Journal Article Ahead of print
PubMed: 42235564 DOI: 10.1016/S2213-2600(26)00149-9
Clinical characteristics and outcomes in the adult cystic fibrosis population in Europe from 2014 to 2024: analysis of the European Cystic Fibrosis Society Patient Registry.
Lancet Respir Med
Authors: Annalisa Orenti, Kris De Boeck, Eitan Kerem, Pierre-Régis Burgel, Anna Zolin, Egil Bakkeheim, Lutz Naehrlich, Elpis Hatziagorou
Affiliations
- Department of Clinical Sciences and Community Health, Dipartimento di Eccellenza 2023-27, Laboratory of Medical Statistics, Biometry and Epidemiology G A Maccacaro, University of Milan, Milan, Italy; IRCCS Ospedale Galeazzi-Sant'Ambrogio, Milano, Italy.
- Paediatric Pulmonology Department, KU Leuven, Leuven, Belgium.
- Department of Paediatrics and Cystic Fibrosis Centre, Hebrew University Medical School, Hadassah Medical Centre, Jerusalem, Israel.
- Department of Respiratory Medicine and National Reference Centre for Cystic Fibrosis, Assistance Publique-Hôpitaux de Paris, Paris, France; Institut Cochin, Université Paris Cité and Inserm U1016, Paris, France.
- Department of Clinical Sciences and Community Health, Dipartimento di Eccellenza 2023-27, Laboratory of Medical Statistics, Biometry and Epidemiology G A Maccacaro, University of Milan, Milan, Italy.
- ERN-lung Cystic Fibrosis Core Network, Frankfurt, Germany; National Resource Centre for Cystic Fibrosis, Oslo University Hospital, Oslo, Norway.
- Department of Paediatrics, Justus-Liebig University, Giessen, Germany.
- Paediatric Pulmonology and Cystic Fibrosis Unit, Hippokration General Hospital, Aristotle University of Thessaloniki, Thessaloniki, Greece.
Abstract
BACKGROUND
Since 2018, important advancements in the medical care of people with cystic fibrosis, particularly the introduction and widespread use of highly effective cystic fibrosis transmembrane conductance regulator modulators, have contributed to the adult cystic fibrosis population growing substantially and has led to an increased need for tailored health-care approaches. Our study aimed to analyse the extent to which the clinical characteristics and treatment outcomes of adults with cystic fibrosis have evolved from 2014 to 2024.
METHODS
The European Cystic Fibrosis Society Patient Registry (ECFSPR), collects annual data for more than 55 000 people with cystic fibrosis. Longitudinal data from 20 countries in Europe with high patient coverage (>85%) from 2014 to 2024 were analysed, representing 80% of the whole ECFSPR cohort. Differences in annual cross-sectional estimates were assessed using regression models.
FINDINGS
Between 2014 and 2024, the number of adults with cystic fibrosis increased by 45·0%, from 50·9% to 60·5% of the total cystic fibrosis population. The number of adults older than 30 years nearly doubled. Among adults with cystic fibrosis who had not received a transplant, mean percent predicted FEV 1 improved from 66·1% to 78·8% (p<0·0001), with most of the gain occurring after 2020. Chronic Pseudomonas aeruginosa infection declined significantly (p<0·0001), whereas mean BMI increased significantly (p<0·0001), halving the proportion of individuals who are underweight. Age-related complications, such as malignancy, increased, whereas cystic fibrosis-specific complications and insulin-treated diabetes declined. The largest improvements were observed in individuals with at least one variant responsive to elexacaftor-tezacaftor-ivacaftor (ETI). The uptake of ETI increased from 2% in 2019 to 71% in 2024, associated with improvements in health indicators.
INTERPRETATION
From 2014 to 2024, the adult cystic fibrosis population in Europe expanded substantially due to marked improvements in treatment, particularly following the availability of ETI triple therapy from 2018-19 onwards. The growing number of people with CF surviving to adulthood is consistent with substantial effects from improved care and cystic fibrosis transmembrane conductance regulator modulators, such as ETI, and highlight evolving care needs of people with cystic fibrosis.
FUNDING
None.
MeSH
Registries, Fibrosis, Cystic Fibrosis Transmembrane Conductance Regulator, Cystic Fibrosis, Therapeutics