The Hawai'i Muscular Dystrophy Surveillance, Tracking, and Research Network (Hawai'i MD STARnet) Project will contribute to the common goals of the existing multi-state MD STARnet Project by collaboratively using traditional population based public health surveillance and research approaches to answer questions about Duchenne and Becker Muscular Dystrophy (DBMD) such as: ? ? How common is DBMD? ? ? Is DBMD equally common in different racial ethnic groups? ? ? What are the early signs and symptoms of DBMD? ? ? Do factors such as type of care or type of mutation affect progression of DBMD? ? ? What services are families receiving? ? ? Do different populations receive different care? ? ? According to the 2006 American Community Survey, 4.4% percent of the U.S. population reported Asian ethnicity alone and 0.14% reported Native Hawaiian/Pacific Island ancestry. Currently, MD STARnet participants of Asian or Pacific Island descent make up only 1% of the study sample which is not representative of the national population. Adding Hawai'i and Guam as MD STARnet sites will increase the number of Asian and Pacific Island participants within the study population. Data gathered by project staff will characterize the complications and long term outcomes of individuals with DBMD who are geographically isolated and may face barriers such as lack of access to health care or information about the management of DBMD within protocol standards. In addition, the project will describe the history and outcome of individuals with DBMD who receive different levels of medical and preventative care. Finally, the culture of Hawai'i and Guam may provide insight into unique methods of caring for children and families with DBMD. The Hawai'i MD STARnet Project will follow the established protocols and procedures of the existing multistate MD STARnet Project. Project participants in Hawai'i and Guam will be recruited through partnerships with Department of Health Programs (Birth Defects, Children with Special Health Needs, and Early Intervention Services), Muscular Dystrophy Association (MDA), MDA clinics, Shriners' Hospital, neuromuscular clinics, genetics clinics, and other pediatric and neurological practices. After obtaining informed consent, data will be gathered by project staff via chart review, family interviews, and long term follow-up of participating families. Hawai'i and Guam data will be de-identified and transferred to the central MD STARnet Data Coordinating Center (DCC) for data analysis and evaluation. The Hawai'i MD STARnet project staff will participate in the evaluation of the data, developing publications, and disseminating the results of the project. ? ? ? ?
Data from the MD STARnet project will define the prevalence of DBMD, as well as the factors affecting disease progression. The results of this research study can have significant national public health and health care implications as we identify: the progression of the disease correlated with treatment protocols;possible differences in disease manifestation in various ethnic groups;genetic and management issues that may affect mild versus severe forms of DBMD;and services or resources that may improve outcomes. The involvement of Hawai`i and Guam as MD STARnet project collaborators will enrich the study sample by providing data from Asian and Pacific Islander populations which are minimally represented in the existing database.
| Conway, Kristin M; Ciafaloni, Emma; Matthews, Dennis et al. (2018) Application of the International Classification of Functioning, Disability and Health system to symptoms of the Duchenne and Becker muscular dystrophies. Disabil Rehabil 40:1773-1780 |
| Gissy, Jacob J; Johnson, Teresa; Fox, Deborah J et al. (2017) Delayed onset of ambulation in boys with Duchenne muscular dystrophy: Potential use as an endpoint in clinical trials. Neuromuscul Disord 27:905-910 |
