- Cisplatin+gemcitabine (CG) showed good efficacy in pancreatic ductal adenocarcinoma (PDAC) with BRCA/PALB2 mutations, and veliparib offered no additional response rate advantage but led to more hematological toxicity.
Why this matters
- The authors suggest that CG should be the standard approach for untreated PDAC.
- Open-label, randomized, multicenter, phase 2 study (n=50).
- Patients with untreated germline BRCA/PALB2 mutations with stage III-IV PDAC were randomized to CG or CG with veliparib (CG+V).
- Funding: NIH and nonindustry sources.
- The response rate was 65.2% for CG and 74.1% for CG+V (P=.55), both of which exceeded the prespecified activity threshold.
- Disease control rate, 78.3% for CG and 100% for CG+V (P=.02).
- Median PFS: for CG, 9.7 vs 10.1 months for CG+V (P=.73).
- Median OS: for CG, 16.4 vs 15.5 months for CG+V (P=.6).
- Overall OS:
- 2-year, 30.6% (95% CI, 17.8%-44.4%).
- 3-year, 17.8% (95% CI, 8.1%-30.7%).
- Grade 3-4 hematologic toxicities, CG vs CG+V:
- neutropenia, 30% vs 48%;
- thrombocytopenia, 9% vs 55%; and
- anemia, 35% vs 52%.
- Small sample size, open-label design.