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1.
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:
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
3.
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:
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