title: Sustained engraftment and resolution of bleeding phenotype after unrelated cord blood hematopoietic stem cell transplantation for severe glanzmann thrombasthenia.
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.
authors: Department of Pediatrics, Division of Hematology and Oncology, Emory University, Children's Healthcare of Atlanta, Atlanta, GA, USA. wes.miller@choa.org
authors: Weston
authors: W
authors: Miller
authors: Amy
authors: A
authors: Dunn
authors: Kuang-Yueh
authors: KY
authors: Chiang
mesh_terms: D036101:Cord Blood Stem Cell Transplantation; D006085:Graft Survival; D018380:Hematopoietic Stem Cell Transplantation; D006470:Hemorrhage / Q000209:etiology / Q000628:therapy; D006801:Humans; D007223:Infant; D008297:Male; D010641:Phenotype; D013915:Thrombasthenia / Q000150:complications / Q000628:therapy*; D014019:Tissue Donors; D019172:Transplantation Conditioning / Q000379:methods