Deferiprone for the treatment of transfusional iron overload in thalassemia

Expert Rev Hematol. 2017 Jun;10(6):493-503. doi: 10.1080/17474086.2017.1318052.

Abstract

Transfusional iron overload can lead to hepatic fibrosis, arrhythmias and congestive heart failure and a number of endocrinopathies. Deferiprone is an oral iron chelator approved for use in the United States as a second line agent for the treatment of transfusional iron overload in patients with thalassemia. Areas covered: This article will review the data regarding the efficacy of deferiprone for iron chelation and prevention and reversal of iron related complications, the drug's adverse effect profile, and the use of this drug in combination regimens. Expert commentary: Extensive data support that deferiprone is particularly efficacious at cardiac iron removal and therefore, a chelator regimen that contains deferiprone is generally recommended when there is significant cardiac iron loading and/or in the setting of iron-related cardiac disease. The most concerning side effects of deferiprone are agranulocytosis and milder forms of neutropenia, which require appropriate monitoring and patient/provider education.

Keywords: Thalassemia; deferiprone; iron chelation; iron overload; magnetic resonance imaging.

Publication types

  • Review

MeSH terms

  • Deferiprone
  • Erythrocyte Transfusion / adverse effects*
  • Humans
  • Iron / metabolism
  • Iron Overload / drug therapy*
  • Iron Overload / etiology
  • Iron Overload / metabolism
  • Myocardium / metabolism
  • Neutropenia / chemically induced
  • Neutropenia / metabolism
  • Pyridones / adverse effects
  • Pyridones / therapeutic use*
  • Thalassemia / metabolism
  • Thalassemia / therapy*

Substances

  • Pyridones
  • Deferiprone
  • Iron