• Tomas Kalincik, Sifat Sharmin, Izanne Roos, Jennifer Massey, Ian Sutton, Barbara Withers, Mark S Freedman, Harold Atkins, Eva Krasulova, Eva Kubala Havrdova, Marek Trneny, Tomas Kozak, Joachim Burman, Richard Macdonell, Øivind Torkildsen, Lars Bø, Anne Kristine Lehmann, Basil Sharrack, and John Snowden.
• CORe, Department of Medicine, University of Melbourne, Parkville, Victoria, Australia tomas.kalincik@unimelb.edu.au.
• J. Neurol. Neurosurg. Psychiatr. 2024 Mar 27.

BackgroundNatalizumab was not shown to modify disability in progressive multiple sclerosis (MS). This matched observational study compared the effectiveness of autologous haematopoietic stem cell transplantation (AHSCT) with natalizumab in progressive MS.MethodsPatients with primary/secondary progressive MS from seven AHSCT MS centres and the MSBase registry, treated with AHSCT or natalizumab, were matched on a propensity score derived from sex, age, Expanded Disability Status Scale (EDSS), number of relapses 12/24 months before baseline, time from MS onset, the most effective prior therapy and country. The pairwise-censored groups were compared on hazards of 6-month confirmed EDSS worsening and improvement, relapses and annualised relapse rates (ARRs), using Andersen-Gill proportional hazards models and conditional negative binomial model.Results39 patients treated with AHSCT (37 with secondary progressive MS, mean age 37 years, EDSS 5.7, 28% with recent disability progression, ARR 0.54 during the preceding year) were matched with 65 patients treated with natalizumab. The study found no evidence for difference in hazards of confirmed EDSS worsening (HR 1.49, 95% CI 0.70 to 3.14) and improvement (HR 1.50, 95% CI 0.22 to 10.29) between AHSCT and natalizumab over up to 4 years. The relapse activity was also similar while treated with AHSCT and natalizumab (ARR: mean±SD 0.08±0.28 vs 0.08±0.25; HR 1.05, 95% CI 0.39 to 2.82). In the AHSCT group, 3 patients experienced febrile neutropenia during mobilisation, 9 patients experienced serum sickness, 6 patients required intensive care unit admission and 36 patients experienced complications after discharge. No treatment-related deaths were reported.ConclusionThis study does not support the use of AHSCT to control disability in progressive MS with advanced disability and low relapse activity.© Author(s) (or their employer(s)) 2024. No commercial re-use. See rights and permissions. Published by BMJ.

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