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Emulating randomised clinical trials in relapsing-remitting multiple sclerosis with non-randomised real-world evidence: an application using data from the MSBase Registry
A. Signori, M. Ponzano, T. Kalincik, S. Ozakbas, D. Horakova, E. Kubala Havrdova, R. Alroughani, F. Patti, J. Kuhle, G. Izquierdo, S. Eichau, B. Yamout, SJ. Khoury, R. Karabudak, P. Grammond, P. Duquette, I. Roos, H. Butzkueven, A. van der Walt,...
Jazyk angličtina Země Anglie, Velká Británie
Typ dokumentu časopisecké články
NLK
ProQuest Central
od 1944-07-01 do Před 6 měsíci
Nursing & Allied Health Database (ProQuest)
od 1944-07-01 do Před 6 měsíci
Health & Medicine (ProQuest)
od 1944-07-01 do Před 6 měsíci
Psychology Database (ProQuest)
od 1944-07-01 do Před 6 měsíci
PubMed
38242680
DOI
10.1136/jnnp-2023-332603
Knihovny.cz E-zdroje
- MeSH
- dospělí MeSH
- fingolimod hydrochlorid * terapeutické užití MeSH
- imunosupresiva terapeutické užití MeSH
- interferon beta 1a * terapeutické užití MeSH
- lidé středního věku MeSH
- lidé MeSH
- randomizované kontrolované studie jako téma * MeSH
- registrace * MeSH
- relabující-remitující roztroušená skleróza * farmakoterapie MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mužské pohlaví MeSH
- ženské pohlaví MeSH
- Publikační typ
- časopisecké články MeSH
BACKGROUND: To mimic as closely as possible a randomised controlled trial (RCT) and calibrate the real-world evidence (RWE) studies against a known treatment effect would be helpful to understand if RWE can support causal conclusions in selected circumstances. The aim was to emulate the TRANSFORMS trial comparing Fingolimod (FTY) versus intramuscular interferon β-1a (IFN) using observational data. METHODS: We extracted from the MSBase registry all the patients with relapsing-remitting multiple sclerosis (RRMS) collected in the period 2011-2021 who received IFN or FTY (0.5 mg) and with the same inclusion and exclusion criteria of the TRANSFORMS RCT. The primary endpoint was the annualised relapse rate (ARR) over 12 months. Patients were 1:1 propensity-score (PS) matched. Relapse-rate ratio (RR) was calculated by mean of a negative binomial regression. RESULTS: A total of 4376 patients with RRMS (1140 in IFN and 3236 in FTY) were selected. After PS, 856 patients in each group were matched. The ARR was 0.45 in IFN and 0.25 in FTY with a significant difference between the two groups (RR: 0.55, 95% CI: 0.45 to 0.68; p<0.001). The result of the emulation was very similar and fell within the 95% CI of that observed in the RCT (RR: 0.49, 95% CI: 0.37 to 0.64; p<0.001) with a standardised difference of 0.66 (p=0.51). CONCLUSIONS: By applying the same inclusion and exclusion criteria used in the RCT and employing appropriate methodology, we successfully replicated the RCT results with only minor discrepancies. Also, even if the confounding bias cannot be fully eliminated, conducting a rigorous target trial emulation could still yield valuable insights for comparative effectiveness research.
Alfred Hospital Melbourne Victoria Australia
American University of Beirut Beirut Lebanon
American University of Beirut Medical Center Beirut Lebanon
Amiri Hospital Kuwait City Kuwait
CHUM MS Center and Department of Neuroscience Université de Montréal Montreal Québec Canada
Department of Health Sciences Section of Biostatistics University of Genova Genoa Italy
Department of Medicine University of Melbourne Melbourne Victoria Australia
Department of Neurology Hacettepe University Faculty of Medicine Ankara Turkey
Department of Neurology Royal Melbourne Hospital Melbourne Victoria Australia
Department of Neurology University Hospital Basel Basel Switzerland
Dokuz Eylul University İzmir Turkey
Hospital Universitario Virgen Macarena Seville Spain
Hotel Dieu de Levis Levis Quebec Canada
Istituto di Ricovero e Cura a Carattere Scientifico Ospedale Policlinico San Martino Genoa Italy
Monash University Central Clinical School Melbourne Victoria Australia
Citace poskytuje Crossref.org
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