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Comparative effectiveness in multiple sclerosis: A methodological comparison
I. Roos, I. Diouf, S. Sharmin, D. Horakova, EK. Havrdova, F. Patti, V. Shaygannejad, S. Ozakbas, G. Izquierdo, S. Eichau, M. Onofrj, A. Lugaresi, R. Alroughani, A. Prat, M. Girard, P. Duquette, M. Terzi, C. Boz, F. Grand'Maison, P. Sola, D....
Language English Country England, Great Britain
Document type Journal Article, Research Support, Non-U.S. Gov't
- MeSH
- Fingolimod Hydrochloride therapeutic use MeSH
- Immunologic Factors therapeutic use MeSH
- Immunosuppressive Agents therapeutic use MeSH
- Humans MeSH
- Natalizumab therapeutic use MeSH
- Recurrence MeSH
- Multiple Sclerosis, Relapsing-Remitting * drug therapy MeSH
- Multiple Sclerosis * drug therapy MeSH
- Propensity Score MeSH
- Treatment Outcome MeSH
- Check Tag
- Humans MeSH
- Publication type
- Journal Article MeSH
- Research Support, Non-U.S. Gov't MeSH
BACKGROUND: In the absence of evidence from randomised controlled trials, observational data can be used to emulate clinical trials and guide clinical decisions. Observational studies are, however, susceptible to confounding and bias. Among the used techniques to reduce indication bias are propensity score matching and marginal structural models. OBJECTIVE: To use the comparative effectiveness of fingolimod vs natalizumab to compare the results obtained with propensity score matching and marginal structural models. METHODS: Patients with clinically isolated syndrome or relapsing remitting MS who were treated with either fingolimod or natalizumab were identified in the MSBase registry. Patients were propensity score matched, and inverse probability of treatment weighted at six monthly intervals, using the following variables: age, sex, disability, MS duration, MS course, prior relapses, and prior therapies. Studied outcomes were cumulative hazard of relapse, disability accumulation, and disability improvement. RESULTS: 4608 patients (1659 natalizumab, 2949 fingolimod) fulfilled inclusion criteria, and were propensity score matched or repeatedly reweighed with marginal structural models. Natalizumab treatment was associated with a lower probability of relapse (PS matching: HR 0.67 [95% CI 0.62-0.80]; marginal structural model: 0.71 [0.62-0.80]), and higher probability of disability improvement (PS matching: 1.21 [1.02 -1.43]; marginal structural model 1.43 1.19 -1.72]). There was no evidence of a difference in the magnitude of effect between the two methods. CONCLUSIONS: The relative effectiveness of two therapies can be efficiently compared by either marginal structural models or propensity score matching when applied in clearly defined clinical contexts and in sufficiently powered cohorts.
Aarhus University Hospital Aarhus Denmark
Azienda Ospedaliera di Rilievo Nazionale San Giuseppe Moscati Avellino Avellino Italy
Bakirkoy Education and Research Hospital for Psychiatric and Neurological Diseases Istanbul Turkey
Brain and Mind Centre Sydney NSW Australia
Center of Neuroimmunology Service of Neurology Hospital Clinic de Barcelona Barcelona Spain
Centro Sclerosi Multipla UOC Neurologia ARNAS Garibaldi Catania Italy
CHUM MS Center and Universite de Montreal Montreal QC Canada
CISSS Chaudière Appalache Levis QC Canada
CORe Department of Medicine University of Melbourne Melbourne VIC Australia
CSSS Saint Jérôme Saint Jerome QC Canada
Department of Medicine Sultan Qaboos University Hospital Al Khodh Oman
Department of Neurology Centro Hospitalar Universitario de Sao Joao Porto Portugal
Department of Neurology Faculty of Medicine University of Debrecen Debrecen Hungary
Department of Neurology Razi Hospital Manouba Tunisia
Department of Neurology University Hospital Ghent Ghent Belgium
Department of Neuroscience Azienda Ospedaliera Universitaria Modena Italy
Division of Neurology Department of Medicine Amiri Hospital Sharq Kuwait
Dokuz Eylul University İzmir Turkey
Geelong Hospital Geelong VIC Australia
Groene Hart Ziekenhuis Gouda The Netherlands
Hacettepe University Ankara Turkey
Haydarpasa Numune Training and Research Hospital Istanbul Turkey
Hospital de Galdakao Usansolo Galdakao Spain
Hospital Fernandez Capital Federal Buenos Aires Argentina
Hospital General Universitario de Alicante Alicante Spain
Hospital Germans Trias i Pujol Badalona Spain
Hospital Universitario Donostia and IIS Biodonostia San Sebastián Spain
Hospital Universitario Virgen Macarena Sevilla Spain
Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán Mexico City Mexico
IRCCS Mondino Foundation Pavia Italy
Isfahan University of Medical Sciences Isfahan Iran
Jewish General Hospital Montreal QC Canada
KTU Medical Faculty Farabi Hospital Trabzon Turkey
Mayis University Samsun Turkey
Nemocnice Jihlava Jihlava Czech Republic
Neuro Rive Sud Greenfield Park QC Canada
Neurology Department King Fahad Specialist Hospital Dammam Khobar Saudi Arabia
Royal Victoria Hospital Belfast UK
South Eastern HSC Trust Belfast UK
St Vincent's University Hospital Dublin Ireland
Universidade Metropolitana de Santos Santos Brazil
University G d'Annunzio Chieti Italy
University Hospital Reina Sofia Cordoba Spain
UOC Neurologia Azienda Sanitaria Unica Regionale Marche AV3 Macerata Italy
References provided by Crossref.org
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- $a Roos, Izanne $u CORe, Department of Medicine, University of Melbourne, Melbourne, VIC, Australia/Neuroimmunology Centre, Department of Neurology, Royal Melbourne Hospital, Melbourne, VIC, Australia $1 https://orcid.org/0000000303713666
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- $a Comparative effectiveness in multiple sclerosis: A methodological comparison / $c I. Roos, I. Diouf, S. Sharmin, D. Horakova, EK. Havrdova, F. Patti, V. Shaygannejad, S. Ozakbas, G. Izquierdo, S. Eichau, M. Onofrj, A. Lugaresi, R. Alroughani, A. Prat, M. Girard, P. Duquette, M. Terzi, C. Boz, F. Grand'Maison, P. Sola, D. Ferraro, P. Grammond, R. Turkoglu, K. Buzzard, O. Skibina, B. Yamou, A. Altintas, O. Gerlach, V. van Pesch, Y. Blanco, D. Maimone, J. Lechner-Scott, R. Bergamaschi, R. Karabudak, C. McGuigan, E. Cartechini, M. Barnett, S. Hughes, MJ. Sa, C. Solaro, C. Ramo-Tello, S. Hodgkinson, D. Spitaleri, A. Soysal, T. Petersen, F. Granella, K. de Gans, P. McCombe, R. Ampapa, B. Van Wijmeersch, A. van der Walt, H. Butzkueven, J. Prevost, JL. Sanchez-Menoyo, G. Laureys, R. Gouider, T. Castillo-Triviño, O. Gray, E. Aguera-Morales, A. Al-Asmi, C. Shaw, N. Deri, T. Al-Harbi, Y. Fragoso, T. Csepany, AP. Sempere, I. Trevino-Frenk, J. Schepel, F. Moore, C. Malpas, T. Kalincik
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- $a BACKGROUND: In the absence of evidence from randomised controlled trials, observational data can be used to emulate clinical trials and guide clinical decisions. Observational studies are, however, susceptible to confounding and bias. Among the used techniques to reduce indication bias are propensity score matching and marginal structural models. OBJECTIVE: To use the comparative effectiveness of fingolimod vs natalizumab to compare the results obtained with propensity score matching and marginal structural models. METHODS: Patients with clinically isolated syndrome or relapsing remitting MS who were treated with either fingolimod or natalizumab were identified in the MSBase registry. Patients were propensity score matched, and inverse probability of treatment weighted at six monthly intervals, using the following variables: age, sex, disability, MS duration, MS course, prior relapses, and prior therapies. Studied outcomes were cumulative hazard of relapse, disability accumulation, and disability improvement. RESULTS: 4608 patients (1659 natalizumab, 2949 fingolimod) fulfilled inclusion criteria, and were propensity score matched or repeatedly reweighed with marginal structural models. Natalizumab treatment was associated with a lower probability of relapse (PS matching: HR 0.67 [95% CI 0.62-0.80]; marginal structural model: 0.71 [0.62-0.80]), and higher probability of disability improvement (PS matching: 1.21 [1.02 -1.43]; marginal structural model 1.43 1.19 -1.72]). There was no evidence of a difference in the magnitude of effect between the two methods. CONCLUSIONS: The relative effectiveness of two therapies can be efficiently compared by either marginal structural models or propensity score matching when applied in clearly defined clinical contexts and in sufficiently powered cohorts.
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