Article
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 - 1 Sept 2022
Keogh Ruth H, Cosgriff Rebecca, Andrinopoulou Eleni-Rosalina, Brownlee Keith G, Carr Siobhán B, Diaz-Ordaz Karla, Granger Emily, Jewell Nicholas P, Lewin Alex, Leyrat Clemence, Schlüter Daniela K, van Smeden Maarten, Szczesniak Rhonda D, Connett Gary J
Abstract excerpt
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...
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