Time to Add Autologous Stem Cell Transplant to Standard Care in RRMS?

Kelli Whitlock Burton

October 02, 2023


Three quarters of patients treated with autologous hematopoietic stem cell transplantation (aHSCT) for relapsing remitting multiple sclerosis (RRMS) were disease free 5 years after therapy and more than half reported improvement in disability, a new study shows.


  • Multicentre retrospective cohort study of 174 patients with RRMS from the Swedish MS registry who were treated with aHSCT before January 1, 2020.

  • Primary endpoints were no evidence of disease activity (NEDA) at 5 years and treatment-related mortality.

  • Secondary endpoints were NEDA at 3 and 10 years; confirmed disability worsening relapse-free survival, and MRI event-free survival at 3, 5, and 10 years; annualized relapse rate after aHSCT; proportion of patients with confirmed disability improvement; and Kurtzke Expanded Disability Status Scale change between baseline and follow-up at 1, 2, and 3 years.


  • 73% (95% CI, 63-81) had no evidence of disease at 5 years and 65% (95% CI, 57-75) at 10 years

  • Treatment-related mortality did not occurred.

  • Annualized relapse rate was 1.7 in the year before to aHSCT treatment and 0.035 during a median 5.5-year follow-up (P < .0001).

  • Of 149 patients with disability at baseline, 54% improved in disability, 37% were stable, and 9% deteriorated.

  • Mean number of adverse events (AEs) was 1.7 per patient for grade 3 events and 0.06 per patient for grade 4 events, with febrile neutropenia being the most common AE.


"Our findings demonstrate that aHSCT for RRMS is feasible within regular healthcare and can be performed without compromising safety," the authors write. "We believe that aHSCT could benefit a greater number of MS patients and should be included as a standard of care for highly active MS."


The study was led by Joachim Burman, Department of Medical Sciences, Uppsala University, Sweden. It was funded by the Center for Clinical Research Dalarna, the Marianne and Marcus Wallenberg Foundation, Region Stockholm, the Swedish Research Council, the Swedish Society for Medical Research, the Swedish Society of Medicine and Uppsala‐Örebro Regional Research Council and was published online September 25, 2023, in the Journal of Neurology Neurosurgery & Psychiatry.


There was no control group, and disease progression data may have been underreported.


Burman reported no financial conflicts. Full disclosures in the original article.

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