Abstract
DEFIBROTIDE: A MAJOR BREAKTHROUGH TO FIGHT VENOOCCLUSIVE DISEASE (VOD) OF HEMATOPOIETIC STEM CELL TRANSPLANTATION

Dick B. S. Brashier, Kedar G. Bandekar*, Htet Wai Moe and Neha Akhoon

ABSTRACT

Venoocclusive disease (VOD) is a serious drawback of the standard Hematopoietic stem cell transplantation (HSCT) therapy. Day+ 100 survival of VOD patients after HSCT is very low (about 20%).[1] Previously the standard treatment for VOD was prophylactic and supportive which showed low evidence of effectiveness and high risk of adverse effects.[5] Recently, FDA approved the first therapy, Defibrotide for the treatment of this severe, rare and life-threatening hepatic VOD on March 30, 2016. Defibrotide was approved for the treatment of adult and pediatric patients suffering from hepatic veno-occlusive disease (VOD) with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).[10] Defibrotide increases tissue plasminogen activator (t-PA) and increases protection of activated endothelial cells from being damaged by chemotherapy, tumor necrosis factor-α (TNF-α), serum starvation and perfusion.[11]

Keywords: Defibrotide, Venoocclusive.


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