Efficacy and safety of using Elexacaftor/Tezacaftor/Ivacaftor in the treatment of children with cystic fibrosis: real-world evidence from Brazil.

OBJECTIVE

Cystic fibrosis (CF) treatment has evolved significantly with the development of CFTR modulators, particularly elexacaftor/tezacaftor/ivacaftor (ETI). This study aimed to evaluate in a real-life context, the efficacy, safety and tolerability of ETI in children and adolescents with CF at a national reference center in Brazil.

METHODS

A cohort of 39 patients (mean age: 11.7 years) who had been using ETI for at least three months were evaluated. Anthropometric data, pulmonary function, sweat chloride concentration, pulmonary exacerbations, antibiotic use, and liver function were assessed over a follow-up period of up to 17 months.

RESULTS

Significant improvements were observed in weight Z-score at three months (p = 0.046) and six months (p = 0.018), as well as absolute weight gain (p < 0.001). Height showed absolute growth, but no significant changes in Z-scores. Sweat chloride concentration decreased by 52.8 mmol/L (p < 0.001). Pulmonary exacerbations and antibiotic use significantly declined (p < 0.001 for both). Despite limitations in spirometry data collection, FEV1 values showed a median increase of 6 percentage points. Oropharyngeal swab cultures for Pseudomonas aeruginosa positivity dropped from 43.6 % to 5.1 %. Safety assessments showed a transient rise in alkaline phosphatase (p = 0.011), but no significant hepatotoxicity. The most common adverse events were increased respiratory secretions (25.6 %) and abdominal pain (15.4 %). One temporary treatment suspension and one dose reduction occurred, but no patient required permanent discontinuation.

CONCLUSIONS

ETI demonstrated effectiveness in improving weight gain, reducing pulmonary exacerbations, and significantly lowering sweat chloride concentration. The treatment was well-tolerated, with a favorable safety profile. These findings align with existing literature, supporting ETI's role as a transformative therapy in pediatric CF management.