Abstract:
Cardiac iron overload causes most deaths in β-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with β-thalassemia in a 1-year prospective, multicenter study. The cardiac iron reduction arm (n = 114) included patients with magnetic resonance myocardial T2* from 5 to 20 ms (indicating cardiac siderosis), left ventricular ejection fraction (LVEF) of 56percent or more, serum ferritin more than 2500 ng-mL, liver iron concentration more than 10 mg Fe-g dry weight, and more than 50 transfused blood units. The prevention arm (n = 78) included otherwise eligible patients whose myocardial T2* was 20 ms or more. The primary end point was the change in myocardial T2* at 1 year. In the cardiac iron reduction arm, the mean deferasirox dose was 32.6 mg-kg per day. Myocardial T2* (geometric mean ± coefficient of variation) improved from a baseline of 11.2 ms (± 40.5percent) to 12.9 ms (± 49.5percent) (+16percent; P .001). LVEF (mean ± SD) was unchanged: 67.4 (± 5.7percent) to 67.0 (± 6.0percent) (-0.3percent; P = .53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (± 25.6percent) to 32.5 ms (± 25.1percent) (±2percent; P = .57) and LVEF increased from baseline 67.7 (± 4.7percent) to 69.6 (± 4.5percent) (+1.8percent; P .001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation. This study is registered at http:-- clinicaltrials.gov as NCT00171821. © 2010 by The American Society of Hematology.