Hepatocellular carcinoma (HCC) is a major cause of mortality worldwide
, and no effective systemic therapy currently exists. Recombinant alph
a-interferon (IFN) has been suggested to have some antitumor efficacy
in this illness, and synergism with 5-fluorouracil (5-FU) has been rep
orted in several gastrointestinal malignancies. We therefore treated 1
0 patients with advanced HCC with combination therapy consisting of 5-
FU 750/mg/m(2) weekly and IFN 9 x 10(6) units three times weekly. Toxi
city was substantial in this cirrhotic population, and included mucosi
tis as well as neurologic and hematologic side effects. There were no
sustained antitumor responses. Median survival among this heavily pret
reated population was 10 months. We were therefore unable to demonstra
te any significant benefit to treatment with 5-FU and IFN in patients
with HCC.