Patients with multiple sclerosis (MS) and treated with autologous hematopoietic stem cell transplantation (AHSCT) have approximately a 50% chance of remaining free from neurologic progression for 5 years after transplant, according to a study published in  JAMA Neurology.

Paolo A Muraro, MD, from the Imperial College London in the UK, and colleagues, conducted a retrospective cohort study in patients who had AHSCT for treatment of MS between January 1995 and December 2006. The Expanded Disability Status Scale (EDSS) score at baseline, information on the administered conditioning regimen and graft manipulation, and at least 1 follow-up visit or report after transplant were also considered for eligibility criteria. Variables of interest included age, disease subtype, baseline EDSS score, number of previous disease-modifying treatments, and intensity of the conditioning regimen. Study end points included progression-free survival (survival in the absence of progression of MS), progression of MS (increase of 1 point on the EDSS scale confirmed at 12 months compared with pretreatment baseline).

The primary objective was the evaluation of the long-term outcomes in MS patients by analyzing progression-free survival, evolution of neurologic disability, overall survival, transplant-related mortality, and late effects, including new autoimmune and malignant disorders. The secondary aim was to examine the association of demographic and MS disease-related and treatment-related variables along with long-term outcomes.

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Data were obtained from 25 centers in 13 countries for 281 eligible patients. The follow-up was 6.6 years, with the last patient evaluated on July 1, 2012. A total of 218 patients (78%) had progressive forms of MS. The median EDSS score at pretreatment baseline was 6.5. The 5-year probability of progression-free survival as assessed by EDSS score was 46%. Progression-free survival in the subgroup with relapsing MS was 82% at 3 years, 78% at 4 years, and 73% at 5 years after AHSCT. Younger age and relapsing forms of MS were associated with better progression-free survival rates.

Overall survival was 93% at 5 years and 84% at 10 years after transplant. Within 100 days of transplant, 8 deaths (2.8%) occurred and were considered as transplant-related mortality. The main factors associated with neurologic progression after transplant were older age (hazard ratio [HR] 1.03), progressive vs relapsing form of MS (2.33), and more than 2 previous disease-modifying therapies (1.65). Higher baseline EDSS was associated with worse overall survival.

“In this study of patients with MS treated with AHSCT and followed up long term, almost half of them survived free from neurological progression for 5 years after transplant,” the authors said.


  1. Muraro PA, Pasquini M, Atkins HL, et al. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurol. 20 February 2017. doi:10.1001/jamaneurol.2016.5867