Featured articlePrognostic factors associated with increased survival in patients with pulmonary arterial hypertension treated with subcutaneous treprostinil in randomized, placebo-controlled trials
Section snippets
Population description
This was a retrospective analysis of patients who were enrolled in 3 trials (P01: 04, 05, 06) of SC treprostinil treatment for PAH between June 25, 1998 and December 1, 2003.7, 21, 22 All non-chronic thromboembolic pulmonary hypertension (non-CTEPH) subjects enrolled in the open-label trial of SC treprostinil were included in the analysis, and all patients were on treprostinil therapy. Patients were followed for as long as they received treprostinil and were censored at the time of
Baseline characteristics
Of the 811 patients in this analysis, 628 (77%) were female, and the mean age was 45 (range 5 to 83) years. The most common diagnosis was IPAH (52%, n = 425). Patients in the database were NYHA FC II (16%, n = 126), NYHA FC III (76%, n = 614) and NYHA FC IV (9%, n = 71) at baseline. Twenty-three percent (186 of 811) of the patients were on concomitant PAH medications, including another prostanoid (8%, which includes patients transitioning to intravenous epoprostenol and patients on concomitant
Discussion
Because of the orphan disease status of PAH and ethical issues related to placebo treatment of patients with this debilitating disease, randomized, controlled trials are rarely adequately designed or powered to assess disease survival or prognostic factors of survival benefit.23 In the absence of well-controlled survival trials, there is a need to identify surrogate markers to accurately predict survival. Using a large database of patients with PAH receiving SC treprostinil, we sought to
Disclosure statement
United Therapeutics provided the authors with full access to the trial data and all statistical analyses, as directed by the authors. MedThink Communications provided editorial assistance and project management under the authors' direction, with financial support from United Therapeutics Corporation.
R.L.B. has served as a paid consultant to Pfizer and has received research funding from the sponsor (United Therapeutics Corp.), Lung Rx, Pfizer, Gilead Sciences and Actelion. He is a paid speaker
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