司替戊醇辅助治疗儿童Dravet综合征的研究进展

• 作 者: 陈文杰
• 机 构: 北京大学人民医院儿科 100044
• 出 处: 国际儿科学杂志
• 发表日期: 2019年7期
• 关键词: Dravet syndrome Stiripentol Anticonvulsant Mechanism Efficacy

Dravet综合征(Dravet syndrome,DS)是一种严重而少见的难治性癫痫类型,目前常用的抗癫痫药物治疗效果不佳且具有耐药性,给患儿及其家庭造成了沉重的心理压力和负担.已有多项研究显示,司替戊醇(stiripentol,STP)添加治疗DS安全有效,治疗机制主要是通过直接作用GABA受体增强GA-BA能神经传导,同时抑制多种肝细胞色素P450酶,升高部分抗癫痫药的血药浓度,从而发挥抗癫痫作用.该文对STP辅助治疗DS的机制和临床疗效的进展作一综述,以期为常规抗癫痫疗法效果不佳的DS患儿提供多一种治疗选择.“,”Dravet syndrome ( DS) , known as severe myoclonic epilepsy of infancy, is a devastating disorder characterized by intractable epilepsy and poor neurodevelopmental outcome. Seizures are refractory to conventional antiepileptic therapy, therefore resulting in heavy psychological pressure and burden. Stiripentol ( STP) is a novel antiepileptic drug, which has been proposed to achieve better seizure control in DS. It acts as a direct GABA receptor agonist by increasing the GABAergic transmission and by prolongating the opening peri-od of the receptor dependent chloride channels. In addition, STP inhibits several isoenzymes of the cytochrome P450 system in the liver involved in the metabolism of other antiepileptics, thus potentiating their effects. By summarizing the relevant researches at home and abroad, we describe an on-going work in the anticonvulsant mechanisms and efficacy of STP, to provide an alternative treatment of DS.