A. Rodriguez Ogando, A. Sabaté-Rotés, A. Moya Bonora, L. Albert de la Torre, J. M. Espín López, C. Labrandero de Lera, Maria Jesus Del Cerro Marin
Gregorio Marañon General University Hospital. Hospital Materno Infantil Gregorio Marañon. Vall d’Hebron University Hospital. La Fe University Hospital. 12 de Octubre University Hospital. Virgen dee la Arrixaca University Hospital. La Paz University Hospital. Ramón y Cajal University Hospital.
Spain
Pediatric Cardiology
Pediatr Cardiol 2025;
DOI: 10.1007/s00246-025-03937-y
Abstract
Macitentan seems to improve outcomes in patients with pulmonary arterial hypertension (PAH). Nevertheless, safety and efficacy in pediatric patients is still to be determined. Multi-center, observational study to assess safety and efficacy of oral macitentan in patients under 18 years-of-age from the Spanish Registry of Pediatric Pulmonary Hypertension with group 1 PAH. Outcome measures included changes in the World Health Organization Functional Class (WHO FC) from baseline to month 6, 6-min walking distance (6MWD), NT-proBNP levels, risk-stratification, echocardiographic and hemodynamic parameters, and Pediatric Quality of Life Inventory. Seventy-four patients (median age 9.6 years) with PAH were included in the study, of which thirty-one had idiopathic/heritable pulmonary arterial hypertension and 43 had PAH associated with congenital heart disease. Significant clinical improvement was observed after 6 months of treatment with macitentan: The percentage of patients on WHO FC III/IV decreased from 48 to 22% (p = 0.001); mean 6MWD (n = 42) increased significantly in around 45 m (p = 0.032); NT-proBNP decreased a mean of – 438.9 ± 175 pg/ml, (p = 0.015). There was a 17% increase in the number of patients meeting all three low-risk parameters of 17% (p = 0.002). Side effects were detected in four patients (5.4%). Macitentan was discontinued in one patient. Disease progression was observed in 6 patients despite macitentan therapy. This study provided evidence on the clinical benefits and good tolerance of macitentan in paediatric population with group I PAH treated in routine clinical practice.
Category
Class I. Idiopathic Pulmonary Hypertension
Class I. Heritable Pulmonary Hypertension
Class I. Pulmonary Hypertension Associated with Congenital Cardiovascular Disease
Medical Therapy. Efficacy or Lack of Efficacy
Medical Therapy. Adverse Effects or Lack of Adverse Effects
Age Focus: Pediatric Pulmonary Vascular Disease
Fresh or Filed Publication: Fresh (PHresh). Less than 1-2 years since publication
Article Access
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