婴儿癫痫性痉挛综合征临床试验评价指标的研究进展

doi:10.20274/j.cnki.issn.1674-3865.2025.03.006

中国中西医结合儿科学 ›› 2025, Vol. 17 ›› Issue (3): 221-225.doi: 10.20274/j.cnki.issn.1674-3865.2025.03.006

婴儿癫痫性痉挛综合征临床试验评价指标的研究进展

解怡涵¹, 梅思颖², 张喜莲²()

300181 天津，天津中医药大学2022级中医儿科学专业研究生（解怡涵）
天津中医药大学第一附属医院儿科/国家中医针灸临床医学研究中心（梅思颖，张喜莲）

收稿日期:2024-12-12 修回日期:2025-02-19 出版日期:2025-06-25 上线日期:2025-06-25
通讯作者: 张喜莲 E-mail:zxl2072@126.com
作者简介:解怡涵(1999?)，女，天津中医药大学2022级硕士研究生在读。研究方向：中医药治疗小儿脑系、肺系病证
基金资助:
国家自然科学基金面上项目(82174434);第五批全国中医临床优秀人才研修项目(国中医药办人教函〔2021〕271号)

Research progress on evaluation indexes for clinical trials of infantile epileptic spasms syndrome

Yihan XIE¹, Siying MEI², Xilian ZHANG²()

^1.Tianjin University of Traditional Chinese Medicine, Tianjin 300181, China
^2.First Teaching Hospital of Tianjin University of Traditional Chinese Medicine, National Clinical Research Center for Chinese Medicine Acupuncture and Moxibustion, Tianjin 300181, China

Received:2024-12-12 Revised:2025-02-19 Published:2025-06-25 Online:2025-06-25
Contact: Xilian ZHANG E-mail:zxl2072@126.com
Supported by:
National Natural Science Foundation(82174434)

摘要/Abstract

摘要：

婴儿癫痫性痉挛综合征（IESS）是一种早发性癫痫性脑病，其临床治疗效果欠佳、预后不良，对IESS治疗方案的探索和优化仍是目前的研究热点。近年来有关治疗IESS的临床试验不断开展，本研究查阅文献，对相关评价指标进行了归纳总结：对于疗效评价，痉挛发作情况是最基本和直接的指标，部分研究会联合脑电图改变综合分析；关于脑电图评价，Krammer评分、波幅负荷和癫痫样放电评分系统（BASED）是评估高度失律程度的常用工具，此外部分脑电图生物标志物有望成为更具标准化的评价指标；远期随访需关注患儿的神经运动发育、发作复发率等，其中神经运动发育情况可选用Gesell量表、Bayley量表、Griffiths量表等进行评价；手术后的远期疗效评价最常使用Engel分级量表和国际抗癫痫联盟（ILAE）分级量表；安全性评价以检测相应指标、报告不良事件和统计不良反应发生率为主；中医药治疗IESS具有一定的优势和潜力，但临床试验稀缺，应进一步发掘中医药特色指标。

关键词: 婴儿癫痫性痉挛综合征, 评价指标, 临床试验, 儿童

Abstract:

Infantile epileptic spasms syndrome (IESS) is a kind of early?onset epileptic encephalopathy, which has a poor clinical outcome and prognosis, and the optimization of the treatment plan for IESS is a hot research topic at present. In recent years, relevant clinical trials have been carried out continuously, and this study summarizes the evaluation indexes for IESS based on the review of literatures: for the evaluation of efficacy, seizure attack is the most basic and direct index, and some of the studies make comprehensive analysis in conjunction with the changes of EEG; regarding the evaluation of EEG, the Krammer score and BASED are commonly used to evaluate the degree of heightened dysrhythmia, and in addition, some of EEG biomarkers are expected to become more standardized evaluation indexes; long?term follow?up needs to focus on the neuromotor development of children, seizure recurrence rate and so on, of which neuromotor development can be evaluated by using scales such as Gesell Scale, Bayley Scale and Griffiths Scale; for the long?term efficacy evaluation after the surgery, the Engel Rating Scale and the ILAE Rating Scale are most commonly used; in the safety evaluation the detection of the corresponding indexes, the reports on adverse events and the records of incidence of adverse reactions are the main indexes; the treatment for IESS with Chinese medicine has certain advantages and potentials, but clinical trials are scarce, and the characteristic indicators of Chinese medicine should be further explored.

Key words: Infantile epileptic spasms syndrome, Evaluation index, Clinical trial, Child

中图分类号:

R742.1

解怡涵, 梅思颖, 张喜莲. 婴儿癫痫性痉挛综合征临床试验评价指标的研究进展[J]. 中国中西医结合儿科学, 2025, 17(3): 221-225.

Yihan XIE, Siying MEI, Xilian ZHANG. Research progress on evaluation indexes for clinical trials of infantile epileptic spasms syndrome[J]. Chinese Pediatrics of Integrated Traditional and Western Medicine, 2025, 17(3): 221-225.

参考文献 33

[1]	Zuberi SM, Wirrell E, Yozawitz E, et al. ILAE classification and definition of epilepsy syndromes with onset in neonates and infants: position statement by the ILAE Task Force on Nosology and Definitions[J]. Epilepsia, 2022, 63(6): 1349-1397.
[2]	中国抗癫痫协会. 临床诊疗指南-癫痫病分册: 2023修订版[M]. 北京: 人民卫生出版社, 2023: 18-19.
[3]	Wilmshurst JM, Ibekwe RC, O’Callaghan FJK. Epileptic spasms- 175 years on: trying to teach an old dog new tricks[J]. Seizure, 2017, 44: 81-86.
[4]	Lux AL, Osborne JP. A proposal for case definitions and outcome measures in studies of infantile spasms and West syndrome: consensus statement of the West Delphi group[J]. Epilepsia, 2004, 45(11): 1416-1428.
[5]	O’Callaghan FJK, Edwards SW, Alber FD, et al. Vigabatrin with hormonal treatment versus hormonal treatment alone(ICISS) for infantile spasms: 18⁃month outcomes of an open⁃label, rando⁃mised con⁃trolled trial[J]. Lancet Child Adolesc Health, 2018, 2(10): 715-725.
[6]	秦霞霞. 生酮饮食治疗婴幼儿发育性癫痫性脑病的有效性和安全性分析[D]. 太原: 山西医科大学, 2023.
[7]	Kapoor D, Sharma S, Garg D, et al. Intravenous methylpredni⁃solone versus oral prednisolone for west syndrome: a randomized open⁃label trial[J]. Indian J Pediatr, 2021, 88(8): 778-784.
[8]	王秋红. 镁离子对一线治疗失败婴儿痉挛症的疗效与安全性研究及罕见基因相关婴儿痉挛症的表型与基因型分析[D]. 北京: 中国人民解放军医学院, 2021.
[9]	马娜,王葆辉. 不同剂量泼尼松联合托吡酯治疗婴儿痉挛症的效果比较[J]. 中国实用医刊,2023,50(12):100-103.
[10]	Kramer U, Sue WC, Mikati MA. Hypsarrhythmia: frequency of variant patterns and correlation with etiology and outcome[J]. Neurology, 1997, 48(1): 197-203.
[11]	Zhang Y, Yu L, Zhou Y, et al. Prognostic utility of hypsarrhy⁃thmia scoring in children with West syndrome after ketogenic diet[J]. Clin Neurol Neurosurg, 2019, 184: 105402.
[12]	Mytinger JR, Hussain SA, Islam MP, et al. Improving the inter⁃rater agreement of hypsarrhythmia using a simplified EEG gra⁃ding scale for children with infantile spasms[J]. Epilepsy Res, 2015, 116: 93-98.
[13]	汤志鸿, 陈伟岗, 尹映仪, 等. 波幅负荷和癫痫样放电评分系统对促肾上腺皮质激素治疗婴儿癫痫性痉挛综合征疗效的评估价值[J]. 癫痫杂志, 2024, 10(4): 308-312.
[14]	Chu YJ, Chang CF, Weng WC, et al. Electroencephalography complexity in infantile spasms and its association with treatment response[J]. Clin Neurophysiol, 2021, 132(2): 480-486.
[15]	Smith RJ, Hu DK, Shrey DW, et al. Computational characteristics of interictal EEG as objective markers of epileptic spasms[J]. Epilepsy Res, 2021, 176: 106704.
[16]	Shrey DW, Kim McManus O, Rajaraman R, et al. Strength and stability of EEG functional connectivity predict treatment response in infants with epileptic spasms[J]. Clin Neurophysiol, 2018, 129(10): 2137-2148.
[17]	董燕, 徐瑞娟, 史亚丽, 等. 激素冲击治疗对婴儿痉挛症患儿脑功能网络属性特征路径长度的影响[J]. 中华实用诊断与治疗杂志, 2022, 36(4): 394-398.
[18]	叶小飞, 胡盼盼, 杨阳, 等. 婴儿痉挛症儿童脑网络特点及ACTH疗效分析[J]. 安徽医学, 2024, 45(10): 1222-1226.
[19]	Trevathan E, Murphy CC, Yeargin⁃Allsopp M. The descriptive epidemiology of infantile spasms among Atlanta children[J]. Epilepsia, 1999, 40(6): 748-751.
[20]	郭英英, 赵瑾珠, 郝燕. Infanib量表和Gesell发育量表在儿童运动发育评估中的一致性研究[J]. 中国中西医结合儿科学, 2024, 16(2): 144-147.
[21]	易受蓉, 罗学荣, 杨志伟, 等. 贝利婴幼儿发展量表在我国的修订(城市版)[J]. 中国临床心理学杂志, 1993, 1(2): 71-75.
[22]	黎思娴,陈彦,姚晨,等. Griffiths发育评估量表在一例难治性癫痫术前评估中的定侧定位作用报道及文献复习[J]. 中华神经医学杂志,2020,19(1):67-69.
[23]	王素娟, 李惠, 杨红, 等. Peabody运动发育量表[J]. 中国康复理论与实践, 2006, 12(2): 181-182.
[24]	Sumanasena SP, Wanigasinghe J, Arambepola C, et al. Effect of intramuscular ACTH versus oral prednisolone on the developmental trajectories of children with west syndrome over 24 months: a randomised control study[J]. Eur J Paediatr Neurol, 2021, 32: 98-105.
[25]	Pavone P, Polizzi A, Marino SD, et al. West syndrome: a comprehensive review[J]. Neurol Sci, 2020, 41(12): 3547-3562.
[26]	Schmid MM, Freudenmann RW, Keller F, et al. Non⁃fatal and fatal liver failure associated with valproic acid[J]. Pharmacopsychiatry, 2013, 46(2): 63-68.
[27]	中国医师协会神经内科医师分会儿童神经专业委员会,中华医学会儿科学分会神经学组. 生酮饮食治疗儿童癫痫性脑病循证指南[J]. 中华实用儿科临床杂志,2019,34(12):881-888.
[28]	刘琦, 窦万臣. 癫痫预后评估量表的研究进展[J]. 癫痫与神经电生理学杂志, 2023, 32(3): 171-175.
[29]	Kwon HE, Kim SH, Kang HC, et al. Epilepsy surgery for pediatric patients with mild malformation of cortical development[J]. Seizure, 2020, 82: 50-55.
[30]	易招师, 查剑, 吴华平, 等. 婴儿痉挛症缓解后复发的危险因素[J]. 中华实用儿科临床杂志, 2016, 31(12): 899-902.
[31]	彭炜. 儿童癫痫性脑病的临床特点和马融教授中药添加治疗EE的临床观察[D]. 天津: 天津中医药大学, 2022.
[32]	王莉莉, 关玲. 艾灸足三里对婴儿痉挛症患者头面部红外热像的影响[J]. 上海针灸杂志, 2015, 34(2): 145-148.
[33]	Wang X, Huang Q, Wu L, et al. Application of peripheral blood cytokine and immunoglobulin detection in ACTH therapy for the treatment of infantile spasms[J]. Front Pediatr, 2024, 12: 1365917.

婴儿癫痫性痉挛综合征临床试验评价指标的研究进展

Research progress on evaluation indexes for clinical trials of infantile epileptic spasms syndrome

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