Palmer K J, Goa K L
Adis International Limited, Auckland, New Zealand.
Drugs. 1993 Feb;45(2):259-94. doi: 10.2165/00003495-199345020-00007.
Defibrotide is a deoxyribonucleic acid derivative extracted from mammalian organs, which has been developed for the treatment of a number of vascular disorders. It appears to increase fibrinolysis and may possess antithrombotic, antiatherosclerotic and anti-ischaemic actions, probably due to its ability to selectively increase prostaglandin I2 and E2 levels and to increase tissue plasminogen activator and decrease plasminogen activator inhibitor function. Defibrotide is available as an intravenous and intramuscular preparation, and also as an oral formulation for long term use. Trials performed to date have provided initial evidence that defibrotide is effective in the treatment of peripheral obliterative arterial disease and acute thrombophlebitis, while preliminary data suggest possible use in preventing fibrin deposition in the circuitry of renal haemodialysis equipment. Efficacy in preventing deep vein thrombosis after surgery has been demonstrated but defibrotide does not appear to offer any therapeutic advantage over heparin. Further clinical experience is required in other disorders, including acute myocardial infarction, Raynaud's phenomenon, renal thrombotic microangiopathy and renal transplant rejection, before adequate assessment of efficacy in these areas can be made. Defibrotide is well tolerated, as assessed in trials of up to 6 months duration, with a low global incidence of adverse events (< 1 to 9%). Mild allergic reactions and gastrointestinal disturbances have occasionally been described, and a hypotensive effect has also infrequently been observed. Thus, available data suggest that defibrotide is a well tolerated agent with little or no anticoagulant activity, which is conveniently available in both parenteral and oral formulations. Initial data indicate that the drug may be a useful alternative in the treatment of peripheral obliterative arterial disease and thrombophlebitis, while its therapeutic potential in other vascular disorders and efficacy relative to established agents remains to be fully determined.
去纤苷是一种从哺乳动物器官中提取的脱氧核糖核酸衍生物，已被开发用于治疗多种血管疾病。它似乎能增强纤维蛋白溶解作用，可能还具有抗血栓形成、抗动脉粥样硬化和抗缺血作用，这可能是因为它能够特异性地提高前列腺素I2和E2水平，并增强组织纤溶酶原激活物活性、降低纤溶酶原激活物抑制剂的功能。去纤苷有静脉注射和肌肉注射制剂，也有可供长期使用的口服制剂。迄今为止进行的试验已提供初步证据表明，去纤苷在治疗外周闭塞性动脉疾病和急性血栓性静脉炎方面有效，而初步数据表明其可能用于预防肾透析设备回路中的纤维蛋白沉积。已证实去纤苷在预防术后深静脉血栓形成方面有效，但与肝素相比，去纤苷似乎没有任何治疗优势。在对包括急性心肌梗死、雷诺现象、肾血栓性微血管病和肾移植排斥反应在内的其他疾病进行充分疗效评估之前，还需要更多的临床经验。在长达6个月的试验评估中，去纤苷耐受性良好，不良事件的总体发生率较低（<1%至9%）。偶尔会出现轻微的过敏反应和胃肠道不适，也很少观察到低血压效应。因此，现有数据表明，去纤苷是一种耐受性良好的药物，几乎没有或没有抗凝活性，有方便的肠胃外和口服制剂。初步数据表明，该药物可能是治疗外周闭塞性动脉疾病和血栓性静脉炎的有用替代品，但其在其他血管疾病中的治疗潜力以及相对于现有药物的疗效仍有待充分确定。
