技术背景“一种精确的医学方法，为患有膝关节骨关节炎的超重和肥胖成人开发和内部优化的最佳运动和减肥治疗”
Technical Background for "A Precision Medicine Approach to Develop and Internally Validate Optimal Exercise and Weight Loss Treatments for Overweight and Obese Adults with Knee Osteoarthritis"
摘要：
我们在“为超重和肥胖成人膝关节骨关节炎患者开发和内部优化最佳运动和减肥治疗的精准医学方法”（Jiang et al. 2020）中为膝关节骨关节炎临床分析中开发的方法提供了额外的统计背景。Jiang et al. 2020提出了一种管道，用于使用精确医学模型学习最佳治疗规则，并使用Z检验将其与零阶模型进行比较。模型性能基于价值函数，这是一个预测每个决策规则未来回报的标量。折叠刀（即，采用留一交叉验证法估计IDEA中多个结果的价值函数及其方差。IDEA是一项随机临床试验，研究了三种干预措施（运动（E），饮食减肥（D）和D+E）对超重和肥胖的膝关节骨关节炎参与者的影响。在本报告中，我们将通过额外的统计背景来扩展讨论和论证。我们详细阐述了精确医学的背景，价值函数的刀切估计及其估计方差的推导，刀切估计的一致性，以及额外的模拟结果，反映了更多的刀切估计的性能。我们建议阅读Jiang et al. 2020，以了解膝关节骨关节炎最佳ITR的临床应用和解释以及对管道的整体理解，并建议使用本文了解基础统计学推导和方法。
英文摘要：
We provide additional statistical background for the methodology developed in the clinical analysis of knee osteoarthritis in "A Precision Medicine Approach to Develop and Internally Validate Optimal Exercise and Weight Loss Treatments for Overweight and Obese Adults with Knee Osteoarthritis" (Jiang et al. 2020). Jiang et al. 2020 proposed a pipeline to learn optimal treatment rules with precision medicine models and compared them with zero-order models with a Z-test. The model performance was based on value functions, a scalar that predicts the future reward of each decision rule. The jackknife (i.e., leave-one-out cross validation) method was applied to estimate the value function and its variance of several outcomes in IDEA. IDEA is a randomized clinical trial studying three interventions (exercise (E), dietary weight loss (D), and D+E) on overweight and obese participants with knee osteoarthritis. In this report, we expand the discussion and justification with additional statistical background. We elaborate more on the background of precision medicine, the derivation of the jackknife estimator of value function and its estimated variance, the consistency property of jackknife estimator, as well as additional simulation results that reflect more of the performance of jackknife estimators. We recommend reading Jiang et al. 2020 for clinical application and interpretation of the optimal ITR of knee osteoarthritis as well as the overall understanding of the pipeline and recommend using this article to understand the underlying statistical derivation and methodology.
机构、总结、中文摘要均由AI分析而来，可能存在错误，请以论文正文为准。
