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）是一组导致骨髓生成障碍和外周血细胞减少的克隆性增殖性骨髓疾病。基于人群的癌症登记数据表明，美国每年诊断出超过10，000例MDS新病例。80%的MDS病例在诊断时为65岁或以上，观察到的3年生存率仅为35%。国会已经确定MDS正在被研究，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链接数据库的可用性为研究MDS患者的护理模式和结局提供了一个独特的机会，这两者都知之甚少。到2008年底，SEER医疗保险数据库将包括2001 - 2005年期间诊断的MDS患者的SEER数据和截至2007年底的这些患者的医疗保险索赔信息。我们建议使用这些数据来（1）评估护理模式，特别是MDS患者在诊断后接受的骨髓检查数量和各种治疗策略的使用，包括最近批准的DNA去甲基化剂，阿扎胞苷和地西他滨;（2）确定影响MDS患者生存的因素后，考虑共病条件;（3）估计MDS患者治疗的直接医疗费用。预计将在研究中纳入约6，716名66岁或以上诊断的MDS患者的基于人群的队列，因此将在年龄，性别，种族，SEER地区和合并症方面与患者匹配的非癌症对照。对于MDS患者，将评价骨髓检查的频率和接受各种治疗（例如输血、铁螯合治疗、生长因子、化疗和脱甲基剂）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统计学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患者的生存率，在考虑到合并症后，这是重要的预测MDS患者的死亡率。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）的医疗保险费用，并评估哪些患者特征影响费用。通过比较MDS患者的费用与在年龄、性别、种族、SEER地区和共病条件上与MDS患者相匹配的非癌症对照的费用，我们将估计归因于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。为了说明2006年医疗保险D部分生效后某些治疗（例如生长因子）覆盖范围的潜在变化，我们将按两个不同的时间段进行分层分析，2001 - 2005年（D部分前）和2006 - 2007年（D部分后）。这项研究将首次使用基于人群的MDS患者样本来评估护理模式和MDS生存率，以调整共病条件。它也将是第一个提供与MDS治疗相关的直接医疗费用的国家估计数。这项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，并对卫生资源分配的政策制定者非常有帮助。公共卫生相关性：骨髓增生异常综合征（MDS）是一组研究不足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6，716例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。这项研究的结果将对MDS患者的临床医生和卫生资源分配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.