Expanding the Impact of New Cystic Fibrosis Therapies in Low- and Middle-Income Countries.

Background: Cystic fibrosis (CF) primarily affects Caucasian populations, with the highest prevalence in countries like Ireland, the UK, Australia, and Canada. Despite significant improvements in survival, pulmonary insufficiency remains the leading cause of death. Factors such as nutrition, chronic Pseudomonas aeruginosa (PsA) infection, genotype, pancreatic status, and cystic fibrosis-related diabetes affect pulmonary function across age groups.

Objective: This review examines disparities in CF care and outcomes between high-income countries (HICs) and low-income countries (LICs), focusing on the impact of CFTR modulators like Elexacaftor/Tezacaftor/Ivacaftor (ETI) and challenges in accessing care in LICs.

Methods: Data from the European CF Society Patient Registry and studies on CF outcomes across regions were reviewed to assess survival trends, pulmonary function, and infection rates among people with CF (pwCF). The effects of CFTR modulator therapies, particularly for F508del carriers, were also evaluated.

Results: In HICs, improvements in survival rates and pulmonary function have been noted, especially with the use of CFTR modulators like ETI. However, in LICs, challenges like limited access to therapies, delayed diagnosis, poor nutrition, and high PsA infection rates lead to poorer outcomes. In regions with fewer F508del carriers, access to care and medications is further limited, exacerbating disparities.

Conclusion: Although CF treatment advancements have improved outcomes in many pwCF, these benefits are not evenly distributed globally. Efforts to improve CF care in LICs, such as increasing awareness, ensuring access to therapies, and establishing specialized clinics, are essential to bridging this gap.