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Adjunctive use of cenobamate for pediatric refractory focal-onset epilepsy: A single-center retrospective study

      Highlights

      • Cenobamate is a useful adjunctive therapy for refractory focal-onset epilepsy in adult patients, but the safety and efficacy is yet to be established in the pediatric population.
      • We performed a retrospective analysis of a pediatric cohort of refractory focal-onset epilepsy receiving cenobamate as an adjunctive therapy.
      • We measured seizure reduction, median reduction in seizure frequency, median dose, responder rate, and treatment-emergent adverse events.
      • Cenobamate showed similar rates of efficacy, safety, and tolerability in the pediatric population when compared to adult data, even when used at maximum dose of 400 mg.

      Abstract

      Objective

      We explored the efficacy and safety profile of cenobamate as an adjunctive therapy in patients with refractory focal-onset epilepsy in the pediatric population.

      Methods

      This was a retrospective, single-center study of cenobamate used as an adjunctive medication in pediatric patients with refractory focal-onset epilepsy . We measured seizure reduction, median reduction in seizure frequency, median dose, responder rate, and treatment-emergent adverse events.

      Results

      We studied the efficacy and safety profile of cenobamate in 21 pediatric patients (mean age 15.9). Cenobamate was up titrated using the prescribed starter pack with final doses ranging from 100 mg to 400 mg daily. The mean and median dose of cenobamate was 209.8 mg (±98.87 mg) and 200 mg (175–275), respectively. For patients weighing less than 50 kg, mean and median dose was 4.0 mg/kg/day (3.20–4.63) and 4.32 mg/kg/day, respectively. Mean and median baseline seizure frequency per month in this cohort was 15.38 and 16, respectively, prior to the introduction of cenobamate. After the adjunctive use of cenobamate, mean and median seizure frequency per month reduced to 7.29 and 1, respectively; median reduction in seizure frequency was 93.7%. Seizure reduction of at least 50% (responder rate) was noted in 13 (62.5%) patients and a seizure reduction of at least 75% noted in 11 (52.4%) patients, similar to that seen in adults. Four patients (19%) achieved seizure freedom. Of the 21 pediatric patients, 9 (42.8%) patients had treatment-emergent adverse events (TEAE) with the most commonly reported symptom being ataxia (5, 23.8%) and sedation (2, 9.5%). Three (14.3%) patients discontinued early due to these side effects. No children developed drug rash with eosinophilia and systemic symptoms (DRESS).

      Conclusion

      Cenobamate demonstrates similar efficacy rates and safety profile within the pediatric population when compared to the published adult data, making it an effective, safe, and tolerable adjunctive medication for children with refractory focal-onset epilepsy, even at the maximum daily dose.

      Keywords

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