Autologous Hematopoietic Cell Transplantation for Treatment-Refractory Relapsing Multiple Sclerosis: Position Statement from the American Society for Blood and Marrow Transplantation

Autor: Robert Lowsky, Paolo A. Muraro, Richard A. Nash, Laura E. Baldassari, Stefanie Sarantopoulos, Mark S. Freedman, James Bowen, Marcelo C. Pasquini, Paul A. Carpenter, Keith M. Sullivan, Christopher Bredeson, Jan Storek, Bipin N. Savani, John R. Corboy, Linda M. Griffith, Harold L. Atkins, Jeffrey A. Cohen, George E. Georges, Navneet S. Majhail
Přispěvatelé: National Institutes of Health
Rok vydání: 2019
Předmět:
Canada
medicine.medical_specialty
Coverage
medicine.medical_treatment
Immunology
indication
Salvage therapy
IMMUNOABLATION
PROGRESSION
Stem cells
Hematopoietic stem cell transplantation
multiple sclerosis
Transplantation
Autologous
ECTRIMS/EAN GUIDELINE
03 medical and health sciences
0302 clinical medicine
LONG-TERM OUTCOMES
Autologous hematopoietic cell transplantation
PEOPLE
Humans
Medicine
Intensive care medicine
Adverse effect
Societies
Medical
Patient Care Team
Salvage Therapy
Transplantation
Science & Technology
business.industry
DISABILITY
Multiple sclerosis
Hematopoietic Stem Cell Transplantation
1103 Clinical Sciences
Retrospective cohort study
ADULTS
Hematology
medicine.disease
United States
Clinical trial
Treatment Outcome
Systematic review
030220 oncology & carcinogenesis
DISEASE-MODIFYING THERAPIES
DOSE IMMUNOSUPPRESSIVE THERAPY
business
Life Sciences & Biomedicine
PHARMACOLOGICAL-TREATMENT
030215 immunology
Zdroj: Biology of Blood and Marrow Transplantation. 25:845-854
ISSN: 1083-8791
Popis: 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.
Databáze: OpenAIRE
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