Efficacy of deferasirox in reducing and preventing cardiac iron overload in β-thalassemia

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 56% 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 inmyocardial 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 (±40.5%) to 12.9 ms (±49.5%) (+16%;P<.001). LVEF (mean ± SD) was unchanged: 67.4 (±5.7%) to 67.0 (±6.0%) (-0.3%;P=.53). In the prevention arm, baseline myocardial T2* was unchanged from baseline of 32.0 ms (±25.6%) to 32.5 ms (±25.1%) (+2%; P=.57) and LVEF increased from baseline 67.7 (±4.7%) to 69.6 (±4.5%) (+1.8%; P<.001). This prospective study shows that deferasirox is effective in removing and preventing myocardial iron accumulation.

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