Comparative Effectiveness and Safety of Newer Agents for Anemia Treatment in HD

描述（由申请人提供）：超过575，000名美国人患有终末期肾病，通常并发贫血，其中大多数患者接受维持透析治疗。这项拟议的工作将比较darbepopolysine alfa的有效性和安全性，darbepopolysine alfa是一种治疗贫血的新型注射药物，与其已建立的表亲epopolysine alfa。同样，ferumoxytol，一种新的静脉补铁治疗贫血将与一个既定的铁补充剂，铁蔗糖进行比较。这两种药物都是根据随机研究的信息批准的，这些研究评估了它们减少贫血的疗效。然而，关于这两种药物的长期疗效和安全性的信息非常有限。 我们建议研究这两种药物的有效性和安全性，与它们的同类药物进行比较。具有科学意义的事件包括短期结局（速发过敏反应、实验室参数，包括血红蛋白、铁饱和度、铁蛋白）和长期结局，包括感染、心血管事件、癌症和死亡率。 我们将使用从医疗保险账单索赔和透析提供者的电子医疗记录中收集的数据来解决这些研究问题。由于设施根据其处方集中可用的内容在这些治疗方案中进行选择，而不一定基于患者特征，因此我们可以利用当设施为整个患者群体切换ESA或铁剂时发生的自然实验。我们将应用现代流行病学技术来消除偏见，并期望提供相对效益和风险的有效估计。 这项研究的结果有可能立即影响和改善终末期肾病患者接受的护理。我们的研究结果可能会提高所接受的护理质量，从而改善这一弱势患者群体的结局。工作的目标和范围与国家糖尿病、消化和肾脏疾病研究所的使命完全一致，更具体地说，与肾脏、泌尿系统和血液系统疾病研究所完全一致，该研究所将考虑这项资助申请。 公共卫生相关性：超过50万美国人的肾脏已经不可逆转地停止工作，这使得这些患者依赖于接受定期的肾透析。贫血在这一人群中很普遍，最近出现了新的治疗方法。我们建议研究其中两种新药达贝泊利和ferumoxytol的长期获益和风险，这将提供重要的新信息，这些信息是优化这些患者安全有效的医疗保健的必要信息。

DESCRIPTION (provided by applicant): More than 575,000 Americans have end-stage renal disease, which is typically complicated by anemia, and most of these patients receive maintenance dialysis treatment. The proposed work will compare the effectiveness and safety of darbepoetin alfa, a novel injectible drug for the treatment of anemia, with its established cousin, epoetin alfa. Similarly, ferumoxytol, a new intravenous iron supplement for the treatment of anemia will be compared with an established iron supplement, iron sucrose. Both drugs were approved using information from randomized studies that evaluated their efficacy on reducing anemia. Very limited information, however, is available for either drug regarding their longer-term efficacy and safety. We are proposing to study the effectiveness and safety of these two medications when compared to their established cousins. Events of scientific interest include short-term outcomes (anaphylaxis, laboratory parameters including hemoglobin, iron saturation, ferritin) and longer- term outcomes including infection, cardiovascular events, cancer, and mortality. We will use already collected data from Medicare billing claims and electronic medical records of dialysis providers to address these research questions. Since facilities choose among these treatment options based on what is available in their formulary, and not necessarily based on patient characteristics, we can exploit natural experiments that occur when facilities switch their ESA or iron agent for their entire patient population. We will apply modern epidemiological techniques to eliminate bias and aspire to provide valid estimates of relative benefits and risks. Findings from the proposed work have the potential to immediately impact and improve the care that patients with end-stage renal disease receive. Our results may improve the quality of care received and, thus, the outcomes of this vulnerable patient population. The aims and scope of work are in full congruence with the mission of the National Institutes of Diabetes and Digestive and Kidney Diseases, and more specifically the Division of Kidney, Urologic, and Hematologic Diseases, which will consider this application for funding. PUBLIC HEALTH RELEVANCE: The kidneys of more than 500,000 Americans have irreversibly stopped working, which renders these patients dependent on receiving regular kidney dialysis. Anemia is widespread in this population and newer treatments for it have recently become available. We propose to study the long-term benefits and risks of two of these new drugs, darbepoetin and ferumoxytol, which will provide important new information that is necessary information to optimize safe and effective heath care of these patients.