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Biol Blood Marrow Transplant. 2019 Feb 19. pii: S1083-8791(19)30139-9. doi: 10.1016/j.bbmt.2019.02.014. [Epub ahead of print]

AUTOLOGOUS HEMATOPOIETIC CELL TRANSPLANTATION FOR TREATMENT-REFRACTORY RELAPSING MULTIPLE SCLEROSIS: POSITION STATEMENT FROM THE AMERICAN SOCIETY FOR BLOOD AND MARROW TRANSPLANTATION.

Author information

1
Mellen Center for MS Treatment and Research, Cleveland Clinic, Cleveland, Ohio, USA.
2
Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
3
Multiple Sclerosis Center, Swedish Neuroscience Institute, Seattle, Washington, USA.
4
Clinical Research Division, Fred Hutchinson Cancer Research Center, Seattle, Washington.
5
Department of Neurology, University of Colorado School of Medicine, Aurora, Colorado, USA.
6
University of Ottawa and Ottawa Hospital Research Institute, Ottawa, Ontario, Canada.
7
Division of Allergy, Immunology and Transplantation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
8
Division of Blood and Marrow Transplantation, Stanford University, Stanford, California, USA.
9
Blood and Marrow Transplant Program, Cleveland Clinic, Cleveland, Ohio, USA.
10
Division of Brain Sciences, Department of Medicine, Imperial College London, London, UK.
11
Blood and Marrow Transplant Program, Colorado Blood Cancer Institute, Denver, Colorado.
12
Department of Hematology and Oncology, Medical College of Wisconsin, Milwaukee, Wisconsin and Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin.
13
Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, North Carolina, USA.
14
Division of Hematology/Oncology, Vanderbilt University Medical Center, Nashville, Tennessee, USA.
15
Blood and Marrow Transplant Program, University of Calgary, Calgary, Alberta.

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.

KEYWORDS:

autologous hematopoietic cell transplantation; coverage; indication; multiple sclerosis; stem cells

PMID:
30794930
DOI:
10.1016/j.bbmt.2019.02.014

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