Nab-paclitaxel plus cisplatin versus gemcitabine plus cisplatin as first-line treatment in advanced biliary tract cancer: results of a multicentre, randomised, phase II trial

Background: the efficacy and safety of conventional first-line chemotherapeutic regimens for the treatment of advanced biliary tract carcinomas (ABTCs) have been unsatisfactory. Objectives: We aimed to explore alternative chemotherapeutic regimens capable of providing a better efficacy and fewer side effects. Design: Multicentre, randomised, phase II clinical trial Methods : Eligible patients with unresectable, recurrent, or metastatic biliary system tumours diagnosed by pathology or cytology between January 2021 and November 2022 were included. The participants were randomised to either a gemcitabine-cisplatin group (GC) or an albumin-paclitaxel-cisplatin group (NC). Progression-free survival (PFS) was the primary outcome, whereas overall survival (OS), and objective response rate (ORR) were the secondary outcomes. Results : The trial enrolled 75 patients and had a median follow-up period of 11 months. The median PFS (mPFS) was 7.8 m (95% confidence interval [CI]: 5.4–14.0 m) in the NC group, and 7.0 m (95%CI: 3.9–10.1 m) in the GC group (p=0.0034, hazard ratio [HR]=0.5136, 95%CI: 0.3136–0.8411). Median OS for the NC group was 12.4 m (95%CI: 7.3–22.3 m) and for the GC group was 12.1 m (95%CI: 6.7–20.7 m), with no significant differences (p=0.4592, HR=0.811, 95%CI: 0.463–1.442). PFS rates at 6 and 8 months were 48.24% vs. 70.8% and 8.51% vs. 32.0% for the NC and the GC group, respectively (p < 0.05). Regarding safety, although patients from both groups experienced thrombocytopenia, those in the NC group demonstrated significantly lower rates (p < 0.05). Conclusion: Compared with the GC group, the NC group showed a significantly increased mPFS, in addition to higher PFS rates at 6 and 8 months. Furthermore, the NC group showed lower haematological toxicity. This suggests that paclitaxel-based regimens may be a viable alternative for first-line treatment of ABTCs. Registration: Clinical Trials.gov identifiers: NCT04692051. Registered October 31, 2018 (Web Links -https://www.chictr.org.cn/showproj.html?proj=38440)