Department of Otolaryngology/Head and Neck Surgery, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
Department of Medicine, Division of Pulmonary and Critical Care Medicine, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina.
Am J Rhinol Allergy. 2020 Jul;34(4):573-580. doi: 10.1177/1945892420912368. Epub 2020 Mar 13.
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.
囊性纤维化（CF）是一种遗传性疾病，可能导致多种全身疾病，并可能导致严重的呼吸功能衰竭。然而，CF 跨膜电导调节因子（CFTR）调节剂的出现改变了具有特定突变的 CF 患者的治疗方法。尽管临床试验强调了这些新疗法可增加肺功能和减少恶化，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，为耳鼻喉科医生（经常照顾 CF 患者的医生）提供对这些药物的基本了解，以及它们对 CF 患者慢性鼻-鼻窦炎（CRS）的影响。
回顾了经临床批准和可获得的 CFTR 调节剂及其使用的具体适应证。此外，还对这些疗法及其对 CF 中 CRS 的影响进行了系统评价。
目前有 4 种 CFTR 调节剂可用于 CF 患者。目前的药物被批准用于门控、残余功能或 F508del 突变。多项报告描述了 CFTR 调节剂在鼻上皮培养物中增加跨上皮离子转运；然而，关于这些调节剂对鼻-鼻窦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 中影响的新数据。
CFTR 调节剂改变了 CF 的治疗方法。这些药物的初步研究显示出 CF 中很有前景的结果；然而，关于 CFTR 调节剂对 CF 相关 CRS 的影响的文献很少，尽管初步结果令人鼓舞。
