Core outcomes in neonatology: development of a core outcome set for neonatal research

Background
Neonatal research evaluates many different outcomes using multiple measures. This can prevent synthesis of trial results in meta-analyses, and selected outcomes may not be relevant to former patients, parents and health professionals.

Objective
To define a core outcome set (COS) for research involving infants receiving neonatal care in a high-income setting.

Design
Outcomes reported in neonatal trials and qualitative studies were systematically reviewed. Stakeholders were recruited for a three-round international Delphi survey. A consensus meeting was held to confirm the final COS, based on the survey results.

Participants
Four hundred and fourteen former patients, parents, healthcare professionals and researchers took part in the eDelphi survey; 173 completed all three rounds. Sixteen stakeholders participated in the consensus meeting.

Results
The literature reviews identified 104 outcomes; these were included in round 1. Participants proposed 10 additional outcomes; 114 outcomes were scored in rounds 2 and 3. Round 1 scores showed different stakeholder groups prioritised contrasting outcomes. Twelve outcomes were included in the final COS: survival, sepsis, necrotising enterocolitis, brain injury on imaging, general gross motor ability, general cognitive ability, quality of life, adverse events, visual impairment/blindness, hearing impairment/deafness, retinopathy of prematurity and chronic lung disease/bronchopulmonary dysplasia.

Conclusions and relevance
A COS for clinical trials and other research studies involving infants receiving neonatal care in a high-income setting has been identified. This COS for neonatology will help standardise outcome selection in clinical trials and ensure these are relevant to those most affected by neonatal care.

Contributors

James William Harrison Webbe1, James M N Duffy2, Elsa Afonso3, Iyad Al-Muzaffar4, Ginny Brunton5, Anne Greenough6, Nigel J Hall7, Marian Knight8, Jos M Latour9,10, Caroline Lee-Davey11, Neil Marlow12, Laura Noakes13, Julie Nycyk14, Angela Richard-Löndt13, Ben Wills-Eve15, Neena Modi16, Chris Gale1

Author affiliations

1 Academic Neonatal Medicine, Imperial College London, London, UK
2 Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, Oxfordshire, UK
3 Neonatal Unit, Rosie Hospital, Cambridge, Cambridgeshire, UK
4 The Neonatal Unit, Royal Glamorgan Hospital, Llantrisant, Rhondda Cynon Taf, UK
5 UCL Institute of Education Centre for Longitudinal Studies, London, UK
6 Department of Women and Children's Health, School of Life Sciences, Faculty of Life Sciences and Medicine, King's College London, London, UK
7 Paediatric Surgery, Southampton General Hospital, Southampton, UK
8 National Perinatal Epidemiology Unit, Oxford, UK
9 School of Nursing and Midwifery, Faculty of Health, Education and Society, Plymouth University, Plymouth, Devon, UK
10 School of Nursing and Midwifery, Faculty of Health Sciences, Curtin University, Perth, Western Australia, Australia
11 Bliss, London, UK
12 Institute for Women's Health, University College London, London, UK
13 Parent of Neonatal Patient, London, UK
14 Neonatal Unit, Birmingham City Hospital, Birmingham, UK
15 Former neonatal patient, London, UK
16 Neonatal Medicine, Imperial College London, London, UK

Publication

Journal: Archives of Disease in Childhood: Fetal and Neonatal Edition
Volume: 9
Issue: 11
Pages: -
Year: 2019
DOI: 10.1136/archdischild-2019-317501

Further Study Information

Current Stage: Not Applicable
Date: January 2016 - March 2018
Funding source(s): This study was funded as part of a Medical Research Council (MRC) Clinician Scientist Fellowship awarded to CG (MR/N008405/1), salary support for JWHW from the Portland Hospital and Mason Medical Research Foundation, and unrestricted funds held by NMo. NMa receives part funding from the Department of Health’s NIHR Biomedical Research Centre’s funding scheme at UCLH/UCL.


Health Area

Disease Category: Neonatal care

Disease Name: Neonatal care

Target Population

Age Range: 0 - 0

Sex: Either

Nature of Intervention: Any

Stakeholders Involved

- Charities
- Clinical experts
- Consumers (caregivers)
- Families
- Patient/ support group representatives
- Researchers
- Consumers (patients)

Study Type

- COS for clinical trials or clinical research

Method(s)

- Delphi process
- Systematic review
- Consensus meeting

Outcomes reported in neonatal trials and qualitative studies were systematically reviewed. Stakeholders were recruited for a three-round international Delphi survey. A consensus meeting was held to confirm the final COS, based on the survey results.