-2Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-4Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit gamma-3Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit alpha-6Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Nuclear receptor ROR-gammaHomo sapiens (human)Potency26.60320.026622.448266.8242AID651802
Spike glycoproteinSevere acute respiratory syndrome-related coronavirusPotency31.62280.009610.525035.4813AID1479145
Gamma-aminobutyric acid receptor subunit alpha-1Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit beta-3Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Gamma-aminobutyric acid receptor subunit beta-2Rattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
TAR DNA-binding protein 43Homo sapiens (human)Potency31.62281.778316.208135.4813AID652104
GABA theta subunitRattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
Inositol hexakisphosphate kinase 1Homo sapiens (human)Potency38.90180.01238.964839.8107AID1645842
ATPase family AAA domain-containing protein 5Homo sapiens (human)Potency30.43320.011917.942071.5630AID651632; AID720516
Ataxin-2Homo sapiens (human)Potency29.84930.011912.222168.7989AID651632
Gamma-aminobutyric acid receptor subunit epsilonRattus norvegicus (Norway rat)Potency3.16231.000012.224831.6228AID885
cytochrome P450 2C9, partialHomo sapiens (human)Potency38.90180.01238.964839.8107AID1645842
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Inhibition Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
ATP-binding cassette sub-family C member 3Homo sapiens (human)IC50 (µMol)133.00000.63154.45319.3000AID1473740
Multidrug resistance-associated protein 4Homo sapiens (human)IC50 (µMol)133.00000.20005.677410.0000AID1473741
Bile salt export pumpHomo sapiens (human)IC50 (µMol)113.37500.11007.190310.0000AID1449628; AID1473738
Canalicular multispecific organic anion transporter 1Homo sapiens (human)IC50 (µMol)133.00002.41006.343310.0000AID1473739
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (250)

Processvia Protein(s)Taxonomy
xenobiotic metabolic processATP-binding cassette sub-family C member 3Homo sapiens (human)
xenobiotic transmembrane transportATP-binding cassette sub-family C member 3Homo sapiens (human)
bile acid and bile salt transportATP-binding cassette sub-family C member 3Homo sapiens (human)
glucuronoside transportATP-binding cassette sub-family C member 3Homo sapiens (human)
xenobiotic transportATP-binding cassette sub-family C member 3Homo sapiens (human)
transmembrane transportATP-binding cassette sub-family C member 3Homo sapiens (human)
leukotriene transportATP-binding cassette sub-family C member 3Homo sapiens (human)
monoatomic anion transmembrane transportATP-binding cassette sub-family C member 3Homo sapiens (human)
transport across blood-brain barrierATP-binding cassette sub-family C member 3Homo sapiens (human)
prostaglandin secretionMultidrug resistance-associated protein 4Homo sapiens (human)
cilium assemblyMultidrug resistance-associated protein 4Homo sapiens (human)
platelet degranulationMultidrug resistance-associated protein 4Homo sapiens (human)
xenobiotic metabolic processMultidrug resistance-associated protein 4Homo sapiens (human)
xenobiotic transmembrane transportMultidrug resistance-associated protein 4Homo sapiens (human)
bile acid and bile salt transportMultidrug resistance-associated protein 4Homo sapiens (human)
prostaglandin transportMultidrug resistance-associated protein 4Homo sapiens (human)
urate transportMultidrug resistance-associated protein 4Homo sapiens (human)
glutathione transmembrane transportMultidrug resistance-associated protein 4Homo sapiens (human)
transmembrane transportMultidrug resistance-associated protein 4Homo sapiens (human)
cAMP transportMultidrug resistance-associated protein 4Homo sapiens (human)
leukotriene transportMultidrug resistance-associated protein 4Homo sapiens (human)
monoatomic anion transmembrane transportMultidrug resistance-associated protein 4Homo sapiens (human)
export across plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
transport across blood-brain barrierMultidrug resistance-associated protein 4Homo sapiens (human)
guanine nucleotide transmembrane transportMultidrug resistance-associated protein 4Homo sapiens (human)
fatty acid metabolic processBile salt export pumpHomo sapiens (human)
bile acid biosynthetic processBile salt export pumpHomo sapiens (human)
xenobiotic metabolic processBile salt export pumpHomo sapiens (human)
xenobiotic transmembrane transportBile salt export pumpHomo sapiens (human)
response to oxidative stressBile salt export pumpHomo sapiens (human)
bile acid metabolic processBile salt export pumpHomo sapiens (human)
response to organic cyclic compoundBile salt export pumpHomo sapiens (human)
bile acid and bile salt transportBile salt export pumpHomo sapiens (human)
canalicular bile acid transportBile salt export pumpHomo sapiens (human)
protein ubiquitinationBile salt export pumpHomo sapiens (human)
regulation of fatty acid beta-oxidationBile salt export pumpHomo sapiens (human)
carbohydrate transmembrane transportBile salt export pumpHomo sapiens (human)
bile acid signaling pathwayBile salt export pumpHomo sapiens (human)
cholesterol homeostasisBile salt export pumpHomo sapiens (human)
response to estrogenBile salt export pumpHomo sapiens (human)
response to ethanolBile salt export pumpHomo sapiens (human)
xenobiotic export from cellBile salt export pumpHomo sapiens (human)
lipid homeostasisBile salt export pumpHomo sapiens (human)
phospholipid homeostasisBile salt export pumpHomo sapiens (human)
positive regulation of bile acid secretionBile salt export pumpHomo sapiens (human)
regulation of bile acid metabolic processBile salt export pumpHomo sapiens (human)
transmembrane transportBile salt export pumpHomo sapiens (human)
cell surface receptor signaling pathway via JAK-STATInterferon betaHomo sapiens (human)
response to exogenous dsRNAInterferon betaHomo sapiens (human)
B cell activation involved in immune responseInterferon betaHomo sapiens (human)
cell surface receptor signaling pathwayInterferon betaHomo sapiens (human)
cell surface receptor signaling pathway via JAK-STATInterferon betaHomo sapiens (human)
response to virusInterferon betaHomo sapiens (human)
positive regulation of autophagyInterferon betaHomo sapiens (human)
cytokine-mediated signaling pathwayInterferon betaHomo sapiens (human)
natural killer cell activationInterferon betaHomo sapiens (human)
positive regulation of peptidyl-serine phosphorylation of STAT proteinInterferon betaHomo sapiens (human)
cellular response to interferon-betaInterferon betaHomo sapiens (human)
B cell proliferationInterferon betaHomo sapiens (human)
negative regulation of viral genome replicationInterferon betaHomo sapiens (human)
innate immune responseInterferon betaHomo sapiens (human)
positive regulation of innate immune responseInterferon betaHomo sapiens (human)
regulation of MHC class I biosynthetic processInterferon betaHomo sapiens (human)
negative regulation of T cell differentiationInterferon betaHomo sapiens (human)
positive regulation of transcription by RNA polymerase IIInterferon betaHomo sapiens (human)
defense response to virusInterferon betaHomo sapiens (human)
type I interferon-mediated signaling pathwayInterferon betaHomo sapiens (human)
neuron cellular homeostasisInterferon betaHomo sapiens (human)
cellular response to exogenous dsRNAInterferon betaHomo sapiens (human)
cellular response to virusInterferon betaHomo sapiens (human)
negative regulation of Lewy body formationInterferon betaHomo sapiens (human)
negative regulation of T-helper 2 cell cytokine productionInterferon betaHomo sapiens (human)
positive regulation of apoptotic signaling pathwayInterferon betaHomo sapiens (human)
response to exogenous dsRNAInterferon betaHomo sapiens (human)
B cell differentiationInterferon betaHomo sapiens (human)
natural killer cell activation involved in immune responseInterferon betaHomo sapiens (human)
adaptive immune responseInterferon betaHomo sapiens (human)
T cell activation involved in immune responseInterferon betaHomo sapiens (human)
humoral immune responseInterferon betaHomo sapiens (human)
positive regulation of T cell mediated cytotoxicityHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
adaptive immune responseHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
antigen processing and presentation of endogenous peptide antigen via MHC class I via ER pathway, TAP-independentHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
regulation of T cell anergyHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
defense responseHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
immune responseHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
detection of bacteriumHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
regulation of interleukin-12 productionHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
regulation of interleukin-6 productionHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
protection from natural killer cell mediated cytotoxicityHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
innate immune responseHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
regulation of dendritic cell differentiationHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
antigen processing and presentation of endogenous peptide antigen via MHC class IbHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycle G2/M phase transitionCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
ER overload responseCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
mitophagyCellular tumor antigen p53Homo sapiens (human)
in utero embryonic developmentCellular tumor antigen p53Homo sapiens (human)
somitogenesisCellular tumor antigen p53Homo sapiens (human)
release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
hematopoietic progenitor cell differentiationCellular tumor antigen p53Homo sapiens (human)
T cell proliferation involved in immune responseCellular tumor antigen p53Homo sapiens (human)
B cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
T cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
response to ischemiaCellular tumor antigen p53Homo sapiens (human)
nucleotide-excision repairCellular tumor antigen p53Homo sapiens (human)
double-strand break repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
protein import into nucleusCellular tumor antigen p53Homo sapiens (human)
autophagyCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in cell cycle arrestCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in transcription of p21 class mediatorCellular tumor antigen p53Homo sapiens (human)
transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
Ras protein signal transductionCellular tumor antigen p53Homo sapiens (human)
gastrulationCellular tumor antigen p53Homo sapiens (human)
neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
protein localizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA replicationCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
determination of adult lifespanCellular tumor antigen p53Homo sapiens (human)
mRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
rRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
response to salt stressCellular tumor antigen p53Homo sapiens (human)
response to inorganic substanceCellular tumor antigen p53Homo sapiens (human)
response to X-rayCellular tumor antigen p53Homo sapiens (human)
response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
positive regulation of gene expressionCellular tumor antigen p53Homo sapiens (human)
cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
viral processCellular tumor antigen p53Homo sapiens (human)
glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
cerebellum developmentCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell growthCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
mitotic G1 DNA damage checkpoint signalingCellular tumor antigen p53Homo sapiens (human)
negative regulation of telomere maintenance via telomeraseCellular tumor antigen p53Homo sapiens (human)
T cell differentiation in thymusCellular tumor antigen p53Homo sapiens (human)
tumor necrosis factor-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
regulation of tissue remodelingCellular tumor antigen p53Homo sapiens (human)
cellular response to UVCellular tumor antigen p53Homo sapiens (human)
multicellular organism growthCellular tumor antigen p53Homo sapiens (human)
positive regulation of mitochondrial membrane permeabilityCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
entrainment of circadian clock by photoperiodCellular tumor antigen p53Homo sapiens (human)
mitochondrial DNA repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
transcription initiation-coupled chromatin remodelingCellular tumor antigen p53Homo sapiens (human)
negative regulation of proteolysisCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of RNA polymerase II transcription preinitiation complex assemblyCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
response to antibioticCellular tumor antigen p53Homo sapiens (human)
fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
circadian behaviorCellular tumor antigen p53Homo sapiens (human)
bone marrow developmentCellular tumor antigen p53Homo sapiens (human)
embryonic organ developmentCellular tumor antigen p53Homo sapiens (human)
positive regulation of peptidyl-tyrosine phosphorylationCellular tumor antigen p53Homo sapiens (human)
protein stabilizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of helicase activityCellular tumor antigen p53Homo sapiens (human)
protein tetramerizationCellular tumor antigen p53Homo sapiens (human)
chromosome organizationCellular tumor antigen p53Homo sapiens (human)
neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
hematopoietic stem cell differentiationCellular tumor antigen p53Homo sapiens (human)
negative regulation of glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
type II interferon-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
cardiac septum morphogenesisCellular tumor antigen p53Homo sapiens (human)
positive regulation of programmed necrotic cell deathCellular tumor antigen p53Homo sapiens (human)
protein-containing complex assemblyCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to endoplasmic reticulum stressCellular tumor antigen p53Homo sapiens (human)
thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
necroptotic processCellular tumor antigen p53Homo sapiens (human)
cellular response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
cellular response to xenobiotic stimulusCellular tumor antigen p53Homo sapiens (human)
cellular response to ionizing radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to UV-CCellular tumor antigen p53Homo sapiens (human)
stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
cellular response to actinomycin DCellular tumor antigen p53Homo sapiens (human)
positive regulation of release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
cellular senescenceCellular tumor antigen p53Homo sapiens (human)
replicative senescenceCellular tumor antigen p53Homo sapiens (human)
oxidative stress-induced premature senescenceCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
oligodendrocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of execution phase of apoptosisCellular tumor antigen p53Homo sapiens (human)
negative regulation of mitophagyCellular tumor antigen p53Homo sapiens (human)
regulation of mitochondrial membrane permeability involved in apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of G1 to G0 transitionCellular tumor antigen p53Homo sapiens (human)
negative regulation of miRNA processingCellular tumor antigen p53Homo sapiens (human)
negative regulation of glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
negative regulation of pentose-phosphate shuntCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
regulation of fibroblast apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
positive regulation of cellular senescenceCellular tumor antigen p53Homo sapiens (human)
positive regulation of intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
negative regulation of transcription by RNA polymerase IINuclear receptor ROR-gammaHomo sapiens (human)
xenobiotic metabolic processNuclear receptor ROR-gammaHomo sapiens (human)
regulation of glucose metabolic processNuclear receptor ROR-gammaHomo sapiens (human)
regulation of steroid metabolic processNuclear receptor ROR-gammaHomo sapiens (human)
intracellular receptor signaling pathwayNuclear receptor ROR-gammaHomo sapiens (human)
circadian regulation of gene expressionNuclear receptor ROR-gammaHomo sapiens (human)
cellular response to sterolNuclear receptor ROR-gammaHomo sapiens (human)
positive regulation of circadian rhythmNuclear receptor ROR-gammaHomo sapiens (human)
regulation of fat cell differentiationNuclear receptor ROR-gammaHomo sapiens (human)
positive regulation of DNA-templated transcriptionNuclear receptor ROR-gammaHomo sapiens (human)
adipose tissue developmentNuclear receptor ROR-gammaHomo sapiens (human)
T-helper 17 cell differentiationNuclear receptor ROR-gammaHomo sapiens (human)
regulation of transcription by RNA polymerase IINuclear receptor ROR-gammaHomo sapiens (human)
negative regulation of protein phosphorylationTAR DNA-binding protein 43Homo sapiens (human)
mRNA processingTAR DNA-binding protein 43Homo sapiens (human)
RNA splicingTAR DNA-binding protein 43Homo sapiens (human)
negative regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
regulation of protein stabilityTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of insulin secretionTAR DNA-binding protein 43Homo sapiens (human)
response to endoplasmic reticulum stressTAR DNA-binding protein 43Homo sapiens (human)
positive regulation of protein import into nucleusTAR DNA-binding protein 43Homo sapiens (human)
regulation of circadian rhythmTAR DNA-binding protein 43Homo sapiens (human)
regulation of apoptotic processTAR DNA-binding protein 43Homo sapiens (human)
negative regulation by host of viral transcriptionTAR DNA-binding protein 43Homo sapiens (human)
rhythmic processTAR DNA-binding protein 43Homo sapiens (human)
regulation of cell cycleTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA destabilizationTAR DNA-binding protein 43Homo sapiens (human)
3'-UTR-mediated mRNA stabilizationTAR DNA-binding protein 43Homo sapiens (human)
nuclear inner membrane organizationTAR DNA-binding protein 43Homo sapiens (human)
amyloid fibril formationTAR DNA-binding protein 43Homo sapiens (human)
regulation of gene expressionTAR DNA-binding protein 43Homo sapiens (human)
inositol phosphate metabolic processInositol hexakisphosphate kinase 1Homo sapiens (human)
phosphatidylinositol phosphate biosynthetic processInositol hexakisphosphate kinase 1Homo sapiens (human)
negative regulation of cold-induced thermogenesisInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol phosphate biosynthetic processInositol hexakisphosphate kinase 1Homo sapiens (human)
xenobiotic metabolic processCanalicular multispecific organic anion transporter 1Homo sapiens (human)
xenobiotic transmembrane transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
negative regulation of gene expressionCanalicular multispecific organic anion transporter 1Homo sapiens (human)
bile acid and bile salt transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
bilirubin transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
heme catabolic processCanalicular multispecific organic anion transporter 1Homo sapiens (human)
xenobiotic export from cellCanalicular multispecific organic anion transporter 1Homo sapiens (human)
transmembrane transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
transepithelial transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
leukotriene transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
monoatomic anion transmembrane transportCanalicular multispecific organic anion transporter 1Homo sapiens (human)
transport across blood-brain barrierCanalicular multispecific organic anion transporter 1Homo sapiens (human)
xenobiotic transport across blood-brain barrierCanalicular multispecific organic anion transporter 1Homo sapiens (human)
cell population proliferationATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of B cell proliferationATPase family AAA domain-containing protein 5Homo sapiens (human)
nuclear DNA replicationATPase family AAA domain-containing protein 5Homo sapiens (human)
signal transduction in response to DNA damageATPase family AAA domain-containing protein 5Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorATPase family AAA domain-containing protein 5Homo sapiens (human)
isotype switchingATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of DNA replicationATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of isotype switching to IgG isotypesATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA clamp unloadingATPase family AAA domain-containing protein 5Homo sapiens (human)
regulation of mitotic cell cycle phase transitionATPase family AAA domain-containing protein 5Homo sapiens (human)
negative regulation of intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorATPase family AAA domain-containing protein 5Homo sapiens (human)
positive regulation of cell cycle G2/M phase transitionATPase family AAA domain-containing protein 5Homo sapiens (human)
negative regulation of receptor internalizationAtaxin-2Homo sapiens (human)
regulation of translationAtaxin-2Homo sapiens (human)
RNA metabolic processAtaxin-2Homo sapiens (human)
P-body assemblyAtaxin-2Homo sapiens (human)
stress granule assemblyAtaxin-2Homo sapiens (human)
RNA transportAtaxin-2Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (84)

Processvia Protein(s)Taxonomy
ATP bindingATP-binding cassette sub-family C member 3Homo sapiens (human)
ABC-type xenobiotic transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
glucuronoside transmembrane transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ABC-type glutathione S-conjugate transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ABC-type bile acid transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ATP hydrolysis activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ATPase-coupled transmembrane transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
xenobiotic transmembrane transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ATPase-coupled inorganic anion transmembrane transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
icosanoid transmembrane transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
ABC-type transporter activityATP-binding cassette sub-family C member 3Homo sapiens (human)
guanine nucleotide transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
protein bindingMultidrug resistance-associated protein 4Homo sapiens (human)
ATP bindingMultidrug resistance-associated protein 4Homo sapiens (human)
ABC-type xenobiotic transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
prostaglandin transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
urate transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
purine nucleotide transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
ABC-type glutathione S-conjugate transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
ABC-type bile acid transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
efflux transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
15-hydroxyprostaglandin dehydrogenase (NAD+) activityMultidrug resistance-associated protein 4Homo sapiens (human)
ATP hydrolysis activityMultidrug resistance-associated protein 4Homo sapiens (human)
glutathione transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
ATPase-coupled transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
xenobiotic transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
ATPase-coupled inorganic anion transmembrane transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
ABC-type transporter activityMultidrug resistance-associated protein 4Homo sapiens (human)
protein bindingBile salt export pumpHomo sapiens (human)
ATP bindingBile salt export pumpHomo sapiens (human)
ABC-type xenobiotic transporter activityBile salt export pumpHomo sapiens (human)
bile acid transmembrane transporter activityBile salt export pumpHomo sapiens (human)
canalicular bile acid transmembrane transporter activityBile salt export pumpHomo sapiens (human)
carbohydrate transmembrane transporter activityBile salt export pumpHomo sapiens (human)
ABC-type bile acid transporter activityBile salt export pumpHomo sapiens (human)
ATP hydrolysis activityBile salt export pumpHomo sapiens (human)
cytokine activityInterferon betaHomo sapiens (human)
cytokine receptor bindingInterferon betaHomo sapiens (human)
type I interferon receptor bindingInterferon betaHomo sapiens (human)
protein bindingInterferon betaHomo sapiens (human)
chloramphenicol O-acetyltransferase activityInterferon betaHomo sapiens (human)
TAP bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
signaling receptor bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
protein bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
peptide antigen bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
TAP bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
protein-folding chaperone bindingHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
transcription cis-regulatory region bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
core promoter sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
TFIID-class transcription factor complex bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription repressor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription activator activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
protease bindingCellular tumor antigen p53Homo sapiens (human)
p53 bindingCellular tumor antigen p53Homo sapiens (human)
DNA bindingCellular tumor antigen p53Homo sapiens (human)
chromatin bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activityCellular tumor antigen p53Homo sapiens (human)
mRNA 3'-UTR bindingCellular tumor antigen p53Homo sapiens (human)
copper ion bindingCellular tumor antigen p53Homo sapiens (human)
protein bindingCellular tumor antigen p53Homo sapiens (human)
zinc ion bindingCellular tumor antigen p53Homo sapiens (human)
enzyme bindingCellular tumor antigen p53Homo sapiens (human)
receptor tyrosine kinase bindingCellular tumor antigen p53Homo sapiens (human)
ubiquitin protein ligase bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase regulator activityCellular tumor antigen p53Homo sapiens (human)
ATP-dependent DNA/DNA annealing activityCellular tumor antigen p53Homo sapiens (human)
identical protein bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase bindingCellular tumor antigen p53Homo sapiens (human)
protein heterodimerization activityCellular tumor antigen p53Homo sapiens (human)
protein-folding chaperone bindingCellular tumor antigen p53Homo sapiens (human)
protein phosphatase 2A bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II-specific DNA-binding transcription factor bindingCellular tumor antigen p53Homo sapiens (human)
14-3-3 protein bindingCellular tumor antigen p53Homo sapiens (human)
MDM2/MDM4 family protein bindingCellular tumor antigen p53Homo sapiens (human)
disordered domain specific bindingCellular tumor antigen p53Homo sapiens (human)
general transcription initiation factor bindingCellular tumor antigen p53Homo sapiens (human)
molecular function activator activityCellular tumor antigen p53Homo sapiens (human)
promoter-specific chromatin bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingNuclear receptor ROR-gammaHomo sapiens (human)
DNA-binding transcription factor activity, RNA polymerase II-specificNuclear receptor ROR-gammaHomo sapiens (human)
DNA-binding transcription repressor activity, RNA polymerase II-specificNuclear receptor ROR-gammaHomo sapiens (human)
DNA-binding transcription factor activityNuclear receptor ROR-gammaHomo sapiens (human)
protein bindingNuclear receptor ROR-gammaHomo sapiens (human)
oxysterol bindingNuclear receptor ROR-gammaHomo sapiens (human)
zinc ion bindingNuclear receptor ROR-gammaHomo sapiens (human)
ligand-activated transcription factor activityNuclear receptor ROR-gammaHomo sapiens (human)
sequence-specific double-stranded DNA bindingNuclear receptor ROR-gammaHomo sapiens (human)
nuclear receptor activityNuclear receptor ROR-gammaHomo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
double-stranded DNA bindingTAR DNA-binding protein 43Homo sapiens (human)
RNA bindingTAR DNA-binding protein 43Homo sapiens (human)
mRNA 3'-UTR bindingTAR DNA-binding protein 43Homo sapiens (human)
protein bindingTAR DNA-binding protein 43Homo sapiens (human)
lipid bindingTAR DNA-binding protein 43Homo sapiens (human)
identical protein bindingTAR DNA-binding protein 43Homo sapiens (human)
pre-mRNA intronic bindingTAR DNA-binding protein 43Homo sapiens (human)
molecular condensate scaffold activityTAR DNA-binding protein 43Homo sapiens (human)
inositol-1,3,4,5,6-pentakisphosphate kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol hexakisphosphate kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol heptakisphosphate kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol hexakisphosphate 5-kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
protein bindingInositol hexakisphosphate kinase 1Homo sapiens (human)
ATP bindingInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol hexakisphosphate 1-kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol hexakisphosphate 3-kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol 5-diphosphate pentakisphosphate 5-kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
inositol diphosphate tetrakisphosphate kinase activityInositol hexakisphosphate kinase 1Homo sapiens (human)
protein bindingCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ATP bindingCanalicular multispecific organic anion transporter 1Homo sapiens (human)
organic anion transmembrane transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ABC-type xenobiotic transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
bilirubin transmembrane transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ABC-type glutathione S-conjugate transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ATP hydrolysis activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ATPase-coupled transmembrane transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
xenobiotic transmembrane transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ATPase-coupled inorganic anion transmembrane transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
ABC-type transporter activityCanalicular multispecific organic anion transporter 1Homo sapiens (human)
protein bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
ATP bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
ATP hydrolysis activityATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA clamp unloader activityATPase family AAA domain-containing protein 5Homo sapiens (human)
DNA bindingATPase family AAA domain-containing protein 5Homo sapiens (human)
RNA bindingAtaxin-2Homo sapiens (human)
epidermal growth factor receptor bindingAtaxin-2Homo sapiens (human)
protein bindingAtaxin-2Homo sapiens (human)
mRNA bindingAtaxin-2Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (55)

Processvia Protein(s)Taxonomy
plasma membraneATP-binding cassette sub-family C member 3Homo sapiens (human)
basal plasma membraneATP-binding cassette sub-family C member 3Homo sapiens (human)
basolateral plasma membraneATP-binding cassette sub-family C member 3Homo sapiens (human)
membraneATP-binding cassette sub-family C member 3Homo sapiens (human)
nucleolusMultidrug resistance-associated protein 4Homo sapiens (human)
Golgi apparatusMultidrug resistance-associated protein 4Homo sapiens (human)
plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
membraneMultidrug resistance-associated protein 4Homo sapiens (human)
basolateral plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
apical plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
platelet dense granule membraneMultidrug resistance-associated protein 4Homo sapiens (human)
external side of apical plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
plasma membraneMultidrug resistance-associated protein 4Homo sapiens (human)
basolateral plasma membraneBile salt export pumpHomo sapiens (human)
Golgi membraneBile salt export pumpHomo sapiens (human)
endosomeBile salt export pumpHomo sapiens (human)
plasma membraneBile salt export pumpHomo sapiens (human)
cell surfaceBile salt export pumpHomo sapiens (human)
apical plasma membraneBile salt export pumpHomo sapiens (human)
intercellular canaliculusBile salt export pumpHomo sapiens (human)
intracellular canaliculusBile salt export pumpHomo sapiens (human)
recycling endosomeBile salt export pumpHomo sapiens (human)
recycling endosome membraneBile salt export pumpHomo sapiens (human)
extracellular exosomeBile salt export pumpHomo sapiens (human)
membraneBile salt export pumpHomo sapiens (human)
extracellular spaceInterferon betaHomo sapiens (human)
extracellular regionInterferon betaHomo sapiens (human)
Golgi membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
endoplasmic reticulumHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
Golgi apparatusHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
plasma membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
cell surfaceHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
ER to Golgi transport vesicle membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
secretory granule membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
phagocytic vesicle membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
early endosome membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
recycling endosome membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
extracellular exosomeHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
lumenal side of endoplasmic reticulum membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
MHC class I protein complexHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
extracellular spaceHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
external side of plasma membraneHLA class I histocompatibility antigen, B alpha chain Homo sapiens (human)
nuclear bodyCellular tumor antigen p53Homo sapiens (human)
nucleusCellular tumor antigen p53Homo sapiens (human)
nucleoplasmCellular tumor antigen p53Homo sapiens (human)
replication forkCellular tumor antigen p53Homo sapiens (human)
nucleolusCellular tumor antigen p53Homo sapiens (human)
cytoplasmCellular tumor antigen p53Homo sapiens (human)
mitochondrionCellular tumor antigen p53Homo sapiens (human)
mitochondrial matrixCellular tumor antigen p53Homo sapiens (human)
endoplasmic reticulumCellular tumor antigen p53Homo sapiens (human)
centrosomeCellular tumor antigen p53Homo sapiens (human)
cytosolCellular tumor antigen p53Homo sapiens (human)
nuclear matrixCellular tumor antigen p53Homo sapiens (human)
PML bodyCellular tumor antigen p53Homo sapiens (human)
transcription repressor complexCellular tumor antigen p53Homo sapiens (human)
site of double-strand breakCellular tumor antigen p53Homo sapiens (human)
germ cell nucleusCellular tumor antigen p53Homo sapiens (human)
chromatinCellular tumor antigen p53Homo sapiens (human)
transcription regulator complexCellular tumor antigen p53Homo sapiens (human)
protein-containing complexCellular tumor antigen p53Homo sapiens (human)
plasma membraneGamma-aminobutyric acid receptor subunit gamma-2Rattus norvegicus (Norway rat)
plasma membraneGlutamate receptor 2Rattus norvegicus (Norway rat)
nucleusNuclear receptor ROR-gammaHomo sapiens (human)
nucleoplasmNuclear receptor ROR-gammaHomo sapiens (human)
nuclear bodyNuclear receptor ROR-gammaHomo sapiens (human)
chromatinNuclear receptor ROR-gammaHomo sapiens (human)
nucleusNuclear receptor ROR-gammaHomo sapiens (human)
virion membraneSpike glycoproteinSevere acute respiratory syndrome-related coronavirus
plasma membraneGamma-aminobutyric acid receptor subunit alpha-1Rattus norvegicus (Norway rat)
plasma membraneGamma-aminobutyric acid receptor subunit beta-2Rattus norvegicus (Norway rat)
intracellular non-membrane-bounded organelleTAR DNA-binding protein 43Homo sapiens (human)
nucleusTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
perichromatin fibrilsTAR DNA-binding protein 43Homo sapiens (human)
mitochondrionTAR DNA-binding protein 43Homo sapiens (human)
cytoplasmic stress granuleTAR DNA-binding protein 43Homo sapiens (human)
nuclear speckTAR DNA-binding protein 43Homo sapiens (human)
interchromatin granuleTAR DNA-binding protein 43Homo sapiens (human)
nucleoplasmTAR DNA-binding protein 43Homo sapiens (human)
chromatinTAR DNA-binding protein 43Homo sapiens (human)
fibrillar centerInositol hexakisphosphate kinase 1Homo sapiens (human)
nucleoplasmInositol hexakisphosphate kinase 1Homo sapiens (human)
cytosolInositol hexakisphosphate kinase 1Homo sapiens (human)
nucleusInositol hexakisphosphate kinase 1Homo sapiens (human)
cytoplasmInositol hexakisphosphate kinase 1Homo sapiens (human)
plasma membraneCanalicular multispecific organic anion transporter 1Homo sapiens (human)
cell surfaceCanalicular multispecific organic anion transporter 1Homo sapiens (human)
apical plasma membraneCanalicular multispecific organic anion transporter 1Homo sapiens (human)
intercellular canaliculusCanalicular multispecific organic anion transporter 1Homo sapiens (human)
apical plasma membraneCanalicular multispecific organic anion transporter 1Homo sapiens (human)
Elg1 RFC-like complexATPase family AAA domain-containing protein 5Homo sapiens (human)
nucleusATPase family AAA domain-containing protein 5Homo sapiens (human)
cytoplasmAtaxin-2Homo sapiens (human)
Golgi apparatusAtaxin-2Homo sapiens (human)
trans-Golgi networkAtaxin-2Homo sapiens (human)
cytosolAtaxin-2Homo sapiens (human)
cytoplasmic stress granuleAtaxin-2Homo sapiens (human)
membraneAtaxin-2Homo sapiens (human)
perinuclear region of cytoplasmAtaxin-2Homo sapiens (human)
ribonucleoprotein complexAtaxin-2Homo sapiens (human)
cytoplasmic stress granuleAtaxin-2Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (687)

Assay IDTitleYearJournalArticle
AID504749qHTS profiling for inhibitors of Plasmodium falciparum proliferation2011Science (New York, N.Y.), Aug-05, Volume: 333, Issue:6043
Chemical genomic profiling for antimalarial therapies, response signatures, and molecular targets.
AID1346987P-glycoprotein substrates identified in KB-8-5-11 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1296008Cytotoxic Profiling of Annotated Libraries Using Quantitative High-Throughput Screening2020SLAS discovery : advancing life sciences R & D, 01, Volume: 25, Issue:1
Cytotoxic Profiling of Annotated and Diverse Chemical Libraries Using Quantitative High-Throughput Screening.
AID1347082qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: LASV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347104qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347094qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347106qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347096qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347099qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347095qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1508630Primary qHTS for small molecule stabilizers of the endoplasmic reticulum resident proteome: Secreted ER Calcium Modulated Protein (SERCaMP) assay2021Cell reports, 04-27, Volume: 35, Issue:4
A target-agnostic screen identifies approved drugs to stabilize the endoplasmic reticulum-resident proteome.
AID1347092qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for A673 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347108qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347103qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1346986P-glycoprotein substrates identified in KB-3-1 adenocarcinoma cell line, qHTS therapeutic library screen2019Molecular pharmacology, 11, Volume: 96, Issue:5
A High-Throughput Screen of a Library of Therapeutics Identifies Cytotoxic Substrates of P-glycoprotein.
AID1347154Primary screen GU AMC qHTS for Zika virus inhibitors2020Proceedings of the National Academy of Sciences of the United States of America, 12-08, Volume: 117, Issue:49
Therapeutic candidates for the Zika virus identified by a high-throughput screen for Zika protease inhibitors.
AID1347083qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lassa (LASV) Arenavirus: Viability assay - alamar blue signal for LASV Primary Screen2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1745845Primary qHTS for Inhibitors of ATXN expression
AID1347093qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347098qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347102qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347097qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347091qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347105qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347090qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347101qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347107qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347086qHTS for Inhibitors of the Functional Ribonucleoprotein Complex (vRNP) of Lymphocytic Choriomeningitis Arenaviruses (LCMV): LCMV Primary Screen - GLuc reporter signal2020Antiviral research, 01, Volume: 173A cell-based, infectious-free, platform to identify inhibitors of lassa virus ribonucleoprotein (vRNP) activity.
AID1347100qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347089qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1745850Viability Counterscreen for Confirmatory qHTS for Inhibitors of ATXN expression
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588499High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain A protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588497High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Botulinum neurotoxin light chain F protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID1745849Viability Counterscreen for CMV-Luciferase Assay of Inhibitors of ATXN expression
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Current protocols in cytometry, Oct, Volume: Chapter 13Microsphere-based flow cytometry protease assays for use in protease activity detection and high-throughput screening.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2006Cytometry. Part A : the journal of the International Society for Analytical Cytology, May, Volume: 69, Issue:5
Microsphere-based protease assays and screening application for lethal factor and factor Xa.
AID588501High-throughput multiplex microsphere screening for inhibitors of toxin protease, specifically Lethal Factor Protease, MLPCN compound set2010Assay and drug development technologies, Feb, Volume: 8, Issue:1
High-throughput multiplex flow cytometry screening for botulinum neurotoxin type a light chain protease inhibitors.
AID504810Antagonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
AID504812Inverse Agonists of the Thyroid Stimulating Hormone Receptor: HTS campaign2010Endocrinology, Jul, Volume: 151, Issue:7
A small molecule inverse agonist for the human thyroid-stimulating hormone receptor.
AID1745846Firefly Luciferase Counterscreen for Inhibitors of ATXN expression
AID1745848Confirmatory qHTS for Inhibitors of ATXN expression
AID1745847CMV-Luciferase Counterscreen for Inhibitors of ATXN expression
AID1347411qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Mechanism Interrogation Plate v5.0 (MIPE) Libary2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID654073Cytotoxicity against human HPLF cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1315402Cytotoxicity against human A549 cells after 72 hrs by MTT assay2016European journal of medicinal chemistry, Aug-25, Volume: 119Design and synthesis of novel pyrazolo[1,5-a]pyrimidine derivatives bearing nitrogen mustard moiety and evaluation of their antitumor activity in vitro and in vivo.
AID625288Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for jaundice2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1191478Cytostatic activity against human CEM cells2015Bioorganic & medicinal chemistry, Feb-01, Volume: 23, Issue:3
Investigation of fatty acid conjugates of 3,5-bisarylmethylene-4-piperidone derivatives as antitumor agents and human topoisomerase-IIα inhibitors.
AID496821Antimicrobial activity against Leishmania2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1185080Antiproliferative activity against human MCF7 cells assessed as growth inhibition after 72 hrs by MTT assay2014ACS medicinal chemistry letters, Jul-10, Volume: 5, Issue:7
Novel hybrids of natural oridonin-bearing nitrogen mustards as potential anticancer drug candidates.
AID50735Mean graph mid point was measured on colon cancer cells.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID45823Cytotoxicity against CEM T-lymphocytes.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1132956Antitumor activity against mouse Walker 256 cells allografted in Sprague-Dawley rat assessed as increase in survival rate at 2.5 mg/kg/day, ip relative to control1978Journal of medicinal chemistry, Aug, Volume: 21, Issue:8
Antitumor agents 32. Synthesis and antitumor activity of cyclopentenone derivatives related to helenalin.
AID496820Antimicrobial activity against Trypanosoma brucei2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1594193Selectivity ratio of IC50 for human SW620 cells under aerobic condition to IC50 for human SW620 cells under hypoxic condition2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID47647Concentration required to inhibit 50% of cell growth on CEM lymphocytes.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID396986Cytotoxicity against human Caov3 cells after 72 hrs by MTT assay2009European journal of medicinal chemistry, May, Volume: 44, Issue:5
Design, cytotoxic and fluorescent properties of novel N-phosphorylalkyl substituted E,E-3,5-bis(arylidene)piperid-4-ones.
AID384309Cytotoxicity against human HPC cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID1916652Cytotoxicity against human MCF7 cells assessed as cell growth inhibition incubated for 24 hrs by MTT assay2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID99876In vitro cytotoxicity against L1210 cells at a dose 10 ug/mL1989Journal of medicinal chemistry, May, Volume: 32, Issue:5
Synthesis of 5-[1-hydroxy(or methoxy)-2-bromo(or chloro)ethyl]-2'-deoxyuridines and related halohydrin analogues with antiviral and cytotoxic activity.
AID615291Antiproliferative activity against human MDA-MB-231 cells after 72 hrs by crystal violet staining2011European journal of medicinal chemistry, May, Volume: 46, Issue:5
Bivalent bendamustine and melphalan derivatives as anticancer agents.
AID458819Selectivity index, ratio of CC50 for human normal cells to CC50 for human HSC4 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID300175Cytotoxicity against human Molt 4/C8 cells2007Bioorganic & medicinal chemistry, Sep-01, Volume: 15, Issue:17
E,E,E-1-(4-Arylamino-4-oxo-2-butenoyl)-3,5-bis(arylidene)-4-piperidones: a topographical study of some novel potent cytotoxins.
AID1191479Cytostatic activity against human HeLa cells2015Bioorganic & medicinal chemistry, Feb-01, Volume: 23, Issue:3
Investigation of fatty acid conjugates of 3,5-bisarylmethylene-4-piperidone derivatives as antitumor agents and human topoisomerase-IIα inhibitors.
AID496829Antimicrobial activity against Leishmania infantum2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID224730Concentration required to inhibit 50% of cell growth on murine L1210 cells.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID1916664Plasma protein binding in human2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID200785The compound was tested for cytotoxicity against SF-188 (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID1079942Steatosis, proven histopathologically. Value is number of references indexed. [column 'STEAT' in source]
AID496828Antimicrobial activity against Leishmania donovani2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID653936Cytotoxicity against human HGF cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1916661Cytotoxicity against human K562 cells assessed as cell growth inhibition incubated for 24 hrs by MTT assay2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID539593Growth inhibition of human NCI60 cells by SBR assay2010Bioorganic & medicinal chemistry letters, Dec-01, Volume: 20, Issue:23
Discovery and selectivity-profiling of 4-benzylamino 1-aza-9-oxafluorene derivatives as lead structures for IGF-1R inhibitors.
AID393871Cytotoxicity against HGF cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID496823Antimicrobial activity against Trichomonas vaginalis2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1439560Cytotoxicity against mouse L1210 cells assessed as growth inhibition after 48 hrs by coulter counter method
AID384303Cytotoxicity against mouse L1210 cells after 48 hrs2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID1594189Cytotoxicity against human SW620 cells incubated for 4 hrs under aerobic condition followed by compound washout and measured after 5 days by SRB assay2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID92134Evaluation for cytotoxicity to human tumor cell lines.1998Journal of medicinal chemistry, Mar-26, Volume: 41, Issue:7
Cytotoxic activities of Mannich bases of chalcones and related compounds.
AID393861Cytotoxicity against human CEM cells after 72 hrs2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID295417Cytotoxicity against HSC2 cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID382490Cytotoxicity against human gingival fibroblast by MTT method2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID712166Cytotoxicity against MMR-deficient human A2780/CP70 cells after 5 days by MTT assay in presence of 10 uM MGMT inactivator Patrin22012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID98496Inhibitory effect on the biosyntheses of RNA in murine L1210 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1439566Cytotoxicity against human HPC after 48 hrs by MTT assay
AID225422Concentration of compound required for 50% cross linking of plasmid pBR322 DNA was investigated using an assay involving linear double stranded DNA2001Journal of medicinal chemistry, Mar-01, Volume: 44, Issue:5
Design, synthesis, and evaluation of a novel pyrrolobenzodiazepine DNA-interactive agent with highly efficient cross-linking ability and potent cytotoxicity.
AID466506Cytotoxicity against human CEM cells2010Bioorganic & medicinal chemistry letters, Mar-01, Volume: 20, Issue:5
Derivatives of aryl amines containing the cytotoxic 1,4-dioxo-2-butenyl pharmacophore.
AID1439567Cytotoxicity against HPLF after 48 hrs by MTT assay
AID224731Concentration required to inhibit 50% of cell growth on murine P388 D1 cells.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID384992Cytotoxicity against mouse L1210 cells after 72 hrs2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID521220Inhibition of neurosphere proliferation of mouse neural precursor cells by MTT assay2007Nature chemical biology, May, Volume: 3, Issue:5
Chemical genetics reveals a complex functional ground state of neural stem cells.
AID209939Inhibitory effect on the biosyntheses of RNA using human Jurkat T cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1474166Liver toxicity in human assessed as induction of drug-induced liver injury by measuring severity class index2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID88789Concentration required to inhibit 50% growth of Human CEM T-lymphocytes cells1998Journal of medicinal chemistry, Mar-26, Volume: 41, Issue:7
Cytotoxic activities of Mannich bases of chalcones and related compounds.
AID1277012Cytotoxicity against HGF assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID540210Clearance in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID55444The compound was tested for cytotoxicity against D283 MR (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID629592Cytotoxicity against human INA-6 cells assessed as viable fractions using annexin V-FITC/propidium iodide staining by flow cytometry2011European journal of medicinal chemistry, Dec, Volume: 46, Issue:12
Anti-tumoral activities of dioncoquinones B and C and related naphthoquinones gained from total synthesis or isolation from plants.
AID364034Cytotoxicity against human KYSE520 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID654083Cytotoxicity against human RPMI8226 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1073152Induction of apoptosis in human JJN-3 cells assessed as early apoptotic cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 13.3%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID1916654Cytotoxicity against human MDA-MB-231 cells assessed as reduction in cell viability at 15 uM incubated for 24 hrs by MTT assay relative to control2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID153691Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12 for mortality range at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID393862Cytotoxicity against mouse L1210 cells after 48 hrs2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID283821Cytotoxicity against human HCT116 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID469472Cytotoxicity against human Scov3 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Mar, Volume: 45, Issue:3
Synthesis, characterization and structure-activity relationship of novel N-phosphorylated E,E-3,5-bis(thienylidene)piperid-4-ones.
AID1777556Antiproliferative activity against human MCF7 cells assessed as inhibition of cell proliferation measured after 72 hrs by CCK-8 assay2021Bioorganic & medicinal chemistry, 09-01, Volume: 45Design and synthesis of β-carboline derivatives with nitrogen mustard moieties against breast cancer.
AID615398Cytostatic activity against mouse L1210 cells after 2 days by coulter counter assay2011European journal of medicinal chemistry, Sep, Volume: 46, Issue:9
Design, synthesis and bioevaluation of novel candidate selective estrogen receptor modulators.
AID588216FDA HLAED, serum glutamic oxaloacetic transaminase (SGOT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID103748In vitro cytotoxicity activity against MCF-72002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID477295Octanol-water partition coefficient, log P of the compound2010European journal of medicinal chemistry, Apr, Volume: 45, Issue:4
QSPR modeling of octanol/water partition coefficient of antineoplastic agents by balance of correlations.
AID1136466Immunosuppressive activity in mouse splenic lymphocytes isolated from 25 mg/kg, ip on days 2,3 and 5 treated CF1 mouse assessed as reduction in sheep red blood cell antigen-induced plaque-forming response (Rvb = 100%)1979Journal of medicinal chemistry, Nov, Volume: 22, Issue:11
Antitumor and antiinflammatory agents: N-benzoyl-protected cyanomethyl esters of amino acids.
AID588212Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in rodents2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID101740In vitro cytotoxicity activity against M4 Beu, by colony forming assay2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID408359Induction of DNA interstrand cross linking in 20 Gy irradiated human H1299 cells at 200 uM after 1 hr by comet assay2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
Synthesis and biological activity of stable and potent antitumor agents, aniline nitrogen mustards linked to 9-anilinoacridines via a urea linkage.
AID24701Degradation under physiological pH 9.0; Instantaneous2000Bioorganic & medicinal chemistry letters, Nov-06, Volume: 10, Issue:21
Stability of bioreductive drug delivery systems containing melphalan is influenced by conformational constraint and electronic properties of substituents.
AID496830Antimicrobial activity against Leishmania major2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID364029Cytotoxicity against human RT4 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID594322Antiproliferative activity against human Molt4/C8 cells assessed as inhibition of cell growth after 48 hrs by ZF-Coulter Counting2011Bioorganic & medicinal chemistry letters, May-01, Volume: 21, Issue:9
Synthesis of novel antimitotic agents based on 2-amino-3-aroyl-5-(hetero)arylethynyl thiophene derivatives.
AID1292276Cytotoxicity against human HSC2 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID462975Binding affinity to DNA in human H1299 cells assessed as formation of DNA cross-links at 200 uM followed by irradiation by modified single cell gel electrophoresis comet assay2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Potent DNA-directed alkylating agents: Synthesis and biological activity of phenyl N-mustard-quinoline conjugates having a urea or hydrazinecarboxamide linker.
AID1594185Cytotoxicity against human MDA-MB-468 cells incubated for 4 hrs under hypoxic condition followed by compound washout and measured after 5 days by SRB assay2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID200783The compound was tested for cytotoxicity against SF-126 (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID444324Cytotoxicity against human CEM cells2009Bioorganic & medicinal chemistry letters, Nov-15, Volume: 19, Issue:22
Design, synthesis and bioevaluation of novel maleamic amino acid ester conjugates of 3,5-bisarylmethylene-4-piperidones as cytostatic agents.
AID385001Cytotoxicity against human HSC4 cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID588215FDA HLAED, alkaline phosphatase increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID398090Growth inhibition of human Molt4/C8 cells after 3 days2009Bioorganic & medicinal chemistry, Jun-01, Volume: 17, Issue:11
2-(3-Aryl-2-propenoyl)-3-methylquinoxaline-1,4-dioxides: a novel cluster of tumor-specific cytotoxins which reverse multidrug resistance.
AID98497Inhibitory effect on the biosyntheses of protein in murine L1210 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID98495Inhibitory effect on the biosyntheses of DNA in murine L1210 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1277025Selectivity index, ratio of CC50 for HPC to CC50 for human HL60 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID625285Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatic necrosis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID354538Cytotoxicity against rat C6 cells at 50 ug/mL to 2.5 mg/mL after 3 days treated 4 hrs before db-cAMP challenge by MTT assay1996Journal of natural products, Dec, Volume: 59, Issue:12
Cell-based screen for identification of inhibitors of tubulin polymerization.
AID153688Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12 for median day of death at an optimal dose of 1 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID596215Cytotoxicity against human HSC3 cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID101741Comparative cellular uptake study performed on M4 Beu (melanoma cell line)2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID1132955Antitumor activity against mouse P388 cells allografted in DBA/2 mouse assessed as increase in survival rate at 25 mg/kg/day, ip administered for 2 weeks relative to control1978Journal of medicinal chemistry, Aug, Volume: 21, Issue:8
Antitumor agents 32. Synthesis and antitumor activity of cyclopentenone derivatives related to helenalin.
AID150684Inhibition of proliferation of murine P388 cells1998Journal of medicinal chemistry, Mar-26, Volume: 41, Issue:7
Cytotoxic activities of Mannich bases of chalcones and related compounds.
AID588219FDA HLAED, gamma-glutamyl transferase (GGT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID1148042Antitumor activity against mouse EAC allografted in CF1 mouse assessed as inhibition of tumor growth at 33 mg/kg/day measured on day 71977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 1. N-Protected vinyl, 1,2-dihaloethyl, and cyanomethyl esters of phenylalanine.
AID1148050Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat assessed as animal survival days at 2.5 mg/kg/day, ip1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID567542Cytotoxicity against human renal cancer cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID744852Cytotoxicity against human HeLa cells after 4 days by Coulter counter analysis2013European journal of medicinal chemistry, May, Volume: 63Anticancer activity of novel hybrid molecules containing 5-benzylidene thiazolidine-2,4-dione.
AID712161Cytotoxicity against human HCT116 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1439593Toxicity in ip dosed mouse
AID1275534Cytotoxicity against human HSC2 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry letters, Feb-15, Volume: 26, Issue:4
1-[3-(2-Hydroxyethylsulfanyl)propanoyl]-3,5-bis(benzylidene)-4-piperidones: A novel cluster of P-glycoprotein dependent multidrug resistance modulators.
AID524792Antiplasmodial activity against Plasmodium falciparum D10 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID1178003Antiproliferative activity against human CEM cells after 72 hrs by coulter counting analysis2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID221020Effect on DNA biosyntheses in leukemia L1210 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID300178Cytotoxicity against human colon cancer cells2007Bioorganic & medicinal chemistry, Sep-01, Volume: 15, Issue:17
E,E,E-1-(4-Arylamino-4-oxo-2-butenoyl)-3,5-bis(arylidene)-4-piperidones: a topographical study of some novel potent cytotoxins.
AID666611Cytotoxicity against human HeLa cells after 72 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and antitumor activity of formononetin nitrogen mustard derivatives.
AID99171Tested in vivo for optimal non-toxic dose against L1210/LPAM cell line in CDF1 mice1989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID99877In vitro cytotoxicity against L1210 cells at a dose 50 ug/mL1989Journal of medicinal chemistry, May, Volume: 32, Issue:5
Synthesis of 5-[1-hydroxy(or methoxy)-2-bromo(or chloro)ethyl]-2'-deoxyuridines and related halohydrin analogues with antiviral and cytotoxic activity.
AID153372Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for tumor free survivors at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID283823Cytotoxicity against human KM12 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID1292273Cytotoxicity against human Molt4/C8 cells after 72 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID382487Cytotoxicity against human HSC2 by MTT method2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID596211Anticancer activity against human K562 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID654077Cytotoxicity against human HCT15 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID588213Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in non-rodents2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID596201Anticancer activity against human CCRF-CEM cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1178005Antiproliferative activity against mouse L1210 cells after 72 hrs by coulter counting analysis2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID425652Total body clearance in human2009Journal of medicinal chemistry, Aug-13, Volume: 52, Issue:15
Physicochemical determinants of human renal clearance.
AID1916660Lipophilicity, logP of the compound in n-octane/water by spectrophotometry2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID1185725Cytotoxicity against human HeLa cells after 72 hrs by Coulter counting method2014European journal of medicinal chemistry, Sep-12, Volume: 842-(4-Chlorobenzyl)-6-arylimidazo[2,1-b][1,3,4]thiadiazoles: synthesis, cytotoxic activity and mechanism of action.
AID1584825Growth inhibition of human MDA-MB-468 cells after 48 hrs by fluorescence polarisation assay2018Journal of medicinal chemistry, 10-25, Volume: 61, Issue:20
Discovery and Optimization of Novel Hydrogen Peroxide Activated Aromatic Nitrogen Mustard Derivatives as Highly Potent Anticancer Agents.
AID444322Cytotoxicity against mouse L1210 cells2009Bioorganic & medicinal chemistry letters, Nov-15, Volume: 19, Issue:22
Design, synthesis and bioevaluation of novel maleamic amino acid ester conjugates of 3,5-bisarylmethylene-4-piperidones as cytostatic agents.
AID153695Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12) for tumor free survivors at an optimal dose of 1 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID408376Cytotoxicity against human Molt4/C8 cells after 72 hrs2008Bioorganic & medicinal chemistry, Jun-01, Volume: 16, Issue:11
E,E-2-Benzylidene-6-(nitrobenzylidene)cyclohexanones: syntheses, cytotoxicity and an examination of some of their electronic, steric, and hydrophobic properties.
AID1185723Cytotoxicity against mouse L1210 cells after 48 hrs by Coulter counting method2014European journal of medicinal chemistry, Sep-12, Volume: 842-(4-Chlorobenzyl)-6-arylimidazo[2,1-b][1,3,4]thiadiazoles: synthesis, cytotoxic activity and mechanism of action.
AID481760Antiproliferative activity against mouse FM3A cells after 48 hrs2010Bioorganic & medicinal chemistry letters, May-01, Volume: 20, Issue:9
Symmetrical alpha-bromoacryloylamido diaryldienone derivatives as a novel series of antiproliferative agents. Design, synthesis and biological evaluation.
AID384306Cytotoxicity against human HSC4 cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID1277002Antiproliferative activity against human Molt4/C8 cells assessed as cell survival after 72 hrs by coulter counter analysis2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID654074Cytotoxicity against human COLO205 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID458803Cytotoxicity against human HPLF cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID588214FDA HLAED, liver enzyme composite activity2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID396987Cytotoxicity against human Scov3 cells after 72 hrs by MTT assay2009European journal of medicinal chemistry, May, Volume: 44, Issue:5
Design, cytotoxic and fluorescent properties of novel N-phosphorylalkyl substituted E,E-3,5-bis(arylidene)piperid-4-ones.
AID1916657Cytotoxicity against human MCF7 cells assessed as cell growth inhibition incubated for 48 hrs by AlamarBlue assay2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID1277013Cytotoxicity against HPC assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1473739Inhibition of human MRP2 overexpressed in Sf9 cell membrane vesicles assessed as uptake of [3H]-estradiol-17beta-D-glucuronide in presence of ATP and GSH measured after 20 mins by membrane vesicle transport assay2013Toxicological sciences : an official journal of the Society of Toxicology, Nov, Volume: 136, Issue:1
A multifactorial approach to hepatobiliary transporter assessment enables improved therapeutic compound development.
AID654079Cytotoxicity against human KM12 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1113479Cytotoxicity against Mus musculus (mouse) L1210 cells assessed as growth inhibition after 48 hr by coulter counter analysis2013Medicinal chemistry research : an international journal for rapid communications on design and mechanisms of action of biologically active agents, , Volume: 22, Issue:4
Synthesis and biological evaluation of 2-(5-substituted-1-((diethylamino)methyl)-2-oxoindolin-3-ylidene)-
AID1079945Animal toxicity known. [column 'TOXIC' in source]
AID153834Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for % increase in life span at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID79237In vitro cytotoxicity in chondrosarcoma cell line (H-EMC-SS) by Alamar blue assay2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID1178013Antileukemic activity against human K562 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1079936Choleostatic liver toxicity, either proven histopathologically or where the ratio of maximal ALT or AST activity above normal to that of Alkaline Phosphatase is < 2 (see ACUTE). Value is number of references indexed. [column 'CHOLE' in source]
AID295422Cytotoxicity against HPLF cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID524796Antiplasmodial activity against Plasmodium falciparum W2 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID1178010Cytotoxicity against human KM12 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID588220Literature-mined public compounds from Kruhlak et al phospholipidosis modelling dataset2008Toxicology mechanisms and methods, , Volume: 18, Issue:2-3
Development of a phospholipidosis database and predictive quantitative structure-activity relationship (QSAR) models.
AID153698Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12) for total dose at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID209940Inhibitory effect on the biosyntheses of protein using human Jurkat T cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1439570Selectivity index, ratio of CC50 for HPLF to CC50 for human HL60 cells
AID596200Anticancer activity against human SW620 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1241933Antiproliferative activity against mouse FM3A cells assessed as inhibition of cell proliferation incubated for 2 days by Coulter counter based assay2015European journal of medicinal chemistry, Aug-28, Volume: 101Design, synthesis and antiproliferative activity of novel heterobivalent hybrids based on imidazo[2,1-b][1,3,4]thiadiazole and imidazo[2,1-b][1,3]thiazole scaffolds.
AID483885Binding affinity to LAT1 in rat brain2010Bioorganic & medicinal chemistry letters, Jun-15, Volume: 20, Issue:12
Regiospecific and conformationally restrained analogs of melphalan and DL-2-NAM-7 and their affinities for the large neutral amino acid transporter (system LAT1) of the blood-brain barrier.
AID1079946Presence of at least one case with successful reintroduction. [column 'REINT' in source]
AID496831Antimicrobial activity against Cryptosporidium parvum2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1449628Inhibition of human BSEP expressed in baculovirus transfected fall armyworm Sf21 cell membranes vesicles assessed as reduction in ATP-dependent [3H]-taurocholate transport into vesicles incubated for 5 mins by Topcount based rapid filtration method2012Drug metabolism and disposition: the biological fate of chemicals, Dec, Volume: 40, Issue:12
Mitigating the inhibition of human bile salt export pump by drugs: opportunities provided by physicochemical property modulation, in silico modeling, and structural modification.
AID98577Cytotoxicity was determined after 48 hr of continuous exposure against L1210 murine leukemia cells2004Journal of medicinal chemistry, May-06, Volume: 47, Issue:10
Cytotoxic alpha-halogenoacrylic derivatives of distamycin A and congeners.
AID653935Selectivity index, ratio of CC50 for human normal cells to CC50 for human HL60 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1277022Selectivity index, ratio of CC50 for HPC to CC50 for human HSC4 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1060756Antitumor activity against mouse B16F10 cells xenografted in C57BL/6 mouse assessed as reduction of tumor size at 100 ug administered as intratumorally for 3 days2014European journal of medicinal chemistry, Jan, Volume: 71Synthesis and analysis of activity of a potential anti-melanoma prodrug with a hydrazine linker.
AID594324Antiproliferative activity against human HeLa cells assessed as inhibition of cell growth after 48 hrs by ZF-Coulter Counting2011Bioorganic & medicinal chemistry letters, May-01, Volume: 21, Issue:9
Synthesis of novel antimitotic agents based on 2-amino-3-aroyl-5-(hetero)arylethynyl thiophene derivatives.
AID199113Antitumor activity against S-180 cells after i.p. administration in mice up to 7 days at a dose of 16 umol/kg2001Bioorganic & medicinal chemistry letters, Jun-04, Volume: 11, Issue:11
Esters of 2-(1-hydroxyalkyl)-1,4-dihydroxy-9,10-anthraquinones with melphalan as multifunctional anticancer agents.
AID393870Cytotoxicity against human HL60 cells after 24 hrs by MTT assay in presence of 10% fetal bovine serum2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID744854Cytotoxicity against mouse FM3A cells after 2 days by Coulter counter analysis2013European journal of medicinal chemistry, May, Volume: 63Anticancer activity of novel hybrid molecules containing 5-benzylidene thiazolidine-2,4-dione.
AID1916648Cytotoxicity against human U2OS cells assessed as cell growth inhibition2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID409954Inhibition of mouse brain MAOA2008Journal of medicinal chemistry, Nov-13, Volume: 51, Issue:21
Quantitative structure-activity relationship and complex network approach to monoamine oxidase A and B inhibitors.
AID1178008Cytotoxicity against human HCC2998 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1292279Cytotoxicity against human HGF cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID354543Cytotoxicity against rat C6 cells assessed as cell release at 50 ug/mL to 2.5 mg/mL after 5 hrs by MTT assay in absence of db-cAMP1996Journal of natural products, Dec, Volume: 59, Issue:12
Cell-based screen for identification of inhibitors of tubulin polymerization.
AID588218FDA HLAED, lactate dehydrogenase (LDH) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID594321Antiproliferative activity against mouse FM3A/0 cells assessed as inhibition of cell growth after 48 hrs by ZF-Coulter Counting2011Bioorganic & medicinal chemistry letters, May-01, Volume: 21, Issue:9
Synthesis of novel antimitotic agents based on 2-amino-3-aroyl-5-(hetero)arylethynyl thiophene derivatives.
AID99422Mean graph mid point was measured on leukemic cells.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID385002Cytotoxicity against human RPMI8226 cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID1439575Selectivity index, ratio of CC50 for human HPC to CC50 for human HSC3 cells
AID1769487Aqueous solubility of the compound
AID526437Selectivity index, ratio of CC50 for normal cells to CC50 for human HL60 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID1073136Induction of apoptosis in human JJN-3 cells assessed as late apoptotic cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 9.59%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID55575The compound was tested for cytotoxicity against D341 (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID596310Toxicity in mouse harboring P-388 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID526439Selectivity index, ratio of CC50 for normal cells to CC50 for human HSC2 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID1178002Antiproliferative activity against human Molt4/C8 cells after 72 hrs by coulter counting analysis2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1777558Cytotoxicity against human MCF-10A cells assessed as inhibition of cell proliferation measured after 72 hrs by CCK-8 assay2021Bioorganic & medicinal chemistry, 09-01, Volume: 45Design and synthesis of β-carboline derivatives with nitrogen mustard moieties against breast cancer.
AID1178012Antileukemic activity against human HL-60(TB) cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID55572The compound was tested for cytotoxicity against D341 MR (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID666612Cytotoxicity against human SGC7901 cells after 72 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and antitumor activity of formononetin nitrogen mustard derivatives.
AID1185081Antiproliferative activity against human Bel7402 cells assessed as growth inhibition after 72 hrs by MTT assay2014ACS medicinal chemistry letters, Jul-10, Volume: 5, Issue:7
Novel hybrids of natural oridonin-bearing nitrogen mustards as potential anticancer drug candidates.
AID384995Cytotoxicity against human SR cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID588217FDA HLAED, serum glutamic pyruvic transaminase (SGPT) increase2004Current drug discovery technologies, Dec, Volume: 1, Issue:4
Assessment of the health effects of chemicals in humans: II. Construction of an adverse effects database for QSAR modeling.
AID462400Toxicity in mouse2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Cytotoxic 2-benzylidene-6-(nitrobenzylidene)cyclohexanones which display substantially greater toxicity for neoplasms than non-malignant cells.
AID384301Cytotoxicity against human Molt 4/C8 cells after 72 hrs2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID625279Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for bilirubinemia2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1185724Cytotoxicity against human CEM cells after 72 hrs by Coulter counting method2014European journal of medicinal chemistry, Sep-12, Volume: 842-(4-Chlorobenzyl)-6-arylimidazo[2,1-b][1,3,4]thiadiazoles: synthesis, cytotoxic activity and mechanism of action.
AID221022Effect on proliferation of leukemia L1210 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID364038Cytotoxicity against human LCLC-103H cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID712170Induction of Escherichia coli pBR322 DNA alkylation assessed as formation of covalent DNA adducts after 2 hrs by Taq polymerase DNA stop assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1292284Selectivity index, ratio of CC50 for human HPLF cells to CC50 for human HSC2 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1079947Comments (NB not yet translated). [column 'COMMENTAIRES' in source]
AID24699Degradation under physiological pH 7.4 in the presence of glutathione2000Bioorganic & medicinal chemistry letters, Nov-06, Volume: 10, Issue:21
Stability of bioreductive drug delivery systems containing melphalan is influenced by conformational constraint and electronic properties of substituents.
AID311524Oral bioavailability in human2007Bioorganic & medicinal chemistry, Dec-15, Volume: 15, Issue:24
Hologram QSAR model for the prediction of human oral bioavailability.
AID138602Percentage inhibition as antitumor activity in carcinoma CF1 male mice.1980Journal of medicinal chemistry, Mar, Volume: 23, Issue:3
Antitumor agents: diazomethyl ketone and chloromethyl ketone analogues prepared from N-tosyl amino acids.
AID496817Antimicrobial activity against Trypanosoma cruzi2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1315404Cytotoxicity against human DU145 cells after 72 hrs by MTT assay2016European journal of medicinal chemistry, Aug-25, Volume: 119Design and synthesis of novel pyrazolo[1,5-a]pyrimidine derivatives bearing nitrogen mustard moiety and evaluation of their antitumor activity in vitro and in vivo.
AID1241932Antiproliferative activity against mouse L1210 cells assessed as inhibition of cell proliferation incubated for 2 days by Coulter counter based assay2015European journal of medicinal chemistry, Aug-28, Volume: 101Design, synthesis and antiproliferative activity of novel heterobivalent hybrids based on imidazo[2,1-b][1,3,4]thiadiazole and imidazo[2,1-b][1,3]thiazole scaffolds.
AID596197Anticancer activity against human HCT15 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1241935Antiproliferative activity against human HeLa cells assessed as inhibition of cell proliferation incubated for 4 days by Coulter counter based assay2015European journal of medicinal chemistry, Aug-28, Volume: 101Design, synthesis and antiproliferative activity of novel heterobivalent hybrids based on imidazo[2,1-b][1,3,4]thiadiazole and imidazo[2,1-b][1,3]thiazole scaffolds.
AID625290Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver fatty2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID540213Half life in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID1439558Cytotoxicity against human Molt4/C8 cells assessed as growth inhibition after 72 hrs by coulter counter method
AID1439561Cytotoxicity against human HL60 cells after 48 hrs by MTT assay
AID1113478Cytotoxicity against Homo sapiens (human) CEM cells assessed as growth inhibition after 72 hr by coulter counter analysis2013Medicinal chemistry research : an international journal for rapid communications on design and mechanisms of action of biologically active agents, , Volume: 22, Issue:4
Synthesis and biological evaluation of 2-(5-substituted-1-((diethylamino)methyl)-2-oxoindolin-3-ylidene)-
AID588211Literature-mined compound from Fourches et al multi-species drug-induced liver injury (DILI) dataset, effect in humans2010Chemical research in toxicology, Jan, Volume: 23, Issue:1
Cheminformatics analysis of assertions mined from literature that describe drug-induced liver injury in different species.
AID91673Mean graph mid point was measured on all cell lines.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID382488Cytotoxicity against human HSC4 cells by MTT method2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID1277011Cytotoxicity against human HL60 cells assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID567536Cytotoxicity against human leukemia cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID364036Cytotoxicity against human DAN-G cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1292283Selectivity index, ratio of CC50 for human HGF cells to CC50 for human HSC4 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1148046Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat assessed as host survival days at 2.5 mg/kg, ip qd1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 1. N-Protected vinyl, 1,2-dihaloethyl, and cyanomethyl esters of phenylalanine.
AID153966Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for mortality range at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1079949Proposed mechanism(s) of liver damage. [column 'MEC' in source]
AID150665Inhibitory activity was tested against P388 cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID98045In vitro cytotoxicity was determined against L1210 leukemia cells from mouse1990Journal of medicinal chemistry, Feb, Volume: 33, Issue:2
Synthesis and antiviral and cytotoxic activity of iodohydrin and iodomethoxy derivatives of 5-vinyl-2'-deoxyuridines, 2'-fluoro-2'-deoxyuridine, and uridine.
AID744855Cytotoxicity against mouse L1210 cells after 2 days by Coulter counter analysis2013European journal of medicinal chemistry, May, Volume: 63Anticancer activity of novel hybrid molecules containing 5-benzylidene thiazolidine-2,4-dione.
AID653928Cytotoxicity against human HSC2 cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID97133Ratio between IC50 values on L1210 and L1210/L-PAM cells2004Journal of medicinal chemistry, May-06, Volume: 47, Issue:10
Cytotoxic alpha-halogenoacrylic derivatives of distamycin A and congeners.
AID1439563Cytotoxicity against human HSC3 cells after 48 hrs by MTT assay
AID228488Glutathione-s-transferase activity of the compound, expressed as percentage of control.2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID1148056Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat assessed as tumor growth inhibition at 2.5 mg/kg/day, ip1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID23271Partition coefficient (logD7.4)1990Journal of medicinal chemistry, Jul, Volume: 33, Issue:7
Structure-activity relationships of antineoplastic agents in multidrug resistance.
AID230140Tested in vitro for the resistance index ratio (ID50) of L1210/LPAM to L1210 cell line1989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID384997Cytotoxicity against human HPC cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID46584Inhibitory activity was tested against CEM cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID1292277Cytotoxicity against human HSC3 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID364033Cytotoxicity against human KYSE510 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1247222Antiproliferative activity against human HepG2 cells incubated for 48 hrs by MTT assay2015Bioorganic & medicinal chemistry letters, Oct-01, Volume: 25, Issue:19
Synthesis, structure-activity relationship and biological evaluation of novel nitrogen mustard sophoridinic acid derivatives as potential anticancer agents.
AID153682Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12 for change in body weight at an optimal dose of 1 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1277018Selectivity index, ratio of CC50 for HGF to CC50 for human HSC3 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID382492Cytotoxicity against human periodontal ligament fibroblast by MTT method2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID166691The compound was tested for cytotoxicity against RG-2 (Rat) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID384305Cytotoxicity against human HSC3 cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID625292Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) combined score2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID152701Cytotoxicity against murine P388 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID92264Tested against human tumor cell lines, mean graph mid point values(MG MID) are determined.1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID596207Anticancer activity against human Molt4/C8 cells after 72 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID425653Renal clearance in human2009Journal of medicinal chemistry, Aug-13, Volume: 52, Issue:15
Physicochemical determinants of human renal clearance.
AID596295Cytotoxicity against human HPLF cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID458806Cytotoxicity against human HSC4 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID153250Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for mortality range at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID45824Inhibitory concentration against CEM T-lymphocytes2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID654082Cytotoxicity against human K562 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1474167Liver toxicity in human assessed as induction of drug-induced liver injury by measuring verified drug-induced liver injury concern status2016Drug discovery today, Apr, Volume: 21, Issue:4
DILIrank: the largest reference drug list ranked by the risk for developing drug-induced liver injury in humans.
AID1277009Cytotoxicity against human HSC3 cells assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1277016Selectivity index, ratio of CC50 for HPC to CC50 for human HSC2 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID653932Cytotoxicity against human HSC4 cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID227705The apoptotic index [(number of human jurkat cells with apoptotic nuclei/number of cells counted) 100]2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID615399Cytostatic activity against human Molt4/C8 cells after 3 days by coulter counter assay2011European journal of medicinal chemistry, Sep, Volume: 46, Issue:9
Design, synthesis and bioevaluation of novel candidate selective estrogen receptor modulators.
AID1277024Selectivity index, ratio of CC50 for HGF to CC50 for human HL60 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID445446Oral bioavailability in human2010Journal of medicinal chemistry, Jan-14, Volume: 53, Issue:1
The permeation of amphoteric drugs through artificial membranes--an in combo absorption model based on paracellular and transmembrane permeability.
AID654078Cytotoxicity against human HT-29 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID496827Antimicrobial activity against Leishmania amazonensis2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1473741Inhibition of human MRP4 overexpressed in Sf9 cell membrane vesicles assessed as uptake of [3H]-estradiol-17beta-D-glucuronide in presence of ATP and GSH measured after 20 mins by membrane vesicle transport assay2013Toxicological sciences : an official journal of the Society of Toxicology, Nov, Volume: 136, Issue:1
A multifactorial approach to hepatobiliary transporter assessment enables improved therapeutic compound development.
AID540212Mean residence time in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID125499Inhibitory activity was tested against Molt4/C8 cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID408377Cytotoxicity against mouse L1210 cells after 48 hrs2008Bioorganic & medicinal chemistry, Jun-01, Volume: 16, Issue:11
E,E-2-Benzylidene-6-(nitrobenzylidene)cyclohexanones: syntheses, cytotoxicity and an examination of some of their electronic, steric, and hydrophobic properties.
AID1292274Cytotoxicity against human CEM cells after 72 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1916647Cytotoxicity against mouse MC3T3-E1 cells assessed as cell growth inhibition2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID594320Antiproliferative activity against mouse L1210 cells assessed as inhibition of cell growth after 48 hrs by ZF-Coulter Counting2011Bioorganic & medicinal chemistry letters, May-01, Volume: 21, Issue:9
Synthesis of novel antimitotic agents based on 2-amino-3-aroyl-5-(hetero)arylethynyl thiophene derivatives.
AID483884Inhibition of LAT1-mediated L-[14C]leucine uptake in Sprague-Dawley rat brain by duel-labeled liquid scintillation counting2010Bioorganic & medicinal chemistry letters, Jun-15, Volume: 20, Issue:12
Regiospecific and conformationally restrained analogs of melphalan and DL-2-NAM-7 and their affinities for the large neutral amino acid transporter (system LAT1) of the blood-brain barrier.
AID567538Cytotoxicity against human colon cancer cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID383748Cytotoxicity against human EJ60 cells after 72 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Synthesis, computational study and cytotoxic activity of new 4-hydroxycoumarin derivatives.
AID1277010Cytotoxicity against human HSC4 cells assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID625284Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatic failure2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID153091Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for tumor free survivors at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID98125Median survival time of treated to the control was determined in L1210 cell line1989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID1439578Selectivity index, ratio of CC50 for human HPC to CC50 for human HSC4 cells
AID458805Cytotoxicity against human HSC2 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID364035Cytotoxicity against human YAPC cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID445445Permeability at pH 6.5 by PAMPA method2010Journal of medicinal chemistry, Jan-14, Volume: 53, Issue:1
The permeation of amphoteric drugs through artificial membranes--an in combo absorption model based on paracellular and transmembrane permeability.
AID235488Selectivity index is the ratio of MG MID value for all cell lines to that of colon cancer cells2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID384307Cytotoxicity against human HL60 cells after 24 hrs by trypan blue exclusion assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID481762Antiproliferative activity against human CEM cells after 48 hrs2010Bioorganic & medicinal chemistry letters, May-01, Volume: 20, Issue:9
Symmetrical alpha-bromoacryloylamido diaryldienone derivatives as a novel series of antiproliferative agents. Design, synthesis and biological evaluation.
AID496825Antimicrobial activity against Leishmania mexicana2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1079934Highest frequency of acute liver toxicity observed during clinical trials, expressed as a percentage. [column '% AIGUE' in source]
AID567541Cytotoxicity against human ovarian cancer cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID1483959Induction of apoptosis in human PBMC after 3 days by V-FITC/ propidium iodide staining-based flow cytometric analysis2017Journal of natural products, 02-24, Volume: 80, Issue:2
Dioncophyllines C
AID625291Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver function tests abnormal2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID1178011Cytotoxicity against human SW620 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1178009Cytotoxicity against human HCT15 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID458802Cytotoxicity against human HPC cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID384310Cytotoxicity against human HPLF cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID153082Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for median day of death at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1079935Cytolytic liver toxicity, either proven histopathologically or where the ratio of maximal ALT or AST activity above normal to that of Alkaline Phosphatase is > 5 (see ACUTE). Value is number of references indexed. [column 'CYTOL' in source]
AID1073142Induction of apoptosis in human XG6 cells assessed as viable cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 83.3%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID295421Cytotoxicity against HGF cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID1277015Selectivity index, ratio of CC50 for HGF to CC50 for human HSC2 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID596297Selectivity index, ratio of average CC50 for normal human cell line to CC50 for human HSC4 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1439569Selectivity index, ratio of CC50 for human HPC to CC50 for human HL60 cells
AID526436Cytotoxicity against human HL60 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID596309Toxicity in mouse harboring L1210 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1473738Inhibition of human BSEP overexpressed in Sf9 cell membrane vesicles assessed as uptake of [3H]-taurocholate in presence of ATP measured after 15 to 20 mins by membrane vesicle transport assay2013Toxicological sciences : an official journal of the Society of Toxicology, Nov, Volume: 136, Issue:1
A multifactorial approach to hepatobiliary transporter assessment enables improved therapeutic compound development.
AID596210Anticancer activity against human HL60 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID153226Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for total dose at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID526446Cytotoxicity against human HPLF cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID221820Inhibitory concentration was evaluated on human Jurkat T-cells after their exposure for 48 hours2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID1079931Moderate liver toxicity, defined via clinical-chemistry results: ALT or AST serum activity 6 times the normal upper limit (N) or alkaline phosphatase serum activity of 1.7 N. Value is number of references indexed. [column 'BIOL' in source]
AID1315401Cytotoxicity against human HepG2 cells after 72 hrs by MTT assay2016European journal of medicinal chemistry, Aug-25, Volume: 119Design and synthesis of novel pyrazolo[1,5-a]pyrimidine derivatives bearing nitrogen mustard moiety and evaluation of their antitumor activity in vitro and in vivo.
AID1292285Selectivity index, ratio of CC50 for human HPLF cells to CC50 for human HSC3 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID153078Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for % increase in life span at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID384991Cytotoxicity against human Molt4/C8 cells after 72 hrs2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID393873Cytotoxicity against human HPLF cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID596196Anticancer activity against human HCC2998 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID227164Average potency of all four cell lines (P388 cell line, L1210 cell line, Molt 4/C8 cell line, CEM T-lymphocytes)2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID526441Selectivity index, ratio of CC50 for normal cells to CC50 for human HSC3 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID496818Antimicrobial activity against Trypanosoma brucei brucei2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID444323Cytotoxicity against human Molt4/C8 cells2009Bioorganic & medicinal chemistry letters, Nov-15, Volume: 19, Issue:22
Design, synthesis and bioevaluation of novel maleamic amino acid ester conjugates of 3,5-bisarylmethylene-4-piperidones as cytostatic agents.
AID44482The compound was tested for cytotoxicity against C6 (Rat) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID1439572Selectivity index, ratio of CC50 for human HPC to CC50 for human HSC2 cells
AID383747Cytotoxicity against human HL60 cells after 72 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Synthesis, computational study and cytotoxic activity of new 4-hydroxycoumarin derivatives.
AID95088IC80 concentrations of the compounds were used in determining the inhibition of protein synthesis using Jurkat T cells.1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID1136468Immunosuppressive activity in mouse splenic lymphocytes isolated from 0.1 mg, ip on days 2,3 and 5 treated CF1 mouse assessed as reduction in sheep red blood cell antigen-induced plaque-forming response (Rvb = 100%)1979Journal of medicinal chemistry, Nov, Volume: 22, Issue:11
Antitumor and antiinflammatory agents: N-benzoyl-protected cyanomethyl esters of amino acids.
AID496819Antimicrobial activity against Plasmodium falciparum2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID458804Cytotoxicity against human HL60 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID384308Cytotoxicity against human HDF cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID712163Cytotoxicity against human HT-29 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1916665Plasma protein binding in human assessed as unbound fraction2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID1185079Antiproliferative activity against human K562 cells assessed as growth inhibition after 72 hrs by MTT assay2014ACS medicinal chemistry letters, Jul-10, Volume: 5, Issue:7
Novel hybrids of natural oridonin-bearing nitrogen mustards as potential anticancer drug candidates.
AID654080Cytotoxicity against human SW620 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID384994Cytotoxicity against human K562 cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID539594Cytotoxicity against human NCI60 cells by SBR assay2010Bioorganic & medicinal chemistry letters, Dec-01, Volume: 20, Issue:23
Discovery and selectivity-profiling of 4-benzylamino 1-aza-9-oxafluorene derivatives as lead structures for IGF-1R inhibitors.
AID384998Cytotoxicity against human HPLF cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID1079932Highest frequency of moderate liver toxicity observed during clinical trials, expressed as a percentage. [column '% BIOL' in source]
AID1113477Cytotoxicity against Homo sapiens (human) HeLa cells assessed as growth inhibition after 96 hr by coulter counter analysis2013Medicinal chemistry research : an international journal for rapid communications on design and mechanisms of action of biologically active agents, , Volume: 22, Issue:4
Synthesis and biological evaluation of 2-(5-substituted-1-((diethylamino)methyl)-2-oxoindolin-3-ylidene)-
AID384312Neurotoxicity in ip dosed mouse by rotarod test2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID235489Selectivity index is the ratio of MG MID value for all cell lines to that of leukemic cells2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID567543Cytotoxicity against human prostate cancer cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID1079937Severe hepatitis, defined as possibly life-threatening liver failure or through clinical observations. Value is number of references indexed. [column 'MASS' in source]
AID1178007Cytotoxicity against human COLO205 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID224732Inhibitory activity against Murine P388 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID47330Lethal dose was determined for antitumor activity against murine leukemia cells (CFU-C)1987Journal of medicinal chemistry, Mar, Volume: 30, Issue:3
Selective cytotoxicity of a system L specific amino acid nitrogen mustard.
AID28892Partition coefficient (logD) (octanol/phosphate buffer)2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID540211Fraction unbound in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID596296Selectivity index, ratio of average CC50 for normal human cell line to CC50 for human HL-60 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1292281Selectivity index, ratio of CC50 for human HGF cells to CC50 for human HSC2 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1079943Malignant tumor, proven histopathologically. Value is number of references indexed. [column 'T.MAL' in source]
AID138285Anticarcinoma activity of Diazomethyl Ketone against Ehrlich ascites Carcinoma pharmacological screens were reported; 6/61980Journal of medicinal chemistry, Mar, Volume: 23, Issue:3
Antitumor agents: diazomethyl ketone and chloromethyl ketone analogues prepared from N-tosyl amino acids.
AID94731Cytotoxicity against L-1210 cells2001Bioorganic & medicinal chemistry letters, Jun-04, Volume: 11, Issue:11
Esters of 2-(1-hydroxyalkyl)-1,4-dihydroxy-9,10-anthraquinones with melphalan as multifunctional anticancer agents.
AID1148041Antitumor activity against mouse EAC allografted in CF1 mouse assessed as tumor volume at 33 mg/kg/day measured on day 71977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 1. N-Protected vinyl, 1,2-dihaloethyl, and cyanomethyl esters of phenylalanine.
AID396988Cytotoxicity against human PC3 cells after 72 hrs by MTT assay2009European journal of medicinal chemistry, May, Volume: 44, Issue:5
Design, cytotoxic and fluorescent properties of novel N-phosphorylalkyl substituted E,E-3,5-bis(arylidene)piperid-4-ones.
AID150512Cytotoxicity evaluated against P388 cells.1998Journal of medicinal chemistry, Oct-08, Volume: 41, Issue:21
4-(beta-Arylvinyl)-3-(beta-arylvinylketo)-1-ethyl-4-piperidinols and related compounds: a novel class of cytotoxic and anticancer agents.
AID526443Selectivity index, ratio of CC50 for normal cells to CC50 for human HSC4 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID384990Cytotoxicity against human CEM cells after 72 hrs2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID629593Cytotoxicity against human PBMC cells assessed as viable fractions using annexin V-FITC/propidium iodide staining by flow cytometry2011European journal of medicinal chemistry, Dec, Volume: 46, Issue:12
Anti-tumoral activities of dioncoquinones B and C and related naphthoquinones gained from total synthesis or isolation from plants.
AID393869Cytotoxicity against human HSC4 cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID1439562Cytotoxicity against human HSC2 cells after 48 hrs by MTT assay
AID654081Cytotoxicity against human HL-60(TB) cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1916649Cytotoxicity against human SAOS-2 cells assessed as cell growth inhibition2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID526445Cytotoxicity against human HPC cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID1439574Selectivity index, ratio of CC50 for HGF to CC50 for human HSC3 cells
AID247839Cytotoxicity against human leukemia cell line K5622004Journal of medicinal chemistry, Sep-09, Volume: 47, Issue:19
Design and synthesis of a nitrogen mustard derivative stabilized by apo-neocarzinostatin.
AID98567Cytotoxicity evaluated against L1210 cells.1998Journal of medicinal chemistry, Oct-08, Volume: 41, Issue:21
4-(beta-Arylvinyl)-3-(beta-arylvinylketo)-1-ethyl-4-piperidinols and related compounds: a novel class of cytotoxic and anticancer agents.
AID91666Cytotoxicity evaluated against human tumor.1998Journal of medicinal chemistry, Oct-08, Volume: 41, Issue:21
4-(beta-Arylvinyl)-3-(beta-arylvinylketo)-1-ethyl-4-piperidinols and related compounds: a novel class of cytotoxic and anticancer agents.
AID615284Cytostatic effect against human MCF7 cells at 10 uM after 72 hrs by crystal violet staining relative to uncreate control2011European journal of medicinal chemistry, May, Volume: 46, Issue:5
Bivalent bendamustine and melphalan derivatives as anticancer agents.
AID712167Cytotoxicity against human A2780 cells after 5 days by MTT assay in presence of 10 uM MGMT inactivator Patrin22012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID98173Cytotoxicity against murine L1210 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID283820Cytotoxicity against human COLO205 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID1664699Cytotoxicity against human MCF7 cells assessed as reduction in cell viability after 48 hrs by MTT assay2020Bioorganic & medicinal chemistry, 08-01, Volume: 28, Issue:15
Tetrazoles as anticancer agents: A review on synthetic strategies, mechanism of action and SAR studies.
AID524791Antiplasmodial activity against Plasmodium falciparum 7G8 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID221824Inhibition of RNA synthesis was evaluated on human Jurkat T-cells after 8 hours.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID94963Inhibitory concentration against Human Jurkat T cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID1178004Antiproliferative activity against human HeLa cells after 72 hrs by coulter counting analysis2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1769491Covalent binding affinity to salmon DNA measured after 30 mins by UV based spectroscopic method
AID95084Inhibitory concentration against Human Jurkat T cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID625287Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatomegaly2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID625283Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for elevated liver function tests2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID139117Volume of fluid collected 8 days after tumor transplantation in mice.1980Journal of medicinal chemistry, Mar, Volume: 23, Issue:3
Antitumor agents: diazomethyl ketone and chloromethyl ketone analogues prepared from N-tosyl amino acids.
AID153086Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for mortality range at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID24698Degradation under physiological pH 7.42000Bioorganic & medicinal chemistry letters, Nov-06, Volume: 10, Issue:21
Stability of bioreductive drug delivery systems containing melphalan is influenced by conformational constraint and electronic properties of substituents.
AID102210Tested in vitro for inhibition after 144 hr against human colon carcinoma LoVo/DX cell line resistant to Doxorubicin (DX)1994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID596195Anticancer activity against human HCT116 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID295419Cytotoxicity against HSC4 cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID99170Tested in vitro for the cytotoxicity as number of viable cells against L1210/LPAM cell line after 72 hr treatment at conc. of 10E-61989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID98915Inhibitory activity was tested against L1210 cells1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID526440Cytotoxicity against human HSC3 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID99059Tested in vitro for inhibition after 48 hr exposure against murine leukemia cell line L12101994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID248055Antiproliferative activity in MCF-7 human breast cancer cells2005Journal of medicinal chemistry, Jan-27, Volume: 48, Issue:2
Antitumor-active cobalt-alkyne complexes derived from acetylsalicylic acid: studies on the mode of drug action.
AID1073143Induction of apoptosis in human XG6 cells assessed as early apoptotic cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 8.62%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID1277004Antiproliferative activity against mouse L1210 cells assessed as cell survival after 48 hrs by coulter counter analysis2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID408375Cytotoxicity against human CEM cells after 72 hrs2008Bioorganic & medicinal chemistry, Jun-01, Volume: 16, Issue:11
E,E-2-Benzylidene-6-(nitrobenzylidene)cyclohexanones: syntheses, cytotoxicity and an examination of some of their electronic, steric, and hydrophobic properties.
AID712156Cytotoxicity against human U251 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1185082Antiproliferative activity against human MGC803 cells assessed as growth inhibition after 72 hrs by MTT assay2014ACS medicinal chemistry letters, Jul-10, Volume: 5, Issue:7
Novel hybrids of natural oridonin-bearing nitrogen mustards as potential anticancer drug candidates.
AID1277008Cytotoxicity against human HSC2 cells assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1473740Inhibition of human MRP3 overexpressed in Sf9 insect cell membrane vesicles assessed as uptake of [3H]-estradiol-17beta-D-glucuronide in presence of ATP and GSH measured after 10 mins by membrane vesicle transport assay2013Toxicological sciences : an official journal of the Society of Toxicology, Nov, Volume: 136, Issue:1
A multifactorial approach to hepatobiliary transporter assessment enables improved therapeutic compound development.
AID1439573Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC2 cells
AID153239Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for % increase in life span at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1079939Cirrhosis, proven histopathologically. Value is number of references indexed. [column 'CIRRH' in source]
AID221023Effect on protein biosyntheses in leukemia L1210 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID1439564Cytotoxicity against human HSC4 cells after 48 hrs by MTT assay
AID596199Anticancer activity against human KM12 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID86328Tested in vitro for the dose inhibiting cell proliferation by 50% against HeLa cell line after 24 hr treatment at conc. of 10E-61989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID1594190Cytotoxicity against human SW620 cells incubated for 4 hrs under hypoxic condition followed by compound washout and measured after 5 days by SRB assay2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID625281Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cholelithiasis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID153984Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for total dose at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID458801Cytotoxicity against human HGF cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID230383Resistance index as the ratio of IC50 against LoVo/Dx to that of LoVo1994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID1439565Cytotoxicity against HGF after 48 hrs by MTT assay
AID1132874Antitumor activity against mouse P388 cells allografted in DBA/2 mouse assessed as survival rate at 25 mg/kg relative to control1978Journal of medicinal chemistry, Jul, Volume: 21, Issue:7
Antitumor agents. 31. Helenalin sym-dimethylethylenediamine reaction products and related derivatives.
AID384304Cytotoxicity against human HSC2 cells after 24 hrs by MTT assay2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID98578Cytotoxicity was determined after 48 hr of continuous exposure against L1210 murine leukemia cells,(cells resistant to melphalan (L-PAM)2004Journal of medicinal chemistry, May-06, Volume: 47, Issue:10
Cytotoxic alpha-halogenoacrylic derivatives of distamycin A and congeners.
AID653933Selectivity index, ratio of CC50 for human normal cells to CC50 for human HSC4 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID97895Inhibitory activity against murine leukemia cells (L1210) was determined1987Journal of medicinal chemistry, Mar, Volume: 30, Issue:3
Selective cytotoxicity of a system L specific amino acid nitrogen mustard.
AID331134Growth inhibition of human HepG2 cells2008Bioorganic & medicinal chemistry letters, May-15, Volume: 18, Issue:10
Possible chemical mechanisms underlying the antitumor activity of S-deoxyleinamycin.
AID283825Cytotoxicity against human HCC2998 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID466507Cytotoxicity against mouse L1210 cells2010Bioorganic & medicinal chemistry letters, Mar-01, Volume: 20, Issue:5
Derivatives of aryl amines containing the cytotoxic 1,4-dioxo-2-butenyl pharmacophore.
AID393867Cytotoxicity against human HSC2 cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID153686Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(12) for % increase in life span at an optimal dose of 1 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID496824Antimicrobial activity against Toxoplasma gondii2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID393868Cytotoxicity against human HSC3 cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID283831Cytotoxicity against human NCI49 cell lines2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID384993Cytotoxicity against human CCRF-CEM cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID1777557Antiproliferative activity against human MDA-MB-231 cells assessed as inhibition of cell proliferation measured after 72 hrs by CCK-8 assay2021Bioorganic & medicinal chemistry, 09-01, Volume: 45Design and synthesis of β-carboline derivatives with nitrogen mustard moieties against breast cancer.
AID466505Cytotoxicity against human Molt4/C8 cells2010Bioorganic & medicinal chemistry letters, Mar-01, Volume: 20, Issue:5
Derivatives of aryl amines containing the cytotoxic 1,4-dioxo-2-butenyl pharmacophore.
AID354542Inhibition of tubulin polymerization in rat C6 cells at 50 ug/mL to 2.5 mg/mL after 4 hrs1996Journal of natural products, Dec, Volume: 59, Issue:12
Cell-based screen for identification of inhibitors of tubulin polymerization.
AID364032Cytotoxicity against human KYSE70 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1277003Antiproliferative activity against human CEM cells assessed as cell survival after 72 hrs by coulter counter analysis2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID712164Cytotoxicity against human DLD1 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID94808In vitro cytotoxic activity against human leukemic cell line K562 after incubation for 1 hour2003Bioorganic & medicinal chemistry letters, Jun-16, Volume: 13, Issue:12
Chemical synthesis and cytotoxicity of dihydroxylated cyclopentenone analogues of neocarzinostatin chromophore.
AID1584442Antiproliferative activity against human NCI60 cells after 48 hrs by SRB assay2018Journal of medicinal chemistry, 10-25, Volume: 61, Issue:20
Discovery of Novel Topoisomerase II Inhibitors by Medicinal Chemistry Approaches.
AID55448The compound was tested for cytotoxicity against D283 (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID96162Inhibitory activity against K-562 (human chronic myeloid Leukemia) cells1993Journal of medicinal chemistry, Apr-02, Volume: 36, Issue:7
In vitro cytotoxicity of GC sequence directed alkylating agents related to distamycin.
AID1277019Selectivity index, ratio of CC50 for HPC to CC50 for human HSC3 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1292286Selectivity index, ratio of CC50 for human HPLF cells to CC50 for human HSC4 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID270650Cytotoxicity against rat C6 glioma cell line2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Discovery of antiglioma activity of biaryl 1,2,3,4-tetrahydroisoquinoline derivatives and conformationally flexible analogues.
AID97907Lethal dose was determined for myelosuppressive activity against murine leukemia cells (L1210)1987Journal of medicinal chemistry, Mar, Volume: 30, Issue:3
Selective cytotoxicity of a system L specific amino acid nitrogen mustard.
AID283822Cytotoxicity against human HCT15 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID524790Antiplasmodial activity against Plasmodium falciparum 3D7 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID526438Cytotoxicity against human HSC2 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID540209Volume of distribution at steady state in human after iv administration2008Drug metabolism and disposition: the biological fate of chemicals, Jul, Volume: 36, Issue:7
Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds.
AID153843Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for median day of death at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID596212Anticancer activity against human RPMI8226 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID594323Antiproliferative activity against human CEM/0 cells assessed as inhibition of cell growth after 48 hrs by ZF-Coulter Counting2011Bioorganic & medicinal chemistry letters, May-01, Volume: 21, Issue:9
Synthesis of novel antimitotic agents based on 2-amino-3-aroyl-5-(hetero)arylethynyl thiophene derivatives.
AID712169Binding affinity to Escherichia coli pBR322 DNA assessed as guanine-N7 alkylation on GC-rich region after 2 hrs by piperidine cleavage assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1277020Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC3 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1315403Cytotoxicity against human MCF7 cells after 72 hrs by MTT assay2016European journal of medicinal chemistry, Aug-25, Volume: 119Design and synthesis of novel pyrazolo[1,5-a]pyrimidine derivatives bearing nitrogen mustard moiety and evaluation of their antitumor activity in vitro and in vivo.
AID712159Cytotoxicity against human A549 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID153233Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for change in body weight at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID409956Inhibition of mouse brain MAOB2008Journal of medicinal chemistry, Nov-13, Volume: 51, Issue:21
Quantitative structure-activity relationship and complex network approach to monoamine oxidase A and B inhibitors.
AID283827Cytotoxicity against human RPMI8226 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID526442Cytotoxicity against human HSC4 cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID55598The compound was tested for cytotoxicity against DAOY MR (human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID1079944Benign tumor, proven histopathologically. Value is number of references indexed. [column 'T.BEN' in source]
AID153245Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for median day of death at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1148054Antineoplastic activity against mouse EAC allografted in CF1 mouse assessed as tumor growth inhibition on day 7 measured at 33 mg/kg1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID625282Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cirrhosis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID567540Cytotoxicity against human melanoma cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID95087IC80 concentrations of the compounds were used in determining the inhibition of RNA synthesis using Jurkat T cells.1999Journal of medicinal chemistry, Apr-22, Volume: 42, Issue:8
Conformational and quantitative structure-activity relationship study of cytotoxic 2-arylidenebenzocycloalkanones.
AID99062Tested in vitro for inhibition after 48 hr exposure against murine leukemia cell line L1210/L-PAM resistant to melphalan (L-PAM)1994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID548203Cytotoxicity against human Caov3 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Dec, Volume: 45, Issue:12
Structure-cytotoxicity relationship in a series of N-phosphorus substituted E,E-3,5-bis(3-pyridinylmethylene)- and E,E-3,5-bis(4-pyridinylmethylene)piperid-4-ones.
AID653930Cytotoxicity against human HSC3 cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID1073144Induction of apoptosis in human XG6 cells assessed as late apoptotic cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 7.78%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID300176Cytotoxicity against human CEM cells2007Bioorganic & medicinal chemistry, Sep-01, Volume: 15, Issue:17
E,E,E-1-(4-Arylamino-4-oxo-2-butenoyl)-3,5-bis(arylidene)-4-piperidones: a topographical study of some novel potent cytotoxins.
AID1148051Antineoplastic activity against mouse EAC allografted in CF1 mouse assessed as animal survival on day 7 measured at 33 mg/kg1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID385000Cytotoxicity against human HSC2 cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID295423Cytotoxicity against HPC cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID1149713Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat at 2.5 mg/kg/day, ip relative to control1977Journal of medicinal chemistry, Jul, Volume: 20, Issue:7
Antitumor agents. 25. Synthesis and antitumor activity of uracil and thymine alpha-methylene-gamma-lactones and related derivatives.
AID567539Cytotoxicity against human CNSC cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID393860Cytotoxicity against human Molt4/C8 cells after 72 hrs2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID396989Cytotoxicity against human A549 cells after 72 hrs by MTT assay2009European journal of medicinal chemistry, May, Volume: 44, Issue:5
Design, cytotoxic and fluorescent properties of novel N-phosphorylalkyl substituted E,E-3,5-bis(arylidene)piperid-4-ones.
AID384302Cytotoxicity against human CEM cells after 72 hrs2008European journal of medicinal chemistry, Apr, Volume: 43, Issue:4
Design, synthesis and antiproliferative activity of some 3-benzylidene-2,3-dihydro-1-benzopyran-4-ones which display selective toxicity for malignant cells.
AID300177Cytotoxicity against mouse L1210 cells2007Bioorganic & medicinal chemistry, Sep-01, Volume: 15, Issue:17
E,E,E-1-(4-Arylamino-4-oxo-2-butenoyl)-3,5-bis(arylidene)-4-piperidones: a topographical study of some novel potent cytotoxins.
AID221821Inhibitory concentration was evaluated on human Jurkat T-cells after their exposure for 48 hours2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID45400Effect on cross resistance of CHO cells resistant to colchicine (CHRC5)1990Journal of medicinal chemistry, Jul, Volume: 33, Issue:7
Structure-activity relationships of antineoplastic agents in multidrug resistance.
AID625280Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for cholecystitis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID99076Concentration required to inhibit 50% growth of L1210 leukemia cells1998Journal of medicinal chemistry, Mar-26, Volume: 41, Issue:7
Cytotoxic activities of Mannich bases of chalcones and related compounds.
AID295420Cytotoxicity against human HL60 cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID153074Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1303) for change in body weight at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID653934Cytotoxicity against human HL60 cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID567544Cytotoxicity against human breast cancer cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID1073147Induction of apoptosis in human JJN-3 cells assessed as viable cells at 15 uM after 48 hrs by annexinV/propidium iodide staining (Rvb = 75.9%)2013Bioorganic & medicinal chemistry, Sep-01, Volume: 21, Issue:17
A 2,6,9-hetero-trisubstituted purine inhibitor exhibits potent biological effects against multiple myeloma cells.
AID283829Cytotoxicity against human SR cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID22528Half life was determined1987Journal of medicinal chemistry, Mar, Volume: 30, Issue:3
Selective cytotoxicity of a system L specific amino acid nitrogen mustard.
AID382489Cytotoxicity against human HL60 cells in presence of RPMI1640 containing 10% fetal bovine serum by trypan blue exclusion test2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID654075Cytotoxicity against human HCC2998 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID666610Cytotoxicity against human DU145 cells after 72 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and antitumor activity of formononetin nitrogen mustard derivatives.
AID1079938Chronic liver disease either proven histopathologically, or through a chonic elevation of serum amino-transferase activity after 6 months. Value is number of references indexed. [column 'CHRON' in source]
AID1148047Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat assessed as tumor growth inhibition at 2.5 mg/kg, ip qd relative to control1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 1. N-Protected vinyl, 1,2-dihaloethyl, and cyanomethyl esters of phenylalanine.
AID234203Therapeutic index is the ratio of LD90 of CFU-C to LD90 of L1210 cell lines1987Journal of medicinal chemistry, Mar, Volume: 30, Issue:3
Selective cytotoxicity of a system L specific amino acid nitrogen mustard.
AID596204Anticancer activity against human COLO205 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID615400Cytostatic activity against human CEM cells after 3 days by coulter counter assay2011European journal of medicinal chemistry, Sep, Volume: 46, Issue:9
Design, synthesis and bioevaluation of novel candidate selective estrogen receptor modulators.
AID666609Cytotoxicity against human HCT116 cells after 72 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and antitumor activity of formononetin nitrogen mustard derivatives.
AID596202Selectivity index, ratio of average CC50 for normal human cell line to CC50 for human HSC2 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID79240Comparative cellular uptake study performed on H-EMC-SS (chondrosarcoma cell line)2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID209832Cytotoxicity against human Jurkat T cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID481761Antiproliferative activity against mouse L1210 cells after 48 hrs2010Bioorganic & medicinal chemistry letters, May-01, Volume: 20, Issue:9
Symmetrical alpha-bromoacryloylamido diaryldienone derivatives as a novel series of antiproliferative agents. Design, synthesis and biological evaluation.
AID481763Antiproliferative activity against human HeLa cells after 48 hrs2010Bioorganic & medicinal chemistry letters, May-01, Volume: 20, Issue:9
Symmetrical alpha-bromoacryloylamido diaryldienone derivatives as a novel series of antiproliferative agents. Design, synthesis and biological evaluation.
AID653931Selectivity index, ratio of CC50 for human normal cells to CC50 for human HSC3 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID283828Cytotoxicity against human HL60 (TB) cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID96948Tested in vitro for the cytotoxicity as number of viable cells against L1210 cell line after 72 hr treatment at conc. of 10E-61989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID1178015Antileukemic activity against human SR cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID221823Apoptotic index calculated using human Jurkat T cells2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID364040Cytotoxicity against human A427 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID596209Anticancer activity against mouse L1210 cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID469474Cytotoxicity against human A549 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Mar, Volume: 45, Issue:3
Synthesis, characterization and structure-activity relationship of novel N-phosphorylated E,E-3,5-bis(thienylidene)piperid-4-ones.
AID1594184Cytotoxicity against human MDA-MB-468 cells incubated for 4 hrs under aerobic condition followed by compound washout and measured after 5 days by SRB assay2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID445447Clearance in human assessed as liver blood flow2010Journal of medicinal chemistry, Jan-14, Volume: 53, Issue:1
The permeation of amphoteric drugs through artificial membranes--an in combo absorption model based on paracellular and transmembrane permeability.
AID496826Antimicrobial activity against Entamoeba histolytica2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID97981Effective dose against L1210 cells.1989Journal of medicinal chemistry, May, Volume: 32, Issue:5
Synthesis of 5-[1-hydroxy(or methoxy)-2-bromo(or chloro)ethyl]-2'-deoxyuridines and related halohydrin analogues with antiviral and cytotoxic activity.
AID270651Cytotoxicity against rat astrocytes2006Journal of medicinal chemistry, Sep-21, Volume: 49, Issue:19
Discovery of antiglioma activity of biaryl 1,2,3,4-tetrahydroisoquinoline derivatives and conformationally flexible analogues.
AID1292280Cytotoxicity against human HPLF cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1132879Antitumor activity against rat Walker 256 cells allografted in Sprague-Dawley rat assessed as survival rate at 2.5 mg/kg relative to control1978Journal of medicinal chemistry, Jul, Volume: 21, Issue:7
Antitumor agents. 31. Helenalin sym-dimethylethylenediamine reaction products and related derivatives.
AID1241934Antiproliferative activity against human CEM cells assessed as inhibition of cell proliferation incubated for 3 days by Coulter counter based assay2015European journal of medicinal chemistry, Aug-28, Volume: 101Design, synthesis and antiproliferative activity of novel heterobivalent hybrids based on imidazo[2,1-b][1,3,4]thiadiazole and imidazo[2,1-b][1,3]thiazole scaffolds.
AID1594188Selectivity ratio of IC50 for human MDA-MB-468 cells under aerobic condition to IC50 for human MDA-MB-468 cells under hypoxic condition2019Bioorganic & medicinal chemistry letters, 05-15, Volume: 29, Issue:10
Prototyping kinase inhibitor-cytotoxin anticancer mutual prodrugs activated by tumour hypoxia: A chemical proof of concept study.
AID1277023Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC4 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID153375Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388 (1313) for total dose at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID1439576Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC3 cells
AID102186Tested in vitro for inhibition after 144 hr exposure against human colon carcinoma LoVo cell line1994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID125183Cytotoxicity against human Molt 4/C8 cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID79238In vitro cytotoxicity in chondrosarcoma cell line (H-EMC-SS) by Hoechst 33342 DNA Fluorometric assay2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID101869In vitro cytotoxicity activity against M4 Dau by colony forming assay2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID1439579Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC4 cells
AID248173Antiproliferative activity in MDA-MB 231 human breast cancer cells2005Journal of medicinal chemistry, Jan-27, Volume: 48, Issue:2
Antitumor-active cobalt-alkyne complexes derived from acetylsalicylic acid: studies on the mode of drug action.
AID1178014Antileukemic activity against human RPMI8226 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID596213Cytotoxicity against human HL60 cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID153974Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for tumor free survivors at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID497005Antimicrobial activity against Pneumocystis carinii2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID384996Cytotoxicity against human HGF cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID55602The compound was tested for cytotoxicity against DAOY(human) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID221951Concentration required to inhibit 50% of cell growth on human Molt 4/C8 cells.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID596214Cytotoxicity against human HSC2 cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1277026Selectivity index, ratio of CC50 for HPLF to CC50 for human HL60 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1483958Induction of apoptosis in human INA-6 cells after 3 days by V-FITC/ propidium iodide staining-based flow cytometric analysis2017Journal of natural products, 02-24, Volume: 80, Issue:2
Dioncophyllines C
AID1148053Antineoplastic activity against mouse EAC allografted in CF1 mouse assessed as ascites volume on day 7 measured at 33 mg/kg (Rvb = 4.1 +/- 1.24 mL)1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID712165Cytotoxicity against MMR-deficient human A2780/CP70 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID86203Tested in vitro for the cytotoxicity as number of viable cells against Hep-2 cell line after 72 hr treatment at conc. of 10E-61989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID100651In vitro cytotoxicity against L1210 murine leukemia cells2000Bioorganic & medicinal chemistry letters, Aug-07, Volume: 10, Issue:15
Phenyl sulfur mustard derivatives of distamycin A.
AID496832Antimicrobial activity against Trypanosoma brucei rhodesiense2010Bioorganic & medicinal chemistry, Mar-15, Volume: 18, Issue:6
Multi-target spectral moment QSAR versus ANN for antiparasitic drugs against different parasite species.
AID1292278Cytotoxicity against human HSC4 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID1916666Plasma protein binding in human by ultrafiltration method2022European journal of medicinal chemistry, Aug-05, Volume: 238Melphalan: Recent insights on synthetic, analytical and medicinal aspects.
AID295774Antitumor activity against human MOLT3 cells in presence of Penicillin-G-amidase after 72 hrs by XTT assay2007Bioorganic & medicinal chemistry, Jun-01, Volume: 15, Issue:11
Remarkable drug-release enhancement with an elimination-based AB3 self-immolative dendritic amplifier.
AID1584783Induction of DNA-interstanded-cross-link formation in [32P]-labeled 49-mer duplex DNA (unknown origin) at 1 mM in absence of H2O2 after 16 hrs by PAGE relative to control2018Journal of medicinal chemistry, 10-25, Volume: 61, Issue:20
Discovery and Optimization of Novel Hydrogen Peroxide Activated Aromatic Nitrogen Mustard Derivatives as Highly Potent Anticancer Agents.
AID1315400Cytotoxicity against human SH-SY5Y cells after 72 hrs by MTT assay2016European journal of medicinal chemistry, Aug-25, Volume: 119Design and synthesis of novel pyrazolo[1,5-a]pyrimidine derivatives bearing nitrogen mustard moiety and evaluation of their antitumor activity in vitro and in vivo.
AID596198Anticancer activity against human HT-29 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID524793Antiplasmodial activity against Plasmodium falciparum Dd2 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID398091Growth inhibition of human CEM cells after 3 days2009Bioorganic & medicinal chemistry, Jun-01, Volume: 17, Issue:11
2-(3-Aryl-2-propenoyl)-3-methylquinoxaline-1,4-dioxides: a novel cluster of tumor-specific cytotoxins which reverse multidrug resistance.
AID654076Cytotoxicity against human HCT116 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID364031Cytotoxicity against human 5637 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1275535Cytotoxicity against human HSC4 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry letters, Feb-15, Volume: 26, Issue:4
1-[3-(2-Hydroxyethylsulfanyl)propanoyl]-3,5-bis(benzylidene)-4-piperidones: A novel cluster of P-glycoprotein dependent multidrug resistance modulators.
AID712168Cytotoxicity against human A2780 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID653929Selectivity index, ratio of CC50 for human normal cells to CC50 for human HSC2 cells2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID221021Effect on RNA biosyntheses in leukemia L1210 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID221825Inhibition of protein synthesis was evaluated on human Jurkat T-cells after 8 hours.2000Journal of medicinal chemistry, Oct-19, Volume: 43, Issue:21
Sequential cytotoxicity: a theory evaluated using novel 2-[4-(3-aryl-2-propenoyloxy)phenylmethylene]cyclohexanones and related compounds.
AID1191480Cytostatic activity against mouse L1210 cells2015Bioorganic & medicinal chemistry, Feb-01, Volume: 23, Issue:3
Investigation of fatty acid conjugates of 3,5-bisarylmethylene-4-piperidone derivatives as antitumor agents and human topoisomerase-IIα inhibitors.
AID1079941Liver damage due to vascular disease: peliosis hepatitis, hepatic veno-occlusive disease, Budd-Chiari syndrome. Value is number of references indexed. [column 'VASC' in source]
AID596208Anticancer activity against human CEM cells after 72 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID1148052Antineoplastic activity against mouse EAC allografted in CF1 mouse assessed as packed cell volume on day 7 measured at 33 mg/kg (Rvb = 32.75 +/- 7.87%)1977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 2. Structure-activity studies on N-protected vinyl, 1,2-dibromoethyl, and cyanomethyl esters of several amino acids.
AID1079933Acute liver toxicity defined via clinical observations and clear clinical-chemistry results: serum ALT or AST activity > 6 N or serum alkaline phosphatases activity > 1.7 N. This category includes cytolytic, choleostatic and mixed liver toxicity. Value is
AID99172Median survival time of treated to the control was determined in L1210/LPAM cell line1989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID1148040Antitumor activity against mouse EAC allografted in CF1 mouse assessed as host survival at 33 mg/kg/day measured on day 71977Journal of medicinal chemistry, Dec, Volume: 20, Issue:12
Antineoplastic agents. 1. N-Protected vinyl, 1,2-dihaloethyl, and cyanomethyl esters of phenylalanine.
AID1664700Cytotoxicity against human HeLa cells assessed as reduction in cell viability after 48 hrs by MTT assay2020Bioorganic & medicinal chemistry, 08-01, Volume: 28, Issue:15
Tetrazoles as anticancer agents: A review on synthetic strategies, mechanism of action and SAR studies.
AID283826Cytotoxicity against human K562 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID596294Cytotoxicity against human HPC cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID39870In vitro cytotoxicity activity against B16, by colony forming assay2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID1439568Selectivity index, ratio of CC50 for HGF to CC50 for human HL60 cells
AID139721Screening of Ehrlich ascites carcinoma cells in mice at 20 mg/kg/day intraperitoneally.1980Journal of medicinal chemistry, Mar, Volume: 23, Issue:3
Antitumor agents: diazomethyl ketone and chloromethyl ketone analogues prepared from N-tosyl amino acids.
AID712162Cytotoxicity against human PANC1 cells after 5 days by MTT assay2012ACS medicinal chemistry letters, Dec-13, Volume: 3, Issue:12
Strategy for Imidazotetrazine Prodrugs with Anticancer Activity Independent of MGMT and MMR.
AID1275536Cytotoxicity against human HL60 cells after 48 hrs by trypan blue assay2016Bioorganic & medicinal chemistry letters, Feb-15, Volume: 26, Issue:4
1-[3-(2-Hydroxyethylsulfanyl)propanoyl]-3,5-bis(benzylidene)-4-piperidones: A novel cluster of P-glycoprotein dependent multidrug resistance modulators.
AID364039Cytotoxicity against human MCF7 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1439559Cytotoxicity against human CEM cells assessed as growth inhibition after 72 hrs by coulter counter method
AID153824Compound was evaluated in vivo for antitumor activity after intraperitoneal administration against P388/L-PAM(14) for change in body weight at an optimal dose of 5 mg/kg/injection1991Journal of medicinal chemistry, Jan, Volume: 34, Issue:1
Heterocyclic quinones. 17. A new in vivo active antineoplastic drug: 6,7-bis(1-aziridinyl)-4-[[3-(N,N-dimethylamino)propyl]amino]-5,8- quinazolinedione.
AID458818Selectivity index, ratio of CC50 for human normal cells to CC50 for human HSC2 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID209833Cytotoxicity against human Jurkat T cells.2001Journal of medicinal chemistry, Feb-15, Volume: 44, Issue:4
A conformational and structure-activity relationship study of cytotoxic 3,5-bis(arylidene)-4-piperidones and related N-acryloyl analogues.
AID1178006Cytotoxicity against human NCI60 cells assessed as inhibition of tumor growth after 24 hrs2014European journal of medicinal chemistry, Apr-22, Volume: 77Novel 3,5-bis(arylidene)-4-oxo-1-piperidinyl dimers: structure-activity relationships and potent antileukemic and antilymphoma cytotoxicity.
AID1277021Selectivity index, ratio of CC50 for HGF to CC50 for human HSC4 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID1292282Selectivity index, ratio of CC50 for human HGF cells to CC50 for human HSC3 cells2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID603111Induction of pEGFP-N1 plasmid DNA interstrand cross-linking at 1 to 5 uM by alkaline agarose gel shift assay2011Bioorganic & medicinal chemistry, Mar-15, Volume: 19, Issue:6
Design, synthesis and antitumor evaluation of phenyl N-mustard-quinazoline conjugates.
AID1277017Selectivity index, ratio of CC50 for HPLF to CC50 for human HSC2 cells2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID469473Cytotoxicity against human Caov3 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Mar, Volume: 45, Issue:3
Synthesis, characterization and structure-activity relationship of novel N-phosphorylated E,E-3,5-bis(thienylidene)piperid-4-ones.
AID80659Inhibition of [3H]thymidine incorporation in human lung adenocarcinoma cells in the absence of PGA enzyme1993Journal of medicinal chemistry, Apr-02, Volume: 36, Issue:7
Prodrugs of doxorubicin and melphalan and their activation by a monoclonal antibody-penicillin-G amidase conjugate.
AID666608Cytotoxicity against human SH-SY5Y cells after 72 hrs by MTT assay2012European journal of medicinal chemistry, Aug, Volume: 54Synthesis and antitumor activity of formononetin nitrogen mustard derivatives.
AID221024Inhibitory activity against L1210 leukemia cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID230380Resistance index as the ratio of IC50 against L1210/L-PAM to that of L1210 cell lines1994Journal of medicinal chemistry, Dec-09, Volume: 37, Issue:25
Synthesis and antitumor activity of a new class of pyrazolo[4,3-e]pyrrolo[1,2-a][1,4]diazepinone analogues of pyrrolo[1,4][2,1-c]benzodiazepines.
AID1439571Selectivity index, ratio of CC50 for HGF to CC50 for human HSC2 cells
AID524795Antiplasmodial activity against Plasmodium falciparum HB3 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID1079948Times to onset, minimal and maximal, observed in the indexed observations. [column 'DELAI' in source]
AID227560Alkylating activity of compound was determined by the NBP assay at 570 nm.2002Journal of medicinal chemistry, May-09, Volume: 45, Issue:10
Synthesis and in vitro evaluation of quaternary ammonium derivatives of chlorambucil and melphalan, anticancer drugs designed for the chemotherapy of chondrosarcoma.
AID567537Cytotoxicity against human NSCLC cells assessed as cell growth after 48 hrs by SRB assay2011European journal of medicinal chemistry, Jan, Volume: 46, Issue:1
Microwave supported synthesis of some novel 1,3-diarylpyrazino[1,2-a]benzimidazole derivatives and investigation of their anticancer activities.
AID238161Dissociation binding constant was determined for apoNCS binding site using fluorescence quenching titration assay; Insufficient change in fluorescence2004Journal of medicinal chemistry, Sep-09, Volume: 47, Issue:19
Design and synthesis of a nitrogen mustard derivative stabilized by apo-neocarzinostatin.
AID653937Cytotoxicity against human hematopoietic progenitor cells after 48 hrs2012European journal of medicinal chemistry, May, Volume: 51Dimeric 3,5-bis(benzylidene)-4-piperidones: a novel cluster of tumour-selective cytotoxins possessing multidrug-resistant properties.
AID364030Cytotoxicity against human RT112 cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID1079940Granulomatous liver disease, proven histopathologically. Value is number of references indexed. [column 'GRAN' in source]
AID526444Cytotoxicity against human HGF cells2010Bioorganic & medicinal chemistry letters, Nov-15, Volume: 20, Issue:22
Sequential cytotoxicity: a theory examined using a series of 3,5-bis(benzylidene)-1-diethylphosphono-4-oxopiperidines and related phosphonic acids.
AID1439577Selectivity index, ratio of CC50 for HGF to CC50 for human HSC4 cells
AID524794Antiplasmodial activity against Plasmodium falciparum GB4 after 72 hrs by SYBR green assay2009Nature chemical biology, Oct, Volume: 5, Issue:10
Genetic mapping of targets mediating differential chemical phenotypes in Plasmodium falciparum.
AID283824Cytotoxicity against human SW620 cells2007European journal of medicinal chemistry, Jan, Volume: 42, Issue:1
Design, synthesis and cytotoxic properties of novel 1-[4-(2-alkylaminoethoxy)phenylcarbonyl]-3,5-bis(arylidene)-4-piperidones and related compounds.
AID1191481Selectivity ratio of IC50 for mouse L1210 cells to IC50 for human CEM cells2015Bioorganic & medicinal chemistry, Feb-01, Volume: 23, Issue:3
Investigation of fatty acid conjugates of 3,5-bisarylmethylene-4-piperidone derivatives as antitumor agents and human topoisomerase-IIα inhibitors.
AID98949Tested in vivo for optimal non-toxic dose against L1210 cell line in CDF1 mice1989Journal of medicinal chemistry, Apr, Volume: 32, Issue:4
Synthesis, DNA-binding properties, and antitumor activity of novel distamycin derivatives.
AID548935Cytotoxicity against human A549 cells after 72 hrs by MTT assay2010European journal of medicinal chemistry, Dec, Volume: 45, Issue:12
Structure-cytotoxicity relationship in a series of N-phosphorus substituted E,E-3,5-bis(3-pyridinylmethylene)- and E,E-3,5-bis(4-pyridinylmethylene)piperid-4-ones.
AID1277014Cytotoxicity against HPLF assessed as cell death after 48 hrs by MTT assay2016Journal of medicinal chemistry, Jan-28, Volume: 59, Issue:2
3,5-Bis(3-alkylaminomethyl-4-hydroxybenzylidene)-4-piperidones: A Novel Class of Potent Tumor-Selective Cytotoxins.
AID744853Cytotoxicity against human CEM cells after 3 days by Coulter counter analysis2013European journal of medicinal chemistry, May, Volume: 63Anticancer activity of novel hybrid molecules containing 5-benzylidene thiazolidine-2,4-dione.
AID596203Selectivity index, ratio of average CC50 for normal human cell line to CC50 for human HSC3 cells2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID295418Cytotoxicity against HSC3 cells2007Bioorganic & medicinal chemistry, May-15, Volume: 15, Issue:10
3-(3,4,5-Trimethoxyphenyl)-1-oxo-2-propene: a novel pharmacophore displaying potent multidrug resistance reversal and selective cytotoxicity.
AID44632The compound was tested for cytotoxicity against C6-MR (Rat) Glioma cell lines1997Journal of medicinal chemistry, May-23, Volume: 40, Issue:11
Modulation of melphalan resistance in glioma cells with a peripheral benzodiazepine receptor ligand-melphalan conjugate.
AID89118Concentration required to inhibit 50% growth of human Molt 4/C8 T-lymphocyte cells1998Journal of medicinal chemistry, Mar-26, Volume: 41, Issue:7
Cytotoxic activities of Mannich bases of chalcones and related compounds.
AID1292275Cytotoxicity against mouse L1210 cells after 48 hrs by MTT assay2016Bioorganic & medicinal chemistry, 05-15, Volume: 24, Issue:10
Tumour-specific cytotoxicity and structure-activity relationships of novel 1-[3-(2-methoxyethylthio)propionyl]-3,5-bis(benzylidene)-4-piperidones.
AID596216Cytotoxicity against human HSC4 cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID364037Cytotoxicity against human SISO cells after 96 hrs by microtiter assay2008European journal of medicinal chemistry, Sep, Volume: 43, Issue:9
Structure-activity relationships of novel heteroaryl-acrylonitriles as cytotoxic and antibacterial agents.
AID625289Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for liver disease2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID382491Cytotoxicity against human pulp cells by MTT method2008European journal of medicinal chemistry, Jan, Volume: 43, Issue:1
Cytotoxic 3,5-bis(benzylidene)piperidin-4-ones and N-acyl analogs displaying selective toxicity for malignant cells.
AID121259Ratio of number of mice that survived after 50 days among eight mice tested2001Bioorganic & medicinal chemistry letters, Jun-04, Volume: 11, Issue:11
Esters of 2-(1-hydroxyalkyl)-1,4-dihydroxy-9,10-anthraquinones with melphalan as multifunctional anticancer agents.
AID300179Cytotoxicity against human leukemia cells2007Bioorganic & medicinal chemistry, Sep-01, Volume: 15, Issue:17
E,E,E-1-(4-Arylamino-4-oxo-2-butenoyl)-3,5-bis(arylidene)-4-piperidones: a topographical study of some novel potent cytotoxins.
AID625286Drug Induced Liver Injury Prediction System (DILIps) training set; hepatic side effect (HepSE) score for hepatitis2011PLoS computational biology, Dec, Volume: 7, Issue:12
Translating clinical findings into knowledge in drug safety evaluation--drug induced liver injury prediction system (DILIps).
AID458817Selectivity index, ratio of CC50 for human normal cells to CC50 for human HL60 cells2010Bioorganic & medicinal chemistry letters, Feb-01, Volume: 20, Issue:3
3,5-Bis(benzylidene)-1-[4-2-(morpholin-4-yl)ethoxyphenylcarbonyl]-4-piperidone hydrochloride: a lead tumor-specific cytotoxin which induces apoptosis and autophagy.
AID125184Inhibitory concentration against proliferation of human Molt 4/C8 cells2002Journal of medicinal chemistry, Jul-04, Volume: 45, Issue:14
Correlations between cytotoxicity and topography of some 2-arylidenebenzocycloalkanones determined by X-ray crystallography.
AID393872Cytotoxicity against human pulp cells after 24 hrs by MTT assay2009European journal of medicinal chemistry, Jan, Volume: 44, Issue:1
The cytotoxic properties and preferential toxicity to tumour cells displayed by some 2,4-bis(benzylidene)-8-methyl-8-azabicyclo[3.2.1] octan-3-ones and 3,5-bis(benzylidene)-1-methyl-4-piperidones.
AID596217Cytotoxicity against human HGF cells after 48 hrs2011Journal of medicinal chemistry, May-12, Volume: 54, Issue:9
3,5-Bis(benzylidene)-1-[3-(2-hydroxyethylthio)propanoyl]piperidin-4-ones: a novel cluster of potent tumor-selective cytotoxins.
AID384999Cytotoxicity against human HL60 cells2008Bioorganic & medicinal chemistry, May-15, Volume: 16, Issue:10
1-Aryl-2-dimethylaminomethyl-2-propen-1-one hydrochlorides and related adducts: A quest for selective cytotoxicity for malignant cells.
AID1347110qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for A673 cells)2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347424RapidFire Mass Spectrometry qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347116qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SJ-GBM2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347109qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for NB1643 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347123qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh41 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347129qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SK-N-SH cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347121qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347122qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347117qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for BT-37 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347119qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for MG 63 (6-TG R) cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347113qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for LAN-5 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347425Rhodamine-PBP qHTS Assay for Modulators of WT P53-Induced Phosphatase 1 (WIP1)2019The Journal of biological chemistry, 11-15, Volume: 294, Issue:46
Physiologically relevant orthogonal assays for the discovery of small-molecule modulators of WIP1 phosphatase in high-throughput screens.
AID1347126qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh30 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347112qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for BT-12 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347128qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for OHS-50 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347125qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Rh18 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347127qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for Saos-2 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347114qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for DAOY cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347124qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for RD cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347115qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for NB-EBc1 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347407qHTS to identify inhibitors of the type 1 interferon - major histocompatibility complex class I in skeletal muscle: primary screen against the NCATS Pharmaceutical Collection2020ACS chemical biology, 07-17, Volume: 15, Issue:7
High-Throughput Screening to Identify Inhibitors of the Type I Interferon-Major Histocompatibility Complex Class I Pathway in Skeletal Muscle.
AID1347118qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for TC32 cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347111qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Confirmatory screen for SK-N-MC cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1159607Screen for inhibitors of RMI FANCM (MM2) intereaction2016Journal of biomolecular screening, Jul, Volume: 21, Issue:6
A High-Throughput Screening Strategy to Identify Protein-Protein Interaction Inhibitors That Block the Fanconi Anemia DNA Repair Pathway.
AID1159550Human Phosphogluconate dehydrogenase (6PGD) Inhibitor Screening2015Nature cell biology, Nov, Volume: 17, Issue:11
6-Phosphogluconate dehydrogenase links oxidative PPP, lipogenesis and tumour growth by inhibiting LKB1-AMPK signalling.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (7,657)

TimeframeStudies, This Drug (%)All Drugs %
pre-19902600 (33.96)18.7374
1990's1340 (17.50)18.2507
2000's1554 (20.30)29.6817
2010's1691 (22.08)24.3611
2020's472 (6.16)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 74.77

According to the monthly volume, diversity, and competition of internet searches for this compound, as well the volume and growth of publications, there is estimated to be very strong demand-to-supply ratio for research on this compound.

MetricThis Compound (vs All)
Research Demand Index74.77 (24.57)
Research Supply Index9.16 (2.92)
Research Growth Index4.52 (4.65)
Search Engine Demand Index137.58 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (74.77)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials1,351 (16.61%)5.53%
Reviews642 (7.89%)6.00%
Case Studies1,123 (13.81%)4.05%
Observational23 (0.28%)0.25%
Other4,995 (61.41%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (706)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
S0833, Modified Total Therapy 3 (TT3) for Newly Diagnosed Patients With Multiple Myeloma (MM): A Phase II SWOG Trial for Patients Aged ≤ 65 Years [NCT01055301]Phase 20 participants (Actual)Interventional2011-07-31Withdrawn(stopped due to lack of accrual)
A Multicenter, Open-label, Phase Ii Study of Dasatinib in Combination With Melphalan and Prednisone (D-MP) in Advanced, Relapsed / Refractory Multiple Myeloma Patients [NCT01116128]Phase 28 participants (Actual)Interventional2008-02-29Terminated(stopped due to difficulty in enrolling patients)
Busulfan, Etoposide, Cytarabine and Melphalan (BuEAM) as a Conditioning for Autologous Stem Cell Transplantation in Patients With Diffuse Large B Cell Lymphoma (DLCBL) Previously Treated With Rituximab Based Regimen [NCT01063439]Phase 242 participants (Anticipated)Interventional2010-01-31Recruiting
Tandem Autologous Stem Cell Transplantation for Patients With Primary Progressive or Poor Risk Recurrent Hodgkin's Disease [NCT00265889]Phase 242 participants (Actual)Interventional2002-02-28Completed
Thalidomide-Dexamethasone Incorporated Into Double Autologous Stem-Cell Transplantation for Patients Less Than 65 Years of Age With Newly Diagnosed Multiple Myeloma [NCT01341262]Phase 2378 participants (Actual)Interventional2002-03-31Completed
A Phase II Study to Evaluate the Safety and Efficacy of IV Busulfan, Melphalan, and Thiotepa (BuMelTT) Followed By Autologous PBSC Infusion for Patients With Hodgkin's Disease and Non-Hodgkin's Lymphoma [NCT00238433]Phase 237 participants (Actual)Interventional2005-03-31Completed
A Phase I/II Pilot Study of Memory-like NK Cells to Consolidate TCRαβ T Cell Depleted Haploidentical Transplant in High-risk AML [NCT06158828]Phase 1/Phase 248 participants (Anticipated)Interventional2024-03-31Not yet recruiting
A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies [NCT04904588]Phase 2300 participants (Anticipated)Interventional2021-09-30Recruiting
A Phase 3, Multicenter, Randomized, Controlled, Open-label Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Subjects With Previously Untreated Multiple Myeloma Who Are Ineligible for High [NCT03217812]Phase 3220 participants (Actual)Interventional2017-11-23Active, not recruiting
Tandem Autologous Stem Cell Transplantation for Patients With Primary Progressive or Recurrent Hodgkin's Disease (A BMT Study), Phase II [NCT00233987]Phase 298 participants (Actual)Interventional2005-10-31Completed
(PRO#11307) Phase III Randomized Study of Autologous Stem Cell Transplantation With High-dose Melphalan Versus High-dose Melphalan and Bortezomib in Patients With Multiple Myeloma 65 Year or Older [NCT01453088]Phase 263 participants (Actual)Interventional2010-06-24Terminated(stopped due to Lack of Accrual)
[NCT01250808]0 participants Expanded AccessAvailable
A Phase I Study of Intensity Modulated Total Marrow Irradiation (IMTMI) in Addition to Fludarabine/Melphalan Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies [NCT02333162]Phase 130 participants (Anticipated)Interventional2014-12-05Recruiting
Pilot Study T Cell Depletion in the Setting of Autologous Stem Cell Transplantation for Patients With Multiple Myeloma [NCT01526096]Phase 115 participants (Actual)Interventional2011-07-12Active, not recruiting
A Randomized Open-label Multicenter Phase III Trial of Melphalan and Dexamethasone (MDex) Versus Bortezomib, Melphalan and Dexamethasone (BMDex) for Untreated Patients With Systemic Light-chain (AL) Amyloidosis [NCT01277016]Phase 3110 participants (Actual)Interventional2011-01-31Completed
Busulfan, Etoposide, Cytarabine, and Melphalan (BuEAM) as a Conditioning for Autologous Stem Cell Transplantation in Patients With T Cell or NK Cell Lymphoma [NCT01178658]Phase 242 participants (Anticipated)Interventional2010-07-31Recruiting
A Phase II Trial of Alpha Beta T-cell and CD19 B-cell Depleted Peripheral Blood Stem Cell Transplantation Using the CliniMACS System for Patients With Non-Malignant Hematologic Disorders From Matched or Mismatched, Related or Unrelated Donors [NCT03615144]Phase 21 participants (Actual)Interventional2018-07-23Terminated(stopped due to Participant accrual low and the study was closed)
A Study of the Pharmacokinetics of Melphalan During Treatment With Melflufen and Dexamethasone in Patients With Relapsed Refractory Multiple Myeloma and Impaired Renal Function [NCT03639610]Phase 235 participants (Actual)Interventional2018-08-28Terminated(stopped due to The sponsor decided to terminate the study following an FDA request of a partial clinical hold.)
A Phase I Trial of Ruxolitinib Combined With Tacrolimus and Sirolimus as Acute Graft-versus-Host Disease (aGVHD) Prophylaxis During Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis [NCT02528877]Phase 10 participants (Actual)Interventional2015-11-30Withdrawn(stopped due to The study design was revised so a new protocol will be opened.)
A Phase I Dose Escalation Trial of WT1-specific Donor-derived T Cells Following T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Relapsed/Refractory Multiple Myeloma [NCT01758328]Phase 129 participants (Anticipated)Interventional2012-12-31Active, not recruiting
Risk-ADAPTed Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation (ADAPT) [NCT06028828]Phase 260 participants (Anticipated)Interventional2023-09-11Recruiting
Pilot Open-label Trial of Nivolumab Combined With Chemotherapy in Refractory Myelodysplastic Syndromes. [NCT03259516]Phase 1/Phase 22 participants (Actual)Interventional2017-05-25Terminated(stopped due to Slow recruitment rate)
An International Multi-center Phase 2 Study to Evaluate the Safety and Efficacy of Sequential Melphalan Hydrochloride for Injection for Use With the Hepatic Delivery System Treatment Followed by Sorafenib in Patients With Unresectable Hepatocellular Carci [NCT02406508]Phase 20 participants (Actual)Interventional2014-10-31Withdrawn(stopped due to No enrollment in the study)
An International Multi-center Phase 2 Study to Evaluate the Efficacy and Safety of Melphalan Hydrochloride for Injection for Use With the Hepatic Delivery System Treatment in Patients With Unresectable Hepatocellular Carcinoma or Intra Hepatic Cholangioca [NCT02415036]Phase 217 participants (Actual)Interventional2014-06-30Terminated(stopped due to Limited enrollment)
A Randomized, Multi-Center, Phase III Study of Allogeneic Stem Cell Transplantation Comparing Regimen Intensity in Patients With Myelodysplastic Syndrome or Acute Myeloid Leukemia (BMT CTN #0901) [NCT01339910]Phase 3272 participants (Actual)Interventional2011-06-30Terminated(stopped due to Accrual terminated as recommended by the data and safety monitoring board.)
High Dose Chemotherapy With Autologous Stem Cell Rescue for Aggressive B Cell Lymphoma and Hodgkin Lymphoma in HIV-infected Patients (BMT CTN #0803) [NCT01141712]Phase 243 participants (Actual)Interventional2010-04-30Completed
An Open-Label, Dose-Escalation, Phase 1/2 Study of the Oral Form of Ixazomib (MLN9708), a Next-Generation Proteasome Inhibitor, Administered in Combination With a Standard Care Regimen of Melphalan and Prednisone in Patients With Newly Diagnosed Multiple [NCT01335685]Phase 1/Phase 261 participants (Actual)Interventional2011-06-27Completed
Ocular Conservative Treatment for Retinoblastoma: Efficacy of the New Management Strategies and Visual Outcome - RETINO 2018 [NCT04681417]Phase 2/Phase 3225 participants (Anticipated)Interventional2021-03-25Recruiting
A Pilot Trial of Blinatumomab Consolidation Post Autologous Stem Cell Transplantation in Patients With DLBCL [NCT03072771]Phase 114 participants (Actual)Interventional2017-08-01Completed
[NCT01342237]Phase 233 participants (Anticipated)Interventional2011-03-31Recruiting
CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplant for Malignant Disease [NCT02061800]Phase 1/Phase 215 participants (Anticipated)Interventional2013-06-03Recruiting
Multicenter Phase II, Double-blind Placebo Controlled Trial of Maintenance Ixazomib After Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Multiple Myeloma (BMT CTN 1302) [NCT02440464]Phase 257 participants (Actual)Interventional2015-08-31Completed
Allogeneic Stem Cell Transplantation for Patients With Hematological Malignancies Using Multiple Unrelated Cord Blood Units [NCT00547196]10 participants (Actual)Interventional2005-08-16Active, not recruiting
A Phase I/II Clinical Trial of NK Cells Administration to Prevent Disease Relapse for Patient With High-Risk Myeloid Malignancies Undergoing Haploidentical Stem-Cell Transplantation [NCT01904136]Phase 1/Phase 290 participants (Anticipated)Interventional2014-04-22Completed
Phase II Study of RO4929097 to Eradicate Residual Disease in Patients With Multiple Myeloma Post Single Autologous Stem Cell Transplant [NCT01251172]Phase 20 participants (Actual)Interventional2010-12-31Withdrawn(stopped due to Lack of Drug Supply)
A Multi-institutional Feasibility Study of Intra-arterial Chemotherapy Given in the Ophthalmic Artery of Children With Retinoblastoma [NCT02097134]Phase 114 participants (Actual)Interventional2014-10-31Completed
HLA-Nonidentical Stem Cell and Natural Killer Cell Transplantation for Children Less the Two Years of Age With Hematologic Malignancies [NCT00145626]Phase 240 participants (Actual)Interventional2004-05-31Completed
Phase II Study of High-Dose Topotecan, Cyclophosphamide and Melphalan for the Treatment of Multiple Myeloma [NCT01039025]Phase 260 participants (Actual)Interventional2002-02-18Completed
"First Line Treatment in HIV-related Large Cell Non Hodgkin Lymphoma at High Risk, Including Early Consolidation With High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell Transplantation" [NCT01045889]Phase 227 participants (Actual)Interventional2007-01-31Completed
Randomized Trial of Unmanipulated Versus Expanded Cord Blood [NCT00067002]Phase 2110 participants (Actual)Interventional2003-04-30Completed
A Randomized Controlled Clinical Trial Comparing Chidamide,Carmustine,Etoposide,Cytarabine and Melphalan With BEAM Regimen Combined With Autologus Hematopoietic Stem Cell Transplantation for the Treatment of Newly Diagnosed PTCL [NCT05931263]Phase 3104 participants (Anticipated)Interventional2023-06-01Recruiting
A Randomized Phase II Trial Comparing Treosulfan and Melphalan With Melphalan Alone as Conditioning Regimen for Autologous Stem Cell Transplantation (ASCT) in Myeloma Patients (TreoMel Trial) [NCT05636787]Phase 2120 participants (Anticipated)Interventional2023-06-06Recruiting
Phase II Trial Of Non-Myeloablative Regimen Combining Melphalan, Fludarabine, And Anti-CD52 Monoclonal Antibody (CAMPATH-1H) Followed By An Unmodified Hematopoietic Cell Transplant From An HLA Compatible Related Or Unrelated Donor For Treatment Of Lymphoh [NCT00027560]Phase 251 participants (Actual)Interventional2001-07-31Completed
Sequential High-Dose Melphalan and Busulfan/Cyclophosphamide Followed by Peripheral Blood Progenitor Cell Rescue, Interferon/Thalidomide and Pamidronate for Patients With Multiple Myeloma [NCT00004088]Phase 277 participants (Actual)Interventional1999-04-13Completed
Phase IIa Study of Addition of Itacitinib With Tacrolimus/Sirolimus Regimen for GVHD Prophylaxis in Fludarabine and Melphalan Non-Myeloablative Conditioning Hematopoietic Cell Transplantation for Acute Leukemias, MDS or MF [NCT04339101]Phase 259 participants (Actual)Interventional2020-11-11Active, not recruiting
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Hematologic Malignancies [NCT04282174]Phase 20 participants (Actual)Interventional2022-09-30Withdrawn(stopped due to Study was split into two new studies before the first participant was enrolled.)
A Phase I Study to Examine the Toxicity of Allogeneneic Stem Cell Transplantation for Relapsed or Therapy Refractory Ewings [NCT02472392]Phase 110 participants (Anticipated)Interventional2013-04-30Completed
A Phase 1/2 Study of Ixazomib as a Replacement for Bortezomib or Carfilzomib for Multiple Myeloma (MM) Patients Recently Relapsed or Refractory to Their Last Combination Regimen Containing Either Bortezomib or Carfilzomib [NCT02206425]Phase 1/Phase 245 participants (Actual)Interventional2014-09-30Completed
Phase 1 Trial for Patients With Advanced Hematologic Malignancies Undergoing Reduced Intensity Allogeneic HCT With a T-cell Depleted Graft With Infusion of Conventional T-cells and Regulatory T-cells [NCT05088356]Phase 140 participants (Anticipated)Interventional2021-09-07Recruiting
Phase I/II Study Utilizing High Dose Busulfan and Melphalan With Escalating Carfilzomib as Conditioning in Autologous Peripheral Blood Stem Cell Transplantation for Patients With Multiple Myeloma [NCT03795597]Phase 1/Phase 236 participants (Anticipated)Interventional2019-05-22Recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies [NCT01652092]30 participants (Anticipated)Interventional2012-09-04Recruiting
Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy [NCT03674411]Phase 222 participants (Actual)Interventional2019-01-02Active, not recruiting
Phase II Study of Clofarabine and High-Dose Melphalan Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation for Myelodysplasia or Acute Leukemia in Remission [NCT01885689]Phase 272 participants (Actual)Interventional2014-02-10Active, not recruiting
Pilot Study Using Myeloablative Busulfan/Melphalan (BuMel) Consolidation Following Induction Chemotherapy for Patients With Newly Diagnosed High-Risk Neuroblastoma [NCT01798004]Phase 1150 participants (Actual)Interventional2013-04-08Active, not recruiting
A Phase II Study of Dasatinib (Sprycel®) (NSC #732517) as Primary Therapy Followed by Transplantation for Adults >/= 18 Years With Newly Diagnosed Ph+ Acute Lymphoblastic Leukemia by CALGB, ECOG and SWOG [NCT01256398]Phase 266 participants (Actual)Interventional2010-12-14Completed
A COG Pilot Study of Intensive Induction Chemotherapy and 131I-MIBG Followed by Myeloablative Busulfan/Melphalan (Bu/Mel) for Newly Diagnosed High-Risk Neuroblastoma [NCT01175356]Phase 199 participants (Actual)Interventional2010-10-04Active, not recruiting
Evaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis [NCT03113162]Phase 115 participants (Anticipated)Interventional2015-05-29Recruiting
Phase III Trial of High-dose Melphalan and Stem Cell Transplantation Versus High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis [NCT02489500]Phase 33 participants (Actual)Interventional2015-06-30Terminated(stopped due to Enrollment held for toxicity evaluation; then closed due to competing trial)
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis [NCT03426969]Early Phase 13 participants (Actual)Interventional2018-01-31Completed
Conservative Treatments of Retinoblastoma [NCT02866136]Phase 2133 participants (Anticipated)Interventional2012-02-29Active, not recruiting
Population Pharmacokinetics (PK) of Melphalan in Pediatric Patients Undergoing Allogeneic Hematopoietic Cell Transplantation (HCT) [NCT03609827]25 participants (Actual)Observational2015-09-01Completed
Phase 1b/2a Trial of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT) From an HLA-partially Matched Related or Unrelated Donor After TCRαβ+ T-cell/CD19+ B-cell Depletion for Patients Who Will Receive a Kidney Transplant (KT) From the Same HSCT/KT [NCT05508009]Phase 1/Phase 212 participants (Anticipated)Interventional2023-01-10Recruiting
Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome [NCT00357565]Phase 220 participants (Anticipated)Interventional2005-11-30Recruiting
Carfilzomib/SAHA Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant for Patients With Refractory/Relapsed Myeloma [NCT02114502]Phase 20 participants (Actual)Interventional2014-09-30Withdrawn
A PHASE I-II MULTICENTER STUDY OF THE CLORETAZINE-DAUNORUBICIN-ARACYTINE COMBINATION FOR THE TREATMENT OF ACUTE MYELOID LEUKEMIA (AML) WITH UNFAVORABLE CYTOGENETICS [NCT00840684]Phase 1/Phase 2135 participants (Anticipated)Interventional2009-01-31Completed
A Single Arm, Open-Label, Phase 2 Study of Melflufen in Combination With Dexamethasone in Patients With Relapsed Refractory Multiple Myeloma Who Are Refractory to Pomalidomide and/or an Anti-CD38 Monoclonal Antibody [NCT02963493]Phase 2157 participants (Actual)Interventional2016-12-28Completed
A Phase I/II Dose Escalation Trial of Clofarabine, in Addition to Melphalan and Thiotepa as Myeloablative Regimen Followed by an Allogeneic Unmodified Hematopoietic Stem Cell Transplant From HLA-Compatible Related or Unrelated Donors for the Treatment of [NCT00423514]Phase 1/Phase 238 participants (Actual)Interventional2006-11-20Completed
High-Dose Doxorubicin and Ifosfamide Followed by Melphalan and Cisplatin for Patients With High-Risk and Recurrent Sarcoma [NCT00002601]Phase 213 participants (Actual)Interventional1994-09-30Completed
A Phase I/II Study of Escalating Doses of Thalidomide in Conjunction With Bortezomib and HIgh Dose Melphalan as a Conditioning Regimen for Autologous Peripheral Blood Stem Cell Transplantation in Patients With Advanced Multiple Myeloma [NCT01242267]Phase 1/Phase 229 participants (Actual)Interventional2010-05-11Completed
A Multicenter Phase 2 Study to Evaluate Subcutaneous Daratumumab in Combination With Standard Multiple Myeloma Treatment Regimens [NCT03412565]Phase 2265 participants (Actual)Interventional2018-04-26Active, not recruiting
A Phase 3, Randomized, Controlled, Open-label Study of VELCADE (Bortezomib) Melphalan-Prednisone (VMP) Compared to Daratumumab in Combination With VMP (D-VMP), in Subjects With Previously Untreated Multiple Myeloma Who Are Ineligible for High-dose Therapy [NCT02195479]Phase 3706 participants (Actual)Interventional2014-12-09Active, not recruiting
An Open-Label, Multicenter, Phase 1b Study of JNJ-54767414 (HuMax CD38) (Anti-CD38 Monoclonal Antibody) in Combination With Backbone Regimens for the Treatment of Subjects With Multiple Myeloma [NCT01998971]Phase 1242 participants (Actual)Interventional2014-02-18Active, not recruiting
Immunotherapy With Ex Vivo-Expanded Cord Blood-Derived CAR-NK Cells Combined With High-Dose Chemotherapy and Autologous Stem Cell Transplantation for B-Cell Lymphoma [NCT03579927]Phase 1/Phase 20 participants (Actual)Interventional2019-10-03Withdrawn(stopped due to Lack of Funding)
A Randomized, Two-period, Cross-over, Phase 2 Study, Comparing Pharmacokinetics, and Assessing Safety and Tolerability of Peripheral and Central i.v. Administration of Melphalan Flufenamide (Melflufen) in RRMM Patients [NCT04412707]Phase 227 participants (Actual)Interventional2020-08-04Terminated(stopped due to The sponsor decided to terminate the study following an FDA request of a partial clinical hold.)
Phase I/II Study of Carboplatin, Melphalan and Etoposide Phosphate in Conjunction With Osmotic Opening of the Blood-Brain Barrier and Delayed Intravenous Sodium Thiosulfate Chemoprotection, in Previously Treated Subjects With Anaplastic Oligodendroglioma [NCT00303849]Phase 1/Phase 233 participants (Actual)Interventional2005-09-15Completed
A Phase II Study of Enhancing Anti-Tetanus Vaccine Response After Autologous Stem Cell Transplantation [NCT02700841]Phase 28 participants (Actual)Interventional2020-01-09Terminated(stopped due to Study terminated prematurely due to poor recruitment.)
Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma (A Multicenter Prospective Single Arm Trial) [NCT04903678]18 participants (Anticipated)Interventional2021-05-01Recruiting
Reduce Intensity Conditioning (RIC) Allogenic Hematopoietic Stem Cell Transplantation (Allo HSCT) for Patients With Relapsed Multiple Myeloma: A Pilot Study [NCT04205240]Phase 21 participants (Actual)Interventional2020-12-22Terminated(stopped due to Poor accrual)
Phase I/II Study of VELCADE®-BEAM and Autologous Hematopoietic Stem Cell Transplantation for Relapsed Indolent Non-Hodgkin's Lymphoma, Transformed or Mantle Cell Lymphoma [NCT00571493]Phase 1/Phase 242 participants (Actual)Interventional2006-04-14Completed
A Randomized Trial for Adults With Newly Diagnosed Acute Lymphoblastic Leukemia [NCT01085617]Phase 31,033 participants (Actual)Interventional2010-12-31Active, not recruiting
Choice of Topotecan or Melphalan in Retinoblastoma Patients [NCT04799002]Phase 350 participants (Anticipated)Interventional2021-03-11Recruiting
Intra-Arterial (IA) Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma (ACPP) and Choroid Plexus Carcinoma (CPC) Prior to Second-Look Surgery [NCT04994977]Phase 16 participants (Anticipated)Interventional2023-05-04Recruiting
Phase 0 Microdose Study to Evaluate the Effect of Melphalan, Bortezomib and Dexamethasone on Cellular Gene-expression in Patients With Multiple Myeloma [NCT02109861]Early Phase 16 participants (Actual)Interventional2014-01-31Active, not recruiting
A Pilot Study Evaluating the Safety of Alternating Systemic Chemotherapy and Intra-Arterial Melphalan Chemotherapy in Children With Newly Diagnosed Advanced Intra-Ocular Retinoblastoma [NCT02116959]Phase 16 participants (Actual)Interventional2014-07-23Terminated(stopped due to Low accrual)
Adoptive Transfer of NY-ESO-1 TCR Engineered Peripheral Blood Mononuclear Cells (PBMC) and Peripheral Blood Stem Cells (PBSC) After a High Dose Melphalan Conditioning Regimen, With Administration of Interleukin-2, in Patients With Multiple Myeloma [NCT03506802]Phase 10 participants (Actual)Interventional2018-07-10Withdrawn(stopped due to No Participants Enrolled)
Superselective Intra-arterial Chemotherapy Treatment for Retinoblastoma- 5 Year Results From Turkey [NCT03935074]30 participants (Actual)Observational2016-08-30Completed
S0115, A Phase II Trial Evaluating Modified High Dose Melphalan (100 mg/m) And Autologous Peripheral Blood Stem Cell Supported Transplant (SCT) For High Risk Patients With Multiple Myeloma And/Or Light Chain Amyloidosis (AL Amyloidosis) (A BMT Study) [NCT00064337]Phase 2104 participants (Actual)Interventional2004-01-31Completed
Pilot Study of Allogeneic/Syngeneic Blood Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas [NCT00043979]Phase 260 participants (Actual)Interventional2002-09-19Completed
Pilot Study Of Allogeneic Peripheral Blood Progenitor Cell Transplantation In Patients With Chemotherapy-Refractory Or Poor-Prognosis Metastatic Breast Cancer [NCT00074269]Phase 25 participants (Actual)Interventional2003-07-31Terminated(stopped due to Terminated early due to poor enrollment)
High-Dose Chemo-Radiotherapy for Patients With Primary Refractory and Relapsed Hodgkin's Disease [NCT00003631]Phase 2118 participants (Actual)Interventional1998-08-31Completed
A Phase II Trial for Patients With Inflammatory (Stage IIIB) and Responsive Metastatic Stage IV Breast Cancer Using Busulfan, Melphalan and Thiotepa Followed by Autologous or Syngeneic PBSC Rescue and 12 Weeks of Post-Engraftment Immunotherapy With Low-Do [NCT00003199]Phase 250 participants (Actual)Interventional1997-11-30Completed
Multiple-center Randomized Study to Compare Fludarabine and Busulfan Versus Fludarabine, Busulfan and Melphalan in Adult Patients With Acute Myeloid Leukemia (AML) and Myelodysplasia Syndrome (MDS) [NCT05991908]Phase 3222 participants (Anticipated)Interventional2023-10-19Recruiting
A Prospective Study of Tocilizumab for the Prevention of Graft Failure and Graft-versus-Host Disease in Haplo-Cord Transplantation [NCT04395222]Phase 221 participants (Actual)Interventional2020-10-07Active, not recruiting
Clinical Study to Evaluate the Safety and Efficacy of Daratumumab and Carfilzomib-based Induction/Consolidation/Maintenance Therapy in Transplant-eligible, Ultra High-risk, Newly Diagnosed Multiple Myeloma [NCT06140966]Phase 254 participants (Anticipated)Interventional2023-10-20Recruiting
Intravitreal Melphalan for Intraocular Retinoblastoma [NCT05504291]Phase 228 participants (Anticipated)Interventional2022-11-04Recruiting
A Multi-Center, Phase 3, Randomized Trial of Matched Unrelated Donor (MUD) Versus HLA-Haploidentical Related (Haplo) Myeloablative Hematopoietic Cell Transplantation for Children, Adolescents, and Young Adults (AYA) With Acute Leukemia or Myelodysplastic [NCT05457556]Phase 3435 participants (Anticipated)Interventional2023-03-15Recruiting
An Intergroup Phase III Randomized Controlled Trial Comparing Melphalan, Prednisone and Thalidomide (MPT) Versus Melphalan, Prednisone and Lenalidomide (Revlimid(TM))(MPR) in Newly Diagnosed Multiple Myeloma Patients Who Are Not Candidates for High-Dose T [NCT00602641]Phase 3306 participants (Actual)Interventional2008-02-29Active, not recruiting
A Phase III Randomized, Double-Blind Study of Maintenance Therapy With CC-5013 (NSC # 703813) or Placebo Following Autologous Stem Cell Transplantation for Multiple Myeloma [NCT00114101]Phase 3460 participants (Actual)Interventional2004-12-15Active, not recruiting
A Pilot Study: Gene Expression and Bacterial Analysis of Oral Mucositis in Patient's Undergoing Melphalan Conditioning and Autologous Stem Cell Transplant [NCT02589860]0 participants (Actual)Observational2015-10-30Withdrawn(stopped due to No accrual)
A National, Open-label, Multicenter, Randomized, Comparative Phase IIb Study of Treatment for Newly Diagnosed Multiple Myeloma Patients Older Than 65 Years With Sequential Melphalan/Prednisone/Velcade (MPV) Followed by Revlimid/Low Dose Dexamethasone (Rd) [NCT01237249]Phase 2250 participants (Actual)Interventional2011-02-28Completed
A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL) [NCT03126916]Phase 3724 participants (Anticipated)Interventional2018-05-14Active, not recruiting
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for B-Cell Non-Hodgkin Lymphoma Using Zevalin, Fludarabine and Melphalan [NCT00577278]Phase 241 participants (Actual)Interventional2007-10-03Active, not recruiting
Randomized Phase II Multi-center Trial of Busulfan, Etoposide, and Cyclophosphamide Versus Busulfan, Etoposide, and Melphalan as Conditioning Therapy for Autologous Stem-cell Transplantation(ASCT) in Patients With Non-Hodgkin's Lymphoma [NCT03794167]Phase 275 participants (Actual)Interventional2012-06-01Completed
Clinical Study of Mitoxantrone Hydrochloride Liposome, Carmostine, Etoposide and Cytarabine as Conditioning Regimen for Autologous Hematopoietic Stem Cell Transplantation in Patients With Lymphoma [NCT05681403]53 participants (Anticipated)Interventional2023-01-15Not yet recruiting
Phase1b/2 Study Combining Hepatic Percutaneous Perfusion With Ipilimumab Plus Nivolumab in Advanced Uveal Melanoma [NCT04283890]Phase 1/Phase 283 participants (Anticipated)Interventional2019-12-04Recruiting
JAK Inhibitor Prior to Allogeneic Stem Cell Transplant for Patients With Primary and Secondary Myelofibrosis: A Prospective Study [NCT02251821]Phase 299 participants (Actual)Interventional2014-10-20Active, not recruiting
Melphalan vs Busulfan+ Cyclophosphamide+ Etoposide Conditioning Regimen for Multiple Myeloma Undergoing Autologous Hematopoietic Stem Cell Transplantation [NCT03385096]Phase 2/Phase 3122 participants (Anticipated)Interventional2018-01-02Recruiting
Busulfan, Etoposide, Cytarabine, and Melphalan (BuEAM) as a Conditioning for Autologous Stem Cell Transplantation in Patients With B Cell Lymphoma Except for Diffuse Large B Cell Lymphoma [NCT01178645]Phase 242 participants (Anticipated)Interventional2010-07-31Recruiting
Clinical Research of Allogeneic Hematopoietic Stem Cell Transplantation for Treatment of Children With Stage 4/M Neuroblastoma: A Prospective, Single-arm, Phase II, Multi-center Trial [NCT05303727]Phase 264 participants (Anticipated)Interventional2022-08-31Not yet recruiting
A Randomized Phase III Trial of Melphalan and Dexamethasone (MDex) Versus Bortezomib, Melphalan and Dexamethasone (BMDex) for Untreated Patients With Systemic Light-Chain (AL) Amyloidosis Ineligible for Autologous Stem-Cell Transplantation [NCT01078454]Phase 311 participants (Actual)Interventional2010-11-30Completed
Efficacy and Safety of Venetoclax Combined With BEAM (Carmustine, Etoposide Cytarabine and Melphalan) Pretreatment in Autologous Stem Cell Transplantation for Diffuse Large B-cell Lymphoma: a Single-center, Randomized Clinical Study [NCT05863845]52 participants (Anticipated)Interventional2023-06-01Not yet recruiting
Randomized Study of Personalized Melphalan Dosing in the Setting of Autologous Transplant [NCT03328936]Phase 20 participants (Actual)Interventional2018-09-01Withdrawn(stopped due to Sponsor decision)
Open-labelled, Multicenter Phase II Clinical Trial of Intravenous Busulfan, Melphalan and Etoposide as Conditioning for Autologous Transplantation in Patients With Poor-risk, Refractory or Relapsed Non-Hodgkin's Lymphoma [NCT03792815]Phase 251 participants (Actual)Interventional2009-10-31Completed
Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults With Hematological Malignancies [NCT05735717]Phase 2150 participants (Anticipated)Interventional2023-05-11Recruiting
A Phase II Prospective International Multicenter Clinical Trial for Eyes With Relapsed Retinoblastoma, With Randomization Depending on the Site of Relapse or on Previous Treatment [NCT04455139]Phase 22 participants (Actual)Interventional2021-11-15Terminated(stopped due to Difficulties in recruiting patients due to changing in treatment standards for the target population)
Pharmacokinetic (PK)-Directed Dosing of Captisol Enabled Melphalan for Patients With Multiple Myeloma or Light Chain (AL) Amyloidosis Undergoing High Dose Therapy and Autologous Hematopoietic Progenitor Cell Transplant [NCT02909036]Phase 146 participants (Actual)Interventional2016-09-30Active, not recruiting
Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma [NCT02605421]Phase 212 participants (Anticipated)Interventional2016-06-30Recruiting
A Single-arm, Multi-Center, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Melphalan/HDS Treatment in Patients With Hepatic-Dominant Ocular Melanoma [NCT02678572]Phase 3102 participants (Actual)Interventional2016-02-01Completed
Dose-Intense Chemotherapy and Stem Cell Rescue in the Treatment of Inflammatory Breast Carcinoma [NCT00003042]Phase 241 participants (Actual)Interventional1997-05-30Active, not recruiting
A Phase II Study of Sirolimus, Tacrolimus and Thymoglobulin®, as Graft-versus-Host- Disease Prophylaxis in Patients Undergoing Unrelated Donor Hematopoietic Cell Transplantation [NCT00691015]Phase 248 participants (Actual)Interventional2008-05-31Completed
Allogeneic Hematopoietic Cell Transplant for Hematological Cancers and Myelodysplastic Syndromes in HIV-Infected Individuals (BMT CTN #0903) [NCT01410344]Phase 220 participants (Actual)Interventional2011-09-30Completed
Clofarabine-melphalan-alemtuzumab Conditioning in Patients With Advanced Hematologic Malignancies [NCT00943592]Phase 1/Phase 282 participants (Actual)Interventional2006-03-31Completed
Myeloma X Relapse (Intensive): A Phase III Study to Determine the Role of a Second Autologous Stem Cell Transplant as Consolidation Therapy in Patients With Relapsed Multiple Myeloma Following Prior High-dose Chemotherapy and Autologous Stem Cell Rescue. [NCT00747877]Phase 3460 participants (Anticipated)Interventional2008-04-30Recruiting
An Open-Label, Phase 1/2 Study of Melflufen and Dexamethasone for Patients With AL Amyloidosis Following at Least One Prior Line of Therapy [NCT04115956]Phase 16 participants (Actual)Interventional2020-08-06Terminated(stopped due to The sponsor decided to terminate the study following an FDA request of a partial clinical hold.)
Targeted Intensification by a Preparative Regimen for Patients With High-grade B-Cell Lymphoma Utilizing Standard-dose Yttrium-90 Ibritumomab Tiuxetan (Zevalin) Radioimmunotherapy (RIT) Combined With High-dose BEAM Followed by Autologous Stem Cell Transpl [NCT00689169]Phase 275 participants (Actual)Interventional2007-08-31Completed
Randomized Phase II Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden in High Risk Patients With Multiple Myeloma or Amyloidosis [NCT00651937]Phase 280 participants (Actual)Interventional2008-03-31Completed
A Phase 1-2 Study of a Novel Conditioning Regimen of Bendamustine and Melphalan Followed by Autologous Stem Cell Transplant for Patients With Multiple Myeloma [NCT00916058]Phase 1/Phase 257 participants (Actual)Interventional2009-04-23Completed
Adoptive Immunotherapy Utilizing Activated Marrow Infiltrating Lymphocytes in the Autologous Transplant Setting in Multiple Myeloma [NCT00566098]Phase 1/Phase 226 participants (Actual)Interventional2007-11-30Completed
Reduced-dose Radiotherapy Following High-dose Chemotherapy and Autologous Stem Cell Transplantation in Patients With Central Nervous System Non-germinomatous Germ Cell Tumors [NCT02784054]Phase 225 participants (Anticipated)Interventional2014-04-30Recruiting
A Phase III Study for Patients Relapsing or Progressing After Autologous Transplantation on Total Therapy 2 (TT2, UARK 98-026): Bortezomib, Thalidomide and Dexamethasone Versus Bortezomib, Melphalan, and Dexamethasone [NCT00573391]Phase 35 participants (Actual)Interventional2006-08-31Terminated(stopped due to low accrual)
Post-Transplant Bendamustine (PT-BEN) for GVHD Prophylaxis [NCT04022239]Phase 1/Phase 240 participants (Anticipated)Interventional2020-03-13Recruiting
Pilot Open-Label Study of Safety and Efficacy of Ruxolitinib Given Peri-Transplant During Reduced Intensity Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients With Myelofibrosis [NCT02917096]Phase 118 participants (Actual)Interventional2016-11-13Completed
Phase II Study of Umbilical Cord Blood-Derived Natural Killer Cells in Conjunction With Elotuzumab, Lenalidomide and High Dose Melphalan Followed by Autologous Stem Cell Transplant for Patients With Multiple Myeloma [NCT01729091]Phase 272 participants (Anticipated)Interventional2013-06-10Active, not recruiting
A Randomized Phase II Dose Finding Study of Revlimid™ and Melphalan in Patients With Previously Untreated Multiple Myeloma [NCT00305812]Phase 251 participants (Actual)Interventional2006-03-09Completed
BMT-03: A Phase I Trial of Total Marrow Irradiation in Addition to High Dose Melphalan Conditioning Prior to Autologous Transplant for Patients With Relapsed or Refractory Multiple Myeloma [NCT02043847]Phase 112 participants (Actual)Interventional2014-01-14Completed
A Trial of Busulfan, Melphalan, Fludarabine and T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Followed by Post Transplantation Donor Lymphocyte Infusions for Patients With Relapsed or High-Risk Multiple Myeloma [NCT01131169]Phase 266 participants (Actual)Interventional2010-05-31Completed
Stem Cell Transplant as Standard Therapy for Symptomatic Multiple Myeloma [NCT00004165]Phase 30 participants Interventional1999-10-31Completed
Partially HLA-Mismatched Related Donor Hematopoietic Stem Cell Transplantation Using Killer Immunoglobulin Receptor and Human Leukocyte Antigen Based Donor Selection [NCT02880293]44 participants (Actual)Interventional2016-08-23Completed
High Dose Ascorbic Acid (HDAA) in Patients With Plasma Cell Disorders [NCT03602235]Phase 19 participants (Anticipated)Interventional2019-03-05Recruiting
A Multi-center Randomized Phase II Study of the Impact of CD34+ Cell Dose on Absolute Lymphocyte Count Following High-Dose Therapy and Autologous Stem Cell Transplantation for Relapsed and Refractory Diffuse Large B-cell Lymphoma (DLBCL) [NCT02570542]Phase 259 participants (Actual)Interventional2015-10-31Active, not recruiting
Autologous Stem Cell Transplantation With Nivolumab in Patients With Multiple Myeloma [NCT03292263]Phase 1/Phase 230 participants (Anticipated)Interventional2017-04-24Recruiting
A Phase I Study of Melphalan, Bendamustine, and Carfilzomib for Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Myeloma [NCT02148913]Phase 118 participants (Actual)Interventional2014-06-30Completed
Allogeneic Stem Cell Transplantation for Patients With Multiple Myeloma: a Pilot Feasibility Study Using a Novel Protocol [NCT02447055]Early Phase 10 participants (Actual)Interventional2015-12-31Withdrawn
A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma [NCT06172296]Phase 3478 participants (Anticipated)Interventional2024-02-14Not yet recruiting
A Phase II Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate Uproleselan (GMI-1271) for GI Toxicity Prophylaxis During Melphalan-Conditioned Autologous Hematopoietic Cell Transplantation (Auto-HCT) for Multiple Myeloma (MM) [NCT04682405]Phase 251 participants (Actual)Interventional2021-05-05Completed
A Pilot Study of Immunotherapy Including Haploidentical NK Cell Infusion Following CD133+ Positively-Selected Autologous Hematopoietic Stem Cells in Children With High Risk Solid Tumors or Lymphomas [NCT02130869]Phase 18 participants (Actual)Interventional2014-10-10Completed
Combined Transplantation of Unmanipulated Haploidentical and a SingleCord Blood Unit for Patients With Hematologic Malignancies [NCT01359254]Phase 21 participants (Actual)Interventional2010-04-30Terminated(stopped due to did not accrue enough patients.)
A Phase I, Open Label Study Evaluating the Safety and Efficacy of Adoptive Transfer of Autologous NY-ESO-1 CD8-TCR Engineered T Cells and NY-ESO-1 CD4-TCR Engineered Hematopoietic Stem Cells (HSC) After a Myeloablative Conditioning Regimen, With Administr [NCT03691376]Phase 14 participants (Actual)Interventional2019-03-08Active, not recruiting
NB2004 Trial Protocol for Risk Adapted Treatment of Children With Neuroblastoma [NCT00410631]Phase 3642 participants (Anticipated)Interventional2004-10-31Recruiting
Phase II Randomized Study of Isolated Limb Perfusion Using Melphalan With or Without Tumor Necrosis Factor in Patients With Unresectable High Grade Soft Tissue Sarcomas of the Extremity [NCT00019968]Phase 20 participants Interventional1999-08-31Completed
Phase II Study of Isolated Hepatic Perfusion With Melphalan Followed By Postoperative Hepatic Arterial Chemotherapy in Patients With Unresectable Colorectal Cancer Metastatic to the Liver [NCT00019760]Phase 20 participants Interventional1999-04-30Completed
A Phase 1 Study to Assess Safety and Tolerability of Tremelimumab and Durvalumab, Administered With High Dose Chemotherapy and Autologous Stem Cell Transplant (HDT/ASCT) [NCT02716805]Phase 16 participants (Actual)Interventional2016-12-13Terminated(stopped due to FDA placed on partial hold due to additional data)
A Multicenter Phase II Trial of Bortezomib (Velcade), Melphalan, and Dexamethasone (V-MD) in Patients With Symptomatic AL-Amyloidosis or Light Chain Deposition Disease [NCT00520767]Phase 235 participants (Actual)Interventional2007-09-30Completed
A Phase II Study of Reduced Intensity Allogeneic Transplantation for Refractory Hodgkin Lymphoma [NCT00908180]Phase 247 participants (Anticipated)Interventional2009-07-31Not yet recruiting
A Reduced Intensity Conditioning Regimen With CD3-Depleted Hematopoietic Stem Cells to Improve Survival for Patients With Hematologic Malignancies Undergoing Haploidentical Stem Cell Transplantation [NCT00566696]Phase 273 participants (Actual)Interventional2007-12-14Completed
Phase III Trial Comparing Treatment With Melphalan+Prednisolon (MP) With Melphalan+Prednisolon+Thalidomide (MPT) for Previously Untreated Elderly Patients With Multiple Myeloma [NCT00934154]Phase 3122 participants (Actual)Interventional2006-03-31Completed
Ex-Vivo Depletion of Myeloma Cells From Peripheral Blood Progenitor Cell Grafts [NCT00968396]Phase 20 participants (Actual)Interventional2013-02-28Withdrawn
Phase II Open Lable Clinical Study Efficacy and Safety of the Holistic Treatment for Young Patients With High-Risk Multiple Myeloma [NCT04008888]50 participants (Anticipated)Interventional2018-01-05Recruiting
A Phase II Trial of MRD (Melphalan, Lenalidomide and Dexamethasone) for Patients With AL Amyloidosis [NCT00679367]Phase 216 participants (Actual)Interventional2008-05-31Completed
T-Cell or Natural Killer (NK) Cell Adback in Patients With Lymphoid Malignancies Receiving Allogeneic Stem Cell Transplantation With Campath-IH Containing Conditioning Regimens [NCT00536978]Phase 222 participants (Actual)Interventional2007-09-30Completed
Ensayo Fase II de Trasplante autólogo de Sangre periférica en Pacientes Con Mieloma múltiple Tras Acondicionamiento Con Busulfan Intravenoso y Melfalan [NCT00804947]Phase 250 participants (Anticipated)Interventional2005-09-30Recruiting
Phase I/II Study of the Combination of Bortezomib With Arsenic Trioxide, Ascorbic Acid and High-Dose Melphalan for Patients With Multiple Myeloma [NCT00469209]Phase 1/Phase 260 participants (Actual)Interventional2006-06-30Completed
A Pilot Study of Lenalidomide, Melphalan and Dexamethasone in AL Amyloidosis [NCT00890552]25 participants (Actual)Interventional2009-04-30Completed
Phase II Trial of High-dose Melphalan and Bortezomib and Stem Cell Transplantation in Patients With AL Amyloidosis [NCT00790647]Phase 210 participants (Actual)Interventional2008-06-30Completed
A Phase I Study of Clofarabine Plus High Dose Melphalan as a Conditioning Regimen for Allogeneic Transplantation [NCT00641030]Phase 120 participants (Actual)Interventional2007-07-31Completed
Allogeneic Hematopoietic Cell Transplantation Using α/β+ T-lymphocyte Depleted Grafts From HLA Mismatched Donors [NCT03615105]Phase 29 participants (Actual)Interventional2018-07-25Active, not recruiting
A Randomized Phase III Study to Compare Bortezomib, Melphalan, Prednisone (VMP) With High Dose Melphalan Followed by Bortezomib, Lenalidomide, Dexamethasone (VRD) Consolidation and Lenalidomide Maintenance in Patients With Newly Diagnosed Multiple Myeloma [NCT01208766]Phase 31,503 participants (Actual)Interventional2011-01-31Active, not recruiting
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen [NCT02776202]Phase 215 participants (Anticipated)Interventional2016-05-31Recruiting
Clinical, Multicenter, Single-arm, Treatment With a Scheme With Low Doses of Bortezomib / Melphalan / Prednisone (MPV) in Patients With Multiple Myeloma (MM) Newly Diagnosed Symptomatic ≥75 Years [NCT02773550]Phase 424 participants (Actual)Interventional2014-01-31Terminated(stopped due to Low recruiment.)
An Open-Label Phase 1/2a Study of the Safety and Efficacy of Melflufen and Dexamethasone in Combination With Either Bortezomib or Daratumumab in Patients With Relapsed or Relapsed-Refractory Multiple Myeloma [NCT03481556]Phase 1/Phase 256 participants (Actual)Interventional2018-04-12Terminated(stopped due to The sponsor decided to terminate the study following an FDA request of a partial clinical hold.)
Comparison of Melphalan-Prednisone (MP) to MP Plus Thalidomide in the Treatment of Newly Diagnosed Very Elderly Patients (> 75 Years) With Multiple Myeloma [NCT00644306]Phase 3232 participants (Actual)Interventional2002-04-30Terminated(stopped due to survival advantage demonstrated)
A Multi-Institutional Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Malignant Neuro-Epithelial and Other Solid Tumors [NCT02653196]Early Phase 11 participants (Actual)Interventional2015-09-30Terminated(stopped due to The Principal Investigator left the institution.)
Phase II Study of Carfilzomib-cyclophosphamide-dexamethasone and High-dose Melphalan (HDT) Followed by Randomization Between Observation or Maintenance With Carfilzomib and Dexamethasone in Patients With Relapsed Multiple Myeloma After HDT [NCT02572492]Phase 2200 participants (Actual)Interventional2015-01-31Active, not recruiting
Haploidentical Hematopoietic Stem Cell Transplantation Using A Novel Clofarabine Containing Conditioning Regimen For Patients With Refractory Hematologic Malignancies [NCT00824135]Phase 134 participants (Actual)Interventional2009-01-31Completed
Intra-arterial (Ophthalmic Artery) Chemotherapy for Retinoblastoma [NCT00857519]10 participants (Actual)Interventional2009-01-31Completed
Phase II Study of VIDL (VP-16, Ifosfamide, Dexamethasone, L-asparaginase) Chemotherapy Followed by High-dose Chemotherapy and Autologous Stem Cell Transplantation in Patients With Stage III/IV Extranodal NK-T-Cell Lymphoma [NCT02544425]Phase 227 participants (Anticipated)Interventional2016-02-21Recruiting
Allogeneic Stem Cell Transplantation for Mantle Cell Lymphoma [NCT00006747]Phase 24 participants (Actual)Interventional2000-11-30Completed
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in [NCT00017680]Phase 225 participants Interventional1999-07-31Completed
Phase 1-2 Study of Injection of Melphalan Into the Ophthalmic Artery in Children With Retinoblastoma [NCT00906113]Phase 1/Phase 210 participants (Anticipated)Interventional2011-06-30Recruiting
A Randomised Phase II Clinical Trial Using Targeted Radiotherapy Delivered by an Yttrium-90 Radio-Labelled Anti-CD66 Monoclonal Antibody With High Dose Melphalan Compared to Melphalan Alone, Prior to Autologous Stem Cell Transplantation for Multiple Myelo [NCT00637767]Phase 225 participants (Actual)Interventional2007-12-01Terminated(stopped due to Low recruitment)
Randomized Phase 3b Study of Three Treatment Regimens in Subjects With Previously Untreated Multiple Myeloma Who Are Not Considered Candidates for High-Dose Chemotherapy and Autologous Stem Cell Transplantation: VELCADE, Thalidomide, and Dexamethasone Ver [NCT00507416]Phase 3502 participants (Actual)Interventional2007-06-30Completed
Infusion of Expanded Cord Blood T Cells Following Cord Blood Transplantation [NCT00972101]Phase 10 participants (Actual)Interventional2009-09-30Withdrawn(stopped due to Development of other studies led to termination without recruitment.)
Effects of Exercise in Combination With Epoetin Alfa During High-Dose Chemotherapy and Autologous Peripheral Blood Stem Cell Transplantation for Multiple Myeloma [NCT00577096]120 participants (Actual)Interventional2001-10-31Completed
A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation [NCT00856388]62 participants (Actual)Interventional2009-01-14Completed
A Phase II Clinical Trial for Untreated Patients With Multiple Myeloma Eligible for Stem Cell Transplant: Lenalidomide (Revlimid®) Plus Low-dose Dexamethasone (Ld x 4 Cycles) Then Stem Cell Collection Followed by Randomization to Continued Ld or Stem Cell [NCT00807599]Phase 267 participants (Actual)Interventional2008-12-10Completed
Zevalin/BEAM/Rituximab vs BEAM/Rituximab With or Without Rituximab Maintenance in Autologous Stem Cell Transplantation for Diffuse Large B-Cell Lymphomas [NCT00591630]Phase 230 participants (Actual)Interventional2007-11-14Completed
Evaluation of Benefit and Side Effects of 131I-MIBG in Combination With Myeloablative Chemotherapy and Autologous Peripheral Blood Stem Cell Transplantation for the Treatment of High-risk Neuroblastoma [NCT00798148]Phase 1/Phase 210 participants (Anticipated)Interventional2008-09-30Recruiting
A Phase I/II Study of Oral Melphalan Combined With LBH589 for Patients With Relapsed or Refractory Multiple Myeloma (MM) [NCT00743288]Phase 1/Phase 240 participants (Actual)Interventional2008-07-31Completed
Phase II Study of Low Intensity Allogeneic Transplantation in Mantle Cell Lymphoma [NCT00720447]Phase 225 participants (Anticipated)Interventional2008-11-30Not yet recruiting
Phase 1 of Exposure Targeted Melphalan Dosing [NCT04483206]Phase 190 participants (Anticipated)Interventional2021-05-20Recruiting
Three-arm Clinical Trial for Patients With Hematologic Malignancies and Mismatched Donors - Haploidentical, 1 Antigen Mismatch Related or Unrelated, and Matched Unrelated Donor (MUD)- Using a T-cell Replete Allograft and High-dose Post-transplant Cyclopho [NCT01010217]Phase 2176 participants (Actual)Interventional2009-11-05Completed
High-Dose Gemcitabine, Busulfan and Melphalan With Hematopoietic Cell Support for Patients With Relapsed/Refractory Hodgkin's Disease [NCT01200329]Phase 281 participants (Actual)Interventional2011-06-30Completed
Anti-CD7 Universal CAR-T Cells for CD7+ T/NK Cell Hematologic Malignancies: a Multi-center, Uncontrolled Trial [NCT04264078]Early Phase 130 participants (Anticipated)Interventional2021-03-01Recruiting
A Phase I Study of Amifostine Followed by High-Dose Escalation of Melphalan With Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis [NCT00052884]Phase 18 participants (Actual)Interventional2004-01-22Terminated(stopped due to Slow accrual and changes in clinical practice)
Phase I/II Study of Oral Lenalidomide and High Dose Melphalan Supported by Autologous Peripheral Blood Stem Cell Infusion for Patients With Multiple Myeloma [NCT01142232]Phase 1/Phase 260 participants (Actual)Interventional2010-08-27Completed
A Phase II Trial of Transplants From HLA-Compatible Related or Unrelated Donors With CD34+ Enriched, T-cell Depleted Peripheral Blood Stem Cells Isolated by the CliniMACS System in the Treatment of Patients With Hematologic Malignancies and Other Lethal H [NCT01119066]Phase 2422 participants (Actual)Interventional2010-05-03Completed
A Study to Compare Bone Marrow Transplantation to Standard Care in Adolescents and Young Adults With Severe Sickle Cell Disease (BMT CTN 1503) [NCT02766465]Phase 2138 participants (Actual)Interventional2016-11-30Completed
A Phase I Study of Reduced Intensity, Sequential Double Umbilical Cord Blood Transplantation Using ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematological Malignancies. [NCT00890500]Phase 112 participants (Actual)Interventional2011-01-31Completed
A Phase II Study of Reduced Intensity Sibling Allogeneic Transplantation for Relapsed, Chemosensitive, PET-positive Hodgkin Lymphoma [NCT00907036]Phase 249 participants (Anticipated)Interventional2009-07-31Not yet recruiting
A Randomized Phase I/II Study of BI-505 in Conjunction With High-dose Melphalan and Autologous Stem Cell Transplantation for Multiple Myeloma [NCT02756728]Phase 1/Phase 25 participants (Actual)Interventional2016-05-31Terminated(stopped due to Full clinical hold from FDA)
A Phase I/II Study of Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation [NCT02750254]Phase 15 participants (Actual)Interventional2016-06-27Terminated(stopped due to Toxicity. Only enrolled patients in phase I portion of trial.)
A Trial of Reduced Intensity Conditioning and Transplantation of Haplotype Mismatched and KIR Class I Epitope-Mismatched Highly Purified CD34 Cells [NCT00085449]Phase 1/Phase 20 participants (Actual)Interventional2006-05-31Withdrawn(stopped due to Funding cut, no patients enrolled)
Allogeneic Stem Cell Transplantation For Multiple Myeloma: A Two Step Approach To Reduce Toxicity Involving High Dose Melphalan and Autologous Stem Cell Transplant Followed By PBSC Allografting After Low Dose TBI [NCT00003954]Phase 1/Phase 240 participants (Anticipated)Interventional1999-03-31Completed
A Phase II Trial of Reduced Intensity Conditioning and Partially HLA-mismatched (HLA-haploidentical) Related Donor Bone Marrow Transplantation for High-risk Solid Tumors [NCT01804634]Phase 260 participants (Anticipated)Interventional2013-03-27Recruiting
Phase I Trial of Tandem Chemotherapy Cycles as Consolidation Therapy for High-Risk Epithelial Ovarian and Primary Peritoneal Cancer Utilizing Intraperitoneal Paclitaxel/IV Cyclophosphamide Followed by IV Topotecan/Intraperitoneal Cisplatin/IV Melphalan Us [NCT00550784]Phase 18 participants (Actual)Interventional2001-01-31Completed
Anastrozole Adjuvant Trial - Study of Anastrozole Compared to NOLVADEX (Tamoxifen Citrate) for Adjuvant Treatment of Early Breast Cancer (Clinical Studies), Tamoxifen Citrate Was Added to Adjunct Cytotoxic Chemotherapy- Treatment of Malignant Joint Tumor. [NCT06154590]100 participants (Anticipated)Observational [Patient Registry]2024-07-31Not yet recruiting
A Pilot Induction Regimen Incorporating Chimeric 14.18 Antibody (ch14.18, Dinutuximab) (NSC# 764038) and Sargramostim (GM-CSF) for the Treatment of Newly Diagnosed High-Risk Neuroblastoma [NCT03786783]Phase 242 participants (Actual)Interventional2019-01-14Active, not recruiting
An Open, Single-centre Non-randomized Phase II Clinical Trial on Intra-arterial Chemotherapy With Melphalan for the Treatment of Retinoblastoma (RTB) in Advanced Intraocular Stage [NCT01393769]Phase 25 participants (Actual)Interventional2009-11-30Terminated(stopped due to Only 5 subjects could be enrolled. Sample of 25 pat. not be achieved (rare disease).)
Addition of Inotuzumab Ozogamicin Pre- and Post-Allogeneic Transplantation [NCT03856216]Phase 244 participants (Anticipated)Interventional2019-10-28Recruiting
Prospective Phase I/II Trial to Jointly Optimize the Administration Schedule(s) and Dose(s) of Melphalan for Injection (Evomela) as a Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Patients With Newly Diagnosed Multiple Myel [NCT03417284]Phase 1/Phase 262 participants (Actual)Interventional2019-10-09Active, not recruiting
Study Characterizing the Impact of Different Therapeutic Strategies on Event Occurrence at 2 Years, 5 Years, 10 Years, and 15 Years, According to Prognostic Groups in Patients With Hodgkin Lymphoma [NCT00920153]Phase 3442 participants (Actual)Interventional2008-05-31Terminated(stopped due to Other new drugs)
Reduced Intensity Conditioning and Transplantation of Partially HLA-Mismatched Peripheral Blood Stem Cells for Patients With Hematologic Malignancies [NCT02581007]Phase 225 participants (Actual)Interventional2015-10-26Completed
Phase I/II Study of Intra-Arterial Melphalan Given With Intra-Arterial Carboplatin, Osmotic Blood-Brain Barrier Disruption and Delayed Otoprotective Sodium Thiosulfate for Patients With Recurrent or Progressive CNS Embryonal or Germ Cell Tumors [NCT00983398]Phase 1/Phase 217 participants (Actual)Interventional2009-07-09Active, not recruiting
Phase III Study of Single Autologous Stem Cell Transplantation Followed by Maintenance Therapy as Front-line Treatment for Myeloma [NCT00892346]Phase 380 participants (Anticipated)Interventional2009-05-31Suspended(stopped due to No avaliability of melphlan in mainland China)
A Phase I/II Dose Escalation Study Assessing the Toxicity and Efficacy of 153-Samarium-EDTMP in Place of TBI in the Conditioning Regimen for PBSCT for Patients With Multiple Myeloma [NCT00602706]Phase 1/Phase 276 participants (Anticipated)Interventional2000-01-31Completed
A Phase III, Randomized, Open-label, 3-arm Study to Determine the Efficacy and Safety of Lenalidomide(REVLIMID) Plus Low-dose Dexamethasone When Given Until Progressive Disease or for 18 Four-week Cycles Versus the Combination of Melphalan, Prednisone, an [NCT00689936]Phase 31,623 participants (Actual)Interventional2008-08-21Completed
Phase I/II Study Of The Combination Of Lenalidomide With High-Dose Melphalan For Autologous Transplant in Patients With Multiple Myeloma [NCT01079936]Phase 1/Phase 261 participants (Actual)Interventional2010-03-31Completed
A Phase 2 Study of Autologous Followed by Nonmyeloablative Allogeneic Transplantation Using Total Lymphoid Irradiation (TLI) and Antithymocyte Globulin (ATG) in Multiple Myeloma Patients [NCT00899847]Phase 29 participants (Actual)Interventional2009-05-31Completed
"Revlimid®, and Metronomic Melphalan in the Management of Higher Risk Myelodysplastic Syndromes (MDS) and CMML: A Phase 2 Study" [NCT00744536]Phase 220 participants (Actual)Interventional2008-01-31Completed
Phase III Randomized Trial of Single vs. Tandem Myeloablative Consolidation Therapy for High-Risk Neuroblastoma [NCT00567567]Phase 3665 participants (Actual)Interventional2007-11-05Completed
Pilot Study of Unrelated Cord Blood Transplantation in Patients With Poor Risk Haematological Malignancies [NCT00916045]Phase 240 participants (Anticipated)Interventional2009-09-30Terminated(stopped due to Recruitment issues)
Autologous Stem Cell Rescue With CD133+ Selected Hematopoietic Progenitor Cells in Patients With High-Risk Neuroblastoma [NCT00539500]Phase 2/Phase 33 participants (Actual)Interventional2007-10-31Terminated(stopped due to Slow Accrual.)
A Randomized, Multi-Center Phase III Trial Comparing Two Conditioning Regimens (CloFluBu and BuCyMel) in Children With Acute Myeloid Leukemia Undergoing Allogeneic Stem Cell Transplantation. [NCT05477589]Phase 3170 participants (Anticipated)Interventional2022-06-07Recruiting
An Open-Label Expanded Access Study of the Melphalan/Hepatic Delivery System (HDS) in Patients With Hepatic Dominant Ocular Melanoma [NCT05022901]Phase 330 participants (Anticipated)Interventional2022-06-10Active, not recruiting
A Phase I/II Study of Vaccination Against Minor Histocompatibility Antigens HA1 or HA2 After Allogeneic Stem Cell Transplantation for Advanced Hematologic Malignancies [NCT00943293]Phase 11 participants (Actual)Interventional2003-05-31Terminated(stopped due to Poor accrual; did not enter phase 2)
Natural Killer Cells in Allogeneic Cord Blood Transplantation [NCT01619761]Phase 113 participants (Actual)Interventional2013-05-03Active, not recruiting
A Phase II Study of GM-CSF (Sargramostim) and Rituximab Following Autologous Transplantation For Relapsed Follicular Lymphoma [NCT00521014]Phase 214 participants (Actual)Interventional2007-10-31Completed
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Myelodysplastic Syndrome [NCT05617625]Phase 250 participants (Anticipated)Interventional2024-03-31Suspended(stopped due to The study has been put on hold while pending CMS approval)
Phase II Single-Blind Randomized Trial Comparing Morbidity of Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy (CRS-HIPEC) Using Mitomycin-C Versus Melphalan for Colorectal Peritoneal Carcinomatosis [NCT03073694]Phase 2100 participants (Anticipated)Interventional2017-07-14Recruiting
UARK 2006-15: A Phase III Randomized Study of Tandem Transplants With or Without Bortezomib (Velcade) and Thalidomide (Thalomid) to Evaluate Its Effect on Response Rate and Durability of Response in Multiple Myeloma Patients [NCT00574080]Phase 320 participants (Actual)Interventional2006-07-31Terminated(stopped due to low accrual)
Phase 2 Study Of High Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support In Patients With Multiple Myeloma And Primary Light Chain Amyloidosis [NCT00007995]Phase 275 participants (Anticipated)Interventional1999-07-31Completed
Non-Ablative Chemotherapeutic Conditioning Before Allogeneic Stem Cell Transplantation [NCT00008307]Phase 252 participants (Anticipated)Interventional1998-04-30Active, not recruiting
A National, Multi-Center, Open-Label Study of Velcade in Combination With Melphalan and Prednisone (V-MP) in Older Untreated Multiple Myeloma Patients. [NCT00388635]Phase 1/Phase 260 participants (Actual)Interventional2004-04-30Completed
A Phase I Study of Isolated Hepatic Perfusion With Escalating Dose Melphalan Followed by Postoperative Hepatic Arterial Floxuridine and Leucovorin for Metastatic Unresectable Colorectal Cancers of the Liver [NCT00001576]Phase 128 participants Interventional1997-07-31Completed
Lenalidomide, Melphalan, Prednisone, and Thalidomide (RMPT) for Relapsed/Refractory Multiple Myeloma [NCT00961467]Phase 244 participants (Actual)Interventional2007-02-28Completed
Dose Escalation Study of 131 I-Metaiodobenzylguanidine (MIBG) With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma - A Phase I Study [NCT00005978]Phase 10 participants Interventional2000-05-31Completed
A Pilot Study Of Tandem High Dose Chemotherapy With Stem Cell Rescue Following Induction Therapy In Children With High Risk Neuroblastoma [NCT00017368]Phase 242 participants (Actual)Interventional2001-04-30Completed
Phase I Study of Escalating Doses of Radiation Therapy Using Helical Tomotherapy in Combination With Fludarabine (FLU) and Melphalan (MEL) as a Preparative Regimen for Allogeneic Hematopoietic Stem Cell (HSC) Transplantation in Patients With Advanced and [NCT00800150]Phase 16 participants (Actual)Interventional2008-11-30Terminated(stopped due to Protocol objective could not be met. A new study with amended eligibility criteria will be developed.)
Infusional Gemcitabine and High-dose Melphalan (HDM) Conditioning Prior to (ASCT) Autologous Stem Cell Transplantation for Patients With Relapsed/Refractory Lymphoma [NCT02295722]Phase 1/Phase 2100 participants (Actual)Interventional2015-04-30Terminated(stopped due to It did not show a significant benefit to justify completing the full target accrual.)
Busulfan and Melphalan With Autologous Hematopoietic Stem Cell Support With Positively-Selected CD133+ Hematopoietic Cells for Children With High Risk Solid Tumors and Lymphomas [NCT00152126]26 participants (Actual)Interventional2003-08-31Completed
Phase II Study of Early Allogeneic Blood Stem Cell Transplantation During Induction-chemotherapy Induced Aplasia in High-risk Acute Myeloid Leukemia [NCT00188136]Phase 232 participants (Actual)Interventional2002-08-31Completed
A Phase II Trial of Busulfan, Melphalan, and Fludarabine With Peri-transplant Palifermin, Followed by a T-Cell Depleted Hematopoietic Stem Cell Transplant From HLA Matched or Mismatched Related or Unrelated Donors in Patients With Advanced Myelodysplastic [NCT00629798]Phase 264 participants (Actual)Interventional2008-02-12Completed
Pharmacogenomic Study to Predict Survival, Best Response and Toxicity in Newly Diagnosed Myeloma Patients Above the Age of 65 Treated With Either a Combination of Melphalan-prednisone-thalidomide or Lenalidomide-dexamethasone [NCT00907452]143 participants (Actual)Interventional2009-07-29Completed
High Dose Sequential Therapy for Poor Risk Recurrent or Refractory Hodgkin's Disease [NCT00544570]30 participants (Anticipated)Interventional1998-04-30Completed
Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study [NCT00160355]Phase 14 participants (Actual)Interventional2005-05-31Completed
A Phase III, Multicentre, Randomized, Double-Blind, Placebo-Controlled, 3-Arm Parallel Group Study To Determine The Efficacy And Safety Of Lenalidomde (Revlimid®) In Combination With Melphalan And Prednisone Versus Placebo Plus Melphalan And Prednisone In [NCT00405756]Phase 3459 participants (Actual)Interventional2007-01-31Completed
Bortezomib (Velcade®) and Reduced-Intensity Allogeneic Stem Cell Transplantation for Patients With Lymphoid Malignancies [NCT00439556]Phase 240 participants (Actual)Interventional2007-02-13Completed
Mini-Allogeneic Peripheral Blood Progenitor Cell Transplantation For Recurrent or Metastatic Breast Cancer [NCT00429572]Phase 219 participants (Actual)Interventional1998-01-31Completed
Adaptive Randomization of Fludarabine-Melphalan Versus Fludarabine-Cyclophosphamide Conditioning Regimen in Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation Using HLA-Matched Related Donor for Metastatic Renal Cell Carcinoma [NCT00429026]Phase 240 participants (Actual)Interventional2004-01-31Terminated(stopped due to Slow accrual, study terminated.)
A Randomized Phase III Trial of Hyperthermic Isolated Limb Perfusion With Melphalan, Tumor Necrosis Factor, and Interferon-Gamma in Patients With Locally Advanced Extremity Melanoma [NCT00001296]Phase 3122 participants Interventional1992-02-29Completed
A Phase II Trial of a Chemotherapy Based Regimen of Intravenous Busulfan (Busulfex), Melphalan and Thiotepa as Myeloablative Regimen Followed by a T- Cell Depleted Allogeneic Hematopoietic Stem Cell Transplant From and HLA-Compatible Donor in the Treatmen [NCT00357396]Phase 210 participants (Actual)Interventional2005-06-30Completed
Non-myeloablative Allogeneic Transplantation for the Treatment of Multiple Myeloma [NCT00185614]Phase 263 participants (Actual)Interventional2000-08-31Completed
Panobinostat Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant for Patients With Refractory/Relapsed Myeloma [NCT02506959]Phase 283 participants (Actual)Interventional2015-09-14Active, not recruiting
Study of Allogeneic Transplantation in Patients With Cutaneous T-Cell Lymphoma (CTCL) [NCT00506129]Phase 233 participants (Actual)Interventional2003-09-30Completed
An Evaluation of Bortezomib (VelcadeR ) Followed by High-Dose Melphalan and Bortezomib (VelcadeR) as Conditioning Regimen for Tandem Peripheral Blood Stem Cell Transplants in Patients With Primary Refractory Multiple Myeloma and Plasma Cell Leukemia [NCT00307086]Phase 230 participants (Actual)Interventional2005-06-30Completed
A Study of Intensive-Dose Melphalan and Topotecan (MT) Followed by Autologous Stem Cell Rescue in Patients With Multiple Myeloma. [NCT00325416]Phase 1/Phase 2177 participants (Actual)Interventional2001-11-19Completed
A Phase II Study of Concurrent Systemic Pembrolizumab and Isolated Limb Infusion (ILI) With Melphalan and Dactinomycin for Patients With Locally Advanced or Metastatic Extremity Sarcoma [NCT04332874]Phase 230 participants (Anticipated)Interventional2020-04-01Recruiting
Multiple Myeloma Treatment With Thalidomide. Three Randomized Studies on Thalidomide as Induction Treatment Before Autotransplant (MY-TAG) or With a Conventional Chemotherapy (MY-DECT) and as Consolidation/Maintenance at Plateau Phase (MY-PLAT). [NCT01070862]Phase 30 participants Interventional2003-05-31Completed
A Phase II Study of High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, Melphalan, Thymoglobulin and Autologous CD34+ Hematopoietic Stem Cell Transplant for the Treatment of Poor Prognosis Multiple Sclerosis [NCT00288626]Phase 225 participants (Actual)Interventional2006-07-31Completed
T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies [NCT00683046]Phase 2204 participants (Actual)Interventional2001-11-30Completed
A Randomized, National, Open-label, Multicenter, Phase III Trial Studying Induction Therapy With Bortezomib/Lenalidomide/Dexamethasone (VRD-GEM), Followed by High-dose Chemotherapy With Melphalan-200 (MEL-200) Versus Busulfan-melphalan (BUMEL), and Consol [NCT01916252]Phase 3460 participants (Anticipated)Interventional2013-09-30Completed
Prospective Observational Study on Isolated Limb Perfusion of Melphalan in Treating Patients With Metastasis or Recidivism of Limb Melanoma or Sarcoma That Are Not Operable [NCT01920516]40 participants (Anticipated)Observational2013-07-31Recruiting
A Phase 2, Open-label, Prospective, Multicenter Study to Evaluate the Efficacy of Intravenous Busulfan and Melphalan as a Conditioning Regimen in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation [NCT01923935]Phase 2105 participants (Anticipated)Interventional2013-01-31Recruiting
Phase I/II Bortezomib, Melphalan and Low Dose TBI Conditioning for Patients Undergoing Autologous Stem Cell Transplantation for Multiple Myeloma [NCT01936090]Phase 111 participants (Actual)Interventional2013-08-31Completed
TCRαβ-depleted Progenitor Cell Graft With Additional Memory T-cell DLI, Plus Selected Use of Blinatumomab, in Naive T-cell Depleted Haploidentical Donor Hematopoietc Cell Transplantation for Hematologic Malignancies [NCT03849651]Phase 2140 participants (Anticipated)Interventional2019-01-31Recruiting
A Phase II Study of Reduced Intensity Conditioning in Pediatric Patients and Young Adults ≤55 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation [NCT01962415]Phase 2100 participants (Anticipated)Interventional2014-02-04Recruiting
A Phase II Trial Of BEAM/Rituximab/Autologous Hematopoietic Stem Cell Transplantation (AHSCT) For Patients With CD20 Positive Non-Hodgkin's Lymphoma [NCT00080886]Phase 268 participants (Actual)Interventional2002-05-08Completed
International, Multi-center, Prospective, Double Randomized, Open Phase III Study Evaluating Thalidomide/Dexamethasone Versus Melphalan/Prednisone as Induction Therapy and Thalidomide/Interferon-alpha Versus Interferon-alpha as Maintenance Therapy in Newl [NCT00205751]Phase 2/Phase 3350 participants (Anticipated)Interventional2001-08-31Completed
Efficacy of 6x R-CHOP Followed by Myeloablative Radiochemotherapy and Autologous Stem Cell Transplantation vs. 3 x (R-CHOP/R-DHAP) Followed by a High Dose ARA-C Containing Myeloablative Regimen and Autologous Stem Cell Transplantation [NCT00209222]Phase 3360 participants (Anticipated)Interventional2004-07-31Recruiting
Panobinostat Combined With High-Dose Gemcitabine/Busulfan/Melphalan With Autologous Stem Cell Transplant for Patients With Refractory/Relapsed Lymphoma [NCT02961816]Phase 20 participants (Actual)Interventional2017-06-30Withdrawn(stopped due to Lack of funding)
A Randomized Study of Purged Versus Unpurged Peripheral Blood Stem Cell Transplant Following Dose Intensive Induction Therapy for High Risk Neuroblastoma [NCT00004188]Phase 3495 participants (Actual)Interventional2001-02-28Completed
A Phase I Trial of High Dose Therapy and Autologous Stem Cell Transplantation Followed by Infusion of Chimeric Antigen Receptor (CAR) Modified T-Cells Directed Against CD19+ B-Cells for Relapsed and Refractory Aggressive B Cell Non-Hodgkin Lymphoma [NCT01840566]Phase 117 participants (Actual)Interventional2013-04-30Active, not recruiting
A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Relapsed or Therapy Refractory Ewing Sarcoma [NCT01969942]Phase 10 participants (Actual)Interventional2013-04-30Withdrawn(stopped due to No subjects meeting study inclusion were enrolled.)
Personalized NK Cell Therapy in CBT [NCT02727803]Phase 2100 participants (Anticipated)Interventional2016-05-19Recruiting
A Phase I/IIa, Open-label, Non-randomized, Study of ASC-101 in Patients With Hematologic Malignancies or Myelodysplastic Syndrome (MDS) Who Are Candidates for Dual-cord Umbilical Cord Blood Transplantation (UCBT) [NCT01983761]Phase 1/Phase 225 participants (Anticipated)Interventional2013-11-30Active, not recruiting
A Phase I-II Trial of Adoptive Immunotherapy Using Haploidentical, Related Donor-Lymphocyte Infusions and IL-2 After Autologous Stem Cell Transplantation for Advanced Lymphoid Malignancies [NCT00248430]Phase 1/Phase 220 participants (Anticipated)Interventional2003-08-31Completed
Induction Therapy With Bortezomib and Dexamethasone Followed by Autologous Stem Cell Transplantation Versus Autologous Stem Cell Transplantation Alone in the Treatment of AL Amyloidosis [NCT01998503]Phase 356 participants (Actual)Interventional2007-12-31Completed
A Phase 2 Multicenter Study of High Dose Chemotherapy With Autologous Stem Cell Transplant Followed by Maintenance Therapy With Romidepsin for the Treatment of T Cell Non-Hodgkin Lymphoma [NCT01908777]Phase 247 participants (Actual)Interventional2013-07-16Active, not recruiting
A Phase I Study of Stem Cell Gene Therapy for HIV Mediated by Lentivector Transduced, Pre-Selected CD34+ Cells [NCT02797470]Phase 1/Phase 211 participants (Actual)Interventional2016-06-23Active, not recruiting
A Multicenter Phase II Study Evaluating BeEAM (Bendamustine, Etoposide, Cytarabine, Melphalan) Prior to Autologous Stem Cell Transplant for First and Second Chemosensitive Relapses in Patients With Follicular Lymphoma [NCT02008006]Phase 221 participants (Actual)Interventional2014-07-09Terminated(stopped due to Insufficient recruitment and unavailability of the treatment)
Intra-arterial Chemotherapy for the Treatment of Intraocular Retinoblastoma [NCT01293539]Phase 210 participants (Actual)Interventional2011-03-31Terminated(stopped due to IAC is now recognized as part of standard of care treatment options in the US.)
"Phase I Study of Yttrium-90 Labeled Anti-CD25 (a-Tac) Monoclonal Antibody Plus BEAM for Autologous Hematopoietic Cell Transplantation (AHCT) in Patients With Primary Refractory or Relapsed Hodgkin Lymphoma, the a-Tac BEAM Regimen" [NCT01476839]Phase 125 participants (Actual)Interventional2012-11-09Active, not recruiting
Phase I Study of Bortezomib With or Without Total Marrow Irradiation (TMI) Using Intensity Modulated Radiation Therapy (IMRT) in Combination With Fludarabine (FLU) and Melphalan (MEL) as a Preparative Regimen for Allogeneic Hematopoietic Stem Cell (HSC) T [NCT01163357]Phase 118 participants (Actual)Interventional2011-01-28Active, not recruiting
Double Dose Intensive Chemo-Radiotherapy With Peripheral Blood Progenitor Cell Rescue for Children With Advanced Stage Neuroblastoma and Sarcomas [NCT00165139]Phase 220 participants (Actual)Interventional1996-01-31Completed
Anti-CD19 Universal CAR-T Cells for CD19+ B Cell Hematologic Malignancies: a Multi-center, Uncontrolled Trial [NCT04264039]Early Phase 130 participants (Anticipated)Interventional2020-04-01Not yet recruiting
AA Phase IIA Open-Label, Randomized, PK Comparative, Cross-Over Study of Melphalan HCL for Injection (Propylene Glycol-Free) and Alkeran for Injection for Myeloablative Conditioning in MM Patients Undergoing Autologous Transplantation [NCT00925782]Phase 224 participants (Actual)Interventional2010-01-31Completed
High Dose Chemotherapy And Autologous Peripheral Blood Stem Cell Rescue For High Risk Acute Leukemia [NCT00008190]Phase 20 participants Interventional1999-03-31Completed
A Phase III Multicenter, Randomized, Open-Label Trial Evaluating High Dose Melphalan Plus Holmium-166-DOTMP Versus High Dose Melphalan Alone When Given In Conjuction With Peripheral Blood Stem Cell Transplantation In Patients With Multiple Myeloma [NCT00008229]Phase 30 participants Interventional2000-08-31Completed
A Phase II Study Of Blood Stem Cell Mobilization With Intravenous Melphalan (60 MG/M2) + G-CSF In Patients With Multiple Myeloma [NCT00008268]Phase 20 participants Interventional2000-08-31Completed
A Phase II Trial of Rituxan and BEAM High-Dose Chemotherapy and Autologous Peripheral Blood Progenitor Transplant for Lymphoma [NCT00007852]Phase 244 participants (Actual)Interventional2000-09-01Completed
A Phase I Dose Escalation Trial to Evaluate Safety and Efficacy of Oral Sorafenib (Nexavar) With Regional Melphalan Via Normothermic Isolated Limb Infusion (ILI) in Patients With Intransit Extremity Melanoma [NCT00565968]Phase 120 participants (Actual)Interventional2007-10-31Completed
UARK 2007-01, A Phase II Pilot Study of the Combination of Melphalan, Bortezomib, Thalidomide and Dexamethasone (MEL-VTD) and Autologous Transplantation for Patients Relapsing or Progressing After Tandem Transplantation [NCT00577668]Phase 20 participants (Actual)Interventional2007-04-30Withdrawn(stopped due to Poor accrual)
MINIALO-VELCADE2005: A Phase II National, Open-label, Multicenter, no Controlled Study of Treated With Bortezomib (Velcade) Multiple Myeloma Patients Pre and Post Allogeneic Haematopoietic Progenitor Cell Transplant With no Myeloablative Conditioning [NCT00564200]Phase 230 participants (Anticipated)Interventional2007-11-30Completed
Phase III Trial of Stem Cell Transplantation Compared to Parenteral Melphalan and Oral Dexamethasone in the Treatment of Primary Systemic Amyloidosis (AL) [NCT00477971]Phase 389 participants (Actual)Interventional2005-10-31Completed
Programma di Terapia Per Pazienti Affetti da Linfoma Diffuso a Grandi Cellule B CD20 Positive [NCT00556127]Phase 294 participants (Actual)Interventional2002-06-30Completed
Phase I/II Trial of Melphalan, Prednisone Plus Lenalidomide in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Stem Cell Transplant [NCT00477750]Phase 1/Phase 233 participants (Actual)Interventional2005-06-30Completed
An Expanded Access Program Protocol for Melphalan Flufenamide in Combination With Dexamethasone in Patients With Triple Class Refractory Multiple Myeloma [NCT04534322]0 participants Expanded AccessApproved for marketing
A Randomized Double-Blind Phase III Study of Ibrutinib During and Following Autologous Stem Cell Transplantation Versus Placebo in Patients With Relapsed or Refractory Diffuse Large B-Cell Lymphoma of the Activated B-Cell Subtype [NCT02443077]Phase 3302 participants (Anticipated)Interventional2016-10-12Active, not recruiting
A Phase 3, Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of Dexamethasone Plus MLN9708 or Physicians Choice of Treatment Administered to Patients With Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis [NCT01659658]Phase 3177 participants (Actual)Interventional2012-12-12Terminated(stopped due to Sponsor's decision)
A Prospective Study of Optimal Cord Selection for Haplo-Cord Transplantation: Targeting the Inherited Paternal Antigen (IPA) and Matching for the Non-Inherited Maternal Antigen (NIMA) [NCT01810588]Phase 2273 participants (Actual)Interventional2012-10-16Active, not recruiting
Reduced Intensity Conditioning With Fludarabine and Busulfan Versus Fludarabine and Melphalan Before Allogeneic Stem Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndrome: A Randomised, Single-Center, Phase III Study [NCT05674539]Phase 3200 participants (Anticipated)Interventional2022-12-28Recruiting
Phase II Trial of a Chemotherapy Alone Regimen of IV Busulfan (Busulfex), Melphalan and Fludarabine as Myeloablative Regimen Followed by an Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplant From an HLA-Identical, or HLA-Non Identical Related o [NCT00582933]Phase 296 participants (Actual)Interventional2001-05-31Completed
Tandem High-dose Chemotherapy and Autologous Stem Cell Rescue in Patients With High-risk Neuroblastoma [NCT00793845]Phase 240 participants (Anticipated)Interventional2008-08-31Completed
An International Single-Arm Study to Provide Further Safety and Efficacy Data on the Bortezomib(Velcade)/Melphalan/Prednisone Regimen in Previously Untreated Transplant Ineligible Multiple Myeloma Patients [NCT00799539]0 participants Expanded AccessNo longer available
Phase I/II Trial to Determine the Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Ste [NCT05436418]Phase 1/Phase 2240 participants (Anticipated)Interventional2022-11-18Recruiting
A Phase I Trial Evaluating Escalating Doses of 211At-Labeled Anti-CD38 Monoclonal Antibody (211At-OKT10-B10) Combined With Melphalan as Conditioning Prior to Autologous Hematopoietic Cell Transplantation for Patients With Multiple Myeloma [NCT04466475]Phase 130 participants (Anticipated)Interventional2024-01-27Recruiting
Lenalidomide and Low Dose Dexamethasone Induction Therapy Followed by Low Dose Melphalan, Prednisone, Lenalidomide and Bortezomib Sequential Maintenance Therapy for Newly Diagnosed High-risk Multiple Myeloma [NCT00691704]Phase 218 participants (Actual)Interventional2008-08-31Completed
S0629, Observational Study of Asymptomatic Waldenstrom's Macroglobulinemia and Phase II Study of Tandem Autologous Transplant and Maintenance Treatment for Patients With Symptomatic Disease [NCT00723658]Phase 20 participants (Actual)Interventional2008-09-30Withdrawn(stopped due to lack of accrual)
A Pilot Study of a Novel Sequential Treatment Utilizing CPX-351 as Salvage Chemotherapy Followed by Allogeneic Stem-Cell Transplantation (SCT) Utilizing a Haplo-cord Graft for Patients With Relapsed or Refractory Leukemia or Myelodysplastic Syndrome [NCT03393611]Phase 114 participants (Actual)Interventional2012-11-30Completed
Belinostat Combined With Azacitidine/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Refractory or Relapsed Lymphoma [NCT02701673]Phase 1/Phase 20 participants (Actual)Interventional2016-06-30Withdrawn
Autologous and Allogeneic Transplantation for T-Cell Lymphoma: Impact of Campath -1H and Soluble CD52 [NCT00505921]Phase 227 participants (Actual)Interventional2003-03-31Terminated(stopped due to Slow Accrual.)
A Phase II Evaluation of High Dose Chemotherapy and Autologous Stem Cell Transplantation for Intestinal T-cell Lymphomas [NCT00669812]Phase 260 participants (Anticipated)Interventional2008-02-29Recruiting
Abatacept for Graft Versus Host Disease Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease: a Sickle Transplant Alliance for Research Trial [NCT02867800]Phase 124 participants (Actual)Interventional2016-07-31Active, not recruiting
Allogeneic Stem Cell Transplantation for Children and Adolescents With CML: Conditioning Regimen, Donor Selection, Supportive Care and Diagnostic Procedures [NCT02707393]Phase 2/Phase 313 participants (Actual)Interventional2009-04-30Completed
High Dose Thiotepa and Melphalan With Autologous Hematopoietic Stem Cell Transplant in Children and Adolescents With Solid Tumors [NCT00607984]20 participants (Anticipated)Interventional2006-06-30Recruiting
A Phase I Study of Hepatic Arterial Infusion of Escalating Dose Melphalan With Venous Filtration for Metastatic Unresectable Cancers of the Liver [NCT00030082]Phase 10 participants Interventional2001-07-31Completed
A Multicenter Phase I/II Dose Escalation Study of Lenalidomide in Combination With Melphalan and Dexamethasone in Subjects With Newly-diagnosed Light-chain (AL)-Amyloidosis [NCT00621400]Phase 1/Phase 227 participants (Actual)Interventional2008-01-31Completed
A Phase II Study of Reduced-Intensity Allogeneic Peripheral Blood Stem Cell Transplantation (PBSCT) for Treatment of Hematologic Malignancies and Hematopoietic Failure States [NCT00997386]Phase 216 participants (Actual)Interventional2009-09-30Completed
Autologous Transplantation of Haematopoietic Stem Cells With Conditioning Including Zevalin + BEAM to Patients Suffering From Refractory Large B-cell Diffuse Lymphom [NCT00646750]Phase 231 participants (Actual)Interventional2008-01-31Completed
An Open-Label, Multicenter, Phase 1 Dose Escalation Study to Evaluate Safety, Tolerability and Anti-tumor Activity of Systemic Buthionine Sulfoximine (BSO) in Combination With Normothermic Isolated Limb Infusion of Melphalan in Subjects With Locally Advan [NCT00661336]Phase 10 participants (Actual)Interventional2008-04-30Withdrawn(stopped due to The study was withdrawn due to lack of patients.)
Bortezomib* and Vorinostat as Maintenance Therapy After Autologous Transplant for Non-Hodgkin Lymphoma Using R-BEAM or BEAM Conditioning Transplant Regimen [NCT00992446]Phase 227 participants (Actual)Interventional2010-09-02Completed
A Randomized, Open-Label, Phase 2 Study of CNTO 328 (Anti-IL-6 Monoclonal Antibody) and VELCADE-Melphalan-Prednisone Compared With VELCADE-Melphalan-Prednisone for the Treatment of Previously Untreated Multiple Myeloma [NCT00911859]Phase 2118 participants (Actual)Interventional2009-06-30Completed
Evaluation of Melphalan+Bortezomib as a Conditioning Regimen for Autologous and Allogeneic Stem Cell Transplants in Multiple Myeloma After Cytoreductive Therapy [NCT00948922]Phase 2124 participants (Actual)Interventional2009-06-18Completed
Safety, Tolerability and Efficacy of Ultra-low Doses of Alkylating Drug Melphalan Inhalations for the Treatment of Non-cystic Fibrosis Bronchiectasis [NCT04278040]Phase 27 participants (Anticipated)Interventional2018-06-20Recruiting
Reduced Intensity Haploidentical Peripheral Blood Stem Cell Transplantation With Post-transplant Cyclophosphamide and Sirolimus/Mycophenolate Mofetil/RGI-2001 Based GVHD Prevention: a Pilot Study [NCT04473911]Phase 125 participants (Actual)Interventional2020-08-14Active, not recruiting
A Phase I Study to Examine the Toxicity of Killer IG-Like Receptor (KIR) Mismatched Umbilical Cord Blood for Pediatric Patients With Malignant Solid Tumors [NCT00436761]Phase 120 participants (Anticipated)Interventional2004-05-31Active, not recruiting
Vorinostat (SAHA) Combined With High-Dose Gemcitabine, Busulfan, and Melphalan With Autologous Hematopoietic Cell Support for Patients With Relapsed or Refractory Lymphoid Malignancies [NCT01421173]Phase 178 participants (Actual)Interventional2011-08-31Completed
IFM 2014-02 Study: A Randomized Phase III Study of Bortezomib-Melphalan 200 Conditioning Regimen Versus Melphalan 200 for Frontline Transplant Eligible Patients With Multiple Myeloma [NCT02197221]Phase 3300 participants (Actual)Interventional2015-01-31Completed
Phase II Study of Melphalan, Arsenic Trioxide, and Ascorbic Acid in Patients With Relapsed or Refractory Multiple Myeloma [NCT00085345]Phase 20 participants (Actual)InterventionalWithdrawn
Risk Adapted Intravenous Melphalan and Adjuvant Thalidomide and Dexamethasone for Untreated Patients With Primary Systemic Amyloidosis [NCT00089167]Phase 20 participants Interventional2002-05-31Completed
A Phase II Trial Of Isolated Hepatic Perfusion (IHP) With Melphalan For Subjects With Metastatic Unresectable Colorectal Cancers Of The Liver With Disease Refractory To First Line Systemic Chemotherapy [NCT00089401]Phase 28 participants (Actual)Interventional2004-07-31Completed
A Multicenter, Open-Label, Single Arm, Phase II Study of Daratumumab as Consolidation/Maintenance Therapy After Autologous Stem Cell Transplantation in Patients With Multiple Myeloma [NCT03346135]Phase 231 participants (Actual)Interventional2019-07-17Active, not recruiting
Propylene Glycol-Free Melphalan HCl (EVOMELA®) in Combination With Fludarabine and Total Body Irradiation Based Reduced Intensity Conditioning for Haploidentical Transplantation [NCT03159702]Phase 243 participants (Anticipated)Interventional2017-12-08Recruiting
Multicenter Phase I/II Study of Haploidentical Hematopoietic Cell Transplantation With CD3/CD19 Depleted Grafts in Patients With Treatment Refractory Hematologic Malignancies [NCT00202917]Phase 1/Phase 261 participants (Actual)Interventional2004-02-29Completed
Phase 1b/2, Multicenter, Open-label Study of Oprozomib, Melphalan, and Prednisone in Transplant Ineligible Patients With Newly Diagnosed Multiple Myeloma [NCT02072863]Phase 1/Phase 29 participants (Actual)Interventional2014-01-31Completed
Loncastuximab Tesirine in Combination With BEAM (Carmustine, Etoposide, Ara-C, Melphalan) Conditioning Regimen Prior to Autologous Stem Cell Transplant (ASCT) and for Maintenance Therapy in Diffuse Large B-Cell Lymphoma (DLBCL) [NCT05228249]Phase 10 participants (Actual)Interventional2023-04-30Withdrawn(stopped due to PI left institution and funding sponsor closed study. Study did not open to accrual, and no participants were enrolled.)
Response-based Treatment for Children With Unresectable Localized Soft Tissue Sarcomas [NCT02784015]Phase 241 participants (Anticipated)Interventional2016-05-31Recruiting
MUK Nine b: OPTIMUM. A Phase II Study Evaluating Optimised Combination of Biological Therapy in Newly Diagnosed High Risk Multiple Myeloma and Plasma Cell Leukaemia. [NCT03188172]Phase 295 participants (Anticipated)Interventional2017-09-28Active, not recruiting
Gemcitabine Combined With Busulfan and Melphalan, With Hematopoietic Cell Transplantation, for Patients With Poor-prognosis Advanced Lymphoid Malignancies [NCT00410982]Phase 1145 participants (Actual)Interventional2006-12-31Completed
A Phase II Study of Peripheral Blood Stem Cell Transplantation (PBSCT)From Haploidentical Related Donors for Treatment of Hematologic Malignancies and Hematopoietic Failure States [NCT00618969]Phase 22 participants (Actual)Interventional2008-02-29Completed
Randomised, Non-blind, Parallel Group Study to Compare Tandem High Dose Melphalan (200mg/m²) Versus Triple Intermediate Dose Melphalan (100mg/m²) and Stem Cell Transplantation in Induction Phase and Prednisolone/IFN Versus IFN in Maintenance Therapy in Ne [NCT00205764]Phase 3212 participants (Anticipated)Interventional1999-03-31Completed
A Randomized Phase III Study of the Treatment of Children and Adolescents With Refractory or Relapsed Acute Myeloid Leukemia [NCT00186966]Phase 3394 participants (Actual)Interventional2002-03-31Completed
Treatment Protocol for High-Risk PNET Brain Tumors in Children With Surgery, Sequential Chemotherapy, Conventional and High-Dose With Peripheral Blood Stem Cell Transplantation and Radiation Therapy [NCT00180791]Phase 271 participants (Anticipated)Interventional2002-07-31Recruiting
Phase II Trial of Reduced Intensity Conditioning Hematopoietic Stem Cell Transplantation for Primary Immune Deficiencies, Immune Dysregulatory Syndromes, and Inherited Bone Marrow Failure Syndromes Using Post-Transplant Cyclophosphamide [NCT04232085]Phase 227 participants (Anticipated)Interventional2020-02-12Recruiting
International Collaborative Treatment Protocol for Infants Under One Year With Acute Lymphoblastic or Biphenotypic Leukemia [NCT00550992]445 participants (Anticipated)Interventional2006-01-31Recruiting
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies Using Melphalan for T-Cell Tolerization [NCT01350258]Phase 1/Phase 28 participants (Actual)Interventional2011-04-30Terminated(stopped due to Poor accrual)
PILOT STUDY OF BUTHIONINE SULFOXIMINE (BSO) IN COMBINATION WITH MELPHALAN FOR HIGH RISK NEUROBLASTOMA PATIENTS [NCT00002730]Phase 10 participants Interventional1996-06-30Completed
PHASE III MULTICENTRE TRIAL OF TREATMENT OF NEUROBLASTOMA IN CHILDREN AND ADOLESCENTS [NCT00002802]Phase 3500 participants (Anticipated)Interventional1990-07-31Completed
A Phase II Trial of ICE Chemotherapy Followed by High Dose BEAM Chemotherapy With Autologous Peripheral Blood Progenitor Cell Transplantation in Patients >= 60 Years Old With Refractory or Relapsed Intermediate Grade Non-Hodgkin's Lymphoma [NCT00002982]Phase 20 participants Interventional1997-01-31Completed
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders [NCT00003336]Phase 26 participants (Actual)Interventional1998-01-31Completed
Phase I/II Study of Lenalidomide Maintenance Following BEAM (+/- Rituximab) for Chemo-Resistant or High Risk Non-Hodgkin?s Lymphoma [NCT01035463]Phase 1/Phase 274 participants (Actual)Interventional2009-11-12Completed
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies [NCT01471067]Phase 133 participants (Actual)Interventional2012-07-13Completed
A Phase II Study Evaluating Intravenous Melphalan With Autologous Whole Blood Stem Cell Transplantation Over Three Cycles In Patients With Castration-Resistant Prostate Cancer (MEL-CAP) [NCT01907009]Phase 2/Phase 329 participants (Actual)Interventional2013-01-31Terminated(stopped due to Early termination)
A National, Open-Label, Multicenter, Randomized, Comparative Phase III Study of Induction Treatment With Melphalan/Prednisone/Velcade Versus Thalidomide / Prednisone / Velcade and Maintenance Treatment With Thalidomide / Velcade Versus Prednisone / Velcad [NCT00443235]Phase 3260 participants (Anticipated)Interventional2005-03-31Completed
An Open-Label, Multicenter, Phase 1/2 Dose Escalation Study to Evaluate Safety, Tolerability and Anti-tumor Activity of Systemic ADH-1 in Combination With Normothermic Isolated Limb Infusion of Melphalan in Subjects With Locally Advanced In-Transit Malign [NCT00421811]Phase 1/Phase 256 participants (Anticipated)Interventional2007-04-30Completed
A Phase I Trial of Zevalin Radioimmunotherapy With High-Dose Melphalan and Stem Cell Transplant for Multiple Myeloma [NCT00477815]Phase 130 participants (Actual)Interventional2005-05-31Completed
Phase I Dose Escalation Trial of High Dose Melphalan Conditioning Regimen With Palifermin for Cytoprotection Followed by Autologous Peripheral Blood Stem Cell Transplantation for Multiple Myeloma [NCT00482846]Phase 138 participants (Actual)Interventional2007-06-30Completed
Phase I Clinical Trial of Dose Escalated Bortezomib + ATO (Arsenic Trioxide) + Melphalan as a Conditioning Regimen for Multiple Myeloma [NCT00504101]Phase 10 participants (Actual)Interventional2007-06-30Withdrawn(stopped due to Temporarily closed to accrual pending availablity of drug.)
A Randomised, International, Open-label, Phase II Study of Peripheral Blood Progenitor Cell (PBPC) Mobilization and Engraftment With Pegfilgrastim or Filgrastim for Autologous Transplantation in Patients With Multiple Myeloma (MM) [NCT00526734]Phase 2100 participants (Anticipated)Interventional2006-02-28Recruiting
A Phase I/II Study of Liposomal Doxorubicin (Doxil)/Melphalan/Bortezomib (Velcade) in Relapsed/Refractory Multiple Myeloma [NCT00516191]Phase 1/Phase 20 participants (Actual)Interventional2004-10-31Withdrawn(stopped due to low accrual)
A Phase III, Randomized Study of Daratumumab, Cyclophosphamide, Bortezomib and Dexamethasone (Dara-VCD) Induction Followed by Autologous Stem Cell Transplant or Dara-VCD Consolidation and Daratumumab Maintenance in Patients With Newly Diagnosed AL Amyloid [NCT06022939]Phase 3338 participants (Anticipated)Interventional2023-10-31Not yet recruiting
Acalabrutinib Plus RICE for Patients With Relapsed/Refractory DLBCL Followed by Autologous Hematopoietic Cell Transplantation and Acalabrutinib Maintenance Therapy [NCT03736616]Phase 247 participants (Anticipated)Interventional2019-08-16Recruiting
A Trial Comparing Single Agent Melphalan to Carmustine, Etoposide, Cytarabine and Melphalan (BEAM) as a Preparative Regimen for Patients With Multiple Myeloma Undergoing High Dose Therapy Followed by Autologous Stem Cell Reinfusion [NCT03570983]Phase 2100 participants (Anticipated)Interventional2018-09-05Recruiting
Phase 2 Study of Personalized r-ATG Dosing to Improve Survival Through Enhanced Immune Reconstitution in Pediatric and Adult Patients Undergoing Ex-vivo CD34-Selected Allogeneic-HCT (PRAISE-IR) [NCT04872595]Phase 256 participants (Anticipated)Interventional2021-04-30Recruiting
High-dose Gemcitabine, Busulfan and Melphalan With Autologous Hematopoietic-Cell Support for Patients With Poor-Risk Myeloma [NCT01237951]Phase 275 participants (Actual)Interventional2010-11-08Completed
Reduced Intensity Hematopoietic Cell Transplantation for Patients With Resistant Langerhans Cell Histiocytosis [NCT00618540]Phase 21 participants (Actual)Interventional2007-01-31Terminated(stopped due to Slow accrual)
Cord Blood Ex-Vivo MSC Expansion Plus Fucosylation to Enhance Homing and Engraftment [NCT03096782]Phase 26 participants (Actual)Interventional2017-10-13Completed
High-Dose Melphalan and Autologous Stem Cell Transplantation (HDM/SCT) in Light-Chain Deposition Disease (LCDD) and Immunoglobulin Deposition Disease (IGDD) [NCT00681044]Phase 25 participants (Actual)Interventional2006-10-31Terminated(stopped due to Poor accrual)
An Open Label, Expanded Access Study of Melphalan Chemosaturation With the Delcath System in Patients With Ocular and Cutaneous Melanoma Metastatic to the Liver [NCT01728051]0 participants Expanded AccessNo longer available
The Treatment of Multiple Myeloma Utilizing VBMCP Chemotherapy Alternating With High-Dose Cyclophosphamide and Alpha2b-Interferon Versus VBMCP: A Phase III Study for Previously Untreated Multiple Myeloma [NCT00002556]Phase 3312 participants (Actual)Interventional1994-07-31Completed
Evaluation of Allogeneic Peripheral Blood Stem Cell Transplants From a Related Donor Without Graft-Versus-Host Prophylaxis in Patients With High Risk of Relapse [NCT00003396]Phase 242 participants (Anticipated)Interventional1998-09-30Completed
Autologous Transplantation With and High Dose BCNU and Melphalan Followed by Consolidation With DCEP Plus Taxol/Cisplatin in Patients With Poor Prognosis Low Grade Non-Hodgkin's Lymphoma and Chronic Lymphocyte Leukemia, Who Have Received < or = 12 Months [NCT00003402]Phase 235 participants (Anticipated)Interventional1999-01-31Completed
A Phase III Study of High-Dose Chemotherapy Using Busulfan, Melphalan and Thiotepa Versus Cyclophosphamide,Thiotepa, Carboplatin Followed by Autologous Stem Cell Transplantation in Patients With High-Risk Primary Stage II or III (Non-Inflammatory) Breast [NCT00003972]Phase 3280 participants (Anticipated)Interventional1998-07-31Completed
Treatment of Newly Diagnosed Childhood AML Using a Timed-Sequential Remission Induction and Consolidation Followed by Single Dose Melphalan With Peripheral Stem Cell Rescue: A POG Pilot Study [NCT00004056]Phase 135 participants (Actual)Interventional1999-10-31Completed
A Phase I/II Study of Targeted Radiotherapy Using Holmium-166-DOTMP With Melphalan and Peripheral Blood Stem Cell Transplantation for Treatment of Multiple Myeloma [NCT00004158]Phase 1/Phase 20 participants Interventional1999-06-30Completed
A Phase II Trial of Tandem Transplantation in AL Amyloidosis [NCT00075621]Phase 262 participants (Actual)Interventional2000-08-31Completed
A Phase II/III Study of Immunomodulation After High Dose Myeloablative Therapy With Autologous Stem Cell Rescue for Refractory/Relapsed Hodgkin Disease [NCT00070187]Phase 2/Phase 324 participants (Actual)Interventional2003-11-30Completed
A Phase II Study of Reduced Dose Post Transplantation Cyclophosphamide as GvHD Prophylaxis in Adult Patients With Hematologic Malignancies Receiving HLA-Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation [NCT06001385]Phase 2170 participants (Anticipated)Interventional2023-11-01Not yet recruiting
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis [NCT03118492]Phase 116 participants (Anticipated)Interventional2017-05-24Recruiting
UARK 2008-03 Phase II Trial for Patients Not Qualifying for TT4 and TT5 Protocols Because of Prior Therapy (No Prior Transplant) [NCT00871013]Phase 2160 participants (Anticipated)Interventional2009-03-31Active, not recruiting
A Random-Assignment Study of Hepatic Arterial Infusion of Melphalan With Venous Filtration Via Peripheral Hepatic Perfusion (PHP) (Delcath System) Versus Best Alternative Care for Ocular and Cutaneous Melanoma Metastatic to the Liver [NCT00324727]Phase 393 participants (Actual)Interventional2006-02-28Completed
The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells [NCT00084695]Phase 225 participants (Anticipated)Interventional2003-09-30Recruiting
UARK 2003-18, A Phase II Study of KIR-Ligand Mismatched Haplo-Identical Natural Killer Cells Transfused Before Autologous Stem Cell Transplant in Relapsed Multiple Myeloma [NCT00089453]Phase 110 participants (Actual)Interventional2003-09-30Completed
Haploidentical Donor Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation With a TLI Based Conditioning Regimen in Patients With Hematologic Malignancies [NCT01807611]Phase 282 participants (Actual)Interventional2013-05-16Completed
A Phase II Study Of Hepatic Arterial Infusion Of Melphalan With Venous Filtration Via Peripheral Hepatic Perfusion (PHP) For Unresectable Primary And Metastatic Cancers Of The Liver [NCT00096083]Phase 256 participants (Actual)Interventional2004-09-30Completed
A Phase II Trial of Isolated Hepatic Perfusion (IHP) and Systemic FOLFOX4 for Subjects With Metastatic Unresectable Colorectal Cancers of the Liver With ≥ 40% Hepatic Tumor Burden [NCT00103298]Phase 20 participants Interventional2004-12-31Completed
Reduced Intensity Conditioning Regimen for Haplo-identical Family Donor Stem Cell Transplants for Hematologic Malignancies With Delayed Add-back of Non-alloreactive T Cells [NCT00104975]Phase 120 participants (Anticipated)Interventional2005-02-28Completed
Neuroblastoma Protocol 2005: Therapy for Children With Advanced Stage High-Risk Neuroblastoma [NCT00135135]Phase 223 participants (Actual)Interventional2005-08-31Completed
A Programme of Treatment Development for Older Patients With Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome [NCT00454480]Phase 2/Phase 32,000 participants (Anticipated)Interventional2006-08-31Completed
Cord Blood Expansion on Mesenchymal Stem Cells [NCT00498316]Phase 198 participants (Actual)Interventional2007-07-03Completed
Phase II Study of Sequential Unrelated Cord Blood Transplantation Using Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis [NCT00133367]Phase 232 participants (Anticipated)Interventional2005-08-31Completed
High Dose Chemotherapy With Stem Cell Rescue Followed By Consolidation Treatment in Patients With Metastatic Hormone-Refractory Prostate Cancer [NCT00003400]Phase 245 participants (Anticipated)Interventional1998-09-30Completed
Phase II Multicenter Trial of Single Autologous Hematopoietic Cell Transplant Followed by Lenalidomide Maintenance for Multiple Myeloma With or Without Vaccination With Dendritic Cell/Myeloma Fusions (BMT CTN 1401) [NCT02728102]Phase 2203 participants (Actual)Interventional2016-07-31Completed
A Multimodality Treatment Approach to Patients With Inflammatory Cancer of the Breast and Locally Advanced Non-Inflammatory Stage III Breast Cancer and Stage IV Breast Cancer [NCT00001193]Phase 2200 participants Interventional1984-11-30Completed
Treatment of High Risk, Inherited Lysosomal And Peroxisomal Disorders by Reduced Intensity Hematopoietic Stem Cell Transplantation [NCT00383448]Phase 238 participants (Actual)Interventional2006-09-30Completed
Allogeneic Stem Cell Transplantation Followed By Adoptive Immunotherapy for Patients With Relapsed and Refractory Hodgkin's Disease [NCT00385788]Phase 252 participants (Actual)Interventional2005-07-31Completed
Randomized Trial Using Standard Dose Versus High Dose Rituximab in Addition to Autologous Transplantation With BEAM for Patients With Diffuse Large B Cell Lymphomas [NCT00472056]Phase 293 participants (Actual)Interventional2005-03-31Completed
A Phase II Trial For High-Risk Myeloma Evaluating Accelerating and Sustaining Complete Remission (AS-CR) by Applying Non-Host-Exhausting and Timely Dose-Reduced Mel-80-CFZ-TD-Pace Transplant(s) With Interspersed Mel-20-CFZ-TD-Pace With CFZ-RD and CFZ-D Ma [NCT02128230]Phase 220 participants (Actual)Interventional2014-06-10Terminated(stopped due to Low enrollment and Futility as determined by Amgen's Carfilzomib NASCR program.)
Umbilical Cord Blood Transplantation From Unrelated Donors [NCT03016806]Phase 130 participants (Anticipated)Interventional2015-06-30Recruiting
Tandem Autologous HCT/Nonmyeloablative Allogeneic HCT From HLA-Matched Related and Unrelated Donors Followed by Bortezomib Maintenance Therapy for Patients With High-Risk Multiple Myeloma [NCT00793572]Phase 232 participants (Actual)Interventional2008-10-31Completed
Combination High Dose Melphalan and Autologous PBSC Transplant With Bortezomib for Multiple Myeloma: A Dose and Schedule Finding Study [NCT00793650]Phase 1/Phase 239 participants (Actual)Interventional2005-05-31Terminated
Combined Haploidentical-Cord Blood Transplantation for Adults and Children [NCT00943800]87 participants (Actual)Interventional2006-10-09Completed
A Pilot Trial of Unrelated Donor Hematopoietic Cell Transplantation for Children With Severe Thalassemia Using a Reduced Intensity Conditioning Regimen (The URTH Trial) [NCT01005576]Phase 221 participants (Actual)Interventional2010-01-31Completed
Sequential Autologous HCT / Nonmyeloablative Allogeneic HCT Using Related, HLA-Haploidentical Donors for Patients With High-Risk Lymphoma, Multiple Myeloma, or Chronic Lymphocytic Leukemia [NCT01008462]Phase 216 participants (Actual)Interventional2010-03-18Completed
Autologous Stem Cell Transplant In Patients With Hodgkin Lymphoma (HL) and Non-Hodgkin Lymphomas (NHL) [NCT03125642]Phase 2150 participants (Anticipated)Interventional2017-04-20Recruiting
Trial Protocol for the Treatment of Children With High Risk Neuroblastoma (NB2004-HR) [NCT00526318]360 participants (Anticipated)Interventional2007-01-31Recruiting
Phase II Study to Evaluate the Efficacy of Upfront Obinutuzumab in Mantle Cell Lymphoma Patients Treated by DHAP Followed by Autologous Transplantation Plus Obinutuzumab Maintenance Then MRD Driven Maintenance [NCT02896582]Phase 286 participants (Actual)Interventional2016-10-31Active, not recruiting
A Phase IIb, Multicenter, Open-Label, Safety and Efficacy Study of High Dose Melphalan HCL for Injection (Propylene Glycol-Free)for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Transplantation [NCT01660633]Phase 261 participants (Actual)Interventional2012-12-31Completed
Isolated Pelvic and Limb Perfusion With 1mg TNFa in the Treatment of Locally Advanced Sarcoma of Pelvis and Limbs' Girdle [NCT00181025]Phase 233 participants (Anticipated)Interventional2004-05-31Recruiting
Phase II Trial of Induction Therapy With Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation in Patients With AL Amyloidosis [NCT01083316]Phase 235 participants (Actual)Interventional2009-09-30Completed
A Feasibility Trial of Post-Transplant Infusion of Allogeneic Regulatory T Cells and Allogeneic Conventional T Cells in Patients With Hematologic Malignancies Undergoing Allogeneic Myeloablative Hematopoietic Cell Transplantation From Haploidentical-Relat [NCT01050764]Phase 1/Phase 210 participants (Actual)Interventional2009-06-30Terminated(stopped due to Safety)
Dose Escalation of Total Marrow Irradiation Added to an Alkylator-Intense Conditioning Regimen for Patients With High Risk or Relapsed Solid Tumors [NCT00623077]Phase 123 participants (Actual)Interventional2005-08-31Terminated(stopped due to Replaced by another study)
A Phase I Study of Post-transplant Autologous Cytokine-induced Killer (CIK) Cells for the Treatment of High-risk Hematologic Malignancies [NCT00477035]Phase 122 participants (Actual)Interventional2006-05-31Completed
A Phase I Study of Bendamustine and Melphalan Conditioning and Autologous Stem Cell Transplantation for Treatment of Multiple Myeloma and Relapsed/Refractory B-cell Lymphoma in Elderly Patients [NCT03352765]Phase 128 participants (Anticipated)Interventional2017-11-20Active, not recruiting
Risk-adapted Therapy for AL Amyloidosis [NCT01527032]Phase 20 participants Interventional2002-09-30Completed
Phase II Trial of Second Autologous Transplantation in AL Amyloidosis [NCT00075608]Phase 212 participants (Actual)Interventional2001-08-31Terminated(stopped due to poor accrual)
A Phase I/II Study of Isolated Limb Infusion and Targeted Gene Therapy for Advanced, Unresectable Extremity Melanoma [NCT01531244]Phase 1/Phase 20 participants (Actual)Interventional2014-12-31Withdrawn
Iberoamerican Phase III International Study, Open, Multicenter, Randomized, Comparative of Thalidomide / Cyclophosphamide / Dexamethasone Versus Thalidomide / Dexamethasone Versus Thalidomide / Melphalan / Prednisone as Induction Therapy Followed by Maint [NCT01532856]Phase 364 participants (Actual)Interventional2007-01-31Active, not recruiting
Dose-Intensive Melphalan and Cyclophosphamide With Autologous Bone Marrow Rescue for Recurrent Medulloblastoma and Germ Cell Tumors [NCT00002594]Phase 231 participants (Actual)Interventional1994-09-30Completed
HIGH-DOSE MELPHALAN CHEMOTHERAPY AND TOTAL BODY RADIATION WITH PERIPHERAL BLOOD STEM-CELL RECONSTITUTION FOR PATIENTS WITH RELAPSING MULTIPLE MYELOMA [NCT00002630]Phase 250 participants (Anticipated)Interventional1993-06-30Completed
Randomised Comparisons, in Myeloma Patients of All Ages, of Thalidomide, Lenalidomide, Carfilzomib and Bortezomib Induction Combinations, and of Lenalidomide and Combination Lenalidomide Vorinostat as Maintenance (Myeloma XI) [NCT01554852]Phase 34,420 participants (Actual)Interventional2010-05-31Active, not recruiting
BEAM Plus Iodine-131 Anti-B1 Antibody and Autologous Hematopoietic Stem Cell Transplantation for Treatment of Recurrent Diffuse Large B-Cell Non-Hodgkin's Lymphoma [NCT00006695]Phase 250 participants (Actual)Interventional2000-04-01Completed
Myeloblative Therapy With Autologous Hematopoietic Stem Cell Transplantation in Patients With Multiple Myeloma and B-Cell Malignancies [NCT00003163]Phase 210 participants (Anticipated)Interventional1997-09-30Active, not recruiting
Randomized, Controlled Study to Compare the Efficacy, Safety and Pharmacokinetics of Melphalan/HDS Treatment Given Sequentially Following Cisplatin/Gemcitabine Versus Cisplatin/Gemcitabine in Patients With IntraHepatic Cholangiocarcinoma [NCT03086993]Phase 2/Phase 3295 participants (Anticipated)Interventional2018-04-10Active, not recruiting
A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Pediatric Solid Tumors With Relapsed or Therapy Refractory Disease [NCT00112645]Phase 110 participants (Anticipated)Interventional2005-04-30Completed
A Phase II Study of Isolated Hepatic Perfusion (IHP) With Melphalan for Metastatic Unresectable Cancers of the Liver [NCT00019786]Phase 267 participants (Anticipated)Interventional1999-08-31Completed
European Ewing Tumour Working Initiative of National Groups Ewing Tumour Studies 1999 (EURO-E.W.I.N.G.99) [NCT00020566]Phase 31,200 participants (Anticipated)Interventional2001-02-28Recruiting
Autologous Transplantation for Multiple Myeloma [NCT00177047]Phase 2/Phase 3363 participants (Actual)Interventional2004-04-20Completed
A Study Of The Treatment Of Metastatic Neuroblastoma In Children More Than One Year Of Age At Diagnosis [NCT00024193]Phase 20 participants Interventional1999-04-30Active, not recruiting
A Randomized Trial Of BEAM Plus PBSCT Versus Single Agent High-Dose Therapy Followed By BEAM Plus PBSCT In Patients With Relapsed Hodgkin's Disease [NCT00025636]Phase 3220 participants (Anticipated)Interventional2001-07-31Active, not recruiting
A Phase II Study Of Mobilization Chemotherapy With GMCSF And GCSF Followed By High Dose Therapy Combined With IL2 Activated Autologous Peripheral Blood Stem Cells Followed By Sequential IL2 Therapy As Treatment For Solid Tumors And Lymphoma [NCT00027937]Phase 20 participants Interventional2001-08-31Completed
A Randomised Phase III Study On The Effect Of The Chimeric Anti-CD20 Monoclonal Antibody (Mabthera) During Sequential Chemotherapy Followed By Autologous Stem Cell Transplantation In Patients With Relapse B-Cell Non-Hodgkin Lymphoma(HOVON 44 STUDY) [NCT00012051]Phase 3340 participants (Anticipated)Interventional2000-09-30Completed
Autologous Followed By Non-Myeloablative Allogeneic Transplant For Multiple Myeloma [NCT00028600]Phase 260 participants (Actual)Interventional2001-11-30Completed
A Phase II Trial of IV Busulfan (Busulfex) and Melphalan as a Preparatory Regimen Prior to Allogeneic Bone Marrow Transplantation for the Treatment of Advanced and High Risk Hematologic Malignancies [NCT00014469]Phase 20 participants Interventional2000-12-31Completed
Treatment of Mantle Cell Lymphomas at Advanced Stages: Prospective Randomized Comparison of Myeloablative Radiochemotherapy Followed by Blood Stem Cell Transplantation Versus Maintenance With Interferon Alpha in First Remission After Initial Cytoreductive [NCT00016887]Phase 30 participants Interventional2000-12-31Active, not recruiting
A Multicenter Dosimetry Trial to Evaluate Radiation Absorbed Dose From Holmium-166-DOTMP in Patients With Multiple Myeloma [NCT00045136]Phase 1/Phase 20 participants Interventional2002-01-31Completed
High-Dose Therapy and Autologous Blood Stem Cell Transplantation (ASCT) Followed by Post-Transplant Immunotherapy With Costimulated Autologous T-Cells in Conjunction With Pneumococcal Conjugate Vaccine Immunization for Patients With Multiple Myeloma [NCT00046852]Phase 1/Phase 20 participants Interventional2001-12-31Completed
A Study of Allogeneic Blood Stem Cell Transplantation With Purine Analog-Based Conditioning For Patients With Advanced Hodgkin's Disease [NCT00423709]46 participants (Actual)Interventional1998-01-31Completed
Ibritumomab Tiuxetan and High-Dose Melphalan as Conditioning Regimen Before Autologous Stem Cell Transplantation for Elderly Patients With Lymphoma in Relapse or Resistant to Chemotherapy. A Multicenter Phase I Trial [NCT00392691]Phase 120 participants (Actual)Interventional2006-10-31Completed
Randomized Study of ICE Plus RITUXIMAB Versus DHAP Plus Rituximab in Previously Treated Patients With Diffuse Large B-cell Lymphoma, Followed by Randomized Maintenance With Rituximab [NCT00137995]Phase 3481 participants (Actual)Interventional2003-06-30Completed
A Randomized, Phase III, Placebo-Controlled Multicenter Study to Demonstrate the Effectiveness and Safety of the Combination Enzyme Tablet (Wobe-Mugos E) as Adjuvant Therapy to Standard of Care Treatment in Patients With Stages II or III Multiple Myeloma [NCT00014339]Phase 30 participants Interventional2000-03-31Active, not recruiting
A Pilot Study of Daunorubicin-cytarabine Liposome (CPX-351) Plus FLT3-inhibitor (Midostaurin) as Induction Therapy for Patients With FLT3 Mutated Acute Myeloid Leukemia Followed by Consolidation With a CD34+-Selected Allograft [NCT04982354]Phase 1/Phase 220 participants (Anticipated)Interventional2022-07-05Recruiting
Neuroblastoma Study Phase II Study of Various Therapies in Patients With Neuroblastoma [NCT00017225]Phase 20 participants Interventional1997-05-31Completed
Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donor After Flu-Mel-PTCy Versus Flu-Mel-ATG Reduced-intensity Conditioning: a Phase II Randomized Study From the Belgian Hematology Society (BHS) [NCT03852407]Phase 2114 participants (Anticipated)Interventional2019-02-04Recruiting
A Phase I/II Clinical Trial of Pomalidomide With Melphalan and Dexamethasone in Patients With Newly Diagnosed Untreated Systemic AL Amyloidosis: Trial Stopped During Phase I [NCT01807286]Phase 13 participants (Actual)Interventional2014-01-31Terminated(stopped due to Sponsor requested termination)
Campath 1H (Alemtuzumab) Combined With High-Dose Therapy and Autologous Stem Cell Transplantation in Chronic Lymphocytic Leukemia [NCT00276809]Phase 230 participants (Anticipated)Interventional2001-06-30Completed
Pivotal Study for High Dose Therapy and Autologous Stem Cell Transplantation in Early Stages of CLL [NCT00275015]Phase 2169 participants (Actual)Interventional1998-01-31Completed
High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma: Standard of Care Considerations [NCT01526603]20 participants (Anticipated)Interventional2012-03-28Recruiting
Phase II Study of Autologous Myeloma-Derived Immunoglobulin Idiotype Conjugated to Keyhole Limpet Hemocyanin Plus Sargramostim (GM-CSF) in Patients With Multiple Myeloma Undergoing Second Autologous Peripheral Blood Stem Cell Transplantation [NCT00019097]Phase 20 participants Interventional1995-07-31Completed
A Phase III Prospective Random Assignment Trial of Regional and Systemic Chemotherapy With or Without Initial Isolated Hepatic Perfusion for Patients With Metastatic Unresectable Colorectal Cancers of the Liver [NCT00020501]Phase 30 participants Interventional2001-03-31Completed
Protocol For The Treatment Of Children And Adolescents With Hodgkin's Disease [NCT00025064]Phase 2260 participants (Anticipated)Interventional2000-01-31Active, not recruiting
Protocol For The Treatment Of Relapsed And Refractory Wilms Tumour And Clear Cell Sarcoma Of The Kidney (CCSK) [NCT00025103]Phase 275 participants (Anticipated)Interventional2001-05-31Active, not recruiting
European Infant Neuroblastoma Study - Stage 2, 3, 4, and 4S; MYCN Amplified Tumors [NCT00025649]Phase 20 participants Interventional1999-07-31Completed
Unrelated Umbilical Cord Blood As An Alternate Source Of Stem Cells Transplantation [NCT00055653]Phase 20 participants Interventional2003-01-31Completed
A Randomized Phase III Study On The Effect Of Thalidomide Combined With Adriamycin, Dexamethasone (AD) And High Dose Melphalan In Patients With Multiple Myeloma [NCT00028886]Phase 3450 participants (Anticipated)Interventional2001-03-31Active, not recruiting
High Risk Neuroblastoma Study 1 Of Siop-Europe [NCT00030719]Phase 3175 participants (Anticipated)Interventional2001-12-31Recruiting
Autologous Blood and Marrow Transplantation for Hematologic Malignancy and Selected Solid Tumors [NCT00060255]Phase 2451 participants (Actual)Interventional1991-12-31Completed
A Pilot Induction Regimen Incorporating Topotecan for Treatment of Newly Diagnosed High Risk Neuroblastoma [NCT00070200]Phase 131 participants (Actual)Interventional2004-03-31Completed
Phase I/II Evaluation of Safety and Activity of Mylotarg Plus Melphalan and Fludarabine as Preparative Therapy for Older or Medically Infirm Patients Undergoing Allogeneic Bone Marrow and Peripheral Blood Stem Cell Transplantation [NCT00038831]Phase 1/Phase 247 participants (Actual)Interventional2001-05-31Completed
A Phase I/II, Multi-Center, Open Label Study of Melphalan, Prednisone, Thalidomide and Defibrotide in Advanced and Refractory Multiple Myeloma Patients [NCT00406978]Phase 1/Phase 224 participants (Actual)Interventional2006-02-28Completed
A Phase I Trial Combining IDEC-Y2B8 And High-Dose Beam Chemotherapy With Hematopoietic Progenitor Cell Transplant In Patients With Relapsed Or Refractory B-Cell Non-Hodgkin's Lymphoma [NCT00058292]Phase 144 participants (Actual)Interventional2000-04-30Completed
A Phase II Study of High-Dose Intravenous Busulfan Plus Melphalan With Allogeneic or Autologous Marrow or Peripheral Blood Progenitor Cell Transplantation for Lymphoid Malignancies or Multiple Myeloma [NCT00427765]Phase 2168 participants (Actual)Interventional2004-12-31Completed
High-dose Chemotherapy for Poor-Prognosis Relapsed Germ-Cell Tumors [NCT00936936]Phase 264 participants (Actual)Interventional2009-06-02Active, not recruiting
A Phase I/II Trial of Venetoclax and BEAM Conditioning Followed by Autologous Stem Cell Transplantation for Patients With Primary Refractory Non-Hodgkin Lymphoma [NCT03583424]Phase 1/Phase 219 participants (Actual)Interventional2018-09-10Active, not recruiting
A Randomized Phase II Trial of Fludarabine/Melphalan 140 VS. Fludarabine/Melphalan 100 Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation for Patients With Multiple Myeloma [NCT00505895]Phase 252 participants (Actual)Interventional2002-01-31Completed
Study of Fludarabine Based Conditioning for Allogeneic Stem Cell Transplantation for Myelofibrosis [NCT00572897]Phase 266 participants (Actual)Interventional2007-08-31Completed
Phase 1/2 Investigator Sponsored Study of Selinexor in Combination With High-Dose Melphalan Before Autologous Hematopoietic Cell Transplantation for Multiple Myeloma [NCT02780609]Phase 1/Phase 222 participants (Actual)Interventional2017-07-20Completed
Phase I Study of Escalating Doses of Total Marrow and Lymphoid Irradiation (TMLI) Combined With Fludarabine and Melphalan as Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients With High-Risk Acute Leukemia or Myelodysplastic Syndro [NCT03494569]Phase 136 participants (Anticipated)Interventional2018-07-06Recruiting
A Comparison of Reduced Dose Total Body Irradiation (TBI) and Cyclophosphamide With Fludarabine and Melphalan Reduced Intensity Conditioning in Adults With Acute Lymphoblastic Leukaemia (ALL) in Complete Remission. (ALL-RIC) [NCT03821610]Phase 2242 participants (Anticipated)Interventional2018-11-22Active, not recruiting
Transplantation of Two Partially Matched Umbilical Cord Blood Units Following Reduced Intensity Conditioning to Enhance Engraftment and Limit Transplant-Related Mortality in Adults With Hematologic Malignancies [NCT00827099]Phase 25 participants (Actual)Interventional2006-06-30Terminated(stopped due to Unacceptable morbidity & mortality)
"A Feasibility Study of Early Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms" [NCT02756572]Phase 230 participants (Actual)Interventional2016-09-22Completed
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis [NCT04384692]Phase 245 participants (Anticipated)Interventional2020-12-18Recruiting
Olaparib Combined With High-Dose Chemotherapy for Refractory Lymphomas [NCT03259503]Phase 150 participants (Actual)Interventional2019-09-13Active, not recruiting
CNCT19 Following Autologous Stem Cell Transplantation in Patients With Relapsed or Refractory Aggressive B-cell Lymphoma [NCT04690192]Phase 1/Phase 220 participants (Anticipated)Interventional2021-01-01Recruiting
Phase I Clinical Trial Using an Engineered Peripheral Blood Graft for Haploidentical Transplantation [NCT02960646]Phase 111 participants (Actual)Interventional2017-01-18Completed
A Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory Regime [NCT01821781]Phase 220 participants (Anticipated)Interventional2013-03-31Recruiting
Intra-arterial Chemotherapy for the Treatment of Progressive Diffuse Intrinsic Pontine Gliomas (DIPG). [NCT01688401]Phase 13 participants (Actual)Interventional2013-03-08Completed
Immune Consolidation With Activated T Cells Armed With OKT3 x Rituxan (Anti-CD3 x Anti-CD20) Bispecific Antibody (CD20Bi) After Peripheral Blood Stem Cell Transplant for High Risk CD20+ Non-Hodgkin's Lymphomas [NCT00244946]Phase 115 participants (Actual)Interventional2004-03-31Completed
Intra-Arterial Melphalan (L-Phenylalanine Mustard) Administered in Conjunction With Osmotic Blood-Brain Barrier Disruption in Patients With Brain Malignancies: A Phase I Study [NCT00253721]Phase 121 participants (Actual)Interventional1998-05-31Terminated(stopped due to Other competing clinical trials affecting enrollment)
Transplantation of Umbilical Cord Blood From Related and Unrelated Donors [NCT00290628]43 participants (Actual)Interventional1999-10-31Terminated(stopped due to Replaced with another study)
Feasibility of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation Followed by Donor Lymphocyte Infusions for Children at High Risk for Complications With Conventional Transplantation [NCT00301860]8 participants (Actual)Interventional2003-01-31Terminated(stopped due to lack of efficacy)
European Infant Neuroblastoma Study Final Protocol [NCT00417053]Phase 30 participants InterventionalActive, not recruiting
A Phase II Trial of Bortezomib + Ascorbic Acid + Melphalan (BAM) Combination Therapy for Patients With Newly Diagnosed Multiple Myeloma [NCT00317811]Phase 235 participants (Anticipated)Interventional2005-11-30Completed
Treatment in First Line of Mantle Cell Lymphoma for Patients Under 66 Years by the VAD-CHLORAMBUCIL -Rituximab Regimen Followed by Intensification and Autologous PBSC Transplantation After Marrow Purging With Rituximab [NCT00285389]Phase 239 participants (Actual)Interventional2002-02-28Completed
2015-12: A Phase II Study Exploring the Use of Early and Late Consolidation/Maintenance With Anti-CD38 (Protein) Monoclonal Antibody to Improve Progression Free Survival in Patients With Newly Diagnosed Multiple Myeloma [NCT03004287]Phase 250 participants (Anticipated)Interventional2017-07-01Active, not recruiting
Phase I/II Study of Liposomal Doxorubicin (Doxil®)/ Melphalan/Bortezomib (Velcade®) in Relapsed/Refractory Multiple Myeloma [NCT00334932]Phase 1/Phase 232 participants (Anticipated)Interventional2006-02-28Recruiting
A Phase II Study of Intravenous Melphalan and Busulfan Followed by HLA-Matched, Allogeneic Peripheral Blood Stem Cell Transplant for the Treatment of Multiple Myeloma [NCT00313625]Phase 220 participants (Anticipated)Interventional2005-09-30Completed
Autologous Stem Cell Transplantation (ASCT) Versus Oral Melphalan and High-Dose Dexamethasone in Patients With AL (Primary)Amyloidosis. A Prospective Randomized Trial . [NCT00344526]Phase 3100 participants Interventional2000-01-31Completed
A Phase II Study Of Pooled Unrelated Donor Umbilical Cord Blood (UCB) Transplant For Patients With Hematologic Malignancies Needing Allogeneic Stem Cell Transplant But Do Not Have A Related HLA-Matched Donor [NCT01500161]Phase 21 participants (Actual)Interventional2011-11-30Terminated(stopped due to Insufficient accruals)
A Phase I Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas [NCT01625351]Phase 123 participants (Actual)Interventional2012-08-20Completed
Selective Intra-arterial Chemotherapy in the Treatment Strategy of Metastatic Spinal Disease [NCT01637766]Phase 112 participants (Actual)Interventional2012-04-30Completed
Use of Umbilical Cord Blood Cell in the Preparative Regimen of Patients With Advanced Hematologic Malignancies Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT00427557]Phase 231 participants (Actual)Interventional2006-10-31Completed
Standard Chemotherapy (CHOP Regimen) Versus Sequential High-Dose Chemotherapy With Autologous Stem Cell Transplantation in Patients With Newly Diagnosed Aggressive Non-Hodgkin's Lymphomas and Poor Prognostic Factors: A Randomized Phase III Study (MISTRAL) [NCT00003215]Phase 3400 participants (Anticipated)Interventional1997-04-30Completed
Combined T Cell Depleted Haploidentical Peripheral Blood Stem Cell and Unrelated Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies Using a Total Lymphoid Irradiation Based Preparative Regimen [NCT02199041]Phase 224 participants (Actual)Interventional2014-07-11Terminated(stopped due to The study was halted early due to slow accrual.)
High Risk Neuroblastoma Study 1 of SIOP-Europe (SIOPEN) [NCT01704716]Phase 33,300 participants (Anticipated)Interventional2002-02-28Recruiting
MRD-Guided Sequential Therapy For Deep Response in Newly Diagnosed Multiple Myeloma - MASTER-2 Trial [NCT05231629]Phase 2300 participants (Anticipated)Interventional2023-12-13Recruiting
CD19-specific T Cell Infusion in Patients With B-Lineage Lymphoid Malignancies [NCT00968760]Phase 134 participants (Actual)Interventional2011-06-20Completed
A Multicenter Phase II Study of Subcutaneous Velcade Plus Oral Melphalan and Prdnisone or Plus Cycloposphamide and Prednisone or Plus Prednisone in Newly Diagnosed Elderly Multiple Myeloma Patients [NCT01190787]Phase 2150 participants (Anticipated)Interventional2010-07-31Active, not recruiting
AUTOLOGOUS TRANSPLANTATION AND STEM CELL BASED-GENE THERAPY FOR THE TREATMENT OF HIV-ASSOCIATED LYMPHOMA [NCT01769911]0 participants (Actual)Interventional2015-02-28Withdrawn
UARK 2008-02, A Phase II Trial for High-risk Myeloma Evaluation Accelerating and Sustaining Complete Remission (AS-CR) by Applying Non-host-exhausting and Timely Dose-reduced MEL-80-VRD-PACE Tandem Transplants [NCT00869232]Phase 290 participants (Actual)Interventional2008-10-31Active, not recruiting
Phase III-study for Evaluation of Induction Therapy Before Stem Cell Mobilization and Tandem High-dose Melphalan in Multiple Myeloma Patients 60 to 70 Years of Age [NCT02288741]Phase 3549 participants (Actual)Interventional2001-08-31Completed
Tandem Autotransplantation for Multiple Myeloma in Participants With Less Than 12 Months of Preceding Therapy, Incorporating Velcade (Bortezomib) With the Transplant Chemotherapy and During Maintenance [NCT01548573]Phase 219 participants (Actual)Interventional2012-05-31Terminated(stopped due to Met study stopping rules)
Intra-arterial Chemotherapy for Retinoblastoma (IAC) [NCT04342572]Phase 15 participants (Actual)Interventional2020-08-11Active, not recruiting
Autologous Blood and Marrow Transplantation for Hematologic Malignancies and Selected Solid Tumors [NCT00536601]174 participants (Actual)Interventional2006-06-29Completed
2015-10: A Phase II Pilot Study of Expanded Natural Killer Cells and Elotuzumab to Eradicate High-Risk Myeloma Post Autologous Stem Cell Transplant [NCT03003728]Phase 20 participants (Actual)Interventional2019-11-01Withdrawn(stopped due to Withdrawal of study support)
Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT) [NCT01626092]3 participants (Actual)Interventional2012-07-11Completed
Phase II Study Assessing the Efficacy and Toxicity of PK--directed Intravenous Busulfan in Combination With High--Dose Melphalan and Bortezomib as Conditioning Regimen for First--Line Autologous Hematopoietic Stem Cell Transplantation in Patients With Mul [NCT01605032]Phase 219 participants (Actual)Interventional2012-02-29Completed
Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation [NCT02007863]2 participants (Actual)Interventional2008-08-31Completed
SPINOZA / שפינוזה. Study With Preparatory INduction Of Zevalin in Aggressive Lymphoma. A Randomized Phase 3 Study of BEAM Versus 90Yttrium Ibritumomab Tiuxetan (Zevalin) / BEAM in Patients Requiring ASCT for Relapsed DLBCL [NCT02366663]Phase 33 participants (Actual)Interventional2015-01-31Terminated(stopped due to Withdrawal of sponsor support)
A Phase I-II Study of Infusional Melphalan + Bortezomib for Myeloablative Therapy Prior to Autologous Transplant for Patients With Multiple Myeloma [NCT02353572]Phase 1/Phase 23 participants (Actual)Interventional2009-11-30Terminated(stopped due to Principle investigator left the institution)
A Randomized Study of Combined Haplo-identical Umbilical Cord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation in Patients With Hematologic Malignancies [NCT01745913]Phase 22 participants (Actual)Interventional2012-10-26Terminated(stopped due to Slow accrual)
Biparental HLA Haplotype Disparate T-cell Depleted Transplants for Patients Lacking an HLACompatible Donor [NCT01598025]3 participants (Actual)Interventional2012-05-02Terminated(stopped due to Closed due to poor accrual)
Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation With Bevacizumab for Advanced Solid Tumor [NCT00523809]Phase 25 participants (Actual)Interventional2007-08-31Terminated(stopped due to Slow accrual.)
A Phase I Study of Isolated Hepatic Portal and Arterial Perfusion (IHP) With Escalating Dose Melphalan for Primary or Metastatic Unresectable Cancers of the Liver [NCT00001587]Phase 130 participants Interventional1997-09-30Completed
A Phase Ib/2 Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell Transplantation [NCT03192397]Phase 1/Phase 235 participants (Actual)Interventional2017-08-09Active, not recruiting
Modulation of Intensive Melphalan (L-PAM) by Buthionine Sulfoximine (BSO) Autologous Stem Cell Support for Resistant or Recurrent High-Risk Neuroblastoma (IND 69-112) [NCT00005835]Phase 131 participants (Actual)Interventional2001-08-31Completed
A Phase I/II Study of a Novel Reduced Intensity Conditioning Regimen for Allogeneic Stem Cell Transplantation in Patients With Multiple Myeloma [NCT00995059]Phase 1/Phase 20 participants (Actual)InterventionalWithdrawn
Phase I-II Single Cycle Melphalan/Total Marrow Irradiation (TMI) and Autologous Stem Cell Transplantation (ASCT) Followed by Maintenance in Patients With High-Risk Myeloma and/or Poor Response to Induction Therapy Within 12 Months of Diagnosis [NCT03100877]Phase 1/Phase 20 participants (Actual)Interventional2018-01-31Withdrawn(stopped due to Feasibility Issues)
Reduced Intensity Matched Sibling Bone Marrow Transplantation for Sickle Cell Anemia in Patients 2-30 Years Old [NCT01877837]Phase 330 participants (Actual)Interventional2011-06-30Completed
High-Dose Immunosuppressive Therapy Using Carmustine, Etoposide, Cytarabine, and Melphalan (BEAM) + Thymoglobulin Followed by Syngeneic or Autologous Hematopoietic Cell Transplantation for Patients With Autoimmune Neurologic Diseases [NCT00716066]Phase 280 participants (Anticipated)Interventional2008-06-30Recruiting
A Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Matched Unrelated Donors in Patients With Advanced Hematologic Malignancies and Hematological Disorders [NCT00544115]Phase 2260 participants (Actual)Interventional2001-10-16Active, not recruiting
A Phase II Trial of Thymoglobulin and Melphalan in Patients With Relapsed Multiple Myeloma [NCT00635024]Phase 21 participants (Actual)Interventional2008-05-31Terminated(stopped due to Due to competing trials, this study is permanenlty closed to patient acrrual.)
Randomized Trial of Activated Marrow Infiltrating Lymphocytes Alone or in Conjunction With an Allogeneic GM-CSF-based Myeloma Cellular Vaccine in the Autologous Transplant Setting in Multiple Myeloma [NCT01045460]Phase 236 participants (Actual)Interventional2010-01-15Completed
Transplantation Of Umbilical Cord Blood From Unrelated Donors In Patients With Haematological Diseases Using A Myeloablative Conditioning Regimen [NCT02310997]Phase 211 participants (Actual)Interventional2011-07-31Terminated(stopped due to Trial closed early due to poor recruitment)
Phase II Study of Revlimid (Lenalidomide), Melphalan, and Dexamethasone (ReMeDex) for Newly Diagnosed Multiple Myeloma Patients Not Undergoing Autologous Transplantation [NCT00843310]Phase 28 participants (Actual)Interventional2008-11-30Terminated(stopped due to Due to slow accrual)
A Phase II Study of High-Dose Melphalan With Hematopoietic Stem Cell Reconstitution for Patients With Primary Systemic Amyloidosis [NCT00003353]Phase 20 participants Interventional1998-11-16Completed
Phase I/II Study of Liposomal Doxorubicin (Doxil®)/Melphalan/Bortezomib (Velcade®) in Relapsed/Refractory Multiple Myeloma [NCT00985907]Phase 1/Phase 213 participants (Actual)Interventional2004-10-28Terminated(stopped due to Low Accrual)
Standard Dose Versus Myeloablative Therapy for Previously Untreated Symptomatic Multiple Myeloma, A Phase III Intergroup Study [NCT00002548]Phase 3899 participants (Actual)Interventional1994-01-31Completed
Phase II Trial of Sequential High-Dose Alkylating Agents in Metastatic Breast Cancer [NCT00002680]Phase 240 participants (Actual)Interventional1994-02-28Completed
Phase 2 Multi-center Study of Anti-Programmed-Death-1 [Anti-PD-1] During Lymphopenic State After High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplant [HDT/ASCT] for Multiple Myeloma [NCT02331368]Phase 232 participants (Actual)Interventional2015-06-30Completed
Phase I Study of Yttrium-90 Labeled Anti-CD25 Monoclonal Antibody Plus Standard BEAM Conditioning for Autologous Hematopoietic Cell Transplantation in Patients With Mature T-Cell Non-Hodgkin Lymphoma: the aTAC BEAM Regimen [NCT02342782]Phase 120 participants (Actual)Interventional2020-06-08Active, not recruiting
A Phase II Trial of The Addition of Ipilimumab (MDX-010) To Isolated Limb Infusion (ILI) With Standard Melphalan and Dactinomycin In The Treatment of Advanced Unresectable Melanoma of The Extremity [NCT01323517]Phase 226 participants (Actual)Interventional2011-02-28Completed
Allogeneic Stem Cell Transplant With a Novel Conditioning Therapy Using Helical Tomotherapy, Melphalan, and Fludarabine in Hematological Malignancies [NCT00544466]Phase 1/Phase 275 participants (Actual)Interventional2006-07-31Active, not recruiting
University of Arkansas (UARK 2001-12), A Phase III Study of DTPACE Followed by Tandem Transplant With MEL 200 Versus MEL/DTPACE Hybrid and DTPACE Consolidation in Patients With Active Multiple Myeloma [NCT00083915]Phase 397 participants (Actual)Interventional2001-06-30Completed
High-dose 131I-MIBG Treatment Incorporated Into Tandem High-dose Chemotherapy and Autologous Stem Cell Transplantation in Patients With High-risk Neuroblastoma [NCT03061656]Phase 240 participants (Anticipated)Interventional2009-01-01Active, not recruiting
Hepatic Arterial Infusion of Autologous Tumor Infiltrating Lymphocytes Preconditioned With Percutaneous Hepatic Perfusion With Melphalan in Patients With Melanoma and Liver Metastases [NCT05903937]Phase 16 participants (Anticipated)Interventional2023-12-31Not yet recruiting
A Phase II Single-Center, Open-Label, Safety and Efficacy Study of Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients Undergoing Autologous Stem Cell Transplantation [NCT02994784]Phase 227 participants (Actual)Interventional2018-01-08Terminated(stopped due to Slow Accrual during COVID pandemic, alternative treatments available. Study halted early.)
Phase II Trial of Lymphodepletion and Anti-PD-1 Blockade to Reduce Relapse in High Risk AML Patients Who Are Not Eligible for Allogeneic Stem Cell Transplantation [NCT02771197]Phase 220 participants (Actual)Interventional2016-09-28Completed
A Phase 1/2 Study of Vadastuximab Talirine Administered in Sequence With Allogeneic Hematopoietic Stem Cell Transplant in Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) [NCT02614560]Phase 1/Phase 214 participants (Actual)Interventional2015-11-30Terminated
A Phase II Study of Busulfan & Melphalan as Conditioning Regimen for ASCT in Patients Who Received Bortezomib Based Induction for Newly Diagnosed Multiple Myeloma Followed by Lenalidomide Maintenance Until Progression. [NCT01702831]Phase 278 participants (Actual)Interventional2013-10-01Completed
A Phase II Multicenter Study of Carfilzomib, Lenalidomide and Dexamethasone (KRd) as Induction Therapy, Followed by High-dose Therapy With Melphalan and Autologous Peripheral Blood Stem Cell Transplantation, Consolidation With KRd, and Maintenance With Le [NCT02415413]Phase 290 participants (Anticipated)Interventional2015-05-31Active, not recruiting
A Phase I/II Study of Escalating Doses of Bortezomib in Conjunction With High Dose Melphalan as a Conditioning Regimen for Autologous Peripheral Blood Stem Cell Transplantation in Patients With Multiple Myeloma [NCT00784823]Phase 1/Phase 232 participants (Actual)Interventional2007-01-31Completed
Busulfan, Cyclophosphamide, and Melphalan Followed by Allogeneic Hematopoietic Cell Transplantation in Patients With Hematological Malignancies [NCT00176839]Phase 2/Phase 311 participants (Actual)Interventional2000-06-07Terminated(stopped due to Replaced by a different study)
UARK 98-026, Total Therapy II - A Phase III Study for Newly Diagnosed Multiple Myeloma Evaluating Anti-Angiogenesis With Thalidomide and Post-Transplant Consolidation Chemotherapy [NCT00083551]Phase 3668 participants (Actual)Interventional1998-08-31Completed
ChiCGB Versus BEAM With Autologous Stem-Cell Transplantation in High-risk Hodgkin and Non-Hodgkin Lymphoma - A Prospective, Multi-centered, Randomized Clinical Trial [NCT05466318]Phase 3306 participants (Anticipated)Interventional2022-07-01Recruiting
A Phase II Trial Of Thalidomide/Dexamethasone Induction Followed By Tandem Melphalan Transplant And Prednisone/Thalidomide Maintenance (A BMT Study) [NCT00040937]Phase 2147 participants (Actual)Interventional2002-06-30Completed
Reduced-Intensity Allogeneic HSC Transplantation From HLA-Matched Related and Unrelated Donors for Patients With Multiple Myeloma - A Multi-Center Trial [NCT00054353]Phase 1/Phase 216 participants (Actual)Interventional2002-10-31Completed
I-Metaiodobenzylguanidine (MIBG) With Intensive Chemotherapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma [NCT00253435]Phase 250 participants (Actual)Interventional2005-09-30Completed
Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases [NCT00038857]Phase 229 participants (Actual)Interventional2001-09-30Completed
Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia [NCT00003816]Phase 2/Phase 3361 participants (Actual)Interventional1998-10-19Completed
Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation [NCT02790515]Phase 252 participants (Anticipated)Interventional2016-07-14Recruiting
A Pilot Study of Post-transplant High Dose Cyclophosphamide (PTCY) as Part of Graft-Versus-Host Disease (GVHD) Prophylaxis in T-Cell Replete HLA-Mismatched Unrelated Donor (MMUD) Ablative and Reduced Intensity Hematopoietic Cell Transplantation (HCT) for [NCT03128359]Phase 238 participants (Actual)Interventional2017-05-30Active, not recruiting
Intravitreal Injections of Melphalan for Retinoblastoma [NCT01558960]10 participants (Anticipated)Interventional2012-03-31Terminated(stopped due to Sufficient findings to draw conclusions)
A Multicenter, Single Arm, Open Label Study of Autologous Stem Cell Transplantation With High Dose Melphalan in Patients With Multiple Myeloma [NCT01572688]60 participants (Anticipated)Interventional2011-11-30Recruiting
A Phase II Study of CHOEP Induction Followed by Gemcitabine/Busulfan/Melphalan Autologous Stem Cell Transplantation for Patients With Newly Diagnosed T-Cell Lymphoma [NCT01746173]Phase 25 participants (Actual)Interventional2013-07-31Terminated(stopped due to Slow accrual and futility)
Autologous Peripheral Blood Stem Cell Transplantation and Maintenance Lenalidomide After High-dose Melphalan for Multiple Myeloma [NCT01617213]Phase 21 participants (Actual)Interventional2012-04-30Terminated(stopped due to Study is no longer needed as recent data have answered the primary hypotheses for this study.)
A Randomized Trial to Compare Busulfan + Melphalan 140 mg/m2 With Melphalan 200 mg/m2 as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation for Multiple Myeloma [NCT01413178]Phase 3205 participants (Actual)Interventional2011-09-30Completed
Phase III Study on Efficacy of Dose Intensification in Patients With Non-metastatic Ewing Sarcoma. [NCT02063022]Phase 3278 participants (Actual)Interventional2009-01-22Completed
Pilot Study of High-Dose Chemotherapy With Busulfan, Melphalan, and Topotecan Followed by Autologous Hematopoietic Stem Cell Transplant in Advanced Stage and Recurrent Tumors [NCT00638898]Phase 125 participants (Actual)Interventional2007-02-26Active, not recruiting
A Phase II Study of Thiotepa Added to Fludarabine and Melphalan as the Preparative Regime for Alternative Donor Transplantation [NCT03342196]Phase 240 participants (Actual)Interventional2018-03-21Active, not recruiting
Treatment Protocol for Relapsed Anaplastic Large Cell Lymphoma of Childhood and Adolescence [NCT00317408]96 participants (Anticipated)Interventional2004-04-30Active, not recruiting
A Phase I/II Clinical Study of JNJ-26866138 (Bortezomib) in Untreated Multiple Myeloma Patients Who Are Not Candidates for Hematopoietic Stem Cell Transplant (HSCT) [NCT00985959]Phase 1101 participants (Actual)Interventional2008-07-31Completed
Lenalidomide Combined With Vorinostat/Gemcitabine/Busulfan/Melphalan With Autologous Stem-Cell Transplantation in Diffuse Large B-Cell Lymphoma of the ABC Subtype [NCT02589145]Phase 1/Phase 28 participants (Actual)Interventional2016-06-22Terminated(stopped due to Closed due to very slow accrual)
Comparative Study of Dexamethasone vs Prednisone (Both in Combination With Melphalan) as Induction Therapy in Untreated Symptomatic Myeloma With an Additional Assessment of Dexamethasone vs no Additional Treatment as Maintenance Therapy in Non-Progressing [NCT00002678]Phase 3595 participants (Actual)Interventional1995-06-02Completed
Phase I of Intrathecal Melphalan in Patients With Recurrent Neoplastic Meningitis [NCT00002750]Phase 16 participants (Anticipated)Interventional1992-12-31Completed
Double Umbilical Cord Blood Transplantation for Patients With Malignant and Non-Malignant Disorders [NCT00801931]Phase 1/Phase 21 participants (Actual)Interventional2007-09-06Terminated(stopped due to Poor accrual)
Clinical Study of Safety and Tolerability of Melphalan Hydrochloride for Injection in the Treatment of Relapsed or Relapsed and Refractory Multiple Myeloma [NCT05438394]Phase 124 participants (Anticipated)Interventional2022-10-13Recruiting
A Phase I Trial of Sequential High Dose Chemotherapy Regimens Followed by Autologous or Syngeneic Peripheral Blood Stem Cell (PBSC) Rescue in Patients With Persistent Stage III/IV Ovarian Cancer [NCT00003080]Phase 10 participants Interventional1996-09-30Completed
Phase I/II Study of Samarium 153 as Part of a Double (Sequential) Autologous Bone Marrow Transplant (ABMT) for Patients With Stage IV Breast Cancer [NCT00003086]Phase 1/Phase 212 participants (Anticipated)Interventional1997-03-31Terminated(stopped due to no participants enrolled in a three year period)
Autologous Peripheral Blood Stem Cell Transplantation With High Dose Melphalan For Treatment Of Primary Amyloidosis (AL) [NCT00002810]Phase 20 participants Interventional1996-05-31Completed
Multiple Cycles of High Dose Chemotherapy Supported With Filgrastim and Peripheral Blood Progenitor Cells in Patients With Metastatic Breast Cancer [NCT00003392]Phase 261 participants (Actual)Interventional1997-09-30Completed
MYELOMA VII MEDICAL RESEARCH COUNCIL WORKING PARTY ON LEUKEMIA IN ADULTS: MYELOMATOSIS THERAPY TRIAL [NCT00002599]Phase 3750 participants (Anticipated)Interventional1994-09-30Active, not recruiting
VIIITH MYELOMATOSIS TRIAL: A RANDOMISED TRIAL OF TREATMENT FOR INDUCING FIRST PLATEAU PHASE ABCM VS 3 COURSES OF ABCM FOLLOWED BY ORAL WEEKLY CYCLOPHOSPHAMIDE [NCT00002653]Phase 31,000 participants (Anticipated)Interventional1993-09-30Active, not recruiting
Rituxan in the Management of Multiple Myeloma [NCT00003554]Phase 20 participants Interventional1998-11-30Completed
A Randomized Trial to Evaluate Early High Dose Therapy and Autologous Bone Marrow Transplantation as Part of Planned Initial Therapy for Poor Risk Intermediate/High Grade NHL [NCT00003578]Phase 3500 participants (Anticipated)Interventional1993-01-31Active, not recruiting
A Phase II Study of Intensive Methotrexate and Cytarabine Followed by High Dose Beam Chemotherapy With Autologous Peripheral Blood Progenitor Cell Transplantation in Patients With Newly Diagnosed Primary Central Nervous System Lymphoma [NCT00003632]Phase 230 participants (Anticipated)Interventional1998-09-30Completed
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Adults and Children With Bone Marrow Failure Syndromes or Inherited Metabolic or Hematologic Diseases [NCT00003662]Phase 290 participants (Anticipated)Interventional1998-08-31Completed
A Phase I Study of the Chemoprotectant Amifostine With Autologous Stem Cell Transplantation for High Risk or Relapsed Pediatric Solid Tumors and Brain Tumors [NCT00003926]Phase 113 participants (Actual)Interventional1998-11-30Terminated(stopped due to Withdrawn due to slow accrual)
A Study of Intensive-Dose Melphalan, Topotecan, and VP-16 Phosphate (MTV) Followed by Autologous Stem Cell Rescue in Patients With Multiple Myeloma [NCT00005792]Phase 1131 participants (Actual)Interventional1998-06-02Completed
A Phase I Trial of Melphalan and Thiotepa Followed by Autologous or Syngeneic Peripheral Blood Stem Cell (PBSC) Rescue in Patients With a Complete Response Following Standard Therapy for Stage III/IV Epithelial Ovarian Cancer [NCT00002977]Phase 145 participants (Anticipated)Interventional1997-01-31Completed
Autologous Stem Cell Transplantation for Poor Prognosis, Relapsed, or Refractory Intermediate-High Grade B-Cell Lymphoma Using Gemcitabine Plus High Dose BCNU and Melphalan Followed by Anti-CD20 Moab (IDEC C2B8, Rituximab, Rituxan) and Consolidative Chemo [NCT00003397]Phase 225 participants (Anticipated)Interventional1998-09-30Completed
A Randomized Phase III Study to Assess Intensification of the Conditioning Regimen for Allogenic Stem Cell Transplantation (ALLO-SCT) for Leukemia or Myelodysplastic Syndrome With a High Risk of Relapse [NCT00002989]Phase 3207 participants (Anticipated)Interventional1997-03-31Active, not recruiting
Interferon Maintenance in Advanced Multiple Myeloma After Using High-Dose Melphalan as Myeloablative Chemotherapy: A Pilot Study [NCT00003007]Phase 20 participants Interventional1996-07-31Completed
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Children and Adults With Hematologic Malignancies [NCT00003661]Phase 23 participants (Actual)Interventional1998-06-30Completed
Autotransplantation for Chronic Myelogenous Leukemia (CML) Followed by Immunotherapy With Ex-Vivo Expanded Autologous T Cells [NCT00003727]Phase 222 participants (Anticipated)Interventional1999-03-31Completed
A Phase II Study of High Dose Late Intensification Therapy in Patients With Chemotherapy Sensitive Multiple Myeloma [NCT00004903]Phase 20 participants Interventional1999-10-31Active, not recruiting
A Randomized Phase III Trial of Sequential High Dose Chemotherapy or Standard Chemotherapy for Optimally Debulked FIGO Stage III and IV Ovarian Cancer [NCT00004921]Phase 30 participants Interventional1998-09-30Completed
Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies [NCT00281879]Phase 2200 participants (Actual)Interventional2006-02-28Terminated
Pilot Study of a Double Isolation Perfusion Schedule Using Melphalan Alone for Intransit Melanoma or Unresectable Sarcoma of the Extremity [NCT00001577]Phase 130 participants Interventional1997-06-30Completed
High-Dose Cytarabine and Idarubicin Induction, High Dose Etoposide and Cyclophosphamide Intensification, Autologous Stem Cell Transplantation and Interleukin-2 Immune Modulation in Previously Untreated De Novo and Secondary Adult Myeloid Leukemia [NCT00002945]Phase 361 participants (Actual)Interventional1996-12-31Completed
Phase II Evaluation of IV Melphalan (L-PAM) and Whole Body Hyperthermia (WBH) for Malignant Melanoma [NCT00002973]Phase 234 participants (Anticipated)Interventional1995-12-31Completed
A Phase I Trial of Busulfan, Thiotepa and Melphalan Followed by Autologous or Syngeneic Peripheral Blood Stem Cell Transplantation and Followed by Total Marrow (Skeletal) Irradiation (TMI) in Patients With High-Risk Ewing's Sarcoma, PNET or Rhabdomyosarco [NCT00003081]Phase 116 participants (Anticipated)Interventional1998-03-31Completed
A Phase II, Open-Label, Pharmacokinetic Study of Propylene Glycol-Free Melphalan HCl for Myeloablative Conditioning in Multiple Myeloma Patients Undergoing Autologous Transplantation [NCT02669615]Phase 224 participants (Actual)Interventional2016-11-01Completed
A Phase II Study of Melphalan HCl for Injection (Propylene Glycol-free), Combined With Carmustine, Etoposide, and Cytarabine (BEAM Regimen) for Myeloablative Conditioning in Lymphoma Patients Undergoing Autologous Stem Cell Transplantation [NCT01969435]Phase 250 participants (Actual)Interventional2014-03-19Completed
Autologous Transplantation With High Dose BCNU and Melphalan Followed by Consolidation With DCEP and Taxol/Cisplatin in Patients With Multiple Myeloma and < or = 12 Months of Standard Therapy [NCT00003399]Phase 20 participants Interventional1998-09-30Completed
Safety and Efficacy Study of KL-7SHRNA Injection Solution in the Treatment of AIDS Patients With Lymphoma [NCT05922384]3 participants (Anticipated)Interventional2023-07-05Recruiting
A RANDOMIZED, MULTICENTER, OPEN LABEL STUDY COMPARING TWO STANDARD TREATMENTS, BORTEZOMIB-MELPHALAN-PREDNISONE (VMP) WITH OR WITHOUT DARATUMUMAB (Dara-VMP) VS LENALIDOMIDE-DEXAMETHASONE (Rd) WITH OR WITHOUT DARATUMUMAB (Dara-Rd) IN AUTOLOGOUS STEM CELL TR [NCT03829371]Phase 4450 participants (Anticipated)Interventional2019-01-03Recruiting
A Randomized, Controlled, Open-label, Phase 3 Study of Melflufen/Dexamethasone Compared With Pomalidomide/Dexamethasone for Patients With Relapsed Refractory Multiple Myeloma Who Are Refractory to Lenalidomide [NCT03151811]Phase 3495 participants (Actual)Interventional2017-06-12Completed
A Multicenter, Open Label Study Of Oral Revlimid And Prednisone (Rp) Followed By Oral Revlimid Melphalan And Prednisone (Mpr) In Newly Diagnosed Elderly Multiple Myeloma Patients [NCT01160107]Phase 246 participants (Actual)Interventional2008-07-31Completed
A PHASE III, MULTI-CENTER, RANDOMIZED OPEN LABEL STUDY OF VELCADE, MELPHALAN, PREDNISONE AND THALIDOMIDE (V-MPT) Versus VELCADE, MELPHALAN, PREDNISONE (V-MP) IN ELDERLY UNTREATED MULTIPLE MYELOMA PATIENTS [NCT01063179]Phase 3511 participants (Actual)Interventional2006-05-31Completed
A PHASE 3, MULTICENTRE, RANDOMIZED, CONTROLLED STUDY TO DETERMINE THE EFFICACY AND SAFETY OF LENALIDOMIDE, MELPHALAN AND PREDNISONE (MPR) Versus MELPHALAN (200 mg/m2) FOLLOWED BY STEM CELL TRANSPLANT IN NEWLY DIAGNOSED MULTIPLE MYELOMA SUBJECTS [NCT00551928]Phase 3402 participants (Actual)Interventional2007-06-30Active, not recruiting
CD45A-Depleted Haploidentical Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation [NCT02259348]Phase 212 participants (Actual)Interventional2014-10-31Terminated(stopped due to Investigator's decision.)
A Phase I Study Using Hepatic Arterial Infusion of Autologous Tumor Infiltrating Lymphocytes in Patients With Melanoma and Liver Metastases [NCT04812470]Phase 16 participants (Anticipated)Interventional2023-02-06Recruiting
A Pilot Study Involving Administration of Combination Anti-Retroviral Therapy and Transplantation of HLA-Matched Sibling Peripheral Blood Stem Cells or Partially HLA-Matched Unrelated Umbilical Cord Blood In Adults With HIV Infection and Hematologic Malig [NCT00003435]Phase 10 participants (Actual)Interventional1998-05-31Withdrawn
A Randomized Prospective Study of Early Intensification Versus Alternating Triple Therapy for Patients With Poor Prognosis Lymphoma [NCT00002835]Phase 3116 participants (Actual)Interventional1995-10-30Completed
A Phase II Trial of Multiple Cycles of Sequential High Dose Chemotherapy for Patients With Chemotherapy Sensitive Relapsed Non-Hodgkin's Lymphoma [NCT00003957]Phase 23 participants (Actual)Interventional1998-12-31Completed
A Randomised Study Comparing CIDEX (CCNU, Oral Idarubicin and Dexamethasone) With Melphalan and Prednisolone in Relapsed Multiple Myeloma [NCT00003603]Phase 3660 participants (Anticipated)Interventional1998-03-31Active, not recruiting
A Randomized Phase III Trial of Hyperthermic Isolated Limb Perfusion and Melphalan With and Without Tumor Necrosis Factor in Patients With Localized Advanced Extremity Melanoma [NCT00003789]Phase 3216 participants (Actual)Interventional1999-03-31Completed
Randomized Study of Rituximab (Mabthera) in Patients With Relapsed Follicular Lymphoma Prior to High-Dose Therapy as In Vivo Purging and to Maintain Remission Following High-Dose Therapy [NCT00005589]Phase 3460 participants (Anticipated)Interventional1999-10-31Completed
Evaluation of Pretargeted Anti-CD20 Radioimmunotherapy Combined With BEAM Chemotherapy and Autologous Stem Cell Transplantation for High-Risk B-Cell Malignancies [NCT02483000]Phase 13 participants (Actual)Interventional2017-02-01Terminated(stopped due to Closed early due to lack of funding)
Immunotherapy With Ex Vivo-Expanded Cord Blood-Derived NK Cells Combined With Rituximab High-Dose Chemotherapy and Autologous Stem Cell Transplant for B-Cell Non-Hodgkin's Lymphoma [NCT03019640]Phase 222 participants (Actual)Interventional2017-10-10Completed
Comparison of High Dose Rapid Schedule With Conventional Schedule Chemotherapy for Stage 4 Neuroblastoma Over the Age of One Year [NCT00365755]Phase 30 participants InterventionalCompleted
Multicenter Phase II Study of Haploidentical Hematopoietic Cell Transplantation With CD3/CD19 Depleted Grafts After a Reduced Intensity Conditioning Regimen for Adult Patients With Acute Leukemia [NCT00961142]Phase 223 participants (Actual)Interventional2009-06-30Terminated(stopped due to slow recruitment)
SHARON: Study of Metastatic Cancers in Patients With a Defect in a Homologous Recombination Gene Using Autologous Stems Cells and Potentiated Redox Cycling to Overcome Drug Resistance to Nitrogen Mustard Derivatives [NCT04150042]Phase 110 participants (Anticipated)Interventional2021-01-13Recruiting
BMT-02: A Phase I Trial of Total Marrow Irradiation in Addition to High Dose Melphalan Conditioning Prior to Autologous Transplant for Multiple Myeloma Following Initial Induction Therapy [NCT02043860]Phase 13 participants (Actual)Interventional2014-01-10Terminated(stopped due to low accrual)
Melphalan Pharmacokinetics in Children Undergoing Hematopoietic Stem Cell Transplantation: A Pilot Study [NCT02390544]Phase 134 participants (Actual)Interventional2015-05-08Completed
Alkylator-Intense Conditioning Followed by Autologous Transplantation for Patients With High Risk or Relapsed Solid or CNS Tumors [NCT01505569]20 participants (Anticipated)Interventional2011-10-20Recruiting
A Phase II Trial of Thiotepa, Busulfan, and Melphalan Followed by Autologous or Syngeneic Marrow or Peripheral Blood Stem Cell Transplantation in Patients With Multiple Myeloma [NCT00003146]Phase 20 participants Interventional1997-11-30Completed
S9805, Phase II Study of Tandem High Dose Melphalan Supported by Peripheral Blood Stem Cell Support in Waldenstrom's Macroglobulinemia (WM) [NCT00003416]Phase 29 participants (Actual)Interventional1998-09-30Completed
A Randomized Phase II Trial Comparing Bendamustine and Melphalan With Melphalan Alone as Conditioning Regimen for Autologous Stem Cell Transplantation (ASCT) in Myeloma Patients [NCT03187223]Phase 2121 participants (Actual)Interventional2017-07-20Completed
B-cell Mature Non-Hodgkin's Lymphoma Treatment Protocol in Children and Adolescents 2021 (B-NHL-M-2021) [NCT05518383]Phase 4300 participants (Anticipated)Interventional2022-05-25Recruiting
A Phase II Trial of Isolated Limb Infusion With Melphalan and Dactinomycin for Regional Melanoma and Soft Tissue Sarcoma of the Extremity [NCT00004250]Phase 235 participants (Anticipated)Interventional1999-08-31Completed
Venetoclax Combining With Fludarabine and Melphalan as Conditioning Regimen Prior to Allogeneic Hematopoietic Stem Cell Transplantation for Older Patients With Hematologic Malignancies [NCT05084027]Phase 250 participants (Anticipated)Interventional2021-10-07Recruiting
A Phase II Trial Of Autologous Stem Cell Transplant Followed By Mini-Allogeneic Stem Cell Transplant In Lieu Of Standard Allogeneic Bone Marrow Transplantation For Treatment Of Multiple Myeloma [NCT00014508]Phase 20 participants Interventional2001-11-19Completed
A Phase II Study of Sirolimus, Tacrolimus and Thymoglobulin, as Graft-versus-Host Prophylaxis in Patients Undergoing Unrelated Donor Hematopoietic Cell Transplantation [NCT00589563]Phase 232 participants (Actual)Interventional2007-05-31Completed
Phase I/II Study of the Combination of Arsenic Trioxide With Ascorbic Acid and High-Dose Melphalan for Patients With Multiple Myeloma [NCT00661544]Phase 1/Phase 248 participants (Actual)Interventional2004-03-31Completed
A Phase II Study of Yttrium-90-Labeled Anti-CD20 Monoclonal Antibody in Combination With High-Dose Beam Followed by Autologous Stem Cell Transplantation for Poor Risk/Relapsed B-Cell Lymphoma [NCT00695409]Phase 2122 participants (Actual)Interventional2008-03-18Completed
High-Dose Sequential Therapy and Single Autologous Transplantation for Multiple Myeloma [NCT00349778]Phase 2102 participants (Actual)Interventional2006-08-31Completed
Epigenetic Priming With 5-Azacytidine Prior to in Vivo T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With High Risk Myeloid Malignancies in Morphologic Remission [NCT02497404]Phase 240 participants (Actual)Interventional2015-02-13Completed
ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (HSCT) IN PATIENTS WITH HIGH RISK HEMOGLOBINOPATHIES LIKE SICKLE CELL DISEASE AND β-THALESSEMIA-MAJOR USING REDUCED INTENSITY CONDITIONING REGIMEN [NCT02435901]Phase 1/Phase 229 participants (Actual)Interventional2008-12-31Completed
A Phase 1/2 Trial Evaluating Treatment of Emergent Graft Versus Host Disease (GvHD) With AP1903 After Planned Donor Infusions (DLIs) of T-cells Genetically Modified With the iCasp9 Suicide Gene in Patients With Hematologic Malignancies [NCT01875237]Phase 1/Phase 23 participants (Actual)Interventional2013-12-27Terminated
A Randomized, Open-label Phase 3 Study of Carfilzomib, Melphalan, and Prednisone Versus Bortezomib, Melphalan, and Prednisone in Transplant-ineligible Patients With Newly Diagnosed Multiple Myeloma [NCT01818752]Phase 3955 participants (Actual)Interventional2013-07-08Completed
Phase 1/2A Study Carfilzomib + High Dose Melphalan as Preparative Regimen for Autologous Hematopoietic Stem Cell Transplantation in Multiple Myeloma [NCT01690143]Phase 1/Phase 245 participants (Actual)Interventional2012-05-31Completed
A Phase 1/2 Trial of Carfilzomib and Melphalan and Conditioning for Autologous Stem Cell Transplantation for Multiple Myeloma (CARAMEL) [NCT01842308]Phase 1/Phase 250 participants (Actual)Interventional2013-06-04Completed
Phase I Study of Escalating Doses of 90Y-DOTA-Anti-CD25 Monoclonal Antibody Added to the Conditioning Regimen of Fludarabine, Melphalan, and Organ Sparing Total Marrow and Lymphoid Irradiation (TMLI) as Conditioning for Allogeneic Hematopoietic Cell Trans [NCT05139004]Phase 112 participants (Anticipated)Interventional2022-07-19Recruiting
A Phase 3, Intergroup Multicentre, Randomized, Controlled 3 Arm Parallel Group Study to Determine the Efficacy and Safety of Lenalidomide in Combination With Dexamethasone (RD) Versus Melphalan, Prednisone and Lenalidomide (MPR) Versus Cyclophosphamide, P [NCT01093196]Phase 3660 participants (Anticipated)Interventional2009-10-31Active, not recruiting
A Phase 3, Multicentre, Randomized, Controlled Study to Determine the Efficacy and Safety of Cyclophosphamide, Lenalidomide and Dexamethasone (CRD) Versus Melphalan (200 mg/m2) Followed By Stem Cell Transplant In Newly Diagnosed Multiple Myeloma Subjects [NCT01091831]Phase 3389 participants (Actual)Interventional2009-07-31Active, not recruiting
Phase 2 Study of Planned Donor Lymphocyte Infusion After Reduced Intensity Allogeneic Stem Cell Transplantation [NCT01518153]Phase 216 participants (Actual)Interventional2012-02-29Terminated(stopped due to Objectives not met.)
A Phase I/II Study Evaluating Escalating Doses of 90Y-BC8-DOTA (Anti-CD45) Antibody Followed by BEAM Chemotherapy and Autologous Stem Cell Transplantation for High-Risk Lymphoid Malignancies [NCT01921387]Phase 1/Phase 220 participants (Actual)Interventional2013-10-09Completed
Immunotherapy for Autologous/Syngeneic Peripheral Blood Stem Cell (PBSC) Transplant Patients as Treatment for Advanced Multiple Myeloma [NCT00006244]Phase 236 participants (Actual)Interventional2000-02-29Completed
A Multi-Center Phase III Study of Autologous Transplantation for Patients With Multiple Myeloma Comparing Melphalan 280 mg/m2 + Amifostine With Melphalan 200 mg/m2 + Amifostine [NCT00217438]Phase 3130 participants (Actual)Interventional2005-07-31Completed
Multicenter Randomized Controlled Clinical Study of Mitoxantrone Hydrochloride Liposome Injection Combined With Carmustine, Etoposide and Cytarabine (Modified BEAM Protocol) for T Cell Lymphoma Underwent Autologous Stem Cell Transplantation [NCT05814718]122 participants (Anticipated)Interventional2023-04-15Not yet recruiting
A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric and Adolescent-Young Adults Patients With High Risk Solid Tumors [NCT04530487]Phase 240 participants (Anticipated)Interventional2020-08-19Recruiting
Nonmyeloablative Allogeneic Stem Cell Transplantation For Relapsed Hodgkin's or Non-Hodgkin's Lymphoma After Autologous Transplantation ( A BMT Study) [NCT00121186]Phase 21 participants (Actual)Interventional2005-07-31Terminated(stopped due to poor accrual)
A Phase II Study of Reduced Intensity Double Umbilical Cord Blood Transplantation Using Fludarabine, Melphalan, and Low Dose Total Body Radiation [NCT01408563]Phase 233 participants (Actual)Interventional2011-12-31Completed
BeEAM (Bendamustine, Etoposide, Cytarabine, Melphalan) Versus CEM (Carboplatin, Etoposide, Melphalan) in Lymphoma Patients as a Conditioning Regimen Before Autologous Hematopoietic Cell Transplantation [NCT05813132]60 participants (Actual)Interventional2022-12-01Completed
An Intention-to-Treat Study of Salvage Chemotherapy Followed by Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of High-Risk or Relapsed Hodgkin Lymphoma [NCT00574496]Phase 225 participants (Actual)Interventional2007-11-13Completed
Single Autologous Transplant Followed by Consolidation and Maintenance for Participants ≥ 65 Years of Age Diagnosed With Multiple Myeloma or a Related Plasma Cell Malignancy [NCT01849783]Phase 241 participants (Actual)Interventional2013-04-04Completed
A Phase II Study of V-BEAM (Bortezomib, Carmustine, Etoposide, Cytarabine, and Melphalan) as Conditioning Regimen Prior to Second Autologous Stem Cell Transplantation for Multiple Myeloma [NCT01653418]Phase 210 participants (Actual)Interventional2012-09-30Terminated(stopped due to Due to the high rate of morbidity and mortality)
A Pilot Study of Low Dose Melphalan and Bortezomib for Treatment of Acute Myelogenous Leukemia and High-Risk Myelodysplastic Syndromes [NCT00789256]26 participants (Actual)Interventional2004-09-30Completed
A Phase II Study of Maintenance Treatment With Sequential Bortezomib, Thalidomide and Dexamethasone Following Autologous Peripheral Blood Stem Cell Transplant in Patients With Multiple Myeloma [NCT00792142]Phase 245 participants (Actual)Interventional2008-01-16Completed
Neuroblastoma Protocol 2012: Therapy for Children With Advanced Stage High-Risk Neuroblastoma [NCT01857934]Phase 2153 participants (Actual)Interventional2013-07-05Active, not recruiting
Single-center, Prospective, Open-label, Comparator Study, Blind for Central Accessor to Access the Efficacy, Safety, and Tolerability of Inhalations of Low-doses of Melphalan in Patients With Pneumonia With Confirmed or Suspected COVID-19 [NCT04380376]Phase 260 participants (Anticipated)Interventional2020-04-30Recruiting
Pharmacokinetic Study of Melphalan in Pediatric Hematopoietic Stem Cell Transplantation [NCT04937634]Phase 120 participants (Anticipated)Interventional2020-09-11Recruiting
A Clinical Study of Low-dose Total Body Irradiation and Fludarabine/Busulfan/Melphalan as a Conditioning Regimen for Secondary Umbilical Cord Blood Transplantation in Patients With Hematological Malignancies Who Relapsed After Allo-HSCT [NCT06125483]38 participants (Anticipated)Interventional2023-11-01Recruiting
Haploidentical Donor Hematopoietic Stem Cell Transplantation [NCT02660281]Phase 174 participants (Actual)Interventional2015-10-31Completed
A Phase II Trial of CD34+ Enriched Transplants From HLA-Compatible Related or Unrelated Donors for Treatment of Patients With Leukemia or Lymphoma [NCT05565105]Phase 2100 participants (Anticipated)Interventional2024-03-31Not yet recruiting
Autologous Transplantation With Gemcitabine and High Dose BCNU Plus Melphalan Followed by Consolidation With DCEP Plus Gemcitabine and Taxol/Cisplatin in Patients With Multiple Myeloma and >12 Months of Standard Therapy [NCT00003401]Phase 20 participants Interventional1999-01-31Completed
High Dose Chemotherapy With Stem Cell Rescue in Recently Diagnosed Patients With Advanced (Stage III and IV) Ovarian Cancer With > 1 cm Residual Disease After Debulking Surgery [NCT00003413]Phase 232 participants (Anticipated)Interventional1998-09-30Completed
Phase I/II Study of Escalating Dose Melphalan With Autologous Pluripotent Hematopoietic Stem Cell Support and Amifostine Cytoprotection in Cancer Patients [NCT00003425]Phase 1/Phase 225 participants (Anticipated)Interventional1997-12-31Completed
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With High Risk Hematologic Malignancies [NCT00003335]Phase 244 participants (Actual)Interventional1998-01-31Completed
Melphalan Intra-arterial Chemotherapy for Choroidal Melanoma Chemoreduction - a Phase I Clinical Trial [NCT05893654]Phase 1/Phase 210 participants (Anticipated)Interventional2021-05-01Enrolling by invitation
A Randomized, Controlled, Open-Label Phase 3 Study of Melflufen in Combination With Daratumumab Compared With Daratumumab in Patients With Relapsed or Relapsed-Refractory Multiple Myeloma [NCT04649060]Phase 354 participants (Actual)Interventional2020-12-21Terminated(stopped due to The sponsor decided to terminate the study due to financial issues following an FDA request for a partial clinical hold.)
Pilot Study of JAK Inhibitor Therapy Followed by Reduced Intensity Haploidentical Transplantation for Patients With Myelofibrosis [NCT04370301]Phase 210 participants (Anticipated)Interventional2021-02-09Recruiting
A Randomised Study of High Dose Chemotherapy/Radiotherapy and Autologous Bone Marrow Transplantation in Patients With High Grade Malignant Non-Hodgkin's Lymphoma (Kiel Classification) According to Prognostic Groups [NCT00003815]Phase 30 participants Interventional1994-06-30Active, not recruiting
High-Dose Consolidation With Escalating Doses of Melphalan and Thiotepa for Stage IV Breast Cancer [NCT00003899]Phase 10 participants Interventional1999-01-31Completed
Phase 1-2 Study of the Combination of Escalated Total Bone Marrow Irradiation (TBMI) by Helicoidal Tomotherapy and a Fixed High-dose Melphalan (140 mg/m²) Followed by Peripheral Stem Cell Rescue (PSC) in First Relapsed Multiple Myeloma. [NCT01794572]Phase 1/Phase 213 participants (Actual)Interventional2013-04-24Terminated(stopped due to Low patient recruitement rate and modification of the therapeutic standard)
A Pilot Study of Whole-body MRI-guided Intensity Modulated Radiation Therapy Combined With Systemic Chemotherapy Followed by High-Dose Chemotherapy With Busulfan, Melphalan and Topotecan and Stem Cell Rescue in Patients With Poor Risk Ewing's Sarcoma [NCT01795430]0 participants (Actual)Interventional2013-07-31Withdrawn(stopped due to No participants enrolled.)
Phase I / II Study Of Carfilzomib (CFZ) Intensification Early After Autologous Transplantation (AHCT) For Plasma Cell Myeloma [NCT01658904]Phase 1/Phase 23 participants (Actual)Interventional2012-07-31Terminated(stopped due to study closed prematurely because investigator left National Institutes of Health)
A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation [NCT01529827]Phase 294 participants (Actual)Interventional2012-02-28Completed
Bevacizumab Combined With High-Dose Gemcitabine, Docetaxel, Melphalan, and Carboplatin in Patients With Advanced Epithelial Ovarian Cancer [NCT00583622]Phase 213 participants (Actual)Interventional2007-12-31Terminated(stopped due to Slow Accrual)
Phase I/II Trial of Carfilzomib Plus Melphalan and Prednisone in Elderly Untreated Patients With Multiple Myeloma. [NCT01279694]Phase 1/Phase 272 participants (Actual)Interventional2010-10-31Completed
A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation [NCT00744692]Phase 122 participants (Actual)Interventional2008-10-31Completed
Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies [NCT01499147]100 participants (Actual)Interventional2000-02-29Completed
Isolated Limb Perfusion for Advanced Melanoma or Sarcoma Limited to Extremity With or Without Distant Metastases [NCT02507076]0 participants (Actual)Interventional2012-04-30Withdrawn(stopped due to no enrollment)
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation for Multiple Myeloma Using a Conditioning Regimen of Fludarabine, Melphalan, and Bortezomib [NCT01453101]Phase 254 participants (Actual)Interventional2010-06-09Completed
Study With High Doses of Chemotherapy, Radiotherapy and Consolidation Therapy With Ciclofosfamide and Anticyclooxygenase 2, for the Metastatic Ewing Sarcoma [NCT02727387]Phase 2155 participants (Actual)Interventional2009-06-01Completed
Y Zevalin, BEAM and Rituximab In Autologous Stem Cell Transplantation (ASCT) For Lymphoma [NCT01538472]Phase 1/Phase 240 participants (Actual)Interventional2003-09-30Completed
A Multicenter Open Label Phase 2 Study of Carfilzomib Weekly Plus Melphalan and Prednisone in Untreated Symptomatic Elderly Multiple Myeloma [NCT02302495]Phase 280 participants (Anticipated)Interventional2014-01-31Active, not recruiting
Lenalidomide Plus Melphalan as a Preparative Regimen for Autologous Stem Cell Transplantation in Relapsed Multiple Myeloma: A Phase 1 / 2 Study [NCT01054196]Phase 1/Phase 252 participants (Actual)Interventional2010-08-31Active, not recruiting
UARK 2013-13, Total Therapy 4B - Formerly 2008-01 - A Phase III Trial for Low Risk Myeloma Ages 65 and Under: A Trial Enrolling Subjects to Standard Total Therapy 3 (S-TT3) [NCT00734877]Phase 3382 participants (Actual)Interventional2008-07-31Active, not recruiting
High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN) [NCT04221035]Phase 3800 participants (Anticipated)Interventional2019-11-05Recruiting
Autologous Stem Cell Transplantation With CD34-Selected Peripheral Blood Stem Cells (PBSC) in Pediatric and Adult Patients With Severe Crohn's Disease [NCT00692939]Phase 1/Phase 220 participants (Anticipated)Interventional2012-06-26Recruiting
"A Phase I/II Clinical Trial of Off-the-shelf NK Cell Administration in Combination With Allogeneic SCT to Decrease Disease Relapse in Patients With High-risk Myeloid Malignancies Undergoing Matched Related, Matched Unrelated, One Antigen Mismatched Unrel [NCT05115630]Phase 1/Phase 224 participants (Anticipated)Interventional2022-04-08Recruiting
Reduced-Intensity Fludarabine, Melphalan, and Total Body Irradiation Conditioning for Transplantation of HLA-Haploidentical Related Hematopoietic Cells (Haplo-HCT) For Patients With Hematologic Malignancies [NCT04191187]Phase 237 participants (Anticipated)Interventional2019-12-06Active, not recruiting
Early HLA Matched Sibling Hematopoietic Stem Cell Transplantation for Children With Sickle Cell Disease: A Sickle Transplant Advocacy and Research Alliance (STAR) Trial [NCT04018937]Phase 258 participants (Anticipated)Interventional2019-03-22Recruiting
A Prospective Study of Bortezomib in Combination With Melphalan and Prednisone for Patients With Previously Untreated Multiple Myeloma [NCT00734149]Phase 245 participants (Actual)Interventional2004-07-31Completed
Induction Therapy With Bortezomib-melphalan and Prednisone (VMP) Followed by Lenalidomide and Dexamethasone (Rd) Versus Carfilzomib, Lenalidomide and Dexamethasone (KRd) Plus/Minus Daratumumab, 18 Cycles, Followed by Consolidation and Maintenance Therapy [NCT03742297]Phase 3462 participants (Actual)Interventional2018-10-22Active, not recruiting
[NCT01274403]Phase 2130 participants (Actual)InterventionalCompleted
A Phase I/II Study of Autologous Stem Cell Transplantation Followed by Nonmyeloablative Allogeneic Stem Cell Transplantation for Patients With Relapsed or Refractory Lymphoma - A Multi-center Trial [NCT00005803]Phase 1/Phase 276 participants (Actual)Interventional1999-09-30Completed
A Randomized Phase II Study Comparing Two Different Conditioning Regimens Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) for Children With Juvenile Myelomonocytic Leukemia (JMML) [NCT01824693]Phase 230 participants (Actual)Interventional2013-06-24Completed
A 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning [NCT05031897]Phase 267 participants (Anticipated)Interventional2021-10-25Recruiting
A Feasibility Study of Myeloablative BEAM Allogeneic Transplantation Followed by Oral Ixazomib Maintenance Therapy in Patients With Relapsed High-Risk Multiple Myeloma [NCT02504359]Phase 111 participants (Actual)Interventional2015-07-20Completed
A Prospective, Open-label, Multicenter, Observational Study to Evaluate the Efficacy and Safety of Bortezomib, Melphalan, Prednisone(VMP) for Initial Treatment in Patients With Multiple Myeloma Who do Not Undergo Autologous Stem Cell Transplantation [NCT02474563]171 participants (Actual)Observational2011-05-31Completed
A Pilot Study to Assess Impact of Low Dose Melphalan on Disease Burden Measured by Next Generation Sequencing Before Autologous Hematopoietic Cell Transplant (AHCT) for Multiple Myeloma Patients [NCT05013437]Early Phase 120 participants (Anticipated)Interventional2023-03-31Recruiting
A Randomized Clinical Trial of Lenalidomide (CC-5013) and Dexamethasone With and Without Autologous Peripheral Blood Stem Cell Transplant in Patients With Newly Diagnosed Multiple Myeloma [NCT01731886]Phase 460 participants (Actual)Interventional2012-09-30Completed
A Pilot Study of Allogeneic Hematopoietic Cell Transplantation for Patients With High Grade Central Nervous System Malignancies [NCT04521946]Phase 10 participants (Actual)Interventional2021-01-14Withdrawn(stopped due to No participants enrolled.)
A Pilot Study of Condensed Busulfan, Melphalan, and Fludarabine Conditioning Prior to Ex-vivo CD34+ Selected Allogeneic Hematopoietic Cell Transplantation [NCT04098393]Phase 145 participants (Anticipated)Interventional2019-09-18Recruiting
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism [NCT00176865]Phase 219 participants (Actual)Interventional2002-08-31Completed
Phase II Study of Tandem Cycle Dose-Intense Chemotherapy of Melphalan and Carboplatin, Thiotepa and Cyclophosphamide (STMP V) ± Trastuzumab Followed by Helical Tomotherapy or Local Regional Radiation Therapy for Stage IV Metastatic and Stage IIIB/C Breast [NCT00182793]Phase 232 participants (Actual)Interventional2005-07-31Completed
BEAM + 131Iodine-Anti-B1 Radioimmunotherapy and Autologous Hematopoietic Stem Cell Transplantation for the Treatment of Recurrent Non-Hodgkin's Lymphoma [NCT00574509]Phase 134 participants (Actual)Interventional1996-03-04Completed
Tandem High-Dose Therapy With Melphalan and Total Marrow Irradiation (TMI) With Peripheral Blood Progenitor Cell Support and Lenalidomide Maintenance in Multiple Myeloma: A Phase I/II Trial [NCT00112827]Phase 1/Phase 254 participants (Actual)Interventional2004-11-30Completed
A Current Practice Study of Rituxan in Patient Receiving BEAM Chemotherapy and Autologous Blood Stem Cell Transplantation for High Risk Lymphoma or Hodgkin's Disease [NCT01702961]75 participants (Actual)Interventional2002-06-30Completed
A Study of Granix to Disrupt the Bone Marrow Microenvironment in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation [NCT02112045]Phase 290 participants (Actual)Interventional2015-01-20Terminated(stopped due to Terminated due to results of interim analysis)
A Pilot Study of a Sequential Regimen of Intensive Chemotherapy Followed by Autologous or Allogeneic Transplantation for Refractory Lymphoma (Non-Hodgkin's and Hodgkin's) and Phase 2 Expansion Cohort [NCT02059239]Phase 1/Phase 234 participants (Actual)Interventional2014-06-04Completed
Azacitidine/Vorinostat/GemBuMel With Autologous Stem-Cell Transplant (SCT) in Patients With Refractory Lymphomas [NCT01983969]Phase 1/Phase 261 participants (Actual)Interventional2013-11-07Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

TrialOutcome
NCT00002601 (4) [back to overview]Toxicities Counts
NCT00002601 (4) [back to overview]5-year Progression-free Survival
NCT00002601 (4) [back to overview]Number of Participants With Grade 3 Bilirubin
NCT00002601 (4) [back to overview]5-year Overall Survival
NCT00003042 (2) [back to overview]Three-year Relapse-free Survival
NCT00003042 (2) [back to overview]Five-year Overall Survival
NCT00003199 (3) [back to overview]Event-free Survival
NCT00003199 (3) [back to overview]Number of Participants With Toxicity of a Combination of Low-dose IL-2 and GM-CSF
NCT00003199 (3) [back to overview]Overall Survival
NCT00003631 (1) [back to overview]Objective Response
NCT00003816 (4) [back to overview]4 Year PFS
NCT00003816 (4) [back to overview]4 yr OS
NCT00003816 (4) [back to overview]CR Rate
NCT00003816 (4) [back to overview]Toxicity/TRM at Day 100
NCT00004088 (2) [back to overview]Three-year Overall Survival
NCT00004088 (2) [back to overview]Progression-free Survival
NCT00005803 (4) [back to overview]Progression Free-survival (PFS)
NCT00005803 (4) [back to overview]Overall Survival (OS)
NCT00005803 (4) [back to overview]Non-Relapse Mortality
NCT00005803 (4) [back to overview]Engraftment of HLA Identical PBSC Allografts
NCT00006244 (6) [back to overview]Proportion of Patients Alive and in Remission
NCT00006244 (6) [back to overview]Time to Disease Progression
NCT00006244 (6) [back to overview]Initial Response to Therapy
NCT00006244 (6) [back to overview]Overall Survival
NCT00006244 (6) [back to overview]Number of Patients ≥56 Years Old Experiencing Grade 3-4 Regimen Related Toxicity
NCT00006244 (6) [back to overview]Number of Patients <56 Years Old Experiencing Grade 3-4 Regimen Related Toxicity
NCT00027560 (6) [back to overview]Extensive Chronic Graft-versus-Host Disease Matched Related Patients
NCT00027560 (6) [back to overview]Overall Survival
NCT00027560 (6) [back to overview]Overall Survival
NCT00027560 (6) [back to overview]Extensive Chronic Graft-versus-Host Disease Unrelated and Mismatched Related Patients
NCT00027560 (6) [back to overview]Acute Graft-versus-Host Disease Unrelated and Mismatched Related Patients
NCT00027560 (6) [back to overview]Acute Graft-versus-Host Disease Matched Related Patients
NCT00038857 (1) [back to overview]Number of Participants With Absolute Neutrophil Count Engraftment
NCT00040937 (2) [back to overview]Overall Survival
NCT00040937 (2) [back to overview]Assess Toxicity of Thalidomide/Dexamethasone as a Pre-transplant Induction Regimen.
NCT00043979 (14) [back to overview]Toxicity
NCT00043979 (14) [back to overview]Number of Participants With Engraftment
NCT00043979 (14) [back to overview]Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)
NCT00043979 (14) [back to overview]Median Progression Free Survival
NCT00043979 (14) [back to overview]Early Post Transplantation Relapse
NCT00043979 (14) [back to overview]Cluster of Differentiation 4 (CD4) Reconstitution
NCT00043979 (14) [back to overview]Median Time to Reach Absolute Neutrophil Count of 500/mm(3)
NCT00043979 (14) [back to overview]Median Survival From Date of Progression
NCT00043979 (14) [back to overview]Number of Participants to Complete Conversion to >95% Donor Chimerism
NCT00043979 (14) [back to overview]Number of Participants With Acute and Chronic GVHD
NCT00043979 (14) [back to overview]Median Time to Reach a Platelet Count of 50,000/mm(3)
NCT00043979 (14) [back to overview]Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy
NCT00043979 (14) [back to overview]Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant
NCT00043979 (14) [back to overview]Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)
NCT00054353 (8) [back to overview]OS
NCT00054353 (8) [back to overview]Relapse Rate
NCT00054353 (8) [back to overview]Engraftment
NCT00054353 (8) [back to overview]PFS
NCT00054353 (8) [back to overview]Response Rate
NCT00054353 (8) [back to overview]Incidence of Acute GVHD (Grades III-IV)
NCT00054353 (8) [back to overview]Incidence of Chronic (Extensive) GVHD
NCT00054353 (8) [back to overview]Non-relapse Mortality
NCT00064337 (2) [back to overview]Hematologic Response
NCT00064337 (2) [back to overview]Overall Survival
NCT00067002 (5) [back to overview]Time To Neutrophil Engraftment
NCT00067002 (5) [back to overview]Number of Participants Severity of Acute GVHD by Treatment Arm
NCT00067002 (5) [back to overview]Rate of Chronic GVHD
NCT00067002 (5) [back to overview]Number of Participants With Engraftment
NCT00067002 (5) [back to overview]Rate of Acute Graft Versus Host Disease (GVHD)
NCT00074269 (7) [back to overview]Frequency of the Induction of Full Donor Chimerism of Lymphocytes as Measured at 1 Month Post Allografting
NCT00074269 (7) [back to overview]Number of Participants With Acute and Chronic Graft-versus-host Disease (GVHD)
NCT00074269 (7) [back to overview]Number of Participants With Adverse Events
NCT00074269 (7) [back to overview]Number of Participants With Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes
NCT00074269 (7) [back to overview]Progression-free Survival
NCT00074269 (7) [back to overview]Response as Measured at 12 Months Post Allografting
NCT00074269 (7) [back to overview]Overall Survival
NCT00083551 (1) [back to overview]Overall Survival
NCT00112827 (3) [back to overview]Maximum Tolerated Dose (MTD)
NCT00112827 (3) [back to overview]Number of Subjects With Response
NCT00112827 (3) [back to overview]Overall Survival
NCT00114101 (4) [back to overview]Time to Progression
NCT00114101 (4) [back to overview]Response to Autologous Hematopoietic Stem-cell Transplant (HSCT) at Day 100
NCT00114101 (4) [back to overview]Number of Participants With Progression, Death or Diagnosis of Second Primary Malignancy
NCT00114101 (4) [back to overview]Overall Survival
NCT00121186 (2) [back to overview]Progression-free Survival
NCT00121186 (2) [back to overview]Overall Survival
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Minimal Residual Disease (MRD)
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Dose of CD34
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Dose of NK Cells
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Engraftment Failure
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Fatal Acute Graft-Versus Host Disease (GVHD)
NCT00145626 (13) [back to overview]Number of Transplant-Related Adverse Outcomes: Regimen-Related Mortality
NCT00145626 (13) [back to overview]One-year Survival
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Disease Status at HSCT
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Donor Type
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Match N/6 HLA Loci
NCT00145626 (13) [back to overview]Factors Affecting One-year Survival: Median Age of Donor at HSCT
NCT00145626 (13) [back to overview]Kinetics of Lymphohematopoietic Reconstitution
NCT00145626 (13) [back to overview]Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation
NCT00176839 (6) [back to overview]Incidence Chronic Graft-versus-host Disease (GVHD)
NCT00176839 (6) [back to overview]Incidence of Relapse
NCT00176839 (6) [back to overview]Probability of Long-term Disease-free Survival (DFS)
NCT00176839 (6) [back to overview]Incidence of Acute Graft-versus-host Disease (GVHD)
NCT00176839 (6) [back to overview]Incidence of Regimen-related Toxicity 100 Days Post Transplant
NCT00176839 (6) [back to overview]Probability of Engraftment
NCT00176865 (9) [back to overview]Number of Subjects Alive at One Year
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 365 Days
NCT00176865 (9) [back to overview]Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)
NCT00176865 (9) [back to overview]Number of Subjects Alive at 100 Days
NCT00176865 (9) [back to overview]Incidence of Chronic Graft Versus Host Disease (cGVHD)
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 180 Days
NCT00176865 (9) [back to overview]Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)
NCT00176865 (9) [back to overview]Percentage of Donor Chimerism at 100 Days
NCT00176865 (9) [back to overview]Number of Subjects With Mixed Chimerism
NCT00177047 (17) [back to overview]Number of Participants Experiencing Incidence of Relapse
NCT00177047 (17) [back to overview]Time to Attainment of CR
NCT00177047 (17) [back to overview]Time to Relapse
NCT00177047 (17) [back to overview]Time to Progression
NCT00177047 (17) [back to overview]Number of Participants With Overall Survival
NCT00177047 (17) [back to overview]Count of Participants Experiencing Transplant Related Mortality
NCT00177047 (17) [back to overview]Number of Participants Achieving a Complete Response
NCT00177047 (17) [back to overview]Number of Participants Achieving a Complete Response
NCT00177047 (17) [back to overview]Number of Participants Achieving a Complete Response
NCT00177047 (17) [back to overview]Number of Participants With Absolute Neutrophil Recovery
NCT00177047 (17) [back to overview]Number of Participants With Disease Progression
NCT00177047 (17) [back to overview]Number of Participants With Overall Survival
NCT00177047 (17) [back to overview]Number of Participants With Infections
NCT00177047 (17) [back to overview]Time to Attainment of CR+PR
NCT00177047 (17) [back to overview]Number of Participants With Overall Survival
NCT00177047 (17) [back to overview]Number of Patients With Extended Disease-free Survival
NCT00177047 (17) [back to overview]Number of Participants With Toxicities
NCT00182793 (2) [back to overview]5-Year Overall Survival Rate
NCT00182793 (2) [back to overview]5-Year Relapse-free Survival Rate
NCT00185614 (4) [back to overview]Acute Graft-vs-Host-Disease (aGvHD)
NCT00185614 (4) [back to overview]Event-free Survival (EFS)
NCT00185614 (4) [back to overview]Relapse Rate
NCT00185614 (4) [back to overview]Overall Survival (OS)
NCT00217438 (2) [back to overview]CR and Near CR Rates
NCT00217438 (2) [back to overview]Relative Toxicities Between Melphalan 280 mg/m^2 or Melphalan 200 mg/m^2
NCT00233987 (4) [back to overview]2-year Progression-free Survival
NCT00233987 (4) [back to overview]Overall Survival
NCT00233987 (4) [back to overview]Number of Patients With Grade 3 Through Grade 5 Adverse Events That Are Related to Study Drug
NCT00233987 (4) [back to overview]Response Rate
NCT00238433 (2) [back to overview]Therapy-Related Toxicities
NCT00238433 (2) [back to overview]Disease-Free Survival
NCT00253435 (4) [back to overview]Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS
NCT00253435 (4) [back to overview]Engraftment DLT
NCT00253435 (4) [back to overview]Event-free Survival (EFS) at 3 Years
NCT00253435 (4) [back to overview]Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion
NCT00265889 (3) [back to overview]Progression-free Survival
NCT00265889 (3) [back to overview]Response Rate
NCT00265889 (3) [back to overview]Number of Patients That Experience Pulmonary Toxicity
NCT00288626 (20) [back to overview]Overall Survival
NCT00288626 (20) [back to overview]Percent Change From Screening in Brain Volume
NCT00288626 (20) [back to overview]Survival From Treatment-Related Mortality
NCT00288626 (20) [back to overview]MS Relapse-Free Survival Probability After Transplant
NCT00288626 (20) [back to overview]Number of New T2-Weighted Lesions From Baseline
NCT00288626 (20) [back to overview]MS Progression-Free Survival Probability After Transplant
NCT00288626 (20) [back to overview]MRI Activity-Free Survival Probability After Transplant
NCT00288626 (20) [back to overview]Event-Free Survival Probability After Transplant
NCT00288626 (20) [back to overview]Disease-Modifying Therapy Survival Probability After Transplant
NCT00288626 (20) [back to overview]Change From Baseline in T2-Weighted Lesion Volume
NCT00288626 (20) [back to overview]Survival From MS-Related Mortality
NCT00288626 (20) [back to overview]Change From Baseline in T1-Weighted Lesion Volume
NCT00288626 (20) [back to overview]Change From Baseline in Number of Gadolinium-Enhanced Lesions
NCT00288626 (20) [back to overview]Change From Baseline in Extended Disability Status Scale (EDSS)
NCT00288626 (20) [back to overview]Time to Platelet Engraftment
NCT00288626 (20) [back to overview]Time to Neutrophil Engraftment
NCT00288626 (20) [back to overview]Percent of Participants Who Experienced All-Cause Morbidity Within 12 Months of Post-HCT
NCT00288626 (20) [back to overview]Percent of Participants Who Experienced All-Cause Morbidity
NCT00288626 (20) [back to overview]Event-Free Survival Probability During the 3 Years After Transplant
NCT00288626 (20) [back to overview]Event-Free Survival Probability During the 5 Years After Transplant
NCT00307086 (1) [back to overview]Overall Survival (OS)
NCT00325416 (4) [back to overview]Phase II Overall Survival (OS)
NCT00325416 (4) [back to overview]Phase II Event Free Survival (EFS)
NCT00325416 (4) [back to overview]Phase I - Maximum Tolerated Dose (MTD) Level
NCT00325416 (4) [back to overview]Phase II Participants - Overall Response Rate
NCT00349778 (3) [back to overview]Number of Participants With Pulmonary Toxicity
NCT00349778 (3) [back to overview]Number of Participants That Relapse After Autologous Transplantation
NCT00349778 (3) [back to overview]Overall Participant Survival (OS)
NCT00357396 (1) [back to overview]Overall Objective Response
NCT00383448 (4) [back to overview]Number of Patients With Chronic Graft Versus Host Disease (GVHD)
NCT00383448 (4) [back to overview]Number of Patients With Donor Cell Engraftment
NCT00383448 (4) [back to overview]Number of Patients With Acute Graft Versus Host Disease (GVHD)
NCT00383448 (4) [back to overview]Number of Patients Whose Death Was Related to the Transplant
NCT00385788 (1) [back to overview]Transplant Related Mortality Rate
NCT00405756 (28) [back to overview]Kaplan Meier Estimates of Overall Survival (OS)
NCT00405756 (28) [back to overview]Kaplan Meier Estimates for Time to Next Antimyeloma Therapy
NCT00405756 (28) [back to overview]Kaplan Meier Estimates for Duration of Response as Determined by the Central Adjudication Committee (CAC)
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Financial Difficulties Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Insomnia Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Nausea and Vomiting Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Pain Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Physical Functioning Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Role Functioning Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Social Functioning Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Body Image Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Side Effects of Treatment Scale
NCT00405756 (28) [back to overview]Number of Participants in Disease Response Categories Representing Their Best Response During the Double-blind Treatment Period
NCT00405756 (28) [back to overview]Summary of Participants With Treatment-Emergent Adverse Events (TEAE) During the Double-Blind Treatment Period
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Future Perspective Scale
NCT00405756 (28) [back to overview]Kaplan Meier Estimates of Progression-free Survival (PFS) Based on the Response Assessment by the Central Adjudication Committee (CAC)
NCT00405756 (28) [back to overview]Kaplan Meier Estimates of Progression-free Survival (PFS) From Start of Maintenance Therapy Period Based on the Response Assessment by the Central Adjudication Committee (CAC)
NCT00405756 (28) [back to overview]Kaplan Meier Estimates of Time to Progression (TTP) Based on the Response Assessment by the Central Adjudication Committee (CAC)
NCT00405756 (28) [back to overview]Time to First Response
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Quality of Life Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Appetite Loss Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Congitive Functioning Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Constipation Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Diarrhoea Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Dyspnoea Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Emotional Functioning Scale
NCT00405756 (28) [back to overview]Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Scale
NCT00423514 (1) [back to overview]Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0
NCT00427557 (1) [back to overview]Number of Participants With Engraftment
NCT00427765 (1) [back to overview]Average Overall Survival Time
NCT00429572 (5) [back to overview]Time to Progressive Disease
NCT00429572 (5) [back to overview]Grade II-IV Toxicity
NCT00429572 (5) [back to overview]Overall Survival
NCT00429572 (5) [back to overview]Number of Participants With Tumor Response
NCT00429572 (5) [back to overview]Number of Participants With Acute or Chronic GVHD And Response to Therapy
NCT00439556 (2) [back to overview]Disease-free Survival
NCT00439556 (2) [back to overview]Number of Participants With Dose Limiting Toxicity (DLT)
NCT00469209 (1) [back to overview]Number of Patients Reaching Complete Response (CR)
NCT00472056 (1) [back to overview]Disease-free Survival (DFS)
NCT00477750 (5) [back to overview]Percentage of Participants With Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 (v3)
NCT00477750 (5) [back to overview]Progression-free Survival
NCT00477750 (5) [back to overview]Overall Survival (OS) at 3 Years
NCT00477750 (5) [back to overview]Patients With Overall Confirmed Response
NCT00477750 (5) [back to overview]Duration of Response (DOR)
NCT00477971 (4) [back to overview]3-Year Progression Free Survival
NCT00477971 (4) [back to overview]Organ Response to Treatment
NCT00477971 (4) [back to overview]Hematologic Response Rate
NCT00477971 (4) [back to overview]3 Year Overall Survival
NCT00505895 (2) [back to overview]Number of Participants With Successful Engraftment at Day 100
NCT00505895 (2) [back to overview]Acute Grade II-IV Graft Versus Host Disease (GVHD)
NCT00505921 (1) [back to overview]Participant Progression Free Survival at 2 Years
NCT00506129 (2) [back to overview]Participant's Response According to Physician's Global Assessment of Clinical Condition (PGA)
NCT00506129 (2) [back to overview]Average Overall Survival (OS) Length
NCT00507416 (8) [back to overview]Change From Baseline in EORTC QLQ-C30 - Global Health Status
NCT00507416 (8) [back to overview]Time to Alternative Therapy
NCT00507416 (8) [back to overview]Progression Free Survival (PFS)
NCT00507416 (8) [back to overview]Percentage of Participants With an Overall Response
NCT00507416 (8) [back to overview]Percentage of Participants With a Complete Response or a Very Good Partial Response
NCT00507416 (8) [back to overview]Percentage of Participants With a Complete Response
NCT00507416 (8) [back to overview]Overall Survival
NCT00507416 (8) [back to overview]Duration of Response
NCT00520767 (3) [back to overview]Complete Hematologic Response
NCT00520767 (3) [back to overview]Overall Survival
NCT00520767 (3) [back to overview]Time to Treatment Failure (TTF)
NCT00521014 (1) [back to overview]Progression-free Survival Rate
NCT00536601 (8) [back to overview]Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)
NCT00536601 (8) [back to overview]Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)
NCT00536601 (8) [back to overview]Regimen-related Toxicity Grade 2-4 in Any Organ (Measured by Bearman Score, Values Range From 0 (None) to 4 (Fatal))
NCT00536601 (8) [back to overview]Response Rate (Complete Remission)
NCT00536601 (8) [back to overview]Overall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)
NCT00536601 (8) [back to overview]Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)
NCT00536601 (8) [back to overview]Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)
NCT00536601 (8) [back to overview]Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)
NCT00539500 (3) [back to overview]Number of Treated Participants With Engraftment Failure at Day 42
NCT00539500 (3) [back to overview]Engraftment Failure Rate
NCT00539500 (3) [back to overview]Number of Participants With Device-related Toxicity Associated With Transplantation of CD133+ Cells
NCT00544115 (2) [back to overview]Two-year Overall Survival
NCT00544115 (2) [back to overview]Neutrophil Engraftment - The Days Till ANC Recovery
NCT00544466 (2) [back to overview]Number of Grade 3 and Above Toxicities of Helical Tomotherapy (HT) in Combination With Fludarabine and Melphalan Followed by Allogeneic Stem Cell Transplantation.
NCT00544466 (2) [back to overview]Overall Survival on Day 180 Days Post-transplant
NCT00547196 (2) [back to overview]Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
NCT00547196 (2) [back to overview]Survival Rate at Day 180 After Allogeneic Transplant From Umbilical Cord Blood (UCB)
NCT00566098 (7) [back to overview]Disease Response
NCT00566098 (7) [back to overview]Hematopoietic Engraftment
NCT00566098 (7) [back to overview]Anti-tumor Immune Responses
NCT00566098 (7) [back to overview]Feasibility of MILs Generation as Assessed by Percentage of Participants With Successful MIL Generation
NCT00566098 (7) [back to overview]Survival
NCT00566098 (7) [back to overview]T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)
NCT00566098 (7) [back to overview]Pneumococcal-specific Vaccine Responses
NCT00566696 (7) [back to overview]Incidence of Regimen-related Mortality
NCT00566696 (7) [back to overview]Overall Survival (OS)
NCT00566696 (7) [back to overview]To Estimate the Rate of Overall Grade III-IV Acute GVHD, and the Rate and Severity of Chronic GVHD in Research Participants.
NCT00566696 (7) [back to overview]Event-free Survival (EFS)
NCT00566696 (7) [back to overview]To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.
NCT00566696 (7) [back to overview]Incidence of Non-hematologic Regimen-related Toxicities
NCT00566696 (7) [back to overview]Disease-Free Survival (DFS)
NCT00567567 (14) [back to overview]Topotecan Systemic Clearance
NCT00567567 (14) [back to overview]Event-free Survival Rate
NCT00567567 (14) [back to overview]Incidence Rate of Local Recurrence
NCT00567567 (14) [back to overview]Peak Serum Concentration of Isotretinoin in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies
NCT00567567 (14) [back to overview]Proportion of Patients With a Polymorphism
NCT00567567 (14) [back to overview]Intraspinal Extension
NCT00567567 (14) [back to overview]Response After Induction Therapy
NCT00567567 (14) [back to overview]Surgical Response
NCT00567567 (14) [back to overview]EFS Pts Non-randomly Assigned to Single CEM (12-18 Mths, Stg. 4, MYCN Nonamplified Tumor/Unfavorable or Indeterminant Histopathology/Diploid DNA Content & Pts>547 Days, Stg.3, MYCN Nonamplified Tumor AND Unfavorable or Indeterminant Histopathology).
NCT00567567 (14) [back to overview]Type of Surgical or Radiotherapy Complication
NCT00567567 (14) [back to overview]OS in Patients 12-18 Months, Stage 4, MYCN Nonamplified Tumor/Unfavorable Histopathology/Diploid DNA Content/Indeterminant Histology/Ploidy and Patients > 547 Days, Stage 3, MYCN Nonamplified Tumor AND Unfavorable Histopathology/Indeterminant Histology
NCT00567567 (14) [back to overview]Enumeration of Peripheral Blood Cluster of Differentiation (CD)3, CD4, and CD8 Cells
NCT00567567 (14) [back to overview]Duration of Greater Than or Equal to Grade 3 Neutropenia
NCT00567567 (14) [back to overview]Duration of Greater Than or Equal to Grade 3 Thrombocytopenia
NCT00571493 (3) [back to overview]Maximum Tolerated Dose (MTD) of Bortezomib
NCT00571493 (3) [back to overview]Preliminary Estimate of Overall Response Rate (ORR)
NCT00571493 (3) [back to overview]Progression-free Survival (PFS), and Overall Survival (OS)
NCT00572897 (6) [back to overview]Transplant-related Mortality
NCT00572897 (6) [back to overview]Response Outcomes
NCT00572897 (6) [back to overview]PLT
NCT00572897 (6) [back to overview]The Primary Endpoint is Progression-free Survival.
NCT00572897 (6) [back to overview]Absolute Neutrophil Count (ANC)
NCT00572897 (6) [back to overview]Overall Survival
NCT00574496 (3) [back to overview]Disease Relapse or Progression as Measured by CT Scan or PET
NCT00574496 (3) [back to overview]Overall Survival
NCT00574496 (3) [back to overview]Progression-free Survival at 1 Year
NCT00577096 (10) [back to overview]Number of Platelet Transfusions Needed to Maintain Adequate Number of Platelets. (Long Term)
NCT00577096 (10) [back to overview]Number of Stem Cell Collection Attempts (Long Term)
NCT00577096 (10) [back to overview]Total Number of Days of Stem Cell Collection (Long Term)
NCT00577096 (10) [back to overview]Number of Stem Cell Collection Attempts (Short Term)
NCT00577096 (10) [back to overview]Hemoglobin Levels Before Chemotherapy and During Transplantation Period (Short Term)
NCT00577096 (10) [back to overview]Hemoglobin Levels Before Chemotherapy and During Transplantation Period (Long Term)
NCT00577096 (10) [back to overview]Total Number of Days of Stem Cell Collection (Short Term)
NCT00577096 (10) [back to overview]Number of Platelet Transfusions Needed to Maintain Adequate Number of Platelets.(Short Term)
NCT00577096 (10) [back to overview]Number of Red Blood Cell Transfusions Needed to Maintain Hemoglobin Levels (Long Term)
NCT00577096 (10) [back to overview]Number of Red Blood Cell Transfusions Needed to Maintain Hemoglobin Levels (Short Term)
NCT00577278 (3) [back to overview]Progression-free Survival at Two Years
NCT00577278 (3) [back to overview]Relapse/Progression Rate at Two Years
NCT00577278 (3) [back to overview]Overall Survival at Two Years
NCT00582933 (1) [back to overview]Death From GVHD
NCT00583622 (1) [back to overview]Participant Response
NCT00589563 (14) [back to overview]Cumulative Incidence of Chronic GVHD
NCT00589563 (14) [back to overview]Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease (GVHD) at Day 100
NCT00589563 (14) [back to overview]Incidence of Disease Relapse/Progression at 2 Years Post HSCT
NCT00589563 (14) [back to overview]Event Free Survival at Two Years Post HSCT
NCT00589563 (14) [back to overview]Non-relapse Mortality at 100 Days Post HSCT
NCT00589563 (14) [back to overview]Non-relapse Mortality at Two Years Post HSCT
NCT00589563 (14) [back to overview]Occurence of Sinusoidal Obstructive Syndrome (SOS)
NCT00589563 (14) [back to overview]Occurrence of Thrombotic Microangiopathy
NCT00589563 (14) [back to overview]Overall Survival at Two Years Post HSCT
NCT00589563 (14) [back to overview]Time to Absolute Neutrophil Count Recovery (Engraftment)
NCT00589563 (14) [back to overview]Time to Platelet Count Recovery (Engraftment)
NCT00589563 (14) [back to overview]Occurence of Infections Including Cytomegalovirus and Epstein-Barr Virus Reactivation
NCT00589563 (14) [back to overview]Severity of Acute GVHD
NCT00589563 (14) [back to overview]Severity of Chronic GVHD
NCT00591630 (1) [back to overview]Number of Participants With a 2-Year Progression-Free Survival (PFS)
NCT00602641 (4) [back to overview]Very Good Partial Response (VGPR) Rate
NCT00602641 (4) [back to overview]Progression-Free Survival (PFS)
NCT00602641 (4) [back to overview]Change in Functional Assessment of Cancer Therapy-Neurotoxicity Trial Outcome Index (FACT-Ntx TOI) Score From Baseline to Cycle 12
NCT00602641 (4) [back to overview]Overall Survival
NCT00618540 (8) [back to overview]Incidence of Chronic GVHD
NCT00618540 (8) [back to overview]Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
NCT00618540 (8) [back to overview]Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD)
NCT00618540 (8) [back to overview]Neutrophil Engraftment
NCT00618540 (8) [back to overview]Overall Survival
NCT00618540 (8) [back to overview]Platelet Engraftment
NCT00618540 (8) [back to overview]Transplantation-related Death
NCT00618540 (8) [back to overview]Disease-free Survival at 12 Months Post Transplantation
NCT00635024 (4) [back to overview]Overall Survival (OS)
NCT00635024 (4) [back to overview]Hematological Response Rate Defined as the Number of Participants Who Achieve a Confirmed Response
NCT00635024 (4) [back to overview]Number of Participants With Severe Non-hematological Adverse Events
NCT00635024 (4) [back to overview]Progression-free Survival (PFS)
NCT00651937 (2) [back to overview]Mean Symptom Severity Burden as Measured by MDASI Scores
NCT00651937 (2) [back to overview]Mean Symptom Severity Burden as Measured by MDASI Scores
NCT00661544 (1) [back to overview]Response Rate
NCT00679367 (3) [back to overview]Number of Organs Improved or Stable Based on Description Below:
NCT00679367 (3) [back to overview]Number of Participants With Hematologic Response
NCT00679367 (3) [back to overview]Number of Participants Removed From Study Due to Toxicities
NCT00683046 (2) [back to overview]Median Overall Survival
NCT00683046 (2) [back to overview]Median Disease-free Survival
NCT00689936 (43) [back to overview]Number of Participants With Adverse Events (AEs) During the Active Treatment Phase
NCT00689936 (43) [back to overview]Shift From Baseline to Most Extreme Postbaseline Value in Absolute Neutrophil Count During the Active Treatment Phase
NCT00689936 (43) [back to overview]Shift From Baseline to Most Extreme Postbaseline Value in Creatinine Clearance (CrCl) During the Active Treatment Phase
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Time to Treatment Failure (TTF) at the Time of Final Analysis
NCT00689936 (43) [back to overview]Change From Baseline in the European Quality of Life-5 Dimensions (EQ-5D) Health Utility Index Score
NCT00689936 (43) [back to overview]Kaplan Meier Estimates for Time to Second-line Anti-myeloma Treatment (AMT)
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Duration of Myeloma Response as Determined by an Investigator Assessment at Time of Final Analysis
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Duration of Myeloma Response as Determined by the IRAC
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Overall Survival at the Time of Final Analysis (OS)
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Time to Second Line Therapy AMT at the Time of Final Analysis
NCT00689936 (43) [back to overview]Kaplan Meier Estimates of Time to Treatment Failure (TTF)
NCT00689936 (43) [back to overview]Shift From Baseline to Most Extreme Postbaseline Value in Platelet Count During the Active Treatment Phase.
NCT00689936 (43) [back to overview]Kaplan-Meier Estimates of PFS Based on the Response Assessment by the Investigator At the Time of Final Analysis
NCT00689936 (43) [back to overview]Kaplan-Meier Estimates of Progression-free Survival (PFS) Based on the Response Assessment by the Independent Review Adjudication Committee (IRAC)
NCT00689936 (43) [back to overview]Percentage of Participants With a Myeloma Response by Adverse Risk Cytogenetic Risk Category Based on IRAC Review.
NCT00689936 (43) [back to overview]Percentage of Participants With a Myeloma Response by Favorable Hyperdiploidy Risk Cytogenetic Risk Category Based on IRAC Review
NCT00689936 (43) [back to overview]Percentage of Participants With a Myeloma Response by Normal Risk Cytogenetic Risk Category Based on IRAC Review
NCT00689936 (43) [back to overview]Percentage of Participants With a Myeloma Response by Uncertain Risk Cytogenetic Risk Category Based on IRAC Review
NCT00689936 (43) [back to overview]Shift From Baseline to Most Extreme Postbaseline Value in Hemoglobin During the Active Treatment Phase
NCT00689936 (43) [back to overview]Percentage of Participants With an Objective Response After Second-line Anti-myeloma Treatment at the Time of Final Analysis
NCT00689936 (43) [back to overview]Percentage of Participants With an Objective Response Based on Investigator Assessment at Time of Final Analysis
NCT00689936 (43) [back to overview]Percentage of Participants With an Objective Response Based on IRAC Review
NCT00689936 (43) [back to overview]Time to First Response Based on the Investigator Assessment at the Time of Final Analysis
NCT00689936 (43) [back to overview]Time to First Response Based on the Review by the IRAC
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Constipation Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Diarrhea Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Dyspnea Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Fatigue Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Financial Difficulties Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Insomnia Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Nausea/Vomiting Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Pain Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain
NCT00689936 (43) [back to overview]Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain
NCT00689936 (43) [back to overview]Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Health Status Domain
NCT00689936 (43) [back to overview]Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Body Image Scale
NCT00689936 (43) [back to overview]Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms Scale
NCT00689936 (43) [back to overview]Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Future Perspective Scale
NCT00689936 (43) [back to overview]Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Side Effects Treatment Scale
NCT00691015 (8) [back to overview]Severity of Acute Graft-versus-host Disease (GVHD)
NCT00691015 (8) [back to overview]Safety, as Defined by Serious Adverse Events and Adverse Events Related to Study Treatment.
NCT00691015 (8) [back to overview]Overall Survival.
NCT00691015 (8) [back to overview]Karnofsky Performance Status Performance Status
NCT00691015 (8) [back to overview]Incidence of Infections, Including Bacterial, Fungal, and Viral Infections (i.e., CMV and EBV Reactivation, Including Post-transplant Lymphoproliferative Disorders)
NCT00691015 (8) [back to overview]Incidence of Chronic GVHD.
NCT00691015 (8) [back to overview]Incidence of Acute Graft-versus-host Disease (GVHD)
NCT00691015 (8) [back to overview]Time to Engraftment (i.e., Absolute Neutrophil Recovery [ANC > 500/mm³] )
NCT00691704 (5) [back to overview]Time to Response
NCT00691704 (5) [back to overview]Overall Survival
NCT00691704 (5) [back to overview]Time to Progression
NCT00691704 (5) [back to overview]Progression Free Survival
NCT00691704 (5) [back to overview]Duration of Response
NCT00695409 (8) [back to overview]Number of Patients With RIT/ZBEAM Developing Therapy Induced MDS and AML
NCT00695409 (8) [back to overview]Time to Platelet Recovery
NCT00695409 (8) [back to overview]Time to Neutrophil Recovery
NCT00695409 (8) [back to overview]Number of Patients With Active Disease at ASCT Achieving CR/PR by Day 100 After ASCT
NCT00695409 (8) [back to overview]2-Year Progression-Free Survival
NCT00695409 (8) [back to overview]2-Year Cumulative Incidence of Progression
NCT00695409 (8) [back to overview]100-Day Treatment-Related Mortality
NCT00695409 (8) [back to overview]2-Year Overall Survival
NCT00734149 (3) [back to overview]Median Duration of Response
NCT00734149 (3) [back to overview]Response
NCT00734149 (3) [back to overview]Number of Patients With Any Grade or Severe Adverse Event
NCT00743288 (5) [back to overview]Time to Progression
NCT00743288 (5) [back to overview]Overall Response Rate (ORR) and Clinical Benefit Rate (CBR) [ORR= Complete Response (CR) + Very Good Partial Response (VGPR) + Partial Response (PR)]; CBR=ORR + Minimal Response (MR)] Following Treatment With Panobinostat and Melphalan
NCT00743288 (5) [back to overview]Duration of Response
NCT00743288 (5) [back to overview]Maximum Tolerated Dose (MTD)
NCT00743288 (5) [back to overview]MTD
NCT00744692 (9) [back to overview]To Describe the Incidence of Grade 3-4 Organ Toxicity
NCT00744692 (9) [back to overview]To Describe the Pace of Neutrophil Recovery
NCT00744692 (9) [back to overview]To Describe the Pace of Platelet Recovery
NCT00744692 (9) [back to overview]To Determine the Overall Survival at day180 Post-transplant
NCT00744692 (9) [back to overview]To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure
NCT00744692 (9) [back to overview]To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)
NCT00744692 (9) [back to overview]To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant
NCT00744692 (9) [back to overview]To Evaluate the Pace of Immune Reconstitution.
NCT00744692 (9) [back to overview]Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.
NCT00784823 (1) [back to overview]The Maximum Tolerated Dose of Bortezomib (MTD)
NCT00789256 (1) [back to overview]Response Rate of the Combination of Bortezomib and Melphalan in Patients With AML and High-risk MDS.
NCT00790647 (4) [back to overview]Number of Participants Surviving at 2 Years
NCT00790647 (4) [back to overview]Number of Participants With Hematologic Response
NCT00790647 (4) [back to overview]Number of Participants Surviving at 1 Year
NCT00790647 (4) [back to overview]Number of Participants Surviving at 100 Days From Transplant
NCT00792142 (3) [back to overview]One Year Overall Survival
NCT00792142 (3) [back to overview]One Year Progression-free Survival (PFS)
NCT00792142 (3) [back to overview]Number of Participants With Adverse Events
NCT00793572 (6) [back to overview]Number of Patients Surviving Progression-free
NCT00793572 (6) [back to overview]Number of Patients Surviving Overall
NCT00793572 (6) [back to overview]Number of Patients With Chronic GVHD
NCT00793572 (6) [back to overview]Number of Patients With Grade II-IV Acute GVHD
NCT00793572 (6) [back to overview]Number of Patients With Non-relapse Mortality
NCT00793572 (6) [back to overview]Number of Patients With Toxicities Related to Bortezomib Maintenance Therapy
NCT00793650 (2) [back to overview]Safety and Engraftment
NCT00793650 (2) [back to overview]Response Rate Using EBMT(European Group for Blood and Bone Marrow Transplan) Criteria at Day +100 After Transplant.
NCT00807599 (2) [back to overview]Progression Free Survival (PFS) Rate at 2 Years After Enrollment in Untreated Patients With Multiple Myeloma.
NCT00807599 (2) [back to overview]Overall Survival
NCT00827099 (2) [back to overview]Number of Patients That Engrafted Blood Counts by 30 Days After Transplant
NCT00827099 (2) [back to overview]Number of Participants With 100 Day Transplant-related Mortality (TRM)
NCT00856388 (8) [back to overview]Rate of Complete Donor Chimerism - Blood
NCT00856388 (8) [back to overview]Median Time to Platelet Engraftment
NCT00856388 (8) [back to overview]Day 100 TRM
NCT00856388 (8) [back to overview]Acute GVHD Grade III-IV
NCT00856388 (8) [back to overview]3 yr Overall Survival
NCT00856388 (8) [back to overview]1 yr Extenstive Chronic GVHD
NCT00856388 (8) [back to overview]Median Time to ANC Engraftment
NCT00856388 (8) [back to overview]Rate of Complete Donor Chimerism - Blood
NCT00890552 (4) [back to overview]Duration of Response
NCT00890552 (4) [back to overview]Event-free Survival (EFS)
NCT00890552 (4) [back to overview]Overall Survival (OS)
NCT00890552 (4) [back to overview]Hematologic Response Rate
NCT00899847 (8) [back to overview]Overall Response Rate (ORR)
NCT00899847 (8) [back to overview]Overall Survival (OS)
NCT00899847 (8) [back to overview]Partial Response Rate (PRR)
NCT00899847 (8) [back to overview]Median Time to Engraftment After Allo-PBSC Transplant
NCT00899847 (8) [back to overview]Median Time to Engraftment After Auto-PBSC Transplant
NCT00899847 (8) [back to overview]Event-free Survival (EFS)
NCT00899847 (8) [back to overview]Complete Response Rate (CRR)
NCT00899847 (8) [back to overview]Incidence of Graft Versus Host Disease (GvHD)
NCT00911859 (9) [back to overview]Overall Survival
NCT00911859 (9) [back to overview]Duration of Response (DOR)
NCT00911859 (9) [back to overview]Change From Baseline to Cycle 9 in Global Health Status/Quality of Life Subscale of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ C30)
NCT00911859 (9) [back to overview]1-year Survival Rate
NCT00911859 (9) [back to overview]1-year Progression-Free Survival (PFS) Rate
NCT00911859 (9) [back to overview]Percentage of Participants Who Achieved Overall Response ie, Complete Response (CR) or Partial Response (PR) - European Group for Blood and Marrow Transplantation (EBMT) Criteria
NCT00911859 (9) [back to overview]Progression-Free Survival (PFS)
NCT00911859 (9) [back to overview]Percentage of Participants Who Achieved Stringent Complete Response (sCR) - International Myeloma Working Group (IMWG) Criteria
NCT00911859 (9) [back to overview]Percentage of Participants Who Achieved Complete Response (CR) - European Group for Blood and Marrow Transplantation (EBMT) Criteria
NCT00916058 (6) [back to overview]Progression-Free Survival (Phase 1)
NCT00916058 (6) [back to overview]Overall Survival at 3 Years (Phase 2)
NCT00916058 (6) [back to overview]Overall Response Rate (Phase 2) - Number of Participants Achieving at Least a Partial Response or Better in Disease Status at Day 100 Post-transplant
NCT00916058 (6) [back to overview]Progression-Free Survival (Phase 2)
NCT00916058 (6) [back to overview]Maximum Tolerated Dose (Phase 1)
NCT00916058 (6) [back to overview]Overall Survival at 2 Years (Phase 1)
NCT00925782 (4) [back to overview]Area Under the Curve (0-t)
NCT00925782 (4) [back to overview]Concentration-Max (Cmax)
NCT00925782 (4) [back to overview]Determination of Engraftment Following Melphalan-alkeran Sequence and Alkeran-melphalan Sequence Followed by ASCT
NCT00925782 (4) [back to overview]Determination of Myeloablation Following Melphalan-alkeran Sequence and Alkeran-melphalan Sequence Followed by ASCT
NCT00943592 (8) [back to overview]Number of Participants With Skin Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation
NCT00943592 (8) [back to overview]Treatment-related Mortality (TRM)
NCT00943592 (8) [back to overview]Number of Participants With Renal Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation
NCT00943592 (8) [back to overview]Number of Participants With Other Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation
NCT00943592 (8) [back to overview]Number of Participants With Hepatic Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation
NCT00943592 (8) [back to overview]Relapse Rate
NCT00943592 (8) [back to overview]Progression-free Survival (PFS)
NCT00943592 (8) [back to overview]Overall Survival (OS)
NCT00943800 (3) [back to overview]Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
NCT00943800 (3) [back to overview]Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years
NCT00943800 (3) [back to overview]Percentage of Participants With Neutrophil Engraftment
NCT00948922 (3) [back to overview]Progression Free Survival (PFS)
NCT00948922 (3) [back to overview]Percentage of Participants With Acute or Chronic Graft-versus-host Disease (GVHD) Following Transplant
NCT00948922 (3) [back to overview]Overall Survival (OS) Rate
NCT00985907 (1) [back to overview]Number of Participants With Dose Limiting Toxicities (DLT) (Phase 1)
NCT00985959 (7) [back to overview]Number of Participants With Dose Limiting Toxicity During the Phase I (Cycle 1)
NCT00985959 (7) [back to overview]Number of Participants With Overall Response (Complete Response [CR] + Partial Response [PR]) - Phase I and II
NCT00985959 (7) [back to overview]Maximum Observed Plasma Concentration (Cmax) of Melphalan - Phase I
NCT00985959 (7) [back to overview]Maximum Observed Plasma Concentration (Cmax) of Prednisolone - Phase I
NCT00985959 (7) [back to overview]Maximum Observed Plasma Concentration (Cmax) of Bortezomib (JNJ-26866138 Alone) - Phase I
NCT00985959 (7) [back to overview]Maximum Observed Plasma Concentration (Cmax) of Bortezomib (JNJ-26866138 in Combination With Melphalan and Prednisolone) - Phase I
NCT00985959 (7) [back to overview]Median Time to First Response - Phase II
NCT00992446 (4) [back to overview]Event-free Survival
NCT00992446 (4) [back to overview]Overall Survival
NCT00992446 (4) [back to overview]Median Time to Disease Progression
NCT00992446 (4) [back to overview]Toxicity of Vorinostat Bortezomib Maintenance Therapy After Autologous Transplant
NCT01005576 (4) [back to overview]Median Time to Platelet Engraftment
NCT01005576 (4) [back to overview]Primary Objective: Event-free Survival at 1 Year.
NCT01005576 (4) [back to overview]Incidence of Disease Recurrence
NCT01005576 (4) [back to overview]Median Time to ANC Engraftment
NCT01008462 (7) [back to overview]Event-Free Survival (EFS)
NCT01008462 (7) [back to overview]Number of Patients Who Engrafted
NCT01008462 (7) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease
NCT01008462 (7) [back to overview]Non-relapse Mortality (NRM)
NCT01008462 (7) [back to overview]Overall Survival
NCT01008462 (7) [back to overview]Number of Patients With Relapsed/Progressive Disease
NCT01008462 (7) [back to overview]Number of Patients Who Had Infections
NCT01010217 (6) [back to overview]Number of Participants With Non-relapse Mortality (NRM)
NCT01010217 (6) [back to overview]Number of Participants With Non Related Mortality (NRM)
NCT01010217 (6) [back to overview]Disease Free Survival
NCT01010217 (6) [back to overview]cGVHD
NCT01010217 (6) [back to overview]Engraftments
NCT01010217 (6) [back to overview]Grade III-IV aGVHD
NCT01035463 (3) [back to overview]Event-free Survival
NCT01035463 (3) [back to overview]Overall Survival
NCT01035463 (3) [back to overview]Maximum Tolerated Dose of Lenalidomide (Phase I)
NCT01045460 (5) [back to overview]Response Rates by Blade Criteria
NCT01045460 (5) [back to overview]Safety as Measured by Grade 3-5 Adverse Events
NCT01045460 (5) [back to overview]Overall Survival
NCT01045460 (5) [back to overview]Progression-free Survival
NCT01045460 (5) [back to overview]Feasibility as Measured by Participant Withdrawal or Removal
NCT01050764 (6) [back to overview]Serious Infections
NCT01050764 (6) [back to overview]Overall Survival (OS), 1 Year
NCT01050764 (6) [back to overview]Median Overall Survival (OS)
NCT01050764 (6) [back to overview]Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells
NCT01050764 (6) [back to overview]Acute Graft-versus-Host-Disease (aGvHD)
NCT01050764 (6) [back to overview]To Measure the Incidence and Severity of Acute and Chronic GvHD
NCT01078454 (1) [back to overview]Proportion of Patients With Hematologic Overall Response (Partial Response [PR]+ Very Good PR [VGPR]+ Amyloid Complete Response [ACR]+ Stringent Complete Response [sCR]) After 3 Months (3 Cycles) of Therapy
NCT01079936 (4) [back to overview]Number of Participants With Response (CR at Day 90)
NCT01079936 (4) [back to overview]Participants With Grade 3 =/> Adverse Events
NCT01079936 (4) [back to overview]Maximum Tolerated Dose (MTD) of Lenalidomide
NCT01079936 (4) [back to overview]Number of Participants With Day 30 DLT (Overall Study, Phase I/Phase II)
NCT01083316 (4) [back to overview]Number of Participants Surviving at 5 Years
NCT01083316 (4) [back to overview]Number of Participants With Disease Response
NCT01083316 (4) [back to overview]Number of Participants Proceeding to Transplant Following Induction
NCT01083316 (4) [back to overview]Number of Participants Surviving at 100 Days Post Transplant
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]Number of Participants With Acute and Chronic GVHD Following T-cell Depleted, CD34+ Progenitor Cell Enriched Transplants Fractionated by the CliniMACS System.
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]Survival and Disease-free Survival (DFS)
NCT01119066 (6) [back to overview]The Incidence of Durable Hematopoietic Engraftment for T-cell Depleted Transplants Fractionated by the CliniMACS System Administered After Each of the Four Disease Targeted Cytoreduction Regimens.
NCT01119066 (6) [back to overview]Incidence of Non-relapse Mortality (Transplant-related Mortality) Following Each Cytoreduction Regimen and a Transplant Fractionated by the CliniMACS System.
NCT01131169 (2) [back to overview]Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS)
NCT01131169 (2) [back to overview]Proportion of Participants With Relapsed Multiple Myeloma Alive at 2 Years
NCT01141712 (12) [back to overview]Immunologic Reconstitution
NCT01141712 (12) [back to overview]HIV Single-Copy Polymerase Chain Reaction (PCR)
NCT01141712 (12) [back to overview]Hematologic Function
NCT01141712 (12) [back to overview]Complete Remission (CR) and/or Partial Response (PR)
NCT01141712 (12) [back to overview]Relapse/Progression
NCT01141712 (12) [back to overview]Treatment-Related Mortality (TRM)
NCT01141712 (12) [back to overview]Progression-Free Survival (PFS)
NCT01141712 (12) [back to overview]Number of Participants Experiencing Toxicity
NCT01141712 (12) [back to overview]Number of Participants Experiencing Infections
NCT01141712 (12) [back to overview]Cumulative Incidence of Platelet Recovery
NCT01141712 (12) [back to overview]Cumulative Incidence of Neutrophil Recovery
NCT01141712 (12) [back to overview]Overall Survival (OS)
NCT01142232 (3) [back to overview]Phase I: Number of Patients With Dose Limiting Toxicity
NCT01142232 (3) [back to overview]Phase II: Treatment-Related Adverse Events Grade 3 or Higher
NCT01142232 (3) [back to overview]Phase II: Overall Response Rate
NCT01175356 (3) [back to overview]Percentage of MIBG Avid Patients Treated With Meta-iodobenxylguanide (MIBG) Labeled With Iodine-131
NCT01175356 (3) [back to overview]Percentage of MIBG Avid Patients Treated With MIBG Labeled With Iodine-131 and Bu/Mel Chemotherapy
NCT01175356 (3) [back to overview]Incidence of Unacceptable Toxicity and Sinusoidal Obstruction Syndrome (SOS), Assessed by Common Terminology Criteria (CTV)v.4.0 for Toxicity Assessment and Grading for I-MIBG
NCT01200329 (2) [back to overview]Overall Survival (OS) of These Patients.
NCT01200329 (2) [back to overview]Event-free Survival (EFS) of Patients
NCT01237951 (4) [back to overview]Overall Survival
NCT01237951 (4) [back to overview]Participants Who Had Measurable Disease at Time of Transplant and Achieved a Stringent Complete Remission
NCT01237951 (4) [back to overview]Progression-free Survival (PFS)
NCT01237951 (4) [back to overview]Percent of Participants Dying From Treatment-Related Complications
NCT01242267 (4) [back to overview]Treatment Free Interval/PFS
NCT01242267 (4) [back to overview]Toxicity Assessment
NCT01242267 (4) [back to overview]Maximum Tolerated Dose of Thalidomide
NCT01242267 (4) [back to overview]Complete Response (CR) and Very Good Partial Response (VgPR) Rate
NCT01256398 (6) [back to overview]Feasibility of Maintenance Therapy in This Patient Population (Restricted to Those Patients Achieving a CR). Feasibility Will be Defined as the Number of Deaths Ocuring.
NCT01256398 (6) [back to overview]Disease Free Survival Defined From the Date of First Induction Complete Response (CR) to Relapse or Death Due to Any Cause
NCT01256398 (6) [back to overview]Disease Free Survival (DFS)
NCT01256398 (6) [back to overview]Probability of Being BCR-ABL Negative in the Bone Marrow and Peripheral Blood at the Completion of the CNS Prophylaxis Course (Restricted to Those Patients Achieving a CR)
NCT01256398 (6) [back to overview]Overall Survival (OS)
NCT01256398 (6) [back to overview]Response
NCT01293539 (1) [back to overview]Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.
NCT01323517 (3) [back to overview]Progression Free Survival at One Year.
NCT01323517 (3) [back to overview]To Define the Immunologic Events and Signatures at the Tumor Site and in the Periphery That Corresponds to Response to Ipilimumab.
NCT01323517 (3) [back to overview]Toxicity of Additional Ipilimumab Will be Evaluated for All Treated Patients Using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0
NCT01335685 (27) [back to overview]AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Time to Progression (TTP) (Phase 2)
NCT01335685 (27) [back to overview]Time to First Response (Phase 2)
NCT01335685 (27) [back to overview]Progression Free Survival (Phase 2)
NCT01335685 (27) [back to overview]Overall Survival (Phase 2)
NCT01335685 (27) [back to overview]Overall Response Rate (ORR)
NCT01335685 (27) [back to overview]Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Duration of Response (DOR) (Phase 2)
NCT01335685 (27) [back to overview]Very Good Partial Response (VGPR) or Better Response Rate (Phase 2)
NCT01335685 (27) [back to overview]Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)
NCT01335685 (27) [back to overview]Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)
NCT01335685 (27) [back to overview]Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)
NCT01335685 (27) [back to overview]Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
NCT01335685 (27) [back to overview]Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)
NCT01335685 (27) [back to overview]Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)
NCT01339910 (10) [back to overview]Number of Participants With Donor Cell Engraftment
NCT01339910 (10) [back to overview]Percentage of Participants With Neutrophil and Platelet Engraftment
NCT01339910 (10) [back to overview]Percentage of Participants With Acute Graft Versus Host Disease (GVHD)
NCT01339910 (10) [back to overview]Percentage of Participants With Treatment-related Mortality
NCT01339910 (10) [back to overview]Percentage of Participants With Relapse-Free Survival (RFS)
NCT01339910 (10) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT01339910 (10) [back to overview]Percentage of Participants With Chronic GVHD
NCT01339910 (10) [back to overview]Number of Participants With Secondary Graft Failure
NCT01339910 (10) [back to overview]Number of Participants With Primary Graft Failure
NCT01339910 (10) [back to overview]Percentage of Participants With Disease Relapse
NCT01408563 (12) [back to overview]1 Year Relapse Rate
NCT01408563 (12) [back to overview]100-day Treatment Related Mortality
NCT01408563 (12) [back to overview]Immune Reconstitution - Median CD4 Count at 12 Months
NCT01408563 (12) [back to overview]Median Thrombopoietin Levels After Transplant
NCT01408563 (12) [back to overview]Median Time to Neutrophil Engraftment
NCT01408563 (12) [back to overview]Median Time to Platelet Engraftment
NCT01408563 (12) [back to overview]Number of Participants With Primary Graft Failure
NCT01408563 (12) [back to overview]Rate of Post-transplant Lymphoma
NCT01408563 (12) [back to overview]Rates of Grade II-IV and Grade III-IV Acute Graft Versus Host Disease (GVHD) at 100 Days
NCT01408563 (12) [back to overview]Relapse-free Survival
NCT01408563 (12) [back to overview]Overall Survival
NCT01408563 (12) [back to overview]The Rate of Chronic GVHD
NCT01410344 (7) [back to overview]Chimerism
NCT01410344 (7) [back to overview]Percentage of Participants With Overall Survival
NCT01410344 (7) [back to overview]Percentage of Participants With Non-Relapse Mortality
NCT01410344 (7) [back to overview]Percentage of Participants Recovering Hematologic Function
NCT01410344 (7) [back to overview]Percentage of Participants With Relapse/Progression
NCT01410344 (7) [back to overview]Percentage of Participants With Chronic Graft-Versus-Host Disease (GVHD)
NCT01410344 (7) [back to overview]Percentage of Participants With Acute Graft-Versus-Host Disease (GVHD)
NCT01413178 (5) [back to overview]Number of Participants That Had Grade 3-4 Toxicities.
NCT01413178 (5) [back to overview]Number of Participants With Complete Response (CR)
NCT01413178 (5) [back to overview]Progression-Free Survival (PFS)
NCT01413178 (5) [back to overview]Treatment-Related Mortality (TRM) Between 2 Arms.
NCT01413178 (5) [back to overview]Overall Survival (OS)
NCT01453088 (2) [back to overview]Progression Free Survival Rate
NCT01453088 (2) [back to overview]Overall Survival Rate
NCT01453101 (3) [back to overview]Progression Free Survival
NCT01453101 (3) [back to overview]Overall Survival (OS)
NCT01453101 (3) [back to overview]Overall Response Rate
NCT01499147 (4) [back to overview]Number of Participants With Moderate to Severe (Grade 2-4) Acute Graft Versus Host Disease (GVHD).
NCT01499147 (4) [back to overview]Number of Participants With Engraftment.
NCT01499147 (4) [back to overview]Time to ANC and Platelet Engraftment
NCT01499147 (4) [back to overview]Participants With 100 Day Transplant-related Mortality.
NCT01518153 (2) [back to overview]Overall Survival (OS)
NCT01518153 (2) [back to overview]Success Rate
NCT01529827 (4) [back to overview]Clinical Response
NCT01529827 (4) [back to overview]Median Time to Neutrophil Engraftment
NCT01529827 (4) [back to overview]Transplant Related Mortality (TRM)
NCT01529827 (4) [back to overview]Progression Free Survival (PFS) at One Year
NCT01538472 (2) [back to overview]Overall Survival Median
NCT01538472 (2) [back to overview]3-Year Overall Survival
NCT01548573 (4) [back to overview]Event-Free Survival (EFS)
NCT01548573 (4) [back to overview]Overall Survival
NCT01548573 (4) [back to overview]Identification of Drug Resistant Genes
NCT01548573 (4) [back to overview]Number of Grade 3 Non-hematologic and Grade 4 Hematologic Serious Adverse Events Associated With the Addition of Bortezomib, Thalidomide, and Dexamethasone Into Autologous Transplant Regimens.
NCT01605032 (5) [back to overview]Overall Response Rate
NCT01605032 (5) [back to overview]Mortality
NCT01605032 (5) [back to overview]Rate of Complete Response as Determined by the IMWG Criteria
NCT01605032 (5) [back to overview]Progression-free Survival
NCT01605032 (5) [back to overview]Overall Survival
NCT01626092 (2) [back to overview]Transplant-Related Mortality
NCT01626092 (2) [back to overview]Donor (Allogeneic) Hematopoietic Engraftment
NCT01653418 (9) [back to overview]Number of Participants With Overall Survival (OS)
NCT01653418 (9) [back to overview]Number of Participants With Progression-free Survival (PFS)
NCT01653418 (9) [back to overview]Time to Platelet Engraftment After V-BEAM.
NCT01653418 (9) [back to overview]Overall Response Rate (ORR)
NCT01653418 (9) [back to overview]Toxicity of V-BEAM
NCT01653418 (9) [back to overview]Very Good Partial Response Rate (VGPR+nCR+sCR+CR)
NCT01653418 (9) [back to overview]Complete Response Rate (Complete Response + Stringent Complete Response)
NCT01653418 (9) [back to overview]Time to Neutrophil Engraftment After V-BEAM.
NCT01653418 (9) [back to overview]Treatment Related Mortality (TRM) of V-BEAM
NCT01658904 (2) [back to overview]Engraftment Failure Transplant Related Mortality
NCT01658904 (2) [back to overview]Number of Participants With Adverse Events
NCT01659658 (21) [back to overview]Change From Baseline in Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-Ntx) Score at Week 28 of the PFS Follow-up
NCT01659658 (21) [back to overview]Change From Baseline in SF-36 Physical Component Summary Score at Week 28 of the PFS Follow-up
NCT01659658 (21) [back to overview]Duration of Hematologic Response
NCT01659658 (21) [back to overview]EuroQol 5-Dimension 3-Level (EQ-5D-3L) Visual Analogue Scale Score
NCT01659658 (21) [back to overview]Number of Hospitalizations
NCT01659658 (21) [back to overview]Number of Participants With Serious Adverse Events (SAEs)
NCT01659658 (21) [back to overview]Overall Survival
NCT01659658 (21) [back to overview]Percentage of Participants With Best Vital Organ (Cardiac and/or Kidney) Response
NCT01659658 (21) [back to overview]Percentage of Participants With Complete Hematologic Response
NCT01659658 (21) [back to overview]Percentage of Participants With Overall Hematologic Response
NCT01659658 (21) [back to overview]Hematologic Disease Progression Free Survival
NCT01659658 (21) [back to overview]Progression Free Survival (PFS)
NCT01659658 (21) [back to overview]2-Year Vital Organ (Heart or Kidney) Deterioration and Mortality Rate
NCT01659658 (21) [back to overview]Change From Baseline in 36-item Short Form General Health Survey (SF-36) Mental Component Summary Score at Week 28 of the PFS Follow-up
NCT01659658 (21) [back to overview]Change From Baseline in Amyloidosis Symptom Scale Total Score at Week 28 of the PFS Follow-up
NCT01659658 (21) [back to overview]Plasma Concentration of Ixazomib
NCT01659658 (21) [back to overview]Number of Participants in Each Category of the EuroQol 5-Dimensional (EQ-5D) Questionnaire Score
NCT01659658 (21) [back to overview]Vital Organ Progression Free Survival
NCT01659658 (21) [back to overview]Time to Vital Organ (Heart or Kidney) Deterioration and Mortality Rate
NCT01659658 (21) [back to overview]Time To Treatment Failure (TTF)
NCT01659658 (21) [back to overview]Time To Subsequent Anticancer Treatment
NCT01690143 (5) [back to overview]Maximum Tolerated Dose (MTD) of Carfilzomib Plus Melphalan as Conditioning for Autologous Hematopoietic Cell Transplantation in Patients With Relapsed Multiple Myeloma(MM) [Phase I Portion of Study]
NCT01690143 (5) [back to overview]Frequency of Grades 3 and 4 Non-hematologic Adverse Events During the Transplant Component ( 135 Days)
NCT01690143 (5) [back to overview]Complete Response (CR) Rate.
NCT01690143 (5) [back to overview]Median Time for Neutrophil and Platelet Engraftment.
NCT01690143 (5) [back to overview]Very Good Partial Response (VGPR) Rate.
NCT01702961 (3) [back to overview]Median Days to Neutrophil Engraftment
NCT01702961 (3) [back to overview]Disease-free Survival
NCT01702961 (3) [back to overview]Number of Participants With Overall Best Response Achieved After Transplantation
NCT01731886 (5) [back to overview]Complete Response Rate
NCT01731886 (5) [back to overview]Overall Survival Rate (OS)
NCT01731886 (5) [back to overview]Progression Free Survival (PFS)
NCT01731886 (5) [back to overview]Progression Free Survival (PFS)
NCT01731886 (5) [back to overview]Overall Survival Rate (OS)
NCT01746173 (2) [back to overview]Induction Response
NCT01746173 (2) [back to overview]24-month Progression-Free Survival Rate
NCT01798004 (1) [back to overview]The Tolerability of BuMel Regimen
NCT01807611 (6) [back to overview]Overall Survival
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Successful Engraftment
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Malignant Relapse
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)
NCT01807611 (6) [back to overview]Event-free Survival
NCT01807611 (6) [back to overview]Number of Transplant Recipients With Transplant-related Mortality (TRM)
NCT01818752 (7) [back to overview]Number of Participants With Adverse Events
NCT01818752 (7) [back to overview]European Organisation for Research and Treatment of Cancer Quality of Life Core Module (EORTC QLQ-C30) Global Health Status/Quality of Life (QOL) Scores
NCT01818752 (7) [back to overview]Progression-Free Survival (PFS)
NCT01818752 (7) [back to overview]Percentage of Participants With ≥ Grade 2 Peripheral Neuropathy
NCT01818752 (7) [back to overview]Overall Survival (OS)
NCT01818752 (7) [back to overview]Overall Response Rate
NCT01818752 (7) [back to overview]Complete Response Rate
NCT01824693 (4) [back to overview]Percent Probability of Event-free Survival (EFS)
NCT01824693 (4) [back to overview]Number of Participants Who Experience Treatment-Related Mortality (TRM) by Day 100
NCT01824693 (4) [back to overview]Percent Probability of 18 Months-relapse Event Between Arms
NCT01824693 (4) [back to overview]Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
NCT01842308 (5) [back to overview]Complete Response Rate at Day 100
NCT01842308 (5) [back to overview]Adverse Event Rate, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0
NCT01842308 (5) [back to overview]Progression Free Percentage at 1 and 2 Years Post Registration
NCT01842308 (5) [back to overview]Percentage of Patients With Complete Responses, Defined as a Complete Response Noted as the Objective Status on Two Consecutive Evaluations (Phase II)
NCT01842308 (5) [back to overview]Determine Maximum Tolerated Dose by the Number of Patients With a DLT Per Dose Level
NCT01849783 (4) [back to overview]Percentage of Participants Able to Complete Full Course Therapy
NCT01849783 (4) [back to overview]Median Progression Free Survival (mPFS)
NCT01849783 (4) [back to overview]Mean Change in Quality-Of-Life Indicators Post-Transplant
NCT01849783 (4) [back to overview]Percentage of Participants With Serious Treatment-Related Complications
NCT01857934 (6) [back to overview]Overall Response Rate [Complete Response + Very Good Partial Response + Partial Response (CR + VGPR + PR)]
NCT01857934 (6) [back to overview]Local Failure Rate and Pattern of Failure
NCT01857934 (6) [back to overview]Feasibility of Delivering hu14.18K322A to 6 Cycles of Induction Therapy
NCT01857934 (6) [back to overview]Event-free Survival (EFS)
NCT01857934 (6) [back to overview]Dose Limiting Toxicity (DLT) or Severe (Grade 3 or 4) VOD With hu14.18K322A With Allogeneic NK Cells in Consolidation
NCT01857934 (6) [back to overview]Dose Limiting Toxicity (DLT)
NCT01875237 (8) [back to overview]Number of Participants Assessed Post Donor Lymphocyte Infusion (DLI): Disease-free Survival & Non-relapse Mortality, Chimerism and GVHD.
NCT01875237 (8) [back to overview]To Assess the Incidence of Acute GvHD Flare After CR/PR Requiring Additional Agent for Systemic Therapy Before Day 56 Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Evaluate the Safety of Donor Lymphocyte Infusion Followed by Dimerizer Drug, AP1903 by Number of Participants With Adverse Events.
NCT01875237 (8) [back to overview]To Assess Post Donor Lymphocyte Infusion (DLI) Chimerism
NCT01875237 (8) [back to overview]To Assess the Proportions of GvHD Response Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Assess the Incidence of GvHD Treatment Failure Post-administration of AP1903.
NCT01875237 (8) [back to overview]To Assess the Proportion of Patients Developing Grade I-IV Acute GvHD
NCT01875237 (8) [back to overview]To Assess the Incidence of Epstein-Barr Virus -PTLD or EBV Reactivation Requiring Therapy Post DLI.
NCT01877837 (2) [back to overview]Number of Participants With Graft Failure
NCT01877837 (2) [back to overview]Overall Survival
NCT01885689 (2) [back to overview]Overall Survival at 2 Years
NCT01885689 (2) [back to overview]Progression-free Survival at 2 Years
NCT01921387 (4) [back to overview]Progression-free Survival Following Autologous Stem Cell Transplant (ASCT)
NCT01921387 (4) [back to overview]The Lowest Antibody (Yttrium 90-BC8-DOTA) Dose (mg/kg) That is Consistent With a Favorable Biodistribution Rate >= 80% in Lymphoma Patients
NCT01921387 (4) [back to overview]Maximum-tolerated Dose (MTD) of Yttrium-90-BC8-DOTA
NCT01921387 (4) [back to overview]Estimated Dose to Tumor Sites Based on the Tumor to Normal Organ Ratios Derived From Dosimetry Estimates Coupled With the Absorbed Dose to Normal Organs Based on the Administered Activity of Yttrium Y 90 Anti-CD45 Monoclonal Antibody BC8
NCT01969435 (11) [back to overview]Disease-free Survival
NCT01969435 (11) [back to overview]Disease-free Survival
NCT01969435 (11) [back to overview]Safety and Toxicity as Measured by Treatment Related Non-hematologic Adverse Events
NCT01969435 (11) [back to overview]Efficacy as Measured by Response Rates
NCT01969435 (11) [back to overview]Overall Survival (OS) Rate
NCT01969435 (11) [back to overview]Treatment-related Mortality (TRM)
NCT01969435 (11) [back to overview]Time to Engraftment (Platelet)
NCT01969435 (11) [back to overview]Time to Engraftment (Neutrophil)
NCT01969435 (11) [back to overview]Relapse Free Survival
NCT01969435 (11) [back to overview]Progression-free Survival Rate (PFS)
NCT01969435 (11) [back to overview]Progression-free Survival (PFS) Rate
NCT01983969 (2) [back to overview]Frequency of DLT
NCT01983969 (2) [back to overview]Participants With Event-free Survival (EFS)
NCT02007863 (1) [back to overview]Number of Successful Unrelated Cord Blood (UCB) Transplants
NCT02059239 (8) [back to overview]Disease Response 30 Days Post-Transplant
NCT02059239 (8) [back to overview]Disease Response at 1 Year Post-Transplant
NCT02059239 (8) [back to overview]Overall Survival at Day 365 Post-Transplant
NCT02059239 (8) [back to overview]Number of Patients Achieving Platelet Engraftment
NCT02059239 (8) [back to overview]Number of Patients Achieving Neutrophil Engraftment
NCT02059239 (8) [back to overview]Disease Response Following Salvage Chemotherapy
NCT02059239 (8) [back to overview]Progression-Free Survival After Stem Cell Transplant
NCT02059239 (8) [back to overview]Transplant-Related Mortality
NCT02097134 (4) [back to overview]Rate of Metastases of Retinoblastoma
NCT02097134 (4) [back to overview]Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy
NCT02097134 (4) [back to overview]Probability of Ocular Salvage
NCT02097134 (4) [back to overview]Number of Patients Experiencing Feasibility Failure
NCT02112045 (7) [back to overview]Number of Participants With Overall Response
NCT02112045 (7) [back to overview]Number of Participants With Platelet Engraftment
NCT02112045 (7) [back to overview]Overall Survival as Measured by Number of Participants Alive at Last Follow-up
NCT02112045 (7) [back to overview]Progression-free Survival as Measured by Number of Participants Without Disease Progression at Last Follow-up
NCT02112045 (7) [back to overview]Number of Participants With Adverse Events
NCT02112045 (7) [back to overview]Number of Participants With Neutrophil Engraftment
NCT02112045 (7) [back to overview]Number of Participants With Complete Response or Stringent Complete Response
NCT02128230 (1) [back to overview]The Remission Rate for Participants With High-risk Myeloma
NCT02195479 (16) [back to overview]Time to Response
NCT02195479 (16) [back to overview]Time to Next Treatment (TNT)
NCT02195479 (16) [back to overview]Time to Disease Progression (TTP)
NCT02195479 (16) [back to overview]Progression Free Survival on Next Line of Therapy (PFS2)
NCT02195479 (16) [back to overview]Progression Free Survival (PFS)
NCT02195479 (16) [back to overview]Percentage of Participants With Very Good Partial Response (VGPR) or Better
NCT02195479 (16) [back to overview]Percentage of Participants With Stringent Complete Response (sCR)
NCT02195479 (16) [back to overview]Percentage of Participants With Negative Minimal Residual Disease (MRD)
NCT02195479 (16) [back to overview]Percentage of Participants With Complete Response (CR) or Better
NCT02195479 (16) [back to overview]Overall Survival (OS)
NCT02195479 (16) [back to overview]Overall Response Rate (ORR)
NCT02195479 (16) [back to overview]Change From Baseline in EuroQol 5 Dimensions-5 Level (EQ-5D-5L) Utility Score
NCT02195479 (16) [back to overview]Duration of Response (DOR)
NCT02195479 (16) [back to overview]Percentage of Participants With Best M-protein Response
NCT02195479 (16) [back to overview]Change From Baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L): Visual Analogue Scale (VAS)
NCT02195479 (16) [back to overview]Change From Baseline in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ)-C30: Emotional Functioning Score
NCT02199041 (9) [back to overview]Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
NCT02199041 (9) [back to overview]Number of Participants With Transplant-related Mortality (TRM)
NCT02199041 (9) [back to overview]Number of Participants With Transplant-related Morbidity
NCT02199041 (9) [back to overview]Number of Participants With Secondary Graft Failure
NCT02199041 (9) [back to overview]Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT
NCT02199041 (9) [back to overview]Number of Participants With Event-free Survival (EFS)
NCT02199041 (9) [back to overview]Number of Participants With Malignant Relapse
NCT02199041 (9) [back to overview]Number of Participants With Neutrophil Engraftment
NCT02199041 (9) [back to overview]Number of Participants With Overall Survival (OS)
NCT02259348 (9) [back to overview]Incidence and Severity of Acute GvHD
NCT02259348 (9) [back to overview]Rate of Transplant-related Mortality (TRM)
NCT02259348 (9) [back to overview]Incidence of Malignant Relapse
NCT02259348 (9) [back to overview]Percentage of Participants Engrafted by Day 42 Post-transplant
NCT02259348 (9) [back to overview]Overall Survival (OS)
NCT02259348 (9) [back to overview]Median Days to Absolute Neutrophil Count (ANC) Engraftment
NCT02259348 (9) [back to overview]Mean of Days to Absolute Neutrophil Count (ANC) Engraftment
NCT02259348 (9) [back to overview]Event-free Survival (EFS)
NCT02259348 (9) [back to overview]Incidence and Severity of Chronic GvHD
NCT02331368 (3) [back to overview]The Estimated Percentage of Patients Alive at 2 Years
NCT02331368 (3) [back to overview]The Estimated Percentage of Patients Alive Without Relapse or Progression at 2 Years
NCT02331368 (3) [back to overview]The Number of Patients That Achieve Complete Response
NCT02366663 (4) [back to overview]Number of Patients With Grade 3-5 Toxicities Graded by the NCI CTCAE Version 4.0
NCT02366663 (4) [back to overview]Number of Patients With Complete or Partial Response at Day 30
NCT02366663 (4) [back to overview]Time to Neutrophil Engraftment
NCT02366663 (4) [back to overview]Time to Platelet Engraftment
NCT02435901 (3) [back to overview]Number of Participants With Sustained Cell Engraftment of Donor Cells
NCT02435901 (3) [back to overview]Event Free Survival; Number of Participants Who Survived at 2 Years
NCT02435901 (3) [back to overview]Assessment of Treatment Related Mortality and Morbidity
NCT02440464 (14) [back to overview]Percentage of Participants With Infections Post-randomization by Time Point
NCT02440464 (14) [back to overview]Percentage of Participants With Infections Post-randomization by Infection Type
NCT02440464 (14) [back to overview]Percentage of Participants With Disease Progression
NCT02440464 (14) [back to overview]Percentage of Participants With Chronic GVHD
NCT02440464 (14) [back to overview]Functional Assessment of Cancer Therapy (FACT) - Bone Marrow Transplant (BMT) Total Score
NCT02440464 (14) [back to overview]Percentage of Participants With Acute GVHD (Grades III-IV)
NCT02440464 (14) [back to overview]Medical Outcomes Study (MOS) - Short Form 36 (SF-36) Score
NCT02440464 (14) [back to overview]Percentage of Participants With Response to Treatment
NCT02440464 (14) [back to overview]Percentage of Participants With Best Response to Treatment After Randomization
NCT02440464 (14) [back to overview]Percentage of Participants With Treatment-Related Mortality (TRM)
NCT02440464 (14) [back to overview]Percentage of Participants With Toxicities Post-randomization by Toxicity Type
NCT02440464 (14) [back to overview]Percentage of Participants With Toxicities Post-randomization by Time Point
NCT02440464 (14) [back to overview]Percentage of Participants With Progression-Free Survival
NCT02440464 (14) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT02483000 (3) [back to overview]Incidence of Toxicity, Defined According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0
NCT02483000 (3) [back to overview]Dosimetry of Yttrium Y 90 DOTA-biotin
NCT02483000 (3) [back to overview]Overall Survival
NCT02489500 (3) [back to overview]Overall Survival
NCT02489500 (3) [back to overview]Number of Participants With Hematologic Response
NCT02489500 (3) [back to overview]Toxicities
NCT02497404 (9) [back to overview]Overall Survival at 6 Months Post-transplant
NCT02497404 (9) [back to overview]Overall Survival at 2 Years Post-Transplant
NCT02497404 (9) [back to overview]Overall Survival at 1 Year Post-Transplant
NCT02497404 (9) [back to overview]High-Risk Extensive Chronic Graft-versus-Host-Disease
NCT02497404 (9) [back to overview]Graft Failure
NCT02497404 (9) [back to overview]Disease Free Survival at 6 Months Post-transplant
NCT02497404 (9) [back to overview]Disease Free Survival at 2 Years Post-transplant
NCT02497404 (9) [back to overview]Disease Free Survival at 1 Year Post-transplant
NCT02497404 (9) [back to overview]Acute Graft-versus-Host Disease (GVHD)
NCT02581007 (4) [back to overview]GVHD Incidence
NCT02581007 (4) [back to overview]Relapse Incidence
NCT02581007 (4) [back to overview]Overall Survival
NCT02581007 (4) [back to overview]Graft Rejection
NCT02614560 (7) [back to overview]Best Response of CR or CRi
NCT02614560 (7) [back to overview]1-year Survival Rate
NCT02614560 (7) [back to overview]Duration of Response
NCT02614560 (7) [back to overview]Overall Survival
NCT02614560 (7) [back to overview]Incidence of Adverse Events
NCT02614560 (7) [back to overview]Incidence of Laboratory Abnormalities
NCT02614560 (7) [back to overview]Rate of MRD Negativity
NCT02669615 (3) [back to overview]Cmax (Pharmacokinetics)
NCT02669615 (3) [back to overview]AUC0-t (Pharmacokinetics)
NCT02669615 (3) [back to overview]Transplant-Related Mortality (TRM) Following Autologous Stem-Cell Transplantation (ASCT)
NCT02716805 (2) [back to overview]Number of Subjects With Treatment-emergent Adverse Events
NCT02716805 (2) [back to overview]Number of Subjects With Best Response According to International Myeloma Working Group (IMWG) Consensus Criteria
NCT02728102 (15) [back to overview]Number of Participants With Minimal Residual Disease (MRD)
NCT02728102 (15) [back to overview]Participants Response to Treatment
NCT02728102 (15) [back to overview]Percentage of Participants With Myeloma Progression of Vaccine and Non-vaccine Arms
NCT02728102 (15) [back to overview]Percentage of Participants With Myeloma Progression in Pairwise Analysis
NCT02728102 (15) [back to overview]Percentage of Participants With Grade 2 and 3 Infections in Pairwise Analysis
NCT02728102 (15) [back to overview]Percentage of Participants With Grade 2 and 3 Infections
NCT02728102 (15) [back to overview]Percentage of Participants With 1-year Response Rate of CR/sCR
NCT02728102 (15) [back to overview]Participants With Grade ≥ 3 Toxicities
NCT02728102 (15) [back to overview]Percentage of Participants With Treatment-related Mortality (TRM)
NCT02728102 (15) [back to overview]Percentage of Participants With Overall Survival in Pairwise Analysis
NCT02728102 (15) [back to overview]Percentage of Participants With Progression-Free Survival
NCT02728102 (15) [back to overview]Number of Grade ≥ 3 Toxicities
NCT02728102 (15) [back to overview]Number of Grade 2 and 3 Infections
NCT02728102 (15) [back to overview]Percentage of Participants With Overall Survival
NCT02728102 (15) [back to overview]Percentage of Participants With Progression-Free Survival in Pairwise Analysis
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - TREATMENT RELATED MORTALITY
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Event-free Survival (EFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - GENDER
NCT02756572 (19) [back to overview]Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 28
NCT02756572 (19) [back to overview]Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Relapsed-free Survival 6 Months After Transplant
NCT02756572 (19) [back to overview]Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Enrollment and Incidence of Early Transplant
NCT02756572 (19) [back to overview]Demonstrate the Feasibility of Collecting Patient-reported Outcomes for Trial Participants
NCT02756572 (19) [back to overview]Treatment Related Mortality Among Patients Who Received Early Transplant vs Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Relapse-free Survival (RFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Received Early Transplant.
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Received Early Transplant.
NCT02756572 (19) [back to overview]Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant
NCT02756572 (19) [back to overview]Event-free Survival (EFS) Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 84
NCT02756572 (19) [back to overview]Acute Graft Versus Host Disease Among Patients Who Received Early Transplant
NCT02756572 (19) [back to overview]Demonstrate the Feasibility of Collecting Resource Utilization Data for Trial Participants
NCT02756572 (19) [back to overview]Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - AGE
NCT02771197 (2) [back to overview]Number of Patients With 2-year Relapse Risk
NCT02771197 (2) [back to overview]Assess Safety of Pembrolizumab by Recording the Number of Participants With Treatment-related Adverse Events
NCT02780609 (5) [back to overview]Overall Survival (OS)
NCT02780609 (5) [back to overview]Phase 1 and Phase 2 Percentage of Participants Treated at Dose Level 3/RP2D With Progression Free Survival (PFS)
NCT02780609 (5) [back to overview]Phase I: Recommended Phase II Dose (RPh2D)
NCT02780609 (5) [back to overview]Rate of Minimal Residual Disease (MRD)
NCT02780609 (5) [back to overview]Complete Response (CR)
NCT02797470 (1) [back to overview]Percentage of Participants Who Achieve a Timely Engraftment
NCT02963493 (9) [back to overview]Overall Response Rate (ORR)
NCT02963493 (9) [back to overview]Functional Status and Well-being: EQ-5D-3L
NCT02963493 (9) [back to overview]Functional Status and Well-being: EORTC QLQ-C30
NCT02963493 (9) [back to overview]Time to Response
NCT02963493 (9) [back to overview]Duration of Response
NCT02963493 (9) [back to overview]Clinical Benefit Rate
NCT02963493 (9) [back to overview]Overall Survival
NCT02963493 (9) [back to overview]Progression Free Survival (PFS)
NCT02963493 (9) [back to overview]Time to Progression
NCT02994784 (8) [back to overview]Number of Participants With Cardiac Dysfunction (New Arrhythmia)
NCT02994784 (8) [back to overview]Neutrophil Engraftment
NCT02994784 (8) [back to overview]Hematologic Overall Response Rate
NCT02994784 (8) [back to overview]Platelet Engraftment
NCT02994784 (8) [back to overview]Treatment Related Mortality
NCT02994784 (8) [back to overview]Participants With Peri-transplant Hospitalizations
NCT02994784 (8) [back to overview]Organ Response
NCT02994784 (8) [back to overview]Number of Participants With Renal Dysfunction
NCT03019640 (2) [back to overview]Number of Participants Who Survived
NCT03019640 (2) [back to overview]Treatment-related Mortality Within 30 Days (TRM30)
NCT03096782 (3) [back to overview]Overall Survival
NCT03096782 (3) [back to overview]Disease-free Survival
NCT03096782 (3) [back to overview]Time to Engraftment
NCT03128359 (3) [back to overview]Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year
NCT03128359 (3) [back to overview]Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading
NCT03128359 (3) [back to overview]Overall Survival (OS) at 1 Year
NCT03151811 (4) [back to overview]Overall Response Rate (ORR)
NCT03151811 (4) [back to overview]Progression Free Survival (PFS)
NCT03151811 (4) [back to overview]Safety and Tolerability: Number of Patients With Treatment-emergent Adverse Events, Including Clinical Laboratory and Vital Signs Abnormalities, as Assessed by CTCAE v4.0
NCT03151811 (4) [back to overview]Duration of Response (DOR)
NCT03412565 (14) [back to overview]Percentage of Participants With Anti-Daratumumab Antibodies
NCT03412565 (14) [back to overview]Maximum Observed Serum Concentration (Cmax) of Daratumumab
NCT03412565 (14) [back to overview]D-VMP, D-Rd, and D-Kd Cohorts: Overall Response Rate (ORR)
NCT03412565 (14) [back to overview]Maximum Observed Serum Concentration (Cmax) of Daratumumab
NCT03412565 (14) [back to overview]Maximum Observed Serum Concentration (Cmax) of Daratumumab
NCT03412565 (14) [back to overview]D-VRd Cohort: Overall Response Rate (ORR)
NCT03412565 (14) [back to overview]D-VRd Cohort: Percentage of Participants With Very Good Partial Response (VGPR) or Better Response
NCT03412565 (14) [back to overview]D-VMP, D-Rd and D-Kd Cohorts: Duration of Response (DOR)
NCT03412565 (14) [back to overview]D-VMP, D-Rd, and D-Kd Cohorts: Percentage of Participants With VGPR or Better Response
NCT03412565 (14) [back to overview]D-VMP, D-Rd, and D-Kd Cohorts: Percentage of Participants With Minimal Residual Disease (MRD) Negative Rate
NCT03412565 (14) [back to overview]Maximum Observed Serum Concentration (Cmax) of Daratumumab
NCT03412565 (14) [back to overview]Percentage of Participants With Infusion-Related Reactions (IRRs)
NCT03412565 (14) [back to overview]Percentage of Participants With CR or Better Response
NCT03412565 (14) [back to overview]Percentage of Participants With Anti-rHuPH20 Antibodies
NCT03481556 (12) [back to overview]Progression-Free Survival (PFS)
NCT03481556 (12) [back to overview]Clinical Benefit Rate (≥ Minimal Response)
NCT03481556 (12) [back to overview]Time to Next Treatment (TTNT)
NCT03481556 (12) [back to overview]Time to Progression (TTP)
NCT03481556 (12) [back to overview]Time to Response (TTR)
NCT03481556 (12) [back to overview]Best Response (BR)
NCT03481556 (12) [back to overview]Duration of Clinical Benefit (DOCB)
NCT03481556 (12) [back to overview]Duration of Response (DOR)
NCT03481556 (12) [back to overview]Overall Survival (OS)
NCT03481556 (12) [back to overview]Phase 1: Number of Participants Who Experienced a Dose Limiting Toxicity (DLT)
NCT03481556 (12) [back to overview]Phase 2: Overall Response Rate (ORR)
NCT03481556 (12) [back to overview]Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE)
NCT03639610 (14) [back to overview]Tmax of Melphalan
NCT03639610 (14) [back to overview]Best Confirmed Response
NCT03639610 (14) [back to overview]Area Under the Curve (Inf) of Melphalan
NCT03639610 (14) [back to overview]Area Under the Curve (0-t) of Melphalan
NCT03639610 (14) [back to overview]Time to Response
NCT03639610 (14) [back to overview]Time to Clinical Benefit
NCT03639610 (14) [back to overview]Cmax of Melphalan
NCT03639610 (14) [back to overview]Clinical Benefit Rate
NCT03639610 (14) [back to overview]Duration of Clinical Benefit
NCT03639610 (14) [back to overview]Progression-free Survival
NCT03639610 (14) [back to overview]Duration of Response
NCT03639610 (14) [back to overview]Overall Response Rate
NCT03639610 (14) [back to overview]Overall Survival
NCT03639610 (14) [back to overview]T1/2 of Melphalan
NCT03786783 (5) [back to overview]Response Rate
NCT03786783 (5) [back to overview]Percentage of Participants With Unacceptable Toxicity
NCT03786783 (5) [back to overview]Overall Survival
NCT03786783 (5) [back to overview]Event-free Survival
NCT03786783 (5) [back to overview]"Percentage of Participants Who Are Feasibility Failure"
NCT04205240 (2) [back to overview]Number of Patients With a Partial Response
NCT04205240 (2) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)
NCT04339101 (3) [back to overview]Cumulative Incidence of Grade II-IV Acute GVHD
NCT04339101 (3) [back to overview]Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year
NCT04339101 (3) [back to overview]Progression Free Survival (PFS)
NCT04395222 (2) [back to overview]Proportion of Failure of the Haplo-Graft
NCT04395222 (2) [back to overview]Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment
NCT04412707 (20) [back to overview]Best Response (Stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Minimal Response (MR), Stable Disease (SD) or Progressive Disease (PD)
NCT04412707 (20) [back to overview]ORR
NCT04412707 (20) [back to overview]PFS
NCT04412707 (20) [back to overview]TTNT
NCT04412707 (20) [back to overview]TTP
NCT04412707 (20) [back to overview]TTR
NCT04412707 (20) [back to overview]Area Under the Plasma Concentration Versus Time Curve AUC(0-inf) of Melflufen and Desethyl-melflufen
NCT04412707 (20) [back to overview]Area Under the Plasma Concentration Versus Time Curve AUC(0-inf) of Melphalan
NCT04412707 (20) [back to overview]Area Under the Plasma Concentration Versus Time Curve AUC(0-t) of Melflufen and Desethyl-melflufen
NCT04412707 (20) [back to overview]Area Under the Plasma Concentration Versus Time Curve AUC(0-t) of Melphalan
NCT04412707 (20) [back to overview]Elimination Half-life (t1/2) of Melflufen, Melphalan and Desethyl-melflufen
NCT04412707 (20) [back to overview]Frequency of Treatment-Emergent Adverse Events (TEAEs) by MedDRA SOC and PT
NCT04412707 (20) [back to overview]Grade 3/4 Treatment-Emergent Adverse Events (TEAEs)
NCT04412707 (20) [back to overview]Grade 5 Treatment-Emergent Adverse Events (TEAEs)
NCT04412707 (20) [back to overview]Peak Plasma Concentration for Melflufen and Desethyl-melflufen
NCT04412707 (20) [back to overview]Peak Plasma Concentration for Melphalan
NCT04412707 (20) [back to overview]Number of Participants With Local Reactions Including Phlebitis at Infusion Site After Peripheral Intravenous Administration
NCT04412707 (20) [back to overview]CBR
NCT04412707 (20) [back to overview]DOCB
NCT04412707 (20) [back to overview]DOR
NCT04649060 (9) [back to overview]Progression Free Survival (PFS)
NCT04649060 (9) [back to overview]Overall Survival (OS)
NCT04649060 (9) [back to overview]Overall Response Rate (ORR)
NCT04649060 (9) [back to overview]Duration of Response (DOR)
NCT04649060 (9) [back to overview]Duration of Clinical Benefit (DOCB)
NCT04649060 (9) [back to overview]Clinical Benefit Rate (CBR)
NCT04649060 (9) [back to overview]Time to Response (TTR)
NCT04649060 (9) [back to overview]Time to Progression (TTP)
NCT04649060 (9) [back to overview]Time to Next Treatment (TTNT)
NCT04682405 (24) [back to overview]Change in Abdominal Pain as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Bristol Stool Scale
NCT04682405 (24) [back to overview]Change in Diarrhea as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Enterocolitis as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Esophageal Pain as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Esophagitis as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Gastritis as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Oral Mucositis as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Proctitis as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Vomiting as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0
NCT04682405 (24) [back to overview]Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0
NCT04682405 (24) [back to overview]Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0
NCT04682405 (24) [back to overview]Change in Nutritional Status as Assessed by Change in Standing Weight
NCT04682405 (24) [back to overview]Median Daily Dose of Anti-diarrheal Medications
NCT04682405 (24) [back to overview]Duration of Hospital Length of Stay
NCT04682405 (24) [back to overview]Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days
NCT04682405 (24) [back to overview]Median Daily Dose of Pain Medications
NCT04682405 (24) [back to overview]Change in Hemorrhoids as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Change in Nausea as Assessed Per CTCAE v5.0
NCT04682405 (24) [back to overview]Time to First Antibiotics
NCT04682405 (24) [back to overview]Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available)
NCT04682405 (24) [back to overview]Time to Neutrophil Engraftment
NCT04682405 (24) [back to overview]Incidence of Clostridium Difficile Infections

Toxicities Counts

Number of patients with grade 3 and 4 toxicities observed during cycles 1 & 2 using the Common Toxicity Criteria Version for Chemotherapy. (NCT00002601)
Timeframe: 2 months after completion of second cycle of treatment.

,
InterventionParticipants (Count of Participants)
AnemiaLeukopeniaNeutropeniaFebrile neutropeniaThrombocytopeniaMucositisBacteremia/sepsisHyperglycemiaPulmonary function impairment (FEV1)Pulmonary infiltratesDiarrheaArrhythmiaHematuriaMoodElevated PTT
Cycle 181313131313251011111
Cycle 2311119111410201010

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5-year Progression-free Survival

Estimated using the product-limit method of Kaplan and Meier. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST), as a 25% increase in the sum of the longest diameter of target lesions, or the appearance of new lesions. (NCT00002601)
Timeframe: Until disease progression, up to 5 Years

Interventionpercentage of participants (Number)
Doxorubicin/Ifosfamide + Melphalan/CDDP + PSCT23

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Number of Participants With Grade 3 Bilirubin

Criteria for early termination of this feasibility study: > 2 patients experience grade 4 or 5 hematologic toxicity or more that 3 patients experience grade 3 hematologic toxicity; > 2 patients experience grade 3 hepatic or gastrointestinal toxicity or > 3 patients are unable to receive the second cycle of treatment; > 2 patients experience grade 5 toxicity related to treatment regimen. (NCT00002601)
Timeframe: 2 years after completion of treatment

Interventionparticipants with Grade 3 Bilirubin (Number)
Doxorubicin/Ifosfamide + Melphalan/CDDP + PSCT3

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5-year Overall Survival

Estimated using the product-limit method of Kaplan and Meier. (NCT00002601)
Timeframe: Until death from any cause, up to 5 years

Interventionpercentage of participants (Number)
Doxorubicin/Ifosfamide + Melphalan/CDDP + PSCT31

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Three-year Relapse-free Survival

Estimated using the product-limit method of Kaplan and Meier. Relapse defined as appearance of any new lesions during or after protocol treatment. (NCT00003042)
Timeframe: From date of mastectomy until date of relapse or death from any cause, 3 years post mastectomy.

Interventionpercentage of participants (Number)
Neoadjuvant Chemo Followed by Surgery & High-dose Chemo With PSC Rescue61.5
High-dose Chemo With Rescue77.8

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Five-year Overall Survival

Estimated using the product-limit method of Kaplan and Meier. Endpoint is defined as death due to any cause. (NCT00003042)
Timeframe: From date of mastectomy until date of death, 5 years post mastectomy.

Interventionpercentage of participants (Number)
Neoadjuvant Chemo Followed by Surgery & High-dose Chemo With PSC Rescue69.2
High-dose Chemo With Rescue70.4

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Event-free Survival

Event-free survival of patients treated for inflammatory (Stage IIIb) and responsive stage IV breast cancer with BUMELTT and PBSC support and low dose immunotherapy with IL2 and GM-CSF. (NCT00003199)
Timeframe: 11 years

InterventionParticipants (Count of Participants)
Stage IIIB DiseaseStage IV Disease
TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer119

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Number of Participants With Toxicity of a Combination of Low-dose IL-2 and GM-CSF

IL-2/GM-CSF toxicity assessed using the NCI Toxicity Criteria. Toxicity was defined as any grade 2, 3, 4 or 5 CNS (except grade 0-3 malaise and fatigue) toxicity; any grade 3, 4, or 5 non-CNS or non-hematological toxicity (except grade 0-3 bilirubin); or any grade 4 or 5 hematological toxicity. (NCT00003199)
Timeframe: 16 Weeks

InterventionParticipants (Count of Participants)
TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer6

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Overall Survival

Overall survival of patients treated for inflammatory (Stage IIIb) and responsive stage IV breast cancer with BUMELTT and PBSC support and low dose immunotherapy with IL2 and GM-CSF. (NCT00003199)
Timeframe: 11 years

InterventionParticipants (Count of Participants)
TX/Maintenance Therapy for Stage IIIB/IV Breast Cancer18

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Objective Response

Determine the overall objective response. CR rate [as measured from the start of ICE, (or high dose CTX) to the end of transplant for those who receive it, or the end of ICE for those who do not].Complete response (CR): No evidence of Hodgkin's disease determined clinically, radiologically or pathologically when indicated (NCT00003631)
Timeframe: 2 years

,,
Interventionparticipants (Number)
FailureMinor Response (MR)Partial Response (PR)Progression Free (P-Free)Complete Response (CR)Progression of Disease (POD)
Group A - Favorable Prognostic Group3214100
Group B - Intermediate Prognostic Group6123121
Group C - Unfavorable Prognostic Group304710

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4 Year PFS

progression free survival estimate at 4 years post BMT (events are disease progression/relapse and death due to any cause) (NCT00003816)
Timeframe: 4 years

Interventionpercentage of participants (Number)
BuCy49.1
CyTBI52.2
FluMel33.2
VpCyTBI26.1
Other40

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4 yr OS

Overall survival estimate at 4 years post BMT (NCT00003816)
Timeframe: 4-year

Interventionpercentage of participants (Number)
BuCy56.4
CyTBI56.5
FluMel38.2
VpCyTBI39.1
Other53.3

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CR Rate

Rate of Complete Remission by Day +100 (NCT00003816)
Timeframe: day 100

InterventionParticipants (Count of Participants)
BuCy42
CyTBI55
FluMel134
VpCyTBI18
Other7

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Toxicity/TRM at Day 100

Death due to treatment related causes before day +100 after BMT (NCT00003816)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
BuCy3
CyTBI4
FluMel31
VpCyTBI4
Other4

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Three-year Overall Survival

Kaplan-Meier estimate at three years post-first transplant of survival. Outcome is death or alive at follow-up (censored). 95 percent confidence interval of the point estimate is calculated using Greenwood's variance. (NCT00004088)
Timeframe: Estimate reported at three years after day 0 of the first cycle of infusion of cells of the tandem transplant.

Interventionproportion of participants (Number)
HD Chemo Followed by PBPC Rescue0.662

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Progression-free Survival

"Kaplan-Meier estimate at three years post-first transplant of survival. Event of interest is the first of Death or Progression. Censoring is Alive in Continuous Complete Remission at date of last follow-up.~95 percent confidence interval of the point estimate is calculated using Greenwood's variance." (NCT00004088)
Timeframe: Estimate reported at three years after day 0 of the first cycle of infusion of cells of the tandem transplant.

Interventionproportion of participants (Number)
HD Chemo Followed by PBPC Rescue0.456

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Progression Free-survival (PFS)

Number of patients surviving without disease by interval. Chemosensitive and chemoresistant subjects will be analyzed separately. (NCT00005803)
Timeframe: From the date of autologous transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission, assessed up to 3 years

,,
InterventionParticipants (Count of Participants)
6 Months1 Year1.5 Years2 Years3 Years
Chemoresistant Group128888
Chemosensitive Group3627252522
Unknown Chemosensitivity Group10000

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Overall Survival (OS)

Number of patients surviving by interval. Chemosensitive and chemoresistant subjects will be analyzed separately. (NCT00005803)
Timeframe: From the date of autologous transplant until the time of death, assessed up to 3 years

,,
InterventionParticipants (Count of Participants)
6 Months1 Year1.5 Years2 Years3 Years
Chemoresistant Group1410988
Chemosensitive Group3931272623
Unknown Chemosensitivity Group10000

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Non-Relapse Mortality

"The rates and accompanying confidence intervals associated with transplant-related mortality will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 100 post-non-myeloablative allografting following mobilization and high-dose chemotherapy with autografting

InterventionParticipants (Count of Participants)
Treatment (Tandem Transplantation)3

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Engraftment of HLA Identical PBSC Allografts

"Number of patients who engrafted by Day 56 post allogeneic transplant. Failure to engraft is defined as the absence of detectable donor cells in the marrow. The rates and accompanying confidence intervals associated with failure of engraftment at day +56 will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 56

InterventionParticipants (Count of Participants)
Treatment (Tandem Transplantation)53

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Proportion of Patients Alive and in Remission

(NCT00006244)
Timeframe: 12.9 Median Years

InterventionParticipants (Count of Participants)
Immunotherapy Treatment4

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Time to Disease Progression

(NCT00006244)
Timeframe: 12.9 years (median)

Interventionyears (Median)
Immunotherapy Treatment1.61

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Initial Response to Therapy

Evaluate initial response to therapy (complete remission, partial remission, stable response, or progression of disease) (NCT00006244)
Timeframe: Evaluated at Day +84-90 Post-Transplant

InterventionParticipants (Count of Participants)
Patients in Complete RemissionPatients in Partial RemissionPatients with Stable resposnePatients with Disease Progression
Immunotherapy Treatment158103

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Overall Survival

Overall survival in Multiple Myeloma patients treated with melphalan, IL2-incubated peripheral blood stem cells, and sequential IL2 and interferon maintenance. (NCT00006244)
Timeframe: 12.9 Median Years

InterventionParticipants (Count of Participants)
Immunotherapy Treatment9

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Overall Survival

(NCT00027560)
Timeframe: 24 months post transplant

Interventionparticipants (Number)
TREATMENT OF LYMPHOHEMATOPOIETIC MALIGNANCIES30

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Overall Survival

(NCT00027560)
Timeframe: 12 months post transplant

Interventionparticipants (Number)
TREATMENT OF LYMPHOHEMATOPOIETIC MALIGNANCIES37

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Number of Participants With Absolute Neutrophil Count Engraftment

Absolute neutrophil engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Baseline to Day 30 post transplant. (NCT00038857)
Timeframe: Day 0 up to Day 30

Interventionparticipant (Number)
Melphalan + Thiotepa + Fludarabine + Rabbit ATG + CD34 PBPC21

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Overall Survival

(NCT00040937)
Timeframe: 4-7 years

Interventionproportion surviving at 4 years (Number)
Treatment Arm.64

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Assess Toxicity of Thalidomide/Dexamethasone as a Pre-transplant Induction Regimen.

To assess Grade 3-5 AE related to thalidomide/dexamethasone when administered as a pre-transplant induction regimen. (NCT00040937)
Timeframe: Induction

InterventionParticipants (Number)
Induction/PBSC Mobilization2

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Toxicity

Here is the number of participants with adverse events. For a detailed list of adverse events see the adverse event module. (NCT00043979)
Timeframe: 16.5 months

InterventionParticipants (Number)
Arm 2-Recipients30

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Number of Participants With Engraftment

Engraftment is defined as rapid conversion to complete donor chimerism and is assessed by blood counts and chimerism, >95% donor engraftment at day 100 in >75% of patients. (NCT00043979)
Timeframe: 100 days

InterventionParticipants (Number)
Arm 2-Recipients23

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Number of Participants Who Experienced Graft Versus Tumor Effect (GVT)

GVT is defined as tumor response after day 42 post-transplantation without cytotoxic therapy. (NCT00043979)
Timeframe: up to day 100

InterventionParticipants (Count of Participants)
Arm 2-Recipients0

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Median Progression Free Survival

Progression free survival was based on the time from on-study date until progression or last follow-up. (NCT00043979)
Timeframe: up to 77 months

InterventionMonths (Median)
Arm 2-Recipients15.9

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Early Post Transplantation Relapse

Participants who experienced recurrence or progression of disease following transplant. (NCT00043979)
Timeframe: up to 300 days

InterventionDays (Median)
Arm 2-Recipients100

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Cluster of Differentiation 4 (CD4) Reconstitution

The median CD4 count with a range of 85-1565 (absolute count) was used to determine recovery and were considered recovered if in this range. The CD4 count was established by flow cytometry testing. (NCT00043979)
Timeframe: Day +28-42

Interventionmm(3) (Median)
Arm 2-Recipients284

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Median Time to Reach Absolute Neutrophil Count of 500/mm(3)

Days for participants to achieve a neutrophil count of 500/mm(3). (NCT00043979)
Timeframe: up to 12 days

InterventionDays (Median)
Arm 2-Recipients9

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Median Survival From Date of Progression

Median survival from date of progression is based on the time from on-study date until progression or last follow-up. (NCT00043979)
Timeframe: up to 77 months

InterventionMonths (Median)
Participants who did not receive a transplant(n=7)Participants who received a transplant (n=23)
Arm 2-Recipients3.319.1

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Number of Participants to Complete Conversion to >95% Donor Chimerism

Participants who tolerated the transplantation regimen and accepted >95% of the donors blood, marrow, and/or tissue. (NCT00043979)
Timeframe: up to 30 days

InterventionParticipants (Count of Participants)
Day +14Day +28
Arm 2-Recipients2323

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Number of Participants With Acute and Chronic GVHD

Acute GVHD as by Modified Glucksberg Criteria occurring before day 100. Chronic GVHD as per Seattle criteria occurring after day 100. (NCT00043979)
Timeframe: up to 5 years or death

,
Interventionparticipants (Number)
acute GVHDchronic GVHD
Recipients -Cyclosporine GVHD Prophylaxis1212
Recipients -Tacrolimus/Sirolimus GVHD Prophylaxis55

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Median Time to Reach a Platelet Count of 50,000/mm(3)

Days for participants to achieve a platelet count of 50,000/mm(3). (NCT00043979)
Timeframe: up to 43 days

InterventionDays (Median)
Arm 2-Recipients15

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Post-Hematopoietic Stem Cell Transplant (HSCT) Radiotherapy

Site of radiotherapy (high energy radiation) and/or toxicity experienced by the participants post HSCT radiotherapy. Grading was preformed using the Modified Glucksberg Criteria. (NCT00043979)
Timeframe: up to 6 cycles or 168 days

InterventionParticipants (Count of Participants)
Chest wall; G2 skinAbdomen; G4 GIPancreas; G4 LFTs, G4 pancreatitisPleura, mediastinum; G4 LFTs, G2 mucositisChest wall; G4 skin, G3 mucositisSpine, skull; G2 nausea+vomiting, G2 fatiguePelvis; G4 enteritisPulmonary (cyberknife)Brain; B3 mucositisWhole lung; G3 mucositis, G3 skin, G5 lungL arm, R shoulder, B/L femur
Arm 2-Recipients11111111111

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Two Year Survival Rate for Patients Undergoing Allo-Hematopoietic Stem Cell Transplant

Participants who are alive at two years following Allo-Hematopoietic Stem Cell Transplant. (NCT00043979)
Timeframe: 2 years

Interventionpercentage of participants (Number)
From date of enrollmentFrom date of transplantation
Arm 2-Recipients39.134.8

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Best Response Post-Hematopoietic Stem Cell Transplant EOCH (Etoposide, Vincristine, Cyclophosphamide, and Doxorubicin)

Response is defined by the Response Evaluation Criteria in Solid Tumors (RECIST). RECIST criteria offer a simplified, conservative, extraction of imaging data for wide application in clinical trials. They presume that linear measures are an adequate substitute for 2-D (dimensional) methods and registers four response categories: Complete response (CR) is disappearance of all target lesions. Partial response (PR) is 30% increase in the sum of the longest diameter of target lesions. Progressive disease (PD) is 20% increase in the sum of the longest diameter of target lesions. Stable disease (SD) is small changes that do not meet above criteria. For the purposes of this study very good partial response ((VGPR) is >75% reduction in disease) was also employed. (NCT00043979)
Timeframe: up to 10 cycles of therapy or 280 days

InterventionParticipants (Count of Participants)
Complete Response (CR)Progressive Disease (PD)Partial Response (PR)Very Good Partial Response (VGPR)
Arm 2-Recipients2442

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OS

Number of subjects surviving. OS will be estimated by the method of Kaplan and Meier. Confidence intervals will be estimated. (NCT00054353)
Timeframe: At 6 months and then every year thereafter, up to 5 years

,
InterventionParticipants (Count of Participants)
6 Months1 Year2 Years3 Years4 Years5 Years
Related Donor854333
Unrelated Donor320000

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Relapse Rate

Number of subjects who relapsed after achieving CR post-transplant. Relapse rate will be summarized using cumulative incidence estimates. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor1
Unrelated Donor1

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Engraftment

Number of subjects who engrafted post-transplant. Engraftment will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor12
Unrelated Donor4

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PFS

PFS will be calculated for all patients from the date of transplant until the time of progression, relapse, death, or the date the patient was last known to be in remission. Progressive disease is defined as greater than 25% increase in serum or urine M proteins compared to best response status after autologous transplant and/or appearance of new lytic bone lesions or plasmocytomas. (NCT00054353)
Timeframe: At 1 year post-transplant

InterventionParticipants (Count of Participants)
Related Donor4
Unrelated Donor1

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Response Rate

Number of subjects who achieved CR post-transplant. Response rate will be summarized using cumulative incidence estimates. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor2
Unrelated Donor1

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Incidence of Acute GVHD (Grades III-IV)

Number of patients with Grade III-IV acute GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor2
Unrelated Donor0

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Incidence of Chronic (Extensive) GVHD

Number of subjects with chronic extensive GVHD post-transplant. Severe GVHD will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Related Donor4
Unrelated Donor1

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Non-relapse Mortality

Early NRM will be monitored in a sequential fashion. (NCT00054353)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Related Donor1
Unrelated Donor1

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Hematologic Response

(NCT00064337)
Timeframe: Until off study

InterventionParticipants (Count of Participants)
Treatment8

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Overall Survival

Time from initial registration until death or date of last contact, whichever occurs first, for up to 5 years from the date of the last patient registration. (NCT00064337)
Timeframe: 5 years from initial registration, or until death, whichever occurred earlier, on average, about 4.5 years

InterventionMonths (Median)
Treatment68

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Time To Neutrophil Engraftment

Engraftment is defined as a sustained ANC > 0.5 x 10^9/L for at least 3 consecutive days. Engraftment date is the first of the 3 days with sustained absolute neutrophil count (ANC) >/= 0.5 x 10^9/L. (NCT00067002)
Timeframe: First 100 days, evaluation and blood tests twice weekly

InterventionDays (Median)
Un-Manipulated CB Arm17
Expanded CB Arm15

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Number of Participants Severity of Acute GVHD by Treatment Arm

The severity of acute GVHD is determined by an assessment of the degree of involvement of the skin, liver, and gastrointestinal tract. The stages of individual organ involvement is assessed using Glucksberg grade (I-IV) where Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT00067002)
Timeframe: Following first 100 days, up to one year

,
InterventionParticipants (Count of Participants)
Grade ≤ 2Grade ≥ 3No GVHD
Expanded CB Arm15227
Un-Manipulated CB Arm20619

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Rate of Chronic GVHD

Number of participants who present GVHD post-transplant and display features of chronic GVHD. Diagnostic and distinctive features of chronic GVHD are present. There are no features of acute GVHD. (NCT00067002)
Timeframe: Up to one year

,
InterventionParticipants (Count of Participants)
Overall Chronic GVHDLimitedExtensive
Expanded CB Arm201413
Un-Manipulated CB Arm16109

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Number of Participants With Engraftment

Engraftment defined as a sustained absolute neutrophil count (ANC) > 0.5 x 10^9/L for at least 3 consecutive days. Engraftment Failure defined as ANC <500/ul by day +42 and participant has no evidence of donor chimerism on bone marrow examination. (NCT00067002)
Timeframe: First 100 days, evaluation and blood tests twice weekly

InterventionParticipants (Count of Participants)
Un-Manipulated CB Arm45
Expanded CB Arm44

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Rate of Acute Graft Versus Host Disease (GVHD)

Number of participants who display features of acute GVHD within 100 days of transplant. (NCT00067002)
Timeframe: Review over first 100 days

InterventionParticipants (Count of Participants)
Un-Manipulated CB Arm29
Expanded CB Arm24

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Frequency of the Induction of Full Donor Chimerism of Lymphocytes as Measured at 1 Month Post Allografting

(NCT00074269)
Timeframe: 1 month post allografting

InterventionParticipants (Count of Participants)
Treatment5

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Number of Participants With Acute and Chronic Graft-versus-host Disease (GVHD)

(NCT00074269)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Treatment3

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Number of Participants With Adverse Events

(NCT00074269)
Timeframe: 5 years post transplant

InterventionParticipants (Count of Participants)
Treatment5

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Number of Participants With Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes

Long-term Engraftment of Allogeneic Stem Cells and Lymphocytes based on cell counts of ANC >1000 for 3 consecutive days and platelet count of >50,000 (NCT00074269)
Timeframe: 30 days post transplant

InterventionParticipants (Count of Participants)
Treatment5

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Progression-free Survival

Progression assessed by CT scan (NCT00074269)
Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 6 months

Interventiondays (Median)
Treatment110

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Response as Measured at 12 Months Post Allografting

response (partial and complete) assessed by CT scan at 12 months post allografting (NCT00074269)
Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 12 months

InterventionParticipants (Count of Participants)
Treatment3

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Overall Survival

(NCT00074269)
Timeframe: 1 year from the time of transplant

Interventiondays (Median)
Treatment279.4

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Overall Survival

Overall Survival at six years after initiating protocol therapy (NCT00083551)
Timeframe: 6 Years

Interventionpercentage of participants (Number)
Thalidomide65
No Thalidomide58

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Maximum Tolerated Dose (MTD)

The highest dose tested (Total Marrow Irradiation) in which there is no treatment related mortality and none or only one patient experienced dose limited toxicity (DLT) attributable to the study drug(s), when at least six were fully treated at that dose and fully followed for toxicity. The MTD is one dose level below the lowest dose tested in which 2 or more patients experienced DLT attributable to the treatment or there was a treatment related death. At least 6 patients will be treated at the MTD. (NCT00112827)
Timeframe: 8 weeks from start of treatment, up to 2 years

InterventioncGy (Number)
Treatment Arm1600

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Number of Subjects With Response

Response defined as complete response or very good partial response. Complete response defined as the absence of bone marrow or blood findings of multiple myeloma on at least 2 measurements at a minimum of a 6 week interval. Thus all evidence of serum and urinary M-components must disappear on electrophoresis as well as by immunofixation studies. The follow-up bone marrow may not contain more than 5% plasma cells on aspiration or core biopsy and no evidence of increasing anemia. Skeletal X-rays must either show recalcification or no change in osteolytic lesions. Resolution of soft tissue plasmocytomas. Very good partial response defined as reduction of bone marrow or blood findings of multiple myeloma on at least 2 measurements at a minimum of a 6 week interval by greater than or equal to 90%. (NCT00112827)
Timeframe: Evaluated after each course until completion of treatment.

InterventionParticipants (Count of Participants)
Phase 1 Cohort 1 (Total TMI Dose: 1000 cGy)3
Phase 1 Cohort 2 (Total TMI Dose: 1200 cGy)3
Phase 1 Cohort 3 (Total TMI Dose: 1400 cGy)2
Phase 1 Cohort 4 & Phase 2 MTD (Total TMI Dose:1600 cGy)20
Phase 1 Cohort 5 (Total TMI Dose: 1800 cGy)4

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Overall Survival

Estimated using the product-limit method of Kaplan and Meier. Event defined as death due to any cause. (NCT00112827)
Timeframe: From date of treatment until the date of death from any cause, assessed up to 14 years

Interventionmonths (Median)
Treatment Arm95.8

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Time to Progression

"Time to progression (TTP) was defined as the date of transplant to date of progression or death due to any cause, whichever occurs first. TTP was estimated using the Kaplan Meier method.~Progression was defined per the International Myeloma Working Group definition as one more of the following:~25% increase in serum M-component (absolute increase >= 0.5g/dl)~25% increase in urine M-component (absolute increase >= 200mg/24hour~25% increase in the difference between involved and uninvolved Free Light Chain levels (absolute increase >= 10mg/dl)~25 % increase in bone marrow plasma cell percentage (absolute increase of >=10%)~Definite development of new bone lesion or soft tissue plasmacytomas~Development of hypercalcemia" (NCT00114101)
Timeframe: Duration of study (up to 10years)

Interventionmonths (Median)
Lenalidomide Maintenance39
Placebo Maintenance21

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Response to Autologous Hematopoietic Stem-cell Transplant (HSCT) at Day 100

"Response was defined according to International Myeloma Working Group criteria (2006)~Complete Response: Complete disappearance of M-protein from serum & urine on immunofixation, normalization of Free Light Chain (FLC) ratio & <5% plasma cells in bone marrow (BM)~Partial Response: >= 50% reduction in serum M-Component and/or Urine M-Component >= 90% reduction or <200 mg per 24 hours; or >= 50% decrease in difference between involved and uninvolved FLC levels~Marginal Response: 25-49% reduction in serum M-component & urine M-component by 50-89% which still exceeds 200mg/24hour~Progressive Disease: Defined in primary outcome measure~Stable Disease: Not meeting any of the criteria above" (NCT00114101)
Timeframe: Day 100

,
Interventionparticipants (Number)
Complete responsePartial responseMarginal responseStable diseaseProgressive diseaseUnknown
Lenalidomide Maintenance67115113800
Placebo Maintenance7910953231

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Number of Participants With Progression, Death or Diagnosis of Second Primary Malignancy

Patients who develop progression (defined in primary outcome measure), died or develop a new primary malignancy (cancer) will summarized in this outcome. (NCT00114101)
Timeframe: Duration of study (up to 10 years)

Interventionparticipants (Number)
Lenalidomide Maintenance92
Placebo Maintenance133

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Overall Survival

Overall Survival was measured from the date of randomization to date of death due to any cause. OS was estimated using the Kaplan Meier method. (NCT00114101)
Timeframe: Duration of study (up to 10 years)

Interventionmonths (Median)
Lenalidomide MaintenanceNA
Placebo MaintenanceNA

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Progression-free Survival

PFS rate at 1 year. (NCT00121186)
Timeframe: 1, 3, and 12 months after protocol treatment, then every 3 months for 1 year, every 6 months for year 2, then annually thereafter until 5 years after registration

Interventionparticipants (Number)
Nonmyeloablative Allogeneic Stem Cell Transplant1

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Overall Survival

OS rate at 1 year. (NCT00121186)
Timeframe: 1, 3, and 12 months after protocol treatment, then every 3 months for 1 year, every 6 months for year 2, then annually thereafter until 5 years after registration

Interventionparticipants (Number)
Nonmyeloablative Allogeneic Stem Cell Transplant1

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Factors Affecting One-year Survival: Minimal Residual Disease (MRD)

Detection of leukemia blasts in bone marrow by flow cytometry (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
Negative for MRDPositive for MRD
Alive21
Expired10
Study Participants31

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Factors Affecting One-year Survival: Median Dose of CD34

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionCD34 X 10^6/kg (Median)
Alive35.2
Expired38.3
Study Participants37.8

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Factors Affecting One-year Survival: Median Dose of NK Cells

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionNKcells X 10^6/kg (Median)
Alive40.2
Expired37.6
Study Participants38.9

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One-year Survival

"The one-year survival of infants with high-risk hematologic malignancies who receive a haploidentical transplant procedure using a total body irradiation (TBI)-excluding conditioning regimen followed by an HLA-nonidentical family donor hematopoietic stem cell (HSC) graft depleted of T cells ex vivo using the CliniMACS CD34+ selection system, with a subsequent infusion of donor NK cells purified ex vivo using the CliniMACS CD3+ depletion and CD56+ enrichment system.~The Kaplan-Meier estimate for one-year survival is reported." (NCT00145626)
Timeframe: One year after transplant

Interventionpercentage of participants (Number)
Study Participants50

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Factors Affecting One-year Survival: Disease Status at HSCT

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
Active DiseaseComplete Remission-1Complete Remission-2Progressive DiseaseRelapse
Alive06010
Expired12103
Study Participants18113

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Factors Affecting One-year Survival: Donor Type

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
FatherMotherUncle
Alive250
Expired421
Study Participants671

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Factors Affecting One-year Survival: Match N/6 HLA Loci

HLA typing determined the degree of match by looking at 6 different HLA loci. The results indicate the number of the 6 loci that matched for each participant. Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

,,
Interventionparticipants (Number)
3/6 HLA Loci4/6 HLA Loci
Alive61
Expired34
Study Participants95

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Factors Affecting One-year Survival: Median Age of Donor at HSCT

Due to small sample size (n=14) and total number of events (n=7), the analysis to check the various factors that affect the one-year survival was not performed (using logistic and cox model). (NCT00145626)
Timeframe: Up to one year after transplant

InterventionYears (Median)
Alive21.5
Expired27.2
Study Participants25.73

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Kinetics of Lymphohematopoietic Reconstitution

The lymphohematopoietic reconstitution will be summarized using summary statistics and assessed in a longitudinal manner and analyzed accordingly. (NCT00145626)
Timeframe: From 0-3 months after HSCT through 4-5 years after HSCT

,,,,,,,
Interventioncells *10^3/µl (Median)
CD3 LymphocyteCD3 Gamma DeltaCD4 LymphocyteCD8 LymphocyteCD19 LymphocyteCD56 LymphocyteCD4/CD8 RatioAbsolute Lymphocyte Value
0-3 Months After HSCT0.220.000.130.010.260.362.600.88
1-2 Years After HSCT1.650.700.960.610.450.221.882.59
2-3 Years After HSCT2.880.241.561.050.560.321.383.76
3-4 Years After HSCT2.650.211.331.100.560.341.303.65
3-6 Months After HSCT0.570.040.420.090.480.335.531.29
4-5 Years After HSCT1.870.381.940.700.430.191.302.40
6-9 Months After HSCT1.150.080.920.290.610.203.361.95
9-12 Months After HSCT2.240.091.370.360.520.191.902.90

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Frequency of and Clinical Relevance of Minimal Residual Disease (MRD) Before and After Transplantation

The presence or absence of MRD before and after the bone marrow transplant (BMT) and its frequency distribution will be obtained for each time point separately. (NCT00145626)
Timeframe: Baseline before HSCT, 1 year post HSCT, and up to 5 years post HSCT

InterventionParticipants (Count of Participants)
Patient 172467502Patient 172467503Patient 172467501Patient 272467501Patient 272467502Patient 272467503Patient 372467501Patient 372467502Patient 372467503Patient 472467501Patient 472467502Patient 472467503
Data Not CollectedPositive MRDNegative MRD
Study Participants: 1 Year Post HSCT1
Study Participants: 5 Years Post HSCT1
Study Participants: 1 Year Post HSCT0
Study Participants: Before HSCT0
Study Participants: Before HSCT1
Study Participants: 5 Years Post HSCT0

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Incidence Chronic Graft-versus-host Disease (GVHD)

Number of participants with chronic GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies. (NCT00176839)
Timeframe: 1 year

Interventionparticipants (Number)
Treatment Arm0

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Incidence of Relapse

Number of patients with relapse after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies. (NCT00176839)
Timeframe: 1 year

Interventionparticipants (Number)
Treatment Arm2

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Probability of Long-term Disease-free Survival (DFS)

Number of participants with long-term disease free survival after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies. (NCT00176839)
Timeframe: 1 year

Interventionparticipants (Number)
Treatment Arm3

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Incidence of Acute Graft-versus-host Disease (GVHD)

Number of participants with acute GVHD after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies. (NCT00176839)
Timeframe: 100 days post-transplant

Interventionparticipants (Number)
Treatment Arm7

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Incidence of Regimen-related Toxicity 100 Days Post Transplant

Number of participants with regimen-related toxicity 100 days post transplant after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies. (NCT00176839)
Timeframe: 100 days post-transplant

Interventionparticipants (Number)
Treatment Arm3

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Probability of Engraftment

Number of participants with engraftment after being treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by HCT for hematological malignancies.. (NCT00176839)
Timeframe: 1 year

Interventionparticipants (Number)
Treatment Arm10

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Number of Subjects Alive at One Year

(NCT00176865)
Timeframe: Day 365

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor7
Arm 3 - Mismatched Double Cord Donors3

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Percentage of Donor Chimerism at 365 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 365

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor81.9
Arm 2 - Matched Unrelated Donor78.6
Arm 3 - Mismatched Double Cord Donors91.7

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Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD)

Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease. (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor3
Arm 3 - Mismatched Double Cord Donors0

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Number of Subjects Alive at 100 Days

(NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor8
Arm 3 - Mismatched Double Cord Donors4

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Incidence of Chronic Graft Versus Host Disease (cGVHD)

Chronic graft versus host disease (cGVHD) is a reaction which typically develops 3 to 6 months after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. (NCT00176865)
Timeframe: 6 months and 1 year

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor0
Arm 3 - Mismatched Double Cord Donors0

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Percentage of Donor Chimerism at 180 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 180

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor88.9
Arm 2 - Matched Unrelated Donor73.3
Arm 3 - Mismatched Double Cord Donors90.5

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Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD)

Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease. (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor1
Arm 2 - Matched Unrelated Donor1
Arm 3 - Mismatched Double Cord Donors0

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Percentage of Donor Chimerism at 100 Days

The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 100

Interventionpercentage of donor cells (Mean)
Arm 1 - Matched Sibling Donor96.5
Arm 2 - Matched Unrelated Donor75.5
Arm 3 - Mismatched Double Cord Donors100

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Number of Subjects With Mixed Chimerism

>10% Donor Cells at Day 100 (NCT00176865)
Timeframe: Day 100

Interventionparticipants (Number)
Arm 1 - Matched Sibling Donor3
Arm 2 - Matched Unrelated Donor8
Arm 3 - Mismatched Double Cord Donors4

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Number of Participants Experiencing Incidence of Relapse

The return of disease after its apparent recovery/cessation. (NCT00177047)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment69

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Time to Attainment of CR

"Mean (STD) among patients achieving complete remission (CR)~Myeloma Response Definitions - Using International Uniform Response Criteria:~Complete Response (CR):~Absence of the original monoclonal paraprotein~<5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy~No increase in size or number of lytic bone lesions~Disappearance of soft tissue plasmacytomas" (NCT00177047)
Timeframe: 12 months post transplant

Interventionmonths (Mean)
Chemotherapy and Transplant Treatment4.6

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Time to Relapse

Mean number of days among patients relapsing (NCT00177047)
Timeframe: 1 year

InterventionDays (Mean)
Chemotherapy and Transplant Treatment182.9

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Time to Progression

Mean number of days among patients progressing (NCT00177047)
Timeframe: 1 year

InterventionDays (Mean)
Chemotherapy and Transplant Treatment159.4

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Number of Participants With Overall Survival

The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate. (NCT00177047)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment328

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Number of Participants Achieving a Complete Response

"Myeloma Response Definitions - Using International Uniform Response Criteria:~Stringent Complete Response (sCR)requires, plus CR:~Normal free light chain ratio~Absence of clonal cells in bone marrow~Complete Response (CR):~Absence of the original monoclonal paraprotein~<5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy~No increase in size or number of lytic bone lesions~Disappearance of soft tissue plasmacytomas." (NCT00177047)
Timeframe: 100 Days post transplant

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment51

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Number of Participants Achieving a Complete Response

"Myeloma Response Definitions - Using International Uniform Response Criteria:~Stringent Complete Response (sCR)requires, plus CR:~Normal free light chain ratio~Absence of clonal cells in bone marrow~Complete Response (CR):~Absence of the original monoclonal paraprotein~<5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy~No increase in size or number of lytic bone lesions~Disappearance of soft tissue plasmacytomas." (NCT00177047)
Timeframe: 12 months post transplant

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment123

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Number of Participants Achieving a Complete Response

"Myeloma Response Definitions - Using International Uniform Response Criteria:~Stringent Complete Response (sCR)requires, plus CR:~Normal free light chain ratio~Absence of clonal cells in bone marrow~Complete Response (CR):~Absence of the original monoclonal paraprotein~<5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy~No increase in size or number of lytic bone lesions~Disappearance of soft tissue plasmacytomas." (NCT00177047)
Timeframe: 6 months post transplant

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment99

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Number of Participants With Absolute Neutrophil Recovery

Hematologic recovery is defined by absolute neutrophil count (ANC) >2500/μl and platelets > 100,000/μl (NCT00177047)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment363

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Number of Participants With Disease Progression

"Myeloma Response Definitions - Using International Uniform Response Criteria:~Progressive Disease (PD)~For patients not in CR or sCR, progressive disease requires one or more of the following:~>25% increase in the level of the serum monoclonal paraprotein, which must also be an absolute increase of at least 0.5 g/dL.~>25% increase in 24-hour urine protein electrophoresis, which must also be an absolute increase of at least 200 mg/24 hours.~Absolute increase in the difference between involved and uninvolved FLC levels (absolute increase must be >10 mg/dl), only in patients without measurable paraprotein in the serum and urine.~>25% increase in plasma cells in a bone marrow aspirate or on trephine biopsy, which must also be an absolute increase of at least 10%.~Definite increase in the size of existing bone lesions or soft tissue plasmacytomas." (NCT00177047)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment34

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Number of Participants With Overall Survival

The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate. (NCT00177047)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment349

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Number of Participants With Infections

Occurrence of infections in the patients by the first 100 days of transplant (NCT00177047)
Timeframe: By first 100 days

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment68

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Time to Attainment of CR+PR

"Mean (STD) among patients achieving complete remission (CR) and partial remission (PR)~Myeloma Response Definitions - Using International Uniform Response Criteria:~Complete Response (CR):~Absence of the original monoclonal paraprotein~<5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy~No increase in size or number of lytic bone lesions~Disappearance of soft tissue plasmacytomas.~Partial Response (PR):~Greater than or equal to 50% reduction in the level of the serum monoclonal paraprotein and/or reduction in 24 hour urinary monoclonal paraprotein either by greater than or equal to 90% or to <200 mg/24 hours in light chain disease.~If the only measurable non-bone marrow parameter is FLC, greater than or equal to 50% reduction in the difference between involved and uninvolved FLC levels or a 50% decrease in level" (NCT00177047)
Timeframe: 12 months post transplant

Interventionmonths (Mean)
Chemotherapy and Transplant Treatment4.3

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Number of Participants With Overall Survival

The percentage of people in a study or treatment group who are alive for a certain period of time after they were diagnosed with or treated for a disease, such as cancer. Also called survival rate. (NCT00177047)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment301

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Number of Patients With Extended Disease-free Survival

Extended disease free survival will be defined as percentage of patients surviving more than 36 months without relapse or disease progression. (NCT00177047)
Timeframe: 36 Months

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment164

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Number of Participants With Toxicities

Occurrence of toxicities by first 100 days of transplant (NCT00177047)
Timeframe: By first 100 days

InterventionParticipants (Count of Participants)
Chemotherapy and Transplant Treatment68

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5-Year Overall Survival Rate

Estimated using the product-limit method of Kaplan and Meier. Patients who were still alive were censored at the date of last follow-up (NCT00182793)
Timeframe: From time of initial PBPC rescue until the date of death from any cause, assessed up to 5 years post treatment.

Interventionpercentage of participants (Median)
All Patients75

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5-Year Relapse-free Survival Rate

Estimated using the product-limit method of Kaplan and Meier. Relapse defined as appearance of any new lesions during or after protocol treatment. Whenever possible, relapses should be documented histologically. (NCT00182793)
Timeframe: From time of initial PBPC rescue until death or disease recurrence (disease progression for patients with stage IV disease), whichever came first, up to 5 years post treatment

Interventionpercentage of participants (Median)
All Patients53

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Acute Graft-vs-Host-Disease (aGvHD)

Development of acute graft-vs-host-disease (aGvHD) within 6 months, for participants receiving Allo-HCT. (NCT00185614)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Auto- Then Allo-HCT7

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Event-free Survival (EFS)

"Event-free survival (EFS) as determined for all participants who received the initial Auto-HCT treatment. Event was defined as any of the following within 3 years of the participant's last infusion of Auto-HCT or Allo-HCT: relapse; death; or last follow-up if there is no data to document the participant remained alive at 3 years." (NCT00185614)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT02424

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Relapse Rate

Relapse rate as determined for all participants who received the initial Auto-HCT treatment. Relapse was protocol-specified as progressive disease, indicated by an increase as compared to pre-Auto-HCT baseline, of serum or urine monoclonal protein >25%; bone marrow plasmacytosis >25%; or bone lesions on skeletal survey (any increase). (NCT00185614)
Timeframe: 3 years

Interventionparticipants (Number)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT22931

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Overall Survival (OS)

Overall Survival (OS) as determined for all participants who received the initial Auto-HCT treatment, as assessed from the date of the last transplant. (NCT00185614)
Timeframe: 3 years

Interventionparticipants (Number)
Auto-HCT onlyAuto-HCT then Allo-HCTAll Participants
Auto- Then Allo-HCT14142

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CR and Near CR Rates

"Per modified European Group for Blood and Marrow Transplant (EBMT) response criteria for definition of response in patients with multiple myeloma treated by high-dose therapy and stem cell transplantation: Complete Response (CR): Complete disappearance of all clinically measurable disease, complete response requires negative tests for monoclonal proteins in serum and urine by immunofixation, no monoclonal plasma cells in marrow specimen by flow cytometry and no evidence of progressive bone disease by skeletal survey. Near Complete Response (nCR): Less than 0.1 gram/dL monoclonal protein detectable in serum by standard protein electrophoresis and less than 50 mg monoclonal protein detectable in urine on 24 hour collection. Less than 5% monoclonal plasma cells detectable in bone marrow by immunohistochemistry. No evidence of progressive bone disease by skeletal survey." (NCT00217438)
Timeframe: Up to 120 days after transplant

Interventionpercentage of participants (Number)
Arm I (High Dose Melphalan, Amifostine Trihydrate, Transplant)39
Arm II (Low Dose Melphalan, Amifostine Trihydrate, Transplant)22

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Relative Toxicities Between Melphalan 280 mg/m^2 or Melphalan 200 mg/m^2

Number of Grade 3-4 adverse events observed in each group from enrollment through the Day 80-120 evaluation. Grade 3-4 events are defined by the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0. (NCT00217438)
Timeframe: Up to day 56 after transplant

InterventionGrade 3-4 adverse events (Number)
Arm I (High Dose Melphalan, Amifostine Trihydrate, Transplant)20
Arm II (Low Dose Melphalan, Amifostine Trihydrate, Transplant)10

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2-year Progression-free Survival

Measured from date of randomization to date of first observation of progressive disease, or death due to any cause (NCT00233987)
Timeframe: At day 60, then every 6 months for 2 years

Interventionpercentage of participants (Number)
High-dose Therapy Plus Tandem Transplant59

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Overall Survival

Measured from date of registration to date of death due to any cause or last contact (NCT00233987)
Timeframe: At day 60, then every 6 months for 2 years, then annually for a total of 7 years

Interventionpercentage of participants (Number)
High-dose Therapy Plus Tandem Transplant91

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Response Rate

Complete Response(CR) is a complete disappearance of all disease with the exception of nodes. No new lesions. previously enlarged organs must have regressed and not be palpable. Bone marrow(BM) must be negative if positive at baseline. Normalization of markers. CR Unconfirmed (CRU) does not qualify for CR above, due to a residual nodal mass or an indeterminate BM. Partial Response(PR) is a 50% decrease in the sum of products of greatest diameters (SPD) for up to 6 identified dominant lesions, including spleenic and hepatic nodules from baseline. No new lesions and no increase in the size of liver, spleen or other nodes. (NCT00233987)
Timeframe: At day 60, then every 6 months for 2 years

Interventionparticipants (Number)
Complete ResponsePartial ResponseUnconfirmed Complete ResponseUnconfirmed Partial ResponseNo ResponseAssessment Inadequate
High-dose Therapy Plus Tandem Transplant158323326

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Disease-Free Survival

The data presented below represents the total number of subjects (out of 36) that reached disease-free survival status during Months 3, 6, 9, 12, 18, and 24 time points. (NCT00238433)
Timeframe: 3, 6, 9, 12, 18, and 24 months post transplantation

InterventionParticipants (Count of Participants)
3 months6 months9 months12 months18 months24 months
Busulfan/Melphalan/Thiotepa343028252422

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Dose Limiting Veno-occlusive Disease (VOD) / Sinusoidal Obstruction Syndrome SOS

"Dose limiting veno-occlusive disease (VOD) defined as:~the presence of hepatomegaly with right upper quadrant tenderness and an elevation of total bilirubin > grade 1, PLUS~the presence of grade 3 abnormalities of any ONE of the following: total bilirubin, hypoalbuminemia, weight gain, or hypoxia without other attribution" (NCT00253435)
Timeframe: Between start of MIBG treatment and 60 days post stem cell infusion

Interventionparticipants (Number)
Poor Risk Patients6
Good Risk Patients0

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Engraftment DLT

"• Engraftment toxicity: delayed engraftment and/or failure to engraft defined as:~neutrophils (ANC) < 500/μL by day 28 post transplant, or~platelets < 20,000 /μL by day 56 post transplant, or~if additional stem cells are required to be infused for any medical reason prior to initial engraftment of neutrophils or platelets." (NCT00253435)
Timeframe: From treatment start until 60 days post stem cell infusion

Interventionparticipants (Number)
Poor Risk Patients2
Good Risk Patients1

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Event-free Survival (EFS) at 3 Years

EFS will be measured from start of treatment until progression, death or start of another treatment - whichever comes first. We report the estimated probability of EFS at 3 years. (NCT00253435)
Timeframe: 3 years since start of treatment

InterventionEstimated probability (Number)
Poor Risk Patients0.20
Good Risk Patients0.38

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Response (Complete Response, Very Good Partial Response, and Partial Response) at 60-days Post Stem Cell Infusion

Tumor response based on evaluation performed on day 60 or at the time of disease progression/recurrence or start of another treatment - whichever comes first. Such evaluations will include 123I-MIBG scan, CT/MRI, urine catecholamine measurement, and bone marrow analysis (for those with marrow disease at study entry). (NCT00253435)
Timeframe: Response assessed 60 days post stem cell infusion

Interventionparticipants (Number)
Poor Risk Patients4
Good Risk Patients3

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Progression-free Survival

Outcome is based on the number of patients who were alive without progression or relapse within 1 year. Progression is defined as a 50% increase in the sum of products of all measurable lesions. (NCT00265889)
Timeframe: one year after second transplant

Interventionparticipants (Number)
Poor Risk Patients18

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Response Rate

Number of patients that receive a Complete Response (CR), Partial Response (PR)or Progression. CR defined as complete disappearance of all measurable and evaluable disease and no new lesions. PR is defined as >/= 50% decrease in the sum of products of all measurable lesions. Progression is defined as a 50% increase in the sum of products of all measurable lesions. (NCT00265889)
Timeframe: One year after second transplant

Interventionparticipants (Number)
Complete ResponseProgressionUnknownExpiredPartial Response
Poor Risk Patients1814230

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Number of Patients That Experience Pulmonary Toxicity

Pulmonary toxicity are due to side effects that medicinal drugs cause to the lungs. (NCT00265889)
Timeframe: One year after second transplant

Interventionparticipants (Number)
Poor Risk Patients9

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Overall Survival

The probability that a participant did not experienced a death estimated at 1, 2, 3, 4, and 5 years following transplant via the Kaplan-Meier Method. Greenwood's formula for standard error was used to calculate 90% confidence intervals. Participants that did not die were censored at the time of last follow-up. (NCT00288626)
Timeframe: From study entry to death, loss to follow-up, or the end of the study, whichever came first, up to 6 years

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT1.01.00.9570.9110.863

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Percent Change From Screening in Brain Volume

Magnetic resonance imaging (MRI) scan techniques measured ventricular volumes and grey and white matter brain volumes. Change from screening was computed as the value at the time point minus the screening value. (NCT00288626)
Timeframe: 8 weeks to 5 years after HCT

InterventionPercent Change (Mean)
8 Weeks Post Transplant6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT-0.8-1.1-1.2-1.6-2.2-2.0-2.3

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MS Relapse-Free Survival Probability After Transplant

"MS clinical relapse is defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit or disability, and lasting over 48 hours. Clinical relapse was determined by the participant's neurologist and was measured as days from transplant to new or worsening neurological symptom relative to baseline.~Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error." (NCT00288626)
Timeframe: 1 to 5 years after HCT

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT0.9580.9150.8690.8690.869

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Number of New T2-Weighted Lesions From Baseline

A T2-weighted magnetic resonance imaging (MRI) scan was used to determine the number of new T2 lesions in the brain relative to Baseline. A value of 0 means that the participant didn't worsen. Values greater than 0 indicate an increase in disease activity from baseline. (NCT00288626)
Timeframe: 6 Months to 5 years after HCT

InterventionLesions per scan (Mean)
6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT0.20.20.20.10.40.1

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MS Progression-Free Survival Probability After Transplant

"MS progression is measured as number of days from transplant to first Kurtzke's Expanded Disability Status Scale (EDSS) increase of more than 0.5 relative to the baseline measurement. EDSS assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS).~Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error." (NCT00288626)
Timeframe: 1 to 5 years after HCT

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT1.00.9130.9130.9130.913

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MRI Activity-Free Survival Probability After Transplant

MS disease activity is measured as days from transplant to first occurrence of >= 2 new MS lesions on Magnetic resonance imaging (MRI) relative to baseline. Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error. (NCT00288626)
Timeframe: 1 to 5 years after HCT

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT0.9580.9580.9580.9100.863

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Event-Free Survival Probability After Transplant

Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence intervals based on Greenwood's formula for standard error. (NCT00288626)
Timeframe: 1, 2, and 4 years after HCT

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant4 Years Post Transplant
HDIT and HCT0.9580.8280.738

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Disease-Modifying Therapy Survival Probability After Transplant

Treatment with disease-modifying therapy was measured by the number of days from transplant to the first treatment with an additional disease-modifying therapy. Examples of therapy include interferon beta-1a, glatiramer acetate, natalizumab, alemtuzumab, other immunosuppressive medications, or experimental therapies directed against MS activity. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. (NCT00288626)
Timeframe: 1 to 5 years after HCT

InterventionProbability (Number)
1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT1.01.01.01.00.950

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Change From Baseline in T2-Weighted Lesion Volume

A T2-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T2 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value. (NCT00288626)
Timeframe: 8 weeks to 5 years after HCT

Interventionmilliliters (Mean)
8 Weeks Post Transplant6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT-0.8-0.7-1.0-1.6-1.9-1.9-2.3

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Change From Baseline in T1-Weighted Lesion Volume

A T1-weighted magnetic resonance imaging (MRI) scan was used to assess the volume of T1 lesions in the brain. Change from baseline was computed as the value at the time point minus the baseline value. (NCT00288626)
Timeframe: 8 weeks to 5 years after HCT

Interventionmilliliter (Mean)
8 Weeks Post Transplant6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT-0.10.00.20.30.40.40.3

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Change From Baseline in Number of Gadolinium-Enhanced Lesions

Multiple sclerosis disease-related lesions were assessed by gadolinium-enhanced magnetic resonance imaging (MRI). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening. (NCT00288626)
Timeframe: 8 weeks to 5 years after HCT

InterventionLesions per scan (Mean)
8 Weeks Post Transplant6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT-2.1-2.3-2.5-2.5-1.3-2.2-2.7

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Change From Baseline in Extended Disability Status Scale (EDSS)

Kurtzke's Expanded Disability Status Scale (EDSS) assesses disability in Multiple Sclerosis patients. Eight functional systems are evaluated: visual, brain stem, pyramidal, cerebellar, sensory, bowel and bladder, cerebral, and ambulation. The overall score ranges from 0 (normal neurological exam) to 10 (death due to MS). Change from baseline was computed as the value at the time point minus the baseline value. A negative value in change from baseline indicates an improvement and a positive value indicates worsening. A change of > 0.5 in EDSS was a treatment-failure criterion. (NCT00288626)
Timeframe: 6 months to 5 years after HCT

Interventionunits on a scale (Mean)
6 Months Post Transplant1 Year Post Transplant2 Years Post Transplant3 Years Post Transplant4 Years Post Transplant5 Years Post Transplant
HDIT and HCT-0.3-0.7-0.8-0.8-0.6-0.9

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Time to Platelet Engraftment

Platelet engraftment, or platelet count recovery, is defined as Platelets > 20,000/μL for two consecutive measurements on different days with no platelet transfusions in the preceding 7 days. Normal range is 150,000-450,000/μL. Reference: http://www.hopkinsmedicine.org/heart_vascular_institute/clinical_services/centers_excellence/womens_cardiovascular_health_center/patient_information/health_topics/platelets.html. (NCT00288626)
Timeframe: From time of graft infusion to time of engraftment, up to 6 years

InterventionDays (Mean)
HDIT and HCT18.5

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Time to Neutrophil Engraftment

Neutrophil engraftment, or neutrophil count recovery, is defined as an Absolute Neutrophil Count (ANC) > 500/ μL for 2 consecutive measurements on different days. Normal range is 1500 to 8000/μL. Reference: http://www.medicinenet.com (NCT00288626)
Timeframe: From time of graft infusion to time of engraftment, up to 6 years

InterventionDays (Mean)
HDIT and HCT10.8

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Percent of Participants Who Experienced All-Cause Morbidity Within 12 Months of Post-HCT

Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher. (NCT00288626)
Timeframe: From the time of Autologous CD34+ Hematopoietic Stem Cell Transplant (HCT) to 1 year after HCT.

InterventionPercentage of Participants (Number)
HDIT and HCT95.8

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Percent of Participants Who Experienced All-Cause Morbidity

Morbidity is the occurrence of NCI Common Terminology Criteria for Adverse Events (CTCAE) v3.0 adverse event grade 3 or higher. (NCT00288626)
Timeframe: From the time of enrollment until completion of the 5-year follow-up, an average of 6 years.

Interventionpercentage of participants (Number)
HDIT and HCT100

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Event-Free Survival Probability During the 3 Years After Transplant

Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. (NCT00288626)
Timeframe: 3 years

InterventionProbability (Number)
HDIT and HCT0.784

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Event-Free Survival Probability During the 5 Years After Transplant

Event-free survival (EFS) is survival without death or disease activity from any one of the following criteria: 1) loss of neurological function, defined as a change in pretransplant Extended Disability Status Scale (EDSS) of > 0.5. 2) Relapse, defined as the development of a new neurological sign and corresponding symptom, or worsening of an existing neurological sign and symptom, localized to central nervous system white matter, resulting in neurological deficit/disability, and lasting over 48 hours. 3) New lesions on magnetic resonance imaging (MRI), defined as presence of 2 or more independent multiple sclerosis brain lesions detected on MRI 1 year or more after stem cell transplant. Kaplan-Meier estimates of survival probability, with 90% confidence interval based on Greenwood's formula for standard error. (NCT00288626)
Timeframe: 5 years

InterventionProbability (Number)
HDIT and HCT0.692

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Overall Survival (OS)

Median overall survival after first peripheral blood stem cell transplant (PBSCT). (NCT00307086)
Timeframe: 40 months post transplant

Interventionmonths (Median)
Autologous PBSCT40

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Phase II Overall Survival (OS)

Time from start of treatment until death from any cause. (NCT00325416)
Timeframe: Phase II - Phase start at 62 months up to 120 months

Interventionmonths (Median)
Age Group A - Melphalan and Topotecan Plus Stem Cell Rescue63
Age Group B - Melphalan and Topotecan Plus Stem Cell Rescue62

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Phase II Event Free Survival (EFS)

Time to treatment failure, which is defined as the time from day 0 to the time of progressive disease. Progressive disease is defined by unequivocal objective evidence and constitutes any of the following: 1). an increase in the total amount of monoclonal protein (M-component from Serum Protein Electrophoresis (SPEP) and/or Urine Protein Electrophoresis (UPEP) with immunofixation) by more than 100% from the lowest level of serum myeloma protein seen after high-dose chemotherapy by serum protein electrophoresis; 2). an increase in the total amount of monoclonal protein above the remission level of the myeloma peak (i.e., an increase of >25% above the lowest level in a 24 hour urine or serum protein; 3). the reappearance of the M-protein if the patient had entered a CR: 4). definite increase in the size (> 1 cm) or number of lytic bone lesions. Compression fractures do not constitute a relapse. (NCT00325416)
Timeframe: Phase II - Phase start at 62 months up to 120 months

Interventionmonths (Median)
Age Group A - Melphalan and Topotecan Plus Stem Cell Rescue13.3
Age Group B - Melphalan and Topotecan Plus Stem Cell Rescue21.3

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Phase I - Maximum Tolerated Dose (MTD) Level

"MTD of topotecan in multiple myeloma patients receiving autologous transplant when give with melphalan 150 mg/m^2 for three days. Two parallel dose escalations were used, one each for young (18-60 years of age) and elderly patients (> 61 years of age). Elderly patients began a dose level once it had been found to be safe for the young cohort. The purpose of this approach was to expand the access of this trial to elderly patients while ensuring safety.~Phase I Dose Escalation: Level 1 - 20 mg/m^2; Level 2 - 30 mg/m^2; Level 4 - 54 mg/m^2; Level 5 - 72 mg/m^2; Level 6 - 96 mg/m^2; Level 7 - 127.8 mg/m^2; Level 8 - 170.1 mg/m^2" (NCT00325416)
Timeframe: Phase I - 5 years, 2 months

Interventionmg/m^2 (Number)
Age Group A - Melphalan and Topotecan Plus Stem Cell Rescue127.8
Age Group B - Melphalan and Topotecan Plus Stem Cell Rescue30

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Phase II Participants - Overall Response Rate

Re-evaluation of participants who had responsive disease prior to transplant. All changes in monoclonal protein and immunoglobulins will be referenced to those levels obtained immediately prior to cyclophosphamide priming chemotherapy. Complete Response (CR): A CR will be defined as the disappearance of the monoclonal protein by immunofixation studies of serum and urine (100x concentrate) and less than or equal to 5% plasma cells in a bone marrow aspirate. Partial Response (PR): 50% - 74% decrease in the measurable monoclonal protein (M-component from an SPEP and/or UPEP with immunofixation). (NCT00325416)
Timeframe: Phase II - Phase start at 62 months up to 120 months

,
Interventionpercentage of participants (Number)
Overall Response RateComplete Response RatePartial Response Rate
Age Group A - Melphalan and Topotecan Plus Stem Cell Rescue652639
Age Group B - Melphalan and Topotecan Plus Stem Cell Rescue571839

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Number of Participants With Pulmonary Toxicity

Pulmonary toxicity was assessed as the incidence of interstitial pneumonitis. (NCT00349778)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
High-Dose Sequential Therapy32

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Number of Participants That Relapse After Autologous Transplantation

Relapse was measured as the number of patients who relapse after high-dose sequential therapy then autologous transplantation (NCT00349778)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
High-Dose Sequential Therapy66

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Overall Participant Survival (OS)

Survival status was assessed 5 years after transplant. (NCT00349778)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
High-Dose Sequential Therapy52

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Overall Objective Response

(NCT00357396)
Timeframe: 2 years

Interventionparticipants (Number)
Very Good Partial Response (VGPR)Complete Response (CR)
Patients With HIGH RISK EWING'S SARCOMA FAMILY TUMOR41

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Number of Patients With Chronic Graft Versus Host Disease (GVHD)

Graft-Versus-Host Disease is a severe complication created by infusion of donor cells into a foreign host. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. (NCT00383448)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treated Patients2

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Number of Patients With Donor Cell Engraftment

Donor Cell Engraftment is defined as the process of transplanted stem cells reproducing new cells. (NCT00383448)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treated Patients26

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Number of Patients With Acute Graft Versus Host Disease (GVHD)

Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Acute GVHD can occur once the donor's cells have engrafted in the transplant recipient. The symptoms typically appear within weeks after transplant. (NCT00383448)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treated Patients3

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Kaplan Meier Estimates of Overall Survival (OS)

Data as of 11 May 2010 cutoff. Overall survival (OS) was defined as the time between randomization and death. Participants who died, regardless of the cause of death, were considered to have had an event. Participants who were lost to follow-up prior to the end of the trial, or who were withdrawn from the trial, were censored at the time of last contact. Participants who were still being treated were censored at the last available date available, or clinical cut-off date, if it was earlier. (NCT00405756)
Timeframe: up to 177 weeks

Interventionweeks (Median)
MPR+RNA
MPR+pNA
MPp+pNA

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Kaplan Meier Estimates for Duration of Response as Determined by the Central Adjudication Committee (CAC)

"Data as of 11 May 2010 cutoff. Duration of myeloma response was defined as the time from the initial response date to the earlier of progressive disease (PD) as determined by the CAC or death on study. PD was based on the European Group for Blood and Marrow Transplantation/International Bone Marrow Transplant Registry/Autologous Bone Marrow Transplant Registry [EBMT/IBMTR/ABMTR] criteria.~PD criteria includes increasing monoclonal paraprotein levels, bone marrow findings, worsening lytic bone disease, progressively enlarging extramedullary plasmacytomas, or hypercalcemia." (NCT00405756)
Timeframe: Up to 149 weeks

Interventionweeks (Median)
MPR+R121.6
MPR+p56.1
MPp+p55.4

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Financial Difficulties Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a problem scale like the financial problems scale = higher level of financial problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=111,123,125)Cycle 7 - approximately Month 7 (n=94,111,112)Cycle 10 - approximately Month 10 (n=84,92,97)Cycle 13 - approximately Month 13 (n=70,72,83)Cycle 16 - approximately Month 16 (n=61,52,63)
MPp+p-2.9-2.1-1.7-4.0-5.3
MPR+p-1.1-0.60.7-0.5-0.6
MPR+R2.42.16.04.81.6

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Insomnia Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the insomnia scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=118,124,128)Cycle 7 - approximately Month 7 (n=100,109,111)Cycle 10 - approximately Month 10 (n=87,94,96)Cycle 13 - approximately Month 13 (n=75,73,83)Cycle 16 - approximately Month 16 (n=64,53,63)
MPp+p-5.0-5.7-1.7-6.8-3.7
MPR+p-1.6-6.4-2.50.9-0.6
MPR+R2.0-1.0-5.0-4.9-4.7

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Nausea and Vomiting Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the nausea/vomiting scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=120,127,130)Cycle 7 - approximately Month 7 (n=99,112,112)Cycle 10 - approximately Month 10 (n=87,95,97)Cycle 13 - approximately Month 13 (n=75,72,83)Cycle 16 - approximately Month 16 (n=64,52,62)
MPp+p-0.00.70.3-0.4-1.3
MPR+p-1.3-0.7-1.4-3.0-4.2
MPR+R3.30.51.90.71.0

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Pain Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the pain scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=120,127,129)Cycle 7 - approximately Month 7 (n=100,112,113)Cycle 10 - approximately Month 10 (n=88,95,97)Cycle 13 - approximately Month 13 (n=74,74,83)Cycle 16 - approximately Month 16 (n=64,53,63)
MPp+p-13.4-11.5-9.8-12.1-12.2
MPR+p-13.8-16.5-15.6-14.9-11.0
MPR+R-14.4-17.8-17.2-13.7-20.3

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Physical Functioning Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score=better level of physical functioning. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=120,127,130)Cycle 7 - approximately Month 7 (n=100,112,112)Cycle 10 - approximately Month 10 (n=88,95,96)Cycle 13 - approximately Month 13 (n=75,74,83)Cycle 16 - approximately Month 16 (n=64,53,63)
MPp+p4.52.75.13.31.1
MPR+p3.38.18.59.77.6
MPR+R1.98.28.98.610.0

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Role Functioning Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score=better level of role functioning. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=119,127,130)Cycle 7 - approximately Month 7 (n=99,112,113)Cycle 10 - approximately Month 10 (n=86,95,95)Cycle 13 - approximately Month 13 (n=74,74,82)Cycle 16 - approximately Month 16 (n=64,53,63)
MPp+p7.46.95.65.77.1
MPR+p3.08.07.511.78.5
MPR+R1.85.79.39.712.2

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Social Functioning Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better level of social functioning. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=115,125,127)Cycle 7 - approximately Month 7 (n=98,111,112)Cycle 10 - approximately Month 10 (n=87,92,97)Cycle 13 - approximately Month 13 (n=72,73,83)Cycle 16 - approximately Month 16 (n=63,52,63)
MPp+p6.06.14.16.29.8
MPR+p0.34.44.57.56.1
MPR+R5.18.310.911.813.2

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Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. EORTC QLQ-MY20 includes four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale; higher score for the disease symptoms scale = higher level of symptomatology. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=113,121,127)Cycle 7 - approximately Month 7 (n=96,109,112)Cycle 10 - approximately Month 10 (n=85,91,95)Cycle 13 - approximately Month 13 (n=72,73,82)Cycle 16 - approximately Month 16 (n=62,51,62)
MPp+p-5.4-6.0-5.4-6.3-3.3
MPR+p-8.7-9.7-7.1-8.8-5.9
MPR+R-8.9-9.0-7.9-7.2-10.5

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Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Body Image Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale. For the body image scale, higher scores = better body image. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=110,117,119)Cycle 7 - approximately Month 7 (n=88,104,108)Cycle 10 - approximately Month 10 (n=79,83,94)Cycle 13 - approximately Month 13 (n=68,72,79)Cycle 16 - approximately Month 16 (n=59,52,61)
MPp+p4.55.23.95.12.7
MPR+p-0.32.6-4.0-0.56.4
MPR+R2.13.87.61.03.4

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Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Side Effects of Treatment Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale; higher score for the side effects scale = higher level of symptomatology. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=113,120,125)Cycle 7 - approximately Month 7 (n=95,108,111)Cycle 10 - approximately Month 10 (n=85,89,94)Cycle 13 - approximately Month 13 (n=72,72,81)Cycle 16 - approximately Month 16 (n=62,50,61)
MPp+p0.61.80.30.3-0.9
MPR+p0.1-1.70.0-1.0-2.9
MPR+R1.30.4-1.6-3.8-2.1

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Number of Participants in Disease Response Categories Representing Their Best Response During the Double-blind Treatment Period

Data as of 11 May 2010 cutoff. Best response was determined by the Central Assessment Committee (CAC) based on the European Group for Blood and Marrow Transplantation (EBMT) criteria: Complete Response (CR)-absence of serum and urine monoclonal paraprotein for 6 weeks, plus no increase in size or number of bone lesions, plus other factors); Partial Response (PR)-not all CR criteria, plus >=50% reduction in serum monoclonal paraprotein plus others; Stable Disease (SD)- not PR or PD; Progressive Disease (PD)- reappearance of monoclonal paraprotein, bone lesions, other; Not Evaluable (NE). (NCT00405756)
Timeframe: Up to 165 weeks

,,
Interventionparticipants (Number)
Complete response (CR)Partial response (PR)Stable disease (SD)Progressive disease (PD)Response not evaluable (NE)
MPp+p5727007
MPR+p5994027
MPR+R151022807

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Summary of Participants With Treatment-Emergent Adverse Events (TEAE) During the Double-Blind Treatment Period

Data as of 11 May 2010 cutoff. Participant counts in different categories of TEAEs during the double-blind treatment period. A TEAE is as any AE occurring or worsening on or after the first treatment of any study drug, and within 30 days after the last dose of the last study drug. Severity grades according to Common Terminology Criteria for Adverse Events v3.0 (CTCAE) on a 1-5 scale: Grade 1= Mild AE, Grade 2= Moderate AE, Grade 3= Severe AE, Grade 4= Life-threatening or disabling AE, Grade 5=Death related to AE. Dose reduction includes reduction with or without interruption. (NCT00405756)
Timeframe: Up to 169 weeks (Double-blind therapy period plus 4 weeks)

,,
Interventionparticipants (Number)
>=1 adverse event (AE)>=1 CTCAE grade 3-4 AE>=1 CTCAE grade 5 AE>=1 serious AE (SAE)>=1 AE related to Lenaldomide/Placebo>=1 AE related to Melphalan>=1AE related to Prednisone>=1 Grade 3-4 AE related to Lenaldomide/Placebo>=1 Grade 3-4 AE related to Melphalan>=1 Grade 3-4 AE related to Prednisone>=1 Grade 5 AE related to Lenalidomide/Placebo>=1 Grade 5 AE related to Melphalan>=1 Grade 5 AE related to Prednisone>=1 SAE related to Lenalidomide/Placebo>=1 SAE related to Melphalan>=1 SAE related to Prednisone>=1 AE leading to Lenalidomide/Placebo withdrawal>=1 AE leading to Melphalan withdrawal>=1 AE leading to Prednisone withdrawal>=1 AE leading to Lenalidomide/Plac dose reduction>=1 AE leading to Melphalan dose reduction>=1 AE leading to Prednisone dose reduction>=1 AE leading to Lenalidomide/Plac dose interrupt>=1 AE leading to Melphalan dose interruption>=1 AE leading to Prednisone dose interruption
MPp+p15310775613112693686222231111151410102621551015
MPR+p15112966214513494117110292113224162419197058782139
MPR+R150137766148140871281183233138271926202071471592528

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Change From Baseline to Cycles 4, 7, 10, 13, 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) In Future Perspective Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. Questions used 4-point scale (1 'Not at All' to 4 'Very Much'). Scores are averaged, and transformed to 0-100 scale. For the future perspective scale, higher score = better perspective of the future. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=112,121,124)Cycle 7 - approximately Month 7 (n=93,108,112)Cycle 10 - approximately Month 10 (n=83,88,97)Cycle 13 - approximately Month 13 (n=71,73,81)Cycle 16 - approximately Month 16 (n=62,52,62)
MPp+p7.69.814.511.914.4
MPR+p4.37.76.66.37.7
MPR+R4.714.617.317.318.5

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Kaplan Meier Estimates of Progression-free Survival (PFS) Based on the Response Assessment by the Central Adjudication Committee (CAC)

"Data as of 11 May 2010 cutoff. PFS was calculated as the time from randomization to the earlier of the first documentation of progressive disease (PD) as determined by the CAC, or death on study due to any cause. PD was based on the European Group for Blood and Marrow Transplantation/International Bone Marrow Transplant Registry/Autologous Bone Marrow Transplant Registry [EBMT/IBMTR/ABMTR] criteria.~PD criteria includes increasing monoclonal paraprotein levels, bone marrow findings, worsening lytic bone disease, progressively enlarging extramedullary plasmacytomas, or hypercalcemia." (NCT00405756)
Timeframe: up to 165 weeks

Interventionweeks (Median)
MPR+R136.1
MPR+p62.1
MPp+p56.1

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Kaplan Meier Estimates of Progression-free Survival (PFS) From Start of Maintenance Therapy Period Based on the Response Assessment by the Central Adjudication Committee (CAC)

"Data as of 11 May 2010 cutoff. PFS calculated from the start of the Maintenance period to the earlier of the first documentation of progressive disease (PD) as determined by the CAC, or death on study due to any cause.~PD was based on the European Group for Blood and Marrow Transplantation/International Bone Marrow Transplant Registry/Autologous Bone Marrow Transplant Registry [EBMT/IBMTR/ABMTR] criteria.~PD criteria includes increasing monoclonal paraprotein levels, bone marrow findings, worsening lytic bone disease, progressively enlarging extramedullary plasmacytomas, or hypercalcemia." (NCT00405756)
Timeframe: Approximately week 37 (start of cycle 10) to week 165

Interventionweeks (Median)
MPR+R112.0
MPR+p32.3

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Kaplan Meier Estimates of Time to Progression (TTP) Based on the Response Assessment by the Central Adjudication Committee (CAC)

"Data as of 11 May 2010 cutoff. TTP was the time between randomization and disease progression as determined by the CAC. PD was based on the European Group for Blood and Marrow Transplantation/International Bone Marrow Transplant Registry/Autologous Bone Marrow Transplant Registry [EBMT/IBMTR/ABMTR] criteria.~PD criteria includes increasing monoclonal paraprotein levels, bone marrow findings, worsening lytic bone disease, progressively enlarging extramedullary plasmacytomas, or hypercalcemia." (NCT00405756)
Timeframe: up to 165 weeks

Interventionweeks (Median)
MPR+R148.1
MPR+p62.7
MPp+p61.3

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Time to First Response

Data as of 11 May 2010 cutoff. Time to first response was defined as the time from start of treatment until first response as assessed by the Central Assessment Committee (CMC) based on European Group for Blood and Marrow Transplantation (EBMT) criteria. (NCT00405756)
Timeframe: Up to 66 weeks

Interventionweeks (Mean)
MPR+R10.0
MPR+p9.3
MPp+p16.2

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Quality of Life Scale

Data as of 11 May 2010 cutoff. EORTC QLC-C30 is a 30-item questionnaire to assess the quality of life in cancer patients. EORTC QLQ-C30 includes functional scales (physical, role, cognitive, emotional, social), global health status, symptom scales (fatigue, pain, nausea/vomiting), and other (dyspnoea, appetite loss, insomnia, constipation/diarrhea, financial difficulties). Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); two used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better quality of life. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=114,121,125)Cycle 7 - approximately Month 7 (n=96,108,110)Cycle 10 - approximately Month 10 (n=84,86,96)Cycle 13 - approximately Month 13 (n=70,70,82)Cycle 16 - approximately Month 16 (n=61,50,62)
MPp+p6.14.26.25.48.1
MPR+p5.68.18.88.87.2
MPR+R2.38.012.47.610.7

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Appetite Loss Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the appetite loss scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=119,125,130)Cycle 7 - approximately Month 7 (n=99,111,111)Cycle 10 - approximately Month 10 (n=87,93,96)Cycle 13 - approximately Month 13 (n=75,72,83)Cycle 16 - approximately Month 16 (n=64,52,63)
MPp+p-5.6-5.7-8.0-4.8-6.4
MPR+p1.9-5.7-5.4-8.8-16.0
MPR+R1.7-3.7-5.0-6.2-7.8

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Congitive Functioning Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better level of cognitive functioning. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=115,125,128)Cycle 7 - approximately Month 7 (n=98,111,113)Cycle 10 - approximately Month 10 (n=87,92,97)Cycle 13 - approximately Month 13 (n=73,73,83)Cycle 16 - approximately Month 16 (n=63,52,63)
MPp+p1.30.7-2.7-1.4-4.0
MPR+p-2.00.1-4.4-3.0-3.5
MPR+R0.32.91.0-0.00.3

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Constipation Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the constipation scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=114,124,128)Cycle 7 - approximately Month 7 (n=96,111,112)Cycle 10 - approximately Month 10 (n=86,93,97)Cycle 13 - approximately Month 13 (n=73,73,81)Cycle 16 - approximately Month 16 (n=63,51,62)
MPp+p-4.9-2.7-1.7-3.3-2.2
MPR+p4.80.6-1.1-2.7-5.2
MPR+R-1.8-3.5-5.0-5.0-1.6

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Diarrhoea Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the diarrhea scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=115,125,124)Cycle 7 - approximately Month 7 (n=98,109,112)Cycle 10 - approximately Month 10 (n=87,92,95)Cycle 13 - approximately Month 13 (n=73,73,80)Cycle 16 - approximately Month 16 (n=63,52,61)
MPp+p3.20.9-0.00.80.5
MPR+p1.9-1.21.4-1.41.3
MPR+R2.33.41.15.510.6

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Dyspnoea Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the dyspnoea scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=117,126,126)Cycle 7 - approximately Month 7 (n=100,110,110)Cycle 10 - approximately Month 10 (n=86,93,96)Cycle 13 - approximately Month 13 (n=73,73,81)Cycle 16 - approximately Month 16 (n=62,53,62)
MPp+p-0.02.13.8-0.01.6
MPR+p-6.4-8.5-4.3-2.3-6.3
MPR+R-2.6-1.7-4.3-5.0-3.2

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Emotional Functioning Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score = better level of emotional functioning. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=115,125,128)Cycle 7 - approximately Month 7 (n=98,111,112)Cycle 10 - approximately Month 10 (n=86,92,97)Cycle 13 - approximately Month 13 (n=73,73,83)Cycle 16 - approximately Month 16 (n=63,52,63)
MPp+p6.85.04.76.66.9
MPR+p2.74.21.61.1-0.2
MPR+R4.88.89.08.29.9

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Change From Baseline to Cycles 4, 7, 10, 13 and 16 in European Organization for Research and Treatment of Cancer Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Scale

Data as of 11 May 2010 cutoff. EORTC QLQ-C30 is a 30-item questionnaire to assess the overall quality of life in cancer patients. Most questions used 4-point scale (1 'Not at All' to 4 'Very Much'); 2 questions used 7-point scale (1 'Very Poor' to 7 'Excellent'). Scores are averaged, and transformed to 0-100 scale; higher score for a symptom scale like the fatigue scale = higher level of symptomatology/problems. (NCT00405756)
Timeframe: Baseline (Day 0), Months 4, 7, 10, 13, 16

,,
Interventionunits on a scale (Mean)
Cycle 4 - approximately Month 4 (n=120,127,129)Cycle 7 - approximately Month 7 (n=100,112,110)Cycle 10 - approximately Month 10 (n=87,95,95)Cycle 13 - approximately Month 13 (n=74,74,82)Cycle 16 - approximately Month 16 (n=64,53,62)
MPp+p-5.1-5.7-6.9-7.5-4.1
MPR+p-5.5-9.5-7.5-10.7-9.7
MPR+R-3.0-7.6-7.5-7.1-10.0

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Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0

(NCT00423514)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL31
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL7

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Number of Participants With Engraftment

Engraftment defined as first of three (3) consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L; assessed from baseline to 100 days post-engraftment. (NCT00427557)
Timeframe: Baseline to 100 days post-engraftment

Interventionparticipants (Number)
Fludarabine + Melphalan + Umbilical Cord Blood Unit27

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Average Overall Survival Time

Average number of years for survival post transplant where overall survival time is measured from date of transplant to disease progression or death for any reason. (NCT00427765)
Timeframe: Baseline(transplantation) to disease progression or death for any reason, up to 6 years.

Interventionyears (Mean)
Busulfan + Melphalan3

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Time to Progressive Disease

Progression-free was measured, by days, at time from transplantation to development to disease or death from any cause, which ever occurred first. (NCT00429572)
Timeframe: Transplant to Progression.

InterventionDays (Median)
Allogeneic Transplantation202

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Grade II-IV Toxicity

Non-hematopoietic toxicity within the first year of transplantation, acute Graft versus Host Disease (GVHD) above National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade I and chronic above Grade I are reported by participant incidence. Broad classification of adverse events (AE) categories based on anatomy and/or pathophysiology; within each category, AEs are listed accompanied by their descriptions of severity (Grade, Grade 1 least severe). (NCT00429572)
Timeframe: Up to one year.

InterventionParticipants (Number)
CardiacPulmonaryGastrointestinalRenalNeurologicalFever/Flu like symptomsInfectionGenitourinarySkin
Allogeneic Transplantation138022322

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Overall Survival

Survival duration was calculated from time of transplantation by number of days. (NCT00429572)
Timeframe: Transplant until death.

InterventionDays (Median)
Allogeneic Transplantation643

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Number of Participants With Tumor Response

Best response recorded from start of treatment until disease progression/recurrence using World Health Organization (WHO) criteria of Complete Response: disappearance of all disease/symptoms > 4 weeks; Partial response, > 50% reduction in sum of products of diameters of each measurable lesion for more than 4 weeks; Stable Disease, no change in tumor size; and Progressive Disease, appearance of new lesions or > 25% increase in sum of products of diameters of any measurable lesions. (NCT00429572)
Timeframe: Baseline to measured progressive disease (post study follow-up period 24 months starting from the date of the last drug administration). Data collected every 4 months.

Interventionparticipants (Number)
Complete ResponseStable DiseasePartial ResponseProgressive Disease
Allogeneic Transplantation5913

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Number of Participants With Acute or Chronic GVHD And Response to Therapy

"Participants diagnosed with Graft versus Host Disease (GVHD) post transplant were divided into either acute (aGVHD), normally observed within the first 100 days post-transplant; and chronic GVHD (cGVHD) cases, normally occur after 100 days, then evaluated and scored according to standard criteria from Consensus conference on acute GVHD grading, Bone Marrow Transplant 1995; 15: 825-828, noted is type of case and whether responds to therapy." (NCT00429572)
Timeframe: Transplant to 1 year post transplant

Interventionparticipants (Number)
aGVHDaGVHD responded to therapycGVHDcGVHD responded to therapy
Allogeneic Transplantation971414

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Disease-free Survival

To determine DFS at 1 year post transplant. (NCT00439556)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Velcade Dose Level 116
Velcade Dose Level 20
Velcade Dose Level 30

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Number of Participants With Dose Limiting Toxicity (DLT)

To determine the maximum tolerated dose(MTD) of velcade and dose limiting toxicity(DLT). A dose limiting toxicity (DLT) was defined as a grade 3-4 neurological toxicity, graft failure, or death due to GvHD. The Commom Terminlogy Criteria for Adverse Events v3.0 was used. (NCT00439556)
Timeframe: From start of treatment to 90 days after the start of treatment

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Transplant, Filgrastim, Tacrolimus)0
Velcade Dose Level 20
Velcade Dose Level 30

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Number of Patients Reaching Complete Response (CR)

Number of participants with CR at Day 180 who had maintained CR for at minimum of 4 weeks, and who had: No monoclonal protein in urine/serum when analyzed by immunofixation electrophoresis; bone marrow normal by morphological examination with <5% plasma cells, <1% aneuploid light chain restricted population by flow cytometry for DNA/cIg; and, while healing of bony lesion is not required, no new lytic lesion should appear. Further compression fracture of spine not considered progressive disease. (NCT00469209)
Timeframe: Baseline through Day 180, with assessments at Day 90 and Day 180

Interventionparticipants (Number)
No Bortezomib4
Bortezomib 1.0 mg/m^21
Bortezomib 1.5 mg/m^24

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Disease-free Survival (DFS)

DFS defined as time from transplantation to disease relapse, disease progression, death during remission, or last follow-up. Evaluation at 3 months and 6 months after transplantation, then every 6 months for 3 years, and then once a year up to 5 years from the transplant date. (NCT00472056)
Timeframe: Up to 5 years from transplant date.

Interventionmonths (Mean)
Standard Dose Rituximab11.33
High Dose Rituximab9.635

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Percentage of Participants With Toxicity, Assessed Using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 (v3)

The overall toxicity rates (percentages) for grade 3 or higher adverse events considered at least possibly related to treatment are reported below. (NCT00477750)
Timeframe: Every cycle during treatment up to 3 years

Interventionpercentage of patients (Number)
Grade 3Grade 4Grade 5
Treatment (Lenalidomide, Melphalan, Prednisone)33670

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Progression-free Survival

"Progression free survival (PFS) is defined as the time from the date of randomization to the date of disease progression or death resulting from any cause, whichever comes first. Progression was defined as any one or more of the following:~An increase of 25% from lowest confirmed response in:~Serum M-component (absolute increase >= 0.5g/dl)~Urine M-component (absolute increase >= 200mg/24hour~Difference between involved and uninvolved Free Light Chain levels (absolute increase >= 10mg/dl~Bone marrow plasma cell percentage (absolute increase of >=10%)" (NCT00477750)
Timeframe: registration to progressive disease (up to 3 years)

Interventionmonths (Median)
Treatment (Lenalidomide, Melphalan, Prednisone)21.4

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Overall Survival (OS) at 3 Years

OS was defined as the time from registration to death due to any cause. Patients who were alive were censored at date of last follow-up. The overall survival at 3 years (a percentage) is reported below. (NCT00477750)
Timeframe: registration to death (up to 3 years)

Interventionpercentage of patients (Number)
Treatment (Lenalidomide, Melphalan, Prednisone)58

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Patients With Overall Confirmed Response

"Response that was confirmed on 2 consecutive evaluations.>~Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixations, normalization of Free Light Chain (FLC) ratio and <=5% plasma cells in bone marrow>~Very Good Partial Response (VGPR): >=90% reduction in serum M-spike, Urine M-spike <100mg per 24 hours>~Partial Response (PR): >=50% reduction in serum M-spike, Urine M-spike >=90% reduction or < 200mg per 24 hours, or >=50% decrease in difference between involved and uninvolved FLC levels or 50% decrease in bone marrow plasma cells" (NCT00477750)
Timeframe: Every cycle during treatment

Interventionparticipants (Number)
CRVGPRPR
Treatment (Lenalidomide, Melphalan, Prednisone)3510

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Duration of Response (DOR)

Duration of response was calculated from documentation of first response to date of progression in the subset of patients who responded. Patients without progression were censored at the date of last tumor evaluation. (NCT00477750)
Timeframe: from first response to progression or death (up to 3 years)

Interventionmonths (Median)
Treatment (Lenalidomide, Melphalan, Prednisone)16.3

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3-Year Progression Free Survival

"Percentage of patients who were progression free at 3 years. The 3-year progression free rate was estimated using the Kaplan Meier method.~Progression is assessed when one of the following occur:~reappearance of monoclonal protein by immunofixation,~Increase in serum monoclonal paraprotein to >25% above the lowest response level,~Increase in urine M-protein to > 25% above the lowest remission value for 24-hour excretion." (NCT00477971)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Low-Dose Melphalan29.1
High-Dose Melphalan + Autologous HSC51.7

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Organ Response to Treatment

"Organ response was evaluated on the basis of improvement of one or more affected organ; only one parameter was required to satisfy the criteria. Response needed to be maintained for a minimum of 3 months to be considered valid.~Renal response required a 50% reduction in 24-hour urine protein excretion (at least 0.5 g/d) with stable creatinine. Cardiac response required one of >= 2-mm reduction in the interventricular septal (IVS) thickness by echocardiogram, or improvement of ejection fraction by >= 20%, or improvement by 2 NYHA classes without an increase in diuretic use. Hepatic response required either >= 50% decrease in (or normalization of) an initially elevated alkaline phosphatase level or reduction in the size of the liver by at least 2 cm by radiographic determination. Gastrointestinal tract improvement was defined as normalization of a low serum carotene level, or reduction of diarrhea to < 50% of previous movements/day, or decrease in fecal fat excretion by 50%." (NCT00477971)
Timeframe: 10 years

Interventionpercentage of participants (Number)
Low-Dose Melphalan26.5
High-Dose Melphalan + Autologous HSC29.1

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Hematologic Response Rate

"Response that was confirmed on 2 consecutive evaluations during treatment. A hematologic response consisted of a Complete response, Very Good Partial Response or Partial Response.~Complete Response (CR): Complete disappearance of M-protein from serum and urine on immunofixation, normalization of Free Light Chain (FLC) ratio and <5% plasma cells in bone marrow.~Very Good Partial Response (VGPR): >=90% reduction in serum M-component; Urine M-Component <=100 mg per 24 hours.~Partial Response (PR): >=50% reduction in serum M-component and/or Urine M-Component >=90% reduction or <200 mg per 24 hours; or >=50% decrease in difference between involved and uninvolved FLC levels." (NCT00477971)
Timeframe: 10 years

Interventionpercentage of participants (Number)
Low-Dose Melphalan55.9
High-Dose Melphalan + Autologous HSC69.1

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3 Year Overall Survival

Percentage of patients who were alive at 3 years. The 3-year survival rate was estimated using the Kaplan Meier method. (NCT00477971)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Low-Dose Melphalan58.8
High-Dose Melphalan + Autologous HSC83.6

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Number of Participants With Successful Engraftment at Day 100

(NCT00505895)
Timeframe: Day 100

Interventionparticipants (Number)
Fludarabine + Melphalan + Stem Cell Infusion27
Fludarabine + Lower-Dose Melphalan + Stem Cell Infusion23

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Acute Grade II-IV Graft Versus Host Disease (GVHD)

Effects of Rituximab as measured by percentage of participants with Acute and Chronic Graft Versus Host Disease (GVHD) incidences after allogeneic transplantation. GVHD occurring anytime after day 90 post transplant was considered chronic GVHD; otherwise it was considered acute GVHD. Acute GVHD status defined as GVHD with maximum grade ≥2. Clinical grading of Acute GVHD (Thomas et al., New England Journal of Medicine (NEJM), 229:895, 1975): Grade 1 to 4. (NCT00505895)
Timeframe: GVHD grading weekly during first 100 days; Annual examinations for nine year study period

,
Interventionpercentage of participants (Number)
Acute GVHDChronic GVHD
Fludarabine + Lower-Dose Melphalan + Stem Cell Infusion2148
Fludarabine + Melphalan + Stem Cell Infusion2229

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Participant Progression Free Survival at 2 Years

Progression-free survival defined as the number of participants without evidence of progression or death after 2 years from stem cell transplant. (NCT00505921)
Timeframe: 2 years

Interventionparticipants (Number)
Campath-1H15

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Participant's Response According to Physician's Global Assessment of Clinical Condition (PGA)

Response defined by Physician's Global Assessment of Clinical Condition (PGA) where Complete Response (CR) is No evidence of disease; 100% improvement; Partial Response (PR), one of following: 1) Very significant clearance ( > 90% to < 100%); only traces of disease remains; 2) Significant improvement ( > 75% to < 90%); some evidence of disease remain; 3) Intermediate between slight and marked improvement; ( > 50% to < 75%); 4) Some improvement ( > 25% to < 50%); however, significant evidence of disease remains; Stable Disease (SD): Disease has not changed from baseline condition (+<25%); Progressive Disease (PD): Disease is worse than at baseline evaluation by > 25% or more. Response confirmed by a second assessment at least 4 weeks following it. Assessments at baseline, pre and post transplant (100 days) then till disease progression or year one. (NCT00506129)
Timeframe: Response assessed pre-transplant and 100 days post transplant with follow up at 1 year.

Interventionparticipants (Number)
Complete Response (CR) Converted Post TransplantCR Prior to TransplantPartial Response (PR)Stable Disease (SD)Progressive Disease (PD)
Fludarabine + Melphalan With PBPC1960016

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Average Overall Survival (OS) Length

OS was defined as the time from transplantation to the date of death from any cause or last follow up, measured in days. (NCT00506129)
Timeframe: Baseline to disease progression, followed up to 5 years post transplant

InterventionDays (Mean)
Fludarabine + Melphalan With PBPC1207

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Change From Baseline in EORTC QLQ-C30 - Global Health Status

"The European Organisation for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact).~The EORTC QLQ-C30 Global Health Status/QOL Scale is scored between 0 and 100, where higher scores indicate better Global Health Status/QOL. Negative changes from baseline indicate deterioration in QOL or functioning and positive changes indicate improvement." (NCT00507416)
Timeframe: Baseline and Day 1 of Cycles 3, 5, 7, 9, 11 and 13

,,
Interventionunits on a scale (Mean)
Cycle 3, Day 1 (n=129, 115, 125)Cycle 5, Day 1 (n=114, 98, 107)Cycle 7, Day 1 (n=89, 79, 84)Cycle 9, Day 1 (n=87, 66, 67)Cycle 11, Day 1 (n=71, 61, 65)Cycle 13, Day 1 (n=67, 52, 61)
Bortezomib and Dexamethasone1.3-4.9-3.3-4.2-11.6-10.2
Bortezomib, Melphalan and Prednisone2.0-0.4-4.7-1.02.81.0
Bortezomib, Thalidomide, and Dexamethasone-4.4-6.1-8.6-8.1-7.9-8.5

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Time to Alternative Therapy

Time to alternative therapy is defined as the time between randomization and alternative therapy. Participants who did not receive alternative therapy were censored at the time of last contact. (NCT00507416)
Timeframe: From randomization until alternative therapy. Median follow-up time was 43 months.

Interventionmonths (Median)
Bortezomib and Dexamethasone19.7
Bortezomib, Thalidomide, and Dexamethasone24.5
Bortezomib, Melphalan and Prednisone19.0

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Progression Free Survival (PFS)

"PFS is defined as the time from randomization to disease progression or death, whichever occurs first. Participants who did not progress and were still alive at the cut-off date were censored at the date of last contact. Response was assessed by the Investigator using the International Myeloma Working Group (IMWG) uniform response criteria.~Progressive disease requires 1 of the following:~Increase of ≥ 25% from nadir in:~Serum M-component (absolute increase ≥ 0.5 g/dl)~Urine M-component (absolute increase ≥ 200 mg/24 hours)~In patients without measurable serum and urine M-protein levels the difference between involved and uninvolved free light chain (FLC) levels (absolute increase > 100 mg/dl)~Bone marrow plasma cell percentage (absolute % ≥ 10%)~Development of new or increase in the size of existing bone lesions or soft tissue plasmacytomas.~Development of hypercalcemia (corrected serum calcium > 11.5 mg/dl) attributed solely to plasma cell proliferative disease" (NCT00507416)
Timeframe: From randomization until disease progression. Median follow-up time was 43 months.

Interventionmonths (Median)
Bortezomib and Dexamethasone14.7
Bortezomib, Thalidomide, and Dexamethasone15.4
Bortezomib, Melphalan and Prednisone17.3

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Percentage of Participants With an Overall Response

"Overall response defined as a best overall response of complete response (CR), very good partial response (VGPR) or partial response (PR), assessed by the Investigator using the IMWG uniform response criteria.~CR: Negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, and <5% plasma cells in bone marrow.~VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥ 90% reduction in serum M-protein plus urine M-protein level <100 mg per 24 hours (h).~PR requires 1 of the following:~≥50% reduction of serum M-protein and 24-h urinary M-protein by ≥ 90% or to <200 mg/24 h, or~If M-protein not measurable, a ≥50% decrease in the difference between involved and uninvolved FLC levels, or~If FLC not measurable, a ≥ 50% reduction in plasma cells, provided baseline bone marrow plasma cell percentage was ≥30%.~If present at baseline, a ≥50% reduction in the size of soft tissue plasmacytomas is also required." (NCT00507416)
Timeframe: Response assessed every other cycle for up to 13 cycles (49 weeks).

Interventionpercentage of participants (Number)
Bortezomib and Dexamethasone73
Bortezomib, Thalidomide, and Dexamethasone80
Bortezomib, Melphalan and Prednisone70

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Percentage of Participants With a Complete Response or a Very Good Partial Response

"Complete response is defined by negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, and <5% plasma cells in bone marrow.~Very good partial response is defined by serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level <100 mg per 24 hours.~Response was assessed by the Investigator using the IMWG uniform response criteria." (NCT00507416)
Timeframe: Response assessed every other cycle for up to 13 cycles (49 weeks).

Interventionpercentage of participants (Number)
Bortezomib and Dexamethasone37
Bortezomib, Thalidomide, and Dexamethasone51
Bortezomib, Melphalan and Prednisone41

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Percentage of Participants With a Complete Response

Participants with a best overall response of complete response, defined as negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, and <5% plasma cells in bone marrow. Response was assessed by the Investigator using the IMWG uniform response criteria. (NCT00507416)
Timeframe: Response assessed every other cycle, for up to 13 cycles (49 weeks).

Interventionpercentage of participants (Number)
Bortezomib and Dexamethasone3
Bortezomib, Thalidomide, and Dexamethasone4
Bortezomib, Melphalan and Prednisone4

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Overall Survival

Overall survival is defined as the time between randomization and death. Participants still alive at the cutoff date or lost to follow-up were censored at the date of last contact. (NCT00507416)
Timeframe: From randomization until death. Median follow-up time was 43 months.

Interventionmonths (Median)
Bortezomib and Dexamethasone49.8
Bortezomib, Thalidomide, and Dexamethasone51.5
Bortezomib, Melphalan and Prednisone53.1

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Duration of Response

Duration of response is defined in participants with an overall response as the time between first documentation of response and disease progression. Responders without disease progression were censored at the last clinical assessment of response. (NCT00507416)
Timeframe: From first documented response until disease progression. Median follow-up time was 43 months.

Interventionmonths (Median)
Bortezomib and Dexamethasone18.3
Bortezomib, Thalidomide, and Dexamethasone22.4
Bortezomib, Melphalan and Prednisone19.8

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Complete Hematologic Response

(NCT00520767)
Timeframe: Up to 12 months

Interventionparticipants (Number)
Melphalan, Dexamethasone, Bortezomib,16

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Overall Survival

time from day of registration until day of death. (NCT00520767)
Timeframe: time from day of registration until 72 months.

Interventionmonth (Median)
Melphalan, Dexamethasone, Bortezomib,31.1

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Time to Treatment Failure (TTF)

Time from start of treatment until date of documented disease progression, removal from protocol due to toxicity, or death from any cause. (NCT00520767)
Timeframe: start of treatment until 72 months

Interventionmonth (Median)
Melphalan, Dexamethasone, Bortezomib,18.1

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Progression-free Survival Rate

"after autologous stem cell transplantation (ASCT). Disease progression is defined using International Workshop Criteria for non-Hodgkin lymphoma37 and is defined as:~≥ 50% increase in products of diameters of any previously identified abnormal node or nodule AND/OR~appearance of any new lesions" (NCT00521014)
Timeframe: up to 3 years

Interventionyears (Mean)
Relapsed Follicular Lymphoma Patients1.759

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Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed. (NCT00536601)
Timeframe: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

InterventionProportion of participants (Number)
BuCy
Acute Leukemia33

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Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed. (NCT00536601)
Timeframe: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

InterventionProportion of participants (Number)
VCpCyTtCpTtC1500
Solid Tumors43050

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Response Rate (Complete Remission)

Response rates will be reported using descriptive statistics. (NCT00536601)
Timeframe: At 100 days

InterventionParticipants (Count of Participants)
Acute Leukemia5
Hodgkin Lymphoma17
Non-Hodgkin Lymphoma54
MM/Amyloid17
Solid Tumors7

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Overall Survival, Presented as the Estimate at 10-yrs (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. (NCT00536601)
Timeframe: Patients are followed up to maximum of 12 years

InterventionProportion of participants (Number)
Acute Leukemia33
Hodgkin Lymphoma61
Non-Hodgkin Lymphoma45
MM/Amyloid39
Solid Tumors46

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Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed. (NCT00536601)
Timeframe: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

InterventionProportion of participants (Number)
Mel120Mel200
MM/Amyloid2213

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Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed. (NCT00536601)
Timeframe: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

InterventionProportion of participants (Number)
BuCyCBV
Hodgkin Lymphoma1443

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Progression-free Survival (PFS) Distribution of Patients With HL, NHL, and MM for Each Disease-specific High-dose Therapy Regimen, Estimate Provided for 10-yr PFS (Median Follow-up Time in Survivors)

Assessed using the product-limit based Kaplan Meier method. Additionally, a 95% confidence interval of the distribution will be computed. (NCT00536601)
Timeframe: From the date of transplantation to the date of first observed disease progression or death due to any cause, assessed up to 12 years

InterventionProportion of participants (Number)
BuCyCBVVCp
Non-Hodgkin Lymphoma22370

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Number of Treated Participants With Engraftment Failure at Day 42

Engraftment failure is defined as if by day +42 participant does not have an absolute neutrophil count (ANC) >500/ul, and has no evidence of donor chimerism on bone marrow examination. (NCT00539500)
Timeframe: Participant evaluation at Day 42

Interventionparticipants (Number)
Transplantation CD133+ Cells0

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Engraftment Failure Rate

Engraftment Failure Rate is number of participants with engraftment failure out of total participants. Engraftment failure is defined as failure to achieve an absolute neutrophil count (ANC) >500/ul by day 42, and has no evidence of donor chimerism on bone marrow examination. (NCT00539500)
Timeframe: Participant evaluation at Day 42, total study up to 3 Years

InterventionParticipant (Number)
Transplantation CD133+ Cells0

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Two-year Overall Survival

Overall survival (OS) was measured from peripheral stem cell infusion to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. Participants were followed up to 2 years after transplant and Kaplan-Meier survival analysis was used to generate the two-year Overall Survival estimate presented. (NCT00544115)
Timeframe: Up to 2 years post transplant

Interventionpercentage of survival probability (Number)
Regimen I58
Regimen II50
Regimen III54
Regimen IV50
Regimen V38
Regimen VI50

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Neutrophil Engraftment - The Days Till ANC Recovery

The primary engraftment endpoint, neutrophil engraftment, is defined as the first of three consecutive days on which the absolute neutrophil count is > 500/µL. The duration and extent of neutrophil engraftment is the time from transplant to neutrophil engraftment. (NCT00544115)
Timeframe: Up to 180 days post transplant

Interventiondays (Median)
Regimen I17
Regimen II16
Regimen III15
Regimen IV14
Regimen V18
Regimen VI16

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Number of Grade 3 and Above Toxicities of Helical Tomotherapy (HT) in Combination With Fludarabine and Melphalan Followed by Allogeneic Stem Cell Transplantation.

Toxicities (adverse events) were evaluated using the modified Bearman Scale and the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. (NCT00544466)
Timeframe: 100 days post treatment

Interventionevents (Number)
Treatment (Enzyme Inhibitor, Radiation Therapy, Transplant)793

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Overall Survival on Day 180 Days Post-transplant

Overall survival (OS) was measured from peripheral stem cell infusion to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. Participants were followed up to 180 days after transplant and Kaplan-Meier survival analysis was used to generate the Overall Survival estimate at 180 days. (NCT00544466)
Timeframe: Up to 180 days post-transplant

InterventionPercent Probability (Number)
Treatment (Enzyme Inhibitor, Radiation Therapy, Transplant)81

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Survival Rate at Day 100 After Allogeneic Transplant From Umbilical Cord Blood (UCB)

Number of surviving patients at Day 100 post-transplant divided by number of patients undergone transplantation. (NCT00547196)
Timeframe: From transplant to Day 100 post-transplant

Interventionpercentage of surviving patients (Number)
Regimen I (FTBI, Cyclophosphamide, Fludarabine)100
Regimen III (TBI, Cyclophosphamide, Fludarabine)100
Regimen IV (Fludarabine, Melphalan)100

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Survival Rate at Day 180 After Allogeneic Transplant From Umbilical Cord Blood (UCB)

Number of surviving patients at Day 180 post-transplant divided by number of patients undergone transplantation. (NCT00547196)
Timeframe: From transplant up to Day 180 post-transplant

Interventionpercentage of surviving patients (Number)
Regimen I (FTBI, Cyclophosphamide, Fludarabine)60
Regimen III (TBI, Cyclophosphamide, Fludarabine)100
Regimen IV (Fludarabine, Melphalan)50

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Disease Response

Percentage of participants with partial or complete response by Bladé criteria. Partial response is defined as a >= 50% decrease in serum paraprotein or 90% decrease in urinary light chains (for participants without measurable serum paraprotein). Complete response is defined as negative serum and urine immunofixation and a bone marrow aspirate with < 5% plasma cells. (NCT00566098)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseStable DiseaseProgressive Disease
MILs in Patients Undergoing an Autologous Peripheral SCT7663

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Hematopoietic Engraftment

Days to absolute neutrophil count > 500 cells per microliter. (NCT00566098)
Timeframe: Up to 1 year

Interventiondays (Median)
MILs in Patients Undergoing an Autologous Peripheral SCT17.9

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Anti-tumor Immune Responses

Myeloma lysate response that measures the T-cell response quantified as %CD3+/CFSE-low/IFN-gamma+. (NCT00566098)
Timeframe: At time of bone marrow harvest, Day 60 post-transplant, Day 180 post-transplant, and Day 360 post-transplant

Intervention%CD3+/CFSE-low/IFN-gamma+ (Mean)
At time of bone marrow harvestDay 60 post-transplantDay 180 post-transplantDay 360 post-transplant
MILs in Patients Undergoing an Autologous Peripheral SCT1.5814.118.112.7

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Feasibility of MILs Generation as Assessed by Percentage of Participants With Successful MIL Generation

Success rate of expanding MILs in vitro and obtaining a protocol-specified product. (NCT00566098)
Timeframe: Up to 1 year

Interventionpercentage of participants (Number)
MILs in Patients Undergoing an Autologous Peripheral SCT100

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Survival

Survival in months for participants who are alive (Overall Survival) and alive without disease progression (Progression-free survival). Disease progression is defined as a change from negative to positive on immunofixation or electrophoresis for participants previously in complete remission or a 25% increase in serum electrophoresis for participants not previously in complete remission. Partial response is defined as a >= 50% decrease in serum paraprotein or 90% decrease in urinary light chains (for participants without measurable serum paraprotein). Complete response is defined as negative serum and urine immunofixation and a bone marrow aspirate with < 5% plasma cells. (NCT00566098)
Timeframe: Up to 129 months

Interventionmonths (Median)
Overall survivalProgression-free survival
MILs in Patients Undergoing an Autologous Peripheral SCT112.112.3

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T-cell Reconstitution as Determined by Absolute Lymphocyte Count (ALC)

ALC counts trending over time. (NCT00566098)
Timeframe: Days 14, 28, 60, 180, and 360

Interventioncells per microliter (Median)
Day 14Day 28Day 60Day 180Day 360
MILs in Patients Undergoing an Autologous Peripheral SCT7561768170014301660

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Pneumococcal-specific Vaccine Responses

CRM-197 Prevnar-specific vaccine responses that measure the T-cell response of the vaccine quantified as %CD3+/CFSE-low/IFN-gamma+. (NCT00566098)
Timeframe: At time of bone marrow harvest, Day 60 post-transplant, Day 180 post-transplant, and Day 360 post-transplant

Intervention%CD3+/CFSE-low/IFN-gamma+ (Mean)
At time of bone marrow harvestDay 60 post-transplantDay 180 post-transplantDay 360 post-transplant
MILs in Patients Undergoing an Autologous Peripheral SCT21.514.423.420.1

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Overall Survival (OS)

Estimate the one-year overall survival (OS) for research participants who receive this study treatment. OS is defined as time from transplantation to death due to any cause. Patient who are alive at the time of analysis will be censored. The estimated percentage of participants with OS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: one year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies71.0

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To Estimate the Rate of Overall Grade III-IV Acute GVHD, and the Rate and Severity of Chronic GVHD in Research Participants.

The rate of Overall Grade III-IV Acute AVHD will be report as the proportion of patients who have Grade III-IV Acute GVHD to the total patients with transplant. The rate of Chronic GVHD will be report as the proportion of patients who have Chronic GVHD to the total patients with transplant. The Severity of Chronic GVHD will be reported using CTCAE grading system, as a number. (NCT00566696)
Timeframe: five years post-transplant

InterventionPercentage of participants (Number)
Rate of Overall Grade III-IV Acute AVHDRate of limited grade Chronic GVHD
High-Risk Hematologic Malignancies22.589.68

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Event-free Survival (EFS)

To determine if one year event-free survival can be improved in pediatric patients undergoing a haploidentical transplant by using a reduced intensity conditioning regimen and a targeted dose T cell depleted donor product. EFS is defined as time from transplantation to the occurrence of relapse or death due to any cause. Patients who are alive at the time of analysis will be censored. The estimated percentage of participants with EFS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: one year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies54.8

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To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.

The Cumulative Incidence of Relapse will be reported as the proportion of number of relapses since transplant to the total number of patients at risk at five years post-transplant. (NCT00566696)
Timeframe: five years post-transplant

InterventionPercentage of participants (Number)
The Cumulative Incidence of Relapse at five year pEstimate±SE
High-Risk Hematologic Malignancies30.08.6

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Disease-Free Survival (DFS)

Estimate the one-year disease-free survival (DFS) for research participants who receive this study treatment. DFS is defined as time from transplantation to the occurrence of relapse or death due to relapse. Patients who are alive at the time of analysis or die due to other causes will be censored at the time of their events. The estimated percentage of participants with DFS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: One year post-transplant

InterventionPercentage of participants (Number)
High-Risk Hematologic Malignancies70.1

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Topotecan Systemic Clearance

Median topotecan systemic clearance for courses 1 and 2. (NCT00567567)
Timeframe: Day 1 of courses 1-2

InterventionL/h/m2 (Median)
Single HST (CEM)28.1
Tandem HST (CEM), Randomly Assigned28.1
Not Assigned28.5

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Event-free Survival Rate

Comparison of EFS curves, starting from the time of randomization, by treatment group (single CEM vs. tandem CEM) (NCT00567567)
Timeframe: Three years, from time of randomization

Interventionpercent probability (Number)
Single HST (CEM)48.8
Tandem HST (CEM), Randomly Assigned61.8

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Incidence Rate of Local Recurrence

Cumulative incidence rate of local recurrence comparison between ANBL0532 patients randomized or assigned to receive single CEM transplant and boost radiation versus the historical A3973 patients who were transplanted and received boost radiation. (NCT00567567)
Timeframe: Up to 3 years

InterventionPercentage 3-year cumulative incidence (Number)
Single HST (CEM)15.7

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Peak Serum Concentration of Isotretinoin in Patients Enrolled on Either A3973, ANBL0032, ANBL0931, ANBL0532 and Future High Risk Studies

Median peak serum concentration level of isotretinoin for patients enrolled on ANBL0532 (NCT00567567)
Timeframe: Day 1 of each course

InterventionMicromolar (Median)
Single HST (CEM)1.00
Tandem HST (CEM), Randomly Assigned1.36
Not Assigned1.26

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Proportion of Patients With a Polymorphism

A chi-square test will be used to test whether the response rate after two cycles of induction therapy and the presence of a polymorphism are independent in the study population. (NCT00567567)
Timeframe: Through completion of a participant's first two cycles during induction, including treatment delays, assessed up to 69 days

InterventionProportion of patients (Number)
Single HST (CEM)0.96
Tandem HST (CEM), Randomly Assigned0.96
Not Assigned0.97

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Intraspinal Extension

Percentage of patients with primary tumors with intraspinal extension. (NCT00567567)
Timeframe: Up to 5 years

InterventionPercentage of patients (Number)
Single HST (CEM)8.25
Tandem HST (CEM), Randomly Assigned9.66
Not Assigned7.78

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Response After Induction Therapy

Per the International Response Criteria: measurable tumor defined as product of longest x widest perpendicular diameter. Elevated catecholamine levels, tumor cell invasion of bone marrow also considered measurable tumor. Complete Response (CR)-no evidence of primary tumor or metastases. Very Good Partial Response (VGPR)->90% reduction of primary tumor; no metastases; no new bone lesions, all pre-existing lesions improved. Partial Response (PR)-50-90% reduction of primary tumor; >50% reduction in measurable sites of metastases; 0-1 bone marrow samples with tumor; number of positive bone sites decreased by >50%. Mixed Response (MR)->50% reduction of any measurable lesion (primary or metastases) with <50% reduction in other sites; no new lesions; <25% increase in any existing lesion. No Response (NR)-no new lesions; <50% reduction but <25% increase in any existing legions. Progressive Disease (PD)-any new/increased measurable lesion by >25%; previous negative marrow positive. (NCT00567567)
Timeframe: Study enrollment to the end of induction therapy

InterventionProportion participants that responded (Number)
Single HST (CEM)0.54
Tandem HST (CEM), Randomly Assigned0.48
Not Assigned0.35

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Surgical Response

Percentage of patients who achieved a surgical complete resection (NCT00567567)
Timeframe: Up to 3 years

InterventionPercentage of patients (Number)
Single HST (CEM)83.98
Tandem HST (CEM), Randomly Assigned84.09
Not Assigned58.89

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EFS Pts Non-randomly Assigned to Single CEM (12-18 Mths, Stg. 4, MYCN Nonamplified Tumor/Unfavorable or Indeterminant Histopathology/Diploid DNA Content & Pts>547 Days, Stg.3, MYCN Nonamplified Tumor AND Unfavorable or Indeterminant Histopathology).

Kaplan-Meier curves of EFS will be plotted, and the proportion of responders to induction therapy will be tabulated. (NCT00567567)
Timeframe: Up to 3 years

Interventionpercent probability (Number)
Single HST (CEM)73.1

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Type of Surgical or Radiotherapy Complication

The Percentage of patients who experienced surgical or radiotherapy complications will be calculated. The complications are: bowel obstruction, chylous leaf, renal injury/atrophy/loss and diarrhea. (NCT00567567)
Timeframe: Up to 3 years

InterventionPercentage of patients (Number)
Single HST (CEM)13.11
Tandem HST (CEM), Randomly Assigned12.50
Not Assigned11.85

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OS in Patients 12-18 Months, Stage 4, MYCN Nonamplified Tumor/Unfavorable Histopathology/Diploid DNA Content/Indeterminant Histology/Ploidy and Patients > 547 Days, Stage 3, MYCN Nonamplified Tumor AND Unfavorable Histopathology/Indeterminant Histology

Kaplan-Meier curves of OS will be plotted, and the proportion of responders to induction therapy will be tabulated. (NCT00567567)
Timeframe: Up to 3 years

Interventionpercent probability (Number)
Single HST (CEM)81.0

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Enumeration of Peripheral Blood Cluster of Differentiation (CD)3, CD4, and CD8 Cells

A descriptive comparison of the median number of T-cells (CD3, CD4, CD8) between treatment arms (single vs. tandem myeloablative regimens) will be performed. (NCT00567567)
Timeframe: Up to 6 months after completion of assigned myeloablation therapy

,
Interventioncells/mm^3 (Median)
CD3CD4CD8
Single HST (CEM)20073104
Tandem HST (CEM), Randomly Assigned255.581151

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Duration of Greater Than or Equal to Grade 3 Neutropenia

A logistic regression model will be used to test the ability of the number of days of neutropenia to predict the presence of a polymorphism. (NCT00567567)
Timeframe: Through completion of a participant's first cycle during induction, including treatment delays, assessed up to 39 days

InterventionDays (Median)
Single HST (CEM)7
Tandem HST (CEM), Randomly Assigned7
Not Assigned7

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Duration of Greater Than or Equal to Grade 3 Thrombocytopenia

A logistic regression model will be used to test the ability of the number of days of thrombocytopenia to predict the presence of a polymorphism. (NCT00567567)
Timeframe: Through completion of a participant's first cycle during induction, including treatment delays, assessed up to 46 days

InterventionDays (Median)
Single HST (CEM)4
Tandem HST (CEM), Randomly Assigned4
Not Assigned4

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Maximum Tolerated Dose (MTD) of Bortezomib

The maximum tolerated dose (MTD) is defined to be the dose cohort below which 3 of 6 patients experience dose limiting toxicity (DLT), or the highest dose cohort of 1.5 mg/m², if 2 DLT were not observed at any dose cohort. (NCT00571493)
Timeframe: 14 months

Interventionmg/m² (Number)
Phase I1.5
Phase II1.0

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Preliminary Estimate of Overall Response Rate (ORR)

To obtain a preliminary estimate of overall response rate (ORR). The overall response rate is calculated as the number of patients who achieved complete response (CR) and partial response (PR) divided by the total number of evaluable patients. (NCT00571493)
Timeframe: 100 day post autologous hematopoietic stem cell transplantation (ASCT), one year post ASCT

Interventionparticipants (Number)
Overall Response Rate (100 days after transplant)Overall Response Rate (1 year after transplant)
Phase II: Overall Response Rate3833

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Progression-free Survival (PFS), and Overall Survival (OS)

To obtain a preliminary estimate of PFS and OS. Overall survival (OS) is defined as time from the first chemotherapy administered on the transplant trial until death from any cause. Progression free survival (PFS)is defined as time from therapy until relapse, progression, or death from any cause. (NCT00571493)
Timeframe: one year post autologous hematopoietic stem cell transplantation (ASCT) , 5 years post ASCT

Interventionpercentage of participants (Number)
Progression Free Survival 1 year after transplantOverall Survival 1 year after transplantProgression Free Survival 5 years after transplantOverall Survival 5 years after transplant
Phase II: Progression Free Survival and Overall Survival83913267

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Response Outcomes

assessed according to the IWG Criteria (NCT00572897)
Timeframe: 180 days

,
Interventionparticipants (Number)
clinical complete responseclinical partial responseclinical improvementstable diseaseprogressive disease
Sibling Donor781120
Unrelated Donor61541

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PLT

Patients with PLT ≥20 × 109/L (NCT00572897)
Timeframe: 2 years

,
Interventionparticipants (Number)
yesno
Sibling Donor284
Unrelated Donor2014

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The Primary Endpoint is Progression-free Survival.

Number of participants alive at 2 years who are progression-free (NCT00572897)
Timeframe: 2 years

Interventionparticipants (Number)
Sibling Donor24
Unrelated Donor11

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Absolute Neutrophil Count (ANC)

Patients with ANC ≥0.5 × 10^9/L (NCT00572897)
Timeframe: 2 years

,
Interventionparticipants (Number)
yesno
Sibling Donor311
Unrelated Donor268

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Overall Survival

The number of patients alive at last follow-up. (NCT00572897)
Timeframe: 73 months

Interventionparticipants (Number)
Sibling Donor25
Unrelated Donor11

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Disease Relapse or Progression as Measured by CT Scan or PET

(NCT00574496)
Timeframe: 3 years

Interventionmonths (Median)
High-Risk or Relapsed Hodgkin Lymphoma34.3

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Overall Survival

(NCT00574496)
Timeframe: up to 8 years

Interventionmonths (Median)
High-Risk or Relapsed Hodgkin Lymphoma70.3

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Progression-free Survival at 1 Year

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions (NCT00574496)
Timeframe: 1 year

Interventionproportion of progression-free pts (Number)
High-Risk or Relapsed Hodgkin Lymphoma33.3

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Number of Platelet Transfusions Needed to Maintain Adequate Number of Platelets. (Long Term)

(NCT00577096)
Timeframe: up to 30 weeks

InterventionPlatelet Transfusions (Mean)
Usual Care3.6
Exercise2.0

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Number of Stem Cell Collection Attempts (Long Term)

(NCT00577096)
Timeframe: up to 30 weeks

InterventionStem Cell Collection Attempts (Mean)
Usual Care1.3
Exercise1.1

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Total Number of Days of Stem Cell Collection (Long Term)

(NCT00577096)
Timeframe: up to 30 weeks

InterventionDays (Mean)
Usual Care4.9
Exercise4.5

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Number of Stem Cell Collection Attempts (Short Term)

(NCT00577096)
Timeframe: up to 15 weeks

InterventionStem Cell Collection Attempts (Mean)
Usual Care1.4
Exercise1.1

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Hemoglobin Levels Before Chemotherapy and During Transplantation Period (Short Term)

Hemoglobin Levels were measured at baseline, before peripheral blood stem cell transplantation (PBSCT), During PBSCT and at hospital discharge. (NCT00577096)
Timeframe: up to 15 weeks

,
Interventiong/dl (Mean)
BaselineBefore transplantationDuring transplantationAt discharge
Exercise11.611.010.410.6
Usual Care12.110.810.110.6

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Hemoglobin Levels Before Chemotherapy and During Transplantation Period (Long Term)

Hemoglobin Levels were measured at baseline, before peripheral blood stem cell transplantation (PBSCT), during PBSCT and at hospital discharge. (NCT00577096)
Timeframe: up to 30 weeks

,
Interventiong/dl (Mean)
BaselineBefore TransplantationDuring TransplanationAt Discharge
Exercise11.712.010.811.0
Usual Care11.512.010.810.9

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Total Number of Days of Stem Cell Collection (Short Term)

(NCT00577096)
Timeframe: up to 15 weeks

InterventionDays (Mean)
Usual Care5.3
Exercise4.0

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Number of Platelet Transfusions Needed to Maintain Adequate Number of Platelets.(Short Term)

(NCT00577096)
Timeframe: up to 15 weeks

InterventionPlatelet transfusions (Mean)
Usual Care3.1
Exercise2.3

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Number of Red Blood Cell Transfusions Needed to Maintain Hemoglobin Levels (Long Term)

The targeted hemoglobin level for each participant was 10-12 g/dl. This is the number of red blood cell (RBC) transfusions administered to participants, as part of the investigational therapy algorithm, in an attempt to alleviate the anemia caused by multiple myeloma and high-dose chemotherapy. The numbers of RBC and platelet transfusions were obtained from the University of Arkansas for Medical Sciences blood bank. (NCT00577096)
Timeframe: up to 30 weeks

InterventionRBC Transfusions (Mean)
Usual Care1.8
Exercise1.0

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Number of Red Blood Cell Transfusions Needed to Maintain Hemoglobin Levels (Short Term)

The targeted hemoglobin level for each participant was 10-12 g/dl. This is the number of red blood cell (RBC) transfusions administered to participants, as part of the investigational therapy algorithm, in an attempt to alleviate the anemia caused by multiple myeloma and high-dose chemotherapy. The numbers of RBC and platelet transfusions were obtained from the University of Arkansas for Medical Sciences blood bank. (NCT00577096)
Timeframe: up to 15 weeks

InterventionRBC Transfusions (Mean)
Usual Care2.3
Exercise1.8

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Progression-free Survival at Two Years

Progression-free survival (PFS) was measured from initial treatment to disease progression or death from any cause, whichever came first. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of survival probability (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan61

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Relapse/Progression Rate at Two Years

The primary endpoint was 2-year cumulative incidence of relapse/progression (RP), defined as time from alloHCT to disease recurrence or progression. Cumulative incidences for RP was generated in a competing-risk setting, given that death events were competing events. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of cumulative incidence (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan20

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Overall Survival at Two Years

Overall survival (OS) was measured from initial treatment to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT00577278)
Timeframe: From the initial treatment to the last disease assessment, up to two years

Interventionpercentage of survival probability (Number)
0.4 mCi 90Y-Ibritumomab Tiuxetan63

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Death From GVHD

To establish the early transplant-related severe morbidity and mortality and 3-the incidence and severity of GvHD. (NCT00582933)
Timeframe: 2 years

Interventionparticipants (Number)
Transplant Patients4

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Participant Response

Number of participants evaluated using Response to Treatment in Solid Tumors (RECIST) with definitions of Complete Response (CR): disappearance of all target lesions; and, Partial Response: at least a 30% decrease in the sum of the longest diameter of target lesions, taking as reference the baseline sum longest diameter. Maintained Continued CR: participants who entered study in a CR and maintained CR post study treatment. Evaluations once a week till Day +30, then Days 30, 60, and 100 then at 6 months or until disease progression. (NCT00583622)
Timeframe: Up to 6 months

Interventionpercentage of participants (Number)
Complete Response (CR)Maintained Continued CRPartial Response (PR)No ResponseNot Evaluable
Bevacizumab + High-Dose Chemotherapy33.342.08.38.38.3

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Cumulative Incidence of Chronic GVHD

Patients were evaluated for the development of chronic GVHD from 101 days post HSCT to last contact or documented evidence of the disease. The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for GVHD were death and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients developing cGVHD (Number)
All Patients62.5

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Cumulative Incidence of Grade II-IV Acute Graft-Versus-Host Disease (GVHD) at Day 100

Patients were evaluated for the development of acute GVHD within the first 100 days post HSCT. The cumulative incidence of grade II-IV acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death and nonengraftment. (NCT00589563)
Timeframe: 100 Days Post Hematopoietic Stem Cell Transplant (HSCT)

InterventionPercentage of patients developing aGVHD (Number)
All Patients37.3

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Incidence of Disease Relapse/Progression at 2 Years Post HSCT

Patients were evaluated for relapse/progression post transplant throughout the study. The cumulative incidence of relapse/progression was determined using competing risk analysis. Competing risks for relapse were non-relapse mortality and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients who relapsed (Number)
All Patients12.5

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Event Free Survival at Two Years Post HSCT

Patients were evaluated for event free survival (EFS) throughout the study. Events were defined as death, relapse, progression, or nonengraftment. Kaplan Meier estimates were calculated as time from HSCT to event. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients with an event (Number)
All Patients61.3

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Non-relapse Mortality at 100 Days Post HSCT

Patients were evaluated for non-relapse mortality (NRM) throughout the study. Non-relapse mortality was considered any death not attributable to relapse or disease progression. The cumulative incidence of NRM was determined using competing risk analysis. Competing risks for NRM were death due to disease progression, relapse and nonengraftment. (NCT00589563)
Timeframe: 100 day point estimate was provided

InterventionPercentage of patients with a NRM (Number)
All Patients9.4

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Non-relapse Mortality at Two Years Post HSCT

Patients were evaluated for non-relapse mortality (NRM) throughout the study. Non-relapse mortality was considered any death not attributable to relapse or disease progression. The cumulative incidence of NRM was determined using competing risk analysis. Competing risks for NRM were death due to disease progression, relapse and nonengraftment. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients with a NRM (Number)
All Patients15.6

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Occurence of Sinusoidal Obstructive Syndrome (SOS)

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. This is the number of participants who developed SOS. (NCT00589563)
Timeframe: Median Follow Up: 28 Months (Range: 1-49 Months)

Interventionparticipants (Number)
All Patients1

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Occurrence of Thrombotic Microangiopathy

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. This is the number of participants who developed TMA. (NCT00589563)
Timeframe: Median Follow Up: 28 Months (Range: 1-49 months)

Interventionparticipants (Number)
All Patients7

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Overall Survival at Two Years Post HSCT

Patients were evaluated for survival (OS) throughout the study. Kaplan Meier estiamtes were calculated for overall survival using time from HSCT to death of any cause or for surviving patients last contact date. (NCT00589563)
Timeframe: 2 year point estimate was provided.

InterventionPercentage of patients who died (Number)
All Patients65.6

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Time to Absolute Neutrophil Count Recovery (Engraftment)

Absolute neutrophil count (ANC) recovery is defined as an ANC of ≥ 0.5 x 10^9/L (500/mm3) for three consecutive laboratory values obtained on different days (NCT00589563)
Timeframe: Patients were evaluated until neutrophil recovery, a median of 15 days post HSCT

InterventionDays (Median)
All Patients14.5

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Time to Platelet Count Recovery (Engraftment)

Platelet recovery is defined as the first date of three consecutive laboratory values ≥ 25 x 10^9 L obtained on different days. (NCT00589563)
Timeframe: Patients were evaluated until platelet recovery, a median of 14 days

InterventionDays (Median)
All Patients14

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Occurence of Infections Including Cytomegalovirus and Epstein-Barr Virus Reactivation

Participants were monitored throughout the trial (median of 28 months) for various infections/complications. (NCT00589563)
Timeframe: Median Follow Up: 28 months (Range: 1-49 months)

Interventionparticipants (Number)
Neither CMV or EBVCMV reactivation onlyEBV onlyBoth CMV and EBV
All Patients16934

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Severity of Acute GVHD

All patients were considered for the evaluation of the severity of acute GVHD. (NCT00589563)
Timeframe: 100 Days Post HSCT

Interventionparticipants (Number)
No Acute GVHDYes - Grade IYes- Grade IIYes- Grade IIIYes - Grade IVNo- Inevaluable (graft failures)
All Patients999104

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Severity of Chronic GVHD

All Patients were considered for the evaluation of chronic GVHD severity. (NCT00589563)
Timeframe: Patients were evaluated until they developed chronic GVHD, a median of 130 days post HSCT

Interventionparticipants (Number)
No Chronic GVHDYes- LimitedYes - ExtensiveNo- Inevaluable (graft failure/died
All Patients44177

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Number of Participants With a 2-Year Progression-Free Survival (PFS)

Response evaluated using the standard criteria response for lymphoma through CT scan. (NCT00591630)
Timeframe: 2 years (beginning day 30 after treatment)

InterventionParticipants (Count of Participants)
Group 1 - Zevalin + BEAM + Rituximab +Stem Cell Transplant6
Group 1 - Zevalin + BEAM + Stem Cell Transplant8
Group 2 - BEAM + Rituximab + Stem Cell Transplant8
Group 2 - BEAM + Stem Cell Transplant5

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Very Good Partial Response (VGPR) Rate

Response evaluation was based on the International Myeloma Working Group (IMWG) response criteria. VGPR rate was defined as patients achieving at least VGPR which include patients who achieving complete response (CR) and VGPR. CR refers to patients who have complete disappearance of an M-protein and no evidence of myeloma in the bone marrow. VGPR refers to patients who meet the following criteria: Serum and urine M-component detectable by immunofixation but not on electrophoresis; Or 90% or greater reduction in serum M-component plus urine M-component <100 mg per 24 hours; If the serum and urine M protein are unmeasurable and the immunoglobulin free light chain parameter is being used to measure response, a ≥ 90% decrease in the difference between involved and uninvolved free light chain (FLC) levels is required in place of the M protein criteria. (NCT00602641)
Timeframe: Assessed every cycle (1 cycle=28 days) for the first 12 cycles, and then every 2 cycles while on treatment. Post treatment assessed every 3 months < 2 years from study entry, every 6 months if 2-5 years, every 12 months if 6-10 years from study entry.

Interventionproportion of participants (Number)
Arm I (MPT-T)0.247
Arm II (mPR-R)0.316

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Progression-Free Survival (PFS)

PFS is defined as the time from randomization to the earlier of progression or death of any cause. (NCT00602641)
Timeframe: Assessed every 3 months for 2 years, then every 6 months for 3 years, then annually for 10 years from the date of randomization.

Interventionmonths (Median)
Arm I (MPT-T)21.0
Arm II (mPR-R)18.7

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Change in Functional Assessment of Cancer Therapy-Neurotoxicity Trial Outcome Index (FACT-Ntx TOI) Score From Baseline to Cycle 12

A combined scale was used to assess the quality of life (QOL) comprising of the well established and validated functional well-being (FWB) and physical well-being (PWB) components of FACT-G version 4 (14 questions), which will address the physical and functional well-being of multiple myeloma patients plus the FACT-neurotoxicity (NTX, 11 questions), which will evaluate symptoms of neurotoxicity. This pooled scale is referred to as the FACT Ntx TOI. The FACT-Ntx TOI has 25 items and the score ranges from 0 (worst possible outcome) to 100 (best possible outcome). (NCT00602641)
Timeframe: Administered at registration, the beginning of cycle 7 d1, the end of cycle 12 d28, then at the end of cycle 18, 24, and 38 d28. For patients who discontinue treatment early, assessed at time of discontinuation and at the next quarterly follow-up visit.

Interventionunits on a scale (Mean)
Arm I (MPT-T)-2.8
Arm II (mPR-R)3.3

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Overall Survival

Overall survival was defined as time from randomization to death from any cause. (NCT00602641)
Timeframe: Assessed every 3 months for 2 years, then every 6 months for 3 years, then annually for 10 years from the date of randomization.

Interventionmonths (Median)
Arm I (MPT-T)52.6
Arm II (mPR-R)47.7

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Incidence of Chronic GVHD

Occurrence of symptoms in any organ system fulfilling the criteria of limited or extensive chronic GvHD (Appendix III), among patients surviving > 90 days with evidence of engraftment. Patients without chronic GvHD will be censored at time of death or last follow-up. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)

The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive <21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning1

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Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD)

The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive <21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant. (NCT00618540)
Timeframe: Day 100 and Month 6

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Neutrophil Engraftment

Incidence of neutrophil recovery and donor chimerism at Day 100. (NCT00618540)
Timeframe: Day 100

Interventionparticipants (Number)
Alemtuzumab Conditioning1

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Overall Survival

Count of patients alive at 1 and 3 years. Deaths from any cause are events. Surviving patients are censored at the date of last contact. (NCT00618540)
Timeframe: Year 1, Year 3

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Platelet Engraftment

Incidence of platelet recovery and donor chimerism at Day 100. (NCT00618540)
Timeframe: Day 100

Interventionparticipants (Number)
Alemtuzumab0

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Disease-free Survival at 12 Months Post Transplantation

"This outcome is defined as survival with resolution of LCH at 12 months post transplant.~Unresolved disease for over 12 months post-transplant, progressive disease after this time period, recurrence of disease and death from any cause are considered events.~Those who survive with resolution of disease are censored at the date of last contact." (NCT00618540)
Timeframe: Year 1

Interventionparticipants (Number)
Alemtuzumab Conditioning0

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Overall Survival (OS)

OS was defined as the time from registration to death of any cause. (NCT00635024)
Timeframe: up to 2 years

Interventionmonths (Median)
Anti-thymocyte Globulin/Melphalan2.9

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Hematological Response Rate Defined as the Number of Participants Who Achieve a Confirmed Response

"Response that was confirmed on 2 consecutive evaluations during the first 4 months of treatment.~Complete Response(CR): Disappearance of M-protein from serum and urine, normalization of Free Light Chain (FLC) ratio and <5% plasma cells in bone marrow.~Very Good Partial Response(VGPR): >=90% reduction in serum M-component; Urine M-Component <100mg per 24hours.~Partial Response(PR): >=50% reduction in serum M-component and/or Urine M-Component >=90% reduction or <200mg per 24hours; or >=50% decrease in difference between involved and uninvolved FLC levels." (NCT00635024)
Timeframe: 4 months

Interventionparticipants (Number)
Anti-thymocyte Globulin/Melphalan0

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Number of Participants With Severe Non-hematological Adverse Events

Severe non-hematologic adverse events were defined as adverse events grade 3 or higher, regardless of attribution to study drug. Adverse events were graded according to the National Cancer Institute Common Toxicity Criteria for Adverse Events (NCI CTCAE version 3.0) (NCT00635024)
Timeframe: every month during treatment, up to 12 months

Interventionparticipants (Number)
YesNo
Anti-thymocyte Globulin/Melphalan10

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Progression-free Survival (PFS)

"PFS was defined as the time from registration to progression or death due to any cause.~Progression was defined as any one or more of the following:~An increase of 25% from lowest confirmed response in:~Serum M-component (absolute increase >= 0.5g/dl)~Urine M-component (absolute increase >= 200mg/24hour~Difference between involved and uninvolved Free Light Chain levels (absolute increase >= 10mg/dl)~Bone marrow plasma cell percentage (absolute increase of >=10%)~Definite development of new bone lesion or soft tissue plasmacytomas" (NCT00635024)
Timeframe: up to 2 years

Interventionmonths (Median)
Anti-thymocyte Globulin/Melphalan2.9

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Mean Symptom Severity Burden as Measured by MDASI Scores

"MD Anderson Symptom Inventory (MDASI) Scale regularly administered during the first year following transplantation. This instrument is brief, easily understood, and provides a measure of the intensities of cancer-related symptoms. Participants rate the intensity of physical, affective, and cognitive symptoms on 0 to 10 numeric scales from not present (score of 0) to as bad as you can imagine (score of 10). Participants also rate amount of interference with daily activities caused by symptoms on 0 to 10 numeric scales from did not interfere (Score of 0) to interfered completely (score of 10)." (NCT00651937)
Timeframe: The first 7 days post-transplant.

Interventionscore on a scale (Mean)
Standard Dose Stem Cell Group4.27
High Dose Stem Cell Group4.26

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Mean Symptom Severity Burden as Measured by MDASI Scores

"MD Anderson Symptom Inventory (MDASI) Scale regularly administered during the first year following transplantation. This instrument is brief, easily understood, and provides a measure of the intensities of cancer-related symptoms. Participants rate the intensity of physical, affective, and cognitive on 0 to 10 numeric scales from not present (score of 0) to as bad as you can imagine (score of 10). Participants also rate amount of interference with daily activities caused by symptoms on 0 to 10 numeric scales from did not interfere (Score of 0) to interfered completely (score of 10)" (NCT00651937)
Timeframe: 28 day course of ASCT

Interventionscore on a scale (Mean)
Standard Dose Stem Cell Group228.4
High Dose Stem Cell Group223.0

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Response Rate

Bone marrow aspirate and biopsy performed to assess complete response and overall response rate. (NCT00661544)
Timeframe: 3, 6 and 12 months

InterventionParticipants (Number)
Complete ResponseOverall Response (Complete + Partial Response)
Arsenic Trioxide + Vitamin C + Melphalan1136

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Number of Organs Improved or Stable Based on Description Below:

"Renal response - > 50% decrease in daily 24 hour proteinuria, without worsening renal insufficiency.~Hepatic response - decrease of 2 centimeters or more of the liver span and/or decrease of the alkaline phosphatase by 50% if elevated at baseline.~Cardiac response - decrease of 2 millimeters or more in mean left ventricular wall thickness in patients with baseline wall thickness > 11 mm or a decrease in New York Heart Association heart failure class.~Autonomic nervous system response - resolution of orthostatic vital signs and symptoms, and resolution of symptoms of gastric atony or of functional ileus.~Gastrointestinal response - a greater than one grade improvement in diarrhea due to biopsy proven amyloid.~Peripheral nervous system response - resolution of clinical signs of peripheral neuropathy." (NCT00679367)
Timeframe: one year

Interventionnumber of organs stable or improved (Number)
Melphalan Revlimid and Dexamethasone10

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Number of Participants With Hematologic Response

"Complete hematologic response: Absence of detectable monoclonal protein in serum or urine by immunofixation electrophoresis, bone marrow biopsy with less than 5% plasma cells without clonal dominance of kappa or lambda isotype, and normal serum free light chain assay.~Partial hematologic response: Amyloid patients have highly individualized measures of disease burden. For patients with detectable and quantifiable monoclonal marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells. For patients with a detectable monoclonal peak on serum or urine protein electrophoresis, a reduction in the peak height of 50% or more. For patients with quantifiable urinary kappa or lambda chain concentration, a 50% reduction in daily light chain excretion (concentration x 24 hour urine volume). For patients with an elevated serum free light chain assay, reduction of 50% or more." (NCT00679367)
Timeframe: one year

Interventionparticipants (Number)
Melphalan Revlimid and Dexamethasone7

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Number of Participants Removed From Study Due to Toxicities

Number of study participants removed from study treatment due to toxicities (NCT00679367)
Timeframe: One year

InterventionParticipants (Count of Participants)
Melphalan Revlimid and Dexamethasone6

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Median Overall Survival

"All patients were administered the following drugs;~Fludarabine 30mg/m2 intravenously daily at the same time over 30 min on days -7,-6,-5,-4, and -3~Melphalan 140mg/m2 IV on day -2~Stem cell infusion on day 0~Campath 20mg IV on day -7,-6,-5,-4, and -3" (NCT00683046)
Timeframe: Patients evaluated continuously with disease specific re-evaluation at day 30, 3 months, 6 months, 1 year, and as indicated thereafter up to 10 years

InterventionDays (Median)
T-cell Depleted Allogeneic Stem Cell Transplantation547

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Median Disease-free Survival

"All patients were administered the following drugs;~Fludarabine 30mg/m2 intravenously daily at the same time over 30 min on days -7,-6,-5,-4, and -3~Melphalan 140mg/m2 IV on day -2~Stem cell infusion on day 0~Campath 20mg IV on day -7,-6,-5,-4, and -3" (NCT00683046)
Timeframe: Patients evaluated continuously with disease specific re-evaluation at day 30, 3 months, 6 months, 1 year, and as indicated thereafter up to 10 years

InterventionDays (Median)
T-cell Depleted Allogeneic Stem Cell Transplantation333

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Number of Participants With Adverse Events (AEs) During the Active Treatment Phase

A TEAE is any AE occurring or worsening on or after the first treatment of any study drug, and within 30 days after the last dose of the last study drug. Severity grades according to Common Terminology Criteria for Adverse Events v3.0 (CTCAE) on a 1-5 scale: Grade 1= Mild AE, Grade 2= Moderate AE, Grade 3= Severe AE, Grade 4= Life-threatening or disabling AE, Grade 5=Death related to AE. A serious AE is any AE occurring at any dose that: • Results in death; • Is life-threatening; • Requires or prolongs existing inpatient hospitalization; • Results in persistent or significant disability/incapacity; • Is a congenital anomaly/birth defect; • Constitutes an important medical event. (NCT00689936)
Timeframe: From first dose of study drug through 28 days following the discontinuation visit from active treatment phase; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

,,
InterventionParticipants (Number)
≥ 1 adverse event (AE)≥ 1 grade (Gr) 3 or 4 AE≥ 1 grade (Gr) 5 AE≥ 1 serious adverse event (SAE)≥ 1 AE related to Lenalidomide/Dex/Mel/Pred/Thal≥ 1 AE related to Lenalidomide≥ 1 AE related to dexamethasone≥ 1 AE related to melphalan≥ 1 AE related to prednisone≥ 1 AE related to thalidomide≥1 AE related to Lenalidomide/Dex or Mel/Pred/Thal≥ 1 Gr 3 or 4 AE related to Len/Dex/Mel/Pred/Thal≥ 1 grade 3 or 4 AE related to Lenalidomide≥ 1 grade 3 or 4 AE related to dexamethasone≥ 1 grade 3 or 4 AE related to melphalan≥ 1 grade 3 or 4 AE related to prednisone≥ 1 grade 3 or 4 AE related to Thalidomide≥1Gr 3 or 4 AE related to Len/Dex or Mel/Pred/Thal≥ 1 Grade 5 AE related to Len/Dex/Mel/Pred/Thal≥ 1 Grade 5 AE related to Lenalidomide≥ 1 Grade 5 AE related to Dexamethasone≥ 1 Grade 5 AE related to melphalan≥ 1 Grade 5 AE related to prednisone≥ 1 Grade 5 AE related to Thalidomide≥1 Grade 5 AE related to Len/Dex or Mel/Pred/Thal≥1 SAE related to Len/Dex/Mel/Pred/Thal≥1 SAE related to Lenalidomide≥1 SAE related to dexamethasone≥1 SAE related to melphalan≥1 SAE related to prednisone≥1 SAE related to thalidomide≥1 SAE related to Len/Dex or Mel/Pred/Thal≥1AE leading to Len/Dex/Mel/Pred/Thal Withdrawal≥1 AE leading to Lenalidomide withdrawal≥1 AE leading to dexamethasone withdrawal≥1 AE leading to melphalan withdrawal≥1 AE leading to prednisone withdrawal≥1 AE leading to Thalidomide withdrawal≥1AE leading to Len/DexOR Mel/Pred/Thal Withdrawal≥1AE leading to Len/Dex/Mel/Pred/Thal reduction≥1 AE leading to Lenalidomide reduction≥1 AE leading to dexamethasone reduction≥1 AE leading to melphalan reduction≥1 AE leading to prednisone reduction≥1 AE leading to thalidomide reduction≥1AE leading to Len/Dex or Mel/Pred/Thal reduction≥1 AE leading to Rd or MPT interruption≥1 AE leading to Lenalidomide interruption≥1 AE leading to dexamethasone interruption≥1 AE leading to melphalan interruption≥1 AE leading to prednisone interruption≥1 AE leading to Thalidomide interruption≥1 AE leading to Len and Dex or MPT interruption
Lenalidomide and Dexamethasone Rd1853643336308501481410000269326290177000104119700051581309700064109931040008421415511800020321301280000241
Lenalidomide and Low-Dose Dexamethasone (Rd)5294535035950648242900026937334222900013117121600011195165130000951571091520009627920317000030368353319000290
Melphalan + Prednisone + Thalidomide (MPT)53948038270527004413264931454230030711831649100065521420075629427153008378146713480019947254241900328324388249

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Shift From Baseline to Most Extreme Postbaseline Value in Absolute Neutrophil Count During the Active Treatment Phase

Neutrophil counts was assessed for participants from baseline grade to most extreme severity grade using the NCI CTCAE v 3.0 grading scale. (NCT00689936)
Timeframe: Randomization to end of treatment or the data cut off of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

,,
Interventionparticipants (Number)
Normal Baseline Grade to Normal Postbaseline GradeNormal Baseline Grade to Grade 1 postbaselineNormal Baseline Grade to Grade 2 postbaselineNormal Baseline Grade to Grade 3 postbaselineNormal Baseline Grade to Grade 4 postbaselineGrade 1 Baseline to Normal postbaselineGrade1 Baseline to Grade 1 postbaselineGrade 1 Baseline to Grade 2 postbaselineGrade 1 Baseline to Grade 3 postbaselineGrade 1 Baseline to Grade 4 postbaselineGrade 2 Baseline to normal postbaselineGrade 2 Baseline to Grade 1 postbaselineGrade 2 Baseline to Grade 2 postbaselineGrade 2 Baseline to Grade 3 postbaselineGrade 2 Baseline to Grade 4 postbaselineGrade 3 Baseline to Normal postbaselineGrade 3 Baseline to Grade 1 postbaselineGrade 3 Baseline to Grade 2 postbaselineGrade 3 Baseline to Grade 3 postbaselineGrade3 Baseline to Grade 4 postbaselineGrade 4 Baseline to Normal postbaseline GradeGrade 4 Baseline to Grade 1 postbaseline GradeGrade 4 Baseline to Grade 2 postbaselineGrade 4 Baseline Grade to Grade 3 postbaselineGrade 4 Baseline to Grade 4 postbaseline
Lenalidomide and Dexamethasone Rd181338510971306111530401111850012200000
Lenalidomide and Low-Dose Dexamethasone (Rd)103961217021781725911141890022001000
Melphalan + Prednisone + Thalidomide (MPT)3779128141452211202101721100000100000

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Shift From Baseline to Most Extreme Postbaseline Value in Creatinine Clearance (CrCl) During the Active Treatment Phase

Renal function was assessed for participants from baseline to the most extreme value in creatinine clearance calculated using the Cockcroft-Gault estimation. (NCT00689936)
Timeframe: Randomization to end of treatment or the data cut off of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

,,
Interventionparticipants (Number)
CrCl< 30 mL/min to CrCl< 30 mL/minCrCl < 30 mL/min to CrCl ≥ 30 but < 50 mL/minCrCl < 30 mL/min to CrCl ≥ 50 but < 80 mL/minCrCl< 30 mL/min to ≥ 80 mL/minCrCl≥ 30 but < 50 mL/min to < 30 mL/minCrCl ≥ 30 but < 50 mL/min to CrCl ≥ 30 but < 50 mLCrCl ≥ 30 but < 50 mL/min to CrCl ≥ 50 but < 80 mLCrCl ≥ 30 but < 50 mL/min to ≥ 80 mL/minCrCl ≥ 50 but < 80 mL to CrCl< 30 mL/minCrCl ≥ 50 but < 80 mL to CrCl ≥ 30 but < 50 mL/minCrCl ≥ 50 but < 80 mL to CrCl ≥ 50 but < 80 mL/minCrCl ≥ 50 but < 80 mL to ≥ 80 mL/minCrCl ≥ 80 mL/min to CrCl< 30 mL/minCrCl ≥ 80 mL/min to CrCl ≥ 30 but < 50 mL/minCrCl ≥ 80 mL/min to CrCl ≥ 50 but < 80 mL/minCrCl ≥ 80 mL/min to CrCl ≥ 80 mL/min
Lenalidomide and Dexamethasone Rd18171482241551201130991010114
Lenalidomide and Low-Dose Dexamethasone (Rd)15187213767904112107006109
Melphalan + Prednisone + Thalidomide (MPT)1919500416520410297009121

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Kaplan Meier Estimates of Time to Treatment Failure (TTF) at the Time of Final Analysis

TTF is defined as the time between the randomization and discontinuation of study treatment for any reason, including disease progression (determined by the investigators assessment based on the IMWG response criteria), treatment toxicity, start of another anti-myeloma therapy (AMT) or death. (NCT00689936)
Timeframe: From date of randomization until the data cut-off date of 21 January 2016; median follow up for all participants was 16.1 months.

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)16.9
Lenalidomide and Dexamethasone Rd1817.2
Melphalan + Prednisone + Thalidomide (MPT)14.1

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Change From Baseline in the European Quality of Life-5 Dimensions (EQ-5D) Health Utility Index Score

EQ-5D is a self-administered questionnaire that assesses health-related quality of life. The EQ-5D descriptive health profile comprises five dimensions of health (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Each dimension has 3 levels of response: No problem (1), some problems (2), and extreme problems (3). A unique EQ-5D health state is defined by combining one level from each of the five dimensions into a single utility index score. EQ-5D index values range from -0.59 to 1.00 where higher EQ-5D scores represent better health status. A positive change from baseline score indicates improvement in health status and better health state. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-0.00.10.10.10.10.0
Lenalidomide and Low-Dose Dexamethasone (Rd)0.00.10.10.10.10.0
Melphalan + Prednisone + Thalidomide (MPT)0.00.10.10.10.10.0

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Kaplan Meier Estimates for Time to Second-line Anti-myeloma Treatment (AMT)

Time to second-line anti-myeloma therapy was defined as time from randomization to the start of another non-protocol anti-myeloma therapy. (NCT00689936)
Timeframe: From date of randomization until the data cut-off of 24 May 2013; median follow-up for all participants was 23.0 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)39.1
Lenalidomide and Dexamethasone Rd1828.5
Melphalan + Prednisone + Thalidomide (MPT)26.7

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Kaplan Meier Estimates of Duration of Myeloma Response as Determined by an Investigator Assessment at Time of Final Analysis

Duration of response was defined as the duration from the time when the response criteria were first met for CR or VGPR or PR based on IMWG criteria until the first date the response criteria were met for progressive disease or until the participant died from any cause, whichever occurred first. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment; data cut-off date of 21 January 2016; median follow-up for responders was 19.9 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)31.5
Lenalidomide and Dexamethasone Rd1821.5
Melphalan + Prednisone + Thalidomide (MPT)22.1

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Kaplan Meier Estimates of Duration of Myeloma Response as Determined by the IRAC

Duration of response was defined as the duration from the time when the response criteria were first met for CR or VGPR or PR based on IMWG criteria until the first date the response criteria were met for progressive disease or until the participant died from any cause, whichever occurred first. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median follow-up for responders was 20.1 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)35.0
Lenalidomide and Dexamethasone Rd1822.1
Melphalan + Prednisone + Thalidomide (MPT)22.3

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Kaplan Meier Estimates of Overall Survival at the Time of Final Analysis (OS)

Overall survival was defined as the time between randomization and death. Participants, who died, regardless of the cause of death, were considered to have had an event. All participants who were lost to follow-up prior to the end of the trial or who were withdrawn from the trial were censored at the time of last contact. Participants who were still being treated were censored at the last available date the participant was known to be alive. (NCT00689936)
Timeframe: From date of randomization to date of data cut-off date of 21 January 2016; median follow-up for all participants was 48.3 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)59.1
Lenalidomide and Dexamethasone Rd1862.3
Melphalan + Prednisone + Thalidomide (MPT)49.1

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Kaplan Meier Estimates of Time to Second Line Therapy AMT at the Time of Final Analysis

Time to second-line anti-myeloma therapy is defined as time from randomization to the start of another non-protocol anti-myeloma therapy. Those who do not receive another anti-myeloma therapy were censored at the last assessment or follow-up visit known to have received no new therapy. (NCT00689936)
Timeframe: From date of randomization until the data cut-off of date 21 January 2016; median follow-up for all participants was 23.0 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)36.7
Lenalidomide and Dexamethasone Rd1828.5
Melphalan + Prednisone + Thalidomide (MPT)26.7

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Kaplan Meier Estimates of Time to Treatment Failure (TTF)

TTF is defined as the time between the randomization and discontinuation of study treatment for any reason, including disease progression (determined by IRAC based on the IMWG response criteria), treatment toxicity, start of another anti-myeloma therapy (AMT) or death. (NCT00689936)
Timeframe: From date of randomization until the data cut-off of 24 May 2013; median follow-up for all participants was 16.1 months.

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)16.9
Lenalidomide and Dexamethasone Rd1817.2
Melphalan + Prednisone + Thalidomide (MPT)14.1

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Shift From Baseline to Most Extreme Postbaseline Value in Platelet Count During the Active Treatment Phase.

Improvement in platelets was assessed for participants from baseline grade to most extreme severity grade using the NCI CTCAE v 3.0 grading scale. (NCT00689936)
Timeframe: Randomization to end of treatment or the data cut off of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

,,
Interventionparticipants (Number)
Normal Baseline Grade to Normal Postbaseline GradeNormal Baseline Grade to Grade 1 postbaselineNormal Baseline Grade to Grade 2 postbaselineNormal Baseline Grade to Grade 3 postbaselineNormal Baseline Grade to Grade 4 postbaselineGrade1 Baseline to Normal postbaseline GradeGrade 1 Baseline to Grade 1 postbaselineGrade 1 Baseline to Grade 2 postbaselineGrade 1 Baseline to Grade 3 postbaselineGrade 1 Baseline to Grade 4 postbaselineGrade 2 Baseline to normal postbaseline GradeGrade 2 Baseline to Grade 1 postbaselineGrade 2 Baseline to Grade 2 postbaselineGrade 2 Baseline to Grade 3 postbaselineGrade 2 Baseline to Grade 4 postbaselineGrade 3 Baseline to Normal postbaseline GradeGrade 3 Baseline to Grade 1 postbaselineGrade 3 Baseline to Grade 2 postbaselineGrade 3 Baseline to Grade 3 postbaselineGrade 3 Baseline to Grade 4 postbaseline
Lenalidomide and Dexamethasone Rd1819721130125338191210132000001
Lenalidomide and Low-Dose Dexamethasone (Rd)19721624154134151020033100002
Melphalan + Prednisone + Thalidomide (MPT)16520827311165171010212200110

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Kaplan-Meier Estimates of PFS Based on the Response Assessment by the Investigator At the Time of Final Analysis

PFS was calculated as the time from randomization to the first documented PD or death due to any cause during the study, which ever occurred first based on the International Myeloma Working Group Uniform Response criteria (IMWG). Those who withdrew for any reason or received another anti-myeloma therapy without documented PD were censored on the date of their last response assessment, prior to receiving any other anti-myeloma therapy. Censoring rules for PFS: - No baseline assessments and no progression or death documented within the 2 scheduled assessments; Death within the lst two assessments without any adequate response assessment; Progression documented between scheduled assessments; Death between adequate assessments; no progression; study discontinuations for reasons other than PD or death; new anti-myeloma started prior to PD; death or PD after an extended lost to follow-up time period (2 or more missed scheduled assessment's). (NCT00689936)
Timeframe: From date of randomization to date of data cut-off date of 21 January 2016; median follow-up for all participants was 17.7 months

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)26.0
Lenalidomide and Dexamethasone Rd1821.0
Melphalan + Prednisone + Thalidomide (MPT)21.9

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Kaplan-Meier Estimates of Progression-free Survival (PFS) Based on the Response Assessment by the Independent Review Adjudication Committee (IRAC)

PFS was calculated as the time from randomization to the first documented PD or death due to any cause during the study, which ever occurred first based on the International Myeloma Working Group Uniform Response criteria (IMWG). Those who withdrew for any reason or received another anti-myeloma therapy without documented PD were censored on the date of their last response assessment, prior to receiving any other anti-myeloma therapy. Censoring rules for PFS: - No baseline assessments and no progression or death documented within the 2 scheduled assessments; Death within the lst two assessments without any adequate response assessment; Progression documented between scheduled assessments; Death between adequate assessments; no progression; study discontinuations for reasons other than PD or death; new anti-myeloma started prior to PD; death or PD after an extended lost to follow-up time period (2 or more missed scheduled assessment's). (NCT00689936)
Timeframe: From date of randomization until the data cut-off date of 24 May 2013. Median follow-up time for all participants was 17.1 months.

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)25.5
Lenalidomide and Dexamethasone Rd1820.7
Melphalan + Prednisone + Thalidomide (MPT)21.2

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Percentage of Participants With a Myeloma Response by Adverse Risk Cytogenetic Risk Category Based on IRAC Review.

Participants were placed in adverse and non-adverse cytogenetic risk categories at baseline and response rates evaluated. Adverse Risk: t(4;14), t(14;16), del(13q) or monosomy 13, del(17p), 1q gain Favorable Hyperdiploidy: : t(11;14), gains of 5/9/15; Normal: a normal result, gains other than 5/9/15, IgH deletion Uncertain risk: probes used for analysis cannot place participant in any of the other risk categories. Objective response = best overall response including CR, VGPR or PR based on the IRAC Review; A CR is negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPRis serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

InterventionPercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)70.0
Lenalidomide and Dexamethasone Rd1869.7
Melphalan + Prednisone + Thalidomide (MPT)58.2

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Percentage of Participants With a Myeloma Response by Favorable Hyperdiploidy Risk Cytogenetic Risk Category Based on IRAC Review

Participants were placed in adverse and non-adverse cytogenetic risk categories at baseline and response rates evaluated. Adverse Risk: t(4;14), t(14;16), del(13q) or monosomy 13, del(17p), 1q gain Favorable Hyperdiploidy: : t(11;14), gains of 5/9/15; Normal: a normal result, gains other than 5/9/15, IgH deletion Uncertain risk: probes used for analysis cannot place participant in any of the other risk categories. Objective response = best overall response including CR, VGPR or PR based on the IRAC Review; A CR is negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPRis serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)80.4
Lenalidomide and Dexamethasone Rd1881.6
Melphalan + Prednisone + Thalidomide (MPT)70.6

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Percentage of Participants With a Myeloma Response by Normal Risk Cytogenetic Risk Category Based on IRAC Review

Participants were placed in adverse and non-adverse cytogenetic risk categories at baseline and response rates evaluated. Adverse Risk: t(4;14), t(14;16), del(13q) or monosomy 13, del(17p), 1q gain Favorable Hyperdiploidy: : t(11;14), gains of 5/9/15; Normal: a normal result, gains other than 5/9/15, IgH deletion Uncertain risk: probes used for analysis cannot place participant in any of the other risk categories. Objective response = best overall response including CR, VGPR or PR based on the IRAC Review; A CR is negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPRis serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of particpants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)80.4
Lenalidomide and Dexamethasone Rd1874.8
Melphalan + Prednisone + Thalidomide (MPT)61.0

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Percentage of Participants With a Myeloma Response by Uncertain Risk Cytogenetic Risk Category Based on IRAC Review

Participants were placed in adverse and non-adverse cytogenetic risk categories at baseline and response rates evaluated. Adverse Risk: t(4;14), t(14;16), del(13q) or monosomy 13, del(17p), 1q gain Favorable Hyperdiploidy: : t(11;14), gains of 5/9/15; Normal: a normal result, gains other than 5/9/15, IgH deletion Uncertain risk: probes used for analysis cannot place participant in any of the other risk categories. Objective response = best overall response including CR, VGPR or PR based on the IRAC Review; A CR is negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPRis serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)60.5
Lenalidomide and Dexamethasone Rd1876.8
Melphalan + Prednisone + Thalidomide (MPT)57.5

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Shift From Baseline to Most Extreme Postbaseline Value in Hemoglobin During the Active Treatment Phase

Hemoglobin was assessed for participants from baseline grade to most extreme severity grade using the NCI CTCAE v 3.0 grading scale. (NCT00689936)
Timeframe: Randomization to end of treatment or the data cut off of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

,,
Interventionparticipants (Number)
Normal Baseline Grade to Normal Postbaseline GradeNormal Baseline Grade to Grade 1 postbaselineNormal Baseline Grade to Grade 2 postbaselineNormal Baseline Grade to Grade 3 postbaselineNormal Baseline Grade to Grade 4 postbaselineGrade 1 Baseline to Normal postbaselineGrade 1 Baseline to Grade 1 postbaselineGrade1 Baseline to Grade 2 postbaselineGrade 1 Baseline to Grade 3 postbaselineGrade 1 Baseline to Grade 4 postbaselineGrade 2 Baseline to normal postbaselineGrade 2 Baseline to Grade 1 postbaselineGrade 2 Baseline to Grade 2 postbaselineGrade 2 Baseline to Grade 3 postbaselineGrade 2 Baseline to Grade 4 postbaselineGrade 3 Baseline to Normal postbaselineGrade 3 Baseline to Grade 1 postbaselineGrade 3 Baseline to Grade 2 postbaselineGrade 3 Baseline to Grade 3 postbaselineGrade 3 Baseline to Grade 4 postbaselineGrade 4 Baseline to Normal postbaselineGrade 4 Baseline to Grade 1 postbaselineGrade 4 Baseline to Grade 2 postbaselineGrade 4 Baseline to Grade 3 postbaselineGrade 4 Baseline to Grade 4 postbaseline
Lenalidomide and Dexamethasone Rd18103081001261231750121354190148300011
Lenalidomide and Low-Dose Dexamethasone (Rd)639800010612825208125484001210500001
Melphalan + Prednisone + Thalidomide (MPT)92541001101232040141334711001010200102

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Percentage of Participants With an Objective Response After Second-line Anti-myeloma Treatment at the Time of Final Analysis

Objective response according to IMWG Uniform Response Criteria was defined as a best overall response including a complete response (CR), very good partial response (VGPR) or partial response (PR) based on the IRAC Review. A CR is defined s: negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPR is serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is: ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment; data cut-off date of 21 January 2016; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)46.2
Lenalidomide and Dexamethasone Rd1853.1
Melphalan + Prednisone + Thalidomide (MPT)45.7

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Percentage of Participants With an Objective Response Based on Investigator Assessment at Time of Final Analysis

Objective response according to IMWG Uniform Response Criteria was defined as a best overall response including a complete response (CR), very good partial response (VGPR) or partial response (PR) based on the IRAC Review. A CR is defined s: negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPR is serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is: ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 21 January 2016; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)80.7
Lenalidomide and Dexamethasone Rd1878.6
Melphalan + Prednisone + Thalidomide (MPT)67.5

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Percentage of Participants With an Objective Response Based on IRAC Review

Objective response according to IMWG Uniform Response Criteria was defined as a best overall response including a complete response (CR), very good partial response (VGPR) or partial response (PR) based on the IRAC Review. A CR is defined as: negative serum and urine on immunofixation, disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in BM; A VGPR is serum and urine M-protein detectable by immunofixation but not on electrophoresis or ≥90% reduction in serum M-protein and urine M-protein level <100 mg/24 hours; A PR is: ≥50% reduction of serum M-Protein and reduction in urinary M-protein by ≥90% or to <200 mg/24 hours. If present at baseline a ≥50% reduction in size of soft tissue plasmacytomas. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionpercentage of participants (Number)
Lenalidomide and Low-Dose Dexamethasone (Rd)75.1
Lenalidomide and Dexamethasone Rd1873.4
Melphalan + Prednisone + Thalidomide (MPT)62.3

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Time to First Response Based on the Investigator Assessment at the Time of Final Analysis

The time to first myeloma response was defined as the time from randomization to the time when the response criteria for at least a PR was first met based on the IMWG criteria assessed by the investigator. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 21 January 2016; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm.

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)1.8
Lenalidomide and Dexamethasone Rd181.8
Melphalan + Prednisone + Thalidomide (MPT)2.8

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Time to First Response Based on the Review by the IRAC

The time to first myeloma response was defined as the time from randomization to the time when the response criteria for at least a PR was first met based on the IMWG criteria. (NCT00689936)
Timeframe: Disease response was assessed every 28 days until end of treatment or the data cut-off date of 24 May 2013; median duration of treatment was 80.2 weeks in the Rd arm; 72 weeks in the Rd18 arm and 67.1 weeks in the MPT arm

Interventionmonths (Median)
Lenalidomide and Low-Dose Dexamethasone (Rd)1.8
Lenalidomide and Dexamethasone Rd181.8
Melphalan + Prednisone + Thalidomide (MPT)2.8

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Change From Baseline in the EORTC QLQ-C30 Appetite Loss Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Appetite Loss Scale is scored between 0 and 100, with a high score indicating a higher level of appetite loss. Negative change from Baseline values indicate improvement in appetite and positive values indicate worsening of appetite. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd182.9-3.3-8.6-6.4-5.1-7.5
Lenalidomide and Low-Dose Dexamethasone (Rd)1.3-5.9-9.8-7.3-8.1-1.0
Melphalan + Prednisone + Thalidomide (MPT)1.0-6.2-13.5-10.5-12.2-2.6

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Change From Baseline in the EORTC QLQ-C30 Cognitive Functioning Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Cognitive Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1, (Baseline) then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-1.71.80.9-1.2-2.8-2.6
Lenalidomide and Low-Dose Dexamethasone (Rd)-1.2-0.7-0.9-1.6-2.2-4.9
Melphalan + Prednisone + Thalidomide (MPT)-1.8-1.5-0.3-0.6-0.7-7.1

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Change From Baseline in the EORTC QLQ-C30 Constipation Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Constipation Scale is scored between 0 and 100, with a high score indicating a higher level of constipation. Negative change from Baseline values indicate improvement in constipation and positive values indicate worsening of constipation. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd186.30.0-5.1-5.2-5.9-7.5
Lenalidomide and Low-Dose Dexamethasone (Rd)8.31.8-2.4-2.4-4.5-7.9
Melphalan + Prednisone + Thalidomide (MPT)18.413.96.83.70.0-2.2

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Change From Baseline in the EORTC QLQ-C30 Diarrhea Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Diarrhea Scale is scored between 0 and 100, with a high score indicating a higher level of diarrhea. Negative change from Baseline values indicate improvement in diarrhea and positive values indicate worsening of diarrhea. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd182.33.46.09.110.96.4
Lenalidomide and Low-Dose Dexamethasone (Rd)3.83.78.211.814.810.8
Melphalan + Prednisone + Thalidomide (MPT)-0.6-2.4-2.2-2.5-1.7-0.5

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Change From Baseline in the EORTC QLQ-C30 Dyspnea Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnoea Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate improvement in symptoms and positive values indicate worsening symptoms. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd183.6-1.9-2.9-1.62.90.8
Lenalidomide and Low-Dose Dexamethasone (Rd)0.9-0.8-2.3-3.5-1.8-1.0
Melphalan + Prednisone + Thalidomide (MPT)4.22.00.1-1.60.47.8

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Change From Baseline in the EORTC QLQ-C30 Emotional Functioning Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Emotional Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd180.13.95.84.93.13.7
Lenalidomide and Low-Dose Dexamethasone (Rd)0.63.84.64.65.82.6
Melphalan + Prednisone + Thalidomide (MPT)1.02.15.55.15.1-0.0

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Change From Baseline in the EORTC QLQ-C30 Fatigue Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate improvement in symptoms and positive values indicate worsening symptoms. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd184.4-3.4-5.9-2.30.1-1.6
Lenalidomide and Low-Dose Dexamethasone (Rd)2.6-2.5-3.7-4.3-3.10.3
Melphalan + Prednisone + Thalidomide (MPT)2.8-1.8-4.5-3.9-4.32.7

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Change From Baseline in the EORTC QLQ-C30 Financial Difficulties Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Financial Difficulties Scale is scored between 0 and 100, with a high score indicating a higher level of financial difficulties. Negative change from Baseline values indicate improvement in financial difficulties and positive values indicate worsening of financial difficulties. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-0.3-0.4-0.31.61.80.5
Lenalidomide and Low-Dose Dexamethasone (Rd)2.11.91.40.42.01.9
Melphalan + Prednisone + Thalidomide (MPT)0.51.90.71.10.45.0

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Change From Baseline in the EORTC QLQ-C30 Insomnia Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Insomnia Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate improvement in symptoms and positive values indicate worsening symptoms. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd183.2-1.3-1.91.11.4-1.6
Lenalidomide and Low-Dose Dexamethasone (Rd)2.10.2-1.2-1.0-0.5-5.2
Melphalan + Prednisone + Thalidomide (MPT)-10.5-8.9-11.6-9.6-6.0-4.5

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Change From Baseline in the EORTC QLQ-C30 Nausea/Vomiting Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Nausea/Vomiting Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate improvement in symptoms and positive values indicate worsening symptoms. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-0.5-2.5-4.0-3.6-2.7-4.2
Lenalidomide and Low-Dose Dexamethasone (Rd)1.8-1.1-1.3-2.2-2.30.4
Melphalan + Prednisone + Thalidomide (MPT)4.0-1.2-3.9-3.9-3.91.0

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Change From Baseline in the EORTC QLQ-C30 Pain Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Pain Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate improvement in symptoms and positive values indicate worsening symptoms. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-4.4-13.1-16.1-14.7-12.4-7.9
Lenalidomide and Low-Dose Dexamethasone (Rd)-5.4-13.4-14.4-14.0-14.4-8.0
Melphalan + Prednisone + Thalidomide (MPT)-7.8-12.1-13.4-14.3-14.7-6.0

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Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Physical Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-1.44.77.67.46.83.0
Lenalidomide and Low-Dose Dexamethasone (Rd)-1.73.44.75.06.9-0.1
Melphalan + Prednisone + Thalidomide (MPT)-0.92.25.36.98.3-0.1

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Change From Baseline in the EORTC QLQ-C30 Role Functioning Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Role Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-4.66.38.69.49.13.8
Lenalidomide and Low-Dose Dexamethasone (Rd)-2.72.46.37.88.0-0.3
Melphalan + Prednisone + Thalidomide (MPT)-2.44.18.211.814.5-1.0

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Change From Baseline in the EORTC QLQ-C30 Social Functioning Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used in clinical research to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Social Functioning Scale is scored between 0 and 100, with a high score indicating better functioning/support. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-2.22.05.23.83.22.7
Lenalidomide and Low-Dose Dexamethasone (Rd)-4.30.74.02.94.2-1.2
Melphalan + Prednisone + Thalidomide (MPT)-1.42.43.45.86.0-3.5

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Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Global Health Status Domain

The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in QOL or functioning and positive values indicate improvement. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Study discontinuation
Lenalidomide and Dexamethasone Rd18-1.34.75.43.25.75.0
Lenalidomide and Low-Dose Dexamethasone (Rd)0.44.85.94.86.4-0.1
Melphalan + Prednisone + Thalidomide (MPT)1.04.36.16.54.80.3

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Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Body Image Scale

EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. EORTC QLQ-MY20 includes four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used a 4-point scale (from 1 'Not at All' to 4 'Very Much'). Scores were averaged, and transformed to a 0-100 scale; for the body image scale, a higher score indicates a better body image. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-1.50.81.5-0.4-0.31.8
Lenalidomide and Low-Dose Dexamethasone (Rd)-4.5-1.7-1.4-1.4-2.3-5.6
Melphalan + Prednisone + Thalidomide (MPT)-1.6-3.0-2.8-2.6-1.1-5.6

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Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Disease Symptoms Scale

EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. EORTC QLQ-MY20 includes four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used a 4-point scale (from 1 'Not at All' to 4 'Very Much'). Scores were averaged, and transformed to a 0-100 scale; a higher score indicates more severe disease symptom(s). (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd18-4.1-10.0-9.9-8.7-6.2-4.5
Lenalidomide and Low-Dose Dexamethasone (Rd)-4.0-9.1-8.8-7.8-8.7-3.5
Melphalan + Prednisone + Thalidomide (MPT)-4.4-7.0-7.9-6.5-7.9-3.7

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Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Future Perspective Scale

EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. EORTC QLQ-MY20 includes four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used a 4-point scale (from 1 'Not at All' to 4 'Very Much'). Scores were averaged, and transformed to a 0-100 scale; for the future perspective scale, a higher score indicates a better perspective of the future. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd183.99.212.312.111.78.8
Lenalidomide and Low-Dose Dexamethasone (Rd)4.78.59.810.812.75.8
Melphalan + Prednisone + Thalidomide (MPT)3.36.38.010.09.53.2

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Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Multiple Myeloma (EORTC QLQ-MY20) Side Effects Treatment Scale

EORTC QLQ-MY20 is a validated questionnaire to assess the overall quality of life in patients with multiple myeloma. EORTC QLQ-MY20 includes four scales: disease symptoms, treatment side-effects, future perspective, and body image. Questions used a 4-point scale (from 1 'Not at All' to 4 'Very Much'). Scores were averaged, and transformed to a 0-100 scale; a higher score represents a more severe overall side effect of treatment. (NCT00689936)
Timeframe: Cycle 1 Day 1 (Baseline), then Months 1, 3, 6, 12, 18 and Discontinuation visit

,,
Interventionunits on a scale (Mean)
Month 1Month 3Month 6Month 12Month 18Discontinuation Visit
Lenalidomide and Dexamethasone Rd184.01.2-0.41.22.3-1.0
Lenalidomide and Low-Dose Dexamethasone (Rd)2.51.01.71.92.20.6
Melphalan + Prednisone + Thalidomide (MPT)5.63.52.94.74.33.8

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Severity of Acute Graft-versus-host Disease (GVHD)

(NCT00691015)
Timeframe: Within 100 days after donor peripheral blood stem cell transplantation (PBSCT) as assessed by Glucksberg criteria

Intervention% of participants with severe aGVHD (Number)
All Participants33.3

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Overall Survival.

(NCT00691015)
Timeframe: At 2 years after PBSCT

Interventionpercentage of participants (Number)
All Participants57.4

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Karnofsky Performance Status Performance Status

"100 - Normal; no complaints; no evidence of disease. 90 - Able to carry on normal activity; minor signs or symptoms of disease. 80 - Normal activity with effort; some signs or symptoms of disease. 70 - Cares for self; unable to carry on normal activity or to do active work. 60 - Requires occasional assistance, but is able to care for most of their personal needs.~50 - Requires considerable assistance and frequent medical care. 40 - Disabled; requires special care and assistance. 30 - Severely disabled; hospital admission is indicated although death not imminent.~20 - Very sick; hospital admission necessary; active supportive treatment necessary.~10 - Moribund; fatal processes progressing rapidly. 0 - Dead" (NCT00691015)
Timeframe: At 90 days after PBSCT

Interventionunits on a scale (Median)
All Participants80

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Incidence of Infections, Including Bacterial, Fungal, and Viral Infections (i.e., CMV and EBV Reactivation, Including Post-transplant Lymphoproliferative Disorders)

(NCT00691015)
Timeframe: Within 6 months after PBSCT

Interventionpercentage of participants (Number)
All Participants80.85

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Incidence of Chronic GVHD.

(NCT00691015)
Timeframe: Within 2 years after PBSCT

Interventionpercentage of participants (Number)
All Participants44.68

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Incidence of Acute Graft-versus-host Disease (GVHD)

(NCT00691015)
Timeframe: Within 100 days after donor peripheral blood stem cell transplantation (PBSCT) as assessed by Glucksberg criteria

Interventionpercentage of participants (Number)
All Participants44.7

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Time to Engraftment (i.e., Absolute Neutrophil Recovery [ANC > 500/mm³] )

(NCT00691015)
Timeframe: post transplant, up to 4 weeks

InterventionDays (Median)
All Participants11

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Time to Response

Time to response will be measured in the population of subjects with a confirmed response as the time from the date of initiation of treatment to the date of the first documentation of a confirmed response. Full restaging was performed at 6, 12, 18, 24, 36, 48, 60, 72 months). Time to response will be estimated and plotted with the Kaplan-Meier method. The median will be calculated with 95% confidence intervals. (NCT00691704)
Timeframe: 6 months

Interventionmonths (Mean)
High-risk Multiple Myeloma2

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Overall Survival

Overall survival is defined as the time from the date of initiation of treatment to the date of death due to any cause. Overall response rate will be estimated with its 80% confidence interval, and the numbers of subjects achieving a sustained complete response (sCR), complete response (CR), very good partial response (VGPR), partial response (PR), or stable disease (SD) will be tabulated. Overall survival will be estimated and plotted with the Kaplan-Meier method. The median will be calculated with 95% confidence intervals. (NCT00691704)
Timeframe: 6 years

Interventionmonths (Mean)
High-risk Multiple Myeloma36

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Time to Progression

Time to progression (TTP) is defined for all patients as the time from initiation of treatment to disease progression with deaths owing to causes other than progression not counted as events, but censored (Durie et al., 2006). (NCT00691704)
Timeframe: 6 years

Interventionmonths (Mean)
High-risk Multiple Myeloma11

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Progression Free Survival

Progression free survival (PFS) is defined for all subjects as the time from the date of initiation of treatment to the date of first documentation of relapse, progression, or death due to any cause. Full restaging was performed at 6, 12, 18, 24, 36, 48, 60, 72 months). PFS survival will be estimated and plotted with the Kaplan-Meier method. The median will be calculated with 95% confidence intervals. (NCT00691704)
Timeframe: 2 years

Interventionparticipants (Number)
PFS at 2 yearsPFS at 1 year
High-risk Multiple Myeloma39

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Duration of Response

The duration of response, defined only among the responders, is measured from start of achieving Partial Response (PR) [first observation of PR before confirmation] to the time of disease progression, with deaths owing to causes other than progression not counted as events, but censored (Durie et al., 2006). Duration of response will be estimated and plotted with the Kaplan-Meier method. The median will be calculated with 95% confidence intervals. (NCT00691704)
Timeframe: 6 years

Interventionmonths (Mean)
High-risk Multiple Myeloma11

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Number of Patients With RIT/ZBEAM Developing Therapy Induced MDS and AML

Patient receiving the full treatment of RIT/ZBEAM developed therapy induced MDS or AML. (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) to onset of therapy induced MDS/AML, assessed up to 5 years

InterventionParticipants (Count of Participants)
Treatment (RIT, ZBEAM, ASCT)0

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Time to Platelet Recovery

Platelet recovery was defined as the first of 7 consecutive days with a platelet count ≥ 20,000/µL with no transfusions. (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) till the first of 7 consecutive days with a platelet count ≥ 20,000/µL with no transfusions

InterventionDays (Median)
Treatment (RIT, ZBEAM, ASCT)12

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Time to Neutrophil Recovery

Neutrophil recovery was defined as the first of 3 consecutive days of an absolute neutrophil count ≥ 500/µL. (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) till the first of 3 consecutive days of an absolute neutrophil count ≥ 500/µL.)

InterventionDays (Median)
Treatment (RIT, ZBEAM, ASCT)10

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Number of Patients With Active Disease at ASCT Achieving CR/PR by Day 100 After ASCT

Responses are assessed using the Revised Criteria for Malignant Lymphoma Response Definitions for Clinical Trials (Cheson et al. 2007). Complete Response (CR) defined as disappearance of all evidence of disease. Partial Response (PR) defined as regression of measurable disease and no new sites. (NCT00695409)
Timeframe: Up to Day 100 post-ASCT

InterventionParticipants (Count of Participants)
Patients With Active Disease at ASCT53

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2-Year Progression-Free Survival

Progression-free survival (PFS) was defined as time from peripheral stem cell infusion to recurrence, progression or death. In a clinical trial, measuring the progression-free survival is one way to see how well a new treatment works. Progression-free survival was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula [Breslow NE, Day NE. Statistical methods in cancer research: volume II, the design and analysis of cohort studies. IARC Sci Publ 1987;82:1-406.] (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) to first observation of progressive disease or death due to any cause, whichever comes first, assessed up to 5 years

InterventionPercentage of Participants (%) (Number)
Treatment (RIT, ZBEAM, ASCT)71

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2-Year Cumulative Incidence of Progression

The cumulative incidence was estimated after taking into account the competing risk of early death. (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) to date of first observation of progressive disease or relapsed disease, assessed up to 5 years

InterventionPercentage of Participants (%) (Number)
Treatment (RIT, ZBEAM, ASCT)28

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2-Year Overall Survival

Overall survival (OS) was measured from peripheral stem cell infusion to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. [Breslow NE, Day NE. Statistical methods in cancer research: volume II, the design and analysis of cohort studies. IARC Sci Publ 1987;82:1-406.] (NCT00695409)
Timeframe: From peripheral stem cell infusion (Day0 ASCT) to death due to any cause, assessed up to 5 years

InterventionPercentage of Participants (%) (Number)
Treatment (RIT, ZBEAM, ASCT)89

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Median Duration of Response

Duration of response is measured from date of first confirmed response until date of disease progression. (NCT00734149)
Timeframe: up to 4 years

Interventionmonths (Median)
Bortezomib+Melphalan+Prednisone: Non-ASCT19.8
Bortezomib+Melphalan+Prednisone: ASCT27.9

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Response

Overall response rate equals complete response and partial response per Southwest Oncology Group Criteria. Measurable, quantifiable protein criteria must be present. Acceptable protein criteria are quantitative immunoglobulin IgG, IgA, IgD, IgE or IgM and/or urine M-component (Bence-Jones protein). If both are present, the quantitative immunoglobulin will be followed for response. Complete Remission: The absence of bone marrow or blood findings of multiple myeloma. This includes disappearance of all evidence of serum and urine M-components on electrophoresis as by immunofixation studies. There must also be no evidence of increasing anemia. Partial Remission: A 50-74% reduction in the quantitative immunoglobulin, and if present, a 50-89% reduction in the urine M-component (Bence-Jones protein). Stable/No Remission): A <50% reduction I nthe quantitative immunoglobulin, or if the patient has light-chain disease only, a <50% reduction in the urine M-component (Bence-Jones protein. (NCT00734149)
Timeframe: 6 weeks following completion of treatment

Interventionparticipants (Number)
Bortezomib+Melphalan+Prednisone42

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Number of Patients With Any Grade or Severe Adverse Event

Number of patients with any grade or severe, defined as ≥ grade 3 by Common Terminology Criteria for Adverse Events (CTCAE) v4.0, adverse events as a measure of safety (NCT00734149)
Timeframe: At any time during the study and up to 30 days after stopping the study drug

Interventionparticipants (Number)
Bortezomib+Melphalan+Prednisone34

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Time to Progression

(NCT00743288)
Timeframe: Time from the start of treatment to progressive disease

Interventionmonths (Median)
Melphalan and Panobinostat1.6

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Overall Response Rate (ORR) and Clinical Benefit Rate (CBR) [ORR= Complete Response (CR) + Very Good Partial Response (VGPR) + Partial Response (PR)]; CBR=ORR + Minimal Response (MR)] Following Treatment With Panobinostat and Melphalan

Responses were evaluated according to criteria modified from those developed by Blade et al., 1998 The reference point for evaluating response improvement is the baseline. This baseline reference point is also valid when a patient has already achieved a response and transitions through into a better response grade. (NCT00743288)
Timeframe: 24 months

,,,,
Interventionparticipants (Number)
CRVGPRPRMRSD (stable disease)Progressive disease (PD)ORR (CR+VGPR+ PR)CBR (ORR+MR)
Melphalan and Panobinostat All Patients0210231433
Melphalan and Panobinostat Schedule A00001200
Melphalan and Panobinostat Schedule B02105133
Melphalan and Panobinostat Schedule C00005200
Melphalan and Panobinostat Schedule D000012900

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Duration of Response

(NCT00743288)
Timeframe: First evidence of PR or better (for overall response) and MR or better (for clinical benefit response) to start of disease progression or death

Interventionmonths (Median)
Melphalan and Panobinostat Schedule B8.1

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Maximum Tolerated Dose (MTD)

Phase 1: to determine the MTD of panobinostat (LBH589) in combination with melphalan to be used in the Phase 2 portion of the study (NCT00743288)
Timeframe: 12 months

Interventionmg LBH589 (Number)
Melphalan and Panobinostat Schedule D320

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MTD

Phase 1: to determine MTD of melphalan in combination with panobinostat to be used in the Phase 2 portion of the study (NCT00743288)
Timeframe: 12 months

Interventionmg/kg melphalan (Number)
Melphalan and Panobinostat Schedule D30.05

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To Describe the Incidence of Grade 3-4 Organ Toxicity

(NCT00744692)
Timeframe: 2 years post transplant

Interventionparticipants (Number)
RIC Cord Blood Transplant0

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To Describe the Pace of Neutrophil Recovery

Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions (NCT00744692)
Timeframe: 42 days post transplant

Interventiondays (Median)
RIC Cord Blood Transplant20

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To Describe the Pace of Platelet Recovery

Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions (NCT00744692)
Timeframe: 180 days post transplant

Interventiondays (Median)
RIC Cord Blood Transplant48

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To Determine the Overall Survival at day180 Post-transplant

To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis (NCT00744692)
Timeframe: 180 days

Interventionpercentage of participants (Number)
RIC Cord Blood Transplant81.8

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To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure

(NCT00744692)
Timeframe: at least 2 years post transplant

Interventionpercentage of patients (Number)
RIC Cord Blood Transplant0

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To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)

To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis (NCT00744692)
Timeframe: 100 days post transplant

Interventionpercentage of participants (Number)
RIC Cord Blood Transplant22.7

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To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant

(NCT00744692)
Timeframe: 2 years post transplant

Interventionparticipants (Number)
RIC Cord Blood Transplant0

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To Evaluate the Pace of Immune Reconstitution.

Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request. (NCT00744692)
Timeframe: 1 year post transplant

Interventioncells/uL (Median)
RIC Cord Blood Transplant805

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Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.

Determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor cells at 180 days) following reduced intensity conditioning regimens in children < 21 years receiving cord blood transplant for non-malignant disorders. (NCT00744692)
Timeframe: 180 days post transplant

Intervention% of participants (Number)
RIC Cord Blood Transplant88

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The Maximum Tolerated Dose of Bortezomib (MTD)

The Maximum Tolerated Dose of Bortezomib (MTD) Will be Defined as the Dose Level Prior to That Resulting in Two Out of Six Patients Experiencing a DLT (NCT00784823)
Timeframe: During dosing of Bortezomib on Day -4 to Day -1 of ASCT

Interventionmg/m2 of bortezomib (Number)
Combined Dose Intense Melphalan With Bortezomib (MTD)1.6

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Response Rate of the Combination of Bortezomib and Melphalan in Patients With AML and High-risk MDS.

Determine disease response to treatment using Cheson 2000 report of an international working group to standardize response criteria for myelodysplastic syndromes. (NCT00789256)
Timeframe: Post Cycle 1 through 28 days post-treatment

Interventionparticipants (Number)
Strata 15
Strata 22

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Number of Participants Surviving at 2 Years

(NCT00790647)
Timeframe: 2 years from transplant

InterventionParticipants (Count of Participants)
Stem Cell Transplant With Bortezomib and Melphalan9

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Number of Participants With Hematologic Response

"complete and partial hematologic response defined as: Complete response: absence of detectable monoclonal protein in serum and urine, and bone marrow biopsy <5% plasma cells with no clonal predominance of kappa or lambda isotype.~Partial response: any one of the following~For patients with detectable and quantifiable marrow plasmacytosis, a reduction of 50% or more in plasma cells as a percentage of nucleated bone marrow cells.~For patients with a detectable monoclonal peak on serum protein electropheresis or urine protein electropheresis, a reduction in the peak height of 50% or more.~For patients with quantifiable urinary kappa or lambda chain concentration, a reduction in daily light chain excretion (concentration x 24-hr urine volume)." (NCT00790647)
Timeframe: one year

Interventionparticipants (Number)
Stem Cell Transplant With Bortezomib and Melphalan6

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Number of Participants Surviving at 1 Year

(NCT00790647)
Timeframe: one year from transplant

Interventionparticipants (Number)
Stem Cell Transplantation With Bortezomib and Melphalan9

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Number of Participants Surviving at 100 Days From Transplant

(NCT00790647)
Timeframe: 100 Days from transplant date

Interventionparticipants (Number)
Stem Cell Transplant With Bortezomib and Melphalan9

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One Year Overall Survival

One year overall survival estimated using the product-limit method of Kaplan and Meier. Defined as the percentage of patients alive at year one after starting treatment. (NCT00792142)
Timeframe: From date of treatment initiation until death from any cause, assessed up to one year.

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant, Maintenance Treatment)95

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One Year Progression-free Survival (PFS)

"PFS estimated using the product-limit method of Kaplan and Meier. Defined as the percentage of patients progression-free at year one after starting treatment.~International Myeloma Working Group uniform response criteria for disease progression:~Increase of > 25% from baseline in Serum M-component and/or (the absolute increase must be > 0.5 g/dl); Urine M-component and/or (the absolute increase must be > 200 mg/24 h; Only in patients without measurable serum and urine M-protein levels:~the difference between involved and uninvolved FLC levels. The absolute increase must be >l0mg/dl.~Bone marrow plasma cell percentage: the absolute % must be > 10%C; Definite development of new bone lesions or soft tissue plasmacytomas.~or definite increase in the size of existing bone lesions or soft tissue plasmacytomas Development of hypercalcemia (corrected serum calcium >11.5 mg/dl or 2.65 mmol/l) that can be attributed solely to the plasma cell proliferative disorder." (NCT00792142)
Timeframe: From start of treatment initiation until disease progression, relapse or death from any cause, assessed up to 1 year.

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant, Maintenance Treatment)88

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Number of Participants With Adverse Events

All grade 3 and above treatment-related adverse events (AEs) during bortezomib/dexamethasone treatment cycles. (NCT00792142)
Timeframe: After 4 months of maintenance therapy

InterventionParticipants (Count of Participants)
LeukopeniaLymphopeniaNeutropeniaThrombocytopeniaCataractFatigueUpper respiratory infectionAnxietyPain in extremitySinus bradycardiaHyperglycemiaHypophosphatemia
Treatment (Stem Cell Transplant, Maintenance Treatment)1132112111143

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Number of Patients Surviving Progression-free

"Number of subjects surviving without progressive disease post-transplant.~Progressive disease criteria:~Greater than 25% increase in serum (absolute increase must be ≥0.5 g/dL) or urine (absolute increase must be ≥200 mg/24h) M proteins compared to best response status after autologous transplant.~Appearance of new lytic bone lesions or plasmacytomas." (NCT00793572)
Timeframe: At 2 years after the autograft

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy14

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Number of Patients Surviving Overall

Number of subjects surviving two years post autologous transplant. (NCT00793572)
Timeframe: At 2 years after the autograft

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy21

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Number of Patients With Chronic GVHD

Number of subjects with classic chronic or chronic extensive GVHD post allogeneic transplant. (NCT00793572)
Timeframe: 1 year post allo

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy10

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Number of Patients With Grade II-IV Acute GVHD

"Number of patients who developed acute GVHD post allogeneic transplant.~aGVHD Stages~Skin:~- a maculopapular eruption involving < 25% BSA~- a maculopapular eruption involving 25 - 50% BSA~- generalized erythroderma~- generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~- bilirubin 2.0 - 3.0 mg/100 mL~- bilirubin 3 - 5.9 mg/100 mL~- bilirubin 6 - 14.9 mg/100 mL~- bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT00793572)
Timeframe: 100 days post allo transplant

InterventionParticipants (Count of Participants)
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy14

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Number of Patients With Non-relapse Mortality

Number of subjects with non-relapse mortalities post allogeneic transplant. (NCT00793572)
Timeframe: 200 and 365 days after allo

InterventionParticipants (Count of Participants)
200 days post allo1 Year post allo
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy11

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Safety and Engraftment

Peripheral blood progenitor cells were collected with either chemo-mobilization (27 of 39, 69%) or growth factor mobilization (12 of 39, 31%). Patients received an average of 9.0 × 10^6/kg CD34+ cells (range, 2.3-65) as their transplant graft. (NCT00793650)
Timeframe: Day 30 after transplant

,
Interventiondays (Median)
Median time for platelet recoveryMedian time for neutrophil recovery
Bortezomib After HD melphalanAll1612
Bortezomib Before HD Melphalan1612

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Response Rate Using EBMT(European Group for Blood and Bone Marrow Transplan) Criteria at Day +100 After Transplant.

"CR :Negative immunofixation on the serum and urine and Disappearance of any soft tissue plasmacytomas and <=5% plasma cells in bone marrow.~VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level <100mg per 24 hour.~Partial Response:>=50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by >=90% or to <200mg per 24 hour." (NCT00793650)
Timeframe: 100 days after transplant

,
Interventionparticipants (Number)
Complete response (CR)VGPR-Very good partial remissionPartial Response
Bortezomib After Melphalan6117
Bortezomib Before Melphalan296

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Progression Free Survival (PFS) Rate at 2 Years After Enrollment in Untreated Patients With Multiple Myeloma.

(NCT00807599)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Continue Lenalidomide and Dexamethasone84.6
Stem Cell Transplant x 1 or x 283.3

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Overall Survival

(NCT00807599)
Timeframe: up to 4 years

Interventionpercentage of participants alive (Number)
Continue Lenalidomide and Dexamethasone95.7
Stem Cell Transplant x 1 or x 290.6

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Number of Patients That Engrafted Blood Counts by 30 Days After Transplant

Number of patients whose Absolute Neutrophil Count (ANC) recovered to >500 x10^3/uL for at least 3 consecutive days after transplant (NCT00827099)
Timeframe: Day 30

Interventionparticipants (Number)
Umbilical Cord Blood Transplant5

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Rate of Complete Donor Chimerism - Blood

"Rate of Complete Donor Chimerism - Blood~Summarized using standard descriptive statistics." (NCT00856388)
Timeframe: Day 30

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)89

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Median Time to Platelet Engraftment

"Median Time to Platelet Engraftment~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Day 100

InterventionDays (Median)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)17.0

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Day 100 TRM

Day 100 Treatment Related Mortality An exact 95% confidence interval will be provided. (NCT00856388)
Timeframe: First 100 days

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)8.44

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Acute GVHD Grade III-IV

"Acute GVHD grade III-IV~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Up to day 100

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)27

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3 yr Overall Survival

3 yr Overall Survival estimated using the Kaplan-Meier method. (NCT00856388)
Timeframe: Up to 4.5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)46.0

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1 yr Extenstive Chronic GVHD

"1 yr Extensive Chronic GVHD~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Up to 4.5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)60

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Median Time to ANC Engraftment

"Median Time to ANC Engraftment~Summarized using standard descriptive statistics along with corresponding 95% confidence intervals." (NCT00856388)
Timeframe: Days 30

InterventionDays (Median)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)16.5

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Rate of Complete Donor Chimerism - Blood

"Rate of Complete Donor Chimerism - Blood~Summarized using standard descriptive statistics." (NCT00856388)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic Stem Cell Transplant)94

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Duration of Response

Assessed as the median value for the time from first partial response until progression; death; or last follow-up. (NCT00890552)
Timeframe: 32 months

Interventionmonths (Median)
Lenalidomide, Melphalan and Dexamethasone (MDR)9.1

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Event-free Survival (EFS)

Assessed as the median value for EFS 12 months after starting MDR treatment (NCT00890552)
Timeframe: 12 months

Interventionmonths (Median)
Lenalidomide, Melphalan and Dexamethasone (MDR)3.15

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Overall Survival (OS)

Participants alive 12 months after starting MDR treatment. (NCT00890552)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Lenalidomide, Melphalan and Dexamethasone (MDR)58

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Hematologic Response Rate

At the end of each treatment cycle (4 weeks), hematologic response rate as assessed. Hematologic response was considered to be amyloid complete response (normal FLC ratio and negative serum and urine immunofixation); very good partial response (difference between involved and uninvolved FLCs [dFLC] < 40 mg/L); or partial response (dFLC decrease > 50%). (NCT00890552)
Timeframe: 8 weeks

Interventionparticipants (Number)
Complete Response (CR)Very good Partial Response (VGPR)Partial Response (PR)No Response (NR)Response not evaluable
Lenalidomide, Melphalan and Dexamethasone (MDR)24891

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Overall Response Rate (ORR)

Overall response rate (ORR) = Complete Response Rate (CRR) + Partial Response Rate (PRR) (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant7

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Overall Survival (OS)

To evaluate the graft versus myeloma effect by monitoring rate of overall survival (OS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled

Interventionpercentage of participants (Number)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant67

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Partial Response Rate (PRR)

"Partial response rate (PRR) was assessed as~> 50% reduction in serum M-protein plus urine M-protein reduction by 90% or < 200 mg/24 hr~If serum M-protein is not measurable, then > 50% reduction in the involved serum free light chain~If involved serum free light chain is not measurable, then > 50% reduction in the bone marrow plasma cell percentage + > 50% reduction in the size of any soft tissue plasmacytoma." (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant4

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Median Time to Engraftment After Allo-PBSC Transplant

"Engraftment is assessed as:~Neutrophil engraftment is > 0.5 x 10⁹/L after cytopenia~Platelet engraftment is > 20 x 10⁹/L after cytopenia" (NCT00899847)
Timeframe: 1 month

InterventionDays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Autologous-Allogeneic Hematopoietic Stem Cell Transplant2410

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Median Time to Engraftment After Auto-PBSC Transplant

"Engraftment is assessed as:~Neutrophil engraftment is > 0.5 x 10⁹/L after cytopenia~Platelet engraftment is > 20 x 10⁹/L after cytopenia" (NCT00899847)
Timeframe: 1 month

InterventionDays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Autologous-Allogeneic Hematopoietic Stem Cell Transplant1115

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Event-free Survival (EFS)

To evaluate the graft versus myeloma effect by monitoring rate of event-free survival (EFS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled

Interventionpercentage of participants (Number)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant44

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Complete Response Rate (CRR)

"Complete response rate (CRR) was assessed as all of:~Negative immunoflixation on the serum and urine~Disappearance of any soft tissue plasmacytomas~< 5% plasma cells in bone marrow" (NCT00899847)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant3

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Incidence of Graft Versus Host Disease (GvHD)

To evaluate the incidence acute GvHD of this tandem autologous/allogeneic transplant setting (NCT00899847)
Timeframe: 2 years after the last participant is enrolled.

InterventionParticipants (Count of Participants)
Autologous-Allogeneic Hematopoietic Stem Cell Transplant1

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Overall Survival

Overall survival is defined as the time interval in days between the date of randomization and the participant's death from any cause. (NCT00911859)
Timeframe: From the date of randomization till the date of death, as assessed up to the end of study (approximately 3 years)

InterventionDays (Median)
Part 2: VMP (Velcade+Melphalan+Prednisone)NA
Part 2: VMP (Velcade+Melphalan+Prednisone) + SiltuximabNA

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Duration of Response (DOR)

DOR was defined as length from the earliest date a participant achieved a complete response (CR) or partial response (PR) to either date for disease progression (including relapse from CR) or the censoring date for progressive disease. Responders without disease progression were censored at the last efficacy assessment for disease progression. (NCT00911859)
Timeframe: From the date participants achieved CR or PR to either date for disease progression (including relapse from CR) or the censoring date for progressive disease, as assessed Up to 30 days after last dose of study medication

InterventionDays (Median)
Part 2: VMP (Velcade+Melphalan+Prednisone)497
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab583

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Change From Baseline to Cycle 9 in Global Health Status/Quality of Life Subscale of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ C30)

"Global health status/quality of life is a subscale of the EORTC QOL C30, which comprises two questions related to overall health/quality of life during the past week. The raw score to each question ranged from 1 (very poor) to 7 (excellent). The raw mean score of health status/quality of life subscale is calculated for each participant and a linear transformation applied to standardize the raw score, so that scores range from 0 to 100; a higher score represents a higher (better) health and quality of life." (NCT00911859)
Timeframe: Baseline (Day 1 predose) and Cycle 9 (Week 54)

InterventionScores on a scale (Mean)
Part 2: VMP (Velcade+Melphalan+Prednisone)14.78
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab8.33

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1-year Survival Rate

Percentage of participants who are alive at the end of year 1 after randomization (NCT00911859)
Timeframe: 1 year

InterventionPercentage of participants (Number)
Part 2: VMP (Velcade+Melphalan+Prednisone)87.8
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab87.5

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1-year Progression-Free Survival (PFS) Rate

The 1-year PFS rate was defined as the percentage of participants surviving 1 year after randomization without disease progression or death. (NCT00911859)
Timeframe: 1 year

InterventionPercentage of participants (Number)
Part 2: VMP (Velcade+Melphalan+Prednisone)77.5
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab72.1

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Percentage of Participants Who Achieved Overall Response ie, Complete Response (CR) or Partial Response (PR) - European Group for Blood and Marrow Transplantation (EBMT) Criteria

CR or PR was assessed using EBMT criteria. CR: disappearance of the original monoclonal paraprotein from the blood and urine on at least 2 determinations for a minimum of 6 weeks by immunofixation studies, <5% plasma cells in the bone marrow on at least 1 determination, if skeletal survey is available: no increase in the size or number of lytic bone lesions (development of a compression fracture does not exclude response), disappearance of soft tissue plasmacytomas for at least 6 weeks; PR: >=50% reduction in the level of serum monoclonal paraprotein for at least 2 determinations 6 weeks apart, if present, reduction in 24-hour urinary light chain excretion by either >=90% or to <200 mg for at least 2 determinations 6 weeks apart, >=50% reduction in the size of soft tissue plasmacytomas (by clinical or radiographic examination) for at least 6 weeks, if skeletal survey is available: no increase in size or number of lytic bone lesions (NCT00911859)
Timeframe: Up to disease progression, approximately 3 years

InterventionPercentage of participants (Number)
Part 2: VMP (Velcade+Melphalan+Prednisone)79.6
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab87.8

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Progression-Free Survival (PFS)

PFS was defined as the time between randomization and either disease progression or death, whichever occurred first. (NCT00911859)
Timeframe: From the date of randomization until disease progression or death, whichever occurred first, as assessed up to the last efficacy assessment for disease progression (approximately 3 years)

InterventionDays (Median)
Part 2: VMP (Velcade+Melphalan+Prednisone)518
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab519

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Percentage of Participants Who Achieved Stringent Complete Response (sCR) - International Myeloma Working Group (IMWG) Criteria

sCR was assesses by IMWG Criteria: Negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, <=5% plasma cells in bone marrow, normal free light chain ratio, absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence. sCR is a CR that has been confirmed by immunofixation + free light chain assay + either bone marrow immunohistochemistry or immunofluorescence (NCT00911859)
Timeframe: Up to disease progression, approximately 3 years

InterventionPercentage of participants (Number)
Part 2: VMP (Velcade+Melphalan+Prednisone)6.1
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab4.1

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Percentage of Participants Who Achieved Complete Response (CR) - European Group for Blood and Marrow Transplantation (EBMT) Criteria

CR was assessed using EMBT criteria: disappearance of the original monoclonal paraprotein from the blood and urine on at least 2 determinations for a minimum of 6 weeks by immunofixation studies, <5% plasma cells in the bone marrow on at least 1 determination, if skeletal survey is available: no increase in the size or number of lytic bone lesions (development of a compression fracture does not exclude response), disappearance of soft tissue plasmacytomas for at least 6 weeks. (NCT00911859)
Timeframe: Up to disease progression, approximately 3 years

InterventionPercentage of participants (Number)
Part 2: VMP (Velcade+Melphalan+Prednisone)22.4
Part 2: VMP (Velcade+Melphalan+Prednisone) + Siltuximab26.5

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Progression-Free Survival (Phase 1)

"Time elapsed between Day 0 and disease progression, as defined by the International Myeloma Working Group (IMWG) disease response criteria. Disease progression is defined as an increase of >25% from lowest response value in any one or more of the following:~Serum M-component and/or (the absolute increase must be > 0.5 g/dL)~Urine M-component and/or (the absolute increase must be > 200 mg/24 h)~Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved free light chain levels. The absolute increase must be > 10 mg/dL~Bone marrow plasma cell percentage; the absolute percentage must be > 10%~Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas~Development of hypercalcaemia (corrected serum calcium > 11.5 mg/dL or 2.65 mmol/L) that can be attributed solely to the plasma cell proliferative disorder" (NCT00916058)
Timeframe: From Day 0 to first incidence of disease progression, up to 1,128 days

InterventionDays (Median)
Phase 1791

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Overall Survival at 3 Years (Phase 2)

Time elapsed between Day 0 and death from any cause, whichever came first, assessed up to 3 years. (NCT00916058)
Timeframe: From Day 0 until time of death, assessed up to 3 years.

InterventionPercentage of participants alive (Median)
Phase 288

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Overall Response Rate (Phase 2) - Number of Participants Achieving at Least a Partial Response or Better in Disease Status at Day 100 Post-transplant

Number of patients achieving at least a partial response or better in disease status at Day 100 post-transplant, as defined by the International Myeloma Working Group (IMWG) disease response criteria. Partial response in disease status is defined by the IMWG as ≥50% reduction of serum M-protein and reduction in 24-h urinary M-protein by ≥90% or to <200 mg per 24 hours; If the serum and urine M-protein are unmeasurable, a ≥50% decrease in the difference between involved and uninvolved FLC levels is required in place of the M-protein criteria; If serum and urine M-protein are unmeasurable, and serum-free light assay is also unmeasurable, ≥50% reduction in plasma cells is required in place of M-protein, provided baseline bone marrow plasma-cell percentage was ≥30%. In addition to these criteria, if present at baseline, a ≥50% reduction in the size (SPD) of soft tissue plasmacytomas is also required (NCT00916058)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Phase 233

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Progression-Free Survival (Phase 2)

"Time elapsed between Day 0 and disease progression, as defined by the International Myeloma Working Group (IMWG) disease response criteria. Disease progression is defined as an increase of >25% from lowest response value in any one or more of the following:~Serum M-component and/or (the absolute increase must be > 0.5 g/dL)~Urine M-component and/or (the absolute increase must be > 200 mg/24 h)~Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved free light chain levels. The absolute increase must be > 10 mg/dL~Bone marrow plasma cell percentage; the absolute percentage must be > 10%~Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas~Development of hypercalcaemia (corrected serum calcium > 11.5 mg/dL or 2.65 mmol/L) that can be attributed solely to the plasma cell proliferative disorder" (NCT00916058)
Timeframe: From Day 0 to first incidence of disease progression, up to 86 months

InterventionMonths (Median)
Phase 247

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Maximum Tolerated Dose (Phase 1)

The Maximum Tolerated Dose was not met in Phase 1 of the study. Phase 2 participants were enrolled at the highest dose administered in Phase 1 (Dose Level 6) and this Outcome Measure is the reported number of dose-limiting toxicities experienced by Phase 1 participants. DLTs were defined as any grade 3 non-hematologic adverse even that did not resolve within 72 hours, any occurrence of a grade 4 non-hematologic adverse event, or failure to engraft with an absolute neutrophil count of 500/mm^3 and platelet count of 20,000/mm^3 untransfused by Day 35 post-transplant. (NCT00916058)
Timeframe: 35 days post-transplant

InterventionParticipants (Count of Participants)
Dose Level 10
Dose Level 21
Dose Level 30
Dose Level 40
Dose Level 50
Dose Level 60

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Overall Survival at 2 Years (Phase 1)

Time elapsed between Day 0 and death from any cause, whichever came first, assessed up to 2 years. (NCT00916058)
Timeframe: From Day 0 until time of death, assessed up to 2 years.

InterventionPercentage of participants alive (Median)
Phase 170

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Area Under the Curve (0-t)

"AUC (0-t) was one of the pharmacokinetic endpoints to confirm pharmacokinetic similarity between Melphalan HCl for Injection (Propylene Glycol-Free) and Alkeran for Injection in patients with multiple myeloma. Pharmacokinetic similarity was defined as a difference of 20% or less in the 90% confidence intervals (CIs) for the ratios of the pharmacokinetic parameters (calculated using log-transformed data) for the two formulations.~The AUC results provided is the summary of Melphalan PK following Melphalan-Alkeran injection in Sequence 1 and Alkeran-melphalan injection in Sequence 2." (NCT00925782)
Timeframe: Day -3 and Day -2

Interventionmin*ng/mL (Geometric Mean)
Melphalan376577
Alkeran341183

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Concentration-Max (Cmax)

"Cmax was one of the pharmacokinetic endpoints to confirm pharmacokinetic similarity between Melphalan HCl for Injection (Propylene Glycol-Free) and Alkeran for Injection in patients with multiple myeloma. Pharmacokinetic similarity was defined as a difference of 20% or less in the 90% confidence intervals (CIs) for the ratios of the pharmacokinetic parameters (calculated using log-transformed data) for the two formulations.~The Cmax results provided is the summary of Melphalan PK following Melphalan-Alkeran injection in Sequence 1 and Alkeran-melphalan injection in Sequence 2." (NCT00925782)
Timeframe: Day -3 and Day -2

Interventionng/mL (Geometric Mean)
Melphalan4374
Alkeran3931

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Determination of Engraftment Following Melphalan-alkeran Sequence and Alkeran-melphalan Sequence Followed by ASCT

"Neutrophil engraftment was defined as the first day of 3 consecutive days where ANC (absolute neutrophil count) was higher than 500/ul. The two drug treatments in this cross-over design were administered on 2 subsequent days (Day -3 and Day -2). No per arm analysis was performed.~Since the two treatments were administered consecutively with 1 day rest, the time to engraftment and engraftment rate was measured after both treatments were administered." (NCT00925782)
Timeframe: 30 days

Interventiondays (Mean)
Open-label, Randomized, Crossover Study11

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Determination of Myeloablation Following Melphalan-alkeran Sequence and Alkeran-melphalan Sequence Followed by ASCT

"ANC <0.5 × 109/L, absolute lymphocyte count (ALC) <0.1 × 109/L, platelet count <20,000/mm3, or bleeding requiring transfusion. The first of 2 consecutive days for which cell counts drop below these cutoff levels was recorded as the date of myeloablation.~Since the two treatments were administered consecutively with 1 day rest, the time to myeloablation and myeloablation rate was measured after both treatments were administered.~Comparison of sequence effect was not planned in the study and due to small sample size, it was not performed." (NCT00925782)
Timeframe: 30 days

Interventiondays (Mean)
Open-label, Randomized, Cross-over Study3

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Number of Participants With Skin Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation

Toxicity was scored according to NCI/CTC version 3 (NCT00943592)
Timeframe: Day 7 until Day 30

Interventionparticipants (Number)
Grade 1-2Grade 3-4
Clofarabine, Melphalan, and Alemtuzumab68

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Number of Participants With Renal Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation

Toxicity was scored according to NCI/CTC version 3 (NCT00943592)
Timeframe: Day 7 until Day 30

Interventionparticipants (Number)
Grade 1-2Grade 3-4Grade 5
Clofarabine, Melphalan, and Alemtuzumab26133

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Number of Participants With Other Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation

Toxicity was scored according to NCI/CTC version 3 (NCT00943592)
Timeframe: Day 7 until Day 30

Interventionparticipants (Number)
Grade 1-2Grade 3-4Grade 5
Clofarabine, Melphalan, and Alemtuzumab1277

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Number of Participants With Hepatic Adverse Events During the Conditioning Regimen Prior to Stem Cell Transplantation

Toxicity was scored according to NCI/CTC version 3 (NCT00943592)
Timeframe: Day 7 until Day 30

Interventionparticipants (Number)
Grade 1-2Grade 3-4
Clofarabine, Melphalan, and Alemtuzumab4830

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Relapse Rate

(NCT00943592)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Clofarabine, Melphalan, and Alemtuzumab29

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Progression-free Survival (PFS)

Progression is defined from stem cell infusion to disease relapse, i.e., recurrence of hematologic malignancy and/or need for treatment after transplant for disease or death from any cause, whichever occurred first. (NCT00943592)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Clofarabine, Melphalan, and Alemtuzumab45

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Overall Survival (OS)

(NCT00943592)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Clofarabine, Melphalan, and Alemtuzumab59

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Percentage of Participants With Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)

"Acute GVHD is defined by the Przepiorka criteria, which stages the degree of organ involvement in the skin, liver, and gastrointestinal (GI) tract, based on severity, with Stage 1+ being least severe and stage 4+ being the most severe. Grading of acute GVHD is as follows: Grade II (skin involvement stages 1+ to 3+, liver 1+, GI tract 1+), Grade III (skin involvement stages 2+ to 3+, liver 1+, GI tract 2+ to 4+), Grade IV (skin involvement stages 4+, Liver 4+).~Chronic GVHD is assessed by NIH consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant. 2005; 11:945-56., for grading criteria. (See Citation: Filipovich AH et al)~The incidence of patients with acute GVHD (Grade II-IV) was determined at 180 days. The incidence of Chronic GVHD by 2 years was reported" (NCT00943800)
Timeframe: Up to 2 years

Interventionpercentage of patients (Number)
Acute GVHD (grade II-IV)Chronic GVHD
Treatment Group16.13.4

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Overall Survival- Percentage of Participants Who Survived at 2 Years and 5 Years

We reported overall survival at 2 years and 5 years after transplant (NCT00943800)
Timeframe: up to 5 years

Interventionpercentage of patients (Number)
Two-year overall survivalFive-year overall survival
Treatment Group43.732.9

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Percentage of Participants With Neutrophil Engraftment

Cumulative incidence of graft failure (neutrophil) by day 28 was reported. Patients who did not have neutrophil engraftment before death was considered as a competing risk. Failure to engraft was defined as lack of evidence of hematopoietic recovery (ANC <500/mm3 and platelet count < 20,000/mm3) by day +35, confirmed by a biopsy revealing a marrow cellularity < 5%. Graft failure was also defined as initial myeloid engraftment by day +35, documented to be of donor origin, followed by a drop in the ANC to < 500/mm3 for more than three days, independent of any myelosuppressive drugs, severe GVHD, CMV, or other infection. (NCT00943800)
Timeframe: Transplant (Day 0) through Day +28

Interventionpercentage of patients (Number)
Treatment Group85.1

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Progression Free Survival (PFS)

PFS: Number of participants, per treatment arm with progression free survival at time of analysis. Survival time will be measured from the date of transplant to the date of progression, death or the last follow-up, whichever comes first. Progressive Disease (PD): Increase of ≥ 25% from lowest response value in any one or more of the following: Serum M-component and/or; Urine M-component and/or; Only in patients without measurable serum and urine M-protein levels; the difference between involved and uninvolved FLC levels. The absolute increase must be > 10 mg/dL; Bone marrow plasma cell percentage; absolute percentage ≥ 10%; Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas; Development of hypercalcemia that can be attributed solely to the plasma cell proliferative disorder. (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

InterventionParticipants (Count of Participants)
A: Allogeneic Stem Cell Transplant17
B: Autologous Stem Cell Transplant25
BE: Group B Expansion19

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Percentage of Participants With Acute or Chronic Graft-versus-host Disease (GVHD) Following Transplant

Percentage of participants with Acute or Chronic GVHD following transplant (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

Interventionpercentage of participants (Number)
A: Allogenic Stem Cell Transplant34

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Overall Survival (OS) Rate

Overall survival in participants with multiple myeloma treated with Bortezomib (Velcade®) containing conditioning regimen and autologous as well as allogeneic transplantation. (NCT00948922)
Timeframe: End of 2 year, post transplant follow-up

Interventionpercentage (Number)
A: Allogeneic Stem Cell Transplant79.2
B: Autologous Stem Cell Transplant89.2
BE: Group B Expansion97.3

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Number of Participants With Dose Limiting Toxicities (DLT) (Phase 1)

Safety assessments will be evaluated as the proportion of patients experiencing the following: treatment-related grade 3 or higher toxicity (hematologic and nonhematologic) and treatment-related death. (NCT00985907)
Timeframe: Up to 2 cycles of treatment, approximately 56 days

InterventionParticipants (Count of Participants)
Doxil® + Melphalan + Velcade (DMV)0

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Number of Participants With Dose Limiting Toxicity During the Phase I (Cycle 1)

Dose limiting toxicity defined as an adverse event or adverse drug reaction experienced by the participants during 6 weeks of treatment Cycle 1 (NCT00985959)
Timeframe: 6 weeks

InterventionParticipants (Number)
Phase I - JNJ-26866138 0.7 mg/m2 Group0
Phase I - JNJ-26866138 1.0 mg/m2 Group0
Phase I - JNJ-26866138 1.3 mg/m2 Group1

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Number of Participants With Overall Response (Complete Response [CR] + Partial Response [PR]) - Phase I and II

Response is evaluated as per the criteria for evaluating disease response and progression in patients with multiple myeloma treated by high-dose therapy and haemopoietic stem cell transplantation (Blade et al. 1998). CR: disappearance of the original monoclonal protein from the blood and urine and <5% plasma cells in the bone marrow on at least 2 determinations for a minimum of 6 weeks; no increase in the size or number of lytic bone lesions; disappearance of soft tissue plasmacytomas for at least 6 weeks. PR: ≥50% reduction in the level of serum monoclonal protein for at least 2 determinations 6 weeks apart; If present, reduction in 24-hour urinary light chain excretion by either ≥90% or to <200 mg for at least 2 determinations 6 weeks apart; ≥50% reduction in the size of soft tissue plasmacytomas for at least 6 weeks; no increase in size or number of lytic bone lesions (NCT00985959)
Timeframe: 54 weeks

InterventionParticipants (Number)
Phase I - JNJ-26866138 0.7 mg/m2 Group6
Phase I - JNJ-26866138 1.0 mg/m2 Group5
Phase I - JNJ-26866138 1.3 mg/m2 Group4
Phase II - JNJ-26866138 1.3 mg/m2 Group60
Total71

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Maximum Observed Plasma Concentration (Cmax) of Melphalan - Phase I

Cmax of melphalan at dose of 9 mg/m2 on Cycle 2/Day 4 (NCT00985959)
Timeframe: Day 4 of Cycle 2

Interventionng/mL (Mean)
Melphalan100.2

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Maximum Observed Plasma Concentration (Cmax) of Prednisolone - Phase I

Cmax of Prednisolone at dose of 60 mg/m2 on Cycle 2/Day 4 (NCT00985959)
Timeframe: Day 4 of Cycle 2

Interventionng/mL (Mean)
Prednisolone1131

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Maximum Observed Plasma Concentration (Cmax) of Bortezomib (JNJ-26866138 Alone) - Phase I

Cmax of bortezomib following intravenous administration of JNJ-26866138 at dose of 0.7, 1.0, and 1.3 mg/m2 on Cycle 1/Day 25 (JNJ-26866138 alone) (NCT00985959)
Timeframe: Day 25 of Cycle 1

Interventionng/mL (Mean)
Phase I - JNJ-26866138 0.7 mg/m2 Group45.43
Phase I - JNJ-26866138 1.0 mg/m2 Group59.42
Phase I - JNJ-26866138 1.3 mg/m2 Group120.3

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Maximum Observed Plasma Concentration (Cmax) of Bortezomib (JNJ-26866138 in Combination With Melphalan and Prednisolone) - Phase I

Cmax of bortezomib following intravenous administration of JNJ-26866138 at dose of 0.7, 1.0, and 1.3 mg/m2 on Cycle 2/Day 4 (combination with melphalan and prednisolone) (NCT00985959)
Timeframe: Day 4 of Cycle 2

Interventionng/mL (Mean)
Phase I - JNJ-26866138 0.7 mg/m2 Group34.40
Phase I - JNJ-26866138 1.0 mg/m2 Group69.50
Phase I - JNJ-26866138 1.3 mg/m2 Group88.87

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Median Time to First Response - Phase II

Time to first response is the duartion of time required to achieve first response to treatment (NCT00985959)
Timeframe: up to 54 weeks

InterventionDays (Median)
Phase II - JNJ-26866138 1.3 mg/m2 Group51

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Event-free Survival

Number of patients alive without disease progression/relapse (NCT00992446)
Timeframe: 6.64 Years Post-Transplant

InterventionParticipants (Count of Participants)
Alive without disease porgressionalive with disease progression
Treatment (Chemotherapy, ASCT, Bortezomib, Vorinostat))145

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Overall Survival

Number of patients alive who received maintenance therapy (NCT00992446)
Timeframe: 6.64 Years Post-Transplant

Interventionparticipants (Number)
AliveDead
Treatment (Chemotherapy, ASCT, Bortezomib, Vorinostat))163

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Median Time to Disease Progression

median days from transplant to relapse/progression (NCT00992446)
Timeframe: time post ASCT to progression

Interventionyears (Median)
Treatment (Chemotherapy, ASCT, Bortezomib, Vorinostat))1.05

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Toxicity of Vorinostat Bortezomib Maintenance Therapy After Autologous Transplant

Number of patients on maintenance therapy post-transplant who experienced grade 3 or higher toxicity per NCI-Common Terminology Criteria for Adverse Events, version 3. The first three months of bortezomib and vorinostat therapy will be used as the time period to evaluate toxicity for stopping rules of the study. Toxicity that meets stopping rules will be determined based on the number of patients that are withdrawn from study for significant toxicity (grade IV, non-hematological, non-metabolic, non-peripheral neuropathy). (NCT00992446)
Timeframe: 3 months after start of maintenance therapy

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, ASCT, Bortezomib, Vorinostat))19

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Median Time to Platelet Engraftment

(NCT01005576)
Timeframe: 100 days

Interventiondays (Median)
Conditioning Regimen27.5

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Primary Objective: Event-free Survival at 1 Year.

(NCT01005576)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Conditioning Regimen17

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Incidence of Disease Recurrence

(NCT01005576)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Conditioning Regimen1

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Median Time to ANC Engraftment

(NCT01005576)
Timeframe: 100 days

Interventiondays (Median)
Conditioning Regimen14

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Event-Free Survival (EFS)

Number of patients surviving without relapsed/progressive disease (NCT01008462)
Timeframe: 1 Year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)9

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Number of Patients Who Engrafted

Number of patients with donor engraftment. (NCT01008462)
Timeframe: Day 84 post-allograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)13

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Number of Patients With Grade II-IV Acute Graft-versus-Host-Disease and/or Chronic Extensive Graft-versus-Host-Disease

"aGVHD The diagnosis of aGVHD is identified through various stages and grading of the disease related to Skin (Rash), Gut (Diarrhea, Nausea/vomiting and/or anorexia) and the liver (Bilirubin) assessed by severity and grading scale outlined in the section Grafts vs Hosts by Sullivan (1999).~GVHD Grades Grade I: 1-2 Skin Rash; No gut or liver involvement Grade II: Stage 1-3 Skin rash; Stage 1 gut and/or stage 1 liver involvement Grade III: Stage 2-4 gut involvement and/or stage 2-4 liver involvement with or without rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death~CGVHD The diagnosis of cGVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD." (NCT01008462)
Timeframe: 1 year post-allograft,

InterventionParticipants (Count of Participants)
Acute Graft-versus-Host-DiseaseChronic extensive Graft-versus-Host-Disease
Treatment (Autologous HCT, Donor HCT)81

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Non-relapse Mortality (NRM)

Number of patients with non-relapse mortalities. (NCT01008462)
Timeframe: 200 days and 1 Year post-allograft

InterventionParticipants (Count of Participants)
200 days1 Year
Treatment (Autologous HCT, Donor HCT)01

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Overall Survival

Number of patients surviving one year post-autograft (NCT01008462)
Timeframe: 1 year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)10

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Number of Patients With Relapsed/Progressive Disease

"Relapse/Progression defined as:~Nodes, liver, and/or spleen ≥50% increased or new by physical exam / imaging studies.~Circulating lymphocytes ≥50% increased by morphology and/or flow cytometry. Richter's transformation by lymph node biopsy ." (NCT01008462)
Timeframe: 1 year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)6

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Number of Patients Who Had Infections

Number of patients who had infections. (NCT01008462)
Timeframe: 1 Year post-autograft

InterventionParticipants (Count of Participants)
Treatment (Autologous HCT, Donor HCT)16

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Number of Participants With Non-relapse Mortality (NRM)

Non-relapse mortality (NRM) is defined as death from any cause other than relapse disease. Bayesian monitoring scheme described in Thall, Simon, and Estey (1996) employed to perform interim monitoring of the data during the course of the trial separately within each group. (NCT01010217)
Timeframe: At 100 days

InterventionParticipants (Count of Participants)
Haplo Arm8
1AgMM Related/Unrelated Donors7
MUD Donor2
Second Transplant0
Myelofibrosis0

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Disease Free Survival

Participants who have survived without their original disease. (NCT01010217)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Haplo Arm50
1AgMM Related/Unrelated Donors24
MUD Donor12
Second Transplant0
Myelofibrosis0

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cGVHD

Chronic graft vs host disease (NCT01010217)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Haplo Arm11
1AgMM Related/Unrelated Donors7
MUD Donor0
Second Transplant0
Myelofibrosis0

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Engraftments

(NCT01010217)
Timeframe: Day 28

InterventionParticipants (Count of Participants)
Haplo Arm80
1AgMM Related/Unrelated Donors50
MUD Donor21
Second Transplant0
Myelofibrosis0

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Grade III-IV aGVHD

Acute Graft vs host disease (NCT01010217)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Haplo Arm11
1AgMM Related/Unrelated Donors7
MUD Donor1
Second Transplant0
Myelofibrosis0

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Event-free Survival

The Kaplan-Meier method will be used to estimate the event-free survival distribution. (NCT01035463)
Timeframe: 1 year

Interventionpercentage of participants (Number)
All Phase I Participants84
All Phase II Participants87

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Overall Survival

The Kaplan-Meier method will be used to estimate the overall survival distribution. This outcome only reports data as it pertains to overall survival at one year. All-cause mortality includes survival for follow up for all subjects on the study. (NCT01035463)
Timeframe: 1 year

Interventionpercentage of participants (Number)
All Phase I Participants100
All Phase II Participants95

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Maximum Tolerated Dose of Lenalidomide (Phase I)

The Maximum Tolerated Dose (MTD) is defined to be the dose cohort below which 3 out of 6 subjects experience dose limiting toxicities during cycle 1. Dose limiting toxicities graded using the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCT01035463)
Timeframe: Cycle 1, 28 days

Interventionmilligrams PO daily (Number)
Treatment (Stem Cell Transplantation)10

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Response Rates by Blade Criteria

"Number of participants with each disease response category utilizing the Blade criteria:~Complete Response (CR): Defined as negative serum and urine immunofixation and a bone marrow aspirate with < 5% plasma cells.~Near Complete Response (nCR): Defined as negative serum and urine paraprotein, positive serum and/or urine immunofixation, and a bone marrow aspirate with < 5% plasma cells.~Very Good Partial Response (VGPR): Defined as negative serum and urine paraprotein with positive serum and/or urine immunofixation; or a 90% decrease in serum paraprotein with urine paraprotein < 100 mg/24 hours.~Partial Response (PR): Defined as a 50-89% decrease in serum paraprotein.~Minimal Response (MR): Defined as a 25-49% decrease in serum paraprotein.~Stable Disease (SD): Defined as not falling into any other response category.~Overall response rate (ORR): Total of CR, nCR, VGPR, and PR." (NCT01045460)
Timeframe: Up to 1 year

,
InterventionParticipants (Count of Participants)
CRnCRVGPRPRMRSDORR
ASCT + MILs23140410
ASCT + MILs + Vaccine52151213

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Safety as Measured by Grade 3-5 Adverse Events

Number of participants who experienced at least one grade 3-5 adverse event by CTCAE 3.0 that was attributed to MILs or the myeloma vaccine. (NCT01045460)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
ASCT + MILs0
ASCT + MILs + Vaccine1

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Overall Survival

Number of participants alive at 5 years (overall survival). (NCT01045460)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
ASCT + MILs7
ASCT + MILs + Vaccine9

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Progression-free Survival

Median number of months that participants were alive without disease relapse or progression (progression-free survival). (NCT01045460)
Timeframe: Up to 5 years

Interventionmonths (Median)
ASCT + MILs15.5
ASCT + MILs + Vaccine18.6

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Feasibility as Measured by Participant Withdrawal or Removal

Number of participants who withdrew or were removed from the study for reasons other than lack of efficacy prior to completion. (NCT01045460)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
ASCT + MILs3
ASCT + MILs + Vaccine3

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Serious Infections

Serious infections are reported as the number of participants experienced serious infections. (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)6

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Overall Survival (OS), 1 Year

Assessed as subjects remaining alive 12 months after CD34+ cell infusion (ie, excludes death due to any cause) (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Number)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)2

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Median Overall Survival (OS)

Reported as the median overall survival (OS) in months from infusion of the hematopoietic stem cells (HSCT) (NCT01050764)
Timeframe: 25 months

Interventionmonths (Median)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)3.7

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Maximum-tolerated Dose (MTD) of Regulatory and Conventional T-cells

The maximum-tolerated dose (MTD) was to be determined based on the safety and feasibility observed for a pre-determined set of cellular dose level combinations of regulatory T-cells (T-reg) and conventional T-cells (T-con). (NCT01050764)
Timeframe: 30 days after HSCT infusion

Interventioncells/kg (Number)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)NA

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Acute Graft-versus-Host-Disease (aGvHD)

The primary outcome was incidence of grade 3 or 4 acute graft-vs-host-disease (aGvHD), reported as the number of participants developing grade 3 or 4 aGvHD. (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)1

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To Measure the Incidence and Severity of Acute and Chronic GvHD

Population of participants that received HSCT and T-reg plus T-con, and developed actue, chronic, or any graft vs host disease (GvHD) (NCT01050764)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Acute GvHDChronic GvHDAny GvHD (actue + chronic)
Allogeneic T-Cell Infusion After Stem Cell Transplant (SCT)123

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Proportion of Patients With Hematologic Overall Response (Partial Response [PR]+ Very Good PR [VGPR]+ Amyloid Complete Response [ACR]+ Stringent Complete Response [sCR]) After 3 Months (3 Cycles) of Therapy

sCR: ACR and no clonal cells in bone marrow (BM) ACR: Negative serum/urine immunofixation (IF), <5% plasma cells in BM, and normal serum FLC ratio VGPR: 1. PR and any of the following; 2. serum/urine M-protein detectable by IF but not measurable (NM) on electrophoresis (EP); (3) ≥90% reduction in serum M-component and urine M-protein <100 mg/24 hr if baseline serum measurable; (4) urine M-component <100 mg/24 hr and NM serum M-protein on serum protein EP if baseline urine measurable; (5) ≥90% drop in the difference between involved and uninvolved FLC levels if only FLC measurable PR: (1) ≥50% drop of serum M-protein and 24-hr urinary M-protein drop by ≥90% or to <200 mg/24 hr if baseline serum/urine measurable; or (2) ≥50% drop of serum M-protein if only serum measurable at baseline; or (3) 24-hr urinary M-protein drop by ≥90% or to <200 mg/24 hr if baseline urine measurable; or (4) ≥ 50% drop in the difference between involved and uninvolved FLC if only FLC measu (NCT01078454)
Timeframe: Assessed at 3 months

InterventionProportion of patients (Number)
Arm A (Mel-Dex)0.33
Arm B (B-Mel-Dex)0.60

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Number of Participants With Response (CR at Day 90)

Response is defined as the event that the participant is alive with complete response (CR) at day 90 (+/-30 days). CR defined as: A) Absence of monoclonal protein in urine and serum when analyzed by immunofixation electrophoresis. B) The bone marrow should be normal by morphological examination with <5% plasma cells. There should be < 1% aneuploid light chain restricted population by flow cytometry for DNA/cIg. C) While healing of bone lesions not required, no new lytic lesion should appear. Further compression fracture of spine will be not considered as progressive disease. (NCT01079936)
Timeframe: Day 90 after stem cell transplant

Interventionparticipants (Number)
25 Mg Lenalidomide0
50 mg Lenalidomide0
75 mg Lenalidomide4
100 mg Lenalidomide4

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Participants With Grade 3 =/> Adverse Events

Number of participants experiencing adverse events above a Grade 3 according to the Common Terminology Criteria for Adverse Events (CTCAE) version 2. (NCT01079936)
Timeframe: Day 90 after stem cell transplant

Interventionparticipants (Number)
25 Mg Lenalidomide3
50 mg Lenalidomide5
75 mg Lenalidomide15
100 mg Lenalidomide17

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Maximum Tolerated Dose (MTD) of Lenalidomide

There were 4 doses of lenalidomide in the dose escalation phase: 25 mg, 50 mg, 75 mg, and 100 mg. The first 12 patients were treated at these dose levels (3 patients per level) and safety assessed at each level. The MTD dose level was to be the level at which participants at each lenalidomide dose level had no dose limiting toxicity (DLT). DLT defined as as regimen-related death, graft failure, grade 3 or 4 atrial fibrillation, grade 4 deep venous thrombosis, or pulmonary embolism before day 30 after auto-HCT. Each participant received a fixed dose of Melphalan plus one of the four doses 25, 50, 75 or 100 mg of Lenalidomide orally for each of 7 days, -8 to -2 pre transplant. (NCT01079936)
Timeframe: Assessed at 21-28 Day Cycle

Interventionmg/day (Number)
Lenalidomide + High-Dose Melphalan100

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Number of Participants With Day 30 DLT (Overall Study, Phase I/Phase II)

Dose limiting toxicity (DLT) was defined as regimen-related death, graft failure, grade 3 or 4 atrial fibrillation, grade 4 deep venous thrombosis, or pulmonary embolism before day 30 after auto-HCT. (NCT01079936)
Timeframe: Day 30 following transplant

Interventionparticipants (Number)
25 Mg Lenalidomide0
50 mg Lenalidomide0
75 mg Lenalidomide2
100 mg Lenalidomide0

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Number of Participants Surviving at 5 Years

(NCT01083316)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
Bortezomib and Dexamethasone29

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Number of Participants With Disease Response

Complete response: Normal serum free light chain ratio and Negative serum and urine immunofixation electrophoresis Very good partial response: Difference in serum free light chains less than 40 mg/L Partial Response: >50% Reduction in the difference in serum free light chains (NCT01083316)
Timeframe: One year

InterventionParticipants (Count of Participants)
Bortezomib and Dexamethasone20

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Number of Participants Proceeding to Transplant Following Induction

(NCT01083316)
Timeframe: 2 months

InterventionParticipants (Count of Participants)
Bortezomib and Dexamethasone30

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Number of Participants Surviving at 100 Days Post Transplant

(NCT01083316)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Bortezomib and Dexamethasone27

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: at 6 months post transplant

,,,
Interventionpercentage of participants (Number)
SurvivalDisease Free Survival
Busulfan, Melphalan and Fludarabine92.489.0
Clofarabine, Melphalan and Thiotepa87.285.1
Melphalan, Fludarabine and Thiotepa90.990.9
Total Body Irradiation, Thiotepa and Cyclophosphamide93.382.5

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Number of Participants With Acute and Chronic GVHD Following T-cell Depleted, CD34+ Progenitor Cell Enriched Transplants Fractionated by the CliniMACS System.

Standard BMT-CTN and IBMTR systems clinical criteria as defined by Rowlings, et al will be used to establish and grade acute GvHD. Chronic GvHD will be diagnosed and graded according to the criteria of Sullivan (CIBMTR). (NCT01119066)
Timeframe: 3 years

,,,
Interventionparticipants (Number)
aGVHD II-IVcGVHD, LimitedcGVHD, Extensive
Busulfan, Melphalan and Fludarabine6041
Clofarabine, Melphalan and Thiotepa1011
Melphalan, Fludarabine and Thiotepa201
Total Body Irradiation, Thiotepa and Cyclophosphamide3428

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: 1 year post transplant

,,,
Intervention% of pts at 1 year post transplant (Number)
SurvivalDisease free survival
Busulfan, Melphalan and Fludarabine83.873.7
Clofarabine, Melphalan and Thiotepa85.183.0
Melphalan, Fludarabine and Thiotepa81.872.7
Total Body Irradiation, Thiotepa and Cyclophosphamide80.868.3

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Survival and Disease-free Survival (DFS)

(NCT01119066)
Timeframe: 2 years post transplant

,,,
Intervention% of pts at 2 years (Number)
SurvivalDisease Free Survival
Busulfan, Melphalan and Fludarabine72.162.1
Clofarabine, Melphalan and Thiotepa63.859.6
Melphalan, Fludarabine and Thiotepa71.662.3
Total Body Irradiation, Thiotepa and Cyclophosphamide68.758.9

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The Incidence of Durable Hematopoietic Engraftment for T-cell Depleted Transplants Fractionated by the CliniMACS System Administered After Each of the Four Disease Targeted Cytoreduction Regimens.

(NCT01119066)
Timeframe: 3 years

,,,
InterventionParticipants (Count of Participants)
EngraftedPrimary Graft FailureLate graft failureNot Evaluable Engraftment
Busulfan, Melphalan and Fludarabine205050
Clofarabine, Melphalan and Thiotepa45020
Melphalan, Fludarabine and Thiotepa10010
Total Body Irradiation, Thiotepa and Cyclophosphamide118011

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Proportion of Participants With Relapsed Multiple Myeloma With Progression-free (PFS)

(NCT01131169)
Timeframe: 2 years

InterventionProportion of participants PFS (Number)
Relapsed Multiple Myeloma0.31

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Proportion of Participants With Relapsed Multiple Myeloma Alive at 2 Years

(NCT01131169)
Timeframe: 2 years

InterventionProportion of pts alive at 2 years (Number)
Relapsed Multiple Myeloma0.54

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Immunologic Reconstitution

Immunologic Reconstitution will be assessed by quantitative immunoglobulin measurement (IgM, IgG and IgA) (NCT01141712)
Timeframe: Year 1

Interventionmg/dL (Median)
IgGIgAIgM
Autologous Transplant109012348.5

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HIV Single-Copy Polymerase Chain Reaction (PCR)

HIV RNA assay will be performed at the specified time points and summarize the assessments of the viral copy number using descriptive statistics. (NCT01141712)
Timeframe: Baseline, Days 100, 180, 365, and 730

Interventioncopies/mL (Median)
BaselineDay 100Day 180Day 365Day 730
Autologous Transplant802988497130

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Hematologic Function

Hematologic function will be defined as ANC > 1500 neutrophils/μL, Hemoglobin> 10g/dL without transfusion support, and platelets > 100,000/μL (NCT01141712)
Timeframe: Days 100 and 365

Interventionparticipants (Number)
Day 100Day 365
Autologous Transplant1123

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Complete Remission (CR) and/or Partial Response (PR)

The frequencies and proportions of patients who have a CR or PR. CR is defined as the disappearance of all evidence of disease, and PR is defined as the regression of measurable disease and no new sites. (NCT01141712)
Timeframe: Day 100

Interventionparticipants (Number)
CRPRRelapse/Progression
Autologous Transplant3612

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Relapse/Progression

Relapse is defined as the appearance of any new lesion more than 1.5 cm in any axis during or at the end of therapy, even if other lesions are decreasing in size. Progression is defined as a lymph node with a diameter of the short axis of less than 1.0 cm must increase by >= 50% and to a size of 1.5 x 1.5 cm or more than 1.5 cm in the long axis. (NCT01141712)
Timeframe: Year 2

Interventionpercentage of participants (Number)
Autologous Transplant12.5

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Progression-Free Survival (PFS)

The time to this event is the time from enrollment until death, relapse/progression, receipt of anti-lymphoma therapy, or last follow up, whichever comes first. Progression-free survival (PFS) will be estimated using the Kaplan Meier product limit estimator. The PFS probability and confidence interval will be calculated at two years post-transplant. (NCT01141712)
Timeframe: Year 2

Interventionpercentage of participants (Number)
Autologous Transplant79.8

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Number of Participants Experiencing Toxicity

Toxicities will be defined by using the version 3.0 NCI Common Terminology Criteria for Adverse Events (CTCAE) criteria. Only grade 3 and higher toxicities will be collected. (NCT01141712)
Timeframe: Year 2

Interventionparticipants (Number)
Autologous Transplant9

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Number of Participants Experiencing Infections

Microbiologically documented infections will be reported by site of disease, date of onset, severity, and resolution, if any. (NCT01141712)
Timeframe: Year 1

Interventionparticipants (Number)
Autologous Transplant22

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Cumulative Incidence of Platelet Recovery

Platelet recovery is defined as a platelet count greater than 20,000/μL for the first of two consecutive labs with no platelet transfusions 7 days prior. (NCT01141712)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Autologous Transplant92.5

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Cumulative Incidence of Neutrophil Recovery

Neutrophil recovery is defined as two consecutive days of absolute neutrophil count (ANC) > 500 neutrophils/μL following the expected nadir. (NCT01141712)
Timeframe: Day 28

Interventionpercentage of participants (Number)
Autologous Transplant97.5

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Overall Survival (OS)

Assess the OS after autologous hematopoietic stem cell transplantation (HCT) for chemotherapy-sensitive aggressive B cell lymphoma or Hodgkin's lymphoma in patients with HIV using BEAM for pre-transplant conditioning. The primary analysis will consist of estimating the 1 year OS probability from the time of transplantation based on the Kaplan-Meier product limit estimator. The 1 year and 2 year OS and confidence interval will be calculated. (NCT01141712)
Timeframe: Year 1 and 2

Interventionpercentage of participants (Number)
1 year2 years
Autologous Transplant87.382

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Phase I: Number of Patients With Dose Limiting Toxicity

The number of patients who had a DLT during the dose finding portion (Phase I) of the trial for the safety of lenalidomide when used in combination with high dose melphalan in the setting of autologous stem cell transplantation in patients with multiple myeloma. (NCT01142232)
Timeframe: up to 1 month

InterventionParticipants (Count of Participants)
Phase I, Dose Level 10
Phase I, Dose Level 20
Phase I, Dose Level 30
Phase I, Dose Level 40

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Phase II: Overall Response Rate

Evaluate the overall response rate of patients receiving therapy. Patients are considered as having a response if their overall response is Partial Response or better (CR+sCR+VGPR+PR). The percentage of patients achieving this and the exact 95% confidence interval will be calculated. Responses will be defined using the response criteria determined by the International Working Group for Multiple Myeloma (CR= Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and 5% plasma cells in bone marrow; sCR=CR as defined above plus normal FLC ratio and absence of clonal cells in bone marrow by immunohistochemistry or immunoflurorescence; VGPR=Serum and urine M-component detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-component plus urine M-component<100 mg per 24 h; PR=>=50% reduction of serum M-protein and reduction in 24-h urinary M-protein by >=90% or to <200mg per 24 h). (NCT01142232)
Timeframe: up to 5 years

Interventionpercentage of participants (Number)
Phase II87.5

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Percentage of MIBG Avid Patients Treated With Meta-iodobenxylguanide (MIBG) Labeled With Iodine-131

Number of MIBG avid patients who receive 131I-MIBG divided by the number of patients evaluable for the feasibility of MIBG endpoint x 100%. (NCT01175356)
Timeframe: Up to 6 weeks after course 5 of induction

InterventionPercentage of participants (Number)
Treatment (131I-MIBG, Chemotherapy)86.8

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Percentage of MIBG Avid Patients Treated With MIBG Labeled With Iodine-131 and Bu/Mel Chemotherapy

Number of MIBG avid patients who receive 131I-MIBG and Bu/Mel divided by the number of patients evaluable for the feasibility of MIBG and Bu/Mel consolidation endpoint x 100%. (NCT01175356)
Timeframe: Up to day -6 of conditioning

InterventionPercentage of participants (Number)
Treatment (131I-MIBG, Chemotherapy)82.2

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Incidence of Unacceptable Toxicity and Sinusoidal Obstruction Syndrome (SOS), Assessed by Common Terminology Criteria (CTV)v.4.0 for Toxicity Assessment and Grading for I-MIBG

Number of patients who had an unacceptable toxicity or experienced SOS. Unacceptable toxicity was defined as CTC Grade 4-5 Pulmonary/Respiratory. (NCT01175356)
Timeframe: Up to 6 weeks after course 5 of induction

InterventionParticipants (Count of Participants)
Treatment (131I-MIBG, Chemotherapy)3

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Overall Survival (OS) of These Patients.

The overall survival is the length of time from the start of treatment (Auto SCT) for the cancer, that patients are diagnosed with are still alive until date of first documented progression or date of death from any cause. It is measured in months and assessed up to 84 months. (NCT01200329)
Timeframe: Beyond 100 days post transplant up to 84 months.

InterventionMonths (Median)
Overall Study Group52

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Event-free Survival (EFS) of Patients

The event-free survival (EFS) of patients with poor prognosis relapse or refractory Hodgkin's disease (HD) after high-dose chemotherapy (HDC) with Gemcitabine/Busulfan/Melphalan (GemBuMel). Event is defined as relapse, tumor progression or death.Progression free survival is the length of time during and after the treatment of disease that a patient lives with the disease but it does not get worse. Toxicity is defined as the treatment related mortality (TRM) rate, which will be evaluated within 30 days post transplant, and this rate will be compared with the 5% maximum rate. For EFS analysis, patients who experience the tumor relapse, disease progression, or death will be considered to be an event. (NCT01200329)
Timeframe: Enrollment up to 2 years post transplant

InterventionParticipants (Count of Participants)
Overall Study Group51

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Overall Survival

Number of participants from ASCT to death or last contact (NCT01237951)
Timeframe: From date of transplant until the date of death from any cause, assessed up to 2 years

InterventionParticipants (Count of Participants)
Gemcitabine/Busulfan/Melphalan49

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Participants Who Had Measurable Disease at Time of Transplant and Achieved a Stringent Complete Remission

Stringent complete remission was defined as negative immunofixation on the serum and urine, disappearance of any soft tissue plasmacytomas, 5% or fewer plasma cells in bone marrow, normal free light chain ratio, and the absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence. (NCT01237951)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Gemcitabine/Busulfan/Melphalan16

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Progression-free Survival (PFS)

Number of participants remain free of progression or death after ASCT (NCT01237951)
Timeframe: From date of transplant until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 2 years.

InterventionParticipants (Count of Participants)
Gemcitabine/Busulfan/Melphalan31

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Treatment Free Interval/PFS

The treatment-free interval after treatment with the combination of thalidomide, bortezomib and melphalan (NCT01242267)
Timeframe: PFS was defined as the time from ASCT to disease progression or death from any cause, an average of 9 months

Interventionmonths (Median)
Phase 1/Phase 29.3

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Toxicity Assessment

Assessment of the toxicities resulting from administration of combinations of thalidomide, bortezomib and melphalan (NCT01242267)
Timeframe: Patients will be hospitalized or in the outpatient transplant facility until engraftment and resolution of serious adverse events, a median of 16 (range, 11-24) days

InterventionSerious Adverse Events (Number)
Phase I - Thalidomide Dose Level 600 mg1
Phase I - Thalidomide Dose Level 800 mg0
Phase I - Thalidomide Dose Level 1000 mg0
Phase 23

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Maximum Tolerated Dose of Thalidomide

Maximum tolerated dose of thalidomide used in conjunction with dose-intense melphalan, bortezomib and autologous (syngeneic) HSC support in the salvage therapy of patients who failed a prior treatment with dose-intense melphalan (NCT01242267)
Timeframe: Dose escalation will be based on the assessment of tolerability determined after the last patient of each cohort reaches day +21.

Interventionmg (Number)
Phase 11000
Phase 21000

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Complete Response (CR) and Very Good Partial Response (VgPR) Rate

The complete response (CR) and very good partial response (VgPR) rate in patients undergoing ASCT using thalidomide, bortezomib and melphalan (NCT01242267)
Timeframe: Assessments will be made from Day +28 after transplantation for 3 months and then at 3 month intervals until demonstration of disease progression and initiation of further therapy, an average of 9 months

InterventionParticipants (Count of Participants)
Phase I - Thalidomide Dose Level 600 mg0
Phase I - Thalidomide Dose Level 800 mg3
Phase I - Thalidomide Dose Level 1000 mg1
Phase 213

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Feasibility of Maintenance Therapy in This Patient Population (Restricted to Those Patients Achieving a CR). Feasibility Will be Defined as the Number of Deaths Ocuring.

Proportions will be estimated based on the combined and individual cohorts. (NCT01256398)
Timeframe: 10 years

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, Transplant)5

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Disease Free Survival Defined From the Date of First Induction Complete Response (CR) to Relapse or Death Due to Any Cause

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: At 3 years after CR

Interventionpercentage of patients (Number)
Treatment (Chemotherapy, Transplant)52.6

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Disease Free Survival (DFS)

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: 10 years

InterventionMonths (Median)
Treatment (Chemotherapy, Transplant)29.7

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Probability of Being BCR-ABL Negative in the Bone Marrow and Peripheral Blood at the Completion of the CNS Prophylaxis Course (Restricted to Those Patients Achieving a CR)

Proportions will be estimated based on the combined and individual cohorts. (NCT01256398)
Timeframe: 10 years

Interventionproportion of participants (Number)
Treatment (Chemotherapy, Transplant)0.667

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Overall Survival (OS)

Estimated using the Kaplan-Meier estimator. Proportions will be estimated using point as well as interval estimators. All interval estimators will be constructed using the finite sample size sampling distribution at the unadjusted two-sided level of 0.05. (NCT01256398)
Timeframe: 10 years

InterventionMonths (Median)
Treatment (Chemotherapy, Transplant)55.9

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Response

Proportion of patients reaching a CR. A CR requires the following: an absolute neutrophil count (segs and bands) >1000/μL, no circulating blasts, platelets >100,000/μL; adequate bone marrow cellularity with trilineage hematopoiesis, and <5% marrow leukemia blast cells. All previous extramedullary manifestations of disease must be absent (e.g., lymphadenopathy, splenomegaly, skin or gum infiltration, testicular masses, or CNS involvement). (NCT01256398)
Timeframe: 10 years

Interventionproportion of participants (Number)
Treatment (Chemotherapy, Transplant).9846

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Number of Patients Who Complete Therapy Without the Need for Additional Treatment Including Systemic Chemotherapy, External Beam Radiation, or Enucleation.

The primary objective of this study is to show that intra-arterial delivery of the chemotherapeutic agent is successful in treating intraocular retinoblastoma, defined as avoiding systemic chemotherapy, external beam radiation, and enucleation. (NCT01293539)
Timeframe: Within the first six months after the initial treatment.

InterventionParticipants (Count of Participants)
Intra-arterial Chemotherapy10

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Progression Free Survival at One Year.

Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions (NCT01323517)
Timeframe: 1 year

Interventionpercentage of participants PFS at 1 year (Number)
Ipilimumab, Melphalan and Dactinomycin58

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To Define the Immunologic Events and Signatures at the Tumor Site and in the Periphery That Corresponds to Response to Ipilimumab.

Summaries of antibody response, comparison of pretreatment with post-ipilimumab and end of treatment will be assessed for percent of CD4, CD8, and CD68 positive cells (NCT01323517)
Timeframe: 2 years

Interventionpercent of positive cells (Mean)
Pretreatment CD4Posttreatment CD4Pretreatment CD8Posttreatment CD8Pretreatment CD68Posttreatment CD68
Ipilimumab, Melphalan and Dactinomycin328271018

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Toxicity of Additional Ipilimumab Will be Evaluated for All Treated Patients Using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 3.0

(NCT01323517)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Ipilimumab, Melphalan and Dactinomycin26

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AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionhr*ng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 29
Arm C: Ixazomib 3.0 mg662.8331527.800
Arm C: Ixazomib 4.0 mg1037.5002680.000
Arm D: Ixazomib 4.0 mg934.8002435.000

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AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionhr*ng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg450.000705.667
Arm B: Ixazomib 4.0 mg806.8241610.500
Arm B: Ixazomib 5.5 mg1612.2501680.000

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Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)

Terminal elimination rate constant, calculated as the negative of the slope of the log-linear regression of the natural logarithm concentration-time curve during the terminal phase. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Intervention1/hour (Mean)
Cycle 1, Day 29
Arm C: Ixazomib 4.0 mg0.005
Arm D: Ixazomib 4.0 mg0.006

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AUCtau: Area Under the Plasma Concentration-time Curve Over the Dosing Interval for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Interventionhr*ng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 11
Arm A: Ixazomib 3.0 mg319.7141227.143
Arm A: Ixazomib 3.7 mg287.0001180.000

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Time to Progression (TTP) (Phase 2)

TTP is defined as time from date of enrollment to date of first documented disease progression (PD). Per IMWG criteria, progressive disease requires any 1 or more of following: Increase of ≥25% from nadir in serum M-component and/or (absolute increase must be ≥0.5 g/dL), urine M-component and/or (absolute increase must be ≥200 mg/24 hour. Participants without measurable serum+urine M-protein levels: difference between involved and uninvolved FLC levels. The absolute increase must be >10 mg/dL. Bone marrow plasma cell percentage: absolute % must be ≥10%. Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in size of existing bone lesions or soft tissue plasmacytomas. Development of hypercalcemia (corrected serum calcium >11.5 mg/dL or 2.85 mmol/L) that can be attributed solely to plasma cell proliferative disorder. (NCT01335685)
Timeframe: From the date of enrollment to the date of the first documented disease progression for up to 5.5 years

Interventionmonths (Median)
Arm B: Ixazomib 4.0 mg (RP2D)22.1

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Time to First Response (Phase 2)

Response is defined as CR, VGPR and PR. Per IMWG criteria, CR:1)Negative immunofixation on serum+urine, 2)Disappearance of any soft tissue plasmacytomas, 3)< 5% plasma cells in bone marrow. VGPR: Serum+urine M-protein detectable by immunofixation but not on electrophoresis/ 90% or >reduction in serum M-protein + urine M-protein level < 100 mg/ 24-hour. PR:1)≥50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by ≥90% or to <200 mg/24-hour. If serum+urine M-protein are unmeasurable, ≥50% decrease in difference between involved and uninvolved FLC levels is required. Else, ≥50% reduction in plasma cells is required in place of M-protein, provided baseline bone marrow plasma cell percentage was ≥30%. In addition, if present at baseline, a ≥50% reduction in size of soft tissue plasmacytomas is required. (NCT01335685)
Timeframe: From the date of enrollment to the date of the first documented response for up to 5.5 years

Interventionmonths (Median)
Arm B: Ixazomib 4.0 mg (RP2D)1.9

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Progression Free Survival (Phase 2)

Progression Free Survival is defined as time in months from start of study treatment to first documentation of objective tumor progression per investigator assessment or up to death due to any cause, whichever occurs first. Per IMWG criteria, progressive disease requires any 1 or more of following: Increase of ≥25% from nadir in serum M-component and/or (absolute increase must be ≥0.5 g/dL), urine M-component and/or (absolute increase must be ≥200 mg/24 hour. Participants without measurable serum+urine M-protein levels: difference between involved and uninvolved FLC levels. The absolute increase must be >10 mg/dL. Bone marrow plasma cell percentage: absolute % must be ≥10%. Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in size of existing bone lesions or soft tissue plasmacytomas. Development of hypercalcemia (corrected serum calcium >11.5 mg/dL or 2.85 mmol/L) that can be attributed solely to plasma cell proliferative disorder. (NCT01335685)
Timeframe: From the date of enrollment to the date of the first documented disease progression or death due to any cause for up to 5.5 years

Interventionmonths (Median)
Arm B: Ixazomib 4.0 mg (RP2D)18.4

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Overall Survival (Phase 2)

Overall Survival is the time in months from start of study treatment to date of death due to any cause. (NCT01335685)
Timeframe: From date of enrollment to date of death, approximately 5.5 years (Approximate median follow-up: 43.6 months)

Interventionmonths (Median)
Arm B: Ixazomib 4.0 mg (RP2D)NA

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Overall Response Rate (ORR)

ORR is defined as percentage of participants with overall response including CR, VGPR, and partial response (PR). Per IMWG criteria, CR:1)Negative immunofixation on serum and urine, 2)Disappearance of any soft tissue plasmacytomas, 3)< 5% plasma cells in bone marrow. VGPR: Serum+urine M-protein detectable by immunofixation but not on electrophoresis/ 90% or >reduction in serum M-protein + urine M-protein level < 100 mg/ 24-hour. PR:1)≥50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by ≥90% or to <200 mg/24-hour. If serum+urine M-protein are unmeasurable, ≥50% decrease in difference between involved and uninvolved FLC levels is required. If serum+urine M-protein are unmeasurable and serum free light assay is also unmeasurable, ≥50% reduction in plasma cells is required in place of M-protein, provided baseline bone marrow plasma cell percentage was ≥30%. In addition, if present at baseline, a ≥50% reduction in size of soft tissue plasmacytomas is required. (NCT01335685)
Timeframe: Day 1 of every other cycle from Day 1 of Cycle 2 (each cycle of 28 days) up to 61 cycles, at end of treatment (Up to 5.5 years)

Interventionpercentage of participants (Number)
Arm A: Ixazomib 3.0 mg86
Arm A: Ixazomib 3.7 mg67
Arm B: Ixazomib 3.0 mg100
Arm B: Ixazomib 4.0 mg65
Arm B: Ixazomib 5.5 mg60
Arm C: Ixazomib 3.0 mg40
Arm C: Ixazomib 4.0 mg67
Arm D: Ixazomib 4.0 mg50

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Duration of Response (DOR) (Phase 2)

DOR is defined as time of first documentation of a confirmed PR or better response to first documented PD or start of alternative therapy. DOR was presented for those achieving CR+VGPR+PR. Per IMWG criteria, CR:1)Negative immunofixation on serum+urine, 2)Disappearance of any soft tissue plasmacytomas, 3)< 5% plasma cells in bone marrow. VGPR: Serum+urine M-protein detectable by immunofixation but not on electrophoresis/ 90% or >reduction in serum M-protein + urine M-protein level < 100 mg/ 24-hour. PR:1)≥50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by ≥90% or to <200 mg/24-hour. If serum+urine M-protein are unmeasurable, ≥50% decrease in difference between involved and uninvolved FLC levels is required. Else, ≥50% reduction in plasma cells is required in place of M-protein, provided baseline bone marrow plasma cell percentage was ≥30%. In addition, if present at baseline, a ≥50% reduction in size of soft tissue plasmacytomas is required. (NCT01335685)
Timeframe: From the time from the date of first documentation of PR or better to the date of first documented disease progression for up to 5.5 years

Interventionmonths (Median)
Arm B: Ixazomib 4.0 mg (RP2D)25.2

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Very Good Partial Response (VGPR) or Better Response Rate (Phase 2)

VGPR or better response rate is defined as percentage of participants with a complete response (CR) and very good partial response (VGPR). Per International Myeloma Working Group Uniform Response Criteria (IMWG), CR: 1) Negative immunofixation on the serum and urine, 2) Disappearance of any soft tissue plasmacytomas and 3) < 5% plasma cells in bone marrow. VGPR: Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein plus urine M-protein level < 100 mg per 24 hour. (NCT01335685)
Timeframe: Day 1 of every other cycle from Day 1 of Cycle 2 (each cycle of 28 days) until death (Up to 5.5 years)

Interventionpercentage of participants (Number)
Arm B: Ixazomib 4.0 mg (RP2D)48

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Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)

Terminal phase elimination half-life (T1/2) is the time required for half of the drug to be eliminated from the plasma. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionhours (Mean)
Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg167.000
Arm B: Ixazomib 4.0 mg130.362
Arm B: Ixazomib 5.5 mg98.900

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Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)

Terminal phase elimination half-life (T1/2) is the time required for half of the drug to be eliminated from the plasma. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Interventionhours (Mean)
Cycle 1, Day 29
Arm C: Ixazomib 4.0 mg163.500
Arm D: Ixazomib 4.0 mg120.050

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Terminal Phase Elimination Half-life (T1/2) for Ixazomib (Phase 1)

Terminal phase elimination half-life (T1/2) is the time required for half of the drug to be eliminated from the plasma. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

Interventionhours (Mean)
Cycle 1, Day 15Cycle 1, Day 29
Arm C: Ixazomib 3.0 mgNA140.575

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Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionhours (Median)
Cycle 1, Day 1Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg1.7500.833
Arm B: Ixazomib 4.0 mg1.0001.000
Arm B: Ixazomib 5.5 mg1.3020.500

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Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionhours (Median)
Cycle 1, Day 1Cycle 1, Day 29
Arm C: Ixazomib 3.0 mg1.5601.500
Arm C: Ixazomib 4.0 mg1.2821.275
Arm D: Ixazomib 4.0 mg0.5670.760

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Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)

Terminal elimination rate constant, calculated as the negative of the slope of the log-linear regression of the natural logarithm concentration-time curve during the terminal phase. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

Intervention1/hour (Mean)
Cycle 1, Day 15Cycle 1, Day 29
Arm C: Ixazomib 3.0 mgNA0.005

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Terminal Elimination Rate Constant (λz) for Ixazomib (Phase 1)

Terminal elimination rate constant, calculated as the negative of the slope of the log-linear regression of the natural logarithm concentration-time curve during the terminal phase. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Intervention1/hour (Mean)
Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg0.004
Arm B: Ixazomib 4.0 mg0.006
Arm B: Ixazomib 5.5 mg0.007

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Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)

Accumulation ratio for AUCtau (Rac) was calculated as area under the curve from time zero to end of dosing interval (AUCtau) on Day 14 divided by area under the curve from time zero to end of dosing interval (AUCtau) on Day 1. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionratio (Mean)
Cycle 1, Day 29
Arm C: Ixazomib 3.0 mg2.632
Arm C: Ixazomib 4.0 mg2.560
Arm D: Ixazomib 4.0 mg2.540

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Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)

Accumulation ratio for AUCtau (Rac) was calculated as area under the curve from time zero to end of dosing interval (AUCtau) on Day 14 divided by area under the curve from time zero to end of dosing interval (AUCtau) on Day 1. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionratio (Mean)
Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg1.700
Arm B: Ixazomib 4.0 mg2.288
Arm B: Ixazomib 5.5 mg1.970

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Observed Accumulation Ratio for AUCtau (Rac) (Phase 1)

Accumulation ratio for AUCtau (Rac) was calculated as area under the curve from time zero to end of dosing interval (AUCtau) on Day 14 divided by area under the curve from time zero to end of dosing interval (AUCtau) on Day 1. (NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Interventionratio (Mean)
Cycle 1, Day 11
Arm A: Ixazomib 3.0 mg4.019
Arm A: Ixazomib 3.7 mg4.120

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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. (NCT01335685)
Timeframe: From first dose of study drug through 30 days after last dose of study drug or until the start of subsequent antineoplastic therapy for up to 5.6 years

,,,,,,,
Interventionparticipants (Number)
During Entire Study Any Adverse EventGrade 3 or Higher Adverse EventSerious Adverse EventAdverse Event With Any Study Drug DiscontinuationAdverse Event With Any Study Drug Reduction
Arm A: Ixazomib 3.0 mg77204
Arm A: Ixazomib 3.7 mg44402
Arm B: Ixazomib 3.0 mg33301
Arm B: Ixazomib 4.0 mg262112813
Arm B: Ixazomib 5.5 mg55323
Arm C: Ixazomib 3.0 mg65423
Arm C: Ixazomib 4.0 mg44212
Arm D: Ixazomib 4.0 mg65124

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Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 29
Arm C: Ixazomib 3.0 mg55.36759.560
Arm C: Ixazomib 4.0 mg50.875109.000
Arm D: Ixazomib 4.0 mg72.080146.400

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Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,,
Interventionng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 15
Arm B: Ixazomib 3.0 mg22.95030.267
Arm B: Ixazomib 4.0 mg53.27885.636
Arm B: Ixazomib 5.5 mg104.225285.000

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Tmax: Time to Reach the Maximum Plasma Concentration (Cmax) for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Interventionhours (Median)
Cycle 1, Day 1Cycle 1, Day 11
Arm A: Ixazomib 3.0 mg1.0201.050
Arm A: Ixazomib 3.7 mg0.5178.000

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Cmax: Maximum Observed Plasma Concentration for Ixazomib (Phase 1)

(NCT01335685)
Timeframe: Pre-dose on Day 1 and at multiple timepoints (up to 8 hours) on Day 11 for Arm A, Day 15 for Arm B and Day 29 for Arms C and D

,
Interventionng/mL (Mean)
Cycle 1, Day 1Cycle 1, Day 11
Arm A: Ixazomib 3.0 mg26.79169.214
Arm A: Ixazomib 3.7 mg39.30022.000

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Number of Participants With Donor Cell Engraftment

Donor cell engraftment will be assessed by donor-recipient chimerism assays. Full donor chimerism is defined as the presence of at least 95% donor cells as a proportion of the total population in the peripheral blood or bone marrow. Graft rejection is defined as the presence of no more than 5% donor cells as a proportion of the total population. Mixed chimerism is defined as the presence of between 5% and 95% donor cells. Mixed or full donor chimerism will be considered evidence of donor engraftment. (NCT01339910)
Timeframe: Days 28 and 100 and 18 months post-transplant

InterventionParticipants (Count of Participants)
Day 2872548419Day 2872548420Day 10072548420Day 1007254841918 Months7254842018 Months72548419
Mixed ChimerismFull Donor ChimerismGraft RejectionDeath Prior to AssessmentUnknown (relapsed or missing assay)
Myeloablative Conditioning Regimen (MAC)86
Reduced Intensity Conditioning (RIC)80
Myeloablative Conditioning Regimen (MAC)9
Reduced Intensity Conditioning (RIC)30
Myeloablative Conditioning Regimen (MAC)1
Myeloablative Conditioning Regimen (MAC)0
Reduced Intensity Conditioning (RIC)0
Myeloablative Conditioning Regimen (MAC)36
Reduced Intensity Conditioning (RIC)22
Myeloablative Conditioning Regimen (MAC)106
Reduced Intensity Conditioning (RIC)86
Myeloablative Conditioning Regimen (MAC)12
Myeloablative Conditioning Regimen (MAC)2
Myeloablative Conditioning Regimen (MAC)6
Reduced Intensity Conditioning (RIC)8
Myeloablative Conditioning Regimen (MAC)71
Reduced Intensity Conditioning (RIC)66
Myeloablative Conditioning Regimen (MAC)4
Reduced Intensity Conditioning (RIC)5
Reduced Intensity Conditioning (RIC)1
Myeloablative Conditioning Regimen (MAC)31
Reduced Intensity Conditioning (RIC)42
Myeloablative Conditioning Regimen (MAC)25
Reduced Intensity Conditioning (RIC)19

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Percentage of Participants With Neutrophil and Platelet Engraftment

Neutrophil engraftment is defined as achieving an absolute neutrophil count greater than 500x10^6/liter for 3 consecutive measurements on different days. The first of the 3 days will be designated the day of neutrophil engraftment. Platelet engraftment is defined as achieving platelet counts greater than 20,000/microliter for consecutive measurements over 7 days without requiring platelet transfusions. The first of the 7 days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 7 days. (NCT01339910)
Timeframe: Days 28 and 60 post-transplant

,
Interventionpercentage (Number)
Neutrophil Engraftment at Day 28Platelet Engraftment at Day 60
Myeloablative Conditioning Regimen (MAC)98.595.5
Reduced Intensity Conditioning (RIC)97.896.2

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Percentage of Participants With Acute Graft Versus Host Disease (GVHD)

"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL~2-3 mg/dL~3.01-6 mg/dL~6.01-15.0 mg/dL~>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT01339910)
Timeframe: Day 100 post-transplant

,
Interventionpercentage (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHD
Myeloablative Conditioning Regimen (MAC)44.713.6
Reduced Intensity Conditioning (RIC)31.66.8

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Percentage of Participants With Relapse-Free Survival (RFS)

Relapse-free survival is defined as survival without relapse of the primary disease. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)67.8
Reduced Intensity Conditioning (RIC)47.3

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Percentage of Participants With Overall Survival (OS)

Overall survival is defined as survival of death from any cause. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)77.5
Reduced Intensity Conditioning (RIC)67.7

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Percentage of Participants With Chronic GVHD

Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. (NCT01339910)
Timeframe: 18 months post-transplant

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)64.0
Reduced Intensity Conditioning (RIC)47.6

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Number of Participants With Secondary Graft Failure

Secondary graft failure is defined by initial neutrophil engraftment followed by subsequent decline in neutrophil counts to less than 500x10^6/liter that is unresponsive to growth factor therapy. (NCT01339910)
Timeframe: 18 months post-transplant

InterventionParticipants (Count of Participants)
Myeloablative Conditioning Regimen (MAC)1
Reduced Intensity Conditioning (RIC)4

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Number of Participants With Primary Graft Failure

Primary graft failure is defined by lack of neutrophil engraftment. (NCT01339910)
Timeframe: 28 days post-transplant

InterventionParticipants (Count of Participants)
Myeloablative Conditioning Regimen (MAC)1
Reduced Intensity Conditioning (RIC)3

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Percentage of Participants With Disease Relapse

Disease Relapse is defined as relapse of the primary disease. (NCT01339910)
Timeframe: 18 months post-randomization

Interventionpercentage (Number)
Myeloablative Conditioning Regimen (MAC)13.5
Reduced Intensity Conditioning (RIC)48.3

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1 Year Relapse Rate

The percentage of participants that relapsed within 12 months. Relapse is defined by either morphological or cytogenetic evidence of the original malignancy consistent with pre-transplant features. (NCT01408563)
Timeframe: 1 year

Interventionpercentage (Number)
Fludarabine/Melphalan/TBI20

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Immune Reconstitution - Median CD4 Count at 12 Months

(NCT01408563)
Timeframe: 1 Year

Interventioncells/mm3 (Median)
Fludarabine/Melphalan/TBI362.4

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Median Thrombopoietin Levels After Transplant

(NCT01408563)
Timeframe: 30 Days

Interventionpg/mL (Median)
Fludarabine/Melphalan/TBI2500

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Median Time to Neutrophil Engraftment

The median number of days measured from the time of transplantation, until the first documented neutrophil engraftment. neutrophil engraftment is defined as the first of 3 consecutive days of absolute neutrophil count > 500 neutrophils per microliter of blood. (NCT01408563)
Timeframe: From the time of transplantation, until the time of neutrophil engraftment, median duration of 24 days

InterventionDays (Median)
Fludarabine/Melphalan/TBI24

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Median Time to Platelet Engraftment

The time to platelet engraftment is measured from the time of transplantation until the time of first documented platelet engraftment. Platelet engraftment is defined as a platelet count ≥ 20,000/µL for three consecutive measurements over three or more days. The first of the three days will be designated the day of platelet engraftment. Subjects must not have had platelet transfusions during the preceding 3 days or in the following 7 days after the day of engraftment, unless the platelet transfusion is being given specifically to achieve a platelet threshold to allow an elective invasive procedure, such as a central catheter removal. (NCT01408563)
Timeframe: From the time of transplantation, until the time of platelet engraftment, median duration of 52 days

InterventionDays (Median)
Fludarabine/Melphalan/TBI52

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Number of Participants With Primary Graft Failure

Primary graft failure is defined as the failure to achieve an absolute neutrophil count (ANC) >500/ µL by day 42, in the absence of relapse. (NCT01408563)
Timeframe: From the time of transplantation until 42 days post transplantation

InterventionParticipants (Count of Participants)
Fludarabine/Melphalan/TBI8

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Rate of Post-transplant Lymphoma

The number of participants that were found to have lymphoma post-transplant. (NCT01408563)
Timeframe: 2.5 years

InterventionParticipants (Count of Participants)
Fludarabine/Melphalan/TBI0

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Rates of Grade II-IV and Grade III-IV Acute Graft Versus Host Disease (GVHD) at 100 Days

"Acute GVHD is assessed using Consensus Criteria:~Organ Classifications:~0: No rash due to GVHD; Bilirubin < 2 mg/dL; < 500 mL diarrhea/ day~1: Maculopapular rash < 25% of body surface; Bilirubin 2-3 mg/dL; 500 to 999 mL diarrhea/ day or persistent nausea with histologic evidence of GVHD in stomach/ duodenum~2: Maculopapular rash 25-50% of body surface; Bilirubin 3.1-6 mg/dL; 1,000 to 1,499 mL diarrhea/ day~3: Maculopapular rash > 50% of body surface; Bilirubin 6.1-15 mg/dL; 1,500 or more mL diarrhea/ day~4: Generalized erythroderma with bullous formation; Bilirubin > 15 mg/dL; Severe abdominal pain with or without ileus~Overall Clinical Grade:~0: No Stage 1-4 of any organ~I: Stage 1-2 rash and no liver or gut involvement~II: Stage 3 rash, or Stage 1 liver involvement, or Stage 1 gut involvement~III: None to Stage 3 skin rash with Stage 2-3 liver involvement, or Stage 2-4 gut involvement~IV: Stage 4 skin rash, or Stage 4 liver involvement" (NCT01408563)
Timeframe: 100 Days

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI16

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Relapse-free Survival

The percentage of participants that have not died or had disease progression by two years. Relapse is defined by either morphological or cytogenetic evidence of the original malignancy consistent with pre-transplant features. (NCT01408563)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI49

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Overall Survival

The percentage of participants alive at two years (NCT01408563)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI55

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The Rate of Chronic GVHD

Chronic Graft Versus Host Disease (GVHD) is assessed using the National Institutes of Health (NIH) consensus criteria. (NCT01408563)
Timeframe: From the time of transplantation until the time of chronic GVHD onset, up to 1 year

Interventionpercentage of participants (Number)
Fludarabine/Melphalan/TBI21

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Chimerism

Donor T-cell and myeloid chimerism will be described separately by conditioning regimen intensity (myeloablative or reduced intensity) according to proportions with mixed chimerism (5-95% donor cells out of all), full chimerism (>95% donor cells), or graft rejection (<5% donor cells). (NCT01410344)
Timeframe: Week 4, Day 100, and 6 months Post-transplant

InterventionParticipants (Count of Participants)
Week 472365409Week 472365410Day 10072365410Day 100723654096 Months723654096 Months72365410
Graft RejectionNo Assay ReportedFull ChimerismMixed ChimerismDead at Assessment
Reduced Intensity Allogeneic Transplant4
Myeloablative Allogeneic Transplant1
Myeloablative Allogeneic Transplant3
Myeloablative Allogeneic Transplant4
Reduced Intensity Allogeneic Transplant5
Myeloablative Allogeneic Transplant2
Reduced Intensity Allogeneic Transplant2
Myeloablative Allogeneic Transplant0
Reduced Intensity Allogeneic Transplant0

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Percentage of Participants With Overall Survival

Overall survival is defined as the time from transplant to death from any cause. (NCT01410344)
Timeframe: Six months, 1 Year, and 2 Years Post-transplant

Interventionpercentage of participants (Number)
6 Months1 Year2 Years
Allogeneic Transplant82.458.850.2

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Percentage of Participants With Non-Relapse Mortality

The events for non-relapse mortality are death due to any cause other than relapse of the underlying malignancy. (NCT01410344)
Timeframe: Day 100, 1 Year, and 2 Years Post-transplant

Interventionpercentage of participants (Number)
Day 1001 Year2 Years
Allogeneic Transplant0.011.818.3

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Percentage of Participants Recovering Hematologic Function

Recovery of hematologic function is described by the time to neutrophil and platelet recovery. Time to neutrophil recovery will be the first of three consecutive days of > 500 neutrophils/μL following the expected nadir. Time to platelet engraftment will be described by the date when platelet count is > 20,000/μL for the first of three consecutive labs with no platelet transfusions 7 days prior. (NCT01410344)
Timeframe: Days 28 and 100 Post-transplant

Interventionpercentage of participants (Number)
Day 28 Neutrophil RecoveryDay 100 Platelet Recovery
Allogeneic Transplant100.094.1

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Percentage of Participants With Relapse/Progression

Relapse/Progression is defined as relapse or progression of the primary malignancy. (NCT01410344)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Allogeneic Transplant29.4

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Percentage of Participants With Chronic Graft-Versus-Host Disease (GVHD)

Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. (NCT01410344)
Timeframe: 1 Year Post-transplant

Interventionpercentage of participants (Number)
Allogeneic Transplant17.6

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Percentage of Participants With Acute Graft-Versus-Host Disease (GVHD)

"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL 1.2-3 mg/dL 2.3.01-6 mg/dL 3.6.01-15.0 mg/dL 4.>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT01410344)
Timeframe: Day 100 Post-transplant

Interventionpercentage of participants (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHD
Allogeneic Transplant41.211.8

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Number of Participants That Had Grade 3-4 Toxicities.

(NCT01413178)
Timeframe: At day 90 post SCT (Stem Cell Transplantation)

,
Interventionparticipants (Number)
Grade 3 - DiarrheaGrade 3 - Elevated ALTGrade 3 - Elevated TbiliGrade 3 - InfectionGrade 3 - MucositisGrade 3 - NauseaGrade 3 - NeuropathyGrade 3 - Neutropenic feverGrade 3 - PneumoniaGrade 3 - TachycardiaGrade 3 - Transient blindnessGrade 3 - AnorexiaGrade 3 - Myocardial ischemiaGrade 3 - Pleural effusionGrade 3 - Rash
Busulfan + Melphalan532161551731111100
Melphalan4005020321000111

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Number of Participants With Complete Response (CR)

Complete response (CR), evaluated 90 days from transplant, defined as (i) negative immunofixation of the multiple myeloma (MM) protein in urine and serum, (ii) disappearance of any soft tissue plasmacytomas, and (iii) less than 5% plasma MM cells in the bone marrow. International Myeloma Working Group uniform response criteria. (NCT01413178)
Timeframe: Evaluated 90 days from transplant.

InterventionParticipants (Count of Participants)
Busulfan + Melphalan12
Melphalan15

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Progression-Free Survival (PFS)

Participants that are still alive and without Multiple Myeloma 3 years after Stem cell Transplantation. (NCT01413178)
Timeframe: 3 years after transplant

InterventionParticipants (Count of Participants)
Busulfan + Melphalan72
Melphalan50

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Overall Survival (OS)

(NCT01413178)
Timeframe: From time of ASCT to 3 years

InterventionParticipants (Count of Participants)
Busulfan + Melphalan91
Melphalan79

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Progression Free Survival Rate

Progression free survival of elderly patients with multiple myeloma treated with either high-dose melphalan versus high-dose melphalan and bortezomib at 3 years (NCT01453088)
Timeframe: Participants will be followed post transplant for a minimum of 3 years, and after that may be monitored as part of the study indefinitely

InterventionParticipants (Count of Participants)
Auto Transplant High Dose Melphalan13
Auto Transplant High Dose Melphalan+Bortezomib11

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Overall Survival Rate

(NCT01453088)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Auto Transplant High Dose Melphalan27
Auto Transplant High Dose Melphalan+Bortezomib27

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Progression Free Survival

The main primary endpoint of this study is two-year progression free survival. Patients are considered a failure with respect to PFS if they die or experience disease progression or relapse. The time to this event is the time from transplantation to relapse/progression, initiation of non-protocol anti-myeloma therapy, or death from any cause. Subjects alive without confirmed disease progression will be censored at the time of last disease evaluation. Deaths without progression are treated as failures no matter when they occur. (NCT01453101)
Timeframe: Subjects will be followed for progression-free survival for at least 36 months

InterventionMonths (Median)
Fludarabine, Melphalan, Bortezomib16.7

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Overall Survival (OS)

Overall survival (OS): Defined as time from the first dose of administration to death from any cause (NCT01453101)
Timeframe: Up to 3 years

Interventionpercentage (Number)
Fludarabine, Melphalan, Bortezomib42

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Overall Response Rate

"Overall response rate: Defined as the composite endpoint of response to treatment which includes Complete Response (CR), Partial Response (PR), stable disease (SD) as defined in International Response Criteria.~International Myeloma Working Group Response Criteria for Multiple Myeloma:~CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and < 5% plasma cells in bone marrow PR: > 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by >90% or to < 200 mg/24 h SD: Not meeting criteria for CR, VGPR, PR, or progressive disease" (NCT01453101)
Timeframe: Up to 3 years

InterventionParticipants (Count of Participants)
Fludarabine, Melphalan, Bortezomib45

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Number of Participants With Moderate to Severe (Grade 2-4) Acute Graft Versus Host Disease (GVHD).

Acute GVHD grade 2-4 was assessed in patients in the FluBU and FluMel groups up to 100 days after transplant. (NCT01499147)
Timeframe: Up to 100 days post-transplant (acute GVHD).

Interventionparticipants (Number)
Fludarabine/Busulfan + ATG2
Fludarabine/Melphalan +ATG1

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Number of Participants With Engraftment.

Median time to ANC engraftment and platelet engraftment in both groups as well as the transfusion requirements measured within 30 days after transplant. (NCT01499147)
Timeframe: Up to 30 days post-transplant

Interventionparticipants (Number)
Fludarabine/Busulfan + ATG18
Fludarabine/Melphalan + ATG12

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Time to ANC and Platelet Engraftment

Days to ANC or platelet engraftment (NCT01499147)
Timeframe: Up to 30 days post-transplant

Interventiondays to ANC and platelet engraftment (Median)
Fludarabine/Busulfan + ATG15
Fludarabine/Melphalan + ATG12

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Overall Survival (OS)

Overall Survival is defined as the interval between day of transplant and day of death. (NCT01518153)
Timeframe: Every 3 months until day of death

Interventiondays (Median)
Stem Cell Transplant + Donor Lymphocyte Infusion246

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Success Rate

Success rate defined as alive, engrafted without grade 3 or 4 GvHD or relapse at day 100 post allogeneic stem cell transplantation followed by donor lymphocyte infusion (DLI). (NCT01518153)
Timeframe: 100 days

Interventionparticipants (Number)
Low Dose Donor T-Cells1
High Dose Donor T-Cells3

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Clinical Response

"Patients will be followed according to response criteria as referenced in BMT SOP Standards of Therapy last updated 2008. Clinical Response = CR + PR.~Complete Response Requires all of the following:~Serum and urine negative for monoclonal proteins by immunofixation~Normal free light chain ratio~Plasma cells in marrow < 5%~Partial Response (PR) Requires any of the following:~- ≥ 50% reduction in current serum monoclonal protein levels > 0.5 g/dL or urine light chain levels > 100 mg/day with a visible peak or free light chain levels > 10mg/dL~Progressive Disease (PD) Requires any of the following:~If progressing from CR, any detectable monoclonal protein or abnormal free light chain ratio (light chain must double)~If progressive from PR or SD, ≥ 50% increase in the serum M protein to > 0.5 g/dL,or ≥ 50% increase in urine M protein to > 200mg/day with visible peak present.~Free light chain increase of ≥ 50% to" (NCT01529827)
Timeframe: In the first 100 days from day 0 of transplant

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic PBSCT)45

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Median Time to Neutrophil Engraftment

Median time to recovery of absolute neutrophil count >=500/uL for 3 consecutive days. Summarized using standard descriptive statistics along with corresponding 95% confidence intervals. (NCT01529827)
Timeframe: Day 100

Interventiondays (Median)
Treatment (Reduced Intensity Allogeneic PBSCT)17

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Progression Free Survival (PFS) at One Year

Assessed using Kaplan Meier and Proportional Hazards (NCT01529827)
Timeframe: day of transplant until progression up to 5 years

Interventionpercentage of participants (Number)
Treatment (Reduced Intensity Allogeneic PBSCT)85

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Overall Survival Median

Overall survival reported as number of days participants alive following treatment up to 5 years with annual follow up till disease progression. Evaluations done every 3 months for 1 year and then every 6 months for 5 years to check on the status of the disease, with long-term follow up as needed. (NCT01538472)
Timeframe: Participant followed from baseline treatment to 5 years, with study total period 8 years (study duration)

Interventiondays (Median)
Y Zevalin + BEAM1299

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3-Year Overall Survival

Number of participants alive 3 years following treatment. Evaluations done every 3 months for 1 year and then every 6 months to check on the status of the disease. (NCT01538472)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Y Zevalin + BEAM78

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Event-Free Survival (EFS)

To determine whether, in comparison to Total Therapy II, the median Event-Free Survival (EFS) can be increased from 4.8 years to 7.2 years, which represents an increase in median EFS of approximately 50%, based on an intent-to-treat analysis. (NCT01548573)
Timeframe: 8 years

Intervention ()
Tandem Autologous Stem Cell Transplant0

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Overall Survival

To determine the median overall survival based on an intent-to-treat analysis, which should exceed 10 years, based on the projected 10-year survival of Total Therapy III, keeping in mind that participants are included in this protocol with up to 12 months of prior therapy. (NCT01548573)
Timeframe: 10 years

Intervention ()
Tandem Transplant in MM <12 Mos of Prior Treatment0

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Identification of Drug Resistant Genes

To determine whether repeated bone marrow samples analyzed for gene expression profiling (GEP) can identify genes related to drug resistance in myeloma. The drug resistant genes or the gene products might then be targeted specifically to eradicate myeloma cells surviving tandem transplantation. (NCT01548573)
Timeframe: 5 years

Intervention ()
Tandem Transplant in MM <12 Mos of Prior Treatment0

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Number of Grade 3 Non-hematologic and Grade 4 Hematologic Serious Adverse Events Associated With the Addition of Bortezomib, Thalidomide, and Dexamethasone Into Autologous Transplant Regimens.

To determine whether bortezomib, thalidomide and dexamethasone with transplant 1 and velcade/gemcitabine with transplant 2 can be safely incorporated into well-tested pre-transplant regimens of high-dose melphalan and carmustine/melphalan in doses equivalent to the BEAM(BCNU, etoposide, arabinoside, melphalan)regimen. Treatment-related toxicities will be compared to those reported in the literature using similar intensive approaches. (NCT01548573)
Timeframe: 2 years

Intervention ()
Tandem Transplant in MM <12 Mos of Prior Treatment0

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Overall Response Rate

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. (NCT01605032)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Complete responsePartial response
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)217

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Mortality

(NCT01605032)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)1

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Rate of Complete Response as Determined by the IMWG Criteria

Number of patients achieved complete response after the treatment regimen (NCT01605032)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)2

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Progression-free Survival

The progression free survival was assessed over a period of 2 years (NCT01605032)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)11

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Overall Survival

The overall survival of patients was measured of a period of 2 years. (NCT01605032)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)18

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Donor (Allogeneic) Hematopoietic Engraftment

Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT. (NCT01626092)
Timeframe: Day 100 Following Hematopoietic Cell Transplant (HCT)

Interventionparticipants (Number)
Intent-To-Treat Patients1

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Number of Participants With Overall Survival (OS)

OS is defined as the duration from the time of transplant to death or last follow-up. (NCT01653418)
Timeframe: Median follow-up of 6 months (range: 6-12 months)

Interventionparticipants (Number)
Expired Day +3Expired Day +18Alive
V-BEAM + Stem Cell Infusion118

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Number of Participants With Progression-free Survival (PFS)

"PFS is defined as the duration from transplant to time of first progression, death, relapse after CR, or the date the patient was last known to be in remission.~Response will be assessed per the International Myeloma Working Group (IMWG) Response Criteria." (NCT01653418)
Timeframe: Median follow-up of 6 months (range: 6.0-12.0 months)

Interventionparticipants (Number)
No relapse/progressionRelapse/progression at 12 months
V-BEAM + Stem Cell Infusion71

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Time to Platelet Engraftment After V-BEAM.

Time to platelet engraftment is defined as the duration between Day 0 to the first day of platelet count sustained at > 20x109/L without transfusion. The median time to neutrophil and platelet engraftment will be reported. (NCT01653418)
Timeframe: Day +100

Interventiondays (Median)
More than 20 x 10^9/LMore than 50 x 10^9/L
V-BEAM + Stem Cell Infusion22.523

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Overall Response Rate (ORR)

"ORR includes Partial Response (PR) + Very Good Partial Response (VGPR) + Complete Response (CR)~Response will be assessed per the International Myeloma Working Group (IMWG) Response Criteria." (NCT01653418)
Timeframe: 3 months following Day +100 visit

Interventionparticipants (Number)
Partial responseVery good partial responseComplete response
V-BEAM + Stem Cell Infusion026

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Toxicity of V-BEAM

"Graded per the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.~Patients are evaluated from first receiving study treatment until a 30-day follow-up after the conclusion of treatment for adverse events not resulting in death. Adverse events resulting in death will be evaluated through Day +100.~This outcomes measures the common toxicities observed. Please refer to the Serious Adverse Event and Other Adverse Event sections of the results for further details." (NCT01653418)
Timeframe: 30 days after end of treatment / Day +100

Interventionparticipants (Number)
Neutropenic feverClostridium difficile colitisNeutropenic colitis without Clostridium difficileSepsisMucositis (grade 1-2)Mucositis (grade 3-4)Diarrhea (grade 3-4)Hepatic toxicity (grade 3-4)Peripheral neuropathy (grade 1-2)Toxic death
V-BEAM + Stem Cell Infusion103338210122

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Very Good Partial Response Rate (VGPR+nCR+sCR+CR)

Response will be assessed per the International Myeloma Working Group (IMWG) Response Criteria. (NCT01653418)
Timeframe: Day +100

Interventionparticipants (Number)
V-BEAM + Stem Cell Infusion8

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Complete Response Rate (Complete Response + Stringent Complete Response)

Defined by the International Myeloma Working Group (IMWG) criteria (NCT01653418)
Timeframe: Day +100

Interventionparticipants (Number)
V-BEAM + Stem Cell Infusion6

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Time to Neutrophil Engraftment After V-BEAM.

Time to neutrophil engraftment is defined as duration between Day 0 to the first day of ANC > 0.5x109/L post transplant when it is sustained for more than three consecutive days. (NCT01653418)
Timeframe: Day +100

Interventiondays (Median)
V-BEAM + Stem Cell Infusion10

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Number of Participants With Adverse Events

Here is the number of participants with adverse events. For a detailed list of adverse events, see the adverse event module. (NCT01658904)
Timeframe: 8 months and 15 days

Interventionparticipants (Number)
Cohort 1- CFZ 20 mg/m^2 (Day 1,2)1

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Change From Baseline in Functional Assessment of Cancer Therapy/Gynecologic Oncology Group-Neurotoxicity (FACT/GOG-Ntx) Score at Week 28 of the PFS Follow-up

The FACT/GOG-Ntx is a participant completed questionnaire that comprises 11 individual items evaluating symptoms of neurotoxicity on a 5-point scale where: 0=not at all (best) to 4=very much for a total possible score of 0 to 44. Symptom scores are inverted so that higher scores of FACT/GOG-Ntx indicate higher quality of life or functioning. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: Baseline, Week 28 of the PFS Follow-up

Interventionscore on a scale (Mean)
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide0.0

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Change From Baseline in SF-36 Physical Component Summary Score at Week 28 of the PFS Follow-up

SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100, where higher scores are associated with less disability and better quality of life. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: Baseline, Week 28 of the PFS Follow-up

Interventionscore on a scale (Mean)
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide10.3

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Duration of Hematologic Response

Duration of hematologic response (DOR) was defined as the time from the date of first documentation of a hematologic response to the date of first documented hematologic disease progression as determined by the investigator. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From time of first documented response to disease progression (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + DexamethasoneNA
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide21.19

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EuroQol 5-Dimension 3-Level (EQ-5D-3L) Visual Analogue Scale Score

The EQ visual analogue scale (VAS) records the participant's self-rated health on a 20 centimeter vertical VAS that ranges from 0 (worst imaginable health state) to 100 (best imaginable health state). Baseline is defined as the value collected at the time closest to, but prior to, the start of study drug administration. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: At Week 28 of the OS follow-up

Interventionscore on a scale (Mean)
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide23.0

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Number of Hospitalizations

A hospitalization was defined as at least one overnight stay in an intensive care unit and/or non-intensive care unit. If a single hospitalization included both an intensive care unit stay and a non-intensive care unit stay, the hospitalization was counted only once (as an intensive care unit stay). The mean number of hospitalizations is reported in this outcome measure. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionhospitalizations (Mean)
Arm A: Ixazomib + Dexamethasone1.8
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide1.4

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Number of Participants With Serious Adverse Events (SAEs)

A SAE was defined as any untoward medical occurrence that at any dose resulted in death, was life-threatening, required inpatient hospitalization or prolongation of an existing hospitalization, resulted in persistent or significant disability or incapacity, was a congenital anomaly/birth defect or medically important event. (NCT01659658)
Timeframe: From first dose of study drug through 30 days after administration of the last dose of study drug (up to 115 months)

InterventionParticipants (Count of Participants)
Arm A: Ixazomib + Dexamethasone44
Arm B: Dexamethasone + Melphalan11
Arm B: Dexamethasone + Cyclophosphamide2
Arm B: Dexamethasone + Thalidomide0
Arm B: Dexamethasone + Lenalidomide17

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Overall Survival

Overall survival was defined as the time from the date of randomization to the date of death. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone69.55
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide43.17

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Percentage of Participants With Best Vital Organ (Cardiac and/or Kidney) Response

Vital organ (heart and kidney) response rate was defined as the percentage of participants who achieved vital organ response according to central laboratory results and ISA criteria as evaluated by an adjudication committee. A vital organ response was defined as response of 1 or 2 of the involved vital organs with no change from Baseline in the rest of involved vital organs. Percentages were rounded off to the nearest decimal. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionpercentage of participants (Number)
Arm A: Ixazomib + Dexamethasone19
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide12

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Percentage of Participants With Complete Hematologic Response

Complete hematologic response was defined as the percentage of participants with CR based on central laboratory results and the 2010 ISA Consensus Criteria as assessed by the investigator. CR: Complete disappearance of M-protein from serum and urine on immunofixation, and normalization of FLC ratio. Percentages were rounded off to the nearest decimal. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionpercentage of participants (Number)
Arm A: Ixazomib + Dexamethasone30
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide17

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Percentage of Participants With Overall Hematologic Response

Overall hematologic response was defined as the percentage of participants with complete response (CR), very good partial response (VGPR) and partial response (PR) based on central laboratory results and the 2010 International Society of Amyloidosis (ISA) Consensus Criteria as assessed by an adjudication committee. CR: Complete disappearance of M-protein from serum and urine on immunofixation, and normalization of free light chain (FLC) ratio. VGPR: differential free light chain (difference between involved and uninvolved FLC levels; dFLC) < 40 mg/L. PR: ≥50% reduction in dFLC. Percentages were rounded off to the nearest decimal. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionpercentage of participants (Number)
Arm A: Ixazomib + Dexamethasone53
Arm B: Dexamethasone + Melphalan58
Arm B: Dexamethasone + Cyclophosphamide30
Arm B: Dexamethasone + Thalidomide50
Arm B: Dexamethasone + Lenalidomide51

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Hematologic Disease Progression Free Survival

Hematologic disease PFS was defined as the time from the date of randomization to the date of first documentation of hematologic PD according to central laboratory results and ISA criteria as evaluated by an adjudication committee, or death due to any cause, whichever occurred first. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone29.50
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide27.73

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Progression Free Survival (PFS)

PFS was defined as the time from the date of randomization to the date of first documentation of hematologic disease progression, or organ (cardiac or renal) progression, or death due to any cause, whichever occurred first according to central laboratory results and ISA criteria as evaluated by the investigator. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone11.86
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide7.62

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2-Year Vital Organ (Heart or Kidney) Deterioration and Mortality Rate

Cardiac (Heart) deterioration was defined as the need for hospitalization for heart failure. Kidney deterioration was defined as progression to end-stage renal disease (ESRD) with the need for maintenance dialysis or renal transplantation. Vital organ deterioration was evaluated by an adjudication committee. Percentages were rounded off to the nearest decimal. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: Up to 2 years

Interventionpercentage of participants (Number)
Arm A: Ixazomib + Dexamethasone47
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide54

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Change From Baseline in 36-item Short Form General Health Survey (SF-36) Mental Component Summary Score at Week 28 of the PFS Follow-up

SF-36 Version 2 is a multipurpose, participant completed, short-form health survey with 36 questions that consists of an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures. Physical component summary (PCS) is mostly contributed by physical function (PF), role physical (RP), bodily pain (BP), and general health (GH). Mental component summary (MCS) is mostly contributed by mental health (MH), role emotional (RE), social function (SF), and vitality (VT). Each component on the SF-36 item health survey is scored from 0 (best) to 100 (worst). Total score ranges from 0-100, where higher scores are associated with less disability and better quality of life. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: Baseline, Week 28 of the PFS Follow-up

Interventionscore on a scale (Mean)
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide2.0

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Change From Baseline in Amyloidosis Symptom Scale Total Score at Week 28 of the PFS Follow-up

The amyloidosis symptom scale questionnaire is a participant completed questionnaire that evaluates symptom severity of 3 symptoms: Swelling, Shortness of Breath and Dizziness, each rated on an 11-point scale where: 0=no symptoms to 10=very severe symptoms. Higher scores indicate worsening of symptoms. Total Score is the sum of all responses from the amyloidosis symptom scale ranging from 0 to 30. Higher scores represent higher levels of symptomatology or problems and a negative change from baseline indicates improvement. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: Baseline, Week 28 of the PFS Follow-up

Interventionscore on a scale (Mean)
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide-16.0

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Plasma Concentration of Ixazomib

As prespecified in the protocol, data for this outcome measure was planned to be collected for ixazomib arm group only. (NCT01659658)
Timeframe: Cycle 1, Day 1: 1, 4 hours postdose, Day 14: 144 hours postdose; Cycle 2, Day 1: predose, Day 14: 144 hours postdose; Cycles 3 to 10, Day 1: predose (cycle length=28 days)

Interventionnanogram per milliliter (ng/mL) (Geometric Mean)
Cycle 1 Day 1: 1 Hour Post-doseCycle 1 Day 14: 4 Hours Post-doseCycle 1 Day 14: 144 Hours Post-doseCycle 2 Day 1: Pre-doseCycle 2 Day 14: 144 Hours Post-doseCycle 3 Day 1: Pre-doseCycle 4 Day 1 Pre-doseCycle 5 Day 1 Pre-doseCycle 6 Day 1: Pre-doseCycle 7 Day 1: Pre-doseCycle 8 Day 1: Pre-doseCycle 9 Day 1: Pre-doseCycle 10 Day 1: Pre-dose
Arm A: Ixazomib + Dexamethasone16.51810.6523.8752.0004.7262.1872.2762.2642.2352.2992.0382.1432.232

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Number of Participants in Each Category of the EuroQol 5-Dimensional (EQ-5D) Questionnaire Score

The European Quality of Life (EuroQOL) 5-Dimensional (EQ-5D) is a patient completed questionnaire consisting of 2 pages: the EQ-5D descriptive system and the EQ visual analogue scale (EQ VAS). The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 3 possible choices: no problems to extreme problems. Higher scores=worsening of the quality of life. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: At Week 28 of the OS follow-up

,
InterventionParticipants (Count of Participants)
Mobility: No Problems in Walking AboutMobility: Some Problem in Walking AboutMobility: Confined to BedSelf-Care: No Problems With Self- CareSelf-Care: Some Problems Washing or DressingSelf-Care: Unable to Wash or DressUsual Activities: No Problems With Performing Usual ActivitiesUsual Activities: Some Problem With Performing Usual ActivitiesUsual Activities: Unable to Performing Usual ActivitiesPain/Discomfort: No Pain or DiscomfortPain/Discomfort: Moderate Pain or DiscomfortPain/Discomfort: Extreme Pain or DiscomfortAnxiety/Depression: Not Anxious or DepressedAnxiety/Depression: Moderately Anxious or DepressedAnxiety/Depression: Extremely Anxious or Depressed
Arm A: Ixazomib + Dexamethasone000000000000000
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide010010010010001

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Vital Organ Progression Free Survival

Vital organ PFS is defined as the time from the date of randomization to the date of first documentation of progression of vital organ (heart or kidney) according to central laboratory results and ISA criteria as evaluated by an adjudication committee, or death due to any cause, whichever occurs first. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone15.77
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide11.01

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Time to Vital Organ (Heart or Kidney) Deterioration and Mortality Rate

Time to vital organ deterioration or death was assessed by the investigator and defined as the time from randomization to vital organ (heart or kidney) deterioration or death, whichever occurs first. Cardiac deterioration is defined as the need for hospitalization for heart failure. Kidney deterioration is defined as progression to ESRD with the need for maintenance dialysis or renal transplantation. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From randomization to time of vital organ deterioration or death (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone38.67
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide26.09

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Time To Treatment Failure (TTF)

TTF was defined as the time from randomization to the date of first documented treatment failure. Treatment failure was defined as: 1) death due to any cause; 2) hematologic progression or major organ progression according to central laboratory results and ISA criteria as evaluated by the investigator; 3) clinically morbid organ disease requiring additional therapy; or 4) withdrawn for any reason. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until discontinuation of study drug due to disease progression or unacceptable toxicity, or death whichever occurs first (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone10.32
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide5.32

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Time To Subsequent Anticancer Treatment

Time to subsequent anticancer therapy was defined as the time from randomization to the first date of subsequent anticancer therapy. Participants without subsequent anticancer therapy were censored at the date of death or last known to be alive. As prespecified in the protocol, data was planned to be collected and analyzed in a combined way for non-ixazomib arm groups in this outcome measure. (NCT01659658)
Timeframe: From first dose of study drug until subsequent anticancer treatment (up to 115 months)

Interventionmonths (Median)
Arm A: Ixazomib + Dexamethasone26.48
Arm B: Dexamethasone + Melphalan/Cyclophosphamide/Thalidomide/Lenalidomide12.45

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Maximum Tolerated Dose (MTD) of Carfilzomib Plus Melphalan as Conditioning for Autologous Hematopoietic Cell Transplantation in Patients With Relapsed Multiple Myeloma(MM) [Phase I Portion of Study]

The maximum tolerated dose of carfilzomib that can be safely combined with high dose melphalan as conditioning regimen prior to autologous hematopoietic cell transplantation in patients with relapsed multiple myeloma meeting eligibility criteria. (NCT01690143)
Timeframe: Up to 4 1/2 months

Interventionmg/m2 (Number)
Patients Undergoing Transplantation56

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Frequency of Grades 3 and 4 Non-hematologic Adverse Events During the Transplant Component ( 135 Days)

Grading of AE's is performed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. (NCT01690143)
Timeframe: Up to 4 1/2 months

InterventionParticipants (Count of Participants)
Patients Undergoing Transplantation22

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Complete Response (CR) Rate.

CR defined as the following: Negative immunofixation of the serum and urine. If only the measurable non-bone marrow parameter was free light chain, normalization of free light chain ratio. < 5% plasma cells in bone marrow. And, disappearance of any soft tissue plasmacytomas. (NCT01690143)
Timeframe: Up to 17 months

InterventionParticipants (Count of Participants)
Patients Undergoing Transplantation10

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Median Time for Neutrophil and Platelet Engraftment.

Neutrophil engraftment is defined as the first of three consecutive days with absolute neutrophil count >500/mm3. Platelet engraftment is defined as the first of 3 consecutive days of platelets > 20,000/mm3 without platelet transfusion in the prior 7 days. (NCT01690143)
Timeframe: Up to 1 month.

Interventiondays (Median)
Patients Undergoing Transplantation10

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Very Good Partial Response (VGPR) Rate.

"VGPR defined as any one of the following:~≥ 90% reduction of serum M-protein; ≥ 90% reduction in 24-hour urinary M-protein or decrease to < 100 mg per 24 hour; ≥ 50% decrease in the difference between involved and uninvolved FLC levels or a 50% decrease in level of involved FLC with 50% decrease in ratio; ≥ 50% reduction in bone marrow plasma cells; ≥ 50% reduction in the size of soft tissue plasmacytomas." (NCT01690143)
Timeframe: Up to 17 months

InterventionParticipants (Count of Participants)
Patients Undergoing Transplantation26

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Median Days to Neutrophil Engraftment

Neutrophil engraftment was recorded as the first day that absolute neutrophil counts (ANC) exceeds 0.5 X 10^9/L for three consecutive readings. (NCT01702961)
Timeframe: 30 days post-transplant

Interventiondays (Median)
BEAM + R: Autologous Stem Cell Transplant11

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Disease-free Survival

Disease-free survival at 12 months post-transplant in patients with Hodgkin's disease or non-Hodgkin's lymphomas (NCT01702961)
Timeframe: 12 months post-transplant

Interventionpercentage of participant (Number)
All patientsHodgkin's DiseaseNon-Hodgkin's Lymphomas
BEAM + R: Autologous Stem Cell Transplant768870

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Number of Participants With Overall Best Response Achieved After Transplantation

Response was summarized as complete remission (CR): disappearance of all evidence of disease; partial remission (PR): regression of measurable disease (>=50% decrease in sum of the product of the diameters (SPD) of up to six of the largest dominant nodes or nodal masses) and no new sites; stable disease (SD): failure to attain CR/PR/PD; relapsed disease or progressive disease (PD): any new lesion or increase by >= 50% of previously involved sites from nadir. (NCT01702961)
Timeframe: 3 months post-transplant

Interventionparticipants (Number)
Complete Remission (CR)Partial Remission (PR)Stable Disease (SD)Relapsed Disease or Progressive Disease (PD)
BEAM + R: Autologous Stem Cell Transplant63804

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Complete Response Rate

The primary objective of this study is to determine the complete response rate of lenalidomide and low-dose dexamethasone versus that of lenalidomide and low-dose dexamethasone followed by autologous peripheral blood stem cell transplant in patients with newly diagnosed multiple myeloma (will include unconfirmed complete response (CR), CR and stringent complete response (sCR)). (NCT01731886)
Timeframe: 3 years

InterventionParticipants (Count of Participants)
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation7
Arm B: Low-dose Dexamethasone7

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Overall Survival Rate (OS)

To compare overall survival in subjects receiving autologous peripheral blood stem cell transplant after undergoing induction therapy with lenalidomide and dexamethasone versus in those receiving only lenalidomide and dexamethasone, followed by lenalidomide maintenance in both arms. Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis. (NCT01731886)
Timeframe: 4 years

Interventionpercentage of participants (Number)
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation79.8
Arm B: Low-dose Dexamethasone78.9

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Progression Free Survival (PFS)

PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse. (NCT01731886)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation52.0
Arm B: Low-dose Dexamethasone47.4

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Progression Free Survival (PFS)

PFS is the length of time during and after the treatment of a disease, such as cancer, that a patient lives with the disease but it does not get worse. (NCT01731886)
Timeframe: 4 years

Interventionpercentage of participants (Number)
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation36.0
Arm B: Low-dose Dexamethasone31.6

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Overall Survival Rate (OS)

To compare overall survival in subjects receiving autologous peripheral blood stem cell transplant after undergoing induction therapy with lenalidomide and dexamethasone versus in those receiving only lenalidomide and dexamethasone, followed by lenalidomide maintenance in both arms. Only patients who achieved at least a partial response (PR) following 4 cycles of induction were included in the analysis. (NCT01731886)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Arm A: Low-dose Dexamethasone + Stem Cell Transplantation100
Arm B: Low-dose Dexamethasone94.7

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Induction Response

Induction response is the defined as the proportion of patients who achieve complete remission (CR) or partial remission (PR) during 6 cycles of induction therapy. Response was assessed was using a combination of CT scans and PET scans. Partial and complete response were categorized according to standard lymphoma response criteria, specifically the Revised Response Criteria (Cheson 2007). Given the cycle length of 3 weeks, induction duration per protocol was 18 weeks. (NCT01746173)
Timeframe: Disease was re-staged at cycles 3 and 6 during induction. Median duration of induction therapy in this study cohort was 6 cycles/18 weeks (range 2-6 cycles).

Interventionproportion of patients (Number)
CHOEP + High Dose Therapy + Auto SCT.60

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24-month Progression-Free Survival Rate

24-month progression-free survival rate is defined as the proportion of patients remaining alive and progression-free at 24 months from start of induction therapy. Disease progression was assessed using a combination of CT scans and PET scans. Progression was categorized according to standard lymphoma response criteria, specifically the Revised Response Criteria (Cheson 2007). (NCT01746173)
Timeframe: Disease was re-staged at cycles 3 and 6 during induction, at day 100 post-ASCT, and in long-term follow-up at months 12, 18, 24 and 36. All patients were evaluable up to month 24.

Interventionproportion of patients (Number)
CHOEP + High Dose Therapy + Auto SCT0.0

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The Tolerability of BuMel Regimen

Number of patients who experience one or more unacceptable toxicities (severe sinusoidal obstruction syndrome [SOS] or Grade 4-5 pulmonary toxicity per Common Toxicity Criteria [CTC] v.4.0) during the Consolidation phase of therapy. (NCT01798004)
Timeframe: Up to 28 days post-consolidation therapy, up to 1 year

Interventionparticipants (Number)
All Patients9

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Overall Survival

The one-year survival is defined by the patient who has not died within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year survival patients and the total number of patients. (NCT01807611)
Timeframe: one year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients59

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Number of Transplant Recipients With Successful Engraftment

Neutrophil engraftment will be determined using the parameters put forth by the Center for International Blood and Marrow Registry. Assessments will be made upon review of daily complete blood count and serial chimerism studies. Successful engraftment for the purposes of this objective will be patients who do not experience graft failure. (NCT01807611)
Timeframe: 42 days post engraftment

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients70

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Number of Transplant Recipients With Malignant Relapse

Bone marrow studies for disease status evaluation will be performed at 1-year post-transplant. Testing will include a research evaluation for minimal residual disease. (NCT01807611)
Timeframe: One-year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients18

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Number of Transplant Recipients With Acute and/or Chronic Graft Versus Host Disease (GVHD)

Acute and chronic graft-vs.-host disease will be evaluated using the standard grading criteria. The estimate will be the number of recipients who experienced GVHD divided by the total number of patients considered in this group. (NCT01807611)
Timeframe: 100 days post-transplant for acute GVHD; one year post-transplant for chronic GVHD .

InterventionParticipants (Count of Participants)
Number of Transplant Recipients With Acute Graft Versus Host Disease (aGVHD)24
Number of Transplant Recipients With Chronic Graft Versus Host Disease (cGVHD)16

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Event-free Survival

The one-year event free survival is defined by the patient who has neither experienced relapse nor death within one year after post transplantation. And the rate is calculated by computing the ratio between total number of one year event free survival patients and the total number of patients. (NCT01807611)
Timeframe: One year post-transplantation

InterventionParticipants (Count of Participants)
Experimental: Transplant Recipients49

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Number of Participants With Adverse Events

"Adverse events (AEs)were graded using National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 4.03, where GRADE 1 = Mild; GRADE 2 = Moderate; GRADE 3 = Severe; GRADE 4 = Life-threatening; GRADE 5 = Fatal.~A serious adverse event is an adverse event that met 1 or more of the following criteria:~Death~Life-threatening~Required inpatient hospitalization or prolongation of an existing hospitalization~Resulted in persistent or significant disability/incapacity~Congenital anomaly/birth defect~Important medical event that jeopardized the participant and may have required medical or surgical intervention to prevent 1 of the outcomes listed above.~Treatment-related adverse events are adverse events considered related to at least 1 investigational product by the investigator, including those with unknown relationship." (NCT01818752)
Timeframe: From the first dose of any study drug up to 30 days after the last dose of any study drug as of the data cut-off date of 15 July 2016; median duration of treatment was 52 weeks in both treatment groups.

,
Interventionparticipants (Number)
All adverse eventsAEs ≥ grade 3Serious adverse eventsLeading to discontinuation of study drugFatal adverse eventsTreatment-related adverse events (TRAEs)TRAEs ≥ grade 3Treatment-related serious adverse eventsTRAE leading to discontinuation of study drugTreatment-related fatal adverse events
Bortezomib, Melphalan, Prednisone4543581987320431285102515
Carfilzomib, Melphalan, Prednisone46035423583314082681365410

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European Organisation for Research and Treatment of Cancer Quality of Life Core Module (EORTC QLQ-C30) Global Health Status/Quality of Life (QOL) Scores

"The EORTC QLQ-C30 is a validated self-rating questionnaire including 30 items used to assess the overall quality of life in cancer patients.~It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact).~The EORTC QLQ-C30 Global Health Status/QOL scale was scored between 0 and 100, with higher scores indicating better Global Health Status/QOL." (NCT01818752)
Timeframe: Baseline, weeks 6, 12, 18, 24, 30, 36, 42 and 48

,
Interventionunits on a scale (Mean)
Baseline (n = 425, 425)Week 6 (n = 412, 407)Week 12 (n = 376, 389)Week 18 (n = 341, 369)Week 24 (n = 320, 345)Week 30 (n = 298, 317)Week 36 (n = 285, 308)Week 42 (n = 275, 288)Week 48 (n = 261, 265)
Bortezomib, Melphalan, Prednisone53.356.255.355.757.361.661.963.362.9
Carfilzomib, Melphalan, Prednisone53.961.162.463.263.363.064.065.065.1

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Progression-Free Survival (PFS)

"Progression-free survival was defined as the time from randomization to the earlier of documented disease progression or death due to any cause. PFS was analyzed using Kaplan-Meier methods. The duration of PFS was censored for participants with no baseline and/or post-baseline disease assessments, who started a new anti-cancer therapy before documentation of disease progression or death, death or disease progression after missed disease assessment of 100 consecutive days or longer, or who were alive without documentation of disease progression before the data cutoff date, including lost to follow-up prior to disease progression.~Participants were evaluated for disease response and progression according to the International Myeloma Working Group Uniform Response Criteria (IMWG-URC), determined centrally using a validated computer algorithm in a blinded manner." (NCT01818752)
Timeframe: From randomization until the data cut-off date of 15 July 2016; median follow-up time for PFS was 21.6.and 22.2 months in the bortezomib and carfilzomib arms respectively.

Interventionmonths (Median)
Bortezomib, Melphalan, Prednisone22.1
Carfilzomib, Melphalan, Prednisone22.3

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Percentage of Participants With ≥ Grade 2 Peripheral Neuropathy

"Neuropathy events were defined as Grade 2 or higher peripheral neuropathy as specified by peripheral neuropathy Standardised Medical Dictionary for Regulatory Activities (MedDRA) Query, narrow (scope) (SMQN) terms.~Peripheral neuropathy was assessed by neurologic exam and graded according to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03:~Grade 1: Asymptomatic; Grade 2: Moderate symptoms, limiting instrumental activities of daily living (ADL) Grade 3: Severe symptoms; limiting self-care ADL; Grade 4: Life-threatening consequences, urgent intervention indicated; Grade 5: Death." (NCT01818752)
Timeframe: From the first dose of any study drug up to 30 days after the last dose of any study drug as of the data cut-off date of 15 July 2016; median duration of treatment was 52 weeks in both treatment groups.

Interventionpercentage of participants (Number)
Bortezomib, Melphalan, Prednisone35.1
Carfilzomib, Melphalan, Prednisone2.5

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Overall Survival (OS)

"Overall survival (OS) was defined as the time from randomization to the date of death (whatever the cause). Participants who were alive or lost to follow-up as of the data analysis cut-off date were censored on the date the patient was last known to be alive.~Median overall survival was estimated using the Kaplan-Meier method." (NCT01818752)
Timeframe: From randomization until the data cut-off date of 15 July 2016; median follow-up time for OS was 22.2 and 22.5 months in the bortezomib and carfilzomib arms respectively.

Interventionmonths (Median)
Bortezomib, Melphalan, PrednisoneNA
Carfilzomib, Melphalan, PrednisoneNA

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Overall Response Rate

"Disease response was evaluated according to the IMWG-URC using a validated computer algorithm. Overall response was defined as the percentage of participants with a best overall response of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR).~sCR: As for CR, normal serum free light chain (SFLC) ratio and no clonal cells in bone marrow (BM).~CR: No immunofixation on serum and urine, disappearance of any soft tissue plasmacytomas and < 5% plasma cells in BM biopsy; VGPR: Serum and urine M-protein detectable by immunofixation but not electrophoresis or ≥ 90% reduction in serum M-protein with urine M-protein <100 mg/24 hours. A ≥ 50% reduction in the size of soft tissue plasmacytomas if present at baseline.~PR: ≥ 50% reduction of serum M-protein and reduction in urine M-protein by ≥ 90% or to < 200 mg/24 hours. A ≥ 50% reduction in the size of soft tissue plasmacytomas if present at baseline." (NCT01818752)
Timeframe: Disease response was assessed every 3 weeks during the first 54 weeks and every 6 weeks thereafter until PD or the data cut-off date of 15 July 2016; median follow-up time was 21.6.and 22.2 months in the bortezomib and carfilzomib arms respectively.

Interventionpercentage of participants (Number)
Bortezomib, Melphalan, Prednisone78.8
Carfilzomib, Melphalan, Prednisone84.3

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Complete Response Rate

"Complete response rate was defined as the percentage of participants in each treatment group who achieved a sCR or CR per the IMWG-URC as their best response.~sCR: As for CR, normal serum free light chain (SFLC) ratio and no clonal cells in bone marrow (BM).~CR: No immunofixation on serum and urine, disappearance of any soft tissue plasmacytomas and < 5% plasma cells in BM biopsy." (NCT01818752)
Timeframe: Disease response was assessed every 3 weeks during the first 54 weeks and every 6 weeks thereafter until PD or the data cut-off date of 15 July 2016; median follow-up time was 21.6.and 22.2 months in the bortezomib and carfilzomib arms respectively.

Interventionpercentage of participants (Number)
Bortezomib, Melphalan, Prednisone23.1
Carfilzomib, Melphalan, Prednisone25.9

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Percent Probability of Event-free Survival (EFS)

Probability of Event-free Survival (EFS) for Patients after 18 months. An event is either treatment related mortality (TRM), primary or secondary graft failure, or relapse/non-response (as defined in protocol section 10). Time to event is time from transplant with patients who die between the start of the conditioning regimen and transplant given a time to event of zero. (NCT01824693)
Timeframe: From transplant up to 18 months

Interventionpercent probability (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)83
Arm II (Busulfan, Fludarabine Phosphate)22

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Percent Probability of 18 Months-relapse Event Between Arms

Probability of patients relapsing at 18 months. A relapse event is defined in protocol section 10.2.3. Time to relapse/non-response is defined as time from transplant to when all criteria of section 10.2.3 are met. (NCT01824693)
Timeframe: From transplant up to 18 months

Interventionpercent probability (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)17
Arm II (Busulfan, Fludarabine Phosphate)55

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Percentage of Participants Who Experience Primary Graft Failure Event Between Arms

Primary Graft failure is defined as the failure to achieve an ANC >= 500/uL after 42 days, determined by 3 consecutive measurements on different days; OR < 5% donor cells in blood or bone marrow by day +42 (as demonstrated by a chimerism assay), without evidence of Juvenile Myelomonocytic Leukemia (JMML). (NCT01824693)
Timeframe: Day 0 - day 540 (18 months) following completion of stem cell transplant

Interventionpercentage of patients (Number)
Arm I (Busulfan, Cyclophosphamide, Melphalan)0
Arm II (Busulfan, Fludarabine Phosphate)0

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Complete Response Rate at Day 100

Complete response rate (CRR) is defined as the percentage of complete responses estimated by the total number of patients who achieve a complete response by day 100 post-transplant divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true complete response rate at day 100 will be calculated. (NCT01842308)
Timeframe: At day 100

Interventionpercentage of patients (Number)
Dose Level 317.07

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Adverse Event Rate, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0

These results are reported in the adverse events section. The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration. (NCT01842308)
Timeframe: Up to 5 years

InterventionParticipants (Count of Participants)
Phase 1: Dose Level 36
Phase 1: Dose Level 23
Phase 1: Dose Level 13
Phase 1: Dose Level 02
Phase 2: Dose Level 335

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Progression Free Percentage at 1 and 2 Years Post Registration

"Progression is an Increase of 25% from lowest value in any of the following (A 25% increase refers to M protein, FLC and bone marrow results and does not refer to bone lesions, soft tissue plasmacytoma or hypercalcemia. The lowest value does not need to be a confirmed value. If the lowest serum Mprotein is ≥ 5 g/dL, an increase in serum M-protein of ≥ 1 g/dL is sufficient to define disease progression.), (In the case where a value is felt to be a spurious result per physician discretion (for example, a possible lab error), that value will not be considered when determining the lowest value.): Serum M-protein (absolute increase must be ≥ 0.5 g/dL) and/or Urine M-protein (absolute increase must be ≥ 200 mg/24 hrs) and/or If the only measurable disease is FLC, the difference between involved and uninvolved FLC levels (absolute increase must be>10 mg/dL) and/or If the only measurable disease is BM, bone marrow PC percentage (absolute increase must be ≥ 10%) (Bone marrow criteria for PD" (NCT01842308)
Timeframe: At 1 year and 2 years

Interventionpercentage of patients (Number)
1 year2 years
Dose Level 390.2475.61

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Percentage of Patients With Complete Responses, Defined as a Complete Response Noted as the Objective Status on Two Consecutive Evaluations (Phase II)

The percentage of successes will be estimated by the number of successes divided by the total number of evaluable patients. Exact binomial 95% confidence intervals for the true success percentages will be calculated. (NCT01842308)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
Dose Level 321.95

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Determine Maximum Tolerated Dose by the Number of Patients With a DLT Per Dose Level

Will be defined as the dose level below the lowest dose that induces dose-limiting toxicity(DLT) in at least one-third of patients. For this study, a DLT is any of the following during the first 2 cycles of treatment; Absolute neutrophil count engraftment* delayed beyond day 21 or Platelet engraftment* delayed beyond day 30, grade 3+ related sensory or motor neuropathy, or grade 4+ related non-neurologic or non-hematologic adverse event(excluding nausea, vomiting, and diarrhea. Reported below are the number of patients who experienced a DLT. (NCT01842308)
Timeframe: Up to day 30

InterventionParticipants (Count of Participants)
Phase 1: Dose Level 31
Phase 1: Dose Level 20
Phase 1: Dose Level 10
Phase 1: Dose Level 00

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Percentage of Participants Able to Complete Full Course Therapy

Percentage of participants able to complete the full course of therapy. (NCT01849783)
Timeframe: Up to 6 years

InterventionParticipants (Count of Participants)
Autologous Stem Cell Transplant24

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Median Progression Free Survival (mPFS)

PFS is defined as the time from the start of DPACE to the date of first documentation of disease progression as assessed by the International Myeloma Working Group response criteria or death due to any cause. Progression is defined using the International Myeloma Working Group response criteria, an increase of greater than or equal to 25% from the lower response value. (NCT01849783)
Timeframe: From the start of DPACE for all participants who have had at least one day of protocol treatment. Up to 6 years.

Interventionmonths (Median)
Autologous Stem Cell Transplant76.4

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Mean Change in Quality-Of-Life Indicators Post-Transplant

Measured using the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ-C30) and Multiple Myeloma module (QLQ-MY20). The EORTC QLQ-C30 includes functional scales (physical, role, emotional, cognitive, and social) and global health status. The EORTC QLQ-MY20 includes disease symptoms and treatment side effects scales. Scores are averaged and transformed to 0-100 scale. For functional and global health status, a positive change from baseline (pre-DPACE) indicates improvement whereas for the symptom scales a negative change from baseline (pre-DPACE) indicates improvement. (NCT01849783)
Timeframe: Pre-DPACE, Pre-maintenance, every 6 months for up to 2 years during maintenance. Up to 6 years.

Interventionscore on a scale (Mean)
Change in Physical Functioning at Pre-MaintenanceChange in Physical Functioning at Maintenance Cycle 6Change in Physical Functioning at Maintenance Cycle 12Change in Physical Functioning at Maintenance Cycle 18Change in Physical Functioning at Maintenance Cycle 24Change in Role Functioning at Pre-MaintenanceChange in Role Functioning at Maintenance Cycle 6Change in Role Functioning at Maintenance Cycle 12Change in Role Functioning at Maintenance Cycle 18Change in Role Functioning at Maintenance Cycle 24Change in Emotional Functioning at Pre-MaintenanceChange in Emotional Functioning at Maintenance Cycle 6Change in Emotional Functioning at Maintenance Cycle 12Change in Emotional Functioning at Maintenance Cycle 18Change in Emotional Functioning at Maintenance Cycle 24Change in Cognitive Functioning at Pre-MaintenanceChange in Cognitive Functioning at Maintenance Cycle 6Change in Cognitive Functioning at Maintenance Cycle 12Change in Cognitive Functioning at Maintenance Cycle 18Change in Cognitive Functioning at Maintenance Cycle 24Change in Social Functioning at Pre-MaintenanceChange in Social Functioning at Maintenance Cycle 6Change in Social Functioning at Maintenance Cycle 12Change in Social Functioning at Maintenance Cycle 18Change in Social Functioning at Maintenance Cycle 24Change in Global Health Status at Pre-MaintenanceChange in Global Health Status at Maintenance Cycle 6Change in Global Health Status at Maintenance Cycle 12Change in Global Health Status at Maintenance Cycle 18Change in Global Health Status at Maintenance Cycle 24Change in Distress Symptoms at Pre-MaintenanceChange in Distress Symptoms at Maintenance Cycle 6Change in Distress Symptoms at Maintenance Cycle 12Change in Distress Symptoms at Maintenance Cycle 18Change in Distress Symptoms at Maintenance Cycle 24Change in Side Effects of Treatment at Pre-MaintenanceChange in Side Effects of Treatment at Maintenance Cycle 6Change in Side Effects of Treatment at Maintenance Cycle 12Change in Side Effects of Treatment at Maintenance Cycle 18Change in Side Effects of Treatment at Maintenance Cycle 24
Autologous Stem Cell Transplant1.3-0.61.15.44.67.48.74.47.06.08.75.51.45.88.30.4-4.6-5.1-6.0-3.6-1.02.7-4.90.8-1.55.40.2-3.1-0.13.46.25.95.73.85.3-3.3-0.7-5.1-2.4-2.7

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Overall Response Rate [Complete Response + Very Good Partial Response + Partial Response (CR + VGPR + PR)]

Per the 1993 INRC: measurable tumor defined as product of the longest x widest perpendicular diameter, elevated catecholamine levels and tumor cels in bone marrow. Complete Response (CR)-no evidence of primary tumor or metastases. Very Good Partial Response (VGPR)->90% reduction of primary tumor; no metastases; no new bone lesions, all pre-existing lesions improved. Partial Response (PR)-50-90% reduction of primary tumor; >50% reduction in measurable sites of metastases; 0-1 bone marrow samples with tumor; number of positive bone sites decreased by >50%. Mixed Response (MR)->50% reduction of any measurable lesion with <50% reduction in other sites; no new lesions; <25% increase in any existing lesion. No Response (NR)-no new lesions; <50% reduction but <25% increase in an any existing lesion. No Response (NR)-no new lesions; <50% reduction but <25% increase in any existing legions. Progressive Disease (PD)-any new/increased measurable lesion by >25%; previous negative marrow positive. (NCT01857934)
Timeframe: After two initial courses of chemotherapy (approximately 6 weeks after enrollment)

InterventionParticipants (Count of Participants)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Antibody (hu14.18K322A)42

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Local Failure Rate and Pattern of Failure

Local failure is defined as relapse or progression of disease at the primary site. The cumulative incidence of local failure will be estimated; competing events will include distant failure or death prior to local failure. (NCT01857934)
Timeframe: Up to 3 years

Interventionpercentage of participants (Number)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Antibody Antibody (hu14.18K322A)1.56

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Feasibility of Delivering hu14.18K322A to 6 Cycles of Induction Therapy

"The study is designed to monitor the feasibility of delivering hu14.18K332A to 6 cycles of Induction chemotherapy. The feasibility of Induction therapy for this study will be to target no worse than 75%. A patient was considered as a failure for the 6 cycles of Induction therapy if the patient failed to complete Induction therapy within 155 days since treatment initiation due to toxicity or disease progression, unless the delay was a result of non-medical issues (e.g. not due to protocol toxicity). The proportion of patients who successfully received hu14.18K322A with 6 cycles of induction chemotherapy was estimated together with a 95% confidence interval. The response rate (CR + VGPR + PR) to 6 cycles of Induction chemoimmunotherapy was estimated together with the 95% confidence intervals" (NCT01857934)
Timeframe: After 6 cycles of induction therapy (approximately 24 weeks after enrollment)

Interventionpercentage of participants (Number)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Plus Antibody (hu14.18K322A)96.8

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Event-free Survival (EFS)

EFS was estimated as time to relapse, progressive disease, secondary neoplasm, or death from any cause from enrollment. The EFS was estimated by Kaplan-Meier method (NCT01857934)
Timeframe: 3 years, from time of enrollment

Interventionpercent of probability (Number)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Plus Antibody (hu14.18K322A)73.7

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Dose Limiting Toxicity (DLT) or Severe (Grade 3 or 4) VOD With hu14.18K322A With Allogeneic NK Cells in Consolidation

Number of patients who experience an unacceptable dose limiting toxicity (per CTCAE v 4.0) including the following toxicities: 1) toxicity requiring the use of pressors, including Grade 4 acute capillary leak syndrome or Grade3 and 4 hypotension; 2) Toxicity requiring ventilation support, including Grade 4 respiratory toxicity; 3) Grade 3 or 4 neurotoxicity with MRI evidence of new CNS thrombi, infarction or bleeding in any subject receiving the hu14.18K322A with NK cell combination; 4) Failure of recovery of ANC > 500/mm3 by day 35 after PBSC infusion. Number of patients who experience Grade 3 or Grade 4 (per Common Toxicity Criteria v 4.0) veno occlusive disease (VOD). (NCT01857934)
Timeframe: During the recovery phase after busulfan/melphalan and PBSC rescue (approximately 24-26 weeks after enrollment)

InterventionParticipants (Count of Participants)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Plus Antibody (hu14.18K322A)0

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Dose Limiting Toxicity (DLT)

Number of patients who experience an unacceptable dose limiting toxicity (per CTCAE v 4.0) including the following toxicities: 1) Toxicity requiring the use of pressors, including Grade 4 acute capillary leak syndrome or Grade 3 and 4 hypotension; 2) Toxicity requiring ventilation support, including Grade 4 respiratory toxicity; 3) Grade 3 or 4 neurotoxicity with MRI evidence of new CNS thrombi, infarction or bleeding. (NCT01857934)
Timeframe: During MRD treatment cycle (approximately 8-12 months after enrollment)

InterventionParticipants (Count of Participants)
NB2012 Therapy (Including Induction, Consolidation, and MRD) Plus Antibody (hu14.18K322A)1

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Number of Participants Assessed Post Donor Lymphocyte Infusion (DLI): Disease-free Survival & Non-relapse Mortality, Chimerism and GVHD.

Participants to assess at 6 months post donor lymphocyte infusion (DLI): disease-free survival & non-relapse mortality, chimerism and GVHD (NCT01875237)
Timeframe: 6 months

,
InterventionParticipants (Count of Participants)
disease-free survivaIchimerism post DLIGVHD post DLInon-relapse mortality
Transplant Only0000
Transplat Plus DLI1110

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To Assess the Incidence of Acute GvHD Flare After CR/PR Requiring Additional Agent for Systemic Therapy Before Day 56 Post-administration of AP1903.

"To assess participants with incidence of acute GvHD flare after CR/PR requiring additional agent (including 2.5 mg/kg/day of prednisone [or methylprednisolone equivalent of 2 mg/kg/day]) for systemic therapy before Day 56 post-administration of AP1903." (NCT01875237)
Timeframe: before Day 56 post AP1903

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Evaluate the Safety of Donor Lymphocyte Infusion Followed by Dimerizer Drug, AP1903 by Number of Participants With Adverse Events.

To evaluate the safety of the infusion of inducible caspase 9 (BPZ-1001) modified T-cells followed by dimerizer drug, AP1903. Safety evaluated by number of participants with Adverse events. (NCT01875237)
Timeframe: up to 3.5 years

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Assess Post Donor Lymphocyte Infusion (DLI) Chimerism

To assess the number of Participants with 100% donor chimerism at 6 months post donor lymphocyte infusion (DLI) (NCT01875237)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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To Assess the Proportions of GvHD Response Post-administration of AP1903.

To assess the proportions of GvHD complete response (CR), partial response (PR), mixed response, no response, and progression among surviving patients at Day 3, 7, 14, 28, and 56 post-administration of AP1903. (NCT01875237)
Timeframe: Day 3, 7, 14, 28, and 56 post-administration of AP1903

InterventionParticipants (Count of Participants)
Day 3 post-administration of AP190371985950Day 3 post-administration of AP190371985951Day 7 post-administration of AP190371985951Day 7 post-administration of AP190371985950Day 14 post-administration of AP190371985951Day 14 post-administration of AP190371985950Day 28 post-administration of AP190371985951Day 28 post-administration of AP190371985950Day 56 post-administration of AP190371985951Day 56 post-administration of AP190371985950
no responsecomplete responseprogressionpartial response
Transplat Plus DLI1
Transplat Plus DLI0
Transplant Only0

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To Assess the Incidence of GvHD Treatment Failure Post-administration of AP1903.

To assess the incidence of GvHD treatment failure, defined as no response, progression, administration of additional therapy for GvHD, or mortality post-administration of AP1903. (NCT01875237)
Timeframe: Day 3, 7, 14, 28, and 56 post-administration of AP1903.

,
InterventionParticipants (Count of Participants)
Day 3 post-administration of AP1903.Day 7 post-administration of AP1903.Day 14 post-administration of AP1903.Day 28 post-administration of AP1903.Day 56 post-administration of AP1903.
Transplant Only00000
Transplat Plus DLI11111

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To Assess the Proportion of Patients Developing Grade I-IV Acute GvHD

To assess the proportion of patients developing grade I-IV acute GvHD by Day 28, 56, and 180 post DLI. (NCT01875237)
Timeframe: Day 28, 56, and 180 post DLI.

,
InterventionParticipants (Count of Participants)
Day 28Day 56Day 180
Transplant Only000
Transplat Plus DLI110

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To Assess the Incidence of Epstein-Barr Virus -PTLD or EBV Reactivation Requiring Therapy Post DLI.

To assess the incidence of Epstein-Barr virus (EBV)-associated lymphoproliferative disorder or EBV reactivation requiring therapy post DLI. (NCT01875237)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Transplant Only0
Transplat Plus DLI1

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Number of Participants With Graft Failure

"Primary endpoint:~In each group, the Number of participants with Graft Failure at the 2 years endpoint will be estimated using the Kaplan Meier product limit estimator." (NCT01877837)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Patients With Sickle Cell Anemia3

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Overall Survival

"Secondary endpoints:~Overall survival: The distribution of time to death from any cause will be estimated by Kaplan- Meier product limit function and plotted. The overall survival will be measured from the time of transplant to any death and patients will be followed for 2 years." (NCT01877837)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Patients With Sickle Cell Anemia23

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Overall Survival at 2 Years

Overall survival (OS) is defined as time from start of protocol treatment to death from any cause. It was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT01885689)
Timeframe: From start of protocol treatment to death due to any cause, or last follow-up, whichever comes first, assessed up to 2 years.

Interventionpercent probability (Number)
Treatment (Clofarabine, Melphalan, Transplant)67

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Progression-free Survival at 2 Years

Progression-free survival (PFS) is defined as time from start of protocol treatment to disease relapse/progression, death or last contact, whichever occurs first. Progression-free survival was estimated using the Kaplan-Meier method; the 95% confidence interval was calculated using Greenwood's formula. (NCT01885689)
Timeframe: From start of protocol treatment to death due to any cause, disease relapse/progression, or last follow-up, whichever comes first, assessed up to 2 years.

Interventionpercent probability (Number)
Treatment (Clofarabine, Melphalan, Transplant)54

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Progression-free Survival Following Autologous Stem Cell Transplant (ASCT)

Estimate the 1 year progression-free survival (PFS) rate after ASCT (NCT01921387)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Treatment (90Y-BC8-DOTA, Chemotherapy, PBSC)12

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The Lowest Antibody (Yttrium 90-BC8-DOTA) Dose (mg/kg) That is Consistent With a Favorable Biodistribution Rate >= 80% in Lymphoma Patients

(NCT01921387)
Timeframe: Up to 5 years

Interventionmg/kg (Number)
Treatment (90Y-BC8-DOTA, Chemotherapy, PBSC)0.75

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Maximum-tolerated Dose (MTD) of Yttrium-90-BC8-DOTA

Single patients will be treated at escalating doses in 2-Gy increments (Table 4) until a DLT is observed. Once a DLT is observed, the second stage will begin at the next lower dose level and patients will be treated in cohorts of 4. (NCT01921387)
Timeframe: Within 30 days post-transplant

InterventionGy - MTD (Number)
Treatment (90Y-BC8-DOTA, Chemotherapy, PBSC)34

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Estimated Dose to Tumor Sites Based on the Tumor to Normal Organ Ratios Derived From Dosimetry Estimates Coupled With the Absorbed Dose to Normal Organs Based on the Administered Activity of Yttrium Y 90 Anti-CD45 Monoclonal Antibody BC8

Will be evaluated among all patients and among those treated at the estimated MTD. (NCT01921387)
Timeframe: Up to 5 years

InterventionmCi (Number)
Treatment (90Y-BC8-DOTA, Chemotherapy, PBSC)52.8

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Disease-free Survival

Percentage of patients who survive without any signs or symptoms of cancer at 1 year. (NCT01969435)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)70

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Disease-free Survival

Percentage of patients who survive without any signs or symptoms of cancer at 2 years. (NCT01969435)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)64

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Efficacy as Measured by Response Rates

"The response rates according to each category of response Complete Response (CR), Partial Response (PR), Stable Disease (SD), and Progressive Disease (PD) will be summarized by the proportion of patients meeting each criterion.~Evaluated using PET or CT scan and Revised Response Criteria for Malignant Lymphoma" (NCT01969435)
Timeframe: Up to Day 100

Interventionpercentage of participants (Number)
Complete responsePartial responseStable diseaseProgressive disease
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)844012

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Overall Survival (OS) Rate

(NCT01969435)
Timeframe: Median follow-up 15.4 months (range 4.7-24.6)

Interventionpercentage of participants (Number)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)10

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Time to Engraftment (Platelet)

Time from the date of transplant to the date of platelet engraftment. (NCT01969435)
Timeframe: Assessed up to day 100

Interventiondays (Median)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)19

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Time to Engraftment (Neutrophil)

Time from the date of the transplant to the date of neutrophil engraftment. (NCT01969435)
Timeframe: Assessed up to day 30

Interventiondays (Median)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)10

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Relapse Free Survival

(NCT01969435)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)0

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Progression-free Survival Rate (PFS)

(NCT01969435)
Timeframe: 1 year

Interventionpercentage of participants (Median)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)70

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Progression-free Survival (PFS) Rate

PFS - Time from start of treatment to the time of progression or death, whichever occurs first. (NCT01969435)
Timeframe: 6 months

Interventionpercentage of participants (Median)
Melphalan, Carmustine, Etoposide, Cytarabine (BEAM)84

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Frequency of DLT

Maximum tolerated dose (MTD) of azacitidine based on DLT was defined as any Grade 4 nonhematologic and noninfectious toxicity or any grade 3 mucositis or skin toxicity lasting > 3 days at peak severity. For dose finding, the continunal reassessment method was used with a target DLT probability per cohort of 25%. Azacitidine doses were chosen adaptively for sucessive cohorts with a minimum size of 2 patients. Toxicity scoring followed the National Cancer Institute Common Toxicity Criteria, version 3. (NCT01983969)
Timeframe: Enrollment up to day 30 post transplant for each dosing cohort

InterventionDose-limiting toxicities (Number)
Azacitidine Dose Level 116
Azacitidine Dose Level 228
Azacitidine Dose Level 340

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Participants With Event-free Survival (EFS)

EFS is defined as the time from transplantation to either relapse, second tumors, or death, whichever occurred first, or last contact. EFS was analzyed by the individual disease groups rather than the cohort dose levels. (NCT01983969)
Timeframe: Enrollment up to 100 days post transplant.

InterventionParticipants (Count of Participants)
DLBCL17
Hodgkin Lymphoma16
T-cell NHL7
Other B-cell Lymphoma5

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Number of Successful Unrelated Cord Blood (UCB) Transplants

The number of patients who received successful UCB transplants as evidenced by absolute neutrophil recovery. (NCT02007863)
Timeframe: 2 Years

Interventionparticipants (Number)
Umbilical Cord Blood + Chemotherapy2

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Disease Response 30 Days Post-Transplant

Proportion of patients achieving a complete remission (CR; disappearance of clinically overt disease with no FDG-avid lesions on PET scan), partial remission (PR; reduction in clinical disease buden and >50% bi-dimensional decrease in tumor size on imaging), stable disease (SD; failure to meet the criteria for CR, PR or PD with no new areas of disease involvement) or progressive disease (PD; the appearance of any new lesion >1.5cm in size or a greater than 50% increase in the diameter of a previously identified node of over 1cm in size), as per Cheson Criteria, 30 days following autologous or allogeneic stem cell transplant (NCT02059239)
Timeframe: 30 days after stem cell transplant

,
InterventionParticipants (Count of Participants)
Complete RemissionPartial RemissionStable DiseaseProgressive DiseaseNot Assessed
Chemo Plus Allogeneic Transplantation73111
Chemo Plus Autologous Transplantation122200

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Disease Response at 1 Year Post-Transplant

Proportion of patients achieving a complete remission (CR; disappearance of clinically overt disease with no FDG-avid lesions on PET scan), partial remission (PR; reduction in clinical disease buden and >50% bi-dimensional decrease in tumor size on imaging), stable disease (SD; failure to meet the criteria for CR, PR or PD with no new areas of disease involvement) or progressive disease (PD; the appearance of any new lesion >1.5cm in size or a greater than 50% increase in the diameter of a previously identified node of over 1cm in size), as per Cheson Criteria, 1 year following autologous or allogeneic stem cell transplant (NCT02059239)
Timeframe: 1 year after stem cell transplant

,
InterventionParticipants (Count of Participants)
Complete RemissionPartial RemissionStable DiseaseProgressive DiseaseNot AssessedPatient Deceased
Chemo Plus Allogeneic Transplantation400207
Chemo Plus Autologous Transplantation1210300

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Overall Survival at Day 365 Post-Transplant

The time from stem cell infusion (Day 0) to death from any cause. (NCT02059239)
Timeframe: From Day 0 until time of death, assessed up to 365 days post-transplant

,
InterventionParticipants (Count of Participants)
AliveDeceased
Chemo Plus Allogeneic Transplantation67
Chemo Plus Autologous Transplantation160

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Number of Patients Achieving Platelet Engraftment

Proportion of patients who successfully achieve platelet engraftment after stem cell transplant, defined as a platelet count of >20k/microL for three consecutive days without transfusion support for seven consecutive days. (NCT02059239)
Timeframe: 74 Days Post-Transplant

InterventionParticipants (Count of Participants)
Chemo Plus Autologous Transplantation16
Chemo Plus Allogeneic Transplantation12

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Number of Patients Achieving Neutrophil Engraftment

Proportion of patients who successfully achieve neutrophil engraftment after stem cell transplant, defined as an absolute neutrophil count of 500/mm3 or for three consecutive days. (NCT02059239)
Timeframe: 35 Days Post-Transplant

InterventionParticipants (Count of Participants)
Chemo Plus Autologous Transplantation16
Chemo Plus Allogeneic Transplantation13

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Disease Response Following Salvage Chemotherapy

Proportion of patients achieving a complete remission (CR; disappearance of clinically overt disease with no FDG-avid lesions on PET scan), partial remission (PR; reduction in clinical disease buden and >50% bi-dimensional decrease in tumor size on imaging), stable disease (SD; failure to meet the criteria for CR, PR or PD with no new areas of disease involvement) or progressive disease (PD; the appearance of any new lesion >1.5cm in size or a greater than 50% increase in the diameter of a previously identified node of over 1cm in size), as per Cheson Criteria, following salvage chemotherapy with Bendamustine. (NCT02059239)
Timeframe: Within 14 days of salvage chemotherapy treatment

,
InterventionParticipants (Count of Participants)
Complete RemissionPartial RemissionStable DiseaseProgressive DiseaseNot Assessed
Chemo Plus Allogeneic Transplantation16261
Chemo Plus Autologous Transplantation48411

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Progression-Free Survival After Stem Cell Transplant

Time elapsed between stem cell transplant (Day 0) and disease progression, as defined by the Cheson Criteria (the appearance of any new lesion >1.5cm in size or a greater than 50% increase in the diameter of a previously identified node of over 1cm in size) (NCT02059239)
Timeframe: Stem cell transplant (Day 0) up to 2 years post-transplant

InterventionMonths (Median)
Chemo Plus Autologous TransplantationNA
Chemo Plus Allogeneic Transplantation8

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Rate of Metastases of Retinoblastoma

The percentage of patients who experience metastases of retinoblastoma will be estimated. Ineligible patients or patients who do not receive any protocol therapy are excluded from this analysis (NCT02097134)
Timeframe: Up to 2 years

InterventionPercentage of Patients (Number)
Melphalan0

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Incidence of Grade 3 or Higher CTCAE Adverse Events Associated With Multiple Doses of IA Chemotherapy

The percentage of patients with at least 1 occurrence of grade 3 or higher CTCAE adverse experience will be provided. Ineligible patients or patients who do not receive any protocol therapy are excluded from reporting of adverse events. (NCT02097134)
Timeframe: Up to 30 days after completion of study treatment

InterventionPercentage of patients (Number)
Melphalan38

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Probability of Ocular Salvage

A patient will be considered an ocular-salvage success if enucleation because of disease progression or toxicity is not required during the 2 years following enrollment. (NCT02097134)
Timeframe: 2 years

InterventionPercentage of patients (Number)
Melphalan36

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Number of Patients Experiencing Feasibility Failure

Feasibility failure is defined as a) interventional radiologist is unable to access the ophthalmic artery for the 1st chemotherapy administration for any reason; b) patient develops central retinal artery occlusion after the 1st or 2nd course that does not reopen by the time the next injection is due; or c) the patient cannot receive all three treatments because of Common Terminology Criteria for Adverse Events (CTCAE) complications grade III or IV that are considered possibly, probably or likely related treatment. (NCT02097134)
Timeframe: Up to 4 months

Interventionparticipants (Number)
Melphalan4

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Number of Participants With Overall Response

"Overall response rate=CR+sCR+VGPR+PR~Complete response (CR), disappearance of monoclonal protein from the blood & urine and <5% plasma cells in bone marrow &disappearance of soft tissue plasmacytomas~Stringent complete response (sCR), CR & normal free light chain ratio & absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence~Very good partial response (VGPR), serum and urine monoclonal protein detectable by immunofixation but not on electrophoresis OR > 90% reduction in serum monoclonal protein with urine monoclonal protein < 100 mg per 24 hours and if present, > 50% reduction in the size of soft tissue plasmacytomas~Partial response (PR), > 50% reduction in the level of the serum monoclonal protein & reduction in urine monoclonal protein & > 50% reduction in the size of soft tissue plasmacytomas & if serum and urine monoclonal protein are unmeasurable and serum free light chain is unmeasurable, a > 50% reduction in plasma cells is required" (NCT02112045)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)41
Control: High Dose Melphalan (HDM)42

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Number of Participants With Platelet Engraftment

Platelet engraftment is defined as an untransfused platelet measurement >20,000/mm3 × 3 consecutive daily assessments. The first of 3 consecutive days for which the untransfused platelet measurement is >20,000/mm3 will be recorded as the date of platelet engraftment. Time to platelet engraftment will be calculated as the time from receiving the date of ASCT to the date of platelet engraftment. Untransfused is defined as no transfusions within 7 days. (NCT02112045)
Timeframe: Up to Day 100

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)44
Control: High Dose Melphalan (HDM)44

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Overall Survival as Measured by Number of Participants Alive at Last Follow-up

OS is defined as the duration from the time of transplant Day 0 to death or last follow-up. (NCT02112045)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)40
Control: High Dose Melphalan (HDM)43

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Progression-free Survival as Measured by Number of Participants Without Disease Progression at Last Follow-up

PFS is defined as the duration from time of transplant Day 0 to time of first progression/clinical relapse, death, or the date the patient was last known to be in remission (NCT02112045)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)37
Control: High Dose Melphalan (HDM)36

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Number of Participants With Adverse Events

-Assessed by NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 (NCT02112045)
Timeframe: Up through Day 30

,
InterventionParticipants (Count of Participants)
Febrile neutropeniaAtrial fibrillationLeft ventricular systolic functionSinus tachycardiaExcessive cerumenImpacted cerumenDry eyeAbdominal painColitisColonic perforationConstipationDiarrheaEnterocolitisEpigastric painEsophagitisHemorrhoidsIleusMucositis oralNauseaOral painUpper gastrointestinal hemorrhageEdema limbsFeverInfusion related reactionMalaisePainAllergic reactionBacteremia-Coagulase negative staphyococcusBacteremia - Enterobacter ClocaeBacteremia - FusobacteriumBacteremia - MSSABacteremia - Pseudomonas AeruginosaBacteremia - Streptococcus mitisCandida (groin)Clostridium difficileLung infectionMucosal infection (oral thrush)Peri-rectal yeast infectionSepsisSkin infectionSplenic infectionUpper respiratory infectionUrinary tract infectionVaginal infectionBleeding from CVC insertion siteFallActivated partial thromboplastin time prolongedAspartate aminotransferase increasedBlood bilirubin increasedCreatinine increasedElectrocardiogram QT corrected interval prolongedWeight lossAnorexiaDehydrationHypernatremiaHyperuricemiaHypoalbuminemiaHypocalcemiaHypokalemiaHyponatremiaHypophosphatemiaBone painMuscle cramps-back/legsRight thigh/leg painAcitinic Keratosis (right shoulder)HeadacheReversible posterior leukoencephalopathy syndromeSyncopeTremorAnxietyDeliriumInsomniaHematuriaProteinuriaCoughDyspneaHypoxiaPleural effusionPulmonary edemaWheezingErythematous nodulesPruritusRash acneiformRash maculo-papularSkin lesionFlushingHematomaHypertensionHypotensionPhlebitisThromboembolic eventAtrial flutterMyocardial infarctionPalpitationsSupraventricular tachycardiaAdrenal insufficiencySIADHDyspepsiaDysphagiaHemorrhoidal hemorrhageOdynophagiaChillsMulti-organ failureNon-cardiac chest painBacteremia - Gram PositiveBacteremia-Serratia liquefaciens/pantoea speciesCatheter related infectionCMV viremiaEsophageal infectionOtitis mediaRhinitis infectiveAlanine aminotransferase increasedAlkaline phosphatase increasedINR increasedHyperglycemiaUncontrolled Diabetes MellitusGeneralized muscle weaknessJaw painJoint effusionEncephalopathyPeripheral sensory neuropathyPresyncopeAcute kidney injuryUrinary frequencyUrinary retentionUrinary tract painScrotal painAtelectasisEpistaxisHiccupsPneumonitisProductive coughRespiratory failureSore throatFacial rash/dermatitisFollicular rash-back/posterior neckRash forehead/backSkin ulceration
Control: High Dose Melphalan (HDM)272131112111111112114511141111111111113111131441111123111311202216135111111112111211511112111111121100000000000000000000000000000000000000000000000
Experimental: Granix and High Dose Melphalan (HDM)3030100020005000001631005100120003103310013020102113406161218251644040001000000000080110020000150311111111111121111111113311111212111111221111111

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Number of Participants With Neutrophil Engraftment

Neutrophil engraftment is defined as ANC ≥ 0.5 × 10^9/L × 3 consecutive daily assessments. The first of 3 consecutive days for which ANC ≥ 0.5 × 109/L will be recorded as the date of neutrophil engraftment. Time to neutrophil engraftment will be calculated as the time from the date of the ASCT to the date of neutrophil engraftment. (NCT02112045)
Timeframe: Up to Day 30

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)44
Control: High Dose Melphalan (HDM)45

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Number of Participants With Complete Response or Stringent Complete Response

"Complete response (CR) requires all of the following:~Disappearance of monoclonal protein by both protein electrophoresis and immunofixation studies from the blood and urine~<5% plasma cells in the bone marrow~Disappearance of soft tissue plasmacytomas~Stringent complete response (sCR) requires all of the following:~CR as defined above~Normal free light chain ratio~Absence of clonal cells in the bone marrow by immunohistochemistry or immunofluorescence" (NCT02112045)
Timeframe: Day +100

InterventionParticipants (Count of Participants)
Experimental: Granix and High Dose Melphalan (HDM)17
Control: High Dose Melphalan (HDM)17

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The Remission Rate for Participants With High-risk Myeloma

Toward improving the clinical outcomes of research subjects with high-risk MM (HR-MM) in the context of the immediately preceding TT5 trial 2008-02 and TT3 trials 2003-33 and 2006-66, TT5B will attempt to accelerate and sustain, at 2 years from starting therapy, the proportion of subjects in complete remission (AS-CR-2) by reducing host-imposed toxicity and thus facilitating timely completion of highly synergistic 8-drug combination therapy, including the next generation proteasome inhibitor, Carfilzomib (CFZ). This will result in avoiding MM re-growth that, we postulate, ensued in TT3 during recovery phases from severe de-conditioning. Furthermore, we speculate that the incidence of positive minimal residual disease (MRD) will be reduced with the addition of one cycle of consolidation therapy. (NCT02128230)
Timeframe: from baseline to either death or study completion for each subject (up to approximately 48 months)

InterventionParticipants (Number)
Total Therapy 5B0

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Time to Response

Time to response, defined as the time between the date of randomization and the first efficacy evaluation that the participant has met all criteria for PR or better. PR: >=50% reduction of serum M-protein and reduction in 24-hour urinary M-protein by >=90% or to <200 mg/24 hours; If the serum and urine M-protein are not measurable, a decrease of >=50% in the difference between involved and uninvolved FLC levels is required in place of the M-protein criteria; If serum and urine M-protein are not measurable, and serum free light assay is also not measurable, >=50% reduction in bone marrow PCs is required in place of M-protein, provided baseline bone marrow plasma cell percentage was >=30%. (NCT02195479)
Timeframe: From randomization to first documented PR or better (up to 2.4 years)

InterventionMonths (Median)
Velcade, Melphalan and Prednisone (VMP)0.82
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)0.79

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Time to Disease Progression (TTP)

TTP: Time from date of randomization to date of first documented evidence of PD or death due to PD, whichever occurs first. PD per IMWG criteria- Increase of 25 % from lowest response value in one of following: Serum and urine M-component (absolute increase >=0.5 gram per deciliter [g/dL] and >=200 mg/24 hours respectively); Only in participants without measurable serum and urine M-protein levels: difference between involved and uninvolved FLC levels (absolute increase >10 milligram per deciliter [mg/dL]); Only in participants without measurable serum and urine M-protein levels and without measurable disease by FLC levels, bone marrow plasma cells (PC)% (absolute % >=10%); Bone marrow PC %: absolute % >10%; Definite development of new bone lesions/soft tissue plasmacytomas or definite increase in size of existing bone lesions/soft tissue plasmacytomas and Development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to the PC proliferative disorder. (NCT02195479)
Timeframe: From randomization to either disease progression or death due to PD whichever occurs first (up to 2.4 years)

InterventionMonths (Median)
Velcade, Melphalan and Prednisone (VMP)19.35
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)NA

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Progression Free Survival (PFS)

PFS- duration from date of randomization to Progressive disease (PD)/death, whichever occurs first. PD per IMWG criteria-Increase of 25% from lowest response value in one of following: Serum and urine M-component (absolute increase >=0.5 gram per deciliter [g/dL] and >=200 milligram [mg]/24 hours respectively); Only participants without measurable serum and urine M-protein levels: difference between involved and uninvolved free light chain (FLC) levels (absolute increase>10 mg/dL); Only participants without measurable serum and urine M-protein levels,without measurable disease by FLC levels,bone marrow Plasma cells (PC) %(absolute % >=10%);Bone marrow PC%: absolute% >10%; Definite development of new bone lesions/soft tissue plasmacytomas/definite increase in size of existing bone lesions/soft tissue plasmacytomas and Development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to the PC proliferative disorder. (NCT02195479)
Timeframe: From randomization to either disease progression or death whichever occurs first (up to 2.4 years)

InterventionMonths (Median)
Velcade, Melphalan and Prednisone (VMP)18.14
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)NA

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Percentage of Participants With Very Good Partial Response (VGPR) or Better

VGPR or better rate was defined as the percentage of participants who achieved VGPR or complete response (CR) (including stringent complete response[sCR]) according to the IMWG criteria during or after the study treatment. VGPR: Serum and urine component detectable by immunofixation but not on electrophoresis, or >= 90% reduction in serum M-protein plus urine M-protein level less than (<) 100 milligram (mg) per 24 hour; CR: negative immunofixation on the serum and urine, Disappearance of any soft tissue plasmacytomas and < 5% plasms cells (PCs) in bone marrow; sCR: CR in addition to having a normal FLC ratio and an absence of clonal cells in bone marrow by immunohistochemistry, immunofluorescence, 2-4 color flow cytometry. (NCT02195479)
Timeframe: From randomization to disease progression (up to 2.4 years)

InterventionPercentage of participants (Number)
Velcade, Melphalan and Prednisone (VMP)49.7
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)71.1

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Percentage of Participants With Stringent Complete Response (sCR)

sCR as per IMWG criteria is CR plus normal free light chain (FLC) ratio and absence of clonal PCs by immunohistochemistry, immunofluorescence or 2- to 4-color flow cytometry. CR: Negative immunofixation on the serum and urine; Disappearance of any soft tissue plasmacytomas; <5% plasma cells (PCs) in bone marrow. (NCT02195479)
Timeframe: From randomization to disease progression (up to 2.4 years)

InterventionPercentage of participants (Number)
Velcade, Melphalan and Prednisone (VMP)9.3
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)20.3

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Percentage of Participants With Negative Minimal Residual Disease (MRD)

The Minimal Residual Disease negativity rate was defined as the percentage of participants who had negative MRD (detection of less than 1 malignant cell among 100,000 normal cells) assessment at any timepoint after the first dose of study drugs by evaluation of bone marrow aspirates or whole blood at 10^-5 threshold. MRD was evaluated by using Deoxyribonucleic acid (DNA) sequencing of immunoglobulin genes. MRD was assessed in participants who achieved complete response or stringent complete response (CR/sCR). (NCT02195479)
Timeframe: From randomization to disease progression (up to 2.4 years)

InterventionPercentage of participants (Number)
Velcade, Melphalan and Prednisone (VMP)6.2
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)22.3

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Percentage of Participants With Complete Response (CR) or Better

CR or better rate was defined as the percentage of participants with a CR or better (i.e. CR and sCR) as per IMWG criteria. CR: as negative immunofixation on the serum and urine and disappearance of soft tissue plasmacytomas and less than (<) 5 percent plasma cells in bone marrow; sCR: CR plus normal free light chain (FLC) ratio and absence of clonal PCs by immunohistochemistry, immunofluorescence or 2- to 4-color flow cytometry. (NCT02195479)
Timeframe: From randomization to disease progression (up to 2.4 years)

InterventionPercentage of participants (Number)
Velcade, Melphalan and Prednisone (VMP)24.4
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)42.6

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Overall Survival (OS)

Overall Survival (OS) was defined as the number of days the date of randomization to date of death. Median Overall Survival was estimated by using the Kaplan-Meier method. (NCT02195479)
Timeframe: From randomization to death (up to approximately 2.4 years)

InterventionMonths (Median)
Velcade, Melphalan and Prednisone (VMP)NA
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)NA

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Overall Response Rate (ORR)

The Overall response rate was defined as the percentage of participants who achieved a partial response (PR) or better, according to the International Myeloma Working Group (IMWG) criteria, during the study or during follow up. IMWG criteria for PR: greater than or equal to (>=) 50 percentage(%) reduction of serum M-protein and reduction in 24 hour urinary M-protein by >=90% or to <200 mg/24 hours, if the serum and urine M-protein are not measurable, a decrease of >=50% in the difference between involved and uninvolved free light chain (FLC) levels is required in place of the M-protein criteria, If serum and urine M-protein are not measurable, and serum free light assay is also not measurable, >=50% reduction in bone marrow plasma cells (PCs) is required in place of M-protein, provided baseline bone marrow plasma cell percentage was >=30%, in addition to the above criteria, if present at baseline, a >=50% reduction in the size of soft tissue plasmacytomas is also required. (NCT02195479)
Timeframe: From randomization to disease progression (up to 2.4 years)

InterventionPercentage of participants (Number)
Velcade, Melphalan and Prednisone (VMP)73.9
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)90.9

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Change From Baseline in EuroQol 5 Dimensions-5 Level (EQ-5D-5L) Utility Score

EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L Visual Analog Scale. The EQ-5D-5L descriptive system provides a profile of the participant's health state 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 response options (no problems, slight problems, moderate problems, severe problems and extreme problems) that reflect increasing levels of difficulty. The participant was asked to indicate his/her current health state by selecting the most appropriate level in each of the 5 dimensions. Responses to the 5 dimension scores were combined and converted into a single preference-weighted health utility index score 0 (0.0- worst health state) to 1 (1.0- better health state) representing the general health status of the individual based on the UK scoring algorithm. (NCT02195479)
Timeframe: Baseline, Months 3, 6, 9, 12 and 18

,
InterventionUnits on a scale (Mean)
BaselineMonth 3Month 6Month 9Month 12Month 18
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)0.570.120.130.160.170.13
Velcade, Melphalan and Prednisone (VMP)0.590.090.120.160.150.13

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Duration of Response (DOR)

DOR: participants with a confirmed response (PR or better) as time between first documentation of response and disease progression, IMWG response criteria, or death due to PD, whichever occurs first. PD: Increase of 25% from lowest response value in any one of following: Serum M-component (absolute increase>=0.5 g/dL); Urine M-component (absolute increase>=200 mg/24 hours); Only participants without measurable serum and urine M-protein levels: difference between involved and uninvolved FLC levels (absolute increase >10 mg/dL); Only participants without measurable serum and urine M-protein levels and without measurable disease by FLC levels, bone marrow PC%(absolute%>=10%); Bone marrow PC's %: absolute%>10%; Definite development of new bone lesions/soft tissue plasmacytomas/definite increase in the size of existing bone lesions or soft tissue plasmacytomas and Development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to PC proliferative disorder. (NCT02195479)
Timeframe: Up to 2.4 years

InterventionMonths (Median)
Velcade, Melphalan and Prednisone (VMP)21.3
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)NA

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Percentage of Participants With Best M-protein Response

Percentage of participants with Best M- protein response of 100% reduction and >=90% to < 100% reduction were assessed. Best M-protein response was defined as the maximal percent reduction or the lowest percent increase from baseline in serum M-protein for participants with measurable heavy chain at baseline or urine M-protein for participants without measurable heavy chain, but with measurable light chain disease at baseline. For participants without measurable heavy chain and light chain disease at baseline, best response in serum free light chain (FLC) was defined as the maximal percent reduction or the lowest percent increase from baseline in the difference between involved and uninvolved serum FLC level (dFLC). (NCT02195479)
Timeframe: Approximately up to 2.4 years

,
InterventionPercentage of participants (Number)
Best M-protein response in serum: 100% reductionBest M-protein response in serum:>= 90 to < 100%Best M-protein response in urine:100% reductionBest M-protein response in urine:>=90 to < 100%Best response in dFLC:100% reductionBest response in dFLC: >=90% to < 100% reduction
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)58.515.290.57.10100.0
Velcade, Melphalan and Prednisone (VMP)38.714.669.413.9077.8

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Change From Baseline in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L): Visual Analogue Scale (VAS)

EQ-5D-5L is a standardized, participant-rated questionnaire to assess health-related quality of life. The EQ-5D-5L includes 2 components: the EQ-5D-5L health state profile (descriptive system) and the EQ-5D-5L Visual Analog Scale. The Visual Analogue Scale is designed to rate the participant's current health state on a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. (NCT02195479)
Timeframe: Baseline, Months 3, 6, 9, 12 and 18

,
InterventionUnits on a scale (Mean)
BaselineMonth 3Month 6Month 9Month 12Month 18
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)57.909.2810.8312.5010.7912.04
Velcade, Melphalan and Prednisone (VMP)60.334.207.409.8910.807.65

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Change From Baseline in European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ)-C30: Emotional Functioning Score

"The EORTC QLQ-C30 is a 30 items self-reporting questionnaire, with a 1 week recall period, resulting in 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning, and social functioning), 1 Global Health Status (GHS) scale, 3 symptom scales (fatigue, nausea and vomiting, and pain), and 6 single symptom items (dyspnea, insomnia, appetite loss, constipation, diarrhea, and financial difficulties). The questionnaire includes 28 items with 4-point Likert type responses from 1-not at all to 4-very much to assess functioning and symptoms; 2 items with 7-point Likert scales (1= poor and 7= excellent) for global health and overall QoL. Scores are transformed to a 0 to 100 scale, with higher scores representing better GHS, better functioning, and more symptoms. Negative change from baseline values indicate deterioration in quality of life or functioning and positive values indicate improvement." (NCT02195479)
Timeframe: Baseline, Months 3, 6, 9, 12 and 18

,
InterventionUnits on a scale (Least Squares Mean)
Month 3Month 6Month 9Month 12Month 18
Daratumumab, Velcade, Melphalan and Prednisone (D-VMP)8.810.611.112.611.4
Velcade, Melphalan and Prednisone (VMP)9.410.511.91112.7

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Number of Participants by Severity With Acute Graft Versus Host Disease (GVHD) in the First 100 Days After HCT

"Cumulative incidence of acute GVHD was estimated using Kalbfleisch-Prentice method. Death is the competing risk event. SAS macro (bmacro252-Excel2007cin) available at St. Jude was used for analysis. Severity of GVHD and stage were determined using the Clinical Oncology Group (COG) Stem Cell Committee Consensus Guidelines for establishing organ stage and overall grade of acute GVHD. Participants are graded on a scale from I to IV, with I being mild and IV being severe.~Overall Clinical Grade (based on the highest stage obtained):~Grade 0: No stage 1-4 of any organ. Grade I: Stage 1-2 skin and no liver or gut involvement. Grade II: Stage 3 skin, or Stage I liver involvement, or Stage 1 gastrointestinal (GI).~Grade III: Stage 0-3 skin, with Stage 2-3 liver, or Stage 2-3 GI. Grade IV: Stage 4 skin, liver or GI involvement.~Due to early close of study, a small number of patients were enrolled. The number of patients who experienced acute GVHD is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
No Acute GVHDGrade IGrade IIGrade IIIGrade IV
Treatment80121

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Number of Participants With Secondary Graft Failure

"The cumulative incidence of secondary graft failure will be estimated using the Kalbfleisch-Prentice method. Deaths due to toxicity and relapse before day 100 are the competing events.~Secondary graft failure or graft rejection will be defined as no evidence of donor chimerism by umbilical cord blood (UCB) and/or haploidentical donor (<10%), or too few cells to perform adequate chimerism analysis, in research participants with prior neutrophil engraftment.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced secondary graft failure is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
Treatment0

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Number of Participants by Severity With Chronic Graft Versus Host Disease (GVHD) in the First 100 Days After HCT

"Cumulative incidence of acute and chronic GVHD was estimated using Kalbfleisch-Prentice method. Death is competing risk event. SAS macro (bmacro252-Excel2007cin) available St. Jude was used for such analysis. Severity of chronic GVHD was evaluated using National Institutes of Health (NIH) Consensus Global Severity Scoring. Mild is considered a better outcome with severe being the worst.~Criteria for grading chronic GVHD:~Mild: 1-2 organs/sites, maximum organ score of 1, and lung score of 1. Moderate: 3 or more organs/sites and maximum organ score of 1 and lung score of 1, OR at least 1 organ/site and maximum organ score of 2 and lung score of 1. Severe: At least 1 organ/site and maximum organ score of 3 and lung score of 2-3.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced chronic GVHD is provided." (NCT02199041)
Timeframe: 100 days after transplantation

InterventionParticipants (Count of Participants)
No Chronic GVHDMildModerateSevere
Treatment11001

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Number of Participants With Event-free Survival (EFS)

"The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided." (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment4

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Number of Participants With Malignant Relapse

"Relapse was evaluated using standard World Health Organization (WHO) criteria for each disease. The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Relapse defined as the recurrence of original disease. Death is the competing risk event. The analysis will be implemented using Statistical Analysis System (SAS) macro (bmacro252-Excel2007cin).~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who experienced malignant relapse is provided" (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment7

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Number of Participants With Neutrophil Engraftment

Neutrophil engraftment is defined as absolute neutrophil count (ANC) recovery of ≥ 0.5 x 10^9/L (500/mm^3) for three consecutive laboratory values obtained on different days (derived from either donor). Date of engraftment is the date of the first of the three consecutive laboratory values. The number of patients engrafted by day +42 post-transplant is provided. (NCT02199041)
Timeframe: Until day 42 post-transplant

InterventionParticipants (Count of Participants)
Treatment11

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Number of Participants With Overall Survival (OS)

"The Kaplan-Meier estimate of OS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) - date of hematopoietic cell transplantation (HCT) and all participants surviving after 1 year post-transplant will be considered as censored.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not die at 1 year post-transplant is provided." (NCT02199041)
Timeframe: One year after transplantation

InterventionParticipants (Count of Participants)
Treatment6

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Incidence and Severity of Acute GvHD

The cumulative incidence of acute GvHD will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The severity of acute GvHD. The number of participants with incidence by grade is given. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe. (NCT02259348)
Timeframe: 100 days post transplantation

Interventionparticipants (Number)
Grade IGrade IIGrade IIIGrade IV
Participants0031

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Incidence of Malignant Relapse

The estimate of cumulative incidence of relapse will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The number of participants with incidence of malignant relapse is given. Relapse was evaluated using standard WHO criteria for each disease. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Percentage of Participants Engrafted by Day 42 Post-transplant

To estimate engraftment by day +42 post-transplant in patients who receive CD45RA-depleted haploidentical donor progenitor cell transplantation following reduced intensity conditioning regimen that includes haploidentical NK cells. Engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventionpercentage of participants (Number)
Participants100

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Overall Survival (OS)

The Kaplan-Meier estimate of OS along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where OS = min (date of last follow-up, date of death) - date of transplant and all participants surviving at the time of analysis without events will be censored. The number of participants surviving to one-year post-transplantation is given. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Median Days to Absolute Neutrophil Count (ANC) Engraftment

ANC engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventiondays (Median)
Participants10

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Mean of Days to Absolute Neutrophil Count (ANC) Engraftment

ANC engraftment is defined as the first of 3 consecutive tests performed on different days of an ANC ≥ 500/mm^3 with evidence of donor cell engraftment. (NCT02259348)
Timeframe: Day 42 post transplantation

Interventiondays (Mean)
Participants10.3

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Event-free Survival (EFS)

The Kaplan-Meier estimate of event-free survival (EFS) along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given. (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
Participants2

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Incidence and Severity of Chronic GvHD

"The cumulative incidence of chronic GvHD will be estimated using Kalbfleisch-Prentice method. Death is the competing risk event. The severity of chronic GvHD will be described. Chronic GvHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with incidence by severity is given." (NCT02259348)
Timeframe: one year post transplantation

Interventionparticipants (Number)
MildModerateSevere
Participants000

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The Estimated Percentage of Patients Alive at 2 Years

(NCT02331368)
Timeframe: 2 Years

Interventionpercentage of patients (Number)
Anti-PD-1 (MK-3475)100

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The Estimated Percentage of Patients Alive Without Relapse or Progression at 2 Years

"Progression is defined as an increase of ≥ 25% from the lowest response value in any one or more of the following:~Serum M-component with an absolute increase ≥ 0.5 g/dL; and/or Urine M-component with an absolute increase ≥ 200 mg/24 hours; and/or Only in patients without measurable serum and urine M-protein levels: the difference between involved and uninvolved FLC levels (absolute increase must be >100mg/l) Bone marrow plasma cell percentage (absolute % must be ≥10% Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas Development of hypercalcemia (corrected serum calcium >11.5mg/100ml) that can be attributed solely to the plasma cell proliferative disorder" (NCT02331368)
Timeframe: 2 Years

Interventionpercentage of patients (Number)
Anti-PD-1 (MK-3475)83

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The Number of Patients That Achieve Complete Response

The primary efficacy endpoint is complete response rate. The complete response rate was estimated by the observed proportion of complete responders at day 180. Complete response (CR) was defined as negative immunofixation of serum and urine and disappearance of any soft tissue plasmacytomas, and <5% plasma cells in bone marrow and negative bone marrow flow cytometry. (NCT02331368)
Timeframe: 180 days post-transplant

InterventionParticipants (Count of Participants)
Anti-PD-1 (MK-3475)7

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Number of Patients With Grade 3-5 Toxicities Graded by the NCI CTCAE Version 4.0

Observed toxicities will be summarized in terms of type and severity. In accordance with the secondary study objectives, descriptive analyses on these data will be performed. (NCT02366663)
Timeframe: Day -21 to Day +100 post-HCT

,
InterventionParticipants (Count of Participants)
AnemiaFebrile neutropeniaHyperglycemiaHyponatremiaINR increasedLymphocyte count decreasedMucositis oralNeutrophil count decreasedObesityOral painPharyngeal mucositisPlatelet count decreasedSore ThroatWhite blood cell decreased
Arm I (ZBEAM)22111222100202
Arm II (BEAM)11000111111111

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Number of Patients With Complete or Partial Response at Day 30

Definition of disease status is based on the article of Revised Response Criteria for Malignant Lymphoma Response Definitions for Clinical Trials (Cheson et al, 2007). Tests used for evaluation of disease status would be physical examination, laboratory testing, bone marrow testing, bone marrow biopsy and aspirate, PET scan, and CT scans of neck, chest, abdomen and pelvis as indicated. (NCT02366663)
Timeframe: Day 0 to Day +30 post-HCT

InterventionParticipants (Count of Participants)
Arm I (ZBEAM)1
Arm II (BEAM)1

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Time to Neutrophil Engraftment

Time to neutrophil engraftment will be compared between treatment arms using a log-rank test, and the cumulative incidence curves will be estimated. (NCT02366663)
Timeframe: Day 0 to Day 100 post-HCT

Interventiondays (Median)
Arm I (ZBEAM)11
Arm II (BEAM)10

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Time to Platelet Engraftment

Time to platelet engraftment will be compared between treatment arms using a log-rank test, and the cumulative incidence curves will be estimated. (NCT02366663)
Timeframe: Day 0 to Day 100 post-HCT

Interventiondays (Median)
Arm I (ZBEAM)22.5
Arm II (BEAM)26

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Number of Participants With Sustained Cell Engraftment of Donor Cells

Sustained stem cell engraftment of donor cells will be evaluated by chimerism (FISH fluorescence in situ hybridization OR VNTR (Variable Number of Tandem Repeats), based on recipient/donor gender, at 30 days, 100 days, 6 months and 1 year following the use of reduced intensity conditioning. (NCT02435901)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Reduced Intensity Regimen29

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Event Free Survival; Number of Participants Who Survived at 2 Years

29 participants will be evaluated for Event Free Survival. (NCT02435901)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Expired Secondary to SepsisExpired Secondary to GVHDSurvived Participants
Reduced Intensity Regimen1226

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Percentage of Participants With Infections Post-randomization by Time Point

All Grade 2 and 3 infections are reported according to the BMT CTN MOP (Manual of Procedures) where a higher grade corresponds to more severe symptoms. The cumulative incidence of severe, life-threatening, or fatal infections (Grade 3), treating death as a competing event, are estimated at 6, 12 and 18 months post randomization using Aalen-Johansen estimators for each treatment group. Comparison of cumulative incidence between the two treatment arms was done using a Z test at fixed time points. (NCT02440464)
Timeframe: 6, 12 and 18 months post-randomization

,
Interventionpercentage of participants (Number)
6 Months Post-randomization12 Months Post-randomization18 Months Post-randomization
Ixazomib Maintenance4.84.84.8
Placebo0.00.00.2

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Percentage of Participants With Infections Post-randomization by Infection Type

All Grade 2 and 3 infections are reported according to the BMT CTN MOP (Manual of Procedures) where a higher grade corresponds to more severe symptoms. The number of participants with post-randomization infections in each treatment arm is described by severity and type of infection. (NCT02440464)
Timeframe: 2 years post-randomization

,
InterventionParticipants (Count of Participants)
Participants with InfectionsMaximum Severity: Grade 2Maximum Severity: Grade 3Participants with Bacterial InfectionParticipants with Viral InfectionParticipants with Fungal InfectionParticipants with Other Infection
Ixazomib Maintenance8623601
Placebo1110121110

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Percentage of Participants With Disease Progression

Disease progression was evaluated using the International Uniform Response Criteria. Participants in sCR/CR must meet at least one of the criteria for disease progression specified in the protocol for sCR/CR participants; those not in sCR/CR must meet at least one of the disease progression criteria specified in the protocol for those not in sCR/CR. The cumulative incidence of progression from randomization will be estimated for each treatment arm using the Aalen-Johansen estimator, with death in remission treated as a competing risk. Initiation of anti-myeloma therapy will be considered evidence of progression. Participants who were event-free at two years post-transplant are censored at that time. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance34.744.7
Placebo27.336.4

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Percentage of Participants With Chronic GVHD

Cumulative incidences of chronic GVHD were determined using the Aalen-Johansen estimator. Death prior to chronic GVHD is treated as the competing risk. Cumulative incidences are compared between treatment arms using Gray's test. Data of chronic GVHD were collected from providers and chart review according to the recommendations of the 2014 NIH Consensus Criteria. Eight organs are scored on a 0-3 scale to reflect degree of chronic GVHD involvement; 3 indicates the worst symptom. Liver and pulmonary function test results and use of systemic therapy for treatment of chronic GVHD are also recorded. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance68.668.6
Placebo63.663.6

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Functional Assessment of Cancer Therapy (FACT) - Bone Marrow Transplant (BMT) Total Score

The FACT-BMT version 4.0 instrument is comprised of the Functional Assessment of Cancer Therapy - General (FACT-G), which evaluates the health-related quality of life (HQL) of patients receiving treatment for cancer, and the BMT Concerns module, that addresses disease and treatment-related questions specific to bone marrow transplant. This scale goes from 0 to 196 where higher scores indicate better functioning. The FACT-BMT Total, which has all items in the FACT-G and BMT modules, was used as the outcome measure. This self-reported questionnaire was completed at transplant, randomization, 6 months following the start of maintenance therapy (which corresponds to 6 months post-randomization), and 24 months post-transplant. Comparisons between treatment groups were performed prior to maintenance, 6 months post-randomization and at 24 months after transplant. Only English and Spanish speaking patients were eligible to participate in the quality of life component of this trial. (NCT02440464)
Timeframe: Randomization, 6-months post-randomization, 24 months post-transplant

,
Interventionscore on a scale (Median)
Randomization6 Months Post-randomization24 Months Post-transplantChange from Randomization to 6 Months Post-randomizationChange from Randomization to 24 Months Post-transplant
Ixazomib Maintenance100.0121.4121.55.012.6
Placebo110.0114.0109.07.03.0

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Percentage of Participants With Acute GVHD (Grades III-IV)

Cumulative incidences of grade III-IV acute GVHD were determined using the Aalen-Johansen estimator. Death prior to acute GVHD is treated as the competing risk. Cumulative incidences are compared between treatment arms using Gray's test. Grading of acute GVHD was derived by consensus grading per BMT Clinical Trials Network (CTN) manual of procedures (MOP). The acute GVHD algorithm calculates the grade based on the organ (skin, GI and liver) stage and etiology/biopsy reported on the weekly GVHD form. Grade I aGVHD is defined as Skin stage of 1-2 and stage 0 for both GI and liver organs. Grade II aGVHD is stage 3 of skin, or stage 1 of GI, or stage 1 of liver. Grade III is stage 2-4 for GI, or stage 2-3 of liver. Grade IV is stage 4 of skin, or stage 4 of liver. Grade 4 is the worst outcome. (NCT02440464)
Timeframe: 100 days post-randomization

Interventionpercentage of participants (Number)
Ixazomib Maintenance9.5
Placebo0.0

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Medical Outcomes Study (MOS) - Short Form 36 (SF-36) Score

The MOS SF-36 instrument is a general assessment of health quality of life with eight components: Physical Functioning, Role Physical, Pain Index, General Health Perceptions, Vitality, Social Functioning, Role Emotional, and Mental Health Index. The scale is 0 to 100 where 0 is maximum disability and 100 is no disability, so the higher the score the more positive the outcome. The Physical Component Summary (PCS) and Mental Component Summary (MCS) were used as the outcome measures in summarizing the SF-36 data for this study. This self-reported questionnaire was completed at transplant, randomization, 6 months following the start of maintenance therapy (which corresponds to 6 months post-randomization), and 24 months post-transplant. Comparisons between treatment groups were performed prior to maintenance, 6 months post-randomization and at 24 months after transplant. Only English and Spanish speaking patients were eligible to participate in the quality of life component of this trial. (NCT02440464)
Timeframe: Randomization, 6 months post-randomization, 24 months post-transplant

,
Interventionscore on a scale (Median)
PCS: RandomizationPCS: 6 Months Post-randomizationPCS: 24 Months Post-transplantPCS: Change from Randomization to 6 Months Post-randomizationPCS: Change from Randomization to 24 Months Post-transplantMCS: RandomizationMCS: 6 Months Post-randomizationMCS: 24 Months Post-transplantMCS: Change from Randomization to 6 Months Post-randomizationMCS: Change from Randomization to 24 Months Post-transplant
Ixazomib Maintenance38.747.945.13.34.751.554.856.1-0.64.9
Placebo41.541.245.71.07.849.954.549.55.3-2.5

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Percentage of Participants With Response to Treatment

Response was assessed using the International Uniform Response Criteria. All disease classifications are relative to the patient's disease status prior to allogeneic transplant (i.e. study entry). Response to treatment after randomization (sCR, CR, VGPR, or PR) is summarized in each arm post-transplant at 18 months and 24 months post-transplant. These response data were summarized separately for patients in sCR/CR at the time of randomization vs. those who were not in sCR/CR at the time of randomization. (NCT02440464)
Timeframe: 18 months and 24 months post-transplant

InterventionParticipants (Count of Participants)
Response to Treatment at 18 Months Post-transplant for Participants in sCR/CR at Randomization72551676Response to Treatment at 18 Months Post-transplant for Participants in sCR/CR at Randomization72551677Response to Treatment at 18 Months Post-transplant for Participants Not in sCR/CR at Randomization72551676Response to Treatment at 18 Months Post-transplant for Participants Not in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants in sCR/CR at Randomization72551676Response to Treatment at 24 Months Post-transplant for Participants in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants Not in sCR/CR at Randomization72551677Response to Treatment at 24 Months Post-transplant for Participants Not in sCR/CR at Randomization72551676
Stringent Complete Response (sCR)Complete Response (CR)Very Good Partial Response (VGPR)Not EvaluablePartial Response (PR)Stable Disease (SD)Progressive Disease (PD)Died Before Evaluation
Ixazomib Maintenance3
Placebo3
Placebo2
Placebo0
Ixazomib Maintenance2
Placebo4
Ixazomib Maintenance4
Placebo1
Placebo6
Ixazomib Maintenance0
Ixazomib Maintenance1
Ixazomib Maintenance5

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Percentage of Participants With Best Response to Treatment After Randomization

Response was assessed using the International Uniform Response Criteria. Best response is the best of all the disease response status at each assessment time point after randomization. The order from best to worst is: Stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Stable Disease (SD), and Progressive Disease (PD). All disease classifications are relative to the patient's disease status prior to allogeneic transplant (i.e. study entry). This outcome was compared between treatment groups using all response data up to 2 years post-transplant. These response data were summarized separately for patients in sCR/CR at the time of randomization vs. those who were not in sCR/CR at the time of randomization. Within each group (in sCR/CR vs not in sCR/CR at randomization), best response to treatment was compared between treatment groups using a Fisher's Exact test instead of a chi-square test because of the small sample size. (NCT02440464)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
Participants in sCR/CR at Randomization72551676Participants in sCR/CR at Randomization72551677Participants Not in sCR/CR at Randomization72551677Participants Not in sCR/CR at Randomization72551676
Partial Response (PR)Stable Disease (SD)Progressive Disease (PD)Stringent Complete Response (sCR)Complete Response (CR)Very Good Partial Response (VGPR)
Ixazomib Maintenance6
Placebo5
Ixazomib Maintenance2
Ixazomib Maintenance0
Placebo0
Placebo1
Ixazomib Maintenance3
Placebo4
Placebo3
Ixazomib Maintenance4
Ixazomib Maintenance1

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Percentage of Participants With Toxicities Post-randomization by Toxicity Type

Toxicities are graded using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0. Higher grades correspond to worse symptoms with the minimum grade being a 0 and the maximum grade being a 5. Toxicities post-randomization are described for each treatment arm by the type of toxicity as well as peak overall grade. (NCT02440464)
Timeframe: 2 years post-randomization

,
InterventionParticipants (Count of Participants)
Maximum Toxicity Grade : 0 - 2Maximum Toxicity Grade : 3Maximum Toxicity Grade : 4Maximum Toxicity Grade : 5Abnormal Liver SymptomsDysgeusiaEncephalopathyGrade 3-5 Allergic ReactionGrade 3-5 AnaphylaxisGrade 3-5 Blood/Lymphatic ToxicityGrade 3-5 Cardiac ToxicityGrade 3-5 FatigueGrade 3-5 FeverGrade 3-5 GI ToxicityGrade 3-5 Hearing LossGrade 3-5 HemorrhageGrade 3-5 Hepatobiliary/PancreasGrade 3-5 HyperthyroidismGrade 3-5 HypothyroidismGrade 3-5 Metabolic ToxicityGrade 3-5 Musculoskeletal ToxicityGrade 3-5 Nervous System ToxicityGrade 3-5 Ocular ToxicityGrade 3-5 Renal ToxicityGrade 3-5 Respiratory ToxicityGrade 3-5 Skin/Subcutaneous Tissue ToxicityGrade 3-5 Vascular ToxicityHepatitisIntestinal ObstructionLiver FailureSevere Muscle Weakness/ParalysisSudden Vision LossTumor Lysis Syndrome
Ixazomib Maintenance8130031000563060010041400212010001
Placebo6141122010873070070163425853100100

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Percentage of Participants With Toxicities Post-randomization by Time Point

Toxicities are graded using NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 4.0. Higher grades correspond to worse symptoms with the minimum grade being a 0 and the maximum grade being a 5. The cumulative incidence of Grade ≥ 3 toxicity was estimated at 6, 12 and 18 months post randomization using Aalen-Johansen estimators for each treatment group. Death from a cause other than toxicity was treated as a competing risk. Comparison of cumulative incidence between the two treatment arms was done using a Z test at fixed time points. (NCT02440464)
Timeframe: 6, 12 and 18 months post-randomization

,
Interventionpercentage of participants (Number)
6 Months Post-randomization12 Months Post-randomization18 Months Post-randomization
Ixazomib Maintenance57.161.961.9
Placebo63.668.272.7

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Percentage of Participants With Progression-Free Survival

The primary endpoint compares progression-free survival as a time to event endpoint from randomization between patients randomized to ixazomib and placebo maintenance in high risk multiple myeloma. Participants are considered a failure of the primary endpoint if they die or suffer from disease progression or if they initiate non-protocol anti-myeloma therapy. Disease progression was evaluated using the International Uniform Response Criteria. Participants must meet one of the criteria for disease progression specified in the protocol. The time to this event is the time from randomization to progression, death, or initiation of non-protocol anti myeloma therapy whichever comes first. The Kaplan-Meier estimator was used to estimate progression-free survival during the 2 year post-transplant follow-up period. Participants who were event-free at two years post-transplant are censored at that time. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance65.355.3
Placebo72.759.1

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Percentage of Participants With Overall Survival (OS)

Overall survival (OS) is defined as freedom from death from any cause. OS post-randomization is estimated for each arm using the Kaplan-Meier estimator and compared between arms using the log rank test. Participants who are alive at two years post-transplant are censored at that time. Confidence intervals for values of 100% were not calculated and are shown as 100%. (NCT02440464)
Timeframe: 12 months and 21 months post-randomization

,
Interventionpercentage of participants (Number)
12 Months Post-randomization21 Months Post-randomization
Ixazomib Maintenance100.094.7
Placebo90.986.4

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Incidence of Toxicity, Defined According to National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0

Descriptive statistics on the number and percent toxicities will be calculated. (NCT02483000)
Timeframe: Up to 30 days after transplant

InterventionParticipants (Count of Participants)
Serious Adverse EventsOther (Not Including Serious) Adverse Events
Treatment (PRIT)22

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Dosimetry of Yttrium Y 90 DOTA-biotin

Assessed using OLINDA dosimetry software. The estimated dose to normal organs and tumor sites will be described based on the tumor to normal organ ratios derived from dosimetry estimates coupled with the absorbed dose to normal organs based on the administered activity of yttrium Y 90 DOTA-biotin. (NCT02483000)
Timeframe: Up to 7 days after infusion

InterventioncGy/mCi (Median)
LiverSpleenLungsKidneysBone MarrowBrain
Treatment (PRIT)2.53.730.22911.43.750.229

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Overall Survival

Overall survival will be estimated. (NCT02483000)
Timeframe: Up to 4 years

InterventionParticipants (Count of Participants)
Treatment (PRIT)2

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Overall Survival

duration of overall survival measured in days (NCT02489500)
Timeframe: 5 years

Interventiondays (Number)
Melphalan43
Melphalan + Bortezomib29

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Number of Participants With Hematologic Response

Hematologic response defined as: at least 50% improvement in the difference between involved and uninvolved free light chains (NCT02489500)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Melphalan0
Melphalan + Bortezomib0

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Toxicities

Number of serious adverse events per participant based on Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0 (NCT02489500)
Timeframe: 100 days

Interventionevents per participant (Mean)
Melphalan8.5
Melphalan + Bortezomib15

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Overall Survival at 6 Months Post-transplant

Number of participants alive at 6 months post-transplant (NCT02497404)
Timeframe: 6 months post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine35

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Overall Survival at 2 Years Post-Transplant

Number of participants alive at 2 years post-transplant (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine15

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Overall Survival at 1 Year Post-Transplant

Number of participants alive at 1 year post-transplant (NCT02497404)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine25

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High-Risk Extensive Chronic Graft-versus-Host-Disease

Number of patients who develop high-risk extensive chronic graft-versus-host disease. Extensive chronic GVHD is defined as generalized skin or multiple organ involvement. High risk chronic GVHD is defined as platelet count of less than 100k/microL (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine2

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Graft Failure

Number of patients who experience graft failure, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to <0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation. (NCT02497404)
Timeframe: 21 days post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine1

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Disease Free Survival at 6 Months Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 6 months post-transplant (NCT02497404)
Timeframe: 6 months post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine25

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Disease Free Survival at 2 Years Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 2 years post-transplant. (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine13

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Disease Free Survival at 1 Year Post-transplant

Number of participants without evidence of progression of underlying malignancy for which the transplant was performed, assessed at 1 year post-transplant. (NCT02497404)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine18

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Acute Graft-versus-Host Disease (GVHD)

Number of patients who develop acute graft-versus-host disease of any grade. (NCT02497404)
Timeframe: 2 years post-transplant

InterventionParticipants (Count of Participants)
5 Azacytidine13

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GVHD Incidence

The number of participants that developed graft-versus-host-disease before or at 100 days after transplant (NCT02581007)
Timeframe: 100 days

Interventionpercentage of patients (Number)
Grades II to IV acute GVHDGrades III to IV acute GVHDModerate chronic GVHDSevere chronic GVHD
Reduced-Intensity Mismatched Transplant2081612

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Relapse Incidence

Number of patients with disease reoccurrence at 1 and 2 years post-transplant (NCT02581007)
Timeframe: 2 years

Interventionpercentage of patients (Number)
1 yr post-transplant2 yr post-transplant
Reduced-Intensity Mismatched Transplant2436

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Overall Survival

Number of participants still alive 2 years after transplant (NCT02581007)
Timeframe: 2 years

Interventionpercentage of patients (Number)
1 yr post-transplant2 yr post-transplant
Reduced-Intensity Mismatched Transplant6856

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Graft Rejection

Measurement of donor cells vs. recipient cells (NCT02581007)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Reduced-Intensity Mismatched Transplant3

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Best Response of CR or CRi

Percentage of patients who achieved a best response of CRi (complete remission with incomplete blood count recovery) or CR (complete remission) (NCT02614560)
Timeframe: 9 weeks

InterventionParticipants (Count of Participants)
Pre-allo (Before Stem Cell Transplant)4

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1-year Survival Rate

1-year survival rate estimated using Kaplan-Meier methods The start date for overall survival is the day of alloSCT. (NCT02614560)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Pre-allo (Before Stem Cell Transplant)0
Post-allo (After Stem Cell Transplant)75

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Duration of Response

Defined as the time from the start of the first documented complete response (CR) or complete remission with incomplete blood count recovery (CRi) to the documentation of relapse or death due to any cause. (NCT02614560)
Timeframe: 9 weeks

Interventionweeks (Median)
Pre-allo (Before Stem Cell Transplant)6.57

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Overall Survival

Defined as the time from the day of alloSCT to the date of death due to any cause. (NCT02614560)
Timeframe: Approximately 96 weeks

Interventionweeks (Median)
Pre-allo (Before Stem Cell Transplant)11.07
Post-allo (After Stem Cell Transplant)NA

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Incidence of Adverse Events

AE: Adverse events; TEAE: Treatment-emergent adverse event. Grades are defined using National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE), v4.03. Grade 1 = mild, no intervention needed; Grade 2 = moderate, minimal intervention needed; Grade 3 = severe or medically significant, hospitalization is required; Grade 4 = life-threatening, urgent intervention needed; Grade 5 = death related to adverse event. An AE is considered serious if it was fatal, life threatening, required hospitalization, was disabling/incapacitating, resulted in a birth defect or congenital anomally, or was otherwise considered to be medically significant. (NCT02614560)
Timeframe: Approximately 1 year

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InterventionParticipants (Count of Participants)
TEAEsAEs Related to Vadatuximab TalirineAEs Leading to Treatment DiscontinuationGrade 3 or Higher TEAEsSerious AEs
Post-allo (After Stem Cell Transplant)87162
Pre-allo (Before Stem Cell Transplant)66065

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Incidence of Laboratory Abnormalities

Number (count) of participants that experienced a Grade 3 or higher laboratory toxicity (hematology and chemistry). Grades are defined using National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE), v4.03. Grade 1 = mild, no intervention needed; Grade 2 = moderate, minimal intervention needed; Grade 3 = severe or medically significant, hospitalization is required; Grade 4 = life-threatening, urgent intervention needed; Grade 5 = death related to adverse event. (NCT02614560)
Timeframe: Approximately 1 year

,
InterventionParticipants (Count of Participants)
Any chemistry testAmylase (iu/l)Alanine aminotransferase (iu/l)Aspartate aminotransferase (iu/l)Total bilirubin (mg/dl)Uric acid (mg/dl)Lipase (iu/l)Sodium (meq/l)Any hematology testHemoglobin (g/dl)Lymphocytes (x10^3/ul)Neutrophils (x10^3/ul)Platlets (x10^3/ul)White blood cell count (x10^3/ul)
Post-allo (After Stem Cell Transplant)30100011501445
Pre-allo (Before Stem Cell Transplant)43113100636666

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Rate of MRD Negativity

Rate of MRD (minimal residual disease) negativity at Day -1 (1 day prior to transplant) and Day 30 post-transplant (Part A only) (NCT02614560)
Timeframe: 30 days

InterventionPercentage of participants (Number)
Day -1 Rate of MRD NegativityDay 30 Rate of MRD Negativity
Pre-allo (Before Stem Cell Transplant)NA75

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Cmax (Pharmacokinetics)

Maximum observed plasma concentration. Derived from the individual raw data. (NCT02669615)
Timeframe: Day -2

Interventionng/ml (Median)
Melphalan HCl for Injection (Propylene Glycol Free)7380

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AUC0-t (Pharmacokinetics)

Area under the plasma concentration-time curve to the last measurable time point (AUC0-t) calculated by the trapezoidal rule. The area under the concentration-time curve (AUC) is calculated to determine the total drug exposure over a period of time. (NCT02669615)
Timeframe: Day -2

Interventionng*min/ml (Median)
Melphalan HCl for Injection (Propylene Glycol Free)533552

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Number of Subjects With Treatment-emergent Adverse Events

Toxicity was graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Treatment-emergent adverse events (TEAEs) were reported based on clinical laboratory tests, physical examinations, and vital signs from the time of enrollment through the end of the study period. DLTs were assessed from the first dose of study drug through the Cycle 2 administration of tremelimumab ± durvalumab post ASCT. DLTs were defined per protocol as lack of neutrophil/platelet engraftment by Day 30 post ASCT; Grade 5 toxicity (treatment-related death); Grade 4 non-hematological toxicity; Grade 3 non-hematological toxicity (with exclusions); isolated Grade 3 electrolyte abnormalities; or immune-related AEs resulting in discontinuation of treatment. (NCT02716805)
Timeframe: up to 14 months

InterventionParticipants (Count of Participants)
Any TEAEMaximum grade 2 TEAEMaximum grade 4 TEAEImmune-related TEAETremelimumab-related TEAEDurvalumab-related TEAESerious TEAETEAE Leading to Treatment DiscontinuationTEAE Meeting DLT Criteria
Cohort 1624151100

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Number of Subjects With Best Response According to International Myeloma Working Group (IMWG) Consensus Criteria

Response was evaluated by appropriate imaging and myeloma serum/urine tests at the start of Cycle 1 and end of study, with response categorized per IMWG consensus criteria, as follows: stringent complete response (sCR) - CR criteria + normal free light chain (FLC) ratio + no clonal cells in bone marrow; CR - negative immunofixation on serum/urine + no soft tissue plasmacytomas + <5% plasma cells in bone marrow; very good partial response (VGPR) - serum/urine M-protein detectable by immunofixation but not electrophoresis OR ≥90% reduction in serum M-protein + urine <100 mg/24h; PR - ≥50% and ≥90% (or <200 mg/24h) reduction of serum + urine M-protein, respectively; progressive disease - increase of ≥25% serum and/or urine M-component, increase of >10 mg/dL in involved and uninvolved FLC levels, bone marrow plasma cells ≥10%, new or larger lesions, or corrected serum calcium >11.5 mg/dL or 2.65 mmol/L [Rajkumar et al. Blood 2011;117:4691-5; Durie et al. Leukemia 2006;20:1467-73]. (NCT02716805)
Timeframe: Up to 14 months

InterventionParticipants (Count of Participants)
sCRVGPRProgressive disease
Cohort 1121

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Number of Participants With Minimal Residual Disease (MRD)

Minimal residual disease (MRD) is defined as the presence of malignant plasma cells detected by multicolor flow cytometry among patients who are in complete remission. Multichannel flow cytometry will be used to establish MRD based on the presence of malignant plasma cells that are CD45 (-/dim), CD38+, CD138+, CD19-, CD56+ kappa or lambda restricted. The number of patients with MRD negative (MRD-) are described using frequencies at pre-randomization and 9th cycle post-randomization and compared between the vaccine arm with the no-vaccine arms combined. (NCT02728102)
Timeframe: Pre-randomization, Post-randomization at Cycle 9

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InterventionParticipants (Count of Participants)
Pre-randomizationPost-randomization at Cycle 9
Lenalidomide With or Without GM-CSF3141
Lenalidomide, Vaccine, and GM-CSF3538

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Participants Response to Treatment

A participant's disease status is evaluated based on the International Uniform Response Criteria per protocol. Before disease progression (PD), all disease classifications including stringent complete response (sCR), complete response (CR), very good partial remission (VGPR), partial response (PR), stable disease (SD) are relative to participant's disease status at study entry. Disease status is 'Not Evaluable' when disease assessment is not required, or disease status is missing. (NCT02728102)
Timeframe: 6 months, 1 year, and 2 years post-transplant and at Cycles 3(Day 57), 6(Day 141), 9(Day 225), 12(Day 309), 15 (Day 393), 18(Day 477), 21(Day 561) and 24(Day 654) of maintenance therapy

InterventionParticipants (Count of Participants)
Disease Status at 6 Months72284442Disease Status at 6 Months72284444Disease Status at 6 Months72284445Disease Status at 12 Months72284444Disease Status at 12 Months72284442Disease Status at 12 Months72284445Disease Status at 24 Months72284442Disease Status at 24 Months72284444Disease Status at 24 Months72284445Disease Status at Cycle 3 (Day 57 Assessment)72284442Disease Status at Cycle 3 (Day 57 Assessment)72284445Disease Status at Cycle 3 (Day 57 Assessment)72284444Disease Status at Cycle 6 (Day 141 Assessment)72284445Disease Status at Cycle 6 (Day 141 Assessment)72284444Disease Status at Cycle 6 (Day 141 Assessment)72284442Disease Status at Cycle 9 (Day 225 Assessment)72284442Disease Status at Cycle 9 (Day 225 Assessment)72284445Disease Status at Cycle 9 (Day 225 Assessment)72284444Disease Status at Cycle 12 (Day 309 Assessment)72284442Disease Status at Cycle 12 (Day 309 Assessment)72284444Disease Status at Cycle 12 (Day 309 Assessment)72284445Disease Status at Cycle 15 (Day 393 Assessment)72284444Disease Status at Cycle 15 (Day 393 Assessment)72284442Disease Status at Cycle 15 (Day 393 Assessment)72284445Disease Status at Cycle 18 (Day 477 Assessment)72284445Disease Status at Cycle 18 (Day 477 Assessment)72284442Disease Status at Cycle 18 (Day 477 Assessment)72284444Disease Status at Cycle 21 (Day 561 Assessment)72284445Disease Status at Cycle 21 (Day 561 Assessment)72284444Disease Status at Cycle 21 (Day 561 Assessment)72284442Disease Status at Cycle 24 (Day 654 Assessment)72284445Disease Status at Cycle 24 (Day 654 Assessment)72284444Disease Status at Cycle 24 (Day 654 Assessment)72284442
Stringent Complete Response (sCR)Complete Response (CR)Very Good Partial Remission (VGPR)Stable Disease (SD)Not EvaluablePartial Response (PR)Progression (PD)Dead
Maintenance Lenalidomide8
Lenalidomide and GM-CSF12
Lenalidomide, Vaccine, and GM-CSF22
Maintenance Lenalidomide9
Maintenance Lenalidomide3
Maintenance Lenalidomide2
Maintenance Lenalidomide0
Maintenance Lenalidomide1
Maintenance Lenalidomide10
Lenalidomide, Vaccine, and GM-CSF8
Lenalidomide and GM-CSF9
Lenalidomide, Vaccine, and GM-CSF19
Lenalidomide and GM-CSF5
Maintenance Lenalidomide14
Lenalidomide, Vaccine, and GM-CSF35
Lenalidomide and GM-CSF15
Lenalidomide, Vaccine, and GM-CSF5
Lenalidomide and GM-CSF6
Lenalidomide, Vaccine, and GM-CSF13
Lenalidomide, Vaccine, and GM-CSF18
Lenalidomide, Vaccine, and GM-CSF28
Lenalidomide and GM-CSF14
Lenalidomide, Vaccine, and GM-CSF1
Lenalidomide, Vaccine, and GM-CSF16
Lenalidomide and GM-CSF8
Lenalidomide, Vaccine, and GM-CSF17
Lenalidomide and GM-CSF7
Maintenance Lenalidomide15
Lenalidomide, Vaccine, and GM-CSF24
Lenalidomide and GM-CSF13
Lenalidomide, Vaccine, and GM-CSF6
Lenalidomide and GM-CSF0
Lenalidomide, Vaccine, and GM-CSF2
Lenalidomide and GM-CSF2
Lenalidomide, Vaccine, and GM-CSF7
Lenalidomide and GM-CSF11
Lenalidomide, Vaccine, and GM-CSF4
Lenalidomide, Vaccine, and GM-CSF3
Lenalidomide, Vaccine, and GM-CSF0
Maintenance Lenalidomide4
Maintenance Lenalidomide13
Lenalidomide, Vaccine, and GM-CSF9
Maintenance Lenalidomide7
Lenalidomide, Vaccine, and GM-CSF20
Maintenance Lenalidomide11
Lenalidomide and GM-CSF17
Maintenance Lenalidomide6
Lenalidomide, Vaccine, and GM-CSF11
Lenalidomide, Vaccine, and GM-CSF15
Lenalidomide, Vaccine, and GM-CSF14
Lenalidomide and GM-CSF16
Lenalidomide and GM-CSF3
Lenalidomide and GM-CSF1
Lenalidomide, Vaccine, and GM-CSF12
Lenalidomide and GM-CSF4

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Percentage of Participants With Myeloma Progression of Vaccine and Non-vaccine Arms

The event for this endpoint is defined as disease progression from CR/sCR or progressive disease for participants not in CR/sCR, or initiation of off protocol antimyeloma therapy. The cumulative incidence of myeloma progression will be compared between the vaccine arm and the combined non-vaccine arms using Gray's test and treating death (without documentation of disease progression) as a competing risk. Participants alive without disease progression at last observation will be censored at the date of last contact. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF20.7
Lenalidomide With or Without GM-CSF11.8

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Percentage of Participants With Myeloma Progression in Pairwise Analysis

This is the pairwise comparison for percentage of participants with Myeloma Progression. The event for this endpoint is defined as disease progression from CR/sCR or progressive disease for participants not in CR/sCR, or initiation of off protocol antimyeloma therapy. The cumulative incidence of myeloma progression will be compared between the vaccine arm, lenalidomide/GM-CSF arm and lenalidomide alone arm using Gray's test and treating death (without documentation of disease progression) as a competing risk. Participants alive without disease progression at last observation will be censored at the date of last contact. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF20.7
Lenalidomide and GM-CSF8.7
Maintenance Lenalidomide15.1

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Percentage of Participants With Grade 2 and 3 Infections in Pairwise Analysis

This is the pairwise comparison for percentage of participants with Grade 2 and 3 infections. Grade 2 and 3 infections, as defined by the BMT CTN Technical MOP, are reported on the study. The cumulative incidence of infections post randomization, treating death as a competing risk, were compared between the vaccine arm, lenalidomide/GM-CSF arm and lenalidomide alone arm using the Gray's test. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF26.6
Lenalidomide and GM-CSF14.2
Maintenance Lenalidomide32.6

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Percentage of Participants With Grade 2 and 3 Infections

Grade 2 and 3 infections, as defined by the BMT CTN Technical MOP, are reported on the study. The cumulative incidence of infections post randomization, treating death as a competing risk, were compared between the vaccine and the combined non-vaccine groups using the Gray's test. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF26.6
Lenalidomide With or Without GM-CSF23.2

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Percentage of Participants With 1-year Response Rate of CR/sCR

The primary objective of this randomized trial is to compare the proportion of patients alive and in complete response (CR or sCR) at one year post transplant between patients receiving DC/myeloma vaccine/GM-CSF with lenalidomide maintenance therapy to those receiving lenalidomide maintenance therapy with or without GM-CSF. Complete Response (CR) is defined to require all the followings: Absence of the original monoclonal paraprotein in serum and urine by routine electrophoresis and by immunofixation; Less than 5% plasma cells in a bone marrow aspirate and also on trephine bone biopsy, if biopsy is performed; No increase in size or number of lytic bone lesions on radiological investigations; Disappearance of soft tissue plasmacytomas. Stringent Complete Response (sCR) is defined to require all the followings in addition to CR: Normal free light chain ratio (FLC); Absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence. (NCT02728102)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF52.9
Lenalidomide With or Without GM-CSF50.0

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Participants With Grade ≥ 3 Toxicities

Toxicities are evaluated using NCI CTCAE version 4.0 at pre-maintenance initiation and during maintenance therapy monthly for the first 4 cycles and then at cycles 6, 9, 15, 21, 24, which correspond to Day 1, 29, 57, 85, 141, 225, 393, 561, and 645 post maintenance initiation. The number of participants experiencing Grade ≥ 3 toxicity are displayed for the vaccine and non-vaccine arms separately. The proportion of participants experiencing Grade ≥ 3 toxicity are compared between the vaccine and non-vaccine arms combined. (NCT02728102)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Lenalidomide, Vaccine, and GM-CSF53
Lenalidomide With or Without GM-CSF49

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Percentage of Participants With Overall Survival in Pairwise Analysis

This is the pairwise comparison for percentage of participants with Overall Survival. Death from any cause is considered as events for this endpoint. The time to event is calculated as time from randomization to death, loss to follow-up or the end of the study, whichever comes first. Patients alive at the time of last observation are considered censored. The Kaplan-Meier estimator will be constructed for each treatment arm. Overall survival are compared between the vaccine arm, lenalidomide/GM-CSF arm and lenalidomide alone arm from time of randomization. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF97
Lenalidomide and GM-CSF100
Maintenance Lenalidomide96.9

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Percentage of Participants With Progression-Free Survival

Death or disease progression will be considered as events for this endpoint. The time to event will be calculated as time from randomization to disease progression, death, initiation of non-protocol anti-myeloma therapy, loss to follow-up or the end of the study, whichever comes first. The Kaplan-Meier estimator will be constructed for each treatment arm. Progression-free survival was compared between the vaccine and the combined non-vaccine arms using the log-rank test. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF79.3
Lenalidomide With or Without GM-CSF88.2

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Number of Grade ≥ 3 Toxicities

Toxicities are evaluated using NCI CTCAE version 4.0 at pre-maintenance initiation and during maintenance therapy monthly for the first 4 cycles and then at cycles 6, 9, 15, 21, 24, which correspond to Day 1, 29, 57, 85, 141, 225, 393, 561, and 645 post maintenance initiation. All Grade ≥ 3 toxicities will be tabulated for treatment arms. Toxicities are categorized by organ system according to the CTCAE. Toxicities that involve multiple questions per organ system are combined in one category. (NCT02728102)
Timeframe: 2 years

,,
InterventionToxicities (Number)
Auditory DisordersBlood and Lymphatic DisordersCardiovascular DisordersGI DisordersGeneral DisordersHepatobiliary/Pancreas DisordersImmune System DisordersInvestigationsMetabolism and Nutrition DisordersMusculoskeletal and Connective Tissue DisordersNervous System DisordersRenal DisordersRespiratory, Thoracic and Mediastinal DisordersSkin and Subcutaneous Tissue DisordersVascular DisordersAbnormal Liver Symptoms
Lenalidomide and GM-CSF040342100115015111
Lenalidomide, Vaccine, and GM-CSF1814155422711415460
Maintenance Lenalidomide03822210132122730

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Number of Grade 2 and 3 Infections

Grade 2 and 3 infections, as defined by the BMT CTN Technical MOP, occurring after randomization will be reported. The incidence of definite and probable viral, fungal and bacterial infections will be tabulated for each patient. (NCT02728102)
Timeframe: 2years

,,
Interventioninfections (Number)
BacterialViralFungalOther
Lenalidomide and GM-CSF3600
Lenalidomide, Vaccine, and GM-CSF101401
Maintenance Lenalidomide61401

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Percentage of Participants With Overall Survival

Death from any cause is considered as events for this endpoint. The time to event is calculated as time from randomization to death, loss to follow-up or the end of the study, whichever comes first. Patients alive at the time of last observation are considered censored. The Kaplan-Meier estimator will be constructed for each treatment arm. Overall survival are compared between the vaccine and the combined non-vaccine arms from time of randomization. (NCT02728102)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF97
Lenalidomide With or Without GM-CSF98.5

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Percentage of Participants With Progression-Free Survival in Pairwise Analysis

This is the pairwise comparison for percentage of participants with Progression-Free Survival. Death or disease progression will be considered as events for this endpoint. The time to event will be calculated as time from randomization to disease progression, death, initiation of non-protocol anti-myeloma therapy, loss to follow-up or the end of the study, whichever comes first. The Kaplan-Meier estimator will be constructed for each treatment arm. Progression-free survival was compared between the vaccine arm, lenalidomide/GM-CSF arm and lenalidomide alone arm. (NCT02728102)
Timeframe: 2 year

Interventionpercentage of participants (Number)
Lenalidomide, Vaccine, and GM-CSF79.3
Lenalidomide and GM-CSF91.3
Maintenance Lenalidomide84.9

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Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant

Overall survival among patients who did not receive early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days after induction day 1

InterventionPercentage of participants (Number)
Did Not Receive Allogeneic HCT on Study75

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Event-free Survival (EFS) Among Patients Who Received Early Transplant

Event-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. Events included death, relapse, and grade 3-4 acute graft vs host disease. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - GENDER

Patients who receive early transplant will be compared to those that don't using the Fisher's exact test for the categorical variables of gender. (NCT02756572)
Timeframe: From time of subject's study enrollment to time of subject's feasibility success assessment (i.e. when subject received transplant within 60 days or when it was determined subject would not proceed with transplant on study)

,
InterventionParticipants (Count of Participants)
FemaleMale
Did Not Receive Allogeneic HCT on Study1011
Received Allogeneic HCT on Study81

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Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 28

"Complete Remission (CR), defined as <5% blasts in bone marrow with hematologic recovery (ANC>1000/ul and platelets >100,000/ml).~CRi, defined as complete remission with insufficient hematologic recovery (ANC<1000/ul or platelets<100,000/ul).~CRp, defined as complete remission but platelets <100,000/ul. Minimal residual disease (MRD), defined as any detectable disease by flow cytometry, cytogenetics, FISH or PCR in a patient otherwise fulfilling remission criteria.~Morphologic leukemia-free state (MLFS), defined as <5% blasts in bone marrow with no hematologic recovery.~Relapse, defined as >5% blasts in bone marrow, flow cytometry, or manual differential; OR treatment for active relapsed disease." (NCT02756572)
Timeframe: Approximately 28 days after early allogeneic HCT

InterventionParticipants (Count of Participants)
CR with MRDCR without MRDCRi with MRDCRi without MRDMLFS with MRDMLFS without MRDRelapse
Received Allogeneic HCT on Study0701000

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Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Relapsed-free Survival 6 Months After Transplant

Success will be defined as observing a 40% of higher 6-month relapse-free survival among patients who received early transplant on study. (NCT02756572)
Timeframe: 6 months after early allogeneic HCT on study

InterventionParticipants (Count of Participants)
No relapse within 6 months post-HCT (feasibility success)Relapse within 6 months post-HCT (feasibility failure)
Received Early Allogeneic HCT on Study62

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Feasibility of Early Allogeneic Hematopoietic Cell Transplant Assessed by Enrollment and Incidence of Early Transplant

Success will be defined as enrollment of at least 30 patients with transplants occurring in 15 of these 30 (50%) within 60 days of enrollment or start of induction chemotherapy with G-CLAM, whichever is earlier. (NCT02756572)
Timeframe: Up to 60 days after start of chemotherapy

InterventionParticipants (Count of Participants)
Received allogeneic HCT on study within 60 days (feasibility success)Did not receive allogeneic HCT on study within 60 days (feasibility failure)
Treatment (Chemotherapy, HCT)921

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Demonstrate the Feasibility of Collecting Patient-reported Outcomes for Trial Participants

The amount of returned patient-reported outcome questionnaires will be summarized for each collection timepoint using percent collection from surviving patients for PRO timepoints. (NCT02756572)
Timeframe: Up to 12 months post-HCT

InterventionParticipants (Count of Participants)
Enrollment PROs returnedPost G-CLAM PROs returnedPre-HCT PROs returned6 months post-HCT PROs returned12 months post-HCT PROs returned
Treatment (Chemotherapy, HCT)2723843

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Relapse-free Survival (RFS) Among Patients Who Received Early Transplant

Relapse-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Relapse-free Survival (RFS) Among Patients Who Received Early Transplant

Relapse-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Overall Survival (OS) Among Patients Who Received Early Transplant.

Overall survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Overall Survival (OS) Among Patients Who Received Early Transplant.

Overall survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 100 days post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study91

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Overall Survival (OS) Among Patients Who Did Not Receive Early Transplant

Overall survival among patients who did not receive early allogeneic HCT on study as estimated with the Kaplan-Meier method. (NCT02756572)
Timeframe: Up to 6 months after induction day 1

InterventionPercentage of participants (Number)
Did Not Receive Allogeneic HCT on Study62

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Event-free Survival (EFS) Among Patients Who Received Early Transplant

Event-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. Events included death, relapse, and grade 3-4 acute graft vs host disease. (NCT02756572)
Timeframe: Up to 6 months post-transplant

InterventionPercentage of participants (Number)
Received Allogeneic HCT on Study82

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Response Assessments After Early Allogeneic Hematopoietic Cell Transplant, Day 84

"Complete Remission (CR), defined as <5% blasts in bone marrow with hematologic recovery (ANC>1000/ul and platelets >100,000/ml).~CRi, defined as complete remission with insufficient hematologic recovery (ANC<1000/ul or platelets<100,000/ul).~CRp, defined as complete remission but platelets <100,000/ul. Minimal residual disease (MRD), defined as any detectable disease by flow cytometry, cytogenetics, FISH or PCR in a patient otherwise fulfilling remission criteria.~Morphologic leukemia-free state (MLFS), defined as <5% blasts in bone marrow with no hematologic recovery.~Relapse, defined as >5% blasts in bone marrow, flow cytometry, or manual differential; OR treatment for active relapsed disease." (NCT02756572)
Timeframe: Approximately 84 days after early allogeneic HCT

InterventionParticipants (Count of Participants)
CR with MRDCR without MRDCRi with MRDCRi without MRDMLFS with MRDMLFS without MRDRelapse
Received Allogeneic HCT on Study0402002

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Acute Graft Versus Host Disease Among Patients Who Received Early Transplant

Acute graft versus host disease (graded II, III, or IV) among patients who received early allogeneic HCT on study. (NCT02756572)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Received Allogeneic HCT on Study0

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Demonstrate the Feasibility of Collecting Resource Utilization Data for Trial Participants

The amount of days of hospitalization (the major driver of costs within the first year) will be collected for resource utilization. (NCT02756572)
Timeframe: Within the first year of induction chemotherapy on study

Interventiondays (Median)
Treatment (Chemotherapy, HCT)49

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Factors That Distinguish Patients Who Received Early Transplant From Those Who Did Not - AGE

Patients who receive early transplant will be compared to those that don't using the Wilcoxon rank sum test for the quantitative variable of age. (NCT02756572)
Timeframe: From time of subject's study enrollment to time of subject's feasibility success assessment (i.e. when subject received transplant within 60 days or when it was determined subject would not proceed with transplant on study)

Interventionyears (Median)
Received Allogeneic HCT on Study55
Did Not Receive Allogeneic HCT on Study57

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Number of Patients With 2-year Relapse Risk

Hypothesis is that following lymphodepleting chemotherapy and pembrolizumab, the 2-year relapse risk will decrease to ≤35% (NCT02771197)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Lymphodepletion Plus Pembrolizumab10

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Overall Survival (OS)

Rate of participants' survival at time of evaluation. (NCT02780609)
Timeframe: at 24 months

Interventionpercent (Number)
Phase 1 Dose Level 3 and Phase 2: Selinexor Plus HDM HCT95.2

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Phase 1 and Phase 2 Percentage of Participants Treated at Dose Level 3/RP2D With Progression Free Survival (PFS)

Progression Free Survival defined as the time from start of treatment to the time of progression or death. (NCT02780609)
Timeframe: at 24 months

Interventionpercent of participants (Number)
Phase 1 Dose Level 3 and Phase 2: Selinexor Plus HDM HCT66.7

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Rate of Minimal Residual Disease (MRD)

Rate of participants who did not have Minimal Residual Disease (MRD) as assessed by flow cytometry. (NCT02780609)
Timeframe: 3 months post HCT

Interventionpercentage of participants (Number)
Phase 1 Level 1: Selinexor Plus HDM HCT33.3
Phase 1 Level 2: Selinexor Plus HDM HCT100
Phase 1 Level 3: Selinexor Plus HDM HCT16.7
Phase 2: Selinexor Plus HDM HCT33.3

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Complete Response (CR)

"Complete response (CR) conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.~tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow.~Complete Response conversion rate. CR: Negative immunofixation of serum and urine, disappearance of any soft tissue plasmacytomas, and ≤ 5% plasma cells in bone marrow." (NCT02780609)
Timeframe: 3 months post HCT

Interventionpercentage of participants with CR (Number)
Phase 1 Level 1: Selinexor Plus HDM HCT0
Phase 1 Level 2: Selinexor Plus HDM HCT33.3
Phase 1 Level 3: Selinexor Plus HDM HCT16.6
Phase 2: Selinexor Plus HDM HCT10

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Percentage of Participants Who Achieve a Timely Engraftment

Timely engraftment is defined as a persistent an absolute neutrophil count (ANC) of at least 500 cells/mm3 and a platelet count of at least 20,000 cells/mm3 for at least 3 days (NCT02797470)
Timeframe: 1 month post-transplant

Interventionpercentage of participants who achieve a (Mean)
Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:0 Ratio60
Treatment (Anti-HIV Gene Transduced CD34+ Cells): 1:1 Ratio75
Treatment (Anti-HIV Gene Transduced CD34+ Cells): 5:1 Ratio100

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Overall Response Rate (ORR)

The overall response rate (ORR) will be estimated as the percentage of patients who achieve sCR, CR, VGPR, or PR as their best response as assessed by the investigator. Response assessed by IMWG (International myeloma working group) criteria sCR-stringent complete response: CR plus Normal FLC (free light chain) ratio and absence of clonal cells in BM CR-complete response: Negative immunofixation in serum/urine; Disappearance of soft tissue plasmacytomas; <5% plasma cells in BM; If only FLC disease, normal FLC ratio (0.26-1.65) VGPR-very good partial response: Serum/urine M-protein detectable by immunofixation but not electrophoresis or ≥90% reduction in serum M-protein and urine M-protein <100 mg/24 h; If only FLC disease, >90% decrease in the difference between involved and uninvolved FLC levels PR-partial response: 50% reduction of serum M-protein and soft tissue plasmacytomas, ≥90% reduction in urinary M-protein or to <200 mg/24 h; other special cases if M-protein unmeasurable (NCT02963493)
Timeframe: Patients were followed until documented progression, unacceptable toxicity, patient/physician decision to withdraw or date of death, whichever came first. Longest time to response in study recorded as 15.3 months. Longest time on treatment 35 months.

InterventionParticipants (Count of Participants)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set53
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease14
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease35

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Functional Status and Well-being: EQ-5D-3L

"Change from baseline in Patient Reported Outcome questionnaire EQ-5D-3L. The EQ-5D-3L questionnaire converts 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression of patient-reported, current-day health status into a health utility score. For the EQ-5D-3L questionnaire, each dimension is scored on an ordinal scale with 3 available levels of response and scores ranging from 1 to 3, no problems, some problems, and extreme problems, respectively. The EQ VAS scores rates health today with anchors ranging from 0 (worst imaginable health state) to 100 (best imaginable health state). No imputation of missing items or composite scores were done. The scores for the 5 dimensions are used to compute a single utility score ranging from zero (0.0) to 1 (1.0) representing the general health status of the individual." (NCT02963493)
Timeframe: To be assessed prior to dosing at Cycle 1, 2, 4, 6, 8, and End of Treatment. 23 patients were ongoing for QoL assessments at data cutoff. QoL was added in Protocol Amendment 4 beginning in Oct. 2018.

,
InterventionChange in composite scale score (Mean)
Baseline valueCycle 2 Day 1 Change from baselineCycle 4 Day 1 Change from baselineCycle 6 Day 1 Change from baselineCycle 8 Day 1 Change from baselineEnd of Treatment Change from baseline
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set0.6748-0.0228-0.0169-0.0786-0.1461-0.0014
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease0.6803-0.05020.0038-0.1486-0.21740.0124

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Functional Status and Well-being: EORTC QLQ-C30

Change from baseline in Patient Reported Outcome questionnaire EORTC QLQ-C30. The EORTC QLQ-C30 includes 30 items resulting in 5 functional scales, 1 Global Health Status scale, 3 symptom scales, and 6 single items. The recall period is 1 week (the past week). The scales are transformed to a 0 (worst) to 100 (best) scale. The QLQ-C30 summary score is calculated as the mean of the combined 13 QLQ-C30 scale and item scores (excluding global QoL and financial impact), with a higher score indicating a better HRQoL. If at least 50% of the items from the scale had been answered, the missing items were assumed to have values equal to the average of those items which were present for that respondent. (NCT02963493)
Timeframe: To be assessed prior to dosing at Cycle 1, 2, 4, 6, 8, and End of Treatment. 23 patients were ongoing for QoL assessments at data cutoff. QoL was added in Protocol Amendment 4 beginning in Oct. 2018.

,
Interventionunits on a scale (Mean)
Baseline valueCycle 2 Day 1 Change from baselineCycle 4 Day 1 Change from baselineCycle 6 Day 1 Change from baselineCycle 8 Day 1 Change from baseline
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set75.50.2-2.6-3.3-7.5
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease74.974.876.973.271.3

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Time to Response

Duration from start of treatment to the first occurrence of a confirmed response of PR or better as assessed by the investigator. See definitions of response in Primary Outcome (ORR). (NCT02963493)
Timeframe: From start of treatment to first confirmed response. Longest time to response in study recorded as 15.3 months.

Interventionmonths (Median)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set2.1
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease2.1

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Duration of Response

Time from first response to progression based on investigator assessment. See definitions of response and progression in ORR and PFS outcomes. (NCT02963493)
Timeframe: From date of response until the date of first documented progression or date of death from any cause, whichever came first. Longest time of response recorded as 36.2 months at study end.

Interventionmonths (Median)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set6.90
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease5.49
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease7.46

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Clinical Benefit Rate

The clinical benefit rate (CBR) will be estimated as the percentage of patients who achieve sCR, CR, VGPR, PR, or MR as their best response as assessed by the investigator. See Primary Outcome (ORR) for definitions of response categories. (NCT02963493)
Timeframe: Patients were followed until documented progression, unacceptable toxicity, patient/physician decision to withdraw or date of death, whichever came first. Longest time on study treatment recorded as 35.0 months at study end.

InterventionParticipants (Count of Participants)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set72
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease17
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease48

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Overall Survival

Time from start of treatment to death (NCT02963493)
Timeframe: From date of first dose of study medication until the date of death from any cause, assessed up to 24 months after study drug discontinuation.

Interventionmonths (Median)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set11.79
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease6.44
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease10.12

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Progression Free Survival (PFS)

Time from start of treatment to either progression or death, whichever comes first as assessed by the investigator using IMWG criteria. Progression of disease is defined by an increase of 25% from the lowest response for either of Serum M-component (absolute increase of ≥ 0.5 g/dL) or Urine M-component (absolute increase of ≥200 mg/ 24h); In patients without measurable M-protein a 25% increase in the difference between involved and uninvolved FLC (free light chain) levels (absolute increase must be >10 mg/dL); If unmeasurable FLC levels, a 25% increase in bone marrow plasma cell percentage (absolute percentage must be >10%); New bone or soft tissue plasmacytomas or definite increase in existing ones; Development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to the plasma cell proliferative disorder (NCT02963493)
Timeframe: Patients were followed until documented progression, unacceptable toxicity, patient/physician decision to withdraw or date of death, whichever came first. Longest follow-up time for PFS recorded as 37.2 months at study end.

Interventionmonths (Median)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set4.27
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Extramedullary Disease2.89
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease3.94

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Time to Progression

Duration from start of treatment to first evidence of disease progression as assessed by the investigator. See definitions of response and progression in ORR and PFS outcomes. (NCT02963493)
Timeframe: From start of treatment to first evidence of disease progression or date of death from any cause, whichever came first. Longest time to progression recorded was 37.2 months at study end.

Interventionmonths (Median)
Melphalan Flufenamide (Melflufen) + Dexamethasone: Full Analysis Set4.4
Melphalan Flufenamide (Melflufen) + Dexamethasone: Patients With Triple Class Refractory Disease4.11

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Number of Participants With Cardiac Dysfunction (New Arrhythmia)

To determine the safety profile of propylene glycol-free melphalan hydrochloride in the conditioning regimen prior to autologous stem cell transplantation in AL amyloidosis patients, including adverse events related to cardiac dysfunction (new arrhythmia). (NCT02994784)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Evomela5

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Neutrophil Engraftment

time to neutrophil engraftment (NCT02994784)
Timeframe: 3 weeks

Interventiondays (Median)
Evomela10

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Hematologic Overall Response Rate

Number of patients with response based on Gertz, Palladini criteria (below) Complete response (CR): Normal serum free light chain ratio,Negative serum and urine immunofixation electrophoresis Very good partial response (VGPR): Difference in serum free light chains less than 40 mg/L Partial Response (PR): >50% Reduction in the difference in serum free light chains Stable Disease (SD): Meets neither criteria for CR, VGPR, PR or PD (NCT02994784)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
Evomela22

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Platelet Engraftment

Assess time to platelet engraftment (NCT02994784)
Timeframe: 100 days

Interventiondays (Median)
Evomela17

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Participants With Peri-transplant Hospitalizations

Number of participants with peri-transplant hospitalizations (NCT02994784)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Evomela23

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Organ Response

"Number of patients with organ response based on Gertz criteria (below)~Kidney: 50% reduction in 24-hour urine protein excretion in the absence of progressive renal insufficiency (defined as a 25% increase in serum creatinine, as long as that is > to an absolute increase of 0.5 mg/dL). In the case of nephrotic syndrome: a decrease in proteinuria to < 1g/24h and an improvement in one of 2 extrarenal features - normalization of serum albumin or resolution of edema and/or discontinuation of diuretics in response to improvement in edema.~Heart: ≥ 2 mm reduction in the interventricular septal (IVS) thickness by echocardiogram, improvement of ejection fraction by ≥ 20% (echocardiogram must be performed at the same institution), or decrease in 2 NYHA classes without increase in diuretic need." (NCT02994784)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Evomela13

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Number of Participants With Renal Dysfunction

To determine the safety profile of propylene glycol-free melphalan hydrochloride in the conditioning regimen prior to autologous stem cell transplantation in AL amyloidosis patients, including adverse events related to renal dysfunction (was acute renal failure is defined as either a >/=1 mg/dL increase in serum creatinine or a doubling of serum creatinine to >/=1.5 mg/dL for at least 2 days.). (NCT02994784)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Evomela2

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Number of Participants Who Survived

Number of Participants Who Survived at day 180. (NCT03019640)
Timeframe: From the time of transplant, assessed up to day 180

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy, NK Infusion, Stem Cell Transplant)16

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Overall Survival

Number of participants alive 1 year post transplant. (NCT03096782)
Timeframe: Up to 12 months after transplant

InterventionParticipants (Count of Participants)
Chemotherapy Plus Cord Blood Transplant4

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Disease-free Survival

Number of participants that were in remission post transplant. (NCT03096782)
Timeframe: Up to12 months

InterventionParticipants (Count of Participants)
Complete Remission at 30 daysComplete Remission at 12 months
Chemotherapy Plus Cord Blood Transplant64

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Time to Engraftment

Number of days from transplant when participants achieved engraftment measure by ANC of 0.5 for three consecutive days. (NCT03096782)
Timeframe: Up to 12 months after transplant

InterventionParticipants (Count of Participants)
Twenty Two DaysTwenty Nine DaysThirty DaysNine DaysThirty Two Days
Chemotherapy Plus Cord Blood Transplant21111

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Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year

Estimates will be calculated using the Kaplan-Meier method, Greenwood formula will be used to calculate standard error (SE), and log-log transformation method will be used to construct 95% confidence intervals. Graft versus host disease (GVHD, acute and chronic), disease status and vital status will be monitored per clinical standard operating procedure. For this endpoint failure is defined as the first occurrence of grade 3 or 4 acute GVHD, or moderate/severe chronic GVHD, or disease relapse (for patients in complete remission at the start of conditioning) or disease progression (for patients with active disease at the start of conditioning) or death (from any cause). Patients not experiencing any of these will be censored at his/her date of last contact. (NCT03128359)
Timeframe: From stem cell infusion to grade 3-4 acute graft versus host disease (GVHD), moderate-severe chronic GVHD, relapse, progression or death (from any cause), whichever occurs first, assessed for up to 1 year.

Interventionpercentage of survival probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)53
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)84

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Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading

Acute graft versus host disease is graded according to the 1994 Keystone Consensus Grading. aGVHD grade was evaluated from day 0 through 100 days post-transplant. The first day of acute GVHD onset at grades 2-4 was used to calculate the cumulative incidence. Relapse/death prior to onset was considered competing events. (NCT03128359)
Timeframe: Up to 100 days post-stem cell infusion

Interventionpercentage of probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)47
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)53

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Overall Survival (OS) at 1 Year

Estimates was calculated using the Kaplan-Meier method, Greenwood formula was used to calculate SE, and log-log transformation method was used to construct 95% confidence intervals. Each patient's vital status was monitored per clinical standard operating procedure. For this endpoint failure is defined as death (from any cause). Patients not experiencing a death event was censored at his/her date of last contact. (NCT03128359)
Timeframe: From start of transplant to death, or last follow up, whichever occurs first, assessed for up to 1 year.

Interventionpercentage of probability (Number)
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis)68
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis)100

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Overall Response Rate (ORR)

ORR defined as the proportion of patients for whom the best overall confirmed response is stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR), as assessed by IRC. (NCT03151811)
Timeframe: From randomization until best response achieved before confirmed progression, or if no progression up to 24 months after end of treatment. Median time to best response was 2.1 and 2.0 months for Arm A and B, respectively.

InterventionParticipants (Count of Participants)
Arm A: Melflufen+Dexamethasone80
Arm B: Pomalidomide+Dexamethasone67

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Progression Free Survival (PFS)

Progression Free Survival defined as the duration in months from randomization until first evidence of confirmed disease progression, as assessed by the Independent Review Committee (IRC) according to the International Myeloma Working Group Uniform Response Criteria (IMWG-URC) (NCT03151811)
Timeframe: From randomization to time of progression, or, if no progression, 24 months after end of treatment

Interventionmonths (Median)
Arm A: Melflufen+Dexamethasone6.83
Arm B: Pomalidomide+Dexamethasone4.93

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Safety and Tolerability: Number of Patients With Treatment-emergent Adverse Events, Including Clinical Laboratory and Vital Signs Abnormalities, as Assessed by CTCAE v4.0

Number of patients with treatment-emergent adverse events, including clinical laboratory and vital signs abnormalities, as assessed by CTCAE v4.0 will be presented. No formal statistical analysis will be performed for safety endpoints. (NCT03151811)
Timeframe: From start of dosing until 30 days after the last dose of study treatment, the median time frame for study treatment was 25.1 and 22.1 months for Arm A and B, respectively.

InterventionParticipants (Count of Participants)
Arm A: Melflufen+Dexamethasone226
Arm B: Pomalidomide+Dexamethasone241

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Duration of Response (DOR)

DOR defined as the duration in months from first documentation of a confirmed response to first evidence of confirmed disease progression or death due to any cause. (NCT03151811)
Timeframe: From first evidence of response until confirmed progression, or if no progression, 24 months after end of treatment

Interventionmonths (Median)
Arm A: Melflufen+Dexamethasone11.17
Arm B: Pomalidomide+Dexamethasone11.07

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Percentage of Participants With Anti-Daratumumab Antibodies

Percentage of participants with antibodies to daratumumab were reported. (NCT03412565)
Timeframe: For D-VRD Arm: Baseline up to 2 years 3 months; For D-VMP, D-Rd and D-Kd Arms: Baseline up to 2 years 7 months

Interventionpercentage of participants (Number)
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)0
D + Bortezomib + Melphalan + Prednisone (D-VMP)0
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)0
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)0

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Maximum Observed Serum Concentration (Cmax) of Daratumumab

Cmax was defined as maximum serum concentration observed following daratumumab administration. Each cycle for: D-VRd cohort is of 21 days, D-VMP cohort is of 42 days and D-Rd and D-Kd cohorts is of 28 days. Each cohort have a treatment period of 84 days. (NCT03412565)
Timeframe: D-VRd: Day 4 of Cycles 1 and 4 and post treatment at Week 8; D-VMP: Day 4 of Cycles 1 and 2 and post treatment at Week 8; D-Rd and D-Kd: Day 4 of Cycles 1 and 3 and post treatment at Week 8

Interventionmicrograms per milliliter (mcg/mL) (Mean)
Cycle 1 Day 4Cycle 4 Day 4Post-treatment Phase Week 8
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)100746263

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D-VMP, D-Rd, and D-Kd Cohorts: Overall Response Rate (ORR)

ORR was defined as the percentage of participants who achieved partial response (PR) or better according to international myeloma working group (IMWG) criteria. IMWG criteria for PR: greater than or equal to (>=) 50 percent (%) reduction of serum M-protein and reduction in 24-hour urinary M-protein by >=90% or to less than (<) 200 milligrams (mg) per 24 hours, If the serum and urine M-protein are not measurable, a decrease of >=50% in the difference between involved and uninvolved free light chain (FLC) levels is required in place of the M-protein criteria, If serum and urine M-protein are not measurable, and serum free light assay is also not measurable, >=50% reduction in bone marrow plasma cells (PCs) is required in place of M-protein, provided baseline bone marrow plasma cell percentage was >=30%. In addition to the above criteria, if present at baseline, a >=50% reduction in the size of soft tissue plasmacytomas is also required. (NCT03412565)
Timeframe: Up to 2 years 3 months

Interventionpercentage of participants (Number)
D + Bortezomib + Melphalan + Prednisone (D-VMP)88.1
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)90.8
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)84.8

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Maximum Observed Serum Concentration (Cmax) of Daratumumab

Cmax was defined as maximum serum concentration observed following daratumumab administration. Each cycle for: D-VRd cohort is of 21 days, D-VMP cohort is of 42 days and D-Rd and D-Kd cohorts is of 28 days. Each cohort have a treatment period of 84 days. (NCT03412565)
Timeframe: D-VRd: Day 4 of Cycles 1 and 4 and post treatment at Week 8; D-VMP: Day 4 of Cycles 1 and 2 and post treatment at Week 8; D-Rd and D-Kd: Day 4 of Cycles 1 and 3 and post treatment at Week 8

Interventionmicrograms per milliliter (mcg/mL) (Mean)
Cycle 1 Day 4Cycle 3 Day 4Post-treatment Phase Week 8
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)13786949.3

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Maximum Observed Serum Concentration (Cmax) of Daratumumab

Cmax was defined as maximum serum concentration observed following daratumumab administration. Each cycle for: D-VRd cohort is of 21 days, D-VMP cohort is of 42 days and D-Rd and D-Kd cohorts is of 28 days. Each cohort have a treatment period of 84 days. (NCT03412565)
Timeframe: D-VRd: Day 4 of Cycles 1 and 4 and post treatment at Week 8; D-VMP: Day 4 of Cycles 1 and 2 and post treatment at Week 8; D-Rd and D-Kd: Day 4 of Cycles 1 and 3 and post treatment at Week 8

Interventionmicrograms per milliliter (mcg/mL) (Mean)
Cycle 1 Day 4Cycle 2 Day 4Post-treatment Phase Week 8
D + Bortezomib + Melphalan + Prednisone (D-VMP)98.6612162

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D-VRd Cohort: Overall Response Rate (ORR)

ORR was defined as the percentage of participants who achieved a PR or better, IMWG criteria, during the study or during follow up. IMWG criteria for PR >= 50% reduction of serum M-protein and reduction in 24 hour urinary M-protein by >=90% or to <200 mg/24 hours, if the serum and urine M-protein are not measurable, a decrease of >=50% in the difference between involved and uninvolved FLC levels is required in place of the M-protein criteria, If serum and urine M-protein are not measurable, and serum free light assay is also not measurable, >=50% reduction in bone marrow PCs is required in place of M-protein, provided baseline bone marrow plasma cell percentage was >=30%, in addition to the above criteria, if present at baseline, a >=50% reduction in the size of soft tissue plasmacytomas is also required. (NCT03412565)
Timeframe: Up to 2 years and 3 months

Interventionpercentage of participants (Number)
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)97.0

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D-VRd Cohort: Percentage of Participants With Very Good Partial Response (VGPR) or Better Response

VGPR or better rate was defined as the percentage of participants who achieved VGPR or complete response (CR) (including stringent complete response [sCR]) according to the IMWG criteria during or after the study treatment. VGPR: Serum and urine component detectable by immunofixation but not on electrophoresis, or >= 90% reduction in serum M-protein plus urine M-protein level <100 mg/24 hour; CR: negative immunofixation on the serum and urine, Disappearance of any soft tissue plasmacytomas and <5% PCs in bone marrow; sCR: CR in addition to having a normal FLC ratio and an absence of clonal cells in bone marrow by immunohistochemistry, immunofluorescence, 2-4 color flow cytometry. (NCT03412565)
Timeframe: Up to 2 years and 3 months

Interventionpercentage of participants (Number)
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)71.6

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D-VMP, D-Rd and D-Kd Cohorts: Duration of Response (DOR)

DOR was defined as the time from the date of initial documented response (PR or better response) to the date of first documented evidence of progressive disease (PD) or death due to PD. PD is defined as an increase of 25% from the lowest response value in one of the following: serum and urine M-component (absolute increase must be >=0.5 gram per deciliter [g/dL] and >=200 mg/24 hours respectively); only in participants without measurable serum and urine M-protein levels the difference between involved and uninvolved FLC levels (absolute increase must be >10 mg/dL); definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas; development of hypercalcemia (corrected serum calcium >11.5 mg/dL) that can be attributed solely to PC proliferative disorder. (NCT03412565)
Timeframe: From baseline up to 2 years 7 months

Interventionmonths (Median)
D + Bortezomib + Melphalan + Prednisone (D-VMP)NA
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)NA
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)NA

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D-VMP, D-Rd, and D-Kd Cohorts: Percentage of Participants With VGPR or Better Response

VGPR or better rate was defined as the percentage of participants who achieved VGPR or CR (including sCR) according to the IMWG criteria during or after the study treatment. VGPR: Serum and urine component detectable by immunofixation but not on electrophoresis, or >= 90% reduction in serum M-protein plus urine M-protein level <100 mg per 24 hour; CR: negative immunofixation on the serum and urine, Disappearance of any soft tissue plasmacytomas and <5% PCs in bone marrow; sCR: CR in addition to having a normal FLC ratio and an absence of clonal cells in bone marrow by immunohistochemistry, immunofluorescence, 2-4 color flow cytometry. (NCT03412565)
Timeframe: From baseline up to 2 years 7 months

Interventionpercentage of participants (Number)
D + Bortezomib + Melphalan + Prednisone (D-VMP)77.6
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)80.0
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)77.3

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D-VMP, D-Rd, and D-Kd Cohorts: Percentage of Participants With Minimal Residual Disease (MRD) Negative Rate

MRD negativity rate was defined as the percentage of participants who were considered MRD negative after MRD testing at any timepoint after the first dose by bone marrow aspirate. MRD negativity rate was assessed by next-generation sequencing at a threshold of <10^5. (NCT03412565)
Timeframe: Up to 2 years and 3 months

Interventionpercentage of participants (Number)
D + Bortezomib + Melphalan + Prednisone (D-VMP)25.4
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)21.5
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)27.3

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Maximum Observed Serum Concentration (Cmax) of Daratumumab

Cmax was defined as maximum serum concentration observed following daratumumab administration. Each cycle for: D-VRd cohort is of 21 days, D-VMP cohort is of 42 days and D-Rd and D-Kd cohorts is of 28 days. Each cohort have a treatment period of 84 days. (NCT03412565)
Timeframe: D-VRd: Day 4 of Cycles 1 and 4 and post treatment at Week 8; D-VMP: Day 4 of Cycles 1 and 2 and post treatment at Week 8; D-Rd and D-Kd: Day 4 of Cycles 1 and 3 and post treatment at Week 8

Interventionmicrograms per milliliter (mcg/mL) (Mean)
Cycle 1 Day 4Cycle 3 Day 4
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)108648

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Percentage of Participants With CR or Better Response

CR or better rate was defined as the percentage of participants with a CR or better response (that is, CR and sCR) as per IMWG criteria. CR: as negative immunofixation on the serum and urine and disappearance of soft tissue plasmacytomas and less than (<) 5 percent plasma cells in bone marrow; sCR: CR plus normal FLC ratio and absence of clonal PCs by immunohistochemistry, immunofluorescence or 2- to 4-color flow cytometry. (NCT03412565)
Timeframe: For D-VRD Arm: Baseline up to 2 years 3 months; For D-VMP, D-Rd and D-Kd Arms: Baseline up to 2 years 7 months

Interventionpercentage of participants (Number)
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)16.4
D + Bortezomib + Melphalan + Prednisone (D-VMP)55.2
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)50.8
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)42.4

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Percentage of Participants With Anti-rHuPH20 Antibodies

Percentage of participants with antibodies to rHuPH20 were reported. (NCT03412565)
Timeframe: For D-VRD Arm: Baseline up to 2 years 3 months; For D-VMP, D-Rd and D-Kd Arms: Baseline up to 2 years 7 months

Interventionpercentage of participants (Number)
Daratumumab (D)+Bortezomib+Lenalidomide+Dexamethasone (D-VRd)6.1
D + Bortezomib + Melphalan + Prednisone (D-VMP)3.1
Daratumumab + Lenalidomide + Dexamethasone (D-Rd)4.8
Daratumumab + Carfilzomib + Dexamethasone (D-Kd)4.7

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Progression-Free Survival (PFS)

PFS was defined as the time in months from date of initiation of therapy to the earlier of confirmed disease progression or death due to any cause. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg10.0
A (Melflufen+Bortezomib+Dex) 40 mg24.30
B (Melflufen+Daratumumab+Dex) 30 mg28.4
B (Melflufen+Daratumumab+Dex) 40 mg9.7

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Clinical Benefit Rate (≥ Minimal Response)

≥ Minimal response for participants included sCR, CR, VGPR, PR and MR. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionpercentage of participants (Number)
A (Melflufen+Bortezomib+Dex) 30 mg86.7
A (Melflufen+Bortezomib+Dex) 40 mg75.0
B (Melflufen+Daratumumab+Dex) 30 mg83.3
B (Melflufen+Daratumumab+Dex) 40 mg74.1

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Time to Progression (TTP)

TTP defined as time from first dose to first documented confirmed progression. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg17.6
A (Melflufen+Bortezomib+Dex) 40 mg24.3
B (Melflufen+Daratumumab+Dex) 30 mg29.2
B (Melflufen+Daratumumab+Dex) 40 mg11.5

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Time to Response (TTR)

Time from Cycle 1 Day 1 to a response of PR or better. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg3.0
A (Melflufen+Bortezomib+Dex) 40 mg2.2
B (Melflufen+Daratumumab+Dex) 30 mg3.3
B (Melflufen+Daratumumab+Dex) 40 mg1.1

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Best Response (BR)

BR defined by International Myeloma Working Group Uniform Response Criteria. BR= Complete Response (CR)/Stringent CR (sCR) defined as below negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and < 5% plasma cells in bone marrow/plus normal free light chain (FLC) ratio and absence of clonal cells in bone marrow by immunohistochemistry or immunofluorescence, Very Good Partial Response (VGPR)= serum and urine M-protein detectable by immunofixation but not on electrophoresis or > 90% reduction in serum M-protein plus urine M-protein level < 100 mg/24 hours, Partial Response (PR)= a > 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by > 90% or to < 200 mg/24 hours, Minimal Response (MR), Stable Disease (SD)= not meeting the criteria for other response options, Progressive Disease (PD)= increase of > 25% from lowest response value in serum/urine M-component or bone marrow plasma cell percentage, or non-evaluable. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

,,,
InterventionParticipants (Count of Participants)
CRsCRVGPRPRMRSDPDNon-evaluableMissing
Regimen A - Melflufen 30mg + Bortezomib + Dexamethasone102911001
Regimen A - Melflufen 40mg + Bortezomib + Dexamethasone013201001
Regimen B - Melflufen 30mg + Daratumumab + Dexamethasone102200001
Regimen B - Melflufen 40mg + Daratumumab + Dexamethasone1161112104

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Duration of Clinical Benefit (DOCB)

DOCB was defined as time in months from the first evidence of confirmed assessment of sCR, CR, VGPR, PR or MR to first confirmed disease progression, or to death due to any cause. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg15.7
A (Melflufen+Bortezomib+Dex) 40 mg21.3
B (Melflufen+Daratumumab+Dex) 30 mg27.20
B (Melflufen+Daratumumab+Dex) 40 mg10.9

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Duration of Response (DOR)

DOR is defined for participants with confirmed objective response (PR or better, which includes PR, VGPR, CR and sCR) as the time from the first documentation of objective response to the first documentation of objective progression or to death due to any cause, whichever occurs first. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg9.0
A (Melflufen+Bortezomib+Dex) 40 mgNA
B (Melflufen+Daratumumab+Dex) 30 mg24.2
B (Melflufen+Daratumumab+Dex) 40 mg9.9

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Overall Survival (OS)

Time from the first dose of melflufen to death due to any cause. (NCT03481556)
Timeframe: Up to 24 months following confirmed disease progression, or initiation of subsequent therapy (a maximum of 198.9 weeks)

Interventionmonths (Median)
A (Melflufen+Bortezomib+Dex) 30 mg33.5
A (Melflufen+Bortezomib+Dex) 40 mgNA
B (Melflufen+Daratumumab+Dex) 30 mg35.0
B (Melflufen+Daratumumab+Dex) 40 mg18.3

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Phase 1: Number of Participants Who Experienced a Dose Limiting Toxicity (DLT)

"Toxicity was graded according to the National Cancer Institute - Common Terminology Criteria for Adverse Events Version 4.03.~DLT criteria that apply to Regimens A and B:~Grade 3 non-hematologic toxicity preventing the administration of > 1 dose of bortezomib or daratumumab during the 1st cycle.~Grade 4 or greater non-hematologic toxicity.~Grade 4 thrombocytopenia (platelet count < 25,000 cells/ mm^3) preventing the administration of > 1 dose of bortezomib or daratumumab during the 1st cycle or with clinically significant bleeding during the 1st cycle.~Grade 4 neutropenia (ANC < 500 cells/mm^3), lasting more than 7 days during the 1st cycle.~Greater than 14 days' delay to meet the criteria for the start of a new cycle (Cycle 2) due to toxicity." (NCT03481556)
Timeframe: Cycle 1: Day 1 to Day 28

InterventionParticipants (Count of Participants)
Regimen A - Melflufen 30mg + Bortezomib + Dexamethasone0
Regimen A - Melflufen 40mg + Bortezomib + Dexamethasone0
Regimen B - Melflufen 30mg + Daratumumab + Dexamethasone0
Regimen B - Melflufen 40mg + Daratumumab + Dexamethasone0

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Phase 2: Overall Response Rate (ORR)

ORR was defined as the percentage of participants who achieved a best confirmed response of Partial Response (PR) or better. (NCT03481556)
Timeframe: Until disease progression, death or initiation of subsequent therapy (a maximum of 194.3 weeks)

Interventionpercentage of participants (Number)
Regimen A - Melflufen 30mg + Bortezomib + Dexamethasone80
Regimen A - Melflufen 40mg + Bortezomib + Dexamethasone75
Regimen B - Melflufen 30mg + Daratumumab + Dexamethasone83.3
Regimen B - Melflufen 40mg + Daratumumab + Dexamethasone70.4

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Number of Participants Who Experienced a Treatment-emergent Adverse Event (TEAE)

TEAEs were defined as adverse events (AEs) that started after the first dose of study medication was administered and within 30 days of the last administration of the study treatment or before start of subsequent anticancer treatment (whichever occurred first) or that worsened after the first dose of study medication was administered. (NCT03481556)
Timeframe: Cycle 1 Day 1 up to a maximum of 198.9 weeks

,,,
InterventionParticipants (Count of Participants)
Any TEAEsAny Grade 3 or Higher TEAEs
A (Melflufen+Bortezomib+Dex) 30 mg1515
A (Melflufen+Bortezomib+Dex) 40 mg88
B (Melflufen+Daratumumab+Dex) 30 mg66
B (Melflufen+Daratumumab+Dex) 40 mg2726

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Tmax of Melphalan

Time of maximum observed concentration (Tmax) (NCT03639610)
Timeframe: Samples were drawn 5-10 minutes after the end of infusion, 2-3 hours after the end of infusion, and 5-7 hours after the end of infusion.

,,
Interventionminutes (Median)
Cycle 1Cycle 2
Cohort 1a, Melflufen 40 mg38.000037.0000
Cohort 1b, Melflufen 30 mg40.000040.0000
Cohort 2a, Melflufen 20 mg37.000036.0000

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Best Confirmed Response

Best confirmed response required 2 consecutive assessments with the same response result made at any time. In case at the second consecutive assessment (made at any time) the response is higher than the previous one, then confirmed response (linked to the first assessment visit) will be the first one (e.g., PR - VGPR consecutive pair will lead to a PR confirmed response at the first visit). In case the second consecutive response is lower than the first one, then confirmed response (linked to the first assessment visit) will be the second one (e.g. CR-VGPR consecutive pair will lead to a VGPR confirmed response at the first visit). (NCT03639610)
Timeframe: Patients were assessed for response after each cycle. After discontinuation of therapy, patients continued to be assessed until documented progression (confirmed on 2 consecutive assessments) or initiation of subsequent therapy (max duration 127.1 weeks).

,,
InterventionParticipants (Count of Participants)
Stringent complete response (sCR)Complete response (CR)Very good partial response (VGPR)Partial response (PR)Minimal response (MR)Stable disease (SD)Progressive disease (PD)Non-evaluable
Cohort 1a, Melflufen 40 mg00371523
Cohort 1b, Melflufen 30 mg02141200
Cohort 2a, Melflufen 20 mg00010030

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Area Under the Curve (Inf) of Melphalan

Area under the concentration-time curve (AUC) from 0 hours to infinity (NCT03639610)
Timeframe: Samples were drawn 5-10 minutes after the end of infusion, 2-3 hours after the end of infusion, and 5-7 hours after the end of infusion.

,,
Interventionminutes*ng/mL (Mean)
Cycle 1Cycle 2
Cohort 1a, Melflufen 40 mg85123.363580365.5468
Cohort 1b, Melflufen 30 mg77173.068972830.2410
Cohort 2a, Melflufen 20 mg31712.904239685.8277

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Area Under the Curve (0-t) of Melphalan

Area under the concentration-time curve (AUC) from 0h to the last measurable concentration. (NCT03639610)
Timeframe: Samples were drawn 5-10 minutes after the end of infusion, 2-3 hours after the end of infusion, and 5-7 hours after the end of infusion.

,,
Interventionminutes*ng/mL (Mean)
Cycle 1Cycle 2
Cohort 1a, Melflufen 40 mg78250.346274415.5556
Cohort 1b, Melflufen 30 mg70303.440067458.0000
Cohort 2a, Melflufen 20 mg27912.712532146.0000

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Time to Response

Time from first dose of therapy to first documented confirmed response of partial response (PR) or better. (NCT03639610)
Timeframe: From initiation of therapy until documented disease response (maximum duration 127.1 weeks).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg2.45
Cohort 1b, Melflufen 30 mg1.18
Cohort 2a, Melflufen 20 mg2.83

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Time to Clinical Benefit

Time to clinical benefit was defined as the time from first dose of therapy to first documented confirmed response of minimal response (MR) or better. (NCT03639610)
Timeframe: Patients were assessed for response after each cycle. After discontinuation of therapy, patients continued to be assessed until documented progression (confirmed on 2 consecutive assessments) or initiation of subsequent therapy (max duration 127.1 weeks).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg1.12
Cohort 1b, Melflufen 30 mg1.12
Cohort 2a, Melflufen 20 mg2.83

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Cmax of Melphalan

Maximum observed concentration (Cmax) (NCT03639610)
Timeframe: Samples were drawn 5-10 minutes after the end of infusion, 2-3 hours after the end of infusion, and 5-7 hours after the end of infusion.

,,
Interventionng/mL (Mean)
Cycle 1Cycle 2
Cohort 1a, Melflufen 40 mg550.1538539.4444
Cohort 1b, Melflufen 30 mg472.2000469.5000
Cohort 2a, Melflufen 20 mg181.2500194.0000

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Clinical Benefit Rate

Clinical benefit rate (CBR) is the proportion of patients who achieved a confirmed minimal response (MR) or better (stringent complete response [sCR], complete response [CR], very good partial response [VGPR], partial response [PR], and MR) as their best response, as assessed by the Investigator. (NCT03639610)
Timeframe: Patients were assessed for response after each cycle (maximum duration 127.1 weeks).

InterventionParticipants (Count of Participants)
Cohort 1a, Melflufen 40 mg11
Cohort 1b, Melflufen 30 mg8
Cohort 2a, Melflufen 20 mg1

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Duration of Clinical Benefit

Duration of clinical benefit (DOCB) was calculated as time in months from the first evidence of confirmed assessment of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), partial response (PR), or minimal response (MR) to first confirmed disease progression, or to death due to any cause. DOCB was defined only for patients with a confirmed MR or better. DOCB was summarized using the Kaplan-Meier (K-M) method. The median DOCB was estimated from the 50th percentile of the corresponding K-M estimates. The 95% confidence interval for median DOCB was constructed using the method of Brookmeyer (Brookmeyer, 1982). (NCT03639610)
Timeframe: Patients were assessed from the first measure of a confirmed MR or better until confirmed progression, death, or initiation of subsequent therapy (maximum duration 127.1 weeks).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg9.26
Cohort 1b, Melflufen 30 mg10.58
Cohort 2a, Melflufen 20 mgNA

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Progression-free Survival

Progression-free survival (PFS) was defined as the time from the date of first study drug (the earliest of melflufen and dexamethasone start date) initiation to the date of first documentation of confirmed PD or death due to any cause, whichever occurred first. Participants were deemed 'progressed' in case of i) unconfirmed progressive disease (PD) as the final response assessment, ii) death after at least one response assessment or PD based on at least two consecutive response assessments at any time, or iii) death before the first response assessment. The distribution of PFS was summarized using the Kaplan-Meier (K-M) method. The median PFS was estimated from the 50th percentile of the corresponding K-M estimates. The 95% confidence interval for median PFS was constructed using the method of Brookmeyer (Brookmeyer, 1982). (NCT03639610)
Timeframe: Patients were assessed from the initiation of therapy until documented disease progression or initiation of new therapy (maximum duration 127.1 weeks).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg8.61
Cohort 1b, Melflufen 30 mg7.66
Cohort 2a, Melflufen 20 mg3.43

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Duration of Response

Duration of response (DOR) is defined as the time from the first evidence of confirmed assessment of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR) to first confirmed disease progression, or to death due to any cause. The distribution of DOR was summarized using the Kaplan-Meier (K-M) method. The median DOR was estimated from the 50th percentile of the corresponding K-M estimates. The 95% confidence interval for median DOR was constructed using the method of Brookmeyer (Brookmeyer, 1982). (NCT03639610)
Timeframe: Patients were assessed for response from the first measure of a confirmed response (PR or better) until confirmed progression, death, or initiation of subsequent therapy (maximum duration 127.1 weeks).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg8.31
Cohort 1b, Melflufen 30 mg13.83
Cohort 2a, Melflufen 20 mgNA

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Overall Response Rate

Overall response rate (ORR) is the percentage of patients who achieved a confirmed response of stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR) as best response, as assessed by the Investigator. (NCT03639610)
Timeframe: Patients were assessed for response after each cycle. After discontinuation of therapy, patients continued to be assessed until disease progression (confirmed on 2 consecutive assessments) (maximum duration 127.1 weeks).

InterventionParticipants (Count of Participants)
Cohort 1a, Melflufen 40 mg10
Cohort 1b, Melflufen 30 mg7
Cohort 2a, Melflufen 20 mg1

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Overall Survival

Overall survival was defined as the time in months from initiation of therapy to death due to any cause. Patients still alive at the end of the study, or lost to follow up, were censored at last day known alive. (NCT03639610)
Timeframe: Patients were followed for overall survival until death or until the last patient in the study had documented progression (confirmed on 2 consecutive assessments) or initiation of subsequent therapy (maximum of 39.2 months).

Interventionmonths (Median)
Cohort 1a, Melflufen 40 mg9.76
Cohort 1b, Melflufen 30 mgNA
Cohort 2a, Melflufen 20 mgNA

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T1/2 of Melphalan

Elimination half-life of melphalan (NCT03639610)
Timeframe: Samples were drawn 5-10 minutes after the end of infusion, 2-3 hours after the end of infusion, and 5-7 hours after the end of infusion.

,,
Interventionminutes (Mean)
Cycle 1Cycle 2
Cohort 1a, Melflufen 40 mg89.139087.5544
Cohort 1b, Melflufen 30 mg98.356890.7844
Cohort 2a, Melflufen 20 mg109.5502136.2193

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Response Rate

Per the revised INRC, response is comprised by responses in 3 components: primary tumor, soft tissue and bone metastases, and bone marrow. Primary and metastatic soft tissue sites were assessed using Response Evaluation Criteria in Solid Tumors and MIBG scans or FDG-PET scans if the tumor was MIBG non-avid. Bone marrow was assessed by histology or immunohistochemistry and cytology or immunocytology. Complete response (CR) - All components meet criteria for CR. Partial response (PR) - PR in at least one component and all other components are either CR, minimal disease (in bone marrow), PR (soft tissue or bone) or not involved (NI; no component with progressive disease (PD). Minor response (MR) - PR or CR in at least one component but at least one other component with stable disease; no component with PD. Stable disease (SD) - Stable disease in one component with no better than SD or NI in any other component; no component with PD. Progressive disease (PD) - Any component with PD. (NCT03786783)
Timeframe: Up to the first 5 cycles of treatment

InterventionPercentage of patients (Number)
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)78.6

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Percentage of Participants With Unacceptable Toxicity

Assessed with National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Assessed by estimation of the combined toxic death and unacceptable toxicity rate during Induction cycles 3-5 together with a 95% confidence interval. (NCT03786783)
Timeframe: Up to the first 5 cycles of treatment

Interventionpercentage of patients (Number)
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)0.0

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Overall Survival

Kaplan-Meier method was used to estimate overall survival (OS). OS was defined as the time from study enrollment to death. 1-year OS is provided. (NCT03786783)
Timeframe: Up to 1 year

InterventionPercent Probability (Number)
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)95.0

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Event-free Survival

Per the revised INRC, progressive disease is: 1) > 20% increase in the longest diameter of the primary tumor, taking as reference the smallest sum and ¬> increase of 5 mm in longest dimension, 2) Any new soft tissue lesion detected by CT/MRI that is MIBG avid or FDG-PET avid, 3) Any new soft tissue lesion seen on CT/MRI that is biopsied and found to be neuroblastoma or ganglioneuroblastoma, 4) Any new bone site that is MIBG avid, 5) Any new bone site that is FDG-PET avid and has CT/MRI findings of tumor or is histologically neuroblastoma or ganglioneuroblastoma 6) A metastatic soft tissue site with > 20% increase in longest diameter, taking as reference the smallest sum on study, and with > 5mm in sum of diameters of target soft tissue lesions, 7) A relative MIBG score ¬> 1.2, 8) Bone marrow without tumor infiltration that becomes >5% tumor infiltration, 9) Bone marrow with tumor infiltration that increases by > 2-fold and has > 20% tumor infiltration on reassessment. (NCT03786783)
Timeframe: Up to 1 year

InterventionPercent Probability (Number)
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)82.6

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"Percentage of Participants Who Are Feasibility Failure"

"Feasibility failures were defined as patients that did not receive >= 75% of the planned dinutuximab doses during Induction cycles 3-5. Assessed by estimation of the feasibility failure rate together with a 95% confidence interval." (NCT03786783)
Timeframe: Up to the first 5 cycles of treatment

InterventionPercentage of patients (Number)
Treatment(Chemotherapy, Dinutuximab, Sargramostim, ASCT, EBRT)0.0

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Number of Patients With a Partial Response

The proportion of each type of response with a 95% CI will be reported for all evaluable patients, assuming a binomial distribution. (NCT04205240)
Timeframe: Approximately 11 months

Interventionpatients (Number)
Treatment (Conditioning Regimen, Stem Cell Transplant)1

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Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)

The event will be onset of grade II-IV aGvHD and time to aGvHD will be defined as the period of time from transplantation to the event of aGvHD. Death and early relapse without aGvHD will be competing risks. Cumulative incidence rate of aGVHD with 95% confidence intervals will be estimated from the cumulative incidence curves. To evaluate the association between patient characteristics and aGVHD, the Gray's test accounting for competing risks will be used to compare the cumulative incidence curves and a proportional hazards model for the sub distribution of competing risks will be used to estimate the hazard ratio. The cumulative incidence of chronic GVHD (cGVHD) will be similarly analyzed. (NCT04205240)
Timeframe: Up to 6 weeks

Interventionparticipants (Number)
Treatment (Conditioning Regimen, Stem Cell Transplant)0

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Cumulative Incidence of Grade II-IV Acute GVHD

Acute GVHD will be graded and staged according to the Consensus Grading. (NCT04339101)
Timeframe: From day 0 (date of stem cell infusion) through 100 days post-transplant

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)4

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Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year

GRFS is defined as time from the date of transplantation to the first time of observing following events: grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first. Kaplan-Meier curve will be generated for GRFS. (NCT04339101)
Timeframe: From the date of transplantation to the first time of observing following events: grade 3-4 acute graft versus host disease (GVHD), chronic GVHD requiring systemic treatment, relapse, or death, whichever occurs first, assessed at 1 year post transplant.

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)56

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Progression Free Survival (PFS)

Kaplan-Meier curve will be generated for PFS. (NCT04339101)
Timeframe: From the date of stem cell infusion to the date of death, disease relapse/progression, or last follow-up, whichever occurs first, assessed at 1 year post transplant

Interventionpercentage of probability (Number)
Treatment (Itacitinib Adipate, Tacrolimus, Sirolimus)70

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Proportion of Failure of the Haplo-Graft

Proportion of patients with a failed haplo-graft, defined as the absence of neutrophil engraftment by Day +21 or a drop in the absolute neutrophil count to <0.3 cells/microL for five consecutive days occurring after initial neutrophil engraftment within the first 3 weeks post-transplantation (second nadir) (NCT04395222)
Timeframe: 21 days post-transplant

InterventionParticipants (Count of Participants)
ATG Group I2
ATG Group II5
ATG Group III1

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Percentage of Subjects With Successful Haplo-derived Neutrophil Engraftment

"This is defined as:~Achieve an absolute neutrophil count (ANC) of 500 cells/microL for three consecutive days with the first on or prior to Day +21 post-transplant, AND~Absence of a second nadir - a drop in the ANC to <300 cells/microL for five consecutive days - after initial neutrophil recovery." (NCT04395222)
Timeframe: 21 days post-transplant

Interventionpercentage of participants (Number)
ATG Group I80
ATG Group II50
ATG Group III0

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Best Response (Stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), Partial Response (PR), Minimal Response (MR), Stable Disease (SD) or Progressive Disease (PD)

To assess best response during the study with the criteria established by the International Myeloma Working Group Uniform Response Criteria (IMWG-URC) for sCR, CR, VGPR, PR, SD and PD (NCT04412707)
Timeframe: From initiation of therapy until disease progression. Median treatment durations for Arm A and Arm B were 29.14 weeks and 12.14 weeks, respectively.

,,
InterventionParticipants (Count of Participants)
Stringent complete responseComplete responseVery good partial responsePartial responseMinimal responseStable diseaseProgressive diseaseNot available
Arm A01034222
Arm B00011614
Overall01045836

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ORR

To assess overall response rate (ORR), including CR/sCR, VGPR and PR, during the study with the criteria established by the IMWG-URC. (NCT04412707)
Timeframe: From initiation of therapy until disease progression. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

InterventionParticipants (Count of Participants)
Arm A4
Arm B1
Overall5

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PFS

To assess progression free survival (PFS) (NCT04412707)
Timeframe: From initiation of therapy until documented disease progression or initiation of new therapy. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

Interventionmonths (Median)
Arm A5.21
Arm B2.89
Overall3.73

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TTNT

To assess time to next treatment (TTNT) (NCT04412707)
Timeframe: From randomization to the date of next anti-myeloma treatment. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

Interventionmonths (Median)
Arm ANA
Arm BNA
OverallNA

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TTP

To assess time to progression (TTP) during the study with the criteria established by the IMWG-URC. (NCT04412707)
Timeframe: From date of randomization until documented disease progression. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

Interventionmonths (Median)
Arm A5.78
Arm B4.80
Overall5.78

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TTR

To assess time to response (TTR) in patients with PR or better during the study with the criteria established by the UMWG-URC. (NCT04412707)
Timeframe: From initiation of therapy until documented disease response. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

InterventionParticipants (Count of Participants)
Arm A4
Arm B1
Overall5

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Area Under the Plasma Concentration Versus Time Curve AUC(0-inf) of Melflufen and Desethyl-melflufen

To evaluate and compare the pharmacokinetic (PK) variable AUC(0-inf) of melflufen and desethyl-melflufen after central and peripheral intravenous infusion of melflufen (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1 - 13 PK samples during and post infusion (28 days cycle)

,
Interventionmin x ng/mL (Geometric Mean)
Melflufen Cycle 1 (0-inf)Meflufen Cycle 2 (0-inf)Desethyl-melflufen Cycle 1 (0-inf)Desethyl-melflufen Cycle 2 (0-inf)
Central Venous Catheter (CVC)2841.233093.96759.25440.65
Peripheral Venous Catheter (PVC)3639.343099.70609.44695.29

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Area Under the Plasma Concentration Versus Time Curve AUC(0-inf) of Melphalan

To evaluate and compare the pharmacokinetic (PK) variable AUC(0-inf) of melphalan after central and peripheral intravenous infusion of melflufen (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1. Samples were collected 5, 10, 15, 20, and 25 minutes after the start of the infusion; immediately before the end of the infusion; and 5, 10, 15, and 30 minutes and 1, 2, and 4 hours after the end of the infusion.

,
Interventionmin*ng/mL (Geometric Mean)
Cycle 1Cycle 2
Central Venous Catheter (CVC)66835.4750835.59
Peripheral Venous Catheter (PVC)54216.7067403.07

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Area Under the Plasma Concentration Versus Time Curve AUC(0-t) of Melflufen and Desethyl-melflufen

To evaluate and compare the pharmacokinetic (PK) variable AUC(0-t) of melflufen and desethyl-melflufen after central and peripheral intravenous infusion of melflufen (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1 - 13 PK samples during and post infusion (28 days cycle)

,
Interventionmin x ng/mL (Geometric Mean)
Melflufen Cycle 1 (0-t)Melflufen Cycle 2 (0-t)Desethyl-melflufen Cycle 1 (0-t)Desethyl-melflufen Cycle 2 (0-t)
Central Venous Catheter (CVC)2828.693078.21639.39391.11
Peripheral Venous Catheter (PVC)3617.033083.74563.34636.06

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Area Under the Plasma Concentration Versus Time Curve AUC(0-t) of Melphalan

To evaluate and compare the pharmacokinetic (PK) variable AUC(0-t) of melphalan after central and peripheral intravenous infusion of melflufen. (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1. Samples were collected 5, 10, 15, 20, and 25 minutes after the start of the infusion; immediately before the end of the infusion; and 5, 10, 15, and 30 minutes and 1, 2, and 4 hours after the end of the infusion.

,
Interventionmin*ng/mL (Geometric Mean)
Cycle 1Cycle 2
Central Venous Catheter (CVC)59543.2046173.13
Peripheral Venous Catheter (PVC)49518.3660273.95

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Elimination Half-life (t1/2) of Melflufen, Melphalan and Desethyl-melflufen

To evaluate elimination half-life (t½) for melflufen, melphalan and desethyl-melflufen after central and peripheral intravenous infusion of melflufen. (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1 - 13 measurements during and post infusion (28 days cycle)

,
Interventionminutes (Mean)
Melphalan Cycle 1Melphalan Cycle 2Melflufen Cycle 1Melflufen Cycle 2Desethyl-melflufen Cycle 1Desethyl-melflufen Cycle 2
Central Venous Catheter (CVC)80.0972.974.455.7423.4917.43
Peripheral Venous Catheter (PVC)72.5178.097.426.1518.4425.04

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Frequency of Treatment-Emergent Adverse Events (TEAEs) by MedDRA SOC and PT

To assess safety and general tolerability of melflufen by collecting non-serious Adverse Events (AEs) from the start of study treatment until 30 days after the last dose of any study drug (melflufen or dexamethasone) or initiation of subsequent therapy whichever occurred first. Serious AEs (SAEs) were collected from the time the subject signed the ICF until 30 days after the last dose of any study drug (melflufen or dexamethasone) or initiation of subsequent therapy whichever occurred first. (NCT04412707)
Timeframe: Median treatment durations for Arm A and Arm B were 29.14 weeks and 12.14 weeks, respectively. The AE reporting period is estimated to be 30 days longer.

,,
InterventionParticipants (Count of Participants)
Patients with at least 1 TEAEBlood and lymphatic system disordersThrombocytopeniaNeutropeniaAnaemiaLeukopeniaInfections and InfestationsPneumoniaCOVID-19 pneumoniaRespiratory tract infectionGeneral disorders and administration site conditionsPyrexiaFatigueGeneral health deteriorationMusculoskeletal and connective tissue disordersArthralgiaBack painBone painInvestigationsSARS-CoV-2 test positiveC-reactive protein increasedInjury, poisoning and procedural complicationsfemur fractureSkin and subcutaneous tissue disordersRash
Arm A1313109926302521041224220022
Arm B1312109717230632252104403200
Overall26252018163135321153293328623222

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Grade 3/4 Treatment-Emergent Adverse Events (TEAEs)

To assess safety and general tolerability of melflufen by collecting non-serious Adverse Events (AEs) from the start of study treatment until 30 days after the last dose of any study drug (melflufen or dexamethasone) or initiation of subsequent therapy whichever occurred first. Serious AEs (SAEs) were collected from the time the subject signed the ICF until 30 days after the last dose of any study drug (melflufen or dexamethasone) or initiation of subsequent therapy whichever occurred first. (NCT04412707)
Timeframe: Median treatment durations for Arm A and Arm B were 29.14 and 12.14 weeks, respectively. The AE reporting period is estimated to be 30 days longer.

,,
InterventionParticipants (Count of Participants)
Blood and lymphatic system disordersThrombocytopeniaNeutropeniaAnaemiaLeukopeniaLymphopeniaFebrile neutropeniaInfections and infestationsPneumoniaCOVID-19 pneumoniaSepsisGeneral disorders and administration site conditionsGeneral physical health deteriorationDeathAstheniaInjury, poisoning and procedural complicationsFemur fractureFemoral neck fractureGastrointestinal disordersStomatitisMusculoskeletal and connective tissue disordersBack painMusculoskeletal chest painNervous system disordersIschaemic strokeVascular disordersHypertension
Arm A: Grade 3/41288521122010000000001101100
Arm B: Grade 3/410107311121100001321111010011
Overall: Grade 3/4221815832243110001321112111111

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Grade 5 Treatment-Emergent Adverse Events (TEAEs)

To assess safety and general tolerability of melflufen by collecting serious adverse events (SAEs) from the signing of the ICF until 30 days after the last dose of any study drug (melflufen or dexamethasone) or initiation of subsequent therapy whichever comes first. (NCT04412707)
Timeframe: From signing of the ICF until 30 days after the last dose of any study drug or initiation of subsequent therapy, whichever comes first. Median treatment durations for Arms A and B were 29.14 weeks and 12.14 weeks. AE reporting period=30 days longer

,,
InterventionParticipants (Count of Participants)
Infections and infestationsPneumoniaCOVID-19 pneumoniaGeneral disorders and administration site conditionsGeneral physical health deteriorationDeathGastrointestinal disordersIleus
Arm A: Grade 500000011
Arm B: Grade 531232100
Overall: Grade 531232111

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Peak Plasma Concentration for Melflufen and Desethyl-melflufen

To evaluate and compare the pharmacokinetic (PK) variable Cmax of melflufen and desethyl-melflufen after central and peripheral intravenous infusion of melflufen. (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1 - 13 PK samples during and post infusion (28 days cycle)

,
Interventionng/mL (Geometric Mean)
Melflufen Cycle 1Melflufen Cycle 2Desethyl-melflufen Cycle 1Desethyl-melflufen Cycle 2
Central Venous Catheter (CVC)123.0141.7511.85111.851
Peripheral Venous Catheter (PVC)151.11127.2716.72116.801

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Peak Plasma Concentration for Melphalan

To evaluate and compare the pharmacokinetic (PK) variable Cmax of melphalan after central and peripheral intravenous infusion of melflufen. (NCT04412707)
Timeframe: Cycle 1 Day 1 and Cycle 2 Day 1. Samples were collected 5, 10, 15, 20, and 25 minutes after the start of the infusion; immediately before the end of the infusion; and 5, 10, 15, and 30 minutes and 1, 2, and 4 hours after the end of the infusion.

,
Interventionng/mL (Geometric Mean)
Cycle 1Cycle 2
Central Venous Catheter (CVC)530.1449.2
Peripheral Venous Catheter (PVC)486.1546.3

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Number of Participants With Local Reactions Including Phlebitis at Infusion Site After Peripheral Intravenous Administration

Assessment of the local tolerability of peripheral intravenous administration of melflufen using the Visual Infusion Phlebitis (VIP) scale. The VIP scale provides a score from 0 to 5, noting an ascending order of severity of inflammation. A score of 0 is the lowest possible score, meaning no inflammation detected, and 5 is the highest score, indicating the most severe reaction. (NCT04412707)
Timeframe: 15 minutes and 4 hours after peripheral intravenous administration, pre- and post-infusion on Day 1 and Day 8

InterventionParticipants (Count of Participants)
Cycle 1 Day 1 Pre-Infusion72564849Cycle 1 Day 1 Post-Infusion72564849Cycle 1 Day 872564849Cycle 2 Day 1 Pre-Infusion72564849
VIP score = 4VIP score = 0VIP score = 1VIP score = 5VIP score = 2VIP score = 3
Peripheral Venous Catheter (PVC)13
Peripheral Venous Catheter (PVC)0
Peripheral Venous Catheter (PVC)10
Peripheral Venous Catheter (PVC)8

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CBR

To assess clinical benefit rate (CBR), i.e., proportion of patients that achieve a confirmed minimal response or better (sCR, CR, VGPR, PR and MR), during the study with the criteria established by the IMWG-URC. (NCT04412707)
Timeframe: To be assessed at the end of study drug treatment. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

InterventionParticipants (Count of Participants)
Arm A8
Arm B2
Overall10

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DOCB

To assess duration of clinical benefit (DOCB) in patients with stringent complete response (sCR), complete response (CR), very good partial response (VGPR), PR, or MR during the study with the criteria established by the IMWG-URC. (NCT04412707)
Timeframe: From first evidence of confirmed assessment of sCR, CR, VGPR, PR or MR to first confirmed disease progression, or to death due to any cause. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

Interventionmonths (Median)
Arm ANA
Arm BNA
OverallNA

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DOR

To assess duration of response (DOR): the time in months from the first evidence of confirmed assessment of sCR, CR, VGPR, or PR to first confirmed disease progression according to the criteria established by the IMWG-URC or to death due to any cause. DOR is defined only for patients with a confirmed PR or better. (NCT04412707)
Timeframe: From confirmed response until disease progression. Maximum treatment durations for Arm A and Arm B were 68.4 and 59.0 weeks, respectively.

Interventionmonths (Median)
Arm ANA
Arm BNA
OverallNA

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Progression Free Survival (PFS)

Time from the date of randomization to the date of first documentation of confirmed progressive disease (PD) or death due to any cause, whichever occurred first. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Median)
Arm A (Melflufen+Dexamethasone+Daratumumab)NA
Arm B (Daratumumab)4.86

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Overall Survival (OS)

Time from randomization to death due to any cause. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Median)
Arm A (Melflufen+Dexamethasone+Daratumumab)11.04
Arm B (Daratumumab)NA

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Overall Response Rate (ORR)

Proportion of patients who achieve a best-confirmed response of stringent Complete Response (sCR), Complete Response (CR), Very Good Partial Response (VGPR), or Partial Response (PR). (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionpercentage of patients (Number)
Arm A (Melflufen+Dexamethasone+Daratumumab)59.3
Arm B (Daratumumab)29.6

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Duration of Response (DOR)

Time from the first evidence of confirmed assessment of sCR, CR, VGPR or PR to first confirmed disease progression, or death due to any cause. DOR is defined only for patients with a confirmed PR or better. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Median)
Arm A (Melflufen+Dexamethasone+Daratumumab)NA
Arm B (Daratumumab)NA

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Duration of Clinical Benefit (DOCB)

Time from first evidence of confirmed assessment of sCR, CR, VGPR, PR, or MR to first confirmed disease progression, or to death due to any cause. DOCB is defined only for patients with a confirmed MR or better. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Median)
Arm A (Melflufen+Dexamethasone+Daratumumab)NA
Arm B (Daratumumab)NA

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Clinical Benefit Rate (CBR)

The proportion of patients who achieve a best confirmed response of sCR, CR, VGPR, PR, or MR. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionpercentage of patients (Number)
Arm A (Melflufen+Dexamethasone+Daratumumab)70.4
Arm B (Daratumumab)48.1

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Time to Response (TTR)

Time from randomization to the date of the first documented confirmed response in a patient who has responded with ≥PR. (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Mean)
Arm A (Melflufen+Dexamethasone+Daratumumab)1.8
Arm B (Daratumumab)1.8

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Time to Progression (TTP)

Time from randomization to the date of the first documented confirmed PD (NCT04649060)
Timeframe: From the date of randomization until the end of study (approximately 12 months).

Interventionmonths (Median)
Arm A (Melflufen+Dexamethasone+Daratumumab)NA
Arm B (Daratumumab)NA

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Change in Abdominal Pain as Assessed Per CTCAE v5.0

Grade 0 abdominal pain is defined as no abdominal pain. Grade 1 abdominal pain is defined as mild pain. Grade 2 abdominal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 abdominal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 abdominal pain defined in the CTCAE v5.0. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.040.000.040.040.170.250.130.250.130.210.290.540.500.460.380.290.220.05
Uproleselan + Standard of Care Melphalan0.000.000.000.160.190.350.120.120.080.310.350.580.540.380.150.230.120.12

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Change in Bristol Stool Scale

Change in Bristol Stool Scale as measured by incidence of Type 7 Bristol Stool Scale: liquid consistency with no solid pieces. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionParticipants (Count of Participants)
Day -3, AMDay -3, PMDay -2, AMDay -2, PMDay -1, AMDay -1, PMDay 0, AMDay 0, PMDay 1, AMDay 1, PMDay 2, AMDay 2, PMDay 3, AMDay 3, PMDay 4, AMDay 4, PMDay 5, AMDay 5, PMDay 6, AMDay 6, PMDay 7, AMDay 7, PMDay 8, AMDay 8, PMDay 9, AMDay 9, PMDay 10, AMDay 10, PMDay 11, AMDay 11, PMDay 12, AMDay 12, PMDay 13, AMDay 13, PMDay 14, AMDay 14, PM
Placebo + Standard of Care Melphalan11210131435681012111210111110121514161716191911977874
Uproleselan + Standard of Care Melphalan000010105132115847612121415141918131310981186332

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Change in Diarrhea as Assessed Per CTCAE v5.0

Grade 0 is defined as no diarrhea, or no change from baseline. Grade 1 is defined as an increase of <4 stools per day over baseline; mild increase in ostomy output compared to baseline. Grade 2 is defined as an increase of 4-6 stools per day over baseline; moderate increase in ostomy output compared to baseline; limiting instrumental ADL. Grade 3 is defined as an increase of >=7 stools per day over baseline; hospitalization indicated; severe increase in ostomy output compared to baseline; limited self care ADL. Grade 4 is defined as life-threatening consequences; urgent intervention indicated. Grade 5 is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.130.130.380.210.250.460.881.081.291.421.381.631.541.921.631.171.130.95
Uproleselan + Standard of Care Melphalan0.000.000.310.190.230.420.650.881.001.191.421.381.541.541.461.231.000.92

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Change in Enterocolitis as Assessed Per CTCAE v5.0

Grade 0 enterocolitis is defined as no enterocolitis. Grade 1 enterocolitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 enterocolitis is defined as abdominal pain; mucus or blood in stool. Grade 3 enterocolitis is defined as severe or persistent abdominal pain; fever; ileus; peritoneal signs. Grade 4 enterocolitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 enterocolitis is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.000.040.080.000.040.040.080.080.250.380.380.250.250.130.05
Uproleselan + Standard of Care Melphalan0.000.000.000.000.080.040.000.040.000.000.120.270.270.230.120.080.040.00

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Change in Esophageal Pain as Assessed Per CTCAE v5.0

Grade 0 esophageal pain is defined as no esophageal pain. Grade 1 esophageal pain is defined as mild pain. Grade 2 esophageal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 esophageal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 esophageal pain defined in the CTCAE v5.0. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.040.040.040.040.130.130.290.460.330.290.330.250.170.130.14
Uproleselan + Standard of Care Melphalan0.000.000.000.000.000.120.040.080.040.190.350.460.460.380.310.190.230.00

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Change in Esophagitis as Assessed Per CTCAE v5.0

Grade 0 esophagitis is defined as no presence of esophagitis. Grade 1 esophagitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 esophagitis is defined as symptomatic; altered GI function; limiting instrumental ADL. Grade 3 esophagitis is defined as severely altered GI function; TPN indicated; elective invasive intervention indicated; limiting self care ADL. Grade 4 esophagitis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 esophagitis is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.000.000.000.000.040.040.170.250.250.250.250.290.210.130.14
Uproleselan + Standard of Care Melphalan0.000.000.000.000.000.080.040.040.040.120.230.580.540.460.380.190.120.04

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Change in Gastritis as Assessed Per CTCAE v5.0

Grade 0 gastritis is defined as no presence of esophagitis. Grade 1 gastritis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 gastritis is defined as symptomatic; altered GI function; medical intervention indicated. Grade 3 gastritis is defined as severely altered eating or gastric function; TPN or hospitalization indicated. Grade 4 gastritis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 gastritis is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.000.040.130.080.040.040.080.080.040.090.130.170.130.090.05
Uproleselan + Standard of Care Melphalan0.000.000.000.000.040.150.000.000.000.080.120.190.230.190.120.080.040.00

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Change in Oral Mucositis as Assessed Per CTCAE v5.0

Grade 0 oral mucositis is defined as no presence of mucositis. Grade 1 oral mucositis is defined as asymptomatic or mild symptoms; intervention not indicated. Grade 2 oral mucositis is defined as moderate pain or ulcer that does not interfere with oral intake; modified diet indicated. Grade 3 oral mucositis is defined as severe pain; interfering with oral intake. Grade 4 oral mucositis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 oral mucositis is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.040.040.000.080.080.080.040.080.040.170.420.330.380.330.380.210.220.23
Uproleselan + Standard of Care Melphalan0.000.000.000.000.000.120.160.080.120.120.310.230.380.350.270.150.150.04

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Change in Proctitis as Assessed Per CTCAE v5.0

Grade 0 proctitis is defined as no proctitis. Grade 1 proctitis is defined as rectal discomfort, intervention not indicated. Grade 2 proctitis is defined as symptomatic (e.g., rectal discomfort, passing blood or mucus); medical intervention indicated; limiting instrumental ADL. Grade 3 proctitis is defined as severe symptoms; fecal urgency or stool incontinence; limiting self-care ADL. Grade 4 proctitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 proctitis is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.000.000.000.000.040.040.040.040.000.080.080.000.000.000.00
Uproleselan + Standard of Care Melphalan0.000.000.000.000.000.040.000.000.000.120.000.000.080.080.000.000.000.00

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Change in Vomiting as Assessed Per CTCAE v5.0

Grade 0 vomiting is defined as no vomiting. Grade 1 vomiting is defined as intervention not indicated. Grade 2 vomiting is defined as outpatient IV hydration; medical intervention indicated. Grade 3 vomiting is defined as tube feeding, TPN, or hospitalization indicated. Grade 4 vomiting is defined as life-threatening consequences. Grade 5 vomiting is defined as death. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.080.290.290.630.500.670.540.460.670.420.420.380.380.290.130.09
Uproleselan + Standard of Care Melphalan0.000.000.040.120.270.350.150.150.190.190.460.620.420.190.150.080.000.12

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Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0

"Questions regarding gastrointestinal toxicities~Responses are scored from 1-5 with 1=never and 5=almost constantly" (NCT04682405)
Timeframe: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)

,
Interventionscore on a scale (Median)
Frequency of nausea - Day -3Frequency of nausea - Day 8Frequency of nausea - date of discharge or Day 14 (whichever is sooner)Frequency of vomiting - Day -3Frequency of vomiting - Day 8Frequency of vomiting - date of discharge or Day 14 (whichever is sooner)Frequency of heartburn - Day -3Frequency of heartburn - Day 8Frequency of heartburn - date of discharge or Day 14 (whichever is sooner)Frequency of bloating - Day -3Frequency of bloating - Day 8Frequency of bloating - date of discharge or Day 14 (whichever is sooner)Frequency of diarrhea - Day -3Frequency of diarrhea - Day 8Frequency of diarrhea - date of discharge or Day 14 (whichever is sooner)Frequency of abdominal pain - Day -3Frequency of abdominal pain - Day 8Frequency of abdominal pain - date of discharge or Day 14 (whichever is sooner)Frequency of bowel incontinence - Day -3Frequency of bowel incontinence - Day 8Frequency of bowel incontinence - date of discharge or Day 14 (whichever is sooner)
Placebo + Standard of Care Melphalan1.004.003.001.002.502.001.001.001.501.002.002.001.004.004.001.002.502.001.001.001.50
Uproleselan + Standard of Care Melphalan1.004.003.001.002.002.001.001.001.001.002.002.001.004.004.001.002.002.001.002.002.00

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Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0

"Questions regarding gastrointestinal toxicities~Responses are scored from 1-5 with 1=no symptoms to 5=severe symptoms" (NCT04682405)
Timeframe: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)

,
Interventionscore on a scale (Median)
Severity of difficulty swallowing - Day -3Severity of difficulty swallowing - Day 8Severity of difficulty swallowing - date of discharge or Day 14 (whichever is sooner)Severity of mouth or throat sores - Day -3Severity of mouth or throat sores - Day 8Severity of mouth or throat sores - date of discharge or Day 14 (whichever is sooner)Severity of decreased appetite - Day -3Severity of decreased appetite - Day 8Severity of decreased appetite - date of discharge or Day 14 (whichever is sooner)Severity of nausea - Day -3Severity of nausea - Day 8Severity of nausea - date of discharge or Day 14 (whichever is sooner)Severity of vomiting - Day -3Severity of vomiting - Day 8Severity of vomiting - date of discharge or Day 14 (whichever is sooner)Severity of heartburn - Day -3Severity of heartburn - Day 8Severity of heartburn - date of discharge or Day 14 (whichever is sooner)Severity of bloating - Day -3Severity of bloating - Day 8Severity of bloating - date of discharge or Day 14 (whichever is sooner)Severity of abdominal pain - Day -3Severity of abdominal pain - Day 8Severity of abdominal pain - date of discharge or Day 14 (whichever is sooner)
Placebo + Standard of Care Melphalan1.001.001.001.001.001.001.004.003.001.003.502.501.002.002.001.001.001.501.002.001.501.002.502.00
Uproleselan + Standard of Care Melphalan1.001.001.001.001.001.001.003.003.001.003.002.001.002.002.001.001.001.001.002.002.001.002.001.00

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Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0

"Questions regarding gastrointestinal, pain, and psychological symptoms interfering with daily activities~Responses are scored from 1-5 with 1=not at all to 5=very much" (NCT04682405)
Timeframe: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)

,
Interventionscore on a scale (Median)
Mouth or throat sore interference - Day -3Mouth or throat sore interference - Day 8Mouth or throat sore interference - date of discharge or Day 14 (whichever is sooner)Decreased appetite interference - Day -3Decreased appetite interference - Day 8Decreased appetite interference - date of discharge or Day 14 (whichever is sooner)Abdominal pain interference - Day -3Abdominal pain interference - Day 8Abdominal pain interference - date of discharge or Day 14 (whichever is sooner)Bowel incontinence interference - Day -3Bowel incontinence interference - Day 8Bowel incontinence interference - date of discharge or Day 14 (whichever is sooner)
Placebo + Standard of Care Melphalan1.001.001.001.003.503.001.001.501.001.001.001.00
Uproleselan + Standard of Care Melphalan1.001.001.001.003.002.001.002.001.001.001.002.00

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Change in Nutritional Status as Assessed by Change in Standing Weight

Patient standing weight in kilograms was taken at specific time points to assess any changes in nutritional status. (NCT04682405)
Timeframe: Day -3, Day 8, and date of discharge or Day 14 (whichever is sooner) (up to be 18 days)

,
Interventionkilograms (Mean)
Day -3Day 8Day 14 or day of discharge, whichever is sooner
Placebo + Standard of Care Melphalan89.4887.2489.44
Uproleselan + Standard of Care Melphalan93.2692.8193.34

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Median Daily Dose of Anti-diarrheal Medications

This is defined as the number of doses of anti-diarrheal medications, such as loperamide or lomotil, that participants took daily. (NCT04682405)
Timeframe: Through date of discharge (up to be 18 days)

Interventionnumber of doses in one day (Median)
Uproleselan + Standard of Care Melphalan2.20
Placebo + Standard of Care Melphalan2.00

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Duration of Hospital Length of Stay

(NCT04682405)
Timeframe: From date of admission for auto-HCT to date of discharge (up to be 18 days)

InterventionDays (Mean)
Uproleselan + Standard of Care Melphalan18.42
Placebo + Standard of Care Melphalan18.46

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Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days

(NCT04682405)
Timeframe: Before conditioning and at day +14 or date of discharge (whichever is sooner) (up to be 18 days)

InterventionDays (Mean)
Uproleselan + Standard of Care Melphalan0.19
Placebo + Standard of Care Melphalan0.00

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Median Daily Dose of Pain Medications

The median daily dose of pain medications is provided as morphine equivalents. (NCT04682405)
Timeframe: Through date of discharge (up to be 18 days)

Interventionmg morphine equivalents (Median)
Uproleselan + Standard of Care Melphalan13.02
Placebo + Standard of Care Melphalan15.65

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Change in Hemorrhoids as Assessed Per CTCAE v5.0

Grade 0 hemorrhoids is defined as no hemorrhoids. Grade 1 hemorrhoids is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 hemorrhoids is defined as symptomatic; banding or medical intervention indicated. Grade 3 hemorrhoids is defined as severe symptoms; invasive intervention indicated. There is no grade 4 or 5 hemorrhoids defined in the CTCAE v5.0. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.000.000.000.000.000.000.000.040.080.080.080.040.080.080.040.080.090.09
Uproleselan + Standard of Care Melphalan0.000.000.000.040.040.080.080.120.120.150.080.080.150.120.150.160.040.08

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Change in Nausea as Assessed Per CTCAE v5.0

Grade 0 nausea is defined as no nausea. Grade 1 nausea is defined as loss of appetite without alteration in eating habits. Grade 2 nausea is defined as oral intake decreased without significant weight loss, dehydration or malnutrition. Grade 3 nausea is defined as inadequate oral caloric or fluid intake; tube feeding, TPN, or hospitalization indicated. There is no grade 4 or 5 nausea defined in the CTCAE v5.0. (NCT04682405)
Timeframe: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)

,
InterventionCTCAE grade (Mean)
Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6Day 7Day 8Day 9Day 10Day 11Day 12Day 13Day 14
Placebo + Standard of Care Melphalan0.080.080.210.420.631.130.921.171.291.331.421.211.381.421.291.000.830.59
Uproleselan + Standard of Care Melphalan0.000.000.120.350.621.041.001.000.921.231.351.231.191.080.650.620.650.20

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Time to First Antibiotics

Measured by the time in days to the first antibiotic dose for bacteremia. (NCT04682405)
Timeframe: Through date of discharge (up to be day 18)

Interventiondays (Median)
Uproleselan + Standard of Care Melphalan10.00
Placebo + Standard of Care Melphalan9.00

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Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available)

(NCT04682405)
Timeframe: Through date of discharge (upto be 18 days)

InterventionParticipants (Count of Participants)
Uproleselan + Standard of Care Melphalan8
Placebo + Standard of Care Melphalan5

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Time to Neutrophil Engraftment

-Defined as ANC ≥0.5 x 10^9/L for 3 consecutive days or ≥1.0 x 10^9/L for 1 day (NCT04682405)
Timeframe: Through date of discharge (up to be 18 days)

InterventionDays (Mean)
Uproleselan + Standard of Care Melphalan12.77
Placebo + Standard of Care Melphalan12.50

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Incidence of Clostridium Difficile Infections

(NCT04682405)
Timeframe: Through date of discharge (up to be 18 days)

InterventionParticipants (Count of Participants)
Uproleselan + Standard of Care Melphalan3
Placebo + Standard of Care Melphalan1

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