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[儿科领域的新型抗癫痫药物]

[New anti-epileptic drugs in Paediatrics].

作者信息

Málaga Ignacio, Sánchez-Carpintero Rocío, Roldán Susana, Ramos-Lizana Julio, García-Peñas Juan José

机构信息

Unidad de Neuropediatría, Hospital Universitario Central de Asturias, Oviedo, España.

Unidad de Neuropediatría, Clínica Universitaria de Navarra, Pamplona, España.

出版信息

An Pediatr (Engl Ed). 2019 Dec;91(6):415.e1-415.e10. doi: 10.1016/j.anpedi.2019.09.008. Epub 2019 Nov 8.

Abstract

It is estimated that about 70 million people all over the world suffer from epilepsy, half of which are children, in whom the prevalence is around 0.5 to 0.8%. Although there are several therapies, the treatment of epilepsy is based mainly on drugs, which, depending on the year of coming onto the market are classified as first, second, or third generation. In this article, a description is presented on the main characteristics of the latest generation of anti-epileptic drugs (lacosamide, eslicarbazepine acetate, brivaracetam, perampanel, retigabine, everolimus and cannabidiol). These, with the exception of retigabine (is not yet on the market), are considered safe and effective in the paediatric population. Everolimus and cannabidiol have very specific indications (tuberous sclerosis, Dravet syndrome, and Lennox Gastaut syndrome), while the rest are indicated in the management of seizures of focal origin in children from 4 years-old. These new molecules have been developed in order to provide a pharmaceutical profile and tolerance superior to the previously available drugs, and it is forecast that as their use increases, their true potential and profile will widen. Furthermore, for the first time in Paediatric Epileptology, the extrapolation of the efficacy data in adults have been used (together with specific safety and pharmacokinetic studies in the paediatric population), in order to speed up their approval for use in the child population.

摘要

据估计,全球约有7000万人患有癫痫,其中一半是儿童,儿童患病率约为0.5%至0.8%。尽管有多种治疗方法,但癫痫治疗主要基于药物,根据上市年份,这些药物可分为第一代、第二代或第三代。本文介绍了最新一代抗癫痫药物(拉科酰胺、醋酸艾司利卡西平、布瓦西坦、吡仑帕奈、瑞替加滨、依维莫司和大麻二酚)的主要特点。除瑞替加滨(尚未上市)外,这些药物在儿科人群中被认为是安全有效的。依维莫司和大麻二酚有非常特定的适应症(结节性硬化症、德雷维特综合征和伦诺克斯-加斯托综合征),而其余药物适用于4岁及以上儿童局灶性发作的治疗。开发这些新分子是为了提供比现有药物更优的药学特性和耐受性,预计随着其使用增加,它们的真正潜力和特性将得到更广泛的展现。此外,在儿科癫痫学领域,首次采用了成人疗效数据的外推法(结合儿科人群的特定安全性和药代动力学研究),以加快这些药物在儿童人群中的获批使用。

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