Impacts of Elexacaftor/Tezacaftor/Ivacaftor: Comparison of Cystic Fibrosis Healthcare Providers’ Observations in Europe and the United States
Year: 2025
Type: preprint
Study design: Observational Study
Abstract: Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with improvements in physical health and well-being. However, there are growing concerns about side-effects related to sleep, cognition and new or worsening mental health in a subset. This study assessed healthcare providers’ perceptions of the positive and negative impacts of ETI and compared provider ratings in Europe to the United States (US). Methods: The European CF Society’s Mental Health Working Group adapted and distributed the survey developed by the CF Foundation’s Mental Health Advisory Committee. It assessed healthcare providers’ observations of the impacts of ETI in these domains: 1) positive and negative physical and psychological effects, 2) sleep difficulties, 3) cognitive difficulties, 4) worsening mental health, and 5) concerns about the future and finances. Results: Seventy-eight healthcare providers across 21 European (EU) countries responded, endorsing similar percentages of positive physical and psychological effects as US providers. However, in Europe lower percentages of MH side-effects (e.g., increased depression/anxiety, suicidal ideation, body image), and concerns about the future (e.g., copays, food insecurity). Conclusions: Across international providers similar positive results were reported. A substantial proportion of PWCF across EU and US experienced ETI side-effects related to sleep, cognition, and new or worsening mental health. However, EU providers reported significantly fewer side-effects related to mental health, negative body image, planning for the future and finances. MH side-effects led to dosing changes in ETI and in the US, they led to changes in MH medications. These international data document the importance of systematically assessing and managing these side-effects.
Institutions: Bambino Gesù Children's Hospital, Assaf Harofeh Medical Center, Innsbruck Medical University, University Hospital Innsbruck, Emma Kinderziekenhuis, Amsterdam University Medical Centers, Massachusetts General Hospital, Instytut Matki i Dziecka, University at Buffalo, State University of New York, National Jewish Health
Language: English
Subfield: Pulmonary and Respiratory Medicine
Field: Medicine
Domain: Health Sciences
Open Access status: gold
OpenAlex ID: W4413619953