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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:
BIBFORM103013
035-os BibID:
(Wos)000597939700001 (Scopus)85097297540
Első szerző:
Berger, Thomas
Cím:
Factors influencing daily treatment choices in multiple sclerosis : practice guidelines, biomarkers and burden of disease / Berger Thomas, Adamczyk-Sowa Monika, Csépány Tünde, Fazekas Franz, Fabjan Tanja Hojs, Horáková Dana, Ledinek Alenka Horvath, Illes Zsolt, Kobelt Gisela, Jazbec Sasa Sega, Klímová Eleonóra, Leutmezer Fritz, Rejdak Konrad, Rozsa Csilla, Sellner Johann, Selmaj Krzysztof, Stourac Pavel, Szilasiová Jarmila, Turcáni Peter, Vachová Marta, Vanecková Manuela, Vécsei László, Havrdová Eva Kubala
Dátum:
2020
ISSN:
1756-2856 1756-2864
Megjegyzések:
At two meetings of a Central European board of multiple sclerosis (MS) experts in 2018 and 2019 factors influencing daily treatment choices in MS, especially practice guidelines, biomarkers and burden of disease, were discussed. The heterogeneity of MS and the complexity of the available treatment options call for informed treatment choices. However, evidence from clinical trials is generally lacking, particularly regarding sequencing, switches and escalation of drugs. Also, there is a need to identify patients who require highly efficacious treatment from the onset of their disease to prevent deterioration. The recently published European Committee for the Treatment and Research in Multiple Sclerosis/European Academy of Neurology clinical practice guidelines on pharmacological management of MS cover aspects such as treatment efficacy, response criteria, strategies to address suboptimal response and safety concerns and are based on expert consensus statements. However, the recommendations constitute an excellent framework that should be adapted to local regulations, MS center capacities and infrastructure. Further, available and emerging biomarkers for treatment guidance were discussed. Magnetic resonance imaging parameters are deemed most reliable at present, even though complex assessment including clinical evaluation and laboratory parameters besides imaging is necessary in clinical routine. Neurofilament-light chain levels appear to represent the current most promising non-imaging biomarker. Other immunological data, including issues of immunosenescence, will play an increasingly important role for future treatment algorithms. Cognitive impairment has been recognized as a major contribution to MS disease burden. Regular evaluation of cognitive function is recommended in MS patients, although no specific disease-modifying treatment has been defined to date. Finally, systematic documentation of real-life data is recognized as a great opportunity to tackle unresolved daily routine challenges, such as use of sequential therapies, but requires joint efforts across clinics, governments and pharmaceutical companies.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:
Therapeutic Advances in Neurological Disorders. - 13 (2020), p. 1-13. -
További szerzők:
Adamczyk-Sowa, Monika
Csépány Tünde (1956-) (neurológus, pszichiáter)
Fazekas, Franz
Fabjan, Tanja Hojs
Horakova, Dana
Horváth Ledinek, Alenka
Illés Zsolt (neurológus, Pécs)
Kobelt, Gisela
Jazbec, Sasa Sega
Klimova, Eleonora
Leutmezer, Fritz
Rejdak, Konrad
Rózsa Csilla
Sellner, Johann
Selmaj, Krzysztof
Stourac, Pavel
Szilasiova, Jarmila
Turčáni, Peter
Vachova Márta
Vanecková, Manuela
Vécsei László (1954-) (neurológus)
Havrdova, Eva
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
3.
001-es BibID:
BIBFORM076645
Első szerző:
Berger, Thomas
Cím:
Management of multiple sclerosis patients in central European countries : current needs and potential solutions / Thomas Berger, Monika Adamczyk-Sowa, Tünde Csépány, Franz Fazekas, Tanja Hojs Fabjan, Dana Horáková, Zsolt Illes, Eleonóra Klimová, Fritz Leutmezer, Konrad Rejdak, Csilla Rozsa, Saša Šega Jazbec, Jarmila Szilasiová, Peter Turčáni, Marta Vachová, László Vécsei, Eva Havrdová
Dátum:
2018
ISSN:
0019-1442
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
Megjelenés:
Therapeutic Advances in Neurological Disorders. - 11 (2018), p. 1-12. -
További szerzők:
Adamczyk-Sowa, Monika
Csépány Tünde (1956-) (neurológus, pszichiáter)
Fazekas, Franz
Fabjan, Tanja Hojs
Horakova, Dana
Illés Zsolt
Klimova, Eleonora
Leutmezer, Fritz
Rejdak, Konrad
Rózsa Csilla
Šega Jazbec, Saša
Szilasiova, Jarmila
Turčáni, Peter
Vachova Márta
Vécsei László (1954-) (neurológus)
Havrdova, Eva
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
4.
001-es BibID:
BIBFORM085749
Első szerző:
Brown, Jeremy William L.
Cím:
The risk of relapse following on-treatment clinically silent lesions in patients with relapsing-remitting multiple sclerosis / Brown, J. W. L., Lugaresi A., Horakova D., Havrdova E., Jokubaitis V., Lechner-Scott J., Trojano M., Min M., Shaw C., Shuey N., Slee M., Mccombe P., Van Pesch V., Van Wijmeersch B., Prevost J., Moore F., Prat A., Girard M., Duquette P., Ayrignac X., Sempere A. Perez, Sanchez-Menoyo J. L., Ramo-Tello C., Csépány Tünde, Hutchinson M., De Luca G., Bergamaschi R., Granella F., Curti E., Tsantes E., Sola P., Ferraro D., Alroughani R., Hupperts R., Al-Harbi T., Sidhom Y., Boz C., Terzi M., Ozakbas S., Soysal A., Pucci E., Izquierdo G., Iuliano G., Rio M. Edite, Spitaleri D., Grammond P., Grand'Maison F., Butzkueven H., Kalincik T.
Dátum:
2017
ISSN:
1352-4585
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idézhető absztrakt
folyóiratcikk
Megjelenés:
Multiple Sclerosis. - 23 : Suppl. 3 (2017), p. 992-994. -
További szerzők:
Lugaresi, Alessandra
Horakova, Dana
Havrdova, Eva
Jokubaitis, Vilija
Lechner-Scott, Jeannette
Trojano, Maria
Min, M.
Shaw, C. A.
Shuey, Neil
Slee, Mark
McCombe, Pamela
Pesch, Vincent van
Van Wijmeersch, Bart
Prevost, Julie
Moore, Fraser
Prat, Alexandre
Girard, Marc
Duquette, Pierre
Ayrignac, X.
Sempere, Perez A.
Sanchez-Menoyo, Jose
Ramo-Tello, Cristina
Csépány Tünde (1956-) (neurológus, pszichiáter)
Hutchinson, Michael
De Luca, Giacomo
Bergamaschi, Roberto
Granella, Franco
Curti, E.
Tsantes, E.
Sola, Patrizia
Ferraro, D.
Alroughani, Raed
Hupperts, Raymond
Al-Harbi, Talal
Sidhom, Youssef
Boz, Cavit
Terzi, Murat
Ozakbas, Serkan
Soysal, Aysun
Pucci, Eugenio
Izquierdo, Guillermo
Iuliano, Gerardo
Rio, Edite M.
Spitaleri, Daniele
Grammond, Pierre
Grand'Maison, Francois
Butzkueven, Helmut
Kalincik, Tomas
Internet cím:
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
5.
001-es BibID:
BIBFORM119146
035-os BibID:
(scopus)85177103067 (wos)001030569800001
Első szerző:
Diouf, Ibrahima
Cím:
Effectiveness of multiple disease-modifying therapies in relapsing-remitting multiple sclerosis : causal inference to emulate a multiarm randomised trial / Diouf Ibrahima, Malpas Charles B., Sharmin Sifat, Roos Izanne, Horakova Dana, Kubala Havrdova Eva, Patti Francesco, Shaygannejad Vahid, Ozakbas Serkan, Eichau Sara, Onofrj Marco, Lugaresi Alessandra, Alroughani Raed, Prat Alexandre, Duquette Pierre, Terzi Murat, Boz Cavit, Grand'Maison Francois, Sola Patrizia, Ferraro Diana, Grammond Pierre, Yamout Bassem, Altintas Ayse, Gerlach Oliver, Lechner-Scott Jeannette, Bergamaschi Roberto, Karabudak Rana, Iuliano Gerardo, McGuigan Christopher, Cartechini Elisabetta, Hughes Stella, Sa Maria Jose, Solaro Claudio, Kappos Ludwig, Hodgkinson Suzanne, Slee Mark, Granella Franco, de Gans Koen, McCombe Pamela A., Ampapa Radek, van der Walt Anneke, Butzkueven Helmut, Sánchez-Menoyo José Luis, Vucic Steve, Laureys Guy, Sidhom Youssef, Gouider Riadh, Castillo-Trivino Tamara, Gray Orla, Aguera-Morales Eduardo, Al-Asmi Abdullah, Shaw Cameron, Al-Harbi Talal M., Csepany Tunde, Sempere Angel P., Trevino Frenk Irene, Stuart Elizabeth A., Kalincik Tomas
Dátum:
2023
ISSN:
0022-3050
Megjegyzések:
Background Simultaneous comparisons of multiple disease-modifying therapies for relapsing-remitting multiple sclerosis (RRMS) over an extended follow-up are lacking. Here we emulate a randomised trial simultaneously comparing the effectiveness of six commonly used therapies over 5 years. Methods Data from 74 centres in 35 countries were sourced from MSBase. For each patient, the first eligible intervention was analysed, censoring at change/discontinuation of treatment. The compared interventions included natalizumab, fingolimod, dimethyl fumarate, teriflunomide, interferon beta, glatiramer acetate and no treatment. Marginal structural Cox models (MSMs) were used to estimate the average treatment effects (ATEs) and the average treatment effects among the treated (ATT), rebalancing the compared groups at 6-monthly intervals on age, sex, birth-year, pregnancy status, treatment, relapses, disease duration, disability and disease course. The outcomes analysed were incidence of relapses, 12-month confirmed disability worsening and improvement. Results 23 236 eligible patients were diagnosed with RRMS or clinically isolated syndrome. Compared with glatiramer acetate (reference), several therapies showed a superior ATE in reducing relapses: natalizumab (HR=0.44, 95% CI=0.40 to 0.50), fingolimod (HR=0.60, 95% CI=0.54 to 0.66) and dimethyl fumarate (HR=0.78, 95% CI=0.66 to 0.92). Further, natalizumab (HR=0.43, 95% CI=0.32 to 0.56) showed a superior ATE in reducing disability worsening and in disability improvement (HR=1.32, 95% CI=1.08 to 1.60). The pairwise ATT comparisons also showed superior effects of natalizumab followed by fingolimod on relapses and disability. Conclusions The effectiveness of natalizumab and fingolimod in active RRMS is superior to dimethyl fumarate, teriflunomide, glatiramer acetate and interferon beta. This study demonstrates the utility of MSM in emulating trials to compare clinical effectiveness among multiple interventions simultaneously.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
MULTIPLE SCLEROSIS
STATISTICS
Megjelenés:
Journal Of Neurology Neurosurgery And Psychiatry. - 94 : 12 (2023), p. 1004-1011. -
További szerzők:
Malpas, Charles B.
Sharmin, Sifat
Roos, Izanne
Horakova, Dana
Kubala Havrdova, Eva
Patti, Francesco
Shaygannejad, Vahid
Ozakbas, Serkan
Eichau, Sara
Onofrj, Marco
Lugaresi, Alessandra
Alroughani, Raed
Prat, Alexandre
Duquette, Pierre
Terzi, Murat
Boz, Cavit
Grand'Maison, Francois
Sola, Patrizia
Ferraro, Diana
Grammond, Pierre
Yamout, Bassem
Altintas, Ayse
Gerlach, Oliver
Lechner-Scott, Jeannette
Bergamaschi, Roberto
Karabudak, Rana
Iuliano, Gerardo
McGuigan, Christopher
Cartechini, Elisabetta
Hughes, Stella
Sá, Maria José
Solaro, Claudio
Kappos, Ludwig
Hodgkinson, Suzanne
Slee, Mark
Granella, Franco
de Gans, Koen
McCombe, Pamela
Ampapa, Radek
Walt, Anneke van der
Butzkueven, Helmut
Sanchez-Menoyo, Jose
Vucic, Steve
Laureys, Guy
Sidhom, Youssef
Gouider, Riadh
Castillo Triviño, Tamara
Gray, Orla
Aguera-Morales, Eduardo
Al-Asmi, Abdullah
Shaw, Cameron
Al-Harbi, Talal
Csépány Tünde (1956-) (neurológus, pszichiáter)
Sempere, Perez A.
Trevino-Frenk, Irene
Stuart, Elizabeth A.
Kalincik, Tomas
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
6.
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:
7.
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:
8.
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:
9.
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:
10.
001-es BibID:
BIBFORM067755
Első szerző:
Kalincik, Tomas
Cím:
Treatment effectiveness of alemtuzumab compared with natalizumab, fingolimod, and interferon beta in relapsing-remitting multiple sclerosis : a cohort study / Tomas Kalincik, J. William L. Brown, Neil Robertson, Mark Willis, Neil Scolding, Claire M. Rice, Alastair Wilkins, Owen Pearson, Tjalf Ziemssen, Michael Hutchinson, Christopher McGuigan, Vilija Jokubaitis, Tim Spelman, Dana Horakova, Eva Havrdova, Maria Trojano, Guillermo Izquierdo, Alessandra Lugaresi, Alexandre Prat, Marc Girard, Pierre Duquette, Pierre Grammond, Raed Alroughani, Eugenio Pucci, Patrizia Sola, Raymond Hupperts, Jeannette Lechner-Scott, Murat Terzi, Vincent Van Pesch, Csilla Rozsa, François Grand'Maison, Cavit Boz, Franco Granella, Mark Slee, Daniele Spitaleri, Javier Olascoaga, Roberto Bergamaschi, Freek Verheul, Steve Vucic, Pamela McCombe, Suzanne Hodgkinson, Jose Luis Sanchez-Menoyo, Radek Ampapa, Magdolna Simo, Tunde Csepany, Cristina Ramo, Edgardo Cristiano, Michael Barnett, Helmut Butzkueven, Alasdair Coles, MSBase Study Group
Dátum:
2017
ISSN:
1474-4422
Megjegyzések:
BackgroundAlemtuzumab, an anti-CD52 antibody, is proven to be more efficacious than interferon beta-1a in the treatment of relapsing-remitting multiple sclerosis, but its efficacy relative to more potent immunotherapies is unknown. We compared the effectiveness of alemtuzumab with natalizumab, fingolimod, and interferon beta in patients with relapsing-remitting multiple sclerosis treated for up to 5 years.MethodsIn this international cohort study, we used data from propensity-matched patients with relapsing-remitting multiple sclerosis from the MSBase and six other cohorts. Longitudinal clinical data were obtained from 71 MSBase centres in 21 countries and from six non-MSBase centres in the UK and Germany between Nov 1, 2015, and June 30, 2016. Key inclusion criteria were a diagnosis of definite relapsing-remitting multiple sclerosis, exposure to one of the study therapies (alemtuzumab, interferon beta, fingolimod, or natalizumab), age 65 years or younger, Expanded Disability Status Scale (EDSS) score 6?5 or lower, and no more than 10 years since the first multiple sclerosis symptom. The primary endpoint was annualised relapse rate. The secondary endpoints were cumulative hazards of relapses, disability accumulation, and disability improvement events. We compared relapse rates with negative binomial models, and estimated cumulative hazards with conditional proportional hazards models.FindingsPatients were treated between Aug 1, 1994, and June 30, 2016. The cohorts consisted of 189 patients given alemtuzumab, 2155 patients given interferon beta, 828 patients given fingolimod, and 1160 patients given natalizumab. Alemtuzumab was associated with a lower annualised relapse rate than interferon beta (0?19 [95% CI 0?14?0?23] vs 0?53 [0?46?0?61], p<0?0001) and fingolimod (0?15 [0?10?0?20] vs 0?34 [0?26?0?41], p<0?0001), and was associated with a similar annualised relapse rate as natalizumab (0?20 [0?14?0?26] vs 0?19 [0?15?0?23], p=0?78). For the disability outcomes, alemtuzumab was associated with similar probabilities of disability accumulation as interferon beta (hazard ratio [HR] 0?66 [95% CI 0?36?1?22], p=0?37), fingolimod (1?27 [0?60?2?70], p=0?67), and natalizumab (0?81 [0?47?1?39], p=0?60). Alemtuzumab was associated with similar probabilities of disability improvement as interferon beta (0?98 [0?65?1?49], p=0?93) and fingolimod (0?50 [0?25?1?01], p=0?18), and a lower probability of disability improvement than natalizumab (0?35 [0?20?0?59], p=0?0006).InterpretationAlemtuzumab and natalizumab seem to have similar effects on annualised relapse rates in relapsing-remitting multiple sclerosis. Alemtuzumab seems superior to fingolimod and interferon beta in mitigating relapse activity. Natalizumab seems superior to alemtuzumab in enabling recovery from disability. Both natalizumab and alemtuzumab seem highly effective and viable immunotherapies for multiple sclerosis. Treatment decisions between alemtuzumab and natalizumab should be primarily governed by their safety profiles.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
Megjelenés:
Lancet Neurology 16 : 4 (2017), p. 271-281. -
További szerzők:
Brown, Jeremy William L.
Robertson, Neil
Willis, Mark
Scolding, Neil
Rice, Claire M.
Wilkins, Alastair
Pearson, Owen
Ziemssen, Tjalf
Hutchinson, Michael
McGuigan, Christopher
Jokubaitis, Vilija
Spelman, Tim
Horakova, Dana
Havrdova, Eva
Trojano, Maria
Izquierdo, Guillermo
Lugaresi, Alessandra
Prat, Alexandre
Girard, Marc
Duquette, Pierre
Grammond, Pierre
Alroughani, Raed
Pucci, Eugenio
Sola, Patrizia
Hupperts, Raymond
Lechner-Scott, Jeannette
Terzi, Murat
Pesch, Vincent van
Rózsa Csilla
Grand'Maison, Francois
Boz, Cavit
Granella, Franco
Slee, Mark
Spitaleri, Daniele
Olascoaga, Javier
Bergamaschi, Roberto
Verheul, Freek
Vucic, Steve
McCombe, Pamela
Hodgkinson, Suzanne
Sanchez-Menoyo, Jose
Ampapa, Radek
Simó Magdolna
Csépány Tünde (1956-) (neurológus, pszichiáter)
Ramo, Cristina
Cristiano, Edgardo
Barnett, Michael
Butzkueven, Helmut
Coles, Alasdair
MSBase Study Group
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
11.
001-es BibID:
BIBFORM058152
Első szerző:
Kalincik, Tomas
Cím:
Switch to natalizumab versus fingolimod in active relapsing-remitting multiple sclerosis / Tomas Kalincik, Dana Horakova, Tim Spelman, Vilija Jokubaitis, Maria Trojano, Alessandra Lugaresi, Guillermo Izquierdo, Csilla Rozsa, Pierre Grammond, Raed Alroughani, Pierre Duquette, Marc Girard, Eugenio Pucci, Jeannette Lechner-Scott, Mark Slee, Ricardo Fernandez-Bolanos, Francois Grand'Maison, Raymond Hupperts, Freek Verheul, Suzanne Hodgkinson, Celia Oreja-Guevara, Daniele Spitaleri, Michael Barnett, Murat Terzi, Roberto Bergamaschi, Pamela McCombe, Jose Sanchez-Menoyo, Magdolna Simo, Tunde Csepany, Gabor Rum, Cavit Boz, Eva Havrdova, Helmut Butzkueven
Dátum:
2015
ISSN:
0364-5134
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
Megjelenés:
Annals Of Neurology. - 77 : 3 (2015), p. 425-435. -
További szerzők:
Horakova, Dana
Spelman, Tim
Jokubaitis, Vilija
Trojano, Maria
Lugaresi, Alessandra
Izquierdo, Guillermo
Rózsa Csilla
Grammond, Pierre
Alroughani, Raed
Duquette, Pierre
Girard, Marc
Pucci, Eugenio
Lechner-Scott, Jeannette
Slee, Mark
Fernandez-Bolanos, Ricardo
Grand'Maison, Francois
Hupperts, Raymond
Verheul, Freek
Hodgkinson, Suzanne
Oreja-Guevara, Celia
Spitaleri, Daniele
Barnett, Michael
Terzi, Murat
Bergamaschi, Roberto
McCombe, Pamela
Sanchez-Menoyo, Jose
Simó Magdolna
Csépány Tünde (1956-) (neurológus, pszichiáter)
Rum Gábor
Boz, Cavit
Havrdova, Eva
Butzkueven, Helmut
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
12.
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
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