Current and future management strategies for relapsed or progressive hepatoblastoma

Rajkumar Venkatramani, Wayne L. Furman, Joerg Fuchs, Steven W. Warmann, Marcio Malogolowkin

Research output: Contribution to journalArticlepeer-review

17 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)221-232
Number of pages12
JournalPediatric Drugs
Issue number4
StatePublished - Jun 25 2012
Externally publishedYes


  • 1-Phosphatidylinositol-3-kinase-inhibitors
  • Angiogenesis-inhibitors
  • Antineoplastics
  • Beta-Catenin-inhibitors
  • Bevacizumab
  • Carboplatin
  • Children
  • Cisplatin
  • Cyclophosphamide
  • Doxorubicin
  • Doxorubicin-liposomal
  • Epidermal-growth-factor-receptor-antagonists
  • Etoposide
  • Fluorouracil
  • fms-like-tyrosine-kinase-3-inhibitors
  • Gene-therapies
  • Hedgehog-cell-signalling- pathway-inhibitors
  • Ifosfamide
  • Immunotherapies
  • Irinotecan
  • Liver cancer
  • Liver-transplant
  • mTOR-protein-inhibitors
  • Oxaliplatin
  • Pazopanib
  • Platelet-derived-growth-factor-receptor-antagonists
  • Poly(ADP-ribose)-polymerase- inhibitors
  • Proto-oncogene-protein-c-akt-inhibitors
  • Proto-oncogene-protein-c-kit inhibitors
  • Proto-oncogene-protein-c-met-inhibitors
  • Proto-oncogene-protein-c-ret- inhibitors
  • Raf-kinase-inhibitors
  • Somatomedin inhibitors
  • Sorafenib
  • Sunitinib
  • Surgery
  • Vincristine
  • Wnt-signalling-pathway-inhibitors.

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pharmacology (medical)


Dive into the research topics of 'Current and future management strategies for relapsed or progressive hepatoblastoma'. Together they form a unique fingerprint.

Cite this