Nivolumab Added to Chemotherapy or Ipilimumab Offers No Survival Benefit in Advanced Biliary Tract Cancer

The addition of nivolumab to chemotherapy or ipilimumab conferred no benefit in progression-free survival (PFS) for patients with advanced biliary tract cancer (BTC) who had not previously received systemic therapy, according to a phase 2 trial.

Due to previous data, Vaibhav Sahai, MBBS, MS, Division of Hematology and Oncology, University of Michigan, Ann Arbor, MI and colleagues wrote, “we hypothesized an enhanced benefit of [immune checkpoint blockade (ICB)] as first-line treatment with anti-PD1 treatment in combination with chemotherapy or as dual ICB.”

The multi-center, open label phase 2 BilT-01 trial enrolled 75 patients, 68 of which received treatment and were evaluable, with advanced BTC with no prior systemic treatment between September 2017 and June 2019. Most patients had intrahepatic cholangiocarcinoma (61.8%) and metastatic disease (89.7%).

Patients were randomized to either 360 mg nivolumab on day 1, plus gemcitabine and cisplatin on days 1 and 8, every 3 weeks for 6 months, followed by 240 mg nivolumab every 2 weeks (Arm A, n = 35), or 240 mg nivolumab every 2 weeks plus 1 mg/kg ipilimumab every 6 weeks (Arm B, n = 33). While most baseline characteristics were similar between arms, there was a significantly higher proportion of patients who had previously undergone surgery in Arm A (n = 13) compared to Arm B (n = 5).

The primary end point was 6-month PFS. Secondary end points were median PFS and median overall survival (OS).

The median months of treatment was 6.6 for Arm A and 2.7 in Arm B. The median follow-up was 32.2 months in Arm A and 31.5 months for Arm B. The 6-month PFS was 59.4% for those patients in Arm A (95% confidence interval [CI], 40.5 to 74.0) and 21.2% for those in Arm B (95% CI, 9.4 to 36.3). The median PFS of Arm A was 6.6 months (95% CI, 3.4 to 7.7), compared to 3.9 months in Arm B (95% CI, 2.3 to 4.5). When compared to Arm B, PFS of Arm A was not found to be significantly better (P = .077) The median OS of Arm A was 10.6 months (95% CI, 6.4 to 24.5) compared to 8.2 months in Arm B (95% CI, 5.8 to 16.9; P = .61).

The study authors concluded that “the observed PFS rates…do not appear to represent an improvement over standard therapy” though “the high OS rate at 2 years in Arm A suggests benefit in a small cohort of patients.”

Source:

Sahai V, Griffith KA, Beg MS, et al. A randomized phase 2 trial of nivolumab, gemcitabine, and cisplatin or nivolumab and ipilimumab in previously untreated advanced biliary cancer: BilT-01. Cancer. Published online July 27, 2022. doi:10.1002/cncr.34394