司替戊醇用于儿童难治性癫痫的安全性与有效性研究进展  

Research progress on the safety and efficacy of stiripentol for treatment of refractory epilepsy in children

作　　者：刘漫漫 王纪文[2] LIU Manman;WANG Jiwen(Department of Pharmacy,Shanghai Children’s Medical Center,School of Medicine,Shanghai Jiao Tong University,Shanghai 200127,China;Department of Neurology,Shanghai Children’s Medical Center,School of Medicine,Shanghai Jiao Tong University,Shanghai 200127,China)

机构地区：[1]上海交通大学医学院附属上海儿童医学中心药剂科,上海200127 [2]上海交通大学医学院附属上海儿童医学中心神经内科,上海200127

出　　处：《药学前沿》2025年第2期355-360,共6页China Pharmacist

摘　　要：约25%的癫痫儿童经两种耐受性良好的抗癫痫发作药物联合治疗后仍不能控制发作,为药物难治性癫痫。司替戊醇(STP)是第二代抗癫痫发作药物,具有独特的药理学机制和药动学特性,通过作用于γ-氨基丁酸(GABA)能系统发挥直接抗惊厥作用,也可通过肝药酶抑制特性增强其他抗癫痫发作药物的疗效,发挥间接抗惊厥作用,其抑制乳酸脱氢酶(LDH)及神经保护作用也得到研究证实。目前,STP在国内外获批作为Dravet综合征一线治疗药物氯巴占及丙戊酸基础上的添加治疗。临床研究显示,STP作为儿童难治性非典型失神癫痫、部分性癫痫发作的添加治疗可能有效,且耐受性较好,短期不良事件主要包括神经系统反应(30%)和胃肠道反应(29%),而食欲减退(16%)和体重减轻(6%)是常见的STP长期应用不良事件,这些不良事件均呈剂量相关性,可通过减少剂量获得缓解。About 25% of children with epilepsy cannot control their seizures after combined treatment with two well tolerated antiepileptic drugs,which is known as drug-resistant epilepsy.Stiripentol(STP)is a second-generation antiepileptic drug with unique pharmacological mechanisms and pharmacokinetic properties.It exerts direct anticonvulsant effects by acting on the gamma aminobutyric acid(GABA)system,and can also enhance the efficacy of other antiepileptic drugs through hepatic enzyme inhibition properties,exerting indirect anticonvulsant effects.Its inhibition of lactate dehydrogenase(LDH)and neuroprotective effects have also been confirmed by research.Currently,STP has been approved both domestically and internationally as an adjunct therapy to firstline treatment drugs for Dravet syndrome,based on chlorpromazine and valproic acid.Clinical studies have shown that STP,as an adjunct therapy for refractory atypical absence epilepsy and partial epileptic seizures in children,may be effective and well tolerated.Short-term adverse events mainly include neurological reactions(30%)and gastrointestinal reactions(29%),while appetite loss(16%)and weight loss(6%)are common long-term adverse events of STP.These adverse events are dose-dependent and can be relieved by reducing the dose.

关 键 词：司替戊醇 难治性癫痫 DRAVET综合征 药物相互作用 儿童 不良反应 

分 类 号：R985[医药卫生—药品]