Abstract
Pulmonary vasodilator therapy is still often an off-label treatment for pulmonary hypertension in children. The aim of this nationwide register-based study was to assess patient characteristics and strategies for pulmonary vasodilator therapy in young Swedish children. Prescription information for all children below seven years of age at treatment initiation, between 2007 and 2017, was retrieved from the National Prescribed Drug Register, and medical information was obtained by linkage to other registers. All patients were categorized according to the WHO classification of pulmonary hypertension. In total, 233 patients had been prescribed pulmonary vasodilators. The treatment was initiated before one year of age in 61% (N = 143). Sildenafil was most common (N = 224 patients), followed by bosentan (N = 29), iloprost (N = 14), macitentan (N = 4), treprostinil (N = 2) and riociguat (N = 2). Over the study period, the prescription rate for sildenafil tripled. Monotherapy was most common, 87% (N = 203), while 13% (N = 20) had combination therapy. Bronchopulmonary dysplasia (N = 82, 35%) and/or congenital heart defects (N = 156, 67%) were the most common associated conditions. Eight percent (N = 18) of the patients had Down syndrome. Cardiac catheterization had been performed in 39% (N = 91). Overall mortality was 13% (N = 30) during the study period. This study provides an unbiased overview of national outpatient use of pulmonary vasodilator therapy in young children. Few cases of idiopathic pulmonary arterial hypertension were found, but a large proportion of pulmonary hypertension associated with congenital heart defects or bronchopulmonary dysplasia. Despite treatment, mortality was high, and additional pediatric studies are needed for a better understanding of underlying pathologies and evidence of treatment effects.
Highlights
Pulmonary hypertension (PH) is associated with considerable morbidity and mortality
ICD diagnosis of PH was found for 223 of 233 children and they were stratified according to World Health Organization (WHO) classification groups 1–5
Down syndrome was identified in 8% (N 1⁄4 18/233) of the total study population and most commonly in WHO group 1, where 25% (N 1⁄4 11/44) of the patients had Down syndrome
Summary
Treatment options for pre-capillary WHO group 1 PH, pulmonary arterial hypertension (PAH), have increased over the last two decades, and modern therapy has improved survival but is still far from curative. Strategies for pulmonary vasodilator therapy in children is often off-label, and especially for non-group 1 PH it is in many cases based on experience and expert opinion rather than on evidence from studies.[1]. 2 | A national study of vasodilator therapy for pulmonary hypertension in young children Jeremiasen et al. The updated version from 2018 included minor changes for better understanding and sorting of PH.[1,4,5]
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
