Guidelines for Use of Stem Cell Transplantation for Refractory MS and NMOSD Published

05/05/2025

A new consensus statement from the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the European Society for Blood and Marrow Transplantation (EBMT) provides updated guidance on the use of autologous hematopoietic stem cell transplantation (AHSCT) in individuals diagnosed with multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD). The statement was published in a recent issue of Nature Reviews Neurology

The recommendations were developed from a focused workshop involving neurologists, hematologists, and transplant experts aiming to clarify AHSCT's role in treatment algorithms. Key indications for AHSCT include aggressive relapsing–remitting MS unresponsive to high-efficacy disease-modifying therapies (DMTs) and certain cases of NMOSD.

Clinical Recommendations:

  • Patient Selection: AHSCT is advised for patients with highly active relapsing–remitting MS who have failed at least one high-efficacy DMT. Consideration is also given to treatment-naive patients with rapidly evolving severe MS. 
    Additional details regarding suitability of AHSCT for patients with progressive MS and general suitability are provided in the report, including assessments required and contraindications. 
  • NMOSD Consideration: AHSCT may be considered for NMOSD patients with severe, treatment-refractory disease, although further research is needed to establish its efficacy in this population. 
  • Multidisciplinary Approach: Implementation of AHSCT requires collaboration among neurologists, hematologists, and transplant specialists to ensure comprehensive patient care.
  • Pre- and Post-Transplant Care: Recommendations include thorough pre-transplant evaluations, including fertility counseling, and structured post-transplant rehabilitation to maximize neurological recovery.

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