TY - JOUR
T1 - Sustained engraftment and resolution of bleeding phenotype after unrelated cord blood hematopoietic stem cell transplantation for severe Glanzmann thrombasthenia
AU - Miller, Weston P
AU - Dunn, Amy
AU - Chiang, Kuang Yueh
PY - 2009/6/1
Y1 - 2009/6/1
N2 - 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.
AB - 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.
KW - Glanzmann thrombasthenia
KW - Hematopoietic stem cell transplantation
KW - Umbilical cord blood transplantation
UR - http://www.scopus.com/inward/record.url?scp=67651209052&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=67651209052&partnerID=8YFLogxK
U2 - 10.1097/MPH.0b013e31819b7228
DO - 10.1097/MPH.0b013e31819b7228
M3 - Article
C2 - 19648793
AN - SCOPUS:67651209052
SN - 1077-4114
VL - 31
SP - 437
EP - 439
JO - Journal of pediatric hematology/oncology
JF - Journal of pediatric hematology/oncology
IS - 6
ER -