Efficacy and Tolerability of Selexipag in Pediatric Patients with Pulmonary Arterial Hypertension: An Updated Systematic Review and Meta-analysis.

Evidence supporting selexipag in pediatric pulmonary arterial hypertension (PAH) is sparse, mainly from small, heterogeneous studies. This meta-analysis assessed its efficacy and tolerability in this population. A systematic search of PubMed, Embase, Web of Science, Cochrane Library, and Scopus was conducted until April 2025. Eligible studies included patients under 18 years diagnosed with PAH and treated with selexipag, reporting clinical outcomes. Two reviewers independently screened, extracted data, and evaluated study quality. Pooled analyses of efficacy and safety outcomes were performed, with subgroup comparisons for add-on therapy versus transition from parenteral prostacyclin analogs. Twelve studies involving 344 pediatric patients (90.6% diagnosed with PAH) were included. Selexipag significantly improved 6-minute walk distance (SMD: - 0.75; 95% CI: - 1.22 to - 0.29; P = 0.002), but did not affect pulmonary vascular resistance index, mean pulmonary artery pressure (mPAP), or NT-proBNP. Sensitivity analysis revealed reduced mPAP in PAH-only studies (SMD: - 0.30; P = 0.03). Adverse events occurred in 62.8%, predominantly mild. Functional improvement was greater in the add-on therapy group, which also experienced more adverse events. Selexipag improved functional capacity and mPAP in pediatric PAH and was generally well tolerated. Further prospective studies are needed to confirm long-term benefits.