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1.
001-es BibID:
BIBFORM103014
035-os BibID:
(Scopus)85107568058 (Wos)000687405300017 (cikkazonosító)103012
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
Van Wijmeersch, Bart
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
2.
001-es BibID:
BIBFORM103017
035-os BibID:
(Wos)000685503300008 (Scopus)85107912293 (cikkazonosító)106180
Első szerző:
De Brouwer, Edward
Cím:
Longitudinal machine learning modeling of MS patient trajectories improves predictions of disability progression / De Brouwer Edward, Becker Thijs, Moreau Yves, Havrdova Eva Kubala, Trojano Maria, Eichau Sara, Ozakbas Serkan, Onofrj Marco, Grammond Pierre, Kuhle Jens, Kappos Ludwig, Sola Patrizia, Cartechini Elisabetta, Lechner-Scott Jeannette, Alroughani Raed, Gerlach Oliver, Kalincik Tomas, Granella Franco, Grand'Maison Francois, Bergamaschi Roberto, José Sá Maria, Van Wijmeersch Bart, Soysal Aysun, Sanchez-Menoyo Jose Luis, Solaro Claudio, Boz Cavit, Iuliano Gerardo, Buzzard Katherine, Aguera-Morales Eduardo, Terzi Murat, Trivio Tamara Castillo, Spitaleri Daniele, Van Pesch Vincent, Shaygannejad Vahid, Moore Fraser, Oreja-Guevara Celia, Maimone Davide, Gouider Riadh, Csepany Tunde, Ramo-Tello Cristina, Peeters Liesbet
Dátum:
2021
ISSN:
0169-2607
Megjegyzések:
Background and Objectives: Research in Multiple Sclerosis (MS) has recently focused on extracting knowledge from real-world clinical data sources. This type of data is more abundant than data produced during clinical trials and potentially more informative about real-world clinical practice. However, this comes at the cost of less curated and controlled data sets. In this work we aim to predict disability progression by optimally extracting information from longitudinal patient data in the real-world setting, with a special focus on the sporadic sampling problem. Methods: We use machine learning methods suited for patient trajectories modeling, such as recurrent neural networks and tensor factorization. A subset of 6682 patients from the MSBase registry is used. Results: We can predict disability progression of patients in a two-year horizon with an ROC-AUC of 0.85, which represents a 32% decrease in the ranking pair error (1-AUC) compared to reference methods using static clinical features. Conclusions: Compared to the models available in the literature, this work uses the most complete patient history for MS disease progression prediction and represents a step forward towards AI-assisted precision medicine in MS.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:
Computer Methods And Programs In Biomedicine. - 208 (2021), p. 1-14. -
További szerzők:
Becker, Thijs
Moreau, Yves
Havrdova, Eva
Trojano, Maria
Eichau, Sara
Ozakbas, Serkan
Onofrj, Marco
Grammond, Pierre
Kuhle, Jens
Kappos, Ludwig
Sola, Patrizia
Cartechini, Elisabetta
Lechner-Scott, Jeannette
Alroughani, Raed
Gerlach, Oliver
Kalincik, Tomas
Granella, Franco
Grand'Maison, Francois
Bergamaschi, Roberto
José Sá, Maria
Van Wijmeersch, Bart
Soysal, Aysun
Sanchez-Menoyo, Jose
Solaro, Claudio
Boz, Cavit
Iuliano, Gerardo
Buzzard, Katherine
Aguera-Morales, Eduardo
Terzi, Murat
Trivio, Tamara Castillo
Spitaleri, Daniele
Pesch, Vincent van
Shaygannejad, Vahid
Moore, Fraser
Oreja-Guevara, Celia
Maimone, Davide
Gouider, Riadh
Csépány Tünde (1956-) (neurológus, pszichiáter)
Ramo-Tello, Cristina
Peeters, Liesbet
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
3.
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
4.
001-es BibID:
BIBFORM107545
035-os BibID:
(cikkazonosító)15706 (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
Van Wijmeersch, Bart
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
5.
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
6.
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:
Saját polcon:
7.
001-es BibID:
BIBFORM103015
035-os BibID:
(cikkazonosító)155 (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
Van Wijmeersch, Bart
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
8.
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
Van Wijmeersch, Bart
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
9.
001-es BibID:
BIBFORM103566
035-os BibID:
(WoS)000874431500025 (Scopus)85141339945
Első szerző:
Roos, Izanne
Cím:
Disease Reactivation After Cessation of Disease-Modifying Therapy in Patients With Relapsing-Remitting Multiple Sclerosis / Roos Izanne, Malpas Charles, Leray Emmanuelle, Casey Romain, Horakova Dana, Havrdova Eva Kubala, Debouverie Marc, Patti Francesco, De Seze Jerome, Izquierdo Guillermo, Eichau Sara, Edan Gilles, Prat Alexandre, Girard Marc, Ozakbas Serkan, Grammond Pierre, Zephir Helene, Ciron Jonathan, Maillart Elisabeth, Moreau Thibault, Amato Maria Pia, Labauge Pierre, Alroughani Raed, Buzzard Katherine, Skibina Olga, Terzi Murat, Laplaud David Axel, Berger Eric, Grand'Maison Francois, Lebrun-Frenay Christine, Cartechini Elisabetta, Boz Cavit, Lechner-Scott Jeannette, Clavelou Pierre, Stankoff Bruno, Prevost Julie, Kappos Ludwig, Pelletier Jean, Shaygannejad Vahid, Yamout Bassem I., Khoury Samia J., Gerlach Oliver, Spitaleri Daniele L. A., Van Pesch Vincent, Gout Olivier, Turkoglu Recai, Heinzlef Olivier, Thouvenot Eric, McCombe Pamela Ann, Soysal Aysun, Bourre Bertrand, Slee Mark, Castillo-Trivino Tamara, Bakchine Serge, Ampapa Radek, Butler Ernest Gerard, Wahab Abir, Macdonell Richard A., Aguera-Morales Eduardo, Cabre Philippe, Ben Nasr Haifa, Van der Walt Anneke, Laureys Guy, Van Hijfte Liesbeth, Ramo-Tello Cristina M., Maubeuge Nicolas, Hodgkinson Suzanne, Sánchez-Menoyo José Luis, Barnett Michael H., Labeyrie Celine, Vucic Steve, Sidhom Youssef, Gouider Riadh, Csepany Tunde, Sotoca Javier, de Gans Koen, Al-Asmi Abdullah, Fragoso Yara Dadalti, Vukusic Sandra, Butzkueven Helmut, Kalincik Tomas, MSBase and OFSEP
Dátum:
2022
ISSN:
0028-3878 1526-632X
Megjegyzések:
Objectives: To evaluate the rate of return of disease activity after cessation of multiple sclerosis (MS) disease-modifying therapy. Methods: This was a retrospective cohort study from two large observational MS registries: MSBase and OFSEP. Patients with relapsing-remitting MS who had ceased a disease-modifying therapy and were followed up for the subsequent 12-months were included in the analysis. The primary study outcome was annualised relapse rate in the 12 months after disease-modifying therapy discontinuation stratified by patients who did, and did not, commence a subsequent therapy. The secondary endpoint was the predictors of first relapse and disability accumulation after treatment discontinuation. Results: 14,213 patients, with 18,029 eligible treatment discontinuation epochs, were identified for seven therapies. Annualised rates of relapse (ARR) started to increase 2-months after natalizumab cessation (month 2-4 ARR, 95% confidence interval): 0.47, 0.43-0.51). Commencement of a subsequent therapy within 2-4 months reduced the magnitude of disease reactivation (mean ARR difference: 0.15, 0.08-0.22). After discontinuation of fingolimod, rates of relapse increased overall (month 1-2 ARR: 0.80, 0.70-0.89), and stabilised faster in patients who started a new therapy within 1-2 months (mean ARR difference: 0.14, -0.01-0.29). Magnitude of disease reactivation for other therapies was low, but reduced further by commencement of another treatment 1-10 months after treatment discontinuation. Predictors of relapse were higher relapse rate in the year before cessation, female sex, younger age and higher EDSS. Commencement of a subsequent therapy reduced both the risk of relapse (HR 0.76, 95%CI 0.72-0.81) and disability accumulation (0.73, 0.65-0.80). Conclusion: The rate of disease reactivation after treatment cessation differs among MS treatments, with the peaks of relapse activity ranging from 1 to 10 months in untreated cohorts that discontinued different therapies. These results suggest that untreated intervals should be minimised after stopping anti-trafficking therapies (natalizumab and fingolimod). Classification of evidence: This study provides class III that disease reactivation occurs within months of discontinuation of multiple sclerosis disease-modifying therapies. Risk of disease activity is reduced by commencement of a subsequent therapy.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:
Neurology. - 99 : 17 (2022), p. e1926-e1944. -
További szerzők:
Malpas, Charles
Leray, Emmanuelle
Casey, Romain
Horakova, Dana
Havrdova, Eva
Debouverie, Marc
Patti, Francesco
De Seze, Jérôme
Izquierdo, Guillermo
Eichau, Sara
Edan, Gilles
Prat, Alexandre
Girard, Marc
Ozakbas, Serkan
Grammond, Pierre
Zephir, Hélène
Ciron, Jonathan
Maillart, Elisabeth
Moreau, Thibault
Amato, Maria Pia
Labauge, Pierre
Alroughani, Raed
Buzzard, Katherine
Skibina, Olga
Terzi, Murat
Laplaud, David
Berger, Eric
Grand'Maison, Francois
Lebrun-Frenay, Christine
Cartechini, Elisabetta
Boz, Cavit
Lechner-Scott, Jeannette
Clavelou, Pierre
Stankoff, Bruno
Prevost, Julie
Kappos, Ludwig
Pelletier, Jean
Shaygannejad, Vahid
Yamout, Bassem
Khoury, Samia J.
Gerlach, Oliver
Spitaleri, Daniele L. A.
Pesch, Vincent van
Gout, Olivier
Turkoglu, Recai
Heinzlef, Olivier
Thouvenot, Eric
McCombe, Pamela
Soysal, Aysun
Bourre, Bertrand
Slee, Mark
Castillo Triviño, Tamara
Bakchine, Serge
Ampapa, Radek
Butler, Ernest
Wahab, Abir
Macdonell, Richard
Aguera-Morales, Eduardo
Cabre, Philippe
Ben Nasr, Haifa
Walt, Anneke van der
Laureys, Guy
Van Hijfte, Liesbeth
Ramo-Tello, Cristina
Maubeuge, Nicolas
Hodgkinson, Suzanne
Sanchez-Menoyo, Jose
Barnett, Michael
Labeyrie, Céline
Vucic, Steve
Sidhom, Youssef
Gouider, Riadh
Csépány Tünde (1956-) (neurológus, pszichiáter)
Sotoca, Javier
de Gans, Koen
Al-Asmi, Abdullah
Fragoso, Yara
Vukusic, Sandra
Butzkueven, Helmut
Kalincik, Tomas
OFSEP and the MSBase investigators
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
10.
001-es BibID:
BIBFORM103021
035-os BibID:
(Wos)000809732300001 (Scopus)85131507832
Első szerző:
Sharmin, Sifat
Cím:
Confirmed disability progression as a marker of permanent disability in multiple sclerosis / Sharmin Sifat, Bovis Francesca, Malpas Charles, Horakova Dana, Havrdova Eva Kubala, Izquierdo Guillermo, Eichau Sara, Trojano Maria, Prat Alexandre, Girard Marc, Duquette Pierre, Onofrj Marco, Lugaresi Alessandra, Grand'Maison Francois, Grammond Pierre, Sola Patrizia, Ferraro Diana, Terzi Murat, Gerlach Oliver, Alroughani Raed, Boz Cavit, Shaygannejad Vahid, van Pesch Vincent, Cartechini Elisabetta, Kappos Ludwig, Lechner-Scott Jeannette, Bergamaschi Roberto, Turkoglu Recai, Solaro Claudio, Iuliano Gerardo, Granella Franco, Van Wijmeersch Bart, Spitaleri Daniele, Slee Mark, McCombe Pamela, Prevost Julie, Ampapa Radek, Ozakbas Serkan, Sanchez-Menoyo Jose Luis, Soysal Aysun, Vucic Steve, Petersen Thor, de Gans Koen, Butler Ernest, Hodgkinson Suzanne, Sidhom Youssef, Gouider Riadh, Cristiano Edgardo, Castillo-Trivino Tamara, Saladino Maria Laura, Barnett Michael, Moore Fraser, Rozsa Csilla, Yamout Bassem, Skibina Olga, van der Walt Anneke, Buzzard Katherine, Gray Orla, Hughes Stella, Sempere Angel Perez, Singhal Bhim, Fragoso Yara, Shaw Cameron, Kermode Allan, Taylor Bruce, Simo Magdolna, Shuey Neil, Al-Harbi Talal, Macdonell Richard, Dominguez Jose Andres, Csepany Tunde, Sirbu Carmen-Adella, Sormani Maria Pia, Butzkueven Helmut, Kalincik Tomas
Dátum:
2022
ISSN:
1351-5101
Megjegyzések:
Background and purpose: The prevention of disability over the long term is the main treatment goal in multiple sclerosis (MS); however, randomized clinical trials evaluate only short-term treatment effects on disability. This study aimed to define criteria for 6-month confirmed disability progression events of MS with a high probability of resulting in sustained long-term disability worsening. Methods: In total, 14,802 6- month confirmed disability progression events were identified in 8741 patients from the global MSBase registry. For each 6-month confirmed progression event (13,321 in the development and 1481 in the validation cohort), a sustained progression score was calculated based on the demographic and clinical characteristics at the time of progression that were predictive of long-term disability worsening. The score was externally validated in the Cladribine Tablets Treating Multiple Sclerosis Orally (CLARITY) trial. Results: The score was based on age, sex, MS phenotype, relapse activity, disability score and its change from baseline, number of affected functional system domains and worsening in six of the domains. In the internal validation cohort, a 61% lower chance of improvement was estimated with each unit increase in the score (hazard ratio 0.39, 95% confidence interval 0.29?0.52; discriminatory index 0.89). The proportions of progression events sustained at 5 years stratified by the score were 1: 72%; 2: 88%; 3: 94%; 4: 100%. The results of the CLARITY trial were confirmed for reduction of disability progression that was >88% likely to be sustained (events with score ?1.5).Conclusions: Clinicodemographic characteristics of 6-month confirmed disability progression events identify those at high risk of sustained long-term disability. This knowledge will allow future trials to better assess the effect of therapy on long-term disability accrual.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:
European Journal Of Neurology. - 29 : 8 (2022), p. 2321-2334. -
További szerzők:
Bovis, Francesca
Malpas, Charles
Horakova, Dana
Havrdova, Eva
Izquierdo, Guillermo
Eichau, Sara
Trojano, Maria
Prat, Alexandre
Girard, Marc
Duquette, Pierre
Onofrj, Marco
Lugaresi, Alessandra
Grand'Maison, Francois
Grammond, Pierre
Sola, Patrizia
Ferraro, Diana
Terzi, Murat
Gerlach, Oliver
Alroughani, Raed
Boz, Cavit
Shaygannejad, Vahid
Pesch, Vincent van
Cartechini, Elisabetta
Kappos, Ludwig
Lechner-Scott, Jeannette
Bergamaschi, Roberto
Turkoglu, Recai
Solaro, Claudio
Iuliano, Gerardo
Granella, Franco
Van Wijmeersch, Bart
Spitaleri, Daniele
Slee, Mark
McCombe, Pamela
Prevost, Julie
Ampapa, Radek
Ozakbas, Serkan
Sanchez-Menoyo, Jose
Soysal, Aysun
Vucic, Steve
Petersen, Thor
de Gans, Koen
Butler, Ernest
Hodgkinson, Suzanne
Sidhom, Youssef
Gouider, Riadh
Cristiano, Edgardo
Castillo Triviño, Tamara
Saladino, Maria Laura
Barnett, Michael
Moore, Fraser
Rózsa Csilla
Yamout, Bassem
Skibina, Olga
Walt, Anneke van der
Buzzard, Katherine
Gray, Orla
Hughes, Stella
Sempere, Perez A.
Singhal, Bhim
Fragoso, Yara
Shaw, Cameron
Kermode, Allan G.
Taylor, Bruce V.
Simó Magdolna
Shuey, Neil
Al-Harbi, Talal
Macdonell, Richard
Dominguez, Jose Andres
Csépány Tünde (1956-) (neurológus, pszichiáter)
Sirbu, Carmen-Adella
Sormani, Maria Pia
Butzkueven, Helmut
Kalincik, Tomas
Internet cím:
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DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
11.
001-es BibID:
BIBFORM119143
035-os BibID:
(scopus)85181176590 (wos)001130397900001
Első szerző:
Spelman, Tim
Cím:
Comparative effectiveness and cost-effectiveness of natalizumab and fingolimod in rapidly evolving severe relapsing-remitting multiple sclerosis in the United Kingdom / Spelman T., Herring W. L., Acosta C., Hyde R., Jokubaitis V. G., Pucci E., Lugaresi A., Laureys G., Havrdova E. K., Horakova D., Izquierdo G., Eichau S., Ozakbas S., Alroughani R., Kalincik T., Duquette P., Girard M., Petersen T., Patti F., Csepany T., Granella F., Grand'Maison F., Ferraro D., Karabudak R., Jose Sa M., Trojano M., van Pesch V., Van Wijmeersch B., Cartechini E., McCombe P., Gerlach O., Spitaleri D., Rozsa C., Hodgkinson S., Bergamaschi R., Gouider R., Soysal A., Prevost J., Garber J., de Gans K., Ampapa R., Simo M., Sanchez-Menoyo J. L., Iuliano G., Sas A., van der Walt A., John N., Gray O., Hughes S., De Luca G., Onofrj M., Buzzard K., Skibina O., Terzi M., Slee M., Solaro C., Ramo-Tello C., Fragoso Y., Shaygannejad V., Moore F., Rajda C., Aguera-Morales E., Butzkueven H.
Dátum:
2024
ISSN:
1369-6998 1941-837X
Megjegyzések:
Aim To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS). Methods Real-world data from the MSBase Registry were obtained for patients with RES-RRMS who were previously either naive to disease-modifying therapies or had been treated with interferon-based therapies, glatiramer acetate, dimethyl fumarate, or teriflunomide (collectively known as BRACETD). Matched cohorts were selected by 3-way multinomial propensity score matching, and the annualized relapse rate (ARR) and 6-month?confirmed disability worsening (CDW6M) and improvement (CDI6M) were compared between treatment groups. Comparative effectiveness results were used in a cost-effectiveness model comparing natalizumab and fingolimod, using an established Markov structure over a lifetime horizon with health states based on the Expanded Disability Status Scale. Additional model data sources included the UK MS Survey 2015, published literature, and publicly available sources. Results In the comparative effectiveness analysis, we found a significantly lower ARR for patients starting natalizumab compared with fingolimod (rate ratio [RR]?=?0.65; 95% confidence interval [CI], 0.57?0.73) or BRACETD (RR = 0.46; 95% CI, 0.42?0.53). Similarly, CDI6M was higher for patients starting natalizumab compared with fingolimod (hazard ratio [HR]?=?1.25; 95% CI, 1.01?1.55) and BRACETD (HR = 1.46; 95% CI, 1.16?1.85). In patients starting fingolimod, we found a lower ARR (RR = 0.72; 95% CI, 0.65?0.80) compared with starting BRACETD, but no difference in CDI6M (HR = 1.17; 95% CI, 0.91?1.50). Differences in CDW6M were not found between the treatment groups. In the base-case cost-effectiveness analysis, natalizumab dominated fingolimod (0.302 higher quality-adjusted life-years [QALYs] and ?17,141 lower predicted lifetime costs). Similar cost-effectiveness results were observed across sensitivity analyses. Conclusions This MSBase Registry analysis suggests that natalizumab improves clinical outcomes when compared with fingolimod, which translates to higher QALYs and lower costs in UK patients with RES-RRMS.
Tárgyszavak:
Orvostudományok
Elméleti orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Multiple sclerosis
natalizumab
fingolimod
real-world data
comparative effectiveness
cost-effectiveness
Megjelenés:
Journal of Medical Economics. - 27 : 1 (2024), p. 109-125. -
További szerzők:
Herring, W. L.
Acosta, C.
Hyde, R.
Jokubaitis, Vilija
Pucci, Eugenio
Lugaresi, Alessandra
Laureys, Guy
Havrdova, Eva
Horakova, Dana
Izquierdo, Guillermo
Eichau, Sara
Ozakbas, Serkan
Alroughani, Raed
Kalincik, Tomas
Duquette, Pierre
Girard, Marc
Petersen, Thor
Patti, Francesco
Csépány Tünde (1956-) (neurológus, pszichiáter)
Granella, Franco
Grand'Maison, Francois
Ferraro, D.
Karabudak, Rana
José Sá, Maria
Trojano, Maria
Pesch, Vincent van
Van Wijmeersch, Bart
Cartechini, Elisabetta
McCombe, Pamela
Gerlach, Oliver
Spitaleri, Daniele
Rózsa Csilla
Hodgkinson, Suzanne
Bergamaschi, Roberto
Gouider, Riadh
Soysal, Aysun
Prevost, Julie
Garber, Justin
de Gans, Koen
Ampapa, Radek
Simó Magdolna
Sanchez-Menoyo, Jose
Iuliano, Gerardo
Sas Attila
Walt, Anneke van der
John, N.
Gray, Orla
Hughes, Stella
De Luca, Giacomo
Onofrj, Marco
Buzzard, Katherine
Skibina, Olga
Terzi, Murat
Slee, Mark
Solaro, Claudio
Ramo-Tello, Cristina
Fragoso, Yara
Shaygannejad, Vahid
Moore, Fraser
Rajda Cecília
Aguera-Morales, Eduardo
Butzkueven, Helmut
Internet cím:
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Intézményi repozitóriumban (DEA) tárolt változat
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