Autologous hematopoietic cell transplantation for treatment-refractory relapsing multiple sclerosis: Position statement from the American Society for Blood and Marrow Transplantation
File(s)
Published version
Author(s)
Type
Journal Article
Abstract
Multiple sclerosis (MS) is a chronic, disabling, immune-mediated, central nervous system demyelinating and degenerative disease. Approved disease modifying therapies may be incompletely effective in some patients with highly active relapsing disease and high risk of disability. Immunoablative or myeloablative therapy followed by autologous hematopoietic cell transplantation (AHCT) has been investigated in retrospective studies, clinical trials, and meta-analyses/systematic reviews as an approach to address this unmet clinical need. On behalf of the American Society for Blood and Bone Marrow Transplantation (ASBMT), a panel of experts in AHCT and MS convened to review available evidence and make recommendations on MS as an indication for AHCT. Review of recent literature identified eight retrospective studies, eight clinical trials, and three meta-analyses/systematic reviews. In aggregate, these studies indicate that AHCT is an efficacious and safe treatment for active relapsing forms of MS to prevent clinical relapses, MRI lesion activity, and disability worsening, and to reverse disability, without unexpected adverse events. Based on the available evidence, the ASBMT recommends that treatment-refractory relapsing MS with high risk of future disability be considered a "standard of care, clinical evidence available" indication for AHCT. Collaboration of neurologists with expertise in treating MS and transplant physicians with experience performing AHCT for autoimmune disease is crucial for appropriate patient selection and optimizing transplant procedures to improve patient outcomes. Transplant centers in the United States and Canada are strongly encouraged to report baseline and outcomes data on patients receiving AHCT for multiple sclerosis to the Center for International Blood and Marrow Transplant Research.
Date Issued
2019-05
Date Acceptance
2019-02-14
Citation
Biology of Blood and Marrow Transplantation, 2019, 25 (5), pp.845-854
ISSN
1083-8791
Publisher
Elsevier
Start Page
845
End Page
854
Journal / Book Title
Biology of Blood and Marrow Transplantation
Volume
25
Issue
5
Copyright Statement
© 2019 American Society for Blood and Marrow Transplantation. This is an open access article under the CC BYNC-ND license. (http://creativecommons.org/licenses/by-nc-nd/4.0/)
Sponsor
National Institutes of Health
Identifier
https://www.ncbi.nlm.nih.gov/pubmed/30794930
PII: S1083-8791(19)30139-9
Grant Number
Clinical Trial No: ITN077AI
Subjects
autologous hematopoietic cell transplantation
coverage
indication
multiple sclerosis
stem cells
Publication Status
Published
Coverage Spatial
United States
Date Publish Online
2019-02-19