Megdanova-Chipeva Vera G, Lamarca Angela, Backen Alison, McNamara Mairéad G, Barriuso Jorge, Sergieva Sonia, Gocheva Lilia, Mansoor Was, Manoharan Prakash, Valle Juan W
Department of Medical Oncology, The Christie NHS Foundation Trust, Manchester M204BX, UK.
Department of Radiotherapy and Medical Oncology, University Hospital "Queen Yoanna" ISUL, 1000 Sofia, Bulgaria.
Cancers (Basel). 2020 Jul 21;12(7):1988. doi: 10.3390/cancers12071988.
Pancreatic neuroendocrine tumours (PanNETs) are rare diseases and a good example of how research is not only feasible, but also of crucial importance in the scenario of rare tumours. Many clinical trials have been performed over the past two decades expanding therapeutic options for patients with advanced PanNETs. Adequate management relies on optimal selection of treatment, which may be challenging for clinicians due to the fact that multiple options of therapy are currently available. A number of therapies already exist, which are supported by data from phase III studies, including somatostatin analogues and targeted therapies (sunitinib and everolimus). In addition, chemotherapy remains an option, with temozolomide and capecitabine being one of the most popular doublets to use. Peptide receptor radionuclide therapy was successfully implemented in patients with well-differentiated gastro-entero-pancreatic neuroendocrine tumours, but with certain questions waiting to be solved for the management of PanNETs. Finally, the role of immunotherapy is still poorly understood. In this review, the data supporting current systemic treatment options for locally advanced or metastatic PanNETs are summarized. Strategies for treatment selection in patients with PanNETs based on patient, disease, or drug characteristics is provided, as well as a summary of current evidence on prognostic and predictive biomarkers. Future perspectives are discussed, focusing on current and forthcoming challenges and unmet needs of patients with these rare tumours.
胰腺神经内分泌肿瘤（PanNETs）是罕见疾病，是一个很好的例子，说明研究不仅可行，而且在罕见肿瘤的情况下至关重要。在过去二十年中进行了许多临床试验，扩大了晚期PanNETs患者的治疗选择。充分的管理依赖于治疗的最佳选择，由于目前有多种治疗选择，这对临床医生来说可能具有挑战性。已经存在多种疗法，这些疗法得到了III期研究数据的支持，包括生长抑素类似物和靶向疗法（舒尼替尼和依维莫司）。此外，化疗仍然是一种选择，替莫唑胺和卡培他滨是最常用的联合用药之一。肽受体放射性核素疗法已成功应用于高分化胃肠胰神经内分泌肿瘤患者，但在PanNETs的管理方面仍有一些问题有待解决。最后，免疫疗法的作用仍知之甚少。在这篇综述中，总结了支持目前局部晚期或转移性PanNETs全身治疗选择的数据。提供了基于患者、疾病或药物特征的PanNETs患者治疗选择策略，以及目前关于预后和预测生物标志物的证据总结。讨论了未来展望，重点关注这些罕见肿瘤患者当前和即将面临的挑战以及未满足的需求。
