Defibrotide treatment was associated with improved overall survival in sinusoidal obstruction syndrome/veno-occlusive disease after allogeneic hematopoietic stem cell transplantation, with a hazard ratio of 0.41 compared to recombinant thrombomodulin.
Defibrotide treatment was associated with improved overall survival compared to recombinant thrombomodulin in multivariate analysis.
The incidence of SOS/VOD among allo-HSCT recipients in this cohort was approximately 6.4%.
At 100 days post-onset, overall survival was numerically higher in the defibrotide group than the rTM group, though the difference was not statistically significant.
Baseline characteristics were broadly similar between the two treatment groups, with no significant differences in disease stage at transplantation or SOS/VOD severity at diagnosis.
Review of the most severe SOS/VOD cases suggested an association between inflammatory status and the clinical course of the disease.
This research studied a serious complication called sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD), which is a condition where small blood vessels in the liver become blocked following bone marrow transplantation. Researchers in Japan compared outcomes in 48 patients who developed this condition over roughly 13 years, dividing them into two groups based on which treatment they received: an older drug called recombinant thrombomodulin (15 patients treated before 2019) versus a newer drug called defibrotide (33 patients treated from 2019 onward). When accounting for differences in patient age and the intensity of their pre-transplant chemotherapy, patients who received defibrotide were about 59% less likely to die during follow-up than those who received recombinant thrombomodulin. While the raw survival numbers at 100 days after diagnosis were better in the defibrotide group (about 47% alive vs. 20% alive), that difference alone was not statistically significant, likely because of the small number of patients involved. However, once statistical adjustments were made for age and treatment intensity, defibrotide showed a meaningful survival benefit. The researchers also observed that inflammation in the body appeared to be connected to how severe SOS/VOD became and how well patients recovered. This research suggests that defibrotide may be a more effective treatment for SOS/VOD than recombinant thrombomodulin, supporting its use in clinical practice. The finding that inflammation may play a role in disease course could point toward future directions for treatment. However, because this was a retrospective study with a relatively small number of patients treated at a single center over different time periods, larger studies would be needed to confirm these results.
Katsuki K, Tachibana T, Izumi A, Nukui J, Arai S, Hirose N, et al.. (2026). Impact of defibrotide on survival in sinusoidal obstruction syndrome / veno-occlusive disease: a retrospective cohort study comparing two sequential treatment eras.. Hematology (Amsterdam, Netherlands). https://doi.org/10.1080/16078454.2026.2721241