Background Ivacaftor (IVA) improves lung function and other extrapulmonary outcomes in people with cystic fibrosis (CF). However, the effect of initiating IVA at earlier versus later ages has not been studied. Methods We conducted an observational cohort study of people in the US CF Foundation Patient Registry aged ≥6 years with ≥1 CF transmembrane conductance regulator–gating mutation to compare the effects of initiating IVA at earlier ages on per cent predicted forced expiratory volume in 1 s (ppFEV1) and pulmonary exacerbation (PEx) outcomes. People with CF were grouped by age at IVA initiation (ages 6–10, 11–15, 16–20 and 21–25 years) to perform three analyses of younger versus older IVA initiation (6–10 vs 11–15, 11–15 vs 16–20 and 16–20 vs 21–25 years). For each analysis, baseline characteristics assessed over 1-year periods at the same age prior to IVA initiation were balanced by standardised mortality/morbidity ratio (SMR) weighting. For each analysis, outcomes were compared over a 5-year outcome assessment period when both groups were in the same age range and receiving IVA. Findings Baseline characteristics were well balanced between younger and older IVA initiator groups after SMR weighting. In the outcome assessment period, younger IVA initiators had significantly higher mean ppFEV1 than older initiators across all comparisons, and those initiating IVA between ages 6–10 and 11–15 years had significantly lower PEx rates. Interpretation Study findings showed the importance of early IVA initiation in people with CF. Data are available upon reasonable request. The data that support the findings of this study are available from the US Cystic Fibrosis Foundation Patient Registry at . The US Cystic Fibrosis Foundation Patient Registry collects and manages its own data and maintains processes for researchers to request summarised data. Restrictions may apply to the availability of these data, which were used under a license agreement for this study.

中文翻译：

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开始使用 ivacaftor 时年龄对囊性纤维化患者肺部结局的影响


背景 依伐卡托 （IVA） 可改善囊性纤维化 （CF） 患者的肺功能和其他肺外结局。然而，尚未研究在早期与晚期开始 IVA 的效果。方法 我们对美国 CF 基金会患者登记处年龄为 ≥6 岁且患有 ≥1 CF 跨膜传导调节因子门控突变的人进行了一项观察性队列研究，以比较在较早年龄开始 IVA 对预测 1 秒用力呼气容积百分比 （ppFEV1） 和肺部恶化 （PEx） 结果的影响。CF 患者按 IVA 开始时的年龄（6-10 岁、11-15 岁、16-20 岁和 21-25 岁）进行分组，对年轻与老年 IVA 开始进行三项分析（6-10 岁与 11-15 岁、11-15 岁与 16-20 岁和 16-20 岁与 21-25 岁）。对于每项分析，通过 标准化死亡率/发病率比 （SMR） 加权平衡 IVA 开始前同一年龄在 1 年内评估的基线特征。对于每项分析，当两组处于相同的年龄范围并接受 IVA 时，比较了 5 年结局评估期的结局。结果 SMR 加权后，年轻和老年 IVA 启动组之间的基线特征平衡良好。在结果评估期间，在所有比较中，年轻的 IVA 启动者的平均 ppFEV1 显著高于年长的启动者，而那些在 6-10 岁和 11-15 岁之间启动 IVA 的人的 PEx 率显着降低。解释研究结果显示，早期 IVA 开始对 CF 患者的重要性。支持本研究结果的数据可从美国囊性纤维化基金会患者登记处获得，网址为 。 美国囊性纤维化基金会患者登记处 （US Cystic Fibrosis Foundation Patient Registry） 收集和管理自己的数据，并维护研究人员请求汇总数据的流程。这些数据的可用性可能受到限制，这些数据是根据本研究的许可协议使用的。