Real-world evidence of subcutaneous treprostinil use in pulmonary arterial hypertension in Argentina

Abstract

Introduction: Pulmonary arterial hypertension is a progressive haemodynamic disease with high morbidity and mortality. Of the different treatments available, the prostacyclin analogues are the drugs of choice for high-risk patients, with treprostinil being the most commonly used drug in Argentina. Methodology: The objective of this study is to perform a retrospective evaluation of the efficacy and safety of subcutaneous treprostinil in regular clinical practice in Argentina in 51 patients with pulmonary arterial hypertension after 12 months of follow-up. Results: The results showed that treatment with subcutaneous treprostinil is associated with a significant improvement in different clinical efficacy parameters: 65% reduction in advanced functional class ( p < 0.0001), 130-m increase in the 6-min walk test ( p < 0.0001), 65% reduction in the pro B-type natriuretic peptide value (–531 pg/dL; p < 0.0001), significant reduction of 15.7% in pulmonary vascular resistance [–1.3 wood units (WU); p < 0.0001], improved cardiac index with an increase of 16.7% (+0.4 L/min/m2; p = 0.002), as well as a high survival rate (92%) and a 44% incidence of combined events (mortality, heart failure, syncope and/or lung transplantation), without a significant increase in previously reported adverse events. The risk stratification evaluation according to ESC/ERS guidelines showed a significant decrease in the proportion of patients at high risk after the treatment period ( p = 0.004). Conclusions: These real-world results corroborate the efficacy and safety of subcutaneous treprostinil, even at high doses, and open up the possibility of improving its current use in clinical practice as a first-line therapy, especially in high-risk patient profiles.