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
{"title":"高效调节剂治疗对囊性纤维化患儿鼻窦炎和嗅觉障碍的影响：一项前瞻性研究方案。","authors":"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","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>Enrolment began in April 2023. 21 participants have been enrolled as of October 2023 with ongoing enrolment at all sites.</p><p><strong>Conclusion: </strong>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.</p>","PeriodicalId":11739,"journal":{"name":"ERJ Open Research","volume":"11 1","pages":""},"PeriodicalIF":4.3000,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726580/pdf/","citationCount":"0","resultStr":"{\"title\":\"The impact of highly effective modulator therapy on sinusitis and dysosmia in young children with cystic fibrosis: a prospective study protocol.\",\"authors\":\"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\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>Enrolment began in April 2023. 21 participants have been enrolled as of October 2023 with ongoing enrolment at all sites.</p><p><strong>Conclusion: </strong>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.</p>\",\"PeriodicalId\":11739,\"journal\":{\"name\":\"ERJ Open Research\",\"volume\":\"11 1\",\"pages\":\"\"},\"PeriodicalIF\":4.3000,\"publicationDate\":\"2025-01-13\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11726580/pdf/\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"ERJ Open Research\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1183/23120541.00137-2024\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"2025/1/1 0:00:00\",\"PubModel\":\"eCollection\",\"JCR\":\"Q1\",\"JCRName\":\"RESPIRATORY SYSTEM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"ERJ Open Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1183/23120541.00137-2024","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/1/1 0:00:00","PubModel":"eCollection","JCR":"Q1","JCRName":"RESPIRATORY SYSTEM","Score":null,"Total":0}
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 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.
期刊介绍：
ERJ Open Research is a fully open access original research journal, published online by the European Respiratory Society. The journal aims to publish high-quality work in all fields of respiratory science and medicine, covering basic science, clinical translational science and clinical medicine. The journal was created to help fulfil the ERS objective to disseminate scientific and educational material to its members and to the medical community, but also to provide researchers with an affordable open access specialty journal in which to publish their work.
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