Comparative Effectiveness: Erythropoietic Stimulating Agents in Treatment of MDS

疗效比较：红细胞生成刺激剂治疗 MDS

• 批准号: 7821746
• 负责人: AMY J DAVIDOFF
• 金额: $ 32.58万
• 依托单位: UNIVERSITY OF MARYLAND BALTIMORE
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-30 至 2011-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7821746
• 关键词: Address; Adopted; Adoption; Adverse event; Affect; Aftercare; Age; Anemia; Applications Grants; Area; Azacitidine; Benefits and Risks; Blood Transfusion; Boxing; CSF3 gene; Cancer Patient; Caring; Caucasoid Race; Cessation of life; Characteristics; Chromosome Deletion; Clinical; Clinical Trials; Clonal Hematopoietic Stem Cell; Cost Control; Cost Measures; Data; Data Set; Data Sources; Databases; Decitabine; Dependence; Development; Diagnosis; Disease Progression; Drug Prescriptions; Dysmyelopoietic Syndromes; Educational workshop; Effectiveness; Elderly; Enrollment; Erythrocyte Transfusion; Event; Foundations; Future; Geographic Locations; Guidelines; Health; Health Services Accessibility; Health Status; Hematologic Neoplasms; Hematologist; Incidence; Insurance; International; Intervention; Iron Overload; Knowledge; Link; Location; Malignant Neoplasms; Measures; Medical; Medicare; Medicare claim; Methods; Modeling; Monitor; Neoplasms; Oncologist; Oral; Outcome; Patients; Pattern; Pharmaceutical Preparations; Pharmacotherapy; Phase; Physicians' Offices; Population; Probability; Provider; Race; Randomized Clinical Trials; Randomized Controlled Clinical Trials; Regional Cancer; Relapse; Relative (related person); Reporting; Research; Research Design; Risk; Role; Safety; Series; Socioeconomic Status; Solid Neoplasm; Supportive care; Survival Analysis; Thrombosis; Time; Transfusion; Treatment Cost; Treatment Failure; United States Centers for Medicare and Medicaid Services; United States Food and Drug Administration; United States National Institutes of Health; Update; beneficiary; cancer therapy; chromosome 5q loss; cohort; comparative effectiveness; cost; cytopenia; disease characteristic; effectiveness research; high risk; improved; lenalidomide; neoplasm registry; patient population; policy implication; population based; response

DESCRIPTION (provided by applicant): This NIH Challenge Grant application addresses the specific Challenge Area (05): Comparative Effectiveness Research, and the specific Challenge Topic, 05-CA-104: Comparative Effectiveness Research in Cancer Treatment. This study will examine patterns of treatment with erythropoietic stimulating agents (ESA) in patients with myelodysplastic syndromes (MDS), and compare clinical benefits, risks, and costs associated with any ESA use, and different levels of ESA exposure. The alternative for low-risk patients with MDS associated anemia is supportive care with red blood cell transfusions and management of iron overload. ESAs have been used for the treatment of anemia in MDS patients for approximately 15 years, with a 20 - 40% response rate and median response duration of two years. High-risk MDS patients and those with poor or lapsed response to ESAs may receive 5 azacitidine or decitabine. Clinicians treating MDS patients have widely adopted ESA use for anemic MDS patients despite the lack of large-scale clinical trial evidence demonstrating longer-term clinical benefits. In 2007 the Food and Drug Administration administered a warning concerning the use of ESAs in patients with cancer related anemia due to apparent increase in thromboembolic events, as well as a possible decrease in relapse-free survival. The absence of large scale randomized trials in MDS precludes definitive conclusions regarding the safety and efficacy of these agents in this population; clinical trials randomizing between ESAs and supportive care would be of limited feasibility in the US. The recent inclusion of MDS as a reportable diagnosis in the SEER database (2001) provides an opportunity to explore the current use of ESAs in MDS patients in the Medicare population. By examining claims data linked to SEER reported MDS diagnoses, the patterns of ESA use in the Medicare population will be described. We will examine the association between ESA use and health outcomes including thromboembolic events, disease progression to AML, and overall survival. SEER-Medicare cases will also be compared to the survival of patients from an historic cohort of untreated patients (International MDS Risk Analysis Workshop (IMRAW) data base) to further estimate the effect of ESA exposure on health outcomes. The effect of ESA use on cost of care will also be examined. Finally, we will use a separate dataset that captures Medicare enrollment and claims data, including Medicare Part D (prescription drug) claims for MDS patients from 2005 through 2008. We will use these data to examine whether patterns of ESA use, transfusions, and other drug therapies have been affected by availability of a new oral agent (lenalidomide) starting in 2006, and the effect of the FDA and CMS regulatory activities, which occurred in 2007. These results will have important treatment and policy implications regarding the relative safety and efficacy of ESAs in this elderly MDS patient population and may inform future CMS coverage decisions. Myelodysplastic syndromes (MDS) are the most common hematologic malignancy in the elderly, with at least 10,000 new cases diagnosed each year. Erythropoietic stimulating agents (ESA) are effective in treating the symptomatic anemia that affects MDS patients, but little is known about longer term positive and negative health effects of ESAs, compared to supportive care with repeated blood transfusions. In this study, we will use data from regional cancer registries that is linked to Medicare enrollment and insurance claims data. We will examine the characteristics of Medicare beneficiaries with MDS, patterns of treatment, safety and effectiveness of ESA use, and costs of care.

描述（由申请人提供）： 此 NIH 挑战补助金申请涉及特定挑战领域 (05)：比较有效性研究，以及特定挑战主题 05-CA-104：癌症治疗的比较有效性研究。本研究将探讨骨髓增生异常综合征 (MDS) 患者的红细胞生成刺激剂 (ESA) 治疗模式，并比较与任何 ESA 使用以及不同水平的 ESA 暴露相关的临床获益、风险和成本。对于患有 MDS 相关性贫血的低风险患者，另一种选择是通过红细胞输注和铁过载管理进行支持治疗。 ESA 用于治疗 MDS 患者贫血已有约 15 年，缓解率为 20 - 40%，中位缓解持续时间为两年。高危 MDS 患者和对 ESA 反应不佳或失效的患者可能会接受 5 粒阿扎胞苷或地西他滨治疗。尽管缺乏大规模临床试验证据证明长期临床益处，但治疗 MDS 患者的临床医生已广泛采用 ESA 治疗贫血 MDS 患者。 2007年，美国食品和药物管理局对患有癌症相关贫血的患者使用ESA发出了警告，因为血栓栓塞事件明显增加，并且无复发生存率可能降低。由于缺乏针对 MDS 的大规模随机试验，无法得出关于这些药物在该人群中的安全性和有效性的明确结论；在美国，在 ESA 和支持性护理之间进行随机临床试验的可行性有限。最近将 MDS 作为可报告的诊断纳入 SEER 数据库（2001 年），这为探索目前 ESA 在医疗保险人群中 MDS 患者中的使用提供了机会。通过检查与 SEER 报告的 MDS 诊断相关的索赔数据，将描述医疗保险人群中 ESA 的使用模式。我们将研究 ESA 使用与健康结果之间的关联，包括血栓栓塞事件、疾病进展为 AML 和总体生存率。 SEER-Medicare 病例还将与未经治疗的历史队列患者的生存率（国际 MDS 风险分析研讨会 (IMRAW) 数据库）进行比较，以进一步估计 ESA 暴露对健康结果的影响。还将研究 ESA 的使用对护理成本的影响。最后，我们将使用一个单独的数据集来捕获 Medicare 登记和索赔数据，包括 2005 年至 2008 年 MDS 患者的 Medicare D 部分（处方药）索赔。我们将使用这些数据来检查 ESA 使用、输血和其他药物治疗受到 2006 年开始的新型口服药物（来那度胺）的上市以及 2007 年 FDA 和 CMS 监管活动的影响。这些结果将对 ESA 在老年 MDS 患者群体中的相对安全性和有效性产生重要的治疗和政策影响，并可能为未来 CMS 覆盖决策提供信息。骨髓增生异常综合征 (MDS) 是老年人中最常见的血液系统恶性肿瘤，每年至少诊断出 10,000 例新病例。促红细胞生成剂 (ESA) 可有效治疗影响 MDS 患者的症状性贫血，但与反复输血的支持治疗相比，人们对 ESA 的长期积极和消极健康影响知之甚少。在本研究中，我们将使用来自地区癌症登记处的与医疗保险登记和保险索赔数据相关的数据。我们将研究患有 MDS 的 Medicare 受益人的特征、治疗模式、ESA 使用的安全性和有效性以及护理费用。