Survival in Childhood Pulmonary Arterial Hypertension

Abstract

I nvestigators from multiple institutions conducted an observational study to describe patient characteristics in children with pulmonary arterial hypertension (PAH) and identify factors related to increased survival. Study participants were children <18 years old diagnosed with PAH, who had been enrolled in the Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management (REVEAL). The registry contains clinical information on pediatric patients enrolled from 2006 through 2009 at 26 sites across the United States.The children with PAH were categorized into subgroups: idiopathic or familial PAH (IPAH/FPAH); PAH associated with connective tissue disease, congenital heart disease (APAH-CHD; repaired or unrepaired), human immunodeficiency virus, and portal hypertension (APAH-PoPH) with and persistent pulmonary hypertension of the newborn.There were 216 children with PAH in the registry; of these, 92% were either in the IPAH/FPAH (56%, n=122) or APAH-CHD (36%; repaired, n=29; unrepaired/partial repair, n=48) subgroups. The median age at diagnosis was 7 years. Girls were more commonly affected (62%). The most common initial symptoms were dyspnea on exertion (DOE) (46% of patients), fatigue (24%), and syncope/presyncope (24%). DOE and syncope/presyncope were significantly more frequent in the IPAH/FPAH subgroup (53% and 36%, respectively) as compared to those with APAH-CHD (30% and 4%, respectively).Significantly more children with IPAH/FPAH responded to acute vasodilator testing, as compared to those with APAH-CHD (35% vs 15%; P = .006). However, among the 162 patients with IPAH/FPAH or APAH-CHD who underwent vasodilator testing, there was no difference in survival between the IPAH/FPAH and APAH-CHD subgroups (overall survival 75%). Older age at diagnosis, higher pulmonary vascular resistance index, lower weight at time of diagnosis, and familial PAH were significantly associated with decreased survival. The authors conclude that identification of factors associated with survival will help clinicians tailor therapy and subsequently improve outcomes.