Cystic Fibrosis Questionnaire-Revised Domain Research Articles

Modulator Therapy in Cystic Fibrosis Patients with cis Variants in F508del Complex Allele: A Short-Term Observational Case Series.

Previous studies reported the influence of cis variants in F508del cystic fibrosis (CF) patients in their responses to CFTR modulators. The current study is a prospective, observational study involving three patients with CF and pancreatic insufficiency, carrying a complex allele including F508del with A238V, I1027T, or L467F. We report clinical data before and after 4 weeks of treatment with tezacaftor (TEZ)/ivacaftor (IVA), elexacaftor (ELX)/TEZ/IVA, and lumacaftor (LUM)/IVA for patients with complex alleles A238V, I1027T, and L467F, respectively. The 50-year-old patient bearing F508del;A238V/D1152H showed a normal sweat test (13 mEq/L) and improvements in forced expiratory volume in the first second (FEV1) (+7 points), body mass index (BMI) (+0.85), and respiratory CF Questionnaire-Revised (CFQ-R) domain (+22.2 points). The 12-year-old patient bearing F508del;I1027T/R709X showed an improvement in a sweat test (−40 mEq/l), FEV1 (+9 points) and the respiratory CFQ-R domain (+16.7 points). No changes in outcomes were observed for the 6-year-old patient F508del;L467F/F508del. Our data highlight that the reported variants do not modify the phenotypic expression of F508del. Searching L467F is crucial in CF patients with F508del nonresponsive to ELX/TEZ/IVA. Further data are needed to evaluate the clinical effect of these variants after a longer follow up.

Health-Related Quality of Life in Adults with Cystic Fibrosis: Familial, Occupational, Social, and Mental Health Predictors.

Background: Cystic Fibrosis (CF) adult patients experience daily physical symptoms and disabilities that may impact their quality of life and mental health. Methods: This prospective study aimed to evaluate the relative contribution of the familial, occupational, and social environment, besides that of the main physical and mental health factors, to the quality of life of CF adult patients using the Cystic Fibrosis Questionnaire-Revised (CFQ-R) in a multivariate model. Results: Fifty patients were analyzed (70% of men; median age of 25 years; median body mass index of 21 kg/m²; median FEV1 of 57%). Anxiety and depression scores were negatively associated with 9 of the 12 CFQ-R domains. When controlling for anxiety and depression, FEV1% and BMI were significant positive predictors of several domains of the CFQ-R. All the familial, occupational, and social components analyzed but one (professional training) were predictors of at least one domain of the CFQ-R. Conclusion: Anxiety and depression explained a greater proportion of the variance than physical variables (age, sex, BMI, FEV1%, and exacerbation in the last year) in CF HRQoL. Many familial, occupational, and social components were also specifically and independently predictors of some HRQoL domains. Their screening might help identifying CF patients eligible for specific interventions, focusing on the impaired QoL dimensions.

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

BackgroundThere 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. Methods125 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. ResultsNo 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. ConclusionHRQOL 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.

Pretransplant Quality of Life and Post-Transplant Survival in Adolescents with Cystic Fibrosis.

Cystic fibrosis (CF) is the most common indication for pediatric lung transplantation and the third most common for adults. The selection of candidates and timing of transplant is challenging and whether there is a survival benefit of this procedure for pediatric patients is controversial. Use of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a well-validated, disease-specific quality of life measure may improve pretransplant referral decision-making. This multicenter study evaluated whether specific domains on the CFQ-R (i.e., Physical Functioning, Respiratory Symptoms), assessed pretransplant, predicted survival 4-year post-transplant (n = 25). A two-step Cox regression, with physical predictors entered in step one (i.e., age, CF-related Diabetes, FEV1% predicted) and the Physical Functioning and Respiratory Symptoms CFQ-R scales entered in step two, was used to assess whether the CFQ-R explained additional and unique variance. Receiver Operating Characteristic (ROC) curves were used to assess the sensitivity and specificity of optimal cut-points of significant CFQ-R domains. The Respiratory Symptoms scale predicted survival 4-year post-transplant (Exp(B) = 0.38, 95% CI = 0.14-1.01; area under the curve = 0.87) and once it was added to the model, no other individual predictors were significant. The incremental improvement beyond the physical parameters approached but did not reach statistical significance (χ2 Δ = 5.79, p = .06). This study suggested that including patient-reported outcomes could aid pretransplant referral decision-making. The Respiratory Symptoms scale in particular may serve as a useful tool to help determine when to refer and evaluate an individual for transplant.

Prevalence of unmet palliative care needs in adults with cystic fibrosis

BackgroundPhysical and emotional burdens impair quality of life (QoL) in many adults with cystic fibrosis (CF). Palliative care (PC) improves QoL in other serious illnesses, yet the full array of palliative needs amenable to PC are unknown in CF. MethodsWe surveyed 164 adults with CF using the Supportive Care Needs Survey 34 (SCNS-34) to assess unmet PC needs across five domains, the Edmonton Symptom Assessment System (ESAS) to assess symptom burden, and the Cystic Fibrosis Questionnaire—Revised (CFQ-R) to assess CF-specific QoL. We assessed associations between SCNS-34 domain scores and respondent characteristics, including symptom burden and FEV1. ResultsMedian age was 29 years; 56% of respondents were male. Median FEV1 was 57% predicted. 78% of respondents reported ≥1 unmet PC need; physical and daily living (72%) and psychological (66%) needs were most prevalent. Symptom burden was correlated with all SCNS-34 domains scores, and strongly correlated with the physical (r = 0.79) and psychological (r = 0.72) domain scores. FEV1 was moderately inversely correlated with the physical domain score (r = −0.41). Forty-four of the 45 inverse correlations between SCNS-34 domain scores and CFQ-R domain scores were significant. Patient-reported depressive and anxiety symptoms were significantly associated with higher scores in five and four SCNS-34 domains, respectively. ConclusionsAdults with CF have substantial unmet PC needs. Patient-reported symptom burden is more strongly associated with reporting unmet PC needs than FEV1. Routine screening of unmet PC needs, using tools such as the SCNS-34, may enable CF care teams to optimize the provision of primary and specialist PC.

Measuring recovery in health-related quality of life during and after pulmonary exacerbations in patients with cystic fibrosis

BackgroundWe explored the time-dependent impact of pulmonary exacerbations (PEx) on health-related quality of life (HRQoL) using Cystic Fibrosis Questionnaire–Revised (CFQ-R) data from 2 large cystic fibrosis (CF) trials. MethodsThis exploratory post-hoc analysis evaluated the impact of PEx on CFQ-R domains of functioning in 80 patients with CF (homozygous for F508del-CFTR), aged ≥14 years randomized to placebo in the TRAFFIC and TRANSPORT trials who experienced 1 PEx. ResultsScores on the CFQ-R were significantly lower within 1 week of PEx start in 8 out of 12 domains (Respiratory Symptoms, Physical Functioning, Emotional Functioning, Health Perceptions, Role Functioning, Social Functioning, Eating, and Vitality). Patients whose PEx was treated with hospitalization or intravenous antibiotics had greater reductions in some domains of HRQoL compared with those treated with oral antibiotics. In the immediate weeks post-PEx, improvement was seen on Emotional Functioning, Respiratory Symptoms, and Health Perceptions, while further decline was seen for Eating, Physical Functioning, Role Functioning, Vitality, and Weight. For some measures (Physical Functioning, Vitality), full recovery to pre-PEx levels took several weeks. ConclusionsPulmonary exacerbations have significant effects on multiple domains of HRQoL, and recovery across multiple domains post-PEx can take several weeks. These findings provide insight into the impact of PEx on patient HRQoL and recovery post-PEx.Clinical trial registration:ClinicalTrials.gov identifiers, NCT01807923 and NCT01807949

WS09.4 The role of resilience in quality of life of adult patients with cystic fibrosis (CF)

Objectives Although several psychological studies on coping and quality of life suggested high levels of resilience in CF patients, empirical data on resilience in CF are barely existent. Thus, the purpose of the study was to investigate resilience in adult CF patients and its impact on quality of life. Methods 57 adult CF patients (18–59 years) participated in the study at their routine medical check-up. In addition to their regular psychological assessment including the Cystic Fibrosis Questionnaire – Revised (CFQ-R), the Resilience Scale (RS) was administered. The constructs were also correlated with physical parameters (age, gender and lung function). Results Patients reported high levels of resilience and overall good quality of life in all domains. Patient's resilience was significantly higher compared to the large healthy reference group (t[2086] = 4.7510, p=0.0001) and was independent of gender (t[55] = 0.826, p=0.412), age (r = 0.076, p=0.573) and lung function (FEV1%, r=0.165, p=0.220). Significant associations between resilience and CFQ-R domains could be confirmed. Conclusion Our study is one of the first attempts to explicitely test resilience in CF patients and could demonstrate high levels of resilience in these patient group and positive correlations with quality of life. These results suggest that resilience could be seen as an important factor on quality of life and coping with CF. Therefore fostering resilience early in patient's life should be encouraged.

Pulmonary function, functional capacity and quality of life in adults with cystic fibrosis

BackgroundAs there are few studies on the impact of respiratory and functional status on the quality of life domains in adults with cystic fibrosis, this study aimed to evaluate the association between respiratory function, functional capacity and quality of life in these subjects. MethodsThis is a cross-sectional study, where adults with clinical and laboratorial diagnoses of CF fibrosis underwent pulmonary function tests, the six-minute walk distance test (6MWT) and responded to the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Descriptive statistics was used to summarize the findings. The associations were tested by means of Pearson's or Spearman tests, and the significance level was set at 5%. ResultsThe 21 patients who completed the study presented with reduced quality of life in all CFQ-R domains, obstructive pulmonary disease and reduced 6MWT distance. The following associations were found between pulmonary function and CFQ-R domains: forced vital capacity – FVC (%) and treatment burden and digestive symptoms (r=−0.433, p<0.05; r=−0.443, p<0.05, respectively), forced expiratory volume in one second – FVC ratio – FEV1/FVC (%) and physical functioning, social and respiratory symptoms (r=0.5, p<0.05; r=0.58, p<0.01; r=0.45, p<0.05, respectively), residual volume (%) and physical functioning (r=0.49, p<0.05), airways’ resistance – Raw and physical functioning and emotional functioning (r=−0.44, p<0.05; r=−0,46, p<0.05, respectively), carbon monoxide diffusing capacity (%pred) and physical functioning (r=−0,51; p<0.05). ConclusionAdults with CF have reduced quality of life, which in part is associated with the severity of their lung function.

Longitudinal Trends in Health-Related Quality of Life in Adults With Cystic Fibrosis

Health-related quality-of-life (HRQOL) measures have been used as patient-reported outcomes in clinical trials in cystic fibrosis (CF), but there are limited data on HRQOL changes over time in adults with CF. The Project on Adult Care in Cystic Fibrosis, a prospective, longitudinal panel study of 333 adults with CF at 10 CF centers in the United States, administered a disease-specific HRQOL measure, the Cystic Fibrosis Questionnaire-Revised (CFQ-R), seven times over 21 months. The CFQ-R assesses both physical and psychosocial domains of health. Growth curve regression models were developed for each CFQ-R domain, adjusting for demographic and clinical characteristics. Between 205 and 303 adults completed surveys (response rate, 70%-93%). Mean age at baseline was 33 years (range, 19-64 years); mean FEV1 % predicted was 59.8% (SD, 22%). Over the 21 months of follow-up, lung function, frequency of pulmonary exacerbations, and nutritional indices were associated with physical CFQ-R domain scores. There were no significant population trends over time in the physical domain scores; however, there were population time trends in three psychosocial domains: treatment burden (+8.9 points/y), emotional functioning (+3.2 points/y), and social functioning (-2.4 points/y). Individual variation in both physical and psychosocial subscales was seen over 21 months. In a longitudinal multicenter population of adults with CF, clinical variables such as FEV1, exacerbation frequency, and weight were correlated with related CFQ-R subscales. For the population as a whole, the physical domains of CFQ-R, such as respiratory symptoms, were stable. In contrast, population changes in several psychosocial domains of CFQ-R suggest that differentiating between the physical and the psychosocial trajectories in health among adults with CF is critical in evaluating patient-reported outcomes.

Association among posture, lung function and functional capacity in cystic fibrosis

AimThe purpose of this study was to evaluate the correlations within pulmonary function, functional capacity, and posture in adult patients with cystic fibrosis (CF). A secondary aim was to evaluate the correlation between patient quality of life and postural assessment variables. MethodA cross-sectional study was conducted on fourteen patients with CF. Patients were subjected to a postural analysis (postural assessment software) and measurements of pulmonary function (spirometry, whole body plethysmography, and carbon monoxide diffusing capacity) and functional capacity (6-min walking test). All patients completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R). ResultsMost patients were male (57%), and the median age of the patients was 24.5 (22–34). The forced expiratory volume in one second, the 6-min walking distance, total lung capacity, and airway resistance were significantly correlated with the vertical alignment of the chest (ρ = −0.57, P < 0.05; ρ = −0.65, P < 0.01; ρ = 0.54, P < 0.05; and ρ = 0.67, P < 0.01, respectively). The ‘physical’ domain of the CFQ-R was significantly correlated with the vertical alignment of the chest (ρ = −0.74, P < 0.01), and the ‘limitations’ domain of the CFQ-R was significantly correlated with the angle of the hip (ρ = −0.55, P < 0.05). ConclusionThe present study shows that abnormalities in pulmonary function and functional capacity are associated with postural changes in adults with CF. However the severity of the postural abnormalities does not negatively influence the CFQ-R domains.

Associação entre postura, função pulmonar e capacidade funcional na fibrose quística

AimThe purpose of this study was to evaluate the correlations within pulmonary function, functional capacity, and posture in adult patients with cystic fibrosis (CF). A secondary aim was to evaluate the correlation between patient quality of life and postural assessment variables. MethodA cross-sectional study was conducted on fourteen patients with CF. Patients were subjected to a postural analysis (postural assessment software) and measurements of pulmonary function (spirometry, whole body plethysmography, and carbon monoxide diffusing capacity) and functional capacity (6-min walking test). All patients completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R). ResultsMost patients were male (57%), and the median age of the patients was 24.5 (22–34). The forced expiratory volume in one second, the 6-min walking distance, total lung capacity, and airway resistance were significantly correlated with the vertical alignment of the chest (ρ = −0.57, P < 0.05; ρ = −0.65, P < 0.01; ρ = 0.54, P < 0.05; and ρ = 0.67, P < 0.01, respectively). The ‘physical’ domain of the CFQ-R was significantly correlated with the vertical alignment of the chest (ρ = −0.74, P < 0.01), and the ‘limitations’ domain of the CFQ-R was significantly correlated with the angle of the hip (ρ = −0.55, P < 0.05). ConclusionThe present study shows that abnormalities in pulmonary function and functional capacity are associated with postural changes in adults with CF. However the severity of the postural abnormalities does not negatively influence the CFQ-R domains.

Quality of life and healthcare utilisation in cystic fibrosis: a multicentre study

The aim of our study was to discover the health status and healthcare utilisation associated with pulmonary exacerbations in cystic fibrosis (CF) and chronic Pseudomonas aeruginosa infection. Patients with CF from five UK CF centres attended two visits, 8-12 weeks apart. They were classified at visit 1 as being in one of the three health states: no current pulmonary exacerbation; "mild" (no hospitalisation) pulmonary exacerbation; and "severe" (hospitalisation) pulmonary exacerbation. All patients completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R) and EuroQol (EQ-5D) and a clinical form, and forced expiratory volume in 1 s (FEV1) was measured at visits 1 and 2. Annual healthcare utilisation data were collected. 94 patients of mean±sd age 28.5±8.2 yrs and FEV1 58.7±26.8% were recruited. 60 patients had no pulmonary exacerbation, 15 had a mild and 19 had a severe pulmonary exacerbation at visit 1. EQ-5D and CFQ-R data showed that the worse the exacerbation, the poorer the health-related quality of life (HRQoL). There were strong relationships between the CFQ-R and EQ-5D domain scores. The mean rate of pulmonary exacerbations per patient per year was 3.6 (1.5 in hospital and 2.2 at home). The mean length of stay per hospital pulmonary exacerbation was 9 days. As exacerbation status worsens, patients experience worse HRQoL. There is a significant healthcare burden associated with treatment of pulmonary exacerbation and long-term prophylaxis.

Six-Minute Walk Test and Health-Related Quality of Life: Objective Tools to Assess Improvement in Cystic Fibrosis Patients Hospitalized for Pulmonary Exacerbation

The 6 Minute Walk Test (6 MWT) has been shown to be a better test for submaximal exercise capacity than other submaximal exercise tests. The cystic fibrosis (CF) Health-Related Quality of Life Cystic Fibrosis Questionnaire-Revised (CFQ-R) has been used in CF patients in order to assess the quality of life. There are a few studies that examine measurements of multiple endpoints simultaneously [e.g., pulmonary function test (PFT), exercise capacity, CFQ-R] with treatment in CF pulmonary exacerbation. We hypothesize that the 6MWT is safe to perform during pulmonary exacerbation, and that 6 MWT, CFQ-R, and PFT improve with treatment in CF pulmonary exacerbation. We further hypothesized that the improvements in 6MWT, CFQ-R, and PFT are related. We performed 6 MWT, PFT, and CFQ-R in 21 CF subjects (age 7–20 years) admitted for pulmonary exacerbation. 6 MWT, PFT, and CFQ-R were completed at admission, at the end of week 1, and at the end of week 2. No complications were noted during the 6MWT.The 6MWT, PFT [forced expiratory volume in one second (FEV1), FVC and forced expiratory flow25%–75% (FEF25%–75%)], and CFQ-R domains (Respiratory and Physical) improved significantly at week 2. The physical domain change correlated with the 6MWT improvement, whereas the respiratory domain change correlated with the FEF 25%–75% improvement at week 2. There was no significant relationship observed between the 6MWT and PFT improvements. In conclusion, 6MWT is a safe and well-tolerated test, and it can be utilized as an adjunct or alternative outcome measure to PFT in acute CF pulmonary exacerbation. In addition, multiple outcome measures, including 6MWT and HRQOL, should be utilized to assess the efficacy of treatment in CF pulmonary exacerbation.

Longitudinal assessment of health‐related quality of life in an observational cohort of patients with cystic fibrosis

Patient-reported outcomes (PROs) are increasingly used to evaluate the efficacy of new treatments and the progression of chronic diseases. The Cystic Fibrosis Questionnaire-Revised (CFQ-R) is a disease-specific, PRO measure of health-related quality of life (HRQOL). We evaluated associations between changes in health status over time and HRQOL in a national CF database. Using the Epidemiologic Study of Cystic Fibrosis (ESCF) data, we identified participants who had completed age-appropriate CFQ-R assessments on two occasions separated by 9-15 months. Next, we developed multivariate regression models to test whether associations existed between (1) changes in respiratory signs/symptoms and changes in the respiratory health domains of the CFQ-R, (2) changes in nutritional health status and changes in the nutritional health domains of the CFQ-R, and (3) changes in treatment complexity and changes in the Treatment Burden scale of the CFQ-R. We analyzed 1,947 pairs of assessments: 337 child (mean age 8.9, range 6-13 years), 581 parent (mean age of child 8.8, range 6-13 years), 398 adolescent (mean age 15.3, range 14-17 years), and 631 adult (mean age 26.9, range 18-73 years). On average, we found little change in both health status indicators and CFQ-R domain scores over 1 year. Significant associations over time, however, were found between increases in respiratory symptoms and worse CFQ-R Respiratory Symptom scores, declining weight and worsening scores on CFQ-R nutritional health domains, and increases in treatment complexity and worsening CFQ-R Treatment Burden scores for parent respondents. Health status and HRQOL changes were small over a 1-year period in this CF population. However, changes in respiratory symptoms and weight were associated with significant changes on relevant CFQ-R scores, indicating that this PRO is sensitive to changes in health status over time.