defibrotide and Wilms-Tumor

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.. 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.. 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.. 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.

Topics: Antineoplastic Combined Chemotherapy Protocols; Biomarkers; Child; Child, Preschool; Female; Fibrinolytic Agents; Hepatic Veno-Occlusive Disease; Humans; Infant; Kidney Neoplasms; Male; Plasminogen Activator Inhibitor 1; Polydeoxyribonucleotides; Wilms Tumor

Sinusoidal obstruction syndrome (SOS) is a life-threatening complication generally occurring after hematopoietic stem cell transplantation. SOS after standard dose chemotherapy in malignancies is rare. Between the year 1995 and 2016, 414 patients were diagnosed with acute lymphoblastic leukemia and 113 patients were diagnosed with Wilms tumor in our institution. Among these patients, 4 patients with acute lymphoblastic leukemia (0.96%) and 2 patients with Wilms tumor (1.7%) developed SOS during treatment. SOS behaves like a local disseminated intravascular coagulation. Defibrotide has proved to be effective in SOS. In this article, we report our experience with defibrotide in SOS.

Topics: Antineoplastic Agents; Child; Hepatic Veno-Occlusive Disease; Humans; Neoplasms; Platelet Aggregation Inhibitors; Polydeoxyribonucleotides; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Wilms Tumor

We report a case of administration compassionate use defibrotide in a 13-year-old girl with Sinusoidal Obstructive Syndrome and thrombocytopenia, also known as Hepatopathy--Thrombocytopenia Syndrome (HTS) during chemotherapy for Wilms' tumor.

Topics: Adolescent; Chemical and Drug Induced Liver Injury; Compassionate Use Trials; Dactinomycin; Female; Humans; Polydeoxyribonucleotides; Syndrome; Thrombocytopenia; Wilms Tumor

Topics: Antineoplastic Agents; Chemotherapy, Adjuvant; Child, Preschool; Female; Hepatic Veno-Occlusive Disease; Humans; Kidney Neoplasms; Liver Function Tests; Neoadjuvant Therapy; Polydeoxyribonucleotides; Treatment Outcome; Ultrasonography, Doppler, Color; Wilms Tumor

Here we report a case of administration of defibrotide in an 11 months old infant with hepatic veno-occlusive disease during chemotherapy for nephroblastoma. He presented with abdominal distension, a weight gain of 15%, ascites, hepatomegaly with right upper quadrant pain, thrombocytopenia and hypertransaminasemia. Despite therapy, his clinical conditions aggravated, and, therefore intravenous administration of defibrotide on a compassionate-use basis was started. The dosage was 15 mg/kg/day in 4 divided doses, which was increased gradually (in 3 days) to 40 mg/kg/day in 4 divided doses. Defibrotide proved safe and effective in resolving clinical symptoms and normalizing serological findings in the syndrome.

Topics: Antineoplastic Agents; Compassionate Use Trials; Hepatic Veno-Occlusive Disease; Humans; Infant; Male; Polydeoxyribonucleotides; Wilms Tumor