High-dose chemotherapy in children with metastatic hepatoblastoma

Background: Despite the advent of effective chemotherapy, a poor prognosis has been reported for patients with metastatic hepatoblastoma. To improve this prognosis, we conducted high-dose chemotherapy with autologous bone marrow rescue in patients with metastatic hepatoblastoma. Methods and results: Three patients were treated with high-dose chemotherapy. In patient 1, high-dose chemotherapy was given after the patient's first pulmonary relapse. Additional pulmonary metastases, which developed more than 6 months after high-dose chemotherapy, were treated by multiple thoracotomy without additional chemotherapy. Patient 2 presented additional pulmonary metastases soon after the end of the first thoracotomy and high-dose chemotherapy. Because of a decreased serum alpha-fetoprotein level after re-excision of the pulmonary metastases, a second round of high-dose chemotherapy was performed. In patient 3, multiple pulmonary metasteses responded to preoperative chemotherapy and disappeared according to the chest computed tomography. Intensive treatment with a high-dose chemotherapeutic regimen was performed at the end of postoperative chemotherapy. All three patients are alive and well, more than 6 years after receiving their diagnosis. Conclusion: The role of high-dose chemotherapy in treatment of metastatic hepatoblastoma could not be clarified, because of the small number of patients. However, the better outcome of our patients indicates that multimodal therapy, including high-dose chemotherapy, may improve the outcome of the patients with metastatic hepatoblastoma.