Novel Therapies in Hemostasis and Transfusion Medicine

止血和输血医学的新疗法

• 批准号: 7116779
• 负责人: JAMES BRUCE BUSSEL
• 金额: $ 29.3万
• 依托单位: WEILL MEDICAL COLL OF CORNELL UNIV
• 依托单位国家: 美国
• 财政年份: 2002
• 资助国家: 美国
• 起止时间: 2002-09-30 至 2007-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7116779
• 关键词: antineoplastics; blood disorder chemotherapy; blood transfusion; blood treatment; clinical research; clinical trials; cooperative study; hemostasis; human subject; human therapy evaluation; immunosuppression; leukocyte transfusion; monoclonal antibody; neutropenia; patient oriented research; thrombocytopenic purpura; thrombopoiesis; thrombopoietic factor; thrombosis

DESCRIPTION (provided by applicant): This application is in response to RFA HL-02-001 entitled Transfusion Medicine/Hemostasis Clinical Network and is a consortium of the New York Presbyterian Hospital - Weill Cornell College of Medicine-Columbia College of Physicians and Surgeons. It provides health care to > 20% of the New York City metropolitan area and has access to > 20 million people within 2 hours. It is an amalgam of physicians and scientists involved in clinical research in Hemostasis and Transfusion Medicine with expertise in Pediatrics, Internal Medicine, Pathology, and Surgery. The group has both the patient population and the clinical expertise required to participate in clinical trials proposed by other centers in the Network. Specific Aim 1 is a clinical trial of refractory ITP, defined as children and adults with ITP who have failed to respond to splenectomy. It intends to focus on the pathophysiology of refractory ITP by comparing two novel treatments. One, rituximab, is an anti-CD20 which depletes the recipient of B cells and should be an effective immunosuppressant in a "pure" autoantibody disease like ITP. There is preliminary data describing its effectiveness which should optimize its use. The other arm intends to use thrombopoietin or mimetic (TPO) to increase the platelet count by stimulating platelet production. This arm hypothesizes that a critical element in refractory ITP is a decreased production of platelets which can be rectified by stimulation with TPO. A registry of splenectomy will be included to facilitate enrollment of eligible patients. Specific Aim 2 intends to optimize granulocytes for transfusion. This would be of great potential benefit to patients with prolonged, severe neutropenia who suffer considerable morbidity and occasional mortality from infection. There is also the high cost of prolonged hospitalizations. In the past, studies of granulocyte transfusion showed little benefit and significant toxicity. The current study will explore different methods of preparation of granulocytes and also novel techniques for evaluation of their efficacy. Specific Aim 3 demonstrates that the consortium is able to participate in protocols for a wide variety of disorders of hemostasis and transfusion medicine. The consortium includes Dr. Grima of the NY Blood Center who annually phereses approximately 20 TTP patients; a leading center, Cornell, for management of patients with alloimmune thrombocytopenia;and a large hemophilia center.

描述（由申请人提供）： 本申请是对标题为“输血医学/止血临床网络”的RFA HL-02-001的回应，是纽约长老会医院-威尔康奈尔医学院-哥伦比亚内外科医学院的联合体。 它为超过20%的纽约市大都会地区提供医疗保健，并在2小时内为超过2000万人提供服务。 它是一个混合的医生和科学家参与临床研究的止血和输血医学与专业知识，在儿科，内科，病理学和外科。 该小组拥有参与网络中其他中心提出的临床试验所需的患者人群和临床专业知识。具体目标1是一项难治性ITP的临床试验，定义为脾切除术无效的儿童和成人ITP。 它旨在通过比较两种新的治疗方法来关注难治性ITP的病理生理学。 一种是利妥昔单抗，它是一种抗CD 20的药物，可以耗尽受体的B细胞，应该是一种有效的免疫抑制剂，用于治疗“纯”自身抗体疾病，如ITP。 有初步数据说明其有效性，应优化其使用。 另一组拟使用血小板生成素或模拟物（TPO）通过刺激血小板生成来增加血小板计数。该分支假设难治性ITP中的关键因素是血小板产生减少，这可以通过TPO刺激来纠正。 将纳入脾切除术的登记研究，以促进合格患者的入组。 具体目标2旨在优化用于输血的粒细胞。 这将是一个巨大的潜在利益，长期，严重的中性粒细胞减少症的患者遭受相当大的发病率和偶尔死亡的感染。 此外，长期住院的费用也很高。 在过去，粒细胞输注的研究表明，几乎没有好处和显着的毒性。 目前的研究将探索不同的方法制备的粒细胞，也是新的技术，以评估其疗效。 具体目标3表明，该联盟能够参与各种止血和输血医学疾病的方案。 该联盟包括纽约血液中心的Grima博士，他每年为大约20名TTP患者提供服务;一个领先的中心，康奈尔大学，用于管理同种免疫性血小板减少症患者;以及一个大型血友病中心。