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
{"title":"Effectiveness and safety of perampanel for pediatric patients with epilepsy: A real-world study from China.","authors":"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","doi":"10.1002/ped4.12463","DOIUrl":null,"url":null,"abstract":"<p><strong>Importance: </strong>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.</p><p><strong>Objective: </strong>To evaluate the effectiveness, retention, and safety of PER in the treatment of children and adolescents with epilepsy in China.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>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.</p><p><strong>Interpretation: </strong>Under routine clinical conditions, PER demonstrated good effectiveness and retention in Chinese children with epilepsy, particularly in those with refractory epilepsy.</p>","PeriodicalId":19992,"journal":{"name":"Pediatric Investigation","volume":"9 2","pages":"172-180"},"PeriodicalIF":1.9000,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12175630/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Investigation","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1002/ped4.12463","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/6/1 0:00:00","PubModel":"eCollection","JCR":"Q2","JCRName":"PEDIATRICS","Score":null,"Total":0}
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.