The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol
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 fibr...
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| Language: | English |
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European Respiratory Society
2025-01-01
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| Series: | ERJ Open Research |
| Online Access: | http://openres.ersjournals.com/content/11/1/00137-2024.full |
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| author | 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 |
| author_facet | 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 |
| author_sort | Christine M. Liu |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-13ece56839174cbca6340602165b2d72 |
| institution | DOAJ |
| issn | 2312-0541 |
| language | English |
| publishDate | 2025-01-01 |
| publisher | European Respiratory Society |
| record_format | Article |
| series | ERJ Open Research |
| spelling | doaj-art-13ece56839174cbca6340602165b2d722025-08-20T03:02:16ZengEuropean Respiratory SocietyERJ Open Research2312-05412025-01-0111110.1183/23120541.00137-202400137-2024The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocolChristine M. Liu0Jakob L. Fischer1Edith T. Zemanick2Jason C. Woods3Karolin K. Markarian4Sean B. Fain5Deborah Froh6Sonya L. Heltshe7Lucas R. Hoffman8Stephen M. Humphries9Elizabeth L. Kramer10Katie Larson Ode11Michael Lewis12Douglas A. Li13Jaime Mata14Sarah S. Milla15Peter J. Niedbalski16Benjamin D. Sawatzky17Myung-Shin Sim18Jillian S. Sullivan19Andrew T. Trout20Christopher H. Goss21Jennifer L. Taylor-Cousar22Daniel M. Beswick23 Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA Department of Pediatrics, University of Colorado School of Medicine, Aurora, CO, USA Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA University of California, Los Angeles, CTSI, David Geffen School of Medicine, Los Angeles, CA, USA Department of Radiology, University of Iowa, Iowa City, IA, USA Department of Pediatrics, University of Virginia, Charlottesville, VA, USA Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA, USA Department of Pediatrics, University of Washington and Seattle Children's Research Institute, Seattle, WA, USA Department of Radiology, National Jewish Health, Aurora, CO, USA Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA Department of Pediatrics, University of Iowa, Iowa City, IA, USA Department of Pediatrics, University of Kansas Medical Center, Kansas City, KS, USA Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA Department of Radiology and Medical Imaging, University of Virginia, Charlottesville, VA, USA Department of Radiology, Children's Hospital Colorado, Aurora, CO, USA Department of Pulmonary, Critical Care, and Sleep Medicine, University of Kansas Medical Center, Kansas City, KS, USA Department of Radiology, University of Vermont, Colchester, VT, USA Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA Department of Pediatrics, University of Vermont, Colchester, VT, USA Department of Pediatrics, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA Department of Medicine and Pediatrics, University of Washington, Seattle, WA, USA Department of Medicine, National Jewish Health, Aurora, CO, USA Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, Los Angeles, CA, USA 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.http://openres.ersjournals.com/content/11/1/00137-2024.full |
| spellingShingle | 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 The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol ERJ Open Research |
| title | The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol |
| title_full | The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol |
| title_fullStr | The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol |
| title_full_unstemmed | The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol |
| title_short | The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol |
| title_sort | impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis a prospective study protocol |
| url | http://openres.ersjournals.com/content/11/1/00137-2024.full |
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