Gemcitabine, cisplatin and nab-paclitaxel phase 2 (MD Anderson and Mayo)

This open-label single-arm phase 2 (NCT02392637) at MD Anderson and Mayo Clinic Arizona enrolled 62 patients with advanced biliary tract cancer between 2015 and 2017; of 60 treated, 63 percent had intrahepatic cholangiocarcinoma, 15 percent extrahepatic and 22 percent gallbladder cancer, and 78 percent had metastatic disease. Starting doses of gemcitabine 1,000, cisplatin 25 and nab-paclitaxel 125 mg/m² on days 1 and 8 of 21-day cycles were reduced to 800, 25 and 100 mg/m² after haematological toxicity in the first 32 patients. Median progression-free survival, the primary endpoint, was 11.8 months (95 percent confidence interval 6.0 to 15.6), the partial response rate 45 percent, disease control 84 percent and median overall survival 19.2 months. The figures compared favourably with the 8.0 and 11.7 months of gemcitabine and cisplatin in ABC-02 and led directly to SWOG S1815, which found no survival benefit; the contrast is a reminder that single-arm phase 2 results in selected academic-centre populations overestimate effects.