Abstracts

Rationale: Treatment resistant epilepsy is a common problem amongst pediatric patients and can lead to significant morbidity. Approximately 30% of children with epilepsy are refractory to anti-seizure medications (ASM). Cenobamate is a novel ASM that reduces neuronal activity and seizures by inhibiting voltage-gated sodium channels in addition to modulating y-aminobutyric acid (GABA-A) ion channels. Cenobamate was recently approved for localization-related epilepsy in adults and was shown to have reduced seizures by up to 65% in randomized controlled trials. The results have been promising in the adult population, but to date there is little data on use of this medication in children.

Methods: This study is an open label retrospective single institution chart review. A total of 20 patients (60% male), ages 2 to 18 years were started on cenobamate for both focal (65%) and generalized treatment resistant epilepsy. Initial dosages ranged from 0.1 to 0.7mg/kg/day with the lowest initial starting dose being 3.125mg daily. Patients were titrated every-other-week to a daily target dose ranging from 0.9 to 8.3mg/kg/day, with the largest daily dose of 200mg daily. Most patients were started on one 12.5mg tablet daily for two weeks followed by the subsequent up-titration. Patients were followed on an outpatient basis for 3-11 months.

Results: A total of 20 patients were started on cenobamate, 16 of whom have had follow up since initiation. These patients had varying degrees of seizure burden prior to medication onset. Of the patients who were seen in follow up, 10 of the 16 (63%) patients showed significant improvement in their seizure frequency. Adverse side effects required discontinuation of the medication occurred in 2 patients. The most common adverse effect was fatigue (4 patients), but other adverse effects encountered included hair loss, drooling and slurred speech, urinary changes (pungent smell), extreme emotional lability and suicidality (one patient). One patient was unable to continue due to financial concerns.

Conclusions: This review suggests that cenobamate may have a role in the management of pediatric epilepsy, as more than 60% of the patients reviewed in this study showed an improvement in seizure frequency. The data also suggests cenobamate may be beneficial for patients with generalized as well as focal epilepsy. Further studies are required, including randomized controlled clinical trials in order to better understand dosing, tolerability and safety of cenobamate in the pediatric population.

Funding: Please list any funding that was received in support of this abstract.: None.