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TOPLINE
In young children with cystic fibrosis (CF), 1 year of elexacaftor-tezacaftor-ivacaftor (ETI) therapy was significantly associated with reduced sinus opacification and increased olfactory bulb volume, suggesting that early intervention may help prevent irreversible impairment of olfactory function.
METHODOLOGY
- Researchers conducted a prospective study across six pediatric CF centers in the US to assess sinonasal and olfactory outcomes in young children after ETI initiation.
- They enrolled 31 children with confirmed CF (mean age, 4.8 years; 55% girls) from May 2023 to December 2024. Children underwent sinus MRI and psychophysical olfactory testing at baseline and 1 year after ETI initiation.
- Olfactory function was assessed using the Pediatric Smell Wheel (PSW; range, 0-11; higher scores indicate better olfaction), and quality of life (QOL) was evaluated using the Brief Questionnaire of Olfactory Disorders (BQOD; range, 0-21; higher scores indicate worse olfactory QOL) and other caregiver-reported QOL surveys.
- Primary outcomes were sinus opacification and olfactory bulb volume.
TAKEAWAY
- After 1 year of ETI therapy, significant reductions in sinus opacification and improvements in olfactory bulb volume were observed, with sinus opacification decreasing by 25.6% (P < .01) and olfactory bulb volume increasing by 7.4 mm³ (P < .05).
- PSW odor identification scores increased by 1.3 points (P < .05) and olfactory cleft opacification decreased by 14.4 percentage points (P < .01).
- Caregiver-reported QOL surveys, including BQOD scores, remained predominantly unchanged after ETI therapy, with no statistically significant differences from baseline.
IN PRACTICE
"Given that prior literature demonstrated lack of improvement in olfactory dysfunction with ETI initiation later in life, these results highlight the potential olfactory benefits of early ETI initiation," the authors wrote.
SOURCE
This study was led by Janice J. Chung, MD, University of California, Los Angeles. It was published online on August 10, 2026, in the Annals of the American Thoracic Society.
LIMITATIONS
The single-arm design included no control group, limiting the ability to attribute the observed reductions in sinus opacification, increases in olfactory bulb volume, and improvements in odor identification to ETI. The modest sample size limited statistical power, and most outcomes were exploratory. No minimal clinically important difference has been established for the PSW in children of this age.
DISCLOSURES
This study was funded by the National Institutes of Health, the Cystic Fibrosis Foundation, and the American Academy of Otolaryngology-Head and Neck Surgery CORE/American Rhinologic Society. Several authors reported receiving grants, consulting fees, or honoraria or having other financial ties with pharmaceutical, device, and imaging companies, including Vertex Pharmaceuticals. Other authors declared having no relevant financial relationships.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
