HEALTH PROFESSIONAL · SOURCE READING
Orthotopic liver transplant
Source: Childhood Liver Cancer Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: January 6, 2025 · Captured 2026-09-09.
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Context: Treatment Option Overview for Childhood Liver Cancer / Surgery
Liver transplants have been associated with significant success in the treatment of children with unresectable hepatic tumors.[19]; [20-22][Level of evidence C1] A review of the world experience has documented a posttransplant survival rate of 70% to 80% for children with hepatoblastomas.[17,23-25] Intravenous vascular invasion, positive lymph nodes, and contiguous extrahepatic spread did not have significant adverse effects on outcome. Adjuvant chemotherapy after transplant may decrease the risk of tumor recurrence, but its use has not been studied definitively in a randomized clinical trial.[26]
Evidence (orthotopic liver transplant):
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27]
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The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27]
The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following:
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following:
Diagnosis.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Diagnosis.
For the study period of 1987 to 2012, the 5-year survival rate was 76% and the graft survival rate was 77% for patients with hepatoblastoma. The survival and graft survival rates were 63% for patients with hepatocellular carcinoma.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Diagnosis.
For the study period of 2009 to 2012, the 3-year survival and graft survival rates were 84% for patients with hepatoblastoma. The survival and graft survival rates were 85% for patients with hepatocellular carcinoma.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following:
Transplant era.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Transplant era.
The death rate by hazard ratio was 1.0 for the period before 2002, 0.72 for the period of 2002 to 2009, and 0.54 for the period of 2009 to 2012.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following:
Medical condition at transplant.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Medical condition at transplant.
For hepatoblastoma patients, the survival rate by hazard ratio was 1.0 for hospitalized patients versus 1.81 for nonhospitalized patients at the time of transplant.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Medical condition at transplant.
For hepatocellular carcinoma patients, the survival rate by hazard ratio was 1.0 for hospitalized patients versus 1.92 for nonhospitalized patients.
The United Network for Organ Sharing (UNOS) database was queried for all patients younger than 18 years with a primary malignant liver tumor who underwent an orthotopic liver transplant between 1987 and 2012 (N = 544). The patients were diagnosed with hepatoblastoma (n = 376, 70%), hepatocellular carcinoma (n = 84, 15%), and other tumors (n = 84, 15%). Patients with hepatocellular carcinoma were older, more often hospitalized at the time of transplant, and more likely to receive a cadaveric organ than were patients with hepatoblastoma.[27] › The 5-year patient survival rate was 73%, and the graft survival rate was 74% for the entire cohort, with most deaths resulting from malignancy. On multivariate analysis, independent predictors of 5-year patient and graft survival included the following: › Medical condition at transplant.
Patients hospitalized in the intensive care unit did not fare worse than patients not in the intensive care unit.
A report of 149 patients with hepatocellular carcinoma younger than 21 years who underwent transplants between 1987 and 2015 used detailed data collected at all U.S. pediatric transplant centers.[19]
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A report of 149 patients with hepatocellular carcinoma younger than 21 years who underwent transplants between 1987 and 2015 used detailed data collected at all U.S. pediatric transplant centers.[19]
The 1-year graft survival rate of about 85% did not differ from the survival rate for patients with hepatoblastoma or biliary atresia. Survival rates continued to decline over time, from 85% at 1 year to 52% at 5 years and 43% at 10 years, a more dramatic decline than that seen for hepatoblastoma or biliary atresia.
A report of 149 patients with hepatocellular carcinoma younger than 21 years who underwent transplants between 1987 and 2015 used detailed data collected at all U.S. pediatric transplant centers.[19]
The survival after transplant did not differ from that of adults who underwent transplant for hepatocellular carcinoma.
A report of 149 patients with hepatocellular carcinoma younger than 21 years who underwent transplants between 1987 and 2015 used detailed data collected at all U.S. pediatric transplant centers.[19]
Of the patients with hepatocellular carcinoma, 22 received a diagnosis after transplant for medical cirrhotic disease such as tyrosinemia. They had a superior outcome, but it was not statistically significant compared with the rest of the patients.
A review of the Surveillance, Epidemiology, and End Results (SEER) Program database and many single-institution series have reported results similar to the UNOS database study described above.[11,20-22,28]; [25][Level of evidence C1]
In a three-institution study of children with hepatocellular carcinoma, the overall 5-year disease-free survival rate was approximately 60%.[29]
Source links and citations
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
Source links and citations
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
The 3-year event-free survival (EFS) rate was 81% for patients with hepatoblastoma who received a transplant (n = 157).
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
The 3-year EFS rate was 62% for patients with hepatocellular carcinoma who received a transplant (n = 18).
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
Of the patients who received a transplant to treat hepatoblastoma, 6.9% had PRETEXT II disease and 15.3% had POSTTEXT I/II disease.
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
Tumor extent did not impact survival (P = NS).
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
Patients who received transplants for salvage (n = 13) and patients who received transplants for primary hepatoblastoma had similar 3-year EFS rates (62% vs. 78%; P = NS).
In a study that used the Society of Pediatric Liver Transplantation (SPLIT) database to identify patients who underwent liver transplant between 2011 and 2019, the following was reported:[30][Level of evidence C2]
Among patients who received transplants for hepatocellular carcinoma, the 3-year EFS rate was poorer in older patients (38% for patients aged ≥8 years vs. 86% for patients aged <8 years; P < .001).
Application of the Milan criteria for UNOS selection of recipients of deceased donor livers is controversial.[31,32] The Milan criteria for liver transplant are directed toward adults with cirrhosis and hepatocellular carcinoma. The criteria do not apply to children and adolescents with hepatocellular carcinoma, especially those without cirrhosis.
Cirrhosis is an underlying risk factor for the development of hepatocellular carcinoma in children who suffer from certain diseases or conditions. These diseases include perinatally acquired hepatitis B, hepatorenal tyrosinemia, progressive familial intrahepatic cholestasis, glycogen storage disease, Alagille syndrome, and other conditions. Improvements in screening methodology have allowed for earlier identification and treatment of some of these conditions, as well as monitoring for development of hepatocellular carcinoma. Nevertheless, because of the poor prognosis of patients with hepatocellular carcinoma, liver transplant should be considered for diseases or conditions that have resulted in early findings of cirrhosis, before the development of liver failure or malignancy.[33]
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Living-donor liver transplant for hepatic malignancy is more common in children than adults, and the outcome is similar to those undergoing cadaveric liver transplant.[34,35] In patients with hepatocellular carcinoma, gross vascular invasion, distant metastases, lymph node involvement, tumor size, and male sex were significant risk factors for recurrence. In one report, 33 patients with hepatoblastoma and 10 patients with hepatocellular carcinoma were treated with living-donor liver transplants. For the hepatoblastoma patients, the 5-year OS rate was 87.4%, and the EFS rate was 75.8%. The 5-year OS and EFS rates were 75.4% for the patients with hepatocellular carcinoma. The presence of renal vein invasion was associated with an increased incidence of recurrence and death (P = .28).[36][Level of evidence C1]
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Publication references
Read the original reference and check its publication notices.
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Preserved source evidence · Not medical advice
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