Introduction of Ivacaftor/Lumacaftor in Children With Cystic Fibrosis Homozygous for F508del in the Netherlands: A Nationwide Real‐Life Study
Lum/iva was generally well tolerated in a real-life, nationwide pediatric cohort. The efficacy of lum/iva was comparable to phase 3 studies in children. LCI and BA analysis as markers of small airway and structural lung disease showed significant improvement which indicates the importance of these parameters to evaluate treatment effects of CF modulators in children.
Authors
- Michiel Bannier (ORCID: https://orcid.org/0000-0003-4823-5872)
- Yuxin Chen (ORCID: https://orcid.org/0000-0003-2549-0838)
- Marianne Nuijsink (ORCID: https://orcid.org/0000-0002-3948-2794)
- Judith M. Vonk (ORCID: https://orcid.org/0000-0001-7531-4547)
- Annelies M. Zwitserloot (ORCID: https://orcid.org/0000-0003-4519-9511)
- Gerard H. Koppelman (ORCID: https://orcid.org/0000-0001-8567-3252)
- Suzanne W. J. Terheggen‐Lagro (ORCID: https://orcid.org/0000-0002-3305-3745)
- Shima Aziz
- Hettie M. Janssens (ORCID: https://orcid.org/0000-0002-2210-9176)
- Peter Merkus (ORCID: https://orcid.org/0000-0002-1977-7384)
- Kate Groot
- Brigitte W.M. Willemse (ORCID: https://orcid.org/0000-0003-3009-0919)
- Harm A.W.M. Tiddens (ORCID: https://orcid.org/0000-0001-5628-6667)
- D. D. Zomer‐ van Ommen
Institutions
- University Medical Center Groningen (NL)
- Radboud University Nijmegen (NL)
- University of Groningen (NL)
- Utrecht University (NL)
- Erasmus MC (NL)
- Radboud University Medical Center (NL)
- University Medical Center Utrecht (NL)
- Maastricht University (NL)
- Nederlandse Cystic Fibrosis Stichting (NL)
- Emma Kinderziekenhuis (NL)
- Haga Hospital (NL)
- Amsterdam University Medical Centers (NL)
- Wilhelmina Children's Hospital (NL)
Publication Details
- Journal
- Pediatric Pulmonology
- Published
- 2025-01-01
- DOI
- https://doi.org/10.1002/ppul.27473
- Citations
- 2
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 3.04
Funders
- Vertex Pharmaceuticals