BACKGROUND: Pulmonary arterial hypertension (PAH) is a common complication among patients with congenital heart disease (CHD). Despite advances in PAH treatment, evidence for the benefits of PAH therapies in CHD-PAH is limited. OBJECTIVES: This analysis aimed to evaluate outcomes in patients with repaired PAH-CHD receiving an approved PAH drug. METHODS: This was a pooled analysis including CHD-PAH patients whose CHD was repaired ≥1 year prior from 3 randomized, placebo-controlled, event-driven studies: GRIPHON (NCT01106014), SERAPHIN (NCT00660179), and COMPASS-2 (NCT00303459). The primary endpoint was time to first confirmed morbidity/mortality (M/M) event. HRs with 95% CIs were determined with random effects models. RESULTS: The analysis included 1,982 patients with PAH, 177 (8.9%) with CHD-PAH. In the overall PAH cohort, the mean age was 48 and 49 years in treatment and placebo groups; 80% and 77% were female. In the CHD-PAH cohort, the mean age was 41 and 39 years; 70% and 66% were female. Overall, ≥98% in each group were World Health Organization functional class II and III at baseline. There was a significant reduction in risk of M/M events vs placebo in the overall PAH and CHD-PAH cohorts: 37% reduction in the overall PAH cohort (HR: 0.63; 95% CI: 0.52-0.77) and 50% reduction in the CHD-PAH population (HR: 0.50; 95% CI: 0.26-0.94). CONCLUSIONS: Treatment with approved PAH drugs provided a similar reduction in M/M risk in patients with repaired CHD-PAH when compared with the overall PAH population. This pooled analysis provides important evidence to guide medical management in this patient population.

Ahmanson UCLA Adult Congenital Heart Disease Center University of California Los Angeles California USA

Cardiology and Center of Cardiovascular Medicine Medical University of Vienna Vienna Austria

CARE Hospitals Hyderabad India

Centre of Postgraduate Medical Education European Health Centre Otwock Poland

Charles University and General University Hospital Prague Czech Republic

David Geffen School of Medicine at University of California Los Angeles Los Angeles California USA

Duke University Health System Durham North Carolina USA

Hannover Medical School Hannover Germany; German Center of Lung Research Hannover Germany

Ignacio Chavez National Heart Institute Mexico Mexico

Johnson and Johnson Titusville New Jersey USA

London Health Sciences Centre Schulich School of Medicine and Dentistry Western University London Ontario Canada

Mater Misericordiae University Hospital Dublin Ireland

Université Paris Saclay APHP Hôpital Bicêtre Le Kremlin Bicêtre France

University of Michigan Health System Ann Arbor Michigan USA

University of Texas Southwestern Medical Center Dallas Texas USA

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