Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries
Disease progression in patients with cystic fibrosis treated with ivacaftor: Data from national US and UK registries
复制标题
DOI：
10.1016/j.jcf.2019.05.015
复制
发表时间：
2020-01-01
影响因子：
5.2
通讯作者：
Bilton, Diana
中科院分区：
文献类型：
--
作者：
Volkova, Nataliya;Moy, Kristin;Bilton, Diana
背景：Ivacaftor是首个针对囊性纤维化（CF）潜在缺陷的CFTR调节剂类药物。这项长期观察性安全性研究评估了在现实环境中接受ivacaftor治疗的CF患者长达5年的疾病进展。方法：来自现有美国和英国CF患者登记的数据被用于评估依活动性因子治疗与未治疗的对照队列中肺功能、营养状况、肺恶化和住院、CF相关糖尿病（CFRD）和铜绿假单胞菌的纵向模式，这些对照队列与年龄、性别和疾病严重程度相匹配。结果：美国分析包括635名接受ivacator治疗的患者和1874名对照者，从上市第1年（2012-2016年）开始随访5年。从预处理基线（2011年）到第5年（2016年）的结果模式评估显示，相对于比较组，ivacaftor治疗的患者肺功能得到了更好的保存（预测FEV1的平均百分比变化，ivacaftor组为-0.7个百分点，而比较组为-8.3个百分点），营养状况得到改善(ivacaftor组平均体重指数变化+2.4 kg/m（2)，而比较组为+1.6 kg/m(2)）。与预处理基线和比较组相比，接受ivacaftor治疗的美国患者在5年随访中病情恶化和住院的频率显著降低。CFRD和铜绿假单胞菌患病率也出现了良好的趋势。规模较小的英国登记处的调查结果在方向上与美国的调查结果相似并一致。结论：这项观察性研究代表了在现实世界中使用ivacaftor治疗的患者中最大的纵向分析。研究结果支持用ivacaftor调节CFTR来改善疾病。(C) 2019作者。Elsevier B.V.出版
Background: Ivacaftor is the first in a class of drugs, CFTR modulators, that target the underlying defect in cystic fibrosis (CF). This long-term observational safety study evaluated CF disease progression in patients treated with ivacaftor in a real-world setting for up to 5 years.Methods: Data from existing US and UK CF patient registries were used to assess longitudinal patterns in lung function, nutritional status, pulmonary exacerbations and hospitalizations, CF-related diabetes (CFRD), and Pseudomonas aeruginosa in ivacaftor-treated vs untreated comparator cohorts matched by age, sex, and disease severity.Results: US analyses included 635 ivacaftor-treated patients and 1874 comparators followed for 5 years from year 1 of market availability (2012-2016). Evaluation of outcome patterns from pretreatment baseline (2011) through year 5 (2016), showed that relative to comparators, ivacaftor-treated patients had better preserved lung function (mean change in percent predicted FEV1, -0.7 percentage points with ivacaftor vs -8.3 percentage points in comparators) and improved nutritional status (mean body mass index change +2.4 kg/m(2) with ivacaftor vs +1.6 kg/m(2) in comparators). US patients treated with ivacaftor had significantly lower frequencies of exacerbations and hospitalizations in each of the 5 years of follow-up relative to pretreatment baseline and comparators. Favorable trends in CFRD and P. aeruginosa prevalence were also observed. Findings from the smaller UK registry were directionally similar to and consistent with US findings.Conclusions: This observational study represents the largest longitudinal analysis of patients treated with ivacaftor in a real-world setting. The findings support disease modification by CFTR modulation with ivacaftor. (C) 2019 The Authors. Published by Elsevier B.V.