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本文引用的文献

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2
N-of-1 Trial of a Statin, Placebo, or No Treatment to Assess Side Effects.一项比较他汀类药物、安慰剂或不治疗以评估副作用的单病例试验。
N Engl J Med. 2020 Nov 26;383(22):2182-2184. doi: 10.1056/NEJMc2031173. Epub 2020 Nov 15.
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How personalised medicine will transform healthcare by 2030: the ICPerMed vision.到 2030 年,个性化医疗将如何改变医疗保健:ICPerMed 的愿景。
J Transl Med. 2020 Apr 28;18(1):180. doi: 10.1186/s12967-020-02316-w.
4
Cardiovascular effect of discontinuing statins for primary prevention at the age of 75 years: a nationwide population-based cohort study in France.75 岁时停止他汀类药物用于一级预防对心血管的影响:法国一项全国范围内基于人群的队列研究。
Eur Heart J. 2019 Nov 14;40(43):3516-3525. doi: 10.1093/eurheartj/ehz458.
5
Addressing Extreme Propensity Scores via the Overlap Weights.通过重叠权重解决极端倾向评分。
Am J Epidemiol. 2019 Jan 1;188(1):250-257. doi: 10.1093/aje/kwy201.
6
Using simulation and optimization approach to improve outcome through warfarin precision treatment.
Pac Symp Biocomput. 2018;23:412-423.
7
Personalized medicine could transform healthcare.个性化医疗可能会改变医疗保健。
Biomed Rep. 2017 Jul;7(1):3-5. doi: 10.3892/br.2017.922. Epub 2017 Jun 2.
8
Optimal decision support rules improve personalize warfarin treatment outcomes.
Annu Int Conf IEEE Eng Med Biol Soc. 2016 Aug;2016:2594-2597. doi: 10.1109/EMBC.2016.7591261.
9
A systematic review of factors that contribute to nocebo effects.对导致反安慰剂效应的因素的系统评价。
Health Psychol. 2016 Dec;35(12):1334-1355. doi: 10.1037/hea0000416. Epub 2016 Sep 22.
10
Statin-Associated Side Effects.他汀类药物相关副作用。
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多目标下个体化他汀类药物治疗处方的决策规则。

Decision rules for personalized statin treatment prescriptions over multi-objectives.

机构信息

Institute for Health Informatics, University of Minnesota, Minneapolis, MN 55455, USA.

Department of Pharmaceutical Care & Health Systems, University of Minnesota, Minneapolis, MN 55455, USA.

出版信息

Exp Biol Med (Maywood). 2023 Dec;248(24):2526-2537. doi: 10.1177/15353702231220660. Epub 2024 Jan 27.

DOI:10.1177/15353702231220660
PMID:38281069
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10854472/
Abstract

In our previous study, we demonstrated the feasibility of producing a proactive statin prescription strategy - a personalized statin treatment plan (PSTP) - using neural networks with big data. However, its non-transparency limited result interpretations and clinical usability. To improve the transparency of our previous approach with minimal compromise to the maximal statin treatment benefit-to-risk ratio, this study proposed a five-step pipeline approach called the decision rules for statin treatment (DRST). Steps 1-3 of our proposed pipeline improved our previous PSTP model in optimizing individual benefit-to-risk ratio; Step 4 used a decision tree model (DRST) to provide straightforward rules in the initial statin treatment plan; Step 5 aimed to evaluate the efficacy of these decision rules by conducting a clinical trial simulation. We included 107,739 de-identified patient data from Optum Labs Database Warehouse in this study. The final decision rules were compact and efficient, resulting from a decision tree with only a maximum depth of 3 and 11 nodes. The DRST identified three factors that are easily obtainable at the point of care: age, low-density lipoprotein cholesterol (LDL-C) level, and age-adjusted Charlson score. Moreover, it also identified six subpopulations that can benefit most from these decision rules. In our clinical trial simulations, DRST was found to improve statin benefit in LDL-C reduction by 4.15 percentage points (pp) and reduce risks of statin-associated symptoms (SAS) and statin discontinuation by 11.71 and 3.96 pp, respectively, when compared to the standard of care. Moreover, these DRST results were only less than 0.6 pp suboptimal to PSTP, demonstrating that building DRST that provide transparency with minimal compromise to the maximal benefit-to-risk ratio of statin treatments is feasible.

摘要

在我们之前的研究中,我们展示了使用神经网络和大数据生成主动他汀类药物处方策略——个性化他汀类药物治疗计划 (PSTP) 的可行性。然而,它的不透明性限制了结果解释和临床可用性。为了提高我们之前方法的透明度,同时最大限度地提高他汀类药物治疗的效益风险比,本研究提出了一种称为他汀类药物治疗决策规则 (DRST) 的五步流程方法。我们提出的方法的步骤 1-3 改进了我们之前的 PSTP 模型,以优化个体效益风险比;步骤 4 使用决策树模型 (DRST) 提供初始他汀类药物治疗计划中的直接规则;步骤 5 旨在通过临床试验模拟评估这些决策规则的疗效。我们在这项研究中纳入了来自 Optum Labs Database Warehouse 的 107739 名去识别患者数据。最终的决策规则是紧凑高效的,这是由于决策树的最大深度只有 3 层和 11 个节点。DRST 确定了三个在护理点很容易获得的因素:年龄、低密度脂蛋白胆固醇 (LDL-C) 水平和年龄调整 Charlson 评分。此外,它还确定了六个最能从这些决策规则中受益的亚组。在我们的临床试验模拟中,与标准治疗相比,DRST 被发现可以提高 LDL-C 降低 4.15 个百分点 (pp) 的他汀类药物效益,并降低他汀类药物相关症状 (SAS) 和他汀类药物停药的风险分别为 11.71 和 3.96 pp。此外,这些 DRST 结果仅比 PSTP 差不到 0.6 pp,表明构建提供透明度且对他汀类药物治疗的最大效益风险比的最小折衷的 DRST 是可行的。