Stem cell transplantation for children with Glanzmann thrombasthenia

British Journal of Haematology
P ConnorM Mathias

Abstract

Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet function disorder. Stem cell transplantation (SCT) is curative, but it is only indicated in selected patients with a severe clinical phenotype or who develop anti-platelet antibodies. SCT have previously been limited to full intensity myeloablative conditioning regimens. This study details the successful outcome of SCT in five consecutive patients with GT, three of whom received reduced intensity conditioning (RIC) with stem cells from non-sibling donors. This is the first time RIC SCT has been reported in GT, and offers the possibility of curative therapy with reduced late effects.

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Citations

Jul 23, 2013·Hämostaseologie·V WiegeringP G Schlegel
Mar 13, 2016·Transfusion Medicine Reviews·Man-Chiu PoonRainer Zotz
Aug 8, 2009·The Journal of Thoracic and Cardiovascular Surgery·Jake GillenFred A Crawford
Dec 23, 2008·Pediatric Blood & Cancer·M Kashif IshaqiA Jamil
Nov 26, 2010·The Laryngoscope·R Raul RosasJames Sidman
Oct 24, 2009·Pediatric Transplantation·Carrie L KitkoPaul A Carpenter
Mar 30, 2010·Haemophilia : the Official Journal of the World Federation of Hemophilia·C SantoroM G Mazzucconi
May 12, 2010·British Journal of Haematology·Jayanthi Alamelu, Ri Liesner
Nov 14, 2012·Expert Review of Hematology·Alan T NurdenPaquita Nurden

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