Lanwei Guo, Jiani Yuan, Lin Cai, Chenxin Zhu, Yan Zheng, Haiyan Yang, Yanyan Liu
{"title":"Disease Burden of Gastrointestinal Tumors in China From 1990 to 2021, an Analysis for the Global Burden of Disease Study 2021","authors":"Lanwei Guo, Jiani Yuan, Lin Cai, Chenxin Zhu, Yan Zheng, Haiyan Yang, Yanyan Liu","doi":"10.1111/jebm.70072","DOIUrl":"10.1111/jebm.70072","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>China faces a significant burden of gastrointestinal tumors driven by socioeconomic, environmental, and lifestyle factors. Using GBD2021 data, this study analyses epidemiological trends and disease burden for six major gastrointestinal tumor cancers (esophagus, gastric, colorectum, liver, pancreas, gallbladder and biliary tract) in China (1990–2021).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The GBD 2021 was used to extract the incidence, mortality, and disability-adjusted life years (DALYs) data of gastrointestinal tumors in China. Age-standardized rates (ASRs) and 95% uncertainty intervals (UIs) were calculated. Temporal trends were assessed by joinpoint regression analysis, and average annual percent change (AAPC) and annual percentage change (APC) were calculated and analyzed stratified by gender and age group.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In 2021, China recorded 1.96 million new gastrointestinal cancer cases, with 1.35 million deaths and 33.07 million DALYs. Gastric cancer led in mortality, and colorectal cancer demonstrated the most rapid incidence growth (AAPC = 1.68). Significant reductions were observed in gastric cancer age-standardized mortality rates (ASMR) (AAPC = –2.44) and esophageal cancer age-standardized disability-adjusted life year rates (ASDR) (AAPC = –2.31). Gender disparities were particularly pronounced in esophageal cancer, with the male-to-female mortality ratio (M/F) escalating from 2.50 (1990) to 4.12 (2021). The age group with the highest mortality burden was 70–74, while the age group with the most significant loss of DALYs was 65–69.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>China has significantly reduced gastrointestinal cancer burden, but gender and age disparities persist, necessitating targeted interventions. Future efforts should focus on tertiary prevention for high-risk groups, especially males and the elderly, while enhancing molecular subtyping and regional data stratification for precision cancer control.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jebm.70072","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145092174","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}
Huai Heng Loh, Siow Phing Tay, Ai Jiun Koa, Mei Ching Yong, Asri Said, Chee Shee Chai, Natasya Marliana Abdul Malik, Anselm Ting Su, Bonnie Bao Chee Tang, Florence Hui Sieng Tan, Norlela Sukor
{"title":"Cardiac Autonomic Dysfunction in Obstructive Sleep Apnea: The Hidden Role of Vitamin D Deficiency","authors":"Huai Heng Loh, Siow Phing Tay, Ai Jiun Koa, Mei Ching Yong, Asri Said, Chee Shee Chai, Natasya Marliana Abdul Malik, Anselm Ting Su, Bonnie Bao Chee Tang, Florence Hui Sieng Tan, Norlela Sukor","doi":"10.1111/jebm.70071","DOIUrl":"10.1111/jebm.70071","url":null,"abstract":"<p>Obstructive sleep apnea (OSA) is a common sleep-breathing disorder, affecting an estimated 9–38% of adults worldwide, with prevalence increasing in parallel with rising obesity rates and aging population [<span>1</span>]. While men are still more likely to be diagnosed with OSA, this condition is becoming more common in women [<span>2</span>]. Coronary artery disease (CAD), a common complication of OSA, is driven by a combination of intermittent hypoxia, metabolic dysfunction, and systemic inflammation, which together accelerate atherosclerosis [<span>3</span>]. This underscores the importance of identifying modifiable factors to mitigate cardiovascular risks in these patients. A key mechanism linking OSA to heightened cardiovascular risks in CAD is cardiac autonomic dysfunction, characterized by increased sympathetic activity and reduced parasympathetic tone. These alterations can be assessed through heart rate variability (HRV), a well-established predictor of adverse cardiovascular outcomes.</p><p>Patients with OSA often exhibit lower serum 25-hydoxyvitamin D [25(OH)D] levels, with a high prevalence of vitamin D deficiency (VDD) compared to those without OSA [<span>4</span>]. OSA and VDD share overlapping risk factors, with interplay between obesity, hypoxia, reduced vitamin D absorption, inflammation, and effects on upper airway muscle function [<span>4</span>]. VDD is linked to higher cardiovascular risk and mortality and may be associated with poorer HRV [<span>5, 6</span>], although evidence remains limited.</p><p>This study aimed to investigate the role of vitamin D in cardiovascular autonomic function among OSA patients, addressing a critical gap in understanding the interplay between VDD, OSA, and cardiovascular risks. Given the contribution of systemic inflammation and endothelial dysfunction to cardiovascular risks in OSA, we also assessed high-sensitivity C-reactive protein (hsCRP) and brachial artery flow-mediated dilatation (BAFMD) as secondary endpoints, providing complementary insights alongside cardiac autonomic function. The study was conducted in accordance with the principles outlined in the Declaration of Helsinki. Ethical approval was obtained from the Medical Research Ethical Committee of Malaysia (NMRR-21-1472-60812) and UNIMAS Medical Ethics Committee (UNIMAS/TNC(PI)/09-65/01). All patients who fulfilled study criteria were recruited after informed consent obtained. This study was part of a broader research effort, <i>Cardiovascular Impacts of RAAS and Vitamin D in Obstructive Sleep Apnea (CARD-OSA)</i>, which sought to explore the complex roles of renin-angiotensin-aldosterone system and vitamin D in OSA and their broader implications for patient health.</p><p>Designed as a cross-sectional study, it was conducted at a tertiary sleep center from June 2022 to May 2024. Briefly, patients aged ≥18 years with a body mass index (BMI) ≥27.5 kg/m<sup>2</sup>, and confirmed OSA were recruited. The BMI cut-off used, following ","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jebm.70071","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145080912","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":"Evaluating Evidence With AI: ChatGPT's Performance on the PEDro Scale in Physical Therapy Research","authors":"Jie Hao, Zixuan Yao, Andréas Remis, Xin Yu","doi":"10.1111/jebm.70074","DOIUrl":"10.1111/jebm.70074","url":null,"abstract":"","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145080958","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":"Chinese Guideline for the Rehabilitation Treatment of Osteoporosis (2024 Edition)","authors":"Haiming Wang, Yi Li, Chengqi He","doi":"10.1111/jebm.70056","DOIUrl":"10.1111/jebm.70056","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <p>Osteoporosis (OP) is one of the most serious health problems, causing a huge economic burden to patients, families, and society. OP rehabilitation treatment plays an important role in pain-relieving, reducing the risk of fracture, improving the ability of daily activities, and promoting the healing of OP fractures, and has been increasingly valued and recommended by domestic and foreign guidelines. This guideline was updated based on the Chinese Expert Consensus on Rehabilitation Intervention for Primary OP 2019 editions and was initiated by the Chinese Society of Physical Medicine and rehabilitation and the West China Hospital of Sichuan University. This guideline development was guided by domestic and international guideline development methods and principles and selected through clinical issue screening and deconstruction and two rounds of Delphi questionnaire consultation. The International Classification of Functioning, Disability and Health (ICF) was used as the theoretical framework, and the Grading of Recommendations Assessment, Development and Evaluation (GRADE) was used based on the best available evidence. The guideline was developed based on the best available evidence, used the GRADE method to grade the quality of evidence and recommendations, and reported according to the Reporting Items for Practice Guidelines in Healthcare. Taking into account patients' preferences and values and the needs of Chinese clinical practice, it puts forward 22 recommendations covering seven aspects (rehabilitation assessment, therapeutic modalities, occupational therapy, assistive devices, cognitive behavior and psychological therapy, traditional Chinese medicine therapy, and health education) to systematically standardize OP rehabilitation.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145054173","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}
Weilong Zhao, Danni Xia, Ziying Ye, Honghao Lai, Mingyao Sun, Jiajie Huang, Jiayi Liu, Jianing Liu, Long Ge
{"title":"From Evidence to Recommendations With Large Language Models: A Feasibility Study","authors":"Weilong Zhao, Danni Xia, Ziying Ye, Honghao Lai, Mingyao Sun, Jiajie Huang, Jiayi Liu, Jianing Liu, Long Ge","doi":"10.1111/jebm.70067","DOIUrl":"10.1111/jebm.70067","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Formulating evidene-based recommendations for practice guidelines is a complex process that requires substantial expertise. Artificial intelligence (AI) is promising in accelerating the guideline development process. This study evaluates the feasibility of leveraging five large language models (LLMs)—ChatGPT-3.5, Claude-3 sonnet, Bard, ChatGLM-4, Kimi chat—to generate recommendations based on structured evidence, assesses their concordance, and explores the potential for AI.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The general and specific prompts were drafted and validated. We searched PubMed to include evidence-based guidelines related to health and lifestyle. We randomly selected one recommendation from every included guideline as the sample and extracted the evidence base supporting the selected recommendations. The prompts and evidence were fed into five LLMs to generate structured recommendations.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>ChatGPT-3.5 demonstrated the highest proficiency in comprehensively extracting and synthesizing evidence to formulate novel insights. Bard consistently adhered to existing guideline principles, aligning its algorithm with these tenets. Claude generated fewer topical recommendations, focusing instead on evidence analysis and mitigating irrelevant information. ChatGLM-4 exhibited a balanced approach, combining evidence extraction with adherence to guideline principles. Kimi showed potential in generating concise and targeted recommendations. Among the six generated recommendations, average consistency ranged from 50% to 91.7%.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The findings of this study suggest that LLMs hold immense potential in accelerating the formulation of evidence-based recommendations. LLMs can rapidly and comprehensively extract and synthesize relevant information from structured evidence, generating recommendations that align with the available evidence.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040250","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":"Developing and Externally Validating the Multivariable Prediction Model for White-Coat Hypertension","authors":"Shali Hao, Xiaomei Zhang, Lingxiao Li, Libin Mo, Yangguang Liu, Jiahuan Li, Wenli Wang, Jiandi Wu, Yuli Huang","doi":"10.1111/jebm.70064","DOIUrl":"10.1111/jebm.70064","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>The white-coat hypertension (WCH) detection by monitoring the out-of-office blood pressure (BP) consumes resources and time. This study aimed at developing the prediction model based on patients’ characteristics obtained from clinical data.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Individuals who participated in two large hospitals health check-up examination were screened. Participants with twice readings of elevated office blood pressure in different visits, while no history of hypertension were included. Combination with home blood pressure monitoring, participants were defined as having WCH or sustained hypertension (SH), respectively. Independent predictors were found by employing multivariate logistic regression on training set. A nomogram was built using independent predictors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In total, 383 outpatients with elevated office blood pressure were enrolled. Two hundred and thirty-three of them from one hospital were included for the development of the prediction model (training sets), and 150 patients from another independent study site were included for external validation (external validation sets). We identified six predictors including office systolic blood pressure, body mass index, sex, total cholesterol, homocysteine, and heart rate being linked to WCH diagnosis. Area under receiver operating characteristic curve (AUC) for the model was 0.792 and 0.692 regarding training and external validation sets, respectively. The calibration curve and decision curve analyses further demonstrated that the model had good performance for distinguishing WCH from SH.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>This prediction model can help clinicians to identify WCH individuals from those with SH, providing an effective tool for guiding personalized recommendations of abnormal blood pressure management.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145029802","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":"Burden of Myelodysplastic Syndromes: A Literature Review of Epidemiological and Humanistic Aspects","authors":"Shitong Xie, Jiajun Yan, Preston Tse, Brittany Humphries, Feng Xie","doi":"10.1111/jebm.70065","DOIUrl":"10.1111/jebm.70065","url":null,"abstract":"<p>Myelodysplastic syndromes (MDS) are a group of clonal neoplastic bone marrow disorders characterized by ineffective hematopoiesis and cytopenia [<span>1</span>]. Given the increasing incidence of MDS in aging populations and its chronic disease burden, understanding its epidemiological trends and humanistic burden is crucial. Despite several reviews that have examined epidemiological or humanistic burden of MD, existing literature is often limited in scope, focusing on specific subgroups, for example, higher-risk MDS, or single geographic regions. Furthermore, heterogeneous study methodologies have led to inconsistent estimates of MDS incidence, prevalence, and mortality, complicating comparisons across populations. The aim of this study was to conduct a scoping literature review to summarize both the epidemiological and humanistic burden of MDS.</p><p>A systematic search was conducted to understand the epidemiological, humanistic, and economic burden of MDS. The search was undertaken from inception to July 13, 2021 in Embase and Medline. A detailed search strategy is presented in Table S1. Bibliographic searches of systematic reviews identified during the screening were conducted to validate the search strategy and identify any additional publications that were not retrieved through the original search. Studies were included if they (1) included patients with MDS and assessed epidemiologic (i.e., prevalence, incidence or mortality) or humanistic (i.e., patient-reported outcome using either a generic or disease-specific instrument) outcomes; (2) had a sample of more than 100, and (3) were published in English. Systematic reviews were excluded, but if relevant, their reference lists were cross-referenced. Study selection involved two phases: an initial screening based on title and abstract followed by a full-text review. Both phases of screening were performed by two reviewers independently and in duplicate. Any discrepancies were solved by discussion between the two reviewers or consulting a third reviewer when necessary. A study mapping process was conducted to match publications reporting on the same study. For included studies, information on study and patient characteristics, and outcomes of interest were extracted by two reviewers independently using a standardized data extraction form. Discrepancies were reconciled by discussion between the two reviewers or in consultation with a third reviewer when necessary.</p><p>The study employed descriptive analyses to summarize the epidemiological and HRQoL outcomes in patients with MDS. The descriptive statistics included age-standardized incidence rate (ASIR), prevalence, and survival rates. Data were organized in Excel and visualized with R using distribution plots and trend graphs.</p><p>A total of 63 papers (56 studies) reported on the epidemiological burden of MDS and 57 papers (46 studies) on the HRQoL burden of MDS. The selection process is summarized in Figure S1. Details on studies repor","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-09-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jebm.70065","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145029841","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}
Luis Furuya-Kanamori, Xanthoula Rousou, Polychronis Kostoulas, Suhail A. R. Doi
{"title":"Examining and Interpreting Doi Plot Asymmetry in Meta-Analyses of Randomized Controlled Trials","authors":"Luis Furuya-Kanamori, Xanthoula Rousou, Polychronis Kostoulas, Suhail A. R. Doi","doi":"10.1111/jebm.70063","DOIUrl":"10.1111/jebm.70063","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <p>Systematic reviews and meta-analyses are considered the highest level of evidence, but their reliability can be undermined by publication bias. Traditional methods for assessing publication bias, such as funnel plots and <i>p</i>-value-based tests (e.g., Egger test), have notable limitations, including reliance on subjective interpretation and dependence on the number of studies included in a meta-analysis (<i>k</i>). The Doi plot and LFK index offer promising alternatives, providing improved visualization and quantification of plot asymmetry. This study revisits the application of the Doi plot and LFK index for detecting publication bias, addresses recent criticisms, and evaluates their performance compared to <i>p</i>-value-based methods using simulation study. Simulations included scenarios with varying study numbers (<i>k</i> = 5, 10, 20, 50), study sample sizes (small, large), and simulated bias level (<i>ρ</i> = 0, –0.3, –0.5, –0.9) generated using the Copas selection model. Diagnostic performance metrics (i.e., sensitivity and specificity) were estimated and compared for the LFK index and Egger test. The LFK index exhibited consistent higher sensitivity across varying <i>k</i> and simulated bias levels. In contrast, the Egger test was highly dependent on <i>k</i>, with sensitivity declining sharply in small meta-analyses (<i>k</i> < 20). Specificity of the LFK index adjusted with random error, while Egger test specificity remained fixed at ∼90%. The Doi plot and LFK index effectively address the limitations of traditional methods, offering robust <i>k</i>-independent performance and more reliable detection of publication bias. These findings support a transition to the Doi plot and LFK index for publication bias assessment in meta-analyses.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1111/jebm.70063","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957108","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}
Yanjiao Shen, Parpia Sameer, Xin Xia, Yuqing Zhang, Jinhui Ma, Qingyang Shi, Qiukui Hao, Xianlin Gu, Wenbo He, Yamin Chen, Na Zhang, Le Wang, Yating Zeng, Xiaoyi Su, Qiang Zong, Qiao Zhi, Sitong Liu, Xinyao Wang, Xinyu Zou, Ying He, Qiong Guo, Borong Wang, Liang Du, Zhengchi Li, Jin Huang, Guyatt Gordon
{"title":"A Systematic Survey of the Optimal Strategy for Dealing With Missing Binary Outcomes in Simulation Studies of Randomized Controlled Trials","authors":"Yanjiao Shen, Parpia Sameer, Xin Xia, Yuqing Zhang, Jinhui Ma, Qingyang Shi, Qiukui Hao, Xianlin Gu, Wenbo He, Yamin Chen, Na Zhang, Le Wang, Yating Zeng, Xiaoyi Su, Qiang Zong, Qiao Zhi, Sitong Liu, Xinyao Wang, Xinyu Zou, Ying He, Qiong Guo, Borong Wang, Liang Du, Zhengchi Li, Jin Huang, Guyatt Gordon","doi":"10.1111/jebm.70058","DOIUrl":"10.1111/jebm.70058","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Aim</h3>\u0000 \u0000 <p>To summarize the optimal strategies for dealing with missing binary outcome data (MBOD) in randomized controlled trials (RCTs) as informed by simulation studies, and to summarize the quality of reporting in these studies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>To identify simulation studies comparing at least two strategies to deal with MBOD and evaluating their performance (bias, coverage and power), we searched MEDLINE, EMBASE, Cochrane Central Register of Controlled Trials via Ovid, Web of Science, and JSTOR from their inception up to December 20, 2023. We evaluated reporting quality using established criteria for simulation studies in medical statistics. We summarized data using descriptive statistics and a narrative synthesis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Our search identified 29,460 citations, of which five proved eligible. Multiple imputation (MI), investigated in five studies, showed consistently good performance in all domains tested for missing completely at random (MCAR) and missing at random (MAR) but with important limitations in missing not at random (MNAR). Complete case analysis (CCA), investigated in four studies of which three addressed model-based CCA, performed well in bias and coverage under MAR and MCAR, but less well for MNAR. One study reported that non-model-based CCA performed poorly with respect to bias under MAR. Non-model-based single imputation, investigated in two studies, showed consistently poor performance across all domains tested for MAR, MCAR and MNAR. One study reported that model-based single imputation performed well with respect to bias under MAR. Regarding reporting quality, all studies reported the aims, dependence of simulated data sets, scenarios and statistical methods evaluated, number of simulations performed, justification of data generation and criteria used to evaluate the simulation performance. None of the studies reported the starting seeds, random number generators and failures occurring during simulation.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Simulation studies address methods to deal with MBOD in RCTs, provided evidence that the MI approach is superior with respect to bias and coverage compared with CCA. Non-model-based single imputation generally performed poorly.</p>\u0000 </section>\u0000 </div>","PeriodicalId":16090,"journal":{"name":"Journal of Evidence‐Based Medicine","volume":"18 3","pages":""},"PeriodicalIF":3.5,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144957088","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}