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Increasing Pulmonary Artery Pulsatile Flow Improves Hypoxic Pulmonary Hypertension in Piglets
Published on: May 11, 2015
Treatment of pulmonary arterial hypertension in children
Matthias Gorenflo1, Victoria C Ziesenitz1
1Department of Pediatric Cardiology and Congenital Heart Diseases, Centre of Child and Adolescent Health, University Hospital Heidelberg, Heidelberg, Germany.
Insights
Pediatric pulmonary arterial hypertension (PAH) treatments are evolving. Current options include bosentan and sildenafil, with new therapies under investigation to improve outcomes for children with PAH.
Area of Science:
- Pediatric Pulmonology
- Pharmacology
- Cardiovascular Medicine
Background:
- Pulmonary arterial hypertension (PAH) significantly impacts children's health.
- Current FDA-approved PAH treatments for pediatric patients include bosentan and sildenafil.
- New targeted therapies are being evaluated for pediatric use to reduce off-label prescribing.
Purpose of the Study:
- To review current treatment options for pediatric pulmonary arterial hypertension (PAH).
- To present an overview of emerging treatment concepts for pediatric PAH, drawing from adult studies.
- To highlight the need for further research and optimized drug development in pediatric PAH.
Main Methods:
- Literature review of current pediatric PAH pharmacotherapy.
- Analysis of existing treatment guidelines and clinical trial data.
- Exploration of novel drug classes and their potential in pediatric PAH management.
Main Results:
- Established treatments for pediatric PAH include endothelin receptor antagonists and phosphodiesterase-5 inhibitors.
- New drug classes like soluble guanylate cyclase stimulators and prostacyclin receptor agonists show promise but require pediatric evaluation.
- Evidence does not support initial combination therapy over monotherapy for pediatric PAH survival, considering diverse etiologies like PAH-CHD.
- Pharmacology in children may be influenced by developmental factors and drug-drug interactions, suggesting a role for therapeutic drug monitoring.
Conclusions:
- There is a critical need for more controlled studies on PAH medications in children, both as monotherapy and combination therapy.
- Pooling data from clinical trials and patient registries is essential for optimizing drug development, trial design, and evidence-based pharmacotherapy for pediatric PAH.
- Further research is required to establish the efficacy and safety of newer PAH therapies in the pediatric population.
Abstract:
Pulmonary arterial hypertension (PAH) is a devastating illness causing already significant morbidity in childhood. Currently approved treatment options for children comprise the endothelin receptor antagonist bosentan, as well as the phosphodiesterase-5 inhibitor sildenafil. But PAH treatment has advanced significantly over the past decade, and new classes of targeted drug therapies, such as stimulators of the soluble guanylate cyclase (riociguat) or prostacyclin receptor agonists (selexipag), are currently evaluated regarding their efficacy and safety in children, in order to limit off-label use. Due to the different etiologies in children, such as PAH-CHD, there is no evidence that initial combination therapy in children is superior to a mono-therapy with respect to survival. Special attention should also be paid to the pharmacology of PAH drugs in children, which might be impacted by ontogeny or drug-drug-interactions. Therapeutic drug monitoring may be useful in pediatric patients. There is a clear need for more controlled studies of PAH medications, alone or in combination therapy in the pediatric age group. Data from clinical trials as well as from patient registries should be pooled to optimize drug development and evaluation, trial design, and evidence-based pharmacotherapy in pediatric patients with PAH. In this review, the current treatment options of pediatric PAH are summarized, and an overview of new treatment concepts, which are already evaluated in adults, is presented.
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