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

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收录情况: ◇ 卓越:梯队期刊 ◇ ESCI ◇ 中华系列

机构: [1]Capital Med Univ, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, Dept Neurol, Beijing, Peoples R China [2]Peking Univ First Hosp, Dept Pediat, Beijing, Peoples R China [3]Capital Med Univ, Beijing Tiantan Hosp, Dept Neurol, Beijing, Peoples R China [4]Peoples Liberat Army Gen Hosp, Med Ctr 7, Dept Neurol Dev, Beijing, Peoples R China [5]Chinese Peoples Liberat Army Gen Hosp, Med Ctr 1, Dept Pediat, Beijing, Peoples R China [6]Peking Univ Peoples Hosp, Dept Pediat, Beijing, Peoples R China [7]Capital Med Univ, Xuanwu Hosp, Dept Neurol, Beijing, Peoples R China [8]Peking Union Med Coll Hosp, Dept Neurol, Beijing, Beijing, Peoples R China [9]Tsinghua Univ, Dept Epilepsy Ctr, Yuquan Hosp, Beijing, Peoples R China [10]China Japan Friendship Hosp, Dept Neurol, Beijing, Peoples R China [11]Capital Med Univ, Beijing Friendship Hosp, Dept Neurol, Beijing, Peoples R China [12]Beijing Haidian Hosp, Dept Funct Neurol, Beijing, Peoples R China [13]Capital Med Univ, Beijing Luhe Hosp, Dept Neurol, Beijing, Peoples R China [14]Childrens Hosp, Capital Inst Pediat, Beijing, Peoples R China
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关键词: Children and adolescents Efficacy Epilepsy Perampanel (PER) Safety

摘要:
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.

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大类 | 4 区 医学
小类 | 4 区 儿科
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大类 | 4 区 医学
小类 | 4 区 儿科
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出版当年[2023]版:
Q2 PEDIATRICS
最新[2023]版:
Q2 PEDIATRICS

影响因子: 最新[2023版] 最新五年平均 出版当年[2023版] 出版当年五年平均 出版前一年[2022版]

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第一作者机构: [1]Capital Med Univ, Beijing Childrens Hosp, Natl Ctr Childrens Hlth, Dept Neurol, Beijing, Peoples R China
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