Univ. of Miami Comprehensive Sickle Cell Center: Basic & Translational Research

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DESCRIPTION (provided by applicant): The aims of this Comprehensive Sickle Cell Center are to (1) establish a research center that promotes collaboration to conduct clinical trials in clinical research in sickle cell disease; (2) rapidly advance access to effective treatment by participating in multi-center Phase II and high-risk clinical trials; (3) improve access to quality sickle cell care throughout the region through the implementation of strong clinical and patient services cores, (4) carry out clinical, patient services, and translation research studies in advance knowledge about the treatment of sickle cell disease, and (5) contribute to the future workforce of professionals who are able to conduct research and provide high-quality service to patients with sickle cell. We will establish (a) collaborative relationships with other programs to provide services to children and adults with sickle cell disease for the purpose of conducting research and disseminating information to the community; (b) a clinical core and will provide the necessary infrastructure for participating in clinical research, and will provide a common resource for assessing stroke risk for all participants in our studies; and (c) a patient services core that will enhance the capacity and outcomes of the newborn screening program, provide just-in-time information about sickle cell disease on the Internet to the community that interacts with individuals with sickle cell disease, and improve continuity of care by establishing a transition program between adolescent and young adult care. We will conduct (a) a clinical research study that focuses on safety and effectiveness of bosenten in the treatment of sickle pain, (b) a patient services research study evaluating an intervention to improve academic achievement, reduce family conflict, and improve health status, and (c) a translational study evaluating the role hypoxia on brain development and the application of this information to an intervention using oxygen supplementation to improve cognitive function in children with chronic hypoxia. We will also establish a training program in sickle cell disease for doctoral-level professionals, and promote careers in sickle cell disease through a high school summer program. This will all be accomplished by an integrated team of basic, clinical, and behavioral scientist working at the University of Miami and in collaboration with other centers. The public health benefit of this project will be improved access to knowledge, new approaches to care, and opportunities to advance knowledge by participation in research.