PURPOSE: Current PAH treatment algorithms encourage a stepwise, add-on approach. At present, the ideal time to add-on prostacyclin therapy remains unknown. TRIUMPH-1 was a 12-week double blind (DB) trial of iTRE in patients stabilized on an oral background therapy with a long term open-label (OL) extension. This analysis compares efficacy outcomes up to 2 years with OL iTRE in patients originally assigned in DB to iTRE (iTRE/ iTRE) or PBO (PBO/ iTRE).
METHODS: 206 subjects (125 US, 81 Rest of World) participated in OL. Six minute walk distance (6MWD), quality of life (MLwHF), and NYHA functional class were assessed every 3 months. Exposure time for all patients was calculated based on time on active iTRE.
RESULTS: Median baseline 6MWD was 359 meters for 97 patients receiving iTRE/iTRE and 365 meters for 109 patients receiving PBO/iTRE. Marked improvements in 6MWD were noted in iTRE/ iTRE as compared with PBO/ iTRE, with median changes of 37, 38, 35 and 26 meters vs. 19, 25, 22, and 5 meters at 6, 12, 18, and 24 months respectively. After 18 and 24 months of therapy respectively, 45% and 38% iTRE/iTRE had improvements in 6MWD of >50m compared to 32% and 27% in PBO/iTRE. A greater frequency of improvement in components of MLwHF was also noted in iTRE/iTRE, as were decreases in functional class (FC) at 24 months compared to baseline (42% iTRE/iTRE vs. 30% PBO/iTRE).
CONCLUSIONS: PAH is a progressive disease often requiring multiple therapies. These data obtained in patients stabilized on an oral PAH medication suggest that the earlier addition of inhaled treprostinil provides long-term improvement in exercise capacity and QOL, while also demonstrating the potential outcome of delaying active therapy in placebo controlled trials.
CLINICAL IMPLICATIONS: PAH can progress insidiously and constant surveillance is required to avoid clinical worsening.
DISCLOSURE: The following authors have nothing to disclose: Raymond Benza, Karl Gotzkowsky, Amanda Jenkins, Lewis Rubin
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