Defitelio is designed to treat hepatic veno-occlusive disease (VOD) associated with multi-organ dysfunction in patients post-hematopoietic stem-cell transplantation.

Mechanism of Action and Effectiveness

Defitelio is used to treat patients with hepatic veno-occlusive disease (VOD), a life-threatening complication following hematopoietic stem-cell transplantation (HSCT). VOD is characterized by endothelial injury and obstruction of the hepatic vasculature, often leading to liver dysfunction and multi-organ failure. The drug exerts its therapeutic effects by improving vascular endothelial function, thereby protecting against vascular damage and enhancing blood flow in the liver. Its role is critical in preventing the progression of VOD into severe hepatic and multi-organ failure, especially in patients with additional renal or pulmonary dysfunction.