Arizona Muscular Dystrophy Surveillance and Research Tracking Network (MD STARnet

亚利桑那州肌营养不良症监测和研究跟踪网络 (MD STARnet

• 批准号: 8247936
• 负责人: CHRISTOPHER MCCORD CUNNIFF
• 金额: $ 55万
• 依托单位: UNIVERSITY OF ARIZONA
• 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 2014-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8247936
• 关键词: Arizona; Muscular; Dystrophy; Surveillance; Research

DESCRIPTION (provided by the applicant): Objective 1: Conduct population-based surveillance and research of Duchenne/Becker muscular dystrophy (DBMD). 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 Principal Investigator meeting per year in Atlanta for investigators to present study findings. PUBLIC HEALTH RELEVANCE: This project will expand the work of the MD STARnet to include additional muscular dystrophies into the surveillance cohort and conduct an in-depth evaluation resulting in a best practices manual for the CDC in implementing future surveillance programs for other rare disorders. This will allow the CDC to better allocate their funds toward data collection and implementation and circumvent some of the start-up involved in planning and designing systems for surveillance activities.

描述（由申请方提供）：目的1：进行基于人群的杜氏/贝克肌营养不良症（DBMD）监测和研究。活动1.1.检查公共卫生实践中现有DBMD数据集的数据效用和质量。通过一个新的或现有的委员会与其他MD STARnet研究中心合作，检查与DBMD当前MD STARnet数据的数据收集、管理和分析相关的数据质量问题。活动1.2.向CDC提供有关DBMD监测的研究中心经验的信息，作为CDC对MD STARnet评估的一部分。活动1.3.优化收集的DBMD患者的临床数据变量数量，最多包括75-100个变量，这些变量可以产生可靠的相关信息，为公共卫生实践提供信息。活动1.4.在资助期的第一年，使用减少的变量数量对新的和随访的DBMD病例进行病历摘要。活动1.5.设计并进行访谈，以确定影响DBMD患者及其家庭的医疗过渡和其他生命阶段问题。目标二：设计并实施基于人群的7种肌营养不良症（肌强直性、先天性、肢带型、Emery-Dreifuss、面肩肱型、远端型和眼咽型）监测。 活动2.1.通过对现有MD STARnet系统的评估，修改或制定：（a）确定病例的方案;（B）病例定义;（c）数据收集工具，以收集关于流行率、人口统计学、保健覆盖范围和护理地点的信息;（d）全面质量控制程序，以解决与数据收集、管理和分析有关的数据质量问题。活动2.2.发展或保持与当地诊所和利益相关者的伙伴关系。活动2.3.在Y2-Y3期间对另外7例肌营养不良症进行一次病历摘要。目的3：评估方法进行基于人口的监测，为九个肌营养不良症。活动3.1。在国家协调中心的领导下，与其他研究中心合作，编写一份总结报告，评估将监测范围扩大到其他肌营养不良症。摘要应包括：（a）使用的方法;（B）吸取的经验教训;（c）优势;（d）挑战;（e）费用;（f）障碍;（g）开展肌肉萎缩症和罕见疾病监测方案的建议。目标4：利用2002-2011年MD STARnet上收集的信息分析数据、发表文章和传播报告。活动4.1。提出并领导至少三份手稿;在其他三份手稿上进行广泛合作。活动4.2.每年至少参加一次在亚特兰大举行的主要研究者会议，以便研究者介绍研究结果。 公共卫生相关性：该项目将扩展MD STARnet的工作，将更多的肌营养不良症纳入监测队列，并进行深入评估，为CDC实施未来其他罕见疾病监测计划提供最佳实践手册。这将使疾病预防控制中心能够更好地将资金分配给数据收集和实施，并避免规划和设计监测活动系统所涉及的一些启动。