New treatment opportunities in pulmonary hypertension and congenital heart disease

Ilja Mark Blok, Annelieke van Riel, Mark J. Schuuring, Arie van Dijk, Barbara Mulder, Berto Bouma

Research output: Contribution to journalMeeting AbstractAcademic


Background: Endothelin receptor antagonists are regarded a cornerstone in treatment of pulmonary arterial hypertension associated with congenital heart disease (PAH-CHD). Currently macitentan, a potential successor of the widely applied bosentan, is being introduced. A recent trial has proven its outstanding effects on exercise capacity and mortality, compared to placebo. However a direct clinical comparison with bosentan is lacking. Using our large longitudinal cohort of PAH-CHD patients, we aim to provide insight in potential benefits of switching bosentan to macitentan.

Methods: In this multicenter prospective cohort study, all adult PAH-CHD patients on bosentan are scheduled to switch to macitentan with continuation of study protocol measurements. These include laboratory tests, 6-minute walk tests (6-MWT), cardiopulmonary exercise tests, echocardiography, MRI and quality of life questionnaires at regular intervals during follow-up. All measurements will be compared with previously collected prospective data during bosentan treatment using paired samples t-tests.

Results: From 2006 a total of 84 PAH-CHD patients started bosentan, of whom 44 patients are currently still alive (mean age 46±14 years, follow-up 6.2±2.6 years, 46% male, 36% Down syndrome). Currently 25 patients were switched to macitentan. No serious adverse events were reported. Three months macitentan treatment resulted in improved 6-MWT (429±110 to 438±122 meters, mean values) and resting saturation (83 to 86%, median values) although non-significant. However macitentan reduced NT-pro-BNP (1143 to 804 ng/L, median values, p=0.05) and high sensitive Troponin-T (0.016 to 0.010 µg/L, median values, p=0.01). These and additional results after treatment switch will be presented.

Conclusion: This is the first study to provide important clinical and safety information on the effect of switching bosentan to macitentan in adult PAH-CHD patients. After three months of macitentan treatment in the first 25 patients significant declines in cardiac markers NT-pro-BNP and Troponin-T are already apparent.
Original languageEnglish
Pages (from-to)A564-A564
JournalJournal of the American College of Cardiology
Issue number10, Suppl.
Publication statusPublished - 2015
Externally publishedYes
Event64th American College of Cardiology (ACC) Annual Scientific Session 2015 - San Diego Convention Center, San Diego, United States
Duration: 14 Mar 201516 Mar 2015
Conference number: 64


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