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Case studies: a guide for researchers, educators, and implementers. 案例研究:研究人员、教育工作者和实现者的指南。
IF 1 Pub Date : 2025-09-21 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2025-001623
Trisha Greenhalgh

Case study is a widely used but poorly understood research method, conducted differently in different disciplines. This paper explores philosophical, theoretical and methodological issues in case study research and outlines how to conduct one. It offers preliminary guidance for policy makers on how to select and use case studies for learning and decision making. In social science research, a case study is a detailed, contextualised account of a clearly delineated, real world phenomenon, prepared prospectively using mostly qualitative methods. Social science case studies can be of various kinds (eg, theoretical or naturalistic, single or multiple, typical or extreme). A public health case study is a historical account of a health threat and how it was managed. An implementation science case study evaluates the implementation of an intervention (usually retrospectively), combining quantitative assessment against predefined objectives with a narrative of how the project unfolded. Educational case studies present real world topics as stories illustrated by data and prompt students to discuss these from different angles. Impact case studies summarise the societal impact of a research programme. Many accounts described as case studies are overly brief and superficial. The paper concludes with a call to improve the quality and consistency (and hence the usefulness) of case studies.

案例研究是一种广泛使用但鲜为人知的研究方法,在不同的学科中进行不同的研究。本文探讨了案例研究中的哲学、理论和方法问题,并概述了如何进行案例研究。它为决策者提供了关于如何选择和使用案例研究进行学习和决策的初步指导。在社会科学研究中,案例研究是对一个清晰描述的现实世界现象的详细的、背景化的描述,主要使用定性方法进行前瞻性准备。社会科学案例研究可以是多种多样的(例如,理论的或自然的,单一的或多重的,典型的或极端的)。公共卫生案例研究是对健康威胁及其管理方式的历史描述。实施科学案例研究评估干预措施的实施(通常是回顾性的),将针对预定义目标的定量评估与项目如何展开的叙述相结合。教育案例研究以数据说明的故事形式呈现现实世界的主题,并促使学生从不同角度讨论这些主题。影响案例研究总结了研究项目的社会影响。许多被描述为案例研究的描述过于简短和肤浅。论文最后呼吁提高案例研究的质量和一致性(以及有用性)。
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引用次数: 0
The critical importance of design decisions in case studies. 案例研究中设计决策的关键重要性。
IF 1 Pub Date : 2025-09-21 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2025-001986
Penelope Hawe
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引用次数: 0
Evaluation of area based socioeconomic inequalities and neonatal mortality rates in France: national population based study. 评价法国基于地区的社会经济不平等和新生儿死亡率:全国基于人口的研究。
IF 1 Pub Date : 2025-09-16 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2024-000954
Victor Sartorius, Héloïse Torchin, Luc Gaulard, Marianne Philibert, Victoria Butler, Monica Saucedo, Catherine Deneux-Tharaux, Jeanne Fresson, Jennifer Zeitlin

Abstract:

Objective: To investigate the magnitude and evolution of inequalities in neonatal mortality rates by using area based socioeconomic indices in France.

Design: National population based study.

Setting: For 2015-20, data from the French National Health Data System (Système National des Données de Santé, SNDS). For 2001-08, neonatal death certificates and aggregate vital statistics data by municipality of residence.

Participants: Live births with a gestational age ≥22 completed weeks to a mother residing in metropolitan France, 2015-20 (4 293 403 live births and 10 869 neonatal deaths), compared with a 2001-08 study (6 202 918 live births and 14 851 neonatal deaths).

Main outcome measures: Differences in neonatal mortality rate (death before day 28 of life) according to the socioeconomic characteristics of the mother's municipality of residence. Comparison with data from a 2001-08 study to assess changes in socioeconomic inequalities and their contribution to the increase in neonatal mortality rate.

Results: The neonatal mortality rate was 2.53 per 1000 live births in 2015-20. Five indicators, previously associated with perinatal mortality, were combined into a perinatal French deprivation index (P-FDep) for the main analysis. P-FDep was categorised into five equal groups (deprivation groups 1-5) for comparison with other research and into 10 equal groups (deprivation groups 1-10) for more granular analyses, with group 1 being the least and group 5 (or group 10) the most deprived group. The rate in the most deprived compared with the least deprived group for P-FDep was 1.71 (95% confidence interval 1.60 to 1.83) times higher, based on the analysis of deprivation groups 1-5. A mortality gradient existed across the groups, translating into 2496 excess deaths (23.3%) when the rate in the least deprived group was applied to all areas. The gradient was more marked when deprivation groups 1-10 were used (relative risk 1.88, 95% CI 1.71 to 2.07 for the highest to the lowest deprived group). Compared with 2001-08 (neonatal mortality rate 2.39 per 1000), the rate remained constant in the least deprived areas, but worsened in the most deprived areas (+10.1% and +11.7% for groups 4 and 5, respectively), increasing the relative risks between the highest and lowest groups, which were 1.54 (95% CI 1.46 to 1.62) for deprivation groups 1-5 and 1.67 (1.55 to 1.79) for deprivation groups 1-10, in 2001-08.

Conclusions: In this study, the socioeconomic level of the mother's place of residence was strongly associated with the neonatal mortality rate. The data showed that inequalities have widened, contributing to the increase in the neonatal mortality rate.

摘要:目的:利用基于区域的社会经济指数,调查法国新生儿死亡率不平等的程度和演变。设计:基于全国人口的研究。背景:2015- 2020年,数据来自法国国家健康数据系统(system National des donnsam, SNDS)。2001- 2008年,按居住城市分列的新生儿死亡证明和汇总生命统计数据。参与者:2015- 2020年居住在法国大都市的母亲的胎龄≥22周的活产(4 293 403例活产和10 869例新生儿死亡),与2001-08年的研究(6 202 918例活产和14 851例新生儿死亡)相比。主要结果测量:根据母亲居住城市的社会经济特征,新生儿死亡率(出生后28天前死亡)的差异。与2001-08年评估社会经济不平等变化及其对新生儿死亡率上升的影响的研究数据的比较。结果:2015- 2020年新生儿死亡率为2.53 / 1000活产。以前与围产期死亡率相关的五个指标被合并成围产期法国剥夺指数(P-FDep)进行主要分析。P-FDep被分成5个相等的组(剥夺组1-5)与其他研究进行比较,并被分成10个相等的组(剥夺组1-10)进行更细致的分析,第1组是最少的,第5组(或第10组)是最被剥夺的组。根据对剥夺组1-5的分析,最剥夺组P-FDep的发生率比最剥夺组高1.71倍(95%可信区间1.60 ~ 1.83)。各群体之间存在死亡率梯度,当将最贫困群体的死亡率应用于所有地区时,死亡人数增加了2496人(23.3%)。当使用剥夺组1-10时,梯度更为明显(剥夺最高至最低组的相对风险为1.88,95% CI为1.71至2.07)。与2001-08年(新生儿死亡率2.39 / 1000)相比,最贫困地区的新生儿死亡率保持不变,但最贫困地区的新生儿死亡率有所恶化(第4组和第5组分别为+10.1%和+11.7%),最高和最低群体之间的相对风险增加,2001-08年,第1-5组的相对风险为1.54(95%可信区间1.46至1.62),第1-10组的相对风险为1.67(95%可信区间1.55至1.79)。结论:在本研究中,母亲居住地的社会经济水平与新生儿死亡率密切相关。数据显示,不平等现象扩大,导致新生儿死亡率上升。
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引用次数: 0
Regression adjustment for causal inference. 因果推理的回归调整。
IF 1 Pub Date : 2025-09-16 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2023-000816
Frederick Ho
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引用次数: 0
Effectiveness of different de-implementation strategies in primary care: systematic review and meta-analysis. 初级保健中不同去实施策略的有效性:系统回顾和荟萃分析。
IF 1 Pub Date : 2025-09-09 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2025-001343
Aleksi Raudasoja, Sameer Parpia, Jussi M J Mustonen, Robin Vernooij, Petra Falkenbach, Yoshitaka Aoki, Anton Barchuk, Marco H Blanker, Rufus Cartwright, Kathryn Crowder, Herney Andres Garcia-Perdomo, Rachel Gutschon, Alex L E Halme, Tuomas P Kilpeläinen, Ilari Kuitunen, Tiina Lamberg, Eddy Lang, Jenifer Matos, Olli P O Nevalainen, Niko K Nordlund, Negar Pourjamal, Eero Raittio, Patrick O Richard, Philippe D Violette, Jorma T Komulainen, Raija Sipilä, Kari A O Tikkinen

Objective: To evaluate the effectiveness of various de-implementation interventions in primary care, targeting care (treatments or tests) that provides no or limited value for patients (low value care).

Design: Systematic review and meta-analysis.

Data sources: Medline and Scopus databases, from inception to 10 July 2024.

Eligibility criteria for selecting studies: Randomised trials comparing de-implementation interventions with placebo or sham intervention, no intervention, or other de-implementation intervention strategies in primary care. Eligible trials provided information on the use of low value care, total volume of care, appropriate care, and health outcomes.

Data extraction and synthesis: Titles, abstracts, and full texts were screened, data were extracted, and risk of bias was assessed independently and in duplicate. Random effects meta-analyses were conducted, and the certainty of evidence was assessed with the Grading of Recommendations Assessment, Development, and Evaluation (GRADE) approach.

Results: 13 008 abstracts were screened and 140 were eligible for inclusion in the study. Median follow-up was 287 days (interquartile range 180-365). In 75 (54%) trials the aim was to reduce the use of antibiotics, in 42 (30%) to reduce other drug treatments, in 17 (12%) to reduce imaging, and in 15 (11%) to reduce laboratory testing. The certainty of the evidence was moderate that provider education combined with audit and feedback reduced the use of targeted low value care (odds ratio 0.73, 95% confidence interval (95% CI) 0.63 to 0.84). Provider education (0.86, 95% CI 0.72 to 1.03), audit and feedback (0.82, 0.67 to 1.00), and patient education (0.70, 0.30 to 1.66), and a combination of these strategies (point estimates for odds ratios ranging from 0.57 to 0.64) may reduce the use of targeted low value care (low certainty of evidence for all).

Conclusions: The results suggested with moderate certainty of evidence that provider education combined with audit and feedback reduced the use of targeted low value care. Individual strategies may slightly reduce the use of targeted low value care, but achieving a meaningful impact on low value care may require the use of multiple strategies. The results may be useful for patients, clinicians, policy makers, and guideline developers when deciding on future de-implementation strategies and research priorities.

Systematic review registration: PROSPERO CRD42023411768.

目的:评估初级保健中各种去实施干预措施的有效性,针对对患者没有或有限价值的护理(低价值护理)(治疗或检查)。设计:系统回顾和荟萃分析。数据来源:Medline和Scopus数据库,从成立到2024年7月10日。选择研究的资格标准:在初级保健中比较去实施干预与安慰剂或假干预、不干预或其他去实施干预策略的随机试验。符合条件的试验提供了关于低价值护理的使用、护理总量、适当护理和健康结果的信息。数据提取和综合:筛选标题、摘要和全文,提取数据,独立评估偏倚风险,一式两份。进行随机效应荟萃分析,并采用分级推荐评估、发展和评价(GRADE)方法评估证据的确定性。结果:筛选了13 008篇摘要,其中140篇符合纳入研究的条件。中位随访为287天(四分位数范围180-365)。75项(54%)试验的目的是减少抗生素的使用,42项(30%)试验的目的是减少其他药物治疗,17项(12%)试验的目的是减少影像学检查,15项(11%)试验的目的是减少实验室检查。证据的确定性是中等的,提供者教育结合审计和反馈减少了有针对性的低价值护理的使用(优势比0.73,95%置信区间(95% CI) 0.63至0.84)。提供者教育(0.86,95% CI 0.72 - 1.03)、审计和反馈(0.82,0.67 - 1.00)和患者教育(0.70,0.30 - 1.66),以及这些策略的组合(优势比的点估计范围为0.57 - 0.64)可能会减少有针对性的低价值护理的使用(所有证据的低确定性)。结论:结果表明,有中等确定性的证据表明,提供者教育结合审计和反馈减少了有针对性的低价值护理的使用。个别策略可能会略微减少有针对性的低价值护理的使用,但要对低价值护理产生有意义的影响,可能需要使用多种策略。研究结果可能对患者、临床医生、政策制定者和指南制定者在决定未来的反实施策略和研究重点时有用。系统评价注册:PROSPERO CRD42023411768。
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引用次数: 0
Prescription rates in different groups of outpatients with covid-19 and other acute respiratory infections: comparative observational study based on German routine data. 不同组门诊covid-19及其他急性呼吸道感染患者的处方率:基于德国常规数据的比较观察研究
IF 1 Pub Date : 2025-09-04 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2024-000992
Lena Marie Paschke, Kerstin Klimke, Maike Below

Objectives: To identify and quantify prescriptions after a covid-19 infection compared with other acute respiratory infections in previously healthy patients and those with chronic disease.

Design: Comparative observational study based on German routine data.

Setting: Ambulatory care of all residents in Germany with statutory health insurance (88% of the German population).

Participants: Adults receiving a diagnosis of covid-19 or an acute respiratory infection between the fourth quarter of 2020 and the second quarter of 2021 who had rarely (70 797 and 173 822 with covid-19 and acute respiratory infection, respectively) or frequently (900 593 and 1 755 691, respectively) accessed outpatient medical care in the past.

Main outcome measures: Difference in differences in the proportion of prescriptions of relevant drugs before and one year after infection.

Results: In patients who used the healthcare system less frequently before their covid-19 infection than afterwards, increases in prescription rates for antidiabetics (difference in differences 0.23%, P=0.007), antithrombotics (0.71%, P=0.02), and cardiovascular drugs like beta blockers (0.25%, P=0.03) were observed compared with patients with other acute respiratory infections. One year after infection, the difference in antidiabetic prescription rates was highest. Although a peak in antihypertensive prescription rates was observed six months after infection, antithrombotics were predominantly prescribed during the acute phase. Conversely, patients who had already used the healthcare system on a regular basis before their infection showed no significant long term increases in prescription rates across the drug groups analysed.

Conclusions: This study supports findings that diseases such as diabetes and cardiovascular disease are more prevalent after covid-19 than after other acute respiratory infections. Because the effect is apparent in real world data, future societal implications should be considered, including increased disease burden and growing demand for medical care owing to the increasing need for drugs.

目的:确定和量化covid-19感染后的处方,并将其与既往健康患者和慢性病患者的其他急性呼吸道感染进行比较。设计:基于德国常规数据的比较观察研究。环境:为所有拥有法定医疗保险的德国居民(占德国人口的88%)提供门诊护理。参与者:在2020年第四季度至2021年第二季度期间被诊断为covid-19或急性呼吸道感染的成年人,他们过去很少(分别为70 797和173 822例患有covid-19和急性呼吸道感染)或经常(分别为900 593和1 755 691)获得门诊医疗服务。主要观察指标:感染前后1年相关药物处方比例差异。结果:与其他急性呼吸道感染患者相比,在感染前使用医疗保健系统次数较少的患者中,抗糖尿病药物(差异0.23%,P=0.007)、抗血栓药物(差异0.71%,P=0.02)和β受体阻滞剂等心血管药物(差异0.25%,P=0.03)的处方率均有所增加。感染后一年,抗糖尿病处方率的差异最大。虽然抗高血压处方率在感染后6个月达到高峰,但抗血栓药物主要是在急性期开的。相反,在感染之前已经定期使用医疗保健系统的患者在分析的药物组中,处方率没有显着的长期增长。结论:本研究支持了新冠肺炎后糖尿病和心血管疾病等疾病比其他急性呼吸道感染后更普遍的研究结果。由于这种影响在现实世界的数据中是明显的,因此应考虑到未来的社会影响,包括由于对药物的需求增加而增加的疾病负担和对医疗保健的需求。
{"title":"Prescription rates in different groups of outpatients with covid-19 and other acute respiratory infections: comparative observational study based on German routine data.","authors":"Lena Marie Paschke, Kerstin Klimke, Maike Below","doi":"10.1136/bmjmed-2024-000992","DOIUrl":"10.1136/bmjmed-2024-000992","url":null,"abstract":"<p><strong>Objectives: </strong>To identify and quantify prescriptions after a covid-19 infection compared with other acute respiratory infections in previously healthy patients and those with chronic disease.</p><p><strong>Design: </strong>Comparative observational study based on German routine data.</p><p><strong>Setting: </strong>Ambulatory care of all residents in Germany with statutory health insurance (88% of the German population).</p><p><strong>Participants: </strong>Adults receiving a diagnosis of covid-19 or an acute respiratory infection between the fourth quarter of 2020 and the second quarter of 2021 who had rarely (70 797 and 173 822 with covid-19 and acute respiratory infection, respectively) or frequently (900 593 and 1 755 691, respectively) accessed outpatient medical care in the past.</p><p><strong>Main outcome measures: </strong>Difference in differences in the proportion of prescriptions of relevant drugs before and one year after infection.</p><p><strong>Results: </strong>In patients who used the healthcare system less frequently before their covid-19 infection than afterwards, increases in prescription rates for antidiabetics (difference in differences 0.23%, P=0.007), antithrombotics (0.71%, P=0.02), and cardiovascular drugs like beta blockers (0.25%, P=0.03) were observed compared with patients with other acute respiratory infections. One year after infection, the difference in antidiabetic prescription rates was highest. Although a peak in antihypertensive prescription rates was observed six months after infection, antithrombotics were predominantly prescribed during the acute phase. Conversely, patients who had already used the healthcare system on a regular basis before their infection showed no significant long term increases in prescription rates across the drug groups analysed.</p><p><strong>Conclusions: </strong>This study supports findings that diseases such as diabetes and cardiovascular disease are more prevalent after covid-19 than after other acute respiratory infections. Because the effect is apparent in real world data, future societal implications should be considered, including increased disease burden and growing demand for medical care owing to the increasing need for drugs.</p>","PeriodicalId":72433,"journal":{"name":"BMJ medicine","volume":"4 1","pages":"e000992"},"PeriodicalIF":10.0,"publicationDate":"2025-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12414209/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145024891","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Common methodological issues in observational epidemiological studies of older adults. 老年人观察性流行病学研究中的常见方法学问题。
IF 1 Pub Date : 2025-09-02 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2025-001332
Emma Nichols, Eleanor Hayes-Larson
{"title":"Common methodological issues in observational epidemiological studies of older adults.","authors":"Emma Nichols, Eleanor Hayes-Larson","doi":"10.1136/bmjmed-2025-001332","DOIUrl":"10.1136/bmjmed-2025-001332","url":null,"abstract":"","PeriodicalId":72433,"journal":{"name":"BMJ medicine","volume":"4 1","pages":"e001332"},"PeriodicalIF":10.0,"publicationDate":"2025-09-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12406907/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145001997","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prognostic models for cardiovascular and kidney outcomes in people with type 2 diabetes: living systematic review and meta-analysis of observational studies. 2型糖尿病患者心血管和肾脏预后模型:观察性研究的实时系统回顾和荟萃分析
IF 1 Pub Date : 2025-08-14 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2025-001369
Daniel G Rayner, Darsh Shah, Si-Cheng Dai, David Gou, Jason Z X Chen, Arnav Agarwal, Reem A Mustafa, Veena Manja, Per Olav Vandvik, Thomas Agoritsas, Farid Foroutan
<p><strong>Objective: </strong>To summarise available evidence regarding the performance metrics of validated prognostic models on cardiovascular and kidney outcomes in adults with type 2 diabetes mellitus.</p><p><strong>Design: </strong>Living systematic review and meta-analysis of observational studies.</p><p><strong>Data sources: </strong>Medline, Embase, Central, and the Cochrane Database of Systematic Reviews from 1 January 2020 to 17 January 2024.</p><p><strong>Eligibility criteria for selecting studies: </strong>Studies validating prognostic models that predicted all cause and cardiovascular mortality, admission to hospital for heart failure, kidney failure, myocardial infarction, or ischaemic stroke in adults with type 2 diabetes mellitus, including people with established cardiovascular disease or chronic kidney disease, or both. Risk models evaluating composite outcomes were not eligible.</p><p><strong>Data synthesis: </strong>For each model and outcome, using a random effects model, the reported discrimination measures were pooled, reported as c statistics. Furthermore, when available, calibration plots were reconstructed and interpreted narratively. The Prediction Model Risk of Bias Assessment (PROBAST) tool was used to assess the risk of bias of each analysed study cohort and the Grading of Recommendations, Assessment, Development, and Evaluations (GRADE) approach to evaluate our certainty in the evidence.</p><p><strong>Results: </strong>6529 publications were identified, of which 35 studies reporting on 13 models were included, all of which were developed for general populations with type 2 diabetes but no established cardiovascular disease or chronic kidney disease. Among the identified models, the Risk Equations for Complications of Type 2 Diabetes (RECODe) and the UK Prospective Diabetes Study Outcomes Model 2 (UKPDS-OM2) evaluated all outcomes except for admission to hospital for heart failure. Relative to a threshold c statistic of 0.7, RECODe had an acceptable discrimination for cardiovascular mortality (0.79, high certainty), probably has an acceptable discrimination for myocardial infarction (0.72, moderate certainty) and stroke (0.71, moderate certainty), and may have an acceptable discrimination for kidney failure (0.76, low certainty). High certainty evidence suggests that UKPDS-OM2 has unacceptable discrimination for myocardial infarction (0.64) and stroke (0.65). RECODe showed acceptable calibration for cardiovascular mortality (high certainty), myocardial infarction (high certainty), and kidney failure (moderate certainty) but had unacceptable calibration for stroke (moderate certainty). UKPDS-OM2 showed acceptable calibration for cardiovascular mortality (moderate certainty), stroke (moderate certainty), and kidney failure (low certainty), but may have unacceptable calibration for myocardial infarction (moderate certainty).</p><p><strong>Conclusion: </strong>13 unique models were identified that evaluated cardiovascul
目的:总结关于2型糖尿病成人心血管和肾脏预后模型性能指标的现有证据。设计:观察性研究的动态系统评价和荟萃分析。数据来源:Medline, Embase, Central和Cochrane系统评价数据库,时间为2020年1月1日至2024年1月17日。选择研究的资格标准:验证预测2型糖尿病成人全因死亡率和心血管死亡率、因心力衰竭、肾衰竭、心肌梗死或缺血性中风住院的预后模型的研究,包括已确诊心血管疾病或慢性肾脏疾病的患者,或两者兼而有之。评估综合结果的风险模型不合格。数据综合:对于每个模型和结果,使用随机效应模型,将报告的歧视措施汇总,报告为c统计量。此外,在有条件的情况下,对校准图进行重建和叙事解释。使用预测模型偏倚风险评估(PROBAST)工具评估每个分析研究队列的偏倚风险,并使用推荐、评估、发展和评估分级(GRADE)方法评估证据的确定性。结果:共纳入6529篇出版物,其中35篇研究报告了13种模型,所有这些研究都是针对2型糖尿病的一般人群开发的,但没有确定的心血管疾病或慢性肾脏疾病。在已确定的模型中,2型糖尿病并发症风险方程(RECODe)和英国前瞻性糖尿病研究结果模型2 (UKPDS-OM2)评估了除因心力衰竭入院外的所有结果。相对于阈值c为0.7的统计量,RECODe对心血管疾病死亡率的鉴别可接受(0.79,高确定性),对心肌梗死(0.72,中等确定性)和中风(0.71,中等确定性)的鉴别可接受,对肾衰竭的鉴别可接受(0.76,低确定性)。高确定性证据表明,UKPDS-OM2对心肌梗死(0.64)和脑卒中(0.65)的鉴别是不可接受的。RECODe对心血管死亡率(高确定性)、心肌梗死(高确定性)和肾衰竭(中等确定性)的校准可接受,但对中风(中等确定性)的校准不可接受。UKPDS-OM2对心血管死亡率(中等确定性)、中风(中等确定性)和肾衰竭(低确定性)的校准可接受,但对心肌梗死(中等确定性)的校准可能不可接受。结论:确定了13种独特的模型来评估2型糖尿病患者的心血管和肾脏预后。RECODe和UKPDS-OM2两个模型评估了除因心力衰竭入院外的所有结果。在所有评估的预后模型中,RECODe在大多数结果的验证研究中具有可接受的辨别和校准;虽然,需要更多的研究直接比较模型。研究注册号:PROSPERO, CRD42023423075。读者注意:这篇文章是一篇生动的系统综述,将根据新出现的证据进行更新。从原始出版之日起,更新可能会在两年内发生。本版本为原文。
{"title":"Prognostic models for cardiovascular and kidney outcomes in people with type 2 diabetes: living systematic review and meta-analysis of observational studies.","authors":"Daniel G Rayner, Darsh Shah, Si-Cheng Dai, David Gou, Jason Z X Chen, Arnav Agarwal, Reem A Mustafa, Veena Manja, Per Olav Vandvik, Thomas Agoritsas, Farid Foroutan","doi":"10.1136/bmjmed-2025-001369","DOIUrl":"10.1136/bmjmed-2025-001369","url":null,"abstract":"&lt;p&gt;&lt;strong&gt;Objective: &lt;/strong&gt;To summarise available evidence regarding the performance metrics of validated prognostic models on cardiovascular and kidney outcomes in adults with type 2 diabetes mellitus.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Design: &lt;/strong&gt;Living systematic review and meta-analysis of observational studies.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Data sources: &lt;/strong&gt;Medline, Embase, Central, and the Cochrane Database of Systematic Reviews from 1 January 2020 to 17 January 2024.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Eligibility criteria for selecting studies: &lt;/strong&gt;Studies validating prognostic models that predicted all cause and cardiovascular mortality, admission to hospital for heart failure, kidney failure, myocardial infarction, or ischaemic stroke in adults with type 2 diabetes mellitus, including people with established cardiovascular disease or chronic kidney disease, or both. Risk models evaluating composite outcomes were not eligible.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Data synthesis: &lt;/strong&gt;For each model and outcome, using a random effects model, the reported discrimination measures were pooled, reported as c statistics. Furthermore, when available, calibration plots were reconstructed and interpreted narratively. The Prediction Model Risk of Bias Assessment (PROBAST) tool was used to assess the risk of bias of each analysed study cohort and the Grading of Recommendations, Assessment, Development, and Evaluations (GRADE) approach to evaluate our certainty in the evidence.&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Results: &lt;/strong&gt;6529 publications were identified, of which 35 studies reporting on 13 models were included, all of which were developed for general populations with type 2 diabetes but no established cardiovascular disease or chronic kidney disease. Among the identified models, the Risk Equations for Complications of Type 2 Diabetes (RECODe) and the UK Prospective Diabetes Study Outcomes Model 2 (UKPDS-OM2) evaluated all outcomes except for admission to hospital for heart failure. Relative to a threshold c statistic of 0.7, RECODe had an acceptable discrimination for cardiovascular mortality (0.79, high certainty), probably has an acceptable discrimination for myocardial infarction (0.72, moderate certainty) and stroke (0.71, moderate certainty), and may have an acceptable discrimination for kidney failure (0.76, low certainty). High certainty evidence suggests that UKPDS-OM2 has unacceptable discrimination for myocardial infarction (0.64) and stroke (0.65). RECODe showed acceptable calibration for cardiovascular mortality (high certainty), myocardial infarction (high certainty), and kidney failure (moderate certainty) but had unacceptable calibration for stroke (moderate certainty). UKPDS-OM2 showed acceptable calibration for cardiovascular mortality (moderate certainty), stroke (moderate certainty), and kidney failure (low certainty), but may have unacceptable calibration for myocardial infarction (moderate certainty).&lt;/p&gt;&lt;p&gt;&lt;strong&gt;Conclusion: &lt;/strong&gt;13 unique models were identified that evaluated cardiovascul","PeriodicalId":72433,"journal":{"name":"BMJ medicine","volume":"4 1","pages":"e001369"},"PeriodicalIF":10.0,"publicationDate":"2025-08-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12359462/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144884411","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of comorbidity measures for predicting mortality and revision surgery after elective primary shoulder replacement surgery based on data from the National Joint Registry and Hospital Episode Statistics for England: population based cohort study. 基于英国国家联合登记和医院事件统计的数据,评估择期原发性肩关节置换术后预测死亡率和翻修手术的合并症措施:基于人口的队列研究。
IF 1 Pub Date : 2025-08-10 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2024-001283
Epaminondas Markos Valsamis, Adrian Sayers, Jie Ma, Paula Dhiman, Stephen E Gwilym, Jonathan L Rees

Objective: To determine the importance of comorbidity measures when predicting mortality and revision surgery after elective primary shoulder replacement surgery.

Design: Population based cohort study.

Setting: Linked data from the National Joint Registry and NHS Hospital Episode Statistics were used to identify all elective primary shoulder replacements in England, 6 January 2012 to 30 March 2022.

Participants: 37 176 consenting patients, aged 18-100 years, who had elective primary shoulder replacement surgery.

Main outcome measures: Risk of mortality at 90 and 365 days, and risk of long term revision surgery after the primary surgery.

Results: 37 176 primary shoulder replacement procedures were included; 102 patients died within 90 days and 445 within 365 days of the primary surgery. 1219 patients had revision surgery over a maximum follow-up period of >10 years. The addition of comorbidity measures derived from Hospital Episode Statistics (Charlson comorbidity index with summary hospital mortality index weights, Elixhauser comorbidity index, and hospital frailty risk score) to simpler models resulted in little improvement in predictive performance. Optimism adjusted performance (C index) of the models that included age, sex, American Society of Anesthesiologists (ASA) grade, and main surgical indication was 0.76 (95% confidence interval (CI) 0.72 to 0.81) for 90 day mortality, 0.74 (0.71 to 0.76) for 365 day mortality, and 0.64 (0.63 to 0.66) for revision surgery. The best performing models that included a comorbidity measure had an optimism adjusted C index of 0.77 (95% CI 0.73 to 0.81) for 90 day mortality, 0.76 (0.74 to 0.78) for 365 day mortality, and 0.65 (0.63 to 0.66) for revision surgery. Heterogeneity in model performance across regions of England was low, and decision curve analysis showed minimal improvement in net benefit when including comorbidity measures.

Conclusions: In this study, patient comorbidity scores added little improvement to simpler models that included age, sex, ASA grade, and main surgical indication for predicting mortality and revision surgery after elective primary shoulder replacement surgery. This improvement needs to be balanced against the additional challenges of routine data linkage to obtain these scores.

目的:确定合并症指标在预测择期原发性肩关节置换术后死亡率和翻修手术中的重要性。设计:基于人群的队列研究。背景:2012年1月6日至2022年3月30日,来自国家联合登记处和NHS医院事件统计的相关数据被用于确定英格兰所有选择性初级肩关节置换术。参与者:37176名同意接受选择性原发性肩关节置换手术的患者,年龄18-100岁。主要结局指标:90天和365天的死亡风险,以及初次手术后长期翻修手术的风险。结果:纳入37 176例原发性肩关节置换术;102例患者在初次手术后90天内死亡,445例在365天内死亡。1219例患者进行了翻修手术,最长随访时间为10年。将来自医院事件统计的共病测量(Charlson共病指数与医院总死亡率指数权重、Elixhauser共病指数和医院虚弱风险评分)添加到更简单的模型中,预测性能几乎没有改善。包括年龄、性别、美国麻醉师学会(ASA)分级和主要手术指征的模型的乐观调整性能(C指数),90天死亡率为0.76(95%可信区间(CI) 0.72 ~ 0.81), 365天死亡率为0.74(0.71 ~ 0.76),翻修手术为0.64(0.63 ~ 0.66)。包含合并症测量的最佳模型,90天死亡率的乐观调整C指数为0.77 (95% CI 0.73至0.81),365天死亡率为0.76(0.74至0.78),翻修手术为0.65(0.63至0.66)。英国各地区模型表现的异质性很低,决策曲线分析显示,当包括合并症测量时,净效益的改善很小。结论:在本研究中,患者合并症评分对包括年龄、性别、ASA等级和主要手术指征在内的预测择期原发性肩关节置换术后死亡率和翻修手术的简单模型几乎没有改善。为了获得这些分数,这种改进需要与常规数据链接的额外挑战相平衡。
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引用次数: 0
Cardiorespiratory fitness in adolescence and risk of type 2 diabetes in late adulthood in one million Swedish men: nationwide sibling controlled cohort study. 100万瑞典男性青少年心肺健康和成年后期2型糖尿病风险:全国同胞对照队列研究
IF 1 Pub Date : 2025-08-07 eCollection Date: 2025-01-01 DOI: 10.1136/bmjmed-2024-001313
Marcel Ballin, Viktor H Ahlqvist, Daniel Berglind, Mattias Brunström, Angel Herraiz-Adillo, Pontus Henriksson, Martin Neovius, Francisco B Ortega, Anna Nordström, Peter Nordström
<p><strong>Objective: </strong>To examine the association between adolescent cardiorespiratory fitness and risk of type 2 diabetes in late adulthood, including the potential influence of unobserved familial confounding on the association.</p><p><strong>Design: </strong>Nationwide sibling controlled cohort study.</p><p><strong>Setting: </strong>Swedish Military Service Conscription Register, Sweden, 1972-95, with Multi-Generation Register for identifying full siblings. National Patient Register and Prescribed Drug Register for data on diagnoses of type 2 diabetes, deaths from National Cause of Death Register, and Statistics Sweden for emigration and socioeconomic data.</p><p><strong>Participants: </strong>1 124 049 Swedish men who participated in mandatory military conscription examinations with completed standardised cardiorespiratory fitness testing. Participants were followed up until 31 December 2023.</p><p><strong>Main outcome measures: </strong>Type 2 diabetes, defined as a composite endpoint of diagnosis in inpatient or specialist outpatient care and dispensation of antidiabetic drug treatment, until 31 December 2023.</p><p><strong>Results: </strong>1 124 049 men, including 477 453 full siblings, with a mean age of 18.3 (standard deviation 0.7) years at baseline were included. During follow-up, 115 958 men (10.3%) and 48 089 full siblings (10.1%) had a first type 2 diabetes event at a median age of 53.4 (interquartile range 47.6-59.3) years. Cardiorespiratory fitness was categorised into deciles (referred to as groups, with group 1 being the lowest fitness level and group 10 the highest). In a cohort analysis, the adjusted hazard ratio in fitness group 2 versus fitness group 1 was 0.83 (95% confidence interval (CI) 0.81 to 0.85), with a difference in the standardised cumulative incidence at age 65 years of 4.3 (95% CI 3.8 to 4.8) percentage points, decreasing to a hazard ratio of 0.38 (0.36 to 0.39; incidence difference 17.8 (17.3 to 18.3) percentage points) in fitness group 10. When comparing full siblings, and thus controlling for all unobserved shared behavioural, environmental, and genetic confounders, the association was replicated, but with a reduction in magnitude. The hazard ratio in fitness group 2 was 0.89 (95% CI 0.85 to 0.94; incidence difference 2.3 (1.3 to 3.3) percentage points) and 0.53 (0.50 to 0.57; incidence difference 10.9 (9.7 to 12.1) percentage points) in fitness group 10. Hypothetically moving all participants in fitness group 1 to fitness group 2 was estimated to prevent 7.2% (95% CI 6.4% to 8.0%) of events at age 65 years in the cohort analysis versus 4.6% (2.6% to 6.5%) in the full sibling analysis, whereas hypothetically moving all participants to fitness group 10 was estimated to prevent 35.6% (34.1% to 37.0%) versus 24.3% (20.5 to 28.0) of events. Indications of effect modification by overweight status were found, where the association was smaller in those with overweight than in those without overweight, parti
目的:探讨青少年心肺健康与成年后期2型糖尿病风险之间的关系,包括未观察到的家族混杂因素对这种关系的潜在影响。设计:全国同胞对照队列研究。背景:1972年至1995年,瑞典兵役征召登记簿(Swedish Military Service征兵登记簿)和多代登记簿(Multi-Generation Register),用于识别全兄妹。国家患者登记册和处方药登记册中的2型糖尿病诊断数据,国家死因登记册中的死亡数据,以及瑞典统计局的移民和社会经济数据。参与者:1 124 049名参加强制性征兵考试并完成标准化心肺功能测试的瑞典男性。参与者被随访至2023年12月31日。主要结局指标:2型糖尿病,定义为住院或专科门诊诊断和降糖药物治疗的复合终点,直至2023年12月31日。结果:纳入1 124 049名男性,包括477 453名全兄妹,基线时平均年龄为18.3岁(标准差0.7)。在随访期间,115 958名男性(10.3%)和48 089名全兄妹(10.1%)首次发生2型糖尿病事件,中位年龄为53.4岁(四分位数间距为47.6-59.3岁)。心肺健康被分为十分位数(称为组,第1组的健康水平最低,第10组的健康水平最高)。在队列分析中,健身组2与健身组1的校正风险比为0.83(95%可信区间(CI) 0.81至0.85),65岁时标准化累积发病率的差异为4.3 (95% CI 3.8至4.8)个百分点,风险比降至0.38(0.36至0.39;健身组发病率差异17.8(17.3 ~ 18.3)个百分点。当对全兄妹进行比较,从而控制所有未观察到的共同行为、环境和遗传混杂因素时,这种关联得到了复制,但程度有所降低。健身2组的危险比为0.89 (95% CI 0.85 ~ 0.94;发病率差2.3(1.3 ~ 3.3)个百分点)和0.53 (0.50 ~ 0.57);健身组发病率差异10.9(9.7 ~ 12.1)个百分点。假设将健身组1的所有参与者转移到健身组2,在队列分析中估计可以预防7.2% (95% CI 6.4%至8.0%)的65岁事件,而在全同胞分析中则为4.6%(2.6%至6.5%),而假设将所有参与者转移到健身组10估计可以预防35.6%(34.1%至37.0%)的事件,而24.3%(20.5至28.0)的事件。研究发现,超重状态改变了影响的迹象,超重人群的相关性小于没有超重的人群,特别是在全兄弟姐妹分析中。结论:研究结果表明,青少年心肺健康对成年后期2型糖尿病的发展可能很重要,但传统的观察性分析可能对影响的程度给出了有偏差的估计。
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