Nationwide survey of the outcomes of living donor liver transplantation for hepatoblastoma in Japan.

Recently, liver transplantation (LT) has been increasingly performed for unresectable hepatoblastoma (HB) with acceptable results. We conducted a national survey of cases undergoing living donor liver transplantation (LDLT) for HB to evaluate their outcomes. Thirty-nine patients (28 males and 11 females with a median age at LDLT of 3.6 years) who had undergone LDLT for HB by the end of 2009 were enrolled in this study. The clinical data were collected from their medical records via a questionnaire survey in 2011 (median follow-up = 4.6 years). Thirteen patients (33.3%) had extrahepatic lesions before LDLT. Thirty-eight patients (97.4%) received chemotherapy, and 15 (38.5%) underwent hepatectomy before LDLT. Twenty-seven patients (69.2%) were alive without recurrence after LDLT, and 12 patients (30.8%) suffered from recurrence. The most common site of recurrence was the lung (9 cases), which was followed by the graft liver (6 cases). The median interval from LDLT to recurrence was 5.5 months. Four of the 9 cases (44.4%) with lung metastasis underwent surgical resection, and 3 were alive without any tumor recurrence. Eight patients died because of tumor recurrence. The multivariate landmark analysis revealed that the independent recurrence risk factors were a high alpha-fetoprotein (AFP) level at diagnosis [≥ 500,000 ng/mL; hazard ratio (HR) = 7.86, P = 0.010], the presence of extrahepatic lesions before LDLT (HR = 3.82, P = 0.042), and a high AFP level at LDLT (≥ 4000 ng/mL; HR = 9.19, P = 0.036). The actuarial 3- and 5-year patient survival rates were 84.3% and 77.3%, respectively. In conclusion, with appropriate timing for scheduled LT, LDLT provides a valuable alternative treatment with excellent results for children with HB.