Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis
Jazyk angličtina Země Velká Británie, Anglie Médium electronic
Typ dokumentu časopisecké články, práce podpořená grantem
PubMed
35637426
PubMed Central
PMC9150358
DOI
10.1186/s12874-022-01623-8
PII: 10.1186/s12874-022-01623-8
Knihovny.cz E-zdroje
- Klíčová slova
- Causal contrasts, Censoring, Effectiveness, Indication bias, Multiple sclerosis, Positivity assumption, Propensity score,
- 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
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- Názvy látek
- fingolimod hydrochlorid MeSH
- natalizumab MeSH
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.
Aarhus University Hospital Neurology PJJ Boulevard DK 8200 Aarhus N Denmark
Arènes UMR 6051 RSMS U 1309 Univ Rennes EHESP CNRS Inserm Rennes France
Assistance Publique Des Hôpitaux de Paris Hôpital Henri Mondor Service de neurologie Créteil France
Assistance Publique Des Hôpitaux de Paris Hôpital Saint Antoine Service de neurologie Paris France
Azienda Ospedaliera Di Rilievo Nazionale San Giuseppe Moscati Avellino Avellino Italy
Central Clinical School Monash University Melbourne Australia
Centre Hospitalier de Pontoise Service de neurologie Pontoise France
Centre Hospitalier de Saint Denis Hôpital Casanova Service de neurologie Saint Denis France
Centre Hospitalier de Versailles Hôpital André Mignot Service de neurologie Le Chesnay France
Centre Hospitalier Intercommunal de Poissy Saint Germain en Laye Service de neurologie Poissy France
Centre Hospitalier Sud Francilien Service de neurologie Corbeil Essonnes France
Centre Hospitalier Universitaire d'Amiens Picardie Site sud Service de neurologie Amiens France
Centre Hospitalier Universitaire de Bordeaux Hôpital Pellegrin Service de neurologie Bordeaux France
Centre Hospitalier Universitaire de Lille Hôpital Salengro Service de neurologie D Lille France
Centre Hospitalier Universitaire de Nîmes Hôpital Carémeau Service de neurologie Nîmes France
Centre Hospitalier Universitaire de Rennes Hôpital Pontchaillou Service de neurologie Rennes France
Centre Hospitalier Universitaire Limoges Hôpital Dupuytren Service de neurologie Limoges France
CHU de Nantes Service de Neurologie and CIC015 INSERM 44093 Nantes France
CHUM and Universite de Montreal Montreal Canada
CISSS Chaudiere Appalache Levis Canada
CSSS Saint Jérôme Saint Jerome Canada
Department of Basic Medical Sciences Neuroscience and Sense Organs University of Bari Bari Italy
Department of Clinical Epidemiology Aarhus University Hospital Aarhus Aarhus Denmark
Department of Clinical Medicine University of Copenhagen Copenhagen Denmark
Department of Emergency and General Medicine Parma University Hospital Parma Italy
Department of Medicine and Surgery University of Parma Parma Italy
Department of Medicine University of Melbourne Melbourne Australia
Department of Neurology Aalborg University Hospital Multiple Sclerosis Unit Aalborg Denmark
Department of Neurology Box Hill Hospital Monash University Melbourne Australia
Department of Neurology Copenhagen University Hospital Herlev Copenhagen Denmark
Department of Neurology Faculty of Medicine University of Debrecen Debrecen Hungary
Department of Neurology John Hunter Hospital Hunter New England Health Newcastle Australia
Department of Neurology The Alfred Hospital Melbourne Australia
Department of Neurology University Hospital of Northern Sealand Copenhagen Denmark
Department of Neuroscience Azienda Ospedaliera Universitaria Modena Italy
Department of Neuroscience Imaging and Clinical Sciences University G d'Annunzio Chieti Italy
Dipartimento Di Scienze Biomediche E Neuromotorie Università Di Bologna Bologna Italy
Division of Neurology Department of Medicine Amiri Hospital Sharq Kuwait
Dokuz Eylul University Konak Izmir Turkey
Eugene Devic EDMUS Foundation 69677 Lyon Bron France
Flinders University Adelaide Australia
Fondation Adolphe de Rothschild de L'œil Et du Cerveau Service de neurologie Paris France
Garibaldi Hospital Catania Italy
GF Ingrassia Department University of Catania Catania Italy
Hacettepe University Ankara Turkey
Haydarpasa Numune Training and Research Hospital Istanbul Turkey
Hopital Notre Dame Montreal Canada
Hospital Universitario Virgen Macarena Seville Spain
INSERM CR1064 44000 Nantes France
IRCCS Istituto Delle Scienze Neurologiche Di Bologna Bologna Italy
Isfahan University of Medical Sciences Isfahan Iran
KTU Medical Faculty Farabi Hospital Trabzon Turkey
Medical Faculty 19 Mayis University Samsun Turkey
Melbourne MS Centre Department of Neurology Royal Melbourne Hospital Melbourne Australia
Monash University Melbourne Australia
Neuro Rive Sud Longueuil QC Canada
Policlinico G Rodolico Catania Italy
Rehabilitation and MS Centre Overpelt and Hasselt University Hasselt Belgium
Rouen University Hospital 76000 Rouen France
Royal Brisbane and Women's Hospital Herston Australia
School of Medicine and Public Health University Newcastle Newcastle Australia
Université Claude Bernard Lyon 1 Faculté de médecine Lyon Est 69000 Lyon France
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