Characterisation of Pediatric Pulmonary Hypertensive Vascular Disease from the PPHNet Registry.

Abman, Steven H; Mullen, Mary P; Sleeper, Lynn A; Austin, Eric D; Rosenzweig, Erika B; Kinsella, John P; Ivy, Dunbar; Hopper, Rachel K; Usha Raj, J; Fineman, Jeffrey; Keller, Roberta L; Bates, Angela; Krishnan, Usha S; Avitabile, Catherine M; Davidson, Alexander; Natter, Marc D; Mandl, Kenneth D

BACKGROUND There are limited data about the range of diseases, natural history, age-appropriate endpoints and optimal care for children with pulmonary hypertension (PH), including the need for developing high quality patient registries of children with diverse forms of PH to enhance care and research. OBJECTIVE To characterise the distribution and clinical features of diseases associated with pediatric PH, including natural history, evaluation, therapeutic interventions and outcomes, as defined by the WSPH Classification. METHODS 1475 patients were enrolled into a multisite registry across the Pediatric Pulmonary Hypertension Network (PPHNet), comprised of 8 interdisciplinary PH programs. RESULTS WSPH Groups 1 (PAH) and 3 (lung disease) were the most common primary classifications (45% and 49% of subjects, respectively). The most common Group 3 conditions were BPD and CDH. Group 1 disease was predominantly associated with congenital heart disease (60%) and idiopathic (23% of Group 1 cases). In comparison with Group 1, Group 3 subjects had better disease resolution (HR=3.1, p<0.001), tended to be younger at diagnosis (0.3 (0.0,0.6) versus 1.6 (0.1,6.9) years (median (IQR); p<0.001), and were more often male (57% versus. 45%, p<0.001). Down syndrome (DS), the most common genetic syndrome in the registry, constituted 11% of the entire PH cohort. CONCLUSIONS We find a striking proportion of pediatric PH patients with Group 3 disorders, reflecting the growing recognition of PH in diverse developmental lung diseases. Greater precision of clinical phenotyping based on disease-specific characterization may further enhance care and research of pediatric PH.