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Current and Future Management Strategies for Relapsed or Progressive Hepatoblastoma

  • Therapy In Practice
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Abstract

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.

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Acknowledgments

No sources of funding were used to prepare this manuscript. The authors have no conflicts of interest that are directly relevant to the content of this review.

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Authors and Affiliations

  1. Division of Hematology/Oncology, Children’s Hospital Los Angeles, Los Angeles, California, USA

    Rajkumar Venkatramani

  2. Department of Hematology/Oncology, St Jude Children’s Research Hospital, Memphis, Tennessee, USA

    Wayne L. Furman

  3. Department of Pediatric Surgery and Pediatric Urology, University of Tübingen, Tübingen, Germany

    Joerg Fuchs & Steven W. Warmann

  4. Division of Hematology/Oncology/Bone Marrow Transplant, Children’s Hospital of Wisconsin and the Medical College of Wisconsin, MFRC 3018, MS 756, 8701 Watertown Plank Rd, Milwaukee, Wisconsin, WI 53226, USA

    Marcio H. Malogolowkin MD

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  1. Rajkumar Venkatramani
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  2. Wayne L. Furman
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  3. Joerg Fuchs
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  4. Steven W. Warmann
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  5. Marcio H. Malogolowkin MD
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Correspondence to Marcio H. Malogolowkin MD.

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Venkatramani, R., Furman, W.L., Fuchs, J. et al. Current and Future Management Strategies for Relapsed or Progressive Hepatoblastoma. Pediatr Drugs 14, 221–232 (2012). https://doi.org/10.2165/11597740-000000000-00000

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