Makridis Konstantin L, Bast Thomas, Prager Christine, Kovacevic-Preradovic Tatjana, Bittigau Petra, Mayer Thomas, Breuer Eva, Kaindl Angela M
Department of Pediatric Neurology, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Center for Chronically Sick Children, Charité-Universitätsmedizin Berlin, Berlin, Germany.
Front Neurol. 2022 Jul 12;13:950171. doi: 10.3389/fneur.2022.950171. eCollection 2022.
In one third of all patients with epilepsy, seizure freedom is not achieved through anti-seizure medication (ASM). These patients have an increased risk of earlier death, poorer cognitive development, and reduced quality of life. Cenobamate (CNB) has recently been approved as a promising novel ASM drug for the treatment of adults with focal-onset epilepsy. However, there is little experience for its application in pediatric patients.
In a multicenter study we evaluated retrospectively the outcome of 16 pediatric patients treated "off label" with CNB.
In 16 patients with a mean age of 15.38 years, CNB was started at an age of 15.05 years due to DRE. Prior to initiation of therapy, an average of 10.56 (range 3-20) ASM were prescribed. At initiation, patients were taking 2.63 (range 1-4) ASM. CNB was increased by 0.47 ± 0.27mg/kg/d every 2 weeks with a mean maximum dosage of 3.1 mg/kg/d (range 0.89-7) and total daily dose of 182.81 mg (range 50-400 mg). Seizure freedom was achieved in 31.3% and a significant seizure reduction of >50% in 37.5%. Adverse events occurred in 10 patients with fatigue/somnolence as the most common. CNB is taken with high adherence in all but three patients with a median follow-up of 168.5 days.
Cenobamate is an effective ASM for pediatric patients suffering from drug-resistant epilepsy. In addition to excellent seizure reduction or freedom, it is well-tolerated. Cenobamate should be considered as a novel treatment for DRE in pediatric patients.
在所有癫痫患者中,三分之一的患者无法通过抗癫痫药物(ASM)实现无癫痫发作。这些患者过早死亡、认知发育较差和生活质量降低的风险增加。司替戊醇(CNB)最近已被批准作为一种有前景的新型ASM药物,用于治疗成人局灶性癫痫发作。然而,其在儿科患者中的应用经验很少。
在一项多中心研究中,我们回顾性评估了16例接受CNB“超说明书用药”治疗的儿科患者的治疗结果。
16例患者的平均年龄为15.38岁,因药物难治性癫痫(DRE)在15.05岁开始使用CNB。在开始治疗前,平均开具了10.56种(范围3 - 20种)ASM。开始治疗时,患者正在服用2.63种(范围1 - 4种)ASM。每2周将CNB剂量增加0.47±0.27mg/kg/d,平均最大剂量为3.1mg/kg/d(范围0.89 - 7),每日总剂量为182.81mg(范围50 - 400mg)。31.3%的患者实现了无癫痫发作,37.5%的患者癫痫发作显著减少>50%。10例患者出现不良事件,最常见的是疲劳/嗜睡。除3例患者外,所有患者对CNB的依从性都很高,中位随访时间为168.5天。
司替戊醇是治疗患有耐药性癫痫的儿科患者的一种有效ASM。除了能显著减少癫痫发作或实现无癫痫发作外,它的耐受性良好。司替戊醇应被视为儿科患者DRE的一种新型治疗方法。