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Beyond immune thrombocytopenia: the evolving role of thrombopoietin receptor agonists

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Abstract

Since its discovery, the thrombopoietin (TPO) pathway has been an important pharmaceutical target for the treatment of thrombocytopenia. The first generation of TPO mimetics included peptide agents sharing homology with endogenous TPO, but these introduced a risk of antibody formation to endogenous TPO and were not successful. However, second-generation TPO mimetics or TPO receptor agonists (RAs) are currently being used to treat thrombocytopenia associated with a number of conditions, such as immune thrombocytopenia (ITP), severe aplastic anaemia (SAA), and hepatitis C virus-associated chronic liver disease. Accumulating efficacy and safety data suggest that the role of TPO-RAs in the treatment of thrombocytopenia may evolve in the near future with broader use of these agents in ITP and SAA, as well as approval in other indications, potentially including myelodysplastic syndromes, chemotherapy-associated thrombocytopenia, and post-transplant thrombocytopenia. This review provides an overview of clinical data on the efficacy and safety of TPO-RAs, emphasising recent findings that may expand their clinical utility.

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Acknowledgments

All listed authors meet the criteria for authorship set forth by the International Committee of Medical Journal Editors. Editorial support for the preparation of this manuscript was funded by Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.

Novartis did not influence the content of the manuscript, nor did the authors receive financial compensation for authoring the manuscript.

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Authors and Affiliations

  1. Hematology Project Foundation/Fondazione Progetto Ematologia, S. Bortolo Hospital, Vicenza, Italy

    Francesco Rodeghiero

  2. Department of Hematology, S. Bortolo Hospital, Vicenza, Italy

    Francesco Rodeghiero & Giuseppe Carli

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  1. Francesco Rodeghiero
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  2. Giuseppe Carli
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Correspondence to Francesco Rodeghiero.

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Conflict of interest

FR has served on Advisory Boards/Speaker’s Bureaus for Amgen, GSK/Novartis, LFB, MedImmune, UCB and Argenx, all outside the submitted work. GC has no conflicts of interest to disclose.

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Rodeghiero, F., Carli, G. Beyond immune thrombocytopenia: the evolving role of thrombopoietin receptor agonists. Ann Hematol 96, 1421–1434 (2017). https://doi.org/10.1007/s00277-017-2953-6

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