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Deriving the PedsUtil health state classification system to measure health utilities for pediatric populations based on the PedsQL: a confirmatory factor analysis. 根据 PedsQL 得出 PedsUtil 健康状况分类系统,以衡量儿科人群的健康效用:确证因子分析。
IF 3.2 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-10-08 DOI: 10.1186/s12955-024-02300-8
Ellen Kim DeLuca, Kim Dalziel, Eve Wittenberg, Angela M Rose, Lisa A Prosser

Background: An important methodological challenge in conducting pediatric economic evaluations is estimating the preference-based health-related quality of life (HRQoL) of children. Current methods are highly variable and there is no single instrument available to value HRQoL consistently across multiple pediatric age groups. The Pediatric Quality of Life Inventory (PedsQL) is a non-preference-based generic HRQoL instrument validated for children 2-18 years, but it cannot be directly used in economic evaluations. The aim of this study was to establish the core dimension structure of the PedsUtil health state classification system using confirmatory factor analysis, which is the first step of deriving a preference-based measure of HRQoL based on the PedsQL.

Methods: Four competing dimension structures of the PedsUtil health state classification system were developed based on published literature and expert opinion. Using data from the Longitudinal Study of Australian Children (LSAC) (n = 45,207), the 4 dimension structures were evaluated using the robust weighted least squares estimation method. The analyses were stratified by 2-year age intervals (from 2 to 17 years) to reflect the study design of the LSAC, as well as special healthcare needs status of the child. Model fit was evaluated by examining standardized factor loadings and various fit indices including the comparative fit index (CFI), Tucker-Lewis Index (TLI), and the root mean square error of approximation (RMSEA). Modification indices and residual correlations were examined to re-specify the models to improve model fit when necessary.

Results: The findings supported a 7-dimension structure (i.e., Physical Functioning, Pain, Fatigue, Emotional Functioning, Social Functioning, School Functioning, and School Absence) of the PedsUtil health state classification system. The 7-dimension model exhibited adequate fit across subgroups with CFI values that ranged from 0.929 to 0.954, TLI values from 0.916 to 0.946, and RMSEA values from 0.058 to 0.102.

Conclusions: This study established the core dimension structure of the PedsUtil health state classification system using confirmatory factor analysis. The 7-dimension structure was found to be applicable across diverse pediatric populations. Research is currently ongoing to select the most representative item within each dimension of the PedsUtil health state classification system and valuation surveys will be fielded to estimate the PedsUtil scoring system.

背景:在进行儿科经济评估时,一个重要的方法论挑战是估算儿童基于偏好的健康相关生活质量(HRQoL)。目前的方法千差万别,而且没有一种工具可以对多个儿科年龄组的儿童进行一致的 HRQoL 估值。儿科生活质量量表(PedsQL)是一种非基于偏好的通用 HRQoL 工具,已在 2-18 岁儿童中得到验证,但不能直接用于经济评估。本研究的目的是利用确证因子分析建立 PedsUtil 健康状况分类系统的核心维度结构,这是根据 PedsQL 得出基于偏好的 HRQoL 测量方法的第一步:根据已发表的文献和专家意见,开发了 PedsUtil 健康状况分类系统的四个竞争维度结构。利用澳大利亚儿童纵向研究(LSAC)的数据(n = 45,207),采用稳健加权最小二乘法估算方法对 4 个维度结构进行了评估。分析按 2 岁年龄间隔(从 2 岁到 17 岁)进行分层,以反映 LSAC 的研究设计以及儿童的特殊医疗保健需求状况。通过检查标准化因子负荷和各种拟合指数(包括比较拟合指数(CFI)、塔克-刘易斯指数(TLI)和均方根近似误差(RMSEA))来评估模型的拟合度。对修正指数和残差相关性进行了检验,以便在必要时对模型进行重新规范,从而提高模型的拟合度:研究结果支持 PedsUtil 健康状况分类系统的 7 维结构(即身体功能、疼痛、疲劳、情绪功能、社会功能、学校功能和缺课)。7维模型在不同亚组中表现出充分的拟合度,CFI值介于0.929至0.954之间,TLI值介于0.916至0.946之间,RMSEA值介于0.058至0.102之间:本研究通过确证因子分析建立了 PedsUtil 健康状况分类系统的核心维度结构。研究发现,7 个维度的结构适用于不同的儿科人群。目前正在进行研究,以在 PedsUtil 健康状况分类系统的每个维度中选择最具代表性的项目,并将开展估值调查,以评估 PedsUtil 评分系统。
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引用次数: 0
The subjective wellbeing of people living with Multiple Sclerosis in Australia: insights from the Personal Wellbeing Index. 澳大利亚多发性硬化症患者的主观幸福感:个人幸福指数的启示。
IF 3.2 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-30 DOI: 10.1186/s12955-024-02278-3
Glen J Henson, Ingrid van der Mei, Bruce V Taylor, Suzi B Claflin, Andrew J Palmer, Julie A Campbell, Gang Chen

Objectives: Subjective wellbeing has been defined as an individual's personal appraisal of their quality of life. Subjective wellbeing is associated with positive health behaviours and improved coping abilities. This study aimed to investigate the subjective wellbeing of people living with multiple sclerosis (MS), using the novel Personal Wellbeing Index, and make comparisons with the general population.

Methods: Cross-sectional data was obtained from the Australian Multiple Sclerosis Longitudinal Study and the How Is Your Life Australian general population study in August-October 2020. Subjective wellbeing was measured as life satisfaction using the Personal Wellbeing Index. This instrument measures life satisfaction globally and in seven life domains, allowing the importance of domain-specific life satisfaction to be explored. Descriptive and multivariable regression analyses were conducted.

Results: One thousand six hundred eighty-three MS and 1,021 general population participants entered the study (mean age 52.4 and 58.6; female 79.9% and 52.4%, respectively). For people living with MS the most important life domains were standard of living and achieving in life. The domain of personal health was more influential for people living with MS (p < 0.01) than the general population. The life domains most susceptible to MS-related disability were personal health, achieving in life, and community connectedness (p < 0.01 for these domains).

Conclusion: Personal health and achieving in life are key domains through which the subjective wellbeing of people living with MS is modified. This study recommends the development of interventions to support healthy perceptions of illness and continued employment as paramount in improving the subjective wellbeing of people living with MS.

目的:主观幸福感被定义为个人对其生活质量的评价。主观幸福感与积极的健康行为和应对能力的提高有关。本研究旨在使用新型个人幸福指数调查多发性硬化症(MS)患者的主观幸福感,并与普通人群进行比较:2020年8月至10月,从澳大利亚多发性硬化症纵向研究和 "您的生活如何 "澳大利亚普通人群研究中获得了横断面数据。主观幸福感通过个人幸福指数(Personal Wellbeing Index)来衡量生活满意度。该工具测量全球和七个生活领域的生活满意度,从而探讨特定领域生活满意度的重要性。研究进行了描述性和多变量回归分析:共有 1683 名多发性硬化症患者和 1021 名普通人群参与研究(平均年龄分别为 52.4 岁和 58.6 岁;女性分别占 79.9% 和 52.4%)。对于多发性硬化症患者来说,最重要的生活领域是生活水平和生活成就。个人健康领域对多发性硬化症患者的影响更大(p 结论:个人健康和实现人生价值是多发性硬化症患者最重要的生活领域:个人健康和生活成就是改变多发性硬化症患者主观幸福感的关键领域。本研究建议制定干预措施,支持多发性硬化症患者对疾病和持续就业的健康认知,这对改善多发性硬化症患者的主观幸福感至关重要。
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引用次数: 0
Cross-cultural adaptation and content validation of the Singapore English version of EQ-5D-Y: a qualitative study. 新加坡英语版 EQ-5D-Y 的跨文化改编和内容验证:一项定性研究。
IF 3.2 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-27 DOI: 10.1186/s12955-024-02290-7
Rachel Lee-Yin Tan, Zhi Min Ng, Le Ann Chen, Michael Herdman, Nan Luo

Background: The EQ-5D-Y is a generic preference-weighted measure for children and adolescents which was developed within Europe. Two versions exist, the EQ-5D-Y-3L (Y-3L) and EQ-5D-Y-5L (Y-5L). This study aimed to cross-culturally adapt the Y-3L and Y-5L for use in Singapore and to assess the content validity, specifically, the relevance and comprehensiveness of the EQ-5D-Y descriptive system (DS) in Asia.

Methods: To culturally adapt the instruments, an expert panel consisting of paediatricians and primary school educators were consulted. Modifications suggested by the expert panel were tested via cognitive debriefing interviews with children aged 8-12 in Singapore. To assess the content validity of the EQ-5D-Y DS, interviews were conducted with both healthy (n = 8) and ill children (n = 6) aged 8-15. In the interviews, children discussed their experience with poor health and commented on the comprehensiveness and relevance of the EQ-5D-Y DS.

Results: The cross-cultural adaptation process led to minor modifications to the UK English Y-3L and Y-5L versions, including using phrases familiar to the local children and adding examples to facilitate understanding. The five health dimensions in the EQ-5D-Y DS were spontaneously elicited when children discussed their experience with poor health. All health dimensions related to poor health elicited from the interviews fell into three broad categories: physical health (e.g. Appetite, Mobility, and Sleep), mental well-being (e.g. Annoyed/Frustrated and Scared/Worried), and social relationships (e.g. Family and Friends). The EQ-5D-Y DS was generally found to be relevant and comprehensive, although some health dimensions that may be relevant to the local population (Social relationship and Appetite) were not covered.

Conclusions: The UK English EQ-5D-Y instruments were adapted to produce the Singapore English EQ-5D-Y instrument that were comprehensible to local children as young as 8 years old. The EQ-5D-Y DS was generally relevant and comprehensive to measure poor health of local children. Future studies should ascertain the benefits of adding bolt-on items related to social relationships and appetite to the EQ-5D-Y DS.

背景EQ-5D-Y 是一种针对儿童和青少年的通用偏好加权测量方法,由欧洲开发。目前有两个版本:EQ-5D-Y-3L(Y-3L)和 EQ-5D-Y-5L(Y-5L)。本研究旨在对 Y-3L 和 Y-5L 进行跨文化调整,以便在新加坡使用,并评估其内容效度,特别是 EQ-5D-Y 描述系统(DS)在亚洲的相关性和全面性:为了对工具进行文化适应性调整,我们咨询了一个由儿科医生和小学教育工作者组成的专家小组。通过对新加坡 8-12 岁儿童进行认知汇报访谈,对专家组提出的修改建议进行了测试。为了评估 EQ-5D-Y DS 的内容效度,我们对 8-15 岁的健康儿童(8 人)和患病儿童(6 人)进行了访谈。在访谈中,孩子们讨论了他们健康状况不佳的经历,并对 EQ-5D-Y DS 的全面性和相关性发表了意见:结果:在跨文化适应过程中,对英国英语 Y-3L 和 Y-5L 版本进行了小幅修改,包括使用当地儿童熟悉的短语和添加例子以方便理解。当儿童讨论他们健康状况不佳的经历时,会自发地引出 EQ-5D-Y DS 中的五个健康维度。访谈中引出的所有与健康状况不佳有关的健康维度可分为三大类:身体健康(如食欲、行动能力和睡眠)、心理健康(如烦恼/沮丧和恐惧/忧虑)和社会关系(如家人和朋友)。尽管 EQ-5D-Y DS 未涵盖一些可能与当地人口相关的健康维度(社会关系和食欲),但总体而言,EQ-5D-Y DS 是相关和全面的:结论:英国英语 EQ-5D-Y 测评工具经过改编后制成了新加坡英语 EQ-5D-Y 测评工具,当地年仅 8 岁的儿童也能理解。EQ-5D-Y DS总体上与衡量当地儿童健康状况不良相关且全面。未来的研究应确定在 EQ-5D-Y DS 中增加与社会关系和食欲有关的附加项目的益处。
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引用次数: 0
Estimating the minimally important difference for the EQ-5D-5L and EORTC QLQ-C30 in cancer. 估算癌症患者 EQ-5D-5L 和 EORTC QLQ-C30 的最小重要差异。
IF 3.2 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-20 DOI: 10.1186/s12955-024-02294-3
Siobhan Bourke, Bryan Bennett, Yemi Oluboyede, Tara Li, Louise Longworth, Sian Bissell O'Sullivan, Julia Braverman, Ioana-Alexandra Soare, James W Shaw

Background: The minimal important difference (MID) is a useful tool to interpret changes in patients' health-related quality of life. This study aims to estimate MIDs for interpreting within-patient change for both components of the EQ-5D-5L questionnaire [EQ-Visual Analogue Scale (EQ-VAS) and utility index] and domains of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) for cancer patients.

Methods: Data were obtained from the Cancer 2015 dataset, a longitudinal cohort of Australian cancer patients. Anchor-based approaches were used to estimate MIDs for the EQ-5D-5L index-based utility index [Australia and the United States (US) tariff sets], EQ-VAS scores, and the EORTC QLQ-C30. Clinical [Eastern Cooperative Oncology Group (ECOG) performance status] and patient-reported (items 29 and 30 of the EORTC QLQ-C30 and the EQ-VAS) anchors were assessed for appropriateness by their correlation strength. Clinical change groups (CCGs) were defined a priori for improvement and deterioration based on estimates used in previous literature. MIDs were estimated via linear regression and distribution-based methods.

Results: For the index-based utility scores in Australia, the anchor-defined MID estimates were 0.01 to 0.06 for improvement and - 0.04 to -0.03 for deterioration, with a weighted value of 0.03 for improvement and deterioration. The EQ-VAS MID estimate was 5 points for both improvement and deterioration. For the EORTC QLQ-C30, changes of at least 3.64 (improvement) and - 4.28 (deterioration) units on the physical functioning scale, 6.31 (improvement) and - 7.11 (deterioration) units on the role functioning scale, 4.65 (improvement) and - 3.41 (deterioration) units on the emotional functioning scale, and 5.41 (improvement) and - 5.56 (deterioration) units on the social functioning scale were estimated to be meaningful.

Conclusion: This study identified lower MIDs for the EQ-5D-5L utility index, EQ-VAS, and EORTC QLQ-C30 domain scores, than those reported previously. The use of a real-world cancer-specific panel dataset may reflect smaller MID estimates that are more applicable to cancer patients in the clinical practice, rather than using MIDs that have been estimated from clinical trials.

背景:最小重要差异(MID)是解释患者健康相关生活质量变化的有用工具。本研究旨在估算最小重要差异,以解释癌症患者的 EQ-5D-5L 问卷的两个组成部分[EQ-视觉模拟量表(EQ-VAS)和效用指数]以及欧洲癌症研究和治疗组织生活质量问卷核心 30(EORTC QLQ-C30)各领域的患者内部变化:数据来自澳大利亚癌症患者纵向队列 "癌症 2015 "数据集。采用基于锚点的方法估算了基于 EQ-5D-5L 指数的效用指数[澳大利亚和美国(US)标准集]、EQ-VAS 评分和 EORTC QLQ-C30 的中位数。临床[东部合作肿瘤学组(Eastern Cooperative Oncology Group,ECOG)表现状态]和患者报告(EORTC QLQ-C30的第29和30项以及EQ-VAS)锚根据其相关强度评估是否合适。临床变化组(CCG)是根据以往文献中使用的估计值预先为病情改善和恶化定义的。通过线性回归和基于分布的方法估算中位数:对于澳大利亚基于指数的效用评分,锚定的 MID 估计值为:改善 0.01 至 0.06,恶化 - 0.04 至 -0.03,改善和恶化的加权值均为 0.03。EQ-VAS 的 MID 估计值为改善和恶化均为 5 点。就 EORTC QLQ-C30 而言,身体功能量表中至少有 3.64 个单位(改善)和-4.28 个单位(恶化)的变化、角色功能量表中至少有 6.31 个单位(改善)和-7.11 个单位(恶化)的变化、情感功能量表中至少有 4.65 个单位(改善)和-3.41 个单位(恶化)的变化、社会功能量表中至少有 5.41 个单位(改善)和-5.56 个单位(恶化)的变化才有意义:本研究确定的 EQ-5D-5L 实用性指数、EQ-VAS 和 EORTC QLQ-C30 领域评分的中位数低于之前的报告。使用真实世界的癌症特异性小组数据集可能反映出较小的 MID 估计值更适用于临床实践中的癌症患者,而不是使用临床试验估计的 MID。
{"title":"Estimating the minimally important difference for the EQ-5D-5L and EORTC QLQ-C30 in cancer.","authors":"Siobhan Bourke, Bryan Bennett, Yemi Oluboyede, Tara Li, Louise Longworth, Sian Bissell O'Sullivan, Julia Braverman, Ioana-Alexandra Soare, James W Shaw","doi":"10.1186/s12955-024-02294-3","DOIUrl":"https://doi.org/10.1186/s12955-024-02294-3","url":null,"abstract":"<p><strong>Background: </strong>The minimal important difference (MID) is a useful tool to interpret changes in patients' health-related quality of life. This study aims to estimate MIDs for interpreting within-patient change for both components of the EQ-5D-5L questionnaire [EQ-Visual Analogue Scale (EQ-VAS) and utility index] and domains of the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30) for cancer patients.</p><p><strong>Methods: </strong>Data were obtained from the Cancer 2015 dataset, a longitudinal cohort of Australian cancer patients. Anchor-based approaches were used to estimate MIDs for the EQ-5D-5L index-based utility index [Australia and the United States (US) tariff sets], EQ-VAS scores, and the EORTC QLQ-C30. Clinical [Eastern Cooperative Oncology Group (ECOG) performance status] and patient-reported (items 29 and 30 of the EORTC QLQ-C30 and the EQ-VAS) anchors were assessed for appropriateness by their correlation strength. Clinical change groups (CCGs) were defined a priori for improvement and deterioration based on estimates used in previous literature. MIDs were estimated via linear regression and distribution-based methods.</p><p><strong>Results: </strong>For the index-based utility scores in Australia, the anchor-defined MID estimates were 0.01 to 0.06 for improvement and - 0.04 to -0.03 for deterioration, with a weighted value of 0.03 for improvement and deterioration. The EQ-VAS MID estimate was 5 points for both improvement and deterioration. For the EORTC QLQ-C30, changes of at least 3.64 (improvement) and - 4.28 (deterioration) units on the physical functioning scale, 6.31 (improvement) and - 7.11 (deterioration) units on the role functioning scale, 4.65 (improvement) and - 3.41 (deterioration) units on the emotional functioning scale, and 5.41 (improvement) and - 5.56 (deterioration) units on the social functioning scale were estimated to be meaningful.</p><p><strong>Conclusion: </strong>This study identified lower MIDs for the EQ-5D-5L utility index, EQ-VAS, and EORTC QLQ-C30 domain scores, than those reported previously. The use of a real-world cancer-specific panel dataset may reflect smaller MID estimates that are more applicable to cancer patients in the clinical practice, rather than using MIDs that have been estimated from clinical trials.</p>","PeriodicalId":12980,"journal":{"name":"Health and Quality of Life Outcomes","volume":"22 1","pages":"81"},"PeriodicalIF":3.2,"publicationDate":"2024-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11416011/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142285965","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
EQ-5D-5L and SF-6Dv2 health utilities scores of diffuse large B-cell lymphoma patients in China 中国弥漫大 B 细胞淋巴瘤患者的 EQ-5D-5L 和 SF-6Dv2 健康效用评分
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-19 DOI: 10.1186/s12955-024-02297-0
Mincai Li, Bingxue Fang, Hongfei Gu, Yawen Jiang
This study evaluates the health-related quality of life (HRQoL) of persons with diffuse large B-cell lymphoma (DLBCL) by using EQ-5D-5L and SF-6Dv2 and compares the measurement properties of the two instruments. DLBCL patients were identified via a patient group and were surveyed using web-based questionnaires. Demographic information, socioeconomic status (SES), clinical characteristics, and EQ-5D-5L and SF-6Dv2 responses were collected and statistically described. The association between the EQ-5D-5L and SF-6Dv2 dimensions were analyzed using the Spearman's correlation coefficient, whereas the correlation of the utility scores was evaluated using Pearson's correlation coefficient. The agreement between the responses of the two instruments were examined using a Bland–Altman (B-A) plot. A one-way analysis of variance (ANOVA) was performed to compare the utility scores across subgroups in different clinical states (a t-test was used if there were two subgroups). In addition, the graded response model (GRM) was used to describe the discrimination ability and difficulty characteristics of the dimensions in the two instruments. In total, 582 valid responses were collected, among which 477 respondents were associated with initial-treatment and 105 respondents were relapsed/refractory (RR) patients. The mean (standard deviation [SD]) EQ-5D-5L and SF-6Dv2 utility scores of the DLBCL patients were 0.828 (0.222) and 0.641 (0.220), respectively. The correlation between the EQ-5D-5L and SF-6Dv2 dimensions ranged from 0.299 to 0.680, and the correlation between their utility scores was 0.787. The B-A plot demonstrated an acceptable but not strong agreement between EQ-5D-5L and SF-6Dv2 utility scores. The GRM model results indicated that all dimensions of each instrument were highly discriminating overall, but EQ-5D-5L had suboptimal discriminative power among patients with good health. Both the EQ-5D-5L and SF-6Dv2 showed valid properties to assess the HRQoL of DLBCL patients. However, utility scores derived from the two instruments had substantial difference, thereby prohibiting the interchangeable use of utilities from the two instruments.
本研究通过使用 EQ-5D-5L 和 SF-6Dv2 评估弥漫大 B 细胞淋巴瘤(DLBCL)患者的健康相关生活质量(HRQoL),并比较两种工具的测量特性。弥漫性大 B 细胞淋巴瘤患者是通过一个患者团体确定的,并使用网络问卷进行了调查。收集了人口统计学信息、社会经济地位(SES)、临床特征、EQ-5D-5L和SF-6Dv2反应,并进行了统计学描述。使用斯皮尔曼相关系数分析了 EQ-5D-5L 和 SF-6Dv2 两个维度之间的关联,而使用皮尔逊相关系数评估了效用评分的相关性。使用布兰德-阿尔特曼(B-A)图检验了两种工具的响应之间的一致性。采用单因素方差分析(ANOVA)来比较不同临床状态下不同亚组的效用得分(如果有两个亚组,则采用 t 检验)。此外,还采用了分级反应模型(GRM)来描述两种工具中各维度的辨别能力和难度特征。共收集到 582 份有效答卷,其中 477 份答卷与初始治疗相关,105 份答卷与复发/难治(RR)患者相关。DLBCL患者的EQ-5D-5L和SF-6Dv2效用评分的平均值(标准差[SD])分别为0.828(0.222)和0.641(0.220)。EQ-5D-5L和SF-6Dv2维度之间的相关性从0.299到0.680不等,其效用得分之间的相关性为0.787。B-A图显示,EQ-5D-5L和SF-6Dv2效用评分之间的一致性可以接受,但并不强。GRM模型结果表明,每种工具的所有维度总体上都具有很高的区分度,但EQ-5D-5L在健康状况良好的患者中的区分度并不理想。EQ-5D-5L和SF-6Dv2都显示出评估DLBCL患者HRQoL的有效特性。但是,这两种工具得出的效用分数有很大差异,因此不能互换使用这两种工具得出的效用分数。
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引用次数: 0
Patient-reported outcome measures for acute rhinosinusitis in adults and children: a systematic review of the quality of existing instruments 针对成人和儿童急性鼻炎的患者报告结果测量方法:对现有工具质量的系统性审查
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-12 DOI: 10.1186/s12955-024-02289-0
Ann-Kristin Baalmann, Sophie Nestler, Theresa Donhauser, Christian Apfelbacher, Katharina Piontek
Acute rhinosinusitis (ARS) is a self-limiting inflammation of the nose and sinuses caused by viral or bacterial infections that requires primarily symptomatic treatment. Patient-reported outcome measures (PROMs) are suitable tools for the assessment of the effectiveness of remedies for ARS from the patient’s perspective in clinical trials and real-world studies. Data regarding the quality of existing PROMs for ARS are limited. To conduct a systematic review of the quality of existing disease-specific PROMs for use in adults and children with ARS according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology, and to derive recommendations for use of the identified instruments in future clinical studies. We systematically searched PubMed, Web of Science and Embase for studies reporting on the development and/or validation of PROMs for ARS. We assessed the methodological quality of each included study, evaluated the quality of measurement properties per PROM and study, and graded the evidence. Based on the overall evidence, we derived recommendations for use of the instruments. We identified four studies on three PROMs measuring symptoms of ARS and quality of life in adults (Sinonasal Outcome Test-16, SNOT-16; Measurement of Acute Rhinosinusitis, MARS; Rhinosinusitis Quality-of-Life Assessment, RhinoQoL). For ARS in children, we identified two studies on two PROMs measuring symptoms of ARS (Pediatric Rhinosinusitis Symptom Scale, PRSS; Sinus Symptom Questionnaire, S5). Our assessment of measurement properties indicates that all instruments require further validation before they can be unrestrictedly recommended for use in future research (COSMIN category B). In particular, there were no content validity studies for any of the identified instruments, but also data on other important measurement properties, e.g., structural validity, are lacking. Currently, no PROM for ARS in adults and children can be unrestrictedly recommended based on the evaluation of their quality. Further validation is required for all identified PROMs. Content validation involving patients and experts should be given priority. OSF ( https://doi.org/10.17605/OSF.IO/VAP8U ).
急性鼻窦炎(ARS)是由病毒或细菌感染引起的鼻腔和鼻窦的一种自限性炎症,主要需要对症治疗。在临床试验和实际研究中,患者报告结果测量(PROMs)是从患者角度评估治疗 ARS 的有效性的合适工具。有关 ARS 现有 PROM 质量的数据十分有限。根据基于共识的健康测量工具选择标准(COSMIN)方法,对用于成人和儿童 ARS 患者的现有疾病特异性 PROMs 的质量进行系统性回顾,并得出在未来临床研究中使用已确定工具的建议。我们系统地检索了 PubMed、Web of Science 和 Embase 中有关 ARS PROMs 开发和/或验证的研究报告。我们评估了每项纳入研究的方法学质量,评价了每个 PROM 和研究的测量属性质量,并对证据进行了分级。根据总体证据,我们得出了使用工具的建议。我们确定了四项关于测量成人 ARS 症状和生活质量的三种 PROM 的研究(鼻窦结果测试-16,SNOT-16;急性鼻炎测量,MARS;鼻炎生活质量评估,RhinoQoL)。对于儿童的 ARS,我们发现了两项关于测量 ARS 症状的 PROM 的研究(儿童鼻炎症状量表 PRSS;窦症状问卷 S5)。我们对测量特性的评估表明,所有工具都需要进一步验证,才能无限制地推荐用于未来的研究(COSMIN B 类)。特别是,所有已确定的工具均未进行内容效度研究,同时也缺乏其他重要测量属性(如结构效度)方面的数据。目前,没有任何一种用于成人和儿童 ARS 的 PROM 可以根据其质量评估结果无限制地推荐使用。所有已确定的 PROM 都需要进一步验证。应优先考虑由患者和专家参与的内容验证。OSF ( https://doi.org/10.17605/OSF.IO/VAP8U )。
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引用次数: 0
A sex-oriented analysis concerning skeletal muscle quantity and quality and associations to quality of life in hospitalized patients with cirrhosis 以性别为导向分析住院肝硬化患者骨骼肌的数量和质量及其与生活质量的关系
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-12 DOI: 10.1186/s12955-024-02295-2
Jie Yang, Gaoyue Guo, Fang Yang, Chaoqun Li, Han Wang, Wanting Yang, Ziyi Yang, Qing Liu, Qian Li, Chao Sun
There is a paucity of data regarding sex-oriented analyses of connection between muscle quantity and quality and health-related quality of life (HRQoL), taking into account the pathophysiological differences of sarcopenia/myosteatosis in males versus females. We sought to investigate the associations between skeletal muscle index (SMI)-defined sarcopenia and intramuscular adipose tissue content (IMAC)-defined myosteatosis and EuroQol-5D (EQ-5D)-defined HRQoL in patients with decompensated cirrhosis concerning sex disparities. Totally, 382 patients were enrolled. The relationship between SMI/IMAC and HRQoL was evaluated with restricted cubic spline and Pearson correlation analyses. Furthermore, association between SMI or sarcopenia and EQ-5D utility index was determined by multiple linear regression, adjusted for age, BMI and concurrent disease severity. The study population comprised evenly distributed male and female patients (190: 192), mean age 61.9 years. The prevalence of sarcopenia (40.5 versus 9.9%, P < 0.001) and SMI (48.8 versus 42.2 cm2/m2, P < 0.001) were significantly higher in males relative to females, with comparable myosteatosis prevalence (15.3 versus 16.7%, P = 0.708). Self-care, usual activities and pain within EQ-5D scale were more prevalent in the sarcopenia compared with non-sarcopenia groups across entire population and stratified by sex. The SMI values exhibited a significantly linear correlation with EQ-5D utility index in male but not female patients (P for non-linearity = 0.281). In multiple analysis, SMI or the presence of sarcopenia was both significantly associated with EQ-5D utility index. Subgroup analyses unveiled no discernible interactions between sarcopenia and EQ-5D utility index. Muscle quantity measured by SMI was associated with declined HRQoL in males rather than females, whereas no associations were found regarding muscle quality measured by IMAC in both sexes. It is tempting to manage sarcopenia by increasing SMI levels as high as possible in hopes of achieving better health consequence. Our findings represent the importance of connecting CT-demarcated body composition abnormalities to meaningful patient-centered outcomes. Future targeted studies with sizable multi-center populations are warranted to clarify this causality, and in consequence develop optimized intervention against sarcopenia/myosteatosis or key determinants concerning impaired HRQoL.
考虑到男性与女性肌肉疏松症/肌营养不良症的病理生理差异,以性别为导向分析肌肉数量和质量与健康相关生活质量(HRQoL)之间联系的数据非常少。我们试图研究失代偿期肝硬化患者的骨骼肌指数(SMI)定义的肌肉疏松症和肌肉内脂肪组织含量(IMAC)定义的肌营养不良症与EQ-5D(EuroQol-5D)定义的HRQoL之间的关系,其中涉及性别差异。共有 382 名患者入选。通过限制性三次样条分析和皮尔逊相关分析评估了 SMI/IMAC 与 HRQoL 之间的关系。此外,SMI 或肌肉疏松症与 EQ-5D 实用指数之间的关系是通过多元线性回归确定的,并对年龄、体重指数和并发疾病严重程度进行了调整。研究人群中男女比例均衡(190:192),平均年龄为 61.9 岁。男性的肌肉疏松症(40.5% 对 9.9%,P < 0.001)和 SMI(48.8 cm2/m2 对 42.2 cm2/m2,P < 0.001)发病率明显高于女性,而肌骨质疏松症的发病率则相当(15.3% 对 16.7%,P = 0.708)。与非肌少症组别相比,在整个人群中,肌少症组别在自我护理、日常活动和 EQ-5D 量表中的疼痛发生率均高于非肌少症组别,并按性别进行了分层。在男性患者中,SMI 值与 EQ-5D 实用性指数呈明显的线性相关,而在女性患者中则不然(非线性 P = 0.281)。在多重分析中,SMI 或肌肉疏松症的存在均与 EQ-5D 实用指数有显著相关性。分组分析显示,肌肉疏松症与 EQ-5D 实用指数之间没有明显的交互作用。以 SMI 测量的肌肉量与 HRQoL 下降相关的是男性而非女性,而以 IMAC 测量的肌肉质量与男女两性均无关联。通过尽可能提高 SMI 水平来控制肌肉疏松症,希望达到更好的健康效果,这很有诱惑力。我们的研究结果表明,将 CT 标记的身体成分异常与以患者为中心的有意义的结果联系起来非常重要。未来有必要对相当规模的多中心人群进行有针对性的研究,以澄清这一因果关系,从而针对肌肉疏松症/骨质疏松症或影响 HRQoL 的关键因素制定优化干预措施。
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引用次数: 0
Dynamic changes in quality of life in older patients with chronic obstructive pulmonary disease: a 7-year follow up 老年慢性阻塞性肺病患者生活质量的动态变化:7 年跟踪调查
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-11 DOI: 10.1186/s12955-024-02296-1
Chun-Hsiang Yu, Sheng-Han Tsai, Jo-Ying Hung, Pei-Fang Su, Chih-Hui Hsu, Xin-Min Liao, Tzuen-Ren Hsiue, Chiung-Zuei Chen
: Chronic obstructive pulmonary disease (COPD) is a major cause of the rapid decline of health-related quality of life (HRQoL), associated with accelerated frailty in older populations. This study aimed to analyse the long-term dynamic changes of HRQoL and the predictive factors for the rapid decline of HRQoL in older patients with COPD. Overall 244 patients with COPD, aged ≧ 65 years from one medical centre were enrolled between March 2012 and July 2020. Further, we prospectively assessed HRQoL scores with utility values, using EuroQol Five-Dimension (EQ-5D) questionnaires. Additionally, long-term dynamic changes in HRQoL were analysed using the Kernel smoothing method and examined the factors contributing to the deterioration of HRQoL using a linear mixed effects model. Older patients with COPD with forced expiration volume (FEV1) < 50% of prediction entered the phase of rapid and continuous decline of HRQoL ~ 2 years after enrolment, but patients with FEV1 ≥ 50% of prediction without rapidly declined HRQoL during 7 years follow up. Therefore, FEV1 < 50% of prediction is a novel predictor for the rapid decline of HRQoL. The course of rapidly declining HRQoL occurred, initially in the usual activities and pain/discomfort domains, followed by the morbidity, self-care, and depression/anxiety domains ~ 2 and 4 years after enrolment, respectively. The mixed effects model indicated that both FEV1 < 50% of prediction and a history of severe acute exacerbation (SAE) requiring hospitalisation were contributing factors for deterioration in HRQoL . Both FEV1 < 50% of prediction and exacerbations requiring hospitalisation were contributing factors for the deterioration of HRQoL in long-term follow up. Additionally, FEV1 < 50% of prediction was a novel predictor for patients entering the phase of rapid decline of HRQoL.
:慢性阻塞性肺疾病(COPD)是导致健康相关生活质量(HRQoL)快速下降的主要原因,与老年人群的加速衰弱有关。本研究旨在分析慢性阻塞性肺病老年患者的 HRQoL 的长期动态变化以及 HRQoL 快速下降的预测因素。在 2012 年 3 月至 2020 年 7 月期间,一家医疗中心共招募了 244 名年龄≧ 65 岁的慢性阻塞性肺病患者。此外,我们还使用 EuroQol Five-Dimension (EQ-5D) 问卷对具有效用值的 HRQoL 分数进行了前瞻性评估。此外,我们还使用核平滑法分析了 HRQoL 的长期动态变化,并使用线性混合效应模型研究了导致 HRQoL 恶化的因素。强迫呼气容积(FEV1)<预测值50%的老年慢性阻塞性肺病患者在入组后2年左右进入HRQoL快速持续下降阶段,但FEV1≥预测值50%的患者在7年随访期间HRQoL没有快速下降。因此,FEV1 < 预测值的 50%是预测 HRQoL 快速下降的一个新指标。HRQoL 快速下降的过程最初发生在日常活动和疼痛/不适领域,随后是发病率、自理能力和抑郁/焦虑领域,分别发生在入组 2 年和 4 年之后。混合效应模型表明,FEV1<预测值的50%和需要住院治疗的严重急性加重(SAE)病史都是导致HRQoL恶化的因素。在长期随访中,FEV1<预测值的 50%和需要住院治疗的病情加重都是导致 HRQoL 恶化的因素。此外,FEV1 < 预测值的 50%是预测患者进入 HRQoL 快速下降阶段的一个新指标。
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引用次数: 0
Validation of the parents’ version of the KINDLR and Kiddy Parents questionnaire in a South African context 在南非验证家长版 KINDLR 和 Kiddy Parents 问卷
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-11 DOI: 10.1186/s12955-024-02292-5
Elmari Deacon, Esmé Jansen van Vuren, Elizabeth Bothma, Chanelle Volschenk, Ruan Kruger
This study aimed to assess the usefulness of the parent version of the KINDLR and the additional items of the Kiddy Parents questionnaire in the South-African context and to validate it as an appropriate tool for measuring health-related quality of life (HRQoL). The ExAMIN Youth SA study was designed to investigate lifestyle behaviours, including psychosocial factors that may adversely impact on cardiovascular health of children. Construct validity was examined by using exploratory and confirmatory factor analysis, while internal consistency was tested by Cronbach’s alpha. The final factor structure was confirmed by model fit indices. The study included children (n = 1088) aged between 5 and 10 years in North-West, South Africa. The reliability coefficients of the original factors could not be reproduced in this data set, with the Cronbach’s alphas ranging between 0.46 and 0.78. With exploratory factor analysis, including the additional items, our data supported a 7-factor structure with acceptable internal consistency (Cronbach’s alpha: 0.68–0.79; Omega: 0.75–0.85) and acceptable model fit indices (CFI: 0.91; TLI: 0.90; RMSEA: 0.05; SRMR: 0.07). Two factors (emotional wellbeing and everyday functioning) further split into separate factors for positive and negative experiences related to each of these dimensions. We confirmed a new factor structure of the parent version of the KINDLR and the additional items of the Kiddy Parents questionnaire, which can be used in the African context. Although the new factor structure has great overlap with the original structure, some items did not contribute to the factors as expected. Language and cultural differences between the original German group and the current South African study group resulted in a different factor structure.
本研究旨在评估 KINDLR 家长版和 Kiddy Parents 问卷附加项目在南非环境中的实用性,并验证其作为测量健康相关生活质量 (HRQoL) 的适当工具的有效性。ExAMIN Youth SA 研究旨在调查生活方式行为,包括可能对儿童心血管健康产生不利影响的社会心理因素。结构效度通过探索性和确认性因子分析进行检验,而内部一致性则通过克朗巴赫α进行检验。模型拟合指数确认了最终的因子结构。研究对象包括南非西北部 5 至 10 岁的儿童(n = 1088)。在这组数据中,原始因子的信度系数无法再现,Cronbach's alphas 介于 0.46 和 0.78 之间。通过探索性因素分析(包括附加项目),我们的数据支持 7 个因素结构,其内部一致性(Cronbach's alpha:0.68-0.79;Omega:0.75-0.85)和模型拟合指数(CFI:0.91;TLI:0.90;RMSEA:0.05;SRMR:0.07)均可接受。两个因子(情绪健康和日常功能)进一步拆分为与每个维度相关的积极和消极体验的独立因子。我们确认了家长版 KINDLR 和 Kiddy Parents 问卷附加项目的新因子结构,该结构可用于非洲环境。虽然新的因子结构与原来的结构有很大的重叠,但有些项目对因子的贡献并没有达到预期。原来的德国研究小组和现在的南非研究小组之间的语言和文化差异导致了不同的因子结构。
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引用次数: 0
How are maternal and fetal outcomes incorporated when measuring benefits of interventions in pregnancy? Findings from a systematic review of cost-utility analyses 在衡量孕期干预措施的益处时,如何纳入孕产妇和胎儿的结果?成本效益分析的系统性审查结果
IF 3.6 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-09-11 DOI: 10.1186/s12955-024-02293-4
Lucy Abel, Helen Dakin, Ting Cai, Richard J. McManus, Abigail McNiven, Oliver Rivero-Arias
Medical interventions used in pregnancy can affect the length and quality of life of both the pregnant person and fetus. The aim of this systematic review was to identify and describe the theoretical frameworks that underpin outcome measurement in cost-utility analyses of pregnancy interventions. Searches were conducted in the Paediatric Economic Database Evaluation (PEDE) database (up to 2017), as well as Medline, Embase and EconLit (2017–2019). We included all cost-utility analyses of any intervention given during pregnancy, published in English. We conducted a narrative synthesis of: study design; outcome construction (life expectancy, quality adjustment, discount rate); and whether the Incremental Cost-Effectiveness Ratio (ICER) was constructed using maternal or fetal outcomes. Where both outcomes were included, methods for combining them were extracted. We identified 127 cost-utility analyses in pregnancy, of which 89 reported QALYs and 38 DALYs. Outcomes were considered solely for the fetus in 59 studies (47%), solely for the pregnant person in 13 studies (10%), and for both in 49 studies (39%). The choice to include or exclude one or both sets of outcomes was not consistent within particular clinical areas. Where outcomes for both mother and baby were included, methods for combining these outcomes varied. Twenty-nine studies summed QALYs/DALYs for maternal and fetal outcomes, with no adjustment. The remaining 20 took a variety of approaches designed to weigh maternal and fetal outcomes differently. These include (1) treating fetal outcomes as a component of maternal quality of life, rather than (or in addition to) an independent individual health outcome; (2) treating the maternal-fetal dyad as a single entity and applying a single utility value to each combination of outcomes; and (3) assigning a shorter time horizon to fetal outcomes to reduce the weight of lifetime fetal outcomes. Each approach made different assumptions about the relative value of maternal and fetal health outcomes, demonstrating a lack of consistency and the need for guidance. Methods for capturing QALY/DALY outcomes in cost-utility analysis in pregnancy vary widely. This lack of consistency indicates a need for new methods to support the valuation of maternal and fetal health outcomes.
孕期医疗干预会影响孕妇和胎儿的寿命和生活质量。本系统性综述旨在确定和描述妊娠干预成本效用分析结果测量的理论框架。我们在儿科经济数据库评估(PEDE)数据库(截至 2017 年)以及 Medline、Embase 和 EconLit(2017-2019 年)中进行了检索。我们纳入了所有以英文发表的孕期干预措施的成本效用分析。我们对以下内容进行了叙述性综述:研究设计;结果构建(预期寿命、质量调整、贴现率);以及增量成本效益比(ICER)是使用母体还是胎儿结果构建的。在同时包含两种结果的情况下,我们还提取了合并两种结果的方法。我们确定了 127 项妊娠成本效用分析,其中 89 项报告了 QALY,38 项报告了 DALY。59项研究(47%)只考虑了胎儿的结果,13项研究(10%)只考虑了孕妇的结果,49项研究(39%)同时考虑了两者的结果。在特定的临床领域中,纳入或排除一组或两组结果的选择并不一致。在同时纳入母亲和婴儿结果的情况下,合并这些结果的方法也各不相同。有 29 项研究将母体和胎儿的 QALYs/DALYs 相加,未作任何调整。其余 20 项研究采用了各种不同的方法,旨在对母体和胎儿的结果进行不同的权衡。这些方法包括:(1)将胎儿结局视为孕产妇生活质量的一个组成部分,而不是(或除了)独立的个体健康结局;(2)将孕产妇-胎儿组合视为一个单一实体,并对每种结局组合应用单一效用值;以及(3)为胎儿结局分配较短的时间跨度,以降低胎儿终生结局的权重。每种方法都对孕产妇和胎儿健康结果的相对价值做出了不同的假设,表明缺乏一致性,需要指导。在妊娠成本效用分析中,获取 QALY/DALY 结果的方法差别很大。这种缺乏一致性的情况表明,需要新的方法来支持对孕产妇和胎儿健康结果的估值。
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Health and Quality of Life Outcomes
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