Deferiprone in the treatment of transfusion-dependent thalassemia: a review and perspective

Abstract

Deferiprone is an orally active iron chelator which has emerged from an extensive search for new treatment of iron overload. Comparative studies have shown that at comparable doses deferiprone may be as effective as deferoxamine in removing body iron. Retrospective and prospective studies have shown that deferiprone monotherapy is significantly more effective than deferoxamine in improving myocardial siderosis in thalassemia major. Agranulocytosis is the most serious side effect associated with the use of deferiprone, occurring in about 1% of the patients. More common but less serious side effects are gastrointestinal symptoms, arthralgia, zinc deficiency, and fluctuating transaminases levels. Deferiprone can be used in combination with desferrioxamine. This regimen of chelation is tolerable and attractive for patients unable to comply with standard deferoxamine infusions or with inadequate response to deferiprone alone. Combination therapy has been effectively used in the management of severe cardiac siderosis.