Autologous hematopoietic stem cell transplantation for severe autoimmune disease with special reference to rheumatoid arthritis

In 1996 an international collaboration began to explore the use of immunoablation and stem cell rescue in the treatment of severe autoimmune disease. Around 500 patients have been so treated according to consensus guidelines, the majority being registered in the combined European League Against Rheumatism and European Group for Blood and Marrow Transplantation (EULAR/EBMT) data registry. Results in terms of toxicity and benefit are different in the different autoimmune diseases, e.g., for rheumatoid arthritis (RA) a low transplant related mortality (TRM) of one patient out of 43 but high relapse rate (around two-thirds), whereas for systemic sclerosis (SSc) a higher TRM (12%) but less relapse. More aggressive immunoablative regimes were associated with more procedure related toxicity, but so far a clear therapeutic advantage has not been demonstrated. An overall actuarial TRM of 9% was observed. Randomized, prospective controlled phase III trials have begun in SSc and will soon commence in RA and multiple sclerosis. More phase I and II data are required for systemic lupus erythematosus and juvenile idiopathic arthritis.