Background: Chemoembolization with lipiodol (iodized oil) is widely used to treat patients with unresectable hepatocellular carcinoma. Severe side effects have been reported, and improved survival has not been clearly demonstrated.
Methods: Patients with unresectable hepatocellular carcinoma who did not have severe liver disease and who met additional entry criteria were randomly assigned to receive either lipiodol chemoembolization (70 mg of cisplatin, 10 ml of lipiodol, and gelatin-sponge [Gelfoam] particles delivered through the hepatic artery) or conservative management involving treatment of complications and pain. Courses of treatment were to be given every two months for a maximum of four courses. The main end point was survival.
Results: The study was stopped in December, 1992, after a sequential analysis showed the lack of the expected benefit from chemoembolization. As of October 1, 1994, 39 of the 50 patients assigned to chemoembolization and 40 of the 46 patients assigned to conservative management had died. Twenty-six patients assigned to chemoembolization received all four courses of treatment. There was no significant difference in survival between the two groups, although there was a trend favoring the chemoembolization group (estimated relative risk of death in the control group, 1.4; 95 percent confidence interval, 0.9 to 2.2; P = 0.13). The comparison of survival between the two groups was not substantially changed by adjustments for differences in base-line and prognostic characteristics (adjusted relative risk, 1.3; 95 percent confidence interval, 0.8 to 2.1; P = 0.31). At one year, the estimated survival rates were 62 percent in the chemoembolization group (95 percent confidence interval, 48.6 to 75.4 percent) and 43.5 percent in the conservative-management group (95 percent confidence interval, 29.2 to 57.8 percent). In the chemoembolization group, tumor growth, as assessed by tumor size and serum alpha-fetoprotein concentration, was reduced and the incidence of portal obstruction was lower than in the conservative-management group. Liver failure occurred after 47 courses of treatment in 30 patients assigned to chemoembolization.
Conclusions: In a group of patients with unresectable hepatocellular carcinoma but without severe liver disease, lipiodol chemoembolization reduced tumor growth, often caused acute liver failure, and did not significantly improve survival.