Three-year outcomes of elexacaftor/tezacaftor/ivacaftor in adults with cystic fibrosis: A real-world study.

INTRODUCTION

CFTR modulators have significantly changed the prognosis of cystic fibrosis (CF), although data on their long-term clinical and microbiological impact in adults in real-world practice remain limited.

OBJECTIVE

To analyze the clinical, functional, and microbiological outcomes at 36 months in adults with CF treated with elexacaftor/tezacaftor/ivacaftor (ETI).

METHODS

A longitudinal observational study was conducted in adults with CF. Lung function, number of exacerbations, weight, and sputum cultures were assessed at baseline and at 12, 24, and 36 months after initiation of ETI.

RESULTS

A total of 55 patients were included (43.6% female; mean age 32±13 years). FEV1 improved at 12 months and remained stable at 36 months. Exacerbations decreased, and weight increased progressively. The proportion of patients with potentially pathogenic microorganisms decreased from 93.1% at baseline to 36.2% at 36 months. Patients without microbiological isolation showed better lung function and fewer exacerbations.

CONCLUSIONS

In real-world clinical practice, ETI is associated with improvements in lung function, weight, and exacerbations, as well as a reduction in the isolation of potentially pathogenic microorganisms in sputum at 36 months.