ArticleBlood advances2022
Defibrotide: real-world management of veno-occlusive disease/sinusoidal obstructive syndrome after stem cell transplant.
Article in Blood advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.
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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.
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Who cites it
18 citing papers in PubMed, 35 citations in OpenAlex.
- Prospective assessment of risk biomarkers of sinusoidal obstruction syndrome after hematopoietic cell transplantation.JCI insight · 2023Trial
- Chronic arsenic poisoning from a realgar-containing traditional medicine causing hepatic veno-occlusive disease and polyserositis: a case report.Journal of medical case reports · 2026Article
- Defibrotide for late-onset sinusoidal obstruction syndrome following umbilical cord blood transplantation: a single-center retrospective study.International journal of hematology · 2026Article
- Review
- Defibrotide for Protecting Against and Managing Endothelial Injury in Hematologic Malignancies and COVID-19.Biomolecules · 2025Review
- Management of liver sinusoidal obstruction syndrome/veno-occlusive disease in adults: a 2025 perspective from an international expert group.Bone marrow transplantation · 2025Review
- The RNA revolution in medicine: from gene regulation to clinical therapeutics.Animal cells and systems · 2025Review
- An international survey to better understand the current incidence, severity, and management of VOD/SOS.Bone marrow transplantation · 2025Article
- Operational procedure sharing pathway in veno-occlusive disease: a Delphi consensus-based recommendations.Frontiers in oncology · 2025Article
- Article
- RNA therapeutics in targeting G protein-coupled receptors: Recent advances and challenges.Molecular therapy. Nucleic acids · 2024Review
- Incidence and risk factors of early onset VOD/SOS differ in younger vs older adults after stem cell transplantation.Blood advances · 2024Article
- Clinical outcomes of early defibrotide discontinuation: a single-center case series.Bone marrow transplantation · 2024Article
- Stem cell transplantation for ALL: you've always got a donor, why not always use it?Hematology. American Society of Hematology. Education Program · 2023Review
- Current incidence, severity, and management of veno-occlusive disease/sinusoidal obstruction syndrome in adult allogeneic HSCT recipients: an EBMT Transplant Complications Working Party study.Bone marrow transplantation · 2023Article
- The Importance of New EBMT Criteria on the Diagnosis of Veno-Occlusive Liver Disease in Children.Journal of clinical medicine · 2023Article
- Article
- A Comparative Study of Porcine and Ovine Derived Defibrotide: Coagulation Profiles, Molecular Characteristics, and DNA Content.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisArticle
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Authors and funding
11 authors at 3 institutions in 1 country.
Funding
Abstract
Hepatic veno-occlusive disease or sinusoidal obstructive syndrome (VOD/SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT). Defibrotide is the only medication approved by the US Food and Drug Administration for the management of severe VOD/SOS after HSCT. We report our center's experience with commercially available defibrotide as treatment of patients with VOD/SOS. We retrospectively identified 28 cases of VOD/SOS, based on the European Society for Blood and Marrow Transplantation criteria, from March 2016 through June 2019. The median day of VOD/SOS onset was 25 days (range, 8-69 days), and defibrotide was initiated on day of diagnosis in 71% of patients. Complete resolution of VOD/SOS occurred in 75% of patients. Day 100 survival was 64% for all HSCT patients and 53% for those with very severe VOD/SOS. Response rates and survival were similar in patients with VOD/SOS after myeloablative or reduced-intensity chemotherapy HSCT. Therapy-related adverse events were mild and included hematuria (43%), epistaxis (18%), and hypotension (11%). Severe hemorrhagic adverse events occurred in 2 patients (pulmonary hemorrhage and upper gastrointestinal hemorrhage; 7%) and both in the setting of progressive VOD/SOS. Early diagnosis, prompt initiation of defibrotide, and minimization of dosing interruptions may be key to successful treatment of VOD/SOS.
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