Veno-occlusive disease in pediatric patients affected by Wilms tumor.
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.


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.
Pediatric Hematology Oncology, Department of Pediatrics, University of Padova, Padova, Italy. simone.cesaro@ospedaleuniverona.it
Simone
S
Cesaro
Monica
M
Spiller
Maria Teresa
MT
Sartori
Rita
R
Alaggio
Mattia
M
Peruzzo
Graziella
G
Saggiorato
Gianni
G
Bisogno
D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects*; D015415:Biomarkers / Q000097:blood; D002648:Child; D002675:Child, Preschool; D005260:Female; D005343:Fibrinolytic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D006504:Hepatic Veno-Occlusive Disease / Q000139:chemically induced* / Q000175:diagnosis / Q000188:drug therapy; D006801:Humans; D007223:Infant; D007680:Kidney Neoplasms / Q000150:complications* / Q000188:drug therapy; D008297:Male; D017395:Plasminogen Activator Inhibitor 1 / Q000097:blood*; D011089:Polydeoxyribonucleotides / Q000009:adverse effects / Q000627:therapeutic use*; D009396:Wilms Tumor / Q000150:complications* / Q000188:drug therapy
D015415:Biomarkers; D005343:Fibrinolytic Agents; D017395:Plasminogen Activator Inhibitor 1; D011089:Polydeoxyribonucleotides; C036901:defibrotide