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<p>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&#xa0;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&#xa0;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&#xa0;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&#xa0;m (p = 0.032); NT‐proBNP decreased a mean of − 438.9 ± 175&#xa0;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.</p>

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Macitentan in Children with Pulmonary Arterial Hypertension: A Multicenter Experience

  • 期刊论文

  • 发表日期：

    2025年7月5日

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  • A. Rodriguez Ogando,
  • A. Sabaté-Rotés,
  • A. Moya Bonora,
  • L. Albert de la Torre,
  • J. M. Espín López,
  • C. Labrandero de Lera,
  • M. J. del Cerro Marin

摘要

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.