Impact of Cystic Fibrosis Transmembrane Conductance Regulator Therapy on Chronic Rhinosinusitis and Health Status: Deep Learning CT Analysis and Patient-reported Outcomes.
Impact of Cystic Fibrosis Transmembrane Conductance Regulator Therapy on Chronic Rhinosinusitis and Health Status: Deep Learning CT Analysis and Patient-reported Outcomes.
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DOI：
10.1513/annalsats.202101-057oc
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发表时间：
2022-01
影响因子：
8.3
通讯作者：
Taylor-Cousar JL
中科院分区：
文献类型：
--
作者：
Beswick DM;Humphries SM;Balkissoon CD;Strand M;Vladar EK;Lynch DA;Taylor-Cousar JL
Elexacaftor，tezacaftor和ivacaftor（ETI）三联组合可改善囊性纤维化（PwCF）患者的肺部健康。然而，它对鼻窦疾病和健康效用的客观衡量标准的影响尚未确定。评估ETI对慢性鼻窦炎（CRS）和一般健康状况的影响，包括计算机断层扫描（CT）、生活质量（QOL）和生产力损失。具有CF跨膜传导调节因子基因型F508 del/F508 del或F508 del/最小功能的临床启动ETI的成人PwCF+CRS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的ETI 6个月后鼻窦CT混浊百分比（%SO）的变化。次要终点包括鼻窦QOL、健康效用值和生产力损失的变化，通过经验证的指标每月进行评估。30个PwCF提供了基线数据; 25个完成了研究。基线时，该队列具有实质性CRS，平均22个问题的鼻窦结局测试（SNOT-22）评分为33.1，平均鼻窦CT %SO为63.7%。6个月随访时，%SO平均改善22.9%（P < 0.001）。对于既往未接受过调节剂治疗的患者，%SO改善趋势更大（P = 0.09）。平均SNOT-22评分和健康效用分别提高了15.3和0.068 [6.8%]（均P <0.007）。出席，活动障碍和整体生产力损失改善（所有P <0.049）。SNOT-22评分和健康效用的改善在一个月内发生，并在研究期间保持改善。ETI与鼻窦CT混浊和生产力损失的实质性改善以及鼻窦QOL和健康效用的临床有意义的改善相关。在研究中，大多数改善都是快速、稳健和持久的。
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% (P < 0.001). %SO improvement trended toward greater magnitude for those naïve to prior modulator therapy (P = 0.09). Mean SNOT-22 scores and health utility improved by 15.3 and 0.068 [6.8%] (all P ⩽ 0.007). Presenteeism, activity impairment and overall productivity loss improved (all P ⩽ 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.