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Background <p>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.</p> Methods <p>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&#xa0;years. Clinical data, biomarker levels, and echocardiograms were collected every 6&#xa0;months.</p> Results <p>In total, 10 children (7 females) were enrolled, with a median age of 14.5&#xa0;years. The median follow-up duration was 29.3&#xa0;months. During follow-up, 4 patients (40%) died. At the 6-month follow-up, improvements were observed in NT-proBNP levels (<i>n</i> = 9), 6-min walk distance (6MWD; <i>n</i> = 8) and WHO functional class (WHO-FC; <i>n</i> = 4). Among surviving patients, paired analysis revealed that 83.3% (5/6) showed an improvement in WHO-FC (<i>P</i> &lt; 0.05), NT-proBNP levels were significantly reduced (<i>P</i> &lt; 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.</p> Conclusion <p>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.</p> Trial registration <p>Not applicable.</p>

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

  • 期刊论文

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  • 发表日期：

    2026年5月8日

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  • Meng Li,
  • Yingchun Wang,
  • Xiaoyu Hu,
  • Haizhao Zhao,
  • Weida Lu,
  • Yuan Ji,
  • Xiaopei Cui

摘要

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.