The University of Texas MD Anderson Cancer Center, Houston, Texas 77030, USA.
Oncologist. 2013;18(5):525-32. doi: 10.1634/theoncologist.2012-0434. Epub 2013 Apr 24.
In the past 3 years, we have witnessed the completion of four randomized phase III studies in neuroendocrine tumors and the approval of two new drugs, everolimus and sunitinib, for the treatment of patients with well-differentiated pancreatic neuroendocrine tumors. These studies demonstrate a shift from case series and single-arm studies toward prospective, randomized controlled clinical trials and evidence-based therapy in the neuroendocrine tumor field. However, the clinical development of these agents also highlights the potential challenges awaiting other new drugs in this area. Herein, we discuss the strengths and weaknesses of the most recent phase II and phase III neuroendocrine tumor studies and discuss how limitations inherent in current trial design can lead to potential pitfalls. We also discuss how trial design can be improved, with the hope of increasing the number of drugs successfully developed to treat patients with neuroendocrine tumors.
在过去的 3 年中，我们见证了四项神经内分泌肿瘤的随机 III 期研究的完成，以及两种新药依维莫司和舒尼替尼被批准用于治疗分化良好的胰腺神经内分泌肿瘤患者。这些研究表明，神经内分泌肿瘤领域已经从病例系列和单臂研究转向了前瞻性、随机对照临床试验和基于证据的治疗。然而，这些药物的临床开发也凸显了该领域其他新药可能面临的挑战。在此，我们讨论了最近的 II 期和 III 期神经内分泌肿瘤研究的优缺点，并讨论了当前试验设计中固有的局限性如何导致潜在的陷阱。我们还讨论了如何改进试验设计，希望能增加成功开发用于治疗神经内分泌肿瘤患者的药物数量。
