Elexacaftor/tezacaftor/ivacaftor and mental health: A workshop report from the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health working group

•This workshop focused on mental health and elexacaftor/tezacaftor/ivacaftor (ETI).•Literature on mental health impacts of ETI, especially pediatric, is limited.•Studies reporting pooled mean effects show no change or improved mental health.•Some people with CF report new or worsening mental health...

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Veröffentlicht in:Journal of cystic fibrosis 2024-11
Hauptverfasser: Bathgate, CJ, Fedele, DA, Tillman, EM, He, J, Everhart, RS, Reznikov, LR, Liu, FF, Kirby, K, Raffensperger, K, Traver, K, Riekert, KA, Powers, SW, Georgiopoulos, AM
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Sprache:eng
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Zusammenfassung:•This workshop focused on mental health and elexacaftor/tezacaftor/ivacaftor (ETI).•Literature on mental health impacts of ETI, especially pediatric, is limited.•Studies reporting pooled mean effects show no change or improved mental health.•Some people with CF report new or worsening mental health symptoms.•Preclinical/mechanistic investigations and innovative clinical studies are needed. This report summarizes the 2023 inaugural annual meeting of the Cystic Fibrosis Foundation's Prioritizing Research in Mental Health (PRIME) working group. This workshop focused on mental health and elexacaftor/tezacaftor/ivacaftor (ETI). We reviewed existing literature and identified key gaps and study design considerations in preclinical work, pharmacokinetics/pharmacodynamics, mood/anxiety, quality of life/self-perception, neuropsychological symptoms, sleep, and symptom management. Limited studies have identified behavioral changes with modulator exposure in rodent models of depression, anxiety, and cognition. Longitudinal human studies reporting mean changes generally show no change or improvement. However, case reports and single-center studies identify subgroups reporting new or worsening symptoms. Future studies should focus on understanding the role of CFTR in the nervous system, defining ETI impacts in preclinical models, and mechanistic investigations. Innovative methods with larger samples and comprehensive assessments are needed to determine the incidence of new/worsening symptoms throughout the lifespan and effective management strategies.
ISSN:1569-1993
1873-5010
1873-5010
DOI:10.1016/j.jcf.2024.11.006