Factors determining long-term outcomes of hepatocellular carcinoma within the Milan criteria: liver transplantation versus locoregional therapy

Patients with hepatocellular carcinoma (HCC) satisfying the Milan criteria are candidates for liver transplantation (LT), but locoregional therapies could be another options for them.A total of 1859 treatment-naïve HCC patients fulfilling the Milan criteria were analyzed. Survival tree analysis was performed to generate survival nodes with similar survival risks in 1729 non-LT group, and compared with the survival of 130 patients who received LT.Among patients who did not receive LT, survival tree analysis classified patients into 6 nodes according to Child-Pugh (CP) score, serum alphafetoprotein (AFP) levels, tumor size, and age, with different mortality risks (5-year survival rate of 87.3%, 77.5%, 65.8%, 64.7%, 44.0%, and 28.7% for nodes 1-6, respectively; P < 0.001). The overall survival of patients in nodes 1 (CP score 5 with AFP levels <5 ng/mL) and 2 (CP score 5 with maximal tumor size <2.5 cm) were comparable with that of patients who received LT (both P > 0.05), but the survival rates of patients in nodes 3 to 6 were worse than that of LT (P < 0.05 for all). In each survival node, survival differed slightly according to initial treatment modality for patients who did not receive LT. For patients who received LT, tumor stage at the time of LT was associated with long-term outcome.Certain groups of non-LT patients showed survival rates that were similar to the survival rates of LT patients. CP score, AFP levels, tumor size, and age were baseline factors that can help estimate the long-term outcomes of non-LT treatment. In addition, tumor stage at the time of LT and specific initial treatment modality in non-LT patients affected the long-term outcomes. These factors can help estimate the long-term outcomes of HCC patients diagnosed within the Milan criteria.