TY - JOUR
T1 - The US national registry for childhood interstitial and diffuse lung disease
T2 - Report of study design and initial enrollment cohort
AU - for the chILD Registry Collaborative
AU - Nevel, Rebekah J.
AU - Deutsch, Gail H.
AU - Craven, Daniel
AU - Deterding, Robin
AU - Fishman, Martha P.
AU - Wambach, Jennifer A.
AU - Casey, Alicia
AU - Krone, Katie
AU - Liptzin, Deborah R.
AU - O'Connor, Michael G.
AU - Kurland, Geoffrey
AU - Taylor, Jane B.
AU - Gower, William A.
AU - Hagood, James S.
AU - Conrad, Carol
AU - Tam-Williams, Jade B.
AU - Fiorino, Elizabeth K.
AU - Goldfarb, Samuel
AU - Sadreameli, Sara C.
AU - Nogee, Lawrence M.
AU - Montgomery, Gregory
AU - Hamvas, Aaron
AU - Laguna, Theresa A.
AU - Bansal, Manvi
AU - Lew, Cheryl
AU - Santiago, Maria
AU - Popova, Antonia
AU - De, Aliva
AU - Chan, Marilynn
AU - Powers, Michael R.
AU - Josephson, Maureen B.
AU - Camburn, Devaney
AU - Voss, Laura
AU - Li, Yun
AU - Young, Lisa R.
N1 - Publisher Copyright:
© 2023 Wiley Periodicals LLC.
PY - 2024/9
Y1 - 2024/9
N2 - Introduction: 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. Methods: 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. Results: We report the study design and selected 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, 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%). Conclusion: This Registry is the largest longitudinal chILD cohort in the United States to date, providing a powerful framework for collaborating centers committed to improving the understanding and treatment of these rare disorders.
AB - Introduction: 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. Methods: 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. Results: We report the study design and selected 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, 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%). Conclusion: This Registry is the largest longitudinal chILD cohort in the United States to date, providing a powerful framework for collaborating centers committed to improving the understanding and treatment of these rare disorders.
KW - NEHI
KW - interstitial lung disease
KW - rare lung disease
UR - http://www.scopus.com/inward/record.url?scp=85164471819&partnerID=8YFLogxK
U2 - 10.1002/ppul.26568
DO - 10.1002/ppul.26568
M3 - Article
C2 - 37401889
AN - SCOPUS:85164471819
SN - 8755-6863
VL - 59
SP - 2236
EP - 2246
JO - Pediatric Pulmonology
JF - Pediatric Pulmonology
IS - 9
ER -