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Glanzmann's thrombasthenia: pathogenesis, diagnosis, and current and emerging treatment options

Authors Solh T, Botsford A, Solh M

Received 8 March 2015

Accepted for publication 29 April 2015

Published 8 July 2015 Volume 2015:6 Pages 219—227


Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 3

Editor who approved publication: Dr Martin H. Bluth

Tia Solh,1 Ashley Botsford,2 Melhem Solh3

1Department of Physician Assistant Studies, Philadelphia College of Osteopathic Medicine, Suwanee, GA, USA; 2College of Health Care Sciences, Nova Southeastern University, Orlando, FL, USA; 3The Blood and Marrow Transplant Group of Georgia, Northside Hospital, Atlanta, GA, USA

Abstract: Glanzmann’s thrombasthenia (GT) is a genetic platelet surface receptor disorder of GPIIb/IIIa (ITG αIIbß3), 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.

Keywords: Glanzmann, thrombasthenia, treatment

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