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New advances in the treatment of adult chronic immune thrombocytopenic purpura: role of thrombopoietin receptor-stimulating agents

Authors Metjian A, Abrams C

Published 4 December 2009 Volume 2009:3 Pages 499—513


Review by Single-blind

Peer reviewer comments 3

Ara Metjian1, Charles S Abrams2

1Department of Medicine, Duke University Medical Center, Durham, NC, USA; 2Department of Medicine, University of Pennsylvania, Philadelphia, PA, USA

Abstract: Decades of basic science and clinical research have led to an increased understanding of the pathophysiology of immune thrombocytopenic purpura (ITP), the processes underlying thrombopoiesis, and the treatment of chronic ITP. Now, new agents are available to treat ITP in a nonimmunosuppressive fashion. Lessons learned from the clinical trials of recombinant human thrombopoietin (TPO) have led to the development of a novel class of compounds: nonimmunogenic agonists of the thrombopoietin receptor. Representing the first nonimmunosuppressive agents to treat chronic refractory ITP in decades, medications such as romiplostim and eltrombopag were recently approved by the US Food and Drug Administration. These new agents offer physicians a new tool for treating difficult cases of ITP in their medical armamentarium. Additional TPO mimetics are also being developed that show promise in vitro, and await future development.

Keywords: thrombocytopenia, immune-mediated thrombocytopenia, eltrombopag, romiplostim, thrombopoietin, thrombopoietin receptor

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