Preference-based Health-related Quality Of Life Research Articles

Burden and preference-based quality of life associated with bullying in children.

The objectives of this study are to assess the association between childhood bullying and preference-based health-related quality of life (QoL) in Australian school children and their parents and estimate quality-adjusted life years (QALYs) associated with bullying chronicity. Children aged 8-10years completed the child health utilities (CHU-9D), while parents completed the Australian quality of life (AQoL-8D). Children were grouped into four categories of bullying involvement (no bullying, victim, perpetrator, or both perpetrator and victim) based on the Revised Olweus Bully/Victim Questionnaire. Parental data were compared across two bullying involvement groups (bullying vs. no bullying). QALYs were calculated for children over two years and comparisons made based on the number of assessments where bullying was reported(baseline, 1- and 2-year follow up). Children who were involved in bullying (victims and/or perpetrators) reported statistically significantly lower mean utility scores compared to children who were not involved in bullying. Parents whose child was involved in bullying had significantly lower mean utility scores compared to parents of children not involved with bullying. There appeared to be a dose-response relationship, with higher QALY losses associated with increasing frequency of reported bullying. Bullying among Australian school children was associated with significantly lower preference-based QoL for themselves and their parents. This study also confirmed the significant burden of disease for bullying among children measured by an incremental decrease in QALY with an increasing chronicity of bullying over time.

A Systematic Literature Review of Preference-Based Health-Related Quality-of-Life Measures Applied and Validated for Use in Childhood and Adolescent Populations in Sub-Saharan Africa

ObjectivesConsideration of health status in children and adolescents now includes broader concepts such as health-related quality-of-life (HRQoL). Globally, there is a need for relevant preference-based HRQoL measures (PBMs) for use in children and adolescents, yet measurement of HRQoL in these groups presents particular challenges. This article systematically reviews the available generic childhood PBMs and their application and cross-cultural validation in sub-Saharan African (sSA). MethodsA systematic review of published literature from January 1, 1990, to February 8, 2017, was conducted using MEDLINE (through OvidSP), EMBASE (OvidSP), EconLit (EBSCOhost), PsycINFO, Web of Science, and PubMed. ResultsA total of 220 full-text articles were included in a qualitative synthesis. Ten generic childhood PBMs were identified, of which 9 were adapted from adult versions and only 1 was developed specifically for children. None of the measures were originally developed in sSA or other resource-constrained settings. The Health Utilities Index Mark 3 (HUI3) and the EQ-5D-Y were the only measures that had been applied in sSA settings. Further, the HUI3 and the EQ-5D-Y were the only generic childhood PBM that attempted to establish cross-cultural validation in sSA. Five of the 6 of these validation studies were conducted using the EQ-5D-Y in a single country, South Africa. ConclusionsThe findings show that application of generic childhood PBMs in sSA settings has hitherto been limited to the HUI3 and EQ-5D-Y. Most adaptations of existing measures take an absolutist approach, which assumes that measures can be used across cultures. Nevertheless, there is also need to ensure linguistic and conceptual equivalence and undertake validation across a range of sSA cultural contexts.

Empirical Validity of a Generic, Preference-Based Capability Wellbeing Instrument (ICECAP-A) in the Context of Spinal Cord Injury.

Assessing the validity of generic instruments across different clinical contexts is an important area of methodological research in economic evaluation and outcomes measurement. Our objective was to examine the empirical validity of a generic, preference-based capability wellbeing instrument (ICECAP-A) in the context of spinal cord injury. This study consisted of a secondary analysis of data collected using an online cross-sectional survey. The survey included questions regarding demographics, injury classifications and characteristics, secondary health conditions, quality of life and wellbeing, and functioning in activities of daily living. Analysis comprised thedescriptive assessment of Spearman's rank correlations between item-/dimension-level data for the ICECAP-A and four preference-based health-related quality of life (HRQoL) instruments, and discriminant and convergent validity approaches to examine 21 evidence-informed or theoretically derived constructs. Constructs were defined using participant and injury characteristics and responses to a range of health, wellbeing and functioning outcomes. Three hundred sixty-fourindividuals completed the survey. Mean index score for the ICECAP-A was 0.761; 12 (3%) individuals reported full capability (upper anchor; score = 1), and there were no reports of zero capabilities (lower anchor; score = 0). The strongest correlations were dominated by items and dimensions on the comparator (HRQoL) instruments that are non-health aspects of quality of life, such as happiness and control over one's life (including self-care). Of 21 hypothesised constructs, 19 were confirmed in statistical tests, the exceptions being the exploratory hypotheses regarding education and age at injury. The ICECAP-A is an empirically valid outcome measure for assessing capability wellbeing in people with spinal cord injury living in a community setting. The extent to which the ICECAP-A provides complementary information to preference-based HRQoL instruments is dependent on the comparator.

The relationship between preference-based health-related quality of life and lifestyle behavior: a cross-sectional study on a community sample of adults who had undergone a health check-up

BackgroundPreference-based Health-Related Quality of Life (HRQL) is one of the most important indicators for calculating QALY (Quality-Adjusted Life Years) in a cost-effectiveness analysis. This study aimed to collect data on healthy individuals’ HRQL based on the preferences of Japanese people who had undergone a comprehensive health check-up, and to examine the influence of relevant factors, such as blood biochemical data and lifestyle behavior.MethodsWe conducted a cross-sectional study targeting people who had undergone a comprehensive health check-up in 2015. Participants were asked to respond to a medical interview sheet. We then examined the utility value, as well as lifestyle habits such as alcohol intake, smoking, and exercise. HRQL was examined using EQ-5D-5L. Using a multiple regression analysis, we examined the influence of related factors, such as lifestyle and biochemical test data.ResultsWe collected 2037 responses (mean age = 54.98 years; 55.0% female). The average preference-based health-related HRQL was 0.936 ± 0.087. A total of 1167 people (57.2%) responded that they were completely healthy. The biochemical test data that were recognized to correlate with HRQL were hemoglobin, total cholesterol, creatinine, all of which were weak (r = − 0.045–0.113). The results of multiple regression analysis showed that significant facts were: being female, age (≧70 year-old), drinking alcohol (sometimes), activity (very often), and lack of sleep.ConclusionsThe HRQL of participants who had undergone a comprehensive health check-up was generally high, and only declined for those over 70 years of age. It is suggested that preference-based HRQL is related to physical activity, and that decrease of activity and lack of sleep leads to a decrease in HRQL.

Effects of Primary Biliary Cholangitis on Quality of Life and Health Care Costs in the United Kingdom

There have been few high-quality studies of the costs, preference-based health-related quality of life (HRQoL) and cost effectiveness of treatments for primary biliary cholangitis (PBC). We aimed to estimate the marginal effects of PBC complications and symptoms, accounting for treatment, on HRQoL and the annual cost of health care in the United Kingdom (UK). These are essential components for evaluation of cost effectiveness and this information will aid in evaluation of new treatments. Questionnaires were mailed to 4583 participants in the UK-PBC research cohort and data were collected on HRQoL and use of the National Health Service (NHS) in the UK from 2015 through 2016. HRQoL was measured using the EQ-5D-5L instrument. The annual cost of resource use was calculated using unit costs obtained from NHS sources. We performed econometric analyses to determine the effects of treatment, symptoms, complications, liver transplantation status, and patient characteristics on HRQoL and annual costs. In an analysis of data from 2240 participants (over 10% of all UK PBC patients), we found that PBC symptoms have a considerable effect on HRQoL. Ursodeoxycholic acid therapy was associated with significantly higher HRQoL regardless of response status. Having had a liver transplant and ascites were also independently associated with reduced HRQoL. Having had a liver transplant (US$4294) and esophageal varices (US$3401) were the factors with the two greatest mean annual costs to the NHS. Symptoms were not independently associated with cost but were associated with reduction in HRQoL for patients, indicating the lack of effective treatments for PBC symptoms. In an analysis of data from 2240 participants in the UK PBC, we found that HRQoL and cost estimates provide greater insight into the relative importance of PBC-related symptoms and complications. These findings provide estimates for health technology assessments of new treatments for PBC.

Health-related quality of life measured using the EQ-5D\u20135\u2009L: population norms for the capital of Iran

ObjectivesEQ-5D is the most commonly used generic preference-based health-related quality of life (HRQoL) measure. The current study aimed at estimating the HRQoL index scores using EQ-5D-5 L measure in the capital of Iran; moreover, identifying some determinants of the HRQoL.MethodsA sample of 3060 subjects was selected by a stratified random sampling method from the general adult population of Tehran. Face-to-face interview was conducted to fill out the questionnaire, in this cross-sectional survey. EQ-5D-5 L utility score were estimated using an interim value set, based on a crosswalk methodology. Additionally, the relationships between HRQoL and sociodemographic characteristics were tested by generalized linear model, using STATA version 13.ResultsThe mean ± standard deviation utility and EQ-VAS scores were 0.79 ± 0.17 and 71.72 ± 19.37. The utility scores ranged 0.61 ± 0.19 in > 69 year-old females to 0.88 ± 0.12 in < 30 year-old males. In mobility, self-care, and usual activity dimensions, most of the respondents reported “no problems” (70.47, 90.62, and 76.34%, respectively). However, in anxiety/depression and pain/discomfort dimensions, most of the respondents had problems (53.23 and 54.03%, respectively). Females had lower utility score than males; the utility score reduced with age increase; the educational level lead to higher utility scores; and the utility scores of individuals without spouse (divorced or widowed) were lower than those of the married individuals and never married ones.ConclusionsThe current study reported HRQoL norm data for the general adult population in the capital of Iran; these data could be very useful for policy making and economic evaluations. A significant percentage of people in Tehran reported anxiety/ depression, which highlights the risk of psychological problems. Effective interventions are needed to increase their HRQoL, especially for the vulnerable groups of the community.

Relationship Between the Wisconsin Stone Quality of Life (WISQOL) and Preference-Based/Health Utility Measures of Health-Related Quality of Life (HRQoL) in Kidney Stone Patients

ObjectiveTo determine preference-based (utility) assessments of health-related quality of life (HRQoL) in kidney stone patients, and evaluate the association between these and disease specific, psychometric health status-based HRQoL scores (obtained via the Wisconsin Stone Quality of Life [WISQOL]). MethodsOne hundred four adults with urolithiasis, as well as 78 young healthy adults without history of urolithiasis (controls) were consecutively enrolled, meeting the predetermined recruitment goal. Each participant completed the SF-36 v2 (from which SF-6D utility is calculated) and EQ-5D questionnaires, while urolithiasis patients additionally completed the WISQOL. Relationship between health utility and WISQOL scores was evaluated using Pearson's test and multivariable linear regression analysis (MVA). Construct validity of the utilities for urolithiasis was assessed by comparing utilities for patients vs controls, and for symptomatic vs asymptomatic patients. ResultsMean (SD) WISQOL standard score, SF-6D, and EQ-5D utilities in stone patients respectively were 63 (29.1), 0.68 (0.16), and 0.83 (0.17). SF-6D and EQ-5D utilities significantly correlated with WISQOL standard score (Pearson's r = 0.87 and 0.58, respectively; each P <.0001). On MVA, WISQOL standard score was a significant predictor of SF-6D and EQ-5D utilities, explaining 72.1% and 33.9% of the respective variances in the utilities. Median SF-6D and EQ-5D utility were significantly lower in patients vs controls (each P ≤.0009), and in symptomatic vs asymptomatic patients (each P ≤ .0002). ConclusionSF-6D, more so than EQ-5D utilities in urolithiasis patients are strongly associated with disease-specific WISQOL scores, suggesting they are optimal for preference-based HRQoL assessment in this population. Construct validity of the utilities in stone disease was demonstrated.

A two-step procedure to generate utilities for the Infant health-related Quality of life Instrument (IQI).

Because of a lack of preference-based health-related quality of life (HRQoL) instruments suitable for infants aged 0-12 months, we previously developed the Infant QoL Instrument (IQI). The present study aimed to generate an algorithm to estimate utilities for the IQI. Via an online survey, respondents from the general population and primary caregivers from China-Hong Kong, the UK, and the USA were presented 10 discrete choice scenarios based on the IQI classification system. An additional sample of respondents from the general population were also asked if they considered the examined health states to be worse than death. Coefficients for the IQI item levels were obtained with a conditional logit model based on the responses of the primary caregivers for IQI states only. These coefficients were then normalized using the rank-ordered logit model based on the responses from the general population who assessed "death" as a choice option. In this way, the values were rescaled from full health (1.0) to death (0.0), and consequently, they became suitable for the computation of quality-adjusted life years. The total sample consisted of 1409 members of the general population and 1229 primary caregivers. Results indicated that, out of the 7 IQI items ("sleeping," "feeding," "breathing," "stooling/poo," "mood," "skin," and "interaction"), "breathing" had the highest impact on the HRQoL of infants. Moreover, except for "stooling," all item levels were statistically significant. The general population sample considered none of the health states as worse than death. The utility value for the worst health state was 0.015 (State 4444444). The IQI is the first generic instrument to assess overall HRQoL in 0-1-year-old infants by providing values and utilities. Using discrete choice experiments, we demonstrated that it is possible to derive utilities of infant health states. The next step will be to collect IQI values in a clinical population of infants and to compare these values with those of other instruments.

Estimating patient-reported outcomes for glaucoma management: A cross-sectional study.

To identify important explanatory variables of four patient-reported outcomes (PROs): vision-related quality of life (VRQoL), preference-based health-related quality of life (HRQoL), social support and community integration and depressive symptoms. Cross-sectional study conducted at one ophthalmic practice in a hospital setting. Patients with a diagnosis of glaucoma or glaucoma suspect (n=250) were sequentially recruited. Patients with language restrictions were excluded. Data were collected through medical chart reviews and face-to-face interviews. The PROs were measured using validated tools. Candidate models for predicting PROs from explanatory variables were constructed using linear and logistic regression, as well as classification and regression trees. Through leave-one-out cross-validation, the performance of each model was assessed in terms of mean absolute error. Use of mobility aids, best corrected visual acuity (BCVA), income, and living arrangements were most predictive of VRQoL, social support, and community integration. Use of mobility aids was also most predictive of the presence of depressive symptoms, and BCVA with preference-based HRQoL. Although promising associations were discovered, the models based on commonly collected clinical variables had limited ability to accurately predict individual patient PROs. Thus, although this study identifies clinical and demographic variables that are most predictive of PROs, routine collection of PROs in clinical practice may be necessary to obtain a complete picture of the quality of life of glaucoma patients.

The construct validity of the Child Health Utility 9D-DK instrument

BackgroundRelative to their application with adults there is currently little information about the application of preference-based health-related quality of life (HRQL) instruments among populations of young people. The Child Health Utility 9D (CHU9D) is a paediatric-specific generic preference-based HRQL instrument, recently translated and linguistically validated into Danish (CHU9D-DK). The purpose of this study was to investigate the construct validity of the CHU9D-DK in a sample of Danish high school students.MethodsAll students attending a Danish High School were invited to participate in a web-based survey in January 2018 (N = 272). The survey included the CHU9D-DK, the young adult version of the Pediatric Quality of Life Inventory™ 4.0 Generic Core Scales (PedsQL), self-reported health status, presence/absence of disability/chronic diseases, life satisfaction, and socio-economic questions. CHU9D-DK utility scores were generated by employing the two scoring algorithms developed from adults in the UK and adolescents in Australia, respectively. Internal consistency, reliability and construct validity of the CHU9D-DK instrument were investigated.ResultsTwo hundred and twenty-eight (84%) students consented to participate and completed the survey. The mean ± (standard deviation) values of the CHU9D-DK utilities were 0.84 (0.11) when the UK adult algorithm was applied and 0.70 (0.22), when the Australian adolescent algorithm was applied. The mean PedsQL score was 82.32 (13.14). The CHU9D-DK showed good internal consistency reliability (Cronbach’s alpha = 0.803). Higher levels of health status and life satisfaction were significantly associated with higher CHU9D-DK utility scores regardless of which scoring algorithm was applied (p-values < 0.001). Students living with a disability/chronic disease exhibited significantly lower utility scores relative to their healthy peers (p-values < 0.05). Higher socio-economic status (approximated by financial situation and frequency of family vacations) was also associated with higher utility scores (p-values < 0.005).ConclusionThe CHU9D-DK demonstrated good psychometric performance overall and shows potential as a valid and reliable instrument for assessing the HRQL of Danish young people.Trial registrationClinicalTrials.gov identifier: NCT03391999, Registered 15 October 2017.

Measuring the impact of chronic conditions and associated multimorbidity on health-related quality of life in the general population in Hong Kong SAR, China: A cross-sectional study.

ObjectivesThe aims of this study were to 1) evaluate the impact of eight common chronic conditions and multimorbidity on preference-based health-related quality of life (HRQoL), and 2) estimate the minimally important difference (MID) in the general population of Hong Kong (HK).DesignData were analyzed using secondary data analysis based on a cross-sectional, population-based validation study of HK’s general population.ParticipantsA representative sample was recruited across eighteen geographical districts in HK, and 1,014 HK Chinese residents aged 18 years and older participated in the survey. The prevalence of chronic conditions among the respondents was 30.3%.InterventionsThe HRQoL was assessed using the locally validated version of EQ-5D-5L. The five-dimension descriptive system, and the utility scores of EQ-5D-5L were used as the dependent variable in the study. Eight common chronic conditions, multimorbidity, and demographic characteristics were defined as predictors in the analysis. Chi-squared test, analysis of variance (ANOVA), logistic regression, and Tobit regression models were used to analyze the data. A simulation-based approach was used to calculate the MID based on instrument-defined single level transitions.ResultsThe findings indicated that respondents with physical disabilities were more likely to report problems on all five dimensions of the EQ-5D-5L than those with other chronic conditions. In addition, respondents with multiple chronic conditions were more likely to report health problems and lower utility scores of EQ-5D-5L. The mean of MID estimates among the respondents in HK was 0.093 (standard deviation = 0.001), which is higher than in other Asian countries.ConclusionsThe findings suggest that having more chronic conditions is strongly associated with a lower HRQoL. Healthcare reforms to address foreseeable challenges arising as more patients live with chronic conditions and multimorbidity could improve the HRQoL of HK citizens.

PNS384 HEALTH-RELATED QUALITY OF LIFE (HRQOL) MEASURED BY PROMIS PREFERENCE SCORE (PROPR) IN THE GENERAL POPULATION OF THE UNITED KINGDOM, FRANCE, AND GERMANY

The PROMIS Preference score (PROPr) is a new generic, societal, and preference-based health-related quality of life (HrQoL) score for Quality-adjusted life years (QALY) in cost-effectiveness analyses, promising to overcome limitations of existing legacy measures such as ceiling effects. It is the first HrQoL score using modern instruments based on Item Response Theory (IRT). The PROPr contains seven PROMIS domains: cognition, depression, fatigue, pain, physical function, sleep disturbance, and ability to participate in social roles and activities. It was valuated in US preferences using standard gamble (SG) online interviews. Aim of this study is to measure HrQoL by PROPr in the general population of three European countries and to compare it to HrQoL measured by the EQ-5D-5L index value. We collected PROMIS-29 Profile and EQ-5D-5L in three samples of the general population in the United Kingdom (n=1,509), France (n=1,501), and Germany (n=1,502). For the missing cognition domain in the PROMIS-29 Profile we assumed population average. Descriptive statistical analyses compared both scores across age and gender, their correlation, and distribution over the full range of health. The mean PROPr (UK 0.48, France 0.54, Germany 0.48; p<0.01) and the mean EQ-5D-5L index value (UK 0.79, France 0.82, Germany 0.88, p<0.01) show significant difference (UK d=0.31; France d=0.29; Germany d=0.39; p<0.01). The Pearson correlation coefficient is similar (UK 0.76; France 0.72; Germany 0.66) compared to 0.53 (95%CI: 0.33; 0.68) in German patients and 0.70 in the US valuation. In all countries, both scores show similar discrimination across gender and age. The PROPr is approximately normally distributed while the EQ-5D-5L index value shows ceiling effects. In three independent samples, our analyses consistently confirm earlier results suggesting that PROPr and EQ-5D-5L index value are not equivalent, effecting QALY in cost-effectiveness analyses. Valuation of PROPr in country-specific preferences with time-trade-off (TTO) would make comparison more valid.

Impact of cyclosporin treatment on health-related quality of life of patients with alopecia areata

Introduction Alopecia areata (AA) is a disfiguring disease with substantial psychological burden. No studies explore the efficacy of pharmacotherapy through health-related quality of life (HRQoL) using both disease-specific and generic quality of life (QoL) instruments. We present the first study to elicit health utility from patients with AA and to evaluate the efficacy of cyclosporin in relation to HRQoL using both measures. Methods Participants with moderate to severe AA from a placebo-controlled randomized trial investigating cyclosporin were administered the generic preference-based HRQoL instrument, Assessment of Quality of Life-8D (AQoL-8D) and the disease-specific HRQoL instrument, Alopecia Areata Symptom Impact Scale (AASIS). HRQoL was measured at each study visit and compared to baseline. Results A number of 32 participants were analyzed. The mean health utility was 0.748. At 3 months, the cyclosporin group had trends for greater improvement in HRQoL across 6 of 8 AQoL-8D dimensions and 5 of 7 AASIS symptom domains compared to placebo. HRQoL was lower than Australian population norms across 6 of 8 AQoL-8D dimensions. Conclusions Patients with AA had a mean health utility of 0.748. Treatment with cyclosporin 4 mg/kg/d for 3 months resulted in trends for improvement of HRQoL across multiple dimensions in both disease-specific and generic measures. Capsule summary The mean health utility for patients with AA was 0.748. Patients with alopecia areata have impaired health-related quality of life across 6 of 8 AQoL-8D dimensions compared to population norms. Treatment with oral cyclosporin for moderate to severe AA resulted in trends for improvement in QoL across multiple dimensions.

Patient-assigned health utility values for controlled and uncontrolled pemphigus vulgaris and foliaceus.

The assessment of health-related quality of life (HRQoL) in patients with pemphigus is now of increasing interest due to the availability of highly effective new therapies. Preference-based HRQoL values or health utilities required for medical and financial decision-making are not yet available directly from pemphigus patients. To obtain health utility values for current health and hypothetical health states from the perspective of pemphigus patients. A cross-sectional questionnaire survey was carried out with pemphigus patients. Disease severity was rated by Autoimmune Bullous Skin Disorder Intensity Score (ABSIS). Patients were asked to evaluate their current health as well as three common hypothetical pemphigus health states [uncontrolled pemphigus vulgaris (PV), uncontrolled pemphigus foliaceus (PF) and controlled PV/PF] by using composite time trade-off (cTTO). Multiple regression was applied to explore determinants of utility values. Responses of 108 patients (64.8% women, mean age 57.4years) were analysed. Mean ABSIS score was 11.6. The mean utility values for the hypothetical uncontrolled PV, uncontrolled PF and controlled PV/PF health states were 0.41, 0.52 and 0.66 with cTTO. The mean cTTO scores for current health were higher compared with the hypothetical health states (0.76; P<0.001). Patients with higher ABSIS, worse pain intensity scores and those having a caregiver reported lower utility values for current health (P<0.05). In pemphigus, HRQoL impairment expressed in utility values seems to be considerable, especially in comparison with other chronic dermatological conditions (e.g. psoriasis, atopic eczema, chronic hand eczema). These health utilities inform physicians, policymakers and funders about the overall extent of health loss in pemphigus and provide evidence to guide medical decisions and cost-effectiveness analyses of treatment strategies. Future research is needed to evaluate the caregiver burden in pemphigus.

Generic preference-based health-related quality of life in children with neurodevelopmental disorders: a scoping review.

AimTo describe how generic preference‐based health‐related quality of life (HRQoL) instruments have been used in research involving children with neurodevelopmental disorders (NDD).MethodA systematic search of nine databases identified studies that used generic preference‐based HRQoL instruments in children with NDD. Data extracted following the Preferred Reporting Items for Systematic Review and Meta‐Analyses extension for Scoping Review guidelines included type of NDD, instrument used, respondent type, justification, and critical appraisal for these selections.ResultsThirty‐six studies were identified: four cost–utility analyses; 15 HRQoL assessments; five economic burden studies; three intervention studies; and nine ‘other’. The Health Utilities Index (Mark 2 and Mark 3) and EuroQoL 5D (EQ‐5D; three‐level EQ‐5D, five‐level EQ‐5D, and the youth version of the EQ‐5D) instruments were most frequently used (44% and 31% respectively). The relatively low use of these instruments overall may be due to a lack of psychometric evidence, inconsistency in justification for and lack of clarity on appropriate respondent type and age, and geographical challenges in applying preference weights.InterpretationThis study highlights the dearth of studies using generic preference‐based HRQoL instruments in children with NDD. The use of cost–utility analysis in this field is limited and validation of these instruments for children with NDD is needed. The quality of data should be considered before guiding policy and care decisions.What this paper adds Limited use of generic preference‐based health‐related quality of life (HRQoL) instruments in studies on children with neurodevelopmental disorders.Only 11% of studies were cost–utility analyses.Inconsistencies in justification for choosing generic preference‐based HRQoL instruments and respondent types.

Economic evaluation of the NET intervention versus guideline dissemination for management of mild head injury in hospital emergency departments

BackgroundEvidence-based guidelines for the management of mild traumatic brain injury (mTBI) in the emergency department (ED) are now widely available, and yet, clinical practice remains inconsistent with the guidelines. The Neurotrauma Evidence Translation (NET) intervention was developed to increase the uptake of guideline recommendations and improve the management of minor head injury in Australian emergency departments (EDs). However, the adoption of this type of intervention typically entails an upfront investment that may or may not be fully offset by improvements in clinical practice, health outcomes and/or reductions in health service utilisation. The present study estimates the cost and cost-effectiveness of the NET intervention, as compared to the passive dissemination of the guideline, to evaluate whether any improvements in clinical practice or health outcomes due to the NET intervention can be obtained at an acceptable cost.Methods and findingsStudy setting: The NET cluster randomised controlled trial [ACTRN12612001286831]. Study sample: Seventeen EDs were randomised to the control condition and 14 to the intervention. One thousand nine hundred forty-three patients were included in the analysis of clinical practice outcomes (NET sample). A total of 343 patients from 14 control and 10 intervention EDs participated in follow-up interviews and were included in the analysis of patient-reported health outcomes (NET-Plus sample). Outcome measures: Appropriate post-traumatic amnesia (PTA) screening in the ED (primary outcome). Secondary clinical practice outcomes: provision of written information on discharge (INFO) and safe discharge (defined as CT scan appropriately provided plus PTA plus INFO). Secondary patient-reported, post-discharge health outcomes: anxiety (Hospital Anxiety and Depression Scale), post-concussive symptoms (Rivermead), and preference-based health-related quality of life (SF6D). Methods: Trial-based economic evaluations from a health sector perspective, with time horizons set to coincide with the final follow-up for the NET sample (2 months post-intervention) and to 1-month post-discharge for the NET-Plus sample. Results: Intervention and control groups were not significantly different in health service utilisation received in the ED/inpatient ward following the initial mTBI presentation (adjusted mean difference $23.86 per patient; 95%CI − $106, $153; p = 0.719) or over the longer follow-up in the NET-plus sample (adjusted mean difference $341.78 per patient; 95%CI − $58, $742; p = 0.094). Savings from lower health service utilisation are therefore unlikely to offset the significantly higher upfront cost of the intervention (mean difference $138.20 per patient; 95%CI $135, $141; p < 0.000). Estimates of the net effect of the intervention on total cost (intervention cost net of health service utilisation) suggest that the intervention entails significantly higher costs than the control condition (adjusted mean difference $169.89 per patient; 95%CI $43, $297, p = 0.009). This effect is larger in absolute magnitude over the longer follow-up in the NET-plus sample (adjusted mean difference $505.06; 95%CI $96, $915; p = 0.016), mostly due to additional health service utilisation. For the primary outcome, the NET intervention is more costly and more effective than passive dissemination; entailing an additional cost of $1246 per additional patient appropriately screened for PTA ($169.89/0.1363; Fieller’s 95%CI $525, $2055). For NET to be considered cost-effective with 95% confidence, decision-makers would need to be willing to trade one quality-adjusted life year (QALY) for 25 additional patients appropriately screened for PTA. While these results reflect our best estimate of cost-effectiveness given the data, it is possible that a NET intervention that has been scaled and streamlined ready for wider roll-out may be more or less cost-effective than the NET intervention as delivered in the trial.ConclusionsWhile the NET intervention does improve the management of mTBI in the ED, it also entails a significant increase in cost and—as delivered in the trial—is unlikely to be cost-effective at currently accepted funding thresholds. There may be a scope for a scaled-up and streamlined NET intervention to achieve a better balance between costs and outcomes.Trial registrationAustralian New Zealand Clinical Trials Registry ACTRN12612001286831, date registered 12 December 2012.

A Norwegian 15D value algorithm: proposing a new procedure to estimate 15D value algorithms

PurposeSo far there is no Norwegian value algorithm to inform healthcare decision making. The 15D health state values estimated with the original 15D valuation procedure tend to be higher than the values of other generic preference-based health-related quality of life (HRQoL) instruments. The main purpose of this study was to use a new 15D valuation procedure to estimate Norwegian 15D health state values and to explore their empirical performance.MethodsThe visual analogue scale was used to collect 15D valuation data in a representative sample of the Norwegian general population. The new procedure used fewer valuation tasks and anchored the 15D health state values in an empirically assessed range. The Norwegian 15D health state values were compared to the values of five HRQoL instruments which were provided by Norwegian residents belonging to seven disease groups and a healthy population.ResultsThe Norwegian 15D health state values ranged from 1 to − 0.52. Compared to 15D health state values estimated with the original procedure, the Norwegian 15D health state values were lower and more in line with values of other HRQoL instruments.ConclusionsThe new 15D valuation procedure is simpler, links the 15D health state values better to the requirements of the QALY model, and provides an empirically-based range. We recommend using the new valuation procedure in future 15D valuation studies, and the Norwegian health state values for use in 15D-based health economic analyses in Norway.

Responsiveness and minimally important difference of SF-6D and EQ-5D utility scores for the treatment of pelvic organ prolapse

Utility preference scores are standardized, generic, health-related quality of life (HRQOL) measures that quantify disease severity and burden and summarize morbidity on a scale from 0 (death) to 1 (optimal health). Utility scores are widely used to measure HRQOL and in cost-effectiveness research. To determine the responsiveness, validity properties, and minimal important difference (MID) of utility scores, as measured by the Short Form 6D (SF-6D) and EuroQol (EQ-5D), in women undergoing surgery for pelvic organ prolapse (POP). This study combined data from 4 large, U.S., multicenter surgical trials enrolling 1321 women with pelvic organ prolapse. We collected condition-specific quality of life data using the Pelvic Floor Distress Inventory (PFDI) and Pelvic Floor Impact Questionnaire (PFIQ). A subset of women completed the SF6D; women in 2 trials also completed the EQ5D. Mean utility scores were compared from baseline to 12 months after surgery. Responsiveness was assessed using effect size (ES) and standardized response mean (SRM). Validity properties were assessed by (1) comparing changes in utility scores at 12 months between surgical successes and failures as defined in each study, and (2) correlating changes in utility scores with changes in the PFDI and PFIQ. MID was estimated using both anchor-based (SF-36 general health global rating scale "somewhat better" vs "no change") and distribution-based methods. The mean SF-6D score improved 0.050, from 0.705 ± 0.126 at baseline to 0.761 ± 0.131 at 12 months (P < .01). The mean EQ-5D score improved 0.060, from 0.810 ± 0.15 at baseline to 0.868 ± 0.15 at 12 months (P < .01). The ES (0.13-0.61) and SRM (0.13-0.57) were in the small-to-moderate range, demonstrating the responsiveness of the SF-6D and EQ-5D similar to other conditions. SF-6D and EQ-5D scores improved more for prolapse reconstructive surgical successes than for failures. The SF-6D and EQ-5D scores correlated with each other (r= 0.41; n= 645) and with condition-specific instruments. Correlations with the PFDI and PFIQ and their prolapse subscales were in the low to moderate range (r= 0.09-0.38), similar to other studies. Using the anchor-based method, the MID was 0.026 for SF-6D and 0.025 for EQ-5D, within the range of MIDs reported in other populations and for other conditions. These findings were supported by distribution-based estimates. The SF-6D and EQ-5D have good validity properties and are responsive, preference-based, utility and general HRQOL measures for women undergoing surgical treatment for prolapse. The MIDs for SF-6D and EQ-5D are similar and within the range found for other medical conditions.

QL1 - VALIDATION OF PROMIS PREFERENCE SCORE (PROPR) IN GERMAN PATIENTS WITH RHEUMATOLOGICAL CONDITIONS

The PROMIS Preference score (PROPr) is a new generic, societal, and preference-based health-related quality of life (HRQoL) score which can be used as utility score for Quality-adjusted life years (QALY) in cost-effectiveness analyses. It is the first of its kind using modern instruments based on Item Response Theory (IRT). The PROPr contains seven PROMIS domains: cognition, depression, fatigue, pain, physical function, sleep disturbance, and ability to participate in social roles and activities. It was developed with the preferences of the US general population. The aim of this study is to validate the PROPr in independent data of German patients and compare it to their outcomes in the established EQ-5D-5L index value. We collected PROPr and EQ-5D-5L index value from 68 patients in the rheumatology ward at Charité University Medicine Berlin between March 18th and Mai 31st 2018. We conducted descriptive statistical analyses comparing both scores across age, gender and conditions, as well as their correlation and distribution over the full range of health. The mean PROPr (0.35, 95%CI: 0.32; 0.38) score and the mean EQ-5D-5L index value (0.75, 95%CI: 0.70; 0.80) differ significantly (d = 0.40, p < 0.001). The Pearson correlation coefficient between both scores was observed to be 0.53 (95%CI: 0.33; 0.68) compared to 0.70 in the US valuation survey. Both scores have similar discrimination across gender but not across age and conditions. While the PROPr is approximately normally distributed, the EQ-5D-5L index value shows a ceiling effect, indicating a limited range of measurement. Our analysis suggests that PROPr and EQ-5D-5L index value are not equivalent in German patients, which would greatly affect QALY in cost-effectiveness analyses. Evaluation of PROPr health states in German preferences and larger samples could resolve this. Further research should address which instrument more accurately reflects health utility.

PCV47 - Preference-Based Health-Related Quality Of Life For Heart Disease Patients In Japan

The study examines preference-based health-related quality of life (utility) for patients with heart disease in Japan. The study targeted patients with heart disease who were hospitalized at 3 hospitals. The study also used New York Heart Association (NYHA) classification for severity level of heart disease. Utility score was obtained by using EQ-5D-5L (EuroQol-5 Dimension-5 Level) in the evaluation for health-related QOL. In the study, an evaluation by therapist was also conducted to examine a difference in survey responses between patient himself/herself (Self) and proxy. For carrying out the study, we had ethical review at each hospital and received consent from each patient in writing. The study conducted regression analysis with use of STATA15.0 for investigating an influence on patient attributes in the statistical processing. The study received responses from total 174 patients with heart disease. The average age and gender was 68.7 ± 12.6 years old and 109 males (62.6%). The disease breakdown was 37 patients for heart failure, 43 patients for cardiac infarction, 10 patients for aorta dissection, 54 patients for valve disease, 18 patients for cardiomyopathy and 12 patients for other diseases. In NYHA classification, it indicates I=43, II=99, III=31, and IV=1. Utility score by Self in each NYHA classification was 0.84, 0.78, 0.64, and 0.57 respectively, and they were solely extracted as significant explanatory variables by regression analysis (p <0.001). In addition, for the responses by proxy, the score was 0.87, 0.78, 0.58, and 0.40 in Proxy 1 and 0.86, 0.76, 0.62, and 0.55 in Proxy 2 respectively. The study found a difference by NYHA in any of these responses. The study found a clear difference in utility score for NYHA classification. It was proved that EQ-5D-5L is an useful tool for preference-based health-related quality of life evaluation for patients with heart disease.