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Pulmonary arterial hypertension (PAH) is a progressive disease characterized by elevation of pulmonary vascular resistance and right ventricular failure. By using advanced therapies to reduce mortality, clinicians focus on improving functional status and quality of life (QOL). The aim of our study was to assess health-related QOL of pediatric patients with PAH. Parents of all children (aged 2-18 years) and patients aged 5-18 years with an appropriate level of intellectual development completed general and cardiac-specific validated surveys (Pediatric Quality of Life Inventory 4.0 and Pediatric Quality of Life Inventory 3.0, respectively). Demographic and clinical information was collected to grade disease severity. Twenty-five children were enrolled, yielding 25 parent reports and 15 patient self-reports. The PAH group had significantly lower scores than healthy children in all domains. Patients with World Health Organization Functional Class I had significantly higher parent proxy scores in School Functioning (P =.029) and in Heart Problems and Symptoms domain (P =.014) Patients with tricuspid annular plane systolic excursion below −2 z score showed impairment in each parent proxy general domain and in the Cognitive Problems score of the Cardiac module (P =.006). In conclusion the QOL of patients with PAH was impaired in every domain compared with healthy children. Patients with reduced right ventricle systolic function showed significantly lower QOL in all core domains. These results point to the need for psychosocial rehabilitation in addition to somatic care to improve the QOL in this severely ill population [ABSTRACT FROM AUTHOR]

Angiotensin-converting enzyme inhibitors (ACEI), such as enalapril, are a cornerstone of treatment for pediatric heart failure which is still used off-label. Using a novel age-appropriate formulation of enalapril orodispersible minitablets (ODMTs), phase II/III open-label, multicenter pharmacokinetic (PK) bridging studies were performed in pediatric patients with heart failure due to dilated cardiomyopathy (DCM) and congenital heart disease (CHD) in five participating European countries. Children were treated for 8 weeks with ODMTs according to an age-appropriate dosing schedule. The primary objective was to describe PK parameters (area under the curve (AUC), maximal concentration (Cmax), time to reach maximal concentration (t-max)) of enalapril and its active metabolite enalaprilat. Of 102 patients, 89 patients (n = 26, DCM; n = 63 CHD) were included in the primary PK endpoint analysis. Rate and extent of enalapril and its active metabolite enalaprilat were described and etiology and age could be identified as potential PK modifying factors. The dosing schedule appeared to be tolerated well and did not result in any significant drug-related serious adverse events. The PK analysis and the lack of severe safety events supports the applied age-appropriate dosing schedule for the enalapril ODMTs. [ABSTRACT FROM AUTHOR]