Abstract
BACKGROUND: Nanoliposomal irinotecan plus fluorouracil with leucovorin (NFF) is the standard treatment regimen after gemcitabine-based therapy in patients with unresectable pancreatic cancer. However, data on the efficacy and safety of NFF in terms of the presence or absence of biliary drainage (BD) or serum bilirubin levels prior to NFF are limited. Therefore, we analyzed whether these factors affect the efficacy and safety of NFF in the real world. METHODS: The NAPOLEON-2 study consisted of a retrospective and a prospective phase. As the retrospective phase, we retrospectively evaluated 161 consecutive patients who received NFF as second- or later line treatment. The primary endpoint was overall survival (OS); other endpoints included progression-free survival, response rate, disease control rate, dose intensity, and adverse events (AEs). We compared the endpoints between the non-BD group and BD group first, and then between the serum total bilirubin ≥ 1.0 mg/dL group and < 1.0 mg/dL group. RESULTS: All patients received gemcitabine prior to NFF. No significant difference in OS was observed between the non-BD and BD groups (9.1 vs 7.6 months; hazard ratio (HR), 1.09; 95% confidence interval (CI), 0.72-1.66; P = 0.69). The rates of severe hematological AEs and biliary tract infections were higher in the BD group than in the non-BD group. A significant difference in OS was noted between the bilirubin ≥ 1.0 mg/dL and < 1.0 mg/dL group (5.4 vs 8.9 months; HR, 2.13; 95%CI, 1.18-3.84; P = 0.01). In addition, the rate of severe hematological AE was higher in the high bilirubin group (56% vs. 26%). CONCLUSIONS: BD had minimal impact on the efficacy of NFF in daily practice. Patients with total bilirubin ≥ 1.0 mg/dL had shorter OS than those with total bilirubin < 1.0 mg/dL.