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. 2025 Jul 30.
doi: 10.1111/epi.18586. Online ahead of print.

Real-world use of cenobamate in pediatric focal epilepsies and developmental epileptic encephalopathies: A multicenter retrospective series

Víctor Soto-Insuga  1 , Adrián Valls Carbó  2 , Anna Gretel Pinzón-Acevedo  2 , Elena González-Alguacil  1 , Juan José García Peñas  1 , Andrea Sariego Jamardo  3 , Gemma Aznar-Laín  4 , Salvador Ibáñez-Micó  5 , Helena Alarcón Martínez  5 , Raquel Buenache  6 , Saray Rekarte  6 , Andrea Parejo Olivera  2   7 , Ezequiel Tuero-Montiel  2 , Jana DomínguezCarral  8 , María López  9 , Ainhoa García Ribes  10 , María Jesús Martínez González  10 , Eva Arias  11 , Adrián García Ron  11 , Susana Boronat  12 , Eulalia Turón Viñas  12 , Dolors Casellas  13 , Virginia Navarro  14 , David Conejo  14 , Elena Miravet  15 , Irene Sánchez-Miranda Román  16 , Antonio Gil Nagel-Rein  16   17 , María Muñoz Cabeza  18 , Patricia Smeyers  19 , Ángel Aledo-Serrano  2
Affiliations

Real-world use of cenobamate in pediatric focal epilepsies and developmental epileptic encephalopathies: A multicenter retrospective series

Víctor Soto-Insuga et al. Epilepsia. .

Abstract

Objective: Cenobamate (CNB) is an anti-seizure medication approved for focal-onset seizures in adults, with growing evidence supporting its use in pediatric drug-resistant epilepsy (DRE). This study evaluates the efficacy, retention, and safety of CNB in children and adolescents, including those with developmental and epileptic encephalopathies (DEEs).

Methods: We conducted a retrospective, multicenter study of 169 pediatric patients (0-18 years) with DRE treated with CNB across centers in Spain. Seizure response (≥50% reduction) and seizure freedom (no seizures in the previous month) were assessed at 3, 6, and 12 months.

Results: At 12 months, CNB showed a retention rate of 89.2%, with 83.9% of patients achieving seizure response and 19.6% reaching seizure freedom. Response rates were 73.4% at 3 months and 77.1% at 6 months, with seizure freedom increasing from 11.8% at 3 months to 21.1% at 6 months. Outcomes were comparable between DEEs and focal epilepsies, indicating broad-spectrum efficacy. Seizure worsening occurred in 4.7% of patients, all with DEEs. After adjusting for seizure type, those patients with focal seizures had higher odds of achieving seizure response (odds ratio [OR] 95% confidence interval [95% CI] 5.46, 1.27-23.52; p = 0.02) and seizure freedom (OR 95% CI 5.32, 1.57-17.97; p < 0.01). Median CNB doses (mg/kg/day) were 1.78 (range 0.37-12.5), 2.5 (range 0.67-12.5), and 3.19 (range 0.96-9.09) at 3, 6, and 12 months, respectively. Adverse events occurred in 44.9% of patients, most commonly somnolence (42.3%) and dizziness (22.4%), which improved with slower titration.

Significance: CNB appears effective and well-tolerated in pediatric DRE, including DEE, with high retention and sustained efficacy over time. Adverse events were generally manageable with dose adjustments, and slow, weight-based titration improved tolerability. Prospective studies are warranted to confirm these findings.

Keywords: antiseizure medications; cenobamate; developmental and epileptic encephalopathies; focal seizures; pediatric epilepsy.

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References

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