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

Developmental associations between victimization and body mass index from 3 to 10 years in a population sample

In the current prospective study, we investigated (1) whether high and low BMI in early childhood puts a child at risk of victimization by their peers, and (2) whether being victimised increases BMI over the short- and long-term, independent of the effect of BMI on victimization. We also examined whether gender moderated these prospective associations. Participants were 1344 children who were assessed yearly from ages 3 -10 years as part of the Québec Longitudinal Study of Child Development (QLSCD). BMI predicted annual increases in victimization for girls aged 6 years and over; for boys aged 7 and 8 years of age, higher BMI reduced victimization over the school year. Further, victimization predicted annual increases in BMI for girls after age 6 years. When these short-term effects were held constant, victimization was also shown to have a three and five-year influence on annual BMI changes for girls from age 3 years. These short- and long-term cross-lagged effects were evident when the effects of family adversity were controlled. The findings support those from previous prospective research showing a link between higher BMI and victimization, but only for girls. Further, being victimised increased the likelihood that girls would put on weight over time, which then increased future victimization. The implications of these prospective findings for interventions are considered

Initial development of the Psychopathic Processing and Personality Assessment (PAPA) across populations

Three studies describe development of the Psychopathic Processing and Personality Assessment (PAPA). Study one outlines a literature review and Expert Delphi (n = 32) to develop the initial PAPA. Study two validates the PAPA with 431 participants (121 male prisoners and 310 university students: 154 men, 156 women), also using the Levenson Self Report Psychopathy scale and a measure of cognitive schema and affect. Study three refined the PAPA, employing it with 50 male students and 40 male forensic psychiatric patients using clinical (interview) assessments of psychopathy: the Psychopathy Checklist – Screening Version and the Affect, Cognitive and Lifestyle assessment. The PAPA comprised four factors; dissocial tendencies; emotional detachment; disregard for others; and lack of sensitivity to emotion. It positively correlated with existing psychopathy measures. Variations across PAPA subscales were noted across samples when associated with clinical measures of psychopathy. Support for the validity of the PAPA was indicated across samples. Directions for research and application are outlined

Longitudinal and cross-sectional modelling of health related quality of life in people with cystic fibrosis

People with cystic fibrosis (CF) must endure up to four hours treatment per day to maintain health and are vulnerable to complications. The Cystic Fibrosis Quality of Life Questionnaire was developed to measure health related quality of life (HRQoL) in the UK. Most studies on HRQoL are cross-sectional in design with HRQoL measured once per patient. However, the Cystic Fibrosis Quality of Life Questionnaire has been used to monitor HRQoL longitudinally with measures taken over a 12 year period at one clinic in the UK. These data were modelled with a binomial distribution for a domain score and with fixed and random coefficients for the patient-level clinical and demographic variables. The longitudinal study included 182 patients whose HRQoL was first measured within a single calendar year and were then followed-up. These data provided an opportunity to compare, directly and by simulation, the modelling of a cross-sectional with the modelling of a longitudinal study and so provided insights into the statistical merits of longitudinal studies compared to cross-sectional studies in HRQoL

Communication of information about reproductive and sexual health in cystic fibrosis. Patients, parents and caregivers' experience

AbstractBackgroundThis review evaluated research concerning communication and information about reproductive and sexual health (RSH) in cystic fibrosis (CF).MethodsPapers in the English language reporting RSH issues in CF, published between January 2000 and December 2010, were included. The review focused on (a) the content of information given to parents and patients, (b) the timing of information, (c) the sources of information, (d) attitudes and emotional reactions, (e) chronic illness and sexual behavior, (f) methodological and cultural considerations and (g) ethical considerations.ResultsEleven papers were identified originating from Australia, the United Kingdom and Poland. Patients and parents expressed the need for up-to-date, CF-related verbal and written information, provided by the CF team and infertility specialists. Patients were often embarrassed to initiate a discussion. Health care providers expressed the need for training to counsel patients in RSH.ConclusionA pro-active discussion of RSH issues is proposed as a standard part of the care-pathway. A list of recommendations is given to initiate this process

Adherence to Ivacaftor is suboptimal

Ivacaftor, the first cystic fibrosis (CF) transmembrane conductance regulator (CFTR) potentiator provides a remarkable example of personalised medicine that has the capacity to transform lives and patient care. For those with a CFTR-G551D mutation, two phase three trials (adult and paediatric age 6+ years) and a follow-up open label trial have demonstrated remarkable and sustained improvements in CFTR function, FEV1 (approximately 10% points) and a reduction in pulmonary exacerbations with Ivacaftor 150 mg every 12 h [1–3]

Implementing the International Committee on Mental Health in Cystic Fibrosis (ICMH) Guidelines: Screening accuracy and referral-treatment pathways

Background: The International Committee on Mental Health (ICMH) published screening guidelines in Cystic Fibrosis (CF). This work 1) evaluated the sensitivity of the recommended screening tools against the ‘gold standard’ clinical psychological assessment and 2) investigated referral and treatment pathways. Methods: Ninety-six participants (79 caregivers; 17 adolescents with CF) completed the screening tools prior to formal assessment. Agreement between screening data and psychological assessment was evaluated, sensitivity analyses performed and referral pathways tracked. Results: All participants with an elevated screen (moderate/severe range) were subsequently assessed as requiring treatment for major depression/anxiety disorders. However, many were referred for treatment without elevated scores. Hence, sensitivity was poor with the recommended threshold score of 10, but with a threshold of 5 the sensitivity was 76% for adults and 46% for adolescents. The area under the ROC curve (diagnostic test ability) was 0.89 for caregivers but lower at 0.68 for adolescents. Conclusion: Mental health screening is complex, particularly in adolescents. Nonetheless, it is a first valuable step to improve mental health care in CF. The need for psychological support is greater than anticipated by the TIDES study

Indicators of pulmonary exacerbation in cystic fibrosis: A Delphi survey of patients and health professionals

Background: There is uncertainty about the most important indicators of pulmonary exacerbations in CF. Methods: Two parallel Delphi surveys in 13 CF centres (UK and Ireland). Delphi 1: 31 adults with CF, ≥ one exacerbation over 12 months. Delphi 2: 38 CF health professionals. Rounds 1 and 2 participants rated their level of agreement with statements relating to indicators of exacerbation; Round 3 participants rated the importance of statements which were subsequently placed in rank order. Results: Objective measurements were of higher importance to health professionals. Feelings of increased debility were rated most important by adults with CF. Conclusions: There were clear differences in perspectives between the two groups as to the most important indicators of an exacerbation. This highlights that CF health professionals should take more cognisance of specific signs and symptoms reported by adults with CF, especially since these may be a precursor to an exacerbation

Understanding the sleep-aggression relationship in a forensic mental health sample

The contribution of cognition to the sleep-aggression relationship is explored via three connected studies, involving adult male forensic patients detained in a high secure hospital. Study 1 included 31 patients, interviewed to examine their experiences of specific sleep problems. In Study 2, 42 patients completed a series of measures examining sleep dysfunction, aggression, and cognition, while Study 3 was designed to impact on sleep via a cognitive approach. In the latter, 48 patients were randomly assigned as part of a feasibility trial to one of three conditions: mindfulness (cognitive approach), sleep education, and treatment as usual. Collectively, the studies demonstrated the multifaceted nature of cognition in the sleep-aggression relationship, with a need to account fully for cognitive factors. A preliminary conceptual model is outlined - the Cognitive Sleep Model for Aggression and Self Harm (CoSMASH), as a direction for future research to consider