Siponimod: Disentangling disability and relapses in secondary progressive multiple sclerosis
| Autor: | Christian Wolf, Ludwig Kappos, Daniela Piani Meier, Nicolas Rouyrre, Amit Bar-Or, Davorka Tomic, Patrick Vermersch, Bruce A.C. Cree, Gavin Giovannoni, Robert J. Fox, Baldur Magnusson, Göril Karlsson, Ralf Gold, Frank Dahlke |
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| Rok vydání: | 2020 |
| Předmět: |
medicine.medical_specialty
secondary progressive multiple sclerosis relapses Clinical Sciences 01 natural sciences Multiple sclerosis 010104 statistics & probability 03 medical and health sciences chemistry.chemical_compound 0302 clinical medicine Physical medicine and rehabilitation Recurrence Benzyl Compounds medicine Humans Disability progression 0101 mathematics Progressive multiple sclerosis Neurology & Neurosurgery business.industry Direct effects Neurosciences Multiple Sclerosis Chronic Progressive medicine.disease progressive multiple sclerosis Siponimod Neurology chemistry Disease Progression Secondary progressive multiple sclerosis Azetidines siponimod Neurology (clinical) progression business Original Research Papers 030217 neurology & neurosurgery |
| Zdroj: | Multiple Sclerosis (Houndmills, Basingstoke, England) Multiple sclerosis (Houndmills, Basingstoke, England), vol 27, iss 10 |
| ISSN: | 1477-0970 |
| Popis: | Background: In multiple sclerosis, impact of treatment on disability progression can be confounded if treatment also reduces relapses. Objective: To distinguish siponimod’s direct effects on disability progression from those on relapses in the EXPAND phase 3 trial. Methods: Three estimands, one based on principal stratum and two on hypothetical scenarios (no relapses, or equal relapses in both treatment arms), were defined to determine the extent to which siponimod’s effects on 3- and 6-month confirmed disability progression were independent of on-study relapses. Results: Principal stratum analysis estimated that siponimod reduced the risk of 3- and 6-month confirmed disability progression by 14%–20% and 29%–33%, respectively, compared with placebo in non-relapsing patients. In the hypothetical scenarios, risk reductions independent of relapses were 14%–18% and 23% for 3- and 6-month confirmed disability progression, respectively. Conclusion: By controlling the confounding impact of on-study relapses on confirmed disability progression, these statistical approaches provide a methodological framework to assess treatment effects on disability progression in relapsing and non-relapsing patients. The analyses support that siponimod may be useful for treating secondary progressive multiple sclerosis in patients with or without relapses. |
| Databáze: | OpenAIRE |
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