Pulmonary arterial hypertension associated with a congenital heart defect: advanced medium-term medical treatment stabilizes clinical condition

OBJECTIVE: Pulmonary arterial hypertension (PAH) associated with congenital heart defect (CHD), and especially Eisenmenger syndrome, is associated with impaired exercise tolerance and reduced quality of life. In this study, we describe medium-term follow-up of adult patients with PAH associated with CHD, treated in a single center with different types of advanced medication. DESIGN: The treatment and clinical course of 15 patients (11 female, median age 53, range 28-74 years) with PAH associated with CHD is retrospectively described. Data on patient characteristics, exercise test, right-heart catheterization, and type of advanced therapy were collected from medical files. Advanced medical therapy consisted of either intravenous prostacyclin, or endothelin receptor antagonists, or phosphodiesterase-5-inhibitors. Additional therapy was started in case of persistent clinical deterioration or insufficient improvement with monotherapy. RESULTS: All patients (10 patients with Eisenmenger syndrome, 5 patients with a closed defect and PAH) were exposed to different durations of advanced medication. Median period of treatment was 2.5 (range 0.7-6.3) years. Atrial septal defect, type secundum, was the most frequent underlying diagnosis (n = 10). Most patients (n = 9) received a combination of advanced medical therapy. Six-minute walk distance (6-MWD) remained unchanged with an increase of 44 +/- 78 m (P = 0.2) and 41 +/- 80 m (P = 0.3) compared with baseline after respectively 1 and 2 years of treatment. Younger age was associated with better performance (beta = -7 m per year, P < 0.05), patients younger than 45 years showed a greater improvement in 6-MWD after 2 years of treatment (P < 0.05). During a mean follow-up of 23 (range 4-58) months, mean pulmonary arterial pressure (53 +/- 24-49 +/- 17 mmHg, P = 0.3) and pulmonary vascular resistance (770 +/- 1090-650 +/- 444 dynes s/cm5, P = 0.7) showed no deterioration. CONCLUSION: Advanced treatment strategies in patients with PAH associated with CHD are useful. The treatment effect seems to be one of disease stabilization and decreasing the rate of deterioration. Younger age was associated with a greater improvement of 6-MWD