Safety and Tolerance of Prostaglandin E1 Therapy in Neonates with Duct-Dependent Congenital Heart Disease: A 10-Year Retrospective Study

Abstract

Introduction: Congenital heart diseases (CHD) are the most common birth defects. Duct-dependent lesions require prostaglandin E1 (PGE1) to maintain ductus arteriosus patency. While life-saving, PGE1 is associated with significant side effects. This study aimed to evaluate the safety and tolerance profile of PGE1 therapy in neonates with ductal-dependent CHD in a real-life neonatal intensive care setting. Methods: A retrospective, descriptive, single-center study was conducted, including 63 newborns with duct-dependent CHD who received PGE1 in the Neonatal Intensive Care Department of Monastir between January 2014 and December 2023. Safety was assessed by the incidence, type, and severity of adverse events clinically attributed to PGE1. Results: The hospital prevalence of duct-dependent CHD was 0.2%. The overall mortality rate was 51%, with 65% of deaths occurring preoperatively. Only 33% of cases had an antenatal diagnosis. PGE1 infusion was started at a mean age of 2.4 days. The mean initial and optimal maintenance doses were 0.067 mcg/kg/min and 0.028 mcg/kg/min, respectively. Adverse effects were frequent, with 85% of patients experiencing at least one event. The most common non-serious events were irritability (22%), fever (17%), and cutaneous flushing (16%). Serious adverse events included apnea (10%), hypotension (13%), and convulsions (5%). Conclusion: PGE1 is an indispensable but high-risk therapy. Our findings highlight a substantial burden of adverse effects, even with low-dose regimens, and a critically high preoperative mortality. This underscores systemic challenges beyond drug safety alone. Improving outcomes necessitates a multifaceted strategy: enhanced antenatal screening, implementation of standardized PGE1 protocols to minimize risks, and optimized surgical pathways to reduce critical delays.