title: Use of allogeneic stem cell transplantation for moderate-severe Glanzmann thrombasthenia.
abstract: Glanzmann thrombasthenia (GT) is a rare, autosomal recessive coagulopathy characterized by either qualitative or quantitative abnormalities of the membrane glycoprotein αIIbβ3 complex leading to bleeding tendencies, ranging from purpura to life-threatening hemorrhage. Although patients can be managed with supportive measures including platelet transfusions, complications such as alloimmunization are possible. Allogeneic stem cell transplantation (ASCT) can be indicated in severe cases of GT. We report the case of an eight-month-old girl diagnosed with moderate-severe GT, who was successfully treated with a reduced-intensity, human leukocyte antigen (HLA)-identical ASCT.
authors.affiliation: a Department of Hematology/Oncology/Transplantation , Ann & Robert H. Lurie Children's Hospital , Chicago , IL , USA .
authors.forename: Amy
authors.initials: A
authors.lastname: Walz
authors.forename: Alicia
authors.initials: A
authors.lastname: Lenzen
authors.forename: Brian
authors.initials: B
authors.lastname: Curtis
authors.forename: Jason
authors.initials: J
authors.lastname: Canner
authors.forename: Jennifer
authors.initials: J
authors.lastname: Schneiderman
mesh_terms: D001792:Blood Platelets / Q000276:immunology / Q000378:metabolism; D005260:Female; D006680:HLA Antigens / Q000235:genetics / Q000276:immunology; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D012720:Severity of Illness Index; D035781:Siblings; D013915:Thrombasthenia / Q000175:diagnosis / Q000628:therapy*; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D006680:HLA Antigens
keywords: Glanzmann thrombasthenia; pediatric hematology/oncology; platelet disorders; stem cell transplant