Arizona Muscular Dystrophy Surveillance and Research Program

描述（由申请人提供）：2002年，美国疾病控制和预防中心（CDC）授予亚利桑那州、科罗拉多州、爱荷华州和纽约州四个试验点合作协议（RFA 02172），以发展一个监测网络，即肌肉萎缩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、基于人群的监测，以表征DBMD及其并发症的流行病学特征；2)对DBMD患儿进行长期随访和跟踪，以描述治疗病例和/或治疗不符合方案标准的病例的病史和结果。指导本研究的假设是，基于人群的监测和综合研究计划在确定DMD/BMD疾病的患病率和建立健康结果数据以及这些疾病的护理类型方面是有效的。研究表明，以人群为基础的个体疾病监测是进行临床和描述性研究的有效第一步。监测提供人口数据，作为与回答问卷调查或临床研究的患有这些疾病的个体样本进行比较的基础。在以临床为基础的人群中进行以志愿者为基础的研究的另一种选择可能产生的数据不能反映患有这种疾病的个体的人群。对于当前的项目，亚利桑那州肌肉萎缩症研究和监测计划（亚利桑那州MD STARnet）建议继续作为MD STARnet的参与站点，其具体目标如下：在美国产生基于人群的DBMD患病率和发病率，特别注意发病率随时间和种族/民族的差异；2. 确定DBMD的早期体征和症状；3. 描述DBMD患者家属接受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；和4。调查DBMD的严重程度或病程是否受到所接受的治疗类型的变化和所确定的突变类型的影响。

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.