Experience with Selexipag in Triple Therapy for Pulmonary Arterial Hypertension in Chinese Children

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 in 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 of children with Group 1 PAH receiving selexipag-based TCT at Qilu Hospital of Shandong University, China. Clinical data, biomarker levels, and echocardiograms were collected every 6 months, and risk score for paediatric pulmonary hypertension (PH) compiled by the European Paediatric Pulmonary Vascular Disease Network (EPPVDN) in 2019 was calculated based on existing data. Results: A total of 11 patients (7 female) were included, with a median age of 13.0 (range 3.1-17.2) years. After 6 months, none of the patients died. Six patients (55%) experienced tolerable adverse reactions. The median N-terminal pro-brain natriuretic peptide decreased by 622 pg/ml (P<0.01), the median functional class changed from III to II (P<0.05), and the median 6-minute walk distance increased by 29 m (P<0.05). Regarding non-invasive risk scores by the EPPVDN, the median high-risk and low-risk scores decreased by 0.2857 (P<0.01) and increased by 0.3077 (P<0.01), respectively. For all 11 children, the one-, two-, and three-year estimated survival rates were 81%, 73%, and 64%, respectively. Conclusion: Our small retrospective study supports the use of selexipag as a viable off-label option in pediatric TCT. Trial registration: Not applicable.