Background: Continuous intravenous Epoprostenol treatment has been shown to be beneficial in patients with severe pulmonary arterial hypertension (PAH). We report the experience of the UK Pulmonary Hypertension Service for Children treating 39 children with this therapy. Patients and methods: The patients were aged 4 months to 17 years (median 5.4 years), male: female ratio was 1: 1.2. Eleven children were treated with Epoprostenol alone. Twenty-eight were also treated with Sildenafil, Bosentan, Nifedipine or a combination of these. Twenty-five children had idiopathic PAH and 14 had PAH associated with congenital heart disease (9), connective tissue disease (2), chronic lung disease (2) or HIV (1). Cardiac catheter studies made before treatment began showed that in 26 patients the pulmonary artery pressure equalled or exceeded the systemic pressure. Thirty-four patients had a pulmonary vascular resistance (PVR) study with a mean PVR of 23 ± 12 units m2. After initial evaluation children were monitored by ECG, transthoracic echocardiography, weight, 6-minute-walk test and WHO functional class. Results: Follow-up was 1–90 months (mean 27). Twenty-four children remain stable and well, 8 have been transplanted and 7 died on treatment. There was no deterioration in the ECG; the 6-minute walk distance and right ventricular function assessed by echocardiography remained stable. WHO functional class improved from 3.6 to 2.6 during the first year (n = 38) (p < 0.001) and remained stable for up to 3 years (n = 17). Weight improved significantly (p < 0.05). A Kaplan Meier survival curve showed that survival was 94.4% at 1 year, 90.3% at 2 years and 83.9% at 3 years.

Conclusions: Epoprostenol treatment improved survival, clinical status and WHO functional class in children with severe PAH. Survival in the children on intravenous Epoprostenol with and without combination therapy was better than that described in adult studies (McLaughlin VV, et al. Circulation 2002; 106: 1477–1482).