Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy: A Review for the Otolaryngologist.
Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy: A Review for the Otolaryngologist.
复制标题
DOI：
10.1177/1945892420912368
复制
发表时间：
2020-07
影响因子：
2.6
通讯作者：
Kimple AJ
中科院分区：
文献类型：
--
作者：
Lee SE;Farzal Z;Daniels MLA;Thorp BD;Zanation AM;Senior BA;Ebert CS Jr;Kimple AJ
囊性纤维化（CF）是一种遗传性疾病，可导致多种全身性疾病和潜在致命的严重呼吸系统损害。然而，CF跨膜传导调节因子（CFTR）调节剂的出现改变了选择性突变CF患者的管理。尽管临床试验强调了这些新疗法增加肺功能和减少恶化的结果，但它们对鼻窦的影响尚未得到很好的描述。我们的目的是回顾CFTR调节剂，为经常护理CF患者的耳鼻喉科医生提供对这些药物及其对CF患者慢性鼻窦炎（CRS）的作用的基本了解。本文综述了临床批准和可用的CFTR调节剂及其使用的具体适应症。此外，对这些治疗方法及其对CF患者CRS的影响进行了系统回顾。目前有四种CFTR调节剂被fda批准用于CF患者。目前的药物被批准用于门控、残余功能或F508del突变。多篇报道描述了CFTR调节剂在鼻上皮培养中增加了跨上皮离子运输；然而，关于这些调节剂对鼻腔健康影响的临床研究仅限于5项研究，这些研究提供了CFTR调节剂在CRS中作用的新数据。CFTR调节剂已经改变了CF的治疗。这些药物的初步研究显示CF有很好的效果；然而，尽管初步结果令人鼓舞，但描述CFTR调制器对cf相关CRS影响的文献很少。
Cystic fibrosis (CF) is a genetic disease that may result in multiple systemic disorders and potentially fatal severe respiratory compromise. However, the advent of CF transmembrane conductance regulator (CFTR) modulators has changed the management of CF for patients with select mutations. Although clinical trials have highlighted increased pulmonary function and decreased exacerbations as a result of these novel therapies, their effect on the sinuses has not been well-described. Our objective is to review the CFTR modulators to provide otolaryngologists, physicians who frequently care for patients with CF, a basic understanding of these drugs and their effects on chronic rhinosinusitis (CRS) in patients with CF. The clinically approved and available CFTR modulators and specific indications for their use are reviewed. Additionally, a systematic review of these therapies and effects on CRS in CF was performed. Four Food and Drug Administration approved CFTR modulators are available for patients with CF. Current drugs are approved for gating, residual function, or F508del mutations. Multiple reports describe CFTR modulators’ increase in transepithelial ion transport in nasal epithelial cultures; however, clinical studies regarding effects of these modulators on sinonasal health are limited to 5 studies that present new data of the effects of CFTR modulators in CRS. CFTR modulators have changed management of CF. Initial studies of these medications demonstrate promising results in CF; however, there is a paucity of literature describing the effect of CFTR modulators on CF-associated CRS, although initial results are encouraging.
登录
查看更多内容
影响因子：
168.9
作者：
Heijerman, Harry G. M.;McKone, Edward F.;Mccoy, Karen S.
通讯作者：
Mccoy, Karen S.
影响因子：
3.1
作者：
Talamo Guevara M;McColley SA
通讯作者：
McColley SA
影响因子：
6.4
作者：
Lim, Dong-Jin;McCormick, Justin;Cho, Do-Yeon
通讯作者：
Cho, Do-Yeon
影响因子：
3.9
作者：
Brewington, John J.;McPhail, Gary L.;Clancy, John P.
通讯作者：
Clancy, John P.
影响因子：
1.1
作者：
Guigui, S.;Wang, J.;Cohen, R. I.
通讯作者：
Cohen, R. I.