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Novel Therapeutic Approaches in the Treatment of Children With Hepatoblastoma

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

Journal of Pediatric Hematology/Oncology: December 2002 - Volume 24 - Issue 9 - p 751-755
Clinical And Laboratory Observations

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.

From the Division of Hematology/Oncology, Department of Pediatrics (H.M.K., P.R.H., M.K.), and the Department of Radiology (C.R.), Northwestern University and Children's Memorial Hospital, Chicago, Illinois; Division of Hematology/Oncology, Department of Pediatrics, Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania (P.H.S.); and the University of Illinois, Rockford, Illinois (T.L.M.).

Submitted for publication December 3, 2001; accepted March 25, 2002.

Address correspondence and reprint requests to Howard M. Katzenstein, M.D., Children's Healthcare of Atlanta at Egleston, Division of Pediatric Hematology/Oncology, 2040 Ridgewood Drive, NE, Suite 100, Atlanta, GA 30322, U.S.A. E-mail:

© 2002 Lippincott Williams & Wilkins, Inc.