Sustained Engraftment and Resolution of Bleeding Phenotype After Unrelated Cord Blood Hematopoietic Stem Cell Transplantation for Severe Glanzmann Thrombasthenia

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Abstract

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 transplantation has been curative; however, suitable donor availability can be a barrier for some patients. We are the first to report the use of umbilical cord blood hematopoietic stem cell transplantation for Glanzmann thrombasthenia.

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