Intravenous treprostinil as an add-on therapy in patients with pulmonary arterial hypertension

Background: In patients with pulmonary arterial hypertension who have an insufficient response to oral or inhaled therapies, current guidelines recommend the use of parenteral prostacyclin analogues, although the efficacy of this approach is unknown.

Methods: This retrospective multicenter study evaluated patients with pulmonary arterial hypertension who received intravenous treprostinil as an add-on therapy. The risk at baseline and follow-up (6-12 months after the initiation of treprostinil) was classified as low, intermediate, or high according to current recommendations. The outcome was measured as transplant-free survival after the initiation of treprostinil therapy.

Results: A total of 126 patients were analyzed, almost all of them pre-treated with combinations of other pulmonary arterial hypertension medications. Before the initiation of intravenous treprostinil, 2 (2%) patients had a low-risk profile; 100 (79%), an intermediate-risk profile; and 24 (19%), a high-risk profile. At follow-up, 24 (19%) patients were classified as low-risk. These patients had a 5-year transplant-free survival rate >90%. In contrast, patients who remained at intermediate or high risk had transplant-free survival rates of 76%, 43%, and 28% at 1, 3, and 5 years, respectively. Failure to reach a low risk at follow-up was an independent predictor of transplant-free survival (hazard ratio, 9.25; 95% confidence interval, 1.20-71.60; p = 0.033 1).

Conclusions: Risk assessment at 6-12 months after the initiation of add-on intravenous treprostinil in patients with an insufficient response to nonparenteral treatments allows the prediction of transplant-free survival over the ensuing years. Achieving a low-risk profile is associated with excellent outcomes, whereas mortality is high in patients who remain at intermediate or high risk.