Pulmonary Institute and CF Center, Carmel Medical Center, Haifa, Israel
B. Rappaport Faculty of Medicine, Technion-Israel Institute of Technology, Haifa, Israel.
Eur Respir Rev. 2020 Mar 20;29(155). doi: 10.1183/16000617.0112-2019. Print 2020 Mar 31.
Drug compounds that augment the production and activity of the cystic fibrosis (CF) transmembrane regulator (CFTR) have revolutionised CF care. Many adults and some children with CF suffer advanced and severe lung disease or await lung transplantation. While the hope is that these drug compounds will prevent lung damage when started early in life, there is an ongoing need to care for people with advanced lung disease. The focus of this review is the accumulating data from clinical trials and case series regarding the benefits of CFTR modulator therapy in people with advanced pulmonary disease. We address the impact of treatment with ivacaftor, lumacaftor/ivacaftor, tezacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor on lung function, pulmonary exacerbations, nutrition and quality of life. Adverse events of the different CFTR modulators, as well as the potential for drug-drug interactions, are discussed.
能够增加囊性纤维化跨膜转导调节因子（CFTR）的产生和活性的药物化合物彻底改变了囊性纤维化的治疗方式。许多患有囊性纤维化的成年人和一些儿童患有晚期和严重的肺部疾病，或者等待肺移植。虽然人们希望这些药物化合物在生命早期开始时可以预防肺部损伤，但仍需要持续关注患有晚期肺部疾病的人群。本综述的重点是关于 CFTR 调节剂治疗对晚期肺部疾病患者的益处的临床试验和病例系列的累积数据。我们探讨了 ivacaftor、lumacaftor/ivacaftor、tezacaftor/ivacaftor 和 elexacaftor/tezacaftor/ivacaftor 治疗对肺功能、肺部恶化、营养和生活质量的影响。讨论了不同 CFTR 调节剂的不良反应事件，以及药物相互作用的可能性。
