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Introduction <p>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&#xa0;years homozygous for <i>F508del</i> mutation or heterozygous with at least one <i>F508del</i> 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.</p> Methods <p>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&#xa0;years with ≥ 1 <i>F508del</i> allele on lung function and healthcare resource utilization (HCRU). Lung function (percent predicted forced expiratory volume in 1&#xa0;s [ppFEV<sub>1</sub>]), hospitalizations, outpatient visits, pulmonary exacerbations (PEx), and prescribed medications were analyzed during baseline and follow-up periods (12&#xa0;months pre- and post-ELX/TEZ/IVA initiation, respectively) and reported as change from baseline.</p> Results <p>A total of 149 individuals were included (mean [standard deviation, SD] age: 32.6 [12.25] years). A mean improvement in ppFEV<sub>1</sub> 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.</p> Conclusion <p>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.</p>

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Real-World Impact of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Italy: A Retrospective Study from a Cystic Fibrosis Center

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  • 出版模式：

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  • 发表日期：

    2025年12月4日

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  • Marco Cipolli,
  • Teja Thorat,
  • Pia C. Pafundi,
  • Alessandro Roggeri,
  • Daniela P. Roggeri,
  • Carlotta Rossi,
  • Emily Pintani,
  • Ilaria Meneghelli,
  • Francesca Lucca,
  • Anna Fratoni,
  • Gabriela Vega-Hernandez

摘要

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.