Longitudinal impact of demographic and clinical variables on health-related quality of life in cystic fibrosis.

OBJECTIVES: The insights that people with cystic fibrosis have concerning their health are important given that aspects of health-related quality of life (HRQoL) are independent predictors of survival and a decrease in lung function is associated with a decrease in HRQoL over time. Cross-sectional data suggest that key variables, other than lung function, are also associated with HRQoL--although study results are equivocal. This work evaluates the relationship between these key demographic and clinical variables and HRQoL longitudinally. DESIGN: Longitudinal observational study. Observations were obtained at seven time points: approximately every 2 years over a 12-year period. SETTING: Large adult cystic fibrosis centre in the UK. PARTICIPANTS: 234 participants aged 14-48 years at recruitment. OUTCOME MEASURE: Nine domains of HRQoL (Cystic Fibrosis Quality of Life Questionnaire) in relation to demographic (age, gender) and clinical measures (forced expiratory volume in 1 s, (FEV1)% predicted, body mass index (BMI), cystic fibrosis-related diabetes, Burkholderia cepacia complex, totally implantable vascular access device, nutritional and transplant status). RESULTS: A total of 770 patient assessments were obtained for 234 patients. The results of random coefficients modelling indicated that demographic and clinical variables were identified as being significant for HRQoL over time. In addition to lung function, transplant status, age, having a totally implantable vascular access device, cystic fibrosis-related diabetes, BMI and B. cepacia complex impacted on many HRQoL domains longitudinally. Gender was important for the domain of body image. CONCLUSIONS: Demographic and changes in clinical variables were independently associated with a change in HRQoL over time. Compared with these longitudinal data, cross-sectional data are inadequate when evaluating the relationships between HRQoL domains and key demographic and clinical variables, as they fail to recognise the full impact of the CF disease trajectory and its treatments on quality of life.

Longitudinal association between lung function and health-related quality of life in cystic fibrosis.

BACKGROUND: Lung function is an important indicator of cystic fibrosis disease status and those with better forced expiratory volume in 1 s (FEV(1))% predicted have tended to report a better health-related quality of life (HRQoL) in cross-sectional studies. The relationship between lung function and HRQoL over time is unknown. This work assesses the natural progression of HRQoL reporting over many years and compares assessments across a whole decade and evaluates the relationship between lung function and HRQoL longitudinally. METHODS: Demographic (age, gender), clinical (FEV(1)% predicted, body mass index, diabetes, Burkholderia cepacia complex, intravenous access device and nutritional status) and HRQoL (Cystic Fibrosis Quality of Life Questionnaire) variables were obtained every 2 years over a 12-year period (seven time points from 1998 to 2010). RESULTS: HRQoL and lung function declined slowly over time and significant decade changes were observed for FEV(1)% predicted and the nine domains of the Cystic Fibrosis Quality of Life Questionnaire. The results of random coefficient modelling indicated that, at the population level, decreasing FEV(1)% predicted was associated with decreasing HRQoL after adjusting for confounding variables. However, the percentage of patients for whom a decrease in lung function was associated with a decrease in HRQoL differed according to the quality of life domain. CONCLUSIONS: HRQoL and FEV(1)% predicted decline slowly; nevertheless, a decrease in lung function predicted a decrease in HRQoL over time.

Cystic fibrosis research in allied health and nursing professions.

This report is the result of the "Allied Health and Nursing Professions Working Group" meeting which took place in Verona, Italy, November 2009, which was organised by the European Cystic Fibrosis Society, and involved 32 experts. The meeting was designed to provide a "roadmap" of high priority research questions that can be addressed by Allied Health Professionals (AHP) and nursing. The other goal was to identify research skills that would be beneficial to AHP and nursing researchers and would ultimately improve the research capacity and capability of these professions. The following tasks were accomplished: 1) a Delphi survey was used to identify high priority research areas and themes, 2) common research designs used in AHP and nursing research were evaluated in terms of their strengths and weaknesses, 3) methods for assessing the clinimetric and psychometric properties, as well as feasibility, of relevant outcome measures were reviewed, and 4) a common skill set for AHPs and nurses undertaking clinical research was agreed on and will guide the planning of future research opportunities. This report has identified important areas and themes for future research which include: adherence; physical activity/exercise; nutritional interventions; interventions for the newborn with CF and evaluation of outcome measures for use in AHP and nursing research. It has highlighted the significant challenges AHPs and nurses experience in conducting clinical research, and proposes strategies to overcome these challenges. It is hoped that this report will encourage research initiatives that assess the efficacy/effectiveness of AHP and nursing interventions in order to improve the evidence base. This should increase the quality of research conducted by these professions, justify services they currently provide, and expand their skills in new areas, with the ultimate goal of improving care for patients with CF.

Symposium 6: Young people, artificial nutrition and transitional care. The nutritional challenges of the young adult with cystic fibrosis: transition.

Cystic fibrosis (CF) is a complex multisystem disorder affecting mainly the gastrointestinal tract and respiratory system. Intestinal malabsorption occurs in approximately 90% of patients. In the past, malnutrition was an inevitable consequence of disease progression, leading to poor growth, impaired respiratory muscle function, decreased exercise tolerance and immunological impairment. A positive association between body weight and height and survival has been widely reported. The energy requirements of patients with CF vary widely and generally increase with age and disease severity. For many young adults requirements will be 120-150% of the age-related estimated average requirement. To meet these energy needs patients are encouraged to eat a high-fat high-energy diet with appropriate pancreatic enzyme supplements. Many patients are unable to achieve an adequate intake as a result of a variety of factors including chronic poor appetite, infection-related anorexia, gastro-oesophageal reflux and abdominal pain. Oral energy supplements and enteral tube feeding are widely used. Nutritional support has been shown to improve nutritional status and stabilise or slow the rate of decline in lung function. With such emphasis on nutritional intake and nutritional status throughout life, poor adherence to therapies and issues relating to body image are emerging. The median survival of patients with CF is increasing. CF is now considered a life-limiting disease of adulthood rather than a terminal childhood illness. With increased longevity new challenges are emerging that include the transition of young adults with CF to adult services, CF-related diabetes, disordered eating, osteoporosis, liver disease and transplantation.

Decision-making about pregnancy for women with cystic fibrosis.

OBJECTIVES: This study aims to generate experiential data about the decision-making process encountered by 12 women with cystic fibrosis (CF) considering pregnancy. DESIGN: This study uses a qualitative grounded theory methodology to analyse the data and develop a conceptual hierarchical model of salient issues. METHODS: Twelve women completed a semi-structured interview accompanied by quantitative self-report measures of quality of life and psychological well-being, and indicators of health and treatment status were used. RESULTS: Descriptive data demonstrated that the sample did not differ significantly from other published samples. Four core categories emerged: (1) impact of the decision; (2) preparation for making and living with the decision; (3) owning the decision; and (4) personal dilemmas. Each comprised a number of overlapping conceptual and subcategories. Influencing factors included the impact of pregnancy on participants' health, and upon their ability to care for the child, the pregnancy experiences of other women with CF, the moral implications of having a child and the support and beliefs of family members. DISCUSSION: The findings extend the current knowledge in this area. We recommend development of guidelines for supporting and advising chronically ill young women about the psychosocial and emotional issues.

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.

Microvascular complications in patients with cystic fibrosis-related diabetes (CFRD).

BACKGROUND: This study aims to quantitatively and qualitatively assess microvascular complications and their risk factors in patients with cystic fibrosis-related diabetes (CFRD) compared to those with type 1 diabetes mellitus (DM1). METHODS: 79 patients with CFRD were matched with 79 patients with DM1 according to sex, age and duration of insulin therapy. Retinopathy, peripheral neuropathy, nephropathy and microalbuminuria were the microvascular complications assessed. Risk factors studied were: smoking, BMI, HbA1c, cholesterol, cholesterol/HDL ratio, diastolic and systolic blood pressure. RESULTS: Both groups had the same number of microvascular complications (29%). CFRD patients showed more microalbuminuria (21% versus 4.1%; p=0.003), while retinopathy was more common in patients with DM1 (24% versus 10%; p=0.044). The prevalences of peripheral neuropathy and nephropathy were similar. Patients with CFRD had lower BMI (p<0.0001), total cholesterol (p<0.0001) and HbA1c (p=0.056) levels, and a lower prevalence of smokers (p<0.0001). Cholesterol/HDL ratio and diastolic and systolic blood pressure were similar in both groups. CONCLUSIONS: The microvascular complications shown by patients with CFRD are similar to those seen in patients with DM1 but with a lower prevalence of retinopathy and a higher prevalence of microalbuminuria. The latter may reflect the influence of other cystic fibrosis-related factors on renal function.

Nutritional status, perceived body image and eating behaviours in adults with cystic fibrosis.

BACKGROUND & AIMS: Achieving and maintaining an ideal nutritional status is the primary aim of the nutritional management of cystic fibrosis (CF). It is unclear how nutritional interventions impact on patients' perceptions and behaviours concerning body image and eating. This work aimed to provide a psychosocial profile and compare CF patients receiving (a) enteral tube feeding, (b) nutritional supplements, (c) no nutritional interventions, and (d) healthy controls. METHODS: A cross-sectional questionnaire design was employed. Age, gender, lung function, and body mass index were recorded. Subjects completed measures of eating attitudes, perceived and desired body shape, body image, self-esteem and quality of life (QoL). RESULTS: A minority of CF patients reported disordered eating. Those receiving nutritional interventions engaged in less dieting behaviour. All CF groups, especially intervention groups, received more pressure from others to eat. For females, control groups desired to be slimmer whereas intervention groups desired to be heavier. Healthy males were content with their body whereas CF males wished to be heavier. Patients receiving enteral tube feeding were less satisfied with their body image, reported lower self-esteem and poorer QoL. CONCLUSION: Body image and eating behaviours are important considerations of nutritional interventions for maintaining QoL.