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Romiplostim

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Abstract

  • ▴ Romiplostim is an Fc-peptide fusion protein (or ‘peptibody’) that stimulates megakaryopoiesis and thrombopoiesis by binding to, and activating, the thrombopoietin receptor.

  • ▴ Because it has no sequence homology to thrombopoietin, romiplostim theoretically avoids the risk of eliciting cross-reacting, neutralizing antibodies to thrombopoietin.

  • ▴ In well designed, 24-week, phase III trials, subcutaneous romiplostim was significantly more effective than placebo in achieving the primary endpoint of a protocol-defined durable platelet response in nonsplenectomized (61% vs 5%) or splenectomized (38% vs 0%) adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP).

  • ▴ Romiplostim was also significantly more effective than placebo with regard to a number of secondary endpoints, including the proportions of patients with an overall (durable plus transient) platelet response or who required ITP rescue medications. The majority of romiplostim-treated patients receiving concurrent ITP drugs were able to reduce or discontinue these therapies.

  • ▴ Platelet response was maintained by most patients during longer-term treatment with romiplostim for up to 3 or 4 years in an open-label extension study.

  • ▴ Romiplostim was generally well tolerated. Almost all adverse events in the phase III studies were of mild-to-moderate intensity; most were unrelated to treatment. Longer-term treatment with romiplostim had an adverse event profile consistent with that observed in the phase III studies.

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Acknowledgments and Disclosures

The manuscript was reviewed by: B. Godeau, Médecine Interne, Hôpital Henri-Mondor, Assistance Publique-Hôpitaux de Paris, Université Paris, Créteil, Paris, France; R. Stasi, Department of Medical Sciences, Ospedale “Regina Apostolorum”, Albano Laziale, Rome, Italy.

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 0754, Auckland, New Zealand

    James E. Frampton & Katherine A. Lyseng-Williamson

  2. Wolters Kluwer Health, Conshohocken, Pennsylvania, USA

    James E. Frampton & Katherine A. Lyseng-Williamson

Authors
  1. James E. Frampton
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  2. Katherine A. Lyseng-Williamson
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Correspondence to James E. Frampton.

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Frampton, J.E., Lyseng-Williamson, K.A. Romiplostim. Drugs 69, 307–317 (2009). https://doi.org/10.2165/00003495-200969030-00006

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  • DOI: https://doi.org/10.2165/00003495-200969030-00006

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