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In a large-scale real-world cohort, autologous hematopoietic stem cell transplantation (AHSCT) appeared to be effective and beneficial for patients with severe forms of MS. AHSCT led to overall durable remission of inflammatory activity and clinical stability, even though 38% were patients with progressive MS.

AHSCT is increasingly used to treat people with aggressive forms of MS. To establish its relative efficacy, randomized-controlled trials comparing AHSCT with high-efficacy treatments are necessary. Of such trials, four are ongoing. Currently, the main source of information on the efficacy of AHSCT are observational studies. Paolo Muraro, from the London Bridge Hospital, MD, in the UK, presented a large retrospective analysis of a cohort of patients with MS were treated with AHSCT between 2002 and 2023 at 14 UK centers¹. The results were focused on effectiveness. Outcomes used were relapse-free survival (RFS), MRI-activity-free survival (MFS), progression-free survival (PFS), no evidence of disease activity (NEDA), and confirmed improvement of the Expanded Disability Status Scale (EDSS).

The study cohort consisted of 363 patients with MS, 209 (62%) of whom had relapsing-remitting MS, and 129 (38%) progressive MS (46 had primary MS). Their median age was 40 years, median EDSS was 6.0, and median disease duration was 10 years.

Treatment-related mortality (TRM) occurred in four patients (1.1%). At 2 and 4 years after AHSCT, RFS was 94.6% and 88.6%. MFS was 88.2% and 78.8%, PFS 83.5% and 62.4%, and NEDA 72.0% and 48.5%. Prof. Muraro considered the PFS results, which are probably the most important for patients, to be good, as well as the NEDA results. He considered an even bigger success than the cumulative EDSS improvement: 24.6% at 2 years and 26.8% at 5 years. The prevalence of this improvement was 24.2% and 20.4%.

Ongoing randomized-controlled trials will further compare the efficacy of AHSCT with high-efficacy disease-modifying therapy, including for patients with relapsing-remitting MS in the STAR-MS trial (ISRCTN88667898) in the UK².

Medical writing support was provided by Michiel Tent

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