A real-world study on selexipag for pulmonary arterial hypertension: a single-center retrospective study

Abstract

Abstract Background Pulmonary arterial hypertension (PAH) is a rare disease with a poor prognosis, although outcomes have improved with the development of pulmonary vasodilators. Epoprostenol, a strong prostacyclin agent, requires continuous intravenous infusion, which can be a burden for patients. Consequently, less invasive PGI₂ derivatives have been developed. The GRIPHON trial (2015) established the efficacy and safety of selexipag, an oral prostacyclin receptor agonist now used worldwide. However, real-world data on its clinical effectiveness and tolerability remain scarce. Purpose We aimed to assess the real-world efficacy and safety of selexipag in patients with PAH. Methods We retrospectively investigated 97 consecutive patients with PAH who received selexipag between 2016 and 2022. We collected data on demographics, treatment details, and clinical outcomes. Efficacy endpoints included changes in pulmonary vascular resistance (PVR), mean pulmonary artery pressure (mPAP), six-minute walk distance (6MWD), and COMPERA risk classification. Safety endpoints included adverse events requiring symptomatic treatment. Results Ninety-seven patients (mean age 50±14 years, 73 females) were analyzed and included idiopathic/heritable PAH (56%), connective tissue disease-associated PAH (16%), and congenital heart disease–associated PAH (20%). The mean maintenance dose of selexipag was 1.9±1.1 mg/day in clinical practice. At 52 weeks after selexipag initiation, PVR (primary endpoint; P0.05) and mPAP (P0.01) significantly decreased, while 6MWD significantly improved (P0.01). Most patients showed stable or improved COMPERA risk classification. Adverse events requiring symptomatic management were observed in 60 patients (62%), with headache (n=41), diarrhea (n=28), nausea (n=14), and jaw pain (n=7) being the most common. Conclusions In the real-world setting of patients with PAH, selexipag demonstrated improvements in hemodynamics, exercise capacity, and risk classification and did not add newly defined safety signals. These findings are supportive to the efficacy and tolerability of a unique oral PGI₂ receptor agonist selexipag for the favorable management of PAH.