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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.


Z L Liu, Y Wang, Y L Liu, J Zhang. Application of defibrotide in hepatic sinusoidal obstruction syndrome induced by hematopoietic stem cell transplantation]. Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology. 2021 Jan 20;29(1):92-96

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PMID: 33541030

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