目的 研究小兒積滯臨床隨機對照試驗的設(shè)計要素，為小兒積滯中藥臨床試驗設(shè)計與評價技術(shù)指南的制定提供文獻研究基礎(chǔ)。方法 計算機系統(tǒng)檢索中文學術(shù)期刊全文數(shù)據(jù)庫（CNKI）、維普中文科技期刊數(shù)據(jù)庫（VIP）、萬方數(shù)據(jù)庫（Wanfang Data）、中國生物醫(yī)學文獻數(shù)據(jù)庫（CMB），Embase、PubMed、Web of Science、Cochrane Library數(shù)據(jù)庫中關(guān)于小兒積滯臨床隨機對照試驗中英文文獻，檢索年限為建庫至2021年12月。對符合納入標準的文獻進行資料提取、分析，提煉小兒積滯臨床試驗設(shè)計與評價的主要技術(shù)要素。結(jié)果 共檢索到文獻1 983篇，根據(jù)文獻納排標準最終納入21篇文獻。目標定位，18項定位于消除或改善全部臨床癥狀，3項選擇消除或改善主要臨床癥狀。試驗總體設(shè)計采用隨機21項，盲法設(shè)計7項；樣本量估算3項；陽性藥對照20項，安慰劑對照1項；多中心研究4項；差異性檢驗設(shè)計20項，非劣效檢驗設(shè)計1項。21項研究均有明確中醫(yī)診斷，16項有明確西醫(yī)診斷標準來源。受試者的選擇與退出，21項研究設(shè)有納入及排除標準。干預(yù)措施，中藥內(nèi)治17項，中藥貼敷1項，中藥貼敷聯(lián)合雙歧桿菌顆粒1項，中成藥聯(lián)合推拿手法治療1項，單獨使用推拿手法治療1項。試驗流程，21項研究均未設(shè)導(dǎo)入期，設(shè)計隨訪研究4項；療程4～28 d，以7 d或14 d為主。有效性評價，18項以疾病臨床有效率為主要評價指標，3項以主要癥狀，或其總分，或以其定義的“改善”或“控制”或“消失”比例，作為主要評價指標；安全性評價，18項對不良事件或不良反應(yīng)進行了詳細描述，7項對實驗室常規(guī)檢查進行描述，5項對生命體征進行描述；全部研究中，有倫理批件號說明2項。結(jié)論 納入的文獻信息較完善，基本涵蓋了小兒積滯臨床試驗設(shè)計與評價的主要技術(shù)要素，對中成藥治療小兒積滯臨床試驗設(shè)計關(guān)鍵環(huán)節(jié)和常見問題進行了分析總結(jié)，具有較高借鑒與參考價值，為《小兒積滯中藥臨床試驗設(shè)計與評價技術(shù)指南》的制定提供了文獻依據(jù)。

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

重大新藥創(chuàng)制專項資助項目（2020ZX09201-008）