Sinus Disease in Young Children With Cystic Fibrosis
This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) in young children with cystic fibrosis (YCwCF). This study involves two groups: children 2-8 years old, inclusive at initial visit, receiving highly effective modulator therapy (HEMT), and a control group of children 2-8 years old, inclusive at initial visit, not receiving HEMT. Outcomes will include sinus magnetic resonance imaging (MRI) scans, olfactory tests, and quality of life surveys obtained over a two-year period.
⁃ HEMT Group:
• Children with documentation of a CF diagnosis
• Age 2-8 years old at first study visit
• CFTR mutation consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor)
• Clinician intent to prescribe ivacaftor or ETI so that enrollment is before start of HEMT
⁃ Non-HEMT/Control Group:
• Children with documentation of a CF diagnosis
• Age 2-8 years at first study visit
• Ineligible for highly effective modulator therapy (ivacaftor or elexacaftor/tezacaftor/ivacaftor) based on CFTR mutation or clinical decision not to initiate HEMT if eligible