Pulmonary therapy · 2025
Real-World Impact of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Italy: A Retrospective Study from a Cystic Fibrosis Center.
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Cipolli Marco, Thorat Teja, Pafundi Pia C, Roggeri Alessandro, Roggeri Daniela P, Rossi Carlotta, Pintani Emily, Meneghelli Ilaria, Lucca Francesca, Fratoni Anna, Vega-Hernandez Gabriela
Аннотация
INTRODUCTION: In Italy, elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in combination with ivacaftor was reimbursed starting July 2021 for use in people with cystic fibrosis (CF) aged ≥ 12 years homozygous for F508del mutation or heterozygous with at least one F508del allele and with a minimal function mutation. This study assessed the impact of ELX/TEZ/IVA on real-world outcomes among this population in Italy.
METHODS: This observational study used data from Verona CF Center from October 2018 to December 2022 to describe the impact of ELX/TEZ/IVA on individuals aged ≥ 12 years with ≥ 1 F508del allele on lung function and healthcare resource utilization (HCRU). Lung function (percent predicted forced expiratory volume in 1 s [ppFEV1]), hospitalizations, outpatient visits, pulmonary exacerbations (PEx), and prescribed medications were analyzed during baseline and follow-up periods (12 months pre- and post-ELX/TEZ/IVA initiation, respectively) and reported as change from baseline.
RESULTS: A total of 149 individuals were included (mean [standard deviation, SD] age: 32.6 [12.25] years). A mean improvement in ppFEV1 of +14.38 percentage points (95% confidence interval [CI] 12.65; 16.10) was observed during the follow-up period. An 87.2% reduction in the annualized PEx rate (rate ratio: 0.128 [95% CI 0.085; 0.187]) and an 85.4% reduction in the annualized hospitalization rate (rate ratio: 0.146 [95% CI 0.096; 0.213]) were observed during follow-up compared to baseline. Reductions in the proportion of individuals requiring prescription medications, including intravenous antibiotics, mucolytic agents, and bronchodilators, were observed.
CONCLUSION: Substantial improvements in lung function and reductions in HCRU were observed after treatment with ELX/TEZ/IVA in the Verona CF Center. Results contribute to the growing evidence of country-specific real-world data on the positive impact of ELX/TEZ/IVA.
METHODS: This observational study used data from Verona CF Center from October 2018 to December 2022 to describe the impact of ELX/TEZ/IVA on individuals aged ≥ 12 years with ≥ 1 F508del allele on lung function and healthcare resource utilization (HCRU). Lung function (percent predicted forced expiratory volume in 1 s [ppFEV1]), hospitalizations, outpatient visits, pulmonary exacerbations (PEx), and prescribed medications were analyzed during baseline and follow-up periods (12 months pre- and post-ELX/TEZ/IVA initiation, respectively) and reported as change from baseline.
RESULTS: A total of 149 individuals were included (mean [standard deviation, SD] age: 32.6 [12.25] years). A mean improvement in ppFEV1 of +14.38 percentage points (95% confidence interval [CI] 12.65; 16.10) was observed during the follow-up period. An 87.2% reduction in the annualized PEx rate (rate ratio: 0.128 [95% CI 0.085; 0.187]) and an 85.4% reduction in the annualized hospitalization rate (rate ratio: 0.146 [95% CI 0.096; 0.213]) were observed during follow-up compared to baseline. Reductions in the proportion of individuals requiring prescription medications, including intravenous antibiotics, mucolytic agents, and bronchodilators, were observed.
CONCLUSION: Substantial improvements in lung function and reductions in HCRU were observed after treatment with ELX/TEZ/IVA in the Verona CF Center. Results contribute to the growing evidence of country-specific real-world data on the positive impact of ELX/TEZ/IVA.