Observational studyBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2016
Defibrotide for Treatment of Severe Veno-Occlusive Disease in Pediatrics and Adults: An Exploratory Analysis Using Data from the Center for International Blood and Marrow Transplant Research.
Observational study in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
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Who cites it
29 citing papers in PubMed, 60 citations in OpenAlex.
- Trial
- Defibrotide Therapy for SARS-CoV-2 ARDS.Chest · 2022Trial
- Risk Score for the Development of Veno-Occlusive Disease after Allogeneic Hematopoietic Cell Transplant.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2018Trial
- Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome.British journal of haematology · 2018Trial
- Drug-induced sinusoidal obstruction syndrome: a real-world pharmacovigilance study based on the FDA Adverse Event Reporting System (FEARS).BMC pharmacology & toxicology · 2025Article
- Defibrotide for Protecting Against and Managing Endothelial Injury in Hematologic Malignancies and COVID-19.Biomolecules · 2025Review
- Article
- Incidence and risk factors of early onset VOD/SOS differ in younger vs older adults after stem cell transplantation.Blood advances · 2024Article
- Defibrotide impact on the acute GVHD disease incidence in pediatric hematopoietic stem cell transplant recipients.Life science alliance · 2023Article
- Real-world use of defibrotide for veno-occlusive disease/sinusoidal obstruction syndrome: the DEFIFrance Registry Study.Bone marrow transplantation · 2023Article
- Molecular Advances in Sinusoidal Obstruction Syndrome/Veno-Occlusive Disease.International journal of molecular sciences · 2023Review
- Prognostic Biomarkers for Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome in Myeloablative Allogeneic Hematopoietic Cell Transplantation: Results from the Blood and Marrow Transplant Clinical Trials Network 1202 Study.Transplantation and cellular therapy · 2023Article
- Inotuzumab ozogamicin in B-cell precursor acute lymphoblastic leukemia: efficacy, toxicity, and practical considerations.Frontiers in immunology · 2023Review
- [Research progress of hepatic sinusoidal obstruction syndrome].Zhonghua gan zang bing za zhi = Zhonghua ganzangbing zazhi = Chinese journal of hepatology · 2022Article
- Influence of creatinine levels on survival in patients with veno-occlusive disease treated with defibrotide.The Korean journal of internal medicine · 2022Article
- [Advances in the diagnosis and management of sinusoidal obstruction syndrome after allogeneic hematopoietic stem cell transplantation].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2021Article
- Introduction of new pediatric EBMT criteria for VOD diagnosis: is it time-saving or money-wasting? : Prospective evaluation of pediatric EBMT criteria for VOD.Bone marrow transplantation · 2020Article
- A case report of severe veno-occlusive disease following autologous stem cell transplantation successfully treated with Defibrotide.European journal of translational myology · 2020Article
- Modified diagnostic criteria, grading classification and newly elucidated pathophysiology of hepatic SOS/VOD after haematopoietic cell transplantation.British journal of haematology · 2020Review
- Publisher Correction: Systematic review of defibrotide studies in the treatment of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).Bone marrow transplantation · 2019Article
Corrections and comments
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Authors and funding
8 authors at 3 institutions in 1 country.
Funding
Abstract
Veno-occlusive disease (VOD) is an early and serious complication of hematopoietic cell transplantation (HCT) that is associated with inferior survival, particularly when it is complicated by multiorgan failure (severe VOD). We evaluated the efficacy of defibrotide in the treatment of severe VOD using observational data from the Center for International Blood and Marrow Transplant Research (CIBMTR). Eight thousand three hundred forty-one patients treated by HCT between 2008 and 2011 were identified from the CIBMTR clinical database; 3.2% met criteria for VOD and 1.2% met criteria for severe VOD. Patients with a diagnosis of VOD as reported to the CIBMTR by their transplanting centers, who had no prior history of cirrhosis, and who had a maximum total bilirubin level > 2.0 mg/dL by day +100 post-HCT were selected for study. Severe VOD was defined as VOD occurring in the setting of renal impairment requiring dialysis or any noninfectious pulmonary abnormality. Patients with severe VOD were divided into 2 groups for analysis: those treated with defibrotide (n = 41) and those not treated with defibrotide (n = 55). Patients in the nondefibrotide group were older, were more likely to be male, were more likely to have a history of previous fungal infection, and had a higher proportion of clinically significant pre-existing disease or organ impairment. Survival rate at day +100 was 39% (95% CI, 24.8% to 54.3%) in patients receiving defibrotide and 30.9% (95% CI, 19.5% to 43.6%) in those not receiving defibrotide. Resolution rate of VOD at day +100 was 51% in the defibrotide group and 29% in the nondefibrotide group (difference, 22.1%; 95% CI, 2.6% to 42%). The results of our study are consistent with previously reported experiences with defibrotide, confirm the poor outcome of this syndrome, and suggest defibrotide is effective in the treatment of severe VOD.
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