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Autologous hematopoietic stem cell transplantation in multiple sclerosis A meta-analysis

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Title: Autologous hematopoietic stem cell transplantation in multiple sclerosis A meta-analysis
Authors: Sormani, MP
Muraro, PA
Schiavetti, I
Signori, A
Laroni, A
Saccardi, R
Mancardi, GL
Item Type: Journal Article
Abstract: Objective: To summarize the evidence on immunoablative therapy followed by autologous hematopoietic stem cell transplantation (aHSCT) to manage severe and treatment-refractory multiple sclerosis (MS). Methods: We collected all the published studies of aHSCT in any form of MS from 1995 to 2016, carefully excluding reports that were updated in subsequent studies. Endpoints were transplant-related mortality (TRM), rate of disease progression, and no evidence of disease activity (NEDA) status. A weighted metaregression based on a Poisson model was run, assessing whether there were study-specific characteristics with an effect on TRM and progression. Results: Fifteen studies including 764 transplanted patients were pooled in the meta-analysis. The pooled estimate of TRM was 2.1% (95% confidence interval [CI] 1.3%–3.4%). TRM was higher in older studies (p = 0.014) and in studies with a lower proportion of patients with relapsing-remitting MS (RRMS) (p = 0.028). A higher baseline Expanded Disability Status Scale (p = 0.013) was also significantly associated with a higher TRM. Pooled rate of progression was 17.1% at 2 years (95% CI 9.7%–24.5%) and 23.3% (95% CI 16.3%–31.8%) at 5 years. Lower 2-year progression rate was significantly associated with higher proportions of patients with RRMS (p = 0.004). The pooled proportion of NEDA patients at 2 years was 83% (range 70%–92%) and at 5 years was 67% (range 59%–70%). Conclusions: The emerging evidence on this therapeutic approach in MS indicates that the largest benefit/risk profile form this therapeutic approach can be obtained in patients with aggressive MS with a relapsing-remitting course and who have not yet accumulated a high level of disability.
Issue Date: 28-Apr-2017
Date of Acceptance: 6-Oct-2016
URI: http://hdl.handle.net/10044/1/53339
DOI: https://dx.doi.org/10.1212/WNL.0000000000003987
ISSN: 1526-632X
Publisher: American Academy of Neurology
Start Page: 2115
End Page: 2122
Journal / Book Title: Neurology
Volume: 88
Issue: 22
Copyright Statement: © 2017 American Academy of Neurology
Keywords: Science & Technology
Life Sciences & Biomedicine
Clinical Neurology
Neurosciences & Neurology
1103 Clinical Sciences
1109 Neurosciences
1702 Cognitive Science
Neurology & Neurosurgery
Publication Status: Published
Appears in Collections:Department of Medicine (up to 2019)