Current Treatment Options in Gastroenteropancreatic Neuroendocrine Carcinoma

Oncologist. 2019 Aug;24(8):1076-1088. doi: 10.1634/theoncologist.2018-0604. Epub 2019 Jan 11.


Poorly differentiated gastroenteropancreatic neuroendocrine carcinomas (GEPNECs) are a rare neoplasm with a bleak prognosis. Currently there are little prospective data available for optimal treatment. This review discusses the current available regimens and the future direction for the treatment of GEPNECs. Treatment plans for GEPNECs are often adapted from those devised for small cell lung cancer; however, differences in these malignancies exist, and GEPNECs require their own treatment paradigms. As such, current first-line treatment for GEPNECs is platinum-based chemotherapy with etoposide. Studies show that response rate and overall survival remain comparable between cisplatin and carboplatin versus etoposide and irinotecan; however, prognosis remains poor, and more efficacious therapy is needed to treat this malignancy. Additional first-line and second-line treatment options beyond platinum-based chemotherapy have also been investigated and may offer further treatment options, but again with suboptimal outcomes. Recent U.S. Food and Drug Administration approval of peptide receptor radionuclide therapy in low- and intermediate-grade neuroendocrine tumors may open the door for further research in its usefulness in GEPNECs. Additionally, the availability of checkpoint inhibitors lends promise to the treatment of GEPNECs. This review highlights the lack of large, prospective studies that focus on the treatment of GEPNECs. There is a need for randomized control trials to elucidate optimal treatment regimens specific to this malignancy. IMPLICATIONS FOR PRACTICE: There are limited data available for the treatment of poorly differentiated gastroenteropancreatic neuroendocrine carcinomas (GEPNECs) because of the rarity of this malignancy. Much of the treatment regimens used in practice today come from research in small cell lung cancer. Given the poor prognosis of GEPNECs, it is necessary to have treatment paradigms specific to this malignancy. The aim of this literature review is to summarize the available first- and second-line GEPNEC therapy, outline future treatments, and highlight the vast gap in the literature.


低分化胃肠胰神经内分泌癌 (GEPNEC) 是一种罕见的肿瘤,预后较差。目前,几乎没有关于可用的最佳治疗的前瞻性数据。本综述讨论了当前可用的方案和 GEPNEC 治疗的未来方向。GEPNEC 的治疗计划通常从针对小细胞肺癌的治疗计划中进行改进;然而,这些恶性肿瘤中存在差异,并且 GEPNEC 需要自己的治疗规范。因此,目前对 GEPNEC 的一线治疗是基于铂类化疗联合依托泊苷。研究表明,顺铂和卡铂与依托泊苷和伊立替康治疗的反应率和总生存率相当;然而,预后仍然很差,需要更有效的疗法来治疗这种恶性肿瘤。除了铂类化疗之外,其他一线和二线治疗方案也已经过研究,可能提供了进一步的治疗方案,但同样产生了不理想的结果。最近美国食品药品管理局批准在低级别和中级别神经内分泌肿瘤中使用肽受体放射性核素治疗,可能为进一步研究其在 GEPNEC 中的有用性打开了一扇大门。此外,免疫检查点抑制剂的可用性为 GEPNEC 的治疗带来了希望。本综述强调了缺乏研究 GEPNEC 治疗的大型前瞻性研究。需要随机对照试验来阐明针对该恶性肿瘤的最佳治疗方案。

实践意义:由于这种恶性肿瘤的罕见性,可用于治疗低分化胃肠胰神经内分泌癌 (GEPNEC) 的数据有限。如今在实践中使用的许多治疗方案来自于对小细胞肺癌的研究。鉴于 GEPNEC 的预后不良,有必要获得针对该恶性肿瘤的治疗规范。本文献综述的目的是总结现有的一线和二线 GEPNEC 疗法,概述未来的治疗方法,并强调文献中的巨大差距。

Keywords: Carcinoma; Current treatment; Neuroendocrine.

Publication types

  • Review

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Carboplatin / therapeutic use
  • Carcinoma, Neuroendocrine / mortality
  • Carcinoma, Neuroendocrine / pathology
  • Carcinoma, Neuroendocrine / therapy*
  • Cisplatin / therapeutic use
  • Clinical Trials as Topic
  • Etoposide / therapeutic use
  • Humans
  • Intestinal Neoplasms / mortality
  • Intestinal Neoplasms / pathology
  • Intestinal Neoplasms / therapy*
  • Irinotecan / therapeutic use
  • Neuroendocrine Tumors / mortality
  • Neuroendocrine Tumors / pathology
  • Neuroendocrine Tumors / therapy*
  • Pancreatic Neoplasms / mortality
  • Pancreatic Neoplasms / pathology
  • Pancreatic Neoplasms / therapy*
  • Prognosis
  • Progression-Free Survival
  • Radiopharmaceuticals / therapeutic use
  • Stomach Neoplasms / mortality
  • Stomach Neoplasms / pathology
  • Stomach Neoplasms / therapy*


  • Radiopharmaceuticals
  • Etoposide
  • Irinotecan
  • Carboplatin
  • Cisplatin

Supplementary concepts

  • Gastro-enteropancreatic neuroendocrine tumor