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Finite Markov chains with absorbing states and mis-specified random effects: application to cognitive data. 具有吸收态和错误指定随机效应的有限马尔可夫链:在认知数据中的应用。
Q3 Medicine Pub Date : 2025-01-01 Epub Date: 2025-01-18 DOI: 10.1080/24709360.2025.2451519
Pei Wang, Changrui Liu, Jiyeon Park, Suzanne L Tyas, Richard J Kryscio

Finite Markov chains with absorbing states are valuable tools for analyzing longitudinal data with categorical responses. However, defining the one-step transition probabilities in terms of fixed and random effects presents challenges due to the large number of unknown parameters involved. To address this, we employ a marginal model to estimate the fixed effects across various choices of the distribution governing the random effects. Subsequently, we utilize an h-likelihood method to estimate the random effects based on these fixed effect estimates. The estimation approach is applied to analyze longitudinal cognitive data from the Nun Study. Our findings highlight that the fixed effects remain relatively robust across a wide range of assumptions. However, the analysis of random effects utilizing tools such as AIC, Q-Q plots, and gradient plots appears to be sensitive to mis-specifications in the distribution of the random effects. Our proposed approach allows researchers to verify the assumptions of random effects and provides more accurate estimation of these effects. Additionally, the precisely estimated random effects enable researchers to identify individuals at high risk for absorbing states (e.g., incurable diseases) and to determine the progression rates for certain diseases.

具有吸收态的有限马尔可夫链是分析具有分类响应的纵向数据的有效工具。然而,由于涉及大量未知参数,根据固定效应和随机效应定义一步转移概率存在挑战。为了解决这个问题,我们采用一个边际模型来估计控制随机效应的分布的各种选择的固定效应。随后,我们利用h-似然方法在这些固定效应估计的基础上估计随机效应。估计方法被应用于分析来自修女研究的纵向认知数据。我们的研究结果强调,在广泛的假设范围内,固定效应仍然相对强劲。然而,利用AIC、Q-Q图和梯度图等工具对随机效应的分析似乎对随机效应分布的错误规范很敏感。我们提出的方法允许研究人员验证随机效应的假设,并提供更准确的估计这些效应。此外,精确估计的随机效应使研究人员能够识别处于吸收状态高风险的个体(例如,不治之症),并确定某些疾病的进展率。
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引用次数: 0
Mean Cost and Cost-Effectiveness Ratios with Censored Data: a Tutorial and SAS® Macros. 平均成本和成本效益比与删节数据:教程和SAS®宏。
Q3 Medicine Pub Date : 2025-01-01 Epub Date: 2025-10-06 DOI: 10.1080/24709360.2025.2565537
Eduard Poltavskiy, Dingning Liu, Shuai Chen, Heejung Bang, Hongwei Zhao

Censoring is an unignorable issue when analyzing survival data and/or medical cost data. Medical costs may be viewed as a type of survival data-in that they accrue over time until an endpoint such as death-or a 'mark' variable. Since Lin et al. (1997) and Mushlin et al. (1998) published landmark papers on this topic, censored cost data have been extensively studied. In this tutorial, we explain how to estimate mean cost and cost-effectiveness ratios, along with three examples under two different data scenarios: when only total cost data (so one observation per person) or longitudinal data (or cost history) are available. We also provide an updated literature review. SAS codes in the supplement could be useful to practitioners and data analysts.

在分析生存数据和/或医疗费用数据时,审查是一个不可忽视的问题。医疗费用可以被视为一种生存数据,因为它们随着时间的推移而累积,直到一个终点(如死亡)或一个“标记”变量。自从Lin等人(1997)和Mushlin等人(1998)发表了关于这一主题的具有里程碑意义的论文以来,审查成本数据得到了广泛的研究。在本教程中,我们将解释如何估计平均成本和成本效益比率,并提供两种不同数据场景下的三个示例:只有总成本数据(即每人一次观察)或纵向数据(或成本历史)可用。我们还提供了最新的文献综述。附录中的SAS代码可能对从业人员和数据分析人员有用。
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引用次数: 0
Adjusting for bias due to measurement error in functional quantile regression models with error-prone functional and scalar covariates. 在容易出错的函数协变量和标量协变量的功能分位数回归模型中调整由于测量误差引起的偏差。
Q3 Medicine Pub Date : 2024-01-01 Epub Date: 2024-10-02 DOI: 10.1080/24709360.2024.2405439
Xiwei Chen, Heyang Ji, Yuanyuan Luan, Roger S Zoh, Lan Xue, Sneha Jadhav, Carmen D Tekwe

Wearable devices enable the continuous monitoring of physical activity (PA) but generate complex functional data with poorly characterized errors. Most work on functional data views the data as smooth, latent curves obtained at discrete time intervals with some random noise with mean zero and constant variance. Viewing this noise as homoscedastic and independent ignores potential serial correlations. Our preliminary studies indicate that failing to account for these serial correlations can bias estimations. In dietary assessments, epidemiologists often use self-reported measures based on food frequency questionnaires that are prone to recall bias. With the increased availability of complex, high-dimensional functional, and scalar biomedical data potentially prone to measurement errors, it is necessary to adjust for biases induced by these errors to permit accurate analyses in various regression settings. However, there has been limited work to address measurement errors in functional and scalar covariates in the context of quantile regression. Therefore, we developed new statistical methods based on simulation extrapolation (SIMEX) and mixed effects regression with repeated measures to correct for measurement error biases in this context. We conducted simulation studies to establish the finite sample properties of our new methods. The methods are illustrated through application to a real data set.

可穿戴设备能够持续监测身体活动(PA),但会产生复杂的功能数据,并且具有较差的特征误差。大多数关于函数数据的工作将数据视为在离散时间间隔内获得的平滑的潜在曲线,其中包含一些均值为零且方差恒定的随机噪声。将这种噪声视为均方差和独立的,忽略了潜在的序列相关性。我们的初步研究表明,不考虑这些序列相关性可能会使估计产生偏差。在饮食评估中,流行病学家经常使用基于食物频率问卷的自我报告测量方法,这种方法容易产生回忆偏差。随着复杂、高维功能和标量生物医学数据的可用性增加,可能容易出现测量误差,因此有必要调整由这些误差引起的偏差,以便在各种回归设置中进行准确的分析。然而,在分位数回归的背景下,解决函数和标量协变量测量误差的工作有限。因此,我们开发了基于模拟外推(SIMEX)和重复测量的混合效应回归的新统计方法,以纠正这种情况下的测量误差偏差。我们进行了模拟研究,以确定我们的新方法的有限样本性质。通过对一个实际数据集的应用说明了这些方法。
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引用次数: 0
The analysis of Salmonella’s ability to survive in different external environments 沙门氏菌在不同外部环境下的生存能力分析
Q3 Medicine Pub Date : 2023-01-02 DOI: 10.1080/24709360.2023.2265277
Wesam R. Kadhum, Lyudmila Sviridova, Dmitry Snegirev
AbstractThe work aims to analyze the survival of the Salmonella pathogen in various objects of the outdoor environment (water, soil). Survival rates for Salmonella isolated in agar-agar from aqueous media (distilled water, tap water, well water, seawater) and soil were investigated. Every seven days, samples were subjected to bacteriological analysis, where they were streaked onto nutrient agar medium at a temperature of 36°C to determine the presence of viable Salmonella. In cases where Salmonella was not detected, microscopic examination was conducted to ascertain the presence of dead bacteria. Seasonal aspects of calf morbidity due to salmonellosis were examined. Salmonella survival in distilled water was maximal and exceeded four months; in well water, it survived two months (p ≤ 0.05 with distilled water); the survival rate in tap and sea water was one month (p ≤ 0.01). Salmonella was viable for more than eight months in artificially contaminated chernozem, five months in grey forest soil (p ≤ 0.05), and for at least three months in the soil at 0°C Salmonella (p ≤ 0.01). Salmonellosis is more common in 4–35% of calves 1–3 months of age. Salmonella can live outdoors, remaining viable and virulent in soil and water for 5–8 months.KEYWORDS: Salmonellaeexternal environmentaquatic environmentsoilsurvivalvirulence Disclosure statementNo potential conflict of interest was reported by the author(s).Data availability statementData will be available on request.
摘要本工作旨在分析沙门氏菌病原菌在室外环境(水、土壤)各种物体中的生存情况。研究了从水(蒸馏水、自来水、井水、海水)和土壤中分离的琼脂沙门氏菌的存活率。每隔7天,对样品进行细菌学分析,在36°C的温度下,将样品放在营养琼脂培养基上,以确定活沙门氏菌的存在。在没有检测到沙门氏菌的情况下,进行显微镜检查以确定死细菌的存在。小牛发病率的季节性方面,由于沙门氏菌病进行了检查。沙门氏菌在蒸馏水中的存活率最高,超过4个月;井水中存活2个月(蒸馏水中p≤0.05);在自来水和海水中的存活率均为1个月(p≤0.01)。沙门菌在人工污染黑钙土中存活8个月以上,在灰色森林土中存活5个月(p≤0.05),在0℃沙门菌土壤中存活3个月以上(p≤0.01)。沙门氏菌病在1-3个月大的犊牛中更为常见,占4-35%。沙门氏菌可在室外存活,在土壤和水中可存活5-8个月。关键词:沙门氏菌;外部环境;水生环境;土壤;数据可用性声明数据可应要求提供。
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引用次数: 0
Notice of duplicate publication: public transportation network scan for rapid surveillance 复发通知:公交网络扫描快速监控
Q3 Medicine Pub Date : 2023-01-02 DOI: 10.1080/24709360.2023.2275481
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引用次数: 0
Global Odds Model with Proportional Odds and Trend Odds Applied to Gross and Microscopic Brain Infarcts. 具有比例赔率和趋势赔率的全局赔率模型应用于大体和微观脑梗塞。
Q3 Medicine Pub Date : 2023-01-01 Epub Date: 2018-07-26 DOI: 10.1080/24709360.2018.1500089
Ana W Capuano, Robert Wilson, Julie A Schneider, Sue E Leurgans, David A Bennett

Medical and epidemiological researchers commonly study ordinal measures of symptoms or pathology. Some of these studies involve two correlated ordinal measures. There is often an interest in including both measures in the modeling. It is common to see analyses that consider one of the measures as a predictor in the model for the other measure as outcome. There are, however, issues with these analyses including biased estimate of the probabilities and a decreased power due to multicollinearity (since they share some predictors). These issues create a necessity to examine both variables as simultaneous outcomes, by assessing the marginal probabilities for each outcome (i.e. using a proportional odds model) and the association between the two outcomes (i.e. using a constant global odds model). In this work we extend this model using a parsimonious option when the constraints imposed by assumptions of proportional marginal odds and constant global odds do not hold. We compare approaches by using simulations and by analyzing data on brain infarcts in older adults. Age at death is a marginal predictor of gross infarcts and also a marginal predictor of microscopic infarcts, but does not modify the association between gross and microscopic infarcts.

医学和流行病学研究人员通常研究症状或病理的顺序测量。其中一些研究涉及两个相关的序数测度。在建模中包含这两个度量通常是一种兴趣。常见的分析是,将其中一项指标视为模型中的预测指标,将另一项指标作为结果。然而,这些分析存在一些问题,包括概率的偏差估计和由于多重共线性而导致的功率下降(因为它们共享一些预测因子)。这些问题产生了将两个变量作为同时结果进行检查的必要性,通过评估每个结果的边际概率(即使用比例优势模型)和两个结果之间的关联(即使用恒定全局优势模型)。在这项工作中,当比例边际赔率和恒定全局赔率的假设所施加的约束不成立时,我们使用简约选项来扩展该模型。我们通过模拟和分析老年人脑梗死的数据来比较方法。死亡年龄是大体梗死的边缘预测因子,也是微观梗死的边缘预报因子,但不能改变大体梗死和微观梗死之间的关联。
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引用次数: 0
A marginal structural model for estimation of the effect of HIV positivity awareness on risky sexual behavior 估计HIV阳性意识对危险性行为影响的边际结构模型
Q3 Medicine Pub Date : 2022-07-03 DOI: 10.1080/24709360.2023.2171537
H. Twabi, Samuel O. M. Manda, D. Small, H. Kohler
In this paper, a Marginal Structural Model (MSM) with inverse probability of treatment weights was used to estimate the causal effect of HIV positivity awareness on condom use and multiple sexual partners using data from the Malawi Longitudinal Study of Families and Health (MLSFH). Cumulative awareness of HIV positivity was measured as the number of times an individual was aware of their positive HIV status. Awareness of HIV positivity was associated with increased condom use (OR=2.22, 95%: (1.79, 2.75)). Only among women was it associated with multiple sexual partners (OR=1.76, 95%: (1.36, 2.28)). The use of MSM (over standard regression models for repeated measures) should be encouraged as it is more suited for assessing the cumulative treatment effects while controlling for time-varying confounders in longitudinal studies. There is a need to up-scale interventions that promote HIV testing, awareness of HIV status, and prevention of HIV transmission.
在本文中,使用马拉维家庭与健康纵向研究(MLSFH)的数据,使用具有治疗权重逆概率的边际结构模型(MSM)来估计HIV阳性意识对避孕套使用和多个性伴侣的因果影响。对艾滋病毒阳性的累计认识是指一个人意识到自己的艾滋病毒阳性状况的次数。对HIV阳性的认识与避孕套使用的增加有关(OR=2.22,95%:(1.79,2.75))。只有在女性中,它与多个性伴侣有关(OR=1.76,95%:(1.36,2.28))。应鼓励使用MSM(重复测量的超标准回归模型),因为它更适合评估累积治疗效果,同时控制时变纵向研究中的混杂因素。有必要加大干预力度,促进艾滋病毒检测、对艾滋病毒状况的认识和预防艾滋病毒传播。
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引用次数: 0
Flexible and robust procedure for subgroup inference 子群推理的灵活鲁棒方法
Q3 Medicine Pub Date : 2022-07-03 DOI: 10.1080/24709360.2022.2127650
Ao Yuan, Anqi Yin, M. Tan
In subgroup analysis of clinical trials and precision medicine, it is important to assess the causal effect of a new treatment against an existing one and classify the new treatment favorable subgroup if it exists. As the original randomization does not apply to comparisons between subgroups, for unbiased estimate the causal inference method will be used, in particular the doubly robust procedure, in which a propensity score model and a regression model need to be specified. As long as one of the models is correctly specified, the causal effect will be estimated unbiased. However, it is known that any subjectively specified model more or less deviates from the true one, and so the doubly robust procedure may still not be robust. To overcome this issue, we apply a recently proposed method to allow the identification of subgroups and causal inference in subgroups. The model is a semiparametric robust and flexible procedure, in which both the propensity score model and the regression model are semiparametric, with monotone constraint on the nonparametric parts. Simulation studies are conducted to evaluate the performance of the proposed method and compare some existing methods. Then the method is applied to analyze a real clinical trial data.
在临床试验和精准医学的亚组分析中,评估新治疗方法与现有治疗方法的因果效应,并对存在的新治疗有利亚组进行分类是很重要的。由于最初的随机化并不适用于子组之间的比较,因此对于无偏估计,将使用因果推理方法,特别是双稳健过程,其中需要指定倾向得分模型和回归模型。只要其中一个模型是正确指定的,因果效应将被无偏估计。然而,众所周知,任何主观指定的模型或多或少都会偏离真实模型,因此双鲁棒过程仍然可能不具有鲁棒性。为了克服这个问题,我们应用了最近提出的一种方法来识别子组和子组中的因果推理。该模型是一种半参数鲁棒灵活过程,其中倾向分数模型和回归模型都是半参数模型,非参数部分具有单调约束。通过仿真研究对所提方法的性能进行了评价,并对现有方法进行了比较。并将该方法应用于实际临床试验数据的分析。
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引用次数: 1
The “exposure-based cross-sectional” study design: a novel observational study design applicable to rare exposures “基于暴露的横断面”研究设计:一种适用于罕见暴露的新型观察性研究设计
Q3 Medicine Pub Date : 2022-07-03 DOI: 10.1080/24709360.2022.2095244
J. Poorolajal
Current epidemiological studies are either inefficient or very expensive and time-consuming when the exposure of interest is very rare. The ‘exposure-based cross-sectional’ study is a new design that can overcome this problem. The ‘exposure-based cross-sectional’ study starts with exposed and unexposed groups. Then, these two groups are compared to determine what proportion of each group have the disease and what proportion do not. It is as if we were conducting a reversed case–control study in which the positions of the disease and exposures are altered. Dissimilar to retrospective cohort studies, the ‘exposure-based cross-sectional’ study does not depend on the basic existing records. This study measures the disease ‘prevalence’ rather than the disease ‘incidence’. The ‘exposure-based cross-sectional’ study design was examined in several real-life epidemiological studies with binary and continuous outcomes. The ‘exposure-based cross-sectional’ study is an efficient, inexpensive, expeditious, and easy to conduct study design for rare exposures. It can be performed for both binary and continuous outcomes.
目前的流行病学研究要么效率低下,要么在感兴趣的接触非常罕见的情况下非常昂贵和耗时。“基于暴露的横断面”研究是一种可以克服这一问题的新设计。“基于暴露的横断面”研究从暴露组和未暴露组开始。然后,将这两组进行比较,以确定每组中有多大比例的人患有该疾病,有多少比例的人没有。这就好像我们在进行一项反向病例对照研究,在该研究中,疾病的位置和暴露被改变了。与回顾性队列研究不同,“基于暴露的横断面”研究不依赖于现有的基本记录。这项研究衡量的是疾病的“流行率”,而不是疾病的“发病率”。“基于暴露的横断面”研究设计在几项具有二元和连续结果的现实流行病学研究中进行了检验。“基于暴露的横断面”研究是一种针对罕见暴露的高效、廉价、快速且易于进行的研究设计。它既可以用于二元结果,也可以用于连续结果。
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引用次数: 0
Propensity score-based adjustment for covariate effects on classification accuracy of bio-marker using ROC curve 利用ROC曲线对生物标志物分类准确性的协变量影响进行基于倾向性评分的调整
Q3 Medicine Pub Date : 2022-07-03 DOI: 10.1080/24709360.2022.2131994
Muntaha Mushfiquee, M. S. Rahman
The potential performance of bio-marker in classifying diseased from healthy population may be affected by baseline covariates (X) that are associated with both the bio-marker (Y) and the disease status (D). Some existing approaches can be able to adjust for the effect of a single covariate at a time. However, several potential covariates can be available in practice for which simultaneous adjustment in the ROC curve is essential. This study proposed a propensity score (PS) based adjustment for the effects of several covariates in the ROC curve. The PS is first derived from a linear transformation of several covariates and the PS-adjusted (and PS-specific) ROC curve was then estimated using the existing non-parametric induced ROC regression framework. The method is illustrated for both continuous and binary bio-markers. The simulation study suggests that the PS-based adjustment performed well by providing a consistent estimate of the true ROC curve and showing robustness to the mis-specification of the propensity score model as well as to a non-linear function of covariates. Further, an application of the method is provided to evaluate the effectiveness of the body-mass-index in classifying patients with hypertension or diabetes after adjusting for the potential covariates such as age, sex, education, socio-economic status.
生物标记物在从健康人群中分类疾病方面的潜在性能可能受到与生物标记物(Y)和疾病状态(D)相关的基线协变量(X)的影响。一些现有的方法可以一次调整单个协变量的影响。然而,在实践中可以获得几个潜在的协变量,ROC曲线的同时调整是必不可少的。本研究提出了一种基于倾向评分(PS)的ROC曲线中几个协变量影响的调整方法。PS首先从几个协变量的线性变换中导出,然后使用现有的非参数诱导ROC回归框架估计PS调整的(和PS特定的)ROC曲线。该方法对连续生物标记和二元生物标记都进行了说明。模拟研究表明,基于PS的调整表现良好,因为它提供了真实ROC曲线的一致估计,并对倾向评分模型的错误规范以及协变量的非线性函数表现出了稳健性。此外,在对年龄、性别、教育程度、社会经济地位等潜在协变量进行调整后,提供了该方法的应用,以评估体重指数在高血压或糖尿病患者分类中的有效性。
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引用次数: 0
期刊
Biostatistics and Epidemiology
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