title: Autologous haematopoietic stem cell transplants for autoimmune disease--feasibility and transplant-related mortality. Autoimmune Disease and Lymphoma Working Parties of the European Group for Blood and Marrow Transplantation, the European League Against Rheumatism and the International Stem Cell Project for Autoimmune Disease.
abstract: This ongoing multicentre prospective phase I/II trial enrolled 74 consecutive patients from 22 centres worldwide with severe autoimmune disease, 35 with rheumatological disorders, 31 with neurological, five with haematological and three with vasculitides. They were treated with autologous peripheral blood or bone marrow transplants according to predetermined criteria. Two patients died after mobilisation before transplant. Seventy-two patients were given 73 transplants, seven bone marrow, and 66 mobilised peripheral blood stem cell transplants. The graft was manipulated to remove T and/or B cells in 43 cases. All 73 transplants engrafted. Five patients died of transplant-related complications: two from bleeding, three from infections. Two patients died of progressive disease. The transplant-related mortality at 1 year of 9% (1-17%; 95% CI) is comparable to the transplant-related mortality of 6% (3-9%; 95% CI) in patients transplanted during the same period in Europe for non-Hodgkin's lymphoma in sensitive relapse (P = 0.39). Sixty patients are evaluable for response, 40 patients (65%) showed some improvement in their disease. Haematopoietic stem cell transplants are feasible for patients with severe refractory autoimmune disease. Transplant-related mortality is comparable to results in patients with non-Hodgkin's lymphoma in responsive relapse. Two-thirds of the patients show at least some response. These preliminary data are promising. Although associated with considerable risk, randomised trials comparing autologous stem cell transplants to conventional therapy are warranted.
authors.affiliation: University Rheumatology Clinic, Felix Platter Hospital, Basel, Switzerland.
authors.forename: A
authors.initials: A
authors.lastname: Tyndall
authors.forename: A
authors.initials: A
authors.lastname: Fassas
authors.forename: J
authors.initials: J
authors.lastname: Passweg
authors.forename: C
authors.initials: C
authors.lastname: Ruiz de Elvira
authors.forename: M
authors.initials: M
authors.lastname: Attal
authors.forename: P
authors.initials: P
authors.lastname: Brooks
authors.forename: C
authors.initials: C
authors.lastname: Black
authors.forename: P
authors.initials: P
authors.lastname: Durez
authors.forename: J
authors.initials: J
authors.lastname: Finke
authors.forename: S
authors.initials: S
authors.lastname: Forman
authors.forename: L
authors.initials: L
authors.lastname: Fouillard
authors.forename: D
authors.initials: D
authors.lastname: Furst
authors.forename: J
authors.initials: J
authors.lastname: Holmes
authors.forename: D
authors.initials: D
authors.lastname: Joske
authors.forename: J
authors.initials: J
authors.lastname: Jouet
authors.forename: I
authors.initials: I
authors.lastname: Kötter
authors.forename: F
authors.initials: F
authors.lastname: Locatelli
authors.forename: H
authors.initials: H
authors.lastname: Prentice
authors.forename: A M
authors.initials: AM
authors.lastname: Marmont
authors.forename: P
authors.initials: P
authors.lastname: McSweeney
authors.forename: M
authors.initials: M
authors.lastname: Musso
authors.forename: H H
authors.initials: HH
authors.lastname: Peter
authors.forename: J A
authors.initials: JA
authors.lastname: Snowden
authors.forename: K
authors.initials: K
authors.lastname: Sullivan
authors.forename: A
authors.initials: A
authors.lastname: Gratwohl
mesh_terms: D000293:Adolescent; D000328:Adult; D001327:Autoimmune Diseases / Q000628:therapy*; D002648:Child; D005240:Feasibility Studies; D005260:Female; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods* / Q000401:mortality*; D006801:Humans; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D014182:Transplantation, Autologous / Q000379:methods / Q000401:mortality