Neonates Effects and Tolerability of Treprostinil in Hypertension with Persistent Pulmonary

Abstract

Objective: The aim of this study was to establish the effects of treprostinil in congenital diaphragmatic hernia (CDH) patients with persistent pulmonary hypertension (PHT) after 1 week of treatment. Drug effects were assessed by oxygenation index (OI), clinical end points, serial biochemical markers, and pre- and posttreatment echocardiogram. Treatment complications were also described.

Study design: This is a quasi-experimental study of neonates with PHT admitted to the NICU within 48 hours showing persistent clinical instability, receiving mechanical ventilation with FiO2 > 60%, milrinone therapy, and inhaled nitric oxide. Clinical data were compared before and after treprostinil treatment.

Results: Seventeen neonates met the inclusion criteria. Median age was 17 days. Before treatment, median OI was 20 (IQR: 12-27). Suprasystemic PHT was estimated by echocardiogram in 8/17 patients; the rest were systemic. After 1 week of treatment, 15/17 patients were alive and median OI was 8 (IQR: 5-12, p = 0.0089). There were no statistically significant changes in laboratory data. Echocardiogram still showed suprasystemic PHT in 20% of patients. Adverse effects included hypotension, hematoma at the infusion site, and surgical persistent ductus arteriosus (PDA) closure in 4/17 patients. Fourteen patients were discharged. The median treatment time was 61 days.

Conclusion: Treprostinil was well tolerated with satisfactory clinical response. Further studies are required to identify early responder subgroups.