Vangelista A, Frascà G M, Raimondi C, Liviano-D'Arcangelo G, Bonomini V
Haemostasis. 1986;16 Suppl 1:51-4. doi: 10.1159/000215342.
Thrombotic microangiopathy (TMA) can occur whenever pathogenetic events lead to fibrin deposition in the microcirculation. It has been suggested that intravascular coagulation is important in the development of renal as well as cerebral lesions. The mortality rate in adults varies from 50 to 70%; chronic or progressive renal failure occurs in approximately two thirds of children over 2 years of age. Poor success may be due to therapy being initiated too late or to inappropriate use of antagonistic drugs, or both. In the last 2 years we have treated 8 patients with TMA (5 with thrombotic thrombocytopenic purpura; 3 with hemolytic uremic syndrome) with defibrotide, a new antithrombotic agent extracted from mammalian lungs. At admission all patients had severe renal involvement (serum creatinine 5.3-14.9 mg/dl) and coagulation abnormalities (low platelet count; high levels of circulating fibrin degradation products). There were neurological manifestations in 6 patients. Defibrotide administration was followed in 6 patients by recovery of renal function. In all patients, defibrotide therapy induced the disappearance of neurological manifestations and normalization of coagulation abnormalities. Defibrotide caused no side effects. All patients are alive after 7-22 months of follow-up.
血栓性微血管病（TMA）在任何致病事件导致微循环中纤维蛋白沉积时均可发生。有人提出，血管内凝血在肾脏和脑部病变的发展中起重要作用。成人的死亡率在50%至70%之间；约三分之二的2岁以上儿童会出现慢性或进行性肾衰竭。治疗效果不佳可能是由于治疗开始得太晚或对抗性药物使用不当，或两者兼而有之。在过去两年中，我们用去纤苷治疗了8例TMA患者（5例血栓性血小板减少性紫癜；3例溶血尿毒综合征），去纤苷是一种从哺乳动物肺中提取的新型抗血栓药物。入院时，所有患者均有严重的肾脏受累（血清肌酐5.3 - 14.9 mg/dl）和凝血异常（血小板计数低；循环纤维蛋白降解产物水平高）。6例患者有神经学表现。6例患者在使用去纤苷后肾功能恢复。在所有患者中，去纤苷治疗使神经学表现消失，凝血异常恢复正常。去纤苷未引起副作用。所有患者在随访7 - 22个月后均存活。
