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

Pennell, D.J. and Porter, J.B. and Cappellini, M.D. and El-Beshlawy, A. and Chan, L.L. and Aydinok, Y. and Elalfy, M.S. and Sutcharitchan, P. and Li, C.K. and Ibrahim, H. and Viprakasit, V. and Kattamis, A. and Smith, G. and Habr, D. and Domokos, G. and Roubert, B. and Taher, A. (2010) Efficacy of deferasirox in reducing and preventing cardiac iron overload in beta-thalassemia. Blood, 115 (12). pp. 2364-2371.

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Cardiac iron overload causes most deaths in beta-thalassemia major. The efficacy of deferasirox in reducing or preventing cardiac iron overload was assessed in 192 patients with beta-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 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 (+/- 40.5%) 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. This study is registered at http://clinicaltrials.gov as NCT00171821. (Blood. 2010; 115: 2364-2371)

Item Type: Article
Subjects: R Medicine
Depositing User: Dr Mohd Faizal Hamzah
Date Deposited: 01 Dec 2015 00:57
Last Modified: 01 Dec 2015 00:57
URI: http://eprints.um.edu.my/id/eprint/14976

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