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Deferasirox: a novel, once-daily oral iron chelator

Maria Domenica Cappellini

Regular blood transfusions for the treatment of chronic anemia inevitably lead to iron overload since humans do not possess a physiological mechanism for removing excess iron. Without appropriate treatment, the cumulative effects of iron overload lead to severe organ damage and, ultimately, death. The iron chelating agent, deferoxamine, has been used effectively for over 40 years and has demonstrated the important morbidity and mortality benefits of chelation therapy. However, the demanding therapeutic regimen of frequent and slow subcutaneous infusions means that compliance can be poor, thereby compromising its effectiveness. Deferasirox (Exjade^®, ICL670) is a new once-daily, oral iron chelator that has recently received approval from the US FDA, European Commission and Swiss (Swissmedic) authorities. The efficacy and safety of deferasirox has been established in a comprehensive and rigorous set of clinical studies involving adult and pediatric patients as young as 2 years of age, and in patients with a wide range of transfusiondependent anemias. These data show the dose–dependent effects of deferasirox and have established that this new agent is as efficacious as the reference standard therapy (deferoxamine) when used at comparable therapeutic doses. The availability of this convenient, effective and well-tolerated therapy represents a significant advance in the management of transfusional hemosiderosis that allows the physician to tailor iron chelation therapy to individual patient needs.