Background: Central nervous system germ cell tumors are heterogeneous and rare pediatric tumors with high chemo-radiosensitivity. Despite the good overall prognosis, non-germinomatous lesions have a higher risk of relapse and may therefore require personalized treatment. Gemcitabine is one of the chemotherapeutic agents used in refractory/relapsed cases. We describe the case of liver veno-occlusive disease from Gemcitabine in a patient with high-risk metastatic non-germinomatous germ cell tumor refractory to first-line treatment. Methods: A 7-year-old male patient diagnosed with metastatic high risk non-germinomatous germ cell tumor of the pineal gland was treated according to SIOP CNS GCT II indications. The patient showed progression during chemotherapy, developing a new left temporal hemorrhagic lesion. In consideration of the high hemorrhagic risk, the patient was started on second-line chemotherapy with a Gemcitabine-Oxaliplatin regimen. A dose of 1000 mg/mq of gemcitabine was administered on days 1 and 8 of a 21-day cycle. The patient received 4 total cycles of chemotherapy obtaining a partial response to treatment with a reduction of over 60% of the hemorrhagic lesion. Approximately 10 days after the last administration of Gemcitabine, the patient showed platelet refractoriness, alteration of liver function, weight gain and contraction of diuresis. In the suspicion of liver veno-occlusive disease, abdominal ultrasound was performed with a study of hepatic flows that confirmed our hypothesis. The patient promptly started treatment with defibrotide and supportive therapy with diuretics, platelets and coagulation factors; abdominal drainage was also necessary. Results: After 21 days of defibrotide, the patient achieved complete resolution of the liver veno-occlusive disease. Brain MRI confirmed the response to chemotherapy and patient started craniospinal irradiation. Conclusion: The majority of veno-occlusive disease occur in patients undergoing bone marrow transplantation. Gemcitabine-induced liver veno-occlusive disease is rare and has never been reported in pediatric age.
Gemcitabine in pediatric refractory CNS GCT: a case of liver veno-occlusive disease
E Miele;
2026-01-01
Abstract
Background: Central nervous system germ cell tumors are heterogeneous and rare pediatric tumors with high chemo-radiosensitivity. Despite the good overall prognosis, non-germinomatous lesions have a higher risk of relapse and may therefore require personalized treatment. Gemcitabine is one of the chemotherapeutic agents used in refractory/relapsed cases. We describe the case of liver veno-occlusive disease from Gemcitabine in a patient with high-risk metastatic non-germinomatous germ cell tumor refractory to first-line treatment. Methods: A 7-year-old male patient diagnosed with metastatic high risk non-germinomatous germ cell tumor of the pineal gland was treated according to SIOP CNS GCT II indications. The patient showed progression during chemotherapy, developing a new left temporal hemorrhagic lesion. In consideration of the high hemorrhagic risk, the patient was started on second-line chemotherapy with a Gemcitabine-Oxaliplatin regimen. A dose of 1000 mg/mq of gemcitabine was administered on days 1 and 8 of a 21-day cycle. The patient received 4 total cycles of chemotherapy obtaining a partial response to treatment with a reduction of over 60% of the hemorrhagic lesion. Approximately 10 days after the last administration of Gemcitabine, the patient showed platelet refractoriness, alteration of liver function, weight gain and contraction of diuresis. In the suspicion of liver veno-occlusive disease, abdominal ultrasound was performed with a study of hepatic flows that confirmed our hypothesis. The patient promptly started treatment with defibrotide and supportive therapy with diuretics, platelets and coagulation factors; abdominal drainage was also necessary. Results: After 21 days of defibrotide, the patient achieved complete resolution of the liver veno-occlusive disease. Brain MRI confirmed the response to chemotherapy and patient started craniospinal irradiation. Conclusion: The majority of veno-occlusive disease occur in patients undergoing bone marrow transplantation. Gemcitabine-induced liver veno-occlusive disease is rare and has never been reported in pediatric age.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione.
