Developing a Neonatal Pulmonary Hypertension Core Outcome Set (NeoPH COS)-A Study Protocol

Area of Science:

• Neonatology
• Pediatric Cardiology
• Clinical Trial Methodology

Background:

• Neonatal pulmonary hypertension (PH) is a rare condition with high mortality.
• Limited evidence-based treatment strategies exist for neonatal PH.
• Inconsistent outcome reporting hinders comparison across studies.

Purpose of the Study:

• To develop a consensus-driven core outcome set (COS) for neonatal PH.
• To standardize outcome reporting in clinical trials for infants with PH.
• To improve the quality of evidence for PH therapies in neonates.

Main Methods:

• International consensus development involving stakeholders (parents, clinicians, researchers).
• Two-stage approach: systematic literature review and qualitative interviews, followed by Delphi survey and consensus meeting.
• Formation of a global advisory group to guide the COS development.

Main Results:

• A core outcome set (COS) for neonatal pulmonary hypertension (PH) is being developed.
• The methodology ensures international applicability and relevance across disciplines.
• The COS aims to enhance trial design and outcome homogeneity.

Conclusions:

• A standardized COS will improve the quality of evidence for neonatal PH treatments.
• This initiative will facilitate better comparison of therapeutic strategies.
• The COS is expected to translate into improved clinical care for newborns with PH.