Quality Of Life In Cystic Fibrosis Research Articles

465 Cystic fibrosis quality of life, symptom burden, and adherence to treatment regimens in the era of highly effective modulator therapies

465 Cystic fibrosis quality of life, symptom burden, and adherence to treatment regimens in the era of highly effective modulator therapies

Physical activity and liver disease affect the fat-free mass in adolescents with cystic fibrosis

Cystic fibrosis (CF) is predominantly a lung disease but is also characterised by impaired skeletal muscularity and a reduction in fat-free mass. We aimed to test the hypothesis that clinical and anthropometric parameters would determine fat-free mass impairment in adolescents with CF. We measured the fat-free mass index (FFMI) using bioelectrical impedance, the lung function using spirometry, the number of shuttles as a measure of exercise tolerance and the reported physical activity in children and young people with CF in a tertiary centre at King’s College Hospital, London, UK. CF-related liver disease was diagnosed by abnormal liver enzymes and/or ultrasonography. We studied 28 children and young people (11 male) with a median (interquartile range (IQR)) age of 15 (13–17) years. They had a median (IQR) FFMI of 13.5 (11.6–15.1) kg/m2. The FFMI significantly correlated with age (rho = 0.568, p = 0.002), number of shuttles (rho = 0.691, p < 0.001) and reported hours of activity per day (rho = 0.426, p = 0.024). The median (IQR) FFMI was significantly higher in male [15.1 (13.1–18.6) kg/m2] compared to female participants [12.7 (11.6–14.1) kg/m2, p = 0.008]. The median (IQR) FFMI was significantly lower in the 10 (36%) participants with liver disease [11.9 (11.5–13.4) kg/m2] compared to the FFMI in the remaining 18 participants without liver disease [14.4 (12.5–15.9) kg/m2, p = 0.027].Conclusion: Fat-free mass increases with increasing age and growth in adolescents with CF. Physical activity exerts a beneficial effect on fat-free mass, and CF-related liver disease negatively affects fat-free mass in adolescents with CF.What is Known:• Health behaviours in adolescence influence lifelong health in cystic fibrosis (CF).• A normal body mass index in CF might fail to reveal a low fat-free mass (FFM), and quality of life in CF is strongly associated with a reduced FFM.What is New:• FFM increases with increasing age and growth in adolescents with CF. • Physical activity exerts a beneficial effect, and liver disease negatively affects FFM in adolescents with CF.

P240 Elexacaftor/tezacaftor/ivacaftor treatment improves cystic fibrosis quality of life over multiple domains

P240 Elexacaftor/tezacaftor/ivacaftor treatment improves cystic fibrosis quality of life over multiple domains

Exploring the nature of perceived treatment burden: a study to compare treatment burden measures in adults with cystic fibrosis [version 1; peer review: 2 approved

Despite the importance of reducing treatment burden for people with cystic fibrosis (CF), it has not been fully understood as a concept. This study aims to quantify the treatment burden perceived by CF adults and explore the association between different validated treatment burden measures. This is a cross-sectional observational study of CF adults attending a single large UK adult center. Participants completed an online survey that contained three different treatment burden scales; CF Questionnaire-Revised (CFQ-R) subscale, CF Quality of Life (CFQoL) subscale, and the generic multimorbidity treatment burden questionnaire (MTBQ). Among 101 participants, the median reported treatment burden by the CFQ-R subscale was 55.5 (IQR 33.3 - 66.6), the CFQoL subscale was 66.6 (IQR 46.6 - 86.6), and the MTBQ reversed global score was 84.6 (IQR 73.1 - 92.3). No correlation was found between respondents' demographic or clinical variables and treatment burden measured via any of the three measures. All treatment burden measures showed correlations against each other. More treatments were associated with high treatment burden as measured by the CFQ-R, CFQoL subscales, and the MTBQ. However, longer treatment time and more complex treatment plans were correlated with high treatment burden as measured by the CFQ-R and CFQoL subscales, but not with the MTBQ. Treatment burden is a substantial issue in CF. Currently, the only available way to evaluate it is with the CF-specific quality of life measure treatment burden subscales (CFQ-R and CFQoL); both indicated that treatment burden increases with more treatments, longer treatment time, and more complex treatments.

The role of modulators in cystic fibrosis related diabetes

The development and introduction of modulator therapies have completely shifted the paradigm for the treatment of cystic fibrosis (CF). Highly effective modulator therapies have driven marked improvements in lung function, exacerbation rate, weight and quality of life in CF patients.However, their effect on CF related diabetes (CFRD) is not well delineated. The role of CF transmembrane conductance regulator (CFTR) in CFRD pathogenesis is inadequately understood and research aimed at deciphering the underlying mechanisms of CFRD continues to evolve. In this review, we summarize what is known regarding the effect of CFTR modulators on CFRD. Small studies using ivacaftor monotherapy in gating mutations have revealed improvement in insulin secretion, glucose tolerance and/or decrease in insulin requirement. However, lumacaftor/ivacaftor studies (primarily in delta F 508 homozygous) have not revealed significant improvement in CFRD or glucose tolerance. No studies are yet available regarding the effect of the highly effective triple therapy (elexacaftor/tezacaftor/ivacaftor) on CFRD or insulin secretion. CFTR modulators might affect development or progression of CFRD through many mechanisms including improving insulin secretion by correcting the CFTR defect directly, improving ductal function, reducing islet inflammation, and improving incretin secretion or by enhancing insulin sensitivity via reduced systemic inflammation and increased physical activity driven by improved lung function and quality of life. On the other hand, they can stimulate appetite and improve gastrointestinal function resulting in increased caloric intake and absorption, driving excessive weight gain and potentially increased insulin resistance. If the defect in insulin secretion is reversible then it is possible that initiation of CFTR modulators at a younger age might help prevent CFRD. Despite the advances in CF management, CFRD remains a challenge and knowledge continues to evolve. Future studies will drive better understanding of the role of highly effective CFTR modulators in CFRD.

Therapeutic effect of nebulized hypertonic saline for muco-obstructive lung diseases: a systematic review and meta-analysis with trial sequential analysis

Overproduction of mucus and impaired clearance play important roles in the pathogenesis of muco-obstructive lung diseases (MOLDs). This study aims to evaluate the therapeutic effect and safety of nebulized hypertonic...

Drug treatments for managing cystic fibrosis-related diabetes.

Drug treatments for managing cystic fibrosis-related diabetes.

The presence of Aspergillus fumigatus is associated with worse respiratory quality of life in cystic fibrosis

BackgroundThe clinical effects of Aspergillus fumigatus in the cystic fibrosis (CF) airway, with the exception of allergic bronchopulmonary aspergillosis, is unclear. MethodsCF adolescents and adults (age 14 years and older) underwent bacterial and semi-selective fungal culture testing to determine the prevalence of fungi in the CF respiratory tract and the independent association between the presence of Aspergillus fumigatus and clinical characteristics. ResultsAspergillus fumigatus (10.3%) and Candida species (57.8%) were the most common filamentous fungi and yeast seen respectively in the sputa of 206 individuals with CF. Inhaled corticosteroid (ICS) use was more common in Aspergillus fumigatus-positive than Aspergillus fumigatusnegative (100% versus 75.8%, p = .01). Aspergillus fumigatus was significantly associated with lower respiratory domain score (β −8.74, 95% CI −16.6, −0.88, p = .03), representing worse respiratory-related quality of life, accounting for demographics, disease characteristics, and the presence of a pulmonary exacerbation. ConclusionThe presence of Aspergillus fumigatus in CF sputum was associated with worse respiratory quality of life in CF in a crosssectional, single center study. Longitudinal analysis examining the clinical implications of Aspergillus fumigatus on respiratory health over time is needed.

Sleep disorders in patients with primary ciliary dyskinesia, cystic fibrosis with and without pancreatic insufficiency

Cystic fibrosis (CF) and primary ciliary dyskinesia (PCD) are characterized by impaired mucociliary clearance causing sinopulmonary infections and airway inflammation. However, they differ in many aspects including multisystemic involvement and disease severity. To compare sleep disorders and their correlation with quality of life (QOL) in three distinct patient populations: CF and pancreatic insufficiency (CF-PI), pancreatic sufficiency (CF-PS) and PCD. Patients completed age appropriate sleep quality questionnaires (SDSC, PSQI), quality of life questionnaires (PedQL, QOL-BE) and the Epworth sleepiness scale (ESS). Medical records were reviewed for clinical data. Eighty patients, 33 CF-PI, 27 CF-PS, and 20 PCD; 40 females; 41 adults and 39 children completed the study. In adults, 66% slept less than 7 h per night, and sleep quality was reduced in 26.5%, 53% reported night wakings and 47% nocturnal snoring or coughing. In children 78% slept more than 8 h, SDSC scores were mostly in the normal range. A significant correlation was found between sleep quality and QOL in both age groups. Global sleep scores were correlated with hemoglobin levels. Despite differences in disease severity and QOL, there were no significant differences in sleep quality between the three groups. Sleep disorders are common and correlate with QOL in CF and PCD. Despite differences in disease characteristics and severity, there are no differences in terms of sleep disorders between CF-PI, CF-PS and PCD patients, suggesting that they may represent indirect outcomes of disease.

Evaluation of quality of life predictors in adolescents and young adults with cystic fibrosis

BackgroundCystic fibrosis (CF) is a chronic disease that has an impact on Health-Related Quality of Life (HRQoL). ObjectivesTo identify demographic and clinical factors associated with HRQoL in adolescents and young adults with CF. MethodsThe sample comprised adolescent and young adult patients with CF. They completed the Cystic Fibrosis Quality of Life (CFQoL) questionnaire, which includes Physical, Social, Treatment, Chest Symptoms, Emotional Functioning, Future Concerns, Relationships, Body Image, and Career dimensions. We examined the relationships between gender, age, body weight, FEV1, pain, sleep, anxiety, depression and HRQoL. ResultsThe sample comprised 95 patients (aged 14–25 years; female/male: 43.1/56.8%). The lowest CFQoL score was observed in Future Concerns. FEV1 and body weight were positively associated with Physical Functioning (ß = 0.21; P < 0.01) and Body Image (ß = 0.30; P< 0.01), respectively. Females perceived themselves more negatively in Future Concerns (ß = −0.26; P< 0.01), Relationships (ß = −0.17; P< 0.01) and Career Concerns (ß = −0.20; P < 0.01) than males. Pain intensity (ß = −0.37), anxiety (ß = −0.39) and poor sleep quality (ß = −0.21) were negatively associated with global CFQoL (P < 0.001). ConclusionsPain intensity, anxiety and quality of sleep have the broadest impact on HRQoL. Regular assessment of psycho-emotional functioning, quality of sleep and pain intensity may improve a patient's well-being.

Respiratory muscle training improves respiratory muscle endurance but not exercise tolerance in children with cystic fibrosis.

Respiratory muscle endurance (RME) training has been shown to increase exercise endurance and lung function in adults with cystic fibrosis (CF). We conducted an interventional study to investigate the effectiveness of RME training on CF-related health outcomes in children. In a crossover trial, 22 children, aged 9-18 years, with CF performed 8 weeks of RME training and standard chest physiotherapy in a randomized sequence separated by a 1 week washout period. All children underwent training sessions using the RME training device before beginning the study. The primary outcomes were RME (in minutes) and exercise endurance (in minutes). Data were analyzed according to the intention-to-treat principle. Sixteen of 22 children (73%) completed the study. Study dropouts tended to be older with more advanced lung disease. After RME training, respiratory muscle endurance significantly increased by 7.03 ± 8.15 min (mean ± standard deviation, P < 0.001), whereas exercise endurance was unchanged by RME training (0.80 ± 2.58 min, P = 0.169). No significant improvement in secondary outcomes (lung function, CF quality of life, and CF clinical score) were observed. The small sample size and short intervention time have to be acknowledged as limitations of our study. RME training led to a significant increase in respiratory muscle endurance in children with CF. However, RME training did not improve exercise endurance or other CF-related health outcomes. Thus, our results do not support the routine use of RME training in the care of children with CF. Future studies in larger populations and with prolonged intervention time may overcome the limitations of our study. Pediatr Pulmonol. 2017;52:331-336. © 2017 Wiley Periodicals, Inc.

Quality of life in adults with cystic fibrosis: the Greek experience.

Recent developments in treatment have steadily raised the median predicted age of survival for people with Cystic Fibrosis (CF). We report the health-related quality of life (HRQoL) in CF adult patients and correlate our findings with the patients' demographic characteristics. The Cystic Fibrosis Quality of Life (CFQoL) questionnaire was answered by 77 CF adult patients. The questionnaire included questions pertaining to age, sex and level of education and covered eight sections of functioning. The highest score was reported in the "Social Functioning" section, while the lowest in the "Concerns for the Future" section. When different age groups were compared, statistical significances were reported in "Physical Functioning", "Interpersonal Relationships", and the "Career Concerns" section, with older patients reporting statistically higher HRQoL scores than younger ones (p < 0.005). No statistically significant difference was reported amongst the scoring between male and female CF patients. When different educational levels were compared, patients that had received a higher educational training scored statistically higher in all but one sections of the questionnaire when compared with patients of a lower educational level (p < 0.005). More than half Greek adult CF patients report that they are capable to participate in social activities but most of them are worried about the outcome of their disease and its effect on their lives.

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.

Relationship Between Clinical Variables and Health-Related Quality of Life in Young Adult Subjects With Cystic Fibrosis.

Health-related quality of life (HRQOL) has received much attention in patients with cystic fibrosis (CF). The goal of this study was to evaluate the association between clinical, lung function, sleep quality, and polysomnographic variables with 2 HRQOL questionnaires, the shorter-version World Health Organization Quality of Life (WHOQOL-BREF) and Cystic Fibrosis Quality of Life (CFQOL) questionnaires, in adult subjects with CF. In a cross-sectional study, 51 subjects underwent clinical evaluation and overnight polysomnography and answered WHOQOL-BREF, CFQOL, Pittsburgh Sleep Quality Index, and Epworth Sleepiness Scale questionnaires. In addition, pulmonary function tests, 6-min walk tests, and echocardiography were performed. For WHOQOL-BREF scores, the sleep quality index was associated with the physical domain; the percent-of-predicted 6-min walk distance (6MWD) and sleepiness scale were associated with the psychological domain; the percent-of-predicted FEV1 and sleep quality index were associated with the social relationship domain; and the sleep quality index was associated with the environment domain. For CFQOL scores, age at diagnosis, clinical score, and sleep quality index were associated with the physical functioning domain; the percent-of-predicted 6MWD and pulmonary arterial systolic pressure were associated with the role domain; sex and sleep quality index were associated with the vitality domain; the apnea-hypopnea index was associated with the emotional functioning domain; sex and body mass index (BMI) were associated with the body image domain; the percent-of-predicted 6MWD and sleep quality index were associated with the health perception domain; age, sex, BMI, and arousal index were associated with the weight domain; age, sex, percent-of-predicted FEV1, percent-of-predicted 6MWD, and pulmonary arterial systolic pressure were associated with the respiratory symptom domain; and the clinical score was associated with the digestive symptom domain. The sleep quality index score, 6MWD, sleepiness scale score, and FEV1 were predictors of WHOQOL-BREF scores. Age at diagnosis, clinical score, sleep quality score, 6MWD, sex, apnea-hypopnea index, BMI, current age, arousal index, FEV1, and pulmonary arterial systolic pressure were predictors of CFQOL scores.

Health-related quality of life of patients suffering from cystic fibrosis.

Cystic fibrosis (CF) is a particular example of a chronic disease with progressive course. Current statistical data shows that the life span of patients with CF has extended. Therefore, it is necessary to undertake interdisciplinary actions aiming at improving the efficiency of medical activities as well as minimalizing the influence of those interventions on the patients' quality of prolonged life. The aim of the study was to depict the QoL of patients suffering from mucoviscidosis and show the differences in the assessment of patients' QoL depending on age and sex. The study covered 30 patients with CF. Mean age of the respondent group was 24.83 ± 6.98; mean age of females 25.8 ± 7.27, whereas of males - 23.5 ± 6.56. A specific questionnaire for testing cystic fibrosis quality of life (CFQoL) of patients was used in the study. Generally patients with CF evaluated their QoL as low. In the study with the use of a CFQoL questionnaire they evaluated their QoL as the lowest in a subscale: future concerns. In most domains women got lower mean values than men. A dependence between patients' QoL and their age was ascertained. The older the patient is, the lower QoL is in the case of subscales (CFQoL): physical, emotional and social functioning, chest symptoms, interpersonal relationships, body image, career concerns. Patients with CF generally evaluate their QoL as low; there are differences in the evaluation of QoL between women and men. There is a relation between patients' QoL their age. The older the patient is, the worse his QoL in most subscales is.

What is the role of noninvasive ventilation in cystic fibrosis?

The use of noninvasive ventilatory support in patients with cystic fibrosis (CF) has increased exponentially over the past 2 decades. This review examines the current knowledge and considers potential future directions for use of noninvasive ventilation in CF patients. Noninvasive ventilation was originally reported as a bridge to transplantation in CF patients with severe respiratory failure but is now used as a long-term treatment modality for patients with respiratory failure independent of transplant status. In 2013 to 2014, over 400 publications on noninvasive ventilation demonstrate its increasing clinical application, however only seven reference CF. Recent technological advances and potential benefits to CF patients are considered. The role of noninvasive ventilation in CF patients in chronic respiratory failure is established, but future prospective studies are needed to determine further indications and optimal timing of this intervention. Developments in both ventilator and interface design may enhance the efficacy of ventilation in CF patients but require careful individualized assessment and regular review. The implications on treatment burden and quality of life in CF also need to be studied.

Determinants of physical activity participation in adults with cystic fibrosis

Introduction: Cystic Fibrosis (CF) is a complex, progressive, life-limiting disease predominantly affecting children and young adults. Regular participation in physical activity is strongly encouraged in those with CF as aerobic fitness has been shown to be an independent predictor of survival, and associated with improved quality of life (QOL). In children with CF anaerobic power, aerobic capacity, QOL and nutrition have been identified as predictors of physical activity participation. Following puberty, participation in physical activity declines in girls with CF.

The impact of nocturnal oxygen desaturation on quality of life in cystic fibrosis

Background Nocturnal oxyhaemoglobin desaturation is common in cystic fibrosis (CF) but the effect on quality of life (QoL) remains unknown. Methods Sixty stable CF outpatients with mean age 31 ± 8 years (mean ± 1SD), BMI 20.8 ± 3.2 kg/m 2 and FEV 1 42 ± 13% predicted had arterial blood gas sampling, lung function testing, overnight pulse oximetry and completed the CF QoL questionnaire, Epworth Sleepiness Scale and Medical Research Council dyspnoea scale. Results 11 (18%) of the CF patients were ‘desaturators,’ (SpO 2 < 90% for ≥ 30% recording time on overnight oximetry). Desaturators had greater difficulty performing their treatments (39 ± 22 vs 61 ± 26, p < 0.01) and more exertional dyspnoea (3.2 ± 0.8 vs 2.0 ± 0.9, p < 0.001) than non-desaturators after controlling for the effects of FEV 1, awake PaO 2 and PaCO 2 (adjusted p-values < 0.01 and 0.04 respectively). Conclusions Nocturnal oxyhaemoglobin desaturation is associated with impaired QoL, independent of the effects of lung function and awake gas exchange, in stable CF outpatients with moderate to severe lung disease.

Randomised placebo controlled trial of non-invasive ventilation for hypercapnia in cystic fibrosis

Background:The clinical benefits of domiciliary non-invasive positive pressure ventilation (NIV) have not been established in cystic fibrosis (CF). We studied the effects of nocturnal NIV on quality of life (QoL),...

Percepção de qualidade de vida de pessoas com fibrose cística: um estudo sobre a adequação dos instrumentos de medida

INTRODUÇÃO: Avaliações de qualidade de vida (QV) na fibrose cística (FC) são tão importantes quanto a avaliação da condição clínica do paciente. Porém, há divergências sobre conceitos e teorias de QV. METODOLOGIA: Onze pacientes brasileiros com FC maiores de 18 anos responderam a perguntas sugeridas pela OMS para a exploração conceitual da QV. A pesquisa foi orientada pela abordagem da grounded theory, e os dados sofreram análise qualitativa. RESULTADOS: Apesar da diversidade de domínios valorizados pelos entrevistados estar de acordo com o caráter multidimensional da QV, há domínios ainda não presentes nas escalas. CONCLUSÕES: Nenhuma escala atualmente disponível abrange todos os aspectos valorados pelos pacientes, o que aponta para a importância de questões idiossincráticas e culturais para a QV na FC. O esclarecimento da relação entre saúde e QV ainda depende de pesquisas futuras.