Transplantation of hematopoietic stem cells in multiple sclerosis

Introduction and method. The idea of treating autoimmune diseases which are resistant to conventional immunodepressive treatment by autologous transplantation of hematopoietic stem cells obtained from peripheral blood (ATHS) is based on the hypothesis that immunoablative treatment would destroy the patients' anti-self lymphocytes and the reinfusion of stem cells would give rise to lymphocytes which would be tolerant of the antigens responsible for the autoimmune response. By July 2000 90 patients with multiple sclerosis (MS) had received a A THS according to data of the European Group for Blood and Marrow Transplantation/European League against Rheumatism (EBMT/EULAR). In the literature there are reports of the results obtained in 43 patients in whom MS was the sole indication for treatment -30 secondary progressive (SP), 10 primary progressive (PP) and 3 progressive-relapsing (PR) MS-. The transplant-related mortality (death within the first 100 days) was 4.6% (2143), in both cases caused by infection. In the study which included 24 patients, the ATHS was considered to have been effective, since 92% of the patients with SPMS had no further progression after 3 years. However, of the patients with PPMS only 40% scored the same or better on the EDSS, as compared to their basal scores, at the end of the study. In any case, this treatment cannot be considered to cure the MS since the probability of patients having no signs of active disease 3 years after transplantation is 12% in SPMS and 0% in PPMS. Conclusions. ATHS remains an experimental treatment. Only in the future will it be clear whether this treatment is really useful for patients with multiple sclerosis.