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A simulation study showed that linear regression and Mann-Whitney test can be used to analyze the days alive and at home by day 30 outcome in a randomized controlled trial 模拟研究表明,在随机对照试验中,线性回归和Mann-Whitney检验可用于分析存活和在家30天(DAH30)的结果。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-16 DOI: 10.1016/j.jclinepi.2025.111674
Jonathan Alistair Cook

Background and Objectives

The aims of the work were to consider the properties of the days alive and at home by day 30 (DAH30) from a statistical perspective, and to conduct a simulation study exploring the use of simple (unadjusted) linear regression and Mann-Whitney test as the method of analysis reflect realized analysis options.

Study Design and Setting

The days alive and at home by day 30 (DAH30) has been proposed a patient-centric outcome, and clinically relevant outcome suitable for clinical trials. It has unusual statistical properties, and suitability of standard statistical analysis methods is unclear. The properties of DAH30 were reviewed. Simulations based upon 1:1 allocation in an randomized controlled trial (RCT) based upon empirical data were conducted reflecting different additive and realized (reflecting the DAH30) treatment effects, sample sizes and distributions with varying and central locations and zero value level. A variety of metrics were used to assess performance (including bias, coverage, and rejection rate).

Results

Linear regression provided a valid estimate of the unadjusted average treatment effect with an additive treatment. This was confirmed in terms of bias, estimation of variance, rejection rate in the absence of an effect, and coverage of the 95% confidence interval for the true realized effect. Mann-Whitney provided greater (power) than linear regression in some situations.

Conclusion

Simple linear regression is a reasonable analytic option for the DAH30 for estimating the average treatment effect in the RCT cohort (ie, an intention to treat, or “treatment policy” estimand) where zero-inflation is relatively low. Mann-Whitney test in some circumstances (small effects and smaller samples sizes) provides better ability (like for like) to detect a difference between the groups.

Plain Language Summary

Simple linear regression can be used to analyze DAH30 outcome in a randomized trial for a range of scenarios which were considered in this study (including relatively proportions of zero values). The DAH30's properties affect the treatment effect than can be estimated. Mann-Whitney test offered better ability to detect a difference of a smaller magnitude for smaller samples sizes.
目的:从统计学角度考虑DAH30的特性,并进行模拟研究,探索使用简单(未调整)线性回归和Mann-Whitney检验作为分析方法,反映实现的分析选项。研究设计和设置:第30天存活和在家的天数(DAH30)已被提出为以患者为中心的指标,并且是适合临床试验的临床相关指标。它具有不同寻常的统计特性,标准统计分析方法的适用性尚不清楚。综述了DAH30的性质。在基于经验数据的随机对照试验(RCT)中进行了基于1:1分配的模拟,模拟了不同添加剂(反映DAH30)的处理效果、样本量以及中心位置和零值水平的分布。使用各种度量来评估性能(包括偏倚、覆盖率、拒绝率)。结果:线性回归对加性处理的未调整平均处理效果提供了有效的估计。这在偏差、方差估计、没有效应时的拒绝率和真实实现效应的95%置信区间的覆盖率方面得到了证实。在某些情况下,Mann-Whitney提供了比线性回归更大的(功率)。结论:在零通货膨胀率相对较低的RCT队列(即治疗意图或“治疗政策”估计)中,简单线性回归是DAH30估计平均治疗效果的合理分析选择。在某些情况下(小的影响和较小的样本量),曼-惠特尼检验提供了更好的能力(同类对同类)来检测组之间的差异。
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引用次数: 0
High-impact trials with genetic and -omics information focus on cancer mutations, are industry-funded, and less transparent 具有遗传和组学信息的高影响试验侧重于癌症突变,由行业资助,透明度较低。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-16 DOI: 10.1016/j.jclinepi.2025.111676
Luigi Russo , Leonardo M. Siena , Sara Farina , Roberta Pastorino , Stefania Boccia , John P.A. Ioannidis

Objectives

To assess how genetics and -omics information is used in the most cited recent clinical trials and to evaluate industry involvement and transparency patterns.

Study Design and Setting

This is a meta-research evaluation using a previously constructed database of the 600 most cited clinical trials published from 2019 to 2022. Trials that utilized genetic or -omics characterization of participants in the trial design, analysis, and results were considered eligible.

Results

132 (22%) trials used genetic or -omics information, predominantly for detection of cancer mutations (n = 101). Utilization included eligibility criteria (n = 59), subgroup analysis (n = 82), and stratification factor in randomization (n = 14). Authors addressed the relevance in the conclusions in 82 studies (62%). 102 studies (77%) provided data availability statements and six had data already available. Most studies had industry funding (n = 111 [84.0%]). Oncology trials were more likely to be industry-funded (90.1% vs 64.5%, P = .001), to have industry-affiliated analysts (43.6% vs 22.6%, P = .036), and to favor industry-sponsored interventions (83.2% vs 58.1% P = .004). When compared to other trials, genetic and -omics trials were more likely to be funded by industry (84% vs 63.9%, P < .001) and tended to be less likely to have full protocols (P = .018) and statistical plans (P = .04) available.

Conclusion

Our study highlights the current underutilization of genetic and -omics technologies beyond testing for cancer mutations. Industry involvement in these trials appears to be more substantial and transparency is more limited, raising concerns about potential bias.
目的:评估遗传学和组学信息如何在最近引用最多的临床试验中使用,并评估行业参与和透明度模式。研究设计和设置:这是一项元研究评估,使用先前构建的数据库,该数据库包含2019年至2022年发表的600项被引用最多的临床试验。在试验设计、分析和结果中利用参与者的遗传或组学特征的试验被认为是合格的。主要发现:132项(22%)试验使用遗传或组学信息,主要用于检测癌症突变(n=101)。应用包括资格标准(n=59)、亚组分析(n=82)和随机分层因素(n=14)。作者在82项研究(62%)的结论中提到了相关性。102项研究(77%)提供了数据可用性声明,6项研究已有数据可用。大多数研究有行业资助(n=111[84.0%])。肿瘤学试验更有可能是由行业资助的(90.1%对64.5%,p=0.001),有行业关联的分析师(43.6%对22.6%,p=0.036),以及支持行业赞助的干预措施(83.2%对58.1%,p=0.004)。与其他试验相比,基因和组学试验更有可能得到工业界的资助(84%对63.9%)。结论:我们的研究强调了目前基因和组学技术在癌症突变检测之外的利用不足。行业对这些试验的参与似乎更大,透明度更有限,引发了对潜在偏见的担忧。
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引用次数: 0
Funding matters: time to update preferred reporting items for systematic reviews and meta-analyses? 资金问题:何时更新PRISMA?
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-15 DOI: 10.1016/j.jclinepi.2025.111678
James Burgert, Georgia C. Richards
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引用次数: 0
How should we assess trustworthiness of randomized controlled trials? 我们应该如何评估随机对照试验的可信度?
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-13 DOI: 10.1016/j.jclinepi.2025.111670
Jack Wilkinson, David Tovey
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引用次数: 0
Exploring the use and usefulness of living guidelines for consumers: international online survey of patients' and carers' views 探索消费者生活指南的使用和有用性:对患者和护理人员观点的国际在线调查。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-13 DOI: 10.1016/j.jclinepi.2025.111671
Anneliese Synnot , Samantha Chakraborty , Jessica Xue , Hui Zhen Cheng , Danielle Berkovic , Tari Turner

Background and Objective

Living guidelines contain continually updated, and potentially changing, clinical recommendations. The implications of living guidelines for consumers (eg, patients, carers, and people with lived experience) - particularly how living guidelines should be developed and disseminated – are yet to be established. The objective of this study was to explore consumers’ views about how best to support the use and usefulness of living guidelines to consumers.

Methods

This study used a qualitative (online survey) design. We invited consumers who were familiar with guidelines (living or conventional) to participate in the study. The survey was distributed globally. Recruitment was conducted via the Australian and international networks of the Australian Living Evidence Collaboration. We invited consumers who were familiar with guidelines (living or conventional) to participate in the study. The survey was distributed globally. Recruitment was conducted via the Australian and international networks of the Australian Living Evidence Collaboration. The 5–10 minute survey collected demographic data then, after introducing the living guidelines concept, asked questions about what living guidelines mean for consumers, how we might make them easy for consumers to find and use, and potential challenges to their use. We analyzed the data using inductive thematic analysis.

Results

Forty-five people (71% women) from 12 countries completed the survey. Participants were enthusiastic about the concept of living guidelines and what they might mean for consumers' ability to make informed health-care decisions and receive best care. They also identified potential challenges related to living guideline dissemination, such as low public awareness of guidelines and confusion about updated recommendations. Participants described practical strategies to support consumers’ awareness and use of, and access to, living guidelines. These included: meaningful involvement of consumers in the development and dissemination of living guidelines; raising awareness by promoting the guidelines widely through trusted health information sources and on social media; and using user-centered formatting and design principles (eg, considering accessibility needs, and publishing lay summaries with plain and culturally-appropriate language).

Conclusion

Consumers suggested a comprehensive range of dissemination strategies to support the use and usefulness of living guidelines to consumers, which largely reflect best practice in conventional guideline dissemination. Promoting and explaining the living nature of guideline recommendations might support their use by consumers. There should also be a close link between the living guidelines and any versions or additional content created for both consumers and clinicians.
背景:生活指南包含不断更新和潜在变化的临床建议。生活准则对消费者(例如病人、护理人员、有生活经验的人)的影响,特别是如何制定和传播生活准则,还有待确定。目的:探讨消费者对如何最好地支持生活指南对消费者的使用和有用性的看法。设计:定性(在线调查)设置和参与者:我们邀请熟悉指南(生活或传统)的消费者参与研究。该调查在全球范围内进行。招募是通过澳大利亚活体证据合作的澳大利亚和国际网络进行的。数据收集和分析:5-10分钟的调查收集了人口统计数据,然后介绍了生活指南的概念,询问了生活指南对消费者意味着什么,我们如何让消费者更容易找到和使用它们,以及使用它们可能面临的挑战。我们采用归纳主题分析法对数据进行分析。结果:来自12个国家的45人(71%为女性)完成了调查。与会者对生活指南的概念以及它们对消费者做出明智的医疗保健决定和获得最佳护理的能力可能意味着什么充满热情。他们还确定了与生活指南传播相关的潜在挑战,例如公众对指南的认知度低以及对更新建议的混淆。与会者介绍了支持消费者认识、使用和获得生活指南的实际策略。这些措施包括:消费者有意义地参与生活准则的制定和传播;通过可信赖的卫生信息来源和社交媒体广泛宣传指南,提高认识;使用以用户为中心的格式和设计原则(例如,考虑可访问性需求,用通俗易懂的语言发布外行摘要)。结论:消费者建议了一系列全面的传播策略,以支持生活指南对消费者的使用和有用性,这在很大程度上反映了传统指南传播的最佳实践。促进和解释指南建议的生活性质可能会支持消费者使用它们。在生活指南和为消费者和临床医生创建的任何版本或附加内容之间也应该有密切的联系。
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引用次数: 0
Larger sample sizes are needed when developing a clinical prediction model using machine learning in oncology: methodological systematic review 当在肿瘤学中使用机器学习开发临床预测模型时,需要更大的样本量:方法学系统评价。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-13 DOI: 10.1016/j.jclinepi.2025.111675
Biruk Tsegaye , Kym I.E. Snell , Lucinda Archer , Shona Kirtley , Richard D. Riley , Matthew Sperrin , Ben Van Calster , Gary S. Collins , Paula Dhiman

Background and Objectives

Having a sufficient sample size is crucial when developing a clinical prediction model. We reviewed details of sample size in studies developing prediction models for binary outcomes using machine learning (ML) methods within oncology and compared the sample size used to develop the models with the minimum required sample size needed when developing a regression-based model (Nmin).

Methods

We searched the Medline (via OVID) database for studies developing a prediction model using ML methods published in December 2022. We reviewed how sample size was justified. We calculated Nmin, which is the Nmin, and compared this with the sample size that was used to develop the models.

Results

Only one of 36 included studies justified their sample size. We were able to calculate Nmin for 17 (47%) studies. 5/17 studies met Nmin, allowing to precisely estimate the overall risk and minimize overfitting. There was a median deficit of 302 participants with the event (n = 17; range: −21,331 to 2298) when developing the ML models. An additional three out of the 17 studies met the required sample size to precisely estimate the overall risk only.

Conclusion

Studies developing a prediction model using ML in oncology seldom justified their sample size and sample sizes were often smaller than Nmin. As ML models almost certainly require a larger sample size than regression models, the deficit is likely larger. We recommend that researchers consider and report their sample size and at least meet the minimum sample size required when developing a regression-based model.
背景:在建立临床预测模型时,有足够的样本量是至关重要的。我们回顾了在肿瘤学中使用机器学习(ML)方法开发二元结果预测模型的研究中样本量的细节,并将用于开发模型的样本量与开发基于回归的模型(Nmin)所需的最小样本量进行了比较。方法:我们在Medline(通过OVID)数据库中检索了2022年12月发表的使用ML方法开发预测模型的研究。我们回顾了样本量的合理性。我们计算了Nmin,这是开发基于回归的模型所需的最小样本量,并将其与用于开发模型的样本量进行了比较。结果:36项纳入的研究中只有一项证明了其样本量的合理性。我们能够计算17项(47%)研究的Nmin。5/17项研究符合Nmin,允许精确估计总体风险并最大限度地减少过拟合。该事件中有302名参与者存在中位数缺陷(n= 17;范围:-21331至2298)开发ML模型时。在17项研究中,另外3项研究满足了精确估计总体风险所需的样本量。结论:利用肿瘤ML建立预测模型的研究很少证明其样本量是合理的,而且样本量通常小于Nmin。由于ML模型几乎肯定需要比回归模型更大的样本量,因此赤字可能更大。我们建议研究人员考虑并报告他们的样本量,并且在开发基于回归的模型时至少满足所需的最小样本量。
{"title":"Larger sample sizes are needed when developing a clinical prediction model using machine learning in oncology: methodological systematic review","authors":"Biruk Tsegaye ,&nbsp;Kym I.E. Snell ,&nbsp;Lucinda Archer ,&nbsp;Shona Kirtley ,&nbsp;Richard D. Riley ,&nbsp;Matthew Sperrin ,&nbsp;Ben Van Calster ,&nbsp;Gary S. Collins ,&nbsp;Paula Dhiman","doi":"10.1016/j.jclinepi.2025.111675","DOIUrl":"10.1016/j.jclinepi.2025.111675","url":null,"abstract":"<div><h3>Background and Objectives</h3><div>Having a sufficient sample size is crucial when developing a clinical prediction model. We reviewed details of sample size in studies developing prediction models for binary outcomes using machine learning (ML) methods within oncology and compared the sample size used to develop the models with the minimum required sample size needed when developing a regression-based model (N<sub>min</sub>).</div></div><div><h3>Methods</h3><div>We searched the Medline (via OVID) database for studies developing a prediction model using ML methods published in December 2022. We reviewed how sample size was justified. We calculated N<sub>min</sub>, which is the N<sub>min</sub>, and compared this with the sample size that was used to develop the models.</div></div><div><h3>Results</h3><div>Only one of 36 included studies justified their sample size. We were able to calculate N<sub>min</sub> for 17 (47%) studies. 5/17 studies met N<sub>min</sub>, allowing to precisely estimate the overall risk and minimize overfitting. There was a median deficit of 302 participants with the event (<em>n</em> = 17; range: −21,331 to 2298) when developing the ML models. An additional three out of the 17 studies met the required sample size to precisely estimate the overall risk only.</div></div><div><h3>Conclusion</h3><div>Studies developing a prediction model using ML in oncology seldom justified their sample size and sample sizes were often smaller than N<sub>min</sub>. As ML models almost certainly require a larger sample size than regression models, the deficit is likely larger. We recommend that researchers consider and report their sample size and at least meet the minimum sample size required when developing a regression-based model.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"180 ","pages":"Article 111675"},"PeriodicalIF":7.3,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143015515","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
What happened next? A survey of review clients evaluating impacts of rapid reviews 接下来发生了什么?对评估快速评审影响的评审客户的调查。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-10 DOI: 10.1016/j.jclinepi.2025.111673
Peter Bragge , Emily C. Clark , Veronica Delafosse , Ngo Cong-Lem , Diki Tsering , Paul Kellner , Alyssa Kostopoulos , Maureen Dobbins

Objectives

End-user evaluation of the impact of evidence syntheses is critical to demonstrating value. This study presents results of a survey evaluating the impact of rapid reviews undertaken by two teams based in Melbourne, Australia, and Hamilton, Canada.

Methods

Clients were invited to participate in a short written survey following delivery of a rapid review. Survey items encompassed reach, usefulness and format; interactions with the review teams; and overall satisfaction.

Results

Twenty-five completed surveys from 53 invitations were received pertaining to 19 rapid reviews conducted between September 2021 and October 2023. Topics encompassed COVID-19, health and behavior change; reports were an average of 31 pages; and were delivered over an average of 62 days. Evaluation findings were positive, with high satisfaction with reports and service delivery; very high satisfaction with report structure and length; good evidence of reach (reports read by decision makers and cited in other documents); and evidence that the rapid reviews made contributions to strategic planning, policy and program funding decisions.

Conclusion

Rapid reviews are making impactful contributions, alongside other inputs, to policy and practice. Further research is required to build this evaluation dataset; examine the balance between timeliness and methodological rigor in evidence synthesis; and explore models of delivery and capacity within and outside of government. It is also critical to promote implementation efforts to harness the full potential of rapid reviews and other evidence syntheses to impact the lives of citizens.
最终用户对证据综合影响的评估对于证明价值至关重要。本研究提出了一项调查的结果,该调查评估了澳大利亚墨尔本和加拿大汉密尔顿的两个小组进行的快速审查的影响。方法:客户被邀请参加一个简短的,书面调查后交付的快速审查。调查项目包括范围、有用性和格式;与评审团队的互动;总体满意度。结果:在2021年9月至2023年10月期间进行的19次快速审查中,收到了53份邀请,完成了25项调查。主题包括COVID-19、健康和行为改变;报告平均为31页;平均交付时间为62天。评价结果是积极的,对报告和提供的服务非常满意;对报告的结构和长度非常满意;良好的影响证据(决策者阅读并在其他文件中引用的报告);并有证据表明,快速审查有助于战略规划、政策和项目资助决策。结论:快速审查与其他投入一起对政策和实践作出了有影响力的贡献。需要进一步的研究来建立这个评估数据集;审查证据合成的及时性和方法严谨性之间的平衡;探索政府内外的交付模式和能力。还必须促进实施工作,充分利用快速审查和其他证据综合的潜力,影响公民的生活。
{"title":"What happened next? A survey of review clients evaluating impacts of rapid reviews","authors":"Peter Bragge ,&nbsp;Emily C. Clark ,&nbsp;Veronica Delafosse ,&nbsp;Ngo Cong-Lem ,&nbsp;Diki Tsering ,&nbsp;Paul Kellner ,&nbsp;Alyssa Kostopoulos ,&nbsp;Maureen Dobbins","doi":"10.1016/j.jclinepi.2025.111673","DOIUrl":"10.1016/j.jclinepi.2025.111673","url":null,"abstract":"<div><h3>Objectives</h3><div>End-user evaluation of the impact of evidence syntheses is critical to demonstrating value. This study presents results of a survey evaluating the impact of rapid reviews undertaken by two teams based in Melbourne, Australia, and Hamilton, Canada.</div></div><div><h3>Methods</h3><div>Clients were invited to participate in a short written survey following delivery of a rapid review. Survey items encompassed reach, usefulness and format; interactions with the review teams; and overall satisfaction.</div></div><div><h3>Results</h3><div>Twenty-five completed surveys from 53 invitations were received pertaining to 19 rapid reviews conducted between September 2021 and October 2023. Topics encompassed COVID-19, health and behavior change; reports were an average of 31 pages; and were delivered over an average of 62 days. Evaluation findings were positive, with high satisfaction with reports and service delivery; very high satisfaction with report structure and length; good evidence of reach (reports read by decision makers and cited in other documents); and evidence that the rapid reviews made contributions to strategic planning, policy and program funding decisions.</div></div><div><h3>Conclusion</h3><div>Rapid reviews are making impactful contributions, alongside other inputs, to policy and practice. Further research is required to build this evaluation dataset; examine the balance between timeliness and methodological rigor in evidence synthesis; and explore models of delivery and capacity within and outside of government. It is also critical to promote implementation efforts to harness the full potential of rapid reviews and other evidence syntheses to impact the lives of citizens.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"180 ","pages":"Article 111673"},"PeriodicalIF":7.3,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142973160","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Accounting for differential exclusions in the Nordic-European initiative on colorectal cancer trial discloses stronger-than-reported effects of screening colonoscopy 考虑到NordICC试验中的差异排除,结肠镜筛查的效果比报道的要强。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-10 DOI: 10.1016/j.jclinepi.2025.111669
Hermann Brenner , Tim Holland-Letz , Michael Hoffmeister , Thomas Heisser

Objectives

Recently, results on colorectal cancer (CRC) incidence and mortality reduction by the offer of screening colonoscopy were reported for the first time from a randomized controlled trial (RCT), the Nordic-European Initiative on Colorectal Cancer (NordICC) trial. Despite randomization, there was a substantially lower proportion of postrandomization exclusions of CRC cases due to cancer registry-recorded date of diagnosis before recruitment in the invited group than in the usual-care group. We aimed to evaluate the impact of such differential exclusions on the trial's effect estimates on CRC risk.

Study Design and Setting

We compared reported postrandomization exclusions of CRC cases due to cancer registry-recorded date of diagnosis, and we derived adjusted effect estimates on CRC risk accounting for the reported differential postrandomization exclusion of CRC cases in the invited group and the usual-care group.

Results

Reported postrandomization exclusion proportions of CRC cases were originally reported as 52/31,472 (0.17%) and 159/63,133 (0.25%) in the invited and usual-care group, respectively, (P < .005) in an analysis, including participants from all four NordICCstudy countries and as 52/28,277 (0.20%) and 164/56,529 (0.29%) in the recent analysis of 10-year follow-up data from three of the countries (P = .018). Accounting for the differential exclusion proportions increased the estimated CRC risk reduction (95% CI) from originally reported 18% (7%–30%) to 25% (95% CI 13%–35%) in intention-to-screen analysis. Estimated reduction of CRC risk among screening attenders increased from originally reported 31% (17%–45%) to 50% (25%–69%) in adjusted per-protocol analysis.

Conclusion

Accounting for differential postrandomization exclusions of CRC cases leads to stronger-than-reported effect estimates in the so far only RCT on long-term effects of screening colonoscopy.
目的:最近,一项随机对照试验(RCT)——北欧-欧洲结直肠癌倡议(NordICC)试验首次报道了结肠镜筛查降低结直肠癌(CRC)发病率和死亡率的结果。尽管进行了随机化,但随机化后由于招募前癌症登记记录的诊断日期而排除CRC病例的比例在邀请组中明显低于常规护理组。我们的目的是评估这种差异排除对试验对结直肠癌风险的影响。研究设计和设置:我们比较了因癌症登记诊断日期而报道的随机化后排除的CRC病例,并对邀请组和常规护理组报道的随机化后排除CRC病例的差异得出了CRC风险的调整效应估计。结果:最初报道的随机化后CRC病例排除比例在邀请组和常规组分别为52/ 31472(0.17%)和159/ 63133(0.25%)。结论:考虑到随机化后CRC病例排除的差异,在迄今为止唯一的关于结肠镜筛查长期效果的随机对照试验中,结果比报道的效果估计更强。
{"title":"Accounting for differential exclusions in the Nordic-European initiative on colorectal cancer trial discloses stronger-than-reported effects of screening colonoscopy","authors":"Hermann Brenner ,&nbsp;Tim Holland-Letz ,&nbsp;Michael Hoffmeister ,&nbsp;Thomas Heisser","doi":"10.1016/j.jclinepi.2025.111669","DOIUrl":"10.1016/j.jclinepi.2025.111669","url":null,"abstract":"<div><h3>Objectives</h3><div>Recently, results on colorectal cancer (CRC) incidence and mortality reduction by the offer of screening colonoscopy were reported for the first time from a randomized controlled trial (RCT), the Nordic-European Initiative on Colorectal Cancer (NordICC) trial. Despite randomization, there was a substantially lower proportion of postrandomization exclusions of CRC cases due to cancer registry-recorded date of diagnosis before recruitment in the invited group than in the usual-care group. We aimed to evaluate the impact of such differential exclusions on the trial's effect estimates on CRC risk.</div></div><div><h3>Study Design and Setting</h3><div>We compared reported postrandomization exclusions of CRC cases due to cancer registry-recorded date of diagnosis, and we derived adjusted effect estimates on CRC risk accounting for the reported differential postrandomization exclusion of CRC cases in the invited group and the usual-care group.</div></div><div><h3>Results</h3><div>Reported postrandomization exclusion proportions of CRC cases were originally reported as 52/31,472 (0.17%) and 159/63,133 (0.25%) in the invited and usual-care group, respectively, (<em>P</em> &lt; .005) in an analysis, including participants from all four NordICCstudy countries and as 52/28,277 (0.20%) and 164/56,529 (0.29%) in the recent analysis of 10-year follow-up data from three of the countries (<em>P</em> = .018). Accounting for the differential exclusion proportions increased the estimated CRC risk reduction (95% CI) from originally reported 18% (7%–30%) to 25% (95% CI 13%–35%) in intention-to-screen analysis. Estimated reduction of CRC risk among screening attenders increased from originally reported 31% (17%–45%) to 50% (25%–69%) in adjusted per-protocol analysis.</div></div><div><h3>Conclusion</h3><div>Accounting for differential postrandomization exclusions of CRC cases leads to stronger-than-reported effect estimates in the so far only RCT on long-term effects of screening colonoscopy.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"180 ","pages":"Article 111669"},"PeriodicalIF":7.3,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142973157","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Assessing the scientific integrity of the collected work of one author or author group 评估一个作者或一个作者小组收集的作品的科学完整性。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-10 DOI: 10.1016/j.jclinepi.2024.111603
Jeremy Nielsen , Esmée M. Bordewijk , Lyle C. Gurrin , Siddharth Shivantha , Madeline Flanagan , Sue Liu , May M. Linn , Kelly X. Zhou , Rik van Eekelen , Nicholas J.L. Brown , Jim Thornton , Ben W. Mol

Objectives

No published methods for research integrity review include both statistical techniques applied to groups of randomized trials and individual assessment of papers. We propose a method based on practical experience of investigating data integrity across the collected papers of an author or author group.

Study Design and Setting

We report our approach to investigating the collected papers of an author or author group suspected of academic misconduct.

Results

In the investigation of the work of an author or author group, we recommend a systematic search for the work of the involved authors in PubMed, Google Scholar, and the Retraction Watch database, as well as a search of trial registries for unpublished clinical trials. Summary information from studies should be tabulated to assess consistency between study registration, execution, and publication. Each paper should be investigated for unfeasible features of the governance, methodology, execution, results, and reporting of the study. Pairwise comparison of baseline and outcome tables between papers may reveal data duplication or unfeasibly large differences between baseline characteristics in similar studies. Assessment of baseline characteristics from multiple randomized trials using Carlisle’s method can determine whether the data are consistent with a properly executed randomization process, as can checking whether reported baseline characteristics follow expected patterns for random variables such as Benford’s law. If serious concerns are raised, a more thorough investigation should be performed by journals, publishers, and institutions.

Conclusion

These methods provide a systematic and reproducible way to assess the collected work of an author or group of authors.

Plain Language Summary

It is increasingly accepted that papers reporting on clinical studies may contain fraudulent or falsified data, often multiple papers by a single author or author group. Based on our experience assessing the research integrity of collections of papers by one author or author group, we present an approach to these investigations that combines published statistical methods with pragmatic assessment of study feasibility. This will help journals and publishers better identify groups of potentially untrustworthy studies.
目的:没有已发表的研究完整性评价方法既包括应用于随机试验组的统计技术,也包括对论文的个人评估。我们提出了一种基于调查一个作者或作者组收集的论文的数据完整性的实践经验的方法。研究设计和设置:我们报告了我们对涉嫌学术不端行为的作者或作者小组的论文进行调查的方法。结果:在调查一个作者或作者组的工作时,我们建议在PubMed、谷歌Scholar和RetractionWatch数据库中系统地搜索相关作者的工作,并在未发表的临床试验中搜索试验注册库。研究的摘要信息应制成表格,以评估研究注册、执行和发表之间的一致性。每篇论文都应该调查研究的治理、方法、执行、结果和报告的不可行的特征。论文间基线表和结果表的两两比较可能会发现数据重复或类似研究中基线特征之间存在不可思议的巨大差异。使用Carlisle的方法评估多个随机试验的基线特征可以确定数据是否与正确执行的随机化过程一致,也可以检查报告的基线特征是否遵循随机变量(如Benford定律)的预期模式。如果提出了严重的担忧,期刊、出版商和机构应该进行更彻底的调查。结论:这些方法为评价单个或多组作者的文集提供了一种系统的、可重复的方法。
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引用次数: 0
Research participation effects and where to find them: a systematic review of studies on alcohol 研究参与效应及其来源:对酒精研究的系统回顾。
IF 7.3 2区 医学 Q1 HEALTH CARE SCIENCES & SERVICES Pub Date : 2025-01-10 DOI: 10.1016/j.jclinepi.2025.111668
Katarina Ulfsdotter Gunnarsson, Elizabeth S. Collier, Marcus Bendtsen

Objectives

The term ‘research participation effects’ (RPEs) is intended to capture features and artifacts of study design that may affect measured outcomes in ways that introduce bias into research findings, impacting inference and outcome validity. This systematic review aims to identify which RPEs have been studied in the context of alcohol research and provide an overview of estimates of RPEs on self-reported alcohol consumption.

Study Design and Setting

This systematic review summarizes the available evidence on RPEs in alcohol research.

Results

Twenty-seven reports were included in the review. The reports included randomized controlled trials (RCTs), studies-within-a-trial, between-subjects experiments, and qualitative investigations. A range of RPEs were addressed as follows: assessment reactivity (N = 15), being randomized to a waiting list control group (N = 3), the impact of obtaining informed consent (N = 2), experimentally induced social desirability (N = 3), and the Hawthorne effect, either specifically by name (N = 2, one quantitative, one qualitative) or described as general RPE presence (N = 2). The literature provided proportionally stronger evidence in favor of assessment reactivity and waiting list designs affecting alcohol outcomes, contrary to obtaining informed consent or inducing social desirability.

Conclusion

Variation in study quality, terminology, and outcome measures hinder comprehensive understanding and discussion of RPEs at present. Improved knowledge of RPEs and their potential long-term consequences in alcohol research, including a unified lexicon, would enhance trial design and improve the certainty of evidence in alcohol research.
目的:术语“研究参与效应”(RPEs)旨在捕捉研究设计的特征和人为因素,这些特征和人为因素可能以引入研究结果偏差、影响推断和结果效度的方式影响测量结果。本系统综述旨在确定在酒精研究的背景下研究了哪些rpe,并概述了自我报告酒精消费的rpe估计。研究设计和设置:本系统综述总结了酒精研究中研究参与效应的现有证据。结果:共纳入27篇报道。这些报告包括随机对照试验、试验中研究、受试者间实验和定性调查。研究涉及一系列RPE:评估反应性(N=15),随机分配到等待名单对照组(N=3),获得知情同意的影响(N=2),实验诱导的社会可取性(N=3),以及霍桑效应,具体名称(N=2,一个定量,一个定性)或描述为一般RPE存在(N=2)。文献提供了比例更强的证据,支持评估反应性和等候名单设计影响酒精结果,而不是获得知情同意或诱导社会期望。结论:目前,研究质量、术语和结果测量的差异阻碍了对rpe的全面理解和讨论。提高对rpe及其在酒精研究中的潜在长期影响的认识,包括统一的词汇,将加强试验设计并提高酒精研究中证据的确定性。
{"title":"Research participation effects and where to find them: a systematic review of studies on alcohol","authors":"Katarina Ulfsdotter Gunnarsson,&nbsp;Elizabeth S. Collier,&nbsp;Marcus Bendtsen","doi":"10.1016/j.jclinepi.2025.111668","DOIUrl":"10.1016/j.jclinepi.2025.111668","url":null,"abstract":"<div><h3>Objectives</h3><div>The term ‘research participation effects’ (RPEs) is intended to capture features and artifacts of study design that may affect measured outcomes in ways that introduce bias into research findings, impacting inference and outcome validity. This systematic review aims to identify which RPEs have been studied in the context of alcohol research and provide an overview of estimates of RPEs on self-reported alcohol consumption.</div></div><div><h3>Study Design and Setting</h3><div>This systematic review summarizes the available evidence on RPEs in alcohol research.</div></div><div><h3>Results</h3><div>Twenty-seven reports were included in the review. The reports included randomized controlled trials (RCTs), studies-within-a-trial, between-subjects experiments, and qualitative investigations. A range of RPEs were addressed as follows: assessment reactivity (<em>N</em> = 15), being randomized to a waiting list control group (<em>N</em> = 3), the impact of obtaining informed consent (<em>N</em> = 2), experimentally induced social desirability (<em>N</em> = 3), and the Hawthorne effect, either specifically by name (<em>N</em> = 2, one quantitative, one qualitative) or described as general RPE presence (<em>N</em> = 2). The literature provided proportionally stronger evidence in favor of assessment reactivity and waiting list designs affecting alcohol outcomes, contrary to obtaining informed consent or inducing social desirability.</div></div><div><h3>Conclusion</h3><div>Variation in study quality, terminology, and outcome measures hinder comprehensive understanding and discussion of RPEs at present. Improved knowledge of RPEs and their potential long-term consequences in alcohol research, including a unified lexicon, would enhance trial design and improve the certainty of evidence in alcohol research.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"179 ","pages":"Article 111668"},"PeriodicalIF":7.3,"publicationDate":"2025-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142973159","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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Journal of Clinical Epidemiology
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