Economic evaluation of a trial exploring the effects of a web-based support tool for parents of children with juvenile idiopathic arthritis.

OBJECTIVE: To explore the cost-effectiveness of a web-based support tool for parents of children with Juvenile Idiopathic Arthritis. METHODS: A multi-centred randomised controlled trial was conducted in paediatric rheumatology centres in England. The WebParC intervention consisted of online information about JIA and its treatment and a toolkit using cognitive-behavioural therapy principles to support parents manage their child's JIA. An economic evaluation was performed alongside the trial involving 220 parents. The primary outcome was the self-report Pediatric Inventory for Parents measure of illness-related parenting stress, with two dimensions; difficulty and frequency. These measures along with costs were assessed post intervention at 4 months and 12 months. Costs were calculated for healthcare usage using a UK NHS economic perspective. Data was collected and analysed on the impact of caring costs on families. Uncertainty around cost effectiveness was explored using bootstrapping and cost-effectiveness acceptability curves. RESULTS: The intervention arm showed improved average Pediatric Inventory for Parents scores for the dimensions of frequency and difficulty, of 1.5 and 3.6 respectively at 4 months and. 0.35 and 0.39 at 12 months, representing improved PIP scores for the intervention arm. At both 4 and 12 month follow up the average total cost per case was higher in the control group when compared with the intervention arm with mean differences of £360 (95% CI £29.6 to £691) at 4 months and £203 (95% CI £16 to £390) at 12 months. The probability of the intervention being cost effective ranged between 49% and 54%. CONCLUSION: The WebParC intervention led to reductions in primary and secondary healthcare resource use and costs at 4 and 12 months. The intervention demonstrated particular savings for rheumatology services at both follow ups. Future economies of scale could be realised by health providers with increased opportunities for cost effectiveness over time. TRIAL REGISTRATION: ISRCTN, ISRCTN13159730.

A mixed model approach to estimate the survivor average causal effect in cluster-randomized trials.

In many medical studies, the outcome measure (such as quality of life, QOL) for some study participants becomes informatively truncated (censored, missing, or unobserved) due to death or other forms of dropout, creating a nonignorable missing data problem. In such cases, the use of a composite outcome or imputation methods that fill in unmeasurable QOL values for those who died rely on strong and untestable assumptions and may be conceptually unappealing to certain stakeholders when estimating a treatment effect. The survivor average causal effect (SACE) is an alternative causal estimand that surmounts some of these issues. While principal stratification has been applied to estimate the SACE in individually randomized trials, methods for estimating the SACE in cluster-randomized trials are currently limited. To address this gap, we develop a mixed model approach along with an expectation-maximization algorithm to estimate the SACE in cluster-randomized trials. We model the continuous outcome measure with a random intercept to account for intracluster correlations due to cluster-level randomization, and model the principal strata membership both with and without a random intercept. In simulations, we compare the performance of our approaches with an existing fixed-effects approach to illustrate the importance of accounting for clustering in cluster-randomized trials. The methodology is then illustrated using a cluster-randomized trial of telecare and assistive technology on health-related QOL in the elderly.

Using the behaviour change wheel and person-based approach to develop a digital self-management intervention for patients with adrenal insufficiency: the Support AI study protocol.

Introduction: Most patients with Adrenal insufficiency (AI) require lifelong glucocorticoid replacement. They need to increase glucocorticoids during physical illness or major stressful situations and require parenteral hydrocortisone in the event of an adrenal crisis. Patients with AI have impaired quality of life and high mortality; approximately 1 in 6-12 patients are hospitalised at least once/year from a potentially preventable adrenal crisis. Adoption of self-management behaviours are crucial; these include adherence to medication, following "sick day rules" and associated behaviours that aid prevention and treatment of adrenal crisis such as symptom monitoring, having extra tablets, carrying a medical-alert ID and injection kit, and self-injecting when necessary. Current patient education is ineffective at supporting self-management behaviour change or reducing adrenal crisis-related hospitalisations. This research study aims to gain an in-depth understanding of the barriers and enablers to self-management for patients with AI and to develop an evidence-based digital self-management behaviour change intervention. Methods: The study is conducted in accordance with the MRC Framework for developing complex interventions. Underpinned by the Behaviour Change Wheel (BCW), the Theoretical Domains Framework (TDF), and the Person-Based Approach, this research will be conducted in two phases: Phase 1 will involve a sequential qualitative/quantitative mixed-methods study involving focus group interviews followed by a cross-sectional survey with patients with AI recruited from patient advocacy groups and endocrine clinics in the UK. Phase 2 will develop the Support AI, a website-based digital behaviour change intervention (DBCI) informed by Phase 1 findings to support self-management for patients with AI. The most appropriate behaviour change techniques (BCTs) will be selected utilising a nominal group technique with an Expert Panel of 10-15 key stakeholders. The design of the Support AI website will be guided by the Person-Based Approach using an Agile iterative "think-aloud" technique with 12-15 participants over 3 usability testing iterations. Conclusion: A theory- and evidence-based digital behaviour change intervention will be developed which will be tested in a feasibility randomised trial following completion of this study. The projected benefit includes cost-effective health care service (reduced hospitalisations and demand for specialist services) and improved health outcomes and quality of life for patients with AI.

The impact of chemotherapy on cognitive performance post-surgery in patients with colorectal cancer: A prospective cohort study.

OBJECTIVES: Subjective reports of cognitive impairment following chemotherapy are frequent in cancer patients. Objective cognitive impairment has been observed in cancer patients regardless of treatment regimen suggesting the relationship between cognitive impairment and chemotherapy is not clear cut. Little research has explored the effects of chemotherapy on cognition following surgery in colorectal cancer (CRC). The present study explored the effects of chemotherapy on cognitive performance in a sample of CRC patients. METHODS: 136 participants were recruited into a prospective cohort study: 78 CRC patients undergoing surgery and adjuvant chemotherapy, 58 CRC patients undergoing surgery only. A battery of neuropsychological tests was administered to participants 4 weeks post-surgery (T1), 12 weeks after first chemotherapy (T2) and 3 months after last chemotherapy (T3) or equivalent time-points. RESULTS: Using the criterion of scoring at least two standard-deviations below the group norm on at least one neuropsychological test, 45%-55% of all CRC patients showed cognitive deficits 10 months after surgery (T3) and 14% on at least 3 tests. However, cognition did not significantly differ between patients who had chemotherapy and those who did not. A time by group interaction effect was found on the composite cognition score using multi-level modelling suggesting a greater improvement in cognition in the surgery only group over time (p < 0.05). CONCLUSIONS: CRC patients display cognitive impairment 10 months after surgery. Chemotherapy did not worsen cognitive impairment but did appear to slow cognitive recovery relative to those undergoing surgery only. The findings demonstrate a clear need for supportive cognitive interventions for all CRC patients following treatment.

A Bayesian Approach for Estimating the Survivor Average Causal Effect When Outcomes Are Truncated by Death in Cluster-Randomized Trials.

Many studies encounter clustering due to multicenter enrollment and nonmortality outcomes, such as quality of life, that are truncated due to death-that is, missing not at random and nonignorable. Traditional missing-data methods and target causal estimands are suboptimal for statistical inference in the presence of these combined issues, which are especially common in multicenter studies and cluster-randomized trials (CRTs) carried out among the elderly or seriously ill. Using principal stratification, we developed a Bayesian estimator that jointly identifies the always-survivor principal stratum in a clustered/hierarchical data setting and estimates the average treatment effect among them (i.e., the survivor average causal effect (SACE)). In simulations, we observed low bias and good coverage with our method. In a motivating CRT, the SACE and the estimate from complete-case analysis differed in magnitude, but both were small, and neither was incompatible with a null effect. However, the SACE estimate has a clear causal interpretation. The option to assess the rigorously defined SACE estimand in studies with informative truncation and clustering can provide additional insight into an important subset of study participants. Based on the simulation study and CRT reanalysis, we provide practical recommendations for using the SACE in CRTs and software code to support future research.

Prevalence and trajectories of subjective cognitive complaints and implications for patient outcomes: A prospective study of haemodialysis patients.

OBJECTIVES: Cognitive impairment is common in haemodialysis patients and is associated with increased hospitalization and mortality. However, subjective cognitive complaints (SCCs), the self-experienced difficulties in everyday cognitive activities, remain poorly understood. This study examined the prevalence and course of SCCs in haemodialysis patients and its longitudinal associations with sociodemographic, clinical and patient-reported variables. DESIGN: Observational prospective study with baseline and 12-month follow-up assessment. METHODS: Based on a validated cut-off point on the Kidney Disease Quality of Life Cognitive Function subscale, haemodialysis patients (N = 159; 40.3% female, mean age 53.62) were classified into cognitive complaint trajectories: (1) resilient (60.4%; no/low SCCs throughout); (2) persistent (8.8%; stable high SCCs); (3) deterioration (17.6%; from no/low to high SCCs); and (4) recovery (13.2%; from high to no/low SCCs). Sociodemographic/clinical characteristics, self-efficacy, self-management skills, adherence, mood and biochemical assays were measured at both assessments and compared among trajectories using mixed ANOVAs. RESULTS: Interaction effects indicated significant improvements in the recovery group in clinical outcomes (i.e., decreased phosphorus and calcium-phosphorus product), self-efficacy and mood over time. Group effects indicated significantly poorer self-efficacy, self-management skills and adherence in the persistent group than other trajectories across both assessments. None of the sociodemographic/clinical characteristics was associated with SCC trajectories. CONCLUSIONS: The extent of SCCs vary over time across haemodialysis patients. Routine screening of SCCs in dialysis settings may help identifying patients at risk of poor self-management and worse prognosis. Strategies that compensate for cognitive lapses may mitigate the perceived cognitive burden of this population.

Psychosocial Risk Factors for Health-Related Quality of Life in Adult Congenital Heart Disease.

BACKGROUND: There is variability in the impact of adult congenital heart disease (ACHD) on health-related quality of life (HRQoL). A greater insight into the impact of ACHD may be gained from investigating HRQoL in various diagnostic groups and considering the importance of psychosocial risk factors for poor HRQoL. OBJECTIVE: We compared the HRQoL of people with ACHD with normative data from the general population and among 4 diagnostic groups and identified risk factors for poor HRQoL in ACHD from a comprehensive set of sociodemographic, clinical, and psychosocial factors. METHODS: We conducted a cross-sectional study with 303 participants from 4 diagnostic groups Simple, Tetralogy of Fallot, Transposition of the Great Arteries, Single Ventricle who completed measures of illness perceptions, coping, social support, mood, and generic and disease-specific HRQoL. Data were analyzed using 1-sample t tests, analysis of variance, and hierarchical multiple regressions. RESULTS: There was diminished psychosocial HRQoL in the Simple group compared with the general population. Consistently significant risk factors for poor HRQoL included younger age, a perception of more severe symptoms due to ACHD, depression, and anxiety. Clinical factors were poor predictors of HRQoL. CONCLUSIONS: The findings highlight the need to develop intervention studies aiming to improve HRQoL in people with ACHD and the routine assessment of illness perceptions and mood problems during key periods in people's lives. This will help address patient misconceptions that could be tackled by clinicians or specialist nurses during routine outpatient appointments and identify people in need of psychological support.

Subjective cognitive complaints in end-stage renal disease: a systematic review and meta-analysis.

Cognitive impairment is common in patients with end-stage renal disease (ESRD) and is associated with compromised quality of life and functional capacity, as well as worse clinical outcomes. Most previous research and reviews in this area were focused on objective cognitive impairment, whereas patients' subjective cognitive complaints (SCCs) have been less well-understood. This systematic review aimed to provide a broad overview of what is known about SCCs in adult ESRD patients. Electronic databases were searched from inception to January 2022, which identified 221 relevant studies. SCCs appear to be highly prevalent in dialysis patients and less so in those who received kidney transplantation. A random-effects meta-analysis also shows that haemodialysis patients reported significantly more SCCs than peritoneal dialysis patients (standardised mean difference -0.20, 95% confidence interval -0.38 to -0.03). Synthesis of longitudinal studies suggests that SCCs remain stable on maintenance dialysis treatment but may reduce upon receipt of kidney transplant. Furthermore, SCCs in ESRD patients have been consistently associated with hospitalisation, depression, anxiety, fatigue, and poorer quality of life. There is limited data supporting a strong relation between objective and subjective cognition but preliminary evidence suggests that this association may be domain-specific. Methodological limitations and future research directions are discussed.

The role of subjective cognitive complaints in self-management among haemodialysis patients: a cross-sectional study.

BACKGROUND: Subjective cognitive complaints refer to self-experienced difficulties with everyday cognitive tasks. Although there has been a fair amount of research on cognitive impairments and cognitive complaints in end-stage renal disease, the practical implications of these complaints remain unclear. The current study aims to examine the associations of cognitive complaints with sociodemographic and clinical variables, mood, as well as key patient-reported outcomes, i.e., self-efficacy, self-management skills, and treatment adherence. METHODS: A total of 305 haemodialysis patients (mean age = 53.97 years, 42.6% female) completed the Kidney Disease Quality of Life Cognitive Function subscale, a brief measure of cognitive complaints. The recommended cut-off point of 60 was used to identify probable cognitive impairment. Measures of self-efficacy, self-management skills (i.e., symptom coping, health monitoring, health service navigation), treatment adherence, and mood symptoms were also administered. Between-group comparisons and correlational analyses were performed to examine associations of cognitive complaints with sociodemographic, clinical, and health behaviour variables. Mediation analyses were also conducted to investigate the mediating role of self-efficacy on the relationship between cognitive complaints and treatment adherence. RESULTS: Nearly a quarter (23.0%) of haemodialysis patients reported cognitive complaints indicative of clinical impairments. Risk of probable impairments was higher for patients with hypertension, diabetes, those diagnosed with end-stage renal disease at an older age, and those with shorter time on dialysis. Subjective cognitive complaints (both rates of probable impairments as per cut-off and continuous scores) were significantly associated with lower disease and treatment self-efficacy, poorer self-management skills, lower treatment adherence, as well as higher symptoms of distress. Mediation analysis indicated that treatment self-efficacy mediated the relationship between cognitive complaints and treatment adherence. CONCLUSIONS: The current study demonstrated the clinical characteristics of haemodialysis patients who report cognitive complaints indicative of probable cognitive impairments and showed the associations of these complaints with self-management outcomes. Future studies should adopt more comprehensive measures of cognitive complaints and longitudinal designs to confirm the current findings.

The Effects of a Web-Based Tool for Parents of Children With Juvenile Idiopathic Arthritis: Randomized Controlled Trial.

BACKGROUND: Juvenile idiopathic arthritis (JIA) is a group of autoinflammatory diseases that cause pain and disability if not controlled by treatment. Parenting a child with JIA is stressful for parents, who express concerns about their child's treatment and may experience anxiety and powerlessness concerning their child's illness. Parenting stress is greater in parents of children with chronic illness than in those with healthy children and is related to poorer psychological adjustment in both parents and children. It is therefore important to develop interventions to support parents. This paper reports the evaluation of a web-based tool that provides information and practical skills to help increase parents' confidence in managing their child's illness and reduce parenting stress. OBJECTIVE: The aim of this study is to evaluate the benefits of a web-based tool (WebParC) for parents of children with recently diagnosed JIA. METHODS: A multicentered randomized controlled trial was conducted at pediatric rheumatology centers in England. We recruited parents of children aged ≤12 years who had been diagnosed with JIA within the previous 6 months. They were randomized to the intervention (WebParC access plus standard care) or the control (standard care alone) and followed up 4 months and 12 months after randomization. Where both parents participated, they were randomized by household to the same trial arm. The WebParC intervention consists of information about JIA and its treatment plus a toolkit, based on cognitive behavioral therapy, to help parents develop skills to manage JIA-related issues. The primary outcome was the self-report Pediatric Inventory for Parents measure of illness-related parenting stress. The secondary outcomes were parental mood, self-efficacy, coping, effectiveness of participation in their child's health care, satisfaction with health care, and child's health-related quality of life. RESULTS: A total of 203 households comprising 220 parents were randomized to the intervention (100/203, 49.3%) or control (103/203, 50.7%) arm. Follow-up assessments were completed by 65.5% (133/203) of the households at 4 months (intervention 60/100, 60%, and control 73/103, 70.9%) and 61.1% (124/203) of the households at 12 months (intervention 58/100, 58%, and control 66/103, 64.1%). A main effect of the trial arm was found on the Pediatric Inventory for Parents: the intervention participants reported less frequency (subscales communication F1,120627=5.37; P=.02, and role function F1,27203=5.40; P=.02) and difficulty (subscales communication F1,2237=7.43; P=.006, medical care F1,2907=4.04; P=.04, and role function F1,821=4.37, P=.04) regarding illness-related stressful events than the control participants. CONCLUSIONS: The WebParC website for parents of children with JIA reduced illness-related parenting stress. This web-based intervention offers a feasible preventive approach for parents of children with JIA and potentially could be adapted and evaluated for parents of children with other chronic illnesses. TRIAL REGISTRATION: International Standard Randomized Controlled Trial Number (ISRCTN) 13159730; http://www.isrctn.com/ISRCTN13159730.

A patient-initiated treatment model for blepharospasm and hemifacial spasm: a randomized controlled trial.

BACKGROUND: To test, in a two-arm, single center, superiority, randomized controlled trial, the effectiveness of and costs associated with a patient-initiated treatment model for people with hemifacial spasm (HFS) and blepharospasm (BEB) in comparison to usual care. METHODS: One hundred and thirty patients with HFS or BEB, aged 18 years or over, were recruited from a nurse-led botulinum toxin type A clinic at an eye hospital in the United Kingdom (UK), completed baseline measures and were randomized (1:1). The intervention group determined their own botulinum toxin type A (BoNT/A) treatment schedule during the trial period (9 months) and received an information leaflet with a "hotline" number to book an appointment. Usual care appointments were scheduled by treating clinicians. Data analysts were blind to study group. The primary outcomes were disease severity and functional disability, as measured by the Jankovic Rating Scale and Blepharospasm Disability Index, respectively. Secondary outcomes included quality of life, anxiety and depression, satisfaction with care, confidence in the service, economic costs and employment days lost. RESULTS: Sixty-five patients were randomized to each group. The intervention demonstrated no statistically significant difference to usual care for any of primary outcomes. On secondary outcomes the levels of anxiety differed significantly (F2, 142.39 = 1.65, p = 0.02), with the intervention arm exhibiting a decrease and the control arm an increase (Hedges' g = - 0.26 [99% CI -0.83, 0.32]). No other statistically significant differences were found for secondary outcomes. Overall healthcare costs and costs to the patient were on average £198.95 less (95% CI -£256.76, £654.67; p = 0.10) per participant for those in the intervention compared to usual care, although this finding was not significant. CONCLUSIONS: We did not observe differences between the patient-initiated treatment model and usual care for people with BEB or HFS, on any primary outcome measure, quality of life, or depression. The patient-initiated treatment model may, however, have the potential to save healthcare costs and reduce anxiety. Patients using this new model were also equally as satisfied in the service and confident in their care as those receiving treatment as usual. TRIAL REGISTRATION: Clinicaltrials.gov ID NCT02577224 , 16th October 2015.

Randomised open-label trial comparing intravenous iron and an erythropoiesis-stimulating agent versus oral iron to treat preoperative anaemia in cardiac surgery (INITIATE trial).

BACKGROUND: Preoperative anaemia is a risk factor for adverse postoperative outcomes after cardiac surgery. Iron deficiency is a frequent cause of low preoperative haemoglobin. An effective treatment for preoperative anaemia associated with iron deficiency has not been determined. METHODS: We conducted a single-centre, open-label, pragmatic randomised trial, enrolling 156 elective cardiac surgery patients who had low preoperative haemoglobin (100-130 g L-1) with iron deficiency (serum ferritin <100 µg L-1 or transferrin saturation <30%) to compare intravenous ferric derisomaltose 1000 mg and darbepoetin 200 µg subcutaneously (intervention group) with oral ferrous sulphate 600 mg daily (control group). The primary outcome was transfusion of at least one unit of allogeneic red cells during surgery and within the following 5 days. Secondary outcomes included the change in haemoglobin concentration between randomisation and surgery, red cell transfusion volume, postoperative blood loss, pre-specified postoperative complications, length of hospital stay, and in-hospital death. RESULTS: The odds of red cell transfusion were lower in the intervention group compared with the control group (adjusted odds ratio=0.33; 95% confidence interval [CI], 0.15-0.75; P=0.008). Of the secondary outcomes, the only significant difference was the increase in haemoglobin between randomisation and surgery, intervention vs control 9.5 g L-1 (95% CI, 6.8-12.2; P<0.001). CONCLUSIONS: In patients with a low preoperative haemoglobin and iron deficiency, preoperative treatment with a single dose of ferric derisomaltose and darbepoetin decreased the proportion of participants who received a perioperative blood transfusion as a consequence of a greater increase in haemoglobin compared with treatment with oral ferrous sulphate. CLINICAL TRIAL REGISTRATION: ISRCTN Number: 41421863; EUDRACT number: 2011-003695-36.

Assistive technology and telecare to maintain independent living at home for people with dementia: the ATTILA RCT.

BACKGROUND: Assistive technology and telecare have been promoted to manage the risks associated with independent living for people with dementia, but there is limited evidence of their effectiveness. OBJECTIVES: This trial aimed to establish whether or not assistive technology and telecare assessments and interventions extend the time that people with dementia can continue to live independently at home and whether or not they are cost-effective. Caregiver burden, the quality of life of caregivers and of people with dementia and whether or not assistive technology and telecare reduce safety risks were also investigated. DESIGN: This was a pragmatic, randomised controlled trial. Blinding was not undertaken as it was not feasible to do so. All consenting participants were included in an intention-to-treat analysis. SETTING: This trial was set in 12 councils in England with adult social services responsibilities. PARTICIPANTS: Participants were people with dementia living in the community who had an identified need that might benefit from assistive technology and telecare. INTERVENTIONS: Participants were randomly assigned to receive either assistive technology and telecare recommended by a health or social care professional to meet their assessed needs (a full assistive technology and telecare package) or a pendant alarm, non-monitored smoke and carbon monoxide detectors and a key safe (a basic assistive technology and telecare package). MAIN OUTCOME MEASURES: The primary outcomes were time to admission to care and cost-effectiveness. Secondary outcomes assessed caregivers using the 10-item Center for Epidemiological Studies Depression Scale, the State-Trait Anxiety Inventory 6-item scale and the Zarit Burden Interview. RESULTS: Of 495 participants, 248 were randomised to receive full assistive technology and telecare and 247 received the limited control. Comparing the assistive technology and telecare group with the control group, the hazard ratio for institutionalisation was 0.76 (95% confidence interval 0.58 to 1.01; p = 0.054). After adjusting for an imbalance in the baseline activities of daily living score between trial arms, the hazard ratio was 0.84 (95% confidence interval 0.63 to 1.12; p = 0.20). At 104 weeks, there were no significant differences between groups in health and social care resource use costs (intervention group - control group difference: mean -£909, 95% confidence interval -£5336 to £3345) or in societal costs (intervention group - control group difference: mean -£3545; 95% confidence interval -£13,914 to £6581). At 104 weeks, based on quality-adjusted life-years derived from the participant-rated EuroQol-5 Dimensions questionnaire, the intervention group had 0.105 (95% confidence interval -0.204 to -0.007) fewer quality-adjusted life-years than the control group. The number of quality-adjusted life-years derived from the proxy-rated EuroQol-5 Dimensions questionnaire did not differ between groups. Caregiver outcomes did not differ between groups over 24 weeks. LIMITATIONS: Compliance with the assigned trial arm was variable, as was the quality of assistive technology and telecare needs assessments. Attrition from assessments led to data loss additional to that attributable to care home admission and censoring events. CONCLUSIONS: A full package of assistive technology and telecare did not increase the length of time that participants with dementia remained in the community, and nor did it decrease caregiver burden, depression or anxiety, relative to a basic package of assistive technology and telecare. Use of the full assistive technology and telecare package did not increase participants' health and social care or societal costs. Quality-adjusted life-years based on participants' EuroQol-5 Dimensions questionnaire responses were reduced in the intervention group compared with the control group; groups did not differ in the number of quality-adjusted life-years based on the proxy-rated EuroQol-5 Dimensions questionnaire. FUTURE WORK: Future work could examine whether or not improved assessment that is more personalised to an individual is beneficial. TRIAL REGISTRATION: Current Controlled Trials ISRCTN86537017. FUNDING: This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 25, No. 19. See the NIHR Journals Library website for further project information.

Many people with dementia living at home are recommended assistive technology and telecare to help them remain living safely and independently in the community. These devices are meant to assist and support activities such as taking medication or cooking, or to raise an alert when there is an issue, such as a fire; however, there is currently little evidence to support such claims. This trial investigated whether or not assistive technology and telecare could delay people moving into residential care and keep them any safer than alternatives, and whether or not they were cost-effective. We recruited 495 people with dementia and their unpaid caregivers, who were randomly assigned to receive either a package of assistive technology and telecare recommended by a health or social care professional or alternative support involving only basic assistive technology and telecare. We monitored the residential status, the use of health-care services and the health and well-being of participants with dementia and their caregivers over a 2-year period. Researchers also spent time with participants to see how they were living with the technology. The trial found no difference in the time that people with dementia with full assistive technology and telecare remained at home, nor any reduction in the number of safety incidents, compared with the participants who received basic assistive technology and telecare only. Full assistive technology and telecare did not increase health and social care costs. It did not improve the well-being of people with dementia or that of their caregivers. People with dementia who had full assistive technology and telecare rated their quality of life poorer than those with basic assistive technology and telecare did, but their caregivers rated their quality of life as about the same as caregivers of people with basic assistive technology and telecare. The technology sometimes averted crises but also disrupted people's everyday lives. These results suggest that assistive technology and telecare for people with dementia provided in real-world conditions may not be as beneficial as previously claimed. The way that assistive technology and telecare services are organised bears further investigation to see how these services could be improved.

Illness Uncertainty in Parents of Children with Juvenile Idiopathic Arthritis.

OBJECTIVE: To gain a better understanding of uncertainty regarding the illness experienced by parents of children with juvenile idiopathic arthritis (JIA). METHODS: Parents/guardians of a child or young person (aged less than 18 years) diagnosed JIA were recruited in the United Kingdom via the National Rheumatoid Arthritis Society JIA group. Semistructured telephone interviews were conducted with the parents. RESULTS: Twenty parents took part, including 19 mothers and one father. Their children with JIA were mostly female (n = 15; 75%) with polyarticular arthritis (n = 12; 60%), averaged 8 years of age, and had been diagnosed for a mean of 3.7 (SD 2.3) years. Parents expressed uncertainty in the following five key domains: diagnosis, cause, symptoms, and prognosis; medical management; impact; parenting uncertainty; and awareness of JIA. All participants expressed uncertainty in at least four of the five domains. Although parents' uncertainty in the early stages of the disease related to lack of information and understanding of JIA, much uncertainty could not be resolved by receipt of information. These included concerns about their child's future and a lack of support with managing the emotional aspects of living with JIA. CONCLUSION: We found that parents' experiences of uncertainty went beyond dealing with the purely medical aspects of JIA. Provision of information about JIA, although essential, is not sufficient to help parents manage the considerable uncertainty they experience about many aspects of their child's JIA. Identifying ways to incorporate support for coping with uncertainty into routine care will be an important way of supporting parents to care for their child with JIA.

Placing assistive technology and telecare in everyday practices of people with dementia and their caregivers: findings from an embedded ethnography of a national dementia trial.

BACKGROUND: Policy makers and care providers see assistive technology and telecare as potential products to support people with dementia to live independently in their homes and communities. Previous research rarely examined how people with dementia and their caregivers actually use such technology. The study examined how and why people living with dementia and their caregivers used assistive technology and telecare in their own homes. METHODS: This study used an ethnographic design embedded within the NIHR-funded Assistive Technology and Telecare to maintain Independent Living At home for people with dementia (ATTILA) randomised controlled trial. We collected 208 h of observational data on situated practices of ten people with dementia and their ten caregivers. We used this data to construct extended cases to explain how technologies supported people with dementia in home and community settings. RESULTS: We identified three themes: placing technology in care, which illustrates how people with dementia and caregivers 'fit' technology into their homes and routines; replacing care with technology, which shows how caregivers replaced normal care practices with ones mediated through technologies; and technology displacing care and everyday life, which highlights how technologies disrupted the everyday lives of people with dementia. DISCUSSION: This study exemplifies unintended and unanticipated consequences for assistive technology and telecare uptake in 'real world' community-based dementia care. It underlines the need to identify and map the context of technological provision over time within the changing lives of people with dementia and their caregivers.

Does prospective acceptability of an intervention influence refusal to participate in a randomised controlled trial? An interview study.

BACKGROUND: The generalizability of findings of Randomised Controlled Trials (RCTs) is undermined by low or biased recruitment. Reasons for participant refusal are infrequently reported in published literature. AIMS: To apply the Theoretical Framework of Acceptability (TFA) to: (1) explore patient-reported reasons for declining to participate in a RCT comparing a new service model (patient-initiated appointments) with standard care (appointments scheduled by clinician) for managing blepharospasm and hemifacial spasm; (2) to explore associations between decliners' perceptions of acceptability and non-participation. METHOD: Eligible patients (n = 242) were approached to participate in the trial. Phase 1: decliners provided a brief reason for refusal. Reasons were analysed descriptively and reviewed against TFA constructs. PHASE 2: Consecutive decliners participated in short semi-structured interviews, to explore their reasons for refusal in more depth. Interviews were transcribed and analysed, with the TFA as a coding framework. RESULTS: Eighty-seven (36%) eligible patients refused trial participation; all provided a reason. From interviews with 15 decliners (17%), four key beliefs about acceptability were identified: happy with standard care (n = 41) (49%), anticipated burden of patient-initiated service, lack of confidence in ability to engage with new service and uncertainties about effectiveness of new service. Two themes reflected non-TFA factors: trial participation a low priority and burden of completing trial documentation. CONCLUSION: Reasons for refusal trial participation included: (a) reasons directly associated with intervention acceptability, and (b) reasons associated with trial participation more broadly. The TFA facilitated identification of problematic aspects of the new appointment booking system which could be addressed to enhance acceptability.

The effectiveness and cost-effectiveness of assistive technology and telecare for independent living in dementia: a randomised controlled trial.

OBJECTIVES: The use of assistive technology and telecare (ATT) has been promoted to manage risks associated with independent living in people with dementia but with little evidence for effectiveness. METHODS: Participants were randomly assigned to receive an ATT assessment followed by installation of all appropriate ATT devices or limited control of appropriate ATT. The primary outcomes were time to institutionalisation and cost-effectiveness. Key secondary outcomes were number of incidents involving risks to safety, burden and stress in family caregivers and quality of life. RESULTS: Participants were assigned to receive full ATT (248 participants) or the limited control (247 participants). After adjusting for baseline imbalance of activities of daily living score, HR for median pre-institutionalisation survival was 0.84; 95% CI, 0.63 to 1.12; P = 0.20. There were no significant differences between arms in health and social care (mean -£909; 95% CI, -£5,336 to £3,345, P = 0.678) and societal costs (mean -£3,545; 95% CI, -£13,914 to £6,581, P = 0.499). ATT group members had reduced participant-rated quality-adjusted life years (QALYs) at 104 weeks (mean - 0.105; 95% CI, -0.204 to -0.007, P = 0.037) but did not differ in QALYs derived from proxy-reported EQ-5D. DISCUSSION: Fidelity of the intervention was low in terms of matching ATT assessment, recommendations and installation. This, however, reflects current practice within adult social care in England. CONCLUSIONS: Time living independently outside a care home was not significantly longer in participants who received full ATT and ATT was not cost-effective. Participants with full ATT attained fewer QALYs based on participant-reported EQ-5D than controls at 104 weeks.

Which factors impact on quality of life for adults with blepharospasm and hemifacial spasm?

Purpose: Benign essential blepharospasm (BEB) and hemifacial spasm (HFS) are debilitating conditions causing spasms to the eyes and/or face and can significantly impact on quality of life (QoL). Initial research has highlighted potential factors impacting on QoL in BEB, but there remains a wealth of demographic, clinical, and psychosocial factors that may contribute to QoL but have not received attention. Methods: Cross-sectional baseline data were collected before a single-masked randomised controlled trial from 130 adults with BEB and HFS recruited from botulinum toxin clinics at Moorfields Eye Hospital, London. QoL was measured using the 24-item Craniocervical Dystonia Questionnaire (CDQ24), which provides a total score and five subscale scores relating to Stigma, Emotional state, Pain, Activities of daily living (ADL), and Social/family life. Treating clinicians provided clinical data. Hierarchical multiple regressions were performed on this baseline data to identify significant predictors of QoL. Results: ADL and Stigma were the areas most impacted upon whilst patients experienced better adjustment in relation to Pain, Social/family life, and Emotional state. CDQ24 Total scores were explained by the model (80% variance) and were significantly associated with appearance concerns, emotional representations, perceived negative consequences of the condition, mood, and dose of botulinum toxin. Conclusions: Patients with BEB and HFS report a detrimental impact on ADL and perceived stigma in relation to their condition. Predominantly, individual perceptions and mood are associated with QoL in this population, rather than demographic and clinical factors, signifying areas to target in the design of future healthcare services or interventions.

Men's beliefs about treatment for erectile dysfunction-what influences treatment use? A systematic review.

Successful treatment of erectile dysfunction (ED) is associated with improvements in quality of life; however, treatment utilisation is sub-optimal. The aim of this systematic review was to identify the rates of ED treatment utilisation and the barriers and enablers men experience when using treatment. We searched: MEDLINE®, Embase, the Cochrane library; AMED; HMIC; HTA; CINAHL; PsychARTICLES; PsychINFO up to August 2018. Data on rates of treatment utilisation and barriers and enablers of utilisation were extracted and summarised. Fifty studies were included. Discontinuation rates ranged from 4.4 to 76% for phosphodiesterase type 5 inhibitors, 18.6 to 79.9% for intracavernosal injections, and 32 to 69.2% for urethral suppositories. In relation to those with a penile prosthesis, 30% discontinued having sex due to, e.g. device complications, lack of partner or a loss of sexual interest. Most research included in the current review examined barriers to treatment utilisation and therefore focussed on reasons for discontinuing treatment. However, a small number explored factors that men found helpful with regards to treatment utilisation. The most prevalent barriers to utilisation were treatment ineffectiveness, side effects, the quality of men's intimate relationships and treatment costs. With regards to treatment enablers, the most salient finding was that men who reported side effects to a healthcare professionals (HCPs) were significantly less likely to discontinue treatment. There were limitations in methodology in that the studies did not use validated measures of treatment utilisation or barriers and enablers and no study used psychological theory to inform the examination of factors that influenced treatment utilisation. This review identifies a number of influential factors relating to ED treatment utilisation and highlights the importance of men's beliefs with regards to ED and its treatment. Beliefs are potentially modifiable and therefore the findings of this review highlight important considerations for HCPs with regards to supporting men to make better use of treatment.

A patient-initiated DMARD self-monitoring service for people with rheumatoid or psoriatic arthritis: a cost-effectiveness analysis.

OBJECTIVE: To determine whether a patient-initiated DMARD self-monitoring service for people on MTX is a cost-effective model of care for patients with RA or PsA. METHODS: An economic evaluation was undertaken alongside a randomized controlled trial involving 100 patients. Outcome measures were quality of life and ESR assessed at baseline and post-intervention. Costs were calculated for healthcare usage using a United Kingdom National Health Service economic perspective. Sensitivity analysis was performed to explore the impact of nurse-led telephone helplines. Uncertainty around the cost-effectiveness ratios was estimated by bootstrapping and analysing the cost-effectiveness planes. RESULTS: Fifty-two patients received the intervention and 48 usual care. The difference in mean cost per case indicated that the intervention was £263 more expensive (P < 0.001; 95% CI: £149.14, £375.86) when the helpline costs were accounted for and £94 cheaper (P = 0.08; 95% CI: -£199.26, £10.41) when these costs were absorbed by the usual service. There were, however, statistically significant savings for the patient (P = 0.02; 95% CI: -£28.98, £3.00). When costs and effectiveness measures of ESR and quality of life measured, using the Short Form-12v1, were combined this did not show the patient-initiated service to be cost-effective at a statistically significant level. CONCLUSION: This patient-initiated service led to reductions in primary and secondary healthcare services that translated into reduced costs, in comparison with usual care, but were not cost-effective. Further work is needed to establish how nurse-led telephone triage services are integrated into rheumatology services and the associated costs of setting up and delivering them. TRIAL REGISTRATION: ClinicalTrials.gov, http://clinicaltrials.gov, ISRCTN21613721.