Objective:

To compare outcomes in pediatric patients with intractable focal seizures taking cenobamate versus their prior antiseizure medications (ASMs) using data from a national claims database.

Background:

Small retrospective studies have reported benefit with off-label cenobamate in pediatric patients with drug-resistant epilepsy.

Design/Methods:

Patients ≤17 years old with a diagnosis of intractable focal seizures taking ≥1 ASM between 5/1/2020-12/31/2022 were identified retrospectively from the HealthVerity Marketplace Private Source 20 claims database. Patients were required to have taken ≥2 prescription fills of cenobamate and have ≥180 days of medical and pharmacy enrollment before and after initiating cenobamate. Patients’ data were analyzed by line of therapy (LOT), whereby a new LOT was identified as the dispensing of a new ASM after ≥30 days without a previous fill. We compared healthcare utilization outcomes (eg, epilepsy-related inpatient days and emergency room [ER] visit days) during the patient’s previous LOTs to outcomes when on cenobamate.

Results:

176 patients with intractable focal seizures (mean age=13.8 years [range: 2-17 years]; 61% male; mean previous LOT=5.5) were included. The median line duration was 333 days for cenobamate vs 365 days for the previous ASMs. The most common previous ASM lines of therapy were cannabidiol (n=31), clobazam (n=23), and lacosamide (n=16). The rate of epilepsy-related inpatient days improved from 5.39 days per year to 4.19 per year, and the rate of epilepsy-related ER days improved from 0.82 days per year to 0.58 per year. Overall, 31.8% of patients on cenobamate required a new ASM to be added.

Conclusions:

Cenobamate was associated with lower inpatient days and ER visits in pediatric patients with intractable focal epilepsy compared to previous therapies. Further studies evaluating the use of cenobamate in pediatric patients with intractable focal seizures are warranted.