| Abstract: | Aim: To investigate the feasibility, efficacy and safety of high‐dose immunosuppressive therapy and autologous peripheral blood stem cell transplantation (PBSCT) with CD34+ cell selection in patients with refractory and severe autoimmune diseases. Methods: Eighteen patients with persistent systemic lupus erythematosus refractory to conventional treatment were enrolled into the study of peripheral blood stem cell transplantation in Peking Union Medical College Hospital from 1999 to 2005. After mobilization and conditioning, the enriched CD34+ cells were reinfused. Disease activity, adverse effects, haematopoietic and immunologic reconstitution were monitored and followed up for at least 6 months. Results: Overall treatment‐related mortality was 5.6% with one patient dying of cytomegalovirus infection. The overall remission rate was 95.8% in the first year after PBSCT. Relapse occurred in three patients (17.6%) in 37, 26, and 19 months post‐transplantation, respectively. Disease Activity Index scores of systemic lupus erythematosus survivors were decreased significantly (P < 0.001). Conclusions: Short‐term effect of autologous peripheral blood stem cell transplantation is promising although treatment‐related mortality and relapses are observed in a subset of patients. High‐dose immunosuppressive therapy followed by autologous peripheral blood stem cell transplantation with CD34+ cell selection is feasible and relatively safe in the treatment of severe and refractory autoimmune diseases. The long‐term effect needs further evaluation and multicentre study. |
