Journal of Clinical Epidemiology最新文献

{"title":"Definitions of Validity Terms for Use in Discussions of Randomized Controlled Trials.","authors":"Yasaman Yazdani, Monica Taljaard, Merrick Zwarenstein","doi":"10.1016/j.jclinepi.2025.111752","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111752","url":null,"abstract":"<p><p>We review existing definitions and usages of validity terms and propose a single definition for each term for use in communicating inferences from Randomized Controlled trials (RCTs).</p><p><strong>Methods: </strong>Two trialists and a statistician reviewed definitions in various dictionaries and literature to identify confusions and propose unified definition for each term.</p><p><strong>Results: </strong>We propose the following disambiguated and mutually coherent set of definitions for validity terms: TARGET POPULATION: A well-defined population, for whom inferences from an RCT are asserted as valid by the investigators or by other users.</p><p><strong>Internal validity: </strong>An assertion that an inference from an RCT is at low risk of confounding (Distortion of the effects of the intervention by differences in prognostic factors between arms of the trial other than the difference in intervention exposure).</p><p><strong>External validity: </strong>An umbrella term asserting that an internally valid inference from an RCT applies to a target population specified by the investigators or by other users, requiring any of three justifications: representativeness, applicability or extrapolatability.</p><p><strong>Representativeness: </strong>An assertion of external validity of an inference based on the statistical representativeness of the participants, as a random sample of the source (and target) population.</p><p><strong>Applicability: </strong>An assertion of the external validity of an inference based on subjective assessment of the similarity in context and in distribution of known predictors of outcome between the participant sample and the target population.</p><p><strong>Extrapolatability: </strong>An assertion of the external validity of an inference from an RCT to a target population based on a common mechanism of action. BIAS: Bias is the opposite of validity and may be internal, due to confounding from systematic error in design, measurement, and analysis; or external, due to mismatch between the population represented by the RCT participants and their setting, and the target population and its context.</p><p><strong>Conclusion: </strong>With wide uptake, this coherent set of definitions for key terms related to validity could improve understanding and design of RCTs.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111752"},"PeriodicalIF":7.3,"publicationDate":"2025-03-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143588012","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}

{"title":"Response to \"The Inappropriateness of Internal Consistency Testing and Factor Analysis for formative indicators: comment on Felicia et al, 2024\".","authors":"Felicia Jia Ler Ang, Mihir Gandhi, Yin Bun Cheung","doi":"10.1016/j.jclinepi.2025.111750","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111750","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111750"},"PeriodicalIF":7.3,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143574609","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}

{"title":"From introducing Table 0 to forgetting Figure 1: it is time for a reporting guideline for publishing with real-world clinical data. Author's reply.","authors":"Jip W T M de Kok, Bas C T van Bussel, Iwan C C van der Horst, Frank van Rosmalen","doi":"10.1016/j.jclinepi.2024.111656","DOIUrl":"10.1016/j.jclinepi.2024.111656","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111656"},"PeriodicalIF":7.3,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142924001","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}

{"title":"From introducing Table 0 to forgetting Figure 1: it is time for a reporting guideline for publishing with real-world clinical data-Response to de Kok et al.","authors":"Carolina Hincapié-Osorno, Raymond J van Wijk, Douwe F Postma, Jan C Ter Maaten, Hjalmar R Bouma, Jacqueline Koeze","doi":"10.1016/j.jclinepi.2024.111655","DOIUrl":"10.1016/j.jclinepi.2024.111655","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111655"},"PeriodicalIF":7.3,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142924002","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}

{"title":"Rethinking Medication Safety in Pregnancy: How Target Trial Emulation and Real-World Data Bridge the Evidence Gap.","authors":"Yanhong Jessika Hu, Joanne M Said, Jeanie L Y Cheong","doi":"10.1016/j.jclinepi.2025.111747","DOIUrl":"10.1016/j.jclinepi.2025.111747","url":null,"abstract":"<p><strong>Objectives: </strong>The exclusion of pregnant women and infants from many randomized controlled trials (RCTs) has left critical gaps in medication safety, complicating clinical decision-making during these sensitive life stages. This commentary explores target trial emulation using real-world data as a robust alternative for advancing medication safety research when RCTs are not feasible.</p><p><strong>Methods: </strong>Target trial emulation replicates the design principles of RCTs within observational data, accounting for the dynamic nature of medication exposure across gestational stages and adjusting for time-varying confounders. While challenges such as unmeasured confounding, selection bias, and violations of positivity assumptions remain, this method provides crucial insights to address current evidence gaps.</p><p><strong>Results: </strong>Information on medication exposure effects will be obtained, which will inform safer medication guidelines in pregnancy and infancy. Future research integrating artificial intelligence-driven tools, open science practices, and robust data governance frameworks will further strengthen the reliability and impact of target trial emulation. Multinational collaboration and data sharing across diverse sources will accelerate the generation of evidence, ultimately advancing medication safety.</p><p><strong>Conclusion: </strong>Target trial emulation, leveraging real-world data, is a promising alternative when traditional clinical trials are not feasible. This approach promotes safer medication use and improves health outcomes for mothers and infants.</p><p><strong>Plain language summary: </strong>Many clinical trials exclude pregnant women and infants, leaving critical gaps in understanding medication safety during pregnancy and early life. Target trial emulation, which applies clinical trial principles to real-world data, offers a promising alternative when traditional trials are not feasible. This method allows researchers to study how medications affect pregnant women and babies at different stages of pregnancy while also considering factors that change over time. While there are still challenges, like unmeasured factors and bias remain, target trial emulation helps fill these knowledge gaps. Future advancements, including AI, Open Science, enhanced data sharing, and international collaboration, can further enhance this method's ability to improve the safety of medications for mothers and infants worldwide.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111747"},"PeriodicalIF":7.3,"publicationDate":"2025-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143538039","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}

{"title":"The Inappropriateness of Internal Consistency Testing and Factor Analysis for Formative Indicators: Comment on Felicia et al, 2024.","authors":"Xin Meng, Daqiu Wang, Yan Huo, Wenhan Shang, Aiping Wang","doi":"10.1016/j.jclinepi.2025.111748","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111748","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111748"},"PeriodicalIF":7.3,"publicationDate":"2025-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143538043","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}

{"title":"Large language models for conducting systematic reviews: on the rise, but not yet ready for use - a scoping review.","authors":"Judith-Lisa Lieberum, Markus Töws, Maria-Inti Metzendorf, Felix Heilmeyer, Waldemar Siemens, Christian Haverkamp, Daniel Böhringer, Joerg J Meerpohl, Angelika Eisele-Metzger","doi":"10.1016/j.jclinepi.2025.111746","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111746","url":null,"abstract":"<p><strong>Background: </strong>Machine learning (ML) promises versatile help in the creation of systematic reviews (SRs). Recently, further developments in the form of large language models (LLMs) and their application in SR conduct attracted attention.</p><p><strong>Objective: </strong>To provide an overview of LLM applications in SR conduct in health research.</p><p><strong>Study design: </strong>We systematically searched MEDLINE, Web of Science, IEEEXplore, ACM Digital Library, Europe PMC (preprints), Google Scholar, and conducted an additional hand search (last search: 26 February 2024). We included scientific articles in English or German, published from April 2021 onwards, building upon the results of a mapping review that has not yet identified LLM applications to support SRs. Two reviewers independently screened studies for eligibility; after piloting, one reviewer extracted data, checked by another.</p><p><strong>Results: </strong>Our database search yielded 8054 hits, and we identified 33 articles from our hand search. We finally included 37 articles on LLM support. LLM approaches covered 10 of 13 defined SR steps, most frequently literature search (n=15, 41%), study selection (n=14, 38%), and data extraction (n=11, 30%). The mostly recurring LLM was GPT (n=33, 89%). Validation studies were predominant (n=21, 57%). In half of the studies, authors evaluated LLM use as promising (n=20, 54%), one quarter as neutral (n=9, 24%) and one fifth as non-promising (n=8, 22%).</p><p><strong>Conclusions: </strong>Although LLMs show promise in supporting SR creation, fully established or validated applications are often lacking. The rapid increase in research on LLMs for evidence synthesis production highlights their growing relevance.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111746"},"PeriodicalIF":7.3,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143532184","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}

{"title":"Editorial, April 2025","authors":"David Tovey,&nbsp;Andrea C. Tricco","doi":"10.1016/j.jclinepi.2025.111749","DOIUrl":"10.1016/j.jclinepi.2025.111749","url":null,"abstract":"","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"180 ","pages":"Article 111749"},"PeriodicalIF":7.3,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143609145","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}

{"title":"Two-decade health-related quality of life and performance on physical function tests in midaged women: findings from a prospective cohort study","authors":"Leticia W. Ribeiro,&nbsp;Gregore I. Mielke,&nbsp;Jenny Doust,&nbsp;Gita D. Mishra","doi":"10.1016/j.jclinepi.2025.111730","DOIUrl":"10.1016/j.jclinepi.2025.111730","url":null,"abstract":"<div><h3>Objectives</h3><div>Health-related quality of life (HRQoL) is more commonly measured in younger populations than objective physical function tests. However, associations between HRQoL and the performance on physical function tests are unclear. This study investigates the association between HRQoL measures across adulthood and performance on physical function tests in midaged women.</div></div><div><h3>Methods</h3><div>Data were derived from 499 women born during 1973–1978 from the Menarche-to-PreMenopause Study, a substudy of the Australian Longitudinal Study on Women's Health. HRQoL was assessed every 3 years from ages 18–23 years to 40–45 years using the eight Short Form Health Survey subscales. Generalized estimating equation models examined the associations between HRQoL over 22 years and three performance tests at a mean age 44.6 years: handgrip strength (kg), chair rise (sec), and standing balance (sec). Worse performance was defined by the lowest tertile of the sample.</div></div><div><h3>Results</h3><div>Several HRQoL subscales showed longitudinal associations with performance. Repeatedly lower scores on nearly all subscales were linked to worse chair rise performance, except for social functioning and mental health. Bodily pain was associated with all three tests; women reporting more pain across the 22-year follow-up showed 50% higher odds of worse chair rise and 30% higher odds of both worse handgrip strength and balance. Women with lower physical functioning scores had higher odds of worse grip (odds ratio 1.4, 95% CI 1.1–1.9) and worse chair rise performances (odds ratio 1.4, 95% CI 1.4–2.6).</div></div><div><h3>Conclusion</h3><div>This study showed poorer HRQoL from early-to-mid adulthood was associated with worse physical performance in midaged Australian women, particularly in the chair rise test.</div></div>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":"181 ","pages":"Article 111730"},"PeriodicalIF":7.3,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143525106","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}

{"title":"Performance of the revised WHO cardiovascular disease risk prediction models for the Middle East and North Africa: a validation study in the Tehran Lipid and Glucose Study.","authors":"Mahin Nomali, Mehdi Yaseri, Saharnaz Nedjat, Fereidoun Azizi, Mohammad Ali Mansournia, Hossein Navid, Goodarz Danaei, Mark Woodward, Noushin Fahimfar, Ewout Steyerberg, Davood Khalili","doi":"10.1016/j.jclinepi.2025.111736","DOIUrl":"https://doi.org/10.1016/j.jclinepi.2025.111736","url":null,"abstract":"<p><strong>Objective: </strong>We aimed to evaluate the performance of the revised WHO models in predicting the 10-year risk of cardiovascular disease (CVD) in Iran, as part of the Middle East and North Africa (MENA) region.</p><p><strong>Study design and setting: </strong>We analyzed data from the Tehran Lipid and Glucose Study (TLGS), including 5162 participants (2241 men) aged 40-80 years without CVD at baseline (the 3^rd examination, 2006-2008), for the occurrence of CVD (myocardial infarction, coronary heart disease death, and stroke). We assessed the statistical performance of original and regionally recalibrated models-both laboratory- and non-laboratory-based- using discrimination (C-statistic) calibration (calibration plot and observed-to-expected[O:E] ratio), and clinical performance applying net benefit (NB), a measure of true positives penalized for a weight of false positives, a decimal value representing the expected proportion of true positive outcomes among total population.</p><p><strong>Results: </strong>During the 10-year follow-up, 307 CVD events occurred. The cumulative incidence of CVD was 9.0% (95% CI: 8.0-10.0%) in men and 4.0% (3.0-5.0%) in women. For the laboratory-based model, the C-statistic was 0.72 (0.68-0.75) in men and 0.83 (0.80-0.86) in women; for the non-laboratory-based model, it was 0.70 (0.66-0.73) and 0.82 (0.79-0.86), respectively. The lab model underpredicted the risk (O:E=1.20[1.00-1.33] for men and 1.40 [1.13-1.60] for women). At the risk threshold of 10%, NB for the lab model was 0.03 (0.02-0.04) for men and 0.01 (0.004-0.01) for women; these values became zero or negative for thresholds over 20%. Regionally recalibrated models overestimated the risk (O:E <1) and showed lower NB.</p><p><strong>Conclusion: </strong>The loss of specificity was not sufficiently offset by the increase in sensitivity provided by the regionally recalibrated models compared to the original models.</p>","PeriodicalId":51079,"journal":{"name":"Journal of Clinical Epidemiology","volume":" ","pages":"111736"},"PeriodicalIF":7.3,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143525089","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}