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.
The Reproductive Molecular Epidemiology Research and Education Program at The University of Iowa will provide expert public health informatics support as the Data Coordinating Center for all project sites funded to conduct surveillance and research of muscular dystrophies and neuromuscular disorders under RFA-DD-14-001. Data collected can aid in providing treatment and support options and identifying unique needs of individuals with these conditions, as well as their families. Findings will be shared with scientiss, clinicians, advocacy groups, and policy makers developing interventions and patient resources.
