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Aarhus University Hospital Neurology ... 1 Arènes UMR 6051 RSMS U 1309 Univ Renn... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Azienda Ospedaliera Di Rilievo Nazion... 1 CHU de Nantes Service de Neurologie a... 1 CHUM and Universite de Montreal Montr... 1 CISSS Chaudiere Appalache Levis Canada 1 CSSS Saint Jérôme Saint Jerome Canada 1 Central Clinical School Monash Univer... 1 Centre Des Neurosciences de Lyon UMR5... 1 Centre Hospitalier Intercommunal de P... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Sud Francilien Ser... 1 Centre Hospitalier Universitaire Dijo... 1 Centre Hospitalier Universitaire Gren... 1
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Aarhus University Hospital Neurology ... 1 Arènes UMR 6051 RSMS U 1309 Univ Renn... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Assistance Publique Des Hôpitaux de P... 1 Azienda Ospedaliera Di Rilievo Nazion... 1 CHU de Nantes Service de Neurologie a... 1 CHUM and Universite de Montreal Montr... 1 CISSS Chaudiere Appalache Levis Canada 1 CSSS Saint Jérôme Saint Jerome Canada 1 Central Clinical School Monash Univer... 1 Centre Des Neurosciences de Lyon UMR5... 1 Centre Hospitalier Intercommunal de P... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Régional Universit... 1 Centre Hospitalier Sud Francilien Ser... 1 Centre Hospitalier Universitaire Dijo... 1 Centre Hospitalier Universitaire Gren... 1
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- Lefort, M
- Sharmin, S
- Andersen, J B
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Vukusic, S
Autor Vukusic, S Service de Neurologie, Sclérose en Plaques, Pathologies de La Myéline Et Neuro-Inflammation, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, 69677, Lyon/Bron, France Centre Des Neurosciences de Lyon, UMR5292, Observatoire Français de La Sclérose en Plaques, INSERM, 1028 et CNRS, 69003, Lyon, France Université, Claude Bernard Lyon 1, Faculté de médecine Lyon Est, 69000, Lyon, France
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Casey, R
Autor Casey, R Service de Neurologie, Sclérose en Plaques, Pathologies de La Myéline Et Neuro-Inflammation, Hôpital Neurologique Pierre Wertheimer, Hospices Civils de Lyon, 69677, Lyon/Bron, France Centre Des Neurosciences de Lyon, UMR5292, Observatoire Français de La Sclérose en Plaques, INSERM, 1028 et CNRS, 69003, Lyon, France Université, Claude Bernard Lyon 1, Faculté de médecine Lyon Est, 69000, Lyon, France Eugene Devic EDMUS Foundation, 69677, Lyon/Bron, France
- Debouverie, M
- Edan, G
- Ciron, J
- Ruet, A
- De Sèze, J
BioMedCentral Open Access od 2001
Directory of Open Access Journals od 2001
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PubMed Central od 2001
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Health & Medicine (ProQuest) od 2009-01-01
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PubMed
35637426
DOI
10.1186/s12874-022-01623-8
Knihovny.cz E-zdroje
BACKGROUND: Natalizumab and fingolimod are used as high-efficacy treatments in relapsing-remitting multiple sclerosis. Several observational studies comparing these two drugs have shown variable results, using different methods to control treatment indication bias and manage censoring. The objective of this empirical study was to elucidate the impact of methods of causal inference on the results of comparative effectiveness studies. METHODS: Data from three observational multiple sclerosis registries (MSBase, the Danish MS Registry and French OFSEP registry) were combined. Four clinical outcomes were studied. Propensity scores were used to match or weigh the compared groups, allowing for estimating average treatment effect for treated or average treatment effect for the entire population. Analyses were conducted both in intention-to-treat and per-protocol frameworks. The impact of the positivity assumption was also assessed. RESULTS: Overall, 5,148 relapsing-remitting multiple sclerosis patients were included. In this well-powered sample, the 95% confidence intervals of the estimates overlapped widely. Propensity scores weighting and propensity scores matching procedures led to consistent results. Some differences were observed between average treatment effect for the entire population and average treatment effect for treated estimates. Intention-to-treat analyses were more conservative than per-protocol analyses. The most pronounced irregularities in outcomes and propensity scores were introduced by violation of the positivity assumption. CONCLUSIONS: This applied study elucidates the influence of methodological decisions on the results of comparative effectiveness studies of treatments for multiple sclerosis. According to our results, there are no material differences between conclusions obtained with propensity scores matching or propensity scores weighting given that a study is sufficiently powered, models are correctly specified and positivity assumption is fulfilled.
- MeSH
- fingolimod hydrochlorid terapeutické užití MeSH
- lidé MeSH
- natalizumab terapeutické užití MeSH
- relabující-remitující roztroušená skleróza * farmakoterapie MeSH
- roztroušená skleróza * farmakoterapie MeSH
- výsledek terapie MeSH
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- lidé MeSH
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- časopisecké články MeSH
- práce podpořená grantem MeSH
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