AUTHOR=Zhao Fen , Ren Ying , Geng Guifu , Zhang Tong , Hu Wandong , Zhang Huan , Jin Ruifeng , Shi Jianguo , Gao Zaifen , Zhang Hongwei , Liu Yong TITLE=Effectiveness and tolerability of perampanel monotherapy in children with newly diagnosed focal epilepsy JOURNAL=Frontiers in Neurology VOLUME=Volume 14 - 2023 YEAR=2023 URL=https://www.frontiersin.org/journals/neurology/articles/10.3389/fneur.2023.1144759 DOI=10.3389/fneur.2023.1144759 ISSN=1664-2295 ABSTRACT=Objective: To examine the clinical effectiveness and tolerability of perampanel (PER) as initial monotherapy in pediatric patients with newly diagnosed focal epilepsy. Methods: A retrospective analysis was performed on 62 children with newly diagnosed focal epilepsy who were treated with PER in the Epilepsy Center at Jinan Children's Hospital from July 2021 to July 2022. The treatment status, prognosis and adverse reactions were followed up for a minimum of 6 months after PER monotherapy initiation. The effectiveness of the patients was estimated by the PER effective rate at 3-, 6- and 12- month follow-up evaluations, and adverse reactions were also recorded. The effective rates of PER in different etiologies and epilepsy syndromes were also statistically analyzed. Results: The effective rates of PER treatment at the different time points of evaluation were 88.7% (3 months), 79.1% (6 months), and 80.4% (12 months). And with PER treatment, seizure freedom varied over time with 61.3%, 71.0%, and 71.7% of patients at the 3-month, 6-month, and the 12-month follow-up, respectively. Among the etiologies of epilepsy, the effective rates of genetic, structural, and unknown were generally above 50% at the 3-month, 6-month, and the 12-month follow-up. Among the epilepsy syndromes, the categories with higher treatment efficacy were self-limiting epilepsy with central temporal symptom (SeLECTs), self-limited epilepsy with autonomic seizures (SeLEAS), and children occipital visual epilepsy (COVE), with the effective rate of above 80%. Adverse events were documented in 22 patients (35.5%), but mild and tolerable. The most common adverse events comprised irritability, drowsiness, dizziness, and increased appetite. Conclusion: PER has a favorable effectiveness and tolerability as initial monotherapy for childhood newly diagnosed focal epilepsy, which could be a potential option for long-term medication in the treatment of focal epilepsy in children. The current study provided potential evidence for PER as initial monotherapy in children with focal epilepsy in clinical practice.