National Heart and Lung Institute, Imperial College London, London, United Kingdom.
Bureau of Nutritional Sciences, Food Directorate, Health Canada, Ottawa, Ontario, Canada.
JAMA Pediatr. 2020 Sep 1;174(9):874-881. doi: 10.1001/jamapediatrics.2020.0578.
Breast milk substitutes (BMS) are important nutritional products evaluated in clinical trials. Concerns have been raised about the risk of bias in BMS trials, the reliability of claims that arise from such trials, and the potential for BMS trials to undermine breastfeeding in trial participants. Existing clinical trial guidance does not fully address issues specific to BMS trials.
To establish new methodological criteria to guide the design, conduct, analysis, and reporting of BMS trials and to support clinical trialists designing and undertaking BMS trials, editors and peer reviewers assessing trial reports for publication, and regulators evaluating the safety, nutritional adequacy, and efficacy of BMS products.
DESIGN, SETTING, AND PARTICIPANTS: A modified Delphi method was conducted, involving 3 rounds of anonymous questionnaires and a face-to-face consensus meeting between January 1 and October 24, 2018. Participants were 23 experts in BMS trials, BMS regulation, trial methods, breastfeeding support, infant feeding research, and medical publishing, and were affiliated with institutions across Europe, North America, and Australasia. Guidance development was supported by an industry consultation, analysis of methodological issues in a sample of published BMS trials, and consultations with BMS trial participants and a research ethics committee.
An initial 73 criteria, derived from the literature, were sent to the experts. The final consensus guidance contains 54 essential criteria and 4 recommended criteria. An 18-point checklist summarizes the criteria that are specific to BMS trials. Key themes emphasized in the guidance are research integrity and transparency of reporting, supporting breastfeeding in trial participants, accurate description of trial interventions, and use of valid and meaningful outcome measures.
Implementation of this guidance should enhance the quality and validity of BMS trials, protect BMS trial participants, and better inform the infant nutrition community about BMS products.
母乳代用品（BMS）是在临床试验中评估的重要营养产品。人们对 BMS 试验的偏倚风险、由此类试验产生的说法的可靠性以及 BMS 试验对试验参与者母乳喂养的潜在影响表示担忧。现有的临床试验指南并没有完全解决 BMS 试验特有的问题。
制定新的方法学标准，指导 BMS 试验的设计、进行、分析和报告，并为设计和开展 BMS 试验的临床试验人员、评估试验报告以用于发表的编辑和同行评审人员、评估 BMS 产品安全性、营养充足性和疗效的监管人员提供支持。
设计、地点和参与者：采用改良 Delphi 方法，于 2018 年 1 月 1 日至 10 月 24 日进行了 3 轮匿名问卷和一次面对面的共识会议。参与者是 23 名 BMS 试验、BMS 监管、试验方法、母乳喂养支持、婴儿喂养研究和医学出版方面的专家，他们来自欧洲、北美和澳大拉西亚的机构。指导方针的制定得到了行业咨询、对已发表的 BMS 试验样本中的方法学问题的分析以及与 BMS 试验参与者和研究伦理委员会的磋商的支持。
最初向专家发送了 73 项源于文献的标准。最终的共识指南包含 54 项基本标准和 4 项推荐标准。18 点清单总结了 BMS 试验特有的标准。指导方针中强调的关键主题是研究诚信和报告的透明度、支持试验参与者的母乳喂养、准确描述试验干预措施以及使用有效和有意义的结果测量。
实施本指南应能提高 BMS 试验的质量和有效性，保护 BMS 试验参与者，并使婴儿营养界更好地了解 BMS 产品。
