Conference: 2017 PH Professional Network Symposium
Release Date: 10.06.2017
Presentation Type: Abstracts
File Download: 2017 PHPN Abstract 1005
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Abstract presented at the 2017 PH Professional Network Symposium held in Bethesda, MD on October 5-7, 2017
To provide insight into the use of macitentan in the real-world, post-marketing setting.
The OPsumit® USers registry (OPUS; NCT02126943) characterizes the use and safety profile of the endothelin receptor antagonist (ERA) macitentan (Opsumit®) in the real-world setting.
Started in April 2014, OPUS is an ongoing long-term, prospective, multicenter, observational drug registry of patients newly treated with macitentan in the US. This analysis reports patient characteristics, treatment patterns, and safety of macitentan in patients with pulmonary arterial hypertension (PAH) enrolled in OPUS.
By April 17, 2016, 688 PAH patients who newly initiated macitentan had enrolled in OPUS; follow-up information was available for 590 patients. The follow-up cohort had a median age of 63 years (range, 19–90) at enrollment and was primarily female (74.2%). In total, 47.9% of patients had been diagnosed with PAH within 6 months, and 16.3% had switched from another ERA. WHO functional class at enrollment was assessed in 454 patients; 38.4% were class I/II and 61.6% were class III/IV. The median 6-minute walk distance was 300 m (range, 30.0-750.0 m; n=308). Treatment patterns with respect to PAH-specific medications are shown in Table 1. During follow-up, 401 (68.0%) patients received concomitant treatment with at least one PAH-specific therapy, primarily with phosphodiesterase type 5 inhibitors (PDE5i). Median exposure time of concomitant medications was as follows: PDE5i 5.5 months (range 0.0–20.6 months); i.v./s.c. prostanoids 3.9 months (0 .0–20.6 months); inhaled prostanoids 5.4 months (0.0–19.2 months); oral prostanoids 1.1 months (0.0–7.6 months); and soluble guanylate cyclase-stimulators 4.0 months (0.0–17.0 months). Comparisons between the overall follow-up cohort and the subset of patients who switched from another ERA showed similar median macitentan exposure time (5.6 months [range 0.1–22.1 months] versus 6.6 months [0.1–19.9 months]; similar proportions of patients exposed to macitentan for >1 year (15.9% versus 20.9%); and similar treatment discontinuation rates (22.7% versus 19.8%). Adverse events (AEs) were the most common reason for discontinuation. In the overall follow-up cohort, 338 patients (57.3%) experienced ≥1 AE during the exposure period, the most common being dyspnea (12.9%) and peripheral edema (8.0%). Twenty patients (3.4%) experienced ≥1 hepatic AE, and 55 (9.3%) experienced ≥1 PAH-related hospitalization. The 12-month Kaplan-Meier survival esti mate was 93% (95% CI: 0.89, 0.95).
OPUS provides further insights into macitentan use in the real-world, post-marketing setting. Specifically, the majority of patients are treated with combination PAH therapy. Further, the safety profile of macitentan in the real-world setting is consistent with that observed in the clinical trial setting when used as monotherapy, in combination with other PAH treatments, and in those who switched to macitentan from another ERA.