Characteristics and prospective two-year follow-up of children with pulmonary arterial hypertension in France

Justification: There is few data describing pulmonary arterial hypertension in children. Objectives: The objective was to describe the epidemiology and management of pulmonary arterial hypertension (PAH) in children, as well as its effects on the quality of life and its consequences, excluding patients with persistent pulmonary hypertension of the newborn and those with pulmonary arterial hypertension due to congenital heart disease. Methods: This multicenter non interventional study included children with pulmonary arterial hypertension who were prospectively followed for two years in 21 French centres. The WHO functional classification, the 6-minute walk distance, the quality of life (CHQ-PF50 questionnaire), the hemodynamics parameters and echodoppler performed were evaluated. Results: Fifty children with a mean age of 8.9 +/- 5.4 years were included from May 2005 to June 2006. The prevalence of pulmonary arterial hypertension was estimated at 3.7 cases per million. The patients had the following types of pulmonary arterial hypertension: idiopathic (60%), familial (10%), associated with congenital heart disease that was not the cause of the pulmonary hypertension (24%), associated with a connective-tissue disease (4%) or with portal hypertension (2%). During follow-up there was an increase in the number of drugs prescribed specifically for pulmonary arterial hypertension (44% patients versus 22% at inclusion). The clinical status, 6-minute walk test and quality of life of the majority of patients remained stable. The survival at one and at two years was estimated at 86% [95% confidence interval (76, 96)] and 82% [95% confidence interval (71, 93)]. Conclusions: The majority of cases of pulmonary arterial hypertension in children are idiopathic /familial. A specific group of pulmonary arterial hypertension occurring with congenital heart disease has been identified and resembles idiopathic pulmonary arterial hypertension. The use of specific treatments for PAH may contribute to the stability of the disease and to better survival. (C) 2010 Elsevier Masson SAS. All rights reserved.