Randomized controlled trial of deferiprone or deferoximine in beta-thalassaemia major patients with asymptomatic myocardial siderosis

Authors
Pennell DJ, Berdoukas V, Karagiorga M, Ladis V, Piga A, Aessopos A,
Gotsis ED, Tanner MA, Smith GC, Westwood MA, Wonke B, Galanello R.

Journal
Blood. 2006; 107:3738-3744.

Background
Cardiac disease is the cause of approximately 70 percent of deaths in beta thalassaemia major. Cardiovascular magnetic resonance (CMR) can measure cardiac iron deposition through the magnetic relaxation parameter T2*. This allows the determination of best chelation strategies for the heart by direct assessment of the clearance of myocardial iron.

Direct measures of myocardial iron using myocardial T2* are providing new insights into cardiac disease in thalassaemia. So far, oral chelation with deferiprone appears to be more effective than deferoxamine in removing cardiac iron.

Methods
This open-label randomized controlled trial of 61 patients previously on deferoxamine examined the change in myocardial T2* measured over 1 year for patients maintained on deferoxamine and those switched to deferiprone. The trial was conducted in 4 centres in Italy and Greece.

Summary
The improvement in cardiac T2* was significantly greater for those patients treated with deferiprone. Also, left ventricular ejection fraction (LVEF) improved significantly more in the deferiprone treated patients vs. deferoxamine patients. Overall, deferiprone monotherapy was superior to deferoxamine over 1 year in improving myocardial siderosis and in asymptomatic patients with beta-thalassemia major.

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