Clinical Trials in Transfusion Medicine and Hemostasis

描述(由申请人提供)： 医疗保健的不断发展、高科技疗法的采用以及血库技术的进步导致了输血疗法的创新。关于使用新的血液产品和止血疾病管理的明智决定产生了对国家临床试验网络的需求，以评估新的治疗方法。明尼苏达大学在成功的输血医学和止血临床试验方面有着悠久的历史。重点临床服务与止血、输液药物之间存在良好的生产关系。通过我们的分子和细胞设施和细胞治疗实验室以及与传统血液供应商的良好关系，可以获得成功生产新型血液产品或为止血条件提供新疗法的资源。因此，我们提出了一种输血药物和一种止血方案，每一种方案都解决了需要在多中心网络中进行科学研究的重要临床问题。初步研究表明，大大高于或低于目前做法的血小板剂量可能会导致总体上减少对血小板的需求，同时保持对血小板减少症患者的足够止血。因此，我们的第一个拟议试验将比较两倍和一半大小的血小板剂量与目前标准剂量的血小板在血小板减少患者中维持止血的能力。第二项临床试验将比较血浆置换和大剂量类固醇治疗实体器官或干细胞移植继发的血栓性微血管病(TM)。 血浆置换对血栓性血小板减少性紫癜患者有明显的益处，但移植后继发的TM有不同的病理生理机制，对于这种形式的TM的最佳治疗方法尚不清楚。这项试验将增加对这一困难临床情况的处理的理解，并将确定这些疗法中的哪一种最有效。我们相信这些临床试验解决了两个极其重要的问题：它们的设计说明了我们开发临床试验的能力，而支持性资源说明确立了我们提供新的血液成分和成功进行临床试验的能力。该项目的研究人员和明尼苏达大学主要临床项目的其他负责人有兴趣并愿意参与这些和其他可能通过这个网络开发的临床试验。

DESCRIPTION (provided by applicant): The continuing evolution in medical care, adoption of high technology therapies, and advances in blood banking technology have led to innovations in transfusion therapy. Wise decisions about the use of novel blood products and the management of hemostatic disorders has created a need for a national clinical trials network to evaluate new therapies. The University of Minnesota has a long history of successful transfusion medicine and hemostasis clinical trials. There is an excellent productive relationship between key clinical services and hemostasis and transfusion medicine. The resources to successfully produce novel blood products or to provide new therapies for hemostatic conditions are available through our Molecular and Cellular Facility and Cell Therapy Laboratory and through excellent relationships with traditional blood suppliers. Therefore we propose a transfusion medicine and a hemostasis protocol each of which addresses important clinical issues that need scientific study in a muiticenter network. Preliminary studies have suggested that doses of platelets substantially higher or lower than present practice might lead to an overall reduction in the need for platelets while maintaining adequate hemostasis in thrombocytopenic patients. Therefore, our first proposed trial will compare doses of platelets two times larger and half as large with the present standard dose of platelets for their ability to maintain hemostasis in thrombocytopenic patients. The second clinical trial will compare plasma exchange with high dose steroids for the treatment of thrombotic microangiopathy (TM) secondary to solid organ or stem cell transplantation. Plasma exchange is clearly beneficial in patients with thrombotic thrombocytopenic purpura but TM secondary to transplantation has a different pathophysiologic mechanism and the optimum treatment for this form of TM is not known. This trial will add to the understanding of the management of this difficult clinical situation and will determine which of these therapies is most efficacious. We believe these clinical trials address two extremely important issues; their design illustrates our ability to develop clinical trials and the supportive resource description establishes our ability to provide novel blood components and carry out successful clinical trials. The investigators in this project and other leaders of major clinical programs at the University of Minnesota are interested and willing to participate in these and other clinical trials that may be developed through this network.