Novel therapeutic approaches in the treatment of children with hepatoblastoma

Howard M. Katzenstein, Cynthia Rigsby, Peter H. Shaw, Torrey L. Mitchell, Paul R. Haut, Morris Kletzel

Research output: Contribution to journalArticlepeer-review

27 Scopus citations


Hepatoblastoma is the most common liver tumor diagnosed in children. Children with persistently unresectable disease, metastatic disease at presentation, recurrent disease, or slowly declining alpha-fetoprotein levels are at high risk for recurrence, exhibit an extremely poor prognosis, and are in desperate need of novel therapeutic agents and strategies. Four high-risk patients were treated. One patient with a local recurrence was treated with irinotecan followed by orthotopic liver transplant. Three patients were treated with tandem high-dose chemotherapy (HDT) with autologous stem cell rescue (two with primary metastatic disease and one with recurrent disease). All three of the patients treated with HDT had relapse (two of them subsequently received irinotecan); the remaining patient underwent surgical resection of a solitary recurrent pulmonary metastasis. Irinotecan demonstrated significant antitumor effects in all three treated patients and was well tolerated. None of the three patients treated with HDT remained disease-free, although the patient who underwent surgical resection of a solitary recurrent pulmonary metastasis remains disease-free 6 years from diagnosis. Further exploration of the use of irinotecan is warranted in high-risk patients with hepatoblastoma.

Original languageEnglish (US)
Pages (from-to)751-755
Number of pages5
JournalJournal of Pediatric Hematology/Oncology
Issue number9
StatePublished - Dec 1 2002
Externally publishedYes


  • Hepatoblastoma
  • High-dose therapy
  • Irinotecan
  • Peripheral blood stem cells
  • Transplant

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Hematology
  • Oncology


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