TY -的T1 -试图找到治疗囊性fibrosis: CFTR biomarkers as outcomes JF - European Respiratory Journal JO - Eur Respir J SP - 1156 LP - 1157 DO - 10.1183/09031936.00029913 VL - 42 IS - 4 AU - De Boeck, Kris Y1 - 2013/10/01 UR - //www.qdcxjkg.com/content/42/4/1156.abstract N2 - From the authors:We thank P. Lebecque for his interest in our paper [1]. His comment as to whether measurement of cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers are truly response measures relates to the recent paper by Durmowicz et al. [2] entitled “Change in sweat chloride as a clinical end point in cystic fibrosis clinical trials: the ivacaftor experience”. These authors also point towards the uncertainty of using established measurements such as sweat chloride as a clinical end-point.We are indeed in the early days of clinical use of CFTR modulators and much is to be learned about how improving CFTR biomarkers relates to clinical efficacy. In our further discussion, we will, as Durmowicz et al. [2] do, discuss only ivacaftor and lumacaftor monotherapy [3–5], since data … ER -