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Published on: August 4, 2021
Subcutaneous treprostinil for pulmonary hypertension in chronic lung disease of infancy
Dina J Ferdman1, Erika B Rosenzweig1, Warren A Zuckerman1
1Division of Pediatric Cardiology, Department of Pediatrics, Columbia University Medical Center, College of Physicians and Surgeons, New York, New York.
Insights
Subcutaneous treprostinil offers a safe and effective alternative for treating severe pulmonary arterial hypertension (PAH) in infants with chronic lung disease. This approach improved cardiac function and reduced respiratory support needs without significant side effects.
Area of Science:
- Pediatrics
- Cardiology
- Neonatology
Background:
- Infants with chronic lung disease can develop severe pulmonary arterial hypertension (PAH).
- Current intravenous prostanoid therapy for severe PAH in infants has complications due to central venous access.
- There is a need for improved delivery of targeted PAH therapies in this vulnerable population.
Purpose of the Study:
- To evaluate the safety and efficacy of continuous subcutaneous treprostinil infusion in infants with severe PAH and chronic lung disease.
- To assess the impact of subcutaneous treprostinil on right ventricular function, pulmonary hypertension, and respiratory support requirements.
Main Methods:
- A case series of 5 former extremely preterm infants with severe PAH secondary to chronic lung disease was retrospectively reviewed.
- Infants received continuous subcutaneous treprostinil infusion.
- Echocardiographic assessments and respiratory support needs were evaluated before and after treatment initiation.
Main Results:
- All 5 infants showed improvements in echocardiographic measures of right ventricular function and estimated pulmonary hypertension.
- Respiratory support requirements decreased within weeks of initiating subcutaneous treprostinil therapy.
- No severe site erythema, bleeding, bruising, or infection occurred, unlike common side effects in adults.
Conclusions:
- Continuous subcutaneous treprostinil infusion is a safe, efficacious, and well-tolerated treatment option for select former premature infants with severe PAH and chronic lung disease.
- This alternative delivery method may be beneficial for infants unresponsive to conventional therapies, avoiding central venous catheter complications.
Abstract:
Pulmonary arterial hypertension (PAH) associated with chronic lung disease of infancy can be a life-threatening disease affecting an increasing number of former premature infants. There is a need for improved delivery of targeted PAH therapies for this subgroup of patients who have severe and persistent PAH despite standard respiratory care for chronic lung disease. Currently infants who have severe PAH despite oral or inhaled therapy receive continuous intravenous prostanoid therapy (mostly epoprostenol), which is complicated because of the need for central venous access and associated catheter-related complications. We present a series of 5 infants who were successfully treated with a continuous infusion of subcutaneous treprostinil, which is a longer-acting prostanoid with similar hemodynamic effects. There were improvements in echocardiographic assessment of right ventricular function and estimated pulmonary hypertension, and in respiratory support required within weeks of therapy. Unlike commonly in adults, these 5 infants had no instances of severe site erythema, bleeding, bruising, or infection. In our experience with 5 former extremely preterm infants who had PAH associated with chronic lung disease, subcutaneous treprostinil was safe, efficacious, and well tolerated. We believe that subcutaneous treprostinil can be beneficial in a select group of former premature infants who have chronic lung disease and severe pulmonary arterial hypertension who have not responded adequately to conservative therapies.
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