Response to therapy as a criterion for awarding priority to patients with hepatocellular carcinoma awaiting liver transplantation.

Background. How to prioritize patients with hepatocellular carcinoma (HCC) for liver transplantation (LT) remains controversial. This study was designed to assess the effectiveness of a policy for prioritizing HCC patients according to their response to pre-LT therapy. Methods. The study period was from 2000 to 2008. Dropout criteria included macroscopic vascular invasion, metastases, and poorly differentiated grade at pre-LT biopsy. A specific treatment algorithm was adopted to treat HCC before LT, and the effect of treatment was evaluated 3 months after listing or after the diagnosis of HCC for patients diagnosed while already on the waiting list. Patients were divided into two groups: group 1, patients with disease that completely or partially responded to therapy; and group 2, patients with stable, progressive, or untreatable disease. Group 2 patients were prioritized for LT unless full restaging and repeat biopsy identified dropout criteria. Results. At the 3-month visit, 62 HCC patients (42%) were assigned to group 2 and 85 (58%) to group 1. Eleven of 12 dropouts due to tumor progression came from group 2 (P.01). Response to therapy was the sole predictor of dropout probability, independent of tumor stage (competing risk analysis). The 42 patients in group 2 who were transplanted had much the same 3-year post-LT survival rate as the 57 transplanted patients in group 1 (with survival rates of 82% and 83%, respectively; P[0.05), but a slightly higher risk of post-LT HCC recurrence (13% and 2%, respectively; P = 0.04). Conclusions. Response to therapy is a potentially effective tool for prioritizing HCC patients for LT.