title: [Treatment of severe systemic autoimmune diseases with autologous peripheral blood stem cell transplantation].
abstract: OBJECTIVE
  To investigate the feasibility, efficacy and safety of high dose immunosuppressive therapy (HDIT) and autologous peripheral blood stem cell transplantation (PBSCT) with CD(34)(+) cell selection in patients with refractory and severe autoimmune diseases.
  
  
  METHODS
  Twenty-one patients with SLE, RA, pSS, SSc or MCTD were enrolled in the study from 1999. Autologous haemopoietic stem cells were mobilized with CTX 3 approximately 4 g/m(2) and granulocyte colony stimulating factor (G-CSF). CD(34)(+) cells were selected by CliniMACS. After conditioning with CTX (200 mg/kg) and pig antithymocyte globulin (ATG, 90 mg/kg) or CTX (150 mg/kg) and total body irradiation (TBI, 4 approximately 6 Gy), the enriched CD(34)(+) cells were reinfused.
  
  
  RESULTS
  All patients completed the mobilization and leukapheresis procedures successfully, and proceeded to receive conditioning and transplantation. Two patients died of complication related to transplantation, one is CMV infection, the other is severe pneumonia during the course of granulocyte deficiency. A MCTD patient completed the stem cell mobilization and died of severe pulmonary hypertension and heart failure before CD(34)(+) cells reinfusing. Two SLE patients relapsed in 26, 37 months respectively and a RA patient relapsed in 15 months after transplantation. Other patients got improved, with SLE-DAI score decreasing from 17 to 4 score and proteinuria decreasing from 6.7 g to 2.3 g in SLE patients; DAS28 score from 7.9 to 2.1 in RA patient; Symptom improved and lab results recovered in SS.
  
  
  CONCLUSIONS
  High dose immunosuppressive therapy followed by autologous peripheral blood stem cell transplantation with CD(34)(+) cell selection is feasible and relative safe. Patients remain free from disease active and improved continuously. Some patients could relapse after transplantation. Long-term effect need to be further observed.
authors.affiliation: Department of Rheumatology, Peking Union Medical College Hospital, Beijing 100730, China.
authors.forename: Yan
authors.initials: Y
authors.lastname: Zhao
authors.forename: Dao-bin
authors.initials: DB
authors.lastname: Zhou
authors.forename: Xiao-mei
authors.initials: XM
authors.lastname: Leng
authors.forename: Shu-jie
authors.initials: SJ
authors.lastname: Wang
authors.forename: Tai-sheng
authors.initials: TS
authors.lastname: Li
authors.forename: Yun
authors.initials: Y
authors.lastname: Duan
authors.forename: Ti
authors.initials: T
authors.lastname: Shen
authors.forename: Yong-qiang
authors.initials: YQ
authors.lastname: Zhao
authors.forename: Jie-ping
authors.initials: JP
authors.lastname: Zhang
authors.forename: Lian-jun
authors.initials: LJ
authors.lastname: Bai
authors.forename: Wei
authors.initials: W
authors.lastname: Cui
authors.forename: Fu-quan
authors.initials: FQ
authors.lastname: Zhang
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: D001172:Arthritis, Rheumatoid / Q000628:therapy; D001327:Autoimmune Diseases / Q000628:therapy*; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000628:therapy*; D008297:Male; D036102:Peripheral Blood Stem Cell Transplantation; D019172:Transplantation Conditioning; D014182:Transplantation, Autologous; D016896:Treatment Outcome