Real-Life in Cystic Fibrosis Pediatric Patients Treated With Kaftrio: A Descriptive Observational Study.

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.