Selexipag-based triple combination therapy significantly reduced median NT-proBNP levels from 587.6 to 181.7 pg/ml in Chinese children with pulmonary arterial hypertension.
Cohort (n=10)
No
Does selexipag-based triple combination therapy improve functional class and biomarkers in pediatric patients with pulmonary arterial hypertension?
Selexipag-based triple combination therapy may improve functional class and NT-proBNP levels in pediatric PAH, though mortality remains high and larger prospective studies are needed.
Absolute Event Rate: 181.7% vs 587.6%
p-value: p=<0.05
Abstract Background Selexipag 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. Methods We 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. Results In 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. Conclusion In 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 registration Not applicable.
Li et al. (2026) conducted a cohort in Pulmonary arterial hypertension (n=10). Selexipag (in triple combination therapy) vs. Baseline was evaluated on NT-proBNP levels (pg/ml) (p=<0.05). Selexipag-based triple combination therapy significantly reduced median NT-proBNP levels from 587.6 to 181.7 pg/ml in Chinese children with pulmonary arterial hypertension.