title: Autologous hematopoietic stem cell transplantation for progressive multiple sclerosis: report of efficacy and safety at three yr of follow up in 21 patients.
abstract: OBJECTIVE
  To observe the efficacy and toxicity of autologous hematopoietic stem cell transplantation (HSCT) in progressive multiple sclerosis (PMS).
  
  
  METHODS
  Twenty-one patients with PMS were treated with autologous HSCT. Stem cells were mobilized with cyclophosphamide (CY) and granulocyte colony-stimulating factor. After conditioning regimen of CY and total body irradiation or BEAM, stem cells were reinfused. CD34+ cell selection of the graft was performed and anti-thymocyte globulin was given for T-cell depletion. The probabilities of confirmed progression-free survival and disease activity-free survival were used to assess the efficacy and the adverse experiences were recorded to detect the toxicities.
  
  
  RESULTS
  The median follow-up time was 42 (6-65) months. The probabilities of confirmed progression-free survival and the disease activity-free survival were 75% and 33.3%, respectively. The principal adverse events included allergy, infection, elevation of liver enzymes, transient neurologic deterioration and depression. Two patients died of severe pneumonia and varicella-zoster virus hepatitis, at 4.5 and 15 months post-transplant, respectively.
  
  
  CONCLUSIONS
  Autologous HSCT seems beneficial to PMS. However, more patients and longer follow up would be required to assess the risk/benefit ratio.
authors.affiliation: Department of Neurology, Shanghai First People's Hospital, Shanghai Jiao Tong University, Shanghai, China. drnixiushi@sina.com
authors.forename: Xiu-Shi
authors.initials: XS
authors.lastname: Ni
authors.forename: Jiang
authors.initials: J
authors.lastname: Ouyang
authors.forename: Wen-Hui
authors.initials: WH
authors.lastname: Zhu
authors.forename: Chong
authors.initials: C
authors.lastname: Wang
authors.forename: Bing
authors.initials: B
authors.lastname: Chen
mesh_terms: D018450:Disease Progression; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008279:Magnetic Resonance Imaging; D009103:Multiple Sclerosis / Q000628:therapy*; D012449:Safety; D013997:Time Factors; D014182:Transplantation, Autologous / Q000379:methods*; D016896:Treatment Outcome