ASPIRIN PROPHYLAXIS IN PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

阿司匹林预防阵发性睡眠性血红蛋白尿

• 批准号: 5201207
• 负责人: S ROSENFELD
• 金额: --
• 依托单位: CLINICAL CENTER
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/5201207
• 关键词: aspirin; blood cell count; cardiovascular disorder chemotherapy; chemoprevention; cytolysis; drug administration rate /duration; flow cytometry; human subject; membrane proteins; paroxysmal nocturnal hemoglobinuria; platelet activation; platelets; venous thrombosis

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic hemolytic disorder characterized by the loss of membrane proteins that protect erythrocytes from complement mediated lysis. It is associated with a high risk of major thrombotic events, most commonly thrombosis of large intraabdominal veins. Most patients who die of their disease die of thrombosis. While the cause of this thrombophilia is unknown, we have recently demonstrated normal levels of platelet activation in patients with PNH. We have written a protocol that is currently under consideration by the NHLBI IRB (minor changes have been requested) for treatment of patients with PNH with aspirin. Patients without a history of thrombosis or bleeding, and with platelet counts over 50,000/mm3, will be screened for platelet activation on at least two occasions by flow cytometry. Patients with evidence of activation will be placed on low-dose aspirin for two weeks, and their degree of platelet activation followed. They will then be reassessed after a period off aspirin.

阵发性睡眠性血红蛋白尿症是一种慢性溶血性疾病 以保护红细胞的膜蛋白的丧失为特征 来自补体介导的溶解。它与高风险的重大 血栓形成事件，最常见的是腹腔内大静脉血栓形成。 大多数死于疾病的患者死于血栓形成。虽然原因 这种血栓形成倾向是未知的，我们最近已经证明正常的 PNH患者的血小板活化水平。我们写了一个 NHLBI IRB目前正在考虑的方案（次要 已要求变更）用于治疗PNH患者， 阿司匹林无血栓形成或出血史，且 血小板计数超过50，000/mm 3，将进行血小板活化筛查 在至少两种情况下通过流式细胞术。证据的患者 激活将放置在低剂量阿司匹林两周， 血小板活化程度。然后，他们将被重新评估后， 停一段时间的阿司匹林