What is the clinical evidence from this study?

Study design: Cohort. Population: Pulmonary arterial hypertension (n=10). Intervention: Selexipag (in triple combination therapy) vs. Baseline. Primary outcome: NT-proBNP levels (pg/ml) (p=<0.05).

What does this research mean for the field?

Selexipag-based triple combination therapy significantly reduces NT-proBNP levels and improves WHO functional class with acceptable safety in Chinese children with pulmonary arterial hypertension. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

What question did this study set out to answer?

This study aims to assess the efficacy and safety of selexipag in combination therapy for children with PAH.

May 10, 2026Open Access

Experience with selexipag in triple therapy for pulmonary arterial hypertension in Chinese children

Q: What are the key findings of this study?

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.

Q: What does this research mean for the field?

Selexipag-based triple combination therapy significantly reduces NT-proBNP levels and improves WHO functional class with acceptable safety in Chinese children with pulmonary arterial hypertension. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

Q: What question did this study set out to answer?

This study aims to assess the efficacy and safety of selexipag in combination therapy for children with PAH.

Key Result

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.

Key Points

This study aims to assess the efficacy and safety of selexipag in combination therapy for children with PAH.
Retrospective single-centre study at Qilu Hospital, including 10 pediatric patients with Group 1 PAH.
Clinical data, biomarker levels, and echocardiographic parameters were collected every 6 months from November 2018 to September 2023.
Follow-up duration averaged 29.3 months with median age of 14.5 years.
40% (4/10) of patients died during follow-up; survival rates were 80%, 70%, and 60% at 1, 2, and 3 years.
Significant improvements in WHO functional class and NT-proBNP levels among surviving patients (P < 0.05).
6-min walk distance showed a non-significant increase and echocardiographic parameters did not significantly change.

Study Design

Type

Cohort (n=10)

Multicenter

Structured PICO

Does selexipag-based triple combination therapy improve functional class and biomarkers in pediatric patients with pulmonary arterial hypertension?

Population

10 Chinese pediatric patients (median age 14.5 years, 70% female) with Group 1 Pulmonary Arterial Hypertension (PAH) confirmed by right heart catheterization, WHO functional class II-IV.

Intervention

Selexipag (initiated at 100-200 μg twice daily, titrated up to max 1600 μg twice daily) as part of triple combination therapy with endothelial receptor antagonists (ERAs) and phosphodiesterase-5 inhibitors (PDE5is).

Outcome

Efficacy (WHO functional class, NT-proBNP levels, 6-minute walk distance, echocardiographic parameters) and safety (adverse events, survival) at 6 months and last available follow-up.

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.

Main Result

Absolute Event Rate: 181.7% vs 587.6%

p-value: p=<0.05

Limitations

Small sample size
Retrospective single-centre design
Lack of a control group
Efficacy assessment relied predominantly on non-invasive parameters
Dosing strategies were not standardised and pharmacokinetic data were unavailable

Abstract

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.

Bookmark

View Full Paper