Evaluation of the Impact of Elexacaftor/Tezacaftor/Ivacaftor on Aerobic Capacity in Children With Cystic Fibrosis Aged 6–11 Years: Actual Observations and Clinical Perspectives
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Margarita Pérez-Ruiz [1] ; Mats te Bos [1] ; Patricia Fernández-García [4] ; Cristina de Manuel [2] ; Ana Morales-Tirado [5] ; Alejandro López-Neyra [4] ; Marta Ruiz de Valbuena [2] ; Margarita Rubio [3] ; Verónica Sanz-Santiago [4]
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[1] Universidad Politécnica de Madrid
Universidad Politécnica de Madrid
Madrid, España
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[2] Hospital Universitario La Paz
Hospital Universitario La Paz
Madrid, España
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[3] Universidad Europea de Madrid
Universidad Europea de Madrid
Madrid, España
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[4] Pediatric Pulmonology Department and Cystic Fibrosis Unit, Hospital Infantil Universitario Infantil Niño Jesús, Madrid, España
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[5] Pediatric Pulmonology Department and Cystic Fibrosis Unit, Hospital Infantil Universitario Ramón y Cajal, Madrid, España
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- Localización: Archivos de bronconeumología: Organo oficial de la Sociedad Española de Neumología y Cirugía Torácica SEPAR y la Asociación Latinoamericana de Tórax ( ALAT ), ISSN 0300-2896, Vol. 61, Nº. 11 (November 2025), 2025, págs. 662-668
- Idioma: inglés
- Texto completo no disponible (Saber más ...)
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Resumen
Background Cystic fibrosis causes exercise limitation due to impaired lung function and other complications, which in turn increases the chance of mortality. CFTR modulators, particularly the elexacaftor/tezacaftor/ivacaftor (ETI) combination, improve lung function in children older than 6 years in real-life studies.
Objective This study aimed to assess the impact of ETI on aerobic capacity in children with CF aged 6–11 years under real-life conditions and to evaluate whether prior CFTR modulator treatment affects these outcomes.
Methods A multicenter, prospective cohort study was conducted with pediatric CF patients. Participants underwent evaluations 6–8 months before ETI (T1), at the start of ETI (T2), and 6–8 months post-treatment (T3). Primary outcomes included cardiorespiratory fitness assessed via peak oxygen consumption (VO2peak) during a cardiopulmonary exercise test (CPET), and secondary outcomes encompassed lung function, quality of life, physical activity, and functional mobility.
Results Of the 28 patients (mean age 9.02 ± 1.59 years), 19 were ETI-naive, and 9 had prior CFTR modulator treatment. Significant improvements were observed in FEV1 (p < 0.001), and several functional mobility tests (30CST, Stair Climb Test, 10MWT). However, VO2peak showed no significant changes between T1 and T3. Quality of life scores improved notably in emotional, eating, and respiratory domains, and a slight improvement was noted in physical activity levels (p = 0.037).
Conclusions ETI treatment significantly enhances lung function and certain aspects of quality of life and physical fitness in pediatric CF patients. However, it does not significantly alter aerobic capacity (VO2peak) within the observed period.
