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Propensity score weighting analysis with complex survey data for estimating population-level treatment effects on survival: a simulation study. 用复杂调查数据进行倾向评分加权分析,估计人群水平治疗对生存的影响:一项模拟研究。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-12-01 Epub Date: 2025-06-07 DOI: 10.1007/s10742-025-00344-x
Lihua Li, Chen Yang, Wei Zhang, Yulei He, John R Pleis, Lauren M Rossen, Bian Liu, Morgan Earp, Madhu Mazumdar

Propensity score weighting (PSW) is a valuable tool for estimating treatment effects on survival outcomes in observational studies. However, there is no clear best practice for applying PSW to complex survey data with survival outcomes. This paper addresses this gap by exploring how to integrate PSW into complex survey with design features (strata, clusters, sampling weights) for unbiased population-level estimates. We evaluate three PSW methods where: Method I: neither the propensity score (PS) model nor the outcome model accounts for the survey design; Method II: the PS model does not account for the survey design, but the outcome model does; Method III: both the PS model and outcome model account for the survey design. Through extensive simulations, we compare performance in estimating absolute treatment effects measured by population survival quantile effects and relative treatment effects measured by population marginal hazard ratios. Mean relative bias, mean absolute bias and coverage probability are estimated for model evaluations under various scenarios, including varying treatment effect magnitude, censoring type and rate, level of PS overlap, presence of outliers and nonresponse. Findings reveal that both survey-weighted Methods II and III outperform the unweighted Method I under most scenarios for both measures of treatment effects, especially when there is a true treatment effect. Both weighted methods II and III are found to perform closely, including when there exists informative censoring, influential outliers, or non-response. We recommend that when considering PSW with complex survey data for estimating population-level treatment effects on survival outcomes, both modelling stages should incorporate survey designs, but it is most critical for the outcome modelling. For illustration, all methods are applied to the public-use 2000-2018 National Health Interview Survey (NHIS) Linked to Mortality Files with mortality information through 2019 to estimate the effect of smoking cessation after a cancer diagnosis on subsequent overall survival.

在观察性研究中,倾向评分加权(PSW)是估计治疗对生存结果影响的一种有价值的工具。然而,对于将PSW应用于具有生存结果的复杂调查数据尚无明确的最佳实践。本文通过探索如何将PSW整合到具有设计特征(地层、聚类、抽样权重)的复杂调查中,以实现无偏总体水平估计,从而解决了这一差距。我们评估了三种PSW方法,其中:方法一:倾向得分(PS)模型和结果模型都不能解释调查设计;方法二:PS模型不考虑调查设计,但结果模型考虑了;方法三:采用PS模型和结果模型进行调查设计。通过广泛的模拟,我们比较了用种群生存分位数效应衡量绝对治疗效果和用种群边际风险比衡量相对治疗效果的表现。估计了不同情景下模型评估的平均相对偏倚、平均绝对偏倚和覆盖概率,包括不同的处理效果大小、审查类型和比率、PS重叠水平、异常值的存在和无反应。研究结果表明,在大多数情况下,对于两种治疗效果的测量,调查加权方法II和III都优于未加权方法I,特别是当存在真实的治疗效果时。发现加权方法II和III的表现都很接近,包括当存在信息审查、有影响力的异常值或无响应时。我们建议在考虑具有复杂调查数据的PSW以估计人群水平治疗对生存结果的影响时,两个建模阶段都应纳入调查设计,但这是结果建模最关键的。例如,所有方法都应用于公共使用的2000-2018年全国健康访谈调查(NHIS)与死亡率文件相关,其中包含到2019年的死亡率信息,以估计癌症诊断后戒烟对随后总体生存的影响。
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引用次数: 0
Difference-in-differences analysis with repeated cross-sectional survey data. 重复横断面调查数据的差异分析。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-10-11 DOI: 10.1007/s10742-025-00364-7
Kerry Ye, Alyssa Bilinski, Youjin Lee

Difference-in-differences (DiD) approach is one of the most widely used approaches for evaluating policy effects. However, traditional DiD methods may not recover the population-level average treatment effect on the treated (ATT) in the absence of population-level panel data, particularly when the composition of units in the treatment group changes over time. In this work, we address the following two challenges when applying DiD methods with repeated cross-sectional (RCS) survey data: (1) heterogeneous compositions of study samples across different time points, and (2) availability of data for only a sample of the population. We introduce a policy-relevant target estimand and establish its identification conditions. We then propose a new weighting approach that incorporates both estimated propensity scores and given survey weights. We establish the theoretical properties of the proposed method and examine its finite-sample performance through simulations. Finally, we apply our proposed method to a real-world data application, estimating the effect of a beverage tax on adolescent soda consumption in Philadelphia.

差异中的差异(DiD)方法是评估政策效果最广泛使用的方法之一。然而,在缺乏人口水平面板数据的情况下,传统的DiD方法可能无法恢复对被治疗者(ATT)的人口水平平均治疗效果,特别是当治疗组的单位组成随时间变化时。在这项工作中,我们解决了在重复横断面(RCS)调查数据中应用DiD方法时面临的以下两个挑战:(1)不同时间点研究样本的异质性组成,以及(2)只有人口样本的数据可用性。引入了一个与政策相关的目标估计,并建立了其识别条件。然后,我们提出了一种新的加权方法,该方法结合了估计的倾向得分和给定的调查权重。我们建立了该方法的理论性质,并通过仿真检验了其有限样本性能。最后,我们将我们提出的方法应用到一个真实世界的数据应用中,估计了饮料税对费城青少年苏打水消费的影响。
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引用次数: 0
The role of spatially varying loadings in dynamic spatial factor models for modeling the opioid syndemic. 空间变化负荷在阿片综合征动态空间因子模型中的作用。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-09-01 Epub Date: 2025-09-16 DOI: 10.1007/s10742-025-00356-7
Eva Murphy, David Kline, Staci A Hepler

Understanding the interactions and spatio-temporal variations of public health outcomes is crucial for gaining insight into interrelated epidemics across different locations and time periods. Dynamic spatial factor models provide a flexible framework for capturing shared variability among multiple outcomes through a latent factor and its corresponding loadings. A common assumption in these models is that factor loadings are spatially constant, implying uniform relationships between outcomes across the study region. However, this assumption may overlook important regional differences in how outcomes relate to the underlying latent factor. In this study, we derive the covariance structure of the outcome vector to highlight how spatially varying versus constant loadings influence the overall correlation structure. We find that when loadings vary across space, the spatial covariance of the outcomes is shaped by both the spatial covariance of the loadings and the latent factors. In contrast, when loadings are spatially constant, the spatial covariance of the outcomes is determined primarily by the latent factors, leading to uniform variation across the spatial domain. To assess these differences in practice, we apply a Bayesian hierarchical spatial dynamic factor model to analyze the opioid syndemic in North Carolina. Our results suggest that incorporating spatially varying loadings provides a more detailed, county-specific understanding of the epidemic. This added flexibility enables a localized interpretation of opioid-related interactions and offers insights that could inform targeted public health interventions.

了解公共卫生结果的相互作用和时空变化对于深入了解不同地点和时间段的相互关联的流行病至关重要。动态空间因子模型提供了一个灵活的框架,通过潜在因子及其相应的负载来捕获多个结果之间的共同变异性。这些模型的一个共同假设是,因子负荷在空间上是恒定的,这意味着整个研究区域的结果之间的关系是一致的。然而,这种假设可能忽略了结果与潜在因素之间的重要区域差异。在本研究中,我们推导了结果向量的协方差结构,以突出空间变化与恒定负荷如何影响整体相关结构。研究发现,当负荷跨空间变化时,结果的空间协方差由负荷的空间协方差和潜在因素共同塑造。相反,当负荷在空间上恒定时,结果的空间协方差主要由潜在因素决定,导致整个空间域的均匀变化。为了在实践中评估这些差异,我们应用贝叶斯层次空间动态因子模型来分析北卡罗来纳州的阿片类药物综合征。我们的研究结果表明,结合空间变化的负荷可以更详细地了解该流行病的具体情况。这种增加的灵活性使阿片类药物相关相互作用的本地化解释成为可能,并为有针对性的公共卫生干预提供信息。
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引用次数: 0
The impact of knowledge of hospitalization on mortality predictions. 住院知识对死亡率预测的影响。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-06-13 DOI: 10.1007/s10742-025-00348-7
Chuanling Qin, Curtis Peterson, William B Weeks, A James O'Malley

Despite the rapid advancement of machine learning algorithms, the important problem of distinguishing patients based on the likelihood of their mortality remains a challenge. In this paper, we investigated the degree to which the incorporation of the time-varying factor, length of hospitalization could contribute to modeling mortality. A two-part modeling approach was proposed to capture the potential heterogeneity over follow-up time and to evaluate the extent to which allowing a predictor based on a fixed-time event like hospitalization (as a time-varying coefficient) enhanced mortality prediction. A test was then conducted to assess whether the association between hospitalization and mortality diminished with continued survival of a patient. Leveraging logistic regression models and the XGBoost procedure, the findings supported the claim that the baseline hospitalization is a risk factor whose importance diminishes the longer the patient survives. While simulation studies and theoretical considerations indicate that the two-part model provides deeper insight into the evolving dynamics of regression coefficients and enhances the prediction accuracy of the marginal probability of mortality, its application to the empirical data that motivated this research yielded less compelling results, a finding that aligns with previous findings. Factors such as class imbalance and the magnitude of heterogeneous effects can significantly impact the performance of the two-part model in empirical datasets.

尽管机器学习算法发展迅速,但基于死亡可能性区分患者的重要问题仍然是一个挑战。在本文中,我们研究了纳入时变因素、住院时间长短对建模死亡率的影响程度。提出了一种两部分建模方法,以捕捉随访时间内的潜在异质性,并评估基于住院等固定时间事件(作为时变系数)的预测器增强死亡率预测的程度。然后进行了一项测试,以评估住院和死亡率之间的关联是否随着患者的持续生存而减弱。利用逻辑回归模型和XGBoost程序,研究结果支持了基线住院是一个风险因素,其重要性随着患者存活时间的延长而降低的说法。虽然模拟研究和理论考虑表明,两部分模型可以更深入地了解回归系数的演变动态,并提高死亡率边际概率的预测精度,但将其应用于推动本研究的经验数据时,结果并不令人信服,这一发现与先前的研究结果一致。类不平衡和异质性效应的大小等因素会显著影响两部分模型在经验数据集上的表现。
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引用次数: 0
Combining multiple sources of relationships in a network to advance understanding of physicians' beliefs regarding peer-effects. 在一个网络中结合多种关系来源,以促进对医生关于同伴效应的信念的理解。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-03-20 DOI: 10.1007/s10742-025-00343-y
Yifan Zhao, Carly A Bobak, Megan A Murphy, Olivia Sacks, Lili Liu, Natasha Ray, Amber E Barnato, A James O'Malley

Patient-sharing physician networks are increasingly recognized as valuable tools for examining physician relationships in healthcare research. However, very few studies have examined the reliability of such networks and summary measures derived from them in relation to directly measured physician relationships. In this paper, we evaluate the level of congruence between a survey-based network derived from survey responses to specific name-generator questions and a patient-sharing network derived from claims data. We also examine the association of summary measures derived from either network with physicians' beliefs about peer influence in medical practice. Statistical models with hierarchical and multiple-membership structures were used to estimate the strength of the associations. We found that a survey measure indicating whether a physician was nominated by others was statistically significantly associated with their survey reported beliefs about peer influence. We also observed notable associations between the physicians' structural importance in the network reflected in their eigenvector and betweenness centrality in the patient-sharing network and their beliefs about peer influence. This study of multi-source network relational information advances our understanding of physician survey responses and yields more precise predictions of physician beliefs toward peer-influence than either data source alone. Overall, we found that patient-sharing networks are an important alternative to directly measured survey-based name-generator questions in health services research and other applications. While patient-sharing networks recover some of the information in directly measured peer physician nominations, they also contain distinct information that is helpful for interpreting healthcare insights.

在医疗保健研究中,患者共享医生网络越来越被认为是检查医生关系的有价值的工具。然而,很少有研究检验了这些网络的可靠性,以及从中得出的与直接测量的医生关系相关的总结措施。在本文中,我们评估了基于调查的网络之间的一致性水平,这些网络来自对特定名称生成器问题的调查回应,而患者共享网络来自索赔数据。我们还研究了来自任一网络的总结措施与医生在医疗实践中对同伴影响的信念的关联。使用分层和多成员结构的统计模型来估计关联的强度。我们发现,一项表明医生是否被他人提名的调查测量结果在统计上显著地与他们的调查报告中关于同伴影响的信念相关。我们还观察到,医生在网络中的结构重要性反映在他们的特征向量和患者共享网络中的中间性中心性和他们对同伴影响的信念之间存在显著的关联。这项多来源网络关系信息的研究促进了我们对医生调查反应的理解,并比单独的任何一个数据源更准确地预测了医生对同行影响的信念。总的来说,我们发现在医疗服务研究和其他应用中,患者共享网络是直接测量的基于调查的名称生成器问题的重要替代方案。虽然患者共享网络在直接测量的同行医生提名中恢复了一些信息,但它们也包含有助于解释医疗保健见解的独特信息。
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引用次数: 0
Practical challenges in mediation analysis: a guide for applied researchers. 中介分析中的实际挑战:应用研究者指南。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-01 Epub Date: 2024-04-12 DOI: 10.1007/s10742-024-00327-4
Megan S Schuler, Donna L Coffman, Elizabeth A Stuart, Trang Q Nguyen, Brian Vegetabile, Daniel F McCaffrey

Mediation analysis is a statistical approach that can provide insights regarding the intermediary processes by which an intervention or exposure affects a given outcome. Mediation analyses rose to prominence, particularly in social science research, with the publication of Baron and Kenny's seminal paper and is now commonly applied in many research disciplines, including health services research. Despite the growth in popularity, applied researchers may still encounter challenges in terms of conducting mediation analyses in practice. In this paper, we provide an overview of conceptual and methodological challenges that researchers face when conducting mediation analyses. Specifically, we discuss the following key challenges: (1) Conceptually differentiating mediators from other "third variables," (2) Extending beyond the single mediator context, (3) Identifying appropriate datasets in which measurement and temporal ordering support the hypothesized mediation model, (4) Selecting mediation effects that reflect the scientific question of interest, (5) Assessing the validity of underlying assumptions of no omitted confounders, (6) Addressing measurement error regarding the mediator, and (7) Clearly reporting results from mediation analyses. We discuss each challenge and highlight ways in which the applied researcher can approach these challenges.

中介分析是一种统计方法,可以提供有关干预或暴露影响给定结果的中介过程的见解。随着巴伦和肯尼的开创性论文的发表,调解分析开始崭露头角,尤其是在社会科学研究领域。现在,调解分析被广泛应用于许多研究学科,包括卫生服务研究。尽管越来越受欢迎,但应用研究人员在实践中进行中介分析方面仍可能遇到挑战。在本文中,我们概述了研究人员在进行中介分析时面临的概念和方法上的挑战。具体来说,我们将讨论以下主要挑战:(1)从概念上区分中介与其他“第三变量”,(2)超越单一中介上下文,(3)确定适当的数据集,其中测量和时间顺序支持假设的中介模型,(4)选择反映感兴趣的科学问题的中介效应,(5)评估没有遗漏混杂因素的潜在假设的有效性,(6)解决有关中介的测量误差,以及(7)清楚地报告中介分析的结果。我们将讨论每个挑战,并强调应用研究人员可以应对这些挑战的方法。
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引用次数: 0
Diabetes Belt has lower efficiency in providing diabetes preventive care than surrounding counties. 糖尿病带提供糖尿病预防保健的效率低于周边县。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-06-01 Epub Date: 2023-08-09 DOI: 10.1007/s10742-023-00310-5
Hyojung Kang, Min-Woong Sohn, Soyoun Kim, Siyao Zhang, Rajesh Balkrishnan, Roger Anderson, Anthony McCall, Timothy McMurry, Jennifer Mason Lobo

Annual preventive care is essential for diabetes patients to reduce the risk of complications including hypoglycemic events and blindness. Our aim was to examine the relative efficiency of Diabetes Belt (DB) and non-Diabetes Belt (NDB) counties in providing recommended preventive care for Medicare beneficiaries with diabetes using available health professional resources and to understand county-level socioeconomic factors associated with inefficient provision of preventive care. A data envelopment analysis (DEA) model was developed to assess relative efficiency of counties in providing diabetes preventive care. Logistic regression was performed to identify socioeconomic characteristics associated with inefficiencies. We used Medicare claims data to extract individual-level information of diabetes preventive service use and obtained county-level estimates of health resources information from the Area Health Resources File. More than 80% of counties had more than 10% inefficiencies on average. Compared to counties in the NDB, the odds of being inefficient were 2.44 times more likely in the DB (OR 2.44, CI 1.67-3.58). Counties with lower median income, with a smaller proportion of non-Hispanic Black population, and in a rural area had higher odds of being inefficient in providing preventive care. Our DEA results showed that counties in the DB and NDB were mostly inefficient. The availability of care providers may be less of a problem than how efficiently the resources are used in providing preventive care. Identifying sources of inefficiency within each community with low resource utilization and developing targeted strategies is needed to improve uptake of preventive care cost-effectively.

对于糖尿病患者来说,每年的预防性保健对于降低低血糖事件和失明等并发症的风险至关重要。我们的目的是检查糖尿病带(DB)和非糖尿病带(NDB)县在利用现有卫生专业资源为糖尿病医疗保险受益人提供推荐的预防保健方面的相对效率,并了解与预防保健提供效率低下相关的县级社会经济因素。采用数据包络分析(DEA)模型评估各县提供糖尿病预防护理的相对效率。进行逻辑回归以确定与效率低下相关的社会经济特征。我们使用医疗保险索赔数据提取糖尿病预防服务使用的个人水平信息,并从区域卫生资源文件中获得县级卫生资源信息估计。超过80%的县的平均低效率超过10%。与新开发银行的国家相比,低效率的可能性是新开发银行的2.44倍(OR 2.44, CI 1.67-3.58)。收入中位数较低、非西班牙裔黑人人口比例较小以及农村地区的县在提供预防保健方面效率较低的可能性更高。我们的DEA结果显示,在DB和NDB的县大多效率低下。保健提供者的可用性可能不是一个问题,而是如何有效地利用资源来提供预防性保健。需要在每个资源利用率低的社区内确定效率低下的根源,并制定有针对性的战略,以经济有效地提高预防保健的接受程度。
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引用次数: 0
Evaluation via simulation of statistical corrections for network nonindependence. 通过模拟评估网络非独立性的统计修正。
IF 1.6 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2024-06-01 Epub Date: 2023-08-12 DOI: 10.1007/s10742-023-00311-4
Luke J Matthews, Megan S Schuler, Raffaele Vardavas, Joshua Breslau, Ioana Popescu

Social processes and social context are increasingly recognized as key factors shaping health-related behaviors and outcomes. One social process that may be acting within social networks is social influence, in which an individual's characteristic (e.g., specific health behavior) is potentially impacted by the corresponding characteristic of connected individuals in the network. In the health services context, healthcare providers who work together and share patients may influence each other through the knowledge transmission or development of clinical practice norms. Although many statistical techniques assume independence of data points, when analyzing data that may reflect social processes acting across a social network, it is imperative to account for the interdependencies (i.e., non-independence) across individuals. In practice, studies account for nonindependence in the context of estimating bivariate relations (correlations or linear regression) using a variety of analytic methods (some of which have previously been shown to yield biased results). To date, it is unclear which methods yield acceptable false positive rates, unbiased coefficient estimates, and acceptable statistical power, because there have been no systematic simulation studies comparing methods for addressing network nonindependence arising from social influence. To address this gap, we compared eight commonly used methods that purport to account for nonindependence using simulated network data. While results indicated that none of the techniques reduced false positive rates to the predicted (nominal) 0.05 level, random sampling of network nodes was the method that yielded the smallest false positive rates, yet came at a price of reduced statistical power. Further methodological development is needed.

社会过程和社会背景日益被认为是形成与健康有关的行为和结果的关键因素。可能在社会网络中起作用的一个社会过程是社会影响,其中个人的特征(例如,特定的健康行为)可能受到网络中连接个体的相应特征的影响。在卫生服务方面,共同工作和分享病人的卫生保健提供者可能通过知识传播或临床实践规范的发展相互影响。虽然许多统计技术假设数据点的独立性,但在分析可能反映跨社会网络的社会过程的数据时,必须考虑到个体之间的相互依赖性(即非独立性)。在实践中,研究使用各种分析方法(其中一些先前已被证明产生有偏差的结果)来估计双变量关系(相关性或线性回归)的背景下解释非独立性。迄今为止,尚不清楚哪些方法产生可接受的假阳性率、无偏系数估计和可接受的统计能力,因为没有系统的模拟研究比较解决由社会影响引起的网络非独立性的方法。为了解决这一差距,我们比较了八种常用的方法,这些方法声称使用模拟网络数据来解释非独立性。虽然结果表明,没有一种技术将假阳性率降低到预测(名义)0.05水平,但网络节点的随机抽样是产生最小假阳性率的方法,但其代价是降低了统计能力。需要进一步发展方法。
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引用次数: 0
ATRAcTR (Authentic Transparent Relevant Accurate Track-Record): a screening tool to assess the potential for real-world data sources to support creation of credible real-world evidence for regulatory decision-making ATRAcTR(真实透明的相关准确跟踪记录):一种筛选工具,用于评估真实世界数据来源的潜力,以支持为监管决策创建可信的真实世界证据。
IF 1.5 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2023-11-29 DOI: 10.1007/s10742-023-00319-w
Marc L. Berger, William H. Crown, Jim Z. Li, Kelly H. Zou
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引用次数: 0
Evaluation of survey delivery methods in a national study of Veteran’s healthcare preferences 退伍军人医疗保健偏好全国研究中的调查方法评估
IF 1.5 Q3 HEALTH CARE SCIENCES & SERVICES Pub Date : 2023-11-28 DOI: 10.1007/s10742-023-00320-3
N. Disher, Jennifer Scott, Anna Tyzik, S. Golden, Georgia Baker, Denise M. Hynes, C. Slatore
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引用次数: 0
期刊
Health Services and Outcomes Research Methodology
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