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001-es BibID:BIBFORM103014
035-os BibID:(Scopus)85107568058 (Wos)000687405300017
Első szerző:Andersen, Johanna Balslev
Cím:The effectiveness of natalizumab vs fingolimod : a comparison of international registry studies / Andersen Johanna B., Sharmin Sifat, Lefort Mathilde, Koch-Henriksen Nils, Sellebjerg Finn, Srensen Per Soelberg, Hilt Christensen Claudia C., Rasmussen Peter V., Jensen Michael B., Frederiksen Jette L., Bramow Stephan, Mathiesen Henrik K., Schreiber Karen I., Horakova Dana, Havrdova Eva K., Alroughani Raed, Izquierdo Guillermo, Eichau Sara, Ozakbas Serkan, Patti Francesco, Onofrj Marco, Lugaresi Alessandra, Terzi Murat, Grammond Pierre, Grand Maison Francois, Yamout Bassem, Prat Alexandre, Girard Marc, Duquette Pierre, Boz Cavit, Trojano Maria, McCombe Pamela, Slee Mark, Lechner-Scott Jeannette, Turkoglu Recai, Sola Patrizia, Ferraro Diana, Granella Franco, Shaygannejad Vahid, Prevost Julie, Skibina Olga, Solaro Claudio, Karabudak Rana, Wijmeersch Bart V., Csepany Tunde, Spitaleri Daniele, Vucic Steve, Casey Romain, Debouverie Marc, Edan Gilles, Ciron Jonathan, Ruet Aurélie, Seze, Jérome D., Maillart Elisabeth, Zephir Hélene, Labauge Pierre Defer Gilles, Lebrun Christine, Moreau Thibault, Berger Eric, Clavelou Pierre, Pelletier Jean, Stankoff Bruno, Gout Olivier, Thouvenot Eric, Heinzlef Olivier, Al-Khedr Abdullatif, Bourre Bertrand, Casez Olivier, Cabre Philippe, Montcuquet Alexis, Wahab Abir, Camdessanché Jean-Philippe, Marousset Aude, Patry Ivania, Hankiewicz Karolina, Pottier Corinne, Maubeuge Nicolas, Labeyrie Céline, Nifle Chantal, Leray Emmanuelle, Laplaud David A., Butzkueven Helmut, Kalincik Tomas, Vukusic Sandra, Magyari Melinda
Dátum:2021
ISSN:2211-0348
Megjegyzések:Background Natalizumab and fingolimod were the first preparations recommended for disease breakthrough in priorly treated relapsing-remitting multiple sclerosis. Of three published head-to-head studies two showed that natalizumab is the more effective to prevent relapses and EDSS worsening. Methods By re-analyzing original published results from MSBase, France, and Denmark using uniform methodologies, we aimed at identifying the effects of differences in methodology, in the MS-populations, and at re-evaluating the differences in effectiveness between the two drugs. We gained access to copies of the individual amended databases and pooled all data. We used uniform inclusion/exclusion criteria and statistical methods with Inverse Probability Treatment Weighting. Results The pooled analyses comprised 968 natalizumab- and 1479 fingolimod treated patients. The on-treatment natalizumab/fingolimod relapse rate ratio was 0.77 (p=0.004). The hazard ratio (HR) for a first relapse was 0.82 (p=0.030), and the HR for sustained EDSS improvement was 1.4 (p=0.009). There were modest differences between each of the original published studies and the replication study, but the conclusions of the three original studies remained unchanged: in two of them natalizumab was more effective, but in the third there was no difference between natalizumab and fingolimod. Conclusion The results were largely invariant to the epidemiological and statistical methods but differed between the MS populations. Generally, the advantage of natalizumab was confirmed.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Multiple Sclerosis and Related Disorders. - 53 (2021), p. 1-15. -
További szerzők:Sharmin, Sifat Lefort, Mathilde Koch-Henriksen, Niels Sellebjerg, Finn Thorup Srensen, Per Hilt Christensen, Claudia C. Rasmussen, Peter Vestergaard Jensen, Michael Broksgaard Frederiksen, Jette Lautrup Bramow, Stephan Mathiesen, Henrik Kahr Schreiber, Karen Horakova, Dana Havrdova, Eva Alroughani, Raed Izquierdo, Guillermo Eichau, Sara Ozakbas, Serkan Patti, Francesco Onofrj, Marco Lugaresi, Alessandra Terzi, Murat Grammond, Pierre Grand Maison, Francois Yamout, Bassem Prat, Alexandre Girard, Marc Duquette, Pierre Boz, Cavit Trojano, Maria McCombe, Pamela Slee, Mark Lechner-Scott, Jeannette Turkoglu, Recai Sola, Patrizia Ferraro, Diana Granella, Franco Shaygannejad, Vahid Prevost, Julie Skibina, Olga Solaro, Claudio Karabudak, Rana Wijmeersch, Bart Van Csépány Tünde (1956-) (neurológus, pszichiáter) Spitaleri, Daniele Vucic, Steve Casey, Romain Debouverie, Marc Edan, Gilles Ciron, Jonathan Ruet, Aurélie Seze, Jérome D. Maillart, Elisabeth Zephir, Hélène Labauge, Pierre Defer, Gilles Lebrun-Frenay, Christine Moreau, Thibault Berger, Eric Clavelou, Pierre Pelletier, Jean Stankoff, Bruno Gout, Olivier Thouvenot, Eric Heinzlef, Olivier Al-Khedr, Abdullatif Bourre, Bertrand Casez, Olivier Cabre, Philippe Montcuquet, Alexis Wahab, Abir Camdessanche, Jean-Philippe Marousset, Aude Patry, Ivania Hankiewicz, Karolina Pottier, Corinne Maubeuge, Nicolas Labeyrie, Céline Nifle, Chantal Leray, Emmanuelle Laplaud, David Butzkueven, Helmut Kalincik, Tomas Vukusic, Sandra Magyari Melinda
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2.

001-es BibID:BIBFORM120944
035-os BibID:(Scopus)85122546501
Első szerző:Brouwer, Edward De
Cím:Corrigendum to Longitudinal machine learning modeling of MS patient trajectories improves predictions of disability progression : [Computer Methods and Programs in Biomedicine, Volume 208, (September 2021) 106180] / Edward De Brouwer, Thijs Becker, Yves Moreau, Eva Kubala Havrdova, Maria Trojano, Sara Eichau, Serkan Ozakbas, Marco Onofrj, Pierre Grammond, Jens Kuhle, Ludwig Kappos, Patrizia Sola, Elisabetta Cartechini, Jeannette Lechner-Scott, Raed Alroughani, Oliver Gerlach, Tomas Kalincik, Franco Granella, Francois Grand'Maison, Roberto Bergamaschi, Maria José Sá, Bart Van Wijmeersch, Aysun Soysal, Jose Luis Sanchez-Menoyo, Claudio Solaro, Cavit Boz, Gerardo Iuliano, Katherine Buzzard, Eduardo Aguera-Morales, Murat Terzi, Tamara Castillo Trivio, Daniele Spitaleri, Vincent Van Pesch, Vahid Shaygannejad, Fraser Moore, Celia Oreja-Guevara, Davide Maimone, Riadh Gouider, Tunde Csepany, Cristina Ramo-Tello, Liesbet Peeters
Dátum:2022
ISSN:0169-2607
Megjegyzések:Background and Objectives Research in Multiple Sclerosis (MS) has recently focused on extracting knowledge from real-world clinical data sources. This type of data is more abundant than data produced during clinical trials and potentially more informative about real-world clinical practice. However, this comes at the cost of less curated and controlled data sets. In this work we aim to predict disability progression by optimally extracting information from longitudinal patient data in the real-world setting, with a special focus on the sporadic sampling problem. Methods We use machine learning methods suited for patient trajectories modeling, such as recurrent neural networks and tensor factorization. A subset of 6682 patients from the MSBase registry is used. Results We can predict disability progression of patients in a two-year horizon with an ROC-AUC of 0.85, which represents a 32% decrease in the ranking pair error (1-AUC) compared to reference methods using static clinical features. Conclusions Compared to the models available in the literature, this work uses the most complete patient history for MS disease progression prediction and represents a step forward towards AI-assisted precision medicine in MS.
Tárgyszavak:Orvostudományok Klinikai orvostudományok hozzászólás
folyóiratcikk
Multiple sclerosis
Machine learning
Longitudinal data
Recurrent neural networks
Electronic health records
Disability progression
Real-world data
Megjelenés:Computer Methods And Programs In Biomedicine. - 213 (2022), p. 1-3. -
További szerzők:Becker, Thijs Moreau, Yves Havrdova, Eva Trojano, Maria Eichau, Sara Ozakbas, Serkan Onofrj, Marco Grammond, Pierre Kuhle, Jens Kappos, Ludwig Sola, Patrizia Cartechini, Elisabetta Lechner-Scott, Jeannette Alroughani, Raed Gerlach, Oliver Kalincik, Tomas Granella, Franco Grand'Maison, Francois Bergamaschi, Roberto Sá, Maria José Wijmeersch, Bart Van Soysal, Aysun Sanchez-Menoyo, Jose Solaro, Claudio Boz, Cavit Iuliano, Gerardo Buzzard, Katherine Aguera-Morales, Eduardo Terzi, Murat Trivio, Tamara Castillo Spitaleri, Daniele Pesch, Vincent van Shaygannejad, Vahid Moore, Fraser Oreja-Guevara, Celia Maimone, Davide Gouider, Riadh Csépány Tünde (1956-) (neurológus, pszichiáter) Ramo-Tello, Cristina Peeters, Liesbet
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3.

001-es BibID:BIBFORM085749
Első szerző:Brown, Jeremy William L.
Cím:The risk of relapse following on-treatment clinically silent lesions in patients with relapsing-remitting multiple sclerosis / Brown, J. W. L., Lugaresi A., Horakova D., Havrdova E., Jokubaitis V., Lechner-Scott J., Trojano M., Min M., Shaw C., Shuey N., Slee M., Mccombe P., Van Pesch V., Van Wijmeersch B., Prevost J., Moore F., Prat A., Girard M., Duquette P., Ayrignac X., Sempere A. Perez, Sanchez-Menoyo J. L., Ramo-Tello C., Csépány Tünde, Hutchinson M., De Luca G., Bergamaschi R., Granella F., Curti E., Tsantes E., Sola P., Ferraro D., Alroughani R., Hupperts R., Al-Harbi T., Sidhom Y., Boz C., Terzi M., Ozakbas S., Soysal A., Pucci E., Izquierdo G., Iuliano G., Rio M. Edite, Spitaleri D., Grammond P., Grand'Maison F., Butzkueven H., Kalincik T.
Dátum:2017
ISSN:1352-4585
Tárgyszavak:Orvostudományok Klinikai orvostudományok idézhető absztrakt
folyóiratcikk
Megjelenés:Multiple Sclerosis. - 23 : Suppl. 3 (2017), p. 992-994. -
További szerzők:Lugaresi, Alessandra Horakova, Dana Havrdova, Eva Jokubaitis, Vilija Lechner-Scott, Jeannette Trojano, Maria Min, M. Shaw, C. A. Shuey, Neil Slee, Mark McCombe, Pamela Pesch, Vincent van Wijmeersch, Bart Van Prevost, Julie Moore, Fraser Prat, Alexandre Girard, Marc Duquette, Pierre Ayrignac, X. Sempere, Perez A. Sanchez-Menoyo, Jose Ramo-Tello, Cristina Csépány Tünde (1956-) (neurológus, pszichiáter) Hutchinson, Michael De Luca, Giacomo Bergamaschi, Roberto Granella, Franco Curti, E. Tsantes, E. Sola, Patrizia Ferraro, D. Alroughani, Raed Hupperts, Raymond Al-Harbi, Talal Sidhom, Youssef Boz, Cavit Terzi, Murat Ozakbas, Serkan Soysal, Aysun Pucci, Eugenio Izquierdo, Guillermo Iuliano, Gerardo Rio, Edite M. Spitaleri, Daniele Grammond, Pierre Grand'Maison, Francois Butzkueven, Helmut Kalincik, Tomas
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4.

001-es BibID:BIBFORM103017
035-os BibID:(Wos)000685503300008 (Scopus)85107912293
Első szerző:De Brouwer, Edward
Cím:Longitudinal machine learning modeling of MS patient trajectories improves predictions of disability progression / De Brouwer Edward, Becker Thijs, Moreau Yves, Havrdova Eva Kubala, Trojano Maria, Eichau Sara, Ozakbas Serkan, Onofrj Marco, Grammond Pierre, Kuhle Jens, Kappos Ludwig, Sola Patrizia, Cartechini Elisabetta, Lechner-Scott Jeannette, Alroughani Raed, Gerlach Oliver, Kalincik Tomas, Granella Franco, Grand'Maison Francois, Bergamaschi Roberto, José Sá Maria, Van Wijmeersch Bart, Soysal Aysun, Sanchez-Menoyo Jose Luis, Solaro Claudio, Boz Cavit, Iuliano Gerardo, Buzzard Katherine, Aguera-Morales Eduardo, Terzi Murat, Trivio Tamara Castillo, Spitaleri Daniele, Van Pesch Vincent, Shaygannejad Vahid, Moore Fraser, Oreja-Guevara Celia, Maimone Davide, Gouider Riadh, Csepany Tunde, Ramo-Tello Cristina, Peeters Liesbet
Dátum:2021
ISSN:0169-2607
Megjegyzések:Background and Objectives: Research in Multiple Sclerosis (MS) has recently focused on extracting knowledge from real-world clinical data sources. This type of data is more abundant than data produced during clinical trials and potentially more informative about real-world clinical practice. However, this comes at the cost of less curated and controlled data sets. In this work we aim to predict disability progression by optimally extracting information from longitudinal patient data in the real-world setting, with a special focus on the sporadic sampling problem. Methods: We use machine learning methods suited for patient trajectories modeling, such as recurrent neural networks and tensor factorization. A subset of 6682 patients from the MSBase registry is used. Results: We can predict disability progression of patients in a two-year horizon with an ROC-AUC of 0.85, which represents a 32% decrease in the ranking pair error (1-AUC) compared to reference methods using static clinical features. Conclusions: Compared to the models available in the literature, this work uses the most complete patient history for MS disease progression prediction and represents a step forward towards AI-assisted precision medicine in MS.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Computer Methods And Programs In Biomedicine. - 208 (2021), p. 1-14. -
További szerzők:Becker, Thijs Moreau, Yves Havrdova, Eva Trojano, Maria Eichau, Sara Ozakbas, Serkan Onofrj, Marco Grammond, Pierre Kuhle, Jens Kappos, Ludwig Sola, Patrizia Cartechini, Elisabetta Lechner-Scott, Jeannette Alroughani, Raed Gerlach, Oliver Kalincik, Tomas Granella, Franco Grand'Maison, Francois Bergamaschi, Roberto José Sá, Maria Wijmeersch, Bart Van Soysal, Aysun Sanchez-Menoyo, Jose Solaro, Claudio Boz, Cavit Iuliano, Gerardo Buzzard, Katherine Aguera-Morales, Eduardo Terzi, Murat Trivio, Tamara Castillo Spitaleri, Daniele Pesch, Vincent van Shaygannejad, Vahid Moore, Fraser Oreja-Guevara, Celia Maimone, Davide Gouider, Riadh Csépány Tünde (1956-) (neurológus, pszichiáter) Ramo-Tello, Cristina Peeters, Liesbet
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5.

001-es BibID:BIBFORM132945
035-os BibID:(scopus)85217750289 (wos)001434985500001
Első szerző:D'hondt, Robbe
Cím:Explainable time-to-progression predictions in multiple sclerosis / D'hondt Robbe, Dedja Klest, Aerts Sofie, Van Wijmeersch Bart, Kalincik Tomas, Reddel Stephen, Havrdova Eva Kubala, Lugaresi Alessandra, Weinstock-Guttman Bianca, Mrabet Saloua, Lalive Patrice, Kermode Allan G., Ozakbas Serkan, Patti Francesco, Prat Alexandre, Tomassini Valentina, Roos Izanne, Alroughani Raed, Gerlach Oliver, Khoury Samia J., van Pesch Vincent, Sá Maria José, Prevost Julie, Spitaleri Daniele, McCombe Pamela, Solaro Claudio, van der Walt Anneke, Butzkueven Helmut, Laureys Guy, Sánchez-Menoyo José Luis, de Gans Koen, Al-Asmi Abdullah, Deri Norma, Csepany Tunde, Al-Harbi Talal, Carroll William M., Rozsa Csilla, Singhal Bhim, Hardy Todd A., Ramanathan Sudarshini, Peeters Liesbet, Vens Celine, MSBase Study Group
Dátum:2025
ISSN:0169-2607
Megjegyzések:Background: Prognostic machine learning research in multiple sclerosis has been mainly focusing on black-box models predicting whether a patients' disability will progress in a fixed number of years. However, as this is a binary yes/no question, it cannot take individual disease severity into account. Therefore, in this work we propose to model the time to disease progression instead. Additionally, we use explainable machine learning techniques to make the model outputs more interpretable. Methods: A preprocessed subset of 29,201 patients of the international data registry MSBase was used. Disability was assessed in terms of the Expanded Disability Status Scale (EDSS). We predict the time to significant and confirmed disability progression using random survival forests, a machine learning model for survival analysis. Performance is evaluated on a time-dependent area under the receiver operating characteristic and the precision-recall curves. Importantly, predictions are then explained using SHAP and Bellatrex, two explainability toolboxes, and lead to both global (population-wide) as well as local (patient visit-specific) insights. Results: On the task of predicting progression in 2 years, the random survival forest achieves state-of-the-art performance, comparable to previous work employing a random forest. However, here the random survival forest has the added advantage of being able to predict progression over a longer time horizon, with AUROC >60% for the first 10 years after baseline. Explainability techniques further validated the model by extracting clinically valid insights from the predictions made by the model. For example, a clear decline in the per-visit probability of progression is observed in more recent years since 2012, likely reflecting globally increasing use of more effective MS therapies. Conclusion: The binary classification models found in the literature can be extended to a time-to-event setting without loss of performance, thus allowing a more comprehensive prediction of patient prognosis. Furthermore, explainability techniques proved to be key to reach a better understanding of the model and increase validation of its behaviour.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Disability progression
Explainable artificial intelligence
Longitudinal data
Multiple sclerosis
Survival analysis
Megjelenés:Computer Methods And Programs In Biomedicine. - 263 (2025), p. 1-23. -
További szerzők:Dedja, Klest Aerts, Sofie Wijmeersch, Bart Van Kalincik, Tomas Reddel, Stephen Havrdova, Eva Lugaresi, Alessandra Weinstock-Guttman, Bianca Mrabet, Saloua Lalive, Patrice H. Kermode, Allan G. Ozakbas, Serkan Patti, Francesco Prat, Alexandre Tomassini, Valentina Roos, Izanne Alroughani, Raed Gerlach, Oliver Khoury, Samia J. Pesch, Vincent van Sá, Maria José Prevost, Julie Spitaleri, Daniele McCombe, Pamela Solaro, Claudio Walt, Anneke van der Butzkueven, Helmut Laureys, Guy (Universitary Hospital Ghent) Sanchez-Menoyo, Jose de Gans, Koen Al-Asmi, Abdullah Deri, Norma Csépány Tünde (1956-) (neurológus, pszichiáter) Al-Harbi, Talal Carroll, William M. Rózsa Csilla Singhal, Bhim Hardy, Todd A. Ramanathan, Sudarshini Peeters, Liesbet Vens, Celine MSBase Study Group
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6.

001-es BibID:BIBFORM119146
035-os BibID:(scopus)85177103067 (wos)001030569800001
Első szerző:Diouf, Ibrahima
Cím:Effectiveness of multiple disease-modifying therapies in relapsing-remitting multiple sclerosis : causal inference to emulate a multiarm randomised trial / Diouf Ibrahima, Malpas Charles B., Sharmin Sifat, Roos Izanne, Horakova Dana, Kubala Havrdova Eva, Patti Francesco, Shaygannejad Vahid, Ozakbas Serkan, Eichau Sara, Onofrj Marco, Lugaresi Alessandra, Alroughani Raed, Prat Alexandre, Duquette Pierre, Terzi Murat, Boz Cavit, Grand'Maison Francois, Sola Patrizia, Ferraro Diana, Grammond Pierre, Yamout Bassem, Altintas Ayse, Gerlach Oliver, Lechner-Scott Jeannette, Bergamaschi Roberto, Karabudak Rana, Iuliano Gerardo, McGuigan Christopher, Cartechini Elisabetta, Hughes Stella, Sa Maria Jose, Solaro Claudio, Kappos Ludwig, Hodgkinson Suzanne, Slee Mark, Granella Franco, de Gans Koen, McCombe Pamela A., Ampapa Radek, van der Walt Anneke, Butzkueven Helmut, Sánchez-Menoyo José Luis, Vucic Steve, Laureys Guy, Sidhom Youssef, Gouider Riadh, Castillo-Trivino Tamara, Gray Orla, Aguera-Morales Eduardo, Al-Asmi Abdullah, Shaw Cameron, Al-Harbi Talal M., Csepany Tunde, Sempere Angel P., Trevino Frenk Irene, Stuart Elizabeth A., Kalincik Tomas
Dátum:2023
ISSN:0022-3050
Megjegyzések:Background Simultaneous comparisons of multiple disease-modifying therapies for relapsing-remitting multiple sclerosis (RRMS) over an extended follow-up are lacking. Here we emulate a randomised trial simultaneously comparing the effectiveness of six commonly used therapies over 5 years. Methods Data from 74 centres in 35 countries were sourced from MSBase. For each patient, the first eligible intervention was analysed, censoring at change/discontinuation of treatment. The compared interventions included natalizumab, fingolimod, dimethyl fumarate, teriflunomide, interferon beta, glatiramer acetate and no treatment. Marginal structural Cox models (MSMs) were used to estimate the average treatment effects (ATEs) and the average treatment effects among the treated (ATT), rebalancing the compared groups at 6-monthly intervals on age, sex, birth-year, pregnancy status, treatment, relapses, disease duration, disability and disease course. The outcomes analysed were incidence of relapses, 12-month confirmed disability worsening and improvement. Results 23 236 eligible patients were diagnosed with RRMS or clinically isolated syndrome. Compared with glatiramer acetate (reference), several therapies showed a superior ATE in reducing relapses: natalizumab (HR=0.44, 95% CI=0.40 to 0.50), fingolimod (HR=0.60, 95% CI=0.54 to 0.66) and dimethyl fumarate (HR=0.78, 95% CI=0.66 to 0.92). Further, natalizumab (HR=0.43, 95% CI=0.32 to 0.56) showed a superior ATE in reducing disability worsening and in disability improvement (HR=1.32, 95% CI=1.08 to 1.60). The pairwise ATT comparisons also showed superior effects of natalizumab followed by fingolimod on relapses and disability. Conclusions The effectiveness of natalizumab and fingolimod in active RRMS is superior to dimethyl fumarate, teriflunomide, glatiramer acetate and interferon beta. This study demonstrates the utility of MSM in emulating trials to compare clinical effectiveness among multiple interventions simultaneously.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
MULTIPLE SCLEROSIS
STATISTICS
Megjelenés:Journal Of Neurology Neurosurgery And Psychiatry. - 94 : 12 (2023), p. 1004-1011. -
További szerzők:Malpas, Charles B. Sharmin, Sifat Roos, Izanne Horakova, Dana Kubala Havrdova, Eva Patti, Francesco Shaygannejad, Vahid Ozakbas, Serkan Eichau, Sara Onofrj, Marco Lugaresi, Alessandra Alroughani, Raed Prat, Alexandre Duquette, Pierre Terzi, Murat Boz, Cavit Grand'Maison, Francois Sola, Patrizia Ferraro, Diana Grammond, Pierre Yamout, Bassem Altintas, Ayse Gerlach, Oliver Lechner-Scott, Jeannette Bergamaschi, Roberto Karabudak, Rana Iuliano, Gerardo McGuigan, Christopher Cartechini, Elisabetta Hughes, Stella Sá, Maria José Solaro, Claudio Kappos, Ludwig Hodgkinson, Suzanne Slee, Mark Granella, Franco de Gans, Koen McCombe, Pamela Ampapa, Radek Walt, Anneke van der Butzkueven, Helmut Sanchez-Menoyo, Jose Vucic, Steve Laureys, Guy Sidhom, Youssef Gouider, Riadh Castillo Triviño, Tamara Gray, Orla Aguera-Morales, Eduardo Al-Asmi, Abdullah Shaw, Cameron Al-Harbi, Talal Csépány Tünde (1956-) (neurológus, pszichiáter) Sempere, Perez A. Trevino-Frenk, Irene Stuart, Elizabeth A. Kalincik, Tomas
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7.

001-es BibID:BIBFORM107545
035-os BibID:(Scopus)85148460657 (WoS)000952991100026
Első szerző:Diouf, Ibrahima
Cím:Variability of the response to immunotherapy among subgroups of patients with multiple sclerosis / Diouf Ibrahima, Malpas Charles B., Sharmin Sifat, Roos Izanne, Horakova Dana, Havrdova Eva Kubala, Patti Francesco, Shaygannejad Vahid, Ozakbas Serkan, Izquierdo Guillermo, Eichau Sara, Onofrj Marco, Lugaresi Alessandra, Alroughani Raed, Prat Alexandre, Girard Marc, Duquette Pierre, Terzi Murat, Boz Cavit, Grand'Maison Francois, Hamdy Sherif, Sola Patrizia, Ferraro Diana, Grammond Pierre, Turkoglu Recai, Buzzard Katherine, Skibina Olga, Yamout Bassem, Altintas Ayse, Gerlach Oliver, van Pesch Vincent, Blanco Yolanda, Maimone Davide, Lechner-Scott Jeannette, Bergamaschi Roberto, Karabudak Rana, Iuliano Gerardo, McGuigan Chris, Cartechini Elisabetta, Barnett Michael, Hughes Stella, Sa Maria José, Solaro Claudio, Kappos Ludwig, Ramo-Tello Cristina, Cristiano Edgardo, Hodgkinson Suzanne, Spitaleri Daniele, Soysal Aysun, Petersen Thor, Slee Mark, Butler Ernest, Granella Franco, de Gans Koen, McCombe Pamela, Ampapa Radek, Van Wijmeersch Bart, van der Walt Anneke, Butzkueven Helmut, Prevost Julie, Sinnige L. G. F., Sanchez-Menoyo Jose Luis, Vucic Steve, Laureys Guy, Van Hijfte Liesbeth, Khurana Dheeraj, Macdonell Richard, Gouider Riadh, Castillo-Trivino Tamara, Gray Orla, Aguera-Morales Eduardo, Al-Asmi Abdullah, Shaw Cameron, Deri Norma, Al-Harbi Talal, Fragoso Yara, Csepany Tunde, Perez Sempere Angel, Trevino-Frenk Irene, Schepel Jan, Moore Fraser, Kalincik Tomas
Dátum:2023
ISSN:1351-5101
Megjegyzések:Background This study assessed the effect of patient characteristics on the response to disease modifying therapy (DMT) in in multiple sclerosis (MS). Methods We extracted data from 61,810 patients from 135 centres across 35 countries from the MSBase registry. The selection criteria were: clinically isolated syndrome or definite MS; follow-up ?1 year; ?3 EDSS scores; and with ?1 score recorded per year. Marginal structural models with interaction terms were used to compare the hazards of 12-month confirmed worsening and improvement of disability, and the incidence of relapses between treated and untreated patients stratified by their characteristics. Results Among 24,344 patients with relapsing MS, those on DMTs experienced 48% reduction in relapse incidence (hazard ratio (HR)=0.52, 95%CI=0.45-0.60), 46% lower risk of disability worsening (HR=0.54, 95%CI=0.41-0.71) and 32% greater chance of disability improvement (HR=1.32, 95%CI=1.09-1.59). The effect of DMTs on EDSS worsening and improvement and the risk of relapses was attenuated with more severe disability. The magnitude of the effect of DMT on suppressing relapses declined with higher prior relapse rate and prior cerebral MRI activity. We did not find any evidence for the effect of age on the effectiveness of DMT. After inclusion of 1985 participants with progressive MS, the effect of DMT on disability mostly depended on MS phenotype, whereas its effect on relapses was driven mainly by prior relapse activity. Conclusions DMT is generally most effective among patients with lower disability and in relapsing MS phenotypes. There is no evidence attenuation of the effect of DMT with age.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:European Journal Of Neurology. - 30 : 4 (2023), p. 1014-1024. -
További szerzők:Malpas, Charles B. Sharmin, Sifat Roos, Izanne Horakova, Dana Havrdova, Eva Patti, Francesco Shaygannejad, Vahid Ozakbas, Serkan Izquierdo, Guillermo Eichau, Sara Onofrj, Marco Lugaresi, Alessandra Alroughani, Raed Prat, Alexandre Girard, Marc Duquette, Pierre Terzi, Murat Boz, Cavit Grand'Maison, Francois Hamdy, Sherif Sola, Patrizia Ferraro, Diana Grammond, Pierre Turkoglu, Recai Buzzard, Katherine Skibina, Olga Yamout, Bassem Altintas, Ayse Gerlach, Oliver Pesch, Vincent van Blanco, Yolanda Maimone, Davide Lechner-Scott, Jeannette Bergamaschi, Roberto Karabudak, Rana Iuliano, Gerardo McGuigan, Christopher Cartechini, Elisabetta Barnett, Michael Hughes, Stella Sá, Maria José Solaro, Claudio Kappos, Ludwig Ramo-Tello, Cristina Cristiano, Edgardo Hodgkinson, Suzanne Spitaleri, Daniele Soysal, Aysun Petersen, Thor Slee, Mark Butler, Ernest Granella, Franco de Gans, Koen McCombe, Pamela Ampapa, Radek Wijmeersch, Bart Van Walt, Anneke van der Butzkueven, Helmut Prevost, Julie Sinnige, L. G. F. Sanchez-Menoyo, Jose Vucic, Steve Laureys, Guy Van Hijfte, Liesbeth Khurana, Dheeraj Macdonell, Richard Gouider, Riadh Castillo Triviño, Tamara Gray, Orla Aguera-Morales, Eduardo Al-Asmi, Abdullah Shaw, Cameron Deri, Norma Al-Harbi, Talal Fragoso, Yara Csépány Tünde (1956-) (neurológus, pszichiáter) Perez Sempere, Angel Trevino-Frenk, Irene Schepel, Jan Moore, Fraser Kalincik, Tomas
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

8.

001-es BibID:BIBFORM083269
Első szerző:Fambiatos, Adam
Cím:Risk of secondary progressive multiple sclerosis : a longitudinal study / Adam Fambiatos, Vilija Jokubaitis, Dana Horakova, Eva Kubala Havrdova, Maria Trojano, Alexandre Prat, Marc Girard, Pierre Duquette, Alessandra Lugaresi, Guillermo Izquierdo, Francois Grand'Maison, Pierre Grammond, Patrizia Sola, Diana Ferraro, Raed Alroughani, Murat Terzi, Raymond Hupperts, Cavit Boz, Jeannette Lechner-Scott, Eugenio Pucci, Roberto Bergamaschi, Vincent Van Pesch, Serkan Ozakbas, Franco Granella, Recai Turkoglu, Gerardo Iuliano, Daniele Spitaleri, Pamela McCombe, Claudio Solaro, Mark Slee, Radek Ampapa, Aysun Soysal, Thor Petersen, Jose Luis Sanchez-Menoyo, Freek Verheul, Julie Prevost, Youssef Sidhom, Bart Van Wijmeersch, Steve Vucic, Edgardo Cristiano, Maria Laura Saladino, Norma Deri, Michael Barnett, Javier Olascoaga, Fraser Moore, Olga Skibina, Orla Gray, Yara Fragoso, Bassem Yamout, Cameron Shaw, Bhim Singhal, Neil Shuey, Suzanne Hodgkinson, Ayse Altintas, Talal Al-Harbi, Tunde Csepany, Bruce Taylor, Jordana Hughes, Jae-Kwan Jun, Anneke van der Walt, Tim Spelman, Helmut Butzkueven, Tomas Kalincik, MSBase Study Group
Dátum:2020
ISSN:1352-4585
Megjegyzések:Background: The risk factors for conversion from relapsing-remitting to secondary progressive multiple sclerosis remain highly contested. Objective: The aim of this study was to determine the demographic, clinical and paraclinical features that influence the risk of conversion to secondary progressive multiple sclerosis. Methods: Patients with adult-onset relapsing?remitting multiple sclerosis and at least four recorded disability scores were selected from MSBase, a global observational cohort. The risk of conversion to objectively defined secondary progressive multiple sclerosis was evaluated at multiple time points per patient using multivariable marginal Cox regression models. Sensitivity analyses were performed. Results: A total of 15,717 patients were included in the primary analysis. Older age (hazard ratio (HR) = 1.02, p < 0.001), longer disease duration (HR = 1.01, p = 0.038), a higher Expanded Disability Status Scale score (HR = 1.30, p < 0.001), more rapid disability trajectory (HR = 2.82, p < 0.001) and greater number of relapses in the previous year (HR = 1.07, p = 0.010) were independently associated with an increased risk of secondary progressive multiple sclerosis. Improving disability (HR = 0.62, p = 0.039) and disease-modifying therapy exposure (HR = 0.71, p = 0.007) were associated with a lower risk. Recent cerebral magnetic resonance imaging activity, evidence of spinal cord lesions and oligoclonal bands in the cerebrospinal fluid were not associated with the risk of conversion. Conclusion: Risk of secondary progressive multiple sclerosis increases with age, duration of illness and worsening disability and decreases with improving disability. Therapy may delay the onset of secondary progression.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Multiple Sclerosis. - 26 : 1 (2020), p. 79-90. -
További szerzők:Jokubaitis, Vilija Horakova, Dana Kubala Havrdova, Eva Trojano, Maria Prat, Alexandre Girard, Marc Duquette, Pierre Lugaresi, Alessandra Izquierdo, Guillermo Grand'Maison, Francois Grammond, Pierre Sola, Patrizia Ferraro, Diana Alroughani, Raed Terzi, Murat Hupperts, Raymond Boz, Cavit Lechner-Scott, Jeannette Pucci, Eugenio Bergamaschi, Roberto Pesch, Vincent van Ozakbas, Serkan Granella, Franco Turkoglu, Recai Iuliano, Gerardo Spitaleri, Daniele McCombe, Pamela Solaro, Claudio Slee, Mark Ampapa, Radek Soysal, Aysun Petersen, Thor Sanchez-Menoyo, Jose Verheul, Freek Prevost, Julie Sidhom, Youssef Wijmeersch, Bart Van Vucic, Steve Cristiano, Edgardo Saladino, Maria Laura Deri, Norma Barnett, Michael Olascoaga, Javier Moore, Fraser Skibina, Olga Gray, Orla Fragoso, Yara Yamout, Bassem Shaw, Cameron Singhal, Bhim Shuey, Neil Hodgkinson, Suzanne Altintas, Ayse Al-Harbi, Talal Csépány Tünde (1956-) (neurológus, pszichiáter) Taylor, Bruce V. Hughes, Jordana Jun, Jae-Kwan Walt, Anneke van der Spelman, Tim Butzkueven, Helmut Kalincik, Tomas MSBase Study Group
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

9.

001-es BibID:BIBFORM126269
035-os BibID:(scopus)85164541334 (wos)000999038400003
Első szerző:Kalincik, Tomas
Cím:Comparative Effectiveness of Autologous Hematopoietic Stem Cell Transplant vs Fingolimod, Natalizumab, and Ocrelizumab in Highly Active Relapsing-Remitting Multiple Sclerosis / Tomas Kalincik, Sifat Sharmin, Izanne Roos, Mark S. Freedman, Harold Atkins, Joachim Burman, Jennifer Massey, Ian Sutton, Barbara Withers, Richard Macdonell, Andrew Grigg, øivind Torkildsen, Lars Bo, Anne Kristine Lehmann, Eva Kubala Havrdova, Eva Krasulova, Marek Trneny, Tomas Kozak, Anneke van der Walt, Helmut Butzkueven, Pamela McCombe, Olga Skibina, Jeannette Lechner-Scott, Barbara Willekens, Elisabetta Cartechini, Serkan Ozakbas, Raed Alroughani, Jens Kuhle, Francesco Patti, Pierre Duquette, Alessandra Lugaresi, Samia J. Khoury, Mark Slee, Recai Turkoglu, Suzanne Hodgkinson, Nevin John, Davide Maimone, Maria Jose Sa, Vincent van Pesch, Oliver Gerlach, Guy Laureys, Liesbeth Van Hijfte, Rana Karabudak, Daniele Spitaleri, Tunde Csepany, Riadh Gouider, Tamara Castillo-Trivino, Bruce Taylor, Basil Sharrack, John A. Snowden, MSBase Study Group Collaborators
Dátum:2023
ISSN:2168-6149 2168-6157
Megjegyzések:IMPORTANCE Autologous hematopoietic stem cell transplant (AHSCT) is available for treatment of highly active multiple sclerosis (MS). OBJECTIVE To compare the effectiveness of AHSCT vs fingolimod, natalizumab, and ocrelizumab in relapsing-remitting MS by emulating pairwise trials. DESIGN, SETTING, AND PARTICIPANTS This comparative treatment effectiveness study included 6 specialist MS centers with AHSCT programs and international MSBase registry between 2006 and 2021. The study included patients with relapsing-remitting MS treated with AHSCT, fingolimod, natalizumab, or ocrelizumab with 2 or more years study follow-up including 2 or more disability assessments. Patients were matched on a propensity score derived from clinical and demographic characteristics. EXPOSURE AHSCT vs fingolimod, natalizumab, or ocrelizumab. MAIN OUTCOMES Pairwise-censored groups were compared on annualized relapse rates (ARR) and freedom from relapses and 6-month confirmed Expanded Disability Status Scale (EDSS) score worsening and improvement. RESULTS Of 4915 individuals, 167 were treated with AHSCT; 2558, fingolimod; 1490, natalizumab; and 700, ocrelizumab. The prematch AHSCT cohort was younger and with greater disability than the fingolimod, natalizumab, and ocrelizumab cohorts; thematched groups were closely aligned. The proportion ofwomen ranged from65% to70%,and themean (SD)age ranged from 35.3 (9.4) to 37.1 (10.6) years. The mean (SD) disease duration ranged from 7.9 (5.6) to 8.7 (5.4) years, EDSS score ranged from 3.5 (1.6) to 3.9 (1.9), and frequency of relapses ranged from0.77 (0.94) to0.86 (0.89) in the preceding year. Compared with the fingolimod group (769 [30.0%]), AHSCT (144 [86.2%]) was associated with fewer relapses (ARR: mean [SD], 0.09 [0.30] vs 0.20 [0.44]), similar risk of disability worsening (hazard ratio [HR], 1.70; 95% CI, 0.91-3.17), and higher chance of disability improvement (HR, 2.70; 95% CI, 1.71-4.26) over 5 years. Compared with natalizumab (730 [49.0%]), AHSCT (146 [87.4%]) was associated withmarginally lower ARR (mean [SD],0.08 [0.31]vs0.10 [0.34]), similar risk of disabilityworsening (HR, 1.06; 95% CI,0.54-2.09), and higher chance of disability improvement (HR, 2.68; 95% CI, 1.72-4.18) over 5 years. AHSCT (110 [65.9%]) and ocrelizumab (343 [49.0%])were associatedwith similarARR (mean [SD],0.09 [0.34]vs0.06 [0.32]), disability worsening (HR, 1.77; 95% CI, 0.61-5.08), and disability improvement (HR, 1.37; 95% CI, 0.66-2.82) over 3 years. AHSCT-related mortality occurred in 1 of 159 patients (0.6%). CONCLUSION In this study, the association of AHSCT with preventing relapses and facilitating recovery from disability was considerably superior to fingolimod and marginally superior to natalizumab. This study did not find evidence for difference in the effectiveness of AHSCT and ocrelizumab over a shorter available follow-up time.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
AHSCT
Fingolimod
Natalizumab
Ocrelizumab
multiple sclerosis
Megjelenés:JAMA Neurology. - 80 : 7 (2023), p. 702-713. -
További szerzők:Sharmin, Sifat Roos, Izanne Freedman, Mark S. Atkins, Harold Burman, Joachim Massey, Jennifer Sutton, Ian Withers, Barbara Macdonell, Richard Grigg, Andrew Torkildsen, øivind Bo, Lars Lehmann, Anne Kristine Kubala Havrdova, Eva Krasulova, Eva Trneny, Marek Kozak, Tomas Walt, Anneke van der Butzkueven, Helmut McCombe, Pamela Skibina, Olga Lechner-Scott, Jeannette Willekens, Barbara Cartechini, Elisabetta Ozakbas, Serkan Alroughani, Raed Kuhle, Jens Patti, Francesco Duquette, Pierre Lugaresi, Alessandra Khoury, Samia J. Slee, Mark Turkoglu, Recai Hodgkinson, Suzanne John, Nevin Maimone, Davide José Sá, Maria Pesch, Vincent van Gerlach, Oliver Laureys, Guy Van Hijfte, Liesbeth Karabudak, Rana Spitaleri, Daniele Csépány Tünde (1956-) (neurológus, pszichiáter) Gouider, Riadh Castillo Triviño, Tamara Taylor, Bruce V. Sharrack, Basil Snowden, John A. MSBase Study Group
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

10.

001-es BibID:BIBFORM083270
035-os BibID:(PMID)31877445
Első szerző:Kunchok, Amy
Cím:Clinical and therapeutic predictors of disease outcomes in AQP4-IgG+ neuromyelitis optica spectrum disorder / Amy Kunchok, Charles Malpas, Petra Nytrova, Eva Kubala Havrdova, Raed Alroughani, Murat Terzi, Bassem Yamout, Jyh Yung Hor, Rana Karabudak, Cavit Boz, Serkan Ozakbas, Javier Olascoaga, Magdolna Simo, Franco Granella, Francesco Patti, Pamela McCombe, Tunde Csepany, Bhim Singhal, Roberto Bergamaschi, Yara Fragoso, Talal Al-Harbi, Recai Turkoglu, Jeannette Lechner-Scott, Guy Laureys, Celia Oreja-Guevara, Eugenio Pucci, Patrizia Sola, Diana Ferraro, Ayse Altintas, Aysun Soysal, Steve Vucic, Francois Grand'Maison, Guillermo Izquierdo, Sara Eichau, Alessandra Lugaresi, Marco Onofrj, Maria Trojano, Mark Marriott, Helmut Butzkueven, Ilya Kister, Tomas Kalincik
Dátum:2020
ISSN:2211-0348
Megjegyzések:Aquaporin-4-IgG positive (AQP4-IgG+) Neuromyelitis Optica Spectrum Disorder (NMOSD) is an uncommon central nervous system autoimmune disorder. Disease outcomes in AQP4-IgG+NMOSD are typically measured by relapse rate and disability. Using the MSBase, a multi-centre international registry, we aimed to examine the impact immunosuppressive therapies and patient characteristics as predictors of disease outcome measures in AQP4-IgG+NMOSD. METHOD: This MSBase cohort study of AQP4-IgG+NMOSD patients examined modifiers of relapse in a multivariable proportional hazards model and expanded disability status score (EDSS) using a mixed effects model. RESULTS: 206 AQP4-IgG+ patients were included (median follow-up 3.7 years). Age (hazard ratio [HR] = 0.82 per decade, p = 0.001), brainstem onset (HR = 0.45, p = 0.009), azathioprine (HR = 0.46, p<0.001) and mycophenolate mofetil (HR = 0.09, p = 0.012) were associated with a reduced risk of relapse. A greater EDSS was associated with age (β = 0.45 (per decade), p<0.001) and disease duration (β = 0.07 per year, p<0.001). A slower increase in EDSS was associated with azathioprine (β = -0.48, p<0.001), mycophenolate mofetil (β = -0.69, p = 0.04) and rituximab (β = -0.35, p = 0.024). INTERPRETATION: This study has demonstrated that azathioprine and mycophenolate mofetil reduce the risk of relapses and disability progression is modified by azathioprine, mycophenolate mofetil and rituximab. Age and disease duration were the only patient characteristics that modified the risk of relapse and disability in our cohort.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Disability
Immunosuppression
Neuromyelitis optica spectrum disorder
Predictors
Relapses
Therapy
Megjelenés:Multiple Sclerosis and Related Disorders. - 38 (2020), p. 1-8. -
További szerzők:Malpas, Charles Nytrova, Petra Havrdova, Eva Alroughani, Raed Terzi, Murat Yamout, Bassem Hor, Jyh Yung Karabudak, Rana Boz, Cavit Ozakbas, Serkan Olascoaga, Javier Simó Magdolna Granella, Franco Patti, Francesco McCombe, Pamela Csépány Tünde (1956-) (neurológus, pszichiáter) Singhal, Bhim Bergamaschi, Roberto Fragoso, Yara Al-Harbi, Talal Turkoglu, Recai Lechner-Scott, Jeannette Laureys, Guy Oreja-Guevara, Celia Pucci, Eugenio Sola, Patrizia Ferraro, Diana Altintas, Ayse Soysal, Aysun Vucic, Steve Grand'Maison, Francois Izquierdo, Guillermo Eichau, Sara Lugaresi, Alessandra Onofrj, Marco Trojano, Maria Marriott, Mark Butzkueven, Helmut Kister, Ilya Kalincik, Tomas
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

11.

001-es BibID:BIBFORM103015
035-os BibID:(Scopus)85130953245 (Wos)000805581400002
Első szerző:Lefort, Mathilde
Cím: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 Seze 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.
Dátum:2022
ISSN:1471-2288
Megjegyzések: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 diferent 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.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:BMC Medical Research Methodology. - 22 : 1 (2022), p. 1-14. -
További szerzők:Sharmin, Sifat Andersen, Johanna Balslev Vukusic, Sandra Casey, Romain Debouverie, Marc Edan, Gilles Ciron, Jonathan Ruet, Aurélie De Seze, Jérôme Maillart, Elisabeth Zephir, Hélène Labauge, Pierre Defer, Gilles Lebrun-Frenay, Christine Moreau, Thibault Berger, Eric Clavelou, Pierre Pelletier, Jean Stankoff, Bruno Gout, Olivier Thouvenot, Eric Heinzlef, Olivier Al-Khedr, Abdullatif Bourre, Bertrand Casez, Olivier Cabre, Philippe Montcuquet, Alexis Wahab, Abir Camdessanche, Jean-Philippe Maurousset, Aude Ben Nasr, Haifa Hankiewicz, Karolina Pottier, Corinne Maubeuge, Nicolas Dimitri-Boulos, D. Nifle, Chantal Laplaud, David Horakova, Dana Havrdova, Eva Alroughani, Raed Izquierdo, Guillermo Eichau, Sara Ozakbas, Serkan Patti, Francesco Onofrj, Marco Lugaresi, Alessandra Terzi, Murat Grammond, Pierre Grand'Maison, Francois Yamout, Bassem Prat, Alexandre Girard, Marc Duquette, Pierre Boz, Cavit Trojano, Maria McCombe, Pamela Slee, Mark Lechner-Scott, Jeannette Turkoglu, Recai Sola, Patrizia Ferraro, Diana Granella, Franco Shaygannejad, Vahid Prevost, Julie Maimone, Davide Skibina, Olga Buzzard, Katherine Walt, Anneke van der Karabudak, Rana Wijmeersch, Bart Van Csépány Tünde (1956-) (neurológus, pszichiáter) Spitaleri, Daniele Vucic, Steve Koch-Henriksen, Niels Sellebjerg, Finn Thorup Soerensen, Per Soelberg Hilt Christensen, Claudia C. Rasmussen, Peter Vestergaard Jensen, Michael Broksgaard Frederiksen, Jette Lautrup Bramow, Stephan Mathiesen, Henrik Kahr Schreiber, Karen Butzkueven, Helmut Magyari Melinda Kalincik, Tomas Leray, Emmanuelle
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

12.

001-es BibID:BIBFORM126432
035-os BibID:(WoS)001077004600002 (Scopus)85181761016
Első szerző:Li, Ying
Cím:Examining the environmental risk factors of progressive-onset and relapsing-onset multiple sclerosis : recruitment challenges, potential bias, and statistical strategies / Li Y., Saul A., Taylor B., Ponsonby A. L., Simpson-Yap S., Blizzard L., Broadley S., Lechner-Scott J., Ausimmune/AusLong Investigators Group, Karabudak R., Patti F., Eichau S., Onofrj M., Ozakbas S., Horakova D., Kubala Havrdova E., Grand'Maison F., Alroughani R., Gerlach O., Amato M. P., Altintas A., Girard M., Duquette P., Blanco Y., Ramo-Tello C., Laureys G., Kalincik T., Khoury S. J., Shaygannejad V., Etemadifar M., Singhal B., Mrabet S., Foschi M., Habek M., John N., Hughes S., McCombe P., Ampapa R., van der Walt A., Butzkueven H., de Gans K., McGuigan C., Oreja-Guevara C., Sa M. J., Petersen T., Al-Harbi T., Sempere A. P., Van Wijmeersch B., Grigoriadis N., Prevost J., Gray O., Castillo-Trivino T., Macdonell R., Lugaresi A., Sajedi S. A., MSBase, van der Mei I.
Dátum:2024
ISSN:0340-5354
Megjegyzések:It is unknown whether the currently known risk factors of multiple sclerosis reflect the etiology of progressive-onset multiple sclerosis (POMS) as observational studies rarely included analysis by type of onset. We designed a case-control study to examine associations between environmental factors and POMS and compared effect sizes to relapse-onset MS (ROMS), which will offer insights into the etiology of POMS and potentially contribute to prevention and intervention practice. This study utilizes data from the Primary Progressive Multiple Sclerosis (PPMS) Study and the Australian Multi-center Study of Environment and Immune Function (the AusImmune Study). This report outlines the conduct of the PPMS Study, whether the POMS sample is representative, and the planned analysis methods. The study includes 155 POMS, 204 ROMS, and 558 controls. The distributions of the POMS were largely similar to Australian POMS patients in the MSBase Study, with 54.8% female, 85.8% POMS born before 1970, mean age of onset of 41.44 ? 8.38 years old, and 67.1% living between 28.9 and 39.4? S. The POMS were representative of the Australian POMS population. There are some differences between POMS and ROMS/controls (mean age at interview: POMS 55 years vs. controls 40 years; sex: POMS 53% female vs. controls 78% female; location of residence: 14.3% of POMS at a latitude ? 28.9?S vs. 32.8% in controls), which will be taken into account in the analysis. We discuss the methodological issues considered in the study design, including prevalence-incidence bias, cohort effects, interview bias and recall bias, and present strategies to account for it. Associations between exposures of interest and POMS/ROMS will be presented in subsequent publications.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Bias
Case-control
Environmental factors
Progressive-onset multiple sclerosis
Subject recruitment.
Megjelenés:Journal Of Neurology. - 271 : 1 (2024), p. 472-485. -
További szerzők:Saul, Alice Taylor, Bruce V. Ponsonby, Anne-Louise Simpson-Yap, Steve Blizzard, Leigh Broadley, Simon Lechner-Scott, Jeannette Karabudak, Rana Patti, Francesco Eichau, Sara Onofrj, Marco Ozakbas, Serkan Horakova, Dana Kubala Havrdova, Eva Grand'Maison, Francois Alroughani, Raed Gerlach, Oliver Amato, Maria Pia Altintas, Ayse Girard, Marc Duquette, Pierre Blanco, Yolanda Ramo-Tello, Cristina Laureys, Guy Kalincik, Tomas Khoury, Samia J. Shaygannejad, Vahid Etemadifar, Masoud Singhal, Bhim Mrabet, Saloua Foschi, Matteo Habek, Mario John, Nevin Hughes, Stella McCombe, Pamela Ampapa, Radek Walt, Anneke van der Butzkueven, Helmut de Gans, Koen McGuigan, Christopher Oreja-Guevara, Celia Sá, Maria José Petersen, Thor Al-Harbi, Talal Sempere, Perez A. Wijmeersch, Bart Van Grigoriadis, Nikolaos Prevost, Julie Gray, Orla Castillo Triviño, Tamara Macdonell, Richard Lugaresi, Alessandra Sajedi, Seyed Aidin Mei, Ingrid van der Csépány Tünde (1956-) (neurológus, pszichiáter) Ausimmune/AusLong Investigators Group MSBase Study Group
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