Manchester Adult Cystic Fibrosis Centre, University Hospital of South Manchester, Manchester, UK;
Manchester Adult Cystic Fibrosis Centre, University Hospital of South Manchester, Manchester, UK; Institute of Inflammation and Repair, University of Manchester, Manchester, UK.
Thorax. 2014 Jun;69(6):586-7. doi: 10.1136/thoraxjnl-2013-204532. Epub 2013 Nov 20.
Clinical trials have revealed that Ivacaftor significantly reduces sweat chloride in patients with cystic fibrosis who carry the G551D mutation. This finding has been incorporated into the commissioning guidelines in the UK with a sweat chloride reduction of 30% or below 60 mmol/L, specified as the main criteria for continued funding of Ivacaftor for individual patients. In a cohort of 24 adults who were prescribed Ivacaftor, there was no correlation between absolute or relative reductions in sweat chloride and improvements in lung function. This questions the validity of sweat chloride as a surrogate marker of clinical efficacy.
临床试验表明，伊伐卡托可显著降低携带 G551D 突变的囊性纤维化患者的汗液氯化物水平。这一发现已被纳入英国的委托指南，规定汗液氯化物降低 30%或低于 60mmol/L 作为继续为个别患者提供伊伐卡托资助的主要标准。在 24 名接受伊伐卡托治疗的成年患者队列中，汗液氯化物的绝对或相对降低与肺功能的改善之间没有相关性。这使得汗液氯化物作为临床疗效替代标志物的有效性受到质疑。
