ABSTRACT: Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. A comparative Phase III trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with beta-thalassemia >/=2 years of age. 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 >/=7 mg Fe/g dry weight (dw) 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 < 7 mg Fe/g dw. The most common adverse events included rash, gastrointestinal disturbances, and mild non-progressive 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
Blood. 01/2006; 107(9):3455-3462.