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Response to bosentan in children with pulmonary hypertension
1Great Ormond Street Hospital for Children, University College London, London, UK.
Insights
Bosentan stabilized children with idiopathic pulmonary arterial hypertension (IPAH), though 60% required additional intravenous epoprostenol. Children with secondary pulmonary hypertension (PH) showed significant improvements with bosentan treatment.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Pharmacological Interventions
Background:
- Pulmonary hypertension (PH) in children presents unique challenges.
- Bosentan, a dual endothelin receptor antagonist, is established for adult PH treatment.
- Early pediatric data on bosentan's efficacy and safety is crucial.
Purpose of the Study:
- To evaluate the early clinical experience of using bosentan in pediatric PH patients.
- To assess bosentan's safety and effectiveness in children with idiopathic pulmonary arterial hypertension (IPAH) and secondary PH.
- To describe treatment outcomes and identify potential adjunctive therapies.
Main Methods:
- Retrospective observational study of 40 children treated with bosentan.
- Patients included those with IPAH and PH associated with congenital heart disease, lung, connective tissue, or HIV.
- Treatment response assessed via WHO functional class, 6-minute walk test, weight, ECG, echocardiography, and need for additional therapies.
Main Results:
- Bosentan was well-tolerated in the pediatric cohort.
- Ninety-five percent of IPAH patients stabilized on bosentan, with 60% requiring supplemental intravenous epoprostenol.
- Children with secondary PH demonstrated significant improvements in WHO class, 6-minute walk test, and weight gain.
Conclusions:
- Bosentan effectively stabilizes children with IPAH, often in conjunction with other therapies like epoprostenol.
- Bosentan treatment led to notable improvements in children with secondary PH.
- This study highlights bosentan as a viable therapeutic option in pediatric pulmonary hypertension management.
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.m2 (range 5.6-42.8). In secondary PH the mean PVR was 18 units.m2 (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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