(47) Longitudinal Assessment of Health-related Quality of Life and Clinical Outcomes in Children and Adolescents with Cystic Fibrosis in Australia

Background: The Australian Cystic Fibrosis Data Registry (ACFDR) collects data from more than 3500 people with cystic fibrosis (CF). The registry currently does not capture health-related quality of life (HRQOL) measures. HRQOL is an important outcome, influencing decisions regarding the efficacy and value of health interventions in clinical settings. The aim of this study was to assess and compare child-reported HRQOL and evaluate relationship with clinical outcomes by analysing 10 years of patient reported outcome measures (PROMs) collected at one ACFDR contributing centre.

Methods: Children and adolescents aged 6–18 years completed their HRQOL using Cystic Fibrosis Questionnaire-Revised (CFQ-R) instrument between 2010 and 2020. Eight HRQOL domains were assessed: respiratory, physical, emotional, body, digestive, treatment burden, emotion and social. Demographic and diagnostic data, as well as body mass index (BMI) and forced expiratory volume in 1-second (FEV1) of study participants were extracted from the ACFDR. Descriptive analysis was used to describe the study population. Spearman’s rank correlation was calculated for CFQ-R domains and BMI and FEV1. P-values < 0.05 were considered to be significant.

Results: 47 children and adolescents completed at least one CFQ-R between 2010 and 2020. Of these 23 (48.9%) were female; 40 (85%) participants were 6-13 years of age, and 22 (46.8%) were F508del homozygous. A Spearman’s rho correlation test demonstrated a strong relationship between FEV1 and CFQ-R body domain (r = 0.328, p = 0.004) in females. A significant negative relationship between FEV1 and CFQ-R treatment body domain (r = - 0.262, p = 0.005) was observed in males. A strong positive relationship was also observed between BMI and CFQ-R physical domain (r= 0.243, p = 0.009, in males only) and the CFQ-R eating domain (r = 0.372, p=0.000 in males and r = 0.342, p=0.003 in females). Additional analyses and associations between HRQOL scores stratified by age groups and gender will be presented at the conference.

Conclusions: The initial findings of our study are consistent with those from the previous literature, suggesting that the CFQ-R is a meaningful clinical endpoint to evaluate clinical interventions in children. Through incorporating the CFQ-R into the ACFDR, this study will provide a patient perspective to clinicians and policy makers, enhancing the treatment and management of children and adolescents with CF.

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