Effectiveness and tolerability of perampanel as first add-on treatment in pediatric patients with focal and generalized epilepsy

IF 2.3 4区医学 Q3 CLINICAL NEUROLOGY

Epilepsy Research Pub Date : 2025-12-01 Epub Date: 2025-10-24 DOI:10.1016/j.eplepsyres.2025.107690

Bárbara Blanco Martínez, Elena Arce Portillo, María Muñoz Cabeza, Mercedes López Lobato, Beatriz Muñoz Cabello, Marta Correa Vela, Laura González Hernández, Carmen Cortés Jiménez, Olga Alonso Luengo

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引用次数: 0

Abstract

Purpose

To compare the effectiveness and safety of perampanel as first add-on vs. late add-on therapy in children with focal or generalized epilepsy.

Methods

Retrospective, single-center study conducted between March 2022 and April 2023, including children ≥ 4 years with focal an generalized epilepsy who started treatment with perampanel in November 2020 or later. Patients were classified into two groups: perampanel as first and as late add-on treatment. Effectiveness and safety data were collected at 3, 6, and 12 months after starting treatment. Primary outcomes were responder rates, seizure-freedom rates, and worsening.

Results

57 patients (45.6 % women) with a mean (SD) age of 8.8 (±3.5) years were included; 27 in the first and 30 in the late add-on group. Effectiveness was consistently higher throughout visits in patients receiving perampanel as first vs. late add-on treatment, with higher response rates (12 months, 95.0 % vs. 55.6 %, p = 0.003), higher seizure-freedom rates (12 months, 65 % vs. 37.0 %, p = 0.058), and fewer patients with worsening (12 months, 0 % vs. 12.8 %, p = 0.031). Retention rates remained high in both groups throughout visits. Sixteen patients discontinued treatment. Reasons were lack of effectiveness, which was more frequent in the late vs. first add-on group, with few patients discontinuing due to adverse events.

Conclusion

The improved effectiveness outcomes of perampanel used as first vs. late add-on therapy in a pediatric population and the favorable safety outcomes support its use as first add-on therapy in pediatric patients with focal and generalized epilepsy.

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perampanel作为小儿局灶性和全身性癫痫患者第一附加治疗的有效性和耐受性。

目的：比较perampanel作为局灶性或全面性癫痫患儿首次附加治疗与晚期附加治疗的有效性和安全性。方法：回顾性、单中心研究于2022年3月至2023年4月进行，纳入≥ 4岁局灶性和全面性癫痫患儿，这些患儿在2020年11月或之后开始使用perampanel治疗。患者分为两组：perampanel作为第一组和晚期附加治疗。在开始治疗后3、6和12个月收集有效性和安全性数据。主要结局是应答率、癫痫解除率和病情恶化。结果：纳入57例患者（45.6% %为女性），平均（SD）年龄为8.8（±3.5）岁；第一个组27人，最后一个组30人。有效性一直在访问病人接受高perampanel作为第一和附加治疗后期,高响应率(12个月,95.0 % 55.6 vs %,p = 0.003),更高的发作率(12个月,65 %与37.0 %,p = 0.058),和更少的患者恶化(12个月,0 % 12.8 vs % p = 0.031)。在整个访问过程中，两组的保留率都很高。16名患者停止治疗。原因是缺乏有效性，这在晚期加药组比首次加药组更常见，很少有患者因不良事件而停药。结论：perampanel作为儿科人群首次与晚期附加治疗的疗效改善以及良好的安全性结果支持其作为局灶性和全面性癫痫儿童患者的首次附加治疗。

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

Epilepsy Research 医学-临床神经学

CiteScore

0.10

自引率

4.50%

发文量

143

审稿时长

62 days

期刊介绍： Epilepsy Research provides for publication of high quality articles in both basic and clinical epilepsy research, with a special emphasis on translational research that ultimately relates to epilepsy as a human condition. The journal is intended to provide a forum for reporting the best and most rigorous epilepsy research from all disciplines ranging from biophysics and molecular biology to epidemiological and psychosocial research. As such the journal will publish original papers relevant to epilepsy from any scientific discipline and also studies of a multidisciplinary nature. Clinical and experimental research papers adopting fresh conceptual approaches to the study of epilepsy and its treatment are encouraged. The overriding criteria for publication are novelty, significant clinical or experimental relevance, and interest to a multidisciplinary audience in the broad arena of epilepsy. Review articles focused on any topic of epilepsy research will also be considered, but only if they present an exceptionally clear synthesis of current knowledge and future directions of a research area, based on a critical assessment of the available data or on hypotheses that are likely to stimulate more critical thinking and further advances in an area of epilepsy research.