Iron chelation therapy and thalassemia - an overview.

New developments in the epidemiology, treatment and prognosis of thalassemia have dramatically altered the approach to the care of affected patients. In the last 30 years, conventional treatment of β-thalassemia major, based primarily on regular blood transfusions and iron chelation therapy with desferrioxamine (DFO) has markedly improved the prognosis of the disease. Adequate administration of parenteral DFO reduces or prevents iron accumulation. The unavailability of DFO (dysferol) for most patients with thalassemia major and the failure of prescribed therapy to prevent complications in other patients have led to a search for alternative iron chelators; one of them, deferiprone (DFP) has been commercially available. Patients may ultimately benefit from having a choice between several chelators, including orally active drugs. Combination therapy and organ-targeted chelation, may soon have a considerable impact on the therapeutic outcome and quality of life of patients with thalassemia.