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Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program

OBJECTIVE: This study investigated early, real‐world outcomes with cenobamate (CNB) in a large series of patients with highly drug‐resistant epilepsy within a Spanish Expanded Access Program (EAP). METHOD: This was a multicenter, retrospective, observational study in 14 hospitals. Inclusion criteria...

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Autores principales: Villanueva, Vicente, Santos‐Carrasco, Daniel, Cabezudo‐García, Pablo, Gómez‐Ibáñez, Asier, Garcés, Mercedes, Serrano‐Castro, Pedro, Castro‐Vilanova, Maria D., Sayas, Débora, Lopez‐Gonzalez, Francisco J., Rodríguez‐Osorio, Xiana, Torres‐Gaona, Gustavo, Saiz‐Diaz, Rosa A., Hampel, Kevin G., Martinez‐Ferri, Meritxell, Aguilar‐Amat, Maria J., Mercedes‐Alvarez, Blanca, García‐Morales, Vanessa, del Villar‐Igea, Ana, Massot‐Tarrús, Andreu, Rodríguez‐Uranga, Juan J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10472366/
https://www.ncbi.nlm.nih.gov/pubmed/37149853
http://dx.doi.org/10.1002/epi4.12757
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author Villanueva, Vicente
Santos‐Carrasco, Daniel
Cabezudo‐García, Pablo
Gómez‐Ibáñez, Asier
Garcés, Mercedes
Serrano‐Castro, Pedro
Castro‐Vilanova, Maria D.
Sayas, Débora
Lopez‐Gonzalez, Francisco J.
Rodríguez‐Osorio, Xiana
Torres‐Gaona, Gustavo
Saiz‐Diaz, Rosa A.
Hampel, Kevin G.
Martinez‐Ferri, Meritxell
Aguilar‐Amat, Maria J.
Mercedes‐Alvarez, Blanca
García‐Morales, Vanessa
del Villar‐Igea, Ana
Massot‐Tarrús, Andreu
Rodríguez‐Uranga, Juan J.
author_facet Villanueva, Vicente
Santos‐Carrasco, Daniel
Cabezudo‐García, Pablo
Gómez‐Ibáñez, Asier
Garcés, Mercedes
Serrano‐Castro, Pedro
Castro‐Vilanova, Maria D.
Sayas, Débora
Lopez‐Gonzalez, Francisco J.
Rodríguez‐Osorio, Xiana
Torres‐Gaona, Gustavo
Saiz‐Diaz, Rosa A.
Hampel, Kevin G.
Martinez‐Ferri, Meritxell
Aguilar‐Amat, Maria J.
Mercedes‐Alvarez, Blanca
García‐Morales, Vanessa
del Villar‐Igea, Ana
Massot‐Tarrús, Andreu
Rodríguez‐Uranga, Juan J.
author_sort Villanueva, Vicente
collection PubMed
description OBJECTIVE: This study investigated early, real‐world outcomes with cenobamate (CNB) in a large series of patients with highly drug‐resistant epilepsy within a Spanish Expanded Access Program (EAP). METHOD: This was a multicenter, retrospective, observational study in 14 hospitals. Inclusion criteria were age ≥18 years, focal seizures, and EAP authorization. Data were sourced from patient clinical records. Primary effectiveness endpoints included reductions (100%, ≥90%, ≥75%, and ≥50%) or worsening in seizure frequency at 3‐, 6‐, and 12‐month visits and at the last visit. Safety endpoints included rates of adverse events (AEs) and AEs leading to discontinuation. RESULTS: The study included 170 patients. At baseline, median epilepsy duration was 26 years and median number of seizures/month was 11.3. The median number of prior antiseizure medications (ASMs) and concomitant ASMs were 12 and 3, respectively. Mean CNB dosages/day were 176 mg, 200 mg, and 250 mg at 3, 6, and 12 months. Retention rates were 98.2%, 94.5%, and 87% at 3, 6, and 12 months. At last available visit, the rate of seizure freedom was 13.3%; ≥90%, ≥75%, and ≥50% responder rates were 27.9%, 45.5%, and 63%, respectively. There was a significant reduction in the number of seizures per month (mean: 44.6%; median: 66.7%) between baseline and the last visit (P < 0.001). Responses were maintained regardless of the number of prior or concomitant ASMs. The number of concomitant ASMs was reduced in 44.7% of patients. The cumulative percentage of patients with AEs and AEs leading to discontinuation were 68.2% and 3.5% at 3 months, 74.1% and 4.1% at 6 months, and 74.1% and 4.1% at 12 months. The most frequent AEs were somnolence and dizziness. SIGNIFICANCE: In this highly refractory population, CNB showed a high response regardless of prior and concomitant ASMs. AEs were frequent but mostly mild‐to‐moderate, and few led to discontinuation.
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spelling pubmed-104723662023-09-02 Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program Villanueva, Vicente Santos‐Carrasco, Daniel Cabezudo‐García, Pablo Gómez‐Ibáñez, Asier Garcés, Mercedes Serrano‐Castro, Pedro Castro‐Vilanova, Maria D. Sayas, Débora Lopez‐Gonzalez, Francisco J. Rodríguez‐Osorio, Xiana Torres‐Gaona, Gustavo Saiz‐Diaz, Rosa A. Hampel, Kevin G. Martinez‐Ferri, Meritxell Aguilar‐Amat, Maria J. Mercedes‐Alvarez, Blanca García‐Morales, Vanessa del Villar‐Igea, Ana Massot‐Tarrús, Andreu Rodríguez‐Uranga, Juan J. Epilepsia Open Original Articles OBJECTIVE: This study investigated early, real‐world outcomes with cenobamate (CNB) in a large series of patients with highly drug‐resistant epilepsy within a Spanish Expanded Access Program (EAP). METHOD: This was a multicenter, retrospective, observational study in 14 hospitals. Inclusion criteria were age ≥18 years, focal seizures, and EAP authorization. Data were sourced from patient clinical records. Primary effectiveness endpoints included reductions (100%, ≥90%, ≥75%, and ≥50%) or worsening in seizure frequency at 3‐, 6‐, and 12‐month visits and at the last visit. Safety endpoints included rates of adverse events (AEs) and AEs leading to discontinuation. RESULTS: The study included 170 patients. At baseline, median epilepsy duration was 26 years and median number of seizures/month was 11.3. The median number of prior antiseizure medications (ASMs) and concomitant ASMs were 12 and 3, respectively. Mean CNB dosages/day were 176 mg, 200 mg, and 250 mg at 3, 6, and 12 months. Retention rates were 98.2%, 94.5%, and 87% at 3, 6, and 12 months. At last available visit, the rate of seizure freedom was 13.3%; ≥90%, ≥75%, and ≥50% responder rates were 27.9%, 45.5%, and 63%, respectively. There was a significant reduction in the number of seizures per month (mean: 44.6%; median: 66.7%) between baseline and the last visit (P < 0.001). Responses were maintained regardless of the number of prior or concomitant ASMs. The number of concomitant ASMs was reduced in 44.7% of patients. The cumulative percentage of patients with AEs and AEs leading to discontinuation were 68.2% and 3.5% at 3 months, 74.1% and 4.1% at 6 months, and 74.1% and 4.1% at 12 months. The most frequent AEs were somnolence and dizziness. SIGNIFICANCE: In this highly refractory population, CNB showed a high response regardless of prior and concomitant ASMs. AEs were frequent but mostly mild‐to‐moderate, and few led to discontinuation. John Wiley and Sons Inc. 2023-05-21 /pmc/articles/PMC10472366/ /pubmed/37149853 http://dx.doi.org/10.1002/epi4.12757 Text en © 2023 The Authors. Epilepsia Open published by Wiley Periodicals LLC on behalf of International League Against Epilepsy. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Original Articles
Villanueva, Vicente
Santos‐Carrasco, Daniel
Cabezudo‐García, Pablo
Gómez‐Ibáñez, Asier
Garcés, Mercedes
Serrano‐Castro, Pedro
Castro‐Vilanova, Maria D.
Sayas, Débora
Lopez‐Gonzalez, Francisco J.
Rodríguez‐Osorio, Xiana
Torres‐Gaona, Gustavo
Saiz‐Diaz, Rosa A.
Hampel, Kevin G.
Martinez‐Ferri, Meritxell
Aguilar‐Amat, Maria J.
Mercedes‐Alvarez, Blanca
García‐Morales, Vanessa
del Villar‐Igea, Ana
Massot‐Tarrús, Andreu
Rodríguez‐Uranga, Juan J.
Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title_full Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title_fullStr Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title_full_unstemmed Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title_short Real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: Outcomes from an Expanded Access Program
title_sort real‐world safety and effectiveness of cenobamate in patients with focal onset seizures: outcomes from an expanded access program
topic Original Articles
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10472366/
https://www.ncbi.nlm.nih.gov/pubmed/37149853
http://dx.doi.org/10.1002/epi4.12757
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