Phase II trial of oral doxifluridine plus oral levo-leucovorin in unresectable hepatocellular carcinoma

Systemic chemotherapy with currently available agents in unresectable HCC has a minimal impact on disease progression and a predictable response rate of 2, day 1 through 5, cycles being repeated every 10 days. Thirty-seven patients with unresectable HCC entered the study and are evaluable for response and toxicity. Three partial responses have been observed, to a global response rate of 8% (95% confidence interval 2-22%). After a median observation time of 12 months, the median survival was 7 months, with a median time to progression of 4 months. Main toxicity was diarrhea; severe in 30% of the patients. One patient died as a result of uncontrollable diarrhea. In view of the limited activity observed, further trials with this schedule are not warranted.