Background: We performed a retrospective analysis of patients with pulmonary arterial hypertension receiving inhaled iloprost in a single centre to evaluate long-term tolerability, safety, and efficacy of chronic inhaled iloprost therapy in children.
Methods: A total of 20 patients with either idiopathic or associated pulmonary arterial hypertension were treated with iloprost between April, 2003 and January, 2010. The median age and weight of the patients were 3.8 years--ranging from 4 months to 19 years--and 12.3 kilograms--ranging from 4 to 73 kilograms-- respectively. Pulmonary arterial hypertension was idiopathic or hereditary in eight patients (40%) and associated with congenital cardiac disease in 12 patients (60%).
Results: Of the 20 patients, 15 had combined therapy--12 patients with two and three patients with three different classes of drugs. In all, six patients died during follow-up. The median follow-up time was 18 months, ranging from 6 to 74 months. The 6-minute walking test was performed in 7 out of 20 patients at baseline and on follow-up. The median 6-minute walking test increased from 420 to 490 metres after iloprost therapy (p = 0.028). After initiation of iloprost therapy, one patient complained of headache and another had a rash around his mouth, none necessitating discontinuation of therapy. Overall compliance with inhaled iloprost was good.
Conclusion: Pulmonary hypertension is associated with significant morbidity and mortality. Careful assessment of each patient and timely combination of specific vasodilator therapy is needed to improve clinical outcomes. This study suggests that inhaled iloprost, with or without concomitant endotelin receptor antagonist and/or phosphodiesterase inhibitor, is safe and efficacious for treatment of pulmonary arterial hypertension in children.