Comparison of the Effect of CFTR Modulators <i>elexacaftor</i>/<i>tezacaftor</i>/<i>ivacaftor</i> and <i>lumacaftor</i>/<i>ivacaftor</i> via Serum Human Epididymis Protein 4 Concentration in <i>p.Phe508del-CFTR</i> Homozygous Cystic Fibrosis Patients.

ABSTRACT: Elevated human epididymis protein 4 (HE4) levels decreased in patients with CF (pwCF) in response to CFTR-specific drugs and negatively correlated with FEV1% predicted values (ppFEV1). Objectives: Although elexacaftor/tezacaftor/ivacaftor (ETI, Kaftrio^®) demonstrates more substantial effectiveness than lumacaftor/ivacaftor (LUM/IVA, Orkambi^®) in pwCF, plasma biomarkers have not been used to compare treatment efficacy. Hence, our aim was to correlate the change in HE4 levels and the clinical effects of these CFTR modulators (CFTRm). Methods: Serum HE4 concentrations were measured in a total of 123 pwCF homozygous for the p.Phe508del-CFTR variant before treatment and 1-6 months after either ETI or LUM/IVA administr

SUBMITTER: Pocsi M 

PROVIDER: S-EPMC12429638 | biostudies-literature | 2025 Sep

REPOSITORIES: biostudies-literature