Efficacy and safety of perampanel monotherapy in Chinese patients with focal‐onset seizures: A single‐center, prospective, real‐world observational study

OBJECTIVE: Efficacy and safety of perampanel monotherapy for treating focal‐onset seizures (FOS) has been barely studied in China. This observational study aimed to evaluate the efficacy and safety of perampanel monotherapy in treating Chinese patients with FOS. METHODS: This single‐center, prospective, real‐world observational study enrolled patients aged ≥4 years with FOS who visited the Epilepsy Out‐Patient Clinic of Nanjing Brain Hospital affiliated to Nanjing Medical University from January 2020 to December 2021. All patients were treated with perampanel monotherapy. Seizure‐freedom rates after 6 and 12 months of treatment were calculated. Adverse events (AEs) were recorded. RESULTS: Seventy patients with FOS were enrolled. The mean maintenance perampanel dose was 4.64 ± 1.55 mg/day. The 6‐ and 12‐month retention rates of perampanel monotherapy were 78.6% (55/70) and 70.0% (49/70), respectively. The 6‐ and 12‐month seizure‐freedom rates were 69.84% (44/63) and 65.08% (41/63), respectively. Patients with focal to bilateral tonic–clonic seizures had significantly higher 6‐month and numerically higher 12‐month seizure freedom rates than patients with focal impaired awareness seizures (P = 0.046 and P = 0.204, respectively). Twenty‐six (37.1%) patients experienced treatment‐emergent AEs, and the most common AE was dizziness. Four (5.7%) patients withdrew from the study due to AEs. No new safety concern was observed. SIGNIFICANCE: This is the first prospective study on the efficacy and safety of perampanel monotherapy in treating Chinese patients with FOS, and perampanel monotherapy was effective and safe in treating Chinese patients aged ≥4 years with FOS up to 12 months. More multicenter, real‐world studies with large sample sizes and longer follow‐ups are needed to further evaluate the long‐term efficacy and safety of perampanel monotherapy.