School of Medicine, Faculty of Health and Medical Sciences, Taylor's University, Kuala Lumpur, Malaysia,
School of Pharmacy, Monash University, Kuala Lumpur, Malaysia,
Neonatology. 2020;117(4):428-435. doi: 10.1159/000506703. Epub 2020 Mar 25.
Research findings based on patient-important outcomes (PIOs) provide more useful conclusions than those that are based on surrogate outcomes. It is unclear to what extent PIOs are represented in neonatal randomized controlled trials (RCTs).
We determined the proportion of PIOs in neonatal RCTs included in Cochrane Neonatal reviews.
We extracted up to 5 outcomes from each RCT included in Cochrane Neonatal reviews published until January 2018, with independent determination of PIOs among authors followed by a discussion leading to a consensus. We defined PIOs as outcomes that matter to patient care, such as clinical events or physiological or laboratory parameters that are widely used to guide management.
Among 6,832 outcomes extracted from 1,874 RCTs included in 276 reviews, 5,349 (78.3%) were considered PIOs; 461 studies (24.5%) included 5 or more PIOs, 1,278 (68.2%) included 1-4 PIOs, while 135 (7.2%) had no PIO included. PIOs were observed more often among dichotomous than among continuous outcomes (94.9 vs. 61.5%; RR: 1.54; 95% CI: 1.50-1.58), and more among subjective than among objective outcomes (95.9 vs. 76.8%; RR: 1.25; 95% CI: 1.22-1.28). Newer studies were more likely to have a greater number of PIOs (adjusted OR: 1.033 [95% CI: 1.025-1.041] with each publication year).
The large and increasing representation of PIOs over the years suggests an improving awareness by neonatal trialists of the need to incorporate important outcomes in order to justify the utilization of resources. Further research should explore the reasons for non-inclusion or non-reporting of PIOs in a small proportion of RCTs.
基于患者重要结局（PIO）的研究结果比基于替代结局的研究结果提供了更有用的结论。目前尚不清楚 PIO 在新生儿随机对照试验（RCT）中的代表性程度如何。
我们确定了 Cochrane 新生儿综述中包含的新生儿 RCT 中 PIO 的比例。
我们从截至 2018 年 1 月发表的 Cochrane 新生儿综述中包含的每项 RCT 中提取了最多 5 个结局，作者独立确定 PIO 后进行讨论，达成共识。我们将 PIO 定义为对患者护理有意义的结局，例如广泛用于指导治疗的临床事件或生理或实验室参数。
从 276 项综述中包含的 1874 项 RCT 中提取的 6832 个结局中，有 5349 个（78.3%）被认为是 PIO；461 项研究（24.5%）包含 5 个或更多 PIO，1278 项研究（68.2%）包含 1-4 个 PIO，而 135 项研究（7.2%）没有包含任何 PIO。二分类结局比连续结局更常见 PIO（94.9%比 61.5%；RR：1.54；95%CI：1.50-1.58），主观结局比客观结局更常见 PIO（95.9%比 76.8%；RR：1.25；95%CI：1.22-1.28）。较新的研究更有可能包含更多的 PIO（调整后的 OR：1.033 [95%CI：1.025-1.041]，每年增加 1 项研究）。
多年来 PIO 的大量和不断增加的代表性表明，新生儿试验者越来越意识到需要纳入重要结局，以证明资源的利用是合理的。进一步的研究应该探讨为什么在一小部分 RCT 中没有纳入或没有报告 PIO。
