Comparative Effectiveness of Treatments for Acute Myeloid Leukemia in the Elderly

描述（由申请人提供）：急性髓性白血病（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。与年轻的AML患者相比，老年AML患者（年龄65岁）的预后更差，中位生存期远低于1年。老年人的生存率较差归因于治疗效果较差、合并症较多和其他患者特征。虽然强化化疗是年轻AML患者的标准治疗，但强化或低强度化疗是否会使老年AML患者受益尚未确定。迄今为止，大多数评价老年AML患者化疗效果的研究仅限于来自一个或几个临床机构的小型患者系列。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患者的三倍。然而，目前还没有比较老年AML患者强化化疗与低强度化疗的成本效益的研究。在拟议的研究中，我们将收集一个队列，其中约有5,000名老年AML患者，他们在2005-2009年期间在监测、流行病学和最终结果项目中被诊断出来，并随访他们在2010年底之前接受的医疗护理。我们将从两个方面评估老年AML患者强化和低强度化疗的比较有效性，即，临床效果和成本效益。强化和低强化治疗的临床有效性将通过8周和1年生存期来衡量，成本效果将通过生存期、质量调整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意义。

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.