Transfusion Medicine/Hemostasis Clinical Research Netwo*

描述(由申请人提供)： 血液学和止血障碍是导致住院、发病率和死亡率的常见原因。然而，这些疾病单独考虑是不常见的，这一事实使它们很难在医疗保健系统的背景下进行研究，在这种情况下，罕见疾病的治疗没有标准化，单一机构的研究不能积累足够数量的患者来得出有意义的结论。出于这个原因，需要一个中心网络来合作研究这种障碍。在本应用程序中，我们将追求两个主要目标。首先，我们将概述凯斯西储大学医学院(CWRU)/克利夫兰大学医院(UHC)在止血和输血医学方面的临床研究活动，并展示将CWRU/UHC纳入止血/输血医学临床研究联盟的好处。我们机构在这方面的优势包括1)在这里接受治疗的大量患有先天性和获得性血液病的成人和儿童患者；2)CWRU/UHC癌症中心，NCI指定的综合性癌症中心，通过它可以提供完善的临床研究基础设施来管理和监督我们的研究活动；以及3)我们的教职员工在召集患者参加NIH赞助的临床试验方面的经验和记录。我们的第二个目标将是为研究两种常见的血小板减少性疾病提供全面的理论基础和实验方法，这两种疾病是肝素相关性血小板减少症(HIT)和免疫性血小板减少性紫癜(ITP)。在HIT方面，我们将评估新诊断的HIT和“无症状血小板减少症”患者亚临床血栓形成的发生率，并评估这些患者是否需要替代抗凝。我们假设这种疗法并不是在所有情况下都是必要的。我们还将直接比较鲁鲁丹和阿格曲班在预防该患者组血栓形成方面的疗效。在我们的第二个项目中，我们将评估大量ITP患者中Hp感染的发生率，并确定根除Hp是否会导致ITP的缓解。我们假设许多ITP患者会对这种干预有反应。这些研究的成功完成将为这些疾病的管理提供重要的见解，这将对他们的管理产生重要和直接的影响。

DESCRIPTION (provided by applicant): Hematologic and hemostatic disorders are common causes of hospital admissions, morbidity and mortality. However, these disorders, considered individually, are uncommon, a fact that has made them difficult to study in the context of a health care system in which therapy of rare disorders is not standardized and single institution studies cannot accrue sufficient numbers of patients to reach meaningful conclusions. For this reason, a network of centers to collaboratively study such disorders is needed. In this application, we will pursue two primary goals. First, we will provide an overview of clinical research activity in hemostasis and transfusion medicine at Case Western Reserve University School of Medicine (CWRU)/University Hospitals of Cleveland (UHC), and demonstrate the benefits of including CWRU/UHC in the hemostasis/transfusion medicine clinical research consortium. The strengths of our institution in this regard include 1) the large volume of adult and pediatric patients with congenital and acquired hematologic disorders who receive care here, 2) the CWRU/UHC Cancer Center, an NCI-designated comprehensive cancer center through which a well established clinical research infrastructure is available for administering and monitoring our research activities, and 3) the experience and track record of our faculty in accruing patients to NIH-sponsored clinical trials. Our second goal will be to provide a comprehensive rationale and experimental approach for studying two common thrombocytopenic disorders, heparin-associated thrombocytopenia (HIT) and immune thrombocytopenic purpura (ITP). With regard to HIT, we will assess the incidence of subclinical thrombosis in newly diagnosed patients with HIT and "asymptomatic thrombocytopenia", and evaluate the need for alternative anticoagulation in these patients. We hypothesize that such therapy may not be necessary in all cases. We will also directly compare the efficacy of refludan and argotraban in preventing the development of thrombi in this patient group. In our second project, we will assess the incidence of H. pylori infection in a large cohort of patients with ITP, and determine whether eradication of H. pylori induces a remission of ITP. We hypothesize that many patients with ITP will respond to this intervention. Successful completion of these studies will provide important insights into the management of each of these disorders that will have an important and immediate impact on their management.