中医药治疗重症肌无力随机对照试验结局指标选用现状研究  

Current Status of the Selection of Outcomes in Randomized Controlled Trials of Traditional Chinese Medicine in the Treatment of Myasthenia Gravis

作　　者：彭思扬 李少红 田煜坤 孟令昊 方瑞英 朱文增[1] PENG Siyang;LI Shaohong;TIAN Yukun;MENG Linghao;FANG Ruiying;ZHU Wenzeng(Department of Acupuncture,Guang'anmen Hospital,China Academy of Chinese Medical Sciences,Beijing 100053,China)

机构地区：[1]中国中医科学院广安门医院针灸科,北京市100053

出　　处：《中国全科医学》2023年第11期1340-1347,共8页Chinese General Practice

基　　金：中国中医科学院科技创新工程(CI2021A01309)——基于真实世界探讨益气温阳法治疗重症肌无力的病例注册登记研究。

摘　　要：背景中药和针刺等中医药疗法在重症肌无力(MG)的治疗中广泛应用,但缺乏高质量的循证医学证据验证其有效性和安全性,原因之一是随机对照试验(RCT)设计中缺乏公认、统一的结局指标,进而导致在开展Meta分析等二次研究时由于指标不同而无法合并数据。构建中医药治疗MG的核心结局指标集有助于临床研究设计与高质量循证证据积累。目的描述中医药治疗MG的RCT中结局指标的选用现状,分析存在的问题并提出建议,推动构建中医药核心结局指标集。方法系统检索中国知网(CNKI)、万方数据知识服务平台(Wanfang Data)、维普网(VIP)、中国生物医学文献服务系统(SinoMed)、Medline、PubMed、Cochrane Library与Web of Science数据库,检索时限从各数据库建库至2022-04-01,检索中医药治疗MG的所有RCT,由2位研究者独立筛选文献、提取信息,对结局指标进行分类并统计使用频次,根据结局指标功能属性划分指标域,分析结局指标选用现状与存在的问题,并提出相应建议。结果共纳入186篇文献,涉及71个结局指标,各指标共计选用724次,全部结局指标大致可分为7个大类:重症肌无力严重程度量表(133次)、重症肌无力生活质量量表(20次)、中医证候积分(70次)、安全性指标(66次)、血生化指标(224次)、肌力变化(3次)与其他(208次)。根据结局指标的功能属性可归纳为7个指标域。单篇文献最少选用1项、最多选用12项结局指标,高频使用的指标包括有效率、重症肌无力绝对和相对评分法、中医证候积分等。纳入文献中仅2篇明确报告了主要结局指标与次要结局指标。单篇文献随访次数为2~7次,多数研究(83.33%,155次)选择随访3次,大部分研究(93.55%,174次)未报告是否在结局指标评价时使用盲法。结论目前中医药治疗MG的RCT中结局指标选用存在较多问题,包括主次结局指标区分不明确、缺少国际公认指标、对安�Background Chinese medicine and acupuncture are widely used in the treatment of myasthenia gravis(MG),but there is a lack of high-quality evidence to verify their effectiveness and safety.One of the reasons is the lack of recognized and unified outcomes in the randomized controlled trial(RCT)design,which leads to the inability to combine data in secondary studies such as meta-analysis.The construction of the core outcome set(COS)of traditional Chinese medicine(TCM)for MG is conducive to the design of clinical research and the accumulation of high-quality evidence.Objective To describe the current situation of outcome selection in RCTs of MG treated with TCM,and analyze the existing problems,then put forward suggestions,promoting the construction of a COS of TCM.Methods Electronic databases of CNKI,Wanfang Data,VIP,SinoMed,Medline,PubMed,Cochrane Library,and Web of Science were systematically searched for RCTs of MG treated with TCM from inception to April 1,2022.Two researchers independently screened the literature,extracted data,classified the outcomes and counted their frequency of use,and divided the outcomes into different outcome domains according to the functional attributes.Then,we analyzed the current status and existing problems in the selection of outcomes,and put forward corresponding suggestions.Results A total of 186 RCTs were included,involving 71 outcomes,with a frequency of use of 724 times in total.The roughly classified seven categories of all outcomes with frequency of use are as follows:MG severity scale(133 times),MG quality of life scale(20 times),TCM syndrome score(70 times),safety outcomes(66 times),blood biochemical outcomes(224 times),muscle strength changes(three times),and others(208 times).According to the functional attributes,the outcomes can be classified into seven outcome domains.A single study selected at least one and at most 12 outcomes.The most frequently used outcomes include effective rate,MG absolute and relative score,and TCM syndrome score.Among the included RCTs,only tw

关 键 词：重症肌无力 结局指标 随机对照试验 中医药 核心指标集 

分 类 号：R746.1[医药卫生—神经病学与精神病学]