Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation
Effects of ivacaftor on severely ill patients with cystic fibrosis carrying a G551D mutation
复制标题
DOI：
10.1016/j.jcf.2013.05.006
复制
发表时间：
2013-12-01
影响因子：
5.2
通讯作者：
Mainz, Jochen G.
中科院分区：
文献类型：
--
作者：
Hebestreit, Helge;Sauer-Heilborn, Annette;Mainz, Jochen G.
背景资料：最近，CFTR增效剂ivacaftor已被证明对携带G551D突变和中度肺功能受损的囊性纤维化患者有效且安全。这项回顾性研究的目的是评估依伐卡托在至少有一个G551D突变的重症患者中的疗效和安全性。
Background: Recently, ivacaftor, a CFTR-potentiator, has been shown to be effective and safe in patients with cystic fibrosis carrying a G551D mutation and moderately impaired lung function. The objective of this retrospective study was to assess efficacy and safety of ivacaftor in severely ill patients with at least one G551D mutation.Methods: Data from 14 patients with a FEV1