Duke-UNC Sickle Cell Disease Clinical Research Network

描述（由申请人提供）： 镰状细胞病（SCD）影响大约91，000名美国人，全世界可能约有500万人。仅在一代人中，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患者存活时间的延长，仍然迫切需要了解如何预防和治疗越来越多的SCD导致的器官损伤患者。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，包括杜克和查佩尔山。 东卡罗莱纳大学也将以资本为基础参与临床研究。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。这些研究中心共同代表了有才华的研究人员、接触患者群体的机会以及在SCD中进行高质量临床研究所需的基础设施。这三个机构在SCD合作研究方面有着悠久的历史，并有一个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，以确保对各个中心的拟议研究进行适当审查，并制定共同的实施方案。我们还将与成果研究核心，数据协调中心和网络的其他临床中心合作，以有效地启动和完成杜克大学中心的常见临床研究试验。我们将根据每例患者的比率和实际入组和随访的受试者人数以及完成每项研究的受试者人数接受奖励。(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及其合作研究者开发，并提交给网络指导委员会。我们已为该提案准备了两项临床研究试验。研究1将研究血管紧张素受体阻滞剂是否可以减缓或逆转SCD高输出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心力衰竭。研究2旨在研究低强度抗凝治疗预防SCD患者血管闭塞发作的效用。将根据患者人群、可行性和对患者护理的影响向网络提出其他研究。我们还包括在本申请中的一个病人的结果研究核心的建议。杜克大学SCD临床研究中心致力于开发和进行至关重要的临床研究 改善预防和治疗。我们有患者群体，强大的临床研究经验和专业知识，以及完成网络目标所需的优秀研究人员。(End摘要） 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