我们评估了5-氟尿嘧啶(5-FU)、阿霉素和链霉素(FAS)治疗分化良好的胰腺神经内分泌肿瘤(PanNETs)的结果及其对后续治疗(依维莫司或替莫唑胺)的影响。我们回顾了1992年至2013年在我们中心治疗的晚期PanNET患者。患者每28天注射5-FU (400 mg/m2)、链霉素(400 mg/m2) (IV, 1-5天)和阿霉素(40 mg/m2 IV，第1天)。采用RECIST 1.1版评估总有效率(ORR)。在243例符合条件的患者中，220例可评估ORR、无进展生存期(PFS)和毒性。大多数(90%)有转移性、无功能的PanNETs;14%既往接受过治疗。对FAS的ORR为41%(95%可信区间[CI]: 36-48%)。中位随访时间为61个月。中位无进展生存期为20个月(95% CI: 15-23个月); median overall survival (OS) was 63 (95% CI: 60-71) months. Cox regression analyses suggested improvement with first-line vs subsequent lines of FAS therapy. Main adverse events ≥ grade 3 were neutropenia (10%) and nausea/vomiting (5.5%). Dose reductions were required in 32% of patients. Post-FAS everolimus (n=108; 68% second line) had a median PFS of 10 (95% CI: 8-14) months. Post-FAS temozolomide (n=60; 53% > fourth line) had an ORR of 13% and median PFS of 5.2 (95% CI: 4-12) months. In this largest reported cohort of PanNETs treated with chemotherapy, FAS demonstrated activity without significant safety concerns. FAS did not appear to affect subsequent PFS with everolimus; this sequence is being evaluated prospectively. Responses were noted with subsequent temozolomide-based regimens although PFS was possibly limited by line of therapy.
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