Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis

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Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis. / 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.

I: BMC Medical Research Methodology, Bind 22, Nr. 1, 155, 2022.

Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › fagfællebedømt

Harvard

Lefort, M, Sharmin, S, Andersen, JB, 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, JP, Maurousset, A, Ben Nasr, H, Hankiewicz, K, Pottier, C, Maubeuge, N, Dimitri-Boulos, D, Nifle, C, Laplaud, DA, Horakova, D, Havrdova, EK, 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, PS, Hilt Christensen, CC, Rasmussen, PV, Jensen, MB, Frederiksen, JL, Bramow, S, Mathiesen, HK, Schreiber, KI, 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, bind 22, nr. 1, 155. https://doi.org/10.1186/s12874-022-01623-8

APA

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., ... 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, 22(1), [155]. https://doi.org/10.1186/s12874-022-01623-8

Vancouver

Lefort M, Sharmin S, Andersen JB, Vukusic S, Casey R, Debouverie M o.a. Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis. BMC Medical Research Methodology. 2022;22(1). 155. https://doi.org/10.1186/s12874-022-01623-8

Author

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. / Impact of methodological choices in comparative effectiveness studies : application in natalizumab versus fingolimod comparison among patients with multiple sclerosis. I: BMC Medical Research Methodology. 2022 ; Bind 22, Nr. 1.

Bibtex

@article{f5b7d650472e4dc6b507a4e83c6b7681,
title = "Impact of methodological choices in comparative effectiveness studies: application in natalizumab versus fingolimod comparison among patients with multiple sclerosis",
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.",
keywords = "Causal contrasts, Censoring, Effectiveness, Indication bias, Multiple sclerosis, Positivity assumption, Propensity score",
author = "M. Lefort and S. Sharmin and Andersen, {J. B.} and S. Vukusic and R. Casey and M. Debouverie and G. Edan and J. Ciron and A. Ruet and {De S{\`e}ze}, J. and E. Maillart and H. Zephir and P. Labauge and G. Defer and C. Lebrun-Frenay and T. Moreau and E. Berger and P. Clavelou and J. Pelletier and B. Stankoff and O. Gout and E. Thouvenot and O. Heinzlef and A. Al-Khedr and B. Bourre and O. Casez and P. Cabre and A. Montcuquet and A. Wahab and Camdessanch{\'e}, {J. P.} and A. Maurousset and {Ben Nasr}, H. and K. Hankiewicz and C. Pottier and N. Maubeuge and D. Dimitri-Boulos and C. Nifle and Laplaud, {D. A.} and D. Horakova and Havrdova, {E. K.} and R. Alroughani and G. Izquierdo and S. Eichau and S. Ozakbas and F. Patti and M. Onofrj and A. Lugaresi and M. Terzi and P. Grammond and F. Grand{\textquoteright}Maison and B. Yamout and A. Prat and M. Girard and P. Duquette and C. Boz and M. Trojano and P. McCombe and M. Slee and J. Lechner-Scott and R. Turkoglu and P. Sola and D. Ferraro and F. Granella and V. Shaygannejad and J. Prevost and D. Maimone and O. Skibina and K. Buzzard and {Van der Walt}, A. and R. Karabudak and {Van Wijmeersch}, B. and T. Csepany and D. Spitaleri and S. Vucic and N. Koch-Henriksen and F. Sellebjerg and Soerensen, {P. S.} and {Hilt Christensen}, {C. C.} and Rasmussen, {P. V.} and Jensen, {M. B.} and Frederiksen, {J. L.} and S. Bramow and Mathiesen, {H. K.} and Schreiber, {K. I.} and H. Butzkueven and M. Magyari and T. Kalincik and E. Leray",
note = "Publisher Copyright: {\textcopyright} 2022, The Author(s).",
year = "2022",
doi = "10.1186/s12874-022-01623-8",
language = "English",
volume = "22",
journal = "B M C Medical Research Methodology",
issn = "1471-2288",
publisher = "BioMed Central Ltd.",
number = "1",

}

RIS

TY - JOUR

T1 - Impact of methodological choices in comparative effectiveness studies

T2 - application in natalizumab versus fingolimod comparison among patients with multiple sclerosis

AU - Lefort, M.

AU - Sharmin, S.

AU - Andersen, J. B.

AU - Vukusic, S.

AU - Casey, R.

AU - Debouverie, M.

AU - Edan, G.

AU - Ciron, J.

AU - Ruet, A.

AU - De Sèze, J.

AU - Maillart, E.

AU - Zephir, H.

AU - Labauge, P.

AU - Defer, G.

AU - Lebrun-Frenay, C.

AU - Moreau, T.

AU - Berger, E.

AU - Clavelou, P.

AU - Pelletier, J.

AU - Stankoff, B.

AU - Gout, O.

AU - Thouvenot, E.

AU - Heinzlef, O.

AU - Al-Khedr, A.

AU - Bourre, B.

AU - Casez, O.

AU - Cabre, P.

AU - Montcuquet, A.

AU - Wahab, A.

AU - Camdessanché, J. P.

AU - Maurousset, A.

AU - Ben Nasr, H.

AU - Hankiewicz, K.

AU - Pottier, C.

AU - Maubeuge, N.

AU - Dimitri-Boulos, D.

AU - Nifle, C.

AU - Laplaud, D. A.

AU - Horakova, D.

AU - Havrdova, E. K.

AU - Alroughani, R.

AU - Izquierdo, G.

AU - Eichau, S.

AU - Ozakbas, S.

AU - Patti, F.

AU - Onofrj, M.

AU - Lugaresi, A.

AU - Terzi, M.

AU - Grammond, P.

AU - Grand’Maison, F.

AU - Yamout, B.

AU - Prat, A.

AU - Girard, M.

AU - Duquette, P.

AU - Boz, C.

AU - Trojano, M.

AU - McCombe, P.

AU - Slee, M.

AU - Lechner-Scott, J.

AU - Turkoglu, R.

AU - Sola, P.

AU - Ferraro, D.

AU - Granella, F.

AU - Shaygannejad, V.

AU - Prevost, J.

AU - Maimone, D.

AU - Skibina, O.

AU - Buzzard, K.

AU - Van der Walt, A.

AU - Karabudak, R.

AU - Van Wijmeersch, B.

AU - Csepany, T.

AU - Spitaleri, D.

AU - Vucic, S.

AU - Koch-Henriksen, N.

AU - Sellebjerg, F.

AU - Soerensen, P. S.

AU - Hilt Christensen, C. C.

AU - Rasmussen, P. V.

AU - Jensen, M. B.

AU - Frederiksen, J. L.

AU - Bramow, S.

AU - Mathiesen, H. K.

AU - Schreiber, K. I.

AU - Butzkueven, H.

AU - Magyari, M.

AU - Kalincik, T.

AU - Leray, E.

N1 - Publisher Copyright: © 2022, The Author(s).

PY - 2022

Y1 - 2022

N2 - 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.

AB - 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.

KW - Causal contrasts

KW - Censoring

KW - Effectiveness

KW - Indication bias

KW - Multiple sclerosis

KW - Positivity assumption

KW - Propensity score

U2 - 10.1186/s12874-022-01623-8

DO - 10.1186/s12874-022-01623-8

M3 - Journal article

C2 - 35637426

AN - SCOPUS:85130953245

VL - 22

JO - B M C Medical Research Methodology

JF - B M C Medical Research Methodology

SN - 1471-2288

IS - 1

M1 - 155

ER -

ID: 314449511