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

摘要： 目的　探讨大剂量甲泼尼龙冲击治疗结节性硬化症（TSC）合并婴儿痉挛症（IS）的早期疗效和安全性。方法　回顾性分析郑州大学第三附属医院2009年7月—2017年7月应用大剂量甲泼尼龙冲击治疗的TSC合并IS患儿47例，其中病程≤2个月18例，病程>2个月29例。根据患儿治疗方法，将其分为首选甲泼尼龙组（27例，为初治患儿，首选大剂量甲泼尼龙冲击治疗）和非首选甲泼尼龙组（20例，为应用其他抗癫痫药物治疗失败后选择大剂量甲泼尼龙冲击治疗，其他抗癫痫药物均达到目标剂量2周以上）。观察痉挛控制时间及前5 d痉挛控制者的比例，治疗1、2周时临床疗效，治疗2周时脑电图转归疗效，病程与临床疗效的关系，不良反应。结果　首选甲泼尼龙组痉挛控制时间均为治疗1周内，前5 d痉挛控制者占66.7%（12/18）；非首选甲泼尼龙组痉挛控制时间均为治疗1周内，前5 d痉挛控制者占57.1%（4/7）。首选甲泼尼龙组治疗1、2周时临床疗效总有效率、完全控制率大于非首选甲泼尼龙组（P<0.05）。两组脑电图转归疗效总有效率比较，差异无统计学意义（P>0.05）；首选甲泼尼龙组脑电图转归疗效完全控制率大于非首选甲泼尼龙组（P<0.05）。治疗2周时病程≤2个月患儿临床疗效总有效率大于病程>2个月患儿（P<0.05）。所有患儿大剂量甲泼尼龙冲击治疗过程中，无严重不良反应发生。结论　大剂量甲泼尼龙冲击治疗TSC合并IS的近期效果肯定，不良反应少而轻，安全性高，且方案简单易行，药源充足，价格便宜，对初治患儿推荐应用，对于其他药物治疗无效的患儿仍可考虑尝试。

关键词: 结节性硬化症, 痉挛, 婴儿, 甲泼尼龙, 治疗结果