Quality of life is poorly correlated to lung disease severity in school-aged children with cystic fibrosis

Phillip Pattie; Sarath Ranganathan; Joanne Harrison; Suzanna Vidmar; Graham L Hall; Rachel E Foong; Alana Harper; Kathryn Ramsey; Danielle Wurzel; AREST CF

doi:10.1016/j.jcf.2021.11.005

Quality of life is poorly correlated to lung disease severity in school-aged children with cystic fibrosis

J Cyst Fibros. 2022 May;21(3):e188-e203. doi: 10.1016/j.jcf.2021.11.005. Epub 2021 Nov 17.

Authors

Phillip Pattie¹, Sarath Ranganathan², Joanne Harrison², Suzanna Vidmar³, Graham L Hall⁴, Rachel E Foong⁴, Alana Harper⁵, Kathryn Ramsey⁵, Danielle Wurzel⁶; AREST CF

Affiliations

¹ Faculty of Medicine, Dentistry and Health Sciences, The University of Melbourne, Australia. Electronic address: phillip.pattie3@gmail.com.
² Infection and Immunity, Murdoch Children's Research Institute, Melbourne, Australia; Department of Paediatrics, The University of Melbourne, Australia; Department of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Australia.
³ Department of Paediatrics, The University of Melbourne, Australia; Clinical Epidemiology and Biostatistics Unit, Murdoch Children's Research Institute, Melbourne, Australia.
⁴ Wal-yan Respiratory Centre, Telethon Kids Institute, Nedlands, Western Australia, Australia; School of Allied Health, Faculty of Health Sciences, Curtin University, Perth, Australia.
⁵ Wal-yan Respiratory Centre, Telethon Kids Institute, Nedlands, Western Australia, Australia.
⁶ Infection and Immunity, Murdoch Children's Research Institute, Melbourne, Australia; Department of Paediatrics, The University of Melbourne, Australia; Department of Respiratory and Sleep Medicine, Royal Children's Hospital, Melbourne, Australia; Allergy and Lung Health Unit, Melbourne School of Population and Global Health, The University of Melbourne, Melbourne, Australia.

PMID: 34801433
DOI: 10.1016/j.jcf.2021.11.005

Abstract

Background: There is no data exclusively on the relationship between health-related quality-of-life (HRQOL) and lung disease severity in early school-aged children with cystic fibrosis (CF). Using data from the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) we assessed the relationships between HRQOL, lung function and structure.

Methods: 125 children aged 6.5-10 years enrolled in the AREST CF program were included from CF clinics at Royal Children's Hospital (RCH), Melbourne (n = 66) and Perth Children's Hospital (PCH), Perth (n = 59), Australia. Demographics, HRQOL measured by Cystic Fibrosis Questionnaire-Revised (CFQ-R), spirometry, multiple-breath washout (MBW) and chest CT were collected across two years. Correlation between CFQ-R scores and lung structure/function parameters and agreement between parent-proxy and child-reported HRQOL were evaluated.

Results: No correlation was observed between most CFQ-R domain scores and FEV1 z-scores, excepting weak-positive correlation with parent CFQ-R Physical (rho = 0.21, CI 0.02-0.37), and Weight (rho = 0.21, CI 0.03-0.38) domain and child Body domain (rho = 0.26, CI 0.00-0.48). No correlation between most CFQ-R domain scores and LCI values was noted excepting weak-negative correlation with parent Respiratory (rho = -0.23, CI ^-0.41-^-0.05), Emotional (rho = -0.24, CI ^-0.43-^-0.04), and Physical (-0.21, CI ^-0.39-^-0.02) domains. Furthermore, structural lung disease on CT data demonstrated little to no association with CFQ-R parent and child domain scores. Additionally, no agreement between child self-report and parent-proxy CFQ-R scores was observed across the majority of domains and visits.

Conclusion: HRQOL correlated poorly with lung function and structure in early school-aged children with CF, hence clinical trials should consider these outcomes independently when determining study end-points.

Keywords: Cystic Fibrosis; Cystic Fibrosis Questionnaire-Revised; Quality of Life; lung function; lung structure.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Australia / epidemiology
Child
Cystic Fibrosis*
Health Status
Humans
Lung / diagnostic imaging
Quality of Life*
Severity of Illness Index