Liu Christine M, Fischer Jakob L, Zemanick Edith T, Woods Jason C, Markarian Karolin K, Fain Sean B, Froh Deborah, Heltshe Sonya L, Hoffman Lucas R, Humphries Stephen M, Kramer Elizabeth L, Ode Katie Larson, Lewis Michael, Li Douglas A, Mata Jaime, Milla Sarah S, Niedbalski Peter J, Sawatzky Benjamin D, Sim Myung-Shin, Sullivan Jillian S, Trout Andrew T, Goss Christopher H, Taylor-Cousar Jennifer L, Beswick Daniel M
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.
ERJ Open Res. 2025 Jan 13;11(1). doi: 10.1183/23120541.00137-2024. eCollection 2025 Jan.
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.
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).
Enrolment began in April 2023. 21 participants have been enrolled as of October 2023 with ongoing enrolment at all sites.
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.
慢性鼻-鼻窦炎（CRS）和嗅觉功能障碍（OD）是囊性纤维化患者中常见的疾病并发症。这些研究较少的合并症对生活质量有显著影响。高效调节剂疗法（HEMT）对患有囊性纤维化的幼儿（YCwCF）这些疾病并发症的影响尚不清楚。本拟开展的研究旨在描述YCwCF中CRS和OD的特征，并评估HEMT在改善这一年轻年龄组鼻窦和嗅觉健康方面的疗效。
这项六中心、前瞻性、观察性研究将招募80名2至8岁的YCwCF。根据临床指征，患者分为两组：接受HEMT的患者和未接受HEMT的患者。两组均接受鼻窦磁共振成像、心理物理学嗅觉测试，并在2年内完成患者或家长报告的生活质量调查。将在开始HEMT之前和之后以及组间比较结果。所有研究点均已获得伦理批准，本研究已在ClinicalTrials.gov（NCT06191640）上注册。
2023年4月开始招募。截至2023年10月，已有21名参与者入组，所有研究点的招募工作仍在进行中。
这项研究预计将为早期启动HEMT在管理YCwCF的CRS和OD方面的潜在益处提供关键见解。它将有助于制定有针对性的干预措施，并有助于理解HEMT在改变这一人群疾病进程中的作用。
