Arizona Muscular Dystrophy Surveillance and Research Tracking Network (MD STARnet

！“#$%#&‘()*$+，-.#/’.0”1！“0/”‘2)3#“，%.0”)45’-.6)7#“-.6)8#99(，：；)！”#$$%&&‘！“()%*+，-(.)！/0！ 目的1：对Duchenne/Becker肌营养不良症进行人群监测和研究 练习1.1。检查用于公共卫生实践的现有DBMD数据集的数据效用和质量。参与： 其他MD STARnet站点通过一个新的或现有的委员会来检查与数据相关的数据质量问题 为DBMD收集、管理和分析当前的MD STARnet数据。 练习1.2。向疾控中心提供有关作为疾控中心一部分的DBMD监测的现场经验的信息 对MD STARnet的评估。 练习1.3。细化在患有DBMD的个人身上收集的临床数据变量的数量，以包括 最多75-100个变量，产生可靠的、相关的信息，为公共卫生实践提供信息。 活动1.4。使用减少的数量对新的和后续的DBMD病例进行病历摘要 在供资期间的第一年期间的变数。 活动1.5。设计并进行访谈，以确定医疗转型和其他影响生命阶段的问题 患有DBMD的个人及其家人。 目标2：设计并实施对另外七种肌营养不良症的基于人群的监测 (肌强直、先天性、四肢紧张症、Emery-Dreifuss、面肩肩、远端和眼咽部) 练习2.1。通过对现有MD STARnet系统的评估，修改或开发：(A)用于确定 病例；(B)病例定义；(C)数据收集工具，收集关于流行率、人口统计、 保健覆盖范围和护理地点；(D)全面的质量控制程序，以解决数据质量问题 与数据收集、管理和分析相关的问题。 练习2.2。发展或维护与当地诊所和利益相关者的伙伴关系 练习2.3。在Y2-Y3期间对另外7例肌营养不良症进行一次病历提取 目标3：评估对9种肌营养不良症进行人群监测的方法学 练习3.1。在国家协调中心的领导下，与其他站点协作制作总结 评估将监测扩展到其他肌营养不良症的报告。摘要应包括：(A) 使用的方法；(B)经验教训；(C)优势；(D)挑战；(E)费用；(F)障碍；(G)建议 进行肌营养不良症和罕见疾病监测项目。 目标4：使用从MD STARnet上收集的信息分析数据、发表文章和发布报告 2002-2011年 练习4.1。提出并领导至少三份手稿；广泛合作撰写另外三份手稿。 练习4.2。每年至少参加一次在亚特兰大举行的PI会议，让调查人员展示研究结果。

!"#$%#&'()*$+,-.#/'.0"1!"0/"'2)3#",%.0")45'-.6)7#"-.6)8#99(,:;)))!! "#$$%&&'!"()%*+,-(.)!/0! Objective 1: Conduct population-based surveillance and research of Duchenne/Becker muscular dystrophy Activity 1.1. Examine data utility and quality of existing DBMD data set for public health practice. Participate with other MD STARnet sites through a new or existing committee to examine data quality issues associated with data collection, management and analysis of current MD STARnet data for DBMD. Activity 1.2. Provide information to CDC regarding site experiences with surveillance for DBMD as part of a CDC assessment of MD STARnet. Activity 1.3. Refine the number of clinical data variables collected on individuals who have DBMD to include a maximum of 75-100 variables that yield reliable, relevant information that informs public health practice. Activity 1.4. Conduct medical record abstraction of new and follow-up DBMD cases using the reduced number of variables during year 1 of the funding period. Activity 1.5. Design and conduct an interview to determine healthcare transition and other life stage issues affecting individuals with DBMD and their families. Objective 2: Design and implement population-based surveillance on seven additional muscular dystrophies (Myotonic, Congenital, Limb-Girdle, Emery-Dreifuss, Facioscapulohumeral, Distal, and Oculopharyngeal) Activity 2.1. Through assessment of the existing MD STARnet system, modify or develop: (a) Protocols for ascertaining cases; (b) Case definitions; (c) Data collection instruments to collect information on prevalence, demographics, healthcare coverage, and locations of care; (d) Comprehensive quality control procedures to address data quality issues associated with data collection, management and analysis. Activity 2.2. Develop or maintain partnerships with local clinics and stakeholders Activity 2.3. Conduct medical record abstraction for seven additional muscular dystrophies once during Y2-Y3 Objective 3: Assess methodology for conducting population-based surveillance for the nine muscular dystrophies Activity 3.1. Under the leadership of the national coordinating center, collaborate with other sites to produce a summary report assessing the expansion of surveillance to other muscular dystrophies. The summary should include: (a) Methods used; (b) Lessons learned; (c) Strengths; (d) Challenges; (e) Costs; (f) Barriers; (g) Recommendations for conducting muscular dystrophy and rare disease surveillance programs. Objective 4: Analyze data, publish articles, and disseminate reports using information collected on MD STARnet from 2002-2011 Activity 4.1. Propose and lead a minimum of three manuscripts; extensively collaborate on three other manuscripts. Activity 4.2. Participate in at least one PI meeting per year in Atlanta, for investigators to present study findings.