Influences on indoor environmental trigger remediation uptake for children and young people with asthma: A scoping review.

INTRODUCTION: Children and young people (CYP) with asthma can benefit from reduced exposure to indoor environmental allergens and triggers but may not consistently have avoidance strategies implemented. To inform future interventions to increase trigger and allergen avoidance and enhance asthma control, a greater understanding of the influences on avoidance behaviours is necessary. METHODS: A systematic scoping review was selected to summarize evidence on what influences family uptake of indoor environmental asthma trigger avoidance strategies for CYP with asthma and identify research gaps. Primary studies of any design, including CYP (≤18 years) with asthma, and/or parent-carers, available in English and conducted since 1993, were eligible. Searches included nine databases, hand-searching reference lists and citation searching. FINDINGS: Thirty-three articles were included and are summarized narratively due to heterogeneity. Influences appear complex and multifactorial and include barriers to strategy uptake, health beliefs and personal motivation. Research specifically related to family understanding of allergic sensitisation status and exposure risks, and how these may inform avoidance implementation is required. Patient and public involvement (PPI) was not reported in included articles, although two studies used participatory methods. CONCLUSION: There is limited research on family asthma trigger management, particularly what influences current management behaviours. Variation in families' ability to identify important triggers, understand exposure risk and consistently reduce exposures warrants further exploratory research to explain how families reach avoidance decisions, and what future interventions should aim to address. Further PPI-informed research to address such gaps, could enable theory-based, person-centred interventions to improve the uptake of asthma trigger remediation. PATIENT OR PUBLIC CONTRIBUTION: An asthma-specific PPI group contributed to the decision-making for the funding for the wider project this review sits within. The findings of this scoping review have informed the subsequent phases of the project, and this was discussed with PPI groups (both adult and CYP groups) when proposing the next phases of the project.

Exploring the challenges of accessing medication for patients with cystic fibrosis.

Reducing treatment burden in cystic fibrosis (CF) is the top research priority for patients and clinicians. Difficulty accessing medication is one aspect of treatment burden. We investigated this with an online survey available globally for patients with CF and healthcare professionals. Almost three quarters of patients with CF in our survey report difficulty getting repeat prescriptions on time, and most community pharmacists experience interrupted supplies of CF-specific medications. These barriers affect emotional and physical health of people with CF. Two-thirds of people with CF would like to get all their CF medication from one place, their CF centre.

Changing landscape: psychological care in the era of cystic fibrosis transmembrane conductance regulator modulators.

PURPOSE OF REVIEW: The current review provides an overview of key psychological issues and challenges for the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator era of care. It discusses research from diagnosis and beyond, to patient-team communication with a particular focus on medical trials, adherence and living with CFTR modulators. RECENT FINDINGS: The impact of the diagnosis on parents is immense and the complexity of treatment now and in the future, are a challenge for both parents and teams. Communicating digitally is starting to become daily practice for many in CF care, with coronavirus disease 2019 accelerating this process. Participating in trials has a psychological impact, but most of all the (delayed) access and timing of accessing CFTR modulators is an important theme. Adherence remains of significance, both to 'old' and 'new' treatments. Living with CF in the era of CFTR modulators is beginning to impact on patients' quality of life, including new possibilities, opportunities and challenges. SUMMARY: Psychological care needs to engage and keep pace with the rapid medical changes. Some care priorities remain the same, including psychological screening and assessment, as well as psychoeducation, communication training and psychotherapy. The presence of CF psychologist in the CF clinic remains as important as ever.

Adherence to Aerosol Therapy in Young People With Cystic Fibrosis: Patient and Parent Perspectives Following Electronic Data Capture.

The benefits of improved treatments for cystic fibrosis (CF) depend on optimal adherence, which remains problematic, particularly to aerosol therapy. In this study, we explored the process of adhering to aerosol therapy from the perspective of both adolescents with CF and their parents. Interviews were conducted individually with six adolescents and six parents, informed by accurate adherence data from an electronically chipped, aerosol device. Interview transcripts from audio-recordings were analyzed using grounded theory method (GTM). Major themes revealed differences in perspective between parent and adolescent, with this relationship mediating the cognitive and emotional processes that play a significant role in adherence behavior. These processes are further influenced by interactions with the aerosol therapy treatment regimen, device characteristics, and the context in which adherence is taking place. Parents and adolescents have different views of treatment and how to manage it. Both need to be addressed if optimal adherence is to be achieved.

The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers.

There remain many treatment uncertainties in cystic fibrosis (CF). With limited resources, research should focus on questions which are most important to the CF community. We conducted a James Lind Alliance Priority Setting Partnership in CF. Research questions were elicited and then prioritised in successive surveys. A workshop agreed the final top 10. Online methods avoided cross infection and widened participation. The elicitation survey had 482 respondents (1080 questions) and prioritisation survey 677 respondents. Participants were drawn equally from the patient and clinical communities globally. We have achieved a consensus on 10 research priorities which will be attractive to funders.

Airway Clearance Techniques for Primary Ciliary Dyskinesia; is the Cystic Fibrosis literature portable?

Primary Ciliary Dyskinesia (PCD) is a rare inherited disease with impaired mucociliary clearance. Airway clearance techniques (ACTs) are commonly recommended for patients with PCD to facilitate mucus clearance, despite a lack of evidence in this group. Current physiotherapy practice in PCD is based on evidence extrapolated from the field of Cystic Fibrosis (CF). This paper focuses on the available evidence and outlines challenges in extrapolating evidence between the conditions for best clinical practice.

The neurological, neuroimaging and neuropsychological effects of playing professional football: Results of the UK five-year follow-up study.

BACKGROUND: Whilst the scientific understanding of mild traumatic brain injury sequelae has advanced, the consequences of neurological insults sustained during football play in the form of multiple concussions and heading remains unclear. METHOD: To the authors' knowledge, this is the first longitudinal prospective study to follow-up a group of footballers and controls over time. Thirty-two elite young professional footballers were recruited and 24 were identified at follow-up. Thirty-three controls were recruited and 17 identified at follow-up. Medical examination, MRI (brain) imaging and detailed neuropsychological data were collected on the footballers at baseline and 5-year follow-up. Medical examination and detailed neuropsychological data were collected on the controls at baseline and 5-year follow-up. RESULTS: All participants had normal neurological examination at both time points. At baseline, 37% of the footballers had sustained minor neurological insults. Between baseline and 5 years, 66% of the footballers had sustained minor neurological insults. No MRI (brain) abnormalities were identified among the footballers at either time point. Regarding the neuropsychology, there was a 6-point IQ difference between footballers and controls, with the footballers being low. Test-re-test analysis on a range of carefully selected neurocognitive tests revealed a picture of good stability in cognitive functioning over this 5-year period. CONCLUSIONS: These longitudinal prospective data indicate no significant neurological, structural brain imaging or neuropsychological change among a sample of young elite professional footballers over the first 5 years of their professional career.

Depression in cystic fibrosis; Implications of The International Depression/Anxiety Epidemiological Study (TIDES) in cystic fibrosis.

Children and adults with chronic diseases, as well as their parents, are at increased risk for depression. Where people with CF do exhibit psychological distress it is linked to poorer adherence and pulmonary function, increased hospitalisations and healthcare costs and decreased quality of life. The International Depression Epidemiological Study (TIDES) evaluated depression and anxiety in CF patients and parent caregivers across eight European countries and the USA. Two national and one international data sets have been published. This paper summarises the findings, offers explanations for differences in results, and outlines the clinical implications with consideration given to if and how recommendations could be integrated into managing CF in the UK.

Presenting life with cystic fibrosis: a Q-methodological approach to developing balanced, experience-based prenatal screening information.

BACKGROUND: Cystic fibrosis (CF) is one of the most common life-threatening genetically inherited conditions and prenatal screening for CF is available in many countries. Genetic counsellors and other health professionals are expected to provide information about the condition in a way that facilitates personal decision making. Knowing what information to deliver about complex genetic conditions to support informed screening decisions can be challenging for health professionals. OBJECTIVE: To solicit views from those with personal experience with CF on which aspects of the condition they consider most important to include in prenatal screening materials. METHODS: Q-methodology; an approach to systematically explore variations in viewpoint that combines factor analytic techniques with qualitative approaches to pattern interpretation. SETTING AND PARTICIPANTS: Twelve adults with CF and 18 parents of affected children were recruited from a regional centre in the UK. RESULTS: Five distinct viewpoints on the items most and least important to include in screening information were identified: Factor 1 the normality of life with CF and increasing life expectancy; Factor 2 the hardships and reduced lifespan. Factor 3 medical interventions and the importance of societal support. Factor 4 longer-term consequences of CF. Factor 5 the ability to adjust to the condition. DISCUSSION: The identification of five different views on what represented the most and least important information to include about CF highlights the challenge of portraying a complex genetic condition in a balanced and accurate manner. Novel ways in which Q-methodology findings can be used to meet this challenge are presented.

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 χ(2). 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.

Self-management education for cystic fibrosis.

BACKGROUND: Self-management education may help patients with cystic fibrosis and their families to choose, monitor and adjust treatment requirements for their illness, and also to manage the effects of illness on their lives. Although self-management education interventions have been developed for cystic fibrosis, no previous systematic review of the evidence of effectiveness of these interventions has been conducted. OBJECTIVES: To assess the effects of self-management education interventions on improving health outcomes for patients with cystic fibrosis and their caregivers SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register (date of the last search: 22 August 2013).We also searched databases through EBSCO (CINAHL; Psychological and Behavioural Sciences Collection; PsychInfo; SocINDEX) and Elsevier (Embase) and handsearched relevant journals and conference proceedings (date of the last searches: 01 February 2014 ). SELECTION CRITERIA: Randomised controlled trials, quasi-randomised controlled trials or controlled clinical trials comparing different types of self-management education for cystic fibrosis or comparing self-management education with standard care or no intervention. DATA COLLECTION AND ANALYSIS: Two authors assessed trial eligibility and risk of bias. Three authors extracted data. MAIN RESULTS: Four trials (involving a total of 269 participants) were included. The participants were children with cystic fibrosis and their parents or caregivers in three trials and adults with cystic fibrosis in one trial. The trials compared four different self-management education interventions versus standard treatment: (1) a training programme for managing cystic fibrosis in general; (2) education specific to aerosol and airway clearance treatments; (3) disease-specific nutrition education; and (4) general and disease-specific nutrition education. Training children to manage cystic fibrosis in general had no statistically significant effects on weight after six to eight weeks, mean difference -7.74 lb (i.e. 3.51 kg) (95% confidence interval -35.18 to 19.70). General and disease-specific nutrition education for adults had no statistically significant effects on: pulmonary function (forced expiratory volume at one second), mean difference -5.00 % (95% confidence interval -18.10 to 8.10) at six months and mean difference -5.50 % (95% confidence interval -18.46 to 7.46) at 12 months; or weight, mean difference - 0.70 kg (95% confidence interval -6.58 to 5.18) at six months and mean difference -0.70 kg (95% confidence interval -6.62 to 5.22) at 12 months; or dietary fat intake scores, mean difference 1.60 (85% confidence interval -2.90 to 6.10) at six months and mean difference 0.20 (95% confidence interval -4.08 to 4.48) at 12 months. There is some limited evidence to suggest that self-management education may improve knowledge in patients with cystic fibrosis but not in parents or caregivers. There is also some limited evidence to suggest that self-management education may result in positively changing a small number of behaviours in both patients and caregivers. AUTHORS' CONCLUSIONS: The available evidence from this review is of insufficient quantity and quality to draw any firm conclusions about the effects of self-management education for cystic fibrosis. Further trials are needed to investigate the effects of self-management education on a range of clinical and behavioural outcomes in children, adolescents and adults with cystic fibrosis and their caregivers.

Monitoring of ECFS quality standards for the clinical management of adults with cystic fibrosis.

BACKGROUND: Although cystic fibrosis (CF) standards of care have been produced and regularly updated, they are not specifically targeting at the adult population. The ECFS Standards of Care Project established an international task force of experts to identify quality standards for adults with CF and assess their adherence. METHODS: This study was composed of two phases. In the first one, a task force of international experts derived from published guidelines and graded ten quality standards for adult CF care using a modified Delphi methodology. In the second phase, an international audit was conducted among adult CF centers to retrospectively validate the quality statements and monitor adherence. RESULTS: The task force identified 10 quality standards specific to the care of adults with CF, mainly based on the 2018 ECFS standards of care. 14 adult CF centers participated in the audit, which showed that most quality standards for the management of CF in adults are met across Europe. Heterogeneity in adherence to standards was found across centers according to geographical setting and centers' characteristics. CONCLUSIONS: The identification of quality standards is a valuable resource for the standardization and monitoring of care delivery across centers taking care of adults with CF.

Standards of care for CFTR variant-specific therapy (including modulators) for people with cystic fibrosis.

Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF. The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators were becoming established as a novel therapy. We have produced interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF. Here we provide evidence-based guidance covering the spectrum of care, established using evidence from systematic reviews and expert opinion. Statements were reviewed by key stakeholders using Delphi methodology, with agreement (≥80%) achieved for all statements after one round of consultation. Issues around accessibility are discussed and there is clear consensus that all eligible people with CF should have access to variant-specific therapy.

Self-management education for cystic fibrosis.

BACKGROUND: Self-management education may help patients with cystic fibrosis and their families to choose, monitor and adjust treatment requirements for their illness, and also to manage the effects of illness on their lives. Although self-management education interventions have been developed for cystic fibrosis, no previous systematic review of the evidence of effectiveness of these interventions has been conducted. OBJECTIVES: To assess the effects of self-management education interventions on improving health outcomes for patients with cystic fibrosis and their caregivers SEARCH STRATEGY: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register (date of the last search: 23 February 2011).We also searched databases through EBSCO (CINAHL; Psychological and Behavioural Sciences Collection; PsychInfo; SocINDEX) and Elsevier (EMBASE) and handsearched relevant journals and conference proceedings (date of the last searches: 30th March 2011). SELECTION CRITERIA: Randomised controlled trials, quasi-randomised controlled trials or controlled clinical trials comparing different types of self-management education for cystic fibrosis or comparing self-management education with standard care or no intervention. DATA COLLECTION AND ANALYSIS: Two authors assessed trial eligibility and risk of bias. Three authors extracted data. MAIN RESULTS: Four trials (involving a total of 269 participants) were included. The participants were children with cystic fibrosis and their parents or caregivers in three trials and adults with cystic fibrosis in one trial. The trials compared four different self-management education interventions versus standard treatment: (1) a training programme for managing cystic fibrosis in general; (2) education specific to aerosol and airway clearance treatments; (3) disease-specific nutrition education; and (4) general and disease-specific nutrition education. Training children to manage cystic fibrosis in general had no statistically significant effects on weight after six to eight weeks, mean difference -7.74 lb (95% confidence interval -35.18 to 19.70). General and disease-specific nutrition education for adults had no statistically significant effects on: pulmonary function (forced expiratory volume at one second), mean difference -5.00 % (95% confidence interval -18.10 to 8.10) at six months and mean difference -5.50 % (95% confidence interval -18.46 to 7.46) at 12 months; or weight, mean difference - 0.70 kg (95% confidence interval -6.58 to 5.18) at six months and mean difference -0.70 kg (95% confidence interval -6.62 to 5.22) at 12 months; or dietary fat intake scores, mean difference 1.60 (85% confidence interval -2.90 to 6.10) at six months and mean difference 0.20 (95% confidence interval -4.08 to 4.48) at 12 months. There is some limited evidence to suggest that self-management education may improve knowledge in patients with cystic fibrosis but not in parents or caregivers. There is also some limited evidence to suggest that self-management education may result in positively changing a small number of behaviours in both patients and caregivers. AUTHORS' CONCLUSIONS: The available evidence from this review is of insufficient quantity and quality to draw any firm conclusions about the effects of self-management education for cystic fibrosis. Further trials are needed to investigate the effects of self-management education on a range of clinical and behavioural outcomes in children, adolescents and adults with cystic fibrosis and their caregivers.

Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials.

In a recent James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) the top priority clinical research question was: "What are effective ways of simplifying the treatment burden of people with CF?" We aimed to summarise the lived experience of treatment burden and suggest research themes aimed at reducing it. An online questionnaire was co-produced and responses subjected to quantitative and thematic analysis. 941 survey responses were received (641 from lay community). People with CF reported a median of 10 (interquartile range: 6-15) current treatments. Seven main themes relating to simplifying treatment burden were identified. Treatment burden is high, extending beyond time taken to perform routine daily treatments, with impact varying according to person-specific factors. Approaches to communication, support, evaluation of current treatments, service set-up, and treatment logistics (obtaining/administration) contribute to burden, offering scope for evaluation in clinical trials or service improvement.

Adherence to nebulised antibiotics in cystic fibrosis.

OBJECTIVE: To examine the adherence of patients with cystic fibrosis to nebulised antibiotics. METHODS: A longitudinal design with adherence data collected over 12 weeks. 38 patients (mean age 24.6 years, S.D. 5.3) were recruited from an adult cystic fibrosis (CF) clinic. Adherence was electronically monitored using a Prodose adaptive aerosol delivery (AAD) device. RESULTS: Three indices of adherence were calculated: mean percentage of times the nebuliser was used as prescribed was 50.0% (S.D.=39.7, range 1.1, 155.6); mean percentage of days fully adhered was 31.6% (S.D.=29.4, range 0, 97.2); mean percentage of days nebuliser used at least once was 57.1% (S.D.=34.2, range 3.3, 100). CONCLUSION: Rates of adherence were generally low. Adherence was not associated with any variables apart from age. There were wide variations between individuals, and differences in rates of adherence depending on how this was defined, with potentially important health consequences for the patients. PRACTICE IMPLICATIONS: Technology that improves medication administration may still be associated with low rates of adherence. Health professionals need to be mindful of the pattern of non-adherence for each individual, and factors which may be influencing this.

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.