An update on the diagnosis and treatment of pediatric pulmonary hypertension

ABSTRACT

Introduction

Pulmonary hypertension (PH) is a heterogeneous disease that mainly affects the pulmonary arterioles, leading to significant morbidity and mortality. Pulmonary hypertension in children from birth to adolescence presents important differences from that of adults. The majority of pediatric pulmonary arterial hypertension (PAH) cases are idiopathic or associated with congenital heart disease. However, the management of pediatric PAH mainly depends on the results of evidence-based adult studies and the clinical experiences of pediatric experts.

Areas covered

This article briefly reviews the recent updates on the definition, classification, and diagnostic evaluation of pediatric PAH and their impact on treatment strategies. The main purpose of this review is to discuss the current pediatric therapies, as well as the prospective therapies, in terms of therapeutic targets, actions, side effects, and dosages.

Expert opinion

Although there is no cure for PAH, recent advances in the form of new treatment options have improved the quality of life and survival rates of PAH patients. PAH-targeted drugs and treatment strategies for adult PAH have not been sufficiently studied in children. However, the growing scientific activity in that field will surely change the treatment option recommendations in pediatric PH from experience-based to evidence-based in the near future.

KEYWORDS:

• Children
• new treatment targets
• pediatrics
• pharmacotherapies
• pulmonary arterial hypertension; guidelines for therapy

Article highlights

• Pulmonary hypertension is a rare and progressive disease

• New definition, revised classification, and updated risk variables are important in diagnosis and follow-up of pediatric PH

• Pediatric PAH exhibits similarities and differences compared to adult PAH.

• Recent advances in drug therapies and new treatment strategies such as early initiation, upfront combination have improved the QOL and survival rates in children with PAH

• Novel therapeutic targets and drugs are needed

The growing number of scientific reports regarding pediatric PAH warrant that the existing knowledge on the treatment strategy of this rare disease could improve.

This box summarizes the key points contained in the article.

Acknowledgments

They would also like to acknowledge Gürkan Olgunturk and Ece Konaklıoğlu for their typing, editing, and technical assistance and Semiha Terlemez and Tuba Atalay for their assistance in data collection.

Declaration of interest

The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose