Duke-UNC Sickle Cell Disease Clinical Research Network

杜克大学-北卡罗来纳大学镰状细胞病临床研究网络

• 批准号: 7393087
• 负责人: Kevin Alan Schulman
• 金额: $ 24.37万
• 依托单位: DUKE UNIVERSITY
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-17 至 2011-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7393087
• 关键词: Adhesives; Adult; Affect; Age; American; Angiotensin Receptor; Anticoagulant therapy; Anticoagulation; Award; Blood Platelets; Cardiac; Cause of Death; Cessation of life; Child; Childhood; Chronic; Clinical; Clinical Research; Clinical Trials; Collaborations; Commit; Conduct Clinical Trials; Data Coordinating Center; Dehydration; Development; Diagnosis; Enrollment; Ensure; Erythrocytes; Functional disorder; Generations; Genetic; Goals; Guidelines; Heart failure; Hemoglobin; Hemoglobin SS; Individual; Injury; Institution; Learning; Leukocytes; Life; Life Expectancy; Medical center; Metabolism; Mission; Multiple Organ Failure; Network-based; Nitric Oxide; North Carolina; Numbers; Organ; Outcome; Outcomes Research; Output; Patient Care; Patients; Pharmacologic Substance; Population; Prevention; Process; Protocols documentation; Pulmonary Hypertension; Range; Rate; Recording of previous events; Reperfusion Injury; Reporting; Research; Research Design; Research Infrastructure; Research Personnel; Role; Sickle Cell; Sickle Cell Anemia; Sickle Hemoglobin; Site; Structure; Support of Research; Symptoms; Therapeutic; Thinking; Thrombophilia; Universities; Vascular Endothelium; Work; abstracting; acute chest syndrome; base; capitate bone; design; experience; hydroxyurea; improved; polymerization; prevent; response; sickling

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

描述（由申请人提供）： 镰状细胞疾病（SCD）影响了大约91,000名美国人，大约有500万人。在仅一代人中，美国纯合血红蛋白SS患者的中位存活率已从14年增加到近50年。随着越来越多的SCD患者的生存更长的时间，仍然需要学习如何预防和治疗SCD导致内龙损伤的患者数量增加。因此，我们的具体目的是：（1）在SCD临床研究网络中建立一个临床研究中心，包括杜克大学和UNC教堂山。 东卡罗来纳大学还将在临床研究中参与。我们的任务是促进和进行所有类型的临床研究，旨在为SCD开发新的和改进的治疗方法。这些站点共同代表有才华的研究人员，与患者人群的访问权以及在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方面有悠久的协作研究历史和协调活动的行政结构。 （2）参加SCD临床研究网络。这将包括与指导委员会和网络小组委员会合作，以确保对各个中心的拟议研究进行适当的审查以及开发常见协议以实施。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一起工作，以有效地开始并完成Duke-unc Center的常见临床研究试验。我们将基于每个患病率以及已入学和遵循的受试者的实际数量以及完成每项研究的实际数量来支持研究奖励。 （3）开发并提出了多中心临床研究试验，旨在识别和验证SCD及其后遗症的治疗选择。 PI及其共同研究人员将开发临床试验，以提交网络指导委员会。我们已经为这项建议准备了两项临床研究试验。研究1将研究因血管紧张素受体阻滞而导致高输出型SCD输出状态引起的心脏重塑，从而改善心脏储备并防止进展到高输出心力衰竭。研究2旨在研究低强度抗凝治疗的实用性，以防止SCD患者的血管熟悉发作。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。我们还将在此应用程序中包括了针对患者结果研究核心的建议。 Duke-unc unc SCD临床研究中心致力于开发和传导至关重要的临床研究 改善预防和治疗。我们拥有患者人群，强大的临床研究经验和专业知识，以及实现网络目标所需的才华横溢的研究人员。 （抽象的结尾） 临床中心

项目成果

Outcomes of inpatients with and without sickle cell disease after high-volume surgical procedures.

患有和不患有镰状细胞病的住院患者在大容量外科手术后的结果。

• DOI: 10.1002/ajh.21520
• 发表时间: 2009
• 期刊: American journal of hematology
• 影响因子: 12.8
• 作者: Dinan,MichaelaA;Chou,Chia-Hung;Hammill,BradleyG;Graham,FeliciaL;Schulman,KevinA;Telen,MarilynJ;Reed,ShelbyD
• 通讯作者: Reed,ShelbyD