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Open Access 11-04-2022 | Multiple Sclerosis | Neurological Update

The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis

Authors: A. G. Willison, T. Ruck, G. Lenz, H. P. Hartung, S. G. Meuth

Published in: Journal of Neurology | Issue 7/2022

Abstract

Autologous haematopoietic stem cell transplantation (aHSCT) is gaining traction as a valuable treatment option for patients affected by severe multiple sclerosis (MS), particularly the relapsing–remitting form. We describe the current literature in terms of clinical trials, observational and retrospective studies, as well as immune reconstitution following transplantation, with a focus on the conditioning regimens used for transplantation. The evidence base predominantly consists of non-randomised, uncontrolled clinical trials or data from retrospective or observational cohorts, i.e. very few randomised or controlled trials. Most often, intermediate-intensity conditioning regimens are used, with promising results from both myeloablative and lymphoablative strategies, as well as from regimens that are low and high intensity. Efficacy of transplantation, which is likely secondary to immune reconstitution and restored immune tolerance, is, therefore, not clearly dependent on the intensity of the conditioning regimen. However, the conditioning regimen may well influence the immune response to transplantation. Heterogeneity of conditioning regimens among studies hinders synthesis of the articles assessing post-aHSCT immune system changes. Factors associated with better outcomes were lower Kurtzke Expanded Disability Status Scale, relapsing–remitting MS, younger age, and shorter disease duration at baseline, which supports the guidance for patient selection proposed by the European Society for Blood and Marrow Transplantation. Interestingly, promising outcomes were described for patients with secondary progressive MS by some studies, which may be worth taking into account when considering treatment options for patients with active, progressive disease. Of note, a significant proportion of patients develop autoimmune disease following transplantation, with alemtuzumab-containing regimens associated with the highest incidence.

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Title: The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis
Authors: A. G. Willison
T. Ruck
G. Lenz
H. P. Hartung
S. G. Meuth
Publication date: 11-04-2022
Publisher: Springer Berlin Heidelberg
Keywords: Multiple Sclerosis
Alemtuzumab
Stem Cell Transplantation
Stem Cell Transplantion
Stem Cell Transplantation
Alemtuzumab
Published in: Journal of Neurology / Issue 7/2022
Print ISSN: 0340-5354
Electronic ISSN: 1432-1459
DOI: https://doi.org/10.1007/s00415-022-11063-5

Keynote webinar | Spotlight on medication adherence

Springer Medicine

The current standing of autologous haematopoietic stem cell transplantation for the treatment of multiple sclerosis

Abstract

Keynote webinar | Spotlight on medication adherence

Springer Medicine

Abstract

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