Autologous blood stem cell transplantation as therapy for autoimmune diseases

Autologous stem cell transplantation (ASCT) is starting to be investigated as a potential therapy for severe refractory autoimmune disease including rheumatological, neurological and haematological diagnoses. Increasing numbers of cases are now reported in the literature. Data from all transplanted patients are being collated in a centralized register by the European Group for Blood and Marrow Transplantation (EBMT) and the European League against Rheumatism (EULAR) to enable effective evaluation of the safety and efficacy of this promising technique. Thus far, results have been encouraging; however, more treatment-related deaths were reported in multiorgan diseases such as scleroderma and less in diseases such as rheumatoid arthritis, which confirms the importance of careful patient selection. Optimization of mobilization, conditioning regimen and graft manipulation is required to maximize efficacy without increasing mortality and morbidity. The use of maintenance therapy after ASCT in order to prevent relapse needs to be explored. Following individual case reports and small cohort studies, the next step is likely to involve multicentre randomized controlled trials.