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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]

Objective To estimate an interim five-level EuroQol five-dimensional (EQ-5D-5L) value set for Poland on the basis of the crosswalk methodology developed by the EuroQol Group. Methods On the basis of data from 3691 respondents from six European countries, the EuroQol Group has developed a method of obtaining interim value sets for the EQ-5D-5L by means of mapping to the available three-level EuroQol five-dimensional (EQ-5D-3L) value sets (“crosswalk” methodology). A significant part of the data in this study came from Polish respondents (n = 972; 26.3%). Poland is the first Central European country with EQ-5D-3L time trade-off–based social value set published. To obtain an interim EQ-5D-5L value set, we applied the crosswalk methodology to the Polish EQ-5D-3L value set. Results Estimated Polish values for 3125 EQ-5D-5L health states are presented. Both EQ-5D-5L and EQ-5D-3L value sets have the same range (from −0.523 to 1.000), but different means (0.448 vs. 0.380) and medians (0.483 vs. 0.403), respectively. Proportionately fewer states worse than dead were observed in the EQ-5D-5L (5.4%) value set than in the EQ-5D-3L (13.2%) value set. Conclusions The crosswalk-based value set is available for use in EQ-5D-5L studies in Poland to calculate health state utilities. It should be considered an interim value set until values based on preferences elicited directly from a sample representative of the Polish general population become available. This study helps users of the crosswalk algorithm understand the properties of the EQ-5D-5L values generated using this method, in comparison to EQ-5D-3L values obtained with the Polish time trade-off value set. It is likely that similar results would be observed for values sets in other countries because the same crosswalk methodology applies across all countries. [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]

Purpose: Systemic lupus erythematosus (SLE) can significantly affect both health and non-health-related quality of life (HRQOL and non-HRQOL). However, of the existent published patient-reported outcome (PRO) tools, none were developed from US patients, an ethnically diverse population. Furthermore, these tools do not address men with SLE or assess non-HRQOL issues. Herein, we present the development and validation of the Lupus Patient-Reported Outcome tool (LupusPRO) and discuss its clinical utility and research value compared with other PRO tools currently available for SLE. Methods: Beginning with a conceptual framework, items for LupusPRO were generated using feedback from women and men with SLE. The tool underwent iterations based on patient feedback and clinimetric and psychometric analyses. Validity (content, construct, and criterion) and reliability (internal consistency and test-retest) for the 44-item LupusPRO tool are presented. Results: Consistent with the conceptual framework, items were identified that were related to HRQOL and non-HRQOL constructs. HRQOL domains included (1) lupus symptoms; (2) physical health (physical function, role physical); (3) pain–vitality; (4) emotional health (emotional function and role emotional); (5) body image; (6) cognition; (7) procreation; and (8) lupus medications. Non-HRQOL domains were (1) available social support and coping; (2) desires–goals; and (3) satisfaction with medical care. Internal consistency reliability (0.68-0.94), test-retest reliability (0.55-0.92), content, construct (r > 0.50 with SF-36), and criterion (r > −0.35 with disease activity) validity were fair to good. Conclusions: LupusPRO is a valid and reliable disease-targeted patient-reported health outcome tool that is generalizable to SLE patients in the United States of varied ethnic backgrounds and either gender. [Copyright &y& Elsevier]

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]

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]

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]

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]

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]