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Optimized LC-MS/MS Method for the High-throughput Analysis of Clinical Samples of Ivacaftor, Its Major Metabolites, and Lumacaftor in Biological Fluids of Cystic Fibrosis Patients
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Papilledema in Children With Cystic Fibrosis Receiving Elexacaftor/Tezacaftor/Ivacaftor: A Multicenter Case Series

Charissa W Kam1, Cameron J McKinzie1, Nicole E Omecene2

  • 1Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, North Carolina, USA.

Pediatric Pulmonology
|December 20, 2024
PubMed

Insights

Papilledema, a vision-threatening condition, was observed in children with cystic fibrosis (CF) using elexacaftor/tezacaftor/ivacaftor (ETI). Unlike previous reports, these cases showed minimal symptoms and normal Vitamin A levels.

Area of Science:

  • Ophthalmology
  • Pediatrics
  • Pulmonology

Background:

  • Elexacaftor/tezacaftor/ivacaftor (ETI) is a groundbreaking treatment for cystic fibrosis (CF).
  • Post-marketing surveillance and case reports have indicated a potential association between ETI use and papilledema/intracranial hypertension in children with CF.
  • Previous reports often linked these events to hypervitaminosis A.

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