What does it mean to be “healthy” when taking elexacaftor/tezacaftor/ivacaftor (ETI)? A qualitative study

Abstract

Background

Elexacaftor/tezacaftor/ivacaftor (ETI) has profoundly affected the health and lives of many people with CF (pwCF). The rapid change in health for pwCF taking ETI provided them an opportunity to reflect on what “being healthy” means. The goal of this secondary analysis was to document changes in the health and well-being of pwCF after starting ETI, beyond the expected physical benefits.

Methods

The Qualitative Understanding of Experiences with the SIMPLIFY Trial (QUEST) study evaluated pwCF's experiences on ETI, including participation in a withdrawal study, treatment burden, and experiences of health. Two sixty-minute interviews were conducted approximately four months apart, audio-recorded and transcribed. A phenomenological approach was used to identify text of interest and create a formal codebook.

Results

Ninety-one pwCF (mean age=27.8 years; range 14–67; 51 % male) and 23 caregivers of the teenagers completed at least one interview. Beyond the expected benefits of ETI, four themes were identified: (1) Night and Day Change in Health, (2) Reduced Cognitive Burden, (3) Shifting to Managing Overall Wellness and Co-Morbidities, and (4) Social/Self Identity Changes.

Conclusion

ETI has raised pwCF's expectations for their health and well-being. Current symptom and QOL measures may no longer capture the less perceptible ways pwCF on ETI experience changes in symptoms or health. CF care may need to adapt to focus more on general health, managing other comorbidities, and planning for the future.