Page last updated: 2024-12-08

deferasirox

Description Research Excerpts Clinical Trials Roles Classes Pathways Study Profile Bioassays Related Drugs Related Conditions Protein Interactions Research Growth Market Indicators

Description

Deferasirox: A triazole and benzoate derivative that acts as a selective iron chelator. It is used in the management of chronic IRON OVERLOAD due to blood transfusion or non-transfusion dependent THALASSEMIA. [Medical Subject Headings (MeSH), National Library of Medicine, extracted Dec-2023]

deferasirox : A member of the class of triazoles, deferasirox is 1,2,4-triazole substituted by a 4-carboxyphenyl group at position 1 and by 2-hydroxyphenyl groups at positions 3 and 5. An orally active iron chelator, it is used to manage chronic iron overload in patients receiving long-term blood transfusions. [Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Cross-References

ID SourceID
PubMed CID214348
CHEMBL ID550348
CHEBI ID49005
SCHEMBL ID61756
SCHEMBL ID62042
SCHEMBL ID1071867
MeSH IDM0490722

Synonyms (114)

Synonym
HY-17359
icl-670a
icl-670
deferasirox
cgp-72670
exjade
deferasirox (jan/usan/inn)
D03669
201530-41-8
exjade (tn)
jadenu (tn)
NCGC00181754-01
4-(3,5-bis-(2-hydroxyphenyl)-(1,2,4)-triazol-1-yl)benzoic acid
deferasiroxum
icl 670
CHEBI:49005 ,
icl 670a
4-[3,5-bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl]benzoic acid
DB01609
deferasiroxum [inn-latin]
4-(3,5-bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl)benzoic acid
icl670
icl670a
benzoic acid, 4-(3,5-bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl)-
bdbm50088376
osveral
CHEMBL550348
FT-0665569
4-[(3z,5e)-3,5-bis(6-oxocyclohexa-2,4-dien-1-ylidene)-1,2,4-triazolidin-1-yl]benzoic acid
cas-201530-41-8
dtxsid1048596 ,
dtxcid1028522
tox21_112926
A814288
4-[3,5-bis(6-oxocyclohexa-2,4-dien-1-ylidene)-1,2,4-triazolidin-1-yl]benzoic acid
BCP9000594
4-[3,5-bis(2-hydroxyphenyl)-1,2,4-triazol-1-yl]benzoic acid
jadenu
unii-v8g4mof2v9
v8g4mof2v9 ,
jadenu sprinkle
hsdb 7844
deferasirox [usan:inn:jan]
smr002544689
MLS003915634
FT-0601564
deferasirox [mart.]
deferasirox [mi]
deferasirox [jan]
deferasirox [vandf]
deferasirox [inn]
deferasirox [usan]
deferasirox [orange book]
deferasirox [ema epar]
deferasirox [usp-rs]
deferasirox [ep monograph]
deferasirox [who-dd]
deferasirox [hsdb]
CS-0901
S1712
AKOS015902587
AKOS015855839
SCHEMBL61756
SCHEMBL62042
BOFQWVMAQOTZIW-UHFFFAOYSA-N
4-[3,5-bis(2-hydroxyphenyl)-[1,2,4]triazol-1-yl]benzoic acid
MLS006010080
tox21_112926_1
NCGC00263572-01
deferasirox, icl670
KE-0220
SCHEMBL1071867
4-((3e,5e)-3,5-bis(6-oxocyclohexa-2,4-dien-1-ylidene)-1,2,4-triazolidin-1-yl)benzoic acid
1044764-54-6
F0001-2380
4-[3,5-bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl]benzoic acid; cgp-72670; icl-670; icl-670a
AB01565800_02
mfcd09951804
AKOS025312553
benzoic acid, 4-[3,5-bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl]-
AC-8972
sr-01000931971
SR-01000931971-2
4-[(3z,5e)-3,5-bis(6-oxo-2,4-cyclohexadien-1-ylidene)-1,2,4-triazolidin-1-yl]benzoic acid, aldrichcpr
HMS3655A04
mfcd09751362
J-013060
4-[bis(2-hydroxyphenyl)-1h-1,2,4-triazol-1-yl]benzoic acid
SW219752-1
BCP05341
Q5251502
deferasirox (exjade)
EX-A4091
SB18926
CCG-268337
4-(3,5-bis(6-oxocyclohexa-2,4-dien-1-ylidene)-1,2,4-triazolidin-1-yl)benzoic acid
deferasirox 100 microg/ml in acetonitrile
JBL ,
EN300-26907573
4-[(3z,5e)-3,5-bis(6-oxocyclohexa-2,4-dien-1ylidene)-1,2,4-triazolidin-1-yl] benzoic acid
4-(3,5-bis(6-oxocyclohexa-2,4-dien-1-ylidene)-1,2,4-triazolidin-1-yl)benzoicacid
D5905
deferasirox (ep monograph)
deferasirox (mart.)
deferasirox oral
deferasorox
deferasiroxum (inn-latin)
deferasirox (usp-rs)
v03ac03
deferasirox oral granules
4-(3,5-bis-(hydroxy-phenyl)-1,2,4-triazol-1-yl)-benzoic acid
Z3041510169
4-{3,5-bis[(1e)-6-oxocyclohexa-2,4-dien-1-ylidene]-1,2,4-triazolidin-1-yl}benzoic acid
PD087125

Research Excerpts

Overview

Deferasirox (DFX) is an oral iron-chelating agent and classified into class II of the Biopharmaceutics Classification System. It inhibits iron metabolism in cancer cells, used in the treatment of iron overload such as hemochromatosis.

ExcerptReferenceRelevance
"Deferasirox (DFX) is an iron-chelating agent effective in treating various kinds of cancers, which inhibits iron metabolism in cancer cells. "( Thermosensitive TMPO-oxidized lignocellulose/cationic agarose hydrogel loaded with deferasirox nanoparticles for photothermal therapy in melanoma.
Mirian, M; Rostami, M; Varshosaz, J; Veisi, H, 2023
)
2.58
"Deferasirox is an active iron chelator, used in the treatment of iron overload such as hemochromatosis. "(
Aguilar Hinojosa, NK; Figueroa Saavedra, EI, 2023
)
2.35
"Deferasirox is an oral iron chelator with established dose-dependent efficacy for the treatment of iron overload secondary to transfusion. "( The challenges of handling deferasirox in sickle cell disease patients older than 40 years.
Benites, BD; Costa, FF; Gilli, SCO; Olalla Saad, ST; Ribeiro, LB; Soares, EA, 2019
)
2.25
"Deferasirox is a once-daily oral iron-chelation agent approved by the US Food and Drug Administration in November 2005. "( Evaluation of Efficacy, Safety, and Satisfaction Taking Deferasirox Twice Daily Versus Once Daily in Patients With Transfusion-Dependent Thalassemia.
Ansari, S; Azarkeivan, A; Bahoush, G; Bazrafshan, A; Haghpanah, S; Jangjou, A; Karimi, M; Shahsavani, A, 2020
)
2.25
"Deferasirox (DFX) is an iron chelator approved for the treatment of iron overload diseases. "( Deferasirox protects against hydrogen peroxide-induced cell apoptosis by inhibiting ubiquitination and degradation of p21
Cao, P; Chang, Y; Duan, X; Fan, Y; Hasem, H; Hou, J; Kuang, Y; Miao, J; Pan, S; Tan, K; Xu, M; Zhou, N, 2020
)
3.44
"Deferasirox (DFX) is an oral iron-chelating agent and classified into class II of the Biopharmaceutics Classification System. "( Solid Dispersion Pellets: An Efficient Pharmaceutical Approach to Enrich the Solubility and Dissolution Rate of Deferasirox.
Abbaspour, M; Ebrahimnejad, P; Farmoudeh, A; Nokhodchi, A; Rezaeiroshan, A, 2020
)
2.21
"Deferasirox (DFR) is a trivalent iron chelator, which was approved to treat iron overload syndrome after transfusion."( Deferasirox, a trivalent iron chelator, ameliorates neuronal damage in hemorrhagic stroke models.
Hara, H; Imai, T; Matsubara, H; Nakamura, S; Ohba, T; Shimazawa, M; Sugiyama, T; Tsuji, S, 2021
)
2.79
"Deferasirox is an effective iron chelator when started at an optimum time and with optimum dose. "( Effectiveness of Deferasirox in Pediatric Thalassemia Patients: Experience from a Tertiary Care Hospital of Odisha.
Debta, N; Jena, RK; Panigrahi, A; Panigrahi, M; Swain, TR,
)
1.91
"Deferasirox is an oral iron chelator used to reduce iron levels in iron-overloaded patients with transfusion-dependent anemia or non-transfusion-dependent thalassemia. "( Effect of Genetic Polymorphisms on the Pharmacokinetics of Deferasirox in Healthy Chinese Subjects and an Artificial Neural Networks Model for Pharmacokinetic Prediction.
Chen, J; Hu, Y; Jiang, B; Lou, H; Ruan, Z; Shao, R; Xu, Y; Yang, D, 2020
)
2.24
"Deferasirox is an oral iron chelator that has been on the market since 2005 and has been a suitable replacement for injectable chelators. "( Sensitive determination of deferasirox in blood of patients with thalassemia using dispersive liquid-liquid microextraction based on solidification of floating organic drop followed by HPLC-UV.
Akramipour, R; Fattahi, N; Golpayegani, MR, 2021
)
2.36
"Deferasirox, ExJade, is an FDA-approved iron chelator used for the treatment of iron overload. "( Deferasirox (ExJade): An FDA-Approved AIEgen Platform with Unique Photophysical Properties.
Brewster, JT; Han, HH; He, XP; Hu, XL; Lynch, VM; Mangel, DN; Sedgwick, AC; Sessler, JL; Shang, Y; Snelson, DW; Steinbrueck, A; Tian, H; Yan, KC, 2021
)
3.51
"Deferasirox is an oral chelator approved for iron overload, which has a potential side effect of renal Fanconi syndrome, with proximal tubular dysfunction and tubular acidosis. "( Deferasirox-associated Fanconi syndrome in adult patients with transfusional iron overload.
Geara, A; Sayani, F; Yui, JC, 2021
)
3.51
"As deferasirox (DFX) is an approved Fe chelator drug, its inability to cross blood brain barrier (BBB) remains a definite obstacle against its use as Fe chelator in the brain."( Destructive effect of iron overload in brain tissue of albino rats: Ameliorative role of silver immobilized organo-modified casein nanocomposite as co-treating agent with Deferasirox.
Adel, RM; Amer, AS; Darwish, AS; Lotfy, RA, 2021
)
1.33
"Deferasirox is an orally active, lipophilic iron chelating drug used on thousands of patients worldwide for the treatment of transfusional iron overload. "( Antioxidant Activity of Deferasirox and Its Metal Complexes in Model Systems of Oxidative Damage: Comparison with Deferiprone.
Kichigina, LA; Kontoghiorghes, GJ; Polyakov, NE; Selyutina, OY; Timoshnikov, VA, 2021
)
2.37
"Deferasirox is an iron chelator agent used in the treatment of diseases with iron overload, such as thalassemia and myelodysplastic syndrome. "( Deferasirox-induced urticarial vasculitis in a patient with myelodysplastic syndrome.
Belli, AA; Dere, Y; Karakus, V; Polat, AK, 2017
)
3.34
"Deferasirox is an oral iron-chelating agent having possible antileukemia and immune modulatory effects. "( Improved survival outcomes and restoration of graft-vs-leukemia effect by deferasirox after allogeneic stem cell transplantation in acute myeloid leukemia.
Cho, BS; Cho, SG; Eom, KS; Hahn, AR; Jeon, YW; Kim, HJ; Kim, YJ; Lee, JW; Lee, S; Lee, SE; Lee, TH; Min, CK; Min, WS; Park, SS; Yoon, JH, 2019
)
2.19
"Deferasirox is a potent NF-kB inhibitor, tested in vivo and on acute myeloid leukemia and MDS cell lines, and this effect may explain in part the phenomenon of hematological improvements reported in case reports and in different clinical trials."( Efficacy and safety of deferasirox in myelodysplastic syndromes.
Alimena, G; Breccia, M, 2013
)
1.42
"Deferasirox is a once-daily, oral iron chelator that was developed out of a need for a long-acting, conveniently-administered chelator for patients with transfusional hemosiderosis. "( The palatability and tolerability of deferasirox taken with different beverages or foods.
Chirnomas, D; Esposito, J; Giardina, PJ; Goldberg, SL; Paley, C; Vichinsky, E, 2013
)
2.11
"Deferasirox is a new treatment of iron overload that is administered orally once-a-day, resulting in better acceptance in patients. "( Hyperchloraemic metabolic acidosis induced by the iron chelator deferasirox: a case report and review of the literature.
Bianchetti, MG; Brazzola, P; Dell'Orto, VG, 2013
)
2.07
"Deferasirox (Exjade(®)) is a once-daily orally administered iron chelator which has been approved for use in the treatment of transfusional-dependent chronic iron overload since 2005. "( Deferasirox: a review of its use for chronic iron overload in patients with non-transfusion-dependent thalassaemia.
Plosker, GL; Shirley, M, 2014
)
3.29
"Deferasirox (DFX) is an effective and well-tolerated oral iron chelator elevating the adherence to iron chelating therapy among patients with iron overload. "( Safety of deferasirox: a retrospective cohort study on the risks of gastrointestinal, liver and renal events.
Chou, HC; Hsiao, FY; Huang, WF; Tsai, YW, 2014
)
2.25
"Deferasirox is an oral iron chelator effective for reduction of body iron in iron-overloaded patients with transfusion-dependent anemias and non-transfusion-dependent thalassemia, with a well-established safety profile."( Clinical pharmacology of deferasirox.
Tanaka, C, 2014
)
1.43
"Deferasirox (DFX) is an oral iron chelator with established efficacy and safety. "( A 5-year follow-up in deferasirox treatment: improvement of cardiac and hepatic iron overload and amelioration in cardiac function in thalassemia major patients.
Cappellini, MD; Cassinerio, E; Consonni, D; Giuditta, M; Orofino, N; Pedrotti, P; Poggiali, E; Roghi, A; Zanaboni, L, 2015
)
2.17
"Deferasirox (DFX) is an orally administered iron chelator approved for use in patients with transfusion-dependent iron overload due to myelodysplastic syndromes (MDS). "( Deferasirox chelation therapy in patients with transfusion-dependent MDS: a 'real-world' report from two regional Italian registries: Gruppo Romano Mielodisplasie and Registro Basilicata.
Aloe Spiriti, MA; Breccia, M; Buccisano, F; Criscuolo, M; D'Addosio, A; Fenu, S; Fragasso, A; Latagliata, R; Maurillo, L; Musto, P; Niscola, P; Piccioni, AL; Refrigeri, M; Tatarelli, C; Trapè, G; Venditti, A; Voso, MT, 2015
)
3.3
"Deferasirox is a new oral iron chelating agent with several cases reporting renal adverse events in recent years. "( Transfusion-dependent thalassaemic patients with renal Fanconi syndrome due to deferasirox use.
Chuang, GT; Lu, MY; Tsai, IJ; Tsau, YK, 2015
)
2.09
"Deferasirox is a novel once-daily, oral iron chelator. "( The long-term efficacy and tolerability of oral deferasirox for patients with transfusion-dependent β-thalassemia in Taiwan.
Chang, HH; Jou, ST; Lin, DT; Lin, KH; Lu, MY; Peng, SS; Yang, YL, 2015
)
2.12
"Deferasirox is an oral iron chelator used widely in the treatment of thalassemia major and other transfusion-dependent hemoglobinopathies. "( Deferasirox at therapeutic doses is associated with dose-dependent hypercalciuria.
Bowden, DK; Doery, JC; Fuller, PJ; Gillespie, MT; Kerr, PG; Larmour, I; Milat, F; Polkinghorne, K; Wong, P, 2016
)
3.32
"Deferasirox is an oral iron chelator."( The potential of deferasirox as a novel therapeutic modality in gastric cancer.
Choi, JH; Kim, JS; Lee, YY; Park, BB; Uhm, J; Won, YW, 2016
)
1.5
"Deferasirox is a standard treatment for chronic transfusional iron overload. "( Proximal muscular atrophy and weakness: An unusual adverse effect of deferasirox iron chelation therapy.
Albert, MH; Blaschek, A; Gerstl, L; Huetker, S; Müller-Felber, W; Teusch, V; Vill, K,
)
1.81
"Deferasirox is an oral iron chelator used to treat patients with transfusion-related iron overload. "( Fanconi Syndrome Secondary to Deferasirox in Diamond-Blackfan Anemia: Case Series and Recommendations for Early Diagnosis.
Arora, S; Athale, UH; Bhatt, MD; Kirby-Allen, M; Papneja, K; Wiernikowski, JT, 2016
)
2.17
"Deferasirox is a once-daily orally active tridentate selective iron chelator which pharmacokinetic disposition could influence treatment efficacy and toxicity."( Deferasirox pharmacokinetic evaluation in β-thalassaemia paediatric patients.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Massano, D; Piga, A; Pirro, E, 2017
)
2.62
"Deferasirox is an oral chelator."( Evaluation of Proteinuria in β-Thalassemia Major Patients With and Without Diabetes Mellitus Taking Deferasirox.
Avazpour, A; Badie, A; Haghpanah, S; Karimi, M; Toosi, F, 2017
)
1.39
"Deferasirox is an oral active iron chelating agent."( The questioning for routine monthly monitoring of proteinuria in patients with β-thalassemia on deferasirox chelation.
Bayhan, T; Gümrük, F; Karabulut, E; Küçüker, H; Tutal, AD; Ünal, Ş; Ünlü, O, 2017
)
1.39
"Deferasirox is an oral iron chelator used to treat blood transfusion-related iron overload."( Deferasirox-induced iron depletion promotes BclxL downregulation and death of proximal tubular cells.
Carrasco, S; Egido, J; Fontecha-Barriuso, M; Gallegos-Villalobos, A; Lopez-Hernandez, FJ; Martin-Sanchez, D; Ortiz, A; Ruiz-Ortega, M; Sanchez-Niño, MD; Sanz, AB, 2017
)
2.62
"Deferasirox is a novel iron chelator formulated as tablets for dispersion (suspension) for once-a-day oral administration. "( Absolute oral bioavailability and disposition of deferasirox in healthy human subjects.
Balez, S; Belleli, R; Robeva, A; Séchaud, R, 2008
)
2.04
"Deferasirox is a new iron chelator approved recently for chelation therapy in iron-overloaded patients. "( Hypocalcemia in a dialysis patient treated with deferasirox for iron overload.
Brewster, UC; McPhedran, P; Yusuf, B, 2008
)
2.04
"Deferasirox is a new oral iron chelator used to treat transfusional iron overload. "( Acute interstitial nephritis due to deferasirox: a case report.
Brosnahan, G; Gokden, N; Swaminathan, S, 2008
)
2.06
"Deferasirox is an oral iron chelator, with a long half-life, that can be given once daily, because it provides a 24-hour chelation. "( Long-term efficacy and safety of deferasirox.
Cappellini, MD, 2008
)
2.07
"Deferasirox is an innovative iron-chelating treatment. "( Fanconi syndrome due to deferasirox.
Delarue, R; Fakhouri, F; Le Quintrec, M; Rafat, C; Ribeil, JA, 2009
)
2.1
"Deferasirox is a novel, orally active agent that provides 24-h chelation with a once-daily dose."( Oral iron chelators.
Cappellini, MD; Pattoneri, P, 2009
)
1.07
"Deferasirox is a novel, once daily oral iron chelator that was recently approved for the treatment of transfusional iron overload."( Deferasirox removes cardiac iron and attenuates oxidative stress in the iron-overloaded gerbil.
Al-Rousan, RM; Blough, ER; Gutta, AK; Kakarla, SK; Laurino, JP; Paturi, S; Walker, EM, 2009
)
2.52
"Deferasirox is a once-daily, orally administered, tridentate iron chelator that is indicated in the treatment of iron overload resulting from regular packed red blood cell transfusions in patients with transfusion-dependent anemias, such as beta-thalassemia, sickle cell disease, myelodysplastic syndrome and other rare anemias. "( Pharmacoeconomic benefits of deferasirox in the management of iron overload syndromes.
Imran, F; Phatak, P, 2009
)
2.09
"Deferasirox (DFX) is a daily oral iron chelator approved in the United States for transfusional iron overload."( Adherence to deferasirox in children and adolescents with sickle cell disease during 1-year of therapy.
Alvarez, O; Lewis, N; Lopez-Mitnik, G; Paley, C; Pow Sang, CD; Robinson, N; Rodriguez-Cortes, H, 2009
)
1.44
"Deferasirox is a once-daily oral iron chelator with established dose-dependent efficacy in both adult and pediatric patients with transfusional iron overload. "( Deferasirox (Exjade) for the treatment of iron overload.
Cappellini, MD; Taher, A, 2009
)
3.24
"Deferasirox (DFX) is a newly developed oral iron chelator that enables effective chelation with once daily administration. "( Hematopoietic recovery after administration of deferasirox for transfusional iron overload in a case of myelodysplastic syndrome.
HATANO, K; MATSUYAMA, T; MORI, M; MUROI, K; NAGAI, T; OKABE, H; OMORI, T; OZAWA, K; QZAKI, K; SUZUKI, T; TOSHIMA, M; UEDA, M; UEHARA, E, 2009
)
2.05
"Deferasirox (ICL670) is a novel once-daily, orally administered iron chelator to treat chronic iron overload in patients with transfusion-dependent anemias. "( Pharmacokinetics, metabolism, and disposition of deferasirox in beta-thalassemic patients with transfusion-dependent iron overload who are at pharmacokinetic steady state.
Bruin, GJ; Glaenzel, U; Hazell, K; Porter, JB; Sechaud, R; Waldmeier, F; Warrington, S, 2010
)
2.06
"Deferasirox (Exjade) is a once-daily, oral iron chelator approved for the treatment of transfusional iron overload. "( Cystatin C levels in patients with beta-thalassemia during deferasirox treatment.
Delaporta, P; Goussetis, E; Hantzi, E; Kattamis, A; Ladis, V; Margeli, A; Papassotiriou, I; Sergounioti, A, 2010
)
2.05
"Deferasirox is an oral alternative to the well studied but inconvenient deferroxamine therapy."( A method to measure deferasirox in plasma using HPLC coupled with MS/MS detection and its potential application.
Bouchet, S; Chauzit, E; Mahon, FX; Micheau, M; Molimard, M; Moore, N; Titier, K, 2010
)
1.41
"Deferasirox is an orally administered iron chelator which is effective on iron overloaded patients with transfusion-dependent anemia."( Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia.
Christoulas, D; Dimopoulou, M; Douskou, M; Mpoutou, E; Plata, E; Sioni, A; Terpos, E; Voskaridou, E, 2011
)
2.53
"Deferasirox is a recently approved oral iron chelator for treatment of patients with transfusion-related iron overload. "( Acquired proximal renal tubular dysfunction in β-thalassemia patients treated with deferasirox.
Barzilai-Birenbaum, S; Krause, I; Pazgal, I; Stark, P; Tamary, H; Yacobovich, J; Yaniv, I, 2010
)
2.03
"Deferasirox is an oral iron-chelating agent taken once-daily by patients with transfusion-dependent iron overload. "( Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia.
Chang, HH; Chang, TT; Chiou, SS; Jou, ST; Liao, YM; Lin, DT; Lin, KH; Lin, PC; Lu, MY; Yang, YL, 2011
)
2.07
"Deferasirox (DFRA) is a new approved oral iron chelator. "( Fanconi syndrome in a patient with β-thalassemia major after using deferasirox for 27 months.
Cheng, CH; Lo, FS; Wei, HY; Yang, CP, 2011
)
2.05
"Deferasirox is a new oral iron chelator. "( [Deferasirox--a new oral iron chelator--review].
Chen, BA; Gao, C; Wang, T, 2010
)
2.71
"Deferasirox is a relatively new oral iron chelator and experience of its use in children is limited."( Safety and efficacy of deferasirox in multitransfused Indian children with β-thalassaemia major.
Chandra, J; Chaudhary, H; Dutta, AK; Pemde, H; Singh, V, 2011
)
1.4
"Deferasirox is a new oral chelator with high iron-binding potency and selectivity."( Deferasirox protects against iron-induced hepatic injury in Mongolian gerbil.
Al-Rousan, RM; Blough, ER; Katta, A; Laurino, J; Rice, KM; Triest, WE; Walker, EM; Wu, M, 2011
)
2.53
"Deferasirox (DFX) is an orally administered iron chelator which has been highly efficient in the treatment of secondary iron overload."( Clinical management of gastrointestinal disturbances in patients with myelodysplastic syndromes receiving iron chelation treatment with deferasirox.
Almeida, A; Angelucci, E; Beris, P; Guerci-Bresler, A; Hofmann, WK; Macwhannell, A; Muus, P; Nolte, F; Porter, J; Schumann, C; Selleslag, D; Sliwa, T; Xicoy, B, 2011
)
1.29
"Deferasirox (DFX) is an oral iron chelator that is used worldwide for the treatment of iron overload. "( Interference of deferasirox with assays for serum iron and serum unsaturated iron binding capacity during iron chelating therapy.
Fujiya, M; Ikuta, K; Ito, S; Kohgo, Y; Sasaki, K; Tanaka, H; Torimoto, Y, 2011
)
2.16
"Deferasirox (Exjade®) is an oral tridentate iron chelator used for reducing iron overload."( Deferasirox: pharmacokinetics and clinical experience.
Campus, S; Galanello, R; Origa, R, 2012
)
2.54
"Deferasirox (DFX) is a relatively new iron chelator approved by the US Food and Drug Administration for treatment of children >2 years of age. "( Efficacy of deferasirox in North Indian β-thalassemia major patients: a preliminary report.
Bansal, D; Marwaha, RK; Panigrahi, I; Vaidya, PC, 2012
)
2.2
"Deferasirox is an oral, easily applicable, and effective iron chelator; baseline hepatotoxicity and nephrotoxicity may increase the development of toxic side effects in children with FAA."( Deferasirox therapy in children with Fanconi aplastic anemia.
Azik, FM; Culha, V; Kara, A; Karakurt, N; Ozkasap, S; Tavil, B; Tunç, B; Yarali, N, 2012
)
2.54
"Deferasirox (DFX) is an oral iron chelator with an established dose-dependent efficacy in transfusion-related iron overload. "( Deferasirox-induced renal impairment in children: an increasing concern for pediatricians.
Cochat, P; Dubourg, L; Hadj-Aïssa, A; Laurain, C; Pondarré, C; Ranchin, B; Sigaudo-Roussel, D, 2012
)
3.26
"Deferasirox is a new oral iron chelator used to treat transfusional iron overload. "( First report of drug-induced esophagitis by deferasirox.
Araki, H; Hara, T; Moriwaki, H; Oyama, M; Tsurumi, H; Yoshikawa, T, 2012
)
2.08
"Deferasirox is a newer chelation therapy that is taken orally once daily."( Lifetime cost-utility analyses of deferasirox in beta-thalassaemia patients with chronic iron overload: a UK perspective.
Chandiwana, D; Karnon, J; Tolley, K; Vieira, J, 2012
)
1.38
"Deferasirox is an iron chelating agent for the treatment of transfusional iron over load in patients with chronic anemia. "( Dried blood spot analysis of an iron chelator--deferasirox and its potential application to therapeutic drug monitoring.
Ajjala, DR; Aleti, R; Kandikere, V; Nirogi, R; Srikakolapu, S; Vurimindi, H, 2012
)
2.08
"Deferasirox is an orally effective iron (Fe) chelator currently used for the treatment of iron-overload disease and has been implemented as an alternative to the gold standard chelator, desferrioxamine (DFO). "( The iron chelator, deferasirox, as a novel strategy for cancer treatment: oral activity against human lung tumor xenografts and molecular mechanism of action.
Ford, SJ; Jansson, PJ; Kalinowski, DS; Kovacevic, Z; Lovejoy, DB; Lui, GY; Obeidy, P; Richardson, DR; Sharp, DM; Tselepis, C, 2013
)
2.16
"Deferasirox (DFRA) is a novel oral chelator agent for treatment of iron overload. "( Safety and efficacy of 4 years of deferasirox treatment for sickle cell disease patients.
Bekiari, E; Mainou, M; Tsapas, A; Vetsiou, E; Vlachaki, E, 2013
)
2.11
"Deferasirox (DFX) is a novel iron chelator that has been shown to have similar efficacy and safety compared with deferoxamine (DFO) in patients with β-thalassemia. "( Cost-utility analysis of oral deferasirox versus infusional deferoxamine in transfusion-dependent β-thalassemia patients.
Javanbakht, M; Karimi, M; Keshtkaran, A; Mashayekhi, A; Nuri, B; Salavati, S, 2013
)
2.12
"Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. "( A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia.
Agaoglu, L; Alberti, D; Athanassiou-Metaxa, M; Aydinok, Y; Bejaoui, M; Cappellini, MD; Capra, M; Coates, T; Cohen, A; Drelichman, G; Fattoum, S; Galanello, R; Giardina, P; Janka-Schaub, G; Kattamis, A; Kilinc, Y; Kourakli-Symeonidis, A; Magnano, C; Marks, P; Olivieri, N; Opitz, H; Perrotta, S; Piga, A; Porter, J; Ressayre-Djaffer, C; Thuret, I; Verissimo, M; Vermylen, C, 2006
)
2.1
"Deferasirox is an orally active iron chelator. "( Deferasirox--an oral agent for chronic iron overload.
Hagemann, TM; Vanorden, HE, 2006
)
3.22
"Deferasirox (ICL670) is a novel once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. "( Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with beta-thalassemia major.
Alberti, D; Belleli, R; Bertrand, Y; Bordone, E; Forni, GL; Foschini, ML; Galanello, R; Hewson, N; Lavagetto, A; Leoni, G; Longo, F; Maseruka, H; Piga, A; Sechaud, R; Zappu, A, 2006
)
2.06
"Deferasirox is a once-daily, oral iron chelator developed for treating transfusional iron overload. "( A randomised comparison of deferasirox versus deferoxamine for the treatment of transfusional iron overload in sickle cell disease.
Alberti, D; Bernaudin, F; Coates, T; Eckman, J; Files, B; Fischer, R; Forni, GL; Fung, E; Hassell, K; Holland, J; Kelly, P; Lane, P; Marks, P; Mueller, BU; Okpala, I; Onyekwere, O; Porter, J; Ressayre-Djaffer, C; Swerdlow, P; Vichinsky, E; Wilson, F, 2007
)
2.08
"Deferasirox is an oral tridentate chelator that mobilizes iron stores by binding selectively to the ferric form of iron."( Deferasirox.
Stumpf, JL, 2007
)
2.5
"Deferasirox is a recently approved once-daily oral iron chelator that has been shown to reduce liver iron concentrations and serum ferritin levels to a similar extent as infusional deferoxamine."( Cost effectiveness of once-daily oral chelation therapy with deferasirox versus infusional deferoxamine in transfusion-dependent thalassaemia patients: US healthcare system perspective.
Baladi, JF; Coates, TD; Delea, TE; Phatak, PD; Sofrygin, O; Thomas, SK, 2007
)
2.02
"Deferasirox (DSX) is a recently developed oral chelator which shows good efficacy and tolerability in patients with transfusional hemosiderosis due to various underlying disorders."( Recent developments in iron chelation therapy.
Cario, H; Janka-Schaub, G; Janssen, G; Jarisch, A; Kohne, E; Strauss, G,
)
0.85
"Deferasirox is an orally administered iron chelator that has been approved for use in the United States, Switzerland, and other countries."( Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia.
Abetz, L; Abish, S; Agaoglu, L; Baladi, JF; Bejaoui, M; Cappellini, MD; Cario, H; Coates, T; Ferster, A; Girot, R; Jeng, M; Lai, ME; Loggetto, S; Mangiagli, A; Opitz, H; Porter, J; Ressayre-Djaffer, C; Rofail, D; Strauss, G; Vichinsky, E; Watman, N; Zoumbos, N, 2007
)
1.29
"Deferasirox is a new orally effective iron chelator which has been shown to be non-inferior to deferoxamine in clinical trials."( Current status of iron overload and chelation with deferasirox.
Choudhry, VP; Naithani, R, 2007
)
1.31
"Deferasirox is a new tridentate oral iron chelator developed by computer remodeling recently approved by FDA for children above 2 years. "( Deferasirox: the new oral iron chelator.
Dubey, AP; Parakh, A; Sudha, S, 2007
)
3.23
"Deferasirox (Exjade) is an oral, once-daily iron chelator widely approved for the treatment of transfusional chronic iron overload. "( Deferasirox : a review of its use in the management of transfusional chronic iron overload.
Keam, SJ; Keating, GM; Yang, LP, 2007
)
3.23
"Deferasirox is an oral iron-chelating agent with favorable pharmacokinetics, including a long half-life allowing continuous 24-hour chelation with once-daily dosing."( Novel treatment options for transfusional iron overload in patients with myelodysplastic syndromes.
Goldberg, SL, 2007
)
1.06
"Deferasirox is a new once-daily oral agent for iron overload that was approved by the US Food and Drug Administration in November 2005."( Deferasirox for transfusion-related iron overload: a clinical review.
Lindsey, WT; Olin, BR, 2007
)
2.5

Effects

Deferasirox monotherapy has a good safety profile and effectively chelates total body iron. The most common adverse events reported in the THALASSA trial were related to mild to moderate gastrointestinal disorders.

Deferasirox has been studied in >700 adult and pediatric patients who had transfusion-related iron overload and underlying thalassemia, sickle cell anemia, myelodysplastic syndrome, Diamond-Blackfan syndrome. The most common adverse events reported in the THALASSA trial were related to mild to moderate gastrointestinal disorders.

ExcerptReferenceRelevance
"Deferasirox has an acceptable tolerability profile, with the most common adverse events reported in the THALASSA trial being related to mild to moderate gastrointestinal disorders."( Deferasirox: a review of its use for chronic iron overload in patients with non-transfusion-dependent thalassaemia.
Plosker, GL; Shirley, M, 2014
)
2.57
"Deferasirox monotherapy has a good safety profile and effectively chelates total body iron. "( Efficacy and safety of deferasirox for reducing total body and cardiac iron in thalassemia.
Ahmed, J; Jankharia, B; Krishnan, P; Merchant, R, 2012
)
2.13
"Deferasirox has a manageable safety profile with favorable patient satisfaction reports."( Novel treatment options for transfusional iron overload in patients with myelodysplastic syndromes.
Goldberg, SL, 2007
)
1.06
"Deferasirox has been reported to inhibit NF-κB activity and suppress phosphorylation of the MAPK pathway."( Identification and overcoming rituximab resistance in diffuse large B-cell lymphoma using next-generation sequencing.
Choi, CW; Jeon, MJ; Kim, DS; Yu, ES, 2023
)
1.63
"Deferasirox has shown antioxidant activity in all three model systems, causing substantial reduction in the rate of oxidation and oxidative damage."( Antioxidant Activity of Deferasirox and Its Metal Complexes in Model Systems of Oxidative Damage: Comparison with Deferiprone.
Kichigina, LA; Kontoghiorghes, GJ; Polyakov, NE; Selyutina, OY; Timoshnikov, VA, 2021
)
1.65
"Deferasirox has nephrotoxic effects in the context of chronic therapy. "( A case of Fanconi syndrome due to a deferasirox overdose and a trial of plasmapheresis.
Powell, JL; Shah, L; Zaritsky, JJ, 2017
)
2.17
"Deferasirox has long-term efficacy and safety in children with TDT and SCA, although higher doses (≥30 mg/kg/d) may be required to achieve iron balance."( Deferasirox in children with transfusion-dependent thalassemia or sickle cell anemia: A large cohort real-life experience from Turkey (REACH-THEM).
Akın, M; Akkaynak, D; Antmen, B; Apak, H; Aral, YZ; Aydogan, G; Ayhan, AC; Biner, B; Çalışkan, Ü; Eren, TG; Ertem, M; Gümrük, F; İrken, G; Işık Balcı, Y; Karakaş, Z; Karasu, G; Kazancı, EG; Koç, A; Koçak, Ü; Küpesiz, OA; Kurtoğlu, E; Meral Güneş, A; Oktay, G; Oymak, Y; Patıroğlu, T; Salcioglu, Z; Şaşmaz, İ; Söker, M; Timur, Ç; Tunç, B; Türkkan, E; Ünal, S; Uygun, V; Vergin, C; Yeşilipek, MA; Yıldırmak, Y, 2019
)
3.4
"Deferasirox (DFX) has recently been used to treat thalassemia with iron overload; however, its long-term effectiveness and safety await multi-year studies. "( Effectiveness and Safety of Deferasirox in Thalassemia with Iron Overload: A Meta-Analysis.
Chen, G; Dou, H; Qin, Y; Zhao, Y, 2019
)
2.25
"Oral deferasirox has been licensed for use in children aged over six years who receive frequent blood transfusions and in children aged two to five years who receive infrequent blood transfusions. In the absence of randomised controlled trials with long-term follow up, there is no compelling evidence to change this conclusion.Worsening iron deposition in the myocardium in patients receiving desferrioxamine alone would suggest a change of therapy by intensification of desferrioxamine treatment or the use of desferrioxamine and deferiprone combination therapy.Adverse events are increased in patients treated with deferiprone compared with desferrioxamine and in patients treated with combined deferiprone and desferrioxamine compared with desferrioxamine alone."( Desferrioxamine mesylate for managing transfusional iron overload in people with transfusion-dependent thalassaemia.
Brunskill, SJ; Chowdhury, O; Doree, C; Fisher, SA; Gooding, S; Roberts, DJ, 2013
)
0.84
"Deferasirox has an acceptable tolerability profile, with the most common adverse events reported in the THALASSA trial being related to mild to moderate gastrointestinal disorders."( Deferasirox: a review of its use for chronic iron overload in patients with non-transfusion-dependent thalassaemia.
Plosker, GL; Shirley, M, 2014
)
2.57
"Deferasirox (DFX) has been licensed for iron chelation in patients with sickle cell disease (SCD), but there is limited data on its long-term efficacy and safety in children. "( Deferasirox for iron chelation in multitransfused children with sickle cell disease; long-term experience in the East London clinical haemoglobinopathy network.
Gadong, N; Hemmaway, C; Kaya, B; Newell, H; Simmons, A; Telfer, P; Tsouana, E; Whitmarsh, S, 2015
)
3.3
"Deferasirox has been identified to cause fatal gastrointestinal hemorrhages, renal tubulopathy, hepatic and renal failure, alopecia and anaphylactic reactions in addition to previously reported fatal or serious toxic side effects such as agranulocytosis, renal and hepatic toxicity, skin rash and gastric intolerance."( Introduction of higher doses of deferasirox: better efficacy but not effective iron removal from the heart and increased risks of serious toxicities.
Kontoghiorghes, GJ, 2010
)
1.37
"Deferasirox monotherapy has a good safety profile and effectively chelates total body iron. "( Efficacy and safety of deferasirox for reducing total body and cardiac iron in thalassemia.
Ahmed, J; Jankharia, B; Krishnan, P; Merchant, R, 2012
)
2.13
"Deferasirox has been studied in patients older than 2 years and appears to be safe, with the most common adverse effects reported being mild, transient nausea, gastrointestinal disturbances, and rash."( Deferasirox--an oral agent for chronic iron overload.
Hagemann, TM; Vanorden, HE, 2006
)
2.5
"Deferasirox has been studied in >700 adult and pediatric patients who had transfusion-related iron overload and underlying thalassemia, sickle cell anemia, myelodysplastic syndrome, Diamond-Blackfan syndrome, or another rare anemia."( Deferasirox.
Stumpf, JL, 2007
)
2.5
"Deferasirox has a manageable safety profile with favorable patient satisfaction reports."( Novel treatment options for transfusional iron overload in patients with myelodysplastic syndromes.
Goldberg, SL, 2007
)
1.06
"Deferasirox has exhibited high potency and a clinically manageable safety profile in preclinical models and in an extensive clinical program."( Effect of food, type of food, and time of food intake on deferasirox bioavailability: recommendations for an optimal deferasirox administration regimen.
Alberti, D; Balez, S; Belleli, R; Cappellini, MD; Dutreix, C; Ford, JM; Forni, GL; Galanello, R; Origa, R; Piga, A; Rivière, GJ; Séchaud, R; Zappu, A, 2008
)
1.31
"Deferasirox (ICL670) has been shown to have rapid accessibility to intracellular labile iron. "( Effect of deferasirox (ICL670) on arterial function in patients with beta-thalassaemia major.
Chan, GC; Cheung, YF; Ha, SY, 2008
)
2.19

Treatment

Deferasirox is a new treatment of iron overload that is administered orally once-a-day. It was not associated with changes in renal or liver function, complete blood count, or transplant immunosuppressive levels.

ExcerptReferenceRelevance
"Deferasirox treatment was not associated with changes in renal or liver function, complete blood count, or transplant immunosuppressive levels."( Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
Andes, D; Anglim, A; Bonilla, H; Ibrahim, AS; Mathisen, GE; Perez, M; Spellberg, B; Walsh, TJ, 2009
)
1.35
"Deferasirox treatment reduced the level of pro inflammatory cytokines (IL-1β, IL-6, TNF-α and INF-γ)."( Deferasirox alleviates DSS-induced ulcerative colitis in mice by inhibiting ferroptosis and improving intestinal microbiota.
Chunyu, W; Gao, X; Liu, S; Peng, M; Ran, L; Sun, L; Wan, J; Wang, Y; Wu, Y; Yang, K; Yang, Y; Yin, M, 2023
)
3.07
"Deferasirox is a new treatment of iron overload that is administered orally once-a-day, resulting in better acceptance in patients. "( Hyperchloraemic metabolic acidosis induced by the iron chelator deferasirox: a case report and review of the literature.
Bianchetti, MG; Brazzola, P; Dell'Orto, VG, 2013
)
2.07
"Deferasirox treatment may be clinically beneficial both by reducing iron overload and by improving hematopoiesis in patients with PRCA."( Deferasirox treatment improved hematopoiesis and led to complete remission in a patient with pure red cell aplasia.
Ando, K; Kojima, M; Machida, S; Miyamoto, M; Moriuchi, M; Ohbayashi, Y; Sato, A, 2013
)
2.55
"Deferasirox is a standard treatment for chronic transfusional iron overload. "( Proximal muscular atrophy and weakness: An unusual adverse effect of deferasirox iron chelation therapy.
Albert, MH; Blaschek, A; Gerstl, L; Huetker, S; Müller-Felber, W; Teusch, V; Vill, K,
)
1.81
"Deferasirox treatment or phlebotomy reduces iron burden in pediatric patients with β- thalassemia major post-HSCT, with a manageable safety profile."( One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation.
Abbas, HA; Cappellini, MD; Inati, A; Kahale, M; Koussa, S; Musallam, KM; Nasr, TA; Porter, JB; Sbeiti, N; Taher, AT, 2017
)
2.13
"Deferasirox treatment was taken post iron loading and was given at 100 mg/kg/day p.o for 1 or 3 months."( Deferasirox removes cardiac iron and attenuates oxidative stress in the iron-overloaded gerbil.
Al-Rousan, RM; Blough, ER; Gutta, AK; Kakarla, SK; Laurino, JP; Paturi, S; Walker, EM, 2009
)
2.52
"Deferasirox chelation treatment regimen was well tolerated and adherence to the regimen was good."( Long-term chelation therapy with deferasirox: effects on cardiac iron overload measured by T2* MRI.
Borsellino, Z; Cuccia, L; Gagliardotto, F; Marocco, MR; Ruffo, GB; Tarantino, R, 2010
)
1.36
"Deferasirox treatment decreased cardiac iron levels by 37% (P < 0.05), and this was associated with decreases in the number of TUNEL-positive cells."( Deferasirox decreases age-associated iron accumulation in the aging F344XBN rat heart and liver.
Arvapalli, RK; Blough, ER; Gadde, MK; Kakarla, SK; Katta, A; Laurino, JP; Paturi, S; Rice, KM; Walker, EM; Wehner, P; Wu, M, 2010
)
2.52
"Deferasirox treatment also was associated with reduced hepatic protein oxidation, superoxide abundance, and cell death."( Deferasirox protects against iron-induced hepatic injury in Mongolian gerbil.
Al-Rousan, RM; Blough, ER; Katta, A; Laurino, J; Rice, KM; Triest, WE; Walker, EM; Wu, M, 2011
)
2.53
"Deferasirox treatment (25 mg/kg/day) was initiated when serum ferritin levels increased to more than 1,000 ng/ml during induction chemotherapy."( Iron chelation treatment with deferasirox prior to high-dose chemotherapy and autologous stem cell transplantation may reduce the risk of hepatic veno-occlusive disease in children with high-risk solid tumors.
Cho, EJ; Chueh, HW; Kim, JY; Koo, HH; Lee, SH; Sung, KW; Yoo, KH, 2012
)
1.39
"Deferasirox treatment was completed as scheduled in 11 (61.1%) of them without dose reduction or discontinuation."( Iron chelation treatment with deferasirox prior to high-dose chemotherapy and autologous stem cell transplantation may reduce the risk of hepatic veno-occlusive disease in children with high-risk solid tumors.
Cho, EJ; Chueh, HW; Kim, JY; Koo, HH; Lee, SH; Sung, KW; Yoo, KH, 2012
)
1.39
"Deferasirox treatment during induction chemotherapy reduces the frequency of VOD during HDCT/autoSCT. "( Iron chelation treatment with deferasirox prior to high-dose chemotherapy and autologous stem cell transplantation may reduce the risk of hepatic veno-occlusive disease in children with high-risk solid tumors.
Cho, EJ; Chueh, HW; Kim, JY; Koo, HH; Lee, SH; Sung, KW; Yoo, KH, 2012
)
2.11
"Deferasirox treatment for 3 or more years reversed or stabilized liver fibrosis in 83% of patients with iron-overloaded β-thalassemia. "( Improvement in liver pathology of patients with β-thalassemia treated with deferasirox for at least 3 years.
Brissot, P; Cappellini, MD; Deugnier, Y; Giannone, V; Griffel, L; Porter, JB; Ropert, M; Turlin, B; Zhang, Y, 2011
)
2.04
"The deferasirox treatment reduced pretreatment ferritin levels below the level of 1,000 ng/mL in a median period of 94 days, and these data were found to be statistically significant (p < 0.05)."( Efficacy and safety of oral deferasirox treatment in the posttransplant period for patients who have undergone allogeneic hematopoietic stem cell transplantation (alloHSCT).
Bahcebasi, S; Cetin, M; Deniz, K; Eser, B; Kaynar, L; Kurnaz, F; Ozturk, A; Pala, C; Sivgin, S; Unal, A; Uzer, E, 2012
)
1.15
"Deferasirox treatment reduced liver iron content 51%."( Deferasirox and deferiprone remove cardiac iron in the iron-overloaded gerbil.
Aguilar, MI; Gonzalez, I; Moats, R; Nelson, M; Nick, H; Otto-Duessel, M; Shimada, H; Wood, JC, 2006
)
2.5
"Deferasirox treatment also enhanced the host inflammatory response to mucormycosis."( The iron chelator deferasirox protects mice from mucormycosis through iron starvation.
Edwards, JE; French, SW; Fu, Y; Gebermariam, T; Husseiny, MI; Ibrahim, AS; Lin, L; Schwartz, J; Skory, CD; Spellberg, BJ, 2007
)
1.39
"Treatment with deferasirox for up to 2 years yielded a sustained reduction in iron burden, with a clinically manageable safety profile."( Deferasirox for the treatment of iron overload in non-transfusion-dependent thalassemia.
Cappellini, MD; Taher, AT; Temraz, S, 2013
)
2.17
"Treatment with deferasirox resulted in a statistically significant (p< 0.01) decrease in plasma ferritin levels as compared to the control group (823 ng/ml ± 56 and 1220 ng/ml ±114, respectively)."( Deferasirox limits cartilage damage following haemarthrosis in haemophilic mice.
Biesma, DH; Coeleveld, K; Lafeber, FP; Mastbergen, SC; Nieuwenhuizen, L; Roosendaal, G; Schutgens, RE, 2014
)
2.18
"Treatment with deferasirox produced a significant reduction in median serum ferritin levels in chelation-naïve patients with MDS from 2679 to 2000 ng/mL (P = 0.0002) and a pronounced decrease in prechelated patients with MDS from 2442 to 2077 ng/mL (P = 0.06)."( Deferasirox treatment of iron-overloaded chelation-naïve and prechelated patients with myelodysplastic syndromes in medical practice: results from the observational studies eXtend and eXjange.
Blumenstengel, K; Gattermann, N; Germing, U; Goebeler, M; Groschek, M; Jarisch, A; Junkes, A; Leismann, O; Losem, C; Procaccianti, M; Schlag, R, 2012
)
2.16
"Treatment with deferasirox significantly improves left ventricular function."( Cardiac iron removal and functional cardiac improvement by different iron chelation regimens in thalassemia major patients.
Brevi, F; Cappellini, MD; Cassinerio, E; Graziadei, G; Milazzo, A; Pattoneri, P; Pedrotti, P; Roghi, A; Zanaboni, L, 2012
)
0.72
"Treatment with deferasirox alone ameliorated anaemia and normalized the serum iron levels and hepatic iron concentration in the animals."( Iron chelation therapy as a treatment for Pythium insidiosum in an animal model.
Alves, SH; Fighera, RA; Flores, MM; Pilotto, MB; Santurio, JM; Weiblen, C; Wolkmer, P; Zanette, RA, 2013
)
0.73

Toxicity

A record 4113 fatalities were reported in 2012. Of 84 patients who discontinued deferasirox therapy, 22 died during the trial. 28 withdrew due to an adverse event (AE) DeferasiroX is generally well tolerated. Most common adverse events were gastrointestinal disturbances and ras.

ExcerptReferenceRelevance
" Primary objectives included assessment of safety and tolerability (measured by adverse events and clinical laboratory monitoring), pharmacokinetics (measured as drug and drug-iron complex), and cumulative net iron excretion (measured by faecal and urine output minus food input)."( Effectiveness and safety of ICL670 in iron-loaded patients with thalassaemia: a randomised, double-blind, placebo-controlled, dose-escalation trial.
Alberti, D; Anderson, JR; Giardina, PJ; Grady, RW; Krebs-Brown, AJ; Nathan, DG; Neufeld, EJ; Nisbet-Brown, E; Olivieri, NF; Séchaud, R; Sizer, KC, 2003
)
0.32
" This article speculates that cyclosporine eye drops would also be useful for any disease causing ectropion or eclabion of the eye as well as toxic epidermal necrolysis-related eye pathology (in particular corneal scarring)."( A review of deferasirox, bortezomib, dasatinib, and cyclosporine eye drops: possible uses and known side effects in cutaneous medicine.
Scheinfeld, N, 2007
)
0.72
" In the interim, 1-year data show that deferasirox was well tolerated, with generally infrequent and mild adverse events."( A safety, pharmacokinetic and pharmacodynamic investigation of deferasirox (Exjade, ICL670) in patients with transfusion-dependent anemias and iron-overload: a Phase I study in Japan.
Hata, T; Ishikawa, T; Kato, J; Kondo, M; Miyazawa, K; Mori, H; Nakao, S; Ohyashiki, K; Omine, M; Ozawa, K; Rojkjaer, L; Taniguchi, J; Tanii, H; Tatsumi, Y; Urabe, A, 2008
)
0.85
" However, despite these fatalities it would appear that there is no regular monitoring of such toxicities or of effects such as the accumulation of toxic metals."( Update on toxicity and efficacy aspects of treatment with deferasirox and its implication on the morbidity and mortality of transfused iron loaded patients.
Kontoghiorghes, GJ, 2008
)
0.59
" Deferasirox is generally well tolerated, with the most common adverse events being gastrointestinal disturbances and rash."( Long-term efficacy and safety of deferasirox.
Cappellini, MD, 2008
)
1.54
" Drug-related adverse events were mostly mild to moderate and resolved without discontinuing treatment."( Efficacy and safety of deferasirox, an oral iron chelator, in heavily iron-overloaded patients with beta-thalassaemia: the ESCALATOR study.
Al Jefri, A; Al Zir, K; Daar, S; El-Beshlawy, A; Elalfy, MS; Habr, D; Hmissi, A; Kriemler-Krahn, U; Taher, A, 2009
)
0.66
" The only adverse effects attributable to deferasirox were rashes in two patients."( Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
Andes, D; Anglim, A; Bonilla, H; Ibrahim, AS; Mathisen, GE; Perez, M; Spellberg, B; Walsh, TJ, 2009
)
0.9
" The adverse event profile in patients who received deferasirox doses of >30 mg/kg per d was consistent with previously published data."( Efficacy and safety of deferasirox doses of >30 mg/kg per d in patients with transfusion-dependent anaemia and iron overload.
Cappellini, MD; Clark, J; Galanello, R; Habr, D; Lawniczek, T; Piga, A; Porter, JB; Taher, A; Vichinsky, E, 2009
)
0.91
"Therapy with deferasirox is safe in paediatric patients with thalassaemia major."( Safety and efficacy of deferasirox in multitransfused Indian children with β-thalassaemia major.
Chandra, J; Chaudhary, H; Dutta, AK; Pemde, H; Singh, V, 2011
)
1.05
" The most common reasons for discontinuation were withdrawal of consent (23·8%), lost to follow-up (9·2%) and adverse events (AEs) (7·6%)."( Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.
Bernaudin, F; Coates, T; Deng, W; Forni, GL; Gardner, R; Giannone, V; Griffel, L; Hassell, K; Heeney, MM; Inusa, B; Kutlar, A; Lane, P; Mathias, L; Porter, J; Tebbi, C; Vichinsky, E; Wilson, F, 2011
)
0.65
"7%) discontinued because of adverse events."( Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up.
Agaoglu, L; Aydinok, Y; Bejaoui, M; Canatan, D; Cappellini, MD; Capra, M; Clark, J; Cohen, A; Dong, V; Drelichman, G; Economou, M; Fattoum, S; Griffel, L; Kattamis, A; Kilinc, Y; Perrotta, S; Piga, A; Porter, JB, 2011
)
0.69
" The DFX treatment-related adverse events were observed."( [Curative effects and safety of deferasirox in treatment of iron overload in children with β-thalassemia major].
Chen, GF; Chen, JJ; Gao, HY; Li, CG; Li, Q, 2011
)
0.65
" Serum liver transaminase elevation was the most common adverse effect, followed by non-progressive elevation in serum creatinine level."( [Curative effects and safety of deferasirox in treatment of iron overload in children with β-thalassemia major].
Chen, GF; Chen, JJ; Gao, HY; Li, CG; Li, Q, 2011
)
0.65
" Serum liver transaminase elevation and non-progressive elevation in serum creatinine level are major adverse effects in DFX treatment."( [Curative effects and safety of deferasirox in treatment of iron overload in children with β-thalassemia major].
Chen, GF; Chen, JJ; Gao, HY; Li, CG; Li, Q, 2011
)
0.65
" Adverse effect of DFX included diarrhea, maculopapular skin rash and transient proteinuria that necessitated temporary stoppage of medication."( Efficacy and safety of deferasirox for reducing total body and cardiac iron in thalassemia.
Ahmed, J; Jankharia, B; Krishnan, P; Merchant, R, 2012
)
0.69
" The most common adverse effects were nausea and vomiting, which occurred in three of the patients (13%)."( Efficacy and safety of oral deferasirox treatment in the posttransplant period for patients who have undergone allogeneic hematopoietic stem cell transplantation (alloHSCT).
Bahcebasi, S; Cetin, M; Deniz, K; Eser, B; Kaynar, L; Kurnaz, F; Ozturk, A; Pala, C; Sivgin, S; Unal, A; Uzer, E, 2012
)
0.67
" Serum ferritin (SF) was measured monthly, and safety assessment included monitoring of adverse events during treatment and of liver and renal parameters."( Deferasirox treatment for myelodysplastic syndromes: "real-life" efficacy and safety in a single-institution patient population.
Alimena, G; Breccia, M; Colafigli, G; Federico, V; Finsinger, P; Latagliata, R; Loglisci, G; Petrucci, L; Salaroli, A; Santopietro, M; Serrao, A, 2012
)
1.82
" The most common adverse events (≥ 10 % of all patients) with suspected drug relationship were diarrhea (n = 25, 46 %), nausea (n = 13, 24 %), upper abdominal pain (n = 8, 15 %), serum creatinine increase (n = 16, 30 %) and rash (n = 5, 9 %)."( Results from a 1-year, open-label, single arm, multi-center trial evaluating the efficacy and safety of oral Deferasirox in patients diagnosed with low and int-1 risk myelodysplastic syndrome (MDS) and transfusion-dependent iron overload.
Baier, M; Gattermann, N; Giagounidis, A; Haase, D; Höchsmann, B; Hofmann, WK; Junkes, A; Leismann, O; Lübbert, M; Lück, A; Nolte, F; Platzbecker, U; Schrezenmeier, H; Schumann, C; Taupitz, M, 2013
)
0.6
" Adverse events (AEs) and laboratory parameters were monitored throughout."( Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy.
Aydinok, Y; Chan, LL; El-Beshlawy, A; Elalfy, MS; Habr, D; Lee, SH; Martin, N; Porter, JB; Sutcharitchan, P; Taher, AT, 2013
)
0.7
" None of the patients presented any serious adverse effects and the treatment was well tolerated."( Safety and efficacy of 4 years of deferasirox treatment for sickle cell disease patients.
Bekiari, E; Mainou, M; Tsapas, A; Vetsiou, E; Vlachaki, E, 2013
)
0.67
" Plasma concentration observed at 15 mg/kg was well above that expected for this dose (40-50 μmol/L), although no adverse clinical events were observed."( Pharmacokinetics and safety of deferasirox in subjects with chronic kidney disease undergoing haemodialysis.
Batty, KT; Ferrari, P; Maker, GL; Olynyk, JK; Siva, B; Trengove, RD, 2013
)
0.68
"A record 4113 fatalities were reported in 2012 in a postmarketing surveillance of patients treated with deferasirox, despite warnings of life-threatening toxic side effects, and the need for regular monitoring and prophylactic measures."( A record number of fatalities in many categories of patients treated with deferasirox: loopholes in regulatory and marketing procedures undermine patient safety and misguide public funds?
Kontoghiorghes, GJ, 2013
)
0.84
" The Fisher's Exact test was used to compare the frequency of subjects who experienced at least one SCA-related adverse event (AE) or serious adverse event (SAE) in each arm and to compare event rates."( Pain and other non-neurological adverse events in children with sickle cell anemia and previous stroke who received hydroxyurea and phlebotomy or chronic transfusions and chelation: results from the SWiTCH clinical trial.
Alvarez, O; Aygun, B; Bonner, M; Flanagan, J; Lockhart, A; Miller, ST; Mueller, BU; Owen, W; Schultz, W; Scott, JP; Ware, RE; Yovetich, NA, 2013
)
0.39
" Adverse events (AEs) secondary to drug administration were reported in 26."( Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: two-year results including pharmacokinetics and concomitant hydroxyurea.
Barrette, S; Files, B; Habr, D; Minniti, CP; Torres, M; Vichinsky, E; Zhang, Y, 2013
)
0.7
" Two independent authors assessed data from extracted randomized trials for efficacy and safety in the measurements of serum ferritin (SF), live iron concentration (LIC), myocardial iron content (MIC), left ventricular ejection fraction (LVEF) and adverse events (AEs)."( Comparative efficacy and safety of deferoxamine, deferiprone and deferasirox on severe thalassemia: a meta-analysis of 16 randomized controlled trials.
Huang, L; Jiang, H; Xia, S; Zhang, W, 2013
)
0.63
"Findings indicated that the most effective and safe iron chelators remains to be proven, and further large-scale, long-term studies are needed."( Comparative efficacy and safety of deferoxamine, deferiprone and deferasirox on severe thalassemia: a meta-analysis of 16 randomized controlled trials.
Huang, L; Jiang, H; Xia, S; Zhang, W, 2013
)
0.63
" The most common adverse events were skin rash and gastrointestinal disturbance."( Efficacy and safety of deferasirox in β-thalassemia major patients in Iran: a prospective study from a single referral center in Iran.
Abdollah Gorji, F; Alavi, S; Arzanian, MT; Ebadi, M; Ghazizadeh, F; Shamsian, B, 2014
)
0.71
"Deferasirox proved as an efficient and safe chelating agent in our patients, specifically in mild to moderate iron overloaded patients."( Efficacy and safety of deferasirox in β-thalassemia major patients in Iran: a prospective study from a single referral center in Iran.
Abdollah Gorji, F; Alavi, S; Arzanian, MT; Ebadi, M; Ghazizadeh, F; Shamsian, B, 2014
)
2.16
" Of 84 patients who discontinued deferasirox therapy, 22 died during the trial, and 28 withdrew due to an adverse event (AE)."( Deferasirox for transfusion-dependent patients with myelodysplastic syndromes: safety, efficacy, and beyond (GIMEMA MDS0306 Trial).
Alimena, G; Angelucci, E; Borin, L; Caocci, G; Cilloni, D; Di Tucci, AA; Fazi, P; Fenu, S; Finelli, C; Latte, G; Molteni, A; Piciocchi, A; Quaresmini, G; Quarta, G; Rivellini, F; Salvi, F; Sanpaolo, G; Santini, V; Storti, S; Tura, S; Vallisa, D; Vignetti, M; Volpe, A; Voso, MT, 2014
)
2.13
" However, the US Food and Drug Administration issued a warning about the potential adverse events associated with DFX in 2010."( Safety of deferasirox: a retrospective cohort study on the risks of gastrointestinal, liver and renal events.
Chou, HC; Hsiao, FY; Huang, WF; Tsai, YW, 2014
)
0.8
" The risks of different adverse events were individually analyzed by Cox proportional hazards models and adjusted by age, sex, concomitant medications, and prior medical conditions."( Safety of deferasirox: a retrospective cohort study on the risks of gastrointestinal, liver and renal events.
Chou, HC; Hsiao, FY; Huang, WF; Tsai, YW, 2014
)
0.8
" More researches are warranted to evaluate the association between DFX use and potential adverse events."( Safety of deferasirox: a retrospective cohort study on the risks of gastrointestinal, liver and renal events.
Chou, HC; Hsiao, FY; Huang, WF; Tsai, YW, 2014
)
0.8
" Drug-related adverse events, reported in 17 patients (56."( Phase IV open-label study of the efficacy and safety of deferasirox after allogeneic stem cell transplantation.
Batlle, M; Belloch, V; Duarte, R; Hernández, D; Jarque, I; Jiménez, M; Jiménez, S; López, J; Rovira, M; Sampol, A; Solano, C; Valcárcel, D; Vallejo, C; Vázquez, L, 2014
)
0.65
" Nephrotoxicity is the most serious and frequent adverse effect of deferasirox treatment."( Deferasirox nephrotoxicity-the knowns and unknowns.
Díaz-García, JD; Gallegos-Villalobos, A; Gonzalez-Espinoza, L; Ortiz, A; Sanchez-Niño, MD; Villarrubia, J, 2014
)
2.08
"DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events."( Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients.
Adly, AM; Elalfy, MS; Elhenawy, YI; Samir, A; Tony, S; Wali, Y, 2015
)
0.65
" DFX was temporarily skipped for adverse events in seven patients (7."( Efficacy and safety of deferasirox estimated by serum ferritin and labile plasma iron levels in patients with aplastic anemia, myelodysplastic syndrome, or acute myeloid leukemia with transfusional iron overload.
Bae, SH; Chung, JS; Hyun, MS; Joo, YD; Kim, H; Kim, HG; Kim, IH; Kim, SH; Kim, YS; Kwon, KY; Lee, GW; Lee, HS; Lee, SM; Lee, WS; Lim, SN; Moon, JH; Park, KT; Ryoo, HM; Sohn, SK, 2015
)
0.73
" The most common investigator-reported drug-related adverse events (AEs) were increased blood creatinine (26."( Efficacy and safety of deferasirox in non-thalassemic patients with elevated ferritin levels after allogeneic hematopoietic stem cell transplantation.
Al-Ali, HK; Albrecht, S; Altamura, S; Bug, G; de Haas, K; Hubert, K; Jaekel, N; Kröger, N; Leismann, O; Lieder, K; Muckenthaler, MU; Niederwieser, D; Platzbecker, U; Stadler, M, 2016
)
0.74
" The most common adverse events (AEs) were increased serum creatinine levels (40."( [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China].
Chang, H; Hu, D; Hu, L; Huang, J; Liu, F; Nie, N; Quan, R; Shao, Y; Shi, J; Tang, X; Xiao, H; Zhang, F; Zhang, J; Zhang, L; Zheng, C; Zheng, Y; Zhou, Y, 2016
)
0.74
" The drug was well tolerated with a clinically manageable safety profile and no major adverse events."( [Efficacy and safety of deferasirox in aplastic anemia patients with iron overload: a single arm, multi-center,prospective study in China].
Chang, H; Hu, D; Hu, L; Huang, J; Liu, F; Nie, N; Quan, R; Shao, Y; Shi, J; Tang, X; Xiao, H; Zhang, F; Zhang, J; Zhang, L; Zheng, C; Zheng, Y; Zhou, Y, 2016
)
0.74
" Selected protocols using DFO, L1, and their combination can be designed for personalized chelation therapy in TI, which can effectively and safely remove all the excess toxic iron and prevent cardiac, liver, and other organ damage."( Efficacy and safety of iron-chelation therapy with deferoxamine, deferiprone, and deferasirox for the treatment of iron-loaded patients with non-transfusion-dependent thalassemia syndromes.
Kontoghiorghe, CN; Kontoghiorghes, GJ, 2016
)
0.66
" At every visit for blood transfusion, all patients were clinically assessed for any adverse effects; liver and renal functions were monitored 6-monthly."( Comparative Efficacy and Safety of Oral Iron Chelators and their Novel Combination in Children with Thalassemia.
Gomber, S; Jain, P; Narang, M; Sharma, S, 2016
)
0.43
" No significant adverse reactions were noticed in either the monotherapy or the combination group."( Comparative Efficacy and Safety of Oral Iron Chelators and their Novel Combination in Children with Thalassemia.
Gomber, S; Jain, P; Narang, M; Sharma, S, 2016
)
0.43
"Oral combination therapy of deferiprone and deferasirox appears to be an efficacious and safe modality to reduce serum ferritin in multi-transfused children with thalassemia."( Comparative Efficacy and Safety of Oral Iron Chelators and their Novel Combination in Children with Thalassemia.
Gomber, S; Jain, P; Narang, M; Sharma, S, 2016
)
0.7
" Adverse effects of deferasirox have been reported in large prospective studies."( Proximal muscular atrophy and weakness: An unusual adverse effect of deferasirox iron chelation therapy.
Albert, MH; Blaschek, A; Gerstl, L; Huetker, S; Müller-Felber, W; Teusch, V; Vill, K,
)
0.69
" The majority of patients (81%) were affected by at least 1 adverse event, with decreased renal creatinine clearance being the most frequent."( CONIFER - Non-Interventional Study to Evaluate Therapy Monitoring During Deferasirox Treatment of Iron Toxicity in Myelodysplastic Syndrome Patients with Transfusional Iron Overload.
Bruch, HR; Dencausse, Y; Heßling, J; Michl, G; Schlag, R; Schneider-Schranz, C; Schulte, C; Skorupa, A; Tesch, H; Wolf, S, 2016
)
0.67
"Deferasirox adverse effects include the following: gastrointestinal disturbance, mild elevations in serum creatinine levels and intermittent proteinuria; these events are dose-dependent and reversible with drug discontinuation, but this solution can lead to an inadequate iron chelation."( Deferasirox pharmacokinetic and toxicity correlation in β-thalassaemia major treatment.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Massano, D; Piga, A; Pirro, E, 2016
)
3.32
" The combination of drugs was well tolerated and no new adverse effects were observed."( Efficacy and Safety of Combined Oral Chelation With Deferiprone and Deferasirox in Children With β-Thalassemia Major: An Experience From North India.
Chandra, J; Dhingra, B; Jain, R; Mahto, D; Parakh, N; Sharma, S, 2017
)
0.69
" A total of 134 adverse events (AEs) were reported in 25 patients (92."( A Phase II, Multicenter, Single-Arm Study to Evaluate the Safety and Efficacy of Deferasirox after Hematopoietic Stem Cell Transplantation in Children with β-Thalassemia Major.
Dag, I; Ertem, M; Karasu, G; Kaya, Z; Kuskonmaz, BB; Ozudogru, O; Uygun, V; Yesilipek, MA, 2018
)
0.71
" Random effects model was used to generate direct, indirect and mixed treatment comparison pooled estimates for the following outcomes: serum ferritin, liver iron concentration (LIC), changes in serum ferritin, mortality, urine iron excretion, adverse events, neutropenia, agranulocytosis and number of patients withdrawing the chelating therapy."( Efficacy and safety of iron chelators in thalassemia and sickle cell disease: a multiple treatment comparison network meta-analysis and trial sequential analysis.
Sivaramakrishnan, G; Sridharan, K, 2018
)
0.48
" DFP/DFO was associated with higher LVEF, low risk of adverse events and reduced end of serum ferritin compared to DFO."( Efficacy and safety of iron chelators in thalassemia and sickle cell disease: a multiple treatment comparison network meta-analysis and trial sequential analysis.
Sivaramakrishnan, G; Sridharan, K, 2018
)
0.48
" Particular attention was paid to mortality, serum ferritin (SF), liver iron concentration (LIC), myocardial iron concentration, and adverse events (AEs)."( Effectiveness and Safety of Deferasirox in Thalassemia with Iron Overload: A Meta-Analysis.
Chen, G; Dou, H; Qin, Y; Zhao, Y, 2019
)
0.81
" We compared changes in liver and heart iron, adverse effects and other outcomes, in patients treated with deferiprone or deferasirox."( Single-center retrospective study of the effectiveness and toxicity of the oral iron chelating drugs deferiprone and deferasirox.
Gallie, BL; Olivieri, NF; Sabouhanian, A, 2019
)
0.93
" Close monitoring of the patients is necessary to detect and manage any possible adverse events."( Evaluation of Efficacy, Safety, and Satisfaction Taking Deferasirox Twice Daily Versus Once Daily in Patients With Transfusion-Dependent Thalassemia.
Ansari, S; Azarkeivan, A; Bahoush, G; Bazrafshan, A; Haghpanah, S; Jangjou, A; Karimi, M; Shahsavani, A, 2020
)
0.8
" No significant difference between the groups was shown in the occurrence of serious and drug-related adverse events."( Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial.
Bejaoui, M; Bonifazi, D; Ceci, A; Christou, S; Cosmi, C; Cuccia, L; Del Vecchio, GC; Della Pasqua, O; El-Beshlawy, A; Felisi, M; Filosa, A; Hassab, H; Kattamis, A; Kreka, M; Maggio, A; Origa, R; Putti, MC; Reggiardo, G; Sherief, L; Spino, M; Telfer, P; Tempesta, B; Tricta, F; Tsang, YC; Vitrano, A; Zaka, A, 2020
)
0.79
"In paediatric patients with transfusion-dependent haemoglobinopathies, deferiprone was effective and safe in inducing control of iron overload during 12 months of treatment."( Evaluation of the efficacy and safety of deferiprone compared with deferasirox in paediatric patients with transfusion-dependent haemoglobinopathies (DEEP-2): a multicentre, randomised, open-label, non-inferiority, phase 3 trial.
Bejaoui, M; Bonifazi, D; Ceci, A; Christou, S; Cosmi, C; Cuccia, L; Del Vecchio, GC; Della Pasqua, O; El-Beshlawy, A; Felisi, M; Filosa, A; Hassab, H; Kattamis, A; Kreka, M; Maggio, A; Origa, R; Putti, MC; Reggiardo, G; Sherief, L; Spino, M; Telfer, P; Tempesta, B; Tricta, F; Tsang, YC; Vitrano, A; Zaka, A, 2020
)
0.79
" They were also monitored monthly for any adverse effects."( Efficacy and Safety of Combined Oral Chelation with Deferiprone and Deferasirox on Iron Overload in Transfusion Dependent Children with Thalassemia - A Prospective Observational Study.
Delhikumar, CG; DivakarJose, RR; Ram Kumar, G, 2021
)
0.86
" The drugs were tolerated well without any serious adverse effects."( Efficacy and Safety of Combined Oral Chelation with Deferiprone and Deferasirox on Iron Overload in Transfusion Dependent Children with Thalassemia - A Prospective Observational Study.
Delhikumar, CG; DivakarJose, RR; Ram Kumar, G, 2021
)
0.86
" Twenty-nine patients who used an alternative chelator either alone or combined, who were not compliant to the treatment, changed the drug due to adverse reactions, and had multiple transfusions and did not complete 4 years of DFX use were excluded."( Safety and efficacy of deferasirox in patients with transfusion-dependent thalassemia: A 4-year single-center experience.
Akıcı, F; Arslantaş, E; Ayçiçek, A; Aydoğan, G; Bayram, C; Odaman Al, I; Özdemir, GN; Şalcıoğlu, Z; Uysalol, EP; Zengin Ersoy, G, 2021
)
0.93

Pharmacokinetics

The geometric mean ratios and 90% confidence intervals for Cmax and AUCtau of digoxin (with/without deferasirox) were 0. Concomitant hydroxyurea administration (n = 28) did not appear to influence the efficacy, safety (including li) or toxicity.

ExcerptReferenceRelevance
"5 to 80 mg/kg to investigate its safety, tolerability, and pharmacokinetics and to obtain preliminary information on pharmacodynamic effects."( Safety, tolerability, and pharmacokinetics of ICL670, a new orally active iron-chelating agent in patients with transfusion-dependent iron overload due to beta-thalassemia.
Alberti, D; Bigler, H; Galanello, R; Piga, A; Rouan, MC; Séchaud, R, 2003
)
0.32
" Pharmacokinetic parameters assessed at the end of each treatment period were compared using the standard statistical analysis for bioequivalence assessment."( Absence of an effect of a single-dose deferasirox on the steady-state pharmacokinetics of digoxin.
Balez, S; Belleli, R; Robeva, A; Sechaud, R, 2008
)
0.62
" Cmax, volume of distribution/bioavailability (Vd/F), and elimination half-life (t(1/2)) were not different between the groups, suggesting bioavailability as the likely discriminant."( Deferasirox pharmacokinetics in patients with adequate versus inadequate response.
Bergmann, AK; Braunstein, J; Chirnomas, D; Finkelstein, Y; Grant, FD; Neufeld, EJ; Paley, C; Pereira, L; Shannon, M; Smith, AL, 2009
)
1.8
" Absorption, distribution, metabolism, and excretion of [14C]deferasirox at pharmacokinetic steady state was investigated in five adult beta-thalassemic patients."( Pharmacokinetics, metabolism, and disposition of deferasirox in beta-thalassemic patients with transfusion-dependent iron overload who are at pharmacokinetic steady state.
Bruin, GJ; Glaenzel, U; Hazell, K; Porter, JB; Sechaud, R; Waldmeier, F; Warrington, S, 2010
)
0.86
"In addition to the pharmacokinetic and pharmacodynamic profile of deferasirox, this review examines the efficacy and safety data from pivotal studies with deferasirox in iron-overloaded patients with various anemias, including thalassemia, sickle cell disease and myelodysplastic syndromes."( Deferasirox: pharmacokinetics and clinical experience.
Campus, S; Galanello, R; Origa, R, 2012
)
2.06
" Concomitant hydroxyurea administration (n = 28) did not appear to influence the efficacy, safety (including liver and kidney function), and pharmacokinetic parameters of deferasirox."( Efficacy and safety of deferasirox compared with deferoxamine in sickle cell disease: two-year results including pharmacokinetics and concomitant hydroxyurea.
Barrette, S; Files, B; Habr, D; Minniti, CP; Torres, M; Vichinsky, E; Zhang, Y, 2013
)
0.89
" However, the pharmacokinetic (PK) disposition of DEFR and the iron-DEFR complex (Fe-[DEFR]2) in this dosing strategy is unclear."( Simultaneous Determination of Plasma Deferasirox and Deferasirox-Iron Complex Using an HPLC-UV System and Pharmacokinetics of Deferasirox in Patients With β-Thalassemia Major: Once-daily Versus Twice-daily Administration.
Chiang, PH; Kuo, PH; Lai, CW; Lin, KH; Lu, MY; Wang, N; Wu, TH; Wu, WH, 2015
)
0.69
" Deferasirox is a once-daily orally active tridentate selective iron chelator which pharmacokinetic disposition could influence treatment efficacy and toxicity."( Deferasirox pharmacokinetic evaluation in β-thalassaemia paediatric patients.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Massano, D; Piga, A; Pirro, E, 2017
)
2.81
"Concerning pharmacokinetic parameters, a higher interindividual variability was shown."( Deferasirox pharmacokinetic and toxicity correlation in β-thalassaemia major treatment.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Massano, D; Piga, A; Pirro, E, 2016
)
1.88
" Area under deferasirox concentration curve over 24h (AUC) values were determined by the mixed log-linear rule, using Kinetica software."( Deferasirox pharmacokinetics evaluation in a woman with hereditary haemochromatosis and heterozygous β-thalassaemia.
Allegra, S; Arduino, A; Cusato, J; D'Avolio, A; De Francia, S; Longo, F; Massano, D; Piga, A; Pirro, E, 2016
)
2.26
"025) and area under the concentration curve (P=0."( Effect of pharmacogenetic markers of vitamin D pathway on deferasirox pharmacokinetics in children.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Longo, F; Massano, D; Piga, A; Pirro, E, 2018
)
0.73
"-24 C>T was associated with the pharmacokinetic variability of deferasirox in Chinese subjects."( ABCC2 c.-24 C>T single-nucleotide polymorphism was associated with the pharmacokinetic variability of deferasirox in Chinese subjects.
Cao, K; Chen, X; Li, N; Lu, C; Lu, Y; Ren, G; Tan, Y; Wang, Y; Zhang, Y; Zhao, D; Zhou, J, 2020
)
1.01
" This study investigated the effects of genetic polymorphisms on the pharmacokinetics of deferasirox in healthy Chinese subjects and constructed a pharmacokinetic prediction model based on physiologic factors and genetic polymorphism data."( Effect of Genetic Polymorphisms on the Pharmacokinetics of Deferasirox in Healthy Chinese Subjects and an Artificial Neural Networks Model for Pharmacokinetic Prediction.
Chen, J; Hu, Y; Jiang, B; Lou, H; Ruan, Z; Shao, R; Xu, Y; Yang, D, 2020
)
1.02
" The plasma defersirox concentration was determined using a validated liquid chromatography-tandem mass spectrometry method, and pharmacokinetic parameters were calculated using the noncompartmental method."( Effect of Genetic Polymorphisms on the Pharmacokinetics of Deferasirox in Healthy Chinese Subjects and an Artificial Neural Networks Model for Pharmacokinetic Prediction.
Chen, J; Hu, Y; Jiang, B; Lou, H; Ruan, Z; Shao, R; Xu, Y; Yang, D, 2020
)
0.8

Compound-Compound Interactions

ExcerptReferenceRelevance
" Therefore, we sought to explore the synergistic effects and possible mechanisms of DFX combination with ELT in MDS cells."( Deferasirox combination with eltrombopag shows anti-myelodysplastic syndrome effects by enhancing iron deprivation-related apoptosis.
Fu, R; Huang, L; Liu, C; Liu, Z; Tian, M, 2022
)
2.16
" Hydroxyurea, when combined with iron chelators such as DFX, provides an additional benefit of iron chelation in patients receiving chronic transfusion therapy."( Iron Chelators, Such as Deferasirox, When Combined With Hydroxyurea, Provide an Additional Benefit of Iron Chelation in Patients Receiving Chronic Transfusion Therapy.
Delicou, S; Koskinas, J; Manganas, K; Xydaki, A, 2022
)
1.03

Bioavailability

deferasirox bioavailability is unaltered when dispersed with orange or apple juice compared with dispersion in water. Oral bioavailability was not affected neither by the degree of dispersion nor by the type of drink used for dispersion.

ExcerptReferenceRelevance
" This was followed by a pharmacokinetic study to assess the relative bioavailability of deferasirox tablets dispersed in two types of soft drinks, dispersed in water, and without dispersion."( Relative bioavailability of deferasirox tablets administered without dispersion and dispersed in various drinks.
Balez, S; Brun, E; Dumortier, T; Dutreix, C; Morisson, S; Pommier, F; Séchaud, R, 2008
)
0.86
" Therefore, the oral bioavailability of deferasirox tablets was not affected neither by the degree of dispersion nor by the type of drink (orange or apple juice versus water) used for dispersion."( Relative bioavailability of deferasirox tablets administered without dispersion and dispersed in various drinks.
Balez, S; Brun, E; Dumortier, T; Dutreix, C; Morisson, S; Pommier, F; Séchaud, R, 2008
)
0.91
"This study shows that deferasirox bioavailability is unaltered when dispersed with orange or apple juice compared with dispersion in water."( Relative bioavailability of deferasirox tablets administered without dispersion and dispersed in various drinks.
Balez, S; Brun, E; Dumortier, T; Dutreix, C; Morisson, S; Pommier, F; Séchaud, R, 2008
)
0.95
" The bioavailability of deferasirox was determined following a single oral dose (20 mg/kg) under fed and fasted conditions in patients."( Effect of food, type of food, and time of food intake on deferasirox bioavailability: recommendations for an optimal deferasirox administration regimen.
Alberti, D; Balez, S; Belleli, R; Cappellini, MD; Dutreix, C; Ford, JM; Forni, GL; Galanello, R; Origa, R; Piga, A; Rivière, GJ; Séchaud, R; Zappu, A, 2008
)
0.9
" The current study evaluated the absolute bioavailability of a single 375-mg oral dose of deferasirox administered in the form of tablets compared with a 130-mg intravenous infusion of deferasirox."( Absolute oral bioavailability and disposition of deferasirox in healthy human subjects.
Balez, S; Belleli, R; Robeva, A; Séchaud, R, 2008
)
0.82
" bioavailability was 26% at the 10 mg/kg dose and showed an overproportional increase at the 100 mg/kg dose, probably because of saturation of elimination processes."( Pharmacokinetics, distribution, metabolism, and excretion of deferasirox and its iron complex in rats.
Boernsen, KO; Bruin, GJ; Faller, T; Nick, H; Schneider, J; Schweitzer, A; Waldmeier, F; Wiegand, H, 2008
)
0.59
" Cmax, volume of distribution/bioavailability (Vd/F), and elimination half-life (t(1/2)) were not different between the groups, suggesting bioavailability as the likely discriminant."( Deferasirox pharmacokinetics in patients with adequate versus inadequate response.
Bergmann, AK; Braunstein, J; Chirnomas, D; Finkelstein, Y; Grant, FD; Neufeld, EJ; Paley, C; Pereira, L; Shannon, M; Smith, AL, 2009
)
1.8
" A promising method to improve the bioavailability of pharmaceutical agents is the rapid expansion of supercritical solutions."( Formation of ultrafine deferasirox particles via rapid expansion of supercritical solution (RESS process) using Taguchi approach.
Asghari, I; Esmaeilzadeh, F, 2012
)
0.69
" In this study, we have conjugated DFOB to derivatives of adamantane or the clinical iron chelator deferasirox to produce lipophilic compounds designed to increase the bioavailability of DFOB to brain cells."( Lipophilic adamantyl- or deferasirox-based conjugates of desferrioxamine B have enhanced neuroprotective capacity: implications for Parkinson disease.
Codd, R; Crouch, PJ; Ganio, G; Liddell, JR; Liu, J; Mok, SS; Obando, D; Volitakis, I; White, AR, 2013
)
0.91
" This finding enriches our bioinspired drug design strategy for Ti(IV)-based anticancer therapeutics, which applies a family of Fe(III) chelators termed chemical transferrin mimetic (cTfm) ligands to inhibit Fe bioavailability in cancer cells."( Expanding the Therapeutic Potential of the Iron Chelator Deferasirox in the Development of Aqueous Stable Ti(IV) Anticancer Complexes.
Benjamín-Rivera, JA; Delgado, Y; Loza-Rosas, SA; Munet-Colón, C; Negrón, LJ; Parks, TB; Rivero, KI; Tinoco, AD; Vázquez-Maldonado, AL; Vázquez-Salgado, AM, 2017
)
0.7
"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
" Low bioavailability of the drug due to insufficient solubility in physiological fluids is the main drawback of DFX."( Solid Dispersion Pellets: An Efficient Pharmaceutical Approach to Enrich the Solubility and Dissolution Rate of Deferasirox.
Abbaspour, M; Ebrahimnejad, P; Farmoudeh, A; Nokhodchi, A; Rezaeiroshan, A, 2020
)
0.77

Dosage Studied

deferasirox allows dose adjustments to be made in response to trends in serum ferritin, to changes in a patient's transfusional iron intake, and to the objectives of treatment. It allows the full benefit of transfusion therapy without the risks associated with iron overload.

ExcerptRelevanceReference
" Patients were randomized and received treatment with deferasirox (n = 296) or deferoxamine (n = 290), with dosing of each according to baseline liver iron concentration (LIC)."( A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia.
Agaoglu, L; Alberti, D; Athanassiou-Metaxa, M; Aydinok, Y; Bejaoui, M; Cappellini, MD; Capra, M; Coates, T; Cohen, A; Drelichman, G; Fattoum, S; Galanello, R; Giardina, P; Janka-Schaub, G; Kattamis, A; Kilinc, Y; Kourakli-Symeonidis, A; Magnano, C; Marks, P; Olivieri, N; Opitz, H; Perrotta, S; Piga, A; Porter, J; Ressayre-Djaffer, C; Thuret, I; Verissimo, M; Vermylen, C, 2006
)
0.91
"To review the available literature on the pharmacology, pharmacokinetics, efficacy, toxicology, adverse effects, drug interactions, and dosage guidelines for deferasirox, an oral iron chelator, in Phase III trials."( Deferasirox--an oral agent for chronic iron overload.
Hagemann, TM; Vanorden, HE, 2006
)
1.97
" Pharmacokinetic data support a once-daily dosing regimen based on body weight."( Phase II clinical evaluation of deferasirox, a once-daily oral chelating agent, in pediatric patients with beta-thalassemia major.
Alberti, D; Belleli, R; Bertrand, Y; Bordone, E; Forni, GL; Foschini, ML; Galanello, R; Hewson, N; Lavagetto, A; Leoni, G; Longo, F; Maseruka, H; Piga, A; Sechaud, R; Zappu, A, 2006
)
0.62
" Clinical phase I and II studies demonstrated an exclusively fecal route of iron excretion, with a long plasma half-life, suitable for once-daily dosing and 24-hour protection from labile iron."( Deferasirox: An effective once-daily orally active iron chelator.
Porter, JB, 2006
)
1.78
"Daily divided dosing of deferasirox changes the relative cardiac and liver iron chelation profile compared with daily single dosing, trading improvements in cardiac iron elimination for less-effective hepatic chelation."( Comparison of twice-daily vs once-daily deferasirox dosing in a gerbil model of iron cardiomyopathy.
Aguilar, M; Moats, R; Nick, H; Otto-Duessel, M; Wood, JC, 2007
)
0.91
" Dosage was determined by baseline liver iron concentration (LIC)."( Relative response of patients with myelodysplastic syndromes and other transfusion-dependent anaemias to deferasirox (ICL670): a 1-yr prospective study.
Alberti, D; Alimena, G; Cappellini, MD; Cario, H; Cazzola, M; Cunningham, MJ; Debusscher, L; Della Porta, M; Ford, JM; Forni, GL; Galanello, R; Gathmann, I; Gattermann, N; Giardina, P; Greenberg, P; Jeng, M; Kwiatkowski, J; Maertens, J; Neufeld, EJ; Olivieri, N; Piga, A; Porter, J; Quarta, G; Rabault, B; Rose, C; Saglio, G; Soulières, D; Stadler, M; Tchernia, G; Vichinsky, E, 2008
)
0.56
" Once-daily dosing with deferasirox > or =20 mg/kg/d provided sustained reduction in LPI levels in these heavily iron-overloaded patients, suggesting 24-h protection from LPI."( Reduction in labile plasma iron during treatment with deferasirox, a once-daily oral iron chelator, in heavily iron-overloaded patients with beta-thalassaemia.
Daar, S; Habr, D; Hmissi, A; Kriemler-Krahn, U; Nick, H; Pathare, A; Taher, A, 2009
)
0.91
" The flexible dosing regimen of deferasirox allows dose adjustments to be made in response to trends in serum ferritin, to changes in a patient's transfusional iron intake, and to the objectives of treatment, allowing the full benefit of transfusion therapy without the risks associated with iron overload."( Managing iron overload in patients with myelodysplastic syndromes with oral deferasirox therapy.
Garcia-Manero, G; Jabbour, E; Kantarjian, HM; Taher, A, 2009
)
0.87
" Effective dosing regimens for inadequately responding patients to deferasirox must be determined."( Deferasirox pharmacokinetics in patients with adequate versus inadequate response.
Bergmann, AK; Braunstein, J; Chirnomas, D; Finkelstein, Y; Grant, FD; Neufeld, EJ; Paley, C; Pereira, L; Shannon, M; Smith, AL, 2009
)
2.03
"0001), reflecting dosage adjustments and ongoing iron intake."( Tailoring iron chelation by iron intake and serum ferritin: the prospective EPIC study of deferasirox in 1744 patients with transfusion-dependent anemias.
Cappellini, MD; Chan, LL; Domokos, G; El-Beshlawy, A; Elalfy, M; Gattermann, N; Giraudier, S; Habr, D; Kattamis, A; Lee, JW; Li, CK; Lin, KH; Porter, J; Rose, C; Roubert, B; Seymour, JF; Taher, A; Thein, SL; Viprakasit, V, 2010
)
0.58
" Excluding the diagnostic dilemma of an improbable failure of DFX chelation, the pathogenesis of this phenomenon remains to be clarified, thus further complicating the problem of ferritin specificity and its role in monitoring chelation efficacy and in adapting DFX dosage in a limited period of treatment."( Paradoxically increased ferritin level in a beta-thalassemia major patient following the start of deferasirox chelation therapy.
Ammirabile, M; Cinque, P; Costantini, S; Di Matola, T; Prossomariti, L; Ricchi, P; Spasiano, A, 2010
)
0.58
" Using a pre-specified dosing algorithm serum ferritin reduction was similar in both groups, mean difference (MD) 375."( Deferasirox for managing transfusional iron overload in people with sickle cell disease.
Antes, G; Bassler, D; Fleeman, N; Meerpohl, JJ; Niemeyer, C; Rücker, G, 2010
)
1.8
"Patients from two Taiwanese hospitals with transfusion-dependent β-thalassemia, including those who showed increasing serum ferritin levels for six consecutive months, with at least one level >2,500 ng/dl, while treated with >30 mg/kg/day of once-daily deferasirox (unresponsive) or developed deferasirox-related adverse events (AEs) at the dosage required to maintain the iron burden balance (intolerant) and were treated twice-daily with the same total daily dose of deferasirox since 2008, were enrolled in the study and evaluated retrospectively by medical record review."( Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia.
Chang, HH; Chang, TT; Chiou, SS; Jou, ST; Liao, YM; Lin, DT; Lin, KH; Lin, PC; Lu, MY; Yang, YL, 2011
)
0.81
"Twice-daily deferasirox dosing is effective in iron chelation and improves tolerability in transfusion-dependent β-thalassemia patients who are unresponsive to or intolerant of once-daily deferasirox."( Improved efficacy and tolerability of oral deferasirox by twice-daily dosing for patients with transfusion-dependent β-thalassemia.
Chang, HH; Chang, TT; Chiou, SS; Jou, ST; Liao, YM; Lin, DT; Lin, KH; Lin, PC; Lu, MY; Yang, YL, 2011
)
1.01
" The standard dosage is not useful to every patient."( [Deferasirox--a new oral iron chelator--review].
Chen, BA; Gao, C; Wang, T, 2010
)
1.27
" Iron burden was substantially reduced with appropriate dosing in patients treated for at least 4 years."( Long-term safety and efficacy of deferasirox (Exjade) for up to 5 years in transfusional iron-overloaded patients with sickle cell disease.
Bernaudin, F; Coates, T; Deng, W; Forni, GL; Gardner, R; Giannone, V; Griffel, L; Hassell, K; Heeney, MM; Inusa, B; Kutlar, A; Lane, P; Mathias, L; Porter, J; Tebbi, C; Vichinsky, E; Wilson, F, 2011
)
0.65
" 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
"30 mL red blood cells/kg/d), mean deferasirox dosing (19."( Response of iron overload to deferasirox in rare transfusion-dependent anaemias: equivalent effects on serum ferritin and labile plasma iron for haemolytic or production anaemias.
Beris, P; Domokos, G; Forni, GL; Habr, D; Lin, KH; Porter, JB; Roubert, B; Taher, A; Thein, SL, 2011
)
0.94
"Based on the current available data, deferasirox treatment is effective with a clinically manageable safety profile although appropriate dosing according to the severity of iron burden and iron intake, together with the careful monitoring of laboratory parameters and adverse events, is recommended."( Deferasirox: pharmacokinetics and clinical experience.
Campus, S; Galanello, R; Origa, R, 2012
)
2.09
"The effect of deferasirox dosing tailored for iron burden and iron loading based on liver iron concentration (LIC) was assessed over 1 year in less versus more heavily iron-overloaded patients in a substudy of the Evaluation of Patients' Iron Chelation with Exjade®."( Efficacy and safety of deferasirox at low and high iron burdens: results from the EPIC magnetic resonance imaging substudy.
Aydinok, Y; Chan, LL; El-Beshlawy, A; Elalfy, MS; Habr, D; Lee, SH; Martin, N; Porter, JB; Sutcharitchan, P; Taher, AT, 2013
)
1.06
" These analyses also evaluated deferasirox dosing strategies for patients with NTDT."( Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients.
Cappellini, MD; Chuncharunee, S; Galanello, R; Habr, D; Karakas, Z; Kattamis, A; Lawniczek, T; Porter, JB; Ros, J; Siritanaratkul, N; Sutcharitchan, P; Taher, AT; Viprakasit, V; Zhang, Y, 2013
)
2.12
" The iron chelating treatment significantly reduced serum ferritin levels administered at a dosage of 20-30 mg/kg/day (p<0."( The oral iron chelator deferasirox might improve survival in allogeneic hematopoietic cell transplant (alloHSCT) recipients with transfusional iron overload.
Akyol, G; Baldane, S; Cetin, M; Eser, B; Kaynar, L; Keklik, M; Kurnaz, F; Pala, C; Sivgin, S; Unal, A; Zararsiz, G, 2013
)
0.7
" Effective DFX dosage is thus defined by assessing the efficacy of this agent in clinical practice."( Elevated total iron-binding capacity as a predictor of response to deferasirox therapy in the setting of chronic iron overload.
Hikota, R; Horiuchi, T; Kato, S; Kimura, F; Kobayashi, A; Kobayashi, S; Maekawa, T; Osawa, Y; Sato, K; Watanabe, J; Yamamura, T, 2014
)
0.64
" This review summarizes the clinical pharmacokinetics, pharmacodynamics, and drug-drug interaction profile of deferasirox, and the claims supporting once-daily dosing for effective chelation."( Clinical pharmacology of deferasirox.
Tanaka, C, 2014
)
0.92
" Quantitative magnetic resonance imaging indicated that dosage escalations up to 100 mg/kg were needed to produce meaningful reductions in iron stores."( Dose titration of deferasirox iron chelation therapy by magnetic resonance imaging for chronic iron storage disease in three adult red bald-headed uakari (Cacajao calvus rubicundus).
Baer, J; Brewer, C; Garner, M; Stadler, CK; Tyszka, JM; Wood, JC, 2014
)
0.74
" Individual patient-tailored dosing of DFX should help to improve iron chelation efficacy and to reduce dose-dependent drug toxicity."( Determination of deferasirox plasma concentrations: do gender, physical and genetic differences affect chelation efficacy?
Forni, GL; Fucile, C; Marini, V; Martelli, A; Mattioli, F; Milano, G; Perrotta, S; Pinto, V; Puntoni, M; Robbiano, L, 2015
)
0.76
" Plasma concentrations were determined at the end of dosing interval (C trough) using an high-performance liquid chromatography-ultraviolet method."( Influence of single-nucleotide polymorphisms on deferasirox C trough levels and effectiveness.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Massano, D; Piga, A, 2015
)
0.67
" However, the pharmacokinetic (PK) disposition of DEFR and the iron-DEFR complex (Fe-[DEFR]2) in this dosing strategy is unclear."( Simultaneous Determination of Plasma Deferasirox and Deferasirox-Iron Complex Using an HPLC-UV System and Pharmacokinetics of Deferasirox in Patients With β-Thalassemia Major: Once-daily Versus Twice-daily Administration.
Chiang, PH; Kuo, PH; Lai, CW; Lin, KH; Lu, MY; Wang, N; Wu, TH; Wu, WH, 2015
)
0.69
"Chromatographic analysis was performed using a solvent delivery system coupled to an HPLC-UV detector to determine the steady-state concentrations of DEFR (CDEFR) and Fe-(DEFR)2 (CFe-[DEFR]2) in β-thalassemia major patients (n = 8) following either once-daily or BID dosing, during which the PK parameters of the 2 dosing schedules were compared."( Simultaneous Determination of Plasma Deferasirox and Deferasirox-Iron Complex Using an HPLC-UV System and Pharmacokinetics of Deferasirox in Patients With β-Thalassemia Major: Once-daily Versus Twice-daily Administration.
Chiang, PH; Kuo, PH; Lai, CW; Lin, KH; Lu, MY; Wang, N; Wu, TH; Wu, WH, 2015
)
0.69
"A total of 28 patients either dosing daily or twice daily were recruited."( Deferasirox-Iron Complex Formation Ratio as an Indicator of Long-term Chelation Efficacy in β-Thalassemia Major.
Chiang, PH; Kuo, PH; Lin, KH; Lin, TH; Lu, MY; Wang, N; Wu, TH; Wu, WH, 2017
)
1.9
" The pooled effects across the different dosing ratios are: serum ferritin, mean difference (MD) 454."( Deferasirox for managing iron overload in people with thalassaemia.
Allert, R; Bassler, D; Bollig, C; Meerpohl, JJ; Motschall, E; Niemeyer, CM; Rücker, G; Schell, LK, 2017
)
1.9
" Drug plasma concentrations at the end of dosing interval (Ctrough) and after 0, 2, 4, 6, and 24 h of drug administration were measured by a HPLC-UV method."( Effect of pharmacogenetic markers of vitamin D pathway on deferasirox pharmacokinetics in children.
Allegra, S; Cusato, J; D'Avolio, A; De Francia, S; Longo, F; Massano, D; Piga, A; Pirro, E, 2018
)
0.73
" Our dose escalating method of deferasirox is useful to identify the optimal dosage of the drug in each patient."( Deferasirox for the treatment of iron overload after allogeneic hematopoietic cell transplantation: multicenter phase I study (KSGCT1302).
Kanamori, H; Kanda, J; Kanda, Y; Koyama, S; Machida, S; Miyazaki, T; Morita, S; Najima, Y; Okamoto, S; Saito, T; Tachibana, T; Takeuchi, M; Tanaka, M; Yamamoto, E, 2018
)
2.21
"Whilst only moderately effective at a dosage of 1000mg/day, deferasirox may be a safe agent for iron removal in iron overloaded peritoneal dialysis patients, as relatively low dialysate iron levels reduces the risk of Yersinia and Rhizopus infection."( Use of deferasirox (Exjade) for iron overload in peritoneal dialysis patients.
Doery, JC; Kaplan, Z; Kerr, PG; Yii, E, 2018
)
1.18
" Dose adjustments were reported in 48% of the patients with dose-escalation strategies being the most frequent reason for dosage increases (49%)."( Tolerability and efficacy of deferasirox in patients with transfusional iron overload: results from a German 2-year non-interventional study.
Albrecht, S; Geer, T; Hebart, H; Hofmann, WK; Jarisch, A; Johr, C; Nolte, F; Nückel, H; Rubanov, O; Schmidt, B; Schumann, C, 2018
)
0.77
" Preventing and successfully managing AEs may help limit their impact on adherence, and following dosage and administration recommendations for iron chelators such as deferasirox may help minimise AEs and optimise treatment in patients with transfusion-dependent thalassaemia and lower-risk MDS."( Optimising management of deferasirox therapy for patients with transfusion-dependent thalassaemia and lower-risk myelodysplastic syndromes.
Aydinok, Y; Kattamis, A; Taher, A, 2018
)
0.98
" Regular monitoring and an adjusted deferasirox dosing strategy per local labels allowed continued iron chelation treatment and control of transfusional iron in the majority of patients on study."( International sentinel site surveillance of patients with transfusional hemosiderosis treated with deferasirox in actual practice setting.
Azmon, A; Beneitez Pastor, D; Bruederle, A; El-Beshlawy, A; Elalfy, M; Gilotti, G; Inusa, B; Soledada Duran Nieto, M; Xicoy, B, 2019
)
1.01
" Group II consisted of 19 people with severe thallium salt poisoning, which in addition to the above treatment, received Deferasirox in a dosage of 500 mg two times per day per os."( Complexions therapy and severe intoxication by Thallium salts.
Babaskina, L; Rayisyan, M; Zakharova, N, 2021
)
0.83
" In the literature, there were three studies showing the boon of twice in a day dosing of deferasirox among transfusional-dependent beta thalassemia patients."( Effectiveness and tolerability of twice daily dosing of deferasirox in unresponsive and intolerant transfusion-dependent beta-thalassemia patients: A narrative review.
Babu, T; Panachiyil, GM; Ravi, MD; Sebastian, J,
)
0.6
" Total annual exposure to DFX was calculated after 12 months and expressed as the accumulated exposure time at a dosage of 20 mg/kg/day."( Dose of deferasirox correlates with its effects, which differ between low-risk myelodysplastic syndrome and aplastic anaemia.
Han, B; Zhang, R, 2022
)
1.16
" The minimum dosage of DFX needed for a significant serum ferritin (SF) decrease was 20 mg/kg/day at 6 months, and the minimum accumulation of DFX had to reach 9 months at 20 mg/kg/day by 12 months for patients with low-risk MDS."( Dose of deferasirox correlates with its effects, which differ between low-risk myelodysplastic syndrome and aplastic anaemia.
Han, B; Zhang, R, 2022
)
1.16
" In the United States, deferiprone has been approved for three times daily dosing since 2011 and has recently gained approval for twice-daily administration."( An evaluation of deferiprone as twice-a-day tablets or in combination therapy for the treatment of transfusional iron overload in thalassemia syndromes.
Badawy, SM; Shah, A; Shah, R, 2023
)
0.91
" Twice-daily administration provides a similar level of iron chelation as three times daily dosing with a comparable side effect profile and increased patient acceptability."( An evaluation of deferiprone as twice-a-day tablets or in combination therapy for the treatment of transfusional iron overload in thalassemia syndromes.
Badawy, SM; Shah, A; Shah, R, 2023
)
0.91
" In principle, iron chelators can be combined with an infinite number of dosing regimens; these involve simultaneous or sequential exposure to the chelators on the same day or alternating the drugs on different days."( Combination chelation therapy.
Aydinok, Y, 2023
)
0.91
[information is derived through text-mining from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Roles (1)

RoleDescription
iron chelatornull
[role information is derived from Chemical Entities of Biological Interest (ChEBI), Hastings J, Owen G, Dekker A, Ennis M, Kale N, Muthukrishnan V, Turner S, Swainston N, Mendes P, Steinbeck C. (2016). ChEBI in 2016: Improved services and an expanding collection of metabolites. Nucleic Acids Res]

Drug Classes (4)

ClassDescription
triazolesAn azole in which the five-membered heterocyclic aromatic skeleton contains three N atoms and two C atoms.
monocarboxylic acidAn oxoacid containing a single carboxy group.
benzoic acidsAny aromatic carboxylic acid that consists of benzene in which at least a single hydrogen has been substituted by a carboxy group.
phenolsOrganic aromatic compounds having one or more hydroxy groups attached to a benzene or other arene ring.
[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 (44)

Potency Measurements

ProteinTaxonomyMeasurementAverage (µ)Min (ref.)Avg (ref.)Max (ref.)Bioassay(s)
LuciferasePhotinus pyralis (common eastern firefly)Potency36.12540.007215.758889.3584AID624030
hypoxia-inducible factor 1 alpha subunitHomo sapiens (human)Potency6.00703.189029.884159.4836AID1224846
RAR-related orphan receptor gammaMus musculus (house mouse)Potency27.18170.006038.004119,952.5996AID1159521; AID1159523
SMAD family member 2Homo sapiens (human)Potency23.91450.173734.304761.8120AID1346924
Fumarate hydrataseHomo sapiens (human)Potency37.22120.00308.794948.0869AID1347053
SMAD family member 3Homo sapiens (human)Potency23.91450.173734.304761.8120AID1346924
TDP1 proteinHomo sapiens (human)Potency27.31710.000811.382244.6684AID686978; AID686979
GLI family zinc finger 3Homo sapiens (human)Potency33.49150.000714.592883.7951AID1259369; AID1259392
AR proteinHomo sapiens (human)Potency8.46210.000221.22318,912.5098AID743035; AID743063
estrogen receptor 2 (ER beta)Homo sapiens (human)Potency3.34910.000657.913322,387.1992AID1259378
nuclear receptor subfamily 1, group I, member 3Homo sapiens (human)Potency7.09010.001022.650876.6163AID1224838; AID1224893
progesterone receptorHomo sapiens (human)Potency6.68240.000417.946075.1148AID1346795
cytochrome P450 family 3 subfamily A polypeptide 4Homo sapiens (human)Potency2.75400.01237.983543.2770AID1645841
EWS/FLI fusion proteinHomo sapiens (human)Potency17.11260.001310.157742.8575AID1259252; AID1259253; AID1259255; AID1259256
glucocorticoid receptor [Homo sapiens]Homo sapiens (human)Potency10.72940.000214.376460.0339AID720691; AID720692; AID720719
retinoic acid nuclear receptor alpha variant 1Homo sapiens (human)Potency24.25890.003041.611522,387.1992AID1159552; AID1159553; AID1159555
retinoid X nuclear receptor alphaHomo sapiens (human)Potency2.68320.000817.505159.3239AID1159527; AID1159531
estrogen-related nuclear receptor alphaHomo sapiens (human)Potency15.65970.001530.607315,848.9004AID1224841; AID1224842; AID1224848; AID1224849; AID1259401; AID1259403
farnesoid X nuclear receptorHomo sapiens (human)Potency26.60110.375827.485161.6524AID743217
estrogen nuclear receptor alphaHomo sapiens (human)Potency25.18060.000229.305416,493.5996AID1259244; AID1259248; AID743069; AID743075; AID743078; AID743079; AID743080; AID743091
polyproteinZika virusPotency37.22120.00308.794948.0869AID1347053
peroxisome proliferator-activated receptor deltaHomo sapiens (human)Potency13.33220.001024.504861.6448AID743215
peroxisome proliferator activated receptor gammaHomo sapiens (human)Potency5.95530.001019.414170.9645AID743191
cytochrome P450, family 19, subfamily A, polypeptide 1, isoform CRA_aHomo sapiens (human)Potency29.84930.001723.839378.1014AID743083
thyroid stimulating hormone receptorHomo sapiens (human)Potency23.86750.001628.015177.1139AID1259385; AID1259395
nuclear factor of kappa light polypeptide gene enhancer in B-cells 1 (p105), isoform CRA_aHomo sapiens (human)Potency4.252719.739145.978464.9432AID1159509
Histone H2A.xCricetulus griseus (Chinese hamster)Potency55.56270.039147.5451146.8240AID1224845; AID1224896
thyroid hormone receptor beta isoform 2Rattus norvegicus (Norway rat)Potency25.44230.000323.4451159.6830AID743065; AID743066; AID743067
nuclear factor erythroid 2-related factor 2 isoform 1Homo sapiens (human)Potency1.33320.000627.21521,122.0200AID743202
Voltage-dependent calcium channel gamma-2 subunitMus musculus (house mouse)Potency29.84930.001557.789015,848.9004AID1259244
Cellular tumor antigen p53Homo sapiens (human)Potency26.60320.002319.595674.0614AID651631
Glutamate receptor 2Rattus norvegicus (Norway rat)Potency29.84930.001551.739315,848.9004AID1259244
[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)
Lysine-specific demethylase 6BHomo sapiens (human)IC50 (µMol)3.95000.01601.66726.9000AID1872471
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)36.50000.20005.677410.0000AID1473741
Lysine-specific demethylase 4AHomo sapiens (human)IC50 (µMol)3.77000.20002.83194.7600AID1872469; AID1885301; AID1885302
Bile salt export pumpHomo sapiens (human)IC50 (µMol)58.40000.11007.190310.0000AID1473738
Lysine-specific demethylase 5AHomo sapiens (human)IC50 (µMol)5.00000.13002.374710.0000AID1872470
Potassium voltage-gated channel subfamily H member 2Homo sapiens (human)IC50 (µMol)3,981.07010.00091.901410.0000AID1207518
Voltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)IC50 (µMol)1,000.00000.00032.25459.6000AID1207552
Sodium channel protein type 5 subunit alphaHomo sapiens (human)IC50 (µMol)290.30990.00033.64849.2000AID1207304; AID1207336
Canalicular multispecific organic anion transporter 1Homo sapiens (human)IC50 (µMol)133.00002.41006.343310.0000AID1473739
Potassium voltage-gated channel subfamily D member 3Homo sapiens (human)IC50 (µMol)50.11871.40005.35009.3000AID1207424
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Activation Measurements

ProteinTaxonomyMeasurementAverageMin (ref.)Avg (ref.)Max (ref.)Bioassay(s)
Flavin reductase (NADPH)Homo sapiens (human)Kd1.70410.07000.79681.7100AID1815675
[prepared from compound, protein, and bioassay information from National Library of Medicine (NLM), extracted Dec-2023]

Biological Processes (258)

Processvia Protein(s)Taxonomy
inflammatory response to antigenic stimulusLysine-specific demethylase 6BHomo sapiens (human)
chromatin remodelingLysine-specific demethylase 6BHomo sapiens (human)
response to activityLysine-specific demethylase 6BHomo sapiens (human)
hippocampus developmentLysine-specific demethylase 6BHomo sapiens (human)
cell fate commitmentLysine-specific demethylase 6BHomo sapiens (human)
endothelial cell differentiationLysine-specific demethylase 6BHomo sapiens (human)
positive regulation of transcription by RNA polymerase IILysine-specific demethylase 6BHomo sapiens (human)
mesodermal cell differentiationLysine-specific demethylase 6BHomo sapiens (human)
cardiac muscle cell differentiationLysine-specific demethylase 6BHomo sapiens (human)
response to fungicideLysine-specific demethylase 6BHomo sapiens (human)
cellular response to hydrogen peroxideLysine-specific demethylase 6BHomo sapiens (human)
positive regulation of cold-induced thermogenesisLysine-specific demethylase 6BHomo sapiens (human)
heart developmentLysine-specific demethylase 6BHomo sapiens (human)
regulation of gene expressionLysine-specific demethylase 6BHomo sapiens (human)
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)
negative regulation of autophagyLysine-specific demethylase 4AHomo sapiens (human)
positive regulation of gene expressionLysine-specific demethylase 4AHomo sapiens (human)
negative regulation of gene expressionLysine-specific demethylase 4AHomo sapiens (human)
cardiac muscle hypertrophy in response to stressLysine-specific demethylase 4AHomo sapiens (human)
apoptotic chromosome condensationLysine-specific demethylase 4AHomo sapiens (human)
response to nutrient levelsLysine-specific demethylase 4AHomo sapiens (human)
positive regulation of neuron differentiationLysine-specific demethylase 4AHomo sapiens (human)
negative regulation of DNA-templated transcriptionLysine-specific demethylase 4AHomo sapiens (human)
negative regulation of astrocyte differentiationLysine-specific demethylase 4AHomo sapiens (human)
chromatin remodelingLysine-specific demethylase 4AHomo sapiens (human)
regulation of gene expressionLysine-specific demethylase 4AHomo 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)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycle G2/M phase transitionCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
ER overload responseCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
mitophagyCellular tumor antigen p53Homo sapiens (human)
in utero embryonic developmentCellular tumor antigen p53Homo sapiens (human)
somitogenesisCellular tumor antigen p53Homo sapiens (human)
release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
hematopoietic progenitor cell differentiationCellular tumor antigen p53Homo sapiens (human)
T cell proliferation involved in immune responseCellular tumor antigen p53Homo sapiens (human)
B cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
T cell lineage commitmentCellular tumor antigen p53Homo sapiens (human)
response to ischemiaCellular tumor antigen p53Homo sapiens (human)
nucleotide-excision repairCellular tumor antigen p53Homo sapiens (human)
double-strand break repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
protein import into nucleusCellular tumor antigen p53Homo sapiens (human)
autophagyCellular tumor antigen p53Homo sapiens (human)
DNA damage responseCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in cell cycle arrestCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediator resulting in transcription of p21 class mediatorCellular tumor antigen p53Homo sapiens (human)
transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
Ras protein signal transductionCellular tumor antigen p53Homo sapiens (human)
gastrulationCellular tumor antigen p53Homo sapiens (human)
neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of neuroblast proliferationCellular tumor antigen p53Homo sapiens (human)
protein localizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA replicationCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell population proliferationCellular tumor antigen p53Homo sapiens (human)
determination of adult lifespanCellular tumor antigen p53Homo sapiens (human)
mRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
rRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
response to salt stressCellular tumor antigen p53Homo sapiens (human)
response to inorganic substanceCellular tumor antigen p53Homo sapiens (human)
response to X-rayCellular tumor antigen p53Homo sapiens (human)
response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
positive regulation of gene expressionCellular tumor antigen p53Homo sapiens (human)
cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of cardiac muscle cell apoptotic processCellular tumor antigen p53Homo sapiens (human)
glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
viral processCellular tumor antigen p53Homo sapiens (human)
glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
cerebellum developmentCellular tumor antigen p53Homo sapiens (human)
negative regulation of cell growthCellular tumor antigen p53Homo sapiens (human)
DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
negative regulation of transforming growth factor beta receptor signaling pathwayCellular tumor antigen p53Homo sapiens (human)
mitotic G1 DNA damage checkpoint signalingCellular tumor antigen p53Homo sapiens (human)
negative regulation of telomere maintenance via telomeraseCellular tumor antigen p53Homo sapiens (human)
T cell differentiation in thymusCellular tumor antigen p53Homo sapiens (human)
tumor necrosis factor-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
regulation of tissue remodelingCellular tumor antigen p53Homo sapiens (human)
cellular response to UVCellular tumor antigen p53Homo sapiens (human)
multicellular organism growthCellular tumor antigen p53Homo sapiens (human)
positive regulation of mitochondrial membrane permeabilityCellular tumor antigen p53Homo sapiens (human)
cellular response to glucose starvationCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to DNA damage by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of apoptotic processCellular tumor antigen p53Homo sapiens (human)
entrainment of circadian clock by photoperiodCellular tumor antigen p53Homo sapiens (human)
mitochondrial DNA repairCellular tumor antigen p53Homo sapiens (human)
regulation of DNA damage response, signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
transcription initiation-coupled chromatin remodelingCellular tumor antigen p53Homo sapiens (human)
negative regulation of proteolysisCellular tumor antigen p53Homo sapiens (human)
negative regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of DNA-templated transcriptionCellular tumor antigen p53Homo sapiens (human)
positive regulation of RNA polymerase II transcription preinitiation complex assemblyCellular tumor antigen p53Homo sapiens (human)
positive regulation of transcription by RNA polymerase IICellular tumor antigen p53Homo sapiens (human)
response to antibioticCellular tumor antigen p53Homo sapiens (human)
fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
negative regulation of fibroblast proliferationCellular tumor antigen p53Homo sapiens (human)
circadian behaviorCellular tumor antigen p53Homo sapiens (human)
bone marrow developmentCellular tumor antigen p53Homo sapiens (human)
embryonic organ developmentCellular tumor antigen p53Homo sapiens (human)
positive regulation of peptidyl-tyrosine phosphorylationCellular tumor antigen p53Homo sapiens (human)
protein stabilizationCellular tumor antigen p53Homo sapiens (human)
negative regulation of helicase activityCellular tumor antigen p53Homo sapiens (human)
protein tetramerizationCellular tumor antigen p53Homo sapiens (human)
chromosome organizationCellular tumor antigen p53Homo sapiens (human)
neuron apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of cell cycleCellular tumor antigen p53Homo sapiens (human)
hematopoietic stem cell differentiationCellular tumor antigen p53Homo sapiens (human)
negative regulation of glial cell proliferationCellular tumor antigen p53Homo sapiens (human)
type II interferon-mediated signaling pathwayCellular tumor antigen p53Homo sapiens (human)
cardiac septum morphogenesisCellular tumor antigen p53Homo sapiens (human)
positive regulation of programmed necrotic cell deathCellular tumor antigen p53Homo sapiens (human)
protein-containing complex assemblyCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to endoplasmic reticulum stressCellular tumor antigen p53Homo sapiens (human)
thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of thymocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
necroptotic processCellular tumor antigen p53Homo sapiens (human)
cellular response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
cellular response to xenobiotic stimulusCellular tumor antigen p53Homo sapiens (human)
cellular response to ionizing radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to gamma radiationCellular tumor antigen p53Homo sapiens (human)
cellular response to UV-CCellular tumor antigen p53Homo sapiens (human)
stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
signal transduction by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
cellular response to actinomycin DCellular tumor antigen p53Homo sapiens (human)
positive regulation of release of cytochrome c from mitochondriaCellular tumor antigen p53Homo sapiens (human)
cellular senescenceCellular tumor antigen p53Homo sapiens (human)
replicative senescenceCellular tumor antigen p53Homo sapiens (human)
oxidative stress-induced premature senescenceCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
oligodendrocyte apoptotic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of execution phase of apoptosisCellular tumor antigen p53Homo sapiens (human)
negative regulation of mitophagyCellular tumor antigen p53Homo sapiens (human)
regulation of mitochondrial membrane permeability involved in apoptotic processCellular tumor antigen p53Homo sapiens (human)
regulation of intrinsic apoptotic signaling pathway by p53 class mediatorCellular tumor antigen p53Homo sapiens (human)
positive regulation of miRNA transcriptionCellular tumor antigen p53Homo sapiens (human)
negative regulation of G1 to G0 transitionCellular tumor antigen p53Homo sapiens (human)
negative regulation of miRNA processingCellular tumor antigen p53Homo sapiens (human)
negative regulation of glucose catabolic process to lactate via pyruvateCellular tumor antigen p53Homo sapiens (human)
negative regulation of pentose-phosphate shuntCellular tumor antigen p53Homo sapiens (human)
intrinsic apoptotic signaling pathway in response to hypoxiaCellular tumor antigen p53Homo sapiens (human)
regulation of fibroblast apoptotic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
positive regulation of reactive oxygen species metabolic processCellular tumor antigen p53Homo sapiens (human)
negative regulation of stem cell proliferationCellular tumor antigen p53Homo sapiens (human)
positive regulation of cellular senescenceCellular tumor antigen p53Homo sapiens (human)
positive regulation of intrinsic apoptotic signaling pathwayCellular tumor antigen p53Homo sapiens (human)
negative regulation of transcription by RNA polymerase IILysine-specific demethylase 5AHomo sapiens (human)
circadian regulation of gene expressionLysine-specific demethylase 5AHomo sapiens (human)
positive regulation of DNA-templated transcriptionLysine-specific demethylase 5AHomo sapiens (human)
regulation of DNA-binding transcription factor activityLysine-specific demethylase 5AHomo sapiens (human)
facultative heterochromatin formationLysine-specific demethylase 5AHomo sapiens (human)
regulation of DNA-templated transcriptionLysine-specific demethylase 5AHomo sapiens (human)
chromatin remodelingLysine-specific demethylase 5AHomo sapiens (human)
megakaryocyte differentiationFlavin reductase (NADPH)Homo sapiens (human)
heme catabolic processFlavin reductase (NADPH)Homo sapiens (human)
negative regulation of insulin receptor signaling pathwayFlavin reductase (NADPH)Homo sapiens (human)
regulation of heart rate by cardiac conductionPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of heart rate by hormonePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of membrane potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
positive regulation of DNA-templated transcriptionPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
potassium ion homeostasisPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
cardiac muscle contractionPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of membrane repolarizationPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of ventricular cardiac muscle cell membrane repolarizationPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
cellular response to xenobiotic stimulusPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
potassium ion transmembrane transportPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
ventricular cardiac muscle cell action potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
membrane repolarizationPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
membrane depolarization during action potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
membrane repolarization during action potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
membrane repolarization during cardiac muscle cell action potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of heart rate by cardiac conductionPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
potassium ion export across plasma membranePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
membrane repolarization during ventricular cardiac muscle cell action potentialPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
regulation of potassium ion transmembrane transportPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
negative regulation of potassium ion transmembrane transportPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
positive regulation of potassium ion transmembrane transportPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
negative regulation of potassium ion export across plasma membranePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
potassium ion import across plasma membranePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
immune system developmentVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
positive regulation of cytosolic calcium ion concentrationVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
heart developmentVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
regulation of cardiac muscle contraction by regulation of the release of sequestered calcium ionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
embryonic forelimb morphogenesisVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
camera-type eye developmentVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
positive regulation of adenylate cyclase activityVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
positive regulation of muscle contractionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
calcium ion transport into cytosolVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
cardiac conductionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
calcium ion transmembrane transport via high voltage-gated calcium channelVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
calcium ion transmembrane transportVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
cardiac muscle cell action potential involved in contractionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
membrane depolarization during cardiac muscle cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
membrane depolarization during AV node cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
cell communication by electrical coupling involved in cardiac conductionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
regulation of heart rate by cardiac conductionVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
regulation of ventricular cardiac muscle cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
membrane depolarization during atrial cardiac muscle cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
calcium ion import across plasma membraneVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
regulation of heart rateSodium channel protein type 5 subunit alphaHomo sapiens (human)
cardiac conduction system developmentSodium channel protein type 5 subunit alphaHomo sapiens (human)
cardiac ventricle developmentSodium channel protein type 5 subunit alphaHomo sapiens (human)
brainstem developmentSodium channel protein type 5 subunit alphaHomo sapiens (human)
sodium ion transportSodium channel protein type 5 subunit alphaHomo sapiens (human)
positive regulation of sodium ion transportSodium channel protein type 5 subunit alphaHomo sapiens (human)
response to denervation involved in regulation of muscle adaptationSodium channel protein type 5 subunit alphaHomo sapiens (human)
telencephalon developmentSodium channel protein type 5 subunit alphaHomo sapiens (human)
cerebellum developmentSodium channel protein type 5 subunit alphaHomo sapiens (human)
sodium ion transmembrane transportSodium channel protein type 5 subunit alphaHomo sapiens (human)
odontogenesis of dentin-containing toothSodium channel protein type 5 subunit alphaHomo sapiens (human)
positive regulation of action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
positive regulation of epithelial cell proliferationSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarizationSodium channel protein type 5 subunit alphaHomo sapiens (human)
cardiac muscle contractionSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of ventricular cardiac muscle cell membrane repolarizationSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of atrial cardiac muscle cell membrane depolarizationSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of atrial cardiac muscle cell membrane repolarizationSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of ventricular cardiac muscle cell membrane depolarizationSodium channel protein type 5 subunit alphaHomo sapiens (human)
cellular response to calcium ionSodium channel protein type 5 subunit alphaHomo sapiens (human)
cardiac muscle cell action potential involved in contractionSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of cardiac muscle cell contractionSodium channel protein type 5 subunit alphaHomo sapiens (human)
ventricular cardiac muscle cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during cardiac muscle cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
atrial cardiac muscle cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
SA node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
AV node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
bundle of His cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during AV node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during SA node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during Purkinje myocyte cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during bundle of His cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
AV node cell to bundle of His cell communicationSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of heart rate by cardiac conductionSodium channel protein type 5 subunit alphaHomo sapiens (human)
membrane depolarization during atrial cardiac muscle cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
regulation of sodium ion transmembrane transportSodium channel protein type 5 subunit alphaHomo 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)
potassium ion transportPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
protein homooligomerizationPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
membrane repolarizationPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
membrane repolarization during cardiac muscle cell action potentialPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
regulation of heart rate by cardiac conductionPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
potassium ion export across plasma membranePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
membrane repolarization during ventricular cardiac muscle cell action potentialPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
ventricular cardiac muscle cell membrane repolarizationPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
action potentialPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Molecular Functions (106)

Processvia Protein(s)Taxonomy
protein bindingLysine-specific demethylase 6BHomo sapiens (human)
beta-catenin bindingLysine-specific demethylase 6BHomo sapiens (human)
histone demethylase activityLysine-specific demethylase 6BHomo sapiens (human)
metal ion bindingLysine-specific demethylase 6BHomo sapiens (human)
histone H3K27me2/H3K27me3 demethylase activityLysine-specific demethylase 6BHomo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingLysine-specific demethylase 6BHomo sapiens (human)
chromatin DNA bindingLysine-specific demethylase 6BHomo sapiens (human)
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 bindingLysine-specific demethylase 4AHomo sapiens (human)
zinc ion bindingLysine-specific demethylase 4AHomo sapiens (human)
ubiquitin protein ligase bindingLysine-specific demethylase 4AHomo sapiens (human)
histone demethylase activityLysine-specific demethylase 4AHomo sapiens (human)
methylated histone bindingLysine-specific demethylase 4AHomo sapiens (human)
histone H3K36 demethylase activityLysine-specific demethylase 4AHomo sapiens (human)
histone H3K36me2/H3K36me3 demethylase activityLysine-specific demethylase 4AHomo sapiens (human)
histone H3K9me2/H3K9me3 demethylase activityLysine-specific demethylase 4AHomo sapiens (human)
histone H3K9 demethylase activityLysine-specific demethylase 4AHomo 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)
transcription cis-regulatory region bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
cis-regulatory region sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
core promoter sequence-specific DNA bindingCellular tumor antigen p53Homo sapiens (human)
TFIID-class transcription factor complex bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription repressor activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription activator activity, RNA polymerase II-specificCellular tumor antigen p53Homo sapiens (human)
protease bindingCellular tumor antigen p53Homo sapiens (human)
p53 bindingCellular tumor antigen p53Homo sapiens (human)
DNA bindingCellular tumor antigen p53Homo sapiens (human)
chromatin bindingCellular tumor antigen p53Homo sapiens (human)
DNA-binding transcription factor activityCellular tumor antigen p53Homo sapiens (human)
mRNA 3'-UTR bindingCellular tumor antigen p53Homo sapiens (human)
copper ion bindingCellular tumor antigen p53Homo sapiens (human)
protein bindingCellular tumor antigen p53Homo sapiens (human)
zinc ion bindingCellular tumor antigen p53Homo sapiens (human)
enzyme bindingCellular tumor antigen p53Homo sapiens (human)
receptor tyrosine kinase bindingCellular tumor antigen p53Homo sapiens (human)
ubiquitin protein ligase bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase regulator activityCellular tumor antigen p53Homo sapiens (human)
ATP-dependent DNA/DNA annealing activityCellular tumor antigen p53Homo sapiens (human)
identical protein bindingCellular tumor antigen p53Homo sapiens (human)
histone deacetylase bindingCellular tumor antigen p53Homo sapiens (human)
protein heterodimerization activityCellular tumor antigen p53Homo sapiens (human)
protein-folding chaperone bindingCellular tumor antigen p53Homo sapiens (human)
protein phosphatase 2A bindingCellular tumor antigen p53Homo sapiens (human)
RNA polymerase II-specific DNA-binding transcription factor bindingCellular tumor antigen p53Homo sapiens (human)
14-3-3 protein bindingCellular tumor antigen p53Homo sapiens (human)
MDM2/MDM4 family protein bindingCellular tumor antigen p53Homo sapiens (human)
disordered domain specific bindingCellular tumor antigen p53Homo sapiens (human)
general transcription initiation factor bindingCellular tumor antigen p53Homo sapiens (human)
molecular function activator activityCellular tumor antigen p53Homo sapiens (human)
promoter-specific chromatin bindingCellular tumor antigen p53Homo sapiens (human)
transcription cis-regulatory region bindingLysine-specific demethylase 5AHomo sapiens (human)
DNA bindingLysine-specific demethylase 5AHomo sapiens (human)
transcription coactivator activityLysine-specific demethylase 5AHomo sapiens (human)
enzyme inhibitor activityLysine-specific demethylase 5AHomo sapiens (human)
protein bindingLysine-specific demethylase 5AHomo sapiens (human)
zinc ion bindingLysine-specific demethylase 5AHomo sapiens (human)
chromatin DNA bindingLysine-specific demethylase 5AHomo sapiens (human)
histone demethylase activityLysine-specific demethylase 5AHomo sapiens (human)
histone H3K4me/H3K4me2/H3K4me3 demethylase activityLysine-specific demethylase 5AHomo sapiens (human)
methylated histone bindingLysine-specific demethylase 5AHomo sapiens (human)
histone bindingLysine-specific demethylase 5AHomo sapiens (human)
biliverdin reductase (NAD(P)H) activityFlavin reductase (NADPH)Homo sapiens (human)
protein bindingFlavin reductase (NADPH)Homo sapiens (human)
FMN reductase (NAD(P)H) activityFlavin reductase (NADPH)Homo sapiens (human)
peptidyl-cysteine S-nitrosylase activityFlavin reductase (NADPH)Homo sapiens (human)
riboflavin reductase (NADPH) activityFlavin reductase (NADPH)Homo sapiens (human)
FMN reductase (NADPH) activityFlavin reductase (NADPH)Homo sapiens (human)
FMN reductase (NADH) activityFlavin reductase (NADPH)Homo sapiens (human)
biliberdin reductase NAD+ activityFlavin reductase (NADPH)Homo sapiens (human)
biliverdin reductase (NADPH) activityFlavin reductase (NADPH)Homo sapiens (human)
transcription cis-regulatory region bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
inward rectifier potassium channel activityPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
voltage-gated potassium channel activityPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
delayed rectifier potassium channel activityPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
protein bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
ubiquitin protein ligase bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
identical protein bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
protein homodimerization activityPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
C3HC4-type RING finger domain bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
voltage-gated potassium channel activity involved in cardiac muscle cell action potential repolarizationPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
scaffold protein bindingPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
voltage-gated potassium channel activity involved in ventricular cardiac muscle cell action potential repolarizationPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
high voltage-gated calcium channel activityVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated calcium channel activity involved in cardiac muscle cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated calcium channel activityVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
protein bindingVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
calmodulin bindingVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
high voltage-gated calcium channel activityVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
metal ion bindingVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
alpha-actinin bindingVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated calcium channel activity involved in cardiac muscle cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated calcium channel activity involved in AV node cell action potentialVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated sodium channel activitySodium channel protein type 5 subunit alphaHomo sapiens (human)
protein bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
calmodulin bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
fibroblast growth factor bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
enzyme bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
protein kinase bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
protein domain specific bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
ankyrin bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
ubiquitin protein ligase bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
transmembrane transporter bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
nitric-oxide synthase bindingSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel activity involved in cardiac muscle cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel activity involved in AV node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel activity involved in bundle of His cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel activity involved in Purkinje myocyte action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel activity involved in SA node cell action potentialSodium channel protein type 5 subunit alphaHomo sapiens (human)
scaffold protein bindingSodium channel protein type 5 subunit alphaHomo 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)
A-type (transient outward) potassium channel activityPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
voltage-gated potassium channel activity involved in cardiac muscle cell action potential repolarizationPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
voltage-gated potassium channel activity involved in ventricular cardiac muscle cell action potential repolarizationPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
protein bindingPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
transmembrane transporter bindingPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
metal ion bindingPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
voltage-gated potassium channel activityPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Ceullar Components (60)

Processvia Protein(s)Taxonomy
nucleusLysine-specific demethylase 6BHomo sapiens (human)
nucleoplasmLysine-specific demethylase 6BHomo sapiens (human)
MLL3/4 complexLysine-specific demethylase 6BHomo sapiens (human)
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)
fibrillar centerLysine-specific demethylase 4AHomo sapiens (human)
nucleusLysine-specific demethylase 4AHomo sapiens (human)
nucleoplasmLysine-specific demethylase 4AHomo sapiens (human)
cytosolLysine-specific demethylase 4AHomo sapiens (human)
pericentric heterochromatinLysine-specific demethylase 4AHomo sapiens (human)
nucleusLysine-specific demethylase 4AHomo sapiens (human)
chromatinLysine-specific demethylase 4AHomo 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)
nuclear bodyCellular tumor antigen p53Homo sapiens (human)
nucleusCellular tumor antigen p53Homo sapiens (human)
nucleoplasmCellular tumor antigen p53Homo sapiens (human)
replication forkCellular tumor antigen p53Homo sapiens (human)
nucleolusCellular tumor antigen p53Homo sapiens (human)
cytoplasmCellular tumor antigen p53Homo sapiens (human)
mitochondrionCellular tumor antigen p53Homo sapiens (human)
mitochondrial matrixCellular tumor antigen p53Homo sapiens (human)
endoplasmic reticulumCellular tumor antigen p53Homo sapiens (human)
centrosomeCellular tumor antigen p53Homo sapiens (human)
cytosolCellular tumor antigen p53Homo sapiens (human)
nuclear matrixCellular tumor antigen p53Homo sapiens (human)
PML bodyCellular tumor antigen p53Homo sapiens (human)
transcription repressor complexCellular tumor antigen p53Homo sapiens (human)
site of double-strand breakCellular tumor antigen p53Homo sapiens (human)
germ cell nucleusCellular tumor antigen p53Homo sapiens (human)
chromatinCellular tumor antigen p53Homo sapiens (human)
transcription regulator complexCellular tumor antigen p53Homo sapiens (human)
protein-containing complexCellular tumor antigen p53Homo sapiens (human)
plasma membraneGlutamate receptor 2Rattus norvegicus (Norway rat)
nucleusLysine-specific demethylase 5AHomo sapiens (human)
nucleoplasmLysine-specific demethylase 5AHomo sapiens (human)
nucleolusLysine-specific demethylase 5AHomo sapiens (human)
nucleusLysine-specific demethylase 5AHomo sapiens (human)
chromatinLysine-specific demethylase 5AHomo sapiens (human)
cytoplasmFlavin reductase (NADPH)Homo sapiens (human)
nucleoplasmFlavin reductase (NADPH)Homo sapiens (human)
cytosolFlavin reductase (NADPH)Homo sapiens (human)
plasma membraneFlavin reductase (NADPH)Homo sapiens (human)
intracellular membrane-bounded organelleFlavin reductase (NADPH)Homo sapiens (human)
extracellular exosomeFlavin reductase (NADPH)Homo sapiens (human)
plasma membranePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
cell surfacePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
perinuclear region of cytoplasmPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
voltage-gated potassium channel complexPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
inward rectifier potassium channel complexPotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
plasma membranePotassium voltage-gated channel subfamily H member 2Homo sapiens (human)
cytoplasmVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
plasma membraneVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
postsynaptic densityVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
membraneVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
Z discVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
dendriteVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
perikaryonVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
postsynaptic density membraneVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
L-type voltage-gated calcium channel complexVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
voltage-gated calcium channel complexVoltage-dependent L-type calcium channel subunit alpha-1CHomo sapiens (human)
caveolaSodium channel protein type 5 subunit alphaHomo sapiens (human)
nucleoplasmSodium channel protein type 5 subunit alphaHomo sapiens (human)
nucleolusSodium channel protein type 5 subunit alphaHomo sapiens (human)
endoplasmic reticulumSodium channel protein type 5 subunit alphaHomo sapiens (human)
plasma membraneSodium channel protein type 5 subunit alphaHomo sapiens (human)
caveolaSodium channel protein type 5 subunit alphaHomo sapiens (human)
cell surfaceSodium channel protein type 5 subunit alphaHomo sapiens (human)
intercalated discSodium channel protein type 5 subunit alphaHomo sapiens (human)
membraneSodium channel protein type 5 subunit alphaHomo sapiens (human)
lateral plasma membraneSodium channel protein type 5 subunit alphaHomo sapiens (human)
Z discSodium channel protein type 5 subunit alphaHomo sapiens (human)
T-tubuleSodium channel protein type 5 subunit alphaHomo sapiens (human)
sarcolemmaSodium channel protein type 5 subunit alphaHomo sapiens (human)
perinuclear region of cytoplasmSodium channel protein type 5 subunit alphaHomo sapiens (human)
voltage-gated sodium channel complexSodium channel protein type 5 subunit alphaHomo 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)
plasma membranePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
sarcolemmaPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
GABA-ergic synapsePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
postsynaptic specialization membranePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
voltage-gated potassium channel complexPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
dendritic spinePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
neuronal cell bodyPotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
postsynaptic membranePotassium voltage-gated channel subfamily D member 3Homo sapiens (human)
[Information is prepared from geneontology information from the June-17-2024 release]

Bioassays (125)

Assay IDTitleYearJournalArticle
AID1347106qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for control Hh wild type fibroblast cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
AID1347096qHTS of pediatric cancer cell lines to identify multiple opportunities for drug repurposing: Primary screen for U-2 OS cells2018Oncotarget, Jan-12, Volume: 9, Issue:4
Quantitative high-throughput phenotypic screening of pediatric cancer cell lines identifies multiple opportunities for drug repurposing.
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.
AID651635Viability Counterscreen for Primary qHTS for Inhibitors of ATXN expression
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
AID1745845Primary qHTS for Inhibitors of ATXN expression
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.
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.
AID1384631Growth inhibition of Saccharomyces cerevisiae harboring FET3 deletion mutant at 400 uM measured for 11 hrs by HOP assay2018Journal of medicinal chemistry, 08-23, Volume: 61, Issue:16
Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol.
AID1872470Inhibition of full-length KDM5A (1 to 1090 residues)(unknown origin) using ARTK(me3)-QTARKSTGGKAPRKQLA-GGK(biotin) as substrate preincubated for 10 mins followed by substrate addition measured after 45 mins by LANCE Ultra assay2022European journal of medicinal chemistry, Mar-05, Volume: 231Drug discovery of histone lysine demethylases (KDMs) inhibitors (progress from 2018 to present).
AID1207276Inhibition of long-lasting type calcium current (ICaL) in HEK293 cells (alpha1C/beta2a/alpha2delta1) cells measured using IonWorks Barracuda automated patch clamp platform
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).
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).
AID1207304Inhibition of fast sodium current (INa) in Chinese Hamster Ovary (CHO) K1 cells transfected with human Nav1.5 measured using IonWorks Quattro automated patch clamp platform
AID561877Toxicity in patient with mucormycosis assessed as appearance of erythematous rashes at 5 to 20 mg/kg/day, po for 14 days2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID1207364Inhibition of slow delayed inward rectifying potassium current (Iks) in Chinese Hamster Ovary (CHO) cells expressing hKvLQT1/hminK measured using IonWorks Quattro automated patch clamp platform
AID1207336Inhibition of fast sodium current (INa) in HEK293 cells transfected with human Nav1.5 measured using IonWorks Quattro automated patch clamp platform
AID460413Inhibition of [59Fe] uptake from [59Fe]transferrin in human SK-N-MC cells assessed as at 50 uM after 3 hrs relative to untreated control2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Conjugates of desferrioxamine B (DFOB) with derivatives of adamantane or with orally available chelators as potential agents for treating iron overload.
AID1885303Inhibition of KDM4A in human KYSE-150 cells assessed as increase in H3K9me3 level2022Journal of medicinal chemistry, 07-28, Volume: 65, Issue:14
Recent Advances with KDM4 Inhibitors and Potential Applications.
AID1885304Antiproliferative activity against human KYSE-150 cells2022Journal of medicinal chemistry, 07-28, Volume: 65, Issue:14
Recent Advances with KDM4 Inhibitors and Potential Applications.
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.
AID1207490Inhibition of rapid delayed inward rectifying potassium current (IKr) in Chinese hamster ovary (CHO) cells stable expressing hERG measured using IonWorks Barracuda automated patch clamp platform
AID561867Toxicity in patient with mucormycosis assessed as creatinine level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 1.6 mg/dl)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID1384633Growth inhibition of Saccharomyces cerevisiae harboring GEF1 deletion mutant at 400 uM measured for 11 hrs by HOP assay2018Journal of medicinal chemistry, 08-23, Volume: 61, Issue:16
Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol.
AID561875Toxicity in patient with mucormycosis assessed as platelet count at 5 to 20 mg/kg/day, po for 14 days (Rvb = 217/uL)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID1872471Inhibition of KDM6B (1043 to end residues) (unknown origin) using ATKAARK(me3)-SAPATGGVKKPHRYRPG-GK(biotin) as substrate preincubated for 10 mins followed by substrate addition measured after 45 mins by LANCE Ultra assay2022European journal of medicinal chemistry, Mar-05, Volume: 231Drug discovery of histone lysine demethylases (KDMs) inhibitors (progress from 2018 to present).
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.
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.
AID1474132Drug concentration at steady state in human at 20 to 40 mg/kg, po QD after 24 hrs2013Toxicological 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.
AID1474134Ratio of drug concentration at steady state in human at 20 to 40 mg/kg, po QD after 24 hrs to IC50 for human MRP4 overexpressed in Sf9 insect cells2013Toxicological 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.
AID454622Cytotoxicity against human MIAPaCa2 cells after 96 hrs by MTT assay2010Bioorganic & medicinal chemistry letters, Jan-15, Volume: 20, Issue:2
Synthesis and antiproliferating activity of iron chelators of hydroxyamino-1,3,5-triazine family.
AID561872Toxicity in patient with mucormycosis assessed as WBC count at 5 to 20 mg/kg/day, po for 14 days (Rvb = 8.1/uL)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID1474133Ratio of drug concentration at steady state in human at 20 to 40 mg/kg, po QD after 24 hrs to IC50 for human BSEP overexpressed in Sf9 insect cells2013Toxicological 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.
AID1207518Inhibition of rapid delayed inward rectifying potassium current (IKr) measured using manual patch clamp assay
AID561874Toxicity in patient with mucormycosis assessed as hemoglobin level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 9 mg/dl)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
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).
AID1207396Inhibition of slow delayed inward rectifying potassium current (Iks) in Chinese Hamster Ovary (CHO) cells transfected with KCNQ1 / Kv1.7 / KvLQT1 and KCNE1/minK measured using IonWorks automated patch clamp platform
AID1815674Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLVRB expressed in Escherichia coli BL21 (DE3) assessed as change in gibbs free energy by isothermal titration calorimetry2022Journal of medicinal chemistry, 02-10, Volume: 65, Issue:3
Repositioning Food and Drug Administration-Approved Drugs for Inhibiting Biliverdin IXβ Reductase B as a Novel Thrombocytopenia Therapeutic Target.
AID1885301Inhibition of KDM4A (unknown origin) measured by formaldehyde dehydrogenase (FDH)-coupled fluorescence assay2022Journal of medicinal chemistry, 07-28, Volume: 65, Issue:14
Recent Advances with KDM4 Inhibitors and Potential Applications.
AID1207424Inhibition of transient outward potassium current (Ito) current in Chinese Hamster Ovary (CHO) K1 cells expressing human Kv4.3 measured using IonWorks Quattro automated patch clamp platform
AID1815675Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLVRB expressed in Escherichia coli BL21 (DE3) assessed as dissociation constant by isothermal titration calorimetry2022Journal of medicinal chemistry, 02-10, Volume: 65, Issue:3
Repositioning Food and Drug Administration-Approved Drugs for Inhibiting Biliverdin IXβ Reductase B as a Novel Thrombocytopenia Therapeutic Target.
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).
AID1815672Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLVRB expressed in Escherichia coli BL21 (DE3) assessed as change in enthalpy by isothermal titration calorimetry2022Journal of medicinal chemistry, 02-10, Volume: 65, Issue:3
Repositioning Food and Drug Administration-Approved Drugs for Inhibiting Biliverdin IXβ Reductase B as a Novel Thrombocytopenia Therapeutic Target.
AID419505Volume of distribution at steady state in human at 130 mg administered 90 mins infusion2009Journal of medicinal chemistry, Jul-23, Volume: 52, Issue:14
In silico prediction of volume of distribution in human using linear and nonlinear models on a 669 compound data set.
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.
AID561869Toxicity in patient with mucormycosis assessed as aspartate transaminase level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 27 U/l)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
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).
AID561878Toxicity in patient with mucormycosis assessed as alanine transaminase level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 21 U/l)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
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.
AID454623Cytotoxicity against human foreskin fibroblasts after 96 hrs by MTT assay2010Bioorganic & medicinal chemistry letters, Jan-15, Volume: 20, Issue:2
Synthesis and antiproliferating activity of iron chelators of hydroxyamino-1,3,5-triazine family.
AID561876Toxicity in patient with mucormycosis assessed as glucose level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 131 mg/dl)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
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.
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.
AID454621Cytotoxicity against human MDA-MB-231 cells after 96 hrs by MTT assay2010Bioorganic & medicinal chemistry letters, Jan-15, Volume: 20, Issue:2
Synthesis and antiproliferating activity of iron chelators of hydroxyamino-1,3,5-triazine family.
AID1885305Antiproliferative activity against human HL-60 cells2022Journal of medicinal chemistry, 07-28, Volume: 65, Issue:14
Recent Advances with KDM4 Inhibitors and Potential Applications.
AID460407Partition coefficient, log P by shake flask method2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Conjugates of desferrioxamine B (DFOB) with derivatives of adamantane or with orally available chelators as potential agents for treating iron overload.
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).
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).
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.
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).
AID1829741Cytotoxicity against human NCI-H460 cells assessed as reduction in cell viability incubated for 24 hrs by CCK8 assay
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).
AID561868Toxicity in patient with mucormycosis assessed as blood urea nitrogen level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 34 mg/dl)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID460410Toxicity in human SK-N-MC cells by MTT method2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Conjugates of desferrioxamine B (DFOB) with derivatives of adamantane or with orally available chelators as potential agents for treating iron overload.
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).
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).
AID1829742Cytotoxicity against human NCI-H460 cells assessed as reduction in cell viability incubated for 48 hrs by CCK8 assay
AID1207552Inhibition of long-lasting type calcium current (hICa) in Chinese Hamster Ovary (CHO) cells expressing hCav1.2 measured using IonWorks Quattro automated patch clamp platform
AID1384635Growth inhibition of wild type Saccharomyces cerevisiae BY4743 at 100 uM measured for 15 hrs in presence of Fe2+2018Journal of medicinal chemistry, 08-23, Volume: 61, Issue:16
Yeast Chemogenomic Profiling Reveals Iron Chelation To Be the Principle Cell Inhibitory Mode of Action of Gossypol.
AID1815673Binding affinity to human N-terminal His6-tagged and thrombin cleavage fused BLVRB expressed in Escherichia coli BL21 (DE3) assessed as change in entropy by isothermal titration calorimetry2022Journal of medicinal chemistry, 02-10, Volume: 65, Issue:3
Repositioning Food and Drug Administration-Approved Drugs for Inhibiting Biliverdin IXβ Reductase B as a Novel Thrombocytopenia Therapeutic Target.
AID1207458Inhibition of rapid delayed inward rectifying potassium current (IKr) in Chinese hamster ovary (CHO) K1 cells stably expressing hERG measured using IonWorks Quattro automated patch clamp platform
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).
AID1885302Inhibition of KDM4A (unknown origin) measured by LANCEUltra assay2022Journal of medicinal chemistry, 07-28, Volume: 65, Issue:14
Recent Advances with KDM4 Inhibitors and Potential Applications.
AID561873Toxicity in patient with mucormycosis assessed as absolute neutrophil count at 5 to 20 mg/kg/day, po for 14 days (Rvb = 5.2/uL)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID1474131AUC in human at 20 to 40 mg/kg, po QD after 24 hrs2013Toxicological 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.
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.
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.
AID1872469Inhibition of KDM4A (1 to 359 residues) (unknown origin) using ARTKQTARK(me3)-STGGKAPRKQLA-GGK(biotin) peptide as substrate preincubated for 10 mins followed by substrate addition measured after 45 mins by LANCE Ultra assay2022European journal of medicinal chemistry, Mar-05, Volume: 231Drug discovery of histone lysine demethylases (KDMs) inhibitors (progress from 2018 to present).
AID460409Toxicity in MDCK cells by MTT method2010Journal of medicinal chemistry, Feb-11, Volume: 53, Issue:3
Conjugates of desferrioxamine B (DFOB) with derivatives of adamantane or with orally available chelators as potential agents for treating iron overload.
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).
AID561870Toxicity in patient with mucormycosis assessed as alkaline phosphatase level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 126 U/l)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
AID561871Toxicity in patient with mucormycosis assessed as total bilirubin level at 5 to 20 mg/kg/day, po for 14 days (Rvb = 0.8 mg/dl)2009Antimicrobial agents and chemotherapy, Jul, Volume: 53, Issue:7
Safety and outcomes of open-label deferasirox iron chelation therapy for mucormycosis.
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).
AID1347170Vero cells viability counterscreen for qRT-PCR qHTS assay of selected 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.
AID1347149Furin counterscreen 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.
AID1347153Confirmatory 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.
AID1347171Orthogonal mCherry assay for qRT-PCR qHTS of selected 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.
AID1347164384 well plate NINDS Rhodamine confirmatory 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.
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.
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.
AID1347152Confirmatory screen NINDS 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.
AID1347161Confirmatory screen NINDS Rhodamine 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.
AID1347150Optimization screen NINDS AMC qHTS for Zika virus inhibitors: Linked NS2B-NS3 protease assay2020Proceedings 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.
AID1347151Optimization of GU AMC qHTS for Zika virus inhibitors: Unlinked NS2B-NS3 protease assay2020Proceedings 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.
AID1347168HepG2 cells viability 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.
AID1347163384 well plate NINDS AMC confirmatory 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.
AID1347172Secondary qRT-PCR qHTS assay for selected 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.
AID1347167Vero cells viability 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.
AID1347169Tertiary RLuc qRT-PCR qHTS assay 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.
AID1347155Optimization screen NINDS Rhodamine qHTS for Zika virus inhibitors: Linked NS2B-NS3 protease assay2020Proceedings 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.
AID1347158ZIKV-mCherry secondary 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.
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.
[information is prepared from bioassay data collected from National Library of Medicine (NLM), extracted Dec-2023]

Research

Studies (804)

TimeframeStudies, This Drug (%)All Drugs %
pre-19900 (0.00)18.7374
1990's0 (0.00)18.2507
2000's157 (19.53)29.6817
2010's522 (64.93)24.3611
2020's125 (15.55)2.80
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Market Indicators

Research Demand Index: 77.80

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 Index77.80 (24.57)
Research Supply Index6.88 (2.92)
Research Growth Index6.91 (4.65)
Search Engine Demand Index134.36 (26.88)
Search Engine Supply Index2.00 (0.95)

This Compound (77.80)

All Compounds (24.57)

Study Types

Publication TypeThis drug (%)All Drugs (%)
Trials136 (16.35%)5.53%
Reviews126 (15.14%)6.00%
Case Studies111 (13.34%)4.05%
Observational20 (2.40%)0.25%
Other439 (52.76%)84.16%
[information is prepared from research data collected from National Library of Medicine (NLM), extracted Dec-2023]

Clinical Trials (93)

Trial Overview

TrialPhaseEnrollmentStudy TypeStart DateStatus
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Other Patients With Rare Chronic Anemia and Transfusional Iron Overload [NCT00303329]Phase 2184 participants (Actual)Interventional2004-03-31Completed
A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators [NCT00235391]Phase 31,683 participants (Actual)Interventional2005-10-31Completed
A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients With Iron Overload [NCT02435212]Phase 2224 participants (Actual)Interventional2015-10-21Active, not recruiting
Open-label, Multicenter, Single Arm, Phase II Study Assessing Treatment Patient Preference for New Deferasirox Formulation (Film-coated Tablet) Compared to the Reference Deferasirox Dispersible Tablet Formulation [NCT02993224]Phase 2148 participants (Actual)Interventional2017-07-27Completed
A Phase II Study of Deferasirox in Patients With Myelodysplastic Syndromes Who Are Anemic With Iron Overload [NCT02943668]Phase 22 participants (Actual)Interventional2017-03-02Terminated(stopped due to Terminated due to low accrual)
Multicenter Prospective Observational Study of the Outcome of Patients With Acute Myeloblastic Leukemia (AML) and Myelodysplastic Syndrome (MDS) Receiving Iron Chelation Therapy (Exjade) After Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) [NCT03659084]150 participants (Anticipated)Observational2016-04-30Recruiting
Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron Overload Patients With β-Thalassemia and Intermediate Thalassemia in Bandarabbas [NCT01369719]138 participants (Actual)Interventional2010-02-28Completed
A PHASE II STUDY OF Azacitidine Plus Deferasirox (ICL670) in Higher Risk Myelodysplastic Syndromes (MDS) [NCT02038816]Phase 21 participants (Actual)Interventional2014-03-31Terminated(stopped due to accrual too slow, insufficient patients)
A Single-arm Interventional Phase IV, Post-authorisation Study Evaluating the Safety of Pediatric Patients With Transfusional Hemosiderosis Treated With Deferasirox Crushed Film Coated Tablets [NCT03372083]Phase 444 participants (Actual)Interventional2018-01-16Completed
Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload [NCT01335035]Phase 430 participants (Actual)Interventional2008-12-31Completed
Effects of Iron Loading and Iron Chelation Therapy on Innate Immunity During Human Endotoxemia [NCT01349699]30 participants (Actual)Interventional2010-02-28Completed
A Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron Overload [NCT01284946]Phase 245 participants (Anticipated)Interventional2011-01-31Recruiting
An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-Risk and INT-1 Myelodysplastic Patients [NCT00117507]Phase 424 participants (Actual)Interventional2005-09-30Completed
The Use of the Calcium Channel Blocker Amlodipine as an Adjuvant Treatment to Iron Chelation for the Prevention of Iron Overload Cardiomyopathy in Patients With Thalassemia [NCT02474420]60 participants (Anticipated)Interventional2015-06-30Recruiting
Low Dose Iron Chelation as TReatment of Oxidative Damage in Patients With Sickle Cell Disease: the TROS Study [NCT05392101]Phase 212 participants (Actual)Interventional2021-07-20Completed
A Multicenter, Open-label, Single Arm, Interventional Phase IV Study, to Evaluate the Effect of Deferasirox on Endocrine Complications in Subjects With Transfusion Dependent Thalassemia [NCT02069886]Phase 40 participants (Actual)Interventional2014-12-31Withdrawn
The Shuttle Effect : Combination Therapy With Deferiprone and Deferasirox in Transfusion-dependent Thalassemia Patients. [NCT02198508]13 participants (Actual)Interventional2007-07-31Completed
Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox) [NCT02233504]Early Phase 125 participants (Anticipated)Interventional2014-08-31Completed
Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea Tarda [NCT00599326]Phase 310 participants (Actual)Interventional2008-01-31Completed
1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload. [NCT01250951]Phase 4111 participants (Actual)Interventional2009-12-31Completed
A Multi-Center, Open-Label, Two-Period Cross-Over, Patient-Pilot Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Iron Chelating Activity of DST-0509 (Deferasirox) Tablets in Thalassemia Patients With Inadequate Response to Standard Chelat [NCT03637556]Phase 225 participants (Actual)Interventional2019-08-20Completed
Early and Low Dose Deferasirox (3.5 mg/kg FCT) to Suppress NTBI and LPI as Early Intervention to Prevent Tissue Iron Overload in Lower Risk MDS [NCT03920657]Phase 211 participants (Actual)Interventional2019-10-04Terminated(stopped due to enrollment failed)
Open-label, Multicenter, Single Arm, Phase III Study to Collect Additional Safety and Efficacy Data With Deferasirox Film-coated Tablets in Patients Completing Study CICL670F2201 [NCT02720536]Phase 353 participants (Actual)Interventional2016-08-16Completed
A Multicenter, Randomized, Comparative Study of Different Deferasirox Administration Regimens on Gastrointestinal (GI) Tolerability in Low or Intermediate (Int-1) Risk MDS Myelodysplastic Syndrome Patients With Transfusional Iron Overload. [NCT01326845]Phase 412 participants (Actual)Interventional2011-12-31Terminated(stopped due to The trial was terminated due to insufficient enrollment.)
A 5-year Open Label, Non-comparative Extension to a Randomized, Open-label, Phase IIa Study to Evaluate Safety, Tolerability and the Effects on Liver Iron Concentration of Repeated Doses of 10 and 20 mg/kg/Day of Deferasirox in Comparison With 40 mg/kg/Da [NCT01033747]Phase 2/Phase 370 participants (Actual)Interventional2003-02-28Completed
A Single-arm, Open-label Study of the Palatability and Tolerability of Deferasirox Taken With Meals, With Different Liquids or Crushed and Added to Food [NCT00845871]Phase 465 participants (Actual)Interventional2009-05-31Completed
An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months [NCT00447694]Phase 230 participants (Actual)Interventional2006-02-28Completed
An Iron Balance Study Comparing Deferasirox, Deferoxamine and the Combination of Both Drugs [NCT00738413]Phase 1/Phase 26 participants (Anticipated)Interventional2008-08-31Recruiting
A Randomized, Open-label, Multi-center, Phase II Study to Evaluate the Safety and Efficacy of Deferasirox (ICL670) 20 mg/kg/Day Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients With Iron Overload From Repeated Blood Transfusions [NCT00110617]Phase 2212 participants (Actual)Interventional2005-05-31Completed
A Multicenter, Randomized, Open-label Phase II Trial Evaluating Deferasirox Compared With Deferoxamine in Patients With Cardiac Iron Overload Due to Chronic Blood Transfusions [NCT00600938]Phase 2197 participants (Actual)Interventional2007-11-30Completed
A Study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in Patients With Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or Other Anaemias Treated With Exjade® (Deferasirox) (The MILE Study) [NCT00673608]Phase 4118 participants (Actual)Interventional2007-11-30Completed
A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/d) in Patients Three to Six Months After Allogeneic Hematopoietic Cell Transplantation in Whom Iron Overload is Present [NCT00654589]Phase 475 participants (Actual)Interventional2008-02-29Completed
A Randomized, Open Label, Phase II Study on Safety and Efficacy of Long Term Treatment of ICL670 Relative to Deferoxamine in Sickle Cell Disease Patients With Transfusional Hemosiderosis [NCT00067080]Phase 2195 participants (Actual)Interventional2003-05-31Completed
A Phase I/II Open Label, Dose Escalation Trial and a Six Month Extension to Explore the Safety and Efficacy of ICL670 in Patients With Iron Overload Resulting From Hereditary Hemochromatosis. [NCT00395629]Phase 1/Phase 249 participants (Actual)Interventional2006-08-31Completed
Phase I Study to Examine the Effect of Deferasirox on Renal Hemodynamics in β-thalassemia Patients With Transfusional Iron Overload [NCT00560820]Phase 111 participants (Actual)Interventional2007-09-30Completed
International Sentinel Site Surveillance of Patients With Transfusional Hemosiderosis Treated With Deferasirox in Actual Practice Setting [NCT01394029]120 participants (Actual)Observational2011-07-31Completed
Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major [NCT02761395]60 participants (Anticipated)Interventional2015-11-30Recruiting
A Phase II, Multicenter, Open-label, Randomized Two-year Study to Evaluate the Efficacy and Safety of Deferasirox Film-coated Tablet Versus Phlebotomy in Patients With Hereditary Hemochromatosis. [NCT03203850]Phase 245 participants (Actual)Interventional2018-01-11Completed
A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity [NCT01044186]Phase 230 participants (Actual)Interventional2003-06-30Completed
A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferasirox in Pediatric Patients With Transfusion Dependent β-th [NCT00390858]Phase 240 participants (Actual)Interventional2003-09-30Completed
An Open Label, Safety and Tolerability Study of Deferasirox for Treatment of Transfusional Iron Overload in Low-risk and INT-1, Myelodysplastic Patients Using Serum Ferritin Monitoring [NCT00110266]Phase 2176 participants (Actual)Interventional2005-07-25Completed
Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox [NCT01905774]36 participants (Actual)Observational2011-03-31Completed
A Prospective Single Arm Study to Assess the Efficacy and Safety of Deferasirox 20 mg/kg BID in Transfusion Dependent Thalassemia Patients Inadequately Responding to Current Treatment With Doses > 35mg/kg QD (Once a Day). [NCT01948817]Phase 20 participants (Actual)Interventional2014-02-28Withdrawn
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload [NCT00564941]Phase 4309 participants (Anticipated)Interventional2007-12-31Completed
An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670)in β-thalassemia Patients With Transfusional Iron Overload [NCT00171210]Phase 3506 participants (Actual)Interventional2004-10-31Completed
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate Efficacy and Safety of Deferasirox in Non-transfusion-dependent Thalassemia Patients With Iron Overload [NCT00873041]Phase 2166 participants (Actual)Interventional2008-11-30Completed
Phase II, Open-label, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of Deferasirox in Combination With Deferoxamine Followed by Deferasirox Monotherapy in Patients With Severe Cardiac Iron Overload Due to Chronic Blood Transfusion (HYP [NCT01254227]Phase 260 participants (Actual)Interventional2011-01-31Completed
A One Year Open Label, Non-comparative Extension to a Randomized, Multicenter, Phase II Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration (LIC) of Repeated Doses of 5-30mg/kg/Day ICL670 Relative to D [NCT01090323]Phase 2185 participants (Actual)Interventional2004-07-31Completed
Phase 2 Study of Deferasirox-calcium-vitamin D3 to Treat Postmenopausal Osteoporosis (PMOP) [NCT02854722]Phase 210 participants (Anticipated)Interventional2018-01-15Recruiting
Multicenter, Open Label, Prospective Study to Evaluate the Efficacy and Safety of Deferasirox 30 mg/kg/Day for 52 Weeks, in Transfusion-dependent Beta-thalassemic Patients With Cardiac MRI T2* < 20 Msec [NCT00879242]Phase 220 participants (Actual)Interventional2009-02-28Completed
A Multi-center, Randomized, Double-blind, Placebo-controlled Clinical Trial of Deferasirox in Patients With Myelodysplastic Syndromes (Low/Int-1 Risk) and Transfusional Iron Overload [NCT00940602]Phase 2225 participants (Actual)Interventional2010-03-22Completed
The Deferasirox-AmBisome Therapy for Mucormycosis (DEFEAT Mucor) Study [NCT00419770]Phase 220 participants (Actual)Interventional2007-10-31Completed
Early Treatment With Deferasirox (Exjade®) in Low Risk MDS - a Prospective Multicentre Single-arm Single-stage Phase II Study - [NCT01058369]Phase 22 participants (Actual)Interventional2010-04-30Terminated(stopped due to Insufficient patient recruitment (only 2 patients))
An Open-label, One-sequence Cross-over Study to Investigate the Effect of Deferasirox on the Pharmacokinetics of Midazolam in Healthy Volunteers [NCT00427505]Phase 122 participants Interventional2006-07-31Completed
Open Label, Multicenter Study to Evaluate Safety/Tolerability and Efficacy of Deferasirox (ICL670) in Myelodysplastic Syndrome Patients With Chronic Transfusional Hemosiderosis. [NCT00469560]Phase 3158 participants (Actual)Interventional2007-06-30Completed
A One-year, Open-label, Single Arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 in Patients Diagnosed With Low and INT-1 Risk Myelodysplastic Syndrome (MDS) and Transfusion-dependent Iron Overload [NCT00481143]Phase 463 participants (Actual)Interventional2007-05-31Completed
Identification of Risk Factors and Measures to Prevent Liver and Pancreas Complications in Pediatric Patients Undergoing a Hematopoietic Stem Cell Transplant (HSCT) [NCT04423237]39 participants (Anticipated)Observational2020-09-30Recruiting
A Multicenter Open Label Phase II Study to Evaluate the Safety and Efficacy of Deferasirox in Combination With Deferoxamine Followed by Transitioning to Deferasirox Monotherapy in β-thalassemia Patients With Severe Cardiac Iron Overload [NCT01459718]Phase 232 participants (Actual)Interventional2011-01-31Terminated(stopped due to The study terminated due to low enrollment.)
A 5 Year Observational Study (Registry) of Children Aged 2 to <6 Years at Enrollment With Transfusional Hemosiderosis Treated With Deferasirox [NCT00466063]108 participants (Actual)Observational2007-05-31Completed
A Study of Efficacy and Safety of Long-term Treatment With Deferasirox in Patients With Beta-thalassemia and Transfusional Hemosiderosis [NCT00171171]Phase 3252 participants (Actual)Interventional2004-05-31Completed
A One Year, Open-label, Single-arm, Multi-center Trial Evaluating the Efficacy and Safety of Oral ICL670 (20 mg/kg/Day) in Patients Diagnosed With Transfusion-dependent Iron Overload [NCT00171821]Phase 31,784 participants (Actual)Interventional2005-04-30Completed
A Prospective Randomized Comparative Study of Efficacy and Safety of Combined Deferiprone (DFP) and Deferasirox Versus DFP and Desferrioxamine (DFO) Therapy in Diseases With Severe Iron Overload [NCT01511848]Phase 2/Phase 360 participants (Anticipated)Interventional2012-02-29Not yet recruiting
A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in B-thalassemia Subjects Administered SPD602 (SSP-004184AQ) or Exjade® (Deferasirox) for [NCT01927913]Phase 20 participants (Actual)Interventional2014-11-20Withdrawn(stopped due to This study was withdrawn until the evaluation of the nonclinical rat findings is complete.)
A Randomized, Comparative, Open Label Phase III Trial on Efficacy & Safety of Long-term Treatment With ICL670 Compared to Deferoxamine in Beta-thalassemia Patients With Transfusional Hemosiderosis [NCT00061750]Phase 3595 participants (Actual)Interventional2003-05-31Completed
Phase II Study of Safety & Efficacy of Deferasirox Given for 1 Year in Patients With Chronic Anemias and Transfusional Hemosiderosis Unable to be Treated With Deferoxamine [NCT00061763]Phase 2175 participants (Actual)Interventional2003-05-31Completed
Pilot Pharmacokinetic Study In Patients With Inadequate Response To Deferasirox (Exjade) [NCT00749515]Phase 415 participants (Actual)Interventional2008-03-31Completed
Deferasirox Treatment and Labile Plasma Iron in Iron Overloaded Patients Who Have Undergone Allogeneic Hematopoietic Stem Cell Transplantation [NCT01159067]Phase 21 participants (Actual)Interventional2010-07-31Terminated(stopped due to Low enrollment)
Multicentre, Randomised, Open Label, Non-inferiority Trial to Evaluate the Efficacy and Safety of Deferiprone Compared to Deferasirox in Patients Aged From 1 Month to Less Than 18 Years Affected by Transfusion Dependent Haemoglobinopathies [NCT01825512]Phase 3435 participants (Actual)Interventional2014-03-17Completed
1-year Extension to CICL670A2402 an Open-label, Multi-center Trial of the Efficacy and Safety of Long-term Treatment With Deferasirox (10 to 20 mg/kg/Day) in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2- Year Duration) [NCT00171301]Phase 4233 participants (Actual)Interventional2005-06-30Completed
A Phase I, Single Center, Open-label, One-sequence Cross-over Study to Investigate the Effect of a Potent Inducer Rifampicin on the Pharmacokinetics of Deferasirox in Healthy Volunteers [NCT00419172]Phase 122 participants (Anticipated)Interventional2007-01-31Completed
A Phase IV , Multicenter ,Open Label ,Non Comparative ,Investigator Initiated Study , Evaluating the Effect of Exjade on Oxidative Stress in Low Risk Myelodysplastic Syndrome Patients With Iron Over Load [NCT00452660]Phase 421 participants (Actual)Interventional2007-05-31Completed
The Impact of Deferasirox on Non-Alcoholic-Steatohepatitis (NASH) - a Prospective Open Label Phase I/II Trial [NCT01278056]Phase 1/Phase 25 participants (Actual)Interventional2010-03-31Completed
Phase I-II Study of Association of Deferasirox, Vitamin D and Azacytidine as Treatment of High Risk MDS (IPSS Int-2 and High) [NCT01718366]Phase 1/Phase 250 participants (Actual)Interventional2013-02-28Active, not recruiting
Impact of Intervention With Deferasirox on the Immune Function of Patients With Hematologic Diseases and Transfusion-Related Iron Overload [NCT01273766]Phase 216 participants (Actual)Interventional2011-01-31Completed
Open-label Study of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients Undergoing Treatment Programs in Comparison With Control Group [NCT01818726]Phase 415 participants (Actual)Interventional2014-06-23Terminated(stopped due to Failure to meet the schedule of patient recruitment)
A Phase II Pilot Study to Assess the Presence of Molecular Factors Predictive for Hematologic Response in Myelodysplastic Syndrome Patients Receiving Deferasirox Therapy in Hematological Centers in Belgium Using Gene Expressing Profiling From Baseline Bon [NCT02663752]Phase 21 participants (Actual)Interventional2016-05-30Terminated
Clinical Importance of Treating Iron Overload in Sickle Cell Disease [NCT00981370]Phase 31 participants (Actual)Interventional2009-04-30Terminated(stopped due to 1 consented patient never started on study drug, lost to follow up)
A Multi-Center, Open-label, Non Comparative, Phase II Trial on Efficacy and Safety of ICL670 Given for 1 Year With Dose Adjustments Based on Serum Ferritin in Patients With Chronic Anemia and Transfusional Hemosiderosis Including an Additional 1 Year Exte [NCT00631163]Phase 2102 participants (Actual)Interventional2007-10-31Completed
Safety of Deferasirox (ICL670) and Deferoxamine (Desferal or DFO) Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload [NCT00901199]Phase 222 participants (Actual)Interventional2007-09-30Completed
An Open-label, Single-dose, Parallel-group Study to Assess the Pharmacokinetics of 20 mg/kg Oral Deferasirox in Patients With Impaired Hepatic Function and Healthy Subjects With Normal Hepatic Function [NCT00432627]Phase 124 participants (Actual)Interventional2006-12-31Completed
An Open-label, Phase II, Randomized, Pilot Study to Assess the Effect in Term of Erythroid Improvement of Deferasirox Combined With Erythropoietin Compared to Erythropoietin Alone in Patients With low-and Int-1-risk Myelodysplastic Syndrome. [NCT01868477]Phase 228 participants (Actual)Interventional2014-01-28Completed
A Phase II Study Examining the Use of Deferasirox, Cholecalciferol, and Azacitidine in the Treatment of Newly Diagnosed Acute Myelogenous Leukemia (AML) in Elderly Patients [NCT02341495]Phase 24 participants (Actual)Interventional2013-02-28Terminated(stopped due to Study was terminated early due to low patient accrual.)
Extension Study of Iron Chelation Therapy With Deferasirox in Patients With Transfusional Iron Overload [NCT00379483]Phase 266 participants (Actual)Interventional2002-07-31Completed
A 2-year, Multi-center, Phase II, Open-label, Fixed-dose, Randomized Comparative Trial of Azacitidine, With or Without Deferasirox in Patients With Higher Risk Myelodysplastic Syndromes [NCT02159040]Phase 21 participants (Actual)Interventional2014-09-11Terminated
Phase 1 Study of Deferasirox in Acute Leukemia Patients Not Treated by Standard Chemotherapy Regimens [NCT02413021]Phase 140 participants (Anticipated)Interventional2016-05-31Not yet recruiting
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old [NCT01724138]Phase 40 participants (Actual)Interventional2013-06-30Withdrawn
Deferasirox Versus Venesection in Patients With Hemochromatosis and for Treatment of Transfusional Siderosis in Myelodysplastic Syndrome: Diagnostics and New Biomarkers. [NCT01892644]Phase 250 participants (Actual)Interventional2013-05-31Terminated(stopped due to Failure to recruit patients with hemochromatosis to the Deferasirox arm)
Open-Label Single-Arm Pilot Study of Deferasirox (Exjade®) in Adult Allogeneic Hematopoietic Stem Cell Transplant Recipients With Transfusional Iron Overload [NCT00602446]Phase 24 participants (Actual)Interventional2007-08-31Terminated(stopped due to Due to slow accrual of patients)
A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation. [NCT01610297]Phase 427 participants (Actual)Interventional2013-09-30Completed
Pilot Study of Deferasirox and Deferiprone Combination Oral Chelation for Individuals With Transfusion Dependent Thalassemia and High Iron Burden [NCT01709032]Phase 1/Phase 29 participants (Actual)Interventional2012-09-30Completed
An Open Label, Multi-center, Efficacy and Safety Study of Deferasirox in Iron Overloaded Patients With Non-transfusion Dependent Thalassemia [NCT01709838]Phase 4134 participants (Actual)Interventional2012-12-06Completed
Phase II Trial Evaluating Low-dose Deferasirox (DFX) in Patients With Low-risk (MDS) Myelodysplastic Syndrome Resistant or Relapsing After ESA Agents (LODEFI) [NCT03387475]Phase 239 participants (Anticipated)Interventional2018-02-20Recruiting
A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet) [NCT02125877]Phase 2173 participants (Actual)Interventional2014-07-08Completed
[information is prepared from clinicaltrials.gov, extracted Sep-2024]

Trial Outcomes

TrialOutcome
NCT00110266 (12) [back to overview]Treatment Compliance to Deferasirox
NCT00110266 (12) [back to overview]Trough Plasma Deferasirox Concentration
NCT00110266 (12) [back to overview]Number of Participants Reporting Adverse Events
NCT00110266 (12) [back to overview]Change in Labile Plasma Iron (LPI)
NCT00110266 (12) [back to overview]Total Iron Levels
NCT00110266 (12) [back to overview]Change in Serum Ferritin From Baseline to Weeks 13, 25, 37 and 53
NCT00110266 (12) [back to overview]Directly Chelatable Iron (DCI)
NCT00110266 (12) [back to overview]Frequency of Hematologic Improvement During the Study
NCT00110266 (12) [back to overview]Serum Transferrin Levels
NCT00110266 (12) [back to overview]Transfusion Requirements
NCT00110266 (12) [back to overview]Transferrin Saturation
NCT00110266 (12) [back to overview]The Prevalence of Hereditary Hemochromatosis Gene (HFE) Gene Mutations
NCT00110617 (4) [back to overview]Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104
NCT00110617 (4) [back to overview]The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment
NCT00110617 (4) [back to overview]Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52
NCT00110617 (4) [back to overview]Absolute Change in Serum Ferritin From Baseline to Week 24
NCT00117507 (8) [back to overview]Number of Participants With Adverse Events and Serious Adverse Events
NCT00117507 (8) [back to overview]Labile Plasma Iron (LPI)
NCT00117507 (8) [back to overview]Absolute Change in Urinary Hepcidin
NCT00117507 (8) [back to overview]Absolute Change in Transferrin Saturation
NCT00117507 (8) [back to overview]Absolute Change in Serum Ferritin From Baseline to Week 52
NCT00117507 (8) [back to overview]Absolute Change in Liver Iron Concentration (LIC) From Baseline to End of Study
NCT00117507 (8) [back to overview]To Evaluate Change in Transfusion Requirements
NCT00117507 (8) [back to overview]Absolute Change in Serum Erythropoietin
NCT00171210 (12) [back to overview]Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
NCT00171210 (12) [back to overview]Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy
NCT00171210 (12) [back to overview]Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID
NCT00171210 (12) [back to overview]Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study
NCT00171210 (12) [back to overview]Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study
NCT00171210 (12) [back to overview]Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study
NCT00171210 (12) [back to overview]Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study
NCT00171210 (12) [back to overview]Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy
NCT00171210 (12) [back to overview]Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID
NCT00171210 (12) [back to overview]Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study
NCT00171210 (12) [back to overview]Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID
NCT00171210 (12) [back to overview]Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event
NCT00171301 (4) [back to overview]Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age
NCT00171301 (4) [back to overview]Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study
NCT00171301 (4) [back to overview]Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category
NCT00171301 (4) [back to overview]Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC)
NCT00235391 (2) [back to overview]Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events
NCT00235391 (2) [back to overview]The Change in Serum Ferritin Values From Baseline Through Completion of the Study
NCT00303329 (4) [back to overview]The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study
NCT00303329 (4) [back to overview]The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study
NCT00303329 (4) [back to overview]The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths
NCT00303329 (4) [back to overview]The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study
NCT00390858 (4) [back to overview]Relative Change in Serum Ferritin Level
NCT00390858 (4) [back to overview]Participants With Adverse Events by Primary System Organ Class (SOC)
NCT00390858 (4) [back to overview]Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)
NCT00390858 (4) [back to overview]Change in Liver Iron Concentration (LIC)
NCT00395629 (2) [back to overview]Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)
NCT00395629 (2) [back to overview]Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)
NCT00419770 (1) [back to overview]Total Adverse Events
NCT00447694 (4) [back to overview]Serum Ferritin and Changes From Baseline in Serum Ferritin During Study
NCT00447694 (4) [back to overview]Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2*
NCT00447694 (4) [back to overview]Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks
NCT00447694 (4) [back to overview]Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks
NCT00599326 (2) [back to overview]Number of Participants Showing Reduction or Elimination of Skin Blistering
NCT00599326 (2) [back to overview]Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level
NCT00600938 (21) [back to overview]Extension Study: Change in Serum Ferritin From Baseline by Month
NCT00600938 (21) [back to overview]Extension Study: The Cardiac Iron Concentration From T2* Values
NCT00600938 (21) [back to overview]Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Cmax)
NCT00600938 (21) [back to overview]Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
NCT00600938 (21) [back to overview]Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Tmax)
NCT00600938 (21) [back to overview]Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Mass Indices (LVMI)
NCT00600938 (21) [back to overview]Core Study: Cardiac Function After 6 and 12 Months Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Systolic Volume Indices (LVESVI)
NCT00600938 (21) [back to overview]Core Study: Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)
NCT00600938 (21) [back to overview]Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Time Points of Concentration Data
NCT00600938 (21) [back to overview]Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Ejection Fraction (LVEF)
NCT00600938 (21) [back to overview]Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)
NCT00600938 (21) [back to overview]Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Systolic Volume Indices (LVESVI)
NCT00600938 (21) [back to overview]Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Mass Indices (LVMI)
NCT00600938 (21) [back to overview]Extension Study: Change From Baseline in Myocardial T2* After 24 Months Treatment
NCT00600938 (21) [back to overview]Core Study: Safety and Tolerability of Deferasirox vs Deferoxamine Over the 12 Months Treatment Period.
NCT00600938 (21) [back to overview]Core Study: Cardiac Function After 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)
NCT00600938 (21) [back to overview]Core Study: Cardiac Function After 6 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)
NCT00600938 (21) [back to overview]Core Study: Cardiac Function and the Proportion of Patients Dropping Out Due to Cardiac Dysfunction After Treatment With Deferasirox vs. Deferoxamine
NCT00600938 (21) [back to overview]Core Study: Change From Baseline in Myocardial T2* (Magnetic Resonance T2-star (T2*) Technique for the Measurement of Tissue Iron) After 12 Months Treatment
NCT00600938 (21) [back to overview]Core Study: Change From Baseline in Myocardial T2* After 6 Months Treatment
NCT00600938 (21) [back to overview]Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Area Under the Plasma Concentration-time Curve for a Dosing Interval (AUCtau)
NCT00602446 (2) [back to overview]Number of Patients Not Completing Treatment
NCT00602446 (2) [back to overview]Reduction in Liver Iron Concentration After Study Drug
NCT00631163 (9) [back to overview]Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1
NCT00631163 (9) [back to overview]Correlation of LIC and Serum Ferritin at Core and Extension Study
NCT00631163 (9) [back to overview]Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup
NCT00631163 (9) [back to overview]Absolute Serum Ferritin Levels Over 2 Years
NCT00631163 (9) [back to overview]Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption
NCT00631163 (9) [back to overview]Absolute Change From Baseline in Serum Ferritin Levels to Year 2
NCT00631163 (9) [back to overview]Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2
NCT00631163 (9) [back to overview]Number of Participants With Clinically Significant Ophthalmological Abnormalities
NCT00631163 (9) [back to overview]Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years
NCT00749515 (6) [back to overview]Number of Participants With Polymorphisms in Genes Known to be, or Potentially Involved, in Deferasirox Disposition
NCT00749515 (6) [back to overview]Half-Life of Deferasirox
NCT00749515 (6) [back to overview]Area Under the Curve of Deferasirox After a Dose of 35 mg/kg
NCT00749515 (6) [back to overview]Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg
NCT00749515 (6) [back to overview]Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg
NCT00749515 (6) [back to overview]Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg
NCT00845871 (4) [back to overview]Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16
NCT00845871 (4) [back to overview]Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption
NCT00845871 (4) [back to overview]Change From Baseline in Serum Ferritin at Week 16
NCT00845871 (4) [back to overview]Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12
NCT00873041 (20) [back to overview]Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter
NCT00873041 (20) [back to overview]Extension Study: Change From Baseline in Hemoglobin at Month 24
NCT00873041 (20) [back to overview]Extension Study: Change From Baseline in Transferrin Saturation at Month 24
NCT00873041 (20) [back to overview]Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24
NCT00873041 (20) [back to overview]Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
NCT00873041 (20) [back to overview]Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study
NCT00873041 (20) [back to overview]Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24
NCT00873041 (20) [back to overview]Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)
NCT00873041 (20) [back to overview]Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe
NCT00873041 (20) [back to overview]Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results
NCT00873041 (20) [back to overview]Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure
NCT00873041 (20) [back to overview]Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate
NCT00873041 (20) [back to overview]Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure
NCT00873041 (20) [back to overview]Core Study: Change From Baseline in Hemoglobin at Month 12
NCT00873041 (20) [back to overview]Core Study: Change From Baseline in Transferrin Saturation at Month 12
NCT00873041 (20) [back to overview]Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24
NCT00873041 (20) [back to overview]Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52
NCT00873041 (20) [back to overview]Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52
NCT00873041 (20) [back to overview]Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter
NCT00873041 (20) [back to overview]Core Study: Change in Serum Ferritin Between Baseline and Second Quarter
NCT00901199 (2) [back to overview]Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months
NCT00901199 (2) [back to overview]Change in Serum Creatinine During 12 Months Combined Chelation Therapy
NCT00940602 (16) [back to overview]Percentage of Participants With Significant Renal Dysfunction
NCT00940602 (16) [back to overview]Time to at Least a 10% Increase From Baseline in Left Ventricular End-diastolic Internal (LVIDD) at Two Consecutive Assessments at Least Two Weeks Apart
NCT00940602 (16) [back to overview]Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia
NCT00940602 (16) [back to overview]Percentage of Participants With Newly Occurring Severe Thrombocytopenia
NCT00940602 (16) [back to overview]Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline
NCT00940602 (16) [back to overview]All Collected Deaths
NCT00940602 (16) [back to overview]Total Number of Infections Requiring Intravenous Antimicrobials
NCT00940602 (16) [back to overview]Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality
NCT00940602 (16) [back to overview]Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart)
NCT00940602 (16) [back to overview]Time to Disease Progression
NCT00940602 (16) [back to overview]Time to at Least a 10% Increase From Baseline in Left Ventricular Internal Systolic Diameter (LVISD) at Two Consecutive Assessments at Least Two Weeks Apart
NCT00940602 (16) [back to overview]Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline
NCT00940602 (16) [back to overview]Percentage of Participants With Major Gastrointestinal Bleeding
NCT00940602 (16) [back to overview]Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response
NCT00940602 (16) [back to overview]Event-free Survival
NCT00940602 (16) [back to overview]Overall Survival
NCT01033747 (2) [back to overview]Relative Change in Serum Ferritin From Baseline to 3.5 Years
NCT01033747 (2) [back to overview]The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox
NCT01090323 (2) [back to overview]Number of Participants With Adverse Events After Start of ICL670
NCT01090323 (2) [back to overview]Change in Serum Ferritin From Start of ICL670 to End of Study
NCT01159067 (2) [back to overview]Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)
NCT01159067 (2) [back to overview]Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment
NCT01254227 (7) [back to overview]Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24
NCT01254227 (7) [back to overview]Time to Achieve From Baseline (FAS) of at Least 10% at Month 24
NCT01254227 (7) [back to overview]Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24
NCT01254227 (7) [back to overview]Change in Cardiac Iron Content From Baseline to Month 6,18 and 24
NCT01254227 (7) [back to overview]Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24
NCT01254227 (7) [back to overview]Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24
NCT01254227 (7) [back to overview]Change in Cardiac Iron Content From Baseline to Month 12
NCT01273766 (3) [back to overview]Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only)
NCT01273766 (3) [back to overview]Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics
NCT01273766 (3) [back to overview]Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections
NCT01459718 (8) [back to overview]Left Ventricular Ejection Fraction (LVEF)
NCT01459718 (8) [back to overview]Time to Response
NCT01459718 (8) [back to overview]Number of Patients Achieving a Complete Response (CR)
NCT01459718 (8) [back to overview]Number of Patients Achieving a Partial Response (PR)
NCT01459718 (8) [back to overview]Number of Patients With Stable Disease (SD)
NCT01459718 (8) [back to overview]Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy
NCT01459718 (8) [back to overview]Change From Baseline in Liver Iron Concentration (LIC)
NCT01459718 (8) [back to overview]Correlation Between Change From Baseline in Serum Ferritin and LIC Levels
NCT01610297 (5) [back to overview]The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L
NCT01610297 (5) [back to overview]Change in Serum Ferritin Level.
NCT01610297 (5) [back to overview]Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
NCT01610297 (5) [back to overview]Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)
NCT01610297 (5) [back to overview]Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability
NCT01709032 (2) [back to overview]Number of Participants With Improvement in Cardiac T2* MRI
NCT01709032 (2) [back to overview]Number of Participants With Improvement in Liver Iron Concentration
NCT01709838 (15) [back to overview]Absolute Change in Health-related Outcomes Using Medical Outcomes Study Form 36 (SF-36v2)
NCT01709838 (15) [back to overview]Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline
NCT01709838 (15) [back to overview]Absolute Change in Serum Ferritin From Baseline After 52 Weeks
NCT01709838 (15) [back to overview]Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw
NCT01709838 (15) [back to overview]PK Parameters: AUCtau
NCT01709838 (15) [back to overview]PK Parameters: Cmax
NCT01709838 (15) [back to overview]PK Parameters: Tmax
NCT01709838 (15) [back to overview]Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period
NCT01709838 (15) [back to overview]Time to Achieving LIC <5 mg Fe/g dw
NCT01709838 (15) [back to overview]Absolute Change in Health-related Outcomes Using the Pediatric Quality of Life Questionnaire (PedsQL™)
NCT01709838 (15) [back to overview]Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome
NCT01709838 (15) [back to overview]Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)
NCT01709838 (15) [back to overview]Absolute Change in LIC From Baseline Over Time
NCT01709838 (15) [back to overview]Plasma Pharmacokinetics (PK) Deferasirox Concentrations
NCT01709838 (15) [back to overview]Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)
NCT01818726 (3) [back to overview]Change in Serum Ferritin Values
NCT01818726 (3) [back to overview]Change in Serum Total Iron-binding Capacity (TIBC)
NCT01818726 (3) [back to overview]Change in Transferrin Saturation With Iron (TSI) Values
NCT01825512 (4) [back to overview]Liver MRI
NCT01825512 (4) [back to overview]Cardiac MRI T2*
NCT01825512 (4) [back to overview]Ferritin Level
NCT01825512 (4) [back to overview]Percentage of Successfully Chelated Patients
NCT01868477 (15) [back to overview]Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Hemoglobin Values up to 24 Weeks
NCT01868477 (15) [back to overview]Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)
NCT01868477 (15) [back to overview]Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
NCT01868477 (15) [back to overview]Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)
NCT01868477 (15) [back to overview]Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks
NCT01868477 (15) [back to overview]Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)
NCT02125877 (12) [back to overview]Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
NCT02125877 (12) [back to overview]Weekly Dose Violation Rate
NCT02125877 (12) [back to overview]Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
NCT02125877 (12) [back to overview]Palatability Questionnaire Score
NCT02125877 (12) [back to overview]Overall Safety as Measured by Frequency of Adverse Events
NCT02125877 (12) [back to overview]Overall Safety as Measured by Changes in Laboratory Values From Baseline
NCT02125877 (12) [back to overview]Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
NCT02125877 (12) [back to overview]Number of Participants With Weekly Average Compliance of Medication Consumption
NCT02125877 (12) [back to overview]Frequency of Selected Gastro-intestinal (GI) Adverse Events
NCT02125877 (12) [back to overview]Dererasirox Plasma Concentration
NCT02125877 (12) [back to overview]Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
NCT02125877 (12) [back to overview]Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
NCT02720536 (10) [back to overview]Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12
NCT02720536 (10) [back to overview]Overview of Number of Participants With Adverse Events
NCT02720536 (10) [back to overview]Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12
NCT02720536 (10) [back to overview]Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12
NCT02720536 (10) [back to overview]Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12
NCT02943668 (4) [back to overview]Proportion of Patients That Achieve Erythroid Hematologic Improvement.
NCT02943668 (4) [back to overview]Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement
NCT02943668 (4) [back to overview]Change in Red Blood Cell (RBC) Transfusion Requirements
NCT02943668 (4) [back to overview]Change in Serum Ferritin Levels
NCT02993224 (10) [back to overview]Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire
NCT02993224 (10) [back to overview]Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
NCT02993224 (10) [back to overview]Change From Baseline in Serum Ferritin Levels
NCT02993224 (10) [back to overview]Change Over Time in Palatability Score of Palatability Questionnaire
NCT02993224 (10) [back to overview]Change Over Time in Aftertaste Score of Palatability Questionnaire
NCT02993224 (10) [back to overview]Number of Participants Preferring Deferasirox DT or Previous Iron Chelation Therapy at Week 4 and Week 24 Based on Preference Questionnaire (Item 2)
NCT02993224 (10) [back to overview]Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
NCT02993224 (10) [back to overview]Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire
NCT02993224 (10) [back to overview]Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods
NCT02993224 (10) [back to overview]Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48
NCT03372083 (29) [back to overview]Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters
NCT03372083 (29) [back to overview]Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox
NCT03372083 (29) [back to overview]Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants
NCT03372083 (29) [back to overview]Absolute Change From Baseline in Body Temperature (°C)
NCT03372083 (29) [back to overview]Number of Participants With Notable Changes in ECG Values From Baseline
NCT03372083 (29) [back to overview]Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline
NCT03372083 (29) [back to overview]Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence
NCT03372083 (29) [back to overview]GI Symptom Score in Participants Pre-treated With Deferasirox
NCT03372083 (29) [back to overview]Palatability Score in Participants Pre-treated With Deferasirox
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring
NCT03372083 (29) [back to overview]GI Symptom Score in Chelation Naive Participants
NCT03372083 (29) [back to overview]Adverse Events Profile
NCT03372083 (29) [back to overview]Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg)
NCT03372083 (29) [back to overview]Absolute Change From Baseline in Serum Ferritin (SF)
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
NCT03372083 (29) [back to overview]Absolute Change From Baseline in Body Weight (kg)
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns
NCT03372083 (29) [back to overview]Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence
NCT03372083 (29) [back to overview]Number of Chelation Naive Participants With Palatability After Taste Item Scoring
NCT03372083 (29) [back to overview]Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks
NCT03372083 (29) [back to overview]Palatability Score in Chelation Naive Participants
NCT03372083 (29) [back to overview]Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring
NCT03372083 (29) [back to overview]Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring
NCT03372083 (29) [back to overview]Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring
NCT03372083 (29) [back to overview]Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring
NCT03372083 (29) [back to overview]Absolute Change From Baseline in Pulse Rate (Bpm)

Treatment Compliance to Deferasirox

Treatment compliance was assessed using records of study medication used, dosages administered, and intervals between visits during the study. Drug accountability was noted by the field monitor during site visits and at the completion of the trial. Participants were asked to return all unused medication at monthly visits. (NCT00110266)
Timeframe: up to 1 year

Interventionparticipants (Number)
20 to < 40 %40 - < 60 %60 - < 80 %80 - < 100 %100 - < 120 %≥ 120 %
Deferasirox214896512

[back to top]

Trough Plasma Deferasirox Concentration

The Pharmacokinetic (PK) parameters were assessed at Week 13, 25, 37 and 49 using Liquid Chromatography with tandem mass spectrometry (LC-MS/MS) method. (NCT00110266)
Timeframe: At Week 13, 25, 37 and 49

Interventionμmol/L (Median)
Week 13Week 25Week 37Week 49
Deferasirox21.20026.70025.20030.700

[back to top]

Number of Participants Reporting Adverse Events

Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the study, whether or not considered related to the participant's participation in the study. Serious Adverse Events include adverse events that result in either of death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal functions, or a congenital anomaly/birth defect. (NCT00110266)
Timeframe: up to 53 Weeks

InterventionParticipants (Count of Participants)
Deferasirox171

[back to top]

Change in Labile Plasma Iron (LPI)

LPI represents the component of non-transferrin bound iron and is an indicator of iron overload. The blood sample for LPI determinations was collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49. LPI was calculated as 1 LPI unit = the quantity of reactive oxygen species produced by approximately 1.5 μM Fe (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37 and 49

InterventionLPI Unit (Median)
BaselineWeek 13Week 25Week 37Week 49
Deferasirox0.30-0.10-0.20-0.30-0.45

[back to top]

Total Iron Levels

Levels of non-transferrin bound iron (NTBI) and serum iron from baseline to 3,6,9 and 12 months (Weeks 13, 25, 37 and 49) were assessed. (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37, 49 and 53

Interventionμg/dL (Median)
BaselineWeek 13Week 25Week 37Week 49Week 53
Deferasirox194.0230.50222.0212.0218.0221.00

[back to top]

Change in Serum Ferritin From Baseline to Weeks 13, 25, 37 and 53

Change in levels of serum ferritin from baseline to 3, 6, 9 and 12 months (Weeks 13, 25, 37 and 53). (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37 and 53

Interventionμg/L (Median)
BaselineWeek 13Week 25Week 37Week 53
Deferasirox2771.5-146.5-167.5-505.0-592.0

[back to top]

Directly Chelatable Iron (DCI)

The blood sample for DCI determinations were collected at Week 1 (prior to first dose) and at Weeks 13, 25, 37 and 49. (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37 and 49

InterventionµM (Median)
BaselineWeek 13Week 25Week 37Week 49
Deferasirox0.000.000.000.000.00

[back to top]

Frequency of Hematologic Improvement During the Study

"Hematologic responses defined by International Working Group response criteria in myelodysplasia. Hematologic Improvement (HI) responses, at least 9 weeks defined as:~Erythroid response (pretreatment, <11 g/dL): Hgb increase by 1.5 g/dL; Relevant reduction units of Red blood cell (RBC) transfusions by absolute number at least 4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 weeks; Only RBC transfusions for Hgb of 9.0 g/dL pretreatment count in RBC transfusion response evaluation; Platelet response (pretreatment,<100x10^9/L): If starting with >20x10^9/L platelets: absolute increase 30x10^9/L, Increase from baseline <20 x10^9/L to >20x10^9/L and by =/> 100%; Neutrophil response (pretreatment, <1.0x10^9/L): =/> 100% increase & absolute increase >0.5x10^9/L; Progression or relapse after HI: At least 1 of the following: =/>50% decrement from max response levels in granulocytes or platelets; Reduction in Hgb by 1.5 g/dL; or Transfusion dependence." (NCT00110266)
Timeframe: up to 1 year

InterventionParticipants (Count of Participants)
Response (Yes)Response (No)Missing
Deferasirox77410

[back to top]

Serum Transferrin Levels

Serum transferrin from baseline to 3, 6, 9 and 12 months of treatment (Visit Weeks 13, 25, 37 and 49) were assessed. (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37, 49 and 53

Interventionmg/dL (Median)
BaselineWeek 13Week 25Week 37Week 49Week 53
Deferasirox156.0161.0160.0158.0160.0161.00

[back to top]

Transfusion Requirements

Number of participants receiving transfusions, the summarized during the study. (NCT00110266)
Timeframe: up to 1 year

InterventionParticipants (Count of Participants)
ScreeningWeek 1 through 13Week 14 through 26Week 27 through 39Week 40 through 52
Deferasirox1631361199872

[back to top]

Transferrin Saturation

Levels of transferrin saturation from baseline to 3, 6, 9 and 12 months. (NCT00110266)
Timeframe: From Baseline to Weeks 13, 25, 37, 49 and 53

Interventionpercentage of saturation (Median)
BaselineWeek 13Week 25Week 37Week 49Week 53
Deferasirox91.00-11.00-11.00-12.50-12.00-18.00

[back to top]

The Prevalence of Hereditary Hemochromatosis Gene (HFE) Gene Mutations

HFE (hemochromatosis) gene mutations in the Myelodysplastic Syndrome (MDS) population, the trial included testing for the C282Y, H63D and S65C HFE gene mutations. One blood sample was collected at Baseline (Week 1), or, if that visit was missing, the sample was taken at Week 13 (Month 3). Only one blood sample was to be taken from each participant. (NCT00110266)
Timeframe: up to Week 13 (Month 3)

InterventionParticipants (Count of Participants)
C282Y72456542H63D72456542S65C72456542
NegativePositive Heterozygous
Deferasirox85
Deferasirox9
Deferasirox70
Deferasirox24
Deferasirox92
Deferasirox2

[back to top]

Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 104

Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 104 for the Deferasirox treatment group. Means were adjusted for the amount of transfused blood. (NCT00110617)
Timeframe: Start of Deferasirox (ICL670) treatment, 104 Weeks

Interventionmg/mL (Least Squares Mean)
Deferasirox (ICL670)-682.6

[back to top]

The Number of Participants With Adverse Events (AEs) in the First 24 Weeks of Treatment

The number of participants with Adverse Events (AEs) overall and according to Medical Dictionary for Regulatory Activities (MedDRA) preferred term greater than or equal to 5% participants in any group by treatment in the first 24 weeks. (NCT00110617)
Timeframe: 24 Weeks

,
Interventionparticipants (Number)
HeadacheSickle cell anaemia with crisisDiarrhoeaVomitingPyrexiaNauseaAbdominal painUpper respiratory tract infectionRashCoughConstipationPain in ExtremityBack PainChest PainOropharyngeal painPruritusAbdominal pain upperNasal congestionUrinary tract infectionArthralgiaNasopharyngitisInsomniaDizzinessInjection site pain
Deferasirox (ICL670)303030211920161014101110877786974320
Deferoxamine (DFO) Then ICL6701785994693723455534013333

[back to top]

Absolute Change in Serum Ferritin After Start of Treatment With Deferasirox (ICL670) to Week 24 and to Week 52

Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 24 and the absolute change in serum ferritin after start of treatment with Deferasirox (ICL670) to week 52 for the Deferasirox treatment group and the Deferoxamine then Deferasirox treatment group. Means were adjusted for the amount of transfused blood. (NCT00110617)
Timeframe: Start of Deferasirox (ICL670) treatment, 24 Weeks, 52 Weeks

,
Interventionmg/mL (Least Squares Mean)
24 weeks from ICL670 treatment start (n=111,47)52 weeks from ICL670 treatment start (n=113,40)
Deferasirox (ICL670)-146.7-487.3
Deferoxamine (DFO) Then ICL670-204.7-545.7

[back to top]

Absolute Change in Serum Ferritin From Baseline to Week 24

Absolute change from baseline serum ferritin after 24 weeks of treatment with Deferasirox (ICL670) and absolute change from baseline serum ferritin after 24 weeks of treatment with Deferoxamine. Means were adjusted for the amount of transfused blood. (NCT00110617)
Timeframe: Baseline, 24 Weeks

Interventionmg/mL (Least Squares Mean)
Deferasirox (ICL670)-173.2
Deferoxamine (DFO) Then ICL670-868.7

[back to top]

Number of Participants With Adverse Events and Serious Adverse Events

An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign [including abnormal laboratory findings], symptom or disease) in a subject or clinical investigation subject after providing written informed consent for participation in the study. Any sign or symptom that occured from first dose of study treatment until end of study treatment. (NCT00117507)
Timeframe: Up To Week 52

InterventionParticipants (Count of Participants)
Adverse eventsSerious adverse events
Deferasirox2411

[back to top]

Labile Plasma Iron (LPI)

LPI represents the component of non-transferrin bound iron and is an indicator of iron overload. The outcome was reported as LPI Unit, where, 1 LPI unit = the quantity of reactive oxygen species produced by approximately 1.5 μM Fe. (NCT00117507)
Timeframe: Baseline to Week 52

InterventionLPI Unit (Mean)
BaselineWeek 52/EOS
Deferasirox0.700.22

[back to top]

Absolute Change in Urinary Hepcidin

Absolute Change in Urinary Hepcidin from baseline (NCT00117507)
Timeframe: Baseline to Week 52

Interventionng (nanogram)/mg (milligram) creatinine (Median)
BaselineWeek 52/EOS
Deferasirox327.6367.32

[back to top]

Absolute Change in Transferrin Saturation

Transferrin Saturation was assessed using magnetic resonance imaging (MRI) mean liver proton transverse relaxation rates (R2) (NCT00117507)
Timeframe: Baseline to Week 52

Interventionpercentage of transferrin saturation (Mean)
BaselineWeek 52/EOS (Change from baseline)
Deferasirox59.38.6

[back to top]

Absolute Change in Serum Ferritin From Baseline to Week 52

Absolute change in serum ferritin after start of treatment with Deferasirox (ICL670). (NCT00117507)
Timeframe: Baseline to Week 52

Interventionμg/L (microgram/litre) (Mean)
BaselineWeek 52/EOS (end of study)
Deferasirox3847.6-729.8

[back to top]

Absolute Change in Liver Iron Concentration (LIC) From Baseline to End of Study

LIC was assessed using magnetic resonance imaging (MRI) mean liver proton transverse relaxation rates (R2). (NCT00117507)
Timeframe: Baseline to Week 52

Interventionmg iron (Fe) per gram of dry weight (dw) (Mean)
BaselineWeek 52/EOS
Deferasirox20.64-4.50

[back to top]

To Evaluate Change in Transfusion Requirements

Change in transfusion requirements from baseline. (NCT00117507)
Timeframe: Baseline to Week 52

Interventionblood tranfusions (Mean)
Number of Transfusions per PatientNumber of Units of Blood Transfused per Patient
Deferasirox15.534.0

[back to top]

Absolute Change in Serum Erythropoietin

Absolute Change in Serum Erythropoietin from baseline. (NCT00117507)
Timeframe: Baseline to Week 52

InterventionIU (international unit)/L (litre) (Median)
BaselineWeek 52/EOS
Deferasirox646.50-79.00

[back to top]

Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy

Relative change in liver iron content (LIC) as measured by biopsy and calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value. (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionpercent of start value (Median)
Crossover-25.4
ICL670-26.0

[back to top]

Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by Biopsy

Measurement of median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through biopsy. Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw). (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionmg Fe/g dw (Median)
Crossover-2.8
ICL670-3.2

[back to top]

Absolute Change in Liver Iron Content From Start of ICL670 Treatment to End of Study Measured by SQUID

Measurement of the median absolute change in liver iron content (LIC) from start of treatment with Deferasirox (ICL670) to end of study obtained through Superconducting Quantum Interfering Device (SQUID). Absolute change = End of study value - start of treatment value. LIC is expressed in mg of iron per gram of liver dry weight (mg Fe/g dw). (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionmg Fe/g dw (Median)
Crossover-1.1
ICL670-1.6

[back to top]

Change in Surrogate Marker: Serum Iron From Start of Treatment With ICL670 to End of Study

"Measurement of the relative change of potential surrogate markers: Serum Iron (µmol/L) from start of treatment with Deferasirox (ICL670) to end of study.~(Serum Iron at the End of Study-Serum Iron at Start of ICL670)/Serum Iron at Start of ICL670*100." (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

InterventionPercent change (Mean)
Crossover1.5
ICL67010.3

[back to top]

Change in Surrogate Marker: Serum Transferrin From Start of Treatment With ICL670 to End of Study

"Measurement of the relative change in percent of potential surrogate marker: Serum Transferrin (g/L) from start of treatment with Deferasirox (ICL670) to end of study.~(Serum Transferrin at the End of Study-Serum Transferrin at Start of ICL670)/Serum Transferrin at Start of ICL670*100." (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

InterventionPercent change (Mean)
Crossover7.7
ICL6707.0

[back to top]

Change in Surrogate Marker: Transferrin Saturation From Start of Treatment With ICL670 to End of Study

"Measurement of the relative change of potential surrogate marker: Transferrin Saturation (Percent) from start of treatment with Deferasirox (ICL670) to end of study.~(Transferrin Saturation at the End of Study-Tranferrin Saturation at Start of ICL670)/Transferrin Saturation at Start of ICL670*100." (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

InterventionPercent change (Mean)
Crossover-5.4
ICL6703.4

[back to top]

Change of Total Body Iron Excretion Rate (TBIE) From Start of ICL670 Treatment to the End of Study

Median change in TBIE (mg/kg/day) from start of treatment with Deferasirox (ICL670) to end of study. (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionmg/kg/day (Median)
Crossover0.37
ICL6700.38

[back to top]

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by Liver Biopsy

Mean absolute change of LIC from start of Deferasirox (ICL670) treatment to the end of study assessed by liver biopsy. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw). (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionmg Fe/g dw (Mean)
Crossover-2.4
ICL670-4.1

[back to top]

Long-term Effect of ICL670 on Hepatic Iron Stores Measured by Means of Liver Iron Content (LIC) as Assessed by SQUID

Mean absolute change in LIC from start of Deferasirox (ICL670) treatment to the end of the study assessed by Superconducting Quantum Interfering Device (SQUID) measurement used as a non-invasive alternative to Biopsy for pediatric participants. Reported in milligrams of Iron per gram dry weight (mg Fe/g dw). (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionmg Fe/g dw (Mean)
Crossover-0.5
ICL670-0.7

[back to top]

Long-term Effect of Treatment With ICL670 on the Changes in Serum Ferritin Levels From Start of ICL670 Treatment to End of Study

Mean Absolute Change in serum ferritin (ug/L) from start of treatment with Deferasirox (ICL670) to end of study taking into account the therapeutic goal which will either be to maintain iron balance or to induce negative iron balance. End of study taken as the mean of, at most, the last three available results after start of treatment with ICL670. (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionμg/L (Mean)
Crossover-122.1
ICL670-527.8

[back to top]

Relative Change in Liver Iron Content From Start of ICL670 Treatment to End of Study as Measured by SQUID

Relative change in liver iron content (LIC) measured by Superconducting Quantum Interfering Device (SQUID), calculated by: End of study value - Start of ICL670 treatment value (absolute change) / Start of ICL670 treatment value. (NCT00171210)
Timeframe: Start of ICL670 treatment, End of Study or study discontinuation (up to 5 years)

Interventionpercent of start value (Median)
Crossover-20.8
ICL670-33.0

[back to top]

Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event

Adverse events results are based on preferred terms with at least 7% of participants in any group. (NCT00171210)
Timeframe: up to 5 years

,
InterventionParticipants (Number)
PyrexiaCoughHeadacheDiarrhoeaVomitingInfluenzaAbdominal painNasopharyngitisNauseaOropharyngeal painPharyngitisBack PainAbdominal pain upperRhinitisBronchitisArthralgiaUpper respiratory tract infectionTransfusion reactionBlood creatinine increasedGastroenteritisAstheniaRashTonsillitisFatiguePain in extremityAcute tonsillitisDyspepsiaUrinary tract infectionUrticariaToothacheSinusitisCholelithiasis
Crossover10985817361655956525057495245413932362231312422222121171414141311
ICL670113110846975707573636255595252454552344737313430293029212323222223

[back to top]

Percentage of Participants With Treatment Success From Core Baseline (BL) to Extension End of Study, by Baseline LIC Level and Age

Success was defined as the percentage of participants with decreased liver iron content (LIC) at the end of extension study compared to core baseline (BL) LIC. Success Criteria: For participants with Baseline LIC from 1 - <7 mg Fe/g dw, success was achieved if LIC level maintained at 1 - <7 mg Fe/g dw. For participants with Baseline LIC ≥7 - <10 mg Fe/g dw, success was achieved if LIC dropped to between 1 and < 7 mg Fe/g dw. For participants with Baseline LIC ≥10 mg Fe/g dw, success was achieved if LIC dropped by at least 3 mg Fe/g dw. LIC was measured by biopsy or magnetic resonance imaging. (NCT00171301)
Timeframe: From Core Study Baseline, to Extension End of Study, Up to 3 Years

,
Interventionpercentage of participants (Mean)
Core Baseline LIC 1-<7 mg Fe/g dw (n=20, 2)Core Baseline LIC 7-<10 mg Fe/g dw (n=18, 8)Core Baseline LIC ≥10 mg Fe/g dw (n=124, 61)
Deferasirox (16 Years or Older)10050.073.8
Deferasirox (Between 2 <16 Years)75.072.276.6

[back to top]

Absolute Change in Serum Ferritin Level Measured From Core Study Baseline (BL) to End of Extension Study

Serum Levels were assessed at core study baseline (BL), 1 year, 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study) in monthly intervals. Serum Ferritin is reported in micrograms per Liter (µg/L). (NCT00171301)
Timeframe: From Baseline of Core Study to End of Extension Study, up to 3 years

Interventionµg/L (Mean)
Deferasirox (Between 2 <16 Years)-1432.51
Deferasirox (16 Years or Older)-1791.91

[back to top]

Absolute Change in Liver Iron Concentration (LIC)Measured by Liver MRI or Liver Biopsy From Core Study Baseline (BL) to End of Extension Study, by LIC Category

"Liver MRI or Liver Biopsy was performed at the core study baseline (BL) and then 1 year and 2 years in the core study, baseline of the extension study and time of discontinuation from the extension visit (end of study). Liver iron content (LIC) is reported in milligram Iron per gram dry weight (mg Fe/g dw).~Absolute change in LIC from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-<7 mg Fe/g dw and ≥7 mg Fe/g dw." (NCT00171301)
Timeframe: From Baseline of Core Study to End of Extension Study, up to 3 years

,
Interventionmg Fe/g dw (Mean)
Core Baseline LIC 1-<7 mg Fe/g dw (n=12, 0)Core Baseline LIC ≥7 mg Fe/g dw (n=107, 41)
Deferasirox (16 Years or Older)NA-8.39
Deferasirox (Between 2 <16 Years)-1.32-9.03

[back to top]

Absolute Change in Serum Ferritin Level for All Participants Measured From Core Study Baseline (BL) to End of Extension Study, by Baseline Liver Iron Content (LIC)

Serum Levels were assessed at core study baseline (BL) and then 1 year and 2 years in core study, baseline of extension study and time of discontinuation from the extension visit (end of study). Serum Ferritin is reported in micrograms per Liter. Absolute change in Serum Ferritin from core study baseline to the end of the extension study is presented for participants with the following two core study baseline LIC levels: 1-<7 mg Fe/g dw and ≥7 mg Fe/g dw. (NCT00171301)
Timeframe: From Baseline of Core Study to End of Extension Study, up to 3 years

Interventionµg/L (Mean)
Core Baseline LIC 1-<7 mg Fe/g dw (n=12)Core Baseline LIC ≥ 7 mg Fe/g dw (n=151)
Deferasirox (All Participants)-369.83-1619.31

[back to top]

Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events

Safety as assessed by the number of participants with death, serious adverse events (SAE), and/or Adverse Events (AEs) leading to study drug interruption or discontinuation. Note: only treatment emergent AEs are summarized. (NCT00235391)
Timeframe: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)

,,,,,
InterventionParticipants (Number)
Number of deathsNon-fatal SAEsAEs leading to discontinuationAEs leading dose adjustment/temporary interruption
≥ 65 Years0222
12 to < 16 Years1271028
16 to < 50 Years412975209
2 to < 6 Years010415
50 to < 65 Years04511
6 to < 12 Years022531

[back to top]

The Change in Serum Ferritin Values From Baseline Through Completion of the Study

The number of participants with Improvement, No Change or Worsening in Serum ferritin category levels at the end of the study compared to baseline. Serum ferritin levels in µg/L were divided into to 6 categories: (<1000), (1000-<2500), (2500-<4000), (4000-<5500), (5500-<7000) and (>=7000). Improvement was defined as a shift to a lower category at the end of study compared to the category at baseline. Worsening was defined as a shift to a higher category at the end of the study compared to the category at baseline. No change was no change in category at end of study from baseline. (NCT00235391)
Timeframe: Baseline to end of study (Median exposure time to drug was approximately 30 weeks; Maximum exposure was 104 weeks)

,,,,,
InterventionParticipants (Number)
ImprovementNo ChangeWorsening
≥ 65 Years231
12 to < 16 Years268755
16 to < 50 Years206570341
2 to < 6 Years176316
50 to < 65 Years4317
6 to < 12 Years2710663

[back to top]

The Change in Liver Iron Content (LIC) as Assessed by Liver Biopsy at Baseline to the End of the Study

Liver iron concentration was monitored at the start of the core study, the end of the core study, and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. (NCT00303329)
Timeframe: Core study Baseline to end of extension study (up to 60 months)

Interventionmg Fe/g dw (Mean)
β-thalassemia Patients-5.17
Rare Anemias Patients-5.10

[back to top]

The Absolute Change in Serum Ferritin (μg/L) Levels From Baseline to the End of the Study

Serum ferritin was monitored monthly and the dose of deferasirox was increased or decreased in steps of 5 to 10 mg/kg/day up to a maximum of 40 mg/kg/day if appropriate, every 3 months. If serum ferritin fell to 500 ng/mL or lower on two consecutive study visits, an interruption of treatment until serum ferritin was more than 500 ng/mL was considered. (NCT00303329)
Timeframe: Core study Baseline to end of extension study (up to 60 months)

,
Interventionμg/L (Mean)
BaselineEnd of StudyAbsolute Change
Rare Anemias Patients3268.82896.0-382.2
β-thalassemia Patients4320.63708.2-612.4

[back to top]

The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths

Safety was assessed using reports of adverse events of all participants in this study. Serious adverse events are those events that resulted in death, were life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. (NCT00303329)
Timeframe: Core study Baseline to the end of the study (up to 60 months)

,
InterventionParticipants (Number)
Adverse EventsSerious Adverse EventsDeaths
Rare Anemias Patients995012
β-thalassemia Patients85362

[back to top]

The Absolute Change in Liver Iron Content (LIC) as Assessed by Superconducting Quantum Interference Device (SQUID) From Baseline to End of Study

Liver iron concentration was monitored at the end of the core study and then at the end of the extension study. High-risk participants, like participants with rare anemia, were excluded from any further potential liver biopsy, except if required and justified by the Investigator for the general care of the participant. Pediatric participants or participants with a medical contraindication to liver biopsy were allowed the use of SQUID in the extension study. (NCT00303329)
Timeframe: Core study Baseline to end of extension study (up to 60 months)

Interventionmg Fe/g dw (Mean)
β-thalassemia Patients0.19
Rare Anemias Patients-2.04

[back to top]

Relative Change in Serum Ferritin Level

Serum levels were drawn at the baseline of the Core Study up to 18 months of the Extension Study. Levels were analyzed for serum ferritin measured in micrograms per Liter. Relative change (%) in serum ferritin level was assessed from Baseline to Extension 18 months. Relative Change = 1 - (Change in ferritin level from Baseline/Baseline level) x 100. (NCT00390858)
Timeframe: Baseline of Core Study to Extension 18 months, up to 2.5 years.

Interventionpercent change (Mean)
Children (<12 Years)62.4
Adolescents ( ≧12 Years)54.9

[back to top]

Participants With Adverse Events by Primary System Organ Class (SOC)

Safety parameters were measured by the number and type of adverse events (AEs). An adverse event is any untoward medical occurence in a patient administered a medicinal product that does not necessarily have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign ( for example, an abnormal laboratory finding), symptom or disease temporally associated with the use of the medicinal product, whether or not this is associated with the use of this medicinal product. (NCT00390858)
Timeframe: 4 year extension + core 1 year

,
Interventionparticipants (Number)
Patients with at least one Adverse Event (AE)General disorders & administration site conditionsRespiratory, thoracic & mediastinal disordersGastrointestinal disordersInfections & infestationsMusculoskeletal & connective tissue disordersInvestigationsNervous system disordersSkin & subcutaneous tissue disordersInjury, poisoning & procedural complicationsEar & labyrinth disordersMetabolism & nutrition disordersEye disordersRenal & urinary disordersHepatobiliary disordersReproductive system & breast disordersCardiac disordersBlood & lymphatic system disordersPsychiatric disordersImmune system disordersSurgical & medical proceduresEndocrine disordersVascular disordersCongenital, familial & genetic disorders
Adolescents ( ≧12 Years)202017171715101388810967855423120
Children (<12 Years)201919181812139121196631011200101

[back to top]

Total Body Iron Elimination (TBIE) Rate (mg/kg/Day)

Total Iron Body Elimination (TBIE) Rate [mg/kg/Day] was calculated for each patient based on SQUID ( Superconducting Quantum Interference Device) results. (NCT00390858)
Timeframe: Baseline of Core Study to End of Extension Study, up to 5 years

,
Interventionmg/kg/Day (Mean)
Core Baseline TBIE (n=19, 20)End of Extension TBIE (n=11,14)
Adolescents ( ≧12 Years)0.40830.4286
Children (<12 Years)0.42920.4939

[back to top]

Change in Liver Iron Concentration (LIC)

Change in Liver Iron Concentration [LIC] measured by means of SQUID (Superconducting Quantum Interference Device). LIC is expressed in milligrams of iron per gram of liver dry weight (mg Fe/g dw) (NCT00390858)
Timeframe: Baseline of Core Study to End of Extension Study, up to 5 years.

,
Interventionmg Fe/g dw (Mean)
Core Baseline LIC (n = 20, 20)End of Extension LIC (n=19, 20)Change from Baseline LIC (n=19, 20)
Adolescents ( ≧12 Years)5.734.66-1.10
Children (<12 Years)6.255.46-0.9

[back to top]

Absolute Change of Serum Ferritin From Baseline to the End of Extension, by Dose Cohort (Extension Per-protocol Population)

Mean absolute change in serum ferritin from baseline to the end of the extension study. (NCT00395629)
Timeframe: 0 to 48 weeks

Interventionµg/L (Mean)
Deferasirox (ICL670) 5 mg/kg/Day-389.1
Deferasirox (ICL670) 10 mg/kg/Day-610.5
Deferasirox (ICL670) 15 mg/kg/Day-571.6

[back to top]

Trough Concentrations of Deferasirox (ICL670), by Dose Cohort (Per-protocol Population)

A blood sample was collected just prior to administration of the next dose of Deferasirox (pre-dose trough level) or approximately 24 hours after the previous dose at weeks 4, 8, 12, 16, 20 and 24. The mean trough concentration at each time point was calculated. (NCT00395629)
Timeframe: 4, 8, 12, 16, 20, and 24 weeks

,,
Interventionµmol/L (Mean)
Week 4 (n=10,14,17)Week 8 (n=10,12,17)Week 12 (n=10,12,13)Week 16 (n=10,11,11)Week 20 (n=9,10,8)Week 24 (n=10,9,6)
Deferasirox (ICL670) 10 mg/kg/Day19.6223.8828.7121.6226.4721.32
Deferasirox (ICL670) 15 mg/kg/Day30.4827.0623.3921.8027.5826.83
Deferasirox (ICL670) 5 mg/kg/Day9.638.8610.5812.3811.7214.62

[back to top]

Total Adverse Events

(NCT00419770)
Timeframe: 30 Days After End of Therapy

InterventionEvents (Number)
Deferasirox16
Placebo16

[back to top]

Serum Ferritin and Changes From Baseline in Serum Ferritin During Study

Serum ferritin will be assessed at each study visit. Analysis was performed in Completer population consists of those participants who had a Week 77 MRI. (NCT00447694)
Timeframe: From Baseline to 25, 49, 77 Week

Interventionμg/L (Mean)
BaselineWeek 25Week 25 Absolute change from baselineWeek 49Week 49 Absolute change from baselineWeek 77Week 77 Absolute change from baseline
Deferasirox4343.754280.77-62.983759.29-593.363179.81-882.74

[back to top]

Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2*

Cardiac T2* was measured in the short axis plane at the widest point of a 4-chamber localizer using custom breath-hold R2* gradient echo sequences modeled after techniques used by Anderson et al (2001) and Westwood et al (2003). (NCT00447694)
Timeframe: From Baseline to 25, 49, 77 Week

Interventionmilliseconds (msec) (Mean)
BaselineWeek 25Week 25 Absolute Change from BaselineWeek 49Week 49 Absolute Change from BaselineWeek 77Week 77 Absolute Change from Baseline
Deferasirox9.9211.731.7711.932.0112.102.18

[back to top]

Left Ventricular Ejection Fraction (LVEF) and Change in Left Ventricular Ejection Fraction From Baseline to 101 Weeks

Magnetic resonance imaging (MRI)-measured cardiac T2* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader. (NCT00447694)
Timeframe: From Baseline to 25, 49, 77 Week

Interventionpercentage of participants (Mean)
BaselineWeek 25Week 25 Absolute change from baselineWeek 49Week 49 Absolute change from baselineWeek 77Week 77 Absolute change from baseline
Deferasirox64.0162.17-1.8461.61-2.6863.840.11

[back to top]

Change From Baseline in Liver Iron Concentration (LIC) Was Measured by MRI R2 From Absolute Change From Baseline to 101 Weeks

MRI evaluation of liver iron concentration has been validated by liver biopsy (St Pierre et al 2005). Studies comparing T2* values of liver iron concentration (LIC) with LIC as assessed by biopsy have confirmed that T2* values reflect liver iron content (Wood et al 2003b). Direct tissue-validation of cardiac T2* measurements in humans has not been performed because endomyocardial biopsy is a dangerous and unreliable indicator of cardiac iron overload (Olson et al 1989, Fitchett et al 1980). However, it has been shown that cardiac T2* accurately reflects cardiac iron in a gerbil iron cardiomyopathy model (Wood et al 2004b). T2* measurements have shown excellent inter-scanner and inter-exam reproducibility, making them suitable for longitudinal monitoring (Westwood et al 2003a, Westwood et al 2003b). (NCT00447694)
Timeframe: From Baseline to 25, 49, 77 Week

Interventionmg Fe/g dw liver (Mean)
BaselineWeek 25Week 25 Absolute Change from BaselineWeek 49Week 49 Absolute Change from BaselineWeek 77Week 77 Absolute Change from Baseline
Deferasirox19.8517.23-2.8917.0-2.8516.62-3.23

[back to top]

Number of Participants Showing Reduction or Elimination of Skin Blistering

The present trial was undertaken to determine if oral deferasirox could be useful in the treatment of PCT. Monthly clinic visits with a physical examination was conducted to assess the skin for blisters. (NCT00599326)
Timeframe: Within 6 months of treatment.

Interventionparticipants (Number)
EliminationReduction
Deferasirox08

[back to top]

Number of Participants Showing Decrease in Ferritin and Urinary Porphyrin Level

Patients with PCT usually have normal or elevated serum iron and ferritin levels as well as increased iron absorption. Phlebotomy is conducted to analyzes the ferritin levels. Urine collection is performed and samples of the urine are analyzed for porphyrin levels. (NCT00599326)
Timeframe: 6 months

Interventionparticipants (Number)
Decrease in ferritin levelDecrease in urinary porphyrin level
Deferasirox86

[back to top]

Extension Study: Change in Serum Ferritin From Baseline by Month

Serum ferritin values was summarized by descriptive statistics. Absolute value and the absolute change from baseline in serum ferritin by month was provided by treatment group. (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
Interventionug/L (Mean)
Month 6 (n=72,28,38,0)Month 12 (n=70,29,40,1)Month 18 (n=66,24,39,1)Month 24 (n=59,24,30,0)
Extension: DFO to DFO-1307.14-1877.00-2426.92-2724.00
Extension: DFO to ICL-1054.87-1223.73-1494.82-1513.23
Extension: ICL to DFONA-498.00-1067.00NA
Extension: ICL to ICL-626.10-988.46-1962.14-2239.03

[back to top]

Extension Study: The Cardiac Iron Concentration From T2* Values

Cardiac iron concentration (derived from T2* values) at baseline, Months 6, 12, 18 and 24 were summarized by descriptive statistics. The absolute change from baseline at Months 6, 12, 18 and 24 were also summarized by treatment group. Lliver iron concentration is expressed in units (mg of iron / g of liver tissue dry weight (dw) (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
Interventionmg Fe/g dw (Mean)
Month 6 (n=71,26,40,1)Month 12 (n=66,29,41,1)Month 18 (n=68,26,39,1)Month 24 (n=63,25,33,1)
Extension: DFO to DFO-0.12-0.12-0.45-0.69
Extension: DFO to ICL-0.08-0.14-0.20-0.34
Extension: ICL to DFO0-0.77-0.24-0.52
Extension: ICL to ICL-0.12-0.38-0.47-0.70

[back to top]

Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Cmax)

The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, maximum plasma concentration (Cmax) (NCT00600938)
Timeframe: 12 Month

Interventionumol/L (Mean)
Deferasirox (ICL). For Extension Labeled as ICL to ICL150.09

[back to top]

Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24

Results of liver iron content (LIC) measurements by MRI was summarized by descriptive statistics. The absolute value and the absolute change from baseline in LIC at Months 6, 12, 18 and 24 were provided by treatment group. (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
Interventionmg Fe/g dw (Mean)
Month 6 (n=71,26,38,1)Month 12 (n=69,29,40,1)Month 18 (n=70,23,38,1)Month 24 (n=60,24,33,1)
Extension: DFO to DFO-12.66-19.44-26.09-26.02
Extension: DFO to ICL-6.30-7.98-10.87-10.96
Extension: ICL to DFO-3.80-3.90-2.90-3.20
Extension: ICL to ICL-4.56-10.22-12.26-15.74

[back to top]

Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Maximum Plasma Concentration (Tmax)

The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, time to reach maximum plasma concentration (Tmax) (NCT00600938)
Timeframe: 12 Month

Intervention(h) (Median)
Deferasirox (ICL). For Extension Labeled as ICL to ICL4.00

[back to top]

Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Mass Indices (LVMI)

An absolute change from baseline in LVMI after 6, and 12 months treatment with deferasirox and DFO was summarized (NCT00600938)
Timeframe: 6 Month, 12 Month

,
Interventiongram/m^2 (Mean)
Change from Baseline at 6 Month (n= 85, 73)Change from Baseline at 12 Month/EOS (n= 91, 81)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL1.014.13
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO3.325.25

[back to top]

Core Study: Cardiac Function After 6 and 12 Months Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Systolic Volume Indices (LVESVI)

An absolute change from baseline in LVESVI after 6 and 12 months treatment with deferasirox and DFO was summarized. Changes in cardiovascular magnetic resonance (CMR) measured left ventricular end systolic after 6 and 12 months treatment. Left ventricular (LV) end-systolic volume indexed to body surface area (ESVI) is a simple yet powerful echocardiographic marker of LV remodeling that can be measured easily. Left ventricular (LV) end-systolic volume (ESV) has been shown to be an important determinant of survival after myocardial infarction (MI) (NCT00600938)
Timeframe: 6 Month, 12 Month

,
InterventionMilliliter (Mean)
Change from baseline at 6 Month (n= 85, 73)Change from Baseline at 12 Month/EOS (n= 91, 81)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL1.81.57
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO0.880.10

[back to top]

Core Study: Core Study: Cardiac Function After 6 and 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)

An absolute change from baseline in LVEDVI after 6, and 12 months treatment with deferasirox and DFO was summarized (NCT00600938)
Timeframe: 6 Month, 12 Month

,
InterventionPercent (Mean)
Change from Baseline at 6 Month (n= 85, 73)Change from Baseline at 12 Month/EOS (n= 91, 81)
Core; Deferasirox (ICL). For Extension Labeled as ICL to ICL1.811.79
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO1.481.10

[back to top]

Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Time Points of Concentration Data

The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. For trough concentration assessments, a 2-mL blood sample was to be taken on arrival at the study site, i.e. prior to the patient receiving the daily deferasirox dose (pre-dose blood sample). A second 2-mL blood sample was to be taken 2 hours later (post-dose sample). At all other visits (Visits 3 - 14), a pre-dose sample was to be taken. For PK profile assessments, 3 blood samples were taken after 1, 2, and 4 hours post-dose in addition to the 2-mL pre-dose (NCT00600938)
Timeframe: Month 1 and month 2 (pre-dose, 1,2 and 4 hours post-dose)

Intervention(umol/L) (Mean)
Month 1, 0 hour (predose)Month 1, 1 hour (post dos)Month 1, 2 hour (post dose)Month 1, 4 hour (post dose)Month 2, 0 hour (predose)Month 2, 1 hour (post dose)Month 2, 2 hour (post dose)Month 2, 4 hour (post dose)
Deferasirox (ICL). For Extension Labeled as ICL to ICL32.2596.32136.47133.3338.66119.48177.19180.76

[back to top]

Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Ejection Fraction (LVEF)

Cardiac function endpoints (LVEF) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
InterventionPercent (Mean)
Month 6 (n=71,26,40,1)Month 12 (n= 66,29,42,1)Month 18 (n=68,26,39,1 )Month 24 (n= 63,25,33,1)
Extension: DFO to DFO-1.80.3-0.8-0.6
Extension: DFO to ICL0.10.0-1.30.2
Extension: ICL to DFO-1.00-10.0-18.0
Extension: ICL to ICL-1.1-0.5-0.10.6

[back to top]

Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Diastolic Volume Indices (LVEDVI)

Cardiac function endpoint (LVEDVI ) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
InterventionmL/m^2 (Mean)
Month 6 (n=69,26,40,1)Month 12 (n=64,28,40,1)Month 18 (n=67,24,35,1)Month 24 (n=60,23,33,0)
Extension: DFO to DFO3.5-0.64.29.5
Extension: DFO to ICL0.53.08.35.4
Extension: ICL to DFO1.04.036.0NA
Extension: ICL to ICL2.02.06.53.4

[back to top]

Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular End Systolic Volume Indices (LVESVI)

Cardiac function endpoints (LVESVI) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes from baseline (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
InterventionmL/m^2 (Mean)
Month 6 (n=69,26,40,1)Month 12 (n=64,28,40,1 )Month 18 (n=67,24,35,1 )Month 24 (n=60,23,33,0 )
Extension: DFO to DFO3.4-0.82.84.3
Extension: DFO to ICL00.64.11.7
Extension: ICL to DFO1228.0NA
Extension: ICL to ICL1.71.52.41.6

[back to top]

Extension Study: Cardiac Function From Baseline to Month 24 by Change in Left Ventricular Mass Indices (LVMI)

Cardiac function endpoints (LVMI) obtained by CMR at baseline, Months 6, 12, 18 and 24 were summarized by means of descriptive statistics. These analyses were conducted for the measured values as well as for the absolute changes (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
Interventiongram/m^2 (Mean)
Month 6 (n=69,26,40,1)Month 12 (n=64,28,40,1)Month 18 (n=67,24,35,1)Month 24 (n=60,23,33,0)
Extension: DFO to DFO1.89.1-0.16.7
Extension: DFO to ICL3.23.44.010.3
Extension: ICL to DFO-6.0137.0NA
Extension: ICL to ICL1.44.24.85.6

[back to top]

Extension Study: Change From Baseline in Myocardial T2* After 24 Months Treatment

The measured T2* values, the ratio (post-baseline / baseline T2*) at Month 6, 12, 18 and 24 was summarized for FAS population along with two-sided 95% CIs. The geometric means of the ratio was presented for all treatment groups (NCT00600938)
Timeframe: Months 6, 12, 18 and 24

,,,
InterventionRatio (Geometric Mean)
Month 6 (n=71,26,40,1)Month 12(n=66, 29, 41, 1)Month 18 (n=68, 26, 39, 1)Month 24 (n=63, 25, 33, 1)
Extension : ICL to ICL1.061.171.241.38
Extension; DFO to ICL1.031.071.131.21
Extension: DFO to DFO1.051.061.181.33
Extension: ICL to DFO1.001.171.051.11

[back to top]

Core Study: Safety and Tolerability of Deferasirox vs Deferoxamine Over the 12 Months Treatment Period.

Number of patients with adverse events, serious adverse events and death (NCT00600938)
Timeframe: 12 Month

,
InterventionParticipants (Number)
At least one AESerious Adverse EventsDeath. None were considered related to study drug.
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL65101
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO69101

[back to top]

Core Study: Cardiac Function After 12 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)

An absolute change from baseline in LVEF after 12 months treatment with deferasirox and compared to.DFO was tested using an analysis of covariance model including baseline left ventricular ejection fraction (LVEF) as a covariate. (NCT00600938)
Timeframe: 12 Month

InterventionPercent (Least Squares Mean)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL-0.5
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO-0.0

[back to top]

Core Study: Cardiac Function After 6 Months of Treatment With Deferasirox vs. Deferoxamine, by Change in Left Ventricular Ejection Fraction (LVEF)

An absolute change from baseline in LVEF after 6 months treatment with deferasirox and DFO was summarized (NCT00600938)
Timeframe: 6 Month

InterventionPercent (Mean)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL-0.95
Deferoxamine (DFO). For Extension Labeled as DFO to DFO-0.37

[back to top]

Core Study: Cardiac Function and the Proportion of Patients Dropping Out Due to Cardiac Dysfunction After Treatment With Deferasirox vs. Deferoxamine

The number of patients withdrawn from the study due to LVEF <50%, T2* <6 ms or significant decreases in T2* ≥ 33% from baseline was provided per treatment group. (NCT00600938)
Timeframe: 12 Month

InterventionParticipants (Number)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL3
Core; Deferoxamine (DFO). For Extension Labeled as DFO to DFO2

[back to top]

Core Study: Change From Baseline in Myocardial T2* (Magnetic Resonance T2-star (T2*) Technique for the Measurement of Tissue Iron) After 12 Months Treatment

Non- inferiority in efficacy of deferasirox compared to deferoxamine (DFO) in treating cardiac iron overload as measured by T2*. A non-inferiority margin of 0.9 (90%) was applied. Due to limitations in performing heart biopsies, T2* (T2 star), a Magnetic Resonance (MR) relaxation parameter expressed in milliseconds, as is an important tool to noninvasively quantify cardiac iron concentration. Studies have shown that myocardial T2* evaluations may predict cardiac events, e.g., impaired (<56%) left ventricular ejection fraction (LVEF) is prevalent among patients with low T2*: found in 62% of patients with T2*<8 ms; 20% with T2* of 8-12 ms; and in 5% with T2* >12 ms (Tanner 2006) (NCT00600938)
Timeframe: 12 Month

InterventionMillisecond (Geometric Mean)
Core: Deferasirox (ICL)1.12
Core: Deferoxamine (DFO)1.07

[back to top]

Core Study: Change From Baseline in Myocardial T2* After 6 Months Treatment

Summary statistics of T2* ratio Month 6/baseline (NCT00600938)
Timeframe: 6 Month

InterventionRatio (Geometric Mean)
Core: Deferasirox (ICL). For Extension Labeled as ICL to ICL1.04
Core: Deferoxamine (DFO). For Extension Labeled as DFO to DFO1.04

[back to top]

Core Study: Single and Repeated Dose Pharmacokinetics of Deferasirox, Area Under the Plasma Concentration-time Curve for a Dosing Interval (AUCtau)

The plasma level of deferasirox (ICL670) obtained in this study was summarized descriptively. Plasma concentration was plotted by patient and by visit. Descriptive statistics included the mean, median, SD, and CV, min and max. deferasirox pharmacokinetics (PK) trough levels over the 12 months of treatment and obtained PK profiles for the 40 mg/kg/day deferasirox dose, area under the plasma concentration-time curve for a dosing interval (AUCtau) (NCT00600938)
Timeframe: 12 Month

Intervention(h.ng/mL) (Mean)
Deferasirox (ICL). For Extension Labeled as ICL to ICL2129.70

[back to top]

Number of Patients Not Completing Treatment

Number of patients who discontinued deferasirox during 6 month daily treatment due to drug related toxicity (NCT00602446)
Timeframe: 6 Months

InterventionParticipants (Number)
Deferasirox Treated0

[back to top]

Reduction in Liver Iron Concentration After Study Drug

Efficacy as measured by reduction in liver iron concentration (LIC) after 6 months of the study drug compared to baseline (LIC at baseline minus LIC at 6 months). This shows the mean reduction for the 3 subjects treated in this study. (NCT00602446)
Timeframe: 6 Months

Interventionmilligrams/gram (Mean)
Deferasirox Treated5.6

[back to top]

Absolute Change From Baseline in Liver Iron Concentration (LIC) to Year 1

Liver iron concentration (LIC), a predictor of iron burden, was measured using relaxation rate magnetic resonance imaging (R2-MRI) technique. Relaxation rate was determined as R2 = 1/relaxation time (T2). The baseline value of LIC of participants was categorized as < 7, ≥ 7 to < 15, and ≥ 15 milligram of iron/tissue dry weight (mg Fe/g dw). A negative change from baseline favored study treatment in reducing LIC. (NCT00631163)
Timeframe: From the Baseline, Year 1 (End of core study)

Interventionmg Fe/g dw (Mean)
Deferasirox-10.9

[back to top]

Correlation of LIC and Serum Ferritin at Core and Extension Study

LIC, a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. The correlation between absolute change in LIC and absolute change in serum ferritin was determined. (NCT00631163)
Timeframe: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)

InterventionCorrelation coefficient (Number)
Deferasirox (Core Study)0.291
Deferasirox (Extension Study)0.325

[back to top]

Absolute Change From Baseline in Liver Iron Concentration (LIC) in Japanese Subgroup

Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC was < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC. (NCT00631163)
Timeframe: From the Baseline, End of Year 1 (End of core study), End of Year 2 (End of extension study)

Interventionmg Fe/g dw (Mean)
Year 1Year 2
Deferasirox-13.9-18.4

[back to top]

Absolute Serum Ferritin Levels Over 2 Years

Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. (NCT00631163)
Timeframe: From the Baseline, Year 1 (End of core study), Year 2 (End of extension study)

,
Interventionnanogram(s)/millilitre (Mean)
Year 1Year 2
All Randomized Participants2653.32092.4
Japanese Participants2903.52114.8

[back to top]

Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Adverse Event of Special Interest (AESI), Discontinuation and Interruption

Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require hospitalisation, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Death was defined as a fatal event leading to permanent cessation of all vital functions of the body. (NCT00631163)
Timeframe: From the Baseline up to Year 2 (End of extension study)

Interventionparticipants (Number)
Adverse EventsSerious Adverse Events (SAE)Serious Adverse Events (SAE) with suspected relationship to study drugDeathAdverse event leading to discontinuation of study drugAdverse event leading to dose adjustment/interruptionAdverse Event of Special Interest (AESI)Adverse Event With Suspected Relationship to Study drug
All Randomized Participants974610614676265

[back to top]

Absolute Change From Baseline in Serum Ferritin Levels to Year 2

Serum ferritin was a marker for the monitoring of chelation therapy. Ferritin protein stores iron and provides overall iron levels, higher ferritin in blood showed more iron content. (NCT00631163)
Timeframe: From the Baseline up to Year 2 (End of extension study)

Interventionnanogram(s)/millilitre (Mean)
All Randomized Participants-677.9
Japanese Participants-892.8

[back to top]

Absolute Change From Baseline in Liver Iron Concentration (LIC) to End of Year 2

Liver Iron Concentration (LIC), a predictor of iron burden, was measured using R2-MRI technique. Relaxation rate was determined as R2 = 1/T2. The baseline value of LIC of participants was categorized as < 7, ≥ 7 to < 15, and ≥ 15 mg Fe/g dw. A negative change from baseline favoured study treatment in reducing LIC. (NCT00631163)
Timeframe: From the Baseline to End of Year 2 (End of extension study)

Interventionmg Fe/g dw (Mean)
Deferasirox-13.5

[back to top]

Number of Participants With Clinically Significant Ophthalmological Abnormalities

Clinically significant changes in left eye and right eye were assessed by the investigator based on methods like visual acuity, slit lamp examination, tonometry and fundus oculi. (NCT00631163)
Timeframe: At 2 years (End of extension study)

Interventionparticipants (Number)
NormalAbnormal, Clinically InsignificantAbnormal, Clinically SignificantNot AvailableTotal
All Randomized Participants24149350

[back to top]

Total Body Iron Elimination Rate (TBIE), Iron Intake, Iron Excretion/Iron Intake and Chelation Efficiency After 2 Years

Total body iron excretion (TBIE)was used to investigate the chelation efficacy of Deferasirox therapy. TBIE rate was estimated based on the iron influx as determined by the amount of red cells transfused and the change in total body iron (TBI) stores. (NCT00631163)
Timeframe: From the Baseline, Year 2 (End of extension study)

,
Interventionmg/kg/day (Mean)
TBIEIron intakeIron excretion/iron intakeChelation efficiency
Deferasirox (Extension Study)0.460.272.000.40
Japanese Participants0.540.272.440.50

[back to top]

Number of Participants With Polymorphisms in Genes Known to be, or Potentially Involved, in Deferasirox Disposition

Polymorphisms in genes known to be, or potentially involved, in deferasirox disposition: UGT1a1 (including the Gilbert syndrome promoter polymorphism, (TA)nTAA),UGT1a3, BRCP/ABCG2, MRP2/ABCC2. These genes were chosen because deferasirox is primarily eliminated by glucuronidation and subsequent biliary excretion. (NCT00749515)
Timeframe: 3 months

,
InterventionParticipants (Count of Participants)
UGT1a1UGT1a3BCRP/ABCG2MRP2/ABCC2
Adequate Responders (Control)5555
Inadequate Responders10101010

[back to top]

Half-Life of Deferasirox

"All patients received the same interventions of deferoxamine challenge, deferasirox dose with pharmacokinetic monitoring. Then we compared responses between patients who were known to be slow responders to deferasirox and those who were known to be rapid responders (chelated well).~Deferoxamine: After a 3-day washout period from all chelation, all patients have a 12 hour infusion of 50mg/kg of deferoxamine with urine collection and pre and post blood sampling to assess iron and Total Iron Binding Capacity (TIBC) by atomic absorption." (NCT00749515)
Timeframe: 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose.

Interventionhour (Mean)
Inadequate Responders6.08
Adequate Responders (Control)7.83

[back to top]

Area Under the Curve of Deferasirox After a Dose of 35 mg/kg

Area Under the Curve (AUC) 0 to 24 hours post dose (NCT00749515)
Timeframe: 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose

Interventionmicromole/liter*hour (Mean)
Inadequate Responders479.59
Adequate Responders (Control)1123.11

[back to top]

Clearance/Bioavailability of Deferasirox in Patients With Poor Response to Deferasirox Compared to Patients With Good Response After a Dose of 35 mg/kg

Clearance/bioavailability (CL/F) (NCT00749515)
Timeframe: 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose.

Interventionliter/hour (Mean)
Inadequate Responders1.30
Adequate Responders (Control)0.61

[back to top]

Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg

Volume of distribution/bioavailability (Vd/F) (NCT00749515)
Timeframe: 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose

Interventionliter (Mean)
Inadequate Responders10.33
Adequate Responders (Control)6.5

[back to top]

Volume of Distribution/Bioavailability of Deferasirox After a Dose of 35 mg/kg

Volume of distribution/bioavailability (Vd/F), adjusted per kilogram body weight (NCT00749515)
Timeframe: 0, 1, 2, 4, 6, 8, 12, and 24 hours post dose

Interventionliter/kilogram (Mean)
Inadequate Responders0.32
Adequate Responders (Control)0.13

[back to top]

Trough Plasma Concentration of Deferasirox at Week 8, Week 12 and Week 16

Blood samples were drawn at every visit as close as possible to 24 hours post dose from each subject participating in the study and trough plasma concentrations were estimated. (NCT00845871)
Timeframe: Pre-dose (0), 1, 2, 4 and 6 hour (post-dose) at Week 8, 12 and 16

Interventionmicromoles per litre (μmol/L) (Median)
Week 8, Deferasirox 20 mg/kgWeek 12, Deferasirox 20 mg/kgWeek 16, Deferasirox 20 mg/kgWeek 8, Deferasirox 30 mg/kgWeek 12, Deferasirox 30 mg/kgWeek 16, Deferasirox 30 mg/kg
Deferasirox36.3066.9527.7520.1534.3054.50

[back to top]

Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs), Discontinuation and Interruption

Adverse events (AEs) were defined as any unfavorable and unintended diagnosis, symptom, sign (including an abnormal laboratory finding), syndrome or disease which either occurs during study, having been absent at baseline, or, if present at baseline, appears to worsen. Serious adverse events (SAEs) were defined as any untoward medical occurrences that result in death, are life threatening, require (or prolong) hospitalization, cause persistent or significant disability/incapacity, result in congenital anomalies or birth defects, or are other conditions which in judgement of investigators represent significant hazards. Subjects who had permanently terminated from the treatment or kept the treatment on hold/deviated from protocol due to adverse event were defined as subjects with permanent discontinuation and temporary interruption, respectively. (NCT00845871)
Timeframe: Day 1 up to Week 16

,,,,,,
InterventionParticipants (Count of Participants)
AEsSAEsAEs leading to permanent discontinuationAEs leading to temporary interruptionDeath
Breakfast (Deferasirox With Liquid)60010
Breakfast (Deferasirox With Soft Food)105220
Deferasirox (Run-in Phase)395040
Dinner (Deferasirox With Liquid)61000
Dinner (Deferasirox With Soft Food)61000
No Meal (Deferasirox With Liquid)246020
No Meal Deferasirox With Soft Food)110100

[back to top]

Change From Baseline in Serum Ferritin at Week 16

Ferritin protein stores iron and provides overall iron levels. Higher ferritin in blood showed higher iron content. Fluctuations from normal serum ferritin levels (500 ng/mL) observed at two consecutive visits led to dose adjustment of deferasirox. (NCT00845871)
Timeframe: Baseline, Week 16 (End of study)

InterventionNanogram/mlillilitre (Mean)
Age 2 to < 10 yearsAge 10 to < 60 yearsAge ≥ 60 years
Deferasirox-198.138.3-593

[back to top]

Percentage of Participants With Differing Palatability Scores at Week 8 and Week 12

Palatability was assessed by participants based on a five-point Facial Hedonic scale defined as: dislike extremely; somewhat dislike; neither like or dislike; somewhat like; like extremely for the meal and method of administration. For participants under 5 years of age, the scale was completed by parent or caregiver. (NCT00845871)
Timeframe: Week 8 and Week 12

,,,,,
Interventionpercentage of participants (Number)
Week 8, Dislike extremelyWeek 8, Somewhat dislikeWeek 8, Neither like or dislikeWeek 8, Somewhat likeWeek 8, Like extremelyWeek 12, Dislike extremelyWeek 12, Somewhat dislikeWeek 12, Neither like or dislikeWeek 12, Somewhat likeWeek 12, Like extremely
Breakfast (Deferasirox With Liquid)028.628.6042.902525050
Breakfast (Deferasirox With Soft Food)1002030400016.733.350
Dinner (Deferasirox With Liquid)014.328.628.628.60033.333.333.3
Dinner (Deferasirox With Soft Food)0033.3066.700066.733.3
No Meal (Deferasirox With Liquid)05.929.417.647.14.34.356.58.726.1
No Meal Deferasirox With Soft Food)014.328.642.914.3010501030

[back to top]

Extension Study: Absolute Change in Serum Ferritin From Baseline to Eighth Quarter

Blood was collected for serum ferritin at Core Baseline and monthly during the Eighth quarter of the Extension Study. Absolute change from Baseline: quarterly average - baseline average. A negative change from baseline indicated improvement. (NCT00873041)
Timeframe: Core Baseline, Eighth Quarter (last 3 months of the study)

Interventionmicrograms/liter (Mean)
Deferasirox-565.9
Placebo/Deferasirox-504.3

[back to top]

Extension Study: Change From Baseline in Hemoglobin at Month 24

Blood was collected for Hemoglobin at Baseline and Month 24. Change from Baseline= Month 24 hemoglobin - Baseline hemoglobin. (NCT00873041)
Timeframe: Core Baseline, Month 24

Interventiong/L (Mean)
Deferasirox-2.6
Placebo/Deferasirox-3.1

[back to top]

Extension Study: Change From Baseline in Transferrin Saturation at Month 24

Blood was collected for transferrin saturation at Baseline and Month 24. Change from baseline= Month 24 transferrin saturation - baseline transferrin saturation. (NCT00873041)
Timeframe: Core Baseline, Month 24

InterventionPercent saturation (Mean)
Deferasirox-5.01
Placebo/Deferasirox1.35

[back to top]

Extension Study: Change in Liver Iron Concentration (LIC) From Baseline at Month 24

LIC was measured by magnetic resonance imaging technique at Baseline and Month 24. A negative change from baseline indicated improvement. (NCT00873041)
Timeframe: Core Baseline, Month 24

Interventionmg iron (Fe)/g dry weight (dw) (Mean)
Deferasirox-7.1
Placebo/Deferasirox-6.7

[back to top]

Extension Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)

"The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for serum ferritin difference from Baseline at Month 24 versus LIC difference from Baseline at Month 24.~A value of 1.0 indicates a perfect correlation." (NCT00873041)
Timeframe: Core Baseline, Month 24

InterventionCorrelation coefficient (Number)
All Randomized Participants0.735

[back to top]

Extension Study: Percentage of Participants Reaching a Liver Iron Concentration (LIC) < 5 mg Fe/g dw From Core Baseline to End of Extension Study

Liver iron concentration was measured at Core Baseline and at the end of the Extension Study. Magnetic Resonance Imaging (MRI) scans were analyzed at a central laboratory to determine the LIC value. The percentage of participants with LIC < 5 mgFe/g dw (milligram iron/gram dry weight) change from Baseline at the end of the Extension Study is reported. (NCT00873041)
Timeframe: Core Baseline to End of Extension Study (up to 24 months)

InterventionPercentage of participants (Number)
Deferasirox39.1
Placebo/Deferasirox37.5

[back to top]

Core Study: Change in Liver Iron Concentration (LIC) From Baseline At Week 24 and Week 52 in Patients With Dose Increases After Week 24

LIC was measured by magnetic resonance imaging technique at baseline, Week 24 and Week 52. Dose Doubling (Dose Increases) began at Week 24. (NCT00873041)
Timeframe: Baseline, Week 24, Week 52

,,
Interventionmg iron (Fe)/g dry weight (dw) (Mean)
Change from baseline at Week 24 (n=26,24,30)Change from baseline at Week 52
10 mg/kg/Day Deferasirox0.69-4.02
5 mg/kg/Day Deferasirox0.56-1.82
Placebo0.940.62

[back to top]

Core Study: Correlation Between Serum Ferritin and LIC (Liver Iron Concentration)

"The correlation between serum ferritin and LIC was investigated using a scatter plot with a regression line for the following cases:~Baseline serum ferritin versus baseline LIC~Serum ferritin difference from baseline at fourth quarter versus difference from baseline in LIC at Week 52.~A value of 1.0 indicates a perfect correlation." (NCT00873041)
Timeframe: Baseline, 52 weeks

InterventionCorrelation coefficient (Number)
BaselineWeek 52 (n=134)
All Randomized Participants0.6530.609

[back to top]

Core Study: Percentage of Participants With Adverse Events Graded Mild, Moderate and Severe

Percentage of Participants with Mild, Moderate and Severe adverse events (AE) any primary system organ class regardless of study drug relationship. A patient with multiple occurrences of an AE is counted only once in the AE category for that treatment. A patient with multiple severity ratings for an AE while on a treatment is only counted once under the maximum rating. (NCT00873041)
Timeframe: 52 Weeks

,,
InterventionPercentage of participants (Number)
MildModerateSevere
10 mg/kg/Day Deferasirox43.616.418.2
5 mg/kg/Day Deferasirox36.427.312.7
Placebo42.921.416.1

[back to top]

Core Study: Percentage of Participants With Notable Abnormal Post-baseline Laboratory Results

"The percentage of participants with notable laboratory results:~Platelet count: (<100 x 10^9/L)~Absolute neutrophils: (<1.5 x 10^9/L)~Alanine aminotransferase (ALT): (>5 x Upper limit normal (ULN) and >2 x baseline).~Aspartate aminotransferase (AST): (>5 x ULN and >2 x baseline)~Serum creatinine: (>33% increase from baseline and >ULN at ≥2 consecutive post-baseline values) Creatinine clearance: (<60 mL/min at ≥2 consecutive post-baseline values)~Urinary protein/creatinine ratio: (≥ 1.0 mg/mg at ≥2 consecutive post-baseline values)" (NCT00873041)
Timeframe: 52 Weeks

,,
InterventionPercentage of participants (Number)
Platelet countAbsolute neutrophilsALTASTSerum creatinineCreatinine clearanceUrinary protein/creatinine ratio
10 mg/kg/Day Deferasirox5.53.601.85.51.80
5 mg/kg/Day Deferasirox5.55.50001.81.8
Placebo10.75.41.81.8000

[back to top]

Core Study: Percentage of Participants With Notably Abnormal Post-baseline Diastolic Blood Pressure

"Diastolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.~A Notably Abnormal Diastolic Blood Pressure was defined as a measurement in one of the following two categories:~High: ≥105 with an increase from baseline ≥15 mmHg~Low: ≤50 with a decrease from baseline ≥15 mmHg" (NCT00873041)
Timeframe: Baseline, 52 Weeks

,,
InterventionPercentage of participants (Number)
HighLow
10 mg/kg/Day Deferasirox0.010.9
5 mg/kg/Day Deferasirox0.014.5
Placebo0.014.3

[back to top]

Core Study: Percentage of Participants With Notably Abnormal Post-baseline Pulse Rate

"Pulse Rate was measured at each visit.~A Notably Abnormal Pulse Rate was defined as a measurement in one of the following two categories:~High: ≥120 with an increase from baseline ≥15 beats per minute (bpm)~Low: ≤50 with a decrease from baseline ≥15 bpm" (NCT00873041)
Timeframe: Baseline, 52 Weeks

,,
InterventionPercentage of participants (Number)
HighLow
10 mg/kg/Day Deferasirox0.01.8
5 mg/kg/Day Deferasirox1.80.0
Placebo3.60.0

[back to top]

Core Study: Percentage of Participants With Notably Abnormal Post-baseline Systolic Blood Pressure

"Systolic blood pressure was measured at each visit after the patient rested in the sitting position for at least 3 minutes.~A Notably Abnormal Systolic Blood Pressure was defined as a measurement in one of the following two categories:~High: ≥180 with an increase from baseline ≥20 mmHg~Low: ≤90 with a decrease from baseline ≥20 mmHg" (NCT00873041)
Timeframe: Baseline, 52 Weeks

,,
InterventionPercentage of participants (Number)
HighLow
10 mg/kg/Day Deferasirox0.05.5
5 mg/kg/Day Deferasirox0.010.9
Placebo1.816.1

[back to top]

Core Study: Change From Baseline in Hemoglobin at Month 12

Blood was collected for Hemoglobin at baseline and Month 12. Change from baseline= Month 12 hemoglobin - baseline hemoglobin. (NCT00873041)
Timeframe: Baseline, Month 12

Interventiong/L (Mean)
5 mg/kg/Day Deferasirox-1.8
10 mg/kg/Day Deferasirox-0.7
Placebo-2.8

[back to top]

Core Study: Change From Baseline in Transferrin Saturation at Month 12

Blood was collected for transferrin saturation at Baseline and Month 12. Change from baseline= Month 12 transferrin saturation - baseline transferrin saturation. (NCT00873041)
Timeframe: Baseline, Month 12

InterventionPercent saturation (Mean)
5 mg/kg/Day Deferasirox-3.79
10 mg/kg/Day Deferasirox-3.64
Placebo3.37

[back to top]

Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 24

LIC was measured by magnetic resonance imaging technique at baseline and Week 24. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 24 with treatment as factor and baseline LIC as covariate. (NCT00873041)
Timeframe: Baseline, Week 24

Interventionmg iron (Fe)/g dry weight (dw) (Least Squares Mean)
5 mg/kg/Day Deferasirox-0.87
10 mg/kg/Day Deferasirox-0.90
Placebo-0.24

[back to top]

Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52

LIC was measured by magnetic resonance imaging technique at baseline and Week 52. Estimates were obtained from an Analysis of Covariance (ANCOVA) model for change in LIC between baseline and Week 52 with treatment as factor and baseline LIC as covariate. (NCT00873041)
Timeframe: Baseline, Week 52

Interventionmg iron (Fe)/g dry weight (dw) (Least Squares Mean)
5 mg/kg/Day Deferasirox-1.95
10 mg/kg/Day Deferasirox-3.80
Placebo0.38

[back to top]

Core Study: Change in Liver Iron Concentration (LIC) in Placebo Patients From Baseline to Week 52

LIC was measured by magnetic resonance imaging technique at baseline and Week 52. The change in liver iron concentration for participants in the placebo arm was used to assess the iron accumulation rate. (NCT00873041)
Timeframe: Baseline, Week 52

Interventionmg iron (Fe)/g dry weight (dw) (Mean)
Placebo0.26

[back to top]

Core Study: Change in Serum Ferritin Between Baseline and Fourth Quarter

"Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.~Fourth quarter serum ferritin average was the average of all serum ferritin values obtained within days 286- End of Study.~Change from baseline: fourth quarter serum ferritin average - baseline serum ferritin average." (NCT00873041)
Timeframe: Baseline, (Day 286 to End of Study [Day 365])

Interventionμg/L (Mean)
5 mg/kg/Day Deferasirox-130.47
10 mg/kg/Day Deferasirox-249.16
Placebo128.63

[back to top]

Core Study: Change in Serum Ferritin Between Baseline and Second Quarter

"Baseline serum ferritin average was the average of all available ferritin values from screening to last sample prior to the first intake of study drug.~Second quarter serum ferritin average was the average of all serum ferritin values obtained within days 106-195.~Change from baseline: second quarter serum ferritin average - baseline serum ferritin average." (NCT00873041)
Timeframe: Baseline, (Day 106 to Day 195)

Interventionμg/L (Mean)
5 mg/kg/Day Deferasirox8.20
10 mg/kg/Day Deferasirox-17.75
Placebo106.45

[back to top]

Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months

Change in liver iron concentration from baseline to 12 months with the use of combined chelation therapy. The change was calculated as the liver iron concentration at 12 months minus the value at baseline. (NCT00901199)
Timeframe: 12 months

Interventionmg/g (Median)
Deferasirox (Exjade) and Desferal (DFO)-6.2

[back to top]

Change in Serum Creatinine During 12 Months Combined Chelation Therapy

Comparison of average serum creatinine over 12 months of combined chelation therapy compared with baseline serum creatinine. (NCT00901199)
Timeframe: 12 months

Interventionmg/dl (Mean)
Deferasirox (Exjade) and Desferal (DFO)0.09

[back to top]

Percentage of Participants With Significant Renal Dysfunction

Significant renal dysfunction was defined as a serum creatinine value ≥ 2 times upper limit of normal (ULN) at two consecutive assessments at least 7 days apart (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox0.7
Placebo0

[back to top]

Time to at Least a 10% Increase From Baseline in Left Ventricular End-diastolic Internal (LVIDD) at Two Consecutive Assessments at Least Two Weeks Apart

Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVIDD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVIDD was available. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
DeferasiroxNA
PlaceboNA

[back to top]

Percentage of Participants With Newly Occurring Moderate or Severe Neutropenia

Moderate or severe neutropenia was defined as neutrophil counts less than 1.0×10E9/L. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox27.7
Placebo26.3

[back to top]

Percentage of Participants With Newly Occurring Severe Thrombocytopenia

Severe thrombocytopenia was defined as platelets counts less than 50×10E9/L. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox10.1
Placebo19.7

[back to top]

Percentage of Participants With Worsening Glucose Metabolism Compared to Baseline

As assessed by an annual glucose tolerance test (OGTT). Worsening glucose metabolism was defined as an increase in glucose metabolism category (normal, impaired glucose metabolism, diabetes mellitus) based on the American Diabetes Association criteria (American Diabetes Association 2009) compared to the baseline result. The last available lab assessment date was used as the cut-off date for the analysis. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox18.1
Placebo18.4

[back to top]

All Collected Deaths

On-treatment deaths were collected from FPFT up to 28 days after study drug discontinuation, for a maximum duration of 2627 days (2599 plus 28 days) (treatment duration ranged from 1 day to 2599 days). Deaths post treatment survival follow up were collected after the on treatment period, up to approx. 7.4 years. Patients who had not experienced any of the non-fatal events from the composite primary endpoint and had not stopped study participation at the time of data cut-off (end of study) were censored. (NCT00940602)
Timeframe: 2627 days, approx. 7.4 years

,
InterventionParticipants (Number)
Total deathsDeaths on-treatmentDeaths post-treatment survival follow up
Deferasirox572433
Placebo331023

[back to top]

Total Number of Infections Requiring Intravenous Antimicrobials

"The total number of infections were counted and summarized per treatment group. For this number, one participant can contribute more than one infection event. Infections were determined from the reported AEs with system organ class Infections and infestations and action taken Concomitant medication taken. Antimicrobial therapy was determined from the reported concomitant medications for participants who had an infection AE. The route of administration needed to be specified as intravenous (i.v.). End of treatment period was defined as the treatment period plus 28 days." (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventioninfections (Number)
Deferasirox253
Placebo111

[back to top]

Time to Study Drug Discontinuation Due to an AE or Laboratory Abnormality

As recorded on the Study Treatment Completion electronic Case Report Form (eCRF), date and reason given.Only participants for whom the reason for stopping study medication was entered as AE or laboratory abnormality were considered. This time to event endpoint was calculated as the date of study drug discontinuation due to an AE or laboratory abnormality minus date of randomization plus 1. Participants who did not discontinue study medication due to an AE or laboratory abnormality were censored at the date of study drug discontinuation. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
DeferasiroxNA
Placebo1022

[back to top]

Time to First Occurrence of Serum Ferritin Level >2 Times the Baseline Value at Two Consecutive Assessments (at Least Two Weeks Apart)

Assessed by blood draw and calculated as follows: Date of first occurrence of serum ferritin >2 times the baseline value at two consecutive assessments (at least two weeks apart), minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when serum ferritin was available. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
DeferasiroxNA
Placebo592

[back to top]

Time to Disease Progression

"Disease progression was defined as follows:~MDS progression: Transition into a higher MDS risk group based on IPSS scoring~Progression to AML: 20 percent or more blasts seen in the bone marrow collected by biopsy or aspirate.~Disease progression was calculated as follows: Date of diagnosis of MDS progression or date of first diagnosis of AML, minus date of randomization plus 1. Participants who neither experienced MDS progression nor progression to AML were censored at the last contact date." (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
DeferasiroxNA
PlaceboNA

[back to top]

Time to at Least a 10% Increase From Baseline in Left Ventricular Internal Systolic Diameter (LVISD) at Two Consecutive Assessments at Least Two Weeks Apart

Assessed by echocardiography and calculated as follows: Date of echocardiography assessment where a minimum of 10% increase of LVISD first occurred, minus date of randomization plus 1. Participants who did not experience such an increase were censored at the last date when LVISD was available. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
Deferasirox1179.0
PlaceboNA

[back to top]

Percentage of Participants With Newly Occurring Hypothyroidism Compared to Baseline

"As assessed by annual measurement of Thyroid Stimulating Hormone (TSH) and free T4. Hypothyroidism was defined as follows and is inclusive of:~Primary hypothyroidism: serum TSH >upper limit of normal (ULN) and free T4 ULN and a free T4 within normal limits. The last available lab assessment date was used as the cut-off date for the analysis." (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox5.4
Placebo3.9

[back to top]

Percentage of Participants With Major Gastrointestinal Bleeding

Major gastrointestinal bleeding was defined as an AE that could include one of the following MedDRA preferred terms: gastric hemorrhage, gastrointestinal hemorrhage, small intestinal hemorrhage, esophageal hemorrhage, large intestinal hemorrhage, rectal hemorrhage, melaena, duodenal ulcer hemorrhage, gastric ulcer hemorrhage, peptic ulcer hemorrhage, large intestinal ulcer hemorrhage, esophageal ulcer hemorrhage, and hematochezia. The end of treatment period was defined as the treatment period plus 28 days. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox5.4
Placebo3.9

[back to top]

Percentage of Participants With Hematologic Improvement (HI) in Terms of Erythroid Response

"HI in terms of erythroid responses was assessed based on International Working Group (IWG) criteria, with improvement defined as follows:~Hemoglobin increase of ≥ 1.5 g/dL OR~Reduction of ≥ 4 RBC transfusions/8 weeks in comparison to pre-treatment values and lasting at least 8 weeks. The last hemoglobin value measured prior to randomization was used as the pre-treatment value. The last available lab assessment date was used as the cut-off date for the analysis." (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventionpercentage of participants (Number)
Deferasirox39.6
Placebo27.6

[back to top]

Event-free Survival

Event-free survival was defined as the time from the date of randomization to the date of the first documented non-fatal event (worsening cardiac function, hospitalization for congestive heart failure, liver function impairment, liver cirrhosis, transformation to AML, as defined in the protocol), or death, whichever occurred first. Participants who did not experience a non-fatal event as of the time of data cut-off (end of study), as well as participants who did not experience a non-fatal event and stopped study participation before the data cut-off, were censored as specified in the protocol. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7 years

Interventiondays (Median)
Deferasirox1440
Placebo1091

[back to top]

Overall Survival

Overall survival was calculated as the date of death (irrespective of cause) minus date of randomization plus 1. (NCT00940602)
Timeframe: Day 1 to end of treatment period, approx. 7.4 years

Interventiondays (Median)
Deferasirox1907
Placebo1509

[back to top]

Relative Change in Serum Ferritin From Baseline to 3.5 Years

The mean percentage change in serum ferritin was evaluated by comparing the serum ferritin level at the start of Deferasirox treatment to the serum ferritin level collected 18 months following the start of the extension study. Serum ferritin is measured in micrograms per Liter. Relative Change = 1- (Change in ferritin level from Baseline/Baseline level) x 100. (NCT01033747)
Timeframe: Baseline to 3.5 years

InterventionPercent change (Mean)
Deferasirox32.4
Deferasirox Crossover33.2

[back to top]

The Relative Change From Baseline in Liver Iron Content (LIC) After Prolonged Use of Deferasirox

The mean percentage change in liver iron content (LIC) as assessed by superconducting quantum interference device (SQUID) was evaluated by comparing the LIC at the start of Deferasirox treatment to the LIC at the end of the 5 year extension study for participants who were treated with Deferasirox for more than 3.5 years. LIC is expressed in milligrams of iron per gram of liver dry weight (mgFe/g dw). Relative change = 1- (Change in LIC from Baseline/Baseline level) x 100. (NCT01033747)
Timeframe: Baseline to 7 Years

InterventionPercent change (Mean)
Deferasirox-21.9
Deferasirox Crossover-23.1

[back to top]

Number of Participants With Adverse Events After Start of ICL670

Safety as assessed by the number of participants with adverse event or death after the start of ICL670. (NCT01090323)
Timeframe: 0 - 60 months

,
Interventionparticipants (Number)
Adverse EventsDeaths
Crossover492
ICL6701311

[back to top]

Change in Serum Ferritin From Start of ICL670 to End of Study

The main efficacy variable was change in serum ferritin in response to therapy with ICL670. Due to variability of serum ferritin, end of study was considered as the mean of at most the last 3 available observations after the start of ICL670. (NCT01090323)
Timeframe: 0 - 60 months

Interventionµg/L (Median)
ICL670-245.5
Crossover-134.3

[back to top]

Number of Patients With Elevated Labile Plasma Iron (LPI) Above Threshold (0.5 Umol/L)

(NCT01159067)
Timeframe: At baseline

InterventionParticipants (Count of Participants)
Arm I0

[back to top]

Number of Patients With Serum Ferritin Level Lower Than 1500 ng/mL After Treatment

Number of patients, whose Serum Ferritin levels are lower than 1500 ng/mL at two consecutive study visits. Serum Ferritin levels are measured at screening (baseline), week 4, 8, 12, 16, 20, 24 and end of study. (NCT01159067)
Timeframe: Assessed through 6 months from the start of treatment

InterventionParticipants (Count of Participants)
Arm I1

[back to top]

Percentage of Participants With T2*>=10 ms and at Least 10% Relative Increase From Baseline at Month 6, 12, 18 and 24

The number of evaluable participants at each visit were used as the denominator for the calculation of proportion at each visit. (NCT01254227)
Timeframe: From the Months 6, 12, 18 and 24

Interventionpercentage of participants (Geometric Mean)
Month 6Month12Month 18Month 24
All Participants12.5019.2333.3347.22

[back to top]

Time to Achieve From Baseline (FAS) of at Least 10% at Month 24

Time from date of start of study treatment to date when first achieving T2* ≥ 10 ms (but at least 10% relative increase from baseline) was summarized using the reverse Kaplan-Meier estimates (1 - Kaplan-Meier estimates) for the FAS. (NCT01254227)
Timeframe: At 24 months

Interventionmilliseconds/ms (Median)
All Participants722.0

[back to top]

Cardiac Iron Concentration Levels From Baseline and at Month 6, 12, 18 and 24

Cardiac iron concentration (mg Fe/g dw) was quantified using the formula (cardiac iron concentration (mg Fe/g dw) = 45 * T2* (ms) ^ (-1.22) and analyzed over time. (NCT01254227)
Timeframe: From the Baseline, Month 6, 12, 18 and Month 24

Interventionmg Fe/g dw (Mean)
BaselineMonth 6Month 12Month 18Month 24
All Participants4.184.313.933.513.14

[back to top]

Change in Cardiac Iron Content From Baseline to Month 6,18 and 24

The change in cardiac iron content was calculated as ratio of Cardiac T2* at different time points; the efficacy endpoint analyses were performed on the Full Analysis Set (FAS). (NCT01254227)
Timeframe: From Baseline to Months 6, 18 and 24

Interventionratio (Geometric Mean)
Month 6Month 18Month 24
All Participants1.021.171.30

[back to top]

Change in Right Ventricular Ejection Fraction (RVEF) From Baseline to Month 6, 12, 18 and 24

Magnetic resonance imaging (MRI)-measured cardiac T2* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader. (NCT01254227)
Timeframe: From the Months 6, 12, 18 and 24

InterventionPercent Ejection Fraction (Mean)
BaselineMonth 6Month 12Month 18Month 24
All Participants67.1-1.2-1.6-2.1-1.4

[back to top]

Change in Left Ventricular Ejection Fraction (LVEF) From Baseline to Month 6, 12, 18 and 24

Magnetic resonance imaging (MRI)-measured cardiac T2* and cardiac function reflected by left and right ventricle ejection fraction. A standardized MRI protocol for T2* acquisition technique will be used in the centers. Images will be reviewed centrally by an expert MRI reader. (NCT01254227)
Timeframe: From the Months 6, 12, 18 and 24

InterventionPercent Ejection Fraction (Mean)
BaselineMonth 6Month 12Month 18Month 24
All Participants66.50.1-0.20.60.9

[back to top]

Change in Cardiac Iron Content From Baseline to Month 12

Cardiac T2* is the most sensitive and reproducible test in detecting myocardial iron load. A cardiac T2* value of <10 ms is defined as severe cardiac iron overload. Participants who do not have baseline T2* or do not have any post-baseline T2* are excluded from the analysis. (NCT01254227)
Timeframe: From Baseline to Month 12

Interventionratio (Geometric Mean)
All Participants1.09

[back to top]

Changes in Mean Neutrophil Values (as Measured by Lab) for Arm 1 (Other Arms Were Used for Calibration Only)

Changes in Neutrophils between baseline and mean neutrophils values during treatment (measured after each dose) (NCT01273766)
Timeframe: Baseline, up to 6 months

Intervention10^9 Neutrophils per Liter (Mean)
Arm I7.8

[back to top]

Need for Hospitalization, Ventilator Support, Exchange Transfusion/Apheresis or Treatment With Antifungals or Antibiotics

Records will be assessed at baseline and prospectively while on study. (NCT01273766)
Timeframe: Baseline, up to 6 months

,
InterventionParticipants (Count of Participants)
Baseline : HospitalizationBaseline : VentilatorBaseline : TransfusionBaseline : Antibiotics1 Month : Hospitalization1 Month : Ventilator1 Month : Transfusion1 Month : Antibiotics2 Month : Hospitalization2 Month : Ventilator2 Month : Transfusion2 Month : Antibiotics3 Month : Hospitalization3 Month : Ventilator3 Month : Transfusion3 Month : Antibiotics4 Month : Hospitalization4 Month : Ventilator4 Month : Transfusion4 Month : Antibiotics5 Month : Hospitalization5 Month : Ventilator5 Month : Transfusion5 Month : Antibiotics6 Month : Hospitalization6 Month : Ventilator6 Month : Transfusion6 Month : Antibiotics
Arm I2000100100000000000100003001
Correlative5105101110111010111120010000

[back to top]

Cumulative Incidence of Documented Bacterial, Fungal, and Viral Infections

Records will be assessed at baseline and prospectively while on study. (NCT01273766)
Timeframe: Baseline, up to 6 months

InterventionParticipants (Count of Participants)
Arm I2
Correlative5

[back to top]

Left Ventricular Ejection Fraction (LVEF)

LVEF % was measured by cardiac magnetic resonance (CMR). (NCT01459718)
Timeframe: 6, 12, 18, 24 months

InterventionPercentage of ejection fraction (Mean)
Baseline6 months12 months18 months24 months
Deferasirox / Deferasirox + Deferoxamine (DFO)6565.464.865.166.2

[back to top]

Time to Response

Time to response was defined as the time from baseline when the participant had severe cardiac iron overload to the time when the participant achieved mild/moderate cardiac overload (T2*>10 milliseconds [ms]). (NCT01459718)
Timeframe: 24 months

Interventionms (Mean)
Deferasirox / Deferasirox + Deferoxamine (DFO)13.0

[back to top]

Number of Patients Achieving a Complete Response (CR)

Complete Response is defined as patients that stop intensive deferasirox -DFO treatment, at any time point during the 24 months of study, based on an improvement in the cardiac Magnetic Resonance Imaging T2 star technique (MRI T2*) value being >10ms, and continue to be treated with deferasirox monotherapy without any further need for reverting back to intensive iron chelation treatment during the 24 months of study. (NCT01459718)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Deferasirox / Deferasirox + Deferoxamine (DFO)4

[back to top]

Number of Patients Achieving a Partial Response (PR)

Partial Response is defined as patients that stop intensive deferasirox -DFO treatment at any time point during the 24 months study and transition to receive deferasirox monotherapy, but due to a deterioration in cardiac MRI T2* to a value < 10 ms revert back to intensive deferasirox -DFO iron chelation therapy during the 24 months of study. (NCT01459718)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Deferasirox / Deferasirox + Deferoxamine (DFO)0

[back to top]

Number of Patients With Stable Disease (SD)

Stable Disease is defined as those patients that never achieved an improvement in the cardiac MRI T2* to values >10ms during the 24 months of study. (NCT01459718)
Timeframe: 24 months

InterventionParticipants (Count of Participants)
Deferasirox / Deferasirox + Deferoxamine (DFO)8

[back to top]

Change From Baseline in Cardiac Iron Overload of Patients in Intensive Iron Chelation Therapy Consisting of Deferasirox-DFO and After Transition to Deferasirox Monotherapy

Cardiac iron overload was determined by cardiac MRI T2*. Cardiac iron overload also was measured by the monthly velocity of heart MRI T2*. (NCT01459718)
Timeframe: Baseline, 6, 12, 18, 24 months

InterventionMilliseconds (ms) (Mean)
6 months12 months18 months24 months
Deferasirox / Deferasirox + Deferoxamine (DFO)0.10.71.32.4

[back to top]

Change From Baseline in Liver Iron Concentration (LIC)

Change from baseline in LIC was determined by change in liver MRI T2*. (NCT01459718)
Timeframe: Baseline, 6, 12, 18, 24 months

Interventionmg of iron/gram of dry weight of liver (Mean)
6 months12 months18 months24 months
Deferasirox / Deferasirox + Deferoxamine (DFO)-5-10.3-10.2-12.4

[back to top]

Correlation Between Change From Baseline in Serum Ferritin and LIC Levels

Spearman correlation coefficients between serum ferritin and LIC changes from baseline levels were reported. (NCT01459718)
Timeframe: Baseline, 6, 12, 18, 24 months

InterventionSpearman correlation coefficient (Number)
6 months12 months18 months24 months
Deferasirox / Deferasirox + Deferoxamine (DFO)0.091-0.0330.3470.273

[back to top]

The Percentage of Patients Reaching Serum Ferritin Levels Lower Than 500 μg/L

Serum Ferritin values between 1000-2500 μg/L are indicative of mild to moderate iron overload while values >2500 μg/L are indicative of severe iron overload and levels constantly higher than 2500 μg/L has been shown to to increase the risk of cardiac complications and endocrine disease. Maintaining levels <1000 μg/L is associated with increased survival and less morbidity. (NCT01610297)
Timeframe: Week 28 and Week 52

InterventionPercentage of Patients (Number)
Week 28 (n=26)Week 52 (n=27)
ICL6707.733.3

[back to top]

Change in Serum Ferritin Level.

Blood samples were collected and serum levels were assessed at study baseline (BL) and at 12 months. (NCT01610297)
Timeframe: Baseline, 12 Months

Interventionng/mL (Mean)
BaselineMonth 12
ICL6701766.81903.56

[back to top]

Change in the Further Parameters of Iron Overload (Cardiac Iron Concentration by Magnetic Resonance Imaging (MRI Examination)

Cardiac MRI values between 10 to 20 milliseconds (ms) are indicative of moderate cardiac iron deposition associated with declining left ventricular ejection fraction and arrhythmias while values <10 ms are indicative of deposition sufficient to risk cardiac decompensation and associated with overt heart failure and mortality. (NCT01610297)
Timeframe: Baseline, 12 month

Interventionms (Mean)
Baseline Cardiac MRI(n= 27)Week 52 Cardiac MRI (n=24)
ICL67026.4828.25

[back to top]

Change in the Further Parameters of Iron Overload (Liver Iron Concentration by Magnetic Resonance Imaging (MRI Examination)

Liver Iron Concentration (LIC) values between 3 and 7 mg Fe / g dry weight are indicative of mild iron deposition, while values between 7 and 15 mg Fe / g dry weight are indicative of moderate iron deposition which have been associated with liver disease. Values >15 mg Fe/g dry weight are indicative of severe iron deposition which is associated with progressive liver fibrosis, increased morbidity and mortality (NCT01610297)
Timeframe: Baseline, 12 month

Interventionmg Fe/g dry liver weight (Mean)
Baseline Liver MRI(n= 27)Week 52 Liver MRI (n=25)
ICL67012.074.62

[back to top]

Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability

To determine the safety; incidence, type and severity of adverse events including renal, hepatic, biochemistry and hematologic parameters of deferasirox in the treatment of iron overload after hematopoietic stem cell transplantation (HSCT) in patients with beta-thalassemia major in 12 months period (NCT01610297)
Timeframe: 12 months

InterventionParticipants (Number)
Adverse eventsSerious adverse eventsDeath
ICL6702530

[back to top]

Number of Participants With Improvement in Cardiac T2* MRI

Improvement in Cardiac T2* MRI from baseline to determine if there is a reduction of cardiac iron burden. (NCT01709032)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Deferasirox and Deferiprone Combination Chelation3

[back to top]

Number of Participants With Improvement in Liver Iron Concentration

Determine the safety of the combination of Deferasirox and Deferiprone for the treatment of subjects with Thalassemia Major and Severe Iron Overload by assessing change in liver iron concentration from baseline to follow-up (NCT01709032)
Timeframe: 12 months

InterventionParticipants (Count of Participants)
Deferasirox and Deferiprone3

[back to top] [back to top]

Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline

Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment (NCT01709838)
Timeframe: Baseline, 52 weeks

Interventionmg Fe/g dw (Mean)
Deferasirox-6.68

[back to top]

Absolute Change in Serum Ferritin From Baseline After 52 Weeks

Absolute change in serum ferritin from baseline after 52 weeks of treatment (NCT01709838)
Timeframe: Baseline, 52 weeks

Interventionng/mL (Mean)
Deferasirox-494.64

[back to top]

Percentage of Participants With Baseline LIC>15 Achieving LIC<5 mg Fe/g dw

The percentage of participants with baseline LIC>15 mg Fe/g dw achieving an LIC <5 mg Fe/g dw during the study (NCT01709838)
Timeframe: 5 years

Interventionpercentage of participants (Number)
Deferasirox51.0

[back to top]

PK Parameters: AUCtau

The pharmacokinetic parameter, AUCtau was determined using non-compartmental method(s) for deferasirox and its iron complex. AUC=area under the concentration-time curve during a dosing interval at steady state (amount × time × volume). (NCT01709838)
Timeframe: pre-dose (0 hour), and at 2, and 4 hours at Week 4

Interventionhr*umol/L (Geometric Mean)
Deferasirox678.2

[back to top]

PK Parameters: Cmax

The pharmacokinetic parameter, Cmax, was determined using non-compartmental method(s) for deferasirox and its iron complex. Cmax (maximum/peak plasma drug concentration after drug administration)=amount × volume (NCT01709838)
Timeframe: pre-dose (0 hour), and at 2, and 4 hours at Week 4

Interventionumol/L (Geometric Mean)
Deferasirox53.367

[back to top]

PK Parameters: Tmax

The pharmacokinetic parameter, Tmax, may be determined using non-compartmental method(s) for deferasirox and its iron complex. Tmax=time to reach maximum/peak concentration following drug administration. (NCT01709838)
Timeframe: pre-dose (0 hour), and at 2, and 4 hours at Week 4

Interventionhr (Geometric Mean)
Deferasirox2.5131

[back to top]

Time From Target LIC of 3 mg Fe/g dw to the First LIC ≥5 mg Fe/g dw in the Follow up Period

Time from the target LIC <3 mg Fe/g dw to the first LIC ≥5 mg Fe/g dw in the follow-up period (NCT01709838)
Timeframe: post-baseline, up to 260 weeks

Interventiondays (Median)
Deferasirox27.4

[back to top]

Time to Achieving LIC <5 mg Fe/g dw

Time to achieving LIC <5 mg Fe/g dw for participants with baseline LIC>15 mg Fe/g dw during the study (NCT01709838)
Timeframe: 5 years

Interventionmonths (Median)
Deferasirox36.3

[back to top] [back to top]

Absolute Change in LIC From Baseline After 52 Weeks of Treatment by Underlying Non-transfusion Dependent Thalassemia (NTDT) Syndrome

Absolute change in liver iron concentration measured by MRI from baseline after 52 weeks of treatment by underlying NTDT syndrome. The 4 underlying disease types: Beta-thalassemia intermedia (N =69), HbE beta-thalassemia (N = 24), Alpha-thalassemia intermedia (HbH disease) (N = 40), Other, specify (N = 1) (NCT01709838)
Timeframe: Baseline, 52 Weeks

Interventionmg Fe/g dw (Mean)
Beta-thalassemia intermediaHbE beta-thalassemiaAlpha-thalassemia intermedia (HbH disease)Other, specify
Deferasirox-6.11-6.18-7.97-6.00

[back to top]

Correlation Analysis for Absolute Change in LIC and Serum Ferritin at Week 24 and EOS (Week 260 + 30 Days Follow-up)

Correlation for absolute change between LIC and serum ferritin was assessed using scatter plots with pearson correlation coefficient and simple linear model. (NCT01709838)
Timeframe: Week 24, End of Study (EOS): Week 260 + 30 days follow up

Interventioncorrelation coefficient (Number)
Week 24Change from Baseline at EOS
Deferasirox0.2990.740

[back to top]

Absolute Change in LIC From Baseline Over Time

Absolute change in serum ferritin from baseline over time up to 260 weeks (NCT01709838)
Timeframe: 24, 52, 76, 104, 128, 156, 180, 208, 232, 260 Weeks

Interventionmg Fe/g dw (Mean)
Week 24Week 52Week 76Week 104Week 128Week 156Week 180Week 208Week 232Week 260
Deferasirox-3.67-7.02-8.93-9.63-10.03-10.20-9.94-10.04-10.58-10.57

[back to top]

Plasma Pharmacokinetics (PK) Deferasirox Concentrations

Blood samples for PK evaluation were collected for a sub-group of patients. The patient had to have been on treatment without dose adjustment or treatment interruption (for any reason) for at least 4 consecutive days prior to scheduled PK sampling visit. If there was a dosage change or interruption within 4 days of the visit, no PK blood samples was collected, and an appropriate comment had to be made on the PK CRF page. (NCT01709838)
Timeframe: Weeks 12 & 24: pre-dose (0hr), 2hr & 4hr post-dose

Interventionhr*umol/L (Geometric Mean)
4 weeks: 0hr pre-dose4 weeks: 2hr post-dose4 weeks: 4hr post-dose
Deferasirox6.51348.55644.652

[back to top]

Serum Ferritin (SF) vs LIC at Baseline and EOS (Week 260 + 30 Days Follow-up)

Correlation between serum ferritin and LIC is assessed using scatter plots with pearson correlation coefficient and simple linear model. (NCT01709838)
Timeframe: Baseline, End of Study (EOS): Week 260 + 30 days follow up

Interventioncorrelation coefficient (Number)
At BaselineChange from Baseline at EOS
Deferasirox0.7300.531

[back to top]

Change in Serum Ferritin Values

"Change from baseline was be summarized descriptively for all on-treatment study visits.~Changes to the planned statistical analysis were related to significant withdrawal of patients from the Per-Protocol Analysis Set due to a large number of patients who discontinued the study (lack of assessments of iron exchange parameters at visits) and deviations from the Protocol affecting the assessment of efficacy parameters. Because of that, the additional efficacy analysis in the Per-Protocol Analysis Set was not performed." (NCT01818726)
Timeframe: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52

,
Interventionng/ml (Mean)
ScreeningWeek 0Week 4Week 8Week 12Week 16Week 20Week 24Week 28Week 32Week 36Week 40Week 44Week 48Week 52
Serum Ferritin Level < 1,000 μg/l769.02727.68640.75623.14653.62647.58646.80680.08526.17687.37699.13717.33661.13709.50655.07
Serum Ferritin Level ≥ 1,000 μg/l2866.142895.622089.622498.582262.122164.012054.231710.671446.071626.281709.071897.051301.801740.001288.80

[back to top]

Change in Serum Total Iron-binding Capacity (TIBC)

Mean change from baseline in serum total iron-binding capacity was summarized descriptively for all on-treatment study visits. (NCT01818726)
Timeframe: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52

,
Interventionμmol/l (Mean)
ScreeningWeek 0Week 4Week 8Week 12Week 16Week 20Week 24Week 28Week 32Week 36Week 40Week 44Week 48Week 52
Serum Ferritin Level < 1,000 μg/l66.6849.1049.0047.4447.2049.5348.6346.5345.9748.2347.5346.3044.1045.7046.77
Serum Ferritin Level ≥ 1,000 μg/l4.5149.9878.7073.9083.5685.3174.3481.90101.9072.8576.5683.5071.5674.3067.10

[back to top]

Change in Transferrin Saturation With Iron (TSI) Values

Mean percentage change from baseline in transferrin saturation with iron was summarized descriptively for all on-treatment study visits. (NCT01818726)
Timeframe: Screening, Week (Wk) 4, Wk 8, Wk 12, Wk 16, Wk 20, Wk 24, Wk 28, Wk 32, Wk 36, Wk 40, Wk 44, Wk 48, Wk 52

,
InterventionPercentage of saturation (Mean)
ScreeningWeek 0Week 4Week 8Week 12Week 16Week 20Week 24Week 28Week 32Week 36Week 40Week 44Week 48Week 52
Serum Ferritin Level < 1,000 μg/l53.7054.8850.1049.5449.0452.7850.655.9052.6749.9047.8343.0733.2744.2043.57
Serum Ferritin Level ≥ 1,000 μg/l88.8087.1973.3772.6872.4368.6068.1474.3065.0575.7565.9466.0569.2271.0783.40

[back to top]

Liver MRI

Change in liver iron concentration (measured using liver MRI), assessed as difference between value at 12 months minus value at baseline. (NCT01825512)
Timeframe: at baseline and after 12 months

Interventionmg/g (Mean)
Deferiprone-0.848
Deferasirox-2.975

[back to top]

Cardiac MRI T2*

Change in cardiac iron concentration (measured using cardiac MRI T2*), assessed as difference between value at 12 months minus value at baseline. MRI T2* is a non-invasive method based on gradient echo (GRE) sequences, where T2* represents the spin-spin relaxation times, measured in milliseconds. The faster the curve decreases (ie, the smaller T2*), the greater amount of iron is in the tissue. Treatment success was assessed as follows: if baseline cardiac T2* was less than 20 ms, an increase of 10% or more after 1 year of treatment was defined as treatment success; if baseline cardiac T2* was more than 20 ms, any increase or a decrease of less than 10% after 1 year of treatment was defined as treatment success. (NCT01825512)
Timeframe: at baseline and after 12 months

Interventionmilliseconds (ms) (Mean)
Deferiprone0.488
Deferasirox1.121

[back to top]

Ferritin Level

Change in serum ferritin level, assessed as difference between value at 12 months minus value at baseline. (NCT01825512)
Timeframe: at baseline and after 12 months

Interventionng/mL (Mean)
Deferiprone-397.583
Deferasirox-398.184

[back to top]

Percentage of Successfully Chelated Patients

Percentage of successfully chelated patients is assessed by serum ferritin levels (in all patients) and cardiac MRI T2* (in patients above 10 years of age able to perform an MRI scan without sedation) (NCT01825512)
Timeframe: at baseline and after 12 months

InterventionParticipants (Count of Participants)
Deferiprone69
Deferasirox80

[back to top]

Absolute Change in Serum Ferritin up to 24 Weeks for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline (NCT01868477)
Timeframe: Baseline up 24 weeks

Interventionng/mL (Median)
Responders - Week 5Responders - Week 9Responders - Week 13Responders - Week 17Non-responders - Week 5Non-responders - Week 9Non-responders - Week 13Non-responders - Week 17Non-responders - Week 21
EPO+DFX at 12-116-13659.574.5-68.3-148220.4-16.6-10.5

[back to top]

Absolute Change in Serum Ferritin up to 24 Weeks for Erythropoietin Alpha Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventionng/mL (Median)
Responders - Week 5Responders - Week 9Responders - Week 13Responders - Week 17Responders - Week 21Non-responders - Week 5Non-responders - Week 9Non-responders - Week 13
Erythropoietin Alpha-98.5-79.024.8-57.8-39.8-352-189-44.5

[back to top]

Summary of Hematologic Improvement in Patients Randomized to EPO+DFX and EPO Alone, Within 24 Weeks of Treatment (Full Analysis Set)

Percentage of participants achieving an hematologic improvement defined as: neutrophil improvement: increase from baseline >0.5 × 10^9/L (baseline = 1.0 × 10^9/L ), platelet improvement: increase from baseline ≥ 30 × 10^9/L (baseline = 100 × 10^9/L), hemoglobin improvement: Hb increase from baseline ≥ 1 g/dL (baseline<11 g/dL) (NCT01868477)
Timeframe: Baseline up to 24 weeks

,
Interventionpercentage of participants (Number)
Hematologic improvementNeutropil improvementPlatelet improvementHemoglobin improvement
Deferasirox + Erythropoietin Alpha45.580.080.060.0
Erythropoietin Alpha10066.750.066.7

[back to top]

Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set) (NCT01868477)
Timeframe: Baseline up to 24 weeks

InterventionThis analysis included patients randomized either (g/dL)
"Responders - Week 972023742"Responders - Week 1372023742"Responders - Week 1772023742"Non-responders - Week 972023742"Non-responders - Week 1772023742
"
EPO+DFX at 12 WeeksMedian

[back to top]

Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set) (NCT01868477)
Timeframe: Baseline up to 24 weeks

InterventionAbsolute change in serum ferritin from baseline (ng/mL)
"Responders - Week 1372023740"Non-responders - Week 972023740"Non-responders - Week 1372023740
"
Deferasirox + Erythropoietin AlphaMedian

[back to top]

Absolute Change From Baseline to Post-baseline Value for Hemoglobin(g/dL)(Full Analysis Set)

Hematological response criteria defined as: Erythroid response: hemoglobin (Hb) increase from baseline >= 1.5 g/dL (baseline < 11 g/dL), neutrophil response: increase from baseline >= 100% and increase > 0.5 × 10^9/L (baseline <1 × 10^9/L), platelet response: increase from baseline >= 30 × 10^9/L (baseline <100 × 10^9/L) according to modified IWG 2006 criteria (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventiong/dL (Mean)
Erythropoietin Alpha1.8
Deferasirox + Erythropoietin Alpha2.1

[back to top]

Absolute Change in Hemoglobin Values up to 24 Weeks

Absolute change in hemoglobin values for patients showing improvement: Hemoglobin improvement Hb increase from baseline ≥ 1 g/dL (baseline<11 g/dL) (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventiong/dL (Mean)
Erythropoietin Alpha1.3
Deferasirox + Erythropoietin Alpha1.4

[back to top]

Difference in Percentage of Patients Achieving Erythroid Response Within 12 Weeks, by Treatment Group (Full Analysis Set)

Difference in percentage of patients achieving an erythroid response within 12 weeks of treatment between the two arms according to modified IWG 2006 criteria increase in hemoglobin (Hb) ≥ 1.5 g/dL. Erythroid response is defined as the increase in Hb from baseline ≥ 1.5 g/dL. Patients achieving erythroid response at least once within 12 weeks were considered responders (NCT01868477)
Timeframe: Baseline up to 12 weeks

Interventionpercentage of participants (Number)
Erythropoietin Alpha41.7
Deferasirox + Erythropoietin Alpha27.3

[back to top]

Summary of Erythroid Response in Participants Randomized to EPO Alone at Baseline and Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

Erythroid response: hemoglobin increase from baseline > = 1.5 g/dL (baseline <11 g/dL) (NCT01868477)
Timeframe: Week 13 up to 24 weeks

Interventionparticipants (Number)
EPO+DFX (12 Weeks)0

[back to top]

Summary of Erythroid Response Within 24 Weeks in Participants Randomized to EPO at Baseline and Not Switched to EPO+DFX After 12 Weeks of Treatment (Full Analysis Set)

Erythroid response: hemoglobin increase from baseline > = 1.5 g/dL (baseline <11 g/dL). Percentages are based on N. Confidence intervals are calculated using Clopper-Pearson method. Hemoglobin value is at time of first response (NCT01868477)
Timeframe: baseline up to 24 weeks

Interventionpercentage of participants (Number)
EPO (24 Weeks)71.4

[back to top]

Absolute Change in Hemoglobin (Hb) From Baseline for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventiong/dL (Median)
Responders - Week 5Responders - Week 9Responders - Week 13Responders - Week 17Responders - Week 21Non-responders - Week 5Non-responders - Week 9Non-responders - Week 13Non-responders - Week 17Non-responders - Week 21
Deferasirox + Erythropoietin Alpha0.71.62.92.41.7-0.10.00.2-0.5-0.6

[back to top]

Absolute Change in Hemoglobin (Hb) From Baseline for EPO+DFX at 12 Weeks Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. The time-course of Hb and its absolute changes from baseline was summarized by descriptive statistics by visit and erythroid response. Patients randomized to EPO and not switching after 12 weeks to EPO+DFX would consist of only responders. (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventiong/dL (Median)
Responders - Week 5Non-responders - Week 5Non-responders - Week 13Non-responders - Week 21
EPO+DFX at 12 Weeks1.20.30.40.0

[back to top]

Absolute Change in Hemoglobin (Hb) From Baseline for Erythropoietin Alpha Arm (Full Analysis Set)

This analysis included patients randomized either to EPO or DFX+EPO at baseline as well as patients who did not have erythroid response at week 12 in the EPO group and switched to combination therapy. (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventiong/dL (Median)
Responders - Week 5Responders - Week 9Responders - Week 13Responders - Week 17Responders - Week 21Non-responders - Week 5Non-responders - Week 9Non-responders - Week 13
Erythropoietin Alpha1.51.91.71.60.8-0.9-1.7-2.5

[back to top]

Absolute Change in Platelets and Neutrophil Levels up to 24 Weeks

Absolute change in platelets and neutrophil levels for participants showing improvement: neutrophil improvement: increase from baseline >0.5 × 10^9/L (baseline = 1.0 × 10^9/L ), platelet improvement: increase from baseline ≥ 30 × 10^9/L (baseline = 100 × 10^9/L) (NCT01868477)
Timeframe: Baseline up to 24 weeks

,
Intervention10^9 cells/L (Mean)
PlateletsNeutrophils
Deferasirox + Erythropoietin Alpha66.32.4
Erythropoietin Alpha58.71.2

[back to top]

Absolute Change in Serum Ferritin up to 24 Weeks for Deferasirox + Erythropoietin Alpha Arm (Full Analysis Set)

Absolute change in serum ferritin from baseline (NCT01868477)
Timeframe: Baseline up to 24 weeks

Interventionng/mL (Median)
Responders - Week 5Responders - Week 9Responders - Week 17Responders - Week 21Non-responders - Week 5Non-responders - Week 17Non-responders - Week 21
Deferasirox + Erythropoietin Alpha-82.5-13916.5-95.5-38.0-123-291

[back to top]

Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary

The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point. (NCT02125877)
Timeframe: weeks -1, 4, 8, 12, 16, 20, 24

,
Interventionscore on a scale (Mean)
week -1 (n=69,65)week 4 (n=60,64)week 8 (n=59,51)week 12 (n=51,45)week 16 (n=48,41)week 20 (n40,39)week 24 (n32,26)
Deferasirox Dispersible Tablet (DFX-DT)1.41.81.41.71.91.51.5
Deferasirox Film-coated Tablet (DFX-FCT)1.91.11.11.00.90.91.2

[back to top]

Weekly Dose Violation Rate

The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100. (NCT02125877)
Timeframe: weeks 1, 4, 8, 12, 16, 20, 24

,
Interventionpercent dose violation (Mean)
week 1 (n=56,53)week 4 (n=58,58)week 8 (n=56,46)week 12 (n=50,41)week 16 (n=48,40)week 20 (n=40,36)week 24 (n=30,24)
Deferasirox Dispersible Tablet (DFX-DT)17.715.818.015.713.522.617.1
Deferasirox Film-coated Tablet (DFX-FCT)15.86.78.410.710.011.310.1

[back to top]

Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)

Blood samples were collected to assess Tmax. (NCT02125877)
Timeframe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose

,
Interventionhour (Median)
week 1 (n=14,15)week 3 (n=14,15)
Deferasirox Dispersible Tablet (DFX-DT)3.572.85
Deferasirox Film-coated Tablet (DFX-FCT)2.002.02

[back to top]

Palatability Questionnaire Score

"The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of no aftertaste. The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point." (NCT02125877)
Timeframe: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)

,
Interventionscore on a scale (Mean)
week 2 (n=69,70)week 3 (n=57,51)week 13 (n=59,62)week 24 (n=63,60)
Deferasirox Dispersible Tablet (DFX-DT)9.08.89.38.8
Deferasirox Film-coated Tablet (DFX-FCT)10.810.810.810.9

[back to top]

Overall Safety as Measured by Frequency of Adverse Events

The percentage of participants with adverse events, serious adverse events and deaths was assessed. (NCT02125877)
Timeframe: 28 weeks

,
InterventionPercentage of participants (Number)
Adverse eventsSAEsDeaths
Deferasirox Dispersible Tablet (DFX-DT)89.515.10
Deferasirox Film-coated Tablet (DFX-FCT)89.718.41.1

[back to top]

Overall Safety as Measured by Changes in Laboratory Values From Baseline

The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary. (NCT02125877)
Timeframe: baseline (BL), 30 weeks

,
InterventionPercentage of participants (Number)
platelet count, notable range: <100 x 10^9/Lplatelet count, extended range: <50 x 10^9/Labsolute neutrophils, notable range: <1.5 x 10^9/Labsolute neut., extended range: <0.5 x 10^9/Lserum creat, 2 cons >33% incr. from BL and >ULNcreat clearance, notable range: 2 cons <60mL/mincreat clearance, extended range: 2 cons <40mL/minurin protein/urin creat ratio, 2 cons >1.0 mg/mgALT, notable range: >5 x ULN and >2 x BLALT, extended range: >10 x ULN and >2 x BLAST, notable range: >5 x ULN and >2 x BLAST, extended range: >10 x ULN and >2 x BL
Deferasirox Dispersible Tablet (DFX-DT)9.33.58.14.74.77.02.32.31.21.201.2
Deferasirox Film-coated Tablet (DFX-FCT)8.05.713.803.42.32.301.101.10

[back to top]

Observed Maximum Plasma Concentration Following Drug Administration (Cmax)

Blood samples were collected to assess Cmax. (NCT02125877)
Timeframe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose

,
Interventionumol/L (Mean)
week 1 (n=14,15)week 3 (n=14,15)
Deferasirox Dispersible Tablet (DFX-DT)74.6118
Deferasirox Film-coated Tablet (DFX-FCT)79.3139

[back to top]

Number of Participants With Weekly Average Compliance of Medication Consumption

A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses. (NCT02125877)
Timeframe: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24

,
InterventionParticipants (Number)
week 1week 2week 3week 4week 5week 6week 7week 8week 9week 10week 11week 12week 13week 14week 15week 16week 17week 18week 19week 20week 21week 22week 23week 24
Deferasirox Dispersible Tablet (DFX-DT)566462585662555653525050495148484343404039383630
Deferasirox Film-coated Tablet (DFX-FCT)536456585851484645464241474242403938373636343324

[back to top]

Frequency of Selected Gastro-intestinal (GI) Adverse Events

The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed. (NCT02125877)
Timeframe: 28 weeks

,
InterventionPercentage of participants (Number)
Any GI adverse eventAbdominal painConstipationDiarrheaNauseaVomiting
Deferasirox Dispersible Tablet (DFX-DT)61.626.715.134.926.722.1
Deferasirox Film-coated Tablet (DFX-FCT)58.626.48.033.327.617.2

[back to top]

Dererasirox Plasma Concentration

Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT. (NCT02125877)
Timeframe: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose

,
Interventionumol/L (Mean)
week 3, pre-dose (n=63,70)week 3, 2 hours post-dose (n=67,76)week 13, pre-dose (n=69.56)week 13, 2 hours post-dose (n=74,59)week 21, pre-dose (n=54,59)week 21, 2 hours post-dose (n=59,64)
Deferasirox Dispersible Tablet (DFX-DT)39.680.837.178.746.689.8
Deferasirox Film-coated Tablet (DFX-FCT)27.395.531.392.543.1105

[back to top]

Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)

Blood samples were collected to assess AUClast. (NCT02125877)
Timeframe: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose

,
Interventionumol/L*h (Mean)
week1 (n=14,15)week 3 (n=13,15)
Deferasirox Dispersible Tablet (DFX-DT)11101590
Deferasirox Film-coated Tablet (DFX-FCT)10402110

[back to top]

Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)

The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point. (NCT02125877)
Timeframe: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)

,
Interventionscore on a scale (Mean)
week 2, adherence (n=70,70)week 2, satisfaction/preference (n=70,70)week 2, concerns (n=70,70)week 3, adherence (n=58,51)week 3, satisfaction/preference (n=58,51)week 3, concerns (n=58,51)week 13, adherence (n=59,64)week 13, satisfaction/preference (n=59,64)week 13, concerns (n=59,64)week 24, adherence (n=63,60)week 24, satisfaction/preference (n=63,60)week 24, concerns (n=63,60)
Deferasirox Dispersible Tablet (DFX-DT)10.35.212.910.95.412.411.25.412.712.55.811.8
Deferasirox Film-coated Tablet (DFX-FCT)7.62.813.87.72.614.07.82.913.67.52.913.7

[back to top]

Change From Baseline Serum Creatinine (Umol/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
Interventionumol/L (Mean)
BaselinePostChange
Baseline55.1NANA
Month 1257.463.56.1
Month 654.462.07.6

[back to top]

Percentage Relative Change From Baseline of Serum Ferritin (%) at Month 6 and 12

The percentage relative change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Percentage relative change = 100 × ([Post - Baseline] / Baseline). Percentage relative change is calculated for each patient individually and then overall descriptive summary statistics is obtained for subjects with a value at baseline and the particular time point. A negative percentage relative change from baseline is regarded as an improvement in this study (NCT02720536)
Timeframe: Baseline, 6 and 12 months

Interventionpercentage of relative change (Mean)
BaselineNA
Month 6-18.61
Month 12-29.08

[back to top]

Overview of Number of Participants With Adverse Events

Numbers represent counts of participants within the categories. An adverse event (AE) was defined as treatment emergent if its onset date is on or after (≥) the first administration of study treatment within this study or events present prior to start of study treatment but increased in severity on or after (≥) the first administration of study treatment within this study but not later than 30 days after the last study treatment in this study (NCT02720536)
Timeframe: Baseline up to approximately 25 months

Interventionnumber of participants (Number)
Adverse events (AEs)Treatment related AEsSevere adverse eventsTreatment related severe adverse eventsSerious adverse events (SAEs)Treatment related SAEsFatal SAEsTreatment related fatal SAEsAEs leading to discontinuationTreatment related AEs leading to discontinuationAEs leading to dose adjust/interruptionAEs requiring additional therapy
Deferasirox52201421301042334

[back to top]

Change From Baseline Platelets (10^9 Cells/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
Intervention10^9 cells/L (Mean)
BaselinePostChange
Baseline330.1NANA
Month 12339.4361.922.4
Month 6336.5344.27.7

[back to top]

Change From Baseline Red Blood Cells (RBC) (10^12 Cells/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
Intervention10^12 cells/L (Mean)
BaselinePostChange
Baseline3.753NANA
Month 123.7463.607-0.139
Month 63.7333.610-0.124

[back to top]

Change From Baseline of Serum Ferritin Level (ug/L) at Month 6 and 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline. A negative change from baseline is regarded as an improvement in this study (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
Interventionug/l (Mean)
BaselinePostChange
Baseline2523.51NANA
Month 122542.761924.49-618.26
Month 62614.122228.94-385.18

[back to top]

Change From Baseline Creatinine Clearance (mL/Min) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
InterventionmL/min (Mean)
BaselinePostChange
Baseline131.9NANA
Month 12129.6119.8-9.8
Month 6131.8116.0-15.8

[back to top]

Change From Baseline Aspartate Aminotransferase/Serum Glutamic Oxaloacetic Transaminase (AST/SGOT) (U/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
InterventionU/L (Mean)
BaselinePostChange
Baseline30.7NANA
Month 1233.725.2-8.5
Month 632.927.6-5.3

[back to top]

Change From Baseline Alanine Aminotransferase/Serum Glutamic Pyruvic Transaminase (ALT/SGPT) (U/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
InterventionU/L (Mean)
BaselinePostChange
Baseline37.4NANA
Month 1239.525.9-13.6
Month 639.428.9-10.5

[back to top]

Change From Baseline White Blood Cells (WBC) (10^9 Cells/L) at Month 6 and Month 12

The change from baseline at each time point is calculated only for subjects with a value at baseline and the particular time point. Post = Post baseline, Change = Post - Baseline (NCT02720536)
Timeframe: Baseline, 6 and 12 months

,,
Intervention10^9 cells/L (Mean)
BaselinePostChange
Baseline9.336NANA
Month 129.1009.007-0.093
Month 69.5599.090-0.469

[back to top]

Proportion of Patients That Achieve Erythroid Hematologic Improvement.

"As defined by the modified International Working Group (IWG) response criteria:~Erythroid response (pretreatment, <11 g/dL):~Hgb increase by ≥ 1.5 g/dL~Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation.~Platelet response (pretreatment, < 100 x 10^9/L)~Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L platelets~Increase from < 20 x 10^9/L to > 20 x 10^9/L and by at least 100%~Neutrophil response (pretreatment, < 1.0 x 10^9/L)~1) At least 100% increase and an absolute increase > 0.5 x 10^9/L" (NCT02943668)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Treatment (Deferasirox)0

[back to top]

Proportion of Patients Who Achieve Granulocyte or Platelet Hematologic Improvement

"As defined by the modified International Working Group (IWG) response criteria:~Erythroid response (pretreatment, <11 g/dL):~Hgb increase by ≥ 1.5 g/dL~Relevant reduction of units of RBC transfusions by an absolute number of at least 4 RBC transfusions/8 wk compared with the pretreatment transfusion number in the previous 8 wk. Only RBC transfusions given for a Hgb of ≤ 0.9 g/dL pretreatment will count in the RBC transfusion response evaluation.~Platelet response (pretreatment, < 100 x 10^9/L)~Absolute increase of ≥ 30 x 10^9/L for patients starting with > 20 x 10^9/L platelets~Increase from < 20 x 10^9/L to > 20 x 10^9/L and by at least 100%~Neutrophil response (pretreatment, < 1.0 x 10^9/L)~1) At least 100% increase and an absolute increase > 0.5 x 10^9/L" (NCT02943668)
Timeframe: At 6 months

InterventionParticipants (Count of Participants)
Treatment (Deferasirox)1

[back to top]

Change in Red Blood Cell (RBC) Transfusion Requirements

Assessed monthly for up to twelve months. (NCT02943668)
Timeframe: Baseline up to 12 months

,
InterventionRBC transfusions/month (Number)
Transfusion requirement at BaselineTransfusion requirement at time off-treatment
Patient 142
Patient 212

[back to top]

Change in Serum Ferritin Levels

Assessed monthly for up to twelve months. (NCT02943668)
Timeframe: Baseline up to 12 months

,
Interventionng/mL (Number)
Baseline1 month2 months3 months4 months5 months6 months7 months8 months9 months10 months11 months12 months
Patient 1602566376010640522360725256974832405439333728NANA
Patient 2755778827583414652776880NANANANANA

[back to top]

Change From Baseline in Gastrointestinal (GI) Symptom Score Based on GI Questionnaire

"The GI symptom score was calculated from responses to 5 questions of the GI questionnaire, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom.~An observer GI symptom questionnaire was administered to those patients who were < 10 years old. The questionnaire was completed by the parents of the participants. All items and the scoring algorithm remained the same as for participants ≥ 10 years old." (NCT02993224)
Timeframe: Baseline (week -1 or, if missing, week -2), week 24, 28 and 48

InterventionScore on a scale (Mean)
Week 24 - participants ≥ 10 years treated with deferasirox DTWeek 24 - participants ≥ 10 years treated with deferasirox FCTWeek 28 - participants ≥ 10 years treated with deferasirox FCTWeek 48 -participants ≥ 10 years treated with deferasirox FCTWeek 24 - participants < 10 years treated with deferasirox DTWeek 24 - participants < 10 years treated with deferasirox FCTWeek 28 - participants < 10 years treated with deferasirox FCTWeek 48 - participants < 10 years treated with deferasirox FCT
Deferasirox DT Followed by Deferasirox FCT1.45.8-1.9-2.20.2-2.0-0.5-0.6

[back to top]

Change From Baseline in Preference Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire

"The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.~The preference/satisfaction domain score consisted of 2 preference/satisfaction items, measured using a 5-point response scale. The preference score was calculated by summing these 2 items, with scores ranging from 2 to 10. Higher scores indicated worse satisfaction.~For participants < 10 years old, an observer version (ObsRO) was administered. Preference score remained the same as for participants ≥ 10 years old." (NCT02993224)
Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

InterventionScore on a scale (Mean)
Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)
Deferasirox DT Followed by Deferasirox FCT0.50.5-1.1-0.90.00.0-0.8-0.9

[back to top]

Change From Baseline in Serum Ferritin Levels

Absolute change from baseline over time in serum ferritin levels (NCT02993224)
Timeframe: From Baseline (Day 1) up to 96 weeks

Interventionmicrogram/liter (ug/L) (Mean)
Week 2 - participants treated with deferasirox DTWeek 4 - participants treated with deferasirox DTWeek 8 - participants treated with deferasirox DTWeek 12 - participants treated with deferasirox DTWeek 16 - participants treated with deferasirox DTWeek 20 - participants treated with deferasirox DTWeek 24 - participants treated with deferasirox DTWeek 28 - participants treated with deferasirox FCTWeek 32 - participants treated with deferasirox FCTWeek 36 - participants treated with deferasirox FCTWeek 40 - participants treated with deferasirox FCTWeek 44 - participants treated with deferasirox FCTWeek 48 - participants treated with deferasirox FCTWeek 52 - participants treated with deferasirox FCTWeek 56 - participants treated with deferasirox FCTWeek 60 - participants treated with deferasirox FCTWeek 64 - participants treated with deferasirox FCTWeek 68 - participants treated with deferasirox FCTWeek 72 - participants treated with deferasirox FCTWeek 76 - participants treated with deferasirox FCTWeek 80 - participants treated with deferasirox FCTWeek 84 - participants treated with deferasirox FCTWeek 88 - participants treated with deferasirox FCTWeek 92 - participants treated with deferasirox FCTWeek 96 - participants treated with deferasirox FCT
Deferasirox DT Followed by Deferasirox FCT363.654393.126462.560510.455519.038540.618644.849679.610623.600679.011739.016748.063833.700760.229903.622980.644863.579921.725971.112888.9171005.7581013.6831117.8491222.4291137.347

[back to top]

Change Over Time in Palatability Score of Palatability Questionnaire

"The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. Among the taste items, first one measured taste on a 5-point response scale. The other two items measured what happened after taking the medicine and how the perceived amount of liquid taken with the medicine was. Responses to these 3 items were combined and converted into a single palatability score using a scoring matrix: each combination of responses on each of 3 items corresponded to a predefined palatability score. E.g. if a participant responded bad to item 1, vomited <30min to item 2 and not enough to item 3, then the palatability score assigned was 0. This score ranged from 0 to 11; higher scores indicated better palatability. For participants <10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm were the same as for participants ≥10 years old. Change in palatability score over time was assessed" (NCT02993224)
Timeframe: Week 4, 24, 28 and 48

InterventionScore on a Scale (Mean)
From Week 4 to Week 24- participants treated with deferasirox DT at Week 24From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48
Deferasirox DT Followed by Deferasirox FCT-0.10.01.11.3

[back to top]

Change Over Time in Aftertaste Score of Palatability Questionnaire

"The palatability questionnaire consisted of 4 items, three items measuring taste and one item measuring aftertaste. The aftertaste item scored on a 5-point response scale with the response option: Very good = 1, Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5. This item offered an additional response option of no aftertaste. The aftertaste score was calculated among participants who had an aftertaste. Higher aftertaste scores indicated a worse aftertaste.~For participants less than (<) 10 years old, an observer palatability questionnaire was administered. Items and scoring algorithm remained the same as for participants greater than or equal to (≥) 10 years old.~Change in aftertaste score over time was assessed" (NCT02993224)
Timeframe: Week 4, 24, 28 and 48

InterventionScore on a Scale (Mean)
From Week 4 to Week 24 in participants treated with deferasirox DT at Week 24From Week 4 to Week 24 in participants treated with deferasirox FCT at Week 24From Week 24 to Week 28 in participants treated with deferasirox FCT at Week 28From Week 24 to Week 48 in participants treated with deferasirox FCT at Week 48
Deferasirox DT Followed by Deferasirox FCT-0.1-0.5-0.5-0.5

[back to top] [back to top]

Change From Baseline in Concerns Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire

"The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns.~The concerns domain score consisted of 3 items to address any concerns or worries with the medication. All 3 items were measured on a 5-point response scale. The concerns score was calculated by summing the 3 items, with scores ranging from 3 to 15. Higher scores indicated fewer concerns. For participants < 10 years old, an observer version (ObsRO) was administered. Concerns score remained the same as for participants ≥ 10 years old." (NCT02993224)
Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

InterventionScore on a scale (Mean)
Week 24 - participants ≥ 10 treated with deferasirox DT (PRO)Week 24 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 28 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 48 - participants ≥ 10 treated with deferasirox FCT (PRO)Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)
Deferasirox DT Followed by Deferasirox FCT-0.1-1.00.30.5-0.4-0.70.10.1

[back to top]

Change From Baseline in Adherence Domain Score of Modified Satisfaction With Iron Chelation (mSICT) Questionnaire

"The mSICT patient reported outcome (PRO) consisted of 15 items that represented 3 domains: Adherence, Preference, and Concerns. The adherence domain score consisted of 6 adherence items, measured using a 5-point response scale. The adherence score was calculated by summing these 6 items, with scores ranging from 6 to 30. Higher scores indicated worse adherence.~For participants <10 years old, an observer version (ObsRO) was administered. The adherence score remained the same as for participants ≥10 years old." (NCT02993224)
Timeframe: Baseline (week 2 or, if missing, week 3), week 24, 28 and 48

InterventionScore on a scale (Mean)
Week 24- participants ≥ 10 years treated with deferasirox DT (PRO)Week 24 - participants ≥ 10 years treated with deferasirox FCT (PRO)Week 28 - participants ≥ 10 years treated with deferasirox FCT (PRO)Week 48 - participants ≥ 10 years treated with deferasirox FCT (PRO)Week 24 - participants < 10 years treated with deferasirox DT (ObsRO)Week 24 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 28 - participants < 10 years treated with deferasirox FCT (ObsRO)Week 48 - participants < 10 years treated with deferasirox FCT (ObsRO)
Deferasirox DT Followed by Deferasirox FCT-0.3-1.00.50.5-0.2-0.70.81.1

[back to top]

Percentage of Consumed Tablet Counts During Deferasirox DT and Deferasirox FCT Treatment Periods

The percentage of consumed tablet counts (compliance) was calculated for each treatment period in the core phase: deferasirox DT (period 1) and deferasirox FCT (period 2). Compliance was defined as the total tablet count consumed divided by total tablet count prescribed and multiplied by 100. Total tablet count consumed was calculated as total number of tablets dispensed minus total number of tablets lost/wasted or returned. Total tablet count prescribed was calculated as the number of tablets that the patient should have taken during this period. If a patient did not return the study drug, the compliance was not calculated. (NCT02993224)
Timeframe: Deferasirox DT: From Baseline up to Week 24. Deferasirox FCT: From Week 25 up to Week 48

InterventionPercentage of tablet counts (Mean)
Deferasirox DT (Baseline up to Week 24)Deferasirox FCT (From Week 25 up to Week 48)
Deferasirox DT Followed by Deferasirox FCT98.6895.07

[back to top]

Number of Participants Selecting Each Reason for Treatment Preference as Assessed by the Preference Questionnaire at Week 28 and Week 48

"The preference questionnaire was a 3 item questionnaire. The first item asked the patients (or parents of young patients from 2 to 9 years old) which medicine they were taking. The second item asked which of the medicines did the patient Like best. Finally, the third item asked the patient why he/she preferred the medicine they chose in the second item. The number of participants who selected each response option for item 3 was assessed. Participants could select multiple reasons for treatment preference at each timepoint." (NCT02993224)
Timeframe: Week 28 and Week 48

InterventionParticipants (Count of Participants)
Aftertaste (Week 28)Can correctly prepare the medicine (Week 28)Convenience (it's not a problem to take your medicine) (Week 28)Easier to remember to take the medicine (Week 28)Gain my personal time with family and friends (Week 28)No/ less pain on the injection site (Week 28)No/ less side effects (Week 28)Number of pills (Week 28)Number of times you have to take the medicine (Week 28)Other (Week 28)Taste (Week 28)Aftertaste (Week 48)Can correctly prepare the medicine (Week 48)Convenience (it's not a problem to take your medicine) (Week 48)Easier to remember to take the medicine (Week 48)Gain my personal time with family and friends (Week 48)No/ less pain on the injection site (Week 48)No/ less side effects (Week 48)Number of pills (Week 48)Number of times you have to take the medicine (Week 48)Other (Week 48)Taste (Week 48)
Deferasirox DT Followed by Deferasirox FCT9351095735315046425301748103613637484147051

[back to top]

Number of Participants With Worst Post-baseline Values in Selected Chemistry Parameters

Safety measured by the worst post-baseline severity grade observed in a patient calculated using the normal/low/high classifications based on local laboratory normal ranges, regardless of the baseline status. Baseline was defined as the last non-missing value on or prior to the first dose. The selected chemistry parameters were: Alanine aminotransferase (ALT), Alkaline phosphatase (ALP), Aspartate aminotransferase (AST), total bilirubin, direct bilirubin, serum creatinine and Urine protein creatinine ratio (UPCR) (Protein/Creatinine represented UPCR). Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Count of Participants)
Alanine Aminotransferase72542906Alkaline Phosphatase72542906Aspartate Aminotransferase72542906Bilirubin72542906Creatinine72542906Direct Bilirubin72542906Protein/Creatinine72542906
NormalLowHighHigh & LowMissing
Deferasirox16
Deferasirox28
Deferasirox25
Deferasirox2
Deferasirox14
Deferasirox30
Deferasirox29
Deferasirox3
Deferasirox20
Deferasirox1
Deferasirox17
Deferasirox6
Deferasirox0

[back to top]

Number of Participants With GI Bowel Movements Item Scoring in Participants Pre-treated With Deferasirox

"The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 (None), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = 5 - 10 and 6 = 11 or more. The GI bowel movements item score was presented descriptively using frequency counts." (NCT03372083)
Timeframe: Baseline, Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

InterventionParticipants (Count of Participants)
Baseline (BL)72542906Week 272542906Week 372542906Week 472542906Week 872542906Week 1272542906Week 1672542906Week 2072542906EOT (Week 24)72542906
345 to 1011 or more
Deferasirox30
Deferasirox26
Deferasirox0
Deferasirox28
Deferasirox1
Deferasirox29
Deferasirox2
Deferasirox25

[back to top]

Number of Participants With GI Bowel Movements Item Scoring in Chelation Naive Participants

"The GI symptom questionnaire consisted of 6 items, 5 of which were scored using a 1-5 rating scale. The sixth item assessed bowel movement frequency during the past week, using 7 response options 0 = 0 (None), 1 = 1, 2 = 2, 3 = 3, 4 = 4, 5 = 5 - 10 and 6 = 11 or more. The GI bowel movements item score was presented descriptively using frequency counts." (NCT03372083)
Timeframe: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

InterventionParticipants (Count of Participants)
Week 272542906Week 372542906Week 472542906Week 872542906Week 1272542906Week 1672542906Week 2072542906EOT (Week 24)72542906
11 or more345 to 10
Deferasirox8
Deferasirox3
Deferasirox1
Deferasirox5
Deferasirox7
Deferasirox2
Deferasirox0
Deferasirox6

[back to top]

Absolute Change From Baseline in Body Temperature (°C)

Absolute change from baseline over time in body temperature measurements was to be provided. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

InterventionCelsius degree (°C) (Mean)
Baseline (BL)Change from BL @ Week 2Change from BL @ Week 3Change from BL @ Week 4Change from BL @ Week 8Change from BL @ Week 12Change from BL @ Week 16Change from BL @ Week 20Change from BL @ EOT (Week 24)
Deferasirox36.43-0.03-0.100.000.02-0.030.100.030.08

[back to top]

Number of Participants With Notable Changes in ECG Values From Baseline

Safety measured by the notable post-baseline changes in ECG values (PR, QRS, QT, QTcF and HR intervals) compared to baseline. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Number)
Deferasirox0

[back to top]

Number of Participants With Clinically Significant Ocular Assessments Changes From Baseline

Safety measured by notable post-baseline changes in Ocular assessments (Distance visual acuity test, Applanation tonometry, lens photography, wide angle fundus photography of the retina and optic nerve). Ocular assessment were required at screening and end of Treatment; during treatment, they were to be performed at the discretion of the investigator based on patient reporting symptoms. Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Number)
Deferasirox0

[back to top]

Number of Participants With Clinically Significant Auditory Assessments Changes From Baseline

Safety measured by notable post-baseline changes in Auditory assessments (comprehensive audiometry threshold examination and speech recognition). Baseline was defined as the last non-missing value on or prior to the first dose. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Number)
Deferasirox0

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Concerns

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Child's perspective overall score-Week 4Child's perspective overall score-Week 12Child's perspective overall score-EOTCaregiver's perspective overall score-Week 4Caregiver's perspective overall score-Week 12Caregiver's perspective overall score-EOT
Deferasirox9.79.48.93.73.93.4

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Type of Medicine Child Like Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
Wk 472542906Wk 1272542906EOT72542906
Tablet (taken once a day)Tablet (taken 3 times a day)Sprinkle powder on foodInjectionI don't knowTablet to dissolve in liquidTablet crushed
Deferasirox1
Deferasirox0
Deferasirox5
Deferasirox3
Deferasirox4
Deferasirox2

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Adherence

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Child's perspective overall score-Week 4Child's perspective overall score-Week 12Child's perspective overall score-EOTCaregiver's perspective overall score-Week 4Caregiver's perspective overall score-Week 12Caregiver's perspective overall score-EOT
Deferasirox-2.4-2.6-1.9-1.4-1.4-1.0

[back to top]

GI Symptom Score in Participants Pre-treated With Deferasirox

The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT. (NCT03372083)
Timeframe: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

InterventionScore (Mean)
Week 2Week 3Week 4Week 8Week 12Week 16Week 20EOT (Week 24)
Deferasirox6.36.06.46.46.56.77.37.6

[back to top]

Palatability Score in Participants Pre-treated With Deferasirox

The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Week 4Week 12EOT (Week 24)
Deferasirox10.710.310.6

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Rank Based on Child's Preference Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency counts. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
Wk 4 -Tablet to dissolve in liquid72542906Wk 12-Tablet to dissolve in liquid72542906EOT-Tablet to dissolve in liquid72542906Wk 4-Tablet (taken once a day)72542906Wk 12-Tablet (taken once a day)72542906EOT-Tablet (taken once a day)72542906Wk 4-Tablet (taken 3 times a day)72542906Wk 12-Tablet (taken 3 times a day)72542906EOT-Tablet (taken 3 times a day)72542906Wk 4-Tablet crushed72542906Wk 12-Tablet crushed72542906EOT-Tablet crushed72542906Wk 4-Sprinkle powder on food72542906Wk 12-Sprinkle powder on food72542906EOT-Sprinkle powder on food72542906Wk 4-Injection72542906Wk 12-Injection72542906EOT-Injection72542906
345612
Deferasirox6
Deferasirox5
Deferasirox0
Deferasirox3
Deferasirox4
Deferasirox2
Deferasirox1
Deferasirox8
Deferasirox9

[back to top]

GI Symptom Score in Chelation Naive Participants

The GI symptom score was calculated from responses to 5 questions, each with a possible score of 1 to 5, for an overall possible score range of 5 to 25, where a lower score represents a less severe GI symptom and a higher score represents a more severe GI symptom. GI symptom scores were summarized using descriptive statistics at week 2, week 3, week 4, week 8, week 12, week 16, week 20 and EOT. (NCT03372083)
Timeframe: Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

InterventionScore (Mean)
Week 2Week 3Week 4Week 8Week 12Week 16Week 20EOT (Week 24)
Deferasirox6.87.17.06.86.27.05.96.8

[back to top]

Adverse Events Profile

Analysis of frequencies for treatment emergent Adverse Event (TEAEs), Serious Adverse Event TEAEs and Deaths due to AEs, through the monitoring of relevant clinical and laboratory safety parameters. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Count of Participants)
On treatment-related AEOn treatment related SAEOn treatment Deaths
Deferasirox2200

[back to top]

Absolute Change From Baseline in Systolic and Diastolic Blood Pressures (mmHg)

Absolute change from baseline over time in systolic and diastolic blood pressures measurements were to be provided. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Interventionmillimetre of mercury (mmHg) (Mean)
Systolic BP-Baseline (BL)Systolic BP-Change from BL @ Week 2Systolic BP-Change from BL @ Week 3Systolic BP-Change from BL @ Week 4Systolic BP-Change from BL @ Week 8Systolic BP-Change from BL @ Week 12Systolic BP-Change from BL @ Week 16Systolic BP-Change from BL @ Week 20Systolic BP-Change from BL @ EOT (Week 24)Diastolic BP-Baseline (BL)Diastolic BP-Change from BL @ Week 2Diastolic BP-Change from BL @ Week 3Diastolic BP-Change from BL @ Week 4Diastolic BP-Change from BL @ Week 8Diastolic BP-Change from BL @ Week 12Diastolic BP-Change from BL @ Week 16Diastolic BP-Change from BL @ Week 20Diastolic BP-Change from BL @ EOT (Week 24)
Deferasirox100.0-0.1-2.3-0.30.3-1.4-1.9-0.6-2.660.00.7-2.11.0-0.61.7-0.5-0.1-0.4

[back to top]

Absolute Change From Baseline in Serum Ferritin (SF)

Absolute change from baseline over time in SF values up to 24 weeks of treatment were to be provided. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (BL), Week 4, Week 8, Week 12, Week 16, EOT (Week 24)

Interventionug/L (Mean)
Baseline (BL)Change from BL @ Week 4Change from BL @ Week 8Change from BL @ Week 12Change from BL @ Week 16Change from BL @ Week 20Change from BL @ EOT (Week 24)
Deferasirox2152.7-118.4-17.5-52.871.9-64.3-140.7

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
Wk 472542906Wk 1272542906EOT72542906
TasteConvenienceNumber of pillsNo/Less side effectsCan correctly prepare the medicineEasier to remember to take the medicineNumber of times he/she has to take the medicineNo/Less pain on the injection siteGain personal time with their family and friendsOtherAftertaste
Deferasirox2
Deferasirox1
Deferasirox0

[back to top]

Absolute Change From Baseline in Body Weight (kg)

Absolute change from baseline over time in body weight measurements was to be provided. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (BL), Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Interventionkilogram (kg) (Mean)
Baseline (BL)Change from BL @ Week 4Change from BL @ Week 8Change from BL @ Week 12Change from BL @ Week 16Change from BL @ Week 20Change from BL @ EOT (Week 24)
Deferasirox15.260.080.190.310.250.430.44

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Participants Pre-treated With Deferasirox: Mean Change From Baseline in Concerns

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT concerns domain scale for child's response had a possible range from 2 to 10, based on two questions and the mSICT concerns domain scale for caregiver's responses had the possible range of 1 to 5 based on one question. A higher score indicated fewer concerns. Only descriptive analysis performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Child's perspective overall score-Week 4Child's perspective overall score-Week 12Child's perspective overall score-EOTCaregiver's perspective overall score-Week 4Caregiver's perspective overall score-Week 12Caregiver's perspective overall score-EOT
Deferasirox1.11.20.80.40.40.2

[back to top]

Modified Satisfaction With Iron Chelation Therapy (Modified SICT) in Chelation Naive Participants: Mean Change From Baseline in Adherence

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT adherence domain consisted of 6 items from the child's perspective and 6 items from the caregiver's perspective, each with a possible score of 1 to 5, for an overall possible score range of 6 to 30. A higher score indicates poorer adherence. Only descriptive analysis performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Child's perspective overall score-Week 4Child's perspective overall score-Week 12Child's perspective overall score-EOTCaregiver's perspective overall score-Week 4Caregiver's perspective overall score-Week 12Caregiver's perspective overall score-EOT
Deferasirox10.49.810.39.09.910.6

[back to top]

Number of Chelation Naive Participants With Palatability After Taste Item Scoring

The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
Wk 472542906Wk 1272542906EOT72542906
BadVery badVery GoodGoodNeither good nor bad
Deferasirox0
Deferasirox5
Deferasirox2
Deferasirox1
Deferasirox4
Deferasirox3

[back to top]

Number of Participants With Selected Gastrointestinal Disorders up to 24 Weeks

To assess the safety of crushed deferasirox FCT with respect to selected gastrointestinal (GI) disorders (esophagitis, stomatitis, mouth ulceration, gastric ulcers, haemorrhage, abdominal pain, diarrhea, nausea, and vomiting). Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (Week 1 Day 1) up to Week 24, plus 30 day safety follow-up.

InterventionParticipants (Count of Participants)
OesophagitisBarrett's esophagitisStomatitisMouth ulcerationGastric ulcerGastrointestinal haemorrhageAbdominal painDiarrhoeaNauseaVomiting
Deferasirox0000002402

[back to top]

Palatability Score in Chelation Naive Participants

The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated separately. Among the taste items, first one measured taste on a five point response scale. The last two items measured what happened after taking the medicine, i.e., swallowed or vomited etc. and how the perceived amount of liquid taken with the medicine was, enough, not enough or too much. The palatability summary score was calculated from these three items using a scoring matrix and the score ranges from 0 to 11. A higher score indicates better palatability. Only descriptive analysis was performed. (NCT03372083)
Timeframe: Week 4, Week 12, EOT (Week 24)

InterventionScore (Mean)
Week 4Week 12EOT (Week 24)
Deferasirox10.410.910.8

[back to top]

Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Rank Based on Child's Preference Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the rank of the medicine (tablet to dissolve in liquid, tablet taken once a day, tablet taken 3 times a day, tablet crushed, sprinkle powder on food and injection), with a range of 1 to 6 (1 being most preferred and 6 being least preferred), based on what the child prefers. These items were presented descriptively using frequency count. (NCT03372083)
Timeframe: Baseline (BL), Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
BL -Tablet to dissolve in liquid72542906Week 4 -Tablet to dissolve in liquid72542906Week 12-Tablet to dissolve in liquid72542906EOT-Tablet to dissolve in liquid72542906BL-Tablet (taken once a day)72542906Week 4-Tablet (taken once a day)72542906Week 12-Tablet (taken once a day)72542906EOT-Tablet (taken once a day)72542906BL-Tablet (taken 3 times a day)72542906Week 4-Tablet (taken 3 times a day)72542906Week 12-Tablet (taken 3 times a day)72542906EOT-Tablet (taken 3 times a day)72542906BL-Tablet crushed72542906Week 4-Tablet crushed72542906Week 12-Tablet crushed72542906EOT-Tablet crushed72542906BL-Sprinkle powder on food72542906Week 4-Sprinkle powder on food72542906Week 12-Sprinkle powder on food72542906EOT-Sprinkle powder on food72542906BL-Injection72542906Week 4-Injection72542906Week 12-Injection72542906EOT-Injection72542906
162345
Deferasirox12
Deferasirox3
Deferasirox5
Deferasirox7
Deferasirox18
Deferasirox11
Deferasirox13
Deferasirox4
Deferasirox0
Deferasirox14
Deferasirox21
Deferasirox6
Deferasirox17
Deferasirox23
Deferasirox10
Deferasirox9
Deferasirox8
Deferasirox2
Deferasirox1
Deferasirox16
Deferasirox19
Deferasirox15
Deferasirox32
Deferasirox28
Deferasirox25
Deferasirox27

[back to top]

Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Reasons Child Preferred Crushed Medicine Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the reason child preferred crushed medicine (taste, aftertaste, convenience, number of pills, no/less side effects, can correctly prepare the medicine, easier to remember to take the medicine, number of times he/she has to take the medicine, no/less pain on the injection site, gain personal time with their family and friends, and other). These items were presented descriptively using frequency counts. (NCT03372083)
Timeframe: Baseline (BL), Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
BL72542906Wk 472542906Wk 1272542906EOT72542906
AftertasteConvenienceNo/Less side effectsGain personal time with their family and friendsOtherCan correctly prepare the medicineEasier to remember to take the medicineTasteNumber of pillsNumber of times he/she has to take the medicineNo/Less pain on the injection site
Deferasirox6
Deferasirox5
Deferasirox2
Deferasirox1
Deferasirox4
Deferasirox3
Deferasirox8
Deferasirox0
Deferasirox7

[back to top]

Modified SICT in Participants Pre-treated With Deferasirox: Number of Participants With Type of Medicine Child Like Scoring

The mSICT questionnaire was to be completed at screening visit 1, week 4, week 12 and EOT. The responses from screening visit 1 for mSICT questionnaire were to be considered as baseline. The modified SICT consisted of 20 items that represented 3 domains: Adherence, Preference and Concerns. The mSICT preference domain consisted of 3 items including the type of medicine the child said he/she liked best (tablet to dissolve in liquid, tablet (taken once a day), tablet (taken 3 times a day), tablet crushed, sprinkle powder on food, injection and I don't know). These items were presented descriptively using frequency counts. (NCT03372083)
Timeframe: Baseline (BL), Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
BL72542906Wk 472542906Wk 1272542906EOT72542906
Tablet to dissolve in liquidTablet (taken once a day)I don't knowTablet (taken 3 times a day)Tablet crushedSprinkle powder on foodInjection
Deferasirox11
Deferasirox14
Deferasirox3
Deferasirox1
Deferasirox13
Deferasirox12
Deferasirox4
Deferasirox9
Deferasirox15
Deferasirox0

[back to top]

Number of Participants Pre-treated With Deferasirox With Palatability After Taste Item Scoring

The palatability (taste and ability to consume medicine) questionnaire consisted of 4 items, three items measuring taste or ability to consume medicine and one item measuring aftertaste. The aftertaste item was treated as a separate item and scored on a 5-point response scale with the response format Very good = 1 (best), Good = 2, Neither good nor bad = 3, Bad = 4, Very bad = 5 (worst). Only descriptive analysis performed using frequency counts. (NCT03372083)
Timeframe: Baseline, Week 4, Week 12, EOT (Week 24)

InterventionParticipants (Count of Participants)
BL72542906Wk 472542906Wk 1272542906EOT72542906
GoodVery badVery GoodNeither good nor badBad
Deferasirox5
Deferasirox15
Deferasirox7
Deferasirox1
Deferasirox13
Deferasirox16
Deferasirox4
Deferasirox9
Deferasirox12
Deferasirox2
Deferasirox0
Deferasirox11
Deferasirox14
Deferasirox3

[back to top]

Absolute Change From Baseline in Pulse Rate (Bpm)

Absolute change from baseline over time in supine pulse rate was to be provided. Only descriptive analysis performed. (NCT03372083)
Timeframe: Baseline (BL), Week 2, Week 3, Week 4, Week 8, Week 12, Week 16, Week 20, EOT (Week 24)

Interventionbeats per minute (bpm) (Mean)
Baseline (BL)Change from BL @ Week 2Change from BL @ Week 3Change from BL @ Week 4Change from BL @ Week 8Change from BL @ Week 12Change from BL @ Week 16Change from BL @ Week 20Change from BL @ EOT (Week 24)
Deferasirox102.4-1.1-2.7-3.1-1.0-0.3-2.20.81.1

[back to top]