Purpose: The safety and effectiveness of
perampanel in clinical settings involving Chinese pediatric patients are limited, as
perampanel has only recently been approved for use in China, in September 2019. We aimed to evaluate the efficacy and tolerability of
perampanel as an adjunctive
therapy for pediatric patients with
epilepsy aged ≥ 4 years in Xinjiang, Northwest China. Methods: Efficacy was assessed by measuring changes in seizure frequency at 3-, 6-, and 12-month follow-up compared with baseline. The baseline was 3 months before the addition of
perampanel, and the seizure frequency was based on the patients' seizure diary. The safety and tolerability depended on the type and frequency of any adverse event during
epilepsy treatment across all pediatric patients. Results: Overall, 67 pediatric patients from 2 different hospitals were enrolled in the study. Among the pediatric patients with
seizures during the baseline period, the effective rates for all seizure types at 3, 6, and 12 months were 59.1%, 58.7%, and 57.4%, respectively. During
perampanel treatment, 34 patients (50.7%) experienced at least 1 adverse reaction. Conclusion: Overall, this real-world retrospective study of pediatric patients validated that
perampanel is an effective treatment option as an adjunctive
therapy among pediatric patients with
epilepsy aged ≥4 years.