Measuring Lifelong Multimorbidity with Patient Perspectives: Implementing and Visualizing the Cumulative Burden Methodology Across Cohorts

项目总结/摘要 背景：多发性硬化症，定义为同时发生两种或两种以上的身体或精神慢性疾病， 正在全球范围内流行并不断上升。新的多模态测量方法提供了对频谱的独特见解， 需要多种共病状况的模式和程度。目前的措施未能提供足够的 从设计，评估和实施多层次的临床相关干预措施的背景。 目的：为了响应PAR-20-179，我们建议开发、可视化和应用一套新的模块化套件， 采取措施和工具，以解决目前影响多阶段成果研究的关键限制因素。 具体目标：（1）在生命过程的各个阶段制定个性化的累积负担措施， 基于患者的观点，使用条件加权的负担权重和风险因素;（2）创建一套 基于网络的可视化供任何类型的队列使用;（3）将累积负担应用于独立的 镰状细胞病患者队列，以测试实施情况并指导未来向其他队列的传播。 研究设计：两个新的措施，这是建立在我们现有的累积负担框架，将 使用圣犹达终身队列（SJLIFE）研究的数据开发，这是一项大型儿科临床队列研究， 癌症幸存者首先，我们将开发一个个性化的措施，多发病风险的个人使用 泊松过程的回归框架，以提供对模式终身变化的见解， 多种慢性疾病的严重程度和相关的关键风险因素。二是落实负担 权重，一组特定于条件的权重，来自效用权重（我们也将计算），使用 SJLIFE数据，以纳入患者观点。要使用/共享度量标准， 将利用软件开发来制作动态的、交互式的基于网络的可视化。最后利用 实施研究的综合框架，我们将应用累积负担法进行研究 在第二个由患有镰状细胞病的成年人和儿童组成的队列中进行多项研究， 国家研究优先事项，并研究其他国家如何将我们的创新措施应用于他们的同龄人。 意义：我们的累积负担措施是为了克服一些紧迫的困难， PAR-20-179确定的当前多项措施。这项拟议的研究旨在建立在我们以前的基础上， 通过结合个性化的风险预测和患者报告的结果来工作。此外，我们认识到， 制定新的措施和公布我们的方法不足以确保广泛使用和影响。到 为了支持有效的实施和传播，我们将创建可视化工具，帮助调查人员共享 累积的负担结果，并研究其应用的障碍和使能因素， 科学方法。我们预计我们的工作将产生一套可视化模板和模块化应用 方法资源，使临床医生，研究人员，倡导团体和政策制定者有机会 揭示日益严重的影响到我们社会几乎每一个亚群体的多重犯罪问题。

PROJECT SUMMARY/ABSTRACT Background: Multimorbidity, defined as the co-occurrence of two or more physical or mental chronic illnesses, is prevalent and rising globally. New measures of multimorbidity that provide unique insights into the spectrum, pattern and magnitude of multiple co-morbid conditions are needed. Current measures fail to provide adequate context from which to design, evaluate, and implement multilevel clinically-relevant interventions. Objective: In response to PAR-20-179, we propose to develop, visualize and apply a modular suite of novel measures and tools to address the critical limitations currently impacting multimorbidity outcomes research. Specific Aims: (1) Develop personalized cumulative burden measures, over various stages of the life-course, based on patient perspectives using condition-weighted burden weights and risk factors; (2) Create a suite of web-based visualizations for use by any type of cohort; (3) Apply the cumulative burden to an independent cohort of sickle cell disease patients to test implementation and guide future dissemination to other cohorts. Study design: Two novel measures, which built on our existing cumulative burden framework, will be developed using data from the St. Jude Lifetime Cohort (SJLIFE) study, a large clinical cohort of pediatric cancer survivors. First, we will develop a personalized measure of multimorbidity risk for an individual using a regression framework of the Poisson process to provide insights into lifelong variations in patterns and magnitude of multiple chronic conditions and associated key risk factors. Second, we will implement burden weights, a set of condition-specific weights derived from utility weights (which we will also calculate) using SJLIFE data in order to incorporate patient perspectives. To use/share the metrics, the Agile approach to software development will be employed to create dynamic, interactive web-based visualizations. Finally, using the consolidated framework for implementation research, we will apply the cumulative burden method to study multimorbidity in a second cohort comprised of adults and children with sickle cell disease in order to advance national research priorities and study how others may our innovative measures to their cohorts. Significance: Our cumulative burden measure was created to overcome some of the pressing difficulties with current multimorbidity measures identified by PAR-20-179. This proposed study seeks to build on our prior work by incorporating personalized risk prediction and patient-reported outcomes. Additionally, we recognize that developing new measures and publishing our methods are insufficient to ensure broad use and impact. To support effective implementation and dissemination, we will create visual tools that help investigators share cumulative burden results and study the barriers and enablers of their application using an implementation science approach. We anticipate our work will yield a suite of visualization templates and modular applied method resources that will enable clinicians, researchers, advocacy groups, and policymakers an opportunity to shed light on the growing problem of multimorbidity affecting nearly every sub-group of our society.