Background: Patients with pulmonary arterial
hypertension (PAH) are managed according to
evidence-based treatment guidelines.
Methods and results: In this single-centre
retrospective analysis, we examined outcomes of
patients with PAH caused by congenital heart disease
(PAH-CHD) with respect to exercise capacity and
survival of adults treated with either bosentan or
sildenafil monotherapy or bosentan-sildenafil dual
therapy between January 2007 and January 2014. Of
the 82 patients analysed, 29 had Down syndrome; 54
(65.8%) received bosentan monotherapy, 16 (19.5%)
sildenafil monotherapy and 12 (14.6%) dual therapy.
Mean treatment duration was 2.5 years for all patients
and 4.1 years for 38 patients treated for ≥2 years.
Pooled patient and treatment data showed initial
improvement followed by stabilisation in mean 6 min
walk distance (6MWD). For Down and non-Down
patients, mean 6MWD increased and then stabilised on
bosentan monotherapy. Mean 6MWD of patients on
dual therapy at the time of analysis was 246.3 m
before PAH-specific therapy initiation, 211.9 m
immediately prior to addition of a second therapy and
214.4 m at last visit while on dual therapy. 1, 2 and 3-
year survival rates for all patients from time of
treatment initiation were 96%, 87% and 80%,
respectively.
Conclusions: For the majority of patients,
monotherapy with a PAH-specific medication provided
improved and sustained exercise benefits. For the small
percentage of patients who required it, add-on therapy
appeared to prevent further deterioration in exercise
capacity but did not improve 6MWD.
CORE (COnnecting REpositories)
CORE (COnnecting REpositories)