Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, Australia.
Sydney School of Public Health, The University of Sydney, Sydney, Australia; Centre for Kidney Research, The Children's Hospital at Westmead, Sydney, Australia; University Francois Rabelais, Tours, France; Department of Nephrology and Clinical Immunology, Tours Hospital, Tours, France; INSERM (U1153), Paris, France.
J Pediatr. 2017 Jul;186:110-117.e11. doi: 10.1016/j.jpeds.2017.03.034. Epub 2017 Apr 24.
To determine the range and heterogeneity of outcomes reported in randomized controlled trials of interventions for children with chronic kidney disease (CKD).
The Cochrane Kidney and Transplant Specialized Register was searched to March 2016. Randomized trials involving children across all stages of CKD were selected. All outcome domains and measurements were extracted from included trials. The frequency and characteristics of the outcome domains and measures were evaluated.
From 205 trials included, 6158 different measurements of 100 different outcome domains were reported, with a median of 22 domains per trial (IQR 13-41). Overall, 52 domains (52%) were surrogate, 38 (38%) were clinical, and 10 (10%) were patient-reported. The 5 most commonly reported domains were blood pressure (76 [37%] trials), relapse/remission (70 [34%]), kidney function (66 [32%]), infection (61 [30%]), and height/pubertal development (51 [25%]). Mortality (14%), cardiovascular disease (4%), and quality of life (1%) were reported infrequently. The 2 most frequently reported outcomes, blood pressure and relapse/remission, had 56 and 81 different outcome measures, respectively.
The outcomes reported in clinical trials involving children with CKD are extremely heterogeneous and are most often surrogate outcomes, rather than clinical and patient-centered outcomes such as cardiovascular disease and quality of life. Efforts to ensure consistent reporting of outcomes that are important to patients and clinicians will improve the value of trials to guide clinical decision-making. In our study, non-English articles were excluded.
确定针对慢性肾脏病（CKD）儿童干预措施的随机对照试验报告的结局范围和异质性。
截至 2016 年 3 月，对 Cochrane 肾脏病和移植专题登记库进行检索。选择纳入各阶段 CKD 儿童的随机试验。从纳入的试验中提取所有结局领域和测量值。评估结局领域和措施的频率和特征。
在 205 项试验中，报告了 100 个不同结局领域的 6158 种不同测量值，中位数为每个试验 22 个领域（IQR 13-41）。总体而言，52 个领域（52%）为替代指标，38 个（38%）为临床结局，10 个（10%）为患者报告结局。报告最多的 5 个领域是血压（76 [37%] 项试验）、复发/缓解（70 [34%]）、肾功能（66 [32%]）、感染（61 [30%]）和身高/青春期发育（51 [25%]）。死亡率（14%）、心血管疾病（4%）和生活质量（1%）报告较少。报告最多的两个结局，血压和复发/缓解，分别有 56 和 81 种不同的结局测量值。
涉及 CKD 儿童的临床试验报告的结局非常异质，且多为替代结局，而非心血管疾病和生活质量等临床和以患者为中心的结局。努力确保一致报告对患者和临床医生重要的结局，将提高试验指导临床决策的价值。在我们的研究中，排除了非英文文章。
