Treatment with ivacaftor is associated with a change from pancreatic exocrine insufficiency (PI) to pancreatic sufficiency (PS) in children with cystic fibrosis and gating mutations, Australian research shows.
In a WA study of 204 children and adolescents with CF, all 12 patients eligible for ivacaftor were pancreatic insufficient (PI) at diagnosis and 11 were on pancreatic enzymes replacement therapy (PERT).
The study, published as a letter in Pediatric Pulmonology, found 36% (4/11 patients) became PS after starting ivacaftor.
“Children are more likely to change function if initial pancreatic elastase is more than 50 μg/g and if ivacaftor is started at a younger age,” it said.
“Potentially children eligible for ivacaftor at 1 year of age with borderline pancreatic function can now be managed without PERT if growing well.”
The study said that two of the four children on ivacaftor experienced recurrent abdominal pain while taking PERT. Their symptoms resolved when PERT was discontinued.
Another child with a residual CFTR function mutation became constipated before repeat testing of pancreatic function.
“There are many causes of abdominal pain and constipation in CF, and change in pancreatic status should be considered,” the researchers said.