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首页> 外文期刊>Pulmonary therapy. >Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience
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Real-World Outcomes Among Patients with Cystic Fibrosis Treated with Ivacaftor: 2012–2016 Experience

机译：患有IVACAFTOR治疗囊性纤维化患者的现实世界结果：2012-2016经验

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IntroductionIn this long-term, postapproval, observational study, data from the US Cystic Fibrosis Foundation Patient Registry and the UK Cystic Fibrosis Registry were used to evaluate the impact of ivacaftor treatment on cystic fibrosis (CF) by comparing outcomes in ivacaftor-treated patients with those in matched untreated comparator patients. Registry data from up to 5?years of ivacaftor availability in the US and up to 4?years of availability in the UK were evaluated.MethodsStarting in the first year of ivacaftor availability, ivacaftor-treated patients in each registry were matched 1:5 to comparator patients who never received ivacaftor. Clinical endpoints were evaluated in annual cross-sectional safety analyses. The key endpoints were death, organ transplants, pulmonary exacerbation, and hospitalization. Relative risks and 95% CIs were calculated to compare the ivacaftor and comparator cohorts in each registry.ResultsHere, we report the complete and final results of the annual cross-sectional safety analyses across the duration of the study, with up to 5?years of follow-up. Data show a pattern of lower risk of death, transplant, pulmonary exacerbation, and hospitalization among ivacaftor-treated patients in both registries.ConclusionsIvacaftor-treated patients had consistently favorable clinical outcomes relative to untreated comparators, and no new safety concerns were identified. While general limitations of observational research apply, these findings support disease modification by CF transmembrane conductance regulator (CFTR) modulator therapy with ivacaftor. Future research of novel CFTR modulators will need to explore alternative methods for comparator selection for evaluation of clinical data given the evolving landscape of CF treatment.
机译：介绍这一长期，未满，观察研究，来自美国囊性纤维化基础患者注册表和英​​国囊性纤维化登记处的数据通过比较IVACAFTOR治疗患者的结果来评估IVACAFTOR治疗对囊性纤维化（CF）的影响那些匹配的未经治疗的比较患者。从最多5年的Ivacaftor可用性的注册管理资源数据在美国和最多4年内获得了4年的可用性。在每次登记处的Ivacafactor治疗的患者中，Ivacafactor治疗的患者的一年中，达到了1：5比较患者从未接受过Ivacaftor。在年横截面安全分析中评估临床终点。关键终点是死亡，器官移植，肺癌和住院。计算了相对风险和95％的CIS，以比较每个注册表中的IVACAFTOR和比较队员.Resultssss，我们报告了研究期间的年度横断面安全分析的完整和最终结果，最多5年？多年跟进。数据显示了患有Ivacafactor治疗的患者中的死亡，移植，肺癌的病症和住院风险较低的模式。结合同期的患者相对于未处理的比较器持续有利的临床结果，并且没有确定新的安全问题。虽然观察研究的一般局限性适用，但这些发现支持CF跨膜电导调节剂（CFTR）调节剂治疗疾病修饰。新型CFTR调制器的未来研究将需要探索用于评估临床资料的比较器选择的替代方法，因为CF治疗的不断发展景观。

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