The professor of neurology at the University of Basel provided insight on a new methodology that allows depiction of patient profiles from baseline characteristics and their associations with individual outcomes. [WATCH NOW: 5 minutes]
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"We saw that cognitive deficits at baseline were predictive of further deterioration in the course of the follow-up. Cognitive assessment seems to be an important clinical sign of the clinical severity of the disease, not only on a cross-sectional level, but also if we look at the following years."
Despite the expansion of disease-modifying therapies for patients with multiple sclerosis (MS), there are still complications with treatment selection, as different clinical and demographic profiles may respond differently depending on their underlying pathophysiology. To estimate the profile of responders to treatment, investigators conducted a post hoc analysis of the phase 3 EXPAND study (NCT01665144), which evaluated the effect of siponimod (Mayzent; Novartis) in patients with secondary progressive MS.
Presented at the 2022 American Academy of Neurology (AAN) Annual Meeting, April 2-7, in Seattle, Washington, investigators adopted an innovative statistical approach combining the concept of defining responders to a therapy according to their baseline profile. The analysis generated a response score derived from baseline characteristics describing treatment effect of patients on 4 clinical end points: Expanded Disability Status Scale, Timed 25-Foot Walk, 9-Hole Peg Test, and Symbol Digit Modalities Test.
Four different responder profiles were obtained and validated, all showing a significant interaction with treatment, thus defining responders to each of the 4 outcomes. Overall, 78% (1290 of 1645) of patients were pronounced siponimod-treatment responders in at least 1 of the 4 clinical outcomes. Ludwig Kappos, MD, FEAN, FAAN, an investigator of the study, believes cognitive assessment is important as an aspect of prognosis but also as measure of treatment effect. Kappos, professor of neurology at the University of Basel, sat down with NeurologyLive® to discuss the analysis, some of the take-home findings, and whether it’s something that can and should be replicated going forward.