Benefits of reviewing pancreatic function in children with cystic fibrosis

Background: Cystic fibrosis is a common cause of pancreatic exocrine insufficiency. Clinically pancreatic insufficiency is treated as a permanent problem. However, new cystic fibrosis transmembrane conductance regulator (CFTR) protein modulator therapies, like ivacaftor, can change pancreatic function in children. Also, children with residual CFTR function mutations may be pancreatic insufficient in infancy and spontaneously become pancreatic sufficient. Aims: To evaluate the benefit of reviewing pancreatic function, testing and diagnosis, of a cohort and repeat testing to patients with the potential to change function. Methods: all children with cystic fibrosis managed at Perth Children’s Hospital at the end of 2019 were reviewed for pancreatic function testing at diagnosis, genotype and current modulator and pancreatic enzyme replacement therapy use. Children with the potential to change pancreatic function were offered repeat testing. Results: 204 children were included. 11 children were prescribed ivacaftor and four of these became pancreatic sufficient. Five children with a residual CFTR function mutation were pancreatic insufficient at diagnosis. Four became pancreatic sufficient. Conclusion: there is benefit in re-evaluating pancreatic function in children with cystic fibrosis regularly, especially if on ivacaftor or pancreatic insufficient with a residual CFTR function mutation.