The Impact of Elexacaftor/tezacaftor/ivacaftor on Cystic Fibrosis Health-related Quality of Life and Decision-making About Daily Treatment Regimens: a Mixed Methods Exploratory Study

Overview

Journal Ther Adv Chronic Dis

Publisher Sage Publications

Date 2024 Aug 2

PMID 39091508

Authors

Melissa Basile

Jennifer Polo

Katherine Henthorne

Joan DeCelie-Germana

Susan Galvin

Janice Wang

Affiliations

Soon will be listed here.

Abstract

Background: Elexacaftor/tezacaftor/ivacaftor (ETI) has reduced many symptoms of cystic fibrosis (CF).

Objectives: We sought to identify the impact of ETI on both symptoms and treatment decisions among adults with CF.

Design: Participants were enrolled in a cross-sectional study. Surveys were sent a RedCap link. Semistructured interviews were administered remotely Microsoft Teams. Interviews were audio recorded and professionally transcribed.

Methods: We assessed Cystic Fibrosis Questionnaire-Revised (CFQ-R) subscales for physical, respiratory, emotion, and treatment, and analyzed semistructured interviews covering CF treatment regimens and daily living. Quantitative and qualitative results were analyzed separately and via a mixed-methods convergence coding matrix.

Results: Twenty-four adults with CF taking ETI were included. CFQ-R subscale scores (mean scores/standard deviation) were physical (82.1/22.8), respiratory (83.7/11.2), emotion (65.3/14.2), and treatment (57.5/20.1). Three themes about decision-making for non-ETI-treatments emerged: (1) How I'm feeling, (2) Not noticing a difference, and (3) Uncertainty about long-term impact of modifying treatment regimens, and we found participants weighed each of these factors in their treatment decisions. Key findings from mixed-methods analysis show that among individuals experiencing higher CFQ-R scores for physical and respiratory compared to emotion and treatment, there were statements indicating that while those participants were experiencing better physical health, many continued their burdensome treatment regimens.

Conclusion: With little long-term data on the impact of reducing non-ETI treatments, participants weighed how they were feeling, treatment efficacy beliefs, and risk tolerance when making treatment decisions.

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