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Synonyms: Defitelio®
defibrotide is an approved drug (EMA (2013), FDA (2016))
Compound class:
Natural product
Comment: Defibrotide is a deoxyribonucleic acid derivative (single-stranded) purified from bovine lung. It has anticoagulant activity. Defibrotide is administered as defibrotide sodium. As this is a natural extract, its composition is not fully resolved, so no chemical structure is provided.
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Summary of Clinical Use  |
| Approved to treat hepatic veno-occlusive disease (VOD) in adults and children following hematopoietic stem cell transplantation (HSCT)- reviewed in [2]. Defibrotide is the first and only approved treatment in Europe for severe VOD. Phase 2I clinical trial results are published in [6]. A Phase 2 trial has been designed to evaluate defibrotide's anticoagulant effect in preventing sinusoidal obstruction syndrome in high-risk sickle cell patients undergoing a familial haploidentical allogeneic stem cell transplantation (NCT02675959) [1]. |
| Mechanism Of Action and Pharmacodynamic Effects |
| Hepatic VOD can occur in patients who receive chemotherapy and HSCT, and it can lead to multiple organ failure and death. Although hepatic VOD is rare (~2% of patients develop the condition after HSCT), around 80% of these patient do not survive. Defibrotide, therefore fills a significant medical need in post-HSCT management. Defibrotide's mechanism of action includes increasing PGI2 generation [3], with PGI2 reducing platelet aggregation. Defibrotide is also reported to have profibrinolytic activity in animal studies [4]. Hepatic VOD can also be known as sinusoidal obstruction syndrome (SOS). |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT02675959 | Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease | Phase 2 Interventional | New York Medical College | ||
