V. Mazzaferro et al., LIVER-TRANSPLANTATION FOR THE TREATMENT OF SMALL HEPATOCELLULAR CARCINOMAS IN PATIENTS WITH CIRRHOSIS, The New England journal of medicine, 334(11), 1996, pp. 693-699
Background. The role of orthotopic liver transplantation in the treatm
ent of patients with cirrhosis and hepatocellular carcinoma is controv
ersial, and determining which patients are likely to have a good outco
me after liver transplantation is difficult. Methods. We studied 48 pa
tients with cirrhosis who had small, unresectable hepatocellular carci
nomas and who underwent liver transplantation. In 94 percent of the pa
tients, the cirrhosis was related to infection with hepatitis B virus,
hepatitis C virus, or both. The presence of tumor was confirmed by bi
opsy or serum alpha-fetoprotein assay. The criteria for eligibility fo
r transplantation were the presence of a tumor 5 cm or less in diamete
r in patients with single hepatocellular carcinomas and no more than t
hree tumor nodules, each 3 cm or less in diameter, in patients with mu
ltiple tumors. Twenty-eight patients with sufficient hepatic function
underwent treatment for the tumor, mainly chemoembolization, before tr
ansplantation. After liver transplantation, the patients were followed
prospectively for a median of 26 months (range, 9 to 54), No anticanc
er treatment was given after transplantation. Results. The overall mor
tality rate was 17 percent. After four years, the actuarial survival r
ate was 75 percent and the rate of recurrence-free survival was 83 per
cent. Hepatocellular carcinoma recurred in four patients (8 percent).
The overall and recurrence-free survival rates at four years among the
35 patients (73 percent of the total) who met the predetermined crite
ria for the selection of small hepatocellular carcinomas at pathologic
al review of the explanted liver were 85 percent and 92 percent, respe
ctively, whereas the rates in the 13 patients (27 percent) whose tumor
s exceeded these limits were 50 percent and 59 percent, respectively (
P=0.01 for overall survival; P=0.002 for recurrence-free survival). In
this group of 48 patients with early-stage tumors, tumor-node-metasta
sis status, the number of tumors, the serum alphafetoprotein concentra
tion, treatment received before transplantation, and 10 other variable
s were not significantly correlated with survival. Conclusions. Liver
transplantation is an effective treatment for small, unresectable hepa
tocellular carcinomas in patients with cirrhosis.