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Charles F. Manski 《The American statistician》2019,73(1):296-304
AbstractA central objective of empirical research on treatment response is to inform treatment choice. Unfortunately, researchers commonly use concepts of statistical inference whose foundations are distant from the problem of treatment choice. It has been particularly common to use hypothesis tests to compare treatments. Wald’s development of statistical decision theory provides a coherent frequentist framework for use of sample data on treatment response to make treatment decisions. A body of recent research applies statistical decision theory to characterize uniformly satisfactory treatment choices, in the sense of maximum loss relative to optimal decisions (also known as maximum regret). This article describes the basic ideas and findings, which provide an appealing practical alternative to use of hypothesis tests. For simplicity, the article focuses on medical treatment with evidence from classical randomized clinical trials. The ideas apply generally, encompassing use of observational data and treatment choice in nonmedical contexts. 相似文献
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Marjolein J. E. Greuter Xiang‐Ming Xu Jie‐Bin Lew Evelien Dekker Ernst J. Kuipers Karen Canfell Gerrit A. Meijer Veerle M. H. Coupé 《Risk analysis》2014,34(5):889-910
Several colorectal cancer (CRC) screening models have been developed describing the progression of adenomas to CRC. Currently, there is increasing evidence that serrated lesions can also develop into CRC. It is not clear whether screening tests have the same test characteristics for serrated lesions as for adenomas, but lower sensitivities have been suggested. Models that ignore this type of colorectal lesions may provide overly optimistic predictions of the screen‐induced reduction in CRC incidence. To address this issue, we have developed the Adenoma and Serrated pathway to Colorectal CAncer (ASCCA) model that includes the adenoma‐carcinoma pathway and the serrated pathway to CRC as well as characteristics of colorectal lesions. The model structure and the calibration procedure are described in detail. Calibration resulted in 19 parameter sets for the adenoma‐carcinoma pathway and 13 for the serrated pathway that match the age‐ and sex‐specific adenoma and serrated lesion prevalence in the COlonoscopy versus COlonography Screening (COCOS) trial, Dutch CRC incidence and mortality rates, and a number of other intermediate outcomes concerning characteristics of colorectal lesions. As an example, we simulated outcomes for a biennial fecal immunochemical test screening program and a hypothetical one‐time colonoscopy screening program. Inclusion of the serrated pathway influenced the predicted effectiveness of screening when serrated lesions are associated with lower screening test sensitivity or when they are not removed. To our knowledge, this is the first model that explicitly includes the serrated pathway and characteristics of colorectal lesions. It is suitable for the evaluation of the (cost)effectiveness of potential screening strategies for CRC. 相似文献
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Panos G. Georgopoulos Christopher J. Brinkerhoff Sastry Isukapalli Michael Dellarco Philip J. Landrigan Paul J. Lioy 《Risk analysis》2014,34(7):1299-1316
A challenge for large‐scale environmental health investigations such as the National Children's Study (NCS), is characterizing exposures to multiple, co‐occurring chemical agents with varying spatiotemporal concentrations and consequences modulated by biochemical, physiological, behavioral, socioeconomic, and environmental factors. Such investigations can benefit from systematic retrieval, analysis, and integration of diverse extant information on both contaminant patterns and exposure‐relevant factors. This requires development, evaluation, and deployment of informatics methods that support flexible access and analysis of multiattribute data across multiple spatiotemporal scales. A new “Tiered Exposure Ranking” (TiER) framework, developed to support various aspects of risk‐relevant exposure characterization, is described here, with examples demonstrating its application to the NCS. TiER utilizes advances in informatics computational methods, extant database content and availability, and integrative environmental/exposure/biological modeling to support both “discovery‐driven” and “hypothesis‐driven” analyses. “Tier 1” applications focus on “exposomic” pattern recognition for extracting information from multidimensional data sets, whereas second and higher tier applications utilize mechanistic models to develop risk‐relevant exposure metrics for populations and individuals. In this article, “tier 1” applications of TiER explore identification of potentially causative associations among risk factors, for prioritizing further studies, by considering publicly available demographic/socioeconomic, behavioral, and environmental data in relation to two health endpoints (preterm birth and low birth weight). A “tier 2” application develops estimates of pollutant mixture inhalation exposure indices for NCS counties, formulated to support risk characterization for these endpoints. Applications of TiER demonstrate the feasibility of developing risk‐relevant exposure characterizations for pollutants using extant environmental and demographic/socioeconomic data. 相似文献
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İlknur Aykurt Karlıbel Seyhan Dülger Meliha Kasapoğlu Aksoy Muhammet Güzelsoy Ali Rıza Türkoğlu Lale Altan 《The aging male》2019,22(2):109-115
Objective: This study aims to investigate the effect of smoking on sexual functions in AS patients.Patients and methods: A total of 67 male AS patients with a median age of 34 years (range: 18–57) reporting sexual activity at least for the past 4 weeks period were included. Patients were divided into smokers (Group 1, n?=?47) and non-smokers (Group 2, n?=?20). Fagerström test for nicotine dependence, smoking history, exhaled carbon monoxide test were recorded for smoking AS patients. Visual analogue scale (VAS), Bath Ankylosing Spondylitis Disease Activity Index (BASDAI), Bath Ankylosing Spondylitis Metrology Index (BASMI), Bath Ankylosing Spondylitis Functional Index (BASFI), Ankylosing Spondylitis Quality of Life (ASQoL), International Index of Erectile Function (IIEF), Beck Depression Inventory (BDI) were filled for both groups.Results: There was no significant difference between smokers and non-smokers in all evaluation parameters. BASMI scores were significantly lower in the mild dependency subgroup as compared to those with moderate or severe dependency (p?=?.005 and p?=?.007, respectively). Total IIEF score and IIEF categories correlated significantly with BASDAI, BASFI, BASMI, ASQoL, pain, fatigue, and cumulative smoking. BDI showed an inverse correlation with the IIEF score and IIEF category (p?.001 r?=?–0.520, p?.001 r?=?–0.508, respectively).Conclusions: Sexual function in AS patients is associated with the pain, fatigue, disease activity, functional status, quality of life, depression as well as the cumulative exposure to smoking, and that sexual functions tend to decline with increasing degree of cigarette dependency. 相似文献
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Heitor de Oliveira Duarte Enrique Lopez Droguett Márcio das Chagas Moura Elainne Christine de Souza Gomes Constança Barbosa Verônica Barbosa Moacyr Araújo 《Risk analysis》2014,34(5):831-846
We developed a stochastic model for quantitative risk assessment for the Schistosoma mansoni (SM) parasite, which causes an endemic disease of public concern. The model provides answers in a useful format for public health decisions, uses data and expert opinion, and can be applied to any landscape where the snail Biomphalaria glabrata is the main intermediate host (South and Central America, the Caribbean, and Africa). It incorporates several realistic and case‐specific features: stage‐structured parasite populations, periodic praziquantel (PZQ) drug treatment for humans, density dependence, extreme events (prolonged rainfall), site‐specific sanitation quality, environmental stochasticity, monthly rainfall variation, uncertainty in parameters, and spatial dynamics. We parameterize the model through a real‐world application in the district of Porto de Galinhas (PG), one of the main touristic destinations in Brazil, where previous studies identified four parasite populations within the metapopulation. The results provide a good approximation of the dynamics of the system and are in agreement with our field observations, i.e., the lack of basic infrastructure (sanitation level and health programs) makes PG a suitable habitat for the persistence and growth of a parasite metapopulation. We quantify the risk of SM metapopulation explosion and quasi‐extinction and the time to metapopulation explosion and quasi‐extinction. We evaluate the sensitivity of the results under varying scenarios of future periodic PZQ treatment (based on the Brazilian Ministry of Health's plan) and sanitation quality. We conclude that the plan might be useful to slow SM metapopulation growth but not to control it. Additional investments in better sanitation are necessary. 相似文献
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