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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:
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:
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
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
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
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
Internet cím:
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
6.
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:
7.
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:
8.
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:
9.
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
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:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
11.
001-es BibID:
BIBFORM103010
035-os BibID:
(Wos)000697081200001 (Scopus)85115657202
Első szerző:
Sharmin, Sifat
Cím:
Natalizumab Versus Fingolimod in Patients with Relapsing-Remitting Multiple Sclerosis : a Subgroup Analysis From Three International Cohorts / Sharmin Sifat, Lefort Mathilde, Andersen Johanna Balslev, Leray Emmanuelle, Horakova Dana, Havrdova Eva Kubala, Alroughani Raed, Izquierdo Guillermo, 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, Prevost Julie, Maimone Davide, Skibina Olga, Buzzard Katherine, Van der Walt Anneke, Van Wijmeersch Bart, Csepany Tunde, Spitaleri Daniele, Vucic Steve, Casey Romain, Debouverie Marc, Edan Gilles, Ciron Jonathan, Ruet Aurélie, De Seze Jérome, Maillart Elisabeth, Zephir Hélene, Labauge Pierre, Defer Gilles, Lebrun-Frénay Christine, Moreau Thibault, Berger Eric, Clavelou Pierre, Pelletier Jean, Stankoff Bruno, Gout Olivier, Thouvenot Eric, Heinzlef Olivier, Al-Khedr Abullatif, Bourre Bertrand, Casez Olivier, Cabre Philippe, Montcuquet Alexis, Wahab Abir, Camdessanché Jean-Philippe, Maurousset Aude, Patry Ivania, Hankiewicz Karolina, Pottier Corinne, Maubeuge Nicolas, Labeyrie Céline, Nifle Chantal, Laplaud David, Koch-Henriksen Niels, Sellebjerg Finn Thorup, Soerensen Per Soelberg, Pfleger Claudia Christina, Rasmussen Peter Vestergaard, Jensen Michael Broksgaard, Frederiksen Jette Lautrup, Bramow Stephan, Mathiesen Henrik Kahr, Schreiber Karen Ingrid, Magyari Melinda, Vukusic Sandra, Butzkueven Helmut, Kalincik Tomas, Danish Multiple Sclerosis Registry, OFSEP and the MSBase investigators
Dátum:
2021
ISSN:
1172-7047
Megjegyzések:
Introduction Natalizumab has proved to be more efective than fngolimod in reducing disease activity in relapsing-remitting multiple sclerosis (RRMS). Whether this association is universal for all patient groups remains to be determined. Objective The aim of this study was to compare the relative efectiveness of natalizumab and fngolimod in RRMS subgroups defned by the baseline demographic and clinical characteristics of interest. Methods Patients with RRMS who were given natalizumab or fngolimod were identifed in a merged cohort from three international registries. Efcacy outcomes were compared across subgroups based on patients' sex, age, disease duration, Expanded Disability Status Scale (EDSS) score, and disease and magnetic resonance imaging (MRI) activity 12 months prior to treatment initiation. Study endpoints were number of relapses (analyzed with weighted negative binomial generalized linear model) and 6-month confrmed disability worsening and improvement events (weighted Cox proportional hazards model), recorded during study therapy. Each patient was weighted using inverse probability of treatment weighting based on propensity score. Results A total of 5148 patients (natalizumab 1989; fngolimod 3159) were included, with a mean ? standard deviation age at baseline of 38 ? 10 years, and the majority (72%) were women. The median on-treatment follow-up was 25 (quartiles 15?41) months. Natalizumab was associated with fewer relapses than fngolimod (incidence rate ratio [IRR]; 95% confdence interval [CI]) in women (0.76; 0.65?0.88); in those aged ? 38 years (0.64; 0.54?0.76); in those with disease duration ? 7 years (0.63; 0.53?0.76); in those with EDSS score < 4 (0.75; 0.64?0.88), < 6 (0.80; 0.70?0.91), and ? 6 (0.52; 0.31?0.86); and in patients with pre-baseline relapses (0.74; 0.64?0.86). A higher probability of confrmed disability improvement on natalizumab versus fngolimod (hazard ratio [HR]; 95% CI) was observed among women (1.36; 1.10?1.66); those aged > 38 years (1.34; 1.04?1.73); those with disease duration > 7 years (1.33; 1.01?1.74); those with EDSS score < 6 (1.21; 1.01?1.46) and ? 6 (1.93; 1.11?3.34); and patients with no new MRI lesion (1.73; 1.19?2.51). Conclusions Overall, in women, younger patients, those with shorter disease durations, and patients with pre-treatment relapses, natalizumab was associated with a lower frequency of multiple sclerosis relapses than fngolimod. It was also associated with an increased chance of recovery from disability among most patients, particularly women and those with no recent MRI activity.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
multiple sclerosis natalizumab fingolimod
Megjelenés:
Cns Drugs. - 35 (2021), p. 1217-1232. -
További szerzők:
Lefort, Mathilde
Andersen, Johanna Balslev
Leray, Emmanuelle
Horakova, Dana
Havrdova, Eva
Alroughani, Raed
Izquierdo, Guillermo
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
Prevost, Julie
Maimone, Davide
Skibina, Olga
Buzzard, Katherine
Walt, Anneke van der
Van Wijmeersch, Bart
Csépány Tünde (1956-) (neurológus, pszichiáter)
Spitaleri, Daniele
Vucic, Steve
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
Patry, Ivania
Hankiewicz, Karolina
Pottier, Corinne
Maubeuge, Nicolas
Labeyrie, Céline
Nifle, Chantal
Laplaud, David
Koch-Henriksen, Niels
Sellebjerg, Finn Thorup
Soerensen, Per Soelberg
Pfleger, Claudia Christina
Rasmussen, Peter Vestergaard
Jensen, Michael Broksgaard
Frederiksen, Jette Lautrup
Bramow, Stephan
Mathiesen, Henrik Kahr
Schreiber, Karen
Magyari Melinda
Vukusic, Sandra
Butzkueven, Helmut
Kalincik, Tomas
Danish Multiple Sclerosis Registry
OFSEP and the MSBase investigators
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
12.
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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