Iloprost for the Treatment of Pulmonary Hypertension in Adults With Congenital Heart Disease

Pulmonary arterial hypertension (PAH), or high blood pressure in the lungs, is common in
patients with congenital heart disease. Historically these patients suffered significant
morbidity and mortality due to a lack of effective therapies. More recently, advanced
therapies which target the mechanisms underlying the development and progression of PAH have
been introduced into clinical care. Oral, intravenous, subcutaneous, and inhaled therapies
are all available for the treatment of PAH. Patients with PAH are first treated with oral
agents (including sildenafil and bosentan). However, if these agents fail to achieve the
desired effect for the patient, intravenous or inhaled therapies may be initiated.
Combination therapy with multiple agents is common in routine clinical care. However, the
most efficacious therapeutic regimen has yet to be delineated. The present study seeks to
evaluate the efficacy of one specific regimen: iloprost, an inhaled prostacyclin derivative,
used in combination with oral therapy (sildenafil and/or bosentan). Iloprost has been
approved by the FDA for use in this patient population. Adults with PAH already receiving
oral therapy will be invited to participate in this study. Iloprost will be added to their
current therapeutic regimen for a period of three months, with pre- and post-treatment
assessments. These will include a cardiopulmonary exercise test, BNP (a blood test), six
minute walking distance, and a quality of life questionnaire.