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Macitentan in Children with Pulmonary Arterial Hypertension: A Multicenter Experience
A Rodriguez Ogando1,2, A Sabaté-Rotés3, A Moya Bonora4
1Department of Paediatric Cardiology, Health Research Institute Gregorio Marañón, Gregorio Marañon General University Hospital, Madrid, Spain. alejandro.rodriguez@salud.madrid.org.
Insights
Macitentan treatment significantly improved outcomes in pediatric patients with pulmonary arterial hypertension (PAH), showing enhanced functional class and exercise capacity. The drug demonstrated good safety and efficacy in this young population.
Area of Science:
- Pediatric Cardiology
- Pulmonary Hypertension Research
- Pharmacological Studies
Background:
- Pulmonary arterial hypertension (PAH) in children requires effective treatment options.
- Macitentan, an endothelin receptor antagonist, has shown promise in adult PAH but requires evaluation in pediatric populations.
- Limited data exists on the safety and efficacy of macitentan in patients under 18 years.
Purpose of the Study:
- To assess the safety and efficacy of oral macitentan in pediatric patients with Group 1 PAH.
- To evaluate clinical outcomes including functional class, exercise capacity, and biochemical markers.
- To determine the drug's tolerability and impact on risk stratification in children with PAH.
Main Methods:
- Multi-center, observational study using data from the Spanish Registry of Pediatric Pulmonary Hypertension.
- Inclusion of 74 pediatric patients (median age 9.6 years) with Group 1 PAH.
- Assessment of outcomes at 6 months, including World Health Organization Functional Class (WHO FC), 6-minute walking distance (6MWD), NT-proBNP, risk parameters, and quality of life.
Main Results:
- Significant improvement in WHO FC, with a decrease in patients in WHO FC III/IV from 48% to 22% (p=0.001).
- Mean 6MWD increased by approximately 45 meters (p=0.032).
- NT-proBNP levels decreased significantly (mean -438.9 pg/ml, p=0.015), with a 17% increase in patients meeting low-risk criteria (p=0.002).
- Adverse events were reported in 5.4% of patients, with macitentan discontinued in one case.
Conclusions:
- Oral macitentan demonstrates significant clinical benefits and good tolerance in pediatric patients with Group 1 PAH.
- The findings support the use of macitentan in routine clinical practice for managing pediatric PAH.
- Further research may explore long-term outcomes and specific PAH subtypes in children.
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.
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