Experience with inhaled iloprost in paediatric pulmonary arterial hypertension

Background: Inhaled iloprost has been approved for the treatment of Pulmonary Arterial Hypertension (PAH) in adults, but there are few studies about its effects in children. Objectives: To review long-term effects and outcomes of inhaled iloprost treatment in children with PAH. Methods: Retrospective review of clinical and haemodynamic evolution in children with PAH after inhaled iloprost therapy. Results: Between 2000-2010 25 patients with PAH were treated with inhaled iloprost (17 female; range 3 months to 12-years-old): 11 idiopathic, 1 pulmonary veno-occlusive disease, 4 associated with congenital heart disease, 7 lung disease, 1 schistosomiasis and 1 congenital metabolic disease. Clinical and haemodynamic parameters at baseline were: WHO class II in 3, III in 16, IV in 6, and median of mean pulmonary arterial pressure 56 mmHg (range 29-90 mmHg). Iloprost was started as initial therapy in 14 patients: 8 as monotherapy and 6 as combined treatment, and in 11 cases was added during the follow-up. Mean dose was 50 μg/day. Median follow-up was 3.6 years (range 1 month to 10 years). There were no serious side effects but facial flushing was often observed in those using a mask. During the follow-up iloprost was discontinued in 1 patient due to abdominal pain, 1 patient was lost, 12 patients improved their functional class, 3 remained stable, 3 received a lung transplant, 1 a heart-lung transplant and 4 died. Conclusions: Inhaled iloprost is well tolerated in children, both as monotherapy and as a combined treatment, with minimal side effects. Although uncontrolled, data suggest long-term clinical benefit from continued therapy in 60% of patients.