Hepatic veno-occlusive disease: a chemotherapy-related toxicity in children with malignancies.
Hepatic veno-occlusive disease (VOD) is a major manifestation of liver toxicity associated with conventional and high-dose chemotherapy in children affected by hematologic malignancies and certain solid tumors. Clinically, patients present with jaundice, painful hepatomegaly, and fluid retention, which may evolve into multi-organ failure, a hallmark of severe disease. The pathogenesis is complex and not completely understood, but the damage to sinusoidal endothelium, typically caused by toxic metabolites released from antineoplastic drugs, is thought to play a crucial role, together with cytokine activation, immune deregulation, and coagulopathy. Diagnosis is based on clinical criteria supported by characteristic ultrasound findings, with the gold standard investigation being hepatic-venous pressure gradient measurement and biopsy. Several treatment options have been tested; the most convincing approach to date is the use of defibrotide, a novel oligonucleotide with antithrombotic and antiplatelet aggregating properties, as well as endothelial-stabilizing effects. This agent, together with other specific forms of supportive care, has shown efficacy in the treatment of established VOD and promising results in the prevention of VOD in pediatric patients receiving chemotherapy.
Cefalo
Maria Giuseppina
MG
Division of Paediatric Oncology, Catholic University of Rome, Rome, Italy.
Maurizi
Palma
P
Arlotta
Annalisa
A
Scalzone
Maria
M
Attinà
Giorgio
G
Ruggiero
Antonio
A
Riccardi
Riccardo
R
D000818:Animals; D000970:Antineoplastic Agents / Q000009:adverse effects* / Q000627:therapeutic use; D002648:Child; D006504:Hepatic Veno-Occlusive Disease / Q000139:chemically induced* / Q000175:diagnosis / Q000503:physiopathology / Q000628:therapy; D006801:Humans; D009369:Neoplasms / Q000150:complications / Q000188:drug therapy*; D011379:Prognosis; D012307:Risk Factors
D000970:Antineoplastic Agents
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