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: 8583443
• 负责人: Rong Wang
• 金额: $ 8.33万
• 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/8583443
• 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）：急性髓细胞性白血病（AML）的特征是髓样谱系中的分化和爆炸细胞的过度增殖。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（E 65岁）相比，其中位生存期的中位数远不到一年。老年人的生存率较差，归因于效率较低的治疗，更多的合并症和其他患者特征。尽管强化化疗是针对年轻AML患者的标准治疗方法，但无论是强化还是低密集型化疗将使老年AML患者受益。迄今为止，大多数评估化学疗法对老年AML患者的影响的研究仅限于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。接受化学疗法的老年AML患者的费用几乎是那些未接受化学疗法的成本。但是，没有研究比较对老年AML患者治疗的强化化疗与低强化化疗的成本效益。在拟议的研究中，我们将组装大约5,000名老年AML患者的队列，这些患者在2005年至2009年期间被诊断出在监视，流行病学和最终结果方案领域，并遵循他们在2010年底获得的医疗服务。我们将评估两种方面的效率和高度疗法的强度效率和低密度化学疗法的比较效率，以及在两方面的效力，以及该方面的效率，以及该方面的效率，即，即效应。密集和低密集型治疗的临床有效性将通过8周和1年的生存率来衡量，成本效益将通过生存时间，质量调整后的生存期和增量的成本效益比来衡量。鉴于人口老龄化以及医疗保险支出的不断增加，拟议的研究的发现不仅将为医生和患者选择治疗方案提供宝贵的信息，而且还具有重大的健康政策影响。