The U.S. National Registry for Childhood Interstitial and Diffuse Lung
Disease: Report of Study Design and Initial Enrollment Cohort
Abstract
Childhood interstitial and diffuse lung disease (chILD) encompasses a
broad spectrum of rare disorders. The Children’s Interstitial and
Diffuse Lung Disease Research Network (chILDRN) established a
prospective registry to advance knowledge regarding etiology, phenotype,
natural history, and management of these disorders. This longitudinal,
observational, multicenter registry utilizes single-IRB reliance
agreements, with participation from 25 chILDRN centers across the U.S.
Clinical data are collected and managed using the Research Electronic
Data Capture (REDCap) electronic data platform. We report the study
design and some elements of the initial Registry enrollment cohort,
which includes 683 subjects with a broad range of chILD diagnoses. The
most common diagnosis reported was neuroendocrine cell hyperplasia of
infancy (NEHI), with 155 (23%) subjects. Components of underlying
disease biology were identified by enrolling sites, with cohorts of
interstitial fibrosis, immune dysregulation, and airway disease being
most commonly reported. Prominent morbidities affecting enrolled
children included home supplemental oxygen use (63%) and failure to
thrive (46%). This Registry is the largest longitudinal chILD cohort in
the U.S. to date, providing a powerful framework for collaborating
centers committed to improving the understanding and treatment of these
rare disorders.