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. 2024 Mar 7;63(3):2301241.
doi: 10.1183/13993003.01241-2023. Print 2024 Mar.

Cystic fibrosis in Europe: improved lung function and longevity - reasons for cautious optimism, but challenges remain

Eitan Kerem  1 , Annalisa Orenti  2 , Arianna Adamoli  2 , Elpis Hatziagorou  3   4 , Lutz Naehrlich  5   4 , Isabelle Sermet-Gaudelus  6   7   8   4 ; and the ECFS Patient Registry Steering Group
Affiliations

Cystic fibrosis in Europe: improved lung function and longevity - reasons for cautious optimism, but challenges remain

Eitan Kerem et al. Eur Respir J. .

Abstract

Background: Prognosis and disease severity in cystic fibrosis (CF) are linked to declining lung function. To characterise lung function by the number of adults in countries with different levels of Gross National Income (GNI), data from the European Cystic Fibrosis Society Patient Registry were utilised.

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.

Results: A consistent improvement in FEV1 % pred and survival was observed among the 47 621 people with CF (pwCF), including subjects with chronic Pseudomonas aeruginosa infection, CFRD and/or undernutrition. Mean values of FEV1 % pred changed from 85% to 94.2% for children and from 63.6% to 74.7% for adults. FEV1 % pred further increased among those carrying the F508del mutation in 2021, when elexacaftor/tezacaftor/ivacaftor 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 lower income countries did not demonstrate a significant annual increase in FEV1 % pred or in the number of adults.

Conclusion: This pan-European analysis demonstrates a consistent improvement in FEV1 % pred, number of adult pwCF and survival over the last decade only in European higher and middle income countries. Urgent action is needed in the lower income countries where such improvement was not observed. The notable improvement observed in pwCF carrying the F508del mutation emphasises the need to develop treatments for all CF mutations.

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Conflict of interest statement

Conflict of interest: L. Naehrlich reports grants from the German Center for Lung Research, Vertex Pharmaceuticals and Mukoviszidose Institute, participation on a trial steering committee for CF STORM, leadership roles as medical lead of the German CF Registry and pharmacovigilance study manager of the ECFSPR, and medical writing from Articulate Science, outside the submitted work. I. Sermet-Gaudelus reports grants, consulting fees and lecture honoraria from Vertex Pharmaceuticals, and a leadership role as medical lead of the French Pediatric CF Network, outside the submitted work. The remaining authors have no potential conflicts of interest to disclose.

Figures

FIGURE 1
FIGURE 1
Forced expiratory volume in 1 s (FEV1) percentage predicted values in European people with cystic fibrosis according to age and year.
FIGURE 2
FIGURE 2
Forced expiratory volume in 1 s (FEV1) percentage predicted values in European people with cystic fibrosis (pwCF) according to age and year: a) in pwCF with chronic Pseudomonas aeruginosa infection, b) in pwCF with CF-related diabetes (CFRD) and c) in underweight pwCF.
FIGURE 3
FIGURE 3
Forced expiratory volume in 1 s (FEV1) percentage predicted values in European people with cystic fibrosis according to age, year and genotype: a) F508del mutation on both alleles (F508del/F508del), b) F508del mutation on one allele and a minimal function mutation on the other allele (F508del/MF), c) two minimal function mutations (excluding F508del) (MF/MF) and d) at least one residual function mutation (RF/other).
FIGURE 4
FIGURE 4
Median forced expiratory volume in 1 s (FEV1) percentage predicted (graph lines) and number of people with cystic fibrosis (bar charts) by age group and year in Europe: a) lower income countries (LICs), b) middle income countries (MICs) and c) higher income countries (HICs). (The three groups are different from the World Bank Gross National Income categories of low, middle and high income countries.)
FIGURE 5
FIGURE 5
Survival probability in European people with cystic fibrosis according to age and year of follow-up.
FIGURE 6
FIGURE 6
Forced expiratory volume in 1 s (FEV1) percentage predicted values according to age and year in Europe: a) lower income countries (LICs), b) middle income countries (MICs) and c) higher income countries (HICs). (The three groups are different from the World Bank Gross National Income categories of low, middle and high income countries.)
FIGURE 7
FIGURE 7
Forced expiratory volume in 1 s (FEV1) percentage predicted values according to Gross National Income (GNI) (as a continuous variable) and year.
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