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Clinical efficacy and safety of switch from bosentan to macitentan in children and young adults with pulmonary arterial hypertension

Published online by Cambridge University Press:  13 December 2017

Ebru Aypar ,
Dursun Alehan ,
Tevfik Karagöz ,
Hayrettin Hakan Aykan  and
İlker Ertugrul
Ebru Aypar*
Affiliation:
Department of Pediatric Cardiology, Hacettepe University, Sihhiye, Ankara, Turkey
Dursun Alehan
Affiliation:
Department of Pediatric Cardiology, Hacettepe University, Sihhiye, Ankara, Turkey
Tevfik Karagöz
Affiliation:
Department of Pediatric Cardiology, Hacettepe University, Sihhiye, Ankara, Turkey
Hayrettin Hakan Aykan
Affiliation:
Department of Pediatric Cardiology, Hacettepe University, Sihhiye, Ankara, Turkey
İlker Ertugrul
Affiliation:
Department of Pediatric Cardiology, Hacettepe University, Sihhiye, Ankara, Turkey
*
Correspondence to: Department of Pediatric Cardiology, Hacettepe University, Ankara, Såhhåye, Turkey. Tel: +90 (312) 305 11 57; Fax: +90 (312) 324 49 90; E-mail: [email protected]
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Abstract

Background

Macitentan is an orally active, potent, dual endothelin receptor antagonist and is the only registered treatment for pulmonary arterial hypertension that significantly reduced morbidity and mortality in a long-term event-driven study.

Aim

Few studies compared the clinical efficacy and safety of switch from bosentan to macitentan only in adult patients with pulmonary arterial hypertension. We aimed to evaluate the clinical efficacy and safety of switch from bosentan to macitentan in children and young adults.

Methods

This is a single-institution, 24-week prospective study. Patients ⩾12 years of age with idiopathic/heritable pulmonary arterial hypertension or related to CHD or residual pulmonary arterial hypertension due to repaired congenital systemic-to-pulmonary shunts and on bosentan therapy were included. Concomitant treatment with oral phosphodiesterase type 5 inhibitors and inhaled prostanoids was allowed. Outcome measures included change from baseline to week 24, in the 6-minute walk distance, functional class, oxygen saturation at rest/after 6-minute walk distance test, systolic pulmonary artery pressure estimated by echocardiography, and brain natriuretic peptide levels. Safety end points included adverse events laboratory abnormalities.

Results

A total of 13 patients – 5 male and 8 female – completed the study. The mean age was 20.3±6.5 years (12–35) and weight was 54.0±14.5 kg (27–75). Five patients were ⩽18 years of age. Macitentan improved 6-minute walk distance from baseline (mean: 466±35 m (300–590)), at 12 weeks (mean: 494±78 m (325–590), +28 m) (p<0.05), and at 24 weeks (mean: 507±58 m (325–625), +41 m) (p<0.05). Macitentan did not significantly change functional class, oxygen saturation at rest/after 6-minute walk distance test, brain natriuretic levels, and systolic pulmonary artery pressure (p>0.05). None of the patients had anaemia, hepatotoxicity, and peripheral oedema.

Conclusions

Our study is the first study that showed that switch from bosentan to macitentan significantly improved exercise capacity in children and young adults with pulmonary arterial hypertension and is well tolerated without any adverse events.

Keywords

Bosentanchildrenendothelin receptor antagonistmacitentanpulmonary arterial hypertension
Type
Original Articles
Information
Cardiology in the Young , Volume 28 , Issue 4 , April 2018 , pp. 542 - 547
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Copyright
© Cambridge University Press 2017 

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