The Clinical Course of Portopulmonary Hypertension and Outcomes With Endothelin Receptor Antagonist Treatment: Observational Study of Data From the US Organ Procurement and Transplantation Network

Abstract

Background. Portopulmonary hypertension (PoPH) occurs in patients with advanced liver disease and can be a contraindication to liver transplant (LT). Improvement of hemodynamic parameters with pulmonary arterial hypertension (PAH) therapies (including endothelin receptor antagonists [ERAs]) may help some patients to become eligible for LT. Methods. We conducted a retrospective secondary data analysis to describe the clinical course and management of PoPH in patients on a US registry LT waitlist and outcomes in patients receiving an ERA. Results. At the time of LT waitlist entry (1996–2019), patient characteristics and disease severity were similar in the 685 patients with PoPH enrolled overall (LT waitlist data set) and the 420 of them who underwent LT (LT data set). Most patients (92.0%) had a model for end-stage liver disease exception granted before entering the LT waitlist. Patients spent a median of 8.9 mo (interquartile range, 3.7–19.7) on the LT waitlist before undergoing LT. Overall, 77.1% of patients received PAH treatment at LT waitlist entry (ERAs, 30.1%). Hemodynamic parameters improved in ≥95% of patients between the first assessment versus the second (median interval, 9 mo) and last assessments (median interval, 14 mo). At the first assessment, 49.6% of patients had mean pulmonary arterial pressure ≥45 mm Hg versus 2.6% and 1.8% of patients at the second and last assessments, respectively; 47.5% of patients had pulmonary vascular resistance >450 dynes·s/cm5 versus 0.9% and 0.2% of patients at the second and last assessments. One-year survival was 90.6% (95% confidence interval [CI], 87.6-92.9) following LT waitlist entry and was 86.4% (95% CI, 82.6-89.5) after LT; 5-y survival was 67.4% (95% CI, 60.0-73.8) while on the LT waitlist (before LT) and was 75.6% (95% CI, 70.4-80.0) following LT. Conclusions. This large US study of patients with PoPH on an LT waitlist confirms that effective PAH treatments can help patients achieve acceptable hemodynamics, providing the opportunity to undergo LT.