Sydney School of Public Health, University of Sydney, Sydney, Australia; Centre for Kidney Research, Children's Hospital at Westmead, Sydney, Australia.
Sydney School of Public Health, University of Sydney, Sydney, Australia; Centre for Kidney Research, Children's Hospital at Westmead, Sydney, Australia.
Semin Nephrol. 2024 May-Jul;44(3-4):151549. doi: 10.1016/j.semnephrol.2024.151549. Epub 2024 Sep 16.
The outcomes reported in trials across all stages of chronic kidney disease (CKD) are highly variable and often do not include outcomes that are directly relevant to patients and caregivers. Frequently, the outcomes reported in trials are often unvalidated surrogate biochemical end points. The omission of outcomes that are meaningful and important to patients can diminish the value of trials in supporting treatment decisions. In response, there have been increasing efforts across many health and medical disciplines to develop core outcome sets, defined as the minimum set of outcomes to be reported in all trials in a specific health area to improve the relevance and consistency of reporting trial outcomes. The international Standardized Outcomes in Nephrology (SONG) initiative was established in 2014 and has since developed seven core outcome sets for different diagnosis and treatment stages of CKD. The core outcomes were based on consensus among patients, caregivers, and health professionals. Each core outcome set includes at least one patient-reported outcome, including fatigue (hemodialysis), life participation (kidney transplantation, peritoneal dialysis, early CKD not yet requiring kidney replacement therapy, children and adolescents, and glomerular disease), and pain (polycystic kidney disease). This article outlines how patient-reported outcomes are currently reported in trials, discusses core patient-reported outcomes that have been established for trials in kidney disease, and outlines strategies for implementing core patient-reported outcomes in trials.
在慢性肾脏病（CKD）的所有阶段的试验中报告的结果变化很大，并且通常不包括与患者和护理人员直接相关的结果。试验中报告的结果通常是未经证实的替代生化终点。省略对患者有意义和重要的结果会降低试验在支持治疗决策方面的价值。有鉴于此，许多健康和医学学科都在努力制定核心结局集，定义为在特定健康领域的所有试验中都要报告的最小一组结局，以提高试验结果报告的相关性和一致性。国际肾脏病标准化结局（SONG）倡议于 2014 年成立，此后为 CKD 的不同诊断和治疗阶段制定了七个核心结局集。核心结局是基于患者、护理人员和卫生专业人员之间的共识。每个核心结局集都至少包括一个患者报告的结局，包括疲劳（血液透析）、生活参与（肾移植、腹膜透析、早期 CKD 尚未需要肾脏替代治疗、儿童和青少年以及肾小球疾病）和疼痛（多囊肾病）。本文概述了目前在试验中报告患者报告结局的情况，讨论了为肾脏病试验制定的核心患者报告结局，并概述了在试验中实施核心患者报告结局的策略。
