title: Haematopoetic stem cell transplantation for refractory autoimmune cytopenia.
abstract: This study describes the outcome of patients receiving haematopoietic stem cell transplantation (HSCT) to treat severe refractory autoimmune cytopenia. The registry of the European Group of Blood and Marrow Transplantation holds data on 36 patients receiving 38 transplants, the first transplant was autologous for 27 and allogeneic for nine patients. Patients had autoimmune haemolytic anaemia (autologous: 5; allogeneic: 2), Evans's syndrome (autologous: 2; allogeneic: 5); immune thrombocytopenia (autologous: 12), pure red cell aplasia (autologous: 4; allogeneic: 1), pure white cell aplasia (autologous: 1; allogeneic 1), or thrombotic thrombocytopenic purpura (autologous: 3). Patients had longstanding disease having failed multiple prior treatments. Among 26 evaluable patients mobilized for autologous HSCT, three died of treatment-related causes, one died of disease progression, seven were non-responders, six patients had transient responses and nine had continuous partial or complete remission. Of the seven evaluable patients receiving allogeneic HSCT, one died of treatment-related complications, one with transient response died of progressive disease and five had a continuous response. Autologous and allogeneic HSCT may induce a response in a subset of patients with autoimmune cytopenia of long duration albeit at the price of considerable toxicity.
authors.affiliation: Basel University Hospitals, Basel, Switzerland. jakob.passweg@unibas.ch
authors.forename: J R
authors.initials: JR
authors.lastname: Passweg
authors.forename: M
authors.initials: M
authors.lastname: Rabusin
authors.forename: M
authors.initials: M
authors.lastname: Musso
authors.forename: Y
authors.initials: Y
authors.lastname: Beguin
authors.forename: S
authors.initials: S
authors.lastname: Cesaro
authors.forename: G
authors.initials: G
authors.lastname: Ehninger
authors.forename: I
authors.initials: I
authors.lastname: Espigado
authors.forename: A
authors.initials: A
authors.lastname: Iriondo
authors.forename: L
authors.initials: L
authors.lastname: Jost
authors.forename: V
authors.initials: V
authors.lastname: Koza
authors.forename: S
authors.initials: S
authors.lastname: Lenhoff
authors.forename: I
authors.initials: I
authors.lastname: Lisukov
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: A
authors.initials: A
authors.lastname: Marmont
authors.forename: P
authors.initials: P
authors.lastname: Philippe
authors.forename: C
authors.initials: C
authors.lastname: Pilatrino
authors.forename: P
authors.initials: P
authors.lastname: Quartier
authors.forename: J
authors.initials: J
authors.lastname: Stary
authors.forename: P
authors.initials: P
authors.lastname: Veys
authors.forename: J
authors.initials: J
authors.lastname: Vormoor
authors.forename: A
authors.initials: A
authors.lastname: Wahlin
authors.forename: F
authors.initials: F
authors.lastname: Zintl
authors.forename: C
authors.initials: C
authors.lastname: Bocelli-Tyndall
authors.forename: A
authors.initials: A
authors.lastname: Tyndall
authors.forename: A
authors.initials: A
authors.lastname: Gratwohl
mesh_terms: D000293:Adolescent; D000328:Adult; D000744:Anemia, Hemolytic, Autoimmune / Q000628:therapy; D001327:Autoimmune Diseases / Q000628:therapy*; D002648:Child; D002675:Child, Preschool; D016208:Databases, Factual; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012010:Red-Cell Aplasia, Pure / Q000628:therapy; D013921:Thrombocytopenia / Q000628:therapy*; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous; D016896:Treatment Outcome