ArticlesClinical features of paediatric pulmonary hypertension: a registry study
Introduction
Pulmonary hypertension with increased pulmonary vascular resistance is associated with substantial morbidity and mortality. The most recent clinical classification defines five pulmonary hypertension groups, with pulmonary arterial hypertension (PAH) being group 1 (the full classification is provided in the webappendix).1 PAH can be idiopathic (IPAH), heritable (HPAH), or associated with conditions (APAH) such as congenital heart disease and can present at any age. It is a rare disease with incidence and prevalence estimates of 2–3 per million and 25–50 per million, respectively.
Without treatment, median survival after diagnosis of IPAH or HPAH has been reported as 2·8 years in adults, but survival in children might be worse.2 Clinical trials and registries have led to substantial progress in treatment of this disorder in adults.2, 3, 4, 5, 6 Although pathobiology and clinical features share similarities in children and adults, paediatric pulmonary hypertension could well differ from adult disease.7, 8, 9 Adult studies alone cannot provide a basis for optimum care for children. However, paediatric pulmonary hypertension is insufficiently characterised. The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry is a global, prospective, observational study designed to provide information about demographics, course, treatment, and outcomes in paediatric pulmonary hypertension.10
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Study design
TOPP is a centre-based, comprehensive registry, which was initiated on Jan 31, 2008. Enrolled patients undergo assessment, treatment, and follow-up according to the judgment of their physicians. No specific therapy or follow-up protocols are part of TOPP. Patients in clinical trials are eligible. Patients were enrolled from 31 centres in 19 countries in five continents (sites and investigators are listed at the end of the report). Patients with pulmonary hypertension caused by left-heart
Results
From Jan 31, 2008, to Feb 15, 2010, 456 patients were enrolled. Enrolment of prevalent and incident patients was stopped at one site on Feb 10 and Aug 19, of 2009, respectively, to prevent overrepresentation of that site. Enrolment of prevalent patients at all other sites was stopped on May 22, 2009, to prevent such patients accounting for more than two-thirds of the total. No further predefined restriction rules were required.
Of the 456 patients in the all-patients cohort, 362 (79%) met all
Discussion
Overall, about half the children enrolled with confirmed pulmonary hypertension had a history of congenital heart disease, which was regarded as causative for the PAH in most cases. In the most recent classification of pulmonary hypertension, a specific threshold for increased pulmonary vascular resistance was abandoned as a criterion for PAH.11 Flow-associated pulmonary hypertension due to large left-to-right systemic-to-pulmonary shunts might have normal pulmonary capillary wedge pressure and
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