Aphase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia

Maria Domenica Cappellini, Alan Cohen, Antonio Piga, Mohamed Bejaoui, Silverio Perrotta, Leyla Agaoglu, Yesim Aydinok, Antonis Kattamis, Yurdanur Kilinc, John Porter, Marcello Capra, Renzo Galanello, Slaheddine Fattoum, Guillermo Drelichman, Carmelo Magnano, Monica Verissimo, Miranda Athanassiou-Metaxa, Patricia Giardina, Alexandra Kourakli-Symeonidis, Gritta Janka-SchaubThomas Coates, Christiane Vermylen, Nancy Olivieri, Isabelle Thuret, Herbert Opitz, Catherine Ressayre-Djaffer, Peter Marks, Daniele Alberti

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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 languageEnglish
Pages (from-to)3455-3462
Number of pages8
JournalBlood
Volume107
Issue number9
DOIs
Publication statusPublished - May 1 2006

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ASJC Scopus subject areas

  • Hematology

Cite this

Cappellini, M. D., Cohen, A., Piga, A., Bejaoui, M., Perrotta, S., Agaoglu, L., Aydinok, Y., Kattamis, A., Kilinc, Y., Porter, J., Capra, M., Galanello, R., Fattoum, S., Drelichman, G., Magnano, C., Verissimo, M., Athanassiou-Metaxa, M., Giardina, P., Kourakli-Symeonidis, A., ... Alberti, D. (2006). Aphase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with β-thalassemia. Blood, 107(9), 3455-3462. https://doi.org/10.1182/blood-2005-08-3430