Wellness in the modulator era: An observational study of the impact of CFTR modulator therapy on the well-being of people with cystic fibrosis.

BACKGROUND

People with cystic fibrosis (PwCF) have experienced substantial improvements in health following use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies. However, less is known about how modulator therapies impact well-being.

METHODS

We used a cross-sectional observational study to identify relationships between CFTR modulator therapies, health-related quality of life (HRQoL), and well-being. Adult PwCF and caregivers of children with CF completed the Wellness in the Modulator Era (Well-ME) survey between June 22 and July 31, 2022. HRQoL was measured with PROMIS Global 10/Global 7 + 2 Parent Proxy. We used a mixed methods analysis to compare experiences and concerns of PwCF who currently (n = 665), no longer (n = 51), or never (n = 184) took modulator therapy.

RESULTS

Adult PwCF taking a modulator (n = 416) reported better PROMIS global physical health than those who no longer (n = 37) or never took a modulator (n = 94) and better PROMIS global mental health than those who never took a modulator. Caregiver-reported HRQoL was similar across children with CF who currently, no longer, or never took a modulator. PwCF taking a modulator reported larger improvements in physical health, quality of life, social well-being, and treatment burden than those who no longer or never took a modulator. Nearly one-quarter (23 %) of PwCF taking modulator therapy reported worsening of mental well-being.

CONCLUSIONS

This study expands our knowledge of well-being among PwCF in the CFTR modulator era as reported by patients and parents. Findings lay the groundwork for establishing future research priorities, policy efforts, and communications in areas that improve well-being for PwCF.

Informations de copyright

Copyright © 2024. Published by Elsevier B.V.

Déclaration de conflit d'intérêts

Declaration of competing interest The authors of this manuscript would like to disclose the following conflict of interest: E.A. reports a salary from the Cystic Fibrosis Foundation. T.K. reports grants from the Cystic Fibrosis Foundation and the National Institutes of Health and consulting fees from the Cystic Fibrosis Foundation. J.K. reports grants from the Cystic Fibrosis Foundation (VANCIT23AO-LAD and NELSON20QI0). C.L. receives an honorarium from the Cystic Fibrosis Foundation for time spent developing a survey. C.R. reports a salary from the Cystic Fibrosis Foundation. E.Y. reports a salary from the Cystic Fibrosis Foundation. A.V.C. reports grants from the Cystic Fibrosis Foundation (VANCIT23AO-LAD and NELSON20QI0), along with an honorarium and reimbursement for personal travel expenses to attend the 2023 CF Foundation Therapeutics Development Network meeting. J.A. reports funding support from the Cystic Fibrosis Foundation and the National Institute of Health, grants from the Cystic Fibrosis Foundation and the National Institute of Health, consulting fees from Anagram Therapeutics, and holds a leadership role in The Obesity Society. C.B. reports grants to her institution from the Cystic Fibrosis Foundation, Vertex Pharmaceuticals, and AstraZeneca, and serves as a board member of the Cystic Fibrosis Foundation's Indiana Chapter. J.C. receives a salary from the Cystic Fibrosis Foundation. R.C. reports an honorarium from the Cystic Fibrosis Foundation for time spent developing a survey. C.F. reports a Cystic Fibrosis Foundation Center Grant for salary support as a CF center director and for the Cystic Fibrosis Foundation STRC. A.G. reports grants from the Cystic Fibrosis Foundation, Dutch Cystic Fibrosis Foundation, National Heart, Lung, and Blood Institute, and Vertex Pharmaceuticals, along with consulting fees and honoraria paid directly from Vertex Pharmaceuticals, Saudi Pediatric Pulmonology Association, and Virginia Commonwealth Universit, along with support to attend meetings from Cystic Fibrosis Foundation, European Cystic Fibrosis Society, French Cystic Fibrosis Society, as well as a Leadership position at European Cystic Fibrosis Society committee and Cystic Fibrosis Foundation committee chair, both providing institutional funding. C.G. reports funding and grants from the National Institutes of Health, FDA, and the Cystic Fibrosis Foundation, along with Consulting fees paid directly from Enterprise Therapeutics Honoraria from Gilead Sciences and Vertex Pharmaceutical, and support to attend meetings from Vertex Pharmaceuticals and Enterprise Therapeutics, as well as Serving as a DSMB Chair for a trial supported by Novartis and the European Commission. Deputy Editor, Annals of the ATS, and Stock in Air Therapeutics. All other authors have nothing to disclose