TRANSFUSION MEDICINE/HEMOSTASIS TRIAL SITE AT PSBC

描述(由申请人提供)： 建议在普吉特湾血液中心(PSBC)设立一个输血医学/止血临床试验场。PSBC是一个地区性血液计划，将所有血液和血液产品分发给西雅图和周围县的患者。预计网络临床试验将在西雅图6家医院中的任何一家进行，这些医院总共有2193张床位，2001年期间，这些医院的20996名患者接受了超过15万种血液产品。总部设在PSBC的四名华盛顿大学医学院教职员工在临床试验方面拥有丰富的经验，他们将与其他试验地点一起参与网络临床试验。这些内科科学家在他们感兴趣的研究领域都是公认的专家，即血小板和粒细胞输注治疗、分离程序及其适应症、止血和血栓形成障碍以及ITP患者。这些医生监督PSBC专门从事血友病护理的止血诊所、凝血实验室、血小板抗体实验室和分离设施。通过这些临床活动，他们了解到除了地区医院的患者外，有各种疾病的患者可能是参加网络临床试验的候选人。 建议进行两个网络临床试验：1)评价低剂量与标准剂量血小板疗法的止血效果和血小板利用率；2)多中心随机对照试验，观察粒细胞集落刺激因子动员的粒细胞输注治疗感染中性粒细胞减少的疗效。第一个试验试图确定与标准剂量相比，低剂量的血小板输注是否能够维持足够的止血，同时减少支持血小板减少患者所需的血小板总数。主要终点将是止血；即，证明随机使用低剂量组的患者在发生WHO二级出血的患者百分比方面并不低于标准闭合组。 第二个试验将评估粒细胞输注治疗的有效性，以确定G-CSF动员的粒细胞输注对中性粒细胞减少症细菌或真菌感染患者的临床疗效。患者将随机接受每日粒细胞输注，同时接受标准护理或仅接受标准护理。主要终点将是每一组患者的比例，这些患者对他们的感染表现出微生物反应，并且在进入研究后至少存活28天。

DESCRIPTION (provided by applicant): A Transfusion Medicine/Hemostasis Clinical Trial Site to be located at the Puget Sound Blood Center (PSBC) is proposed. PSBC is a regional blood program that distributes all blood and blood products to patients in Seattle and surrounding counties. It is anticipated that network clinical trials will be conducted at any of 6 Seattle hospitals that have a total of 2,193 beds, and, during 2001, 20,996 patients in these hospitals received over 150,000 blood products. Four University of Washington medical school faculty, based at PSBC, with extensive experience in clinical trials will participate with other trial sites in the network clinical trials. These physician scientists are all recognized experts in their areas of research interest; i.e., platelet and granulocyte transfusion therapy, apheresis procedures and their indications, disorders of hemostasis and thrombosis, and patients with ITP. These physicians supervise a hemostasis clinic at PSBC that specializes in hemophilia care, a coagulation laboratory, a platelet antibody laboratory, and an apheresis facility. Through these clinical activities, they are aware of patients with a variety of disorders - besides those in area hospitals - who might be candidates for participation in network clinical trials. Two network clinical trials are proposed: 1) "Evaluate The Hemostatic Efficacy And Platelet Utilization Rates Of Low Versus Standard Dose Platelet Therapy;" and 2) A Multicenter Randomized Controlled Trial On The Efficacy Of Transfusion Of G-CSF Mobilized Granulocytes To Neutropenic Patients With Infection." The first trial seeks to determine whether low dose compared to standard dose platelet transfusions are able to maintain adequate hemostasis while decreasing the total number of platelets required to support thrombocytopenic patients. The primary endpoint will be hemostasis; i.e., to demonstrate non-inferiority of patients randomized to the low dose arm compared to those in the standard close arm in the percentage of patients that develop WHO Grade 2 bleeding. The second trial will evaluate the effectiveness of granulocyte transfusion therapy to determine the clinical efficacy of G-CSF mobilized granulocyte transfusions for neutropenic patients with bacteria or fungal infections. Patients will be randomized to receive daily granulocyte transfusions along with standard care or standard care alone. The primary endpoint will be the proportion of patients in each arm who demonstrate both a microbial response to their infection and who also survive at least 28 days from study entry.