Ann Am Thorac Soc. 2025 Sep 10. doi: 10.1513/AnnalsATS.202501-052OC. Online ahead of print.
ABSTRACT
Background Although advances in care have improved cystic fibrosis (CF) outcomes in higher-income countries (HICs), the situation remains alarming in lower-income countries (LICs). Methods People with CF (pwCF) enrolled in the European Cystic Fibrosis Society Patient Registry (ECFSPR) and carrying at least one F508del variant allele were evaluated in 2017 and in 2022 for predicted percent forced expiratory volume (ppFEV1), underweight status, and chronic Pseudomonas aeruginosa (Pa) infection, according to the gross national income (GNI) per capita divided into three terciles (low-income countries, LICs; middle-income countries, MICs; and high-income countries, HICs). Survival was evaluated in the periods 2013-2017 and 2018-2022. Generalized linear models and Cox regression models were fitted. Findings From the 31,723 pwCF reported in ECFSPR in 2022, 13.5% lived in LICs, 19.9% in MICs, and 66.6% in HICs. PwCF living in LICs had a significantly lower median survival age, reduced ppFEV1, and higher prevalence of Pa infection and underweight status compared with pwCF from MICs and HICs. Data modeling indicated that avoiding underweight status and Pa infection would increase survival by 42 years for pwCF living in LICs. Access to CFTR modulators would further increase their survival by 15 to 29 years depending on their nutrition and infection status, resulting in a survival up to 82 years in the best-case scenario. Interpretation Access to CFTR modulators equalizes survival between LICs and higher income countries within Europe. Optimizing care practices and social determinants of health remains crucial in LICs.
PMID:40929684 | DOI:10.1513/AnnalsATS.202501-052OC
