Effectiveness and safety of perampanel for pediatric patients with epilepsy: A real-world study from China.

IF 1.9 4区 医学 Q2 PEDIATRICS
Pediatric Investigation Pub Date : 2025-03-11 eCollection Date: 2025-06-01 DOI:10.1002/ped4.12463
Xiaohui Wang, Taoyun Ji, Maomao Liu, Xiaofei Wang, Zhixian Yang, Sanmei Wang, Liping Zou, Jiong Qin, Xiaotun Ren, Liankun Ren, Liri Jin, Jie Shi, Dantao Peng, Kui Chen, Jindong Dai, Nan Zhang, Jun Wang, Tianyu Song, Fang Fang, Yuehua Zhang, Qun Wang
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引用次数: 0

Abstract

Importance: Perampanel (PER) is used less extensively in children than in adults. Currently, there is a lack of data from PER clinical studies with large sample sizes in Chinese children and adolescents with epilepsy, especially those with refractory epilepsy.

Objective: To evaluate the effectiveness, retention, and safety of PER in the treatment of children and adolescents with epilepsy in China.

Methods: This was a multicenter, prospective, observational study. Children and adolescents with epilepsy who received PER as adjunctive therapy were included. The primary effectiveness endpoint was the proportion of patients achieving a ≥50% reduction in seizure frequency after 6 months of treatment compared to baseline. The secondary effectiveness endpoints included retention and seizure-free rates. The safety outcome was the incidence of treatment-emergent adverse events (TEAEs).

Results: A total of 240 patients with epilepsy were enrolled in the study. Prior to initiating PER treatment, approximately 87.9% of them took two or more antiseizure medications. After a 6-month treatment regimen with PER, 70.4% of the patients experienced a reduced seizure frequency of at least 50%, and 22.1% achieved complete seizure freedom. The retention rate was 90.2%. TEAEs were reported by 89 patients, leading to the discontinuation of PER in seven cases. No severe TEAEs were observed in this study.

Interpretation: Under routine clinical conditions, PER demonstrated good effectiveness and retention in Chinese children with epilepsy, particularly in those with refractory epilepsy.

perampanel治疗小儿癫痫患者的有效性和安全性:一项来自中国的真实世界研究。
重要性:Perampanel (PER)在儿童中的应用比在成人中的应用要少。目前,中国癫痫儿童和青少年,特别是难治性癫痫儿童和青少年,缺乏大样本量的PER临床研究数据。目的:评价PER治疗儿童和青少年癫痫的有效性、保持性和安全性。方法:这是一项多中心、前瞻性、观察性研究。儿童和青少年癫痫患者接受PER作为辅助治疗。主要有效性终点是治疗6个月后癫痫发作频率较基线降低≥50%的患者比例。次要有效性终点包括滞留率和无癫痫发作率。安全性指标是治疗中出现的不良事件(teae)的发生率。结果:共纳入240例癫痫患者。在开始PER治疗之前,大约87.9%的患者服用了两种或两种以上的抗癫痫药物。经过6个月的PER治疗方案,70.4%的患者癫痫发作频率降低了至少50%,22.1%的患者癫痫发作完全自由。保留率为90.2%。89例患者报告了teae,其中7例导致PER停止。本研究未见严重teae。解释:在常规临床条件下,PER在中国癫痫患儿,特别是难治性癫痫患儿中表现出良好的有效性和保持性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Pediatric Investigation
Pediatric Investigation Medicine-Pediatrics, Perinatology and Child Health
CiteScore
3.30
自引率
0.00%
发文量
176
审稿时长
12 weeks
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