Autologous hematopoietic stem cell transplantation for autoimmune diseases

Bone Marrow Transplant. 2005 May;35(9):869-79. doi: 10.1038/sj.bmt.1704892.


Experimental data and early phase I/II studies suggest that high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation (HSCT) can arrest progression of severe autoimmune diseases. We have evaluated the toxicity and disease response in 473 patients with severe autoimmune disease treated with autologous HSCT between 1995 and 2003, from 110 centers participating in the European Group for Blood and Marrow Transplantation (EBMT) autoimmune disease working party database. Survival, transplant-related mortality, treatment response and disease progression were assessed. In all, 420 patients (89%; 86+/-4% at 3 years, median follow-up 20 months) were alive, 53 (11%) had died from transplant-related mortality (N=31; 7+/-3% at 3 years) or disease progression (N=22; 9+/-4% at 3 years). Of 370 patients, 299 evaluable for response (81%) showed a treatment response, which was sustained in 213 (71% of responders). Response was associated with disease (P<0.001), was better in patients who received cyclophosphamide during mobilization (relative risk (RR)3.28 (1.57-6.83)) and was worse with increasing age (>40 years, RR0.29 (0.11-0.82)). Disease progression was associated with disease (P<0.001) and conditioning intensity (high intensity, RR1; intermediate intensity, RR1.81 (0.96-3.42)); low intensity, RR2.34 (1.074-5.11)). These data from the collective EBMT experience support the hypothesis that autologous HSCT can alter disease progression in severe autoimmune disease.

Publication types

  • Clinical Trial
  • Clinical Trial, Phase III
  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Autoimmune Diseases / mortality
  • Autoimmune Diseases / therapy*
  • Bone Marrow Transplantation* / methods
  • Bone Marrow Transplantation* / mortality
  • Cyclophosphamide / administration & dosage
  • Female
  • Graft Survival
  • Hematopoietic Stem Cell Mobilization / methods
  • Humans
  • Immunosuppressive Agents / administration & dosage
  • Male
  • Peripheral Blood Stem Cell Transplantation* / methods
  • Peripheral Blood Stem Cell Transplantation* / mortality
  • Transplantation Conditioning
  • Transplantation, Autologous


  • Immunosuppressive Agents
  • Cyclophosphamide