Defibrotide for children and adults with hepatic veno-occlusive disease post hematopoietic cell transplantation.
BACKGROUND Hepatic veno-occlusive disease (VOD) is a rare and potentially severe complication of chemotherapy. We describe five patients who developed VOD after chemotherapy for Wilms tumor (WT) and evaluate the role of plasminogen activator inhibitor-1 (PAI-1) and defibrotide for diagnosis and therapy of VOD, respectively. PATIENTS AND METHODS Thirty-five patients treated from 2002 to 2009 for WT were eligible. Diagnosis of VOD was according McDonald's criteria that required two of the following: jaundice, hepatomegaly and/or right upper quadrant pain, weight gain with or without ascites. RESULTS VOD occurred in 5 of 35 patients (14%) after 21-105 days from starting chemotherapy. Two patients developed multiorgan failure (MOF). PAI-1 was high in four patients who were tested. Three patients were treated with defibrotide and no side effects were reported while two patients received supportive measures only. Four patients recovered and three of them received defibrotide. They are all alive and well after a median follow-up of 35 months. One of two patients not treated with defibrotide died of MOF. CONCLUSIONS PAI-1 levels were abnormal in WT-VOD. Defibrotide was a safe, well-tolerated, and potentially efficacious therapy in this group of patients. Further prospective study is needed in WT-VOD to confirm these data.