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1.

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with histiocytosis in Europe.

Methods

We conducted a cross-sectional study of patients with histiocytosis from France, Germany, Italy, Spain, Bulgaria, the UK, and Sweden. Data on demographic characteristics, health resource utilisation, informal care, loss of labour productivity and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.

Results

A total of 134 patients (35 France, 32 Germany, 30 Italy, 24 Spain, 7 Bulgaria, 4 UK and 2 Sweden) completed the questionnaire. The average annual costs ranged from € 6832 to € 33,283 between countries, the year of reference being 2012. Estimated direct healthcare costs ranged from € 1698 to € 18,213; direct nonhealthcare costs ranged from € 2936 to € 17,622 and labour productivity losses ranged from € 1 to € 8855. The mean EQ-5D score for adult histiocytosis patients was estimated at between 0.32 and 0.85, and the mean EQ-5D visual analogue scale score was estimated at between 50.00 and 66.50.

Conclusion

The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of histiocytosis patients from a broad perspective (societal costs). This type of analysis is very scarce in international literature for rare diseases in comparison with other illnesses. We conclude that histiocytosis patients incur considerable societal costs and experience substantial deterioration in HRQOL.
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2.

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with juvenile idiopathic arthritis (JIA) in Europe.

Methods

We conducted a cross-sectional study of patients with JIA from Germany, Italy, Spain, France, the United Kingdom, Bulgaria, and Sweden. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D-5L) questionnaire.

Results

A total of 162 patients (67 Germany, 34 Sweden, 33 Italy, 23 United Kingdom, 4 France, and 1 Bulgaria) completed the questionnaire. Excluding Bulgarian results, due to small sample size, country-specific annual health care costs ranged from €18,913 to €36,396 (reference year: 2012). Estimated direct healthcare costs ranged from €11,068 to €22,138; direct non-healthcare costs ranged from €7837 to €14,155 and labor productivity losses ranged from €0 to €8715. Costs are also shown to differ between children and adults. The mean EQ-5D index score for JIA patients was estimated at between 0.44 and 0.88, and the mean EQ-5D visual analogue scale score was estimated at between 62 and 79.

Conclusions

JIA patients incur considerable societal costs and experience substantial deterioration in HRQOL in some countries. Compared with previous studies, our results show a remarkable increase in annual healthcare costs for JIA patients. Reasons for the increase are the inclusion of non-professional caregiver costs, a wider use of biologics, and longer hospital stays.
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3.

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with Duchenne muscular dystrophy (DMD) in Europe.

Methods

We conducted a cross-sectional study of patients with DMD from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden, and the UK. Data on demographic characteristics, healthcare resource utilization, informal care, labor productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire. Costs have been estimated from a societal perspective adopting a bottom-up approach.

Results

A total of 422 questionnaires were included in the study; 268 of which were collected from patients with DMD and 154 from caregivers. The average annual cost per person in 2012 ranged from €7657 in Hungary to €58,704 in France. Direct non-healthcare costs are the main component of whole costs and informal care is the main driver of non-healthcare costs. Costs are also shown to differ between children and adults. With regard to HRQOL of adult patients, the EQ-5D VAS score and EQ-5D index scores were 50.5 and 0.24, respectively. The corresponding EQ-5D VAS and EQ-5D index scores for caregivers were 74.7 and 0.71, respectively.

Conclusions

We have estimated the average annual cost per patient with DMD in eight European countries adopting a social perspective, and to our knowledge this is the first study with such a wide perspective. The results on costs show a considerable gap between Eastern and Western European countries. Non-healthcare costs range from 64 to 89 % of overall costs and informal care is to a great extent the main driver of this cost category. The HRQOL of people with DMD is much lower than that of the general population.
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4.

Objective

The aim of this study was to determine the economic burden from a societal perspective and health-related quality of life (HRQOL) of patients with Prader-Willi syndrome (PWS) in Europe.

Methods

We conducted a cross-sectional study of patients with PWS from Spain, Bulgaria, Hungary, Germany, Italy, the UK, Sweden and France. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.

Results

A total of 261 patients completed the questionnaire. The average annual costs ranged from € 3937 to € 67,484 between countries; the reference year for unit prices was 2012. Direct healthcare costs ranged from € 311 to € 18,760, direct non-healthcare costs ranged from € 1269 to € 44,035, and loss of labour productivity ranged from € 0 to € 2255. Costs were also shown to differ between children and adults. The mean EQ-5D index score for adult PWS patients ranged between 0.40 and 0.81 and the mean EQ-5D visual analogue scale score ranged between 51.25 and 90.00.

Conclusion

The main strengths of this study lie in our bottom-up approach to costing and in the evaluation of PWS patients from a broad societal perspective. This type of analysis is very scarce in the international literature on rare diseases in comparison with other illnesses. We conclude that PWS patients incur considerable societal costs and experience substantial deterioration in HRQOL.
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5.

Background

The aim of this study was to determine the social/economic costs and health-related quality of life (HRQOL) of patients with epidermolysis bullosa (EB) in eight EU member states.

Methods

We conducted a cross-sectional study of patients with EB from Bulgaria, France, Germany, Hungary, Italy, Spain, Sweden and the United Kingdom. Data on demographic characteristics, health resource utilisation, informal care, labour productivity losses, and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.

Results

A total of 204 patients completed the questionnaire. Average annual costs varied from country to country, and ranged from €9509 to €49,233 (reference year 2012). Estimated direct healthcare costs ranged from €419 to €10,688; direct non-healthcare costs ranged from €7449 to €37,451 and labour productivity losses ranged from €0 to €7259. The average annual cost per patient across all countries was estimated at €31,390, out of which €5646 accounted for direct health costs (18.0 %), €23,483 accounted for direct non-healthcare costs (74.8 %), and €2261 accounted for indirect costs (7.2 %). Costs were shown to vary across patients with different disability but also between children and adults. The mean EQ-5D score for adult EB patients was estimated at between 0.49 and 0.71 and the mean EQ-5D visual analogue scale score was estimated at between 62 and 77.

Conclusion

In addition to its negative impact on patient HRQOL, our study indicates the substantial social/economic burden of EB in Europe, attributable mostly to high direct non-healthcare costs.
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6.

Objectives

Our goal was to provide data on the economic burden and health-related quality of life (HRQOL) of patients with cystic fibrosis (CF) and their caregivers in Europe.

Methods

A cross-sectional study was carried out on adults and children with CF in eight European countries. Patients completed an anonymous questionnaire regarding their socio-demographic characteristics, use of healthcare services and presence of a caregiver. Costs were calculated with a bottom-up approach using unit costs from each participating country, and HRQOL was assessed using EQ-5D. The principal caregiver also answered a questionnaire on their characteristics, HRQOL and burden.

Results

A total of 905 patients with CF was included (399 adults and 506 children). The total average annual cost per patient varied from €21,144 in Bulgaria to €53,256 in Germany. Adults had higher direct healthcare costs than children, but children had much higher informal care costs (P < 0.0001). Total costs increased with patients’ level of dependence. In adults, mean utility fell between 0.640 and 0.870, and the visual analogue scale ranged from 46.0 to 69.7. There was no difference in caregiver HRQOL regardless of whether they cared for an adult or a child. However, caregivers who looked after a child had a significantly higher burden (P = 0.0013).

Conclusions

Our study highlights the burden of CF in terms of costs and decreased HRQOL for both patients and their caregivers throughout Europe.
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7.

Objective

The aim of this study was to determine the economic burden from a societal perspective and the health-related quality of life (HRQOL) of patients with systemic sclerosis (SSc; scleroderma) in Europe.

Methods

We conducted a cross-sectional study of patients with SSc (involving both localised and systemic sclerosis) from Germany, Italy, Spain, France, the UK, Hungary and Sweden. Data on demographic characteristics, healthcare resource utilisation, informal care, labour productivity losses and HRQOL were collected from the questionnaires completed by patients or their caregivers. HRQOL was measured with the EuroQol 5-domain (EQ-5D) questionnaire.

Results

A total of 589 patients completed the questionnaire. The rate of patients with localised scleroderma, limited cutan and diffuse cutan SSc were 28, 68 and 4 %, respectively. Average annual costs varied from country to country and ranged from € 4607 to € 30,797 (reference year: 2012). Estimated direct healthcare costs ranged from € 1413 to € 17,300; direct non-healthcare costs ranged from € 1875 to € 4684 and labour productivity losses ranged from € 1701 to € 14,444. The mean EQ-5D index score for adult SSc patients varied from 0.49 to 0.75 and the mean EQ-5D visual analogue scale score was between 58.72 and 65.86.

Conclusion

The main strengths of this study lie in our bottom-up approach to costing and our evaluation of SSs patients from a broad societal perspective. This type of analysis is very unusual in the international literature on rare diseases in comparison with other illnesses. We concluded that SSc patients incur considerable societal costs and experience substantial deterioration in HRQOL.
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8.

Objective

To estimate the social/economic costs of fragile X syndrome (FXS) in Europe and to assess the health-related quality of life (HRQOL) of patients and caregivers.

Methods

A cross-sectional study was conducted in a sample of European countries. Patients were recruited through patients’ associations. Data on their resource use and absence from the labour market were retrospectively obtained from an online questionnaire. Costs were estimated by a bottom-up approach and the EuroQol-5 Domain (EQ-5D) questionnaire was used to measure patients’ and caregivers’ HRQOL.

Results

Five countries were included in the analysis. The mean annual cost of FXS per patient varied from €4951 in Hungary to €58,862 in Sweden. Direct non-healthcare costs represented the majority of costs in all countries but there were differences in the share incurred by formal and informal care among those costs. Costs were also shown to differ between children and adults. Mean EQ-5D utility score for adult patients varied from 0.52 in France (n = 42) to 0.73 in Hungary (n = 2), while for caregivers this score was consistently inferior to 0.87.

Conclusion

Our findings underline that, although its prevalence is low, FXS is costly from a societal perspective. They support the development of tailored policies to reduce the consequences of FXS on both patients and their relatives.
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9.

Background

Although tea consumption has been reported to have various health benefits in humans, its association with health-related quality of life (HRQOL) has not been investigated directly. We aimed to examine the relationship between tea consumption and HRQOL among older Chinese adults.

Methods

We analyzed community-based cross-sectional data of 5,557 older Chinese individuals aged 60 years or older who participated in the Weitang Geriatric Diseases study. Information on tea consumption and HRQOL assessed by the European Quality of Life-5 dimensions (EQ-5D) were collected by questionnaires. We estimated the relationship of tea consumption and the EQ-5D index score using linear regression models and the association between tea consumption and self-reported EQ-5D health problems using logistic regression models.

Results

The EQ-5D index score was higher for habitual tea drinkers than their counterparts. In multivariate linear analyses controlling for socio-demographic conditions, health conditions, and lifestyle habits, the differences in ED-5D index score between individuals with and without tea drinking habits was 0.012 (95% confidence interval, 0.006-0.017). In multivariate logistic analyses, habitual tea drinking was inversely associated with reporting of problems in EQ-5D dimensions mobility (odds ration [OR], 0.44; 95% CI: 0.23-0.84); pain/discomfort (OR, 0.74; 95% CI: 0.61-0.90); and anxiety/depression (OR, 0.60; 95% CI: 0.38-0.97). These associations were more evident for black or oolong tea than green tea.

Conclusion

Habitual tea consumption was associated with better HRQOL in older adults.
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10.

Purpose

Blood donors are considered to be one of the healthiest populations, but relatively little is known about their perceived quality of life. The objective was to examine HRQOL in donors infected with HIV, HBV, HCV or HTLV and a comparison group.

Methods

Donors with confirmed viral infection (cases) and donors who tested false-positive (controls) participated in a multicenter study of US blood donors (2010–2013), funded by the National Heart, Lung and Blood Institute (NHLBI). HRQOL was measured by the EuroQol Five Dimension (EQ-5D) instrument and EQ-5D visual analogue scale (VAS). The lower 25th ‰ of EQ-5D index or VAS score of controls was defined as a “lower HRQOL.”

Results

A total of 1574 controls completed the HRQOL assessment with a mean EQ-5D index of 0.94 (SD = 0.10) and EQ-VAS of 87.6 (SD = 10.6). Mean EQ-5D index for 192 HIV-, 315 HCV- and 195 HTLV-positive donors were significantly lower than the controls (0.86, 0.83 and 0.87; SD = 0.18, 0.20 and 0.16, respectively, p < 0.001). HBV-positive donors (n = 290) had a similar mean EQ-5D index (0.93, SD = 0.14, p = 0.05) to controls. Anxiety/depression was reported by 34 % of cases, compared with 13 % of controls. In multivariable modeling, the odds of lower HRQOL in HIV, HBV, HCV and HTLV cases were 2.1, 1.6, 2.6 and 2.3 times that of controls, respectively (p < 0.05).

Conclusions

HRQOL reported by blood donors with recent viral infections was relatively high but lower than controls. On average, HRQOL among HCV-positive donors was the lowest and HBV-positive donors reported scores similar to donors without infection.
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11.

Purpose

To assess the effectiveness of a preventive health program and vitamin D status in improving the health-related quality of life (HRQOL) of older residents of Canada.

Design

We analyzed baseline and follow-up data of 2119 volunteers of a community program that promotes healthy lifestyles and encourages vitamin D supplementation. We examined the program effect on each of the five dimensions of the EQ-5D-5L, HRQOL score, and quality-adjusted life years (QALYs) using multivariable regression methods. We further examined the specific contribution of vitamin D status as quantified by serum 25-hydroxyvitamin D (25(OH)D).

Results

Problems with mobility, usual activities, pain/discomfort, and depression/anxiety were reported less during follow-up compared to baseline. On average, participants’ HRQOL had improved by 0.018 units at 6 months and 0.025 units at 1 year of follow-up. Improvements in vitamin D status were independently associated with improvements in HRQOL and in QALYs. As per 25 nmol/L increase in 25(OH)D, there was a 0.002 increase in HRQOL and a 0.001 increase in QALYs.

Conclusions

This study documents the benefits of a real-world preventive health program to HRQOL. It is the first to reveal that improvements in vitamin D status parallel improvements in HRQOL among healthy community dwellers. The study further suggests that the preventive health program and supplementation with vitamin D are cost-effective interventions.
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12.

Background

There is a growing interest in policy making for using utility measures and identifying algorithms to convert disease-specific measures into utilities.

Objectives

To analyse the relationship between EQ-5D, Dermatology Life Quality Index (DLQI) and Psoriasis Area and Severity Index (PASI) in psoriasis. To transform DLQI scores, and key clinical, demographic and health service utilisation variables into utilities.

Methods

A cross-sectional questionnaire survey of 200 consecutive adult patients with moderate to severe psoriasis was carried out in two Hungarian university clinics. The relationship between the outcome measures were analysed with correlations and with the known-groups method. Bivariate and multivariate regression algorithms on EQ-5D scores were formulated.

Results

The mean age of respondents was 51 years (SD = 12.9), 68.5 % were male, and 51.5 % received biological therapy. Median EQ-5D, DLQI, and PASI scores were 0.73, 3.0, and 3.45, respectively. EQ-5D showed a moderate correlation with the DLQI and with the PASI (r s = ?0.48 and ?0.43, p < 0.05). Strong correlation was found between DLQI and PASI (r s = 0.81, p < 0.05). DLQI and PASI discriminated better among groups categorised by the localisation of the lesions than EQ-5D. Presence of psoriasis on the neck and/or décolletage was associated with the greatest health related quality of life (HRQOL) impairment. Ten variables were incorporated in a multivariate algorithm that accounted for 48.8 % of EQ-5D variance (ANOVA p < 0.001).

Conclusions

This study provided the first evidence that patients with visible psoriatic lesions have significantly worse HRQOL compared to those with non-visible lesions, measured not only with DLQI but also with EQ-5D. In addition to demographic and clinical variables, our model included health service utilisation variables related to psoriasis, and explained higher proportion of EQ-5D variance than any previous findings in the literature.
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13.

Objectives

To assess the health-related quality of life (HRQOL) of patients with mucopolysaccharidosis (MPS) and their caregivers and to quantify the disease-related costs from a societal perspective.

Methods

In the context of a multi-country study of rare diseases (BURQOL-RD project), a cross-sectional survey was performed among MPS patients in seven European countries. Data on demographic characteristics, health resource utilization, informal care, and loss of labor productivity were collected. The EQ-5D, Barthel index (BI), and Zarit burden interview (ZBI) questionnaires were used to assess patients’ and their informal caregivers’ quality of life, patients’ functional ability, and caregivers’ burden, respectively.

Results

Altogether, 120 patients (children 62 %, females 40 %) and 66 caregivers completed the questionnaire. Patients’ mean age was 16.5 years and median age at diagnosis was 3 years. Adult patients’ average EQ-5D and EQ VAS scores varied across countries from 0.13 to 0.43 and 30.0 to 62.2, respectively, mean BI was 46.7, and ZBI was 32.7. Mean informal care time was 51.3 h/week. The mean total annual cost per patient (reference year 2012) was €24,520 in Hungary, €25,993 in France, €84,921 in Italy, €94,384 in Spain, and €209,420 in Germany. Costs are also shown to differ between children and adults. Direct costs accounted for most of the costs in all five countries (80, 100, 99, 98, and 93 %, respectively).

Conclusions

MPS patients experience substantial loss of HRQOL and their families take a remarkable part in their care. Although utilization of health and social care resources varies significantly across countries, MPS incurs considerable societal costs in all the countries studied.
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14.

Objective

The aim of this study was to compare health-related quality of life (HRQOL) between type 2 diabetic patients with and without locomotive syndrome, which is a risk for becoming bedridden because of deteriorating locomotive organs.

Subjects and methods

A total of 135 patients with type 2 diabetes mellitus (69.2 ± 10.2 years) were enrolled in this cross-sectional study. HRQOL was evaluated by the Euro-QOL-5 (EQ-5D), and locomotive syndrome was evaluated by “loco-check,” established the Japanese Orthopedic Association. Clinical data, such as anthropometric parameters, blood and urine examination results, blood pressure (BP), drug usage, and psychological distress, were analyzed.

Results

Average HbA1c in all patients was 7.2 ± 1.0 %, and 74 patients (54.8 %) were identified as having the locomotive syndrome. EQ-5D scores were significantly lower in type 2 diabetic patients with locomotive syndrome than in those without locomotive syndrome, even after adjusting for age, sex, body mass index (BMI), and HbA1c. By the multiple regression analysis, locomotive syndrome and age were identified as determinant factors of HRQOL in type 2 diabetic patients.

Conclusion

In type 2 diabetic patients, lower HRQOL was a characteristic feature of subjects with locomotive syndrome, even after adjusting for confounding factors. Prevention or management of locomotive syndrome may be beneficial for improving HRQOL in type 2 diabetic patients.
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15.

Purpose

To determine whether insurance status modifies the association between injuries and health-related quality of life (HRQOL) in a nationally representative sample of US adults.

Methods

This is a longitudinal, observational study using the pooled 2000–2006 Medical Expenditure Panel Survey (MEPS). A total of 50,225 adults (age ≥18) with or without injuries were included in the study. HRQOL was evaluated using the EuroQoL Health Index (EQ-5D), visual analog scale (VAS), and the SF-12 physical component score (PCS) and mental component score (MCS). A categorical variable of injury-insurance combinations was created (not injured-privately insured, not injured-publically insured, not injured-uninsured, injured-privately insured, injured-publically insured, or injured-uninsured) and was included in the linear regression models. Adjustment covariates included age, gender, education, race, diabetes, hypertension, and baseline self-reported health. Healthcare utilization was also examined among the study population by injury status and across insurance groups.

Results

Seven hundred and ten individuals reported injuries. Adjusted analyses showed that injured individuals with public insurance had lower EQ-5D (?0.25), VAS (?11.4), PCS (?8.5), and MCS (?4.9) than the privately insured controls, while uninsured had EQ-5D, VAS, PCS and MCS that were, respectively, ?0.12, ?7.2, ?2.6 and ?4.1 relative to privately insured controls. With the exception of hospital discharges, healthcare utilization among uninsured individuals was lower than those with public or private insurance.

Conclusions

We found injured individuals to have lower HRQOL than those without injuries, and this effect was exacerbated by insurance status. These findings call for interventions aimed to narrow the outcome disparity among injured in the US.
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16.

Purpose

Health-related quality of life (HRQoL) of pulmonary TB patients has not been assessed in Pakistan. We assessed self-reported HRQoL of pulmonary TB patients in Karachi, Pakistan utilizing the EQ-5D and EQ-VAS prior to, during, and after completion of TB treatment.

Methods

We enrolled 226 pulmonary TB patients in a longitudinal cohort study. Health-utility scores were estimated by the EQ-5D five dimensions and the EQ-Visual Analogue Scale (VAS) at baseline (month 0) and each monthly follow-up visit until treatment completion at month 6. Repeated-measures ANOVA was used to investigate effect of time into treatment on EQ-5D and EQ-VAS scores.

Results

EQ-5D health utility and EQ-VAS scores increase with treatment progression. For the enrolled TB patients, the mean EQ-5D utility scores more than doubled from 0.43 to 0.88, p?<?.001, effect size η2?=?0.40 from treatment initiation to treatment completion.

Conclusion

Perceived HRQoL of TB patients improves with treatment progression. This can inform targeted treatment plans as well as TB policy and funding for high-burden countries.
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17.

Purpose

We aim to describe the impact of diabetic retinopathy (DR) on health-related quality of life (HRQOL) among community-dwelling Chinese adults who had been previously diagnosed with type 2 diabetes mellitus (T2DM).

Methods

A community-based survey including 913 patients with T2DM was conducted in Suzhou, China. Retinopathy lesions were graded according to the Airlie House classification system of the Early Treatment Diabetic Retinopathy Study. The HRQOL was measured by the Chinese version of the EuroQol Group’s five-level EuroQol five-dimensional questionnaire (EQ-5D-5L). A Gamma distribution with log link was incorporated into linear regression models to assess the associations between DR and EQ-5D-5L health utility score.

Results

The mean EQ-5D-5L index scores were 0.971?±?0.082 among individuals with unilateral DR and 0.970?±?0.145 among those with bilateral DR, which were lower compared with those without DR (0.986?±?0.045, P?=?0.02). In multivariate analysis adjusting for confounders, people with bilateral DR reported lower the EQ-5D index scores compared with those without DR. The presence of DR was significantly associated with problems in usual activities (odds ratio [OR]?=?0.16, P?=?0.02, comparing participants with unilateral vs. no DR; OR?=?0.11; P?=?0.01, comparing participants with bilateral vs. no DR). No significant variations in EQ-5D-5L index scores as well as different domains of health problems between individuals with unilateral and bilateral DR were observed (P?>?0.05).

Conclusion

Chinese T2DM patients with bilateral DR tend to report lower HRQOL scores compared with those without DR, especially in health problems associated with usual activities.
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18.

Background

Accurate assessment of preference-based health-related quality of life is important in determining the value of asthma interventions.

Objective

To examine the sensitivity and responsiveness of the EQ-5D and the AQL-5D to differences in asthma control measured by the Asthma Control Questionnaire (ACQ-5).

Methods

The Observational Study of Asthma Control and Outcomes was a prospective survey of persistent asthma patients ≥12 years old in Kaiser Colorado. Patients received a survey three times in 1 year, including the ACQ-5, AQL-5D and EQ-5D-3L (including VAS). Censored Least Absolute Deviations (CLAD) and logistic regression were used, controlling for sociodemographics and smoking.

Results

There were 6666 completed surveys (1799 individuals completed all three survey waves). After controlling for covariates, each one-point increase in ACQ-5 was associated with a decrease of 0.066, 0.058, 0.074 and 6.12 in EQ-5D(US), EQ-5D(UK), AQL-5D and VAS scores. Uncontrolled asthma (ACQ-5 > 1.5) was associated with a decrease of 0.15, 0.17, 0.11 and 10, respectively (vs. ACQ ≤ 1.5). AQL-5D scores were statistically significantly different across categories of ACQ-5 scores of 0.5 (the minimum clinically important difference [MCID]), while EQ-5D scores were not significant across most categories. The AQL-5D appeared more robust to changes in control over time (responsiveness) compared to EQ-5D-3L.

Conclusion

The AQL-5D appears more responsive to changes in asthma control over time and more sensitive to detecting differences corresponding to the ACQ-5 MCID than the EQ-5D-3L. Using the EQ-5D-3L without an asthma-specific measure such as the AQL-5D may miss clinically important changes in asthma control.
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19.

Background

Conventionally, models used for health state valuation data have been parametric. Recently, a number of researchers have investigated the use of non-parametric Bayesian methods in this area.

Objectives

In this paper, we present a non-parametric Bayesian model to estimate a preference-based index for a five-dimensional health state classification, namely EQ-5D.

Methods

A sample of 2997 members of the UK general population valued 43 health states selected from a total of 243 health states defined by the EQ-5D using time trade-off technique. Findings from non-parametric modelling are reported in this paper and compared to previously used parametric estimations. The impact of respondent characteristics on health state valuations is also reported.

Results

The non-parametric models were found to be better at predicting scores in populations with different distributions of characteristics than observed in the survey sample. Additionally, non-parametric models were found to be better at allowing for the impact of respondent characteristics to vary by health state. The results show an important age effect with sex having some effect.

Conclusion

The non-parametric Bayesian models provide more realistic and better utility estimates from the EQ-5D than previously used parametric models have done. Furthermore, the model is more flexible in estimating the impact of covariates.
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20.

Purpose

In the original SF-6D valuation study, the analytical design inherited conventions that detrimentally affected its ability to predict values on a quality-adjusted life year (QALY) scale. Our objective is to estimate UK values for SF-6D states using the original data and multi-attribute utility (MAU) regression after addressing its limitations and to compare the revised SF-6D and EQ-5D value predictions.

Methods

Using the unaltered data (611 respondents, 3503 SG responses), the parameters of the original MAU model were re-estimated under three alternative error specifications, known as the instant, episodic, and angular random utility models. Value predictions on a QALY scale were compared to EQ-5D3L predictions using the 1996 Health Survey for England.

Results

Contrary to the original results, the revised SF-6D value predictions range below 0 QALYs (i.e., worse than death) and agree largely with EQ-5D predictions after adjusting for scale. Although a QALY is defined as a year in optimal health, the SF-6D sets a higher standard for optimal health than the EQ-5D-3L; therefore, it has larger units on a QALY scale by construction (20.9 % more).

Conclusions

Much of the debate in health valuation has focused on differences between preference elicitation tasks, sampling, and instruments. After correcting errant econometric practices and adjusting for differences in QALY scale between the EQ-5D and SF-6D values, the revised predictions demonstrate convergent validity, making them more suitable for UK economic evaluations compared to original estimates.
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