Registry-based randomized controlled trials (RRCTs) can provide internally valid results in a real-world context at relatively low effort and cost. However, the main characteristics, the extent to which the registry is utilized (eg, proportion of data from registry) and registry-related limitations are not well characterized. This methodological review of RRCTs aims to analyze the trial design features, investigate potential usage options, and identify possible limitations of using registry data for randomized controlled trials (RCTs).
Study Design and Setting
A systematic search in PubMed for ongoing and published RRCTs was conducted up to February 2, 2023. Studies that reported at least one outcome derived from a registry were included. Study selection was independently performed by two reviewers. All data were extracted into a standardized table, and descriptive statistics were generated.
Results
We included 162 RRCTs (41 protocols and 121 studies). Most RRCTs were multicenter trials (n = 127; 78.4%) comprising a large number of participants (median = 1787; range = 41 to 683,927) and a long follow-up period (median = 60 months; range = 1 to 367 months) with a minimal loss to follow-up. The inclusion criteria of participants were mostly broadly defined. Types of interventions ranged from surgical procedures to behavioral interventions, and almost half of the interventions (46.9%) had a preventive purpose. The main registry outcome was mostly a clinical endpoint (40.1%) or a composite endpoint of major clinical events (30.9%) that was objectively measurable. We found different degrees of registry utilization, ranging from the exclusive use of long-term monitoring of previously published data to the more comprehensive registry utilization for patient recruitment, endpoint collection, and long-term follow-up. Limitations related to the use of registry data comprised potential coding errors or incomplete data (eg, due to under-recording of mild cases). In addition, technical challenges must be considered (eg, failed linkages or time-delayed data entry).
Conclusion
A broad spectrum of potential usage options and usage extent of registry data exist. Our analysis suggests that in many cases, the potential of using registry data and thus their benefits were not fully utilized. In addition, the study illustrates that there is not a single, unified methodology for designing RRCTs but that registries can support RCTs in various ways. Therefore, future RRCTs should specify for what purposes and to what extent registries were utilized. Moreover, a clear definition and taxonomy of RRCTs appears necessary for facilitating future dialogue and research on RRCTs.
{"title":"A methodological review identified several options for utilizing registries for randomized controlled trials","authors":"Luisa Urban , Nina Haller , Dawid Pieper , Tim Mathes","doi":"10.1016/j.jclinepi.2024.111614","DOIUrl":"10.1016/j.jclinepi.2024.111614","url":null,"abstract":"<div><h3>Objectives</h3><div>Registry-based randomized controlled trials (RRCTs) can provide internally valid results in a real-world context at relatively low effort and cost. However, the main characteristics, the extent to which the registry is utilized (eg, proportion of data from registry) and registry-related limitations are not well characterized. This methodological review of RRCTs aims to analyze the trial design features, investigate potential usage options, and identify possible limitations of using registry data for randomized controlled trials (RCTs).</div></div><div><h3>Study Design and Setting</h3><div>A systematic search in PubMed for ongoing and published RRCTs was conducted up to February 2, 2023. Studies that reported at least one outcome derived from a registry were included. Study selection was independently performed by two reviewers. All data were extracted into a standardized table, and descriptive statistics were generated.</div></div><div><h3>Results</h3><div>We included 162 RRCTs (41 protocols and 121 studies). Most RRCTs were multicenter trials (<em>n</em> = 127; 78.4%) comprising a large number of participants (median = 1787; range = 41 to 683,927) and a long follow-up period (median = 60 months; range = 1 to 367 months) with a minimal loss to follow-up. The inclusion criteria of participants were mostly broadly defined. Types of interventions ranged from surgical procedures to behavioral interventions, and almost half of the interventions (46.9%) had a preventive purpose. The main registry outcome was mostly a clinical endpoint (40.1%) or a composite endpoint of major clinical events (30.9%) that was objectively measurable. We found different degrees of registry utilization, ranging from the exclusive use of long-term monitoring of previously published data to the more comprehensive registry utilization for patient recruitment, endpoint collection, and long-term follow-up. Limitations related to the use of registry data comprised potential coding errors or incomplete data (eg, due to under-recording of mild cases). In addition, technical challenges must be considered (eg, failed linkages or time-delayed data entry).</div></div><div><h3>Conclusion</h3><div>A broad spectrum of potential usage options and usage extent of registry data exist. Our analysis suggests that in many cases, the potential of using registry data and thus their benefits were not fully utilized. In addition, the study illustrates that there is not a single, unified methodology for designing RRCTs but that registries can support RCTs in various ways. Therefore, future RRCTs should specify for what purposes and to what extent registries were utilized. Moreover, a clear definition and taxonomy of RRCTs appears necessary for facilitating future dialogue and research on RRCTs.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111614"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142677474","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111606
Michael Colacci , Yu Qing Huang , Gemma Postill , Pavel Zhelnov , Orna Fennelly , Amol Verma , Sharon Straus , Andrea C. Tricco
Background and Objectives
Clinical machine learning (ML) technologies can sometimes be biased and their use could exacerbate health disparities. The extent to which bias is present, the groups who most frequently experience bias, and the mechanism through which bias is introduced in clinical ML applications is not well described. The objective of this study was to examine instances of bias in clinical ML models. We identified the sociodemographic subgroups PROGRESS that experienced bias and the reported mechanisms of bias introduction.
Methods
We searched MEDLINE, EMBASE, PsycINFO, and Web of Science for all studies that evaluated bias on sociodemographic factors within ML algorithms created for the purpose of facilitating clinical care. The scoping review was conducted according to the Joanna Briggs Institute guide and reported using the PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) extension for scoping reviews.
Results
We identified 6448 articles, of which 760 reported on a clinical ML model and 91 (12.0%) completed a bias evaluation and met all inclusion criteria. Most studies evaluated a single sociodemographic factor (n = 56, 61.5%). The most frequently evaluated sociodemographic factor was race (n = 59, 64.8%), followed by sex/gender (n = 41, 45.1%), and age (n = 24, 26.4%), with one study (1.1%) evaluating intersectional factors. Of all studies, 74.7% (n = 68) reported that bias was present, 18.7% (n = 17) reported bias was not present, and 6.6% (n = 6) did not state whether bias was present. When present, 87% of studies reported bias against groups with socioeconomic disadvantage.
Conclusion
Most ML algorithms that were evaluated for bias demonstrated bias on sociodemographic factors. Furthermore, most bias evaluations concentrated on race, sex/gender, and age, while other sociodemographic factors and their intersection were infrequently assessed. Given potential health equity implications, bias assessments should be completed for all clinical ML models.
背景：临床机器学习（ML）技术有时可能存在偏见，使用这些技术可能会加剧健康差异。关于临床机器学习应用中存在偏差的程度、最常出现偏差的群体以及引入偏差的机制，目前还没有很好的描述。本研究的目的是检查临床 ML 模型中的偏差实例。我们确定了出现偏倚的社会人口亚群（使用 PROGRESS-Plus 框架），以及报告的偏倚引入机制。 方法：我们检索了 MEDLINE、EMBASE、PsycINFO 和 Web of Science，以查找所有评估为促进临床护理而创建的 ML 算法中社会人口因素偏倚的研究。范围界定综述根据 JBI 指南进行，并使用范围界定综述的 PRISMA 扩展报告：我们确定了 6448 篇文章，其中 760 篇报告了临床 ML 模型，91 篇（12.0%）完成了偏倚评估并符合所有纳入标准。大多数研究只评估了一个社会人口因素（n=56，61.5%）。最常评估的社会人口因素是种族（n=59，64.8%），其次是性/性别（n=41，45.1%）和年龄（n=24，26.4%），有一项研究（1.1%）评估了交叉因素。在所有研究中，74.7%（n=68）报告存在偏见，18.7%（n=17）报告不存在偏见，6.6%（n=6）未说明是否存在偏见。87%的研究报告称存在针对社会经济弱势群体的偏见：结论：大多数被评估为存在偏差的 ML 算法都显示出社会人口因素方面的偏差。此外，大多数偏倚评估都集中在种族、性别和年龄上，而其他社会人口因素及其交叉因素很少得到评估。考虑到潜在的健康公平影响，所有临床 ML 模型都应完成偏倚评估。
{"title":"Sociodemographic bias in clinical machine learning models: a scoping review of algorithmic bias instances and mechanisms","authors":"Michael Colacci , Yu Qing Huang , Gemma Postill , Pavel Zhelnov , Orna Fennelly , Amol Verma , Sharon Straus , Andrea C. Tricco","doi":"10.1016/j.jclinepi.2024.111606","DOIUrl":"10.1016/j.jclinepi.2024.111606","url":null,"abstract":"<div><h3>Background and Objectives</h3><div>Clinical machine learning (ML) technologies can sometimes be biased and their use could exacerbate health disparities. The extent to which bias is present, the groups who most frequently experience bias, and the mechanism through which bias is introduced in clinical ML applications is not well described. The objective of this study was to examine instances of bias in clinical ML models. We identified the sociodemographic subgroups PROGRESS that experienced bias and the reported mechanisms of bias introduction.</div></div><div><h3>Methods</h3><div>We searched MEDLINE, EMBASE, PsycINFO, and Web of Science for all studies that evaluated bias on sociodemographic factors within ML algorithms created for the purpose of facilitating clinical care. The scoping review was conducted according to the Joanna Briggs Institute guide and reported using the PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) extension for scoping reviews.</div></div><div><h3>Results</h3><div>We identified 6448 articles, of which 760 reported on a clinical ML model and 91 (12.0%) completed a bias evaluation and met all inclusion criteria. Most studies evaluated a single sociodemographic factor (<em>n</em> = 56, 61.5%). The most frequently evaluated sociodemographic factor was race (<em>n</em> = 59, 64.8%), followed by sex/gender (<em>n</em> = 41, 45.1%), and age (<em>n</em> = 24, 26.4%), with one study (1.1%) evaluating intersectional factors. Of all studies, 74.7% (<em>n</em> = 68) reported that bias was present, 18.7% (<em>n</em> = 17) reported bias was not present, and 6.6% (<em>n</em> = 6) did not state whether bias was present. When present, 87% of studies reported bias against groups with socioeconomic disadvantage.</div></div><div><h3>Conclusion</h3><div>Most ML algorithms that were evaluated for bias demonstrated bias on sociodemographic factors. Furthermore, most bias evaluations concentrated on race, sex/gender, and age, while other sociodemographic factors and their intersection were infrequently assessed. Given potential health equity implications, bias assessments should be completed for all clinical ML models.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111606"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142631723","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111617
Andrada Ciuca , Siddharth Banka , Tara Clancy , Simon Jones , Jamie J. Kirkham , William G. Newman , Katherine Payne , Ramona Moldovan
Objectives
Advanced therapy medicinal products (ATMPs) are medicines based on genes, tissues, or cells and can include gene therapy, somatic-cell therapy, and tissue-engineered medicines. Patient-reported outcomes (PROs) are reports on health and well-being that come directly from the individual without external interpretation. Patient-reported outcome measures (PROMs) are questionnaires aimed at assessing the individual and subjective experience with health and other psychosocial aspects. The aim of the present review is to assess the extent and quality of PROs and PROMs used in orphan ATMP trials.
Study Design and Setting
The database from National Health Service Special Pharmacy Service horizon scanning was searched on 27 March 2024 to identify all ATMPs for orphan conditions. Clinical trial protocols were included in this review if they investigated ATMPs for orphan conditions and were published in clinical trial databases.
Results
A total of 100 trials were included. These accounted for 64 conditions. Only 37% (37/100) of the trials included PROs. Overall, 17 different types of PROs were identified across the trials. Quality of life (QoL) and health-related quality of life (HRQoL) were the most frequent PROs found in 18% (18/100) and 13% (13/100) of the trials, respectively. A total of 33 PROMs were identified. Of these, 57% (19/33) were HRQoL (89% [17/19]) or QoL (11% [2/19]) measures. Of the HRQoL measures identified, 71% (12/17) were disease specific and 29% (5/17) were generic. Of the non-QoL PROMs, 29% (4/14) were designed to measure pain and 71% (10/14) PROMs focused on other psychological outcomes, including anxiety and depression.
Conclusion
Our results show that only 37% of the orphan ATMP trials include patient-reported outcomes and measures. This highlights the urgent need for relevant PROs/PROMs that capture benefits and harms and assimilation of existing PROMs for better comparison between or within conditions. It is essential to include and reflect the patients' experience so that those intended to benefit from the research have the opportunity to influence its direction.
{"title":"Patient-reported outcomes and measures are under-utilised in advanced therapy medicinal products trials for orphan conditions","authors":"Andrada Ciuca , Siddharth Banka , Tara Clancy , Simon Jones , Jamie J. Kirkham , William G. Newman , Katherine Payne , Ramona Moldovan","doi":"10.1016/j.jclinepi.2024.111617","DOIUrl":"10.1016/j.jclinepi.2024.111617","url":null,"abstract":"<div><h3>Objectives</h3><div>Advanced therapy medicinal products (ATMPs) are medicines based on genes, tissues, or cells and can include gene therapy, somatic-cell therapy, and tissue-engineered medicines. Patient-reported outcomes (PROs) are reports on health and well-being that come directly from the individual without external interpretation. Patient-reported outcome measures (PROMs) are questionnaires aimed at assessing the individual and subjective experience with health and other psychosocial aspects. The aim of the present review is to assess the extent and quality of PROs and PROMs used in orphan ATMP trials.</div></div><div><h3>Study Design and Setting</h3><div>The database from National Health Service Special Pharmacy Service horizon scanning was searched on 27 March 2024 to identify all ATMPs for orphan conditions. Clinical trial protocols were included in this review if they investigated ATMPs for orphan conditions and were published in clinical trial databases.</div></div><div><h3>Results</h3><div>A total of 100 trials were included. These accounted for 64 conditions. Only 37% (37/100) of the trials included PROs. Overall, 17 different types of PROs were identified across the trials. Quality of life (QoL) and health-related quality of life (HRQoL) were the most frequent PROs found in 18% (18/100) and 13% (13/100) of the trials, respectively. A total of 33 PROMs were identified. Of these, 57% (19/33) were HRQoL (89% [17/19]) or QoL (11% [2/19]) measures. Of the HRQoL measures identified, 71% (12/17) were disease specific and 29% (5/17) were generic. Of the non-QoL PROMs, 29% (4/14) were designed to measure pain and 71% (10/14) PROMs focused on other psychological outcomes, including anxiety and depression.</div></div><div><h3>Conclusion</h3><div>Our results show that only 37% of the orphan ATMP trials include patient-reported outcomes and measures. This highlights the urgent need for relevant PROs/PROMs that capture benefits and harms and assimilation of existing PROMs for better comparison between or within conditions. It is essential to include and reflect the patients' experience so that those intended to benefit from the research have the opportunity to influence its direction.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111617"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142752251","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111612
Gui Liberali , Eric Boersma , Hester Lingsma , Jasper Brugts , Diederik Dippel , Jan Tijssen , John Hauser
<div><h3>Objectives</h3><div>To evaluate real-time (day-to-day) adaptation of randomized controlled trials (RCTs) with delayed endpoints – a “forward-looking optimal-experimentation” form of response-adaptive randomization. To identify the implied tradeoffs between lowered mortality, CIs, statistical power, potential arm misidentification, and endpoint rate change during the trial.</div></div><div><h3>Study Design and Setting</h3><div>Using data from RCTs in acute myocardial infarction (30,732 patients in the Global Utilization of Streptokinase and Tissue Plasminogen Activator for Occluded Coronary Arteries, GUSTO-1) and coronary heart disease (12,218 patients in the EURopean trial On reduction of cardiac events with Perindopril in stable coronary Artery disease, EUROPA), we resample treatment-arm assignments and expected endpoints to simulate (1) real-time assignment, (2) forward-looking assignments adapted after observing a fixed number of patients (“blocks”), and (3) a variant that balances RCT and real-time assignments. Blinded real-time adaptive randomizations (RTARs) adjust day-to-day arm assignments by optimizing the tradeoff between assigning the (likely) best treatment and learning about endpoint rates for future assignments.</div></div><div><h3>Results</h3><div>Despite delays in endpoints, real-time assignment quickly learns which arm is superior. In the simulations, by the end of the trials, real-time assignment allocated more patients to the superior arm and fewer patients to the inferior arm(s) resulting in less mortality over the course of the trial. Endpoint rates and odds ratios were well within (resampling) CIs of the RCTs, but with tighter CIs on the superior arm and less-tight CIs on the inferior arm(s) and the odds ratios. The variant and patient-block-based adaptation each provides intermediate levels of benefits and costs. When endpoint rates change within a trial, real-time assignment improves estimation of the end-of-trial superior-arm endpoint rates, but exaggerates differences relative to inferior arms. Unlike most response-adaptive randomizations, real-time assignment automatically adjusts to reduce biases when real changes are larger.</div></div><div><h3>Conclusion</h3><div>Real-time assignment improves patient outcomes within the trial and narrows the CI for the superior arm. Benefits are balanced with wider CIs on inferior arms and odds ratios. Forward-looking variants provide intermediate benefits and costs. In no simulations, was an inferior arm identified as statistically superior.</div></div><div><h3>Plain Language Summary</h3><div>Randomized controlled trials (RCT) are the gold standard in clinical trials — typically half of the patients are assigned to a new drug or procedure and the other half to a placebo (or the current best option). Typically, half of the patients might get an inferior drug or treatment. We explore a method, real-time adaptive randomization (RTAR), that uses information observed up to the t
{"title":"Real-time adaptive randomization of clinical trials","authors":"Gui Liberali , Eric Boersma , Hester Lingsma , Jasper Brugts , Diederik Dippel , Jan Tijssen , John Hauser","doi":"10.1016/j.jclinepi.2024.111612","DOIUrl":"10.1016/j.jclinepi.2024.111612","url":null,"abstract":"<div><h3>Objectives</h3><div>To evaluate real-time (day-to-day) adaptation of randomized controlled trials (RCTs) with delayed endpoints – a “forward-looking optimal-experimentation” form of response-adaptive randomization. To identify the implied tradeoffs between lowered mortality, CIs, statistical power, potential arm misidentification, and endpoint rate change during the trial.</div></div><div><h3>Study Design and Setting</h3><div>Using data from RCTs in acute myocardial infarction (30,732 patients in the Global Utilization of Streptokinase and Tissue Plasminogen Activator for Occluded Coronary Arteries, GUSTO-1) and coronary heart disease (12,218 patients in the EURopean trial On reduction of cardiac events with Perindopril in stable coronary Artery disease, EUROPA), we resample treatment-arm assignments and expected endpoints to simulate (1) real-time assignment, (2) forward-looking assignments adapted after observing a fixed number of patients (“blocks”), and (3) a variant that balances RCT and real-time assignments. Blinded real-time adaptive randomizations (RTARs) adjust day-to-day arm assignments by optimizing the tradeoff between assigning the (likely) best treatment and learning about endpoint rates for future assignments.</div></div><div><h3>Results</h3><div>Despite delays in endpoints, real-time assignment quickly learns which arm is superior. In the simulations, by the end of the trials, real-time assignment allocated more patients to the superior arm and fewer patients to the inferior arm(s) resulting in less mortality over the course of the trial. Endpoint rates and odds ratios were well within (resampling) CIs of the RCTs, but with tighter CIs on the superior arm and less-tight CIs on the inferior arm(s) and the odds ratios. The variant and patient-block-based adaptation each provides intermediate levels of benefits and costs. When endpoint rates change within a trial, real-time assignment improves estimation of the end-of-trial superior-arm endpoint rates, but exaggerates differences relative to inferior arms. Unlike most response-adaptive randomizations, real-time assignment automatically adjusts to reduce biases when real changes are larger.</div></div><div><h3>Conclusion</h3><div>Real-time assignment improves patient outcomes within the trial and narrows the CI for the superior arm. Benefits are balanced with wider CIs on inferior arms and odds ratios. Forward-looking variants provide intermediate benefits and costs. In no simulations, was an inferior arm identified as statistically superior.</div></div><div><h3>Plain Language Summary</h3><div>Randomized controlled trials (RCT) are the gold standard in clinical trials — typically half of the patients are assigned to a new drug or procedure and the other half to a placebo (or the current best option). Typically, half of the patients might get an inferior drug or treatment. We explore a method, real-time adaptive randomization (RTAR), that uses information observed up to the t","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111612"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142669987","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2025.111710
Gustav Nilsonne, Susanne Wieschowski, Nicholas J DeVito, Maia Salholz-Hillel, Love Ahnström, Till Bruckner, Katarzyna Klas, Tarik Suljic, Samruddhi Yerunkar, Natasha Olsson, Carolina Cruz, Karolina Strzebonska, Lars Småbrekke, Mateusz T Wasylewski, Johan Bengtsson, Martin Ringsten, Aminul Schuster, Tomasz Krawczyk, Themistoklis Paraskevas, Eero Raittio, Luca Herczeg, Jan-Ole Hesselberg, Sofia Karlsson, Ronak Borana, Matteo Bruschettini, Shai Mulinari, Karely Lizárraga, Maximilian Siebert, Nicole Hildebrand, Shreya Ramakrishnan, Perrine Janiaud, Emmanuel Zavalis, Delwen Franzen, Kim Boesen, Lars G Hemkens, Florian Naudet, Sofie Possmark, Rebecca M Willén, John P Ioannidis, Daniel Strech, Cathrine Axfors
Objective: To systematically evaluate timely reporting of clinical trial results at medical universities and university hospitals in the Nordic countries.
Study design and setting: In this cross-sectional study, we included trials (regardless of intervention) registered in the EU Clinical Trials Registry and/or ClinicalTrials.gov, completed 2016-2019, and led by a university with medical faculty or university hospital in Denmark, Finland, Iceland, Norway, or Sweden. We identified summary results posted at the trial registries, and conducted systematic manual searches for results publications (e.g., journal articles, preprints). We present proportions with 95% confidence intervals (CI), and medians with interquartile range (IQR).
Protocol: https://osf.io/wua3r RESULTS: Among 2,112 included clinical trials, 1,650 (78.1%, 95%CI 76.3-79.8%) reported any results during our follow-up; 1,097 (51.9%, 95%CI 49.8-54.1%) reported any results within 2 years of the global completion date; and 48 (2.3%, 95%CI 1.7-3.0%) posted summary results in the registry within 1 year. Median time from global completion date to results reporting was 690 days (IQR 1,103). 856/1,681 (50.9%) of ClinicalTrials.gov-registrations were prospective. Denmark contributed approximately half of all trials. Reporting performance varied widely between institutions.
Conclusion: Missing and delayed results reporting of academically led clinical trials is a pervasive problem in the Nordic countries. We relied on trial registry information, which can be incomplete. Institutions, funders, and policy makers need to support trial teams, ensure regulation adherence, and secure trial reporting before results are permanently lost.
{"title":"Results reporting for clinical trials led by medical universities and university hospitals in the Nordic countries was often missing or delayed.","authors":"Gustav Nilsonne, Susanne Wieschowski, Nicholas J DeVito, Maia Salholz-Hillel, Love Ahnström, Till Bruckner, Katarzyna Klas, Tarik Suljic, Samruddhi Yerunkar, Natasha Olsson, Carolina Cruz, Karolina Strzebonska, Lars Småbrekke, Mateusz T Wasylewski, Johan Bengtsson, Martin Ringsten, Aminul Schuster, Tomasz Krawczyk, Themistoklis Paraskevas, Eero Raittio, Luca Herczeg, Jan-Ole Hesselberg, Sofia Karlsson, Ronak Borana, Matteo Bruschettini, Shai Mulinari, Karely Lizárraga, Maximilian Siebert, Nicole Hildebrand, Shreya Ramakrishnan, Perrine Janiaud, Emmanuel Zavalis, Delwen Franzen, Kim Boesen, Lars G Hemkens, Florian Naudet, Sofie Possmark, Rebecca M Willén, John P Ioannidis, Daniel Strech, Cathrine Axfors","doi":"10.1016/j.jclinepi.2025.111710","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111710","url":null,"abstract":"<p><strong>Objective: </strong>To systematically evaluate timely reporting of clinical trial results at medical universities and university hospitals in the Nordic countries.</p><p><strong>Study design and setting: </strong>In this cross-sectional study, we included trials (regardless of intervention) registered in the EU Clinical Trials Registry and/or ClinicalTrials.gov, completed 2016-2019, and led by a university with medical faculty or university hospital in Denmark, Finland, Iceland, Norway, or Sweden. We identified summary results posted at the trial registries, and conducted systematic manual searches for results publications (e.g., journal articles, preprints). We present proportions with 95% confidence intervals (CI), and medians with interquartile range (IQR).</p><p><strong>Protocol: </strong>https://osf.io/wua3r RESULTS: Among 2,112 included clinical trials, 1,650 (78.1%, 95%CI 76.3-79.8%) reported any results during our follow-up; 1,097 (51.9%, 95%CI 49.8-54.1%) reported any results within 2 years of the global completion date; and 48 (2.3%, 95%CI 1.7-3.0%) posted summary results in the registry within 1 year. Median time from global completion date to results reporting was 690 days (IQR 1,103). 856/1,681 (50.9%) of ClinicalTrials.gov-registrations were prospective. Denmark contributed approximately half of all trials. Reporting performance varied widely between institutions.</p><p><strong>Conclusion: </strong>Missing and delayed results reporting of academically led clinical trials is a pervasive problem in the Nordic countries. We relied on trial registry information, which can be incomplete. Institutions, funders, and policy makers need to support trial teams, ensure regulation adherence, and secure trial reporting before results are permanently lost.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111710"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143124071","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2025.111708
Lena Fischer, Leon Vincent Schewe, Fülöp Scheibler, Rahel Wollny, Corinna Schaefer, Torsten Karge, Thomas Langer, Jan Berghold, Ivan D Florez, Andrew Hutchinson, Sheyu Li, Marta Maes-Carballo, Zachary Munn, Lilisbeth Perestelo-Perez, Livia Puljak, Anne Stiggelbout, Dawid Pieper
Objectives: To review the methods used to develop and integrate patient decision aids (PDAs) based on the recommendations of clinical practice guidelines (CPGs).
Study design and setting: We conducted a scoping review covering bibliographic databases (PubMed, Embase; searched until December 2023), grey literature, references, and expert consultations to identify eligible documents. Documents published from 2000 onwards and describing methods related to guideline-based PDA development or linking CPGs and PDAs were included. Two reviewers independently selected and analyzed the documents. Results were synthesized and presented narratively.
Results: Based on 24 included documents, we categorized their methods into four topics. For topic (1), the selection of CPG recommendations for which PDAs are (most) needed, we found a total of 14 selection factors across n=11 documents, with uncertainty/variability in patient preferences and trade-offs between options being the most frequently mentioned. Topic (2) (n=24) covers methods for developing and/or updating guideline-based PDAs, such as forming a multidisciplinary development group, using CPGs and their evidence summaries along with other sources as the evidence base, and using digital solutions for semi-automated development and updating. Topic (3) (n=12) covers methods for PDA quality assessment and/or user testing, such as finalizing and approving the PDAs after a review and feedback process from the CPG group and an iterative user testing process. Topic (4) (n=20) covers methods for linking CPGs and PDAs, often through digital strategies.
Conclusion: We identified heterogeneous methods for developing and integrating PDAs based on CPG recommendations. Empirical testing is required to determine the most useful and practically feasible (combination of) methods. CPG organizations should focus on establishing adequate methods for linking CPG and PDA development to foster shared decision making.
{"title":"Scoping review indicates heterogeneous methods for developing and integrating patient decision aids in the context of clinical practice guidelines.","authors":"Lena Fischer, Leon Vincent Schewe, Fülöp Scheibler, Rahel Wollny, Corinna Schaefer, Torsten Karge, Thomas Langer, Jan Berghold, Ivan D Florez, Andrew Hutchinson, Sheyu Li, Marta Maes-Carballo, Zachary Munn, Lilisbeth Perestelo-Perez, Livia Puljak, Anne Stiggelbout, Dawid Pieper","doi":"10.1016/j.jclinepi.2025.111708","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111708","url":null,"abstract":"<p><strong>Objectives: </strong>To review the methods used to develop and integrate patient decision aids (PDAs) based on the recommendations of clinical practice guidelines (CPGs).</p><p><strong>Study design and setting: </strong>We conducted a scoping review covering bibliographic databases (PubMed, Embase; searched until December 2023), grey literature, references, and expert consultations to identify eligible documents. Documents published from 2000 onwards and describing methods related to guideline-based PDA development or linking CPGs and PDAs were included. Two reviewers independently selected and analyzed the documents. Results were synthesized and presented narratively.</p><p><strong>Results: </strong>Based on 24 included documents, we categorized their methods into four topics. For topic (1), the selection of CPG recommendations for which PDAs are (most) needed, we found a total of 14 selection factors across n=11 documents, with uncertainty/variability in patient preferences and trade-offs between options being the most frequently mentioned. Topic (2) (n=24) covers methods for developing and/or updating guideline-based PDAs, such as forming a multidisciplinary development group, using CPGs and their evidence summaries along with other sources as the evidence base, and using digital solutions for semi-automated development and updating. Topic (3) (n=12) covers methods for PDA quality assessment and/or user testing, such as finalizing and approving the PDAs after a review and feedback process from the CPG group and an iterative user testing process. Topic (4) (n=20) covers methods for linking CPGs and PDAs, often through digital strategies.</p><p><strong>Conclusion: </strong>We identified heterogeneous methods for developing and integrating PDAs based on CPG recommendations. Empirical testing is required to determine the most useful and practically feasible (combination of) methods. CPG organizations should focus on establishing adequate methods for linking CPG and PDA development to foster shared decision making.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111708"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143124073","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111573
Shi Wu Wen, Na Zeng, Daniel Krewski, Mark C. Walker
{"title":"When results from clinical trials are different from observational studies","authors":"Shi Wu Wen, Na Zeng, Daniel Krewski, Mark C. Walker","doi":"10.1016/j.jclinepi.2024.111573","DOIUrl":"10.1016/j.jclinepi.2024.111573","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111573"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142480175","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111634
Rayan Hojeij , Pia Brensing , Michael Nonnemacher , Bernd Kowall , Ursula Felderhoff-Müser , Marcel Dudda , Christian Dohna-Schwake , Andreas Stang , Nora Bruns
Objectives
The performance of injury severity scores (ISSs), used widely to quantify injury severity and predict outcomes, has not been investigated in German pediatric cases. This study aims to identify the most feasible and accurate injury score predictor of mortality in German children with trauma using International Classification of Diseases 10 (ICD-10).
Study Design and Setting
Between 2014 and 2020, a retrospective observational cohort study of hospital admissions cases aged <18 years with injury-related ICD-10 codes, using the German hospital database (GHD), was conducted. The maximum abbreviated injury scale and ISS were calculated using the International Classification of Diseases-Abbreviated Injury Scale (ICD-AIS) map provided by the Association for the Advancement of Automotive Medicine, adjusted to the German modification of the ICD-10 classification. The survival risk ratio was used to calculate the single-worst ICD-derived injury (single International Classification of Disease Injury Severity Score [ICISS]) and a multiplicative ICISS. Logistic regressions were conducted for each of the four above-mentioned scores (predictors) to predict in-hospital mortality (outcome) in the selected trauma population and within four clinically relevant subgroups using discrimination and calibration.
Results
1,720,802 were trauma patients, and ICD-AIS mapping was possible in 1,328,377 cases. Cases with mapping failure (n = 392,425; 22.8%) were younger and had a higher mortality rate were excluded from the performance analysis. ICISS-derived scores had a better discrimination and calibration than ICD-AIS based scores in the overall cohort and all four subgroups (area under the curve [AUC] ranges between 0.985 and 0.998 vs 0.886 and- 0.972, respectively).
Conclusion
Empirically derived measures of injury severity were superior to ICD-AIS mapped scores in the GHD to predict mortality in pediatric trauma patients. Given the high percentage of mapping failure and high mortality among cases with single-coded injury, the single ICISS may be the most suitable measure of injury severity in this group of patients.
{"title":"Performance of ICD-10-based injury severity scores in pediatric trauma patients using the ICD-AIS map and survival rate ratios","authors":"Rayan Hojeij , Pia Brensing , Michael Nonnemacher , Bernd Kowall , Ursula Felderhoff-Müser , Marcel Dudda , Christian Dohna-Schwake , Andreas Stang , Nora Bruns","doi":"10.1016/j.jclinepi.2024.111634","DOIUrl":"10.1016/j.jclinepi.2024.111634","url":null,"abstract":"<div><h3>Objectives</h3><div>The performance of injury severity scores (ISSs), used widely to quantify injury severity and predict outcomes, has not been investigated in German pediatric cases. This study aims to identify the most feasible and accurate injury score predictor of mortality in German children with trauma using International Classification of Diseases 10 (ICD-10).</div></div><div><h3>Study Design and Setting</h3><div>Between 2014 and 2020, a retrospective observational cohort study of hospital admissions cases aged <18 years with injury-related ICD-10 codes, using the German hospital database (GHD), was conducted. The maximum abbreviated injury scale and ISS were calculated using the International Classification of Diseases-Abbreviated Injury Scale (ICD-AIS) map provided by the Association for the Advancement of Automotive Medicine, adjusted to the German modification of the ICD-10 classification. The survival risk ratio was used to calculate the single-worst ICD-derived injury (single International Classification of Disease Injury Severity Score [ICISS]) and a multiplicative ICISS. Logistic regressions were conducted for each of the four above-mentioned scores (predictors) to predict in-hospital mortality (outcome) in the selected trauma population and within four clinically relevant subgroups using discrimination and calibration.</div></div><div><h3>Results</h3><div>1,720,802 were trauma patients, and ICD-AIS mapping was possible in 1,328,377 cases. Cases with mapping failure (<em>n</em> = 392,425; 22.8%) were younger and had a higher mortality rate were excluded from the performance analysis. ICISS-derived scores had a better discrimination and calibration than ICD-AIS based scores in the overall cohort and all four subgroups (area under the curve [AUC] ranges between 0.985 and 0.998 vs 0.886 and- 0.972, respectively).</div></div><div><h3>Conclusion</h3><div>Empirically derived measures of injury severity were superior to ICD-AIS mapped scores in the GHD to predict mortality in pediatric trauma patients. Given the high percentage of mapping failure and high mortality among cases with single-coded injury, the single ICISS may be the most suitable measure of injury severity in this group of patients.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111634"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142796472","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111616
Lesley Uttley , Louise Falzon , Jennifer A. Byrne , Andrea C. Tricco , Marcus R. Munafò , David Moher , Thomas Stoeger , Limbanazo Matandika , Cyril Labbé , Florian Naudet
Background
Research culture is strongly influenced by academic incentives and pressures such as the imperative to publish in academic journals, and can influence the nature and quality of the evidence we produce.
Objective
The purpose of this rapid scoping review is to capture the breadth of differential pressures and contributors to current research culture, drawing together content from empirical research specific to the health and biomedical sciences.
Study Design and Setting
PubMed and Web of Science were searched for empirical studies of influences and impacts on health and biomedical research culture, published between January 2012 and April 2024. Data charting extracted the key findings and relationships in research culture from included papers such as workforce composition; equitable access to research; academic journal trends, incentives, and reproducibility; erroneous research; questionable research practices; biases vested interests; and misconduct. A diverse author network was consulted to ensure content validity of the proposed framework of i) inclusivity, ii) transparency, iii) rigor, and iv) objectivity.
Results
A growing field of studies examining research culture exists ranging from the inclusivity of the scientific workforce, the transparency of the data generated, the rigor of the methods used and the objectivity of the researchers involved. Figurative diagrams are presented to storyboard the links between research culture content and findings.
Conclusion
The wide range of research culture influences in the recent literature indicates the need for coordinated and sustained research culture conversations. Core principles in effective research environments should include inclusive collaboration and diverse research workforces, rigorous methodological approaches, transparency, data sharing, and reflection on scientific objectivity.
{"title":"Research culture influences in health and biomedical research: rapid scoping review and content analysis","authors":"Lesley Uttley , Louise Falzon , Jennifer A. Byrne , Andrea C. Tricco , Marcus R. Munafò , David Moher , Thomas Stoeger , Limbanazo Matandika , Cyril Labbé , Florian Naudet","doi":"10.1016/j.jclinepi.2024.111616","DOIUrl":"10.1016/j.jclinepi.2024.111616","url":null,"abstract":"<div><h3>Background</h3><div>Research culture is strongly influenced by academic incentives and pressures such as the imperative to publish in academic journals, and can influence the nature and quality of the evidence we produce.</div></div><div><h3>Objective</h3><div>The purpose of this rapid scoping review is to capture the breadth of differential pressures and contributors to current research culture, drawing together content from empirical research specific to the health and biomedical sciences.</div></div><div><h3>Study Design and Setting</h3><div>PubMed and Web of Science were searched for empirical studies of influences and impacts on health and biomedical research culture, published between January 2012 and April 2024. Data charting extracted the key findings and relationships in research culture from included papers such as workforce composition; equitable access to research; academic journal trends, incentives, and reproducibility; erroneous research; questionable research practices; biases vested interests; and misconduct. A diverse author network was consulted to ensure content validity of the proposed framework of i) inclusivity, ii) transparency, iii) rigor, and iv) objectivity.</div></div><div><h3>Results</h3><div>A growing field of studies examining research culture exists ranging from the inclusivity of the scientific workforce, the transparency of the data generated, the rigor of the methods used and the objectivity of the researchers involved. Figurative diagrams are presented to storyboard the links between research culture content and findings.</div></div><div><h3>Conclusion</h3><div>The wide range of research culture influences in the recent literature indicates the need for coordinated and sustained research culture conversations. Core principles in effective research environments should include inclusive collaboration and diverse research workforces, rigorous methodological approaches, transparency, data sharing, and reflection on scientific objectivity.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111616"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142711243","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}
Pub Date : 2025-02-01DOI: 10.1016/j.jclinepi.2024.111622
Elizabeth Varghese , Anny Briola , Titouan Kennel , Abby Pooley , Richard A. Parker
Objectives
Stepped wedge cluster randomized trials (SW-CRTs) are an appealing study design because they enable sequential roll out of an intervention across clusters, bringing logistical advantages. This review aimed to evaluate the design rationale, design features, stepped wedge diagram, and analytical approaches of SW-CRTs published in high-impact medical journals from 2020 to 2023, focusing particularly on adherence to key guidelines from the Consolidated Standards of Reporting Trials extension to SW-CRTs.
Study Design and Setting
We conducted searches across PubMed and Cochrane Central Registry of Controlled Trials databases for SW-CRTs published between January 2020 and December 2023 in eight high-impact journals. Eligibility criteria included peer-reviewed publications of randomized SW-CRTs involving human participants, published in English.
Results
Of the 23 SW-CRTs included in the review, 70% had "stepped wedge" explicitly mentioned in their titles. Most studies (96%) included a stepped wedge diagram, but only 65% of these diagrams clearly communicated the duration of each time period. There was considerable variability in design features, including number of sequences (median of 7, range 3–20) and clusters (median of 15, range 9–19). The majority of trials (78%) provided robust justifications for selecting a SW-CRT design, for example, citing practical or logistical constraints. However, 22% of the studies offered less convincing rationales. Generalized linear mixed models were the most frequent analysis method employed.
Conclusion
Our review has highlighted areas for improvement in the presentation of SW-CRTs, particularly in clearly indicating the duration of time periods within diagrams and providing robust justifications for selecting a SW-CRT design.
Plain Language Summary
The stepped wedge cluster randomized trial (SW-CRT) is a type of study design that introduces interventions to different groups at different times. This review examined reports of SW-CRTs published in top medical journals from 2020 to 2023 to see if they followed certain guidelines such as including the word “stepped wedge” in their title. A total of 23 SW-CRTs were included in the review, with 70% mentioning "stepped wedge" in the title. Most (96%) included diagrams, but only 65% showed the duration of each time period clearly. There was variability in design, such as variations in the number of sequences and groups. 78% gave valid reasons for using SW-CRTs, citing practical benefits, whereas 22% did not give convincing reasons. This review suggests that improvements can be made in the presentation of stepped wedge diagrams and in the reporting of SW-CRTs. Researchers should clearly report the length of time periods and provide strong justifications for their design choice.
{"title":"A systematic review of stepped wedge cluster randomized trials in high impact journals: assessing the design, rationale, and analysis","authors":"Elizabeth Varghese , Anny Briola , Titouan Kennel , Abby Pooley , Richard A. Parker","doi":"10.1016/j.jclinepi.2024.111622","DOIUrl":"10.1016/j.jclinepi.2024.111622","url":null,"abstract":"<div><h3>Objectives</h3><div>Stepped wedge cluster randomized trials (SW-CRTs) are an appealing study design because they enable sequential roll out of an intervention across clusters, bringing logistical advantages. This review aimed to evaluate the design rationale, design features, stepped wedge diagram, and analytical approaches of SW-CRTs published in high-impact medical journals from 2020 to 2023, focusing particularly on adherence to key guidelines from the Consolidated Standards of Reporting Trials extension to SW-CRTs.</div></div><div><h3>Study Design and Setting</h3><div>We conducted searches across PubMed and Cochrane Central Registry of Controlled Trials databases for SW-CRTs published between January 2020 and December 2023 in eight high-impact journals. Eligibility criteria included peer-reviewed publications of randomized SW-CRTs involving human participants, published in English.</div></div><div><h3>Results</h3><div>Of the 23 SW-CRTs included in the review, 70% had \"stepped wedge\" explicitly mentioned in their titles. Most studies (96%) included a stepped wedge diagram, but only 65% of these diagrams clearly communicated the duration of each time period. There was considerable variability in design features, including number of sequences (median of 7, range 3–20) and clusters (median of 15, range 9–19). The majority of trials (78%) provided robust justifications for selecting a SW-CRT design, for example, citing practical or logistical constraints. However, 22% of the studies offered less convincing rationales. Generalized linear mixed models were the most frequent analysis method employed.</div></div><div><h3>Conclusion</h3><div>Our review has highlighted areas for improvement in the presentation of SW-CRTs, particularly in clearly indicating the duration of time periods within diagrams and providing robust justifications for selecting a SW-CRT design.</div></div><div><h3>Plain Language Summary</h3><div>The stepped wedge cluster randomized trial (SW-CRT) is a type of study design that introduces interventions to different groups at different times. This review examined reports of SW-CRTs published in top medical journals from 2020 to 2023 to see if they followed certain guidelines such as including the word “stepped wedge” in their title. A total of 23 SW-CRTs were included in the review, with 70% mentioning \"stepped wedge\" in the title. Most (96%) included diagrams, but only 65% showed the duration of each time period clearly. There was variability in design, such as variations in the number of sequences and groups. 78% gave valid reasons for using SW-CRTs, citing practical benefits, whereas 22% did not give convincing reasons. This review suggests that improvements can be made in the presentation of stepped wedge diagrams and in the reporting of SW-CRTs. Researchers should clearly report the length of time periods and provide strong justifications for their design choice.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"178 ","pages":"Article 111622"},"PeriodicalIF":7.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142781874","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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