Efficacy and safety of perampanel in genetic generalized epilepsy: A retrospective, single-center study in China

Perampanel (PER) is a selective non-competitive AMPA receptor antagonist approved for treating focal and generalized seizures. However, its efficacy in genetic generalized epilepsy (GGE) has not been extensively studied in Chinese populations. This retrospective, single-center study enrolled 54 patients with GGE treated with PER between March 2021 and November 2023. To ensure data quality and minimize bias, we implemented standardized data collection procedures including: (1) systematic documentation using standardized seizure diaries, (2) regular follow-up assessments at predefined intervals, and (3) rigorous application of inclusion/exclusion criteria. Efficacy was assessed by seizure freedom rate, responder rate (≥50 % seizure reduction) and retention rate at 3, 6, 12 months and last follow-up. Safety was evaluated by monitoring adverse events. At last follow-up (mean 14 ± 4.95 months), the overall seizure freedom rate was 53.7 % and responder rate was 70.4 %. Idiopathic generalized epilepsy (IGE) patients showed better outcomes compared to non-IGE patients (seizure freedom: 63.6 % vs 10 %; responder rate: 79.5 % vs 30 %). PER demonstrated highest efficacy in generalized tonic-clonic seizures (80.4 % responder rate), followed by myoclonic (70.8 %) and absence seizures (50 %). Among epilepsy syndromes, GTCA showed the best response (100 % responder rate), followed by JME (83.3 %). The mean effective dose was 3.86 mg/day in the seizure-free group. Treatment-emergent adverse events occurred in 18.5 % of patients, with dizziness (18.5 %) being most common, leading to discontinuation in 3.7 % of cases. PER demonstrated favorable efficacy and tolerability in Chinese patients with GGE, particularly in IGE patients and those with generalized tonic-clonic seizures. Lower doses were associated with better outcomes, suggesting careful dose titration may optimize therapeutic benefits.