Journal of Clinical Epidemiology最新文献

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The GIN-McMaster Guideline Development Checklist extension for engagement GIN-McMaster指南开发清单(GDC)扩展参与。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-17 DOI: 10.1016/j.jclinepi.2025.111727
Jennifer Petkovic , Olivia Magwood , Thomas W. Concannon , Elie A. Akl , Joanne Khabsa , Lyubov Lytvyn , Vivian Welch , Marc T. Avey , Soumyadeep Bhaumik , Angus Gunn , Ana Marusic , Lawrence Mbuagbaw , Zachary Munn , Marisha E. Palm , Danielle Pollock , Michael Saginur , Imad Bou Akl , Thurayya Arayssi , Asma Ben Brahem , Tammy Clifford , Peter Tugwell
{"title":"The GIN-McMaster Guideline Development Checklist extension for engagement","authors":"Jennifer Petkovic , Olivia Magwood , Thomas W. Concannon , Elie A. Akl , Joanne Khabsa , Lyubov Lytvyn , Vivian Welch , Marc T. Avey , Soumyadeep Bhaumik , Angus Gunn , Ana Marusic , Lawrence Mbuagbaw , Zachary Munn , Marisha E. Palm , Danielle Pollock , Michael Saginur , Imad Bou Akl , Thurayya Arayssi , Asma Ben Brahem , Tammy Clifford , Peter Tugwell","doi":"10.1016/j.jclinepi.2025.111727","DOIUrl":"10.1016/j.jclinepi.2025.111727","url":null,"abstract":"<div><h3>Objectives</h3><div>Better engagement of diverse groups of interest-holders in the development of health guidelines has been proposed to improve their usefulness, implementability, and acceptability. Guidelines shape clinical or public health practice decision-making. Trustworthy guidelines are systematically developed documents that include actionable statements based on evidence and a formal, structured and transparent decision process. This paper describes the Guidelines International Network (GIN)-McMaster Guideline Development Checklist (GDC) Extension for Engagement to assist developers with engaging multiple interest-holders throughout all topics of guideline development.</div></div><div><h3>Study Design and Setting</h3><div>To produce this checklist extension, we conducted a 3-phase mixed methods study. First, we identified 10 groups of interest-holders to be engaged in health guideline development: patients, the public, providers, program managers, principal investigators, payers/purchasers of health services, payers/funders of health research, policymakers, peer-reviewed journal editors, and product makers identified in previous work and recruited coleads to represent these groups (<em>n</em> = 26 total).</div></div><div><h3>Results</h3><div>We conducted a series of reviews to identify existing methods and barriers/facilitators for engagement, approaches to managing conflicts of interest, and describe the impacts of engagement on the guideline development process. The results of these reviews informed the development of an online survey for which we received 195 responses. We clarified these results through 43 key informant interviews with interest-holders. The final GDC extension checklist was determined based on consensus methods with our coleads.</div></div><div><h3>Conclusion</h3><div>This paper presents the GIN-McMaster GDC Extension for Engagement. This checklist provides interest-holder–informed recommendations for providing advice/feedback or participating in decision-making in guideline development.</div></div><div><h3>Plain Language Summary</h3><div>Health-care guidelines are formal recommendations for diagnosing and managing medical conditions or public health concerns. The steps involved in developing guidelines include from planning the process, defining the focus of the guideline and the questions it should answer, and summarizing the evidence. The Guidelines International Network-McMaster Guideline Development checklist includes 146 steps of guideline development grouped into 18 topics. Our work extends this checklist to provide guidance on how to engage with people from different groups of ‘interest-holders’ throughout guideline development. These interest-holders include (1) patients, (2) members of the public, (3) health-care providers, (4) people who manage and operate health programs, (5) researchers, (6) people who pay for or purchase health services, (7) policymakers, (8) people who pay for health research","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111727"},"PeriodicalIF":7.3,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143460550","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
Review of the quality of reporting of statistical analysis plans for cluster randomized trials 聚类随机试验统计分析计划报告质量的评价。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-16 DOI: 10.1016/j.jclinepi.2025.111726
Jacqueline Y. Thompson , Julia Shaw , Samuel I. Watson , Yixin Wang , Clare Robinson , Monica Taljaard , Karla Hemming
{"title":"Review of the quality of reporting of statistical analysis plans for cluster randomized trials","authors":"Jacqueline Y. Thompson ,&nbsp;Julia Shaw ,&nbsp;Samuel I. Watson ,&nbsp;Yixin Wang ,&nbsp;Clare Robinson ,&nbsp;Monica Taljaard ,&nbsp;Karla Hemming","doi":"10.1016/j.jclinepi.2025.111726","DOIUrl":"10.1016/j.jclinepi.2025.111726","url":null,"abstract":"<div><h3>Background and Objectives</h3><div>The guideline for the content of Statistical Analysis Plans (SAPs) outlines recommendations for items to be included in SAPs. As yet there is no specific tailoring of this guideline for Cluster Randomized Trials (CRTs). There has also been no assessment of reporting quality of SAPs against this guideline. Our intention is to identify how well a sample of SAPs for CRTs are adhering to the reporting of key items in the current guidelines, as well as additional analysis aspects considered to be important in CRTs.</div></div><div><h3>Methods</h3><div>We include (i) fully published standalone SAPs identified via Ovid-MEDLINE and (ii) SAPs published as supplementary material or appendices to the final published report identified by searching an existing database of nearly 800 CRTs.</div></div><div><h3>Results</h3><div>The search identified 85 unique SAPs: 26 were published in standalone format and 59 were supplementary material to the full trial report. There was mixed clarity in reporting of items related to the current guideline (eg, most (61/85, 72%) reported what covariates will be included in any adjustment; but fewer (26/85, 31%) reported what method will be used to estimate the absolute measure of effect). Considering additional aspects important for CRTs, the majority (79/85, 93%) included a plan to allow for clustering in the analysis; but fewer (10/40, 25%) reported how a small number of clusters would be accommodated (this was only considered relevant for the subset of CRTs with fewer than 40 clusters). Few (5/85, 6%) reported how the intracluster correlation would be estimated. Few clearly reported statistical targets of inference: in only two SAPs (2/85, 2%) it was clear whether the objectives were related to the individual or cluster-level average; in trials where relevant, only three (3/70, 4%) clearly reported whether the objectives were related to the marginal or cluster-specific effect.</div></div><div><h3>Conclusion</h3><div>This review has identified specific areas of poor quality of reporting that might need additional consideration when developing the guidance for the reporting of SAPs for CRTs.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111726"},"PeriodicalIF":7.3,"publicationDate":"2025-02-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143442696","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
Author’s reply: “The importance of properly specifying your target trial emulation: commentary on Mésidor et al.” 作者的回复是:“正确指定目标试验模拟的重要性:对msamsidor等人的评论。”
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-13 DOI: 10.1016/j.jclinepi.2025.111723
Miceline Mésidor, Caroline Sirois, Jason Robert Guertin, Paul Poirier, Claudia Blais, Denis Talbot
{"title":"Author’s reply: “The importance of properly specifying your target trial emulation: commentary on Mésidor et al.”","authors":"Miceline Mésidor,&nbsp;Caroline Sirois,&nbsp;Jason Robert Guertin,&nbsp;Paul Poirier,&nbsp;Claudia Blais,&nbsp;Denis Talbot","doi":"10.1016/j.jclinepi.2025.111723","DOIUrl":"10.1016/j.jclinepi.2025.111723","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"180 ","pages":"Article 111723"},"PeriodicalIF":7.3,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426697","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
"Treatment effects properly defined are not due to placebo: response to Schmidt et al": author's reply. "正确定义的治疗效果并非安慰剂所致:对施密特等人的回应":作者回复。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-13 DOI: 10.1016/j.jclinepi.2025.111721
Stefan Schmidt, Martin Loef, Thomas Ostermann, Harald Walach
{"title":"\"Treatment effects properly defined are not due to placebo: response to Schmidt et al\": author's reply.","authors":"Stefan Schmidt, Martin Loef, Thomas Ostermann, Harald Walach","doi":"10.1016/j.jclinepi.2025.111721","DOIUrl":"10.1016/j.jclinepi.2025.111721","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111721"},"PeriodicalIF":7.3,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Quantitative assessment of inconsistency in meta-analysis using decision thresholds with two new indices 用两个新指标的决策阈值定量评估meta分析的不一致性。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-13 DOI: 10.1016/j.jclinepi.2025.111725
Bernardo Sousa-Pinto , Ignacio Neumann , Rafael José Vieira , Antonio Bognanni , Manuel Marques-Cruz , Sara Gil-Mata , Simone Mordue , Clareece Nevill , Gianluca Baio , Paul Whaley , Guido Schwarzer , James Steele , Gavin Stewart , Holger J. Schünemann , Luís Filipe Azevedo
{"title":"Quantitative assessment of inconsistency in meta-analysis using decision thresholds with two new indices","authors":"Bernardo Sousa-Pinto ,&nbsp;Ignacio Neumann ,&nbsp;Rafael José Vieira ,&nbsp;Antonio Bognanni ,&nbsp;Manuel Marques-Cruz ,&nbsp;Sara Gil-Mata ,&nbsp;Simone Mordue ,&nbsp;Clareece Nevill ,&nbsp;Gianluca Baio ,&nbsp;Paul Whaley ,&nbsp;Guido Schwarzer ,&nbsp;James Steele ,&nbsp;Gavin Stewart ,&nbsp;Holger J. Schünemann ,&nbsp;Luís Filipe Azevedo","doi":"10.1016/j.jclinepi.2025.111725","DOIUrl":"10.1016/j.jclinepi.2025.111725","url":null,"abstract":"<div><h3>Objectives</h3><div>In evidence synthesis, inconsistency is typically assessed visually and with the <em>I</em><sup>2</sup> and the Q statistics. However, these measures have important limitations (i) if there are few primary studies of small sample sizes or (ii) if there are multiple studies with precise estimates. In addition, with the increasing use of decision thresholds (DT), for example in Grading of Recommendations Assessment, Development and Evaluation evidence to decision (EtD) frameworks, inconsistency judgments can be anchored around DTs. In this article, we developed quantitative measures to assess inconsistency based on DTs.</div></div><div><h3>Study Design and Setting</h3><div>We developed two measures to quantify inconsistency based on DTs – the decision inconsistency (<em>DI</em>) and the across-studies inconsistency (<em>ASI</em>) indices. The <em>DI</em> and the <em>ASI</em> are based on the distribution of the posterior samples studies’ effect sizes (ES) across interpretation categories defined by DTs. We developed these indices for the Bayesian context, followed by a frequentist extension.</div></div><div><h3>Results</h3><div>The <em>DI</em> informs on the <em>overall inconsistency of ESs</em> across interpretation categories, while the <em>ASI</em> quantifies how <em>different</em> studies are compared to each other (in relation to interpretation categories) based on absolute effects. A <em>DI</em> ≥ 50% and an ASI ≥ 25% are suggestive of important inconsistency. We provide an R package (metainc) and a web tool (<span><span>https://metainc.med.up.pt/</span><svg><path></path></svg></span>) to support the computation of the <em>DI</em> and <em>ASI</em>, including in the context of sensitivity analyses assessing the impact of potential uncertainty in inconsistency.</div></div><div><h3>Conclusion</h3><div>The <em>DI</em> and the <em>ASI</em> can contribute to quantitatively assess inconsistency, particularly as DTs are gaining recognition in evidence synthesis and health decision-making.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111725"},"PeriodicalIF":7.3,"publicationDate":"2025-02-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426738","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Treatment effects, properly defined, are not due to placebo: response to Schmidt et al. 正确定义的治疗效果不是由于安慰剂:对Schmidt等人的反应。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-12 DOI: 10.1016/j.jclinepi.2025.111722
Stephen Rhodes
{"title":"Treatment effects, properly defined, are not due to placebo: response to Schmidt et al.","authors":"Stephen Rhodes","doi":"10.1016/j.jclinepi.2025.111722","DOIUrl":"10.1016/j.jclinepi.2025.111722","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111722"},"PeriodicalIF":7.3,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143425788","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cochrane reviews’ authorship has become more gender-diverse but remains geographically concentrated: a meta-research study Cochrane Reviews的作者变得更加性别多样化,但仍保持地域集中:一项元研究。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-11 DOI: 10.1016/j.jclinepi.2025.111719
Ahmad Sofi-Mahmudi , Jana Stojanova , Elpida Vounzoulaki , Eve Tomlinson , Ana Beatriz Pizarro , Sahar Khademioore , Etienne Ngeh , Amin Sharifan , Lucy Elauteri Mrema , Alexis Ceecee Britten-Jones , Santiago Castiello de Obeso , Vivian A. Welch , Lawrence Mbuagbaw , Peter Tugwell
{"title":"Cochrane reviews’ authorship has become more gender-diverse but remains geographically concentrated: a meta-research study","authors":"Ahmad Sofi-Mahmudi ,&nbsp;Jana Stojanova ,&nbsp;Elpida Vounzoulaki ,&nbsp;Eve Tomlinson ,&nbsp;Ana Beatriz Pizarro ,&nbsp;Sahar Khademioore ,&nbsp;Etienne Ngeh ,&nbsp;Amin Sharifan ,&nbsp;Lucy Elauteri Mrema ,&nbsp;Alexis Ceecee Britten-Jones ,&nbsp;Santiago Castiello de Obeso ,&nbsp;Vivian A. Welch ,&nbsp;Lawrence Mbuagbaw ,&nbsp;Peter Tugwell","doi":"10.1016/j.jclinepi.2025.111719","DOIUrl":"10.1016/j.jclinepi.2025.111719","url":null,"abstract":"<div><h3>Objectives</h3><div>The aim of this study was to examine the distribution of country, region, language, and gender diversity in the authorship of Cochrane reviews and compare it to non-Cochrane systematic reviews.</div></div><div><h3>Study Design and Setting</h3><div>We retrieved all published articles from the Cochrane Library (until November 6, 2023) using a web crawling technique that extracted prespecified data fields, including publication date, review category, and author affiliations. For comparison, non-Cochrane systematic reviews were identified through PubMed using E-utility calls. We determined the country, region of affiliations and gender of the first, corresponding, and last authors for Cochrane reviews; the same fields were determined for first authors only for non-Cochrane reviews due to data availability. Trends in geographical and gender diversity over time were evaluated using logistic regression. Fisher's exact test was used for comparisons. Diversity trends between Cochrane and non-Cochrane reviews were explored through visual presentation, Pearson's product-moment correlation, and the Granger Causality Test.</div></div><div><h3>Results</h3><div>This comprehensive analysis included 22,681 Cochrane reviews and 224,484 non-Cochrane reviews. Cochrane reviews showed increasing diversity in several areas: representation of first authors from non-English speaking countries rose substantially (from 16.7% in 1996 to 42.8% in 2023), and female first authorship more than tripled (from 15.0% in 1996 to 55.6% in 2023). Representation from lower-and-middle-income countries (LMICs) in Cochrane reviews has declined recently (from a peak of 23.2% in 2012 to 18.4% in 2023). Among Cochrane Review Groups, diversity varied notably, with Sexually Transmitted Infections achieving the highest representation from LMICs (68.1% of first authors). In 2023, non-Cochrane reviews showed higher representation from non-English speaking countries (56.9%) and LMICs (50.8%) compared to Cochrane reviews. The patterns of gender diversity between Cochrane and non-Cochrane reviews showed strong correlations for female first authorship (<em>r</em> = 0.829, <em>P</em> &lt; .001), suggesting parallel evolution over time.</div></div><div><h3>Conclusion</h3><div>Both Cochrane and non-Cochrane reviews demonstrate important progress in author diversity, particularly in gender representation and inclusion of authors from non-English speaking countries. While non-Cochrane reviews show stronger representation from LMICs, both review sources reflect the evolving landscape of global evidence synthesis.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111719"},"PeriodicalIF":7.3,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143416241","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
Endpoint assessment via routinely collected data generates estimates comparable to randomized controlled trial data: analysis of a cluster-randomized trial on fall injury prevention 终点评估通过常规收集的数据产生的估计与随机对照试验数据相当:对跌倒损伤预防的集群随机试验的分析。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-10 DOI: 10.1016/j.jclinepi.2025.111718
David A. Ganz , Erich J. Greene , Nancy K. Latham , Michael Kane , Lillian C. Min , Thomas M. Gill , David B. Reuben , Peter Peduzzi , Denise Esserman
{"title":"Endpoint assessment via routinely collected data generates estimates comparable to randomized controlled trial data: analysis of a cluster-randomized trial on fall injury prevention","authors":"David A. Ganz ,&nbsp;Erich J. Greene ,&nbsp;Nancy K. Latham ,&nbsp;Michael Kane ,&nbsp;Lillian C. Min ,&nbsp;Thomas M. Gill ,&nbsp;David B. Reuben ,&nbsp;Peter Peduzzi ,&nbsp;Denise Esserman","doi":"10.1016/j.jclinepi.2025.111718","DOIUrl":"10.1016/j.jclinepi.2025.111718","url":null,"abstract":"<div><h3>Background and Objectives</h3><div>Routinely collected data (RCD) from healthcare claims and encounters are increasingly used for outcomes in randomized trials; however, methods for estimating the validity and relative precision of RCD-derived outcomes compared to those from conventional outcome ascertainment are limited. We developed an approach to measuring validity and relative precision of RCD and quantifying uncertainty.</div></div><div><h3>Methods</h3><div>We reanalyzed data from the Strategies to Reduce Injuries and Develop Confidence in Elders (STRIDE) cluster-randomized, controlled trial. Eighty-six primary care practices in 10 US healthcare systems were randomized to either a multifactorial intervention delivered by nurse falls care managers, or enhanced usual care, with 5451 persons age ≥ 70 at increased fall injury risk enrolled in the study. We estimated the hazard ratio (HR) and confidence interval (CI) for STRIDE's primary outcome (time to first serious fall injury) using original study data and RCD. The ratio of the RCD HR to original HR (“ratio of HRs”) measured validity. The confidence limit ratio (CLR; upper divided by lower confidence limits of CI) measured precision, with the ratio of the CLR with RCD to the CLR from the original study data (“ratio of CLRs”), measuring relative precision. We estimated uncertainty around the ratio of HRs and ratio of CLRs using bootstrapped 95% CIs and performed sensitivity analyses to assess the effects of adaptations needed to use RCD.</div></div><div><h3>Results</h3><div>Among the original sample of 5451 study participants, 5036 (92%) were linked to RCD. The intervention to control HR was 0.91 (95% CI: 0.78–1.07) in RCD, compared to 0.92 (95% CI: 0.80–1.06) in the original data. Using all RCD through STRIDE's administrative end date, the ratio of HRs was 1.00 (95% CI: 0.89–1.11) and ratio of CLRs was 1.03 (95% CI: 0.96–1.06). The CI around ratio of HRs was about three-fold wider for RCD than for the original STRIDE data in individuals who linked to RCD. Relative precision of RCD improved with increased length of follow-up.</div></div><div><h3>Conclusion</h3><div>Relying solely on RCD to ascertain the primary outcome in STRIDE would have resulted in similar point estimates and confidence limits for the treatment effect as in the original data. However, there was meaningful uncertainty around the estimate of validity. Efforts to validate RCD-derived outcomes for use as clinical trial endpoints should include measurement of uncertainty around validity estimates.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111718"},"PeriodicalIF":7.3,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143411412","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
Trial emulation to assess the effect of surgery on survival when there are competing risks, with application to patients with thoracic aortic aneurysms 试验模拟评估存在竞争风险时手术对生存的影响,并应用于胸主动脉瘤患者。
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-08 DOI: 10.1016/j.jclinepi.2025.111714
James Murray , Caroline Chesang , Steve Large , Colin Bicknell , Carol Freeman , Ruth H. Keogh , Linda D. Sharples
{"title":"Trial emulation to assess the effect of surgery on survival when there are competing risks, with application to patients with thoracic aortic aneurysms","authors":"James Murray ,&nbsp;Caroline Chesang ,&nbsp;Steve Large ,&nbsp;Colin Bicknell ,&nbsp;Carol Freeman ,&nbsp;Ruth H. Keogh ,&nbsp;Linda D. Sharples","doi":"10.1016/j.jclinepi.2025.111714","DOIUrl":"10.1016/j.jclinepi.2025.111714","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Objectives&lt;/h3&gt;&lt;div&gt;This study extends methods to estimate average causal effect of aneurysm repair surgery on (i) overall survival and (ii) aneurysm-related mortality, accounting for competing risks using data from the Effective Treatment for Thoracic Aortic Aneurysm (ETTAA) cohort.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Study Design and Setting&lt;/h3&gt;&lt;div&gt;ETTAA, a prospective cohort study, recruited 886 patients between 2014 and 2018. Patients were linked to UK national hospital and mortality databases by National Health Service digital and followed-up for later surgeries and deaths. We compared a strategy of open or endovascular surgery (whichever appropriate) within 12 months of enrollment to ETTAA with no surgery within 12 months using the trial emulation framework and cloning-censoring-weighting (CCW) analysis. Key confounders at baseline were controlled for using inverse probability weighting methods.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;In complete case analysis, if everyone received surgery within a 12-month grace period, an estimated 7-year survival probability was 57.4% (95% CI: 47.3%, 67.4%) vs 49.9% (44.0%, 55.0%) if no one received surgery. This benefit was primarily attributable to reduction in aneurysm-related deaths (difference −8.7%, 95% CI: −14.0%, −3.9%), with no significant effect on deaths from other causes. The findings were consistent under sensitivity analyses, including multiple imputation of missing confounders. Our CCW approach addressed selection-for-treatment, allowed for surgery to be received within a grace period, and used appropriate methods to separate aneurysm-related mortality from competing risks.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclusion&lt;/h3&gt;&lt;div&gt;The study demonstrates the utility of trial emulation and counterfactual methods in estimation of causal effects on competing risks using observational data. The findings suggest a benefit for aneurysm-related survival up to 7 years after enrollment.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Plain Language Summary&lt;/h3&gt;&lt;div&gt;This study shows how to estimate effects of surgery on different causes of death, when we cannot do a clinical trial, and illustrates this using an example from heart surgery. The aorta is the main artery that carries oxygen-rich blood from the heart to the body. In some people, a part of the vessel wall becomes weak and loses its elastic properties, so it doesn’t return to its normal shape after the blood has passed through. This can lead to swelling or bulging in the aorta, called an aneurysm. A thoracic aortic aneurysm, or TAA for short, is an aneurysm in the section of the aorta in the chest (&lt;span&gt;&lt;span&gt;https://www.bhf.org.uk/informationsupport/conditions/thoracic-aortic-aneurysm&lt;/span&gt;&lt;svg&gt;&lt;path&gt;&lt;/path&gt;&lt;/svg&gt;&lt;/span&gt;).&lt;/div&gt;&lt;div&gt;We have used data from the Effective Treatment for Thoracic Aortic Aneurysm (ETTAA) study, which investigated aneurysm growth rates, patient outcomes, quality of life, and costs, in 886 patients diagnosed with TAA. ETTAA compared two surgical treatments, &lt;em&gt;Ope","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111714"},"PeriodicalIF":7.3,"publicationDate":"2025-02-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143392380","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
Editors’ Choice March 2025 编辑选择2025年3月
IF 7.3 2区 医学
Journal of Clinical Epidemiology Pub Date : 2025-02-08 DOI: 10.1016/j.jclinepi.2025.111720
Andrea C. Tricco, David Tovey
{"title":"Editors’ Choice March 2025","authors":"Andrea C. Tricco,&nbsp;David Tovey","doi":"10.1016/j.jclinepi.2025.111720","DOIUrl":"10.1016/j.jclinepi.2025.111720","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"179 ","pages":"Article 111720"},"PeriodicalIF":7.3,"publicationDate":"2025-02-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143422178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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