Veno-Occlusive Disease Treatment with Defibrotide

Defibrotide for Treatment of Veno-Occlusive Disease of the Liver

Protocol Description

The purpose of this study is to administer the drug Defibrotide in patients with veno-occlusive disease (VOD), a complication that can result from chemotherapy, radiation therapy and other agents used in marrow transplants. VOD is a failure of blood flow through the liver that can create a life-threatening condition requiring immediate treatment. VOD can result in jaundice, liver dysfunction, weight gain and swelling of the abdomen. Defibrotide is an experimental drug that is not approved by the Food and Drug Administration, however it is believed to prevent the formation of blood clots when given by vein every 6 hours, usually for 14 days.

Eligibility Criteria

Children and young adults who have been diagnosed with veno-occlusive disease (VOD) will be asked to be participants in this study.
Boys: All ages
Girls: All ages


Participants will undergo a physical examination including blood work and an abdominal ultrasound. Defibrotide will be administered intravenously every 6 hours, usually for 14 days. It is possible that additional courses of this drug may be administered.
Visits: Requires hospitalization
Duration: 2 weeks

Status: Open for Enrollment

Source(s) of Support


Primary Investigator

Rakesh Goyal, MD

Contact Information

To get started, please contact:
Rakesh Goyal, MD