Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function
Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function
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DOI：
10.1016/j.jcf.2020.06.012
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发表时间：
2021-03-25
影响因子：
5.2
通讯作者：
Martin, Clemence
中科院分区：
文献类型：
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作者：
Burgel, Pierre-Regis;Durieu, Isabelle;Martin, Clemence
背景：3 期试验已证明 lumacaftor-ivacaftor (LUMA-IVA) 在 Phe508del CFTR 突变纯合子囊性纤维化 (CF) 患者中的安全性和有效性，且 1 s 内预测用力呼气量百分比 (ppFEV(1)) 在 40 至 90 之间。已向所有 ppFEV(1) 水平的患者授予营销授权。方法：评估该药物的安全性和有效性与 ppFEV(1) 患者相比，ppFEV(1) = 90 的患者在治疗第一年的 LUMA-IVA [40-90[。对现实世界研究期间收集的数据进行分析，其中包括 2016 年法国所有 47 个 CF 中心开始 LUMA-IVA 的所有年龄 >= 12 岁的患者。结果：827 名患者根据治疗开始时的 ppFEV(1) 分为 3 个亚组（ppFEV(1) = 90，n = 97）。 ppFEV(1) = 90 时治疗中断率较高 (17.5%)。在不间断治疗的患者中，ppFEV(1) 显着增加，出现在 ppFEV(1) [40-90[亚组 (+2.9%，P
Background: Phase 3 trials have demonstrated the safety and efficacy of lumacaftor-ivacaftor (LUMA-IVA) in patients with cystic fibrosis (CF) homozygous for the Phe508del CFTR mutation and percent predicted forced expiratory volume in 1 s (ppFEV(1)) between 40 and 90. Marketing authorizations have been granted for patients at all levels of ppFEV(1).Methods: To evaluate the safety and effectiveness of LUMA-IVA over the first year of treatment in patients with ppFEV(1) = 90 in comparison with those with ppFEV(1) [40-90[. Analysis of data collected during a real world study, which included all patients aged >= 12 years who started LUMA-IVA in 2016 across all 47 French CF centers.Results: 827 patients were classified into 3 subgroups according to ppFEV(1) at treatment initiation (ppFEV(1) = 90, n = 97). Treatment discontinuation rate was higher in ppFEV(1) = 90 (17.5%). In patients with uninterrupted treatment, significant increase in ppFEV(1) occurred in the ppFEV(1) [40-90[subgroup (+2.9%, P