| First author [Ref.] | Patients n | Description | Age yrs | End-points | Results |
| Barst [33] | 19 | Pharmacokinetics and efficacy of bosentan investigated | NS (3–15) | PharmacokineticsHaemodynamics | Pharmacokinetics of bosentan in children with PAH comparable to healthy adults |
| 6MWD | Improvement in haemodynamics | ||||
| WHO FC | WHO FC improved or stabilised | ||||
| Beghetti [21] | 36 | Pharmacokinetic study of novel paediatric formulation of bosentan | 7.0 (2–11) | Pharmacokinetics | An exposure plateau appears to be reached in children at the dose of 2mg·kg−1 |
| Gilbert [34] | 7 | Uncontrolled study of bosentan in patients with PAH-CHD | 3.8# (1.5–6.4) | Clinical parametersEchocardiography | Significant reduction in right ventricular systolic pressure |
| Haemodynamics | |||||
| Simpson [36] | 7 | Open-label study of children with IPAH treated with bosentan, compared with historical controls | NS | Survival | Improved survival and delayed disease progression |
| Addition of epoprostenol due to disease progression | |||||
| Rosenzweig [35] | 86 | Long-term retrospective study of patients treated with bosentan over median exposure 14 months | 11 (0.8–18) | WHO FC | 46% improved WHO FC |
| Haemodynamics | Improved haemodynamics | ||||
| Survival | |||||
| Maiya [20] | 40 | Long-term retrospective study of children with IPAH and PAH-CHD treated with bosentan | 8.3 (0.6–16) | WHO FCStabilisation of the | Children with IPAH stabilised (with addition of epoprostenol) |
| clinical condition6MWD | WHO FC improved significantly for patients with PAH-CHD | ||||
| van Loon [37] | 10 | Long-term response to bosentan in children compared to adults | 11.8 (4.7–17.3) | WHO FC | Improved 6MWD |
| Transcutaneous oxygen saturation | Survival not significantly different between adults and children | ||||
| Heart rate and blood pressure | Persistence of treatment effect lower in children | ||||
| 6MWD | Improvement in 6MWD at 1 yr but then declined | ||||
| Survival | WHO FC improvement persisted over 1.5 yrs | ||||
| Persistence of treatment effect |
Data for age presented as mean (range), unless otherwise indicated. #: median (range); CHD: congenital heart disease; IPAH: idiopathic PAH; NS: not specified; 6MWD: 6-min walk distance; WHO FC: World Health Organization functional class.