布瓦西坦辅助治疗全面性癫痫患儿的长期耐受性和疗效：一项开放标签随访试验的亚组分析

IF 2.3 3区医学 Q2 BEHAVIORAL SCIENCES

Epilepsy & Behavior Pub Date : 2025-07-29 DOI:10.1016/j.yebeh.2025.110569

Dimitrios Bourikas , Sami Elmoufti , Jan-Peer Elshoff , Allison Little , Kristy Pucylowski , Brian Moseley , Lieven Lagae

{"title":"布瓦西坦辅助治疗全面性癫痫患儿的长期耐受性和疗效：一项开放标签随访试验的亚组分析","authors":"Dimitrios Bourikas , Sami Elmoufti , Jan-Peer Elshoff , Allison Little , Kristy Pucylowski , Brian Moseley , Lieven Lagae","doi":"10.1016/j.yebeh.2025.110569","DOIUrl":null,"url":null,"abstract":"<div><h3>Objectives</h3><div>To assess long-term safety, tolerability, and efficacy of adjunctive brivaracetam in children with generalized-onset seizures.</div></div><div><h3>Methods</h3><div>Subgroup analysis of a Phase 3, open-label trial (N01266/NCT01364597; ≤5 mg/kg/day brivaracetam [maximum 200 mg/day]) in patients aged ≥ 1 month to < 17 years with generalized-onset seizures at core trial entry. Seizure-related outcomes were assessed for patients aged < 2/≥2 years using daily record-card data. Kaplan-Meier–estimated retention and change in Achenbach Child Behavior Checklist (CBCL) scores were assessed post hoc.</div></div><div><h3>Results</h3><div>Of 257 patients receiving brivaracetam, 68 had generalized-onset seizures (50.0 % male; mean age: 6.65 years; median modal brivaracetam dose 3.6 mg/kg/day). Twenty-eight (41.2 %) patients completed the trial; most common reasons for discontinuation (≥10 % patients) were adverse event (22.1 %), lack of efficacy (17.6 %), and withdrawn consent (11.8 %). Kaplan-Meier–estimated retention at 1, 3, and 5 years was 61.8 %, 47.7 %, and 43.3 %, respectively. Sixty-one (89.7 %) patients had treatment-emergent adverse events (serious 25 [36.8 %]). In patients aged < 2/≥2 years (n = 12/n = 33), median percent reduction in 28-day-adjusted total seizure frequency from baseline to end of evaluation was 66.7 %/56.9 %; 50.0 %/60.6 % had ≥ 50 % reduction in all seizures. Mean changes from baseline to last evaluation in Achenbach CBCL 1.5–5 raw syndrome scores (n = 18) were all around 0 (minimal/small amplitude). Mean changes in CBCL 6–18 raw syndrome scores (n = 20) showed small numerical improvements for most syndromes. At last evaluation, most patients (range: 61.1 %–100 %) remained in baseline T-score categories.</div></div><div><h3>Conclusions</h3><div>Long-term adjunctive brivaracetam was well tolerated and efficacious in children with generalized-onset seizures. Behavior/emotional function were generally stable or slightly improved.</div></div>","PeriodicalId":11847,"journal":{"name":"Epilepsy & Behavior","volume":"171 ","pages":"Article 110569"},"PeriodicalIF":2.3000,"publicationDate":"2025-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Long-term tolerability and efficacy of adjunctive brivaracetam in pediatric patients with generalized-onset seizures: Subgroup analysis of an open-label, follow-up trial\",\"authors\":\"Dimitrios Bourikas , Sami Elmoufti , Jan-Peer Elshoff , Allison Little , Kristy Pucylowski , Brian Moseley , Lieven Lagae\",\"doi\":\"10.1016/j.yebeh.2025.110569\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<div><h3>Objectives</h3><div>To assess long-term safety, tolerability, and efficacy of adjunctive brivaracetam in children with generalized-onset seizures.</div></div><div><h3>Methods</h3><div>Subgroup analysis of a Phase 3, open-label trial (N01266/NCT01364597; ≤5 mg/kg/day brivaracetam [maximum 200 mg/day]) in patients aged ≥ 1 month to < 17 years with generalized-onset seizures at core trial entry. Seizure-related outcomes were assessed for patients aged < 2/≥2 years using daily record-card data. Kaplan-Meier–estimated retention and change in Achenbach Child Behavior Checklist (CBCL) scores were assessed post hoc.</div></div><div><h3>Results</h3><div>Of 257 patients receiving brivaracetam, 68 had generalized-onset seizures (50.0 % male; mean age: 6.65 years; median modal brivaracetam dose 3.6 mg/kg/day). Twenty-eight (41.2 %) patients completed the trial; most common reasons for discontinuation (≥10 % patients) were adverse event (22.1 %), lack of efficacy (17.6 %), and withdrawn consent (11.8 %). Kaplan-Meier–estimated retention at 1, 3, and 5 years was 61.8 %, 47.7 %, and 43.3 %, respectively. Sixty-one (89.7 %) patients had treatment-emergent adverse events (serious 25 [36.8 %]). In patients aged < 2/≥2 years (n = 12/n = 33), median percent reduction in 28-day-adjusted total seizure frequency from baseline to end of evaluation was 66.7 %/56.9 %; 50.0 %/60.6 % had ≥ 50 % reduction in all seizures. Mean changes from baseline to last evaluation in Achenbach CBCL 1.5–5 raw syndrome scores (n = 18) were all around 0 (minimal/small amplitude). Mean changes in CBCL 6–18 raw syndrome scores (n = 20) showed small numerical improvements for most syndromes. At last evaluation, most patients (range: 61.1 %–100 %) remained in baseline T-score categories.</div></div><div><h3>Conclusions</h3><div>Long-term adjunctive brivaracetam was well tolerated and efficacious in children with generalized-onset seizures. Behavior/emotional function were generally stable or slightly improved.</div></div>\",\"PeriodicalId\":11847,\"journal\":{\"name\":\"Epilepsy & Behavior\",\"volume\":\"171 \",\"pages\":\"Article 110569\"},\"PeriodicalIF\":2.3000,\"publicationDate\":\"2025-07-29\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Epilepsy & Behavior\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://www.sciencedirect.com/science/article/pii/S1525505025003099\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q2\",\"JCRName\":\"BEHAVIORAL SCIENCES\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Epilepsy & Behavior","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S1525505025003099","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q2","JCRName":"BEHAVIORAL SCIENCES","Score":null,"Total":0}

引用次数: 0

摘要

目的评价布伐西坦辅助治疗儿童全面性癫痫发作的长期安全性、耐受性和疗效。方法：3期开放标签试验(N01266/NCT01364597；≤5mg /kg/天布伐西坦[最大200mg /天])，年龄≥1个月~ 1岁的患者；17岁，在核心试验开始时出现全身性癫痫发作。对老年患者的癫痫相关结局进行评估；2/≥2年使用日记录卡数据。事后评估阿肯巴赫儿童行为检查表（CBCL）得分的kaplan - meier估计保留和变化。结果257例接受布瓦西坦治疗的患者中，68例发生全局性癫痫发作(50.0%男性；平均年龄：6.65岁；布瓦西坦中位剂量3.6 mg/kg/天)。28例（41.2%）患者完成了试验；最常见的停药原因（≥10%的患者）是不良事件（22.1%）、缺乏疗效（17.6%）和撤回同意（11.8%）。kaplan - meier估计，1年、3年和5年的保留率分别为61.8%、47.7%和43.3%。61例（89.7%）患者出现治疗后出现的不良事件（严重25例（36.8%））。老年人<；2/≥2年（n = 12/n = 33），从基线到评估结束，28天调整后总癫痫发作频率的中位数百分比减少为66.7% / 56.9%；50.0% / 60.6%患者所有癫痫发作减少≥50%。从基线到最后一次评估时，Achenbach CBCL 1.5-5原始综合征评分（n = 18）的平均变化都在0左右（最小/小振幅）。CBCL 6-18原始证候评分（n = 20）的平均变化显示大多数证候的数值改善很小。最后一次评估，大多数患者（范围：61.1% - 100%）保持在基线t评分类别。结论布伐西坦长期辅助治疗全面性癫痫患儿耐受性好，疗效显著。行为/情绪功能总体稳定或略有改善。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

Long-term tolerability and efficacy of adjunctive brivaracetam in pediatric patients with generalized-onset seizures: Subgroup analysis of an open-label, follow-up trial

查看原文本刊更多论文

Long-term tolerability and efficacy of adjunctive brivaracetam in pediatric patients with generalized-onset seizures: Subgroup analysis of an open-label, follow-up trial

Objectives

To assess long-term safety, tolerability, and efficacy of adjunctive brivaracetam in children with generalized-onset seizures.

Methods

Subgroup analysis of a Phase 3, open-label trial (N01266/NCT01364597; ≤5 mg/kg/day brivaracetam [maximum 200 mg/day]) in patients aged ≥ 1 month to < 17 years with generalized-onset seizures at core trial entry. Seizure-related outcomes were assessed for patients aged < 2/≥2 years using daily record-card data. Kaplan-Meier–estimated retention and change in Achenbach Child Behavior Checklist (CBCL) scores were assessed post hoc.

Results

Of 257 patients receiving brivaracetam, 68 had generalized-onset seizures (50.0 % male; mean age: 6.65 years; median modal brivaracetam dose 3.6 mg/kg/day). Twenty-eight (41.2 %) patients completed the trial; most common reasons for discontinuation (≥10 % patients) were adverse event (22.1 %), lack of efficacy (17.6 %), and withdrawn consent (11.8 %). Kaplan-Meier–estimated retention at 1, 3, and 5 years was 61.8 %, 47.7 %, and 43.3 %, respectively. Sixty-one (89.7 %) patients had treatment-emergent adverse events (serious 25 [36.8 %]). In patients aged < 2/≥2 years (n = 12/n = 33), median percent reduction in 28-day-adjusted total seizure frequency from baseline to end of evaluation was 66.7 %/56.9 %; 50.0 %/60.6 % had ≥ 50 % reduction in all seizures. Mean changes from baseline to last evaluation in Achenbach CBCL 1.5–5 raw syndrome scores (n = 18) were all around 0 (minimal/small amplitude). Mean changes in CBCL 6–18 raw syndrome scores (n = 20) showed small numerical improvements for most syndromes. At last evaluation, most patients (range: 61.1 %–100 %) remained in baseline T-score categories.

Conclusions

Long-term adjunctive brivaracetam was well tolerated and efficacious in children with generalized-onset seizures. Behavior/emotional function were generally stable or slightly improved.

求助全文

通过发布文献求助，成功后即可免费获取论文全文。去求助

来源期刊

Epilepsy & Behavior 医学-行为科学

CiteScore

5.40

自引率

15.40%

发文量

385

审稿时长

43 days

期刊介绍： Epilepsy & Behavior is the fastest-growing international journal uniquely devoted to the rapid dissemination of the most current information available on the behavioral aspects of seizures and epilepsy. Epilepsy & Behavior presents original peer-reviewed articles based on laboratory and clinical research. Topics are drawn from a variety of fields, including clinical neurology, neurosurgery, neuropsychiatry, neuropsychology, neurophysiology, neuropharmacology, and neuroimaging. From September 2012 Epilepsy & Behavior stopped accepting Case Reports for publication in the journal. From this date authors who submit to Epilepsy & Behavior will be offered a transfer or asked to resubmit their Case Reports to its new sister journal, Epilepsy & Behavior Case Reports.