Document Type

Article

Publication Date

7-2015

Abstract

Glanzmann’s thrombasthenia (GT) is a genetic platelet surface receptor disorder of GPIIb/IIIa (ITG αIIbβ3), either qualitative or quantitative, which results in faulty platelet aggregation and diminished clot retraction. Spontaneous mucocutaneous bleeding is common and can lead to fatal bleeding episodes. Control and prevention of bleeding among patients with GT is imperative, and remains challenging. Local measures, including anti-fibrinolytic therapy, with or without platelet transfusions, used to be the mainstay of therapy. However, in recent years the use of recombinant factor VIIa (rFVIIa) has increased significantly, with excellent response rates in treating and preventing hemorrhage among GT patients. Gene therapy and stem cell transplantation offer a potential cure of this disease, but both are costly and remain experimental at this point. This manuscript offers a comprehensive review of our understanding of GT and the available treatment options.

Publication Title

Journal of Blood Medicine

Volume

2015

Issue

6

First Page

219

Last Page

227

Comments

This article was published in Journal of Blood Medicine, Volume 2015, Issue 6, July 2015, pages 219-227.

The published version is available at http://dx.doi.org/10.2147/JBM.S71319

Copyright © Solh et al. and licensed CC-BY

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