Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure

Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure
Richardson PG, (2016) Blood, (Link to Abstract)

 

Veno-occlusive disease is a well-known complication of HSCT with a high mortality in patients developing multi organ failure of >80%. The authors assessed 102 patients with a diagnosis of veno-occlusive disease (VOD) and multi-organ failure. These patients were given defibrotide 25mg/kg/d and were compared to 32 historical controls. Survival at +100 days was 38.2% in the defibrotide group vs. 25% in controls (p=0.01). Day +100 complete response was seen in 25.5% in the treatment group vs. 12.5% in controls. Adverse events were encountered in a similar incidence in the two groups. Day +180 survival was not statistically significant but the authors point out that the causes of death were not directly related to VOD.


* Defibrotide 25mg/kg/d in patients with multi-organ failure was safe and survival was improved at +100 days but not at +180 days. In conclusion, defibrotide can be given in multi-organ failure due to VOD but long-term survival benefits were not shown.