Comprehensive Sickle Cell Center at JHU and UAB

约翰霍普金斯大学和阿拉巴马大学综合镰状细胞中心

• 批准号: 7640587
• 负责人: James F Casella
• 金额: $ 138.97万
• 依托单位: JOHNS HOPKINS UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-06-18 至 2012-01-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7640587
• 关键词: Comprehensive; Sickle; Cell; Center; JHU

DESCRIPTION (provided by applicant): The new Comprehensive Sickle Cell Center at Johns Hopkins University and University of Alabama (JHU/UAB CSCC) will represent a collaboration between two institutions with strong records of research, patient services and clinical care for patients with sickle cell disease (SCD). The center will have 3 goals: 1) through separate but complementary Clinical Cores at both JHU and UAB, to provide state-of-the-art comprehensive care for adult and pediatric SCD patients at their respective institutions, plus infrastructure to carry out inter-Center collaborative and intra-Center translational research at both institutions; 2) through our Patient Services Core, to provide high quality and improved patient services by evaluating, coordinating, and expanding diagnostic, educational, counseling and legal services for children and adults with SCD and their families, 3) through our Research Projects, to carry out and stimulate new SCD-related basic science and clinical research within both universities and increase collaborations between JHU, UAB, and other Centers. An Administrative Core based at JHU will coordinate and implement local and collaborative CSCC activities. Four research projects are proposed. Our inter-Center trial, will assess the usefulness of hydroxyurea therapy for patients with conditional transcranial Doppler measurements. This trial has the potential to prevent the need for transfusions and provide primary prevention for stroke in a large percentage of patients with SCD at risk for this common and devastating complication. Our Patient Services Project will explore the effect of local public health services on mortality outcomes for SCD. Through this project, we will identify the critical public health interventions of high impact that may account for the marked state-to-state variability in SCD mortality. Our Basic Science Project will take advantage of unique resources (the SIT Trail Biologic Repository and JHU Center for Proteomics) to study potential biomarkers for silent cerebral infarction, a leading cause of neurologic morbidity in SCD. Our Translational Project will exploit ongoing basic research observations on the role of NO and oxidative damage in priapism, to inform a clinical trial that will assess the effect of sildenafil, a modulator of NO function in vivo, for the treatment of priapism in SCD. Our center will take advantage of the rich basic science and clinical research resources in the combined JHU/UAB environment to improve care, services and quality of life for SCD patients locally and nationally.

描述（由申请人提供）：约翰·霍普金斯大学和阿拉巴马大学（JHU/UAB CSCC）的新综合镰状细胞中心将代表两个机构之间为镰状细胞疾病患者（SCD）的研究，患者服务和临床护理的两家机构之间的合作。该中心将有3个目标：1）通过JHU和UAB的单独但互补的临床核心，为各自机构的成人和儿科SCD患者提供最新的综合护理，以及在这两个机构的基础设施中进行基础设施，以进行中心间协作和内部中心的转化； 2）通过我们的患者服务核心，通过评估，与SCD及其家人的儿童和成人评估，协调，协调，教育，教育，咨询和法律服务，提供高质量和改进的患者服务，3）通过我们的研究项目，通过我们的研究项目，刺激并刺激新的SCD与SCD相关的基础科学和临床研究，并增加了大学内的协作，并增加了JHU，JHU，UAB，UAB，UAB，UAB和其他Centers。基于JHU的行政核心将协调并实施本地和协作的CSCC活动。提出了四个研究项目。我们的中心试验将评估有条件经颅多普勒测量的患者羟基脲治疗的有用性。该试验有可能防止输血的需求，并在很大一部分患有这种常见和毁灭性并发症的SCD患者中为中风提供初级预防。我们的患者服务项目将探讨当地公共卫生服务对SCD死亡率结果的影响。通过该项目，我们将确定对高影响力的关键公共卫生干预措施，这些干预措施可能解释了SCD死亡率的州对州的明显变异性。我们的基础科学项目将利用独特的资源（SIT TRAIL生物学存储库和JHU蛋白质组学中心）来研究沉默的脑梗塞的潜在生物标志物，这是SCD神经系统发病率的主要原因。我们的翻译项目将利用正在进行的基础研究观察结果，内容涉及NO和氧化损害在Priapism中的作用，以告知一项临床试验，该试验将评估Sildenafil（在体内NO功能的调节剂）对SCD中的Priapism治疗的效果。我们的中心将利用JHU/UAB环境中丰富的基础科学和临床研究资源，以改善本地和全国SCD患者的护理，服务和生活质量。