关键词: 结节性硬化症, 痉挛, 婴儿, 甲泼尼龙, 治疗结果

Abstract: Objective　To investigate the efficacy and safety of intravenous high-dose methylprednisolone pulse in treatment of tuberous sclerosis complex（TSC）combined with infantile spasms（IS）.Methods　A retrospective analysis was carried out on 47 children with TSC combined with IS treated in the Third Affiliated Hospital of Zhengzhou University from July 2009 to July 2017，18 cases with duration less than or equal to 2 months and 29 cases longer than 2 months.According to treatment methods，subjects were divided into preferred methylprednisolone group（27 cases）and non-preferred methylprednisolone group（20 cases）.Methylprednisolone group was the first choice for children，and the first choice was high-dose methylprednisolone pulse.The non-preferred methylprednisolone group received high-dose methylprednisolone pulse therapy after failure of other antiepileptic drugs，the other antiepileptic drugs all reached the target dose of more than 2 weeks.The time of spasm controlling and proportion of spasm controllers in the first 5 days were observed.After the treatment，clinical effect in 1 and 2 weeks，EEG outcome in 2 weeks，the relationship between course of disease and clinical effect，and the adverse reactions were recorded.Results　The control time of spasm in the preferred methylprednisolone group was within 1 week of treatment，and the proportion of spasm controllers in the first 5 days was 66.7%（12/18）；the control time of spasm in the non-preferred methylprednisolone group was within 1 week of treatment，and the proportion of spasm controllers in the first 5 days was 57.1%（4/7）.At the 1st and 2nd week of treatment，the total effective rate and complete control rate of methylprednisolone group were higher than those of non-preferred methylprednisolone group（P<0.05）.There was no significant difference in the effective rate of EEG outcome between the two groups（P>0.05）.The complete control rate of EEG outcome in the methylprednisolone group was higher than that in the non-preferred methylprednisolone group（P<0.05）.The total effective rate of clinical efficacy in children with disease course ≤ 2 months was greater than that in patients with disease course > 2 months（P<0.05）.No serious adverse reactions occurred during the pulse treatment of high-dose methylprednisolone in all children.Conclusion　Application of intravenous high-dose methylprednisolone pulse in treatment of TSC combined with IS is simple and effective，and its short-term control effect of spasm is affirmative，with few and light adverse effects.With high safety，simple and feasible treatment，sufficient drug source and low price，it is a recommended application for children who are newly treated or had no response to other treatments.

Key words: Tuberous sclerosis；Spasms, infantile；Methylprednisolone；Treatment outcome