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.

Keywords: population registry; prostacyclin; prostanoids; pulmonary arterial hypertension; retrospective studies; survival rate.

Figure 1.

Consort diagram.

Figure 2.

Effect of the treprostinil treatment on different variables at baseline and during the follow-up period (after 3, 6, 9 and 12 months of treatment). (a) Effect of the treatment with treprostinil on the 6-min walk test (6MWT). (b) Effect of the treatment with treprostinil on pro B-type natriuretic peptide (pro-BNP). (c) Effect of the treatment with treprostinil on tricuspid annular plane systolic excursion (TAPSE) value.

Figure 3.

Patient distribution by FC throughout the 12-month treatment period with treprostinil.

Figure 4.

Survival analysis by Kaplan–Meier curves. (a) Overall mortality (survival) during the treatment period. A survival rate of 92% after 12 months is observed with four deaths. The deaths occurred: one after 6 months of treatment, another one after 9 months and the remaining two after 12 months. The causes were: two cases of sudden death, one case of heart failure and another from infection. Three patients were at high risk and one at intermediate risk according to ESC/ERS guidelines. (b) Survival for the combined variable of mortality, hospitalisation for heart failure, syncope and/or lung transplantation. An overall incidence of 43.1% is observed; this means that 22 patients presented at least one of the events included in the combined variable during the treatment period (17 patients suffered from heart failure, 4 patient deaths and 1 patient presented recurrent syncope).

Figure 5.

Survival analysis by Kaplan–Meier curves for the combined variable of mortality, hospitalisation for heart failure, syncope and/or lung transplantation during the treatment period. (a) Analysis according to pro- BNP values. There is a significantly greater incidence (21, 53%) of combined events in the patients with high levels of pro-BNP after the 12 months of treatment. (b) Analysis according to functional class (FC) levels. Patients with advanced FC present a greater incidence of combined events (20; 43.4%) versus the group without advanced FC (2; 40%) after 12 months of treatment.