Comparative Effectiveness of Treatments for Acute Myeloid Leukemia in the Elderly

描述(申请人提供)：急性髓系白血病(AML)的特征是髓系细胞分化受阻和原始细胞过度增殖。它是美国最常见的白血病类型，主要是老年人的一种疾病。与年轻患者相比，老年AML患者(年龄65岁)预后更差，中位生存期远低于一年。老年人的存活率较低被归因于治疗效果较差、更多的合并症以及其他患者特征。虽然强化化疗是年轻急性髓细胞白血病患者的标准治疗方法，但强化化疗或低强度化疗是否会使老年急性髓细胞白血病患者受益尚未确定。到目前为止，大多数评估老年AML患者化疗效果的研究都局限于一个或几个临床机构的小患者系列。近年来，老年急性髓细胞白血病患者的医疗保险支出稳步增长。接受化疗的老年AML患者的费用几乎是没有接受化疗的患者的三倍。然而，目前还没有研究比较强化化疗和低强度化疗治疗老年AML患者的成本-效果。在这项拟议的研究中，我们将汇集2005-2009年间在监测、流行病学和最终结果计划领域确诊的大约5,000名老年AML患者，并跟踪他们在2010年底之前接受的医疗护理。我们将从临床疗效和成本效益两个方面评估强化化疗和低强度化疗治疗老年AML患者的比较效果。强化和低强度治疗的临床疗效将通过8周和1年的生存期来衡量，成本-效果将通过生存期、质量调整后的生存期和增量成本-效果比来衡量。鉴于人口老龄化和医疗保险支出的持续上升，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，而且还具有重大的卫生政策影响。

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.