Pediatr Pulmonol. 2025 Nov;60(11):e71367. doi: 10.1002/ppul.71367.
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.
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) were found.
CONCLUSIONS: Across international providers similar positive results were reported. A substantial proportion of PWCF across EU and United States 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 United States, they led to changes in MH medications. These international data document the importance of systematically assessing and managing these side-effects.
PMID:41222007 | DOI:10.1002/ppul.71367
