• 【鉴定三个家族中5D型远端关节置换术中的三个新的ECEL1突变。】 复制标题 收藏 收藏
    DOI:10.1111/cge.12226 复制DOI
    作者列表:Shaheen R,Al-Owain M,Khan AO,Zaki MS,Hossni HA,Al-Tassan R,Eyaid W,Alkuraya FS
    BACKGROUND & AIMS: :Arthrogryposis refers to congenital contracture in at least two different body parts. When distal joints are primarily involved, the term distal arthrogryposis (DA) is used. The recognition of clinically distinct subtypes of DA has proven very useful in mapping the disease genes for this genetically heterogeneous condition. DA5D is characterized by ocular involvement usually in the form of ptosis and incomitant strabismus, but extraocular manifestations have also been reported. In a multiplex consanguineous family with DA5D, we combined autozygosity mapping and exome sequencing to identify a novel mutation in ECEL1. This was followed by targeted sequencing of this gene in another two extended consanguineous family with the same phenotype, which revealed two additional novel homozygous mutations. Our results support the recent identification of mutations in ECEL1 as a disease gene in DA5D and expand the clinical and allelic spectrum of this condition.
    背景与目标: 甲状旁腺病是指至少两个不同身体部位的先天性挛缩。当主要涉及远端关节时,将使用术语远端关节固定术(DA)。事实证明,DA的临床独特亚型的识别对于针对这种遗传异质性疾病的疾病基因作图非常有用。 DA5D的特点是眼部受累通常以上睑下垂和斜视性斜视的形式出现,但也有眼外表现的报道。在具有DA5D的多重血缘家族中,我们结合了纯合子作图和外显子组测序来鉴定ECEL1中的新突变。然后在具有相同表型的另外两个近亲家族中对该基因进行靶向测序,这揭示了另外两个新的纯合突变。我们的结果支持了最近鉴定出DA5D中ECEL1作为疾病基因的突变,并扩大了这种疾病的临床和等位基因谱。
  • 【使用多发性硬化症影响量表评估健康状态效用值:从MSIS-29版本2到EQ-5D和SF-6D的映射。】 复制标题 收藏 收藏
    DOI:10.1016/j.jval.2012.07.007 复制DOI
    作者列表:Hawton A,Green C,Telford C,Zajicek J,Wright D
    BACKGROUND & AIMS: OBJECTIVES:The 29-item Multiple Sclerosis Impact Scale (MSIS-29) is a psychometrically validated patient-reported outcome measure increasingly used in trials of treatments for multiple sclerosis. However, it is non-preference-based and not amenable for use across policy decision-making contexts. Our objective was to statistically map from the MSIS-29, version 2, to the EuroQol five-dimension (EQ-5D) and the six-dimension health state short form (derived from short form 36 health survey) (SF-6D) to estimate algorithms for use in cost-effectiveness analyses. METHODS:The relationships between MSIS-29, version 2, and EQ-5D and SF-6D scores were estimated by using data from a cohort of people with multiple sclerosis in South West England (n=672). Six ordinary least squares (OLS), Tobit, and censored least adjusted deviation (CLAD) regression analyses were conducted on estimation samples, including the use of subscale and item scores, squared and interaction terms, and demographics. Algorithms from models with the smallest estimation errors (mean absolute error [MAE], root mean square error [RMSE], normalized RMSE) were then assessed by using separate validation samples. RESULTS:Tobit and CLAD. For the EQ-5D, the OLS models including subscale squared terms, and item scores and demographics performed comparably (MAE 0.147, RMSE 0.202 and MAE 0.147, RMSE 0.203, respectively), and estimated scores well up to 3 years post-baseline. Estimation errors for the SF-6D were smaller (OLS model including squared terms: MAE 0.058, RMSE 0.073; OLS model using item scores and demographics: MAE 0.059, RMSE 0.08), and the errors for poorer health states found with the EQ-5D were less pronounced. CONCLUSIONS:We have provided algorithms for the estimation of health state utility values, both the EQ-5D and SF-6D, from scores on the MSIS-29, version 2. Further research is now needed to determine how these algorithms perform in practical decision-making contexts, when compared with observed EQ-5D and SF-6D values.
    背景与目标: 目的:29项多发性硬化症影响量表(MSIS-29)是一种经心理计量学验证的患者报告的结局指标,越来越多地用于治疗多发性硬化症的试验中。但是,它不是基于首选项的,并且不适合在决策决策环境中使用。我们的目标是从MSIS-29(版本2)到EuroQol五维(EQ-5D)和六维健康状态简表(来自于36卫生调查的简表)(SF-6D)进行统计映射,估算用于成本效益分析的算法。
    方法:使用来自英格兰西南部多发性硬化症人群的数据(n = 672),估计MSIS-29(版本2)与EQ-5D和SF-6D评分之间的关​​系。对估计样本进行了六个普通最小二乘(OLS),Tobit和删失最小调整偏差(CLAD)回归分析,包括使用小数和项目得分,平方和交互作用项以及人口统计学。然后,通过使用单独的验证样本评估来自估计误差最小(平均绝对误差[MAE],均方根误差[RMSE],归一化RMSE)的模型中的算法。
    结果:Tobit和CLAD。对于EQ-5D,OLS模型(包括次级量表平方项)以及项目得分和人口统计数据具有可比性(分别为MAE 0.147,RMSE 0.202和MAE 0.147,RMSE 0.203),并且估计分数在基线后长达3年。 SF-6D的估计误差较小(OLS模型包括平方项:MAE 0.058,RMSE 0.073; OLS模型使用项目评分和人口统计:MAE 0.059,RMSE 0.08),而EQ-5D发现的较差健康状态误差不太明显。
    结论:我们提供了根据MSIS-29版本2的得分估算健康状态效用值(EQ-5D和SF-6D)的算法,现在需要进一步研究以确定这些算法在实际决策中的性能与观察到的EQ-5D和SF-6D值进行比较时。
  • 【[德国疗养院的生活质量-使用EQ-5D问卷调查的结果]。】 复制标题 收藏 收藏
    DOI:10.1055/s-0030-1267946 复制DOI
    作者列表:Alagic V,Staudinger B
    BACKGROUND & AIMS: :In the course of reformation of the health-care system the impact of measuring the quality of life steadily rises. The current study reports the disease-comprehensive quality of life of the elderly who live in nursing homes and compares these results with the reference value of the German population. Furthermore, the results from the different nursing homes are compared with one another in order to prove the direct influence of the nursing home itself on the subjective quality of life of the habitants. The survey was conducted with the generic questionnaire EQ-5D which is an approved index-instrument for health-related quality of life measurement and has been translated into 102 languages so far. In total 342 persons from 8 different nursing homes took part in the survey, at least 25 persons from each nursing home. On the one hand the results show interesting divergences in the quality of life level between habitants and the German population. Differences of at least 2- up to 10-fold values have been proved. For example, there was a discrepancy in the fields of problems related to the habitant's sex, which was not documented in the study of German population. On the other hand high discrepancies when comparing the nursing homes among each other could be proved, although the age structure as well as the distribution between the sexes in at least 7 nursing homes was identical. So it seems that a direct impact of the nursing home on the quality of life of the habitants could be shown. The present study raises many new questions which implicate the need for further surveys. These findings may have a proximate effect on the future health-care system, which will have to involve age-based health-care provision as well as innovative concepts of caring.
    背景与目标: :在医疗体系改革过程中,衡量生活质量的影响稳步上升。当前的研究报告了住在疗养院的老年人的疾病综合生活质量,并将这些结果与德国人口的参考值进行了比较。此外,将来自不同疗养院的结果相互比较,以证明疗养院本身对居民主观生活质量的直接影响。该调查是使用通用问卷EQ-5D进行的,该问卷是用于健康相关生活质量测量的认可索引仪器,迄今为止已被翻译成102种语言。来自8个不同疗养院的342人参加了调查,每个疗养院至少25人。一方面,结果表明居民与德国人口在生活质量上存在有趣的差异。已证明至少有2到10倍的差异。例如,在与居民性别有关的问题领域中存在差异,这在德国人口研究中并未得到记录。另一方面,尽管至少7个疗养院的年龄结构以及男女之间的分布相同,但在相互比较疗养院时仍可证明存在高度差异。因此,似乎可以看出疗养院对居民生活质量的直接影响。本研究提出了许多新的问题,这些问题意味着需要进行进一步的调查。这些发现可能会对未来的医疗体系产生重大影响,未来的医疗体系必须包括基于年龄的医疗保健以及创新的护理概念。
  • 【中国的人口健康状况:EQ-5D结果按年龄,性别和社会经济状况提供,来自2008年《国家卫生服务调查》。】 复制标题 收藏 收藏
    DOI:10.1007/s11136-010-9762-x 复制DOI
    作者列表:Sun S,Chen J,Johannesson M,Kind P,Xu L,Zhang Y,Burström K
    BACKGROUND & AIMS: PURPOSE:To measure and analyse national EQ-5D data and to provide norms for the Chinese general population by age, sex, educational level, income and employment status. METHODS:The EQ-5D instrument was included in the National Health Services Survey 2008 (n = 120,703) to measure health-related quality of life (HRQoL). All descriptive analyses by socio-economic status (educational level, income and employment status) and by clinical characteristics (discomfort during the past 2 weeks, diagnosed with chronic diseases during the past 6 months and hospitalised during the past 12 months) were stratified by sex and age group. RESULTS:Health status declines with advancing age, and women reported worse health status than men, which is in line with EQ-5D population health studies in other countries and previous population health studies in China. The EQ-5D instrument distinguished well for the known groups: positive association between socio-economic status and HRQoL was observed among the Chinese population. Persons with clinical characteristics had worse HRQoL than those without. CONCLUSIONS:This study provides Chinese population HRQoL data measured by the EQ-5D instrument, based on a national representative sample. The main findings for different subgroups are consistent with results from EQ-5D population studies in other countries, and discriminative validity was supported.
    背景与目标: 目的:测量和分析国家EQ-5D数据,并按年龄,性别,教育程度,收入和就业状况为中国普通人群提供规范。
    方法:EQ-5D仪器被纳入《 2008年美国国家卫生服务调查》(n = 120,703),以测量与健康相关的生活质量(HRQoL)。所有按社会经济状况(教育程度,收入和就业状况)和临床特征(过去2周内的不适,过去6个月内被诊断为慢性疾病并在过去12个月内住院)的描述性分析均按性别进行了分层和年龄段。
    结果:健康状况随着年龄的增长而下降,女性报告的健康状况比男性差,这与其他国家的EQ-5D人口健康研究以及中国以前的人口健康研究一致。 EQ-5D仪器在已知人群中表现出色:在中国人口中,社会经济地位与HRQoL之间呈正相关。具有临床特征的人的HRQoL要比没有此特征的人差。
    结论:本研究基于全国代表性样本,提供了由EQ-5D仪器测量的中国人群HRQoL数据。不同亚组的主要发现与其他国家的EQ-5D人口研究的结果一致,并支持判别效度。
  • 【设计基于4d和5d过渡金属酞菁的分子磁性纽扣。】 复制标题 收藏 收藏
    DOI:10.1038/s41598-017-03920-5 复制DOI
    作者列表:Ferriani P,Heinze S,Bellini V
    BACKGROUND & AIMS: :The field of molecular spintronics exploits the properties of organic molecules possessing a magnetic moment, either native in the form of radicals or induced by the insertion of transition metal magnetic ions. To realize logic or storage molecular spin-tronics devices, molecules with stable different magnetic states should be deposited on a substrate, and switching between the states controllably achieved. By means of a first-principles calculations, we have devised a functional molecule exhibiting different magnetic states upon structural changes induced by current injection. We investigate the prototypical case of non-planar M-Phthalocyanine (MPc), where M is a transition-metal ion belonging to the 4d and 5d series. We find that for ZrPc and HfPc deposited on a graphene decorated Ni(111) substrate, two different structural conformations could be stabilized, for which the molecules attain different magnetic states depending on the position of the M ion - whether above the Pc or between the Pc and the substrate -, acting therefore as molecular magnetic button. Our work indicates an intuitive way to engineer a magnetic molecular switch with tailored properties, starting from the knowledge of the basic atomic properties of elements and surfaces.
    背景与目标: :分子自旋电子学领域利用具有磁矩的有机分子的性质,该分子既可以自由基的形式天然存在,也可以通过过渡金属磁性离子的插入而诱发。为了实现逻辑或存储分子自旋电子器件,应将具有稳定的不同磁态的分子沉积在基板上,并在状态之间进行可控地切换。通过第一性原理计算,我们设计了一种功能分子,该功能分子在电流注入引起的结构变化时表现出不同的磁态。我们调查了非平面M-酞菁(MPc)的典型情况,其中M是属于4d和5d系列的过渡金属离子。我们发现,对于沉积在石墨烯修饰的Ni(111)衬底上的ZrPc和HfPc,可以稳定两种不同的结构构象,根据M离子的位置-分子在Pc之上还是在Pc之间,分子获得不同的磁态。 PC和基板-因此充当分子磁性按钮。我们的工作表明,从对元素和表面的基本原子特性的了解开始,设计具有定制特性的磁性分子开关的直观方法。
  • 【EQ-5D维度的患者和一般人群权重的比较。】 复制标题 收藏 收藏
    DOI:10.1002/hec.1362 复制DOI
    作者列表:Mann R,Brazier J,Tsuchiya A
    BACKGROUND & AIMS: :This paper examines the differences in health state evaluations given by patients when they are asked to value their own current states, and those given by members of the general population who were asked to value hypothetical health states. Patient data consist of 4137 observations on EQ-5D profiles and Visual Analogue Scale (VAS) obtained from 3376 patients, covering eight different conditions. General population data are taken from the EQ-5D valuation set. Two analyses were carried out. In the first, the patient self-rated VAS was compared with population VAS values for the same health states. In the second, the patient self-rated VAS values were modelled, and the regression coefficients were compared with the corresponding coefficients from the general population study. The first analysis resulted in a statistically significant mean difference of -0.012 (0.647 for patient VAS, 0.659 from the population value set). The second analysis found statistically significant differences between the coefficients for the EQ-5D health dimensions Pain/Discomfort, Mobility and Anxiety/Depression. Anxiety/Depression had the largest impact on the patient model compared with Pain/Discomfort in the general population model. A further regression analysis suggests that the magnitude of disagreement between patient self-rated VAS model and the population VAS model depends on the patients' condition.
    背景与目标: :本文探讨了当患者被要求评估自己的当前状态时,患者提出的健康状态评估与被要求评估假设的健康状态的普通人群所进行的健康状态评估之间的差异。患者数据包括从3376名患者中获得的关于EQ-5D概况和视觉模拟量表(VAS)的4137个观察结果,涵盖了八种不同的情况。一般人口数据取自EQ-5D评估集。进行了两次分析。首先,将患者自我评估的VAS与相同健康状态的人群VAS值​​进行比较。第二,对患者的自评VAS值进行建模,并将回归系数与一般人群研究中的相应系数进行比较。首次分析得出的统计学上的平均差异为-0.012(患者VAS为0.647,人群值组为0.659)。第二项分析发现,EQ-5D健康维度疼痛/不适,活动能力和焦虑/抑郁的系数之间存在统计学上的显着差异。与一般人群模型中的疼痛/不适相比,焦虑/抑郁对患者模型的影响最大。进一步的回归分析表明,患者自我评估的VAS模型与总体VAS模型之间的分歧程度取决于患者的状况。
  • 【ECEL1中的突变会导致5D型远端关节炎。】 复制标题 收藏 收藏
    DOI:10.1016/j.ajhg.2012.11.014 复制DOI
    作者列表:
    BACKGROUND & AIMS: :Distal arthrogryposis (DA) syndromes are the most common of the heritable congenital-contracture disorders, and ~50% of cases are caused by mutations in genes that encode contractile proteins of skeletal myofibers. DA type 5D (DA5D) is a rare, autosomal-recessive DA previously defined by us and is characterized by congenital contractures of the hands and feet, along with distinctive facial features, including ptosis. We used linkage analysis and whole-genome sequencing of a multiplex consanguineous family to identify in endothelin-converting enzyme-like 1 (ECEL1) mutations that result in DA5D. Evaluation of a total of seven families affected by DA5D revealed in five families ECEL1 mutations that explain ~70% of cases overall. ECEL1 encodes a neuronal endopeptidase and is expressed in the brain and peripheral nerves. Mice deficient in Ecel1 exhibit perturbed terminal branching of motor neurons to the endplate of skeletal muscles, resulting in poor formation of the neuromuscular junction. Our results distinguish a second developmental pathway that causes congenital-contracture syndromes.
    背景与目标: :远距离关节角膜增生症(DA)综合征是可遗传的先天性挛缩性疾病中最常见的一种,约50%的病例是由编码骨骼肌纤维收缩蛋白的基因突变引起的。 5D型DA(DA5D)是我们先前定义的一种罕见的常染色体隐性DA,其特征是手脚先天性挛缩以及包括上睑下垂在内的独特面部特征。我们使用连锁分析和多重血缘家族的全基因组测序来鉴定内皮素转化酶样1(ECEL1)突变,从而导致DA5D。对总共受DA5D影响的7个家庭的评估显示,有5个家庭的ECEL1突变可解释约70%的病例。 ECEL1编码神经元内肽酶,并在大脑和周围神经中表达。缺乏Ecel1的小鼠表现出运动神经元向骨骼肌终板扰动的末端分支,导致神经肌肉接头形成不良。我们的结果区分了导致先天性收缩综合征的第二条发育途径。
  • 【视觉和健康结局,通过活动清单和EQ-5D进行评估,以评估视力障碍。】 复制标题 收藏 收藏
    DOI:10.1111/aos.13430 复制DOI
    作者列表:Macedo AF,Ramos PL,Hernandez-Moreno L,Cima J,Baptista AMG,Marques AP,Massof R,Santana R
    BACKGROUND & AIMS: PURPOSE:Generic instruments to assess health utilities can be used to express the burden of health problems in widely used indexes. That is in contrast with what can be obtained with condition-specific instruments, outcomes are very specific and difficult to compare across conditions. The purpose of this study was to assess health and visual outcomes and its determinants in patients with visual impairment (VI) using the EQ-5D-3L and the Activity Inventory (AI). METHODS:Participants were recruited in different hospitals during the PCVIP-study. A total of 134 patients with acuity 0.30 logMAR or less in the better eye were interviewed. The AI includes 46 goals split between three objectives: social functioning, recreation and daily living, and was used to measure visual ability. The EQ-5D consists of five questions covering one domain each and was used to provide a measure of health states. Responses to each domain were combined to produce a single individual index. RESULTS:The AI and the EQ-5D-3L showed enough discriminatory power between VI levels (p < 0.001), and their results were strongly correlated r(134) = 0.825, (p < 0.001). Explanatory factors for visual ability were level of VI in better eye, age and gender, R2  = 0.43, (p < 0.001). Explanatory factors for the EQ-5D-3L were level of VI in the better eye, comorbidities and gender, R2  = 0.36, (p < 0.001). CONCLUSION:Our results showed that the EQ-5D-3L is useful when characterizing the burden of VI and to compute, when necessary, quality-adjusted-life-years (QALY) changes due to VI. However, it is important to consider that the EQ-5D-3L uses a coarse response scale, assesses a limited spectrum of domains and is influenced by comorbidities. This might limit its responsiveness to small changes in visual ability.
    背景与目标: 目的:评估健康效用的通用工具可用于在广泛使用的指标中表达健康问题的负担。这与使用特定条件的仪器所获得的结果相反,结果是非常特定的,并且很难在不同条件之间进行比较。这项研究的目的是使用EQ-5D-3L和活动清单(AI)评估视力障碍(VI)患者的健康和视觉结果及其决定因素。
    方法:参加者在PCVIP研究期间被招募到不同的医院。总共对134只视力≤0.30logMAR或更小视力的患者进行了访谈。 AI包括46个目标,分为三个目标:社交功能,娱乐和日常生活,并用于衡量视觉能力。 EQ-5D由五个问题组成,每个问题涵盖一个领域,用于衡量健康状况。合并对每个域的响应以生成单个索引。
    结果:AI和EQ-5D-3L在VI水平之间显示出足够的辨别力(p <0.001),并且它们的结果与r(134)= 0.825(p <0.001)有很强的相关性。视觉能力的解释性因素是更好的眼睛,年龄和性别的VI水平,R2 = 0.43,(p <0.001)。 EQ-5D-3L的解释性因素是更好的眼睛中的VI水平,合并症和性别,R2 = 0.36,(p <0.001)。
    结论:我们的结果表明,EQ-5D-3L在表征VI的负担以及在必要时计算VI导致的质量调整生命年(QALY)变化时非常有用。但是,重要的是要考虑到EQ-5D-3L使用粗略的响应量表,评估有限的域谱并受合并症影响。这可能将其响应能力限制为视觉能力的微小变化。
  • 【评估银屑病中EQ-5D-3L和EQ-5D-5L仪器的心理测量特性。】 复制标题 收藏 收藏
    DOI:10.1007/s00403-017-1743-2 复制DOI
    作者列表:Yfantopoulos J,Chantzaras A,Kontodimas S
    BACKGROUND & AIMS: :The aim of this study was to assess the psychometric properties of the five-level (EQ-5D-5L) in comparison with the standard three-level (EQ-5D-3L) classification systems of the EQ-5D in a sample of psoriatic patients. Psoriatic subjects (n = 396) recruited from 16 private practicing centers from various areas of Greece self-completed the two EQ-5D versions and the Dermatology Life Quality Index, while information was also collected on socio-demographics, clinical characteristics and comorbidities. The EQ-5D-5L and EQ-5D-3L were evaluated in terms of agreement, feasibility, ceiling effects, redistribution properties, inconsistency, informativity, and convergent and known-groups validity. Missing values were negligible in both versions. The agreement between the EQ-5D-5L and the EQ-5D-3L was very high (ICC = 0.94), while the largest differences were identified for subjects with moderate health status. Ceiling effects decreased in the EQ-5D-5L system by 14.08% (p < 0.05), with "anxiety/depression" showing the highest relative reduction (-10.31%; p < 0.05). Overall inconsistency was rather low (1.7%) and respondents preferred to report milder problems in the EQ-5D-5L. Absolute informativity improved by 56.42% in the EQ-5D-5L, while relative informativity declined by 9.24%, with only "anxiety/depression" demonstrating a small increase (6.77%). Both instruments demonstrated good convergent and known-groups validity, with evidence of a slightly better convergent performance and discriminatory efficiency of the EQ-5D-5L. In conclusion, both instruments demonstrated consistency, valid redistribution and good construct validity. The EQ-5D-5L system may be preferable to the EQ-5D-3L in psoriatic patients, as it demonstrated a marginally better performance in terms of reduced ceiling effects, increased informativity, and improved convergent and known-groups validity efficiency, particularly in the domain of "anxiety/depression".
    背景与目标: :这项研究的目的是评估EQ-5D的标准三级(EQ-5D-3L)分类系统与五级(EQ-5D-5L)的心理测量特性。银屑病患者。从希腊各个地区的16个私人执业中心招募的银屑病受试者(n = 396)自行完成了两个EQ-5D版本和皮肤病生活质量指数,同时还收集了有关社会人口统计学,临床特征和合并症的信息。对EQ-5D-5L和EQ-5D-3L进行了协议,可行性,上限效应,重新分配属性,不一致,信息性以及聚合组和已知组有效性的评估。在两个版本中,缺失值均可以忽略不计。 EQ-5D-5L和EQ-5D-3L之间的协议非常高(ICC = 0.94),而对于中等健康状态的受试者则发现了最大的差异。在EQ-5D-5L系统中,天花板效应降低了14.08%(p <0.05),其中``焦虑/抑郁''相对降低最高(-10.31%; p <0.05)。总体不一致率很低(1.7%),并且受访者更喜欢报告EQ-5D-5L中的轻微问题。 EQ-5D-5L的绝对信息量提高了56.42%,而相对信息量下降了9.24%,只有“焦虑/沮丧”表现出小幅增长(6.77%)。两种工具都表现出良好的收敛性和已知群体的效度,并证明EQ-5D-5L的收敛性和鉴别效率稍好。总之,这两种工具都显示出一致性,有效的重新分配和良好的构造效度。在银屑病患者中,EQ-5D-5L系统可能比EQ-5D-3L更可取,因为在降低天花板效应,增加信息量以及提高会聚和已知人群的有效性方面,该系统表现出略佳的性能,特别是在“焦虑/抑郁”的领域。
  • 【使用多种建模方法从FACT-B映射到EQ-5D-5 L的功能:来自中国乳腺癌患者的数据。】 复制标题 收藏 收藏
    DOI:10.1186/s12955-019-1224-8 复制DOI
    作者列表:Yang Q,Yu XX,Zhang W,Li H
    BACKGROUND & AIMS: BACKGROUND:The Functional Assessment of Cancer Therapy-Breast (FACT-B) is the most commonly used scale for assessing quality of life in patients with breast cancer. The lack of preference-based measures limits the cost-utility of breast cancer in China. The goal of this study was to explore whether a mapping function can be established from the FACT-B to the EQ-5D-5 L when the EQ-5D health-utility index is not available. METHODS:A cross-sectional survey of adults with breast cancer was conducted in China. All patients included in the study completed the EQ-5D-5 L and the disease-specific FACT-B questionnaire, and demographic and clinical data were also collected. The Chinese tariff value was used to calculate the EQ-5D-5 L utility scores. Five models were evaluated using three different modelling approaches: the ordinary least squares (OLS) model, the Tobit model and the two-part model (TPM). Total scores, domain scores, squared terms and interaction terms were introduced into models. The goodness of fit, signs of the estimated coefficients, and normality of prediction errors of the model were also assessed. The normality of the prediction error is determined by calculating the root mean squared error (RMSE), the mean absolute deviation (MAD), and the mean absolute error (MAE). Akaike information criteria (AIC) and Bayes information criteria (BIC) were also used to assess models and predictive performances. The OLS model was followed by simple linear equating to avoid regression to the mean. RESULTS:The performance of the models was improved after the introduction of the squared terms and the interaction terms. The OLS model, including the squared terms and the interaction terms, performed best for mapping the EQ-5D-5 L. The explanatory power of the OLS model was 70.0%. The AIC and BIC of this model were the smallest (AIC = -705.106, BIC = -643.601). The RMSE, MAD and MAE of the OLS model, Tobit model and TPM were similar. The MAE values of the 5-fold cross-validation of the multiple models in this study were 0.07155~0.08509; meanwhile, the MAE of the TPM was the smallest, followed by that of the OLS model. The OLS regression proved to be the most accurate for the mean, and linearly equated scores were much closer to observed scores. CONCLUSIONS:This study establishes a mapping algorithm based on the Chinese population to estimate the EQ-5D-5 L index of the FACT-B and confirms that OLS models have higher explanatory power and that TPMs have lower prediction error. Given the accuracy of the mean prediction and the simplicity of the model, we recommend using the OLS model. The algorithm can be used to calculate EQ-5D scores when EQ-5D data are not directly collected in a study.
    背景与目标: 背景:乳腺癌的功能评估(FACT-B)是评估乳腺癌患者生活质量最常用的量表。缺乏基于优惠的措施限制了中国乳腺癌的成本效用。这项研究的目的是探索当EQ-5D健康效用指数不可用时,是否可以建立从FACT-B到EQ-5D-5 L的映射功能。
    方法:在中国进行了一项成人乳腺癌横断面调查。纳入研究的所有患者均填写了EQ-5D-5 L和针对疾病的FACT-B问卷,并收集了人口统计学和临床​​数据。使用中国关税价值来计算EQ-5D-5 L实用得分。使用三种不同的建模方法评估了五个模型:普通最小二乘(OLS)模型,Tobit模型和两部分模型(TPM)。将总分数,领域分数,平方项和交互项引入模型。还评估了拟合优度,估计系数的符号以及模型的预测误差的正态性。预测误差的正态性是通过计算均方根误差(RMSE),平均绝对偏差(MAD)和平均绝对误差(MAE)来确定的。 Akaike信息标准(AIC)和Bayes信息标准(BIC)也用于评估模型和预测性能。 OLS模型后面是简单的线性等式,以避免回归到均值。
    结果:引入平方项和交互项后,模型的性能得到改善。包含平方项和交互项的OLS模型最适合映射EQ-5D-5 L,OLS模型的解释力为70.0%。该模型的AIC和BIC最小(AIC = -705.106,BIC = -643.601)。 OLS模型,Tobit模型和TPM的RMSE,MAD和MAE相似。本研究中多个模型的5倍交叉验证的MAE值为0.07155〜0.08509;同时,TPM的MAE最小,其次是OLS模型。 OLS回归被证明是最准确的平均值,并且线性方程得分更接近观察到的得分。
    结论:本研究建立了基于中国人口的映射算法,以估计FACT-B的EQ-5D-5 L指数,并确认OLS模型具有较高的解释力,而TPM具有较低的预测误差。鉴于均值预测的准确性和模型的简单性,我们建议使用OLS模型。当未在研究中未直接收集EQ-5D数据时,该算法可用于计算EQ-5D分数。
  • 【精神卫生患者中EQ-5D和SF-6D效用的比较。】 复制标题 收藏 收藏
    DOI:10.1002/hec.1125 复制DOI
    作者列表:Lamers LM,Bouwmans CA,van Straten A,Donker MC,Hakkaart L
    BACKGROUND & AIMS: :Various preference-based measures of health are available for use as an outcome measure in cost-utility analysis. The aim of this study is to compare two such measures EQ-5D and SF-6D in mental health patients. Baseline data from a Dutch multi-centre randomised trial of 616 patients with mood and/or anxiety disorders were used. Mean and median EQ-5D and SF-6D utilities were compared, both in the total sample and between severity subgroups based on quartiles of SCL-90 scores. Utilities were expected to decline with increased severity. Both EQ-5D and SF-6D utilities differed significantly between patients of adjacent severity groups. Mean utilities increased from 0.51 at baseline to 0.68 at 1.5 years follow-up for EQ-5D and from 0.58 to 0.70 for SF-6D. For all severity subgroups, the mean change in EQ-5D utilities as well as in SF-6D utilities was statistically significant. Standardised response means were higher for SF-6D utilities. We concluded that both EQ-5D and SF-6D discriminated between severity subgroups and captured improvements in health over time. However, the use of EQ-5D resulted in larger health gains and consequent lower cost-utility ratios, especially for the subgroup with the highest severity of mental health problems.
    背景与目标: :各种基于偏好的健康度量都可以用作成本效用分析中的一种结果度量。这项研究的目的是比较精神卫生患者中的两种此类措施EQ-5D和SF-6D。使用来自616名患有情绪和/或焦虑症患者的荷兰多中心随机试验的基线数据。根据SCL-90得分的四分位数,比较了总样本中和严重程度亚组之间的EQ-5D和SF-6D平均值和中位数效用。实用程序预计会随着严重程度的增加而下降。相邻严重程度组的患者之间,EQ-5D和SF-6D实用程序均存在显着差异。 EQ-5D的平均效用从基线的0.51增加到1.5年后的0.68,SF-6D从0.58增至0.70。对于所有严重程度亚组,EQ-5D实用程序和SF-6D实用程序的平均变化均具有统计学意义。 SF-6D实用程序的标准响应方式更高。我们得出的结论是,EQ-5D和SF-6D区分了严重程度亚组,并随着时间的推移获得了健康方面的改善。但是,使用EQ-5D可以带来更大的健康收益,并因此降低成本-效用比,尤其是对于精神健康问题严重程度最高的亚组。
  • 【比较EQ-5D评估研究:系统的审查和方法学报告清单。】 复制标题 收藏 收藏
    DOI:10.1177/0272989X13480852 复制DOI
    作者列表:Xie F,Gaebel K,Perampaladas K,Doble B,Pullenayegum E
    BACKGROUND & AIMS: BACKGROUND:There has been a growing interest around the world in developing country-specific scoring algorithms for the EQ-5D. This study systematically reviews all existing EQ-5D valuation studies to highlight their strengths and limitations, explores heterogeneity in observed utilities using meta-regression, and proposes a methodological checklist for reporting EQ-5D valuation studies. METHODS:. We searched Medline, EMBASE, the National Health Service Economic Evaluation Database (NHS EED) via Wiley's Cochrane Library, and Wiley's Health Economic Evaluation Database from inception through November 2012, as well as bibliographies of key papers and the EuroQol Plenary Meeting Proceedings from 1991 to 2012 for English-language reports of EQ-5D valuation studies. Two reviewers independently screened the titles and abstracts for relevance. Three reviewers performed data extraction and compared the characteristics and scoring algorithms developed in the included valuation studies. RESULTS:. Of the 31 studies included in the review, 19 used the time trade-off (TTO) technique, 10 used the visual analogue scale (VAS) technique, and 2 used both TTO and VAS. Most studies included respondents from the general population selected by random or quota sampling and used face-to-face interviews or postal surveys. Studies valued between 7 and 198 total states, with 1-23 states valued per respondent. Different model specifications have been proposed for scoring. Some sample or demographic factors, including gender, education, percentage urban population, and national health care expenditure, were associated with differences in observed utilities for moderate or severe health states. CONCLUSIONS:. EQ-5D valuation studies conducted to date have varied widely in their design and in the resulting scoring algorithms. Therefore, we propose the Checklist for Reporting Valuation Studies of the EQ-5D (CREATE) for those conducting valuation studies.
    背景与目标: 背景:在世界范围内,针对EQ-5D的特定于发展中国家的评分算法的兴趣日益浓厚。这项研究系统地回顾了所有现有的EQ-5D评估研究,以突出它们的优势和局限性,使用元回归分析观测到的公用事业中的异质性,并提出了用于报告EQ-5D评估研究的方法清单。
    方法:。从开始到2012年11月,我们通过Wiley的Cochrane图书馆搜索了Medline,EMBASE,国家卫生服务经济评估数据库(NHS EED)和Wiley的卫生经济评估数据库,以及1991年至1991年的主要论文书目和EuroQol全体会议记录。 2012年用于EQ-5D评估研究的英语报告。两名审稿人独立筛选了标题和摘要以确保相关性。三名审阅者进行了数据提取,并比较了包括在评估研究中的特征和评分算法。
    结果:。该评价纳入的31项研究中,有19项使用了时间权衡(TTO)技术,有10项使用了视觉模拟量表(VAS)技术,还有2项同时使用了TTO和VAS。大多数研究包括通过随机抽样或定额抽样从普通人群中选择的受访者,并使用了面对面访谈或邮政调查的方式。研究评估了7至198个总州之间的价值,每个受访者评估了1至23个州的价值。已经提出了用于评分的不同模型规格。一些样本或人口统计因素,包括性别,教育程度,城市人口百分比和国家卫生保健支出,与中度或重度健康状态的观测公用事业差异有关。
    结论:迄今为止,进行的EQ-5D评估研究在设计和产生的评分算法上千差万别。因此,我们为进行估值研究的人员提出了EQ-5D估值研究报告清单(CREATE)。
  • 【欧洲生活质量(EQ-5D)衡量生活质量的方法可预测接受护理的受HIV感染的成年人的死亡率,急诊室利用率和出院率。】 复制标题 收藏 收藏
    DOI:10.1186/1477-7525-5-5 复制DOI
    作者列表:Mathews WC,May S
    BACKGROUND & AIMS: BACKGROUND:Health-related quality of life (HR-QOL) is a relevant and quantifiable outcome of care. We implemented HR-QOL assessment at all primary care visits at UCSD Owen Clinic using EQ-5D. The study aim was to estimate the prognostic value of EQ-5D for survival, hospitalization, and emergency department (ED) utilization after controlling for CD4 and HIV plasma viral load (pVL). METHODS:We conducted a retrospective analysis of HIV clinic based cohort (1996-2000). The EQ-5D includes single item measures of: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each item is coded using 3-levels (1 = no problems; 2 = some problems; 3 = severe problems). The instrument includes a global rating of current health using a visual analog scale (VAS) ranging from 0 (worst imaginable) to 100 (best imaginable). An additional single item measure of health change (better, much the same, worse) was included. A predicted VAS (pVAS) was estimated by regressing the 5 EQ-5D health states on VAS using reference cell coding of health states and random effects linear models. Survival models were fit using Cox modelling. Hospitalization and ED rate models were estimated using population-averaged Poisson models. RESULTS:965 patients met eligibility criteria. 12% were female; 42% were non-white. Median time-at-risk was 1.2 years. Median CD4 was 233. Median log10(pVL) was 4.6. 47 deaths occurred. In two Cox models controlling for CD4 and pVL, the adjusted hazard ratios (aHR) for VAS and pVAS as time-varying covariates were 0.73 (95% CI: 0.63-0.83) and 0.66 (95% CI: 0.56-0.77) respectively, for every 10 point increase in (p)VAS rating. In Poisson regression models predicting ED visit rates and hospital discharge rates controlling for current CD4 and pVL, each of the EQ-5D health dimensions, VAS, and health change items were significantly (p < 0.05) associated with the outcomes. For ED visit rates, the adjusted incidence rate ratios (aIRR) were 0.86 (0.83-0.89) and 0.79 (0.75-0.82) for VAS and pVAS, respectively. For hospital discharge rates, the aIRR's were 0.85 (0.82-0.88) and 0.79 (0.75-0.82) for VAS and pVAS, respectively. CONCLUSION:EQ-5D is a brief and prognostically useful predictor of mortality, hospitalization, and ED utilization among adults under care for HIV infection, even after adjusting for CD4 and HIV plasma viral load.
    背景与目标: 背景:健康相关的生活质量(HR-QOL)是护理的相关且可量化的结果。我们使用EQ-5D在UCSD欧文诊所的所有初级保健就诊中实施了HR-QOL评估。研究目的是评估控制CD4和HIV血浆病毒载量(pVL)后,EQ-5D对生存,住院和急诊科(ED)利用的预后价值。
    方法:我们对1996-2000年基于HIV临床研究的队列进行了回顾性分析。 EQ-5D包括以下各项的单项指标:活动能力,自我护理,日常活动,疼痛/不适和焦虑/抑郁。每个项目使用3级编码(1 =没问题; 2 =某些问题; 3 =严重的问题)。该仪器使用视觉模拟量表(VAS)对当前健康状况进行总体评分,其范围从0(可想象的最差)到100(可想象的最好)。还包括一项其他的健康变化单项指标(更好,大致相同,更差)。通过使用健康状态的参考细胞编码和随机效应线性模型对VAS上的5个EQ-5D健康状态进行回归,可以估算出预测的VAS(pVAS)。使用Cox模型拟合生存模型。住院和急诊室率模型是使用人口平均泊松模型估算的。
    结果:965名患者符合入选标准。女性占12%; 42%为非白人。中位风险时间为1.2年。 CD4中位数为233。log10(pVL)中位数为4.6。发生了47人死亡。在控制CD4和pVL的两个Cox模型中,VAS和pVAS的时变协变量的调整后风险比(aHR)分别为0.73(95%CI:0.63-0.83)和0.66(95%CI:0.56-0.77), (p)VAS评级每提高10点,就可以达到一次。在预测当前CD4和pVL的ED访视率和出院率的Poisson回归模型中,EQ-5D健康维度,VAS和健康变化项均与结果显着相关(p <0.05)。对于急诊就诊率,VAS和pVAS的调整后发病率比(aIRR)分别为0.86(0.83-0.89)和0.79(0.75-0.82)。对于医院出院率,VAS和pVAS的aIRR分别为0.85(0.82-0.88)和0.79(0.75-0.82)。
    结论:EQ-5D是即使在调整了CD4和HIV血浆病毒载量之后,在接受HIV感染的成年人中,死亡率,住院率和ED利用率的简短且对预后有用的预测指标。
  • 【评估响应系统之间的等效性:将Rasch模型应用于3级和5级EQ-5D。】 复制标题 收藏 收藏
    DOI:10.1097/MLR.0b013e31805371aa 复制DOI
    作者列表:Pickard AS,Kohlmann T,Janssen MF,Bonsel G,Rosenbloom S,Cella D
    BACKGROUND & AIMS: BACKGROUND:Expansion of the EQ-5D health state classifier to 5 levels (EQ-5D-5L) has been proposed to improve discriminative and evaluative properties, but current preference-based algorithms were developed for a 3-level (EQ-5D-3L) structure. The objectives were to examine equivalency of meaning between 3L and 5L response systems, and to psychometrically derive a system of weights that facilitate conversion of 3L preference-based algorithms to a 5L system. METHODS:Rasch models were used to examine the equivalency of the 3L and 5L systems using 2 datasets where health status was assessed using the 3L and 5L: a Dutch study of primarily hypothetical health state assessments and a US-based multicenter study of 423 cancer patients. Category-specific mean values of latent person parameters (using maximum likelihood estimation) for the levels of the 3L and 5L systems were estimated. RESULTS:Means on the latent continuum pertaining to level 3 in the 5L system and level 2 in the 3L (some problems) were similar for both datasets, suggesting equivalence of these levels. Extremes of the 5L response structure consistently broadened the measurement continuum. By anchoring "no problems" as 0 disutility, disutility weights from EQ-5D-3L were transformed into weights for EQ-5D-5L using ratios of logit distances between person means for 5L and 3L calibrated for each dimension using the Rasch model. CONCLUSIONS:This study illustrates the rich potential for modern psychometric techniques both to examine equivalency when health status measures are modified as well as to inform preference-based measurement systems using existing value sets.
    背景与目标: 背景:已提出将EQ-5D健康状态分类器扩展到5个级别(EQ-5D-5L)以提高判别和评估属性,但目前针对3个级别(EQ-5D-3L)开发了基于偏好的算法) 结构体。目的是检查3L和5L响应系统之间的含义是否相等,并从心理上得出一个权重系统,该权重系统有助于将基于3L偏好的算法转换为5L系统。
    方法:使用Rasch模型通过2个数据集检查3L和5L系统的等效性,其中使用3L和5L评估健康状况:主要是假设的健康状况评估的荷兰研究和基于美国的423名癌症患者的多中心研究。估计了3L和5L系统级别的潜在人员参数的类别特定平均值(使用最大似然估计)。
    结果:在两个数据集中,与5L系统中的第3级和3L中的第2级(一些问题)有关的潜在连续统的均值相似,表明这些级别的等效性。 5L响应结构的极端特性不断扩大了测量范围。通过将“无问题”锚定为0无效度,使用Rasch模型针对每个维度校准的5L和3L人均均对数距离的比率,将EQ-5D-3L的无效度权重转换为EQ-5D-5L的权重。
    结论:本研究说明了现代心理计量学技术的巨大潜力,既可用于检查健康状况测度被修改时的等效性,又可利用现有值集为基于偏好的测度系统提供信息。
  • 【EQ-5D,howRu和牛津臀部和膝盖得分在评估髋关节和膝盖置换的效果方面的表现。】 复制标题 收藏 收藏
    DOI:10.1186/s12913-016-1759-x 复制DOI
    作者列表:Benson T,Williams DH,Potts HW
    BACKGROUND & AIMS: BACKGROUND:We aimed to compare the performance of EQ-5D-3 L and howRu, which are short generic patient-reported outcome measures (PROMs), in assessing the outcome of hip and knee replacements, using the Oxford Hip Score (OHS) and the Oxford Knee Scores (OKS) for comparison. METHODS:Outcome was assessed as the difference between pre-surgery and 6-month post-surgery scores. We used a large sample from the NHS PROMs database, which used EQ-5D-3 L, and a small cohort of patients having the same operations collected by MyClinicalOutcomes (MCO), which used howRu. Both cohorts completed the OHS (hips) or the OKS (knees). RESULTS:The change (outcome) between pre-op and post-op scores as measured by howRu was greater than that measured by EQ-5D, relative to that measured by OHS or OKS. For hip replacements, the correlation for change measured by howRu and OHS was r = 0.77 (0.66-0.85). The corresponding correlation for change measured by EQ-5D Index and OHS was r = 0.64 (0.63-0.64). For knee replacements the correlation between change in howRu and OKS was r = 0.86 (0.75-0.92); between EQ-5D Index and OKS r = 0.59 (0.58-0.60). CONCLUSIONS:For hip and knee replacement, the outcome measured by howRu was more highly correlated with that measured by the condition-specific Oxford Hip and Knee Scores than were EQ-5D Index or EQ-VAS. The magnitude of change before and after surgery was also greater.
    背景与目标: 背景:我们的目的是使用牛津髋关节评分(OHS)和EQ-5D-3 L和howRu(这是患者报告的简短通用结果指标(PROM))的性能进行比较,以评估髋关节和膝关节置换的结果。牛津膝盖得分(OKS)进行比较。
    方法:将结果评估为术前和术后6个月得分之间的差异。我们使用了来自NHS PROMs数据库的大量样本,该样本使用了EQ-5D-3L,以及一小群通过MyClinicalOutcomes(MCO)收集的具有相同手术的患者,并使用了HowRu。两个队列都完成了OHS(臀部)或OKS(膝盖)。
    结果:相对于OHS或OKS而言,由howRu衡量的术前和术后分数之间的变化(结果)大于EQ-5D的变化。对于髋关节置换,通过howRu和OHS测得的变化相关性为r = 0.77(0.66-0.85)。通过EQ-5D指数和OHS测得的变化的相应相关性是r = 0.64(0.63-0.64)。对于膝关节置换,howRu和OKS的变化之间的相关性为r = 0.86(0.75-0.92);在EQ-5D索引和OKS之间的r = 0.59(0.58-0.60)。
    结论:对于髋关节和膝关节置换,与EQ-5D指数或EQ-VAS相比,通过howRu测得的结局与通过特定条件的牛津臀部和膝盖得分测得的结局具有更高的相关性。手术前后的变化幅度也更大。

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