Elexacaftor-Tezacaftor- Ivacaftor improves sinonasal outcomes in cystic fibrosis.
Elexacaftor-Tezacaftor- Ivacaftor improves sinonasal outcomes in cystic fibrosis.
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DOI：
10.1016/j.jcf.2022.03.002
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发表时间：
2022-09
影响因子：
5.2
通讯作者：
Zemke, Anna C.
中科院分区：
文献类型：
--
作者：
Stapleton, Amanda L.;Kimple, Adam J.;Goralski, Jennifer L.;Nouraie, S. Mehdi;Branstetter, Barton F.;Shaffer, Amber;Pilewski, Joseph M.;Senior, Brent A.;Lee, Stella E.;Zemke, Anna C.
许多囊性纤维化 (CF) 患者患有慢性鼻窦炎，导致鼻塞、鼻窦感染和反复手术。 Elexacaftor-tezacaftor-ivacaftor 是一种高效调节剂疗法，被批准用于 6 岁或以上患有 CF 且至少有一个 F508del 等位基因或其他反应性突变的个体。目前的研究检验了 ELX/TEZ/IVA 改善 CF 鼻窦疾病的假设。该研究是在两个地点进行的前/后观察性队列研究。参与者在开始 ELX/TEZ/IVA 之前接受了一次研究访问，并在治疗中位 9 个月时接受了第二次访问。每次就诊均包括鼻窦 CT 扫描、硬性鼻内窥镜检查和汗液氯化物测量。在研究期间，按照预定的时间间隔，通过 22 项鼻腔结果测试来测量症状。回归模型用于测试症状、内窥镜检查和 CT 量表的改善情况。该研究招募了 34 名受试者，平均年龄为 27 岁（范围 12-60 岁）。治疗后 7 天内，症状得到改善，并在第 28 天达到稳定水平。内镜下结痂得到缓解，鼻息肉病得到改善，息肉尺寸或消退程度有所减小。通过治疗，CT 射线照片显示鼻窦混浊和粘膜增厚有所改善。在 ELX/TEZ/IVA 治疗中，鼻窦症状快速且持久地改善了至少 180 天。 ELX/TEZ/IVA 改善了疾病的客观测量，包括内窥镜和 CT 检查结果。
Many individuals with cystic fibrosis (CF) have chronic rhinosinusitis resulting in nasal obstruction, sinus infections, and repeated surgeries. Elexacaftor-tezacaftor-ivacaftor is a highly effective modulator therapy approved for individuals aged 6 years or older with CF who have at least one F508del allele or other responsive mutation. The current study tests the hypothesis that ELX/TEZ/IVA improves sinonasal disease in CF. The study was a pre/post, observational cohort study conducted at two sites. Participants underwent a study visit prior to starting ELX/TEZ/IVA and a second visit at a median of 9 months on therapy. Each visit included sinus CT scan, rigid nasal endoscopy, and sweat chloride measurement. Symptoms were measured with the 22 item Sinonasal Outcome Test at scheduled intervals during the study. Regression models were used to test for improvement in symptoms, endoscopy, and CT scales. The study enrolled 34 individuals, with a median age of 27 years (range 12–60). Symptoms improved within 7 days of therapy and plateaued by day 28. Endoscopic crusting resolved and nasal polyposis improved, with a decrease in size or resolution of polyps. Sinus opacification and mucosal thickening improved on CT radiographs with treatment. Sinonasal symptoms improved rapidly and durably for at least 180 days on ELX/TEZ/IVA therapy. Objective measures of disease including endoscopic and CT findings improved with ELX/TEZ/IVA.
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影响因子：
6.4
作者：
Bacon DR;Stapleton A;Goralski JL;Ebert CS Jr;Thorp BD;Nouraie M;Shaffer AD;Senior BA;Lee SE;Zemke AC;Kimple AJ
通讯作者：
Kimple AJ
影响因子：
8.3
作者：
Habib, Al-Rahim R.;Buxton, Jane A.;Quon, Bradley S.
通讯作者：
Quon, Bradley S.
影响因子：
7.8
作者：
Chan, Dylan K.;McNamara, Sharon;Parikh, Sanjay R.
通讯作者：
Parikh, Sanjay R.
影响因子：
5.2
作者：
Beswick, Daniel M.;Humphries, Stephen M.;Taylor-Cousar, Jennifer L.
通讯作者：
Taylor-Cousar, Jennifer L.
影响因子：
5.2
作者：
Rasmussen, Jacob;Aanaes, Kasper;von Buchwald, Christian
通讯作者：
von Buchwald, Christian