Impact of CFTR modulators on caregiver burden in cystic fibrosis: a cross-sectional study in Brazil.
5 affiliations
- 1Unidos pela Vida - Brazilian Institute of Cystic Fibrosis Care, Supera Consulting and Federal University of Paraná (UFPR), Curitiba, Brazil. [email protected].
- 2Postgraduate Program in Pharmaceutical Sciences, Department of Pharmacy, Federal University of Paraná (UFPR), Curitiba, Paraná, Brazil. [email protected].
- 3Unidos pela Vida - Brazilian Institute of Cystic Fibrosis Care and Supera Consulting, Curitiba, Brazil.
- 4Postgraduate Program in Pharmaceutical Sciences, Department of Pharmacy, Federal University of Paraná (UFPR), Curitiba, Paraná, Brazil.
- 5Unidos pela Vida - Brazilian Institute of Cystic Fibrosis Care and Federal University Fluminense, Rio de Janeiro, Brazil.
Abstract
Objective
To assess caregiver burden, health-related quality of life, and work productivity among primary caregivers of individuals with cystic fibrosis (CF) in Brazil, comparing caregivers of patients using elexacaftor/tezacaftor/ivacaftor (ETI) with those of non-users, and analyzing differences according to the age group of the care recipient.Methods
This cross-sectional study was conducted between September and November 2025 using an online questionnaire. Eligible participants were primary caregivers of individuals with CF living in Brazil. Collected data included sociodemographics, CFTR modulator use, caregiving time, and out-of-pocket health-related expenses. Validated instruments included the 12-item Zarit Burden Interview (ZBI-12), the EQ-5D-3L (utility index and Visual Analogue Scale), and the Work Productivity and Activity Impairment Questionnaire for Caregivers (WPAI: CG). Descriptive analyses and group comparisons were stratified by ETI use and age group of the care recipient.Results
A total of 174 caregivers from all Brazilian regions participated; 90.2% were mothers, with a median age of 35 years (IQR: 28-45). The individuals receiving care had a median age of 11 years (IQR: 7-14) among modulator users and 3 years (IQR: 1-7) among non-users, and 44.8% used CFTR modulators. Caregivers of non-modulator users dedicated significantly more time to daily caregiving (300 min [IQR: 180-300] vs. 180 min [IQR: 90-240]; p = 0.002) and reported more frequent consultations (p = 0.003). ZBI-12 scores were higher in the no-modulator group (20.0 [IQR: 16.0-25.2] vs. 15.5 [IQR: 11.2-23.0]; p = 0.006), with the greatest difference observed among caregivers of children under 5 years (20.0 vs. 14.0; p = 0.003), a group currently without access to modulators through the public health system. EQ-VAS was lower in the no-modulator group (65.0 [IQR: 50.0-80.0] vs. 70.0 [IQR: 50.0-80.0]; p = 0.041). Absenteeism (15.8% [IQR: 0.0-28.6] vs. 0.0% [IQR: 0.0-16.4]; p = 0.034), presenteeism (50.0% [IQR: 5.0-60.0] vs. 20.0% [IQR: 0.0-50.0]; p = 0.048), and activity impairment (50.0% [IQR: 22.5-70.0] vs. 30.0% [IQR: 0.0-60.0]; p = 0.011) were significantly higher among caregivers without modulator access.Conclusion
Use of CFTR modulators was associated with lower caregiver burden, better health perception, and reduced work productivity loss.
Related
- Hidden Impact of Hemodialysis on Informal Caregivers: A Cross-Sectional Study of Caregiver Burden in North India
- Bridging primary and palliative care: A cross-sectional study of the relationship between perceived access to family physicians and caregiver burden.
- Caring while aging: a cross-sectional study in Lebanon on caregiver burden, mental health, and the moderating effects of social and financial resources.
- Caregiver Burden in an Online Sample of Family Caregivers: Cross-Sectional Associations with Anticipatory Grief and Family Needs
- Caregiver burden, family resilience, and depressive and anxiety symptoms in parents of depressed adolescents: a cross-sectional mediation study
BibTeX
@article{oliveira2026impact,
title = {{Impact of CFTR modulators on caregiver burden in cystic fibrosis: a cross-sectional study in Brazil.}},
author = {Verônica Del Gragnano Stasiak Bednarczuk Oliveira and Vinícius Bednarczuk Oliveira and Marise Basso Amaral and Marilis Dallarmi Miguel},
journal = {Orphanet journal of rare diseases},
year = {2026},
month = jul,
doi = {10.1186/s13023-026-04514-z},
url = {https://europepmc.org/article/MED/42920171},
note = {CC BY},
}
RIS
TY - JOUR
TI - Impact of CFTR modulators on caregiver burden in cystic fibrosis: a cross-sectional study in Brazil.
AU - Verônica Del Gragnano Stasiak Bednarczuk Oliveira
AU - Vinícius Bednarczuk Oliveira
AU - Marise Basso Amaral
AU - Marilis Dallarmi Miguel
PY - 2026
DA - 2026/07/01
JO - Orphanet journal of rare diseases
DO - 10.1186/s13023-026-04514-z
UR - https://europepmc.org/article/MED/42920171
AB - Objective To assess caregiver burden, health-related quality of life, and work productivity among primary caregivers of individuals with cystic fibrosis (CF) in Brazil, comparing caregivers of patients using elexacaftor/tezacaftor/ivacaftor (ETI) with those of non-users, and analyzing differences according to the age group of the care recipient.Methods This cross-sectional study was conducted between September and November 2025 using an online questionnaire. Eligible participants were primary caregivers of individuals with CF living in Brazil. Collected data included sociodemographics, CFTR modulator use, caregiving time, and out-of-pocket health-related expenses. Validated instruments included the 12-item Zarit Burden Interview (ZBI-12), the EQ-5D-3L (utility index and Visual Analogue Scale), and the Work Productivity and Activity Impairment Questionnaire for Caregivers (WPAI: CG). Descriptive analyses and group comparisons were stratified by ETI use and age group of the care recipient.Results A total of 174 caregivers from all Brazilian regions participated; 90.2% were mothers, with a median age of 35 years (IQR: 28-45). The individuals receiving care had a median age of 11 years (IQR: 7-14) among modulator users and 3 years (IQR: 1-7) among non-users, and 44.8% used CFTR modulators. Caregivers of non-modulator users dedicated significantly more time to daily caregiving (300 min [IQR: 180-300] vs. 180 min [IQR: 90-240]; p = 0.002) and reported more frequent consultations (p = 0.003). ZBI-12 scores were higher in the no-modulator group (20.0 [IQR: 16.0-25.2] vs. 15.5 [IQR: 11.2-23.0]; p = 0.006), with the greatest difference observed among caregivers of children under 5 years (20.0 vs. 14.0; p = 0.003), a group currently without access to modulators through the public health system. EQ-VAS was lower in the no-modulator group (65.0 [IQR: 50.0-80.0] vs. 70.0 [IQR: 50.0-80.0]; p = 0.041). Absenteeism (15.8% [IQR: 0.0-28.6] vs. 0.0% [IQR: 0.0-16.4]; p = 0.034), presenteeism (50.0% [IQR: 5.0-60.0] vs. 20.0% [IQR: 0.0-50.0]; p = 0.048), and activity impairment (50.0% [IQR: 22.5-70.0] vs. 30.0% [IQR: 0.0-60.0]; p = 0.011) were significantly higher among caregivers without modulator access.Conclusion Use of CFTR modulators was associated with lower caregiver burden, better health perception, and reduced work productivity loss.
SN - 1750-1172
ER -