• Johannes Lorscheider, Alessio Signori, Suvitha Subramaniam, Pascal Benkert, Sandra Vukusic, Maria Trojano, Jan Hillert, Anna Glaser, Robert Hyde, Tim Spelman, Melinda Magyari, Frederik Elberling, Luigi Pontieri, Nils Koch-Henriksen, Per Soelberg Sørensen, Oliver Gerlach, Alexandre Prat, Marc Girard, Sara Eichau, Pierre Grammond, Dana Horakova, Cristina Ramo-Tello, Izanne Roos, Katherine Buzzard, Jeanette Lechner Scott, José Luis Sánchez-Menoyo, Raed Alroughani, Julie Prévost, Jens Kuhle, Orla Gray, Guillaume Mathey, Laure Michel, Jonathan Ciron, Jérôme De Sèze, Elisabeth Maillart, Aurelie Ruet, Pierre Labauge, Helene Zephir, Arnaud Kwiatkowski, Anneke van der Walt, Tomas Kalincik, Helmut Butzkueven, Italian MS Register, Observatoire Français de la Sclérose en Plaques (OFSEP), MSBase Study Group, Swedish MS Registry, and Big MS Data Network.
• Department of Neurology, University Hospital Basel, Basel, Switzerland johannes.lorscheider@usb.ch.
• J. Neurol. Neurosurg. Psychiatr. 2024 Dec 6.

BackgroundEffectiveness of disease-modifying treatment (DMT) in people affected by primary progressive multiple sclerosis (PPMS) is limited. Whether specific subgroups may benefit more from DMT in a real-world setting remains unclear. Our aim was to investigate the potential effect of DMT on disability worsening among patients with PPMS stratified by different disability trajectories.MethodsWithin the framework of the Big MS Data network, we merged data from the Observatoire Français de la Sclérose en Plaques, the Swedish and Italian MS registries, and MSBase. We identified patients with PPMS that started DMT or were never treated during the observed period. Subpopulations with comparable baseline characteristics were selected by propensity score matching. Disability outcomes were analysed in time-to-recurrent event analyses, which were repeated in subclasses with different disability trajectories determined by latent class mixed models.ResultsOf the 3243 included patients, we matched 739 treated and 1330 untreated patients with a median follow-up of 3 years after pairwise censoring. No difference in the risk of confirmed disability worsening (CDW) was observed between the groups in the fully matched dataset (HR 1.11, 95% CI 0.97 to 1.23, p=0.127). However, we found a lower risk for CDW among the class of treated patients with an aggressive disability trajectory (n=360, HR 0.68, 95% CI 0.50 to 0.92, p=0.014).ConclusionsIn line with previous studies, our data suggest that DMT does not ameliorate disability worsening in PPMS, in general. However, we observed a beneficial effect of DMT on disability worsening in patients with aggressive predicted disability trajectories.© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY. Published by BMJ.

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