The US national registry for childhood interstitial and diffuse lung disease: Report of study design and initial...

Area of Science:

• Pediatric Pulmonology
• Rare Diseases
• Clinical Research

Background:

• Childhood interstitial and diffuse lung disease (chILD) represents a group of rare pediatric respiratory conditions.
• Understanding the etiology, phenotype, natural history, and management of chILD is crucial for improving patient outcomes.
• The Children's Interstitial and Diffuse Lung Disease Research Network (chILDRN) initiated a prospective registry to address knowledge gaps.

Purpose of the Study:

• To establish a large, longitudinal cohort of children diagnosed with interstitial and diffuse lung diseases.
• To gather comprehensive clinical data for analyzing the characteristics and progression of various chILD subtypes.
• To facilitate collaborative research efforts aimed at advancing the understanding and treatment of chILD.

Main Methods:

• A longitudinal, observational, multicenter registry design was employed.
• Data collection occurred across 25 chILDRN centers in the U.S. utilizing a single Institutional Review Board (IRB) agreement.
• Clinical data were managed through the Research Electronic Data Capture (REDCap) platform.

Main Results:

• The initial cohort comprised 683 subjects with diverse chILD diagnoses.
• Neuroendocrine cell hyperplasia of infancy was the most frequent diagnosis (23%).
• Commonly reported disease components included interstitial fibrosis, immune dysregulation, and airway disease, with high rates of supplemental oxygen use (63%) and failure to thrive (46%).

Conclusions:

• The chILDRN Registry represents the largest U.S. longitudinal cohort for childhood interstitial and diffuse lung disease.
• This registry provides a robust platform for multicenter collaboration to enhance chILD research.
• The findings underscore the significant morbidity associated with chILD, highlighting the need for continued investigation and improved therapeutic strategies.