Background

RePHyNe is a voluntary disease-based online registry of infants under 3 months of age with pulmonary hypertension (PH) which aims to support high-quality research through the creation of a large international dataset.

Purpose of the Registry

Treatment options for neonatal PH are limited, inadequately studied and most babies are treated with unlicensed or off-label medicines whose safety and efficacy remain unproven. Although neonatal PH is a serious and diverse condition, we still know relatively little about its incidence, basic disease mechanisms, natural history, effective treatment and long-term outcomes. This lack of information contrasts starkly with the wealth of data available in adult and paediatric PH.

Inhaled nitric oxide, the only approved drug for the treatment of PH in term and near-term infants, was first licensed over 20 years ago and there currently are no licensed treatments available for preterm neonates, the population in which PH is most prevalent. In comparison, over 10 new drugs have been approved in adult and paediatric patients with PH. We urgently need new data sources to inform the development and authorisation of treatment for neonatal PH.

A registry is an organised system that uses observational methods to collect uniform data on a population defined by a particular disease, condition or exposure, and that is followed over time. Numerous PH registries have been developed in adult and paediatric populations and have contributed to the successful development of new diagnostic and therapeutic approaches and international consensus guidelines.

RePHyNe is a voluntary international ‘disease-based’ registry that will collect information on newborn babies < 3 months of age (corrected for prematurity) who are diagnosed with PH, either clinically or on specified echocardiographic criteria.

  • Data will be collected prospectively
  • All information will be pseudo-anonymised at source
  • Data will be submitted to the Registry using an online form

Aims and Objectives

  1. To establish an international group of key opinion leaders in neonatal PH
  2. To agree a consensus-based definition of neonatal PH.
  3. To agree a dataset including data fields relating to demographics, diagnostics and therapeutic interventions.
  4. To agree a definitive neonatal PH core outcome set developed using consensus methodology.
  5. To construct a web-based (disease registry) database allowing submission of pseudo-anonymised patient-level data.
  6. To characterise the incidence, demographics, predisposing factors, treatments and clinical course of neonatal PH and the variation in these characteristics by geographical site and subtype of neonatal PH.
  7. To develop classification system based on aetiological diagnosis, clinical and echocardiographic phenotypes.
  8. To study the relationship between various biomarkers (including echocardiographic indices and genetic markers), short-term response to treatment and medium- and long-term outcomes in neonatal PH.
  9. To identify risk factors for mortality and adverse long-term neurodevelopmental outcome in neonatal PH.
  10. To develop a baseline illness severity (risk) score.
  11. To describe the range and trends in treatments offered to patients with neonatal PH.