elexacaftor/tezacaftor/ivacaftor CFTR 调节器疗法对囊性纤维化患者内窥镜鼻窦手术率的影响。

IF 7.2 2区 医学 Q1 OTORHINOLARYNGOLOGY International Forum of Allergy & Rhinology Pub Date : 2024-07-03 DOI:10.1002/alr.23400
Graham Pingree BA, Mihai Bentan BS, Thomas Fitzpatrick MD, Theodore Schuman MD
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引用次数: 0

摘要

背景:Elexacaftor/tezacaftor/ivacaftor(ETI)是一种囊性纤维化跨膜受体(CFTR)联合调节剂,已证明可改善囊性纤维化(CF)患者的肺部预后。然而,ETI 对功能性内窥镜鼻窦手术(FESS)的影响仍不清楚:方法:在 TriNetX Analytics 研究网络中查询了 2012 年至 2023 年期间接受鼻窦手术的 CF 患者,该网络包含 1.2 亿份全球去标识化电子病历。根据相关因素,将在接受 FESS 之前服用 ETI 的患者(n = 6056)与未服用 CFTR 调节剂的对照组 CF 患者(n = 37906)和服用其他 FDA 批准的 CFTR 调节剂(tezacaftor/ivacaftor、lumacaftor/ivacaftor 和 ivacaftor)的患者(n = 2437)进行倾向评分匹配。主要结果是接受 FESS 的绝对风险降低率 (ARR)。次要结果包括接受 FESS 后 0 至 6 个月、6 至 12 个月和 12 至 24 个月 CF 相关肺部恶化和入院的绝对风险降低率:结果:与未使用 CFTR 调节剂的 CF 患者相比,使用 ETI 对 FESS 有显著的 ARR(2.12%;95% 置信区间 [CI]:1.5-2.75;P 值):在一个大型数据集中,与未使用 CFTR 调节剂或使用其他 CFTR 调节剂的患者相比,使用 ETI 的 CF 患者发生 FESS、肺部恶化和入院的风险显著降低,这表明 CF 患者的鼻窦疾病和整体健康状况有所改善。
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Impact of elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy on rates of endoscopic sinus surgery in cystic fibrosis

Background

Elexacaftor/tezacaftor/ivacaftor (ETI), a combination cystic fibrosis transmembrane receptor (CFTR) modulator, has demonstrated improved pulmonary outcomes in individuals with cystic fibrosis (CF). However, ETI's impact on functional endoscopic sinus surgery (FESS) remains unclear.

Methods

The TriNetX Analytics Research Network, consisting of 120 million global de-identified electronic medical records, was queried from 2012 to 2023 for subjects with CF who underwent sinus surgery.1 Patients on ETI prior to FESS (n = 6,056) were propensity score matched to control individuals with CF not on CFTR modulators (n = 37,906) and those on other FDA-approved CFTR modulators (tezacaftor/ivacaftor, lumacaftor/ivacaftor, and ivacaftor) (n = 2437) based on relevant factors. The primary outcome was the absolute risk reduction (ARR) of undergoing FESS. Secondary outcomes included ARR of CF-related pulmonary exacerbations and hospital admission from 0 to 6, 6 to 12, and 12 to 24 months following FESS.

Results

ETI use demonstrated a significant ARR for FESS when compared to CF patients not on CFTR modulators (2.12%; 95% confidence interval [CI] 1.5–2.75; p-value < 0.0001) and those on other CFTR modulators (4.7%; 95% CI 3.54–5.85; p-value < 0.0001). No significant differences occurred in secondary outcomes between ETI and non-CFTR modulator groups, except for reduced CF-related pulmonary exacerbations from 0 to 6 months post-FESS. Additionally, a significant reduction in pulmonary exacerbations was observed at all time points and hospital admissions within 6 months following FESS compared to those using other CFTR modulators.

Conclusions

In a large dataset, CF patients on ETI demonstrated significantly reduced risk of FESS, pulmonary exacerbations, and hospital admission compared to patients not on CFTR modulators or those on other CFTR modulators, suggesting improved sinonasal disease and overall health status in CF.

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来源期刊
CiteScore
11.70
自引率
10.90%
发文量
185
审稿时长
6-12 weeks
期刊介绍: International Forum of Allergy & Rhinologyis a peer-reviewed scientific journal, and the Official Journal of the American Rhinologic Society and the American Academy of Otolaryngic Allergy. International Forum of Allergy Rhinology provides a forum for clinical researchers, basic scientists, clinicians, and others to publish original research and explore controversies in the medical and surgical treatment of patients with otolaryngic allergy, rhinologic, and skull base conditions. The application of current research to the management of otolaryngic allergy, rhinologic, and skull base diseases and the need for further investigation will be highlighted.
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