Eitan Kerem, Annalisa Orenti, Arianna Adamoli, Elpis Hatziagorou, Lutz Naehrlich, Isabelle Sermet-Gaudelus, ECFS Patient Registry Steering Group

European Respiratory Journal 2024; DOI: 10.1183/13993003.01241-2023

Abstract

Background Prognosis and disease severity in cystic fibrosis (CF) is linked to declining lung function. To characterize lung function by the number of adults in countries with different level of Gross National Income (GNI), data from the European Cystic Fibrosis Society Patient Registry was utilized.

Methods Annual data including age, forced expiratory volume in 1 s (FEV₁), anthropometry, genotype, respiratory cultures and CF related diabetes (CFRD) were retrieved between 2011 and 2021. All countries were stratified into GNI per capita to reflect differences within Europe.

Findings A consistent improvement in FEV₁ percent of predicted (FEV₁pp) and survival was observed among the 47 621 people with CF (pwCF), including subjects with chronic Pseudomonas aeruginosa, CFRD, and/or undernutrition. Mean values of FEV₁pp changed from 85 to 94.2 for children and from 63.6 to 74.7 for adults. FEV₁pp further increased among those carrying the F508del mutation in 2021, when elexacaftortezacaftorivacaftor (ETI) was available. The number of adult pwCF increased from 13 312 in 2011 to 21 168 in 2021, showing a 60% increase. PwCF living in European countries with lower income (LIC) did not demonstrate a significant annual increase in FEV₁pp or in number of adults.

Interpretation This pan-European analysis demonstrates a consistent improvement in FEV₁pp, number of adult pwCF and survival over the last decade only in European higher and middle-income countries. An urgent action is needed in the LIC where such improvement was not observed. The notable improvement observed pwCF carrying the F508del mutation emphasizes the need to develop treatments for all CF mutations.