Long-term course of pulmonary arterial hypertension in adults with congenital heart disease under targeted therapy: a retrospective analysis of a single tertiary center

Abstract

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. Methods:In this cross-sectional study we retrospectively examined clinical, demographic, and cardiaccatheterization data and medical management for PAH-CHD, and analyzed clinical course and midterm outcome.Results: Over up to 14 years (median, 6.2 years), 103 PAH-CHD patients (66% female) receiving targeted PAH-therapy for pre-tricuspid-shunt (15.5%), post-tricuspid-shunt (32.0%), and complex CHD (52.4%) were followed. Based on modified clinical European Society of Cardiology (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%). Changes in targeted PAH therapy were observed 249 times, with up to 6 (median, 2) therapy changes over a median period of 1.3 years. Over the study course, the medical treatment strategy changed towards 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 systemicto-pulmonary shunt or ES showed the best survival rates. Mortality was remarkably higher in patients with severe PVD in complex CHD and Fontan patients.Conclusions: 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 towards combination therapy was observed during the study course. We consider survival rates under targeted PAH medication to be favorable, particularly in the ES subgroup. Nevertheless, further research is needed to optimize the use of PAH medication, especially in patients with complex CHD.

Invasive Hemodynamic and Vasoreactivity Testing with Inhaled Iloprost in Children with Pulmonary Arterial Hypertension Associated with Congenital Heart Defects

“…those with PAH-CHD. [2,3] However, patients with severe pulmonary vascular disease (PVD), particularly Eisenmenger syndrome, still have considerable mortality and morbidity, and their prognostic evaluation and management are challenging. [4] Acute vasoreactivity testing (AVT) and reversibility of pulmonary vascular resistance (PVR) are often used as indicators to determine defect correction in patients with elevated PVR in CHD.…”

“…[1] Improvements in current PAH therapies such as prostacyclin, endothelin receptor antagonists (ERAs), and phosphodiesterase type-5 (PDE-5) inhibitors have become available and are used to successfully treat patients with PAH, including those with PAH-CHD. [2,3] However, patients with severe pulmonary vascular disease (PVD), particularly Eisenmenger syndrome, still have considerable mortality and morbidity, and their prognostic evaluation and management are challenging. [4] Acute vasoreactivity testing (AVT) and reversibility of pulmonary vascular resistance (PVR) are often used as indicators to determine defect correction in patients with elevated PVR in CHD.…”

Healthcare status of adults with pulmonary hypertension due to congenital heart disease

“…Medical records were reviewed for healthcare-related information, sociodemographic parameters, relevant VSD [13] Ebstein anomaly [29] d-TGA [81] d-TGA [7] PS [27] CoA [71] PA + VSD [5] Aortic aneurysm [14] TOF [69] ASD [4] MVPS [13] VSD [56] AVSD [4] IAA [1] ASD [49] ccTGA [2] ccTGA [27] Mitral valve disease [2] Connective tissue disease [23] CoA [2] AVSD [19] Ebstein anomaly [2] TA [17] PAPVC [2] PA + VSD [15] TOF [2] DIV [12] TA [2] Mitral valve disease [12] DIV [1] Cardiomyopathy, congenital [11] DORV (Fallot-Type) [1] PDA [10] DORV + TGA [1] AVSD, partial [9] AVSD, partial [1] Subaortic stenosis [9] PA + IVS [8] PAS [7] DORV (Fallot-Type) [7] TAC [6] DORV + TGA [5] HCM [4] PAPVC [4] TAPVC [4] HLHS [1] Others* [12] *…”

“…There is a gap in knowledge regarding the awareness level of patients and their primary care providers about their own disease and, the appropriate level of care needed. In addition, there is a great need for targeted medical advice (10)(11)(12).…”