title: Stem cell transplantation for children with Glanzmann thrombasthenia.
abstract: Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet function disorder. Stem cell transplantation (SCT) is curative, but it is only indicated in selected patients with a severe clinical phenotype or who develop anti-platelet antibodies. SCT have previously been limited to full intensity myeloablative conditioning regimens. This study details the successful outcome of SCT in five consecutive patients with GT, three of whom received reduced intensity conditioning (RIC) with stem cells from non-sibling donors. This is the first time RIC SCT has been reported in GT, and offers the possibility of curative therapy with reduced late effects.
authors.affiliation: Department of Haematology, Central Middlesex Hospital, North West London NHS Trust, London, UK. connor807@aol.com
authors.forename: P
authors.initials: P
authors.lastname: Connor
authors.forename: K
authors.initials: K
authors.lastname: Khair
authors.forename: R
authors.initials: R
authors.lastname: Liesner
authors.forename: P
authors.initials: P
authors.lastname: Amrolia
authors.forename: P
authors.initials: P
authors.lastname: Veys
authors.forename: P
authors.initials: P
authors.lastname: Ancliff
authors.forename: M
authors.initials: M
authors.lastname: Mathias
mesh_terms: D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006086:Graft vs Host Disease; D006801:Humans; D008297:Male; D033581:Stem Cell Transplantation / Q000379:methods*; D013915:Thrombasthenia / Q000628:therapy*; D018183:Transplantation Chimera; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome