title: Long-term outcomes of hematopoietic stem cell transplantation for severe treatment-resistant autoimmune cytopenia in children.
abstract: We analyzed the long-term outcomes of pediatric patients registered in the European Group for Blood and Marrow Transplantation database who underwent hematopoietic stem cell transplantation (HSCT) for severe treatment refractory autoimmune cytopenia. With a median follow-up of 100 months, event-free survival was 54% overall, with no significant difference between allogeneic HSCT (n = 15) and autologous HSCT (n = 7) recipients (58% versus 42%; P = .50). Despite a trend toward failure of response or relapse after autologous HSCT compared with allogeneic HSCT, the difference was not significant (43% versus 13%; P = .30). Treatment-related mortality was high in both HSCT groups (29% and 16%; P = .09). Based on the limited numbers of subjects in this retrospective analysis, both allogeneic and autologous HSCT may induce complete and persistent responses in approximately one-half of pediatric patients with severe refractory autoimmune cytopenia, although treatment-related toxicity is high.
authors.affiliation: Institute of Maternal and Child Health, IRCCS Burlo Garofolo Trieste, Italy. rabusin@burlo.trieste.it
authors.forename: Marco
authors.initials: M
authors.lastname: Rabusin
authors.forename: J A
authors.initials: JA
authors.lastname: Snowden
authors.forename: Paul
authors.initials: P
authors.lastname: Veys
authors.forename: Pierre
authors.initials: P
authors.lastname: Quartier
authors.forename: Jean-Hughes
authors.initials: JH
authors.lastname: Dalle
authors.forename: Catharina
authors.initials: C
authors.lastname: Dhooge
authors.forename: Paolo
authors.initials: P
authors.lastname: Di Bartolomeo
authors.forename: Maria
authors.initials: M
authors.lastname: Gonzalez-Vicent
authors.forename: Brenda
authors.initials: B
authors.lastname: Gibson
authors.forename: Arturo
authors.initials: A
authors.lastname: Iriondo
authors.forename: Herbert
authors.initials: H
authors.lastname: Juergens
authors.forename: Igor
authors.initials: I
authors.lastname: Lisukov
authors.forename: Chiara
authors.initials: C
authors.lastname: Messina
authors.forename: Valerie
authors.initials: V
authors.lastname: Mialou
authors.forename: Colin Graham
authors.initials: CG
authors.lastname: Steward
authors.forename: Christian
authors.initials: C
authors.lastname: Urban
authors.forename: Marleen
authors.initials: M
authors.lastname: Renard
authors.forename: Nagua
authors.initials: N
authors.lastname: Giurici
authors.forename: Christina
authors.initials: C
authors.lastname: Peters
authors.forename: Manuela
authors.initials: M
authors.lastname: Badoglio
authors.forename: Luca
authors.initials: L
authors.lastname: Ronfani
authors.forename: Giorgio
authors.initials: G
authors.lastname: Dini
authors.forename: Dominique
authors.initials: D
authors.lastname: Farge
authors.forename: Riccardo
authors.initials: R
authors.lastname: Saccardi
mesh_terms: D000293:Adolescent; D001327:Autoimmune Diseases / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007223:Infant; D008297:Male; D009503:Neutropenia / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D012008:Recurrence; D012189:Retrospective Studies; D016019:Survival Analysis; D013921:Thrombocytopenia / Q000276:immunology / Q000473:pathology / Q000628:therapy*; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome