Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.
- Citation:
- Pennell, D. J., J. B. Porter, M. D. Cappellini, L. L. Chan, A. El-Beshlawy, Y. Aydinok, H. Ibrahim, C. - K. Li, V. Viprakasit, M. S. Elalfy, et al., "Deferasirox for up to 3 years leads to continued improvement of myocardial T2* in patients with β-thalassemia major.", Haematologica, vol. 97, issue 6, pp. 842-8, 2012 Jun.
Abstract:
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.