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

by Selleckchem | Sep 02 2023

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.

Comments:

The study you mentioned aimed to investigate the positive and negative impacts of the medication Elexacaftor/tezacaftor/ivacaftor (ETI) on individuals with cystic fibrosis (CF). ETI has been known to have significant clinical benefits for CF patients, but there have also been reports of side effects, particularly related to neuropsychiatric changes. The study was conducted by the Cystic Fibrosis Foundation's Mental Health Advisory Committee, and they distributed a survey to CF care teams across the United States to gather clinician observations of patient experiences with ETI.

The survey consisted of 26 items and focused on 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. Seventy-five healthcare providers from pediatric, adult, and combined centers responded to the survey.

The results indicated that ETI was associated with positive physical effects and increased optimism, which were reported by a significant proportion of respondents (in the upper quartiles of 50%-100%). These positive effects were perceived to have a notable impact on daily functioning. On the other hand, sleep difficulties and cognitive difficulties were reported by 1%-24% of respondents, with relatively mild impacts on functioning. Psychological symptoms such as increased stress, depression, anxiety, and the need for new psychiatric medications were reported by 1%-24% of respondents, with moderate impacts. Concerns about the future and finances were reported by 1%-24% of respondents, but these concerns had minimal impacts.

In conclusion, the study found that healthcare providers frequently observed positive physical effects of ETI in CF patients. However, there were also reports of negative side effects, including sleep disturbances and worsening psychological functioning. These findings suggest that CF teams should systematically monitor these side effects. The national-level data gathered from this study can serve as a starting point for evaluating the prevalence and consequences of these side effects and can inform future research in this area.