Reduction in abdominal symptoms (CFAbd-Score), faecal M2-pyruvate-kinase and Calprotectin over one year of treatment with Elexacaftor-Tezacaftor-Ivacaftor in people with CF aged ≥12 years – The RECOVER study

Journal of Cystic Fibrosis(2023)

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摘要
•The present study uses the first validated CF-specific patient reported outcome measure focusing on gastrointestinal symptoms (CFAbd-Score) to demonstrate that ETI leads to substantial and sustained reduction in GI-symptom burden.•Symptom improvement is rapid, being evident at 1 month, peaking 2 months and stabilising thereafter.•Previous studies assessing effects of ETI on GI-symptoms had generated conflicting results.•For the first time, significant improvements in faecal M2-pyruvate Kinase (M2-PK), a marker of cell proliferation were seen on ETI, in addition to reductions in faecal calprotectin as previously reported.
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关键词
CFTR,modulation,gastro-intestinal (GI),patient reported outcome measure (PROM),faecal elastase
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