Abstract
Multiple sclerosis (MS)is a chronic, disabling, immune-mediated, demyelinating and degenerative disease of the central nervous system. Approved disease-modifying therapies may be incompletely effective in some patients with highly active relapsing disease and high risk of disability. The use of 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. A review of recent literature identified 8 retrospective studies, 8 clinical trials, and 3 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 relapse, magnetic resonance imaging-detectable lesion activity, and worsening disability 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 transplantation physicians with experience performing AHCT for autoimmune disease is crucial for ensuring appropriate patient selection and optimizing transplantation procedures to improve patient outcomes. Transplantation 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.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 845-854 |
| Number of pages | 10 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 25 |
| Issue number | 5 |
| DOIs | |
| State | Published - May 2019 |
Funding
Financial disclosure: L.E.B. is supported by National Multiple Sclerosis Society Sylvia Lawry Physician Fellowship Award FP-1606-24540 . P.A.M. is supported by the National Institute for Health Research (NIHR) Biomedical Research Centre funding scheme. The views expressed are those of the authors and not necessarily those of the NIHR. The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the National Institute of Allergy and Infectious Diseases , National Institutes of Health, or any other agency of the US Government. Financial disclosure: L.E.B. is supported by National Multiple Sclerosis Society Sylvia Lawry Physician Fellowship Award FP-1606-24540. P.A.M. is supported by the National Institute for Health Research (NIHR) Biomedical Research Centre funding scheme. The views expressed are those of the authors and not necessarily those of the NIHR. The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the National Institute of Allergy and Infectious Diseases, National Institutes of Health, or any other agency of the US Government. Conflict of interest statement: J.A.C. has received compensation for consulting for Alkermes, Biogen, Convelo, EMD Serono, ERT, Gossamer Bio, Novartis, and ProValuate; speaking for Mylan and Synthon; and serving as an editor of Multiple Sclerosis Journal. L.E.B. has received compensation for consulting for Teva. J.D.B. has received compensation from Acorda Therapeutics, Biogen, EMD Serono, Genentech, Genzyme, Novartis, and Teva; holds stock options in Amgen; and has received research support from Acorda Therapeutics, Alexion, Alkermes, Allergan, Biogen, Genentech, Genzyme, GlaxoSmithKline, Novartis, Roche, and Sanofi-Aventis. J.R.C. has received compensation for consulting for Mylan and Novartis, speaking for Prime CME, and serving as an editor for Neurology: Clinical Practice, and has received research support from MedDay and Novartis. M.S.F. has received compensation for consulting for Actelion, Bayer Healthcare, Biogen, Celgene, Chugai, Clene Nanomedicine, EMD Canada, Genzyme, Merck Serono, Novartis, Hoffman La-Roche, PendoPharm, Sanofi-Aventis, and Teva Canada Innovation; for serving as a member of a company advisory board, board of directors, or other similar group for Actelion, BayerHealthcare, Biogen, Clene Nanomedicine, Hoffman la-Roche, Merck Serono, MedDay, Novartis, and Sanofi-Aventis; and for participating in a speaker's bureau for Sanofi-Genzyme; and has received research or educational grants from Genzyme Canada. N.S.M. has consulted for Anthem and served on advisory boards for Atara Biotherapeutics and Incyte. P.A.M. has received travel support and speaker honoraria from Bayer HealthCare, Bayer Pharma, Biogen, Merck-Serono, and Sanofi Aventis. M.C.P. has served as a consultant for Medigene and on an advisory board for Pfizer. S.S. has received a laboratory grant and consulting fees for Gilead and has served on an advisory board for Pharmacyclics. The other authors report no conflicts of interest. Financial disclosure: See Acknowledgments on page XXXX. Financial disclosure: L.E.B. is supported by National Multiple Sclerosis Society Sylvia Lawry Physician Fellowship Award FP-1606-24540. P.A.M. is supported by the National Institute for Health Research (NIHR)Biomedical Research Centre funding scheme. The views expressed are those of the authors and not necessarily those of the NIHR. The content and opinions expressed are solely the responsibility of the authors and do not represent the official policy or position of the National Institute of Allergy and Infectious Diseases, National Institutes of Health, or any other agency of the US Government. Conflict of interest statement: J.A.C. has received compensation for consulting for Alkermes, Biogen, Convelo, EMD Serono, ERT, Gossamer Bio, Novartis, and ProValuate; speaking for Mylan and Synthon; and serving as an editor of Multiple Sclerosis Journal. L.E.B. has received compensation for consulting for Teva. J.D.B. has received compensation from Acorda Therapeutics, Biogen, EMD Serono, Genentech, Genzyme, Novartis, and Teva; holds stock options in Amgen; and has received research support from Acorda Therapeutics, Alexion, Alkermes, Allergan, Biogen, Genentech, Genzyme, GlaxoSmithKline, Novartis, Roche, and Sanofi-Aventis. J.R.C. has received compensation for consulting for Mylan and Novartis, speaking for Prime CME, and serving as an editor for Neurology: Clinical Practice, and has received research support from MedDay and Novartis. M.S.F. has received compensation for consulting for Actelion, Bayer Healthcare, Biogen, Celgene, Chugai, Clene Nanomedicine, EMD Canada, Genzyme, Merck Serono, Novartis, Hoffman La-Roche, PendoPharm, Sanofi-Aventis, and Teva Canada Innovation; for serving as a member of a company advisory board, board of directors, or other similar group for Actelion, BayerHealthcare, Biogen, Clene Nanomedicine, Hoffman la-Roche, Merck Serono, MedDay, Novartis, and Sanofi-Aventis; and for participating in a speaker's bureau for Sanofi-Genzyme; and has received research or educational grants from Genzyme Canada. N.S.M. has consulted for Anthem and served on advisory boards for Atara Biotherapeutics and Incyte. P.A.M. has received travel support and speaker honoraria from Bayer HealthCare, Bayer Pharma, Biogen, Merck-Serono, and Sanofi Aventis. M.C.P. has served as a consultant for Medigene and on an advisory board for Pfizer. S.S. has received a laboratory grant and consulting fees for Gilead and has served on an advisory board for Pharmacyclics. The other authors report no conflicts of interest.
Keywords
- Autologous hematopoietic cell transplantation
- Coverage
- Indication
- Multiple sclerosis
- Stem cells
ASJC Scopus subject areas
- Hematology
- Transplantation