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Berges, Julian [Verfasser:in]; Graeber, Simon Y. [Verfasser:in]; Hämmerling, Susanne Hedwig [Verfasser:in]; Yu, Yin [Verfasser:in]; Krümpelmann, Arne [Verfasser:in]; Stahl, Mirjam [Verfasser:in]; Hirtz, Stephanie [Verfasser:in]; Scheuermann, Heike [Verfasser:in]; Mall, Marcus A. [Verfasser:in]; Sommerburg, Olaf [Verfasser:in]

Effects of lumacaftor—ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years

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  • Medientyp: E-Artikel
  • Titel: Effects of lumacaftor—ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years
  • Beteiligte: Berges, Julian [Verfasser:in]; Graeber, Simon Y. [Verfasser:in]; Hämmerling, Susanne Hedwig [Verfasser:in]; Yu, Yin [Verfasser:in]; Krümpelmann, Arne [Verfasser:in]; Stahl, Mirjam [Verfasser:in]; Hirtz, Stephanie [Verfasser:in]; Scheuermann, Heike [Verfasser:in]; Mall, Marcus A. [Verfasser:in]; Sommerburg, Olaf [Verfasser:in]
  • Erschienen: 30 May 2023
  • Erschienen in: Frontiers in pharmacology ; 14(2023) vom: Mai, Artikel-ID 1188051, Seite 1-9
  • Sprache: Englisch
  • DOI: 10.3389/fphar.2023.1188051
  • Identifikator:
  • Entstehung:
  • Anmerkungen:
  • Beschreibung: Rationale: Lumacaftor/ivacaftor was approved for the treatment of patients with cystic fibrosis who are homozygous for F508del aged 2 years and older following positive results from phase three trials. However, the improvement in CFTR function associated with lumacaftor/ivacaftor has only been studied in patients over 12 years of age, while the rescue potential in younger children is unknown.Methods: In a prospective study, we aimed to evaluate the effect of lumacaftor/ivacaftor on the CFTR biomarkers sweat chloride concentration and intestinal current measurement as well as clinical outcome parameters in F508del homozygous CF patients 2-11 years before and 8-16 weeks after treatment initiation.Results: A total of 13 children with CF homozygous for F508del aged 2-11 years were enrolled and 12 patients were analyzed. Lumacaftor/ivacaftor treatment reduced sweat chloride concentration by 26.8 mmol/L (p = 0.0006) and showed a mean improvement in CFTR activity, as assessed by intestinal current measurement in the rectal epithelium, of 30.5% compared to normal (p = 0.0015), exceeding previous findings of 17.7% of normal in CF patients homozygous for F508del aged 12 years and older.Conclusion: Lumacaftor/ivacaftor partially restores F508del CFTR function in children with CF who are homozygous for F508del, aged 2-11 years, to a level of CFTR activity seen in patients with CFTR variants with residual function. These results are consistent with the partial short-term improvement in clinical parameters.
  • Zugangsstatus: Freier Zugang