title: Autologous haematopoietic stem cell transplantation with an intermediate intensity conditioning regimen in multiple sclerosis: the Italian multi-centre experience.
abstract: BACKGROUND
  Over recent years numerous patients with severe forms of multiple sclerosis (MS) refractory to conventional therapies have been treated with intense immunosuppression followed by autologous haematopoietic stem cell transplantation (AHSCT). The clinical outcome and the toxicity of AHSCT can be diverse, depending on the various types of conditioning protocols and on the disease phase.
  
  
  OBJECTIVE
  To report the Italian experience on all the consecutive patients with MS treated with AHSCT with an intermediate intensity conditioning regimen, named BEAM/ATG, in the period from 1996 to 2008.
  
  
  METHODS
  Clinical and magnetic resonance imaging outcomes of 74 patients were collected after a median follow-up period of 48.3 (range = 0.8-126) months.
  
  
  RESULTS
  Two patients (2.7%) died from transplant-related causes. After 5 years, 66% of patients remained stable or improved. Among patients with a follow-up longer than 1 year, eight out of 25 subjects with a relapsing-remitting course (31%) had a 6-12 months confirmed Expanded Disability Status Scale improvement > 1 point after AHSCT as compared with one out of 36 (3%) patients with a secondary progressive disease course (p = 0.009). Among the 18 cases with a follow-up longer than 7 years, eight (44%) remained stable or had a sustained improvement while 10 (56%), after an initial period of stabilization or improvement with median duration of 3.5 years, showed a slow disability progression.
  
  
  CONCLUSIONS
  This study shows that AHSCT with a BEAM/ATG conditioning regimen has a sustained effect in suppressing disease progression in aggressive MS cases unresponsive to conventional therapies. It can also cause a sustained clinical improvement, especially if treated subjects are still in the relapsing-remitting phase of the disease.
authors: Department of Neuroscience, Ophthalmology and Genetics, University of Genoa, Genoa, Italy.
authors: G L
authors: GL
authors: Mancardi
authors: M P
authors: MP
authors: Sormani
authors: M
authors: M
authors: Di Gioia
authors: L
authors: L
authors: Vuolo
authors: F
authors: F
authors: Gualandi
authors: M P
authors: MP
authors: Amato
authors: E
authors: E
authors: Capello
authors: D
authors: D
authors: Currò
authors: A
authors: A
authors: Uccelli
authors: A
authors: A
authors: Bertolotto
authors: C
authors: C
authors: Gasperini
authors: A
authors: A
authors: Lugaresi
authors: E
authors: E
authors: Merelli
authors: G
authors: G
authors: Meucci
authors: L
authors: L
authors: Motti
authors: M R
authors: MR
authors: Tola
authors: E
authors: E
authors: Scarpini
authors: A M
authors: AM
authors: Repice
authors: L
authors: L
authors: Massacesi
authors: R
authors: R
authors: Saccardi
mesh_terms: D000293:Adolescent; D000328:Adult; D016009:Chi-Square Distribution; D004185:Disability Evaluation; D018450:Disease Progression; D018572:Disease-Free Survival; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality; D006801:Humans; D007558:Italy; D053208:Kaplan-Meier Estimate; D008279:Magnetic Resonance Imaging; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000175:diagnosis / Q000401:mortality / Q000601:surgery*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000175:diagnosis / Q000401:mortality / Q000601:surgery*; D011237:Predictive Value of Tests; D012042:Registries; D012720:Severity of Illness Index; D013997:Time Factors; D019172:Transplantation Conditioning / Q000009:adverse effects / Q000379:methods* / Q000401:mortality; D014182:Transplantation, Autologous; D016896:Treatment Outcome; D055815:Young Adult