SOS for veno-occlusive disease: defibrotide prophylaxis

Defibrotide

“…1 VOD is a rare and life-threatening disease that affects approximately 20% of patients who receive high-dose myeloablative conditioning therapy or HSCT. [2][3][4][5][6] The disease is characterized clinically by increased serum bilirubin, hepatomegaly, fluid retention, and weight gain. 2,7 Its pathophysiology is of complex vascular origin consisting of hepatic venules and sinusoidal endothelial injury.…”

“…4 Typically, mild to moderate cases of VOD are self-limiting and resolve with minimal treatment; however, severe cases are associated with liver failure, hepatorenal syndrome, and multiorgan failure, with a mortality rate as high as 84%. 2,3,6,8 Defibrotide is the only approved drug for the treatment of VOD with renal or pulmonary dysfunction following HSCT; however, patients may also be helped by supportive care, including diuresis, transfusion, renal replacement therapy, and analgesia. 1,8 Experimental approaches to the prevention of VOD include administration of ursodeoxycholic acid, heparin, glutamine, antithrombin III, and prostaglandin E 1 ; however, these methodologies have shown limited efficacy.…”

Nutrition Challenges in a Patient With Sinusoidal Obstructive Syndrome Following an Allogeneic Stem Cell Transplant

“…The syndrome is characterized by hyperbilirubinemia, jaundice, weight gain, ascites, and painful hepatomegaly that develops within approximately 10–20 days after the start of cyclophosphamide‐based cytoreductive therapy and later after other myeloablative regimens 2 (Table 1). Symptoms are related to the narrowing and occlusion of hepatic venules, sinusoidal fibrosis, and hepatocyte necrosis as a result of chemotherapy toxicity 3 , 4 . The incidence of SOS varies from ~5%–60% and is more common after allogeneic HSCT than after autologous HSCT 5 .…”