Project Details

A trial of phosphodiesterase-5 inhibitor in neonatal congenital diaphragmatic hernia (TOP-CDH)

Michelle Yang, MD

Summary

BACKGROUND: Neonates born with congenital diaphragmatic hernia (CDH) have elevated morbidity and mortality secondary to pulmonary hypertension. GAP: Neonates with CDH have increased vasoreactivity leading to pulmonary hypertensive (PH) crises post-hernia repair with cardiorespiratory instability. Administering sildenafil post-operatively may improve cardiorespiratory stability by alleviating PH, subsequently decreasing morbidity. HYPOTHESIS: Neonates with CDH and post-repair PH will have improved left ventricular eccentricity index measured by echocardiogram, after 14 days administration of enteral sildenafil compared to placebo. METHODS: This study is a double-blinded, randomized, placebo controlled trial comparing 14-days of enteral sildenafil citrate 1mg/kg every 8 hours to placebo administration. Neonates managed at Primary Children’s Hospital with echocardiographic evidence of pulmonary hypertension by left ventricular eccentricity index ≥ 1.4 following CDH repair will be approached for enrollment. RESULTS: Pending. IMPACT: Data generated will be used to design an extramurally funded multi-center randomized controlled trial to determine long-term effects of sildenafil therapy for PH in neonates with CDH. This project aims to advance the overall goal of understanding on how to best optimize cardiopulmonary care, leading to improved outcomes for neonates with CDH.