More than 65% of patients with germline BRCA/PALB2-mutated pancreatic ductal adenocarcinoma responded to treatment with combination cisplatin and gemcitabine, according to results of a study reported in the Journal of Clinical Oncology. Two- and three-year survival rates reached 31% and 18%, respectively.  

Researchers for the open-label, multicenter, phase II clinical trial randomly assigned 50 patients to receive cisplatin, gemcitabine, and veliparib (arm A) or the cisplatin-gemcitabine combination without veliparib (arm B). The disease control rate was 100% and 78.3% for arms A and B, respectively. Median progression-free survival was 10.1 and 9.7 months and median overall survival 15.5 and 16.4 months, respectively, for arms A and B. When exploratory analysis looked at BRCA status, progression-free survival was 6.8 and 11.3 months and overall survival 14 and 20.2 months for BRCA1 and BRCA2, respectively.  

Seventy-four percent of patients in arm A and 26% in arm B required at least one dose reduction or discontinuation because of toxicity, primarily hematologic. Patients in arm A experienced grade 3 or 4 anemia (52%), thrombocytopenia (55%), and neutropenia (41%); in arm B the rates were 35%, 9%, and 30%, respectively.  

The researchers concluded that cisplatin plus gemcitabine was well tolerated and should be the first-line, standard-of-care treatment for patients with BRCA-mutated advanced pancreatic ductal carcinoma. “The doublet is the recommendation moving forward,” the researchers reported. “We believe these data define a reference regimen for germline BRCA-mutated and PALB2-mutated pancreatic cancer.”