Continued improvement in myocardial T2* over two years of deferasirox therapy in beta-thalassemia major patients with cardiac iron overload.

摘要

BACKGROUND: The efficacy of cardiac iron chelation in transfusion-dependent patients has been demonstrated in one-year prospective trials. Since normalization of cardiac T2* takes several years, the efficacy and safety of deferasirox was assessed for two years in patients with beta-thalassemia major in the cardiac sub-study of the EPIC trial. DESIGN AND METHODS: Eligible patients with myocardial T2* greater than 5 to less than 20 ms received deferasirox, with the primary endpoint being the change in T2* from baseline to two years. RESULTS: Baseline myocardial T2* was severe (> 5 to /= 20 ms) in 56.7 of patients. In those with severe cardiac iron overload at baseline, 42.9 improved to the moderate-to-mild group. The incidence of drug-related adverse events did not increase during the extension relative to the core study and included (>/= 5) increased serum creatinine, rash and increased alanine aminotransferase. CONCLUSIONS: Continuous treatment with deferasirox for two years with a target dose of 40 mg/kg/d continued to remove iron from the heart in patients with beta-thalassemia major and mild, moderate and severe cardiac siderosis. (Clinicaltrials.gov identifier: NCT 00171821).