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  • 文章类型: Journal Article
    背景:健康相关生活质量(HRQL)已成为心脏病学的重要结果参数。MOS36-Item简式健康调查(SF-36)和PROMIS-29是两种广泛使用的通用指标,可提供综合HRQL评分。SF-36是一种使用了几十年的成熟仪器,可以汇总为身体(PCS)和心理(MCS)组件汇总分数。还提出了用于相关分量得分(PCSc和MCSc)的替代评分算法。PROMIS-29是一种较新但越来越多地使用的HRQL措施。类似于SF-36,身体和心理健康总结得分可以从PROMIS-29领域得分得出,基于相关因子解。到目前为止,PROMIS-29的分数不能直接与SF-36的结果相当,使研究结果的汇总复杂化。因此,我们的目标是提供将PROMIS-29数据转换为完善的SF-36分量汇总分数的算法.
    方法:来自柏林血管事件长期观察(BeLOVE)研究的n=662名参与者的数据用于估计线性回归模型,其中PROMIS-29领域得分或汇总的PROMIS-29身体/心理健康汇总得分作为预测因子,SF-36身体/心理成分汇总得分作为结果。来自随后的评估点(n=259)的数据用于评估经验和预测的SF-36评分之间的一致性。
    结果:PROMIS-29领域得分以及PROMIS-29健康总结得分对PCS显示出较高的预测价值,PCSc,和MCSc(R2≥70%),和MCS的中等预测值(R2=57%,R2=40%,分别)。将回归系数应用于新数据后,大多数模型的经验和预测的SF-36分量汇总得分高度相关(r>0.8).经验分数和预测分数之间的平均差异可以忽略不计(|SMD|<0.1)。
    结论:这项研究提供了易于应用的算法,可以将PROMIS-29数据转换为心血管人群中完善的SF-36身体和心理成分汇总得分。应用于新数据,经验和预测的SF-36分数之间的一致性很高.然而,对于SF-36心理成分汇总分数,与原始因子模型(MCS)相比,在相关模型(MCSc)下发现的预测要好得多。此外,作为相关的副产品,我们的研究证实了在心脏病患者中相对较新的PROMIS-29健康总结评分的结构效度.
    BACKGROUND: Health-related quality of life (HRQL) has become an important outcome parameter in cardiology. The MOS 36-ltem Short-Form Health Survey (SF-36) and the PROMIS-29 are two widely used generic measures providing composite HRQL scores. The domains of the SF-36, a well-established instrument utilized for several decades, can be aggregated to physical (PCS) and mental (MCS) component summary scores. Alternative scoring algorithms for correlated component scores (PCSc and MCSc) have also been suggested. The PROMIS-29 is a newer but increasingly used HRQL measure. Analogous to the SF-36, physical and mental health summary scores can be derived from PROMIS-29 domain scores, based on a correlated factor solution. So far, scores from the PROMIS-29 are not directly comparable to SF-36 results, complicating the aggregation of research findings. Thus, our aim was to provide algorithms to convert PROMIS-29 data to well-established SF-36 component summary scores.
    METHODS: Data from n = 662 participants of the Berlin Long-term Observation of Vascular Events (BeLOVE) study were used to estimate linear regression models with either PROMIS-29 domain scores or aggregated PROMIS-29 physical/mental health summary scores as predictors and SF-36 physical/mental component summary scores as outcomes. Data from a subsequent assessment point (n = 259) were used to evaluate the agreement between empirical and predicted SF-36 scores.
    RESULTS: PROMIS-29 domain scores as well as PROMIS-29 health summary scores showed high predictive value for PCS, PCSc, and MCSc (R2 ≥ 70%), and moderate predictive value for MCS (R2 = 57% and R2 = 40%, respectively). After applying the regression coefficients to new data, empirical and predicted SF-36 component summary scores were highly correlated (r > 0.8) for most models. Mean differences between empirical and predicted scores were negligible (|SMD|<0.1).
    CONCLUSIONS: This study provides easy-to-apply algorithms to convert PROMIS-29 data to well-established SF-36 physical and mental component summary scores in a cardiovascular population. Applied to new data, the agreement between empirical and predicted SF-36 scores was high. However, for SF-36 mental component summary scores, considerably better predictions were found under the correlated (MCSc) than under the original factor model (MCS). Additionally, as a pertinent byproduct, our study confirmed construct validity of the relatively new PROMIS-29 health summary scores in cardiology patients.
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  • 文章类型: Journal Article
    开发一种基于深度学习的模型,该模型能够在来自心脏MRI的原始T1图上沿长轴和短轴方向分割左心室(LV)心肌。使用手动分割作为参考标准,对来自50名健康志愿者和75名患者的天然心肌T1图进行了模型训练。基于U-Net架构,我们使用两种不同的训练指标(Sørensen-Dice系数=DSC和Intersection-over-Union=IOU)系统地优化了模型设计,两个不同的激活函数(ReLU和LeakyReLU)和不同数量的训练时期。使用DSC度量和ReLU激活函数在35个时期内进行的训练取得了最高的整体性能(T110.6±17.9ms的平均误差,平均DSC0.88±0.07)。模型结果与地面实况之间的一致性极限为-35.5至36.1ms。这优于两个人类评估者之间的协议(-34.7至59.1ms)。对于长轴视图,分割同样准确(平均误差T1:6.77±8.3ms,平均DSC:0.89±0.03),对于短轴图像(平均误差ΔT1:11.6±19.7ms,平均DSC:0.88±0.08)。可以在长轴和短轴方向上以非常高的精度对天然心肌T1图进行全自动分割和定量分析。
    To develop a deep learning-based model capable of segmenting the left ventricular (LV) myocardium on native T1 maps from cardiac MRI in both long-axis and short-axis orientations. Models were trained on native myocardial T1 maps from 50 healthy volunteers and 75 patients using manual segmentation as the reference standard. Based on a U-Net architecture, we systematically optimized the model design using two different training metrics (Sørensen-Dice coefficient = DSC and Intersection-over-Union = IOU), two different activation functions (ReLU and LeakyReLU) and various numbers of training epochs. Training with DSC metric and a ReLU activation function over 35 epochs achieved the highest overall performance (mean error in T1 10.6 ± 17.9 ms, mean DSC 0.88 ± 0.07). Limits of agreement between model results and ground truth were from -35.5 to + 36.1 ms. This was superior to the agreement between two human raters (-34.7 to + 59.1 ms). Segmentation was as accurate for long-axis views (mean error T1: 6.77 ± 8.3 ms, mean DSC: 0.89 ± 0.03) as for short-axis images (mean error ΔT1: 11.6 ± 19.7 ms, mean DSC: 0.88 ± 0.08). Fully automated segmentation and quantitative analysis of native myocardial T1 maps is possible in both long-axis and short-axis orientations with very high accuracy.
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  • 文章类型: Journal Article
    目的:这项研究考察了伦理,legal,和生育保护的社会影响,强调它对生育的重要性,选修卵子冷冻,和死后辅助生殖,以及它对接受性别确认手术的变性人的影响。
    方法:对600篇文章进行综合分析,专注于各种学科,包括生物伦理学,心理学,和社会学,探索公共和医疗保健专业人员的知识,患者体验,和监管约束。
    结果:文献正在增长,表明人们越来越认识到FP\的重要性。关键主题包括咨询和知情决策的中心地位,尤其是在生育和EEF方面,以及围绕知情同意和相关个人自主性的道德辩论。分析强调了当前研究中的西方中心偏见,强调需要进行更具包容性和文化敏感性的研究。
    结论:这项研究要求对FP有一个细微的理解,倡导考虑道德的政策,文化,和社会维度。这表明跨学科研究解决已发现的差距的必要性,特别是在理解非西方观点和确保全球公平获得FP服务方面。此外,审查强调了整合以患者为中心的方法和道德框架以指导FP实践和政策的重要性,确保他们尊重不同的价值观,满足个人的需求。
    OBJECTIVE: The study examines the ethical, legal, and social implications of fertility preservation, highlighting its importance across oncofertility, elective egg freezing, and posthumous assisted reproduction, as well as its impact on transgender individuals undergoing gender-affirming surgeries.
    METHODS: A comprehensive analysis of 600 articles, focusing on a diverse range of disciplines, including bioethics, psychology, and sociology, to explore public and healthcare professionals\' knowledge, patient experiences, and regulatory constraints.
    RESULTS: The body of literature is growing, indicating increasing recognition of FP\'s significance. Key themes included the centrality of counseling and informed decision-making, especially in oncofertility and EEF, and ethical debates surrounding informed consent and the autonomy of involved individuals. The analysis underscored a western-centric bias in current research, emphasizing the need for more inclusive and culturally sensitive studies.
    CONCLUSIONS: The study calls for a nuanced understanding of FP, advocating for policies that consider ethical, cultural, and social dimensions. It suggests the necessity for interdisciplinary research to address identified gaps, particularly in understanding non-Western perspectives and ensuring equitable access to FP services globally. Moreover, the review emphasizes the importance of integrating patient-centric approaches and ethical frameworks to guide FP practices and policies, ensuring they respect diverse values and meet individuals\' needs.
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  • 文章类型: Journal Article
    背景:检测葡萄糖-6-磷酸脱氢酶(G6PD)缺乏症是治疗疟疾的重要考虑因素。G6PD缺乏可能导致疟疾治疗期间的溶血性贫血,因此,确定G6PD缺乏症在疟疾治疗策略中非常重要。
    方法:本报告提供了范围审查的结果以及证据和差距图,供G6PD近患者试验指南开发小组考虑,以支持间日疟原虫的根治。本范围审查调查了G6PD缺乏症的常见诊断测试以及决策的重要背景和其他因素。这些因素包括世界卫生组织(世卫组织)指南制定手册建议的六个考虑因素,这些因素对确定建议的方向和力度很重要,并包括“可接受性”,\'可行性,\'\'权益,结果的\'\'估值,\'\'性别\'和\'人权\'。本范围审查的目的是为未来的系统审查和证据综合提供指导,这可以更好地为制定世卫组织关于将G6PD缺乏症检测作为疟疾治疗战略一部分的建议提供信息。
    结果:进行了全面搜索,包括出版,任何文章的同行评审文献,研究G6PD诊断测试和“可接受性”因素的任何研究设计和方法,\'可行性,\'\'权益,结果的\'\'估值,\'\'性别\'和\'人权\'。从搜索中确定了1152项研究,其中14人被确定有资格纳入本次审查。这些研究包含来自21个独特国家的数据,这些国家将G6PD诊断测试视为疟疾治疗策略的一部分。上下文和附加因素之间的关系,G6PD缺乏症的诊断测试和研究方法在总体证据和差距中提出,这表明大多数证据是诊断测试的背景因素,和标准G6PD(SD生物传感器)测试。
    结论:本范围审查产生了动态证据和差距图,对G6PD诊断测试领域内的新兴证据具有反应性。证据和差距图提供了所有可用文献的全面描述,这些文献涉及对决策重要的背景和其他因素,关于特定的G6PD诊断测试。调查感兴趣的背景因素的大多数可用数据与定量G6PD诊断测试有关。虽然可以对这些数据进行正式的定性综合,作为系统审查的一部分,数据可能太异,这是不合适的。这些结果现在可用于为世卫组织G6PD指导发展小组的未来方向提供信息,以支持间日疟原虫的根治。
    BACKGROUND: Testing for glucose-6-phosphate dehydrogenase (G6PD) deficiency is an important consideration regarding treatment for malaria. G6PD deficiency may lead to haemolytic anaemia during malaria treatment and, therefore, determining G6PD deficiency in malaria treatment strategies is extremely important.
    METHODS: This report presents the results of a scoping review and evidence and gap map for consideration by the Guideline Development Group for G6PD near patient tests to support radical cure of Plasmodium vivax. This scoping review has investigated common diagnostic tests for G6PD deficiency and important contextual and additional factors for decision-making. These factors include six of the considerations recommended by the World Health Organization (WHO) handbook for guideline development as important to determining the direction and strength of a recommendation, and included \'acceptability\', \'feasibility,\' \'equity,\' \'valuation of outcomes,\' \'gender\' and \'human rights\'. The aim of this scoping review is to inform the direction of future systematic reviews and evidence syntheses, which can then better inform the development of WHO recommendations regarding the use of G6PD deficiency testing as part of malaria treatment strategies.
    RESULTS: A comprehensive search was performed, including published, peer-reviewed literature for any article, of any study design and methodology that investigated G6PD diagnostic tests and the factors of \'acceptability\', \'feasibility,\' \'equity,\' \'valuation of outcomes,\' \'gender\' and \'human rights\'. There were 1152 studies identified from the search, of which 14 were determined to be eligible for inclusion into this review. The studies contained data from over 21 unique countries that had considered G6PD diagnostic testing as part of a malaria treatment strategy. The relationship between contextual and additional factors, diagnostic tests for G6PD deficiency and study methodology is presented in an overall evidence and gap, which showed that majority of the evidence was for the contextual factors for diagnostic tests, and the \'Standard G6PD (SD Biosensor)\' test.
    CONCLUSIONS: This scoping review has produced a dynamic evidence and gap map that is reactive to emerging evidence within the field of G6PD diagnostic testing. The evidence and gap map has provided a comprehensive depiction of all the available literature that address the contextual and additional factors important for decision-making, regarding specific G6PD diagnostic tests. The majority of data available investigating the contextual factors of interest relates to quantitative G6PD diagnostic tests. While a formal qualitative synthesis of this data as part of a systematic review is possible, the data may be too heterogenous for this to be appropriate. These results can now be used to inform future direction of WHO Guideline Development Groups for G6PD near patient tests to support radical cure of P. vivax malaria.
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  • 文章类型: Journal Article
    背景:国际儿科肿瘤学会全球制图计划旨在描述当地的儿科肿瘤能力。这里,我们报告了拉丁美洲的数据。
    方法:一项由10个问题组成的调查分布在儿科肿瘤学服务的主席之间。中心根据患者数量分为高(HVC;每年100例或更多新病例),中型(MVC;31-99例),和低容量中心(LVC;30例或更少),分别。确定了国家转诊中心(NRC)。
    结果:确定了20个国家的307个中心(271个答复),264个回答是可评估的,占预期病例的78%(每年21,359例)。77%的患者在公共中心接受治疗,包括民间社会组织的额外支持。我们发现,66%的患者在70个卓越中心接受治疗,包括21个NRC。每21名新诊断患者中就有一名儿科肿瘤学家(NRC为44),在84%的中心,护士轮转到其他服务。25%的中心缺乏姑息治疗小组。拥有公共资金的LVC拥有姑息治疗团队或训练有素的儿科肿瘤外科医生的可能性大大降低。社会心理,药房,超过93%的中心提供营养服务。21个NRC中有9个在校园内没有放射治疗设施。
    结论:拉丁美洲大多数患有癌症的儿童在公共HVC中接受治疗。儿科肿瘤学家很少,专业护士和外科医生,和姑息治疗小组,特别是在有公共资金的中心。
    BACKGROUND: The International Society of Paediatric Oncology Society Global Mapping Program aims to describe the local pediatric oncology capacities. Here, we report the data from Latin America.
    METHODS: A 10-question survey was distributed among chairs of pediatric oncology services. Centers were classified according to patient volume into high- (HVC; 100 or more new cases per year), medium- (MVC; 31-99 cases), and low-volume centers (LVC; 30 cases or less), respectively. National referral centers (NRC) were identified.
    RESULTS: Total 307 centers in 20 countries were identified (271 responded), and 264 responses were evaluable, accounting for 78% of the expected cases (21,359 cases per year). Seventy-seven percent of patients are treated in public centers, including additional support by civil society organizations. We found that 66% of the patients are treated in 70 centers of excellence, including 21 NRC. There was a median of one pediatric oncologist every 21 newly diagnosed patients (44 for NRC), and in 84% of the centers, nurses rotated to other services. A palliative care team was lacking in 25% of the centers. LVC with public funding have significantly lower probability of having a palliative care team or trained pediatric oncology surgeons. Psychosocial, pharmacy, and nutrition services were available in more than 93% of the centers. No radiotherapy facility was available on campus in nine of 21 NRC.
    CONCLUSIONS: Most children with cancer in Latin America are treated in public HVC. There is a scarcity of pediatric oncologists, specialized nurses and surgeons, and palliative care teams, especially in centers with public funding.
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  • 文章类型: Journal Article
    目标:AQoL-6D,通用的基于偏好的度量,是EQ-5D-5L的一种有吸引力的替代方案,用于评估慢性心力衰竭(HF)患者的健康状况,鉴于其扩大的范围。然而,没有马来西亚的价值观,AQoL-6D无法生成健康状态效用值(HSUV)来支持当地的经济评估。这项研究旨在开发用于预测HF人群中AQoL-6D的EQ-5D-5LHSUV的算法。
    方法:使用来自419名HF门诊患者的多中心队列的横断面数据。使用五组解释变量和8个模型(普通最小二乘,Tobit,审查最小绝对偏差,广义线性模型,两部分模型[TPM],基于β回归的模型,调整后的有限因变量混合模型,和多项序数回归[MLOGIT])。通过十倍交叉验证的平均绝对误差[MAE]和均方根误差[RMSE]来评估模型的预测性能)。还以图形方式检查了潜在的预测偏差。表现最好的模型,RMSE最低,没有偏差,然后被识别。
    结果:在评估的模型中,TPM,其中包括年龄,性别,和五个AQoL-6D维度得分作为预测因子,似乎是从AQoL-6D直接预测EQ-5D-5LHSUV的最佳性能模型。TPM产生最低的MAE(0.0802)和RMSE(0.1116),并证明了HSUV>0.2的预测准确性,没有明显的偏差。为响应映射开发的MGOGIT模型具有次优的预测准确性。
    结论:这项研究开发了潜在有用的映射算法,用于在无法获得直接EQ-5D-5L数据时,从HF患者的AQoL-6D反应中生成马来西亚EQ-5D-5LHSUV。
    OBJECTIVE: The AQoL-6D, a generic preference-based measure, is an appealing alternative to EQ-5D-5L for assessing health status in patients with chronic heart failure (HF), given its expanded scope. However, without a Malaysian value set, the AQoL-6D cannot generate health state utility values (HSUVs) to support local economic evaluations. This study intended to develop algorithms for predicting EQ-5D-5L HSUVs from AQoL-6D in an HF population.
    METHODS: Cross-sectional data from a multi-centre cohort of 419 HF outpatients were used. Both direct and indirect mapping approaches were attempted using five sets of explanatory variables and 8 models (ordinary least squares, Tobit, censored least absolute deviations, generalised linear model, two-part model [TPM], beta regression-based model, adjusted limited dependent variable mixture model, and multinomial ordinal regression [MLOGIT]). The models\' predictive performance was assessed through ten-fold cross-validated mean absolute error [MAE] and root mean squared error [RMSE]). Potential prediction bias was also examined graphically. The best-performing models, with the lowest RMSE and no bias, were then identified.
    RESULTS: Among the models evaluated, TPM, which included age, sex, and five AQoL-6D dimension scores as predictors, appears to be the best-performing model for directly predicting EQ-5D-5L HSUVs from AQoL-6D. TPM yielded the lowest MAE (0.0802) and RMSE (0.1116), and demonstrated predictive accuracy for HSUVs >0.2 without significant bias. A MLOGIT model developed for response mapping had suboptimal predictive accuracy.
    CONCLUSIONS: This study developed potentially useful mapping algorithms for generating Malaysian EQ-5D-5L HSUVs from AQoL-6D responses among patients with HF when direct EQ-5D-5L data is unavailable.
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  • 文章类型: Journal Article
    在减数分裂期间,spo11产生DNA双链断裂诱导重组,在此过程中,共价连接到断裂两侧的5'末端。这种“共价复合物”在野生型细胞中是瞬时的,但积累的核酸酶突变体无法启动修复。此处提供的CC-seq方法详细介绍了如何在同步酿酒酵母减数分裂细胞中以链特异性核苷酸分辨率精度在全基因组范围内绘制这些Po11复合物的位置。
    During meiosis, Spo11 generates DNA double-strand breaks to induce recombination, becoming covalently attached to the 5\' ends on both sides of the break during this process. Such Spo11 \"covalent complexes\" are transient in wild-type cells, but accumulate in nuclease mutants unable to initiate repair. The CC-seq method presented here details how to map the location of these Spo11 complexes genome-wide with strand-specific nucleotide-resolution accuracy in synchronized Saccharomyces cerevisiae meiotic cells.
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  • 文章类型: Journal Article
    背景:基于偏好的健康相关生活质量(HRQoL)测量,例如EQ-5D或SF-6D,对卫生经济评价至关重要。然而,它们很少被纳入强直性脊柱炎(AS)的临床试验.这项研究旨在开发映射算法,以根据巴斯强直性疾病活动指数(BASDAI)和巴斯强直性脊柱炎功能指数(BASFI)预测EQ-5D-3L和EQ-5D-5L健康效用得分。
    方法:从山东省最大的三级医院招募AS患者,中国,2019年12月至2020年10月。采用方便抽样法根据以下标准选择患者:(1)符合纽约标准诊断为AS;(2)年龄18岁及以上;(3)无精神障碍;(4)能够理解问卷;(5)无严重并发症。有243名患者完成了面对面的问卷调查,排除5例关键变量缺失值的病例。普通最小二乘,审查最小绝对偏差,Tobit,直接方法中的调整有限因变量混合模型和β混合模型(BM)以及响应方法中的有序logit和多项式logit(Mlogit)模型用于开发映射算法。平均绝对误差,均方根误差,使用Spearman相关系数和一致性相关系数来获得预测性能。
    结果:238例AS患者的平均年龄为35.19(SD=9.59)岁,男性占多数(74.47%)。观察到的EQ-5D-3L和EQ-5D-5L健康效用值分别为0.88(SD=0.12)和0.74(SD=0.27),分别。EQ-5D-5L与BASDAI和BASFI的概念重叠高于EQ-5D-3L。Mlogit是EQ-5D-3L性能最好的型号,BM在预测EQ-5D-5L方面表现出比其他直接和间接映射模型更好的性能。
    结论:这项研究表明,EQ-5D-5L,而不是EQ-5D-3L,应选择作为中国AS患者HRQoL的目标结局指标,BM映射算法可用于预测BASDAI和BASFI的EQ-5D-5L值,以进行卫生经济学评价。
    BACKGROUND: Preference-based measures of health-related quality of life (HRQoL), such as the EQ-5D or the SF-6D, are essential for health economic evaluation. However, they are rarely included in clinical trials of ankylosing spondylitis (AS). This study aims to develop mapping algorithms to predict EQ-5D-3L and EQ-5D-5L health utility scores from the Bath Ankylosing Disease Activity Index (BASDAI) and the Bath Ankylosing Spondylitis Functional Index (BASFI).
    METHODS: Patients with AS were recruited from the largest tertiary hospital in Shandong province, China, between December 2019 and October 2020. Patients were selected by convenience sampling method according to the following criteria: (1) diagnosed with AS according to the New York criteria; (2) aged 18 years and above; and (3) without mental disorders; (4) able to understand the questionnaires; (5) without serious complications. There were 243 patients who completed the face-to-face questionnaire survey, and 5 cases with missing values in key variables were excluded. Ordinary least squares, censored least absolute deviations, Tobit, adjusted limited dependent variable mixture model and beta-mixture model (BM) in the direct approach and ordered logit and multinomial logit (Mlogit) model in the response approach were used to develop mapping algorithms. Mean absolute error, root mean square error, Spearman\'s correlation coefficient and concordance correlation coefficient were used to access predictive performance.
    RESULTS: The 238 patients with AS had a mean age of 35.19 (SD = 9.59) years, and the majority (74.47%) were male. The observed EQ-5D-3L and EQ-5D-5L health utility values were 0.88 (SD = 0.12) and 0.74 (SD = 0.27), respectively. The EQ-5D-5L had higher conceptual overlap with the BASDAI and BASFI than the EQ-5D-3L did. The Mlogit was the best-performing model for the EQ-5D-3L, and the BM showed better performance in predicting EQ-5D-5L than other direct and indirect mapping models did.
    CONCLUSIONS: This study demonstrates that the EQ-5D-5L, rather than EQ-5D-3L, should be selected as the target outcome measure of HRQoL in patients with AS in China, and the BM mapping algorithm could be used to predict EQ-5D-5L values from BASDAI and BASFI for health economic evaluation.
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  • 文章类型: Journal Article
    背景:口服阿立哌唑在抑制患有抽动障碍的儿童和青少年抽动方面具有良好的临床疗效和安全性。本研究旨在从中国医疗系统的角度评估和比较高剂量和低剂量阿立哌唑在儿童和青少年抽动障碍中的成本效益。
    方法:对146例抽动障碍患者进行问卷调查,其中144人完成了EQ-5D-Y和YGTSS。使用两种映射算法,建立了四个模型将YGTSS转换为EQ-5D-Y实用程序。我们构建了包含疗效和安全性的决策树模型,以根据我们的映射函数比较高剂量和低剂量阿立哌唑的成本效益。
    结果:在我们的决策树模型中选择了具有模型1(YGTSS总评分)的GLM作为首选函数。基本病例成本-效果分析表明,与低剂量阿立哌唑相比,大剂量阿立哌唑可提高0.001QALYs的有效性,并增加197.99美元的总成本,导致每QALY的ICER为174339.22美元,超过人均国内生产总值的三倍。因此,对于患有抽动障碍的儿童患者,大剂量阿立哌唑不太可能是一种具有成本效益的选择.单因素敏感性分析和概率敏感性分析表明,这些结果是稳健的。
    结论:根据现有数据,低剂量阿立哌唑可能是安全的,有效,和儿童和青少年抽动障碍的经济剂量。
    结论:我们研究的主要局限性是缺乏直接用于成本效益分析的效用。我们通过映射函数间接获得了抽动障碍患者的效用。这可能会引入一些偏见和不确定性。在我们的模型中使用德国的直接医疗费用是一个限制。尽管我们使用购买力平价将其转换为中国的等值,在解释本研究结果时应谨慎行事。
    BACKGROUND: Oral aripiprazole exhibits favorable clinical efficacy and safety in the suppression of tics in children and adolescents with tic disorders. This study aims to evaluate and compare the cost-effectiveness of high-dose and low-dose aripiprazole in children and adolescents with tic disorders from the perspective of the Chinese healthcare system.
    METHODS: A questionnaire survey was conducted on 146 patients with tic disorders, of whom 144 completed EQ-5D-Y and YGTSS. Four models were built to convert YGTSS onto EQ-5D-Y utility using two mapping algorithms. We constructed a decision tree model containing efficacy and safety to compare the cost-effectiveness of high-dose and low-dose aripiprazole based on our mapping function.
    RESULTS: The GLM with model 1 (YGTSS total tic scores) was selected as the preferred function in our decision tree model. The base case cost-effectiveness analysis showed that compared to low-dose aripiprazole, high-dose aripiprazole improves effectiveness by 0.001QALYs and increases the overall cost by $197.99, resulting in an ICER of $174339.22 per QALY, which exceeds three times the gross domestic product per capita. Hence, high-dose aripiprazole is not likely to be a cost-effective option for child patients with tic disorders. One-way sensitivity analysis and probabilistic sensitivity analysis showed that these results is robust.
    CONCLUSIONS: On the basis of currently available data, low-dose aripiprazole may be a safe, effective, and economical dosage for children and adolescents with tic disorders.
    CONCLUSIONS: The main limitation of our study is the lack of utility directly used for cost-effectiveness analysis. We obtained the utility of patients with tic disorders indirectly by the mapping function. This may introduce some bias and uncertainty. And it is a limitation to use the direct medical costs of Germany in our model. Although we converted it to the equivalent value of China using purchasing power parities, caution should be exercised when interpreting the results of this study.
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  • 文章类型: Journal Article
    大麦条纹或黄色锈病(BYR)由条锈病引起。大麦(Psh)是大麦生产的重要制约因素。这种疾病最好通过遗传抗性来控制,这被认为是综合疾病管理中最经济和可持续的组成部分。在这项研究中,我们评估了多种疾病环境下一组国际大麦基因型(n=266)对Psh的抗性多样性(厄瓜多尔,印度,和墨西哥)使用全基因组关联研究(GWAS)。确定了与Psh抗性相关的四个数量性状基因座(QTL)(三个在1H染色体上,一个在7H染色体上)。通过在五个双亲群体中绘制对Psh的抗性来验证QTL,它检测到染色体1H上的关键基因组区域(种群Pompadour/Zhoungdamei,Pompadour/Zug161,和CI9214/Baudin),3H(里卡多/格斯),和7H(Fumai8/Baronesse)。RpshQ的QTL。GWAS和RpshQ检测到GWA.1H.1。使用双亲定位种群检测到的Bau.1H在整个环境中最一致和稳定,并且可能是相同的抗性区域。RpshQ.通过富集目标区域,使用populationCI9214/Baudin饱和Bau.1H,在Morex参考基因组v.2中,将抗性基因座置于7.9和8.1Mbp之间(侧翼为标记sun_B1H_03,靠近Rpsh_1H和sun_B1H_KASP_02的0.7cM,在1HS的远端3.2cM)。竞争性等位基因特异性PCR(KASP)标记sun_B1H_KASP_01共分离用于RpshQ。开发了Bau.1H。该标记在50个澳大利亚大麦品种上得到了验证,在六种基因型中显示出明确的等位基因区分和存在(Baudin,Fathom,旗舰,灌浆,Sakurastar,和Shepherd)。该标记可用于可靠的标记辅助选择和Psh抗性的金字塔化,并使抗条锈病的遗传基础多样化。
    Barley stripe or yellow rust (BYR) caused by Puccinia striiformis f. sp. hordei (Psh) is a significant constraint to barley production. The disease is best controlled by genetic resistance, which is considered the most economical and sustainable component of integrated disease management. In this study, we assessed the diversity of resistance to Psh in a panel of international barley genotypes (n = 266) under multiple disease environments (Ecuador, India, and Mexico) using genome-wide association studies (GWASs). Four quantitative trait loci (QTLs) (three on chromosome 1H and one on 7H) associated with resistance to Psh were identified. The QTLs were validated by mapping resistance to Psh in five biparental populations, which detected key genomic regions on chromosomes 1H (populations Pompadour/Zhoungdamei, Pompadour/Zug161, and CI9214/Baudin), 3H (Ricardo/Gus), and 7H (Fumai8/Baronesse). The QTL RpshQ.GWA.1H.1 detected by GWAS and RpshQ.Bau.1H detected using biparental mapping populations co-located were the most consistent and stable across environments and are likely the same resistance region. RpshQ.Bau.1H was saturated using population CI9214/Baudin by enriching the target region, which placed the resistance locus between 7.9 and 8.1 Mbp (flanked by markers sun_B1H_03, 0.7 cM proximal to Rpsh_1H and sun_B1H_KASP_02, 3.2 cM distal on 1HS) in the Morex reference genome v.2. A Kompetitive Allele Specific PCR (KASP) marker sun_B1H_KASP_01 that co-segregated for RpshQ.Bau.1H was developed. The marker was validated on 50 Australian barley cultivars, showing well-defined allelic discrimination and presence in six genotypes (Baudin, Fathom, Flagship, Grout, Sakurastar, and Shepherd). This marker can be used for reliable marker-assisted selection and pyramiding of resistance to Psh and in diversifying the genetic base of resistance to stripe rust.
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