Abstract

Although the combination of cisplatin and gemcitabine (GEM) is considered the standard first-line chemotherapy against unresectable hilar cholangiocarcinoma (HC), its efficacy is discouraging. The present randomized open-label clinical trial aimed to evaluate the efficacy and safety of the GEM plus S-1 (GEM-S-1) combination against unresectable HC. Twenty-five patients per group were randomly assigned to receive GEM, S-1 or GEM-S-1. Neutropenia (56%) and leukopenia (40%) were the most common chemotherapy-related toxicities in the GEM-S-1 group. Median overall survival (OS) in the GEM-S-1, GEM and S-1 groups was 11, 10 and 6 months, respectively. GEM plus S-1 significantly improved OS compared to S-1 monotherapy (OR=0.68; 95%CI, 0.50–0.90; P=0.008). Median progression-free survival (PFS) times in the GEM-S-1, GEM and S-1 groups were 4.90, 3.70 and 1.60 months, respectively. GEM plus S-1 significantly improved PFS compared to S-1 monotherapy (OR=0.50; 95%CI, 0.27–0.91; P=0.024). Response rates were 36%, 24% and 8% in the GEM-S-1, GEM and S-1 groups, respectively. A statistically significant difference was found in response rates between the gemcitabine-S-1 and S-1 groups (36% vs 8%, P=0.017). Patients with CA19-9<466 U/ml were more responsive to chemotherapeutic agents than those with CA19-9≥571 U/ml (88.9% vs 0%, P<0.001). We conclude that the combination of GEM plus S-1 provides a better OS, PFS and response rate than S-1 monotherapy, but it did not significantly differ from GEM monotherapy. (ChiCTR-TRC-14004733).

Highlights

  • Hilar cholangiocarcinoma (HC) is the most frequently identified cholangiocarcinoma and accounts for 58–66% of cholangiocarcinoma cases in China [1, 2]

  • No significant differences were found among the three groups in age, gender, carbohydrate antigen 19-9 (CA19-9), carcinoembryonic antigen (CEA), BismuthCorlette classification, International Union for Cancer Control (UICC) stage, metastases or biliary drainage (P>0.05)

  • The GEM plus S-1 combination clearly provides benefits in unresectable pancreatic cancer [20], discrepant findings have been published for this combination in unresectable HC [21, 22]

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Summary

Introduction

Hilar cholangiocarcinoma (HC) is the most frequently identified cholangiocarcinoma and accounts for 58–66% of cholangiocarcinoma cases in China [1, 2]. A preclinical study indicated that S-1 and GEM had synergistic effects [15], and a multicenter phase II study showed that this combination was promising for treating advanced biliary tract cancer [16]. It had a relative small sample size (a total of 35 patients) and among the 35 patients, 14 patients had gallbladder cancer, 14 had intrahepatic cholangiocarcinoma and 7 patients had received previous surgical resection. The present prospective study assessed 75 cases of unresectable HC to evaluate the efficacy and safety of GEM and S-1 combined chemotherapy

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