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Reported Adverse Events in a Multicenter Cohort of Patients Ages 6-18 Years with Cystic Fibrosis and at Least One F508del Allele Receiving Elexacaftor/Tezacaftor/Ivacaftor.

Vito TerlizziCristina FevolaSantiago PrestiAlice CastaldoValeria DaccòLaura ClautAngela SepeFabio MajoRosaria CasciaroIrene EspositoPamela VitulloMarta SalviPatrizia TroianiFrancesca FiciliGiuseppe Fabio ParisiStefano PantanoStefano CostaGiuseppina LeonettiNicola PalladinoGiovanni TaccettiPaolo BonomiDonatello Salvatore
Published in: The Journal of pediatrics (2024)
This real-world study highlights significant variability in reported AEs. Our findings suggest that ETI is a safe and well-tolerated therapy in children and adolescents with CF. However, further long-term safety and effectiveness investigations are warranted.
Keyphrases
  • cystic fibrosis
  • end stage renal disease
  • newly diagnosed
  • ejection fraction
  • randomized controlled trial
  • chronic kidney disease
  • peritoneal dialysis
  • prognostic factors
  • double blind