Using Cenobamate for managing idiopathic generalized epilepsy: A single-center experience and systematic literature review.

OBJECTIVE: To evaluate the single-center experience of Cenobamate's (CNB) off-label use in adults with idiopathic generalized epilepsy (IGE). METHODS: We conducted a retrospective review of electronic health records within a large healthcare system to identify IGE patients prescribed CNB. Extensive clinical and medication-related data were extracted, including monthly seizure frequency at baseline, 3, 6, and 12 months, and at last follow-up. We used CNB retention, a marker of its potential effectiveness and tolerability, for 12 months as the primary outcome of interest. Statistical analysis, including Kaplan-Meier survival analysis, was performed to estimate CNB retention over time. We also performed a systematic literature review investigating seizure reduction after CNB use in IGE. RESULTS: Sixteen IGE patients (median age = 36 years; 11 females) treated with CNB were included. Twelve (75%) had JME. Median epilepsy duration was 18 years, and a median of 8 ASMs were tried before CNB use. Among the 14 patients with at least a 12-month follow-up, 8 (57%) achieved the primary outcome. Median CNB retention based on survival analysis was 34 months, and 31% of patients stopped CNB within 3 months. At 3, 6, and 12 months, ≥50% seizure reduction was seen in 75%, 86%, and 63% of patients, respectively. Of 8 patients on CNB at last follow-up (median CNB use of 21 months), 3 (38%) were seizure-free. Median ER visits declined significantly in the 6 months post-CNB (p = 0.028; Cohen's d = 0.66). Fatigue, dizziness, and cognitive symptoms were the most frequently reported adverse effects. Systematic review revealed 3 articles with 4 IGE patients whose seizure outcomes were similar to our findings. SIGNIFICANCE: The study found CNB's use in IGE to be effective, including decreased ER visits. These findings provide preliminary evidence to suggest that off-label use of CNB can be considered in drug-resistant IGE patients. PLAIN LANGUAGE SUMMARY: Cenobamate (CNB), an anti-seizure medicine, was studied for off-label use in 16 adults with drug-resistant idiopathic generalized epilepsy (IGE). Most had juvenile myoclonic epilepsy and long-standing seizures despite many prior treatments. Over half continued CNB for at least 12 months, and around 30% stopped within 3 months. At 3, 6, and 12 months, 63%-86% had at least 50% fewer seizures; three patients became seizure-free. Emergency visits dropped significantly after starting CNB. Common side effects were fatigue, dizziness, and cognitive issues. These findings suggest CNB can help reduce seizures and healthcare use in adults with difficult-to-treat IGE, warranting further study.