Long-term Outcomes of Autologous Peripheral Blood Stem Cell Transplantation for Refractory Rheumatic Diseases

2017 
Objective. We investigated the long-term outcomes of autologous peripheral blood stem cell transplantation (PBSCT) to treat refractory rheumatic diseases. Methods. Patients who underwent PBSCT for refractory rheumatic diseases at our institution be-tween 2002 and 2005 were assessed for outcomes including treatment response, adverse events, damage accrual, and survival at 6 months and last follow-up. Results. Eleven patients, including six with systemic lupus erythematosus (SLE), four with sys-temic sclerosis (SSc), and one with Still`s disease were treated with PBSCT. In SLE patients, two showed complete response, two partial response, and two expired. One patient who expired responded completely two months after transplantation but dis-continued treatment by choice and expired at six months due to an SLE flare. Long-term, two patients went into remission with-out organ damage, one patient went into remission with organ damage, and one had low disease activity with organ damage. Of the four patients with SSc, two showed a complete response, one a partial response, and there was one trans-plantation- related death at six months. At the last record notation, two remained in remission without relapse and one was lost to follow-up. The Still`s disease patient partially responded at six months and was in remission at the last record notation. Conclusion. The ten-year survival rate was 70% with a 40% recurrence rate and 20% treatment-related mortality rate. (J Rheum Dis 2017;24:149-156)
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