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1.
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:
2.
001-es BibID:
BIBFORM103017
035-os BibID:
(Wos)000685503300008 (Scopus)85107912293 (cikkazonosító)106180
Első szerző:
De Brouwer, Edward
Cím:
Longitudinal machine learning modeling of MS patient trajectories improves predictions of disability progression / De Brouwer Edward, Becker Thijs, Moreau Yves, Havrdova Eva Kubala, Trojano Maria, Eichau Sara, Ozakbas Serkan, Onofrj Marco, Grammond Pierre, Kuhle Jens, Kappos Ludwig, Sola Patrizia, Cartechini Elisabetta, Lechner-Scott Jeannette, Alroughani Raed, Gerlach Oliver, Kalincik Tomas, Granella Franco, Grand'Maison Francois, Bergamaschi Roberto, José Sá Maria, Van Wijmeersch Bart, Soysal Aysun, Sanchez-Menoyo Jose Luis, Solaro Claudio, Boz Cavit, Iuliano Gerardo, Buzzard Katherine, Aguera-Morales Eduardo, Terzi Murat, Trivio Tamara Castillo, Spitaleri Daniele, Van Pesch Vincent, Shaygannejad Vahid, Moore Fraser, Oreja-Guevara Celia, Maimone Davide, Gouider Riadh, Csepany Tunde, Ramo-Tello Cristina, Peeters Liesbet
Dátum:
2021
ISSN:
0169-2607
Megjegyzések:
Background and Objectives: Research in Multiple Sclerosis (MS) has recently focused on extracting knowledge from real-world clinical data sources. This type of data is more abundant than data produced during clinical trials and potentially more informative about real-world clinical practice. However, this comes at the cost of less curated and controlled data sets. In this work we aim to predict disability progression by optimally extracting information from longitudinal patient data in the real-world setting, with a special focus on the sporadic sampling problem. Methods: We use machine learning methods suited for patient trajectories modeling, such as recurrent neural networks and tensor factorization. A subset of 6682 patients from the MSBase registry is used. Results: We can predict disability progression of patients in a two-year horizon with an ROC-AUC of 0.85, which represents a 32% decrease in the ranking pair error (1-AUC) compared to reference methods using static clinical features. Conclusions: Compared to the models available in the literature, this work uses the most complete patient history for MS disease progression prediction and represents a step forward towards AI-assisted precision medicine in MS.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:
Computer Methods And Programs In Biomedicine. - 208 (2021), p. 1-14. -
További szerzők:
Becker, Thijs
Moreau, Yves
Havrdova, Eva
Trojano, Maria
Eichau, Sara
Ozakbas, Serkan
Onofrj, Marco
Grammond, Pierre
Kuhle, Jens
Kappos, Ludwig
Sola, Patrizia
Cartechini, Elisabetta
Lechner-Scott, Jeannette
Alroughani, Raed
Gerlach, Oliver
Kalincik, Tomas
Granella, Franco
Grand'Maison, Francois
Bergamaschi, Roberto
José Sá, Maria
Van Wijmeersch, Bart
Soysal, Aysun
Sanchez-Menoyo, Jose
Solaro, Claudio
Boz, Cavit
Iuliano, Gerardo
Buzzard, Katherine
Aguera-Morales, Eduardo
Terzi, Murat
Trivio, Tamara Castillo
Spitaleri, Daniele
Pesch, Vincent van
Shaygannejad, Vahid
Moore, Fraser
Oreja-Guevara, Celia
Maimone, Davide
Gouider, Riadh
Csépány Tünde (1956-) (neurológus, pszichiáter)
Ramo-Tello, Cristina
Peeters, Liesbet
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
3.
001-es BibID:
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:
4.
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:
5.
001-es BibID:
BIBFORM083270
035-os BibID:
(cikkazonosító)101868 (PMID)31877445
Első szerző:
Kunchok, Amy
Cím:
Clinical and therapeutic predictors of disease outcomes in AQP4-IgG+ neuromyelitis optica spectrum disorder / Amy Kunchok, Charles Malpas, Petra Nytrova, Eva Kubala Havrdova, Raed Alroughani, Murat Terzi, Bassem Yamout, Jyh Yung Hor, Rana Karabudak, Cavit Boz, Serkan Ozakbas, Javier Olascoaga, Magdolna Simo, Franco Granella, Francesco Patti, Pamela McCombe, Tunde Csepany, Bhim Singhal, Roberto Bergamaschi, Yara Fragoso, Talal Al-Harbi, Recai Turkoglu, Jeannette Lechner-Scott, Guy Laureys, Celia Oreja-Guevara, Eugenio Pucci, Patrizia Sola, Diana Ferraro, Ayse Altintas, Aysun Soysal, Steve Vucic, Francois Grand'Maison, Guillermo Izquierdo, Sara Eichau, Alessandra Lugaresi, Marco Onofrj, Maria Trojano, Mark Marriott, Helmut Butzkueven, Ilya Kister, Tomas Kalincik
Dátum:
2020
ISSN:
2211-0348
Megjegyzések:
Aquaporin-4-IgG positive (AQP4-IgG+) Neuromyelitis Optica Spectrum Disorder (NMOSD) is an uncommon central nervous system autoimmune disorder. Disease outcomes in AQP4-IgG+NMOSD are typically measured by relapse rate and disability. Using the MSBase, a multi-centre international registry, we aimed to examine the impact immunosuppressive therapies and patient characteristics as predictors of disease outcome measures in AQP4-IgG+NMOSD. METHOD: This MSBase cohort study of AQP4-IgG+NMOSD patients examined modifiers of relapse in a multivariable proportional hazards model and expanded disability status score (EDSS) using a mixed effects model. RESULTS: 206 AQP4-IgG+ patients were included (median follow-up 3.7 years). Age (hazard ratio [HR] = 0.82 per decade, p = 0.001), brainstem onset (HR = 0.45, p = 0.009), azathioprine (HR = 0.46, p<0.001) and mycophenolate mofetil (HR = 0.09, p = 0.012) were associated with a reduced risk of relapse. A greater EDSS was associated with age (β = 0.45 (per decade), p<0.001) and disease duration (β = 0.07 per year, p<0.001). A slower increase in EDSS was associated with azathioprine (β = -0.48, p<0.001), mycophenolate mofetil (β = -0.69, p = 0.04) and rituximab (β = -0.35, p = 0.024). INTERPRETATION: This study has demonstrated that azathioprine and mycophenolate mofetil reduce the risk of relapses and disability progression is modified by azathioprine, mycophenolate mofetil and rituximab. Age and disease duration were the only patient characteristics that modified the risk of relapse and disability in our cohort.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Disability
Immunosuppression
Neuromyelitis optica spectrum disorder
Predictors
Relapses
Therapy
Megjelenés:
Multiple Sclerosis and Related Disorders. - 38 (2020), p. 1-8. -
További szerzők:
Malpas, Charles
Nytrova, Petra
Havrdova, Eva
Alroughani, Raed
Terzi, Murat
Yamout, Bassem
Hor, Jyh Yung
Karabudak, Rana
Boz, Cavit
Ozakbas, Serkan
Olascoaga, Javier
Simó Magdolna
Granella, Franco
Patti, Francesco
McCombe, Pamela
Csépány Tünde (1956-) (neurológus, pszichiáter)
Singhal, Bhim
Bergamaschi, Roberto
Fragoso, Yara
Al-Harbi, Talal
Turkoglu, Recai
Lechner-Scott, Jeannette
Laureys, Guy
Oreja-Guevara, Celia
Pucci, Eugenio
Sola, Patrizia
Ferraro, Diana
Altintas, Ayse
Soysal, Aysun
Vucic, Steve
Grand'Maison, Francois
Izquierdo, Guillermo
Eichau, Sara
Lugaresi, Alessandra
Onofrj, Marco
Trojano, Maria
Marriott, Mark
Butzkueven, Helmut
Kister, Ilya
Kalincik, Tomas
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
6.
001-es BibID:
BIBFORM114473
035-os BibID:
(Scopus)85148502832 (WoS)000951172400001
Első szerző:
Roos, Izanne
Cím:
Comparative effectiveness in multiple sclerosis : A methodological comparison / Roos Izanne, Diouf Ibrahima, Sharmin Sifat, Horakova Dana, Havrdova Eva Kubala, Patti Francesco, Shaygannejad Vahid, Ozakbas Serkan, Izquierdo Guillermo, Eichau Sara, Onofrj Marco, Lugaresi Alessandra, Alroughani Raed, Prat Alexandre, Girard Marc, Duquette Pierre, Terzi Murat, Boz Cavit, Grand'Maison Francois, Sola Patrizia, Ferraro Diana, Grammond Pierre, Turkoglu Recai, Buzzard Katherine, Skibina Olga, 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:
7.
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:
8.
001-es BibID:
BIBFORM103333
035-os BibID:
(Scopus)85090969423 (WOS)000607095300025
Első szerző:
Roos, Izanne
Cím:
Delay from treatment start to full effect of immunotherapies for multiple sclerosis / Roos Izanne, Leray Emmanuelle, Frascoli Federico, Casey Romain, Brown J. William L., Horakova Dana, Havrdova Eva K., Trojano Maria, Patti Francesco, Izquierdo Guillermo, Eichau Sara, Onofrj Marco, Lugaresi Alessandra, Prat Alexandre, Girard Marc, Grammond Pierre, Sola Patrizia, Ferraro Diana, Ozakbas Serkan, Bergamaschi Roberto, Sá Maria José, Cartechini Elisabetta, Boz Cavit, Granella Franco, Hupperts Raymond, Terzi Murat, Lechner-Scott Jeannette, Spitaleri Daniele, Van Pesch Vincent, Soysal Aysun, Olascoaga Javier, Prevost Julie, Aguera-Morales Eduardo, Slee Mark, Csepany Tunde, Turkoglu Recai, Sidhom Youssef, Gouider Riadh, Van Wijmeersch Bart, McCombe Pamela, Macdonell Richard, Coles Alasdair, Malpas Charles B., Butzkueven Helmut, Vukusic Sandra, Kalincik Tomas, MSBase and OFSEP investigators
Dátum:
2020
ISSN:
0006-8950
Megjegyzések:
In multiple sclerosis, treatment start or switch is prompted by evidence of disease activity. Whilst immunomodulatory therapies reduce disease activity, the time required to attain maximal effect is unclear. In this study we aimed to develop a method that allows identification of the time to manifest fully and clinically the effect of multiple sclerosis treatments (Ơtherapeutic lag') on clinical disease activity represented by relapses and progression-of-disability events. Data from two multiple sclerosis registries, MSBase (multinational) and OFSEP (French), were used. Patients diagnosed with multiple sclerosis, minimum 1-year exposure to treatment, minimum 3-year pretreatment follow-up and yearly review were included in the analysis. For analysis of disability progression, all events in the subsequent 5-year period were included. Density curves, representing incidence of relapses and 6-month confirmed progression events, were separately constructed for each sufficiently represented therapy. Monte Carlo simulations were performed to identify the first local minimum of the first derivative after treatment start; this point represented the point of stabilization of treatment effect, after the maximum treatment effect was observed. The method was developed in a discovery cohort (MSBase), and externally validated in a separate, non-overlapping cohort (OFSEP). A merged MSBase-OFSEP cohort was used for all subsequent analyses. Annualized relapse rates were compared in the time before treatment start and after the stabilization of treatment effect following commencement of each therapy. We identified 11 180 eligible treatment epochs for analysis of relapses and 4088 treatment epochs for disability progression. External validation was performed in four therapies, with no significant difference in the bootstrapped mean differences in therapeutic lag duration between registries. The duration of therapeutic lag for relapses was calculated for 10 therapies and ranged between 12 and 30 weeks. The duration of therapeutic lag for disability progression was calculated for seven therapies and ranged between 30 and 70 weeks. Significant differences in the pre- versus post-treatment annualized relapse rate were present for all therapies apart from intramuscular interferon beta-1a. In conclusion we have developed, and externally validated, a method to objectively quantify the duration of therapeutic lag on relapses and disability progression in different therapies in patients more than 3 years from multiple sclerosis onset. Objectively defined periods of expected therapeutic lag allows insights into the evaluation of treatment response in randomized clinical trials and may guide clinical decision-making in patients who experience early on-treatment disease activity. This method will subsequently be applied in studies that evaluate the effect of patient and disease characteristics on therapeutic lag.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
multiple sclerosis
therapeutic lag
Megjelenés:
Brain. - 143 : 9 (2020), p. 2742-2756. -
További szerzők:
Leray, Emmanuelle
Frascoli, Federico
Casey, Romain
Brown, J. William L.
Horakova, Dana
Havrdova, Eva
Trojano, Maria
Patti, Francesco
Izquierdo, Guillermo
Eichau, Sara
Onofrj, Marco
Lugaresi, Alessandra
Prat, Alexandre
Girard, Marc
Grammond, Pierre
Sola, Patrizia
Ferraro, Diana
Ozakbas, Serkan
Bergamaschi, Roberto
Sá, Maria José
Cartechini, Elisabetta
Boz, Cavit
Granella, Franco
Hupperts, Raymond
Terzi, Murat
Lechner-Scott, Jeannette
Spitaleri, Daniele
Pesch, Vincent van
Soysal, Aysun
Olascoaga, Javier
Prevost, Julie
Aguera-Morales, Eduardo
Slee, Mark
Csépány Tünde (1956-) (neurológus, pszichiáter)
Turkoglu, Recai
Sidhom, Youssef
Gouider, Riadh
Van Wijmeersch, Bart
McCombe, Pamela
Macdonell, Richard
Coles, Alasdair
Malpas, Charles B.
Butzkueven, Helmut
Vukusic, Sandra
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:
BIBFORM094875
035-os BibID:
(WoS)000677275500001 (Scopus)85099300704
Első szerző:
Roos, Izanne
Cím:
Determinants of therapeutic lag in multiple sclerosis / Izanne Roos, Emmanuelle Leray, Federico Frascoli, Romain Casey, J. William L. Brown, Dana Horakova, Eva Kubala Havrdova, Marc Debouverie, Maria Trojano, Francesco Patti, Guillermo Izquierdo, Sara Eichau, Gilles Edan, Alexandre Prat, Marc Girard, Pierre Duquette, Marco Onofrj, Alessandra Lugaresi, Pierre Grammond, Jonathan Ciron , Aurélie Ruet, Serkan Ozakbas, Jérôme De Seze, Céline Louapre, Hélène Zephir, Maria José Sá, Patrizia Sola, Diana Ferraro, Pierre Labauge, Gilles Defer, Roberto Bergamaschi, Christine Lebrun-Frenay, Cavit Boz, Elisabetta Cartechini, Thibault Moreau, David Laplaud, Jeannette Lechner-Scott, Francois Grand'Maison, Oliver Gerlach, Murat Terzi, Franco Granella, Raed Alroughani, Gerardo Iuliano, Vincent Van Pesch, Bart Van Wijmeersch, Daniele L. A. Spitaleri, Aysun Soysal, Eric Berger, Julie Prevost, Eduardo Aguera-Morales, Pamela McCombe, Tamara Castillo Triviño, Pierre Clavelou, Jean Pelletier, Recai Turkoglu, Bruno Stankoff, Olivier Gout, Eric Thouvenot, Olivier Heinzlef, Youssef Sidhom, Riadh Gouider, Tunde Csepany, Bertrand Bourre, Abdullatif Al Khedr, Olivier Casez, Philippe Cabre, Alexis Montcuquet, Abir Wahab, Jean-Philippe Camdessanche, Aude Maurousset, Ivania Patry, Karolina Hankiewicz, Corinne Pottier, Nicolas Maubeuge, Céline Labeyrie, Chantal Nifle, Alasdair Coles, Charles B. Malpas, Sandra Vukusic, Helmut Butzkueven, Tomas Kalincik, MSBase and OFSEP study groups
Dátum:
2021
ISSN:
1352-4585
Megjegyzések:
Background: A delayed onset of treatment effect, termed therapeutic lag, may influence the assessment of treatment response in some patient subgroups. Objectives: The objective of this study is to explore the associations of patient and disease characteristics with therapeutic lag on relapses and disability accumulation. Methods: Data from MSBase, a multinational multiple sclerosis (MS) registry, and OFSEP, the French MS registry, were used. Patients diagnosed with MS, minimum 1 year of exposure to MS treatment and 3 years of pre-treatment follow-up, were included in the analysis. Studied outcomes were incidence of relapses and disability accumulation. Therapeutic lag was calculated using an objective, validated method in subgroups stratified by patient and disease characteristics. Therapeutic lag under specific circumstances was then estimated in subgroups defined by combinations of clinical and demographic determinants. Results: High baseline disability scores, annualised relapse rate (ARR) ? 1 and male sex were associated with longer therapeutic lag on disability progression in sufficiently populated groups: females with expanded disability status scale (EDSS) < 6 and ARR < 1 had mean lag of 26.6 weeks (95% CI = 18.2-34.9), males with EDSS < 6 and ARR < 1 31.0 weeks (95% CI = 25.3-36.8), females with EDSS < 6 and ARR ? 1 44.8 weeks (95% CI = 24.5-65.1), and females with EDSS ? 6 and ARR < 1 54.3 weeks (95% CI = 47.2-61.5). Conclusions: Pre-treatment EDSS and ARR are the most important determinants of therapeutic lag.
Tárgyszavak:
Orvostudományok
Klinikai orvostudományok
idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Neurology
multiple sclerosis
observational study
therapeutic lag
Megjelenés:
Multiple Sclerosis. - 27 : 12 (2021), p. 1838-1851. -
További szerzők:
Leray, Emmanuelle
Frascoli, Federico
Casey, Romain
Brown, J. William L.
Horakova, Dana
Havrdova, Eva
Debouverie, Marc
Trojano, Maria
Patti, Francesco
Izquierdo, Guillermo
Eichau, Sara
Edan, Gilles
Prat, Alexandre
Girard, Marc
Duquette, Pierre
Onofrj, Marco
Lugaresi, Alessandra
Grammond, Pierre
Ciron, Jonathan
Ruet, Aurélie
Ozakbas, Serkan
De Seze, Jérôme
Louapre, Céline
Zephir, Hélène
Sá, Maria José
Sola, Patrizia
Ferraro, Diana
Labauge, Pierre
Defer, Gilles
Bergamaschi, Roberto
Lebrun-Frenay, Christine
Boz, Cavit
Cartechini, Elisabetta
Moreau, Thibault
Laplaud, David
Lechner-Scott, Jeannette
Grand'Maison, Francois
Gerlach, Oliver
Terzi, Murat
Granella, Franco
Alroughani, Raed
Iuliano, Gerardo
Pesch, Vincent van
Van Wijmeersch, Bart
Spitaleri, Daniele L. A.
Soysal, Aysun
Berger, Eric
Prevost, Julie
Aguera-Morales, Eduardo
McCombe, Pamela
Castillo Triviño, Tamara
Clavelou, Pierre
Pelletier, Jean
Turkoglu, Recai
Stankoff, Bruno
Gout, Olivier
Thouvenot, Eric
Heinzlef, Olivier
Sidhom, Youssef
Gouider, Riadh
Csépány Tünde (1956-) (neurológus, pszichiáter)
Bourre, Bertrand
Al Khedr, Abdullatif
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
Coles, Alasdair
Malpas, Charles B.
Vukusic, Sandra
Butzkueven, Helmut
Kalincik, Tomas
MSBase and OFSEP study groups
Internet cím:
Szerző által megadott URL
DOI
Intézményi repozitóriumban (DEA) tárolt változat
Borító:
Saját polcon:
10.
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:
11.
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:
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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DOI
Intézményi repozitóriumban (DEA) tárolt változat
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