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，以及全世界大约 500 万人。仅用了一代人的时间，美国纯合血红蛋白 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 患者存活时间越来越长，仍然迫切需要了解如何预防和治疗越来越多的 SCD 引起的内脏器官损伤患者。因此，我们的具体目标是： (1) 在由杜克大学和北卡罗来纳大学教堂山分校组成的 SCD 临床研究网络中建立一个临床研究中心。 东卡罗来纳大学也将按人头参与临床研究。我们的使命是促进和开展旨在开发新的和改进的 SCD 治疗方法的所有类型的临床研究。这些站点共同代表了才华横溢的研究人员、接触患者群体的机会以及进行高质量 SCD 临床研究所需的基础设施。这三个机构在 SCD 方面有着悠久的合作研究历史，并拥有协调活动的行政结构。 (2) 参与SCD临床研究网络。这将包括与网络的指导委员会和小组委员会合作，确保对各个中心提出的研究进行适当审查，并制定共同的实施方案。我们还将与结果研究核心、数据协调中心和网络中的其他临床中心合作，在杜克大学-北卡罗来纳大学中心高效地启动和完成常见的临床研究试验。我们将根据每位患者的比率以及登记和随访以及完成每项研究的受试者的实际数量来接受研究支持奖励。 (3) 开发并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 及其合作研究人员开发，并提交给网络指导委员会。我们为此提案准备了两项临床研究试验。研究1将研究是否可以通过血管紧张素受体阻断来减缓或逆转由SCD高输出量状态引起的心脏重塑，从而改善心脏储备并防止进展为高输出量心力衰竭。研究 2 旨在调查低强度抗凝治疗在预防 SCD 患者血管闭塞发作方面的效用。将根据患者群体、可行性以及对患者护理的影响向网络提出更多研究。我们还在本申请中纳入了患者结果研究核心的提案。杜克大学-北卡罗来纳大学 SCD 临床研究中心致力于开发和开展极其重要的临床研究 以提高预防和治疗水平。我们拥有实现网络目标所需的患者群体、丰富的临床研究经验和专业知识以及才华横溢的研究人员。 （摘要完） 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