title: A pilot trial for severe, refractory systemic autoimmune disease with stem cell transplantation.
abstract: OBJECTIVE
  To evaluate the feasibility, efficacy, and safety of high dose immunosuppressive therapy (HDIT) and autologous hemopoietic stem cell transplantation (HSCT) with CD34+ cell selection in patients with severe, refractory autoimmune diseases.
  
  
  METHODS
  Twenty-six patients with persistent systemic lupus erythematosus (SLE), rheumatoid arthritis (RA), primary Sjögren'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 activity, 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 another 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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Rheumatology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing 100730.
authors.forename: Xiao-Mei
authors.initials: XM
authors.lastname: Leng
authors.forename: Yan
authors.initials: Y
authors.lastname: Zhao
authors.forename: Dao-Bing
authors.initials: DB
authors.lastname: Zhou
authors.forename: Huifen
authors.initials: H
authors.lastname: Situ
authors.forename: Tai-Sheng
authors.initials: TS
authors.lastname: Li
authors.forename: Ti
authors.initials: T
authors.lastname: Shen
authors.forename: Yong-Qiang
authors.initials: YQ
authors.lastname: Zhao
authors.forename: Xiao-Feng
authors.initials: XF
authors.lastname: Zeng
authors.forename: Feng-Chun
authors.initials: FC
authors.lastname: Zhang
authors.forename: Yi
authors.initials: Y
authors.lastname: Dong
authors.forename: Fu-Lin
authors.initials: FL
authors.lastname: Tang
mesh_terms: D000293:Adolescent; D000328:Adult; D018952:Antigens, CD34 / Q000032:analysis; D001172:Arthritis, Rheumatoid / Q000276:immunology / Q000628:therapy; D001327:Autoimmune Diseases / Q000276:immunology / Q000628:therapy*; D003520:Cyclophosphamide / Q000008:administration & dosage / Q000627:therapeutic use*; D004305:Dose-Response Relationship, Drug; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage / Q000627:therapeutic use; D008180:Lupus Erythematosus, Systemic / Q000276:immunology / Q000628:therapy; D008297:Male; D010865:Pilot Projects; D012008:Recurrence; D012859:Sjogren's Syndrome / Q000276:immunology / Q000628:therapy; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous
chemical_list: D018952:Antigens, CD34; D007166:Immunosuppressive Agents; D003520:Cyclophosphamide