Will hematopoietic stem cell transplantation cure human autoimmune diseases?
Hematopoietic stem cell transplantation is becoming an accepted therapy for severe autoimmune diseases, and over 1,000 patients have been transplanted worldwide. Diseases include neurological (multiple sclerosis, others), rheumatological (systemic sclerosis, systemic lupus erythematosus, rheumatoid arthritis, vasculitis), haematological (aplastic anemia, single line immune mediated cytopenias) and others. The aim of this article is not to review the copious specific literature, but to analyze whether the up to now existing evidence satisfies the requirements of cure. Prospective randomized trials have been launched by the European Group of Blood and Marrow Transplantation (EBMT). Autologous transplantation, by far the most widely utilized because of its safety, has been shown to possess a powerful disease-arresting effect, but whether the attendant immune reconstitution ("re-education") will finally lead to cure is not demonstrated. The experience with allogeneic transplantation is too limited to draw even tentative conclusions. A Graft-versus-Autoimmunity effect has been ascertained both experimentally and clinically, but cure of autoimmunity by this procedure has not been demonstrated. Unexpected relapses in spite of full donor chimerism have been published. Further experience and studies are necessary.
Marmont
Alberto M
AM
II Division of Hematology and Stem Cell Transplantation Center, Azienda Ospedaliera-Universitaria S. Martino, Genova, Italy. alberto.marmont@hsanmartino.it
D000818:Animals; D001327:Autoimmune Diseases / Q000276:immunology / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000276:immunology / Q000502:physiology; D006801:Humans; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D014184:Transplantation, Homologous