Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2024 Jun;59(6):1614-1621.
doi: 10.1002/ppul.26953. Epub 2024 Mar 8.

Exploring perceptions of and decision-making about CFTR modulators

Lee Landess  1 , Mary G Prieur  2 , Ashley R Brown  2 , Elisabeth P Dellon  1
Affiliations

Exploring perceptions of and decision-making about CFTR modulators

Lee Landess et al. Pediatr Pulmonol. 2024 Jun.

Abstract

Introduction: Cystic fibrosis (CF) treatment has increasingly focused on highly effective modulators. Despite measurable benefits of modulators, there is little guidance for CF care team members on providing education and support to patients regarding initiation of these therapies. We aimed to explore patient, caregiver, and clinician perceptions of modulators and influences on decisions about starting cystic fibrosis transmembrane regulator (CFTR) modulators.

Methods: We conducted semistructured interviews with CF clinicians, adults with CF, and caregivers of children with CF. We reviewed audio recordings and coded responses to identify central themes.

Results: We interviewed 8 CF clinicians, 9 adults with CF, and 11 caregivers of children with CF. Themes centered on emotional responses to modulator availability, influences on decision-making, concerns about side effects, impact of modulators on planning for the future, the benefits of the multidisciplinary CF care team in supporting treatment decisions, and the unique needs of people with CF who are not eligible for modulators. Clinicians described changes in conversations about modulators since the approval of elexacaftor/tezacaftor/ivacaftor, specifically greater willingness to prescribe with less nuanced conversations with patients and/or caregivers regarding their use.

Conclusion: Based on perspectives and experiences of CF clinicians, adults with CF, and caregivers of children with CF, we suggest clinicians approach conversations about CFTR modulators thoughtfully and thoroughly, utilizing the multidisciplinary model of CF care in exploring patient and caregiver emotions while filling in knowledge gaps, asking about treatment goals beyond potential clinical benefit, and having compassionate conversations with those who are ineligible for modulators.

Keywords: communication; cystic fibrosis; decision making.

PubMed Disclaimer

References

REFERENCES

    1. Cystic Fibrosis Foundation. About cystic fibrosis. Accessed October 2023. http://www.cff.org/AboutCF/
    1. Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809‐1819.
    1. Gentzsch M, Mall MA. Ion channel modulators in cystic fibrosis. Chest. 2018;154(2):383‐393.
    1. Food and Drug Administration. FDA approves new breakthrough therapy for cystic fibrosis. 2019.
    1. Cystic Fibrosis Foundation Patient Registry 2022 Annual Data Report. Bethesda, Maryland 2022. Accessed October 2023. https://www.cff.org/sites/default/files/2021-11/Patient-Registry-Annual-...

MeSH terms

LinkOut - more resources