目的:在1个月至18岁的耐药性癫痫(DRE)等待癫痫手术的患者中,评估perampanel(PER)在现实世界中的有效性和耐受性。
方法:在这项多中心研究中,选择2020年1月至2021年6月接受PER治疗的1个月至18岁DRE患者.研究结果是PER治疗的有效性,报告为癫痫发作频率和癫痫发作自由率的降低。在开始PER后30、60、90、120、150和180天评估有效性。根据患者家属和医生的观察,耐受性情况报告为不良事件。
结果:85例接受PER治疗的患者被纳入研究。辅助PER的平均初始剂量和平均最大剂量为2mg/天和5.8mg/天,分别。平均发作频率(率/周)为41.3、25.4、18.9、14.3、11.2、11.1和8.9次发作,30、60、90、120、150和180天,分别;与基线相比,所有时间点的平均癫痫发作频率均显著降低(p<0.001)。180天,64.9%(37/57)的患者癫痫发作减少≥75%,36.8%(21/57)的患者癫痫发作自由。困倦,共济失调,和行为变化是观察到的常见不良事件,这些改善后的剂量减少PER。由于副作用或不耐受,不需要停止PER。
结论:在现实世界中,在患有DRE的儿科和青少年患者中,PER在癫痫发作控制方面具有良好的耐受性和有效性。
OBJECTIVE: To evaluate the effectiveness and tolerability of perampanel (PER) in real-world settings in patients between 1 month and 18 years of age with drug resistant epilepsy (DRE) waiting for epilepsy surgery.
METHODS: In this multicenter
study, patients between 1 month and 18 years of age with DRE treated with PER between January 2020 and June 2021 were selected. The
study outcome was effectiveness of PER treatment reported as reduction in seizure frequency and seizure freedom rate. Effectiveness was assessed at 30, 60, 90, 120, 150 and 180 days after initiation of PER. Tolerability profiles were reported as adverse events according to the observations of the patients\' family members and physician.
RESULTS: Eighty-five patients treated with PER were included in the
study. The mean initial dose and mean maximum dose of adjunctive PER was 2 mg/day and 5.8 mg/day, respectively. The mean seizure frequency (rate/week) was 41.3, 25.4, 18.9, 14.3, 11.2, 11.1 and 8.9 seizures at baseline, 30, 60, 90, 120, 150 and 180 days, respectively; the reduction in the mean seizure frequency at all timepoints was significant compared at the baseline (p<0.001). At 180 days, ≥75% seizure reduction was seen in 64.9% (37/57) of the patients and seizure freedom was achieved in 36.8% (21/57). Drowsiness, ataxia, and behavioral changes were the common adverse events observed, and these improved after the dose of PER was reduced. No discontinuation of PER was required due to side effects or intolerance.
CONCLUSIONS: In real-world settings, PER is well tolerated and effective in seizure control in pediatric and adolescent patients with DRE.