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: 10331128
• 负责人: GRAHAM A. COLDITZ
• 金额: $ 66.37万
• 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/10331128
• 关键词: Adult; African American; 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; Population; 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.

项目摘要/摘要 多发性骨髓瘤（MM）是一种常见且致命的血液系统恶性肿瘤。 MM的治疗迅速 不断发展。尽管这些新疗法可大大提高生存率，但中位生存期仍然为43-- 诊断83个月。在所有癌症网站中，MM的管理是最昂贵的，部分可以 归因于建议的多药方案。尽管健康和经济如此重要 负担和MM治疗的快速变化景观，MM不是癌症的癌症部位之一 干预和监视建模网络（CISNET）。因此，MM缺乏对设置的比较建模 预防和控制中的目标和政策优先级。此外，与乳腺癌或结直肠癌不同 癌症，没有对MM或风险管理策略的基于人群的筛查 预先态度（MGUS和闷烧的毫米）。 MM需要比较建模来评估 有希望的干预策略，尤其是在初次阶段。防止/控制这种毁灭性的 疾病，必须在实施之前证明这些干预措施的潜力。而且， 长期以来，MM（发病率和生存）的种族差异明显。没有任何基于价值的 预防和治疗策略，MM健康差异将继续恶化。此孵化器程序 将包括两个建模组，以在协调的协调下进行比较建模 中心。我们的计划将评估预防或治疗MM的新型策略 MM负担和减轻MM差异。我们计划相对建立，校准和验证证据 - 基于MM CARE连续体的MM建模（AIM 1）。使用所提出的相对建模，我们 （1）评估新型MM预防策略对被诊断为MGU的高危患者的影响（AIM 2）; （2）评估新型治疗方案的成本效益以及指南申请 诊断为MM的患者的治疗（AIM 3）； （3）评估在什么条件下以及在 通过拟议的新颖干预，可以实现消除种族差异的目标 策略（目标4）。拟议的MM孵化器计划在其能力上具有重要的能力1）建立证据 - 相对于固体区域，基于MM的比较模型，该疾病区域缺乏这种模型 已经进行了这种建模的肿瘤，以指导干预措施和政策； 2）提供基于证据的评估 在实施任何昂贵的临床试验之前； 3）在各个阶段探索新颖的干预/治疗 毫米; 4）检查指南推荐疗法的价值，提供证据以告知变更 指南，因此MGU和MM管理的当前临床实践发生了变化。提议 MGU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 范式从当前的MGU/MM管理转变。因此，这是向前推动田地的重要步骤。