P-69 Efficacy and safety of surufatinib in intrahepatic cholangiocarcinoma: Preliminary results of a real-world study

When applied in the second-line treatment for biliary tract cancer patients (pts), surufatinib (a small-molecule inhibitor of VEGFR1-3, FGFR1 and CSF-1R) monotherapy has offered moderate clinical efficacy and has demonstrated favorable tolerability and safety profiles. This real-world study was to evaluate the efficacy and safety of surufatinib in the treatment for intrahepatic cholangiocarcinoma (ICC) pts. In particular, pts who received resection for ICC with positive margins would be eligible for our study. This population indicates a poor prognosis, for their tumor environment is complex, which is an obstacle to R0 resection. However, no proper treatment for this population has been recommended yet. This is an ongoing single-arm, multi-center, open-label real-world study conducted in China. The study would enroll 200 pts with unresectable or surgical resection with positive margins ICC. Pts would receive surufatinib with or without combination as adjuvant (namely pts with positive margin after resection), first- or further-line therapy, at a proper dose (200-300mg) judged by physicians once per day in 28-day cycles. The primary endpoint is progression-free survival (PFS). If available, tumor assessments were performed every 2 cycles ± 7 days according to RECIST version 1.1. By Feb 28, 2023, a total of 27 eligible pts had been enrolled, of whom 11 (40.74%) were female. The median age was 64 (range: 41-80). 11 (40.74%), 10 (37.04%), 5 (18.52%), and 1 (3.70%) received surufatinib as adjuvant, first-, second-, and third-line as monotherapy or combination, respectively. The median OS was not reached. With a median follow-up time of 12.3 (range: 2.30-16.66) mo, the global mPFS was 8.71 (95% CI: 6.64-14.8) mo. The mPFS of pts who received surufatinib as adjuvant, first-, and further-line were 8.13 (95% CI: 5.85-10.8), 9.66 (95% CI: 5.59-NA), and 8.71 (95% CI: 7.26-NA) mo, respectively. Among those with primary lesion unresected (n=16, for the rest 11 pts who had received tumor resection were not suitable for RECIST v1.1), 6 (37.50%) pts achieved PR, 9 (56.25%) pts achieved SD, and 1 (6.25%) suffered PD. The DCR was 93.75%. Globally, 4 pts died during the study, and no treatment-related death was reported. By then, Hypertension (n=2), fever (n=1), liver dysfunction (n=1), anorexia (n=1), and diarrhea (n=1) had been reported as AEs. More data of deaths and AEs are being collected. Surufatinib exhibited promising efficacy and manageable toxicity on pts with ICC in the real world.