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Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis
- Source :
- Lefort, M, Sharmin, S, Andersen, J B, Vukusic, S, Casey, R, Debouverie, M, Edan, G, Ciron, J, Ruet, A, De Sèze, J, Maillart, E, Zephir, H, Labauge, P, Defer, G, Lebrun-Frenay, C, Moreau, T, Berger, E, Clavelou, P, Pelletier, J, Stankoff, B, Gout, O, Thouvenot, E, Heinzlef, O, Al-Khedr, A, Bourre, B, Casez, O, Cabre, P, Montcuquet, A, Wahab, A, Camdessanché, J P, Maurousset, A, Ben Nasr, H, Hankiewicz, K, Pottier, C, Maubeuge, N, Dimitri-Boulos, D, Nifle, C, Laplaud, D A, Horakova, D, Havrdova, E K, Alroughani, R, Izquierdo, G, Eichau, S, Ozakbas, S, Patti, F, Onofrj, M, Lugaresi, A, Terzi, M, Grammond, P, Grand’Maison, F, Yamout, B, Prat, A, Girard, M, Duquette, P, Boz, C, Trojano, M, McCombe, P, Slee, M, Lechner-Scott, J, Turkoglu, R, Sola, P, Ferraro, D, Granella, F, Shaygannejad, V, Prevost, J, Maimone, D, Skibina, O, Buzzard, K, Van der Walt, A, Karabudak, R, Van Wijmeersch, B, Csepany, T, Spitaleri, D, Vucic, S, Koch-Henriksen, N, Sellebjerg, F, Soerensen, P S, Hilt Christensen, C C, Rasmussen, P V, Jensen, M B, Frederiksen, J L, Bramow, S, Mathiesen, H K, Schreiber, K I, Butzkueven, H, Magyari, M, Kalincik, T & Leray, E 2022, ' Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis ', BMC Medical Research Methodology, vol. 22, no. 1, 155 . https://doi.org/10.1186/s12874-022-01623-8, BMC Medical Research Methodology, BMC Medical Research Methodology, 2022, 22 (1), pp.155. ⟨10.1186/s12874-022-01623-8⟩, BMC Medical Research Methodology, BioMed Central, 2022, 22 (1), pp.155. ⟨10.1186/s12874-022-01623-8⟩, Lefort, M, Sharmin, S, Andersen, J B, Vukusic, S, Casey, R, Debouverie, M, Edan, G, Ciron, J, Ruet, A, De Sèze, J, Maillart, E, Zephir, H, Labauge, P, Defer, G, Lebrun-Frenay, C, Moreau, T, Berger, E, Clavelou, P, Pelletier, J, Stankoff, B, Gout, O, Thouvenot, E, Heinzlef, O, Al-Khedr, A, Bourre, B, Casez, O, Cabre, P, Montcuquet, A, Wahab, A, Camdessanché, J P, Maurousset, A, Ben Nasr, H, Hankiewicz, K, Pottier, C, Maubeuge, N, Dimitri-Boulos, D, Nifle, C, Laplaud, D A, Horakova, D, Havrdova, E K, Alroughani, R, Izquierdo, G, Eichau, S, Ozakbas, S, Patti, F, Onofrj, M, Lugaresi, A, Terzi, M, Grammond, P, Grand'Maison, F, Yamout, B, Prat, A, Girard, M, Duquette, P, Boz, C, Trojano, M, McCombe, P, Slee, M, Lechner-Scott, J, Turkoglu, R, Sola, P, Ferraro, D, Granella, F, Shaygannejad, V, Prevost, J, Maimone, D, Skibina, O, Buzzard, K, Van der Walt, A, Karabudak, R, Van Wijmeersch, B, Csepany, T, Spitaleri, D, Vucic, S, Koch-Henriksen, N, Sellebjerg, F, Soerensen, P S, Hilt Christensen, C C, Rasmussen, P V, Jensen, M B, Frederiksen, J L, Bramow, S, Mathiesen, H K, Schreiber, K I, Butzkueven, H, Magyari, M, Kalincik, T & Leray, E 2022, ' Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis ', BMC Medical Research Methodology, vol. 22, no. 1, 155 . https://doi.org/10.1186/s12874-022-01623-8
- Publication Year :
- 2022
-
Abstract
- 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. OFSEP was supported by a grant provided by the French State and handled by the "Agence Nationale de la Recherche", within the framework of the "Investments for the Future" program, under the reference ANR10-COHO-002, by the Eugène Devic EDMUS Foundation against multiple sclerosis and by the ARSEP Foundation. ML has recieved travel grant from ARSEP foundation for this project. The Clinical Outcomes Research unit at the University of Melbourne received funding from NHMRC (grant number
- Subjects :
- MESH: Fingolimod Hydrochloride
Indication bias
Multiple Sclerosis
MESH: Multiple Sclerosis, Relapsing-Remitting
Propensity score
Epidemiology
Positivity assumption
[SDV]Life Sciences [q-bio]
Health Informatics
Effectiveness
Multiple sclerosis
Multiple Sclerosis, Relapsing-Remitting
Multiple Sclerosi
Humans
Fingolimod Hydrochloride/therapeutic use
MESH: Treatment Outcome
Censoring
Natalizumab/therapeutic use
MESH: Humans
Fingolimod Hydrochloride
Natalizumab
Effectivene
Causal contrasts
MESH: Natalizumab
MESH: Multiple Sclerosis
Multiple Sclerosis, Relapsing-Remitting/drug therapy
Causal contrast
Treatment Outcome
Multiple Sclerosis/drug therapy
Indication bia
Human
Subjects
Details
- Language :
- English
- ISSN :
- 14712288
- Database :
- OpenAIRE
- Journal :
- Lefort, M, Sharmin, S, Andersen, J B, Vukusic, S, Casey, R, Debouverie, M, Edan, G, Ciron, J, Ruet, A, De Sèze, J, Maillart, E, Zephir, H, Labauge, P, Defer, G, Lebrun-Frenay, C, Moreau, T, Berger, E, Clavelou, P, Pelletier, J, Stankoff, B, Gout, O, Thouvenot, E, Heinzlef, O, Al-Khedr, A, Bourre, B, Casez, O, Cabre, P, Montcuquet, A, Wahab, A, Camdessanché, J P, Maurousset, A, Ben Nasr, H, Hankiewicz, K, Pottier, C, Maubeuge, N, Dimitri-Boulos, D, Nifle, C, Laplaud, D A, Horakova, D, Havrdova, E K, Alroughani, R, Izquierdo, G, Eichau, S, Ozakbas, S, Patti, F, Onofrj, M, Lugaresi, A, Terzi, M, Grammond, P, Grand’Maison, F, Yamout, B, Prat, A, Girard, M, Duquette, P, Boz, C, Trojano, M, McCombe, P, Slee, M, Lechner-Scott, J, Turkoglu, R, Sola, P, Ferraro, D, Granella, F, Shaygannejad, V, Prevost, J, Maimone, D, Skibina, O, Buzzard, K, Van der Walt, A, Karabudak, R, Van Wijmeersch, B, Csepany, T, Spitaleri, D, Vucic, S, Koch-Henriksen, N, Sellebjerg, F, Soerensen, P S, Hilt Christensen, C C, Rasmussen, P V, Jensen, M B, Frederiksen, J L, Bramow, S, Mathiesen, H K, Schreiber, K I, Butzkueven, H, Magyari, M, Kalincik, T & Leray, E 2022, ' Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis ', BMC Medical Research Methodology, vol. 22, no. 1, 155 . https://doi.org/10.1186/s12874-022-01623-8, BMC Medical Research Methodology, BMC Medical Research Methodology, 2022, 22 (1), pp.155. ⟨10.1186/s12874-022-01623-8⟩, BMC Medical Research Methodology, BioMed Central, 2022, 22 (1), pp.155. ⟨10.1186/s12874-022-01623-8⟩, Lefort, M, Sharmin, S, Andersen, J B, Vukusic, S, Casey, R, Debouverie, M, Edan, G, Ciron, J, Ruet, A, De Sèze, J, Maillart, E, Zephir, H, Labauge, P, Defer, G, Lebrun-Frenay, C, Moreau, T, Berger, E, Clavelou, P, Pelletier, J, Stankoff, B, Gout, O, Thouvenot, E, Heinzlef, O, Al-Khedr, A, Bourre, B, Casez, O, Cabre, P, Montcuquet, A, Wahab, A, Camdessanché, J P, Maurousset, A, Ben Nasr, H, Hankiewicz, K, Pottier, C, Maubeuge, N, Dimitri-Boulos, D, Nifle, C, Laplaud, D A, Horakova, D, Havrdova, E K, Alroughani, R, Izquierdo, G, Eichau, S, Ozakbas, S, Patti, F, Onofrj, M, Lugaresi, A, Terzi, M, Grammond, P, Grand'Maison, F, Yamout, B, Prat, A, Girard, M, Duquette, P, Boz, C, Trojano, M, McCombe, P, Slee, M, Lechner-Scott, J, Turkoglu, R, Sola, P, Ferraro, D, Granella, F, Shaygannejad, V, Prevost, J, Maimone, D, Skibina, O, Buzzard, K, Van der Walt, A, Karabudak, R, Van Wijmeersch, B, Csepany, T, Spitaleri, D, Vucic, S, Koch-Henriksen, N, Sellebjerg, F, Soerensen, P S, Hilt Christensen, C C, Rasmussen, P V, Jensen, M B, Frederiksen, J L, Bramow, S, Mathiesen, H K, Schreiber, K I, Butzkueven, H, Magyari, M, Kalincik, T & Leray, E 2022, ' Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis ', BMC Medical Research Methodology, vol. 22, no. 1, 155 . https://doi.org/10.1186/s12874-022-01623-8
- Accession number :
- edsair.doi.dedup.....b76c9aa3b89340f07462e048e392906e
- Full Text :
- https://doi.org/10.1186/s12874-022-01623-8