西罗莫司治疗儿童结节性硬化症合并癫痫的临床研究

Clinical study of sirolimus in children with tuberous sclerosis complicated with epilepsy

目的探讨西罗莫司治疗儿童结节性硬化症合并癫痫的疗效及安全性。方法选取2015年5月~2018年6月于我院确诊的结节性硬化症合并癫痫患儿144例,随机分为实验组和对照组各72例,实验终末最后入组106例,其中实验组58例,对照组48例,实验组予西罗莫司联合抗癫痫药物治疗,对照组予常规抗癫痫药物治疗。观察两组治疗24周、48周后的临床疗效,观察西罗莫司的不良反应及安全性。结果治疗24周、48周后实验组总有效率分别为70.69%、79.31%,脑电图好转率分别为72.41%、77.59%;对照组总有效率分别为33.33%、52.08%,脑电图好转率分别为37.50%、47.92%,差异有统计学意义(P<0.05)。实验组的口腔溃疡发生率相对较高,两组患者的其他不良反应发生率比较,差异无统计学意义(P>0.05)。结论西罗莫司治疗儿童结节性硬化症合并癫痫具有良好的临床疗效,且安全性好,不良反应少。

Objective To investigate the efficacy and safety of sirolimus in the treatment of tuberous sclerosis complicated with epilepsy in children.Methods A total of 144 children with tuberous sclerosis and epilepsy diagnosed in our hospital from May 2015 to June 2018 were randomly divided into the study group and the control group,with 72 cases in each group.At the end of the study,106 cases were included,with 58 cases in the study group and 48 cases in the control group.The study group was given sirolimus combined with antiepileptic drugs,and the control group was given conventional antiepileptic drugs.The clinical efficacy of the two groups after 24 weeks and 48 weeks of treatment,the adverse reactions and safety of sirolimus were compared between the two groups.Results After 24 weeks and 48 weeks of treatment,the total effective rate in the study group was 70.69%and 79.31%,respectively,and the EEG improvement rates were 72.41%and 77.59%,respectively;while the total effective rate in the control group was 33.33%and 52.08%,respectively,and the EEG improvement rates were 37.50%and 47.92%,respectively,and the differences were statistically significant(P<0.05).The incidence of oral ulcers in the study group was relatively high,and there was no significant difference in the incidence of other adverse reactions between the two groups(P>0.05).Conclusion Sirolimus has remarkable efficacy in treating tuberous sclerosis complicated with epilepsy in children,and has higher safety and fewer adverse reactions.