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

Treatment Options for Stages I and II

  • Hepatoblastoma of pure fetal histology: For tumors of pure fetal histology, complete surgical resection followed by watchful waiting or single-agent doxorubicin.[1]

    In the Children's Oncology Group (COG) study COG-P9645, stage I pure fetal histology hepatoblastomas with two or fewer mitoses per 10 high power fields were not treated with chemotherapy. Completely excised tumor of purely fetal and favorable histology may be carefully followed without further therapy.[1] A small focus of undifferentiated small cell histology within an otherwise pure fetal histology tumor must be treated with aggressive chemotherapy.[2]

  • Hepatoblastoma with non–pure fetal histology: Gross surgical excision followed by four courses of combination chemotherapy with cisplatin, vincristine, and fluorouracil or cisplatin and doxorubicin or cisplatin alone.[3,4,5,6]

    Combination chemotherapy has been demonstrated to have significant benefit in children with hepatoblastoma. Cisplatin-based chemotherapy has resulted in a survival rate of greater than 90% for children with postsurgical stage I and stage II disease.[3,4,5,7]

    A randomized clinical trial demonstrated comparable efficacy with cisplatin/vincristine/fluorouracil and cisplatin/doxorubicin in the treatment of hepatoblastoma. Although outcome was nominally higher for children receiving cisplatin/doxorubicin, this difference was not statistically significant, and the combination of cisplatin/vincristine/fluorouracil was significantly less toxic than the doses of cisplatin/doxorubicin, to which it was compared.[6]

Treatment Options for Stage III

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  • Chemotherapy followed by reassessment of surgical resectability followed by complete surgical resection.

    In approximately 75% of children and adolescents with initially unresectable hepatoblastoma, tumors can be rendered resectable with cisplatin-based preoperative chemotherapy, and 60% to 65% will survive disease-free.[8]

    A North American randomized clinical trial demonstrated comparable efficacy with cisplatin/vincristine/fluorouracil and cisplatin/doxorubicin in the treatment of hepatoblastoma. Although outcome was nominally higher for children receiving cisplatin/doxorubicin, this difference was not statistically significant, and the combination of cisplatin/vincristine/fluorouracil was significantly less toxic than the doses of cisplatin/doxorubicin used.[6]

    A combination of ifosfamide, cisplatin, and doxorubicin has also been successfully used in the treatment of advanced-stage disease.[9] A regimen of intensified platinum therapy with alternating cisplatin and carboplatin was associated with a decrease in event-free survival (EFS).[10]

  • Chemotherapy followed by reassessment of surgical resectability. If the primary tumor remains unresectable, an orthotopic liver transplantation may be performed.

    Patients whose tumors remain unresectable should be considered for liver transplantation.[5,11,12,13,14,15] In the presence of features predicting unresectability, early coordination with a pediatric liver transplant service is desirable.[16]

  • An alternative treatment approach of transarterial chemoembolization for surgically unresectable disease.[17,18]
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