Defibrotide
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.
Overview
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.
Approvals
| 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) |
Trials
topRecruiting now (live from ClinicalTrials.gov)
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Latest papers
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