Peer-Reviewed Publication
CHEST Pulm2024;2(4):100080.December 1, 2024Journal Article
Using Real-World Research to Study the Impact of Chronic Daily Therapy Discontinuation in Cystic Fibrosis: The Home-Reported Outcomes in Cystic Fibrosis 2 Study Design.
Bradley H Rosen1, Kevin J Psoter2, Kathryn A Sabadosa3, Georgene E Hergenroeder4, Lisa L Bendy5, Nell Meosky Luo6, Connie Zhang6, Clement L Ren4, Cynthia D Brown1
1Division of Pulmonary, Critical Care, Sleep, and Occupational Medicine, Department of Medicine, Indiana University School of Medicine, Indianapolis, IN.
2Division of General Pediatrics (K. J. P.), Department of Pediatrics, Johns Hopkins School of Medicine, Baltimore.
3Cystic Fibrosis Foundation (K. A. S.), Bethesda, MD.
4Division of Pulmonary and Sleep Medicine (G. E. H. and C. L. R.), Department of Pediatrics, Children's Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA.
5Division of Pulmonary, Allergy, and Sleep Medicine (L. L. B.), Department of Pediatrics, Riley Hospital for Children, Indiana University School of Medicine, Indianapolis, IN.
6Folia Health, Inc. (N. M. L. and C. Z.), Boston, MA.
Abstract
BACKGROUND: Chronic daily therapies (CDTs) are the foundation of clinical care for people with cystic fibrosis (CF), but these therapies impose considerable burden. In the era of elexacaftor/tezacaftor/ivacaftor (ETI) therapy, it is not clear if CDT discontinuation would lead to a greater decrease in lung function.
RESEARCH QUESTION: In people with CF who are taking or about to start ETI, does CD…
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