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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:BIBFORM126277
035-os BibID:(scopus)85201493485
Első szerző:De Brouwer, Edward
Cím:Machine-learning-based prediction of disability progression in multiple sclerosis : an observational, international, multi-center study / De Brouwer E., Becker T., Werthen-Brabants L., Dewulf P., Iliadis D., Dekeyser C., Laureys G., Van Wijmeersch B., Popescu V., Dhaene T., Deschrijver D., Waegeman W., De Baets B., Stock M., Horakova D., Patti F., Izquierdo G., Eichau S., Girard M., Prat A., Lugaresi A., Grammond P., Kalincik T., Alroughani R., Grand'Maison F., Skibina O., Terzi M., Lechner-Scott J., Gerlach O., Khoury S. J., Cartechini E., Van Pesch V., Sa M. J., Weinstock-Guttman B., Blanco Y., Ampapa R., Spitaleri D., Solaro C., Maimone D., Soysal A., Iuliano G., Gouider R., Castillo-Trivino T., Sánchez-Menoyo J. L., Laureys G., van der Walt A., Oh J., Aguera-Morales E., Altintas A., Al-Asmi A., de Gans K., Fragoso Y., Csepany T., Hodgkinson S., Deri N., Al-Harbi T., Taylor B., Gray O., Lalive P., Rozsa C., McGuigan C., Kermode A., Sempere A. P., Mihaela S., Simo M., Hardy T., Decoo D., Hughes S., Grigoriadis N., Sas A., Vella N., Moreau Y., Peeters L.
Dátum:2024
ISSN:2767-3170
Megjegyzések:Background Disability progression is a key milestone in the disease evolution of people with multiple sclerosis (PwMS). Prediction models of the probability of disability progression have not yet reached the level of trust needed to be adopted in the clinic. A common benchmark to assess model development in multiple sclerosis is also currently lacking. Methods Data of adult PwMS with a follow-up of at least three years from 146 MS centers, spread over 40 countries and collected by the MSBase consortium was used. With basic inclusion criteria for quality requirements, it represents a total of 15, 240 PwMS. External validation was performed and repeated five times to assess the significance of the results. Transparent Reporting for Individual Prognosis Or Diagnosis (TRIPOD) guidelines were followed. Confirmed disability progression after two years was predicted, with a confirmation window of six months. Only routinely collected variables were used such as the expanded disability status scale, treatment, relapse information, and MS course. To learn the probability of disability progression, state-of-the-art machine learning models were investigated. The discrimination performance of the models is evaluated with the area under the receiver operator curve (ROC-AUC) and under the precision recall curve (AUC-PR), and their calibration via the Brier score and the expected calibration error. All our preprocessing and model code are available at https://gitlab.com/edebrouwer/ms_benchmark, making this task an ideal benchmark for predicting disability progression in MS. Findings Machine learning models achieved a ROC-AUC of 0?71 ? 0?01, an AUC-PR of 0?26 ? 0?02, a Brier score of 0?1 ? 0?01 and an expected calibration error of 0?07 ? 0?04. The history of disability progression was identified as being more predictive for future disability progression than the treatment or relapses history. Conclusions Good discrimination and calibration performance on an external validation set is achieved, using only routinely collected variables. This suggests machine-learning models can reliably inform clinicians about the future occurrence of progression and are mature for a clinical impact study.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Machine learning
disability progression
multiple sclerosis
Megjelenés:PLOS Digital Health. - 3 : 7 (2024), p. 1-25. -
További szerzők:Becker, Thijs Werthen-Brabants, Lorin Dewulf, Pieter Iliadis, Dimitrios Dekeyser, Cathérine Laureys, Guy Wijmeersch, Bart Van Popescu, Veronica Dhaene, Tom Deschrijver, Dirk Waegeman, Willem De Baets, Bernard Stock, Michael J. Horakova, Dana Patti, Francesco Izquierdo, Guillermo Eichau, Sara Girard, Marc Prat, Alexandre Lugaresi, Alessandra Grammond, Pierre Kalincik, Tomas Alroughani, Raed Grand'Maison, Francois Skibina, Olga Terzi, Murat Lechner-Scott, Jeannette Gerlach, Oliver Khoury, Samia J. Cartechini, Elisabetta Pesch, Vincent van Sá, Maria José Weinstock-Guttman, Bianca Blanco, Yolanda Ampapa, Radek Spitaleri, Daniele Solaro, Claudio Maimone, Davide Soysal, Aysun Iuliano, Gerardo Gouider, Riadh Castillo Triviño, Tamara Sanchez-Menoyo, Jose Laureys, Guy (Universitary Hospital Ghent) Walt, Anneke van der Oh, Jiwon Aguera-Morales, Eduardo Altintas, Ayse Al-Asmi, Abdullah de Gans, Koen Fragoso, Yara Csépány Tünde (1956-) (neurológus, pszichiáter) Hodgkinson, Suzanne Deri, Norma Al-Harbi, Talal Taylor, Bruce V. Gray, Orla Lalive, Patrice H. Rózsa Csilla McGuigan, Christopher Kermode, Allan G. Sempere, Perez A. Mihaela, Simu Simó Magdolna Hardy, Todd A. Decoo, Danny Hughes, Stella Grigoriadis, Nikolaos Sas Attila Vella Norbert Moreau, Yves Peeters, Liesbet
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3.

001-es BibID:BIBFORM126270
035-os BibID:(scopus)85190160325 (wos)001201473600001
Első szerző:Dekeyser, Cathérine
Cím:Routine CSF parameters as predictors of disease course in multiple sclerosis : an MSBase cohort study / Dekeyser C., Hautekeete M., Cambron M., Van Pesch V., Patti F., Kuhle J., Khoury S., Lechner Scott J., Gerlach O., Lugaresi A., Maimone D., Surcinelli A., Grammond P., Kalincik T., Habek M., Willekens B., Macdonell R., Lalive P., Csepany T., Butzkueven H., Boz C., Tomassini V., Foschi M., Sánchez-Menoyo J. L., Altintas A., Mrabet S., Iuliano G., Sa M. J., Alroughani R., Karabudak R., Aguera-Morales E., Gray O., de Gans K., van der Walt A., McCombe P. A., Deri N., Garber J., Al-Asmi A., Skibina O., Duquette P., Cartechini E., Spitaleri D., Gouider R., Soysal A., Van Hijfte L., Slee M., Amato M. P., Buzzard K., Laureys G.
Dátum:2024
ISSN:0022-3050 1468-330X
Megjegyzések:Background: It remains unclear whether routine cerebrospinal fluid (CSF) parameters can serve as predictors of multiple sclerosis (MS) disease course. Methods: This large-scale cohort study included persons with MS with CSF data documented in the MSBase registry. CSF parameters to predict time to reach confirmed Expanded Disability Status Scale (EDSS) scores 4, 6 and 7 and annualised relapse rate in the first 2 years after diagnosis (ARR2) were assessed using (cox) regression analysis. Results: In total, 11 245 participants were included of which 93.7% (n=10 533) were persons with relapsing-remitting MS (RRMS). In RRMS, the presence of CSF oligoclonal bands (OCBs) was associated with shorter time to disability milestones EDSS 4 (adjusted HR=1.272 (95% CI, 1.089 to 1.485), p=0.002), EDSS 6 (HR=1.314 (95% CI, 1.062 to 1.626), p=0.012) and EDSS 7 (HR=1.686 (95% CI, 1.111 to 2.558), p=0.014). On the other hand, the presence of CSF pleocytosis (?5 cells/?L) increased time to moderate disability (EDSS 4) in RRMS (HR=0.774 (95% CI, 0.632 to 0.948), p=0.013). None of the CSF variables were associated with time to disability milestones in persons with primary progressive MS (PPMS). The presence of CSF pleocytosis increased ARR2 in RRMS (adjusted R2=0.036, p=0.015). Conclusions: In RRMS, the presence of CSF OCBs predicts shorter time to disability milestones, whereas CSF pleocytosis could be protective. This could however not be found in PPMS. CSF pleocytosis is associated with short-term inflammatory disease activity in RRMS. CSF analysis provides prognostic information which could aid in clinical and therapeutic decision-making.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
CLINICAL NEUROLOGY
CSF
MULTIPLE SCLEROSIS
NEUROIMMUNOLOGY
Megjelenés:Journal of Neurology, Neurosurgery, and Psychiatry . - 95 : 11 (2024), p. 1021-1031. -
További szerzők:Hautekeete, Matthias Cambron, Melissa Pesch, Vincent van Patti, Francesco Kuhle, Jens Khoury, Samia J. Lechner Scott, Jeanette Gerlach, Oliver Lugaresi, Alessandra Maimone, Davide Surcinelli, Andrea Grammond, Pierre Kalincik, Tomas Habek, Mario Willekens, Barbara Macdonell, Richard Lalive, Patrice H. Csépány Tünde (1956-) (neurológus, pszichiáter) Butzkueven, Helmut Boz, Cavit Tomassini, Valentina Foschi, Matteo Sanchez-Menoyo, Jose Altintas, Ayse Mrabet, Saloua Iuliano, Gerardo Sá, Maria José Alroughani, Raed Karabudak, Rana Aguera-Morales, Eduardo Gray, Orla de Gans, Koen Walt, Anneke van der McCombe, Pamela Deri, Norma Garber, Justin Al-Asmi, Abdullah Skibina, Olga Duquette, Pierre Cartechini, Elisabetta Spitaleri, Daniele Gouider, Riadh Soysal, Aysun Van Hijfte, Liesbeth Slee, Mark Amato, Maria Pia Buzzard, Katherine Laureys, Guy
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4.

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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5.

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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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

6.

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:

7.

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:

8.

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:

9.

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:

10.

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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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

11.

001-es BibID:BIBFORM135463
035-os BibID:(Scopus)105025260703 (WoS)001643679600001
Első szerző:Lizak, Nathaniel
Cím:Managing reactivation of multiple sclerosis during treatment with natalizumab / Nathaniel Lizak, Sifat Sharmin, Dana Horáková, Eva Kubala Havrdova, Sara Eichau, Anneke van der Walt, Helmut Butzkueven, Jeannette Lechner-Scott, Katherine Buzzard, Olga Skibina, Oliver Gerlach, Alexandre Prat, Marc Girard, Pierre Duquette, Raed Alroughani, Francesco Patti, Francois Grand'Maison, Maria José Sá, Eduardo Aguera-Morales, Suzanne Hodgkinson, Pierre Grammond, Jens Kuhle, Bassem Yamout, Samia J. Khoury, Serkan Ozakbas, Tunde Csepany, Nevin John, Guy Laureys, Murat Terzi, Maria Pia Amato, Cavit Boz, Abdullah Al-Asmi, Elisabetta Cartechini, Riadh Gouider, Saloua Mrabet, Izanne Roos, Tomas Kalincik, MSBase Study Group
Dátum:2025
ISSN:1352-4585
Megjegyzések:Background: Following natalizumab failure, it is unknown whether switching to alternative high-efficacy therapies offers superior effectiveness over continuing natalizumab. Objective: To compare different treatment strategies following natalizumab failure. Methods: Patients suffering a relapse during natalizumab treatment with adequate follow-up were identified from the MSBase registry. Following natalizumab failure, natalizumab continuation was compared to switching to anti-CD20 therapies/alemtuzumab/lower-efficacy therapies and treatment discontinuation. The primary outcome was the risk of further relapses. Secondary outcomes included risk of subsequent magnetic resonance imaging (MRI) activity, confirmed disability worsening and disease-activity-free survival. Multivariable proportional hazards models compared outcomes during time-varying therapy exposures. Four sensitivity analyses were conducted with varied inclusion criteria and treatment failure definitions. Results: Of 1553 patients experiencing a relapse during natalizumab treatment, 1037 met the inclusion criteria. Following natalizumab failure, switch to anti-CD20 therapy was associated with a lower relapse risk (heart rate (HR) = 0.48, 95% confidence interval (CI) = 0.27?0.84) compared to continuing natalizumab; no differences were observed in MRI or disability outcomes. Treatment de-escalation or cessation was associated with increased relapse risk (HR = 1.46, 95% CI = 1.15?1.85; HR = 2.08, 95% CI = 1.223.55, respectively). We did not find evidence of a difference for switching to alemtuzumab. Sensitivity analyses replicated primary findings. Conclusion: This exploratory study indicates that switching to anti-CD20 therapies following natalizumab failure is associated with a >50% reduction in relapse risk. No differences were seen in secondary outcomes, despite consistent trends. Clinicians may consider anti-CD20 therapies following natalizumab failure, noting further research is needed to confirm differences in MRI and disability outcomes.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Multiple sclerosis
natalizumab
disease-modifying treatment
relapses
treatment failure
Megjelenés:Multiple Sclerosis. - 32 : 1 (2025), p. 121-133. -
További szerzők:Sharmin, Sifat Horakova, Dana Havrdova, Eva Eichau, Sara Walt, Anneke van der Butzkueven, Helmut Lechner-Scott, Jeannette Buzzard, Katherine Skibina, Olga Gerlach, Oliver Prat, Alexandre Girard, Marc Duquette, Pierre Alroughani, Raed Patti, Francesco Grand'Maison, Francois Sá, Maria José Aguera-Morales, Eduardo Hodgkinson, Suzanne Grammond, Pierre Kuhle, Jens Yamout, Bassem Khoury, Samia J. Ozakbas, Serkan Csépány Tünde (1956-) (neurológus, pszichiáter) John, Nevin Laureys, Guy Terzi, Murat Amato, Maria Pia Boz, Cavit Al-Asmi, Abdullah Cartechini, Elisabetta Gouider, Riadh Mrabet, Saloua Roos, Izanne Kalincik, Tomas MSBase Study Group
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

12.

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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