Real-world efficacy and safety of surufatinib in advanced neuroendocrine neoplasm.

634 Background: Surufatinib is an oral tyrosine kinase inhibitor that targets VEGFR1/2/3, FGFR1, and CSF-1R, exerting anti-angiogenic and immune-modulating effects. Currently approved for neuroendocrine tumors, this study assesses its real-world efficacy and safety. Methods: This prospective study enrolled adults with histologically confirmed neuroendocrine neoplasms receiving either surufatinib monotherapy (300 mg) or surufatinib-based combination therapy (250 mg). Primary endpoint is PFS. Secondary endpoints included ORR, DCR, OS, and safety. Results: As of Aug 27, 2025, 58 NEN patients were enrolled and had at least one efficacy assessment. Median age was 53.5 years (range: 22–71), with 50.0% female. Median Ki-67 index was 8% (range: 1%–80%). Primary tumors originated predominantly from the pancreas (67.2%), with 69.0% presenting liver metastasis. Pathological subtypes comprised NET (91.4%) and NEC (8.6%), with PPGL (8.6%) and G3 NET (5.2%) observed. Median follow-up was 26.7 months (mo). NEN demonstrated mPFS of 20.0 mo, with NET showing comparable outcomes (20.0 mo), whereas NEC exhibited markedly inferior survival (3.7 mo). Among NET subtypes, p-NET achieved the longest mPFS (28.1 mo), followed by ep-NET and PPGL, both reaching 17.8 mo. Key efficacy and subgroup analysis are shown (Table). Among 7 NET patients who progressed on monotherapy and sequentially initiated surufatinib-based combination therapy, 3 cases progressed post-combination initiation, while 4 remain on therapy with durations of 38.5, 35.6, 14.9, and 6 months. PFS remains immature from treatment commencement. The 18-month PFS rate was 71.4% (95% CI: 44.7%-100.0%) in the sequential cohort versus 59.4% (95% CI: 43.9%-79.3%) in non-sequential controls. Common treatment-emergent adverse events (TEAEs) included hypertension (48.3%), proteinuria (36.2%), and diarrhea (24.1%). Conclusions: This real-world evidence study demonstrates that MDT-guided individualized therapy significantly enhances surufatinib efficacy, with improved PFS and DCR versus registry data (ORR consistent), while maintaining favorable safety. Notably, sequential surufatinib combination therapy after monotherapy progression yields substantial clinical benefits. Further analyses will optimize precision treatment strategies for advanced neuroendocrine tumors. Clinical trial information: ChiCTR2100049999 . NEN NET NEC p-NET ep-NET PPGL N=58 N=53 N=5 N=37 N=16 N=5 PR 8 8 0 5 3 0 SD 47 44 3 31 13 5 PD 3 1 2 1 0 0 ORR 13.8% 15.1% 0.0% 13.5% 18.8% 0.0% DCR 94.8% 98.1% 60.0% 97.3% 100.0% 100.0% mPFS (95%Cl), months 20.0 (14.3-30.5) 20.0 (15.3-30.5) 3.7 (1.9-NA) 28.1 (14.3-NA) 17.8 (13.7-21.7) 17.8 (8.6-NA)