Cystic Fibrosis Questionnaire-Revised Research Articles

Psychological resilience and intolerance of uncertainty in coping with cystic fibrosis

BackgroundAnxiety and depression are lower than to be expected in a considerable portion of cystic fibrosis (CF) patients. This outcome might be a result of substantial resilience and/or tolerance of uncertainty in coping with adversity. Research into resilience in cystic fibrosis is in its infancy. Methods57 adult CF patients participated in the study during their routine medical checkup. In addition to regular psychological assessment, the Intolerance of Uncertainty Scale (IUS) and the Resilience Scale (RS) were administered. The relative importance of IUS and RS in predicting quality of life in CF was explored. Bivariate correlations and predictive value of variables in multiple regressions on subscales of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) were calculated. ResultsRemarkably, resilience (personal competence and acceptance) was clearly elevated, whereas intolerance of uncertainty was comparable to healthy reference groups. In multiple regressions, personal competence emerged as strongest resilience variable in the prediction of quality of life. ConclusionsCF patients in our study seem to be particularly resilient rather than cognitively avoidant. At this stage of research, fostering personal competence in CF patients is most promising in improving quality of life.

Validating chest MRI to detect and monitor cystic fibrosis lung disease in a pediatric cohort.

Computed Tomography (CT) is the gold standard to assess bronchiectasis and trapped air in cystic fibrosis (CF) lung disease, but has the disadvantage of radiation exposure. Magnetic Resonance Imaging (MRI) is a radiation free alternative. To validate MRI as outcome measure by: correlating MRI scores for bronchiectasis and trapped air with clinical parameters, and by comparing those MRI scores with CT scores. In patients with CF (aged 5.6-17.4 years), MRI and CT were alternated annually during routine annual check-ups between July 2007 and January 2010. Twenty-three children had an MRI performed 1 year prior to CT, 34 children had a CT 1 year prior to MRI. Bronchiectasis and trapped air were scored using the CF-MRI and CF-CT scoring system. CF-MRI scores were correlated with clinical parameters: FEV1 , Pseudomonas aeruginosa, pulmonary exacerbations and patient-reported respiratory symptoms measured on the Cystic Fibrosis Questionnaire-Revised (CFQ-R), using Spearman's correlation coefficient. MRI and CT scores were compared using intra-class correlation coefficients (ICC) and Bland-Altman plots. Fifty-seven patients who had an MRI, CT and CFQ-R during the study period were included. CF-MRI bronchiectasis correlated with FEV1 , Pseudomonas aeruginosa, pulmonary exacerbations and patient-reported respiratory symptoms. CF-MRI trapped air only correlated with FEV1 and Pseudomonas aeruginosa. ICCs between MRI and CT bronchiectasis and trapped air were 0.41 and 0.35 respectively. MRI tended to overestimate bronchiectasis compared to CT. The associations between CF-MRI scores and several important clinical parameters further contributes to the validation of MRI. MRI provides different information than CT.

Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor

The cystic fibrosis (CF) transmembrane conductance regulator (CFTR) gating mutation G551D prevents sufficient ion transport due to reduced channel-open probability. Ivacaftor, an oral CFTR potentiator, increases the channel-open probability. To further analyze improvements in weight and body mass index (BMI) in two studies of ivacaftor in patients aged ≥6 years with CF and the G551D mutation. Patients were randomized 1:1 to ivacaftor 150 mg or placebo every 12 h for 48 weeks. Primary end point (lung function) was reported previously. Other outcomes included weight and height measurements and CF Questionnaire-Revised (CFQ-R). Studies included 213 patients (aged ≤ 20 years, n = 105; aged > 20 years, n = 108). In patients ≤20 years, adjusted mean change from baseline to week 48 in body weight was 4.9 versus 2.2 kg (ivacaftor vs. placebo, p = 0.0008). At week 48, change from baseline in mean weight-for-age z-score was 0.29 versus -0.06 (p < 0.0001); change in mean BMI-for-age z-score was 0.26 versus -0.13 (p < 0.0001). In patients >20 years, adjusted mean change from baseline to week 48 in body weight was 2.7 versus -0.2 kg (p = 0.0003). Mean BMI change at week 48 was 0.9 versus -0.1 kg/m(2) (p = 0.0003). There was no linear correlation evident between changes in body weight and improvements in lung function or sweat chloride. Significant CFQ-R improvements were seen in perception of eating, body image, and sense of ability to gain weight. Nutritional status improved following treatment with ivacaftor for 48 weeks.

Validity of a Spanish Version of the Leicester Cough Questionnaire in Children With Cystic Fibrosis

BackgroundCystic fibrosis (CF) patients present chronic cough as one of the main symptoms, which has an important effect on quality of life and social relations. Our goal was to validate the Spanish version of the Leicester Cough Questionnaire (LCQ) in a group of children and teenagers with CF. MethodsAfter adapting to Spanish by standardized translation and retro-translation methodology, a sample of 58 stable CF patients from 7 to 18years were recruited from three CF specialized centers in Spain. The questionnaire was administered twice; the second administration (LCQ2) was performed between 2 and 4 weeks later than the first one (LCQ1), in order to analyze the reliability and validity of the Spanish version. To correlate results with health related quality of life (HRQoL) we used the Cystic Fibrosis Questionnaire-Revised (CFQ-R). ResultsPopulation was composed by 62% male, age 11.7±3.1years and body mass index (BMI) 19±3kg/m2. Total scores from LCQ were: LCQ1 19 (17.75–21) vs LCQ2 19 (16–21) (P=.199). Cronbach's Alpha coefficient was 0.83 for the LCQtotal and for each specific domain was: 0.82 LCQphysical; 0.74 LCQpsychological and 0.62 LCQsocial. Intraclass correlation coefficient was: 0.69 LCQphysical; 0.59 LCQpsychological; 0.45 LCQsocial and 0.71 LCQtotal (good reliability). Relations with CFQ-R showed moderated and significant results: LCQtotalr=0.51 (P<.001) and respiratory symptoms domain r=0.67 (P<.05). ConclusionThe Spanish version of the Leicester Cough Questionnaire is reliable and valid for children and adolescents with CF and it has good relations with health related quality of life in this population.

Validación de la versión en español del Cuestionario de Tos Leicester en niños con fibrosis quística

BackgroundCystic fibrosis (CF) patients present chronic cough as one of the main symptoms, which has an important effect on quality of life and social relations. Our goal was to validate the Spanish version of the Leicester Cough Questionnaire (LCQ) in a group of children and teenagers with CF. MethodsAfter adapting to Spanish by standardized translation and retro-translation methodology, a sample of 58 stable CF patients from 7 to 18years were recruited from three CF specialized centers in Spain. The questionnaire was administered twice; the second administration (LCQ2) was performed between 2-4 weeks later than the first one (LCQ1), in order to analyse the reliability and validity of the Spanish version. To correlate results with health related quality of life (HRQoL) we used the Cystic Fibrosis Questionnaire-Revised (CFQ-R). ResultsPopulation was composed by 62% male, age 11.7±3.1years and body mass index (BMI) 19±3kg/m2. Total scores from LCQ were: LCQ1 19 (17.75-21) vs LCQ2 19 (16-21) (P=.199). Cronbach's Alpha coefficient was 0.83 for the LCQtotal and for each specific domain was: 0.82 LCQphysical; 0.74 LCQpsychological and 0.62 LCQsocial. Intraclass correlation coefficient was: 0.69 LCQphysical; 0.59 LCQpsychological; 0.45 LCQsocial and 0.71 LCQtotal (good reliability). Relations with CFQ-R showed moderated and significant results: for the LCQtotal: respiratory symptom domain r = 0,51 (P<.001) and physical capacity domain r = 0,62 (P<.05). ConclusionThe Spanish version of the Leicester Cough Questionnaire is reliable and valid for children and adolescents with CF and it has good relations with health related quality of life in this population.

Effect of ivacaftor treatment in patients with cystic fibrosis and the G551D-CFTR mutation: patient-reported outcomes in the STRIVE randomized, controlled trial.

BackgroundCystic fibrosis (CF) is an inherited, rare autosomal recessive disease that results in chronically debilitating morbidities and high premature mortality. We evaluated how ivacaftor treatment affected CF symptoms, functioning, and well-being, as measured by the Cystic Fibrosis Questionnaire-Revised (CFQ-R), a widely-used patient-reported outcome (PRO) measure.MethodsSTRIVE, a double-blind, placebo-controlled randomized trial, evaluated ivacaftor (150 mg) in CF patients aged 12+ with the G551D-CFTR mutation for 48 weeks. Treatment effect analysis used a mixed-effects repeated measures model. Treatment benefit analyses applied the cumulative distribution function and a categorical analysis of change scores (“improvement,” “no change,” or “decline”). Content-based interpretation examined treatment effect on specific item responses.ResultsData from 152 patients with a baseline CFQ-R assessment were analyzed. The treatment effect analysis favored treatment with ivacaftor over placebo on the Body Image, Eating, Health Perceptions, Physical Functioning, Respiratory, Social Functioning, Treatment Burden, and Vitality scales. Findings were supported by the analysis of categorical change. On all CFQ-R scales, the percentage of patients who improved was greater for ivacaftor. In the content-based analysis, the treatment benefit was characterized by better scores across a broad range of domains.ConclusionsResults illustrate broad benefits of ivacaftor treatment across many domains: respiratory symptoms, physical and social functioning, health perceptions, and vitality, as measured by the CFQ-R. The breadth of improvements reflects the systemic mechanism of action of ivacaftor compared to other therapies. Findings support the patient-reported value of ivacaftor treatment in this patient population.Trial RegistrationClinicalTrials.gov NCT00909532Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-015-0293-6) contains supplementary material, which is available to authorized users.

Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial

Ivacaftor has been previously assessed in patients with cystic fibrosis with Gly551Asp-CFTR or other gating mutations. We assessed ivacaftor in patients with Arg117His-CFTR, a residual function mutation. We did a 24-week, placebo-controlled, double-blind, randomised clinical trial, which enrolled 69 patients with cystic fibrosis aged 6 years and older with Arg117His-CFTR and percentage of predicted forced expiratory volume in 1 s (% predicted FEV1) of at least 40. We randomly assigned eligible patients (1:1) to receive placebo or ivacaftor 150 mg every 12 h for 24 weeks. Randomisation was stratified by age (6-11, 12-17, and ≥18 years) and % predicted FEV1 (<70, ≥70 to ≤90, and >90). The primary outcome was the absolute change from baseline in % predicted FEV1 through week 24. Secondary outcomes included safety and changes in sweat chloride concentrations and Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain scores. An open-label extension enrolled 65 of the patients after washout; after 12 weeks, we did an interim analysis. After 24 weeks, the treatment difference in mean absolute change in % predicted FEV1 between ivacaftor (n=34) and placebo (n=35) was 2·1 percentage points (95% CI -1·13 to 5·35; p=0·20). Ivacaftor treatment resulted in significant treatment differences in sweat chloride (-24·0 mmol/L, 95% CI -28·01 to -19·93; p<0·0001) and CFQ-R respiratory domain (8·4, 2·17 to 14·61; p=0·009). In prespecified subgroup analyses, % predicted FEV1 significantly improved with ivacaftor in patients aged 18 years or older (treatment difference vs placebo: 5·0 percentage points, 95% CI 1·15 to 8·78; p=0·01), but not in patients aged 6-11 years (-6·3 percentage points, -11·96 to -0·71; p=0·03). In the extension study, both placebo-to-ivacaftor and ivacaftor-to-ivacaftor groups showed % predicted FEV1 improvement (absolute change from post-washout baseline at week 12: placebo-to-ivacaftor, 5·0 percentage points [p=0·0005]; ivacaftor-to-ivacaftor, 6·0 percentage points [p=0·006]). We did not identify any new safety concerns. The studies are registered with ClinicalTrials.gov (the randomised, placebo-controlled study, number NCT01614457; the open-label extension study, number NCT01707290). Although this study did not show a significant improvement in % predicted FEV1, ivacaftor did significantly improve sweat chloride and CFQ-R respiratory domain scores and lung function in adult patients with Arg117His-CFTR, indicating that ivacaftor might benefit patients with Arg117His-CFTR who have established disease. Vertex Pharmaceuticals Incorporated.

WS09.5 Effect of ivacaftor on wellness, quality of life and cognitive function in adults with cystic fibrosis and G551D mutation

The effects of the potentiating agent ivacaftor on well-being and cognitive function are little known in adults with CF and G551D mutation. Objectives To evaluate the effects of ivacaftor on wellness, quality of life (QOL) and cognitive function in adults with CF; to evaluate a new wellness score for adults with CF. Methods In a placebo controlled crossover trial with 28 days of treatment in 20 adult patients the following were measured at 5 timepoints: The Cystic Fibrosis Questionnaire-Revised (CFQ-R), The Alfred Wellness Score for CF (AweScoreCF) and the Montreal Objective Cognitive Function Assessment (MOCA). Results See the table. In the CFQ-R change in social, health, physical, role and overall QOL improved significantly as did energy, exercise participation and total wellness in the AweScoreCF. There was strong agreement between the CFQ-R and the AweScoreCF. Patients found the AweScoreCF (10 questions) quicker to complete compared to the CFQ-R (50 questions). There were more completed AweScoreCF questionnaires than CFQ-R. There were some improvements in the MOCA in some patients following treatment. EndpointPercentage change from baseline, median (IQR)PlaceboIvacaftorCFQ-R18.1 (−30.8, 67.0)90.6 (41.7, 139.5) * AweScoreCF−0.5 (−6.9, 5.9)10.2 (3.8, 16.6) * MOCA0.1 (−0.7, 0.9)0.7 (−0.2, 1.5)*p Conclusions Treatment with ivacaftor resulted in significant improvements in quality of life, increased wellness, energy and exercise participation. There was strong agreement between the CFQ-R and AweScoreCF. The AweScoreCF may be a useful wellness assessment tool in the CF clinic. Study supported by Vertex Inc.

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.

Comparison of health-related quality of life among children with cystic fibrosis and their parents in two Eastern European countries

BackgroundIn the optimal care of cystic fibrosis (CF) patients, not only medical parameters are respected but also health-related quality of life (HRQOL). The aim of our study was to compare HRQOL of CF patients from two Eastern European countries. Methods141 patients with CF (6–18years) and 102 parents completed the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Data about disease severity, type of children's education and questions about parents' employment status were collected. ResultsIn the patient group, a significant difference was found only in Treatment burden, whereas in the parent group, there were significant differences in Treatment burden, Emotional functioning, Eating and Digestive symptoms between the two countries. School attendance was revealed as an important factor influencing HRQOL. ConclusionsObserved differences in evaluation of HRQOL may be caused by different therapeutic and diagnostic challenges between countries. To identify possible presence of psychosocial problems, monitoring of HRQOL is recommended.

Halotherapy in Patients with Cystic Fibrosis: A Pilot Study

Objectives: Cystic fibrosis (CF) is a complex genetic disorder involving the lower and upper respiratory tract. The purpose of this study is to evaluate the effect of Halotherapy on sinusitis symptoms, dyspnea, pulmonary function tests, and quality of life in CF patients. Study design: This was a pilot open-label before-and-after-study. Settings: The study was performed at the Salt Room® Orlando. Participants were from a single CF care center, and were enrolled in the study between January and June, 2012. Subjects and methods: Patients with clinically stable CF were included in the study. Participants received 9 sessions of HT, 45 minutes each, completed over a 3-week period. Study endpoints include: FEV1 and FVC, Borg dyspnea index test, Cystic Fibrosis Questionnaire-Revised (CFQ-R), and the Sino-Nasal Outcome Test (SNOT-20). Results: Twelve patients completed the study protocol. FEV1 and FVC did not change significantly (p= 0.49 and 0.87, respectively). SNOT-20 score improved by 0.62 points (95% CI -1.03 to -0.2, P = 0.007). There was a trend for improvement in Borg Dyspnea index; the mean score decreased by 0.79 (95% CI -1.64 to 0.05, P=0.065). There was significant improvement in the physical domain, the health perception domain, and the digestive domain.

Coping styles in adults with cystic fibrosis: implications for emotional and social quality of life

As life expectancy increases, interest has grown surrounding the factors that may influence quality of life (QOL) for people with cystic fibrosis (CF). The aim of the current study was to examine which specific coping styles were positively or negatively associated with social and emotional QOL in a CF sample. One hundred and twenty-two respondents aged 18 and over were recruited through an online support group. Respondents completed the ‘CF Questionnaire-Revised (CFQ-R)’ and the ‘Brief COPE’. The CFQ-R is a disease-specific instrument designed to measure the impact of CF on nine QOL domains and the Brief COPE is a 28 item questionnaire which assesses 14 coping scales. A multivariate regression model revealed that higher substance abuse and disengagement was associated with lower emotional QOL whereas greater use of religion, instrumental coping and acceptance was positively associated with emotional QOL. Active coping was linked to better social QOL and a negative association was reported between distraction coping with both emotional and social domains. Given the burden of CF, ascertaining which factors enhance or diminish emotion and social well-being is now an integral component of QOL research. The current findings may therefore have value in informing clinical interventions which aim to cater for the psychological needs of individuals with CF.

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.

Mapping the EQ-5D index from the cystic fibrosis questionnaire-revised using multiple modelling approaches.

BackgroundThis study was designed to develop a mapping algorithm to estimate EQ-5D utility values from Cystic Fibrosis Questionnaire-Revised (CFQ-R) data.MethodsA cross-sectional survey of adults with cystic fibrosis (CF) was conducted in the UK. The survey consisted of the CFQ-R, the EQ-5D and a background questionnaire. Eight regression models, exploring item and domain level predictors, were evaluated using three different modelling approaches: ordinary least squares (OLS), Tobit, and a two-part model (TPM). Predictive performance in each model was assessed by intraclass correlations, information criteria (Bayesian information criteria and Alkaike information criteria), and root mean square error (RMSE).ResultsThe survey was completed by 401 participants. For all modelling approaches the best performing item level model included all items, and the best performing domain level model included the CFQ-R Physical-, Role- and Emotional-functioning, Vitality, Eating Disturbances, Weight, and Digestive Symptoms domains and a selection of squared terms. Overall, the item level TPM, including age and gender covariates performed best within sample validation, but OLS and TPM domain models with squared terms performed best out-of-sample and are recommended for mapping purposes.ConclusionsDomain and item level models using all three modelling approaches reached an acceptable degree of predictive performance with domain models performing well in out-of-sample validation. These mapping functions can be applied to CFQ-R datasets to estimate EQ-5D utility values for economic evaluations of interventions for patients with cystic fibrosis. Further research evaluating model performance in an independent sample is encouraged.Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-015-0224-6) contains supplementary material, which is available to authorized users.

Depression and Anxiety among Parents of Children with Cystic Fibrosis Related to the Children’s Health Related Quality of Life

The aim was to examine depression and anxiety among parents of children with Cystic Fibrosis and the association between the parents’ symptoms and the child’s quality of life as rated by the parents. Parents completed HADS (Hospital Anxiety and Depression Scale), and CES-D (Center of Epidemiologic Studies Depression Scale), and assessed the children’s quality of life with CFQ-R (Cystic Fibrosis Questionnaire-Revised). Anxiety amongst the parents was higher than the general population (m = 6.55, SD = 3.54, p < 0.001). The level of depression does not differ from that of the general population. Mothers showed more anxiety symptoms than fathers (p < 0.001). Gender differences were not significant for depression symptoms. There is a strong association between the fathers’ self-rated depression and their rating of their child’s health. Both children and parents should be paid attention to in order to identify mental ill-health and take measures in good time.

Quality of life in children with CF: Psychometrics and relations with stress and mealtime behaviors.

The purpose of this study was to evaluate the utility of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) with toddlers and preschool-aged children. Clinically relevant relations between health-related quality of life (HRQOL), stress, and mealtime behaviors have not been examined. It was hypothesized that problematic mealtime behaviors and increased stress would be negatively associated with HRQOL. Parents of 73 children (2-6 years) with CF completed questionnaires assessing their children's generic (PedsQL) and CF-specific HRQOL, parenting and CF-specific stress, and mealtime behaviors. CFQ-R Physical, Eating, and Respiratory HRQOL subscales had acceptable to strong reliability (alphas = 0.73-0.86); other scales approached acceptable reliability. Lower CF-specific stress was associated with higher CFQ-R Eating HRQOL (B = -0.84; P < 0.05) scores. Fewer eating problems were associated with higher CFQ-R Eating (B = -1.17; P < 0.0001) and Weight HRQOL (B = -0.78; P < 0.01) scores. As hypothesized, problematic eating and higher CF-specific stress was associated with lower CF-specific HRQOL. The CFQ-R has promise for use in young children with CF, but will need to be modified to exchange items not relevant to preschoolers with items that are more relevant to this age group.

Initial evaluation of the Parent Cystic Fibrosis Questionnaire—Revised (CFQ-R) in infants and young children

BackgroundThere is an urgent need to evaluate treatments for young children with cystic fibrosis (CF); however, efforts have been hampered by a lack of reliable, practical endpoints. To examine whether a patient-reported outcome could be reliable in children 4 to 60months of age, we assessed the psychometric properties of the modified Parent Cystic Fibrosis Questionnaire—Revised (CFQ-R) using data from the Infant Study of Inhaled Saline (ISIS). We also characterized patterns of symptom presentation and daily functioning in children in this age range to inform future measure development. MethodsParents (N=314) completed the CFQ-R and Treatment Adherence Questionnaire (TAQ) at five quarterly visits, as well as a weekly Parent Symptom Diary. ResultsThe Parent CFQ-R demonstrated good construct validity and adequate internal consistency (α's .58–.75). Associations with age, TAQ, and Parent Symptom Diary were observed. The Treatment Burden scale demonstrated responsiveness to change. ConclusionsParents were reliable observers of young children's symptoms and daily functioning, and PROs show promise for this age group. Research is needed to identify key symptoms in infants and young children with CF, and to develop a parent proxy PRO according to FDA/EMA guidelines.

Factors influencing quality of life and disease severity in Hungarian children and young adults with cystic fibrosis.

BackgroundThe aim of our study was to evaluate factors affecting cystic fibrosis (CF) patients’ health-related quality of life (HRQoL) and to assess the level of agreement on HRQol between children and their parents.MethodsFifty-nine patients (mean age: 14.03 ± 4.81 years) from 5 Hungarian CF centres completed the survey. HRQoL was measured using The Cystic Fibrosis Questionnaire-Revised (CFQ-R). Parents were asked to fill out a questionnaire about their smoking habits, educational level and history of chronic illness. Disease severity was assessed using the physician-reported Shwachman-Kulczycki (SK) score system. Spirometry, Body Mass Index (BMI) percentile (pc), hospitalisation and Pseudomonas aeruginosa (PA) infection were examined as physiologic parameters of CF, and the impact of these factors on HRQoL was assessed. A multivariate regression analysis was performed to identify the most important factors affecting HRQoL. The level of significance was set to 0.05.ResultsPassive smoking and parental educational level and chronic diseases status did not have a significant impact on the patients’ HRQoL (p > 0.05). Significantly lower SK scores and spirometry values were found in low BMI pc patients (p < 0.001), in hospitalised (p < 0.01) and in PA-infected patients (p < 0.01), than in the adequate-weight, non-hospitalised and PA culture-negative subgroup. Lower CFQ-R scores were detected in hospitalised patients than in non-hospitalised patients in their Physical functioning domain. PA-infected patients had HRQoL scores that were significantly worse in the Body image (p < 0.01) and Respiratory symptoms (p < 0.05) domains than the PA culture-negative patients. Patients with a low BMI pc (<25th BMI pc) had significantly lower scores in the Eating, Body image and Treatment burden domains, than the adequate-weight patients (>25th BMI pc) (p < 0.01). A strong child–parent agreement was found in the Physical functioning domain (r = 0.77, p < 0.01).ConclusionsPassive smoking, parental educational level and chronic diseases of parents do not affect the HRQoL of CF patients. In contrast, hospitalisation, PA infection and malnutrition have a significant and negative impact on patients’ HRQoL and the clinical severity of the disease. Parents and children were consistent in their scoring of symptoms and behaviours that were observable.

Pulmonary exacerbations and parent-reported outcomes in children

Pulmonary exacerbations (PEs) are used as clinical endpoints in infants and preschool children with cystic fibrosis (CF); however, their characteristics and impact in this age range are poorly understood. We used data from the Infant Study of Inhaled Saline, a multicenter trial of inhaled hypertonic versus isotonic saline, to describe PEs in children with CF <6 years and evaluate associations between PEs and parent-reported outcomes assessed by a weekly parent questionnaire (10 items) and three scales of the Cystic Fibrosis Questionnaire-Revised (CFQ-R), and other participant characteristics. There were 659 PEs among 253 of the 321 ISIS participants (mean age 2.3 years) during 287 participant-years follow-up. Of the 659 PEs, 636 (97%) were treated with oral and 45 (7%) with IV antibiotics (not mutually exclusive). Among 222 participants with PEs who had completed parent questionnaires during a PE and at baseline, 9 of the 10 symptoms were each present in a statistically significantly higher proportion of participants during a PE than at baseline. Lower (worse) baseline Respiratory Symptom and Physical Functioning CFQ-R scores were significantly associated with higher PE rate: rate ratio 1.08 (95%CI: 1.02, 1.14) and 1.21 (1.07, 1.36) per 10 point lower score in respective scale. A higher PE rate was also significantly associated with worse CFQ-R Respiratory Symptom and Physical Functioning scores at the end of the study, adjusted for baseline scores. Though most PEs did not require IV antibiotics, PEs appeared to have a negative impact on parent-reported health outcomes in infants and preschoolers with CF. Pediatr Pulmonol. 2015; 50:236-243. © 2014 Wiley Periodicals, Inc.

CF-CATS: An uncontrolled feasibility study of using tai chi for adults with cystic fibrosis

IntroductionCystic fibrosis (CF) can adversely affect quality of life. This study on tai chi (TC) for cystic fibrosis (CF) was an uncontrolled pilot trial assessing the feasibility of a future controlled trial. MethodsAdults with CF, recruited from the Royal Brompton Hospital, UK, were taught weekly TC (based on the “eternal spring” set) for 5 weeks in participants’ homes. They continued with DVD and home practice for 5 further weeks. Cystic fibrosis questionnaire revised (CFQ-R), hospital anxiety and depression scale and brief pain inventory were completed at baseline, 5 and 10 weeks. Healthcare use, TC practice, symptoms, and wellbeing were recorded weekly, medical data at baseline and 10 weeks. Data compared before and after TC. Two online focus groups explored experiences and feedback on research methods. ResultsEleven patients were recruited, mean age 38, five working/studying, baseline predicted FEV1 between 26% and 74%. Treatment constraints and respiratory symptoms (CFQ-R) significantly improved (mean decrease 27.78 (p=0.023) and 11.90 (p=0.05) respectively). Numerous health fluctuations were recorded.TC was perceived as a “powerful tool”, relaxing and calming. Challenges to TC practice included hospitalisation, poor health, other treatments and forgetting movements. The DVD was helpful. No problems participating were reported. ConclusionTC may reduce CF treatment impact, improve respiratory symptoms, self-efficacy and sleep. Participants’ poor and fluctuating health and the small sample potentially affected results. Such research appears appropriate and feasible. Suggestions for a future trial include measurements of quality of life, dypsnoea, self-efficacy, coping, sleep, exercise levels, longer follow up and frequent assessment.