Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor

Danielle Munce; Meerana Lim; Kathryn Akong

doi:10.1002/ppul.25065

Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor

Pediatr Pulmonol. 2020 Dec;55(12):3381-3383. doi: 10.1002/ppul.25065. Epub 2020 Oct 22.

Authors

Danielle Munce¹, Meerana Lim^{1

2}, Kathryn Akong^{1

2}

Affiliations

¹ Department of Pediatrics, University of California, San Diego, California, USA.
² Division of Pediatric Respiratory Medicine, Rady Children's Hospital, San Diego, California, USA.

PMID: 32910556
DOI: 10.1002/ppul.25065

Abstract

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.

Keywords: fecal elastase; pancreatic enzyme replacement therapy; pancreatitis.

Publication types

Case Reports

MeSH terms

Aminophenols / therapeutic use*
Child
Chloride Channel Agonists / therapeutic use*
Cystic Fibrosis / drug therapy*
Cystic Fibrosis Transmembrane Conductance Regulator / agonists
Cystic Fibrosis Transmembrane Conductance Regulator / genetics
Exocrine Pancreatic Insufficiency / drug therapy*
Female
Humans
Male
Mutation
Pancreas / drug effects
Quinolones / therapeutic use*

Substances

Aminophenols
CFTR protein, human
Chloride Channel Agonists
Quinolones
Cystic Fibrosis Transmembrane Conductance Regulator
ivacaftor