Health and Quality of Life Outcomes最新文献

Background: Individuals with heart failure (HF) experience various symptoms making both diagnosis and disease burden estimates challenging. While HF-specific patient-reported outcome measures (PROMs) are widely used, their focus on clinical cohorts limits their generalizability. Preference-based measures like the EQ-5D enable standardized health-related quality of life (HRQoL) comparisons across conditions, supporting resource allocation decisions. The CDC's Healthy Days (HD) Survey-a simple two-question tool that can be mapped to the EQ-5D-offers a broader approach to tracking HRQoL but remains underutilized in HF populations.

Methods: Using a nationally representative U.S. sample, we mapped HD Survey responses to EQ-5D utility scores to compare HRQoL between individuals with and without HF and examined changes in HRQoL over time. We assessed whether HD-derived scores align with HF-specific utility measures to support population-level health monitoring.

Results: Individuals with HF report significantly more physically unhealthy days (8.46 vs. 3.42) and mentally unhealthy days (5.42 vs. 3.86) compared to those without HF. HF respondents are, on average, 20 years older than those without HF, consistent with HF's prevalence in older adults. The likelihood of an HF diagnosis is similar for men and women but higher among non-Hispanic whites and blacks than Hispanics and other races. Those with HF are more likely to have health insurance. Adjusting for age, sex, race, and insurance, mean EQ-5D utility scores for individuals with and without HF are 0.785 (95% CI: 0.714-0.825) and 0.840 (95% CI: 0.827-0.851), respectively. Utility scores for HF patients remain significantly lower than those without HF up to 10 years post-diagnosis.

Conclusion: HF reduces HRQoL by 6.55%, surpassing the clinically significant threshold of a 1-2% decrement. These findings highlight the potential of the HD Survey to inform public health monitoring and underscore the need for tailored interventions to address HRQoL deficits in HF populations.

Background: Patient-reported outcomes (PROs) in systemic rheumatic diseases (SRDs) are in the forefront of clinical research. However, a comprehensive evaluation of PROs in pivotal trials supporting SRD drug approval is lacking.

Objective: This study aims to systematically characterize the use of PROs in pivotal trials supporting the US Food and Drug Administration (FDA) approval of SRDs treatments and to assess the quality of reporting.

Methods: We reviewed the pivotal trials supporting the approval of SRD indications by FDA since July 2013 to assess the use of PROs, including specific PRO measures (PROMs) and types of endpoints designated. Quality of PRO reporting was assessed according to a modified ISOQoL criteria.

Results: From July 1st, 2013, to June 30th, 2024, the FDA approved 43 new SRD indications based on 67 pivotal trials, with 58 trials included in the final analysis. PROs served as multiple types of endpoints in most trials. All 58 reviewed trials utilized PROs as secondary or exploratory endpoints. The numbers of trials that employed PROs as components of primary endpoints, co-primary endpoints, and key secondary endpoints, were 47(81.0%), 4(6.9%), 45(77.6%), respectively. Notably, the inclusion of PROs as components of composite primary endpoints or co-primary endpoints (100% vs. 8.3%, P < 0.001) and key secondary endpoints (93.5% vs. 16.7%, P < 0.001) were significantly higher in inflammatory arthritis compared to other SRDs. Regarding PROM types, 37 trials (63.8%) reported both generic and disease-specific PROMs, covering a broad range of domains. Quality of PRO reporting, influenced by disease type and the presence of additional PRO reports, was moderate to poor in 45 trials (81.8%). Key reporting elements, such as the PRO hypothesis, mode of PROMs completion, and extent and reasons for missing PRO data, were documented in fewer than 30% of the trials.

Conclusion: PROs significantly impact SRDs drug approval decisions, especially for inflammatory arthritis. However, the overall quality of PRO reporting in pivotal trials of SRDs is suboptimal and needs improvement. Our study provides a comprehensive summary of PRO application in SRDs trials, highlighting the need for strengthening PRO utilization in non-arthritis SRDs and improving PRO reporting quality in future studies.

Background: Entering the age of 80 or 100, individuals' functional decline and the need for assistance in daily activities increase dramatically, resulting in an inevitable increase in unmet long-term care (LTC) needs. Understanding unmet LTC needs in late life and their association with quality of life is essential for effective health planning and resource allocation. We aim to estimate the prevalence of unmet needs for LTC and the association of unmet needs for LTC and Health-related Quality of life (HRQOL), among the Chinese oldest-old population.

Methods: Data were drawn from the 2017 China Hainan Centenarian Cohort Study. All centenarians and a representative sample of individuals aged between 80 and 99 years old in Hainan province, China were included. Self-perceived unmet LTC needs were reported by the respondents. EQ-5D score was calculated from EQ-5D-3L questionnaire to measure HRQOL in this study. We conducted Tobit regression and the Ordered Probit Model to examine the cross-sectional associations between unmet needs for LTC and HRQOL.

Results: 1,444 respondents (mean age 95.75 years [SD 9.13]) were included. The prevalence of unmet LTC needs was 32.69%, and it was higher in rural residents and people with economic deprivation. The results showed that oldest-old individuals with unmet needs for LTC reported lower QALY scores (β=-0.04, p < 0.01). In addition, Unmet LTC needs were significantly correlated with poorer outcomes in mobility (β = 0.18, p < 0.05), self-care (β = 0.19, p < 0.05), pain or discomfort (β = 0.27, p < 0.01), and anxiety or depression (β = 0.09, p < 0.01).

Conclusions: The prevalence of unmet LTC needs was higher in China than its counterpart in high-income countries, especially among those with socio-economic deprivation. Individuals with unmet LTC needs experience lower quality-adjusted life years (QALYs) and higher levels of disabilities across multiple dimensions of health, including mobility, usual activities, self-care, pain/discomfort and depression/anxiety. These findings underscore the importance of addressing unmet LTC needs to improve the overall health outcomes and quality of life for the oldest-old population.

Background: Children of female sex workers (FSWs) in Bangladesh grow up in a challenging socio-economic environment characterized by parental separation, substance abuse, alcoholism, and limited access to healthcare, including oral health services. This study aimed to assess the oral health-related quality of life (OHRQoL) and its associative factors among these children.

Materials and methods: A cross-sectional study was conducted between March 2023 and February 2024 with a sample of 180 FSW mothers/institutional caregivers and their school-going children, aged 7 to 17. OHRQoL was assessed using the CPQ_8 - 10 (Child Perception Questionnaire) for children aged 7 to 11, and the OIDP (Oral Impacts on Daily Performances) for adolescents aged 12 to 17. The Decayed, Missing and Filled Teeth (DMFT/dmft) index was used to assess dental caries, while the gingival index evaluated gingival health. The plaque index and calculus index were employed to assess plaque and calculus levels, respectively.

Results: Among children aged 7 to 11, oral symptoms (Mean = 5.36, SD = 3.72) and functional limitations (Mean = 4.57, SD = 4.42) were the most affected parameters. For children aged 12 to 17, 78.2% reported oral impacts on their daily performances (Mean = 6.50, SD = 6.84). Caries status (β = 0.361, p = 0.001) and place of residence (β = 0.329, p = 0.032) were significantly associated with higher CPQ_8 - 10 scores. A higher OIDP score was associated with gingivitis (β = 0.265, p = 0.035).

Conclusion: The study reveals that children aged 7 to 11 in Daulatdia with caries had significantly poorer OHRQoL, while gingivitis was associated with a higher OIDP score in children aged 12 to 17. OHRQoL evaluation is vital in determining the effectiveness of therapeutic and preventive measures aimed at improving the oral health of this vulnerable population. REGISTRY AND REGISTRATION NO.

Of the study: The Institutional Review Board /Ethics Review Committee (IRB/ERC) of North South University reviewed and approved this study(2023/OR-NSU/IRB/0204).

Objective: This study aims to translate and validate the CarerQol in a sample of informal caregivers in China.

Method: The CarerQol was translated following a standard process. Validation data was collected from multiple sources, including hospitals, patient associations, and community health centers between September 2023 and December 2024 in China. Caregivers of individuals with disability or long-term care needs were recruited. The psychometric properties of the CarerQol-7D were evaluated through ceiling and floor effects, factorial structure, convergent and divergent validity, known-groups validity, and test-retest reliability.

Results: A total of 324 participants completed the survey and provided validated responses. The mean CarerQol level sum score was 12.8 (SD = 2.9). The CarerQol-7D showed no ceiling or floor effects at either dimension or scale levels. Test-retest reliability of the CarerQol-7D was satisfactory, as supported by ICC and Gwet's AC1 measures. Correlation analysis confirmed all hypothesized pairs of dimensions and level sum scores/utility scores between CarerQol-7D, SF-6Dv2, and ICECAP-A. The CarerQol-7D successfully distinguished between risk groups through ANOVA testing. Confirmatory factor analysis revealed that a one-factor model of the CarerQol-7D fit the data.

Conclusion: This study provides the Chinese version of the CarerQol and confirms its sound psychometric properties for informal caregivers in China. These findings demonstrate the CarerQol's value in cost-effectiveness analyses of caregiving interventions and strategies.

Background: Evaluating health-related quality of life (HRQoL) in the general population is essential for establishing benchmarks for health outcome assessments. This study aimed to generate population norms for the EQ-5D-5L dimensions, EQ-VAS (EuroQol-Visual Analogue Scale) scores, and EQ-5D-5L index scores in Iran, stratified by sex and age.

Methods: Data for this cross-sectional study were gathered through face-to-face interviews with 3,518 adults from the general population across nine provinces in Iran, employing a multistage sampling approach. Respondents assessed their own health state across the EQ-5D-5L dimensions-mobility, self-care, usual activities, pain/discomfort, and anxiety/depression-along with the EQ-VAS to evaluate their overall health. The EQ-5D-5L score was calculated using the value set derived from the Iranian general population. Dimension scores and index values (EQ-5D-5L index and EQ-VAS score) were analyzed using multiple logistic regression and generalized linear model (GLM), respectively.

Results: The estimated mean ± standard deviation [SD] of EQ-5D-5L index for the general population of Iran was 0.789 ± 0.258, while the EQ-VAS score was estimated at 74.34 ± 18.67. Among the study participants, 35.8% reported being in the best health state (11111), while the remaining 64.2% experienced problems in at least one of the five dimensions. The most commonly reported problems were related to anxiety/depression (49.2%), followed by pain/discomfort (45.3%). Regression analyses revealed that females reported significantly more problems across the five dimensions of the EQ-5D-5L and had lower EQ-5D-5L index and EQ-VAS scores compared to males. Anxiety/depression were more prevalent among younger individuals, while problems in other dimensions tended to increase with age.

Conclusions: The findings indicate that nearly two-thirds of respondents experienced problems in at least one dimension, with anxiety/depression being the most prevalent, particularly among younger individuals. Females reported lower utility scores and more problems across all dimensions in all age groups. To effectively improve the health status of the Iranian population and ensure optimal resource allocation, it is vital to develop and implement targeted interventions that specifically address the needs highlighted in this study.

Background: Hip fractures are a significant public health concern due to increasing numbers, high mortality and negative impact on health-related quality of life (HRQoL). Socioeconomic position (SEP) affects various health outcomes, but the specific impact on HRQoL and satisfaction after hip fracture remains underexplored. This study assesses whether education and household income influence patient-reported outcome measures (PROMs) after hip fractures, measured by three visual analog scales: EQ-VAS, pain-VAS, and satisfaction-VAS.

Methods: This was a nationwide retrospective cohort study using linked data from the Norwegian Hip Fracture Register and Statistics Norway. PROMs assessed at 4, 12, and 36 months postoperatively in 35,206 hip fracture patients from 2015 to 2018 were included. The SEP data included household income and education levels. Covariance analyses were conducted to evaluate differences in mean VAS scores for general health (EQ-VAS), pain from the operated hip (Pain-VAS), and satisfaction with the result of the operation (Satisfaction-VAS). Analyses adjusted for age, sex, vital status, cognitive impairment, treatment type, and education or income when not used as independent variable.

Results: The study included 23,649 women (67.2%) and 11,557 men (32.8%) with median age 83 years. Lower education was linked to worse EQ-VAS and Pain-VAS scores at all follow-ups and to lower Satisfaction-VAS at 12 and 36 months in both unadjusted and adjusted analyses. Lowest level of income had significant lower EQ-VAS at all follow-ups, lower Pain-VAS at 12 months, and lower Satisfaction-VAS at 4 months. There were increasing differences in mean VAS-scores during follow-up. At 36 months the adjusted differences in mean EQ-VAS between highest and lowest level of income was - 2,51 (-4.04 -0.99). Differences across education levels were even stronger associated; -3.58 (-5.19 to -1.98). Mean differences in Pain-VAS between medium and low education compared to high were 4.30 (2.91 to 5.69) and 5.58 (4.08 to 7.08), respectively. Lower levels of education also had significant negative differences in Satisfaction-VAS at 36 months follow-up -4.06(-5.86 to -2.26).

Conclusions: Lower education and income were significantly associated with worse HRQoL and satisfaction after hip fracture. The clinical relevance of these findings warrants further investigation. Addressing SEP disparities should be integral to hip-fracture care strategies aiming to improve postoperative outcomes.

Objective: The Audit of Diabetes-Dependent Quality of Life (ADDQoL) is a widely used instrument for assessing quality of life in Type 2 Diabetes Mellitus (T2DM). However, it does not directly yield health utility values essential for economic evaluations. This study developed mapping algorithms to predict EQ-5D-5L and SF-6Dv2 utility values from ADDQoL scores in T2DM patients in China.

Methods: Cross-sectional data from 800 T2DM patients in China, stratified by age, sex, and geographical region, were divided into development (80%) and validation (20%) groups. Pearson correlation analyses were conducted to assess the conceptual overlap between ADDQoL and the EQ-5D-5L and SF-6Dv2. Six predictor sets and six regression methods were explored to map ADDQoL scores to EQ-5D-5L and SF-6Dv2 utility values, respectively. Model performance was evaluated using mean absolute error (MAE), root mean square error (RMSE), and intraclass correlation coefficient (ICC).

Results: For the development group, the mean (SD) ADDQoL Average Weighted Impact (AWI) score was - 2.426 (1.052), and the mean (SD) utility values for EQ-5D-5L and SF-6Dv2 were 0.928 (0.092) and 0.791 (0.133), respectively. Among all 36 alternative mapping models each for EQ-5D-5L and SF-6Dv2, the best performance was consistently observed in the two-part models that included the ADDQoL AWI, the first overview item, and their squared terms. For the algorithm mapping to EQ-5D-5L utility values, it achieved a MAE of 0.067, a RMSE of 0.095, and an ICC of 0.414; For the algorithm mapping to SF-6Dv2 utility values, the corresponding metrics were an MAE of 0.099, an RMSE of 0.120, and an ICC of 0.517.

Conclusions: This study provides a mapping framework to estimate EQ-5D-5L and SF-6Dv2 utility values from ADDQoL scores. These algorithms could be used to support economic evaluations, specifically tailored for Chinese T2DM populations.

Background: The Short-Form 36 (SF-36), a widely used patient-reported outcome (PRO), is a questionnaire completed by patients measuring health outcomes in clinical trials. The PRO scores can be discrete, bounded, and skewed. Various statistical methods have been suggested to analyse PRO data, but their results may not be presented on the same scale as the original score, making it difficult to interpret and compare different approaches. This study aims to unify and compare the estimates from different statistical methods for analysing PROs, particularly the SF-36, in randomised controlled trials (RCTs), using standardised effect size (SES) summary measure.

Methods: SF-36 outcomes were analysed using ten statistical methods: multiple linear regression (MLR), median regression (Median), Tobit regression (Tobit), censored absolute least deviation regression (CLAD), beta-binomial regression (BB), binomial-logit-normal regression (BLN), ordered logit model (OL), ordered probit model (OP), fractional logistic regression (Frac), and beta regression (BR). Each SF-36 domain score at a specific follow-up in three clinical trials was analysed. The estimated treatment coefficients and SESs were generated, compared, and interpreted. Model fit was evaluated using the Akaike information criterion.

Results: Estimated treatment coefficients from the untransformed scale-based methods (Tobit, Median, & CLAD) deviated from MLR, whereas the SESs from Tobit produced almost identical values. Transformed scale-based methods (OL, OP, BB, BLN, Frac, and BR) shared a similar pattern, except that OL generated higher absolute coefficients and BLN produced higher SESs than other methods. The SESs from Tobit, BB, OP, and Frac had better agreement against MLR than other included methods.

Conclusions: The SES is a simple method to unify and compare estimates produced from various statistical methods on different scales. As these methods did not produce identical SES values, it is crucial to comprehensively understand and carefully select appropriate statistical methods, especially for analysing PROs like SF-36, to avoid drawing wrong estimates and conclusions using clinical trial data. Future research will focus on simulation analysis to compare the estimation accuracy and robustness of these methods.

Background: In many settings, Tuberculosis (TB) represents a catastrophic life event that substantially impairs a person's Health-Related Quality of Life (HRQoL). We aimed to measure HRQoL among people with TB in Viet Nam at initiation and throughout treatment.

Methods: This study took place in four provinces from Oct-2020 to Sep-2022. Persons initiated on TB treatment were consecutively recruited across three pathways to access care: passive case finding (NTP); active case finding (ACF); and private sector engagement (PPM). We conducted the EuroQol-5-Dimension-5-Level (EQ-5D-5L) survey during the intensive, continuation, and post-treatment phase. We described participant characteristics, assessed the survey's psychometric properties, and calculated utility indexes using a Vietnamese value set. We reported these alongside visual analog scale (EQ-VAS) scores and EQ-5D-5L dimensions by treatment stage, care pathway and other participant characteristics. Mixed-effect Tobit models were fitted to identify relevant associations with HRQoL, which we compared to general population benchmarks.

Results: We recruited 585 participants (23.6% female) with a median age of 51 years. EQ-5D-5L dimensions at baseline showed that 53.8% experienced pain/discomfort and 35.0% felt anxiety/depression, while 33.8%, 30.4%, and 9.6% reported problems with carrying out usual activities, mobility, and self-care, respectively. The mean utility index was 0.83 (95% confidence interval: [0.82, 0.85]) and mean EQ-VAS was 67.1 (95%CI: [65.6, 68.6]). Post-treatment, HRQoL improved significantly on all dimensions and composite measures. While utility indexes were at parity with general population benchmarks (0.90; 95%CI: [0.89, 0.92] vs. 0.91), self-reported EQ-VAS scores remained significantly lower (79.4; 95%CI: [78.1, 80.6] vs. 87.4). HRQoL was higher at baseline in the ACF versus the NTP cohorts on utility index (0.87 vs. 0.82; p = 0.003) and EQ-VAS score (70.4 vs. 65.5; p = 0.015). The EQ-5D-5L tool demonstrated moderate to high validity on Cronbach's alpha (0.75 ≤ α ≤ 0.84) and Spearman's rho (0.4679 ≤ ρ₀ ≤ 0.5651) across treatment stages and various known groups.

Conclusion: TB significantly impairs HRQoL among affected Vietnamese people. While treatment partially remedies these impairments, they may persist post-TB. Hence, physical, psychological and social rehabilitation during and after therapy should receive more attention. We found evidence that ACF may mitigate TB-related declines in HRQoL, but tailored studies are needed to substantiate these findings.