Myelodysplastic Syndromes: Patterns of Care and Outcomes

描述(申请人提供)：骨髓增生异常综合征(MDS)是一组克隆性增殖性骨髓疾病，导致骨髓生成障碍和外周血细胞减少。基于人群的癌症登记数据表明，美国每年新诊断的MDS病例超过1万例。80%的MDS病例在确诊时年龄在65岁或以上，观察到的三年存活率只有35%。国会已确定MDS研究不足，并敦促国家癌症研究所和其他联邦机构进一步开展MDS研究。SEER-Medicare关联数据库的可获得性为研究MDS患者的护理模式和结果提供了一个独特的机会，这两者都鲜为人知。到2008年底，SEER-Medicare数据库将包括2001-2005年间确诊的MDS患者的SEER数据，以及截至2007年底的这些患者的Medicare索赔信息。我们建议使用这些数据来(1)评估护理模式，特别是MDS患者在确诊后接受的骨髓检查的数量以及各种治疗策略的使用，包括最近批准的DNA去甲基化药物阿扎替丁和地西他滨；(2)在考虑并存情况后确定影响MDS患者生存的因素；以及(3)估计与MDS患者治疗相关的直接医疗成本。预计这项研究将包括大约6716名被诊断为66岁或以上的MDS患者的基于人群的队列，以及在年龄、性别、种族、SEER地区和其他疾病方面与患者匹配的非癌症对照。对于MDS患者，将评估骨髓检查的频率和接受各种治疗(如输血、铁络合疗法、生长因子、化疗和去甲基化药物)的患者的百分比。我们还将评估护理模式是否受到以个人或社区为基础的人口和社会经济特征的影响。此外，我们将评估哪些因素/特征影响MDS患者的生存，在考虑到对预测MDS患者死亡率很重要的并存情况后。此外，我们将估计MDS患者从确诊到2007年底或死亡日期(以较早者为准)产生的医疗保险成本，并评估哪些患者特征会影响成本。通过比较MDS患者和在年龄、性别、种族、SEER地区和合并症方面与MDS患者匹配的非癌症对照组的成本，我们将估计可归因于MDS的医疗成本。所有分析将分别针对MDS作为一个组和主要MDS亚型进行。为了考虑到2006年联邦医疗保险D部分生效后某些治疗(例如增长因素)的承保范围可能发生的变化，我们将按两个不同的时间段进行分层分析，2001-2005(D部分之前)和2006-2007(D部分后)。这项拟议的研究将首次使用大量以人群为基础的MDS患者样本来评估针对并存条件调整的护理模式和MDS生存。它还将首次提供与MDS治疗有关的直接医疗费用的国家估计数。这项拟议研究的结果将对临床医生做出有关MDS患者的护理和治疗的决策有价值，并对政策制定者在分配卫生资源方面非常有帮助。公共卫生相关性：骨髓增生异常综合征(MDS)是一组未被充分研究的血液系统恶性肿瘤，最常见于65岁或以上的老年人。研究人员建议使用相关的癌症登记文件和大约6716名MDS患者的医疗保险索赔来研究护理和生存模式，并估计与MDS治疗相关的直接医疗成本。这项研究的结果将对照顾MDS患者的临床医生和分配卫生资源的政策制定者都有价值。

DESCRIPTION (provided by applicant): The myelodysplastic syndromes (MDS) are a group of clonal proliferative bone marrow disorders that result in dysmyelopoiesis and peripheral blood cytopenias. Population-based cancer registry data indicate that over 10,000 new cases of MDS are diagnosed in the US annually. Eighty percent of MDS cases are 65 years or older at the time of diagnosis, and observed three-year survival is only 35%. The Congress has identified MDS as being understudied and urged the National Cancer Institute and other federal agencies to further MDS research. The availability of the SEER-Medicare linked database provides a unique opportunity to study the patterns of care and outcomes in MDS patients, both of which are poorly understood. By the end of 2008, the SEER-Medicare database will include SEER data on MDS patients who were diagnosed during 2001 - 2005 and Medicare claims information on these patients up through the end of 2007. We propose to use these data to (1) assess the patterns of care, especially the number of bone marrow tests MDS patients received after diagnosis and the use of various treatment strategies, included the recently approved DNA demethylating agents, azacitidine and decitabine; (2) identify factors that influence the survival of MDS patients after accounting for comorbid conditions; and (3) estimate the direct medical costs related to the treatment of MDS patients. A population-based cohort of approximately 6,716 MDS patients diagnosed at 66 years or older are expected to be included in the study, so will non-cancer controls matched to patients on age, gender, race, SEER region, and comordid conditions. For MDS patients, the frequency of bone marrow tests and the percentage of patients receiving various treatments (e.g. blood transfusions, iron chelation therapy, growth factors, chemotherapy, and demethylating agents) will be evaluated. We will also assess whether the patterns of care are influence by individual or community-based demographic and socioeconomic characteristics. In addition, we will evaluate which factors/characteristics affect the survival of MDS patients, after taking into account comorbid conditions that are important in predicting mortality in MDS patients. Furthermore, we will estimate the Medicare costs incurred by MDS patients from diagnosis through the end of 2007, or date of death, whichever is earlier, and assess which patient characteristics influence costs. By comparing the costs of MDS patients with the costs of non-cancer controls who are matched to MDS patients on age, gender, race, SEER region, and comorbid conditions, we will estimate the medical costs that are attributable to MDS. All analyses will be conducted separately for MDS as one group and for major MDS subtypes. To account for potential changes in the coverage for certain treatments (e.g. growth factors) after Medicare Part D took effect in 2006, we will stratify analyses by two different time periods, 2001 - 2005 (Pre Part D) and 2006 - 2007 (Post Part D). The proposed study will be the first ever to use a large population-based sample of MDS patients to evaluate the patterns of care and MDS survival adjusting for comorbid conditions. It will also be the first to provide a national estimate of the direct medical costs related to the treatment of MDS. Findings from the proposed study will be valuable to clinicians making decisions about the care and treatment of MDS patients and very helpful to policy makers in the allocation of health resources. PUBLIC HEALTH RELEVANCE: The myelodysplastic syndromes (MDS), a group of understudied malignancies of the blood system, are most common in elderly people 65 years or older. The investigators propose to use linked cancer registry files and Medicare claims of about 6,716 MDS patients to study the patterns of care and survival and estimate the direct medical costs related to the treatment of MDS. Findings from the study will be valuable to both clinicians caring for MDS patients and policy maker allocating health resources.