Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, Sydney, Australia; University Francois Rabelais, Tours, France; Department of Nephrology and Clinical Immunology, Tours Hospital, Tours, France; INSERM, U1246, Tours, France.
Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Westmead, Sydney, Australia.
Am J Kidney Dis. 2018 Jul;72(1):62-74. doi: 10.1053/j.ajkd.2017.11.010. Epub 2018 Feb 21.
Clinical trials are most informative for evidence-based decision making when they consistently measure and report outcomes of relevance to stakeholders. We aimed to assess the scope and consistency of outcomes reported in trials for hemodialysis.
Systematic review.
SETTING & POPULATION: Adults requiring maintenance hemodialysis enrolled in clinical trials.
All Cochrane systematic reviews of interventions published by August 29, 2016, and the trials published and registered in ClinicalTrials.gov since January 2011.
Any hemodialysis-related interventions.
Frequency and characteristics of the reported outcome domains and measures.
From the 362 trials, we extracted and classified 10,713 outcome measures (a median of 21 [IQR, 10-39] per trial) into 81 different outcome domains, of which 42 (52%) were surrogate; 25 (31%), clinical; and 14 (17%), patient reported. The number of outcome measures reported significantly changed over time. The 5 most commonly reported domains were all surrogates: phosphate (125 [35%] trials), dialysis adequacy (120 [33%]), anemia (115 [32%]), inflammatory markers (114 [31%]), and calcium (109 [30%]). Mortality, cardiovascular diseases, and quality of life were reported very infrequently (73 [20%], 44 [12%], and 32 [9%], respectively).
For feasibility, we included a sampling frame that included only trials identified in Cochrane systematic reviews or ClinicalTrials.gov.
Outcomes reported in clinical trials involving adults receiving hemodialysis are focused on surrogate outcomes, rather than clinical and patient-centered outcomes. There is also extreme multiplicity and heterogeneity at every level: domain, measure, metric, and time point. Estimates of the comparative effectiveness of available interventions are unreliable and improvements over time have been inconsistent.
临床试验在基于证据的决策中最具信息性，当它们始终一致地测量和报告与利益相关者相关的结果时。我们旨在评估血液透析临床试验报告结果的范围和一致性。
系统评价。
需要维持性血液透析的成年患者参加临床试验。
截至 2016 年 8 月 29 日发布的所有 Cochrane 系统评价干预措施，以及自 2011 年 1 月以来在 ClinicalTrials.gov 上发布和注册的试验。
任何与血液透析相关的干预措施。
从 362 项试验中，我们提取并分类了 10713 项结果测量指标（中位数为每个试验 21[IQR，10-39]），分为 81 个不同的结果领域，其中 42 个（52%）是替代指标；25 个（31%）为临床指标；14 个（17%）为患者报告指标。报告的结果测量指标数量随时间显著变化。报告最多的 5 个领域都是替代指标：磷酸盐（125[35%]个试验）、透析充分性（120[33%]个）、贫血（115[32%]个）、炎症标志物（114[31%]个）和钙（109[30%]个）。死亡率、心血管疾病和生活质量报告非常少见（分别为 73[20%]、44[12%]和 32[9%]）。
为了可行性，我们只包括了在 Cochrane 系统评价或 ClinicalTrials.gov 中确定的试验抽样框架。
涉及接受血液透析的成年患者的临床试验报告的结果主要集中在替代结果上，而不是临床和以患者为中心的结果。在每个层面，即领域、测量指标、度量和时间点，都存在着极端的多样性和异质性。现有干预措施的相对有效性估计是不可靠的，而且随着时间的推移，改进情况也不一致。
