Sustained engraftment and resolution of bleeding phenotype after unrelated cord blood hematopoietic stem cell transplantation for severe glanzmann thrombasthenia.

Abstract excerpt

Glanzmann thrombasthenia is a rare, autosomal recessive, qualitative platelet disorder resulting from abnormal platelet surface glycoprotein IIb/IIIa. In phenotypically severe cases, medical management is often challenging. Although definitive hemostasis can be achieved with platelet transfusion, alloimmunization and subsequent platelet refractoriness remain a real risk. To date, only hematopoietic stem cell...

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