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

描述(由申请人提供)： 这个综合镰刀细胞中心的目标是(1)建立一个研究中心，促进合作进行镰状细胞病临床研究的临床试验；(2)通过参与多中心II期和高风险临床试验，迅速促进获得有效治疗的机会；(3)通过实施强大的临床和患者服务核心，改善整个区域获得优质镰状细胞护理的机会；(4)提前开展临床、患者服务和翻译研究研究；以及(5)促进未来能够开展研究并为镰状细胞患者提供高质量服务的专业人员队伍。我们将建立(A)与其他项目的合作关系，为患有镰状细胞疾病的儿童和成人提供服务，以进行研究并向社区传播信息；(B)临床核心，并将为参与临床研究提供必要的基础设施，并将为我们研究中的所有参与者提供评估中风风险的共同资源；以及(C)患者服务核心，将加强新生儿筛查计划的能力和结果，在互联网上向与镰状细胞疾病患者互动的社区提供及时的镰状细胞疾病信息，并通过建立青少年和年轻成人护理之间的过渡计划来改善护理的连续性。我们将进行(A)一项临床研究研究，重点是波生丁治疗镰刀痛的安全性和有效性；(B)一项患者服务研究，评估一项旨在提高学业成绩、减少家庭冲突和改善健康状况的干预措施；以及(C)一项转译研究，评估低氧对大脑发育的作用，并将这一信息应用于使用氧气补充以改善慢性低氧儿童认知功能的干预措施。我们还将为博士级专业人员建立镰状细胞病培训计划，并通过高中暑期计划促进镰状细胞病的职业生涯。这一切都将由迈阿密大学基础、临床和行为科学家组成的综合团队完成，并与其他中心合作。该项目的公共卫生效益将是更好地获得知识、新的护理方法以及通过参与研究促进知识发展的机会。

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.