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

There is increasing evidence supporting the use of cenobamate in adults with focal epilepsy yet pediatric data remain limited.

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.

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%.

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.