Efficacy and safety of deferiprone (Ferriprox), an oral iron-chelating agent, in pediatric patients

ABSTRACT

BACKGROUND: Iron overload is a predictable and life-threatening complication in patients dependent on the regular transfusion of RBCs. The aims of this study were to investigate the efficacy and safety of deferiprone in a variety of pediatric hematologic and/or oncologic patients with a high iron overload. METHODS: Seventeen patients (age 1.1-20.4 years; median 10.6 years) from 7 hospitals who were treated with deferiprone from 2006 to 2009 were enrolled in this study. Medical records of enrolled patients were reviewed retrospectively. RESULTS: Serum ferritin levels were 4,677.8+/-1,130.9 microgram/L at baseline compared to 3,363.9+/-1,149.7 microgram/L at the end of deferiprone treatment (P=0.033). Only 1 patient developed neutropenia as a complication. CONCLUSION: Deferiprone treatment is relatively safe for pediatric patients suffering from various hematologic and oncologic diseases that require RBC transfusions as part of treatment. However, the potential development of critical complications such as agranulocytosis and/or neutropenia remains a concern.