目的 研究小儿积滞临床随机对照试验的设计要素，为小儿积滞中药临床试验设计与评价技术指南的制定提供文献研究基础。方法 计算机系统检索中文学术期刊全文数据库（CNKI）、维普中文科技期刊数据库（VIP）、万方数据库（Wanfang Data）、中国生物医学文献数据库（CMB），Embase、PubMed、Web of Science、Cochrane Library数据库中关于小儿积滞临床随机对照试验中英文文献，检索年限为建库至2021年12月。对符合纳入标准的文献进行资料提取、分析，提炼小儿积滞临床试验设计与评价的主要技术要素。结果 共检索到文献1 983篇，根据文献纳排标准最终纳入21篇文献。目标定位，18项定位于消除或改善全部临床症状，3项选择消除或改善主要临床症状。试验总体设计采用随机21项，盲法设计7项；样本量估算3项；阳性药对照20项，安慰剂对照1项；多中心研究4项；差异性检验设计20项，非劣效检验设计1项。21项研究均有明确中医诊断，16项有明确西医诊断标准来源。受试者的选择与退出，21项研究设有纳入及排除标准。干预措施，中药内治17项，中药贴敷1项，中药贴敷联合双歧杆菌颗粒1项，中成药联合推拿手法治疗1项，单独使用推拿手法治疗1项。试验流程，21项研究均未设导入期，设计随访研究4项；疗程4～28 d，以7 d或14 d为主。有效性评价，18项以疾病临床有效率为主要评价指标，3项以主要症状，或其总分，或以其定义的“改善”或“控制”或“消失”比例，作为主要评价指标；安全性评价，18项对不良事件或不良反应进行了详细描述，7项对实验室常规检查进行描述，5项对生命体征进行描述；全部研究中，有伦理批件号说明2项。结论 纳入的文献信息较完善，基本涵盖了小儿积滞临床试验设计与评价的主要技术要素，对中成药治疗小儿积滞临床试验设计关键环节和常见问题进行了分析总结，具有较高借鉴与参考价值，为《小儿积滞中药临床试验设计与评价技术指南》的制定提供了文献依据。

Objective To study the design elements of clinical randomized controlled trials of children with stagnation, and to provide a literature research basis for the development of technical guidelines for clinical trial design and evaluation of children with stagnation. Methods Computer system search CNKI, VIP, Wanfang Data, CMB, Embase, Pubmed, Web of Science, Cochrane Library database on the Chinese and English literature on pediatric stagnant randomized controlled trials, the search period is December 2021. The literature that meets the inclusion criteria was extracted and analyzed, and the main technical elements of the design and evaluation of pediatric accumulation clinical trials were extracted. Results A total of 1 983 articles were retrieved, and 21 articles were finally included according to the inclusion criteria. Target positioning: 18 items are targeted to eliminate or improve all clinical symptoms, and three items are selected to eliminate or improve main clinical symptoms. The overall design of the trial was 21 items at random and seven items were designed by blind method, three sample size estimates, 20 positive drug controls and one placebo control, four multicenter studies. There are 20 differences test designs and one non inferiority test design. All 21 studies have a clear diagnosis of traditional Chinese medicine and 16 have a clear source of diagnostic criteria of western medicine. For the selection and withdrawal of subjects, inclusion and exclusion criteria were set for all studies. Intervention measures: 17 items were treated internally with Chinese medicine, one item was applied with Chinese medicine, one item was applied with Chinese medicine combined with bifidobacterium granules, one item was treated with Chinese patent medicine combined with massage manipulation, and one item was treated with massage manipulation alone. In the trial process, 21 studies had no introduction period, and four follow-up studies were designed. The course of treatment is four to 28 days, mainly seven or 14 days. Effectiveness evaluation: 18 items take the clinical efficiency of disease as the main evaluation index, and three items take the main symptoms, or their total scores, or the defined "improvement", "control" or "disappearance" ratio as the main evaluation index. Safety evaluation: 18 items described adverse events/reactions in detail, seven items described routine laboratory tests, and five items described vital signs. In all the studies, there are two ethical approval documents. Conclusion The literature information included in this study is relatively complete, which basically covers the main technical elements of the design and evaluation of the clinical trial for children with stagnation, and analyzes and summarizes the key links and common problems in the design of the trial for treating children with stagnation with Chinese patent medicine. It has high reference value, and provides a documentary basis for the formulation of Guideline on design and evaluation of clinical trials for Chinese medicine in common pediatric diseases: Food accumulation and stagnation in children.

R965.3

重大新药创制专项资助项目（2020ZX09201-008）