PCV2 - Characterizing Patients Treated With Macitentan For Pulmonary Arterial Hypertension (PAH) In The U.S. Opsumit Users Registry (OPUS)

Abstract

Health plans look beyond randomized controlled trials (RCTs) in making formulary decisions. There are three approved endothelin receptor antagonists, but no head-to-head trials. Real world evidence (RWE) informs the application of RCT results to healthcare decision making by assessing care and patient outcomes in routine clinical practice. RWE is derived from registries, medical records, pragmatic trials, and claims databases. OPUS registry (NCT02126943) is a US-based, multi-center, longitudinal, observational drug registry of newly treated macitentan patients. The objective of this analysis is to describe clinical characteristics, co-morbidities, and treatment of PAH patients with macitentan. Patients newly treated with macitentan (initiated < 30 days prior to enrollment visit) were included in the registry, regardless of prior or concomitant PAH therapy. Planned observation period was at least one year. Data collected at enrollment or follow-up included: demographics, clinical characteristics, outcomes, adverse events, macitentan treatment and registry discontinuation. As of June 2017, 1136 patients with PAH had follow-up data--74.2% females and 25.8% males. Of this cohort, 44.5% of patients were > 65 years of age; 57.9% patients had idiopathic PAH, and 24.9%, PAH associated with connective tissue disease. Common baseline comorbidities included: hypertension (52.6%), edema (24.5%), signs of right heart failure (15.8%), and diabetes mellitus (22.5%). Median exposure to macitentan was 9.6 months. Macitentan was initiated as monotherapy in 41.7%, and double or triple combination therapy in 58.2% of patients. Most common adverse events were dyspnea (18.0%) and peripheral edema (8.9%). 12.4% of patients experienced ≥1 PAH-related hospitalization. OPUS reports macitentan use and safety in real world clinical settings. Reported comorbidities are consistent with other large registries and claims analyses. Macitentan is initiated most commonly as double or triple combination therapy. PAH related hospitalizations were lower than previously reported in both claims analysis and registries.