Abstract
BackgroundNeonatal encephalopathy is a complex syndrome in infants that predominantly affects the brain and other organs. The leading cause is a lack of oxygen in the blood reaching the brain. Neonatal encephalopathy can result in mortality or complications later in life, including seizures, movement disorders and cerebral palsy. Treatment options for neonatal encephalopathy are limited mainly to therapeutic hypothermia, although other potential treatments are emerging. However, evaluations of the effectiveness of treatments are challenging because of heterogeneity and inconsistency in outcomes measured and reported between trials. In this paper, we detail how we will develop a core outcome set to standardise outcomes measured and reported upon for interventions for the treatment of neonatal encephalopathy.MethodsWe will systematically review the literature to identify outcomes reported previously in randomised trials and systematic reviews of randomised trials. We will identify outcomes important to parents or caregivers of infants diagnosed with and who have received treatment for neonatal encephalopathy. We will do this by conducting in person or by video teleconferencing interviews with parents or caregivers in high-income and low- to middle-income countries. Stakeholders with expertise in neonatal encephalopathy (parents/caregivers, healthcare providers and researchers) will rate the importance of identified outcomes in an online Delphi survey using either a three-round Delphi survey or a “Real-Time” Delphi survey to which stakeholders will be allocated at random. Consensus meetings will take place by video conference to allow for an international group of stakeholder representatives to discuss and vote on the outcomes to include in the final core outcome set (COS).DiscussionMore research is needed on treatments for neonatal encephalopathy. Standardising outcomes measured and reported in evaluations of the effectiveness of interventions for the treatment of neonatal encephalopathy will improve evidence synthesis and improve results reported in systematic reviews and meta-analysis in this area. Overall, this COS will allow for improved treatments to be identified, heterogeneity in research to be reduced, and overall patient care to be enhanced.Trial registrationThis study is registered in the Core Outcome Measures for Effectiveness (COMET) database http://www.comet-initiative.org/Studies/Details/1270.
Highlights
Interventions for the treatment of neonatal encephalopathy vary depending on the underlying cause of encephalopathy
Phase 1: Systematic review Research question: what are the outcomes reported in studies for the treatment of neonatal encephalopathy? We will carry out a systematic review of randomised trials and systematic reviews of randomised trials evaluating the effectiveness of interventions for the treatment of neonatal encephalopathy to identify and collate reported outcomes
Phase 4: Consensus meeting(s) Objective The objective of the consensus meeting is to achieve agreement on the final core outcome set (COS) through an online meeting of international key stakeholders with expertise in neonatal encephalopathy. In this part of the COS development process, we are incorporating a randomised trial to identify whether different outcomes are prioritised when using a Real-Time Delphi method compared with a Multi-Round Delphi method in the development of a COS on interventions for the treatment of neonatal encephalopathy
Summary
Background and objectives 2a2b Scope 3b 3c METHODS StakeholdersDescribe the specific objectives with reference to developing a COS 3aDescribe the intervention(s) that will be covered by the COS Describe the context of use for which the COS is to be appliedInformation sources5b Consensus processDescribe how outcomes may be dropped/combined, with reasonsConsensus definition 7bANALYSIS Outcome scoring/feedback7a Describe the procedure for determining how outcomes will be added/combined/ dropped from consideration during the consensus process Missing data. Neonatal encephalopathy is a complex syndrome in infants that predominantly affects the brain and other organs. We detail how we will develop a core outcome set to standardise outcomes measured and reported upon for interventions for the treatment of neonatal encephalopathy. Neonatal encephalopathy is an umbrella term encompassing a complex neurological syndrome in infants born at 35 weeks of gestation or later (as reported by the American College of Obstetricians and Gynecologists’ Task Force on Neonatal Encephalopathy [1]). Lee et al [2] estimated that, in 2010, approximately 1.15 million infants globally had developed neonatal encephalopathy associated with intrapartum events, of which 96% were born in low- and middle-income countries. Neonatal encephalopathy is associated with early mortality in the newborn and with long-term morbidity, including poor neurodevelopmental outcomes [4,5,6]. Foetal and neonatal risk factors include hypoxic ischaemia, giving rise to hypoxic ischaemic encephalopathy [8, 13], systemic infection, intracranial infections (viral or bacterial) [14], metabolic disorders (including mitochondrial disorders and organic acidaemias) [15], neonatal stroke [16], genetic and epigenetic risk factors [17], intracranial haemorrhage, epileptic syndromes and neurodegenerative disorders [16] among others [3]
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