Efficacy and Tolerability of Selexipag in Pediatric Patients with Pulmonary Arterial Hypertension: An Updated Systematic Review and Meta-analysis.
Vasiliki Patsiou, Triantafyllia Grantza, Thomas Chrysochoidis-Trantas et al.
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In 30 seconds
This systematic review and meta-analysis evaluated selexipag efficacy and tolerability in children under 18 with pulmonary arterial hypertension (PAH). Twelve studies included 344 patients, 90.6% of whom had PAH. Selexipag was associated with improved 6-minute walk distance (SMD: - 0.75; 95% CI: - 1.22 to - 0.29; P = 0.002), while pooled analyses found no effect on pulmonary vascular resistance index, mean pulmonary artery pressure, or NT-proBNP. Adverse events occurred in 62.8% and were predominantly mild. Functional improvement was greater with add-on therapy, alongside more adverse events.
Key findings
- 6-minute walk distance improved with selexipag (SMD: - 0.75; 95% CI: - 1.22 to - 0.29; P = 0.002).
- Pooled analyses found no effect on pulmonary vascular resistance index, mean pulmonary artery pressure, or NT-proBNP.
- In sensitivity analysis of PAH-only studies, mean pulmonary artery pressure decreased (SMD: - 0.30; P = 0.03).
- Adverse events occurred in 62.8%, predominantly mild; add-on therapy showed greater functional improvement and more adverse events.
Why it matters
The findings suggest a potential functional benefit of selexipag in pediatric PAH, while highlighting that effects on several haemodynamic and biomarker outcomes were not demonstrated in pooled analyses. The reported adverse-event frequency and differences between treatment subgroups are relevant when interpreting tolerability and efficacy. The evidence remains limited and heterogeneous, so the results do not establish long-term benefit.
What to keep in mind
Evidence came from small, heterogeneous studies, and the review calls for further prospective studies to confirm long-term benefits.