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Original Investigation | Pacific Coast Surgical Association

Effect of Liver Transplant on Long-term Disease-Free Survival in Children With Hepatoblastoma and Hepatocellular Cancer

Thomas A. Pham, MD1; Amy M. Gallo, MD1; Waldo Concepcion, MD1; Carlos O. Esquivel, MD, PhD1; C. Andrew Bonham, MD1
[+] Author Affiliations
1Division of Abdominal Transplantation, Department of Surgery, Stanford University School of Medicine, Palo Alto, California
JAMA Surg. 2015;150(12):1150-1158. doi:10.1001/jamasurg.2015.1847.
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Introduction  Hepatoblastoma (HBL) and hepatocellular cancer (HCC) are the most common primary hepatic malignant neoplasms in childhood. Given the rarity of these childhood tumors and their propensity to present at advanced stages, updated long-term data are needed.

Objective  To determine the efficacy of liver transplant in children with HBL or HCC.

Design, Participants, and Setting  This single-institution retrospective medical record review and analysis spanned from January 1, 1997, through September 17, 2014, at Stanford University School of Medicine. A total of 40 patients younger than 18 years underwent liver transplant for treatment of HBL (n = 30) or HCC (n = 10) during the study period, with follow-up until September 17, 2014. Patients who underwent transplant for HCC included those with tumors that were greater in size than what is proposed by the Milan (a single tumor measuring ≤5 cm or ≤3 nodules measuring ≤3 cm) and University of California, San Francisco (single tumor measuring ≤6.5 cm or ≤3 nodules measuring ≤4.5 cm and a total diameter of ≤8 cm), criteria.

Main Outcomes and Measures  Disease-free and overall patient survival and graft survival.

Results  Using a Kaplan-Meier survival analysis, 1-, 5-, and 10-year disease-free survival after liver transplant was 93%, 82%, and 82%, respectively, for 30 patients with HBL and 90%, 78%, and 78%, respectively, for 10 patients with HCC. Risk factors associated with HBL recurrence after transplant included having pretreatment extent of disease stage IV lesions and a longer waiting list time and being older at the time of the transplant. Recurrence was found in 2 of 7 patients with HBL and pretransplant metastases, which were not found to be an independent risk factor for recurrence. Patients with HCC larger than the proposed Milan and University of California, San Francisco, criteria experienced good 5-year disease-free (82%) and overall (78%) survival after transplant. Being older at the time of transplant (18 vs 11 years; P = .04) and the presence of metastatic disease (1 patient vs none; P = .05) were associated with HCC tumor recurrence.

Conclusions and Relevance  Liver transplant combined with chemotherapy is an excellent treatment that provides long-term disease-free survival in children diagnosed with advanced HBL and HCC. Early addition to a waiting list and aggressive multimodal therapy provide excellent results. Transplant should still be considered in children with HCC larger than the Milan and University of California, San Francisco, criteria.

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Figure.
Kaplan-Meier Survival in Patients Who Underwent Liver Transplant for Hepatoblastoma (HBL) and Hepatocellular Cancer (HCC)

A, Patient survival at 1, 5, and 10 years is 93%, 89%, and 84%, respectively, for HBL and 90%, 90%, and 72%, respectively, for HCC (log-rank test, P = .23). B, Disease-free survival at 1, 5, and 10 years is 93%, 82%, and 82%, respectively, for HBL and 90%, 78%, and 78%, respectively, for HCC (log-rank test, P = .11). C, Graft survival at 10, 40, and 70 months is 86%, 83%, and 83%, respectively, for HBL and 100%, 100%, and 85%, respectively, for HCC (log-rank test, P = .11). We found no significant difference between patients with HBL and HCC in all 3 comparisons as determined by log-rank test.

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