Authors
Mohamad Mohty, Régis Peffault de Latour, Myriam Labopin, Vian Amber, Amélie Petitprez, Deborah Gutierrez, Nalina Dronamraju, Hélène Labussière-Wallet, Didier Blaise
Published in
Bone marrow transplantation. Jun 20, 2026. Epub Jun 20, 2026.
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.
PMID:
42321404
Bibliographic data and abstract were imported from PubMed on 20 Jun 2026.
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