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Allogeneic stem cell transplantation for Glanzmann thrombasthenia
Author(s) -
Ishaqi M. Kashif,
ElHayek M.,
Gassas Adam,
Khanani M.,
Trad Omar,
Baroudi M.,
Jamil A.
Publication year - 2009
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.21888
Subject(s) - medicine , thrombasthenia , sibling , glanzmann's thrombasthenia , hematopoietic stem cell transplantation , platelet , platelet disorder , platelet transfusion , transplantation , stem cell , platelet membrane glycoprotein , immunology , pediatrics , surgery , gastroenterology , platelet aggregation , psychology , developmental psychology , biology , genetics
Glanzmann thrombasthenia (GT) is a rare autosomal recessive bleeding disorder characterized by normal platelet count, but lack of platelet aggregation. The molecular basis is linked to quantitative and/or qualitative abnormalities of the membrane glycoprotein IIb/IIIa complexes. Usually it is associated with mild bleeding but may lead to severe and potentially fatal hemorrhages. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment. However, because of the risks associated with HSCT, it is generally not recommended unless there are life threatening hemorrhages, or the patient has developed refractoriness to platelet transfusion due to antibody formation. Herein, we report an 11‐year‐old female from United Arab Emirates (UAE) with severe GT and anti platelet alloimmunization successfully treated with HSCT from her HLA‐identical sibling. Pediatr Blood Cancer 2009;52:682–683. © 2008 Wiley‐Liss, Inc.