目的:评估在现实条件下用于治疗PERMIT研究中的特发性全身性癫痫(IGE)患者时的perampanel(PER)。
方法:跨国公司,回顾性,PERMIT汇总分析探讨了在17个国家的临床实践中治疗的局灶性和全身性癫痫患者中使用PER的情况.此亚组分析包括IGE的PERMIT参与者。保留时间点和有效性测量时间点分别为3、6和12个月(最后一次观察,定义为“上次访问”,也适用于有效性)。按癫痫发作类型评估有效性(总癫痫发作,全身性强直-阵挛性癫痫发作[GTCS],肌阵挛性癫痫发作,失神癫痫发作),并包括≥50%的应答率和癫痫发作自由率(定义为至少自上次访问以来没有癫痫发作)。在整个PER治疗过程中监测安全性/耐受性,并通过记录不良事件(AE)的发生率进行评估。包括精神病不良事件和导致治疗中断的不良事件。
结果:完整分析集包括544名IGE患者(51.9%的女性;平均年龄,33.3年;平均癫痫持续时间,18.1年)。在3、6和12个月时,92.4%,85.5%和77.3%的参与者继续接受PER治疗,分别(留存人口,n=497)。在最后一次访问中,响应者和癫痫发作自由率是,分别,74.2%和54.6%(总缉获量),81.2%和61.5%(GTCS),85.7%和66.0%(肌阵挛性癫痫发作),和90.5%和81.0%(癫痫发作缺失)(有效性人群,n=467)。不良事件发生在42.9%的患者中,包括易怒(9.6%),头晕/眩晕(9.2%)和嗜睡(6.3%)(耐受性人群,n=520)。12个月内因不良事件而停止治疗的比例为12.4%。
结论:PERMIT研究的这一亚组分析表明,在日常临床实践条件下给药,PER对IGE患者的有效性和良好的耐受性。这些发现与临床试验证据一致,支持PER用作治疗IGE的广谱抗癫痫药物。
This study was undertaken to evaluate perampanel (PER) when used under real-world conditions to treat people with idiopathic generalized epilepsy (IGE) included in the PERaMpanel pooled analysIs of effecTiveness and tolerability (PERMIT) study.
The multinational, retrospective, pooled analysis PERMIT explored the use of PER in people with focal and generalized epilepsy treated in clinical practice across 17 countries. This subgroup analysis included PERMIT participants with IGE. Time points for retention and effectiveness measurements were 3, 6, and 12 months (last observation carried forward, defined as \"last visit,\" was also applied to effectiveness). Effectiveness was evaluated by seizure type (total seizures, generalized tonic-clonic seizures [GTCS], myoclonic seizures, absence seizures) and included ≥50% responder rate and seizure freedom rate (defined as no seizures since at least the previous visit). Safety/tolerability was monitored throughout PER treatment and evaluated by documenting the incidence of adverse events (AEs), including psychiatric AEs and those leading to treatment discontinuation.
The Full Analysis Set included 544 people with IGE (51.9% women, mean age = 33.3 years, mean epilepsy duration = 18.1 years). At 3, 6, and 12 months, 92.4%, 85.5%, and 77.3% of participants were retained on PER treatment, respectively (Retention Population, n = 497). At the last visit, responder and seizure freedom rates were, respectively, 74.2% and 54.6% (total seizures), 81.2% and 61.5% (GTCS), 85.7% and 66.0% (myoclonic seizures), and 90.5% and 81.0% (absence seizures) (Effectiveness Population, n = 467). AEs occurred in 42.9% of patients and included irritability (9.6%), dizziness/vertigo (9.2%), and somnolence (6.3%) (Tolerability Population, n = 520). Treatment discontinuation due to AEs was 12.4% over 12 months.
This subgroup analysis of the PERMIT study demonstrated the effectiveness and good tolerability of PER in people with IGE when administered under everyday clinical practice conditions. These findings are in line with clinical trial evidence, supporting PER\'s use as broad-spectrum antiseizure medication for the treatment of IGE.