Skill Checkup: A Man Who Had Autologous Hematopoietic Stem Cell Transplantation Developed Acute Graft-vs-Host Disease

James L. Harper, MD


February 10, 2022

Historically, low-dose tissue plasminogen activator was used to increase fibrin degradation in veno-occlusive disease. However, it has achieved responses in less than one third of patients. Furthermore, tissue plasminogen activator treatment may result in fatal hemorrhage, and its use is not recommended particularly in the presence of multiorgan failure, which is seen in severe veno-occlusive disease.

In the United States, defibrotide is the only agent with proven efficacy for the treatment of severe or very severe veno-occlusive disease. Its mechanism involves two distinct elements: protection of endothelial cells and restoration of the thrombotic-fibrinolytic balance. US Food and Drug Administration approval was based on findings of a phase 3 trial (N = 102) in which survival and complete response rates were significantly higher in patients receiving defibrotide compared with 32 historical controls. Survival at day 100 after hematopoietic stem cell transplantation was 38.2% in the defibrotide group and 25% in the control group.

Other approaches that have been used include antithrombin III replacement and high-dose methylprednisolone.


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