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Pediatr Endocrinol Rev. 2008 Oct;6 Suppl 1:208-13.

Treatment of iron overload in thalassemia.

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Ospedale S. Eugenio, Thalassemia Unit, P. le dell'Umanesimo10, 00144 Rome, Italy.


Iron overload, characterized by excessive iron deposition, occurs commonly in patients with hereditary or refractory anemias such as beta-thalassemia major, sickle cell anemia, and myelodysplastic syndromes, whose anemia is managed with frequent blood transfusions. Without adequate iron chelation therapy, almost all patients with beta-thalassemia will accumulate potentially fatal iron levels. Myocardial siderosis and resulting cardiac complications are among the leading causes of death in such patients. Unfortunately, even with the administration of effective subcutaneous iron chelation therapy with desferrioxamine (DFO), over 50% of patients die before the age of 35 years, largely because of poor compliance with subcutaneous chelation regimens. Recently introduced oral chelation agents, deferiprone and deferasirox, are associated with higher compliance rates, and greater reductions in cardiac iron levels, than those achieved with DFO. This article reviews the pharmacologic properties and clinical efficacy of currently available iron chelation therapies in the management of transfusional chronic iron overload.

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