Racioethnic and Socioeconomic Impact on Outcomes in Pediatric Cardiomyopathy

项目摘要 心脏病是儿童疾病相关死亡的主要原因。一个主要的贡献者被扩大了 心肌病(DCM)，近三分之一的患者在第一年死亡或接受心脏移植 在确诊后，几乎一半在5年内。虽然儿童扩张性心肌炎的遗传基础越来越多 在心脏移植之外被认识到，对于患有这种毁灭性疾病的儿童来说，几乎没有选择 疾病，例证了临床上未得到满足的需要，以更好地了解与疾病预后相关的因素 对患有扩张型心肌炎的儿童进行干预，制定更有效的干预措施。虽然一些关键的相关因素是临床上的， 社会人口因素也可能起到一定作用。国家心肺血液研究所赞助 1994年开始的儿科心肌病登记(PCMR)及其后来的遗传和生物标记物研究 包含大约4,000例儿童和青少年心肌病的数据，其中一半以上 都是扩张型心肌炎。PCMR企业提供丰富的纵向数据库，并包含关键信息 这将使我们能够评估这一人群在健康结果方面的种族差异 高死亡率、高发病率和高卫生保健利用率。 拟议研究的具体目的是：1)根据种族/民族描述临床 心脏移植名单的结果比率和死亡与心脏移植的组合，以及 用于评估健康差距的措施类别，包括社会经济地位(SES)、保险 基于症状学超声心动图特征、功能状态和健康状况的病情、严重程度- 相关的生活质量和地理位置；2)确定SES和临床结果之间的关联；以及 种族/民族和社会经济地位的相对影响；以及3)估计#年种族-民族差距的程度。 临床结果，利用两种不同的健康差距概念，即根据2010年健康人 和医学研究所(2003年)。提议的项目有几个新的特征，包括应用程序 卫生保健利用方法对健康结果的影响，以检查种族差异；使用 地理编码以确定邻域级别的SES；以及具体实施统计方法 专为差异研究而设计。Sleeper、Wilkinson和Lipshultz博士提出的研究小组， 扎斯拉夫斯基博士与PCMR的研究人员合作，有着悠久的卓有成效的合作历史。 他们拥有临床和统计学专业知识，可以成功地执行这一项目，以识别种族 扩张型心肌病儿童临床结局的差异。

Project Summary Heart disease is a leading cause of disease-related mortality in childhood. A major contributor is dilated cardiomyopathy (DCM), with nearly one-third of patients dying or receiving a heart transplant in the first year following diagnosis, and almost half within 5 years. While the genetic basis for pediatric DCM is increasingly recognized outside of cardiac transplantation, few options exist for children suffering from this devastating disease, exemplifying the clinically unmet need to better understand the factors related to outcomes for children with DCM and develop more effective interventions. While some of the key correlates are clinical, sociodemographic factors may also play a role. The National Heart, Lung and Blood Institute-sponsored Pediatric Cardiomyopathy Registry (PCMR), which began in 1994, and its later genetic and biomarker studies contain data on approximately 4,000 cases of cardiomyopathy in children and adolescents of which over half are cases of DCM. The PCMR enterprise offers a rich, longitudinal database, and contains key information that will allow us to evaluate racioethnic disparities in health outcomes for this population that is characterized by high mortality, morbidity and health care utilization. The specific aims of the proposed research are 1) To describe, according to racial/ethnic group, clinical outcome rates of heart transplant listing and the composite of death and heart transplant, as well as the classes of measures used to evaluate health disparities, including socioeconomic status (SES), insurance status, severity of disease based on symptomatology echocardiographic profile, functional status and health- related quality of life, and geography; 2) To determine the association between SES and clinical outcomes, and the relative impacts of race/ethnicity and SES; and 3) To estimate the magnitude of racioethnic disparities in clinical outcomes, utilizing two different constructs of health disparity, i.e., according to Healthy People 2010 and the Institute of Medicine (2003). The proposed project has several novel features including the application of health care utilization methodologies to health outcomes to examine racioethnic disparities; the use of geocoding to determine neighborhood-level SES; and implementation of statistical methodologies specifically designed for disparities research. The proposed research team of Drs. Sleeper, Wilkinson and Lipshultz, with Dr. Zaslavsky consulting, in conjunction with PCMR investigators, has a long history of productive collaboration and they possess the clinical and statistical expertise to successfully execute this project to identify racioethnic disparities in the clinical outcomes of children with DCM.