Endoscopic radiofrequency ablation plus a novel oral 5-fluorouracil compound versus radiofrequency ablation alone for unresectable extrahepatic cholangiocarcinoma

Background and Aims: Endoscopic radiofrequency ablation (RFA) is a new ablative treatment for unresectable extrahepatic cholangiocarcinoma (EHCC). A novel 5-fluorouracil compound, S-1 (Taiho Pharmaceutical Co, Ltd, Tokushima Plant. Japan), has been widely used as a key drug with first-line or second-line chemotherapy for the treatment of advanced cholangiocarcinoma. The aim of this study was to evaluate the clinical efficacy and safety of endoscopic RFA combined with S-1 for the treatment of unresectable locally advanced EHCC.Methods: Patients with unresectable EHCC were prospectively randomized to 1 of 2 groups: the RFA + S-1 group and the RFA group. Median overall survival (OS), stent patency time, Karnofsky performance status (KPS) score, and adverse events rate were analyzed.Results: The median OS was longer in the RFA + S-1 group (n = 37) than that in the RFA group (n = 38) (16.0 months [95% confidence interval, 13.1-19.0] vs 11.0 months [95% confidence interval, 9.7-12.3]; P < .001). Stent patency time was significantly longer in the RFA + S-1 group than that in the RFA group (6.6 1.5 vs 5.6 +/- .1 months, P = .014). KPS scores at postoperative month 9 (51.6 +/- 17.0 vs 40.4 +/- 16.4, P = .012) and month 12 (35.2 +/- 18.3 vs 23.9 +/- 11.4, P = .014) were all higher in the RFA + S-1 group than those in the RFA group (P < .05). The incidence of ERCP-related adverse events was not significantly different between RFA+S-1 and RFA groups (8.1% vs 10.5%, P > .05).Conclusions: For the treatment of locally advanced EHCC, endoscopic RFA combined with S-1 is associated with longer survival and stent patency and improved functional status than RFA alone.