title: Deferiprone: new preparation. Poorly assessed.
abstract: Deferiprone, an oral iron chelator, has been licensed in Europe for the treatment of iron overload in patients with beta thalassaemia who cannot be treated with deferoxamine because of adverse effects or the difficult administration schedule. (2) The clinical dossier is thin and methodologically mediocre. Even the preclinical assessment dossier is insufficient (there are no studies of carcinogenicity or hepatotoxicity). (3) The only available comparative trial, involving a small number of patients, suggests that deferiprone is less effective than deferoxamine in reducing ferritin levels and hepatic iron stores. (4) A non comparative trial suggests that long-term deferiprone therapy would not only fail, but would also increase the risk of liver fibrosis in some patients. This doubt is not dispelled by the other available non-comparative trials. (5) Deferiprone carries a risk of agranulocytosis in an estimated 1.2% of patients. (6) Deferiprone is teratogenic in two animal species.
mesh_terms: D000380:Agranulocytosis / Q000139:chemically induced*; D002170:Canada; D002986:Clinical Trials as Topic; D005060:Europe; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use; D019190:Iron Overload; D011728:Pyridones / Q000009:adverse effects / Q000627:therapeutic use*; D016896:Treatment Outcome; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones