Evaluating Long-Term Effectiveness of Cystic Fibrosis Modulator Therapies after Rapid Adoption: A Dual-Approach Study
Ivacaftor was associated with improved ppFEV1 across all age groups, with the magnitude of improvement roughly 50% of that observed in clinical trials. The results support the need to account for modulator initiation bias and the use of within-subject analysis in future CFTR (cystic fibrosis transmembrane conductance regulator) modulator effectiveness studies, but caution is advised in younger individuals because of developmental changes that may affect pre- and post-treatment comparability.
Authors
- Eleni‐Rosalina Andrinopoulou (ORCID: https://orcid.org/0000-0002-5372-4163)
- Sanja Stanojevic (ORCID: https://orcid.org/0000-0001-7931-8051)
- David J. Pasta (ORCID: https://orcid.org/0000-0003-2637-9293)
- Michael S. Schechter (ORCID: https://orcid.org/0000-0002-8473-3767)
- Theodore G. Liou (ORCID: https://orcid.org/0000-0003-0121-4847)
- Christopher H. Goss (ORCID: https://orcid.org/0000-0001-8602-0309)
- Ruth H. Keogh (ORCID: https://orcid.org/0000-0001-6504-3253)
- Elizabeth A. Cromwell (ORCID: https://orcid.org/0000-0002-5106-0607)
- Rhonda D. Szczesniak (ORCID: https://orcid.org/0000-0003-0705-715X)
- Nicole Mayer-Hamblett (ORCID: https://orcid.org/0000-0001-5538-9197)
- Wayne J. Morgan (ORCID: https://orcid.org/0000-0002-6346-8140)
- Claire Wainwright (ORCID: https://orcid.org/0000-0001-8389-3809)
- Weiji Su (ORCID: https://orcid.org/0000-0002-5426-9764)
- Pedro Miranda Afonso (ORCID: https://orcid.org/0000-0001-6708-9597)
- Bruce C. Marshall
- Grace C Zhou
- Joshua S. Ostrenga
Institutions
- University of London (GB)
- Cincinnati Children's Hospital Medical Center (US)
- Dalhousie University (CA)
- St. Jude Children's Research Hospital (US)
- University of Arizona (US)
- Inserm (FR)
- The University of Queensland (AU)
- Eli Lilly (United States) (US)
- University of Washington (US)
- Université Paris Cité (FR)
- University of Utah (US)
- Cystic Fibrosis Foundation (US)
- Erasmus MC (NL)
- London School of Hygiene & Tropical Medicine (GB)
- Children’s Health Queensland Hospital and Health Service (AU)
- Hôpital Cochin (FR)
- Quantitative BioSciences (US)
- Cancer Research And Biostatistics (US)
- Cystic Fibrosis Research Foundation (IT)
- DMA Health Strategies (US)
- Cardiovascular Center Frankfurt (DE)
- Institut Cochin (FR)
- Pediatrics and Genetics (US)
- GGD Amsterdam (NL)
- Pulmonary and Critical Care Associates (US)
- Children's Hospital of Richmond at VCU (US)
- University of Cincinnati (US)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Annals of the American Thoracic Society
- Published
- 2025-09-08
- DOI
- https://doi.org/10.1513/annalsats.202503-267oc
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 0.00