A cohort of 50 patients with untreated locally advanced or metastatic pancreatic ductal adenocarcinoma and a germline mutation in BRCA1/2 or PALB2 was randomized to receive gemcitabine/cisplatin with or without veliparib in this multicenter phase II trial. The response rate was numerically but not statistically superior in the veliparib triplet group. The median progression-free and overall survival were also similar between groups.
These results exceeded pre-study thresholds of efficacy, with particularly encouraging 2-year (31%) and 3-year (18%) survival rates for the entire cohort.
Gemcitabine plus cisplatin represents the optimal first-line option for patients with pancreatic cancer with these germline mutations.