American journal of epidemiology最新文献

{"title":"Triple challenges-small sample size in both exposure and control groups to scan rare maternal outcomes in a signal identification approach: a simulation study.","authors":"Thuy N Thai, Almut G Winterstein, Elizabeth A Suarez, Jiwei He, Yueqin Zhao, Di Zhang, Danijela Stojanovic, Jane Liedtka, Abby Anderson, José J Hernández-Muñoz, Monica Munoz, Wei Liu, Inna Dashevsky, Elizabeth Messenger-Jones, Elizabeth Siranosian, Judith C Maro","doi":"10.1093/aje/kwae151","DOIUrl":"10.1093/aje/kwae151","url":null,"abstract":"<p><p>There is a dearth of safety data on maternal outcomes after perinatal medication exposure. Data-mining for unexpected adverse event occurrence in existing datasets is a potentially useful approach. One method, the Poisson tree-based scan statistic (TBSS), assumes that the expected outcome counts, based on incidence of outcomes in the control group, are estimated without error. This assumption may be difficult to satisfy with a small control group. Our simulation study evaluated the effect of imprecise incidence proportions from the control group on TBSS' ability to identify maternal outcomes in pregnancy research. We simulated base case analyses with \"true\" expected incidence proportions and compared these with imprecise incidence proportions derived from sparse control samples. We varied parameters that have an impact on type I error and statistical power (exposure group size, outcome's incidence proportion, and effect size). We found that imprecise incidence proportions generated by a small control group resulted in inaccurate alerting, inflation of type I error, and removal of very rare outcomes for TBSS analysis due to \"zero\" background counts. Ideally, the control size should be at least several times larger than the exposure size to limit the number of false positive alerts and retain statistical power for true alerts. This article is part of a Special Collection on Pharmacoepidemiology.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":"1805-1813"},"PeriodicalIF":5.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141449402","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 consultants 1.","authors":"","doi":"10.1093/aje/kwae429","DOIUrl":"10.1093/aje/kwae429","url":null,"abstract":"","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":"i-iv"},"PeriodicalIF":5.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142674876","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":"Statistical approaches for the integration of external controls in a cystic fibrosis clinical trial: a simulation and an application.","authors":"Mark N Warden, Sonya L Heltshe, Noah Simon, Stephen J Mooney, Nicole Mayer-Hamblett, Amalia S Magaret","doi":"10.1093/aje/kwae148","DOIUrl":"10.1093/aje/kwae148","url":null,"abstract":"<p><p>Development of new therapeutics for a rare disease such as cystic fibrosis is hindered by challenges in accruing enough patients for clinical trials. Use of external controls from well-matched historical trials can reduce prospective trial sizes, and this approach has supported regulatory approval of new interventions for other rare diseases. Here we consider 3 statistical methods that incorporate external controls into a hypothetical clinical trial of a new treatment to reduce pulmonary exacerbations in cystic fibrosis patients: (1) inverse probability weighting, (2) bayesian modeling with propensity-score-based power priors, and (3) hierarchical bayesian modeling with commensurate priors. We compare the methods via simulation study and in a real clinical-trial data setting. Simulations showed that bias in the treatment effect was less than 4% using any of the methods, with type I error (or in the bayesian cases, posterior probability of the null hypothesis) usually less than 5%. Inverse probability weighting was sensitive to similarity in prevalence of the covariates between historical and prospective trial populations. The commensurate prior method performed best with real clinical trial data. Using external controls to reduce trial size in future clinical trials holds promise and can advance the therapeutic pipeline for rare diseases. This article is part of a Special Collection on Pharmacoepidemiology.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":"1796-1804"},"PeriodicalIF":5.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11637530/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141449400","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":"Cardiorenal effects of angiotensin-converting enzyme inhibitors and angiotensin receptor blockers among people underrepresented in trials: analysis of routinely collected data with emulation of a reference trial (ONTARGET).","authors":"Paris J Baptiste, Angel Y S Wong, Anna Schultze, Catherine M Clase, Clémence Leyrat, Elizabeth Williamson, Emma Powell, Johannes F E Mann, Marianne Cunnington, Koon Teo, Shrikant I Bangdiwala, Peggy Gao, Laurie Tomlinson, Kevin Wing","doi":"10.1093/aje/kwae137","DOIUrl":"10.1093/aje/kwae137","url":null,"abstract":"<p><p>Cardiovascular disease is a leading cause of death globally. Angiotensin-converting enzyme inhibitors (ACEi) and angiotensin receptor blockers (ARB), compared in the ONTARGET trial (Ongoing Telmisartan Alone and in Combination with Ramipril Global Endpoint Trial), each prevent cardiovascular disease. However, trial results may not be generalizable, and their effectiveness in underrepresented groups is unclear. Using trial emulation methods within routine-care data to validate findings, we explored the generalizability of ONTARGET results. For people prescribed an ACEi/ARB in the UK Clinical Practice Research Datalink GOLD dataset from January 1, 2001, to July 31, 2019, we applied trial criteria and propensity-score methods to create an ONTARGET trial-eligible cohort. Comparing ARB with ACEi, we estimated hazard ratios for the primary composite trial outcome (cardiovascular death, myocardial infarction, stroke, or hospitalization for heart failure) and secondary outcomes. Because the prespecified criteria were met, confirming trial emulation, we then explored treatment heterogeneity among 3 trial-underrepresented subgroups: females, persons aged ≥75 years, and those with chronic kidney disease. In the trial-eligible population (n = 137 155), results for the primary outcome demonstrated similar effects of ARB and ACEi (hazard ratio = 0.97; 95% CI, 0.93-1.01), meeting the prespecified validation criteria. When extending this outcome to trial-underrepresented groups, similar treatment effects were observed by sex, age, and chronic kidney disease. This suggests that ONTARGET trial findings are generalizable to trial-underrepresented subgroups. This article is part of a Special Collection on Pharmacoepidemiology.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":"1785-1795"},"PeriodicalIF":5.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11637514/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141417218","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":"A prospective cohort study of persistent endocrine-disrupting chemicals and perceived stress.","authors":"Samantha Schildroth, Amelia K Wesselink, Traci N Bethea, Birgit Claus Henn, Alexa Friedman, Victoria Fruh, Chad M Coleman, Sharonda M Lovett, Anissa I Vines, Andreas Sjodin, Julianne Cook Botelho, Antonia M Calafat, Ganesa Wegienka, Jennifer Weuve, Donna D Baird, Lauren A Wise","doi":"10.1093/aje/kwae088","DOIUrl":"10.1093/aje/kwae088","url":null,"abstract":"<p><p>Persistent endocrine-disrupting chemicals (EDCs) can dysregulate the stress response. We evaluated associations between persistent EDCs and perceived stress among participants in the Study of Environment, Lifestyle, and Fibroids (n = 1394), a prospective cohort study of Black women. Participants completed the Perceived Stress Scale 4 (PSS-4) at baseline and every 20 months through 60 months (score range: 0-16); higher scores indicate higher stress. Endocrine-disrupting chemicals, including per- and polyfluoroalkyl substances, polychlorinated biphenyls (PCBs), polybrominated diphenyl ethers (PBDEs), and organochlorine pesticides, were quantified in plasma samples at baseline. We fit bayesian kernel machine regression and linear mixed-effects models to estimate associations of EDCs (as a mixture and individually) with PSS-4 scores at baseline and at each follow-up visit, respectively. Increasing percentiles of the mixture were not strongly associated with PSS-4 scores at baseline, and no interactions were observed among EDCs. Several individual EDCs (eg, perfluorodecanoic acid, PCB 118, PBDE 99) were associated with higher PSS-4 scores at baseline or follow-up, and other EDCs (eg PCB 138/158) were associated with lower PSS-4 scores at baseline or follow-up. The directionality of associations for individual EDCs was inconsistent across follow-up visits. In conclusion, specific EDCs may be associated with perceived stress in Black women. This article is part of a Special Collection on Environmental Epidemiology.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":"1729-1740"},"PeriodicalIF":5.0,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11637482/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141157085","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":"Body size and occupational physical activity during the life course and gastrointestinal cancers.","authors":"Michele Sassano, Mahdi Nalini, Monireh Sadat Seyyedsalehi, Gholamreza Roshandel, Hossein Poustchi, Masoud Khoshnia, Farin Kamangar, Sanford M Dawsey, Christian Abnet, Reza Malekzadeh, Paolo Boffetta, Arash Etemadi","doi":"10.1093/aje/kwae438","DOIUrl":"https://doi.org/10.1093/aje/kwae438","url":null,"abstract":"<p><p>Evidence on the associations of body size and occupational physical activity (PA) during the life course with gastrointestinal cancers is inconclusive. We analyzed data from the Golestan Cohort Study, a population-based prospective cohort study from Iran, with 50,045 individuals aged 40-75 years enrolled during 2004-2008 and followed through April 2023. Body size during adolescence, early adulthood, and at cohort baseline was assessed using validated pictograms. Large adolescent body size was associated with total gastrointestinal cancers (hazard ratio, HR: 1.09; 95% confidence interval, CI: 1.02-1.16) and liver cancer (HR: 1.49; 95% CI: 1.11-2.01). Large early-adulthood body size was associated with liver cancer (HR: 1.85; 95% CI: 1.35-2.52). Compared with normal weight during all stages, overweight or obesity during adolescence only or early adulthood only were associated with stomach cancer, esophageal squamous cell carcinoma, and liver cancer. Lower levels of adolescent occupational PA were associated with colon cancer (HR: 1.39; 95% CI: 1.10-1.75), while lower levels at cohort baseline were positively associated with colon cancer and inversely associated with rectal cancer. Our findings suggest that timing of obesity during the life course might differently affect risk of gastrointestinal cancers, while occupational PA was found to be associated with colon cancer only.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":""},"PeriodicalIF":5.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824014","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":"Reparations for African Enslavement in the U.S. and Black Survival Using the Panel Study of Income Dynamics.","authors":"Jourdyn A Lawrence, Jaquelyn L Jahn, Joy Shi, Kathryn E W Himmelstein, Justin M Feldman, Natalia Linos, Mary T Bassett","doi":"10.1093/aje/kwae444","DOIUrl":"https://doi.org/10.1093/aje/kwae444","url":null,"abstract":"<p><p>Activists, policymakers, and scholars increasingly have advocated for reparations payments to Black Americans to redress the harms of enslavement and discriminatory practices that followed. This study examined the effects of a hypothetical monetary reparations intervention on all-cause premature and overall mortality among Black adults in the U.S. We used the Black-white wealth gap to calculate monetary costs, modeling the effects of wealth influxes of $905,426.10 (in 2019 USD) to each Black household (the amount necessary to eliminate the mean Black-white wealth gap), distributed over 10 years. We applied a target trial emulation framework to data in the Panel Study on Income Dynamics (n=16,010). Each Black household head or spouse/partner was followed from baseline until death, incomplete follow-up, 18 years after baseline, or the end of follow-up in 2019, whichever occurred first. Using the g-formula to account for time-fixed and time-varying confounders, we found a 29% reduction in premature mortality and a 25.6% reduction in overall mortality among Black adults under the reparations intervention. Our findings provide evidence that reparations are a lifesaving and justice-promoting social policy that could significantly contribute to efforts to eliminate health inequities.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":""},"PeriodicalIF":5.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142823524","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":"Sample composition and HIV prevention indicator differences using physical vs. virtual venue recruitment of men who have sex with men in San Francisco.","authors":"Izzy Chiu, Moranda Tate, Dillon Trujillo, Bow Suprasert, Alex Marr, Sean Arayasirikul, Erin C Wilson, Henry F Raymond, Willi McFarland","doi":"10.1093/aje/kwae443","DOIUrl":"https://doi.org/10.1093/aje/kwae443","url":null,"abstract":"<p><p>During the COVID-19 pandemic, the sampling method for the National HIV Behavioral Surveillance (NHBS) in San Francisco changed from physical venue time-location sampling (TLS) to online or virtual venue TLS for men who have sex with men (MSM). We present differences in the samples of MSM recruited using physical venue TLS in 2017 and virtual venue TLS in 2021. We further assess changes in preventive and risk behaviors from 2017 to 2021 after controlling for differences in the sample compositions with multivariable Poisson models using GLM with robust standard errors. Both sampling methods exceeded their targeted sample size of 500 (physical venue TLS n=502, virtual venue TLS n=505). Compared to physical venue TLS, the virtual venue TLS sample had fewer persons experiencing homelessness and incarceration, and more persons with health insurance and post-graduate degrees. After adjusting for these differences and age, race, and employment status, PrEP use increased from 2017 to 2021. The use of several non-injection drugs also increased, namely marijuana, poppers, ketamine, psilocybin, and LSD. We found virtual venue recruitment of MSM to be a viable approach for tracking trends in HIV-related behaviors, with notable appeal given possible future pandemic lockdowns of physical venues and changing socialization patterns.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":""},"PeriodicalIF":5.0,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142823533","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":"Effects of hypothetical low versus moderate-to-high intensity weight management regimens on knee replacements.","authors":"Takahiro Miki, Upul Cooray, Masashi Kanai, Yuta Hagiwara, Masayasu Murakami, Ken Osaka, Takaaki Ikeda","doi":"10.1093/aje/kwae446","DOIUrl":"https://doi.org/10.1093/aje/kwae446","url":null,"abstract":"<p><p>This study aimed to investigate the efficacy of a hypothetical biennial weight management regimen in reducing the necessity for knee replacement (KR) surgery among middle-aged and older adults with or at a higher risk for knee osteoarthritis (OA). Data from the Osteoarthritis Initiative cohort in the US, comprising community-dwelling adults aged 45-79 years at high risk for or with symptomatic knee OA who underwent baseline assessments from September 2008 to December 2010 were used. Subsequent evaluations were conducted at 12, 24, and 96 months. A hypothetical biennial weight management regimen, ranging from low to moderate-to-high intensity, was employed as the exposure, whereas the incidence of KR during follow-up was the outcome. This study, using a targeted learning approach to estimate hypothetical weight management regimens adjusted for time-varying confounding and attrition due to loss to follow-up, revealed that a 7.5% reduction in body mass index (high-intensity) resulted in a notable decrease in KR rates, from 5.93% [95% confidence interval (CI), 5.64-6.21] in the baseline maintenance regimen (low-intensity) to 3.60% (95% CI, 2.70-4.50) in the high-intensity regimen. In conclusion, a hypothetical weight management regimen significantly reduces the need for KR surgery in adults with knee OA.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":""},"PeriodicalIF":5.0,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142824016","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":"Understanding lipidomics associations and the lipoprotein-related caveats in population epidemiology.","authors":"Siyu Zhao, Pauli Ohukainen, Johannes Kettunen, Marjo-Riitta Järvelin, Mika Kähönen, Terho Lehtimäki, Jorma Viikari, Olli T Raitakari, Ville-Petteri Mäkinen, Mika Ala-Korpela","doi":"10.1093/aje/kwae445","DOIUrl":"https://doi.org/10.1093/aje/kwae445","url":null,"abstract":"<p><p>Mass spectrometry lipidomics is becoming customary to analyse serum/plasma samples in epidemiology. The measurables are molecular constituents of lipoprotein particles, but very little is known on the consequences of adjusting lipidomics data with lipoprotein measures. We studied two population cohorts with 5,657 and 2,036 participants. LC-MS/MS lipidomics was applied to analyse 24 molecular lipid classes and NMR spectroscopy to quantify seven lipoprotein lipids plus apolipoprotein A-I (apoA-I) and B (apoB). The associations of these measures were analysed via partial Spearman's correlations. The effects of nine different lipoprotein adjustments on these interrelationships were assessed. Multivariable regression modelling with these adjustments was also performed for the associations between the lipidomics data and BMI. These novel large-scale lipidomics data and their associations between the lipoprotein measures were coherent in both population cohorts, confirming the compatibility of the analytical approaches. Simulated data were generated to corroborate the mediation effects. The lipoprotein-related lipid-transport and metabolism inherently mediate the lipidomics associations as evident from the striking effects of the lipoprotein adjustments. These effects and their relevance to the interpretations of lipidomics data are presented and discussed in detail for the first time. The combined lipoprotein lipid adjustments appear prone to overadjustment and arbitrary biases.</p>","PeriodicalId":7472,"journal":{"name":"American journal of epidemiology","volume":" ","pages":""},"PeriodicalIF":5.0,"publicationDate":"2024-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142823558","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}