Comprehensive Sickle Cell Center at JHU and UAB

描述(由申请人提供)：约翰霍普金斯大学和阿拉巴马大学的新的综合镰状细胞中心(JHU/UAB CSCC)将代表着两个机构之间的合作，这两个机构在研究、患者服务和临床护理方面为镰状细胞病(SCD)患者提供了良好的记录。该中心将有3个目标：1)通过JHU和UAB独立但互补的临床中心，在各自的机构为成人和儿科SCD患者提供最先进的综合护理，以及在两个机构进行中心间协作和中心内翻译研究的基础设施；2)通过我们的患者服务核心，通过评估、协调和扩大对SCD儿童和成人及其家属的诊断、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，在两所大学开展和促进与SCD相关的新的基础科学和临床研究，并加强JHU、UAB和其他中心之间的合作。总部设在JHU的行政核心将协调和实施地方和协作的CSCC活动。提出了四个研究课题。我们的中心间试验将评估羟基脲治疗对有条件的经颅多普勒测量患者的有效性。这项试验有可能避免输血的需要，并在很大比例的SCD患者中提供中风的一级预防，这些患者面临这种常见和破坏性并发症的风险。我们的患者服务项目将探索当地公共卫生服务对SCD死亡率结果的影响。通过这个项目，我们将确定可能导致SCD死亡率在州与州之间存在显著差异的具有高影响力的关键公共卫生干预措施。我们的基础科学项目将利用独特的资源(SIT Trail生物库和JHU蛋白质组学中心)来研究无症状性脑梗塞的潜在生物标记物，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关于NO和氧化损伤在异常勃起中的作用的基础研究观察，为一项临床试验提供信息，该临床试验将评估体内NO功能调节剂西地那非治疗SCD异常勃起的效果。我们中心将利用JHU/UAB联合环境下丰富的基础科学和临床研究资源，改善当地和全国SCD患者的护理、服务和生活质量。

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.