Erik J. Nelson, Ella Cook, Samara Nelson, Rebecca Brown, Megan Pierce, Ashley Bangerter Seelos, Heather Stickle and Michael Johansen
Brigham Young University. Utah State University. Indiana University School of Medicine.
United States
BioMed Central Pediatrics
BMC Pediatr 2023; 23:
DOI: 10.1186/s12887-023-03860-2
Abstract
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.
Category
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Quality of Life Associated with Pulmonary Vascular Disease
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Filed (PHiled). Greater than 1-2 years since publication
Article Access
Free PDF File or Full Text Article Available Through PubMed or DOI: Yes