Sorafenib in pediatric hepatocellular carcinoma from a clinician perspective

Hepatocellular Carcinoma (HCC) is a rare tumor in children and normally carries poor outcomes. The most frequently employed chemotherapy regimen includes cisplatin and doxorubicin (PLADO), but this combination offers limited efficacy. Sorafenib is a multi-tyrosine kinase inhibitor which, following positive studies in adults with HCC, has begun to be introduced in conjunction with PLADO in pediatric oncology with some encouraging results. Based on these findings, the use of sorafenib is become more common in children with unresectable and/or metastatic HCC. The care of patients receiving sorafenib requires appropriate expertise and standardized pediatric guidelines are lacking. An increasing number of children with HCC are expected to receive sorafenib in the years to come. Pediatric oncology clinicians have a key role in identifying side effects early and clinicians caring for children receiving sorafenib need to be familiar with these. This review article provides suitable and practical information on sorafenib for educational development to optimize clinical care and facilitate enhanced patient/parent education. The article addresses specific areas including mechanisms of action, pre-clinical and clinical evidence, dosing and drug administration and toxicities of sorafenib. Clinical research and recommendations for managing sorafenib-related side effects are discussed. Underpinned by research, this article provides pediatric oncology clinicians with the knowledge required to deliver optimal care to children receiving sorafenib.