Page last updated: 2024-12-04

busulfan

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Cross-References

ID SourceID
PubMed CID2478
CHEMBL ID820
CHEBI ID28901
SCHEMBL ID4373
MeSH IDM0003079

Synonyms (228)

Synonym
AC-198
AB00051929-11
AB00051929-10
DIVK1C_000847
KBIO1_000847
NCI60_041640
NCIMECH_000192
bisulfex
busulfano
busulfanum
butane-1,4-diyl dimethanesulfonate
1,4-bis(methanesulfonoxy)butane
tetramethylene bis(methanesulfonate)
CHEBI:28901 ,
SPECTRUM_000092
PRESTWICK_989
tetramethylene {bis[methanesulfonate]}
4-methylsulfonyloxybutyl methanesulfonate
1, {4-bis[methanesulfonoxy]butane}
SPECTRUM5_000928
BSPBIO_001920
methanesulfonic acid, tetramethylene ester
NCGC00090905-01
NCGC00090905-02
1,4-butanediol, dimethanesulphonate
tetramethylenester kyseliny methansulfonove [czech]
1,4-butanediol, dimethanesulfonate
myelosanum
ai3-25012
einecs 200-250-2
busulfex
busulfanum [inn-latin]
busulfano [inn-spanish]
1,4-dimethanesulphonyloxybutane
1,4-butanediol dimethanesulphonate
brn 1791786
myeleukon
ccris 418
1,4-butanediyl dimethanesulfonate
milecitan
tetramethylene dimethane sulfonate
mablin
nsc-750
nsc 750
misulban
1,4-dimethylsulfonoxybutane
busulfan ,
1,4-butanediol dimethanesulfonate
mielosan
nci-c01592
myeloleukon
myleran
1,4-dimesyloxybutane
buzulfan
busulphan
1,4-dimethanesulfonoxybutane
mielucin
C06862
1,4-dimethanesulfonyloxybutane
sulfabutin
citosulfan
myelosan
mitostan
nsc750 ,
mielevcin
gt 2041
55-98-1
mileran
1,4-dimethylsulfonyloxybutane
g.t. 41
tetramethylene bis[methanesulfonate]
sulphabutin
1,4-bis(methanesulfonyloxy)butane
2041 c. b.
1,4-butanediol dimethylsulfonate
c.b. 2041
cb 2041
wln: ws1&o4osw1
gt 41
an 33501
1,4-bis[methanesulfonoxy]butane
x 149
mylecytan
leucosulfan
DB01008
D00248
busulfan (jp17/usp/inn)
IDI1_000847
NCGC00090905-04
NCGC00090905-05
NCGC00090905-03
NCGC00090905-06
KBIO2_003080
KBIO2_000512
KBIOGR_000698
KBIO2_005648
KBIOSS_000512
KBIO3_001420
SPECTRUM3_000320
SPECTRUM4_000259
SPBIO_000253
SPECTRUM2_000067
NINDS_000847
SPECTRUM1500152
NCGC00090905-07
MLS001076666
smr000058613
HMS2091O09
B1022
inchi=1/c6h14o6s2/c1-13(7,8)11-5-3-4-6-12-14(2,9)10/h3-6h2,1-2h3
covzyzsdywqreu-uhfffaoysa-
CHEMBL820
HMS502K09
FT-0663910
HMS1920I07
AKOS003614975
NCGC00090905-09
NCGC00090905-08
NCGC00090905-10
HMS3259G15
busulfan [usp:inn:ban:jan]
tetramethylenester kyseliny methansulfonove
g1ln9045dk ,
unii-g1ln9045dk
2041 c.b.
hsdb 7605
NCGC00259397-01
tox21_201848
NCGC00254038-01
tox21_300318
nsc755916
nsc-755916
pharmakon1600-01500152
tox21_111038
dtxcid10910
dtxsid3020910 ,
cas-55-98-1
HMS2233H04
CCG-35458
mitosan
busulphane
FT-0623291
NCGC00090905-11
busulfan [ep monograph]
busulfan [who-ip]
busulfan [jan]
busulfan [mi]
busulfan [who-dd]
busulfan [ep impurity]
busulfan [mart.]
busulfan [vandf]
busulfan [usp monograph]
tetramethylene di(methanesulfonate)
busulfan [inn]
busulfan fresenius kabi
busulfan [orange book]
busulfan [ema epar]
busulfanum [who-ip latin]
busulfan [hsdb]
myelosanum [who-ip]
busulfan [iarc]
busilvex
S1692
busulfex iv
4-(methanesulfonyloxy)butyl methanesulfonate
HMS3370E11
gtpl7136
HY-B0245
NC00498
SCHEMBL4373
tox21_111038_1
NCGC00090905-12
KS-5212
Z276508890
CB-2041 ,
methanesulfonic
4-[(methylsulfonyl)oxy]butyl methanesulfonate #
2041cb
busulfan;
AB00051929_12
AB00051929_14
mfcd00007562
bdbm50237623
(1,4-bis(methanesulfonyloxy)butane)
busulfan, analytical standard, for drug analysis
busulfan, european pharmacopoeia (ep) reference standard
SR-01000765405-3
SR-01000765405-2
sr-01000765405
SR-01000765405-7
HMS3655A21
busulfan; butane-1,4-diyl di(methanesulfonate)
SBI-0051300.P003
HMS3712A20
SW198555-3
busulfan/myleran
busulfan (myleran, busulfex)
Q348922
AMY33355
129316-96-7
D88731
butane-1,4-diyldimethanesulfonate
EN300-118686
SY029743
1,4-butanediol dimesylate
busulfan (iarc)
methanesulfonic acid tetramethylene ester
1,4-di(methylsulfonyloxy)butane
busulfan (ep monograph)
tetramethylene bis(methanesulphonate)
bussulfam
joacamine
misulfan
1,4-dimethanesulphonoxybutane
1,4-di(methanesulfonyloxy)butane
busulfan (mart.)
tetramethylenedimethanesulfonate
l01ab01
gt-41
busulfan (ep impurity)
methanesulphonic acid, tetramethylene ester
1,4-bis(methanesulphonoxy)butane
1,4-bitanediol dimethanesulfonate esters
bus
1, 4-bis(methanesulfonoxy)butane
busulfan (usp monograph)
wr-19508
1,4-dimethylsulphonoxy-butane

Research Excerpts

Overview

Busulfan is a bifunctional alkylating agent that is widely used before hematopoietic stem cell transplantation (HSCT), in combination with other chemotherapeutic drugs. Busulfan has been proven to possess toxic side effects on testicles.

ExcerptReferenceRelevance
"Busulfan (Bu) is an alkylating agent that significantly inhibits spermatogenesis."( Analysis of the Reversible Impact of the Chemodrug Busulfan on Mouse Testes.
Beaudoin, C; Caira, F; De Haze, A; Garcia, M; Holota, H; Monrose, M; Saru, JP; Thirouard, L; Volle, DH, 2021
)
1.59
"Busulfan is a bifunctional alkylating agent that is widely used before hematopoietic stem cell transplantation (HSCT), in combination with other chemotherapeutic drugs. "( Can Published Population Pharmacokinetic Models of Busulfan Be Used for Individualized Dosing in Chinese Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation? An External Evaluation.
Chen, S; Hu, J; Huang, H; Huang, W; Li, D; Lin, S; Liu, M; Ren, J; Wu, X; Yang, T, 2022
)
2.42
"Busulfan is an anti-cancer drug that leads to testicular problems in humans."( Antioxidant and anti-inflammatory protective effects of rutin and kolaviron against busulfan-induced testicular injuries in rats.
Abarikwu, SO; Amadi, BA; John, IG; Mgbudom-Okah, CJ; Njoku, RC; Onuah, CL, 2022
)
1.67
"Busulfan is a commonly used alkylating agent in the conditioning regimens of hematopoietic cell transplantation (HCT). "( Busulfan dose Recommendation in Inherited Metabolic Disorders: Population Pharmacokinetic Analysis.
Gupta, AO; Illamola, SM; Jennissen, CA; Long, SE; Long-Boyle, JR; Lund, TC; Orchard, PJ; Takahashi, T, 2022
)
3.61
"Busulfan (Bu) is an alkylating drug used in many preparative regimens before hematopoietic stem cell transplantation (HSCT). "( Impact of Glutathione S-Transferase Polymorphisms on Busulfan Pharmacokinetics and Outcomes of Hematopoietic Stem Cell Transplantation.
Al Balushi, K; Al-Hunaini, M; Al-Khabori, M; Al-Moundhri, M; Al-Rawahi, M; Al-Rawas, A; Al-Riyami, I; Al-Zadjali, S; Dennison, D, 2022
)
2.41
"Busulfan is an antineoplastic medication that is broadly utilized for cancer treatment. "( Ellagic acid effects on testis, sex hormones, oxidative stress, and apoptosis in the relative sterility rat model following busulfan administration.
Aghajari, ZA; Jaberi, KR; Jahromi, BN; Khorchani, MJ; Koohpeyma, F; Mahmoodi, M; Mahmoudikohani, F; Movahedpour, A; Noroozi, S; Rostami, A; Saki, F; Vakili, S, 2022
)
2.37
"Busulfan (Bus), is an alkylating agent widely used in chemotherapy which has been proven to possess toxic side effects on testicles. "( Ameliorate effects of resveratrol and l-carnitine on the testicular tissue and sex hormones level in busulfan induced azoospermia rats.
Forouzanfar, M; Hafezi, H; Shariatic, M; Vahdati, A, 2022
)
2.38
"Busulfan is an alkylating agent commonly used in cancer chemotherapy. "( Mechanisms underlying impaired spermatogenic function in orchitis induced by busulfan.
Dong, Z; Wang, D; Xu, S; Zhao, J; Zhao, L, 2023
)
2.58
"Busulfan (Bu) is an alkylating agent commonly used at high doses in the preparative regimens of hematopoietic stem cell transplantation (HSCT). "( Impact of valproic acid on busulfan pharmacokinetics: In vitro assessment of potential drug-drug interaction.
Al-Enezi, BF; Al-Hasawi, N; Matar, KM, 2023
)
2.65
"Busulfan is an alkylating agent widely used in the conditioning of hematopoietic stem cell transplantation possessing a complex metabolism and a large interindividual and intra-individual variability, especially in children. "( Individualizing busulfan dose in specific populations and evaluating the risk of pharmacokinetic drug-drug interactions.
Combarel, D; Delahousse, J; Paci, A; Tran, J; Vassal, G, 2023
)
2.7
"Busulfan (BU) is an alkylating agent used as a conditioning agent prior to hematopoietic stem cell (HSC) transplantation as it is known to be cytotoxic to host hematopoietic stem and progenitor cells. "( Screening for urinary markers predicting hematopoietic stem cell injury induced by busulfan using genetically diverse mice.
Gao, R; Guan, B; Liu, X; Meng, A; Sun, Y; Wang, X; Zhang, L, 2023
)
2.58
"Busulfan is an alkylating agent used as part of conditioning chemotherapy regimens prior to allogeneic hematopoietic cell transplant (allo-HCT). "( Utilization of a Population Pharmacokinetic Model to Improve a Busulfan Test-Dose Strategy in Allogeneic Hematopoietic Cell Transplant Recipients.
Armistead, PM; Crona, DJ; DeVane, SC; Dunlap, TC; Kardouh, M; Kemper, RM; Ptachcinski, JR; Shaw, JR; Symonds, A; Weiner, DL, 2023
)
2.59
"Busulfan (BUS) is an anticancer agent with serious adverse effects on various body organs, including the lung and testis. "( Sitagliptin ameliorates busulfan-induced pulmonary and testicular injury in rats through antioxidant, anti-inflammatory, antifibrotic, and antiapoptotic effects.
Abbas, MA; Ali, EA; Tayel, SG, 2023
)
2.66
"Busulfan is an alkylating drug routinely used in conditioning regimens for allogeneic hematopoietic cell transplantation (allo-HCT). "( Association between busulfan exposure and survival in patients undergoing a CD34+ selected stem cell transplantation.
Boelens, JJ; Boulad, F; Cancio, MI; Cho, C; Curran, KJ; Devlin, S; Flynn, J; Giralt, SA; Gyurkocza, B; Jakubowski, AA; Kernan, NA; Klein, E; Kung, AL; Kunvarjee, BM; Lin, A; O'Reilly, RJ; Papadopoulos, EB; Perales, MA; Prockop, S; Proli, A; Scaradavou, A; Scordo, M; Shaffer, BC; Shah, G; Spitzer, B; Tamari, R, 2023
)
2.68
"Busulfan is a widely used cancer chemotherapeutic agent. "( The impact of busulfan on the testicular structure in prepubertal rats: A histological, ultrastructural and immunohistochemical study.
Abd El-Hay, RI; Gawish, SA; Hamed, WHE; Mostafa Omar, N; Refat El-Bassouny, D, 2023
)
2.71
"Busulfan is an alkylating agent and functions as a myeloablative and anti-leukemic chemotherapy drug. "( Liquid Chromatography-Tandem Mass Spectrometry Method for the Quantification of Plasma Busulfan.
Clinton Frazee, C; Garg, U; Munar, A, 2024
)
3.11
"Busulfan is a bifunctional alkylating agent used for myeloablative conditioning and in the treatment of chronic myeloid leukemia due to its ability to cause DNA damage. "( Maternal exposure to busulfan reduces the cell number in the somatosensory cortex associated with delayed somatic and reflex maturation in neonatal rats.
Albuquerque, G; Costa-de-Santana, BJR; Gouveia, HJCB; Lacerda, DC; Manhães-de-Castro, R; Mendonça, CR; Toscano, AE; Visco, DB, 2020
)
2.32
"Busulfan (Bu) is a key component of several conditioning regimens used before hematopoietic stem cell transplantation (HSCT). "( Busulfan systemic exposure and its relationship with efficacy and safety in hematopoietic stem cell transplantation in children: a meta-analysis.
Fan, D; Feng, X; Li, J; Wu, Y; Yang, C; Zhang, J; Zhao, L; Zhu, G, 2020
)
3.44
"Busulfan (Bu) is an alkylating agent commonly used in preparative regimens for hematologic malignant and non-malignant patients undergoing hematopoietic stem cell transplantation (HSCT). "( UPLC-Tandem Mass Spectrometry for Quantification of Busulfan in Human Plasma: Application to Therapeutic Drug Monitoring.
Alshemmari, SH; Anwar, A; Matar, KM; Refaat, S, 2020
)
2.25
"Busulfan is an alkylating agent routinely used in conditioning regimens prior to allogeneic hematopoietic cell transplantation (HCT) for various nonmalignant disorders, including inborn errors of metabolism. "( Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients.
Dvorak, CC; Kharbanda, S; Long-Boyle, J; Shukla, P, 2020
)
2.33
"Busulfan is an alkylating agent used in chemotherapy conditioning regimens prior to hematopoietic stem cell transplantation (HSCT). "( Electrophilic reactivity of the Busulfan metabolite, EdAG, towards cellular thiols and inhibition of human thioredoxin-1.
Dao, N; Hoang, S; Myers, AL, 2020
)
2.28
"Busulfan (BU) is an alkylating agent with a narrow therapeutic index and high intraindividual pharmacokinetic variability used in conditioning therapy for hematopoietic stem cell transplantation. "( Dried Plasma Spots and Oral Fluid as Alternative Matrices for Therapeutic Drug Monitoring of Busulfan: Analytical Method Development and Clinical Evaluation.
Antunes, MV; da Silva, ACC; Granzotto, FCN; Linden, R; Lizot, LF, 2021
)
2.28
"Busulfan (Bu) is an old drug, but is still well recommended as an alkylating agent during conditioning therapy, before hematopoietic stem cell transplantation. "( Comparison of Two Analytical Methods for Busulfan Therapeutic Drug Monitoring.
Bartoli, A; De Gregori, S; Manzoni, F; Tinelli, C, 2021
)
2.33
"Busulfan is an alkylating chemotherapeutic agent that is routinely prescribed for leukemic patients to induce myelo-ablation. "( The Therapeutic Potential of Amniotic Fluid-Derived Stem Cells on Busulfan-Induced Azoospermia in Adult Rats.
El Mulla, KF; Ibrahim, HF; Medwar, AY; Safwat, SH; Zeitoun, TM, 2021
)
2.3
"Busulfan is an alkylating agent used in allogeneic hematopoietic stem cell transplantation for various malignant and nonmalignant disorders. "( Evaluation of a Nanoparticle-Based Busulfan Immunoassay for Rapid Analysis on Routine Clinical Analyzers.
Baburina, I; Courtney, JB; Gardiner, J; Gill, RV; Hilaire, MR; Meng, QH; Milone, MC; Salamone, SJ; Shaw, LM, 2021
)
2.34
"Busulfan (BSU) is a chemotherapeutic drug that can cause subfertility or sterility in males. "( Impaired spermatogenesis caused by busulfan is partially ameliorated by treatment with conditioned medium of adipose tissue derived mesenchymal stem cells.
Abbaszadeh, HA; Abdi, S; Abdollahifar, MA; Aliaghaei, A; Azad, N; Ebrahimi, V; Fadaei Fathabadi, F; Faraji Sani, M; Ghanimat, F; Movahedi, M; Raoofi, A, 2022
)
2.44
"Busulfan is a chemotherapy agent used in hematopoietic stem cell transplant (HSCT) conditioning regimens. "( Comparison of levetiracetam versus phenytoin/fosphenytoin for busulfan seizure prophylaxis at a pediatric institution.
Garrity, L; Hughes, K; Lane, A; Nelson, AS; Teusink-Cross, A, 2021
)
2.3
"Busulfan (Bu) is an alkylating agent routinely used for conditioning regimens before allogeneic stem cell transplantation (allo-SCT). "( Feasibility and Efficacy of a Pharmacokinetics-Guided Busulfan Conditioning Regimen for Allogeneic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis in Adult Patients with Hematologic Malignancie
Bartoli, A; Bramanti, S; Castagna, L; De Gregori, S; De Philippis, C; Giordano, L; Mannina, D; Mariotti, J; Pieri, G; Roperti, M; Sarina, B; Valli, V, 2021
)
2.31
"Busulfan is a popular antileukemia chemotherapeutic alkylating agent widely known to induce variety of serious adverse effects including chemobrain-related cognitive impairments and dysfunction in male reproductive system. "( Kolaviron abates busulfan-induced episodic memory deficit and testicular dysfunction in rats: The implications for neuroendopathobiological changes during chemotherapy.
Adebayo, OG; Atuadu, V; Ben-Azu, B; Edesiri, TP; Nwangwa, EK; Oyovwi, MO; Ozegbe, QEB; Rotu, RA; Victor, E, 2021
)
2.4
"Busulfan (Bu) is a common component of conditioning regimens before hematopoietic stem cell transplantation (HSCT) and is known for high interpatient pharmacokinetic (PK) variability. "( Precision dosing of intravenous busulfan in pediatric hematopoietic stem cell transplantation: Results from a multicenter population pharmacokinetic study.
Ansari, M; Ben Hassine, K; Bittencourt, H; Bredius, RGM; Daali, Y; Kassir, N; Krajinovic, M; Lewis, V; Nath, CE; Nava, T; Shaw, PJ; Théoret, Y; Uppugunduri, CRS, 2021
)
2.35
"Busulfan is a cytotoxic agent used in preconditioning for hematopoietic stem cell transplantation. "( A Significant Influence of Metronidazole on Busulfan Pharmacokinetics: A Case Report of Therapeutic Drug Monitoring.
Choi, JY; Chung, H; Hong, CR; Hong, KT; Kang, HJ; Lee, S; Park, KD; Shin, HY; Yu, KS, 2017
)
2.16
"Busulfan is an anticancer drug caused variety of adverse effects for patients with cancer. "( Protective effect of L-carnitine and L-arginine against busulfan-induced oligospermia in adult rat.
Abd-Elrazek, AM; Ahmed-Farid, OAH, 2018
)
2.17
"Busulfan is a chemotherapeutic agent used to treat chronic myelogenous leukemia and other myeloproliferative disorders. "( Increased Sat2 expression is associated with busulfan-induced testicular Sertoli cell injury.
Hao, J; Li, G; Liao, X; Liu, Y; Wu, M; Wu, Y; Xian, Y, 2017
)
2.16
"Busulfan (Bu) is an alkylating agent with a limited therapeutic margin and exhibits inter-patient variability in pharmacokinetics (PK). "( Clarifying busulfan metabolism and drug interactions to support new therapeutic drug monitoring strategies: a comprehensive review.
Andersson, BS; Champlin, RE; Ghose, R; Kawedia, JD; Kramer, MA; Myers, AL; Nieto, Y, 2017
)
2.29
"Busulfan (Bu) is a key component of conditioning regimens used before hematopoietic stem cell transplantation (SCT) in children. "( GSTA1 Genetic Variants and Conditioning Regimen: Missing Key Factors in Dosing Guidelines of Busulfan in Pediatric Hematopoietic Stem Cell Transplantation.
Ansari, M; Bittencourt, H; Curtis, PH; Daudt, LE; Duval, M; Krajinovic, M; Nava, T; Rezgui, MA; Théoret, Y; Uppugunduri, CRS, 2017
)
2.12
"Busulfan is an alkylating agent used in pre-transplant conditioning for patients undergoing hematopoietic stem cell transplantation. "( A novel drug interaction between busulfan and blinatumomab.
Lee, J; Oh, A; Patel, P; Quigley, JG; Rondelli, D; Sweiss, K; Ye, R, 2019
)
2.24
"Busulfan is an alkane sulphonate currently used as an anticancer drug and to prepare azoospermic animal models, because it selectively destroys differentiated spermatogonia in the testes. "( Busulfan administration produces toxic effects on epididymal morphology and inhibits the expression of ZO-1 and vimentin in the mouse epididymis.
Cai, Y; Fang, F; Ni, K; Shang, J; Shen, S; Xiong, C; Zhang, X; Zhao, Q, 2017
)
3.34
"Busulfan (Bu) is an alkylating agent used as part of the conditioning regimen in pediatric patients before hematopoietic stem cell transplantation. "( Therapeutic Drug Monitoring of Busulfan for the Management of Pediatric Patients: Cross-Validation of Methods and Long-Term Performance.
Ansari, M; Chalandon, Y; Choong, E; Daali, Y; Doffey-Lazeyras, F; Kuntzinger, M; Lo Piccolo, R; Marino, D; Peters, C; Uppugunduri, CRS, 2018
)
2.21
"Busulfan is a major component of chemotherapy conditioning in hematopoietic cell transplantation (HCT). "( Retrospective study of the digestive tract mucositis derived from myeloablative and non-myeloablative/reduced-intensity conditionings with busulfan in hematopoietic cell transplantation patient.
Bezinelli, LM; Carvalho, DLC; Corrêa, L; da Silva, CC; Eduardo, FP; Ferreira, MH; Gobbi, M; Hamerschlak, N; Rosin, FCP, 2019
)
2.16
"Busulfan is an anti-cancer chemotherapeutic drug and is often used as conditioning regimens prior to bone marrow transplant for treatment of chronic myelogenous leukemia. "( Gosha-Jinki-Gan Recovers Spermatogenesis in Mice with Busulfan-Induced Aspermatogenesis.
Hayashi, S; Hirayanagi, Y; Itoh, M; Kuramasu, M; Nagahori, K; Naito, M; Ogawa, Y; Qu, N; Sakabe, K; Suyama, K; Terayama, H, 2018
)
2.17
"Busulfan is a widely used chemotherapeutic drug for chronic myelogenous leukemia and bone marrow transplantation. "( Melatonin protects spermatogonia from the stress of chemotherapy and oxidation via eliminating reactive oxidative species.
Lin, Z; Mi, J; Song, H; Sun, Z; Wei, R; Xia, Q; Yang, Y; Zhang, X; Zou, K, 2019
)
1.96
"Busulfan (Bu) is a DNA-alkylating agent used for myeloablative conditioning in stem cell transplantation in children and adults. "( Evaluation of effects of busulfan and DMA on SOS in pediatric stem cell recipients.
Bartelink, I; Boelens, J; Boos, J; Diestelhorst, C; Hempel, G; Kerl, K; Trame, MN, 2014
)
2.15
"Busulfan is a chemotherapeutic drug that induces sterility, azoospermia and testicular atrophy. "( Regeneration of spermatogenesis in a mouse model of azoospermia by follicle-stimulating hormone and oestradiol.
Akhondi, MM; Jafarian, A; Lakpour, N; Pejhan, N; Sadeghi, MR; Salehkhou, S, 2014
)
1.85
"Busulfan is an anti-leukemic, DNA alkylating agent that is used in conditioning regimens for patients undergoing hematopoietic stem cell transplantation. "( Development and validation of a liquid chromatography-tandem mass spectrometry assay to quantify plasma busulfan.
French, D; Long-Boyle, JR; Ritchie, JC; Sujishi, KK, 2014
)
2.06
"Busulfan (Bu) is an integral part of conditioning regimens for patients with sickle cell anemia (SCA) undergoing transplantation. "( New insights into the pharmacokinetics of intravenous busulfan in children with sickle cell anemia undergoing bone marrow transplantation.
Alfieri, C; Andreani, M; De Angelis, G; Dinallo, V; Gallucci, C; Gaziev, J; Ialongo, C; Isgrò, A; Lucarelli, G; Marziali, M; Mozzi, AF; Nguyen, L; Paciaroni, K; Petain, A; Ribersani, M; Sodani, P; Testi, M, 2015
)
2.11
"Busulfan is an alkylating agent used for conditioning patients undergoing hematopoietic stem cell transplantation with a narrow therapeutic range and highly variable pharmacokinetics. "( Individualizing oral busulfan dose after using a test dose in patients undergoing hematopoietic stem cell transplantation: pharmacokinetic characterization.
Bariani, C; Carneiro, WJ; Cunha, LC; de Moraes Arantes, A; de Oliveira Neto, JR; Effting, C; Queiroz Labre, LV; Rodrigues, AR; Rodrigues, CR, 2015
)
2.18
"Busulfan is an alkylating agent used to ablate bone marrow cells before hematopoietic stem cell transplantation. "( High-Throughput Validated Method for the Quantitation of Busulfan in Plasma Using Ultrafast SPE-MS/MS.
Danso, D; Enger, R; Jannetto, PJ; Langman, LJ, 2015
)
2.1
"Busulfan is a commonly used chemotherapeutic agent in myeloablative conditioning regimens for allogeneic hematopoietic cell transplantation (allo-HCT). "( Subacute hepatic necrosis mimicking veno-occlusive disease in a patient with HFE H63D homozygosity after allogeneic hematopoietic cell transplantation with busulfan conditioning.
Boyer, MW; Chen, S; Chen, X; Hildebrandt, GC; McDonald, GB; Osborn, JD, 2015
)
2.06
"Busulfan is a chemotherapy drug that has side effects on spermatogonial stem cells (SSC). "( Gonadotoxic effects of busulfan in two strains of mice.
Bordignon, V; Chemeris, RO; Comim, FV; Glanzner, WG; Gonçalves, PB; Gutierrez, K; Rigo, ML, 2016
)
2.19
"Busulfan is an alkylating agent widely used in the ablation of bone marrow cells before hematopoietic stem cell transplant. "( A Simple Liquid Chromatography Tandem Mass Spectrometry Method for Quantitation of Plasma Busulfan.
Abdel-Rahman, S; Dalal, J; Deng, S; Frazee, C; Garg, U; Kiscoan, M, 2016
)
2.1
"Busulfan is a commonly used antineoplastic agent to condition/ablate bone marrow cells before hematopoietic stem cell transplant. "( High-Throughput Quantitation of Busulfan in Plasma Using Ultrafast Solid-Phase Extraction Tandem Mass Spectrometry (SPE-MS/MS).
Danso, D; Jannetto, PJ; Langman, LJ; Robert, E, 2016
)
2.16
"Busulfan (Bu) is a key component of the conditioning regimen prior to HSCT."( Influence of glutathione S-transferase gene polymorphisms on busulfan pharmacokinetics and outcome of hematopoietic stem-cell transplantation in thalassemia pediatric patients.
Ansari, M; Bittencourt, H; Cappelli, B; Duval, M; Huezo-Diaz, P; Krajinovic, M; Marktel, S; Rezgui, MA, 2016
)
1.4
"Busulfan (BU) is a key compound in conditioning myeloablative regimens for children undergoing hematopoietic stem cell transplantation (HSCT). "( Influence of GST gene polymorphisms on busulfan pharmacokinetics in children.
Ansari, M; Champagne, MA; Duval, M; Krajinovic, M; Lauzon-Joset, JF; Théoret, Y; Vachon, MF, 2010
)
2.07
"Busulfan (Bu) is a DNA-alkylating drug used in myeloablative pretransplant conditioning therapy for patients with myeloid leukemia (ML). "( 5-Aza-2'-deoxycytidine sensitizes busulfan-resistant myeloid leukemia cells by regulating expression of genes involved in cell cycle checkpoint and apoptosis.
Andersson, BS; Champlin, RE; Corn, P; Li, Y; Murray, D; Valdez, BC, 2010
)
2.08
"Busulfan is a chemotherapy drug widely used as part of conditioning regimens for patients undergoing bone marrow transplantation (BMT). "( Quantification of busulfan in plasma using liquid chromatography electrospray tandem mass spectrometry (HPLC-ESI-MS/MS).
Ritchie, JC; Snyder, ML, 2010
)
2.14
"Busulfan is a chemotherapeutic agent commonly used for myeloablative conditioning regimens such as in the treatment of chronic myelogenous leukemia. "( An automated method for supporting busulfan therapeutic drug monitoring.
Johnson-Davis, KL; Juenke, JM; McMillin, GA; Miller, KA, 2011
)
2.09
"Busulfan is a bifunctional alkylating agent widely used in pretransplant conditioning regimens in patients undergoing stem cell transplantation for hematologic malignancies. "( Busulfan and metronidazole: an often forgotten but significant drug interaction.
Andersson, BS; Culotta, KS; Gulbis, AM; Jones, RB, 2011
)
3.25
"Busulfan is an alkylating agent widely used in chemotherapy, but with severe side effects. "( Porous metal organic framework nanoparticles to address the challenges related to busulfan encapsulation.
Ben Yahia, M; Chalati, T; Couvreur, P; Gref, R; Horcajada, P; Maurin, G; Serre, C, 2011
)
2.04
"Busulfan (Bu) is an important component of the myeloablative conditioning regimen prior to hematopoietic stem cell transplantation (HSCT) especially in children. "( A simplified method for busulfan monitoring using dried blood spot in combination with liquid chromatography/tandem mass spectrometry.
Ansari, M; Daali, Y; Dayer, P; Déglon, J; Desmules, J; Gumy-Pause, F; Ozsahin, H; Théorêt, Y; Uppugunduri, CR; Versace, F, 2012
)
2.13
"Busulfan (Bu) is an important component of some myeloablative regimens prior to stem cell transplantation (SCT). "( The effect of metronidazole on busulfan pharmacokinetics in patients undergoing hematopoietic stem cell transplantation.
Aschan, J; Hassan, M; Hentschke, P; Ljungman, P; Nilsson, C; Ringdén, O, 2003
)
2.05
"Busulfan is a common component of pretransplant conditioning but has an erratic and unpredictable bioavailability when administered orally."( Intravenous busulfan-based conditioning prior to allogeneic hematopoietic stem cell transplantation: myeloablation with reduced toxicity.
Avigdor, A; Ben-Bassat, I; Bielorai, B; Hardan, I; Nagler, A; Shimoni, A; Toren, A; Yeshurun, M, 2003
)
1.42
"Busulfan is an alkylating agent widely used in combination chemotherapy regimens followed by allogeneic or autologous hematopoietic stem cell transplantation (HSCT). "( High-performance thin-layer chromatography with a derivatization procedure, a suitable method for the identification and the quantitation of busulfan in various pharmaceutical products.
Bouligand, J; Bourget, P; Mercier, L; Paci, A; Vassal, G, 2004
)
1.97
"Busulfan is an alkylating agent used in preparative regimens before bone marrow transplantation (BMT). "( A simple approximation for busulfan dose adjustment in adult patients undergoing bone marrow transplantation.
Akria, L; Hoffer, E; Krivoy, N; Rowe, JM; Scherb, I; Tabak, A, 2004
)
2.06
"Busulfan is an alkylizing agent used instead of radiotherapy for pre-graft preparation before bone marrow grafts in children."( [Busulfan and cycosporin in bone graft children].
Aulagner, G; Bertolle, V; Bleyzac, N; Martin, P, 2004
)
1.96
"Busulfan (BUS) is a highly toxic antineoplastic bifunctional-alkylating agent and has a narrow therapeutic window. "( Kinetic of genotoxic expression in the pharmacodynamics of busulfan.
Cruz-Vallejo, VL; Mendiola-Cruz, MT; Miranda-Pasaye, S; Morales-Ramírez, P; Vallarino-Kelly, T, 2006
)
2.02
"Busulfan is an effective myeloablative conditioning agent for HSCT. "( Pretransplant conditioning in adults and children: dose assurance with intravenous busulfan.
Barnes, YJ; Fisher, VL; Nuss, SL, 2006
)
2
"IV busulfan is a convenient, safe, and effective conditioning agent used in HSCT that has a predictable pharmacokinetic profile."( Pretransplant conditioning in adults and children: dose assurance with intravenous busulfan.
Barnes, YJ; Fisher, VL; Nuss, SL, 2006
)
1.18
"Busulfan (BU) is a unique alkylating agent that primarily targets slowly proliferating or nonproliferating cells in the body, leading to various normal tissue damage while killing leukemia cells. "( Busulfan selectively induces cellular senescence but not apoptosis in WI38 fibroblasts via a p53-independent but extracellular signal-regulated kinase-p38 mitogen-activated protein kinase-dependent mechanism.
Bai, A; Probin, V; Wang, Y; Zhou, D, 2006
)
3.22
"The busulfan is an alkylating agent widely used for the treatment of haematological malignancies and nonmalignant disorders. "( Busulfan loading into poly(alkyl cyanoacrylate) nanoparticles: physico-chemistry and molecular modeling.
Aymes-Chodur, C; Chacun, H; Couvreur, P; Ghermani, NE; Gref, R; Layre, AM; Poupaert, J; Requier, D; Richard, J, 2006
)
2.33
"Busulfan is an example of a drug eliminated through glutathione S-transferase (GST)-catalyzed conjugation with reduced glutathione (GSH). "( Induction of glutathione synthesis explains pharmacodynamics of high-dose busulfan in mice and highlights putative mechanisms of drug interaction.
Bouligand, J; Connault, E; Daudigeos, E; Deroussent, A; Morizet, J; Opolon, P; Paci, A; Re, M; Simonnard, N; Vassal, G, 2007
)
2.01
"Busulfan is an alkylating agent used in a conditioning regimen prior to bone marrow transplantation. "( Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study.
Aulagner, G; Bertholle-Bonnet, V; Bertrand, Y; Bleyzac, N; Galambrun, C; Mialou, V; Souillet, G, 2007
)
2.06
"Busulfan is a useful, sufficiently safe drug in the treatment of patients with CML. "( Busulfan.
Buggia, I; Locatelli, F; Regazzi, MB; Zecca, M, 1994
)
3.17
"Busulfan+melphalan is a safe and feasible conditioning regimen for APBSCT in MM with acceptable toxicity and a high objective response rate, which may result in prolonged survival."( Busulfan and melphalan as conditioning regimen for autologous peripheral blood stem cell transplantation in multiple myeloma.
Alegre, A; Arranz, R; Cámara, R; Casado, F; Fernández-Villalta, MJ; Figuera, A; Lamana, M; Requena, MJ; Steegman, JL; Tomás, JF, 1995
)
2.46
"Busulfan is a GST substrate with a high K(m) relative to concentrations achieved clinically (1-8 microM)."( Busulfan-glutathione conjugation catalyzed by human liver cytosolic glutathione S-transferases.
Czerwinski, M; Gibbs, JP; Slattery, JT, 1996
)
2.46
"Busulfan (BU) is an alkylating drug frequently used to prepare patients for bone marrow transplantation (BMT). "( Itraconazole can increase systemic exposure to busulfan in patients given bone marrow transplantation. GITMO (Gruppo Italiano Trapianto di Midollo Osseo).
Alessandrino, EP; Bosi, A; Buggia, I; Dallorso, A; Locatelli, F; Majolino, I; Pession, A; Regazzi, MB; Rosti, G; Rotoli, B; Zecca, M,
)
1.83
"Busulfan is an alkylating agent commonly used in the treatment of chronic myelogenous leukemia and in combination with cyclophosphamide in preparation for allogeneic bone marrow transplantation. "( Characterization of the mechanisms of busulfan resistance in a human glioblastoma multiforme xenograft.
Ali-Osman, F; Bigner, DD; Colvin, OM; Elion, GB; Friedman, HS; Griffith, OW; Hare, CB; Keir, S; Marcelli, SL; Petros, WP, 1997
)
2.01
"Busulfan is an alkylating agent commonly used to ablate marrow before hematopoietic stem cell transplantation. "( Therapeutic monitoring of busulfan in hematopoietic stem cell transplantation.
Risler, LJ; Slattery, JT, 1998
)
2.04
"Busulfan (BU) is a widely used alkylating agent for antineoplastic therapy and marrow ablation in preparation for bone marrow transplantation (BMT). "( Routine analysis of plasma busulfan by gas chromatography-mass fragmentography.
Lai, WK; Law, LK; Li, CK; Pang, CP; Wong, R; Yuen, PM, 1998
)
2.04
"Busulfan is a carcinostatic which is used for myelocytic leukemia. "( Cataract induced by short-term administration of large doses of busulfan: a case report.
Amemiya, T; Kaida, T; Ogawa, T, 1999
)
1.98
"Busulfan is an alkylating agent currently used in the myeloablative conditioning regimen before stem cell transplantation. "( The pharmacodynamic effect of busulfan in the P39 myeloid cell line in vitro.
Hassan, M; Hassan, Z; Hellström-Lindberg, E, 2001
)
2.04
"Busulfan is a frequently used chemotherapeutic agent in conditioning regimens prior to transplantations."( [High-dose busulfan--monitoring plasma levels and dosage adjustments in adults].
Kalous, O; Malásková, L; Mayer, J, 2001
)
1.42
"Busulfan (BU) is a widely used myeloablative and antineoplastic agent in clinical bone marrow transplantation (BMT). "( Optimization of busulfan dosage in children undergoing bone marrow transplantation: a pharmacokinetic study of dose escalation.
Graham, ML; Grochow, LB; Jones, RJ; Santos, GW; Wagner, JE; Yeager, AM, 1992
)
2.07
"Busulfan is an alkylating agent that is widely used in preparative regimens for bone marrow transplantation (BMT). "( Pharmacokinetics of busulfan: correlation with veno-occlusive disease in patients undergoing bone marrow transplantation.
Braine, HG; Brundrett, RB; Chen, TL; Colvin, OM; Grochow, LB; Jones, RJ; Santos, GW; Saral, R, 1989
)
2.04
"Busulfan is a bifunctional alkylating agent that appears to be cytotoxic to slowly proliferating or non-proliferating stem cell compartments, although its specific molecular and cellular mechanisms are unknown. "( Toxicological review of busulfan (Myleran).
Bishop, JB; Wassom, JS, 1986
)
2.02

Effects

Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU. Busulfan has a very narrow therapeutic index, and acute toxicity may be related to absorption and disposition of the drug and metabolites.

Busulfan has been shown to be mutagenic to microorganisms, mammalian cells in culture, Drosophila, and rodents. Busulfan exposure has previously been linked to clinical outcomes, hence the need for therapeutic drug monitoring (TDM)

ExcerptReferenceRelevance
"Busulfan has a narrow therapeutic window and a well-established exposure-response relationship with important clinical outcomes."( Population Pharmacokinetic Model of Intravenous Busulfan in Hematopoietic Cell Transplantation: Systematic Review and Comparative Simulations.
Al-Kofahi, M; Brown, SJ; Jaber, MM; Takahashi, T, 2023
)
1.89
"Busulfan has a narrow therapeutic index and its concentration was found to correlate with VOD."( Busulfan clearance does not predict the development of hepatic veno-occlusive disease in patients undergoing hematopoietic stem cell transplantation.
Al-Huneini, M; Al-Khabori, M; Al-Rawas, A; Al-Za'abi, M; Dennison, D; Salman, B, 2020
)
2.72
"Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU as part of the preparative regimen for allogeneic transplants. "( Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant.
Brown, V; Bruce, K; Calder, C; Domm, J; Evans, M; Frangoul, H; Ho, R; Kassim, A; Maheshwari, S; Manes, B; Yang, E; Yeh, RF, 2014
)
2.26
"Busulfan has a very narrow therapeutic index, and acute toxicity may be related to absorption and disposition of the drug and metabolites."( Busulfan use in hematopoietic stem cell transplantation: pharmacology, dose adjustment, safety and efficacy in adults and children.
Bentur, Y; Hoffer, E; Krivoy, N; Lurie, Y; Rowe, JM, 2008
)
2.51
"Busulfan has a narrow therapeutic range, and in children, pharmacokinetic variability has been found to be high even after the use of intravenous (i.v.) busulfan. "( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
2.06
"Busulfan has a narrow therapeutic index, giving rise to major liver toxicity (veno-occlusive disease), and a wide interpatient and intrapatient pharmacokinetic variability."( Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study.
Aulagner, G; Bertholle-Bonnet, V; Bertrand, Y; Bleyzac, N; Galambrun, C; Mialou, V; Souillet, G, 2007
)
1.34
"Busulfan exposure has previously been linked to clinical outcomes, hence the need for therapeutic drug monitoring (TDM). "( Busulfan target exposure attainment in children undergoing allogeneic hematopoietic cell transplantation: a single day versus a multiday therapeutic drug monitoring regimen.
Bartelink, IHI; Bognàr, TT; de Kanter, CTMK; Egberts, ACGT; Kingma, JSJ; Lalmohamed, AA; Lindemans, CAC; Smeijsters, EHE; van der Elst, KCMK, 2023
)
3.8
"Busulfan has a narrow therapeutic window and a well-established exposure-response relationship with important clinical outcomes."( Population Pharmacokinetic Model of Intravenous Busulfan in Hematopoietic Cell Transplantation: Systematic Review and Comparative Simulations.
Al-Kofahi, M; Brown, SJ; Jaber, MM; Takahashi, T, 2023
)
1.89
"busulfan (Bu) has been the main conditioning regimens for allogeneic hematopoietic cell transplantation (alloHCT) for young patients with acute myelogenous leukemia (AML) eligible for a myeloablative conditioning (MAC) regimen."( Long-Term Outcomes of Patients with Acute Myelogenous Leukemia Treated with Myeloablative Fractionated Total Body Irradiation TBI-Based Conditioning with a Tacrolimus- and Sirolimus-Based Graft-versus-Host Disease Prophylaxis Regimen: 6-Year Follow-Up fro
Al Malki, MM; Aldoss, I; Ali, H; Aribi, A; Armenian, S; Budde, E; Dadwal, S; Dandapani, S; Forman, SJ; Hui, S; Khaled, S; Marcucci, G; Mei, M; Mokhtari, S; Murata-Collins, J; Nakamura, R; Peng, K; Pullarkat, V; Salhotra, A; Sandhu, KS; Snyder, D; Spielberger, R; Stein, A; Teh, JB; Wong, J; Yang, D, 2020
)
1.28
"Busulfan has a narrow therapeutic index and its concentration was found to correlate with VOD."( Busulfan clearance does not predict the development of hepatic veno-occlusive disease in patients undergoing hematopoietic stem cell transplantation.
Al-Huneini, M; Al-Khabori, M; Al-Rawas, A; Al-Za'abi, M; Dennison, D; Salman, B, 2020
)
2.72
"Busulfan (Bu) has been used in combination with fludarabine (Flu; BuFlu) or cyclophosphamide (Cy; BuCy) as conditioning for allogeneic hematopoietic stem cell transplantation (HSCT). "( Pharmacokinetics of intravenous busulfan as condition for hematopoietic stem cell transplantation: comparison between combinations with cyclophosphamide and fludarabine.
Hino, Y; Kato, J; Kikuchi, T; Koda, Y; Matsumoto, K; Mishina, T; Mori, T; Morita, K; Nagao, Y; Ohwada, C; Okamoto, S; Onizuka, M; Onoda, M; Sakaida, E; Shimizu, H; Shono, K; Takeda, Y; Yokota, A; Yokoyama, H, 2021
)
2.35
"Busulfan has been commonly used to develop such a model, but 30%-87% of mice die when administered an intraperitoneal injection of 40 mg kg"( Establishing a nonlethal and efficient mouse model of male gonadotoxicity by intraperitoneal busulfan injection.
Chen, HC; Deng, CC; Deng, CH; Li, XY; Liu, GH; Lv, LY; Ouyang, B; Sun, XZ; Xie, Y; Yao, JH; Zhang, C,
)
1.07
"Busulfan (BU) has a narrow therapeutic window and the average concentration of BU at steady state (Css) is critical for successful engraftment in children receiving BU as part of the preparative regimen for allogeneic transplants. "( Targeted Busulfan therapy with a steady-state concentration of 600-700 ng/mL in patients with sickle cell disease receiving HLA-identical sibling bone marrow transplant.
Brown, V; Bruce, K; Calder, C; Domm, J; Evans, M; Frangoul, H; Ho, R; Kassim, A; Maheshwari, S; Manes, B; Yang, E; Yeh, RF, 2014
)
2.26
"Busulfan has been widely used to treat CML."( Busulfan inhibits growth of human osteosarcoma through miR-200 family microRNAs in vitro and in vivo.
Li, F; Liu, Y; Mei, Q; Quan, H; Xu, H, 2014
)
2.57
"Busulfan and etoposide have been used as myeloablative therapy for autologous hematopoietic stem cell transplantation (HSCT) in adults with acute myeloid leukemia (AML) for > 20 years. "( A phase I study of targeted, dose-escalated intravenous busulfan in combination with etoposide as myeloablative therapy for autologous stem cell transplantation in acute myeloid leukemia.
Ai, WZ; Andreadis, C; Benet, LZ; Damon, LE; Gaensler, KM; Kaplan, LD; Koplowicz, YB; Linker, CA; Logan, AC; Mannis, GN; Martin, TG; Olin, RL; Sayre, PH; Smith, CC; Sudhindra, A; Venstrom, JM; Wolf, JL, 2015
)
2.11
"busulfan (Bu) has been shown to be highly effective as a pretransplant conditioning regimen in acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and myelodysplastic syndrome (MDS)."( Long-Term Outcomes after Treatment with Clofarabine ± Fludarabine with Once-Daily Intravenous Busulfan as Pretransplant Conditioning Therapy for Advanced Myeloid Leukemia and Myelodysplastic Syndrome.
Alatrash, G; Alousi, AM; Andersson, BS; Champlin, RE; Chen, J; de Lima, M; Fox, PS; Garber, HR; Hosing, C; Janbey, S; Jones, RB; Kebriaei, P; Ning, J; Popat, U; Rondon, G; Shpall, EJ; Thall, PF; Valdez, BC; Worth, LL, 2016
)
1.37
"Busulfan has a very narrow therapeutic index, and acute toxicity may be related to absorption and disposition of the drug and metabolites."( Busulfan use in hematopoietic stem cell transplantation: pharmacology, dose adjustment, safety and efficacy in adults and children.
Bentur, Y; Hoffer, E; Krivoy, N; Lurie, Y; Rowe, JM, 2008
)
2.51
"High busulfan plasma levels have been shown to increase the chance of venoocclusive disease and low levels are associated with recurrence of disease or graft rejection."( Determination of busulfan in human plasma using an ELISA format.
Agarwal, G; Courtney, JB; Fleisher, M; Gonzalez-Espinoza, R; Harney, R; Juenke, JM; Li, Y; Lundell, G; Mathew, A; McMillin, GA; Salamone, SJ, 2009
)
1.15
"Busulfan has a narrow therapeutic range, and in children, pharmacokinetic variability has been found to be high even after the use of intravenous (i.v.) busulfan. "( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
2.06
"Busulfan has been investigated widely for more than three decades leading to a large and precise handling of this agent with numerous studies on activity and pharmacokinetics and pharmacodynamics. "( Pharmacology of dimethanesulfonate alkylating agents: busulfan and treosulfan.
Galaup, A; Paci, A, 2013
)
2.08
"Busulfan has been widely used, but irradiation has been often suggested as an alternative."( Irradiated mouse testes efficiently support spermatogenesis derived from donor germ cells of mice and rats.
Meistrich, ML; Shao, S; Zhang, Z,
)
0.85
"Busulfan has a narrow therapeutic index, giving rise to major liver toxicity (veno-occlusive disease), and a wide interpatient and intrapatient pharmacokinetic variability."( Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study.
Aulagner, G; Bertholle-Bonnet, V; Bertrand, Y; Bleyzac, N; Galambrun, C; Mialou, V; Souillet, G, 2007
)
1.34
"Busulfan has been used widely at conventional dosages (1-12 mg/d) for the treatment of patients with chronic myelogenous leukemia (CML). "( Busulfan.
Buggia, I; Locatelli, F; Regazzi, MB; Zecca, M, 1994
)
3.17
"Busulfan has been previously only available in an oral formulation due to its poor water solubility. "( A phase I/II study of multiple-dose intravenous busulfan as myeloablation prior to stem cell transplantation.
Apperley, J; Craddock, C; Eades, A; Goldman, J; Hassan, M; Kanfer, E; Matthews, J; Nilsson, C; Olavarria, E; Timms, A, 2000
)
2.01
"Busulfan has been the drug most widely used during pregnancy, and it appears not to cause fetal malformations when used alone."( Chronic myelocytic leukemia and the myeloproliferative diseases during the child-bearing years.
Miller, JB, 1976
)
0.98
"Busulfan has been postulated block the formation of new erythropoietin responsive cells (ERC) from hematopoietic stem cells (CFU)."( Increase in erythroid colony formation in rabbits following the administration of testosterone.
Fisher, JW; Moriyama, Y, 1975
)
0.98
"Busulfan has been shown to be mutagenic to microorganisms, mammalian cells in culture, Drosophila, and rodents."( Toxicological review of busulfan (Myleran).
Bishop, JB; Wassom, JS, 1986
)
1.3

Actions

Busulfan did cause a small number of DNA strand breaks to be formed in human cells.

ExcerptReferenceRelevance
"busulfan displays a smaller interpatient variability in exposure compared to oral busulfan (CV of 24% after i.v."( Toxicity and pharmacokinetics of i.v. busulfan in children before stem cell transplantation.
Boos, J; Fleischack, G; Gruhn, B; Hempel, G; Klingebiel, T; Mürdter, T; Oechtering, D; Schiltmeyer, B; Schwab, M; Vormoor, J; Würthwein, G, 2005
)
1.32
"Busulfan did however cause a small number of DNA strand breaks to be formed in human cells."( In vitro studies on the mechanism of action of hepsulfam in chronic myelogenous leukemia patients.
Adlakha, A; Cook, CA; Furmanski, P; Gibson, NW; Hincks, JR; Johnson, CS, 1990
)
1

Treatment

Treatment with Busulfan plus Fludarabine depleted lymphocytes only weakly. LGF treatment in busulfan-treated animals that have suffered a disruption of spermatogenesis can accelerate the reactivation of this process.

ExcerptReferenceRelevance
"busulfan treatment, no adverse physiological effects or body weight loss were detected."( Establishment of effective and safe recipient preparation for germ-cell transplantation with intra-testicular busulfan treatment in pre-pubertal Barbari goats.
Chauhan, MS; Kharche, SD; Pathak, M; Pawaiya, RVS; Quadri, SA; Singh, MK; Singh, SP; Soni, YK, 2022
)
1.65
"Busulfan-treated mice presented with dose-dependent clinical signs including signs of anemia in some individuals."( Model Characterization: Total Body Irradiation or Busulfan for Conditioning in Human Cell Therapy Toxicology and Tumorigenicity Studies using NOD/SCID/IL2Rγnull (NSG) Mice.
Abrahim, MA; Authier, S; Ballesteros, C; Li, C; Wong, K,
)
1.11
"Busulfan treated group received a single dose of busulfan (40 mg/kg), then animals were subdivided to three subgroups; IIa, IIb, IIc which were sacrificed after four, ten and twenty weeks, respectively, from the beginning of the experiment."( The impact of busulfan on the testicular structure in prepubertal rats: A histological, ultrastructural and immunohistochemical study.
Abd El-Hay, RI; Gawish, SA; Hamed, WHE; Mostafa Omar, N; Refat El-Bassouny, D, 2023
)
1.99
"busulfan treatment)."( Targeting myeloid cells to the brain using non-myeloablative conditioning.
Böttcher, C; Fernández-Klett, F; Gladow, N; Priller, J; Rolfes, S, 2013
)
1.11
"Busulfan treatment in juvenile rats resulted in abnormal root development, depending on the stage at which Bu was administered. "( Effect of the antineoplastic agent busulfan on rat molar root development.
Kawano, Y; Kinoshita-Kawano, S; Mitomi, T, 2014
)
2.12
"Busulfan treatment significantly reduced the number of circulating platelets in guinea-pigs by 85.5%, but had no significant effect on the number of circulating leukocytes. "( Bradykinin and capsaicin induced airways obstruction in the guinea pig are platelet dependent.
Keir, SD; Page, CP; Spina, D, 2015
)
1.86
"Busulfan treatment did not affect edema and hemorrhage in P-selectin- or PSGL-1-deficient mice, suggesting that the effect by busulfan is dependent on P-selectin and PSGL-1 expression."( Platelets control leukocyte recruitment in a murine model of cutaneous arthus reaction.
Fujimoto, M; Hara, T; Hasegawa, M; Iwata, Y; Komura, K; Muroi, E; Ogawa, F; Sato, S; Shimizu, K; Takenaka, M; Yanaba, K, 2010
)
1.08
"Busulfan treatment as a chemotherapeutic agent has been considered an alternative approach in xenograft model because it offers a simple, convenient, effective, and less toxic conditioning regimen."( Human B cell development and antibody production in humanized NOD/SCID/IL-2Rγ(null) (NSG) mice conditioned by busulfan.
Choi, B; Chun, E; Kim, M; Kim, SJ; Kim, ST; Lee, KY; Yoon, K, 2011
)
2.02
"In busulfan-treated ZW larvae, the presence of the typical central cavity and expression of a high level of aromatase mRNA confirmed the ovarian phenotype."( Busulfan-mediated germ cell depletion does not alter gonad differentiation in the urodele amphibian Pleurodeles waltl.
Al-Asaad, I; Chardard, D; Chesnel, A; Dumond, H; Flament, S, 2012
)
2.34
"In busulfan-treated thrombocytopenic animals, there was no evidence for either neointimal development or thrombus formation."( Role of platelet activation in catheter-induced vascular wall injury.
Bertipaglia, B; Chiavegato, A; Filipetto, C; Gamba, P; Gasparotto, L; Sartore, S; Taiani, J; Zolpi, E, 2004
)
0.84
"Both busulfan treatments resulted in a significant reduction in PGCs when compared to controls."( Production of germline chimeric chickens following the administration of a busulfan emulsion.
D'Costa, S; Pardue, SL; Petitte, JN; Song, Y, 2005
)
1.01
"Both busulfan treatment doses caused earlier occurrence of persistent estrus, with dose-dependence as compared to controls."( Reduction of primordial follicles caused by maternal treatment with busulfan promotes endometrial adenocarcinoma development in donryu rats.
Maekawa, A; Shirota, M; Taya, K; Watanabe, G; Yoshida, M, 2005
)
1.02
"Busulfan treatment caused a statistically significant decrease in the number of seminiferous tubules containing germ cells."( Testicular xenografts: a novel approach to study cytotoxic damage in juvenile primate testis.
Ehmcke, J; Jahnukainen, K; Schlatt, S, 2006
)
1.06
"Busulfan treatment can be optimized by targeted steady-state concentration or with the use of intravenous preparations."( Risk assessment in haematopoietic stem cell transplantation: conditioning.
Aschan, J, 2007
)
1.06
"Busulfan treatment caused acute declines in testis volume and sperm counts, indicating a disruption of spermatogenesis."( Characterization, cryopreservation, and ablation of spermatogonial stem cells in adult rhesus macaques.
Hermann, BP; Hobbs, RM; Lin, CC; McFarland, D; Orwig, KE; Pandolfi, PP; Rodriguez, M; Schatten, GP; Sheng, Y; Shuttleworth, JJ; Sukhwani, M; Tomko, J, 2007
)
1.06
"In busulfan-treated embryos, diffuse cell death was evident in both ectoderm and mesoderm, peaking at E13."( Busulfan-induced central polydactyly, syndactyly and cleft hand or foot: a common mechanism of disruption leads to divergent phenotypes.
Naruse, T; Oberg, KC; Ogino, T; Takagi, M; Takahara, M, 2007
)
2.3
"Busulfan treatment produced no obvious change in Itm2b expression in epididymis or vas deferens."( Differential expression and regulation of integral membrane protein 2b in rat male reproductive tissues.
Deng, WB; Gao, F; Liang, XH; Mills, N; Rengaraj, D; Yang, ZM, 2008
)
1.07
"In busulfan-treated rats, compared to controls, hypothalamic LH-RH content was decreased by 52%, whereas pituitary LH and FSH concentrations were increased by 60 and 43% respectively."( Effect of prenatal treatment with busulfan on the hypothalamo-pituitary axis, genital tract and testicular histology of prepubertal male rats.
Barenton, B; Hochereau-de Reviers, MT; Perreau, C; Viguier-Martinez, MC, 1984
)
1.06
"Busulfan treatment induced decrease of total volumes of intertubular tissue and of Leydig cells per testis, of accessory glands, and of germ cells from type A spermatogonia to zygotene spermatocytes."( Effects of supplementation with impuberal or adult testicular protein extracts on genital tract and testicular histology as well as hormonal levels in adult busulfan treated rats.
Hochereau-de-Reviers, MT; Perreau, C; Viguier-Martinez, MC,
)
1.05
"Busulfan treated patients who are now deceased, have had a median survival of 35 months (range, 13-108) and actuarial analysis shows the total busulfan treated population to have an expected median survival of 48 months."( Busulfan versus hydroxyurea in long-term therapy of chronic myelogenous leukemia.
Bolin, RW; Hamman, RF; Robinson, WA; Sutherland, J, 1982
)
2.43
"When busulfan-treated polycythemic mice received 5 mg of dexamethasone together with Ep injections they responded to the test dose of Ep with very minor increases in iron incorporation, which would indicate that a very small increase in the ERC population induced by Ep took place."( Inhibition by dexamethasone of erythropoietin-induced amplification of the erythropoietin-responsive cell compartment.
Alippi, RM; Bozzini, CE; Giglio, MJ, 1981
)
0.72
"The busulfan-treated patients had an increased cumulative incidence of veno-occlusive disease of the liver, ie, 12% compared with 1% in the TBI group (P = .009)."( A randomized trial comparing busulfan with total body irradiation as conditioning in allogeneic marrow transplant recipients with leukemia: a report from the Nordic Bone Marrow Transplantation Group.
Lenhoff, S; Ljungman, P; Nikoskelainen, J; Parkkali, T; Remberger, M; Ringdén, O; Ruutu, T; Sallerfors, B; Vindeløv, L; Volin, L, 1994
)
1.06
"Busulfan-treated patients had an increased risk of veno-occlusive disease (VOD) of the liver (12% v 1%, P =.01) and hemorrhagic cystitis (32% v 10%, P =.003)."( Increased risk of chronic graft-versus-host disease, obstructive bronchiolitis, and alopecia with busulfan versus total body irradiation: long-term results of a randomized trial in allogeneic marrow recipients with leukemia. Nordic Bone Marrow Transplanta
Jacobsen, N; Lenhoff, S; Ljungman, P; Mellander, L; Nikoskelainen, J; Parkkali, T; Remberger, M; Ringdén, O; Ruutu, T; Sallerfors, B; Vindeløv, L; Volin, L, 1999
)
1.24
"Busulfan treatment reduced testis receptor levels by 35% (P less than 0.05) after 35 days (maximum effect), and the effect was reversed after recovery (85 d)."( Multiple sites of action of the vitamin D endocrine system: FSH stimulation of testis 1,25-dihydroxyvitamin D3 receptors.
Anderson, MB; Christakos, S; Huang, HF; Osmundsen, BC; Walters, MR, 1989
)
1
"Busulfan treatment was given for one month and the infiltration had disappeared ten weeks after initiation of therapy."( [Diabetes insipidus in chronic myeloid leukemia. Remission of hypophyseal infiltration during busulfan treatment].
Eichhorn, P; Furrer, J; Haller, D; Hämmerli, R; Rhyner, K; Steuli, R, 1988
)
1.22
"Busulfan-treated tumor-bearing mice (BUTUM), busulfan-treated control mice (BUCON), tumor-bearing mice with no busulfan (TUM), and normal controls (CON) were sacrificed on days 4, 7, 11, 14, and 17 after tumor implantation."( Concurrent activation of granulocytes and osteoclasts in busulfan-suppressed bone marrow in response to transplantation of a mammary carcinoma in mice.
Lee, MY; Lottsfeldt, JL; McCracken, CH, 1988
)
1.24
"When busulfan treatment was resumed, hematologic response and decrease of hepatomegaly and splenomegaly reoccurred."( Remission of chronic idiopathic myelofibrosis to busulfan treatment.
Chang, JC; Gross, HM, 1988
)
0.98
"In busulfan-treated mice, polyploid-like mitoses with PCC were seen."( Occurrence of premature chromosome condensation in mouse spermatogonia treated with busulfan.
Hitotsumachi, S; Kikuchi, Y; Yamamoto, KI, 1986
)
1.01
"Treatment with busulfan had no significant effect on bronchoconstriction induced by the direct acting spasmogens histamine or methacholine."( Bradykinin and capsaicin induced airways obstruction in the guinea pig are platelet dependent.
Keir, SD; Page, CP; Spina, D, 2015
)
0.76
"Treatment with Busulfan plus Fludarabine depleted lymphocytes only weakly."( Impact of 3 different short-term chemotherapy regimens on lymphocyte-depletion and reconstitution in melanoma patients.
Appay, V; Canellini, G; Laurent, J; Leyvraz, S; Papaioannou, A; Rimoldi, D; Romero, P; Rufer, N; Speiser, DE; Touvrey, C; Vicari, M; Voelter, V, 2010
)
0.7
"LGF treatment in busulfan-treated animals that have suffered a disruption of spermatogenesis can accelerate the reactivation of this process in most of the tubules, as shown in the histological analysis."( Effect of liver growth factor on both testicular regeneration and recovery of spermatogenesis in busulfan-treated mice.
Arenas, MI; Díaz-Gil, JJ; Gutierrez-Adan, A; Lobo, MV; Pérez-Cerezales, S; Pérez-Crespo, M; Pericuesta, E, 2011
)
0.92
"Treatment with busulfan or interferon-α is usually effective in hydroxyurea failures."( Polycythemia vera and essential thrombocythemia: 2012 update on diagnosis, risk stratification, and management.
Tefferi, A, 2012
)
0.72
"Treatment with busulfan was stopped in March 1993 due to bone marrow suppression."( Long-term cytogenetic remission with ubenimex monotherapy in a case of chronic myeloid leukemia.
Fujimaki, K; Ishigatsubo, Y; Kanamori, H; Koharazawa, H; Taguchi, J; Takabayashi, M; Takasaki, H; Yamaji, S, 2004
)
0.66
"Treatment with busulfan results in the inhibition of cell cycle progression and apoptosis of tumor cells."( Reduced expression of Rho guanine nucleotide dissociation inhibitor-alpha modulates the cytotoxic effect of busulfan in HEK293 cells.
Beck, JF; Bien, S; Kroemer, HK; Reimer, J; Ritter, CA; Sonnemann, J; Wieland, T, 2007
)
0.89
"Rats treated with busulfan to induce thrombocytopenia exhibited a 90% decrease in circulating platelets."( Involvement of nitric oxide and cyclooxygenase products in photoactivation-induced microvascular occlusion.
Lentsch, AB; Lindberg, RA; Miller, FN; Slaaf, DW, 1994
)
0.61
"Mice treated with busulfan develop chronic latent marrow aplasia characterized by near-normal peripheral leukocyte counts, hematocrits, and marrow cellularity but very reduced stem cell (CFU-S) and progenitor cell (CFU-GM) populations. "( Long-term marrow cultures from mice with busulfan-induced chronic latent aplasia.
Caro, J; Erslev, AJ; Halka, KG, 1987
)
0.87
"Mice treated with busulfan also demonstrate residual marrow damage as evidenced by incomplete recovery of marrow CFU-S to pretreatment levels."( Late effects of chemotherapy on hematopoietic progenitor cells.
Adler, S; Fried, W, 1985
)
0.59

Toxicity

CloFluBu provides a less toxic alternative to conventional conditioning regimens, with adequate antileukemic activity. SSC of Balb/C mice are more sensitive to the toxic effects of busulfan then those of Swiss mice.

ExcerptReferenceRelevance
"Drug-induced pulmonary disease is an infrequent, but clinically significant, toxic manifestation of antineoplastic drug therapy."( Pulmonary toxicity of antineoplastic drugs.
Willson, JK, 1978
)
0.26
" We have recently shown that hepsulfam was more toxic to L1210 leukemia cells than was busulfan."( Mechanisms of toxicity of hepsulfam in human tumor cell lines.
Cook, C; Gibson, NW; Hincks, JR; Pacheco, DY, 1990
)
0.5
" To determine the drug-related nonhematologic toxic effects of this new regimen without confounding factors associated with allogeneic transplantation, we conducted a pilot study using this new regimen in 20 patients with acute myeloid leukemia (AML) in first remission prior to autologous unpurged marrow transplantation."( Acute toxicity and first clinical results of intensive postinduction therapy using a modified busulfan and cyclophosphamide regimen with autologous bone marrow rescue in first remission of acute myeloid leukemia.
Beelen, DW; Graeven, U; Mahmoud, HK; Quabeck, K; Sayer, HG; Schaefer, UW, 1989
)
0.5
" It is worth noting that both the analogs were definitely less toxic to the host mice than busulfan."( Antitumor activity and neutrophil-selective hematopoietic toxicity of busulfan analogs in mice.
Kato, T; Kawazoe, Y; Kimoto, H; Kohda, K; Ohta, Y; Suzumura, Y, 1988
)
0.73
" Thus, we have developed a safe chemotherapy schedule for transplantation that avoids the need for radiotherapy and can, if necessary, be repeated after a 3-month interval."( Busulphan and cyclophosphamide cause little early toxicity during displacement bone marrow transplantation in fifty children.
Barnes, R; Downie, CJ; Hobbs, JR; Hugh-Jones, K; Shaw, PJ, 1986
)
0.27
" Azoospermia at 56 days after treatment, which is a result of stem cell killing, was achieved at doses of over 30 mg/kg; this dose is below the LD50 for animal survival, which was over 40 mg/kg."( Effects of busulfan on murine spermatogenesis: cytotoxicity, sterility, sperm abnormalities, and dominant lethal mutations.
Bucci, LR; Meistrich, ML, 1987
)
0.66
"To evaluate the toxic extramedullary morbidity of the conditioning treatment with BuCy for BMT, as well as the usefulness of pentoxyphyllin (PTX) and methylprednisolone (MP) in the prophylaxis of mucositis."( [Extramedullary toxicity in bone marrow transplantation using busulfan and cyclophosphamide conditioning].
Maldonado, J; Pascual, MJ, 1995
)
0.53
" There were seven acute toxic deaths (8%) and in total 15 patients experienced life-threatening or fatal toxicity."( Extramedullary toxicity of a conditioning regimen containing busulphan, cyclophosphamide and etoposide in 84 patients undergoing autologous and allogenic bone marrow transplantation.
Bernstein, E; Brodsky, I; Bulova, S; Crilley, P; Marks, DI; Mullaney, R; Resnick, K; Styler, MJ; Topolsky, D, 1995
)
0.29
" The major toxic effects were leukopenia and thrombocytopenia, which, however, were manageable and rapidly reversible."( Maintenance chemotherapy with oral treosulfan following first-line treatment in patients with advanced ovarian cancer: feasibility and toxicity.
Kuhn, W; Meden, H; Wittkop, Y,
)
0.13
" We have examined the possibility that BU may exert part of its toxic effects via DNA alkylation at the O6 position of guanine as this might provide an approach to improving the conditioning regimen."( O6-Benzylguanine potentiates BCNU but not busulfan toxicity in hematopoietic stem cells.
Blokland, I; Boudewijn, A; Down, JD; Margison, GP; McGown, AT; Ploemacher, RE; Watson, AJ; Westerhof, GR; Wood, M, 2001
)
0.57
" A relationship between exposure to busulphan, expressed as an area under the plasma concentration time curve (AUC), and effect and/or adverse effects, such as veno-occlusive disease (VOD), was reported."( The effect of modulation of glutathione cellular content on busulphan-induced cytotoxicity on hematopoietic cells in vitro and in vivo.
Alsadi, S; Edgren, M; Hägglund, H; Hassan, M; Hassan, Z; Hellström-Lindberg, E, 2002
)
0.31
" In summary, the combination of treosulfan and fludarabine is a safe and efficient conditioning regimen."( Treosulfan and fludarabine: a new toxicity-reduced conditioning regimen for allogeneic hematopoietic stem cell transplantation.
Casper, J; Dölken, G; Freund, M; Hammer, U; Hartung, G; Kiefer, T; Kleine, HD; Knauf, W; Knopp, A; Steiner, B; Wegener, R; Wilhelm, S; Wolff, D, 2004
)
0.32
" BU, given as four single daily doses of 150 mg/m2, is appropriate and safe in all age groups of children."( Busulphan given as four single daily doses of 150 mg/m2 is safe and effective in children of all ages.
Berry, A; Earl, JW; Nath, C; Shaw, PJ, 2004
)
0.32
" No case of severe hepatic veno-occlusive disease or other serious toxic events occurred."( Toxicity and pharmacokinetics of i.v. busulfan in children before stem cell transplantation.
Boos, J; Fleischack, G; Gruhn, B; Hempel, G; Klingebiel, T; Mürdter, T; Oechtering, D; Schiltmeyer, B; Schwab, M; Vormoor, J; Würthwein, G, 2005
)
0.6
" qd is a safe and effective regimen for allogeneic transplantation and is at least clinically equivalent to every 6 h dosing schemes using either oral or parenteral Bu."( Modification of the Bu/Cy myeloablative regimen using daily parenteral busulfan: reduced toxicity without the need for pharmacokinetic monitoring.
Agura, ED; Berryman, RB; Fay, JW; Mamlouk, K; Pineiro, LA; Sandler, I; Saracino, G; Vance, EA; White, M, 2005
)
0.56
" Reduced-intensity allogeneic SCT may offer a less toxic approach to patients with AML."( Preliminary results of the safety of immunotherapy with gemtuzumab ozogamicin following reduced intensity allogeneic stem cell transplant in children with CD33+ acute myeloid leukemia.
Bhatia, M; Bradley, B; Cairo, MS; Cooney, E; Del Toro, G; Foley, S; Garvin, J; George, D; Harrison, L; Hawks, R; Militano, O; Roman, E; Satwani, P; Unal, E; van de Ven, C; Wolownik, K, 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
"Neonatal administration of diethylstilbestrol (DES) to rodents has adverse effects on spermatogenesis."( Neonatal administration of diethylstilbestrol has adverse effects on somatic cells rather than germ cells.
Adachi, T; Fukata, H; Koh, KB; Komiyama, M; Mori, C; Toyama, Y, 2006
)
0.33
" The responses and adverse events of the 2 groups were assessed."( [Efficacy and toxicity of intravenous busulfan-based conditioning before allogeneic peripheral blood stem cell transplantation for leukemia].
Li, J; Luo, SK; Peng, AH; Tong, XZ; Xu, DR; Zhang, GC; Zheng, D; Zou, WY, 2007
)
0.61
" It is also important to consider the busulfan drug-drug interactions and adverse drug reactions that can develop during the therapeutic process."( Busulfan use in hematopoietic stem cell transplantation: pharmacology, dose adjustment, safety and efficacy in adults and children.
Bentur, Y; Hoffer, E; Krivoy, N; Lurie, Y; Rowe, JM, 2008
)
2.06
"5 mg/kg/day; n = 10 in each group) for 2 or 4 weeks to assess the optimal administration period for detection of the toxic effects on ovarian morphology."( Collaborative work on evaluation of ovarian toxicity. 5) Two- or four-week repeated-dose studies and fertility study of busulfan in female rats.
Iwasaki, S; Kudo, S; Masumoto, Y; Miyata, Y; Nishi, M; Sakurada, Y, 2009
)
0.56
" We concluded that prefilled-syringe method is ease and safe way to administer Busulfex on scheduled time."( [Development of new mixing method of Busulfex injection for the purpose of improvement of medical safety method: the prefilled syringe method].
Dan, K; Dobashi, A; Dobashi, Y; Hamada, M; Katayama, S; Senoo, M; Shimizu, H; Tajika, K, 2009
)
0.35
"IVBu is a safe and effective and offers the benefit of predictable and consistent systemic exposure."( Safety, efficacy, and pharmacokinetics of intravenous busulfan in children undergoing allogeneic hematopoietic stem cell transplantation.
Chan, KW; Hayashi, RJ; Kadota, R; Kletzel, M; Mezzi, K; Nieder, ML; Przepiorka, D; Wall, DA; Yeager, AM, 2010
)
0.61
" Our experience shows that targeting busulfan AUC above the range used in previous multicenter trials appears safe and may contribute to sustained engraftment in SCD."( Safety and efficacy of targeted busulfan therapy in children undergoing myeloablative matched sibling donor BMT for sickle cell disease.
Chiang, KY; Haight, AE; Horan, J; Hutcherson, D; McPherson, ME; Olson, E, 2011
)
0.93
" An understanding of structure-activity relationships (SARs) of chemicals can make a significant contribution to the identification of potential toxic effects early in the drug development process and aid in avoiding such problems."( Developing structure-activity relationships for the prediction of hepatotoxicity.
Fisk, L; Greene, N; Naven, RT; Note, RR; Patel, ML; Pelletier, DJ, 2010
)
0.36
" In search of less toxic alternatives, we hypothesized that combination of busulfan (Bu), fludarabine (Flu) and clofarabine (Clo) would provide superior efficacy."( The synergistic cytotoxicity of clofarabine, fludarabine and busulfan in AML cells involves ATM pathway activation and chromatin remodeling.
Andersson, BS; Champlin, RE; Li, Y; Murray, D; Valdez, BC, 2011
)
0.84
" Patients were evaluated for engraftment, adverse events, graft-versus-host disease, non-relapse mortality, relapse incidence, overall survival and disease-free survival."( Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial.
Baumgart, J; Beelen, DW; Blau, IW; Casper, J; Einsele, H; Finke, J; Freund, M; Giebel, S; Gramatzki, M; Holowiecki, J; Kienast, J; Larsson, K; Markiewicz, M; Mylius, HA; Pichlmeier, U; Repp, R; Ruutu, T; Schnitzler, M; Stelljes, M; Stuhler, G; Trenschel, R; Uharek, L; Volin, L; Zander, AR, 2011
)
0.37
" Non-hematologic adverse events of grade III-IV in severity included mainly infections and gastrointestinal symptoms (80% and 22% of the patients, respectively)."( Reduced-toxicity conditioning with treosulfan and fludarabine in allogeneic hematopoietic stem cell transplantation for myelodysplastic syndromes: final results of an international prospective phase II trial.
Baumgart, J; Beelen, DW; Blau, IW; Casper, J; Einsele, H; Finke, J; Freund, M; Giebel, S; Gramatzki, M; Holowiecki, J; Kienast, J; Larsson, K; Markiewicz, M; Mylius, HA; Pichlmeier, U; Repp, R; Ruutu, T; Schnitzler, M; Stelljes, M; Stuhler, G; Trenschel, R; Uharek, L; Volin, L; Zander, AR, 2011
)
0.37
" Multivariate analysis (MVA) identified comorbidity score (HCT-CI) >2 and advanced disease as adverse factors with no independent impact of regimen."( Allo-SCT for AML and MDS with treosulfan compared with BU-based regimens: reduced toxicity vs reduced intensity.
Danylesko, I; Nagler, A; Shem-Tov, N; Shimoni, A; Volchek, Y; Yerushalmi, R, 2012
)
0.38
" This treosulfan-based preparation proved to be safe and effective for thalassemia patients given allogeneic HSCT."( Allogeneic hematopoietic stem cell transplantation in thalassemia major: results of a reduced-toxicity conditioning regimen based on the use of treosulfan.
Bernardo, ME; Bertaina, A; Caocci, G; Contoli, B; Giorgiani, G; La Nasa, G; Locatelli, F; Mastronuzzi, A; Pagliara, D; Pinto, RM; Piras, E; Vacca, A; Zecca, M, 2012
)
0.38
"Intravenous BU divided four times daily (q6 h) has been shown to be safe and effective in pediatric allo-SCT recipients."( The pharmacokinetics and safety of twice daily i.v. BU during conditioning in pediatric allo-SCT recipients.
Baxter-Lowe, LA; Bhatia, M; Bradley, MB; Cairo, MS; Duffy, D; Garvin, JH; George, D; Geyer, MB; Harrison, L; Le Gall, JB; Militano, O; Milone, MC; Morris, E; Satwani, P; Schwartz, J; Shaw, LM; van de Ven, C; Waxman, IM, 2013
)
0.39
" 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
"Patients with class 3 thalassemia with high-risk features for adverse events after high-dose chemotherapy with hematopoietic stem cell transplantation (HSCT) are difficult to treat, tending to either suffer serious toxicity or fail to establish stable graft function."( Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control.
Andersson, BS; Anurathapan, U; Charoenkwan, P; Chuansumrit, A; Hongeng, S; Issaragrisil, S; Jetsrisuparb, A; Pakakasama, S; Rujkijyanont, P; Sirachainan, N; Sirireung, S; Songdej, D; Srisala, S; Sruamsiri, R; Ungkanont, A, 2013
)
0.39
" Our data indicate that Flu (160 mg/m(2)) with targeted myeloablative Bu (90 mg·h/L) is less toxic than and equally effective as BuCy (Mel) in patients with similar indications for allo-HCT."( Fludarabine and exposure-targeted busulfan compares favorably with busulfan/cyclophosphamide-based regimens in pediatric hematopoietic cell transplantation: maintaining efficacy with less toxicity.
Bartelink, IH; Bierings, MB; Boelens, JJ; de Wildt, A; Gerhardt, CE; Lindemans, CA; van Maarseveen, EM; van Reij, EM; Versluys, B, 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
)
0.4
"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
)
0.4
"Modified Bu/Flu as a new RIC regimen is well tolerated and safe for patients who need allogeneic hematopoietic stem cell transplantation, especially in older patients and/or patients with severe comorbidities."( [The efficacy and safety of modified busulfan/fludarabine conditioning regimen in elderly or drug-intolerable patients with hematologic malignancies].
Chen, H; Fu, HX; Huang, XJ; Liu, DH; Liu, KY; Sun, YQ; Tang, FF; Wang, FR; Wang, Y; Xu, LP, 2013
)
0.66
" The FB3-ATG2 regimen is safe and efficient in both lymphoid and myeloid disorders."( Feasibility of the fludarabine busulfan 3 days and ATG 2 days reduced toxicity conditioning in 51 allogeneic hematopoietic stem cell transplantation: a single-center experience.
Chantepie, SP; Gac, AC; Reman, O, 2014
)
0.69
" Because a safe Bu exposure was unknown in patients receiving this combination, Bu was initially targeted to a total area under the concentration-time curve (AUC) of 20,000 μM × minute."( Safety and efficacy of targeted-dose busulfan and bortezomib as a conditioning regimen for patients with relapsed multiple myeloma undergoing a second autologous blood progenitor cell transplantation.
Akpek, G; Armstrong, E; Elekes, A; Freytes, CO; Kato, K; Patil, S; Ratanatharathorn, V; Reece, DE; Rodriguez, TE; Sahovic, E; Shaughnessy, PJ; Smith, A; Solomon, SR; Stadtmauer, EA; Toro, JJ; Tricot, GJ; White, DJ; Yeh, RF; Yu, LH; Zhao, C, 2014
)
0.68
" These findings revealed that SSC of Balb/C mice are more sensitive to the toxic effects of busulfan then those of Swiss mice."( Gonadotoxic effects of busulfan in two strains of mice.
Bordignon, V; Chemeris, RO; Comim, FV; Glanzner, WG; Gonçalves, PB; Gutierrez, K; Rigo, ML, 2016
)
0.97
" Our study indicated that intratesticular injection busulfan for the preparation of recipients in mice is safe and feasible."( Testicular Busulfan Injection in Mice to Prepare Recipients for Spermatogonial Stem Cell Transplantation Is Safe and Non-Toxic.
Chen, X; Hao, H; He, Y; Liang, M; Liu, L; Qin, T; Qin, Y; Wang, C; Wang, D; Zhao, X, 2016
)
1.07
" Moreover, it appears to be safe with a low NRM rate among high-risk patients."( The efficacy and safety of a new reduced-toxicity conditioning with 4 days of once-daily 100 mg/m(2) intravenous busulfan associated with fludarabine and antithymocyte globulins prior to allogeneic stem cell transplantation in patients with high-risk myel
Blaise, D; Calmels, B; Castagna, L; Chabannon, C; Crocchiolo, R; Devillier, R; El-Cheikh, J; Faucher, C; Furst, S; Granata, A; Harbi, S; Lemarie, C; Vey, N; Wanquet, A; Weiller, PJ, 2016
)
0.65
"Many adverse drug reactions are caused by the cytochrome P450 (CYP)-dependent activation of drugs into reactive metabolites."( Development of a cell viability assay to assess drug metabolite structure-toxicity relationships.
Jones, LH; Nadanaciva, S; Rana, P; Will, Y, 2016
)
0.43
" In conclusion, the beneficial properties of SKEO pre-treatment and co-treatment by its herbal potent anti-oxidants may reduce adverse effects of chemotherapy in the reproductive system in a rodent system."( Busulfan-mediated oxidative stress and genotoxicity decrease in sperm of Satureja Khuzestanica essential oil-administered mice.
Nasimi, P; Roohi, S; Tabandeh, MR, 2018
)
1.92
" In conclusion, heat shock with busulfan treatment is a safe method to prepare the recipient of SSC transplantation in mice."( The Safe Recipient of SSC Transplantation Prepared by Heat Shock With Busulfan Treatment in Mice.
Gao, W; Jia, H; Ma, W; Wang, J, 2018
)
1
" While the toxicity of busulfan is well investigated, little is known about the toxic effects of its impurities."( Daily Intravenous Infusion of Busulfan Impurity 5 for 4 Days Is Not Associated With Toxic Effects in the Rat.
Bothe, MK; Cade, D; Franckenstein, D; Quatresous, E; Westphal, M,
)
0.73
" TBC combined with thiotepa is highly toxic to the skin with various cutaneous manifestations."( Skin toxicity following treosulfan-thiotepa-fludarabine-based conditioning regimen in non-malignant pediatric patients undergoing hematopoietic stem cell transplantation.
Altman Kohl, S; Even-Or, E; Molho-Pessach, V; Stepensky, P; Zaidman, I, 2020
)
0.56
" In the safety outcomes, incidents of veno-occlusive disease (VOD) were recorded, as well as other adverse events."( Busulfan systemic exposure and its relationship with efficacy and safety in hematopoietic stem cell transplantation in children: a meta-analysis.
Fan, D; Feng, X; Li, J; Wu, Y; Yang, C; Zhang, J; Zhao, L; Zhu, G, 2020
)
2
"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
)
0.56
" Trends toward improved survival were seen in patients transplanted for myeloid disease using bone marrow as stem cell source who achieved a busulfan AUC > 4000 μmol*min/day with two-year relapse-free survival approaching 80% CONCLUSIONS: This conditioning regimen is safe and effective in patients with high-risk leukemias, particularly myeloid disease."( Reduced-toxicity conditioning regimen with busulfan, fludarabine, rATG, and 400 cGy TBI in pediatric patients undergoing hematopoietic stem cell transplant for high-risk hematologic malignancies.
Chaudhury, S; Duerst, RE; Jacobsohn, D; Kletzel, M; Kwon, S; Rossoff, J; Schneiderman, J; Tse, WT, 2021
)
1.09
" In this study, we aimed to investigate the side effect of Bu/Cy and Bu/Flu regimens on our patients who underwent allogeneic bone marrow transplantation."( Adverse Effects of Busulfan Plus Cyclophosphamide versus Busulfan Plus Fludarabine as Conditioning Regimens for Allogeneic Bone Marrow Transplantation.
Hajifathali, A; Mabani, M; Mehdizadeh, M; Parkhideh, S; Rezvani, H; Salari, S, 2021
)
0.95
"The therapeutic-related adverse effects of the two conditioning regimens in patients who underwent allogeneic bone marrow transplant were almost similar."( Adverse Effects of Busulfan Plus Cyclophosphamide versus Busulfan Plus Fludarabine as Conditioning Regimens for Allogeneic Bone Marrow Transplantation.
Hajifathali, A; Mabani, M; Mehdizadeh, M; Parkhideh, S; Rezvani, H; Salari, S, 2021
)
0.95
" Grade 3 adverse events included mucositis, diarrhea, and liver transaminitis (n = 3 each)."( Adding venetoclax to fludarabine/busulfan RIC transplant for high-risk MDS and AML is feasible, safe, and active.
Antin, JH; Brock, J; Cutler, CS; DeAngelo, DJ; Fell, G; Garcia, JS; Gooptu, M; Ho, VT; Karp, HQ; Kim, AS; Kim, HT; Koreth, J; Letai, A; Lindsley, RC; Loschi, F; Lucas, F; Mashaka, T; Murdock, HM; Nikiforow, S; Potter, D; Romee, R; Ryan, J; Shapiro, R; Soiffer, RJ; Stone, RM, 2021
)
0.9
"We prospectively studied clofarabine-fludarabine-busulfan (CloFluBu)-conditioning in allogeneic hematopoietic cell therapy (HCT) for lymphoid and myeloid malignancies and hypothesized that CloFluBu provides a less toxic alternative to conventional conditioning regimens, with adequate antileukemic activity."( Clofarabine-fludarabine-busulfan in HCT for pediatric leukemia: an effective, low toxicity, TBI-free conditioning regimen.
Bierings, M; Boelens, JJ; Bresters, D; de Koning, CCH; Kollen, WJ; Lankester, AC; Lindemans, CA; Nierkens, S; Versluijs, AB, 2022
)
1.28
" Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is curative and safe at the pediatric age but remains underperformed in adults."( Curative allogeneic hematopoietic stem cell transplantation following reduced toxicity conditioning in adults with primary immunodeficiency.
Asnafi, V; Catherinot, É; Cheminant, M; Couderc, LJ; Fischer, A; Hermine, O; Jeljeli, M; Lanternier, F; Lortholary, O; Mahlaoui, N; Marçais, A; Moshous, D; Neven, B; Picard, C; Salvator, H; Suarez, F; van Endert, P, 2022
)
0.72
" However, considering a wide variation in the effects of busulfan among animal species, its dosage and route of infusion need optimization to prepare effective and safe recipients."( Establishment of effective and safe recipient preparation for germ-cell transplantation with intra-testicular busulfan treatment in pre-pubertal Barbari goats.
Chauhan, MS; Kharche, SD; Pathak, M; Pawaiya, RVS; Quadri, SA; Singh, MK; Singh, SP; Soni, YK, 2022
)
1.18
" The prediction of transplantation-related mortality (TRM) using the Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score and an arbitrary upper age limit of 55 years for administering myeloablative conditioning (MAC) are common strategies to ensure a safe procedure."( Myeloablative Dose of Busulfan and Fludarabine Combined with In Vivo T Cell Depletion Is Safe and Effective Conditioning for Acute Myeloid Leukemia and Myelodysplastic Syndrome Patients.
Alshehri, H; Anteh, S; Avenoso, D; Bourlon, C; Bouziana, S; Dazzi, F; de Farias, M; Dragoi, OD; Gameil, A; Hannah, G; Kenyon, M; Krishnamurthy, P; Kulasekararaj, A; Leung, YT; Mehra, V; Pagliuca, A; Potter, V; Serpenti, F; Shah, MN; Slonim, LB; Wood, H, 2023
)
1.22

Pharmacokinetics

Busulfan pharmacokinetic (PK) monitoring of the area under the concentration-time curve (AUC) is necessary to minimize adverse events associated with both under- and over-dosing of busulfan during hematopoietic stem cell transplantation. This IV form is expected to reduce the high pharmacokinetics variability exhibited with oral busulfa.

ExcerptReferenceRelevance
" The elimination half-life for the last dose in young children was shorter (2."( Pharmacokinetics of high-dose busulphan in relation to age and chronopharmacology.
Aschan, J; Bekassy, AN; Ehrsson, H; Hassan, M; Ljungman, P; Lönnerholm, G; Oberg, G; Smedmyr, B; Taube, A; Wallin, I, 1991
)
0.28
" Concentrations were fit by a one-compartment pharmacokinetic model with first-order absorption."( Pharmacokinetics of busulfan: correlation with veno-occlusive disease in patients undergoing bone marrow transplantation.
Braine, HG; Brundrett, RB; Chen, TL; Colvin, OM; Grochow, LB; Jones, RJ; Santos, GW; Saral, R, 1989
)
0.6
" Administration of busulfan as crushed rather than whole tablets reduced the delay time for appearance of busulfan in plasma but had no effect on absorption or other pharmacokinetic parameters."( Busulfan pharmacokinetics using a single daily high-dose regimen in children with acute leukemia.
Brian, RJ; Earl, JW; Scharping, CE; Shaw, PJ, 1994
)
2.06
" We investigated a 6-h and a 34-h infusion of VP-16 to compare pharmacokinetic parameters and toxicity."( Pharmacokinetics of high-dose VP-16: 6-hour infusion versus 34-hour infusion.
Bewermeier, P; Hossfeld, DK; Krüger, W; Mross, K; Reifke, J; Stockschläder, M; Zander, A, 1994
)
0.29
" VP-16 concentrations were measured in all plasma samples by high-performance liquid chromatography (HPLC) and electrochemical detection, and the results were analyzed with a pharmacokinetic data analysis system."( Pharmacokinetics of undiluted or diluted high-dose etoposide with or without busulfan administered to patients with hematologic malignancies.
Bewermeier, P; Hossfeld, DK; Krüger, W; Mross, K; Stockschläder, M; Zander, A, 1994
)
0.52
"All calculated pharmacokinetic parameters in the two patient groups (VP-16 administered diluted or undiluted) were similar."( Pharmacokinetics of undiluted or diluted high-dose etoposide with or without busulfan administered to patients with hematologic malignancies.
Bewermeier, P; Hossfeld, DK; Krüger, W; Mross, K; Stockschläder, M; Zander, A, 1994
)
0.52
"The two administration modes for VP-16, either diluted or undiluted, are bioequivalent in pharmacokinetic terms."( Pharmacokinetics of undiluted or diluted high-dose etoposide with or without busulfan administered to patients with hematologic malignancies.
Bewermeier, P; Hossfeld, DK; Krüger, W; Mross, K; Stockschläder, M; Zander, A, 1994
)
0.52
" In adults, pharmacodynamic studies have shown a positive correlation between high-systemic exposure of the drug and venocclusive disease (VOD)."( Aspects concerning busulfan pharmacokinetics and bioavailability.
Ehrsson, H; Hassan, M; Ljungman, P, 1996
)
0.62
"The development and validation of a high-performance liquid chromatographic (HPLC) assay for determination of busulfan concentrations in human plasma for pharmacokinetic studies is described."( Validation of a high-performance liquid chromatographic assay method for pharmacokinetic evaluation of busulfan.
Barnett, MJ; Burns, RB; Embree, L; Fung, HC; Heggie, JR; Knight, G; Ng, CS; Spinelli, JJ; Wu, M, 1997
)
0.72
" Pharmacokinetic parameters in individual patients have been related to short-term toxicity and risk of relapse after HSCT."( Pharmacokinetics of intravenous busulfan and evaluation of the bioavailability of the oral formulation in conditioning for haematopoietic stem cell transplantation.
Deeg, J; Ehninger, G; Ehrsam, M; Schmidt, H; Schneider, A; Schuler, US, 1998
)
0.58
" The mean AUC, Css, Cmax and MRV were significantly higher in group B as compared with group A for both doses 1 and 13."( Pharmacokinetics of oral busulphan in children with beta thalassaemia major undergoing allogeneic bone marrow transplantation.
Aigrain, EJ; Chandy, M; Dennison, D; Kanagasabapathy, AS; Krishnamoorthy, R; Poonkuzhali, B; Quernin, MH; Srivastava, A, 1999
)
0.3
"002) longer elimination half-life in group A (10."( The effect of busulphan on the pharmacokinetics of cyclophosphamide and its 4-hydroxy metabolite: time interval influence on therapeutic efficacy and therapy-related toxicity.
Abdel-Rehim, M; Astner, L; Békassy, A; Bielenstein, M; Georén, S; Hassan, M; Hassan, Z; Ljungman, P; Nilsson, C; Oberg, G; Ringdén, O, 2000
)
0.31
" Pharmacokinetic dosing of BU may be important for prevention of NRM but does not appear to influence the risk of relapse in this largely pediatric population with AML."( Busulfan pharmacokinetics do not predict relapse in acute myeloid leukemia.
Baker, KS; Blazar, BR; Bostrom, B; DeFor, T; Ramsay, NK; Woods, WG, 2000
)
1.75
" Bu was used as reference solution, the pharmacokinetic analysis indicated an average bioavailability of oral high-dose Bu of 69%, ranging from <10% to virtually 100%."( Acute safety and pharmacokinetics of intravenous busulfan when used with oral busulfan and cyclophosphamide as pretransplantation conditioning therapy: a phase I study.
Andersson, BS; Blume, KG; Champlin, RE; Chow, DS; Hu, WW; Madden, T; Tran, HT; Vaughan, WP, 2000
)
0.56
"A Phase I dose escalation and pharmacokinetic study of the alkylating cytotoxic agent treosulfan was conducted to evaluate the maximum tolerated dose and the dose-limiting toxicities in patients with advanced malignancies rescued by autologous peripheral blood stem cell transplantation."( Clinical phase I dose escalation and pharmacokinetic study of high-dose chemotherapy with treosulfan and autologous peripheral blood stem cell transplantation in patients with advanced malignancies.
Baumgart, J; Berdel, WE; Bojko, P; Bornhäuser, M; Casper, J; Ehninger, G; Freund, M; Harstrick, A; Hilger, RA; Josten, KM; Oberhoff, C; Scheulen, ME; Schindler, AE; Seeber, S; Wolf, HH, 2000
)
0.31
"The current practice for the dose calculation of most anticancer agents is based on body surface area in m2, although lower interpatient variation in pharmacokinetic parameters has been reported with pharmacokinetically guided administration."( Pharmacokinetically guided administration of chemotherapeutic agents.
Beijnen, JH; Mathôt, RA; Schellens, JH; van den Bongard, HJ, 2000
)
0.31
" Pharmacokinetic analysis of plasma busulfan levels alerted staff to the dosing error."( Accidental busulfan overdose: enhanced drug clearance with hemodialysis in a child with Wiskott-Aldrich syndrome.
Ben-Ari, J; Bentur, Y; Davidovitz, M; Gamzu, Z; Hoffer, E; Krivoy, N; Stein, J; Tabak, A; Yaniv, I, 2001
)
0.98
"The contribution of the bone marrow to in vivo erythropoietin (EPO) elimination was evaluated by determining EPO pharmacokinetic (PK) parameters in five adult sheep in a paired manner before and after chemotherapy-induced marrow ablation."( Changes in erythropoietin pharmacokinetics following busulfan-induced bone marrow ablation in sheep: evidence for bone marrow as a major erythropoietin elimination pathway.
Chapel, S; Hohl, RJ; McGuire, EM; Schmidt, RL; Veng-Pedersen, P; Widness, JA, 2001
)
0.56
" This method was used to determine the pharmacokinetic parameters of busulfan after the first administration of 1 mg/kg orally, in 13 children receiving the drug as part of the preparative regimen for bone marrow transplantation."( Quantification of busulfan in plasma by liquid chromatography-ion spray mass spectrometry. Application to pharmacokinetic studies in children.
Aigrain, EJ; Duval, M; Litalien, C; Quernin, MH; Vilmer, E, 2001
)
0.88
"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
)
0.84
" Although busulfan is used frequently in children as part of a myeloablative regimen prior to bone marrow transplantation, pharmacokinetic data on intravenous busulfan in children are scarce."( Pharmacokinetics of intravenous busulfan in children prior to stem cell transplantation.
Ball, L; Bredius, R; Cremers, S; den Hartigh, J; Schoemaker, R; Twiss, I; Vermeij, P; Vossen, J, 2002
)
1
" Pharmacokinetic studies were done using 11 samples with the first and fourth doses of Bu."( Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Andersson, BS; Brown, C; Chaudhry, A; Duggan, P; Glick, S; Gyonyor, E; Morris, D; Quinlan, D; Ruether, JD; Russell, JA; Stewart, D; Tran, HT, 2002
)
0.63
" Variability due to drug metabolism remains, but simplified pharmacokinetic study may be employed to achieve a specific target AUC."( A limited sampling strategy for pharmacokinetic directed therapy with intravenous busulfan.
Carey, D; Perry, S; Salzman, DE; Vaughan, WP; Westfall, AO, 2002
)
0.54
" Pharmacokinetic parameters were derived by modeling the raw data to fit first-order single compartment kinetics."( Plasma pharmacokinetics of high-dose oral busulfan in children and adults undergoing bone marrow transplantation.
Blazar, B; Bostrom, B; Bruns, A; Enockson, K; Johnson, A, 2003
)
0.58
" Epo's elimination kinetics was studied using a very sensitive tracer interaction method (TIM) before and after chemical ablation of BM as an indirect way of evaluating the EpoR through an assortment of pharmacokinetic parameters (VM, KM, K, and CL) used in differentiating the EpoR population in newborn and adult sheep."( A differential pharmacokinetic analysis of the erythropoietin receptor population in newborn and adult sheep.
Al-Huniti, NH; Chapel, S; Hohl, RJ; Schmidt, RL; Sedars, EM; Veng-Pedersen, P; Widness, JA, 2003
)
0.32
" Plasma concentration-time data were analysed by population pharmacokinetic modelling using NONMEM."( Population pharmacokinetics of oral busulfan in children.
Boos, J; Ehninger, G; Gruhn, B; Hempel, G; Jenke, A; Klingebiel, T; Mürdter, TE; Ritter, CA; Schiltmeyer, B; Schwab, M; Würthwein, G, 2003
)
0.59
" The influence of different covariates on the pharmacokinetic parameters was tested."( Population pharmacokinetics of oral busulfan in children.
Boos, J; Ehninger, G; Gruhn, B; Hempel, G; Jenke, A; Klingebiel, T; Mürdter, TE; Ritter, CA; Schiltmeyer, B; Schwab, M; Würthwein, G, 2003
)
0.59
" Median busulfan clearance was 109 mL/min/m2 and median daily area-under-the-plasma-concentration-versus-time-curve was 4871 micromol-min, with negligible interdose variability in pharmacokinetic parameters."( Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS.
Andersson, BS; Champlin, RE; Couriel, D; de Lima, M; Giralt, S; Jones, R; Korbling, M; Madden, T; Pierre, B; Russell, JA; Shahjahan, M; Shpall, EJ; Thall, PF; Wang, X, 2004
)
1.07
" This pharmacokinetic study was conducted to evaluate the ability of the apparent oral clearance obtained after administering a lower (0."( Intraindividual variability in busulfan pharmacokinetics in patients undergoing a bone marrow transplant: assessment of a test dose and first dose strategy.
Decker, J; Garbriel, D; Harvey, D; Kirby, S; Lindley, C; McCune, J; Petros, WP; Serody, J; Shea, T; Shord, S; Wiley, J, 2004
)
0.61
" Pharmacokinetic studies were performed after the first dose."( Intravenous busulfan in children prior to stem cell transplantation: study of pharmacokinetics in association with early clinical outcome and toxicity.
Ball, LM; Bredius, RG; Cremers, SC; den Hartigh, J; Egeler, RM; Lankester, AC; Teepe-Twiss, IM; Vossen, JM; Zwaveling, J, 2005
)
0.71
" This IV form is expected to reduce the high pharmacokinetic variability exhibited with oral busulfan and as a result, to better target the plasma area under the curve (AUC)."( Intravenous busulfan in adults prior to haematopoietic stem cell transplantation: a population pharmacokinetic study.
Leger, F; Lennon, S; Nguyen, L; Puozzo, C, 2006
)
0.93
"A population pharmacokinetic analysis was performed in 30 patients who received an intravenous busulfan and cyclophosphamide regimen before hematopoietic stem cell transplantation."( Population pharmacokinetics of intravenous busulfan in patients undergoing hematopoietic stem cell transplantation.
Nakashima, D; Takama, H; Takaue, Y; Tanaka, H; Ueda, R, 2006
)
0.82
"Therapeutic drug monitoring is used to minimize toxicity and maximize the therapeutic efficacy of busulfan, which shows high intra- and interpatient pharmacokinetic variability and erratic oral absorption."( Development of a pharmacokinetic and Bayesian optimal sampling model for individualization of oral busulfan in hematopoietic stem cell transplantation.
Booker, BM; Bullock, JM; Capozzi, D; Loughner, J; McCarthy, PL; Shaw, LM; Smith, PF, 2006
)
0.77
" Pharmacokinetic parameters were calculated from plasma busulfan concentration."( Influence of glutathione S-transferase A1 polymorphism on the pharmacokinetics of busulfan.
Iga, T; Kanda, Y; Kubota, T; Kusama, M; Matsukura, Y; Matsuno, K; Ogawa, S, 2006
)
0.81
"Busulfan pharmacokinetic (PK) data showed the area under the curve (AUC), drug half-life, and drug clearance were consistent within each dose group and similar to those reported in children."( Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model.
Donahue, RE; Hsieh, MM; Kang, EM; Krouse, A; Metzger, M; Sadelain, M; Tisdale, JF, 2006
)
3.22
"We performed a Phase I and pharmacokinetic study of once-daily, intravenously administered busulfan in the setting of a reduced-intensity preparative regimen and matched sibling donor allogeneic stem cell transplantation for treatment of metastatic renal cell carcinoma."( Phase I and pharmacokinetic study of once-daily dosing of intravenously administered busulfan in the setting of a reduced-intensity preparative regimen and allogeneic hematopoietic stem cell transplantation as immunotherapy for renal cell carcinoma.
Alexander, W; Bachier, C; LeMaistre, C; Pollack, M; Ririe, D; Shaughnessy, P; Splichal, J; Tran, H, 2006
)
0.78
" Pharmacokinetic studies were performed after the first dose."( Once-daily intravenous busulfan in children prior to stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Bredius, RG; den Hartigh, J; Egeler, RM; Guchelaar, HJ; Lankester, AC; Maarten Bredius, RG; Zwaveling, J, 2006
)
0.64
" Intra- and inter-day precision and accuracy of the measurement fulfilled analytical criteria accepted in pharmacokinetic studies."( Determination of treosulfan in plasma and urine by HPLC with refractometric detection; pharmacokinetic studies in children undergoing myeloablative treatment prior to haematopoietic stem cell transplantation.
Grund, G; Główka, FK; Wachowiak, J; Łada, MK, 2007
)
0.34
" The pharmacokinetic (PK) properties of F-ara-A (9-beta-D-arabinosyl-2-fluoradenine) before and after application of busulfan were prospectively investigated in 16 patients with hematological malignancies."( F-ara-A pharmacokinetics during reduced-intensity conditioning therapy with fludarabine and busulfan.
Bergeman, T; Bonin, M; Bornhauser, M; Ehninger, G; Illmer, T; Leopold, T; Pursche, S; Schleyer, E, 2007
)
0.77
" Busulfan has a narrow therapeutic index, giving rise to major liver toxicity (veno-occlusive disease), and a wide interpatient and intrapatient pharmacokinetic variability."( Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study.
Aulagner, G; Bertholle-Bonnet, V; Bertrand, Y; Bleyzac, N; Galambrun, C; Mialou, V; Souillet, G, 2007
)
1.53
" The population pharmacokinetic analysis revealed a clearance of 86."( Cytotoxicity of dimethylacetamide and pharmacokinetics in children receiving intravenous busulfan.
Boos, J; Gruhn, B; Hempel, G; Klingebiel, T; Lanvers-Kaminsky, C; Oechtering, D; Vormoor, J, 2007
)
0.56
" In the (T)BU/CY patients only, the association of the pharmacokinetic data with liver toxicity, relapse, and survival was evaluated."( Cyclophosphamide following targeted oral busulfan as conditioning for hematopoietic cell transplantation: pharmacokinetics, liver toxicity, and mortality.
Batchelder, A; Cole, S; Deeg, HJ; Gooley, T; McCune, JS; McDonald, GB; Phillips, B; Schoch, HG, 2007
)
0.61
"The objectives of this study were to develop a population pharmacokinetic model and to determine the covariates affecting the pharmacokinetics of busulfan in Japanese pediatric patients who received high-dose oral busulfan as a conditioning regimen before hematopoietic stem cell transplantation."( Population pharmacokinetics of oral busulfan in young Japanese children before hematopoietic stem cell transplantation.
Ariyoshi, N; Kitada, M; Miura, G; Nakamura, H; Nakazawa, K; Okada, K; Sato, T, 2008
)
0.82
" Blood samples (4-10) were collected after the first dose, busulphan concentrations were measured and pharmacokinetic parameters, including clearance (CL) and area under the concentration-time curve (AUC), were determined using the Kinetica software (Innaphase)."( Variability in the pharmacokinetics of intravenous busulphan given as a single daily dose to paediatric blood or marrow transplant recipients.
Earl, JW; Nath, CE; Pati, N; Shaw, PJ; Stephen, K, 2008
)
0.35
" 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
"The effect of glutathione S-transferase variants on pediatric busulfan metabolism was investigated by noncompartmental and population pharmacokinetic modeling."( Glutathione S-transferase A1 genetic variants reduce busulfan clearance in children undergoing hematopoietic cell transplantation.
Baker, KS; Brundage, R; Cao, Q; Farag-El Maasah, S; Jacobson, PA; Johnson, L; Langer, E; Orchard, PJ; Remmel, R; Ross, JA; Wang, X, 2008
)
0.84
" Pharmacokinetic parameters were estimated by using nonlinear mixed-effect modeling (NONMEM)."( Glutathione S-transferase polymorphisms are not associated with population pharmacokinetic parameters of busulfan in pediatric patients.
Bartelink, IH; Boelens, JJ; Bredius, RG; den Hartigh, J; Guchelaar, HJ; Press, RR; van Derstraaten, TR; Zwaveling, J, 2008
)
0.56
"Lifespan-based pharmacodynamic (PD) models of cellular response assume that the lifespan of cells is predetermined at the time of cellular production, despite recognized changes in the cellular environment following production that may alter the survival of the cells."( Pharmacodynamic modeling of the effect of changes in the environment on cellular lifespan and cellular response.
Freise, KJ; Schmidt, RL; Veng-Pedersen, P; Widness, JA, 2008
)
0.35
" Pharmacokinetic parameters were evaluated following first dose using a two-compartment disposition model."( Pharmacokinetics of high-dose i.v. treosulfan in children undergoing treosulfan-based preparative regimen for allogeneic haematopoietic SCT.
Grund, G; Główka, FK; Karaźniewicz-Łada, M; Wachowiak, J; Wróbel, T, 2008
)
0.35
" The goal of this retrospective analysis was to evaluate any age-related pharmacokinetic and pharmacodynamic differences in pediatric patients who received BU as a conditioning agent."( Evaluating pharmacokinetics and pharmacodynamics of intravenous busulfan in pediatric patients receiving bone marrow transplantation.
Ikeda, A; Kim, AH; Moore, TB; Tse, JC, 2009
)
0.59
" Results of clinical outcome of previous studies performed to optimize busulfan and CsA therapy by controlling their pharmacokinetic variability by means of maximum a posteriori (MAP) Bayesian individualization of both drugs are presented."( The use of pharmacokinetic models in paediatric onco-haematology: effects on clinical outcome through the examples of busulfan and cyclosporine.
Bleyzac, N, 2008
)
0.79
" Multivariate regression models showed that, other than the drug dose and age, the GSTM1 genotype was the best predictor of first-dose pharmacokinetic variability."( Influence of GST gene polymorphisms on busulfan pharmacokinetics in children.
Ansari, M; Champagne, MA; Duval, M; Krajinovic, M; Lauzon-Joset, JF; Théoret, Y; Vachon, MF, 2010
)
0.63
" Population pharmacokinetic modeling and determination of an optimal pharmacokinetic sampling schedule over 6 hours could allow for personalizing these busulfan doses in the outpatient clinic."( Development of a population pharmacokinetics-based sampling schedule to target daily intravenous busulfan for outpatient clinic administration.
Blough, DK; McCune, JS; O'Donnell, PV; Pawlikowski, MA; Salinger, DH; Vicini, P, 2010
)
0.78
"We developed a population pharmacokinetic model of oral busulfan in Japanese adults."( Population pharmacokinetic study of a test dose oral busulfan in Japanese adult patients undergoing hematopoietic stem cell transplantation.
Hara, S; Jimi, S; Ogata, K; Sasaki, N; Takamatsu, Y; Tamura, K; Yukawa, E, 2010
)
0.86
" Pharmacokinetic (PK) data were compared with those previously collected in patients (n=127) treated with phenytoin for seizure prophylaxis."( Influence on Busilvex pharmacokinetics of clonazepam compared to previous phenytoin historical data.
Bacigalupo, A; Buzyn, A; Cahn, JY; Carreras, E; Kröger, N; Puozzo, C; Sanz, G; Vernant, JP, 2010
)
0.36
" We retrospectively compared outcomes of a consecutive series of 51 AML patients treated with oral busulfan (1 mg/kg every 6 hours for 4 days) and cyclophosphamide (60 mg/kg IV × 2 days) - (Bu/Cy) with 100 consecutive AML patients treated with pharmacokinetic targeted IV busulfan (AUC < 6000 μM/L*min per day × 4 days) and fludarabine (40 mg/m2 × 4 days) - (t-IV Bu/Flu)."( Pharmacokinetic targeting of intravenous busulfan reduces conditioning regimen related toxicity following allogeneic hematopoietic cell transplantation for acute myelogenous leukemia.
Anasetti, C; Fernandez, HF; Field, T; Kharfan-Dabaja, MA; Kim, J; Nishihori, T; Perez, L; Perkins, J; Pidala, J, 2010
)
0.84
") busulfan can produce a more consistent pharmacokinetic profile than oral formulations can, but nonetheless, significant interpatient variability is evident."( Influence of GST gene polymorphisms on the clearance of intravenous busulfan in adult patients undergoing hematopoietic cell transplantation.
Bae, KS; Choi, Y; Han, SB; Hur, EH; Kim, DY; Kim, SD; Lee, JH; Lee, KH; Lim, HS; Lim, SN; Noh, GJ; Yun, SC, 2011
)
1.33
"A prospective clinical trial was performed in order to validate the pharmacokinetic (PK) and clinical benefits of a new dosing schedule of intravenous busulfan (IV Bu) in children."( Weight-based strategy of dose administration in children using intravenous busulfan: clinical and pharmacokinetic results.
Bertrand, Y; Bordigoni, P; Demeocq, F; Doz, F; Esperou, H; Frappaz, D; Galambrun, C; Gentet, JC; Méchinaud, F; Michel, G; Neven, B; Nguyen, L; Socié, G; Valteau-Couanet, D; Vassal, G; Yakouben, K, 2012
)
0.81
" A one-compartment population model was used to estimate individual pharmacokinetic parameters."( Effect of genetic polymorphisms in genes encoding GST isoenzymes on BU pharmacokinetics in adult patients undergoing hematopoietic SCT.
den Hartigh, J; Guchelaar, HJ; ten Brink, MH; van der Straaten, T; von dem Borne, PA; Wessels, JA; Zwaveling, J, 2012
)
0.38
"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
"Busulfan pharmacokinetic (PK) monitoring of the area under the concentration-time curve (AUC) is necessary to minimize adverse events associated with both under- and over-dosing of busulfan during hematopoietic stem cell transplantation (HSCT)."( Clinical consequences of analytical variance and calculation strategy in oral busulfan pharmacokinetics.
Clarke, W; Lombardi, L; Olson, MT, 2011
)
2.04
"Busulfan has a narrow therapeutic range, and in children, pharmacokinetic variability has been found to be high even after the use of intravenous (i."( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
2.06
"The clinical advantage of pharmacokinetic (PK)-directed-based dosing on intravenous (i."( Pharmacokinetic-directed high-dose busulfan combined with cyclophosphamide and etoposide results in predictable drug levels and durable long-term survival in lymphoma patients undergoing autologous stem cell transplantation.
Ali, Z; Dada, MO; Flowers, CR; Graiser, M; Hutcherson, DA; McMillan, S; Waller, EK; Zhang, H, 2012
)
0.66
" The authors conducted an observational study aiming to investigate the behavior and ontogeny of IV Bu pharmacokinetic (PK) disposition, and to reevaluate the consistency of the BW-based dosing in very young children with rare diseases."( Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: the results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation.
Abdi, ZD; Bleyzac, N; Broutin, S; Dalle, JH; Galambrun, C; Kemmel, V; Moshous, D; Neven, B; Nguyen, L; Paci, A; Pétain, A; Vassal, G, 2012
)
0.63
" Covariates were selected on the basis of their known or theoretical relationships with busulfan pharmacokinetics and were plotted independently against the individual pharmacokinetic parameters and the weighted residuals of the model without covariates to visualize relations."( Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing.
Bartelink, IH; Bierings, MB; Boelens, JJ; Bredius, RG; Danhof, M; Egberts, AC; Hempel, G; Knibbe, CA; Nath, CE; Shaw, PJ; Wang, C; Zwaveling, J, 2012
)
0.87
" Busulfan analysis was carried out by gas chromatography-mass spectrometry and data analysed using a NONMEM population pharmacokinetic approach."( Busulfan pharmacokinetics following intravenous and oral dosing regimens in children receiving high-dose myeloablative chemotherapy for high-risk neuroblastoma as part of the HR-NBL-1/SIOPEN trial.
Amiel, M; Brock, P; Ladenstein, R; Nguyen, L; Paci, A; Riggi, M; Valteau-Couanet, D; Vassal, G; Veal, GJ, 2012
)
2.73
"Busulfan plasma concentrations obtained from 38 patients receiving IV busulfan and 25 patients receiving oral busulfan, were fitted simultaneously using a one-compartment pharmacokinetic model."( Busulfan pharmacokinetics following intravenous and oral dosing regimens in children receiving high-dose myeloablative chemotherapy for high-risk neuroblastoma as part of the HR-NBL-1/SIOPEN trial.
Amiel, M; Brock, P; Ladenstein, R; Nguyen, L; Paci, A; Riggi, M; Valteau-Couanet, D; Vassal, G; Veal, GJ, 2012
)
3.26
"The reduced pharmacokinetic variability and improved control of busulfan AUC observed following IV administration support its utility within the ongoing HR-NBL-1/SIOPEN trial."( Busulfan pharmacokinetics following intravenous and oral dosing regimens in children receiving high-dose myeloablative chemotherapy for high-risk neuroblastoma as part of the HR-NBL-1/SIOPEN trial.
Amiel, M; Brock, P; Ladenstein, R; Nguyen, L; Paci, A; Riggi, M; Valteau-Couanet, D; Vassal, G; Veal, GJ, 2012
)
2.06
" The present study provides an integration of pharmacokinetic and genetic data of 63 adults with acute myeloid leukemia (AML) preconditioned for HSCT with high dose oral BU, with the aim of defining biomarkers predictive of poor BU metabolism."( Pharmacokinetic and pharmacogenetic analysis of oral busulfan in stem cell transplantation: prediction of poor drug metabolism to prevent drug toxicity.
Efrati, E; Elkin, H; Froymovich, L; Krivoy, N; Rowe, JM; Zuckerman, T, 2012
)
0.63
"Recently a pediatric pharmacokinetic (PK) model was developed for busulfan to explain the wide variability in PK of busulfan in children, as this variability is known to influence the outcome of hematopoietic stem cell transplantation in terms of toxicity and event free survival."( Predictive performance of a busulfan pharmacokinetic model in children and young adults.
Bartelink, IH; Bierings, MB; Boelens, JJ; Chiesa, R; Cuvelier, GD; Danhof, M; Egberts, TC; Knibbe, CA; Slatter, MA; van Kesteren, C; Wynn, RF, 2012
)
0.91
" For the purpose of efficient function as platelet substitute, H12-(ADP)-liposomes should potentially have both acceptable pharmacokinetic and biodegradable properties under conditions of an adaptation disease including thrombocytopenia induced by anticancer drugs."( Pharmacokinetic study of adenosine diphosphate-encapsulated liposomes coated with fibrinogen γ-chain dodecapeptide as a synthetic platelet substitute in an anticancer drug-induced thrombocytopenia rat model.
Doi, M; Fujiyama, A; Handa, M; Ikeda, Y; Maruyama, T; Otagiri, M; Taguchi, K; Takeoka, S; Ujihira, H; Watanabe, H, 2013
)
0.39
" Plasma busulfan concentrations were determined by HPLC-UV and the pharmacokinetic parameters were calculated using the WinNonlin program."( Influence of fludarabine on the pharmacokinetics of oral busulfan during pretransplant conditioning for hematopoietic stem cell transplantation.
Colturato, VA; de Castro, FA; Lanchote, VL; Simões, BP; Voltarelli, JC, 2013
)
1.07
"A physiologically based pharmacokinetic (PBPK) model was established and evaluated describing the pharmacokinetics (PK) of the DNA-alkylating agent Busulfan in adults in order to predict the systemic Busulfan drug exposure in both plasma and toxicity-related organs."( Physiologically based pharmacokinetic modelling of Busulfan: a new approach to describe and predict the pharmacokinetics in adults.
Boos, J; Diestelhorst, C; Hempel, G; Kangarloo, SB; McCune, JS; Russell, J, 2013
)
0.84
"When growing up, the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of drugs change, which may alter the effect of drugs."( Towards evidence-based dosing regimens in children on the basis of population pharmacokinetic pharmacodynamic modelling.
Admiraal, R; Boelens, JJ; Bredius, RG; Knibbe, CA; Tibboel, D; van Kesteren, C, 2014
)
0.4
" Pharmacokinetic data to optimize treosulfan dosing are scarce in this patient population."( Pharmacokinetics of treosulfan in pediatric patients undergoing hematopoietic stem cell transplantation.
Ackaert, O; Bredius, RG; den Hartigh, J; Guchelaar, HJ; Lankester, AC; Smiers, FJ; Ten Brink, MH; Zwaveling, J, 2014
)
0.4
"We developed a new population pharmacokinetic (PopPK) model for intravenous (i."( Population pharmacokinetics of intravenous busulfan in children: revised body weight-dependent NONMEM® model to optimize dosing.
Boos, J; Diestelhorst, C; Hempel, G; McCune, JS, 2014
)
0.67
"A physiologically based pharmacokinetic (PBPK) model of the DNA-alkylating agent busulfan was slightly modified and scaled from adults to children in order to predict the systemic busulfan drug exposure in children."( Predictive performance of a physiologically based pharmacokinetic model of busulfan in children.
Boos, J; Diestelhorst, C; Hempel, G; Kangarloo, SB; McCune, JS; Russell, J, 2014
)
0.86
"Population pharmacokinetic (PK) studies of busulfan in children have shown that individualized model-based algorithms provide improved targeted busulfan therapy when compared with conventional dose guidelines."( Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use.
Bartelink, I; Cowan, MJ; Dvorak, CC; French, D; Horn, B; Law, J; Long-Boyle, JR; Musick, L; Savic, R; Yan, S, 2015
)
0.97
"One hundred and eighty-five patients were accrued; 174 provided useable pharmacokinetic data."( Effect of age on the pharmacokinetics of busulfan in patients undergoing hematopoietic cell transplantation; an alliance study (CALGB 10503, 19808, and 100103).
Alyea, EP; Beumer, JH; Blum, W; Christner, SM; Devine, S; Egorin, MJ; Holleran, JL; Jiang, C; Kolitz, JE; Larson, RA; Lewis, LD; Linker, C; Owzar, K; Ratain, MJ; Vij, R, 2014
)
0.67
" This study aimed to assess busulfan pharmacokinetic variability in pretransplant conditioning regimens using an analytical method validated by high-performance liquid chromatography coupled to diode array detector (HPLC/PDA)."( Individualizing oral busulfan dose after using a test dose in patients undergoing hematopoietic stem cell transplantation: pharmacokinetic characterization.
Bariani, C; Carneiro, WJ; Cunha, LC; de Moraes Arantes, A; de Oliveira Neto, JR; Effting, C; Queiroz Labre, LV; Rodrigues, AR; Rodrigues, CR, 2015
)
1.03
"Eight patients who used the test dose (TD) of 1 mg/kg busulfan 10 days before conditioning were included, and 10 serial blood samples were collected to determine: the elimination half-life (t1/2), total area under the curve (AUCT), total clearance (Cl(T)/F), and plasma concentration at steady state (C(ss)), using a monocompartmental model and first-order kinetics."( Individualizing oral busulfan dose after using a test dose in patients undergoing hematopoietic stem cell transplantation: pharmacokinetic characterization.
Bariani, C; Carneiro, WJ; Cunha, LC; de Moraes Arantes, A; de Oliveira Neto, JR; Effting, C; Queiroz Labre, LV; Rodrigues, AR; Rodrigues, CR, 2015
)
0.98
"High variability in the assessed pharmacokinetic parameters was observed, with a 38% variation in C(ss) between TD and conditioning regimen; Cl(T)/F decreased by 30%, suggesting drug accumulation after multiple-dose regimen."( Individualizing oral busulfan dose after using a test dose in patients undergoing hematopoietic stem cell transplantation: pharmacokinetic characterization.
Bariani, C; Carneiro, WJ; Cunha, LC; de Moraes Arantes, A; de Oliveira Neto, JR; Effting, C; Queiroz Labre, LV; Rodrigues, AR; Rodrigues, CR, 2015
)
0.74
" Pharmacokinetic treatment with targeted intravenous busulfan combined with fludarabine (BuFlu) was developed as a preparative regimen for acute leukemia and myelodysplasia."( Myeloablative intravenous pharmacokinetically targeted busulfan plus fludarabine as conditioning for allogeneic hematopoietic cell transplantation in patients with non-Hodgkin lymphoma.
Anasetti, C; Ayala, E; Figueroa, J; Kharfan-Dabaja, MA; Kim, J; Locke, F; Nishihori, T; Perkins, J; Riches, M; Yue, B, 2015
)
0.91
" In conclusion, once-daily administration of ivBu has a stable pharmacokinetic profile, and was safely performed in Japanese patients."( Pharmacokinetics study of once-daily intravenous busulfan in conditioning regimens for hematopoietic stem cell transplantation.
Akahoshi, Y; Ashizawa, M; Ishihara, Y; Kako, S; Kanda, J; Kanda, Y; Kawamura, K; Kikuchi, M; Kimura, S; Matsumoto, K; Morita, K; Nakano, H; Nakasone, H; Nishida, J; Oshima, K; Sakamoto, K; Sato, M; Tanaka, Y; Tanihara, A; Terasako-Saito, K; Ugai, T; Wada, H; Yamasaki, R; Yamazaki, R, 2015
)
0.67
" This regimen was associated with durable donor engraftment and relatively low rates of regimen related toxicity (RRT); future alemtuzumab pharmacokinetic studies may improve outcomes, by allowing targeted alemtuzumab clearance to reduce graft rejection and promote more rapid immune reconstitution."( Unrelated donor hematopoietic stem cell transplantation for the treatment of non-malignant genetic diseases: An alemtuzumab based regimen is associated with cure of clinical disease; earlier clearance of alemtuzumab may be associated with graft rejection.
Abdel-Azim, H; Crooks, GM; Kapoor, N; Khazal, S; Kohn, DB; Mahadeo, KM; Shah, AJ; Zhao, Q, 2015
)
0.42
"A population pharmacokinetic (PPK) analysis was conducted to describe the influence of GSTA1 polymorphisms on intravenous busulfan in adults undergoing allogeneic hematopoietic stem cell transplantation."( Population pharmacokinetics and pharmacodynamics of busulfan with GSTA1 polymorphisms in patients undergoing allogeneic hematopoietic stem cell transplantation.
Choi, B; Han, N; Ji, E; Kim, IW; Kim, MG; Kim, T; Oh, JM; Park, S, 2015
)
0.88
" Pharmacokinetic parameters were determined in six children who received intravenous treosulfan (dose range 12-24 g/m(2)) in combination with fludarabine prior to blood or marrow transplantation."( Development and Validation of a High Pressure Liquid Chromatography-UV Method for the Determination of Treosulfan and Its Epoxy Metabolites in Human Plasma and Its Application in Pharmacokinetic Studies.
Byrne, JA; Earl, JW; Fraser, CJ; Koyyalamudi, SR; Kuzhiumparambil, U; Nath, CE; O'Brien, TA; Shaw, PJ, 2016
)
0.43
" Then EBDM and DEB have the same elimination half-life as TREO, but the levels of EBDM and DEB in the body, including plasma, are much lower than TREO on account of their inherently high clearance."( Formation Rate-Limited Pharmacokinetics of Biologically Active Epoxy Transformers of Prodrug Treosulfan.
Główka, FK; Karbownik, A; Kasprzyk, A; Romański, M; Szałek, E, 2016
)
0.43
"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
)
1.01
" 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
)
0.73
"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
)
1.01
" 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
)
1.11
" Pharmacokinetic evaluation of a busulfan test dose before the start of the conditioning regimen would allow for all conditioning regimen doses to be given at the calculated optimized dose."( Evaluation of the Pharmacokinetics and Efficacy of a Busulfan Test Dose in Adult Patients Undergoing Myeloablative Hematopoietic Cell Transplantation.
Canadeo, A; Eastwood, D; Hamadani, M; Hari, P; Oxencis, C; Pasquini, M; Urmanski, A; Waggoner, M; Weil, E; Zook, F, 2017
)
0.99
" The daily pharmacokinetic analysis revealed that the clearance reduced to 57% of that before the coadministration."( A Significant Influence of Metronidazole on Busulfan Pharmacokinetics: A Case Report of Therapeutic Drug Monitoring.
Choi, JY; Chung, H; Hong, CR; Hong, KT; Kang, HJ; Lee, S; Park, KD; Shin, HY; Yu, KS, 2017
)
0.72
"Using pharmacokinetic parameters obtained from 385 drug administrations in 352 children aged from 1 month to 18 years, treated with five drugs (dactinomycin, busulfan, carboplatin, cyclophosphamide and etoposide), individual exposures (area under the plasma drug concentration versus time curve; AUC) obtained using doses rounded according to the published NHSE tables were calculated and compared with those obtained by standard dose calculation methods."( Investigating the potential impact of dose banding for systemic anti-cancer therapy in the paediatric setting based on pharmacokinetic evidence.
Boddy, AV; Chatelut, E; Osborne, C; Paci, A; Veal, GJ; White-Koning, M, 2018
)
0.68
"Based on pharmacokinetic data for these five drugs, the results generated support the implementation of NHSE dose-banding tables."( Investigating the potential impact of dose banding for systemic anti-cancer therapy in the paediatric setting based on pharmacokinetic evidence.
Boddy, AV; Chatelut, E; Osborne, C; Paci, A; Veal, GJ; White-Koning, M, 2018
)
0.48
"The aim of this study is to develop a population pharmacokinetic (PopPK) model for intravenous busulfan in children that incorporates variants of GSTA1, gene coding for the main enzyme in busulfan metabolism."( Incorporation of GSTA1 genetic variations into a population pharmacokinetic model for IV busulfan in paediatric hematopoietic stem cell transplantation.
Ansari, M; Bittencourt, H; Daudt, LE; Duval, M; Huezo-Diaz Curtis, P; Kassir, N; Krajinovic, M; Litalien, C; Nava, T; Rezgui, MA; Théoret, Y; Uppugunduri, CRS, 2018
)
0.92
" non-malignant), age, conditioning regimen and GSTA1 diplotypes were evaluated as covariates of pharmacokinetic parameters by using nonlinear mixed effects analysis."( Incorporation of GSTA1 genetic variations into a population pharmacokinetic model for IV busulfan in paediatric hematopoietic stem cell transplantation.
Ansari, M; Bittencourt, H; Daudt, LE; Duval, M; Huezo-Diaz Curtis, P; Kassir, N; Krajinovic, M; Litalien, C; Nava, T; Rezgui, MA; Théoret, Y; Uppugunduri, CRS, 2018
)
0.7
"Allometric approaches are widely used for interspecies scaling for the prediction of pharmacokinetic (PK) parameters during drug development."( Multistep Unified Models Using Prior Knowledge for the Prediction of Drug Clearance in Neonates and Infants.
Forshee, R; Jiang, Z; Mahmood, I; Tegenge, MA, 2018
)
0.48
" For that purpose, pharmacokinetic analyses are ongoing within clinical phase II and III trials."( Clinical bioanalysis of treosulfan and its epoxides: The importance of collected blood processing for valid pharmacokinetic results.
Główka, F; Romański, M, 2018
)
0.48
" The significance of the elimination half-life of treosulfan and its epoxides for successful conditioning prior to HSCT is also raised."( Treosulfan Pharmacokinetics and its Variability in Pediatric and Adult Patients Undergoing Conditioning Prior to Hematopoietic Stem Cell Transplantation: Current State of the Art, In-Depth Analysis, and Perspectives.
Główka, FK; Romański, M; Wachowiak, J, 2018
)
0.48
" There is still an important variation in Bu pharmacokinetic between patients that is associated with an increased risk of toxicity and graft failure."( Pharmacokinetics-adapted Busulfan-based myeloablative conditioning before unrelated umbilical cord blood transplantation for myeloid malignancies in children.
Ansari, M; Benadiba, J; Bittencourt, H; Cellot, S; Duval, M; Krajinovic, M; Teira, P; Vachon, MF, 2018
)
0.78
" A population pharmacokinetic analysis with the objective of personalizing therapy in Japanese patients was conducted by integrating pediatric patient data with adult patient data."( Population Pharmacokinetic Analysis of Busulfan in Japanese Pediatric and Adult HCT Patients.
Funaki, T; Kawazoe, A; Kim, S, 2018
)
0.75
" The objective of this retrospective study was to determine the pharmacokinetic impact of using ideal body weight as the initial dosing weight in obese as compared to non-obese transplant recipients."( Pharmacokinetic and clinical outcomes when ideal body weight is used to dose busulfan in obese hematopoietic stem cell transplant recipients.
Griffin, SP; Hsu, JW; Murthy, HS; Richards, AI; Wheeler, SE; Wiggins, LE, 2019
)
0.74
" We used an intensive daily pharmacokinetic monitoring method for busulfan dosing in children for effective myeloablation and to reduce toxicity."( Favorable Outcome of Post-Transplantation Cyclophosphamide Haploidentical Peripheral Blood Stem Cell Transplantation with Targeted Busulfan-Based Myeloablative Conditioning Using Intensive Pharmacokinetic Monitoring in Pediatric Patients.
Cheon, JE; Choi, JY; Hong, CR; Hong, KT; Jang, IJ; Kang, HJ; Park, JD; Park, KD; Shin, HY; Song, SH; Yu, KS, 2018
)
0.92
" Busulfan Cmax as well as weight <9 kg or age <3 years were identified as independent predictors of VOD in logistic regression analysis."( Maximal concentration of intravenous busulfan as a determinant of veno-occlusive disease: a pharmacokinetic-pharmacodynamic analysis in 293 hematopoietic stem cell transplanted children.
Bertrand, Y; Bleyzac, N; Goutelle, S; Neely, M; Philippe, M; Rushing, T, 2019
)
1.7
" The objective of this study was to evaluate busulfan exposure from a pharmacokinetic (PK)-guided dosing strategy using a test dose."( Evaluation of a Test Dose Strategy for Pharmacokinetically-Guided Busulfan Dosing for Hematopoietic Stem Cell Transplantation.
Alexander, MD; Armistead, PM; Chung, Y; Coghill, JM; Davis, JM; Ivanova, A; Jamieson, KJ; Ptachcinski, JR; Rao, KV; Riches, ML; Serody, JS; Sharf, AA; Shaw, JR; Shea, TC; Vincent, BG; Wood, WA, 2019
)
1.01
"This meta-analysis was conducted to derive an integrated conclusion about the influence of glutathione S-transferase (GST) genetic polymorphisms on busulfan pharmacokinetic (PK) parameters and veno-occlusive disease (VOD)."( Effect of glutathione S-transferase genetic polymorphisms on busulfan pharmacokinetics and veno-occlusive disease in hematopoietic stem cell transplantation: A meta-analysis.
Choi, B; Ji, E; Kim, K; Kim, MG; Kwak, A; Oh, JM, 2019
)
0.95
" The aim of this study was to develop a population pharmacokinetic (PK) model of treosulfan in paediatric haematopoietic stem cell transplantation recipients and to explore the effect of potential covariates on treosulfan PK."( Population pharmacokinetics of treosulfan in paediatric patients undergoing hematopoietic stem cell transplantation.
Bertaina, A; Guchelaar, HJ; Lankester, AC; Locatelli, F; Moes, DJAR; van der Stoep, MYEC; Zwaveling, J, 2019
)
0.51
"This study aimed to develop a population pharmacokinetic (PPK) model to investigate the impact of GSTA1, GSTP1, and GSTM1 genotypes on busulfan pharmacokinetic (PK) variability in Chinese adult patients."( Population pharmacokinetic analysis of intravenous busulfan: GSTA1 genotype is not a predictive factor of initial dose in Chinese adult patients undergoing hematopoietic stem cell transplantation.
Chen, B; Hao, C; Hu, J; Huang, J; Li, Z; Liang, W; Sun, Y; Yang, W; Zhang, W, 2020
)
1.01
" However, TDM-based dosing may be challenging if intra-individual pharmacokinetic variability (also denoted inter-occasion variability [IOV]) occurs during therapy."( Intra-individual Pharmacokinetic Variability of Intravenous Busulfan in Hematopoietic Stem Cell-Transplanted Children.
Bertrand, Y; Bleyzac, N; Ducher, M; Goutelle, S; Guitton, J; Leclerc, V; Marsit, H; Neely, M; Philippe, M; Rushing, T, 2020
)
0.8
" We calculated individual pharmacokinetic parameters on each day of therapy using a published population pharmacokinetic model of busulfan and analyzed their changes."( Intra-individual Pharmacokinetic Variability of Intravenous Busulfan in Hematopoietic Stem Cell-Transplanted Children.
Bertrand, Y; Bleyzac, N; Ducher, M; Goutelle, S; Guitton, J; Leclerc, V; Marsit, H; Neely, M; Philippe, M; Rushing, T, 2020
)
1.01
" The population pharmacokinetic model was established in NONMEM software with a first-order estimation method with interaction."( Population pharmacokinetic approach for evaluation of treosulfan and its active monoepoxide disposition in plasma and brain on the basis of a rat model.
Danielak, D; Główka, F; Kasprzyk, A; Romański, M; Teżyk, A, 2020
)
0.56
"One-compartment pharmacokinetic model best described changes in the concentrations of treosulfan in plasma, and EBDM concentrations in plasma and in the brain."( Population pharmacokinetic approach for evaluation of treosulfan and its active monoepoxide disposition in plasma and brain on the basis of a rat model.
Danielak, D; Główka, F; Kasprzyk, A; Romański, M; Teżyk, A, 2020
)
0.56
"The developed population pharmacokinetic model is the first that allows the prediction of treosulfan and EBDM concentrations in rat plasma and brain."( Population pharmacokinetic approach for evaluation of treosulfan and its active monoepoxide disposition in plasma and brain on the basis of a rat model.
Danielak, D; Główka, F; Kasprzyk, A; Romański, M; Teżyk, A, 2020
)
0.56
" From a large cohort of 540 patients, we developed a Bu population pharmacokinetic model based on body weight (BW) and maturation concepts to reduce IIV and optimize exposure."( New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning.
Bondu, S; Bourget, P; Broutin, S; Dalle, JH; De Berranger, E; Devictor, B; Dufour, C; Faivre, L; Galambrun, C; Gandemer, V; Jubert, C; Kemmel, V; Mir, O; Moshous, D; Neven, B; Nguyen, L; Paci, A; Petain, A; Poinsignon, V; Vannier, JP; Vassal, G, 2020
)
0.77
" This multi-institutional prospective study compared pharmacokinetic (PK) parameters of Bu between BuFlu and BuCy."( Pharmacokinetics of intravenous busulfan as condition for hematopoietic stem cell transplantation: comparison between combinations with cyclophosphamide and fludarabine.
Hino, Y; Kato, J; Kikuchi, T; Koda, Y; Matsumoto, K; Mishina, T; Mori, T; Morita, K; Nagao, Y; Ohwada, C; Okamoto, S; Onizuka, M; Onoda, M; Sakaida, E; Shimizu, H; Shono, K; Takeda, Y; Yokota, A; Yokoyama, H, 2021
)
0.9
"This study aimed to predict the presence and mechanism of busulfan drug-drug interactions (DDIs) in hematopoietic stem cell transplantation (HSCT) using pharmacokinetic interaction (PKI) network-based molecular structure similarity and network pharmacology."( Predicting the presence and mechanism of busulfan drug-drug interactions in hematopoietic stem cell transplantation using pharmacokinetic interaction network-based molecular structure similarity and network pharmacology.
Hao, C; Hu, J; Huang, J; Ma, X; Wang, L; Yang, W; Zhang, W, 2021
)
1.13
" Bu shows wide pharmacokinetic (PK) variability among patients."( Feasibility and Efficacy of a Pharmacokinetics-Guided Busulfan Conditioning Regimen for Allogeneic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis in Adult Patients with Hematologic Malignancie
Bartoli, A; Bramanti, S; Castagna, L; De Gregori, S; De Philippis, C; Giordano, L; Mannina, D; Mariotti, J; Pieri, G; Roperti, M; Sarina, B; Valli, V, 2021
)
0.87
"Busulfan (Bu) is a common component of conditioning regimens before hematopoietic stem cell transplantation (HSCT) and is known for high interpatient pharmacokinetic (PK) variability."( Precision dosing of intravenous busulfan in pediatric hematopoietic stem cell transplantation: Results from a multicenter population pharmacokinetic study.
Ansari, M; Ben Hassine, K; Bittencourt, H; Bredius, RGM; Daali, Y; Kassir, N; Krajinovic, M; Lewis, V; Nath, CE; Nava, T; Shaw, PJ; Théoret, Y; Uppugunduri, CRS, 2021
)
2.35
"The aim of this work is the development of a mechanistic physiologically-based pharmacokinetic (PBPK) model using in vitro to in vivo extrapolation to conduct a drug-drug interaction (DDI) assessment of treosulfan against two cytochrome p450 (CYP) isoenzymes and P-glycoprotein (P-gp) substrates."( Evaluation of the drug-drug interaction potential of treosulfan using a physiologically-based pharmacokinetic modelling approach.
Balazki, P; Baumgart, J; Beelen, DW; Böhm, S; Hemmelmann, C; Hilger, RA; Martins, FS; Ring, A; Schaller, S, 2022
)
0.72
" However, considering the comprehensive treosulfan-based conditioning treatment schedule and the respective pharmacokinetic properties of the concomitantly used drugs (eg, half-life), the potential for interaction on all evaluated mechanisms would be low (AUCR < 1."( Evaluation of the drug-drug interaction potential of treosulfan using a physiologically-based pharmacokinetic modelling approach.
Balazki, P; Baumgart, J; Beelen, DW; Böhm, S; Hemmelmann, C; Hilger, RA; Martins, FS; Ring, A; Schaller, S, 2022
)
0.72
" As of 2020, there is no population pharmacokinetic (popPK) model for busulfan in Chinese pediatric patients."( Can Published Population Pharmacokinetic Models of Busulfan Be Used for Individualized Dosing in Chinese Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation? An External Evaluation.
Chen, S; Hu, J; Huang, H; Huang, W; Li, D; Lin, S; Liu, M; Ren, J; Wu, X; Yang, T, 2022
)
1.21
"The study objective was to develop a population pharmacokinetic model for busulfan to comprehensively examine drug-drug interactions in paediatric patients undergoing haematopoietic stem cell transplantation."( Characterization of drug-drug interactions on the pharmacokinetic disposition of busulfan in paediatric patients during haematopoietic stem cell transplantation conditioning.
Dunn, A; Gobburu, JVS; Ivaturi, V; Moffett, BS, 2022
)
1.18
" Population pharmacokinetic (popPK) models enable description of busulfan PK and optimization of exposure, which leads to improvement of event-free survival after HCT."( Busulfan dose Recommendation in Inherited Metabolic Disorders: Population Pharmacokinetic Analysis.
Gupta, AO; Illamola, SM; Jennissen, CA; Long, SE; Long-Boyle, JR; Lund, TC; Orchard, PJ; Takahashi, T, 2022
)
2.4
" In addition to nomograms, therapeutic drug monitoring (TDM) of busulfan measuring plasmatic concentrations to estimate busulfan pharmacokinetic parameters can be used."( Individualizing busulfan dose in specific populations and evaluating the risk of pharmacokinetic drug-drug interactions.
Combarel, D; Delahousse, J; Paci, A; Tran, J; Vassal, G, 2023
)
1.49
" Pharmacokinetic (PK)-guided test-dose strategies have been shown to improve the number of patients achieving busulfan exposure goals and improve clinical outcomes."( Utilization of a Population Pharmacokinetic Model to Improve a Busulfan Test-Dose Strategy in Allogeneic Hematopoietic Cell Transplant Recipients.
Armistead, PM; Crona, DJ; DeVane, SC; Dunlap, TC; Kardouh, M; Kemper, RM; Ptachcinski, JR; Shaw, JR; Symonds, A; Weiner, DL, 2023
)
1.36
" Model-informed precision dosing (MIPD) based on population pharmacokinetic (popPK) models has been implemented in the clinical settings."( Population Pharmacokinetic Model of Intravenous Busulfan in Hematopoietic Cell Transplantation: Systematic Review and Comparative Simulations.
Al-Kofahi, M; Brown, SJ; Jaber, MM; Takahashi, T, 2023
)
1.17

Compound-Compound Interactions

Cyclophosphamide (Cy) in combination with busulfan (Bu) or total body irradiation (TBI) is the most commonly used myeloablative conditioning regimen in patients with chronic myeloid leukemia (CML). To evaluate the efficacy and toxicity of two different etoposide (VP-16) dosages (30 or 45 mg/kg) as conditioning therapy followed by stem cell transplantation.

ExcerptReferenceRelevance
" Etoposide at a dose of 60-65 mg/kg in combination with TBI and cyclophosphamide was associated with a significantly increased incidence of life threatening or fatal toxicities compared with a combination using a dose of 25-50 mg/kg (15 of 24 vs."( Etoposide in combination with cyclophosphamide and total body irradiation or busulfan as conditioning for marrow transplantation in adults and children.
Cahill, R; Deeg, HJ; Gadner, H; Ortlieb, M; Peters, C; Spitzer, TR; Tefft, MC; Torrisi, J; Urban, C, 1994
)
0.52
"Although toxicities with bone marrow transplant preparative regimens containing etoposide in combination with cyclophosphamide and total body irradiation or busulfan were frequently severe, treatment related mortality risk was believed to be acceptably low."( Etoposide in combination with cyclophosphamide and total body irradiation or busulfan as conditioning for marrow transplantation in adults and children.
Cahill, R; Deeg, HJ; Gadner, H; Ortlieb, M; Peters, C; Spitzer, TR; Tefft, MC; Torrisi, J; Urban, C, 1994
)
0.71
" At our institution, we are currently testing the efficacy and toxicity of regimens in which cytarabine or diaziquone are administered in combination with Bu and Cy."( New preparative regimens with diaziquone or cytarabine in combination with busulfan and cyclophosphamide.
Devine, SM; Geller, RB; Holland, HK; Saral, R; Wingard, JR, 1993
)
0.52
"The authors present results of chronic myeloid leukemia (CML) treatment with recombinant and leukocytic interferon in monotherapy and in combination with myelosan."( [Use of recombinant alpha2-interferon in combination with myelosan for the treatment of patients with chronic myeloid leukemia].
Bondare, DK; Feldmane, GIa; Mauritsas, M; Petukhov, VI; Strozha, I,
)
0.13
"To evaluate the efficacy and toxicity of two different etoposide (VP-16) dosages (30 or 45 mg/kg) in combination with busulfan/cyclophosphamide as conditioning therapy followed by stem cell transplantation in acute myeloid leukemia (AML), 90 patients with AML received either 30 mg/kg (n = 60) or 45 mg/kg (n = 30) etoposide in combination with busulfan (16 mg/kg) and cyclophosphamide (120 mg/kg)."( Dose-dependent effect of etoposide in combination with busulfan plus cyclophosphamide as conditioning for stem cell transplantation in patients with acute myeloid leukemia.
Braumann, D; Colberg, H; del Valle, F; Erttmann, R; Fiedler, W; Finkenstein, F; Holstein, K; Kabisch, H; Kröger, N; Krüger, W; Kuse, R; Mayer, U; Metzner, B; Renges, H; Sonnen, R; Sonnenberg, S; Stute, N; Zabelina, T; Zander, AR, 2000
)
0.76
"5-FU combined with busulfan could impair the hematopoietic stem cells and resulted in an irreversible aplastic anemia in rats, making a rat aplastic anemia model with hematopoietic stem cell failure."( [A novel rat aplastic anemia model induced by 5-fluorouracil combined with busulfan].
Chu, J; Ding, S; Liu, A; Wang, S; Xu, S; Zhao, J, 2001
)
0.87
"The safety and efficacy of oral metronomic low-dose treosulfan chemotherapy in combination with the cyclooxygenase-2 (COX-2) inhibitor rofecoxib as a compound with antiangiogenic potential, a therapeutic regimen optimally targeting endothelial cells instead of tumor cells, were assessed in pretreated advanced melanoma patients."( Metronomic oral low-dose treosulfan chemotherapy combined with cyclooxygenase-2 inhibitor in pretreated advanced melanoma: a pilot study.
Gille, J; Kaufmann, R; Spieth, K, 2003
)
0.32
"In this study we have used a standardised ATP-based tumour chemosensitivity assay (ATP-TCA) to measure the activity of gefitinib alone or in combination with different cytotoxic drugs (cisplatin, gemcitabine, oxaliplatin and treosulfan) against a variety of solid tumours (n = 86), including breast, colorectal, oesophageal and ovarian cancer, carcinoma of unknown primary site, cutaneous and uveal melanoma, non-small cell lung cancer (NSCLC) and sarcoma."( The in vitro effect of gefitinib ('Iressa') alone and in combination with cytotoxic chemotherapy on human solid tumours.
Cree, IA; Di Nicolantonio, F; Glaysher, S; Johnson, P; Knight, LA; Mercer, S; Sharma, S; Whitehouse, P, 2004
)
0.32
" Interestingly, gefitinib had both positive and negative effects when used in combination with different cytotoxics."( The in vitro effect of gefitinib ('Iressa') alone and in combination with cytotoxic chemotherapy on human solid tumours.
Cree, IA; Di Nicolantonio, F; Glaysher, S; Johnson, P; Knight, LA; Mercer, S; Sharma, S; Whitehouse, P, 2004
)
0.32
" Dose-escalated treosulphan (3 x 12 or 3 x 14 g/m2) combined with cyclophosphamide (Cy) was chosen for a new preparative regimen before allogeneic haematopoietic stem cell transplantation in 18 patients (median age 44, range 19-64 years) with haematological malignancies, considered ineligible for other myeloablative preparative regimens."( Dose-escalated treosulphan in combination with cyclophosphamide as a new preparative regimen for allogeneic haematopoietic stem cell transplantation in patients with an increased risk for regimen-related complications.
Basara, N; Baumgart, J; Beelen, DW; Casper, J; Fauser, AA; Freund, M; Hahn, JR; Hertenstein, B; Hilger, RA; Holler, E; Mylius, HA; Pichlmeier, U; Scheulen, ME; Trenschel, R, 2005
)
0.33
" In conclusion, although TH produced only a moderate effect against BCL1 leukemia when used alone, its combination with CY is promising and should be tested further in allogeneic murine models and clinical studies."( The effect of high-dose thiotepa, alone or in combination with other chemotherapeutic agents, on a murine B-cell leukemia model simulating autologous stem cell transplantation.
Abdul-Hai, A; Ergas, D; Resnick, IB; Shapira, MY; Slavin, S; Weiss, L, 2007
)
0.34
" We evaluated the efficacy and safety of yttrium-90-ibritumomab tiuxetan ((90)Y-ibritumomab) combined with intravenous busulfan, cyclophosphamide, and etoposide (Bu/Cy/E) followed by ASCT in patients with relapsed or refractory B-cell non-Hodgkin's lymphoma (NHL)."( Yttrium-90-ibritumomab tiuxetan in combination with intravenous busulfan, cyclophosphamide, and etoposide followed by autologous stem cell transplantation in patients with relapsed or refractory B-cell non-Hodgkin's lymphoma.
Choi, YH; Huh, J; Jang, G; Kang, BW; Kim, C; Kim, S; Kim, SW; Kim, WS; Lee, DH; Lee, JS; Lee, SS; Ryu, JS; Suh, C, 2010
)
0.81
" Gastrointestinal toxicity was the dose-limiting factor of Treo in combination with TBI."( Preclinical analysis of treosulfan in combination with total body irradiation as conditioning regimen prior to bone marrow transplantation in rats.
Baumgart, J; Casper, J; Freund, M; Hofmeister-Mielke, N; Pichlmeier, U; Sender, V; Sievert, K; Teifke, JP; Vogel, H; Wolff, D, 2009
)
0.35
"To evaluat the efficacy and safety of myeloablative allogeneic hematopoietic stem cell transplantation (allo-HSCT) combined with imatinib for advanced chronic myeloid leukemia (CML), 15 patients with accelerated phase (n=6) or blast crisis (n=9) were enrolled in this study."( Imatinib combined with myeloablative allogeneic hematopoietic stem cell transplantation for advanced phases of chronic myeloid leukemia.
Cai, Z; Han, X; He, J; Huang, H; Li, L; Lin, M; Luo, Y; Shi, J; Tan, Y; Xie, W; Ye, X; Zhao, Y; Zheng, Y, 2011
)
0.37
"A clinical study of triple drug combination (aprepitant+palonosetron+ dexamethasone) was carried out to evaluate its efficacy in preventing both acute and delayed emesis after high-dose chemotherapy (HDC) with busulphan+cyclophosphamide (BuCy) before hematopoietic stem cell transplantation (HSCT)."( Triple drug combination in the prevention of nausea and vomiting following busulfan plus cyclophosphamide chemotherapy before allogeneic hematopoietic stem cell transplantation.
Gawronski, K; Mlot, B; Oborska, S; Pielichowski, W; Rzepecki, P; Wasko-Grabowska, A,
)
0.36
"Patients treated with the triple drug combination had significantly higher response rates than those receiving palonosetron or ondansetron (+ dexamethasone) during both the acute and delayed phases: highly effective in early + late phases: 55 vs."( Triple drug combination in the prevention of nausea and vomiting following busulfan plus cyclophosphamide chemotherapy before allogeneic hematopoietic stem cell transplantation.
Gawronski, K; Mlot, B; Oborska, S; Pielichowski, W; Rzepecki, P; Wasko-Grabowska, A,
)
0.36
"This triple drug combination was more effective than ondansetron or palonosetron (+ dexamethasone) in preventing acute (especially), and delayed nausea and vomiting following BuCy chemotherapy before HSCT."( Triple drug combination in the prevention of nausea and vomiting following busulfan plus cyclophosphamide chemotherapy before allogeneic hematopoietic stem cell transplantation.
Gawronski, K; Mlot, B; Oborska, S; Pielichowski, W; Rzepecki, P; Wasko-Grabowska, A,
)
0.36
" We sought to determine the safety of GO in combination with busulfan/cyclophosphamide (Bu/Cy) conditioning before allogeneic hematopoietic stem cell transplantation (alloSCT)."( A Phase I study of gemtuzumab ozogamicin (GO) in combination with busulfan and cyclophosphamide (Bu/Cy) and allogeneic stem cell transplantation in children with poor-risk CD33+ AML: a new targeted immunochemotherapy myeloablative conditioning (MAC) regim
Baxter-Lowe, LA; Bhatia, M; Cairo, MS; Dela Cruz, F; Duffy, D; Foley, S; Garvin, JH; George, D; Hawks, R; Jin, Z; Le Gall, J; Morris, E; Satwani, P; Schwartz, J; van de Ven, C, 2012
)
0.86
" busulfan combined with fludarabine and analyzed the outcomes."( Highly variable pharmacokinetics of once-daily intravenous busulfan when combined with fludarabine in pediatric patients: phase I clinical study for determination of optimal once-daily busulfan dose using pharmacokinetic modeling.
Ahn, HS; Han, EJ; Jang, IJ; Jang, MK; Kang, HJ; Kim, H; Kim, NH; Lee, JW; Lee, SH; Park, KD; Shin, HY; Song, SH; Yu, KS; Yuk, YJ, 2012
)
1.53
" busulfan (Bu) in combination with clofarabine (Clo) in patients with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation (SCT)."( Clofarabine combined with busulfan provides excellent disease control in adult patients with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation.
Alousi, A; Andersson, BS; Basset, R; Champlin, RE; Ciurea, S; de Lima, M; Hosing, C; Jones, RB; Kebriaei, P; Khouri, I; Ledesma, C; Nieto, Y; Parmar, S; Popat, U; Qazilbash, M; Shpall, EJ, 2012
)
1.59
" This study evaluates the toxicity of escalating doses of topotecan alone or in combination with thiotepa or treosulfan."( Escalating topotecan in combination with treosulfan has acceptable toxicity in advanced pediatric sarcomas.
Bauer, F; Burdach, S; Filipiak-Pittroff, B; von Luettichau, I; Wawer, A, 2013
)
0.39
"Cyclophosphamide combined with total body irradiation (Cy/TBI) or busulfan (BuCy) are the most widely used myeloablative conditioning regimens for allotransplants."( Better leukemia-free and overall survival in AML in first remission following cyclophosphamide in combination with busulfan compared with TBI.
Ahn, KW; Aljurf, M; Arora, M; Avalos, B; Bolwell, BJ; Bredeson, C; Cahn, JY; Copelan, EA; Cortes, J; Costa, LJ; de Lima, M; Gale, RP; Hale, GA; Halter, J; Hamadani, M; Hamilton, BK; Horowitz, MM; Inamoto, Y; Kalaycio, ME; Kamble, RT; Litzow, MR; Loren, AW; Marks, DI; Maziarz, RT; Olavarria, E; Roy, V; Saber, W; Sabloff, M; Savani, BN; Schouten, HC; Seftel, M; Szer, J; Ustun, C; van Besien, K; Waller, EK; Weisdorf, DJ; Wirk, B; Zhu, X, 2013
)
0.84
" We therefore investigated the antiproliferative effects of nilotinib in combination with drugs that are usually used for conditioning: the alkylating agents mafosfamide, treosulfan, and busulfan."( In vitro testing of drug combinations employing nilotinib and alkylating agents with regard to pretransplant conditioning treatment of advanced-phase chronic myeloid leukemia.
Dreger, P; Fruehauf, S; Ho, AD; Jens Zeller, W; Luft, T; Radujkovic, A; Topaly, J, 2014
)
0.59
"Treatment of imatinib-sensitive, BCR-ABL-positive K562 and LAMA84 cells with nilotinib in combination with mafosfamide, treosulfan, or busulfan resulted in synergistic (CI < 1), additive (CI ~ 1), and predominantly antagonistic (CI > 1) effects, respectively."( In vitro testing of drug combinations employing nilotinib and alkylating agents with regard to pretransplant conditioning treatment of advanced-phase chronic myeloid leukemia.
Dreger, P; Fruehauf, S; Ho, AD; Jens Zeller, W; Luft, T; Radujkovic, A; Topaly, J, 2014
)
0.61
"Cyclophosphamide (Cy) in combination with busulfan (Bu) or total body irradiation (TBI) is the most commonly used myeloablative conditioning regimen in patients with chronic myeloid leukemia (CML)."( Comparison of outcomes of allogeneic transplantation for chronic myeloid leukemia with cyclophosphamide in combination with intravenous busulfan, oral busulfan, or total body irradiation.
Ahn, KW; Aljurf, MD; Alyea, EP; Arora, M; Avalos, BR; Bolwell, BJ; Bredeson, CN; Cahn, JY; Copelan, EA; Cortes, J; Costa, LJ; Fasan, O; Gale, RP; Ghosh, N; Grunwald, MR; Hale, GA; Hamadani, M; Hamilton, BK; Horowitz, MM; Inamoto, Y; Kalaycio, ME; Kamble, RT; Khoury, HJ; Litzow, MR; Loren, AW; Marks, DI; Maziarz, RT; Ramanathan, M; Saber, W; Savani, BN; Schouten, HC; Szer, J; Ustun, C; van Besien, KW; Waller, EK; Weisdorf, DJ; Wirk, B; Zhu, X, 2015
)
0.88
"This study assessed the potential cytotoxicity of RX against JAK2-positive human cell lines (SET-2 and HEL), either alone or in combination with hydroxyurea, busulphan, rapamycin or LY294002."( Pro-Apoptotic Activity of Ruxolitinib Alone and in Combination with Hydroxyurea, Busulphan, and PI3K/mTOR Inhibitors in JAK2-Positive Human Cell Lines.
Cebula-Obrzut, B; Chojnowski, K; Majchrzak, A; Smolewski, P; Szymańska, J; Treliński, J,
)
0.13
" Fifty-eight patients (median age, 67 years; range, 54 to 76) received radioimmunotherapy followed by fludarabine 150 mg/m(2) and busulfan 8 mg/kg combined with either 75 mg (n = 26) or 50 mg (n = 32) alemtuzumab."( Reduced-Intensity Conditioning Combined with (188)Rhenium Radioimmunotherapy before Allogeneic Hematopoietic Stem Cell Transplantation in Elderly Patients with Acute Myeloid Leukemia: The Role of In Vivo T Cell Depletion.
Bornhäuser, M; Ehninger, G; Kotzerke, J; Schetelig, J; Schneider, S; Strumpf, A; Wunderlich, G, 2015
)
0.62
"To retrospectively analyze the safety and efficacy of busulfan (BU) combined with cyclophosphamide (CY) as the conditioning regimen of autologous hematopoietic stem cell transplantation (auto-HSCT) in patients with multiple myeloma (MM)."( [Busulfan Combined with Cyclophosphamide as the Conditioning Regimen in Patients with Multiple Myeloma Treated by Autolo-gous Hematopoietic Stem Cell Transplantation].
Fu, CC; Gu, B; Jin, S; Li, WY; Wang, PF; Xu, Y; Zhou, HF; Zhou, J, 2015
)
1.58
"We conducted a retrospective analysis to compare outcomes in adult patients with acute lymphoblastic leukemia (ALL) who underwent allogeneic hematopoietic cell transplantation (allo-HCT) with conditioning regimens containing cyclophosphamide (CY) in combination with total body irradiation (TBI), oral busulfan (p."( Comparison of Cyclophosphamide Combined with Total Body Irradiation, Oral Busulfan, or Intravenous Busulfan for Allogeneic Hematopoietic Cell Transplantation in Adults with Acute Lymphoblastic Leukemia.
Atsuta, Y; Doki, N; Fukuda, T; Inoue, Y; Kako, S; Kanamori, H; Kurokawa, M; Mitsuhashi, K; Mizuta, S; Morishima, Y; Nagamura-Inoue, T; Onizuka, M; Ozawa, Y; Shigematsu, A; Takahashi, S; Tanaka, J, 2016
)
0.84
"To investigate the efficacy and safety of haploidentical allo-HSCT in combination of reduced intensity preconditioning combined with cyclophosphamid (CTX)-induced immune tolerance after transplanitation for treatment of severe aplastic anemia (SAA)."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 2016
)
0.43
"A total of 15 patients with SAA received the haploidentical allo-HSCT of reduced intensity preconditioning combined with CTX-induced immune tolerance after transplartation in the General hospital of Beijing military command of chinese PLA from June 2012 to December 2014."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 2016
)
0.43
"The haploidentical allo-HSCT of reduced intensity preconditioning combined with CTX-induced immune tolerance after transplantation is safet and effective for SAA patients, that may be applied to clinical therapy."( [Clinical Efficacy of Haploidentical Allo-HSCT of Reduced Intensity Preconditioning Combined with Induced Immune Tolerance after Transplantation for Severe Aplastic Anemia].
Chen, HR; Chen, P; Guo, Z; He, XP; Liu, XD; Lou, JX; Yang, K; Zhang, Y, 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
" Despite decades of use, mechanisms of Bu PK-based drug-drug interactions (DDIs), as well as the negative downstream effects of these DDIs, have not been fully characterized."( Clarifying busulfan metabolism and drug interactions to support new therapeutic drug monitoring strategies: a comprehensive review.
Andersson, BS; Champlin, RE; Ghose, R; Kawedia, JD; Kramer, MA; Myers, AL; Nieto, Y, 2017
)
0.84
"Lentiviral vector gene therapy combined with low-exposure, targeted busulfan conditioning in infants with newly diagnosed SCID-X1 had low-grade acute toxic effects and resulted in multilineage engraftment of transduced cells, reconstitution of functional T cells and B cells, and normalization of NK-cell counts during a median follow-up of 16 months."( Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Abdelsamed, H; Aldave Becerra, JC; Church, JA; Condori, J; Cowan, MJ; Cross, SJ; da Matta Ain, AC; De Ravin, SS; Dokmeci, E; Dowdy, J; Gottschalk, S; Janssen, W; Kang, G; Li, C; Lockey, T; Long-Boyle, JR; Love, JT; Ma, Z; Malech, HL; Mamcarz, E; Maron, G; Meagher, MM; Puck, JM; Ryu, BY; Sorrentino, BP; Tang, X; Triplett, B; van der Watt, H; Weiss, MJ; Youngblood, B; Zhou, S, 2019
)
1.01
" Additional drugs reported in combination with the Treo-Flu backbone are thiotepa and melphalan."( Treosulfan in combination with fludarabine as part of conditioning treatment prior to allogeneic hematopoietic stem cell transplantation.
Ussowicz, M, 2020
)
0.56
"This study aimed to predict the presence and mechanism of busulfan drug-drug interactions (DDIs) in hematopoietic stem cell transplantation (HSCT) using pharmacokinetic interaction (PKI) network-based molecular structure similarity and network pharmacology."( Predicting the presence and mechanism of busulfan drug-drug interactions in hematopoietic stem cell transplantation using pharmacokinetic interaction network-based molecular structure similarity and network pharmacology.
Hao, C; Hu, J; Huang, J; Ma, X; Wang, L; Yang, W; Zhang, W, 2021
)
1.13
"Logistic function models were established to predict busulfan DDIs based on the assumption that an approved drug tends to interact with the drug used in HSCT (DH) if structurally similar to the drugs in the PKI network of the DH."( Predicting the presence and mechanism of busulfan drug-drug interactions in hematopoietic stem cell transplantation using pharmacokinetic interaction network-based molecular structure similarity and network pharmacology.
Hao, C; Hu, J; Huang, J; Ma, X; Wang, L; Yang, W; Zhang, W, 2021
)
1.14
" In this study, cyclophosphamide combined with busulfan was used to establish an animal model."( Experimental study for the establishment of a chemotherapy-induced ovarian insufficiency model in rats by using cyclophosphamide combined with busulfan.
Feng, X; Ling, L; Luo, Y; Tang, D; Wang, Y; Xiong, Z; Zhang, W, 2021
)
1.08
"The aim of this work is the development of a mechanistic physiologically-based pharmacokinetic (PBPK) model using in vitro to in vivo extrapolation to conduct a drug-drug interaction (DDI) assessment of treosulfan against two cytochrome p450 (CYP) isoenzymes and P-glycoprotein (P-gp) substrates."( Evaluation of the drug-drug interaction potential of treosulfan using a physiologically-based pharmacokinetic modelling approach.
Balazki, P; Baumgart, J; Beelen, DW; Böhm, S; Hemmelmann, C; Hilger, RA; Martins, FS; Ring, A; Schaller, S, 2022
)
0.72
"The study objective was to develop a population pharmacokinetic model for busulfan to comprehensively examine drug-drug interactions in paediatric patients undergoing haematopoietic stem cell transplantation."( Characterization of drug-drug interactions on the pharmacokinetic disposition of busulfan in paediatric patients during haematopoietic stem cell transplantation conditioning.
Dunn, A; Gobburu, JVS; Ivaturi, V; Moffett, BS, 2022
)
1.18
" 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
"Total body irradiation (TBI) at a dose of 12 Gy combined with cyclophosphamide (CyTBI12Gy) is one of the standard myeloablative regimens for patients with acute myeloid leukemia (AML) treated with allogeneic hematopoietic cell transplantation (allo-HCT)."( Fludarabine versus cyclophospamide in combination with myeloablative total body irradiation as conditioning for patients with acute myeloid leukemia treated with allogeneic hematopoietic cell transplantation. A study from the Acute Leukemia Working Party
Arat, M; Bourhis, JH; Cornelissen, JJ; Giebel, S; Grillo, G; Hilgendorf, I; Kröger, N; Labopin, M; Maertens, J; Mohty, M; Nagler, A; Poiré, X; Salmenniemi, U; Savani, B; Schroeder, T; Spyridonidis, A; Swoboda, R, 2023
)
0.91
" The aim of the present study was to assess and evaluate the potential drug-drug interaction (DDI) between Bu and VPA."( Impact of valproic acid on busulfan pharmacokinetics: In vitro assessment of potential drug-drug interaction.
Al-Enezi, BF; Al-Hasawi, N; Matar, KM, 2023
)
1.21
" Combined with the strong rationale of busulfan PK/PD relationships, factors altering its clearance (e."( Individualizing busulfan dose in specific populations and evaluating the risk of pharmacokinetic drug-drug interactions.
Combarel, D; Delahousse, J; Paci, A; Tran, J; Vassal, G, 2023
)
1.53
"This review aims to provide an overview of the current knowledge on busulfan pharmacokinetics, its pharmacokinetics variabilities in pediatric populations, drug-drug interactions (DDI), and their consequences regarding dose individualization."( Individualizing busulfan dose in specific populations and evaluating the risk of pharmacokinetic drug-drug interactions.
Combarel, D; Delahousse, J; Paci, A; Tran, J; Vassal, G, 2023
)
1.49
"In conclusion, ATG and PTCy combined with Flu-based increased intensity conditioning regimen is effective for acute leukemia in children."( Intensified conditioning regimen with fludarabine combined with post-transplantation cyclophosphamide for haploidentical allogeneic hematopoietic stem cell transplantation in children with high-risk acute leukemia.
Cao, J; Chen, D; Li, S; Liu, X; Lu, Y; Pei, R; Wang, T; Xu, X; Ye, P; Zheng, ZZ, 2023
)
0.91

Bioavailability

Busulfan is a common component of pretransplant conditioning but has an erratic and unpredictable bioavailability when administered orally. The busulfan formulation (1% powder form or crystal form) and dose (milligrams per kilogram) influenced the absorption rate constant.

ExcerptReferenceRelevance
" Alterations in bioavailability (absorption or first pass elimination) or in actual volume of distribution may account for these differences in drug disposition."( Busulfan disposition in children.
Blazar, B; Grochow, LB; Krivit, W; Whitley, CB, 1990
)
1.72
" The lag time for the start of absorption, the time absorption ends, and the absorption rate constant showed some interindividual variations."( Busulfan kinetics.
Beran, M; Ehrnebo, M; Ehrsson, H; Hassan, M, 1983
)
1.71
" The absorption rate of busulfan tablets in our model was as unpredictable as documented in clinical trials."( Use of a water-soluble busulfan formulation--pharmacokinetic studies in a canine model.
Blanz, J; Deeg, HJ; Ehninger, G; Ehrsam, M; Renner, U; Schuler, U; Storb, R; Zeller, KP, 1995
)
0.91
" However, recently it was shown that busulfan bioavailability varied by 2-fold in adults (0."( Aspects concerning busulfan pharmacokinetics and bioavailability.
Ehrsson, H; Hassan, M; Ljungman, P, 1996
)
0.9
" Bioavailability of BU powder capsules was on average 70% (range, 44-94%)."( Pharmacokinetics of intravenous busulfan and evaluation of the bioavailability of the oral formulation in conditioning for haematopoietic stem cell transplantation.
Deeg, J; Ehninger, G; Ehrsam, M; Schmidt, H; Schneider, A; Schuler, US, 1998
)
0.58
" An intravenous form of busulphan would overcome the problems caused by inter-individual variability and bioavailability of busulphan and most probably minimize the problems with dose adjustment during therapy."( Liposomal busulphan: bioavailability and effect on bone marrow in mice.
Hassan, M; Hassan, Z; Nilsson, C, 1998
)
0.3
" In a pharmacologic study of the bioavailability of treosulfan in a capsule formulation, patients with relapsed ovarian carcinoma were treated with alternating doses of oral and intravenous (i."( Investigation of bioavailability and pharmacokinetics of treosulfan capsules in patients with relapsed ovarian cancer.
Baumgart, J; Hilger, RA; Jacek, G; Kredtke, S; Oberhoff, C; Scheulen, ME; Seeber, S, 2000
)
0.31
"The high and relatively constant bioavailability of treosulfan indicates that capsules provide a satisfactory noninvasive treatment alternative."( Investigation of bioavailability and pharmacokinetics of treosulfan capsules in patients with relapsed ovarian cancer.
Baumgart, J; Hilger, RA; Jacek, G; Kredtke, S; Oberhoff, C; Scheulen, ME; Seeber, S, 2000
)
0.31
" Busulfan is well absorbed after oral administration, exhibits low protein binding and is metabolised through conjugation with glutathione to form a thiophenium ion."( Plasma concentration monitoring of busulfan: does it improve clinical outcome?
Gibbs, JP; McCune, JS; Slattery, JT, 2000
)
1.49
"The unpredictable intestinal absorption and erratic bioavailability of oral busulfan (Bu) has limited the drug's use in high-dose pretransplantation conditioning therapy."( Acute safety and pharmacokinetics of intravenous busulfan when used with oral busulfan and cyclophosphamide as pretransplantation conditioning therapy: a phase I study.
Andersson, BS; Blume, KG; Champlin, RE; Chow, DS; Hu, WW; Madden, T; Tran, HT; Vaughan, WP, 2000
)
0.79
" Busulfan is a common component of pretransplant conditioning but has an erratic and unpredictable bioavailability when administered orally."( Intravenous busulfan-based conditioning prior to allogeneic hematopoietic stem cell transplantation: myeloablation with reduced toxicity.
Avigdor, A; Ben-Bassat, I; Bielorai, B; Hardan, I; Nagler, A; Shimoni, A; Toren, A; Yeshurun, M, 2003
)
1.61
"008) and clearance corrected by bioavailability (0."( Influence of glutathione S-transferase A1 polymorphism on the pharmacokinetics of busulfan.
Iga, T; Kanda, Y; Kubota, T; Kusama, M; Matsukura, Y; Matsuno, K; Ogawa, S, 2006
)
0.56
" This treatment leads to a wide variability in bioavailability and side effects such as the veino-occlusive disease."( Busulfan loading into poly(alkyl cyanoacrylate) nanoparticles: physico-chemistry and molecular modeling.
Aymes-Chodur, C; Chacun, H; Couvreur, P; Ghermani, NE; Gref, R; Layre, AM; Poupaert, J; Requier, D; Richard, J, 2006
)
1.78
" Busulfan pharmacokinetic parameter estimates (Ka, first order absorption rate constant; Vs, volume of distribution related to the body weight; and Cl/F, apparent clearance) were estimated by using the nonparametric adaptative grid (NPAG) algorithm in patients divided into four groups according to initial diagnosis: metabolic diseases, hemoglobinopathies, hematological malignancies, and immune deficiencies."( Influence of underlying disease on busulfan disposition in pediatric bone marrow transplant recipients: a nonparametric population pharmacokinetic study.
Aulagner, G; Bertholle-Bonnet, V; Bertrand, Y; Bleyzac, N; Galambrun, C; Mialou, V; Souillet, G, 2007
)
1.53
" 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
" The busulfan formulation (1% powder form or crystal form) and dose (milligrams per kilogram) influenced the absorption rate constant."( Population pharmacokinetics of oral busulfan in young Japanese children before hematopoietic stem cell transplantation.
Ariyoshi, N; Kitada, M; Miura, G; Nakamura, H; Nakazawa, K; Okada, K; Sato, T, 2008
)
1.13
" Bypassing the oral route to achieve 100% bioavailability translated into improved control over drug administration, with increased safety and reliability of generating therapeutic Bu levels, maximizing antileukemic efficacy."( Busulfan in hematopoietic stem cell transplantation.
Andersson, BS; Ciurea, SO, 2009
)
1.8
" Total clearances were calculated for all 277 patients with oral Bu and compared to those with IV Bu, with the ratio of IV-to-oral clearance representing the absolute bioavailability of the oral form."( Exposure equivalence between IV (0.8 mg/kg) and oral (1 mg/kg) busulfan in adult patients.
Léger, F; Nguyen, L; Puozzo, C, 2009
)
0.59
" Oral Bu bioavailability was about 80% when calculated from the ratio of IV-to-oral total clearances."( Exposure equivalence between IV (0.8 mg/kg) and oral (1 mg/kg) busulfan in adult patients.
Léger, F; Nguyen, L; Puozzo, C, 2009
)
0.59
"Oral bioavailability (F) is a product of fraction absorbed (Fa), fraction escaping gut-wall elimination (Fg), and fraction escaping hepatic elimination (Fh)."( Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
Chang, G; El-Kattan, A; Miller, HR; Obach, RS; Rotter, C; Steyn, SJ; Troutman, MD; Varma, MV, 2010
)
0.36
" Furthermore, bioavailability was calculated to be between 93% and 99% for the development data set."( Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children.
Bergstrand, M; Boos, J; Hempel, G; Karlsson, MO; Trame, MN, 2011
)
0.66
"Our evaluation, based on clinical profiles and a virtual validation set, revealed: i) NLF sometimes overestimated the absorption rate constant Ka, ii) TZE overestimated AUC over 280% when Ka is small, and iii) MLR underestimated AUC over 30% when the elimination rate constant Ke is small."( Limited sampling strategies to estimate the area under the concentration-time curve. Biases and a proposed more accurate method.
Bax, L; Fukumoto, M; Kohno, A; Morishita, Y; Tsuruta, H, 2012
)
0.38
" Intravenous (IV) Bu has more predictable bioavailability and a safer toxicity profile than the oral formulation."( Allogeneic hematopoietic stem-cell transplantation for acute myeloid leukemia in remission: comparison of intravenous busulfan plus cyclophosphamide (Cy) versus total-body irradiation plus Cy as conditioning regimen--a report from the acute leukemia worki
Aljurf, M; Ben Othman, T; Campos, A; Labopin, M; Masszi, T; Michallet, M; Mohty, M; Nagler, A; Passweg, J; Poire, X; Rocha, V; Sengelov, H; Shimoni, A; Socie, G; Unal, A; Veelken, H; Volin, L; Yakoub-Agha, I, 2013
)
0.6
"3 l h(-1) for the absorption rate constant, volume of distribution and oral clearance, respectively."( Busulfan dosing algorithm and sampling strategy in stem cell transplantation patients.
de Castro, FA; Della Pasqua, O; Lanchote, VL; Piana, C; Simões, BP, 2015
)
1.86
"Parenteral administration of Busulfan (BU) conquers the bioavailability and biovariability related issues of oral BU by maintaining the plasma drug concentration in therapeutic range with minimal fluctuations thereby significantly reducing the side effects."( Development and characterization of an organic solvent free, proliposomal formulation of Busulfan using quality by design approach.
Chobisa, D; Monpara, J; Patel, K; Patel, M; Vavia, P, 2018
)
0.99
" Intravenous Busulfan (Bu) combined with therapeutic drug monitoring-guided dosing has been increasingly used, with more predictable bioavailability and better outcomes comparing to oral Bu."( Pharmacokinetics-adapted Busulfan-based myeloablative conditioning before unrelated umbilical cord blood transplantation for myeloid malignancies in children.
Ansari, M; Benadiba, J; Bittencourt, H; Cellot, S; Duval, M; Krajinovic, M; Teira, P; Vachon, MF, 2018
)
1.15
"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

Dosage Studied

The maintenance of certain busulfan plasma concentration during its whole administration with the help of the repeated adjustments of its dosage can reduce the toxicity caused by this agent. The authors aimed to develop a population PK model for busulfans in children that can reliably achieve therapeutic exposure.

ExcerptRelevanceReference
" However, even when BU is given at a dosage that does not significantly affect the HS-P, CFU-S recovery is delayed, suggesting that BU affects the CFU-S in a manner that differs qualitatively from that of CY."( Effects of cyclophosphamide and of busulfan on spleen colony-forming units and on hematopoietic stroma.
Barone, J; Fried, W; Kedo, A, 1977
)
0.53
"We investigated the effect of two variables, the time interval between drug administration and transplant removal and the dosage of the drug, on the prolongation of rat heart allograft survival via donor pretreatment."( Prolongation of rat cardiac allograft survival by donor pretreatment. Impact of dose and timing of drug administration.
Häyry, P; Soots, A, 1979
)
0.26
"Male Wistar Strain rats of known fertility were given subcutaneous dosage of three drugs, alpha-chlorohydrin, amino-alpha-chlorohydrin, and busulphan alone or in combination for a period of thirty days."( Agonistic properties of low doses of antifertility compounds in male rats.
Hafez, ES; Shandilya, LN; Snydle, FE, 1979
)
0.26
" Radiographic appearances consistent with busulphan lung were not reported in any of the 195 B patients (receiving a mean dosage of 464 mg of busulphan over 301 days) or of the 192 C patients but were present in one of the 201 patients receiving placebo."( An investigation of the chest radiographs in a controlled trial of busulphan, cyclophosphamide, and a placebo after resection for carcinoma of the lung.
Fox, W; Roy, DC; Simon, G; Stephens, R; Stott, H, 1976
)
0.26
" The diffusion chamber dose-response curves were compared to the progenitor cell survivals estimated by the spleen colony technique."( The effect of various cytotoxic agents on bone marrow progenitor cells as measured by diffusion chamber assays.
Lamerton, LF; Squires, DJ, 1975
)
0.25
" The 30-day survival was 87-100% in mice transplanted after 10-40 mg/kg busulfan and 79% after TBI, but fell to 54% and 33%, respectively, after 80 mg/kg and 100 mg/kg busulfan, suggesting that this latter dosage range represents the LD50 for single-dose busulfan in young C57BL/6 mice given stem cell rescue."( Growth retardation and depigmentation of hair after high-dose busulfan and congenic hematopoietic cell transplantation in mice.
Farmer, ER; Shinn, C; Wingard, JR; Yeager, AM; Yeager, MJ, 1992
)
0.76
" To optimize BU dosage in young BMT recipients, we developed a dosage regimen based on body surface area (BSA) and determined BU pharmacokinetics and BU-associated toxicities."( Optimization of busulfan dosage in children undergoing bone marrow transplantation: a pharmacokinetic study of dose escalation.
Graham, ML; Grochow, LB; Jones, RJ; Santos, GW; Wagner, JE; Yeager, AM, 1992
)
0.63
"Recent studies have reported that the pharmacokinetics of high-dose busulfan in bone marrow transplantation (BMT) are age-dependent: with the usual dosage of 16 mg/kg over 4 days, systemic exposure is two to four times lower in children than in adults."( Is 600 mg/m2 the appropriate dosage of busulfan in children undergoing bone marrow transplantation?
Challine, D; Deroussent, A; Gouyette, A; Hartmann, O; Koscielny, S; Lemerle, J; Valteau-Couanet, D; Vassal, G, 1992
)
0.79
" The frequency, time to onset, and duration of GVHD were similar among the three CSP dosage groups."( Induction of cutaneous graft-versus-host disease by administration of cyclosporine to patients undergoing autologous bone marrow transplantation for acute myeloid leukemia.
Altomonte, V; Farmer, ER; Hess, AD; Jones, RJ; Santos, GW; Vogelsang, GB; Yeager, AM, 1992
)
0.28
" The results of analysis of sister chromatid exchange (SCE) showed no irreversible damage on chromosomal DNA of the patients at the dosage used (0."( [Further study on the treatment of severe beta-thalassemia with myleran].
Liang, Z, 1991
)
0.28
" We conclude that the busulphan dosage for children must be reconsidered and that further studies are urgently needed to develop an optimal therapy."( Pharmacokinetics of high-dose busulphan in relation to age and chronopharmacology.
Aschan, J; Bekassy, AN; Ehrsson, H; Hassan, M; Ljungman, P; Lönnerholm, G; Oberg, G; Smedmyr, B; Taube, A; Wallin, I, 1991
)
0.28
" The curves of globin chain synthesis ratios were found to be related to dosage and the duration of myleran treatment."( [Influence of myleran on globin chain synthesis ratio in anemic rhesus monkeys].
Ao, ZH, 1989
)
0.28
" G-CSF was given at variable dosage based on neutrophil count."( Granulocyte colony-stimulating factor and neutrophil recovery after high-dose chemotherapy and autologous bone marrow transplantation.
Dodds, A; Fox, RM; Green, MD; Layton, JE; Lusk, J; Maher, D; Morstyn, G; Sheridan, WP; Souza, L; Wolf, M, 1989
)
0.28
"/100 g body weight) in intact rats and those in Group B, a shift in the dose-response curve to the right was observed in the latter, suggesting that the sensitivity of testicular response to gonadotrophin was lower in germ cell-depleted rats."( Significance of germ cells for the expression of testicular endocrine function in rats.
Kasuga, F; Shiota, K; Takahashi, M, 1989
)
0.28
"The technique of high-dose chemotherapy and bone-marrow transplantation takes advantage of any potential dose-response effect in the treatment of cancer and the ability of infused marrow to circumvent severe myelotoxicity."( High-dose chemotherapy with busulphan and cyclophosphamide and bone-marrow transplantation for drug-sensitive malignancies in adults: a preliminary report.
Boyd, AW; Brodie, GN; Green, MD; Griffiths, JD; McGrath, KM; Russell, DM; Scarlett, JD; Sheridan, WP; Thomas, RJ; Vaughan, SL, 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
)
0.27
" Our standard dose of cyclophosphamide was 2 g/m2 daily for 4 days, but this dosage was reduced if necessary so as not to exceed 75 mg/kg/day."( Busulphan and cyclophosphamide cause little early toxicity during displacement bone marrow transplantation in fifty children.
Barnes, R; Downie, CJ; Hobbs, JR; Hugh-Jones, K; Shaw, PJ, 1986
)
0.27
"It is concluded that there is no evidence that either of the two cytotoxic drugs in the dosage prescribed improved survival for the two-year period of observation, though a final evaluation of the adjuvant chemotherapy as studied in this investigation will have to await the results at five years."( Study of cytotoxic chemotherapy as an adjuvant to surgery in carcinoma of the bronchus. Report by a Medical Research Council Working Party.
, 1971
)
0.25
" High dose animals had greater initial responses than low dosed dogs but long term responses were not significantly different."( Autologous bone marrow infusion following high dose chemotherapy of the canine transmissible venereal tumor (TVT).
Epstein, RB; Sarpel, SC, 1980
)
0.26
" The dose-response relationships between BUS and SCEs in vitro were found to be similar for bone marrow and lymphocytes."( Spontaneous and busulphan-induced sister-chromatid exchange (SCE) frequencies in haematologically normal human bone marrow and lymphocytes.
Honeycombe, JR, 1981
)
0.26
" The probability of developing acute leukemia in this study was not significantly correlated to the total cumulative dosage of Treosulfan."( Acute non-lymphocytic leukemia in patients with ovarian carcinoma following long-term treatment with Treosulfan (= dihydroxybusulfan).
Ernst, P; Ersbøl, J; Hou-Jensen, K; Knudtzon, S; Larsen, MS; Nissen, NI; Pedersen-Bjergaard, J; Rose, C; Sørensen, HM, 1980
)
0.47
"0448) than did those who received octreotide at the 150 micrograms dosage level."( Randomized trial of loperamide versus dose escalation of octreotide acetate for chemotherapy-induced diarrhea in bone marrow transplant and leukemia patients.
Davidson, TG; Dix, SP; Geller, RB; Gilmore, CE; Holland, HK; Lin, LS; Topping, DL; Wingard, JR, 1995
)
0.29
" Busulfan was quantitated in plasma samples at 10 time points within the 6 h dosing interval using HPLC before and after dose numbers 1, 2, 5, 13 and 14."( Busulfan pharmacokinetics in bone marrow transplant patients: is drug monitoring warranted?
Blanz, J; Ehninger, G; Kühnle, A; Kumbier, I; Mewes, K; Proksch, B; Schroer, S; Schuler, U; Zeller, KP, 1994
)
2.64
" The difference in clearance between children and adults was not statistically significant when normalized to body surface area, which most probably shows that busulfan dosage should be calculated on the basis of surface area rather than body weight."( Busulfan bioavailability.
Békàssy, A; Bolme, P; Hassan, M; Kållberg, N; Ljungman, P; Ringdén, O; Starý, J; Syrůcková, Z; Wallin, I, 1994
)
1.93
" The pharmacokinetics of busulfan was studied during the 6-hour dosing interval on the third day of therapy by use of a high-performance liquid chromatographic assay."( Disposition of high-dose busulfan in pediatric patients undergoing bone marrow transplantation.
Bonetti, F; Buggia, I; Locatelli, F; Pregnolato, M; Quaglini, S; Regazzi, MB; Zecca, M, 1993
)
0.89
" The optimal dosage of busulfan needed to achieve bone marrow (BM) displacement in young children with malignant or nonmalignant disease remains to be defined."( Busulfan disposition below the age of three: alteration in children with lysosomal storage disease.
Boland, I; Challine, D; Fischer, A; Gluckman, E; Ledheist, F; Lemerle, S; Rahimy, C; Souillet, G; Vassal, G; Vilmer, E, 1993
)
2.04
" These patients received a total of 16 doses of busulfan dosed as 1 mg/kg/dose q 6 h beginning at 09."( Association of busulfan area under the curve with veno-occlusive disease following BMT.
Davidson, TG; Devine, SM; Dix, SP; Geller, RB; Gilmore, CE; Heffner, LT; Hillyer, CD; Holland, HK; Jerkunica, I; Lin, LS; Mullins, RE; Saral, R; Wingard, JR; Winton, EF; York, RC, 1996
)
0.9
" Thus, targeting avoided much of the variability in BU Css seen in other studies and appears to have allowed for an increase in oral dosing from 8 mg/kg to 10."( Busulfan, cyclophosphamide and fractionated total body irradiation for autologous or syngeneic marrow transplantation for acute and chronic myelogenous leukemia: phase I dose escalation of busulfan based on targeted plasma levels.
Appelbaum, FR; Bensinger, WI; Buckner, CD; Clift, R; Demirer, T; Fefer, A; Lambert, K; Sanders, J; Slattery, JT; Storb, R, 1996
)
1.74
" Several investigators have expressed their concern about the dosage in children and many suggested higher doses based on the body surface area for young children."( Aspects concerning busulfan pharmacokinetics and bioavailability.
Ehrsson, H; Hassan, M; Ljungman, P, 1996
)
0.62
" The present model is reliable and adequate for studying more patients, with a long-term follow-up combined with drug monitoring in correlation with drug efficacy and toxicity to define the optimal busulphan dosage required."( Busulphan kinetics and limited sampling model in children with leukemia and inherited disorders.
Fasth, A; Gerritsen, B; Haraldsson, A; Hassan, M; Karlsson, M; Kumlien, S; Sandström, M; Syrůcková, Z; van den Berg, H; Vossen, J, 1996
)
0.29
" The superiority of a therapeutic regimen in chronic phase CML seems to primarily depend on whether its pharmacology permits a sufficiently high dosage to achieve the necessary reduction of tumor burden."( Current aspects of drug therapy in Philadelphia-positive CML: correlation of tumor burden with survival.
Hehlmann, R; Heimpel, H, 1996
)
0.29
"Etoposide is one of the few drugs being used in conditioning regimens because of the ease with which its dosage can be escalated by a factor of 6 compared to the normal dose."( The pharmacokinetics and toxicity of two application schedules with high-dose VP-16 in patients receiving an allogeneic bone marrow transplantation.
Bewermeier, P; Hossfeld, DK; Kruger, W; Mross, K; Reifke, J; Zander, A, 1996
)
0.29
" Plasma concentrations of BU at steady state (C(SS)BU) during the dosing interval were measured for each patient."( Marrow transplantation for chronic myeloid leukemia: the influence of plasma busulfan levels on the outcome of transplantation.
Anasetti, C; Appelbaum, FR; Bensinger, WI; Bowden, R; Bryant, E; Buckner, CD; Chauncey, T; Clift, RA; Deeg, HJ; Doney, KC; Flowers, M; Gooley, T; Hansen, JA; Martin, PJ; McDonald, GB; Nash, R; Petersdorf, EW; Radich, J; Sanders, JE; Schoch, G; Slattery, JT; Soll, E; Stewart, P; Storb, R; Storer, B; Sullivan, KM; Thomas, ED; Witherspoon, RP, 1997
)
0.53
"Fifty-two BMT institutions were sent a questionnaire asking for details of dosing for busulphan, cyclophosphamide (bu-cy), cyclosporin and methotrexate (CSA-MTX)."( Variability in determination of body weight used for dosing busulphan and cyclophosphamide in adult patients: results of an international survey.
Grigg, A; Harun, MH; Szer, J, 1997
)
0.3
"Busulphan (BU) pharmacokinetic (PK) studies in children undergoing bone marrow transplantation suggest that individual BU dosing may be necessary to optimise BU systemic exposure."( An improved limited sampling method for individualised busulphan dosing in bone marrow transplantation in children.
Calderwood, S; Chattergoon, DS; Doyle, J; Freedman, MH; Klein, J; Koren, G; Saunders, EF, 1997
)
0.3
" Routine dosing on the basis of BSA or AIBW in adults and adolescents does not require a specific accommodation for the obese."( The impact of obesity and disease on busulfan oral clearance in adults.
Appelbaum, FR; Corneau, B; Gibbs, JP; Gooley, T; Murray, G; Slattery, JT; Stewart, P, 1999
)
0.58
" However, factors other than absorption influence the AUC, and individualization of dosing may be required even with intravenous administration of the drug."( Myeloablation by intravenous busulfan and hematopoietic reconstitution with autologous marrow in a canine model.
Deeg, HJ; Ehninger, G; Ehrsam, M; Renner, U; Schuler, US; Shulman, H; Storb, R; Yu, C, 1999
)
0.59
" Busulfan plasma levels were measured by gas chromatography-mass spectrometry after the first daily dose of the 4-day dosing regimen."( Up-regulation of glutathione S-transferase activity in enterocytes of young children.
Baldassano, RN; Gibbs, JP; Liacouras, CA; Slattery, JT, 1999
)
1.21
" Although busulfan has only limited activity against solid tumors, the high doses achievable in the CSF following intrathecal administration coupled with the steep dose-response relationships of alkylating agents, provide rationale for further evaluation of this agent."( Intrathecal busulfan treatment of human neoplastic meningitis in athymic nude rats.
Archer, GE; Bigner, DD; Colvin, OM; Friedman, AH; Friedman, HS; Fuchs, HE; McCullough, W; McLendon, RE; Rose, M; Sampson, JH; Sands, H, 1999
)
1.08
"High dosage busulfan (1 mg/kg orally every 6 hours x 16 doses) is frequently used in preparative regimens for haemopoietic stem cell transplantation (HSCT)."( Plasma concentration monitoring of busulfan: does it improve clinical outcome?
Gibbs, JP; McCune, JS; Slattery, JT, 2000
)
0.96
"We investigated whether adjusting the oral busulfan (BU) dosage on the basis of early pharmacokinetic data to achieve a targeted drug exposure could reduce transplant-related complications in children with advanced hematologic malignancies."( Individualizing high-dose oral busulfan: prospective dose adjustment in a pediatric population undergoing allogeneic stem cell transplantation for advanced hematologic malignancies.
Chan, KW; Choroszy, M; Danielson, M; Felix, EA; Madden, T; Petropoulos, D; Przepiorka, D; Sprigg-Saenz, HA; Tran, HT; Worth, LL, 2000
)
0.86
" The aim of this study was to determine the appropriate dosing of etoposide in combination with busulfan and cyclophosphamide in this setting."( Hematopoietic stem cell transplantation (HSCT) with a conditioning regimen of busulfan, cyclophosphamide, and etoposide for children with acute myelogenous leukemia (AML): a phase I study of the Pediatric Blood and Marrow Transplant Consortium.
Coppes, MJ; Gamis, A; Hagg, R; Kamani, N; Mustafa, MM; Sandler, ES; Wall, D, 2000
)
0.75
" Pharmacokinetic dosing of BU may be important for prevention of NRM but does not appear to influence the risk of relapse in this largely pediatric population with AML."( Busulfan pharmacokinetics do not predict relapse in acute myeloid leukemia.
Baker, KS; Blazar, BR; Bostrom, B; DeFor, T; Ramsay, NK; Woods, WG, 2000
)
1.75
" These data suggest that dose adjustment based on first dose PK data could allow uniformity of busulfan dosing for patients receiving SCT."( A phase I/II study of multiple-dose intravenous busulfan as myeloablation prior to stem cell transplantation.
Apperley, J; Craddock, C; Eades, A; Goldman, J; Hassan, M; Kanfer, E; Matthews, J; Nilsson, C; Olavarria, E; Timms, A, 2000
)
0.78
" Prospective studies of pharmacokinetically guided versus surface area-based administration should be performed to validate pharmacokinetic-pharmacodynamic relationships and to facilitate optimal dosage of anticancer agents in the clinic."( Pharmacokinetically guided administration of chemotherapeutic agents.
Beijnen, JH; Mathôt, RA; Schellens, JH; van den Bongard, HJ, 2000
)
0.31
"Chronic low-dose busulfan therapy results in lung injury in 4-6% of patients after several years of treatment and once the cumulative dosage begins to approach 3g."( Idiopathic pneumonia syndrome following myeloablative chemotherapy and autologous transplantation.
Bilgrami, SF; Bona, RD; Clive, JM; Edwards, RL; Feingold, JM; Kapur, D; McNally, D; Metersky, ML; Naqvi, BH; Raible, D; Tutschka, PJ, 2001
)
0.65
" Pharmacokinetic analysis of plasma busulfan levels alerted staff to the dosing error."( Accidental busulfan overdose: enhanced drug clearance with hemodialysis in a child with Wiskott-Aldrich syndrome.
Ben-Ari, J; Bentur, Y; Davidovitz, M; Gamzu, Z; Hoffer, E; Krivoy, N; Stein, J; Tabak, A; Yaniv, I, 2001
)
0.98
" Anticipatory changes in hydroxyurea dosage or the maintenance of a constant dose did not abolish periodicity, but a change in therapy to the non-cycle-specific drug anagrelide dampened and abolished the cycling."( Hydroxyurea and periodicity in myeloproliferative disease.
Bennett, M; Grunwald, AJ, 2001
)
0.31
"The aims of the present study were (1) to investigate and quantify the pharmacokinetics, including inter-occasion variability and covariate relationships, of busulphan in BMT patients and (2) to develop a user-friendly initial dosing and therapeutic drug monitoring (TDM) strategy for the treatment of those patients with busulphan."( Population pharmacokinetic analysis resulting in a tool for dose individualization of busulphan in bone marrow transplantation recipients.
Bekassy, A; Hassan, M; Hassan, Z; Jonsson, EN; Karlsson, MO; Ljungman, P; Nilsson, C; Oberg, G; Ringden, O; Sandström, M, 2001
)
0.31
"Optimisation of busulfan dosage in patients undergoing bone marrow transplantation is recommended in order to reduce toxic effects associated with high drug exposure."( Quantification of busulfan in plasma by liquid chromatography-ion spray mass spectrometry. Application to pharmacokinetic studies in children.
Aigrain, EJ; Duval, M; Litalien, C; Quernin, MH; Vilmer, E, 2001
)
0.99
" These results indicate that targeted busulfan dosing optimizes allogeneic engraftment in children."( Target dose adjustment of busulfan in pediatric patients undergoing bone marrow transplantation.
Bolinger, AM; Cowan, MJ; Glidden, DV; Norstad, D; Risler, LJ; Slattery, JT; Sultan, DH; Zangwill, AB, 2001
)
0.88
"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
)
0.84
" The maintenance of certain busulfan plasma concentration during its whole administration with the help of the repeated adjustments of its dosage can reduce the toxicity caused by this agent and improve the results of bone marrow or peripheral blood stem cell transplantations."( [High-dose busulfan--monitoring plasma levels and dosage adjustments in adults].
Kalous, O; Malásková, L; Mayer, J, 2001
)
0.99
" Plasma determinations were performed after the first dosing at 0, 15, 30, 60, 90, 120, 180, 240, 300, and 360 min."( Therapeutic monitoring of busulfan in pediatric bone marrow transplantation.
Arush, MW; Elhasid, R; Hoffer, E; Krivoy, N; Rowe, JM; Stein, J; Tabak, A; Yaniv, I,
)
0.43
" To our knowledge, this is one of the few cases of ROC in which partial remissions using conventionally dosed chemotherapy were achieved repeatedly despite a unfavorable relapse-free interval after high-dose chemotherapy for primary disease."( Individualized long-term chemotherapy for recurrent ovarian cancer after failing high-dose treatment.
Breidenbach, M; Kurbacher, CM; Mallmann, P; Rein, DT, 2002
)
0.31
" form of busulfan (Bu) has prompted investigation of administration schedules other than the 4-times-daily dosage commonly used with oral Bu."( Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Andersson, BS; Brown, C; Chaudhry, A; Duggan, P; Glick, S; Gyonyor, E; Morris, D; Quinlan, D; Ruether, JD; Russell, JA; Stewart, D; Tran, HT, 2002
)
1.04
" 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
" Responders were treated at all three decitabine dose levels, and no dose-response correlation was observed."( Long-term follow-up of a phase I study of high-dose decitabine, busulfan, and cyclophosphamide plus allogeneic transplantation for the treatment of patients with leukemias.
Andersson, B; Champlin, R; Couriel, D; de Lima, M; Donato, M; Gajewski, J; Giralt, S; Kantarjian, H; Khouri, I; Ravandi, F; Shahjahan, M; van Besien, K, 2003
)
0.56
" Recent data revealed immunosuppressive characteristics and substantial haematopoietic stem cell toxicity after repeated dosing of mice."( Antileukaemic activity of treosulfan in xenografted human acute lymphoblastic leukaemias (ALL).
Baumgart, J; Becker, M; Fichtner, I, 2003
)
0.32
" The dosing varied between 13 and 20 mg/kg with seven patients receiving a dose of 600 mg/m(2)."( Population pharmacokinetics of oral busulfan in children.
Boos, J; Ehninger, G; Gruhn, B; Hempel, G; Jenke, A; Klingebiel, T; Mürdter, TE; Ritter, CA; Schiltmeyer, B; Schwab, M; Würthwein, G, 2003
)
0.59
" Variability in one patient during the 4 days of treatment (interoccasion variability, IOV) for Cl/F (10%) and V/F (19%) were calculated to be less than interindividual variability, fulfilling the condition for individualization of busulfan dosage regimens."( Population pharmacokinetics of oral busulfan in children.
Boos, J; Ehninger, G; Gruhn, B; Hempel, G; Jenke, A; Klingebiel, T; Mürdter, TE; Ritter, CA; Schiltmeyer, B; Schwab, M; Würthwein, G, 2003
)
0.78
" However, because the steepness of the dose-response curves indicates that direct administration of busulfan is not ideal for this purpose, 15 mg busulfan kg(-1) was administered to pregnant females at various times between day 10."( Dose-response of RAG2-/-/gammac-/- mice to busulfan in preparation for spermatogonial transplantation.
Betteridge, KJ; Foster, RA; Hahnel, AC; Moisan, AE, 2003
)
0.8
" Adverse effects of IFN were similar in both age groups, but IFN dosage to achieve treatment goals was lower in older patients."( Chronic myeloid leukemia in the elderly: long-term results from randomized trials with interferon alpha.
Berger, U; Engelich, G; Gnad, U; Hasford, J; Hehlmann, R; Heimpel, H; Heinze, B; Hochhaus, A; Hossfeld, DK; Kolb, HJ; Löffler, H; Maywald, O; Metzgeroth, G; Pfirrmann, M; Pralle, H; Queisser, W; Reiter, A, 2003
)
0.32
" Previous work has suggested a cumulative dosage of 16 mg/kg for haematopoietic transplantation in children less than 3 years of age, but only limited data are available in infants."( Intravenous busulfan for allogeneic hematopoietic stem cell transplantation in infants: clinical and pharmacokinetic results.
Champagne, MA; Dalle, JH; Duval, M; Gardiner, J; Larocque, D; Shaw, L; Taylor, C; Theoret, Y; Vachon, MF; Wall, D, 2003
)
0.7
"We studied the pharmacokinetics of intravenous busulfan (Bu) in children in order to further optimize intravenous Bu dosing in relation to toxicity and survival."( Intravenous busulfan in children prior to stem cell transplantation: study of pharmacokinetics in association with early clinical outcome and toxicity.
Ball, LM; Bredius, RG; Cremers, SC; den Hartigh, J; Egeler, RM; Lankester, AC; Teepe-Twiss, IM; Vossen, JM; Zwaveling, J, 2005
)
0.96
" The BU dosing began at 15 mg/kg, escalating in 1 mg/kg increments in groups of 4 patients."( Dose escalation of busulfan with pentoxifylline and ciprofloxacin in patients with breast cancer undergoing autologous transplants.
Bensinger, WI; Buckner, CD; Holmberg, L; Lilleby, K; Slattery, JT; Storb, R, 2004
)
0.65
"5 min), and the whole procedure can be completed in 4-5 h, which would permit dose corrections after the third dose allowing earlier and better dosing adjustments towards the target level of busulfan."( Development of a rapid and specific assay for detection of busulfan in human plasma by high-performance liquid chromatography/electrospray ionization tandem mass spectrometry.
Azevedo, Dde A; dos Reis, EO; Lima, EL; Suarez-Kurtz, G; Vianna-Jorge, R, 2005
)
0.76
" Significant interpatient and intrapatient variations in pharmacokinetics require individualizing the dosage based on area under the time-versus-concentration curve."( Tandem mass spectrometry method for the quantification of serum busulfan.
Kellogg, MD; Law, T; Rifai, N; Sakamoto, M, 2005
)
0.57
"Busulfan pharmacokinetics and toxicity were monitored in young rhesus macaques at two dosing levels (4 and 6 mg/kg)."( Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model.
Donahue, RE; Hsieh, MM; Kang, EM; Krouse, A; Metzger, M; Sadelain, M; Tisdale, JF, 2006
)
3.22
"Busulfan pharmacokinetic studies suggest that an individual dosing strategy may be necessary to optimise systemic exposure in order to decrease toxicity and improve outcome in haematopoietic stem cell transplantation."( Quantification of busulfan in saliva and plasma in haematopoietic stem cell transplantation in children : validation of liquid chromatography tandem mass spectrometry method.
Gröschl, M; Holter, W; Kuhlen, M; Rascher, W; Rauh, M; Stachel, D, 2006
)
2.11
"To provide clinical insights into dosing and administration of IV busulfan, a conditioning agent for hematopoietic stem cell transplantation (HSCT)."( Pretransplant conditioning in adults and children: dose assurance with intravenous busulfan.
Barnes, YJ; Fisher, VL; Nuss, SL, 2006
)
0.8
"An understanding of the pharmacokinetic principles underlying the relationship between the therapeutic window for busulfan and optimal HSCT outcomes will facilitate proper dosing and administration of IV busulfan."( Pretransplant conditioning in adults and children: dose assurance with intravenous busulfan.
Barnes, YJ; Fisher, VL; Nuss, SL, 2006
)
0.77
" We utilized an infant rhesus monkey model to identify an optimal dosage of busulfan that results in efficient long-term gene marking with minimal toxicities."( Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector.
Burns, TS; Choi, C; Dorey, FJ; Fisher, J; Fletcher, MD; Jimenez, DF; Kahl, CA; Kohn, DB; Leapley, AC; Lee, CI; Pepper, K; Petersen, D; Tarantal, AF; Ultsch, MN, 2006
)
0.95
"Increasing dosages of busulfan resulted in increased area-under-the-curve (AUC) with some variability at each dosage level, suggesting interindividual variation in clearance."( Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector.
Burns, TS; Choi, C; Dorey, FJ; Fisher, J; Fletcher, MD; Jimenez, DF; Kahl, CA; Kohn, DB; Leapley, AC; Lee, CI; Pepper, K; Petersen, D; Tarantal, AF; Ultsch, MN, 2006
)
1.04
"Mean BU exposure (AUCss) (+/-DS) before dosage modification range from 3192 to 12 180 ng h/mL."( Absence of veno-occlussive disease in a cohort of multiple myeloma patients undergoing autologous stem cell transplantation with targeted busulfan dosage.
Broto, A; Brunet, S; Clopés, A; Farré, R; Mangues, MA; Martino, R; Moreno, E; Queraltó, JM; Sierra, J; Sureda, A, 2006
)
0.54
" Individualising BU dosage in MM patients undergoing ASCT we observed the absence of VOD."( Absence of veno-occlussive disease in a cohort of multiple myeloma patients undergoing autologous stem cell transplantation with targeted busulfan dosage.
Broto, A; Brunet, S; Clopés, A; Farré, R; Mangues, MA; Martino, R; Moreno, E; Queraltó, JM; Sierra, J; Sureda, A, 2006
)
0.54
" After April 1999, 31 patients similar in all pretransplantation risk assessments received the same regimen except that intravenous (IV) Bu was substituted for PO Bu and pharmacokinetic-directed (PKD) dosing was attempted to achieve an area under the concentration time curve of 1000-1500 micromol/min for each dose."( Improved outcomes in intermediate- and high-risk aggressive non-Hodgkin lymphoma after autologous hematopoietic stem cell transplantation substituting intravenous for oral busulfan in a busulfan, cyclophosphamide, and etoposide preparative regimen.
Aggarwal, C; Carabasi, MH; Gupta, S; Katz, RO; Nance, AG; Salzman, DE; Saylors, GB; Tilden, AB; Vaughan, WP, 2006
)
0.53
"We studied the pharmacokinetics and clinical outcome of a new once-daily intravenous area under the curve-targeted dosing scheme for busulfan based on body surface area."( Once-daily intravenous busulfan in children prior to stem cell transplantation: study of pharmacokinetics and early clinical outcomes.
Bredius, RG; den Hartigh, J; Egeler, RM; Guchelaar, HJ; Lankester, AC; Maarten Bredius, RG; Zwaveling, J, 2006
)
0.85
" Non-linear modulating influence was registered even at low dosage of cytostatics which actually could not affect leukemic cell viability."( [Modulating effect of antileukemia drugs on the cellular susceptibility in chronic myeloid leukemia to cytotoxic lymphocytes].
Antonenko, EV; Cherepovich, VS; Grinëv, VV; Lotkova, ES; Shakhlevich, EV, 2006
)
0.33
"The objective of this study was to characterize the pharmacokinetics (PK) of intravenous busulfan in pediatric patients and provide dosing recommendations."( Population pharmacokinetic-based dosing of intravenous busulfan in pediatric patients.
Booth, BP; Dagher, R; Fuller, D; Gobburu, JV; Griebel, D; Lennon, S; Mehta, M; Rahman, A; Sahajwalla, C, 2007
)
0.81
"5-fold difference in dosing rate, (3) negligible variability in dose-to-dose pharmacokinetics and negligible interdose accumulation with once-daily administration, and (4) no change in pharmokinetic parameter(s) with concomitant use of imidazole antifungals, oral contraceptives, or phenytoin."( Pharmacokinetics of once-daily IV busulfan as part of pretransplantation preparative regimens: a comparison with an every 6-hour dosing schedule.
Andersson, BS; Champlin, RE; Couriel, D; de Lima, M; Jones, RB; Madden, T; Nguyen, J; Pierre, B; Roberson, S; Shpall, EJ; Thapar, N, 2007
)
0.62
" Additional BU dosing owing to nausea and/or vomiting occurred in 28 patients (18%) and five patients (3%) were hospitalized."( Home administration of high-dose oral busulfan in patients undergoing hematopoietic stem cell transplantation.
Connaghan, DG; Emami, M; Holland, HK; Matthews, RH; Morris, LE, 2007
)
0.61
" 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
)
0.62
"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
)
0.9
" 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
)
0.62
"In order to assess the performance of Bayesian individualization of busulfan (BU) dosage regimens, veno-occlusive disease (VOD) rate was monitored for paediatric patients undergoing allogeneic bone marrow transplantation (BMT)."( Risk-adjusted monitoring of veno-occlusive disease following Bayesian individualization of busulfan dosage for bone marrow transplantation in paediatrics.
Brice, K; Claire, G; Gilles, A; Nathalie, B; Valerie, B; Valerie, M; Yves, B, 2008
)
0.8
" 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
" Monte Carlo simulations were then performed to assess busulfan dosing regimens based on GSTA1 genotypes."( Glutathione S-transferase A1 genetic variants reduce busulfan clearance in children undergoing hematopoietic cell transplantation.
Baker, KS; Brundage, R; Cao, Q; Farag-El Maasah, S; Jacobson, PA; Johnson, L; Langer, E; Orchard, PJ; Remmel, R; Ross, JA; Wang, X, 2008
)
0.84
" Measurement of individual plasma busulfan levels during oral or intravenous dosing to obtain an AUC is likely to provide the necessary elements to monitor the drug disposition, ensuring efficacy and preventing toxicity of patients undergoing HSCT."( Busulfan use in hematopoietic stem cell transplantation: pharmacology, dose adjustment, safety and efficacy in adults and children.
Bentur, Y; Hoffer, E; Krivoy, N; Lurie, Y; Rowe, JM, 2008
)
2.07
" However there is still at least a threefold variation in exposures achieved by the same dose of IV Bu in different individuals and a small proportion of patients will experience toxic exposures with current dosing regimens."( Therapeutic drug monitoring of busulfan in transplantation.
Kangarloo, SB; Russell, JA, 2008
)
0.63
" For an optimal area under the curve (AUC) targeting, a new Bu dosing regimen [i."( Integration of modelling and simulation into the development of intravenous busulfan in paediatrics: an industrial experience.
Nguyen, L, 2008
)
0.58
" 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
)
0.9
" 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
" IV Bu dosing was mostly based on the ideal body weight index while actual body weight or adjusted ideal body weight indexes were mostly used for oral."( Exposure equivalence between IV (0.8 mg/kg) and oral (1 mg/kg) busulfan in adult patients.
Léger, F; Nguyen, L; Puozzo, C, 2009
)
0.59
" An intravenous dosing of busulfan based on body weight was used."( [Efficacy and toxicity of intravenous busulfan-based conditioning treatment before hAematopoietic stem cell transplantation in children: preliminary report].
Debski, R; Krenska, A; Styczyński, J; Wysocki, M,
)
0.7
" All patients had individualized dosing based on pharmacokinetics after the first dose of intravenous BU."( Influence of GST gene polymorphisms on busulfan pharmacokinetics in children.
Ansari, M; Champagne, MA; Duval, M; Krajinovic, M; Lauzon-Joset, JF; Théoret, Y; Vachon, MF, 2010
)
0.63
" busulfan, variability in the systemic concentrations of busulfan after weight-based dosing and the association between busulfan plasma exposure and outcome in HCT patients have led to the continued use of therapeutic drug monitoring of busulfan."( Busulfan in hematopoietic stem cell transplant setting.
Holmberg, LA; McCune, JS, 2009
)
2.71
" PK of the first dose IVBu was determined to calculate a single dosage adjustment, and with the 9th and 13th doses to confirm steady-state PK."( Safety, efficacy, and pharmacokinetics of intravenous busulfan in children undergoing allogeneic hematopoietic stem cell transplantation.
Chan, KW; Hayashi, RJ; Kadota, R; Kletzel, M; Mezzi, K; Nieder, ML; Przepiorka, D; Wall, DA; Yeager, AM, 2010
)
0.61
"The targeted AUC was achieved with the first dose in 17/24 (71%) of the children using the age-adjusted dosing approach."( Safety, efficacy, and pharmacokinetics of intravenous busulfan in children undergoing allogeneic hematopoietic stem cell transplantation.
Chan, KW; Hayashi, RJ; Kadota, R; Kletzel, M; Mezzi, K; Nieder, ML; Przepiorka, D; Wall, DA; Yeager, AM, 2010
)
0.61
" Whether PK-based busulfan dosing can achieve further improvements in this setting is worthy of study."( Superior survival after replacing oral with intravenous busulfan in autologous stem cell transplantation for non-Hodgkin lymphoma with busulfan, cyclophosphamide and etoposide.
Andresen, S; Bolwell, BJ; Copelan, EA; Curtis, J; Dean, RM; Kalaycio, ME; Pohlman, B; Rybicki, LA; Smith, SD; Sobecks, RM; Sweetenham, JW, 2010
)
0.94
" The results suggest that a dosage of 30 mg kg(-1) is optimal for the ablative treatment with busulfan used to prepare the recipient mice."( Optimal dose of busulfan for depleting testicular germ cells of recipient mice before spermatogonial transplantation.
Wang, DZ; Yuan, YL; Zheng, XM; Zhou, XH, 2010
)
0.93
" Maintenance of plasma busulfan concentrations using repeated measurements and proper adjustment of dosage can reduce busulfan-related toxicity and improve treatment outcomes."( Quantification of busulfan in plasma using liquid chromatography electrospray tandem mass spectrometry (HPLC-ESI-MS/MS).
Ritchie, JC; Snyder, ML, 2010
)
1.01
" Bu in different dosing protocols, (2) compare intrasubject variability of Bu PK over repeated administrations; (3) examine the effect of concomitant administration of fludarabine on Bu PK, and (4) examine the effect of plasma concentrations of glutathione (GSH), the cosubstrate in Bu metabolism, on Bu clearance."( Linearity and stability of intravenous busulfan pharmacokinetics and the role of glutathione in busulfan elimination.
Almog, S; Gopher, A; Halkin, H; Hassoun, E; Kurnik, D; Loebstein, R; Nagler, A; Shimoni, A, 2011
)
0.64
" Bu dosing and continued until 24 hours after the last dose."( Influence on Busilvex pharmacokinetics of clonazepam compared to previous phenytoin historical data.
Bacigalupo, A; Buzyn, A; Cahn, JY; Carreras, E; Kröger, N; Puozzo, C; Sanz, G; Vernant, JP, 2010
)
0.36
" A dose-response relationship between busulfan exposure and outcome is known."( Phase I study of dose-escalated busulfan with fludarabine and alemtuzumab as conditioning for allogeneic hematopoietic stem cell transplant: reduced clearance at high doses and occurrence of late sinusoidal obstruction syndrome/veno-occlusive disease.
Artz, AS; Del Cerro, P; Godley, LA; Hart, J; Horowitz, S; Innocenti, F; Larson, RA; O'Donnell, PH; Odenike, OM; Pai, RK; Stock, W; Undevia, SD; Van Besien, K, 2010
)
0.92
" In the remaining 53 patients, the first-dose AUC was within the target range and no dosing adjustments were required."( Pharmacokinetic targeting of i.v. BU with fludarabine as conditioning before hematopoietic cell transplant: the effect of first-dose area under the concentration time curve on transplant-related outcomes.
Anasetti, C; Ayala, E; Fancher, K; Fernandez, H; Field, T; Gardiner, JA; Kharfan-Dabaja, MA; Kim, J; Miller, S; Milone, MC; Perez, L; Perkins, J; Shaw, LM; Tate, C, 2011
)
0.37
" These data suggest that personalizing either IV or oral busulfan dosing cannot be simplified on the basis of GSTA1 or GSTM1 genotype."( Pharmacogenetics of intravenous and oral busulfan in hematopoietic cell transplant recipients.
Abbasi, N; Blough, DK; Deeg, HJ; Ho, RJ; Kelly, EJ; Knutson, JA; McCune, JS; O'Donnell, PV; Pawlikowski, MA; Vadnais, B, 2011
)
0.88
" busulfan dosage scheme."( Influence of GST gene polymorphisms on the clearance of intravenous busulfan in adult patients undergoing hematopoietic cell transplantation.
Bae, KS; Choi, Y; Han, SB; Hur, EH; Kim, DY; Kim, SD; Lee, JH; Lee, KH; Lim, HS; Lim, SN; Noh, GJ; Yun, SC, 2011
)
1.51
"A prospective clinical trial was performed in order to validate the pharmacokinetic (PK) and clinical benefits of a new dosing schedule of intravenous busulfan (IV Bu) in children."( Weight-based strategy of dose administration in children using intravenous busulfan: clinical and pharmacokinetic results.
Bertrand, Y; Bordigoni, P; Demeocq, F; Doz, F; Esperou, H; Frappaz, D; Galambrun, C; Gentet, JC; Méchinaud, F; Michel, G; Neven, B; Nguyen, L; Socié, G; Valteau-Couanet, D; Vassal, G; Yakouben, K, 2012
)
0.81
"The new dosing schedule using IV Bu provides adequate therapeutic targeting from the first administration, with low toxicity and good disease control in high-risk children."( Weight-based strategy of dose administration in children using intravenous busulfan: clinical and pharmacokinetic results.
Bertrand, Y; Bordigoni, P; Demeocq, F; Doz, F; Esperou, H; Frappaz, D; Galambrun, C; Gentet, JC; Méchinaud, F; Michel, G; Neven, B; Nguyen, L; Socié, G; Valteau-Couanet, D; Vassal, G; Yakouben, K, 2012
)
0.61
" The prevalence of overweight and obese children has also increased in the pediatric cancer setting, causing substantial concern over proper chemotherapeutic dosing in this population."( Busulfan dosing in children with BMIs ≥ 85% undergoing HSCT: a new optimal strategy.
Browning, B; Donaldson, A; Halverson, T; Kletzel, M; Shinkle, M; Thormann, K, 2011
)
1.81
" Busulfan dosing is complex due to wide interpatient variability in pharmacokinetics and a narrow therapeutic range."( An automated method for supporting busulfan therapeutic drug monitoring.
Johnson-Davis, KL; Juenke, JM; McMillin, GA; Miller, KA, 2011
)
1.56
"Lower dosage of total body irradiation (TBI) and chemotherapy in reduced-intensity conditioning (RIC) regimens prior to allogeneic stem cell transplantation have reduced the toxicity of the conditioning and non-relapse mortality."( Intermediate intensity conditioning regimen containing FLAMSA, treosulfan, cyclophosphamide, and ATG for allogeneic stem cell transplantation in elderly patients with relapsed or high-risk acute myeloid leukemia.
Brossart, P; Chemnitz, JM; Hallek, M; Holtick, U; Krause, A; Scheid, C; Shimabukuro-Vornhagen, A; Theurich, S; von Lilienfeld-Toal, M, 2012
)
0.38
" We proposed a systematic classification scheme using FDA-approved drug labeling to assess the DILI potential of drugs, which yielded a benchmark dataset with 287 drugs representing a wide range of therapeutic categories and daily dosage amounts."( FDA-approved drug labeling for the study of drug-induced liver injury.
Chen, M; Fang, H; Liu, Z; Shi, Q; Tong, W; Vijay, V, 2011
)
0.37
" Busulfan clearance was not associated with sex or age, but was associated with the day of dosing and conditioning regimen (P = ."( Accurate targeting of daily intravenous busulfan with 8-hour blood sampling in hospitalized adult hematopoietic cell transplant recipients.
Blough, DK; Deeg, HJ; McCune, JS; McDonald, GB; O'Donnell, PV; Pawlikowski, MA; Rezvani, A; Yeh, RF, 2012
)
1.56
"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
" However, when both the test and the treatment doses were administered at the same infusion rate, clearance of the drug between the 2 dosing days was equivalent."( Development and validation of a test dose strategy for once-daily i.v. busulfan: importance of fixed infusion rate dosing.
Bahlis, NJ; Brown, CB; Chaudhry, MA; Daly, A; Duggan, P; Geddes, M; Kangarloo, SB; Magliocco, AM; Naveed, F; Ng, ES; Quinlan, D; Russell, JA; Savoie, ML; Shafey, M; Stewart, DA; Storek, J; Wu, J; Yang, M; Yue, P; Zacarias, N, 2012
)
0.61
"Analytical variance and the AUC calculation method play a considerable role in the clinical management of busulfan dosing during HSCT."( Clinical consequences of analytical variance and calculation strategy in oral busulfan pharmacokinetics.
Clarke, W; Lombardi, L; Olson, MT, 2011
)
0.81
"To evaluate the best method for dosing busulfan in children, we retrospectively analyzed two different data sets from three different dosing regimens by means of population pharmacokinetics using NONMEM."( Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children.
Bergstrand, M; Boos, J; Hempel, G; Karlsson, MO; Trame, MN, 2011
)
0.92
"9 years, who belonged to the once-daily intravenous busulfan dosing regimen."( Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children.
Bergstrand, M; Boos, J; Hempel, G; Karlsson, MO; Trame, MN, 2011
)
0.91
" On the basis of the final models, we simulated two dosing schemes according to allometric BW and BSA showing that we estimated to include about 30% more patients into the proposed therapeutic area under the curve (AUC) range of 900 to 1,500 μM*min and could, furthermore, achieve a reduction in the AUC variability when dosed according to the labeled European Medicines Agency (EMA) dosing recommendation."( Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children.
Bergstrand, M; Boos, J; Hempel, G; Karlsson, MO; Trame, MN, 2011
)
0.66
"We recommend a BSA or an allometric BW dosing regimen for individualizing busulfan therapy in children to reduce variability in busulfan exposure and to improve safety and efficacy of busulfan treatment."( Population pharmacokinetics of busulfan in children: increased evidence for body surface area and allometric body weight dosing of busulfan in children.
Bergstrand, M; Boos, J; Hempel, G; Karlsson, MO; Trame, MN, 2011
)
0.89
"The clinical advantage of pharmacokinetic (PK)-directed-based dosing on intravenous (i."( Pharmacokinetic-directed high-dose busulfan combined with cyclophosphamide and etoposide results in predictable drug levels and durable long-term survival in lymphoma patients undergoing autologous stem cell transplantation.
Ali, Z; Dada, MO; Flowers, CR; Graiser, M; Hutcherson, DA; McMillan, S; Waller, EK; Zhang, H, 2012
)
0.66
"Intravenous (IV) busulfan (Bu) dosing approved in Europe based on 5 body weight (BW) strata has been validated for targeting Bu exposures in children undergoing hematopoietic stem-cell transplantation and with mostly malignant diseases."( Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: the results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation.
Abdi, ZD; Bleyzac, N; Broutin, S; Dalle, JH; Galambrun, C; Kemmel, V; Moshous, D; Neven, B; Nguyen, L; Paci, A; Pétain, A; Vassal, G, 2012
)
0.97
" Bu dosing either adjusted according to the final model or with the approved EU labeling yields similar targeting performances."( Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: the results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation.
Abdi, ZD; Bleyzac, N; Broutin, S; Dalle, JH; Galambrun, C; Kemmel, V; Moshous, D; Neven, B; Nguyen, L; Paci, A; Pétain, A; Vassal, G, 2012
)
0.63
" The BW-based dosing strategy recommended in Europe proved to be consistent on a large paediatric cohort representative of the population heterogeneity observed in hematopoietic stem-cell transplantation."( Pharmacokinetic behavior and appraisal of intravenous busulfan dosing in infants and older children: the results of a population pharmacokinetic study from a large pediatric cohort undergoing hematopoietic stem-cell transplantation.
Abdi, ZD; Bleyzac, N; Broutin, S; Dalle, JH; Galambrun, C; Kemmel, V; Moshous, D; Neven, B; Nguyen, L; Paci, A; Pétain, A; Vassal, G, 2012
)
0.63
"1 to 26 years of age, elucidate patient characteristics that explain the variability in exposure between patients and optimize dosing accordingly."( Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing.
Bartelink, IH; Bierings, MB; Boelens, JJ; Bredius, RG; Danhof, M; Egberts, AC; Hempel, G; Knibbe, CA; Nath, CE; Shaw, PJ; Wang, C; Zwaveling, J, 2012
)
0.64
" Based on the final pharmacokinetic-model, an individualized dosing nomogram was developed."( Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing.
Bartelink, IH; Bierings, MB; Boelens, JJ; Bredius, RG; Danhof, M; Egberts, AC; Hempel, G; Knibbe, CA; Nath, CE; Shaw, PJ; Wang, C; Zwaveling, J, 2012
)
0.64
"The model-based individual dosing nomogram is expected to result in predictive busulfan exposures in patients ranging between 3 and 65 kg and thereby to a safer and more effective conditioning regimen for HSCT in children."( Body weight-dependent pharmacokinetics of busulfan in paediatric haematopoietic stem cell transplantation patients: towards individualized dosing.
Bartelink, IH; Bierings, MB; Boelens, JJ; Bredius, RG; Danhof, M; Egberts, AC; Hempel, G; Knibbe, CA; Nath, CE; Shaw, PJ; Wang, C; Zwaveling, J, 2012
)
0.87
" Analytical methods developed for Bu routine monitoring were aimed at using low volumes of biological fluids and development of simple procedures to facilitate the dosage adjustment."( A simplified method for busulfan monitoring using dried blood spot in combination with liquid chromatography/tandem mass spectrometry.
Ansari, M; Daali, Y; Dayer, P; Déglon, J; Desmules, J; Gumy-Pause, F; Ozsahin, H; Théorêt, Y; Uppugunduri, CR; Versace, F, 2012
)
0.69
"The new DBS method facilitates earlier dosage adjustment during Bu therapy by its specific and simple procedure using 5 μL of whole blood."( A simplified method for busulfan monitoring using dried blood spot in combination with liquid chromatography/tandem mass spectrometry.
Ansari, M; Daali, Y; Dayer, P; Déglon, J; Desmules, J; Gumy-Pause, F; Ozsahin, H; Théorêt, Y; Uppugunduri, CR; Versace, F, 2012
)
0.69
" Still, in different mammalian species wide variability in optimal doses of busulfan has been demonstrated, whereas in birds, the dosage has not yet been optimized."( The effect of busulfan treatment on endogenous spermatogonial stem cells in immature roosters.
Golovan, S; Tagirov, M, 2012
)
0.97
" Clo 40 mg/m(2) was given once daily, with each dose followed by pharmacokinetically dosed Bu infused over 3 hours daily for 4 days, followed by hematopoietic SCT 2 days later."( Clofarabine combined with busulfan provides excellent disease control in adult patients with acute lymphoblastic leukemia undergoing allogeneic hematopoietic stem cell transplantation.
Alousi, A; Andersson, BS; Basset, R; Champlin, RE; Ciurea, S; de Lima, M; Hosing, C; Jones, RB; Kebriaei, P; Khouri, I; Ledesma, C; Nieto, Y; Parmar, S; Popat, U; Qazilbash, M; Shpall, EJ, 2012
)
0.68
" In conclusion, GST and ABCB1 genotyping may assist care-givers in personalizing BU dosage with less trial-and-error and may enable preemptive identification of patients at risk for BU toxicity."( Pharmacokinetic and pharmacogenetic analysis of oral busulfan in stem cell transplantation: prediction of poor drug metabolism to prevent drug toxicity.
Efrati, E; Elkin, H; Froymovich, L; Krivoy, N; Rowe, JM; Zuckerman, T, 2012
)
0.63
" A model for the dosage adjustment with the inclusion of genetic and non-genetic factors should be evaluated in a future prospective validation cohort."( Glutathione S-transferase gene variations influence BU pharmacokinetics and outcome of hematopoietic SCT in pediatric patients.
Ansari, M; Bittencourt, H; Champagne, M; Desjean, C; Duval, M; Krajinovic, M; Labuda, M; Mezziani, S; Peters, C; Przybyla, C; Rezgui, MA; Rousseau, J; Théoret, Y; Uppugunduri, CR; Vachon, MF, 2013
)
0.39
" The authors sought to characterize initial dosing and TDM of IV busulfan, along with factors associated with busulfan clearance, in 729 children who underwent busulfan TDM from December 2005 to December 2008."( Variation in prescribing patterns and therapeutic drug monitoring of intravenous busulfan in pediatric hematopoietic cell transplant recipients.
Baker, KS; Barrett, JS; Bemer, MJ; Blough, DK; Gamis, A; Kelton-Rehkopf, MC; McCune, JS; Winter, L, 2013
)
0.85
" To solve this problem, we proposed a new dosing scheme, in which the amount of dose was adjusted dynamically according to the estimated precision of the AUC estimator."( A proposed dynamic dosing scheme to secure the target plasma drug concentrations based on the precision of the AUC estimate.
Tsuruta, H; Wada, T, 2013
)
0.39
" Assuming an increase in clearance overtime as found in our previous investigation, separate time factors for the two different dosing schedules included in the dataset were tested."( Population pharmacokinetics of dimethylacetamide in children during standard and once-daily IV busulfan administration.
Bartelink, IH; Boelens, JJ; Boos, J; Hempel, G; Trame, MN, 2013
)
0.61
" The rapid clearance with different dosing in patients of different body weights indicates that it is safe to use DMA in children in both a once and four times daily regimen."( Population pharmacokinetics of dimethylacetamide in children during standard and once-daily IV busulfan administration.
Bartelink, IH; Boelens, JJ; Boos, J; Hempel, G; Trame, MN, 2013
)
0.61
"Intravenous (IV) busulfan (Bu) combined with therapeutic drug monitoring-guided dosing is associated with better event-free survival (EFS), lower transplant-related mortality."( Association between busulfan exposure and outcome in children receiving intravenous busulfan before hematopoietic stem cell transplantation.
Ansari, M; Bittencourt, H; Champagne, MA; Desjean, C; Duval, M; Krajinovic, M; Mezziani, S; Peters, C; Rezgui, MA; Théoret, Y; Vachon, MF, 2014
)
1.07
"A busulfan concentration monitoring and dosing service has been provided by Christchurch Hospital since 1998."( Thirteen years' experience of pharmacokinetic monitoring and dosing of busulfan: can the strategy be improved?
Allen, KM; Barclay, ML; Begg, EJ; Buffery, PJ; Chin, PK; Moore, GA, 2014
)
1.36
" The relationship of CL and body weight for the IV group was used to develop a revised IV dosing schedule."( Thirteen years' experience of pharmacokinetic monitoring and dosing of busulfan: can the strategy be improved?
Allen, KM; Barclay, ML; Begg, EJ; Buffery, PJ; Chin, PK; Moore, GA, 2014
)
0.64
" Dose adjustment was made in 47% and 34% of patients dosed IV and orally, respectively, after which there was a trend to more patients achieving the target AUC."( Thirteen years' experience of pharmacokinetic monitoring and dosing of busulfan: can the strategy be improved?
Allen, KM; Barclay, ML; Begg, EJ; Buffery, PJ; Chin, PK; Moore, GA, 2014
)
0.64
" The relationship between CL and body weight was used to revise the initial IV dosing schedule."( Thirteen years' experience of pharmacokinetic monitoring and dosing of busulfan: can the strategy be improved?
Allen, KM; Barclay, ML; Begg, EJ; Buffery, PJ; Chin, PK; Moore, GA, 2014
)
0.64
" In study 1, dose-response relationships were investigated in three-day-old pigs (Landrace × Yorkshire × Duroc, n = 6)."( Intestinal response to myeloablative chemotherapy in piglets.
Heilmann, C; Müller, K; Petersen, BL; Pontoppidan, PL; Sangild, PT; Shen, RL; Thymann, T, 2014
)
0.4
" This article presents an outline on performing a population PK/PD study and translating these results into rational dosing regimens, with the development and prospective evaluation of PK/PD derived evidence-based dosing regimen being discussed."( Towards evidence-based dosing regimens in children on the basis of population pharmacokinetic pharmacodynamic modelling.
Admiraal, R; Boelens, JJ; Bredius, RG; Knibbe, CA; Tibboel, D; van Kesteren, C, 2014
)
0.4
" 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
" Pharmacokinetic data to optimize treosulfan dosing are scarce in this patient population."( Pharmacokinetics of treosulfan in pediatric patients undergoing hematopoietic stem cell transplantation.
Ackaert, O; Bredius, RG; den Hartigh, J; Guchelaar, HJ; Lankester, AC; Smiers, FJ; Ten Brink, MH; Zwaveling, J, 2014
)
0.4
"We utilized a new reduced intensity conditioning (RIC) containing of new dosage of intravenous Bu (9."( [The efficacy and safety of modified busulfan/fludarabine conditioning regimen in elderly or drug-intolerable patients with hematologic malignancies].
Chen, H; Fu, HX; Huang, XJ; Liu, DH; Liu, KY; Sun, YQ; Tang, FF; Wang, FR; Wang, Y; Xu, LP, 2013
)
0.66
") busulfan in children to evaluate the optimal method to personalize its dosing without concentration-time data."( Population pharmacokinetics of intravenous busulfan in children: revised body weight-dependent NONMEM® model to optimize dosing.
Boos, J; Diestelhorst, C; Hempel, G; McCune, JS, 2014
)
1.39
" The dosing nomogram for every 6 h administration derived from the final model is: dose[mg]=target AUC[mg×h/L]×3."( Population pharmacokinetics of intravenous busulfan in children: revised body weight-dependent NONMEM® model to optimize dosing.
Boos, J; Diestelhorst, C; Hempel, G; McCune, JS, 2014
)
0.67
"We revised our prior dosing nomogram after validation in a separate cohort of children."( Population pharmacokinetics of intravenous busulfan in children: revised body weight-dependent NONMEM® model to optimize dosing.
Boos, J; Diestelhorst, C; Hempel, G; McCune, JS, 2014
)
0.67
"Series of observations indicate PK/PD variability challenging the accuracy of the body-weight based busulfan (Bu) dosing schedule for (HSCT) conditioning therapy."( The importance of therapeutic drug monitoring (TDM) for parenteral busulfan dosing in conditioning regimen for hematopoietic stem cell transplantation (HSCT) in children.
Branova, R; Janeckova, D; Keslova, P; Klapkova, E; Malis, J; Prusa, R; Riha, P; Sedlacek, P; Tesfaye, H, 2014
)
0.85
" Overall, patients dosed by DBW were more likely to undershoot goal AUC (-12."( Busulfan dosing (Q6 or Q24) with adjusted or actual body weight, does it matter?
Awan, FT; Clemmons, AB; DeRemer, DL; Evans, S, 2015
)
1.86
" The authors aimed to develop a population PK model for busulfan in children that can reliably achieve therapeutic exposure (concentration at steady state) and implement a simple model-based tool for the initial dosing of busulfan in children undergoing hematopoietic cell transplantation."( Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use.
Bartelink, I; Cowan, MJ; Dvorak, CC; French, D; Horn, B; Law, J; Long-Boyle, JR; Musick, L; Savic, R; Yan, S, 2015
)
0.95
" The final population PK model was implemented into a clinician-friendly Microsoft Excel-based tool and used to recommend initial doses of busulfan in a group of 21 pediatric patients prospectively dosed based on the population PK model."( Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use.
Bartelink, I; Cowan, MJ; Dvorak, CC; French, D; Horn, B; Law, J; Long-Boyle, JR; Musick, L; Savic, R; Yan, S, 2015
)
0.91
"When compared with the conventional dosing guidelines, the model-based algorithm demonstrates significant improvement for providing targeted busulfan therapy in children and young adults."( Population pharmacokinetics of busulfan in pediatric and young adult patients undergoing hematopoietic cell transplant: a model-based dosing algorithm for personalized therapy and implementation into routine clinical use.
Bartelink, I; Cowan, MJ; Dvorak, CC; French, D; Horn, B; Law, J; Long-Boyle, JR; Musick, L; Savic, R; Yan, S, 2015
)
0.9
" Body weight (BW), or adjusted ideal body weight (AIBW)-based dosing (WBD) algorithm, has been used in hematopoietic stem cell transplantation (HSCT)."( Dosing algorithm revisit for busulfan following IV infusion.
Armstrong, E; Kato, K; Le Gallo, C; Rock, E; Wang, X; Wang, Y, 2015
)
0.71
" Dosing algorithm was developed based on derived covariate model of CL."( Dosing algorithm revisit for busulfan following IV infusion.
Armstrong, E; Kato, K; Le Gallo, C; Rock, E; Wang, X; Wang, Y, 2015
)
0.71
" The proposed dosing algorithm was dose (mg) = (31."( Dosing algorithm revisit for busulfan following IV infusion.
Armstrong, E; Kato, K; Le Gallo, C; Rock, E; Wang, X; Wang, Y, 2015
)
0.71
"The proposed dosing algorithm can significantly improve the sub-exposure of Bu."( Dosing algorithm revisit for busulfan following IV infusion.
Armstrong, E; Kato, K; Le Gallo, C; Rock, E; Wang, X; Wang, Y, 2015
)
0.71
" We aimed this retrospective study for comparison of weight- and age-based dosing in terms of clinical outcomes such as time to engraftment, early complications, EFS, OS, and toxicity profiles in children receiving iv Bu."( Clinical comparison of weight- and age-based strategy of dose administration in children receiving intravenous busulfan for hematopoietic stem cell transplantation.
Avci, Z; Azik, F; Gürlek Gökçebay, D; Isik, P; Kara, A; Ozbek, N; Tavil, B; Tunc, B, 2015
)
0.63
"The aim of this investigation was to develop a model-based dosing algorithm for busulfan and identify an optimal sampling scheme for use in routine clinical practice."( Busulfan dosing algorithm and sampling strategy in stem cell transplantation patients.
de Castro, FA; Della Pasqua, O; Lanchote, VL; Piana, C; Simões, BP, 2015
)
2.09
" A one compartment model was selected as basis for sampling optimization and subsequent evaluation of a suitable dosing algorithm."( Busulfan dosing algorithm and sampling strategy in stem cell transplantation patients.
de Castro, FA; Della Pasqua, O; Lanchote, VL; Piana, C; Simões, BP, 2015
)
1.86
" Based on simulation scenarios, a dosing algorithm was identified, which ensures target exposure values are attained after a test dose."( Busulfan dosing algorithm and sampling strategy in stem cell transplantation patients.
de Castro, FA; Della Pasqua, O; Lanchote, VL; Piana, C; Simões, BP, 2015
)
1.86
"The use of the proposed dosing algorithm in conjunction with a sparse sampling scheme may contribute to considerable improvement in the safety and efficacy profile of patients undergoing treatment for stem cell transplantation."( Busulfan dosing algorithm and sampling strategy in stem cell transplantation patients.
de Castro, FA; Della Pasqua, O; Lanchote, VL; Piana, C; Simões, BP, 2015
)
1.86
" Even though dosing and timing might have been the reasons for this failure, it might also be that TREO does not reach the brain in sufficient amount."( Transport of treosulfan and temozolomide across an in-vitro blood-brain barrier model.
Hupert, M; Linz, U; Santiago-Schübel, B; Stab, J; Wagner, S; Wien, S, 2015
)
0.42
" Multiple pediatric busulfan dosing guidelines aim to achieve this target."( Performance of Busulfan Dosing Guidelines for Pediatric Hematopoietic Stem Cell Transplant Conditioning.
Dupuis, LL; Egeler, RM; Gassas, A; Gerges, S; Grunebaum, E; Liu, WJ; Schechter, T; Zao, JH, 2015
)
1.09
" 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
)
1.58
" Therapeutic drug monitoring (TDM)-guided dosing to reach the conventional area under the concentration-time curve (AUC) target range of 900-1500 μmol min/L is associated with better outcomes."( Should busulfan therapeutic range be narrowed in pediatrics? Experience from a large cohort of hematopoietic stem cell transplant children.
Bergeron, C; Bertrand, Y; Bleyzac, N; Fonrose, X; Girard, P; Goutelle, S; Guitton, J; Philippe, M, 2016
)
0.89
" 32 male Wistar rats were injected with a double dosage of 15 ml/kg busulfan with 14 days interval."( Effect of human recombinant granulocyte colony-stimulating factor on rat busulfan-induced testis injury.
Amidi, F; Khanlarkhani, N; Mortezaee, K; Najafi, A; Naji, M; Pasbakhsh, P; Sobhani, A; Zendedel, A, 2016
)
0.9
" They used it to simulate optimal initial busulfan dosages, and in a blinded manner, they compared dosage adjustments using the model in the BestDose software to dosage adjustments calculated by noncompartmental estimation of area under the time-concentration curve at a national reference laboratory in a cohort of patients not included in model building."( Accurately Achieving Target Busulfan Exposure in Children and Adolescents With Very Limited Sampling and the BestDose Software.
Bayard, D; Bleyzac, N; Fu, X; Goutelle, S; Neely, M; Philippe, M; Rushing, T; Schumitzky, A; van Guilder, M, 2016
)
0.99
"BestDose accurately calculates busulfan intravenous dosage requirements to achieve target plasma exposures in children up to 18 years of age and 110 kg using only 2 blood samples per adjustment compared with 6-9 samples for standard noncompartmental dose calculations."( Accurately Achieving Target Busulfan Exposure in Children and Adolescents With Very Limited Sampling and the BestDose Software.
Bayard, D; Bleyzac, N; Fu, X; Goutelle, S; Neely, M; Philippe, M; Rushing, T; Schumitzky, A; van Guilder, M, 2016
)
1.01
" However, the application of different sampling strategies and pharmacokinetic approaches results in different dosing recommendations and ostensibly different outcomes."( Intravenous busulfan dose individualization - impact of modeling approach on dose recommendation.
Abdel-Rahman, SM; Casey, KL; Dalal, J; Garg, U, 2016
)
0.81
" 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
)
1.11
" To facilitate a therapeutic dose-monitoring protocol, we transitioned from Bu6 to daily Bu dosing for patients with Hodgkin and non-Hodgkin lymphoma undergoing autologous stem cell transplantation (ASCT)."( Daily Weight-Based Busulfan with Cyclophosphamide and Etoposide Produces Comparable Outcomes to Four-Times-Daily Busulfan Dosing for Lymphoma Patients Undergoing Autologous Stem Cell Transplantation.
Andresen, S; Bolwell, BJ; Carlstrom, KD; Dean, R; Gerds, A; Hamilton, B; Hill, BT; Jagadeesh, D; Kalaycio, M; Liu, H; Majhail, NS; Pohlman, B; Rybicki, L; Sobecks, R, 2016
)
0.76
"The traditional approach for model-based initial dosing is based on the use of a single vector of typical population parameters for targeting a specific exposure."( A Nonparametric Method to Optimize Initial Drug Dosing and Attainment of a Target Exposure Interval: Concepts and Application to Busulfan in Pediatrics.
Bertrand, Y; Bleyzac, N; Goutelle, S; Neely, M; Philippe, M, 2017
)
0.66
" The aim of this study was to derive a novel once-daily intravenous (IV) busulfan dosing nomogram for pediatric patients undergoing HSCT using a population pharmacokinetic (PK) model."( Pediatric patients undergoing hematopoietic stem cell transplantation can greatly benefit from a novel once-daily intravenous busulfan dosing nomogram.
Choi, JY; Hong, CR; Hong, KT; Kang, HJ; Lee, H; Lee, JW; Park, KD; Rhee, SJ; Shin, HY; Song, SH; Yu, KS, 2017
)
0.89
" TDM remains an important tool for the appropriate dosing of Bu in preparative regimens of hematopoietic stem cell transplantation, especially in populations with genetic admixture."( Therapeutic drug monitoring-guided dosing of busulfan differs from weight-based dosing in hematopoietic stem cell transplant patients.
Al-Farsi, K; Al-Huneini, M; Al-Khabori, M; Al-Kindi, S; Al-Rawas, A; Al-Za'abi, M; Dennison, D; Salman, B; Tauro, M, 2017
)
0.71
"Owing to its narrow therapeutic range and high pharmacokinetic variability, optimal dosing for busulfan is important to minimise overexposure-related systemic toxicity and underexposure-related graft failure."( Pharmacometabolomics for predicting variable busulfan exposure in paediatric haematopoietic stem cell transplantation patients.
Ahn, HS; Cho, JY; Hong, KT; Jang, IJ; Kang, HJ; Kim, B; Lee, JW; Park, KD; Shin, HY; Yu, KS, 2017
)
0.93
" Despite intravenous (IV) administration and dosing recommendations based on age and weight, reports have revealed interindividual variability in Bu pharmacokinetics and the outcomes of hematopoietic stem cell transplantation."( Therapeutic Drug Monitoring of Busulfan for the Management of Pediatric Patients: Cross-Validation of Methods and Long-Term Performance.
Ansari, M; Chalandon, Y; Choong, E; Daali, Y; Doffey-Lazeyras, F; Kuntzinger, M; Lo Piccolo, R; Marino, D; Peters, C; Uppugunduri, CRS, 2018
)
0.77
" In our experience with pediatric patients, test dose PK failed to reliably predict daily dosing requirements with large discrepancies from predicted AUC targets."( Test Dose Pharmacokinetics in Pediatric Patients Receiving Once-Daily IV Busulfan Conditioning for Hematopoietic Stem Cell Transplant: A Reliable Approach?
Brooks, KM; De Ravin, SS; Gall, JBL; George, JM; Hickstein, DD; Hughes, T; Jarosinski, P; Kang, E; Kumar, P; Shah, NN, 2018
)
0.71
"Currently, there is an urgent need to establish the optimal dosing of TREO in conditioning prior to hematopoietic stem cell transplantation, especially in children."( Clinical bioanalysis of treosulfan and its epoxides: The importance of collected blood processing for valid pharmacokinetic results.
Główka, F; Romański, M, 2018
)
0.48
" Of note, the optimal dosing of the prodrug is still unresolved, especially in infants."( Treosulfan Pharmacokinetics and its Variability in Pediatric and Adult Patients Undergoing Conditioning Prior to Hematopoietic Stem Cell Transplantation: Current State of the Art, In-Depth Analysis, and Perspectives.
Główka, FK; Romański, M; Wachowiak, J, 2018
)
0.48
" Intravenous Busulfan (Bu) combined with therapeutic drug monitoring-guided dosing has been increasingly used, with more predictable bioavailability and better outcomes comparing to oral Bu."( Pharmacokinetics-adapted Busulfan-based myeloablative conditioning before unrelated umbilical cord blood transplantation for myeloid malignancies in children.
Ansari, M; Benadiba, J; Bittencourt, H; Cellot, S; Duval, M; Krajinovic, M; Teira, P; Vachon, MF, 2018
)
1.15
" Using this model, the plasma concentrations for once-daily dosing were simulated to adapt new dosage regimens for the benefit and convenience of both patients and medical staff."( Population Pharmacokinetic Analysis of Busulfan in Japanese Pediatric and Adult HCT Patients.
Funaki, T; Kawazoe, A; Kim, S, 2018
)
0.75
" Conditioning with treosulfan alone at nonmyeloablative dosing (3."( Bone Marrow Transplantation after Nonmyeloablative Treosulfan Conditioning Is Curative in a Murine Model of Sickle Cell Disease.
Devadasan, D; Goldman, FD; Pawlik, KM; Sun, CW; Townes, TM; Westin, ER; Wu, LC, 2018
)
0.48
"Weight-based dosing of intravenous busulfan is widely used in hematopoietic cell transplantation."( Pharmacokinetic and clinical outcomes when ideal body weight is used to dose busulfan in obese hematopoietic stem cell transplant recipients.
Griffin, SP; Hsu, JW; Murthy, HS; Richards, AI; Wheeler, SE; Wiggins, LE, 2019
)
1.02
" We used an intensive daily pharmacokinetic monitoring method for busulfan dosing in children for effective myeloablation and to reduce toxicity."( Favorable Outcome of Post-Transplantation Cyclophosphamide Haploidentical Peripheral Blood Stem Cell Transplantation with Targeted Busulfan-Based Myeloablative Conditioning Using Intensive Pharmacokinetic Monitoring in Pediatric Patients.
Cheon, JE; Choi, JY; Hong, CR; Hong, KT; Jang, IJ; Kang, HJ; Park, JD; Park, KD; Shin, HY; Song, SH; Yu, KS, 2018
)
0.92
" These findings may have important implications for busulfan dosing and therapeutic drug monitoring practice in HSCT children."( Maximal concentration of intravenous busulfan as a determinant of veno-occlusive disease: a pharmacokinetic-pharmacodynamic analysis in 293 hematopoietic stem cell transplanted children.
Bertrand, Y; Bleyzac, N; Goutelle, S; Neely, M; Philippe, M; Rushing, T, 2019
)
1.04
"Targeted busulfan dosing helps limit chemotherapy-related toxicity and optimize disease outcomes in hematopoietic stem cell transplantation (HCT)."( Evaluation of a Test Dose Strategy for Pharmacokinetically-Guided Busulfan Dosing for Hematopoietic Stem Cell Transplantation.
Alexander, MD; Armistead, PM; Chung, Y; Coghill, JM; Davis, JM; Ivanova, A; Jamieson, KJ; Ptachcinski, JR; Rao, KV; Riches, ML; Serody, JS; Sharf, AA; Shaw, JR; Shea, TC; Vincent, BG; Wood, WA, 2019
)
1.17
" The RIC and MAC regimens were dosed at the average daily area under the concentration-vs-time curve (AUC) of 4000 µMol min and 5000-6000 µMol min, or total course AUC of 16,000 µMol min and 20,000-24,000 µMol min, respectively; PK-guided dosing removes overlap in systemic Bu exposure."( Reduced intensity vs. myeloablative conditioning with fludarabine and PK-guided busulfan in allogeneic stem cell transplantation for patients with AML/MDS.
Alatrash, G; Andersson, BS; Champlin, RE; Chen, J; Crain, AK; Di Stasi, A; Jones, RB; Kidwell, KM; Popat, U; Shpall, EJ; Thall, PF; Zope, M, 2019
)
0.74
"Imprecise blood collections or cross-contamination of samples may lead to inaccurate drug concentration results and, subsequently, undesired low or high drug dosage calculations."( Pharmacokinetics: Unique Challenges in Blood Monitoring for Oncology Nurses.
Andersson, BS; Gulbis, AM; Kalariya, N; Kawedia, JD; Myers, AL, 2019
)
0.51
"Busulfan is used in myeloablative schemes for hematopoietic stem cell transplantation (HSCT), with monitoring of dosage through the area under the curve (AUC) of the drug plasma concentration (µMol min)."( Pharmacokinetics analysis results are similar for oral compared to intravenous busulfan in patients undergoing hematopoietic stem cell transplantation, except for the earlier onset of mucositis. A controlled clinical study.
de Lima, M; Esteves, I; Fernandes, JF; Hamerschlak, N; Kerbauy, FR; Oliveira, JSR; S Andersson, B; Santos, FPS; Seber, A, 2019
)
2.18
" A model-based dosing table is presented to target an exposure of 1650 mg*h/L (population median) for different weight and age groups."( Population pharmacokinetics of treosulfan in paediatric patients undergoing hematopoietic stem cell transplantation.
Bertaina, A; Guchelaar, HJ; Lankester, AC; Locatelli, F; Moes, DJAR; van der Stoep, MYEC; Zwaveling, J, 2019
)
0.51
"It is well known that pharmacokinetics (PK)-guided busulfan (BU) dosing increases engraftment rates and lowers hepatotoxicity in patients undergoing hematopoietic cell transplantation (HCT)."( Busulfan Pharmacokinetics and Precision Dosing: Are Patients with Fanconi Anemia Different?
Boulad, F; Davies, SM; Emoto, C; Fukuda, T; Fuller, K; Mehta, PA; Seyboth, B; Teusink-Cross, A; Vinks, AA; Wilhelm, J, 2019
)
2.21
" We identified busulfan dosing frequency during allogeneic hematopoietic cell transplantation (HCT) conditioning as a potential target for optimization."( Costs and Outcomes with Once-Daily versus Every-6-Hour Intravenous Busulfan in Allogeneic Hematopoietic Cell Transplantation.
Bassett, B; Jenkins, P; Kim, T; Rezvani, AR; Singhal, S; Tierney, DK, 2020
)
1.15
" At our institution, Bu was previously administered with fixed weight-based dosing (WBD) in combination with cyclophosphamide (Cy) and etoposide (E) for patients with non-Hodgkin lymphoma (NHL) undergoing autologous stem cell transplantation (ASCT)."( Therapeutic Dose Monitoring of Busulfan Is Associated with Reduced Risk of Relapse in Non-Hodgkin Lymphoma Patients Undergoing Autologous Stem Cell Transplantation.
Bolwell, B; Copelan, EA; Dean, RM; Gerds, AT; Hamilton, BK; Hill, BT; Jagadeesh, D; Kalaycio, ME; Lucena, M; Majhail, NS; Pohlman, B; Rybicki, LA; Sobecks, RM; Urban, TA, 2020
)
0.84
" The main objectives were: (i) to establish a mechanistic pharmacokinetic-pharmacodynamic (PKPD) model for the treatment and engraftment effects on neutrophil counts comparing busulfan and treosulfan-based conditioning, and (ii) to explore current dosing schedules with respect to time to HSCT."( Modelling of neutrophil dynamics in children receiving busulfan or treosulfan for haematopoietic stem cell transplant conditioning.
Chiesa, R; Doncheva, B; Prunty, H; Solans, BP; Standing, JF; Trocóniz, IF; Veys, P, 2020
)
1
" However, TDM-based dosing may be challenging if intra-individual pharmacokinetic variability (also denoted inter-occasion variability [IOV]) occurs during therapy."( Intra-individual Pharmacokinetic Variability of Intravenous Busulfan in Hematopoietic Stem Cell-Transplanted Children.
Bertrand, Y; Bleyzac, N; Ducher, M; Goutelle, S; Guitton, J; Leclerc, V; Marsit, H; Neely, M; Philippe, M; Rushing, T, 2020
)
0.8
" DMSPE coupling with liquid chromatography with tandem mass spectrometry (LC-MS/MS) was implemented for the determination of busulfan dosage in plasma samples."( Dispersive micro solid phase extraction of busulfan from plasma samples using novel mesoporous sorbent prior to determination by HPLC-MS/MS.
Ghaffary, S; Hamidi, S; Jahed, FS; Nejati, B, 2020
)
1.03
" Appropriate dosing of busulfan during the preparative phase is critical for a successful allograft; if blood concentrations get too high significant liver toxicity can occur, if blood concentrations are too low, then graft-versus-host disease (GVHD) can develop."( Analysis of Busulfan in Plasma by Liquid Chromatography-Tandem Mass Spectrometry (LC-MS/MS).
Breaud, AR; Clarke, W; Pablo, A, 2020
)
1.25
" 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
)
0.76
" In 3 successive clinical trials, which included either γ-retroviral (γ-RV) or lentiviral (LV) vectors, subjects were conditioned with BU using different dosing nomograms."( Busulfan Pharmacokinetics in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Gene Therapy.
Ansari, M; Bradford, KL; Candotti, F; Garabedian, E; Gaspar, HB; Kohn, DB; Krajinovic, M; Liu, S; Shaw, KL; Tse, J; Wang, X, 2020
)
2
" A new dosing nomogram was evaluated to better fit the population pharmacokinetic model."( New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning.
Bondu, S; Bourget, P; Broutin, S; Dalle, JH; De Berranger, E; Devictor, B; Dufour, C; Faivre, L; Galambrun, C; Gandemer, V; Jubert, C; Kemmel, V; Mir, O; Moshous, D; Neven, B; Nguyen, L; Paci, A; Petain, A; Poinsignon, V; Vannier, JP; Vassal, G, 2020
)
0.77
" Median Bu area under the curve (AUC) was 1179 µmol/L × min compared to 1025 with the EMA dosing nomogram for children <9 kg."( New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning.
Bondu, S; Bourget, P; Broutin, S; Dalle, JH; De Berranger, E; Devictor, B; Dufour, C; Faivre, L; Galambrun, C; Gandemer, V; Jubert, C; Kemmel, V; Mir, O; Moshous, D; Neven, B; Nguyen, L; Paci, A; Petain, A; Poinsignon, V; Vannier, JP; Vassal, G, 2020
)
0.77
"This new model made it possible to propose a novel dosing nomogram that better considered children below 16 kg of BW and allowed better initial exposure as compared to existing dosing schedules."( New dosing nomogram and population pharmacokinetic model for young and very young children receiving busulfan for hematopoietic stem cell transplantation conditioning.
Bondu, S; Bourget, P; Broutin, S; Dalle, JH; De Berranger, E; Devictor, B; Dufour, C; Faivre, L; Galambrun, C; Gandemer, V; Jubert, C; Kemmel, V; Mir, O; Moshous, D; Neven, B; Nguyen, L; Paci, A; Petain, A; Poinsignon, V; Vannier, JP; Vassal, G, 2020
)
0.77
" The combination of model-based dosing and therapeutic drug monitoring (TDM) of busulfan pharmacokinetics (PK) to a lower exposure target has the potential to reduce the regimen-related toxicity while opening marrow niches sufficient for engraftment in diseases such as mucopolysaccharidosis type I (MPS I)."( Lower Exposure to Busulfan Allows for Stable Engraftment of Donor Hematopoietic Stem Cells in Children with Mucopolysaccharidosis Type I: A Case Report of Four Patients.
Dvorak, CC; Kharbanda, S; Long-Boyle, J; Shukla, P, 2020
)
1.12
" Prophylactic T cell depletion via antithymocyte globulin (ATG) during ASCT conditioning is one of the standards of care for GVHD prophylaxis, although the optimal dosing strategy is still unclear."( Antithymocyte globulin administration in patients with profound lymphopenia receiving a PBSC purine analog/busulfan-based conditioning regimen allograft.
Bene, MC; Blin, N; Chevallier, P; Debord, C; Dubruille, V; Duquesne, A; Eveillard, M; Garnier, A; Gastinne, T; Guillaume, T; Jullien, M; Le Bourgeois, A; Le Bris, Y; Le Gouill, S; Mahe, B; Moreau, P; Peterlin, P; Tessoulin, B; Touzeau, C; Wuilleme, S, 2020
)
0.77
"Controlled studies are needed to confirm the clinical outcome value of twice-daily vs once-daily aspirin dosing and the therapeutic role of pegylated interferons and direct oral anticoagulants."( Polycythemia vera and essential thrombocythemia: 2021 update on diagnosis, risk-stratification and management.
Barbui, T; Tefferi, A, 2020
)
0.56
" Patient weight, age, glutathione-S-transferase A1 (GSTA1) genotype and busulfan dosing day/time were the most commonly identified factors affecting CL."( Review of the Pharmacokinetics and Pharmacodynamics of Intravenous Busulfan in Paediatric Patients.
Fraser, CJ; Hennig, S; Lawson, R; Staatz, CE, 2021
)
1.09
"Outcomes were compared in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome who received either myeloablative, fractionated busulfan (f-Bu) dosed to achieve an area under the curve of 20,000 μmol per minute (f-Bu20K) over 2 weeks (n = 84) or a standard, nonfractionated, lower busulfan dose regimen of 16,000 μmol per minute (Bu16K) over 4 days (n = 78)."( Fractionated busulfan myeloablative conditioning improves survival in older patients with acute myeloid leukemia and myelodysplastic syndrome.
Alousi, AM; Andersson, BS; Bashir, Q; Champlin, RE; Chen, J; Ciurea, SO; Hosing, C; Kebriaei, P; Marin, D; Mehta, RS; Olson, AL; Oran, B; Popat, UR; Rezvani, K; Saliba, RM; Shpall, EJ; Valdez, BC, 2021
)
1.19
" Here, in collaboration with the Dutch Association for Quality Assessment in Therapeutic Drug Monitoring and Clinical Toxicology, a novel interlaboratory proficiency program for the quantitation in plasma, pharmacokinetic modeling, and dosing of busulfan was designed."( Quality Control of Busulfan Plasma Quantitation, Modeling, and Dosing: An Interlaboratory Proficiency Testing Program.
Dupuis, LL; Franssen, EJF; Huitema, ADR; Kweekel, DM; McCune, JS; Punt, AM; Ritchie, JC; van Maarseveen, E; Yeh, RF, 2021
)
1.13
"To assess the ability of model-based personalised dosing tools to estimate busulfan exposure (i) in comparison to clinically used intensive sampling exposure estimation procedure, (ii) using limited sampling strategies and (iii) to predict changes in busulfan clearance during busulfan treatment."( Evaluation of two software using Bayesian methods for monitoring exposure and dosing once-daily intravenous busulfan in paediatric patients receiving haematopoietic stem cell transplantation.
Fraser, CJ; Hennig, S; Lawson, R; Paterson, L, 2021
)
1.06
"Data on intravenous busulfan dosing for patients with 4 consecutive days were entered into Bayesian forecasting software, InsightRX and NextDose."( Evaluation of two software using Bayesian methods for monitoring exposure and dosing once-daily intravenous busulfan in paediatric patients receiving haematopoietic stem cell transplantation.
Fraser, CJ; Hennig, S; Lawson, R; Paterson, L, 2021
)
1.16
" Reduction of busulfan clearance from day 1 to 4 of once daily dosing was confirmed and should be considered when adjusting doses."( Evaluation of two software using Bayesian methods for monitoring exposure and dosing once-daily intravenous busulfan in paediatric patients receiving haematopoietic stem cell transplantation.
Fraser, CJ; Hennig, S; Lawson, R; Paterson, L, 2021
)
1.19
" The mice were treated with busulfan and cyclophosphamide combined chemotherapy, and the appropriate dosage was determined by evaluating the myeloablative effect and drug toxicity."( Proliferation kinetics of immune cells during early phase of bone marrow transplantation in mouse model based on chemotherapy conditioning.
Chen, C; DA, Z; Fu, B; Li, X; Lu, Y; Zhou, J, 2021
)
0.92
" The use of PK-guided Bu dosing leads to improved overall survival (OS) and progression-free survival (PFS) compared with fixed-dose administration in a variety of hematologic diseases."( Feasibility and Efficacy of a Pharmacokinetics-Guided Busulfan Conditioning Regimen for Allogeneic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis in Adult Patients with Hematologic Malignancie
Bartoli, A; Bramanti, S; Castagna, L; De Gregori, S; De Philippis, C; Giordano, L; Mannina, D; Mariotti, J; Pieri, G; Roperti, M; Sarina, B; Valli, V, 2021
)
0.87
"25), if concomitantly administered drugs are dosed either 2 hours before or 8 hours after the 2-hour intravenous infusion of treosulfan."( Evaluation of the drug-drug interaction potential of treosulfan using a physiologically-based pharmacokinetic modelling approach.
Balazki, P; Baumgart, J; Beelen, DW; Böhm, S; Hemmelmann, C; Hilger, RA; Martins, FS; Ring, A; Schaller, S, 2022
)
0.72
" The 1-compartment open linear popPK model, which was built by Su-jin Rhee et al (model H), incorporating the patient's body surface area, age, dosing day, and aspartate aminotransferase as significant covariates had preferable predictability than other popPK models."( Can Published Population Pharmacokinetic Models of Busulfan Be Used for Individualized Dosing in Chinese Pediatric Patients Undergoing Hematopoietic Stem Cell Transplantation? An External Evaluation.
Chen, S; Hu, J; Huang, H; Huang, W; Li, D; Lin, S; Liu, M; Ren, J; Wu, X; Yang, T, 2022
)
0.97
" The aim of this study was to compare the pharmacokinetic parameters of Bu after oral dosing between patients receiving phenytoin and those receiving levetiracetam prophylaxis."( Decreased Systemic Busulfan Exposure After Oral Dosing With Concomitant Levetiracetam Compared With Phenytoin.
Artul, T; Efrati, E; Henig, I; Kurnik, D; Lurie, Y; Nassar, L; Scherb, I; Yehudai-Ofir, D; Zuckerman, T, 2022
)
1.05
" Body size-based dosing is used for most anticancer drugs used in infants."( Clinical pharmacology of cytotoxic drugs in neonates and infants: Providing evidence-based dosing guidance.
Barnett, S; Bérénos, IM; Carruthers, V; Huitema, ADR; Kong, J; Lalmohamed, A; Nijstad, AL; Parke, E; Tweddle, DA; Veal, GJ; Zwaan, CM, 2022
)
0.72
" We then compared the probability of cAUC within the range of 80 to 100 mg · h/L by the developed dosing regimen versus conventional regimen."( Busulfan dose Recommendation in Inherited Metabolic Disorders: Population Pharmacokinetic Analysis.
Gupta, AO; Illamola, SM; Jennissen, CA; Long, SE; Long-Boyle, JR; Lund, TC; Orchard, PJ; Takahashi, T, 2022
)
2.16
" Therefore, this assay could be implemented as a valid alternative for LC methods in clinical laboratories on different open-channel clinical chemistry analyzers, resulting in shorter turn-around times for reporting busulfan TDM results with subsequent faster dosage adjustments."( Automation in Busulfan Therapeutic Drug Monitoring: Evaluation of an Immunoassay on two Routine Chemistry Analyzers.
Oyaert, M; Stove, V; Verougstraete, N; Verstraete, AG, 2022
)
1.27
" Loboob at a dosage of 140mg/kg improved sperm viability."( Preserving effect of Loboob (a traditional multi-herbal formulation) on sperm parameters of male rats with busulfan-induced subfertility.
Badr, P; Bahmanpour, S; Dabbaghmanesh, MH; Jahromi, BN; Keshavarz, M; Koohpeyma, F; Najib, FS; Namazi, N; Noori, A; Poordast, T; Zare, N, 2022
)
0.93
" In conclusion, therapeutic drug monitoring (TDM) and individualization of Bu dosage are essential to improve the efficacy and safety of busulfan-based regimen in Chinese pediatric HSCT recipients."( Clinical outcomes of individualized busulfan-dosing in hematopoietic stem cell transplantation in Chinese children undergoing with therapeutic drug monitoring.
Cao, J; Feng, SQ; Hu, MZ; Hu, T; Li, JH; Li, JJ; Liu, R; Shao, DF; Shi, XD; Song, ZL; Tang, RH; Wang, XY; Xuan, LT; Yue, M; Zhai, MN; Zhang, HF; Zhang, L; Zhang, ZX; Zhong, DX, 2022
)
1.2
" Getting more insight into the association between busulfan exposure and the development of VOD/SOS enables further optimization of dosing and treatment strategies."( Association Between the Magnitude of Intravenous Busulfan Exposure and Development of Hepatic Veno-Occlusive Disease in Children and Young Adults Undergoing Myeloablative Allogeneic Hematopoietic Cell Transplantation.
Ansari, M; Bartelink, IH; Bittencourt, H; Boelens, JJ; Bognàr, T; Bredius, RGM; Chiesa, R; Cowan, MJ; Cuvelier, GDE; Dvorak, C; Egberts, TCG; Güngör, T; Hassan, M; Hempel, G; Kletzel, M; Krajinovic, M; Lalmohamed, A; Long-Boyle, JR; Lund, T; Marktel, S; Nath, CE; Orchard, PJ; Rademaker, CMA; Savic, RM; Shaw, PJ; Slatter, MA; Théoret, Y; Versluys, AB; Wynn, RF, 2022
)
1.23
" According to our single feature recommendation, following the busulfan dosage was the most effective for preventing VOD/SOS."( Prediction and recommendation by machine learning through repetitive internal validation for hepatic veno-occlusive disease/sinusoidal obstruction syndrome and early death after allogeneic hematopoietic cell transplantation.
Cho, BS; Cho, SG; Eom, KS; Hwang, HJ; Jung, J; Kim, HJ; Kim, YJ; Lee, E; Lee, JW; Lee, S; Lee, SE; Min, CK; Min, GJ; Park, MS; Park, S; Park, SS; Yoon, JH, 2022
)
0.96
" The efficacy and safety of both dosing strategies were compared using a systematic review and meta-analysis."( Fixed-dose administration and pharmacokinetically guided adjustment of busulfan dose for patients undergoing hematopoietic stem cell transplantation: a meta-analysis and cost-effectiveness analysis.
Chen, C; Chen, T; Guo, J; He, X; Liu, M; Zheng, B, 2022
)
0.95
" In 2019, we changed Bu dosing from 4×/day (Bu-4) to 1×/day (Bu-1) for ease of application."( Impact of busulfan pharmacokinetics on outcome in adult patients receiving an allogeneic hematopoietic cell transplantation.
Battegay, R; Halter, J; Heim, D; Medinger, M; Passweg, JR; Rentsch, KM; Seydoux, C, 2022
)
1.12
" Recipients with Bu(35 mg/kg)/Cy(100 mg/kg) therapy had brain injury, increased neuroinflammation, diminished neurogenesis and cognitive abnormalities, whereas animals given a lesser dosage had no such brain damages."( Effects of Chemotherapy on Neuroinflammation, Neuronal Damage, Neurogenesis, and Behavioral Performance in Bone Marrow Transplantation Recipient Mice.
Bu, XL; Fan, H; Liu, ZH; Tan, CR; Tu, YF; Wang, YJ; Xu, MY; Yu, ZY; Zeng, GH, 2022
)
0.72
"This study aimed to characterize the population pharmacokinetics (PK) of busulfan focusing on how busulfan clearance (CL) changes over time during once-daily administration and assess different methods for measuring busulfan exposure and the ability to achieve target cumulative exposure under different dosing adjustment scenarios in pediatric stem cell transplantation recipients."( Population pharmacokinetic model for once-daily intravenous busulfan in pediatric subjects describing time-associated clearance.
Fraser, CJ; Hennig, S; Lawson, R; Mitchell, R; O'Brien, T; Ramachandran, S; Staatz, CE; Teague, L, 2022
)
1.2
"An extended release dosage form based on encapsulating the challenging drug busulfan within microspheres of the biodegradable, biocompatible and biosynthesized poly(3-hydroxybutyrate-co-3-hydroxyvalerate) (PHBV) polyester was achieved."( Biosynthesized poly(3-hydroxybutyrate-co-3-hydroxyvalerate) as biocompatible microcapsules with extended release for busulfan and montelukast.
Aboulsoud, M; Alamry, KA; Alenazi, E; Alsafadi, D; Hussein, MA; Ibrahim, MI; Safi, E, 2022
)
1.16
" However, considering a wide variation in the effects of busulfan among animal species, its dosage and route of infusion need optimization to prepare effective and safe recipients."( Establishment of effective and safe recipient preparation for germ-cell transplantation with intra-testicular busulfan treatment in pre-pubertal Barbari goats.
Chauhan, MS; Kharche, SD; Pathak, M; Pawaiya, RVS; Quadri, SA; Singh, MK; Singh, SP; Soni, YK, 2022
)
1.18
" Here, we compared post-transplant outcomes after individualized versus fixed busulfan dosage in intermediate-risk AML who achieved CR prior to allograft focusing on pre-transplant flow-MRD."( Individualized busulfan dosing improves outcomes compared to fixed-dose administration in pre-transplant minimal residual disease-positive acute myeloid leukemia patients with intermediate-risk undergoing allogeneic stem cell transplantation in CR.
Ayuk, F; Bacher, U; Badbaran, A; Dadkhah, A; Freiberger, P; Janson, D; Klyuchnikov, E; Kröger, N; Langebrake, C; Massoud, R; Wolschke, C, 2023
)
1.49
" Model-informed dosing is recommended for patients under 2 years."( Evaluation of treosulfan cumulative exposure in paediatric patients through population pharmacokinetics and dosing simulations.
Chung, J; Fraser, C; Keogh, SJ; Kohli, S; Lee, S; McLachlan, AJ; Nath, CE; O'Brien, T; Rosser, SPA; Shaw, PJ, 2023
)
0.91
" We evaluated two myeloablative conditioning dosing ranges of intravenous (IV) busulfan (Bu) in combination with fludarabine in 70 patients."( Evaluation of different pharmacokinetically guided IV busulfan exposure ranges on adult patient outcomes after hematopoietic stem cell transplantation.
Ahmad, S; Edgar, CM; Guo, M; Mcvey, CP; Mori, S; Patel, RD; Rivera-Robles, N; Varela, JC; Yi, F, 2023
)
1.39
" More prospective clinical trials with a larger population are needed to validate the optimal dosing of LEV for BIS prophylaxis in patients undergoing HSCT."( Optimal regimen of levetiracetam for prevention of busulfan-induced seizure in patients undergoing hematopoietic stem cell transplantation: A review of available evidence.
Hadjibabaie, M; Honarmand, H; Kiumarsi, A; Rostami, T; Shahrami, B; Tavajohi, R, 2023
)
1.16
"To ensure effective busulfan exposure in pediatrics, different weight-based nomograms have been established to determine busulfan dosage and provided improved results (65-80% of patients correctly exposed)."( Individualizing busulfan dose in specific populations and evaluating the risk of pharmacokinetic drug-drug interactions.
Combarel, D; Delahousse, J; Paci, A; Tran, J; Vassal, G, 2023
)
1.58
" Study objective was to evaluate the effect of day 1 TDM-guided dosing (regimen d1) versus days 1 + 2 TDM-guided dosing (regimen d1 + 2) on attaining adequate busulfan exposure."( Busulfan target exposure attainment in children undergoing allogeneic hematopoietic cell transplantation: a single day versus a multiday therapeutic drug monitoring regimen.
Bartelink, IHI; Bognàr, TT; de Kanter, CTMK; Egberts, ACGT; Kingma, JSJ; Lalmohamed, AA; Lindemans, CAC; Smeijsters, EHE; van der Elst, KCMK, 2023
)
2.55
"Although exposure-directed busulfan (BU) dosing can improve allogeneic hematopoietic stem cell transplantation outcomes, there is still large variability in BU exposure with test dose alone due to changes in BU clearance caused by drug interactions."( Effects of combined test dose and therapeutic drug monitoring strategy in exposure-directed busulfan.
Akasaka, T; Anzai, N; Arai, Y; Arima, N; Iemura, T; Ikeda, T; Imada, K; Ishikawa, T; Itoh, M; Kanda, J; Kitano, T; Kitawaki, T; Kondo, T; Maeda, T; Matsumoto, K; Moriguchi, T; Nohgawa, M; Takaori-Kondo, A; Takeoka, T; Ueda, A; Ueda, Y; Watanabe, M; Yago, K; Yamashita, K; Yonezawa, A, 2023
)
1.43
" Our results indicate once-daily, intravenous busulfan PK in adult allo-HCT recipients receiving MAC dosing can be reasonably described by a popPK model, and the use of a sparse PK sampling strategy may be feasible for determining target exposure attainment following MAC dosing."( Utilization of a Population Pharmacokinetic Model to Improve a Busulfan Test-Dose Strategy in Allogeneic Hematopoietic Cell Transplant Recipients.
Armistead, PM; Crona, DJ; DeVane, SC; Dunlap, TC; Kardouh, M; Kemper, RM; Ptachcinski, JR; Shaw, JR; Symonds, A; Weiner, DL, 2023
)
1.41
" Model-informed precision dosing (MIPD) based on population pharmacokinetic (popPK) models has been implemented in the clinical settings."( Population Pharmacokinetic Model of Intravenous Busulfan in Hematopoietic Cell Transplantation: Systematic Review and Comparative Simulations.
Al-Kofahi, M; Brown, SJ; Jaber, MM; Takahashi, T, 2023
)
1.17
" As a result, dosing levels can vary significantly from patient to patient."( Liquid Chromatography-Tandem Mass Spectrometry Method for the Quantification of Plasma Busulfan.
Clinton Frazee, C; Garg, U; Munar, A, 2024
)
1.67
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (5)

RoleDescription
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.
insect sterilantA chemosterilant intended to sterilize insects.
antineoplastic agentA substance that inhibits or prevents the proliferation of neoplasms.
teratogenic agentA role played by a chemical compound in biological systems with adverse consequences in embryo developments, leading to birth defects, embryo death or altered development, growth retardation and functional defect.
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.
[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 (1)

ClassDescription
methanesulfonate esterAn organosulfonic ester resulting from the formal condensation of methanesulfonic acid with the hydroxy group of an alcohol, phenol, heteroarenol, or enol.
[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 (29)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Chain A, JmjC domain-containing histone demethylation protein 3AHomo sapiens (human)Potency89.12510.631035.7641100.0000AID504339
Chain A, 2-oxoglutarate OxygenaseHomo sapiens (human)Potency25.11890.177814.390939.8107AID2147
15-lipoxygenase, partialHomo sapiens (human)Potency31.62280.012610.691788.5700AID887
TDP1 proteinHomo sapiens (human)Potency24.84460.000811.382244.6684AID686978; AID686979
apical membrane antigen 1, AMA1Plasmodium falciparum 3D7Potency39.81070.707912.194339.8107AID720542
aldehyde dehydrogenase 1 family, member A1Homo sapiens (human)Potency39.81070.011212.4002100.0000AID1030
thyroid stimulating hormone receptorHomo sapiens (human)Potency39.81070.001318.074339.8107AID926
cytochrome P450 family 3 subfamily A polypeptide 4Homo sapiens (human)Potency5.49500.01237.983543.2770AID1645841
EWS/FLI fusion proteinHomo sapiens (human)Potency8.31640.001310.157742.8575AID1259253; AID1259256
glucocorticoid receptor [Homo sapiens]Homo sapiens (human)Potency3.54810.000214.376460.0339AID588532
retinoic acid nuclear receptor alpha variant 1Homo sapiens (human)Potency30.73140.003041.611522,387.1992AID1159552
retinoid X nuclear receptor alphaHomo sapiens (human)Potency20.66220.000817.505159.3239AID1159527; AID1159531; AID588544
estrogen-related nuclear receptor alphaHomo sapiens (human)Potency30.73140.001530.607315,848.9004AID1224841; AID1259401
pregnane X nuclear receptorHomo sapiens (human)Potency50.11870.005428.02631,258.9301AID720659
estrogen nuclear receptor alphaHomo sapiens (human)Potency9.00740.000229.305416,493.5996AID743079
aryl hydrocarbon receptorHomo sapiens (human)Potency36.55830.000723.06741,258.9301AID743085; AID743122
nuclear factor of kappa light polypeptide gene enhancer in B-cells 1 (p105), isoform CRA_aHomo sapiens (human)Potency0.000619.739145.978464.9432AID1159509
thyroid hormone receptor beta isoform 2Rattus norvegicus (Norway rat)Potency21.75610.000323.4451159.6830AID743065
ubiquitin carboxyl-terminal hydrolase 2 isoform aHomo sapiens (human)Potency15.84890.65619.452025.1189AID927
nuclear factor erythroid 2-related factor 2 isoform 1Homo sapiens (human)Potency74.97800.000627.21521,122.0200AID651741
gemininHomo sapiens (human)Potency0.56230.004611.374133.4983AID624297
lethal factor (plasmid)Bacillus anthracis str. A2012Potency25.11890.020010.786931.6228AID912
lamin isoform A-delta10Homo sapiens (human)Potency0.79430.891312.067628.1838AID1487
Disintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)Potency15.84891.584913.004325.1189AID927
[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)422.66670.11007.190310.0000AID1443980; AID1449628; AID1473738
Matrix metalloproteinase-9Homo sapiens (human)IC50 (µMol)5.21500.00000.705310.0000AID625178
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 (108)

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)
skeletal system developmentMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of protein phosphorylationMatrix metalloproteinase-9Homo sapiens (human)
proteolysisMatrix metalloproteinase-9Homo sapiens (human)
apoptotic processMatrix metalloproteinase-9Homo sapiens (human)
embryo implantationMatrix metalloproteinase-9Homo sapiens (human)
cell migrationMatrix metalloproteinase-9Homo sapiens (human)
extracellular matrix disassemblyMatrix metalloproteinase-9Homo sapiens (human)
macrophage differentiationMatrix metalloproteinase-9Homo sapiens (human)
collagen catabolic processMatrix metalloproteinase-9Homo sapiens (human)
cellular response to reactive oxygen speciesMatrix metalloproteinase-9Homo sapiens (human)
endodermal cell differentiationMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of apoptotic processMatrix metalloproteinase-9Homo sapiens (human)
negative regulation of apoptotic processMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of DNA bindingMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of epidermal growth factor receptor signaling pathwayMatrix metalloproteinase-9Homo sapiens (human)
ephrin receptor signaling pathwayMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of keratinocyte migrationMatrix metalloproteinase-9Homo sapiens (human)
cellular response to lipopolysaccharideMatrix metalloproteinase-9Homo sapiens (human)
cellular response to cadmium ionMatrix metalloproteinase-9Homo sapiens (human)
cellular response to UV-AMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of release of cytochrome c from mitochondriaMatrix metalloproteinase-9Homo sapiens (human)
regulation of neuroinflammatory responseMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of receptor bindingMatrix metalloproteinase-9Homo sapiens (human)
response to amyloid-betaMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of vascular associated smooth muscle cell proliferationMatrix metalloproteinase-9Homo sapiens (human)
negative regulation of epithelial cell differentiation involved in kidney developmentMatrix metalloproteinase-9Homo sapiens (human)
negative regulation of intrinsic apoptotic signaling pathwayMatrix metalloproteinase-9Homo sapiens (human)
negative regulation of cation channel activityMatrix metalloproteinase-9Homo sapiens (human)
negative regulation of cysteine-type endopeptidase activity involved in apoptotic signaling pathwayMatrix metalloproteinase-9Homo sapiens (human)
extracellular matrix organizationMatrix metalloproteinase-9Homo sapiens (human)
positive regulation of epidermal growth factor receptor signaling pathwayDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
response to hypoxiaDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
neutrophil mediated immunityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
germinal center formationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of leukocyte chemotaxisDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
proteolysisDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
membrane protein ectodomain proteolysisDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cell adhesionDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
Notch receptor processingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of cell population proliferationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
response to xenobiotic stimulusDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of T cell chemotaxisDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
protein processingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
signal releaseDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
B cell differentiationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of cell growthDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of cell migrationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
response to lipopolysaccharideDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of chemokine productionDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of tumor necrosis factor productionDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
regulation of mast cell apoptotic processDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
T cell differentiation in thymusDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cell adhesion mediated by integrinDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
wound healing, spreading of epidermal cellsDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
amyloid precursor protein catabolic processDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of blood vessel endothelial cell migrationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of cyclin-dependent protein serine/threonine kinase activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of epidermal growth factor-activated receptor activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of epidermal growth factor receptor signaling pathwayDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
spleen developmentDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cell motilityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
defense response to Gram-positive bacteriumDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cellular response to high density lipoprotein particle stimulusDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
commissural neuron axon guidanceDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
negative regulation of cold-induced thermogenesisDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of G1/S transition of mitotic cell cycleDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of tumor necrosis factor-mediated signaling pathwayDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
positive regulation of vascular endothelial cell proliferationDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
Notch signaling pathwayDisintegrin and metalloproteinase domain-containing protein 17Homo 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)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (41)

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)
endopeptidase activityMatrix metalloproteinase-9Homo sapiens (human)
metalloendopeptidase activityMatrix metalloproteinase-9Homo sapiens (human)
serine-type endopeptidase activityMatrix metalloproteinase-9Homo sapiens (human)
protein bindingMatrix metalloproteinase-9Homo sapiens (human)
collagen bindingMatrix metalloproteinase-9Homo sapiens (human)
peptidase activityMatrix metalloproteinase-9Homo sapiens (human)
metallopeptidase activityMatrix metalloproteinase-9Homo sapiens (human)
zinc ion bindingMatrix metalloproteinase-9Homo sapiens (human)
identical protein bindingMatrix metalloproteinase-9Homo sapiens (human)
endopeptidase activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
metalloendopeptidase activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
Notch bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
interleukin-6 receptor bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
integrin bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
protein bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
peptidase activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
metallopeptidase activityDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
SH3 domain bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cytokine bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
PDZ domain bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
tumor necrosis factor bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
metal ion bindingDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
metalloendopeptidase activity involved in amyloid precursor protein catabolic processDisintegrin and metalloproteinase domain-containing protein 17Homo 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)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (30)

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 regionMatrix metalloproteinase-9Homo sapiens (human)
extracellular spaceMatrix metalloproteinase-9Homo sapiens (human)
collagen-containing extracellular matrixMatrix metalloproteinase-9Homo sapiens (human)
extracellular exosomeMatrix metalloproteinase-9Homo sapiens (human)
tertiary granule lumenMatrix metalloproteinase-9Homo sapiens (human)
ficolin-1-rich granule lumenMatrix metalloproteinase-9Homo sapiens (human)
extracellular spaceMatrix metalloproteinase-9Homo sapiens (human)
cell-cell junctionDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
focal adhesionDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
ruffle membraneDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
Golgi membraneDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cytoplasmDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
endoplasmic reticulum lumenDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cytosolDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
plasma membraneDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
cell surfaceDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
actin cytoskeletonDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
membraneDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
apical plasma membraneDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
membrane raftDisintegrin and metalloproteinase domain-containing protein 17Homo sapiens (human)
plasma membraneDisintegrin and metalloproteinase domain-containing protein 17Homo 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)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (176)

Assay IDTitleYearJournalArticle
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.
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.
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
AID444054Oral bioavailability in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
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.
AID1079943Malignant tumor, proven histopathologically. Value is number of references indexed. [column 'T.MAL' in source]
AID444058Volume of distribution at steady state in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID396072Induction of apoptosis in imatinib mesylate-resistant human K562 cells after 48 hrs2008European journal of medicinal chemistry, Nov, Volume: 43, Issue:11
Synthesis and induction of G0-G1 phase arrest with apoptosis of 3,5-dimethyl-6-phenyl-8-(trifluoromethyl)-5,6-dihydropyrazolo[3,4-f][1,2,3,5]tetrazepin-4(3H)-one.
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.
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.
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.
AID1152955Antiproliferative activity against human SiMa cells at 10 to 100 uM after 72 hrs by MTT assay2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID396071Induction of apoptosis in human K562 cells after 48 hrs2008European journal of medicinal chemistry, Nov, Volume: 43, Issue:11
Synthesis and induction of G0-G1 phase arrest with apoptosis of 3,5-dimethyl-6-phenyl-8-(trifluoromethyl)-5,6-dihydropyrazolo[3,4-f][1,2,3,5]tetrazepin-4(3H)-one.
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.
AID1079945Animal toxicity known. [column 'TOXIC' in source]
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]
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.
AID977599Inhibition of sodium fluorescein uptake in OATP1B1-transfected CHO cells at an equimolar substrate-inhibitor concentration of 10 uM2013Molecular pharmacology, Jun, Volume: 83, Issue:6
Structure-based identification of OATP1B1/3 inhibitors.
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.
AID216591In vitro cytotoxicity causing 50% growth inhibition was determined against WiDr (human colon carcinoma) cell line2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
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.
AID678713Inhibition of human CYP2C9 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using 7-methoxy-4-trifluoromethylcoumarin-3-acetic acid as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
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]
AID1079939Cirrhosis, proven histopathologically. Value is number of references indexed. [column 'CIRRH' in source]
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.
AID1636356Drug activation in human Hep3B cells assessed as human CYP2C9-mediated drug metabolism-induced cytotoxicity measured as decrease in cell viability at 300 uM pre-incubated with BSO for 18 hrs followed by incubation with compound for 3 hrs in presence of NA2016Bioorganic & medicinal chemistry letters, 08-15, Volume: 26, Issue:16
Development of a cell viability assay to assess drug metabolite structure-toxicity relationships.
AID1636357Drug activation in human Hep3B cells assessed as human CYP3A4-mediated drug metabolism-induced cytotoxicity measured as decrease in cell viability at 300 uM pre-incubated with BSO for 18 hrs followed by incubation with compound for 3 hrs in presence of NA2016Bioorganic & medicinal chemistry letters, 08-15, Volume: 26, Issue:16
Development of a cell viability assay to assess drug metabolite structure-toxicity relationships.
AID123389Hematological effects in female CDF1 mice at the dose of 30 mg, in platelet cells2000Journal of medicinal chemistry, Apr-20, Volume: 43, Issue:8
Synthesis and preliminary biological evaluations of CC-1065 analogues: effects of different linkers and terminal amides on biological activity.
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.
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.
AID1152954Antiproliferative activity against human Kelly cells at 10 to 100 uM after 72 hrs by MTT assay2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID444056Fraction escaping gut-wall elimination in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
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).
AID444055Fraction absorbed in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID1649880Growth inhibition of human DU145 cells after 5 days by SRB assay2020Journal of medicinal chemistry, 06-11, Volume: 63, Issue:11
Design and Synthesis of a Trifunctional Molecular System "Programmed" to Block Epidermal Growth Factor Receptor Tyrosine Kinase, Induce High Levels of DNA Damage, and Inhibit the DNA Repair Enzyme (Poly(ADP-ribose) Polymerase) in Prostate Cancer Cells.
AID678714Inhibition of human CYP2C19 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using 3-butyryl-7-methoxycoumarin as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
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.
AID215102In vitro cytotoxicity causing 50% growth inhibition was determined against 22B (human head and neck squamous) cell line2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
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.
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.
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).
AID1649878Growth inhibition of human 22Rv1 cells after 5 days by SRB assay2020Journal of medicinal chemistry, 06-11, Volume: 63, Issue:11
Design and Synthesis of a Trifunctional Molecular System "Programmed" to Block Epidermal Growth Factor Receptor Tyrosine Kinase, Induce High Levels of DNA Damage, and Inhibit the DNA Repair Enzyme (Poly(ADP-ribose) Polymerase) in Prostate Cancer Cells.
AID124034Hematological effects in female CDF1 mice at the dose of 30 mg, in white blood cells2000Journal of medicinal chemistry, Apr-20, Volume: 43, Issue:8
Synthesis and preliminary biological evaluations of CC-1065 analogues: effects of different linkers and terminal amides on biological activity.
AID625276FDA Liver Toxicity Knowledge Base Benchmark Dataset (LTKB-BD) drugs of most concern for DILI2011Drug discovery today, Aug, Volume: 16, Issue:15-16
FDA-approved drug labeling for the study of drug-induced liver injury.
AID80664In vitro cytotoxicity causing 50% growth inhibition was determined against H322 (human non small cell lung cancer) cells2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
AID425652Total body clearance in human2009Journal of medicinal chemistry, Aug-13, Volume: 52, Issue:15
Physicochemical determinants of human renal clearance.
AID678712Inhibition of human CYP1A2 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using ethoxyresorufin as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
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.
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).
AID588209Literature-mined public compounds from Greene et al multi-species hepatotoxicity modelling dataset2010Chemical research in toxicology, Jul-19, Volume: 23, Issue:7
Developing structure-activity relationships for the prediction of hepatotoxicity.
AID1079946Presence of at least one case with successful reintroduction. [column 'REINT' in source]
AID1079932Highest frequency of moderate liver toxicity observed during clinical trials, expressed as a percentage. [column '% BIOL' in source]
AID1152959Increase in glycolysis in human Kelly cells assessed as conversion of glucose to lactate at 100 uM after 12 hrs relative to untreated control2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID1443980Inhibition of human BSEP expressed in fall armyworm sf9 cell plasma membrane vesicles assessed as reduction in vesicle-associated [3H]-taurocholate transport preincubated for 10 mins prior to ATP addition measured after 15 mins in presence of [3H]-tauroch2010Toxicological sciences : an official journal of the Society of Toxicology, Dec, Volume: 118, Issue:2
Interference with bile salt export pump function is a susceptibility factor for human liver injury in drug development.
AID1079948Times to onset, minimal and maximal, observed in the indexed observations. [column 'DELAI' in source]
AID1079940Granulomatous liver disease, proven histopathologically. Value is number of references indexed. [column 'GRAN' in source]
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).
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.
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.
AID1152956Antiproliferative activity against human SK-N-SH cells at 10 to 100 uM after 72 hrs by MTT assay2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
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).
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).
AID1079934Highest frequency of acute liver toxicity observed during clinical trials, expressed as a percentage. [column '% AIGUE' in source]
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).
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.
AID9816In vitro cytotoxicity causing 50% growth inhibition was determined against A270 (human ovarian cancer)cell line2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
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).
AID1079947Comments (NB not yet translated). [column 'COMMENTAIRES' in source]
AID1152957Increase in glycolysis in human LS cells assessed as conversion of glucose to lactate at 100 uM after 12 hrs relative to untreated control2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID444057Fraction escaping hepatic elimination in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID1636440Drug activation in human Hep3B cells assessed as human CYP2D6-mediated drug metabolism-induced cytotoxicity measured as decrease in cell viability at 300 uM pre-incubated with BSO for 18 hrs followed by incubation with compound for 3 hrs in presence of NA2016Bioorganic & medicinal chemistry letters, 08-15, Volume: 26, Issue:16
Development of a cell viability assay to assess drug metabolite structure-toxicity relationships.
AID444051Total clearance in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID1079944Benign tumor, proven histopathologically. Value is number of references indexed. [column 'T.BEN' in source]
AID588210Human drug-induced liver injury (DILI) modelling dataset from Ekins et al2010Drug metabolism and disposition: the biological fate of chemicals, Dec, Volume: 38, Issue:12
A predictive ligand-based Bayesian model for human drug-induced liver injury.
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.
AID1079949Proposed mechanism(s) of liver damage. [column 'MEC' in source]
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).
AID43174In vitro cytotoxicity causing 50% growth inhibition was determined against C26-10 (murine colon carcinoma) cell line2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
AID1152958Increase in glycolysis in human SiMa cells assessed as conversion of glucose to lactate at 100 uM after 12 hrs relative to untreated control2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID977602Inhibition of sodium fluorescein uptake in OATP1B3-transfected CHO cells at an equimolar substrate-inhibitor concentration of 10 uM2013Molecular pharmacology, Jun, Volume: 83, Issue:6
Structure-based identification of OATP1B1/3 inhibitors.
AID1079942Steatosis, proven histopathologically. Value is number of references indexed. [column 'STEAT' in source]
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).
AID678715Inhibition of human CYP2D6 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using 4-methylaminoethyl-7-methoxycoumarin as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
AID678716Inhibition of human CYP3A4 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using diethoxyfluorescein as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
AID444053Renal clearance in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
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).
AID1079937Severe hepatitis, defined as possibly life-threatening liver failure or through clinical observations. Value is number of references indexed. [column 'MASS' in source]
AID1152953Antiproliferative activity against human LS cells at 10 to 100 uM after 72 hrs by MTT assay2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
AID311524Oral bioavailability in human2007Bioorganic & medicinal chemistry, Dec-15, Volume: 15, Issue:24
Hologram QSAR model for the prediction of human oral bioavailability.
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.
AID444052Hepatic clearance in human2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID425653Renal clearance in human2009Journal of medicinal chemistry, Aug-13, Volume: 52, Issue:15
Physicochemical determinants of human renal clearance.
AID444050Fraction unbound in human plasma2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Physicochemical space for optimum oral bioavailability: contribution of human intestinal absorption and first-pass elimination.
AID100106In vitro cytotoxicity causing 50% growth inhibition was determined against LL (murine non small cell lung cancer) cell line2001Bioorganic & medicinal chemistry letters, Mar-26, Volume: 11, Issue:6
Synthesis and in vitro cytotoxicity of novel long chain busulphan analogues.
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.
AID678722Covalent binding affinity to human liver microsomes assessed per mg of protein at 10 uM after 60 mins presence of NADPH2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
AID119060Hematological effects in female CDF1 mice at the dose of 30mg, in red blood cells2000Journal of medicinal chemistry, Apr-20, Volume: 43, Issue:8
Synthesis and preliminary biological evaluations of CC-1065 analogues: effects of different linkers and terminal amides on biological activity.
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).
AID1649879Growth inhibition of human PC3 cells after 5 days by SRB assay2020Journal of medicinal chemistry, 06-11, Volume: 63, Issue:11
Design and Synthesis of a Trifunctional Molecular System "Programmed" to Block Epidermal Growth Factor Receptor Tyrosine Kinase, Induce High Levels of DNA Damage, and Inhibit the DNA Repair Enzyme (Poly(ADP-ribose) Polymerase) in Prostate Cancer Cells.
AID115426Mean corpuscular hemoglobin concentration (MCHC)2000Journal of medicinal chemistry, Apr-20, Volume: 43, Issue:8
Synthesis and preliminary biological evaluations of CC-1065 analogues: effects of different linkers and terminal amides on biological activity.
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).
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).
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]
AID678717Inhibition of human CYP3A4 assessed as ratio of IC50 in absence of NADPH to IC50 for presence of NADPH using 7-benzyloxyquinoline as substrate after 30 mins2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
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]
AID678718Metabolic stability in human liver microsomes assessed as high signal/noise ratio (S/N of >100) by measuring GSH adduct formation at 100 uM after 90 mins by HPLC-MS analysis2012Chemical research in toxicology, Oct-15, Volume: 25, Issue:10
Preclinical strategy to reduce clinical hepatotoxicity using in vitro bioactivation data for >200 compounds.
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.
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.
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]
AID1152948Cellular uptake in human SK-N-SH cells assessed as noradrenaline transporter-mediated uptake at 1 x 10'-4 M after 15 mins by [3H]-noradrenaline uptake assay2014Bioorganic & medicinal chemistry letters, Jun-15, Volume: 24, Issue:12
Synthesis and biological effects of new hybrid compounds composed of benzylguanidines and the alkylating group of busulfan on neuroblastoma cells.
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.
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.
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.
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.
AID1745845Primary qHTS for Inhibitors of ATXN expression
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
AID540299A screen for compounds that inhibit the MenB enzyme of Mycobacterium tuberculosis2010Bioorganic & medicinal chemistry letters, Nov-01, Volume: 20, Issue:21
Synthesis and SAR studies of 1,4-benzoxazine MenB inhibitors: novel antibacterial agents against Mycobacterium tuberculosis.
AID588519A screen for compounds that inhibit viral RNA polymerase binding and polymerization activities2011Antiviral research, Sep, Volume: 91, Issue:3
High-throughput screening identification of poliovirus RNA-dependent RNA polymerase inhibitors.
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.
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.
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.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (4,772)

TimeframeStudies, This Drug (%)All Drugs %
pre-19901734 (36.34)18.7374
1990's688 (14.42)18.2507
2000's833 (17.46)29.6817
2010's1067 (22.36)24.3611
2020's450 (9.43)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 63.27

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 Index63.27 (24.57)
Research Supply Index8.63 (2.92)
Research Growth Index4.60 (4.65)
Search Engine Demand Index112.98 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (63.27)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials645 (12.99%)5.53%
Reviews303 (6.10%)6.00%
Case Studies488 (9.82%)4.05%
Observational18 (0.36%)0.25%
Other3,513 (70.73%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (534)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
CD34+Stem Cell Selection for Patients Receiving Partially Matched Family or Matched Unrelated Adult Donor Allogeneic Stem Cell Transplantations for Malignant and Non-Malignant Disease [NCT01049854]Phase 220 participants (Actual)Interventional2011-09-30Completed
Imatinib Mesylate, Busulfan, Fludarabine, Antithymocyte Globulin and Allogeneic Stem Cell Transplantation for Chronic Myelogenous Leukemia [NCT00499889]Phase 242 participants (Actual)Interventional2003-02-28Terminated(stopped due to Support issue.)
A Pilot Study of Intravenous, Targeted-Dose Busulfan Monotherapy as Conditioning for Autologous Hematopoietic Progenitor Cell Transplantation in High-Risk AML [NCT00363467]3 participants (Actual)Interventional2006-05-31Terminated(stopped due to Low accrual)
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
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
Allogeneic Hematopoietic Cell Transplantation for Patients With Non-Malignant Disorders Using Treosulfan, Fludarabine, and Thiotepa [NCT03980769]Phase 240 participants (Anticipated)Interventional2021-05-05Recruiting
Phase II Study of Reduced-Intensity Allogeneic Stem Cell Transplant for High-Risk Chronic Lymphocytic Leukemia (CLL) [NCT01027000]Phase 268 participants (Actual)Interventional2010-02-28Completed
A Phase I Clinical Trial for the Treatment of ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene [NCT01639690]Phase 110 participants (Anticipated)Interventional2012-07-31Active, not recruiting
Haplo-identical Hematopoietic Stem Cell Transplantation Following Reduced-intensity Conditioning in Children With Neuroblastoma [NCT01156350]Phase 210 participants (Anticipated)Interventional2011-09-30Not yet recruiting
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
Dose-reduced Versus Standard Conditioning Followed by Allogeneic Stem Cell Transplantation in Patients With MDS or sAML: A Randomised Phase III Study (RICMAC) [NCT01203228]Phase 3129 participants (Actual)Interventional2004-05-31Terminated
Hematopoietic Stem Cell Transplantation Using Bone Marrow Or Peripheral Blood Stem Cells From Matched, Unrelated, Volunteer Donors [NCT00054327]Phase 234 participants (Actual)Interventional2000-11-30Completed
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
High-dose Busulfan, High-dose Cyclophosphamide, and Allogeneic Bone Marrow Transplantation for Leukemia, Myelodysplastic Syndromes, Multiple Myeloma and Lymphoma [NCT01177371]Phase 213 participants (Actual)Interventional1988-03-31Completed
Related and Unrelated Donor Hematopoietic Stem Cell Transplant for DOCK8 Deficiency [NCT01176006]Phase 290 participants (Anticipated)Interventional2010-10-05Recruiting
[NCT01338675]Phase 1/Phase 25 participants (Anticipated)Interventional2011-01-31Recruiting
Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With Elongation Factor 1 Alpha Shortened (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency [NCT02999984]Phase 1/Phase 210 participants (Actual)Interventional2016-12-16Completed
Vorinostat With Gemcitabine/Clofarabine/Busulfan for Allogeneic Transplantation for Aggressive Lymphomas [NCT04220008]Phase 230 participants (Anticipated)Interventional2023-06-01Not yet recruiting
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.)
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
A Pilot Study of Myeloablative Allogeneic or Haploidentical Stem Cell Transplantation With High Dose PT-Cy in Relapsed/Refractory AML [NCT02057770]Phase 125 participants (Actual)Interventional2014-02-28Terminated(stopped due to Low accrual rate)
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
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 Randomized Phase II Study to Compare ATG or Post-Transplant Cyclophosphamide to Calcineurin Inhibitor-Methotrexate as GVHD Prophylaxis After Myeloablative Unrelated Donor Peripheral Blood Stem Cell Transplantation [NCT03602898]Phase 20 participants (Actual)Interventional2021-06-01Withdrawn(stopped due to Insufficient funding)
Phase I Trial of Escalated Doses of Targeted Marrow Irradiation (TMI) Combined With Fludarabine and Busulfan as Conditioning Regimen for Allogeneic Hematopoietic Progenitor Cell Transplantation [NCT02129582]Phase 114 participants (Actual)Interventional2014-11-05Completed
Modified TBF Regimen as Conditioning Regimen Prior to Allogeneic Hematopoietic Cell Transplantation for T Cell Acute Lymphoblastic Leukemia/Lymphoblastic Lymphoma:Phase II Study [NCT05598593]Phase 270 participants (Anticipated)Interventional2022-10-23Recruiting
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis [NCT01087398]Phase 2/Phase 310 participants (Anticipated)Interventional2009-09-30Recruiting
A Phase I Open Label Study of IV Busulfan (Busulfex®) in Multiple Myeloma Patients 65 Years of Age or Older, or With Renal Insufficiency Undergoing Autologous Transplantation [NCT00934232]Phase 113 participants (Actual)Interventional2009-08-31Terminated(stopped due to PI left the institution)
Total Body Irradiation/ Fludarabine/ Busulfan/ Cyclophosphamide (TFBC) Combined With Umbilical Cord Blood Transplantation (UCBT) in the Treatment of High-risk Malignant Hematological Diseases [NCT05929092]40 participants (Anticipated)Interventional2023-06-01Recruiting
A Phase II Study of Intensity Modulated Total Marrow Irradiation (IM-TMI) in Addition to Fludarabine/Busulfan Conditioning for Allogeneic Transplantation in High Risk AML and Myelodysplastic Syndromes [NCT03121014]Phase 238 participants (Anticipated)Interventional2017-04-24Recruiting
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
Allogeneic Transplantation Using Venetoclax, Timed Sequential Busulfan,Cladribine, and Fludarabine Conditioning in Patients With AML and MDS [NCT02250937]Phase 2116 participants (Anticipated)Interventional2014-10-27Active, 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
Ruxolitinib in Combination With High Dose Therapy and Autologous Stem Cell Transplantation for Myelofibrosis [NCT02469974]0 participants (Actual)Interventional2015-05-31Withdrawn(stopped due to no enrollments)
Phase I/II Study Using Prophylactic Donor Lymphocyte Infusion Early Post-Transplant After Allogeneic Hematopoietic Cell Transplantation Using Post-Transplantation Cyclophosphamide for High-Risk Hematologic Malignancies [NCT05327023]Phase 1/Phase 2430 participants (Anticipated)Interventional2022-05-23Recruiting
Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases [NCT04965597]Phase 240 participants (Anticipated)Interventional2022-04-19Recruiting
CBA Versus FBA Conditioning Followed by Allogeneic HSCT in Treatment of High Risk and Refractory AML [NCT03384212]Phase 3120 participants (Anticipated)Interventional2016-08-01Recruiting
Busulfan+ Cyclophosphamide+ Etoposide vs Busulfan+ Cyclophosphamide Conditioning Regimen for Diffuse Large B-cell Lymphoma Undergoing Autologous Hematopoietic Stem Cell Transplantation [NCT03229616]Phase 2/Phase 3122 participants (Anticipated)Interventional2017-07-05Recruiting
A Pilot Study to Evaluate the Feasibility, Safety and Engraftment of Zinc Finger Nuclease (ZFN) CCR5 Modified CD34+ Hematopoietic Stem/Progenitor Cells (SB-728mR-HSPC) in HIV-1 (R5) Infected Patients [NCT02500849]Phase 112 participants (Anticipated)Interventional2016-03-10Active, not recruiting
Autologous Bone Marrow Transplantation for Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndrome - A Phase II Pilot Study [NCT00004899]Phase 20 participants Interventional1999-10-31Completed
Decitabine+ Fludarabine+Busulfan Conditioning Regimen for Elderly Acute Myeloid Leukemia in Complete Remission Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT03530085]Phase 2/Phase 360 participants (Anticipated)Interventional2018-06-15Recruiting
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
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 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
Treatment Plan for Hematologic Malignancies Using Intravenous Busulfan and Cyclophosphamide Instead of Total Boby Irradiation (TBI) and Cyclophosphamide to Examine Results, Success and Side Effects of Treatment With Chemotherapy Only, as a Preparative The [NCT01339988]Phase 410 participants (Anticipated)Interventional2011-06-30Not yet recruiting
Feasibility Study on Allogeneic Hematopoietic Stem Cell Transplantation Following Fludarabine-Busulfan-ALS-based Reduced-intensity Conditioning in Children With Hematological Malignancy or Solid Tumor Not Responding to Standard Therapy or for Which the In [NCT00750126]Phase 230 participants (Anticipated)Interventional2007-04-30Recruiting
A 5 Day Course of Fludarabine and Cytarabine Followed by Full Intensity Allogeneic Stem Cell Transplantation (FA5-Bucy) in Treating Patients With High-risk, Recurrent or Refractory Acute Leukemia and Advanced Myelodysplastic Syndrome [NCT02328950]50 participants (Anticipated)Observational2014-12-31Recruiting
Chidamide Combined With Cladribine/Gemcitabine/Busulfan (ChiCGB) With Autologous Stem-Cell Transplantation in High-risk Hodgkin and Non-Hodgkin Lymphoma [NCT03602131]Phase 230 participants (Anticipated)Interventional2019-01-01Not yet recruiting
A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRαβ+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediat [NCT03619551]Phase 264 participants (Anticipated)Interventional2018-10-22Recruiting
Randomized Study Comparing i.v. Busulfan (Busilvex®) Plus Fludarabine (BuFlu) Versus Busilvex® Plus Cyclophosphamide (BuCy2) as Conditioning Regimens Prior AlloHSCT in Patients (Age >= 40 and =<65 Years) With AML in Complete Remission. [NCT01191957]Phase 3252 participants (Actual)Interventional2008-01-31Completed
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders [NCT01050855]Phase 275 participants (Anticipated)Interventional2008-01-31Recruiting
A Randomized Placebo-controlled Phase II Trial of Irradiated, Adenovirus Vector Transferred GM-CSF Secreting Autologous Leukemia Cell Vaccination (GVAX) Versus Placebo Vaccination in Patients With Advanced MDS/AML After Allogeneic Hematopoietic Stem Cell [NCT01773395]Phase 2123 participants (Actual)Interventional2013-01-08Terminated(stopped due to Recommendation by the Data and Safety Monitoring Board due to efficacy concerns)
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
Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency [NCT03765632]Phase 1/Phase 213 participants (Actual)Interventional2018-01-03Completed
High-dose Post-transplantation Cyclophosphamide as Graft Versus-host Disease Prophylaxis After Allogeneic Hematopoietic Stem Cell Transplantation [NCT02294552]Phase 2200 participants (Actual)Interventional2014-10-31Completed
Outcomes of Patients After Allogenic Hematopoietic Cell Transplantation With Decitabine-containing Conditioning Regimen and Acetylcysteine Treatment [NCT04945096]Phase 3100 participants (Anticipated)Interventional2021-07-01Not yet recruiting
Multi-Institutional Prospective Pilot Study of Lupron to Enhance Lymphocyte Immune Reconstitution Following Allogeneic Bone Marrow Transplantation in Post-Pubertal Children and Adults With Molecular Imaging Evaluation [NCT01338987]Phase 276 participants (Actual)Interventional2011-04-19Completed
A Phase 1/2 Trial of Uproleselan Combined With High Dose Busulfan Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for Patients With Chemotherapy Resistant Acute Myeloid Leukemia [NCT05569512]Phase 1/Phase 228 participants (Anticipated)Interventional2022-10-06Recruiting
Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa [NCT00478244]7 participants (Actual)Interventional2007-04-30Terminated(stopped due to Competing studies)
Phase II Study of High-Dose Busulfan and Cyclophosphamide Followed by Allogeneic Bone Marrow Transplantation for Patients With Acute Myelogenous Leukemia [NCT00004896]Phase 20 participants Interventional1999-10-31Completed
UARK 2003-25: A Phase I/II Open Label Study of IV Busulfan (Busulfex®) in Multiple Myeloma Patients Older Than 65 Years of Age or With Renal Insufficiency [NCT00113919]Phase 1/Phase 214 participants (Actual)Interventional2004-06-30Terminated(stopped due to Due to poor accrual)
Busulfan Plus Clofarabine Followed by Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Acute Lymphoblastic Leukemia or Lymphoma, or Biphenotypic Leukemia [NCT00990249]Phase 2120 participants (Actual)Interventional2009-10-31Completed
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
Matched Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplantation With a Conditioning Regimen of Targeted Busulfan, Cyclophosphamide, and Thymoglobulin [NCT00611351]Phase 25 participants (Actual)Interventional2005-06-07Completed
Fludarabine/Clofarabine/Busulfan Combined With SAHA in Patients Receiving Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia [NCT02083250]Phase 170 participants (Actual)Interventional2014-03-06Completed
A Phase 1 Study of Adding Venetoclax to a Reduced Intensity Conditioning Regimen and to Maintenance in Combination With a Hypomethylating Agent After Allogeneic Hematopoietic Cell Transplantation for Patients With High Risk AML, MDS, and MDS/MPN Overlap S [NCT03613532]Phase 178 participants (Anticipated)Interventional2018-10-24Recruiting
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
Itacitinib to Prevent Graft Versus Host Disease [NCT04859946]Phase 130 participants (Anticipated)Interventional2022-01-11Recruiting
A Phase 1/2, Open-Label, Multicenter, Single-Arm Study to Assess the Safety, Tolerability, and Efficacy of BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease [NCT03653247]Phase 1/Phase 28 participants (Anticipated)Interventional2019-03-06Active, not recruiting
Treatment of Acute Myelogenous Leukemia With Busulfan and Etoposide Followed by Autologous or Syngeneic Stem Cell Rescue and Low-Dose Interleukin 2 (IL-2) Immunotherapy [NCT00003875]Phase 230 participants (Actual)Interventional1998-10-13Completed
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
Umbilical Cord Blood Transplant for Congenital Pediatric Disorders [NCT00950846]40 participants (Actual)Interventional2009-09-30Completed
A Randomized Study of Once Daily Fludarabine-Clofarabine Versus Fludarabine Alone Combined With Intervenous Busulfan Followed by Allogeneic Hemopoietic Stem Cell Transplantation for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS) [NCT01471444]Phase 3256 participants (Actual)Interventional2011-11-02Completed
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
Sequential and Personalized Pharmacokinetic-guided Busulfan Administration in the Frame of the Conditiong Regimen for Allogeneic Haematopoietic Stem Cell Transplantation in Patients With Malignant Hemopathies Ineligible for the Standard Myeloablative Cond [NCT04451200]Phase 282 participants (Anticipated)Interventional2020-11-30Not yet recruiting
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
Busulfan+ Cyclophosphamide+ Etoposide Conditioning Regimen for Primary Central Nervous System Lymphoma Undergoing Autologous Hematopoietic Stem Cell Transplantation [NCT03733327]Phase 2/Phase 320 participants (Anticipated)Interventional2018-11-30Recruiting
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
Addition of Gemcitabine to the Standard Reduced Busulfan and Cyclophosphamide (BUCY2) Pre Allogeneic Hematopoietic Stem Cell Transplantation Conditioning for Acute Lymphoblastic Leukemia [NCT03339700]Phase 215 participants (Anticipated)Interventional2018-09-15Recruiting
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 2a Study of Once Daily Intravenous Busulfan With Bortezomib, Followed by an Autologous Hematopoietic Stem Cell Transplant (HSCT) in Subjects With Relapsed Multiple Myeloma After Prior Autologous HSCT [NCT01009840]Phase 230 participants (Actual)Interventional2010-05-31Completed
Allograft of Hematopoietic Stem Cells With Reduced-intensity Conditioning From a HLA-haploidentical Family Donor: Phase II Study of Combined Immunosuppression Before and After Transplantation [NCT00740467]Phase 250 participants (Anticipated)Interventional2008-01-31Recruiting
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
A Study of Outcomes and Toxicity of Busulfex as Part of a High Dose Chemotherapy Preparative Regimen in Autologous Hematopoietic Stem Cell Transplantation for Patients With Plasma Cell Myeloma [NCT00941720]Phase 271 participants (Actual)Interventional2009-06-11Completed
Phase II Study of Allogeneic Transplant of Hematopoietic Stem Cells From a Compatible Family Donor in the Treatment of Patients Over 55 Years With Hematological Malignancies [NCT00806767]Phase 282 participants (Anticipated)Interventional2007-03-31Completed
Hematopoietic Stem Cell Transplantation for the Treatment of Older Patients With Acute Myelogenous Leukemia [NCT00623935]Phase 256 participants (Actual)Interventional2007-03-31Completed
Reduced Intensity Stem Cell Transplantation (RIST) for Patients With Hematological Malignancies Conditioned With Fludarabine and Busulfan [NCT00619645]Phase 28 participants (Actual)Interventional2007-06-30Completed
Phase II Trial of Clofarabine With Parenteral Busulfan (Busulfex®) Followed by Allogeneic Related or Unrelated Donor Transplantation for the Treatment of Hematologic Malignancies and Diseases [NCT00852163]Phase 220 participants (Actual)Interventional2007-03-31Completed
Reduced Intensity Conditioning Regimen for Low- and Intermediate-risk Myelodysplastic Syndrome Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation [NCT03412266]Phase 250 participants (Anticipated)Interventional2018-02-01Recruiting
A Multicenter Pilot Study of Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation With In-vivo T-cell Depletion to Evaluate the Role of NK Cells and KIR Mis-matches in Relapsed or Refractory High-risk Neuroblastoma. [NCT00874315]Phase 20 participants (Actual)Interventional2008-09-30Withdrawn(stopped due to lack of accrual)
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
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02 [NCT00258427]Phase 214 participants (Actual)Interventional2002-03-26Completed
Phase II Study of Multimodality Therapy in Mantle Cell Lymphoma [NCT00004231]Phase 20 participants Interventional1999-10-31Completed
Autologous Blood Stem Cell Transplantation in Patients With Hodgkin's Disease and Non-Hodgkin's Lymphoma Who Have Had Prior Radiation Therapy [NCT00004171]Phase 20 participants Interventional1999-10-31Completed
Phase II Study Evaluating the Efficacy of Allogeneic Transplant Conditioning With Adaptive Dose Busulfan Intravenous (Busilvex®) in Patients at High Risk of Carrying Blood Diseases [NCT02483325]Phase 233 participants (Actual)Interventional2014-09-30Completed
A Phase II Pediatric Study of a Graft-VS.-Host Disease (GVHD) Prophylaxis Regimen With no Calcineurin Inhibitors After Day +60 Post First Allogeneic Hematopoietic Cell Transplant for Hematological Malignancies [NCT05579769]Phase 232 participants (Anticipated)Interventional2022-11-04Recruiting
CBA Versus FBA Conditioning Followed by Haploidentical Allogeneic HSCT in Treatment of High Risk and Refractory AML [NCT03384225]Phase 3120 participants (Anticipated)Interventional2016-08-01Recruiting
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.)
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major [NCT03171831]Phase 430 participants (Anticipated)Interventional2017-04-01Recruiting
A Pilot Trial Examining Myeloid-Derived Suppressor Cells and Checkpoint Immune Regulators' Expression in Allogeneic Stem Cell Transplant Recipients Using Myeloablative Busulfan and Fludarabine [NCT02916979]Phase 120 participants (Actual)Interventional2016-09-06Completed
A Phase I/II Open-Label Study of ECT-001-Expanded Cord Blood Transplantation in Pediatric and Young Adult (<21year) Patients With High-Risk and Very High-Risk Myeloid Malignancies [NCT04990323]Phase 1/Phase 212 participants (Anticipated)Interventional2021-12-01Recruiting
A Randomized Study of Once Daily IV Busulfan With Fludarabine With Hemopoietic Stem Cell Transplantation for Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS) [NCT00469144]Phase 3233 participants (Actual)Interventional2005-06-30Completed
To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study [NCT06069180]Phase 4160 participants (Anticipated)Interventional2023-11-15Recruiting
Phase I/II Study Using Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists [NCT05463133]Phase 1/Phase 250 participants (Anticipated)Interventional2022-07-08Recruiting
A Phase II Randomized Controlled Trial Comparing GVHD-Reduction Strategies for Allogeneic Peripheral Blood Transplantation (PBSCT) for Patients With Acute Leukemia or Myelodysplastic Syndrome: Selective Depletion of CD45RA+ Naïve T Cells (TND) vs. Post-Tr [NCT03970096]Phase 2120 participants (Anticipated)Interventional2019-11-19Recruiting
Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide [NCT02282904]Phase 1/Phase 27 participants (Actual)Interventional2014-10-23Terminated
Phase 3, Open Label, Multi-centre, Randomised Controlled International Study in Ewing Sarcoma [NCT00987636]Phase 3907 participants (Actual)Interventional2009-10-01Completed
Collection of Plasma Samples Using Sodium Heparin From Subjects Undergoing Intravenous Busulfan Treatment [NCT05711732]150 participants (Anticipated)Observational2023-05-15Not yet recruiting
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
A Phase I-II Study of Busulfan-fludarabine Conditioning and T-cell Depleted Allogeneic Stem Cell Transplantation for Patients With Advanced Hematologic Malignancies [NCT00943319]Phase 1/Phase 250 participants (Actual)Interventional2012-03-31Completed
A Phase III Trial in Adult Acute Myeloid Leukemia: Daunorubicin Dose-Intensification Prior to Risk-Allocated Autologous Stem Cell Transplantation [NCT00049517]Phase 3657 participants (Actual)Interventional2002-12-19Completed
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
Allogeneic Stem Cell Transplant for Children and Adolescents With Acute Lymoblastic Leukemia FORUM - Pharmacogenomic Study (add-on Study) [NCT02670564]Phase 41,000 participants (Anticipated)Interventional2013-04-30Recruiting
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study [NCT04009525]Phase 4800 participants (Anticipated)Interventional2019-06-01Recruiting
A Pilot Study of Sequential Autologous Stem Cell Transplantation and Immunotherapy With Reduced Intensity Allogeneic Stem Cell Transplant for High Risk Neuroblastoma [NCT00670410]Phase 115 participants (Actual)Interventional2003-11-30Terminated(stopped due to Poor accrual)
[NCT02577094]Phase 1/Phase 20 participants (Actual)InterventionalWithdrawn
Allogeneic Transplantation for Pediatric Leukemias With Unrelated Donors Using Fludarabine, Busulfan, 400 cGy Total Body Irradiation, and Thymoglobulin [NCT00679536]Phase 1/Phase 230 participants (Anticipated)Interventional2008-05-31Recruiting
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
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen [NCT00796068]Phase 2130 participants (Actual)Interventional2009-02-24Completed
Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome [NCT00885833]Phase 1/Phase 25 participants (Anticipated)Interventional2007-02-28Completed
Rasburicase to Prevent Graft -Versus-Host Disease [NCT00513474]Phase 146 participants (Actual)Interventional2008-01-31Completed
Determination of Optimal Busulfan Dose Using Pharmacokinetic Modeling in Hematopoietic Stem Cell Transplantation [NCT01018446]Phase 130 participants (Anticipated)Interventional2008-12-31Completed
Busulfan-Fludarabine-Clofarabine With Allogeneic Stem Cell Transplantation for Advanced Refractory Acute Myeloid Leukemia, Myelodysplastic Syndrome, and Advanced, Gleevec Refractory Chronic Myeloid Leukemia. A Randomized Phase II Study. [NCT00469014]Phase 272 participants (Actual)Interventional2006-09-30Completed
Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia [NCT00301834]Phase 235 participants (Actual)Interventional2005-01-31Completed
Allogeneic Hematopoietic Cell Transplantation for Patients With Acute Myelogenous Leukemia in Remission Using HLA-Matched Sibling Donors, HLA-Matched Unrelated Donors, or HLA-Mismatched Familial Donors - A Phase 2 Study [NCT01020734]Phase 2263 participants (Actual)Interventional2011-05-31Completed
A Phase I Study of Targeted, Dose-Escalated Intravenous Busulfan and Bolus Etoposide as Preparative Therapy for Patients With Acute Myeloid Leukemia Undergoing Autologous Stem Cell Transplantation [NCT01048827]Phase 112 participants (Actual)Interventional2009-11-30Completed
Phase II Study of Maintenance Therapy With Decitabine (NSC #127716) Following Standard Induction and Cytogenetic Risk-Adapted Intensification in Previously Untreated Patients With AML < 60 Years [NCT00416598]Phase 2546 participants (Actual)Interventional2006-11-15Completed
A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT03600909]Phase 23 participants (Actual)Interventional2018-05-15Terminated(stopped due to Accrual has been slow)
Randomized Trial of GVHD Prophylasxis With Post-transplantation Cyclophocphomide (PTCy) or Thymoglobulin in Unrelated SCT Recepients With Chronic Myeloproliferative Neoplasms and Myelodisplatic Syndrome [NCT02627573]Phase 232 participants (Actual)Interventional2015-07-31Terminated(stopped due to Poor recruitment)
Chemo Sensitization Before Hematopoietic Stem Cell Transplantation With a CXCR4 Antagonist in Patients With Acute Leukemia in Complete Remission: Pilot Study [NCT02605460]Phase 220 participants (Anticipated)Interventional2014-02-28Recruiting
Treatment of Severe (Types II and III) Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation [NCT00705120]Phase 19 participants (Actual)Interventional1995-11-30Completed
A Phase II Trial of Myeloablative Conditioning and Transplantation of Partially HLA-mismatched Bone Marrow for Patients With Hematologic Malignancies [NCT00796562]Phase 2107 participants (Actual)Interventional2008-12-31Completed
Randomized Comparison of Once-daily Intravenous Busulfan Plus Cyclophosphamide Versus Fludarabine as a Conditioning Regimen for Allogeneic Hematopoietic Cell Transplantation in Leukemia and Myelodysplastic Syndrome [NCT00774280]Phase 3130 participants (Actual)Interventional2002-05-31Completed
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic Malignancies [NCT01760655]Phase 262 participants (Actual)Interventional2012-12-24Completed
G-CSF and Plerixafor With Busulfan and Fludarabine for Allogeneic Stem Cell Transplantation for Myeloid Leukemias [NCT00822770]Phase 1/Phase 247 participants (Actual)Interventional2009-01-31Completed
Reduced Intensity Conditioning Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies at High Risk for Transplant Related Mortality With Standard Transplantation [NCT00795132]Phase 247 participants (Actual)Interventional2004-04-30Completed
Post Transplant Cyclophosphamide for Unrelated and Related Allogeneic Hematopoietic Stem Cell Transplantation for Hematological Malignancies [NCT01349101]Phase 280 participants (Actual)Interventional2011-02-10Completed
A Phase II, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of OTI-010 in Subjects Who Receive HLA-Identical Sibling Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies [NCT00081055]Phase 20 participants (Actual)InterventionalWithdrawn
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
The Efficiency and Safety of N-acetylcysteine for Prevention of Thrombotic Events After Allogenic Hematopoietic Stem Cell Transplantation [NCT05907486]Phase 3260 participants (Anticipated)Interventional2023-08-01Not yet recruiting
High-dose Chemotherapy With Autologous Hematopoietic Stem Cell Transplantation as Adjuvant Treatment for Triple Negative Breast Cancer Patients Without Complete Pathological Response to Neoadjuvant Chemotherapy [NCT02670109]Phase 220 participants (Anticipated)Interventional2018-02-01Recruiting
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
Pilot Study of Allogeneic Stem Cell Transplantation From Unrelated Donors for Patients With Severe Homozygous Beta 0/+ Thalassemia or Severe Variants of Beta 0/+ Thalassemia [NCT00578292]10 participants (Actual)Interventional2004-02-29Terminated(stopped due to Stem cell transplant was determined SOC for this disease (study is not relevant))
A First-in-Human Study of HLA-Partially to Fully Matched Allogenic Cryopreserved Deceased Donor Bone Marrow Transplantation for Patients With Hematologic Malignancies [NCT05589896]Phase 1/Phase 212 participants (Anticipated)Interventional2023-11-30Recruiting
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease [NCT02629120]Phase 1/Phase 244 participants (Actual)Interventional2015-12-17Active, not recruiting
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency [NCT01306019]Phase 1/Phase 219 participants (Actual)Interventional2012-09-25Suspended(stopped due to Clones representing 10% or more of the subject patients myeloid lineage have been detected, no evidence of malignancy found.)
Autologous Bone Marrow Transplant for Children With AML in First Complete Remission: Use of Marker Genes to Investigate the Biology of Marrow Reconstitution and the Mechanism of Relapse [NCT00667927]Phase 117 participants (Actual)Interventional1991-03-31Completed
Clinical Phase III Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Reduced-intensity Conditioning (RIC) Prior to Allogeneic Haematopoietic Stem Cell Transplantation in Patients With AML or MDS Considered Ineligible to Standard C [NCT00822393]Phase 3570 participants (Actual)Interventional2008-11-24Completed
A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies [NCT02793544]Phase 280 participants (Actual)Interventional2016-12-31Completed
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.)
Thiotepa-Clofarabine-Busulfan With Allogeneic Stem Cell Transplant for High Risk Malignancies [NCT00857389]Phase 260 participants (Actual)Interventional2009-03-02Completed
Idarubicin+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Intermediate-risk Acute Myeloid Leukemia Undergoing Autologous Hematopoietic Stem Cell Transplantation [NCT02671708]Phase 2/Phase 3153 participants (Actual)Interventional2016-01-31Completed
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity [NCT04339777]Phase 266 participants (Anticipated)Interventional2020-09-22Recruiting
Phase II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Cord Blood Transplantation in Pediatric Acute Myeloid Leukemia [NCT00887042]Phase 1/Phase 235 participants (Anticipated)Interventional2006-06-30Active, not recruiting
Umbilical Cord Blood (UCB) Allogeneic Stem Cell Transplant for Hematologic Malignancies [NCT01093586]Phase 214 participants (Actual)Interventional2007-09-30Completed
Pilot Study of Reduced Intensity Haematopoietic Stem Cell Transplantation in Patients With Poor Risk Myelodysplastic Syndrome and Acute Myeloid Leukaemia Utilising Conditioning With Fludarabine, Busulphan and Thymoglobulin [NCT00915811]Phase 220 participants (Anticipated)Interventional2007-06-30Terminated(stopped due to FB ATG is now a standard for sib allo MDS patients)
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)
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
Romidepsin Therapy in Conditioning and Maintenance in Patients With T-Cell Malignancies Receiving Allogeneic Stem Cell Transplant [NCT02512497]Phase 110 participants (Anticipated)Interventional2017-12-08Recruiting
Safety and Efficacy Study of Busulfan/FLAG Conditioning Regimen in Patients With Relapsed/Refractory Acute Leukemia Undergoing Allogeneic Peripheral Blood Stem Cell Transplantation [NCT02784561]Phase 480 participants (Anticipated)Interventional2016-07-31Not yet recruiting
Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Myeloid Tumors Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT04098653]Phase 2/Phase 3196 participants (Anticipated)Interventional2019-09-30Recruiting
Prospective Phase II Clinical Trial of Myeloablative Conditioning Regimen With Fludarabine and Busulfan Plus 400 cGy Total Body Irradiation for Hematologic Malignancies [NCT00815568]Phase 2114 participants (Anticipated)Interventional2008-08-31Recruiting
Busulfan, Cyclophosphamide, Imatinib Mesylate and Autologous Stem Cell Transplantation in Patients With Chronic Myelogenous Leukemia [NCT01003054]Phase 224 participants (Actual)Interventional2005-03-31Completed
Phase I/II Study of Myeloablative Allogeneic Stem Cell Transplantation for Aggressive Hematologic Malignancies Using Clofarabine and Busulfan x 4 (Clo/BU4) Regimen [NCT00556452]Phase 1/Phase 246 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)
A Phase I Study Using Submyeloablative DOsing of Intravenous Busulfan (Busulfex) for Refractory Brain Tumors [NCT00836628]Phase 10 participants (Actual)Interventional2008-07-31Withdrawn(stopped due to slow accrual)
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
Phase II Study of Radiolabeled BC8 (Anti-CD45) Antibody Combined With Busulfan and Cyclophosphamide as Treatment for Acute Myelogenous Leukemia in First Remission Followed by HLA-Identical Related Peripheral Blood Stem Cell Transplantation [NCT00005940]Phase 218 participants (Actual)Interventional1999-10-31Completed
[NCT00006054]0 participants Interventional2000-03-31Terminated
Allogeneic Peripheral Blood Progenitor Cell Transplantation in Patients With Incurable Solid Tumors: A Phase I Study [NCT00006126]Phase 10 participants (Actual)Interventional1999-09-30Withdrawn(stopped due to Unable to accrue subjects.)
Phase I Study of Intrathecal Spartaject-Busulfan in Children With Neoplastic Meningitis [NCT00006246]Phase 128 participants (Actual)Interventional2000-11-30Completed
Autologous Transplantation for Chronic Myelogenous Leukemia Using Retrovirally Marked Peripheral Blood Progenitor Cells Obtained After In Vivo Cyclophosphamide/G-CSF Priming [NCT00005986]Phase 20 participants Interventional2000-08-31Terminated(stopped due to Principal investigator left the university.)
Autologous Peripheral Blood Stem Cell Mobilization and Transplantation for Myelofibrosis [NCT00006367]Phase 20 participants Interventional2000-05-31Completed
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
A Phase II Open-label, Multicenter, Non Randomized Study Evaluating the Efficacy and the Safety of Clofarabine in Combination With IV Busulfan and Thymoglobulin (CBT) as a Reduced Intensity Conditioning Regimen Prior to Allogeneic Stem Cell Transplantatio [NCT00863148]Phase 230 participants (Actual)Interventional2009-10-31Completed
Prospective , Multicentric, Randomized Phase II Study, Evaluating the Role of Cranial Radiotherapy or Intensive Chemotherapy With Hematopoietic Stem Cell Rescue After Conventional Chemotherapy for Primary Central Nervous System in Young Patients (< 60 y) [NCT00863460]Phase 2140 participants (Actual)Interventional2008-10-03Active, not recruiting
A Pilot Study of High Dose Busulfan Combined With IL2/GM-CSF Activated Autologous/Syngeneic PBSC, Sequential IL2/GM-CSF Therapy and Alpha Interferon Maintenance Therapy as Treatment of CML [NCT00005948]Phase 20 participants Interventional2000-01-31Completed
Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation Following Reduced Intensity Conditioning in Treating Patients With Multiple Myeloma [NCT00802568]Phase 248 participants (Anticipated)Interventional2007-04-30Completed
A Pilot Study of Reduced Intensity Allogeneic Stem Cell Transplantation in Patients With Chronic Myeloid Leukemia (CML) and Adoptive Cellular Immunotherapy Only in Patients With Persistent Disease and Matched Family Donors [NCT00531310]Phase 25 participants (Actual)Interventional2003-01-31Terminated(stopped due to Poor accrual)
A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB) [NCT00881556]Early Phase 13 participants (Actual)Interventional2009-08-20Terminated
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
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
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
Phase 2 Study of Autologous Hematopoietic Cell Transplantation for Core-binding Factor Positive Acute Myeloid Leukemia in the First Complete Remission [NCT01050036]39 participants (Anticipated)Interventional2010-01-31Recruiting
High-dose Chemotherapy and Autologous Stem Cell Transplant or Consolidating Conventional Chemotherapy in Primary CNS Lymphoma - Randomized Phase III Trial [NCT02531841]Phase 3250 participants (Anticipated)Interventional2014-07-31Recruiting
Timed Sequential Busulfan and Post Transplant Cyclophosphamide for Allogeneic Transplantation [NCT02861417]Phase 2204 participants (Actual)Interventional2016-08-05Active, not recruiting
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
Conditioning for Hematopoietic Cell Transplantation With Fludarabine Plus Targeted IV Busulfan and GVHD Prophylaxis With Thymoglobulin, Tacrolimus and Methotrexate in Patients With Myeloid Malignancies [NCT01056614]Phase 223 participants (Actual)Interventional2004-09-30Completed
Allogeneic Stem Cell Transplantation Followed By Targeted Immune Therapy In Average Risk Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia (AML/MDS/JMML) [NCT01020539]Phase 118 participants (Actual)Interventional2002-09-11Completed
An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies [NCT04547049]Phase 3160 participants (Anticipated)Interventional2020-09-01Recruiting
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
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
Allogeneic Bone Marrow Transplantation With Matched Unrelated Donors for Patients With Hematologic Malignancies Using a Preparative Regimen of Busulfan, Cyclophosphamide, and Fludarabine [NCT00208923]Phase 255 participants (Anticipated)Interventional1998-07-31Completed
Phase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency [NCT00228852]Phase 1/Phase 20 participants InterventionalCompleted
Olaparib Combined With High-Dose Chemotherapy for Refractory Lymphomas [NCT03259503]Phase 150 participants (Actual)Interventional2019-09-13Active, not recruiting
Autologous Blood or Marrow Transplantation for Aggressive Non-Hodgkin's Lymphoma Based on Early [18F] FDG-PET Scanning [NCT00238368]Phase 259 participants (Actual)Interventional2004-02-29Completed
Rituximab, Methotrexate, Procarbazine, Vincristine, Lenalidomide (RL-MPV) Followed by BBC (BCNU, Busulfan, Cyclophosphamide) High-dose Chemotherapy With Auto-HCT and Maintenance Therapy With Nivolumab in Newly Diagnosed Primary CNS Lymphoma [NCT05425654]Phase 230 participants (Anticipated)Interventional2021-05-17Recruiting
Allogeneic Stem Cell Transplantation for Children and Adolescents With Acute Lymphoblastic Leukaemia [NCT01949129]Phase 2/Phase 31,000 participants (Anticipated)Interventional2013-04-30Recruiting
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)
Personalized Monitoring of Intravenous Busulfan Dosing for Patients With Lymphoma Undergoing Autologous Stem Cell Transplantation. [NCT01959477]Early Phase 133 participants (Actual)Interventional2014-03-31Completed
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
Prospective and Multicentre Evaluation of 3 Different Doses of IV Busulfan Associated With Fludarabine and Thymoglobuline in the Conditioning of Allogeneic Stem Cell Transplantation (SCT) From a Matched Related or Unrelated Donor in Patients With Poor Pro [NCT01985061]Phase 2177 participants (Anticipated)Interventional2013-12-31Recruiting
Sequential Myeloablative Autologous Stem Cell Transplantation Followed by Allogeneic Non-Myeloablative Stem Cell Transplantation for Patients With Poor Risk Lymphomas [NCT01181271]Phase 242 participants (Actual)Interventional2010-08-31Completed
MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions [NCT02582775]Phase 217 participants (Actual)Interventional2016-03-31Completed
Pilot Study of the Safety and Feasibility of Autologous Peripheral Blood Stem Cell Transplantation for Patients With Relapsed AIDS-Related Lymphoma [NCT00005824]Phase 20 participants Interventional2000-11-30Completed
A Phase III Randomized Study Comparing Busulfan-Total Body Irradiation Versus Cyclophosphamide-Total Body Irradiation Preparative Regimen in Patients With Advanced Myelodysplastic Syndrome (MDS) or MDS-Related Acute Myeloid Leukemia (AML) Undergoing HLA-I [NCT00005866]Phase 3240 participants (Anticipated)Interventional2000-02-29Completed
A Multi-center, Phase 2, Single-Arm, Open-Label Exploratory Study of Individually- Optimized Conditioning Using Pharmacokinetics [PK]-Directed Dose Adjustment of Once Daily Intravenous Busulfan, Followed by Autologous Hematopoietic Stem Cell Transplant in [NCT00948090]Phase 2207 participants (Actual)Interventional2010-01-31Completed
[NCT00005893]0 participants Interventional2000-06-30Completed
Phase III Randomized Study of Induction Chemotherapy With or Without MDR-Modulation With PSC-833 (NSC # 648265, IND # 41121) Followed by Cytogenetic Risk-Adapted Intensification Therapy Followed by Immunotherapy With rIL-2 (NSC # 373364, IND # 1969) vs. O [NCT00006363]Phase 3720 participants (Actual)Interventional2000-11-30Completed
High Dose Chemotherapy And Autologous Peripheral Blood Stem Cell Rescue For High Risk Acute Leukemia [NCT00008190]Phase 20 participants Interventional1999-03-31Completed
Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus [NCT04370795]Phase 1/Phase 230 participants (Anticipated)Interventional2024-01-03Enrolling by invitation
Allogeneic Stem Cell Transplantation for Myelofibrosis and Myelodysplastic Syndrome Using Reduced Intensity Busulfan and Fludarabine Conditioning [NCT00475020]Phase 263 participants (Actual)Interventional2006-01-04Completed
Ex Vivo Expansion of Mafosfamide Purged CD34+ Cells in Patients With Acute Leukemia [NCT00245115]Phase 17 participants (Actual)Interventional2005-10-31Terminated(stopped due to Expiration of study supply)
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis [NCT00578435]Phase 225 participants (Anticipated)Interventional1994-01-31Completed
Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders [NCT05293509]Phase 20 participants (Actual)Interventional2022-03-02Withdrawn(stopped due to 0 accrual)
Multi-Center Phase II Randomized Controlled Trial of Naïve T Cell Depletion for Prevention of Chronic Graft-Versus-Host Disease in Children and Young Adults [NCT03779854]Phase 268 participants (Anticipated)Interventional2019-08-29Recruiting
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
HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease [NCT00578643]Phase 215 participants (Actual)Interventional2004-03-31Completed
Hematopoietic Stem Cell Transplantation From Haploidentical Donors in Patients With Hematological Malignancies Using a Treosulfan-Based Preparative Regimen [NCT04195633]Phase 260 participants (Anticipated)Interventional2021-01-25Recruiting
"Phase II Study of a Reduced-toxicity Submyeloablative Conditioning Regimen Prior to Allogeneic Stem Cell Transplantation in Patients With Hematological Malignancies" [NCT00841724]Phase 282 participants (Actual)Interventional2009-01-31Completed
Allogeneic Bone Marrow Transplantation From HLA Identical Related Donors for Patients With Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, or Hemoglobin SB0/+ Thalassemia [NCT00578344]8 participants (Actual)Interventional2005-07-31Terminated(stopped due to Terminated due to no new subject enrollment during the last 3 year period.)
Non-myeloablative Allogeneic Stem Cell Transplantation With Match Unrelated Donors for Treatment of Hematologic Malignancies and Renal Cell Carcinoma and Aplastic Anemia [NCT00295997]35 participants (Anticipated)Interventional2005-05-31Active, not recruiting
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
Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia [NCT00669890]Phase 112 participants (Actual)Interventional2004-05-31Terminated(stopped due to PI left institution)
International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia - Incorporating an Embedded Dose Finding Study for Gemtuzumab Ozogamicin in Combination With Induction Chemotherapy [NCT02724163]Phase 3700 participants (Anticipated)Interventional2016-04-30Recruiting
Early Diagnosis and Stem Cell Transplantation for Severe Immunodeficiency Diseases [NCT00613561]Phase 225 participants (Anticipated)Interventional2007-12-31Recruiting
Unrelated Donor Hematopoietic Stem Cell Transplantation After Nonmyeloablative Conditioning For Patients With Hematological Malignancies [NCT00627666]Phase 252 participants (Anticipated)Interventional2003-01-31Completed
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
Allogeneic Stem Cell Transplant to Induce Mixed Donor Chimerism in Patients With Sickle Cell Disease and Thalassemia [NCT00408447]Phase 253 participants (Actual)Interventional2004-09-30Active, not recruiting
Phase II Study for Safety and Efficacy of BEB (Bendamustine, Etoposide, Busulfan) Conditioning Regimen for ASCT in Non-Hodgkin's Lymphoma [NCT02836639]Phase 220 participants (Anticipated)Interventional2016-02-29Recruiting
Graft-versus-host Disease Prophylaxis With Post-transplantation Cyclophosphamide and Ruxolitinib in Patients With Myelofibrosis [NCT02806375]Phase 1/Phase 220 participants (Actual)Interventional2016-01-31Completed
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
Fractionated Busulfan Combined With Chidamide/Fludarabine/Cytarabine (ChiFAB) With Allogeneic Hematopoietic Stem-Cell Transplantation in Non-remission Myeloid Malignancies: a Phase II Study [NCT05807659]Phase 218 participants (Anticipated)Interventional2023-03-16Recruiting
[NCT00006056]40 participants Interventional2000-03-31Active, not recruiting
A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies [NCT00423826]0 participants Expanded Access2007-01-31No longer available
Präferenz-Studie Bei älteren Patientinnen Mit Ovarialkarzinomrezidiv: Treosulfan Oral vs. intravenös [NCT00170690]Phase 3123 participants (Actual)Interventional2004-08-31Completed
Dose-escalation Study of Graft-versus-host Disease Prophylaxis With High-dose Post-transplantation Bendamustine in Patients With Refractory Acute Leukemia [NCT02799147]Phase 1/Phase 227 participants (Actual)Interventional2016-06-30Completed
Azacitidine Maintenance Therapy After Allogeneic Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML) [NCT00813124]Phase 224 participants (Actual)Interventional2008-12-31Completed
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
Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease [NCT00186810]Phase 215 participants (Actual)Interventional1992-12-31Completed
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
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation [NCT00301912]Phase 20 participants (Actual)Interventional2002-01-31Withdrawn(stopped due to Withdrawn because study never opened to accrual)
Hemophagocytic Lymphohistiocytosis [NCT00334672]Phase 3288 participants (Anticipated)Interventional2006-03-31Active, not recruiting
Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft [NCT00638820]Phase 23 participants (Actual)Interventional2007-09-30Terminated(stopped due to Excess toxicity)
T Cell Receptor Alpha/Beta T Cell Depleted (α/β TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA) [NCT03579875]Phase 248 participants (Anticipated)Interventional2018-11-13Recruiting
Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Advanced Hematological Malignancies After Treosulfan-based Conditioning Therapy - A Clinical Phase II Study [NCT01063647]Phase 1/Phase 256 participants (Actual)Interventional2001-11-30Completed
Busulfan Plus Cyclophosphamide vs Total Body Irradiation Plus Cyclophosphamide Conditioning Regimen for Standard-risk Acute Lymphocytic Leukemia Undergoing HLA-matched Allogeneic Hematopoietic Stem Cell Transplantation [NCT02670252]Phase 3550 participants (Actual)Interventional2016-01-31Completed
International Collaborative Treatment Protocol for Infants Under One Year With Acute Lymphoblastic or Biphenotypic Leukemia [NCT00550992]445 participants (Anticipated)Interventional2006-01-31Recruiting
A Phase II Trial of Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched Peripheral Blood Stem Cells for Patients With Hematologic Malignancies [NCT00782379]Phase 220 participants (Actual)Interventional2008-10-31Completed
Granulocyte Colony-stimulating Factor+Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT02744742]Phase 2/Phase 3202 participants (Actual)Interventional2016-04-18Completed
AUTOLOGOUS BONE MARROW TRANSPLANTATION USING C-MYB (LR-3001) ANTISENSE OLIGODEOXYNUCLEOTIDE TREATED BONE MARROW IN CHRONIC MYELOGENOUS LEUKEMIA IN CHRONIC OR ACCELERATED PHASE [NCT00002592]Phase 240 participants (Anticipated)Interventional1993-06-30Completed
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
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies [NCT01471067]Phase 133 participants (Actual)Interventional2012-07-13Completed
Haploidentical Hematopoietic Stem Cell Transplantation in the Treatment of Hematological Malignancies Using CAMPATH-1H [NCT00458250]Phase 110 participants (Anticipated)Interventional2006-09-30Completed
Randomized Multicenter Treatment Optimization Study In Chronic Myeloid Leukemia (CML) Interferon-a Vs. Allogeneic Stem Cell Transplantation Vs. High-Dose Chemotherapy Followed By Autografting And Interferon-a Maintainance In Early Chronic Phase [NCT00025402]Phase 31,000 participants (Anticipated)Interventional1997-07-31Active, not recruiting
Phase II Trial of Fludarabine Combined With Intravenous Treosulfan and Allogeneic Hematopoietic Stem-cell Transplantation in Patients With Chemo-refractory or Previously Untreated Acute Myeloid Leukemia and Myelodysplastic Syndrome. [NCT00491634]Phase 224 participants (Anticipated)Interventional2007-06-30Completed
NON-T-CELL DEPLETED HLA-HAPLOIDENTICAL FAMILIAL DONOR HEMATOPOIETIC CELL TRANSPLANTATION AFTER REDUCED INTENSITY CONDITIONING [NCT00521430]30 participants (Anticipated)Interventional2004-04-30Completed
Allogeneic Transplant for Hematological Malignancy [NCT00176930]330 participants (Actual)Interventional2001-10-31Terminated(stopped due to replaced by another study ; Trial re-written as MT2015-29)
Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation [NCT00516152]Phase 236 participants (Anticipated)Interventional2002-11-30Completed
Rituximab For Prevention Of Acute Graft-Versus-Host Disease (GVHD) After Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (HCT) [NCT01044745]Phase 220 participants (Actual)Interventional2009-12-10Terminated(stopped due to Study was closed to accrual for safety related to the frequency of BK infections.)
Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe) [NCT04942730]Phase 240 participants (Anticipated)Interventional2021-01-21Recruiting
A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT02143830]Phase 270 participants (Anticipated)Interventional2014-04-30Recruiting
Phase I/II Study De-intensifying Exposure of Post-transplantation Cyclophosphamide as GVHD Prophylaxis After HLA-haploidentical Hematopoietic Cell Transplantation for Hematologic Malignancies [NCT03983850]Phase 1/Phase 2400 participants (Anticipated)Interventional2019-07-09Recruiting
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
Cord Blood Ex-Vivo MSC Expansion Plus Fucosylation to Enhance Homing and Engraftment [NCT03096782]Phase 26 participants (Actual)Interventional2017-10-13Completed
Pre-administration of Rabbit Antithymocyte Globulin to Optimize Donor T-Cell Engraftment Following Reduced Intensity Allogeneic Peripheral Blood Progenitor Cell Transplantation From Matched-Related Donors [NCT00787761]Phase 224 participants (Actual)Interventional2007-04-30Completed
Sequential Myeloablative Stem Cell Transplantation and Reduced Intensity Allogeneic Stem Cell Transplantation in Patients With Refractory or Recurrent Non-Hodgkin's Lymphoma and Hodgkin's Disease [NCT00802113]Phase 1/Phase 230 participants (Actual)Interventional2003-06-30Completed
Targeted Busulfan, Fludarabine, Etoposide Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Childhood and Adolescent Acute Lymphoblastic Leukemia [NCT02047578]Phase 238 participants (Anticipated)Interventional2014-02-05Recruiting
Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen [NCT00919503]Phase 298 participants (Actual)Interventional2009-07-31Completed
HLA Matched Related and Unrelated Bone Marrow Transplantation With Busulfan/Cyclophosphamide and Post Transplantation Cyclophosphamide for Hematological Malignancies [NCT00134017]Phase 2142 participants (Actual)Interventional2004-06-30Completed
BUSULFAN AND CYCLOPHOSPHAMIDE FOR CYTOREDUCTION OF PATIENTS WITH ACUTE AND CHRONIC LEUKEMIAS AND MYELODYSPLASTIC SYNDROMES UNDERGOING ALLOGENEIC BONE MARROW TRANSPLANTATION WHO CANNOT BE TREATED WITH TOTAL BODY IRRADIATION [NCT00002502]Phase 20 participants Interventional1992-07-31Completed
Pilot Study for Matched-Related Allogeneic Bone Marrow Transplantation for Metastatic Malignant Melanoma [NCT00003060]Phase 16 participants (Anticipated)Interventional1995-03-31Terminated(stopped due to lack of patient accrual)
Randomized Trial of Autologous GVHD for Refractory Lymphoma [NCT00003414]Phase 350 participants (Anticipated)Interventional1997-10-31Completed
Phase I Study of Intrathecal Spartaject-Busulfan in Patients With Neoplastic Meningitis [NCT00003462]Phase 120 participants (Anticipated)Interventional1998-04-30Completed
Matched Unrelated and Haploidentical Bone Marrow Transplantation for Hematologic Malignancies [NCT00003960]Phase 236 participants (Anticipated)Interventional1998-04-30Completed
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
The Value of High Dose Versus Standard Dose ARA-C During Induction and of IL-2 After Intensive Consolidation/Autologous Stem Cell Transplantation in Patients (Age 15-60 Years) With Acute Myelogenous Leukemia. A Randomized Phase II Trial of the EORTC and t [NCT00004128]Phase 32,000 participants (Anticipated)Interventional1999-09-30Active, not recruiting
Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study [NCT02248597]Phase 227 participants (Actual)Interventional2015-02-25Completed
Phase II Study of IV Busulfan Combined With 12 cGy of Fractionated Total Body Irradiation (FTBI) and Etoposide (VP-16) as a Preparative Regimen for Allogeneic Bone Marrow Transplantation for Patients With Advanced Hematological Malignancies [NCT00534430]Phase 230 participants (Actual)Interventional2000-02-29Active, not recruiting
A Pilot Study of Using Filgrastim-Primed Bone Marrow in Human Leukocyte Antigen (HLA) Matched Related Donor Allogenetic Bone Marrow Transplantation for Patients With Hematologic Malignancies and Non-Malignancies [NCT00253552]4 participants (Actual)Interventional2004-05-31Terminated(stopped due to Terminated at request of PI as study was outdated.)
Pilot Study on Allogeneic Stem Cell Transplantation Following Conditioning With Fludarabine and an Alkylating Agent in Patients With High-Risk Chronic Lymphocytic Leukemia [NCT00281983]Phase 1/Phase 2100 participants (Actual)Interventional2000-06-30Completed
Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies [NCT00301951]Phase 17 participants (Actual)Interventional2004-09-30Completed
Pilot Study of Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies [NCT00304018]Phase 15 participants (Actual)Interventional2002-10-31Completed
Low-Dose Allogeneic Peripheral Blood Stem Cell Transplantation for High-Risk Low Grade Hematologic Malignancies [NCT00296023]25 participants (Actual)Interventional1999-01-31Completed
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
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome [NCT01861106]Phase 2144 participants (Anticipated)Interventional2013-07-24Recruiting
Secondary Transplantation Using Moderate Dose Busulfan as Conditioning for a Patient With Partial Reconstitution Post Initial Allogeneic Transplantation [NCT00092937]Phase 12 participants (Actual)Interventional2004-09-23Completed
The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells [NCT00084695]Phase 225 participants (Anticipated)Interventional2003-09-30Recruiting
Alloreactive NK Cells With Busulfan, Fludarabine and Thymoglobulin for Allogeneic Stem Cell Transplantation for AML and MDS [NCT00402558]Phase 115 participants (Actual)Interventional2006-05-31Completed
Pilot Trial of Two Dose Levels of Thymoglobulin® as Part of a Myeloablative-Conditioning for a HLA Identical Matched Related Donor (MRD) Stem Cell Transplant With Cyclosporine (CsA) as Posttransplant Graft vs Host Disease (GvHD) Prophylaxis [NCT00093587]0 participants Interventional2004-08-31Active, not recruiting
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
Allogeneic Hematopoietic Cell Transplantation for Patients With Hematologic Disorders Who Are Ineligible or Inappropriate for Treatment With a More Intensive Therapeutic Regimen [NCT00448201]Phase 271 participants (Actual)Interventional2011-01-07Completed
Autologous Purged Hematopoietic Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML) [NCT00446173]Phase 20 participants (Actual)Interventional2007-03-31Withdrawn(stopped due to Terminated due to low accrual.)
Cord Blood Expansion on Mesenchymal Stem Cells [NCT00498316]Phase 198 participants (Actual)Interventional2007-07-03Completed
Umbilical Cord Blood Transplant for Children With Myeloid Hematological Malignancies (UCAML) [NCT01247701]16 participants (Actual)Interventional2010-11-30Completed
Busulfan and Fludarabine as Conditioning for Allogeneic Stem Cell [NCT00156858]200 participants (Anticipated)Interventional1998-01-31Recruiting
Venetoclax to Improve Outcomes of Fractionated Busulfan Regimen in Patients With High-Risk AML and MDS [NCT04708054]Phase 2100 participants (Anticipated)Interventional2021-10-21Recruiting
A Prospective Trial to Evaluate the Role of In Vivo T Cell Depletion by Campath® (Alemtuzumab) in Reduction of Transplant Related Mortality in Transplantation From HLA-Class I or Class II Mismatched, Unrelated Donors [NCT00555048]Phase 1/Phase 21 participants (Actual)Interventional2007-09-30Terminated(stopped due to Low accrual)
Phase II Study of the Addition of Azacitidine (NSC#102816) to Reduced-Intensity Conditioning Allogeneic Transplantation for Myelodysplasia (MDS) and Older Patients With AML [NCT01168219]Phase 268 participants (Actual)Interventional2010-07-15Completed
CD34 Selected Allogeneic Hematopoietic Cell Transplantation With Myeloablative Conditioning and CD8+ Memory T Cell Infusion For Patients With Myelodysplastic Syndrome, Acute Leukemia, and Chronic Myelogenous Leukemia [NCT04151706]Phase 220 participants (Anticipated)Interventional2020-02-27Suspended(stopped due to Interim analysis)
Reduced-intensity, Related-donor Allogeneic BMT With Fludarabine, Busulfan, and High-dose Posttransplantation Cyclophosphamide for Hematologic Malignancies [NCT01135329]Phase 215 participants (Actual)Interventional2010-08-31Terminated(stopped due to The stopping rule was met and hence the study was closed)
Umbilical Cord Blood Transplantation From Unrelated Donors [NCT03016806]Phase 130 participants (Anticipated)Interventional2015-06-30Recruiting
Reduced-Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Malignant Hematological Diseases [NCT00582894]17 participants (Actual)Interventional2005-02-28Completed
Allogeneic Hematopoietic Stem Cell Transplantation For The Treatment Of High Risk Multiple Myeloma With Reduced Toxicity Myeloablative Conditioning Regimen [NCT00615589]Phase 222 participants (Actual)Interventional2008-02-29Terminated(stopped due to Low accrual)
Combined Haploidentical-Cord Blood Transplantation for Adults and Children [NCT00943800]87 participants (Actual)Interventional2006-10-09Completed
A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT00987480]Phase 245 participants (Actual)Interventional2009-09-25Completed
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders [NCT01043640]Phase 246 participants (Actual)Interventional2009-12-31Completed
A Phase II Study of Hematopoietic Stem Cell Therapy for Young Adults With Severe Sickle Cell Disease [NCT01565616]Phase 222 participants (Actual)Interventional2012-03-31Completed
Population Pharmacokinetics, Effectiveness and Safety of Antineoplastic Drugs in Elderly Patients [NCT05467189]500 participants (Anticipated)Observational [Patient Registry]2021-01-01Recruiting
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC) [NCT01666080]30 participants (Anticipated)Interventional2012-08-31Recruiting
Chidamide Combined With Cladribine/Gemcitabine/Busulfan (ChiCGB) With Autologous Stem-Cell Transplantation in Relapsed and Refractory Diffuse Large B Cell Lymphoma [NCT03151876]Phase 293 participants (Anticipated)Interventional2017-06-12Recruiting
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)
Pacritinib Prior to Transplant for Patients With Myeloproliferative Neoplasms (MPN) [NCT02410551]Phase 24 participants (Actual)Interventional2015-06-15Terminated
Multi-Center Study Using Allogeneic Stem Cell Transplantation Following Reduced Intensity Chemotherapy in Patients With Hemoglobinopathies [NCT00153985]Phase 22 participants (Actual)Interventional2004-03-31Completed
Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis) [NCT00176917]Phase 241 participants (Actual)Interventional1999-05-31Completed
A Phase I/II Combination Study of Topotecan, Fludarabine, Cytosine Arabinoside and G-CSF (T-FLAG) Induction Therapy in Patients With Poor Prognosis AML, MDS and Relapsed/Refractory ALL Followed by Maintenance of Either PBSC Transplant or 13 Cis-Retinoic A [NCT00003619]Phase 1/Phase 20 participants Interventional1998-02-28Completed
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
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
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 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
Sibling Donor Cord Blood Banking and Transplantation [NCT00029380]Phase 230 participants (Anticipated)Interventional1999-01-31Completed
Allogeneic Stem Cell Transplantation Following Nonmyeloablative Chemotherapy in Patients With Hemoglobinopathies [NCT00034528]Phase 22 participants (Actual)Interventional2001-09-30Terminated(stopped due to Due to slow recruitment)
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
Dose Finding Study of Gelonin Purging of Autologous Stem Cells for Transplantation of Patients With AML/MDS in First or Subsequent Remission [NCT00043810]Phase 1/Phase 23 participants (Actual)Interventional2002-07-31Terminated
Conditioning With Targeted Busulfan, Cyclophosphamide and Thymoglobulin for Allogeneic Marrow or Peripheral Blood Stem Cell (PBSC) Transplantation for Myelodysplasia and Myeloproliferative Disorders [NCT00054340]Phase 1/Phase 20 participants Interventional2002-10-31Completed
Treatment of Newly Diagnosed Childhood Acute Myeloid Leukemia (AML) Using Intensive MRC-Based Therapy and Gemtuzumab Ozogamicin (GMTZ): A COG Pilot Study [NCT00070174]Phase 2350 participants (Actual)Interventional2003-12-31Completed
Idarubicin and Ara-C in Combination With Gemtuzumab-Ozogamicin (IAGO) for Young Untreated Patients, Without an HLA Identical Sibling, With High Risk MDS or AML Developing After a Preceding Period With MDS During 6 Months Duration: A Phase II Study [NCT00077116]Phase 231 participants (Actual)Interventional2003-11-30Completed
Fludarabine And Busulfan As Conditioning For Patients With Chronic Myeloid Leukemia Or Myelodysplastic Syndrome Transplanted With Hematopoietic Stem Cells From HLA-Compatible Related Or Unrelated Donors [NCT00027924]Phase 20 participants Interventional2001-10-31Completed
"Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation for the Treatment of Less Advanced Myelodysplasi" [NCT00024050]Phase 20 participants Interventional2001-02-28Completed
Non-Myeloablative Allogeneic Hematopoietic Cell Transplantation For Patients With Disease Relapse Or Myelodysplasia After Prior Autologous Transplantation [NCT00053196]Phase 282 participants (Actual)Interventional2002-12-31Completed
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 Single-arm, Single-center Trial of Prophylactic Tocilizumab for Acute GVHD Prevention After Haploidentical HSCT. [NCT04688021]Phase 246 participants (Anticipated)Interventional2020-12-03Recruiting
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
Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia [NCT00040469]Phase 215 participants Interventional2000-08-31Terminated(stopped due to accrual was slow and sporadic so the study was closed)
Hematopoietic Stem Cell Transplantation With Targeted Busulfan and Fludarabine in Pediatric AML [NCT01274195]Phase 227 participants (Anticipated)Interventional2011-01-31Recruiting
A Phase I/II Study of Immunologically Engineered rhG-CSF Mobilized Peripheral Blood Stem Cells (PBSC) for Allogeneic Transplant From HLA Identical, Related Donors for Treatment of Myeloid Malignancies [NCT00049634]Phase 1/Phase 230 participants (Anticipated)Interventional2002-01-31Completed
Reduced Intensity Allogeneic Stem Cell Transplantation With Matched Unrelated Donors for Patients With Hematologic Malignancies [NCT00818961]Phase 236 participants (Actual)Interventional2005-05-31Terminated(stopped due to terminated early due to meeting end point with fewer patients than anticipated)
Immunoablative Protocol for Allogeneic Related and Unrelated Hematopoietic Peripheral Blood Stem Cell Transplant (HPBSC) [NCT00179764]68 participants (Actual)Interventional2000-03-10Completed
Allogeneic and Matched Unrelated Donor Stem Cell Transplantation for Congenital Immunodeficiencies or Patients With Autoinflammatory/Immunodysregulatory Conditions: Busulfan-Based Conditioning With Campath- 1H or h-ATG, Radiation, and Sirolimus [NCT00426517]Early Phase 148 participants (Actual)Interventional2007-01-19Completed
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
Allogeneic Hematopoietic Cell Transplantation for Patients With Hematologic Disorders Who Are Undergoing Dose-Adjusted Treatment With A Maximally Intensive Busulfex-Based Therapeutic Regimen [NCT00448357]Phase 1/Phase 254 participants (Actual)Interventional2005-10-31Completed
A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With High Risk Hemoglobinopathy Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism [NCT00427661]8 participants (Actual)Interventional2002-06-30Completed
Phase I/II Trial of Fludarabine in Combination With Intravenous Busulfan and Allogeneic Progenitor Cell Support for Patients With Hematologic Malignancies [NCT00506857]Phase 1/Phase 282 participants (Actual)Interventional2003-11-30Completed
Cladribine Dose Escalation in Conditioning Regimen Prior to Allo-HSCT for Refractory Acute Leukemia and Myelodysplastic Syndromes [NCT03235973]Phase 129 participants (Anticipated)Interventional2018-04-28Recruiting
Busulfan (IV) and Fludarabine Followed by Post-allogeneic Transplantation Cyclophosphamide for Graft-versus-Host Disease Prophylaxis in Patients With Hematologic Malignancies. [NCT00800839]Phase 256 participants (Actual)Interventional2008-09-30Completed
Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis [NCT00775931]Phase 2/Phase 37 participants (Actual)Interventional2008-08-31Completed
Busulfan and Cyclophosphamide Followed By Allogeneic Hematopoietic Cell Transplantation In Patients With Hematological Malignancies [NCT01685411]5 participants (Actual)Interventional2013-01-31Terminated
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome [NCT05027945]Phase 237 participants (Anticipated)Interventional2023-02-23Recruiting
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis [NCT04384692]Phase 245 participants (Anticipated)Interventional2020-12-18Recruiting
An Multicenter, Randomized, Controlled, Prospective Clinical Study of Mitoxantrone Liposome Combined With PTCy as Conditioning Regimen in Allo-HSCT in Acute Leukemia [NCT05739630]Phase 2/Phase 360 participants (Anticipated)Interventional2023-01-01Recruiting
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Genetic Rare Disease [NCT02034630]Phase 1/Phase 25 participants (Anticipated)Interventional2014-07-21Recruiting
Rituximab, Methotrexate, Procarbazine and Vincristine Followed by High-dose Chemotherapy With Autologous Stem-cell Rescue in Newly-diagnosed Primary CNS Lymphoma (PCNSL) [NCT00596154]Phase 233 participants (Actual)Interventional2004-12-31Completed
A Phase II Study to Evaluate the Efficacy of Posttransplant Cyclophosphamide for Prevention of Chronic Graft-versus-Host Disease After Allogeneic Peripheral Blood Stem Cell Transplantation [NCT01427881]Phase 243 participants (Actual)Interventional2011-09-30Completed
Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders [NCT00176878]Phase 2/Phase 310 participants (Actual)Interventional2000-06-30Completed
A Pilot Study of Temozolomide and O-Benzylguanine for Treatment of High-Grade Glioma, Using Autologous Peripheral Blood Stem Cells Genetically Modified for Chemoprotection [NCT00253487]0 participants Interventional2005-08-31Completed
Transplantation of Umbilical Cord Blood From Related and Unrelated Donors [NCT00290628]43 participants (Actual)Interventional1999-10-31Terminated(stopped due to Replaced with another study)
European Infant Neuroblastoma Study Final Protocol [NCT00417053]Phase 30 participants InterventionalActive, not recruiting
Assessment of Respiratory Functions After a Conditioning Regimen With Busulfan for Allograft or Autograft in a Unicenter Pediatric and Prospective Population [NCT00328029]Phase 235 participants (Anticipated)Interventional2006-07-31Recruiting
Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission [NCT00305708]Phase 1/Phase 240 participants (Anticipated)Interventional2000-08-31Completed
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
Pharmacokinetic Study of Intravenous Busulfan as Conditioning Regimen for Allogeneic Stem Cell Transplantation in Adult Patients With Acute Leukemia [NCT01498016]Phase 260 participants (Anticipated)Interventional2011-11-30Recruiting
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)
Phase II Trial of Reduced Intensity Conditioning (RIC) and Allogeneic Transplantation of Umbilical Cord Blood (UCB) From Unrelated Donors in Patients With Hematologic Malignancies [NCT01622556]Phase 26 participants (Actual)Interventional2012-01-31Terminated
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
Clofarabine, Gemcitabine, and Busulfan Followed by Allogeneic Stem Cell Transplantation for Chronic Lymphocytic Leukemia (CLL) [NCT01629511]Phase 1/Phase 215 participants (Actual)Interventional2012-11-21Terminated
A Pilot Study of an Immunosuppressive and Myeloablative Preparative Regimen for Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation (HSCT) for Severe Sickle Cell Disease [NCT01279616]Phase 28 participants (Actual)Interventional2010-09-30Terminated(stopped due to PI moving to a different institution.)
Trial of Allogeneic BMT for Hematologic Malignancies Using HLA-matched Related or Unrelated Donors With Fludarabine and IV Busulfan as Pre-transplant Conditioning Followed by Post-transplant Immunosuppression With High-dose Cyclophosphamide [NCT00809276]Phase 1/Phase 292 participants (Actual)Interventional2009-05-31Completed
High Risk Neuroblastoma Study 1 of SIOP-Europe (SIOPEN) [NCT01704716]Phase 33,300 participants (Anticipated)Interventional2002-02-28Recruiting
Multi-center Single Arm Phase II Study of Myeloablative Allogeneic Stem Cell Transplantation for Non-remission Acute Myeloblastic Leukemia (AML) Using Clofarabine and Busulfan x 4 (CloBu4) Regimen [NCT01457885]Phase 275 participants (Actual)Interventional2011-11-30Completed
Reduced-Intensity Preparative Regiment With Fludarabine, Busulfan, And Alemtuzumab (Campath 1H) Followed By Allogeneic Hematopoietic Stem Cell Transplant For Malignant And Non-Malignant Hematological Diseases [NCT00579111]Phase 1/Phase 24 participants (Actual)Interventional2007-06-30Terminated(stopped due to slow accrual)
Cyclophosphamide-Busulfan Versus Busulfan-Cyclophosphamide as Conditioning Regimen Before Allogeneic Hematopoietic Stem Cell Transplantation for Leukemia: a Prospective Randomized Study to Assess Liver Toxicity [NCT01779882]72 participants (Actual)Interventional2013-01-31Completed
Allogeneic Transplantation After a Conditioning With Thiotepa, Busulfan and Fludarabin for the Treatment of Refractory/Early Relapsed Aggressive B-cell Non Hodgkin Lymphomas: a Phase II Multi-Center Trial [NCT01786018]Phase 242 participants (Anticipated)Interventional2013-02-28Recruiting
Evaluation of Plasmatic Levels of Busulfan in Patients Undergoing Hematopoietic Stem Cell Transplantation [NCT01800643]Phase 2/Phase 3100 participants (Anticipated)Interventional2010-03-31Recruiting
Phase II Study of Reduced Intensity Conditioning With Busulfan/Clofarabine Followed by Allogeneic Stem Cell Transplantation [NCT01643668]Phase 234 participants (Actual)Interventional2012-07-31Completed
Prospective, Phase II Randomized Study to Compare Busulfan-fludarabine Reduced-intensity Conditioning (RIC) With Thiotepa-fludarabine RIC Regimen Prior to Allogeneic Transplantation of Hematopoietic Cells for the Treatment of Myelofibrosis [NCT01814475]Phase 262 participants (Actual)Interventional2011-07-31Completed
Partially HLA Mismatched (Haploidentical) Allogeneic Bone Marrow Transplantation for Patients With Hematologic Malignancies [NCT01749293]Phase 23 participants (Actual)Interventional2012-08-30Terminated(stopped due to The protocol design is being reconfigured in order to open a new study.)
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With Secondary Acute Myeloid Leukemia Evolving From MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT05449899]Phase 2/Phase 3232 participants (Anticipated)Interventional2022-07-31Recruiting
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
Killer Immunoglobulin-like Receptor (KIR) Incompatible Unrelated Donor Hematopoietic Cell Transplantation (SCT) for AML With Monosomy 7, -5/5q-, High FLT3-ITD AR, or Refractory and Relapsed Acute Myelogenous Leukemia (AML) in Children: A Children's Oncolo [NCT00553202]Phase 2158 participants (Actual)Interventional2008-01-31Completed
Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation as Second Transplantation for Patients With Disease Relapse or Myelodysplasia After Prior Autologous Transplantation [NCT01118013]Phase 26 participants (Actual)Interventional2010-12-31Terminated
Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc) [NCT00684255]Phase 11 participants (Actual)Interventional2007-08-31Terminated(stopped due to inactive)
A Pilot Study Using High Dose Busulfan and Bortezomib as Part of Allogeneic Transplant Conditioning Regimen for High Risk Multiple Myeloma Patients. [NCT01534143]Phase 21 participants (Actual)Interventional2012-02-29Terminated(stopped due to Data was not collected, because funding was unavailable to continue study.)
ALLOGENEIC AND SYNGENEIC MARROW TRANSPLANTATION IN PATIENTS WITH ACUTE NON-LYMPHOCYTIC LEUKEMIA [NCT00002547]Phase 2280 participants (Actual)Interventional1987-08-31Completed
NK Cells With HLA Compatible Hematopoietic Transplantation for High Risk Myeloid Malignancies [NCT01823198]Phase 1/Phase 263 participants (Actual)Interventional2013-06-11Completed
T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant [NCT01621477]Phase 234 participants (Actual)Interventional2012-08-31Terminated(stopped due to Study was terminated in August 2016 due to replacement by a new study.)
A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells [NCT03538899]Phase 1/Phase 225 participants (Anticipated)Interventional2018-05-31Recruiting
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
T-Cell Depleted Double UCB With Post Transplant IL-2 for Refractory Myeloid Leukemia [NCT01464359]Phase 23 participants (Actual)Interventional2011-10-31Terminated(stopped due to Slow accrual)
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)
PRO#0118: A Phase I Study of Decitabin in Combination With Fludarabin and Busulfan as a Reduced Intensity Conditioning Regimen for the Treatment of Myeloid Malignancies [NCT01455506]Phase 120 participants (Actual)Interventional2009-05-31Completed
RADIOLABELED BC8 (ANTI-CD45) ANTIBODY COMBINED WITH BUSULFAN AND CYCLOPHOSPHAMIDE AS TREATMENT FOR ACUTE MYELOGENOUS LEUKEMIA IN FIRST OR SECOND REMISSION OR UNTREATED FIRST RELAPSE FOLLOWED BY HLA-IDENTICAL RELATED MARROW TRANSPLANTATION [NCT00002554]Phase 230 participants (Anticipated)Interventional1993-11-30Completed
Phase II Study of Timed Sequential Busulfan in Combination With Fludarabine in Allogeneic Stem Cell Transplantation [NCT01572662]Phase 2201 participants (Actual)Interventional2012-04-11Completed
Natural Killer (NK) Cells and Nonmyeloablative Stem Cell Transplantation for Chronic Myelogenous Leukemia (CML) [NCT01390402]Phase 26 participants (Actual)Interventional2012-01-31Completed
Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism [NCT00176852]Phase 2/Phase 322 participants (Actual)Interventional2002-06-30Completed
An International Randomised Clinical Trial of Therapeutic Interventions to Assess the Effects on Outcome in Adults With Acute Myeloid Leukaemia and High Risk Myelodysplasia Undergoing Allogeneic Stem Cell Transplantation. [NCT04217278]Phase 2/Phase 3333 participants (Actual)Interventional2020-01-27Active, not recruiting
Risk Stratification Directed Conditioning Regimen for Haploidentical HSCT in SAA [NCT03821987]55 participants (Anticipated)Interventional2018-12-17Recruiting
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 Study Of Allogeneic Transplant For Older Patients With AML In First Morphologic Complete Remission Using A Non-Myeloablative Preparative Regimen [NCT00070135]Phase 2121 participants (Actual)Interventional2004-01-31Completed
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)
A Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation [NCT01596699]Phase 216 participants (Actual)Interventional2012-05-24Terminated(stopped due to Triggering of futility rule; Interim analysis suggested no statistical superiority over historic controls)
The Therapeutic Window of Busulfan in Children With Haemopoietic Stem Cell Transplantation:A Multicenter Study in China [NCT04786002]500 participants (Anticipated)Observational2020-11-01Recruiting
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
ALLOGENEIC MARROW OR PERIPHERAL BLOOD STEM CELL TRANSPLANTATION FOR AGNOGENIC MYELOID METAPLASIA WITH MYELOFIBROSIS [NCT00002792]Phase 220 participants (Anticipated)Interventional1996-06-30Completed
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies [NCT01384513]Phase 240 participants (Actual)Interventional2011-08-04Completed
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
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
Phase 2 Study of a Reduced-toxicity Myeloablative Conditionning Regimen Using Fludarabine and Full Doses of iv Busulfan in Pediatric Patients Not Eligible for Standard Myeloablative Conditioning Regimens [NCT01572181]Phase 250 participants (Actual)Interventional2012-04-30Completed
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)
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
Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for TP53+ Myeloid Tumors Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT04123392]Phase 2/Phase 3196 participants (Anticipated)Interventional2019-10-31Recruiting
Allogeneic Stem Cell Transplantation With 3-days Busulfan Plus Fludarabine as Conditioning in Patients With Relapsed or Refractory T-, NK/T-cell Lymphomas [NCT02859402]Phase 234 participants (Anticipated)Interventional2016-12-31Recruiting
Phase III Study on Efficacy of Dose Intensification in Patients With Non-metastatic Ewing Sarcoma. [NCT02063022]Phase 3278 participants (Actual)Interventional2009-01-22Completed
In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders [NCT00176826]Phase 2/Phase 322 participants (Actual)Interventional2000-09-30Terminated(stopped due to Replaced by another protocol)
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation [NCT00176904]Phase 2/Phase 3135 participants (Actual)Interventional1995-01-31Completed
Tacrolimus and Sirolimus as Graft Versus Host Disease Prophylaxis After Allogeneic Non-myeloablative Peripheral Blood Stem Cell Transplantation [NCT00282282]Phase 231 participants (Actual)Interventional2006-01-31Completed
Busulfan Dose Escalation Study Based on AUC in the Setting of Busulfan/Fludarabine Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation (HCT) [NCT00361140]Phase 472 participants (Actual)Interventional2005-08-31Completed
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
Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide [NCT03910452]Early Phase 130 participants (Anticipated)Interventional2019-10-28Recruiting
Treatment Protocol for Relapsed Anaplastic Large Cell Lymphoma of Childhood and Adolescence [NCT00317408]96 participants (Anticipated)Interventional2004-04-30Active, not recruiting
Sirolimus and Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation [NCT00350181]Phase 211 participants (Actual)Interventional2006-08-31Completed
Conditioning For Hematopoietic Cell Transplantation With Fludarabine Plus Targeted IV Busulfan and GVHD Prophylaxis With Thymoglobulin, Tacrolimus and Methotrexate in Patients With Myeloid Malignancies [NCT00346359]Phase 240 participants (Anticipated)Interventional2006-03-31Completed
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)
AUTOLOGOUS STEM CELL TRANSPLANTATION FOR ACUTE MYELOID LEUKEMIA IN SECOND REMISSION: A PHASE II STUDY [NCT00002768]Phase 251 participants (Actual)Interventional1996-06-30Completed
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)
Allogeneic Peripheral Blood Progenitor Cell Transplantation Using Histocompatible Sibling-Matched Donor Cells After High-Dose Busulfan/Cyclophosphamide as Therapy for Hematologic Malignancies [NCT00003116]Phase 266 participants (Actual)Interventional1997-05-31Completed
A PHASE III STUDY IN CHILDREN WITH UNTREATED ACUTE MYELOGENOUS LEUKEMIA (AML) OR MYELODYSPLASTIC SYNDROME (MDS) [NCT00002798]Phase 3880 participants (Actual)Interventional1996-08-31Completed
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
INDUCTION WITH ALL-TRANS RETINOIC ACID IN COMBINATION WITH IDARUBICIN AND INTENSIVE CONSOLIDATION FOLLOWED BY BONE MARROW TRANSPLANTATION OR A RANDOMIZED MAINTENANCE TREATMENT DEPENDING UPON THE AMOUNT OF MINIMAL RESIDUAL DISEASE IN ACUTE PROMYELOCYTIC LE [NCT00002701]Phase 3750 participants (Anticipated)Interventional1995-10-31Active, not recruiting
CAMP-010: PHASE I/II STUDY OF IN VIVO PURGING FOLLOWED BY HIGH DOSE CHEMOTHERAPY, AUTOLOGOUS HEMATOPOIETIC STEM CELL INFUSION AND IMMUNOTHERAPY IN PATIENTS WITH CHRONIC MYELOID LEUKEMIA [NCT00002761]Phase 1/Phase 20 participants (Actual)Interventional1996-02-29Withdrawn(stopped due to PI left institution)
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 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
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Children and Adults With Hematologic Malignancies [NCT00003661]Phase 23 participants (Actual)Interventional1998-06-30Completed
A Multicenter Study of Unrelated Umbilical Cord Blood as an Alternate Source of Stem Cells for Transplantation [NCT00003913]Phase 2390 participants (Anticipated)Interventional1998-12-31Completed
T-Cell Depletion for Graft-Versus-Host Disease (GVHD) Prevention in High Risk Matched and Mismatched Allogeneic Bone Marrow Transplantation [NCT00005641]Phase 20 participants Interventional1997-09-30Terminated(stopped due to low study accrual)
Allogeneic Bone Marrow Transplantation for Hematologic Malignancies: A Treatment Approach Based on Risk of Relapse and Toxicity [NCT00005797]Phase 2125 participants (Actual)Interventional1993-03-31Completed
Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies [NCT00281879]Phase 2200 participants (Actual)Interventional2006-02-28Terminated
RANDOMIZED PHASE III STUDY OF INDUCTION (ICE VS MICE VS DCE) AND INTENSIVE CONSOLIDATION (IDIA VS NOVIA VS DIA) FOLLOWED BY BONE MARROW TRANSPLANTATION IN ACUTE MYELOGENOUS LEUKEMIA: AML 10 PROTOCOL [NCT00002549]Phase 31,520 participants (Anticipated)Interventional1993-11-30Active, not recruiting
PROSPECTIVE CONTROLLED STUDY FOR THE OPTIMIZATION OF THERAPY IN CHRONIC MYELOID LEUKEMIA (CML): MULTICENTRIC STUDY FOR THE EVALUATION OF INTERFERON ALPHA VS ALLOGENIC BM TRANSPLANTATION WITH CHEMOTHERAPY IN CML [NCT00002771]Phase 3750 participants (Anticipated)Interventional1995-01-31Active, not recruiting
A PHASE III RANDOMIZED STUDY COMPARING G-CSF MOBILIZED PERIPHERAL BLOOD STEM CELLS WITH MARROW AS THE SOURCE OF STEM CELLS FOR ALLOGENEIC TRANSPLANTS FROM HLA IDENTICAL, RELATED DONORS FOR THE TREATMENT OF CHRONIC MYELOID LEUKEMIA [NCT00002789]Phase 3100 participants (Anticipated)Interventional1996-05-31Completed
Randomized Trial of Busulfan or Total Body Irradiation Conditioning Regimens for Children With Acute Lymphoblastic Leukemia [NCT00002961]Phase 343 participants (Actual)Interventional1995-10-31Terminated(stopped due to poor accrual)
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
Gemcitabine/Clofarabine/Busulfan and Allogeneic Transplantation for Aggressive Lymphomas [NCT01701986]Phase 1/Phase 280 participants (Anticipated)Interventional2012-10-25Active, not recruiting
A Phase II Study of Total Marrow Irradiation, Busulfan, and Alpha-Interferon Followed by Allogeneic Peripheral Blood Stem Cell or Marrow Transplantation for Treatment of Patients With Advanced Multiple Myeloma. [NCT00003195]Phase 20 participants Interventional1997-12-31Completed
A Phase II Trial of Intensive Chemotherapy and Autotransplantation for Patients With Newly Diagnosed Anaplastic Oligodendroglioma [NCT00588523]Phase 260 participants (Actual)Interventional2002-09-30Completed
Autologous Bone Marrow Transplantation for Non-M3 Acute Myeloid Leukemia (AML) in First Remission in Patients NCT00534469]Phase 260 participants (Actual)Interventional2000-02-08Active, not recruiting
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT05453552]Phase 2/Phase 3242 participants (Anticipated)Interventional2022-07-01Recruiting
A Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haploidentical Peripheral Blood Stem Cell Transplantation [NCT03018223]Phase 132 participants (Actual)Interventional2017-01-31Completed
G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT04713956]Phase 2/Phase 3242 participants (Anticipated)Interventional2021-01-15Recruiting
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 Multi-center Phase III Study Comparing Myeloablative to Nonmyeloablative Transplant Conditioning in Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia [NCT00322101]Phase 325 participants (Actual)Interventional2006-01-31Completed
Reduced Toxicity Conditioning Prior to Unrelated Cord Cell Transplantation for High Risk Myeloid Malignancies [NCT02333838]Phase 231 participants (Actual)Interventional2015-05-31Active, not recruiting
Autologous Blood and Marrow Transplantation for Hematologic Malignancies and Selected Solid Tumors [NCT00536601]174 participants (Actual)Interventional2006-06-29Completed
Allogeneic Hematopoietic Stem Cell Transplantation as Initial Salvage Therapy for Patients With Primary Induction Failure Acute Myeloid Leukemia Refractory to High-Dose Cytarabine-Based Induction Chemotherapy [NCT02441803]Phase 211 participants (Actual)Interventional2015-09-14Active, not recruiting
A Phase 3, Randomized, Open-label, Active-Comparator-Controlled Clinical Study to Evaluate the Safety and Efficacy of Bomedemstat (MK-3543/IMG-7289) Versus Best Available Therapy (BAT) in Participants With Essential Thrombocythemia Who Have an Inadequate [NCT06079879]Phase 3300 participants (Anticipated)Interventional2023-11-27Not yet recruiting
FLUCLORIC: Randomized Multicentric Phase III Study Comparing the Efficacy of 2 Reduced Intensity Conditioning Regimens (Clofarabine/Busulfan vs Fludarabine/Busulfan) in Adults With AML and Eligible to Allogeneic Stem Cell Transplantation [NCT05917405]Phase 2302 participants (Anticipated)Interventional2023-09-14Recruiting
A Phase I/II Study of High-Dose Deoxyazacytidine, Busulfan, and Cyclophosphamide With Allogeneic Stem Cell Transplantation for Hematologic Malignancies [NCT00002831]Phase 1/Phase 224 participants (Actual)Interventional1995-08-01Completed
PROSPECTIVE RANDOMISED STUDY TO COMPARE INTERFERON-ALPHA-n1 (WELLFERON) VS 'IDAC' CHEMOTHERAPY AND AUTOGRAFTING FOLLOWED BY INTERFERON ALPHA-n1 (WELLFERON) IN PATIENTS WITH NEWLY DIAGNOSED CHRONIC PHASE CHRONIC MYELOID LEUKEMIA [NCT00002868]Phase 3744 participants (Anticipated)Interventional1997-11-20Completed
A Phase II Study of High-Dose Intravenous Busulfan and Fludarabine With Allogeneic Marrow and Peripheral Blood Progenitor Cell Transplantation for Acute Myeloid Leukemia and Myelodysplastic Syndromes [NCT00502905]Phase 2200 participants (Actual)Interventional2003-10-31Completed
Once Daily Intravenous Busulfex as Part of Reduced-toxicity Conditioning for Patients With Relapsed/Refractory Hodgkin's and Non-Hodgkin's Lymphomas Undergoing Allogeneic Hematopoietic Progenitor Cell Transplantation - A Multicenter Phase II Study [NCT01203020]Phase 222 participants (Actual)Interventional2010-10-12Completed
High-Dose Chemoradiotherapy With Stem Cell Allogeneic Cellular Rescue in Patients With Relapsed or Refractory Hematologic Malignancy - A Phase I/II Study [NCT00004907]Phase 1/Phase 20 participants Interventional1999-10-31Completed
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
A Randomized Phase II Study of Treosulfan, Fludarabine and Low-Dose TBI as Conditioning for Allogeneic Hematopoietic Cell Transplantation in Patients With Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML) [NCT01894477]Phase 2102 participants (Actual)Interventional2013-11-30Active, not recruiting
A Randomized Controlled Study on the Efficacy and Safety of MA-BUCY2 Protocol in the Conditioning of Haploidentical Stem Cell Transplantation in Patients With High-risk Acute Myeloid Leukemia [NCT05814731]264 participants (Anticipated)Interventional2023-04-15Not yet recruiting
ADA Gene Transfer Into Hematopoietic Stem/Progenitor Cells for the Treatment of ADA-SCID [NCT00598481]Phase 212 participants (Actual)Interventional2002-10-02Completed
A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells [NCT01512888]Phase 1/Phase 228 participants (Anticipated)Interventional2016-08-17Suspended(stopped due to voluntary hold)
Cyclophosphamide Followed by Intravenous Busulfan as Conditioning for Hematopoietic Cell Transplantation in Patients With Myelofibrosis, Acute Myeloid Leukemia, or Myelodysplastic Syndrome. [NCT00445744]52 participants (Actual)Interventional2006-12-31Completed
An Adaptive Phase I/II Study of the Safety of CD4+ T Lymphocytes and CD34+ Hematopoietic Stem/Progenitor Cells Transduced With LVsh5/C46, a Dual Anti-HIV Gene Transfer Construct, With and Without Conditioning With Busulfan in HIV-1 Infected Adults Previou [NCT01734850]Phase 1/Phase 213 participants (Actual)Interventional2013-04-30Completed
A Phase II Randomized Study to Assess Outcomes With Treosulfan-Based Versus Clofarabine-Based Conditioning in Patients With Myelodysplastic Syndromes With Excess Blasts (MDS-EB), or Acute Myeloid Leukemia (AML) Undergoing Allogeneic Hematopoietic Cell Tra [NCT04994808]Phase 280 participants (Anticipated)Interventional2023-08-11Recruiting
Neuroblastoma Protocol 2012: Therapy for Children With Advanced Stage High-Risk Neuroblastoma [NCT01857934]Phase 2153 participants (Actual)Interventional2013-07-05Active, not recruiting
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency [NCT03315078]Phase 1/Phase 213 participants (Anticipated)Interventional2012-04-30Recruiting
Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia [NCT00003816]Phase 2/Phase 3361 participants (Actual)Interventional1998-10-19Completed
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
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders [NCT04644016]Phase 231 participants (Anticipated)Interventional2020-11-20Recruiting
A Phase II Trial of Intensive Chemotherapy and Autotransplantation for Patients With Newly Diagnosed Anaplastic Oligodendroglioma [NCT00003101]Phase 260 participants (Anticipated)Interventional1997-08-31Completed
A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With High Risk Hematologic Malignancies [NCT00003335]Phase 244 participants (Actual)Interventional1998-01-31Completed
Haplocompatible Transplant Using TCRα/β Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) [NCT03597594]Phase 1/Phase 24 participants (Actual)Interventional2021-09-02Active, not recruiting
PRO#1278: A Phase III Study of Fludarabine and Busulfan Versus Fludarabine, Busulfan and Low Dose Total Body Irradiation in Patients Receiving an Allogeneic Hematopoietic Stem Cell Transplant [NCT01366612]Phase 353 participants (Actual)Interventional2010-06-16Terminated(stopped due to Lack of Accrual)
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, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1αS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals [NCT01380990]Phase 1/Phase 236 participants (Actual)Interventional2012-11-15Completed
Phase II Trial of Pentostatin and Targeted Busulfan as a Novel Reduced Intensity Regimen for Allogeneic Hematopoietic Stem Cell Transplantation Using Laboratory-Guided (CD4-guided) Immunosuppression. [NCT00496340]Phase 242 participants (Actual)Interventional2007-07-31Completed
Busulfan, Fludarabine, Donor Stem Cell Transplant, and Cyclophosphamide in Treating Participants With Multiple Myeloma or Myelofibrosis [NCT03303950]Phase 26 participants (Actual)Interventional2018-03-30Terminated(stopped due to Slow accrual)
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation [NCT00668564]Phase 218 participants (Actual)Interventional2008-03-31Terminated(stopped due to Replaced by another study)
Unrelated or Partially Matched Allogeneic Donor Stem Cells for Lymphoma, Myeloma, and Chronic Lymphocytic Leukemia [NCT00612716]Phase 26 participants (Actual)Interventional1999-10-06Completed
Pediatric Blood & Marrow Transplant Consortium (PBMTC) Phase II Myeloablative Haploidentical BMT With Post-transplantation Cyclophosphamide for Pediatric Patients With Hematologic Malignancies [NCT02120157]Phase 235 participants (Actual)Interventional2015-07-02Completed
Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies [NCT01499147]100 participants (Actual)Interventional2000-02-29Completed
A Pilot Study to Evaluate Efficacy and Safety of Multiplexed shRNA-modified CD34+ Cells in HIV-infected Patients. [NCT03517631]Phase 16 participants (Anticipated)Interventional2018-02-27Recruiting
Clofarabine Pre-conditioning Followed by Hematopoietic Stem Cell Transplant With Post-Transplant Cyclophosphamide for Non-remission Acute Myeloid Leukemia [NCT04002115]Phase 22 participants (Actual)Interventional2020-06-03Terminated(stopped due to terminated due to low accrual)
Safety and Feasibility of Gene Transfer After Frontline Chemotherapy for Non-Hodgkin Lymphoma in AIDS Patients Using Peripheral Blood Stem/Progenitor Cells Treated With a Lentivirus Vector-Encoding Multiple Anti-HIV RNAs [NCT01961063]Phase 13 participants (Anticipated)Interventional2015-12-31Active, not recruiting
Itacitinib to Prevent Graft Versus Host Disease [NCT04127721]Phase 20 participants (Actual)Interventional2020-09-22Withdrawn(stopped due to 0 ACTUAL Enrollment must have Overall Recruitment Status)
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
Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies [NCT04776850]Early Phase 10 participants (Actual)Interventional2020-12-29Withdrawn(stopped due to Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.)
Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA Complementary DNA (cDNA) by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) [NCT01852071]Phase 1/Phase 246 participants (Actual)Interventional2013-08-02Completed
Cord Blood Transplantation for Hematologic Malignancies and Bone Marrow Failure Syndromes [NCT00003270]Phase 220 participants (Actual)Interventional1997-09-04Completed
PRESERVE: A Multi-Center Trial Using Banked, Cryopreserved HLA-Mismatched Unrelated Donor Bone Marrow and Post-Transplantation Cyclophosphamide in Allogeneic Transplantation for Patients With Hematologic Malignancies [NCT05170828]Phase 10 participants (Actual)Interventional2022-09-30Withdrawn(stopped due to Change in Study Design)
Adoptive Transfer of NY-ESO-1 TCR Engineered Peripheral Blood Mononuclear Cells (PBMC) and Peripheral Blood Stem Cells (PBSC) After a Myeloablative Conditioning Regimen, With Administration of Interleukin-2, in Patients With Advanced Malignancies [NCT03240861]Phase 15 participants (Actual)Interventional2017-07-26Terminated(stopped due to slow accrual)
Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Mixed-lineage-leukemia (MLL)-Rearranged Acute Leukemia Undergoing Allogeneic Hematopoietic Stem Cell Transplantation [NCT03596892]Phase 2/Phase 3122 participants (Anticipated)Interventional2018-07-31Recruiting
Autologous Transplant as Treatment for Favorable or Intermediate Risk MRD-Negative AML Patients After Initial Induction Therapy [NCT03515707]Phase 20 participants (Actual)Interventional2018-07-10Withdrawn(stopped due to PI recommended closure)
A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine [NCT01071239]Phase 21 participants (Actual)Interventional2009-04-30Completed
Allogeneic Bone Marrow Transplantation for Patients With Chronic Myelogenous Leukemia in the Chronic Phase or Multiple Myeloma [NCT00004181]Phase 20 participants Interventional1999-10-31Completed
High-Risk Neuroblastoma Study 2 of SIOP-Europa-Neuroblastoma (SIOPEN) [NCT04221035]Phase 3800 participants (Anticipated)Interventional2019-11-05Recruiting
Reduced Intensity Conditioning Regimen for Elderly or High Comorbidity Burden Patients Receiving Haploidentical Hematopoietic Stem Cell Transplantation [NCT03412409]Phase 250 participants (Anticipated)Interventional2018-02-01Recruiting
Phase I/II Study of Sorafenib Added to Busulfan and Fludarabine Conditioning Regimen in Patients With Relapsed/Refractory AML Undergoing Stem Cell Transplantation [NCT03247088]Phase 1/Phase 274 participants (Anticipated)Interventional2017-07-30Recruiting
A Phase I/II Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplant for the Treatment of Patients With Hematologic Malignancies Using Busulfan, Fludarabine and Total Body Irradiation [NCT00245037]Phase 1/Phase 2147 participants (Actual)Interventional2005-06-30Completed
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
Phase I/II Study of Intravenous (IV) Busulfan and Etoposide (VP-16) Combined With Escalated Doses of Large Field Image-Guided Intensity Modulated Radiation Therapy (IMRT) Using Helical Tomotherapy as a Preparative Regimen for Allogeneic Hematopoietic Stem [NCT00540995]Phase 1/Phase 225 participants (Actual)Interventional2007-06-11Terminated(stopped due to Unable to safely escalate to TMLI doses that were hypothesized to be effective and less toxic than FTBI. Likely due to the giving of Busulfan prior to radiation delivery. Therefore, the study was abandoned and no further patients were accrued.)
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
Azacitidine/Vorinostat/GemBuMel With Autologous Stem-Cell Transplant (SCT) in Patients With Refractory Lymphomas [NCT01983969]Phase 1/Phase 261 participants (Actual)Interventional2013-11-07Completed
Decitabine Followed by Bone Marrow Transplant and High-Dose Cyclophosphamide for the Treatment of Relapsed and Refractory Acute Myeloid Neoplasms [NCT01707004]Phase 220 participants (Actual)Interventional2013-05-16Completed
Autologous Peripheral Blood Stem Cell Transplant for Acute Non-Lymphocytic Leukemia (ANLL) [NCT00630565]Phase 2/Phase 311 participants (Actual)Interventional2006-07-26Terminated(stopped due to Slow Accrual)
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

TrialOutcome
NCT00003199 (3) [back to overview]Event-free Survival
NCT00003199 (3) [back to overview]Overall Survival
NCT00003199 (3) [back to overview]Number of Participants With Toxicity of a Combination of Low-dose IL-2 and GM-CSF
NCT00003270 (1) [back to overview]Progression-free Survival
NCT00003816 (4) [back to overview]4 Year PFS
NCT00003816 (4) [back to overview]Toxicity/TRM at Day 100
NCT00003816 (4) [back to overview]CR Rate
NCT00003816 (4) [back to overview]4 yr OS
NCT00003875 (4) [back to overview]Toxicity Associated With High-dose Busulfan and Etoposide Followed by Stem Cell Rescue
NCT00003875 (4) [back to overview]Proportion of Patients Who Relapsed Associated With the Regimen
NCT00003875 (4) [back to overview]Overall Survival of Patients on Busulfan and Etoposide Followed by Stem Cell Rescue and Aldesleukin
NCT00003875 (4) [back to overview]Toxicity Associated With Aldesleukin Treatment After Stem Cell Rescue
NCT00004088 (2) [back to overview]Three-year Overall Survival
NCT00004088 (2) [back to overview]Progression-free Survival
NCT00049517 (3) [back to overview]Overall Survival (Induction Phase)
NCT00049517 (3) [back to overview]Disease-free Survival (Consolidation Phase)
NCT00049517 (3) [back to overview]Overall Survival (Consolidation Phase)
NCT00054327 (5) [back to overview]Rates of Durable Engraftment
NCT00054327 (5) [back to overview]Toxicity as Measured by CTC v2.0
NCT00054327 (5) [back to overview]Graft-versus-host Disease (GVHD)
NCT00054327 (5) [back to overview]Incidence of Recurrent Disease
NCT00054327 (5) [back to overview]Number of Patients With Overall Survival at 2 Years.
NCT00070135 (3) [back to overview]Non-relapse Mortality (NRM)
NCT00070135 (3) [back to overview]2 Year Disease Free Survival In Unrelated Donor Recipient Group
NCT00070135 (3) [back to overview]2 Year DFS for All Patients
NCT00134017 (5) [back to overview]Relapse
NCT00134017 (5) [back to overview]Chimerism
NCT00134017 (5) [back to overview]Days to Engraftment
NCT00134017 (5) [back to overview]Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD)
NCT00134017 (5) [back to overview]Non-relapse Mortality
NCT00153985 (3) [back to overview]Solid Organ Toxicity Related to the Conditioning Regimen.
NCT00153985 (3) [back to overview]Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.
NCT00153985 (3) [back to overview]The Incidence of Grade II-IV Acute Graft vs. Host Disease.
NCT00176826 (7) [back to overview]Number of Patients Surviving (Disease-free)
NCT00176826 (7) [back to overview]Number of Patients With Treatment Related Mortality.
NCT00176826 (7) [back to overview]Number of Patients With III-IV Graft-Versus-Host Disease (GVHD)
NCT00176826 (7) [back to overview]Number of Patients With Graft Failure
NCT00176826 (7) [back to overview]Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD)
NCT00176826 (7) [back to overview]Time to Transplant Engraftment
NCT00176826 (7) [back to overview]Number of Patients Surviving (Disease-free)
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 Engraftment
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 Long-term Disease-free Survival (DFS)
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 6 Months
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 100 Days
NCT00176852 (15) [back to overview]The Incidence of Chimerism at 1 Year
NCT00176852 (15) [back to overview]Overall Survival
NCT00176852 (15) [back to overview]The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
NCT00176852 (15) [back to overview]The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
NCT00176852 (15) [back to overview]Overall Survival
NCT00176852 (15) [back to overview]The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
NCT00176852 (15) [back to overview]Disease Free Survival
NCT00176852 (15) [back to overview]Disease Free Survival
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
NCT00176852 (15) [back to overview]Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
NCT00176852 (15) [back to overview]The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
NCT00176878 (6) [back to overview]Number of Patients With Succcessful Engraftment After Transplantation
NCT00176878 (6) [back to overview]Number of Patients With Disease Recurrence
NCT00176878 (6) [back to overview]Number of Patients With Chronic Graft Versus Host Disease
NCT00176878 (6) [back to overview]Number of Patients Alive (Survival) at 2 Years
NCT00176878 (6) [back to overview]Number of Patients With Grade 2-4 Acute Graft Versus Host Disease
NCT00176878 (6) [back to overview]Number of Patients Alive at Three Years (Survival)
NCT00176904 (5) [back to overview]Number of Patients With Chronic Graft-Versus-Host Disease
NCT00176904 (5) [back to overview]Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease
NCT00176904 (5) [back to overview]Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease
NCT00176904 (5) [back to overview]Overall Donor Engraftment
NCT00176904 (5) [back to overview]Overall Survival
NCT00176917 (4) [back to overview]Number of Patients Who Failed Engraftment.
NCT00176917 (4) [back to overview]Number of Patients Surviving on Study
NCT00176917 (4) [back to overview]Mean Percentage of Donor Cells in Study Population (Chimerism).
NCT00176917 (4) [back to overview]Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).
NCT00176930 (10) [back to overview]Number of Participants With Persistence or Relapse of Malignancy at 5 Years Post Transplant
NCT00176930 (10) [back to overview]Number of Participants With Persistence or Relapse of Malignancy at 2 Years Post Transplant
NCT00176930 (10) [back to overview]Number of Participants With Neutrophil Engraftment
NCT00176930 (10) [back to overview]Number of Participants With Chronic Graft-Versus-Host Disease
NCT00176930 (10) [back to overview]Number of Participants With Acute Graft-versus-host Disease (GVHD)
NCT00176930 (10) [back to overview]Number of Participants Who Were Alive at 5 Year Post Transplant
NCT00176930 (10) [back to overview]Number of Participants Who Were Alive at 2 Year Post Transplant
NCT00176930 (10) [back to overview]Number of Participants Experiencing Engraftment Failure
NCT00176930 (10) [back to overview]Number of Participants Experiencing Disease-Free Survival at 5 Years Post Transplant
NCT00176930 (10) [back to overview]Number of Participants Experiencing Disease-Free Survival at 2 Years Post Transplant
NCT00238433 (2) [back to overview]Disease-Free Survival
NCT00238433 (2) [back to overview]Therapy-Related Toxicities
NCT00245037 (7) [back to overview]Acute Graft-Versus-Host Disease (aGVHD) Outcome
NCT00245037 (7) [back to overview]Chronic Graft-Versus-Host Disease (cGVHD) Outcome
NCT00245037 (7) [back to overview]Non-relapse Mortality
NCT00245037 (7) [back to overview]Overall Survival
NCT00245037 (7) [back to overview]Progression-Free Survival
NCT00245037 (7) [back to overview]Relapse Mortality
NCT00245037 (7) [back to overview]Regimen-Related Toxicities
NCT00258427 (8) [back to overview]Number of Participants Experiencing Acute Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Chronic Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Chronic Graft-Versus-Host Disease
NCT00258427 (8) [back to overview]Number of Participants Experiencing Major Infections
NCT00258427 (8) [back to overview]Number of Participants Experiencing Overall Survival
NCT00258427 (8) [back to overview]Number of Participants Experiencing Relapse
NCT00258427 (8) [back to overview]Number of Participants Experiencing Graft Failure
NCT00258427 (8) [back to overview]Number of Participants Experiencing Acute Graft-Versus-Host Disease
NCT00265889 (3) [back to overview]Response Rate
NCT00265889 (3) [back to overview]Progression-free Survival
NCT00265889 (3) [back to overview]Number of Patients That Experience Pulmonary Toxicity
NCT00282282 (3) [back to overview]Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation
NCT00282282 (3) [back to overview]Disease Response.
NCT00282282 (3) [back to overview]Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus.
NCT00301834 (5) [back to overview]Disease-free Survival With Correction of Disease at One Year Post Transplantation
NCT00301834 (5) [back to overview]Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation
NCT00301834 (5) [back to overview]Cytomegalovirus (CMV) Viral Infection and Disease Symptoms
NCT00301834 (5) [back to overview]Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation
NCT00301834 (5) [back to overview]Treatment-related Mortality at 100 Days and 1 Year Post Transplantation
NCT00322101 (6) [back to overview]Donor Cell Engraftment
NCT00322101 (6) [back to overview]Incidence and Severity of Acute and Chronic Graft-vs-host Disease
NCT00322101 (6) [back to overview]Incidence of Disease Progression/Relapse
NCT00322101 (6) [back to overview]Overall Survival
NCT00322101 (6) [back to overview]Progression-free Survival
NCT00322101 (6) [back to overview]Non-relapse Mortality
NCT00357396 (1) [back to overview]Overall Objective Response
NCT00361140 (2) [back to overview]Non-relapse Mortality
NCT00361140 (2) [back to overview]Severe Venous Occlusive Disease (VOD)/ Sinusoidal Obstructive Syndrome (SOS)
NCT00363467 (5) [back to overview]1 Year Event-free Survival
NCT00363467 (5) [back to overview]1 Year Overall Survival
NCT00363467 (5) [back to overview]100-day Non-relapse Mortality
NCT00363467 (5) [back to overview]Severe Regimen-related Toxicity
NCT00363467 (5) [back to overview]Successful Autologous Stem Cell Collection
NCT00416598 (2) [back to overview]Number of Participants Who Completed Maintenance Decitabine.
NCT00416598 (2) [back to overview]Disease-free Survival (DFS) Rate at 1 Year
NCT00426517 (8) [back to overview]Days to Absolute Neutrophil Recovery (ANC)
NCT00426517 (8) [back to overview]Participants With Established Stable Mixed Chimerism
NCT00426517 (8) [back to overview]Stem Cell Transplant Engraftment
NCT00426517 (8) [back to overview]Engraftment Without Development of GVHD
NCT00426517 (8) [back to overview]Incidence of Cytomegalovirus (CMV) Disease
NCT00426517 (8) [back to overview]Number of RBC Transfusions Per Subject
NCT00426517 (8) [back to overview]Days to Platelet Recovery
NCT00426517 (8) [back to overview]Days to CD3 Count Greater Than 100 u/L
NCT00427661 (3) [back to overview]Development of GVHD Within 1 Year of BMT
NCT00427661 (3) [back to overview]Engraftment at 1 Year Post BMT.
NCT00427661 (3) [back to overview]Incidence of Grade 2-4 Acute GVHD.
NCT00427765 (1) [back to overview]Average Overall Survival Time
NCT00445744 (2) [back to overview]Effectiveness of Cyclophosphamide/Busulfan Regimen in Reducing Regimen-related Liver Toxicity
NCT00445744 (2) [back to overview]Non-relapse Mortality (NRM) (Patients With AML/MDS)
NCT00448201 (4) [back to overview]5-year Disease-free Survival
NCT00448201 (4) [back to overview]Treatment-related Mortality
NCT00448201 (4) [back to overview]Complete or Mixed Donor Chimerism at 30, 60, and 90 Days Post-transplant
NCT00448201 (4) [back to overview]Graft-vs-host Disease at 6 Months Post-transplant
NCT00448357 (6) [back to overview]Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant
NCT00448357 (6) [back to overview]Number of Participants With Dose Limiting Toxicities (DLTs)
NCT00448357 (6) [back to overview]Overall Survival
NCT00448357 (6) [back to overview]Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen
NCT00448357 (6) [back to overview]Incidence of DNA Chimerism in Patients Between One Month Post Transplant
NCT00448357 (6) [back to overview]Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912)
NCT00469014 (1) [back to overview]Treatment Related Mortality
NCT00469144 (2) [back to overview]3 Year Progression Free Survival
NCT00469144 (2) [back to overview]Treatment-related Mortality (TRM)
NCT00475020 (2) [back to overview]Efficacy of This Therapy 3 Years Post-transplant
NCT00475020 (2) [back to overview]Rate of Non-relapse Mortality at 100 Days Post-transplant
NCT00478244 (10) [back to overview]Number of Patients With Neutrophil Engraftment
NCT00478244 (10) [back to overview]Overall Survival
NCT00478244 (10) [back to overview]Number of Patients With Transplant-Related Mortality
NCT00478244 (10) [back to overview]Number of Patients With Resistance to Blister Formation
NCT00478244 (10) [back to overview]Number of Patients With Platelet Engraftment
NCT00478244 (10) [back to overview]Number of Patients With Detectable Collagen Type VII
NCT00478244 (10) [back to overview]Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)
NCT00478244 (10) [back to overview]Number of Patients With Acute Graft-Versus-Host Disease (GVHD)
NCT00478244 (10) [back to overview]Number of Patients With Donor Derived Cells in Skin
NCT00478244 (10) [back to overview]Number of Patients With >70% Donor Chimerism
NCT00496340 (10) [back to overview]Incidence of Greater Than or Equal to 50% Donor Chimerism
NCT00496340 (10) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT00496340 (10) [back to overview]Cumulative Incidence of Hematopoietic Cell Engraftment
NCT00496340 (10) [back to overview]Rate of T-cell (CD3+) and Myeloid (CD33+) Chimerism by Day +28
NCT00496340 (10) [back to overview]Time to Incidence of Graft Versus Host Disease (GVHD)
NCT00496340 (10) [back to overview]Incidence of Infections
NCT00496340 (10) [back to overview]Non-relapse Mortality Rate (NRM)
NCT00496340 (10) [back to overview]Rate of T-cell (CD3+) and Myeloid (CD33+) Chimerism by Day +100
NCT00496340 (10) [back to overview]Percentage of Participants With Progression Free Survival (PFS)
NCT00496340 (10) [back to overview]Incidence of Graft Versus Host Disease (GVHD)
NCT00499889 (3) [back to overview]Participants' With mCR Response to Post Transplant DLI
NCT00499889 (3) [back to overview]Number of Participants in Complete Molecular Remission at 1 Year
NCT00499889 (3) [back to overview]Participants' With mCR Response to Post Transplant Imatinib Mesylate Therapy
NCT00502905 (1) [back to overview]Number of Participants With Successful Engraftment
NCT00506857 (2) [back to overview]Maximum Tolerated Dose (MTD)
NCT00506857 (2) [back to overview]Number of Participants With Graft Versus Host Disease (GVHD)
NCT00513474 (3) [back to overview]Number of Participant With Adverse Events (AE)
NCT00513474 (3) [back to overview]Uric Acid Levels
NCT00513474 (3) [back to overview]Percentage of Participants With Grades II to IV Acute Graft-Versus-Host Disease (aGVHD)
NCT00534430 (4) [back to overview]Overall Survival Comparing Diagnosis Groups
NCT00534430 (4) [back to overview]Cumulative Incidence of Relapse With Transplant-related Death as the Competing Risk: Diagnosis Groups Are Compared.
NCT00534430 (4) [back to overview]Overall Survival at 5 Years Post-Transplant.
NCT00534430 (4) [back to overview]Disease-free Survival at Five Years Post-transplant
NCT00534469 (2) [back to overview]Disease-Free Survival at 2-Year Post-Transplant
NCT00534469 (2) [back to overview]Disease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk
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]Response Rate (Complete Remission)
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]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]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)
NCT00540995 (1) [back to overview]Maximum Tolerated Dose (MTD) of Intensity-modulated Radiation Therapy (Phase I)
NCT00544115 (2) [back to overview]Two-year Overall Survival
NCT00544115 (2) [back to overview]Neutrophil Engraftment - The Days Till ANC Recovery
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)
NCT00553202 (2) [back to overview]Cumulative Incidence of NK Cell Reconstitution
NCT00553202 (2) [back to overview]Overall Survival (OS)
NCT00555048 (7) [back to overview]Lowest Dose of Alemtuzumab Associated With Transplant-related Mortality
NCT00555048 (7) [back to overview]Disease Relapse
NCT00555048 (7) [back to overview]Extensive Chronic GVHD
NCT00555048 (7) [back to overview]Grades III-IV Acute Graft-vs-host Disease (GVHD)
NCT00555048 (7) [back to overview]Graft Failure
NCT00555048 (7) [back to overview]Life-threatening Infection
NCT00555048 (7) [back to overview]Overall Survival
NCT00556452 (4) [back to overview]Regimen Related Toxicities
NCT00556452 (4) [back to overview]Two-year Overall Survival for All Cases.
NCT00556452 (4) [back to overview]One-year Overall Survival Rate for AML
NCT00556452 (4) [back to overview]Five Year Overall Survival for All Cases
NCT00578292 (8) [back to overview]Engraftment Rate After Transplant
NCT00578292 (8) [back to overview]Immune Reconstitution
NCT00578292 (8) [back to overview]Hematopoietic Reconstitution
NCT00578292 (8) [back to overview]Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)
NCT00578292 (8) [back to overview]Number of Participants With CHRONIC GVHD
NCT00578292 (8) [back to overview]Event-free Survival
NCT00578292 (8) [back to overview]Number of Participants With Infectious Complications
NCT00578292 (8) [back to overview]Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)
NCT00578344 (2) [back to overview]Number of Participants With Immune Response to Immunization After BMT in Participants With SCD, Hemoglobin SC, or Hemoglobin Sb0/+.
NCT00578344 (2) [back to overview]Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan.
NCT00578643 (2) [back to overview]Number of Patients That Have Complete Donor Chimerism After Transplant.
NCT00578643 (2) [back to overview]Percentage of Participants With Engraftment
NCT00579111 (2) [back to overview]Number of Patients With Successful Donor Engraftment
NCT00579111 (2) [back to overview]Number of Patients With Treatment Related Grade III or IV Non-hematological Toxicity
NCT00582894 (4) [back to overview]Number of Participants Experiencing Engraftment Donor Chimerism (EDC)
NCT00582894 (4) [back to overview]Number of Participants Experiencing Transplant Related Mortality (TRM)
NCT00582894 (4) [back to overview]Number of Participants Overall Survival as a Function of Time.
NCT00582894 (4) [back to overview]Number of Participants Relapse-Free
NCT00582933 (1) [back to overview]Death From GVHD
NCT00588523 (2) [back to overview]Number of Participants Evaluated for Toxicities
NCT00588523 (2) [back to overview]Progession Free Survival
NCT00598481 (3) [back to overview]CD3+ Cell Counts
NCT00598481 (3) [back to overview]Rate of Severe Infections
NCT00598481 (3) [back to overview]Survival
NCT00611351 (4) [back to overview]Transplantation-related Mortality at 100 Days Post-transplantation
NCT00611351 (4) [back to overview]Overall Survival
NCT00611351 (4) [back to overview]Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
NCT00611351 (4) [back to overview]Event-free Survival
NCT00612716 (9) [back to overview]Number of Participants With Relapse of Malignancy
NCT00612716 (9) [back to overview]Number of Participants With Platelet Engraftment
NCT00612716 (9) [back to overview]Number of Participants With Persistence Disease
NCT00612716 (9) [back to overview]Number of Participants With Neutrophil Engraftment
NCT00612716 (9) [back to overview]Number of Participants With Grade 3-4 Acute Graft-versus-host Disease
NCT00612716 (9) [back to overview]Number of Participants With Engraftment Failure
NCT00612716 (9) [back to overview]Number of Participants With 2 Year Overall Survival
NCT00612716 (9) [back to overview]Number of Participants With 1 Year Overall Survival
NCT00612716 (9) [back to overview]Number of Participants With of Chronic GVHD.
NCT00615589 (5) [back to overview]The Percentage of Patients Alive 1 Year Post Transplant
NCT00615589 (5) [back to overview]The Percentage of Patients Free From Progression at 1 Year
NCT00615589 (5) [back to overview]Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression
NCT00615589 (5) [back to overview]Percentage of Patients With Treatment Related Mortality (TRM)
NCT00615589 (5) [back to overview]Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)
NCT00619645 (2) [back to overview]Number of Patients With Day 100 Transplant-related Mortality
NCT00619645 (2) [back to overview]Number of Patients Alive 24 Months Post Day 100 Transplant
NCT00623935 (2) [back to overview]Percentage of Participants With Relapse Free Survival at 1 Year
NCT00623935 (2) [back to overview]Percentage of Participants Alive at 1 Year
NCT00638820 (3) [back to overview]Number of Patients With Transplant Related Toxicity
NCT00638820 (3) [back to overview]Number of Patients With Transplant Related Death
NCT00638820 (3) [back to overview]Number of Patients Achieving Donor Cell Engraftment
NCT00668564 (2) [back to overview]Number of Patients Achieving Engraftment
NCT00668564 (2) [back to overview]Overall Survival
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]Safety, as Defined by Serious Adverse Events and Adverse Events Related to Study Treatment.
NCT00691015 (8) [back to overview]Severity of Acute Graft-versus-host Disease (GVHD)
NCT00691015 (8) [back to overview]Time to Engraftment (i.e., Absolute Neutrophil Recovery [ANC > 500/mm³] )
NCT00691015 (8) [back to overview]Overall Survival.
NCT00691015 (8) [back to overview]Karnofsky Performance Status Performance Status
NCT00775931 (4) [back to overview]Transplant Related Toxicity
NCT00775931 (4) [back to overview]Transplant Related Mortality at 100 Days
NCT00775931 (4) [back to overview]Number of Patients Who Achieved Donor Cell Engraftment
NCT00775931 (4) [back to overview]Incidence of Grade II - IV Acute Graft-versus-host Disease
NCT00782379 (11) [back to overview]Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)
NCT00782379 (11) [back to overview]Number of Patients Who Experienced Severe Graft-versus-host Disease (GVHD)(Grade 3 or 4)
NCT00782379 (11) [back to overview]Overall Survival at 12 Months
NCT00782379 (11) [back to overview]Overall Survival at Day 100
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 60 Post-transplantation
NCT00782379 (11) [back to overview]Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 90 Post-transplantation
NCT00782379 (11) [back to overview]Disease Free Survival at 12 Months
NCT00782379 (11) [back to overview]Disease Free Survival at Day 100
NCT00782379 (11) [back to overview]Incidence of Graft Rejection for Patients at Day 100
NCT00782379 (11) [back to overview]Non-relapse Mortality at 1 Year After Peripheral Blood Stem Cell Transplantation (PBSCT)
NCT00787761 (8) [back to overview]T-cell and Myeloid Chimerism at Days 180 Post-transplantation (>90%)
NCT00787761 (8) [back to overview]T-cell and Myeloid Chimerism at Days 90 Post-transplantation (>90% Chimerism)
NCT00787761 (8) [back to overview]Disease-free Survival (DFS) at 24 Months
NCT00787761 (8) [back to overview]Number of Patients Who Experience Severe (Grade 3 or 4) Acute Graft-versus-host Disease
NCT00787761 (8) [back to overview]Number of Patients Experiencing Extensive Chronic Graft Versus Host Disease (GVHD)
NCT00787761 (8) [back to overview]Non-relapse Mortality (NRM) at Day 180 Post-transplantation
NCT00787761 (8) [back to overview]Overall Survival (OS) at 24 Months
NCT00787761 (8) [back to overview]Achievement of > 90% (Full) Donor Chimerism in the T-cell Lineage as Measured by PCR at Day 30 Post-transplantation
NCT00795132 (3) [back to overview]Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments
NCT00795132 (3) [back to overview]Number of Participants Who Experienced Transplantation-related Mortality (TRM)
NCT00795132 (3) [back to overview]Two Year Overall Survival
NCT00796068 (12) [back to overview]Non-relapse Mortality
NCT00796068 (12) [back to overview]Incidence of Clinically Significant Infections
NCT00796068 (12) [back to overview]Incidence of Acute or Chronic Graft-versus-host Disease (GVHD)
NCT00796068 (12) [back to overview]Duration (Days) Until Participants Obtained Platelet Engraftment
NCT00796068 (12) [back to overview]Number of Participants Surviving up to 2 Years Without Disease Progression
NCT00796068 (12) [back to overview]The Number of Participants Alive at Two-years Follow up.
NCT00796068 (12) [back to overview]Number of Patients With Non-relapse Mortality (NRM)
NCT00796068 (12) [back to overview]Number of Participants With Secondary Graft Failure
NCT00796068 (12) [back to overview]Number of Participants With Graft Failure/Rejection
NCT00796068 (12) [back to overview]Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100
NCT00796068 (12) [back to overview]Incidence of Relapse or Disease Progression
NCT00796068 (12) [back to overview]Number of Participants Surviving by 1 Year
NCT00796562 (6) [back to overview]Engraftment as Measured by Donor Chimerism
NCT00796562 (6) [back to overview]Survival
NCT00796562 (6) [back to overview]Non-relapse Mortality
NCT00796562 (6) [back to overview]Acute GVHD
NCT00796562 (6) [back to overview]Chronic GVHD
NCT00796562 (6) [back to overview]Relapse
NCT00800839 (6) [back to overview]2-year Progression-Free Survival
NCT00800839 (6) [back to overview]Day-100 Treatment-Related Mortality
NCT00800839 (6) [back to overview]Cumulative Incidence of Grade III to IV Acute GVHD
NCT00800839 (6) [back to overview]Cumulative Incidence of Grade II to IV Acute GVHD
NCT00800839 (6) [back to overview]2-year Overall Survival
NCT00800839 (6) [back to overview]Rate of Engraftment
NCT00802113 (7) [back to overview]Time to Platelet Engraftment
NCT00802113 (7) [back to overview]Total Number of Subjects That Experienced Transplant-related Mortality (TRM)
NCT00802113 (7) [back to overview]Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT)
NCT00802113 (7) [back to overview]Total Number of Subjects With Partial Response or Stable Disease Following MAC AutoSCT
NCT00802113 (7) [back to overview]Total Number of Subjects With Grade II-IV Acute Graft-versus-Host-Disease (GVHD)
NCT00802113 (7) [back to overview]Time to Neutrophil Engraftment
NCT00802113 (7) [back to overview]Total Number of Subjects With a Disease Relapse or Progression Following MAC AutoSCT
NCT00809276 (1) [back to overview]To Determine the Optimal Regimen of Post-graft Immunosuppression With High-dose Cy Following Fludarabine, Busulfan, and Transplantation of Fully HLA-matched Bone Marrow That Leads to an Acceptable Incidence of Grades III/IV Acute GVHD
NCT00813124 (1) [back to overview]Number of Participants With Molecular Response
NCT00818961 (11) [back to overview]Complete Donor Chimerism
NCT00818961 (11) [back to overview]Neutrophil Recovery
NCT00818961 (11) [back to overview]Number of Patients Requiring the Use of Donor Leukocyte Infusion (DLI) for Early Mixed T-cell Chimerism
NCT00818961 (11) [back to overview]Overall Survival at 1 Year
NCT00818961 (11) [back to overview]Platelet Engraftment
NCT00818961 (11) [back to overview]Survival at Day 100
NCT00818961 (11) [back to overview]Non-relapse Mortality at Day 100
NCT00818961 (11) [back to overview]Number of Patients Experiencing Chronic Graft Versus Host Disease
NCT00818961 (11) [back to overview]Number of Patients Experiencing Grade 2-4 Acute Graft-versus-host Disease Post-transplant
NCT00818961 (11) [back to overview]Non-relapse Mortality at 1 Year Post-transplant
NCT00818961 (11) [back to overview]Number of Patients Experiencing Veno-occlusive Disease (VOD) Post-transplant
NCT00822770 (3) [back to overview]Number of Participants Alive With no Disease Progression at Time of Allo Transplant
NCT00822770 (3) [back to overview]Number of Participants With Grade 4 Dose Limiting Toxicity to Determine Maximum Tolerated Dose (MTD) Plerixafor
NCT00822770 (3) [back to overview]Engraftment Response Rate: Number of Transplanted Participants With Complete Chimerism at Day 30
NCT00857389 (7) [back to overview]Relapse Rate of Participants Treated With Thiotepa, Busulfan, and Clofarabine
NCT00857389 (7) [back to overview]Overall Survival Rate
NCT00857389 (7) [back to overview]Number of Participants With Serious Adverse Events
NCT00857389 (7) [back to overview]Number of Participants With Disease Free Survival
NCT00857389 (7) [back to overview]Graft vs Host Disease (GVHD)
NCT00857389 (7) [back to overview]Engraftment
NCT00857389 (7) [back to overview]Number of Participants With Survival Rate at 100 Days Post-transplant
NCT00919503 (10) [back to overview]Number of Patients With Grade II-IV Acute Graft-versus-host Disease
NCT00919503 (10) [back to overview]Immune Reconstitution Following Hematopoietic Cell Transplantation
NCT00919503 (10) [back to overview]Disease Response at One Year Following Hematopoietic Cell Transplantation
NCT00919503 (10) [back to overview]Non-relapse Mortality
NCT00919503 (10) [back to overview]Number of Participants With Infections
NCT00919503 (10) [back to overview]Number of Patients With of Chronic Graft-versus-host Disease
NCT00919503 (10) [back to overview]Overall Survival
NCT00919503 (10) [back to overview]Preliminary Efficacy
NCT00919503 (10) [back to overview]Donor Chimerism CD3 at 100 Days Post Transplant
NCT00919503 (10) [back to overview]Donor Chimerism CD33 at Day 100 Post Transplant
NCT00941720 (3) [back to overview]Relapse-free Survival
NCT00941720 (3) [back to overview]Pulmonary Toxicity
NCT00941720 (3) [back to overview]Overall Survival
NCT00943319 (3) [back to overview]Maximum Tolerated Dose
NCT00943319 (3) [back to overview]Disease Free Survival
NCT00943319 (3) [back to overview]Overall Survival
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
NCT00948090 (4) [back to overview]Number of Transplant-related Death Events Until Day 100.
NCT00948090 (4) [back to overview]Number of Progression Events in 2 Years.
NCT00948090 (4) [back to overview]Overall Response Rate
NCT00948090 (4) [back to overview]Number of Death Events in 2 Years.
NCT00950846 (6) [back to overview]Number of Participants With Chronic GvHD
NCT00950846 (6) [back to overview]Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT00950846 (6) [back to overview]Number of Participants With Donor Engraftment After Transplant.
NCT00950846 (6) [back to overview]Incidence of Severe Grade III-IV Acute GvHD at Day 100.
NCT00987480 (4) [back to overview]Overall Survival at 3 Years
NCT00987480 (4) [back to overview]Disease-free Survival at 3 Years
NCT00987480 (4) [back to overview]The Incidence of Acute GvHD
NCT00987480 (4) [back to overview]The Incidence of Early Transplant Related Mortality
NCT00990249 (1) [back to overview]Treatment-Related Mortality (TRM) Defined as Non Relapse Mortality (NRM)
NCT01009840 (10) [back to overview]Percentage of Participants With Overall Disease Response at Month 6
NCT01009840 (10) [back to overview]Ratio Area Under Curve (AUC)/Target AUC
NCT01009840 (10) [back to overview]Progression-free Survival
NCT01009840 (10) [back to overview]Percentage of Participants With Transplant-Related Mortality
NCT01009840 (10) [back to overview]Overall Survival
NCT01009840 (10) [back to overview]Percentage of Participants With Progression-free Survival Events
NCT01009840 (10) [back to overview]Percentage of Participants With Hepatic Veno-Occlusive Disease Based on Baltimore Criteria
NCT01009840 (10) [back to overview]Percent Difference Between Area Under Curve (AUC) and Target AUC
NCT01009840 (10) [back to overview]Percent Change in IV Busulfan Dose
NCT01009840 (10) [back to overview]Percentage of Participants With Overall Survival Events
NCT01027000 (1) [back to overview]2-year Progression-free Survival in Early Disease Participants
NCT01043640 (12) [back to overview]Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)
NCT01043640 (12) [back to overview]Donor Cell Chimerism Following Transplant
NCT01043640 (12) [back to overview]Donor Cell Chimerism Following Transplant
NCT01043640 (12) [back to overview]Donor Cell Chimerism Following Transplant
NCT01043640 (12) [back to overview]Donor Cell Chimerism Following Transplant
NCT01043640 (12) [back to overview]Number of Patients Who Died Peri-Transplant
NCT01043640 (12) [back to overview]Donor Cell Chimerism Following Transplant
NCT01043640 (12) [back to overview]Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)
NCT01043640 (12) [back to overview]Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)
NCT01043640 (12) [back to overview]Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
NCT01043640 (12) [back to overview]Number of Patients With Donor Derived Engraftment
NCT01043640 (12) [back to overview]Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
NCT01044745 (4) [back to overview]Number of Participants With Grades II-IV Acute GVHD
NCT01044745 (4) [back to overview]Event-free Survival
NCT01044745 (4) [back to overview]Overall Survival
NCT01044745 (4) [back to overview]Transplant-related Mortality (TRM)
NCT01093586 (14) [back to overview]Overall Survival
NCT01093586 (14) [back to overview]Recurrence or Relapse
NCT01093586 (14) [back to overview]Recurrence or Relapse
NCT01093586 (14) [back to overview]Toxicity Related to UCB Transplantation and Cytoreduction as Assessed by CTC v3.0
NCT01093586 (14) [back to overview]Incidence of Acute Graft-versus-host Disease (GVHD)
NCT01093586 (14) [back to overview]Hematologic Engraftment
NCT01093586 (14) [back to overview]Overall Survival
NCT01093586 (14) [back to overview]Disease Free
NCT01093586 (14) [back to overview]Disease Free
NCT01093586 (14) [back to overview]Incidence of Chronic GVHD
NCT01093586 (14) [back to overview]Transplant Related Mortality
NCT01093586 (14) [back to overview]Transplant Related Mortality
NCT01093586 (14) [back to overview]Occurrence of Serious Infections
NCT01093586 (14) [back to overview]Overall Survival
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]Survival and Disease-free Survival (DFS)
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.
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]Number of Participants With Acute and Chronic GVHD Following T-cell Depleted, CD34+ Progenitor Cell Enriched Transplants 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
NCT01135329 (1) [back to overview]Graft Failure
NCT01168219 (3) [back to overview]100-day Mortality
NCT01168219 (3) [back to overview]Overall Survival (OS)
NCT01168219 (3) [back to overview]Progression-free Survival
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]Percentage of MIBG Avid Patients Treated With Meta-iodobenxylguanide (MIBG) Labeled With Iodine-131
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
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for All Participants
NCT01181271 (12) [back to overview]Cumulative Incidence of Grades II to IV Acute Graft Versus Host Disease (GVHD)
NCT01181271 (12) [back to overview]Cumulative Incidence of Extensive Chronic Graft-versus-host-disease
NCT01181271 (12) [back to overview]Cumulative Incidence of Disease Relapse
NCT01181271 (12) [back to overview]Peripheral Blood All-cell Donor Chimerism
NCT01181271 (12) [back to overview]Number of Days After Allogeneic Transplant Until Absolute Neutrophil Count Was Equal to or Greater Than 500/uL
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for Participants Undergoing Only Autologous Transplant
NCT01181271 (12) [back to overview]Estimated Two Year Overall Survival Rate for Participants Undergoing Only Autologous Transplant
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for All Participants
NCT01181271 (12) [back to overview]Cumulative Incidence of Non-relapse Mortality
NCT01181271 (12) [back to overview]Estimated Two Year Progression Free Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants
NCT01200329 (2) [back to overview]Event-free Survival (EFS) of Patients
NCT01200329 (2) [back to overview]Overall Survival (OS) of These Patients.
NCT01203020 (9) [back to overview]Non-Relapse Mortality (NRM) Following RTC Transplantation at 1 Year.
NCT01203020 (9) [back to overview]Non-Relapse Mortality (NRM) Following RTC Transplantation at 2 Years.
NCT01203020 (9) [back to overview]Percentage of 1 Year Overall Survival (OS)
NCT01203020 (9) [back to overview]Rates of Acute and Chronic Graft Versus Host Disease (GVHD).
NCT01203020 (9) [back to overview]Relapse Rate (RR) Following Transplantation at 2-year.
NCT01203020 (9) [back to overview]Relapse Rate (RR) Following Transplantation at 1-year.
NCT01203020 (9) [back to overview]Percentage of 2-year Progression Free Survival (PFS)
NCT01203020 (9) [back to overview]Percentage of 2 Year Overall Survival (OS)
NCT01203020 (9) [back to overview]Percentage of 1-year Progression Free Survival (PFS)
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]Percent of Participants Dying From Treatment-Related Complications
NCT01237951 (4) [back to overview]Progression-free Survival (PFS)
NCT01237951 (4) [back to overview]Overall Survival
NCT01247701 (7) [back to overview]Number of Participants With Platelet Engraftment
NCT01247701 (7) [back to overview]Number of Participants With Neutrophil Engraftment
NCT01247701 (7) [back to overview]Number of Participants With Relapse Rate After Transplant
NCT01247701 (7) [back to overview]Number of Participants With Chronic GvHD
NCT01247701 (7) [back to overview]Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
NCT01247701 (7) [back to overview]Number of Participants With Donor Engraftment After Transplant.
NCT01247701 (7) [back to overview]Number of Participants With Severe Acute GVHD Grade III-IV
NCT01338987 (4) [back to overview]Number of Adverse Events Related to Study Drug Experienced by Participants After Second Bone Marrow Transplant (BMT)
NCT01338987 (4) [back to overview]Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)
NCT01338987 (4) [back to overview]Percentage of B Cells at One Year Post-transplant in Participants Who Did/Did Not Receive Leuprolide Following Bone Marrow Transplant (BMT)
NCT01338987 (4) [back to overview]Time to Engraftment in First Transplant Recipients Only With Median Thoracic Spine Standardized Uptake Values (SUV) of 1.4 or Greater Than Those Patients With SUV's Less Than 1.4
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 Chronic GVHD
NCT01339910 (10) [back to overview]Percentage of Participants With Treatment-related Mortality
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 Relapse-Free Survival (RFS)
NCT01339910 (10) [back to overview]Percentage of Participants With Acute Graft Versus Host Disease (GVHD)
NCT01339910 (10) [back to overview]Percentage of Participants With Overall Survival (OS)
NCT01339910 (10) [back to overview]Percentage of Participants With Disease Relapse
NCT01366612 (1) [back to overview]To Compare the Relapse Rate at 1 Year of Patients With Myeloid Malignancies Receiving Each Treatment
NCT01380990 (14) [back to overview]Infection Rate
NCT01380990 (14) [back to overview]ADA Activity in Erythrocytes
NCT01380990 (14) [back to overview]VCN in Peripheral Blood Mononuclear Cells (PBMCs)
NCT01380990 (14) [back to overview]OS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
NCT01380990 (14) [back to overview]Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT01380990 (14) [back to overview]Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes
NCT01380990 (14) [back to overview]VCN in CD19+ B Cells
NCT01380990 (14) [back to overview]VCN in CD3+ T Cells
NCT01380990 (14) [back to overview]Frequency of Vector Integration Into Known Protooncogenes (3 Years)
NCT01380990 (14) [back to overview]Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT01380990 (14) [back to overview]Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)
NCT01380990 (14) [back to overview]Change From Baseline in CD3+ T Cell Counts (3 Years)
NCT01380990 (14) [back to overview]Change From Baseline in CD3+ T Cell Counts (1 Year)
NCT01380990 (14) [back to overview]EvFS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)
NCT01390402 (1) [back to overview]Number of Participants With Molecular Complete Remission at 3 Month Post Transplant
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]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 With Acute Graft-Versus-Host Disease (GVHD)
NCT01413178 (5) [back to overview]Number of Participants With Complete Response (CR)
NCT01413178 (5) [back to overview]Treatment-Related Mortality (TRM) Between 2 Arms.
NCT01413178 (5) [back to overview]Progression-Free Survival (PFS)
NCT01413178 (5) [back to overview]Overall Survival (OS)
NCT01413178 (5) [back to overview]Number of Participants That Had Grade 3-4 Toxicities.
NCT01427881 (9) [back to overview]Non-relapse Mortality
NCT01427881 (9) [back to overview]Donor Engraftment
NCT01427881 (9) [back to overview]Chronic GVHD Requiring Systemic Immunosuppressive Treatment
NCT01427881 (9) [back to overview]Hematologic Recovery
NCT01427881 (9) [back to overview]Graft Failure
NCT01427881 (9) [back to overview]Disease-free Survival
NCT01427881 (9) [back to overview]Persistent or Recurrent Malignancy After HCT
NCT01427881 (9) [back to overview]Grades II-IV and III-IV Acute GVHD
NCT01427881 (9) [back to overview]Overall Survival
NCT01457885 (3) [back to overview]Incidence of Relapse
NCT01457885 (3) [back to overview]Cumulative Incidence of Non Relapse Mortality (NRM)
NCT01457885 (3) [back to overview]The Percentage of Patients Alive at 1 Year
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Transplant-Related Mortality
NCT01464359 (9) [back to overview]Incidence of Graft Failure
NCT01464359 (9) [back to overview]Disease Free Survival
NCT01464359 (9) [back to overview]Clinical Disease Response
NCT01464359 (9) [back to overview]Duration of Survival
NCT01464359 (9) [back to overview]Clinical Disease Response
NCT01464359 (9) [back to overview]Incidence of Acute Graft-Versus-Host Disease
NCT01471444 (4) [back to overview]Overall Survival (OS) Post Transplant at 1, 3 and 5 Years
NCT01471444 (4) [back to overview]Progression-Free Survival (PFS)
NCT01471444 (4) [back to overview]Number of Participants With Non Relapse Mortality at 100 Day Post Transplant
NCT01471444 (4) [back to overview]Number of Participants in the Study Who Are With no Grade 3 or 4 Acute Graft-versus-host Disease at Any Time During the First 100 Days Post Transplant.
NCT01499147 (4) [back to overview]Time to ANC and Platelet Engraftment
NCT01499147 (4) [back to overview]Participants With 100 Day Transplant-related Mortality.
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.
NCT01565616 (8) [back to overview]Transplant Related Outcomes
NCT01565616 (8) [back to overview]Event -Free Survival Rate
NCT01565616 (8) [back to overview]PROMIS-57 Scores Health Related Quality of Life
NCT01565616 (8) [back to overview]Chronic Graft Versus Host Disease (GVHD)
NCT01565616 (8) [back to overview]Graft Failure
NCT01565616 (8) [back to overview]Overall Survival
NCT01565616 (8) [back to overview]Acute Graft Versus Host Disease (GVHD)
NCT01565616 (8) [back to overview]Time to Neutrophil and Platelet Engraftment
NCT01572662 (3) [back to overview]Overall Survival
NCT01572662 (3) [back to overview]Non-Relapse Mortality Rate (NRM)
NCT01572662 (3) [back to overview]Overall Survival
NCT01596699 (3) [back to overview]Mixed-donor Chimerism Rate of Patients With High-risk Myeloid Malignancies (Stratum B)
NCT01596699 (3) [back to overview]Number of Participants With Treatment-Related Adverse Events as a Measure of Safety and Tolerability
NCT01596699 (3) [back to overview]Engraftment Rate of Patients With Non-malignant Diseases (Stratum A)
NCT01605032 (5) [back to overview]Rate of Complete Response as Determined by the IMWG Criteria
NCT01605032 (5) [back to overview]Overall Response Rate
NCT01605032 (5) [back to overview]Progression-free Survival
NCT01605032 (5) [back to overview]Mortality
NCT01605032 (5) [back to overview]Overall Survival
NCT01621477 (7) [back to overview]Disease-Free Survival (DFS)
NCT01621477 (7) [back to overview]Event-Free Survival (EFS)
NCT01621477 (7) [back to overview]Incidence of Malignant Relapse
NCT01621477 (7) [back to overview]Number of Participants With Transplant Related Mortality (TRM)
NCT01621477 (7) [back to overview]One-year Survival (OS)
NCT01621477 (7) [back to overview]Incidence and Severity of Acute Graft Versus Host Disease (GVHD)
NCT01621477 (7) [back to overview]Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)
NCT01629511 (3) [back to overview]100 Day Treatment Related Mortality (TRM)
NCT01629511 (3) [back to overview]Time-to-engraftment
NCT01629511 (3) [back to overview]Overall Survival
NCT01643668 (9) [back to overview]Donor Stem Cell Engraftment: Platelet Count
NCT01643668 (9) [back to overview]Cumulative Incidence and Severity of Acute GVHD Within 100 Days Post Transplant
NCT01643668 (9) [back to overview]Cumulative Incidence of Non-relapse Mortality
NCT01643668 (9) [back to overview]Progression-Free and Overall Survival
NCT01643668 (9) [back to overview]Assessment of Donor Stem Cell Engraftment: ANC Count
NCT01643668 (9) [back to overview]Infection-related Complications
NCT01643668 (9) [back to overview]Cumulative Incidence of Chronic GVHD at One Year
NCT01643668 (9) [back to overview]Grade 3 or 4 Toxicities
NCT01643668 (9) [back to overview]Incidence of Hepatic Veno-occlusive Disease
NCT01685411 (15) [back to overview]Count of Participants With Disease Free Survival
NCT01685411 (15) [back to overview]Counts of Participants With Disease Free Survival
NCT01685411 (15) [back to overview]Number of Participant Who Were Alive at 2 Years Post Transplant
NCT01685411 (15) [back to overview]Number of Participant Who Were Alive at 5 Years Post Transplant
NCT01685411 (15) [back to overview]Percentage of Participants With Relapse
NCT01685411 (15) [back to overview]Percentage of Participants With Chronic Graft-Versus-Host Disease
NCT01685411 (15) [back to overview]Percentage of Participants With Treatment-Related Toxicity
NCT01685411 (15) [back to overview]Percentage of Participants With Engraftment Failure
NCT01685411 (15) [back to overview]Count of Participants Who Achieved Neutrophil Engraftment
NCT01685411 (15) [back to overview]Percentage of Participants With Chronic Graft-Versus-Host Disease
NCT01685411 (15) [back to overview]Percentage of Participants With Acute Graft-Versus-Host Disease by Grade
NCT01685411 (15) [back to overview]Percentage of Participants With Treatment-Related Toxicity
NCT01685411 (15) [back to overview]Percentage of Participants With Relapse
NCT01685411 (15) [back to overview]Number of Participant Who Were Alive at 7 Years Post Transplant
NCT01685411 (15) [back to overview]Count of Participants With Disease Free Survival
NCT01707004 (8) [back to overview]Time to Neutrophil Recovery
NCT01707004 (8) [back to overview]Number of Participants With Primary Graft Failure
NCT01707004 (8) [back to overview]Overall Survival (OS)
NCT01707004 (8) [back to overview]Percentage of Participants With Platelet Recovery by Day 30
NCT01707004 (8) [back to overview]Progression Free Survival
NCT01707004 (8) [back to overview]Cumulative Incidence of Chronic GVHD According to BMTCTN
NCT01707004 (8) [back to overview]Cumulative Incidence of Grade III-IV Acute GVHD
NCT01707004 (8) [back to overview]Number of Participants With Complete Remission After Transplantation
NCT01734850 (17) [back to overview]Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)
NCT01734850 (17) [back to overview]Number of Participants With HIV-1 Tropism Shift
NCT01734850 (17) [back to overview]Mean Cell Dose for CD4+ Cells (Ttn)
NCT01734850 (17) [back to overview]Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product
NCT01734850 (17) [back to overview]Percent Cal-1 Marking in Peripheral Blood
NCT01734850 (17) [back to overview]Total Area Under the Curve (AUC) for Busulfan
NCT01734850 (17) [back to overview]Number of Participants With Severe or Life-threatening AEs Related to CSL202
NCT01734850 (17) [back to overview]Number of Participants With Severe and Life-threatening Adverse Events (AEs)
NCT01734850 (17) [back to overview]Number of Participants With Predominant Integration Site Analysis
NCT01734850 (17) [back to overview]HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement
NCT01734850 (17) [back to overview]Number of Participants With the Presence of Replication-competent Retrovirus
NCT01734850 (17) [back to overview]Cal-1 C46 Expression in Peripheral Blood
NCT01734850 (17) [back to overview]Cal-1 Marking in Bone Marrow
NCT01734850 (17) [back to overview]Cal-1 Marking in GALT (25-35 cm)
NCT01734850 (17) [back to overview]Mean Cell Dose for CD34+ Cells (HSPCtn)
NCT01734850 (17) [back to overview]Cal-1 sh5 Expression in Peripheral Blood
NCT01734850 (17) [back to overview]CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement
NCT01746173 (2) [back to overview]Induction Response
NCT01746173 (2) [back to overview]24-month Progression-Free Survival Rate
NCT01749293 (3) [back to overview]Number of Participants That Engrafted and the Number of Participants That Had Full Donor Chimerism at Day 60
NCT01749293 (3) [back to overview]Number of Participants That Had an Event Free Survival Rate
NCT01749293 (3) [back to overview]Number of Participants That Had an Overall Survival Rate
NCT01773395 (5) [back to overview]Percentage of Participants Experiencing Grade 3 or Higher Non-Hematologic or Grade 4 or Higher Hematologic Adverse Events
NCT01773395 (5) [back to overview]18-Month Progression Free Survival
NCT01773395 (5) [back to overview]18-Month Overall Survival
NCT01773395 (5) [back to overview]Percentage of Participants Experiencing Acute and Chronic Graft-Versus-Host Disease
NCT01773395 (5) [back to overview]Percentage of Participants With Relapse and/or Non-Relapse Mortality
NCT01798004 (1) [back to overview]The Tolerability of BuMel Regimen
NCT01823198 (3) [back to overview]Number of Participants Who Experienced Dose-limiting Toxicities (DLT)
NCT01823198 (3) [back to overview]Overall Survival
NCT01823198 (3) [back to overview]Number of Participants With Grade 3 Toxicities
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]Percent Probability of Event-free Survival (EFS)
NCT01824693 (4) [back to overview]Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
NCT01852071 (11) [back to overview]OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
NCT01852071 (11) [back to overview]Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT01852071 (11) [back to overview]Reduction in Deoxyadenosine Nucleotide (dAXP) in Erythrocytes
NCT01852071 (11) [back to overview]Severe Infection Rate Excluding the First Three Months After Treatment
NCT01852071 (11) [back to overview]Vector Copy Number (VCN) in Peripheral Blood (PB) Granulocytes.
NCT01852071 (11) [back to overview]Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT01852071 (11) [back to overview]ADA Activity in Erythrocytes
NCT01852071 (11) [back to overview]Change From Baseline in CD3+ T Cell Counts (2 Years)
NCT01852071 (11) [back to overview]EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
NCT01852071 (11) [back to overview]Number of Single Integration Sites Representing >30% of the Total Integration Sites (2 Years)
NCT01852071 (11) [back to overview]VCN in Peripheral Blood Mononuclear Cells (PBMCs)
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)
NCT01857934 (6) [back to overview]Overall Response Rate [Complete Response + Very Good Partial Response + Partial Response (CR + VGPR + PR)]
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
NCT01894477 (2) [back to overview]Number of Participants With Acute GVHD, Graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0
NCT01894477 (2) [back to overview]Number of Participants That Did Not Progress Within 6 Months
NCT01983969 (2) [back to overview]Participants With Event-free Survival (EFS)
NCT01983969 (2) [back to overview]Frequency of DLT
NCT02007863 (1) [back to overview]Number of Successful Unrelated Cord Blood (UCB) Transplants
NCT02120157 (11) [back to overview]Cumulative Incidence of Chronic GVHD
NCT02120157 (11) [back to overview]Cumulative Incidence of Non-relapse Mortality
NCT02120157 (11) [back to overview]Incidence of Donor Cell Engraftment
NCT02120157 (11) [back to overview]Number of Participants With Donor Cell Engraftment
NCT02120157 (11) [back to overview]Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4
NCT02120157 (11) [back to overview]Primary and Secondary Graft Failure
NCT02120157 (11) [back to overview]Steroid and Non-steroid Immunosuppressants
NCT02120157 (11) [back to overview]Steroid and Non-steroid Immunosuppressants Use Duration
NCT02120157 (11) [back to overview]Survival
NCT02120157 (11) [back to overview]Survival
NCT02120157 (11) [back to overview]Time to Neutrophil and Platelet Recovery
NCT02248597 (5) [back to overview]12 Month Disease Free Survival Probability
NCT02248597 (5) [back to overview]Overall Survival
NCT02248597 (5) [back to overview]Progression Free Survival
NCT02248597 (5) [back to overview]Rate of Acute GvHD
NCT02248597 (5) [back to overview]Relapse-free Mortality
NCT02282904 (2) [back to overview]To Determine the Safety of This Allogeneic HSCT Approach in Patients With CGD Including Transplant Related Toxicity, the Incidence of Acute and Chronic Graft-versus-host Disease, Immune Reconstitution, Overalland Disease-free Survival.
NCT02282904 (2) [back to overview]To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD
NCT02999984 (10) [back to overview]Change From Baseline in Quality of Life Measures (2 Years)
NCT02999984 (10) [back to overview]Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT02999984 (10) [back to overview]EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
NCT02999984 (10) [back to overview]Change From Baseline in CD3+ T Cell Counts (2 Years)
NCT02999984 (10) [back to overview]Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)
NCT02999984 (10) [back to overview]Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)
NCT02999984 (10) [back to overview]OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)
NCT02999984 (10) [back to overview]Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)
NCT02999984 (10) [back to overview]Severe Infections Excluding First 3 Months After Treatment
NCT02999984 (10) [back to overview]Time to Cessation of IgRT for Those Who Stopped (2 Years)
NCT03018223 (4) [back to overview]Progression Free Survival (PFS)
NCT03018223 (4) [back to overview]Overall Survival (OS)
NCT03018223 (4) [back to overview]Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)
NCT03018223 (4) [back to overview]Incidence of Chronic GVHD
NCT03096782 (3) [back to overview]Time to Engraftment
NCT03096782 (3) [back to overview]Disease-free Survival
NCT03096782 (3) [back to overview]Overall Survival
NCT03128359 (3) [back to overview]Overall Survival (OS) at 1 Year
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
NCT03303950 (8) [back to overview]Non-relapse Mortality (NRM) at Day 100
NCT03303950 (8) [back to overview]Non-relapse Mortality (NRM) at Day 365
NCT03303950 (8) [back to overview]Number of Participants With Minimal Residual Disease (MRD) Response
NCT03303950 (8) [back to overview]Overall Survival at One Year
NCT03303950 (8) [back to overview]Disease Free Survival at One Year
NCT03303950 (8) [back to overview]Incidence of Acute Graft Versus Host Disease (GVHD)
NCT03303950 (8) [back to overview]Number of Participants With Different Clinical Responses
NCT03303950 (8) [back to overview]Incidence of Chronic GVHD
NCT04002115 (7) [back to overview]Neutrophil Engraftment
NCT04002115 (7) [back to overview]Severity of Chronic GVHD
NCT04002115 (7) [back to overview]Severity of Acute Graft-versus-host Disease (GVHD)
NCT04002115 (7) [back to overview]Rate of Chronic GVHD
NCT04002115 (7) [back to overview]Rate of Acute Graft-versus-host Disease (GVHD)
NCT04002115 (7) [back to overview]Non-relapse Related Mortality
NCT04002115 (7) [back to overview]Complete Remission (CR) Rate at Day 30 Post HSCT

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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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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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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Progression-free Survival

time to disease progression or death due to any cause (NCT00003270)
Timeframe: 1 year

Intervention% of participants (Number)
Arm 150

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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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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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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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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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Toxicity Associated With High-dose Busulfan and Etoposide Followed by Stem Cell Rescue

Toxicity is defined as any grade 3 or grade 4 toxicity per the Bearman toxicity grading criteria following Busulfan and Etoposide high-dose chemotherapy, stem cell transplant, and the inability to recover sufficiently by day 100 to start IL-2 therapy. (NCT00003875)
Timeframe: Day -7 of transplant to 100 days post transplant

InterventionParticipants (Count of Participants)
Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy)2

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Proportion of Patients Who Relapsed Associated With the Regimen

(NCT00003875)
Timeframe: From date of transplant to date of death from any cause, assessed up to 178 months

InterventionParticipants (Count of Participants)
Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy)16

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Overall Survival of Patients on Busulfan and Etoposide Followed by Stem Cell Rescue and Aldesleukin

Estimated by the method of Kaplan and Meier. (NCT00003875)
Timeframe: From date of transplant to date of death from any cause, assessed up to 178 months

InterventionParticipants (Count of Participants)
Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy)14

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Toxicity Associated With Aldesleukin Treatment After Stem Cell Rescue

Toxicity during IL-2 therapy of any of the following per NCI Common Toxicity version 3: grade 2, 3, 4, or 5 CNS (except grade 0-3 malaise, fatigue, anxiety and depression) toxicity; grade 3, 4, or 5 non-CNS or non-hematologic toxicity; any grade 4 or 5 hematologic toxicity. (NCT00003875)
Timeframe: IL-2 administration to one month after completion of IL-2 treatment

InterventionParticipants (Count of Participants)
Treatment (Chemo, Stem Cell Rescue, Interleukin Therapy)6

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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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Overall Survival (Induction Phase)

Overall survival is defined as the time from randomization in the induction phase to death. (NCT00049517)
Timeframe: Assessed during the first 4 months, then at least every three months for 2 years. then every six months until 5 years after study entry and every 12 months thereafter.

Interventionmonths (Median)
Standard Daunorubicin15.7
High-dose Daunorubicin23.7

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Disease-free Survival (Consolidation Phase)

Disease-free survival is defined from the time of the confirmation of a complete remission via biopsy to the relapse of the disease. (NCT00049517)
Timeframe: Assessed during the first 4 months, then at least every three months for 2 years. then every six months until five years after study entry, and every 12 months thereafter.

InterventionMonths (Median)
Autologous HCT15.0
Go/Autologous HCT13.6

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Overall Survival (Consolidation Phase)

Overall survival is defined as the time from randomization in the consolidation phase to death. (NCT00049517)
Timeframe: Assessed during the first 4 months, then at least every three months for 2 years. then every six months until five years after study entry, and every 12 months thereafter.

InterventionMonths (Median)
Autologous HCT35.5
Go/Autologous HCT27.9

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Rates of Durable Engraftment

Number of days that patients take to reach engraftment defined as time to hematologic engraftment will be defined as ANC >500/µl and platelets >20K/µl without transfusion support. (NCT00054327)
Timeframe: at day 42

Interventiondays (Mean)
Regimen A17.9
Regimen B-115.75
Regimen B-213
Regimen B-318.25
Regimen C13.9
Regimen D10.5

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Toxicity as Measured by CTC v2.0

Number of patients that experience grade 3 or above toxicity. See serious adverse event list for toxicities. (NCT00054327)
Timeframe: at 100 days post transplant

Interventionparticipants (Number)
Regimen A0
Regimen B-10
Regimen B-21
Regimen B-32
Regimen C2
Regimen D0

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Graft-versus-host Disease (GVHD)

Number of patients that develop acute graft-versus-host disease by grades 0-4. Grade O is no development of GVHD. Grade 1-4 is increase severity of skin, liver and gut involvement with 1 being least severe and 4 being most severe. (NCT00054327)
Timeframe: at 100 days post transplant

,,,,,
Interventionparticipants (Number)
Grade 0Grade 1Grade 2Grade 3Grade 4
Regimen A02620
Regimen B-111200
Regimen B-210101
Regimen B-310030
Regimen C11530
Regimen D00002

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Incidence of Recurrent Disease

Number of patients that have disease recurrence. (NCT00054327)
Timeframe: at day 100 post transplant

Interventionparticipants (Number)
Regimen A4
Regimen B-12
Regimen B-20
Regimen B-31
Regimen C2
Regimen D0

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Number of Patients With Overall Survival at 2 Years.

(NCT00054327)
Timeframe: at 2 years from transplant

Interventionparticipants (Number)
Regimen A5
Regimen B-12
Regimen B-22
Regimen B-31
Regimen C5
Regimen D1

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Non-relapse Mortality (NRM)

Percentage of patients who died due to causes other than relapse (NCT00070135)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)16

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2 Year Disease Free Survival In Unrelated Donor Recipient Group

"Percentage of participants who were alive and relapse free at 2 years for patients who were matched with an unrelated donor for transplant. The 2 year disease free survival, with 95% confidence interval, was estimated using the Kaplan Meier method.~A relapse is defined as any of the following:~Reappearance of leukemia blasts cells in peripheral blood~>5% blasts in the marrow, not attributable to another cause (e.g., bone marrow regeneration)~If there are no circulating blasts, but the marrow contains 5-20% blasts, a repeat bone marrow ≥ 1 week later with >5% blasts is necessary to meet the criteria for relapse~The development of extramedullary leukemia or leukemic cells in the cerebral spinal fluid" (NCT00070135)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)40

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2 Year DFS for All Patients

Percentage of participants who were alive and relapse free at 2 years for all patients. The 2 year disease free survival, with 95% confidence interval, was estimated using the Kaplan Meier method. (NCT00070135)
Timeframe: Up to 2 years

Interventionpercentage of participants (Number)
Treatment (Fludarabine, Busulfan, Allogeneic PBSC)42

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Relapse

Percentage of participants who developed relapse or progressive disease. (NCT00134017)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Bone Marrow Transplant44

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Chimerism

Number of patients who achieved 100% donor chimerism. (NCT00134017)
Timeframe: Day 30, Day 60

InterventionParticipants (Count of Participants)
Day 30Day 60
Bone Marrow Transplant99101

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Days to Engraftment

Median number of days to neutrophil and platelet engraftment. (NCT00134017)
Timeframe: Up to one year

Interventiondays (Median)
Neutrophil engraftment, related donorsNeutrophil engraftment, unrelated donorsPlatelet engraftment, related donorsPlatelet engraftment, unrelated donors
Bone Marrow Transplant23253135

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Percentage of Participants Who Develop Acute Graft-versus-host Disease (GVHD)

Percentage of participants who developed grades II-IV and grades III-IV acute 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 I (skin involvement stages 1+ to 2+, with no liver or GI involvement), 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+). (NCT00134017)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Grades II-IV acute GVHDGrades III-IV acute GVHD
Bone Marrow Transplant4910

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Non-relapse Mortality

Percentage of participants who died for BMT-related reasons. (NCT00134017)
Timeframe: Day 100, 2 years

Interventionpercentage of participants (Number)
Day 1002 years
Bone Marrow Transplant615

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Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy.

Outcome was measured by ANC >500 for three consecutive days prior to day 30 after PBSC infusion, >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods prior to day 45 after PBSC infusion and >25% of hematopoietic cells are donor derived as determined by molecular chimerism assays or cytogenetic methods after day 180 after PBSC infusion. (NCT00153985)
Timeframe: 3 years

Interventionparticipants (Number)
Transplant for Severe Hemoglobinopathies2

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The Incidence of Grade II-IV Acute Graft vs. Host Disease.

Outcome was measured by incidence and severity of acute and chronic GVHD following donor stem cell infusion. (NCT00153985)
Timeframe: 3 years

Interventionparticipants (Number)
Transplant for Severe Hemoglobinopathies2

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Number of Patients Surviving (Disease-free)

(NCT00176826)
Timeframe: 1 year

Interventionparticipants (Number)
Intent-To-Treat14

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Number of Patients With III-IV Graft-Versus-Host Disease (GVHD)

(NCT00176826)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Intent-To-Treat1

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Number of Patients With Graft Failure

(NCT00176826)
Timeframe: Day 100 Post transplant

Interventionparticipants (Number)
Intent-To-Treat2

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Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD)

(NCT00176826)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Intent-To-Treat4

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Time to Transplant Engraftment

(NCT00176826)
Timeframe: Day 100 Post Transplant

Interventiondays (Mean)
Intent-To-Treat19.8

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Number of Patients Surviving (Disease-free)

(NCT00176826)
Timeframe: 3 years

Interventionparticipants (Number)
Intent-To-Treat10

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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 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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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 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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The Incidence of Chimerism at 6 Months

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)8

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: 1 year

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)97
MA Bu/Cy (B)100
RIC Cy/Flu/TBI (A2)100

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The Incidence of Chimerism at 100 Days

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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The Incidence of Chimerism at 1 Year

The number of patients whose blood and/or bone marrow contains > 10% donor cells. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)1
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)8

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Overall Survival

Number of patients alive 100 days after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)12

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

The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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

The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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Overall Survival

Number of patients alive 1 year after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)12

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The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)

The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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Disease Free Survival

Number of patients alive without disease 100 days after transplant. (NCT00176852)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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Disease Free Survival

Number of patients alive without disease 1 year after transplant. (NCT00176852)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)3
MA Bu/Cy (B)5
RIC Cy/Flu/TBI (A2)11

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: pre-transplant

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)100
MA Bu/Cy (B)98
RIC Cy/Flu/TBI (A2)99

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Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment

"The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is perfect health and 0 is death." (NCT00176852)
Timeframe: 2 years

Interventionunits on a scale (Median)
RIC Bu/Flu (A) (Discontinued)100
MA Bu/Cy (B)100
RIC Cy/Flu/TBI (A2)100

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The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)

The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant. (NCT00176852)
Timeframe: 6 months

InterventionParticipants (Count of Participants)
RIC Bu/Flu (A) (Discontinued)0
MA Bu/Cy (B)0
RIC Cy/Flu/TBI (A2)0

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Number of Patients With Succcessful Engraftment After Transplantation

"Number of patients who received non-genotypic identical marrow or cord blood cells using a non-myeloablative preparative regimen and exhibited engraftment at Day 42." (NCT00176878)
Timeframe: 42 Days

InterventionParticipants (Number)
Bone Marrow Failure Patients10

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Number of Patients With Disease Recurrence

Number of patients who exhibited disease recurrence at 2 years. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients0

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Number of Patients With Chronic Graft Versus Host Disease

Number of patients who exhibited chronic (normally occurs after 100 days) Graft Versus Host Disease at 2 years post transplant. Chronic graft-versus-host-disease, over its long-term course, can also cause damage to the connective tissue and exocrine glands. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients3

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Number of Patients Alive (Survival) at 2 Years

Calculated from day 1 of transplant to last contact. (NCT00176878)
Timeframe: 2 years

InterventionParticipants (Number)
Bone Marrow Failure Patients6

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Number of Patients With Grade 2-4 Acute Graft Versus Host Disease

Number of patients with Grade 2, 3 and 4 Acute (normally observed within the first 100 days) Graft Versus Host Disease. Acute GVHD is staged as follows: overall grade (skin-liver-gut) with each organ staged individually from a low of 1 to a high of 4. Patients with grade IV GVHD usually have a poor prognosis. Grade 2 = moderate, Grade 3 = severe, Grade 4 = life threatening. (NCT00176878)
Timeframe: 100 Days

InterventionParticipants (Number)
Bone Marrow Failure Patients5

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Number of Patients Alive at Three Years (Survival)

Number of subjects who survived 3 years post-transplant. (NCT00176878)
Timeframe: 3 years

InterventionParticipants (Number)
Bone Marrow Failure Patients6

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Number of Patients With Chronic Graft-Versus-Host Disease

Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome. (NCT00176904)
Timeframe: 1 Year Post Transplant

InterventionParticipants (Number)
Patients Treated With Stem Cell Transplant13

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Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease

Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening. (NCT00176904)
Timeframe: Day 100

InterventionParticipants (Number)
Patients Treated With Stem Cell Transplant34

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Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease

Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening. (NCT00176904)
Timeframe: Day 100

InterventionParticipants (Number)
Patients Treated With Stem Cell Transplant13

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Overall Donor Engraftment

Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%. (NCT00176904)
Timeframe: Day 100

InterventionParticipants (Number)
Patients Treated With Stem Cell Transplant123

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Overall Survival

Number of patients alive at designated timepoints after transplant. (NCT00176904)
Timeframe: 100 Days, 1 Year and 3 Years

InterventionParticipants (Number)
Day 1001 Year3 Years
Patients Treated With Stem Cell Transplant1209281

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Number of Patients Who Failed Engraftment.

Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42. (NCT00176917)
Timeframe: Day 42 Post Transplant

InterventionParticipants (Number)
Transplant Patients1

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Number of Patients Surviving on Study

Number of patients surviving (alive) at specified timepoints. (NCT00176917)
Timeframe: at 100 days, 1 year, and 3 years post transplant

InterventionParticipants (Number)
Day 100 Post Transplant1 Year Post Transplant3 Years Post Transplant
Transplant Patients372827

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Mean Percentage of Donor Cells in Study Population (Chimerism).

Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP). (NCT00176917)
Timeframe: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year

InterventionPercentage (Mean)
21 Days Post Transplant42 Days Post Transplant60 Days Post Transplant100 Days Post Transplant6 Months Post Transplant1 Year Post Transplant
Transplant Patients85.873.284.681.181.691.5

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Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD).

Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease. (NCT00176917)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Number)
Transplant Patients2

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Number of Participants With Persistence or Relapse of Malignancy at 5 Years Post Transplant

Defined as the return of disease after its apparent recovery/cessation. Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT00176930)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
PBSC: No TBI4
Marrow : No TBI4
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC69
Marrow20
Umbilical Cord Blood0
Co-Enroll From MT04030

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Number of Participants With Persistence or Relapse of Malignancy at 2 Years Post Transplant

Defined as the return of disease after its apparent recovery/cessation. Patients with leukemia and lymphoma involving the BM and multiple myeloma will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients with lymphoma and myeloma will have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT00176930)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
PBSC: No TBI3
Marrow : No TBI4
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC63
Marrow17
Umbilical Cord Blood0
Co-Enroll From MT04030

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Number of Participants With Neutrophil Engraftment

Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater. (NCT00176930)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
PBSC: No TBI11
Marrow : No TBI13
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG1
PBSC206
Marrow83
Umbilical Cord Blood2
Co-Enroll From MT04032

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Number of Participants With Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Determine the incidence of chronic GVHD 1 year post transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. (NCT00176930)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
PBSC: No TBI4
Marrow : No TBI2
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC87
Marrow14
Umbilical Cord Blood0
Co-Enroll From MT04031

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

"Acute Graft-Versus-Host Disease (aGVHD) is a severe short-term complication created by infusion of donor cells into a foreign host. Determine the incidence of grade II-IV acute graft-versus-host disease (GVHD) at day 100 post transplant. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging.~Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level." (NCT00176930)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
PBSC: No TBI6
Marrow : No TBI2
UCB : No TBI1
UCB : No TBI/Bu/Cy/ATG0
PBSC89
Marrow23
Umbilical Cord Blood0
Co-Enroll From MT04030

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Number of Participants Who Were Alive at 5 Year Post Transplant

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. (NCT00176930)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
PBSC: No TBI3
Marrow : No TBI9
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC100
Marrow54
Umbilical Cord Blood2
Co-Enroll From MT04032

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Number of Participants Who Were Alive at 2 Year Post Transplant

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. (NCT00176930)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
PBSC: No TBI5
Marrow : No TBI11
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC122
Marrow59
Umbilical Cord Blood2
Co-Enroll From MT04032

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Number of Participants Experiencing Engraftment Failure

Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets. (NCT00176930)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
PBSC: No TBI0
Marrow : No TBI0
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC1
Marrow0
Umbilical Cord Blood0
Co-Enroll From MT04030

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Number of Participants Experiencing Disease-Free Survival at 5 Years Post Transplant

Disease-Free Survival is the length of time during and after medication or treatment during which the disease being treated (usually cancer) does not get worse. It is sometimes used as a metric to study the health of a person with a disease to try to determine how well a new treatment is working. (NCT00176930)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
PBSC: No TBI3
Marrow : No TBI9
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC85
Marrow48
Umbilical Cord Blood2
Co-Enroll From MT04032

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Number of Participants Experiencing Disease-Free Survival at 2 Years Post Transplant

Disease-Free Survival is the length of time during and after medication or treatment during which the disease being treated (usually cancer) does not get worse. It is sometimes used as a metric to study the health of a person with a disease to try to determine how well a new treatment is working. (NCT00176930)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
PBSC: No TBI4
Marrow : No TBI9
UCB : No TBI0
UCB : No TBI/Bu/Cy/ATG0
PBSC100
Marrow52
Umbilical Cord Blood2
Co-Enroll From MT04032

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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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Acute Graft-Versus-Host Disease (aGVHD) Outcome

"Grading of Acute GVHD:~Severity of Individual Organ Involvement:~Skin~1 a maculopapular eruption involving less than 25% of the body surface~2 a maculopapular eruption involving 25-50% of the body surface~3 generalized erythroderma~4 generalized erythroderma with bullous formation and/or with desquamation Liver~1 bilirubin 2.0-3.0mg/100mL~2 bilirubin 3-5.9mg/100mL~3 bilirubin 6-14.9mg/100mL~4 bilirubin >15mg/100mL Gut Diarrhea is graded +1 to +4 in severity. Nausea/vomiting and/or anorexia caused by GVHD is assigned as +1 in severity Diarrhea~1 <1000mL of liquid stool/day~2 >1,000mL of stool/day~3 >1,500mL of stool/day~4 2,000mL of stool/day, severe abdominal pain, with or without ileus~Severity of GVHD:~Grade 1 +1 to +2 skin rash; No gut or liver involvement Grade 2 +1 to +3 skin rash;+1 GI involvement and/or +1 liver" (NCT00245037)
Timeframe: Day 100, Month 6

Interventionpercentage of analyzed participants (Number)
Day 100Month 6
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)5560

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Chronic Graft-Versus-Host Disease (cGVHD) Outcome

"Grading of Chronic GVHD:~Limited: Localized skin involvement and/or hepatic dysfunction due to chronic GVHD~Extensive:~One or more of the following:~Generalized skin involvement Liver histology showing chronic aggressive hepatitis, bridging necrosis or cirrhosis Involvement of the eye: Schirmer's test with <5 mm wetting Involvement of minor salivary glands or oral mucosa demonstrated on labial biopsy Involvement of any other target organ~Chronic GVHD Severity:~Mild: Signs and symptoms of cGVHD do not interfere substantially with function and do not progress once appropriately treated with local therapy or standard systemic therapy.~Moderate: Signs and symptoms of cGVHD interfere somewhat with function despite appropriate therapy or are progressive through first line systemic therapy.~Severe: Signs and symptoms of cGVHD limit function substantially despite appropriate therapy or are progressive through second line systemic therapy" (NCT00245037)
Timeframe: Years 1, 2 and 3

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)64.66667.3

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Non-relapse Mortality

Percent of subjects with non-relapse mortality two years after conditioning with busulfan with fludarabine/200 cGy TBI in patients with hematologic malignancies at moderate to high risk for graft rejection and/or relapse of underlying disease. (NCT00245037)
Timeframe: Two years post-transplant

InterventionParticipants (Count of Participants)
Year 1Year 2
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)2733

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Overall Survival

The percentage of overall patient survival (out of 147 participants) for Years 1, 2, 3 and 5. (NCT00245037)
Timeframe: Years 1, 2, 3 and 5

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3Year 5
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)60484229

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Progression-Free Survival

"The percentage of progression-free patients (out of 147 participants) at Years 1, 2, 3, and 5.~Definition of Disease Progression:~MM/Plasma Cell: Increasing bone pain or increase in serum/urine monoclonal protein by 25%.~CLL/NHL/HD: New sites of lymphadenopathy; ≥ 25% increase in lymph node size; Blood or bone marrow involvement with clonal B-cells; Increase of ≥ 25% bone marrow involvement; ≥ 25% increase in blood involvement with clonal B-cells.~AML/ALL: Any incidence of relapse (>5% blasts) by evaluation of the bone marrow aspirate.~CML: Inability to control platelet or granulocyte counts; Increase in baseline number of metaphases demonstrating the Ph+ chromosome by >25%; Any other new cytogenetic abnormality; Transformation to accelerated phase or blast crisis.~MDS/MPD: Any evidence by morphologic or flow cytometric evaluation of the bone marrow aspirate of new blasts (>5%) or worsening cytopenia or cytogenetic evidence of recurrence." (NCT00245037)
Timeframe: Years 1, 2, 3, and 5

Interventionpercentage of analyzed participants (Number)
Year 1Year 2Year 3Year 5
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)48393529

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Relapse Mortality

The percentage of patients (out of 147 participants) who relapsed at Years 1 and 2. Relapse is defined as the presence of >5% blasts by morphology on a post-transplant bone marrow aspirate. (NCT00245037)
Timeframe: Years 1 and 2

Interventionpercentage of analyzed participants (Number)
Year 1Year 2
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI)1320

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Number of Participants Experiencing Acute Graft-Versus-Host Disease

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00258427)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host. (NCT00258427)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cellsinto a foreign host. (NCT00258427)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Major Infections

Number of participants experiencing Major Infections by the end of treatment (NCT00258427)
Timeframe: Day 1 through 1 year post-transplant

InterventionParticipants (Count of Participants)
Marrow Isolex3
USB Arm8
Marrow Clinimacs2
Sibling withoutCliniMACS1

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Number of Participants Experiencing Overall Survival

Overall Survival - Number of patients alive at 1 year post transplant (NCT00258427)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex1
USB Arm4
Marrow Clinimacs1
Sibling withoutCliniMACS1

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Number of Participants Experiencing Relapse

Patients with leukemia will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. (NCT00258427)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm2
Marrow Clinimacs1
Sibling withoutCliniMACS0

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Number of Participants Experiencing Graft Failure

Graft failure is defined as absolute neutrophil count( ANC ) <5 x 10^8/L by day 30. (NCT00258427)
Timeframe: Day 30

InterventionParticipants (Count of Participants)
Marrow Isolex1
USB Arm0
Marrow Clinimacs0
Sibling withoutCliniMACS0

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Number of Participants Experiencing Acute Graft-Versus-Host Disease

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00258427)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Marrow Isolex0
USB Arm1
Marrow Clinimacs0
Sibling withoutCliniMACS0

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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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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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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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Percentage of Participants With ≥90 Percent Donor-derived Hematopoeisis Around 100 Days Post Transplantation

The percentage of participants with ≥90 percent donor-derived hematopoeisis was assessed around day +100 using peripheral blood chimerism. (NCT00282282)
Timeframe: 100 days

Interventionpercentage of participants (Number)
Tacrolimus and Sirolimus78

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Disease Response.

Disease response was assessed as 2 year progression-free survival. The median follow-up time was 1.84 years. The percentage of participants with who reached this timepoint with no disease progression are reported. (NCT00282282)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Tacrolimus and Sirolimus GVHD Prophylaxis48

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Incidence of Grade II-IV Acute GVHD (aGVHD) Developing by Day 100 Following Non-myeloablative PBSC Transplantation Using Tacrolimus and Sirolimus.

All participants received tacrolimus and sirolimus in this one arm study. There were no participants considered unevaluable for this measure (deceased prior to day 100). The total number of people who developed grade II-IV aGVHD before day 100 are reported here. (NCT00282282)
Timeframe: 100 days

Interventionparticipants (Number)
Tacrolimus and Sirolimus5

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Disease-free Survival With Correction of Disease at One Year Post Transplantation

"Patients deemed alive and well at follow-up timepoint later than 1-year post-transplantation" (NCT00301834)
Timeframe: 1 year post-transplantation

Interventionparticipants (Number)
Single Arm - Transplant Pre-conditioning Per Study Protocol22

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Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation

Peripheral blood chimerism studies were performed by quantitative real time polymerase chain reaction (qPCR) evaluation of differential short tandem repeat DNA sequences (NCT00301834)
Timeframe: 6 weeks post-transplant

Interventionparticipants (Number)
Single Arm - Transplant Pre-conditioning Per Study Protocol31

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Cytomegalovirus (CMV) Viral Infection and Disease Symptoms

polymerase chain reaction testing for presence of CMV weekly until at least day +100 then every 2 weeks until T-cell reconstitution as defined by cluster of differentiation 4 (CD4) > 200 cells/mm3. Median time to T-cell reconstitution was 6 months. (NCT00301834)
Timeframe: Up to one year post-transplant

,
Interventionparticipants (Number)
Positive CMV Viral Load AssaySymptomatic CMV disease
CMV Seronegative Participants10
CMV Seropositive Participants120

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Toxicity Grade ≥ 3 From Start of Conditioning Through the First Year Post Transplantation

(NCT00301834)
Timeframe: 1 year post-transplantation

Interventionparticipants (Number)
Grade 3-4 acute Graft-Versus-Host DiseaseGrade 3-4 mucositis
Single Arm216

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Treatment-related Mortality at 100 Days and 1 Year Post Transplantation

(NCT00301834)
Timeframe: 100 days and 1 year

Interventionparticipants (Number)
Transplantation-related mortality 0-100 daysTransplantation-related mortality 100-365 days
Single Arm - Transplant Pre-conditioning Per Study Protocol21

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Donor Cell Engraftment

Chimerism analysis was performed in patients who recieved nonmyeloablative tranplsnat. In this group the definition of engraftment was a CD3 count greater than 50%. In the myeloablative group, engraftment was defined as an absolute neutrophil count greater than 50%. (NCT00322101)
Timeframe: After stem cell infusion to day 28

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)11
Arm II (Myeloablative Regimen)11

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Incidence and Severity of Acute and Chronic Graft-vs-host Disease

(NCT00322101)
Timeframe: After transplantation

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)1
Arm II (Myeloablative Regimen)4

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Incidence of Disease Progression/Relapse

Disease progression/relapse was defined by IWG criteria (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)7
Arm II (Myeloablative Regimen)2

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Overall Survival

(NCT00322101)
Timeframe: At 2 years

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)6
Arm II (Myeloablative Regimen)6

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Progression-free Survival

IWG criteria was used to determine disease progression (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)7
Arm II (Myeloablative Regimen)5

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Non-relapse Mortality

(NCT00322101)
Timeframe: At 100 days

Interventionparticipants (Number)
Arm I (Nonmyeloablative Regimen)0
Arm II (Myeloablative Regimen)0

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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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Non-relapse Mortality

The number of participants dead due to causes unrelated to relapse within the first 100 days post transplant. (NCT00361140)
Timeframe: 100 days

Interventionparticipants (Number)
AUC 60003
AUC 75004
AUC 90002

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Severe Venous Occlusive Disease (VOD)/ Sinusoidal Obstructive Syndrome (SOS)

The number of subjects with severe VOD / SOS; severity staged according to criteria set forth by McDonald G.B., Hinds M.S., Fisher L.D., et al. Veno-occlusive disease of the liver and multiorgan failure after bone marrow transplantation: a cohort study of 355 patients. Ann Intern Med. 1993;118:255-267. Assessed within the first 100 days post transplant. (NCT00361140)
Timeframe: 100 days

Interventionparticipants (Number)
AUC 60000
AUC 75001
AUC 90002

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1 Year Event-free Survival

Number of participants alive and without disease relapse at 1 year posttransplant (NCT00363467)
Timeframe: 1 year post transplant

Interventionparticipants (Number)
Autologous Hematopoietic Progenitor Cell Transplantation2

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1 Year Overall Survival

Number of participants alive at 1 year posttransplant (NCT00363467)
Timeframe: 1 year post transplant

Interventionparticipants (Number)
Autologous Hematopoietic Progenitor Cell Transplantation2

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100-day Non-relapse Mortality

100-day non-relapse mortality is the number of participants who died before day 100 posttransplant from causes other than relapsed disease (NCT00363467)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
Autologous Hematopoietic Progenitor Cell Transplantation0

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Successful Autologous Stem Cell Collection

Number of subjects who were able to collect at least 2 million CD34+ cells/kg (NCT00363467)
Timeframe: At time of stem cell collection

Interventionparticipants (Number)
Autologous Hematopoietic Progenitor Cell Transplantation2

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Number of Participants Who Completed Maintenance Decitabine.

To determine feasibility of decitabine maintenance, this outcome measures the number of participants who completed all 8 planned cycles of decitabine maintenance as per protocol. (NCT00416598)
Timeframe: Up to 5 years

Interventionparticipants (Number)
Decatibine Maintenance62

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Disease-free Survival (DFS) Rate at 1 Year

"For participants who achieved a complete remission (CR), this is the percentage of participants who were alive and relapse free at 1 year. The 1 year rate, with 95% confidence interval, was estimated using the Kaplan-Meier method~A CR is defined as those with > 20% cellularity of bone marrow biopsy, no presence of extramedullary leukemia for AML, <5 % myeloblast cells for bone marrow with peripheral blood and normal complete blood count (absolute neutrophils > 1000 mL and platelets >= 100,000 mL)." (NCT00416598)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Decatibine Maintenance80

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Days to Absolute Neutrophil Recovery (ANC)

Recovery is defined as an absolute neutrophil count (ANC) of ≥ 0.5 x 109 /L (500/mm3 ) for three consecutive laboratory values obtained on different days. Date of ANC recovery is the date of the first of three consecutive laboratory values where the ANC is ≥ 0.5 x 109 /L. (NCT00426517)
Timeframe: 1 year

InterventionDays (Mean)
Matched Related Donor Stem Cell Transplant19.42
Matched Unrelated Donor Stem Cell Transplant77.44
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)25.5
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Participants With Established Stable Mixed Chimerism

Number of participants with myeloid chimerism of greater than 10% of donor cells at 1 year post transplant (NCT00426517)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Matched Related Donor Stem Cell Transplant7
Matched Unrelated Donor Stem Cell Transplant30
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)3
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Stem Cell Transplant Engraftment

Engraftment of allogeneic or matched unrelated (including cord blood) hematopoietic progenitor cells using moderate-dose busulfan and Campath-1H with or without whole body irradiation so as to attain phenotypic correction of congenital immunodeficiencies. (NCT00426517)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Matched Related Donor Stem Cell Transplant7
Matched Unrelated Donor Stem Cell Transplant31
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)3
Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood0

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Engraftment Without Development of GVHD

Participants who achieved engraftment without development of graft versus host disease (GVHD). (NCT00426517)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Matched Related Donor Stem Cell Transplant6
Matched Unrelated Donor Stem Cell Transplant30
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)3
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Incidence of Cytomegalovirus (CMV) Disease

Number of events of Cytomegalovirus disease based on clinical sequelae that requires treatment (not reactivation) (NCT00426517)
Timeframe: 1 year

InterventionNumber of events (Mean)
Matched Related Donor Stem Cell Transplant0
Matched Unrelated Donor Stem Cell Transplant0
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)0
Matched Unrelated Donor Transplant (MUD-CGD) Cord Blood0

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Number of RBC Transfusions Per Subject

Average number of red blood cell (RBC) transfusion per subject (NCT00426517)
Timeframe: 1 year

Interventiontransfusions per person (Mean)
Matched Related Donor Stem Cell Transplant1.71
Matched Unrelated Donor Stem Cell Transplant7.08
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)4
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Days to Platelet Recovery

Platelet recovery is defined as platelet value ≥ 20 × 109/L for three consecutive days and no platelet transfusions administered for previous seven consecutive days. The date of platelet recovery is the date of the first of three consecutive laboratory values ≥ 20 × 109/L. (NCT00426517)
Timeframe: 1 year

InterventionDays (Mean)
Matched Related Donor Stem Cell Transplant31
Matched Unrelated Donor Stem Cell Transplant31.9
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)32.6
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Days to CD3 Count Greater Than 100 u/L

Rapidity of immune reconstitution based on number of days to CD3 count greater than 100 u/L. (NCT00426517)
Timeframe: 1 year

InterventionDays (Mean)
Matched Related Donor Stem Cell Transplant355
Matched Unrelated Donor Stem Cell Transplant284
Matched Unrelated Donor Stem Cell Transplant (MUD-non CGD)16
Matched Unrelated Donor Transplant (MUD-CGD) Cord BloodNA

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Development of GVHD Within 1 Year of BMT

GVHD is assessed by physical exam, bloodwork and biopsy. (NCT00427661)
Timeframe: 1 year

Interventionparticipants (Number)
AHSC in Severe SCD2

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Engraftment at 1 Year Post BMT.

Measurement of total PBMC chimerism (NCT00427661)
Timeframe: 1 year

Interventionparticipants (Number)
AHSC in Severe SCD6

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Incidence of Grade 2-4 Acute GVHD.

(NCT00427661)
Timeframe: 100 days

Interventionparticipants with grade 2-4 AGVHD (Number)
Experimental1

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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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Non-relapse Mortality (NRM) (Patients With AML/MDS)

Cumulative incidence rate with death as a competing risk, assessed at day 100. (NCT00445744)
Timeframe: Up to day 200

Interventionpercent (Number)
Treatment (Cyclophosphamide, Busulfan, Transplant)17

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5-year Disease-free Survival

The length of time post-transplant that the patient survives without any signs or symptoms of that cancer. (NCT00448201)
Timeframe: Year 5

Interventionpercentage of participants (Number)
All Trial Participants31

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Complete or Mixed Donor Chimerism at 30, 60, and 90 Days Post-transplant

"Complete chimerism is defined as 100% donor cells detected, suggesting complete hematopoietic replacement. Mixed donor chimerism means host cells are detected in particular cells like lymphocytes. Five to 90% donor cells set the criteria for mixed chimerism (MC).~Chimerism was not tabulated on day 30." (NCT00448201)
Timeframe: Days 30, 60, and 90

,
Interventionpercentage of patients (Number)
Complete DonorMixed Donor
Day 608218
Day 908713

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Graft-vs-host Disease at 6 Months Post-transplant

"Graft-vs-host disease (GVHD) can be mild, moderate or severe depending on the differences in tissue type between patient and donor. Its symptoms can include:~Rashes, which include burning and redness, that erupt on the palms or soles and may spread to the trunk and eventually to the entire body~Blistering, causing the exposed skin surface to flake off in severe cases~Nausea, vomiting, abdominal cramps, diarrhea and loss of appetite, which can indicate that the gastrointestinal (digestive) tract is affected~Jaundice, or a yellowing of the skin, which can indicate liver damage~Excessive dryness of the mouth and throat, leading to ulcers~Dryness of the lungs, vagina and other surfaces~Acute GVHD - Can occur soon after the transplanted cells begin to appear in the recipient. Acute GVHD ranges from mild, moderate or severe, and can be life-threatening if its effects are not controlled.~Extensive chronic GVHD - Usually occurs at about three months post-transplant." (NCT00448201)
Timeframe: 6 Months

Interventionpercentage of participants (Number)
Acute GVHDExtensive Chronic GVHD
All Trial Participants1330

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Incidence of Graft vs Host Disease in Patients Between One Month and Two Years Post Transplant

"GVHD can be mild, moderate or severe depending on the differences in tissue type between patient and donor. GVHD can be acute or chronic. Its symptoms can include:~Rashes, which include burning and redness, that erupt on the palms or soles and may spread to the trunk and eventually to the entire body~Blistering, causing the exposed skin surface to flake off in severe cases~Nausea, vomiting, abdominal cramps, diarrhea and loss of appetite, which can indicate that the gastrointestinal (digestive) tract is affected~Jaundice, or a yellowing of the skin, which can indicate liver damage~Excessive dryness of the mouth and throat, leading to ulcers~Dryness of the lungs, vagina and other surfaces" (NCT00448357)
Timeframe: 100 days post transplant

,,
InterventionParticipants (Count of Participants)
Acute GVHD grade >=IIAcute GVHD grades III and IVChronic GVHD; intermediate/severe
High Busulfan AUC Tertile1132
Intermediate Busulfan AUC Tertile1046
Low Busulfan AUC Tertile724

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Number of Participants With Dose Limiting Toxicities (DLTs)

Dose limiting toxicity will be defined as any irreversible grade 3 or any grade 4 non-hematologic toxicity that is related to busulfan infusion and not graft vs host disease or late infection after recovery from the initial period of myelosuppression. The maximum tolerated dose (MTD) is defined as the dose with probability of dose limiting toxicity (DLT) of 0.25. The dose of continuous infusion IV busulfan based on blood levels derived from a test dose in conjunction with fludarabine and alemtuzumab plus tacrolimus for GVHD prophylaxis. (NCT00448357)
Timeframe: first 6 weeks or 42 days following stem cell infusion

InterventionDLTs (Number)
Dose Level 11
Dose Level 21
Dose Level 31
Dose Level 42
Dose Level 52

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Overall Survival

Percentage of participants alive at 3 years post transplant (NCT00448357)
Timeframe: Three years post-transplant

Interventionpercentage of participants (Number)
Low Busulfan AUC Tertile (5078)28
Intermediate Busulfan AUC Tertile (6372)39
High Busulfan AUC Tertile (7605)55

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Capacity of Test Dosing of Busulfan That Would Result in the Desired Area Under the Curve Concentration Exposure of Patients Receiving a Full-dose Busulfan Regimen

Test doses of busulfan were administered and plasma levels were measured to determine a targeted AUC dosing estimate. The capacity is reported as the precision with which these test dose goals predicted the actual 90-hour mean AUC levels.Dose targeting precision was estimated by root mean squared error. (NCT00448357)
Timeframe: Day -15 to Day -11

Interventionpercentage of error (Number)
Dose Level 111.7
Dose Level 24.9
Dose Level 310.2
Dose Level 411.1
Dose Level 515.9

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Incidence of DNA Chimerism in Patients Between One Month Post Transplant

Deoxyribonucleic acid (DNA) chimerism is a measure identifying the genetic profiles of the transplant recipient and of the donor and then evaluating the extent of mixture in the recipient's blood, bone marrow, or other tissue. (NCT00448357)
Timeframe: 30 days post transplant

InterventionParticipants (Count of Participants)
Whole blood chimerism-AnyWhole blood chimerism->=95% donorT Cell chimerism-AnyT Cell chimerism>=95% donor
Experimental: GVHD Prophylaxis49474630

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Three-year Relapse-free Survival (RFS) Rate at the Maximum Tolerated Dose Identified During Phase I of the Trial (Target AUC 6912)

Relapse is defined as new or increased sites of disease or positive one marrow after a complete response (CR). The RFS was calculated as the percentage of patients who were alive and without relapse at 3 years (NCT00448357)
Timeframe: Three years post-transplant

Interventionpercentage of participants (Number)
Low Busulfan AUC Tertile22
Intermediate Busulfan AUC Tertile39
High Busulfan AUC Tertile43

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3 Year Progression Free Survival

"PFS defined as length of time either due to disease recurrence or death, from the time of stem cell infusion (Bone marrow or PBPC) to 3 years.~Response Criteria is measured by the bone marrow aspirate to determine it has leukemic blast. Bone marrow blast less than 5% is considered to be a complete response." (NCT00469144)
Timeframe: 3 years

InterventionDays (Median)
Fixed-Dose Busulfan + Fludarabine42
Adjusted Dose Busulfan + Fludarabine56

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Efficacy of This Therapy 3 Years Post-transplant

Efficacy Assessed as Number of Participants with Overall Survival, Leukemia Progression, Primary Graft Failure and Complete Hematological Response. Primary graft failure is defined as failure to achieve an ANC >/= 0.5 x 10 (9)/L for 3 consecutive days and evidence of donor chimerism by Day +28. Complete hematological response is defined by hemoglobin >/= 120 g/L; or achievement of transfusion independence, with stable Hb > 110 g/L, for RBC transfusion-dependent participants; Spleen not palpable; platelet count 150 x 10 (9)/L; White blood cell 4 x 10 (9)/L to 10 x 10(9)/L. (NCT00475020)
Timeframe: Up to 3 years post-transplant

InterventionParticipants (Count of Participants)
Overall survival at 3 yearsLeukemia progressionPrimary graft failuresComplete hematological remission
Participants With Myelofibrosis and Myelodysplastic Syndrome2527044

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Rate of Non-relapse Mortality at 100 Days Post-transplant

To evaluate the safety of Fludarabine/Busulfan as conditioned regimen for allogeneic stem cell transplantation in patients with myelofibrosis/myelodysplastic syndrome at 100 days post-transplant (NCT00475020)
Timeframe: Non-relapse mortality at 100 days post-transplant

InterventionParticipants (Count of Participants)
InfectionsOrgan FailuresSudden death: likely due to ischemic cardiac event
Participants With Myelofibrosis and Myelodysplastic Syndrome121

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Number of Patients With Neutrophil Engraftment

Number of patients with an absolute neutrophil count >5 x 10^8 cells/liter for 3 consecutive days. (NCT00478244)
Timeframe: Day 42 Post Transplant

Interventionparticipants (Number)
Evaluable Patients6

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Overall Survival

Survival is defined as the number of patients that were alive post transplant. (NCT00478244)
Timeframe: 1 year and 2 years Post Transplant

Interventionparticipants (Number)
1 Year Post Transplant2 Years Post Transplant
Evaluable Patients55

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Number of Patients With Resistance to Blister Formation

Resistance to Blister Formation demonstrated by response to negative pressure. (NCT00478244)
Timeframe: Month 1 through Month 24 Inclusive

Interventionparticipants (Number)
Evaluable Patients2

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Number of Patients With Platelet Engraftment

Number of patients with a platelet count >5 x 10^10 cells/liter for 3 consecutive measurements. (NCT00478244)
Timeframe: Day 180 Post Transplant

Interventionparticipants (Number)
Evaluable Patients5

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Number of Patients With Detectable Collagen Type VII

Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers. (NCT00478244)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Evaluable Patients5

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Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)

Number of patients with cGVHD; a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00478244)
Timeframe: Day 365 Post Transplant

Interventionparticipants (Number)
Evaluable Patients0

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

Number of patients with GVHD. Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00478244)
Timeframe: Day 100 Post Transplant

Interventionparticipants (Number)
Evaluable Patients1

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Number of Patients With Donor Derived Cells in Skin

Number of patients who had donor skin chimerism - donor cells in the patient's epidermis (a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease). (NCT00478244)
Timeframe: Day 90 Post Transplant

Interventionparticipants (Number)
Evaluable Patients6

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Number of Patients With >70% Donor Chimerism

Number of patients with donor chimerism - percentage of donor cells in the patient via the peripheral blood or bone marrow. (NCT00478244)
Timeframe: Days 21, 100, 180, 365 and 730 Post Transplant

Interventionparticipants (Number)
Day 21Day 100Day 180Day 365Day 730
Evaluable Patients65555

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Incidence of Greater Than or Equal to 50% Donor Chimerism

The primary endpoint was achievement of >/= 50% donor chimerism in CD3+ peripheral blood lymphocytes by day +28 (± 7) after allogeneic hematopoietic cell transplantation (allo-HCT). (NCT00496340)
Timeframe: 28 days post-transplant

Interventionpercentage of participants (Number)
Conditioning Followed by HCT100

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Percentage of Participants With Overall Survival (OS)

OS at 2 years post-transplant. OS, defined as time from day of hematopoietic cell infusion to death from any cause. (NCT00496340)
Timeframe: 2 years post-transplant

Interventionpercentage of participants (Number)
Conditioning Followed by HCT68

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Cumulative Incidence of Hematopoietic Cell Engraftment

Hematologic engraftment: defined as time to achieve an absolute neutrophil count (ANC) >/= 500/µl for 3 consecutive days or a platelet count of >/= 20,000//µl without the need for platelet support. (NCT00496340)
Timeframe: 28 days post-transplant

Interventionpercentage of participants (Number)
Conditioning Followed by HCT97.6

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Rate of T-cell (CD3+) and Myeloid (CD33+) Chimerism by Day +28

Median Percentage of Donor Cells in Study Population (Chimerism). (NCT00496340)
Timeframe: 28 days post-transplant

Interventionpercentage of cells (Median)
CD3+CD33+
Conditioning Followed by HCT87100

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Time to Incidence of Graft Versus Host Disease (GVHD)

"The median time from allo-HCT to the initiation of tacrolimus (TAC) taper.~The median time to onset of acute GVHD (aGVHD). Clinical manifestations of acute GVHD include a classic maculopapular rash; persistent nausea and/or emesis; abdominal cramps with diarrhea; and a rising serum bilirubin concentration." (NCT00496340)
Timeframe: Up to 2 years post-transplant

Interventiondays (Median)
Initiation of TAC TaperOnset of aGVHD
Conditioning Followed by HCT6233

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Incidence of Infections

Infections: Incidence of infections (opportunistic and non-opportunistic) following conditioning. (NCT00496340)
Timeframe: Up to 2 years post-transplant

Interventionparticipants (Number)
Cytomegalovirus (CMV) ReactivationEpstein-Barr virus (EBV) ReactivationPost-transplantation Lymphoproliferative DisorderViral InfectionsLung Infections
Conditioning Followed by HCT173152

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Non-relapse Mortality Rate (NRM)

The cumulative incidence of NRM after allo-HCT. (NCT00496340)
Timeframe: Up to 2 years post-transplant

Interventionpercentage of participants (Number)
Day +1002 Years
Conditioning Followed by HCT217

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Rate of T-cell (CD3+) and Myeloid (CD33+) Chimerism by Day +100

Median Percentage of Donor Cells in Study Population (Chimerism). (NCT00496340)
Timeframe: 100 days post-transplant

Interventionpercentage of cells (Median)
CD3+CD33+
Conditioning Followed by HCT96100

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Percentage of Participants With Progression Free Survival (PFS)

PFS at 2 years post-transplant. PFS, defined as time from day of hematopoietic cell infusion to disease relapse. Relapsed disease: Disease was in complete remission post-transplant but returned (e.g., >5% blast in bone marrow or any peripheral blasts). (NCT00496340)
Timeframe: 2 years post-transplant

Interventionpercentage of participants (Number)
Conditioning Followed by HCT55

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Incidence of Graft Versus Host Disease (GVHD)

"By day +100, the cumulative incidence of GVHD, acute of grades 2-4, and 3-4.~At 2 years, the cumulative incidence of chronic GVHD of any severity according to National Institutes of Health (NIH) consensus criteria. Diagnosis of chronic GVHD requires the presence of at least one diagnostic clinical sign of chronic GVHD or the presence of at least one distinctive manifestation confirmed by pertinent biopsy or other relevant tests in the same or another organ. Furthermore, other possible diagnoses for clinical symptoms must be excluded. No time limit is set for the diagnosis of chronic GVHD.~At 2 years, the cumulative incidence of moderate/severe chronic GVHD." (NCT00496340)
Timeframe: Up to 2 years post-transplant

Interventionpercentage of participants (Number)
Day +100 aGVHD Grade 2-4Day +100 aGVHD Grade 3-42 Years, Chronic GVHD of Any Severity2 Years, Moderate/Severe Chronic GVHD
Conditioning Followed by HCT59196958

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Participants' With mCR Response to Post Transplant DLI

Number of participants with response of molecular complete remission (mCR) to DLI as treatment for residual disease after transplant. Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests. (NCT00499889)
Timeframe: 1 year

InterventionParticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin4

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Number of Participants in Complete Molecular Remission at 1 Year

Participants at 1 year in molecular remission, post transplant, post imatinib mesylate and donor lymphocyte infusion (DLI). Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests (this test is most commonly used in clinical trials). (NCT00499889)
Timeframe: Baseline to 1 year

Interventionparticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin21

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Participants' With mCR Response to Post Transplant Imatinib Mesylate Therapy

Number of participants with response of molecular complete remission (mCR) to Imatinib Mesylate therapy as treatment for residual disease after transplant. Molecular remission is a complete remission with no evidence of disease in the blood cells and/or bone marrow using sensitive polymerase chain reaction (PCR) tests. (NCT00499889)
Timeframe: 1 Year

InterventionParticipants (Number)
Mesylate, Busulfan, Fludarabine + Antithymocyte Globulin10

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Number of Participants With Successful Engraftment

Successful Engraftment defined as first of 3 consecutive days with Absolute neutrophil count (ANC) equal to or more than 0.5 * 10^9/L. Failure to engraft by day +30 considered primary engraftment failure. Study period one week prior to transplant through post Day 28. (NCT00502905)
Timeframe: Study period one week prior to transplant through post Day 28

Interventionparticipants (Number)
Busulfan + Fludarabine192

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Maximum Tolerated Dose (MTD)

"Continual reassessment method (four times a day) used to determine an MTD, with a target toxicity probability of 20%, where toxicity is defined as grade 3 or 4 conventional toxicity [National Cancer Institute Common Toxicity Criteria (NCI-CTC)]. Participant evaluation in a cohort with each modality is 30 days." (NCT00506857)
Timeframe: 1 month

Interventionmg/kg (Number)
Busulfan + Fludarabine11.2

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Number of Participants With Graft Versus Host Disease (GVHD)

Tacrolimus and Methotrexate used for acute graft versus host disease (aGVHD) prophylaxis, clinical grading AGVHD criteria (Days 1-100): Grade 1: + to ++ skin rash; no gut involvement; no decrease in clinical performance status; Grade 2: + to +++ skin rash; + gut involvement and/or + liver involvement; mild decrease in performance status; Grade 3: ++ to +++ skin rash; ++ to +++ gut involvement and/or ++ to ++++ liver involvement; marked decrease in performance status; Grade 4: Similar to Grade 3 with ++ to ++++ organ involvement and extreme decrease in performance status. (NCT00506857)
Timeframe: 5 years

InterventionParticipants (Number)
Grade 2Grade 3-4
Tacrolimus + Methotrexate188

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Number of Participant With Adverse Events (AE)

An 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. (NCT00513474)
Timeframe: Up to 71 months

InterventionParticipants (Count of Participants)
Rasburicase Group21
Control Group21

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Uric Acid Levels

Blood was collected and analyzed at a laboratory for serum uric acid levels reported in milligrams(mg)/deciliter(dL). Data is presented for those participants who experienced Grade II to IV aGVHD and those participants who did not experience Grade II to IV aGVHD at pre-transplant and post-transplant. (NCT00513474)
Timeframe: Pre-transplant Day -7 to Day -1 and Post-transplant Day 0 to Day 6

,
Interventionmg/dL (Mean)
Day -7Day -6Day -5Day -4Day -3Day -2Day -1Day 0Day 1Day 2Day 3Day 4Day 5Day 6
Control Group4.1573.4192.9672.5792.3581.8671.712.1632.6712.7782.8052.7582.5792.653
Rasburicase Group0.10.0750.0860.10.0670.0810.4380.9381.6242.0762.2712.5482.5952.705

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Percentage of Participants With Grades II to IV Acute Graft-Versus-Host Disease (aGVHD)

"aGVHD severity was determined using International Bone Marrow Transplant Registry (IBMTR) scale stage and grade of the skin, liver and gut. Stage 1: Skin=maculopapular rash <25% of body surface; Liver=Bilirubin 2-3 mg/dL and Gut=500-999 mL diarrhea/day or peristent nausea with histologic evidence of GvHD. Stage 2: Skin=maculopapular rash 25-50% of body surface; Liver=Bilirubin 3.1-6 mg/dL and Gut=1000-1499 mL diarrhea/day. Stage 3: Skin=maculopapular rash >50% of body surface; Liver=Bilirubin 6.1-15 mg/dL and Gut=≥1500 mL diarrhea/day. Stage 4: Skin=generalized erythroderma with bulla formation; Liver=Bilirubin >15 mg/dL and Gut=severe abdominal pain.~Grade 1: Stage 1-2 rash; no liver or gut involvement. Grade II: Stage 3 rash, or stage 1 liver involvement, or stage 1 gut involvement. Grade III: None to stage 3 skin rash with stage 2-3 liver, or stage 2-4 gut involvement. Grade IV: Stage 4 skin rash, or stage 4 liver involvement." (NCT00513474)
Timeframe: Up to 71 months

Interventionpercentage of participants (Number)
Rasburicase Group24
Control Group57

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Overall Survival Comparing Diagnosis Groups

Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant

Interventionpercentage of survival probability (Number)
AML/1CR67
AML/R150
AML/IF30
Active ALL33

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Overall Survival at 5 Years Post-Transplant.

Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant

Interventionpercentage of survival probability (Number)
Busulfan, FTBI and VP1640

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Disease-free Survival at Five Years Post-transplant

Kaplan-Meier estimate of an event of relapse or death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of transplant to five years post-transplant

Interventionpercentage of survival probability (Number)
Busulfan, FTBI and VP1640

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Disease-Free Survival at 2-Year Post-Transplant

Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment. (NCT00534469)
Timeframe: Estimate at 2 years post treatment

InterventionProportion of pts alive in remission (Number)
HD ARA-C Intermediate Cytogenetics0.65

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Disease-Free Survival at 2-Year Post-Transplant by Cytogenetic Risk

Kaplan-Meier estimates at 2-years post-transplant, with 95% confidence intervals based on logit limit transformation of Greenwood's variance. Outcome is death or relapse, censor is alive in continual complete remission at the date of last clinical disease assessment. (NCT00534469)
Timeframe: Kaplan-Meier estimate 2 years post treatment

InterventionProportion of pts alive in remission (Number)
Unfavorable Risk Cytogenetics0.33
Intermediate Risk Cytogenetics0.75
Favorable Risk Cytogenetics0.75
Unknown Risk: Rejected Cytogenetics Study0.73

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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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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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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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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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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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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)
BuCyCBV
Hodgkin Lymphoma1443

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Maximum Tolerated Dose (MTD) of Intensity-modulated Radiation Therapy (Phase I)

The highest dose tested in which fewer than 33% of patients experienced DLT attributable to the study treatment when at least six patients were treated at the dose and are evaluable for toxicity. The MDT is one dose level below the DLT level. At least six patients will be treated at the MTD. (NCT00540995)
Timeframe: from initial treatment date to Day 30 post-transplant

InterventioncGy (Number)
Arm I: 1200cGy1200

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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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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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Cumulative Incidence of NK Cell Reconstitution

Cumulative incidence of successful reconstitution to donor level is calculated. (NCT00553202)
Timeframe: At 5 years from HSCT date

InterventionPercentage of participants (Number)
Treatment (Chemotherapy and Allogeneic SCT)48.1

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Overall Survival (OS)

OS - Time from HSCT until death (NCT00553202)
Timeframe: At 5 years from HSCT date

InterventionPercentage of participants (Number)
Treatment (Chemotherapy and Allogeneic SCT)45.9

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Disease Relapse

Count of participants with disease relapse at 1 year (NCT00555048)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Alemtuzumab0

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Extensive Chronic GVHD

Count of participants with extensive chronic GVHD at 1 year (NCT00555048)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Alemtuzumab1

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Grades III-IV Acute Graft-vs-host Disease (GVHD)

(NCT00555048)
Timeframe: Up to 100 days

InterventionParticipants (Count of Participants)
Alemtuzumab0

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Graft Failure

Count of participants with graft failure at day 100 (NCT00555048)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Alemtuzumab0

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Life-threatening Infection

(NCT00555048)
Timeframe: Up to 180 days

InterventionParticipants (Count of Participants)
Alemtuzumab0

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Overall Survival

Count of surviving participants at 1 year (NCT00555048)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Alemtuzumab1

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Two-year Overall Survival for All Cases.

Percent Overall Survival (OS) at two years for all patients. (NCT00556452)
Timeframe: 2 years

Interventionpercent overall survival (Number)
Clo/BU428

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One-year Overall Survival Rate for AML

Percent Overall Survival (OS) for at one year for subjects with Acute Myeloid Leukemia (AML). (NCT00556452)
Timeframe: 1 year

Interventionpercent overall survival (Number)
Clo/BU448

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Five Year Overall Survival for All Cases

The number of patients alive at 5 years (NCT00556452)
Timeframe: five years

InterventionParticipants (Count of Participants)
Clo/BU46

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Engraftment Rate After Transplant

Engraftment is defined as an absolute neutrophil count (ANC) >500/microL x 3 days. (NCT00578292)
Timeframe: up to 30 days

Interventionproportion of participants (Number)
Bone Marrow or Stem Cell Infusion1

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Immune Reconstitution

Immune reconstitution: defined as absolute lymphocyte count (ALC) >1000x10e3/microL (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion8

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Hematopoietic Reconstitution

Hematopoietic: defined as transfusion independence. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion7

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Number of Participants With Stable Mixed Hematopoietic Chimerism (HC)

Stable hematopoietic chimerism is defined as having 50-99 percent of donor cells. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion3

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Number of Participants With CHRONIC GVHD

Chronic GVHD is graded by NIH guidelines for chronic GVHD, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3 where 0 means no chronic GVHD, and 3 is the highest stage of chronic GVHD. (NCT00578292)
Timeframe: Assessed monthly from month 3 to month 12

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion1

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Event-free Survival

Event-free survival is calculated from the date of transplant to the date of graft failure, disease recurrence or death from any cause. (NCT00578292)
Timeframe: up to 2 years post transplant

Interventionprobability of event-free survival (Number)
Bone Marrow or Stem Cell Infusion0.7

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Number of Participants With Infectious Complications

All AEs and SAEs (including infections) will be collected for evaluation of infectious complications. (NCT00578292)
Timeframe: up to day 100

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion7

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Number of Participants With Transient Mixed Hematopoietic Chimerism (HC)

Transient mixed hematopoietic chimerism is defined as any mixed chimerism return to 100 percent donor. (NCT00578292)
Timeframe: 1 year post-transplant

InterventionParticipants (Count of Participants)
Bone Marrow or Stem Cell Infusion0

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Number of Participants With Immune Response to Immunization After BMT in Participants With SCD, Hemoglobin SC, or Hemoglobin Sb0/+.

Vaccine response will be measured via a humoral immunity panel evaluating streptococcus pneumonia IgG antibodies (microgram/mL). Panels are obtained pre and 1+ month post vaccination. Standard recommendations define a normal responder as having >4 fold increase in antibody level in 50-70% of the serotypes found in the vaccine. (NCT00578344)
Timeframe: up to 12 months

InterventionParticipants (Count of Participants)
Allogeneic BMT/SCT Transplant0

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Number of Participants Evaluated for Evidence of Recovery of Organ Function Measured Via MRI or PET Scan.

Assess Number of participants that recovered organ function diagnosed with sickle cell disease (SCD) or sickle hemoglobin variants after undergoing allogeneic SCT/BMT from HLA genotype identical donors. (NCT00578344)
Timeframe: One year

InterventionParticipants (Count of Participants)
StableProgressive
Allogeneic BMT/SCT Transplant42

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Number of Patients That Have Complete Donor Chimerism After Transplant.

To estimate the likelihood of complete donor chimerism for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. (NCT00578643)
Timeframe: 120 days post transplant

Interventionparticipants (Number)
Allogeneic Unrelated Transplant13

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Percentage of Participants With Engraftment

To estimate the engraftment rate for patients with CGD using busulfan, cyclophosphamide, fludarabine and alemtuzumab (Campath 1H) as conditioning therapy for SCT from 5/6 or 6/6 HLA-matched unrelated or 5/6 or 6/6 HLA phenotype-matched related donors. (NCT00578643)
Timeframe: 28 days post transplant

Interventionpercentage of participants (Number)
Allogeneic Unrelated Transplant100

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Number of Patients With Successful Donor Engraftment

Each patient will be classified as a success or failure. A success will be defined as engraftment of at least 35% of cells 100 days after transplant. (NCT00579111)
Timeframe: 100 days

Interventionparticipants (Number)
HLA-identical Sibling Transplant2
Unrelated Matched or Single Antigen Mismatched Transplant1

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Number of Participants Experiencing Engraftment Donor Chimerism (EDC)

(NCT00582894)
Timeframe: At time of study termination

InterventionParticipants (Number)
Reduced Intensity Regimen17

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Number of Participants Overall Survival as a Function of Time.

(NCT00582894)
Timeframe: 100 days post transplant

InterventionParticipants (Number)
Reduced Intensity Regimen11

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Number of Participants Relapse-Free

(NCT00582894)
Timeframe: 100 days post-transplant

InterventionParticipants (Number)
Reduced Intensity Regimen5

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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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Number of Participants Evaluated for Toxicities

(NCT00588523)
Timeframe: up to 2 years

InterventionParticipants (Count of Participants)
Participants With Newly Diagnosed Anaplastic Oligodendroglioma60

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Progession Free Survival

To determine the duration of disease control of newly diagnosed pure and mixed anaplastic oligodendrogliomas treated with dose-intensive chemotherapy requiring hematopoietic stem cell support. (NCT00588523)
Timeframe: 2 years

Intervention% of participants without progression (Number)
Participants With Newly Diagnosed Anaplastic Oligodendroglioma85.7

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CD3+ Cell Counts

T-lymphocyte counts (CD3+): mean T-lymphocyte at Baseline and 3 years post gene therapy. Samples were taken from peripheral venous whole blood and tested by cytofluorometry; values are means (10^6/L). (NCT00598481)
Timeframe: baseline up to 3 years post gene therapy

Intervention10^6 cells/L (Geometric Mean)
BaselineYear 1Year 2Year 3
Gene Therapy ITT Population112.5348.3633831.1

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Rate of Severe Infections

Severe infections were defined as those that required hospitalization or those that prolonged hospitalization. The rate of infection was estimated as number of severe infections over person-years of observation (free from severe infections) before and after treatment administration. The first 3 months after gene therapy were not considered in the post-gene therapy analysis, because all subjects were hospitalized during this period. (NCT00598481)
Timeframe: Before Treatment and 3-months post-treatment up to 3 years

InterventionSevere Infections per person year (Number)
Severe Infections (Before Gene Therapy)1.1
Severe Infections (After Gene Therapy)0.429

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Survival

From post-treatment to up to 3 years (NCT00598481)
Timeframe: baseline to 3 years post gene therapy

InterventionParticipants (Count of Participants)
Gene Therapy (Pivotal)12

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Transplantation-related Mortality at 100 Days Post-transplantation

(NCT00611351)
Timeframe: at the 100 days post-transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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Overall Survival

(NCT00611351)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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

(NCT00611351)
Timeframe: at day 100 post transplantation

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic4

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Event-free Survival

(NCT00611351)
Timeframe: 2 years post transplant

InterventionParticipants (Count of Participants)
Unrelated Donor Allogeneic2

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Number of Participants With Relapse of Malignancy

the return of disease after its apparent recovery/cessation. (NCT00612716)
Timeframe: 3 Years

InterventionParticipants (Count of Participants)
Allogeneic Transplantation1

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Number of Participants With Platelet Engraftment

Time to platelets > 20,000 (first of 3 consecutive days) with no platelet transfusions for seven days and percentage of patients with platelet engraftment >50,000 by day 100. (NCT00612716)
Timeframe: Day 180

InterventionParticipants (Count of Participants)
Allogeneic Transplantation2

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Number of Participants With Persistence Disease

the return of disease after its apparent recovery/cessation. (NCT00612716)
Timeframe: 3 Years

InterventionParticipants (Count of Participants)
Allogeneic Transplantation1

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Number of Participants With Neutrophil Engraftment

Time to 1st 3 consecutive days with absolute neutrophil count (ANC) > 5 x 10^8/L and percentage of patients with neutrophil recovery by day 42 (Cumulative incidence). (NCT00612716)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Allogeneic Transplantation5

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Number of Participants With Grade 3-4 Acute Graft-versus-host Disease

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. (NCT00612716)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Allogeneic Transplantation3

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Number of Participants With Engraftment Failure

Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets. (NCT00612716)
Timeframe: 3 Months

InterventionParticipants (Count of Participants)
Allogeneic Transplantation1

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Number of Participants With 2 Year 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.~Overall survival will be defined as time from date enrollment to date of death or censored at the date of last documented contact for patients still alive." (NCT00612716)
Timeframe: 2 year

InterventionParticipants (Count of Participants)
Allogeneic Transplantation1

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Number of Participants With 1 Year 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.~Overall survival will be defined as time from date enrollment to date of death or censored at the date of last documented contact for patients still alive." (NCT00612716)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Allogeneic Transplantation2

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Number of Participants With of Chronic GVHD.

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. (NCT00612716)
Timeframe: 1 Year

InterventionParticipants (Count of Participants)
Allogeneic Transplantation0

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The Percentage of Patients Alive 1 Year Post Transplant

The primary objective is overall survival, one year from the time of transplant. (NCT00615589)
Timeframe: 1 Year

Interventionpercentage of patients (Number)
Flu-Bu461

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The Percentage of Patients Free From Progression at 1 Year

"One of the secondary outcomes that will be measured is progression free survival at 1 Year.~Progressive Disease (PD) is defined as a >25% increase in serum monoclonal paraprotein, a >25% increase in 24-hour urinary light chain excretion, a >25% increase in plasma cells in bone marrow aspirate, an increase in the size or the development of new bone lesions/soft tissue plasmacytomas, or the development of hypercalcemia." (NCT00615589)
Timeframe: 1 Year

Interventionpercentage of patients (Number)
Flu-Bu440

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Non Relapse Mortality (NRM) at 1 Year and 3 yearsThe Percentage of Deaths Not Attributable to Disease Relapse or Progression

Non relapse mortality, defined as the percentage of deaths not attributable to disease relapse or progression at 1 year and at 3 years. (NCT00615589)
Timeframe: 3 years

Interventionpercentage of deaths (Number)
NRM at 1 YearNRM at 3 Years
Flu-Bu41929

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Percentage of Patients With Acute and Chronic Graft Versus Host Disease (GVHD)

"Incidence of acute (Stage II-IV and Stage III-IV) and chronic GVHD (any stage) were analyzed.~Acute GVHD Grading:~Stage II - Skin, 25-50% BSA (Body Surface Area); Liver, 3.1-6mg/dl bilirubin; Gut, 1000-1500ml/day diarrhea Stage III - Skin, generalized erythroderma; Liver, 6.1-15mg/dl bilirubin; Gut, >1500ml/day diarrhea Stage IV - Skin, bullae; Liver, >15mg/dl bilirubin; Gut, pain +/- ileus" (NCT00615589)
Timeframe: 100 days, 2 years

Interventionpercentage of participants (Number)
Grade II-IV Acute GVHDGrade III-IV Acute GVHDChronic GVHD
Flu-Bu4482355

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Number of Patients Alive 24 Months Post Day 100 Transplant

Patients will be followed for survival for 24 months post day 100 transplant. (NCT00619645)
Timeframe: 24 months post day 100 transplant

InterventionParticipants (Count of Participants)
RIST for Heme Malignancies3

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Percentage of Participants With Relapse Free Survival at 1 Year

The primary objective was to determine the 1 year relapse free survival rate (RFS) for individuals > 55 years in age with Acute myeloid leukemia (AML) in Complete Remission (CR) or Partial Remission (PR) who undergo a 7-8/8 HLA- matched unrelated donor transplant using a reduced intensity regimen. (NCT00623935)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Fludarabine Plus Busulfan56

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Percentage of Participants Alive at 1 Year

One of the secondary objectives was to determine overall survival for patients > 55 years in age with AML undergoing full or reduced transplant with the best available donor. (NCT00623935)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Fludarabine Plus Busulfan58

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Number of Patients Achieving Donor Cell Engraftment

Number of patients with persistent presence of donor-derived cells at Day 100 (NCT00638820)
Timeframe: Day 100

InterventionParticipants (Number)
Patients With Osteopetrosis Who Received Transplant2

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Number of Patients Achieving Engraftment

Rate of successful engraftment - patients who achieved and sustained donor engraftment; donor chimerism by day 100 of at least 90% after undergoing hematopoietic stem cell transplantation. (NCT00668564)
Timeframe: Day 100

InterventionParticipants (Number)
Intent-to-Treat14

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Overall Survival

Number of patients alive at timepoints. (NCT00668564)
Timeframe: Day 100, 1 Year, 3 Years

InterventionParticipants (Number)
Day 1001 Year
Intent-to-Treat1412

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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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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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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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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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Number of Patients Who Achieved Donor Cell Engraftment

(NCT00775931)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Marrow Graft Transplant Conditioning5
Cord Blood Transplant Conditioning0

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Incidence of Grade II - IV Acute Graft-versus-host Disease

(NCT00775931)
Timeframe: by Day 100 after transplant

InterventionParticipants (Count of Participants)
Marrow Graft Transplant Conditioning2
Cord Blood Transplant Conditioning0

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Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)

(NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant2

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Number of Patients Who Experienced Severe Graft-versus-host Disease (GVHD)(Grade 3 or 4)

Number of patients who experienced post-transplant complication (GVHD) as seen by clinical evidence (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant2

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Overall Survival at 12 Months

Overall survival, defined as a patient being alive after transplant, is without regard to disease status. (NCT00782379)
Timeframe: 12 months

Interventionparticipants (Number)
Haploidentical Transplant14

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Overall Survival at Day 100

Overall survival is assessed, without regard to disease status, post-transplant, at Day 100. (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant17

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR (polymerase chain reaction) for STR/VNTR (short tandme repeat/variable number tandem repeat) will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 30

Interventionparticipants (Number)
Haploidentical Transplant18

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 60 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 60

Interventionparticipants (Number)
Haploidentical Transplant18

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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 90 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 90

Interventionparticipants (Number)
Haploidentical Transplant13

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Disease Free Survival at 12 Months

Disease free survival refers to patients with no evidence of disease after transplant, at the 12 month time point. (NCT00782379)
Timeframe: 12 months

Interventionparticipants (Number)
Haploidentical Transplant7

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Disease Free Survival at Day 100

Disease free survival refers to patients with no evidence of disease after transplant, at the Day 100 time point. (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant16

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Incidence of Graft Rejection for Patients at Day 100

Number of patients who experienced graft rejection by Day 100 (NCT00782379)
Timeframe: Day 100

Interventionparticipants (Number)
Haploidentical Transplant0

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Non-relapse Mortality at 1 Year After Peripheral Blood Stem Cell Transplantation (PBSCT)

Non-relapse mortality refers to whether a patient dies of causes related to something other than the primary disease. (NCT00782379)
Timeframe: 1 year

Interventionparticipants (Number)
Haploidentical Transplant2

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T-cell and Myeloid Chimerism at Days 180 Post-transplantation (>90%)

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00787761)
Timeframe: 180 days

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan19

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T-cell and Myeloid Chimerism at Days 90 Post-transplantation (>90% Chimerism)

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00787761)
Timeframe: Day 90

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan17

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Disease-free Survival (DFS) at 24 Months

Disease Free survival is measured by the amount of time a patient spends in a disease free state after being transplanted. (NCT00787761)
Timeframe: 24 months

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan13

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Number of Patients Who Experience Severe (Grade 3 or 4) Acute Graft-versus-host Disease

number of patients who experienced post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea. (NCT00787761)
Timeframe: Day 100

Interventionparticipants (Number)
Severe Graft Versus Host Disease2

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Number of Patients Experiencing Extensive Chronic Graft Versus Host Disease (GVHD)

Patients who had post-transplant complication (GVHD) as seen by clinical evidence including but not limited to skin rash, elevated liver function tests, nausea/vomiting/diarrhea. (NCT00787761)
Timeframe: 2 years

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan13

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Non-relapse Mortality (NRM) at Day 180 Post-transplantation

non-relapse mortality refers to the death of a patient for causes other than relapsed disease. (NCT00787761)
Timeframe: Day 180

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan0

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Overall Survival (OS) at 24 Months

Overall survival refers to the length of time a patient is alive after transplant regardless of whether they have progressive or relapsed disease. (NCT00787761)
Timeframe: 24 months

Interventionparticipants (Number)
RIC Transplant Using ATG, Busulfan, Fludarabine and Cytoxan16

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Achievement of > 90% (Full) Donor Chimerism in the T-cell Lineage as Measured by PCR at Day 30 Post-transplantation

Chimerism analysis of peripheral blood mononuclear cells using PCR for STR/VNTR will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00787761)
Timeframe: Day 30

Interventionparticipants (Number)
Transplant Recipients12

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Number of High Risk Pediatric Patients With Successful Sustained Donor Engraftments

Assessed donor engraftment in very high risk pediatric patients. (NCT00795132)
Timeframe: 24 months

Interventionparticipants (Number)
Related BM PBSC16
Unrelated BM PBSC18
Unrelated Cord Blood10

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Two Year Overall Survival

The probability that a given patient will be alive two years after transplantation. (NCT00795132)
Timeframe: 24 months

Interventionprobability (Mean)
Related BM PBSC0.501
Unrelated BM PBSC0.395
Unrelated Cord Blood0.438

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Non-relapse Mortality

Death of any cause other than relapse or disease progression was considered. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)8
Arm II (High Risk for Graft Failure)10

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Incidence of Clinically Significant Infections

Collected and graded according to the modified National Cancer Institute Common Toxicity Criteria. (NCT00796068)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)53
Arm II (High Risk for Graft Failure)61

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

Overall GVHD is determined based on the organ stage, response to treatment and whether GVHD was a major cause of death. Chronic GVHD will be defined according to the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Described as mild, moderate, or severe as graded according to the attached Organ Scoring Sheet. Symptoms consistent with both chronic and acute GVHD occurring after day 100 will be considered overlap chronic GVHD syndrome. (NCT00796068)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)7
Arm II (High Risk for Graft Failure)6

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Duration (Days) Until Participants Obtained Platelet Engraftment

Summarized using a range and median of measure in days until participants reached platelet engraftment. Platelet engraftment was defined as the first of 7 consecutive days when the platelet count exceeded 20 x 109/L (untransfused). (NCT00796068)
Timeframe: At 6 months

Interventiondays (Median)
Arm I (Low Risk for Graft Failure)31
Arm II (High Risk for Graft Failure)31

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Number of Participants Surviving up to 2 Years Without Disease Progression

Progression-free survival is the time interval from start of treatment to documented evidence of disease progression. Disease progression was defined by European LeukemiaNet 2017 criteria and International Working Group (IWG) within 3 years of transplant. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)37
Arm II (High Risk for Graft Failure)41

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The Number of Participants Alive at Two-years Follow up.

Overall survival of participants after two-years of follow up. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)50
Arm II (High Risk for Graft Failure)44

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Number of Patients With Non-relapse Mortality (NRM)

Defined as death from any cause without sign of disease progression or relapse. Loss to follow up are censored, whereas disease relapse or progression are considered competing risks. (NCT00796068)
Timeframe: At day -200

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)7
Arm II (High Risk for Graft Failure)6

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Number of Participants With Secondary Graft Failure

Secondary graft failure is defined as decline of neutrophil count to <500/ul with loss of donor chimerism after day 55 (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)0
Arm II (High Risk for Graft Failure)0

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Number of Participants With Graft Failure/Rejection

"Patients will be considered primary graft failure provided they meet any criteria listed below, in the absence of documented disease persistence/recurrence or active bone marrow infection (ex. Cytomegalovirus (CMV)):~i. Absence of 3 consecutive days with neutrophils >500/u1 combined with host CD3+ peripheral blood chimerism ≥50% at day 42 ii. Absence of 3 consecutive days with neutrophils >500/u1 under any circumstances at day 55 iii. Death after day 28 with neutrophil count <100/u1 without any evidence of engraftment (< 5% donor CD3+) iv. Primary autologous count recovery with < 5% donor CD3+ peripheral blood chimerism at count recovery and without relapse" (NCT00796068)
Timeframe: Up to 100 days

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)2
Arm II (High Risk for Graft Failure)5

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Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100

Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. Diagnosing and grading acute GVHD is based on clinical findings (the organ stage, response to treatment and whether GVHD was a major cause of death) and frequently varies between transplant centers and independent reviewers. (NCT00796068)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)36
Arm II (High Risk for Graft Failure)37

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Incidence of Relapse or Disease Progression

Cumulative incidence estimates using non-relapse mortality as competitive event. (NCT00796068)
Timeframe: Up to 2 years

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)17
Arm II (High Risk for Graft Failure)11

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Number of Participants Surviving by 1 Year

Overall survival was measured from the first day of CBT infusion until death from any cause. (NCT00796068)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I (Low Risk for Graft Failure)51
Arm II (High Risk for Graft Failure)47

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Engraftment as Measured by Donor Chimerism

Percentage of participants who achieved donor chimerism >=95%. (NCT00796562)
Timeframe: Day 60

Interventionpercentage of participants (Number)
Myeloablative Haploidentical BMT91

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Survival

Percentage of participants alive (overall survival) and alive without disease relapse, progression, or diagnosis of myeloid malignancy (event-free survival). Estimated using Kaplan-Meier method. (NCT00796562)
Timeframe: 1 year, 2 years, 3 years

Interventionpercentage of participants (Number)
Overall survival, 1 yearOverall survival, 2 yearsOverall survival, 3 yearsEvent-free survival, 1 yearEvent-free survival, 2 yearsEvent-free survival, 3 years
Myeloablative Haploidentical BMT735754585250

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Non-relapse Mortality

Number of participants deceased for reasons other than disease relapse or progression. (NCT00796562)
Timeframe: Day 100, 1 year

InterventionParticipants (Count of Participants)
100 days1 year
Myeloablative Haploidentical BMT1010

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Acute GVHD

"Percentage of participants who experience grade II-IV or III-IV acute graft-versus-host-disease (GVHD) by Przepiorka criteria. The stages are defined as follows:~Stage II: Rash on >50% of skin, bilirubin 2-3 mg/dL, or diarrhea 500-1000 mL/day~Stage III: Bilirubin 3-15 mg/dL or diarrhea >1000 mL/day~Stage IV: Generalized erythroderma with bullae or bilirubin >15 mg/dL This outcome was estimated using proportional subdistribution hazard regression model for competing risks (Fine and Gray 1999)" (NCT00796562)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Grade II-IVGrade III-IV
Myeloablative Haploidentical BMT114

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Chronic GVHD

Percentage of participants who experience chronic GVHD. Chronic GVHD is graded using NIH consensus criteria and Seattle criteria. This outcome was estimated using proportional subdistribution hazard regression model for competing risks (Fine and Gray 1999) (NCT00796562)
Timeframe: 6 months, 12 months

Interventionpercentage of participants (Number)
6 months12 months
Myeloablative Haploidentical BMT415

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Relapse

Percentage of participants who experienced disease progression or relapse. This outcome was estimated using proportional subdistribution hazard regression model for competing risks (Fine and Gray 1999) (NCT00796562)
Timeframe: 1 year, 3 years

Interventionpercentage of participants (Number)
1 year3 years
Myeloablative Haploidentical BMT3543

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2-year Progression-Free Survival

Progression-free survival (PFS) is defined as the interval between day of transplant and day of death or disease progression. (NCT00800839)
Timeframe: First 25-35 days post transplant and then every 3 months for a maximum of 2 years

Interventionpercentage of participants (Number)
Busulfan + Fludarabine + Cyclophosphamide26

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Cumulative Incidence of Grade III to IV Acute GVHD

Graft Versus Host Disease (GVHD) defined as grade 3 to 4 GVHD within first 100 days post transplant. Death or disease progression before diagnosis of GVHD were considered competing risks in the estimation of the incidence of acute GVHD. (NCT00800839)
Timeframe: 100 days post transplant

Interventionpercentage of incidence (Number)
Busulfan + Fludarabine + Cyclophosphamide22

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Cumulative Incidence of Grade II to IV Acute GVHD

Graft Versus Host Disease (GVHD) defined as grade 2 to 4 GVHD within first 100 days post transplant. Death or disease progression before diagnosis of GVHD were considered competing risks in the estimation of the incidence of acute GVHD. (NCT00800839)
Timeframe: 100 days post transplant

Interventionpercentage of incidence (Number)
Busulfan + Fludarabine + Cyclophosphamide53

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2-year Overall Survival

Overall Survival (OS) is defined as the interval between day of transplant and day of death. (NCT00800839)
Timeframe: First 25-35 days post transplant and then every 3 months for a maximum of 2 years

Interventionpercentage of participants (Number)
Busulfan + Fludarabine + Cyclophosphamide33

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Rate of Engraftment

Engraftment defined as the evidence of donor derived cells (more than 5%) by bone marrow chimerism studies in the presence of neutrophil recovery by day 28 post stem cell (SC) infusion. Engraftment date is the first day of three (3) consecutive days that the ANC exceeds 0.5 x109/L. Delayed engraftment is defined as the evidence of engraftment beyond day 28 post SC infusion achieved after the administration of therapeutic (high dose) hematopoietic growth factors. (NCT00800839)
Timeframe: From engraftment to 60 days post transplant

Interventiondays (Median)
Busulfan + Fludarabine + Cyclophosphamide18

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Time to Platelet Engraftment

Following MAC AutoSCT, the median time to platelet recovery will be measured. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventiondays (Mean)
Arm A - Family Donor22
Arm B - Unrelated Cord Blood or Adult53

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Total Number of Subjects With a Complete Response (CR) Following Myeloablative Conditioning (MAC) and Autologous Stem Cell Transplantation (AutoSCT)

Complete Response is defined as the complete resolution of B symptoms (i.e., weight loss, night sweats and fever) and normalization of all sites of disease on the basis of physical exam, bone marrow biopsy, and imaging studies. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor4
Arm B - Unrelated Cord Blood or Adult12

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Total Number of Subjects With Partial Response or Stable Disease Following MAC AutoSCT

Total includes subjects with partial response and patients with stable disease, defined as <50% reduction in measurable disease or the uninterrupted persistence of B symptoms. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor4
Arm B - Unrelated Cord Blood or Adult9

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Total Number of Subjects With Grade II-IV Acute Graft-versus-Host-Disease (GVHD)

The criteria for grading is based on extent of organ involvement (i.e., Skin, Liver and Gut - rash on >50% of skin, bilirubin 2-3 mg/dl, diarrhea > 500 ml/day) with Grade II being better outcome and Grade IV being worse outcome. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventionparticipants (Number)
Arm A - Family Donor0
Arm B - Unrelated Cord Blood or Adult7

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Time to Neutrophil Engraftment

Following MAC AutoSCT, the median time to neutrophil (PMN) recovery will be measured. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

Interventiondays (Mean)
Arm A - Family Donor16
Arm B - Unrelated Cord Blood or Adult24

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Total Number of Subjects With a Disease Relapse or Progression Following MAC AutoSCT

Includes subjects with any measurable growth of disease in a previously affected site or detection of disease in a new site confirmed by biopsy. (NCT00802113)
Timeframe: Up to 1 year post-transplantation

InterventionParticipants (Count of Participants)
Arm A - Family Donor1
Arm B - Unrelated Cord Blood or Adult3

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To Determine the Optimal Regimen of Post-graft Immunosuppression With High-dose Cy Following Fludarabine, Busulfan, and Transplantation of Fully HLA-matched Bone Marrow That Leads to an Acceptable Incidence of Grades III/IV Acute GVHD

Percentage of participants with grade III-IV acute graft versus host disease (GVHD). GVHD is graded on a combination of skin symptoms (rash), gut symptoms (diarrhea), and liver symptoms (using a lab test called bilirubin). Grades range from I to IV, where I is the least severe and IV is the most severe. (NCT00809276)
Timeframe: 1 year

Interventionpercentage of participants (Number)
BuFlu Transplant15

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Number of Participants With Molecular Response

Molecular Remission defined as two consecutive bone marrow samples done one month apart with negative PCR( polymerase chain reaction) tor CML. (NCT00813124)
Timeframe: 12 month post BMT

InterventionParticipants (Count of Participants)
Complete ResponsePartial ResponseProgressive Disease
Azacytidine Maintenance After Allotx804

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Complete Donor Chimerism

Complete donor chimerism (defined as >/= 95% donor cells in peripheral blood CD3+ and CD33+ was measured. (NCT00818961)
Timeframe: 2 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation26

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Neutrophil Recovery

The number of patients experiencing neutrophil recovery post transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Number of Patients Requiring the Use of Donor Leukocyte Infusion (DLI) for Early Mixed T-cell Chimerism

DLI is used for patients with mixed chimerism following transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation19

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Overall Survival at 1 Year

Evaluation of overall survival at 1 year (# of patients who are alive at 1 year post-transplant) (NCT00818961)
Timeframe: 1 year

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation26

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Platelet Engraftment

The number of patients experiencing platelet engraftment post-transplant (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Survival at Day 100

Survival at Day 100 (NCT00818961)
Timeframe: 100 day

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation35

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Non-relapse Mortality at Day 100

patients are evaluable for their cause of death at Day 100 (NCT00818961)
Timeframe: Day 100

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation1

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Number of Patients Experiencing Chronic Graft Versus Host Disease

(NCT00818961)
Timeframe: >100 days post-transplant

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation20

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Number of Patients Experiencing Grade 2-4 Acute Graft-versus-host Disease Post-transplant

patients experiencing acute graft versus host disease post-transplant (NCT00818961)
Timeframe: patients were followed for 2 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation15

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Non-relapse Mortality at 1 Year Post-transplant

Number of patients who died of non-relapse causes at one year. this is in clusive of all patients who were transplanted on study even though only 10 patients died at by 1 year time point. This outcome will be referenced in the donor chimerism outcome. Only 26/36 patients were eligible for this time point as that is all that were alive. (NCT00818961)
Timeframe: 1 year

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation4

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Number of Patients Experiencing Veno-occlusive Disease (VOD) Post-transplant

Patients will be evaluated up to 4 years post transplant (NCT00818961)
Timeframe: 4 years

Interventionparticipants (Number)
Hematopoietic Stem Cell Transplantation0

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Number of Participants Alive With no Disease Progression at Time of Allo Transplant

In Phase II portion of study, number of participants with treatment failure defined as either disease recurrence or death, measured from start of treatment to allo transplant at Day 0. (NCT00822770)
Timeframe: Baseline till transplant, Day -9 to Day 0, to 10 days

Interventionpercentage of participants (Number)
Participants inParticipants not in Complete Remission
Overall Study: Plerixafor + G-CSF1616

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Number of Participants With Grade 4 Dose Limiting Toxicity to Determine Maximum Tolerated Dose (MTD) Plerixafor

Phase I determination of MTD dose of Plerixafor in combination with a fixed dose of Filgrastim where dose limiting toxicity defined as any grade 4 non-hematologic toxicity observed within 28 days from Day 0 (day of transplant). (NCT00822770)
Timeframe: 28 day cycle (Plerixafor Day -7 to Day -4)

Interventionparticipants (Number)
0 mcg/kg daily80 mcg/kg daily160 mcg/kg daily240 mcg/kg daily
Phase I Plerixafor + G-CSF1474

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Engraftment Response Rate: Number of Transplanted Participants With Complete Chimerism at Day 30

Number of participants with complete chimerism at day 30 where defined as: Engraftment: first day of three (3) consecutive days that Absolute neutrophil count (ANC) exceeds 0.5 X 109/L. Subsequent chimerism studies must demonstrate the presence of donor derived cells. Graft Failure: failure to achieve an ANC >0.5 X 109/L for 3 consecutive days within 28 days after transplantation or a decline of ANC <0.5 x 109/L for three consecutive days after initial documented engraftment unless this is correlated with progression / recurrence of the underlying malignancy. (NCT00822770)
Timeframe: 30 Days post engraftment

InterventionParticipants (Count of Participants)
Overall Study: Plerixafor + G-CSF32

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Relapse Rate of Participants Treated With Thiotepa, Busulfan, and Clofarabine

Relapse Rate will be estimated using the Kaplan-Meier method. (NCT00857389)
Timeframe: Up to 2 years post transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine26

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Overall Survival Rate

Overall Survival Rate will be estimated using the Kaplan-Meier method. (NCT00857389)
Timeframe: Up to 3 years post transplant

Interventiondays (Median)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine320

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Number of Participants With Serious Adverse Events

Severity of toxicities graded according to the NCI Common Toxicity Criteria Adverse Effects (CTCAE) v3.0. Standard reporting guidelines followed for adverse events. Safety data summarized by category, severity and frequency. (NCT00857389)
Timeframe: up to 30 days post transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine52

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Number of Participants With Disease Free Survival

Kaplan-Meier product limit method to estimate the disease free survival. (NCT00857389)
Timeframe: Up to 2 years post transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine32

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Graft vs Host Disease (GVHD)

Severity of toxicities graded according to the NCI Common Toxicity Criteria Adverse Effects (CTCAE) v3.0. Standard reporting guidelines followed for adverse events. Safety data summarized by category, severity and frequency. (NCT00857389)
Timeframe: Up to 30 days post transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine34

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Engraftment

Engraftment is most commonly defined as the first of three consecutive days of achieving a sustained peripheral blood neutrophil count of >500 × 10^6/L . (NCT00857389)
Timeframe: up to 100 days post transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine52

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Number of Participants With Survival Rate at 100 Days Post-transplant

The toxicities will be monitored and scored on a daily basis following the methods of Simon R. Practical Bayesian Guideline for Phase IIB Clinical Trials. A Bayesian stopping rule will be used to stop the trial if there is a 90% chance that the true toxicity rate exceeds the target toxicity rate of 0.25. (NCT00857389)
Timeframe: 100 days post-transplant

InterventionParticipants (Count of Participants)
Conditioning Reg- Thiotepa, Busulfan, and Clofarabine45

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Number of Patients With Grade II-IV Acute Graft-versus-host Disease

Number of patients diagnosed with overall grade II-IV acute GVHD by Day 100 post transplant (NCT00919503)
Timeframe: Day 100 post transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)44
Regimen B (UBCT)8

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Immune Reconstitution Following Hematopoietic Cell Transplantation

Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)39
Regimen B (UBCT)4

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Disease Response at One Year Following Hematopoietic Cell Transplantation

Number of patients with no evidence of disease at one year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)78
Regimen B (UBCT)8

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Non-relapse Mortality

Number of patients who experienced non-relapse mortality by 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)2
Regimen B (UBCT)5

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Number of Participants With Infections

Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant (NCT00919503)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)57
Regimen B (UBCT)9

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Number of Patients With of Chronic Graft-versus-host Disease

Number of patients diagnosed with chronic GVHD and requiring systemic immunosuppression within 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)29
Regimen B (UBCT)5

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Overall Survival

Number of patients alive at 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)80
Regimen B (UBCT)9

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Preliminary Efficacy

Number of patients engrafted (>5% donor CD3+ peripheral blood chimerisms) at 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant

InterventionParticipants (Count of Participants)
Regimen A (PBSCT and BMT)83
Regimen B (UBCT)10

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Donor Chimerism CD3 at 100 Days Post Transplant

Number of patients with peripheral blood donor chimerism for CD3 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. (NCT00919503)
Timeframe: Day 100 post transplant

,
InterventionParticipants (Count of Participants)
Greater than equal to 95%50 - 94%5-49%Less than 5%
Regimen A (PBSCT and BMT)492960
Regimen B (UBCT)7211

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Donor Chimerism CD33 at Day 100 Post Transplant

Number of patients with peripheral blood donor chimerism for CD33 less than 5%, 5-49%, 50-94% and greater than or equal to 95% at 100 days post transplant. (NCT00919503)
Timeframe: 100 days post transplant

,
InterventionParticipants (Count of Participants)
Greater than equal to 95%50-94%5-49%Less than 5%
Regimen A (PBSCT and BMT)72840
Regimen B (UBCT)7221

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Relapse-free Survival

Number of participants without progressive disease at the end of the study period, using the definitions for complete response, partial response and progressive disease from the International Myeloma Working Group . (NCT00941720)
Timeframe: at 6 months

Interventionparticipants (Number)
Busulfan Treatment48

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Pulmonary Toxicity

Toxicity criteria will be assessed and graded according to the National Cancer Institute (NCI) Common Terminology Criteria (CTC) v3.0. Estimated using exact 95% binomial confidence intervals. (NCT00941720)
Timeframe: At 6 months

Interventionpercentage of participants (Number)
Busulfan Treatment3.5

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Overall Survival

Number of patients alive at the end of the study period (NCT00941720)
Timeframe: at 6 months

Interventionparticipants (Number)
Busulfan Treatment53

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Maximum Tolerated Dose

Maximally tolerated area under the curve of intravenous busulfan (Busulfan®) in combination with fludarabine as conditioning regimen for transplantation with in-vivo T-cell depletion. The number reported will be an Area Under the Curve (AUC) measure reported in µmol-min/L. (NCT00943319)
Timeframe: 5 years

Interventionmmol-min/L (Number)
Busulfan and Fludarabine6800

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Disease Free Survival

Disease Free Survival measured by median survival time in days (NCT00943319)
Timeframe: 5 years

Interventiondays (Median)
Busulfan and Fludarabine172

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Overall Survival

Overall Survival measured as median survival in days (NCT00943319)
Timeframe: 5 years

Interventiondays (Median)
Busulfan and Fludarabine161

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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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Number of Progression Events in 2 Years.

The time of Progression-Free Survival (PFS) was defined as the time from transplantation to the occurrence of the event that was death or first recurrence of progressive disease. (NCT00948090)
Timeframe: 2 years

InterventionEvent (Number)
Hodgkin's Lymphoma (≤ 65 Years)41
Hodgkin's Lymphoma (> 65 Years)1
Non-Hodgkin's Lymphoma (≤ 65 Years)46
Non-Hodgkin's Lymphoma (> 65 Years)8

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Overall Response Rate

The overall response status is complete response and not complete response (partial remission, primary refractory/primary induction failure, stable disease, progressive disease, and relapse) at Baseline and each of the scheduled follow-up time points. (NCT00948090)
Timeframe: Baseline, Day 100, Month 6, 12, 24, Early termination and End of Trial (within 30 days of the trial termination)

,,,
InterventionParticipants (Number)
Baseline-Complete Response (N=65, 0, 121, 16)Baseline-Not Complete Response (N=65, 1, 121, 16)Day 100-Complete Response (N=57, 0, 114, 12)Day 100-Not Complete Response (N=57, 0, 114, 12)Month 6-Complete Response (N=45, 0, 98, 11)Month 6-Not Complete Response (N=45, 0, 98, 11)Month 12-Complete Response (N=32, 0, 88, 8)Month 12-Not Complete Response (N=32, 0, 88, 0)Month 24-Complete Response (N=13, 0, 28, 6)Month 24-Not Complete Response (N=13, 0, 28, 0)Early Term-Complete Response (N=40, 0, 41, 0)Early Term-Not Complete Response (N=40, 0, 41, 5)End of Trial-Complete Response (N=22, 0, 71, 9)End of Trial-Not Complete Response(N=22, 0, 71, 9)
Hodgkin's Lymphoma (> 65 Years)01000000000000
Hodgkin's Lymphoma (≤ 65 Years)3530352230152210112238202
Non-Hodgkin's Lymphoma (> 65 Years)124849280600581
Non-Hodgkin's Lymphoma (≤ 65 Years)7249852984147117262437656

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Number of Death Events in 2 Years.

The time of overall survival was defined as the time from transplantation to death of all causes. (NCT00948090)
Timeframe: 2 years

InterventionDeaths (Number)
Hodgkin's Lymphoma (≤ 65 Years)13
Hodgkin's Lymphoma (> 65 Years)1
Non-Hodgkin's Lymphoma (≤ 65 Years)28
Non-Hodgkin's Lymphoma (> 65 Years)6

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Number of Participants With Chronic GvHD

Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3. (NCT00950846)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant Treatment Plan1

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Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 1 year

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.868

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Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 100 days

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.947

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Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT00950846)
Timeframe: 3 years

Interventionprobability of overall survival (Number)
Umbilical Cord Blood Transplant Treatment Plan0.868

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Number of Participants With Donor Engraftment After Transplant.

To evaluate donor engraftment at 100 days, 6 and 12 months after transplant. (NCT00950846)
Timeframe: 100 days, 6 months and 12 months

InterventionParticipants (Count of Participants)
100 days6 months12 months
Umbilical Cord Blood Transplant Treatment Plan363333

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Incidence of Severe Grade III-IV Acute GvHD at Day 100.

Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD. (NCT00950846)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant Treatment Plan1

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Overall Survival at 3 Years

Overall Survival is defined as time from date of transplant to event (death from any cause) or last follow-up. (NCT00987480)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected80

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Disease-free Survival at 3 Years

"Defined as time from date of transplant to relapse, graft rejection or graft failure, or death.~Primary non-engraftment is diagnosed when the participants fails to achieve an ANC >/= 500/ul at any time in the first 28 days post-transplant.~For participants with MDS or AML, relapse will be analyzed as to type and genetic origin of the MDS/leukemic cells. These will be defined by an increasing number of blasts in the marrow over 5% by the presence of circulating peripheral blasts, or by the presence of blasts in any extramedullary site. Cytogenetic analysis of the marrow and/or peripheral blood will also be obtained for the diagnosis of relapse." (NCT00987480)
Timeframe: 3 years

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected77.8

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The Incidence of Acute GvHD

(NCT00987480)
Timeframe: 100 days

Interventionpercentage of participants (Number)
Chemotherapy-based Cytoreductive Regimen Plus a CD34+ Selected6.7

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Percentage of Participants With Overall Disease Response at Month 6

The percentage of participants reported in each disease category by International Myeloma Working Group (IMWG) uniform response criteria for Multiple Myeloma 6 months after autologous Hematopoietic stem cell transplant. Overall Disease Response categories were: [stringent Complete Response (sCR)=CR + normal Free Light Chain (FLC) ratio + absence of clonal cells in bone marrow], [Complete Response (CR)=Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and ≤5% plasma cells in bone marrow], [Very Good Partial Response (VGPR)=Serum and urine M-protein detectable by immunofixation but not on electrophoresis or 90% or greater reduction in serum M-protein + urine M-protein level <100 mg/24 hour], [Partial Response (PR)=≥50% reduction of serum M-protein and reduction in 24 hour urine M-protein by ≥90% or to <200 mg per 24 hour], [Stable Disease (SD)=Not meeting criteria for CR, VGPR, PR or progressive disease] or [Progressive Disease (PD)]. (NCT01009840)
Timeframe: 6 Months

InterventionPercentage of participants (Number)
sCR or CR or VGPRPR or SD or PDNot Assessed
IV Busulfan20.070.010.0

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Ratio Area Under Curve (AUC)/Target AUC

A test dose of IV busulfan 0.8 mg/kg was infused at Baseline (Day -12 to -9) to verify a target busulfan integrated AUC of 20,000 μM*min with a range of 16,000 to 24,000. If necessary the dose could be adjusted. The PK-directed dose recommendation based on the test dose was administered at Day -5. Blood samples for PK analysis were collected at 0, 15, 30 minutes after the End of Infusion and 240, 300, 360 minutes after start of the infusion. Gas chromatography with mass selective detection (GC-MS) was used to determine the busulfan level in plasma. The ratio was calculated: AUC/Target AUC. (NCT01009840)
Timeframe: Baseline (Day -12 to -9), Day -5

InterventionRatio (Median)
IV Busulfan1.00

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Progression-free Survival

Progression-free Survival (PFS) defined as the time from transplantation to the occurrence of the event that was death or first recurrence of progressive disease (PD) by IMWG criteria. PD was defined as an Increase of ≥25% from the lowest response value in any one or more of the following: 1)Serum M-component and/or (the absolute increase must be ≥0.5 g/dL), 2)Urine M-component and/or (the absolute increase must be ≥200 mg/24 hr), 3)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, 4)Bone marrow plasma cell percentage; the absolute percentage must be ≥10%, 5)Definite development of new bone lesions or soft tissue plasmacytomas or definite increase in the size of existing bone lesions or soft tissue plasmacytomas and/or 6)Development of hypercalcaemia that can be attributed solely to the plasma cell proliferative diso (NCT01009840)
Timeframe: 6 Months

InterventionDays (Median)
IV Busulfan191

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Overall Survival

Overall Survival was defined as the time in days from transplantation to death due to all causes. (NCT01009840)
Timeframe: 6 Months

InterventionDays (Median)
IV BusulfanNA

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Percentage of Participants With Progression-free Survival Events

Progression-free survival events are death or first recurrence of progressive disease by International Myeloma Working Group Criteria. (NCT01009840)
Timeframe: 6 Months

InterventionPercentage of participants (Number)
IV Busulfan50.0

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Percentage of Participants With Hepatic Veno-Occlusive Disease Based on Baltimore Criteria

The Baltimore criteria for veno-occlusive disease was defined as the development of hyperbilirubinemia with serum bilirubin > 2 mg/dl within 21 days after transplantation and at least 2 of the following clinical signs and symptoms: (1) hepatomegaly, usually painful, (2) > 5% weight gain, or (3) ascites. (NCT01009840)
Timeframe: 6 Months

InterventionPercentage of participants (Number)
IV Busulfan0.0

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Percent Difference Between Area Under Curve (AUC) and Target AUC

A test dose of IV busulfan 0.8 mg/kg was infused at Baseline (Day -12 to -9) to verify a target busulfan integrated AUC of 20,000 μM*min with a range of 16,000 to 24,000. If necessary the dose could be adjusted. The PK-directed dose recommendation based on the test dose was administered at Day -5. Blood samples for PK analysis were collected at 0, 15, 30 minutes after the End of Infusion and 240, 300, 360 minutes after start of the infusion. GC-MS was used to determine the busulfan level in plasma. The percent difference was calculated between AUC and the Target AUC. (NCT01009840)
Timeframe: Baseline (Day -12 to -9), Day -5

InterventionPercent difference (Median)
IV Busulfan-0.22

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Percent Change in IV Busulfan Dose

The percent change in dose is relative to the busulfan dose administered at the Baseline Visit (Day -12 to -9) when a dose of 0.8 mg/kg was administered and on Day -5 when the Seattle Cancer Care Alliance recommended PK-adjusted dose was administered. (NCT01009840)
Timeframe: Baseline (Day -12 to -9), Day -5

InterventionPercent change (Median)
IV Busulfan7.55

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Percentage of Participants With Overall Survival Events

Overall Survival Event was death. (NCT01009840)
Timeframe: 6 Months

InterventionPercentage of participants (Number)
IV Busulfan13.3

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2-year Progression-free Survival in Early Disease Participants

"Percentage of participants who were alive and progression free at 2 years for participants with early disease stage. The 2 year progression free survival, with 95% confidence interval, was estimated using the Kaplan Meier method.~A progression is defined as one of the following events:~>= 50% increase in the products of at least two lymph nodes on two consecutive determinations two weeks apart (at least one lymph node must be >= 2 cm); appearance of new palpable lymph nodes.~>= 50% increase in the size of the liver and/or spleen as determined by measurement below the respective costal margin; appearance of palpable hepatomegaly or splenomegaly, which was not previously present.~> 50% increase in peripheral blood lymphocytes with an absolute increase > 5000/μL.~Transformation to a more aggressive histology (i.e., Richter's syndrome or prolymphocytic leukemia with >= 56% prolymphocytes)." (NCT01027000)
Timeframe: 2 years post-registration

Interventionpercentage of participants (Median)
Treatment (Combination of Chemotherapy and Transplant)79.5

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Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 100

Interventionpercentage of donor cells (Mean)
Transplant Patients90

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 28

Interventionpercentage of donor cells (Mean)
Transplant Patients95

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 42

Interventionpercentage of donor cells (Mean)
Transplant Patients93

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: One year

Interventionpercentage of donor cells (Mean)
Transplant Patients99

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Number of Patients Who Died Peri-Transplant

Peri-transplant is defined as within 100 days of transplant. (NCT01043640)
Timeframe: By Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Donor Cell Chimerism Following Transplant

Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: 6 months

Interventionpercentage of donor cells (Mean)
Transplant Patients94

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Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients5

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Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients5

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Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients32

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Number of Patients With Donor Derived Engraftment

Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells. (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients42

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Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)

"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant

InterventionParticipants (Count of Participants)
Transplant Patients2

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Number of Participants With Grades II-IV Acute GVHD

Determined with death as a competing risk. Defined and staged using the 1994 consensus conference modifications of the Glucksberg criteria. (NCT01044745)
Timeframe: At day 100

InterventionParticipants (Count of Participants)
Treatment (Rituximab and Allogeneic HCT Transplant)16

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Event-free Survival

Estimated using Kaplan-Meier estimator. (NCT01044745)
Timeframe: From the date of transplant with relapse/progression or death as censored events, up to 2 years

Interventionmonths (Median)
Treatment (Rituximab and Allogeneic HCT Transplant)12

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Overall Survival

Estimated using Kaplan-Meier estimator. (NCT01044745)
Timeframe: From the date of transplant with death from any cause as a censored event, up to 2 years

Interventionmonths (Median)
Treatment (Rituximab and Allogeneic HCT Transplant)12

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Overall Survival

Number of participants that were alive. (NCT01093586)
Timeframe: At 2 years

InterventionParticipants (Count of Participants)
Arm I4

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Recurrence or Relapse

Number of subjects that had disease recurrence (NCT01093586)
Timeframe: one year in patients post UCBT

InterventionParticipants (Count of Participants)
Arm I2

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Recurrence or Relapse

Number of subjects that had disease recurrence (NCT01093586)
Timeframe: two years post transplant

InterventionParticipants (Count of Participants)
Arm I2

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

Number of participants that had acute GVHD (NCT01093586)
Timeframe: At 100 days

InterventionParticipants (Count of Participants)
Arm I11

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Hematologic Engraftment

Number of participants that were able to complete engraftment by day 42. (NCT01093586)
Timeframe: On day +42

InterventionParticipants (Count of Participants)
Arm I10

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Overall Survival

Number of participants that were alive. (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I6

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Disease Free

Number of participants that were disease free (NCT01093586)
Timeframe: At 2 years

InterventionParticipants (Count of Participants)
Arm I4

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Disease Free

Number of participants that were disease free (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I4

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Incidence of Chronic GVHD

Number of participants that have chronic GVHD. Chronic GVHD will be diagnosed and graded on clinical and histological criteria from the Center for International Blood and Marrow Transplant Research (CIBMTR) (NCT01093586)
Timeframe: At 1 year

InterventionParticipants (Count of Participants)
Arm I1

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Occurrence of Serious Infections

Number of participants that had infections (NCT01093586)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Arm I13

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Overall Survival

Number of participants alive at 180 days post engraftment. (NCT01093586)
Timeframe: On day +180

InterventionParticipants (Count of Participants)
Arm I8

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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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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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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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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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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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Graft Failure

Percentage of participants who failed to engraft. (NCT01135329)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Transplant8

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100-day Mortality

The number of death reported within the first 100 days after transplant. (NCT01168219)
Timeframe: Up to 100 days post-treatment

InterventionParticipants (Count of Participants)
Treatment (Chemotherapy and Transplant)10

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Overall Survival (OS)

Overall survival rate (percentage) at 2 and 5 years is defined as the percentage of patients who are still alive 2 and 5 years after date of transplantation respectively. Estimated using the Kaplan-Meier product limit estimator. (NCT01168219)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
OS at 2 yearsOS at 5 years
Treatment (Chemotherapy and Transplant)45.731.2

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Progression-free Survival

"Progression-free survival rate (percentage) at 2 and 5 years is defined as the percentage of patients who are alive and progression free at 2 and 5 years from date of transplantation respectively.~AML progression is defined as:~Reappearance of leukemia blast cells in peripheral blood and > 5% blasts in marrow~If no circulating blasts, but the marrow contains 5-20% blasts, a repeat bone marrow >= 1 week later with > 5% blasts~Development of extramedullary leukemia MDS progression is defined as~For patients with <5% bone marrow blasts: ≥50% increase in blasts to >5% blasts~For patients with 5-10% bone marrow blasts: ≥50% increase to >10% blasts~Any of the following: Reappearance of prior documented characteristic cytogenetic abnormality or refractory cytopenias with unequivocal evidence of dysplasia on bone marrow biopsy/aspirate" (NCT01168219)
Timeframe: Up to 5 years

Interventionpercentage of patients (Number)
PFS at 2 yearsPFS at 5 years
Treatment (Chemotherapy and Transplant)41.226.9

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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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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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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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Estimated Two Year Overall Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants

(NCT01181271)
Timeframe: Two-years after Allogeneic Transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant89

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Estimated Two Year Overall Survival Rate for All Participants

(NCT01181271)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant83

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Cumulative Incidence of Grades II to IV Acute Graft Versus Host Disease (GVHD)

Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01181271)
Timeframe: within 200 days after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant13.8

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Cumulative Incidence of Extensive Chronic Graft-versus-host-disease

Extensive chronic graft versus-host-disease (GVHD) was defined as GVHD that required systemic immunosuppression. (NCT01181271)
Timeframe: 1-year after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant37.9

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Cumulative Incidence of Disease Relapse

(NCT01181271)
Timeframe: 2-years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant17.2

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Peripheral Blood All-cell Donor Chimerism

Successful donor stem cell engraftment is defined as when ≥ 80% of hematopoietic elements are donor-derived as determined by chimerism assays from peripheral blood at day 100 after non-myeloablative allogeneic stem cell transplantation. (NCT01181271)
Timeframe: 100 days post allogeneic transplant

Interventionpercentage of donor-derived elements (Median)
Autologous Then Allogeneic Transplant95

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Number of Days After Allogeneic Transplant Until Absolute Neutrophil Count Was Equal to or Greater Than 500/uL

(NCT01181271)
Timeframe: within 28 days after allogeneic transplant

Interventiondays (Median)
Autologous Then Allogeneic Transplant12

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Estimated Two Year Progression Free Survival Rate for Participants Undergoing Only Autologous Transplant

(NCT01181271)
Timeframe: Two Years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant46

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Estimated Two Year Overall Survival Rate for Participants Undergoing Only Autologous Transplant

(NCT01181271)
Timeframe: two years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant69

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Estimated Two Year Progression Free Survival Rate for All Participants

(NCT01181271)
Timeframe: 2 years

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant64

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Cumulative Incidence of Non-relapse Mortality

Non-relapse mortality is defined as participants who die from causes other than their underlying disease relapse, such as infection or graft versus host disease (NCT01181271)
Timeframe: 2-years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant11.1

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Estimated Two Year Progression Free Survival Rate for Participants Undergoing Both Autologous and Allogeneic Transplants

(NCT01181271)
Timeframe: 2 years after allogeneic transplant

Interventionpercentage of participants (Number)
Autologous Then Allogeneic Transplant72

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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 (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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Non-Relapse Mortality (NRM) Following RTC Transplantation at 1 Year.

Percentage of participants NRM following RTC transplantation at 1-year. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant17.1

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Non-Relapse Mortality (NRM) Following RTC Transplantation at 2 Years.

Percentage of participants NRM following RTC transplantation at 2-year. (NCT01203020)
Timeframe: At 2 years

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant24.6

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Percentage of 1 Year Overall Survival (OS)

Percentage of participants--1 year overall survival (OS) following transplantation. (NCT01203020)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant63.6

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Rates of Acute and Chronic Graft Versus Host Disease (GVHD).

Count/Percentage rates of acute and chronic graft versus host disease (GVHD). (NCT01203020)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Acute GVHDChronic GVHD
Allogeneic Hematopoietic Progenitor Cell Transplant53

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Relapse Rate (RR) Following Transplantation at 2-year.

Percentage of participants Relapse Rates (RR) following transplantation at 2-year. (NCT01203020)
Timeframe: At 2 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant39.7

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Relapse Rate (RR) Following Transplantation at 1-year.

Percentage of participants Relapse Rates (RR) following transplantation at 1-year. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant28.7

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Percentage of 2-year Progression Free Survival (PFS)

Percentage of 2-year progression free survival (PFS) of patients with chemotherapy refractory Hodgkin's and non-Hodgkin's lymphoma (NHL) undergoing reduced-toxicity conditioning (RTC) with once daily intravenous Busulfex and fludarabine. (NCT01203020)
Timeframe: At 2 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant45.5

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Percentage of 2 Year Overall Survival (OS)

Percentage of participants-- 2 Year Overall Survival (OS) post transplant (NCT01203020)
Timeframe: At 2nd year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant59.1

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Percentage of 1-year Progression Free Survival (PFS)

Percentage of 1-year progression free survival (PFS) of patients with chemotherapy refractory Hodgkin's and non-Hodgkin's lymphoma (NHL) undergoing reduced-toxicity conditioning (RTC) with once daily intravenous Busulfex and fludarabine. (NCT01203020)
Timeframe: At 1 year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Progenitor Cell Transplant59.1

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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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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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Number of Participants With Platelet Engraftment

Achievement of untransfused platelet count > 20 x 10^9/L on three consecutive days (NCT01247701)
Timeframe: Day 180

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant13

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Number of Participants With Neutrophil Engraftment

Achievement of absolute neutrophil count > 0.5 x 10^9/L on three consecutive days (NCT01247701)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant15

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Number of Participants With Relapse Rate After Transplant

To assess relapse rate at 1 and 3 years after transplant. Cumulative incidence of relapse was calculated from the date of umbilical cord blood transplant using the competing risk method as described in Gray(1988) with death prior to relapse as the competing risk. Participants still alive without a date of relapse were censored at the time of the last follow-up. (NCT01247701)
Timeframe: 1 and 3 years

Interventionpercentage of participants (Number)
1 year3 year
Umbilical Cord Blood Transplant53.353.3

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Number of Participants With Chronic GvHD

Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3. (NCT01247701)
Timeframe: 1 year

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant1

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Overall Survival at 100 Days, 1 Year, and 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.

To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies. (NCT01247701)
Timeframe: 100 days, 1 year, and 3 years

Interventionprobability of overall survival (Number)
100 days1-Year3-Year
Umbilical Cord Blood Transplant0.9230.9230.923

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Number of Participants With Donor Engraftment After Transplant.

To evaluate donor engraftment at 100 days, 6, 9, 12, 24, and 36 months after transplant. (NCT01247701)
Timeframe: 100 days, 6, 9, 12, 24 and 36 months

InterventionParticipants (Count of Participants)
100 days6 months9 months12 months24 months36 months
Umbilical Cord Blood Transplant1166663

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Number of Participants With Severe Acute GVHD Grade III-IV

Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD. (NCT01247701)
Timeframe: Day 100

InterventionParticipants (Count of Participants)
Umbilical Cord Blood Transplant2

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Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0)

Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. (NCT01338987)
Timeframe: Date treatment consent signed to date off study, approximately 79 months and 11 days.

InterventionParticipants (Count of Participants)
Males That Did Not Receive Leuprolide for 1st Transplant10
Males Randomized to Receive Leuprolide for 1st Transplant8
Females That Received Leuprolide for 1st Transplant20
Matched Related Donors for Transplant4
Recipients of 2nd Transplant2

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Percentage of B Cells at One Year Post-transplant in Participants Who Did/Did Not Receive Leuprolide Following Bone Marrow Transplant (BMT)

B cell percentage is defined as the percentage of lymphocytes that are B cells. (NCT01338987)
Timeframe: after first Bone Marrow Transplant, approximately 12 months post-transplant

Interventionpercentage of cells (Median)
Males that received leuprolideMales that did not receive leuprolideFemales who all received leuprolide
Transplant Recipient22.121.914.3

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Time to Engraftment in First Transplant Recipients Only With Median Thoracic Spine Standardized Uptake Values (SUV) of 1.4 or Greater Than Those Patients With SUV's Less Than 1.4

18F-FLT imaging was performed serially on patients post transplant to identify the level of uptake of 18F-FLT at a day +5 to +12 scan and the day at which neutrophils recover to >500 (i.e., subclinical bone-marrow recovery within 5 days of Bone Marrow Transplantation (BMT infusion)). On each image for each patient, the region of interest was drawn within each thoracic medullary space (n=12), generating the SUV for each space. The mean of these was calculated for each scan. The analysis was the median of the means of the SUV of the thorax values of the day 5-12 scan (averaged the SUV of the thorax for each patient and then took the medians of these). (NCT01338987)
Timeframe: 18F FLT scan done between days +5 to +12 and then time from that scan to engraftment measured

InterventionDays (Median)
Patients with SUV 1.4 or greaterPatients with SUV less than 1.4
Transplant Recipient515

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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 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 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 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 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 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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To Compare the Relapse Rate at 1 Year of Patients With Myeloid Malignancies Receiving Each Treatment

(NCT01366612)
Timeframe: 1 year

InterventionPercent (Number)
Group 138.9
Group 218.8

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Infection Rate

The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. (NCT01380990)
Timeframe: 36 months

InterventionInfection rate per person per year (Number)
OTL-101* On-Study Subjects0.14
OTL-101* On-Study and CUP Subjects0.14
HSCT Controls Without MRD0.13
HSCT Controls With MRD0.17
All HSCT Controls0.16

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ADA Activity in Erythrocytes

ADA enzyme activity was assessed as a measure of successful engraftment of genetically modified Hematopoietic stem progenitor cells (HSPCs), as it marks sustained gene expression from the normal ADA transgene. (NCT01380990)
Timeframe: 36 months

Interventionnmol/h/mg (Median)
OTL-101* On-Study Subjects638.0
OTL-101* On-Study and CUP Subjects490.5
HSCT Controls Without MRD86.5
HSCT Controls With MRD1.0
All HSCT Controls23.0

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VCN in Peripheral Blood Mononuclear Cells (PBMCs)

Engraftment of transduced cells was assessed using vector gene marking in PBMCs (NCT01380990)
Timeframe: 36 months

Interventioncopies/cell (Median)
OTL-101* On-Study Subjects0.600
OTL-101* On-Study and CUP Subjects0.625

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OS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)

Overall survival (OS) is defined as the percentage of subjects alive at 36 months post- treatment with OTL-101* or HSCT (NCT01380990)
Timeframe: 36 months

Interventionpercentage of participants (Number)
OTL-101* On-Study Subjects100
OTL-101* On-Study and CUP Subjects100
HSCT Controls Without MRD100
HSCT Controls With MRD88.89
All HSCT Controls92.86

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Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101* or HSCT (NCT01380990)
Timeframe: 12 months

Interventionpercentage of participants (Number)
OTL-101* On-Study Subjects100
OTL-101* On-Study and CUP Subjects100
HSCT Controls Without MRD100
HSCT Controls With MRD100
All HSCT Controls100

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Reduction in Deoxyadenosine Triphosphate (dATP) in Erythrocytes

Decreased dATP levels coincide with increased ADA enzyme activity, detoxification was used as a marker of correction of the defective ADA gene. The threshold for detoxification was <100 μmol/L. (NCT01380990)
Timeframe: 36 months

Interventionumol/L (Median)
OTL-101* On-Study Subjects50.0
OTL-101* On-Study and CUP Subjects50.0
HSCT Controls Without MRD0.0
HSCT Controls With MRD114.0
All HSCT Controls90.0

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VCN in CD19+ B Cells

Engraftment of transduced cells was assessed using vector gene marking in CD19+ B Cells (NCT01380990)
Timeframe: 36 months

Interventioncopies/cell (Median)
OTL-101* On-Study Subjects0.880
OTL-101* On-Study and CUP Subjects1.190

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VCN in CD3+ T Cells

Engraftment of transduced cells was assessed using vector gene marking (NCT01380990)
Timeframe: 36 months

Interventioncopies/cell (Median)
OTL-101* On-Study Subjects1.065
OTL-101* On-Study and CUP Subjects1.160

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Frequency of Vector Integration Into Known Protooncogenes (3 Years)

"Vector Integration Site Analysis (VISA) allowed determination of the distribution of vector integration sites in each subject's genome, as well as the relative clonal abundance. VISA was to be considered abnormal for a subject if, in 2 or more instances during the course of follow-up, a single integration site was found to represent >30% of the total integration sites detected.~There were no instances of clonal proliferation in the course of the 36 month follow-up for on-study and CUP subjects; hence, a detailed analysis of the frequency of clonal expansion associated with vector integration near proto-oncogenes was not generated." (NCT01380990)
Timeframe: 36 months

Intervention% of integration in known protooncogenes (Number)
OTL-101* On-Study Subjects0
OTL-101* On-Study and CUP Subjects0

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Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT01380990)
Timeframe: 12 months

Interventionpercentage of participants (Number)
OTL-101* On-Study Subjects100
OTL-101* On-Study and CUP Subjects100
HSCT Controls Without MRD100
HSCT Controls With MRD100
All HSCT Controls100

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Vector Copy Number (VCN) in Granulocytes Fraction (Neutrophils)

Engraftment of transduced cells was assessed using vector gene marking in granulocytes (neutrophils) (NCT01380990)
Timeframe: 36 months

Interventioncopies/cell (Median)
OTL-101* On-Study Subjects0.240
OTL-101* On-Study and CUP Subjects0.280

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Change From Baseline in CD3+ T Cell Counts (3 Years)

Immune reconstitution was assessed by change in CD3+ T Cell counts over time. (NCT01380990)
Timeframe: 36 months

Intervention10e9 cells/L (Median)
OTL-101* On-Study Subjects1.060
OTL-101* On-Study and CUP Subjects1.090

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Change From Baseline in CD3+ T Cell Counts (1 Year)

Immune reconstitution was assessed by change in CD3+ T Cell counts over time. (NCT01380990)
Timeframe: 12 months

Intervention10e9 cells/L (Geometric Mean)
OTL-101* On-Study Subjects3.58
OTL-101* On-Study and CUP Subjects3.18

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EvFS of Subjects Treated With IMP With Those of Patients Treated With Allogeneic HSCT (3 Years)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT01380990)
Timeframe: 36 months

Interventionpercentage of participants (Number)
OTL-101* On-Study Subjects90.00
OTL-101* On-Study and CUP Subjects95.00
HSCT Controls Without MRD80.00
HSCT Controls With MRD60.00
All HSCT Controls66.67

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Number of Participants With Molecular Complete Remission at 3 Month Post Transplant

Molecular Complete Remission is defined as participant alive and engrafted with molecular complete remission 100 days post transplant where molecular complete response is no BCR-ABL transcripts detected and engraftment is defined as the evidence of donor derived cells (more than 95%) by chimerism studies in the presence of neutrophil recovery by day 28 post stem cell infusion. (NCT01390402)
Timeframe: Baseline to up to 4 months post-transplant

Interventionparticipants (Number)
NK Infusion + Chemotherapy3

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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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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 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 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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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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Non-relapse Mortality

Defined as death in the absence of recurrent or progressive malignancy after HCT. Non-relapse morality will be assessed with the use of cumulative incidence plots. This secondary endpoint will be characterized and presented as a cumulative incidence. (NCT01427881)
Timeframe: At 2 years

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)14

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Donor Engraftment

Donor engraftment is defined as the count (percent) of patients with full donor chimerism. Full donor chimerism is defined as at least 95% donor CD3 cells in peripheral blood. (NCT01427881)
Timeframe: At day 28

InterventionParticipants (Count of Participants)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)6

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Chronic GVHD Requiring Systemic Immunosuppressive Treatment

Chronic GVHD will be defined by National Institutes of Health (NIH) criteria and requiring systemic treatment. A reduction in the cumulative incidence of GVHD from ~35% to ~15% at 1 year would represent a reasonable goal. A sample size of 42 patients provides 90% power to observe such a difference with one-side 5% type-1 error. (NCT01427881)
Timeframe: At 1 year after transplantation

Interventionpercent of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)16

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Hematologic Recovery

Descriptive statistics will be used to assess the median days of neutrophil and platelet recovery. The day of neutrophil recovery is defined as the first day of three consecutive lab values on different days, after the conditioning regimen-induced nadir of blood counts, that the absolute neutrophil count is > 500/uL. The day of platelet recovery is defined as the first day of three consecutive lab values on different days, after the conditioning regimen-induced nadir of blood counts, that the platelet count is >= 20,000/uL without platelet transfusion support in the seven days prior. (NCT01427881)
Timeframe: Up to day +100

Interventiondays (Median)
Neutrophil EngraftmentPlatelet Engraftment
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)1914

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Graft Failure

Descriptive statistics will be used to assess the incidence of primary graft failure and secondary graft failure. Primary graft failure is defined as failure to achieve a sustained neutrophil count of >= 500/uL by >= 28 days post-transplant. Secondary graft failure is defined as the decline in neutrophil count to < 500/uL after achieving engraftment which is unrelated to infection or drug effect and is unresponsive to stimulation by growth factors. (NCT01427881)
Timeframe: By greater than or equal to 28 days post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)2

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Disease-free Survival

Disease-free survival will be evaluated as Kaplan-Meier estimates. (NCT01427881)
Timeframe: At 1 year post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)73.8

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Persistent or Recurrent Malignancy After HCT

Recurrent or progressive malignancy will be assessed with the use of cumulative incidence plots. Recurrent malignancy will be defined by hematologic criteria. Recurrent malignancy will also be defined as any unplanned medical intervention designed to prevent progression of malignant disease in patients who have molecular, cytogenetic or flow-cytometric evidence of malignant cells after transplantation. (NCT01427881)
Timeframe: At 2 years

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)17

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Grades II-IV and III-IV Acute GVHD

Grades II-IV and III-IV GVHD will be assessed with the use of cumulative incidence plots. (NCT01427881)
Timeframe: Through day +100 post-transplant

Interventionpercentage of patients (Number)
Grades II-IV GVHDGrades III-IV GVHD
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)770

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Overall Survival

Overall survival will be evaluated as Kaplan-Meier estimates. (NCT01427881)
Timeframe: At 1 year post-transplant

Interventionpercentage of patients (Number)
Treatment (TBI, PBSCT, and Cyclophosphamide GVHD Prophylaxis)75.6

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Incidence of Relapse

(NCT01457885)
Timeframe: 2 years

Interventionpercentage of patients (Number)
CloBu4 Regimen55

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Cumulative Incidence of Non Relapse Mortality (NRM)

Percentage of patients passed without relapse/recurrence at 1 year. (NCT01457885)
Timeframe: 1 year

Interventionpercentage of patients (Number)
CloBu4 Regimen21

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The Percentage of Patients Alive at 1 Year

Overall survival was calculated following transplant using a CloBu4 conditioning regimen for patients with non-remission AML (NCT01457885)
Timeframe: 1 year

Interventionpercentage of patients (Number)
CloBu4 Regimen32

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Duration of Survival

(NCT01464359)
Timeframe: 2 years after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Duration of Survival

(NCT01464359)
Timeframe: 1 year after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Incidence of Graft Failure

Incidence of graft failure defined as an absolute neutrophil count of less than 500/uL and a bone marrow that is less than 5% cellular (marrow aplasia) (NCT01464359)
Timeframe: Day 42

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Disease Free Survival

The primary endpoint is a disease free survival at 3 months in patients with chemotherapy refractory AML after a double T-cell depleted (TCD) umbilical cord blood (UCB) transplantation where one TCD unit is activated overnight in IL-2 followed by the administration of two courses of IL-2 three times a week for 6 doses beginning on day +3 and on day +60 to expand UCB-derived NK cells in vivo. (NCT01464359)
Timeframe: At 3 months

Interventionparticipants (Number)
Patients With Acute Myelogenous Leukemia1

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Clinical Disease Response

Defined as leukemia clearance and complete remission. Patients will be followed for disease response for 2 years from transplantation unless: consent is withdrawal, patient is unevaluable - if a patient is not evaluable, follow only untilthe resolution or stabilization of treatment related toxicity, new anti-cancer treatment is started, patient is discharged to hospice (terminal) care. (NCT01464359)
Timeframe: 2 Years from Transplantation

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia2

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Duration of Survival

(NCT01464359)
Timeframe: 6 months after Transplantation.

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia1

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Clinical Disease Response

Defined as leukemia clearance and complete remission. Patients will be followed for disease response for 1 year from transplantation unless: consent is withdrawal, patient is unevaluable - if a patient is not evaluable, follow only until the resolution or stabilization of treatment related toxicity, new anti-cancer treatment is started, patient is discharged to hospice (terminal) care. (NCT01464359)
Timeframe: 1 Year from Transplantation

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia2

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Incidence of Acute Graft-Versus-Host Disease

(NCT01464359)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Patients With Acute Myelogenous Leukemia0

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Overall Survival (OS) Post Transplant at 1, 3 and 5 Years

Number of participants in the study who are alive and disease free at 1, 3 and 5 years post transplant. (NCT01471444)
Timeframe: Post transplant after 1, 3 and 5 years

,
InterventionParticipants (Count of Participants)
1 Year Post Transplant3 Year Post Transplant5 Year Post Transplant
Arm A (Flu+Bu)836964
Arm B (Flu+Clo+Bu)826963

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Progression-Free Survival (PFS)

Number of events with progression free survival. (Progression is defined as more than 5% blast in the peripheral blood or bone marrow biopsy.) or expired from treatment related mortality post transplant. (NCT01471444)
Timeframe: From day of transplant to disease of progression or death of any cause, whichever came first, assessed up to 5 years

InterventionNumber of events (Number)
Arm A (Flu+Bu)69
Arm B (Flu+Clo+Bu)61

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Number of Participants With Non Relapse Mortality at 100 Day Post Transplant

Number of participants expired from complications other than relapsed disease at 100 day Post Transplant. (NCT01471444)
Timeframe: 100 day Post Transplant

InterventionParticipants (Count of Participants)
Arm A (Flu+Bu)3
Arm B (Flu+Clo+Bu)6

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Number of Participants in the Study Who Are With no Grade 3 or 4 Acute Graft-versus-host Disease at Any Time During the First 100 Days Post Transplant.

Number of participants in the study who are with no Grade 3 or 4 acute graft-versus-host disease at any time during the first 100 days post transplant. (NCT01471444)
Timeframe: 100 days post transplant

InterventionParticipants (Count of Participants)
Arm A (Flu+Bu)125
Arm B (Flu+Clo+Bu)115

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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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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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Event -Free Survival Rate

Event-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Bone Marrow Transplant Recipients19

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Chronic Graft Versus Host Disease (GVHD)

Chronic GVHD was graded according to the National Institutes of Health (NIH) 2014 Consensus Criteria Diagnosis and scoring the severity of chronic GVHD is determined by evaluating symptoms of the skin, nails, hair, mouth, eyes, genitalia, gastrointestinal tract, liver, lungs, muscles, fascia and joints, immune function as well as other symptoms such as ascites and neuropathy. Chronic GVHD is graded as mild, moderate or severe based on the number of organ sites impacted and the severity of symptoms. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Mild Chronic GVHDModerate Chronic GVHDSevere Chronic GVHD
Bone Marrow Transplant Recipients321

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Graft Failure

Primary graft failure occurs when a transplant recipient does not achieve donor chimerism following a bone marrow transplant. Secondary graft failure occurs when graft fails after donor chimerism had initially occurred. (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Primary graft failureSecondary graft failure
Bone Marrow Transplant Recipients01

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Overall Survival

Overall survival is defined as survival with or without sickle cell disease after hematopoietic cell transplantation (HCT). (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Bone Marrow Transplant Recipients20

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Acute Graft Versus Host Disease (GVHD)

"Acute GVHD was graded according to the Center for International Blood and Marrow Transplant Research (CIBMTR) consensus criteria. Clinical manifestations of acute GVHD include skin, liver, and gastrointestinal symptoms. Grading of acute GVHD is determined by size of maculopapular rash, bilirubin and stool output. Acute GVHD grades range from 0 to 4 with 0 indicating no GVHD and 4 representing the most severe grade.~Grade II is defined as a maculopapular rash over 25-50% of body surface area (BSA), bilirubin of 3.1 to 6 mg/dL, and stool output of 1000-1500 mL/d (for adults).~Grade III is defined as a maculopapular rash over more than 50% of BSA, bilirubin of 6.1 to 15 mg/dL, and stool output of greater than 1500 mL/d (for adults).~Grade IV is defined as generalized erythroderma with bullous formation, bilirubin greater than 15 mg/dL, and severe abdominal pain with or without ileus." (NCT01565616)
Timeframe: 1 year after transplant

InterventionParticipants (Count of Participants)
Grade II Acute GVHDGrade III Acute GVHDGrade IV Acute GVHD
Bone Marrow Transplant Recipients310

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Time to Neutrophil and Platelet Engraftment

Time to neutrophil engraftment is defined as the first of 3 measurements on different days when the patient has an absolute neutrophil count of at least 500/µL after conditioning. Time to Platelet engraftment is defined as the first day of a minimum of 3 measurements on different days that the patient has achieved a platelet count > 50,000/µL, without receiving a platelet transfusion in the previous 7 days. (NCT01565616)
Timeframe: 1 year after transplant

InterventionDays (Median)
Neutrophil engraftmentPlatelet
Bone Marrow Transplant Recipients1721

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Overall Survival

Number of participants that were diseased free and alive 3 years post-transplant. (NCT01572662)
Timeframe: Up to 3 years post-transplant

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l16
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.47

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Non-Relapse Mortality Rate (NRM)

Number of participants expired within the first 100 days after transplant not due to relapsed disease. (NCT01572662)
Timeframe: 100 days

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l2
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.8

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Overall Survival

Number of participants that are disease free and alive one year post transplant. (NCT01572662)
Timeframe: Up to 1 year post-transplant

InterventionParticipants (Count of Participants)
Arm 1: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 16,000 Umol/l29
Arm 2: Participants w/Hematologic Malignancies Treated w/Fludarabine/TS Busulfan AUC 20,000umol/l.93

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Mixed-donor Chimerism Rate of Patients With High-risk Myeloid Malignancies (Stratum B)

Full-donor chimerism will be defined by as ≥99% donor cells by Short Tandem Repeat (STR) analysis in all cell lines (CD3, CD14/15, and CD19) in peripheral blood. The historic control for Stratum B was determined using the 20 patients who were transplanted from 2005 - 2010 with Busulfan (BU)-based regimens and who retrospectively would have been eligible for the current trial. Of these 20 patients, at 100 days post-HCT, only 8 (40%) patients had full-donor chimerism. If 5 patients in Stratum B experienced mixed-donor chimerism at Day 100, we will close this stratum early for failing to achieve superior donor cell engraftment compared to standard-of-care. (NCT01596699)
Timeframe: Participants will have peripheral blood chimerism assessed at Day 100 post hematopoietic stem cell transplant and then monthly until stable.

Interventionpercentage of participants (Number)
Stratum B: Patients With Myeloid Malignancies66.67

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Engraftment Rate of Patients With Non-malignant Diseases (Stratum A)

Engraftment will be defined as the development of an Absolute Neutrophil Count (ANC) >500 for 3 consecutive days plus donor CD14/15 cells >70%. The engraftment rate in the population used for historical control is 40%. If 3 patients in Stratum A experience graft rejection, this stratum will close early for failing to achieve superior engraftment compared to standard-of-care. (NCT01596699)
Timeframe: Participants will have engraftment blood studies starting approximately Day 30 post hematopoietic stem cell transplant and then monthly until stable. Average study participation is approximately 5 years.

Interventionpercentage of participants (Number)
Stratum A: Patients With Non-Malignancies66.67

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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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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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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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Mortality

(NCT01605032)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
Treatment (Busulfan, Melphalan, Bortezomib, Autologous PBSCT)1

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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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Disease-Free Survival (DFS)

Estimate the DFS at one-year post-transplantation. The event is defined as relapse or death due to relapse. The number of participants who did not relapse up to one year post transplant is reported. (NCT01621477)
Timeframe: one year post transplant

Interventionparticipants (Number)
Treatment7

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Event-Free Survival (EFS)

Estimate the EFS at one-year post-transplantation. The event is defined as relapse or death due to any cause. The number of participants who were alive without relapse at one year post-transplant is reported. (NCT01621477)
Timeframe: one year post transplant

Interventionparticipants (Number)
Treatment4

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Incidence of Malignant Relapse

Estimate the incidence of malignant relapse at one year post-transplant. The number of participants with malignant relapse or progressive disease is given. Relapse was evaluated using standard WHO criteria for each disease. (NCT01621477)
Timeframe: One year post transplantation.

Interventionparticipants (Number)
Treatment10

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One-year Survival (OS)

Evaluate the number of participants alive at 1 year. The number of participants surviving to one-year post-transplantation is given. (NCT01621477)
Timeframe: One year post transplant

Interventionparticipants (Number)
Treatment7

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Incidence and Severity of Acute Graft Versus Host Disease (GVHD)

The number of participants with acute GVHD is given, organized by grade. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe. (NCT01621477)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
No Acute GVHDGrade IGrade IIGrade IIIGrade IV
Treatment93131

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Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)

The severity of chronic GVHD will be described. Chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with chronic GVHD is given, organized by severity. (NCT01621477)
Timeframe: 100 days post transplant

Interventionparticipants (Number)
No Chronic GVHDMildModerateSevere
Treatment15020

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Time-to-engraftment

The number of days until participants by dose level reach engraftment. (NCT01629511)
Timeframe: 30 days post transplant

,,
Interventionparticipants (Number)
Day 10Day 11Day 12Day 13Day 15Day 17<10 days - Graft Failure
Gemcitabine Dose Level 11210000
Gemcitabine Dose Level 32401001
Gemcitabine Dose Level 40010110

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Overall Survival

Will be estimated by the method of Kaplan and Meier. Time-to-event distributions as function of patient baseline covariates will be evaluated using Bayesian time-to-event regression modeling. (NCT01629511)
Timeframe: Up to 1 year post transplant

InterventionParticipants (Count of Participants)
Gemcitabine Dose Level 13
Gemcitabine Dose Level 20
Gemcitabine Dose Level 34
Gemcitabine Dose Level 42

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Donor Stem Cell Engraftment: Platelet Count

Platelet recovery was defined as having a platelet count of at least 20,000 platelets/uL of blood on 2 consecutive measurements without transfusional support prior to day +100 after BuClo RIC HSCT. (NCT01643668)
Timeframe: 1, 2, 3, 4, 8, and 14 weeks post transplant

InterventionParticipants (Count of Participants)
Platelet Engraftmnet AchievedPlatelet Engraftmnet Not Achieved
Treatment Arm330

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Cumulative Incidence and Severity of Acute GVHD Within 100 Days Post Transplant

The percentage of participants who experienced grades 2-4 and grades 3-4 acute graft-versus-host disease (GVHD) by 100 days post transplantation. GVHD is a condition that can occur following an allogenic stem cell transplantation when the donated bone marrow or peripheral stem cells view the recipients body as foreign and the donated cell/marrow attack the body. Acute GVHD is generally observed within the first 100 days post transplant. Acute GVHD is associated with increased treatment related morbidity and mortality. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. The grade of the GVHD is determined by grading GHVD associated adverse events. Associated adverse events were graded using Common Terminology Criteria for Adverse Events (CTCAE) version 4 which uses the same mild, moderate, severe, life threatening grading system as the overall GHVD assessment. (NCT01643668)
Timeframe: 100 days

Interventionpercentage of Participants (Number)
Grades II-IV acute GVHDGrades III-IV acute GVHD
Treatment Arm2112

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Cumulative Incidence of Non-relapse Mortality

The percentage of participants that experienced non-relapse mortality (NRM) at day 100 and 1 year after BuClo RIC SCT. Non-relapse mortality is any mortality that is not associated with or proceeded by disease progression of prior cancers. (NCT01643668)
Timeframe: 100 days, 1 year

Interventionpercentage of participants who died (Number)
100 Days1 Year
Treatment Arm5.924

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Progression-Free and Overall Survival

The 1-year and 2-year progression-free and overall survival measured from the time of stem cell transplantation. Progression is the recurrence or increase in the number of cancer cells found in the body. (NCT01643668)
Timeframe: 1 year, 2 years

Interventionpercentage of participants (Number)
Progression Free Survival at 1 yearProgression Free Survival at 2 yearsOverall Survival at 1 yearOverall Survival at 2 years
Treatment Arm50505656

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Assessment of Donor Stem Cell Engraftment: ANC Count

Patients are considered to have achieved donor cell engraftment if they have an absolute neutrophil count (ANC) of at least 500 cells/uL of blood for 3 consecutive measurements and at least 75% of hematopoietic elements are donor-derived as determined by chimerism assays from peripheral blood prior to day +40 after Busulfan/Clofarabine (BuClo) reduced intensity allogeneic stem cell transplantation (NCT01643668)
Timeframe: 1, 2, 3, and 4 weeks after transplantation

InterventionParticipants (Count of Participants)
Neutrophil Engraftment AchievedNeutrophil Engraftment Not Achieved
Treatment Arm330

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Cumulative Incidence of Chronic GVHD at One Year

The percentage of participants who experienced chronic Graft Versus Host Disease (GVHD) by one year. GVHD is a condition that can occur following an allogenic stem cell transplantation when the donated bone marrow or peripheral stem cells view the recipients body as foreign and the donated cell/marrow attack the body. Chronic GVHD normally occurs after the first 100 days post transplantation. Chronic GVHD can adversely influence long term survival. (NCT01643668)
Timeframe: 1 year

Interventionpercentage of participants (Number)
Treatment Arm44

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Grade 3 or 4 Toxicities

The number of participants that experienced the specified grade 3 and 4 non-hematological toxicities during treatment and follow-up as assessed by Common Terminology Criteria for Adverse Events version 4(CTAE v 4.0). Grade 3 toxicity is considered to be severe and grade 4 is considered to be life threatening. (NCT01643668)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Bacterial InfectionViral InfectionFungal InfectionMucositisIncreased bilirubinIncreased alanine aminotransferaseIncreased aspartate aminotransferaseEngraftment SyndromeFebrile NeutropeniaSepsisAcute Renal FailureDiffuse alveolar hemorrhageIdiopathic pneumonia syndrome
Treatment Arm10121111021100

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Incidence of Hepatic Veno-occlusive Disease

The number of participants that experienced hepatic veno-occlusive disease (VOD). VOD is a condition in which some of the small veins in the liver are obstructed. (NCT01643668)
Timeframe: 2 years

InterventionParticipants (Count of Participants)
Treatment Arm0

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Count of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 7 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Counts of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant3

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Number of Participant Who Were Alive at 2 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 2 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant4

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Number of Participant Who Were Alive at 5 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 5 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant3

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Percentage of Participants With Relapse

The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 1 Year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant40

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Percentage of Participants With Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 1 Year

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Percentage of Participants With Engraftment Failure

Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets. (NCT01685411)
Timeframe: Day 42

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Count of Participants Who Achieved Neutrophil Engraftment

Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater. (NCT01685411)
Timeframe: By Day 42

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant5

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Percentage of Participants With Chronic Graft-Versus-Host Disease

Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 6 Months

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant0

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Percentage of Participants With Acute Graft-Versus-Host Disease by Grade

Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Grade 2-4Grade 3-4
Allogeneic Hematopoietic Stem Cell Transplant4020

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Percentage of Participants With Relapse

The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years

Interventionpercentage of participants (Number)
Allogeneic Hematopoietic Stem Cell Transplant40

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Number of Participant Who Were Alive at 7 Years Post Transplant

Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 7 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Count of Participants With Disease Free Survival

The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 5 Years

InterventionParticipants (Count of Participants)
Allogeneic Hematopoietic Stem Cell Transplant1

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Time to Neutrophil Recovery

Defined as achieving an absolute neutrophil count (ANC) greater than or equal to 500/ul for three consecutive measurements on different days. Will be summarized with mean and standard deviation or median and interquartile range, and the change will be tested using a one-sample paired t-test at a two-tailed significance level of 0.05. (NCT01707004)
Timeframe: Up to 1 year

Interventiondays (Mean)
Decitabine + Bone Marrow Transplant16

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Number of Participants With Primary Graft Failure

Defined as less than 5% donor chimerism in the cluster of differentiation (CD)3 and CD33 selected cell populations at any time after transplantation. Will be analyzed using KM method. (NCT01707004)
Timeframe: Day 30

InterventionParticipants (Count of Participants)
Decitabine + Bone Marrow Transplant0

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Overall Survival (OS)

Will be analyzed using Kaplan-Meier (KM) method, and OS will be obtained from the KM estimates along with 95% confidence intervals. (NCT01707004)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Decitabine + Bone Marrow Transplant64.7

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Percentage of Participants With Platelet Recovery by Day 30

Platelet recovery is defined as the first day of a platelet count greater than 20,000/mm^3 with no platelet transfusions. Will be summarized with mean and standard deviation or median and interquartile range, and the change will be tested using a one-sample paired t-test at a two-tailed significance level of 0.05. (NCT01707004)
Timeframe: Up to day 30

Interventionpercentage with plt engraftment, day 30 (Number)
Decitabine + Bone Marrow Transplant58

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Progression Free Survival

(NCT01707004)
Timeframe: Up to 1 year

Interventiondays (Median)
Decitabine + Bone Marrow Transplant141

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Cumulative Incidence of Chronic GVHD According to BMTCTN

Will be summarized with a proportion and a 95% confidence interval. (NCT01707004)
Timeframe: Up to 1 year

Interventionpercentage (Number)
Decitabine + Bone Marrow Transplant40.7

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Cumulative Incidence of Grade III-IV Acute GVHD

Determined by the standard bone marrow transplant (BMT) Clinical Trials Network criteria (BMTCTN). Will be analyzed using KM method, a Graft versus Host Disease (GVHD) grade III-IV will be obtained from the KM estimates along with 95% confidence intervals. (NCT01707004)
Timeframe: Day 100

Interventionpercentage of participants (Number)
Decitabine + Bone Marrow Transplant27.8

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Number of Participants With Complete Remission After Transplantation

(NCT01707004)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Decitabine + Bone Marrow Transplant14

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Cal-1 Marking in Gut-associated Lymphoid Tissue (GALT) (10-15 cm)

Samples were collected via endoscopic biopsy from the sigmoid colon: 10-15 cm from the anal margin (NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventioncopies/cell (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.81210.8121
Cohort 2 (CSL202 With 1 Busulfan Dose)0.00070.0004
Cohort 3 (CSL202 With 2 Busulfan Doses)0.00220.0001

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Number of Participants With HIV-1 Tropism Shift

Shift from R5 to X4 or dual/mixed tropism (NCT01734850)
Timeframe: Up to 48 weeks

InterventionParticipants (Count of Participants)
Cohort 1 (CSL202 With No Busulfan)0
Cohort 2 (CSL202 With 1 Busulfan Dose)0
Cohort 3 (CSL202 With 2 Busulfan Doses)0

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Mean Cell Dose for CD4+ Cells (Ttn)

(NCT01734850)
Timeframe: Up to 48 weeks

InterventionNumber (10^8 cells) (Mean)
Cohort 1 (CSL202 With No Busulfan)16.940
Cohort 2 (CSL202 With 1 Busulfan Dose)81.855
Cohort 3 (CSL202 With 2 Busulfan Doses)49.553

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Percent Transduction Efficiency of CD4+ Cells (Ttn) and CD34+ Cells (HSPCtn) of Final Cell Product

(NCT01734850)
Timeframe: Up to 48 weeks

,,
InterventionPercent transduction efficiency (Mean)
CD4+ Cells (Ttn)CD34+ Cells (HSPCtn)
Cohort 1 (CSL202 With No Busulfan)41.058.6
Cohort 2 (CSL202 With 1 Busulfan Dose)29.226.3
Cohort 3 (CSL202 With 2 Busulfan Doses)66.831.3

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Percent Cal-1 Marking in Peripheral Blood

(NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventionpercent Cal-1 marking (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.35000.0000
Cohort 2 (CSL202 With 1 Busulfan Dose)0.76500.1275
Cohort 3 (CSL202 With 2 Busulfan Doses)3.12000.1275

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Total Area Under the Curve (AUC) for Busulfan

Cohort 3: Total AUC = first dose AUC value + second dose AUC value (NCT01734850)
Timeframe: Up to 48 weeks

Interventionmicromolar*min (Mean)
Cohort 2 (CSL202 With 1 Busulfan Dose)6521.5
Cohort 3 (CSL202 With 2 Busulfan Doses)8296.8

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Number of Participants With Severe and Life-threatening Adverse Events (AEs)

(NCT01734850)
Timeframe: Up to 48 weeks

,,
InterventionParticipants (Count of Participants)
SevereLife-threatening
Cohort 1 (CSL202 With No Busulfan)00
Cohort 2 (CSL202 With 1 Busulfan Dose)43
Cohort 3 (CSL202 With 2 Busulfan Doses)44

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Number of Participants With Predominant Integration Site Analysis

Vector Integration Site Analysis performed only when Cal-1 Marking is >= 1%. (NCT01734850)
Timeframe: Up to 48 weeks

InterventionParticipants (Count of Participants)
Cohort 1 (CSL202 With No Busulfan)NA
Cohort 2 (CSL202 With 1 Busulfan Dose)NA
Cohort 3 (CSL202 With 2 Busulfan Doses)NA

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HIV Viral Load at Baseline Screening and Week 48 or at Anti-retroviral Therapy (ART) Re-commencement

(NCT01734850)
Timeframe: Up to 48 weeks

,,
InterventionLog10 (copies/mL) (Mean)
Baseline screeningWeek 48 or at ART re-commencement
Cohort 1 (CSL202 With No Busulfan)4.594.56
Cohort 2 (CSL202 With 1 Busulfan Dose)4.514.70
Cohort 3 (CSL202 With 2 Busulfan Doses)4.284.61

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Number of Participants With the Presence of Replication-competent Retrovirus

(NCT01734850)
Timeframe: Up to 48 weeks

InterventionParticipants (Count of Participants)
Cohort 1 (CSL202 With No Busulfan)0
Cohort 2 (CSL202 With 1 Busulfan Dose)0
Cohort 3 (CSL202 With 2 Busulfan Doses)0

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Cal-1 C46 Expression in Peripheral Blood

C46 relative expression will be analyzed by reverse transcriptase-quantitative polymerase chain reaction (RT-qPCR) and normalized to the expression of β2-microglobulin (β2M) mRNA (NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventionunits of relative expression (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.00000.0000
Cohort 2 (CSL202 With 1 Busulfan Dose)1.04960.0000
Cohort 3 (CSL202 With 2 Busulfan Doses)2.17010.0000

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Cal-1 Marking in Bone Marrow

(NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventioncopies/cell (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.00070.0007
Cohort 2 (CSL202 With 1 Busulfan Dose)0.05390.0015
Cohort 3 (CSL202 With 2 Busulfan Doses)0.00210.0009

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Cal-1 Marking in GALT (25-35 cm)

Samples were collected via endoscopic biopsy from the sigmoid colon: 25-35 cm from the anal margin (NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventioncopies/cell (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.02550.0255
Cohort 2 (CSL202 With 1 Busulfan Dose)0.00030.0003
Cohort 3 (CSL202 With 2 Busulfan Doses)0.00680.0055

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Mean Cell Dose for CD34+ Cells (HSPCtn)

(NCT01734850)
Timeframe: Up to 48 weeks

InterventionNumber (10^6 cells/kg body weight) (Mean)
Cohort 1 (CSL202 With No Busulfan)2.1
Cohort 2 (CSL202 With 1 Busulfan Dose)2.4
Cohort 3 (CSL202 With 2 Busulfan Doses)10.6

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Cal-1 sh5 Expression in Peripheral Blood

sh5 relative expression will be analyzed by RT-qPCR and normalized to the expression of RNU38B microRNA (NCT01734850)
Timeframe: Up to 48 weeks

,,
Interventionunits of relative expression (Mean)
Peak markingWeek 48
Cohort 1 (CSL202 With No Busulfan)0.00000.0000
Cohort 2 (CSL202 With 1 Busulfan Dose)0.20270.0000
Cohort 3 (CSL202 With 2 Busulfan Doses)1.69330.0000

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CD4+ Count at Baseline Screening and Week 48 or at ART Re-commencement

(NCT01734850)
Timeframe: Up to 48 weeks

,,
InterventionCD4+ cells/cubic mm (Mean)
Baseline screeningWeek 48 or at ART re-commencement
Cohort 1 (CSL202 With No Busulfan)680.75553.3
Cohort 2 (CSL202 With 1 Busulfan Dose)575.13383.5
Cohort 3 (CSL202 With 2 Busulfan Doses)668.63245.5

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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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Number of Participants That Engrafted and the Number of Participants That Had Full Donor Chimerism at Day 60

To estimate the number of participants that had engraftment rates and the number of participants that had full donor chimerism at Day 60 in patients undergoing an HLA haploidentical stem cell transplant with post transplant high dose cyclophosphamide. (NCT01749293)
Timeframe: Up to Day 60 post-transplant.

InterventionParticipants (Count of Participants)
Haploidentical Transplant0

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Number of Participants That Had an Event Free Survival Rate

To estimate the number of participants who had an event free survival rate (NCT01749293)
Timeframe: Up to 1 year post-transplant

InterventionParticipants (Count of Participants)
Haploidentical Transplant0

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Number of Participants That Had an Overall Survival Rate

To estimate the number of participants that had an overall survival (OS) rate (NCT01749293)
Timeframe: Up to one year post-transplant.

InterventionParticipants (Count of Participants)
Haploidentical Transplant1

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Percentage of Participants Experiencing Grade 3 or Higher Non-Hematologic or Grade 4 or Higher Hematologic Adverse Events

Percentage of participants experiencing grade 3 or higher non-hematologic or grade or higher hematologic adverse events evaluated by CTCAE v5.0. (NCT01773395)
Timeframe: 18 Months

InterventionPercentage of participants (Number)
GVAX3.33
Placebo0

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18-Month Progression Free Survival

"Progression-Free Survival (PFS) at 18 months after randomization. Progression-free survival is defined as time from randomization to progression of disease or death whichever occurs first. Patients with relapse/progressive disease who re-enter remission after vaccination or upon withdrawal of immune suppression alone will not be considered as having disease progression. The primary analysis will be performed using the modified intention-to-treat (ITT) principle, i.e., all transplanted and eligible patients who receive at least one vaccination will be included in the analysis according to the treatment arm they are randomized to. Patients who have not progressed and are alive are censored at the date the patient is known to be progression-free.~Progression is defined by either morphological evidence of acute leukemia or MDS consistent with pre-transplant features~is defined by either morphological evidence of acute leukemia or MDS consistent with pre-transplant features" (NCT01773395)
Timeframe: 18 months

InterventionPercentage (Number)
GVAX53
Placebo55

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18-Month Overall Survival

Assessment of overall survival at 18 months. Participants alive at last contact are censored at last contact. (NCT01773395)
Timeframe: 18 Month

InterventionPercentage of participants (Number)
GVAX63
Placebo59

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Percentage of Participants Experiencing Acute and Chronic Graft-Versus-Host Disease

"Percentage of participants with acute and chronic graft-versus-host disease (GVHD). The time frame for the acute incidence is 1 year and for chronic is 3 years.Grading of aGVHD was derived by consensus grading (Przepiorka 1995). The aGVHD algorithm calculates the grade based on the organ (skin, GI and liver) stage and etiology/biopsy reported. Skin staging: Stage 1. <25% rash; 2. 25-50%; 3. >50%; 4. generalized erythroderma with bullae. GI staging: Stage 1. Diarrhea>500ml/d or persistent nausea; 2. >1000ml/d; 3. >1500ml/d; 4. Large volume diarrhea and severe abdominal pain +- ileus. Liver staging: Stage 1. bilirubin 2-3mg/dl; 2. bili 3-6 mg/dl; 3. bili 6-15 mg/dl; 4. bili>15mg/dl. Grade 4 is the worst outcome.~Chronic GVHD is classified using the NIH Consensus Criteria. 8 organs will be scored on a 0-3 scale to reflect degree of cGVHD involvement 0 being none, 1 mild, 2 moderate 3 severe. Liver and pulmonary function test results will also be recorded. Severe is the worst outcome" (NCT01773395)
Timeframe: 1 and 3 years

,
InterventionPercentage of participants (Number)
Grade 2-4 aGVHD @1yrGrade 3-4 aGVHD @1yrChronic GVHD @3yrsMod-severe cGVHD @3yrs
GVAX34164723
Placebo1205933

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Percentage of Participants With Relapse and/or Non-Relapse Mortality

Percentage of participants with relapse and/or non-relapse mortality at 18 months from registration. (NCT01773395)
Timeframe: 18 Months

,
InterventionPercentage of participants (Number)
Non-relapse MortalityRelapse
GVAX1730
Placebo7.737

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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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Number of Participants Who Experienced Dose-limiting Toxicities (DLT)

Participants that experienced DLT related to the NK Cells post transplant at different dose levels. (NCT01823198)
Timeframe: Up to 42 days

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6222
Phase I: NK Cell Dose Level 2_10^7123
Phase I: NK Cell Dose Level 3_ 3x10^7022
Phase I: NK Cell Dose Level 4_ 10^8022
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^80129

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Overall Survival

Participants that survived between day of transplant and day of death on different dose levels. (NCT01823198)
Timeframe: Up to 2 years

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6212
Phase I: NK Cell Dose Level 2_10^7010
Phase I: NK Cell Dose Level 3_ 3x10^7003
Phase I: NK Cell Dose Level 4_ 10^8012
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^8072

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Number of Participants With Grade 3 Toxicities

Number of participants that had grade 3 toxicities up to day 42. (NCT01823198)
Timeframe: Up to day 42

,,,,,
InterventionParticipants (Count of Participants)
Group A: NK cells from KIR mismatched haplo donorsGroup B: NK cells from KIR mismatched cord blood donorsGroup C: NK cells from matched related donors
Phase I: NK Cell Dose Level 1_10^6212
Phase I: NK Cell Dose Level 2_10^7122
Phase I: NK Cell Dose Level 3_ 3x10^7012
Phase I: NK Cell Dose Level 4_ 10^8012
Phase II: NK Cell Dose Level 3_3x10^7010
Phase II: NK Cell Dose Level 4_ 10^80116

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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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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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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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OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

OS is defined as the percentage of subjects alive at 24 months post- treatment with OTL-101 or HSCT (NCT01852071)
Timeframe: 24 months

Interventionpercentage of participants (Number)
OTL-101 Gene Therapy100
HSCT Controls Without MRD85.71
HSCT Controls With MRD90.91
All HSCT Control Group88.00

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Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with OTL-101 or HSCT (NCT01852071)
Timeframe: 12 months

Interventionpercentage of participants (Number)
OTL-101 Gene Therapy100
HSCT Controls Without MRD85.71
HSCT Controls With MRD100
All HSCT Control Group92.31

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Reduction in Deoxyadenosine Nucleotide (dAXP) in Erythrocytes

Decreased dAXP levels coincide with increased ADA enzyme activity, detoxification was used to demonstrate functional ADA enzyme production from the introduced ADA transgene. The threshold for detoxification was <100 μmol/L. (NCT01852071)
Timeframe: 24 months

Interventionumol/mL (Median)
OTL-101 Gene Therapy0.0330
HSCT Controls Without MRD0.0360
All HSCT Control Group0.0360

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Severe Infection Rate Excluding the First Three Months After Treatment

The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning. (NCT01852071)
Timeframe: 24 months

InterventionInfection rate per person per year (Number)
OTL-101 Gene Therapy0.20
HSCT Controls Without MRD0.56
HSCT Controls With MRD0.15
All HSCT Control Group0.36

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Vector Copy Number (VCN) in Peripheral Blood (PB) Granulocytes.

Vector copy number in the PB granulocyte fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days. VCN analysis was performed by Droplet Digital PCR (ddPCR) on DNA extracted from peripheral blood granulocytes. (NCT01852071)
Timeframe: 24 months

Interventioncopies/cell (Median)
OTL-101 Gene Therapy0.093

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Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT01852071)
Timeframe: 12 months

Interventionpercentage of participants (Number)
OTL-101 Gene Therapy100
HSCT Controls Without MRD64.29
HSCT Controls With MRD100
All HSCT Control Group80.77

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ADA Activity in Erythrocytes

ADA enzyme activity measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV; persistence of ADA enzyme activity over time demonstrates successful engraftment and differentiation of genetically modified HSC. (NCT01852071)
Timeframe: 24 months

Interventionnmol/h/mg (Median)
OTL-101 Gene Therapy105.50
HSCT Controls Without MRD48.500
HSCT Controls With MRD1.000
All HSCT Control Group2.000

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Change From Baseline in CD3+ T Cell Counts (2 Years)

Immune reconstitution was assessed by change in CD3+ T Cell counts at baseline to Month 24. (NCT01852071)
Timeframe: 24 months

Interventioncells/μL (Median)
OTL-101 Gene Therapy569.0
HSCT Controls Without MRD340.0
HSCT Controls With MRD538.0
All HSCT Control Group395.5

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EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT01852071)
Timeframe: 24 months

Interventionpercentage of participants (Number)
OTL-101 Gene Therapy100
HSCT Controls Without MRD50.00
HSCT Controls With MRD63.64
All HSCT Control Group56.00

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Number of Single Integration Sites Representing >30% of the Total Integration Sites (2 Years)

Vector Integration Site Analysis (VISA) allowed determination of the distribution of vector integration sites in each subject's genome, as well as the relative clonal abundance. VISA was to be considered abnormal for a subject if, in 2 or more instances during the course of follow-up, a single integration site was found to represent >30% of the total integration sites detected. (NCT01852071)
Timeframe: 24 months

Interventionnumber of participants (Number)
OTL-101 Gene Therapy0

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VCN in Peripheral Blood Mononuclear Cells (PBMCs)

PBMC VCN is a measure of the accumulation of peripheral blood leukocytes arising from engrafted, genetically modified HSC. VCN analysis was performed by ddPCR on DNA extracted from PBMC. (NCT01852071)
Timeframe: 24 months

Interventioncopies/cell (Median)
OTL-101 Gene Therapy0.972

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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)

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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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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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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Number of Participants With Acute GVHD, Graded by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0

(NCT01894477)
Timeframe: Up to 84 days

InterventionParticipants (Count of Participants)
Arm A (Treosulfan, Fludarabine Phosphate)29
Arm B (Treosulfan, Fludarabine Phosphate, TBI)50

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Number of Participants That Did Not Progress Within 6 Months

Progression is defined as relapse (NCT01894477)
Timeframe: At 6 months post-transplant

InterventionParticipants (Count of Participants)
Arm A (Treosulfan, Fludarabine Phosphate)20
Arm B (Treosulfan, Fludarabine Phosphate, TBI)48

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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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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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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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Cumulative Incidence of Chronic GVHD

Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD). (NCT02120157)
Timeframe: 2 years

Interventionpercent (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS11

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Cumulative Incidence of Non-relapse Mortality

Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies. (NCT02120157)
Timeframe: Day 180

Interventionpercent (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS0

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Incidence of Donor Cell Engraftment

Incidence of donor cell engraftment measured as the percentage of donor cell engraftment. (NCT02120157)
Timeframe: 60 days

Interventionpercentage of donor cell engraftment (Number)
Haploidentical BMT With PTCy for Acute Leukemias and MDS84

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Number of Participants With Donor Cell Engraftment

Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT. (NCT02120157)
Timeframe: Day 60

InterventionParticipants (Count of Participants)
Haploidentical BMT With PTCy for Acute Leukemias and MDS27

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Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4

Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe). (NCT02120157)
Timeframe: 100 days

Interventionpercent (Number)
Grades 2-4Grades 3-4
Haploidentical BMT With PTCy for Acute Leukemias and MDS100

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Primary and Secondary Graft Failure

Incidence (measured as a percentage) of primary and secondary graft failure. (NCT02120157)
Timeframe: 2 years

Interventionpercentage of graft failure (Number)
PrimarySecondary
Haploidentical BMT With PTCy for Acute Leukemias and MDS160

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Steroid and Non-steroid Immunosuppressants

Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD. (NCT02120157)
Timeframe: Two Years

InterventionParticipants (Count of Participants)
Steroid immunosuppressantsNon-steroid immunosuppressants
Haploidentical BMT With PTCy for Acute Leukemias and MDS42

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Steroid and Non-steroid Immunosuppressants Use Duration

Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD. (NCT02120157)
Timeframe: Two Years

Interventionmonths (Number)
Steroid immunosuppressantsNon-steroid immunosuppressants
Haploidentical BMT With PTCy for Acute Leukemias and MDS1819

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Survival

Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage. (NCT02120157)
Timeframe: up to 1 years

Interventionpercent (Number)
overall survival (OS)progression-free survival (PFS)disease-free survival (DFS)event-free survivalRelapse-free GVHD-free survival (GRFS)
Haploidentical BMT With PTCy for Acute Leukemias and MDS7768686865

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Survival

Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage. (NCT02120157)
Timeframe: up to 2 years

Interventionpercent (Number)
overall survival (OS)progression-free survival (PFS)disease-free survival (DFS)event-free survivalRelapse-free GVHD-free survival (GRFS)
Haploidentical BMT With PTCy for Acute Leukemias and MDS7364646452

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Time to Neutrophil and Platelet Recovery

Time to neutrophil and platelet recovery in median days (NCT02120157)
Timeframe: 100 days

Interventiondays (Median)
neutrophilplatelet
Haploidentical BMT With PTCy for Acute Leukemias and MDS2221

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12 Month Disease Free Survival Probability

The percentage of patients who experience death or disease relapse by one year will be calculated and a corresponding 95% confidence interval will be constructed using the normal approximation for binomial proportions. The survival function will be estimated and plotted using the method of Kaplan and Meier. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Overall Survival

The survival function will be estimated and plotted using the method of Kaplan and Meier. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)66.7

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Progression Free Survival

Kaplan-Meier estimation of the percentage of patients who are alive without progressive disease at one year. (NCT02248597)
Timeframe: At 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Rate of Acute GvHD

Kaplan-Meier estimation of the rate of acute GvHD in the study population at one year with a 95% confidence interval. (NCT02248597)
Timeframe: 12 months

Interventionpercentage of participants (Number)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)51.8

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Relapse-free Mortality

Non-relapse mortality will be defined as time from registration to death due to anything other than relapse of hematological malignancy. Patients who relapse will be treated as a competing risk. (NCT02248597)
Timeframe: At 12 months

Interventiondays (Mean)
Treatment (Stem Cell Transplant With GVHD Prophylaxis)86.8

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To Determine the Efficacy of This Allogeneic Transplant Approach in Reconstituting Normal Hematopoiesis and Reversing the Clinical Phenotype of CGD

Patient will have donor chimerism of greater than 20% and resolution of infection or autoimmunity at end of follow up (NCT02282904)
Timeframe: 5 years

InterventionParticipants (Count of Participants)
Greater than 20% donor chimerismResolution of inflammation or infection
CGD Recipient77

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Change From Baseline in Quality of Life Measures (2 Years)

Assessment of quality of life was measured by the Lansky Performance Status Scale. The maximum score on the Lansky Performance Scale is 100 - the child is fully active and able to carry on normal activity with no special care needed. The minimum score is 10 - the child is completely disabled, not even passive play. The scores at baseline (pre-treatment with OTL-101) and scores at Month 24 post-treatment with OTL-101 were compared to establish if there were any changes in the child's score in this timeframe. (NCT02999984)
Timeframe: 24 months

InterventionLansky Performance Score (Median)
Gene Therapy0

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Event-free Survival (EvFS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogeneic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT02999984)
Timeframe: 12 Months

Interventionpercentage of participants (Number)
Percentage event-free at 12 monthsPercentage without reinstitution of PEG-ADA by 12 monthsPercentage without rescue HSCT by 12 months
Gene Therapy90.0090.00100

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EvFS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

"Event-free survival is defined as the percentage of subjects alive with no event, an event being the resumption of PEG-ADA ERT or the need for a rescue allogenic Hematopoietic Stem Cell Transplant (HSCT), or death." (NCT02999984)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Percentage event-free at 24 monthsPercentage without reinstitution of PEG-ADA by 24 monthsPercentage without rescue HSCT by 24 months
Gene Therapy90.0090.00100

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Change From Baseline in CD3+ T Cell Counts (2 Years)

Immune reconstitution was assessed by change in CD3+ T Cell counts over time. (NCT02999984)
Timeframe: 24 months

Interventioncells/μL (Median)
Gene Therapy418.5

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Percentage of Participants With Treatment Efficacy After Treatment With OTL 101 (6 Months)

"Efficacy of OTL-101 treatment at 6 months post OTL-101 infusion based on the following parameters and thresholds:~ADA enzyme activity in erythrocytes above baseline/pretreatment level (i.e., >0 units). ADA enzyme activity is measured to assess the amount of functional gene product produced from the normal ADA transgene delivered by EFS-ADA LV.~Absolute CD3+ T cell counts ≥200 cells/μL. Increase in CD3+ T cell counts is a marker of immune reconstitution.~Granulocyte samples positive for vector sequences by quantitative Polymerase Chain Reaction (PCR) (≥1/10,000 cells). Vector copy number (VCN) in the Peripheral Blood (PB) Granulocytes fraction that was T cell depleted, is a surrogate for amount of engrafted genetically modified Hematopoietic stem cell (HSC) that are producing granulocytes every 3-5 days." (NCT02999984)
Timeframe: 6 months

Interventionpercentage of participants (Number)
% of subjects with an increase from baseline in RBC ADA activity% of subjects with CD3+ T-cell count >=200 cells/μL% of subjects with detectable gene-marked granulocytes by quantitative PCR >=1/10,000 cells
Gene Therapy10010090

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Overall Survival (OS) of Subjects Treated With Investigational Medicinal Product (IMP) (1 Year)

Overall survival is defined as the percentage of subjects alive at 12 months post- treatment with cryopreserved OTL-101. (NCT02999984)
Timeframe: 12 Months

Interventionpercentage of participants (Number)
Gene Therapy100

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OS of Subjects Treated With Investigational Medicinal Product (IMP) (2 Years)

OS is defined as the percentage of subjects alive at 24 months post- treatment with cryopreserved OTL-101. (NCT02999984)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Gene Therapy100

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Percentage of Patients Who Stopped Immunoglobulin Replacement Therapy (IgRT) (2 Years)

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. Indications for considering the discontinuation of immunoglobulin replacement therapy included: absolute CD4+ >200, absolute B cell >100/μl, IgA or IgM > lower limit of normal for age or gene marking >1% detectable in B cells. (NCT02999984)
Timeframe: 24 months

Interventionpercentage of participants (Number)
Gene Therapy78

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Severe Infections Excluding First 3 Months After Treatment

The infections of interest in this study were severe infections or opportunistic infectious episodes, defined as infections requiring hospitalization or prolonging hospitalization and/or documented infections by opportunistic pathogens. Infections that took place in the first 3 months of follow-up post treatment were excluded from calculations to avoid possible bias introduced in the data by the effects of conditioning. (NCT02999984)
Timeframe: 24 months

InterventionInfections per person per year (Number)
Gene Therapy0.12

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Time to Cessation of IgRT for Those Who Stopped (2 Years)

Use of immunoglobulin replacement therapies prior to and after gene therapy were monitored. For subjects who stopped IgRT during the study, the time of cessation was recorded. (NCT02999984)
Timeframe: 24 months

Interventionmonths (Median)
Gene Therapy11.6

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Progression Free Survival (PFS)

Progression-free survival defined by the time interval from transplant to relapse/recurrence, to death or to last follow-up. Reported as percentage of participants who are disease free one year post HCT. (NCT03018223)
Timeframe: Up to 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis56.6

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Overall Survival (OS)

Overall survival is defined as time from transplant to death or last follow-up, and is reported as percentage of surviving participants. (NCT03018223)
Timeframe: Up to 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis70.2

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Incidence of Grade II-IV Acute Graft vs. Host Disease (GVHD)

Cumulative incidence of grade II-IV acute GVHD by day 100 after HCT. Acute GVHD organ staging and assessment of overall grade will use standard consensus criteria. The cumulative incidence of acute and chronic GVHD will be estimated, considering malignancy relapse and non-relapse death as competing risk events. (NCT03018223)
Timeframe: 100 days post hematopoietic cell transplant (HCT)

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis18.8

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Incidence of Chronic GVHD

Cumulative incidence of chronic GVHD by 1 year. Chronic GVHD diagnosis follow National Institutes of Health (NIH) Consensus guidelines. (NCT03018223)
Timeframe: 1 year post HCT

Interventionpercentage of participants (Number)
Conditioning/HCT/GVHD Prophylaxis20.0

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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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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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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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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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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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Non-relapse Mortality (NRM) at Day 100

NRM is defined as death due to GVHD, infections, sepsis, organ (lung, liver, kidney) toxicity. Death from underlying disease (i.e. progression or relapse) is not considered NRM. (NCT03303950)
Timeframe: Up to day 100

InterventionParticipants (Count of Participants)
All Participants - Treatment1

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Non-relapse Mortality (NRM) at Day 365

NRM is defined as death due to GVHD, infections, sepsis, organ (lung, liver, kidney) toxicity. Death from underlying disease (i.e. progression or relapse) is not considered NRM. (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Participants - Treatment2

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Number of Participants With Minimal Residual Disease (MRD) Response

After bone marrow transplant, bone marrow was collected every 3-6 months (as clinically indicated per treating investigator) for up to one year after bone marrow transplant. Bone marrow was evaluated by a clinical pathologist for any evidence of multiple myeloma (MM) or myelofibrosis (MF). Evidence of MM or MF in the bone marrow is referred to as minimal residual disease (MRD). A participant with evidence of MRD is MRD-positive. A participant with no evidence of MRD is MRD-negative, which is considered an MRD response. This outcome reports the number participants with MRD responses any time between bone marrow transplant up to one year of follow-up. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment5

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Overall Survival at One Year

Overall survival is defined as the number of participants remaining alive up to one year following stem cell transplant. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment3

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Disease Free Survival at One Year

Disease free survival is defined as an absence of disease relapse or progression up to one year following stem cell transplant. (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
All Particpants - Treatment3

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Incidence of Acute Graft Versus Host Disease (GVHD)

Cases of acute graft versus host disease (GVHD) will be diagnosed by the treating physician and will be reported as a count of participants with acute GVHD. (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Particpants - Treatment2

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Number of Participants With Different Clinical Responses

Clinical Responses were determined by disease-specific criteria taking into account multiple clinical and molecular markers. Multiple Myeloma (MM) response was determined using International Myeloma Working Group (IMWG) consensus criteria for response. Participants with MM had either Stringent Complete Response (sCR) or Very Good Partial Response (VGPR). Myelofibrosis (MF) response was determined using International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus criteria. Participants with MF had either Complete Response (CR) or Stable Disease (SD) (also referred to in the protocol as no response). (NCT03303950)
Timeframe: Up to 1 year

InterventionParticipants (Count of Participants)
Stringent Complete Response (sCR)Very Good Partial Response (VGPR)Complete Response (CR)Stable Disease (SD)
All Particpants - Treatment1121

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Incidence of Chronic GVHD

Chronic GVHD will be assessed based on criteria established by the National Institutes of Health Consensus Development Project in 2005, and recently updated in 2014. This will be reported as a count of participants diagnosed with chronic GVHD (NCT03303950)
Timeframe: Up to day 365

InterventionParticipants (Count of Participants)
All Particpants - Treatment1

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Neutrophil Engraftment

Rates of engraftment, defined as the first day of Absolute Neutrophil Count (ANC) greater than 500 for the first of three consecutive days (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Severity of Chronic GVHD

The highest overall grade (1-4) of chronic GvHD experienced by participants as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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

The highest grade (1-4) of acute GvHD experienced by participants as measured from day of transplantation to Day +100 using the Glucksberg criteria (NCT04002115)
Timeframe: 100 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Rate of Chronic GVHD

The rate of any grade (1-4) of Chronic GvHD as measured from Day +100 to Year 1 post-transplantation using the Glucksberg criteria. (NCT04002115)
Timeframe: 1 year

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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

The rate of any grade (1-4) of acute GvHD as measured from day of transplantation to Day +100 using the Glucksberg criteria. (NCT04002115)
Timeframe: 100 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^20

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Complete Remission (CR) Rate at Day 30 Post HSCT

The CR rate at 30 days (Day +30) post stem cell transplant infusion (NCT04002115)
Timeframe: 30 days

Interventionpercentage of Participants (Number)
Clofarabine 30 mg/m^2100

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