Iron chelation therapy in sickle-cell disease and other transfusion-dependent anemias

Regular red cell transfusion therapy may be life-saving or may reduce complications substantially in several hematological disorders. The inevitable consequence of repeated transfusions is iron loading, which, if untreated, leads to organ failure and death. Chelation therapy with deferoxamine is the standard of care for patients who have transfusional iron overload. The necessity to administer this drug parenterally limits compliance; this has prompted the search for a safe and effective orally-administered chelator. Deferiprone, the first extensively studied orally active chelator, is now licensed for use in Europe for patients who are unable to use deferoxamine effectively or safely. ICL670, a newer oral chelator, is being tested in large clinical trials. Combined therapies, potentially including transfusional methods to reduce iron loading with parenteral and oral chelators, may improve compliance and efficacy in some patients who are transfused chronically.