Trial reportBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2010
Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial.
Trial report in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to 2 registered trials, which are not on this map. Cited by 87 papers, 2 of them syntheses that pooled it.
What it found
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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.
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.
Using Ultrasound Elastography to Predict Development of Sinusoidal Obstruction Syndrome
Using Ultrasound Elastography to Predict Development of Hepatic Sinusoidal Obstruction Syndrome
Who cites it
87 citing papers in PubMed, 2 syntheses or guidelines pooled it, 231 citations in OpenAlex.
- CROATIAN GUIDELINES FOR THE DIAGNOSIS AND TREATMENT OF PORTAL HYPERTENSION.Acta clinica Croatica · 2024Guideline
- Veno-occlusive disease/sinusoidal obstruction syndrome after haematopoietic stem cell transplantation: Middle East/North Africa regional consensus on prevention, diagnosis and management.Bone marrow transplantation · 2017Guideline
- Trial
- Analysis of Time to Complete Response after Defibrotide Initiation in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome after Hematopoietic Cell Transplantation.Transplantation and cellular therapy · 2021Trial
- Defibrotide enhances fibrinolysis in human endotoxemia - a randomized, double blind, crossover trial in healthy volunteers.Scientific reports · 2019Trial
- Clinical effects of recombinant thrombomodulin and defibrotide on sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation.Bone marrow transplantation · 2019Trial
- Acute Graft-vs.-Host Disease-Associated Endothelial ActivationFrontiers in immunology · 2019Trial
- Defibrotide for the treatment of hepatic veno-occlusive disease/sinusoidal obstruction syndrome following nontransplant-associated chemotherapy: Final results from a post hoc analysis of data from an expanded-access program.Pediatric blood & cancer · 2018Trial
- Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome.British journal of haematology · 2018Trial
- Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure.Blood · 2016Trial
- Sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation: Incidence, risk factors and outcomes.Bone marrow transplantation · 2016Trial
- Escalation to High-Dose Defibrotide in Patients with Hepatic Veno-Occlusive Disease.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2015Trial
- Defibrotide for late-onset sinusoidal obstruction syndrome following umbilical cord blood transplantation: a single-center retrospective study.International journal of hematology · 2026Article
- Assessment of Risks and Outcomes of Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease in Allogeneic Stem Cell Transplant Recipients Including Potentially Undiagnosed Cases-A Multicenter Canadian Study.Current oncology (Toronto, Ont.) · 2026Article
- Evaluating the safety profile of defibrotide in sickle cell disease: anHaematologica · 2025Article
- The Liver in Hematologic Malignancies: Lymphoma, Leukemia, and Myeloma.Clinics in liver disease · 2025Review
- Defibrotide for Protecting Against and Managing Endothelial Injury in Hematologic Malignancies and COVID-19.Biomolecules · 2025Review
- Non-infectious pulmonary complications after haematopoietic progenitor transplantation: a diagnostic approach.Journal of thoracic disease · 2024Review
- Prevention and management of acute toxicities from conditioning regimens during hematopoietic stem cell transplantation.Clinical hematology international · 2024Review
- Defibrotide impact on the acute GVHD disease incidence in pediatric hematopoietic stem cell transplant recipients.Life science alliance · 2023Article
27 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
25 authors at 14 institutions in 2 countries.
Funding
Abstract
Therapeutic options for severe hepatic veno-occlusive disease (VOD) are limited and outcomes are dismal, but early phase I/II studies have suggested promising activity and acceptable toxicity using the novel polydisperse oligonucleotide defibrotide. This randomized phase II dose-finding trial determined the efficacy of defibrotide in patients with severe VOD following hematopoietic stem cell transplantation (HSCT) and identified an appropriate dose for future trials. Adult and pediatric patients received either lower-dose (arm A: 25 mg/kg/day; n = 75) or higher-dose (arm B: 40 mg/kg/day; n = 74) i.v. defibrotide administered in divided doses every 6 hours for > or =14 days or until complete response, VOD progression, or any unacceptable toxicity occurred. Overall complete response and day +100 post-HSCT survival rates were 46% and 42%, respectively, with no significant difference between treatment arms. The incidence of treatment-related adverse events was low (8% overall; 7% in arm A, 10% in arm B); there was no significant difference in the overall rate of adverse events between treatment arms. Early stabilization or decreased bilirubin was associated with better response and day +100 survival, and decreased plasminogen activator inhibitor type 1 (PAI-1) during treatment was associated with better outcome; changes were similar in both treatment arms. Defibrotide 25 or 40 mg/kg/day also appears effective in treating severe VOD following HSCT. In the absence of any differences in activity, toxicity or changes in PAI-1 level, defibrotide 25 mg/kg/day was selected for ongoing phase III trials in VOD.
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Identifiers
What OpenQuestion holds
Registered trials
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.