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A main complication of β-thalassemia major is organ dysfunction due to iron overload. Initial studies of deferasirox, an oral iron chelator that became available a few years ago, suggested that it effectively reduced the iron burden and could substitute for parenteral deferoxamine (DFO).
Now, a multinational group of investigators report 5-year follow-up data from a prospective, randomized study comparing the safety and effectiveness of deferasirox versus DFO in patients with β-thalassemia (Blood 2006; 107:3455). After the first year, 153 children and 143 adult patients received deferasirox, and an additional 120 children and 139 adults crossed over from DFO to deferasirox. Most patients received deferasirox doses of either 15 to <25 mg/kg/d…