How Often Are Patient-Important Outcomes Represented in Neonatal Randomized Controlled Trials? An Analysis of Cochrane Neonatal Reviews
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Updated: Dec 25, 2025

A Novel Method for Involving Women of Color at High Risk for Preterm Birth in Research Priority Setting
Published on: January 12, 2018
Nai Ming Lai1,2, Denise Yin Xian Leom3, Wen Li Chow4
1School of Medicine, Faculty of Health and Medical Sciences, Taylor's University, Kuala Lumpur, Malaysia, lainm@doctors.org.uk.
Background:
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).
Objectives:
We determined the proportion of PIOs in neonatal RCTs included in Cochrane Neonatal reviews.
Methods:
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.
Results:
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).
Conclusions:
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.
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