Treatment of Chronic Thromboembolic Pulmonary Hypertension (CTEPH): Real-World Experience with Macitentan

Abstract

Purpose To describe real-world experience with macitentan in patients with CTEPH enrolled in the OPsumit® USers registry (OPUS; NCT02126943). Methods OPUS is an ongoing, long-term, prospective, US, multicenter, observational drug registry of patients newly treated with macitentan. This subgroup analysis describes the clinical characteristics, treatment patterns and safety of macitentan in CTEPH patients in OPUS. Results As of April 2018, OPUS included 45 CTEPH patients with follow-up data. Of these, 35 patients were not candidates for pulmonary endarterectomy (PEA), 7 had persistent pulmonary hypertension (PH) post-PEA, 1 had persistent PH post-balloon pulmonary angioplasty (BPA), 1 was bridging to PEA/BPA and the information was not known for 1 patient. At enrollment, median age (range) was 67 (18-87) years; 64.4% were female. Median (Q1, Q3) time from CTEPH diagnosis was 15.6 (7.8, 40.4) months. Use of macitentan in double or triple combination therapy regimens is shown in the Table for CTEPH patients and patients with pulmonary arterial hypertension (PAH) enrolled in OPUS. In CTEPH, macitentan was initiated as monotherapy in 13 (28.9%) patients, as double combination therapy in 31 (68.9%) patients, and as triple combination therapy in 1 (2.2%) patient. Macitentan was combined with riociguat in 80.6% of patients receiving double combination therapy. Median (Q1, Q3) exposure to macitentan was 9.9 (2.5, 19.9) months. At least 1 adverse event was experienced by 32 (71.1%) patients; the most common were dyspnea (15.6%), dizziness (11.1%) and pneumonia (11.1%). One patient died during the follow-up period. Conclusion OPUS provides insight into real-world use of macitentan. When used in CTEPH, macitentan was received in combination with other PAH therapies in approximately 70% of patients, and most frequently combined with riociguat. There were no unexpected safety findings in CTEPH patients.