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Treatment of Hepatoblastoma With High-dose Chemotherapy and Stem Cell Rescue: The Pediatric Blood and Marrow Transplant Consortium Experience and Review of the Literature

Karski, Erin E. MD; Dvorak, Christopher C. MD; Leung, Wing MD; Miller, Weston MD; Shaw, Peter J. MD; Qayed, Muna MD; Katsanis, Emmanuel MD; Feusner, James H. MD

Journal of Pediatric Hematology/Oncology: July 2014 - Volume 36 - Issue 5 - p 362–368
doi: 10.1097/MPH.0000000000000130
Original Articles

Background: Children with high-risk or relapsed hepatoblastoma continue to represent treatment challenges. Multiple case reports have documented the use of high-dose chemotherapy with stem cell rescue (HDC) for this population; however, the efficacy and appropriate use of HDC remains unclear.

Procedure: A literature search was performed to identify cases of hepatoblastoma that were treated with HDC. Additional patients were identified by a query through the Pediatric Blood and Marrow Transplant Consortium. All cases were categorized as undergoing HDC as part of their initial treatment or for relapsed disease. Overall survival (OS) and event-free survival (EFS) proportions were calculated for each group. Subgroup analyses were performed looking at the effects of remission status, initial stage, and relapse site.

Results: Forty-two patients were identified. Thirty-one patients received HDC as part of their initial treatment and 55% were long-term survivors with 48% event-free. Eleven received HDC for relapsed disease and 64% were long-term survivors, 36% without events.

Conclusions: It is difficult to draw firm conclusions from a small number of nonrandomized patients who had different stages, treatments, and events before undergoing HDC. However, our calculated EFS and OS proportions are consistent with current data using multimodal therapy without HDC, suggesting that HDC (at least as currently delivered) for hepatoblastoma may not be beneficial.

*Department of Pediatrics, University of California San Francisco, San Francisco

#Children’s Hospital and Research Center Oakland, Oakland, CA

St. Jude Children’s Research Hospital, Memphis, TN

Department of Pediatrics, University of Minnesota, Minneapolis, MN

§Children’s Hospital at Westmead, University of Sydney, NSW, Sydney

Department of Pediatrics, Emory University School of Medicine, Atlanta, GA

Department of Pediatrics, University of Arizona, Tucson, AZ

Supported by NIH T32 CA128583 (E.E.K.); no additional funding sources of conflicts of interest to report.

The authors declare no conflict of interest.

Reprints: Erin E. Karski, MD, 505 Parnassus Avenue, M649, San Francisco, CA 94143 (e-mail:

Received July 4, 2013

Accepted January 24, 2014

Copyright © 2014 Wolters Kluwer Health, Inc. All rights reserved.