Impact of elexacaftor/tezacaftor/ivacaftor on lung function, nutritional status, pulmonary exacerbation frequency and sweat chloride in people with cystic fibrosis: real-world evidence from the German CF Registry
Research output: Contribution to journal › Research article › Contributed › peer-review
Contributors
- Department of Paediatrics
- University of Duisburg-Essen
- Catholic Hospital Bochum gGmbH
- University of Lübeck
- Mukoviszidose Institut gGmbH
- Innsbruck Medical University
- Hannover Medical School (MHH)
- University Hospital Frankfurt
- University Hospital Münster
- HMU Health and Medical University
- STAT-UP Statistical Consulting & Data Science GmbH
- Justus Liebig University Giessen
Abstract
BACKGROUND: Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) improves multiple clinical outcomes in people with cystic fibrosis (pwCF) with at least one F508del allele. This study evaluated the real-world impact of ETI on lung function, nutritional status, pulmonary exacerbation frequency, and sweat chloride concentrations in a large group of pwCF.
METHODS: This observational cohort study used data from the German CF Registry for pwCF who received ETI therapy and were followed up for a period of 12 months.
FINDINGS: The study included 2645 pwCF from 67 centres in Germany (mean age 28.0 ± 11.5 years). Over the first year after ETI was initiated, percent predicted forced expiratory volume in 1 s (ppFEV1) increased by 11.3% (95% confidence interval [CI] 10.8-11.8, p < 0.0001), body mass index (BMI) z-score increased by 0.3 (95% CI 0.3-0.4, p < 0.0001) in individuals aged 12 to <18 years and BMI in adults increased by 1.4 kg/m2 (95% CI 1.3-1.4, p < 0.0001), pulmonary exacerbations decreased by 75.9% (p < 0.0001) and mean sweat chloride concentration decreased by 50.9 mmol/L (95% CI -52.6, -49.3, p < 0.0001). Improvements in ppFEV1 over the first year of therapy were greater in pwCF who had not previously received cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy (12.6% [95% CI 11.9-13.4] vs. 9.7% [95% CI 9.0-10.5] in those with prior CFTR modulator treatment.
INTERPRETATION: These real-world data are consistent with the findings of randomised clinical trials, and support the use of ETI as a highly effective treatment option for pwCF who have at least one F508del allele.
FUNDING: None.
Details
Original language | English |
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Article number | 100690 |
Pages (from-to) | 100690 |
Journal | The Lancet Regional Health - Europe |
Volume | 32 |
Publication status | Published - Sept 2023 |
Peer-reviewed | Yes |
External IDs
PubMedCentral | PMC10405057 |
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Scopus | 85166298302 |