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    Childhood Liver Cancer Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment Option Overview

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    Surgical resection for metastatic disease

    Surgical resection of distant disease has also contributed to the cure of children with hepatoblastoma. Resection of pulmonary metastases is recommended when the number of metastases is limited [26,27,28] and is often performed at the same time as resection of the primary tumor. When possible, resection of areas of locally invasive disease, such as in the diaphragm, and of isolated brain metastasis is recommended.[29]

    Chemotherapy

    In recent years, virtually all children with hepatoblastoma have been treated with chemotherapy, and in some centers, even children with resectable hepatoblastoma are treated with preoperative chemotherapy, which may reduce the incidence of surgical complications at the time of resection.[25,30,31]

    Presurgical chemotherapy

    In an international study, pre-resection neoadjuvant chemotherapy (doxorubicin and cisplatin) was given to all children with hepatoblastoma with or without metastases. The chemotherapy was well tolerated. Following chemotherapy, and excluding those who received liver transplant (less than 5% of patients), complete resection was obtained in 87% of children. This strategy resulted in an overall survival (OS) of 75% at 5 years after diagnosis for all children entered in the study.[30] Identical overall results were seen in a follow-up international study.[31] The International Society of Pediatric Oncology Epithelial Liver Tumor Group (SIOPEL) compared cisplatin alone with cisplatin and doxorubicin in patients with preoperative standard-risk hepatoblastoma. Standard-risk was defined as tumor confined to the liver and not involving more than three sectors. The rates of resection were similar for the cisplatin (95%) and cisplatin/doxorubicin (93%) groups, as were OS (95% and 93%), respectively.[32][Level of evidence:1iiA] SIOPEL has reported a pilot study of high-risk hepatoblastoma patients. In SIOPEL-3HR, cisplatin alternating with carboplatin/doxorubicin was administered in a dose intensive fashion. In 74 patients with PRETEXT stage 4 tumors, 22 of whom also had metastases, 31 became resectable and 26 underwent transplant. The 3-year OS of this group was 69% ± 11%. The 3-year OS of all patients with metastases was 62% ± 12%.[16] In a second trial, cisplatin was dose-intensified (timing, every 2 weeks) in a single-arm prospective study. Three-year event-free survival (EFS) was 76% and OS was 83%. Toxicity was significant but acceptable.[28][Level of evidence: 2A]

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