Introduction: Elexacaftor, Tezacaftor, Ivacaftor (ETI) became available in the UK in August 2020 to treat people with Cystic Fibrosis (CF) aged > 12 years. We report a real-world study of clinical outcomes in young people treated with ETI at our CF centre within the first two years of its availability. Methods: Participants aged 12 to 17 were identified within our clinic, with demographic data supplemented by the UK CF registry. Comprehensive outcome data spanning two years pre and two years post-initiation of CFTR modulators were compiled from various local sources, including patient records, medication delivery logs, and clinical notes. Results: Of the 62 patients started on ETI (32 male, mean age 13.3 years), most (76%) were homozygous for the F508del mutation. Three discontinuations occurred: one pregnancy, two related to side effects. Adherence was high (Proportion of Days covered >90% both years). Following ETI initiation there was a significant increase in mean FEV1% (+11.7 units; 95% CI 7.4 - 15.6), sustained throughout the two-year treatment period. There was no association between baseline lung function and the degree of improvement or rate of decline post-treatment. Improvements were similar for all treatable genotypes. There was a small increase in BMI z-score at four months of treatment, returning to baseline by 24 months. There was a marked reduction in the need for intravenous antibiotics. Conclusions: ETI use in adolescents in a real-world setting led to sustained improvements in health outcomes, consistent with those seen in open trial extension studies

中文翻译：

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Elexacaftor-Tezacaftor-Ivacaftor 治疗对年轻囊性纤维化患者的现实影响：一项纵向研究

简介：Elexacaftor、Tezacaftor、Ivacaftor (ETI) 于 2020 年 8 月在英国上市，用于治疗年龄 > 12 岁的囊性纤维化 (CF) 患者。我们报告了一项针对在我们的 CF 中心接受 ETI 治疗的年轻人的临床结果的真实世界研究，该研究是在该中心成立的头两年内进行的。方法：在我们的诊所内确定 12 至 17 岁的参与者，并由英国 CF 登记处补充人口统计数据。CFTR 调节剂启动前两年和启动后两年的综合结果数据是根据各种当地来源编制的，包括患者记录、给药日志和临床记录。结果：在开始接受 ETI 的 62 名患者中（32 名男性，平均年龄 13.3 岁），大多数 (76%) 是 F508del 突变纯合子。发生了 3 次停药：1 次是怀孕，2 次是与副作用有关。依从性很高（两年的覆盖天数比例均> 90%）。ETI 开始后，平均 FEV1% 显着增加（+11.7 单位；95% CI 7.4 - 15.6），并在整个两年治疗期间持续。基线肺功能与治疗后改善程度或下降率之间没有关联。所有可治疗基因型的改善都是相似的。治疗 4 个月时，BMI z 评分略有增加，24 个月时恢复到基线。静脉注射抗生素的需求显着减少。结论：在现实世界中，青少年使用 ETI 可以持续改善健康结果，这与开放试验扩展研究中所见的结果一致