中医药治疗孤独症谱系障碍随机对照试验结局指标分析

Analysis of outcome indicators of randomized controlled trials of traditional Chinese medicine in the treatment of autism spectrum disorder

关键词:  孤独症谱系障碍  中医药  随机对照试验  中医药核心指标集  结局指标

Key words:  autism spectrum disorder  traditional Chinese medicine  randomized controlled trial  core indicator set of traditional Chinese medicine  outcome indicator

摘要:

[目的] 整理中医药治疗孤独症谱系障碍（ASD）的随机对照试验（RCTs）结局指标，分析中存在的问题，为其临床试验设计及构建核心结局指标集提供依据。[方法] 计算机检索2018年1月1日至2023年12月31日中国知网（CNKI）、万方数据知识服务平台（WanFang Database）、中国生物医学文献服务系统（SinoMed）、维普中文期刊服务平台（VIP）、PubMed、Web of Science、Embase、The Cochrane Library数据库中医药治疗ASD的RCTs文献，通过文献筛选和资料提取汇总结局指标及其测量工具并进行整理和分析。[结果] 共纳入72项研究，总样本量为5 207例，单项研究样本量为18~169 例，治疗周期为30 d~10个月；12项研究报告了ASD的中医辨证分型，64项研究报告了ASD的西医诊断标准，17项研究报告了中医诊断标准；有41项研究进行了疗效评价，共使用了13种有效率测量工具。所有研究一共涉及54个结局指标，归类进入8个指标域：临床有效率1项；中医病证类1项；症状/体征类共有35项；理化检测类13项，生活质量类3项，安全性事件类1项，远期预后和经济学评估未评估。单项RCT报告指标最少有1个，最多达8个，所有54个结局指标共应用259次，结局指标应用最多的是儿童孤独症评定量表评分（CARS），共计50次，其次是孤独症行为量表评分（ABC）共报道了45次，临床有效率指标共报道了41次。[结论] 中医药治疗ASD的RCTs结局指标应用现状在临床研究中存在着试验设计方法学的不足，结局指标数量、选择的差异性大、疗效指标设计要素多，忽视不良结局指标、中医药特色指标不足等问题。研究者在遴选结局指标时，应对各种量表和临床疗效参考标准的质量及其适用性进行合理评估，注重中医病证特点，并选用权威可靠工具，助力构建中医药治疗ASD的核心指标集。

Abstract:

[Objective] To sort out the outcome indicators of randomized controlled trials(RCTs) in the treatment of autism spectrum disorder(ASD) with traditional Chinese medicine(TCM),and analyze the existing problems，so as to provide a basis for the design of clinical trials and the construction of core outcome indicators set. [Methods] From January 1，2018 to December 31，2023，CNKI，WanFang Database，SinoMed，VIP，PubMed，Web of Science，Embase，and the Cochrane Library databases were searched for RCTs of TCM treatment of ASD. Through literature screening and data extraction，the outcome indicators and measurement tools were summarized and analyzed. [Results] A total of 72 studies were included，with a total sample size of 5 207 cases and a single study sample size of 18 to 169 cases. The treatment period was 30 days to10 months. The 12 studies reported the TCM syndrome differentiation and classification of ASD，64 studies reported the Western medical diagnostic criteria for ASD，and 17 studies reported the TCM diagnostic criteria. A total of 41 studies were evaluated the efficacy，and 13 effective measurement tools were used. A total of 54 outcome indicators were included in all studies，which were classified into 8 index domains：one clinical effective rate，one TCM syndrome，35 symptoms/signs in total， 13 physical and chemical tests，3 items of quality of life，and one item of safety event. And the long-term prognosis and economic assessment were not assessed. A single RCT reported a minimum of one and a maximum of 8 indicators，and all 54 outcomes were used 259 times. The most frequently applied outcome indicator is the children’s autism rating scale score(CARS)，ａ ｔotal of 50 times，followed by the autism behavior scale score(ABC),wｈich has been reported 45 times，and the clinical efficiency index has been reported 41 times. [Conclusion] The application status of RCTs outcome indicators in the treatment of ASD with traditional Chinese medicine has some problems in clinical research，such as the lack of trial design methodology，large differences in the number and selection of outcome indicators，many elements in the design of efficacy indicators，the neglect of adverse outcome indicators，and insufficient characteristic indicators of TCM. When selecting outcome indicators，researchers should reasonably evaluate the quality and applicability of various scales and clinical efficacy reference standards，pay attention to the characteristics of TCM disease syndromes，and select authoritative and reliable tools to help construct the core index set of TCM in the treatment of ASD.