The Burden Patients with Myasthenia Gravis Experience in Terms of Breathing, Fatigue, Sleep, Mental Health, Discomfort and Usual Activities in Comparison to the General Population.

INTRODUCTION: Myasthenia gravis (MG) is a rare neuromuscular disorder marked by a variable combination of weakness of eye, bulbar, respiratory, axial, and limb muscles. This study compared the experience of people with MG regarding breathing, fatigue, sleep, pain/discomfort, mental health, and usual activities with the general population. METHODS: The MyRealWorld-MG digital, multinational study enrolled patients with MG and collected demographics, PROMIS-Dyspnea, PROMIS-Sleep Disturbance, FACIT-Fatigue, EQ-5D-5L, Health Utilities Index (HUI-3), Hospital Anxiety and Depression Scale (HADS), MG-Activities of Daily Living (MG-ADL), and MG-Quality-of-Life (MG-QoL-15r). Comparisons with the general population were based on PROMIS population norms, published literature, or on data from a digital, multinational, observational study which enrolled a representative sample of the general population (POPUP). RESULTS: In MyRealWorld-MG (N = 2074), patients experienced higher intensity, frequency, and duration of PROMIS shortness of breath than a US population (p < 0.0001). Patients with MG had higher PROMIS-Sleep Disturbance scores than POPUP (53.7 vs 50.0, p < 0.0001), and 54.9% of patients had clinically severe FACIT-Fatigue scores vs 6.8% in POPUP (p < 0.0001). Among patients with MG, 69.6% and 18.5% had moderate-to-severe HADS-Anxiety and HADS-Depression compared to 20.3% and 6.9% in POPUP (p < 0.001). Statistically significant and strong associations were found between fatigue, sleep, dyspnea, usual activities, and emotions. All outcomes worsened with more severe disease. CONCLUSION: A considerable burden was observed in this comparison of breathing, sleep, fatigue, mental health, and usual activities between patients with MG and the general population, using data from two international studies and published population norms. Even mildly affected patients had significantly worse outcomes than the general population.

People Diagnosed with Myasthenia Gravis have Lower health-related quality of life and Need More Medical and Caregiver Help in Comparison to the General Population: Analysis of Two Observational Studies.

INTRODUCTION: Myasthenia gravis (MG) is a neuromuscular disease causing extreme muscular fatigue, triggering problems with vision, swallowing, speech, mobility, dexterity, and breathing. This analysis intended to estimate the health-related quality-of-life impact, the medical burden, and the need for caregiver help of people diagnosed with MG. METHODS: MyRealWorld-MG (MRW) is an observational study among adults diagnosed with MG in 9 countries. The General Population Norms (POPUP) observational study enrolled representative members of the general population in 8 countries. In both digital studies, respondents entered personal characteristics and provided data on medical conditions, EQ-5D-5L, HUI3, MG-Activities of Daily Living (MG-ADL), sick leave, caregiver help, and medical care utilization. RESULTS: In MRW (n = 1859), 58.4% of respondents had moderate-to-severe MG. Average utility values were lower in MRW versus POPUP (0.739 vs. 0.843 for EQ-5D-5L; 0.493 vs. 0.746 for HUI3), and declined with more severe disease (0.872, 0.707, 0.511 EQ-5D-5L utilities and 0.695, 0.443, 0.168 HUI3 utilities for mild, moderate, and severe MG, respectively). Taking sick leave in the past month was 2.6 times more frequent among people diagnosed with MG compared to the general population (34.4% vs. 13.2%) and four times more people diagnosed with MG reported needing help from a caregiver (34.8% vs. 8.3%). Use of medical care was twice as likely in MRW in comparison with POPUP (51.9% vs. 24.6%). CONCLUSION: This direct comparison of people diagnosed with MG and the general population using two large international studies revealed significant negative impact of MG. Results were consistent across all outcomes, in all countries.

Concordance between patient- and physician-reported Myasthenia Gravis Activities of Daily Living (MG-ADL) scores.

INTRODUCTION/AIMS: Myasthenia gravis (MG) is a neuromuscular disease characterized by abnormal skeletal muscle fatiguability. The MG Activities of Daily Living (MG-ADL) scale assesses eight symptoms and is often used as primary endpoint in MG clinical trials where it is completed by neurologists. However, in observational studies, patients frequently complete the MG-ADL scale independently of their neurologist. In this study we aimed to assess the concordance between self- and physician-reported MG-ADL scores. METHODS: An international observational study was conducted among adult patients with MG scheduled for a routine visit or who entered the hospital via emergency services. Consenting patients and physicians completed the MG-ADL. Concordance between assessments was calculated using Gwet's agreement coefficient (Gwet's AC) for the MG-ADL individual items and the intraclass correlation coefficient (ICC) for the MG-ADL total score. RESULTS: Data were collected from 137 patients (63% female; mean age, 57.7 years). Physicians assessed the patient's symptoms as slightly more severe (8.1 vs 7.5 MG-ADL total score, respectively), corresponding to a difference of 0.6 on a range from 0 to 24. The ICC for the MG-ADL total score between the patient and the physician assessment was 0.94 (95% confidence interval, 0.89 to 0.95), showing excellent concordance. Gwet's AC showed substantial to almost perfect agreement for all items, except eyelid droop, for which the agreement was moderate. DISCUSSION: Our results demonstrate that patients and neurologists have a concordant assessment of the patient's MG symptoms when using the MG-ADL scale. This evidence supports patient self-administration of the MG-ADL in clinical practice and research.

Patient-reported burden of myasthenia gravis: baseline results of the international prospective, observational, longitudinal real-world digital study MyRealWorld-MG.

OBJECTIVES: Myasthenia gravis (MG) is a rare, chronic, autoimmune neuromuscular disease which can affect functional and mental aspects of health and health-related quality of life (HRQoL). This study aims to obtain detailed knowledge of the impact of MG on HRQoL in a broad population from the perspective of the patient. DESIGN: Prospective, observational, digital, longitudinal real-world study. SETTING: Adult patients with MG from seven countries (USA, Japan, Germany, UK, Italy, Spain and Canada) downloaded a mobile application onto their phones and entered data about themselves and their MG. OUTCOME MEASURES: Data was collected using the following general and disease-specific patient-reported outcome measurements: EuroQol 5 Domains Health-Related Quality of Life Questionnaire (EQ-5D-5L), Myasthenia Gravis Activities of Daily Living (MG-ADL), Myasthenia Gravis Quality of Life 15-item revised scale (MG-QoL-15r), Hospital Anxiety and Depression Scale (HADS) and Health Utilities Index III (HUI3). Patients were categorised by their self-assessed Myasthenia Gravis Foundation of America (MGFA) class (I-V). RESULTS: Baseline results of 841 participants (mean age 47 years, 70% women) are reported . The distribution across the MGFA classes was: 13.9%, 31.0%, 38.1%, 15.5% and 1.6% for classes I-V. The MGFA class was a strong predictor of all aspects of HRQoL, measured with disease-specific and with generic instruments. The domains in which patients with MG most frequently mentioned problems were usual activities, anxiety and depression, tiredness, breathing and vision. The mean total MG-ADL Score was positively associated with increasing MGFA classes: 2.7, 4.4, 6.3 and 8.4 for MGFA classes I-IV. Mean baseline EQ-5D-5L utility was also associated with MGFA classes and was 0.817, 0.766, 0.648 and 0.530 for MGFA class I-IV. CONCLUSIONS: MG has a large impact on key aspects of health and HRQoL. The impact of this disease increases substantially with increasing disease severity.

What is measured by the composite, single-item pain/discomfort dimension of the EQ-5D-5L? An exploratory analysis.

PURPOSE: This study examines the EQ-5D-5L pain/discomfort dimension by drawing comparisons with five other pain and discomfort items (pain severity, discomfort severity, pain frequency, discomfort frequency and pain interference) collected in the Australian psychometric study for the EQ Health and Wellbeing instrument. METHODS: Participants, recruited via a market research company, completed an online survey. Methods of analyses included the assessment of descriptive statistics, variation in reporting patterns using chi-square tests and cross-tabulations, correlation analyses, ordered univariate logistic regression, and discriminatory power analyses (Shannon index (H') and Shannon Evenness index (J')). RESULTS: Survey data from 514 participants were used. Compared with EQ-5D-5L pain/discomfort, there was a higher proportion of respondents reporting some level of impairment on at least one of the pain severity and discomfort severity items (74% versus 81%). Correlation with EQ-5D-5L pain/discomfort was strongest for pain severity (r = 0.83) and weakest for discomfort frequency (r = 0.41); the same inferences were drawn for predictive ability. Adding any additional pain or discomfort items to the EQ-5D-5L increased the absolute informativity (H') but not the relative informativity (J'). When replacing EQ-5D-5L pain/discomfort with separate pain and/or discomfort items - i.e., adding items to a modified 'EQ-4D-5L'-absolute informativity increased, while relative informativity increased only when pain interference and frequency-related items (independently or in combination) were added. CONCLUSION: The EQ-5D-5L pain/discomfort dimension captures aspects of pain more than aspects of discomfort. Potential reasons include the absence of descriptors or because pain is mentioned first in the composite item.

Adding a fatigue item to the EQ-5D-5L improves its psychometric performance in the general population.

BACKGROUND: Fatigue is a common and often disturbing sequela of serious chronic health conditions. In the widely applied HRQL instrument, the EQ-5D, this aspect is not included directly, for its assumed lack of additional information. We investigated the validity of this assumption by determining the gain-if any-of an additional fatigue item to the EQ-5D-5L in a general population sample. METHODS: A Dutch general population sample (including diseased people) completed a web-based survey including the EQ-5D-5L and the Rivermead Post-Concussion Symptoms Questionnaire (RPQ). The RPQ fatigue item was used to create the EQ-5D-5L + Fatigue. We head-to-head compared the psychometric performance contrasting the EQ-5D-5L and EQ-5D-5L + Fatigue: distribution (e.g. ceiling), informativity cf. Shannon's indices, convergent validity, domain dependency, and explanatory power. Results were compared between subgroups with and without ≥ 1 chronic health condition. RESULTS: The study population consisted of 3027 persons of whom 52% had a chronic health condition. The mean EQ-5D-5L utility score was 0.83 and 48% experienced some degree of fatigue. Adding the fatigue item to the EQ-5D-5L decreased the ceiling effect, increased absolute informativity (H' = 6.44 vs. H' = 4.90) and relative informativity (J' = 0.46 vs. J' = 0.42). The extra fatigue item slightly increased convergent validity (Spearman's rank correlation coefficient = - 0.61 vs. - 0.62). Domain dependency analysis showed that all EQ-5D-5L domains are dominant over the fatigue item. Explanatory power of the EQ-5D-5L + Fatigue was higher compared to the EQ-5D-5L (R2 = 0.42 vs. 0.39). The gain is substantially larger in the subgroup with chronic health conditions. CONCLUSIONS: Adding a fatigue item to the EQ-5D-5L improved all psychometric performance criteria of the enriched instrument in the general population. Effects are substantially larger in the subgroup with chronic health conditions, indicating that adding a fatigue item to the EQ-5D-5L is especially relevant in evaluating the HRQL of diseased people.

Health Inequality Analysis in Europe: Exploring the Potential of the EQ-5D as Outcome.

Objective: This study explored the additive value of the multi-item EuroQol 5-Dimension 5-Level (EQ-5D-5L) as an outcome measure in health inequality analyses, relative to the single-item EuroQol visual analog scale (EQ VAS). Methods: A sample comprising the general population from Italy, the Netherlands, and United Kingdom (UK) completed the EQ-5D-5L and the EQ VAS. The level of education was selected as a proxy for socio-economic status (SES). EQ-5D-5L level sum scores (LSS) were compared against EQ VAS scores. Stratified and multivariable analyses were used to study the associations between SES and the LSS/EQ VAS relative to the presence of chronic health conditions. Results: A total of 10,172 people participated in this study. In the UK and Netherlands, the LSS was worst for respondents with a low educational level and better for respondents with middle and high educational levels. For Italy, the LSS was best for respondents with a middle educational level compared to respondents with low and high educational levels. The same patterns were observed for the EQ VAS, but differences were slightly smaller. Multivariable analyses showed generally stronger predictive relations in the UK, and with the LSS. The presence of chronic health conditions and being unable to work were independent strong predictors, canceling out the effects of education. Conclusions: In three different European countries, the EQ-5D measures show the presence of education-dependent health inequalities, which are universally explained in regression analysis by independently the presence of chronic health conditions and the inability to work. In stratified analysis, the EQ-5D-5L LSS discriminates slightly better between participants with different levels of SES compared to the EQ VAS.

The relation between EQ-5D and fatigue in a Dutch general population sample: an explorative study.

BACKGROUND: Fatigue negatively influences health-related quality of life. It is questionable whether fatigue is sufficiently covered by the EQ-5D. This study investigated whether fatigue is covered by the existing domains of the EQ-5D. METHODS: A Dutch general population sample completed the EQ-5D (3L and 5L version) and the Rivermead Post-Concussion Symptoms Questionnaire (RPQ), of which the fatigue item was used. Outcomes were compared between participants with and without a chronic health condition. Convergent validity was assessed, and multivariate regression analyses was used to predict the RPQ fatigue item from the EQ-5D-3L and EQ-5D-5L domains separately. RESULTS: 3027 people completed the survey, of whom 52% had ≥ 1 chronic health condition. Fatigue was reported by 48% of the participants. Fatigue was moderately correlated to the EQ-5D domains 'pain/discomfort', 'usual activities', and 'anxiety/depression' for the 3L (r = 0.379-0.426) and 5L version (r = 0.411-0.469). For the 5L, also a moderate correlation with 'mobility' (r = 0.335) was observed. The remaining correlations were weak. All EQ-5D-3L and 5L domains except for 'mobility' were significantly associated with the RPQ fatigue item (unstandardized Beta = - 0.20-0.67; p < 0.01 to p = 0.04). Comparable outcomes were found for participants with and without ≥ 1 chronic health condition. CONCLUSIONS: The extent to which fatigue is covered by the EQ-5D domains is small to moderate, with the EQ-5D-5L being slightly more sensitive to capture fatigue compared to the EQ-5D-3L. An extra fatigue item for the EQ-5D may add value, as fatigue is not fully captured by the existing domains, both in people with and without a chronic health condition.

The added value of extending the EQ-5D-5L with an itching item for the assessment of health-related quality of life of burn patients: an explorative study.

INTRODUCTION: Health-related quality of life (HRQL) is an important outcome in burn care and research. An advantage of a generic HRQL instrument, like the EQ-5D, is that it enables comparison of outcomes with other conditions and the general population. However, the downside is that it does not include burn specific domains, like scar issues or itching. Adding extra items to a generic instrument might overcome this issue. This study explored the potential and added value of extending the EQ-5D-5L with a burn-specific item, using a itching item as an example. METHODS: The EQ-5D-5L and the Patient and Observer Scar Assessment Scale (POSAS) was completed by adult patients 5-7 years after injury. A separate POSAS itching item was used to study the added value of an itching item for the EQ-5D-5L. The EQ-5D-5L + Itching was created by adding the POSAS itching item to the EQ-5D-5L. Five psychometric properties were compared between EQ-5D-5L and EQ-5D-5L + Itching: distribution (e.g. ceiling), informativity cf. Shannon's indices, convergent validity, dimension dependency, and explanatory power respectively. RESULTS: A total of 243 patients were included, of whom 49% reported any itching on the POSAS. Adding an itching item to the EQ-5D-5L decreased the ceiling effect, and resulted in increased absolute informativity (H' = 4.76 vs. H' = 3.64) and relative informativity (J' = 0.34 vs. J' = 0.31). The extra itching item decreased the convergent validity (Spearman's rank correlation coefficient = -0.51 vs. -0.59). Mutual dependency of dimensions existed, showing that all other items were dominant over the itching item. Adding the itching item to the standard EQ-5D-5L barely improved explanatory power (49.3% vs. 49.0%). CONCLUSIONS: PThe present study showed adding a burn-specific item to the EQ-5D-5L is possible and has potential. However, 5 to 7 years after injury, adding an itching item to the EQ-5D-5L provides little additional information; the gain in terms of added value is relatively small. Apart from instances where itching information is specifically needed, a strong case is not present for adding an itching item to the EQ-5D-5L for long-term (>5 yr after burns) HRQL assessment in burn patients. In early time periods after burn, the added value might be greater and we recommend exploring this potential in future studies, ideally on multiple timepoints after burn.

US population norms for the EQ-5D-5L and comparison of norms from face-to-face and online samples.

PURPOSE: Normative scores (norms) allow for comparisons between population(s) of interest and the general population, which is useful for burden of disease studies and cost-effectiveness analysis. The primary aim of this study was to estimate US visual analogue scale (EQ VAS) and utility-based norms for the EQ-5D-5L using the face-to-face sample. The secondary aim was to compare norms estimated in the face-to-face and online populations. METHODS: This study estimated population norms from two general population surveys: (a) face-to-face and (b) online. In these surveys, respondents provided their health state using the EQ-5D-5L health classifier and the EQ VAS. Descriptive statistics, including mean, standard deviation (SD), 95% confidence interval, and median for the 5L utility and EQ VAS were estimated for each sample and across relevant respondent characteristics to serve as the basis for US EQ-5D-5L norms RESULTS: Face-to-face sample respondents (n = 1134) were representative of the US adult general population. In this sample, mean (SD) utility decreased with increasing age until age 45 or greater (age 45-54: 0.816 (0.249) age 55-64: 0.815 (0.243) age 65-74: 0.824 (0.217) age 75 + : 0.811 (0.218)). With increasing age, more problems were reported on all dimensions except anxiety/depression; a smaller proportion of respondents age 65 and older reported problems with anxiety/depression (23.8%) as compared to the youngest respondents (42.1%). Online (n = 2018) mean utility and EQ VAS values were consistently lower than the face-to-face sample. CONCLUSIONS: The availability of US EQ-5D-5L norms facilitates interpretation and understanding of general population and patient health.

Exploring the relation between the EQ-5D-5L pain/discomfort and pain and itching in a sample of burn patients.

BACKGROUND: The EQ-5D domain pain/discomfort (PD) uses one item to capture pain and other aspects of discomfort, like itching. This study explored how pain, itching and the EQ-5D-5L PD domain relate to each other in a sample of burn patients. METHODS: Adult burn patients completed the EQ-5D-5L and the Patient and Observer Scar Assessment Scale (POSAS) 5-7 years after sustaining their injury. The POSAS includes a separate pain and an itching item. Spearman's correlation coefficient established the association between the EQ-5D-5L PD and the POSAS pain and itching item. With multivariable regression analysis the linear association between the POSAS pain and itching item and EQ-5D-5L PD domain was tested. RESULTS: Data from 245 patients were included. Mean EQ-5D-5L index value was 0.87 and 39.2% reported at least slight problems on the EQ-5D-5L PD domain. Most patients gave corresponding answers on the EQ-5D-5L PD domain and on the POSAS pain (73%) and itching (70%) item. Spearman correlation coefficients of the EQ-5D-5L PD domain with the POSAS pain and itching were 0.468 (p < 0.001) and 0.473 (p < 0.001), respectively. Among respondents with pain and without itching and respondents with itching and without pain, Spearman correlation coefficients were 0.585 (p = 0.076) and 0.408 (p = 0.001), respectively. POSAS pain (unstandardized Beta = 0.14) and POSAS itching (unstandardized Beta = 0.08) were significantly associated with EQ-5D-5L PD domain (p < 0.001). CONCLUSIONS: Our findings indicate that, in a sample of burn patients, pain and itching are captured by the broader EQ-5D-5L PD domain. The EQ-5D-5L PD domain can thus be used to assess pain and itching in relation to HRQL, but the POSAS pain and itching items are more sensitive. The EQ-5D-5L is, however, no replacement of the POSAS when the POSAS is used for its primary aim; assessment of scar quality. TRIAL REGISTRATION: Netherlands Trial Register (NTR6407).

Does the EQ-5D-5L benefit from extension with a cognitive domain: Testing a multi-criteria psychometric strategy in trauma patients.

PURPOSE: This study investigated the psychometric yield of extension of the EQ-5D-5L with a cognitive domain (EQ-5D+C) in a mixed cohort of trauma patients with repeated data. METHODS: A stratified sample of patients that presented at the emergency department filled out a follow-up survey 6 and 12 months after trauma. The surveys included the EQ-5D-5L+C, EQ-VAS, and the impact of events scale-revised (IES-R), a validated post-traumatic stress disorder (PTSD) self-assessment scale. Generally, results of the EQ-5D and EQ-5D+C were compared. Psychometrics included the following: distributional features (ceiling/floor effects), discriminatory performance, convergent validity with the EQ-VAS as reference, and responsiveness to change. Psychometric properties were compared between predefined subgroups based on conditions with cognitive impact (Traumatic Brain Injury (TBI)/PTSD). RESULTS: In total, 1799 trauma patients responded 6 and 12 months after trauma, including 107 respondents with PTSD, and 273 with TBI. Six months post-trauma, ceiling of the EQ-5D (26.3%) was reduced with 2.2% with the additional cognitive domain. Using EQ-VAS as reference, convergent validity increased slightly with the addition of the cognitive domain: correlation increasing from 0.651 to 0.664. Cognitive level was found to slightly improve over time in TBI (delta: 0.04) and PTSD patients (delta: 0.05), while (almost) no change was found in patients without TBI and PTSD. CONCLUSION: Adding a cognitive domain to the EQ-5D-5L slightly improved measurement properties and better captured change in health status for trauma patients with TBI and PTSD. Inclusion of the cognitive domain in the EQ-5D-5L when measuring in populations with cognitive problems should be considered.

Population norms for the EQ-5D-3L: a cross-country analysis of population surveys for 20 countries.

This study provides EQ-5D population norms for 20 countries (N = 163,838), which can be used to compare profiles for patients with specific conditions with data for the average person in the general population in a similar age and/or gender group. Descriptive EQ-5D data are provided for the total population, by gender and by seven age groups. Provided index values are based on European VAS for all countries, based on TTO for 11 countries and based on VAS for 10 countries. Important differences exist in EQ-5D reported health status across countries after standardizing for population structure. Self-reported health according to all five dimensions and EQ VAS generally decreased with increasing age and was lower for females. Mean self-rated EQ VAS scores varied from 70.4 to 83.3 in the total population by country. The prior living standards (GDP per capita) in the countries studied are correlated most with the EQ VAS scores (0.58), while unemployment appeared to be significantly correlated in people over the age of 45 only. A country's expenditure on health care correlated moderately with higher ratings on the EQ VAS (0.55). EQ-5D norms can be used as reference data to assess the burden of disease of patients with specific conditions. Such information, in turn, can inform policy-making and assist in setting priorities in health care.

Logical inconsistencies in time trade-off valuation of EQ-5D-5L health states: Whose fault is it?

INTRODUCTION: Inconsistency in the time trade-off (TTO) task in EQ-5D-5L occurs when a respondent gives a higher value to a logically worse health state, the occurrence of inconsistency compromises the quality of the data. It is not yet clear which factors are associated with individual level inconsistency. Relating inconsistency to the characteristics of the respondent, interviewer, and the interview process could be helpful in understanding the causes of inconsistency. The objective of this paper is to discover the factors associated with individual level inconsistencies. METHODS: Twenty interviewers interviewed 1,296 respondents and each respondent valued 10 health states using the EQ-VT platform in 5 cities in China. At the respondent level, inconsistency was identified in terms of severity and quantity and related to the respondent's background characteristics, the time and iterations spent on the wheelchair example task, and the formal TTO tasks, using multilevel multinomial regression analyses. Interviewers' impact on inconsistencies was analyzed using single level multinomial regression analyses. RESULTS: In the full dataset, slight inconsistency was more related to the interview process (Time spent on TTO task: RRR = 1.246 with 95%CI: 1.076,1.441; time spent on Wheelchair example: RRR = 0.815 with 95%CI:0.699,0.952) while severe inconsistency was more related to respondent's gender (Gender: RRR = 2.347 with 95%CI:1.429,3.855). One Interviewer (Interviewer 7: RRR = 7.335 with 95%CI:1.908,28.195) and interviewer's experience (Sequence: RRR = 0.511 with 95%CI:0.385,0.678) in general showed strong influence over inconsistency in the TTO task. CONCLUSION: In conclusion, logical inconsistency in the valuation of EQ-5D-5L health states is associated not only with respondents' characteristics but also with interviewers' performance and the interview process. The role of interviewers and the importance of interviewer training may be more crucial than hitherto believed. This finding could be generalizable to other interviewer-administered health-state valuation study.

Views of the UK General Public on Important Aspects of Health Not Captured by EQ-5D.

INTRODUCTION: The EQ-5D is a standardised instrument designed for use as a generic measure of health outcome. It was (and is) intended to provide information about a 'common core' of dimensions known to be relevant across a range of conditions; however, the five dimensions may not fully capture the health-related impacts of certain conditions. This study analyses the views of the UK general public about important aspects of health considered to be missing from the instrument. METHODS: Survey respondents were asked whether there are any aspects of health they consider to be important but are not captured by the EQ-5D, and, if so, what these aspects are. The responses (text comments) were analysed using content analysis with analyst triangulation. Data were collected from a broadly representative sample of the general public via a paper questionnaire administered as part of face-to-face interviews. RESULTS: Data are available for 436 respondents, 179 of whom suggested aspects of health they considered important but not captured by the five EQ-5D dimensions. These were organised into 22 themes. Sensory deprivation and mental health were the health aspects most commonly mentioned by respondents. CONCLUSIONS: Respondents identified several important aspects of health that are not covered by the EQ-5D descriptive system. This study can provide the basis for more detailed qualitative and quantitative research-in particular to examine the views of different patient groups-to inform further review of the EQ-5D descriptive system. The results also have implications for the sensitivity of other generic measures.

Validity of EQ-5D-5L in stroke.

PURPOSE: To assess EQ-5D-5L (5L) validity in patients with acute stroke, in comparison with EQ-5D-3L (3L). METHODS: Cross-sectional study of 408 patients during index hospitalization. We compared 5L and 3L in terms of feasibility, frequency of unique health states, ceiling effect and discriminatory power (informativity). We assessed construct validity in terms of known-groups validity and convergent validity of 5L dimensions with other stroke outcome measures. RESULTS: The overall proportion of patients with acute stroke reporting 'no problems' with 3L-6.1 % was further reduced to 5.6 % with 5L (relative reduction of 8.2 %). The highest improvement in relative discriminatory power, when moving from 3L to 5L, was noticed in pain/discomfort and anxiety/depression dimensions (Shannon Evenness Index 0.91 for both 5L dimensions; relative increase 34.4 and 29.1 %, respectively). Known-groups validity tests confirmed prior hypotheses: Health state utilities were lower in following subpopulations-females, patients with high modified Rankin Scale (mRS) score, low Barthel Index (BI) or VAS score, patients with subarachnoid hemorrhage or intracerebral hemorrhage, and when proxy respondent was used. Convergence of EQ-5D-5L dimensions with mRS, BI and EQ VAS was improved or at least the same as for 3L dimensions. CONCLUSIONS: Results support the validity of the EQ-5D-5L descriptive system as a generic health outcome measure in patients with acute stroke, demonstrating some psychometric advantages in comparison with EQ-5D-3L.

Comparing responsiveness of the EQ-5D-5L, EQ-5D-3L and EQ VAS in stroke patients.

AIMS: To date, evidence to support the construct validity of the EQ-5D-5L has primarily focused on cross-sectional data. The aims of this study were to examine the responsiveness of EQ-5D-5L in patients with stroke and to compare it with responsiveness of EQ-5D-3L and visual analogue scale (EQ VAS). METHODS: We performed an observational longitudinal cohort study of patients with stroke. At 1 week and 4 months post-stroke, patients were assessed with modified Rankin Scale (mRS) and Barthel Index (BI) and were administered the EQ-5D-5L and EQ-5D-3L, including the EQ VAS. The EQ-5D-5L index scores were derived using the crosswalk methodology developed by the EuroQol Group. We classified patients according to two external criteria, based on mRS or BI, into 3 categories: 'improvement,' 'stable' or 'deterioration'. We assessed the responsiveness of each measure in each patient subgroup using: effect size (ES), standardized response mean (SRM), F-statistic, relative efficiency and area under the receiver operating characteristic curve. RESULTS: A total of 112 patients (52% females; mean age 70.6 years; 93% ischemic stroke) completed all the instruments at both occasions. In subjects with clinical improvement, EQ-5D-5L was consistently responsive, showing moderate ES (0.51-0.71) and moderate to large SRM (0.69-0.86). In general, EQ-5D-3L index appeared to be more responsive (ES 0.63-0.82; SRM 0.77-1.06) and EQ VAS less responsive (ES 0.51-0.65; SRM 0.59-0.69) than EQ-5D-5L index. CONCLUSIONS: The EQ-5D-5L index, based on the crosswalk value set, seems to be appropriately responsive in patients with stroke, 4 months after disease onset. As far as EQ-5D-5L index is scored according to crosswalk approach, the EQ-5D-3L index appears to be more responsive in stroke population.

The validity of the EQ-5D-3L items: an investigation with type 2 diabetes patients from six European countries.

BACKGROUND: Most previous studies concerning the validity of the EQ-5D-3L items refer to applications of only a single language version of the EQ-5D-3L in only one country. Therefore, there is little information concerning the extent to which the results can be generalised across different language versions and/or different countries. Here the validity of the EQ-5D-3L items is investigated for six different language versions in six different countries. METHODS: Data came from 1341 type 2 diabetes patients (England: 289; Finland: 177; Germany: 255; Greece: 165; the Netherlands: 354; Spain: 101). The relationships of the five EQ-5D-3L items with seven different test variables (age, gender, education, previous stroke, problems with heart, problems with lower extremities, problems with eyes), were analysed for each combination of item and test variable. For each combination two logistic regression models with the dichotomised EQ-5D-3L item as dependent variable were computed. The first model contained the test variable and dummy coded countries as independent variables, the second model additionally the terms for the interaction between country and test variable. Statistically significant better fit of the second model was taken as evidence for country specific differences regarding the relationship. When such differences could be attributed mainly to one country the analyses were repeated without the data from this country. Validity was investigated with the remaining data using results of the first models. RESULTS: Due to lack of variation in the Spanish data only 31 of the originally intended 35 interaction tests could be performed. Only three of these yielded a significant result. In all three cases the Spanish data deviated most. Without the Spanish data only 1 of the 35 interaction tests yielded a significant result. With 3 exceptions, the tendency of reporting problems increased with age, female gender, lower education, previous stroke, heart problems, problems with lower extremities and problems with eyes for all EQ-5D-3L items. CONCLUSION: The results concerning the European Spanish version are ambiguous. However, the items of the English, Finnish, German, Greek and Dutch versions of the EQ-5D-3L relate in substantially the same way to the test variables. Mostly, these relationships indicate the items' validity.

Interim EQ-5D-5L Value Set for Poland: First Crosswalk Value Set in Central and Eastern Europe.

OBJECTIVE: To estimate an interim five-level EuroQol five-dimensional (EQ-5D-5L) value set for Poland on the basis of the crosswalk methodology developed by the EuroQol Group. METHODS: On the basis of data from 3691 respondents from six European countries, the EuroQol Group has developed a method of obtaining interim value sets for the EQ-5D-5L by means of mapping to the available three-level EuroQol five-dimensional (EQ-5D-3L) value sets ("crosswalk" methodology). A significant part of the data in this study came from Polish respondents (n = 972; 26.3%). Poland is the first Central European country with EQ-5D-3L time trade-off-based social value set published. To obtain an interim EQ-5D-5L value set, we applied the crosswalk methodology to the Polish EQ-5D-3L value set. RESULTS: Estimated Polish values for 3125 EQ-5D-5L health states are presented. Both EQ-5D-5L and EQ-5D-3L value sets have the same range (from -0.523 to 1.000), but different means (0.448 vs. 0.380) and medians (0.483 vs. 0.403), respectively. Proportionately fewer states worse than dead were observed in the EQ-5D-5L (5.4%) value set than in the EQ-5D-3L (13.2%) value set. CONCLUSIONS: The crosswalk-based value set is available for use in EQ-5D-5L studies in Poland to calculate health state utilities. It should be considered an interim value set until values based on preferences elicited directly from a sample representative of the Polish general population become available. This study helps users of the crosswalk algorithm understand the properties of the EQ-5D-5L values generated using this method, in comparison to EQ-5D-3L values obtained with the Polish time trade-off value set. It is likely that similar results would be observed for values sets in other countries because the same crosswalk methodology applies across all countries.