Officials with the U.S. Food and Drug Administration (FDA) recently approved a new treatment, Defitelio (defibrotide sodium), for people who need stem cell or bone marrow transplants.
These patients have hepatic veno-occlusive disease (VOD) as well as lung or kidney abnormalities. Typically, they need hematopoietic stem cell transplantation (HSCT) as part of their treatments. Patients have chemotherapy treatments just before they receive an HSCT treatment.
This is the first time that the FDA has approved of a treatment for severe hepatic VOD, which is a life-threatening, rare liver condition. This condition is found in patients with chemotherapy and HSCT, as some of the liver’s veins are blocked over time. This results in the liver swelling and losing blood flow, causing liver damage.
“The approval of Defitelio fills a significant need in the transplantation community to treat this rare but frequently fatal complication in patients who receive chemotherapy and HSCT,” Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, said.
The worst kind of hepatic VOD gives the patient kidney and liver failure. Approximately two percent of patient develop the severe form of hepatic VOD after they receive HSCT; approximately 80 percent of these patients die.