Clinical Trials in Transfusion Medicine and Hemostasis

描述（由申请人提供）： 医疗保健的持续发展、高科技疗法的采用以及血库技术的进步导致了输血治疗的创新。 关于使用新型血液制品和止血疾病管理的明智决策，需要建立一个国家临床试验网络来评估新疗法。 明尼苏达大学在输血医学和止血临床试验方面有着悠久的成功历史。 关键临床服务与止血和输血医学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，可以成功生产新型血液制品或提供新的止血疗法。 因此，我们提出了一个输血医学和止血协议，其中每一个都解决了重要的临床问题，需要在一个多中心网络的科学研究。 初步研究表明，血小板的剂量大大高于或低于目前的做法可能会导致血小板的需要，同时保持足够的止血在血小板减少症患者的整体减少。 因此，我们的第一个试验将比较两倍大和一半大的血小板剂量与目前的标准剂量的血小板，以维持血小板减少症患者的止血能力。 第二项临床试验将比较血浆置换与高剂量类固醇治疗继发于实体器官或干细胞移植的血栓性微血管病（TM）。 血浆置换对血栓性血小板减少性紫癜患者明显有益，但继发于移植的TM具有不同的病理生理机制，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种困难的临床情况的管理的理解，并将确定这些疗法中哪一种最有效。 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，支持性资源描述确立了我们提供新型血液成分和成功开展临床试验的能力。 该项目的研究人员和明尼苏达大学主要临床项目的其他领导者有兴趣并愿意参与这些和其他可能通过该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.