Liver Transplantation for Pediatric Hepatoblastoma and Hepatocellular Carcinoma: Out of the Unknown.

Abstract

Withadvances inchemotherapy, surgical treatment, andpostoperative care, the prognosis for children with hepatoblastoma (HBL) has improved significantly during the past 3 decades. The combined use of liver transplantation and chemotherapy isnowthepreferred treatment option for unresectableHBL.1 Inpediatric patientswithhepatocellular carcinoma (HCC), transplantationhasbeenassociatedwith improved disease-free survival compared with conventional treatment with chemotherapy and resection, particularly in children with advanced-stage tumors or chronic underlying liver disease.2 In this issue of JAMA Surgery, Pham et al3 report excellent long-term outcomes at their center after liver transplantation for childhood HBL and HCC, with 5-year disease-free survival rates of 82% and 78%, respectively. Similar to other published reports in the literature, this study is limited by its retrospective nature and the small numbers of patients. The authors proposed several risk factors associated with tumor recurrence after transplantation, but proper multivariable analysis was not performed. Furthermore, patient selection protocols can vary from one transplant center to another, which may lead to notable differences in observed outcomes. The Pediatric Liver Unresectable Tumor Observatory (PLUTO) registry was initiated in 2006 by the International Childhood Liver Tumors Strategy Group (SIOPEL) and now contains prospectively collected data on more than 200 pediatric patients who have undergone transplant for unresectable liver tumors in 79 centers around the world. The primary objectives of the registry are (1) to fully define the indications for liver transplantation in the treatment of pediatric liver tumors, (2) to identify the optimal timing for transplantation, (3) to define the role of pretransplant and posttransplant chemotherapy, and (4) to determine the best strategy for immunosuppression.4 An interim report of the registry5 has demonstrated an expected long-term survival rate of approximately 90% after liver transplantation for unresectable HBL. For children with HCC in the setting of underlying liver disease, liver transplantation produces excellent long-term survival and cure rates, even without the use of chemotherapy. In pediatric patients without chronic liver disease who develop unresectable HCC tumors, Related article page 1150 Research Original Investigation Survival in ChildrenWith Hepatoblastoma and Hepatocellular Cancer