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Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis

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Muraro et al NRN 2017 complete accepted version for deposit.pdfAccepted version1.78 MBAdobe PDFView/Open
Title: Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis
Authors: Muraro, PA
Martin, R
Mancardi, GL
Nicholas, R
Sormani, MP
Saccardi, R
Item Type: Journal Article
Abstract: Autologous haematopoietic stem cell transplantation (AHSCT) is a multistep procedure that enables destruction of the immune system and its reconstitution from haematopoietic stem cells. Originally developed for the treatment of haematological malignancies, the procedure has been adapted for the treatment of severe immune-mediated disorders. Results from ~20 years of research make a compelling case for selective use of AHSCT in patients with highly active multiple sclerosis (MS), and for controlled trials. Immunological studies support the notion that AHSCT causes qualitative immune resetting, and have provided insight into the mechanisms that might underlie the powerful treatment effects that last well beyond recovery of immune cell numbers. Indeed, studies have demonstrated that AHSCT can entirely suppress MS disease activity for 4–5 years in 70–80% of patients, a rate that is higher than those achieved with any other therapies for MS. Treatment-related mortality, which was 3.6% in studies before 2005, has decreased to 0.3% in studies since 2005. Current evidence indicates that the patients who are most likely to benefit from and tolerate AHSCT are young, ambulatory and have inflammatory MS activity. Clinical trials are required to rigorously test the efficacy, safety and cost-effectiveness of AHSCT against highly active MS drugs.
Issue Date: 16-Jun-2017
Date of Acceptance: 1-Jun-2017
URI: http://hdl.handle.net/10044/1/50510
DOI: https://dx.doi.org/10.1038/nrneurol.2017.81
ISSN: 1759-4758
Publisher: Nature Publishing Group
Start Page: 391
End Page: 405
Journal / Book Title: Nature Reviews Neurology
Volume: 13
Issue: 7
Copyright Statement: © 2017 Macmillan Publishers Limited, part of Springer Nature. All rights reserved.
Sponsor/Funder: The UK Stem Cell Foundation
Funder's Grant Number: 944/10
Keywords: Science & Technology
Life Sciences & Biomedicine
Clinical Neurology
Neurosciences & Neurology
DOSE IMMUNOSUPPRESSIVE THERAPY
BONE-MARROW-TRANSPLANTATION
SEVERE AUTOIMMUNE-DISEASES
SINGLE-CENTER EXPERIENCE
EPSTEIN-BARR-VIRUS
WORKING PARTY
T-CELLS
EUROPEAN GROUP
CONDITIONING REGIMEN
FOLLOW-UP
Publication Status: Published
Appears in Collections:Department of Medicine (up to 2019)