title: Long-term follow-up results after autologous haematopoietic stem cell transplantation for severe systemic sclerosis.
abstract: OBJECTIVE
  Systemic sclerosis (SSc) is a generalised autoimmune disease, causing morbidity and a reduced life expectancy, especially in patients with rapidly progressive diffuse cutaneous SSc. As no proven treatment exists, autologous haematopoietic stem cell transplantation (HSCT) is employed as a new therapeutic strategy in patients with a poor prognosis. This study reports the effects on survival, skin and major organ function of HSCT in patients with severe diffuse cutaneous SSc.
  
  
  METHODS
  A total of 26 patients were evaluated. Peripheral blood stem cells were collected using cyclophosphamide (4 g/m2) and rHu G-CSF (5 to 10 microg/kg/day) and were reinfused after positive CD34+ selection. For conditioning, cyclophosphamide 200 mg/kg was used.
  
  
  RESULTS
  After a median follow-up of 5.3 (1-7.5) years, 81% (n = 21/26) of the patients demonstrated a clinically beneficial response. The Kaplan-Meier estimated survival at 5 years was 96.2% (95% CI 89-100%) and at 7 years 84.8% (95% CI 70.2-100%) and event-free survival, defined as survival without mortality, relapse or progression of SSc, resulting in major organ dysfunction was 64.3% (95% CI 47.9-86%) at 5 years and 57.1% (95% CI 39.3-83%) at 7 years.
  
  
  CONCLUSIONS
  This study confirms that autologous HSCT in selected patients with severe diffuse cutaneous SSc results in sustained improvement of skin thickening and stabilisation of organ function up to 7 years after transplantation.
authors.affiliation: Department of Rheumatology, Radboud University Nijmegen Medical Centre, P.O.Box 9101, 6500 HB Nijmegen, The Netherlands. M.Vonk@reuma.umcn.nl
authors.forename: M C
authors.initials: MC
authors.lastname: Vonk
authors.forename: Z
authors.initials: Z
authors.lastname: Marjanovic
authors.forename: F H J
authors.initials: FH
authors.lastname: van den Hoogen
authors.forename: S
authors.initials: S
authors.lastname: Zohar
authors.forename: A V M B
authors.initials: AV
authors.lastname: Schattenberg
authors.forename: W E
authors.initials: WE
authors.lastname: Fibbe
authors.forename: J
authors.initials: J
authors.lastname: Larghero
authors.forename: E
authors.initials: E
authors.lastname: Gluckman
authors.forename: F W M B
authors.initials: FW
authors.lastname: Preijers
authors.forename: A P J
authors.initials: AP
authors.lastname: van Dijk
authors.forename: J J
authors.initials: JJ
authors.lastname: Bax
authors.forename: P
authors.initials: P
authors.lastname: Roblot
authors.forename: P L C M
authors.initials: PL
authors.lastname: van Riel
authors.forename: J M
authors.initials: JM
authors.lastname: van Laar
authors.forename: D
authors.initials: D
authors.lastname: Farge
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D003520:Cyclophosphamide / Q000627:therapeutic use; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D019650:Hematopoietic Stem Cell Mobilization / Q000379:methods; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D016014:Linear Models; D008297:Male; D008875:Middle Aged; D009017:Morbidity; D019653:Myeloablative Agonists / Q000627:therapeutic use; D011994:Recombinant Proteins; D012595:Scleroderma, Systemic / Q000401:mortality / Q000628:therapy*; D015996:Survival Rate; D019172:Transplantation Conditioning / Q000379:methods; D014182:Transplantation, Autologous
chemical_list: D019653:Myeloablative Agonists; D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; D003520:Cyclophosphamide