Clinical utility of treprostinil in the treatment of pulmonary arterial hypertension: an evidence-based review

Figures & data

Core evidence clinical impact summary

Outcome measure	Evidence	Implications
Disease-oriented evidence	Clinical trials	Parenteral and inhaled treprostinil have been shown to be safe and effective in improving exercise capacity, functional class, and dyspnea scoresImproved clinical outcomes have not been consistently demonstrated with oral therapy
Patient-oriented evidence	Clinical trials	Parenteral and inhaled treprostinil have demonstrated improved quality of life, while oral therapy has notAdverse events associated with parenteral and oral therapy should be monitored for safety and tolerability Inhaled therapy has been shown to be well tolerated without serious adverse events
Economic evidence	None currently	No formal cost-effective analysis has been conducted. However, costs should be considered when selecting a specific formulation in comparison to other available PAH therapies

Table 1 Summary of treprostinil clinical trials

Outcome measure	Evidence	Implications
Disease-oriented evidence	Clinical trials	Parenteral and inhaled treprostinil have been shown to be safe and effective in improving exercise capacity, functional class, and dyspnea scoresImproved clinical outcomes have not been consistently demonstrated with oral therapy
Patient-oriented evidence	Clinical trials	Parenteral and inhaled treprostinil have demonstrated improved quality of life, while oral therapy has notAdverse events associated with parenteral and oral therapy should be monitored for safety and tolerability Inhaled therapy has been shown to be well tolerated without serious adverse events
Economic evidence	None currently	No formal cost-effective analysis has been conducted. However, costs should be considered when selecting a specific formulation in comparison to other available PAH therapies

Note: ^aRetrospective subgroup analysis of pulmonary arterial hypertension-associated connective tissue disease subjects from a previously published multicenter, randomized, double-blind, placebo-controlled trial.

Abbreviations: ↑, increase; ↓, decrease; 6MWD, 6-minute walk distance; CI, cardiac index; CO, cardiac output; DB, double blind; MC, multicenter; MPAP, mean pulmonary artery pressure; NYHA, New York Heart Association; PLC, placebo controlled; PVR, pulmonary vascular resistance; PVRI, pulmonary vascular resistance index; QOL, quality of life; R, randomized; WHO, World Health Organization.