Liu Z L, Wang Y, Liu Y L, Zhang J
Beijing Youan Hospital, Capital Medical University, Beijing 100069, China.
Beijing Tongren Hospital, Capital Medical University, Beijing 100730, China.
Zhonghua Gan Zang Bing Za Zhi. 2021 Jan 20;29(1):92-96. doi: 10.3760/cma.j.cn501113-20190404-00106.
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.
肝窦阻塞综合征(HSOS)是造血干细胞移植(HSCT)患者最常见的早期并发症。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天时死亡率可超过80%。去纤苷是目前用于预防和治疗HSCT后HSOS最安全、最有效的药物。它是目前美国唯一批准用于治疗HSCT后伴有肺/肾功能不全的HSOS的药物。此外,它已在欧盟获批用于治疗HSCT后严重HSOS病例。本文简要描述和总结了去纤苷在HSCT后HSOS中的研究进展、临床应用、药代动力学、疗效、安全性、剂量及治疗情况。