Erythropoiesis-stimulating agent use among chronic kidney disease patients

慢性肾病患者使用红细胞生成刺激剂

• 批准号: 8323896
• 负责人: Dennis Joseph Cotter
• 金额: $ 11.49万
• 依托单位: MEDICAL TECHNOLOGY AND PRACTICE PATTERNS
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-09-01 至 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8323896
• 关键词: Accounting; Address; Affect; American; Anemia; Cardiovascular Diseases; Cardiovascular system; Caring; Chronic Kidney Failure; Chronic Kidney Insufficiency; Classification; Clinical; Clinical Practice Guideline; Consensus; Creatinine; Data; Dialysis procedure; Disease Progression; District of Columbia; Dose; End stage renal failure; Ensure; Erythropoiesis; Erythropoietin; Evaluation; Foundations; Goals; Government; Guidelines; Health; Healthy People 2010; Hematocrit procedure; Hemoglobin; Hemoglobin concentration result; Intervention; Iron; Kidney; Kidney Diseases; Kidney Transplantation; Laboratories; Lead; Life; Medicare; Methods; Modeling; Morbidity - disease rate; National Institute of Diabetes and Digestive and Kidney Diseases; Observational Study; Outcome; Patients; Pharmaceutical Preparations; Pilot Projects; Population; Printing; Process; Protocols documentation; Provider; Randomized; Regimen; Research; Risk; Selection Bias; Staging; Stratification; Structural Models; Techniques; Time; Uncertainty; United States Dept. of Health and Human Services; adverse outcome; base; cost; cost effective; evidence base; improved; innovation; mortality; nephrogenesis; prevent; randomized trial; research study; treatment strategy

DESCRIPTION (provided by applicant): Background. There has been a 30% increase in chronic kidney disease (CKD) over the past decade. Anemia almost invariably develops in patients with progressive chronic renal insufficiency, and is associated with a number of adverse outcomes. Erythropoiesis-stimulating agents (ESA therapy) - approved in 1989 for use among the renal disease population - is used to correct anemia. When to start ESA therapy - at what hematocrit level - and at what threshold level to maintain hematocrit throughout treatment is unknown. For example, should ESA therapy be initiated when patients are severely anemic (i.e., hematocrit below 30%) or only moderately anemic (i.e., hematocrit between 30 - 33%)? Even more fundamental, providers appear to be uncertain about the clinical benefits to be derived from epoetin therapy for their anemic CKD patients. Objective. We will examine three possible treatment strategies to address the following research question: Do different anemia management treatment strategies among the CKD population result in different patient outcomes with regard to progression of CKD, cardiovascular disease, and mortality? The strategies that will be examined in this project include: 1) ESAs are not prescribed for patients with anemia; 2) ESAs are prescribed for severely anemic patients (hct < 30%); and 3) ESAs are prescribed for moderately anemic patients (hct between 30 - 33%). Methods. We propose to use an observational study using innovative causal modeling techniques that 'mimics' RCTs by appropriately handling time-dependent confounding between therapy and intermediate clinical outcomes. Specifically, patients who do not follow one of the three strategies listed above throughout the study will generate time- dependent selection bias when conventional statistical techniques are used. Instead, Marginal Structural Models (MSMs) can handle such biases with the caveat that treatment-to-treatment data (including ESA dosing information and hematocrit values) must be used in the modeling process. We propose to use Kaiser Permanente Mid-Atlantic data containing treatment to treatment information as well as detailed laboratory data (GFR, creatinine, iron) and medication use. Significance. Notably, despite the lack of consensus for the use of ESAs, and the large number of CKD patients who never receive therapy, the volume of ESAs administered to predialysis patients has increased 5-fold in the last decade. Our application - to examine different strategies for initiating and maintaining ESA therapy - has important implications for both anemia management and its costs among the burgeoning CKD population.

简介(申请人提供)：背景。在过去的十年里，慢性肾脏疾病(CKD)的发病率增加了30%。贫血几乎总是发生在进行性慢性肾功能不全的患者中，并与许多不良后果有关。红细胞生成刺激剂(ESA疗法)于1989年被批准用于肾脏疾病人群，用于纠正贫血。何时开始ESA治疗-在什么红细胞压积水平-以及在整个治疗过程中将红细胞压积维持在什么阈值水平是未知的。例如，当患者患有重度贫血(即红细胞压积低于30%)或仅中度贫血(即红细胞压积在30%-33%之间)时，是否应该开始ESA治疗？更根本的是，供应商似乎不确定促红细胞生成素治疗贫血CKD患者的临床益处。目标。我们将检查三种可能的治疗策略来解决以下研究问题：CKD人群中不同的贫血管理治疗策略是否会在CKD的进展、心血管疾病和死亡率方面导致不同的患者结局？该项目将审查的策略包括：1)不给贫血患者开ESA；2)给重度贫血患者开ESA(Hct&lt；30%)；3)给中度贫血患者开ESA(Hct在30%-33%之间)。方法：研究方法。我们建议使用一项观察性研究，使用创新的因果建模技术，通过适当处理治疗和中间临床结果之间的依赖时间的混淆来“模拟”随机对照试验。具体地说，当使用常规统计技术时，在整个研究过程中不遵循上面列出的三种策略之一的患者将产生时间相关的选择偏差。取而代之的是，边际结构模型(MSM)可以处理这种偏差，但需要注意的是，必须在建模过程中使用治疗到治疗的数据(包括ESA剂量信息和红细胞压积数值)。我们建议使用Kaiser Permanente中大西洋地区的数据，其中包含治疗信息以及详细的实验室数据(GFR、肌酐、铁)和药物使用情况。意义重大。值得注意的是，尽管对ESA的使用缺乏共识，而且大量CKD患者从未接受过治疗，但在过去十年中，对透析前患者应用ESA的数量增加了5倍。我们的应用--研究启动和维持ESA治疗的不同策略--对迅速增长的CKD人群中的贫血管理及其成本具有重要的影响。