SOLAR: a noninterventional study of outcomes over the long term of people with cystic fibrosis (CF) aged ≥6 years treated with elexacaftor/tezacaftor/ivacaftor using data from the French CF Registry.

INTRODUCTION: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has demonstrated efficacy in people with cystic fibrosis (pwCF) aged ≥6 years with ≥1 F508del allele in clinical trials. This registry-based cohort study aimed to describe real-world use and outcomes up to 36 months post-treatment initiation among pwCF aged ≥6 years treated with ELX/TEZ/IVA in France. METHODS: Outcomes included per cent predicted forced expiratory volume in 1 s (ppFEV1), intravenous-treated pulmonary exacerbations (PEx), hospitalisations, nutrition, lung transplantation, mortality and, among pwCF ≥18 years, quality of life (CFQ-R). All results are presented by population (≥12 years and 6-11 years). In the ≥12 years cohort, a subgroup analysis of pwCF with advanced lung disease (ALD), defined as ppFEV1 <40% at baseline, is presented. RESULTS: The study included 553 pwCF aged 6-11 years, with a mean±sd age of 8.8±1.9 years at baseline, ppFEV1 of 91.3±17.6% and mean follow-up of 8.9 months. In addition, 3313 pwCF aged ≥12 years were included, with a mean±sd age at baseline of 27.6±11.7 years, ppFEV1 of 65.4±24.8% and mean follow-up of 21.2 months. In the cohort aged 6-11 years, ppFEV1 improved by +9.6% (95% CI: 7.6-11.6) at 12 months and i.v.-treated PEx annual rate decreased from 0.15 (95% CI: 0.11-0.18) to 0.02 (0.00-0.04) after 0 to 12 months post-ELX/TEZ/IVA initiation. Among pwCF ≥12 years ppFEV1 improved post-ELX/TEZ/IVA initiation by +16.2% (95% CI: 15.6-16.8) at 24 months. i.v.-treated PEx annual rate decreased from 0.92 (95% CI: 0.88-0.95) during baseline to 0.14 (95% CI: 0.12-0.15) after 12 to 24 months post-ELX/TEZ/IVA initiation. Similar benefits to those in the ≥12 years population were observed in the ALD subgroup. CONCLUSION: This study confirms the longer term, transformational clinical benefit of ELX/TEZ/IVA in pwCF aged ≥6 years in a French population, demonstrating sustained improvements across multiple health outcomes, including in pwCF with ALD that were not part of the pivotal clinical trials.