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EUROPEAN RESPIRATORY REVIEW, 2009;18: 18-23. doi:10.1183/09059180.00011109
© 2009 the European Respiratory Society
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Paediatric pulmonary hypertension: monitoring progress and identifying unmet needs

M. Beghetti

CORRESPONDENCE: M. Beghetti, Unité de Cardiologie, Hôpital des Enfants, 6 rue Willy-Donzé, CH-1211 Geneva 14, Switzerland. Fax: 41 22993824580. E-mail: maurice.beghetti{at}hcuge.ch

Received: December 8, 2008
Accepted December 17, 2008

ABSTRACT

Recent advances in the field of pulmonary hypertension (PH) have provided clinicians with a range of treatment options, but effective disease management in children presents a unique challenge. The present article will discuss the steps being taken to address unmet needs in paediatric PH.

Understanding the epidemiology of paediatric PH is essential to guide management decisions, but such epidemiological data are scarce. The first international paediatric PH registry, Tracking Outcomes in Paediatric Pulmonary Hypertension (TOPP), promises to become a vital resource.

Studies of PH therapies are rare in children, and treatment of paediatric PH is generally guided by the adult treatment algorithm, with some adaptations. However, invasive management options, such as continuous prostacyclin infusion, even if effective, are challenging in children, and further research is required to develop appropriate treatment strategies, formulations and doses for paediatric PH. Measures of treatment success must also be defined, and the applicability of end-points from adult clinical studies remains an open question.

In summary, further epidemiological and treatment data are needed for paediatric pulmonary hypertension. The international TOPP registry will provide a valuable insight, but this must be complemented by research and development of adapted paediatric therapies. Dedicated childhood pulmonary hypertension services would optimise the diagnosis and management of this life-threatening disease.

KEYWORDS: Drug therapy, epidemiology, paediatric, pulmonary hypertension, registries, review






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