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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: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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3.

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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4.

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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5.

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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6.

001-es BibID:BIBFORM103011
035-os BibID:(Scopus)85102090793 (Wos)000656637200025
Első szerző:Kalincik, Tomas
Cím:Effect of Disease-Modifying Therapy on Disability in Relapsing-Remitting Multiple Sclerosis Over 15 Years / Kalincik Tomas, Diouf Ibrahima, Sharmin Sifat, Malpas Charles, Spelman Tim, Horakova Dana, Havrdova Eva Kubala, Trojano Maria, Izquierdo Guillermo, Lugaresi Alessandra, Prat Alexandre, Girard Marc, Duquette Pierre, Grammond Pierre, Jokubaitis Vilija, van der Walt Anneke, Grand'Maison Francois, Sola Patrizia, Ferraro Diana, Shaygannejad Vahid, Alroughani Raed, Hupperts Raymond, Terzi Murat, Boz Cavit, Lechner-Scott Jeannette, Pucci Eugenio, Van Pesch Vincent, Granella Franco, Bergamaschi Roberto, Spitaleri Daniele, Slee Mark, Vucic Steve, Ampapa Radek, McCombe Pamela, Ramo-Tello Cristina, Prevost Julie, Olascoaga Javier, Cristiano Edgardo, Barnett Michael, Saladino Maria Laura, Sanchez-Menoyo Jose Luis, Hodgkinson Suzanne, Rozsa Csilla, Hughes Stella, Moore Fraser, Shaw Cameron, Butler Ernest, Skibina Olga, Gray Orla, Kermode Allan, Csepany Tunde, Singhal Bhim, Shuey Neil, Piroska Imre, Taylor Bruce, Simo Magdolna, Sirbu Carmen-Adella, Sas Attila, Butzkueven Helmut, MSBase Study Group
Dátum:2021
ISSN:0028-3878 1526-632X
Megjegyzések:Objective To test the hypothesis that immunotherapy prevents long-term disability in relapsing-remitting multiple sclerosis (MS), we modeled disability outcomes in 14,717 patients. Methods We studied patients from MSBase followed for ?1 year, with ?3 visits, ?1 visit per year, and exposed to MS therapy, and a subset of patients with ?15-year follow-up. Marginal structural models were used to compare the cumulative hazards of 12-month confirmed increase and decrease in disability, Expanded Disability Status Scale (EDSS) step 6, and the incidence of relapses between treated and untreated periods. Marginal structural models were continuously readjusted for patient age, sex, pregnancy, date, disease course, time from first symptom, prior relapse history, disability, and MRI activity. Results A total of 14,717 patients were studied. During the treated periods, patients were less likely to experience relapses (hazard ratio 0.60, 95% confidence interval [CI] 0.43?0.82, p = 0.0016), worsening of disability (0.56, 0.38?0.82, p = 0.0026), and progress to EDSS step 6 (0.33, 0.19?0.59, p = 0.00019). Among 1,085 patients with ?15-year follow-up, the treated patients were less likely to experience relapses (0.59, 0.50?0.70, p = 10?9) and worsening of disability (0.81, 0.67?0.99, p = 0.043). Conclusion Continued treatment with MS immunotherapies reduces disability accrual by 19%?44% (95% CI 1%?62%), the risk of need of a walking aid by 67% (95% CI 41%?81%), and the frequency of relapses by 40?41% (95% CI 18%?57%) over 15 years. This study provides evidence that disease-modifying therapies are effective in improving disability outcomes in relapsing-remitting MS over the long term. Classification of Evidence This study provides Class IV evidence that, for patients with relapsing-remitting MS, long-term exposure to immunotherapy prevents neurologic disability.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Neurology. - 96 : 5 (2021), p. e783-e797. -
További szerzők:Diouf, Ibrahima Sharmin, Sifat Malpas, Charles Spelman, Tim Horakova, Dana Havrdova, Eva Trojano, Maria Izquierdo, Guillermo Lugaresi, Alessandra Prat, Alexandre Girard, Marc Duquette, Pierre Grammond, Pierre Jokubaitis, Vilija Walt, Anneke van der Grand'Maison, Francois Sola, Patrizia Ferraro, Diana Shaygannejad, Vahid Alroughani, Raed Hupperts, Raymond Terzi, Murat Boz, Cavit Lechner-Scott, Jeannette Pucci, Eugenio Pesch, Vincent van Granella, Franco Bergamaschi, Roberto Spitaleri, Daniele Slee, Mark Vucic, Steve Ampapa, Radek McCombe, Pamela Ramo-Tello, Cristina Prevost, Julie Olascoaga, Javier Cristiano, Edgardo Barnett, Michael Saladino, Maria Laura Sanchez-Menoyo, Jose Hodgkinson, Suzanne Rózsa Csilla Hughes, Stella Moore, Fraser Shaw, Cameron Butler, Ernest Skibina, Olga Gray, Orla Kermode, Allan G. Csépány Tünde (1956-) (neurológus, pszichiáter) Singhal, Bhim Shuey, Neil Piroska Imre Taylor, Bruce V. Simó Magdolna Sirbu, Carmen-Adella Sas Attila Butzkueven, Helmut MSBase Study Group
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7.

001-es BibID:BIBFORM083272
Első szerző:Kalincik, Tomas
Cím:Immunotherapy prevents long-term disability in relapsing multiple sclerosis over 15 years / Tomas Kalincik, Sifat Sharmin, Charles Malpas, Tim Spelman, Dana Horakova, Eva Kubala Havrdova, Maria Trojano, Guillermo Izquierdo, Alessandra Lugaresi, Alexandre Prat, Marc Girard, Pierre Duquette, Pierre Grammond, Vilija Jokubaitis, Anneke van der Walt, Francois Grand'Maison, Patrizia Sola, Diana Ferraro, Vahid Shaygannejad, Raed Alroughani, Raymond Hupperts, Murat Terzi, Cavit Boz, Jeannette Lechner-Scott, Eugenio Pucci, Vincent Van Pesch, Franco Granella, Roberto Bergamaschi, Daniele Spitaleri, Mark Slee, Steve Vucic, Radek Ampapa, Pamela McCombe, Cristina Ramo-Tello, Julie Prevost, Javier Olascoaga, Edgardo Cristiano, Michael Barnett, Maria Laura Saladino, Jose Luis Sanchez-Menoyo, Suzanne Hodgkinson, Csilla Rozsa, Stella Hughes, Fraser Moore, Cameron Shaw, Ernest Butler, Olga Skibina, Orla Gray, Allan Kermode, Tunde Csepany, Bhim Singhal, Neil Shuey, Imre Piroska, Bruce Taylor, Magdolna Simo, Carmen-Adella Sirbu, Attila Sas, Helmut Butzkueven, MSBase Study group
Dátum:2019
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:bioRxiv. - 2019 (2019), p. 1-41. -
További szerzők:Sharmin, Sifat Malpas, Charles Spelman, Tim Horakova, Dana Havrdova, Eva Trojano, Maria Izquierdo, Guillermo Lugaresi, Alessandra Prat, Alexandre Girard, Marc Duquette, Pierre Grammond, Pierre Jokubaitis, Vilija Walt, Anneke van der Grand'Maison, Francois Sola, Patrizia Ferraro, Diana Shaygannejad, Vahid Alroughani, Raed Hupperts, Raymond Terzi, Murat Boz, Cavit Lechner-Scott, Jeannette Pucci, Eugenio Pesch, Vincent van Granella, Franco Bergamaschi, Roberto Spitaleri, Daniele Slee, Mark Vucic, Steve Ampapa, Radek McCombe, Pamela Ramo-Tello, Cristina Prevost, Julie Olascoaga, Javier Cristiano, Edgardo Barnett, Michael Saladino, Maria Laura Sanchez-Menoyo, Jose Hodgkinson, Suzanne Rózsa Csilla Hughes, Stella Moore, Fraser Shaw, Cameron Butler, Ernest Skibina, Olga Gray, Orla Kermode, Allan G. Csépány Tünde (1956-) (neurológus, pszichiáter) Singhal, Bhim Shuey, Neil Piroska Imre Taylor, Bruce V. Simó Magdolna Sirbu, Carmen-Adella Sas Attila Butzkueven, Helmut MSBase Study Group
Internet cím:Intézményi repozitóriumban (DEA) tárolt változat
Borító:

8.

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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Borító:

9.

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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10.

001-es BibID:BIBFORM135460
035-os BibID:(Scopus)105002792941 (WoS)001470351300001
Első szerző:Müller, Jannis
Cím:Standardized Definition of Progression Independent of Relapse Activity (PIRA) in Relapsing-Remitting Multiple Sclerosis / Jannis Müller, Sifat Sharmin, Johannes Lorscheider, Serkan Ozakbas, Rana Karabudak, Dana Horakova, Bianca Weinstock-Guttman, Vahid Shaygannejad, Masoud Etemadifar, Raed Alroughani, Francesco Patti, Sara Eichau, Alexandre Prat, Alessandra Lugaresi, Valentina Tomassini, Allan G. Kermode, Maria Pia Amato, Recai Turkoglu, Ayse Altintas, Katherine Buzzard, Aysun Soysal, Anneke van der Walt, Helmut Butzkueven, Yolanda Blanco, Oliver Gerlach, Samia J. Khoury, Michael Barnett, Nevin John, Jeannette Lechner-Scott, Matteo Foschi, Andrea Surcinelli, Vincent van Pesch, Julie Prevost, Maria Jose Sa, Davide Maimone, Marie D'hooghe, Stella Hughes, Suzanne Hodgkinson, Chris McGuigan, Elisabetta Cartechini, Bruce Taylor, Daniele Spitaleri, Mark Slee, Pamela McCombe, Bassem Yamout, Pascal Benkert, Jens Kuhle, Ludwig Kappos, Izanne Roos, Tomas Kalincik, PGCertBiostat, MSBase Study Group
Dátum:2025
Megjegyzések:Importance: Progression independent of relapse activity (PIRA) is a significant contributor to long-term disability accumulation in relapsing-remitting multiple sclerosis (MS). Prior studies have used varying PIRA definitions, hampering the comparability of study results. Objective: To compare various definitions of PIRA. Design, setting, and participants: This cohort study involved a retrospective analysis of prospectively collected data from the MSBase registry from July 2004 to July 2023. The participants were patients with MS from 186 centers across 43 countries who had clinically definite relapsing-remitting MS, a complete minimal dataset, and 3 or more documented Expanded Disability Status Scale (EDSS) assessments. Exposure: Three-hundred sixty definitions of PIRA as combinations of the following criteria: baseline disability (fixed baseline with re-baselining after PIRA, or plus re-baselining after relapses, or plus re-baselining after improvements), minimum confirmation period (6, 12, or 24 months), confirmation magnitude (EDSS score at/above worsening score or at/above threshold compared with baseline), freedom from relapse at EDSS score worsening (90 days prior, 90 days prior and 30 days after, 180 days prior and after, since previous EDSS assessment, or since baseline), and freedom from relapse at confirmation (30 days prior, 90 days prior, 30 days before and after, or between worsening and confirmation). Main outcome and measure: For each definition, we quantified PIRA incidence and persistence (ie, absence of a 3-month confirmed EDSS improvement over ?5 years). Results: Among 87 239 patients with MS, 33 303 patients fulfilled the inclusion criteria; 24 152 (72.5%) were female and 9151 (27.5%) were male. At the first visits, the mean (SD) age was 36.4 (10.9) years; 28 052 patients (84.2%) had relapsing-remitting MS, and the median (IQR) EDSS score was 2.0 (1.0-3.0). Participants had a mean (SD) 15.1 (11.9) visits over 8.9 (5.2) years. PIRA incidence ranged from 0.141 to 0.658 events per decade and persistence from 0.753 to 0.919, depending on the definition. In particular, the baseline and confirmation period influenced PIRA detection. The following definition yielded balanced incidence and persistence: a significant disability worsening compared with a baseline (reset after each PIRA event, relapse, and EDSS score improvement), in absence of relapses since the last EDSS assessment, confirmed with EDSS scores (not preceded by relapses within 30 days) that remained above the worsening threshold for at least 12 months. Conclusion and relevance: Incidence and persistence of PIRA are determined by the definition used. The proposed standardized definition aims to enhance comparability among studies.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:JAMA Neurology. - 82 : 6 (2025), p. 614-625. -
További szerzők:Sharmin, Sifat Lorscheider, Johannes Ozakbas, Serkan Karabudak, Rana Horakova, Dana Weinstock-Guttman, Bianca Shaygannejad, Vahid Etemadifar, Masoud Alroughani, Raed Patti, Francesco Eichau, Sara Prat, Alexandre Lugaresi, Alessandra Tomassini, Valentina Kermode, Allan G. Amato, Maria Pia Turkoglu, Recai Altintas, Ayse Buzzard, Katherine Soysal, Aysun Walt, Anneke van der Butzkueven, Helmut Blanco, Yolanda Gerlach, Oliver Khoury, Samia J. Barnett, Michael John, Nevin Lechner-Scott, Jeannette Foschi, Matteo Surcinelli, Andrea Pesch, Vincent van Prevost, Julie Sa, Maria Jose Maimone, Davide D'hooghe, Marie Hughes, Stella Hodgkinson, Suzanne McGuigan, Christopher Cartechini, Elisabetta Taylor, Bruce V. Spitaleri, Daniele Slee, Mark McCombe, Pamela Yamout, Bassem Benkert, Pascal Kuhle, Jens Kappos, Ludwig Roos, Izanne Kalincik, Tomas Csépány Tünde (1956-) (neurológus, pszichiáter) MSBase Study Group PGCertBiostat
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11.

001-es BibID:BIBFORM132947
035-os BibID:(scopus)105011413182 (wos)001536298500003
Első szerző:Pirmani, Ashkan
Cím:Personalized federated learning for predicting disability progression in multiple sclerosis using real-world routine clinical data / Pirmani Ashkan, De Brouwer Edward, Arany Ádám, Oldenhof Martijn, Passemiers Antoine, Faes Axel, Kalincik Tomas, Ozakbas Serkan, Gouider Riadh, Willekens Barbara, Horakova Dana, Havrdova Eva Kubala, Patti Francesco, Prat Alexandre, Lugaresi Alessandra, Tomassini Valentina, Grammond Pierre, Cartechini Elisabetta, Roos Izanne, Boz Cavit, Alroughani Raed, Amato Maria Pia, Buzzard Katherine, Lechner-Scott Jeannette, Guimaraes Joana, Solaro Claudio, Gerlach Oliver, Soysal Aysun, Kuhle Jens, Sanchez-Menoyo Jose Luis, Spitaleri Daniele, Csepany Tunde, Van Wijmeersch Bart, Ampapa Radek, Prevost Julie, Khoury Samia J., Van Pesch Vincent, John Nevin, Maimone Davide, Weinstock-Guttman Bianca, Laureys Guy, McCombe Pamela, Blanco Yolanda, Altintas Ayse, Al-Asmi Abdullah, Garber Justin, Van der Walt Anneke, Butzkueven Helmut, de Gans Koen, Rozsa Csilla, Taylor Bruce, Al-Harbi Talal, Sas Attila, Rajda Cecilia, Gray Orla, Decoo Danny, Carroll William M., Kermode Allan G., Fabis-Pedrini Marzena, Mason Deborah, Perez-Sempere Angel, Simu Mihaela, Shuey Neil, Singhal Bhim, Cauchi Marija, Hardy Todd A., Ramanathan Sudarshini, Lalive Patrice, Sirbu Carmen-Adella, Hughes Stella, Castillo Trivino Tamara, Peeters Liesbet M., Moreau Yves
Dátum:2025
ISSN:2398-6352
Megjegyzések:Early prediction of disability progression in multiple sclerosis (MS) remains challenging despite its critical importance for therapeutic decision-making. We present the first systematic evaluation of personalized federated learning (PFL) for 2-year MS disability progression prediction, leveraging multi-center real-world data from over 26,000 patients. While conventional federated learning (FL) enables privacy-aware collaborative modeling, it remains vulnerable to institutional data heterogeneity. PFL overcomes this challenge by adapting shared models to local data distributions without compromising privacy. We evaluated two personalization strategies: a novel AdaptiveDualBranchNet architecture with selective parameter sharing, and personalized fine-tuning of global models, benchmarked against centralized and client-specific approaches. Baseline FL underperformed relative to personalized methods, whereas personalization significantly improved performance, with personalized FedProx and FedAVG achieving ROC-AUC scores of 0.8398 ? 0.0019 and 0.8384 ? 0.0014, respectively. These findings establish personalization as critical for scalable, privacy-aware clinical prediction models and highlight its potential to inform earlier intervention strategies in MS and beyond.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:npj Digital Medicine. - 8 : 1 (2025), p. 1-15. -
További szerzők:De Brouwer, Edward Arany Ádám Oldenhof, Martijn Passemiers, Antoine Faes, Axel Kalincik, Tomas Ozakbas, Serkan Gouider, Riadh Willekens, Barbara Horakova, Dana Havrdova, Eva Patti, Francesco Prat, Alexandre Lugaresi, Alessandra Tomassini, Valentina Grammond, Pierre Cartechini, Elisabetta Roos, Izanne Boz, Cavit Alroughani, Raed Amato, Maria Pia Buzzard, Katherine Lechner-Scott, Jeannette Guimaraes, Joana Solaro, Claudio Gerlach, Oliver Soysal, Aysun Kuhle, Jens Sanchez-Menoyo, Jose Spitaleri, Daniele Csépány Tünde (1956-) (neurológus, pszichiáter) Wijmeersch, Bart Van Ampapa, Radek Prevost, Julie Khoury, Samia J. Pesch, Vincent van John, Nevin Maimone, Davide Weinstock-Guttman, Bianca Laureys, Guy (Universitary Hospital Ghent) McCombe, Pamela Blanco, Yolanda Altintas, Ayse Al-Asmi, Abdullah Garber, Justin Walt, Anneke van der Butzkueven, Helmut de Gans, Koen Rózsa Csilla Taylor, Bruce V. Al-Harbi, Talal Sas Attila Rajda Cecília Gray, Orla Decoo, Danny Carroll, William M. Kermode, Allan G. Fabis-Pedrini, Marzena Mason, Deborah Perez-Sempere, Angel Simu, Mihaela Shuey, Neil Singhal, Bhim Cauchi, Marija Hardy, Todd A. Ramanathan, Sudarshini Lalive, Patrice H. Sirbu, Carmen-Adella Hughes, Stella Castillo Triviño, Tamara Peeters, Liesbet Moreau, Yves
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Borító:

12.

001-es BibID:BIBFORM114473
035-os BibID:(Scopus)85148502832 (WoS)000951172400001
Első szerző:Roos, Izanne
Cím:Comparative effectiveness in multiple sclerosis : A methodological comparison / Roos Izanne, Diouf Ibrahima, Sharmin Sifat, 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, 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, McGuigan Chris, Cartechini Elisabetta, Barnett Michael, Hughes Stella, Sa Maria José, Solaro Claudio, Ramo-Tello Cristina, Hodgkinson Suzanne, Spitaleri Daniele, Soysal Aysun, Petersen Thor, Granella Franco, de Gans Koen, McCombe Pamela, Ampapa Radek, Van Wijmeersch Bart, van der Walt Anneke, Butzkueven Helmut, Prevost Julie, Sanchez-Menoyo Jose Luis, Laureys Guy, Gouider Riadh, Castillo-Trivino Tamara, Gray Orla, Aguera-Morales Eduardo, Al-Asmi Abdullah, Shaw Cameron, Deri Norma, Al-Harbi Talal, Fragoso Yara, Csepany Tunde, Sempere Angel Perez, Trevino-Frenk Irene, Schepel Jan, Moore Fraser, Malpas Charles, Kalincik Tomas, MSBase study group
Dátum:2023
ISSN:1352-4585
Megjegyzések: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.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Observational
causal inference
multiple sclerosis
Megjelenés:Multiple Sclerosis. - 29 : 3 (2023), p. 326-332. -
További szerzők:Diouf, Ibrahima Sharmin, Sifat 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 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 McGuigan, Christopher Cartechini, Elisabetta Barnett, Michael Hughes, Stella Sá, Maria José Solaro, Claudio Ramo-Tello, Cristina Hodgkinson, Suzanne Spitaleri, Daniele Soysal, Aysun Petersen, Thor Granella, Franco de Gans, Koen McCombe, Pamela Ampapa, Radek Wijmeersch, Bart Van Walt, Anneke van der Butzkueven, Helmut Prevost, Julie Sanchez-Menoyo, Jose Laureys, Guy 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) Sempere, Perez A. Trevino-Frenk, Irene Schepel, Jan Moore, Fraser Malpas, Charles Kalincik, Tomas MSBase Study Group
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