Sydney School of Public Health, The University of Sydney, Sydney, Australia.
Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, Australia.
Clin J Am Soc Nephrol. 2022 Jan;17(1):53-64. doi: 10.2215/CJN.07840621. Epub 2021 Dec 30.
Outcomes reported in trials in adults with glomerular disease are often selected with minimal patient input, are heterogeneous, and may not be relevant for clinical decision making. The Standardized Outcomes in Nephrology-Glomerular Disease (SONG-GD) initiative aimed to establish a core outcome set to help ensure that outcomes of critical importance to patients, care partners, and clinicians are consistently reported.
DESIGN, SETTING, PARTICIPANTS, AND MEASUREMENTS: We convened two 1.5-hour workshops in Melbourne, Australia, and Washington, DC, United States. Attendees were identified purposively with 50 patients/care partners and 88 health professionals from 19 countries; 51% were female. Patients and care partners were from the United States, Australia, and Canada, and had experience of a glomerular disease with systemic features (=9), kidney-limited nephrotic disease (=9), or other kidney-limited glomerular disease (=8). Attendees reviewed the results of the SONG-GD Delphi survey and aims of the workshop and then discussed potential core outcomes and their implementation in trials among moderated breakout groups of eight to 12 people from diverse backgrounds. Transcripts of discussions were analyzed thematically.
Three themes were identified that supported the proposed core outcomes: limiting disease progression, stability and control, and ensuring universal relevance (, applicable across diverse populations and settings). The fourth theme, preparedness for implementation, included engaging with funders and regulators, establishing reliable and validated measures, and leveraging existing endorsements for patient-reported outcomes.
Workshop themes demonstrated support for kidney function, disease activity, death, life participation, and cardiovascular disease, and these were established as the core outcomes for trials in adults with glomerular disease. Future work is needed to establish the core measures for each domain, with funders and regulators central to the uptake of the core outcome set in trials.
成人肾小球疾病试验报告的结果往往是在患者参与度极小的情况下选择的，结果具有异质性，且可能与临床决策无关。标准化肾脏病-肾小球疾病结局（SONG-GD）倡议旨在建立一个核心结局集，以帮助确保报告对患者、照护者和临床医生至关重要的结果。
设计、地点、参与者和测量：我们在澳大利亚墨尔本和美国华盛顿特区举行了两次为时 1.5 小时的研讨会。与会者是通过从 19 个国家中确定的 50 名患者/照护者和 88 名卫生专业人员确定的，其中 51%为女性。患者和照护者来自美国、澳大利亚和加拿大，有全身性肾小球疾病（=9）、肾脏局限型肾病综合征（=9）或其他肾脏局限型肾小球疾病（=8）的患病经历。与会者审查了 SONG-GD Delphi 调查的结果和研讨会的目标，然后在由来自不同背景的 8 至 12 人组成的小组中讨论了潜在的核心结局及其在试验中的实施。讨论的记录通过主题分析进行了分析。
确定了三个支持提出的核心结局的主题：限制疾病进展、稳定性和控制以及确保普遍相关性（适用于不同人群和环境）。第四个主题是准备实施，包括与资助者和监管者合作、建立可靠和经过验证的措施以及利用现有的患者报告结局认可。
研讨会主题表明支持肾功能、疾病活动度、死亡、生活参与度和心血管疾病，这些被确定为成人肾小球疾病试验的核心结局。未来需要确定每个领域的核心测量方法，资助者和监管者是在试验中采用核心结局集的核心。
