The experimental drug defibrotide reversed severe veno-occlusive disease (VOD) of the liver in more than one third of the stem cell transplant recipients enrolled in the study. VOD is a type of potentially fatal liver damage that can result from the high doses of chemotherapy given prior to a transplant. The findings, which will be published in the Dec. 15 issue of Blood, have been posted as a "First Edition Paper" on the journal's web site (www.bloodjournal.org).
"Stem cell transplant patients suffering from severe veno-occlusive disease are at a very high risk of death, with a mortality rate in excess of 90 percent. The results from this study are compelling, especially given the remarkable safety of the drug in this extremely sick population," says Paul G. Richardson, MD, a hematologic oncologist at Dana-Farber who led the multi-center study. "This trial, which is the largest of its kind to date, confirms prior reports from smaller studies, and provides a strong platform for us to move forward with further trials of defibrotide, both in therapy for established VOD and its prevention."
Five to 60 percent of stem cell transplant patients develop VOD, which ranges in
severity from mild and reversible to severe and almost always fatal. Stem cell
recipients have an elevated risk for VOD due to the high-dose chemotherapy they
undergo prior to transplantation and the type of transplant they have, with
allogeneic (unrelated or related donor stem cells) transplant recipients being
at greater risk than autologous recipients (using the patient's own stem cells).
Chemotherapy drugs can damage the cells lining the small blood vessels in the
liver as well
Contact: Bill Schaller
Dana-Farber Cancer Institute