Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder — recommendations from ECTRIMS and the EBMT
File(s)
Accepted version
Author(s)
Type
Journal Article
Abstract
Autologous haematopoietic stem cell transplantation (AHSCT) is a treatment option for relapsing forms of multiple sclerosis (MS) that are refractory to disease-modifying therapy (DMT). AHSCT after failure of high-efficacy DMT in aggressive forms of relapsing–remitting MS is a generally accepted indication, yet the optimal placement of this approach in the treatment sequence is not universally agreed upon. Uncertainties also remain with respect to other indications, such as in rapidly evolving, severe, treatment-naive MS, progressive MS, and neuromyelitis optica spectrum disorder (NMOSD). Furthermore, treatment and monitoring protocols, rehabilitation and other supportive care before and after AHSCT need to be optimized. To address these issues, we convened a European Committee for Treatment and Research in Multiple Sclerosis Focused Workshop in partnership with the European Society for Blood and Marrow Transplantation Autoimmune Diseases Working Party, in which evidence and key questions were presented and discussed by experts in these diseases and in AHSCT. Based on the workshop output and subsequent written interactions, this Consensus Statement provides practical guidance and recommendations on the use of AHSCT in MS and NMOSD. Recommendations are based on the available evidence, or on consensus when evidence was insufficient. We summarize the key evidence, report the final recommendations, and identify areas for further research.
Date Issued
2025-03-01
Date Acceptance
2024-12-11
Citation
Nature Reviews Neurology, 2025, 21 (3), pp.140-158
ISSN
1759-4766
Publisher
Nature Research
Start Page
140
End Page
158
Journal / Book Title
Nature Reviews Neurology
Volume
21
Issue
3
Copyright Statement
Copyright © 2025 Springer Nature. This is the author’s accepted manuscript made available under a CC-BY licence in accordance with Imperial’s Research Publications Open Access policy (www.imperial.ac.uk/oa-policy)
License URL
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/39814869
PII: 10.1038/s41582-024-01050-x
Subjects
Humans
Neuromyelitis Optica
Hematopoietic Stem Cell Transplantation
Multiple Sclerosis
Transplantation, Autologous
Publication Status
Published
Coverage Spatial
England
Date Publish Online
2025-01-15