Comp C-Data Coordinating Center for Surveillance and Research

描述（由申请人提供）：肌营养不良监测、跟踪和研究网络（MD STARnet）成立于2002年，对杜氏和贝克肌营养不良（DBMD）患者进行基于人群的监测和长期随访。自2004年以来一直在进行DBMD的纵向监测。积极的后续行动开始于2007年，通过电话采访DBMD病例的主要照顾者，以确定医疗干预措施和家庭特征，地理，保险状况和社会支持对临床结果的影响。其他后续管理包括邮寄调查成人DBMD病例及其护理人员，以检查这些人的需求和生活质量，最近描述了从儿科到成人临床护理的过渡以及青少年后期和成人DBMD病例的其他生活经历。2012年，MD STARnet扩展到包括另外7种MD（先天性、远端、Emery-Dreifuss、面肩肱、肢带、肌强直和眼咽）的横断面监测。我们的爱荷华州研究中心是2002年MD STARnet的四个特许获奖者之一，并于2006年和2011年成功退款。自2003年成立以来，爱荷华州研究中心还一直担任国家MD STARnet数据协调中心（DCC）。作为DCC，我们成功地创建并实施了一个基础设施，为所有基于人群的DBMD纵向监测和研究活动以及其他MD的基于人群的横断面监测提供信息学专业知识和支持。我们建议继续作为DCC，为MD和选定的神经肌肉疾病（NMD）的监测和研究提供专家信息学支持。具体而言，我们的目标是：1）开发和维护用于监测数据收集和分析的软件应用程序、数据库和相关文件; 2）管理每个项目中心收集的监测数据的汇总、清理和传播; 3）分发和支持监测数据收集、临床审查和数据分析所需的应用程序和数据库; 4）通过参加工作组、委员会和面对面会议提供技术援助; 5）参与项目评估计划的规划和实施。爱荷华州的网站已经成功地与资金收到建立和运作的DCC，我们是热情继续这样的工作。继续使用我们经验丰富的DCC基础设施将允许有效利用项目资源，以获得信息学专业知识，并支持MD的监测和研究活动，并将监测活动扩展到选定的NMD。

DESCRIPTION (provided by applicant): The Muscular Dystrophy surveillance, tracking, and Research Network (MD STARnet) was established in 2002 to conduct population-based surveillance and long-term follow-up of individuals with Duchenne and Becker muscular dystrophy (DBMD). Longitudinal surveillance for DBMD has been ongoing since 2004. Active follow-up commenced in 2007 by administering telephone interviews to primary caregivers of DBMD cases to determine medical interventions and influence of family characteristics, geography, insurance status, and social support on clinical outcomes. Additional follow-up administered included mailed surveys to adult DBMD cases and their caregivers to examine needs and quality of life for these individuals and, most recently to describe transitions from pediatric to adult clinical care and other life experiences for late-adolescent and adult DBMD cases. In 2012, the MD STARnet was expanded to include cross-sectional surveillance for seven additional MDs (congenital, distal, Emery-Dreifuss, facioscapulohumeral, limb-girdle, myotonic, and oculopharyngeal). Our Iowa Site was one of four charter awardees for the MD STARnet in 2002 and was successfully refunded in 2006 and 2011. The Iowa Site has also served as the national MD STARnet Data Coordinating Center (DCC) since it was established in 2003. As the DCC, we successfully created and implemented an infrastructure that provided informatics expertise and support for all population-based DBMD longitudinal surveillance and research activities and for population-based cross-sectional surveillance for the additional MDs. We propose to continue as the DCC to provide expert informatics support for surveillance and research of MDs and selected neuromuscular diseases (NMDs). Specifically, we aim to: 1) develop and maintain software applications, databases, and related documentation for surveillance data collection and analysis; 2) manage the pooling, cleaning, and dissemination of surveillance data collected by each project site; 3) distribute and provide support for application and databases required for surveillance data collection, clinical review, and data analysis; 4) provide technical assistance through participation in workgroups, committees, and in-person meetings; and 5) participate in the planning and implementation of the project evaluation plan. The Iowa Site has been successful with funding received to establish and operate the DCC, and we are enthusiastic about continuing such work. Continued use of our experienced DCC infrastructure will permit an efficient use of project resources for informatics expertise and support for surveillance and research activities for MDs and expansion of surveillance activities to selected NMDs.