高效调节剂治疗对囊性纤维化微生物组和炎症的影响。
The Impact of Highly Effective Modulator Therapy on Cystic Fibrosis Microbiology and Inflammation.
机构信息
Department of Pediatrics, University of Michigan Medical School, L2221 UH South, 1500 East Medical Center Drive, Ann Arbor, MI 48109-5212, USA.
Department of Pediatrics, College of Physicians and Surgeons, Columbia University, Columbia University Medical Center, 650West 168th Street, New York, NY 10032, USA.
出版信息
Clin Chest Med. 2022 Dec;43(4):647-665. doi: 10.1016/j.ccm.2022.06.007.
Abstract
Highly effective cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator therapy (HEMT) corrects the underlying molecular defect causing CF disease. HEMT decreases symptom burden and improves clinical metrics and quality of life for most people with CF (PwCF) and eligible cftr mutations. Improvements in measures of pulmonary health suggest that restoration of function of defective CFTR anion channels by HEMT not only enhances airway mucociliary clearance, but also reduces chronic pulmonary infection and inflammation. This article reviews the evidence for how HEMT influences the dynamic and interdependent processes of infection and inflammation in the CF airway, and what questions remain unanswered.
摘要
高效的囊性纤维化跨膜电导调节因子（CFTR）调节剂治疗（HEMT）纠正了导致 CF 疾病的潜在分子缺陷。HEMT 降低了大多数 CF 患者（PwCF）和有资格的 cftr 突变患者的症状负担，并改善了临床指标和生活质量。肺健康指标的改善表明，HEMT 通过恢复有缺陷的 CFTR 阴离子通道的功能，不仅增强了气道黏液纤毛清除功能，还减少了慢性肺部感染和炎症。本文综述了 HEMT 如何影响 CF 气道中感染和炎症的动态和相互依存过程的证据，以及仍未解答的问题。
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