Introduction
Traditionally, treatment options for patients with pulmonary arterial hypertension
associated with congenital heart disease (PAH-CHD) are limited. Bosentan has been
shown to improve pulmonary haemodynamics and exercise tolerance short term but long
term clinical studies are lacking.
Aim
To report long term efficacy and safety data with endothelin receptor antagonists
(ERA) in patients with PAH associated CHD.
Methods
Prospective, open label, uncontrolled, single centre study of 53 patients (33 females,
17 Trisomy 21, mean age 34 ± 12 years) prescribed ERA (48 bosentan, 5 sitaxentan) from 2003 to August 2009. Outcome
measurements of oxygen saturation (SaO2), WHO functional class, 6-minute walk test
distance (6MWD) and adverse events were analysed.
Results
Mean duration of therapy was 15 ± 13 months in 53 patients with CHD. Four patients failed ERA, seven died (five progressive
RHF) and one delisted from transplantation. No abnormal liver transaminases occurred
on bosentan, with one case on sitaxentan. After 3, 6, 12, 18 and 24 months of treatment
a significant improvement was seen in WHO functional class (mean 3.15 vs 2.8 vs 2.5
vs 2.5 vs 2.4 vs 2.4; p < 0.01) and 6MWD (344 ± 18 vs 392 ± 17 vs 411 ± 17 vs 420 ± 17 vs 442 ± 18 vs 417 ± 23: p < 0.0005, p < 0.01) compared with baseline. The Trisomy 21 and PAH-CHD showed a significant improvement
in 6MWD at 6 and 12 months (263 ± 24 vs 348 ± 29 vs 360 ± 32, p < 0.01, p < 0.05) respectively. No changes in SaO2, BNP, RV or LV function were demonstrated during
follow-up.
Conclusion
This large single centre study demonstrates that endothelin receptor antagonism is
an effective and safe treatment in PAH associated CHD with or without Trisomy 21.
The improvements in exercise tolerance are similar to reported benefits in other forms
of PAH.
Keywords
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Article info
Publication history
Accepted:
July 8,
2010
Received in revised form:
July 5,
2010
Received:
January 22,
2010
Identification
Copyright
© 2010 Published by Elsevier Inc. All rights reserved.
