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Eltrombopag

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Abstract

  • ▴ Eltrombopag, an orally administered, small-molecule non-peptide thrombopoietin receptor agonist, selectively binds to the transmembrane domain of the thrombopoietin receptor on the surface of platelets, megakaryocytes and megakaryocyte precursor cells. The drug acts via the Janus Kinase/Signal Transducer and Activator of Transcription (JAK/STAT) pathway to activate megakaryocyte proliferation and differentiation in bone marrow progenitor cells, similar to those observed with endogenous thrombopoietin.

  • ▴ Platelet counts are increased as a result of eltrombopag therapy, and the drug has shown good clinical efficacy in adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP) in randomized, double-blind, placebo-controlled, multi-centre, phase II dose-finding and phase III trials.

  • ▴ After 6 weeks of therapy in the phase III trial, eltrombopag 50 mg/day was associated with a significantly higher response rate (proportion of patients with a platelet count of ≥50 000 cells/μL at day 43; primary endpoint) than placebo in adult patients with chronic ITP.

  • ▴ In addition, the proportion of patients with ITP achieving a platelet count of >200 000 cells/μL and discontinuing treatment due to protocol-defined treatment-cessation criteria, was ≈8-fold higher with eltrombopag than with placebo.

  • ▴ Eltrombopag therapy for 6 weeks also significantly decreased the incidence of WHO-defined bleeding compared with placebo.

  • ▴ Eltrombopag was generally well tolerated in clinical trials, with an adverse events profile that did not differ significantly from that with placebo.

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Acknowledgments and Disclosures

The manuscript was reviewed by: G. Cheng, Department of Medicine & Therapeutics, Prince of Wales Hospital, Shatin, Hong Kong; M. Kuwana, Department of Internal Medicine, Keio University School of Medicine, Tokyo, Japan.

The preparation of this review was not supported by any external funding. During the peer review process, the manufacturer of the agent under review was offered an opportunity to comment on this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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Authors and Affiliations

  1. Wolters Kluwer Health ¦ Adis, 41 Centorian Drive, Private Bag 65901, Mairangi Bay, North Shore, Auckland, 0754, New Zealand

    Karly P. Garnock-Jones & Susan J. Keam

  2. Wolters Kluwer Health, Philadelphia, Pennsylvania, USA

    Karly P. Garnock-Jones & Susan J. Keam

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  1. Karly P. Garnock-Jones
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  2. Susan J. Keam
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Correspondence to Karly P. Garnock-Jones.

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Garnock-Jones, K.P., Keam, S.J. Eltrombopag. Drugs 69, 567–576 (2009). https://doi.org/10.2165/00003495-200969050-00005

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