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Keogh RH, Cosgriff R, Andrinopoulou ER, et al. Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials. Thorax. 2021;doi: 10.1136/thoraxjnl-2020-216265.
Keogh, R. H., Cosgriff, R., Andrinopoulou, E. R., Brownlee, K. G., Carr, S. B., Diaz-Ordaz, K., Granger, E., Jewell, N. P., Lewin, A., Leyrat, C., Schlüter, D. K., van Smeden, M., Szczesniak, R. D., & Connett, G. J. (2021). Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials. Thorax, . https://doi.org/10.1136/thoraxjnl-2020-216265
Keogh, Ruth H, et al. "Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials." Thorax vol. (2021). doi: https://doi.org/10.1136/thoraxjnl-2020-216265
Keogh RH, Cosgriff R, Andrinopoulou ER, Brownlee KG, Carr SB, Diaz-Ordaz K, Granger E, Jewell NP, Lewin A, Leyrat C, Schlüter DK, van Smeden M, Szczesniak RD, Connett GJ. Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials. Thorax. 2021 Sep 23; doi: 10.1136/thoraxjnl-2020-216265. Epub 2021 Sep 23. PMID: 34556554.
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Thorax. 2021 Sep 23; doi: 10.1136/thoraxjnl-2020-216265. Epub 2021 Sep 23.

Projecting the impact of triple CFTR modulator therapy on intravenous antibiotic requirements in cystic fibrosis using patient registry data combined with treatment effects from randomised trials.

Thorax

Ruth H Keogh, Rebecca Cosgriff, Eleni-Rosalina Andrinopoulou, Keith G Brownlee, Siobhán B Carr, Karla Diaz-Ordaz, Emily Granger, Nicholas P Jewell, Alex Lewin, Clemence Leyrat, Daniela K Schlüter, Maarten van Smeden, Rhonda D Szczesniak, Gary J Connett

Affiliations

  1. Department of Medical Statistics, London School of Hygiene & Tropical Medicine, London, UK [email protected].
  2. Cystic Fibrosis Trust, London, UK.
  3. Department of Biostatistics, Erasmus Medical Center, Rotterdam, The Netherlands.
  4. Department of Paediatric Respiratory Medicine, Royal Brompton and Harefield NHS Foundation Trust, London, UK.
  5. Department of Medical Statistics, London School of Hygiene & Tropical Medicine, London, UK.
  6. Department of Public Health, Policy and Systems, Institute of Population Health, University of Liverpool, Liverpool, UK.
  7. Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
  8. Division of Biostatistics & Epidemiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA.
  9. Department of Pediatrics, University of Cincinnati, Cincinnati, Ohio, USA.
  10. National Institute for Health Research, Southampton Respiratory Biomedical Research Centre, University Hospital Southampton NHS Foundation Trust, Southampton, UK.

PMID: 34556554 DOI: 10.1136/thoraxjnl-2020-216265

Abstract

BACKGROUND: Cystic fibrosis (CF) is a life-threatening genetic disease, affecting around 10 500 people in the UK. Precision medicines have been developed to treat specific CF-gene mutations. The newest, elexacaftor/tezacaftor/ivacaftor (ELEX/TEZ/IVA), has been found to be highly effective in randomised controlled trials (RCTs) and became available to a large proportion of UK CF patients in 2020. Understanding the potential health economic impacts of ELEX/TEZ/IVA is vital to planning service provision.

METHODS: We combined observational UK CF Registry data with RCT results to project the impact of ELEX/TEZ/IVA on total days of intravenous (IV) antibiotic treatment at a population level. Registry data from 2015 to 2017 were used to develop prediction models for IV days over a 1-year period using several predictors, and to estimate 1-year population total IV days based on standards of care pre-ELEX/TEZ/IVA. We considered two approaches to imposing the impact of ELEX/TEZ/IVA on projected outcomes using effect estimates from RCTs: approach 1 based on effect estimates on FEV

RESULTS: ELEX/TEZ/IVA is expected to result in significant reductions in population-level requirements for IV antibiotics of 16.1% (~17 800 days) using approach 1 and 43.6% (~39 500 days) using approach 2. The two approaches require different assumptions. Increased understanding of the mechanisms through which ELEX/TEZ/IVA acts on these outcomes would enable further refinements to our projections.

CONCLUSIONS: This work contributes to increased understanding of the changing healthcare needs of people with CF and illustrates how Registry data can be used in combination with RCT evidence to estimate population-level treatment impacts.

© Author(s) (or their employer(s)) 2021. Re-use permitted under CC BY. Published by BMJ.

Keywords: clinical epidemiology; cystic fibrosis; respiratory infection

Conflict of interest statement

Competing interests: SBC reports personal fees and other from Chiesi Pharmaceuticals, non-financial support and other from Vertex, other from Zambon, other from Insmed, outside the submitted work.

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Grant support