Pancreatic, nutritional and clinical outcomes in children 0-5 years with cystic fibrosis during the first 2 years of CFTR modulator therapy (PaNC): a multicentre study.

Abstract

Hypothesis: Infants and young children 4 months to 5 years of age who are commenced on ETI or IVA will have improved and sustained growth and nutritional outcomes, exocrine pancreatic function and sweat chloride levels. Primary aim: To evaluate longitudinal patterns in growth and nutrition in children 4 months to 5 years of age with CF during the first 2 years of treatment with ETI or IVA. Secondary aim: To describe changes in pancreatic function, clinical outcomes and nutritional interventions in children 4 months to 5 years of age with CF during the first 2 years of treatment with ETI or IVA. Longitudinal, ‘real world’ studies are required to better understand the breadth and significance of CF exocrine pancreatic involvement in children with CF who are exposed to early CFTR correction as infants and children and to better understand the nutritional implications.