Loading
Defibrotide (Defitelio) is the only approved treatment for hepatic veno-occlusive disease, a life-threatening liver complication of high-dose chemotherapy before stem cell transplant, in patients whose kidneys or lungs are also failing.
Defibrotide was approved by the FDA in March 2016 for adults and children with hepatic veno-occlusive disease (sinusoidal obstruction syndrome) with renal or pulmonary dysfunction after haematopoietic stem cell transplantation, on a phase 3 trial against historical controls in which survival at day 100 was 38% versus 25%, supported by an expanded-access programme of several hundred patients; the EU authorised Defitelio in 2013 for severe VOD after HSCT in patients over one month old. A subsequent phase 3 prophylaxis trial in high-risk patients (HARMONY) did not meet its primary endpoint, so use remains therapeutic. Bleeding is the main risk and it is contraindicated with systemic anticoagulants or thrombolytics. Marketed by Jazz Pharmaceuticals.
Mixture of single-stranded oligonucleotides from porcine intestinal mucosa that enhances plasmin-mediated fibrinolysis, reduces endothelial activation and protects sinusoidal endothelial cells; the mechanism is not fully elucidated.
1.Defibrotide slips into a pocket on its target.
| Region | Year | Indication |
|---|---|---|
| US | 2016 | Hepatic veno-occlusive disease with renal or pulmonary dysfunction after HSCT, adults and children |
| EU | 2013 | Severe hepatic veno-occlusive disease after HSCT in patients over one month (Defitelio) |
Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.
Query for this drug: (TITLE:"Defibrotide" OR ABSTRACT:"Defibrotide" OR TITLE:"Defitelio" OR ABSTRACT:"Defitelio") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Defibrotide, not a curated reading list.
Shares Allogeneic stem cell transplantation and the tag supportive.
Shares Allogeneic stem cell transplantation and the tag supportive.
Shares Autologous stem cell transplant (high-dose therapy) and the tag supportive.
Shares Autologous stem cell transplant (high-dose therapy) and the tag supportive.
Shares Autologous stem cell transplant (high-dose therapy) and the tag supportive.
Shares Allogeneic stem cell transplantation and the tag supportive.
Shares Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure, Study 2005-01, Autologous stem cell transplant (high-dose therapy).
Shares Autologous stem cell transplant (high-dose therapy), Allogeneic stem cell transplantation.