Phase II study of second line gemcitabine single chemotherapy for biliary tract cancer patients with 5-fluorouracil refractoriness

Aim We conducted this phase II study in an effort to evaluate the efficacy and safety of a gemcitabine single chemotherapy as a second-line treatment for biliary tract cancer (BTC) patients who evidenced disease progression after the administration of 5-fluorouracil (5-FU)-based palliative chemotherapy. Patients and Method Patients treated previously with 5-FU-based palliative treatment as a BTC were enrolled in this study. Treatment consisted of gemcitabine at a dosage of 1,250 mg/m² administered intravenously over a 30-minute period on days 1 and 8 of each 21-day cycle until progression. Results Between Feb. 2006 and July 2009, a total of 32 patients were assigned to treatment groups. 16 patients (50%) had cancers of intra-hepatic cholangiocarcinoma, 12 patients (37.5%) had gall bladder cancer, and 4 patients (12.5%) had extra-hepatic cholangiocarcinoma. In the 29 patients whose tumor responses were evaluated, two achieved a partial response, with an overall response rate of 6.9% (95% confidence interval [CI]: 0.0–16.7%). Six patients (20.7%) evidenced stable disease and 21 patients (72.4%) evidenced progression during the course of treatment. The median follow-up duration was 23.2 months (range: 3.0–53.1 months). The median time to progression (TTP) was 1.6 months (95% CI: 1.3–1.9 months), and the median overall survival (OS) time was 4.1 months (95% CI: 2.7–5.5 months). Poor performance status (ECOG 2) in patients was predictive of shorter TTP. Lower albumin levels (<3.5 g/dL) in patients were predictive of shorter TTP and OS. Conclusions Despite first salvage chemotherapy in the phase II study for patients with 5-FU refractory BTC, the results in terms of RR, TTP, and OS were lower than expected. However, selected patients with good performance status and sufficient albumin levels may have derived some survival benefits from salvage chemotherapy.