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Apatinib combined with CAPTEM (capecitabine plus temozolomide) has demonstrated preliminary activity in neuroendocrine tumours, yet head-to-head comparative efficacy data against standard-of-care sunitinib remain lacking. This study aims to evaluate the efficacy and safety of both regimens in advanced GEP-NETs using real-world data. This retrospective analysis examined patients with advanced GEP-NETs treated with either apatinib plus CAPTEM or sunitinib.Baseline characteristics (e.g. pathological grade, hepatic tumour burden) were balanced between groups using 1:1 propensity score matching (PSM). The primary endpoint was progression-free survival (PFS), with secondary endpoints including overall survival (OS), objective response rate (ORR), and safety. A total of 150 patients were included in the analysis, with 52 patients in each group after PSM. Following matching, the median PFS was significantly longer in the apatinib plus CAPTEM group compared with the sunitinib group (not reached NR 95% CI: 36.6–NR vs 17.9 months 95% CI: 8.0–18.0; hazard ratio HR = 0.17, 95% CI: 0.08–0.36; p < 0.001). The combination therapy group also demonstrated a significant OS benefit (52.0 months 95% CI: 48.1–58.0 vs 16.4 months 95% CI: 12.7–22.1; HR = 0.10, 95% CI: 0.05–0.22; p < 0.001). The ORR was numerically higher in the combination group than in the sunitinib group (65.4% vs 50.0%, p = 0.164). Regarding safety, the sunitinib group exhibited higher rates of hand-foot syndrome and diarrhoea, whilst the combination group did not experience any unexpected severe toxicities. In a PSM-matched cohort of advanced GEP-NETs, apatinib combined with CAPTEM was associated with improved PFS and OS, together with a numerically elevated ORR. Given the retrospective design and the potential for residual confounding, these findings should be considered hypothesis-generating and warrant prospective validation.
Tiegang Li (Fri,) studied this question.