Neonatal Encephalopathy PhD Training Network, Health Research Board, Dublin, Ireland
Health Research Board -Trials Methodology Research Network (HRB-TMRN), University of Galway, Galway, Ireland.
BMJ Paediatr Open. 2024 Sep 25;8(1):e002510. doi: 10.1136/bmjpo-2024-002510.
Neonatal encephalopathy (NE) is a multi-organ condition potentially leading to death or long-term neurodisability. Therapeutic hypothermia is the standard treatment for NE; however, long-term impairments remain common. Studies of new treatments for NE often measure and report different outcomes. Core outcome sets (COSs), a minimum set of outcomes to be measured and reported in all studies for a condition, address this problem. This paper aimed to identify outcomes reported (primary, secondary, adverse events and other reported outcomes) in (1) randomised trials and (2) systematic reviews of randomised trials of interventions for the treatment of NE in the process of developing a COS for interventions for the treatment of NE.
We completed a systematic search for outcomes used to evaluate treatments for NE using MEDLINE, Embase, Cochrane CENTRAL, the Cochrane Database of Systematic Reviews and the WHO International Clinical Trials Registry Platform. Two reviewers screened all included articles independently. Outcomes were extracted verbatim, similar outcomes were grouped and outcome domains were developed.
386 outcomes were reported in 116 papers, from 85 studies. Outcomes were categorised into 18 domains. No outcome was reported by all studies, a single study reported 11 outcomes and it was not explicitly stated that outcomes had input from parents.
Heterogeneity in reported outcomes means that synthesis of studies evaluating new treatments for NE remains difficult. A COS, that includes parental/family input, is needed to ensure consistency in measuring and reporting outcomes, and to enable comparison of randomised trials.
新生儿脑病（NE）是一种多器官疾病，可能导致死亡或长期神经残疾。治疗性低温是 NE 的标准治疗方法；然而，长期损伤仍然很常见。NE 的新治疗方法的研究通常测量和报告不同的结果。核心结局集（COS）是一种最小的结局集，用于测量和报告一种疾病的所有研究，以解决这个问题。本文旨在确定在为 NE 治疗干预措施制定 COS 的过程中，（1）随机试验和（2）随机试验系统评价中报告的结局（主要、次要、不良事件和其他报告结局）。
我们使用 MEDLINE、Embase、Cochrane CENTRAL、Cochrane 系统评价数据库和世界卫生组织国际临床试验注册平台，对用于评估 NE 治疗的干预措施的结局进行了系统搜索。两名评审员独立筛选所有纳入的文章。逐字提取结局，将相似的结局进行分组，并开发结局领域。
从 85 项研究的 116 篇论文中报告了 386 个结局。结局分为 18 个领域。没有一个结局被所有研究报告，一项研究报告了 11 个结局，也没有明确说明结局是来自父母的意见。
报告结局的异质性意味着评估 NE 新治疗方法的研究综合仍然很困难。需要一个包括父母/家庭意见的 COS，以确保在测量和报告结局方面的一致性，并能够比较随机试验。
