Observational studyBone marrow transplantation2021
A multicentre, multinational, prospective, observational registry study of defibrotide in patients diagnosed with veno-occlusive disease/sinusoidal obstruction syndrome after haematopoietic cell transplantation: an EBMT study.
Observational study in Bone marrow transplantation, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03032016 (A Multi-centre, Multinational, Prospective Observational Registry to Collect Safety and Outcome Data in Patients Diagnosed With Severe Hepatic Veno-occlusive Disease), which is not on this map. Cited by 13 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
A Multi-centre, Multinational, Prospective Observational Registry to Collect Safety and Outcome Data in Patients Diagnosed With Severe Hepatic Veno-occlusive Disease (VOD) Following Hematopoietic Stem Cell Transplantation (HSCT) and Treated With Defitelio®
Who cites it
13 citing papers in PubMed, 18 citations in OpenAlex.
- Time to resolution of veno-occlusive disease/sinusoidal obstruction syndrome post-HCT in defibrotide-treated adults from two European registries.Bone marrow transplantation · 2026Observational
- Defibrotide for late-onset sinusoidal obstruction syndrome following umbilical cord blood transplantation: a single-center retrospective study.International journal of hematology · 2026Article
- Detecting Endothelial Compromise Objectively through Dynamic EASIX scoring (DECODE) - predictive utility of EASIX and modified EASIX scores for endothelial complications post hematopoietic cell transplant.Frontiers in oncology · 2026Article
- Defibrotide for Protecting Against and Managing Endothelial Injury in Hematologic Malignancies and COVID-19.Biomolecules · 2025Review
- Liver stiffness measurements predict Sinusoidal Obstructive Syndrome after hematopoietic stem cell transplantation.Bone marrow transplantation · 2024Article
- HokUS-10 scoring system predicts the treatment outcome for sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation.Scientific reports · 2023Article
- Hypofibrinolysis in pediatric patients with veno-occlusive disease in hematopoietic stem cell transplantation.Journal of cancer research and clinical oncology · 2023Article
- Cemiplimab-Associated Sinusoidal Obstruction Syndrome.ACG case reports journal · 2023Article
- Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.Bone marrow transplantation · 2023Article
- Kidney Injury in Children after Hematopoietic Stem Cell Transplant.Current oncology (Toronto, Ont.) · 2023Review
- Defibrotide-treated patients with anicteric or icteric veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic cell transplantation: an EBMT study.Bone marrow transplantation · 2022Article
- Defibrotide Prophylaxis of Sinusoidal Obstruction Syndrome in Adults Treated With Inotuzumab Ozogamicin Prior to Hematopoietic Stem Cell Transplantation.Frontiers in oncology · 2022Article
- Systematic screening and focused evaluation for veno-occlusive disease/sinusoidal obstructive syndrome (VOD/SOS) following allogeneic stem cell transplant is associated with earlier diagnosis and prompt institution of defibrotide treatment.Frontiers in transplantation · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
16 authors at 13 institutions in 4 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Severe hepatic veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a potentially life-threatening complication of haematopoietic cell transplantation (HCT). This multinational, prospective, observational study (NCT03032016), performed by the EBMT, enrolled patients treated with defibrotide from April 2015 to July 2018. This analysis focused on defibrotide-treated patients with VOD/SOS post-HCT. The primary endpoint was incidence of serious adverse events (SAEs) of interest up to 12 months post-HCT in patients with severe VOD/SOS. Overall, 104 defibrotide-treated patients with VOD/SOS post-HCT were enrolled: 62 had severe VOD/SOS and comprised the primary study population, including 36 with multi-organ dysfunction/failure (MOD/MOF). SAEs of interest occurred in 20 of 62 (32%) severe VOD/SOS patients; the most common by category were infection (24%) and bleeding (13%). In patients with severe VOD/SOS, the Kaplan-Meier-estimated Day 100 survival rate was 73% (95% CI: 60%, 82%) with VOD/SOS resolution by Day 100 in 45 of 62 (73%) patients. MOD/MOF resolved in 19 of 36 (53%) patients with MOD/MOF at VOD/SOS diagnosis. Results from this multicentre registry study build on prior defibrotide studies supporting the utility of defibrotide for the treatment of VOD/SOS post-HCT. These results provide additional real-world evidence of the effectiveness and safety of defibrotide in patients with VOD/SOS post-HCT.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.