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Macitentan in infants and children with pulmonary hypertensive vascular disease. Feasibility, tolerability and
Sulaima Albinni1, Imre Pavo1, Erwin Kitzmueller1
1Department of Paediatrics and Adolescent Medicine, Paediatric Heart Centre Vienna, Medical University of Vienna, Vienna, Austria.
Insights
Macitentan is a feasible and well-tolerated treatment for pediatric pulmonary arterial hypertension. Careful monitoring for edema during introduction is recommended in children and infants.
Area of Science:
- Pediatric Cardiology
- Pharmacology
- Pulmonary Hypertension
Background:
- Pulmonary arterial hypertension (PAH) treatment in adults is established with macitentan.
- Limited data exist on macitentan's use in pediatric patients.
- This study addresses the applicability and practical aspects of macitentan in children.
Purpose of the Study:
- To evaluate the feasibility and tolerability of macitentan in pediatric patients with pulmonary arterial hypertension.
- To assess safety parameters including liver function and blood counts.
- To report on practical aspects of introducing macitentan in a pediatric population.
Main Methods:
- Prospective, single-center study of 24 pediatric patients (0.1-23 years) with PAH.
- Macitentan introduced per a weight-adjusted dosing protocol.
- Clinical parameters, liver function, and blood counts monitored over time.
Main Results:
- Macitentan was feasible and generally well-tolerated in 22 of 24 patients.
- Two patients discontinued due to edema.
- Liver function and blood counts remained stable; no significant adverse events reported.
Conclusions:
- Macitentan introduction is feasible and mostly well-tolerated in pediatric PAH patients.
- Edema requires special attention during macitentan initiation.
- This is the first study on macitentan applicability in infants and children; larger trials are needed.
Abstract:
Macitentan is a safe and effective substance for treatment of adults with pulmonary arterial hypertension. Data on its use in paediatric patients are limited. In this single-centre prospective study, we report on our experience with macitentan in children focusing on applicability and practical aspects. Between December 2014 and July 2018, macitentan was introduced to paediatric patients according to a dosing protocol adjusted to body weight. Blood pressure, heart rate, saturation and clinical symptoms were recorded daily during introduction. Liver function parameters and haemoglobin levels were measured at baseline, four weeks and three months after initiation and after one year of treatment. Twenty-four patients (14 male, 10 female) were enrolled for treatment with macitentan. The mean age was 10.7 ± 7.6 years (range: 0.1 year-23 years). Fifteen out of 24 patients were World Health Organization functional class (FC) II, 7 patients in FC III and 2 patients in FC IV. Twenty out of 24 patients (83%) received additional advanced therapy with sildenafil and/or prostacyclines. We had two early discontinuations because of clinical relevant oedema. In the remaining 22 patients, macitentan was well tolerated. Liver function parameters and blood count levels remained stable during the observational time. The introduction of macitentan was feasible and mostly well tolerated in paediatric patients. Special attention should be paid to oedema during introduction of the drug. To the best of our knowledge, this is the first study to report on its applicability in infants and children. However, larger prospective trials are warranted to verify these preliminary findings.
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