Gu Yanxia, Li Yue, Li Wei, Chen Feng, Wu Chunfeng, Chen Jing
Department of Neurology, Children's Hospital of Nanjing Medical University, Nanjing, China.
Department of Pharmacy, Pharmaceutical Sciences Research Center, Children's Hospital of Nanjing Medical University, Nanjing, China.
Epilepsia Open. 2024 Dec;9(6):2209-2218. doi: 10.1002/epi4.13043. Epub 2024 Sep 18.
To assess the efficacy and safety of perampanel (PER) as primary monotherapy in patients aged 4-18 years old with epilepsy.
A single-center, prospective, observational study was conducted from October 2021 to October 2023, to evaluate PER monotherapy's efficacy and safety as initial therapy for pediatric epilepsy. Changes in seizure frequency, safety, and retention rate were observed at 3, 6, 9, and 12 months after initiating PER primary monotherapy.
A total of 124 children aged 4-15 years (mean age = 8.25 ± 2.50 years) were included in the Analysis Sets. The retention rates at 3, 6, 9, and 12 months were 88.71% (110/124), 84.68% (105/124), 78.26% (90/115), and 71.58% (68/95), respectively. Seizure freedom rates at 3, 6, 9, and 12 months were 85.45%, 79.09%, 76.24%, and 75.31%, respectively. The responder rates (≥50% but <100%) at the same endpoints were 9.09%, 14.55%, 12.87%, and 7.41%, respectively. Seizure freedom rate of PER was independent of age at PER initiation, seizure onset age, gender, baseline frequency, seizure types, and family history of epilepsy (p > 0.05) but associated with duration of treatment (p = 0.001) and maintenance dose (p = 0.022). Additionally, 124 patients were included in the safety analysis set. The overall adverse event rate was 38.71% (48/124), with irritability (19 cases, 15.32%) and dizziness (18 cases, 14.52%) being the most common adverse effects. One patient discontinued PER monotherapy within 1 month due to unbearable itching of the skin.
PER monotherapy as the primary anti-seizure medication (ASM) for pediatric epilepsy demonstrates high efficacy and safety in real-world clinical treatment. Patients who respond well to this drug and adhere to long-term treatment can achieve favorable seizure control. Furthermore, patients achieving seizure freedom with a relatively lower dose can opt for the same dose as the maintenance dose.
This study provided the efficacy and safety of PER monotherapy as the primary ASM for Chinese pediatric epilepsy. In total, 124 patients took part. The seizure freedom rates were over 70% at different observation points (OPs), along with a retention rate of 71.58% at the 12-month OP. Most of adverse effects observed were mild to moderate.
评估吡仑帕奈（PER）作为4至18岁癫痫患者一线单药治疗的疗效和安全性。
于2021年10月至2023年10月进行了一项单中心、前瞻性观察性研究，以评估PER单药治疗作为小儿癫痫初始治疗的疗效和安全性。在开始PER一线单药治疗后的3、6、9和12个月观察癫痫发作频率、安全性和保留率的变化。
分析集纳入了124名4至15岁儿童（平均年龄 = 8.25 ± 2.50岁）。3、6、9和12个月时的保留率分别为88.71%（110/124）、84.68%（105/124）、78.26%（90/115）和71.58%（68/95）。3、6、9和12个月时的无癫痫发作率分别为85.45%、79.09%、76.24%和75.31%。相同终点时的反应率（≥50%但<100%）分别为9.09%、14.55%、12.87%和7.41%。PER的无癫痫发作率与开始使用PER时的年龄、癫痫发作起始年龄、性别、基线频率、癫痫发作类型和癫痫家族史无关（p > 0.05），但与治疗持续时间（p = 0.001）和维持剂量（p = 0.022）有关。此外，124名患者纳入安全性分析集。总体不良事件发生率为38.71%（48/124），最常见的不良反应为易怒（19例，15.32%）和头晕（18例，14.52%）。1例患者因皮肤瘙痒难忍在1个月内停用PER单药治疗。
PER单药治疗作为小儿癫痫的一线抗癫痫药物（ASM）在实际临床治疗中显示出高疗效和安全性。对该药物反应良好并坚持长期治疗的患者可实现良好的癫痫控制。此外，以相对较低剂量实现无癫痫发作的患者可选择相同剂量作为维持剂量。
本研究提供了PER单药治疗作为中国小儿癫痫一线ASM的疗效和安全性。共有124名患者参与。不同观察点的无癫痫发作率均超过70%，12个月观察点的保留率为71.58%。观察到的大多数不良反应为轻至中度。
