去纤苷在造血干细胞移植诱发的肝窦阻塞综合征中的应用

Application of defibrotide in hepatic sinusoidal obstruction syndrome induced by hematopoietic stem cell transplantation

肝窦阻塞综合征(HSOS)是造血干细胞移植(HSCT)患者早期最常见的并发症,据报道HSCT后HSOS的平均发病率为13.7%(0~62%),HSOS可能伴有多器官功能衰竭,发病100 d时病死率可超过80%。去纤苷是目前用于预防和治疗HSCT后HSOS最安全有效的药物,是目前美国唯一批准治疗HSCT后伴有肺/肾功能障碍的HSOS的药物,在欧盟国家被批准用于治疗HSCT后的严重HSOS。现概括性描述去纤苷在HSCT后HSOS中的临床应用,简述去纤苷的药理作用、药效和安全性、用药剂量、治疗等方面的研究进展。

Hepatic sinusoidal obstruction syndrome(HSOS)is the most common early complication in patients with hematopoietic stem cell transplantation(HSCT).According to reports,the average incidence of HSOS post-HSCT is 13.7%(0~62%).HSOS may be accompanied by multiple organ failure,and the mortality rate can exceed 80%at 100 days of onset.Defibrotide is currently the safest and most effective drug used to prevent and treat HSOS post-HSCT.It is currently the only drug approved in the United States for the treatment of HSOS with lung/renal dysfunction post-HSCT.In addition,it has been approved in the European Union for the treatment of severe HSOS cases post-HSCT.This article briefly describes and summarizes the research progress,clinical application,pharmacokinetics,efficacy,safety,dose and treatment of defibrotide in HSOS post-HSCT.