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The EQ Health and Well-being, EQ-HWB (25-item) and the EQ-HWB-S (9-item), are new generic measures of health and well-being. The purpose of this study was to examine the measurement properties of the EQ-HWB and EQ-HWB-S measures in relation to the EQ-5D-5L among the Italian general population. A cross-sectional survey was conducted from October 2020 to February 2021, followed by secondary analysis of collected data from Italian adults. This analysis included response pattern distributions, correlation strength, and known-group comparison. Known-group comparison was assessed using effect sizes (ES) across health conditions, caregiver status, and social care usage. The EQ-HWB-S index-based score was based on the UK pilot value set, whereas the Italian value set was used for scoring the EQ-5D-5L index. Out of the 1182 participants, 461 reported having a chronic condition, 185 identified as caregivers, and 42 were social care users. EQ-HWB items (7.5%) showed fewer ceiling effects than EQ-5D-5L items (34.7%). Strong correlations (r s > 0.5) were found between overlapping EQ-HWB and EQ-5D-5L items. EQ-HWB-S and EQ-5D-5L index scores demonstrated similar discrimination based on symptomatic chronic conditions (ES d = 0.68 vs d = 0.71), but EQ-HWB-S had slightly higher ES for social care users (ES d = 0.84 vs d = 0.74). Initial evidence supports the validity of EQ-HWB/EQ-HWB-S because outcome measures in the Italian population. EQ-HWB-S performed comparably to EQ-5D-5L among patients and was better in differentiating social care users. A slight decrease in discriminative properties for caregivers was observed when transitioning from EQ-HWB to EQ-HWB-S. • The EQ Health and Well-being, EQ-HWB (25-item) and EQ-HWB-S (9-item) were developed to meet the need for a single instrument applicable across different beneficiaries of health and social care services. The EQ-HWB is intended for use in clinical practice and population health surveys, whereas the EQ-HWB-S is designed for use in economic evaluations across health, social care, and public health. • This study aimed to assess the psychometric performance of the EQ-HWB/EQ-HWB-S in the general Italian population, including patients, caregivers, and social care users. It evaluated ceiling/floor effects, convergent validity, and discriminative ability against EQ-5D-5L. • Results support the validity of EQ-HWB/EQ-HWB-S as outcome measures in the Italian population. EQ-HWB-S performed comparably to EQ-5D-5L among patients and was better in differentiating social care users. There was a slight decrease in discriminative properties for caregivers was observed when transitioning from EQ-HWB to EQ-HWB-S. This study contributes to the growing body of literature on the EQ-HWB/EQ-HWB-S, highlighting its potential as a prominent measure in health and social care. [ABSTRACT FROM AUTHOR]

Objective: To derive a US-based value set for the EQ-5D-5L questionnaire using an international, standardized protocol developed by the EuroQol Group.Methods: Respondents from the US adult population were quota-sampled on the basis of age, sex, ethnicity, and race. Trained interviewers guided participants in completing composite time trade-off (cTTO) and discrete choice experiment (DCE) tasks using the EuroQol Valuation Technology software and routine quality control measures. Data were modeled using a Tobit model for cTTO data, a mixed logit model for DCE data, and a hybrid model that combined cTTO and DCE data. Model performance was compared on the basis of logical ordering of coefficients, statistical significance, parsimony, and theoretical considerations.Results: Of 1134 respondents, 1062, 1099, and 1102 respondents provided useable cTTO, DCE, and cTTO or DCE responses, respectively, on the basis of quality control criteria and interviewer judgment. Respondent demographic characteristics and health status were similar to the 2015 US Census. The Tobit model was selected as the preferred model to generate the value set. Values ranged from -0.573 (55 555) to 1 (11 111), with 20% of all predicted health states scores less than 0 (ie, worse than dead).Conclusions: A societal value set for the EQ-5D-5L was developed that can be used for economic evaluations and decision making in US health systems. The internationally established, standardized protocol used to develop this US-based value set was recommended by the EuroQol Group and can facilitate cross-country comparisons. [ABSTRACT FROM AUTHOR]

Purpose The objective of this study was to compare the health-related quality of life (HRQL) burden across different types of advanced cancer with the use of 2 widely used patient-reported outcome measures, the generic EQ-5D and the cancer-specific Functional Assessment of Cancer Therapy-General (FACT-G). Methods Patients with advanced cancer of the bladder, brain, breast, colon/rectum, head/neck, hepatobiliary tract/pancreas, kidney, lung, lymphoma, ovary, or prostate completed the EQ-5D and FACT-G. HRQL domains and summary scores were compared across types of cancer, using regression models to adjust for age and sex. Findings Approximately 50 patients with each type of cancer were recruited (total, 534 patients). According to EQ-5D dimensions, the highest proportion of problems was associated with prostate (mobility); prostate and kidney (self-care); head/neck, bladder, and lung (usual activities); breast, head/neck, and lung (pain/discomfort); and lymphoma and lung (anxiety/depression) cancers. EQ-5D visual analogue scale scores were lowest for breast and head/neck cancers; US index-based scores were lowest for breast and lung cancers and highest for brain cancer. For the FACT-G, physical well-being scores were lowest for head/neck, hepatobiliary, and kidney cancers; emotional well-being scores were lowest for hepatobiliary cancer, and functional well-being scores were lowest for head/neck, hepatobiliary, and bladder cancers. Implications The overall and dimension-specific HRQL burden of advanced cancer depended on the type of cancer. Such results may aid clinicians and patients in better understanding of how cancer site can affect HRQL and functioning in different ways. [ABSTRACT FROM AUTHOR]

Objectives: The EuroQol Group recently released youth (Y) and 5-level (5L) versions of its 3-level EQ-5D instrument (3L) that measures health-related quality of life. In this study, we (1) compare 3L, Y, and 5L responses among US adults and (2) assess construct validity. Study Design and Setting: Using a nationally representative sample of US adults (N 5 2,619), we collected 3L, Y, and 5L responses in random order and estimated their associations and their relationship with a 0 to 100 numerical visual analog scale. Results: The prevalence of US adults in the best possible EQ-5D state (i.e., 11111) was lower for the Y (38%) and 5L (35%) than for the 3L (44%), capturing more health problems. However, the prevalence of extreme responses in pain/discomfort and anxiety/depression decreased substantially between the 3L and 5L (from 44% to 17% and from 29% to 13%, respectively). Conclusion: Compared with the 3L, the Y and 5L versions describe population health as having more, yet milder, health problems. Although the 5L may have advantages in patient populations in which extreme problems are more prevalent, population studies or studies that follow patients from childhood may consider using the Y. [ABSTRACT FROM AUTHOR]

Abstract: Objective: Although utility-based algorithms have been developed for the Functional Assessment of Cancer Therapy (FACT), their properties are not well known compared with those of generic utility measures such as the EQ-5D. Our objective was to compare EQ-5D and FACT preference-based scores in cancer patients. Methods: A retrospective analysis was conducted on cross-sectional data collected from 472 cancer patients who completed both FACT-General and the EQ-5D. Preference-based scores were calculated by using published scoring functions for the EQ-5D (Dolan P. Modeling valuations for EuroQol health states. Med Care 1997;35:1095–108; Shaw JW, Johnson JA, Coons SJ. US valuation of the EQ-5D health states: development and testing of the D1 valuation model. Med Care 2005;43:203–20) and FACT (Dobrez D, Cella D, Pickard AS, et al. Estimation of patient preference-based utility weights from the Functional Assessment of Cancer Therapy-General. Value Health 2007;10:266–72; Kind P, Macran S. Eliciting social preference weights for Functional Assessment of Cancer Therapy-Lung health states. Pharmacoeconomics 2005;23:1143–53; Cheung YB, Thumboo J, Gao F, et al. Mapping the English and Chinese versions of the Functional Assessment of Cancer Therapy-General to the EQ-5D utility index. Value Health 2009;12:371–6). Scores were compared on the basis of clinical severity by using Eastern Cooperative Oncology Group performance status ratings by physicians and patients. Relative efficiency of each scoring function was examined by using ratios of F statistics. Results: Mean scores for the overall cohort were lowest when using Kind and Macran''s FACT UK societal algorithm (0.55, SD 0.09) and highest when using Dobrez et al.''s FACT US patient algorithm (0.83, SD 0.08). Mean difference scores associated with clinical severity, when extrapolated to quality-adjusted life-years (QALYs), had a range of 0.18 QALYs gained using FACT (Kind and Macran) to 0.45 QALYs gained using the EQ-5D (Dolan). However, relative efficiencies suggested that FACT (Kind and Macran) scores may provide greater statistical power to detect significant differences based on clinical severity. Conclusions: We found important differences in utilities scores estimated by each algorithm, with FACT-based algorithms tending to underestimate the QALY benefit compared with algorithms based on the EQ-5D. These differences highlight some of the challenges in using disease-specific preference-based measures for decision making despite potentially more relevant disease-specific content. [Copyright &y& Elsevier]

Objective: The D1 model that was developed to predict US societal preferences for EQ-5D health states addressed several important conceptual and statistical issues. However, it has been criticized for being too complex, failing to account for the nonnormal distribution of health state values, and the transformation of preferences for worse-than-death health states before estimation. This research was conducted to develop an improved model for predicting median preferences for EQ-5D health states for the US population. Methods: Probability-weighted least absolute deviations regression was used to fit models to the time trade-off data collected in the US Valuation of the EQ-5D Health States study. No transformation was applied to the values for states considered worse than death. Several model specifications that differed with respect to explanatory variables were evaluated using two-sample cross-validation. Results: The best-fitting model included only fixed effects for moderate or severe problems in each of the 5 EQ-5D dimensions and excluded a constant. This specification yielded rank correlations between observed and predicted values and median observed and predicted values of 0.635 and 0.991, respectively, as well as a median absolute error of 0.026. The predicted median preferences ranged from 1.00 for full health, to –0.81 for the worst possible health state. Conclusions: Due to its simplicity and robustness, a median model is superior to other models for predicting US population preferences for EQ-5D health states. The predictions of this model are suggested for use in applications that require US societal health state values. [ABSTRACT FROM AUTHOR]

Background: The European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (QLQ-C30) is a widely used quality-of-life measure in oncology. The ability to translate QLQ-C30 responses into utility scores would further expand its use in medical decision-making. The aims of this study were to: 1) map QLQ-C30 responses onto patient time trade-off utility scores; and 2) compare a multiattribute approach to a global evaluation approach to modeling utility scores. Methods: Two distinct approaches were applied to data from 1432 cancer patients. The multiattribute approach used psychometric analysis and expert input to select a subset of functioning and symptom scale items for modeling. The second approach focused on global health and quality-of-life items based on a conceptual model. Model selection criteria included parsimony, statistical significance and logical consistency of parameter estimates, predictive accuracy, number of states described, and scale range. Results: The optimal multiattribute model included nine variables for five items from different scales, described 144 unique states, predicted values ranging from 0.63 to 1.00, but it had poor predictive accuracy (cross-validation pseudo- R2 = 0.056). The best-fitting global approach-based model described 24 unique states using eight indicators for two items from one scale (plus a constant) and predicted values ranging from 0.17 to 1.00 (cross-validation pseudo- R2 = 0.127). Conclusions: Multiattribute models produced a greater number of unique predicted values, while global models exhibited more desirable statistical properties and a wider range of values. The recommended models will enable users to predict cancer patients’ utilities from existing and future QLQ-C30 data sets. [ABSTRACT FROM AUTHOR]

Abstract: Objectives: Informal caregivers often serve as proxy raters of Health-Related Quality of Life (HRQL) when patients cannot report on their own behalf. Caregiver depression has been associated with bias in proxy ratings, but few studies have examined the role of caregiver pain. The aim of this study was to determine if caregiver depressive symptoms and/or pain systematically affected patient–proxy agreement on patient HRQL after stroke. Study Design and Settings: Secondary data analysis of 95 stroke patients and their caregivers (dyads) at 6 months poststroke. Caregiver depressive symptoms were measured by Center for Epidemiologic Studies Depression Rating Scale, and pain was measured using EQ-5D. Using multivariate regression, we examined the effect of caregiver depressive symptoms and pain on patient–proxy difference scores on Health Utilities Index 3 (HUI3) attributes and Short Form-36 domains of vitality and social functioning. Results: Caregiver depressive symptoms and pain were associated with significant differences in patient–proxy scores on HUI3 pain (P <0.05). Depressed caregivers underestimated pain experienced by patients, and caregivers with pain overestimated patient pain. Additionally, an interaction between caregiver depressive symptoms and pain was identified. Conclusion: Presence of pain and depressive symptoms in caregivers can significantly affect perceptions of pain in stroke patients. Results suggest that caregivers with pain and/or depression may provide more biased proxy assessments of pain. [Copyright &y& Elsevier]

Purpose: Diseases are often described and studied in isolation, yet there is increasing recognition of the complex interrelatedness of diseases and treatments in patients with multiple chronic diseases. Our objective was to describe the impact of selected diseases involving chronic inflammation (chronic obstructive pulmonary disease [COPD], osteoarthritis, and rheumatoid arthritis) on mortality. Methods: We identified a cohort aged 55 to 64 years with one or more chronic conditions. Clusters of mutually exclusive disease combinations were created. Five-year all-cause mortality was determined and the relative risk (RR) of mortality was estimated when COPD, osteoarthritis, and rheumatoid arthritis were added to clusters. Results: In 741,847 persons the 5-year mortality rates were lowest among persons with one condition and increased with more chronic conditions. The presence of osteoarthritis in a cluster was an exception where the risk was lower compared with that cluster without osteoarthritis: COPD (RR = 0.73 [95% confidence interval (CI), 0.65, 0.81]); ischemic heart disease (0.63 [0.52, 0.76]); hypertension (0.77 [0.71, 0.83]); dementia (0.63 [0.42, 0.93]); depression (0.65 [0.50, 0.84]); hypertension plus diabetes (0.85 [0.77, 0.93]); and ischemic heart disease plus hypertension (0.83 [0.73, 0.94]). Conclusions: The association between osteoarthritis and lower rates of mortality is notable and replicating these findings to explore causal relationships is important. [Copyright &y& Elsevier]

Objectives: Clinical trials and community-based studies often include the Center for Epidemiologic Studies-Depression scale (CES-D) as a measure of depression outcome. We compared responses to symptom-related items on the CES-D by depressed stroke and primary-care patients for several purposes: 1) to illustrate the use of Item Response Theory (IRT)-based (Rasch) models for comparing scale functioning across different patient subgroups; and 2) to inform clinicians and outcome researchers about scale functioning and depressive symptomatology in stroke- compared with primary care-based depression. Methods: Two data sources were analyzed, including 32 depressed patients who were 3 months poststroke, and 366 depressed primary-care patients. Presence of depression was based on a CES-D score 16 or higher. Rasch models were used to assess item fit and compare item hierarchies between depressed primary-care and stroke patients. Results: Item hierarchies were similar for poststroke depression and primary care-based depression. Interpersonal disruption items were the most difficult to endorse for both groups. No items misfit the scale in primary-care depression. Items relating to restless sleep, unfriendliness, and crying slightly misfit the scale in stroke patients, that is, may measure a different trait. Differential item functioning (DIF) between the groups was identified for items relating to appetite, restless sleep, crying, and feeling disliked. Conclusions: Results generally supported the use of the CES-D as measure of depression outcome, particularly in primary care-based depression. DIF may imply that slightly different clusters of depressive symptoms are reported by depressed stroke patients compared with primary care, but this is conjectural given the small stroke sample size and the same items have been previously associated with bias in studies of large nonstroke samples. This study found Rasch models to be useful tools to investigate scale performance for different clinical applications. [ABSTRACT FROM AUTHOR]

Alzheimer's disease is an irreversible neurodegenerative disease, which accounts for most dementia cases. Neuroinflammation is increasingly recognised for its roles in Alzheimer's disease pathogenesis which, in part, links amyloid-beta to neuronal death. Neuroinflammatory signalling can be exhibited by neurons themselves, potentially leading to widespread neuronal cell death, although neuroinflammation is commonly associated with glial cells. The presence of the inflammasomes such as nucleotide-binding leucine-rich repeat receptors protein 1 in neurons accelerates amyloid-beta -induced neuroinflammation and has been shown to trigger neuronal pyroptosis in murine Alzheimer's disease models. However, the pathways involved in amyloid-beta activation of inflammasomes have yet to be elucidated. In this study, a gene trap mutagenesis approach was utilised to resolve the genes functionally involved in inflammasome signalling within neurons, and the mechanism behind amyloid-beta-induced neuronal death. The results indicate that amyloid-beta significantly accelerated neuroinflammatory cell death in the presence of a primed inflammasome (the NLR family pyrin domain-containing 1). The mutagenesis screen discovered the atypical mitochondrial Ras homolog family member T1 as a significant contributor to amyloid-beta-induced inflammasome -mediated neuronal death. The mutagenesis screen also identified two genes involved in transforming growth factor beta signalling, namely Transforming Growth Factor Beta Receptor 1 and SNW domain containing 1. Additionally, a gene associated with cytoskeletal reorganisation, SLIT-ROBO Rho GTPase Activating Protein 3 was found to be neuroprotective. In conclusion, these genes could play important roles in inflammasome signalling in neurons, which makes them promising therapeutic targets for future drug development against neuroinflammation in Alzheimer's disease. [ABSTRACT FROM AUTHOR]

Several measures have been used or developed to capture the health and well-being of caregivers, including the EQ Health and Well-being (EQ-HWB) and its short form, EQ-HWB-S. This study aimed to evaluate the psychometric properties and construct validity of the EQ-HWB/EQ-HWB-S in a US caregiver population. A cross-sectional survey was conducted involving 504 caregivers. Eligible participants were 18+ years old, provided unpaid care to a relative/friend aged 18+ in the past 6 months, and spent on average of at least 1 hour per week caregiving. Survey included the following measures: EQ-HWB, Adult Social Care Outcomes Toolkit for Carers-Carer, CarerQol, and EQ-5D-5L. Psychometric properties were assessed using response distributions, floor/ceiling effects, Spearman's correlation for convergent validity, and effect sizes (ES) for known-group validity based on caregiving situations and intensity. The average age of caregivers was 49.2 (SD = 15.4), with 57.5% being female. More than half (54.4%) reported high caregiving intensity, and 68.3% lived with the care recipient. The EQ-HWB-S index showed a strong positive correlation with the EQ-5D-5L (r s = 0.72), Adult Social Care Outcomes Toolkit for Carers (r s = 0.54), and CarerQol (r s = 0.54) indices. Notably, the EQ-HWB-S index showed the largest ES among measures in differentiating caregiving scenarios with a large ES for caregiver's general health (d = 1.00) and small ES for caregiving intensity (d = 0.39). Results support construct validity of EQ-HWB and EQ-HWB-S as measures for assessing health and well-being of adult informal caregivers in comparison with other validated instruments. Differing levels of known-group validity across anchors emphasize the importance of selecting appropriate measures for caregivers, depending on research question and/or intervention aims. • Various preference-based measures, including Adult Social Care Outcomes Toolkit for Carers-Carer, CarerQol-7D, and EQ-5D-5L, can be used to assess the health and quality of life of informal caregivers. The EQ Health and Well-being (EQ-HWB) was developed as a broader measure of health and well-being, including for caregivers. However, its psychometric properties have yet to be examined in relation to other validated measures, specifically in caregivers. • This study evaluated the psychometric properties of EQ-HWB and its short form, EQ-HWB-S, in a heterogeneous US caregiver population, demonstrating construct validity with other preference-based measures. Notably, care recipients' self-perceived burden to caregivers proved to be a valid anchor-based criterion for distinguishing groups based on the influence of caregiving on their health and well-being. • The findings highlight the importance of selecting appropriate measures for assessing health and well-being of informal caregivers. This choice should depend on the specific aims of the intervention being evaluated and perspective of economic evaluation. Using the appropriate measures can lead to more accurate assessments of the effectiveness and cost-effectiveness of interventions with impacts on broader quality of life and well-being, which is essential for informed decision making in healthcare policy. [ABSTRACT FROM AUTHOR]

Objectives: The EQ Health and Wellbeing Short (EQ-HWB-S) is a new broad generic measure of health and wellbeing for use in economic evaluations of interventions across healthcare, social care, and public health. This measure conceptually overlaps with the 5-level version EQ-5D (EQ-5D-5L), while expanding on the coverage of health and social care related dimensions. This study aims to examine the extent to which the EQ-HWB-S and EQ-5D-5L overlap and are different.Methods: A sample of US-based respondents (n = 903; n = 400 cancer survivors and n = 503 general population) completed a survey administered via an online panel. The survey included the EQ-HWB item pool (62 items, including 11 items used in this analysis), EQ-5D-5L, and questions about sociodemographic and health characteristics. The analysis included (Spearman's) correlations, the comparison of patterns of response (distributions and ceiling effects), and the ability to discriminate between known groups.Results: Moderate to strong associations were found between conceptually overlapping dimensions of the EQ-5D-5L and the EQ-HWB-S (rs > 0.5, P < .001). Among respondents reporting full health on the EQ-5D-5L (n = 161, 18.23%), the EQ-HWB-S identified ceiling effects, particularly with the item "feeling exhausted." Most EQ-5D-5L and EQ-HWB-S items demonstrated discriminative ability among those with and without physical and mental conditions, yielding medium (> 0.5) to large effect sizes (> 0.8). Nevertheless, only EQ-HWB-S items distinguished between caregivers and noncaregivers and those with low and high caregiver burden, albeit with small effect sizes (0.2-0.5).Conclusions: Results indicate a convergence between the measures, especially between overlapping dimensions, lending support to the validity of the EQ-HWB-S. The EQ-HWB-S performed similarly or better than the EQ-5D-5L among patient groups and is better able to differentiate among caregivers and respondents closer to full health. [ABSTRACT FROM AUTHOR]

This study assessed the content validity of generic and condition-specific preference-based measures (PBMs) with patients treated for cancer, evaluated against 10 Consensus-Based Standards for the Selection of Health Measurement Instruments criteria for good content validity, to best inform measurement strategies regarding the use of PBMs in oncology development programs and real-world applications. Individual, semistructured interviews were conducted with patients who received drug treatment for cancer in the United Kingdom (n = 47) and the United States (n = 49). During the interview, patients completed 3 generic PBMs (EQ-5D-5L, EuroQol Health and Wellbeing measure–Short Form, Château Santé Base) and 2 condition-specific PBMs (Quality of Life Utility-Core 10 Dimension, Functional Assessment of Cancer Therapy Eight Dimension [FACT-8D]). Interviews were conducted via teleconference, audio recorded, and transcribed verbatim. Transcripts were coded using thematic and content analysis methods. Condition-specific measures were evaluated as having better relevancy than generic PBMs. Overall, the FACT-8D was evaluated as holding the best content validity in terms of relevancy, and the EuroQol Health and Wellbeing measure–Short Form received the most favorable evaluation of relevancy for generic PBMs. All measures demonstrated comparable comprehensiveness, with all suggested by patients to be missing concepts. The EQ-5D-5L was evaluated best in terms of comprehensibility. This was followed by the Quality of Life Utility-Core 10 Dimension and FACT-8D; both received similar evaluations. All measures were generally seen by patients as adequate in capturing appropriate aspects of health-related quality of life for measuring cancer outcomes, although together condition-specific measures were evaluated as having better relevancy than generic PBMs. Further health-related quality of life instrument development is encouraged, particularly with regard to the longer-term detrimental impacts of cancer and treatment side effects. Other developments could include new cancer-specific tools inclusive of conventional health items, treatment impacts, and psychological items. • Generic preference-based measures (PBMs) are recommended to assess patient-reported outcomes across interventions and to allow for comparative assessments across unrelated health conditions and interventions. Generic PBMs may exclude dimensions relevant to specific conditions, whereas condition-specific PBMs can contain items related to specific symptoms. Recent work has identified several concepts that are relevant to health-related quality of life and psychological wellbeing, which are not measured by existing outcome measures. • Patients who had received drug treatment for cancer in the United Kingdom and the United States evaluated 3 generic and 2 condition-specific PBMs during qualitative interviews. All measures demonstrated comparable comprehensiveness, missing concepts considered by patients as important to measure for their cancer and cancer treatment experiences. Language and format were frequently challenged, with patients suggesting changes to multiple items across all measures. Recall periods were frequently raised as problematic because of the substantial variation in cancer symptoms within a given day or week. • Given the burden of cancer symptoms and drug treatment effects, capturing all relevant outcomes of people with cancer is necessary to inform accurate decision making for cancer technologies. Modification of existing measures should be considered with regard to measurement gaps identified. New cancer-specific tools could be considered, inclusive of conventional health items, treatment impacts, and psychological items. These developments have a large potential for impact given the high proportions of oncology health technology assessment submissions. [ABSTRACT FROM AUTHOR]

OFF Episodes occur in people with Parkinson's disease when their medication wears off, and motor and/or non-motor symptoms emerge. Existing measures used to assess OFF Episodes focus on the time spent in OFF Episodes through diaries or by identifying symptoms, but they are limited in their ability to capture the severity and functional impact of OFF episodes. The aim of this study was to develop and validate a new instrument, called "OFFELIA," that measures the impact of OFF episodes on the quality of life of individuals with Parkinson's disease. Participants completed a cross-sectional questionnaire, "Impact and Communication on OFF Periods," while enrolled in the online clinical study Fox Insights. The data collected was used to develop OFFELIA. Psychometric testing was performed on 18 candidate items using classical, exploratory factor analysis, and item response theory methods. 569 individuals with Parkinson's disease completed the questionnaire. All items were retained for the final measure, with 17 items aggregated into two multi-item scales (functioning and psychological well-being) and one item reported separately as it did not function well with the other items (employment). Known group comparisons based on average duration, frequency and unpredictability of OFF episodes indicated that OFFELIA subscales were more sensitive than existing generic and condition-specific measures. Initial evidence supports the validity of OFFELIA, a new instrument that assesses the impact of OFF periods on daily life. This instrument can be used in assessing clinical therapeutic strategies targeting OFF episodes in Parkinson's disease. • OFFELIA is a new patient-reported outcome measure for Parkinson's Disease. • OFFELIA assesses off episodes' impact on quality of life in people with Parkinson's. • Evidence supports validity and reliability of OFFELIA. • OFFELIA aids clinicians assessing treatment's effect on off episodes. • OFFELIA measures impact of interventions on off episodes in trials. [ABSTRACT FROM AUTHOR]

Objectives: Existing measures for estimating quality-adjusted life-years are mostly limited to health-related quality of life. This article presents an overview of the development the EQ-HWB (EQ Health and Wellbeing), which is a measure that encompasses health and wellbeing.Methods: Stages: (1) Establishing domains through reviews of the qualitative literature informed by a conceptual framework. (2) Generation and selection of items to cover the domains. (3) Face validation of these items through qualitative interviews with 168 patients, social care users, general population, and carers across 6 countries (Argentina, Australia, China, Germany, United Kingdom, United States). (4) Extensive psychometric testing of candidate items (using classical, factor analysis, and item response theory methods) on > 4000 respondents in the 6 countries. Stakeholders were consulted throughout.Results: A total of 32 subdomains grouped into 7 high-level domains were identified from the qualitative literature and 97 items generated to cover them. Face validation eliminated 36 items, modified 14, and added 3. Psychometric testing of 64 items found little difference in missing data or problems with response distribution, the conceptual model was confirmed except in China, and most items performed well in the item response theory in all countries. Evidence was presented to stakeholders in 2 rounds of consultation to inform the final selection of items for the EQ-HWB (25-item) and the short version of EQ-HWB (9-items).Conclusions: EQ-HWB measures have been developed internationally for evaluating interventions in health, public health, and social care including the impact on patients, social care users, and carers. [ABSTRACT FROM AUTHOR]

Objectives: The development of measures such as the EQ-HWB (EQ Health and Wellbeing) requires selection of items. This study explored the psychometric performance of candidate items, testing their validity in patients, social carer users, and carers.Methods: Article and online surveys that included candidate items (N = 64) were conducted in Argentina, Australia, China, Germany, United Kingdom, and the United States. Psychometric assessment on missing data, response distributions, and known group differences was undertaken. Dimensionality was explored using exploratory and confirmatory factor analysis. Poorly fitting items were identified using information functions, and the function of each response category was assessed using category characteristic curves from item response theory (IRT) models. Differential item functioning was tested across key subgroups.Results: There were 4879 respondents (Argentina = 508, Australia = 514, China = 497, Germany = 502, United Kingdom = 1955, United States = 903). Where missing data were allowed, it was low (UK article survey 2.3%; US survey 0.6%). Most items had responses distributed across all levels. Most items could discriminate between groups with known health conditions with moderate to large effect sizes. Items were less able to discriminate across carers. Factor analysis found positive and negative measurement factors alongside the constructs of interest. For most of the countries apart from China, the confirmatory factor analysis model had good fit with some minor modifications. IRT indicated that most items had well-functioning response categories but there was some evidence of differential item functioning in many items.Conclusions: Items performed well in classical psychometric testing and IRT. This large 6-country collaboration provided evidence to inform item selection for the EQ-HWB measure. [ABSTRACT FROM AUTHOR]

Objective: The goal of this study was to estimate an algorithm to convert responses to the Functional Assessment of Cancer Therapy—General (FACT-G) to time trade-off (TTO) utilities based on utilities for current health elicited from cancer patients. Methods: Data for 1433 cancer patients were randomly separated into construction and validation samples. Four FACT-G questions were selected for inclusion based on correlation with Eastern Clinical Oncology Group—Performance Status (ECOG-PS) scores and TTO utilities. Item response theory was used to collapse response categories. Ordinary least squares regression with the constant constrained to one was used to estimate the algorithm. Results: The algorithm estimated mean utility for the full validation sample within three points of observed mean utility (0.805 vs. 0.832, P < 0.01). Mean utilities were wellpredicted (mean absolute difference < 0.03, P > 0.05) for most subgroups defined by ECOG-PS and Short Form-36 physical functioning scores, and responses to the FACT-G overall quality of life item. Nevertheless, the algorithm systematically overpredicted utilities for poorer health states. Conclusions: A FACT-G-based algorithm of cancer patient utilities was developed that estimates group mean utility scores with accuracy comparable to other indirect preference-based measures of health-related quality of life. Patient-based preferences for health outcomes of cancer treatment may be useful in multiple situations, such as managing resources within cancer centers and in understanding health states preferences among cancer experienced patients before and after treatment. [ABSTRACT FROM AUTHOR]

Background: Four visual analog scales were constructed to assess sensory and affective components of operative pain. The Surgical Pain Scales (SPS) measure pain while at rest, pain during normal activities, pain during work or exercise, and pain unpleasantness. Study design: Longitudinal data from 2,164 patients in a randomized trial of laparoscopic versus open hernia repair established the reliability, validity, and sensitivity to change of the SPS. Correlations and t-tests were used to determine their psychometric properties compared with the SF-36 health status instrument. Results: Intraclass correlation coefficients of 0.95 for the sensory scales and 0.94 for the unpleasantness rating confirmed that the SPS produced reliable measurements. Correlations ranging from 0.44 to 0.60 between the visual analog scales and the bodily pain dimension on the SF-36 and significant differences between SPS levels for patients requiring more and less time to resume normal activities (p≤0.015 to p≤0.002) supported the validity of the scales. Clinical responsiveness was demonstrated by a 33.5-mm reduction (standard error = 1.4 mm) in the mean rating on a 150-mm scale measuring pain during normal activities for patients reporting postoperative improvement on the bodily pain dimension (p≤0.0001). Conclusions: The Surgical Pain Scales demonstrated excellent psychometric properties in this study population. The SPS can be used to compare pain levels between groups at a single point in time or to track change for individual patients over time or after operations. Individualized pain management interventions can be tailored based on the sensory and effective ratings. [Copyright &y& Elsevier]

Objectives: To develop a cancer-specific multi-attribute utility instrument derived from the Functional Assessment of Cancer Therapy - General (FACT-G) health-related quality of life (HRQL) questionnaire.Methods: We derived a descriptive system based on a subset of the 27-item FACT-G. Item selection was informed by psychometric analyses of existing FACT-G data (n = 6912) and by patient input (n = 82). We then conducted an online valuation survey, with participants recruited via an Australian general population online panel. A discrete choice experiment (DCE) was used, with attributes being the HRQL dimensions of the descriptive system and survival duration, and 16 choice-pairs per participant. Utility decrements were estimated with conditional logit and mixed logit modeling.Results: Eight HRQL dimensions were included in the descriptive system: pain, fatigue, nausea, sleep, work, social support, sadness, and future health worry; each with 5 levels. Of 1737 panel members who accessed the valuation survey, 1644 (95%) completed 1 or more DCE choice-pairs and were included in analyses. Utility decrements were generally monotonic; within each dimension, poorer HRQL levels generally had larger utility decrements. The largest utility decrements were for the highest levels of pain (-0.40) and nausea (-0.28). The worst health state had a utility of -0.54, considerably worse than dead.Conclusions: A descriptive system and preference-based scoring approach were developed for the FACT-8D, a new cancer-specific multi-attribute utility instrument derived from the FACT-G. The Australian value set is the first of a series of country-specific value sets planned that can facilitate cost-utility analyses based on items from the FACT-G and related FACIT questionnaires containing FACT-G items. [ABSTRACT FROM AUTHOR]

Objectives: The wording of the Hungarian EQ-5D-3L and EQ-5D-5L descriptive systems differ a great deal. This study aimed to (1) develop EQ-5D-3L and EQ-5D-5L value sets for Hungary from a common sample, and (2) compare how level wording affected valuations.Methods: In 2018 to 2019, 1000 respondents, representative of the Hungarian general population, completed composite time trade-off tasks. Pooled heteroscedastic Tobit models were used to estimate value sets. Value set characteristics, single-level transition utilities from adjacent corner health states, and mean transition utilities for all possible health states were compared between the EQ-5D-3L and EQ-5D-5L.Results: Health utilities ranged from -0.865 to 1 for the EQ-5D-3L and -0.848 to 1 for the EQ-5D-5L. The relative importance of the 5 EQ-5D-5L dimensions was as follows: mobility, pain/discomfort, self-care, anxiety/depression, and usual activities. A similar preference ranking was observed for the EQ-5D-3L with self-care being more important than pain/discomfort. The EQ-5D-5L demonstrated lower ceiling effects (range of utilities for the mildest states: 0.900-0.958 [3L] vs 0.955-0.965 [5L]) and better consistency of mean transition utilities across the range of scale. Changing "confined to bed" (3L) to "unable to walk" (5L) had a large positive impact on utilities. Smaller changes with more negative wording in the other dimensions (eg, "very much anxious/feeling down a lot" [3L] vs "extremely anxious/depressed" [5L]) had a modest negative impact on utilities.Conclusion: This study developed value sets of the EQ-5D-3L and EQ-5D-5L for Hungary. Our findings contribute to the understanding of how the wording of descriptive systems affects the estimates of utilities. [ABSTRACT FROM AUTHOR]

Cost-effectiveness models that present results in terms of cost per quality-adjusted life-year for health technologies are used to inform policy decisions in many parts of the world. Health state utilities (HSUs) are required to calculate the quality-adjusted life-years. Even when clinical studies assessing the effectiveness of health technologies collect data on HSUs to populate a cost-effectiveness model, which rarely happens, analysts typically need to identify at least some additional HSUs from alternative sources. When possible, HSUs are identified by a systematic review of the literature, but, again, this rarely happens. In 2014, ISPOR established a Good Practices for Outcome Research Task Force to address the use of HSUs in cost-effectiveness models. This task force report provides recommendations for researchers who identify, review, and synthesize HSUs for use in cost-effectiveness models; analysts who use the results in models; and reviewers who critically appraise the suitability and validity of the HSUs selected for use in models. The associated Minimum Reporting Standards of Systematic Review of Utilities for Cost-Effectiveness checklist created by the task force provides criteria to judge the appropriateness of the HSUs selected for use in cost-effectiveness models and is suitable for use in different international settings. [ABSTRACT FROM AUTHOR]

Background Discrete choice experiments (DCEs) are increasingly used to value aspects of health. An issue with their adoption is that results may be sensitive to the order in which dimensions of health are presented in the valuation task. Findings in the literature regarding order effects are discordant at present. Objectives To quantify the magnitude of order effect of quality-of-life (QOL) dimensions within the context of a DCE designed to produce country-specific value sets for the EORTC Quality of Life Utility Measure-Core 10 dimensions (QLU-C10D), a new utility instrument derived from the widely used cancer-specific QOL questionnaire, the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30. Methods The DCE comprised 960 choice sets, divided into 60 versions of 16 choice sets, with each respondent assigned to a version. Within each version, the order of QLU-C10D QOL dimensions was randomized, followed by life duration in the last position. The DCE was completed online by 2053 individuals in France and Germany. We analyzed the data with a series of conditional logit models, adjusted for repeated choices within respondent. We used F tests to assess order effects, correcting for multiple hypothesis testing. Results Each F test failed to reject the null hypothesis of no position effect: 1) all QOL order positions considered jointly; 2) last QOL position only; 3) first QOL position only. Furthermore, the order coefficients were small relative to those of the QLU-C10D QOL dimension levels. Conclusions The order of presentation of QOL dimensions within a DCE designed to provide utility weights for the QLU-C10D had little effect on level coefficients of those QOL dimensions. [ABSTRACT FROM AUTHOR]

Allogeneic hematopoietic stem cell transplantation (HSCT) is rarely performed in adult patients with sickle cell disease (SCD). We utilized the chemotherapy-free, alemtuzumab/total body irradiation 300 cGy regimen with sirolimus as post-transplantation immunosuppression in 13 high-risk SCD adult patients between November 2011 and June 2014. Patients received matched related donor (MRD) granulocyte colony–stimulating factor–mobilized peripheral blood stem cells, including 2 cases that were ABO incompatible. Quality-of-life (QoL) measurements were performed at different time points after HSCT. All 13 patients initially engrafted. A stable mixed donor/recipient chimerism was maintained in 12 patients (92%), whereas 1 patient not compliant with sirolimus experienced secondary graft failure. With a median follow-up of 22 months (range, 12 to 44 months) there was no mortality, no acute or chronic graft-versus-host disease (GVHD), and no grades 3 or 4 extramedullary toxicities. At 1 year after transplantation, patients with stable donor chimerism have normalized hemoglobin concentrations and improved cardiopulmonary and QoL parameters including bodily pain, general health, and vitality. In 4 patients, sirolimus was stopped without rejection or SCD-related complications. These results underscore the successful use of a chemotherapy-free regimen in MRD HSCT for high-risk adult SCD patients and demonstrates a high cure rate, absence of GVHD or mortality, and improvement in QoL including the applicability of this regimen in ABO mismatched cases (NCT number 01499888). [ABSTRACT FROM AUTHOR]

Objectives There is limited understanding of the effects of bleeding episodes on the daily lives of patients with congenital hemophilia with inhibitors and their caregivers. This analysis of the Dosing Observational Study in Hemophilia examined the impact of acute bleeding episodes on work, school, and family activities. Methods Patients and caregivers participated in a diary study for 90 or more days or until patients experienced four bleeding episodes. All bleed treatments, interference with daily activities, and quality-of-life assessments were captured in daily records. Patients and caregivers reported planned workdays or school days eligible to be “lost” so as to differentiate from days lost because of disability or nonworking status, weekends, and vacations. Results Diaries were completed for 39 patients (18 adults and 21 children). Bleeding episodes that continued for 3 or more days (16.4%) accounted for most of the major changes to family plans. For the 38 patients with bleeding episodes, 47% of 491 bleed days fell on planned workdays or school days; the remainder fell on weekends, holidays, or nonworkdays or non–school days and therefore did not count as “lost days.” Patients reported a loss of productivity on a greater percentage of eligible bleed days than did caregivers (3.9% vs. 0.8%, respectively). Patients and caregivers reported 13.5%/9.3% fully missed and 3.5%/7.6% partially missed days. Conclusions This study demonstrated that in hemophilia with inhibitors, bleeding episodes interfere with the daily activities of patients and their caregivers. Furthermore, documenting only lost days underestimated the impact of bleeding episodes because of the high percentage of days without planned work or school. [ABSTRACT FROM AUTHOR]

Abstract: Introduction: Quality-of-life (QOL) assessments in frequently bleeding patients with congenital hemophilia with inhibitors and their families are confounded by preexisting arthropathy and family circumstances. Periodic QOL assessments typically made on nonbleed days may not provide complete reflections of the burden on patients/families. Aim: To evaluate the impact of bleeding episodes on patients/caregivers/families and the association between monthly QOL scores and patients'' average diary experiences. Methods: Frequently bleeding inhibitor patients (≥four bleeds in 3 months), or their caregivers, provided daily assessment of EuroQol five-dimensional questionnaire and EuroQol five-dimensional questionnaire visual analogue scale, pain (11-point Likert scale), and family anxiety/stress/activity change over 3 to 6 months. QOL scores were stratified by bleed/nonbleed days. Results: Patient QOL assessments were recorded by 37 of the 39 enrolled patients/caregivers (3771 of 3777 eligible dairy days, 472 bleed/3299 nonbleed days). Median (range) diary duration was 91 (66–180) days, with 8.2% (0%–72.2%) bleed days. Mean health scores were significantly worse on bleed days than on nonbleed days (P < 0.0001 for all): EuroQol five-dimensional questionnaire index, 0.66 versus 0.82; visual analogue scale health, 69.7 versus 77.4; and pain score, 4.1 versus 1.8. Bleed days also had higher (P < 0.001) proportions of days with abnormalities in family anxiety/stress (42% vs. 30%) and family activity changes (34% vs. 21%). Conclusions: Assessing the impact of hemophilia with inhibitors on patient/family QOL typically includes periodic (likely nonbleed day) evaluations reflecting baseline abnormalities. Daily assessment, however, indicated that frequent acute bleeds impair QOL beyond patient''s nonbleed day baseline. New approaches are required to assess the cumulative impact of frequent acute bleeds on patients and their families. [Copyright &y& Elsevier]

Abstract: Background: A five-level version of the EuroQol five-dimensional (EQ-5D) descriptive system (EQ-5D-5L) has been developed, but value sets based on preferences directly elicited from representative general population samples are not yet available. The objective of this study was to develop values sets for the EQ-5D-5L by means of a mapping (“crosswalk”) approach to the currently available three-level version of the EQ-5D (EQ-5D-3L) values sets. Methods: The EQ-5D-3L and EQ-5D-5L descriptive systems were coadministered to respondents with conditions of varying severity to ensure a broad range of levels of health across EQ-5D questionnaire dimensions. We explored four models to generate value sets for the EQ-5D-5L: linear regression, nonparametric statistics, ordered logistic regression, and item-response theory. Criteria for the preferred model included theoretical background, statistical fit, predictive power, and parsimony. Results: A total of 3691 respondents were included. All models had similar fit statistics. Predictive power was slightly better for the nonparametric and ordered logistic regression models. In considering all criteria, the nonparametric model was selected as most suitable for generating values for the EQ-5D-5L. Conclusions: The nonparametric model was preferred for its simplicity while performing similarly to the other models. Being independent of the value set that is used, it can be applied to transform any EQ-5D-3L value set into EQ-5D-5L index values. Strengths of this approach include compatibility with three-level value sets. A limitation of any crosswalk is that the range of index values is restricted to the range of the EQ-5D-3L value sets. [Copyright &y& Elsevier]

Background: Improvement in day-to-day functioning is a valued outcome of surgical intervention. A new functional status assessment instrument, the Activities Assessment Scale (AAS), was designed for a randomized clinical trial evaluating laparoscopic versus open hernia repair procedures. Study design: The study data set included 2,164 patients at baseline and 1,562 patients at 3-month followup. Only male patients were enrolled in the trial. The psychometric characteristics of the AAS were examined in statistical analyses of cross-sectional and longitudinal data from the trial. Correlational analyses, factor analyses, and t-tests were used to evaluate scale performance. Results: We found that the AAS was a reliable measure (Cronbach’s Coefficient Alpha =0.85) in the patient population studied. Factor analyses identified three subscales (sedentary activities; ambulatory activities; work and exercise activities). Construct validity was demonstrated by a correlation of 0.65 between the AAS and the physical functioning (PF) dimension of the SF-36 (p &#60; 0.001); comparisons between clinical subgroups further confirmed its validity (p &#60; 0.001). Patients reporting improvement on the physical functioning dimension after surgery showed an effect size of 1.20 for preoperative-postoperative change in their AAS scores. Conclusions: The AAS has been demonstrated to be a reliable, valid, and clinically responsive instrument that can be used to evaluate patient functioning after hernia surgery. It is easy to administer and requires less than 5 minutes of patient time to complete. This measurement system may prove useful in assessing surgical outcomes in both research and office practice settings. [Copyright &y& Elsevier]

Introduction: Social distancing requirements during COVID-19 pose a challenge to conducting traditional academic detailing, which typically involves in-person peer education visits to improve patient outcomes. The main alternative is to conduct virtual academic detailing delivered through web-based technology, but this approach is fraught with many challenges. This study aimed to examine the feasibility and acceptability of a virtual academic detailing program implemented among health care providers.Methods: The academic detailing program focused on appropriate opioid prescribing and chronic non-cancer pain management among a sample of providers. An initial in-person visit was followed by a virtual visit up to 8 weeks later. Videoconferencing was used to conduct the virtual visit with telephone as a backup. Feasibility was assessed whether the virtual visits could happen, and acceptability was assessed by provider satisfaction. Validated measures of Provider Satisfaction with Academic Detailing (PSAD) and Detailer Assessment of Visit Effectiveness (DAVE) with a 5-point Likert-type scale were used. Higher scores corresponded to higher satisfaction and greater perceived effectiveness. Non-parametric and parametric statistical tests were used to compare instrument summary scores across visits and between groups. Pairwise analyses across visits only included instrument responses for providers who participated in both visits and completed both surveys in their entirety.Results: There were 127 (90 %) initial in-person visits completed out of 141 visits scheduled, with a survey response rate of 96 %. Out of 120 virtual follow-up visits scheduled, 92 (77 %) were conducted, and 56 surveys (61 %) were collected. There was a high level of satisfaction with the initial and follow up virtual academic detailing visits, though, among providers who participated in both visits and had completed surveys (n = 50), initial visits had slightly higher scores (mean difference = -2.94 [95 % Confidence intervals: -4.38, -1.50], p < 0.001). There was no significant difference in detailer perception across the two visits as seen in the scale summary score (0.05 [-0.56, 0.66], p = 0.86) and two individually reported items related to feasibility (0.07 [-0.29, 0.42], p = 0.72) and conversation (-0.05 [-0.28, 0.17], p = 0.63). Forty-one (44.6 %) virtual visits were conducted using WebEx, where video and screen sharing of visit content was possible, while the remaining 51 (55.4 %) were conducted using a telephone. There was no significant difference in provider satisfaction between WebEx vs. telephone visits (-1.47 [-4.99, 2.05], p = 0.82). Provider satisfaction was also not impacted by any technical difficulties as reported by the detailer (-0.04 [-3.30, 3.38], p = 0.98).Conclusion: The results slightly favor in-person visits and suggest that virtual detailing visits need to incorporate strategies that minimize technical difficulties and prevent participants from defaulting to less favorable technology. Future research opportunities include evaluating the effectiveness of a virtual versus in-person delivery of AD program on outcomes such as providers' opioid prescribing behavior. [ABSTRACT FROM AUTHOR]