Sydney School of Public Health, The University of Sydney, Sydney, Australia.
Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, Sydney, Australia.
Nephrol Dial Transplant. 2024 Jul 31;39(8):1310-1321. doi: 10.1093/ndt/gfae010.
Many outcomes of high priority to patients and clinicians are infrequently and inconsistently reported across trials in chronic kidney disease (CKD), which generates research waste and limits evidence-informed decision making. We aimed to generate consensus among patients/caregivers and health professionals on critically important outcomes for trials in CKD prior to kidney failure and the need for kidney replacement therapy, and to describe the reasons for their choices.
This was an online two-round international Delphi survey. Adult patients with CKD (all stages and diagnoses), caregivers and health professionals who could read English, Spanish or French were eligible. Participants rated the importance of outcomes using a Likert scale (7-9 indicating critical importance) and a Best-Worst Scale. The scores for the two groups were assessed to determine absolute and relative importance. Comments were analysed thematically.
In total, 1399 participants from 73 countries completed Round 1 of the Delphi survey, including 628 (45%) patients/caregivers and 771 (55%) health professionals. In Round 2, 790 participants (56% response rate) from 63 countries completed the survey including 383 (48%) patients/caregivers and 407 (52%) health professionals. The overall top five outcomes were: kidney function, need for dialysis/transplant, life participation, cardiovascular disease and death. In the final round, patients/caregivers indicated higher scores for most outcomes (17/22 outcomes), and health professionals gave higher priority to mortality, hospitalization and cardiovascular disease (mean difference >0.3). Consensus was based upon the two groups yielding median scores of ≥7 and mean scores >7, and the proportions of both groups rating the outcome as 'critically important' being >50%. Four themes reflected the reasons for their priorities: imminent threat of a health catastrophe, signifying diminishing capacities, ability to self-manage and cope, and tangible and direct consequences.
Across trials in CKD, the outcomes of highest priority to patients, caregivers and health professionals were kidney function, need for dialysis/transplant, life participation, cardiovascular disease and death.
在慢性肾脏病（CKD）的临床试验中，许多对患者和临床医生非常重要的结局指标报告得很少且不一致，这造成了研究浪费，并限制了循证决策。我们旨在让患者/照顾者和卫生专业人员在肾衰竭和需要肾脏替代治疗之前就 CKD 临床试验的关键重要结局达成共识，并描述他们选择这些结局的原因。
这是一项在线两轮国际 Delphi 调查。患有 CKD（所有阶段和诊断）的成年患者、照顾者和能够阅读英语、西班牙语或法语的卫生专业人员有资格参加。参与者使用李克特量表（7-9 表示非常重要）和最佳最差量表对结局的重要性进行评分。评估两组的分数以确定绝对和相对重要性。对评论进行了主题分析。
共有 1399 名来自 73 个国家的参与者完成了 Delphi 调查的第一轮，其中包括 628 名（45%）患者/照顾者和 771 名（55%）卫生专业人员。在第二轮中，来自 63 个国家的 790 名参与者（56%的回复率）完成了调查，其中包括 383 名（48%）患者/照顾者和 407 名（52%）卫生专业人员。总体排名前五的结局为：肾功能、需要透析/移植、生活参与、心血管疾病和死亡。在最后一轮，患者/照顾者对大多数结局的评分较高（22 项结局中有 17 项），而卫生专业人员则更重视死亡率、住院率和心血管疾病（平均差值>0.3）。达成共识的依据是两组的中位数得分均≥7，且两组对该结局的评分均>50%。四个主题反映了他们优先级别的原因：健康灾难迫在眉睫、能力逐渐下降、自我管理和应对能力、以及切实和直接的后果。
在 CKD 的临床试验中，患者、照顾者和卫生专业人员最关心的结局是肾功能、需要透析/移植、生活参与、心血管疾病和死亡。
