Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers' observations across US centers

Christina J. Bathgate, Emily Muther, Anna M. Georgiopoulos, Beth Smith, Laura Tillman, Sonia Graziano, Marieke Verkleij, Paula Lomas, Alexandra Quittner

Research output: Contribution to journalArticleAcademicpeer-review

8 Citations (Scopus)


Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with unprecedented clinical improvements, transforming the management of cystic fibrosis (CF). However, side effects with implications for safety and well-being have been reported, including neuropsychiatric changes. This study aimed to better characterize the emerging positive and negative impacts of ETI. Methods: The Cystic Fibrosis Foundation's Mental Health Advisory Committee distributed a 26-item survey to US CF care teams to assess clinician observations of patient-reported experiences with ETI. Survey responses measured the prevalence of these effects in five domains: (1) positive physical and psychological effects, (2) sleep difficulties, (3) cognitive difficulties, (4) worsening mental health, and (5) concerns about the future and finances. Results: Seventy-five healthcare providers responded from a pediatric, adult, and combined centers. Positive physical effects of ETI and increased optimism were reported in the upper quartiles (50%–100%) and rated as having a significant impact on daily functioning. Sleep and cognitive difficulties were reported in 1%–24%, with slight impacts on functioning, and psychological symptoms (e.g., increased stress, depression, anxiety) and new psychiatric medications were reported in 1%–24%, with moderate impacts. Concerns about the future were reported in 1%–24%, with minimal impacts. Conclusion: Across US centers, providers most often observed positive physical effects of ETI. However, a variety of negative side effects were also reported, including sleep disruptions and worsening psychological functioning, which should be systematically monitored by CF teams. These national-level data are a first step in evaluating the prevalence and consequences of these side effects and can directly inform future studies.
Original languageEnglish
Pages (from-to)2469-2477
Number of pages9
JournalPediatric pulmonology
Issue number9
Early online date2023
Publication statusPublished - Sept 2023


  • Trikafta
  • anxiety
  • cognitive difficulties
  • depression
  • elexacaftor/tezacaftor/ivacaftor
  • insomnia
  • mental health

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