Title:Targeted Therapies in Patients with Pulmonary Arterial Hypertension
Due to Congenital Heart Disease
Volume: 20
Issue: 4
Author(s): Adriana Mares, Debabrata Mukherjee, Richard A. Lange and Nils P. Nickel*
Affiliation:
- Department of Internal Medicine, Division of Cardiology, Texas Tech University Health Sciences Center, El Paso,
Texas, 79905, USA
- Department of Internal Medicine, Division of Pulmonology and Critical Care Medicine, Texas
Tech University Health Sciences Center, El Paso, Texas, 79905, USA
Keywords:
Congenital heart disease, pulmonary arterial hypertension, targeted therapies, Eisenmenger syndrome, repaired congenital defects, PVR.
Abstract:
Pulmonary arterial hypertension (PAH) is a devastating cardiovascular disease leading to
right heart failure and death if untreated. Medical therapies for PAH have evolved substantially over the
last decades and are associated with improvements in functional class, quality of life, and survival.
PAH-targeted therapies now consist of multiple inhaled, oral, subcutaneous, and intravenous therapies
targeting the phosphodiesterase, guanylate cyclase, endothelin and prostacyclin pathways.
Patients with congenital heart disease (CHD) are at high risk of developing PAH and growing evidence
exists that PAH-targeted therapy can be beneficial in PAH-CHD. However, the PAH-CHD patient population
is challenging to treat due to the heterogeneity and complexity of their cardiac lesions and associated
comorbidities. Furthermore, most high-quality randomized placebo-controlled trials investigating
the effects of PAH-targeted therapies only included a minority of PAH-CHD patients. Few randomized,
controlled trials have investigated the effects of PAH-targeted therapy in pre-specified PAH-CHD populations.
Consequently, the results of these clinical trials cannot be extrapolated broadly to the PAH-CHD
population.
This review summarizes the data from high-quality clinical PAH treatment trials with a specific focus
on the PAH-CHD population.
