Deferasirox for up to 3 years leads to continued improvement of myocardial T2☆ in patients with β-thalassemia major

Background Prospective data on cardiac iron removal are limited beyond one year and longer-term studies are, therefore, important. Design and Methods Seventy-one patients in the EPIC cardiac substudy elected to continue into the 3rd year, allowing cardiac iron removal to be analyzed over three years. Results Mean deferasirox dose during year 3 was 33.6 +/- 9.8 mg/kg per day. Myocardial T2*, assessed by cardiovascular magnetic resonance, significantly increased from 12.0 ms +/- 39.1% at baseline to 17.1 ms +/- 62.0% at end of study (P < 0.001), corresponding to a decrease in cardiac iron concentration (based on ad hoc analysis of T2*) from 2.43 +/- 1.2 mg Fe/g dry weight (dw) at baseline to 1.80 +/- 1.4 mg Fe/g dw at end of study (P < 0.001). After three years, 68.1% of patients with baseline T2* 10 to <20 ms normalized (>= 20 ms) and 50.0% of patients with baseline T2* >5 to <10 ms improved to 10 to <20 ms. There was no significant variation in left ventricular ejection fraction over the three years. No deaths occurred and the most common investigator-assessed drug-related adverse event in year 3 was increased serum creatinine (n=9, 12.7%). Conclusions Three years of deferasirox treatment along with a clinically manageable safety profile significantly reduced cardiac iron overload versus baseline and normalized T2* in 68.1% (32 of 47) of patients with T2* 10 to <20 ms.