Real-world effectiveness and safety of second- or third-line pegylated liposomal irinotecan plus 5-fluorouracil and folinic acid in pancreatic ductal adenocarcinoma in Spain

Treatment with pegylated nanoliposomal irinotecan (nal-IRI) plus 5-fluorouracil/leucovorin (folinic acid; 5-FU/LV) has demonstrated remarkable efficacy for metastatic pancreatic ductal adenocarcinoma (PDAC) in clinical trials. However, real-world data on the effectiveness of nal-IRI+5-FU/LV is heterogeneous and is lacking in Spain. To assess the effectiveness and safety of nal-IRI+5-FU/LV in real-life PDAC patients in Spain. A multicenter retrospective study was conducted. Patients aged ⩾18 years who had received at least one cycle of nal-IRI+5-FU/LV as second- or third-line therapy for PDAC were included. The primary endpoint was overall survival (OS) from nal-IRI+5-FU/LV treatment initiation and OS from the diagnosis of metastatic disease (metOS). Overall, 200 evaluable patients were included (⩾3 metastatic sites: 22%; liver/lung metastases: 71.5%/36.9%; and Eastern Cooperative Oncology Group 0-1: 87% at nal-IRI+5FU/LV treatment initiation). Patients received a median of four cycles of nal-IRI+5FU/LV for 2.8 months (range 1.4-7.2), and the treatment was received in the second line by 80% of the patients. The median OS was 7.2 months (6- and 12-month OS rates: 58.1% and 28.9%, respectively), with 27.2% of the patients achieving OS ⩾12 months. The median metOS was 17.5 months, with 30.2% of the patients experiencing metOS ⩾ 24 months. The median progression-free survival (PFS) was 3.7 months (6- and 12-month PFS rate: 37.6% and 15.3%, respectively). The disease control rate was 35.5%. The median CA 19-9 levels decreased by at least 50% in 28.2% of the cases during treatment. Overall, 36% of the patients experienced at least one grade 3-4 adverse event during treatment, the most common being diarrhea (42.6%) and asthenia (30.9%). This real-world study shows that treatment with nal-IRI+5-FU/LV for advanced or metastatic PDAC affords benefit in terms of survival, radiological and CA 19-9 response, and PFS comparable to that reported in the clinical trial setting with a manageable safety profile.