Abstract
Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. A comparative phase 3 trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with β-thalassemia aged 2 years or older. 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). The primary endpoint was maintenance or reduction of LIC; secondary endpoints included safety and tolerability, change in serum ferritin level, and net body iron balance. In both arms, patients with LIC values of 7 mg Fe/g dry weight (dw) or higher had significant and similar dose-dependent reductions in LIC and serum ferritin, and effects on net body iron balance. However, the primary endpoint was not met in the overall population, possibly due to the fact that proportionally lower doses of deferasirox relative to deferoxamine were administered to patients with LIC values less than 7 mg Fe/g dw. The most common adverse events included rash, gastrointestinal disturbances, and mild nonprogressive increases in serum creatinine. No agranulocytosis, arthropathy, or growth failure was associated with deferasirox administration. Deferasirox is a promising once-daily oral therapy for the treatment of transfusional iron overload.
| Original language | English |
|---|---|
| Pages (from-to) | 3455-3462 |
| Number of pages | 8 |
| Journal | Blood |
| Volume | 107 |
| Issue number | 9 |
| DOIs | |
| Publication status | Published - May 1 2006 |
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ASJC Scopus subject areas
- Hematology
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Aphase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia. / Cappellini, Maria Domenica; Cohen, Alan; Piga, Antonio; Bejaoui, Mohamed; Perrotta, Silverio; Agaoglu, Leyla; Aydinok, Yesim; Kattamis, Antonis; Kilinc, Yurdanur; Porter, John; Capra, Marcello; Galanello, Renzo; Fattoum, Slaheddine; Drelichman, Guillermo; Magnano, Carmelo; Verissimo, Monica; Athanassiou-Metaxa, Miranda; Giardina, Patricia; Kourakli-Symeonidis, Alexandra; Janka-Schaub, Gritta; Coates, Thomas; Vermylen, Christiane; Olivieri, Nancy; Thuret, Isabelle; Opitz, Herbert; Ressayre-Djaffer, Catherine; Marks, Peter; Alberti, Daniele.
In: Blood, Vol. 107, No. 9, 01.05.2006, p. 3455-3462.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Aphase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia
AU - Cappellini, Maria Domenica
AU - Cohen, Alan
AU - Piga, Antonio
AU - Bejaoui, Mohamed
AU - Perrotta, Silverio
AU - Agaoglu, Leyla
AU - Aydinok, Yesim
AU - Kattamis, Antonis
AU - Kilinc, Yurdanur
AU - Porter, John
AU - Capra, Marcello
AU - Galanello, Renzo
AU - Fattoum, Slaheddine
AU - Drelichman, Guillermo
AU - Magnano, Carmelo
AU - Verissimo, Monica
AU - Athanassiou-Metaxa, Miranda
AU - Giardina, Patricia
AU - Kourakli-Symeonidis, Alexandra
AU - Janka-Schaub, Gritta
AU - Coates, Thomas
AU - Vermylen, Christiane
AU - Olivieri, Nancy
AU - Thuret, Isabelle
AU - Opitz, Herbert
AU - Ressayre-Djaffer, Catherine
AU - Marks, Peter
AU - Alberti, Daniele
PY - 2006/5/1
Y1 - 2006/5/1
N2 - Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. A comparative phase 3 trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with β-thalassemia aged 2 years or older. 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). The primary endpoint was maintenance or reduction of LIC; secondary endpoints included safety and tolerability, change in serum ferritin level, and net body iron balance. In both arms, patients with LIC values of 7 mg Fe/g dry weight (dw) or higher had significant and similar dose-dependent reductions in LIC and serum ferritin, and effects on net body iron balance. However, the primary endpoint was not met in the overall population, possibly due to the fact that proportionally lower doses of deferasirox relative to deferoxamine were administered to patients with LIC values less than 7 mg Fe/g dw. The most common adverse events included rash, gastrointestinal disturbances, and mild nonprogressive increases in serum creatinine. No agranulocytosis, arthropathy, or growth failure was associated with deferasirox administration. Deferasirox is a promising once-daily oral therapy for the treatment of transfusional iron overload.
AB - Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. A comparative phase 3 trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with β-thalassemia aged 2 years or older. 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). The primary endpoint was maintenance or reduction of LIC; secondary endpoints included safety and tolerability, change in serum ferritin level, and net body iron balance. In both arms, patients with LIC values of 7 mg Fe/g dry weight (dw) or higher had significant and similar dose-dependent reductions in LIC and serum ferritin, and effects on net body iron balance. However, the primary endpoint was not met in the overall population, possibly due to the fact that proportionally lower doses of deferasirox relative to deferoxamine were administered to patients with LIC values less than 7 mg Fe/g dw. The most common adverse events included rash, gastrointestinal disturbances, and mild nonprogressive increases in serum creatinine. No agranulocytosis, arthropathy, or growth failure was associated with deferasirox administration. Deferasirox is a promising once-daily oral therapy for the treatment of transfusional iron overload.
UR - http://www.scopus.com/inward/record.url?scp=33646414765&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=33646414765&partnerID=8YFLogxK
U2 - 10.1182/blood-2005-08-3430
DO - 10.1182/blood-2005-08-3430
M3 - Article
C2 - 16352812
AN - SCOPUS:33646414765
VL - 107
SP - 3455
EP - 3462
JO - Blood
JF - Blood
SN - 0006-4971
IS - 9
ER -