Sydney School of Public Health, The University of Sydney, NSW, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, NSW, Australia.
College of Medicine and Public Health, Flinders University, Adelaide, Australia.
Am J Kidney Dis. 2020 Oct;76(4):533-545. doi: 10.1053/j.ajkd.2020.03.014. Epub 2020 Jul 10.
RATIONALE & OBJECTIVE: The inconsistency in outcomes reported and lack of patient-reported outcomes across trials in children with chronic kidney disease (CKD) limits shared decision making. As part of the Standardized Outcomes in Nephrology (SONG)-Kids initiative, we aimed to generate a consensus-based prioritized list of critically important outcomes to be reported in all trials in children with CKD.
An online 2-round Delphi survey in English, French, and Hindi languages.
SETTINGS & PARTICIPANTS: Patients (aged 8-21 years), caregivers/family, and health care professionals (HCPs) rated the importance of outcomes using a 9-point Likert scale (7-9 indicating critical importance) and completed a Best-Worst Scale.
We assessed the absolute and relative importance of outcomes. Comments were analyzed thematically.
557 participants (72 [13%] patients, 132 [24%] caregivers, and 353 [63%] HCPs) from 48 countries completed round 1 and 312 (56%) participants (28 [40%] patients, 64 [46%] caregivers, and 220 [56%] HCPs) completed round 2. Five outcomes were common in the top 10 for each group: mortality, kidney function, life participation, blood pressure, and infection. Caregivers and HCPs rated cardiovascular disease higher than patients. Patients gave lower ratings to all outcomes compared with caregivers/HCPs except they rated life participation (round 2 mean difference, 0.1), academic performance (0.1), mobility (0.4), and ability to travel (0.4) higher than caregivers and rated ability to travel (0.4) higher than HCPs. We identified 3 themes: alleviating disease and treatment burden, focusing on the whole child, and resolving fluctuating and conflicting goals.
Most participants completed the survey in English.
Mortality, life participation, kidney function, and blood pressure were consistently highly prioritized by patients, caregivers, and HCPs. Patients gave higher priority to some lifestyle-related outcomes compared with caregivers/HCPs. Establishing critically important outcomes for all trials in children with CKD may improve consistent reporting of survival, kidney health, and clinical and life impact outcomes that are meaningful for decision making.
儿童慢性肾脏病(CKD)试验报告结果不一致,且缺乏患者报告结局,限制了共同决策。作为肾脏病标准化结局倡议(SONG)-儿童项目的一部分,我们旨在制定一份基于共识的、优先考虑的重要结局清单,以便在所有儿童 CKD 试验中报告。
在英语、法语和印地语中进行了两轮在线德尔菲调查。
患者(8-21 岁)、照顾者/家属和医疗保健专业人员(HCPs)使用 9 分 Likert 量表(7-9 表示至关重要)对结局的重要性进行评分,并完成最佳最差量表。
我们评估了结局的绝对和相对重要性。对评论进行了主题分析。
来自 48 个国家的 557 名参与者(72 名[13%]患者、132 名[24%]照顾者和 353 名[63%] HCPs)完成了第一轮,312 名(56%)参与者(28 名[40%]患者、64 名[46%]照顾者和 220 名[56%] HCPs)完成了第二轮。每组排名前 10 的有 5 个共同的结局:死亡率、肾功能、生活参与度、血压和感染。与患者相比,照顾者和 HCPs 对心血管疾病的评分更高。与照顾者/HCPs 相比,患者对所有结局的评分均较低,但他们对生活参与度(第二轮平均差异为 0.1)、学业表现(0.1)、活动能力(0.4)和旅行能力(0.4)的评分更高,对旅行能力(0.4)的评分高于 HCPs。我们确定了 3 个主题:减轻疾病和治疗负担、关注整个儿童、解决波动和冲突的目标。
大多数参与者以英语完成了调查。
死亡率、生活参与度、肾功能和血压一直是患者、照顾者和 HCPs 高度优先考虑的事项。与照顾者/HCPs 相比,患者对某些与生活方式相关的结局给予了更高的优先级。为所有儿童 CKD 试验确定重要结局可能会改善生存、肾脏健康以及对决策有意义的临床和生活影响结局的一致报告。