Oral eniluracil/5-fluorouracil in patients with inoperable hepatocellular carcinoma

Background: Conventional systemic chemotherapy currently available for patients with inoperable hepatocellular carcinoma is ineffective. The purpose of this study was to evaluate the safety and efficacy of eniluracil/5-fluorouracil (5-FU) in the treatment of patients with this highly refractory disease. Patients and methods: This multicenter. open-label study evaluated a 28-day oral regimen of 5-FU (1 mg/m(2) twice daily) plus the dihydropyrimidine dehydrogenase inhibitor, eniluracil (10 mg/m(2) twice daily). in patients with chemotherapy-naive or anthiacycline-refractory inoperable hepatocellular carcinoma. Results: A total of 36 patients enrolled into the study. No patient showed a confirmed partial or complete tumor response, although nine patients (25%) had a best response of stable disease. The median duration of progression-free survival was 9.6 weeks [95% confidence interval (CI) 9.1-10.6 weeks]. and the median duration of overall survival was 32.7 weeks (95% CI 17.4-71.6 weeks). Eniluracil/5-FU was well tolerated. Diarrhea, the most frequent treatment-related non-hematological toxicity. occur-red in 11 patients (31%). Hematological toxicities were infrequent and usually mild. Conclusions: Eniluracil/5-FU as a 28-day oral outpatient regimen is well tolerated by patients with inoperable hepatocellular carcinoma, although minimal activity was observed when given as monotherapy at the dose used in this study.