Comp C-Data Coordinating Center for Surveillance and Research

描述(由申请人提供)：肌营养不良症监测、跟踪和研究网络(MD STARnet)成立于2002年，旨在对Duchenne和Becker肌营养不良症(DBMD)患者进行基于人群的监测和长期随访。自2004年以来一直在进行对DBMD的纵向监测。积极的后续行动始于2007年，对DBMD病例的主要照顾者进行电话访谈，以确定医疗干预措施以及家庭特征、地理位置、保险状况和社会支持对临床结果的影响。实施的其他后续行动包括对成年DBMD病例及其照顾者进行邮寄调查，以检查这些人的需求和生活质量，最近还描述了从儿科到成人临床护理的过渡以及青少年晚期和成年DBMD病例的其他生活经历。2012年，MD STARnet扩大到包括另外7个MD的横断面监测(先天性、远端、Emery-Dreifuss、面肩-肱骨、四肢带、肌强直和眼咽部)。我们的爱荷华州站点是2002年MD STARnet的四个特许权获得者之一，并在2006和2011年成功退款。爱荷华州的这个站点自2003年成立以来，一直是国家MD STARnet数据协调中心(DCC)。作为发展协调中心，我们成功地创建和实施了一个基础设施，为所有基于人口的DBMD纵向监测和研究活动以及为额外的MD提供基于人口的横断面监测提供信息学专门知识和支持。我们建议继续作为疾病预防控制中心，为MDS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。具体地说，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、数据库和相关文档；2)管理每个项目点收集的监测数据的汇集、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；以及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收到了建立和运营DCC的资金，我们对继续这样的工作充满热情。继续使用我们经验丰富的DCC基础设施将使我们能够有效利用项目资源，用于信息学专门知识，支持MD的监测和研究活动，并将监测活动扩大到选定的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.