Abstract
Background: The mainstays of irresectable hepatoblastoma (HB) treatment are surgical resection and cisplatin based (CB) chemotherapy (CHT). However, adequate patient selection is a key to achieve acceptable disease-free survival in patients with unresectable HB undergoing liver transplantation (LT).
Procedure: This single-center retrospective analysis of 28 children with HB submitted to LDLT from 1996 to 2019 aimed at determining the pre-transplant factors associated with worse post-transplant event-free survival. The clinical variables collected were gender, age, PELD score (Pediatric End-Stage Liver Disease scoring system), type of neoadjuvant CHT (CB versus other regimens), pre- and post- CHT AFP levels, %AFP reduction post CHT (AFP pre-CHT – AFP post- CHT /AFP pre- CHT), PRETEXT stage, primary versus rescue LDLT, time between diagnosis and LDLT, presence of metastases at diagnosis, follow-up time.
Results: Patients were divided in groups according to the occurrence of the event (recurrence/death) after LDLT – 10 patients in the event-yes and 18 patients in the event-no. Probability of 5-y event-free survival was 63.9%. AFP reduction < 70% (HR=4.33, 95%CI 1.1 to 16.95, p=0.03), and time from diagnosis to LT > 12 months (HR=4.11, 95%CI 1.14 to 14.76, p=0.03) were associated with higher recurrence/death in the Cox regression analysis. Alpha-fetoprotein (AFP) reduction post-CHT > 70% had a good performance in determining disease-free survival, with a calculated AUC of 0.8.
Conclusion: LT for HB is the preferred treatment option for unresectable HB, with no distant metastasis and adequate response to CHT. AFP reduction < 70%, and time from diagnosis to LT > 12 months were associated with higher recurrence/death However, due to the limited number of patients in this study, a larger number of patients is required to corroborate these findings.