Health Utility Measures Research Articles

The contribution of pain in determining the health status of cancer patients with bone metastases: A secondary analysis of data from three Phase III registration trials

We aimed to provide a simple, descriptive health-status profile for cancer patients with bone metastases, based on the EuroQol EQ-5D, a tool commonly used to measure health utility scores, and to evaluate its association with the Brief Pain Inventory (BPI), a legacy pain-assessment tool. Although pain is one of five health-status dimensions measured by the EQ-5D, our understanding of how pain relates to the other EQ-5D dimensions is limited. We derived data from 5500 patients with bone metastases who completed the EQ-5D and BPI. Regression analyses examined how BPI severity and interference scores correlated with EQ-5D utility scores and how BPI items associated with EQ-5D items, for the entire sample and by disease-type subgroup. Regardless of cancer site, the percentage of patients reporting moderate/severe problems in each of the five EQ-5D dimensions were pain/discomfort, 78%; usual activities, 58%; mobility, 55%; anxiety/depression, 57%; and self-care, 26%. BPI pain interference explained more of the variability in the EQ-5D utility scores than did pain severity (R2 =41% vs. 34%). BPI worst pain, average pain, pain now, interference with general activity, and interference with work significantly predicted EQ-5D pain/discomfort, with odds ratio estimates <1. Pain/discomfort was the worst-rated dimension of the EQ-5D in this population, but the relationship of this item to BPI pain severity was modest, suggesting that the single pain item of the EQ-5D may be of limited utility in studies for which pain is an endpoint. Health-status dimensions include more than pain. We examine the contribution of pain severity and pain-related functional interference in determining the health status of cancer patients with bone metastases. The pain dimension from a health-status measure may be an inadequate metric in clinical trials/clinical practice when pain is an important outcome.

Health-Related Quality-Of-Life And Utility Measures Impact In A Randomized Controlled Study Of Fine Needle Aspiration Cytology (FNAC) Versus Watchful Observation In Patients With Incidental Symptomatic Thyroid Nodules

To present the impact of treatment on health-related quality-of-life (HRQOL) and health utility measures from the randomized controlled trial (ClinicalTrials.gov Identifier: NCT02398721) that investigated the FNAC versus watchful observation in patients with symptomatic benign thyroid nodules. Health utility and HRQOL were evaluated using the EuroQoL 5-dimension 5-level (EQ-5D-5L), 6-item Short Form Health Survey (SF-6D), cancer-specific Functional Assessment of Cancer Therapy-general (FACT-G) and generic 12-item Short Form Health Survey (SF-12v2) at baseline, 3-month, 6-month and 12-month assessments. A repeated measure analysis of variance (ANOVA) evaluated differences in HRQOL scores between treatment groups over time. Multiple imputation was used to impute missing data at each time point. HRQOL data completion rates were 99.7% at baseline, 92.7% at 3-month, 93.9% at 6-month and 92.7% at 12-month after baseline. There were significant mean differences in SF-6D, EQ-5D-5L, FACT-G and SF-12v2 over time except PWB, EWB of FACT-G and mental health of SF-12v2. Mean change of utility scores from baseline between groups did not exceed minimal important difference. No significant treatment group by time interactions were found in all HRQOL and utility scores except in the vitality domain and PCS of SF-12v2 (p-value = 0.033; 0.024). When compared to watchful observation, FNAC intervention was associated with better vitality and physical-related HRQOL scores but did not provide better preservation of utility score improvement over the 12-month period.

Measuring health utility in varying pregnancy contexts among a diverse cohort of pregnant women

ObjectiveTo contribute to decision analysis by estimating utility, defined as an individual's valuation of specific health states, for different pregnancy contexts. Study designCross-sectional analysis of data from pregnant women recruited at pregnancy testing clinics during June 2014–June 2015. Utility was measured using the visual analog scale (VAS), PROMIS GSF-derived utility, standard gamble (SG), and time-trade-off (TTO) approaches. Six dimensions of pregnancy context were assessed including: intention, desirability, planning, timing, wantedness, and happiness. Multivariable regression modeling was used to examine the associations between pregnancy context and utility while controlling for women's sociodemographic and health characteristics. ResultsAmong 123 participants with diverse characteristics, aged 27±6 years, with mean gestation of 7.5±3 weeks, few reported optimal pregnancy contexts. Mean utility of the pregnancy state varied across contexts, whether measured with VAS (0.28–0.91), PROMIS GSF-derived utility (0.66–0.75), SG (0.985–1.00) or TTO (0.9990–0.99999). The VAS-derived mean utility score for unintended pregnancy was 0.68 (95% CI 0.59, 0.77). Multivariable regression analysis demonstrated significant disutility of unintended pregnancy, as well as all other unfavorable pregnancy contexts, when measured by VAS. In contrast, PROMIS GSF-derived utility only detected a significant reduction in utility among ambivalent compared to wanted pregnancy, while SG and TTO did not show meaningful differences in utility across pregnancy contexts. ConclusionsUnintended pregnancy is associated with significant patient-reported disutility, as is pregnancy occurring in other unfavorable contexts. VAS-based measurements provide the most nuanced measures of the utility for pregnancy in varying contexts. ImplicationsDecision analyses, including assessments of the cost-effectiveness of pregnancy related interventions, should incorporate measures of the utility of pregnancy in various contexts.

Patient-Proxy Agreement on the Health-Related Quality of Life One Year After Traumatic Brain Injury

ObjectiveTo examine the level of agreement between patients and their proxies on a measure of health-related quality of life (HRQL) 1 year after traumatic brain injury (TBI). DesignA cross-sectional study. SettingFour hospitals in Taipei. ParticipantsParticipants (N=176) were patients with TBI (n=88) identified from discharge records of 4 hospitals in Taiwan and family members or friends (n=88) who could answer questions regarding the patient's health and HRQL 1 year after the injury. InterventionsNot applicable. Main Outcome MeasuresA health profile of the brief Taiwanese version of the World Health Organization Quality of Life (WHOQOL-BREF) and 2 health utility measures of the time tradeoff (TTO) and standard gamble (SG). ResultsCompared with proxy responses, patients provided higher ratings on the domains of the WHOQOL-BREF and lower ratings on the TTO and SG. Intraclass correlation coefficients between patient and proxy ratings varied among the 4 domains of the WHOQOL-BREF, in which levels of agreement were moderate or good for physical capacity (.69) and low for psychological well-being (.45), social relationships (.24), and the environment (.32). Levels of agreement were extremely low for the TTO (0.0) and SG (.10). A profile analysis showed that patients' ratings on the WHOQOL-BREF, on average, were significantly greater than those of their proxies (mean difference, 3.07), with a similar distribution of scatter responses (mean difference, .002) and shape agreement (mean difference, .57). Greater variations in the patient-proxy level of agreement appeared in patients who were younger and had more severe injuries, and also with parent and child proxies. ConclusionsTo assess the HRQL of patients with a TBI, the level of patient-proxy agreement was adequate for the physical domain but was lower for the social and environmental domains, and the agreement was very low for the TTO and SG. Furthermore, a patient's age and injury severity, and the patient-proxy' relationship may affect the agreement.

Influences of Socio-Demographic Factors and Health Utilization Factors on Patient-Centered Provider Communication

ABSTRACTPatient-centered care has been documented as a measure of quality of health care and has been associated with positive health outcomes. However, the effect of health utilization on improving patient-centered communication has not been investigated. This study examined the effect of three important kinds of health utilization: routine check-up, frequency of provider visits in the last year, and quality of health care to patient-centered provider communication. Cross-sectional data from 3,608 respondents to Health Information National Trends Survey-Cycle 4 2014 were analyzed. Multiple regressions were used to examine the association of sociodemographic factors and health utilization to patient-centered provider communication. Results showed that adults above 50 years and women reported higher patient-centered provider communication. Hispanic and Asian versus White respondents reported poorer patient-centered provider communication. Respondents with routine checkups between 1 and 2 years, 2 and 5 years, 5 or more years and none were all negatively associated with patient-centered provider communication in comparison with routine checkup within 1 year. Respondents who didn’t visit health provider within past year had poorer patient-centered provider communication when compared to those who visited once. Finally, higher quality of healthcare experience was associated with higher patient-centered provider communication. Thus, this study highlights that race and ethnicity, age, and gender are significant factors that influence patient-centered provider communication; and specifically higher quality of healthcare experience, one provider visit within past year, and annual routine checkup as measures of health utilization predicts improved patient-centered provider communication.

Valuing Non-Fatal Health Risks: Monetary and Health-Utility Measures

Les indicateurs évaluant les politiques environnementales, sanitaires et de prévention doivent être cohérents avec les préférences des individus affectés, mais également avec les préférences sociales quant à la distribution des risques sanitaires dans la population. Deux types de mesures sont largement employées : monétaires (comme le consentement à payer) ou fondées sur des états de santé pondérés par l’utilité (comme les années de vie ajustées de la qualité ou de l’incapacité), les deux étant sommées pour toute la population. Les secondes, en imposant plus de structure que les premières, font souvent apparaître les préférences individuelles comme incohérentes. Pour la même raison, elles permettent toutefois de limiter les erreurs cognitives et les autres sources d’incohérence propres à l’évaluation monétaire. Cet article présente des preuves empiriques et théoriques permettant de comparer ces deux types d’indicateurs et d’étudier leurs covariations.

Cost-Effectiveness Analysis of Percutaneous Vertebroplasty for Osteoporotic Compression Fractures.

Single-center, single-arm, prospective time-series study. To assess the cost-effectiveness and improvement in quality of life (QOL) of percutaneous vertebroplasty (PVP). PVP is known to relieve back pain and increase QOL for osteoporotic compression fractures. However, the economic value of PVP has never been evaluated in Japan where universal health care system is adopted. We prospectively followed up 163 patients with acute vertebral osteoporotic compression fractures, 44 males aged 76.4±6.0 years and 119 females aged 76.8±7.1 years, who underwent PVP. To measure health-related QOL and pain during 52 weeks observation, we used the European Quality of Life-5 Dimensions (EQ-5D), the Rolland-Morris Disability Questionnaire (RMD), the 8-item Short-Form health survey (SF-8), and visual analogue scale (VAS). Quality-adjusted life years (QALY) were calculated using the change of health utility of EQ-5D. The direct medical cost was calculated by accounting system of the hospital and Japanese health insurance system. Cost-effectiveness was analyzed using incremental cost-effectiveness ratio (ICER): Δ medical cost/Δ QALY. After PVP, improvement in EQ-5D, RMD, SF-8, and VAS scores were observed. The gain of QALY until 52 weeks was 0.162. The estimated lifetime gain of QALY reached 1.421. The direct medical cost for PVP was ¥286,740 (about 3061 US dollars). Cost-effectiveness analysis using ICER showed that lifetime medical cost for a gain of 1 QALY was ¥201,748 (about 2154 US dollars). Correlations between changes in EQ-5D scores and other parameters such as RMD, SF-8, and VAS were observed during most of the study period, which might support the reliability and applicability to measure health utilities by EQ-5D for osteoporotic compression fractures in Japan as well. PVP may improve QOL and ameliorate pain for acute osteoporotic compression fractures and be cost-effective in Japan.

EQ-5D-5L and SF-6D Utility Measures in Symptomatic benign Thyroid Nodules: Acceptability and Psychometric Evaluation.

The aim of this study was to examine the acceptability, validity, and reliability of the EuroQoL Five-Dimension Five-Level (EQ-5D-5L) and Short-Form Six-Dimension (SF-6D) health utility measures in patients with symptomatic benign thyroid nodules. Data from a randomized controlled trial (ClinicalTrials.gov identifier: NCT02398721) of 294 patients with symptomatic benign thyroid nodules were utilized for this psychometric evaluation of health-related quality of life (HR-QOL) measurement. Three HR-QOL questionnaires-the generic 12-item Short Form Health Survey (SF-12v2), EQ-5D-5L, and SF-6D-were interviewer-administered at baseline and 2weeks afterwards. Responses to SF-6D were transformed to SF-6D utility scores using a Hong Kong population scoring algorithm derived by standard gamble, whereas responses to EQ-5D-5L were mapped onto EQ-5D-3L response via interim mapping algorithms and then converted to EQ-5D-5L utility scores using a Chinese-specific value set. Construct validity was determined by evaluating Spearman correlation between SF-12v2 scores and utility scores. Two-week test-retest reliability was assessed using intra-class correlation coefficient. No significant (>15%) floor and ceiling effects were observed for SF-6D utility scores. The SF-6D utility scores had a moderate Spearman rank correlation with the SF-12v2 domain score providing evidence for adequate construct validity. The SF-6D utility scores showed good test-retest reliability (0.794; range 0.696-0.860). Better reliability was observed in SF-6D utility scores than in EQ-5D-5L utility scores. While the EQ-5D-5L instrument was less reproducible, the SF-6D instrument appeared to be an applicable, valid, and reliable measure in assessing the HR-QOL of Chinese patients with symptomatic benign thyroid nodules. The impact of utility score selection on the effectiveness and cost effectiveness of clinical interventions targeted to these patients needs further exploration. NCT02398721, ClinicalTrials.gov.

Multiple sclerosis in Canada 2011 to 2031: results of a microsimulation modelling study of epidemiological and economic impacts.

The objective of our study was to present model-based estimates and projections on current and future health and economic impacts of multiple sclerosis (MS) in Canada over a 20-year time horizon (2011-2031). Using Statistics Canada's Population Health Microsimulation Model (POHEM) framework, specifically the population-based longitudinal, microsimulation model named POHEM-Neurological, we identified people with MS from health administrative data sources and derived incidence and mortality rate parameters from a British Columbia population-based cohort for future MS incidence and mortality projections. We also included a utility-based measure (Health Utilities Index Mark 3) reflecting states of functional health to allow projections of health-related quality of life. Finally, we estimated caregiving parameters and health care costs from Canadian national surveys and health administrative data and included them as model parameters to assess the health and economic impact of the neurological conditions. The number of incident MS cases is expected to rise slightly from 4051 cases in 2011 to 4794 cases per 100 000 population in 2031, and the number of Canadians affected by MS will increase from 98 385 in 2011 to 133 635 in 2031. The total per capita health care cost (excluding out-of-pocket expenses) for adults aged 20 and older in 2011 was about $16 800 for individuals with MS, and approximately $2500 for individuals without a neurological condition. Thus, after accounting for additional expenditures due to MS (excluding out-of-pocket expenses), total annual health sector costs for MS are expected to reach $2.0 billion by 2031. As well, the average out-of-pocket expenditure for people with MS was around $1300 annually throughout the projection period. MS is associated with a significant economic burden on society, since it usually affects young adults during prime career- and family-building years. Canada has a particularly high prevalence of MS, so research such as the present study is essential to provide a better understanding of the current and future negative impacts of MS on the Canadian population, so that health care system policymakers can best plan how to meet the needs of patients who are affected by MS. These findings also suggest that identifying strategies to prevent MS and more effectively treat the disease are needed to mitigate these future impacts.

Evaluating the Effects of a Targeted Home Visiting Program on Maternal and Child Health Outcomes

We evaluate the effects of home visiting targeted towards disadvantaged first-time mothers on maternal and child health outcomes. Our analysis exploits a randomized controlled trial and combines rich longitudinal survey data with unique administrative health data. In a context in which the target group has comprehensive health care access, we find that home visiting has no effects on most types of health utilization, health behaviors, and physical health measures. However, the intervention has a remarkably robust and sizable positive effect on maternal mental health, reducing depressions reported in the survey data and prescriptions of psycholeptics recorded in the administrative data.

Does cognitive decline decrease health utility value in older adult patients with cancer?

Cognitive decline is common among older adults with cancer. The present study aimed to investigate the impact of cognitive decline on health utility value in older adults suffering from cancer. Consecutive patients aged 65 years or older with a primary diagnosis of malignant lymphoma or multiple myeloma were recruited. Patients were asked to complete the EuroQoL-5 (EQ-5D) scale to measure health utility and the Mini-Mental State Examination to assess cognitive decline. The potential impact of cognitive decline was investigated with univariate analysis. A multivariate regression analysis was conducted to control for potential confounding factors. Complete data were obtained from 87 patients, 29% of whom had cognitive decline. The mean ± SE EQ-5D score for patients with cognitive decline was significantly lower than that for those without cognitive decline (0.67 ± 0.04 vs 0.79 ± 0.03, t = 2.38, P = 0.02). However, multiple regression analysis showed that cognitive decline was not significantly associated with EQ-5D scores. Female sex and lower performance scores (worse physical condition) were significantly associated with EQ-5D scores. Cognitive decline may be involved in decreased health utility value in older adult patients with cancer. However, this effect does not seem to be independent, and the patient's physical condition may be a relevant confounding factor.

Early Efforts By Medicare Accountable Care Organizations Have Limited Effect On Mental Illness Care And Management

People with mental illness use more health care and have worse outcomes than those without such illnesses. In response to incentives to reduce spending, accountable care organizations (ACOs) may therefore attempt to improve their management of mental illness. We examined changes in mental health spending, utilization, and quality measures associated with ACO contracts in the Medicare Shared Savings Program and Pioneer model for beneficiaries with mental illness, using Medicare claims for the period 2008-13 and difference-in-differences comparisons with local non-ACO providers. Pioneer contracts were associated with lower spending on mental health admissions in the first year of the contract, an effect that was attenuated in the second year. Otherwise, ACO contracts were associated with no changes in mental health spending or readmissions, outpatient follow-up after mental health admissions, rates of depression diagnosis, or mental health status. These results suggest that ACOs have not yet focused on mental illness or have been largely unsuccessful in early efforts to improve their management of it.

A structured review of health utility measures and elicitation in advanced/metastatic breast cancer.

BackgroundHealth utilities are increasingly incorporated in health economic evaluations. Different elicitation methods, direct and indirect, have been established in the past. This study examined the evidence on health utility elicitation previously reported in advanced/metastatic breast cancer and aimed to link these results to requirements of reimbursement bodies.MethodsSearches were conducted using a detailed search strategy across several electronic databases (MEDLINE, EMBASE, Cochrane Library, and EconLit databases), online sources (Cost-effectiveness Analysis Registry and the Health Economics Research Center), and web sites of health technology assessment (HTA) bodies. Publications were selected based on the search strategy and the overall study objectives.ResultsA total of 768 publications were identified in the searches, and 26 publications, comprising 18 journal articles and eight submissions to HTA bodies, were included in the evidence review. Most journal articles derived utilities from the European Quality of Life Five-Dimensions questionnaire (EQ-5D). Other utility measures, such as the direct methods standard gamble (SG), time trade-off (TTO), and visual analog scale (VAS), were less frequently used. Several studies described mapping algorithms to generate utilities from disease-specific health-related quality of life (HRQOL) instruments such as European Organization for Research and Treatment of Cancer Quality of Life Questionnaire – Core 30 (EORTC QLQ-C30), European Organization for Research and Treatment of Cancer Quality of Life Questionnaire – Breast Cancer 23 (EORTC QLQ-BR23), Functional Assessment of Cancer Therapy – General questionnaire (FACT-G), and Utility-Based Questionnaire-Cancer (UBQ-C); most used EQ-5D as the reference. Sociodemographic factors that affect health utilities, such as age, sex, income, and education, as well as disease progression, choice of utility elicitation method, and country settings, were identified within the journal articles. Most submissions to HTA bodies obtained utility values from the literature rather than exploring the HRQOL data obtained during clinical development. This was critiqued by the National Institute for Health and Clinical Excellence (NICE). Furthermore, the impact of age on utilities was highlighted by NICE and it was suggested that an age match of the study population should be attempted.ConclusionHealth utilities are recorded across the globe to varying extents and using differing elicitation methods. Manufacturers seeking reimbursement need to be aware of the country-specific requirements for elicitation of health utilities.

Asthma and asthma-related health care utilization among people without disabilities and people with physical disabilities

BackgroundPrevious research has shown that people with disabilities have higher rates of some chronic diseases and receive poorer disease-specific care than their counterparts without disabilities. Yet, little is known about the relationship between asthma and disability. ObjectiveThis study examines whether differences in the prevalence of asthma, asthma flare, and asthma-related measures of health care quality, utilization and cost exist among people with physical limitations (PL) and without any limitations. MethodsData from the 2004–2010 Medical Expenditure Panel Survey were pooled to compare outcomes for working-age adults (18–64) with PL to those with no limitations. ResultsPeople with PL had higher rates of asthma (13.8% vs. 5.9%, p < 0.001) and recent asthma flare (52.6% vs. 39.6%, p < 0.001) than people without limitations. There were no differences in health care quality, utilization or cost between people with PL and people without limitations in multivariate analyses. ConclusionsAlthough there are no differences in asthma-related quality or utilization of health care, people with PL have poorer asthma control than people without limitations. Research is needed to determine what factors (e.g., focus on other acute ailments, perceptions that asthma control cannot improve) are related to this outcome. Future research must also examine differences in asthma severity, and its impact on asthma control and health care-related outcomes, among people with and without disabilities.

A Medicare Current Beneficiary Survey-Based Investigation of Alternative Primary Care Models in Nursing Homes: Cost and Utilization Differences.

The current study used the Medicare Current Beneficiary Survey-Based (MCBS) Cost and Use files for 2006-2008 to investigate whether health care costs and service utilization of nursing home residents varied with nurse practitioner (NP) and physician assistant (PA) involvement, compared to the use of medical doctors (MDs) only. The sample included Medicare beneficiaries 65 and older residing in a nursing home for the entire study year (433 annual observations). A generalized estimating equations procedure was used to assess whether health care cost and utilization measures varied by cohort. Point estimates indicated that the annual per-person cost of non-institutional services (total medical cost less the cost of the nursing home itself) was $3,847 and $3,170 more for individuals in the MD-only and MD-dominant cohorts, respectively, compared to those in the NP/PA-dominant cohort. [Res Gerontol Nurs. 2016; 9(3):115-122.].

A single question about a respondent's perceived financial ability to pay monthly bills explains more variance in health utility scores than absolute income and assets questions.

To evaluate a general question about ability to meet monthly bills as an alternative to direct questions about income and assets in health utility studies. We used data from the National Health Measurement Study-a US nationally representative telephone survey collected in 2005-2006. It included health utility measures (EuroQol-5D-3L, Health Utilities Index Mark 3, Short Form-6D, and Quality of Well-being Index) and household income, assets, and financial ability to meet monthly bills questions. Each utility score was regressed on: income and assets (Model 1); difficulty paying bills (DPB) (Model 2); income, assets, and DPB (Model 3). All models used survey weights and adjusted for demographics and education. Among 3666 respondents, as income and assets increased, DPB decreased. The DPB question had fewer missing values (n=30) than income (n=311) or assets (n=373). Model 2 (DPB only) explained more variance in health utility than Model 1 (income and assets only). Including all measures (Model 3) had very modest improvement in R (2), e.g., values were 0.112 (Model 1), 0.166 (Model 2), and 0.175 (Model 3) for EuroQol-5D-3L. The single question on DPB yields more information and has less missing values than the traditionally used income and assets questions.

Suicidal Attempt With Intentional Poisoning Seems a Comorbid Illness With an Increased Burden.

Background:In measuring health utilities, the primary reason for selecting patients as a source for valuations is that they directly experience the impact of the disease.Objectives:Accordingly, the aim of this study was to examine the variation in generic utility measures with respect to acute poisonings by including a comparison between those subjects who had high intention and low intention to commit suicide.Patients and Methods:We evaluated the responses of patients who had attempted suicide and were admitted to a toxicology ward. We used multiple methods, including TTO, VAS, and EQ-5D.Results:We reviewed the collected questionnaires of one hundred patients admitted to the medical toxicology ward of Emam Reza teaching hospital in Mashhad, Iran. Our results show that the mental state after an incomplete suicide attempt can present either a real desire for suicide or a desire for attention from relatives and rejection of life problems.Conclusions:This study demonstrates that the mental states associated with specific diseases should not be ignored in evaluating health states. Although there are benefits to relying on expert panels and the general population in evaluating various health states, attention to the particular health states of the patients (taking into account their associated mental well-being) should also be utilized.

The Significant Contribution of Pain in Determining the Health Status of Patients with Multiple Myeloma (MM)

BACKGROUND: Most cancer patients with bone metastases report significant pain and substantial impairment in their ability to function. In particular, patients with MM are susceptible to bone pain because of bone damage. Patient-reported outcomes such as a general health outcome measure and a legacy pain instrument may provide valuable information in determining the impact of this condition. The goal of this study was to provide a descriptive profile of health status for cancer patients using the EQ-5D-3L (EQ-5D), a commonly used tool to measure health utility scores. Another goal is to evaluate the predictive information provided by the Brief Pain Inventory (BPI), a self-report pain assessment tool designed to capture pain severity and interference in patients. These information may be useful in estimating the health status of patients where the EQ-5D was not administered.METHODS: Data was pooled across three denosumab registrational trials '20050136', '20050103', and '20050244'. Patients completed the EQ-5D and the BPI at baseline. Eligible patients had histologically confirmed advanced cancers, radiographic evidence of at least one bone metastasis (or lytic bone lesion from multiple myeloma), ECOG of 2 or better and adequate organ function. Using data from patients with MM (N=168), descriptive statistics were used to present summary information on health-related quality of life states and utility scores. Linear regressions were used to examine how BPI severity and interference subscale scores relate with EQ-5D utility scores. Ordinal regressions were used to evaluate how well BPI items predict EQ-5D individual items. Bonferroni adjustments were made when evaluating the relationships of BPI items to individual EQ-5D items. These analyses were repeated using data across several groups of cancer patients to determine if similar results can be obtained (breast cancer, N=2,044, prostate cancer, N=1,819 and others, N=1,509).RESULTS: About 9% of MM patients reported perfect health compared with 11% of all cancer patients. Of the 5 dimensions measured by the EQ-5D, the MM cohort rated pain (80%) the highest with moderate to extreme problems. This was followed by usual activities (68%), mobility (63%), anxiety/depression (49%), and self-care (36%). Similar order was observed across all cancer groups, but with slightly lower percentages for most items: pain (78%), usual activities (58%), mobility (55%), anxiety/depression (57%), and self-care (26%). Although pain interference subscale score explained more of the variability in the EQ-5D utility scores compared with pain severity items in the MM cohort (39% vs 31%) and for all patients (41% vs 34%), model fit was less than ideal (mean squared error=0.23 - 0.25). Hence, BPI items were fitted into individual EQ-5D items. Ordinal regression models fitting BPI items into individual EQ-5D items for the MM cohort showed good predictive power as measured by the concordance index c for mobility (86%), self-care (80%), usual activities (83%), pain (90%) and anxiety/depression (78%). For all patients, similar model fit were obtained for mobility (83%), self-care (78%), usual activities (81%), pain (90%) and anxiety/depression (72%). Further examination of the ordinal regression models across all groups showed that three BPI pain severity items, 'pain at its worst', 'average pain' and 'pain now' in addition to interference with activity and interference with work significantly predicted the EQ-5D pain item. Five interference items, activity, walking ability, work, sleep and enjoyment of life significantly predicted the EQ-5D usual activities item. Finally, 'pain now' and four interference items, relationship with others, enjoyment of life, mood, and activity significantly predicted the EQ-5D anxiety/depression item.CONCLUSIONS: Patients with MM reported several functional limitations on the EQ-5D. Majority of the patients reported moderate to extreme problems in all EQ-5D dimensions except self-care. Model fit of ordinal regression models relating BPI items with EQ-5D items are reasonable underscoring the benefit of using the BPI for planning patient care and health status evaluation. This consistency was also found across cancer groups that primarily include breast and prostate. The resultant model demonstrates the significant contribution of pain severity and interference in determining health status in this patient population. DisclosuresMendoza:Amgen Inc.: Consultancy. Shi:Amgen Inc.: Consultancy. Ma:Amgen Inc.: Employment. Zhang:Amgen Inc.: Employment. Qian:Amgen Inc.: Employment. Cleeland:Amgen Inc.: Consultancy.

Pressure UlceR Programme Of reSEarch (PURPOSE): using mixed methods (systematic reviews, prospective cohort, case study, consensus and psychometrics) to identify patient and organisational risk, develop a risk assessment tool and patient-reported outcome Quality of Life and Health Utility measures

BackgroundThe Pressure UlceR Programme Of reSEarch (PURPOSE) consisted of two themes. Theme 1 focused on improving our understanding of individuals’ and organisational risk factors and on improving the quality of risk assessments (work packages 1–3) and theme 2 focused on developing patient-reported outcome measures (work packages 4 and 5).MethodsThe programme comprised 21 individual pieces of work. Pain: (1) multicentre pain prevalence study in acute hospitals, (2) multicentre pain prevalence study in community localities incorporating (3) a comparison of case-finding methods, and (4) multicentre, prospective cohort study. Severe pressure ulcers: (5) retrospective case study, (6) patient involvement workshop with the Pressure Ulcer Research Service User Network for the UK (PURSUN UK) and (7) development of root cause analysis methodology. Risk assessment: (8) systematic review, (9) consensus study, (10) conceptual framework development and theoretical causal pathway, (11) design and pretesting of draft Risk Assessment Framework and (12) field test to assess reliability, validity, data completeness and clinical usability. Quality of life: (13) conceptual framework development (systematic review, patient interviews), (14 and 15) provisional instrument development, with items generated from patient interviews [from (1) above] two systematic reviews and experts, (16) pretesting of the provisional Pressure Ulcer Quality of Life (PU-QOL) instrument using mixed methods, (17) field test 1 including (18) optimal mode of administration substudy and item reduction with testing of scale formation, acceptability, scaling assumptions, reliability and validity, and (19) field test 2 – final psychometric evaluation to test scale targeting, item response categories, item fit, response bias, acceptability, scaling assumptions, reliability and validity. Cost–utility: (20) time trade-off task valuations of health states derived from selected PU-QOL items, and (21) validation of the items selected and psychometric properties of the new Pressure Ulcer Quality of Life Utility Index (PUQOL-UI).Key findingsPain: prevalence studies – hospital and community patients experience both pressure area-related and pressure ulcer pain; pain cohort study – indicates that pain is independently predictive of category 2 (and above) pressure ulcer development. Severe pressure ulcers: these were more likely to develop in contexts in which clinicians failed to listen to patients/carers or recognise/respond to high risk or the presence of an existing pressure ulcer and services were not effectively co-ordinated; service users found the interactive workshop format valuable; including novel components (interviews with patients and carers) in root cause analysis improves the quality of the insights captured. Risk assessment: we developed a Pressure Ulcer Risk Assessment Framework, the PURPOSE-T, incorporating the Minimum Data Set, a screening stage, a full assessment stage, use of colour to support decision-making, and decision pathways that make a clear distinction between patients with an existing pressure ulcer(s) (or scarring from previous ulcers) who require secondary prevention and treatment and those at risk who require primary prevention (http://medhealth.leeds.ac.uk/accesspurposet). Quality of life: the final PU-QOL instrument consists of 10 scales to measure pain, exudate, odour, sleep, vitality, mobility/movement, daily activities, emotional well-being, self-consciousness and appearance, and participation (http://medhealth.leeds.ac.uk/puqol-ques). Cost–utility: seven items were selected from the PU-QOL instrument for inclusion in the PUQOL-UI (http://medhealth.leeds.ac.uk/puqol-ui); secondary study analysis indicated that item selection for the PUQOL-UI was appropriate and that the index was acceptable to patients and had adequate levels of validity.ConclusionsThe PURPOSE programme has provided important insights for pressure ulcer prevention and treatment and involvement of service users in research and development, with implications for patient and public involvement, clinical practice, quality/safety/health service management and research including replication of the pain risk factor study, work exploring ‘best practice’ settings, the impact of including skin status as an indicator for escalation of preventative interventions, further psychometric evaluation of PU-QOL and PUQOL-UI the measurement of ‘disease attribution.’FundingThe National Institute for Health Research Programme Grants for Applied Research programme.

Quality of Life and Risks Associated with Systemic Anti-inflammatory Therapy versus Fluocinolone Acetonide Intraocular Implant for Intermediate Uveitis, Posterior Uveitis, or Panuveitis: Fifty-four–Month Results of the Multicenter Uveitis Steroid Treatment Trial and Follow-up Study

To evaluate the risks and quality-of-life (QoL) outcomes of fluocinolone acetonide implant versus systemic therapy with corticosteroid and immunosuppression when indicated for intermediate uveitis, posterior uveitis, and panuveitis. Additional follow-up of a randomized trial cohort. Two hundred fifty-five patients with intermediate uveitis, posterior uveitis, or panuveitis, randomized to implant or systemic therapy. Randomized subjects with intermediate uveitis, posterior uveitis, or panuveitis (479 eyes) were followed up over 54 months, with 79.2% completing the 54-month visit. Local and systemic potential complications of the therapies and self-reported health utility and vision-related and generic health-related QoL were studied prospectively. Among initially phakic eyes, cataract and cataract surgery occurred significantly more often in the implant group (hazard ratio [HR], 3.0; P = 0.0001; and HR, 3.8; P < 0.0001, respectively). In the implant group, most cataract surgery occurred within the first 2 years. Intraocular pressure elevation measures occurred more frequently in the implant group (HR range, 3.7-5.6; all P < 0.0001), and glaucoma (assessed annually) also occurred more frequently (26.3% vs. 10.2% by 48 months; HR, 3.0; P = 0.0002). In contrast, potential complications of systemic therapy, including measures of hypertension, hyperlipidemia, diabetes, bone disease, and hematologic and serum chemistry indicators of immunosuppression toxicity, did not differ between groups through 54 months. Indices of QoL initially favored implant therapy by a modest margin. However, all summary measures of health utility and vision-related or generic health-related QoL were minimally and nonsignificantly different by 54 months, with the exception of the 36-item Short-Form Health Survey physical component summary score, which favored implant by a small margin at 54 months (3.17 on a scale of 100; P = 0.01, not adjusted for multiple comparisons). Mean QoL results were favorable in both groups. These results suggest that fluocinolone acetonide implant therapy is associated with a clinically important increased risk of glaucoma and cataract with respect to systemic therapy, suggesting that careful monitoring and early intervention to prevent glaucoma is warranted with implant therapy. Systemic therapy subjects avoided a significant excess of toxicities of systemic corticosteroid and immunosuppressive therapies in the trial. Self-reported QoL measures initially favored implant therapy, but over time the measures converged, with generally favorable QoL in both groups.