Quality of life amongst adolescents and young adults with cystic fibrosis: correlations with clinical outcomes

Abstract

OBJECTIVES:The current study sought to evaluate the quality of life of young patients with cystic fibrosis and correlate these results with the clinical parameters indicative of disease severity.METHODS:This cross-sectional study applied the validated Portuguese version of a cystic fibrosis specific quality of life questionnaire to clinically stable patients aged 14 to 21 years old. The correlations between the questionnaire domain scores and forced expiratory volume in one second (FEV1) values, the Shwachman-Kulczycki score, and body mass index were assessed, and correlations were considered as significant when p<0.05.RESULTS:A total of 31 patients (11 females; 16.4±2.3 years old) were evaluated, and the median scores on the questionnaire domains ranged from 66.7 to 100. A significant correlation was found between body mass index and the weight (r=0.43, p=0.016) and the eating questionnaire domains (r=0.44, p=0.013); between FEV1 and the physical (r=0.53, p=0.002) and treatment burden (r=0.41, p=0.023) domains; and between the Shwachman-Kulczycki score and the physical (r=0.39, p=0.03), health (r=0.41, p=0.023), and role (r=0.37, p=0.041) domains. A significant difference was found amongst patients with FEV1 values above or below 60% of the predicted value with regard to the role and health domains. No differences in the scores were found according to gender.CONCLUSIONS:The current cystic fibrosis specific quality of life questionnaire scores exhibited wide variability across all domains; however, they indicated a relatively satisfactory quality of life amongst the patients studied. Certain domains exhibited significant correlations with clinical parameters; thus, this instrument has consistent associations with clinical outcomes.

Efficacy and safety of elexacaftor plus tezacaftor plus ivacaftor versus tezacaftor plus ivacaftor in people with cystic fibrosis homozygous for F508del-CFTR: a 24-week, multicentre, randomised, double-blind, active-controlled, phase 3b trial

“…Increases in BMI are likely associated with improvements in several non-respiratory domains of the CFQ-R, specifically the Eating and Weight domains, for which significant correlations to BMI have previously been reported. 27 Taken together, these results demonstrate that ELX/TEZ/IVA improves CFTR function substantially, leading to broad clinical benefit.…”

Correspondence between lung function and symptom measures from the Cystic Fibrosis Respiratory Symptom Diary–Chronic Respiratory Infection Symptom Score (CFRSD-CRISS)

“…In a study conducted in Portugal, the authors evaluated the relationship between the CFQ-R and pulmonary function and found correlation coefficients ranging from 0.4-0.5 for outcomes such as treatment burden, digestive symptoms, and physical functioning [ 24 ]. Similarly weak to moderate correlations were found between pulmonary metrics and the CFQ-R in a Brazilian population [ 25 ]. Previous work using the population from the STOP study also found little correlation between the change in EQ-5D-5 L and lung function over the time that PEx were being treated [ 26 ].…”

Acceptability of patient reported outcome measures (PROMs) in a cystic fibrosis data registry

“…Both PROMs are reported to require approximately 10 min to complete. 17 18 We wished to understand the views of key stakeholders on national PROM administration through the ACFDR. Therefore, we conducted a qualitative study with the aim of assessing the perspectives of patients with CF, caregivers and clinicians on the usefulness and practicality of incorporating a CF-specific PROM into the ACFDR.…”