Health Utilities Index Mark Research Articles

Cross-sectional validation of the PROMIS-Preference scoring system by its association with social determinants of health.

PROMIS-Preference (PROPr) is a generic, societal, preference-based summary score that uses seven domains from the Patient-Reported Outcomes Measurement Information System (PROMIS). This report evaluates construct validity of PROPr by its association with social determinants of health (SDoH). An online panel survey of the US adult population included PROPr, SDoH, demographics, chronic conditions, and four other scores: the EuroQol-5D-5L (EQ-5D-5L), Health Utilities Index (HUI) Mark 2 and Mark 3, and the Short Form-6D (SF-6D). Each score was regressed on age, gender, health conditions, and a single SDoH. The SDoH coefficient represents the strength of its association to PROPr and was used to assess known-groups validity. Convergent validity was evaluated using Pearson correlations between different summary scores and Spearman correlations between SDoH coefficients from different summary scores. From 4142 participants, all summary scores had statistically significant differences for variables related to education, income, food and financial insecurity, and social interactions. Of the 42 SDoH variables tested, the number of statistically significant variables was 27 for EQ-5D-5L, 17 for HUI Mark 2, 23 for HUI Mark 3, 27 for PROPr, and 27 for SF-6D. The average SDoH coefficients were -0.086 for EQ-5D-5L, -0.039 for HUI Mark 2, -0.063 for HUI Mark 3, -0.064 for PROPr, and -0.037 for SF-6D. Despite the difference in magnitude across the measures, Pearson correlations were 0.60 to 0.76 and Spearman correlations were 0.74 to 0.87. These results provide evidence of construct validity supporting the use of PROPr monitor population health in the general US population.

Safety and cost-effectiveness of individualised screening for diabetic retinopathy: the ISDR open-label, equivalence RCT

Aims/hypothesisUsing variable diabetic retinopathy screening intervals, informed by personal risk levels, offers improved engagement of people with diabetes and reallocation of resources to high-risk groups, while addressing the increasing prevalence of diabetes. However, safety data on extending screening intervals are minimal. The aim of this study was to evaluate the safety and cost-effectiveness of individualised, variable-interval, risk-based population screening compared with usual care, with wide-ranging input from individuals with diabetes.MethodsThis was a two-arm, parallel-assignment, equivalence RCT (minimum 2 year follow-up) in individuals with diabetes aged 12 years or older registered with a single English screening programme. Participants were randomly allocated 1:1 at baseline to individualised screening at 6, 12 or 24 months for those at high, medium and low risk, respectively, as determined at each screening episode by a risk-calculation engine using local demographic, screening and clinical data, or to annual screening (control group). Screening staff and investigators were observer-masked to allocation and interval. Data were collected within the screening programme. The primary outcome was attendance (safety). A secondary safety outcome was the development of sight-threatening diabetic retinopathy. Cost-effectiveness was evaluated within a 2 year time horizon from National Health Service and societal perspectives.ResultsA total of 4534 participants were randomised. After withdrawals, there were 2097 participants in the individualised screening arm and 2224 in the control arm. Attendance rates at first follow-up were equivalent between the two arms (individualised screening 83.6%; control arm 84.7%; difference −1.0 [95% CI −3.2, 1.2]), while sight-threatening diabetic retinopathy detection rates were non-inferior in the individualised screening arm (individualised screening 1.4%, control arm 1.7%; difference −0.3 [95% CI −1.1, 0.5]). Sensitivity analyses confirmed these findings. No important adverse events were observed. Mean differences in complete case quality-adjusted life-years (EuroQol Five-Dimension Questionnaire, Health Utilities Index Mark 3) did not significantly differ from zero; multiple imputation supported the dominance of individualised screening. Incremental cost savings per person with individualised screening were £17.34 (95% CI 17.02, 17.67) from the National Health Service perspective and £23.11 (95% CI 22.73, 23.53) from the societal perspective, representing a 21% reduction in overall programme costs. Overall, 43.2% fewer screening appointments were required in the individualised arm.Conclusions/interpretationStakeholders involved in diabetes care can be reassured by this study, which is the largest ophthalmic RCT in diabetic retinopathy screening to date, that extended and individualised, variable-interval, risk-based screening is feasible and can be safely and cost-effectively introduced in established systematic programmes. Because of the 2 year time horizon of the trial and the long time frame of the disease, robust monitoring of attendance and retinopathy rates should be included in any future implementation.Trial registrationISRCTN 87561257FundingThe study was funded by the UK National Institute for Health Research.Graphical abstract

Impact of Superior Canal Dehiscence Syndrome on Health Utility Values: A Prospective Case-Control Study.

Introduction: Superior canal dehiscence syndrome (SCDS) is a condition characterized by a defect in the bone overlying the superior semicircular canal, creating a third mobile window into the inner ear. Patients can experience disabling symptoms and opt for surgical management. Limited data are available on the impact of SCDS on health-related quality of life (HRQoL) and disease-specific HRQoL more specifically.Objective: To perform a prospective analysis on generic HRQoL in SCDS patients compared to healthy age-matched controls.Methods: A prospective study was performed on patients diagnosed with SCDS and who did not undergo reconstructive surgery yet. Patients were recruited between November 2017 and January 2020 and asked to complete the Health Utility Index (HUI) Mark 2 (HUI2)/Mark 3 (HUI3) questionnaire. For the control group, age-matched participants without otovestibular pathology or other chronic pathology were recruited. The multi-attribute utility function (MAUF) score was calculated for the HUI2 and HUI3. Results of both groups were compared using the Mann-Whitney U test.Results: A total of 20 patients completed the questionnaire. Age ranged from 37 to 79 years with a mean age of 56 years (45% males and 55% females). The control group consisted of 20 participants with a mean age of 56.4 years and ranged from 37 to 82 years (35% males and 65% females). For the case group, median HUI2 MAUF score was 0.75 and median HUI3 MAUF score was 0.65. For the control group, the median scores were 0.88 and 0.86 respectively. There was a statistically significant difference for both HUI2 (p = 0.024) and HUI3 (p = 0.011). SCDS patients had a worse generic HRQoL than age-matched healthy controls. One patient with unilateral SCDS had a negative HUI3 MAUF score (−0.07), indicating a health-state worse than death.Conclusion: SCDS patients have significantly lower health utility values than an age-matched control group. This confirms the negative impact of SCDS on generic HRQoL, even when using an instrument that is not designed to be disease-specific but to assess health state in general. These data can be useful to compare impact on HRQoL among diseases.

Cost-effectiveness Analysis of Abdominal-based Autogenous Tissue and Tissue-expander Implant following Mastectomy.

Background:Patients who had undergone both autologous abdominal tissue (AAT) and tissue expander and implant (TE/I) breast reconstruction reported satisfaction with their reconstruction. While aesthetics and quality of life are important, the cost associated with these procedures must also be considered when choosing one method over the other. The objective of this study was to determine whether AAT-based breast reconstruction is cost-effective compared with 2-stage TE/I reconstruction at a 12-month follow-up.Methods:Thirty-five patients consented and complied to participate in the study with a follow-up of 12 months. The effectiveness of both AAT and TE/I was measured using the Health Utilities Index Mark 3 (HUI-3). From the HUI-3 results, quality-adjusted life years were calculated for each reconstructive approach. Direct healthcare and productivity costs were captured from surgeon billing codes, patient files, and patient diaries. The perspectives of both the Ministry of Health and of society were considered.Results:From the perspectives of both the Ministry of Health and of society, AAT was less effective and more costly when compared with TE/I.Conclusions:In this economic evaluation, TE/I dominated AAT, in that TE/I was more effective and less costly as compared with AAT from the perspectives of both the Ministry of Health and of society at 12 months of follow-up. This conclusion should be interpreted with caution due to a small sample size, the short timespan of the study, and the nonrandomized study design.

A Scoring Algorithm for Deriving Utility Values from the Neuro-QoL for Patients with Multiple Sclerosis.

The Neuro-QoL is a standardized approach to assessing health-related quality of life in people with neurological conditions, including multiple sclerosis (MS). Item banks were developed with item response theory (IRT) methodology so items are calibrated along a continuum of each construct. The purpose of this study was to develop a preference-based scoring algorithm for the Neuro-QoL to derive utilities that could be used in economic modeling. With input from neurologists, 6 Neuro-QoL domains were selected based on relevance to MS and used to define health states for a utility elicitation study in the United Kingdom. General population participants and individuals with MS valued the health states and completed questionnaires (including Neuro-QoL short forms). The Neuro-QoL Utility Scoring System (NQU) was derived based on multi-attribute utility theory using data from the general population sample. Single-attribute disutility functions for 6 Neuro-QoL domains were estimated using isotonic regression with linear interpolation and then combined with a multiplicative model. NQU validity was assessed using MS participant data. Interviews were completed with 203 general population participants (50.2% female; mean age = 45.0 years) and 62 participants with MS (62.9% female; mean age = 46.1 years). Mean (SD) NQU scores were 0.94 (0.06) and 0.82 (0.13) for the general population and MS samples, respectively. The NQU demonstrated known-groups validity by differentiating among subgroups categorized based on level of disability. The NQU demonstrated convergent validity via correlations with generic measures (0.66 and 0.63 with EQ-5D-5L and Health Utilities Index Mark 3, respectively; both P < 0.001). With the NQU, utilities can be derived from any MS treatment group, subgroup, or patient sample who completes items from 6 Neuro-QoL domains. Because the Neuro-QoL is frequently used with MS patients, the NQU greatly expands the options for quantifying outcomes in cost-utility analyses conducted to inform allocation of resources for MS treatment.

Comparing the Clinical and Cost-Effectiveness of Abdominal-based Autogenous Tissue and Tissue-Expander Implant: A Feasibility Study.

To determine the superiority of autologous abdominal tissue (AAT) or tissue-expander implant (TE/I) reconstruction, a robust comparative cohort study is required. This study sought to determine the feasibility of a future large pragmatic cohort study comparing clinical and cost-effectiveness of AAT and TE/I at 12 months postoperative. Potential participants were screened during consultation with their surgeon. Three health-related quality-of-life scales, the Health Utility Index Mark 3, the 12-Item Short Form Health Survey, and the BREAST-Q were used preoperatively, 1, 6, and 12 months postoperatively. Direct medical costs and postoperative patient/caregiver productivity loss were collected using patient diaries. Feasibility was assessed through patient recruitment rates and compliance of patients and study staff to complete required study documentation. Sixty-three patients consented to participate, 44 completed baseline questionnaires; the feasibility objective of recruiting 80% of eligible patients was not met. A 90% completion rate for patient questionnaires was seen at 1-month follow-up and decreased up to 12 months. Quality-adjusted life years were calculated at 0.77 and 0.89 for the AAT and TE/I group, respectively. Case report form completion by study staff and patient diary completion was moderate and low, respectively. Collaborating with hospital case-costing specialists to identify direct medical costs was reliable and efficient. A future large-scale study is feasible. However, due to a diminishing rate of questionnaire completion, almost twice as many patients need to be recruited than expected to have adequate power. Cost data collection from hospital sources was reliable. Case report forms need to be tailored more toward a busy hospital setting.

Adult Measures of General Health and Health-Related Quality of Life.

s was very high (intraclass correlation coefficient 0.996). Any disagreements were resolved through a discussion between all authors. Given the large number of potentially relevant measures identified, only those measures that were used at least 4 times in the screened abstracts were selected for this review. Consequently, this report provides reviews of 4 generic health profiles: the Medical Outcomes Study Short Form 36 and Short Form 12 Health Surveys, the Nottingham Health Profile, and the Sickness Impact Profile, and 4 health utility measures: the Medical Outcomes Study Short Form 6D, the Health Utilities Index Mark 3, Quality of Well-Being Scale (selfadministered), and the Assessment of Quality of Life Scale. Although the EuroQol 5-domain is also frequently used in rheumatology, the review of this measure is included in Measures of Disability article in this issue. MEDICAL OUTCOMES STUDY SHORT FORM 36-ITEM (SF-36) AND SHORT FORM 12-ITEM (SF-12) HEALTH SURVEYS

Health-Related QOL and Economic Burden of Chronic Pruritus

Chronic pruritus (CP) has considerable implications for QOL. However, its impact on health-related QOL and economic burden is not fully characterized. We administered a cross-sectional survey on 132 patients with CP using the Health Utilities Index Mark 3 instrument. Normative data from healthy adults (n= 4,187) were obtained from the Joint Canada/US Survey of Health. Quality-adjusted life-year loss and economic costs were estimated on the basis of Health Utilities Index Mark 3 scores of patients with CP versus controls. Patients with CP had lower overall health performance than the control (0.56 ± 0.03 vs. 0.86 ± 0.003, P < 0.001). In multivariable regression, CP was associated with worse overall health performance (coefficient=-0.30, 95% confidence interval=-0.33 to-0.27), most accentuated in the domains of pain (coefficient=-0.24, confidence interval=-0.28 to-0.21) and emotion (coefficient=-0.11, confidence interval=-0.13 to-0.10). The reduced Health Utilities Index Mark 3 score correlated with 5.5 average lifetime quality-adjusted life-years lost per patient. Using conservative estimates for willingness to pay, the quality-adjusted life-year loss translated to an individual lifetime economic burden of $274,921 and a societal burden of $88.8 billion. CP is associated with significant QOL impairment. The economic burden of CP highlights the necessity for further research into management options.

Assessment of Speech Understanding After Cochlear Implantation in Adult Hearing Aid Users

Cochlear implants were approved for use in adults in the 1980s, but use remains low owing to a lack of awareness regarding cochlear implantation candidacy criteria and expected outcomes. There have been limited, small series examining the safety and effectiveness of cochlear implantation in adult hearing aid (HA) users with and without mild cognitive impairment (MCI). To investigate the safety and effectiveness of a single-ear cochlear implant in a group of optimized adult HA users with and without MCI across a variety of domains. In this nonrandomized controlled trial, a multicenter, prospective, repeated-measures investigation was conducted at 13 US institutions. The setting was academic and community-based cochlear implant programs. Eligible participants were 100 adults (aged >18 years) with postlinguistic onset of bilateral moderate sloping to profound or worse sensorineural hearing loss (≤20 years' duration). Fluent English speakers underwent an optimized bilateral HA trial for at least 30 days. Individuals with aided Consonant-Vowel Nucleus-Consonant (CNC) word score in quiet of 40% or less correct in the ear to be implanted and 50% or less correct in the contralateral ear were offered cochlear implants. The first participant was enrolled on February 20, 2017, and the last participant was enrolled on May 3, 2018. The final follow-up was on December 21, 2018. Participants received the same cochlear implant system and contralateral HA. The primary outcome measure was speech understanding in quiet (CNC word score) using both the cochlear implant and opposite ear HA. Secondary outcome measures included the following: adverse events; speech understanding in noise (AzBio signal-to-noise ratio of +10 db [+10 SNR]) Health Utilities Index Mark 3 (HUI3); Speech, Spatial, and Qualities of Hearing Questionnaire 49 (SSQ49); and Montreal Cognitive Assessment (MoCA). The median age at cochlear implantation of the 96 patients included in the trial was 71 years (range, 23-91 years), and 62 patients (65%) were male. Three serious adverse events requiring revision surgery occurred, and all resolved without sequelae. By 6 months after activation, the absolute marginal mean change in CNC word score and AzBio +10 SNR was 40.5% (95% CI, 35.9%-45.0%) and 24.1% (95% CI, 18.9%-29.4%), respectively. Ninety-one percent (87 of 96) of participants had a clinically important improvement (>15%) in the CNC word score in the implant ear. Mild cognitive impairment (MoCA total score ≤25) was observed in 48 of 81 study participants (59%) at baseline. Speech perception marginal mean improvements were similar between individuals with and without baseline MCI, with values of 40.9% (95% CI, 35.2%-46.6%) and 39.6% (95% CI, 31.8%-47.4%), respectively, for CNC word score and 27.5% (95% CI, 21.0%-33.9%) and 17.8% (95% CI, 9.0%-26.6%), respectively, for AzBio +10 SNR. Statistically significant and clinically important improvements in the HUI3 and SSQ49 were evident at 6 months. The findings of this nonrandomized controlled trial seem to indicate that cochlear implants are safe and effective in restoring speech understanding in both quiet and noise and improve quality of life in individuals with and without MCI. ClinicalTrials.gov Identifier: NCT03007472.

Hearing and Quality-of-Life Outcomes After Cochlear Implantation in Adult Hearing Aid Users 65 Years or Older

Hearing loss, especially moderate to severe forms, has the potential to negatively affect an individual's physical, social, emotional, and cognitive well-being. Moreover, having ineffective binaural hearing increases difficulty understanding speech in noise and leads to a greater degree of social isolation and loneliness and a reduced quality of life (QoL). To explore the audiometric and holistic effects of cochlear implantation in a group of adults 65 years or older compared with an optimized bilateral hearing aid condition. This ad hoc secondary analysis of a prospective, single-subject, repeated-measures nonrandomized clinical trial included 13 cochlear implantation centers across the United States. Participants 65 years or older with postlingual bilateral moderate-to-profound sensorineural hearing loss with aided Consonant-Vowel Nucleus-Consonant (CNC) word scores in quiet of 40% or less in the ear to undergo implantation and 50% or less in the contralateral ear were included in the analysis. Baseline QoL testing was performed after 1 month of optimized bilateral hearing aid use. Participants were enrolled from February 20, 2017, to May 3, 2018, and follow-up was completed December 21, 2018. Data were analyzed from March 25, 2019, to March 31, 2020. Unilateral implantation with a slim, modiolar cochlear implant device. Hearing aid use in the contralateral ear was required through the 6-month primary end-point interval. The primary objective was to evaluate speech perception before and 6 months after activation of a new cochlear implant. Secondary objectives were QoL metrics in the everyday listening condition before and 6 months after implantation. Seventy participants (51 men [73%]) with a median age of 74 (range, 65-91) years were included in the analysis. No major adverse events occurred. Mixed-model analysis with estimated marginal means and 95% CIs compared preimplantation baseline performance with 6-month postimplantation performance. A clinically important improvement in CNC words was shown in the bimodal condition, with a mean difference of 37.2% (95% CI, 32.0%-42.4%), and in the unilateral (cochlear implant only) condition, with a mean difference of 44.1% (95% CI, 39.0%-49.2%). A clinically important improvement in noise (AzBio sentences signal-to-noise ratio of +10 dB) was also shown, with a mean difference of 21.6% (95% CI, 15.7%-27.5%) in the bimodal condition and 24.5% (95% CI, 18.3%-30.7%) in the unilateral condition. The Health Utilities Index Mark 3 multiple-attribute score improved by 0.186 (95% CI, 0.136-0.234); the Speech, Spatial, and Qualities of Hearing Scale total score improved by 2.58 (95% CI, 2.18-2.99); and a novel Device Use Questionnaire reported 94% of participants were satisfied with overall hearing in the everyday listening condition. This subgroup analysis of patients 65 years or older enrolled in a within-subject clinical trial of cochlear implantation demonstrated clinically meaningful audiometric and QoL benefit with an acceptable risk profile. These findings suggest that cochlear implantation in older adults may facilitate the concept of healthy aging. ClinicalTrials.gov Identifier: NCT03007472.

Mapping clinical outcomes to generic preference-based outcome measures: development and comparison of methods.

Cost-effectiveness analysis using quality-adjusted life-years as the measure of health benefit is commonly used to aid decision-makers. Clinical studies often do not include preference-based measures that allow the calculation of quality-adjusted life-years, or the data are insufficient. 'Mapping' can bridge this evidence gap; it entails estimating the relationship between outcomes measured in clinical studies and the required preference-based measures using a different data set. However, many methods for mapping yield biased results, distorting cost-effectiveness estimates. Develop existing and new methods for mapping; test their performance in case studies spanning different preference-based measures; and develop methods for mapping between preference-based measures. Fifteen data sets for mapping from non-preference-based measures to preference-based measures for patients with head injury, breast cancer, asthma, heart disease, knee surgery and varicose veins were used. Four preference-based measures were covered: the EuroQoL-5 Dimensions, three-level version (n = 11), EuroQoL-5 Dimensions, five-level version (n = 2), Short Form questionnaire-6 Dimensions (n = 1) and Health Utility Index Mark 3 (n = 1). Sample sizes ranged from 852 to 136,327. For mapping between generic preference-based measures, data from FORWARD, the National Databank for Rheumatic Diseases (which includes the EuroQoL-5 Dimensions, three-level version, and EuroQoL-5 Dimensions, five-level version, in its 2011 wave), were used. Mixture-model-based approaches for direct mapping, in which the dependent variable is the health utility value, including adaptations of methods developed to model the EuroQoL-5 Dimensions, three-level version, and beta regression mixtures, were developed, as were indirect methods, in which responses to the descriptive systems are modelled, for consistent multidirectional mapping between preference-based measures. A highly flexible approach was designed, using copulas to specify the bivariate distribution of each pair of EuroQoL-5 Dimensions, three-level version, and EuroQoL-5 Dimensions, five-level version, responses. A range of criteria for assessing model performance is proposed. Theoretically, linear regression is inappropriate for mapping. Case studies confirm this. Flexible, direct mapping methods, based on different variants of mixture models with appropriate underlying distributions, perform very well for all preference-based measures. The precise form is important. Case studies show that a minimum of three components are required. Covariates representing disease severity are required as predictors of component membership. Beta-based mixtures perform similarly to the bespoke mixture approaches but necessitate detailed consideration of the number and location of probability masses. The flexible, bi-directional indirect approach performs well for testing differences between preference-based measures. Case studies drew heavily on EuroQoL-5 Dimensions. Indirect methods could not be undertaken for several case studies because of a lack of coverage. These methods will often be unfeasible for preference-based measures with complex descriptive systems. Mapping requires appropriate methods to yield reliable results. Evidence shows that widely used methods such as linear regression are inappropriate. More flexible methods developed specifically for mapping show that close-fitting results can be achieved. Approaches based on mixture models are appropriate for all preference-based measures. Some features are universally required (such as the minimum number of components) but others must be assessed on a case-by-case basis (such as the location and number of probability mass points). Further research is recommended on (1) the use of the monotonicity concept, (2) the mismatch of trial and mapping distributions and measurement error and (3) the development of indirect methods drawing on methods developed for mapping between preference-based measures. This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 24, No. 34. See the NIHR Journals Library website for further project information. This project was also funded by a Medical Research Council grant (MR/L022575/1).

A Review of the Methods Used to Generate Utility Values in NICE Technology Assessments for Children and Adolescents.

This review summarizes and critically examines methods used to generate utilities for child and adolescent health states in previous National Institute for Health and Care Excellence (NICE) technology assessments (TA) and highly specialized technology (HST) evaluations. We identified all NICE TA and HST evaluations in which the licensed indication for the technology included people younger than 18 and included in the review all evaluations using a cost-utility analysis. The review includes 40 TA and HST evaluations. Most assessments generated utility values with the EQ-5D scored using the adult version of the EQ-5D either exclusively (n= 16) or alongside other utility measures and direct elicitation methods of patient own utility (n= 17), although 7 did not use the EQ-5D. Eight assessments used both the EQ-5D child- and adolescent-specific preference-based measures: Health Utilities Index Mark 2 (n= 6), child- and adolescent-specific preference-based measure for atopic dermatitis (n= 1), and youth version of the EQ-5D (EQ-5D-Y) valued using the adult EQ-5D value set (n= 1) or generated using mapping and valued using the adult EQ-5D value set (n= 2). Some cost-utility analyses used age adjustment (utility subtractions, weights, and published mapping formulae) from the adult EQ-5D UK population norms to reflect the general population or disease-free health for children and adolescents (n= 9), and 1 assessment assumed full health (utility value of 1). The review found limited use of child and adolescent population-specific measures to generate health state utility values for children and adolescents in NICE technology assessments. Often assessments involve the use of an adult-specific measure to reflect the health of children.

Health status and psychological outcomes after trauma: A prospective multicenter cohort study.

Survival after trauma has considerably improved. This warrants research on non-fatal outcome. We aimed to identify characteristics associated with both short and long-term health status (HS) after trauma and to describe the recovery patterns of HS and psychological outcomes during 24 months of follow-up. Hospitalized patients with all types of injuries were included. Data were collected at 1 week 1, 3, 6, 12, and 24 months post-trauma. HS was assessed with the EuroQol-5D-3L (EQ-5D-3L) and the Health Utilities Index Mark 2 and 3 (HUI2/3). For the screening of symptoms of post-traumatic stress, anxiety and depression, the Impact of Event Scale (IES) and the Hospital Anxiety and Depression Scale (HADS) subscale anxiety (HADSA) and subscale depression (HADSD) were used. Recovery patterns of HS and psychological outcomes were examined with linear mixed model analyses. A total of 4,883 patients participated (median age 68 (Interquartile range 53-80); 50% response rate). The mean (Standard Deviation (SD)) pre-injury EQ-5D-3L score was 0.85 (0.23). One week post-trauma, mean (SD) EQ-5D-3L, HUI2 and HUI3 scores were 0.49 (0.32), 0.61 (0.22) and 0.38 (0.31), respectively. These scores significantly improved to 0.77 (0.26), 0.77 (0.21) and 0.62 (0.35), respectively, at 24 months. Most recovery occurred up until 3 months. At long-term follow-up, patients of higher age, with comorbidities, longer hospital stay, lower extremity fracture and spine injury showed lower HS. The mean (SD) scores of the IES, HADSA and HADSD were respectively 14.80 (15.80), 4.92 (3.98) and 5.00 (4.28), respectively, at 1 week post-trauma and slightly improved over 24 months post-trauma to 10.35 (14.72), 4.31 (3.76) and 3.62 (3.87), respectively. HS and psychological symptoms improved over time and most improvements occurred within 3 months post-trauma. The effects of severity and type of injury faded out over time. Patients frequently reported symptoms of post-traumatic stress. ClinicalTrials.gov identifier: NCT02508675.

Development of a multiplicative, multi-attribute utility function and eight single-attribute utility functions for the Health Utilities Index Mark 3 in Japan

BackgroundThe Health Utilities Index Mark 3 (HUI3) is a generic multi-attribute, preference-based system for assessing health-related quality of life. It is widely used overseas as an outcome measure and for estimating quality-adjusted life years. We aimed to estimate a multi-attribute and eight single-attribute utility functions for the HUI3 system based on community preferences in Japan. We conducted two preference surveys in this study. The first survey was designed to estimate a model of utility function and collect preference scores, and the second survey was designed to evaluate predictive validity of the utility function and provide independent scores. Values obtained from the feeling thermometer and standard gamble scores obtained from using a chance board were included in the preference scale. We recruited 1043 respondents (age: 20–79 years) from five cities in Japan through the general population classified by sex and age groups. Respondents were further randomly divided into a modeling group (n = 774) and a direct group (n = 263).ResultsWe acquired the estimation for eight single-attribute and a global multi-attribute utility function. The minimum expected multi-attribute utility score was − 0.002. The intraclass correlation coefficient between the directly measured utility score and the score generated by multi-attribute function in terms of 53 health conditions was 0.742.ConclusionsThe HUI3 scoring function developed in Japan has a strong theoretical and empirical basis. It will be useful in future to predict the directly measured score of health technology assessments in Japan.

Cerebral palsy in Canada, 2011-2031: results of a microsimulation modelling study of epidemiological and cost impacts

The objective of our study was to present model-based estimates and projections on current and future health and economic impacts of cerebral palsy in Canada over a 20-year time horizon (2011-2031). We used Statistics Canada's Population Health Model (POHEM)-Neurological to simulate individuals' disease states, risk factors and health determinants and to describe and project health outcomes, including disease incidence, prevalence, life expectancy, health-adjusted life expectancy, health-related quality of life and health care costs over the life cycle of Canadians. Cerebral palsy cases were identified from British Columbia's health administrative data sources. A population-based cohort was then used to generate the incidence and mortality rates, enabling the projection of future incidence and mortality rates. A utility-based measure (Health Utilities Index Mark 3) was also included in the model to reflect various 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 cerebral palsy. Although the overall crude incidence rate of cerebral palsy is projected to remain stable, newly diagnosed cases of cerebral palsy will rise from approximately 1800 in 2011 to nearly 2200 in 2031. In addition, the number of people with the condition is expected to increase from more than 75 000 in 2011 to more than 94 000 in 2031. Direct health care costs in constant 2010 Canadian dollars were about $11 700 for children with cerebral palsy aged 1-4 years versus about $600 for those without the condition. In addition, people with cerebral palsy tend to have longer periods in poorer health-related quality of life. Individuals with cerebral palsy will continue to face challenges related to an ongoing need for specialized medical care and a rising need for supportive services. Our study offers important insights into future costs and impacts associated with cerebral palsy and provides valuable information that could be used to develop targeted health programs and strategies for Canadians living with this condition.

Clinical severity measures and quality-of-life burden in patients with mycosis fungoides and Sézary syndrome: comparison of generic and dermatology-specific instruments.

Given the severe symptom burden and chronic nature of mycosis fungoides (MF) and Sézary syndrome (SS), effective assessment of quality of life (QoL) is essential to guiding patient-centred care in this population. In this study, we aim to provide a comprehensive assessment of QoL in early- and advanced-stage MF/SS and to assess the correlation of traditional measures of clinical severity with QoL measures. Between July 2017 and April 2019, outpatients at an academic medical centre with either MF/SS (n=115) or general dermatology concerns (n=115) completed generic and dermatology-specific QoL instruments [Health Utilities Index Mark 3 (HUI3), RAND 36-Item Short-Form Health Survey (SF-36), Skindex-29, visual analogue scale for itch (VAS itch) and 5-D pruritus scale]. The mean scores of MF/SS patients were compared to that of controls using multivariable regression models adjusted for demographics and medical comorbidities. Cluster analysis of the QoL instruments and clinical severity measures (e.g. stage and body-surface-area involvement) was performed. Patients with MF/SS scored significantly worse than controls on all QoL instruments used, with advanced-stage (IIB-IVB) disease having the worst QoL impairment. Early-stage (IA-IIA) and advanced-stage MF/SS patients had significantly reduced overall health status (HUI3; P<0.05), with largest decrements in social functioning and usual role functioning due to physical and emotional health (SF-36; all P<0.05). MF/SS had significantly worse skin-specific impairment than controls, with advanced-stage disease reporting the most severe skin-specific burden (Skindex-29, P<0.05). Clinical severity measures had a weak correlation with generic (|rs |=0.02-0.27) and moderate correlation with dermatology-specific instruments (|rs |=0.41-0.53). MF/SS have a significant impact on multiple domains of patients' QoL, including social, emotional and physical functioning. Current clinical measures do not adequately address QoL outcomes, underscoring the need for integrating formal disease-specific QoL assessment into the routine evaluation of MF/SS patients.

Japanese health utilities index mark 3 (HUI3): measurement properties in a community sample

BackgroundThe McMaster Health Utilities Index Mark 3 (HUI3) is a generic multi-attribute, preference-based system for assessing health-related quality of life (HRQOL). This study describes the translation procedures and cultural adaptation of the Japanese HUI3 and its measurement properties in a community sample.MethodsThe Japanese HUI3 was developed through forward and back translations in cooperation with the developers of the HUI. Acceptability, comprehensibility of questionnaires, and test-retest reliability were assessed. In a community survey of a total of 3860 people (age: 41 ± 14.3, male/female: 2651/1209), the Canadian scoring function was used to calculate utility scores. Construct validity was assessed by examining the relationship between 20 personal characteristics and utility scores.ResultsLinear regression estimates demonstrated a significant negative relation between HUI3 utility score and low education, male gender, poor interpersonal relationships, older age, and a higher number of chronic diseases. Single-attribute utility scores were associated with chronic conditions in the manner expected. The community samples were relatively healthy. More than 90% of the respondents were distributed in levels 1 and 2 in all attributes except cognition. Interpretability of utility score was assessed by estimation of the relationship between visual analogue scale (VAS) and the self-rated health and utility score. Independence of attributes was assessed. For only 3 of the 28 possible cross-comparisons among the 8 attributes were correlations coefficients greater than 0.25.ConclusionTranslation and adaptation of the HUI3 questionnaire into Japanese was successful, but the sample size and selection bias limit the interpretation of our study conclusions.

Socioeconomic disparities in life and health expectancy among the household population in Canada.

Life expectancy (LE) and health expectancy have increased throughout much of the world. However, these gains have not been shared equally across all population groups. Socioeconomic disparities exist, though varied methodologies and data sources have made it difficult to ascertain changes over time in Canada. The 1996 and 2011 Canadian Census Health and Environment Cohorts, with a five-year mortality follow-up, were used to estimate the LE of the household population at ages 25 and 65, according to individual-level education and income. Health status was measured by the Health Utilities Index Mark 3 instrument in two national population health surveys and was used to adjust LE to estimate health-adjusted life expectancy (HALE). Disparities in LE and HALE, and differences between cohorts, were examined. LE, HALE and the ratio of HALE to LE were greater at higher levels of education or income. A stepwise gradient was also observed by level of education within and across income quintiles, with people in the lowest combined education and income categories at the greatest disadvantage. Disparities were wider in the 2011 cohort compared with the 1996 cohort, but not necessarily to the same extent for both sexes or at different ages. In Canada, education-related and income-related disparities in life and health expectancy persist and may be wider than they were in the past. This underscores the importance of ongoing data development for routine monitoring of trends in mortality and morbidity, which can, in turn, inform policy development and planning to advance health equity.

The effect of ventilation tube insertion on quality of life in children with persistent otitis media with effusion.

To determine the effect of ventilation tube (VT) surgery on quality of life (QoL) in children with persistent otitis media with effusion (OME). Secondary analysis of trial data (oral steroids versus placebo for persistent OME), comparing QoL by history of VT surgery performed between 5weeks and 12months post-randomisation. Multilevel regression models were used to identify the association between VT surgery and QoL scores at 12months, controlling for pre-exposure risk factors associated with surgery, including pre-surgery hearing level. Ear, nose and throat (ENT), paediatric audiology and audiovestibular medicine (AVM) departments in Wales and England. A total of 327 children aged 2-8years with OME symptoms for at least three months and audiometry-proven bilateral hearing loss with VT surgery status. Otitis Media questionnaire (OM8-30) and Paediatric Quality of Life Inventory (PedsQL) total and subscale scores, and the Health Utilities Index Mark 3 (HUI3) at 12months post-randomisation. Participants who had VT surgery had no significant difference in OM8-30, PedsQL or HUI total scores. OM8-30 hearing difficulty (HD) subscale scores at 12months were better in those who had VT surgery (adjusted mean difference (aMD)=-0.46 (95% confidence interval: -0.69 to -0.23), P<.001), and this varied by when the surgery occurred (aMD for surgery between 5weeks and 6months=-0.4 [-0.67 to -0.13], P=.004 and between 6 and 12months=-0.54, [-0.87 to -0.22], P=.001). Ventilation tube surgery was associated with an improvement in HD-related functional health status but no change in overall QoL.

A vision 'bolt-on' increases the responsiveness of EQ-5D: preliminary evidence from a study of cataract surgery.

(1) To evaluate the effect of adding a vision dimension ('bolt-on') to the 5-level EQ-5D (EQ-5D-5L) and 3-level EQ-5D (EQ-5D-3L) on their responsiveness, and (2) to compare the responsiveness of a vision 'bolt-on' EQ-5D-3L (EQ-5D-3L + V) with SF-6D and Health Utilities Index Mark 3 (HUI3) to the benefit of cataract surgery. Sixty-three patients were assessed before and after their cataract surgery using the EQ-5D-3L, EQ-5D-5L, SF-6D, HUI3, as well as a 3-level and a 5-level vision dimension. Preference-based indices were calculated using available value sets for EQ-5D-3L, EQ-5D-3L + V, EQ-5D-5L, SF-6D, and HUI3, and non-preference-based indices were calculated using the sum-score method for EQ-5D-5L and EQ-5D-5L + V (vision bolt-on EQ-5D-5L). Responsiveness was assessed using the standardized response mean (SRM) and F-statistic. Among preference-based indices, mean changes from pre to post-surgery in EQ-5D-3L + V and EQ-5D-3L indices were 0.031 and 0.018, respectively. The mean changes for EQ-5D-5L, SF-6D and HUI3 indices were 0.020, 0.012 and 0.105, respectively. The SRM (F-statistic) for EQ-5D-3L + V and EQ-5D-3L indices were 0.458 (13.2) and 0.098 (0.6), respectively. The responsiveness of EQ-5D-3L + V was better than EQ-5D-5L, SF-6D; the responsiveness of HUI3 was better than all other measures. Using non-preference-based indices, mean change for EQ-5D-5L + V and EQ-5D-5L were 0.067 and 0.017, respectively. The corresponding SRM (F-statistic) were 0.709 (31.7) and 0.295 (5.4). Preliminary evidence from our study suggests that a vision 'bolt-on' may increase the responsiveness of EQ-5D-3L and EQ-5D-5L to change in health outcomes experienced by patients undergoing cataract surgery. In absence of the preference-based vision bolt-on EQ-5D-5L index, HUI3 was the most responsive measure.