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Real-world outcomes of elexacaftor/tezacaftor/ivacaftor in a cohort of children and adolescents with cystic fibrosis in Brazil

Renata Wrobel Folescu Cohen Paloma Fernandes Coelho Leonardo Yllel Rocha Julia Vieira da Cunha Moreira N. Barboza Katty Anne Carvalho Marins Viviane Mauro Correa Meyer Patrícia Fernandes Barreto Machado Costa Tania Wrobel Folescu

Oct 2026 · Respiratory Research & Clinical Practice · 0 citations · 10 references

Abstract

Objective: To describe real-world clinical outcomes associated with elexacaftor/ tezacaftor/ivacaftor (ETI) in people with cystic fibrosis (pwCF) followed at a pediatric referral center in Brazil. Methods: This was a retrospective analysis nested within a prospective cohort study conducted between February of 2023 and February of 2026. We included individuals who had confirmed cystic fibrosis; who had initiated treatment with ETI; and who were followed for at least six months. Outcomes assessed included BMI-for-age z-score (BAZ), percent predicted FEV1 , sweat chloride concentration (SCC), microbiological data, and adverse events. Data were collected from baseline prior to ETI initiation and from follow-up visits at 6-12 months after initiation of ETI treatment. Results: A total of 108 pwCF (males, 50.9%; mean age, 10.2 ± 3.7 years) were included. Genotypes were F/F (46.3%), F/non-F (47.2%), and non-F/non-F (6.5%). ETI was associated with significant improvements in BAZ and FEV1 percentage points (ppFEV1 ), with a mean individual increase of 16.9 percentage points in lung function. Median SCC decreased from 95 mmol/L to 41 mmol/L, with most pwCF falling below the diagnostic threshold. No significant correlation was observed between SCC reduction and changes in BAZ or ppFEV1 . The proportion of individuals with at least one positive respiratory culture for Pseudomonas aeruginosa declined from 54.63% before ETI to 23.58% within 6-12 months of treatment (p < 0.0001). Adverse events were mild: 5.6% developed rash requiring temporary interruption; two had elevated creatine phosphokinase levels; and no clinically significant liver enzyme elevation or permanent discontinuation occurred. Conclusions: ETI was effective and well tolerated, leading to meaningful improvements in nutritional status, lung function, and cystic fibrosis transmembrane conductance regulator restoration, as well as to reduced P. aeruginosa detection, in this real-world pediatric cohort

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