title: A prospective, randomized, controlled trial of autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: a position paper.
abstract: BACKGROUND
  Haematopoietic stem cell transplantation (HSCT) has been tried in the last 15 years as a therapeutic option in patients with poor-prognosis autoimmune disease who do not respond to conventional treatments. Worldwide, more than 600 patients with multiple sclerosis (MS) have been treated with HSCT, most of them having been recruited in small, single-centre, phase 1-2 uncontrolled trials. Clinical and magnetic resonance imaging outcomes from case series reports or Registry-based analyses suggest that a major response is achieved in most patients; quality and duration of response are better in patients transplanted during the relapsing-remitting phase than in those in the secondary progressive stage.
  
  
  OBJECTIVE
  An interdisciplinary group of neurologists and haematologists has been formed, following two international meetings supported by the European and American Blood and Marrow Transplantation Societies, for the purpose of discussing a controlled clinical trial, to be designed within the new scenarios of evolving MS treatments.
  
  
  CONCLUSIONS
  Objectives of the trial, patient selection, transplant technology and outcome assessment were extensively discussed. The outcome of this process is summarized in the present paper, with the goal of establishing the background and advancing the development of a prospective, randomized, controlled multicentre trial to assess the clinical efficacy of HSCT for the treatment of highly active MS.
authors.affiliation: Hematology Department, Careggi University Hospital, Florence, Italy. riccardo.saccardi@aouc.unifi.it
authors.forename: R
authors.initials: R
authors.lastname: Saccardi
authors.forename: M S
authors.initials: MS
authors.lastname: Freedman
authors.forename: M P
authors.initials: MP
authors.lastname: Sormani
authors.forename: H
authors.initials: H
authors.lastname: Atkins
authors.forename: D
authors.initials: D
authors.lastname: Farge
authors.forename: L M
authors.initials: LM
authors.lastname: Griffith
authors.forename: G
authors.initials: G
authors.lastname: Kraft
authors.forename: G L
authors.initials: GL
authors.lastname: Mancardi
authors.forename: R
authors.initials: R
authors.lastname: Nash
authors.forename: M
authors.initials: M
authors.lastname: Pasquini
authors.forename: R
authors.initials: R
authors.lastname: Martin
authors.forename: P A
authors.initials: PA
authors.lastname: Muraro
mesh_terms: D000293:Adolescent; D000328:Adult; D017326:Clinical Trials, Phase III as Topic / Q000379:methods*; D003299:Cooperative Behavior; D004185:Disability Evaluation; D005060:Europe; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D007391:International Cooperation; D008875:Middle Aged; D015337:Multicenter Studies as Topic / Q000379:methods*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000175:diagnosis / Q000601:surgery*; D011446:Prospective Studies; D016032:Randomized Controlled Trials as Topic / Q000379:methods*; D012107:Research Design; D012720:Severity of Illness Index; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D014481:United States; D055815:Young Adult