Comparative Effectiveness of Treatments for Acute Myeloid Leukemia in the Elderly

老年人急性髓系白血病治疗的比较疗效

• 批准号: 8693973
• 负责人: Rong Wang
• 金额: $ 8.08万
• 依托单位: YALE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2013
• 资助国家: 美国
• 起止时间: 2013-07-02 至 2015-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8693973
• 关键词: Acute Myelocytic Leukemia; Address; Affect; Age; Aging; Area; Attention; Blast Cell Proliferation; Bone Marrow Cells; Caring; Characteristics; Clinical; Clinical effectiveness; Comorbidity; Data; Diagnosis; Disease; Economic Burden; Effectiveness; Elderly; Equilibrium; Expenditure; Grant; Health Policy; Hospitalization; Individual; Institution; Length; Link; Measures; Medical; Medicare; Medicare claim; Myelogenous; Observational Study; Outcome; Patients; Physicians; Population; Randomized; Randomized Clinical Trials; Reporting; SEER Program; Series; Supportive care; Time; Treatment Cost; United States; aged; cancer epidemiology; chemotherapy; clinical practice; cohort; comparative effectiveness; comparison group; cost; cost effectiveness; design; disease characteristic; effective therapy; experience; improved; leukemia; older patient; policy implication; population based; programs; public health relevance; randomized trial; response; standard care

DESCRIPTION (provided by applicant): Acute myeloid leukemia (AML) is characterized by arrest of differentiation in the myeloid lineage and an over proliferation of blast cells. It is th most common type of leukemia in the United States and is primarily a disease of the elderly. Compared with their younger counterparts, elderly AML patients (age e 65 years) have worse outcome, with a median survival far less than one year. The poorer survival in the elderly has been attributed to less effective therapy, more comorbidities, and other patient characteristics. Although intensive chemotherapy is a standard treatment for younger AML patients, whether intensive or low-intensive chemotherapy will benefit elderly AML patients is not established. To date, most studies evaluating the effects of chemotherapy in elderly AML patients have been limited to small patient series from one or few clinical institutions. Medicare expenditure for elderly AML patients has steadily increased over recent years. The cost for elderly AML patients who received chemotherapy was almost three times higher than those who did not. However, there is no existing study comparing the cost-effectiveness of intensive chemotherapy versus low- intensive chemotherapy for the treatment of elderly AML patients. In the proposed study, we will assemble a cohort of approximately 5,000 elderly AML patients who were diagnosed in the Surveillance, Epidemiology and End Results program area during 2005-2009 and follow the medical care they received through the end of 2010. We will evaluate the comparative effectiveness of intensive and low-intensive chemotherapy for the treatment of elderly AML patients in two aspects, i.e., the clinical effectiveness and the cost-effectiveness. The clinical effectiveness of intensive and low-intensive treatment will be measured by 8-week and 1-year survival, and the cost-effectiveness will be measured by the length of survival, quality adjusted survival, and incremental cost-effectiveness ratio. Given the aging of the population and the continuous rise in Medicare expenditure, findings from the proposed study will not only provide valuable information for physicians and patients to choose treatment options, but also have significant health policy implications.

描述（由申请人提供）：急性髓系白血病（AML）的特征是髓系分化停滞和母细胞过度增殖。它是美国最常见的白血病类型，主要是老年人的疾病。与年轻患者相比，老年 AML 患者（年龄 65 岁）的预后较差，中位生存期远低于一年。老年人的生存率较差是由于治疗效果较差、合并症较多以及其他患者特征所致。尽管强化化疗是年轻 AML 患者的标准治疗，但强化化疗或低强度化疗是否有益于老年 AML 患者尚不清楚。迄今为止，大多数评估化疗对老年 AML 患者效果的研究仅限于来自一个或少数临床机构的小规模患者系列。近年来，老年 AML 患者的医疗保险支出稳步增加。接受化疗的老年 AML 患者的费用几乎是未接受化疗的患者的三倍。然而，目前尚无研究比较强化化疗与低强度化疗治疗老年 AML 患者的成本效益。在拟议的研究中，我们将收集2005-2009年间在监测、流行病学和最终结果项目领域诊断的约5,000名老年AML患者组成的队列，并跟踪他们截至2010年底接受的医疗护理。我们将从临床效果和成本效益两个方面评估强化和低强度化疗治疗老年AML患者的比较效果。强化和低强化治疗的临床效果将通过8周和1年生存率来衡量，成本效益将通过生存时间、质量调整生存率和增量成本效益比来衡量。鉴于人口老龄化和医疗保险支出的持续增长，拟议研究的结果不仅将为医生和患者选择治疗方案提供有价值的信息，而且对卫生政策也具有重大影响。