Deferasirox: An Alternative to Phlebotomy for Hemochromatosis-Induced Iron Overload?

Hereditary hemochromatosis (HH) is one of the most common genetic disorders and is associated with substantial long-term morbidity and mortality. The mainstay of treatment is intermittent therapeutic phlebotomy. However, not all patients tolerate phlebotomy, which is also contraindicated in severe heart disease and severe anemias.

To test the safety and efficacy of a potential alternative to phlebotomy in this setting, investigators conducted an industry-sponsored, phase I/II, dose-escalation trial of the oral iron-chelating agent deferasirox (Exjade). The researchers studied 49 patients with HH who were homozygous for the C282Y mutation, had no history of cirrhosis, and had ferritin levels between 300 and 2000 ng/mL. Patients received 5, 10…