Comparison of effects of oral deferiprone and subcutaneous desferrioxamine on myocardial iron concentrations and ventricular function in beta-thalassaemia

Background Despite the introduction of the parenteral iron chelator desferrioxamine more than 30 years ago, 50% of patients with thalassaemia major die before the age of 35 years, predominantly from iron-induced heart failure. The only alternative treatment is oral deferiprone, but its longterm efficacy on myocardial iron concentrations is unknown. Methods We compared myocardial iron content and cardiac function in 15 patients receiving long-term deferiprone treatment with 30 matched thalassaemia major controls who were on long-term treatment with desferrioxamine. Myocardial iron concentrations were measured by a new magnetic-resonance T2* technique, which shows values inversely related to tissue iron concentration. Findings The deferiprone group had significantly less myocardial iron (median 34•0 ms vs 11•4 ms, p=0•02) and higher ejection fractions (mean 70% [SD 6•5] vs 63% [6•9], p=0•004) than the desferrioxamine controls. Excess myocardial iron (T2* <20 ms) was less common in the deferiprone group than in the desferrioxamine controls (four [27%] vs 20 [67%], p=0•025), as was severe (T2* <10 ms) iron overload (one [7%] vs 11 [37%], p=0•04). The odds ratio for excess myocardial iron in the desferrioxamine controls versus the deferiprone group was 5•5 (95% CI 1•2–28•8). Interpretation Conventional chelation treatment with subcutaneous desferrioxamine does not prevent excess cardiac iron deposition in two-thirds of patients with thalassaemia major, placing them at risk of heart failure and its complications. Oral deferiprone is more effective than desferrioxamine in removal of myocardial iron.