Abstract
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.
Footnotes
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Conflict of interest: Dr. Abman has nothing to disclose.
Conflict of interest: Dr. Mullen reports grants from NHLBI, during the conduct of the study; personal fees from Actelion, outside the submitted work;
Conflict of interest: Dr. Sleeper reports grants from NHLBI (sub from U. Colorado)Ð during the conduct of the study; .
Conflict of interest: Dr. Austin has nothing to disclose.
Conflict of interest: Dr. Rosenzweig reports grants from NIH, during the conduct of the study; .
Conflict of interest: Dr. Keller reports grants from NHLBI, during the conduct of the study; .
Conflict of interest: Dr. The University of Colorado contracts with Actelion, Bayer, Gilead, and United Therapeutics for Dr Ivy to be a consultant and preform clinical research trials.
Conflict of interest: Dr. Hopper has nothing to disclose.
Conflict of interest: Dr. Raj has nothing to disclose.
Conflict of interest: Dr. Fineman has nothing to disclose.
Conflict of interest: Dr. Keller reports grants from NHLBI, during the conduct of the study; .
Conflict of interest: Dr. Bates has nothing to disclose.
Conflict of interest: Dr. Krishnan has nothing to disclose.
Conflict of interest: Dr. Avitabile has nothing to disclose.
Conflict of interest: Dr. Davidson has nothing to disclose.
Conflict of interest: Dr. Natter reports grants from National Heart, Lung, and Blood Institute (NHLBI), during the conduct of the study; .
Conflict of interest: Dr. Kenneth Mandl reports no o specific conflicts, however in the interest of full disclosure, reports personal fees from Merck, and philanthropy from Eli Lily to his lab, during the conduct of the study;
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- Received September 12, 2020.
- Accepted May 14, 2021.
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