Elexacaftor/tezacaftor/ivacaftor and inflammation in children and adolescents with cystic fibrosis: a retrospective dual-center cohort study.

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Tác giả: Silvia Campana, Daniela Dolce, Cristina Fevola, Angela Pepe, Novella Ravenni, Donatello Salvatore, Giovanni Taccetti, Vito Terlizzi

Ngôn ngữ: eng

Ký hiệu phân loại:

Thông tin xuất bản: England : Therapeutic advances in respiratory disease , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 50632

BACKGROUND: Cystic fibrosis (CF) is characterized by chronic neutrophilic inflammation in the airways. Elexacaftor/tezacaftor/ivacaftor (ETI) therapy has demonstrably improved clinical outcomes and quality of life in people with CF (pwCF), but its effects on systemic inflammatory parameters remain unclear. OBJECTIVE: To evaluate the impact of ETI on systemic inflammation in children and adolescents with CF. DESIGN: Retrospective, dual-center observational, propensity score-matching study of pediatric pwCF on ETI. METHODS: PwCF aged ⩽ 18 years treated with ETI at two Italian reference centers were included in this study. Data on immunoglobulins (Ig) (A, G, and M), γ-globulin, leukocyte levels, percent predicted forced expiratory volume in the first second (ppFEV1), sweat chloride (SC) concentration, and sputum cultures were collected at baseline, 12, and 24 months of treatment. Laboratory data of a control group (pwCF, not in ETI therapy, same demographic characteristics as the study group) were also collected. RESULTS: Sixty-six patients (30 males, median age: 12 years, F508del homozygous: 23) were included. Mean IgG levels (SD) significantly decreased ( CONCLUSION: ETI treatment improved respiratory outcomes and significantly reduced values of IgG, IgA, γ-globulin, and leukocytes, suggesting an effect on the systemic inflammatory response. Further research is warranted to elucidate the role of inflammatory parameters in monitoring response to therapy.
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