Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, California.
Department of Radiology.
Ann Am Thorac Soc. 2022 Jan;19(1):12-19. doi: 10.1513/AnnalsATS.202101-057OC.
Elexacaftor, tezacaftor, and ivacaftor (ETI) in triple combination improves pulmonary health for people with cystic fibrosis (PwCF). However, its impact on objective measures of sinus disease and health utility is unestablished. To evaluate the impact of ETI on chronic rhinosinusitis (CRS) and general health status incorporating computed tomography (CT), quality-of-life (QOL) and productivity loss. Adult PwCF+CRS with CF transmembrane conductance regulator genotype F508del/F508del or F508del/minimal function who clinically initiated ETI participated in a prospective, observational study. The primary endpoint was change in percent sinus CT opacification (%SO) after 6 months of ETI assessed via deep learning-based methods. Secondary endpoints included changes in sinonasal QOL, health utility value and productivity loss, which were evaluated monthly via validated metrics. 30 PwCF provided baseline data; 25 completed the study. At baseline, the cohort had substantial CRS, with mean 22-question SinoNasal Outcome Test (SNOT-22) score 33.1 and mean sinus CT %SO 63.7%. At 6-month follow-up, %SO improved by mean 22.9% ( < 0.001). %SO improvement trended toward greater magnitude for those naïve to prior modulator therapy ( = 0.09). Mean SNOT-22 scores and health utility improved by 15.3 and 0.068 [6.8%] (all ⩽ 0.007). Presenteeism, activity impairment and overall productivity loss improved (all ⩽ 0.049). Improvements in SNOT-22 scores and health utility occurred by one month and remained improved over the study. ETI is associated with substantial improvements in sinus CT opacification and productivity loss, and clinically meaningful improvements in sinonasal QOL and health utility. Most improvements were rapid, robust, and durable over the study.
依伐卡托、泰它卡托和艾维卡托三联组合改善了囊性纤维化（CF）患者的肺部健康。然而，其对鼻窦疾病和健康效用的客观测量的影响尚未确定。评估三联组合对慢性鼻-鼻窦炎（CRS）的影响，以及包括计算机断层扫描（CT）、生活质量（QOL）和生产力损失在内的一般健康状况。患有 CF 跨膜电导调节因子基因型 F508del/F508del 或 F508del/最小功能的成年 CF 伴 CRS 患者，在临床开始使用依伐卡托三联组合后，参与了一项前瞻性、观察性研究。主要终点是通过基于深度学习的方法评估 6 个月时依伐卡托三联组合治疗后鼻窦 CT 混浊百分比（%SO）的变化。次要终点包括通过验证后的指标评估的每月变化，包括鼻-鼻窦生活质量、健康效用值和生产力损失的变化。30 名 CF 患者提供了基线数据；25 名患者完成了研究。在基线时，该队列具有显著的 CRS，平均 22 项鼻-鼻窦炎结局测试（SNOT-22）评分为 33.1，鼻窦 CT 混浊百分比（%SO）平均为 63.7%。在 6 个月的随访时，%SO 平均改善了 22.9%（ ⁇ 0.001）。对于那些之前未接受调节剂治疗的患者，%SO 改善的幅度更大（ ⁇ = 0.09）。平均 SNOT-22 评分和健康效用值分别改善了 15.3 和 0.068 [6.8%]（均 ⁇ 0.007）。出勤障碍、活动障碍和总体生产力损失得到改善（均 ⁇ 0.049）。SNOT-22 评分和健康效用的改善在一个月内出现，并在研究期间保持改善。依伐卡托三联组合与鼻窦 CT 混浊和生产力损失的显著改善以及鼻-鼻窦生活质量和健康效用的有临床意义的改善相关。大多数改善在研究期间是迅速、稳健和持久的。
