Observational studies

观察性研究
  • 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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  • 文章类型: Journal Article
    将一个层分为两个以最大程度地减少许多协变量中的层内不平衡的最佳方法是什么?我们将其公式化为整数程序,并通过线性程序的随机舍入来近似求解。线性程序可以将人的一部分分配给每个细化的阶层。随机舍入将分数人视为概率,使用有偏见的硬币将完整的人分配到地层中。随机舍入是一种经过充分研究的理论技术,可以近似某些不可解决的整数程序的最佳解决方案。当一个阶层中的人数相对于协变量的数量很大时,我们证明了以下新结果:(I)随机舍入以分割地层几乎没有随机化,所以它非常类似于线性规划松弛而不分裂完整的人;(ii)线性松弛和随机舍入解在不可达的整数规划解上放置下界和上界;并且由于(i),这些界限通常很接近,从而批准可用的随机舍入解决方案。我们使用一项观察性研究进行了说明,该研究通过形成由使用倾向评分从5735中选出的2016年患者组成的匹配对来平衡许多协变量。相反,我们形成5个倾向评分层,并将它们细化为10个层,获得良好的协变量平衡,同时保留所有患者。CRAN的R包optrefine实现了该方法。补充材料可在线获得。
    What is the best way to split one stratum into two to maximally reduce the within-stratum imbalance in many covariates? We formulate this as an integer program and approximate the solution by randomized rounding of a linear program. A linear program may assign a fraction of a person to each refined stratum. Randomized rounding views fractional people as probabilities, assigning intact people to strata using biased coins. Randomized rounding is a well-studied theoretical technique for approximating the optimal solution of certain insoluble integer programs. When the number of people in a stratum is large relative to the number of covariates, we prove the following new results: (i) randomized rounding to split a stratum does very little randomizing, so it closely resembles the linear programming relaxation without splitting intact people; (ii) the linear relaxation and the randomly rounded solution place lower and upper bounds on the unattainable integer programming solution; and because of (i), these bounds are often close, thereby ratifying the usable randomly rounded solution. We illustrate using an observational study that balanced many covariates by forming matched pairs composed of 2016 patients selected from 5735 using a propensity score. Instead, we form 5 propensity score strata and refine them into 10 strata, obtaining excellent covariate balance while retaining all patients. An R package optrefine at CRAN implements the method. Supplementary materials are available online.
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  • 文章类型: Journal Article
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  • 文章类型: Journal Article
    背景:关于手术治疗与事件发生时间终点的比较研究为临床实践提供了大量证据,但是生存数据分析的准确使用和混杂偏差的控制仍然是巨大的挑战。
    方法:这是对2021年发表在四本普通医学期刊和五本普通外科期刊上的具有生存结果的外科研究的调查。对两个最关心的统计问题进行了评估,包括通过倾向评分分析(PSA)或多变量分析以及Cox模型中比例风险(PH)假设的混淆控制。
    结果:共纳入74项研究,包括63项观察性研究和11项随机对照试验。在观察性研究中,在外科肿瘤学和非肿瘤学研究中使用PSA的研究比例相似(40.9%对36.8%,P=0.762)。然而,前者报告的PH假设评估比例明显低于后者(13.6%对42.1%,P=0.020)。25项观察性研究(25/63)使用PSA方法,但其中三分之二(17/25)显示PSA后基线数据的平衡不清楚.PSA后的PH假设测试比例略低于PSA前,但差异无统计学意义(24.0%对28.0%,P=0.317)。对生存分析中的混杂控制以及不遵守PH假设的替代解决方案提出了全面建议。
    结论:本研究强调了PSA前后观察性手术研究中PH假设评估的次优报告。在统计方法的基本假设方面需要努力和达成共识。
    BACKGROUND: Comparative studies on surgical treatments with time-to-event endpoints have provided substantial evidence for clinical practice, but the accurate use of survival data analysis and the control of confounding bias remain big challenges.
    METHODS: This was a survey of surgical studies with survival outcomes published in four general medical journals and five general surgical journals in 2021. The two most concerned statistical issues were evaluated, including confounding control by propensity score analysis (PSA) or multivariable analysis and testing of proportional hazards (PH) assumption in Cox model.
    RESULTS: A total of 74 studies were included, comprising 63 observational studies and 11 randomized controlled trials. Among the observational studies, the proportion of studies utilizing PSA in surgical oncology and non-oncology studies was similar (40.9 % versus 36.8 %, P = 0.762). However, the former reported a significantly lower proportion of PH assumption assessments compared to the latter (13.6 % versus 42.1 %, P = 0.020). Twenty-five observational studies (25/63) used PSA methods, but two-thirds of them (17/25) showed unclear balance of baseline data after PSA. And the proportion of PH assumption testing after PSA was slightly lower than that before PSA, but the difference was not statistically significant (24.0 % versus 28.0 %, P = 0.317). Comprehensive suggestions were given on confounding control in survival analysis and alternative resolutions for non-compliance with PH assumption.
    CONCLUSIONS: This study highlights suboptimal reporting of PH assumption evaluation in observational surgical studies both before and after PSA. Efforts and consensus are needed with respect to the underlying assumptions of statistical methods.
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  • 文章类型: Journal Article
    牛皮癣是一种慢性免疫介导的影响皮肤的疾病,指甲,和/或关节。它与全身性炎症有关,也可能与动脉粥样硬化性心血管疾病(ASCVD)的风险增加有关。这项研究的目的是确定银屑病患者ASCVD的总体风险,并根据ASCVD类型和银屑病的严重程度评估风险。这是一项观察性研究的系统评价和荟萃分析,报告了银屑病与一种或多种ASCVD临床类型之间的关联。我们通过PubMed在线搜索医学文献分析和检索系统(MEDLINE),摘录医学数据库(EMBASE),Scopus,比勒费尔德学术搜索引擎(BASE),和谷歌学者从他们的记录开始到2023年7月的英语相关研究。研究选择和数据提取由四名独立评审员进行。总共21项观察性研究(三项横断面研究,一个病例控制,和17个队列)被包括在这篇综述中,代表总共778,049名牛皮癣患者和16,881,765名无牛皮癣的对照受试者。纳入的研究有不同程度的协变量调整,因此,他们的发现可能会受到残留的混淆。所有荟萃分析都使用调整后的效应大小,并基于随机效应模型。然而,队列研究与非队列研究(病例对照和横断面研究)分开分析.银屑病和ASCVD之间存在显著关联(队列研究:风险比(HR),1.21;95%置信区间(CI),1.14至1.28;I2=63%;p<0.001;非队列研究:比值比(OR),1.60;95%CI,1.34至1.92;I2=31%;p=0.23)。银屑病也与心肌梗死显著相关(队列研究:HR,1.20;95%CI,1.10~1.31;I2=60%;p<0.001;非队列研究:OR,1.57;95%CI,1.15至2.15;I2=74%;p=0.05),冠状动脉疾病(队列研究:HR,1.20;95%CI,1.13~1.28;I2=67%;p<0.001;非队列研究:OR,1.60;95%CI,1.34至1.92;I2=31%;p=0.23),主动脉瘤(HR,1.45;95%CI,1.04至2.02;I2=67%;p=0.08),但与缺血性卒中无关(HR,1.14;95%CI,0.96至1.36;I2=44%;p=0.17)。就银屑病的严重程度进行汇总分析显示,两者均为轻度(队列研究:HR,1.17;95%CI,1.08~1.26;I2=74%;p<0.001;非队列研究:OR,1.54;95%CI,1.25至1.90;I2=0%;p=0.50)和严重(队列研究:HR,1.43;95%CI,1.23~1.65;I2=65%;p<0.001;非队列研究:OR,1.65;95%CI,1.29~2.12;I2=25%;p=0.26)银屑病与ASCVD显著相关。银屑病(包括轻度和重度疾病)与ASCVD的风险增加有关。包括冠状动脉疾病(CAD)和主动脉瘤(AA)。所有成人银屑病患者应优先考虑ASCVD风险评估和预防。未来的观察性研究调查银屑病和ASCVD之间的关联应该进行更全面的协变量调整。
    Psoriasis is a chronic immune-mediated disease affecting the skin, nails, and/or joints. It is associated with systemic inflammation and may also be linked to an increased risk of atherosclerotic cardiovascular disease (ASCVD). The objectives of this study were to determine the overall risk of ASCVD in patients with psoriasis and to evaluate the risk according to ASCVD type and the severity of psoriasis. This was a systematic review and meta-analysis of observational studies reporting the association between psoriasis and one or more of the clinical types of ASCVD. We searched Medical Literature Analysis and Retrieval System Online (MEDLINE) via PubMed, Excerpta Medica Database (EMBASE), Scopus, Bielefeld Academic Search Engine (BASE), and Google Scholar for relevant studies in the English language from the beginning of their records to July 2023. Study selection and data extraction were conducted by four independent reviewers. A total of 21 observational studies (three cross-sectional, one case-control, and 17 cohort) were included in this review, representing a total of 778,049 patients with psoriasis and 16,881,765 control subjects without psoriasis. The included studies had varying degrees of covariate adjustment, and thus, their findings may have been subject to residual confounding. All the meta-analyses used the adjusted effect sizes and were based on the random-effects model. However, the cohort studies were analysed separately from the non-cohort studies (the case-control and cross-sectional studies). There was a significant association between psoriasis and ASCVD (cohort studies: hazard ratio (HR), 1.21; 95% confidence interval (CI), 1.14 to 1.28; I2 = 63%; p < 0.001; non-cohort studies: odds ratio (OR), 1.60; 95% CI, 1.34 to 1.92; I2 = 31%; p = 0.23). Psoriasis was also significantly associated with myocardial infarction (cohort studies: HR, 1.20; 95% CI, 1.10 to 1.31; I2 = 60%; p < 0.001; non-cohort studies: OR, 1.57; 95% CI, 1.15 to 2.15; I2 = 74%; p = 0.05), coronary artery disease (cohort studies: HR, 1.20; 95% CI, 1.13 to 1.28; I2 = 67%; p < 0.001; non-cohort studies: OR, 1.60; 95% CI, 1.34 to 1.92; I2 = 31%; p = 0.23), aortic aneurysm (HR, 1.45; 95% CI, 1.04 to 2.02; I2 = 67%; p = 0.08) but not with ischaemic stroke (HR, 1.14; 95% CI, 0.96 to 1.36; I2 = 44%; p = 0.17). Pooled analysis in terms of the severity of psoriasis showed that both mild (cohort studies: HR, 1.17; 95% CI, 1.08 to 1.26; I2 = 74%; p < 0.001; non-cohort studies: OR, 1.54; 95% CI, 1.25 to 1.90; I2 = 0%; p = 0.50) and severe (cohort studies: HR, 1.43; 95% CI, 1.23 to 1.65; I2 = 65%; p < 0.001; non-cohort studies: OR, 1.65; 95% CI, 1.29 to 2.12; I2 = 25%; p = 0.26) psoriasis were significantly associated with ASCVD. Psoriasis (including mild and severe disease) is associated with an increased risk of ASCVD, including coronary artery disease (CAD) and aortic aneurysm (AA). ASCVD risk assessment and prevention should be prioritised in all adult psoriasis patients. Future observational studies investigating the association between psoriasis and ASCVD should conduct a more comprehensive adjustment of covariates.
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  • 文章类型: Letter
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  • 文章类型: Editorial
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  • 文章类型: Systematic Review
    吸入皮质类固醇(ICS)治疗已被证明可以降低COPD加重的风险。应仅适用于未通过双重长效支气管扩张剂治疗得到充分控制且每年加重≥2次,血液嗜酸性粒细胞计数≥300个细胞/µL的COPD患者。ICS治疗在COPD患者的指南之外广泛使用,使ICS退出成为一个重要的考虑因素。本系统综述旨在对ICS戒断对加重频率的影响进行最新分析。肺功能(FEV1)的变化,并确定停药后恢复ICS治疗的COPD患者的比例。
    纳入比较ICS停药与ICS继续治疗的随机对照试验(RCT)和观察性研究。CochraneCentral,WebofScience,CINHAL,搜索Embase和OVIDMedline。使用CochraneRoB2工具和纽卡斯尔-渥太华量表评估偏倚风险。采用GRADE对随机对照试验进行质量评价。ICS戒断RCT事后分析的荟萃分析,通过血液嗜酸性粒细胞计数(BEC)分层,进行了。
    10项随机对照试验(6642例患者随机分组)和6项观察性研究(160,029例患者)纳入结果。当停用ICS并维持长效支气管扩张剂治疗时,ICS退出试验组和继续试验组的加重频率或肺功能变化无一致差异.这些影响的证据质量中等。对于停药后恢复ICS治疗的患者比例(估计范围为12-93%的参与者),没有足够的证据得出确切的结论。
    COPD患者退出ICS治疗是安全可行的,但应同时维持支气管扩张治疗以获得最佳结果。
    UNASSIGNED: Inhaled corticosteroid (ICS) therapy has been demonstrated to reduce the risk of COPD exacerbations. It should only be prescribed to COPD patients who are not adequately controlled by dual long-acting bronchodilator therapy and who have ≥2 exacerbations per year and a blood eosinophil count ≥300cells/µL. ICS therapy is widely prescribed outside guidelines to COPD patients, making ICS withdrawal an important consideration. This systematic review aims to provide an up-to-date analysis of the effect of ICS withdrawal on exacerbation frequency, change in lung function (FEV1) and to determine the proportion of COPD patients who resume ICS therapy following withdrawal.
    UNASSIGNED: Randomised controlled trials (RCTs) and observational studies which compared ICS withdrawal with ICS continuation treatment were included. Cochrane Central, Web of Science, CINHAL, Embase and OVID Medline were searched. Risk of bias was assessed using the Cochrane RoB2 tool and the Newcastle-Ottawa Scale. Quality assessment of RCTs was conducted using GRADE. Meta-analysis of post-hoc analyses of RCTs of ICS withdrawal, stratified by blood eosinophil count (BEC), was undertaken.
    UNASSIGNED: Ten RCTs (6642 patients randomised) and 6 observational studies (160,029 patients) were included in the results. When ICS was withdrawn and long-acting bronchodilator therapy was maintained, there was no consistent difference in exacerbation frequency or lung function change between the ICS withdrawal and continuation trial arms. The evidence for these effects was of moderate quality. There was insufficient evidence to draw a firm conclusion on the proportion of patients who resumed ICS therapy following withdrawal (estimated range 12-93% of the participants).
    UNASSIGNED: Withdrawal of ICS therapy from patients with COPD is safe and feasible but should be accompanied by maintenance of bronchodilation therapy for optimal outcomes.
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  • 文章类型: Journal Article
    目的:超声(US)可以检测临床可疑关节痛(CSA)患者的亚临床关节炎症,这是有价值的预测类风湿关节炎(RA)的发展。在大多数研究协议中,手和前脚都被扫描,但目前还不清楚美国的前脚是否有预测RA的额外价值,特别是由于健康个体的MTP关节滑膜肥大也很常见。为了探索省略前足扫描的可能性,我们确定前足US是否对CSA患者的RA发展具有额外的预测价值。
    方法:两个独立队列的CSA患者进行了手和前足的US检查。我们分析了完整的美国协议的RA发展和美国阳性之间的关联,完整的US方案,校正了健康人前脚的灰度(GS)发现,以及无前脚的方案。
    结果:总计,对298例CSA患者进行了研究。在美国呈阳性的患者中,亚临床关节炎症主要存在于手部(90-86%).只有10-14%的患者仅在前脚有亚临床关节炎症。在两个队列中,US阳性与炎症性关节炎的发展有关,完整方案的HR为2.6(95CI0.9-7.5)和3.1(95CI1.5-6.4),3.1(95CI1.3-7.7)和2.7(95CI1.3-5.4),用于完整的美国协议,和3.1(95CI1.4-6.9)和2.8(95CI1.4-5.6),没有前脚。AUROC在两个队列中是相等的。
    结论:当US用于预测CSA患者的RA发展时,可以省略前足。这是由于发现前脚部的亚临床关节炎症而手部没有伴随的炎症是罕见的。
    OBJECTIVE: Ultrasound (US) can detect subclinical joint-inflammation in patients with clinically suspect arthralgia (CSA), which is valuable as predictor for rheumatoid arthritis (RA) development. In most research protocols both hands and forefeet are scanned, but it is unclear if US of the forefeet has additional value for predicting RA, especially since synovial hypertrophy in MTP-joints of healthy individuals is also common. To explore the possibility to omit scanning of the forefeet we determined if US of the forefeet is of additional predictive value for RA-development in CSA patients.
    METHODS: CSA patients of two independent cohorts underwent US of the hands and forefeet. We analyzed the association between RA-development and US-positivity for the full US-protocol, the full US-protocol with correction for Gray Scale(GS)-findings in the forefeet of healthy and the protocol without-forefeet.
    RESULTS: In total, 298 CSA patients were studied. In patients with a positive US, subclinical joint-inflammation was mostly present in the hands (90-86%). Only 10-14% of patients had subclinical joint-inflammation solely in the forefeet. US-positivity was associated with inflammatory arthritis development in both cohorts, with HRs 2.6(95%CI 0.9-7.5) and 3.1(95%CI 1.5-6.4) for the full protocol, 3.1(95%CI 1.3-7.7) and 2.7(95%CI 1.3-5.4) for the full US-protocol with correction, and 3.1(95%CI 1.4-6.9) and 2.8(95%CI 1.4-5.6) without the forefeet. AUROCs were equal across both cohorts.
    CONCLUSIONS: The forefeet can be omitted when US is used for the prediction of RA-development in CSA patients. This is due to the finding that subclinical joint-inflammation in the forefeet without concomitant inflammation in the hands is infrequent.
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  • 文章类型: Journal Article
    背景:生活质量指标对于肿瘤患者以及死亡率和疾病进展等传统终点越来越重要。统计工具,如WinRatio,赢得赔率,和净效益优先考虑使用复合终点的临床显著结果。在随机试验中,Win统计提供治疗组和对照组之间的公平比较。然而,它们在观察性研究中的使用因混杂变量而变得复杂。倾向得分(PS)匹配减轻了混杂变量,但可能会减少样本量,影响必胜统计分析的力量。或者,PS匹配可以对样本进行分层,保留样本量。本研究旨在评估这些方法对决策的长期影响,尤其是结直肠癌患者。方法:一个激励的例子涉及来自ReSARCh观察性研究(2016-2021)的局部晚期直肠腺癌患者队列,位于12厘米的肛门边缘。这些患者接受了观察和等待方法(WW)或经肛门局部切除术(LE)。Win统计数据比较了WW和LE对复合结局的影响(总生存期,复发,造口术的存在,和直肠切除术)。对于匹配的获胜统计数据,我们使用了Matsouaka等人提出的鲁棒推理技术。(2022),对于分层的胜利统计数据,我们应用了Dong等人提出的方法。(2018)。一项模拟研究评估了平衡和不平衡组中匹配和分层获胜统计数据的覆盖概率,计算置信区间包含WR真实值的频率,NB,和WO跨越1000次模拟。结果:结果表明,当单独考虑疗效结果时,LE方法的疗效更好(WR:0.47(0.01至1.14);NB:-0.16(-0.34至0.02);WO:0.73(0.5至1.05))。然而,当QoL结果包括在分析中时,估计更接近1(WR:0.87(0.06至2.06);WO:0.93(0.61至1.4))和0(NB:-0.04(-0.25至0.17)),这表明LE对造口术和直肠切除的治疗效果有负面影响。此外,基于仿真研究,我们的发现强调了与分层获胜统计相比,匹配在覆盖概率方面的卓越表现(匹配WR:97%与分层WR:在高不平衡情况下为33.3%;匹配WR:98%与分层WR:中等不平衡情况下的34.4%;匹配WR:99.2%与分层WR:在低不平衡设置中为37.4%)。结论:总之,我们的研究从统计意义上解释了winstatistics的结果,提供对成对比较在观测设置中的应用的见解,促进其使用,以改善癌症患者的预后。
    Background: Quality-of-life metrics are increasingly important for oncological patients alongside traditional endpoints like mortality and disease progression. Statistical tools such as Win Ratio, Win Odds, and Net Benefit prioritize clinically significant outcomes using composite endpoints. In randomized trials, Win Statistics provide fair comparisons between treatment and control groups. However, their use in observational studies is complicated by confounding variables. Propensity score (PS) matching mitigates confounding variables but may reduce the sample size, affecting the power of win statistics analyses. Alternatively, PS matching can stratify samples, preserving the sample size. This study aims to assess the long-term impact of these methods on decision making, particularly in colorectal cancer patients. Methods: A motivating example involves a cohort of patients from the ReSARCh observational study (2016-2021) with locally advanced adenocarcinoma of the rectum, situated up to 12 cm from the anal verge. These patients underwent either a watch-and-wait approach (WW) or trans-anal local excision (LE). Win statistics compared the effects of WW and LE on a composite outcome (overall survival, recurrence, presence of ostomy, and rectum excision). For matched win statistics, we used robust inference techniques proposed by Matsouaka et al. (2022), and for stratified win statistics, we applied the method proposed by Dong et al. (2018). A simulation study assessed the coverage probability of matched and stratified win statistics in balanced and unbalanced groups, calculating how often the confidence intervals included the true values of WR, NB, and WO across 1000 simulations. Results: The results suggest a better efficacy of the LE approach when considering efficacy outcomes alone (WR: 0.47 (0.01 to 1.14); NB: -0.16 (-0.34 to 0.02); and WO: 0.73 (0.5 to 1.05)). However, when QoL outcomes are included in the analyses, the estimates are closer to 1 (WR: 0.87 (0.06 to 2.06); WO: 0.93 (0.61 to 1.4)) and to 0 (NB: -0.04 (-0.25 to 0.17)), indicating a negative impact of the treatment effect of LE regarding the presence of ostomy and the excision of the rectum. Moreover, based on the simulation study, our findings underscore the superior performance of matched compared to stratified win statistics in terms of coverage probability (matched WR: 97% vs. stratified WR: 33.3% in a high-imbalance setting; matched WR: 98% vs. stratified WR: 34.4% in a medium-imbalance setting; and matched WR: 99.2% vs. stratified WR: 37.4% in a low-imbalance setting). Conclusions: In conclusion, our study sheds light on the interpretation of the results of win statistics in terms of statistical significance, providing insights into the application of pairwise comparison in observational settings, promoting its use to improve outcomes for cancer patients.
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