Myelodysplastic Syndromes: Patterns of Care and Outcomes

描述（由申请人提供）：骨髓增生异常综合征（MDS）是一组克隆增殖性骨髓疾病，导致骨髓增生异常和外周血细胞减少。基于人群的癌症登记数据表明，在美国每年诊断出超过10,000例MDS新病例。80%的MDS患者在确诊时年龄在65岁或以上，观察到的三年生存率仅为35%。国会已经确定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研究MDS。SEER-Medicare关联数据库的可用性为研究MDS患者的护理模式和结果提供了独特的机会，这两者都知之甚少。到2008年底，SEER-Medicare数据库将包括2001年至2005年诊断的MDS患者的SEER数据，以及2007年底之前这些患者的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患者在诊断后接受骨髓检查的次数和各种治疗策略的使用，包括最近批准的DNA去甲基化药物、阿扎胞苷和地西他滨；(2)在考虑合并症后，确定影响MDS患者生存的因素；(3)估算与MDS患者治疗相关的直接医疗费用。预计该研究将纳入约6,716名诊断年龄在66岁或以上的MDS患者，因此非癌症对照将在年龄、性别、种族、SEER区域和合并症方面与患者相匹配。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、铁螯合治疗、生长因子、化疗和去甲基化药物）的患者百分比。我们还将评估护理模式是否受到个人或社区人口和社会经济特征的影响。此外，在考虑了对预测MDS患者死亡率很重要的合并症后，我们将评估哪些因素/特征影响MDS患者的生存。此外，我们将估计MDS患者从诊断到2007年底或死亡日期（以较早者为准）所产生的医疗保险费用，并评估哪些患者特征会影响成本。通过比较MDS患者的费用与与MDS患者在年龄、性别、种族、SEER区域和合并症方面相匹配的非癌症对照者的费用，我们将估计可归因于MDS的医疗费用。所有分析将分别对MDS作为一个组和主要MDS亚型进行。为了解释2006年医疗保险D部分生效后某些治疗（如生长因子）覆盖范围的潜在变化，我们将按两个不同时期进行分层分析，2001 - 2005年（D部分前）和2006 - 2007年（D部分后）。拟议的研究将是有史以来第一个使用基于大量人群的MDS患者样本来评估护理模式和MDS生存调整合并症的情况。它也将是第一个提供与MDS治疗有关的直接医疗费用的国家估计。该研究结果将对临床医生制定MDS患者的护理和治疗决策有价值，并对政策制定者在卫生资源分配方面有很大帮助。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费用。这项研究的发现将对临床医生照顾MDS患者和政策制定者分配卫生资源有价值。

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.