Treatment options for paediatric pulmonary arterial hypertension

Abstract

Pulmonary arterial hypertension (PAH) is a serious, progressive condition, which can present idiopathically or secondary to conditions such as systemic sclerosis or congenital heart disease. The condition exists in both adult and paediatric forms, which possess several similar characteristics. Adult and paediatric PAH can, however, be distinguished based on underlying pathology and the presence of age-specific conditions, some of which are related to poor lung development in children. Improved knowledge of vascular biology has led to the development of several PAH-specific therapies, which have demonstrated clinical benefits in adults, including improved exercise capacity and prolonged survival. Treatment data in paediatric PAH are scarce. Although limited, the existing data indicate that current treatments for paediatric PAH are well tolerated and effective, at least in the short-and medium-term. Nevertheless, the current guidelines for clinicians, which recommend use of the adult treatment algorithm in paediatric patients, appear justified when judged according to the available evidence. However, further randomised, controlled trials are necessary to increase the evidence base for treatment of paediatric PAH, especially in relation to age-specific conditions. At present, early initiation of treatment and combination pharmacological therapy may offer the most promising courses of action to improve outcomes in paediatric PAH.

Circulating Endothelial Cells in Refractory Pulmonary Hypertension in Children: Markers of Treatment Efficacy and Clinical Worsening

“…In recent years, the available data on targeted PAH therapies suggest that they are generally well tolerated and effective in children, which is generally the case in adults [2], [3]. In adult patients, several studies have addressed the question of combined therapy at the time of clinical worsening [4].…”

“…Furthermore, dose formulation and the role of prostacyclin analogues are also challenging issues in the management of children with PAH. Decision-making often varies between centers [2], [3], [7] and NYHA functional evaluation is also difficult in younger children. For these reasons, biomarkers of treatment response and/or predictors anticipating any change in clinical status would be of great value in managing children with PAH.…”

Contemporary Strategy of Pulmonary Hypertension Management in Pediatrics

“…Независимо от причины ЛГ, всем больным показаны ограничения физических нагрузок. При этом тренировка дыхательной системы и дозированные физические нагрузки могут повысить толерантность к физическим нагрузкам и улучшить качество жизни [1][2][3][4][5][6][7][8][40][41].…”

Neonatal and Childhood Pulmonary Hypertension - An Update (2012)

“…For symptomatic patients who failed to improve with monodrug treatment, combination therapy may exert synergistic effects through targeting different molecular pathways [124]. Unfortunately, randomized-controlled trials in children with PAH-CHD are lacking, although in clinical settings more and more children receive combination therapies [84], perhaps supporting general effectiveness, safety and tolerability [125].…”

“…These approaches have shown encouraging results in preclinical animal models and application to human disease is being considered [20,125,[131][132][133].…”