Time for a gut check: Pancreatic sufficiency resulting from CFTR modulator use

Related Experiment Video

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Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein
09:59

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

Published on: March 9, 2015

12.6K

Rosemary Megalaa1, Vani Gopalareddy2, Elizabeth Champion1

  • 1Division of Pediatric Pulmonology, Atrium Health, Levine Children's Hospital, Charlotte, NC.

Pediatric Pulmonology
|May 9, 2019
PubMed

Insights

Pancreatic exocrine insufficiency in cystic fibrosis may improve with treatment. A 10-year-old child regained pancreatic function after 4 years on ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) modulator.

Area of Science:

  • Medical Research
  • Genetics
  • Pediatrics

Background:

  • Pancreatic exocrine insufficiency (PEI) is a common complication of cystic fibrosis (CF), often considered irreversible.
  • Genetic factors determine PEI in CF, impacting digestion and nutrient absorption.
  • Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators are emerging therapies for CF.

Observation:

  • A 10-year-old patient presented with lifelong PEI since birth.
  • The patient was treated with ivacaftor, a CFTR corrector drug, for four years.
  • Pancreatic function was assessed over the treatment period.

Findings:

  • The patient demonstrated a significant regain of pancreatic exocrine function after four years of ivacaftor therapy.
  • This suggests that pancreatic function in CF may not be entirely irreversible, even in cases of long-standing insufficiency.
  • CFTR modulator therapy showed potential for restoring pancreatic function.

Implications:

  • This case highlights the potential for CFTR modulators to reverse or improve pancreatic exocrine function in cystic fibrosis patients.
  • It suggests a possible therapeutic window for intervention, even in individuals with PEI since birth.
  • Further research is warranted to explore the long-term effects and mechanisms of CFTR modulators on pancreatic function in CF.

Keywords:
cystic fibrosismodulator therapypancreatic insufficiency

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