Trustworthiness of randomized trials in endocrinology—A systematic survey

BackgroundTrustworthy (i.e. low risk of bias) randomized clinical trials (RCTs) play an important role in evidence-based decision making. We aimed to systematically assess the risk of bias of trials published in high-impact endocrinology journals.MethodsWe searched the MEDLINE/PubMed database between 2014 and 2016 for phase 2–4 RCTs evaluating endocrine-related therapies. Reviewers working independently and in duplicate used the Cochrane Risk of Bias Tool (CCRBT) to determine the extent to which the methods reported protected the results of each RCT from bias.ResultsWe assessed 292 eligible RCTs, of which 40% (116) were judged to be at low risk, 43% (126) at moderate, and 17% (50) at high risk of bias. Blinding of outcome assessment was the least common domain reported 43% (125), while selective reporting of outcomes was the most common 97% (282). In multivariable analysis, RCTs with a parallel design (OR 2.4; 95% CI; 1.2–4.6) and funded by for-profit sources (OR 2.2; 95% CI; 1.3–3.6) were more likely to be at low risk of bias.ConclusionsTrustworthy evidence should ultimately shape care to improve the likelihood of desirable patient outcomes. Six out-of 10 RCTs published in top endocrine journals are at moderate/high-risk of bias. Improving this should be a priority in endocrine research.**背景**：具备可信性（即偏倚风险（risk of bias）较低）的随机对照试验（randomized clinical trials, RCTs）在循证决策（evidence-based decision making）中发挥着关键作用。本研究旨在系统评估发表于高影响力内分泌学期刊的临床试验的偏倚风险。 **方法**：本研究于2014至2016年间在MEDLINE/PubMed数据库中检索评估内分泌相关治疗的2~4期随机对照试验。由两名独立开展评审的研究者使用Cochrane偏倚风险工具（Cochrane Risk of Bias Tool, CCRBT），对各项随机对照试验报告中用于规避偏倚的方法学质量进行评估。 **结果**：本研究共纳入292项符合入组标准的随机对照试验，其中40%（116项）被评定为偏倚风险较低，43%（126项）为中等风险，17%（50项）为高风险。在偏倚风险评估的各维度中，结局评估设盲的报告率最低，仅为43%（125项）；而结局选择性报告的报告率最高，达97%（282项）。多变量分析（multivariable analysis）结果显示，采用平行设计（parallel design）的随机对照试验（比值比（odds ratio, OR）=2.4；95%置信区间（confidence interval, CI）：1.2~4.6）以及接受营利性资助来源（for-profit sources）资助的随机对照试验（OR=2.2；95%CI：1.3~3.6）更易呈现较低的偏倚风险。 **结论**：可信的循证证据最终应指导临床诊疗实践，以提升患者获得理想结局的概率。在顶级内分泌学期刊发表的随机对照试验中，六成存在中至高偏倚风险。改善此类研究的方法学质量应成为内分泌领域研究的优先事项。