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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
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2.

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
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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
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4.

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
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5.

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
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6.

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
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7.

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
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8.

001-es BibID:BIBFORM103021
035-os BibID:(Wos)000809732300001 (Scopus)85131507832
Első szerző:Sharmin, Sifat
Cím:Confirmed disability progression as a marker of permanent disability in multiple sclerosis / Sharmin Sifat, Bovis Francesca, Malpas Charles, Horakova Dana, Havrdova Eva Kubala, Izquierdo Guillermo, Eichau Sara, Trojano Maria, Prat Alexandre, Girard Marc, Duquette Pierre, Onofrj Marco, Lugaresi Alessandra, Grand'Maison Francois, Grammond Pierre, Sola Patrizia, Ferraro Diana, Terzi Murat, Gerlach Oliver, Alroughani Raed, Boz Cavit, Shaygannejad Vahid, van Pesch Vincent, Cartechini Elisabetta, Kappos Ludwig, Lechner-Scott Jeannette, Bergamaschi Roberto, Turkoglu Recai, Solaro Claudio, Iuliano Gerardo, Granella Franco, Van Wijmeersch Bart, Spitaleri Daniele, Slee Mark, McCombe Pamela, Prevost Julie, Ampapa Radek, Ozakbas Serkan, Sanchez-Menoyo Jose Luis, Soysal Aysun, Vucic Steve, Petersen Thor, de Gans Koen, Butler Ernest, Hodgkinson Suzanne, Sidhom Youssef, Gouider Riadh, Cristiano Edgardo, Castillo-Trivino Tamara, Saladino Maria Laura, Barnett Michael, Moore Fraser, Rozsa Csilla, Yamout Bassem, Skibina Olga, van der Walt Anneke, Buzzard Katherine, Gray Orla, Hughes Stella, Sempere Angel Perez, Singhal Bhim, Fragoso Yara, Shaw Cameron, Kermode Allan, Taylor Bruce, Simo Magdolna, Shuey Neil, Al-Harbi Talal, Macdonell Richard, Dominguez Jose Andres, Csepany Tunde, Sirbu Carmen-Adella, Sormani Maria Pia, Butzkueven Helmut, Kalincik Tomas
Dátum:2022
ISSN:1351-5101
Megjegyzések:Background and purpose: The prevention of disability over the long term is the main treatment goal in multiple sclerosis (MS); however, randomized clinical trials evaluate only short-term treatment effects on disability. This study aimed to define criteria for 6-month confirmed disability progression events of MS with a high probability of resulting in sustained long-term disability worsening. Methods: In total, 14,802 6- month confirmed disability progression events were identified in 8741 patients from the global MSBase registry. For each 6-month confirmed progression event (13,321 in the development and 1481 in the validation cohort), a sustained progression score was calculated based on the demographic and clinical characteristics at the time of progression that were predictive of long-term disability worsening. The score was externally validated in the Cladribine Tablets Treating Multiple Sclerosis Orally (CLARITY) trial. Results: The score was based on age, sex, MS phenotype, relapse activity, disability score and its change from baseline, number of affected functional system domains and worsening in six of the domains. In the internal validation cohort, a 61% lower chance of improvement was estimated with each unit increase in the score (hazard ratio 0.39, 95% confidence interval 0.29?0.52; discriminatory index 0.89). The proportions of progression events sustained at 5 years stratified by the score were 1: 72%; 2: 88%; 3: 94%; 4: 100%. The results of the CLARITY trial were confirmed for reduction of disability progression that was >88% likely to be sustained (events with score ?1.5).Conclusions: Clinicodemographic characteristics of 6-month confirmed disability progression events identify those at high risk of sustained long-term disability. This knowledge will allow future trials to better assess the effect of therapy on long-term disability accrual.
Tárgyszavak:Orvostudományok Klinikai orvostudományok idegen nyelvű folyóiratközlemény külföldi lapban
folyóiratcikk
Megjelenés:European Journal Of Neurology. - 29 : 8 (2022), p. 2321-2334. -
További szerzők:Bovis, Francesca Malpas, Charles Horakova, Dana Havrdova, Eva Izquierdo, Guillermo Eichau, Sara Trojano, Maria Prat, Alexandre Girard, Marc Duquette, Pierre Onofrj, Marco Lugaresi, Alessandra Grand'Maison, Francois Grammond, Pierre Sola, Patrizia Ferraro, Diana Terzi, Murat Gerlach, Oliver Alroughani, Raed Boz, Cavit Shaygannejad, Vahid Pesch, Vincent van Cartechini, Elisabetta Kappos, Ludwig Lechner-Scott, Jeannette Bergamaschi, Roberto Turkoglu, Recai Solaro, Claudio Iuliano, Gerardo Granella, Franco Van Wijmeersch, Bart Spitaleri, Daniele Slee, Mark McCombe, Pamela Prevost, Julie Ampapa, Radek Ozakbas, Serkan Sanchez-Menoyo, Jose Soysal, Aysun Vucic, Steve Petersen, Thor de Gans, Koen Butler, Ernest Hodgkinson, Suzanne Sidhom, Youssef Gouider, Riadh Cristiano, Edgardo Castillo Triviño, Tamara Saladino, Maria Laura Barnett, Michael Moore, Fraser Rózsa Csilla Yamout, Bassem Skibina, Olga Walt, Anneke van der Buzzard, Katherine Gray, Orla Hughes, Stella Sempere, Perez A. Singhal, Bhim Fragoso, Yara Shaw, Cameron Kermode, Allan G. Taylor, Bruce V. Simó Magdolna Shuey, Neil Al-Harbi, Talal Macdonell, Richard Dominguez, Jose Andres Csépány Tünde (1956-) (neurológus, pszichiáter) Sirbu, Carmen-Adella Sormani, Maria Pia Butzkueven, Helmut Kalincik, Tomas
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Intézményi repozitóriumban (DEA) tárolt változat
Borító:

9.

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
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