The quality and reporting of neuroendocrine tumour (NET) clinical trials has previously been found to be heterogeneous impairing trial interpretability. We aimed to perform an updated review of the quality of phase II/III clinical trials in NET to assess if trial design and reporting have improved since 2011. We performed a PubMed search for phase II/III trials evaluating systemic anticancer therapies or liver-directed therapies published between 2011 and 2018. Data collected comprised administrative data, study population characteristics, endpoints, reporting and statistical design parameters, and results. Sixty studies were included (5218 patients): 50 phase II and 10 phase III trials. Study populations were heterogeneous: 52% of trials allowed tumours from various primary sites, 28% allowed both well- and poorly-differentiated tumour morphology or did not specify, and 57% did not report proliferative indices and/or tumour grade in ≥80% of the study population. Only 36% of trials mandated radiological disease progression on participant enrolment using a validated measure. Statistical design and primary endpoint were clearly defined in 67% and 88% of trials, respectively. Toxicity (88%), radiological response rate (85%) and progression-free survival/time to progression (82%) were well reported in a majority of trials, but health-related quality of life was included in the minority. Of the randomised trials (n = 11), study populations were more homogeneous and study design was more often clearly defined; however, only 45% mandated radiological progression at baseline as measured per Response Evaluation Criteria In Solid Tumours, and reporting of health-related quality of life (55%) remained suboptimal. The design and reporting of NET clinical trials, predominantly of single-arm phase II trials, remains suboptimal and has not considerably improved over time despite the growth in our knowledge of the biology and unique characteristics of NETs. Higher quality is seen in randomised trials, although certain design and reporting elements remain inadequate in some studies. We must prioritise the design and conduct of NET clinical trials to effectively inform future research and guide practice change.

中文翻译：

---

神经内分泌肿瘤临床试验的质量；我们从错误中吸取教训了吗？II期和III期临床试验的评估

先前已发现神经内分泌肿瘤 (NET) 临床试验的质量和报告存在异质性，损害了试验的可解释性。我们旨在对 NET 中 II/III 期临床试验的质量进行更新审查，以评估自 2011 年以来试验设计和报告是否有所改善。我们在 PubMed 中搜索了评估系统性抗癌疗法或肝脏导向的 II/III 期试验疗法发表于 2011 年至 2018 年。收集的数据包括管理数据、研究人群特征、终点、报告和统计设计参数以及结果。共纳入 60 项研究（5218 名患者）：50 项 II 期试验和 10 项 III 期试验。研究人群具有异质性：52% 的试验允许来自不同原发部位的肿瘤，28% 的患者允许高分化和低分化的肿瘤形态或未具体说明，57% 未报告≥80% 的研究人群中的增殖指数和/或肿瘤分级。只有 36% 的试验要求使用经过验证的措施在参与者登记时出现放射疾病进展。统计设计和主要终点分别在 67% 和 88% 的试验中得到明确定义。大多数试验都很好地报告了毒性（88%）、放射学反应率（85%）和无进展生存期/进展时间（82%），但少数试验包括与健康相关的生活质量。在随机试验（n = 11）中，研究人群更加同质，研究设计更明确；然而，根据实体瘤反应评估标准测量，基线时只有 45% 的放射学进展是强制性的，并且与健康相关的生活质量 (55%) 的报告仍然不理想。NET 临床试验的设计和报告，主要是单臂 II 期试验，尽管我们对 NET 的生物学和独特特征的了解有所增长，但仍不理想，并且随着时间的推移并没有显着改善。尽管某些研究中某些设计和报告要素仍然不足，但随机试验的质量更高。我们必须优先考虑 NET 临床试验的设计和实施，以有效地为未来的研究提供信息并指导实践的改变。主要是单臂 II 期试验，尽管我们对 NET 的生物学和独特特征的了解有所增长，但仍然不是最理想的，并且随着时间的推移并没有显着改善。尽管某些研究中某些设计和报告要素仍然不足，但随机试验的质量更高。我们必须优先考虑 NET 临床试验的设计和实施，以有效地为未来的研究提供信息并指导实践的改变。主要是单臂 II 期试验，尽管我们对 NET 的生物学和独特特征的了解有所增长，但仍然不是最理想的，并且随着时间的推移并没有显着改善。尽管某些研究中某些设计和报告要素仍然不足，但随机试验的质量更高。我们必须优先考虑 NET 临床试验的设计和实施，以有效地为未来的研究提供信息并指导实践的改变。