Arizona Muscular Dystrophy Surveillance and Research Program

亚利桑那州肌营养不良症监测和研究计划

• 批准号: 7231273
• 负责人: CHRISTOPHER MCCORD CUNNIFF
• 金额: $ 47.3万
• 依托单位: UNIVERSITY OF ARIZONA
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-09-01 至 2011-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7231273
• 关键词: Duchenne muscular dystrophy; children; clinical research; cooperative study; epidemiology; gene environment interaction; health care quality; human population study; human subject; muscular dystrophy; socioeconomics

DESCRIPTION (provided by applicant): In 2002, the Centers for Disease Control and Prevention (CDC) awarded four sites - Arizona, Colorado, Iowa and New York - cooperative agreements (RFA 02172) to develop a surveillance network, the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet). The overall objectives are: 1) to conduct active, population-based surveillance of Duchenne and Becker Muscular Dystrophy (DBMD) to characterize the epidemiology of DBMD and its complications and 2) to develop long-term follow-up and tracking of children with DBMD to describe the history and outcome of treated cases and/or those cases with treatments not consistent with protocol standards. The hypothesis guiding this research is that population-based surveillance and integrated research programs are effective in determining the prevalence of DMD/BMD disorders and establishing data on health outcomes and the types of care for these disorders. Research has shown that population-based surveillance for individual disorders is an effective first step to clinical and descriptive studies. Surveillance provides population data as a basis for making comparisons to the samples of individuals with these disorders that respond to questionnaires or clinical studies. The alternative of volunteer-based research in clinic-based populations may produce data that do not reflect the population of individuals with the disorder. For the current project, the Arizona Muscular Dystrophy Research and Surveillance Program (Arizona MD STARnet) proposes to continue as a participating MD STARnet site with the following specific aims: 1. Generate population-based prevalence and incidence rates for DBMD in the United States with particular attention to differences in rates over time and by race/ethnicity; 2. Identify the early signs and symptoms of DBMD; 3. Describe the medical and social services received and quality of life of families of patients with DBMD and whether these vary by race/ethnicity and socioeconomic status; and 4. Investigate whether the severity or course of DBMD is influenced by variation in type of care received and by type of mutation identified.

描述(由申请人提供)：2002年，疾病控制和预防中心授予四个地点--亚利桑那州、科罗拉多州、爱荷华州和纽约州的合作协议(02172)，以发展一个监测网络，即肌营养不良症监测跟踪和研究网络(MD STARnet)。总体目标是：1)对Duchenne和Becker肌营养不良症(DBMD)进行积极的、以人群为基础的监测，以确定DBMD及其并发症的流行病学特征；2)对DBMD儿童进行长期跟踪和跟踪，以描述接受治疗的病例和/或那些治疗方案不符合标准的病例的历史和结果。指导这项研究的假设是，基于人群的监测和综合研究计划在确定DMD/BMD疾病的流行率以及建立关于这些疾病的健康结果和护理类型的数据方面是有效的。研究表明，以人群为基础的个体疾病监测是临床和描述性研究的有效第一步。监测提供人口数据，作为与对问卷或临床研究作出反应的患有这些疾病的个人样本进行比较的基础。在以临床为基础的人群中进行基于志愿者的研究的替代方案可能产生的数据不能反映患有这种疾病的个体的数量。在目前的项目中，亚利桑那州肌营养不良症研究和监测计划(Arizona MD STARnet)建议继续作为参与的MD STARnet网站，具体目标如下：1.在美国生成DBMD的基于人群的流行率和发病率，特别关注不同时间和种族/民族的发病率差异；2.识别DBMD的早期迹象和症状；3.描述DBMD患者家属获得的医疗和社会服务以及生活质量，以及这些是否因种族/民族和社会经济地位而异；以及4.调查DBMD的严重程度或病程是否受到接受的护理类型的变化和所识别的突变类型的影响。