A national registry for childhood interstitial and diffuse lung diseases in the United States

Introduction: Childhood interstitial and diffuse lung disease (chILD) encompasses a broad spectrum of rare pulmonary disorders. Our objectives are to advance knowledge on clinical features, management, and outcomes of this population. Methods: The Children’s Interstitial and Diffuse Lung Disease Research Network (ChILDRN) established a longitudinal observational study in 2016 using a national platform for single IRB reliance agreements with 13 participating sites across the United States. Results: 254 subjects have been enrolled to date. Specific chILD diagnoses and clinical characteristics are summarized in Table 1. Overall mean age at study enrollment was 101±73 months. Identified morbidity included home oxygen supplementation in 71% at any time and 44% with ongoing requirement. Failure to thrive was noted in 53%. 46% of subjects had undergone lung biopsy; genetic studies were used in diagnosis for 23%. Pulmonary function abnormalities varied based on disease subgroup. Conclusions: The first multicenter prospective study of chILD in the U.S. indicates substantial morbidity with variable phenotypes in different forms of chILD. This cohort provides a framework for future longitudinal studies focused on elucidation of the genetic and molecular underpinnings of these disorders, development of targeted therapies, and optimization of supportive care.