Development and Content of Core Outcome Sets in Neonatology-A Scoping Review.

For this scoping review, we searched PubMed (MEDLINE), Web of Science and Scopus databases on April 1, 2025. The complete search strategy is provided in the Supporting Information . The search results were uploaded to Covidence software for screening. Two authors (IK and MH) independently screened the abstracts. Disagreements were resolved by consensus initially, with resolution using a third author (PK) when required. Two authors (IK and PK) screened the full reports in a similar manner. The reference lists of included articles were searched for additional eligible articles. We did not search the grey literature (conference abstracts and other non‐peer reviewed publications). We included all consensus studies developing core outcome sets for neonates regardless of the methodology. We defined a neonate as an infant less than 28 days of age. The COS study was included if it had clear domains relevant to the newborn infant. Thus, studies in which the primary aim was to assess maternal health or complications during pregnancy, but which reported neonatal outcomes, were included. We excluded studies which did not clearly develop a COS. For example, studies that described outcomes of published trials or conducted interviews on outcome importance, but did not present clear COS, were excluded. We sought to identify (1) clinical scenarios for which COS were developed and (2) the most common proposed outcomes. We used descriptive statistics to characterise the COS, the methods used to develop them, and the participants who were included in the process. Two authors screened abstracts and full text articles. Covidence software was used in the screening and extraction process. In cases of disagreement, the two reviewers sought consensus. A third reviewer was used in cases of persisting disagreements. Two authors (IK and MH) performed data extraction independently. The following information was extracted: authors, year of publication, outcome set development methods, participants and results. This study has been reported according to the Preferred Reporting Items in Systematic Reviews and Meta‐Analyses—Scoping Review extension guideline [ 12 ]. The protocol was prospectively published in Open Science Framework and is available from: https://osf.io/yngbd/ .

A total of 34 publications, published between 2016 and 2025, were included in the final analysis [ 8 , 9 , 10 , 11 , 13 , 14 , 15 , 16 , 17 , 18 , 19 , 20 , 21 , 22 , 23 , 24 , 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 ]. (Figure  S1 ) They used mostly modified Delphi and Delphi consensus methods, and the median number of rounds was three (IQR 2–3) (Table  1 ) All but one of the COS were based on a systematic literature search to identify possible outcomes, and all 34 COS held official final consensus meetings. The median number of participants was 128. All the studies that reported the participant characteristics included neonatologists and parent representatives (Table  1 ). Characteristics of the included core outcome set development studies. The majority of the reports encompassed both mothers' and infants' outcomes ( n  = 24), reflecting that most interventions took place during pregnancy. The 34 COS included and covered six separate themes (Table  2 ). Some of the COS were extremely specific, such as vasa previa or twin‐to‐twin transfusion syndrome COS, whereas there were COS for overall pregnancy, labour and neonatal outcomes (Table  2 ). These 34 COS proposed a total of 62 distinct neonatal outcomes (Table  S1 ). The median number of proposed outcomes per COS was eight, ranging from four to nineteen (Table  1 ) The most common outcomes were neonatal mortality, preterm birth, birth weight and NICU admission (Figure  1 ) The main themes for outcomes are presented in Table  3 . Core outcome sets classified into subgroups based on the topic. The 10 most common outcomes included in the 34 core outcome sets. Outcomes classified into thematic subgroups. Broncho‐ pulmonary dysplasia (BPD) Central‐nervous‐ system infection

core outcome set neonatal intensive care unit

The main strength was the systematic search and data extraction process which improved the reliability of this study. The main limitation is the lack of comparison of what were important outcomes for the different stakeholder groups, as the studies included rarely separated the results for these groups. Future COS studies should report results stratified by these subgroups in order to better understand the relative importance of different outcomes to different stakeholders.

This scoping review identified 34 COS that included outcomes for neonates. The most commonly proposed outcomes were neonatal mortality, preterm birth, birth weight, NICU admission and neurodevelopment. In 2020, an overall COS for neonatal care in high‐income countries proposed 12 core outcomes. Interestingly, the four stakeholder groups (patients and parents, nurses and therapists, doctors, and researchers) all ranked three outcomes (mortality, necrotizing enterocolitis, and sepsis) in their top four [ 8 ]. Unfortunately, the majority of the other included COS did not report the outcome rankings by stakeholder group, which would have been valuable for evaluating potential differences in preferences. A previous study has found that parents of neonates may rank outcomes differently than clinicians [ 43 ]. A more recent survey found that parents valued quality of life measures higher than health complications [ 44 ]. The aim of a core outcome set is to improve the standardisation of outcomes selected for future studies and systematic reviews. However, a persisting challenge in neonatal research is the lack of consistent outcome definitions, which reflects both the complexity of neonatal conditions and the ongoing development of clinical understanding and consensus [ 45 ]. Many of the presented outcomes presented in the included COS were either overly broad—such as ‘adverse events’, or ‘quality of life’, which can be assessed using various methods—or inconsistently defined, as in the case of bronchopulmonary dysplasia and sepsis [ 46 , 47 ]. Some COS addressed this issue by also providing explicit definitions and recommended measurement tools to enhance clarity and comparability [ 42 ]. As all the COS included were based on the COMET initiative, the methods were comparable. The majority used outcomes extracted from published studies and conducted a Delphi or a modified Delphi process consisting of 2–3 rounds followed by a final consensus meeting. Parents were included in the COS process in all studies, and the majority also had other stakeholders such as researchers, policy makers, and regulators involved. A key challenge for COS initiatives has been that, despite the increasing number of published sets, their consistent implementation in clinical research and practice remains limited [ 6 ].

We found that the neonatal core outcome sets were heterogeneous in terms of development and selected outcomes. The COS mostly used a modified Delphi process with 3 rounds and consensus meetings. The most proposed core outcomes were neonatal mortality, gestational age, birth weight, and need for NICU admission. Future studies and reviews should focus on including outcomes that are important to clinicians and families in order to improve the consistency and relevance of the reporting.

Clinical trials seek to identify safe and effective therapies by analysing outcomes that are important to patients, families and health care professionals. The variability in reported outcomes across clinical trials and observational studies presents a major challenge to synthesising evidence and translating findings into clinical practice. The COMET initiative was launched to improve reporting by developing core outcome sets (COS) [ 1 ]. COS offer a standardised approach to outcome selection and reporting, ensuring that studies measure and report outcomes that are relevant, comparable, and meaningful to clinicians, researchers, and families [ 2 , 3 , 4 , 5 ]. Overall, the uptake of COS to clinical studies has been inconsistent [ 6 ]. In neonatal studies, the use of COS is especially important due to the diverse nature of neonatal morbidities and the long‐term implications of early‐life interventions. Furthermore, the outcomes measured in clinical studies have not always aligned with those most valued by families [ 7 ]. Examples include cranial ultrasound findings with unclear long‐term significance and the need for supplemental oxygen at a single time‐point (e.g., 36 + 0 weeks of gestation). An overall COS for neonatal intensive care unit care in high‐income countries was developed and published in 2020 and included 12 outcomes: 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 [ 8 ]. COS for specific health issues including pre‐eclampsia, congenital abnormalities and neonatal sepsis have been published in recent years [ 9 , 10 , 11 ]. The aim of this scoping review was to identify neonatal patient groups for whom COS have been developed. Secondary aims included the identification of the outcomes which were included and the processes by which these COS were developed.

The authors declare no conflicts of interest.

Data S1. Appendix S1.