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: Weston Miller, Amy Dunn, Kuang-Yueh Chiang
• Journal: Journal of pediatric hematology/oncology (J Pediatr Hematol Oncol) Vol. 31 Issue 6 Pg. 437-9 (Jun 2009) ISSN: 1536-3678 [Electronic] United States
• PMID: 19648793 (Publication Type: Case Reports, Journal Article)

Topics

• Cord Blood Stem Cell Transplantation
• Graft Survival
• Hematopoietic Stem Cell Transplantation
• Hemorrhage (etiology, therapy)
• Humans
• Infant
• Male
• Phenotype
• Thrombasthenia (complications, therapy)
• Tissue Donors
• Transplantation Conditioning (methods)