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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-Harthi, Helal F. Csépány Tünde (1956-) (neurológus, pszichiáter) Sempere, Perez A. Trevino-Frenk, Irene Stuart, Elizabeth A. Kalincik, Tomas
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2.

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
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3.

001-es BibID:BIBFORM083269
Első szerző:Fambiatos, Adam
Cím:Risk of secondary progressive multiple sclerosis : a longitudinal study / Adam Fambiatos, Vilija Jokubaitis, Dana Horakova, Eva Kubala Havrdova, Maria Trojano, Alexandre Prat, Marc Girard, Pierre Duquette, Alessandra Lugaresi, Guillermo Izquierdo, Francois Grand'Maison, Pierre Grammond, Patrizia Sola, Diana Ferraro, Raed Alroughani, Murat Terzi, Raymond Hupperts, Cavit Boz, Jeannette Lechner-Scott, Eugenio Pucci, Roberto Bergamaschi, Vincent Van Pesch, Serkan Ozakbas, Franco Granella, Recai Turkoglu, Gerardo Iuliano, Daniele Spitaleri, Pamela McCombe, Claudio Solaro, Mark Slee, Radek Ampapa, Aysun Soysal, Thor Petersen, Jose Luis Sanchez-Menoyo, Freek Verheul, Julie Prevost, Youssef Sidhom, Bart Van Wijmeersch, Steve Vucic, Edgardo Cristiano, Maria Laura Saladino, Norma Deri, Michael Barnett, Javier Olascoaga, Fraser Moore, Olga Skibina, Orla Gray, Yara Fragoso, Bassem Yamout, Cameron Shaw, Bhim Singhal, Neil Shuey, Suzanne Hodgkinson, Ayse Altintas, Talal Al-Harbi, Tunde Csepany, Bruce Taylor, Jordana Hughes, Jae-Kwan Jun, Anneke van der Walt, Tim Spelman, Helmut Butzkueven, Tomas Kalincik, MSBase Study Group
Dátum:2020
ISSN:1352-4585
Megjegyzések:Background: The risk factors for conversion from relapsing-remitting to secondary progressive multiple sclerosis remain highly contested. Objective: The aim of this study was to determine the demographic, clinical and paraclinical features that influence the risk of conversion to secondary progressive multiple sclerosis. Methods: Patients with adult-onset relapsing?remitting multiple sclerosis and at least four recorded disability scores were selected from MSBase, a global observational cohort. The risk of conversion to objectively defined secondary progressive multiple sclerosis was evaluated at multiple time points per patient using multivariable marginal Cox regression models. Sensitivity analyses were performed. Results: A total of 15,717 patients were included in the primary analysis. Older age (hazard ratio (HR) = 1.02, p < 0.001), longer disease duration (HR = 1.01, p = 0.038), a higher Expanded Disability Status Scale score (HR = 1.30, p < 0.001), more rapid disability trajectory (HR = 2.82, p < 0.001) and greater number of relapses in the previous year (HR = 1.07, p = 0.010) were independently associated with an increased risk of secondary progressive multiple sclerosis. Improving disability (HR = 0.62, p = 0.039) and disease-modifying therapy exposure (HR = 0.71, p = 0.007) were associated with a lower risk. Recent cerebral magnetic resonance imaging activity, evidence of spinal cord lesions and oligoclonal bands in the cerebrospinal fluid were not associated with the risk of conversion. Conclusion: Risk of secondary progressive multiple sclerosis increases with age, duration of illness and worsening disability and decreases with improving disability. Therapy may delay the onset of secondary progression.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Multiple Sclerosis. - 26 : 1 (2020), p. 79-90. -
További szerzők:Jokubaitis, Vilija Horakova, Dana Kubala Havrdova, Eva Trojano, Maria Prat, Alexandre Girard, Marc Duquette, Pierre Lugaresi, Alessandra Izquierdo, Guillermo Grand'Maison, Francois Grammond, Pierre Sola, Patrizia Ferraro, Diana Alroughani, Raed Terzi, Murat Hupperts, Raymond Boz, Cavit Lechner-Scott, Jeannette Pucci, Eugenio Bergamaschi, Roberto Pesch, Vincent van Ozakbas, Serkan Granella, Franco Turkoglu, Recai Iuliano, Gerardo Spitaleri, Daniele McCombe, Pamela Solaro, Claudio Slee, Mark Ampapa, Radek Soysal, Aysun Petersen, Thor Sanchez-Menoyo, Jose Verheul, Freek Prevost, Julie Sidhom, Youssef Van Wijmeersch, Bart Vucic, Steve Cristiano, Edgardo Saladino, Maria Laura Deri, Norma Barnett, Michael Olascoaga, Javier Moore, Fraser Skibina, Olga Gray, Orla Fragoso, Yara Yamout, Bassem Shaw, Cameron Singhal, Bhim Shuey, Neil Hodgkinson, Suzanne Altintas, Ayse Al-Harbi, Talal Csépány Tünde (1956-) (neurológus, pszichiáter) Taylor, Bruce V. Hughes, Jordana Jun, Jae-Kwan Walt, Anneke van der Spelman, Tim Butzkueven, Helmut Kalincik, Tomas MSBase Study Group
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4.

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

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
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6.

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, Yamou 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
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7.

001-es BibID:BIBFORM116385
035-os BibID:(Scopus)85176495277 (WOS)001063488100001
Első szerző:Sharmin, Sifat
Cím:The risk of secondary progressive multiple sclerosis is geographically determined but modifiable / Sharmin Sifat, Roos Izanne, Simpson-Yap Steve, Charles Malpas, Marina M. Sánchez, Serkan Ozakbas, Dana Horakova, Eva K. Havrdova, Francesco Patti, Raed Alroughani, Guillermo Izquierdo, Sara Eichau, Cavit Boz, Magd Zakaria, Marco Onofrj, Alessandra Lugaresi, Bianca Weinstock-Guttman, Alexandre Prat, Marc Girard, Pierre Duquette, Murat Terzi, Maria Pia Amato, Rana Karabudak, Francois Grand`Maison, Samia J. Khoury, Pierre Grammond, Jeannette Lechner-Scott, Katherine Buzzard, Olga Skibina, Anneke van der Walt, Helmut Butzkueven, Recai Turkoglu, Ayse Altintas, Davide Maimone, Allan Kermode, Nevin Shalaby, Vincent V. Pesch, Ernest Butler, Youssef Sidhom, Riadh Gouider, Saloua Mrabet, Oliver Gerlach, Aysun Soysal, Michael Barnett, Jens Kuhle, Stella Hughes, Maria J. Sa, Suzanne Hodgkinson, Celia Oreja-Guevara, Radek Ampapa, Thor Petersen, Cristina Ramo-Tello, Daniele Spitaleri, Pamela McCombe, Bruce Taylor, Julie Prevost, Matteo Foschi, Mark Slee, Chris McGuigan, Guy Laureys, Liesbeth V. Hijfte, Koen de Gans, Claudio Solaro, Jiwon Oh, Richard Macdonell, Eduardo Aguera-Morales, Bhim Singhal, Orla Gray, Justin Garber, Bart V. Wijmeersch, Mihaela Simu, Tamara Castillo-Triviño, Jose L. Sanchez-Menoyo, Dheeraj Khurana, Abdullah Al-Asmi, Talal Al-Harbi, Norma Deri, Yara Fragoso, Patrice H. Lalive, L. G. F. Sinnige, Cameron Shaw, Neil Shuey, Tunde Csepany, Angel P. Sempere, Fraser Moore, Danny Decoo, Barbara Willekens, Claudio Gobbi, Jennifer Massey, Todd Hardy, John Parratt, Tomas Kalincik, the MSBase investigators
Dátum:2023
ISSN:0006-8950
Megjegyzések:Geographical variations in the incidence and prevalence of multiple sclerosis have been reported globally. Latitude as a surrogate for exposure to ultraviolet radiation but also other lifestyle and environmental factors are regarded as drivers of this variation. No previous studies evaluated geographical variation in the risk of secondary progressive multiple sclerosis, an advanced form of multiple sclerosis that is characterized by steady accrual of irreversible disability.We evaluated differences in the risk of secondary progressive multiple sclerosis in relation to latitude and country of residence, modified by high-to-moderate efficacy immunotherapy in a geographically diverse cohort of patients with relapsing-remitting multiple sclerosis. The study included relapsing-remitting multiple sclerosis patients from the global MSBase registry with at least one recorded assessment of disability. Secondary progressive multiple sclerosis was identified as per clinician diagnosis. Sensitivity analyses used the operationalized definition of secondary progressive multiple sclerosis and the Swedish decision tree algorithm. A proportional hazards model was used to estimate the cumulative risk of secondary progressive multiple sclerosis by country of residence (latitude), adjusted for sex, age at disease onset, time from onset to relapsing-remitting phase, disability (Multiple Sclerosis Severity Score) and relapse activity at study inclusion, national multiple sclerosis prevalence, government health expenditure, and proportion of time treated with high-to-moderate efficacy disease-modifying therapy. Geographical variation in time from relapsing-remitting phase to secondary progressive phase of multiple sclerosis was modelled through a proportional hazards model with spatially correlated frailties.We included 51 126 patients (72% female) from 27 countries. The median survival time from relapsing-remitting phase to secondary progressive multiple sclerosis among all patients was 39 (95% confidence interval: 37 to 43) years. Higher latitude [median hazard ratio = 1.21, 95% credible interval (1.16, 1.26)], higher national multiple sclerosis prevalence [1.07 (1.03, 1.11)], male sex [1.30 (1.22, 1.39)], older age at onset [1.35 (1.30, 1.39)], higher disability [2.40 (2.34, 2.47)] and frequent relapses [1.18 (1.15, 1.21)] at inclusion were associated with increased hazard of secondary progressive multiple sclerosis. Higher proportion of time on high-to-moderate efficacy therapy substantially reduced the hazard of secondary progressive multiple sclerosis [0.76 (0.73, 0.79)] and reduced the effect of latitude [interaction: 0.95 (0.92, 0.99)]. At the country-level, patients in Oman, Tunisia, Iran and Canada had higher risks of secondary progressive multiple sclerosis relative to the other studied regions.Higher latitude of residence is associated with a higher probability of developing secondary progressive multiple sclerosis. High-to-moderate efficacy immunotherapy can mitigate some of this geographically co-determined risk. By analysing longitudinal data from 27 countries, Sharmin et al. reveal a geographically varying risk of conversion to secondary progressive disease in patients with multiple sclerosis. Higher latitude of residence increases the risk while high-to-moderate efficacy immunotherapies reduce the risk substantially.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
disease-modifying therapy
geography
health expenditure
latitude
secondary progressive multiple sclerosis
Megjelenés:Brain. - 146 : 11 (2023), p. 4633-4644. -
További szerzők:Roos, Izanne Simpson-Yap, Steve Malpas, Charles Sánchez, Marina M. Ozakbas, Serkan Horakova, Dana Havrdova, Eva Patti, Francesco Alroughani, Raed Izquierdo, Guillermo Eichau, Sara Boz, Cavit Zakaria, Magd Onofrj, Marco Lugaresi, Alessandra Weinstock-Guttman, Bianca Prat, Alexandre Girard, Marc Duquette, Pierre Terzi, Murat Amato, Maria Pia Karabudak, Rana Grand'Maison, Francois Khoury, Samia J. Grammond, Pierre Lechner-Scott, Jeannette Buzzard, Katherine Skibina, Olga Walt, Anneke van der Butzkueven, Helmut Turkoglu, Recai Altintas, Ayse Maimone, Davide Kermode, Allan G. Shalaby, Nevin Pesch, Vincent van Butler, Ernest Sidhom, Youssef Gouider, Riadh Mrabet, Saloua Gerlach, Oliver Soysal, Aysun Barnett, Michael Kuhle, Jens Hughes, Stella Sá, Maria José Hodgkinson, Suzanne Oreja-Guevara, Celia Ampapa, Radek Petersen, Thor Ramo-Tello, Cristina Spitaleri, Daniele McCombe, Pamela Taylor, Bruce V. Prevost, Julie Foschi, Matteo Slee, Mark McGuigan, Christopher Laureys, Guy Hijfte, Liesbeth V. de Gans, Koen Solaro, Claudio Oh, Jiwon Macdonell, Richard Aguera-Morales, Eduardo Singhal, Bhim Gray, Orla Garber, Justin Van Wijmeersch, Bart Simu, Mihaela Castillo Triviño, Tamara Sanchez-Menoyo, Jose Khurana, Dheeraj Al-Asmi, Abdullah Al-Harbi, Talal Deri, Norma Fragoso, Yara Lalive, Patrice H. Sinnige, L. G. F. Shaw, Cameron Shuey, Neil Csépány Tünde (1956-) (neurológus, pszichiáter) Sempere, Perez A. Moore, Fraser Decoo, Danny Willekens, Barbara Gobbi, Claudio Massey, Jennifer Hardy, Todd A. Parratt, John Kalincik, Tomas the MSBase investigators
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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
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001-es BibID:BIBFORM083268
Első szerző:Zhou, Yuan
Cím:Redefining the Multiple Sclerosis Severity Score (MSSS) : the effect of sex and onset phenotype / Yuan Zhou, Suzi B. Claflin, Jim Stankovich, Ingrid van der Mei, Steve Simpson, Richard H. Roxburgh, Tomas Kalincik, Leigh Blizzard, Alessandra Lugaresi, Raed Alroughani, Seyed Aidin Sajedi, Helmut Butzkueven, Eugenio Pucci, Daniele L. A. Spitaleri, Franco Granella, Edgardo Cristiano, Bassem Yamout, Stella Hughes, Riadh Gouider, José Luis Sánchez Menoyo, Javier Olascoaga, Chris McGuigan, Cameron Shaw, Allan G. Kermode, Krisztian Kasa, Talal Al-Harbi, Ayse Altintas, Guy Laureys, Yara Fragoso, Todd A. Hardy, Tunde Csepany, Carmen-Adella Sirbu, Danny Decoo, Attila Sas, Jose C. Alvarez-Cermeño, Karim Kotkata, Jorge Millán-Pascual, Bruce V. Taylor
Dátum:2020
ISSN:1352-4585
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:Multiple Sclerosis. - 26 : 13 (2020), p. 1765-1774. -
További szerzők:Claflin, Suzi B. Stankovich, Jim Mei, Ingrid van der Simpson, Steve Roxburgh, Richard H. Kalincik, Tomas Blizzard, Leigh Lugaresi, Alessandra Alroughani, Raed Sajedi, Seyed Aidin Butzkueven, Helmut Pucci, Eugenio Spitaleri, Daniele L. A. Granella, Franco Cristiano, Edgardo Yamout, Bassem Hughes, Stella Gouider, Riadh Sánchez Menoyo, José Luis Olascoaga, Javier McGuigan, Christopher Shaw, Cameron Kermode, Allan G. Kása Krisztián Al-Harbi, Talal Altintas, Ayse Laureys, Guy Fragoso, Yara Hardy, Todd A. Csépány Tünde (1956-) (neurológus, pszichiáter) Sirbu, Carmen-Adella Decoo, Danny Sas Attila Alvarez-Cermeño, Jose C. Kotkata, Karim Millán-Pascual, Jorge Taylor, Bruce V.
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