氨己烯酸治疗儿童结节性硬化症相关癫痫的疗效及安全性研究进展  

Progress in efficacy and safety of vigabatrin in treatment of epilepsy associated with tuberous sclerosis complex in children

作　　者：朱思齐(综述) 王纪文(审校)[1] Zhu Siqi;Wang Jiwen(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神经内科,200127

出　　处：《国际儿科学杂志》2023年第5期302-305,共4页International Journal of Pediatrics

基　　金：国家重点研发计划(2019YFA0801900)。

摘　　要：结节性硬化症(tuberous sclerosis complex,TSC)是一种常染色体显性遗传皮肤神经综合征,临床表现多样,其中癫痫是最常见的神经系统表现。氨己烯酸(vigabatrin,VGB)为抑制性神经递质γ-氨基丁酸(GABA)类似物,通过不可逆结合GABA转氨酶减少GABA的降解,从而提高中枢神经系统GABA水平,发挥抗癫痫作用,适用于TSC相关各类型癫痫的治疗。近年来,VGB在TSC相关癫痫中的预防作用引起了广泛关注。VGB使用过程中可能存在一定程度的不良反应,其中最值得关注的为视野及中枢神经系统方面的改变。该文就VGB治疗TSC相关癫痫的疗效及安全性进行综述。Tuberous sclerosis complex(TSC)is an autosomal dominant skin-nerve syndrome with diverse clinical manifestations.Epilepsy is the most common neurological manifestation.Vigabatrin(VGB)is an inhibitory neurotransmitterγ-aminobutyric acid(GABA)analogue.It reduces the degradation of GABA by irreversibly binding to GABA transaminase,thereby increasing the level of GABA in the central nervous system and exerting anti-epileptic effects.It is suitable for the treatment of various types of epilepsy related to TSC.In recent years,the preventive effect of VGB in TSC-related epilepsy has attracted wide attention.There is a certain degree of adverse reactions during the use of VGB,the most noteworthy of which is the visual field and central nervous system changes.This article reviews the efficacy and safety of VGB in the treatment of TSC-related epilepsy.

关 键 词：氨己烯酸 结节性硬化症 癫痫 婴儿痉挛 

分 类 号：R725.9[医药卫生—儿科] R742.1[医药卫生—临床医学]