Annals of clinical epidemiology最新文献

{"title":"Designing Single-Arm Clinical Trials: Principles, Applications, and Methodological Considerations.","authors":"Shuna Yao, Qingyao Shang, Meishuo Ouyang, Heng Zhou, Zhihua Yao, Yanyan Liu, Sheng Luo","doi":"10.37737/ace.25011","DOIUrl":"10.37737/ace.25011","url":null,"abstract":"<p><p>Single-arm trials (SATs) are clinical studies without a parallel control group, serving as a vital alternative to randomized controlled trials (RCTs) in scenarios where traditional trial designs are impractical. These trials are particularly relevant in rare diseases, advanced malignancies, novel treatment modalities, and life-threatening conditions, where ethical concerns, logistical challenges, or small patient populations limit the feasibility of RCTs. SATs enable expedited evaluation of therapeutic interventions, often forming the foundation for regulatory approvals. This article explores the principles, applications, and methodological considerations of SATs. Their advantages include smaller sample size requirements, faster timelines, and regulatory acceptance by agencies such as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA). Despite these benefits, SATs face challenges, such as potential biases due to the lack of a control group, limitations in endpoints, and reliance on historical controls that may compromise result validity. Best practices in SAT design are outlined, including refining scientific questions, defining eligibility criteria, selecting clinically meaningful endpoints, and employing robust statistical methods like Simon's two-stage design and Bayesian approaches.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 3","pages":"90-98"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12279405/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144693066","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}

{"title":"Propensity score analysis revisited.","authors":"Yohei Hashimoto, Hideo Yasunaga","doi":"10.37737/ace.25012","DOIUrl":"10.37737/ace.25012","url":null,"abstract":"<p><p>Propensity score (PS) is the probability of the exposure being assigned, conditional on the observed baseline covariates. Many observational studies have used PS analyses to investigate the effects of exposure on outcomes. This report reviews the five steps of PS analyses: 1) calculating PS; 2) checking the overlap of PS; 3) implementing a matching or weighting method including PS matching, inverse-probability-of-treatment weighting, standardized mortality ratio weighting, matching weighting, and overlap weighting; 4) diagnosing the covariate balance; and 5) comparing the outcomes. Two groups are often compared in PS analyses; however, three-group comparisons can provide clinicians with more benefits in many situations in routine clinical practice. Thus, we describe not only two-group comparisons but also three-group comparisons by introducing a few studies that used generalized PS to compare three groups.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 3","pages":"99-104"},"PeriodicalIF":0.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12279407/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144693067","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}

{"title":"Risk factors for nephropathy in individuals with new-onset type 2 diabetes undergoing treatment for hypertension: A retrospective analysis using the Diagnosis Procedure Combination database.","authors":"Tomoko Ishigaki Suzuki, Mari Saito Oba, Kohei Uemura","doi":"10.37737/ace.25009","DOIUrl":"10.37737/ace.25009","url":null,"abstract":"<p><strong>Background: </strong>Diabetic nephropathy is a common complication of diabetes. We investigated the risk factors for diabetic nephropathy in individuals newly diagnosed with type 2 diabetes.</p><p><strong>Methods: </strong>Data from the Japanese Diagnosis Procedure Combination in-patient database (April 2008 to December 2018) were analyzed. The endpoint was subsequent diabetic nephropathy diagnosis or as the time when estimated glomerular filtration rate become <60 ml/min/1.73 m². Candidate risk factors included age, Hemoglobin A1c, log-transformed triglyceride, total cholesterol, and high-density lipoprotein cholesterol levels, body mass index, and estimated glomerular filtration rate. Eligible individuals with type 2 diabetes without complications who had pre- and post-diagnosis Hemoglobin A1c and serum creatinine measurements, and a history of hypertension or cardiovascular disease pre-diagnosis. Those with pre-existing kidney diseases, nephropathy onset pre-diagnosis, estimated glomerular filtration rate <60 ml/min/1.73 m² on or before diabetes diagnosis, or age <20 years at diabetes diagnosis were excluded. A multivariate Cox proportional hazards model (p = 0.2 backward selection) was employed.</p><p><strong>Results: </strong>Of 2,664 eligible individuals (1,775 men, 889 women), 325 men and 175 women developed diabetic nephropathy during follow-up. Cumulative incidence within 5 years was 29.0% in men and 32.5% in women. Age and estimated glomerular filtration rate in both sexes, and total cholesterol in men were significant.</p><p><strong>Conclusions: </strong>Age, estimated glomerular filtration rate, and lipid pose potential risks for diabetic nephropathy onset within 5 years of diabetes diagnosis in individuals with hypertension. Collectively, our findings highlight the importance of early monitoring and intervention in this high-risk.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 3","pages":"71-79"},"PeriodicalIF":0.0,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12279404/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144693068","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}

{"title":"Changes in the Use of Telephone Follow-ups among Patients with Urologic Malignancies in Japan.","authors":"Keiko Fujino, Naoya Masumori, Shiro Hinotsu","doi":"10.37737/ace.25010","DOIUrl":"10.37737/ace.25010","url":null,"abstract":"<p><strong>Background: </strong>This study aimed to compare telephone follow-ups for patients with urologic malignancies in Japan before and during the COVID-19 pandemic.</p><p><strong>Methods: </strong>Using the database of the Japan Medical Data Center Co., Ltd., we identified patients with urologic malignancies who underwent at least one telephone follow-up between April 2014 and March 2020.The self-controlled case series method was used to calculate the incidence rate ratio of telephone follow-up utilization for the pandemic using the pre-pandemic period as a reference.</p><p><strong>Results: </strong>Of the 289 patients, 31 were women. Their median age was 80 years, and the median observation period was 28 months.The incidence rate ratio for telephone follow-up utilization during the pandemic was 17.8 compared to that of the pre-pandemic period. In an analysis stratified by type of carcinoma, the incidence rate ratios were higher in all strata than they were before the pandemic. However, among male patients with genital malignancies, particularly prostate cancers, the incidence rate ratio was lower than in other strata.According to analysis stratified by age, the usage of telephone follow-up for men in their 50s or younger was particularly low. Additionally, the interval between face-to-face visits increased in patients over 60 years-of-age.</p><p><strong>Conclusions: </strong>The telephone follow-up among patients with urologic malignancies in Japan increased significantly during the early phase of the COVID-19 pandemic. Our results indicate that telephone follow-up can potentially be a valuable patient-doctor communication tool.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 3","pages":"80-89"},"PeriodicalIF":0.0,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12279406/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144693065","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}

{"title":"Adverse events, including fractures, among older patients receiving mirogabalin versus pregabalin: A retrospective cohort study using a large claims database in Japan.","authors":"Kanako Makito, Akira Okada, Hideo Yasunaga","doi":"10.37737/ace.25008","DOIUrl":"https://doi.org/10.37737/ace.25008","url":null,"abstract":"<p><strong>Background: </strong>Mirogabalin has a mechanism similar to that of pregabalin in the treatment of neuropathic pain. However, it remains unclear whether these drugs differ in terms of serious side effects, such as fall-related fractures, in older patients. This study aimed to investigate whether mirogabalin is associated with a decrease in adverse events, including fall-related fractures, compared with pregabalin.</p><p><strong>Methods: </strong>We performed a retrospective cohort study using the DeSC database, a large administrative claims database in Japan. This study included 130,244 patients ≥65 years taking mirogabalin or pregabalin between April 2019 and May 2021. The primary outcome was defined as the occurrence of fractures or switching to other medications and was compared between those receiving mirogabalin and pregabalin using Kaplan-Meier curves and multivariable Cox proportional hazards models. A sensitivity analysis was performed regarding patients who received mirogabalin or pregabalin without other analgesic medications at the initial dose.</p><p><strong>Results: </strong>During a median follow-up of 2.8 months, 29,686 (22.8%) and 100,558 (77.2%) received mirogabalin and pregabalin, respectively. The rates of the outcome in the mirogabalin and pregabalin groups were 50.1 and 42.8 per 100 person-years. Cox regression analysis showed that mirogabalin was associated with a lower risk of the outcome (hazard ratio, 0.93; 95% confidence interval, 0.87-1.00). However, sensitivity analysis did not demonstrate a difference in the outcome between the mirogabalin and pregabalin groups without other analgesic medications (hazard ratio, 0.93; 95% confidence interval, 0.86-1.01).</p><p><strong>Conclusions: </strong>Our analyses suggest that the outcome may be less likely in the mirogabalin group; however, the difference appears to be clinically insignificant. Further studies are warranted to confirm these findings.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 2","pages":"61-68"},"PeriodicalIF":0.0,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11982630/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144060122","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}

{"title":"Estimation of the adjusted risk difference for very rare events, large samples, and extreme exposure frequency: Application of Vaccine Effectiveness, Networking, and Universal Safety study data.","authors":"Shuntaro Sato, Yurika Kawazoe, Fumiko Murata, Megumi Maeda, Haruhisa Fukuda","doi":"10.37737/ace.25007","DOIUrl":"https://doi.org/10.37737/ace.25007","url":null,"abstract":"<p><strong>Background: </strong>The post-authorization safety study of a vaccine is an important public health task, and its results contribute to the decisions about whether to recommend a vaccination by estimating not only the risk ratio but also the risk difference. There are few reports of adjusted risk differences. We evaluated the statistical performance of the adjusted risk difference and its variance under a post-authorization safety study's settings (rare events, large sample, extreme exposure frequency).</p><p><strong>Methods: </strong>Adjusted risk differences were estimated using ordinary least squares estimators in a linear regression model with a binary outcome, and their variances were estimated using the standard error from ordinary least squares and four types of robust variance. In a simulation, we evaluated the risk differences' performances using bias, coverage, and power and using data from the Vaccine Effectiveness, Networking, and Universal Safety study as an example of an actual post-authorization safety study.</p><p><strong>Results: </strong>The adjusted risk difference using ordinary least squares was not biased. Compared to the ordinary least squares' standard error, the robust variance achieved more appropriate coverage and higher power. With actual data, including 2 × 2 tables of exposure and outcome with zero, both the ordinary least squares and robust variance could be estimated.</p><p><strong>Conclusions: </strong>In post-authorization safety study settings, the estimation of the risk difference using ordinary least squares and robust variance showed better performance than the typical ordinary least squares. These findings may prove beneficial for reporting risk difference in extreme settings such as post-authorization safety studies.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 2","pages":"50-60"},"PeriodicalIF":0.0,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11982631/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144014822","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}

{"title":"Erratum to: Introduction to Mendelian randomization.","authors":"Shiu Lun Au Yeung, Shan Luo, Masao Iwagami, Atsushi Goto","doi":"10.37737/ace.25004E","DOIUrl":"https://doi.org/10.37737/ace.25004E","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.37737/ace.25004.].</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 2","pages":"69"},"PeriodicalIF":0.0,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11982634/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144056662","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}

{"title":"Cross-sectional Survey of Allergic Diseases in Staff and Their Families at Designated Allergic Disease Medical Hospitals in Japan: Calculation of Age-adjusted Prevalence.","authors":"Yasunori Ito, Taisuke Kato, Koichi Yoshida, Kyohei Takahashi, Yuma Fukutomi, Mizuho Nagao, Tatsuki Fukuie, Hiroshi Matsuzaki, Minoru Gotoh, Akio Tanaka, Satoshi Konno, Junichiro Tezuka, Naoki Kajita, Yuri Matsubara, Masakazu Takahashi, Yosikazu Nakamura, Yuichi Adachi","doi":"10.37737/ace.25005","DOIUrl":"https://doi.org/10.37737/ace.25005","url":null,"abstract":"<p><strong>Background: </strong>Continuous epidemiological surveys using consistent methodologies are essential for assessing the prevalence of allergic diseases. In 2021, a cross-sectional survey on allergic diseases was conducted, targeting staff and their families at Designated Allergic Disease Medical Hospitals across 41 prefectures in Japan. By 2022, these hospitals had been established in all 47 prefectures of Japan.</p><p><strong>Methods: </strong>A prevalence survey of allergic diseases was conducted in 2022 using the same questionnaire as that used in 2021, encompassing hospitals across all 47 prefectures. Age-adjusted prevalence rates were calculated using the 2015 population model for comparison with the 2021 survey data.</p><p><strong>Results: </strong>The 2022 survey included 76 hospitals and 24,444 participants (males, 10,668; women, 13,776; median age, 35 years; interquartile range, 18-50 years). The overall prevalence of allergic diseases was 63.0% (95% confidence interval:62.3-63.6). Age-adjusted prevalence of a history per 100,000 people for each allergic disease was as follows (; 95% confidence interval): bronchial asthma (13,279; 12,776-13,782), atopic dermatitis (12,786; 12,379-13,193), food allergies (13,143; 12,689-13,596), perennial allergic rhinitis (28,132; 27,673-28,982), pollinosis (38,959; 38,216-39,703), allergic conjunctivitis (19,751; 19,188-20,313), metal allergies (2,111; 1,898-2,325), drug allergies (4,478; 4,149-4,806), and anaphylaxis (1,602; 1,417-1,787). The prevalence of perennial allergic rhinitis, pollinosis, and allergic conjunctivitis was higher than that in 2021.</p><p><strong>Conclusions: </strong>This survey is the first in Japan to calculate the age-adjusted prevalence of allergic diseases. Moreover, pollinosis is the most common allergic disease in Japan.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 2","pages":"39-45"},"PeriodicalIF":0.0,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11982633/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144057064","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}

{"title":"DeSC database.","authors":"Hideo Yasunaga","doi":"10.37737/ace.25006","DOIUrl":"https://doi.org/10.37737/ace.25006","url":null,"abstract":"<p><p>The DeSC database is a commercially available administrative claims and health checkup database in Japan. The DeSC database contains health insurance claims data from three types of health insurers: the National Health Insurance (Kokuho), Health Insurance Societies (Kempo), and the Advanced Elderly Medical Service System. A previous study has shown that the population in the DeSC database is representative of the entire population of Japan. Our literature search identified 56 original articles conducted using the DeSC database between April 2022 and September 2024. Although the number of studies using the DeSC database is increasing, there are still opportunities to enhance research on various topics.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 2","pages":"46-49"},"PeriodicalIF":0.0,"publicationDate":"2025-01-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11982632/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144061686","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}

{"title":"Introduction to Mendelian randomization.","authors":"Shiu Lun Au Yeung, Shan Luo, Masao Iwagami, Atsushi Goto","doi":"10.37737/ace.25004","DOIUrl":"10.37737/ace.25004","url":null,"abstract":"<p><p>Mendelian randomization (MR), i.e. instrumental variable analysis using genetic instruments, is an approach that incorporates population genetics to improve causal inference. Given that genetics are randomly allocated at conception, this resembles the randomization process in randomized controlled trials and hence is more resistant to unobserved confounding compared to conventional observational studies (e.g. cohort studies). The seminar paper briefly described the origin of MR and its underlying assumptions (relevance, independence, and exclusion restriction). This was followed by introducing one sample MR designs (in which instrument-exposure and instrument-outcome associations are derived from the same sample) and one sample MR design (in which instrument-exposure and instrument-outcome associations are derived from different samples). The seminar paper then summarized key aspects of MR studies, such as instrument selection, data sources for conducting MR studies, and statistical analyses. Variations of MR design were also introduced, such as how this design can inform the effect of drug targets (drug target MR). The STROBE-MR checklist and relevant MR guidelines were introduced. The seminar paper concluded by discussing the credibility crisis of MR studies.</p>","PeriodicalId":517436,"journal":{"name":"Annals of clinical epidemiology","volume":"7 1","pages":"27-37"},"PeriodicalIF":0.0,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11799858/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143384887","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}