小儿厌食症临床试验设计与评价技术要点的文献研究

目的]系统评估、提炼小儿厌食症临床试验设计与评价技术要点,为试验设计的标准化和试验结果的相互比较提供可行性。方法]检索国内外权威数据库中的小儿厌食症/厌食及其相关疾病随机对照临床试验(RCT)文献,纳入文献要求符合厌食症及其相关疾病诊断标准,干预措施为中医药、化学药、行为疗法,语种为中英文。结果]共检索出文献1 321篇,最终纳入21篇。其临床定位,14项国内研究中,均以改善食欲不振症状或和增加食量为主,同时探讨改善生长和营养状况3项;7项国外研究中,以改善摄食行为、食欲不振症状、摄食量、小儿生长和营养状况为主,分别为4、1、1、1项。试验总体设计,选择阳性药、安慰剂、行为疗法和空白对照,分别为14、4、1、2项;采用双盲法10项,多中心试验9项,有样本量估算1项;采用差异性检验19项,非劣效检验、优效性检验各1项。中、西医诊断标准,主要参考或符合世界卫生组织(WHO)国际疾病分类、国内外专业学会或权威书籍,以及中国高等院校统编教材标准。受试者选择,具有纳入标准、排除标准、脱落剔除标准设计,分别为21、18、7项。干预措施,采用中医药、化学药、行为干预治疗分别为11项、4项、3项,联合治疗3项;疗程在10 d～8周,以2～4周居多。有效性评价,14项国内研究中,以食欲食量症状改善综合评价为主8项,以中医症状整体评价为主6项;7项国外研究中,以摄食行为量表评价为主4项,食欲不振改善、管饲喂养与口服卡路里的比例、体质量为主要指标各1项。安全性观察,有明确指标设计12项。此外,全部21项研究中,有伦理审批表达仅1项。结论]文章纳入的RCT文献信息完善、质量较高,研究结果涵盖了小儿厌食症中药临床试验设计的基本要素,具有一定的借鉴和参考价值。

A literature study on the design and evaluation techniques of clinical trials for infantile anorexia

Objective] To systematically evaluate and refine the technical points of clinical trial design and evaluation of infantile anorexia,so as to provide feasibility for the standardization of trial design and the comparison of trial results.Methods] The radomized controlled trial(RCT) literatures of infantile anorexia/anorexia and its related diseases were searched in the authoritative database at home and abroad. The included literature required compliance with the diagnostic criteria for anorexia and related diseases. The intervention measures were Chinese medicine,chemical medicine and behavior therapy,and the languages were Chinese and English.Results] A total of 1321 articles were retrieved and 21 were included. In its clinical positioning,14 domestic studies have focused on improving anorexia or increasing food intake,while 3 items were exploring the improvement of growth and nutritional status,7 foreign studies were mainly focused on improving feeding behavior,anorexia symptoms,food intake,child growth and nutritional status,which were 4 items,1 item,1 item and 1 item respectively. The overall design of the trial included 14 items of positive drugs,4 items of placebo,1 item of behavioral therapy and 2 items of blank control,10 items of double-blind method,9 items of multicenter trial,1 item of sample size estimation,19 items of difference test,1 item of non-inferiority test and 1 item of superiority test. The diagnostic criteria of Chinese and Western medicine mainly refer to or comply with the World Health Organization(WHO) international classification of diseases,professional societies or authoritative books at home and abroad,as well as the standard of teaching materials compiled by Chinese colleges and universities. Participants were selected with 21 items,18 items and 7 items respectively for inclusion criteria,exclusion criteria and shedding criteria. The intervention measures included 11 items of traditional Chinese medicine,4 items of chemical drugs,3 items of behavioral intervention and 3 items of combined therapy,and the course of treatment ranged from 10 days to 8 weeks,mostly from 2 to 4 weeks. Effectiveness evaluation,in 14 domestic studies,comprehensive evaluation of appetite appetite improvement was mainly 8 items,and overall evaluation of traditional Chinese medicine symptoms was 6 items;in 7 foreign studies,4 items were mainly evaluated by feeding behavior scale;improved appetite loss,tube feeding and oral caloric ratio,and body weight as the main indicators were 1 item respectively. Safety observation,there are 12 items have clear index design. In addition,there was only one was expressed in ethical approval in all 21 studies.Conclusion] The RCT literature included in this paper has perfect information and high quality,and the research results cover the basic elements of clinical trial design of traditional Chinese medicine for children with anorexia,which can be used for reference value.