1. Median overall survival was greater in the pembrolizumab group compared to the control group (12.7 months vs. 10.9 months).
2. Treatment-related adverse events were comparable among patients in either group.
Evidence Rating Level: 1 (Excellent)
Study Rundown: Biliary tract cancers are a complex group of epithelial tumors with a poor prognosis. The first-line therapy for advanced biliary tract cancer includes chemotherapy with gemcitabine and cisplatin. Immune checkpoint inhibitors, such as pembrolizumab, may be effective in treating biliary tract cancer although their benefits remain unclear. This randomized controlled trial aimed to compare the safety and efficacy of adding pembrolizumab to gemcitabine and cisplatin in patients with advanced biliary tract cancer. The primary outcome was the median overall survival while the key secondary outcome included frequency of grade 3 or 4 adverse events. According to study results, patients in the pembrolizumab group demonstrated significantly increased overall survival without any significant differences in safety outcomes compared to the control group. This study was strengthened by a randomized design with patients from all over the world, thus adding to its generalizability.
Click to read the study in The Lancet
In-depth [randomized-controlled trial]: Between Oct 4, 2019, and Jun 8, 2021, 1564 patients were screened for eligibility across 175 sites globally. Included were patients ≥ 18 years with histologically confirmed locally advanced or metastatic biliary tract cancer and an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. Altogether, 1069 patients (533 in pembrolizumab plus gemcitabine and cisplatin and 536 in placebo plus gemcitabine and cisplatin) were included in the final analysis. The primary outcome of overall survival was significantly greater in the pembrolizumab group (12.7 months, 95% confidence interval [CI] 11.5-13.6) versus the placebo group (10.9 months, 95% CI 9.9-11.6, hazard ratio [HR] 0.83, p=0.0034). The secondary endpoint of safety was comparable with 70% of patients in the pembrolizumab and 69% in the placebo group reporting treatment-related adverse events (accounting for 2% and 1% of deaths, respectively). Findings from this study suggest that pembrolizumab added to gemcitabine and cisplatin improved overall survival among patients with biliary tract cancer.
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