An evaluation of deferiprone as twice-a-day tablets or in combination therapy for the treatment of transfusional iron overload in thalassemia syndromes

ABSTRACT

Introduction

Regular blood transfusions in patients with thalassemia syndromes can cause iron overload resulting in complications including cirrhosis, heart problems, or endocrine abnormalities. To prevent iron overload toxicity in these patients, three iron chelators are currently FDA-approved for use: deferoxamine, deferasirox, and deferiprone. In the United States, deferiprone has been approved for three times daily dosing since 2011 and has recently gained approval for twice-daily administration.

Areas Covered

A PubMed literature search was performed with the keywords ‘deferiprone’ and ‘thalassemia.’ Relevant original research studying deferiprone’s effects on transfusional iron overload in patients with thalassemia syndromes was included. Exclusion criteria included case reports and review papers. Deferiprone is effective at reducing serum ferritin levels in patients with iron overload. Twice-daily administration provides a similar level of iron chelation as three times daily dosing with a comparable side effect profile and increased patient acceptability.

Expert Opinion

New studies are highlighting deferiprone’s potential for combination therapy with either deferoxamine or deferasirox to improve iron chelation. Deferiprone’s ability to significantly decrease cardiac and liver iron content can be utilized in other transfusion-dependent hematologic conditions, as evidenced by its recent approval for use in the United States for sickle cell disease or other anemias.

KEYWORDS:

• Deferiprone
• L1
• ferriprox
• chelation
• chelator
• thalassemia
• hemoglobinopathy
• transfusion
• iron
• iron overload
• ferritin

Article highlights

• Deferiprone (DFP) is one of the three iron chelators used to treat transfusional iron overload in patients with thalassemia syndromes and can be used alone or as a dual-chelation therapy.

• DFP has shown to be just as efficacious as other iron chelators in lowering serum ferritin and total body iron.

• Single and dual chelation DFP have both shown to be superior in lowering cardiac iron content compared to other iron chelators.

• It has been standard for DFP to be administered in three divided daily doses; however, the FDA recently approved a twice-a-day tablet formulation in May 2020.

• Twice-daily administration of DFP is just as efficacious and can improve medication adherence due to more convenient administration and lower pill burden.

• DFP has also shown potential for use in other hematological conditions due to its ability to decrease cardiac and liver iron content. DFP has been approved for use in sickle cell disease or other anemias by the US FDA in 2021.

Declaration of interest

The authors have no 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose. Chiesi provided a scientific accuracy review at the request of the journal editor.

Additional information

Funding

This project was supported by grant (K23HL150232, PI: Badawy) from the National Heart, Lung, and Blood Institute of the National Institutes of Health. The content is solely the responsibility of the authors and does not necessarily represent the National Institutes of Health.