Triplett BM (2015), Biology of Blood and Marrow Transplantation (Link to abstract)
Defibrotide is considered to be a safe and effective treatment for hepatic VOD at doses of 25 mg/kg/day. In this prospective clinical trial, 34 patients received escalating doses of up to 110 mg/kg/day of defibrotide. There was no increased toxicity and no increased bleeding episodes for doses up to 100 mg/kg/day. Increased toxicity and bleeding were observed in doses of 110 mg/kg/day (n=5 patients treated with this dose). However, the outcomes of treatment with escalated doses were similar to results from previous cohorts of patients who were treated with standard defibrotide doses.
* This study suggests that escalation to higher doses of defibrotide is safe, but not more effective than standard dosing.