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.
Central pontine and extrapontine myelinolysis are uncommon disorders characterized by distinctive clinical features and typical findings on neuroimaging. Only a few cases are reported in the pediatric age group. We describe the case of a leukemic, malnourished 14-year-old boy with a high serum sodium concentration that gradually increased to 170 mmol/L. During a septic shock episode, hydration with a low sodium concentration at the rate of 104 mL/h for 24 h was administered. A rapid correction of the high serum sodium occurred, exceeding 0.5 mmol/L/h. The following day the patient developed rapid and progressive neurological impairment with clinical features characteristic of central pontine and extrapontine myelinolysis. Magnetic resonance imaging confirmed the diagnosis 11 days later. The patient was treated with steroids and intravenous immunoglobulins. He achieved an almost full neurological recovery and radiological improvement. The reported case demonstrates that central pontine and extrapontine myelinolysis can occur after excessively rapid correction of hypernatremia.
Nausea and vomiting are respectively the first and third most relevant chemotherapy-associated side effects in pediatric oncology, despite the introduction of new antiemetic agents. Symptoms change according to the antineoplastic agents used, and are influenced by age since emotional and psychological aspects play an important role. Studies conducted on new antiemetic therapies usually focus on adult populations affected by tumors, whereas there are very few series including pediatric patients, who are even more severely affected by this specific side effect.
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