Papilledema in Children With Cystic Fibrosis Receiving Elexacaftor/Tezacaftor/Ivacaftor: A Multicenter Case Series

Pediatr Pulmonol. 2024 Dec 20:e27455. doi: 10.1002/ppul.27455. Online ahead of print.

Abstract

Following the approval of elexacaftor/tezacaftor/ivacaftor (ETI), there have been post-marketing reports and published cases of papilledema and intracranial hypertension in children with cystic fibrosis (CF) taking ETI. In those reports, the patients often presented with marked symptoms and concomitant hypervitaminosis A. In this multicenter case series, we report eight cases of papilledema in children with CF taking ETI that were diagnosed via routine eye exam, the majority of whom presented with minimal to no symptoms and all had normal serum Vitamin A levels.