Researchers affiliated with International Childhood Liver Tumor Strategy Group (SIOPEL) 2 trial have reported that neoadjuvant and adjuvant treatment with Platinol® (cisplatin) alone is as effective as Adriamycin® (doxorubicin) plus Platinol, with less toxicity, in children with standard-risk hepatoblastoma. The details of this study were published in the October 22, 2009 issue of the New England Journal of Medicine.[1]

Hepatoblastoma is an uncommon malignant liver tumor occurring in infants and children. Hepatoblastoma is usually accompanied by an elevation in serum alpha-fetoprotein (AFP). Absence of an elevated AFP is considered an adverse prognostic sign. Usual treatment is with neoadjuvant and adjuvant chemotherapy, surgical resection, and, in some cases, liver transplantation.  Intrahepatic tumor extension and lung metastases are also adverse prognostic findings. Patients are currently stratified into standard-risk and high-risk groups. Standard-risk patients have tumor that is confined to the liver and not involving more than three hepatic sections. High-risk hepatoblastoma involves the entire liver, the portal vein, the right and left branches, or all three hepatic veins, or the inferior vena cava. High-risk patients can also have intra-abdominal disease and/or lung metastases. 

Neoadjuvant and adjuvant chemotherapy has usually involved Platinol and an anthracycline such as Adriamycin or Ellence® (epirubicin). Alternative regimens include Platinol with 5-FU and vincristine. The current study was carried out to determine if an anthracycline was necessary for the treatment of standard-risk children with hepatoblastoma.

This study included 126 children with standard-risk hepatoblastoma who were randomly allocated to receive pre- and postoperative Platinol or Platinol plus Adriamycin.

• The complete remission rate was 95% for the Platinol alone group and 93% for patients in the Platinol and Adriamycin group.
• 3-year event-free survival was 83% for patients receiving Platinol alone and 85% for patients receiving Platinol and Adriamycin.
• 3-year overall survival was 95% for patients receiving Platinol alone and 98% in patients receiving Platinol and Adriamycin.
• Grade 3-4 toxicities occurred in 20.6% of patients receiving Platinol alone and 74.4% in patients receiving Platinol and Adriamycin.

Comments: A less toxic regimen of Platinol alone appears to be the new standard for treating standard-risk hepatoblastoma in children.

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