Macitentan for pulmonary arterial hypertension related to repaired congenital heart disease: real-world UK experience

Abstract Introduction Patients with pulmonary arterial hypertension (PAH) following congenital heart disease (CHD) repair are an emerging population at increased risk of complications. Small numbers such patients have been included in studies evaluating the efficacy macitentan, but since obtaining a license for use this population, limited real-world data published. Purpose To describe safety and macitentan therapy adults repaired PAH-CHD. Methods We conducted retrospective observational study PAH-CHD newly started on 2 UK specialist centres between 2014 2020. Clinical variables, including WHO functional class (FC), 6-minute walk (6MW) or incremental shuttle (ISW) distance, EMPHASIS-10 score were recorded prior to after initiation therapy. Results Overall, 49 included, 63.3% female, median age 40.8 [26.7–53.7] years Macitentan was as part initial PAH therapeutic regimen 18 (36.7%) add-on existing 31 (63.3%) patients, whom over third (35.5%) switched from another endothelin receptor antagonist. At time initiation, most (68.4%) (FC) III, while 26.3% FC II 5.3% I. Baseline distance (n=31, 72.1%) 355.0 [263.5–461.2] m (n=12, 27.9%) 220.0 [175.0–302.5] m. (n=39) 24 [10–36]. After 12.7 [9.8–16.1] months therapy, objective exercise capacity improved by 11.8 [−4.3–36.9]% (p=0.01). Health-related quality life least 5 points 41.9% there no significant change overall group (0.0 [−20.3–18.1]% change, p=0.78). stopped 4 (8.2%) due adverse event patient choice, (10.2%) clinical worsening absence improvement. 1 year 6 (12.2%) had further escalated triple died. Conclusions treated highly symptomatic tolerance. well-tolerated led improvement capacity. Funding Acknowledgement Type funding sources: Private grant(s) and/or Sponsorship.