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| Verfasst von: | Berges, Julian [VerfasserIn]  |
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| | Graeber, Simon Y. [VerfasserIn] |
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| | Hämmerling, Susanne Hedwig [VerfasserIn] |
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| | Yu, Yin [VerfasserIn] |
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| | Krümpelmann, Arne [VerfasserIn] |
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| | Stahl, Mirjam [VerfasserIn] |
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| | Hirtz, Stephanie [VerfasserIn] |
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| | Scheuermann, Heike [VerfasserIn] |
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| | Mall, Marcus A. [VerfasserIn] |
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| | Sommerburg, Olaf [VerfasserIn] |
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| Titel: | 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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| Verf.angabe: | Julian Berges, Simon Y. Graeber, Susanne Hämmerling, Yin Yu, Arne Krümpelmann, Mirjam Stahl, Stephanie Hirtz, Heike Scheuermann, Marcus A. Mall and Olaf Sommerburg |
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| E-Jahr: | 2023 |
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| Jahr: | 30 May 2023 |
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| Umfang: | 9 S. |
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| Fussnoten: | Gesehen am 03.07.2023 |
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| Titel Quelle: | Enthalten in: Frontiers in pharmacology |
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| Ort Quelle: | Lausanne : Frontiers Media, 2010 |
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| Jahr Quelle: | 2023 |
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| Band/Heft Quelle: | 14(2023) vom: Mai, Artikel-ID 1188051, Seite 1-9 |
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| ISSN Quelle: | 1663-9812 |
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| Abstract: | 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. |
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| DOI: | doi:10.3389/fphar.2023.1188051 |
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| URL: | kostenfrei: Volltext: https://doi.org/10.3389/fphar.2023.1188051 |
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| | kostenfrei: Volltext: https://www.frontiersin.org/articles/10.3389/fphar.2023.1188051 |
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| | DOI: https://doi.org/10.3389/fphar.2023.1188051 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| K10plus-PPN: | 1851464883 |
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| Verknüpfungen: | → Zeitschrift |
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| Lokale URL UB: |  Zum Volltext |
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Effects of lumacaftor—ivacaftor therapy on cystic fibrosis transmembrane conductance regulator function in F508del homozygous patients with cystic fibrosis aged 2-11 years / Berges, Julian [VerfasserIn]; 30 May 2023 (Online-Ressource)
