Abstracts

Rationale: An estimated 65 million people worldwide suffer from epilepsy with partial seizures being the most common form. In some patients, seizures are not improved even while taking multiple prescribed anti-seizure medications (ASMs). Cenobamate was approved by the U.S. Food and Drug Administration (FDA) in November 2019 to treat partial seizures in adults after its initial testing by SK Life Science, beginning in 2013. Prior studies demonstrated robust efficacy in adults. To date, no studies exist to determine its efficacy in children and adolescents, nor in subpopulations excluded from prior studies (i.e., patients with a history of rash). We reviewed our real-world data in adolescents and adults to help discover effectiveness for these populations and to support use of this new medication.

Methods: We performed a retrospective chart review of all patients presenting with a diagnosis of epilepsy who were prescribed cenobamate at Le Bonheur Children’s Hospital. Data collected include demographic data, the number of prior anti-seizure medications, history of rash to prior ASMs, seizure frequency pre- and post-exposure to cenobamate, daily dosages per age group, and any novel adverse effects.

Results: Overall, 41 patients included in this study had prior exposure to an average of 13.3 ASMs before pursuing treatment with cenobamate, and 80% (n=33) had prior epilepsy surgery or device treatment. Of the 41 patients involved in the study (ages 13–44), 31.8% were between the ages of 12–17 years, and 68.2% were 18 years of age or older. The average duration of treatment (to date) is 8.1 months. Twenty-two (54%) were responders (i.e., they obtained at least a 50% decrease in the number of partial seizures); 58.3% of adolescent patients and 51.7% of adults obtained this extent of seizure improvement. Two patients discontinued the medication—one due to increased seizure frequency, and one due to a lack of improvement. Of the 5 patients with prior history of rash associated with ASMs, none experienced a rash related to cenobamate, and of the 25 patients with a prior history of drug allergy to any medicine, none experienced a rash after taking cenobamate. The average daily dosage for cenobamate was 188.5 mg/day in adolescents and 212.1 mg/day in adults.

Conclusions: This study provided evidence to support the findings of efficacy from previous regulatory trials of cenobamate, extending it into the adolescent age group. No new adverse effects were reported. In addition, none of our patients with prior history of rash to any medication or specifically to other anti-seizure medications experienced a rash from cenobamate (using the approved dose titration), indicating the medication can be used safely in this population. In this group of patients with very refractory epilepsy, based on their number of prior ASMs and surgical procedures, over 50% of the adolescent (12–17 years of age) and adult (18+) patients showed at least 50% seizure reduction following the addition of cenobamate to their prescribed medical regimen.

Funding: Please list any funding that was received in support of this abstract.: Le Bonheur Neuroscience Institute.