Deferasirox.

PURPOSE: The pharmacology, clinical efficacy, adverse effects and toxicities, and the economic issues that should be considered in using deferasirox are reviewed.
SUMMARY: Iron overload is a complication of the chronic blood transfusions used to treat several hematologic disorders. To date, management of transfusional iron overload has consisted of chelation therapy with parenteral deferoxamine. Although survival rates improve with adequate chelation, an estimated one third to one half of patients are not compliant with deferoxamine therapy, largely because of the discomfort and demanding nature of the regimen. In 2005, the Food and Drug Administration approved the labeling for deferasirox for the treatment of chronic overload due to transfusional hemosiderosis. Deferasirox is an oral tridentate chelator that mobilizes iron stores by binding selectively to the ferric form of iron. 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. The largest clinical study to date demonstrated the noninferiority of deferasirox 20 or 30 mg/kg/day compared with subcutaneous infusions of deferoxamine >/=35 mg/kg/day administered five days weekly in a subgroup of patients with higher hepatic iron burdens. Deferasirox has been well tolerated in clinical trials. Nearly 97% of participants in a comparative study stated that they preferred deferasirox over their previous deferoxamine treatment.
CONCLUSION: Deferasirox, a tridentate oral chelator approved for the treatment of chronic iron overload due to blood transfusions, offers a promising alternative for patients unwilling or unable to comply with deferoxamine therapy.