Real-world safety and effectiveness of cenobamate in patients with focal onset seizures: outcomes from an Expanded Access Program.

Fecha de publicación: 07/05/2023 Fecha Ahead of Print: 07/05/2023

Autores de IIS La Fe

• Vicente Enrique Villanueva Haba

  Autor

• Asier Gomez Ibañez

  Autor

• Mercedes Garcés Sánchez

  Autor

Participantes ajenos a IIS La Fe

• Santos-Carrasco, Daniel
• Cabezudo-Garcia, Pablo
• Serrano-Castro, Pedro
• Castro-Vilanova, Maria D.
• Sayas, Debora
• Lopez-Gonzalez, Francisco J.
• Rodriguez-Osorio, Xiana
• Torres-Gaona, Gustavo
• Saiz-Diaz, Rosa A.
• Hampel, Kevin G.
• Martinez-Ferri, Meritxell
• Aguilar-Amat, Maria J.
• Mercedes-Alvarez, Blanca
• Garcia-Morales, Vanessa
• del Villar-Igea, Ana
• Massot-Tarrus, Andreu
• Rodriguez-Uranga, Juan J.

Abstract

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 multicentre, retrospective, observational study in 14 hospitals. Inclusion criteria were age =18years, focal seizures and EAP authorization. Data were sourced from patient clinical records. Primary effectiveness endpoints included reductions (100%, =90%, =75%, =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 26years 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.

© 2023 The Authors. Epilepsia Open published by Wiley Periodicals LLC on behalf of International League Against Epilepsy.

Keywords

• antiseizure medication; epilepsy; refractory