In patients with pulmonary arterial hypertension (PAH), treatment with a short course of parenteral treprostinil lead to more than 2 times the mean total daily dose (TDD) of orally administered treprostinil previously reported in de novo initiators at week 16.

The open-label, multicenter EXPEDITE study was conducted, with findings reported at the American Thoracic Society (ATS) 2023 International Conference, which was held this May in Washington, DC.

The investigators sought to evaluate the efficacy of inducting rapid parenteral treprostinil for attaining twice the real-world dose of oral treprostinil at 16 weeks in patients with PAH. Real-world data had shown a mean TDD of 6 mg of oral treprostinil at 16 weeks for prostacyclin de novo individuals with PAH. Individuals with PAH who had received prior parenteral treprostinil in the same analysis achieved higher doses at the same timepoint.

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Twenty-nine eligible prostacyclin-naive individuals with PAH and a Registry to Evaluate Early and Long-Term PAH Disease Management (REVEAL) Risk Score less than 10 were enrolled in the study. Parenteral treprostinil was titrated over 2 to 8 weeks in all participants. Baseline was defined as the time “prior to parenteral treprostinil initiation.” All individuals transitioned to oral treprostinil over 1 to 21 days, with titration persisting through week 16.

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The primary study endpoint was the percentage of participants who attained an oral treprostinil TDD of 12 mg of greater at week 16. Secondary study endpoints were changes in clinical response and tolerability of the treatment from baseline to week 16.

Overall, 62% of the study participants were female. Characteristics of the 29 participants were as follows:

  • Idiopathic PAH: 41%
  • World Health Organization (WHO) Functional Class (FC) II: 52%
  • WHO FC III: 48%
  • Participants receiving 1 background therapy: 21%
  • Participants receiving 2 background therapies: 38%
  • Participants receiving 3 background therapies: 41%
  • Baseline mean 6-minute walk distance: 367±84.3 m
  • Baseline geometric mean N-terminal pro-brain natriuretic peptide (NT-proBNP): 439±3.9 ng/L

Results of the study showed that at week 16, mean oral treprostinil TDD was 16.4 mg, and 79.3% of the participants had met the primary endpoint. Also at week 16, 68% of participants reported improvement in WHO FC, with 46% attaining WHO FC I and 39% attaining WHO FC II.

The median change in NT-proBNP was –134 ng/L (geometric mean ratio, 0.6; P =.0026). In fact, the proportion of participants with an NT-proBNP of less than 300 ng/L increased 2-fold to 68%. The median change in 6-minute walk distance was +25 m (least squares mean change, +18.8 m; P =.0078).

The mean change in right atrial area was –3.0 cm2, with the proportion of participants with a right atrial area less than 18 cm2 more than doubling to 61%. Positive changes were reported in additional echocardiographic parameters, Borg Dyspnea Score, and quality of life as well. There were no unanticipated adverse events reported.

“Patients had clinically meaningful improvements in quality of life, right heart imaging, and clinical variables associated with risk status,” the researchers noted. “These results support short[-]course parenteral treprostinil induction as an effective approach to quickly reach efficacious doses of oral treprostinil,” they concluded.


Kingrey JF, Miller CE, Balasubramanian V, et al. Remodulin induction facilitates rapid achievement of therapeutic doses of oral treprostinil: results from the EXPEDITE study. Presented at: American Thoracic Society (ATS) 2023 International Conference, Washington, DC; May 19-24, 2023..