Descriptive study of the effects of treprostinil and/or epoprostenol in newborn infants with inhaled NO refractory persistent pulmonary hypertension of the newborn (PPHN)

Charlotte Mazepa, Sébastien Mur, Géraldine Gascoind, Laurent Storme, Nicolas Joram, Caroline Viard, Yves Dulac, Marine Butini, Sophie Breinig
Children’s Hospital, CHU de Toulouse and Université de Toulouse. Lille University Hospital. Jeanne-de-Flandre Hospital and CHU de Lille. Nantes University Hospital. Hôpital Femme-Mère-Enfant, Hospices civils de Lyon and Université Claude-Bernard-Lyon.
France

Therapeutics
Therapie 2026;
DOI: 10.1016/j.therap.2026.03.001

Abstract
Background: Epoprostenol and treprostinil, prostacyclin analogues, are used in some neonatal intensive care units (NICU) in the treatment of persistent pulmonary hypertension of the newborn (PPHN) refractory to inhaled nitric oxide (iNO). The aim of this retrospective multicentric descriptive study was to assess the profile and evolution of these newborns.
Methods: Inclusion of neonates born ≥ 34 weeks of gestational age (WGA) and aged ≤ 28 days of life, presenting clinical signs of PPHN (respiratory and/or hemodynamic lability), and at least one echocardiographic sign of pulmonary hypertension. We collected data on the use of prostacyclin analogues, their tolerance, and their efficacy, by collecting clinical, biological, and echocardiographic data of these newborns.
Results: Seventy patients were included from 4 French NICU. It appears that there may be an improvement in pulmonary hypertension (PH) at the initiation of prostacyclin analogues, showing by an increase in post-ductal SpO2, a decrease in FiO2 and an improvement in PH’level on echocardiography. Only four patients needed a treatment discontinuation due to poor hemodynamic tolerance.
Conclusion: Our study highlighted the safety of the use of epoprostenol and treprostinil in neonates suffering from iNO refractory PPHN, without significant adverse effect. It appears to be necessary to carry out further study to establish recommendations for the management of PPHN, and also to define more precisely the place of treprostinil and epoprostenol in this pathology.

Category
Class I. Persistent Pulmonary Hypertension of the Newborn
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects

Age Focus: Pediatric Pulmonary Vascular Disease

Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication

Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: Yes

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