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Neonates Effects and Tolerability of Treprostinil in Hypertension with Persistent Pulmonary
Mariela Jozefkowicz1, Dora Fabiana Haag2, María Teresa Mazzucchelli1
1Neonatal Intensive Care Unit, Children's Hospital SAMIC Prof. Dr. Juan P. Garrahan, Buenos Aires, Argentina.
Insights
Treprostinil treatment improved oxygenation in congenital diaphragmatic hernia (CDH) patients with persistent pulmonary hypertension (PHT). The drug was well-tolerated, showing satisfactory clinical response in neonates with PHT.
Area of Science:
- Neonatal Medicine
- Pediatric Cardiology
- Pulmonary Hypertension
Background:
- Congenital diaphragmatic hernia (CDH) can lead to persistent pulmonary hypertension (PHT) in neonates.
- PHT in CDH patients presents significant clinical challenges and requires effective treatment strategies.
Purpose of the Study:
- To evaluate the efficacy and safety of treprostinil in neonates with CDH and PHT.
- To assess the impact of treprostinil on oxygenation index (OI) and clinical outcomes.
Main Methods:
- A quasi-experimental study involving 17 neonates with PHT.
- Treprostinil treatment was administered to mechanically ventilated neonates receiving other therapies.
- Clinical data, including OI and echocardiograms, were compared before and after 1 week of treprostinil treatment.
Main Results:
- After 1 week, 15/17 patients survived, with a significant reduction in median OI (20 to 8, p=0.0089).
- Echocardiograms showed persistent suprasystemic PHT in 20% of patients post-treatment.
- Adverse effects included hypotension and PDA closure in 4/17 patients; treprostinil was generally well-tolerated.
Conclusions:
- Treprostinil demonstrated a satisfactory clinical response and improved oxygenation in CDH patients with PHT.
- Further research is needed to identify patient subgroups who respond best to early treprostinil treatment.
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.
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