A PILOT TRIAL FOR SEVERE ,REFRACTORY SYSTEMIC AUTOIMMUNE DISEASE WITH STEM CELL TRANSPLANTATION

Objective To evaluate the feasibility, efficacy, and safety of high dose immunosuppressive therapy (HDIT) and autolo- gous hemopoietic stem cell transplantation (HSCT) with CD34+ cell selection in patients with severe, refractory autoim- mune diseases. Methods Twenty-six patients with persistent systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), primary Sjgren’s syndrome (pSS), or systemic sclerosis (SSc) who had been treated unsuccessfully with conventional treatment were enrolled in the trial in Peking Union Medical College Hospital from September 1999 to June 2004. The patients received HDIT with 200 mg/kg cyclophosphamide followed by an infusion of autologous stem cells that were CD34 selected. Disease acti- vity, adverse effect, hemopoietic and immune reconstitution, and time to recurrence of disease were monitored. Results Overall treatment related mortality was 7.7% (2/26) with 1 patient died of cytomegalovirus infection and an- other of severe pneumonia. Relapse occurred in 3 SLE patients (17.6%) in 37, 26, and 19 months posttransplantation respectively, and 1 RA patient in 15 months posttransplantation. SLE Disease Activity Index (SLEDAI) scores of SLE survivors decreased significantly (P < 0.01). RA patients recorded a drop of Disease Activity Score 28 (DAS 28). The pSS patient remained symptoms free up to now, more than 50 months after the transplantation. Conclusion HSCT can be performed relative safely in patients with severe autoimmune disease. Short-term effect of HSCT is promising. However treatment related mortality and relapse were observed in a subset of patients.

Objective To evaluate the feasibility, efficacy, and safety of high dose immunosuppressive therapy （HDIT） and autolognus hemopoietic stem cell transplantation （HSCT） with CD^34＋ cell selection in patients with severe, refractory autoim mune diseases. Methods Twenty-six patients with persistent systemic lupus erythematosus （SLE）, rheumatoid arthritis （RA）, primary Sjogren＇s syndrome （pSS）, or systemic sclerosis （SSc） who had been treated unsuccessfully with conventional treatment were enrolled in the trial in Peking Union Medical College Hospital from September 1999 to June 2004. The patients received HDIT with 200 mg/kg cyclophosphamide followed by an infusion of autologous stem cells that were CD34 selected. Disease acti- vity, adverse effect, hemopoietic and immune reconstitution, and time to recurrence of disease were monitored. Overall treatment related mortality was 7.7% （2/26） with 1 patient died of cytomegalovirus infection and an other of severe pneumonia. Relapse occurred in 3 SLE patients （17.6%） in 37, 26, and 19 months posttransplantation respectively, and 1 RA patient in 15 months posttransplantation. SLE Disease Activity Index （SLEDAI） scores of SLE survivors decreased significantly （P 〈 0.01）. RA patients recorded a drop of Disease Activity Score 28 （DAS 28）. The pSS patient remained symptoms free up to now, more than 50 months aider the transplantation. Conclusion HSCT can be performed relative safely in patients with severe autoimmune disease. Short-term effect of HSCT is promising. However treatment related mortality and relapse were observed in a subset of patients.