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

Area of Science:

• Medical research
• Pulmonology
• Genetics

Background:

• Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs.
• Kaftrio, a combination therapy of Elexacaftor, Tezacaftor, and Ivacaftor, was approved in 2020 for CF patients aged 12 and older.
• Understanding Kaftrio's long-term effects in pediatric populations is crucial.

Purpose of the Study:

• To evaluate the clinical outcomes of one year of Kaftrio treatment in pediatric CF patients.
• To compare clinical characteristics before and after treatment initiation.
• To assess the impact of Kaftrio on lung function, sweat chloride levels, and BMI in adolescents.

Main Methods:

• Observational, descriptive, and longitudinal study design.
• Inclusion of pediatric CF patients (over 12 years) with at least one F508del mutation.
• Data collection included lung function tests (FEV1, FVC, MMEF), sweat chloride levels, and BMI z-scores pre- and post-treatment.

Main Results:

• Significant improvements observed in lung function: FEV1 z-score (+1.1), FVC (+0.56), and MMEF 25/75 (+1.53).
• Marked reduction in sweat chloride levels by 25.50 points.
• Increase in BMI-for-age z-score (+0.39) and a transient increase in cough/secretions in 61.53% of patients.

Conclusions:

• Kaftrio demonstrates significant efficacy in improving lung function and nutritional status (BMI) in pediatric CF patients.
• The therapy effectively reduces sweat chloride levels, a key biomarker for CF.
• While generally well-tolerated, transient respiratory symptoms may occur upon initiation.