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.
Footnotes
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Conflict of interest: CML: NIDCD grant related to this work.
Conflict of interest: JLF: None to disclose.
Conflict of interest: ETZ: In the last 36 months, she has received grants to her institution from the Cystic Fibrosis Foundation, National Institutes of Health, and Vertex Pharmaceuticals Incorporated; has received fees from the Cystic Fibrosis Foundation and Vertex Pharmaceuticals Incorporated related to consultation, participation on advisory boards, and grant review committees. She served as chair of the CFF Therapeutics Development Network steering committee, reviewed grants for CF Canada, and received travel support from the European CF Society Clinical Trials Network for speaking at their annual meeting.
Conflict of interest: JCW: In the last 36 months, he has received grants to his institution from the Cystic Fibrosis Foundation, the National Institutes of Health, Vertex Pharmaceuticals Incorporated; has received fees from Polarean LLC related to consultation on clinical and translational research.
Conflict of interest: KKM: None to disclose.
Conflict of interest: SBF: In the last 36 months, SBF served as a scientific advisor for Polarean LLC, and received research funding from Polarean LLC, Siemens Healthineers INC, and GE Healthcare INC for development of pulmonary CT and MRI methods.
Conflict of interest: DF: In the lasts 36 months, DF has received grant support from the CF Foundation unrelated to this work and Vertex Pharmaceuticals
Conflict of interest: SLH: In the last 36 months SH received grant support from the Cystic Fibrosis Foundation. Unrelated to this work he has received grant support from National Institutes of Health and Boehringer Ingelheim, as well as service contracts from Calyx paid to his institution.
Conflict of interest: LRH: In the past 36 months, LRH received grant funding from the CF Foundation and the NIH.
Conflict of interest: ELK: In the last 36 months, she has received grant support from the National Institutes of Health and the Cystic Fibrosis Foundation.
Conflict of interest: KLO: In the last 36 months, she has received grant support from the National Institutes of Health, the Cystic Fibrosis Foundation, and Rhythm Pharmaceuticals, Inc unrelated to this work
Conflict of interest: ML: ML participated in an advisory board serving as a consult for Pfizer to discuss RSV vaccines on 9/20/2023.
Conflict of interest: DAL: In the last 36 months, DAL has received grant support from the CF Foundation unrelated to this work.
Conflict of interest: JM: In the last 36 months, he has received grants to his institution from the Cystic Fibrosis Foundation, the National Institutes of Health, the Focused Ultrasound Foundation, iThriv and Polarean LLC; He has received fees from Polarean LLC related to consultation on clinical and translational research.
Conflict of interest: SSM: None to disclose.
Conflict of interest: PJN: In the last 36 months, he has received grants from the National Institutes of Health, American Heart Association, and Scleroderma Foundation.
Conflict of interest: BDS: None to disclose.
Conflict of interest: MSS: None to disclose.
Conflict of interest: JSS: In the last 36 months, JSS has received honoraria from the American Academy of Pediatrics to serve on the PREP-SA editorial board, unrelated to this work
Conflict of interest: ATT: In the last 36 months, he has received grant to his institution from the Cystic Fibrosis Foundation, National Institutes of Health, GE Healthcare, Siemens Healthineers, and Perspectum Inc. He is a consultant for GE Healthcare. He serves as an Assistant Editor for Pediatric Radiology.
Conflict of interest: CHG: In the last 36 months, he has received grants from the National Institutes of Health, the Cystic Fibrosis Foundation, the Federal Drug Administration; has received fees from Enterprise Therapeutics for providing clinical trial design advice. He received honoraria from Gilead Sciences to serve as grant review committee chair and from Vertex Pharmaceuticals for speaking at the UK LEAD conference. He served as a DSMB Chair for a trial supported by Novartis and the European Commission. He serves as the Deputy Editor of the Annals of the American Thoracic Society. He has stock in Air Therapeutics.
Conflict of interest: JLTC: Personal financial relationships with commercial interests relevant to medicine within the past 3 years: As faculty in an institution that is part of the CF TDN, I have been site Pl on studies for Vertex, 4DMT, and Eloxx; I have done consulting/provided clinical trial design advice for Vertex and 4DMT; I served as Chair of a Data Monitoring Committee for AbbVie (complete). Personal financial support from a non-commercial source relevant to medicine within the past 3 years: I have received grant funding from the CFF and NIH. Professional Memberships: CFF Clinical Research Executive Committee; CFF TDN Sexual Health, Reproduction, and Gender Research Working Group (SHARING); CFF Racial Justice Working Group; ATS International Conference Committee Chair-Elect and Respiratory Health Awards Committee Member (Scientific Grant Review and Clinical Problems Programming Committee-complete); Emily's Entourage Scientific Advisory Board; National Institutes of Health/National Heart, Blood, Lung Institute Clinical Trials Review Study Section.
Conflict of interest: DMB: In the last 36 months, DMB has received grant support from CF Foundation and American Rhinologic Society. Unrelated to this work, DMB has received grant support from the International Society of Inflammation and Allergy of the Nose and the American Rhinologic Society, honoraria, and consulting fees from Amgen, on medicolegal cases, and at Garner Health (equity).
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- Received February 12, 2024.
- Accepted July 9, 2024.
- Copyright ©The authors 2024
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