Reporting on behalf of the EULAR and EBMT Autoimmune Disease Stem Cell Project. High dose immunoablative therapy with autologous haemopoietic stem cell transplantation (HSCT) has been used to treat selected patients with severe autoimmune disease (AD) in over 400 cases world wide.334 transplants (327 autologous) are registered in the combined EULAR/EBMT data base, target ADs and numbers being: multiple sclerosis-102, SSc-70, RA-41, JIA-35, SLE-25, dermatomyositis-6, Bechets-3, vasculitis 6 and others. Patients were from 78 transplant centres in 22 countries with a median follow up of 16(1–81) months. Four basic conditioning regimens were used in phase I and II pilot studies, with an unmanipulated graft product in 85 cases. A transplant related mortality (TRM) of 9% overall was observed, with inter disease differences (12.5% in SSc and only one TRM in RA). Around 2/3 of patients responded with improvement or stabilisation. In SSc, 69% achieved a 25% or greater improvement in skin score, and in secondary progressive MS, 78% a 3 year progression free survival. Relapse was more common with RA and JIA, mostly being a synovitis easy to control with agents which were ineffective pre transplant. Randomised, controlled prospective trials are underway in SSc (ASTIS Trial: HSCT versus monthly pulse cyclophosphamide)), and planned for RA and MS. Trials in JIA are being discussed and further phase I/II data are required for SLE and vasculitis. These will be presented, together with immune reconstitution data, sometimes predicting relapse, and new developments in HSCT, especially non myeloablative “mini” allograft techniques. This may be applicable to full sibling matched selected patients.
Biggs M, Passweg J, Furst D, et al. PhaseI/I trial of autologous stem cell transplantation in systemic sclerosis: procedure related mortality and impact on skin disease. Ann Rheum Dis. 2001, in press
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