Hatziagorou Elpis, Fieuws Steffen, Orenti Annalisa, Naehrlich Lutz, Krivec Uros, Mei-Zahav Meir, Jung Andreas, De Boeck Kris
Cystic Fibrosis Unit, Hippokration General Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
KU Leuven - University of Leuven, Interuniversity Institute for Biostatistics and Statistical Bioinformatics, Leuven, Belgium.
ERJ Open Res. 2023 May 22;9(3). doi: 10.1183/23120541.00449-2022. eCollection 2023 May.
To examine the trajectory of forced expiratory volume in 1 s (FEV) using data from the European Cystic Fibrosis Society patient registry (ECFPR) collected from 2008 to 2016, the era before highly effective modulator therapy (HEMT). We evaluated risk factors for FEV decline.
The study population included patients with a confirmed diagnosis of cystic fibrosis recorded in the ECFPR (2008-2016). The evolution of FEV % predicted (%FEV) with age, and the yearly change in %FEV were evaluated. Risk factors considered were cystic fibrosis transmembrane conductance regulator () mutation class, gender, age at diagnosis, neonatal screening, meconium ileus, sweat chloride concentration at diagnosis and country's income level.
We used 199 604 FEV recordings from 38 734 patients. The fastest decline was seen during puberty and in patients diagnosed before the age of 10 years. Males had a higher %FEV, but a higher yearly %FEV loss between the ages of 15 and 25 years. We showed stabilisation and even improvement in %FEV over age in adults with a class III mutation, but a steady decline in patients homozygous for F508del or with both mutations of classes I/II. A faster decline in %FEV was found in patients from low-income countries compared to a similar %FEV evolution in patients from middle- and high-income countries.
These longitudinal FEV data reflect the reality of cystic fibrosis across Europe in the era pre-HEMT, and can serve as baseline for comparison with the post-HEMT era. The similar evolution in middle- and high-income countries underlines opportunities for low-income countries.
利用2008年至2016年欧洲囊性纤维化协会患者登记处（ECFPR）收集的数据，即高效调节剂疗法（HEMT）之前的时期，研究1秒用力呼气量（FEV）的变化轨迹。我们评估了FEV下降的风险因素。
研究人群包括ECFPR（2008 - 2016年）中确诊为囊性纤维化的患者。评估了预测FEV百分比（%FEV）随年龄的变化以及%FEV的年变化。考虑的风险因素有囊性纤维化跨膜传导调节因子（）突变类型、性别、诊断时年龄、新生儿筛查、胎粪性肠梗阻、诊断时汗液氯化物浓度和国家收入水平。
我们使用了来自38734名患者的199604份FEV记录。青春期以及10岁前诊断的患者FEV下降最快。男性的%FEV较高，但在15至25岁之间每年的%FEV损失较高。我们发现，具有III类突变的成年患者的%FEV随年龄增长而稳定甚至改善，但F508del纯合子患者或同时具有I/II类两种突变的患者则持续下降。与中高收入国家患者相似的%FEV变化相比，低收入国家患者的%FEV下降更快。
这些纵向FEV数据反映了HEMT时代之前欧洲囊性纤维化的实际情况，可作为与HEMT时代之后进行比较的基线。中高收入国家的相似变化突出了低收入国家的机会。
