Comparative modeling of multiple myeloma across myeloma control continuuum: prevention, treatment, and disparity reduction

跨骨髓瘤控制连续体的多发性骨髓瘤比较模型：预防、治疗和缩小差异

• 批准号: 10491296
• 负责人: GRAHAM A. COLDITZ
• 金额: $ 64.38万
• 依托单位: WASHINGTON UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2021
• 资助国家: 美国
• 起止时间: 2021-09-20 至 2026-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10491296
• 关键词: Adult; African American population; Area; Breast; Cancer Intervention and Surveillance Modeling Network; Cessation of life; Clinical Trials; Colorectal Cancer; Continuity of Patient Care; Development; Diagnosis; Disease; Economic Burden; Evaluation; Goals; Guidelines; Hematologic Neoplasms; Incidence; Incubators; Intervention; Life; Modeling; Monoclonal gammopathy of uncertain significance; Multiple Birth Offspring; Multiple Myeloma; Neoplasms; Obesity; Patients; Policies; Precancerous Conditions; Precision therapeutics; Prevention; Prevention strategy; Regimen; Reporting; Research; Risk; Route; Solid Neoplasm; Treatment Protocols; Universities; Washington; base; cancer care; cancer site; caucasian American; clinical practice; cohort; comparative; cost; cost-effectiveness evaluation; disease natural history; disparity reduction; evidence base; flexibility; health disparity; health economics; high risk; improved; innovation; malignant breast neoplasm; novel; novel strategies; population based; premalignant; prevent; process optimization; programs; racial disparity; screening; survival outcome; survivorship; treatment disparity; trend

PROJECT SUMMARY/ABSTRACT Multiple myeloma (MM) is a common and lethal hematologic malignancy. Treatments of MM have been rapidly evolving. While these new treatments improve survival considerably, the median survival still ranges from 43- 83 months at diagnosis. Among all cancer sites, the management of MM is the most costly, which in part can be attributable to guideline recommended multidrug regimens. Despite such significant health and economic burdens and rapid changing landscape for MM treatments, MM is not one of the cancer sites in the Cancer Intervention and Surveillance Modeling Network (CISNET). Therefore, MM lacks comparative modeling to set goals and policy prioritization in MM prevention and control. Moreover, unlike breast cancer or colorectal cancer, there exists no population-based screening for MM or risk managed strategies for those with premalignant conditions (MGUS and smoldering MM). MM requires comparative modeling to evaluate promising intervention strategies, particularly at premalignant stages. To prevent/control this devastating disease, it is imperative to demonstrate the potentials of these interventions before implementation. Moreover, marked racial disparities in MM (both incidence and survival) is long-established. Without any value-based strategies for prevention and treatment, MM health disparities will continue to worsen. This Incubator Program will include two modeling groups to conduct comparative modeling under the coordination of the coordinating center. Our Program will evaluate novel strategies in preventing or treating MM with the goals of reducing the burden of MM and mitigating MM disparities. We plan to comparatively build, calibrate, and validate evidence- based MM modeling across the MM care continuum (Aim 1). Using the proposed comparatively modeling, we will (1) assess the impacts of novel MM prevention strategies in high-risk patients diagnosed with MGUS (Aim 2); (2) evaluate the cost-effectiveness of novel treatment regimens as well as guideline-recommended treatments in patients diagnosed with MM (Aim 3); and (3) assess whether, under what conditions, and in which ways the goal of eliminating racial disparities can be achieved through the proposed novel intervention strategies (Aim 4). The proposed MM Incubator Program is significant in its capability to 1) build evidence- based comparative modeling for MM, a disease area that lacks of such modeling, relative to the areas of solid tumors already with such modeling, to guide interventions and policies; 2) provide evidence-based evaluation before implementation of any costly clinical trial; 3) explore novel interventions/treatments at various stages of MM; and 4) examine the value of guideline-recommended therapies, providing evidence to inform changes in guidelines and thus a shift in current clinical practice of MGUS and MM management. The proposed intervention strategies for MGUS and MM patients are innovative, with the goals to prevent and control MM and reduce MM disparities. Successful completion of this study will provide evidence in tangible metrics to urge a paradigm shift from current MGUS/MM management. It is therefore a vital step to move the field forward.

项目总结/摘要 多发性骨髓瘤（MM）是一种常见的恶性血液病。MM的治疗已经迅速 进化虽然这些新的治疗方法大大提高了生存率，但中位生存率仍在43- 83个月诊断在所有癌症部位中，MM的管理是最昂贵的，这在一定程度上可以 可归因于指南推荐的多药治疗方案。尽管如此，健康和经济 由于MM治疗的负担和快速变化的景观，MM不是癌症中的癌症部位之一， 干预和监测建模网络（CISNET）。因此，MM缺乏比较建模来设置 在MM预防和控制方面的目标和政策优先次序。此外，与乳腺癌或结肠直肠癌不同， 癌症，目前还没有基于人群的MM筛查或针对患有MM的患者的风险管理策略。 癌前病变（MGUS和阴燃MM）。MM需要比较建模来评估 有希望的干预策略，特别是在癌前阶段。为了防止/控制这种毁灭性的 因此，在实施这些干预措施之前，必须证明这些干预措施的潜力。此外，委员会认为， MM的明显种族差异（发病率和生存率）早已确立。没有任何价值基础 随着预防和治疗战略的实施，MM的健康差距将继续恶化。孵化器计划 将包括两个模型组，在协调员的协调下进行比较模型 中心我们的项目将评估预防或治疗MM的新策略，目的是减少MM的发病率。 减轻MM的负担和减轻MM的差异。我们计划建立，校正，和验证证据- 基于MM护理连续体的MM建模（目标1）。使用所提出的比较建模，我们 将（1）评估新型MM预防策略对诊断为MGUS的高危患者的影响（Aim 2）;（2）评价新治疗方案的成本-效果以及指南推荐的 诊断为MM的患者的治疗（目标3）;以及（3）评估是否，在什么条件下， 通过拟议的新干预措施，消除种族差异的目标可以通过哪些方式实现 战略（目标4）。拟议的MM孵化器计划的重要性在于：1）建立证据- 基于MM的比较建模，相对于实体瘤的区域， 肿瘤已经与这样的建模，以指导干预措施和政策; 2）提供循证评估 在实施任何昂贵的临床试验之前; 3）在不同阶段探索新的干预措施/治疗方法， MM;以及4）检查指南推荐的治疗的价值，提供证据以告知 指南，从而改变了MGUS和MM管理的当前临床实践。拟议 MGUS和MM患者的干预策略是创新的，其目标是预防和控制MM 并减少MM差异。这项研究的成功完成将提供有形指标的证据，以敦促 从目前的MGUS/MM管理模式转变。因此，这是推动该领域向前发展的重要一步。