Chen Celine, Luca Stephanie, Hansen Abigail, Wherrett Diane K, Witteman Holly O, Chakraborty Pranesh, L'Espérance Audrey, Wilson Michael G, McGavock Jonathan, Delorme Sasha, Hayeems Robin Z
Child Health Evaluative Sciences, Hospital for Sick Children Research Institute, Toronto, Ontario, Canada.
University of Toronto Temerty Faculty of Medicine, Toronto, Ontario, Canada.
BMJ Open. 2025 Jun 8;15(6):e099537. doi: 10.1136/bmjopen-2025-099537.
Type 1 diabetes is a chronic autoimmune disease that often presents with diabetic ketoacidosis at diagnosis. Since detection of type 1 diabetes risk is possible using genetic risk scores and autoantibody assays, prevention of diabetic ketoacidosis or delayed onset of type 1 diabetes may be possible and may improve outcomes. Several pilot screening programmes for type 1 diabetes risk have emerged worldwide but outcomes measured in these screening programmes are heterogeneous, making it difficult to compare and synthesise findings across studies. To improve the standardisation of outcome reporting and measurement, we aim to develop a patient-oriented core outcome set for studies of type 1 diabetes risk screening.
This five-step protocol was developed in alignment with the COS-STAndardised Protocol Statement and the Core Outcome Measures in Effectiveness Trials framework. The five steps will include: (1a) conducting a rapid literature review, (1b) gathering input on candidate outcomes from members of the public, (2) combining literature and public input to prepare a preliminary list of outcomes, (3) conducting Delphi surveys with a range of stakeholders to begin to establish consensus on outcomes, (4) holding a final consensus meeting to establish consensus on outcomes and (5) establishing the outcome measurement instruments for the core outcome set.
Ethics approval has been provided by The Hospital for Sick Children Research Ethics Board. The core outcome set will be distributed to researchers and clinicians involved in diabetes screening and clinical care, patient and family networks, research funders, journal editors, public health experts, and policymakers. Disseminated materials will be tailored to the various end users in the form of publication through academic journals, policy briefs, conferences, educational webinars, websites and social media.
1型糖尿病是一种慢性自身免疫性疾病，诊断时常常伴有糖尿病酮症酸中毒。由于使用遗传风险评分和自身抗体检测能够检测1型糖尿病风险，预防糖尿病酮症酸中毒或延缓1型糖尿病发病或许可行，且可能改善预后。全球已出现了多项针对1型糖尿病风险的试点筛查项目，但这些筛查项目所测量的结果具有异质性，使得跨研究比较和综合研究结果变得困难。为提高结果报告和测量的标准化程度，我们旨在为1型糖尿病风险筛查研究制定一套以患者为导向的核心结局集。
本五步方案是依据《COS标准化方案声明》和《有效性试验核心结局指标》框架制定的。这五个步骤将包括：（1a）进行快速文献综述，（1b）收集公众对候选结局的意见，（2）结合文献和公众意见编制初步结局清单，（3）与一系列利益相关者进行德尔菲调查，以开始就结局达成共识，（4）召开最终共识会议以就结局达成共识，（5）为核心结局集确定结局测量工具。
已获得病童医院研究伦理委员会的伦理批准。核心结局集将分发给参与糖尿病筛查和临床护理的研究人员和临床医生、患者及家庭网络、研究资助者、期刊编辑、公共卫生专家和政策制定者。传播材料将根据不同终端用户的需求进行定制，以学术期刊发表、政策简报、会议、教育网络研讨会、网站和社交媒体等形式呈现。
