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Eisenmenger syndrome (ES) is a known complication of congenital heart disease associated with unrepaired systemic to pulmonary shunts. Evidence for use of targeted pulmonary arterial hypertension therapy in ES is limited. The early experience using ambrisentan was evaluated in a cohort of consecutive patients with ES who initiated ambrisentan at Columbia University''s Pulmonary Hypertension Center from January 1, 2007, to August 1, 2008. Effects of ambrisentan on rest and exercise systemic arterial oxygen saturation (SaO2), exercise capacity, functional status, hemoglobin levels, and hemodynamics were evaluated and compared using paired Student''s t tests. Seventeen patients were evaluated at short-term (mean 163 ± 57 days) and longer term (mean 2.5 ± 0.5 years) follow-up. At short-term follow-up, there was an improvement in exercise capacity (6-minute walking distance 389 ± 74 vs 417 ± 77 m, p = 0.03, n = 11) and maintenance of rest SaO2 (89 ± 7% vs 89% ± 6%, p = 0.75, n = 15), exercise SaO2 (75 ± 15% vs 77% ± 15%, p = 0.33, n = 11), functional class (improvement in 2 patients, no change in 13), and hemoglobin (16.5 ± 2.8 vs 15.8 ± 1.8 g/dl, p = 0.11, n = 14). At longer term follow-up compared to baseline and short-term follow-up, there was stability of exercise capacity, SaO2, functional class, and hemoglobin. In conclusion, in this single-center cohort of patients with ES, ambrisentan was safe and was associated with increasing exercise capacity at short-term follow-up, with patients maintaining SaO2, functional class, and hemoglobin, and with no significant evidence of clinical deterioration at longer term follow-up. Additional studies are required to further assess the efficacy of ambrisentan in patients with ES. [Copyright &y& Elsevier]

Before 2001, intravenous epoprostenol was the only approved drug for patients with idiopathic pulmonary arterial hypertension (IPAH) or familial pulmonary arterial hypertension (FPAH) who were nonresponsive to high-dose calcium channel blockade. The investigators report transitioning select pediatric patients with IPAH or FPAH from intravenous epoprostenol to oral and/or inhaled agents for pulmonary arterial hypertension. A retrospective review was performed on all pediatric patients with IPAH or FPAH treated at Columbia University (1987 to 2008) who transitioned off epoprostenol to oral or inhaled drugs. Criteria for transition included functional class I or II, mean pulmonary arterial pressure <35 mm Hg, normal cardiac index, and age >6 years. Hemodynamic and clinical data were obtained at baseline (before epoprostenol), at peak epoprostenol dose, and after epoprostenol discontinuation. Fourteen of 104 pediatric patients with IPAH or FPAH transitioned off epoprostenol to oral or inhaled drugs from April 2003 to July 2008. Of the 14 subjects, 13 transitioned off epoprostenol successfully to oral or inhaled drugs. No significant changes in functional class, hemodynamics, or exercise data were seen after epoprostenol (mean follow-up duration 7.0 ± 5.8 months) compared to peak epoprostenol, but further improvement was reported in World Health Organization functional class (p <0.005) after epoprostenol discontinuation. After successful epoprostenol discontinuation, 77% of patients were treated with endothelin receptor antagonists, 69% with phosphodiesterase-5 inhibitors, 38% with calcium channel blockers, and 8% inhaled iloprost. At the cut-off date (May 2009), there was 100% survival and 93% transition success. In conclusion, in carefully selected children with IPAH or FPAH initiated on intravenous epoprostenol before the availability of nonparenteral therapy, transition to oral or inhaled therapy for pulmonary arterial hypertension appears safe, with efficacy maintained when performed with close follow-up at a pulmonary hypertension specialty center. [Copyright &y& Elsevier]