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Published on: May 11, 2015
Effects of long-term bosentan in children with pulmonary arterial hypertension
Erika Berman Rosenzweig1, D Dunbar Ivy, Allison Widlitz
1Division of Pediatric Cardiology, New York Presbyterian Hospital, New York, New York 10032, USA. esb14@columbia.edu
Insights
Bosentan therapy is safe and effective for treating pediatric pulmonary arterial hypertension (PAH), improving functional class and survival rates. Long-term outcomes show sustained benefits with this endothelin receptor antagonist.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Pharmacological Interventions
Background:
- Pulmonary arterial hypertension (PAH) in children requires effective long-term treatment.
- Bosentan, an endothelin receptor antagonist, shows promise but requires further pediatric data.
- Limited information exists on the long-term efficacy and safety of bosentan in pediatric PAH patients.
Purpose of the Study:
- To evaluate the long-term outcomes of pediatric patients with pulmonary arterial hypertension (PAH) treated with bosentan.
- To assess the safety and efficacy of bosentan, with or without prostanoid therapy, in children with PAH.
- To analyze the impact of bosentan on hemodynamics, functional class, and survival in pediatric PAH.
Main Methods:
- Retrospective analysis of 86 children with PAH receiving bosentan, with or without prostanoids.
- Collection of hemodynamic data, World Health Organization (WHO) functional class, and safety information.
- Kaplan-Meier survival analysis and comparison of outcomes based on initial WHO functional class.
Main Results:
- Bosentan treatment was continued in 79% of patients, with a median exposure of 14 months.
- 90% of patients experienced improved or unchanged WHO functional class; hemodynamics significantly improved.
- One- and two-year survival estimates were 98% and 91%, respectively, with lower risk in earlier functional classes.
Conclusions:
- Bosentan, with or without prostanoid therapy, is a safe and effective treatment for pediatric PAH.
- The study supports the long-term use of bosentan in children with pulmonary arterial hypertension.
- Early initiation of bosentan in less severe functional classes is associated with better outcomes.
Objectives:
This study investigated the long-term outcome of children with pulmonary arterial hypertension (PAH) treated with bosentan therapy, with or without concomitant prostanoid therapy.
Background:
Bosentan, an oral endothelin ET(A)/ET(B) receptor antagonist, improves hemodynamics and exercise capacity in adults with PAH; however, limited data are available on its long-term effects in children.
Methods:
In this retrospective study, 86 children with PAH (idiopathic, associated with congenital heart or connective tissue disease) started bosentan with or without concomitant intravenous epoprostenol or subcutaneous treprostinil therapy. Hemodynamics, World Health Organization (WHO) functional class, and safety data were collected.
Results:
At the cutoff date, 68 patients (79%) were still treated with bosentan, 13 (15%) were discontinued, and 5 (6%) had died. Median exposure to bosentan was 14 months. In 90% of the patients (n = 78), WHO functional class improved (46%) or was unchanged (44%) with bosentan treatment. Mean pulmonary artery pressure and pulmonary vascular resistance decreased (64 +/- 3 mm Hg to 57 +/- 3 mm Hg, p = 0.005 and 20 +/- 2 U x m2 to 15 +/- 2 U x m2, p = 0.01, respectively; n = 49). Kaplan-Meier survival estimates at one and two years were 98% and 91%, respectively. The risk for worsening PAH was lower in patients in WHO functional class I/II at bosentan initiation than in patients in WHO class III/IV at bosentan initiation.
Conclusions:
These data suggest that bosentan, an oral endothelin ET(A)/ET(B) receptor antagonist, with or without concomitant prostanoid therapy, is safe and efficacious for the treatment of PAH in children.
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