Risk factors for forced expiratory volume in 1 s decline in European patients with cystic fibrosis: data from the European Cystic Fibrosis Society Patient Registry
Elpis Hatziagorou, Steffen Fieuws, Annalisa Orenti, Lutz Naehrlich, Uroš Krivec, Meir Mei‐Zahav, Andreas Jung, K. De Boeck, on behalf of the ECFSPR Collaborative Group, ECFSPR Collaborative Group, Pfleger Andreas, Géraldine Daneau, L. Elise, Petrova Guergana, Pavel Dřevı́nek, Milan Maçek, Hanne Vebert Olesen, Pierre‐Régis Burgel, Lydie Lemonnier-Videau, Andrea Párniczky, G. Fletcher, Rita Padoan, Anna Zolin, Elīna Aleksejeva, Kęstutis Malakauskas, Vincent Gulmans, Stojka Fustik, Ivana Arnaudova Danevskai, O. Turcu, L. Pereira, Liviu Pop, E. Kondratyeva, Milan Rodić, H. Kayserová, María Dolores Pastor‐Vivero, Isabelle de Monestrol, Anders Lindblad, Deni̇z Doğru, Halyna Makukh, Siobhán B. Carr, Rebecca Cosgriff
Abstract
Aim To examine the trajectory of forced expiratory volume in 1 s (FEV 1 ) using data from the European Cystic Fibrosis Society patient registry (ECFPR) collected from 2008 to 2016, i.e. the era before highly effective modulator therapy (HEMT). We evaluated risk factors for FEV 1 decline. Methods The study population included patients with a confirmed diagnosis of cystic fibrosis recorded in the ECFPR (2008–2016). The evolution of FEV 1 % predicted (%FEV 1 ) with age, and the yearly change in %FEV 1 were evaluated. Risk factors considered were cystic fibrosis transmembrane conductance regulator ( CFTR ) mutation class, gender, age at diagnosis, neonatal screening, meconium ileus, sweat chloride concentration at diagnosis and country's income level. Results We used 199 604 FEV 1 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 1 , but a higher yearly %FEV 1 loss between the ages of 15 and 25 years. We showed stabilisation and even improvement in %FEV 1 over age in adults with a class III CFTR mutation, but a steady decline in patients homozygous for F508del or with both mutations of classes I/II. A faster decline in %FEV 1 was found in patients from low-income countries compared to a similar %FEV 1 evolution in patients from middle- and high-income countries. Conclusions These longitudinal FEV 1 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.