Results of the single-center study EvalUation of Pulmonary Hypertension Risk factors AssociaTEd with Survival (EUPHRATES) support using macitentan (Opsumit®) to treat idiopathic pulmonary arterial hypertension (PAH) and congenital heart disease-associated PAH.

“Mono- or sequential combination macitentan therapies were associated with significant and sustained benefits in clinical, neurohumoral, and echocardiographic measures of pulmonary hemodynamics and right ventricular function without any signal suggesting the failure of macitentan therapies in congenital heart disease-associated PAH subgroup,” the authors wrote in the Anatolian Journal of Cardiology.

Treatment with macitentan in mono- or sequential combination therapies led to sustained improvements in functional class, 6-minute walk distance test, N-terminal pro-brain natriuretic peptide (NT-proBNP), echocardiographic measures of pulmonary hemodynamics, and right ventricular longitudinal function. Improvements were noted regardless of disease etiology.

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Furthermore, the authors found that patients classified as low risk in risk model assessment at baseline and at first control, ie, 3 to 6 months after macitentan initiation, had improved survival compared to patients at moderate or high risk.

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A Cox proportional hazards regression model identified age, gender, and log-NT-proBNP as independent predictors of mortality in time-fixed analyses, as well as idiopathic PAH and log-NT-proBNP in time-dependent analyses. The levels of hemoglobin and hepatic enzymes remained stable throughout the follow-up period.

The study enrolled a subgroup of 221 patients with PAH from the EUPHRATES study. Most patients were given dual (52%) or triple (34%) sequential combination macitentan therapies, while 13.6% were given macitentan monotherapy. Patients were followed up for a median period of 1068 days.

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The authors pointed out some limitations to the study, including its retrospective nature and the lack of randomization between macitentan and other endothelin receptor antagonist therapies.


Kaymaz C, Tanyeri S, Tokgoz HC, et al. The consistent effectiveness and safety of macitentan therapies across idiopathic and congenital heart disease-associated pulmonary arterial hypertension: a single-center experience. Anatol J Cardiol. 2022;26(10):778-787. doi:10.5152/AnatolJCardiol.2022.1889