We conducted a multi-institutional retrospective analysis of patients with advanced high-grade NEN of the gastroenteropancreatic tract or of unknown origin seen between February 2016 and April 2023 who received treatment with frontline mFOLFIRINOX.
A total of 35 patients were included (G3 NETs: n=2; NECs: n=25; MiNENs: n=8; stage III: n=5; stage IV: n=30). The objective response rate was 77% (complete response: 3%; partial response: 74%). Median progression-free survival was 12 months (95% CI, 9.2-16.2 months) and median overall survival was 20.6 months (95% CI, 17.2-30.6 months). No significant differences in efficacy were seen according to primary site, histopathology, and Ki-67 proliferative index. All 5 patients with stage III disease who received mFOLFIRINOX obtained an objective response and underwent radical surgery or definitive radiotherapy with curative intent, with a recurrence rate of 40%. Grade 3 or 4 adverse events were observed in 43% of patients (mainly neutropenia and diarrhea). Females were at significantly increased risk of developing severe toxicities.
mFOLFIRINOX shows antitumor activity against high-grade NENs. Well-designed, prospective clinical trials are needed to assess the efficacy of mFOLFIRINOX in both the neoadjuvant and metastatic settings.
方法:我们对2016年2月至2023年4月期间接受一线mFOLFIRINOX治疗的胃肠胰道高级NEN患者或来源不明患者进行了多机构回顾性分析。
结果:共纳入35例患者(G3NETs:n=2;NECs:n=25;MiNENs:n=8;III期:n=5;IV期:n=30)。客观应答率为77%(完全应答:3%;部分应答:74%)。中位无进展生存期为12个月(95%CI,9.2-16.2个月),中位总生存期为20.6个月(95%CI,17.2-30.6个月)。根据原发部位,疗效无显著差异,组织病理学,和Ki-67增殖指数。所有5例接受mFOLFIRINOX治疗的III期患者均获得客观缓解,并接受根治性手术或根治性放疗,复发率为40%。在43%的患者中观察到3级或4级不良事件(主要是中性粒细胞减少和腹泻)。女性发生严重毒性的风险显着增加。
结论:mFOLFIRINOX对高级NENs具有抗肿瘤活性。精心设计,需要前瞻性临床试验来评估mFOLFIRINOX在新辅助治疗和转移治疗中的疗效.