High-dose chemotherapy in children with metastatic hepatoblastoma

Background : Despite the advent of effective chemotherapy, a poor prognosis has been reported for patients with metastatichepatoblastoma. To improve this prognosis, we conducted high-dose chemotherapy with autologous bone marrow rescue in patients withmetastatic hepatoblastoma. Methods and results : Three patients were treated with high-dose chemotherapy. In patient 1, high-dose chemotherapywas given after the patient's first pulmonary relapse. Additional pulmonary metastases, which developed more than 6 monthsafter high-dose chemotherapy, were treated by multiple thoracotomy without additional chemotherapy. Patient 2 presented additionalpulmonary metastases soon after the end of the first thoracotomy and high-dose chemotherapy. Because of a decreased serum alpha-fetoproteinlevel after re-excision of the pulmonary metastases, a second round of high-dose chemotherapy was performed. In patient 3, multiplepulmonary metasteses responded to preoperative chemotherapy and disappeared according to the chest computed tomography. Intensivetreatment with a high-dose chemotherapeutic regimen was performed at the end of postoperative chemotherapy. All three patients are aliveand 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-dosechemotherapy, may improve the outcome of the patients with metastatic hepatoblastoma.