Advances in iron chelation therapy: transitioning to a new oral formulation

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Iron overload is a concern for patients who require repeated red-blood-cell transfusions due to conditions such as sickle cell disease, thalassemia, or myelodysplastic syndromes. The recommended treatment for removing excess iron in these patients is iron chelation therapy. Currently available iron chelators include deferoxamine, which is administered by injection, and deferasirox and deferiprone, both of which are administered orally. Adherence to iron chelator therapy is an important consideration and may be affected by side effects. A new formulation of deferasirox, a film-coated tablet (FCT), has the potential to improve adherence by offering greater flexibility in administration compared with the original formulation of deferasirox, a dispersible tablet (DT) for oral suspension. This review provides an overview of the currently available iron chelator formulations, with a focus on a comparison between deferasirox DT for oral suspension and deferasirox FCT. The new formulation may be associated with fewer side effects and has increased bioavailability. In addition, alternative strategies for iron chelation, such as combining two different iron chelators, will be discussed.

Keywords: deferasirox, deferiprone, deferoxamine, iron chelation, myelodysplastic syndromes, sickle cell disease, thalassemia.

Abbreviations: AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; DT, dispersible tablet; FCT, film-coated tablet; FDA, US Food and Drug Administration; GI, gastrointestinal; ICT, iron chelation therapy; IM, intramuscular; IV, intravenous; LIC, liver iron concentration; MDS, myelodysplastic syndromes; MRI, magnetic resonance imaging; NF, National Formulary; NTBI, non-transferrin-bound iron; NTDT, non–transfusion-dependent thalassemia; RBC, red blood cell; SC, subcutaneous; SCD, sickle cell disease; TDT, transfusion-dependent thalassemia

Citation: Shah NR. Advances in iron chelation therapy: transitioning to a new oral formulation. Drugs in Context 2017; 6: 212502. DOI: 10.7573/dic.212502

Disclosure and potential conflicts of interest: Nirmish R. Shah, MD, is a member of a Novartis Pharmaceuticals Corporation speakers’ bureau. The international Committee of Medical Journal Editors (ICMJE) Potential Conflicts of Interest form for the author is available for download at:

Acknowledgments: The author was responsible for all content and editorial decisions and received no honoraria related to the development of this publication. The author performed the research, writing, and review of all drafts of this manuscript and approved the final draft.

Funding Declaration: Editorial assistance was provided by Jennifer Lee, PhD, of Phase Five Communications, Inc., supported by Novartis Pharmaceuticals Corporation, which had no other involvement in the development of this publication.

Copyright: Copyright © 2017 Shah NR. Distributed under the terms of the Creative Commons License Deed CC BY NC ND 3.0 which allows anyone to copy, distribute, and transmit the article provided it is properly attributed in the manner specified below. No commercial use without permission.

Correct attribution: Copyright © 2017 Shah NR. Published by Drugs in Context under Creative Commons License Deed CC BY NC ND 3.0.

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Correspondence: Nirmish R. Shah, MD, Duke University School of Medicine, Durham, NC, USA.

Provenance: submitted; externally peer reviewed

Submitted: 8 February 2017; Peer review comments to author: 27 February 2017; Revised manuscript received: 2 May 2017; Publication: 16 June 2017

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