2268 Estimating the profile of responders to treatment: do different patients show benefits on different outcomes?

• Published in: BMJ neurology open 2022-08, Vol.4 (Suppl 1), p.A27-A27
• Main Authors: Macdonell, Richard, Bovis, Francesca, Kappos, L, Arnould, S, Goeril, K, Piani Meier, D, Sormani, MP
• Format: Article
• Language: English
• Subjects: Abstracts; Clinical outcomes
• ISSN: 2632-6140
• DOI: 10.1136/bmjno-2022-ANZAN.70

ObjectiveThis study aims to identify the baseline profile characteristic of super-responders to siponimod in the Phase 3 EXPAND study on four domains of progression (Expanded Disability Status Scale [EDSS]; upper limb function using the 9-hole peg test [9HPT]; ambulation using the timed-25-fooot walk test [T25FWT]; and cognitive processing speed using the single digit modalities test [SDMT]) using and innovative statistical approachMethodsThis is a post-hoc analysis of the phase III EXPAND trial comparing siponimod (n=1099) vs placebo (n=546) in SPMS. A response score was derived from baseline characteristics describing participants with a more pronounced treatment effect on each of the 4 clinical endpoints and evaluated optimal division into non-responders/responders according to Zhao L et al. 2013.ResultsIn the whole cohort, the effect of siponimod on time to confirmed progression for each of the 4 outcomes was:EDSS: HR=0.79, p=0.0103;9HPT: HR=0.86, p=0.23;T25FW: HR=0.95, p=0.53;SDMT: HR=0.75, p=0.001.Four different responder profiles (RSP) were obtained and validated, all showing a significant interaction with treatment, thus defining responders to each of the 4 outcomes. Overall, 1290/1645(78%) patients were pronounced siponimod-treatment responders in at least one of the 4 clinical outcomes.Conclusions78% of SPMS patients had a large treatment benefit with siponimod on at least one of the 4 clinical outcomes.