Current and future management strategies for relapsed or progressive hepatoblastoma

Paediatr Drugs. 2012 Aug 1;14(4):221-32. doi: 10.2165/11597740-000000000-00000.


Hepatoblastoma is the most common primary malignant neoplasm of the liver in children. Improvements in chemotherapy and surgical techniques have increased survival rates for those with localized disease. The prognosis for patients with progressive or relapsed disease continues to be dismal. Complete resection by surgery or liver transplantation is necessary for cure. Few conventional chemotherapy agents have demonstrated activity in progressive or relapsed hepatoblastoma. Irinotecan has shown activity in relapsed and progressive hepatoblastoma. The efficacy of high-dose chemotherapy in this setting is unknown. Newer targeted agents that 'selectively' interfere with pathway targets involved in tumor growth and progression such as insulin-like growth factor, phosphatidylinositol 3-kinase (PI3K), Akt, and mammalian target of rapamycin (mTOR) are currently under development. Because of the rarity of hepatoblastoma, only a small minority of these agents will ever be evaluated in children with this disorder. Gene-directed therapy and immunotherapy have shown promising results in the preclinical setting, and should be investigated as future treatment options for advanced hepatoblastoma.

Publication types

  • Review

MeSH terms

  • Animals
  • Antineoplastic Agents / therapeutic use
  • Combined Modality Therapy
  • Genetic Therapy
  • Hepatoblastoma / drug therapy
  • Hepatoblastoma / surgery
  • Hepatoblastoma / therapy*
  • Humans
  • Immunotherapy
  • Liver Neoplasms / drug therapy
  • Liver Neoplasms / surgery
  • Liver Neoplasms / therapy*
  • Liver Transplantation
  • Molecular Targeted Therapy
  • Neoplasm Recurrence, Local


  • Antineoplastic Agents