Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the Liver
| Status: | No longer available |
|---|---|
| Conditions: | Gastrointestinal |
| Therapuetic Areas: | Gastroenterology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 4/26/2017 |
| Start Date: | November 2003 |
Compassionate Use of Defibrotide for Patients With Veno-Occlusive Disease
Veno-occlusive disease (VOD) of the liver is a significant complication for some patients
undergoing hematopoietic stem cell transplantation. This disease is thought to be a toxicity
secondary to chemotherapy or radiation-induced damage to the epithelial cells of the blood
vessels in the liver. VOD is categorized as mild, moderate or severe. Historically, there
has been no method to treat the disease. Recently, however, there have been investigations
into the use of a new agent called defibrotide.
The primary purpose of this protocol is to provide defibrotide to patients with severe VOD.
Because this drug has not been approved by the FDA, use of this medication under the
auspices of this IND treatment plan is for compassionate use only.
undergoing hematopoietic stem cell transplantation. This disease is thought to be a toxicity
secondary to chemotherapy or radiation-induced damage to the epithelial cells of the blood
vessels in the liver. VOD is categorized as mild, moderate or severe. Historically, there
has been no method to treat the disease. Recently, however, there have been investigations
into the use of a new agent called defibrotide.
The primary purpose of this protocol is to provide defibrotide to patients with severe VOD.
Because this drug has not been approved by the FDA, use of this medication under the
auspices of this IND treatment plan is for compassionate use only.
Secondary objectives of this protocol include the following:
- To describe the toxicities of defibrotide in patients with VOD.
- To describe the response rate of VOD in patients receiving defibrotide.
- To describe the toxicities of defibrotide in patients with VOD.
- To describe the response rate of VOD in patients receiving defibrotide.
Inclusion Criteria:
Patients with clinical or pathologic diagnosis of veno-occlusive disease. The following
criteria will be used:
1. Hyperbilirubinemia (greater than or equal to 2mg/dl) and two of the following:
(hepatomegaly, ascites, or weight gain [greater than or equal to 5% of baseline]);
OR,
2. Patient who do not fulfill criteria in #1 but who have pathologic or radiographic
evidence of VOD.
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