Comparative Effectiveness of Treatments for Acute Myeloid Leukemia in the Elderly

描述（由申请人提供）：急性髓系白血病（AML）的特点是髓系谱系分化受阻和母细胞过度增殖。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（65岁）的预后较差，中位生存期远少于1年。老年人较差的生存率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，但强化或低强化化疗是否有利于老年AML患者尚不确定。迄今为止，大多数评估化疗对老年AML患者影响的研究仅限于来自一个或几个临床机构的小患者系列。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化化疗治疗老年AML患者的成本-效果。在拟议的研究中，我们将收集一个大约5000名老年AML患者的队列，这些患者在2005-2009年期间在监测、流行病学和最终结果项目领域被诊断出来，并跟踪他们到2010年底接受的医疗护理。我们将从临床效果和成本-效果两个方面评价强化化疗和低强化化疗治疗老年AML患者的比较效果。以8周生存期和1年生存期衡量强化治疗和低强化治疗的临床疗效，以生存期长短、质量调整生存期、增量成本-效果比衡量成本-效果。鉴于人口老龄化和医疗保险支出的持续增长，本研究的结果不仅为医生和患者选择治疗方案提供了有价值的信息，而且具有重要的卫生政策意义。

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.