去纤苷用于治疗伴有多器官功能衰竭的肝静脉闭塞病/窦性阻塞综合征。
Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome with multiorgan failure.
作者信息
Richardson Paul G, Grupp Stephan A, Pagliuca Antonio, Krishnan Amrita, Ho Vincent T, Corbacioglu Selim
机构信息
Jerome Lipper Multiple Myeloma Center, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA, USA.
Pediatric Oncology, The Children's Hospital of Philadelphia & the Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
出版信息
Int J Hematol Oncol. 2017 Nov;6(3):75-93. doi: 10.2217/ijh-2017-0015. Epub 2017 Aug 11.
Abstract
Hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome (VOD/SOS), is a potentially life-threatening and unpredictable complication of hematopoietic stem cell transplantation (HSCT). Characterized by a prothrombotic-hypofibrinolytic state, VOD/SOS typically presents with hyperbilirubinemia, ascites, weight gain and painful hepatomegaly; VOD/SOS with multiorgan failure may be associated with >80% mortality. Treatment has been mainly supportive. However, defibrotide is now approved in the USA for treatment of hepatic VOD/SOS with renal or pulmonary dysfunction following HSCT and in the European Union for treatment of severe hepatic VOD/SOS post-HSCT. evidence suggests defibrotide may restore thrombotic-fibrinolytic balance at the endothelial level and protect endothelial cells. Defibrotide has demonstrated significant reduction in VOD/SOS-related mortality and resolved VOD/SOS-related symptoms, with a manageable safety profile.
摘要
肝静脉闭塞病，也称为窦性阻塞综合征（VOD/SOS），是造血干细胞移植（HSCT）一种潜在的危及生命且不可预测的并发症。VOD/SOS的特征是血栓形成-纤溶活性低下状态，通常表现为高胆红素血症、腹水、体重增加和肝肿大疼痛；伴有多器官功能衰竭的VOD/SOS死亡率可能超过80%。治疗主要是支持性的。然而，去纤苷目前在美国被批准用于治疗HSCT后伴有肾或肺功能障碍的肝VOD/SOS，在欧盟被批准用于治疗HSCT后的严重肝VOD/SOS。有证据表明，去纤苷可能在内皮水平恢复血栓形成-纤溶平衡并保护内皮细胞。去纤苷已显示出VOD/SOS相关死亡率显著降低，解决了VOD/SOS相关症状，且安全性可控。
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