Disclosing Recovery: A pilot randomized controlled trial of a patient decision aid to improve disclosure processes for people in treatment for opioid use disorder.

INTRODUCTION: People engaged in treatment for opioid use disorder (OUD) report struggling with whether and how to disclose, or share information about their OUD history and/or treatment with others. Yet, disclosure can act as a gateway to re-establishing social connection and support during recovery. The current study describes a pilot randomized controlled trial of Disclosing Recovery: A Decision Aid and Toolkit, a patient decision aid designed to facilitate disclosure decisions and build disclosure skills. METHODS: Participants (n = 50) were recruited from a community-based behavioral health organization in 2021-2022 and randomized to receive the Disclosing Recovery intervention versus an attention-control comparator. They responded to surveys immediately after receiving the intervention as well as one month following the intervention at a follow-up appointment. Primary outcome analyses examined indicators of implementation of the intervention to inform a future efficacy trial. Secondary outcome analyses explored impacts of the intervention on the decision-making process, disclosure rates, and relationships. RESULTS: Participants were successfully recruited, randomized, and retained, increasing confidence in the feasibility of future efficacy trials to test the Disclosing Recovery intervention. Moreover, participants in the Disclosing Recovery intervention agreed that the intervention is acceptable, feasible, and appropriate. They additionally reported a higher quality of their decision-making process and decisions than participants in the comparator condition. At their follow-up appointment, participants with illicit opioid use who received the Disclosing Recovery intervention were less likely to disclose than those who received the comparator condition. Moreover, significant interactions between illicit opioid use and the intervention condition indicated that participants without illicit opioid use who received the Disclosing Recovery intervention reported greater closeness to and social support from their planned disclosure recipient than those who received the comparator condition. CONCLUSIONS: The Disclosing Recovery intervention appears to be an acceptable, feasible, and appropriate patient decision aid for addressing disclosure processes among people in treatment for OUD. Moreover, preliminary results suggest that it shows promise in improving relationship closeness and social support in patients without illicit opioid use. More testing is merited to determine the intervention's efficacy and effectiveness in improving relationship and treatment outcomes for people in treatment for OUD.

The WHO Mental Health Gap Action Programme for mental, neurological, and substance use conditions: the new and updated guideline recommendations.

The WHO Mental Health Gap Action Programme (mhGAP) guideline update reflects 15 years of investment in reducing the treatment gap and scaling up care for people with mental, neurological, and substance use (MNS) conditions. It was produced by a guideline development group and steering group, with support from topic experts, using quantitative and qualitative evidence and a systematic review of use of mhGAP. 90 recommendations from the 2015 guideline update were validated and endorsed for use in their current format. These are joined by 30 revised recommendations and 18 new recommendations, including a new module on anxiety. Psychological interventions are emphasised as treatments and digitally delivered interventions feature across many modules, as well as updated recommendations for psychotropic medicines. Research gaps identified include the need for evidence from low-resource settings and on the views of people with lived experience of MNS conditions. The revised recommendations ensure that mhGAP continues to offer high-quality, timely, transparent, and evidence-based guidance to support non-specialist health workers in low-income and middle-income countries in providing care to individuals with MNS conditions.

Psychosocial interventions for carers of people with severe mental and substance use disorders: a systematic review and meta-analysis.

BACKGROUND: Severe mental disorders - such as schizophrenia, bipolar disorder, and substance use disorders - exert a negative impact not only on affected people but also on their carers. To support carers of people with severe mental disorders, several psychosocial interventions have been developed. METHODS: This systematic review and meta-analysis aimed to assess whether psychosocial interventions for carers of persons with schizophrenia, bipolar disorder, or substance use disorders produce benefit/harm with respect to a series of outcomes - including subjective and objective burden, depressive symptoms, well-being/quality of life, sleep, skills/knowledge, self-efficacy, physical health - as compared to standard support/support as usual or other control conditions. RESULTS: In carers of persons with schizophrenia, psychoeducational interventions were associated with significant improvement in personal burden, well-being, and knowledge about the illness; and a supportive-educational intervention with an improvement in personal burden. In carers of persons with bipolar disorder, psychoeducational interventions were associated with significant improvement in personal burden and depressive symptoms; family-led supportive interventions with an improvement in family burden; family-focused intervention and online "mi.spot" intervention with a significant reduction in depressive symptoms. Psychosocial interventions used for carers of persons with substance use disorders were found to be overall effective on the level of well-being, but the low number of trials did not allow detection of differences between the various psychosocial interventions. CONCLUSIONS: The quality of the evidence ranged from very low to moderate, suggesting the need for further better-quality research.

Qualitative evidence regarding the experience of receiving and providing care for mental health conditions in non-specialist settings in low-income and middle-income countries: a systematic review of reviews.

QUESTION: This review of reviews synthesises qualitative evidence on the experiences of receiving and providing care and treatment for mental health conditions in non-specialist settings in low-income and middle-income countries (LMICs), and the factors that influence the provision and uptake of such services. STUDY SELECTION AND ANALYSIS: Database searches were conducted in PubMed/MEDLINE, EMBASE, PsycINFO, CINAHL, Scopus, African Index Medicus and Global Index Medicus, supplemented by screening repositories of systematic reviews protocols and contacting authors. The evidence synthesis drew on deductive and inductive approaches: a framework analysis approach was used for the initial coding structure, after which the results synthesis was refined further through reviewing and regrouping the initial coding through thematic synthesis principles. FINDINGS: Nine reviews met inclusion criteria and reported on a range of factors related to the provision and uptake of mental healthcare by non-specialist health workers in LMICs: (1) health worker competency, (2) availability of resources, (3) recipient-related and provider-related characteristics, (4) service accessibility, (5) sociocultural acceptability and (6) vulnerable groups for whom barrier to care were potentially exacerbated. CONCLUSIONS: This review provides nuanced and contextualised insights regarding the experiences of receiving and providing care for mental health conditions in LMICs, including barriers influencing service provision and uptake. It is important to ensure mental healthcare in non-specialist settings in LMICs is delivered in a manner which is feasible, acceptable and culturally appropriate in order to improve access to care, reducing stigma and promoting better overall health and well-being for individuals and communities.

Reducing stigma and improving access to care for people with mental health conditions in the community: protocol for a multi-site feasibility intervention study (Indigo-Local).

Background: Stigma and discrimination towards people with mental health conditions by their communities are common worldwide. This can result in a range of negative outcomes for affected persons, including poor access to health care. However, evidence is still patchy from low- and middle-income countries (LMICs) on affordable, community-based interventions to reduce mental health-related stigma and to improve access to mental health care. Methods: This study aims to conduct a feasibility (proof-of-principle) pilot study that involves developing, implementing and evaluating a community-based, multi-component, public awareness-raising intervention (titled Indigo-Local), designed to reduce stigma and discrimination and to increase referrals of people with mental health conditions for assessment and treatment. It is being piloted in five LMICs - China, Ethiopia, India, Nepal and Tunisia - and includes several key components: a stakeholder group workshop; a stepped training programme (using a 'Training of Trainers' approach) of community health workers (or similar cadres of workers) and service users that includes repeated supervision and booster sessions; awareness-raising activities in the community; and a media campaign. Social contact and service user involvement are instrumental to all components. The intervention is being evaluated through a mixed-methods pre-post study design that involves quantitative assessment of stigma outcomes measuring knowledge, attitudes and (discriminatory) behaviour; quantitative evaluation of mental health service utilization rates (where feasible in sites); qualitative exploration of the potential effectiveness and impact of the Indigo-Local intervention; a process evaluation; implementation evaluation; and an evaluation of implementation costs. Discussion: The outcome of this study will be contextually adapted, evidence-based interventions to reduce mental health-related stigma in local communities in five LMICs to achieve improved access to healthcare. We will have replicable models of how to involve people with lived experience as an integral part of the intervention and will produce knowledge of how intervention content and implementation strategies vary across settings. The interventions and their delivery will be refined to be acceptable, feasible and ready for larger-scale implementation and evaluation. This study thereby has the potential to make an important contribution to the evidence base on what works to reduce mental health-related stigma and discrimination and improve access to health care.

The development and validation of the Discrimination and Stigma Scale Ultra Short for People Living with Dementia (DISCUS-Dementia).

BACKGROUND: The recent World Health Organization (WHO) blueprint for dementia research and Lancet Commission on ending stigma and discrimination in mental health has identified a gap around dementia-related measures of stigma and discrimination that can be used in different cultural, language and regional contexts. AIMS: We aimed to characterise experiences of discrimination, and report initial psychometric properties of a new tool to capture these experiences, among a global sample of people living with dementia. METHOD: We analysed data from 704 people living with dementia who took part in a global survey from 33 different countries and territories. Psychometric properties were examined, including internal consistency and construct validity. RESULTS: A total of 83% of participants reported discrimination in one or more areas of life, and this was similar across WHO Regions. The exploratory factor analysis factor loadings and scree plot supported a unidimensional structure for the Discrimination and Stigma Scale Ultra Short for People Living with Dementia (DISCUS-Dementia). The instrument demonstrated excellent internal consistency, with most of the construct validity hypotheses being confirmed and qualitative responses demonstrating face validity. CONCLUSIONS: Our analyses suggest that the DISCUS-Dementia performs well with a global sample of people living with dementia. This scale can be integrated into large-scale studies to understand factors associated with stigma and discrimination. It can also provide an opportunity for a structured discussion around stigma and discrimination experiences important to people living with dementia, as well as planning psychosocial services and initiatives to reduce stigma and discrimination.

How strong should my anchor be for estimating group and individual level meaningful change? A simulation study assessing anchor correlation strength and the impact of sample size, distribution of change scores and methodology on establishing a true meaningful change threshold.

PURPOSE: Treatment benefit as assessed using clinical outcome assessments (COAs), is a key endpoint in many clinical trials at both the individual and group level. Anchor-based methods can aid interpretation of COA change scores beyond statistical significance, and help derive a meaningful change threshold (MCT). However, evidence-based guidance on the selection of appropriately related anchors is lacking. METHODS: A simulation was conducted which varied sample size, change score variability and anchor correlation strength to assess the impact of these variables on recovering the simulated MCT for interpreting individual and group-level results. To assess MCTs derived at the individual-level (i.e. responder definitions; RDs), Receiver Operating Characteristic (ROC) curves and Predictive Modelling (PM) analyses were conducted. To assess MCTs for interpreting change at the group-level, the mean change method was conducted. RESULTS: Sample sizes, change score variability and magnitude of anchor correlation affected accuracy of the estimated MCT. For individual-level RDs, ROC curves were less accurate than PM methods at recovering the true MCT. For both methods, smaller samples led to higher variability in the returned MCT, but higher variability still using ROC. Anchors with weaker correlations with COA change scores had increased variability in the estimated MCT. An anchor correlation of around 0.50-0.60 identified a true MCT cut-point under certain conditions using ROC. However, anchor correlations as low as 0.30 were appropriate when using PM under certain conditions. For interpreting group-level results, the MCT derived using the mean change method was consistently underestimated regardless of the anchor correlation. CONCLUSION: Sample size and change score variability influence the necessary anchor correlation strength when recovering individual-level RDs. Often, this needs to be higher than the commonly accepted threshold of 0.30. Stronger correlations than 0.30 are required when using the mean change method. Results can assist researchers selecting and assessing the quality of anchors.

Measuring discrimination experienced by people with a mental illness: replication of the short-form DISCUS in six world regions.

BACKGROUND: The Discrimination and Stigma Scale (DISC) is a patient-reported outcome measure which assesses experiences of discrimination among persons with a mental illness globally. METHODS: This study evaluated whether the psychometric properties of a short-form version, DISC-Ultra Short (DISCUS) (11-item), could be replicated in a sample of people with a wide range of mental disorders from 21 sites in 15 countries/territories, across six global regions. The frequency of experienced discrimination was reported. Scaling assumptions (confirmatory factor analysis, inter-item and item-total correlations), reliability (internal consistency) and validity (convergent validity, known groups method) were investigated in each region, and by diagnosis group. RESULTS: 1195 people participated. The most frequently reported experiences of discrimination were being shunned or avoided at work (48.7%) and discrimination in making or keeping friends (47.2%). Confirmatory factor analysis supported a unidimensional model across all six regions and five diagnosis groups. Convergent validity was confirmed in the total sample and within all regions [ Internalised Stigma of Mental Illness (ISMI-10): 0.28-0.67, stopping self: 0.54-0.72, stigma consciousness: -0.32-0.57], as was internal consistency reliability (α = 0.74-0.84). Known groups validity was established in the global sample with levels of experienced discrimination significantly higher for those experiencing higher depression [Patient Health Questionnaire (PHQ)-2: p < 0.001], lower mental wellbeing [Warwick-Edinburgh Well-being Scale (WEMWBS): p < 0.001], higher suicidal ideation [Beck Hopelessness Scale (BHS)-4: p < 0.001] and higher risk of suicidal behaviour [Suicidal Ideation Attributes Scale (SIDAS): p < 0.001]. CONCLUSIONS: The DISCUS is a reliable and valid unidimensional measure of experienced discrimination for use in global settings with similar properties to the longer DISC. It offers a brief assessment of experienced discrimination for use in clinical and research settings.

Training for mental health professionals in responding to experienced and anticipated mental health-related discrimination (READ-MH): protocol for an international multisite feasibility study.

BACKGROUND: Mental health and other health professionals working in mental health care may contribute to the experiences of stigma and discrimination among mental health service users but can also help reduce the impact of stigma on service users. However, few studies of interventions to equip such professionals to be anti-stigma agents took place in high-income countries. This study assesses the feasibility, potential effectiveness and costs of Responding to Experienced and Anticipated Discrimination training for health professionals working in mental health care (READ-MH) across low- and middle-income countries (LMICs). METHODS: This is an uncontrolled pre-post mixed methods feasibility study of READ-MH training at seven sites across five LMICs (China, Ethiopia, India, Nepal and Tunisia). OUTCOME MEASURES: knowledge based on course content, attitudes to working to address the impact of stigma on service users and skills in responding constructively to service users' reports of discrimination. The training draws upon the evidence bases for stigma reduction, health advocacy and medical education and is tailored to sites through situational analyses. Its content, delivery methods and intensity were agreed upon through a consensus exercise with site research teams. READ-MH will be delivered to health professionals working in mental health care immediately after baseline data collection; outcome measures will be collected post-training and 3 months post-baseline, followed by qualitative data collection analysed using a combined deductive and inductive approach. Fidelity will be rated during the delivery of READ-MH, and data on training costs will be collected. Quantitative data will be assessed using generalised linear mixed models. Qualitative data will be evaluated by thematic analysis to identify feedback about the training methods and content, including the implementability of the knowledge and skills learned. Pooled and site-specific training costs per trainee and per session will be reported. CONCLUSIONS: The training development used a participatory and contextualised approach. Evaluation design strengths include the diversity of settings, the use of mixed methods, the use of a skills-based measure and the knowledge and attitude measures aligned to the target population and training. Limitations are the uncertain generalisability of skills performance to routine care and the impact of COVID-19 restrictions at several sites limiting qualitative data collection for situational analyses.

How do the number of missing daily diary days impact the psychometric properties and meaningful change thresholds arising from a weekly average summary score?

PURPOSE: Quality of life research often collects daily information and averages this over a week, producing a summary score. When data are missing, arbitrary rules (such as requiring at least 4/7 observations) are used to determine whether a patient's summary score is created or set to missing. This simulation work aimed to assess the impact of missing data on the estimates produced by summary scores, the psychometric properties of the resulting summary score estimates and the impact on interpretation thresholds. METHODS: Complete longitudinal data were simulated for 1000 samples of 400 patients with different day-to-day variability. Data were deleted from these samples in line with missingness mechanisms to create scenarios with up to six days of missing data. Summary scores were created for complete and missing data scenarios. Summary score estimates, psychometric properties and meaningful change estimates were assessed for missing data scenarios compared to complete data. RESULTS: In most cases, the 4/7 day rule was supported, but this depended on daily variability. Fewer days of data were sometimes acceptable, but this was also dependent on the proportion of patients with missing data. Tables and figures allow researchers to assess the potential impact of missing data in their own studies. CONCLUSIONS: This work suggests that the missing data rule used to create summary scores impacts on the estimate, measurement properties and interpretation thresholds. Although a general rule of 4/7 days is supported, the way the summary score is derived does not have a uniform impact across psychometric analyses. Recommendations are to use the 4/7 rule, but plan for sensitivity analyses with other missing data rules.

Training for mental health professionals in responding to experienced and anticipated mental health related discrimination (READ-MH): protocol for an international multisite feasibility study.

Background: Mental health and other health professionals working in mental health care may contribute to the experiences of stigma and discrimination among mental health service users, but can also help reduce the impact of stigma on service users. However the few studies of interventions to equip such professionals to be anti-stigma agents those took place in High-Income Countries. This study assesses the feasibility, potential effectiveness and costs of Responding to Experienced and Anticipated Discrimination training for health professionals working in mental health care (READ-MH) across Low- and Middle-Income Countries (LMICs). Methods: This is an uncontrolled pre-post mixed methods feasibility study of READ-MH training at seven sites across five LMICs (China, Ethiopia, India, Nepal, and Tunisia). Outcome measures: knowledge based on course content; attitudes to working to address the impact of stigma on service users; and skills in responding constructively to service users' reports of discrimination. The training draws upon the evidence bases for stigma reduction, health advocacy and medical education and is tailored to sites through situational analyses. Its content, delivery methods and intensity were agreed through a consensus exercise with site research teams. READ-MH will be delivered to health professionals working in mental health care immediately after baseline data collection; outcome measures will be collected post-training and three months post-baseline, followed by qualitative data collection. Fidelity will be rated during delivery of READ-MH, and data on training costs will be collected. Quantitative data will be assessed using generalised linear mixed models. Qualitative data will be evaluated by thematic analysis to identify feedback about the training methods and content, including the implementability of the knowledge and skills learned. Pooled and site-specific training costs per trainee and per session will be reported. Conclusions: The training development used a participatory and contextualized approach. Evaluation design strengths include the diversity of settings; the use of mixed methods; the use of a skills-based measure; and knowledge and attitude measures aligned to the target population and training. Limitations are the uncertain generalisability of skills performance to routine care, and the impact of COVID-19 restrictions at several sites limiting qualitative data collection for situational analyses.

Health-related quality of life in pediatric patients with partial onset seizures or primary generalized tonic-clonic seizures receiving adjunctive perampanel.

RATIONALE: Study 311 (E2007-G000-311; NCT02849626) was a Phase 3, multicenter, open-label single-arm study of adjunctive perampanel oral suspension in pediatric patients (aged 4 to <12 years) with partial-onset seizures (POS) (with/without secondarily generalized tonic-clonic seizures [SGTCS]) or primary generalized tonic-clonic seizures (PGTCS). Health-related quality of life (HRQoL) was an exploratory endpoint initially analyzed through simple descriptive summaries. The aim of this post hoc analysis was to provide a more thorough assessment of HRQoL. METHODS: This analysis focused on EQ-5D-Y data collected at Baseline, Week 23, and Week 52. Individual dimensions, visual analog scale (VAS) and summed misery index (MI) were evaluated at all visits and compared by seizure type (POS versus SGTCS versus PGTCS), age (4 to <7 versus 7 to <12), and use of concomitant enzyme-inducing antiepileptic drugs (EIAEDs) (yes versus no). Paretian Classification of Health Change (PCHC) analysis summarized the proportion of patients who showed improvement or deterioration in HRQoL. Waterfall plots assessed changes in EQ-5D-Y scores by treatment-emergent adverse events (TEAEs) and by reduction in seizure frequency. Health state utility values associated with differing seizure frequency states were estimated using a linear mixed model. RESULTS: One hundred and fifteen patients completed EQ-5D-Y at relevant study visits (Seizure type: POS n = 84 [of which 21 had SGTCS], PGTCS n = 31; Age: 4 to <7 years n = 30, 7 to <12 years n = 85; Concomitant EIAEDs: Yes n = 35, No n = 80). Completion rates out of those expected to complete EQ-5D-Y were high at both timepoints (84.4% at Week 23 and 97.2% at Week 52). Overall, VAS/MI remained stable over time (did not exceed minimal important difference); this was similar according to seizure type, age, and EIAED usage. In patients with 'no problems' on any EQ-5D-Y dimension at Baseline, nearly all retained their full health at Week 23 (94.7%), and all retained it at Week 52 (100.0%). PCHC analysis showed fewer patients with POS experienced deterioration in EQ-5D-Y than patients with PGTCS at Week 23 (24.1% versus 42.1%). Not experiencing a TEAE, or remaining seizure-free, was associated with improvements in VAS score at Week 23 compared to those experiencing TEAEs or seizures, respectively. Health state utility values (HSUVs) were estimated as follows: seizure free (LS Mean 0.914 [95% CIs 0.587, 1.240]), ≥1 seizure per year (0.620 [0.506, 0.734]), ≥1 seizure per month (0.596 [0.338, 0.855]), ≥1 seizure per week (0.284 [-0.014, 0.582]). CONCLUSIONS: An in-depth analysis of EQ-5D-Y data allowed for a more nuanced exploration of HRQoL than previous descriptive summaries. Our findings provide evidence that perampanel as adjunctive therapy did not result in deterioration of patient HRQoL. The association between TEAEs or remaining seizure-free and HRQoL warrants further exploration. Increasing seizure frequency was associated with decreasing HSUVs; these can inform cost-effectiveness modeling of perampanel and other therapies aiming to reduce seizure frequency in pediatric patients.

Improving patient-clinician communication following nephrectomy in renal cell carcinoma: Development, content validation and pilot testing of a conversation aid tool.

OBJECTIVES: This study developed, and established the content validity, of a conversation aid tool (CAT) for use in clinical practice with renal cell carcinoma (RCC) patients who receive a curative nephrectomy and are at high-risk of recurrence. The CAT was pilot tested in a sample of RCC patients to establish whether the CAT increases knowledge of RCC, treatment options (such as adjuvant therapy), and care options. METHODS: A cross-sectional, mixed methods design was used involving initial, exploratory interviews with RCC patients, RCC specialists and a steering group. Further content validation interviews were conducted with RCC patients and specialists. A web-based survey was conducted with RCC patients (N = 60), to compare the CAT versus a standard of care (SOC) consultation comparator tool on patient knowledge. RESULTS: Findings from exploratory interviews were used to develop the CAT. Content validation interviews demonstrated that the CAT was well understood and relevant to RCC patients. The web-based survey demonstrated that viewing the CAT significantly improved participants knowledge of RCC, and care options, when compared to the SOC. CONCLUSION: The findings highlight that the CAT is a relevant, comprehensive and well-understood tool for use in the post-nephrectomy consultation. PRACTICE IMPLICATIONS: Use of the CAT may increase patient knowledge of RCC and care options.

Health utilities and quality of life in individuals with tuberous sclerosis complex (TSC) who experience epileptic seizures: A web-based survey.

OBJECTIVES: Individuals with tuberous sclerosis complex (TSC) experience a wide range of health impacts, including epileptic seizures, negatively impacting their health-related quality of life (HRQoL). Health state utility values (HSUVs) are index values representing HRQoL and are used as key inputs for health economic analyses. Such data are currently very limited in the TSC population. The objective of this study was to generate HSUVs for TSC health states, defined by the number and type of seizures experienced in the previous week, and to compare with UK normative values. METHODS: This cross-sectional study involved 186 participants (individuals with TSC = 61, caregivers reporting for individuals with TSC = 125) from Europe and North America who completed a web-based survey. Participants completed the [EuroQol - 5 dimensions - 3 levels] (self-report version for individuals with TSC or proxy version 1 for caregivers). RESULTS: The mean age of individuals with TSC was 27.3 years (self-reported: 41.3 years, caregiver-reported: 20.5 years); 56% were males. Most individuals with TSC (71%) reported experiencing between one and ten seizures in the week prior to participating in the study. The most frequently reported type of seizure was focal: simple partial (50%). Across all participants (combined self-report and caregiver-report), the mean HSUV was 0.474 (95% confidence interval [CI]: 0.424-0.524), significantly lower than the UK norm (0.856, 95%CI: 0.848-0.864) [1]. Mean HSUV and HRQoL scores were consistently lower when reported by caregivers than when self-reported by individuals with TSC (HSUV = 0.351 vs. 0.727). This is in part because caregivers reported for individuals with TSC who experienced more frequent and severe seizures than those who were able to self-report. HSUVs incrementally decreased with the experience of more frequent (1-5 per week: HSUV = 0.666 vs. >20: HSUV = 0.290) and more severe seizures (focal: simple partial: HSUV = 0.450 vs. generalized: convulsive: HSUV = 0.194). CONCLUSIONS: The HRQoL and HSUV index scores indicate substantial impairment among individuals with TSC; HSUVs were shown to decrease considerably with increases in seizure frequency or seizure severity, indicating that more burdensome seizure health states are associated with poorer HRQoL.

Measuring Health-Related Quality of Life in Tuberous Sclerosis Complex - Psychometric Evaluation of Three Instruments in Individuals With Refractory Epilepsy.

Tuberous sclerosis complex (TSC) is a rare genetic disease associated with significant disease burden and considerable impact on health-related quality of life (HRQL). Currently no disease-specific clinical outcome assessments evaluate HRQL in individuals with TSC. A multi-center phase III study EXIST-3 (NCT01713946) assessed the efficacy and safety of two trough exposure ranges (Low exposure, LE: 3-7 ng/mL and high exposure, HE: 9-15 ng/mL) of adjunctive everolimus in patients aged 2-65 years with TSC and refractory partial-onset seizures (N = 366). Three age-specific HRQL measures were included as secondary endpoints including: quality of life in childhood epilepsy (QOLCE; caregiver-report for aged 2- < 11), the Quality of Life in Epilepsy Inventory for Adolescents-48 (QOLIE-AD-48; self-report, aged ≥ 11- < 18), and the Quality of Life in Epilepsy Inventory-31-Problems (QOLIE-31-P; self-report, aged ≥ 18). Intellectual ability was evaluated using the Wechsler Non-Verbal (WNV) Scale of Ability. Post hoc analyses were performed on the core phase primary data from EXIST-3 to evaluate the psychometric properties of the HRQL measures and calculate meaningful change estimates. Results showed that a significant subset of the trial sample (4-21 year olds) scored in the intellectual disability range, as assessed by the WNV. Psychometric analyses of the three epilepsy measures (including reliability, validity, and ability to detect change) supported the appropriateness for use in TSC. Distribution-based meaningful change estimates were generated for each HRQL measure, with estimates for the QOLIE-31-P total score largely consistent with the published literature. To our knowledge, this is the first evaluation using clinical trial data to establish the psychometric properties of the QOLCE, QOLIE-AD-48, and QOLIE-31-P for use in individuals with TSC. These findings increase confidence in the measures as valid and reliable for use in clinical trials and future research in patients with TSC.

Content Validity of a Willingness to Quit Tool for Use with Current Smokers in Clinical Practice.

INTRODUCTION: Despite reductions in rates of smoking in the past decade, smoking remains one of the most significant public health concerns. Quitting smoking can result in reductions in a number of serious health conditions. The brief Willingness to Quit (WTQ) tool can be used in routine clinical practice to assess current willingness to quit and engage a patient-physician dialogue regarding smoking cessation. The overall aim of this study was to validate the content of a WTQ tool for use with current smokers in clinical practice. METHODS: In-depth, qualitative interviews were conducted with 12 current smokers and five physicians. The interview was divided into two sections: concept elicitation (CE) followed by cognitive debriefing (CD). During CE, participants were asked questions exploring the different factors that can impact an individual's willingness to quit smoking. During CD, participants were given a copy of the WTQ tool and asked to comment on their level of understanding and interpretability of the items and the feasibility of completing the tool in clinical practice. RESULTS: All of the current smokers (n = 12) and physicians (n = 5) interviewed indicated that the items were understandable and relevant to assess willingness to quit. The tool was considered simple and suitable for use in clinical practice. CONCLUSION: The WTQ tool is a brief tool to assess willingness to quit and to engage communication between patients and physicians. All smokers should be offered smoking cessation support and facilitating a discussion on willingness to quit further supports a personalized quit plan. FUNDING: Pfizer Inc.

Anticipated and experienced discrimination amongst people with schizophrenia, bipolar disorder and major depressive disorder: a cross sectional study.

BACKGROUND: The unfair treatment of individuals with severe mental illness has been linked to poorer physical and mental health outcomes. Additionally, anticipation of discrimination may lead some individuals to avoid participation in particular life areas, leading to greater isolation and social marginalisation. This study aimed to establish the levels and clinical and socio-demographic associations of anticipated and experienced discrimination amongst those diagnosed with a schizophrenia and comparator severe mental illnesses (bipolar and major depressive disorders). METHODS: This study was a cross-sectional analysis of anticipated and experienced discrimination from 202 individuals in South London (47% with schizophrenia, 32% with depression and 20% with bipolar disorder). RESULTS: 93% of the sample anticipated discrimination and 87% of participants had experienced discrimination in at least one area of life in the previous year. There was a significant association between the anticipation and the experience of discrimination. Higher levels of experienced discrimination were reported by those of a mixed ethnicity, and those with higher levels of education. Women anticipated more discrimination than men. Neither diagnosis nor levels of functioning were associated with the extent of discrimination. Clinical symptoms of anxiety, depression and suspiciousness were associated with more experienced and anticipated discrimination respectively. CONCLUSIONS: The unfair treatment of individuals with severe mental illnesses remains unacceptably common. Population level interventions are needed to reduce levels of discrimination and to safeguard individuals. Interventions are also required to assist those with severe mental illness to reduce internalised stigma and social avoidance.