氨己烯酸治疗结节性硬化症伴癫痫的疗效及近期安全性分析

Vigabatrin for seizures treatment in patients with tuberous sclerosis complex: an efficacy and safety study

目的 观察氨己烯酸(Vigabatrin,VGB)治疗结节性硬化症(Tuberous sclerosis complex,TSC)伴癫痫的疗效、耐受性及用药6个月内的安全性.方法 选取2015年1月-12月于解放军总医院儿童医学中心诊治的41例TSC伴癫痫患儿,应用VGB治疗,起始剂量20 mg/(kg·d),维持剂量50~ 100 mg/(kg·d).基线水平的发作频率由患儿父母或监护人完成评价,观察治疗后3、6个月的疗效、不良反应及安全性,进行自身对照观察.疗效判定标准:癫痫发作减少100%为完全控制;减少75% ~99%为显效;减少50%~ 74%为有效;减少<50%为无效.并观察用药期间出现的不良反应.结果 治疗3、6个月后完全控制率分别为51.2%、57.9%,总有效率(完全控制+显效+有效)分别为90.2%、89.5%.治疗6个月内,l例因疗效差及药品购买困难退出(2.4%);发生不良反应14例,主要不良反应有嗜睡、兴奋、多动、肌阵挛等,均较轻微且持续时间短,无患儿出现临床医生可察觉的视野改变.结论 VGB治疗TSC伴癫痫患儿疗效可靠,不良反应小,耐受性及近期安全性好.

Obective To evaluate the efcacy,tolrability and safety of vigabatrin(VGB)for seizure treatment in patients with tuberous sclerosis complex(TSC).Methods Forty-one epilepsy patients with tuberous sclersis complex,adnited from January 2015 to December 2015,were included in our study;they were treated with VGB with an initial dose of 20 mg/(kg.d),and a maintenance dose of 50~100 mg/(kg.d).Baseline seizure frequeney were evaluated by the parents or the guardian,and investigated the eficacy,tolerability,adverse reactions and safety in 3 and 6 months after treatment,and cornpared with the baseline.The treatment outcomes were evaluated by seizure frequency as completely seizure free(100%seizure reduction),markedly efctivee(75%~99%seirure reduction),efletive(50%~74%seizure reduction)and invalid(<50%seizure reduction).Adverse reactions were observed during treatment.Results The completely seizure free rates after 3 and 6 months treatment were 51.2%and57.9%;and the total efective rates(completely seiure free+markedly efctive+ecive)were 90.2%and 89.5%..During the 6 months,only one patients stopped VGB use because of the poor eficacy and the dificulties to buy this medieine.14 patients appeared adversc reactions,including drowsiness,agitation,hyperactivity and myoclonus,which were transient and mild.No patients had clinically perceivable visual-field changcs on clinical examination.Conclusion VGB is a ffctive treament in TSC patients with epilepsy,and have a good security in short term.