Health status and therapy decisions in children with cystic fibrosis in Germany and Austria who switched treatment from LUM/IVA to ELX/TEZ/IVA: Interim analysis from the noninterventional DECIDE study 809–127

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Introduction: DECIDE is an ongoing, 5-year, noninterventional, single-arm, chart review study following the CF treatment journey of children aged 2–11 years (y) in Germany and Austria with CF who are homozygous for F508del-CFTR and received lumacaftor/ivacaftor (LUM/IVA) therapy. Here, we describe the interim results for health status and healthcare decision-making among young children who switched to elexacaftor/tezacaftor/IVA (ELX/TEZ/IVA). Methods: The interim analysis (IA; data cutoff April 2024) included children who switched to ELX/TEZ/IVA and had ³12 months follow-up after ELX/TEZ/IVA initiation. Descriptive analyses compared the 12 months post-ELX/TEZ/IVA initiation to the 12 months before initiating ELX/TEZ/IVA (baseline). Endpoints included reasons for changes in CFTR modulator therapy and other CF-related treatments, healthcare resource utilization, and clinical outcomes. Results: Sixty-three participants were included in this IA. Most participants were female (69.8%). At ELX/TEZ/IVA initiation, the mean age (standard deviation [SD]) was 11.5 y (1.7), mean BMI for age z-score (SD) was –0.31 (0.96), and mean ppFEV1 (SD) was 87.9 (15.3). Compared to the baseline period, ELX/TEZ/IVA treatment led to a mean ppFEV1 improvement of 9.9 (SD: 10.2). In 79.4% (n=50/63), “physician’s preference” was included as the main reason for switching to ELX/TEZ/IVA; 82.5% (n=52/63) participants switched directly from LUM/IVA. None of the participants discontinued ELX/TEZ/IVA or switched to another CFTR modulator within 12 months after initiation. When comparing baseline to follow-up, reductions in the number of pulmonary exacerbations (27 vs 17) and CF-related hospitalizations (23 vs 7) were observed. Conclusions: These findings provide real-world insights into the decision making of physicians in clinical settings in Germany and Austria highlighting the positive impact of ELX/TEZ/IVA on children with CF, including those with well-preserved ppFEV1 at baseline. Publication History Article published online: 28 February 2025 © 2025. Thieme. All rights reserved. Georg Thieme Verlag KG Oswald-Hesse-Straße 50, 70469 Stuttgart, Germany

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