目的:ESPRITE(研究508;NCT03836924)评估了真实世界的安全性,耐受性,12岁以上局灶性发作性癫痫(FOS)患者的辅助治疗和疗效,伴有或不伴有双侧强直阵挛性癫痫发作(FBTCS),在印度。
方法:ESPRITE是一种前瞻性,多中心,单臂,观察,具有6个月治疗期的IV期研究。患者年龄≥12岁,并已被处方为FOS的辅助治疗,有或没有FBTCS。评估包括治疗引起的不良事件(TEAE;主要终点)的发生率,从基线开始每28天癫痫发作频率减少的中位数百分比,50%的反应率,和癫痫-自由率。
结果:总体而言,纳入200名患者(安全性分析集中的199名患者和完成主要疗效分析中的所有访视的174名患者)。18.1%(n=36/199)的患者报告了TEAE(严重程度均为轻度或中度)(最常见的是头晕[3.0%]和易怒[2.0%])。在2.0%的患者中报告了导致停药的TEAE;没有发生死亡或严重的TEAE。6个月时,癫痫发作频率减少的中位数百分比为100.0%,50%的应答率为83.3%,癫痫发作自由率为49.4%。
结论:在年龄≥12岁的FOS患者中,辅助perampanel(平均每日剂量为4mg/天)具有良好的耐受性和有效性,有或没有FBTCS,来自印度。
OBJECTIVE: ESPRITE (
Study 508; NCT03836924) evaluated the real-world safety, tolerability, and efficacy of adjunctive perampanel in patients aged ≥12 years with focal-onset seizures (FOS), with or without focal to bilateral tonic-clonic seizures (FBTCS), in India.
METHODS: ESPRITE was a prospective, multicenter, single-arm, observational, Phase IV
study with a 6-month Treatment Period. Patients were aged ≥12 years and had been prescribed perampanel for adjunctive treatment of FOS, with or without FBTCS. Assessments included incidence of treatment-emergent adverse events (TEAEs; primary endpoint), median percent reduction in seizure frequency per 28 days from baseline, 50% responder rates, and seizure-freedom rates.
RESULTS: Overall, 200 patients were enrolled (199 patients in the Safety Analysis Set and 174 patients who completed all visits in the main efficacy analyses). TEAEs (all mild or moderate in severity) were reported in 18.1% (n = 36/199) of patients (the most common were dizziness [3.0%] and irritability [2.0%]). TEAEs leading to discontinuation of perampanel were reported in 2.0% of patients; no deaths or serious TEAEs occurred. At 6 months, median percent reduction in seizure frequency was 100.0%, 50% responder rate was 83.3%, and seizure-freedom rate was 49.4%.
CONCLUSIONS: Adjunctive perampanel (at a mean daily dose of 4 mg/day) was shown to be well tolerated and effective in patients aged ≥12 years with FOS, with or without FBTCS, from India.
CONCLUSIONS: Many patients do not receive adequate treatment for epilepsy and need effective seizure control medications. In this 6-month clinical
study, 199 patients from India, aged 12 years or older, added perampanel to the anti-seizure medications they were already taking. At 6 months, 49% of patients experienced no seizures since starting perampanel and seizure frequency was reduced by half in 83% of patients. Side effects occurred in 18% of patients (most commonly dizziness and irritability) and caused 2% to stop perampanel; no deaths were reported. Perampanel was an effective and generally safe added medication for patients with epilepsy from India.