# Effectiveness and Safety of Perampanel in Refractory Focal Epilepsy: Real‐World Evidence From a Chinese Cohort

## TL;DR

The study shows that perampanel, when added to existing treatments, effectively reduces seizures in patients with hard-to-treat focal epilepsy, with good safety over a year.

## Contribution

This study provides real-world evidence on perampanel's effectiveness and safety in a Chinese cohort with refractory focal epilepsy.

## Key findings

- Perampanel achieved sustained responder rates of about 66% and seizure freedom of about 27% over 12 months.
- Higher doses and late add-on use of perampanel predicted better outcomes, while adults experienced fewer adverse events.

## Abstract

To evaluate the effectiveness and safety of perampanel (PER) as an add‐on treatment in patients with refractory focal epilepsy.

This single‐center, retrospective observational study consecutively enrolled patients with refractory focal epilepsy who initiated PER as adjunctive therapy between September 2020 and October 2021. The primary outcomes included PER effectiveness (≥ 50% seizure reduction), treatment retention rates, and adverse event (AE) profiles, systematically evaluated at 3‐, 6‐, and 12‐month follow‐up intervals.

This study included 190 patients (median age 27 years; 51.6% female). At 3/6/12‐month follow‐ups, response rates were 65.5%/64.9%/66.1%, seizure‐free rates 26.2%/26.2%/26.8%, and retention rates 88.4%/69.0%/66.8%, respectively. Logistic regression analysis indicated that females had a lower response rate (OR = 0.38, 95% CI = 0.20.74, p = 0.005). Responders had a higher PER dose (≥ 6 mg) than nonresponders at all follow‐ups. Patients receiving late add‐on PER treatment had significantly higher response and seizure‐free rate than those receiving early add‐on therapy. AEs were reported in 41.1% of patients, mostly mild to moderate in severity. The most frequent AEs were dizziness (26.3%), somnolence (13.0%), and psychiatric symptoms (12.11%). Additionally, patients aged ≥ 18 years had a lower risk of AEs (OR = 0.41, 95% CI: 0.19–0.88, p = 0.023).

PER demonstrated clinically meaningful efficacy and acceptable safety in refractory focal epilepsy, with sustained response rates and seizure freedom over 12 months. Male gender, higher doses, and late add‐on use predicted better outcomes, while adults experienced fewer adverse events. These real‐world findings support PER's role in treatment‐resistant cases.

This retrospective real‐world study of 190 Chinese patients with refractory focal epilepsy demonstrated that adjunctive PER achieved sustained responder rates (∼66%) and seizure freedom (∼27%) over 12 months, with an acceptable safety profile. Male gender, higher doses, and late add‐on predicted better outcomes, while adults experienced fewer adverse events.