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Short-Term Treprostinil Use in Infants with Congenital Diaphragmatic Hernia following Repair
Emma Olson1, Leslie A Lusk2, Jeffrey R Fineman1
1Division of Pediatric Critical Care, Department of Pediatrics, Benioff Children's Hospital, University of California San Francisco, San Francisco, CA.
Insights
Treprostinil effectively treated severe pulmonary hypertension in two infants with congenital diaphragmatic hernia. This marks the first report of successful short-term treprostinil use in this neonatal population.
Area of Science:
- Neonatal Medicine
- Pediatric Cardiology
- Pulmonary Hypertension Research
Background:
- Congenital diaphragmatic hernia (CDH) can lead to severe pulmonary hypertension (PH) in neonates.
- Pulmonary hypertension poses a significant mortality risk in infants with CDH.
Observation:
- Two neonates diagnosed with CDH presented with severe pulmonary hypertension at six weeks of age.
- Standard treatments were insufficient to manage the critical condition.
Findings:
- Short-term administration of treprostinil resulted in rapid clinical improvement in both infants.
- Follow-up cardiac catheterization revealed a dramatic reduction in pulmonary hypertension severity.
- Both infants were successfully weaned off treprostinil, indicating a positive treatment response.
Implications:
- This study presents the first documented cases of successful short-term treprostinil therapy for neonatal pulmonary hypertension secondary to CDH.
- Treprostinil may represent a viable therapeutic option for managing severe pulmonary hypertension in neonates with CDH.
- Further research is warranted to establish optimal dosing and long-term outcomes of treprostinil in this population.
Abstract:
We describe 2 infants with congenital diaphragmatic hernia with severe pulmonary hypertension at 6 weeks. Treprostinil was used with rapid clinical improvement. Repeat cardiac catheterization showed dramatic improvement. Both infants were weaned off the drug, representing the first reports of successful short-term treprostinil use in neonates with congenital diaphragmatic hernia.
