B107-16 Long-term Impact of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Patients With ≥1 F508del Mutation: Results From Four Years of Real-world Data
Observational cohort study shows improved lung function and quality of life in cystic fibrosis patients, suggesting sustained benefits from therapy.
Key Points
To evaluate the long-term impact of elexacaftor/tezacaftor/ivacaftor on various health parameters in cystic fibrosis patients with the F508del mutation.
Single-center, observational cohort study conducted over 48 months at University Medicine Essen.
Followed 106 cystic fibrosis patients with at least one F508del allele receiving ETI therapy.
Assessed lung function, BMI, exacerbations, sweat chloride, quality of life, and laboratory parameters.
Mean ppFEV1 increased by 0.5 L (95% CI 0.39-0.62; p<0.001).
BMI rose by 0.64 kg/m² (95% CI 0.55-4.27; p<0.0001), pulmonary exacerbations decreased by 28.9% (p<0.001).
HbA1c decreased by 2.31% (95% CI 2.3-4.62; p<0.001) and CFQ-R total scores improved by 46.7 points (95% CI 20.3-32.6; p<0.001).