Efficacy and safety of liposomal irinotecan in patients with advanced pancreatic cancer: A prospective, multicenter, real-world study.

Abstract

720 Background: Liposomal irinotecan, a liposome-encapsulated form of irinotecan, effectively improves drug stability, preserves the active lactone ring structure, and enhances targeted distribution. It is approved for the treatment of advanced pancreatic cancer. This study aimed to evaluate the efficacy and safety of liposomal irinotecan in a real-world population with advanced unresectable pancreatic cancer. Methods: This real-world observational study comprised two cohorts. Cohort 1 included patients undergoing first-line treatment. Patients receiving liposomal irinotecan-containing regimens were assigned to the experimental group, while those administered AG regimen (nab-paclitaxel + gemcitabine) were placed in the control group. Cohort 2 is a single-arm group, consisted of patients receiving liposomal irinotecan-containing regimens as second-line treatment. The primary endpoint was progression-free survival (PFS). Secondary endpoints included objective response rate (ORR), overall survival (OS), and safety. Results: As of September, 2025, 521 patients were enrolled, including 357 in Cohort 1 (202 in the liposomal irinotecan group and 155 in the AG group) and 164 in Cohort 2. Baseline characteristics were generally balanced in Cohort 1. The most common regimens in the liposomal irinotecan group were liposomal irinotecan + oxaliplatin + fluoropyrimidine (70.3%). Among the 151 and 123 patients in the liposomal irinotecan and AG groups who had at least one tumor assessment, ORR was 24.5% (95% CI: 17.9-32.1) and 12.2% (95% CI: 7.0-19.3), and disease control rates (DCR) were 80.1% (95% CI: 72.9-86.2) and 69.9% (95% CI: 61.0-77.9), respectively. The median PFS was 8.7 months (95% CI 4.9-12.4) in liposomal irinotecan group and 5.8 (95% CI 4.2-7.3) months in AG group. The median OS was not reached in either group. The incidence of adverse events (AEs) were 73.3% and 63.2%, and grade ≥3 AEs occurred in 17.3% and 28.4% of patients, respectively. Baseline characteristics in Cohort 2 were consistent with epidemiological data. The most common regimens were liposomal irinotecan + fluoropyrimidine (52.4%) and liposomal irinotecan + oxaliplatin + fluoropyrimidine (22.0%). Among 113 patients with at least one tumor assessment, ORR was 15.9% (95% CI: 9.7-24.0) and DCR was 65.5% (95% CI: 56.0-74.2). Disease progression or death occurred in 73 subjects, with a median PFS of 5.9months (95% CI: 4.6-7.1). Thirteen patients died, and median OS was not reached. During treatment, 121 (73.8%) patients experienced AEs, and 39 (23.8%) had grade ≥3 AEs. No unexpected adverse events were observed. Conclusions: The results of this study demonstrated that liposomal irinotecan-containing regimens exhibit favorable efficacy and manageable safety in advanced unresectable pancreatic cancer in real world, warranting further investigation and follow-up. Clinical trial information: ChiCTR2300078041 .