Duke-UNC Sickle Cell Disease Clinical Research Network

描述(由申请人提供)： 镰状细胞病(SCD)影响着大约91,000名美国人，可能是全球约500万人。仅在一代人的时间里，美国纯合子血红蛋白SS患者的中位生存期就从14年增加到近50年。随着越来越多的SCD患者存活时间更长，仍然迫切需要了解如何预防和治疗越来越多的SCD导致的内脏损伤患者。因此，我们的具体目标是：(1)在SCD临床研究网络中建立一个临床研究中心，由杜克大学和北卡罗来纳大学教堂山分校组成。东卡罗来纳大学也将参与临床研究。我们的使命是促进和开展旨在开发SCD新的和改进的治疗方法的所有类型的临床研究。这些网站共同代表了才华横溢的研究人员、接触患者群体的途径以及开展SCD高质量临床研究所需的基础设施。这三个机构在SCD方面的合作研究有很长的历史，并有协调活动的行政结构。(2)参与SCD临床研究网络。这将包括与指导委员会和该网络的小组委员会合作，确保适当审查各中心提出的研究报告，并制定共同的执行议定书。我们还将与结果研究核心、数据协调中心和网络的其他临床中心合作，高效地启动和完成杜克-北卡罗来纳大学中心的常见临床研究试验。我们将接受根据每个患者的比率和登记和跟踪的实际受试者数量以及完成每项研究的人的研究支持奖。(3)开发和提出多中心临床研究试验，旨在确定和验证治疗SCD及其后遗症的治疗方案。临床试验将由PI和她的合作调查员开发，并提交给该网络的指导委员会。我们已经为这项提议准备了两项临床研究试验。研究1将探讨血管紧张素受体阻滞剂能否减缓或逆转由SCD的高输出状态引起的心脏重构，从而改善心脏储备，防止进展为高输出心力衰竭。研究2旨在探讨低强度抗凝治疗预防SCD患者血管闭塞的有效性。将根据患者群体、可行性和对患者护理的影响向该网络提出更多研究。我们还在这份申请中包括了一项关于患者结局研究核心的建议。杜克-北卡罗来纳大学SCD临床研究中心致力于开发和开展至关重要的临床研究 加强预防和治疗。我们拥有患者群体、丰富的临床研究经验和专业知识，以及实现网络目标所必需的有才华的研究人员。(摘要结束) 临床中心

DESCRIPTION (provided by applicant): Sickle cell disease (SCD) affects approximately 91,000 Americans and probably about five million people worldwide. In just one generation, the median survival of patients with homozygous hemoglobin SS in the U.S. has increased from 14 years to nearly 50 years. As more and more patients with SCD survive longer, there remains a critical need to learn how to prevent and treat the increasing number of patients with endorgan damage resulting from SCD. Thus our specific aims are to: (1) Establish a Clinical Research Center in the SCD Clinical Research Network, comprising Duke and UNC Chapel Hill. East Carolina University will also participate on a capitated basis in clinical studies. Our mission is to promote and conduct all types of clinical research designed to develop new and improved treatments for SCD. Together these sites represent talented investigators, access to patient populations, and the infrastructure requisite to conduct high quality clinical research in SCD. These three institutions have a long history of collaborative research in SCD and an administrative structure to coordinate activities. (2) Participate in the SCD Clinical Research Network. This will include working with the Steering Committee and subcommittees of the network to ensure appropriate review of proposed studies from individual centers and development of common protocols for implementation. We will also work with the Outcomes Research Core, the Data Coordinating Center and the other Clinical Centers of the network to efficiently start and complete common clinical research trials at the Duke-UNC Center. We will accept awards for the support of research based on per-patient rates and the actual numbers of subjects who are enrolled and followed, and who complete each study. (3) Develop and propose multi-center clinical research trials designed to identify and validate therapeutic options in the treatment of SCD and its sequelae. Clinical trials will be developed by the PI and her co-investigators for submission to the network's Steering Committee. We have prepared two clinical research trials for this proposal. Study 1 will investigate whether cardiac remodeling due to the high output state of SCD can be slowed or reversed with angiotensin receptor blockade, thus improving cardiac reserve and preventing progression to high output heart failure. Study 2 is designed to investigate the utility of low intensity anticoagulation therapy to prevent vaso-occlusive episodes in patients with SCD. Additional studies will be proposed to the Network based on patient populations, feasibility, and impact on patient care. We also include in this application a proposal for a Patient Outcomes Research Core. The Duke-UNC SCD Clinical Research Center is committed to developing and conduction critically important clinical studies to improve prevention and treatment. We have the patient populations, strong clinical research experience and expertise, and talented investigators necessary to accomplish the goals of the Network. (End of Abstract) CLINICAL CENTER