Deferiprone for the treatment of transfusional iron overload in thalassemia

ABSTRACT

Introduction: 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
• iron chelation
• deferiprone
• magnetic resonance imaging
• iron overload

Declaration of interest

JL Kwiatkowski has served as a consultant for Agios and Ionis Pharmaceuticals. She is the principal investigator in an ongoing study of deferiprone in sickle cell anemia and other transfusion-dependent anemias sponsored by Anopharma. She is an investigator in clinical trials sponsored by Novartis and Agios pharmaceuticals and Bluebird Bio, inc. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Additional information

Funding

This paper was not funded.