Janice Abbott

Development of a disease specific health related quality of life measure for adults and adolescents with cystic fibrosis

BACKGROUND Health related quality of life (HRQoL) measurement is important in determining the impact of disease on daily functioning and subsequently informing interventions. In cystic fibrosis (CF) generic HRQoL measures have been employed but these may not be sufficiently specific. The aim of the current work was to develop and validate a disease specific HRQoL measure for adults and adolescents with cystic fibrosis. METHODS Areas of concern to adults and adolescents with CF were identified by unstructured interviews, self-administered questionnaires, consultation with multidisciplinary specialist staff, a review of the relevant literature, and examination of other HRQoL measures. Items for the questionnaire were generated on the basis of this process. Continued evaluation and development of the Cystic Fibrosis Quality of Life (CFQoL) questionnaire was undertaken by a process of statistical analysis and continued feedback from patients. The full testing and validation of the CFQoL questionnaire took place over four phases: (1) initial item generation and testing of a preliminary questionnaire, (2) testing and validation of the second version of the questionnaire, (3) test-retest reliability of a third and final version of the questionnaire, and (4) sensitivity testing of the final version of the questionnaire. RESULTS Nine domains of functioning were identified using principal components analysis with varimax rotation. Internal reliability of the identified domains was demonstrated using Cronbach alpha coefficients (range 0.72–0.92) and item to total domain score correlations. Concurrent validity (ranger = 0.64–0.74), discriminatory ability between different levels of disease severity, sensitivity across transient changes in health (effect size range, moderate d = 0.56 to large d = 1.95), and test-retest reliability (r = 0.74–0.96) were also found to be robust. CONCLUSIONS The CFQoL questionnaire is a fully validated disease specific measure consisting of 52 items across nine domains of functioning which have been identified by, and are of importance to, adolescents and adults with cystic fibrosis. This measure will be useful in clinical trials and longitudinal studies.

Prevalence of depression and anxiety in patients with cystic fibrosis and parent caregivers: results of The International Depression Epidemiological Study across nine countries

Background Individuals with chronic diseases and parent caregivers are at increased risk for symptoms of depression and anxiety. Prevalence of psychological symptoms was evaluated in adolescents and adults with cystic fibrosis (CF) and parent caregivers across nine countries. Methods Patients with CF, ages 12 years and older, and caregivers of children with CF, birth to18 years of age, completed measures of depression and anxiety across 154 CF centres in Europe and the USA. Psychological symptoms were compared across countries using χ2. Logistic regression examined extent of comorbid symptoms, predictors of depression and anxiety, and concordance between parent and adolescent symptomatology. Results Psychological symptoms were reported by 6088 patients with CF and 4102 parents. Elevated symptoms of depression were found in 10% of adolescents, 19% of adults, 37% of mothers and 31% of fathers. Elevations in anxiety were found in 22% of adolescents, 32% of adults, 48% of mothers and 36% of fathers. Overall, elevations were 2–3 times those of community samples. Participants reporting elevated anxiety were more likely to report depression (ORs: adolescents=14.97, adults=13.64, mothers=15.52, fathers=9.20). Significant differences in reports of depression and anxiety were found by patient age and parent respondent. Concordance between 1122 parent–teen dyads indicated that adolescents whose parents reported depression were more likely to be elevated on depression (OR=2.32). Similarly, adolescents whose parents reported anxiety were more likely to score in the elevated range on the anxiety measure (OR=2.22). Conclusions Symptoms of depression and anxiety were elevated in both patients with CF and parents across several European countries and the USA. Annual screening of psychological symptoms is recommended for both patients and parents.

A cost description of an adult cystic fibrosis unit and cost analyses of different categories of patients.

BACKGROUND: There is little information on the costs of running an adult cystic fibrosis centre. The aim of this study was to provide detailed costs to assist funding and planning for these patients. METHODS: The cost of a regional adult cystic fibrosis centre serving 119 cystic fibrosis patients, categorised according to four treatment regimens, was determined. District health authority, family health service authority, and voluntary resources used from April 1989 to March 1990 were determined, with appropriate bases for allocation of costs and patient based costs from local information. RESULTS: The total annual cost of treating the 119 patients was 980,646 pounds, with an average cost 8241 pounds per patient. An outpatient reviewed at three monthly intervals cost 2792 pounds a year; an outpatient receiving intravenous antibiotics cost 8606 pounds; an inpatient receiving intravenous antibiotics cost 13,501 pounds; and a patient needing a high level of care cost 19,955 pounds. Medication accounted for 57% (561,395 pounds) of the total cost. CONCLUSIONS: This analysis has helped us to secure funding for patients with cystic fibrosis and it facilitates the prediction of future requirements. The study also indicates the limitations of using average patient costs and difficulties as a result of the poorly structured British National Health Service accounting and information systems.

International Committee on Mental Health in Cystic Fibrosis: Cystic Fibrosis Foundation and European Cystic Fibrosis Society consensus statements for screening and treating depression and anxiety

Studies measuring psychological distress in individuals with cystic fibrosis (CF) have found high rates of both depression and anxiety. Psychological symptoms in both individuals with CF and parent caregivers have been associated with decreased lung function, lower body mass index, worse adherence, worse health-related quality of life, more frequent hospitalisations and increased healthcare costs. To identify and treat depression and anxiety in CF, the CF Foundation and the European CF Society invited a panel of experts, including physicians, psychologists, psychiatrists, nurses, social workers, a pharmacist, parents and an individual with CF, to develop consensus recommendations for clinical care. Over 18 months, this 22-member committee was divided into four workgroups: Screening; Psychological Interventions; Pharmacological Treatments and Implementation and Future Research, and used the Population, Intervention, Comparison, Outcome methodology to develop questions for literature search and review. Searches were conducted in PubMed, PsychINFO, ScienceDirect, Google Scholar, Psychiatry online and ABDATA by a methodologist at Dartmouth. The committee reviewed 344 articles, drafted statements and set an 80% acceptance for each recommendation statement as a consensus threshold prior to an anonymous voting process. Fifteen guideline recommendation statements for screening and treatment of depression and anxiety in individuals with CF and parent caregivers were finalised by vote. As these recommendations are implemented in CF centres internationally, the process of dissemination, implementation and resource provision should be closely monitored to assess barriers and concerns, validity and use.

Ways of coping with cystic fibrosis: implications for treatment adherence

Purpose: How individuals cope with aspects of cystic fibrosis (CF) has the potential to influence their self management and the course of their disease. To evaluate how individuals cope with CF, a disease specific coping scale was developed and validated. A second objective of the work was to examine the relationship between coping styles and treatment adherence. Methods: The development of the coping scale constituted a longitudinal design. A cross-sectional questionnaire design was used to examine the coping-adherence relationship. The development and validation of the coping scale comprised three phases: (1) Initially, 60 patients were interviewed to identify CF concerns. From this information a list of 23 concerns were recorded; (2) Eighty-three patients were interviewed to identify CF coping responses. For each concern, they were asked what they did or thought to ease the worry. A list of 24 coping strategies were recorded that formed a comprehensive set of items as to how people with CF act, feel and think about aspects of their disease; and (3) Further development and testing of the questionnaire involved 174 patients completing the measure. Four distinct ways of coping with CF were identified by factor analysis. These were termed optimistic acceptance, hopefulness, distraction and avoidance. The cronbach alpha coefficients were 0.74 (optimistic acceptance), 0.69 (hopefulness), 0.71 (distraction) and 0.76 (avoidance). To evaluate the relationship between coping and treatment adherence 60 patients completed the CF Coping Questionnaire and the Manchester Adult Cystic Fibrosis Compliance Questionnaire. Results: Compared with patients who were non-adherent, those who were adherent scored higher on the optimistic acceptance scale (physiotherapy p < 0.05, enzymes p < 0.003, vitamins p < 0.05) and hopefulness scale (physiotherapy p < 0.002, enzymes p < 0.001). Those who were partially adherent reported using distraction as a way of coping to a greater extent than adherent or non-adherent patients (all ps 0.05). Nonadherent patients used avoidance strategies to a greater extent than those who were adherent (physiotherapy p < 0.05, enzymes p < 0.04), although interestingly, adherence with exercise was associated with avoidant coping (p < 0.004). Conclusions: The degree of adherence to treatments was influenced by a persons style of coping. The identification of effective coping strategies to aid both long-term psychological and clinical well-being should improve the management of nonadherence.PURPOSE How individuals cope with aspects of cystic fibrosis (CF) has the potential to influence their self management and the course of their disease. To evaluate how individuals cope with CF, a disease specific coping scale was developed and validated. A second objective of the work was to examine the relationship between coping styles and treatment adherence. METHODS The development of the coping scale constituted a longitudinal design. A cross-sectional questionnaire design was used to examine the coping-adherence relationship. The development and validation of the coping scale comprised three phases: (1) Initially, 60 patients were interviewed to identify CF concerns. From this information a list of 23 concerns were recorded; (2) Eighty-three patients were interviewed to identify CF coping responses. For each concern, they were asked what they did or thought to ease the worry. A list of 24 coping strategies were recorded that formed a comprehensive set of items as to how people with CF act, feel and think about aspects of their disease; and (3) Further development and testing of the questionnaire involved 174 patients completing the measure. Four distinct ways of coping with CF were identified by factor analysis. These were termed optimistic acceptance, hopefulness, distraction and avoidance. The cronbach alpha coefficients were 0.74 (optimistic acceptance), 0.69 (hopefulness). 0.71 (distraction) and 0.76 (avoidance). To evaluate the relationship between coping and treatment adherence 60 patients completed the CF Coping Questionnaire and the Manchester Adult Cystic Fibrosis Compliance Questionnaire. RESULTS Compared with patients who were non-adherent, those who were adherent scored higher on the optimistic acceptance scale (physiotherapy p < 0.05, enzymes p < 0.003, vitamins p < 0.05) and hopefulness scale (physiotherapy p < 0.002, enzymes p < 0.001). Those who were partially adherent reported using distraction as a way of coping to a greater extent than adherent or non-adherent patients (all ps < 0.05). Non-adherent patients used avoidance strategies to a greater extent than those who were adherent (physiotherapy p < 0.05, enzymes p < 0.04), although interestingly, adherence with exercise was associated with avoidant coping (p < 0.004). CONCLUSIONS The degree of adherence to treatments was influenced by a persons style of coping. The identification of effective coping strategies to aid both long-term psychological and clinical well-being should improve the management of non-adherence.

Contemporary psychosocial issues in cystic fibrosis: treatment adherence and quality of life

This review focuses on the two major psychosocial areas currently topical in cystic fibrosis research: treatment adherence and quality of life. First, the paper discusses the importance of evaluating adherence to treatments and medical advice, its measurement, predictors of adherence and non-adherence and whether the data should be used to inform clinical practice and policy decisions. Second, the purpose of evaluating quality of life in cystic fibrosis and an overview of the data are discussed. Both areas have suffered from problems with terminology and definition, inappropriate methodologies and hence difficulties with data interpretation. Future directions for research are advocated.

Health-related quality of life in adults with cystic fibrosis: The role of coping

OBJECTIVE This study examined the role of coping in explaining health-related quality of life (HRQoL) in cystic fibrosis (CF). METHODS Coping, quality of life, and clinical variables were assessed. The CF Coping Scale measures four ways of coping: optimistic acceptance, hopefulness, distraction, and avoidance. The Cystic Fibrosis Quality of Life Questionnaire (CFQoL) comprises 9 domains: physical, social, treatment, chest symptoms, emotional, body image, relationships, career, and future. Step-wise multiple regression and path analysis were used to model the associations between coping and CFQoL domains, taking into account important demographic and clinical factors. RESULTS One hundred and sixteen people with CF were recruited to the study. Their mean FEV(1)% predicted and BMI were 59.3% and 21.2 kg/m(2), respectively. Mean scores were highest for optimism and lowest for distraction coping. Coping had an inequitable influence across the CFQoL domains: it had negligible influence on domains that incorporate symptoms and aspects of physical functioning but considerable influence on psychosocial domains. Optimism and distraction were strongly associated with emotional responses, social functioning, and interpersonal relationships. A high level of optimism was associated with a better HRQoL and high levels of distraction with a poorer HRQoL. For some domains, optimism and distraction had a counterbalancing effect so the difference between them could be an important predictor of HRQoL. CONCLUSION Coping has emerged as an important factor in explaining some quality-of-life domains but not others. This has important implications especially when employing HRQoL as an outcome measure in clinical trials.

Measuring and reporting quality of life outcomes in clinical trials in cystic fibrosis: a critical review

Good quality clinical trials are essential to inform the best cystic fibrosis (CF) management and care, by determining and comparing the effectiveness of new and existing therapies and drug delivery systems. The formal inclusion of quality of life (QoL) as an outcome measure in CF clinical trials is becoming more common. Both an appropriate QoL measure and sound methodology are required in order to draw valid inferences about treatments and QoL. A review was undertaken of randomised controlled trials in cystic fibrosis where QoL was measured. EMBASE, MEDLINE and ISI Web of Science were searched to locate all full papers in the English language reporting randomised controlled trials in cystic fibrosis, published between January 1991 and December 2004. All Cochrane reviews published before December 2004 were hand searched. Papers were included if the authors had reported that they had measured QoL or well being in the trial. 16 trials were identified. The interventions investigated were: antibiotics (4); home versus hospital administration of antibiotics (1); steroids (1); mucolytic therapies (6); exercise (3) and pancreatic enzymes (1). Not one trial evaluated in this review provided conclusive results concerning QoL. This review highlights many of the pitfalls of QoL measurement in CF clinical trials and provides constructive information concerning the design and reporting of trials measuring QoL.

Can Health-related Quality of Life Predict Survival in Adults with Cystic Fibrosis?

RATIONALE Advances in the management of cystic fibrosis have led to a significant improvement in survival, although marked differences between individuals are still observed. The value of patient-reported health-related quality of life scores in predicting survival in adults with cystic fibrosis is unknown. OBJECTIVES To evaluate whether patient-reported health-related quality of life could predict survival in cystic fibrosis. METHODS From 1996 to 1997 a consecutive series of 223 patients were recruited to evaluate the Cystic Fibrosis Quality of Life Questionnaire. Demographic (age, sex), clinical (FEV(1)% predicted, body mass index, diabetes, B. Cepacia complex, intravenous access device, nutritional and lung transplant status) and health-related quality of life variables were recorded (Cystic Fibrosis Quality of Life Questionnaire and the SF-36). These data were used as baseline measures to explore the prognostic association of health-related quality of life and subsequent survival. MEASUREMENTS AND MAIN RESULTS At the census date (December 31, 2006) 154 (69.1%) adults were alive, 66 (29.6%) had died, and three (1.3%) were lost to follow-up. Cox proportional hazards models and bootstrapping procedures examined if health-related quality of life domains predicted survival after adjusting for the demographic and clinical factors. The physical functioning domain of the Cystic Fibrosis Quality of Life Questionnaire and the pain domain of the Short Form-36 had the strongest statistical associations with survival. CONCLUSIONS Aspects of patient-reported quality of life serve as prognostic measures of survival beyond a number of previously known factors in cystic fibrosis. This needs to be investigated further in a larger longitudinal study.

Validation of the SF-36 for the assessment of quality of life in adolescents and adults with cystic fibrosis

BACKGROUND Generic health-related quality of life measures are often applied to disease groups without assessment of their psychometric properties. The current work assesses the properties of the Short Form 36-item (SF-36) questionnaire in a British sample of adolescents and adults with cystic fibrosis (CF). METHODS Two hundred and twenty-three adolescents and adults with CF completed the SF-36 with a further 185 approached and not responding by non-completion of the questionnaire. The structure and internal reliability of the instrument was assessed by principal components analysis, Cronbach alpha coefficients and item to domain correlations. Differences between disease severity groups were assessed by analysis of variance. RESULTS Factor analysis of the SF-36 scores broadly confirmed domain structures for the SF-36. Cronbach alpha coefficients were high (range 0.82-0.91) and item-to-same domain correlations were stronger than item-to-unrelated domain correlations. Examination of differences between mild, moderate and severe disease states revealed four significant main effects for: physical functioning, role limitation due to physical functioning, general health perceptions and energy and vitality. The analysis also revealed the presence of numerous ceiling effects across domains. CONCLUSIONS The domain structure of the SF-36 was demonstrated to be robust. However, the discriminatory ability of the measure was disappointing. The presence of ceiling effects and the low frequency of differences between intermediate disease severity groups indicated that the SF-36 was not discriminatory with respect to mild disease states or progression of illness.