Adjunctive cenobamate in children and adolescents – real-world data from a retrospective multicenter study

Background: There is increasing evidence supporting the use of cenobamate in adults with focal epilepsy yet pediatric data remain limited. Methods: We performed a retrospective multicenter real-world study in pediatric epilepsy referral centers. Patients were included if they had drug-resistant epilepsy between the ages of 0-21 years and received cenobamate with at least 6 months follow-up. The primary outcome was a ≥50% reduction in seizure frequency at 6 and 12 months. Secondary outcomes were retention rates, seizure freedom, adverse effects, and change in concomitant anti-seizure medicines (ASMs). We extensively characterized seizure and epilepsy types, etiology, previous and concomitant ASMs, history of epilepsy surgery and neuromodulation and developmental background. Results: Of 94 patients included, median age of epilepsy onset was 4.3 years, with developmental delay in 72%. Cenobamate was initiated after a median of 7 previous ASMs at a median age of 14 years, (range 1.2–21.8 years, SD 4.18) with mean final dosing of 3.22 mg/kg ≥50% seizure reduction at 6 months was achieved in 41%, increasing to 54% at 12 months. Median follow-up duration was 12 months with 40% retention. Adverse effects were reported in 24% of patients at 6 months. Dose reduction of concomitant ASMs was performed in 55% of patients with other ASMs discontinued in 35%. Conclusion: Our study provides evidence that cenobamate is an effective and well-tolerated ASM in children and adolescents, including in the younger pediatric population. Our findings support the administration of cenobamate in children with focal, drug-resistant epilepsy as adjunctive therapy alongside the use of more established ASMs.