Decision Models to Compare Treatments in Older Patients with AML

比较老年 AML 患者治疗方法的决策模型

• 批准号: 8577297
• 负责人: MARTINE EXTERMANN
• 金额: $ 35.62万
• 依托单位: H. LEE MOFFITT CANCER CTR & RES INST
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-09-18 至 2017-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8577297
• 关键词: Acute Myelocytic Leukemia; Adjuvant; Affect; Age; Agreement; Area; Benefits and Risks; Biological; Calibration; Characteristics; Clinical; Clinical Trials; Comorbidity; Computer software; Cytogenetics; Data; Databases; Decision Aid; Decision Analysis; Decision Making; Decision Modeling; Discrimination; Dose; Effectiveness; Elderly; Equilibrium; Foundations; Geographic Locations; Goals; Hematology; In complete remission; Life; Literature; Living Costs; Malignant - descriptor; Markov Chains; Medicare; Meta-Analysis; Modeling; Outcome; Patients; Patterns of Care; Population; Process; Quality of life; Regimen; Research; Review Literature; Risk; Sampling; Solid; Subgroup; Supportive care; Testing; Time; Treatment Effectiveness; Underserved Population; aged; base; cancer care; care delivery; chemotherapy; cohort; comparative effectiveness; compare effectiveness; cost effectiveness; design; evidence base; functional status; improved; malignant breast neoplasm; model design; models and simulation; mortality; older patient; patient population; preference; prognostic; programs; prospective; public health relevance; systematic review; tool; treatment strategy

DESCRIPTION (provided by applicant): Eighty percent of acute myelogenous leukemias (AML) occur beyond the age of 60, 35% occur beyond the age of 75, and 10% beyond the age of 85. Between 1975 and 2001, the 5-year survival of younger AML patients has more than doubled. Yet the survival of patients above the age of 65 remains dismal, with no progress over that period. Even pending the emergence of new strategies to target the biological challenges of AML in the elderly, significant progress could be made by optimizing the choices of treatment strategies. For example, in the Swedish population, AML patients aged 70-79 years who live in an area where a higher proportion are treated with intensive approach have a 2-year survival of 20% instead of 7%[1]. Also, for poor risk patients, new alternate strategies are emerging, such as hypomethylating agents, that might offer better outcomes than intensive induction chemotherapy. Therefore, we hypothesize that decision models which allow comparing treatment options more objectively might result in improved treatment decisions and outcomes for this population of patients who are underrepresented in clinical trials. Such an approach has been successful in breast cancer, with programs such as Adjuvant!Online. Aims: In this project we plan to design decision models to compare effectiveness and early risks of different treatment approaches for AML in patients older than age 70, and to test them on a sample of patients from the Moffitt Total Cancer Care/Malignant hematology database. Approach: We will first conduct a systematic review of the literature for articles focusing on the treatment of AML i the elderly, and conduct meta-analyses of the results. After that, we will develop decision models using TreeAge Pro. This software creates micro simulation models using Markov processes to model the outcomes of AML and its treatment over time. We will focus on the following outcomes: 1-year survival, 2-year survival, 1- month mortality, and complete response rates. The approaches compared will be: intensive induction chemotherapy, low-dose chemotherapy, hypomethylating agents, and supportive care. We will control for the impact of age, cytogenetics, functional status and comorbidity level. Once the models are developed, we will test them in Moffitt's Total Cancer Care/malignanthematology database, which is a large prospective database that will provide about 900 AML patients above the age of 70 for this study. We hypothesize that our models will correlate well with the outcome of patients in the database and accurately order the benefits of the treatment choices for various prognostic subgroups. Significance: If successful, this project will provide the first available decision mode for evaluating the outcomes of AML in patients aged 70 and above. Such a model could then be tested as a clinical decision aid, as a tool for comparative effectiveness, quality of life, and cot-effectiveness studies and might contribute in the longer term to improved outcomes for this underserved population.

描述（由申请人提供）：80％的急性骨髓性白血病（AML）发生在60岁以上，35％发生在75岁以上，超过85岁以上的10％。在1975年至2001年之间，年轻AML患者的5年生存率增加了一倍以上。然而，65岁以上的患者的存活仍然令人沮丧，在此期间没有进展。即使是在针对AML在老年人中的生物学挑战的新策略的出现之后，也可以通过优化治疗策略的选择来取得重大进展。例如，在瑞典人口中，生活在以较高比例治疗的地区的70-79岁的AML患者的2年生存率为20％，而不是7％[1]。同样，对于风险较差的患者，新的替代策略正在出现，例如降压药，可能比密集诱导化疗更好。因此，我们假设允许更客观地比较治疗方案的决策模型可能会改善治疗决策和对临床试验中人数不足的患者人群的结果。这种方法在乳腺癌方面取得了成功，辅助！目的：在这个项目中，我们计划设计决策模型，以比较70岁以下患者的AML的有效性和早期治疗方法的早期风险，并对来自Moffitt Total Cancer Care/allignants血液学数据库的患者样本进行测试。方法：我们将首先对文献进行系统的综述，以针对重点介绍AML I老年人的文章，并进行结果的荟萃分析。之后，我们将使用Treage Pro开发决策模型。该软件使用Markov工艺创建微型仿真模型，以建模AML及其治疗的结果。我们将重点关注以下结果：1年生存，2年生存，1个月的死亡率和完整的应答率。这些方法将是：密集诱导化疗，低剂量化疗，低甲基化剂和支持性护理。我们将控制年龄，细胞遗传学，功能状态和合并症水平的影响。一旦开发了模型，我们将在莫菲特的总癌症护理/恶性肿瘤数据库中对其进行测试，该数据库是一个很大的前瞻性数据库，可为本研究提供约900名AML患者。我们假设我们的模型将与数据库中患者的结果很好地相关，并准确订购了各种预后亚组的治疗选择的好处。意义：如果成功，该项目将提供第一个可用的决策模式，以评估70岁及以上患者的AML结果。然后，可以将这种模型作为临床决策辅助，作为比较有效性，生活质量和COT效应研究的工具，并可能在长期内做出贡献，以改善服务不足的人群的结果。