Chevidence Lab of Child and Adolescent Health, Children's Hospital of Chongqing Medical University, Chongqing, China.
National Clinical Research Center for Child Health and Disorders, Chongqing, China.
JAMA Pediatr. 2022 Nov 1;176(11):1131-1141. doi: 10.1001/jamapediatrics.2022.3181.
Developing core outcome sets is essential to ensure that results of clinical trials are comparable and useful. A number of core outcome sets in pediatrics have been published, but a comprehensive in-depth understanding of core outcome sets in this field is lacking.
To systematically identify core outcome sets in child health, collate the diseases to which core outcome sets have been applied, describe the methods used for development and stakeholder participation, and evaluate the methodological quality of existing core outcome sets.
MEDLINE, SCOPUS, Cochrane Library, and CINAHL were searched using relevant search terms, such as clinical trials, core outcome, and children, along with relevant websites, such as Core Outcome Measures in Effectiveness Trials (COMET). Four researchers worked in teams of 2, performed literature screening and data extraction, and evaluated the methodological quality of core outcome sets using the Core Outcome Set-Standards for Development (COS-STAD).
A total of 77 pediatric core outcome sets were identified, mainly developed by organizations or researchers in Europe, North America, and Australia and mostly from the UK (22 [29%]) and the US (22 [29%]). A total of 77 conditions were addressed; the most frequent International Classification of Diseases, 11th Revision category was diseases of the digestive system (14 [18%]). Most of the outcomes in pediatric core outcome sets were unordered (34 [44%]) or presented in custom classifications (29 [38%]). Core outcome sets used 1 or more of 8 development methods; the most frequent combination of methods was systematic review/literature review/scoping review, together with the Delphi approach and consensus for decision-making (10 [14%]). Among the 6 main types of stakeholders, clinical experts were the most frequently involved (74 [100%]), while industry representatives were rarely involved (4 [5%]). Only 6 core outcome sets (8%) met the 12 criteria of COS-STAD.
Future quality of pediatric core outcome sets should be improved based on the standards proposed by the COMET initiative, while core outcome sets methodology and reporting standards should be extended to pediatric populations to help improve the quality of core outcome sets in child health. In addition, the COMET outcome taxonomy should also add items applicable to children.
制定核心结局集对于确保临床试验结果具有可比性和有用性至关重要。已经发布了许多儿科核心结局集，但对该领域核心结局集的综合深入了解仍有所欠缺。
系统地确定儿童健康领域的核心结局集，整理已应用于核心结局集的疾病，描述其开发方法和利益相关者的参与情况，并评估现有核心结局集的方法学质量。
使用相关的搜索词（如临床试验、核心结局和儿童）在 MEDLINE、SCOPUS、Cochrane Library 和 CINAHL 中进行了搜索，并查阅了 Core Outcome Measures in Effectiveness Trials (COMET) 等相关网站。四名研究人员组成 2 人一组，进行文献筛选和数据提取，并使用核心结局集标准化开发标准（COS-STAD）评估核心结局集的方法学质量。
共确定了 77 项儿科核心结局集，主要由欧洲、北美和澳大利亚的组织或研究人员制定，主要来自英国（22 项[29%]）和美国（22 项[29%]）。共涉及 77 种疾病；最常见的国际疾病分类第 11 版类别是消化系统疾病（14 项[18%]）。儿科核心结局集中的大多数结局都是无序的（34 项[44%]）或呈现自定义分类（29 项[38%]）。核心结局集使用了 1 种或多种 8 种开发方法；最常见的方法组合是系统评价/文献综述/范围综述，结合德尔菲法和共识决策（10 项[14%]）。在 6 种主要利益相关者类型中，临床专家是最常参与的（74 项[100%]），而工业代表很少参与（4 项[5%]）。只有 6 项核心结局集（8%）符合 COS-STAD 的 12 项标准。
未来儿科核心结局集的质量应根据 COMET 倡议提出的标准进行改进，同时应将核心结局集方法学和报告标准扩展到儿科人群，以帮助提高儿童健康核心结局集的质量。此外，COMET 结局分类法也应增加适用于儿童的项目。
