Patient-reported outcomes in chronic hepatitis delta: An exploratory analysis of the Phase III MYR301 trial of bulevirtide.

BACKGROUND & AIMS: Once-daily treatment of chronic hepatitis delta (CHD) with bulevirtide is well tolerated and associated with significant reductions in HDV RNA in the blood and in biochemical liver disease activity. This study explored the effects of 48-week bulevirtide treatment on health-related quality of life (HRQoL) in patients with CHD. METHODS: In an open-label, randomised, Phase 3 trial, 150 patients with CHD and compensated liver disease were stratified by liver cirrhosis status and randomised 1:1:1 to no treatment (control), bulevirtide 2 mg/day, or bulevirtide 10 mg/day for 48 weeks. HRQoL was evaluated by the following patient-reported outcome (PRO) instruments at baseline, 24 weeks, and 48 weeks: EQ-5D-3L, Hepatitis Quality of Life Questionnaire (HQLQ), and Fatigue Severity Scale (FSS). RESULTS: Patient characteristics and HRQoL scores were balanced at baseline between the treatment (2 mg, n = 49; 10 mg, n = 50) and control (n = 51) groups. Patients receiving 2-mg bulevirtide reported significant improvements compared with controls on the HQLQ domains of role physical, hepatitis-specific limitations, and hepatitis-specific health distress. Numerically higher scores for general health, hepatitis-specific limitations, and hepatitis-specific health distress domains were reported by patients with cirrhosis who received bulevirtide vs control. FSS scores remained stable across treatment groups throughout. At week 48, patients in the 2-mg group showed greater mean improvement from baseline in health status compared with controls on the EQ-5D-3L visual analogue scale. CONCLUSION: PROs indicate that 48-week treatment with bulevirtide monotherapy may improve aspects of HRQoL in patients with CHD. IMPACT AND IMPLICATIONS: Bulevirtide 2 mg is the only approved treatment for patients with chronic hepatitis delta (CHD) in the EU. Patients with CHD have worse quality of life scores than those with chronic hepatitis B. Bulevirtide treatment for 48 weeks reduced HDV RNA and alanine aminotransferase levels and was well tolerated among patients with CHD. For the first time, this study shows that patients who received bulevirtide therapy for 48 weeks reported improvements in physical and hepatitis-related quality of life domains compared to those who did not receive therapy (control group). CLINICAL TRIAL REGISTRATION: ClinicalTrials.gov Identifier, NCT03852719.

Impact of multiple sclerosis comorbidities on quality of life and job activity.

BACKGROUND: Multiple sclerosis (MS) is frequently accompanied by comorbid conditions. OBJECTIVES: This study aimed to determine the prevalence of key comorbid conditions in people with multiple sclerosis (pwMS) and assess their impact on quality of life and work-related activities. METHODS: A cross-sectional study involving 755 pwMS from two third-level Italian MS centers was conducted. Comorbidities were identified from medical records, and quality of life was assessed using the EQ-5D-3L questionnaire. Work-related challenges were evaluated using the Multiple Sclerosis Questionnaire for Job Difficulties (MSQ-Job). RESULTS: 53.8% of pwMS had at least one comorbidity. Hypertension, depression, and anxiety were the most prevalent. Comorbidity presence was associated with reduced quality of life scores in almost all EQ-5D-3L domains and greater job difficulties in all but one MSQ-Job domain. CONCLUSION: Comorbidities in pwMS are prevalent and have a profound influence on quality of life and work-related activities. This comprehensive study offers new insights into the role of comorbidities in MS within the Italian context, emphasizing the need for a multidisciplinary approach in MS management. Further research is crucial to deepen our understanding of these findings in the broader Italian MS community.

Health inequity assessment in Brazil: is EQ-5D-3L sensible enough to detect differences among distinct socioeconomic groups?

BACKGROUND: Multidimensional health-related quality of life (HRQOL) instruments, such as the EQ-5D, are increasingly used to assess inequalities in health. However, it is necessary to explore the ability of these instruments to capture differences between population groups, especially in low/middle-income countries. This study aimed to investigate whether the EQ-5D-3L instrument can detect differences in HRQOL between groups of different socioeconomic status (SES) in Brazil. METHODS: Data collection occurred during the Brazilian EQ-5D-3L valuation study and included respondents aged 18 to 64 years enrolled in urban areas. SES was aggregated into three categories: "higher" (A and B), "intermediate" (C) and "lower" (D and E). EQ-5D-3L index was calculated considering the Brazilian value set. A mixed-effects regression model was estimated with random effects on individuals and marginal effects on SES, sex, and educational attainment. Odds ratios for the chance of reporting problems for each EQ-5D dimension were estimated by logistic regression. RESULTS: A total of 9,148 respondents were included in the study. Mean age was 37.80 ± 13.13 years, 47.4% were men and the majority was ranked as classes B or C (38.4% and 50.7%, respectively). Participants in lower SES classes reported increasingly poorer health compared to individuals in higher classes. The mean EQ-5D-3L index decreased as SES deteriorates being significantly higher for classes A and B (0.874 ± 0.14) compared to class C (0.842 ± 0.15) and classes D and E (0.804 ± 0.17) (p < 0.001). The same was observed for the mean EQ-VAS scores (84.0 ± 13.8 in classes A and B, 81.0 ± 17 in class C and 78.3 ± 18.7 in class C [p < 0.001]). The multivariate analysis confirmed that SES is an independent factor that effects EQ-5D-3L index measures. Participants in intermediate and lower SES classes have a statistically significant lower EQ-5D-3L index compared to participants in classes A and B, regardless of age, sex, and educational attainment. CONCLUSION: In a Brazilian population sample, the EQ-5D-3L instrument was able to detect important differences between groups with distinct socioeconomic statuses (SES). The EQ-5D-3L is useful for exploring inequities in health.

Comparison of the measurement properties and consistency between the EQ-5D-3L and EQ-5D-Y-3L in adolescents aged 15-17 in China.

OBJECTIVE: To evaluate and compare the measurement properties and consistency between the Chinese versions of EQ-5D-3L and EQ-5D-Y-3L among Chinese adolescent populations aged 15-17 years. METHODS: Chinese adolescents aged 15-17 studying in high school were recruited through online survey. Social-demographic characteristics and self-reported EQ-5D-3L and EQ-5D-Y-3L responses were collected in the survey. The consistency of responses between the two measures was assessed using redistribution property, and the consistency of utility values was assessed by intraclass correlation coefficients (ICC). Convergent validity and known-group validity were examined using Spearman's rank correlation, F-test and effect sizes, respectively. Sensitivity was compared using relative efficiency (RE). RESULTS: 762 respondents (48.8% male; age 15-17 years;) were recruited. The EQ-5D-3L showed a more severe ceiling effect than EQ-5D-Y-3L (78.2% vs. 66.0%). Respondents reported higher proportions of having problems in four dimensions using the EQ-5D-Y-3L than using the EQ-5D-3L. The consistency of corresponding dimensions between the two measures was relatively good, while non-negligible proportions of inconsistency were observed in "pain/discomfort" (11.4%) and "anxiety/depression" (15.7%) dimensions. The ICC of the utility values between the EQ-5D-3L and EQ-5D-Y-3L was 0.852 (p < 0.001). The Spearman's rank correlation (range: 0.385-0.620) indicated an acceptable convergent validity between the correlative dimensions of the EQ-5D-3L and EQ-5D-Y-3L. The EQ-5D-Y-3L had a higher efficiency than the EQ-5D-3L at detecting differences across EQ VAS subgroups (ES = 1.793 for EQ-5D-3L, ES = 1.920 for EQ-5D-Y-3L). Mixed results were observed in sensitivity. CONCLUSIONS: Both the EQ-5D-3L and EQ-5D-Y-3L are demonstrated to be valid and generally consistent for measuring HRQoL among adolescents aged 15-17 years in China. Respondents reported higher proportions of having problems using the EQ-5D-Y-3L than using the EQ-5D-3L. More research is warranted to compare the discriminant validity and test-retest reliability between the two measures.

Long-term quality of life and influencing factors in elderly patients after vertebroplasty based on the EQ-5D-3L Chinese utility scoring system: a retrospective study.

BACKGROUND: The quality of life of elderly patients after vertebroplasty is influenced by various factors. Although the EuroQol 5-Dimension 3-Level (EQ-5D-3L) scale has been widely used to assess quality of life, the factors affecting the long-term postoperative quality of life of elderly vertebroplasty patients in China have not been thoroughly studied. METHODS: This retrospective study included 519 patients aged 65 years and older who underwent elective vertebroplasty. We collected baseline data from these patients and conducted telephone follow-ups 12 months postoperation to evaluate their EQ-5D-3L health utility scores and EuroQol Visual Analogue Scale (EQ-VAS) scores. Univariate and multivariate linear regression models were used to analyse the factors affecting quality of life. RESULTS: Of the 519 patients, the majority were female (78.0%), aged 65 to 95 years, with an average age of 75.2 years. Twelve months postoperation, pain/discomfort was the most commonly reported issue for 68.4% of patients. The median EQ-5D-3L health utility score was 0.783, with a range between 0.450 and 0.887; the median EQ-VAS score was 75, ranging from 60 to 85. Multivariate linear regression analysis indicated that older age, hormone use, higher American Society of Anesthesiologists (ASA) grades, nondrinking habits, and low albumin levels were found to be independent risk factors affecting long-term quality of life in elderly patients after vertebroplasty. Additionally, a history of tumours, the number of vertebral compression fractures, and bone mineral density were also crucial influencing factors. CONCLUSIONS: Based on the use of the EQ-5D-3L Chinese utility scoring system, we evaluated the quality of life of patients aged 65 and above 12 months after vertebroplasty. This study identified several factors related to postoperative quality of life in elderly vertebroplasty patients, providing crucial evidence for further clinical decisions and patient education.

Development of a disease-specific health utility score for chronic obstructive pulmonary disease from a discrete choice experiment patient preference study.

OBJECTIVES: While patient input to health technology assessment (HTA) has traditionally been of a qualitative nature, there is increasing interest to integrate quantitative evidence from patient preference studies into HTA decision making. Preference data can be used to generate disease-specific health utility data. We generated a health utility score for patients with chronic obstructive pulmonary disease (COPD) and consider its use within HTAs. METHODS: Based on qualitative research, six symptoms were identified as important to COPD patients: shortness of breath, exacerbations, chronic cough, mucus secretion, sleep disturbance, and urinary incontinence. We employed a discrete choice experiment (DCE) and the random parameter logistic regression technique to estimate utility scores for all COPD health states. The relationship between patients' COPD health utility scores, self-perceived COPD severity, and EQ-5D-3L utility scores was analyzed, with data stratified according to disease severity and comorbidity subgroups. RESULTS: The COPD health utility score had face validity, with utility scores negatively correlated with patients' self-perceived COPD severity. The correlation between the COPD health utility scores and EQ-5D-3L values was only moderate. While patient EQ-5D-3L scores were impacted by comorbidities, the COPD health utility score was less impacted by comorbid conditions. CONCLUSIONS: Our COPD utility measure, derived from a DCE, provides a patient-centered health utility score and is more sensitive to the COPD health of the individual and less sensitive to other comorbidities. This disease-specific instrument should be considered alongside generic health-related quality of life instruments when valuing new COPD therapies in submissions to licensing and reimbursement agencies.

Health-Related Quality of Life in Patients with Spinocerebellar Ataxia: a Validation Study of the EQ-5D-3L.

Although health-related quality of life (HRQoL) has developed into a crucial outcome parameter in clinical research, evidence of the EQ-5D-3L validation performance is lacking in patients with spinocerebellar ataxia (SCA) types 1, 2, 3, and 6. The objective of this study is to assess the acceptability, validity, reliability, and responsiveness of the EQ-5D-3L. For n = 842 predominantly European SCA patients of two longitudinal cohort studies, the EQ-5D-3L, PHQ-9 (Patient Health Questionnaire), and ataxia-specific clinical assessments (SARA: Scale for Assessment and Rating of Ataxia; ADL: activities of daily living as part of Friedreich's Ataxia Rating Scale; INAS: Inventory of Non-Ataxia Signs) were assessed at baseline and multiple annual follow-ups. The EQ-5D-3L was evaluated regarding acceptability, distribution properties, convergent and known-groups validity, test-retest reliability, and effect size measures to analyze health changes. The non-item response was low (EQ-5D-3L index: 0.8%; EQ-VAS: 3.4%). Ceiling effects occurred in 9.9% (EQ-5D-3L) and 3.0% (EQ-VAS) with a mean EQ-5D-3L index of 0.65 ± 0.21. In total, convergent validity showed moderate to strong Spearman's rho (rs > 0.3) coefficients comparing EQ-5D-3L and EQ-VAS with PHQ-9, SARA, ADL, and INAS. EQ-5D-3L could discriminate between groups of age, SARA, ADL, and INAS. Intra-class correlation coefficients (EQ-5D-3LICC: 0.95/EQ-VASICC: 0.88) and Kappa statistics (range 0.44 to 0.93 for EQ-5D-3L items) indicated tolerable reliability. EQ-5D-3L shows small (effect size < 0.3) to moderate (effect size 0.3-0.59) health changes regarding ataxia severity. The analysis confirms an acceptable, reliable, valid, and responsive recommended EQ-5D-3L in SCA patients, measuring the HRQoL adequately, besides well-established clinical instruments.

In search of a 'pan-European value set'; application for EQ-5D-3L.

OBJECTIVES: Country-specific value sets for the EQ-5D are available which reflect preferences for health states elicited from the general population. This allows the transformation of responses on EQ-5D to health state utility values. Only twelve European countries possess country-specific value sets and no value set reflecting the preferences of Europe exists. We aim to estimate a 'pan-European' value set for the EQ-5D-3L, reflecting the preferences for health states of the European population that could help to evaluate health care from the perspective of the European decision-maker. METHODS: We systematically assessed and compared the methodologies of available EQ-5D-3L time trade-off (TTO) value sets from twelve European countries: Denmark, France, Germany, Hungary, Italy, Netherlands, Poland, Portugal, Romania, Slovenia, Spain and UK. Using their published coefficients, a dataset with utility values for all 243 health states was simulated. Different modelling techniques and model specifications including interaction terms were tested. Model selection was based on goodness-of-fit criteria. We also explored results with application of population size weights. RESULTS: Methodological, procedural and analytical characteristics of the included EQ-5D-3L valuation studies were quite comparable. An OLS based model was the preferred model to represent European preferences. Weighting with population size made little difference. CONCLUSIONS: EQ-5D-3L valuation studies were considered of sufficient comparability to form the basis for a new 'pan-European' value set. The method used allows for an easy update when new national value sets become available.

Scale agreement, ceiling and floor effects, construct validity, and relative efficiency of the PROPr and EQ-5D-3L in low back pain patients.

BACKGROUND: The PROMIS Preference score (PROPr) is a new health state utility (HSU) score that aims to comprehensively incorporate the biopsychosocial model of health and apply favorable psychometric properties from the descriptive PROMIS system to HSU measurements. However, minimal evidence concerning comparisons to the EQ-5D-3L and the PROPr's capability to differentiate clinical severity are available. Therefore, the aim of this study was to compare the PROPr to the EQ-5D-3L in terms of scale agreement, ceiling/floor effects, distribution, construct validity, discriminatory power, and relative efficiency (RE) in terms of the Oswestry Disability Index (ODI) for patients with low back pain (LBP). METHODS: We used intra-class correlation coefficients (ICC) and Bland-Altman plots to compare the PROPr and EQ-5D-3L with regared to scale agreement in a cross-sectional routine sample of LBP patients. For distribution, we used the Pearson's coefficient for skewness and for ceiling/floor effects, a 15%-top/bottom threshold. For convergent validity, we used Pearson's correlation coefficients. For known-groups validity, we applied a linear regression with interaction terms (predictors sex, age, and ODI level) and an analysis of variance (ANOVA). For discriminatory power, we calculated the effect size (ES) using Cohen's d and the ratio of the area under the receiver-operating characteristics curves (AUROC-ratio = AUROCPROPr/AUROCEQ-5D-3L). RE was measured using the ratio of F-values (RE = FPROPr/FEQ-5D-3L). RESULTS: Of 218 LBP patients, 50.0% were female and the mean age was 61.8 years. The mean PROPr (0.20, 95%CI: 0.18; 0.22) and EQ-5D-3L scores (0.55, 95%CI: 0.51; 0.58) showed low agreement (d = 0.35, p < 0.001; ICC 0.27, 95%CI: -0.09; 0.59). The PROPr's distribution was positively skewed, whereas the EQ-5D-3L's was negative. Neither tool showed ceiling/floor effects, but all EQ-5D-3L dimensions did. Pearson correlation was r = 0.66 (95%CI: 0.58; 0.73). Differences were invariant to sex and age but not to ODI severity: ESEQ-5D-3L > ESPROPr and RE < 1 in higher ODI severity; ESEQ-5D-3L < ESPROPr and RE > 1 in lower ODI severity. AUROC-ratios did not show significant differences in terms of ODI severity. CONCLUSIONS: All PROPr and EQ-5D-3L biopsychosocial dimensions of health showed impairment in LPB patients. The capability of EQ-5D-3L and PROPr to differentiate ODI levels depends on ODI severity. Joint application of both tools may provide additional information.

Population norms for the EQ-5D-3L and EQ-5D-5L in Romania.

BACKGROUND: The majority of patient reported outcome measures (PROMs) don't have population norms in Romania. This is the case with the EQ-5D as well. Therefore, we aimed to estimate population norms for the Romanian versions of the EQ-5D-5L, EQ-5D-3L, their indexes, and the EQ-VAS. METHODS: A cross-sectional survey was conducted in all regions of Romania from November 2018 to November 2019. A three-stage probability sampling procedure stratified by region and settlement size was used to select a representative sample. Interviews were computer-assisted and conducted in respondents' homes by trained interviewers. Health status was assessed with the EQ-5D-5L, the EQ-5D-3L and the EQ VAS. Descriptive statistics were used to estimate population norms by age groups and sex for the EQ-5D-5L, the EQ-5D-3L, their indexes and the EQ VAS. Population norms were weighted using survey weights. Indexes for the EQ-5D questionnaires were estimated using the recently developed Romanian value sets. RESULTS: Data from 1,649 interviews was analysed in the present study. Survey weights were used so that sex and place of residence ratios for the weighted sample matched the Romanian general population distribution. Participants' mean age was 47.4 years (SE = 1.157) and 50.3% of them reported being in good health. The dimension for which people reported the highest number of problems for both questionnaires was the pain/discomfort dimension. Men aged 35 plus reported fewer problems with pain/discomfort than women for both the EQ-5D-5L and EQ-5D-3L. Health decreased with age as shown by the decrease from age group 18-24 to age group 75 plus in the indexes of both questionnaires: from 0.977 (SE = 0.005) to 0.765 (SE = 0.017) for EQ-5D-5L and from 0.981 (SE = 0.005) to 0.784 (SE = 0.019) for EQ-5D-3L. There was 29.9 points drop in the EQ VAS score between the youngest and oldest group. CONCLUSIONS: Population norms for the Romanian versions of the EQ-5D-5L, EQ-5D-3L, their indexes, and the EQ VAS are now available. These can now be used as reference values by healthcare professionals, researchers and decision-makers leading to a further development of health-related quality of life research in Romania.

Healthcare utilization and health-related quality of life of severe asthma patients in Singapore.

BACKGROUND: Notwithstanding unequivocal consensus on the disproportionate effect of severe asthma (SA) on asthma morbidity, healthcare utilization, quality of life, work impairment and socioeconomic burden, the burden of SA patients in Singapore has not been appraised. OBJECTIVES: To determine the burden of disease and extent of quality of life impairment in SA patients in Singapore. METHODS: A cross-sectional analysis of SA patients seen in Singapore General Hospital (2020-2021) to investigate emergency healthcare utilization, oral corticosteroid (OCS) burden and health-related quality of life (HRQoL) with primary endpoint EuroQoL-5 Dimension three-level (EQ-5D-3L) scores. The empirical measurement properties of the EQ-5D utility index in SA were comprehensively assessed through multivariate regression analyses. RESULTS: A total of 336 SA patients were recruited, 51.2% of SA patients had at least one acute healthcare resource utilization during the previous year, with 25.6% of patients having an emergency healthcare visit to the hospital. Overall mean (SD) EQ-5D-3L and EQ-5D-3L utility scores in SA patients were 6.22 (1.51) and 0.77 (0.30), respectively. EQ-5D utility scores were 0.14 lower in uncontrolled vs controlled asthma and 0.09 lower in the presence of severe exacerbation, whereas barely changed by maintenance OCS dose and airflow limitation. CONCLUSION: SA patients were found to have high disease burden, high healthcare resource utilization and OCS use, low biologics usage, poor HRQoL and utility in comparison with other chronic diseases.

A head-to-head comparison of the measurement properties of EQ-5D-3L and EQ-5D-5L in Chinese family caregivers of cancer patients.

BACKGROUND: Although both EQ-5D-3L(3L) and EQ-5D-5L(5L) have demonstrated good measurement properties in several patient populations, there is currently limited evidence comparing the measurement properties of 3L and 5L in family caregivers (FCs) of cancer patients. PURPOSE: This study aimed to compare the measurement properties of 3L and 5L in a sample of family caregivers of cancer patients. METHODS: A consecutive sample of FCs of cancer patients recruited from three tertiary hospitals were invited to complete the two versions of the EQ-5D in two rounds of interviews. We compared i) the ceiling effect using the McNemar's test, ii) test-retest reliability using intraclass correlation coefficient (ICC) and Cohen's Kappa, iii) convergent validity using Spearman's rank correlation coefficient, iv) known-group validity using F-statistic, v) and discriminant capacity using ordinal logistic regression. RESULTS: A total of 416 FCs completed the baseline questionnaire and 120 caregivers completed the follow-up questionnaire. Ceiling effects were smaller in 5L (12.5%) than in 3L (20.7%). The convergent validity (r = 0.344-0.771), known-groups validity (Fratio5L/3L = 2.06-4.09), discriminant capacity (ES = 0.341-0.396), and test-retest reliability (ICC = 0.725) of the 5L were slightly better than those of the 3L in China. CONCLUSION: The current study found both 3L and 5L to be suitable for use by FCs of cancer patients. However, 5L showed superior measurement properties compared to 3L and therefore could be the preferred instrument when EQ-5D data of cancer patients FCs is required.

Health-related quality of life among people who inject drugs in Australia.

PURPOSE: There is limited research on health-related quality of life (HRQoL) among people who inject drugs (PWID). We aimed to evaluate factors associated with HRQoL among a cohort of PWID in Australia. METHODS: Participants were enrolled in an observational cohort study (the LiveRLife Study) between 2014 and 2018 at 15 sites in Australia. They provided fingerstick whole-blood samples for point-of-care HCV RNA testing and underwent transient elastography to assess liver disease. Participants completed the EQ-5D-3L survey at enrolment. Regression models were used to assess the impact of clinical and socioeconomic characteristics on the EQ-5D-3L scores. RESULTS: Among 751 participants (median age, 43 years; 67% male), 63% reported injection drug use in the past month, 43% had current HCV infection, and 68% had no/mild liver fibrosis (F0/F1). The mean EQ-5D-3L and EQ-VAS scores were 0.67 and 62, respectively, for the overall study population. There was no significant difference in the EQ-5D-3L scores among people with and without recent injecting drug use (mean: 0.66 vs. 0.68, median: 0.73 vs. 0.78, P = 0.405), and among people receiving and not receiving opioid agonist therapy (mean: 0.66 vs. 0.68, median: 0.73 vs. 0.76, P = 0.215). Participants who were employed were found to have the highest mean EQ-5D-3L (0.83) and EQ-VAS scores (77). The presence of current HCV infection, liver fibrosis stage, and high-risk alcohol consumption had little impact on HRQoL. CONCLUSIONS: The study findings provide important HRQoL data for economic evaluations, useful for guiding the allocation of resources for HCV elimination strategies and interventions among PWID.

Quality of Life since Pre-Ataxic Phases of Spinocerebellar Ataxia Type 3/Machado-Joseph Disease.

Although health-related quality of life (HRQoL) has been increasingly valued in healthcare and in clinical trials, there is scarce information about it in spinocerebellar ataxia type 3/Machado-Joseph disease (SCA3/MJD). This study describes the HRQoL results obtained from ataxic SCA3/MJD subjects, and their non-ataxic offspring included in the BIGPRO (Biomarkers and genetic modifiers in a study of presymptomatic and symptomatic SCA3/MJD carriers) study. Demographic data, clinical scales, and HRQoL instruments EQ-5D-3L and SF-36 were collected. Subjects at 50% risk were genotyped in a double-blind manner. The time left until the onset of the disease was estimated for mutation carriers with a SARA < 3 and combined with disease duration of ataxic subjects (TimeToAfterOnset). Analyses were performed using PASW Statistics version 18.0, R version 4.0.0, and G*Power 3.1, and p < 0.05 was considered statistically significant. Twenty-three ataxic carriers, 33 pre-ataxic carriers, and 21 controls were enrolled. Significant differences between ataxic carriers and controls were seen in EQ-VAS, EQ-5D Index, and in some domains of EQ-5D-3L and SF-36. EQ-5D Index showed the best effect size between ataxic and controls (Cohen's d = 2.423). Stepwise changes were seen in pre-ataxic subjects, although not statistically significant. TimeToAfterOnset correlated with EQ-5D Index, EQ-VAS, and SF-36 Physical functioning, Role Physical, Pain, and General Health. EQ-5D Index and EQ-VAS correlated with clinical scales in the ataxic group. These results suggest that HRQoL worsens among carriers since pre-ataxic stages and that they might encompass the underlying disease process. In this cohort, SF-36 Physical Functioning, SF-36 General health, and especially EQ-5D Index and EQ-VAS were the best HRQoL instruments to be used as ancillary evidence to support biological and social meanings for future interventions.

Predicting Patient-Level 3-Level Version of EQ-5D Index Scores From a Large International Database Using Machine Learning and Regression Methods.

OBJECTIVES: This study aimed to evaluate the performance of machine learning and regression methods in the prediction of 3-level version of EQ-5D (EQ-5D-3L) index scores from a large diverse data set. METHODS: A total of 30 studies from 3 countries were combined. Predictions were performed via eXtreme Gradient Boosting classification (XGBC), eXtreme Gradient Boosting regression (XGBR) and ordinary least squares (OLS) regression using 10-fold cross-validation and 80%/20% partition for training and testing. We evaluated 6 prediction scenarios using 3 samples (general population, patients, total) and 2 predictor sets: demographic and disease-related variables with/without patient-reported outcomes. Model performance was evaluated by mean absolute error and percent of predictions within clinically irrelevant error range and within correct health severity group (EQ-5D-3L index <0.45, 0.45-0.926, >0.926). RESULTS: The data set involved 26 318 individuals (clinical settings n = 6214, general population n = 20 104) and 26 predictor variables plus diagnoses. Using all predictors and the total sample, mean absolute error values were 0.153, 0.126, and 0.131, percent of predictions within clinically irrelevant error range were 47.6%, 39.5%, and 37.4%, and within the correct health severity group were 56.3%, 64.9%, and 63.3% by XGBC, XGBR, and OLS, respectively. The performance of models depended on the applied evaluation criteria, the target population, the included predictors, and the EQ-5D-3L index score range. CONCLUSIONS: Regression models (XGBR and OLS) outperformed XGBC, yet prediction errors were outside the clinically irrelevant error range for most respondents. Our results highlight the importance of systematic patient-reported outcome (EQ-5D) data collection. Dialogs between artificial intelligence and outcomes research experts are encouraged to enhance the value of accumulating data in health systems.

A head-to-head comparison of the EQ-5D-3L index scores derived from the two EQ-5D-3L value sets for China.

OBJECTIVE: Two EQ-5D-3L (3L) value sets (developed in 2014 and 2018) co-exist in China. The study examined the level of agreement between index scores for all the 243 health states derived from them at both absolute and relative levels and compared the responsiveness of the two indices. METHODS: Intraclass correlations coefficient (ICC) and Bland-Altman plot were adopted to assess the degree of agreement between the two indices at the absolute level. Health gains for 29,403 possible transitions between pairs of 3L health states were calculated to assess the agreement at the relative level. Their responsiveness for the transitions was assessed using Cohen effect size. RESULTS: The mean (SD) value was 0.427 (0.206) and 0.649 (0.189) for the 3L2014 and 3L2018 index scores, respectively. Although the ICC value showed good agreement (i.e., 0.896), 88.9% (216/243) of the points were beyond the minimum important difference limit according to the Bland-Altman plot. The mean health gains for the 29,403 health transitions was 0.234 (3L2014 index score) and 0.216 (3L2018 index score). The two indices predicted consistent transitions in 23,720 (80.7%) of 29,403 pairs. For the consistent pairs, Cohen effective size value was 1.05 (3L2014 index score) or 1.06 (3L2018 index score); and the 3L2014 index score only yielded 0.007 more utility gains. However, the results based on the two measures varied substantially according to the direction and magnitude of health change. CONCLUSION: The 3L2014 and 3L2018 index scores are not interchangeable. The choice between them is likely to influence QALYs estimations.

Obtaining EQ-5D-3L utility index from the health status scale of traditional Chinese medicine (TCM-HSS) based on a mapping study.

BACKGROUND: Almost all traditional Chinese medicine (TCM) quality of life measures are non-preference-based measures (non-PBMs), which do not provide utilities for cost-utility analysis in pharmacoeconomic evaluation. Whereas the mapping has become a new instrument to obtain utilities, which builds a bridge between non-PBMs and PBMs. PURPOSE: To develop mapping algorithms from the health status scale of traditional Chinese medicine (TCM-HSS) onto the three-level EuroQol five-dimensional questionnaire (EQ-5D-3L). METHODS: The cross-sectional data were collected by questionnaire survey from a tertiary hospital visit population and community residents in China, and randomly divided into training and validation set by 2:1. Based on the training set, direct and indirect mapping methods (7 regression methods and 4 model specifications) were conducted to establish alternative models, which were comprehensively evaluated based on the validation set by mean absolute error, root mean square error, and Spearman correlation coefficient between predicted and observed values. Based on the whole sample, the preferred mapping algorithm was developed. RESULTS: A total of 639 samples were included, with an average age of 45.24 years and 61.66% of respondents were female. The mean EQ-5D-3L index was 0.9225 [SD = 0.1458], and the mean TCM-HSS index was 3.4144 [SD = 3.1154]. The final mapping algorithm was a two-part regression model including the TCM-HSS subscales, interaction terms, and demographic covariates (age and gender). The prediction performance was good. The mean error was 0.0003, the mean absolute error was 0.0566, the root mean square error was 0.1039, and 83.10% of the prediction errors were within 0.1; the Spearman correlation coefficient between predicted and observed EQ-5D-3L values was 0.6479. CONCLUSION: It is the first study to develop a mapping algorithm between the TCM-HSS and EQ-5D-3L, which demonstrates excellent prediction accuracy and estimates utility value for economic evaluation from TCM quality of life measures.

Comparison of EQ-5D-3L and 5L versions following operative fixation of closed ankle fractures.

PURPOSE: To undertake the first testing and comparison of measurement properties for the EuroQol EQ-5D-3L and 5L in patients with ankle problems. METHODS: The cross-sectional postal survey of 959 patients aged ≥ 18 years, who underwent surgical treatment (ORIF) for unstable and closed ankle fractures in Eastern Norway. Both the EQ-5D-3L and 5L were included in a postal questionnaire in 2015, 3-6 years post surgery. Missing data, floor and ceiling effects, and response consistency were assessed. Tests of validity included comparisons with scores for the SF-36 and widely used ankle-specific instruments. The 5L version was assessed for test-retest reliability. RESULTS: There were 567 (59%) respondents; 501 completed both versions and 182 (61%) the 5L retest questionnaire. The 5L outperformed the 3L in tests of data quality and classification efficiency. Correlations with scores for other instruments largely met expectations, those for the 5L being slightly higher. All 5L scores had acceptable levels of reliability. For the 5L index, the smallest detectable differences for group and individual comparisons were 0.02 and 0.20, respectively. CONCLUSION: The 5L outperformed the 3L in terms of data quality, number of health states assessed and tests of validity. The 5L is recommended in research and other applications following surgery for ankle fracture but further testing including responsiveness to change is recommended at clinically relevant follow-up periods.

Predictors of EQ-5D-3L outcomes amongst injured Maori: 1-year post-injury findings from a New Zealand cohort study.

PURPOSE: Maori, the Indigenous population of New Zealand (NZ), are at higher risk of problems with health-related quality of life (HRQoL) 12 months following injury. This paper examines pre-injury sociodemographic and health characteristics and injury-related factors, including healthcare access, and their association with HRQoL outcomes 12 months after injury. METHODS: The Prospective Outcomes of Injury Study recruited 2856 injured New Zealanders aged 18-64 years from the entitlement claims register of the country's no-fault injury insurance agency. One-fifth (n = 566) of the cohort were Maori. Information on predictors and outcomes, with the exception of injury and hospitalisation, was obtained directly from participants at approximately 3 and 12 months post-injury. The outcomes of interest were responses to the five dimensions of the EQ-5D-3L and a dichotomous measure obtained by summing scored responses to each question. Modified Poisson regression was used to identify predictors of each outcome at 12 months post-injury. RESULTS: Predictors differed by outcome. Being female, experiencing EQ-5D-3L problems pre-injury, having ≥ 2 chronic conditions pre-injury, perceiving one's injury to be a threat of long-term disability, and having trouble accessing health services for injury were common predictors of EQ-5D-3L problems at 12 months post-injury for Maori. CONCLUSION: Opportunities exist to improve HRQoL outcomes by identifying individuals in the early stages of injury recovery who may benefit from further treatment and support.

Health-related quality of life among patients with gallstone disease: a systematic review and meta-analysis of EQ-5D utility scores.

PURPOSE: Gallstone disease or cholelithiasis is a chronic illness that usually presents with pain in the abdomen, vomiting and indigestion leading to impaired quality of life. EQ-5D utility score is a validated measure of health-related quality of life (HRQoL). We systematically reviewed the literature and synthesised EQ-5D utility scores among patients with gallstone disease and its improvement on treatment. METHODS: We have systematically searched observational studies reporting EQ-5D utility scores of gallstone disease in PubMed, Scopus, and Embase databases, from inception until February 2021. We selected the studies adhering to the PRISMA guidelines. The selected studies were reviewed, and the EQ-5D utility values of pre and post cholecystectomy were pooled using the random-effects model. RESULT: From identified 4,817 records of database search, eleven studies predominantly from western countries with 2,189 participants were included for systematic review and meta-analysis. The pooled EQ-5D and visual analogue scores were 0.87 (0.82 to 0.91, I2 = 93.73%) and 83.30 (60.59 to 106.12, I2 = 99.30%) respectively with high heterogeneity. The pooled EQ-5D and EQ-5D visual analogue scores post cholecystectomy treatment were 0.93 (0.91 to 0.95, I2 = 90.17%) and 91.7 (85.99 to 96.35, I2 = 97.93%) respectively. The mean difference between the baseline and post intervention were 0.05 (0.01 to 0.10, I2 = 93.50%) and 10.58 (-8.63 to 29.79, I2 = 98.32%) for EQ-5D and visual analogue scores respectively with high heterogeneity between the studies. CONCLUSION: The pooled mean difference indicates improvement in HRQoL after cholecystectomy but with high heterogeneity. Further high-quality studies from Asian countries are required for globally representative quantification and precise estimates of HRQoL among gallstone diseases. PROSPERO REGISTRATION ID: CRD42021234467.