Aster De Vadder, Lotte Lemloh, Bartolomeo Bo, Lennart Hale, Neil Patel, Andreas Mueller, Florian Kipfmueller
Children’s Hospital, University of Bonn. Royal Hospital for Children.
Germany and United Kingdom
European Journal of Pediatrics
Eur J Pediatr 2025; 184:
DOI: 10.1007/s00431-025-06019-6
Abstract
Congenital diaphragmatic hernia (CDH)-associated pulmonary hypertension (PH) is associated with high morbidity and mortality. Pulmonary vasodilative management is challenging and some patients with CDH are unresponsive to inhaled nitric oxide or sildenafil. Bosentan, an enterally-administered endothelin-1 receptor antagonist, reducing pulmonary vascular resistance may play a role in the treatment of CDH-PH. The aim is to evaluate the efficacy and safety of bosentan as an adjunctive therapy for CDH-PH. We report a case series of all CDH neonates who received oral bosentan as an adjunctive therapy for treatment of PH between 2013 and 2021 at our institution. Bosentan was administered at a median enteral dose of 2 mg/kg/day. Main outcomes were improved PH severity on echocardiography, oxygenation, and respiratory support after starting bosentan. Patients were compared according to improvement in PH after 1 week of treatment (responder vs. non-responder). Fifty CDH neonates received oral adjunctive bosentan therapy. Survival to discharge was 58%. Improved PH was observed in 54 and 72% of patients after 1 and 2 weeks respectively (p < 0.001). Respiratory status ameliorated significantly after 2 weeks compared to baseline, with a reduction of ECMO treatment from 30 to 0% and an increase in patients receiving non-invasive or no respiratory support from 18 to 40%. Oxygenation did not improve over 2 weeks, possibly biased by the changes in the respiratory status and other contributing factors to the pathophysiology of CDH.
Conclusion: Bosentan is effective in the treatment of neonates with CDH-PH and was associated with improved PH severity and respiratory status. Adverse effects were minimal and consistent with previous studies.
Category
Class III. Pulmonary Hypertension Associated with Lung Hypoplasia
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