Autologous haematopoietic stem cell transplantation (AHSCT) is generally considered a treatment option for relapsing multiple sclerosis (MS) refractory to disease-modifying therapy (DMT). However, since its optimal placement in the MS therapeutic algorithm is still uncertain, a Focused Workshop of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), in partnership with the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party was organised in March 2022 to review current knowledge and provide recommendations on the use of AHSCT in MS and neuromyelitis optica spectrum disorders (NMOSD) [1].
They recommend that AHSCT should be considered as escalation therapy in highly active MS (frequent relapses, incomplete recovery from relapses, high frequency of new MRI lesions, rapid disability accumulation) for MS patients in whom at least one high-efficacy DMT has failed. The ideal patient profile would comprise: young age (<45 years, although biologically fit patients >55 years might be considered on an individual basis), short disease duration (<10 years), Expanded Disability Status Scale (EDSS) score <6.0, but higher scores may be acceptable due to relapse in previous months. Progressive phenotypes may be included, provided there is clinical and radiological evidence of inflammatory activity. AHSCT may be considered as first-line therapy in severe, rapidly evolving MS, within a clinical trial or research study [2].
In NMOSD, evidence is still insufficient to support AHSCT outside clinical trials, mostly due to the availability of highly effective treatments, but it could be considered as a rescue therapy in otherwise unresponsive NMOSD or as an induction therapy for aggressive disease, especially with conditioning regimens including anti-CD20 or other antibody-depleting strategies [2].
Key Points:
- AHSCT should be offered in certain MS patient profiles, generally after failure of high-efficacy DMT, before the development of irreversible disability.
- AHSCT may be considered as first-line therapy in severe, rapidly evolving MS with a poor prognosis, as part of a clinical trial or a research study, whenever possible.
- AHSCT is not recommended in late-stage MS, typically progressive, but it may be offered in early progressive disease with clear clinical and/or radiological evidence of inflammation.
- More information on the optimal positioning of AHSCT in MS management will be available from ongoing randomised clinical trials in which AHSCT is being compared with high-efficacy DMTs in relapsing-remitting MS.
- AHSCT may be considered in treatment-refractory NMOSD with adapted therapeutic protocols.
References:
[1] European Committee for Treatment and Research in Multiple Sclerosis. Highlights from the 9th ECTRIMS Focused Workshop, 2022.
ectrims.eu/app/uploads/2022/03/9th-ECTRIMS-Focused-Workshop-2022.pdf
[2] Muraro P.A., Mariottini A., Greco R. et al. Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder – recommendations from ECTRIMS and the EBMT. Nat Rev Neurol. 2025; online ahead of print. doi: 10.1038/s41582-024-01050-x. https://pubmed.ncbi.nlm.nih.gov/39814869/
Co-authors:
Paolo Preziosa, Neuroimaging Research Unit, Division of Neuroscience; Neurology Unit, IRCCS San Raffaele Scientific Institute, "Vita-Salute" San Raffaele University, Milan, Italy
Publish on behalf of the Scientific Panel on Multiple sclerosis