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

Abstract

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 between 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 systemic 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 discontinued treatment by choice and expired at six months due to an SLE flare. Long-term, two patients went into remission without 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 transplantation-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.