title: Indication for allogeneic stem cell transplantation in Glanzmann's thrombasthenia.
abstract: Glanzmann's thrombasthenia (GT) is an autosomal recessive disorder characterized by a lack of thrombocyte aggregation due to the absence of thrombocyte glycoproteins IIb and αIIbβ3. The role of haematopoietic stem cell transplantation (HSCT) in GT remains controversial. However, HSCT offers the only curative approach for patients with a severe clinical phenotype. In this review, we will discuss the limitation of current status evidence and the specific risk of GT, in particular the alloimmunization and refractoriness to thrombocyte infusions. 19 successful HSCT in 18 GT type I patients have been reported. Mean age at transplantation was 5 years. All patients are still alive. The majority received sibling bone marrow transplant with busulfan and cyclophosphamid conditioning. GvHD incidence was within the normal range, but 10 patients showed alloimmunization of thrombocytes. Median follow up is 25 months.
authors.affiliation: Verena Wiegering, MD, Dept. of Paediatric Haematology, Oncology, Paediatric Stem Cell Transplantation Program, University Children's Hospital Wuerzburg,, Josef-Schneider-Str. 2, 97080 Wuerzburg, Germany, E-mail: Wiegering_V@klinik.uni-wuerzburg.de.
authors.forename: V
authors.initials: V
authors.lastname: Wiegering
authors.forename: K
authors.initials: K
authors.lastname: Sauer
authors.forename: B
authors.initials: B
authors.lastname: Winkler
authors.forename: M
authors.initials: M
authors.lastname: Eyrich
authors.forename: P G
authors.initials: PG
authors.lastname: Schlegel
mesh_terms: D019317:Evidence-Based Medicine; D006801:Humans; D011183:Postoperative Complications / Q000401:mortality*; D015995:Prevalence; D012307:Risk Factors; D033581:Stem Cell Transplantation / Q000401:mortality* / Q000706:statistics & numerical data*; D015996:Survival Rate; D013915:Thrombasthenia / Q000401:mortality* / Q000601:surgery*; D014184:Transplantation, Homologous / Q000401:mortality / Q000706:statistics & numerical data; D016896:Treatment Outcome
keywords: Glanzmann thrombasthenia; allogeneic stem cell transplantation; alloimmunization