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Hoffmann, R. A wiki for the life sciences where authorship matters. Nature Genetics (2008)

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 3(rd) 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.[1]


  1. Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major. Pennell, D.J., Porter, J.B., Cappellini, M.D., Chan, L.L., El-Beshlawy, A., Aydinok, Y., Ibrahim, H., Li, C.K., Viprakasit, V., Elalfy, M.S., Kattamis, A., Smith, G., Habr, D., Domokos, G., Roubert, B., Taher, A. Haematologica (2012) [Pubmed]
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