Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease.
Impact of CFTR modulator use on outcomes in people with severe cystic fibrosis lung disease.
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DOI：
10.1183/16000617.0112-2019
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发表时间：
2020-03-31
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影响因子：
--
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中科院分区：
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增加囊性纤维化(CF)跨膜调节因子(CFTR)的产生和活性的药物化合物使囊性纤维化的护理发生了革命性的变化。许多成人和一些患有CF的儿童患有晚期严重的肺部疾病或等待肺移植。虽然人们希望这些药物化合物在生命早期就能防止肺损伤，但仍有必要照顾患有晚期肺部疾病的人。这篇综述的重点是从临床试验和病例系列中积累的关于CFTR调节剂治疗晚期肺部疾病的益处的数据。我们讨论了使用异丙酚、异丙酚/异丙酚、替扎卡福/异丙卡福和异丙卡福/替扎卡福治疗对肺功能、肺恶化、营养和生活质量的影响。不同的CFTR调节剂的不良事件，以及潜在的药物相互作用，进行了讨论。CFTR调节剂在改善肺功能和加重病情方面是有效的，即使是在晚期CF。然而，药物不良反应和药物相互作用频繁。Http://bit.ly/37aN81E
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. CFTR modulators are efficacious in improving lung function and exacerbations, even in advanced CF. However, adverse drug reactions and drug interactions are frequent. http://bit.ly/37aN81E
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