A real-world retrospective analysis to evaluate the efficacy and safety of surufatinib in the treatment of advanced neuroendocrine neoplasms in China

Surufatinib, an oral vascular immune kinase inhibitor targeting angiogenesis and immune escape, is approved in China for the treatment of unresectable locally advanced or metastatic neuroendocrine tumors (NETs). This real - world study aimed to evaluate its efficacy and safety in neuroendocrine neoplasms (NENs), including neuroendocrine carcinomas (NECs). From 2022 to 2024, 21 patients (12 with NET and 9 with NEC) at Nanjing Drum Tower Hospital received surufatinib - based therapy. The median follow - up period was 690 days. The primary endpoint, the disease control rate (DCR), was 81% (95% confidence interval [CI]: 58.1 - 94.6), and the objective response rate (ORR) was 14% (95% CI: 3.0 - 36.3). The ORR was 8.3% (95% CI 0.2 - 38.5) in NET and 44.4% (95% CI 13.7 - 78.8) in NEC, with no significant difference by Fisher’s exact test (P=0.119). The median progression - free survival (mPFS) was 9.7 months (95% CI: 2.5 - 15.9), while the median overall survival (mOS) had not been reached. For first - line treatment, the mPFS was 9.7 months (95% CI: 3.5 - 15.9) in NET patients and 15.1 months (95% CI: 3.8 - 26.5) in NEC patients; for second - line treatment, it was 6.7 months (95% CI: 0.9 - 12.4) in NET patients and 4.6 months (95% CI: 1.7 - 7.6) in NEC patients. Treatment - related adverse events (TRAEs) of grade ≥3 occurred in 28% of patients, and no treatment - related deaths were reported. Patients with TRAEs ≥1 (hazard ratio [HR] 0.349, P = 0.048) had longer PFS. This study supported the activity of surufatinib in advanced NENs; subgroup findings, including those for NEC, were exploratory and required confirmation.