Journal article
The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol
ERJ open research, Vol.11(1), 00137-2024
01/2025
DOI: 10.1183/23120541.00137-2024
PMCID: PMC11726580
PMID: 39811548
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 CF (YCwCF) on these disease complications is unknown. This proposed study aims to characterize 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-center, prospective, observational study will enroll 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 two 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 Enrollment began in April 2023. Twenty-one participants have been enrolled as of October 2023 with ongoing enrollment 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.
Details
- Title: Subtitle
- The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol
- Creators
- Christine M. Liu - University of California, Los AngelesJakob L. Fischer - University of California, Los AngelesEdith T. Zemanick - Children's Hospital ColoradoJason C. Woods - Cincinnati Children's Hospital Medical CenterKarolin K. Markarian - University of California, Los AngelesSean B. Fain - University of IowaDeborah Froh - University of VirginiaSonya L. Heltshe - Seattle Children's HospitalLucas R. Hoffman - University of WashingtonStephen M. Humphries - National Jewish HealthElizabeth L. Kramer - University of Cincinnati Medical CenterKatie Larson Ode - University of IowaMichael Lewis - University of Kansas Medical CenterDouglas A. Li - University of California, Los AngelesJaime Mata - University of VirginiaSarah S. Milla - Children's Hospital ColoradoPeter J. Niedbalski - University of Kansas Medical CenterBenjamin D. Sawatzky - University of VermontMyung-Shin Sim - University of California, Los AngelesJillian S. Sullivan - University of VermontAndrew T. Trout - Cincinnati Children's Hospital Medical CenterChristopher H. Goss - University of WashingtonJennifer L. Taylor-Cousar - National Jewish HealthDaniel M. Beswick - University of California, Los Angeles
- Resource Type
- Journal article
- Publication Details
- ERJ open research, Vol.11(1), 00137-2024
- DOI
- 10.1183/23120541.00137-2024
- PMID
- 39811548
- PMCID
- PMC11726580
- NLM abbreviation
- ERJ Open Res
- ISSN
- 2312-0541
- eISSN
- 2312-0541
- Language
- English
- Electronic publication date
- 08/08/2024
- Date published
- 01/2025
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Radiology; Electrical and Computer Engineering; Endocrinology and Diabetes; Stead Family Department of Pediatrics; Health, Sport, and Human Physiology
- Record Identifier
- 9984696889902771
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