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

Abstract

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.