Autologous stem cell transplantation (ASCT) is starting to be investigated
as a potential therapy for severe refractory autoimmune disease including r
heumatological, neurological and haematological diagnoses. Increasing numbe
rs 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 agains
t Rheumatism (EULAR) to enable effective evaluation of the safety and effic
acy of this promising technique. Thus far, results have been encouraging; h
owever, more treatment-related deaths were reported in multiorgan diseases
such as scleroderma and less in diseases such as rheumatoid arthritis, whic
h confirms the importance of careful patient selection. Optimization of mob
ilization, conditioning regimen and graft manipulation is required to maxim
ize efficacy without increasing mortality and morbidity. The use of mainten
ance therapy after ASCT in order to prevent relapse needs to be explored. F
ollowing individual case reports and small cohort studies, the next step is
likely to involve multicentre randomized controlled trials.