ArticleBMC pediatrics2026
Experience with selexipag in triple therapy for pulmonary arterial hypertension in Chinese children.
Article in BMC pediatrics, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundSelexipag is an orally effective prostacyclin receptor agonist that has been approved for treating pulmonary arterial hypertension (PAH) in adults but is still used off-label in children. This study aimed to evaluate the efficacy and safety of selexipag as part of triple combination therapy (TCT) with endothelial receptor antagonists (ERAs) and phosphodiesterase-5 inhibitors (PDE5is) in Chinese children with PAH.
methodsWe conducted a retrospective single-centre study including pediatric patients with Group 1 PAH who received selexipag-based TCT at Qilu Hospital of Shandong University from November 2018 to September 2023. A total of 10 pediatric patients were enrolled, with ages ranging from 8.9 to 17.2 years. Clinical data, biomarker levels, and echocardiograms were collected every 6 months.
resultsIn total, 10 children (7 females) were enrolled, with a median age of 14.5 years. The median follow-up duration was 29.3 months. During follow-up, 4 patients (40%) died. At the 6-month follow-up, improvements were observed in NT-proBNP levels (n = 9), 6-min walk distance (6MWD; n = 8) and WHO functional class (WHO-FC; n = 4). Among surviving patients, paired analysis revealed that 83.3% (5/6) showed an improvement in WHO-FC (P < 0.05), NT-proBNP levels were significantly reduced (P < 0.05), and 6MWD exhibited a non-significant increasing trend. No statistically significant changes were observed in echocardiographic parameters. The 1-, 2-, and 3-year transplant-free survival rates were 80%, 70%, and 60%, respectively. Selexipag was generally well tolerated, and no patients discontinued treatment due to adverse events.
conclusionIn this small retrospective cohort, selexipag-based TCT was associated with improvements in WHO-FC and NT-proBNP levels and acceptable safety in Chinese children with PAH. Nevertheless, our findings are limited by the small sample size and retrospective single-centre design, highlighting the need for larger prospective studies.
trial registrationNot applicable.
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