Iron-Chelation Therapy with Oral Deferiprone — Toxicity or Lack of Efficacy?

Abstract

Red-cell transfusion therapy in patients with homozygous β-thalassemia (thalassemia major) decreases the complications of severe anemia and prolongs survival.¹ However, the effectiveness of transfusions is limited by the tissue damage resulting from iron overload, a consequence of the body's limited capacity to excrete iron. Each 250 ml of transfused red cells adds about 250 mg of elemental iron to the body. The end-organ manifestations of iron overload in patients with β-thalassemia — cirrhosis, hepatocellular carcinoma, cardiac failure, diabetes mellitus, and hypopituitarism — are similar to those in patients with hereditary hemochromatosis, and they shorten life expectancy.¹ There is a direct . . .