Observational studies

观察性研究
  • 文章类型: Journal Article
    背景:基于实践的研究网络(PBRN)研究通常在与现实条件相匹配的环境中评估临床干预措施的有效性。牙科PBRN有能力聚集,identify,并分析来自不同种族的患者人群的大量数据,民族,社会经济,和地理背景。这些牙科研究涵盖了广泛的医疗保健方面,包括预防,诊断,症状和疾病治疗,提高质量,护理协调。
    方法:可以在牙科PBRN中采用广泛的研究方法来研究这些主题,包括随机对照试验。牙科PBRN已经从主要关注病例观察发展到利用先进的网络基础设施并在多个区域和国家站点之间进行合作。除了产生许多高影响力的同行评审出版物外,研究结果改善了临床护理。然而,PBRN遇到挑战,例如研究能力的可持续性(严重依赖资助机构的持续支持),不同的研究文化,并且必须设计既可行又与日常临床实践相关的研究。认识到现实世界证据的关键作用,重要的是对牙科PBRN基础设施的持续投资以及从业者参与全国网络活动的可行机会。
    结论:基于实践的研究网络研究利用了一个重要的研究背景,在这个背景下研究了一系列可以采用多种研究方法的临床主题。然而,维持生产网络需要战略努力,持续的财政支持,和定制的组织技能。
    BACKGROUND: Practice-Based Research Network (PBRN) studies typically assess the effectiveness of clinical interventions in settings that match real-world conditions. Dental PBRNs have the capacity to amass, identify, and analyze vast amounts of data from patient populations that include diverse racial, ethnic, socio-economic, and geographic backgrounds. These dental studies encompass a broad spectrum of healthcare aspects, including prevention, diagnosis, symptom and disease treatment, quality enhancement, and care coordination.
    METHODS: An extensive range of research methodologies can be employed within dental PBRNs to investigate these topics, including randomized controlled trials. Dental PBRNs have evolved from primarily focusing on case observations to leveraging advanced network infrastructure and collaborating across multiple regional and national sites. In addition to producing numerous high-impact peer-reviewed publications, study results have led to improved clinical care. However, PBRNs encounter challenges, such as the sustainability of research capacity (relying heavily on ongoing support from funding agencies), diverse research cultures, and an imperative to design studies that are both feasible and relevant to everyday clinical practice. Recognizing the pivotal role of real-world evidence, it is important to have sustained investment in dental PBRN infrastructure and feasible opportunities for practitioners to participate in network activities nationwide.
    CONCLUSIONS: Practice-Based Research Network studies capitalize on an important research context within which to investigate a range of clinical topics that can employ multiple research methodologies. However, sustaining productive networks requires strategic effort, ongoing financial support, and customized organizational skills.
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  • 文章类型: Journal Article
    目的:用于数据的元数据欧洲药品管理局资助的项目(EUPAS39322),定义了一组元数据来描述现实世界的数据源(RWDS),并在原型目录中试行了元数据收集,以帮助调查人员通过研究进行数据源的可发现性。
    方法:元数据列表是根据对现有元数据目录和建议的审查而创建的,结构化面试,利益相关者调查,和技术研讨会。原型的设计符合FAIR原则(可找到,可访问,可互操作,可重用),使用MOLGENIS软件。元数据收集由来自欧洲各地的15个数据访问合作伙伴(DAP)进行试点。
    结果:总共在六个领域中定义了442个元数据变量:机构(连接到数据源的组织);数据库(由组织维持的数据收集);数据源(涵盖共同基础人群的可链接数据库的集合);研究;(机构)网络;和通用数据模型(CDM)。原型中总共记录了26个机构。每个DAP填充一个数据源及其所选数据库的元数据。数据库的数量因数据源而异;最常见的数据库是医院管理记录和药房分配记录(每个10个数据源)。从符合不同CDM的三个数据源中成功提取了定量元数据,并将其输入到原型中。
    结论:最终确定了元数据列表,一个原型被成功填充,并制定了良好的实践指南。建立和维护RWDS的元数据目录将需要大量努力来支持数据源的可发现性和欧洲研究的可重复性。
    OBJECTIVE: Metadata for data dIscoverability aNd study rEplicability in obseRVAtional studies (MINERVA), a European Medicines Agency-funded project (EUPAS39322), defined a set of metadata to describe real-world data sources (RWDSs) and piloted metadata collection in a prototype catalogue to assist investigators from data source discoverability through study conduct.
    METHODS: A list of metadata was created from a review of existing metadata catalogues and recommendations, structured interviews, a stakeholder survey, and a technical workshop. The prototype was designed to comply with the FAIR principles (findable, accessible, interoperable, reusable), using MOLGENIS software. Metadata collection was piloted by 15 data access partners (DAPs) from across Europe.
    RESULTS: A total of 442 metadata variables were defined in six domains: institutions (organizations connected to a data source); data banks (data collections sustained by an organization); data sources (collections of linkable data banks covering a common underlying population); studies; networks (of institutions); and common data models (CDMs). A total of 26 institutions were recorded in the prototype. Each DAP populated the metadata of one data source and its selected data banks. The number of data banks varied by data source; the most common data banks were hospital administrative records and pharmacy dispensation records (10 data sources each). Quantitative metadata were successfully extracted from three data sources conforming to different CDMs and entered into the prototype.
    CONCLUSIONS: A metadata list was finalized, a prototype was successfully populated, and a good practice guide was developed. Setting up and maintaining a metadata catalogue on RWDSs will require substantial effort to support discoverability of data sources and reproducibility of studies in Europe.
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  • 文章类型: Journal Article
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  • 文章类型: Journal Article
    这份手稿总结了第一作者在2024年卡尔文·施瓦贝兽医流行病学和预防医学终身成就奖研讨会上发表的演讲,授予JanSargeant博士.流行病学研究在理解暴露与健康结果之间的复杂关系中起着至关重要的作用。然而,从这些调查中得出的结论的准确性取决于对暴露变量的精心选择和测量。适当的暴露变量选择对于了解疾病病因至关重要,但通常情况下,我们无法直接测量感兴趣的风险敞口变量,而是使用代理度量来评估风险敞口。不适当地使用代理措施可能会导致对真正的利益暴露做出错误的结论。这些错误可能导致对风险敞口和结果之间关联的估计有偏差。这种偏见的后果超出了研究的关注,因为可以根据有缺陷的证据做出健康决定。认识到并减轻这些偏见对于提供可靠的证据来指导卫生政策和干预措施至关重要。最终有助于改善人口健康结果。为了应对这些挑战,研究人员必须采用严格的方法进行暴露变量选择和验证研究,以最大程度地减少测量误差。
    This manuscript summarizes a presentation delivered by the first author at the 2024 symposium for the Calvin Schwabe Award for Lifetime Achievement in Veterinary Epidemiology and Preventive Medicine, which was awarded to Dr. Jan Sargeant. Epidemiologic research plays a crucial role in understanding the complex relationships between exposures and health outcomes. However, the accuracy of the conclusions drawn from these investigations relies upon the meticulous selection and measurement of exposure variables. Appropriate exposure variable selection is crucial for understanding disease etiologies, but it is often the case that we are not able to directly measure the exposure variable of interest and use proxy measures to assess exposures instead. Inappropriate use of proxy measures can lead to erroneous conclusions being made about the true exposure of interest. These errors may lead to biased estimates of associations between exposures and outcomes. The consequences of such biases extend beyond research concerns as health decisions can be made based on flawed evidence. Recognizing and mitigating these biases are essential for producing reliable evidence that informs health policies and interventions, ultimately contributing to improved population health outcomes. To address these challenges, researchers must adopt rigorous methodologies for exposure variable selection and validation studies to minimize measurement errors.
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  • 文章类型: Journal Article
    目的:已经报道了系统性硬化症(SSc)和SSc间质性肺病(ILD)的表现和结局的种族和种族差异。然而,先前的研究限制了多样性。我们的目标是评估是否存在与ILD相关的种族/民族差异,SSc和ILD之间的时间间隔以及急诊科(ED)就诊或住院率。
    方法:从综合卫生系统的纵向健康记录中提取了756例SSc患者的临床和社会人口统计学变量。Logistic回归模型分析了SSc-ILD时协变量与ILD和年龄的关联。使用互补的对数-对数回归模型分析医疗保健结果。
    结果:总体而言,该队列中33.7%的患者有ILD代码,亚洲人的赔率增加(赔率比[OR],2.60;95%置信区间[CI],1.29-5.28;p=0.008)与白人患者相比。西班牙裔的SSc-ILD年数的预测年龄较年轻(估计,-6.5;95%CI,-13--0.21;p=0.04)和黑人/非裔美国人患者(-10;95%CI-16--4.9;p<0.001)与白人患者相比。黑人/非洲裔美国患者更有可能在SSc代码之前有ILD代码(59%,白人患者为20.6%),从SSc到ILD的最短间隔(3个月)。黑人/非裔美国人(HR,2.59;95%CI1.47-4.49;p=0.001)和西班牙裔患者(HR2.29;95%CI1.37-3.82;p=0.002)的ED就诊率较高。
    结论:我们发现SSc-ILD的几率因种族/民族而异,小型化患者的就诊年龄较早,和更高的ED访问率。
    OBJECTIVE: Racial and ethnic differences in presentation and outcomes have been reported in systemic sclerosis (SSc) and SSc-interstitial lung disease (ILD). However, prior studies have limited diversity. We aim to evaluate if there are racial/ethnic differences associated with ILD, time intervals between SSc and ILD and with emergency department (ED) visit or hospitalization rates.
    METHODS: Clinical and sociodemographic variables were extracted for 756 patients with SSc from longitudinal health records in an integrated health-system. Logistic regression models analyzed the association of covariates with ILD and age at SSc-ILD. Healthcare outcomes were analyzed with complementary log-log regression models.
    RESULTS: Overall, 33.7% of patients in the cohort had an ILD code, with increased odds for Asian (odds ratio [OR], 2.60; 95% confidence interval [CI], 1.29-5.28; p=0.008) compared with White patients. The predicted age in years of SSc-ILD was younger for Hispanic (estimate, -6.5; 95% CI, -13--0.21; p = 0.04) and Black/African American patients (-10; 95% CI -16--4.9; p < 0.001) compared with White patients. Black/African American patients were more likely to have an ILD code before an SSc code (59% compared with 20.6% of White patients), and the shortest interval from SSc to ILD (3 months). Black/African American (HR, 2.59; 95% CI 1.47-4.49; p = 0.001) and Hispanic patients (HR 2.29; 95% CI 1.37- 3.82; p = 0.002) had higher rates of an ED visit.
    CONCLUSIONS: We found that odds of SSc-ILD differed by racial/ethnic group, minoritized patients had earlier age of presentation, and greater rates of an ED visit.
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  • 文章类型: Journal Article
    背景:尽管大多数网络荟萃分析(NMA)使用来自随机临床试验(RCT)的汇总数据(AD),其他研究设计(例如,队列研究和其他非随机研究,NRS)可以提供有关相对治疗效果的信息。研究的个体参与者数据(IPD),当可用时,对于调整重要的参与者特征以及更好地处理网络中的异质性和不一致性,都优于AD。
    结果:我们开发了R包crossnma,以执行交叉格式(IPD和AD)和交叉设计(RCT和NRS)NMA和网络元回归(NMR)。在R环境中使用另一个吉布斯采样器(JAGS)软件将模型实现为贝叶斯三级分层模型。R包crossnma包含自动创建JAGS模型的函数,重新格式化数据(基于用户输入),评估收敛性并总结结果。我们通过使用六个比较四个治疗方法的试验网络来证明Crosnma内的工作流程。
    结论:R包crossnma使用户能够在贝叶斯框架中使用不同数据类型执行NMA和NMR,并有助于纳入所有类型的证据,以识别偏差风险的差异。
    BACKGROUND: Although aggregate data (AD) from randomised clinical trials (RCTs) are used in the majority of network meta-analyses (NMAs), other study designs (e.g., cohort studies and other non-randomised studies, NRS) can be informative about relative treatment effects. The individual participant data (IPD) of the study, when available, are preferred to AD for adjusting for important participant characteristics and to better handle heterogeneity and inconsistency in the network.
    RESULTS: We developed the R package crossnma to perform cross-format (IPD and AD) and cross-design (RCT and NRS) NMA and network meta-regression (NMR). The models are implemented as Bayesian three-level hierarchical models using Just Another Gibbs Sampler (JAGS) software within the R environment. The R package crossnma includes functions to automatically create the JAGS model, reformat the data (based on user input), assess convergence and summarize the results. We demonstrate the workflow within crossnma by using a network of six trials comparing four treatments.
    CONCLUSIONS: The R package crossnma enables the user to perform NMA and NMR with different data types in a Bayesian framework and facilitates the inclusion of all types of evidence recognising differences in risk of bias.
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  • 文章类型: Journal Article
    克拉屈滨与其他有效的多发性硬化症(MS)免疫疗法之间的比较缺乏。
    为了比较克拉屈滨与芬戈莫德的疗效,那他珠单抗,奥克瑞珠单抗和阿仑珠单抗治疗复发缓解型MS。
    接受克拉屈滨治疗的复发缓解型MS患者,芬戈莫德,那他珠单抗,在全球MSBase队列和另外两个英国中心中发现了奥克瑞珠单抗或阿仑珠单抗.患者被跟踪6/12,并有3个人残疾评估。使用倾向评分对患者进行匹配。四个成对分析比较了年复发率(ARR)和残疾结果。
    符合条件的队列包括853(芬戈莫德),464(那他珠单抗),1131(奥克雷珠单抗),123例(阿仑单抗)或493例(克拉屈滨)患者。克拉屈滨的ARR低于芬戈莫德(0.07vs.0.12,p=0.006),ARR高于那他珠单抗(0.10vs.0.06,p=0.03),奥克瑞珠单抗(0.09vs.0.05,p=0.008)和阿仑珠单抗(0.17vs.0.04,p<0.001)。与克拉屈滨相比,使用芬戈莫德(风险比(HR)1.08,95%置信区间(CI)0.47-2.47)或阿仑珠单抗(HR0.73,95%CI0.26-2.07)治疗的患者的残疾恶化风险没有差异,但在接受那他珠单抗(HR0.35,95%CI0.13-0.94)和奥克瑞珠单抗(HR0.45,95%CI0.26-0.78)治疗的患者中更低.没有证据表明残疾改善有差异。
    克拉屈滨是一种有效的治疗方法,可以看作是芬戈莫德的有效性提高,但效果不如最有效的静脉MS疗法。
    UNASSIGNED: Comparisons between cladribine and other potent immunotherapies for multiple sclerosis (MS) are lacking.
    UNASSIGNED: To compare the effectiveness of cladribine against fingolimod, natalizumab, ocrelizumab and alemtuzumab in relapsing-remitting MS.
    UNASSIGNED: Patients with relapsing-remitting MS treated with cladribine, fingolimod, natalizumab, ocrelizumab or alemtuzumab were identified in the global MSBase cohort and two additional UK centres. Patients were followed for ⩾6/12 and had ⩾3 in-person disability assessments. Patients were matched using propensity score. Four pairwise analyses compared annualised relapse rates (ARRs) and disability outcomes.
    UNASSIGNED: The eligible cohorts consisted of 853 (fingolimod), 464 (natalizumab), 1131 (ocrelizumab), 123 (alemtuzumab) or 493 (cladribine) patients. Cladribine was associated with a lower ARR than fingolimod (0.07 vs. 0.12, p = 0.006) and a higher ARR than natalizumab (0.10 vs. 0.06, p = 0.03), ocrelizumab (0.09 vs. 0.05, p = 0.008) and alemtuzumab (0.17 vs. 0.04, p < 0.001). Compared to cladribine, the risk of disability worsening did not differ in patients treated with fingolimod (hazard ratio (HR) 1.08, 95% confidence interval (CI) 0.47-2.47) or alemtuzumab (HR 0.73, 95% CI 0.26-2.07), but was lower for patients treated with natalizumab (HR 0.35, 95% CI 0.13-0.94) and ocrelizumab (HR 0.45, 95% CI 0.26-0.78). There was no evidence for a difference in disability improvement.
    UNASSIGNED: Cladribine is an effective therapy that can be viewed as a step up in effectiveness from fingolimod, but is less effective than the most potent intravenous MS therapies.
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  • 文章类型: Journal Article
    许多退伍军人从非VA机构获得退伍军人事务部(VA)购买的护理,但对退伍军人考虑这种选择的因素知之甚少。在2020年5月至2021年8月之间,我们调查了VA购买的符合护理资格的VA患者,了解选择在哪里接受护理的障碍和促进者。我们检查了前往VA设施的旅行时间与他们选择VA购买的护理(在非VA环境中接受的VA付费医疗保健)与VA设施之间的关联,以及这种关联是否因不信任而改变。我们收到了1,662份回复,692份(42%)选择了VA设施。80%的人表示,优质护理是影响他们决定的三大因素。不信任度最高且距最近的VA设施>1小时的受访者选择VA的预测概率(PP)最低(PP15%;95%置信区间:10%-20%)。退伍军人重视护理质量。VA和其他医疗保健系统应考虑以患者为中心的方法来改善和宣传质量并减少不信任。
    Many Veterans receive Department of Veterans Affairs (VA)-purchased care from non-VA facilities but little is known about factors that Veterans consider for this choice. Between May 2020 and August 2021, we surveyed VA-purchased care-eligible VA patients about barriers and facilitators to choosing where to receive care. We examined the association between travel time to their VA facility and their choice of VA-purchased care (VA-paid health care received in non-VA settings) versus VA facility and whether this association was modified by distrust. We received 1,662 responses and 692 (42%) chose a VA facility. Eighty percent reported quality care was in their top three factors that influenced their decision. Respondents with the highest distrust and who lived >1 hr from the nearest VA facility had the lowest predicted probability (PP) of choosing VA (PP 15%; 95% confidence interval: 10%-20%). Veterans value quality of care. VA and other health care systems should consider patient-centered ways to improve and publicize quality and reduce distrust.
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  • 文章类型: Journal Article
    目的:比较性能(协变量平衡,稳定平衡权重(SBW)与倾向得分权重(PSW)的有效样本量[ESS])。使用两个应用案例来比较性能:(案例1)组间基线协变量的极端不平衡和(案例2)组间样本量的实质性差异。
    方法:使用PremierHealthcare数据库,我们选择的患者(病例1)在2000年1月至2020年6月期间接受了两种不同双极镊子之一的外科手术,或(病例2)在2000年1月至2020年3月期间接受了两种不同不可吸收外科缝线之一的神经系统手术.基于选择的协变量产生对治疗(ATT)权重的平均治疗效果。SBW使用两种技术实现:(1)“网格搜索”以最低目标绝对标准化平均差(SMD)找到最小方差的权重;(2)在预先指定的SMD容差下找到最小方差的权重。PSW和SBW方法在加权后的SMD上进行了比较,不平衡协变量的数量,和ATT加权对照组的ESS。
    结果:在两项研究中,两种SBW技术都实现了改善的协变量平衡。所有方法的加权后ESS低于未加权对照组的原始样本量;然而,SBW方法在对照组中获得了较高的ESS。使用SBW应用变量特异性SMD阈值增加ESS的敏感性分析,表现优于PSW。
    结论:在此应用示例中,与PSW相比,基于优化的SBW方法为协变量平衡目标的预设提供了足够的灵活性,并导致更好的加权后协变量平衡和更大的ESS.
    OBJECTIVE: To compare the performance (covariate balance, effective sample size [ESS]) of stable balancing weights (SBW) versus propensity score weighting (PSW). Two applied cases were used to compare performance: (Case 1) extreme imbalance in baseline covariates between groups and (Case 2) substantial discrepancy in sample size between groups.
    METHODS: Using the Premier Healthcare Database, we selected patients who (Case 1) underwent a surgical procedure with one of two different bipolar forceps between January 2000 and June 2020, or (Case 2) a neurological procedure using one of two different nonabsorbable surgical sutures between January 2000 and March 2020. Average treatment effects on the treated (ATT) weights were generated based on selected covariates. SBW was implemented using two techniques: (1) \"grid search\" to find weights of minimum variance at the lowest target absolute standardized mean difference (SMD); (2) finding weights of minimum variance at prespecified SMD tolerance. PSW and SBW methods were compared on postweighting SMDs, the number of imbalanced covariates, and ESS of the ATT-weighted control group.
    RESULTS: In both studies, improved covariate balance was achieved with both SBW techniques. All methods suffered from postweighting ESS that was lower than the unweighted control group\'s original sample size; however, SBW methods achieved higher ESS for the control groups. Sensitivity analyses using SBW to apply variable-specific SMD thresholds increased ESS, outperforming PSW.
    CONCLUSIONS: In this applied example, the optimization-based SBW method provided ample flexibility with respect to prespecification of covariate balance goals and resulted in better postweighting covariate balance and larger ESS as compared with PSW.
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  • 文章类型: Journal Article
    谷物是世界上许多日常饮食的基础。最近,由于其植物化学物质的含量,人们对全谷物的有益特性产生了相当大的兴趣,特别是多酚。尽管如此,最全面的数据库中报告的谷物类食品中多酚成分的现有数据仍未更新.许多谷类食品和酚类化合物缺失,包括有色素的.报告谷物多酚摄入量的观察性流行病学研究有限且不一致,尽管实验研究表明膳食多酚对心血管疾病具有保护作用,糖尿病,和癌症。由于食物中存在大量化合物,影响其含量的因素很多,因此估算多酚的摄入量是复杂的,如植物品种,收获季节,食品加工和烹饪,使得消费数据与食品成分数据难以匹配。Further,应该考虑到食物成分表和食用食物以不同的方式分类。本工作概述了几个现有数据库中报告的多酚含量的可用数据,就存在而言,缺少和没有数据,并讨论了评估谷物多酚消费量的方法的优缺点。此外,这篇综述表明,我们更需要纳入最新的谷类食物成分数据,并统一收集谷类食物数据的标准化程序和适当的膳食摄入评估工具.
    Cereals are the basis of much of the world\'s daily diet. Recently, there has been considerable interest in the beneficial properties of wholegrains due to their content of phytochemicals, particularly polyphenols. Despite this, the existing data on polyphenolic composition of cereal-based foods reported in the most comprehensive databases are still not updated. Many cereal-based foods and phenolic compounds are missing, including pigmented ones. Observational epidemiological studies reporting the intake of polyphenols from cereals are limited and inconsistent, although experimental studies suggest a protective role for dietary polyphenols against cardiovascular disease, diabetes, and cancer. Estimating polyphenol intake is complex because of the large number of compounds present in foods and the many factors that affect their levels, such as plant variety, harvest season, food processing and cooking, making it difficult matching consumption data with data on food composition. Further, it should be taken into account that food composition tables and consumed foods are categorized in different ways. The present work provides an overview of the available data on polyphenols content reported in several existing databases, in terms of presence, missing and no data, and discusses the strengths and weaknesses of methods for assessing cereal polyphenol consumption. Furthermore, this review suggests a greater need for the inclusion of most up-to-date cereal food composition data and for the harmonization of standardized procedures in collecting cereal-based food data and adequate assessment tools for dietary intake.
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