A large-scale retrospective study found that treatment with defibrotide can be an effective treatment for post-HCT veno-occlusive disease (VOD) and severe VOD (VOD with multi-organ failure). Researchers analyzed 8,341 transplant outcomes reported to CIBMTR and found that 3.2% met criteria for VOD and 1.2% met criteria for severe VOD. Survival rate at day 100 in patients receiving defibrotide (n=41) and in patients not receiving defibrotide (n=55) was 39% vs. 30.9%, respectively, and 100-day resolution rate of VOD was 51% in the defibrotide group and 29% in the non-defibrotide group. Based on poor outcomes associated with VOD, the authors conclude that their results are consistent with previously reported experiences with defibrotide and suggest defibrotide is effective in the treatment of severe VOD.
Strouse C, et al. Biol Blood Marrow Transplant