Diagnosis and treatment of pediatric pulmonary arterial hypertension

ABSTRACT

Introduction: Pediatric pulmonary arterial hypertension (PAH) remains a rare and severe disease with a poor prognosis. PAH may be idiopathic, heritable or associated with systemic conditions in particular associated with congenital heart disease.

Areas covered: A thorough and extensive diagnostic approach is required for a correct diagnosis. The outcome has improved over the last decade with a better diagnostic approach and with the initiation of new targeted therapies. However, there is still significant progress to achieve as there is still no cure for this devastating disease.

Expert opinion: Adapted clinical studies to define the best therapeutic approach are needed. Even if the treatment approach is still mainly derived from adult data and expert consensus, several studies and registries are currently underway and should deliver important information in the next future.

This review aims to give an overview of the current diagnosis and treatment strategies of PAH.

KEYWORDS:

• Paediatric
• pulmonary arterial hypertension
• treatment
• diagnosis

Article highlights

• Expertise and exhaustive diagnostic evaluation of the disease are mandatory to manage and follow up these patients.

• The use of targeted therapies still requires clinical studies to define what would be the best treatment approach.

• Even if cardiac catheterization remains the gold standard, non-invasive tools seem to be promising for evaluation and follow-up of PAH in the future, with advantages of being cost-effective and accessible.

• A more aggressive treatment approach with combination therapy is an attracting option.

• Interventional and surgical approaches may have a therapeutic role in the management of PAH and a better definition of the indication and the procedure strategy is needed in the next future.

This box summarizes key points contained in the article.

Declaration of interest

F Lador has received support from Actelion Pharmaceuticals, MSD, and Bayer. M Beghetti has received grant/research support from Actelion Pharmaceuticals Ltd and Bayer HealthCare, has been a consultant for Actelion Pharmaceuticals Ltd, Bayer HealthCare, Eli Lilly & Co., and GlaxoSmithKline, and has attended speakers bureau(s) for Actelion Pharmaceuticals Ltd. The authors have no other 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 apart from those disclosed.

Reviewer disclosures

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

Additional information

Funding

This paper was not funded.