Real-world use of cenobamate in pediatric focal epilepsies and developmental epileptic encephalopathies: A multicenter retrospective series.

IF 6.6 1区医学 Q1 CLINICAL NEUROLOGY

Epilepsia Pub Date : 2025-07-30 DOI:10.1111/epi.18586

Víctor Soto-Insuga, Adrián Valls Carbó, Anna Gretel Pinzón-Acevedo, Elena González-Alguacil, Juan José García Peñas, Andrea Sariego Jamardo, Gemma Aznar-Laín, Salvador Ibáñez-Micó, Helena Alarcón Martínez, Raquel Buenache, Saray Rekarte, Andrea Parejo Olivera, Ezequiel Tuero-Montiel, Jana DomínguezCarral, María López, Ainhoa García Ribes, María Jesús Martínez González, Eva Arias, Adrián García Ron, Susana Boronat, Eulalia Turón Viñas, Dolors Casellas, Virginia Navarro, David Conejo, Elena Miravet, Irene Sánchez-Miranda Román, Antonio Gil Nagel-Rein, María Muñoz Cabeza, Patricia Smeyers, Ángel Aledo-Serrano

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引用次数: 0

Abstract

Objective: Cenobamate (CNB) is an anti-seizure medication approved for focal-onset seizures in adults, with growing evidence supporting its use in pediatric drug-resistant epilepsy (DRE). This study evaluates the efficacy, retention, and safety of CNB in children and adolescents, including those with developmental and epileptic encephalopathies (DEEs).

Methods: We conducted a retrospective, multicenter study of 169 pediatric patients (0-18 years) with DRE treated with CNB across centers in Spain. Seizure response (≥50% reduction) and seizure freedom (no seizures in the previous month) were assessed at 3, 6, and 12 months.

Results: At 12 months, CNB showed a retention rate of 89.2%, with 83.9% of patients achieving seizure response and 19.6% reaching seizure freedom. Response rates were 73.4% at 3 months and 77.1% at 6 months, with seizure freedom increasing from 11.8% at 3 months to 21.1% at 6 months. Outcomes were comparable between DEEs and focal epilepsies, indicating broad-spectrum efficacy. Seizure worsening occurred in 4.7% of patients, all with DEEs. After adjusting for seizure type, those patients with focal seizures had higher odds of achieving seizure response (odds ratio [OR] 95% confidence interval [95% CI] 5.46, 1.27-23.52; p = 0.02) and seizure freedom (OR 95% CI 5.32, 1.57-17.97; p < 0.01). Median CNB doses (mg/kg/day) were 1.78 (range 0.37-12.5), 2.5 (range 0.67-12.5), and 3.19 (range 0.96-9.09) at 3, 6, and 12 months, respectively. Adverse events occurred in 44.9% of patients, most commonly somnolence (42.3%) and dizziness (22.4%), which improved with slower titration.

Significance: CNB appears effective and well-tolerated in pediatric DRE, including DEE, with high retention and sustained efficacy over time. Adverse events were generally manageable with dose adjustments, and slow, weight-based titration improved tolerability. Prospective studies are warranted to confirm these findings.

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cenobamate在小儿局灶性癫痫和发育性癫痫性脑病中的实际应用：一项多中心回顾性研究。

目的：Cenobamate （CNB）是一种被批准用于成人局灶性癫痫发作的抗癫痫药物，越来越多的证据支持其用于儿科耐药癫痫（DRE）。本研究评估了CNB在儿童和青少年（包括发育性和癫痫性脑病患者）中的有效性、保留性和安全性。方法：我们对西班牙各中心接受CNB治疗的169例DRE患儿（0-18岁）进行了一项回顾性多中心研究。在第3、6和12个月时评估发作反应（≥50%减少）和发作自由（前一个月无发作）。结果：12个月时，CNB保留率为89.2%，83.9%的患者癫痫发作缓解，19.6%的患者癫痫发作自由。缓解率在3个月时为73.4%，6个月时为77.1%，癫痫发作自由度从3个月时的11.8%增加到6个月时的21.1%。结果在dei和局灶性癫痫之间具有可比性，表明广谱疗效。4.7%的患者癫痫发作加重，所有患者均为dei。调整癫痫发作类型后，局灶性癫痫发作患者获得癫痫发作反应的几率更高(优势比[OR] 95%可信区间[95% CI] 5.46, 1.27-23.52；p = 0.02)和癫痫发作自由度(OR 95% CI 5.32, 1.57-17.97；p意义：CNB对儿童DRE（包括DEE）有效且耐受性良好，具有高保留率和持续疗效。不良事件通常可以通过剂量调整来控制，缓慢的、基于体重的滴定可以改善耐受性。有必要进行前瞻性研究来证实这些发现。

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

Epilepsia 医学-临床神经学

CiteScore

10.90

自引率

10.70%

发文量

319

审稿时长

2-4 weeks

期刊介绍： Epilepsia is the leading, authoritative source for innovative clinical and basic science research for all aspects of epilepsy and seizures. In addition, Epilepsia publishes critical reviews, opinion pieces, and guidelines that foster understanding and aim to improve the diagnosis and treatment of people with seizures and epilepsy.