Introduction <p>Elexacaftor/tezacaftor/ivacaftor (ETI) has been shown to substantially improve clinical outcomes among people living with cystic fibrosis (pwCF). The impact of ETI on health care resource utilization in the context of universal health care is largely unknown. We aimed to assess the impact of ETI on hospital and non-hospital health care resource utilization in a national cohort of pwCF up to 2 years after ETI initiation.</p> Methods <p>We included all pwCF aged 12 years or older in the Danish Cystic Fibrosis Cohort initiating ETI therapy between 1 September 2020 and 31 December 2022. The following health care contacts were reported: acute and elective hospitalizations, acute and elective outpatient contacts, general practitioner (GP) visits, other specialist visits, physiotherapist/chiropractor visits, pharmacy visits, and blood sampling appointments. Pre- and post-ETI data were analyzed using logistic and linear regression models estimating number of visits, days in hospital, and odds ratios (ORs) for one monthly contact.</p> Results <p>A total of 283 pwCF initiated ETI in the study period. At 24&#xa0;months post-ETI, utilization of the following health care resources was reduced: elective hospitalizations [OR 0.20 (95% CI: 0.08; 0.50)], elective outpatient hospital contacts [0.70 (0.57; 0.86)], pharmacy visits [0.56 (0.45; 0.71)], and blood sampling appointments [0.61 (0.49; 0.77)]. Number of contacts per month was reduced for the aforementioned outcomes, as well as number of days in hospital for elective hospitalizations. A downward but not statistically significant trend was observed for acute hospitalizations. No significant change was observed for acute outpatient visits, GP visits, other specialist visits, or visits to a physiotherapist/chiropractor.</p> Conclusion <p>In a national cohort of pwCF, ETI was associated with substantial reductions in elective hospitalizations, elective outpatient contacts, duration of elective hospitalizations, pharmacy visits, and blood sampling appointments, sustained 2 years post-ETI initiation. These findings highlight the real-world effectiveness of ETI in the context of a universal health care system.</p>

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Elexacaftor/Tezacaftor/Ivacaftor for Cystic Fibrosis: Impact on Hospitalizations and Health Care Resource Utilization in a Universal Health Care Setting

  • Hans Kristian Råket,
  • Mikkel Zöllner Ankarfeldt,
  • Joanna Nan Wang,
  • Tacjana Pressler,
  • Søren Jensen-Fangel,
  • Tavs Qvist,
  • Daniel Faurholt-Jepsen,
  • Espen Jimenez-Solem,
  • Janne Petersen,
  • Camilla Bjørn Jensen,
  • Christian Leo-Hansen,
  • Thomas Bryrup,
  • Inger Hee Mabuza Mathiesen,
  • Christine Højte Dahl,
  • Mette Frahm Olsen,
  • Esben Herborg Henriksen,
  • Terese Katzenstein,
  • Marianne Skov,
  • Bibi Uhre Nielsen,
  • Karlen Stade Bader Larsen,
  • Hanne Vebert Olesen,
  • Majbritt Jeppesen,
  • Espen Jimenez Solem

摘要

Introduction

Elexacaftor/tezacaftor/ivacaftor (ETI) has been shown to substantially improve clinical outcomes among people living with cystic fibrosis (pwCF). The impact of ETI on health care resource utilization in the context of universal health care is largely unknown. We aimed to assess the impact of ETI on hospital and non-hospital health care resource utilization in a national cohort of pwCF up to 2 years after ETI initiation.

Methods

We included all pwCF aged 12 years or older in the Danish Cystic Fibrosis Cohort initiating ETI therapy between 1 September 2020 and 31 December 2022. The following health care contacts were reported: acute and elective hospitalizations, acute and elective outpatient contacts, general practitioner (GP) visits, other specialist visits, physiotherapist/chiropractor visits, pharmacy visits, and blood sampling appointments. Pre- and post-ETI data were analyzed using logistic and linear regression models estimating number of visits, days in hospital, and odds ratios (ORs) for one monthly contact.

Results

A total of 283 pwCF initiated ETI in the study period. At 24 months post-ETI, utilization of the following health care resources was reduced: elective hospitalizations [OR 0.20 (95% CI: 0.08; 0.50)], elective outpatient hospital contacts [0.70 (0.57; 0.86)], pharmacy visits [0.56 (0.45; 0.71)], and blood sampling appointments [0.61 (0.49; 0.77)]. Number of contacts per month was reduced for the aforementioned outcomes, as well as number of days in hospital for elective hospitalizations. A downward but not statistically significant trend was observed for acute hospitalizations. No significant change was observed for acute outpatient visits, GP visits, other specialist visits, or visits to a physiotherapist/chiropractor.

Conclusion

In a national cohort of pwCF, ETI was associated with substantial reductions in elective hospitalizations, elective outpatient contacts, duration of elective hospitalizations, pharmacy visits, and blood sampling appointments, sustained 2 years post-ETI initiation. These findings highlight the real-world effectiveness of ETI in the context of a universal health care system.