Parkinson's disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial.
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SUBMITTER: Zahra M
PROVIDER: S-EPMC7053067 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
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Parkinson's disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial.
Zahra Mehdi M Durand-Zaleski Isabelle I Górecki Michal M Walleser Autiero Silke S Barnett Gillian G Schüpbach W M Michael WMM
Health and quality of life outcomes 20200302 1
<h4>Background</h4>A utility value is a health-related quality of life metric (HRQoL) metric used in a cost-effectiveness analysis. While utilities as outcomes in the treatment of advanced Parkinson's disease (PD) with deep brain stimulation (DBS) are available, they do not currently exist for PD with early motor complications. The objectives of this study were to predict utilities from observed disease-specific HRQoL data using two mapping algorithms, and investigate their performance in terms ...[more]
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Project description:PurposeThe most widely used generic questionnaire to estimate the quality of life for yielding quality-adjusted life years in economic evaluations is EQ-5D. Country-specific population value sets are required to use EQ-5D in economic evaluations. The aim of this study was to establish an EQ-5D-3L value set for Russia.MethodsA representative sample aged 18+ years was recruited from the Russia`s general population. Computer-assisted face-to-face interviews were conducted based on the standardized valuation protocol using EQ-Portable Valuation Technology. Population preferences were elicited utilizing both composite time trade-off (cTTO) and discrete choice experiment (DCE) techniques. To estimate the value set, a hybrid regression model combining cTTO and DCE data was used.ResultsA total of 300 respondents who successfully completed the interview were included in the primary analysis. 120 (40.0%) respondents reported no health problems of any dimension, and 56 (18.7%) reported moderate health problems in one dimension of the EQ-5D-3L. Median self-rated health using EQ-VAS was 80 with IQR 70-90. Comparing cTTO and DCE-predicted values for 243 health states resulted in a similar pattern. This supports the use of hybrid models. The predicted value based on the preferred model for the worst health state "33333" was -0.503. Mobility dimension had the most significant impact on the utility decrement, and anxiety/depression had the lowest decrement.ConclusionDetermining a Russian national value set may be considered the first step towards promoting cost-utility analysis use to increase comparability among studies and improve the transferability of healthcare decision-making in Russia.
| S-EPMC8233249 | biostudies-literature
Project description:ObjectivesTo obtain utility estimates suitable for use in economic models for chronic spontaneous (idiopathic) urticaria (CSU).MethodsPatient-level data from three randomized clinical trials-ASTERIA I, ASTERIA II and GLACIAL-were analysed. Health states were derived from the Urticaria Activity Score over 7 days (UAS7); higher scores denote greater activity. The health state score ranges were urticaria free: 0; well-controlled urticaria: 1-6; mild urticaria: 7-15; moderate urticaria: 16-27; and severe urticaria: 28-42. The mean EQ-5D utilities were calculated for each health state. A mixed model was used to predict the EQ-5D according to UAS7 health states in a pooled data set containing all treatment arms and time points from the three trials. Pooled trial data were validated through visual comparisons and interaction terms. Fixed and random effects for trials and patients were included, along with the following covariates: UAS7 health state at baseline (moderate or severe); presence of angioedema at baseline and during follow-up; duration of CSU; number of previous CSU medications; visit; current treatment; and patient age and sex.ResultsThere was a consistent improvement in EQ-5D utilities as urticaria activity decreased. The mean utilities ranged from 0.710 (severe urticaria) to 0.780 (moderate urticaria), 0.829 (mild urticaria), 0.862 (well-controlled urticaria) and 0.894 (urticaria free). Sensitivity and subgroup analyses confirmed the robustness of the results.ConclusionThe results suggest that EQ-5D utility scores increase with decreasing urticaria activity. EQ-5D utility scores enable the health-related quality of life of CSU patients to be compared with that of patients with other diseases.
| S-EPMC4828466 | biostudies-literature
Project description:Background The adult versions EQ-5D-3L and EQ-5D-5L have been extensive compared. This is not the case for the EQ-5D youth versions. The study aim was to compare the measurement properties and responsiveness of EQ-5D-Y-3L and EQ-5D-Y-5L in paediatric patients. Methods A sample of patients 8–16 years old with different diseases and a wide range of disease severity was asked to complete EQ-5D-Y-3L, EQ-5D-Y-5L, PedsQL Generic Core Scale, and selected, appropriate disease-specific instruments, three times. EQ-5D-Y-3L and EQ-5D-Y-5L were compared in terms of: feasibility, (re-)distribution properties, discriminatory power, convergent validity, test–retest reliability, and responsiveness. Results 286 participating patients suffered from one of the following diseases: major beta-thalassemia, haemophilia, acute lymphoblastic leukaemia, acute illness. Missing responses were comparable between versions of the EQ-5D-Y, suggesting comparable feasibility. The number of patients in the best health state (level profile 11111) was equal in both EQ-5D-Y versions. The projection of EQ-5D-Y-3L scores onto EQ-5D-Y-5L for all dimensions showed that the two additional levels in EQ-5D-Y-5L slightly improved the accuracy of patients in reporting their problems, especially if severe. Convergent validity with PedsQL and disease-specific measures showed that the two EQ-5D-Y versions performed about equally. Test–retest reliability (EQ-5D-Y-3L 0.78 vs EQ-5D-Y-5L 0.84), and sensitivity for detecting health changes, were both better in EQ-5D-Y-5L. Conclusions Extending the number of levels did not give clear superiority to EQ-5D-Y-5L over EQ-5D-Y-3L based on the criteria assessed in this study. However, increasing the number of levels benefitted EQ-5D-Y performance in the measurement of moderate to severe problems and especially in longitudinal study designs. Supplementary Information The online version contains supplementary material available at 10.1186/s12955-021-01889-4.
| S-EPMC8591892 | biostudies-literature
Project description:Background and purpose - To better detect small changes in postoperative outcome following total hip replacement (THR), the Swedish Hip Arthroplasty Register (SHAR) has decided to change from the EQ-5D-3L (3L) to the EQ-5D-5L (5L). To enable comparison of results obtained with use of the 2 versions of EQ-5D, transferal of results between the questionnaires used is necessary. We assessed the measurement properties of the EQ-5D-5L compared with the EQ-5D-3L, preoperatively and 1-year postoperatively in a Swedish THR population.Patients and methods - Patients eligible for elective THR during 2015 in Western Sweden were invited to the study. With a 2-week separation, the 3L and 5L questionnaires were administered to patients before and 1 year after surgery. Comparing the 2 versions of the EQ-5D, we investigated redistribution of responses, ceiling and floor effects, EQ VAS correlations (Spearman's rank correlation coefficient, rs), and EQ VAS scores for different severity levels by dimension (univariable ordinary least square regression).Results - The additional severity levels of the 5L version were frequently used on both measurement occasions (preoperative mobility 5%, self-care 17%, usual activities 20%, pain 5% and anxiety 3%, postoperative mobility 6%, self-care 5%, usual activities 8%, pain 9%, and anxiety 5%). Ceiling effects of the 3L version diminished overall by 7% using the 5L version. The correlations between the 2 EQ VAS scores obtained with the 3L and 5L instruments were strong both pre- (rs = 0.71) and postoperatively (rs = 0.87). Estimated EQ VAS scores for different levels of severity were consistent for all dimensions except for the mobility dimension of the preoperative 5L version and the anxiety dimension in the postoperative 5L version.Interpretation - Our findings support that the 5L has a higher resolution than the 3L version regarding description of health-related quality of life in patients undergoing THR in Sweden. The EQ VAS scores for different levels of severity agree well between the EQ-5D versions. This could potentially be used to develop a crosswalk value set for transforming 3L to 5L responses in this patient group.
| S-EPMC8023878 | biostudies-literature
Project description:IntroductionThe EQ-5D-Y-3L is a generic measure of health-related quality of life in children and adolescents. Although the Brazilian-Portuguese EQ-5D-Y-3L version is available, there is no value set for it, hampering its use in economic evaluations. This study aimed to elicit a Brazilian EQ-5D-Y-3L value set based on preferences of the general adult population.MethodsTwo independent samples of adults participated in an online discrete choice experiment (DCE) survey and a composite time trade-off (cTTO) face-to-face interview. The framing was "considering your views for a 10-year-old child". DCE data were analyzed using a mixed-logit model. The 243 DCE predicted values were mapped into the observed 28 cTTO values using linear and non-linear mapping approaches with and without intercept. Mapping approaches' performance was assessed to estimate the most valid method to rescale DCE predicted values using the model fit (R2), Akaike Information Criteria (AIC), root mean squared error (RMSE), and mean absolute error (MAE).ResultsA representative sample of 1376 Brazilian adults participated (DCE, 1152; cTTO, 211). The linear mapping without intercept (R2 = 96%; AIC, - 44; RMSE, 0.0803; MAE, - 0.0479) outperformed the non-linear without intercept (R2 = 98%; AIC, - 63; RMSE, 0.1385; MAE, - 0.1320). Utilities ranged from 1 (full health) to - 0.0059 (the worst health state). Highest weights were assigned to having pain or discomfort (pain/discomfort), followed by walking about (mobility), looking after myself (self-care), doing usual activities (usual activities), and feeling worried, sad, or unhappy (anxiety/depression).ConclusionThis study elicited the Brazilian EQ-5D-Y-3L value set using a mixed-logit DCE model with a power parameter based on a linear mapping without intercept, which can be used to estimate the quality-adjusted life-years for economic evaluations of health technologies targeting the Brazilian youth population.
| S-EPMC11343814 | biostudies-literature
Project description:BackgroundThe Hungarian health technology assessment guidelines recommend the use of the EuroQol instrument family in quality-adjusted life-year calculations. However, no national value set exists for the EQ-5D-Y-3L or any other youth-specific instrument.ObjectiveThis study aims to develop a national value set of the EQ-5D-Y-3L for Hungary based on preferences of the general adult population.MethodsThis study followed the international valuation protocol for the EQ-5D-Y-3L. Two independent samples, representative of the Hungarian general adult population in terms of age and sex were recruited to complete online discrete choice experiment (DCE) tasks and composite time trade-off (cTTO) tasks by computer-assisted personal interviews. Adults valued hypothetical EQ-5D-Y-3L health states considering the health of a 10-year-old child. DCE data were modelled using a mixed logit model with random-correlated coefficients. Latent DCE utility estimates were mapped onto mean observed cTTO utilities using ordinary least squares regression.ResultsOverall, 996 and 200 respondents completed the DCE and cTTO surveys, respectively. For each domain, the value set resulted in larger utility decrements with more severe response levels. The relative importance of domains by level 3 coefficients was as follows: having pain or discomfort > feeling worried, sad or unhappy > mobility > doing usual activities > looking after myself. Overall, 12.3% of all health states had negative utilities in the value set, with the worst health state having the lowest predicted utility of - 0.485.ConclusionThis study developed a national value set of the EQ-5D-Y-3L for Hungary. The value set enables to evaluate the cost utility of health technologies for children and adolescents based on societal preferences in Hungary.
| S-EPMC9485017 | biostudies-literature
Project description:BACKGROUND:The EQ-5D has been frequently used in national health surveys. This study is a head-to-head comparison to assess how expanding the number of levels from three (EQ-5D-3L) to five in the new EQ-5D-5L version has improved its distribution, discriminatory power, and validity in the general population. METHODS:A representative sample (N?=?7554) from the Catalan Health Interview Survey 2011-2012, aged ?18, answered both EQ-5D versions, and we evaluated the response redistribution and inconsistencies between them. To assess validity of this redistribution, we calculated the mean of the Visual Analogue Scale (VAS), which measures perceived health. The discriminatory power was examined with Shannon Indices, calculated for each dimension separately. Spanish preference value sets were applied to obtain utility indices, examining their distribution with statistics of central tendency and dispersion. We estimated the proportion of individuals reporting the best health state in EQ-5D-5L and EQ-5D-3L within groups of specific chronic conditions and their VAS mean. RESULTS:A very small reduction in the percentage of individuals with the best health state was observed, from 61.8% in EQ-5D-3L to 60.8% in EQ-5D-5L. In contrast, a large proportion of individuals reporting extreme problems in the 3 L version moved to severe problems (level 4) in the 5 L version, particularly for pain/discomfort (75.5%) and anxiety/depression (66.4%). The average proportion of inconsistencies was 0.9%. The pattern of the perceived health VAS mean confirmed the hypothesis established a priori, supporting the validity of the observed redistribution. Shannon index showed that absolute informativity was higher in the 5 L version for all dimensions. The means (SD) of the Spanish EQ-5D-3L and EQ-5D-5L indices were 0.87 (0.25) and 0.89 (0.22). The proportion of individuals with the best health state within each specific chronic condition was very similar, regardless of the EQ-5D version (??30% in half of the 28 chronic conditions). CONCLUSION:Although the proportion of individuals with the best possible health state is still very high, our findings support that the increase of levels provided by the EQ-5D-5L contributed to the validity and discriminatory power of this new version to measure health in general population, as in the national health surveys.
| S-EPMC6097421 | biostudies-literature
Project description:EQ-5D is a 5-item questionnaire instrument designed to measure health-related quality of life. It is extremely important, since it is used to measure health benefits in many studies providing evidence for reimbursement decisions by the National Institute for Health and Care Excellence in England and similar policy bodies in other countries. EQ-5D has been redesigned in a more detailed form (EQ-5D-5L), but much existing cost-effectiveness evidence is based on the older version (EQ-5D-3L). Statistical mapping from one version to another is widely used, exploiting data from multi-instrument surveys incorporating both variants. However, little is known about the robustness of data from such multi-instrument surveys. We design a randomized experiment to investigate whether inclusion of both versions at different stages in a single interview gives a reliable picture of the relationship between health measures from the two instruments and embed it in individual interviews from the UK Understanding Society household panel. We find that sequencing of the two versions of EQ-5D within an interview has a significant impact not only on the resulting data but also on the estimated mapping models. We illustrate the non-negligible effects in two real-world cost-effectiveness examples and discuss the implications for future multi-instrument survey design.
| S-EPMC9303872 | biostudies-literature
Project description:Limited utility data on patients suffering from overactive bladder (OAB) are available in the literature. The objectives of this study were to estimate utility values in patients with OAB using the generic EQ-5D questionnaire and the OAB-5D disease specific questionnaire, to investigate the relationship between utilities and symptoms, and to evaluate the sensitivity of the two instruments to changes in symptom severity.Analyses were based on pooled data from three large multicenter randomized 12-week placebo-controlled trials (SCORPIO, ARIES, CAPRICORN). Patients completed a micturition diary, EQ-5D and OAB-q (a quality of life questionnaire from which OAB-5D is derived) at baseline and at weeks 4, 8 and 12. Time trade-off tariffs elicited from UK population were applied to obtain utilities from both instruments. Repeated measures regressions were used to estimate EQ-5D and OAB-5D utilities by micturition frequency and incontinence severity level. As a test of sensitivity of the instruments, utility changes from baseline to week 12 were estimated by symptomatic response (improvement, stable or worsening).The sample included 4427 patients. Mean utilities (± standard deviation) across all visits were 0.82 (± 0.21) for EQ-5D and 0.86 (±0.09) for OAB-5D. Correlation between EQ-5D and OAB-5D was 0.34 (p < 0.0001). Both OAB-5D and EQ-5D utilities increased as OAB symptoms improved. Utility values were similar for severe levels of symptoms, but higher with OAB-5D than with EQ-5D for mild cases. Micturitions and incontinence had similar impact on EQ-5D utilities, but micturitions had greater impact on OAB-5D utilities than incontinence. Changes from baseline in OAB-5D utilities differed significantly according to symptomatic response. Changes in EQ-5D utilities were not significantly associated with changes in micturition frequency and weakly associated with changes in incontinence severity among patients with mild symptoms at baseline.This study showed that both EQ-5D and OAB-5D can detect changes in severity of OAB, especially in severe cases. However, OAB-5D is more sensitive than EQ-5D in measuring differences between treatments in milder cases. Both OAB-5D and EQ-5D-although leading to different results-may be useful to derive utilities from clinical trial data and perform cost-effectiveness analyses.Clinical Trials NCT00689104, NCT00662909, NCT00912964.
| S-EPMC3842710 | biostudies-literature
Project description:European countries more than ever face shifts towards aging societies with accompanying challenges for health and aged care services. Economic evaluation has mainly relied on health measures such as EQ-5D across populations and conditions. We want to know how well the EQ-5D performs in the target population to avoid bias to the disadvantage of older adults and care-dependents. Therefore, we aim to explore differences and similarities of EQ-5D-3L and EQ-5D-5L in comparison to the old-age specific WHOQOL-OLD instrument in a sample of older adults receiving aged care services. We collected data from n = 329 older adults (≥65 years) receiving aged care services in Germany; the majority was at least 80 years and had varying care needs. We assessed instruments' feasibility, test-retest reliability, instruments' association and sensitivity to known-group differences. In terms of feasibility and test-retest reliability both EQ-5D versions performed better than the WHOQOL-OLD. All measures differentiated well between groups based on aspects of general health and care levels. The analysis of relationship between measures indicated that EQ-5D and WHOQOL-OLD assess partially overlapping, but distinct constructs. We found no clear evidence of superiority of either EQ-5D version over the other. The EQ-5D-5L performed better in terms of test-retest reliability and stronger correlations with WHOQOL-OLD facets. We conclude that using the WHOQOL-OLD alongside EQ-5D in this sample added further information on different aspects of quality of life.
| S-EPMC10456181 | biostudies-literature