A multicenter, prospective real-world study of irinotecan liposome (II)–based combination regimens in patients with pancreatic ductal adenocarcinoma.

Abstract

e16399 Background: Pancreatic ductal adenocarcinoma (PDAC) is a highly lethal malignancy with limited therapeutic options in advanced stages. Building upon the foundational evidence from the PAN-HEROIC-1 trial which demonstrated the clinical benefits of irinotecan liposome (II) and supported its approval for unresectable locally advanced or metastatic PDAC in China, this study aimed to evaluate the real-world safety and effectiveness of irinotecan liposome (II)-based combination regimens in patients with PDAC. Methods: This multicenter, prospective, real-world study included patients with pathologically or radiologically confirmed PDAC who were considered eligible by investigators for irinotecan liposome (II)-based combination regimens incorporating chemotherapy, chemoradiation, radiotherapy, immunotherapy, or targeted therapy. Patients were assigned to three cohorts according to treatment stage at enrollment: perioperative, first-line, and later-line treatment. Observational data were collected including adverse events, baseline characteristics, therapeutic regimens, and tumor response. The primary endpoint was treatment-emergent adverse events (TEAEs); secondary endpoints included overall survival (OS) and real-world progression-free survival (rwPFS), real-world time to progression (rwTTP), real-world objective response rate (rwORR), real-world disease control rate (rwDCR), real-world disease-free survival (rwDFS) and real-world duration of treatment (rwDOT). Results: As of August 30, 2025, a total of 236 patients were enrolled (perioperative, n = 23; first-line, n = 98; later-line, n = 115). The median age was 62.0 years (range, 33.0–83.0), with males and females accounting for 57.2% and 42.8%, respectively. Tumors were located in pancreatic head (25.4%), pancreatic body (11.4%), pancreatic tail (11.4%) and other pancreatic sites (51.7%). The most frequent metastasis site was the liver (39.0%). Baseline CA19-9 ≥37 U/mL was observed in 81.8% of patients. Any-grade TEAEs occurred in 36.9% of patients, with the most common being anemia (7.2%), nausea (6.8%), diarrhea (6.8%) and leukopenia (5.1%); grade ≥3 TEAEs occurred in 7.2% of patients. Median rwPFS was not reached in any cohort; the 6-month rwPFS rates were 83.3%, 66.4%, and 41.1% for the perioperative, first-line, and later-line cohorts, respectively. The median OS was not reached in any of the three cohorts. Conclusions: This real-world study revealed that the combination regimen based on irinotecan liposome (II) showed a manageable safety profile and encouraging effectiveness signals. Follow-up and patient enrollment are ongoing.