Department of Pulmonology, University Hospital of Nancy, 9 Rue Du Morvan, 54500 Vandoeuvre-lès-Nancy, France; Development, Adaptation and Disadvantage. Cardiorespiratory Regulations and Motor Control (EA 3450 DevAH), University of Lorraine, 9 Avenue de La Forêt de Haye, 54505 Vandoeuvre-lès-Nancy, France.
Development, Adaptation and Disadvantage. Cardiorespiratory Regulations and Motor Control (EA 3450 DevAH), University of Lorraine, 9 Avenue de La Forêt de Haye, 54505 Vandoeuvre-lès-Nancy, France; Lung Function Testing Lab, Children's University Hospital of Nancy, 9 Rue Du Morvan, 54500 Vandoeuvre-lès-Nancy, France.
Pulm Pharmacol Ther. 2023 Oct;82:102248. doi: 10.1016/j.pupt.2023.102248. Epub 2023 Aug 9.
The drug combination elexacaftor-tezacaftor-ivacaftor (ETI) proved highly effective in the improvement of the respiratory symptoms, the percentage of predicted forced expiratory volume in 1 s (FEV1), and to reduce rates of pulmonary exacerbations in people with cystic fibrosis (CF) with at least one F508del mutation. The objectives of the study were to evaluate the impact of ETI on the daily treatment burden due to patient decision and the evolution of lung function parameters at 6 months of treatment in real life.
A single-center observational study was realized including adult patients starting ETI therapy from March 10, 2020 to April 5, 2022. Clinical characteristics were collected at initiation (T0) and at 6 months (T6) of treatment. Outcome measures included names and number of chronic daily medications, respectively lung function parameters: FEV1, forced vital capacity (FVC), FEV1/FVC ratio, peak expiratory flow (PEF), forced expiratory flow at 25-75% of FVC (FEF25-75), β-angle and FEF50/PEF ratio.
Sixty-five patients were included with a mean age of 29.4 ± 8.5 years old, 48% of them F508del homozygous previously treated by lumacaftor-ivacaftor. At T6, the median number of daily medications decreased from 13 [2-24] to 9 [1-19] (p < 0.001). All the studied functional respiratory parameters were improved: FEV1 +18%, FVC +14%, FEF25-75% + 18% (all p < 0.001), as well the airflow obstruction: FEV1/FVC +6%, FEF50/PEF by 0.1 ± 0.1 and β-angle by 10° ± 13° (all p ≤ 0.007).
ETI therapy can reduce the daily treatment burden in real-life at 6 months of treatment, increase a large number of lung function parameters and improve airflow obstruction.
依伐卡托与泰比卡托和艾地卡托的复方制剂（ETI）在改善囊性纤维化（CF）患者的呼吸道症状、第 1 秒用力呼气量（FEV1）预计百分比以及降低至少携带一个 F508del 突变的 CF 患者的肺部恶化率方面表现出了高度有效性。本研究的目的是评估 ETI 对患者决策导致的日常治疗负担以及真实生活中治疗 6 个月时肺功能参数演变的影响。
开展了一项单中心观察性研究，纳入了自 2020 年 3 月 10 日至 2022 年 4 月 5 日期间开始 ETI 治疗的成年患者。在治疗起始时（T0）和 6 个月时（T6）收集临床特征。主要结局指标包括慢性每日药物名称和数量，以及肺功能参数：FEV1、用力肺活量（FVC）、FEV1/FVC 比值、呼气峰流速（PEF）、用力呼出 25%至 75%肺活量时的流量（FEF25-75）、β-角度和 FEF50/PEF 比值。
共纳入 65 例患者，平均年龄 29.4±8.5 岁，48%为 F508del 纯合子，之前曾接受过 lumacaftor-ivacaftor 治疗。在 T6 时，每日药物数量中位数从 13[2-24]降至 9[1-19]（p<0.001）。所有研究的功能性呼吸参数均得到改善：FEV1 增加 18%，FVC 增加 14%，FEF25-75%增加 18%（均 p<0.001），气流阻塞也得到改善：FEV1/FVC 增加 6%，FEF50/PEF 增加 0.1±0.1，β-角度增加 10°±13°（均 p≤0.007）。
ETI 治疗在 6 个月的治疗后可以减轻真实生活中的日常治疗负担，增加大量的肺功能参数，并改善气流阻塞。
