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Published on: June 27, 2025
Quantifying side effects and caregiver burdens of pediatric pulmonary hypertension therapies
Erik J Nelson1, Ella Cook2, Samara Nelson3
1Department of Public Health, Brigham Young University, 2148 LSB, Provo, UT, 84660, USA. Erik_nelson@byu.edu.
Insights
Pediatric pulmonary hypertension (PH) treatments cause significant side effects and family burdens. Research is needed for labeled therapies to improve care for children with PH.
Area of Science:
- Pediatric pulmonology
- Pharmacology
- Family medicine
Background:
- Pulmonary hypertension (PH) is a serious pediatric condition, but treatments are primarily evaluated in adults.
- Pediatric PH therapies are often used off-label, necessitating further investigation.
Purpose of the Study:
- To assess side effect profiles of common pediatric PH treatments.
- To understand the burdens on families caring for children with PH.
Main Methods:
- Online survey distributed through a Facebook group for families of children with PH.
- 139 parents completed the survey regarding PH treatments and care access.
Main Results:
- Most children used multiple medications; Treprostinil, Selexipag, and PDE5 inhibitors were associated with the most side effects.
- Common side effects include skin flushing, headache, nasal congestion, pain, and nausea.
- Significant travel burdens exist, with most families traveling over 20 miles and for at least 60 minutes for care.
Conclusions:
- A wide range of treatment combinations and side effects were observed in pediatric PH.
- Caring for a child with PH imposes substantial economic, emotional, and time burdens on families.
- Further research is crucial to understand side effect implications and support labeled use of therapies.
Background And Objectives:
Pulmonary hypertension (PH) is a rare, but serious disease among children. However, PH has been primarily evaluated among adults. Consequently, treatment therapies have not been fully evaluated among pediatric populations and are used in an 'off label' manner. The purpose of this study was to estimate the side effect profiles of the most commonly prescribed pediatric PH therapies and to understand the burdens placed upon families caring for children living with PH.
Methods:
Participants were recruited online through the "Families of children with pulmonary hypertension" Facebook group and asked to complete a survey about PH treatments.
Results:
A total of 139 parents of a child living with PH completed the survey. Almost all children used ≥ 1 medication to treat PH, with 52% using ≥ 3 medications. The highest average number of side effects was reported by users of Treprostinil, Selexipag and type-5 phosphodiesterase (PDE5) inhibitors. The most common side effects were skin flushing, headache, nasal congestion, joint/muscle pain, and nausea. In terms of accessing care, 81% travel ≥ 20 miles and 68% travel for ≥ 60 min to receive care.
Conclusions:
We found an array of treatment combinations employed to mitigate symptoms of PH in children, with a wide range of side effects. We also found a large, unseen economic, emotional, and time burden of caring for a child living with PH. Further research is warranted to understand the clinical implications of these side effects to move towards labeled usage of these therapies rather than post-hoc off-label usage.
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