313 French pulmonary arterial hypertension registry in children: 2-year follow-up data

Abstract

Pulmonary arterial hypertension (PAH) is a devastating disease with poor survival but there are limited data describing its impact in the paediatric population. The objectives of this national prospective registry were to collect clinical and epidemiological data and to investigate the outcome of children with PAH. All consecutive patients <18 years with PAH seen in 16 referral PAH centres in France were included and evaluated after 1 and 2 years of follow-up for WHO functional class (FC), 6-minute walk distance (6MWD) and quality of life (QoL; CHQ-PF50 questionnaire). Persistent pulmonary hypertension of the newborn and PAH due to congenital heart disease (CHD) were excluded. Fifty patients were included between May 2005 and June 2006. Mean age at diagnosis was 8.7 ± 5.4 years and male/female ratio was 1/1. The prevalence of pediatric PAH was 4.2 cases/million. A history of prematurity, cancer and major surgery was noticed in respectively 19%, 6% and 4% of patients. At inclusion, 28% of patients were in WHO FC III or IV. Aetiology of PAH was idiopathic in 60%; familial in 10%, associated with but not due to CHD in 24%, related to connective tissue disease in 4% and to portal hypertension in 2%. Nine patients (018%) died during the 2-year follow up. Survival estimates at 1 and 2 years were 86% and 82%. Seventy three percents of patients improved or did not change WHO FC (n = 44). Patients remained stable regarding 6MWD (n = 25), hemodynamics (n = 11), and QoL (n = 19). During the 2-year follow-up, combination of PAH-specific therapies was increasingly prescribed (44% patients vs. 22% at inclusion). This first national paediatric registry showed (1) the presence of multiple PAH aetiologies in children, that could be different from those seen in adults, (2) the association with other co-morbidities, and (3) the stabilization or even improvement in patient's condition with the therapeutic management recommended in the current era.