Neoadjuvant Chemotherapy ( FOLFIRINOX or Gemcitabine With Nab‐Paclitaxel) for Borderline‐Resectable Pancreatic Cancer: Long‐Term Results of a Randomized Controlled Trial ( NUPAT ‐01)

BACKGROUND: Initial results of NUPAT-01 suggest the feasibility of multidrug neoadjuvant chemotherapy for borderline-resectable pancreatic cancer and favorable survival of patients, and we report on the long-term results of this study. METHODS: In this multicenter, phase II trial (NUPAT-01), patients with borderline-resectable pancreatic cancer were randomly assigned to receive neoadjuvant chemotherapy with either FOLFIRINOX (original regimen) or gemcitabine with nab-paclitaxel (GEM/nab-PTX) and underwent subsequent surgery if feasible. The primary endpoint was the R0 resection rate. RESULTS: Fifty-one eligible patients were randomly assigned to FOLFIRINOX (n = 26) or GEM/nab-PTX (n = 25). Forty-three patients underwent surgery, and R0 resection was achieved in 33 patients. An Intention-to-treat (ITT) analysis revealed a 3-year overall survival of 51.0% and a 5-year overall survival of 35.3% with a median survival time of 36.5 months. No significant difference between the FOLFIRINOX group and the GEM/nab-PTX group was found in the ITT analysis or in patient survival after surgery. However, patients with a favorable response to chemotherapy had significantly better disease-free survival. CONCLUSION: These results suggest no significant difference between the benefit of FOLFIRINOX and GEM/nab-PTX as neoadjuvant chemotherapy for borderline-resectable pancreatic cancer, and the survival of patients depends not on the regimen but on the response to chemotherapy.