Hubert Dominique, Marguet Christophe, Benichou Jacques, DeSouza Cynthia, Payen-Champenois Catherine, Kinnman Nils, Chandarana Keval, Munck Anne, Fajac Isabelle
Respiratory Medicine and National Cystic Fibrosis Reference Center, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France.
Pediatric Respiratory Diseases and Cystic Fibrosis Center, Rouen University Hospital, INSERM CIC1404, EA 2656 UNI ROUEN, Normandy University, Rouen, France.
Pulm Ther. 2021 Dec;7(2):455-468. doi: 10.1007/s41030-021-00158-5. Epub 2021 Jun 8.
Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from a real-world study (BRIO) to assess the effectiveness of ivacaftor in people with cystic fibrosis (pwCF) in France.
BRIO was an observational study conducted at 35 centers in France. Both pwCF initiating ivacaftor treatment and those already taking ivacaftor were included and prospectively followed for 24 months. The primary objective was to evaluate the effect of ivacaftor on percent predicted forced expiratory volume in 1 s (ppFEV); secondary objectives were evaluating the effect of ivacaftor on clinical effectiveness, healthcare resource utilization (HCRU), and safety.
A total of 129 pwCF were enrolled; 58.9% were aged < 18 years; 64.3% had a G551D-CFTR allele. Mean age at ivacaftor initiation was 19.1 years (range, 2-64 years); ppFEV increased by a least squares mean of 8.49 percentage points in the first 6 months and was sustained through 36 months of ivacaftor use. Growth metrics increased during the first 12 months post-ivacaftor and remained stable. The rate of pulmonary exacerbations (PEx) decreased during the 12 months post-ivacaftor compared with the 12 months pre-ivacaftor; estimated rate ratios (95% CI) were 0.57 (0.43-0.75) for PEx events and 0.25 (0.13-0.48) for PEx requiring hospitalization. No new safety concerns were identified; no deaths occurred.
The results from this real-world study of ivacaftor usage in France were consistent with prior clinical trial outcomes, confirming the clinical effectiveness of ivacaftor, as well as an associated reduction in HCRU.
依伐卡托是一种囊性纤维化跨膜传导调节因子（CFTR）增强剂，已在3期试验中显示出临床益处。我们报告了一项真实世界研究（BRIO）的结果，以评估依伐卡托在法国囊性纤维化患者（pwCF）中的有效性。
BRIO是在法国35个中心进行的一项观察性研究。纳入了开始使用依伐卡托治疗的pwCF患者以及已经在服用依伐卡托的患者，并对其进行了为期24个月的前瞻性随访。主要目标是评估依伐卡托对1秒用力呼气容积预测值百分比（ppFEV）的影响；次要目标是评估依伐卡托对临床疗效、医疗资源利用（HCRU）和安全性的影响。
共纳入129例pwCF患者；58.9%的患者年龄<18岁；64.3%的患者携带G551D-CFTR等位基因。开始使用依伐卡托时的平均年龄为19.1岁（范围为2至64岁）；在最初6个月内，ppFEV的最小二乘均值增加了8.49个百分点，并在使用依伐卡托的36个月内持续保持。生长指标在依伐卡托治疗后的前12个月内增加，并保持稳定。与依伐卡托治疗前的12个月相比，依伐卡托治疗后的12个月内肺部恶化（PEx）发生率降低；PEx事件的估计发生率比值（95%CI）为0.57（0.43-0.75），因PEx需要住院治疗的发生率比值为0.
