Evaluation of Treatments and Services to People with Duchenne Muscular Dystrophy

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DESCRIPTION (provided by applicant): Project Summary Taken together, Duchenne muscular dystrophy (DMD) and the milder allelic variant Becker muscular dystrophy (BMD) are the most common recessive form of muscular dystrophy. The birth prevalence of DMD has been estimated at 1 in 3,500 (2.9 per 10,000) male births and BMD at 1 in 18,518 (0.5 per 10,000) male births. DBMD are progressive diseases that affect multiple systems including muscular, skeletal, respiratory, and cardiovascular. In addition, social, cognitive and psychological development can be negatively affected by the disease. As a result, a comprehensive, multidisciplinary approach to the care of individuals with DMD is optimal. In order to promote such care, recommendations for treatments and services for individuals with DMD have been developed and include those by the American Academy of Neurology and Child Neurology Society on corticosteroid treatment, American Thoracic Society on respiratory care, American Academy of Pediatrics on cardiac surveillance recommendations and, more recently, the 2010 Care Considerations on whole care developed by panels of experts in the care of DMD and coordinated by the US Centers for Disease Control and Prevention (CDC). Over the past decade, the Muscular Dystrophy Surveillance Tracking and Research Network (MD STARnet) has conducted active, population-based surveillance of DBMD to characterize the epidemiology and develop long-term follow-up and tracking of children with DBMD across four sites in Arizona, Colorado, Iowa, and western New York. For the current project, we propose to utilize and update data collected by the MD STARnet to systematically evaluate agreement between treatments and services provided to individuals who have DMD and compare treatment received to the recommendations documented in the 2010 DMD Care Considerations. This will identify gaps between recommended standards of care and actual care received. Additionally, we will survey health care providers identified by the MD STARnet to identify barriers and challenges involved in the delivery of treatments and services. The geographical area covered by the four sites of the MD STARnet mirrors the socio-demographic characteristics of people with DMD in the United States. Although international efforts to examine the uptake of recommended care in DMD are underway (e.g., TREAT-NMD, Canadian Pediatric Neuromuscular group), similar efforts have not been undertaken in the United States. Our proposed project will allow a comparative evaluation of the implementation of the DMD Care Considerations in a population-based sample of individuals with DMD within the US. This project has the potential of having a major public health impact by evaluating the uptake of care considerations and identifying barriers that may prevent the delivery of recommended health care to individuals with DMD. The dissemination of the findings will inform health care providers dedicated to DMD of the potential disparities between actual and recommended care and provide potential solutions for reducing these discrepancies.