Department of Medical Oncology, Beaujon University Hospital (AP-HP, Paris 7 Diderot), Clichy, France.
Endocrinol Metab Clin North Am. 2010 Dec;39(4):811-26. doi: 10.1016/j.ecl.2010.09.006.
Neuroendocrine tumors (NETs) are rare malignancies that arise from endocrine cells located in various anatomic locations, with a dramatic increase in incidence during the last 30 years. Limited therapeutic options are currently available for patients with advanced well-differentiated NETs, including carcinoids and pancreatic NETs. Streptozotocin-based chemotherapy and somatostatin analogues are drugs that are currently used for the treatment of progressive metastatic NETs. Recently, sunitinib demonstrating efficacy in pancreatic islet cell carcinomas has opened a new avenue for the treatment of NETs, and further trials shall be considered in NET types such as carcinoids, poorly differentiated neuroendocrine carcinomas, and several other endocrine tumors that depend on vascular endothelial growth factor (VEGF)/VEGF receptor for angiogenesis. In addition, drugs with distinct mechanisms of action, such as mammalian target of rapamycin inhibitors, currently investigated in phase 3 trials, may also supply novel options to control tumor growth and metastasis. Although acknowledged as rare tumors, recent data demonstrated the feasibility of large randomized trials in this disease. Furthermore, data from large trials also showed the importance of selecting an appropriate patient population when designing randomized studies. This review focuses on novel therapeutic approaches in the treatment of well-differentiated NETs. Based on recent data, novel strategies may now be designed using those anticancer agents to optimize the current treatment of patients with NETs.
神经内分泌肿瘤（NET）是一种罕见的恶性肿瘤，起源于分布于各种解剖位置的内分泌细胞，在过去 30 年中发病率显著增加。目前，对于晚期分化良好的 NET 患者，包括类癌和胰腺 NET，治疗选择有限。基于链脲佐菌素的化疗和生长抑素类似物是目前用于治疗进展性转移性 NET 的药物。最近，在胰腺胰岛细胞瘤中显示出疗效的舒尼替尼为 NET 的治疗开辟了新途径，并且在依赖血管内皮生长因子（VEGF）/VEGF 受体进行血管生成的类癌、低分化神经内分泌癌和其他几种内分泌肿瘤等 NET 类型中，应考虑进一步试验。此外，目前正在进行 3 期试验的具有不同作用机制的药物，如哺乳动物雷帕霉素靶蛋白抑制剂，也可能为控制肿瘤生长和转移提供新的选择。尽管被认为是罕见肿瘤，但最近的数据证明了在这种疾病中进行大型随机试验的可行性。此外，大型试验的数据还表明，在设计随机研究时选择合适的患者人群非常重要。本文重点介绍治疗分化良好的 NET 的新治疗方法。基于最近的数据，现在可能会使用这些抗癌药物设计新的策略，以优化 NET 患者的当前治疗。
