The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol

Christine M Liu; Jakob L Fischer; Edith T Zemanick; Jason C Woods; Karolin K Markarian; Sean B Fain; Deborah Froh; Sonya L Heltshe; Lucas R Hoffman; Stephen M Humphries; Elizabeth L Kramer; Katie Larson Ode; Michael Lewis; Douglas A Li; Jaime Mata; Sarah S Milla; Peter J Niedbalski; Benjamin D Sawatzky; Myung-Shin Sim; Jillian S Sullivan; Andrew T Trout; Christopher H Goss; Jennifer L Taylor-Cousar; Daniel M Beswick

doi:10.1183/23120541.00137-2024

The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol

ERJ Open Res. 2025 Jan 13;11(1):00137-2024. doi: 10.1183/23120541.00137-2024. eCollection 2025 Jan.

Authors

Christine M Liu¹, Jakob L Fischer¹, Edith T Zemanick^{2

3}, Jason C Woods⁴, Karolin K Markarian⁵, Sean B Fain⁶, Deborah Froh⁷, Sonya L Heltshe⁸, Lucas R Hoffman^{8

9}, Stephen M Humphries¹⁰, Elizabeth L Kramer^{4

11}, Katie Larson Ode¹², Michael Lewis¹³, Douglas A Li¹⁴, Jaime Mata¹⁵, Sarah S Milla^{16

17}, Peter J Niedbalski¹⁸, Benjamin D Sawatzky¹⁹, Myung-Shin Sim¹⁴, Jillian S Sullivan²⁰, Andrew T Trout^{4

21}, Christopher H Goss⁹, Jennifer L Taylor-Cousar^{22

23}, Daniel M Beswick¹

Affiliations

¹ Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA.
² Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA.
³ Children's Hospital Colorado, Breathing Institute, Aurora, CO, USA.
⁴ Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
⁵ University of California, Los Angeles, CTSI, David Geffen School of Medicine, Los Angeles, CA, USA.
⁶ Department of Radiology, University of Iowa, Iowa City, IA, USA.
⁷ Department of Pediatrics, University of Virginia, Charlottesville, VA, USA.
⁸ Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA, USA.
⁹ Department of Medicine and Pediatrics, University of Washington, Seattle, WA, USA.
¹⁰ Department of Radiology, National Jewish Health, Aurora, CO, USA.
¹¹ Department of Pediatrics, University of Cincinnati College of Medicine, Cincinnati, OH, USA.
¹² Department of Pediatrics, University of Iowa, Iowa City, IA, USA.
¹³ Department of Pediatrics, University of Kansas Medical Center, Kansas City, KS, USA.
¹⁴ Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
¹⁵ Department of Radiology and Medical Imaging, University of Virginia, Charlottesville, VA, USA.
¹⁶ Department of Radiology, Children's Hospital Colorado, Aurora, CO, USA.
¹⁷ Department of Radiology, University of Colorado School of Medicine, Aurora, CO, USA.
¹⁸ Department of Pulmonary, Critical Care, and Sleep Medicine, University of Kansas Medical Center, Kansas City, KS, USA.
¹⁹ Department of Radiology, University of Vermont, Colchester, VT, USA.
²⁰ Department of Pediatrics, University of Vermont, Colchester, VT, USA.
²¹ Department of Radiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
²² Department of Medicine, National Jewish Health, Aurora, CO, USA.
²³ Department of Pediatrics, National Jewish Health, Aurora, CO, USA.

Abstract

Background: Chronic rhinosinusitis (CRS) and olfactory dysfunction (OD) are prevalent disease complications in people with cystic fibrosis. These understudied comorbidities significantly impact quality of life. The impact of highly effective modulator therapy (HEMT) in young children with cystic fibrosis (YCwCF) on these disease complications is unknown. This proposed study aims to characterise CRS and OD in YCwCF and assess the efficacy of HEMT in improving sinus and olfactory health in this young age group.

Methods: This six-centre, prospective, observational study will enrol 80 YCwCF aged 2-8 years. Patients are divided into two groups: those receiving HEMT and those not on HEMT based on clinical indication. Both groups undergo sinus magnetic resonance imaging, psychophysical olfactory tests, and complete patient- or parent-reported quality of life surveys over 2 years. Outcomes will be compared before and after initiation of HEMT and between groups. Ethical approval has been obtained for all sites, and this study has been registered on ClinicalTrials.gov (NCT06191640).

Results: Enrolment began in April 2023. 21 participants have been enrolled as of October 2023 with ongoing enrolment at all sites.

Conclusion: This investigation is expected to provide critical insights into the potential benefits of early HEMT initiation in managing CRS and OD in YCwCF. It will assist in developing targeted interventions and contribute to the understanding of HEMT's role in altering the disease course in this demographic.

Associated data

ClinicalTrials.gov/NCT06191640