Peng V Wu1, Arun Rangaswami2. 1. Division of Hematology/Oncology/Stem Cell Transplantation & Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, 1000 Welch Rd. Suite 300, Palo Alto, CA, 94304, USA. 2. Division of Hematology/Oncology, Department of Pediatrics, University of California San Francisco, 550 16th St., 3rd Floor, San Francisco, CA, 94158, USA. Arun.Rangaswami@ucsf.edu.
Abstract
PURPOSE OF REVIEW: As the most common pediatric primary liver cancer with rising incidence, hepatoblastoma remains challenging to treat. Here, we review the current understanding of the biology of hepatoblastoma and discuss how recent advances may lead to new treatment modalities. RECENT FINDINGS: Standard chemotherapy regimens including cisplatin, in addition to surgery, have led to high cure rates among patients with low stage hepatoblastoma; however, metastatic and relapsed disease continue to have poor outcomes. Recent genomics and functional studies in cell lines and mouse models have established a central role for the Wnt/β-catenin pathway in tumorigenesis. Targeted agents and immunotherapy approaches are emerging as potential treatment avenues. With recent gains in knowledge of the genomic and transcriptomic landscape of hepatoblastoma, new therapeutic mechanisms can now be explored to improve outcomes for metastatic and relapsed hepatoblastoma and to reduce the toxicity of current treatments.
PURPOSE OF REVIEW: As the most common pediatric primary liver cancer with rising incidence, hepatoblastoma remains challenging to treat. Here, we review the current understanding of the biology of hepatoblastoma and discuss how recent advances may lead to new treatment modalities. RECENT FINDINGS: Standard chemotherapy regimens including cisplatin, in addition to surgery, have led to high cure rates among patients with low stage hepatoblastoma; however, metastatic and relapsed disease continue to have poor outcomes. Recent genomics and functional studies in cell lines and mouse models have established a central role for the Wnt/β-catenin pathway in tumorigenesis. Targeted agents and immunotherapy approaches are emerging as potential treatment avenues. With recent gains in knowledge of the genomic and transcriptomic landscape of hepatoblastoma, new therapeutic mechanisms can now be explored to improve outcomes for metastatic and relapsed hepatoblastoma and to reduce the toxicity of current treatments.
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