Observational studyClinical pediatrics2025
Real-Life in Cystic Fibrosis Pediatric Patients Treated With Kaftrio: A Descriptive Observational Study.
Observational study in Clinical pediatrics, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In 2020, Kaftrio, a combination of Elexacaftor, Tezacaftor, and Ivacaftor, gained approval for treating cystic fibrosis (CF) in patients from the age of 12 years. This study aims to analyze 1 year of treatment with Kaftrio in pediatric patients, comparing their clinical characteristics with pre-treatment data. This is an observational, descriptive, and longitudinal study in patients with CF older than 12 years with at least 1 F508del mutation treated with Kaftrio for 1 year. Forced expiratory volume in 1 second (FEV1) z-score increased by +1.1 (95% confidence interval [CI] = 0.55 to 1.64), forced vital capacity (FVC) by +0.56 (95% CI = 0.10 to 1.04), and maximal mid-expiratory flow (MMEF) 25/75 improved by +1.53 (95% CI = 0.59 to 2.47). In addition, a reduction of 25.50 points (95% CI = -37.95 to -13.06) in sweat chloride levels was observed. Body mass index (BMI)-for-age z-score (WHO 2006/2007) increased +0.39 (95% CI = 0.02 to 0.77). A transient increase in cough and secretions was noted in 61.53% after starting treatment. Kaftrio improves lung function and BMI and also reduces respiratory exacerbations and sweat chloride levels.
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