Question What standardized set of outcomes should be measured and reported in all clinical trials and research studies on neonatal sepsis treatments?
Findings A core outcome set (COS) for neonatal sepsis treatments was created with the involvement of diverse stakeholders. The COS includes 9 key outcomes: all-cause mortality, need for mechanical ventilation, brain injury on imaging, neurologic status at discharge, escalation of antimicrobial therapy, central nervous system infections, multiorgan dysfunction, neurodevelopmental impairment, and quality of life of parents.
Meaning This COS will help to standardize what sepsis outcomes are measured and reported and therefore improve the potential for synthesis across research in this field.
Abstract
Importance Sepsis is one of the leading causes of neonatal mortality. There is heterogeneity in the outcomes measured and reported in studies of neonatal sepsis. To address this challenge, a core outcome set (COS) for research on neonatal sepsis was needed.
Objective The Neonatal Sepsis Core Outcome Set (NESCOS) project aims to develop a COS for research evaluating the effectiveness of neonatal sepsis treatments.
Evidence Review For this consensus statement, the research team obtained ethics approval and used a 4-stage process: (1) a systematic review of qualitative studies, (2) a real-time Delphi (RTD) survey to identify important outcomes for consensus meetings, (3) consensus meetings to finalize the COS, and (4) dissemination of the findings. The study was conducted from May 2, 2022, to October 27, 2023. The steering group and project participants consisted of health care workers, researchers, academics, parents, and parent representatives from low-, middle-, and high-income countries. An RTD survey and consensus meetings were conducted, with measures including a 9-point Likert scale rating (where 1 indicated not at all important and 9 indicated critically important) for outcome importance and a minimum 80% agreement threshold among stakeholders for final COS inclusion. The systematic review identified 19 outcomes, which were combined with outcomes from previous systematic reviews of clinical trials.
Findings The RTD survey included 306 participants, leading to the identification of 55 outcomes for further discussion in consensus meetings. The finalized COS comprises 9 outcomes: all-cause mortality, need for mechanical ventilation, brain injury on imaging, neurologic status at discharge, escalation of antimicrobial therapy, central nervous system infections, multiorgan dysfunction, neurodevelopmental impairment, and quality of life of parents.
Conclusions and Relevance This consensus-based COS for research on neonatal sepsis treatments will help standardize the outcomes measured and reported, enhancing the comparability of research findings. Future efforts should focus on establishing standardized and reliable methods for measuring these outcomes.
Petek Eylul Taneri, MD, PhD1,2; Linda Biesty, PhD2,3; Jamie J. Kirkham, PhD4; Eleanor J. Molloy, MD, PhD5,6,7,8; Richard A. Polin, MD9; Aoife Branagan, MSc5,6; Kondwani Kawaza, MD, PhD10; Mandy Daly11; James L. Wynn, MD12; Lívia Nagy Bonnard13; Fiona A. Quirke, PhD14; Niranjan Kissoon, MBBS15,16; Magdalena Ohaja, PhD2; Kateregga Bazilio17; Eric Giannoni, PhD18; Luregn J. Schlapbach, MD, PhD19,20; Denise Suguitani21; Tobias Strunk, MD, PhD22; Barbara J. Stoll, MD23,24; Declan Devane, PhD1,2,3
Disease Category: Neonatal care
Disease Name: Sepsis
Age Range: 0 - 1
Sex: Either
Nature of Intervention: Any
- Clinical experts
- Families
- Patient/ support group representatives
- Researchers
- COS for clinical trials or clinical research
- COS for practice
- Consensus meeting
- Delphi process
- Literature review
- Systematic review
The study will be conducted in four stages: (1) systematic review, (2) Delphi survey (3) consensus meetings and (4) dissemination and implementation strategy.