Efficacy and Safety of Oral Treprostinil Monotherapy for the Treatment of Pulmonary Arterial Hypertension

• DOI: 10.1161/circulationaha.112.124388
• Publication Date: 2013-01-11T07:02:00Z
• Authors: Zhi-Cheng Jing; Keyur Parikh; Tomas Pulido; Carlos Jerjes-San...; R. James White; Roblee Allen; Adam Torbicki; Kai-Feng Xu; David Yehle; Kevin Laliberte; Carl Arneson; Lewis J. Rubin
• Keywords: Treprostinil

Background— Pulmonary arterial hypertension (PAH) is a progressive, fatal disease with no cure. Parenteral and inhaled prostacyclin analogue therapies are effective for the treatment of PAH, but complicated administration requirements can limit use these in patients less severe disease. This study was designed to evaluate safety efficacy oral treprostinil diolamine as initial de novo PAH. Methods Results— Three hundred forty-nine (intent-to-treat population) not receiving endothelin receptor antagonist or phosphodiesterase type-5 inhibitor background therapy were randomized (treprostinil, n=233; placebo, n=116). The primary analysis population (modified intent-to-treat) included 228 n=151; n=77) access 0.25-mg tablets at randomization. end point change from baseline 6-minute walk distance week 12. Secondary points Borg dyspnea index, clinical worsening, symptoms 12 effect intent-to-treat 23.0 m ( P =0.0125). For population, improvements observed peak (26.0 m; =0.0001) trough (17.0 =0.0025) plasma drug concentrations. Other than an improvement combined distance/Borg score, there significant changes secondary points. Oral generally well tolerated; most common adverse events (intent-to-treat) headache (69%), nausea (39%), diarrhea (37%), pain jaw (25%). Conclusions— improves exercise capacity PAH other treatment. could provide convenient, first-line option requiring more intensive therapy. Clinical Trial Registration:— URL: http://www.clinicaltrials.gov . Unique identifier: NCT00325403