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Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor
Author(s) -
Munce Danielle,
Lim Meerana,
Akong Kathryn
Publication year - 2020
Publication title -
pediatric pulmonology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.866
H-Index - 106
eISSN - 1099-0496
pISSN - 8755-6863
DOI - 10.1002/ppul.25065
Subject(s) - ivacaftor , cystic fibrosis , cystic fibrosis transmembrane conductance regulator , medicine , pancreatic function , malabsorption , exocrine pancreatic insufficiency , pancreatic disease , pancreatic enzymes , gastroenterology , endocrinology , pancreatitis , pancreas
Exocrine pancreatic insufficiency (EPI), which leads to malabsorption and poor weight gain, is seen in 85% of patients with cystic fibrosis (CF). EPI is treated with pancreatic enzyme replacement therapy taken with each meal. The highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulator, ivacaftor, restores CFTR function in patients with responsive mutations. It is a widely held view that EPI in CF is irreversible due to the complete destruction of pancreatic ducts and acinar cells. We describe three pediatric CF patients with EPI who were started on ivacaftor, and subsequently showed evidence of restored exocrine pancreatic function with clinical and biochemical parameters.