Performing a 6MWT is feasible and practical in young children. This study provides data on normal children against which the performance of sick children and the response to therapeutic intervention can be judged.

Objective: To describe an early experience of treating 40 children with the dual endothelin receptor antagonist bosentan, which is known to be safe and effective in adults with pulmonary hypertension (PH). Design: In this retrospective, observational study the UK Service for Pulmonary Hypertension for children treated 40 children with bosentan, 20 with idiopathic pulmonary arterial hypertension (IPAH) (mean age 8.03 years, range 1.2-17) and 20 with PH associated with other conditions (congenital heart disease, parenchymal lung or connective tissue disease, or HIV). Their mean age was 8.3 years (range 0.6-16 years). Patients: 39 patients were in World Health Organization (WHO) class III and IV, and all had shown recent deterioration. In IPAH the mean pulmonary vascular resistance (PVR) was 21.7 units?m 2 (range 5.6-42.8). In secondary PH the mean PVR was 18 units?m 2 (range 4.9-49). No child had a positive response to vasodilator testing with nitric oxide. Interventions: Bosentan was given as first line treatment to 25. Nine were given intravenous epoprostenol. Children were treated for a mean of 12.7 months (range 2-24 months). Main outcome measures: Response to treatment was judged by WHO functional class, six minute walk test, weight, ECG and echocardiographic findings, and need to add additional treatment. Results: Bosentan was well tolerated. In the IPAH group 19 (95%) stabilised with bosentan treatment but 12 (60%) patients needed combined treatment with epoprostenol. In secondary PH, WHO class, six minute walk test, and weight gain improved significantly. Conclusion: Bosentan helped stabilise children with IPAH but intravenous epoprostenol was also needed by 60%. Children with secondary PH improved.

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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).

Aim: To present the experience of the UK Pulmonary Hypertensive Service for Children at Great Ormond Street Hospital in treating children with “Eisenmenger” type physiology with the dual endothelin receptor antagonist bosentan. Patients and methods: Fourteen inoperable patients were included in the study. These were selected based on a history of an unrestrictive intracardiac communication and established right to left shunt at rest. There was a female: male ratio 1: 0.75 and age range at initiation of treatment of 7–18.6 years (mean 12.5 yrs). Six patients had Trisomy 21. The cardiac diagnoses were: 6 large VSDs, 6 CAVSD (1 of which was not Trisomy 21) and 2 more complex congenital heart disease cases that underwent palliative surgery (DILV, DOLV, TGA, PA banding and DORV, TGA, VSD, PS, Waterston). Twelve patients received antiplatelet/anticoagulation therapy. WHO functional class, haemoglobin (Hb), oxygen saturation, 6 min walk tests, echocardiograms and ECGs were assessed before starting treatment with bosentan and after 6 and 12 months of follow up. Twelve patients had a cardiac catheterisation before treatment commenced. The parameters were compared for patients before treatment and at 6 and 12 months post treatment with paired t-test and ANOVA. Results: All patients were severely symptomatic the mean WHO functional class being 3.2 (range 3–4). The mean Hb was 18.3 g/dl (range 13.4–22.9). The mean PVR of all patients was 30.98 units m2 (7.6–57). The Trisomy 21 patients had a mean PVR of 34.4 units m2 and non Trisomy 21 patients had a mean of 28.6 units m2. Mean oxygen saturation at rest was 78.9% (range 67–93), mean 6 min walk test distance was 223.8 m (n = 10, range 108–360) and the lowest oxygen saturation during the walk test was 59.7% (42–79) at 6 min or below. Mean duration of therapy was 15.2 months (range 6–34 months). Bosentan was well tolerated in all patients but discontinued in one due to non compliance with monthly screening blood tests. All patients remained stable and their echocardiogram and ECG findings were unchanged throughout. After 6 months mean WHO functional class had decreased significantly to 2.96 (p = 0.029), whilst Hb and oxygen saturation at rest had not changed. Ten of the 14 patients were on treatment for more than a year and after 12 months the WHO functional class was maintained at the same improved level. The Hb and oxygen saturation at rest remained unchanged. After 12 months the 6 min walk mean distance had increased from 211 to 253 metres (non significant). Conclusion: Patients treated with bosentan for up to 1 year showed clinical improvement and remained stable. After 6 months treatment WHO functional class had significantly improved and improvement was sustained for up to 1 year.