The Lynx Group
Cholangiocarcinoma News

Phase 2 Study Assessing Tolerability, Efficacy, and Biomarkers for Durvalumab ± Tremelimumab and Gemcitabine/Cisplatin in Chemo-Naïve Advanced BTC

Web Exclusives — May 30, 2020

Immunotherapies have shown early promising efficacy in some patients with biliary tract cancer (BTC). The authors assessed durvalumab (D) (anti–PD-L1) ± tremelimumab (T) (anti–CTLA-4), and gemcitabine/cisplatin (GemCis) as first-line treatment in Korean patients with BTC. The research team also performed an extensive biomarker analysis.1

Patients were first enrolled in the biomarker cohort to receive 1 cycle of gemcitabine 1000 mg/m2 + cisplatin 25 mg/m2 on days 1 and 8, followed by GemCis + D 1120 mg and tremelimumab 75 mg, every 3 weeks until disease progression. Subsequent patients were allocated to GemCis + D (3-combo cohort) or GemCis + D+T (4-combo cohort) until disease progression. In all cohorts, tumor biopsies were obtained pretreatment, after 1 cycle, and at disease progression. Blood samples for circulating tumor DNA were obtained at every cycle. Tumor mutational burden was assessed on pretreatment tumor samples, and PD-L1 expression was assessed on pre- and post-treatment tumor biopsy samples. A total of 121 patients were enrolled. Median follow-up durations were 28.5 months, 11.3 months, and 11.9 months in the biomarker cohort, 3-combo cohort, and 4-combo cohort arms, respectively.

Objective response rate and median overall survival were improved in cohorts 3 and 4 compared with the biomarker cohort. The most common adverse events (any grade) were nausea (59.5%), neutropenia (54.5%), and pruritus (54.5%). Candidate biomarkers were identified that may be indicative of response to these therapies. The most common grade 3/4 events were neutropenia (50.4%), anemia (35.5%), and thrombocytopenia (16.5%). The addition of immunotherapy to chemotherapy was tolerable and showed very promising efficacy. The combination of D + Gem/Cis is being investigated in the global phase 3 TOPAZ-1 trial (NCT03875235). number NCT03046862.


  1. Oh D-Y, et al. ASCO 2020. Abstract 4520.

Subscribe to CCA News

Stay up to date with personalized medicine by subscribing to recieve the free CCA News print publication or weekly e‑Newsletter.

I'd like to recieve: