Hemostasis Clinical Research Network Protocols

描述(由申请人提供)： 患有止血障碍的患者面临长期的问题和风险、艰难的治疗决定以及与治疗本身相关的风险。这些风险和并发症中的许多可以通过适当的干预策略来减少或预防。在治疗发病率相对较低的出血性疾病患者方面的进展要求：1)临床研究利用多机构合作网络，以获得足够数量的患者以获得有意义的答案；2)教育患者参与此类研究的极端重要性。该项目的目的是在杜兰大学开发一个止血核心临床研究单位，作为NIH输血医学/止血临床研究网络的一部分。这个单位将利用杜兰出血障碍中心的一些基本基础设施和服务来开发，该中心致力于护理和促进止血障碍患者的发展。一旦成立，这个核心研究单位将从出血疾病中心、骨髓移植单位以及整个杜兰附属医院和诊所网络提供止血患者的临床试验。此外，作为这项提案的一部分，已经制定了两项临床试验方案。首先，一项关于激活的凝血酶原复合体浓缩物(APCCs)预防使用抑制剂的血友病A患者的前瞻性随机交叉研究，是一项长期方案，旨在评估APCCs在预防这一非常复杂的患者组中的出血效果。第二个短期方案，利巴韦林治疗HIV相关性免疫性血小板减少性紫癜(ITP)的前瞻性研究，旨在测试一种治疗HIV-ITP的新药理药物。

DESCRIPTION (provided by applicant): Patients with disorders of hemostasis are subject to long term problems and risks, difficult treatment decisions and risks associated with treatment itself. Many of these risks and complications can be lessened or prevented by appropriate intervention strategies. Advances in the management of patients with bleeding disorders of relatively low incidence require: 1) that clinical studies utilize a multi-institutional cooperative network in order to accrue sufficient numbers of patients to achieve meaningful answers; and 2) that patients be educated on the vital importance of participation in such studies. The purpose of this project is to develop a Hemostasis Core Clinical Research Unit at Tulane University as part of the NIH Transfusion Medicine/Hemostasis Clinical Research Network. This Unit will be developed using some of the basic infrastructure and services of the Tulane Center for Bleeding Disorders, which is dedicated to the care and advancement of patients with disorders of hemostasis. Once established, this Core Research Unit will provide access to clinical trials for hemostasis patients from the Center for Bleeding Disorders, the Bone Marrow Transplant Unit and from throughout the network of Tulane affiliated hospitals and clinics. In addition, two clinical trial protocols have been developed as part of this proposal. The first, A prospective randomized crossover study of activated prothrombin complex concentrates (aPCCs) as prophylactic therapy in hemophilia A patients with inhibitors, is a long-term protocol designed to evaluate the efficacy of aPCCs in the prevention of bleeds in this very complicated group of patients. The second, short-term protocol, A prospective study of ribavirin for the treatment of HIVassociated immunopathic thrombocytopenic purpura (ITP), was developed to test a novel pharmacologic agent in the management of HIV-ITP.