title: Autologous haematopoietic stem cell transplantation for systemic lupus erythematosus: data from the European Group for Blood and Marrow Transplantation registry.
abstract: OBJECTIVE
  Patients with systemic lupus erythematosus (SLE) refractory to conventional immunosuppression suffer substantial morbidity and mortality due to active disease and treatment toxicity. Immunoablation followed by autologous stem cell transplantation (ASCT) is a novel therapeutic strategy that potentially offers new hope to these patients.
  
  
  METHODS
  This retrospective survey reviews the efficacy and safety of ASCT in 28 SLE patients from eight centres reported to the European Group for Blood and Marrow Transplantation (EBMT) registry between 2001 and 2008.
  
  
  RESULTS
  Median disease duration before ASCT was 52 (nine to 396) months, 25/28 SLE patients (89%) were female, age 29 (16-48) years. At the time of ASCT, eight (one to 11) American College of Rheumatology (ACR) diagnostic criteria for SLE were present and 17 (60%) patients had nephritis. Peripheral blood stem cells were mobilized with cyclophosphamide and granulocyte-colony stimulating factor in 93% of patients, and ex vivo CD34 stem cell selection was performed in 36%. Conditioning regimens were employed with either low (n = 10) or intermediate (18) intensities. With a median follow-up of 38 (one to 110) months after ASCT, the five-year overall survival was 81 ± 8%, disease-free survival was 29 ± 9%, relapse incidence (RI) was 56 ± 11% and non-relapse mortality was 15 ± 7%. Graft manipulation by CD34+ selection was associated with a lower RI (p = 0.001) on univariate analysis. There were five deaths within two years after ASCT: three caused by infection, one by secondary autoimmune disease and one by progressive SLE.
  
  
  CONCLUSIONS
  Our data further support the concept of immunoablation and ASCT to re-induce long-term clinical and serologic remissions in refractory SLE patients even in the absence of maintenance therapy. This study also suggests a beneficial effect of ex vivo graft manipulation on prevention of relapses post-transplantation in SLE.
authors.affiliation: Addenbrooke's Hospital, Department of Medicine, UK.
authors.forename: B
authors.initials: B
authors.lastname: Alchi
authors.forename: D
authors.initials: D
authors.lastname: Jayne
authors.forename: M
authors.initials: M
authors.lastname: Labopin
authors.forename: Alexander
authors.initials: A
authors.lastname: Demin
authors.forename: V
authors.initials: V
authors.lastname: Sergeevicheva
authors.forename: T
authors.initials: T
authors.lastname: Alexander
authors.forename: F
authors.initials: F
authors.lastname: Gualandi
authors.forename: B
authors.initials: B
authors.lastname: Gruhn
authors.forename: J
authors.initials: J
authors.lastname: Ouyang
authors.forename: P
authors.initials: P
authors.lastname: Rzepecki
authors.forename: G
authors.initials: G
authors.lastname: Held
authors.forename: A
authors.initials: A
authors.lastname: Sampol
authors.forename: J
authors.initials: J
authors.lastname: Voswinkel
authors.forename: P
authors.initials: P
authors.lastname: Ljungman
authors.forename: A
authors.initials: A
authors.lastname: Fassas
authors.forename: M
authors.initials: M
authors.lastname: Badoglio
authors.forename: R
authors.initials: R
authors.lastname: Saccardi
authors.forename: D
authors.initials: D
authors.lastname: Farge
mesh_terms: D000293:Adolescent; D000328:Adult; D005260:Female; D019538:Health Care Surveys; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000628:therapy*; D008297:Male; D008875:Middle Aged; D012042:Registries; D012189:Retrospective Studies; D055502:Secondary Prevention; D014182:Transplantation, Autologous; D055815:Young Adult