Observational studyBone marrow transplantation2026
Time to resolution of veno-occlusive disease/sinusoidal obstruction syndrome post-HCT in defibrotide-treated adults from two European registries.
Observational study in Bone marrow transplantation, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Authors and funding
9 authors.
Funding
Abstract
We report a pooled analysis of real-world data of adult patients from two European registries of defibrotide use (DEFIFrance and EBMT Post-Authorization Safety Study [EBMT PASS]) to provide insights into the duration of defibrotide therapy to achieve resolution of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) symptoms by severity. Overall, 275 defibrotide-treated adults with VOD/SOS post-hematopoietic cell transplantation (HCT) were included (severe VOD/SOS, n = 215; nonsevere VOD/SOS, n = 60). Median time from the start of defibrotide treatment to VOD/SOS resolution was 18.5 and 16.5 days in patients with severe and nonsevere VOD/SOS, respectively. Among patients with VOD/SOS resolution, symptoms resolved after >21 days in 31% (n = 40/128) and 32% (n = 14/44) with severe and nonsevere VOD/SOS, respectively. In all patients with VOD/SOS resolution independent of severity (n = 174), the Kaplan-Meier-estimated survival at Day 100 post-HCT was 82% (95% confidence interval [CI]: 75%, 87%); this was 78% (95% CI: 70%, 84%) and 91% (95% CI: 78%, 96%) in patients with severe and nonsevere VOD/SOS, respectively. Serious treatment-emergent adverse events of interest occurred in 31% of patients with VOD/SOS resolution. This pooled analysis supports the utility of defibrotide treatment for VOD/SOS and highlights that treatment may often need to be continued beyond 21 days to achieve resolution.
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