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新的综合镰状细胞中心（JHU/UAB CSCC）将代表两家机构之间的合作，这两家机构在镰状细胞病（SCD）患者的研究、患者服务和临床护理方面有着良好的记录。该中心将有3个目标：1)通过JHU和UAB的独立但互补的临床核心，为各自机构的成人和儿科SCD患者提供最先进的综合护理，以及在两个机构开展中心间合作和中心内转化研究的基础设施；2)通过我们的患者服务核心，通过评估、协调和扩大对患有SCD的儿童和成人及其家庭的诊断、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新的SCD相关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CSCC活动。提出了四个研究项目。我们的中心间试验将评估羟基脲治疗有条件经颅多普勒测量患者的有效性。这项试验有可能防止输血的需要，并为很大比例的有这种常见和破坏性并发症风险的SCD患者提供卒中的初级预防。我们的病人服务计划将探讨本地公共卫生服务对慢性阻塞性肺病死亡率的影响。通过这个项目，我们将确定具有重大影响的关键公共卫生干预措施，这些干预措施可能解释SCD死亡率在州与州之间的显著差异。我们的基础科学项目将利用独特的资源（SIT Trail biological Repository和JHU Center for Proteomics）来研究无症状脑梗死的潜在生物标志物，无症状脑梗死是SCD中神经系统疾病的主要原因。我们的转化项目将利用正在进行的关于NO和氧化损伤在阴茎勃起功能障碍中的作用的基础研究观察结果，为临床试验提供信息，以评估西地那非（一种体内NO功能调节剂）治疗SCD阴茎勃起功能障碍的效果。我们的中心将利用JHU/UAB联合环境中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.