Long-Term Course of Pulmonary Arterial Hypertension in Adults with Congenital Heart Disease under Targeted Therapy: A Retrospective Analysis of a Single Tertiary Center

Background: Pulmonary arterial hypertension (PAH), a common complication in adults with congenital heart disease (CHD), leads to significant morbidity and mortality. Targeted PAH medication is available, but PAH-CHD patient data are limited. Several questions regarding indication, treatment escalation, and combination therapy remain unanswered. The aim of this study was therefore to evaluate PAH-specific treatment in adults with PAH-CHD to better understand PAH-specific therapy management.

Method: In this cross-sectional study, we retrospectively examined clinical, demographic, and cardiac-catheterization data and medical management for PAH-CHD, and analyzed clinical course and midterm outcome.

Results: Over up to 14 years (median, 6.2), 103 PAH-CHD patients (66% female) receiving targeted PAH-therapy for pretricuspid shunt (15.5%), posttricuspid shunt (32.0%), and complex CHD (52.4%) were followed up. Based on modified clinical ESC classification, patients were assigned to the following subgroups: Eisenmenger syndrome (ES) (45.6%), severe pulmonary vascular disease (PVD) in complex CHD (20.4%), post-repair patients (19.4%), prevalent systemic-to-pulmonary shunt (3.9%), coincidental/small defects (0%), and Fontan circulation (10.7%).

Over the study course, the medical treatment strategy changed toward combination therapy (baseline: 13.6%; study end: 41%), resulting mostly in stabilized functional class or even improvement in cases of prevalent systemic-to-pulmonary shunt, ES, and patients with repaired CHD. Functional class deterioration, however, was seen in patients with severe PVD due to complex CHD, and Fontan patients.

Of the 103 patients in the study, 25 died (24.3%). Patients with repaired CHD and patients with systemic-to-pulmonary shunt or ES showed the best survival rates. Mortality was remarkably higher in patients with severe PVD in complex CHD and Fontan patients.

Conclusion: Many patients with PAH-CHD benefited from targeted PAH therapy over a median period of 6.2 years. Treatment decisions after targeted PAH-medication initiation were based mainly on clinical assessment. To counteract disease progression, an escalation toward combination therapy was observed during the study course. We consider survival rates under targeted PAH medication to be favorable, particularly in ES. Nevertheless, further research is needed to optimize the use of PAH medication, especially in patients with complex CHD.

Publication History

Article published online:
28 January 2023