ma VOLUME 69 Mayo Clinic Proceedings JUNE 1994 Orthotopic Liver Transplantation for Preoperative Early-Stage Hepatocellular Carcinoma CHEE-KIAT TAN, M.B.,B.S.,* GREGORY J. GORES, M.D., JEFFERY L. STEERS, M.D., MICHAEL K. PORAYKO, M.D., J. EILEEN HAY, M.D., JORGE RAKELA, M.D., RUSSELL H. WIESNER, M.D., AND RUUD A. F. KROM, M.D., PH.D. • Objective: To report our experience with orthotopic liver transplantation (OLT) for highly se- lected patients with early-stage hepatocellular carci- noma (HCC). • Design: We retrospectively analyzed the demo- graphic, clinical, pathologic, and survival data on 21 patients with HCC who underwent OLT at the Mayo Clinic between 1985 and 1993. • Material and Methods: The 21 patients were cat- egorized into three groups: (1) those with incidental HCC (no evidence of HCC preoperatively), (2) those with a unicentric hepatic lesion without vascular inva- sion, and (3) those with an increased serum a-fetopro- tein (AFP) concentration but no detectable mass le- sion in the liver. • Results: For the-seven patients with incidental HCC, the 2-year disease-free survival was 68.5%. For the eight patients with a mass lesion, the 2-year dis- ease-free survival was only 50%. Operative staging revealed more advanced stage disease than had been found on preoperative assessment in five of these eight patients. For the six patients with an increased serum AFP value but no mass lesion, the 2-year disease-free survival was 80%. Tumor recurrence was the major cause of all deaths in this series. • Conclusion: Disease-free survival for patients with radiographie early-stage HCC was suboptimal because of understaging of the disease preoperatively. In contrast, our initial experience with OLT for pa- tients with an increased serum AFP value in the ab- sence of a mass lesion in the liver was favorable. (Mayo Clin Proc 1994; 69:509-514) AFP = -fetoprotein; HCC = hepatocellular carcinoma; OLT = orthotopic liver transplantation; TNM = staging based on tu- mor, nodes, and metastatic involvement Primary hepatocellular carcinoma (HCC) is one of the most common malignant lesions worldwide. No proven or uni- versally accepted medical therapy is available for HCC, and For accompanying editorial, see page 599 surgical removal provides the only possibility for long-term benefit. Many lesions, however, are unresectable because of From the Division of Gastroenterology and Internal Medicine (C.K.T., G.J.G.. M.K.P., J.E.H., J.R., R.H.W.) and Division of Transplantation Sur- gery (J.L.S., R.A.F.K.), Mayo Clinic Rochester, Rochester, Minnesota. Current address: Singapore General Hospital, Singapore. This study was supported in part by the Mayo and Gainey Foundations. Address reprint requests to Dr. G. J. Gores, Division of Gastroenterology, Mayo Clinic Rochester, 200 First Street SW, Rochester, MN 55905. the site of involvement, underlying liver disease, or extent of the malignant process. Even when resection is feasible, patients often have tumor recurrence, and 5-year survival rates are only 30 to 36%.'"3 The advent of orthotopic liver transplantation (OLT) of- fered a new and promising alternative mode of surgical treatment of HCC. Patients whose HCC was once consid- ered unresectable because of coexisting liver disease or ex- tensive malignant disease could undergo transplantation with a hope of cure. Both the malignant tumor itself and the cirrhotic liver, which is fertile soil for the development of metachronous malignant lesions, are removed by OLT. Overall, however, the results of OLT for advanced HCC have been dismal the 5-year survival rate has been only 20%.4 Because survival of patients who undergo OLT for Mayo Clin Proc 1994; 69:509-514 509 © 1994 Mayo Foundation for Medical Education and Research