Section of Surgical Endocrinology, Department of Surgical Oncology, University of Texas MD Anderson Cancer Center, Houston, Texas, USA.
Department of Endocrine Oncology, University Medical Center Utrecht, Utrecht, The Netherlands.
Endocr Relat Cancer. 2020 Aug;27(8):T9-T25. doi: 10.1530/ERC-19-0441.
The PanNET Working Group of the 16th International Multiple Endocrine Neoplasia Workshop (MEN2019) convened in Houston, TX, USA, 27-29 March 2019 to discuss key unmet clinical needs related to PanNET in the context of MEN1, with a special focus on non-functioning (nf)-PanNETs. The participants represented a broad range of medical scientists as well as representatives from patient organizations, pharmaceutical industry and research societies. In a case-based approach, participants addressed early detection, surveillance, prognostic factors and management of localized and advanced disease. For each topic, after a review of current evidence, key unmet clinical needs and future research directives to make meaningful progress for MEN1 patients with nf-PanNETs were identified. International multi-institutional collaboration is needed for adequately sized studies and validation of findings in independent datasets. Collaboration between basic, translational and clinical scientists is paramount to establishing a translational science approach. In addition, bringing clinicians, scientists and patients together improves the prioritization of research goals, assures a patient-centered approach and maximizes patient involvement. It was concluded that collaboration, research infrastructure, methodologic and reporting rigor are essential to any translational science effort. The highest priority for nf-PanNETs in MEN1 syndrome are (1) the development of a data and biospecimen collection architecture that is uniform across all MEN1 centers, (2) unified strategies for diagnosis and follow-up of incident and prevalent nf-PanNETs, (3) non-invasive detection of individual nf-PanNETs that have an increased risk of metastasis, (4) chemoprevention clinical trials driven by basic research studies and (5) therapeutic targets for advanced disease based on biologically plausible mechanisms.
第 16 届国际多发性内分泌肿瘤工作组（MEN2019）的 PanNET 工作组于 2019 年 3 月 27 日至 29 日在美国休斯顿召开会议，讨论 MEN1 背景下与 PanNET 相关的关键未满足的临床需求，特别关注无功能性（nf）-PanNET。与会者代表了广泛的医学科学家以及来自患者组织、制药行业和研究协会的代表。在基于案例的方法中，参与者讨论了早期检测、监测、局部和晚期疾病的预后因素和管理。对于每个主题，在回顾现有证据、关键未满足的临床需求和未来研究方向之后，确定了为 MEN1 伴 nf-PanNET 患者取得有意义进展的方向。需要开展国际多机构合作，以进行足够规模的研究并验证独立数据集的研究结果。基础、转化和临床科学家之间的合作对于建立转化科学方法至关重要。此外，将临床医生、科学家和患者聚集在一起可以改善研究目标的优先级，确保以患者为中心的方法并最大限度地提高患者的参与度。会议得出的结论是，合作、研究基础设施、方法学和报告严谨性对于任何转化科学努力都是必不可少的。MEN1 综合征中 nf-PanNET 的最高优先级是：(1) 开发一种数据和生物样本收集架构，使其在所有 MEN1 中心都保持一致；(2) 制定统一的诊断和随访方案，用于新诊断和已确诊的 nf-PanNET；(3) 开发用于检测具有转移风险的个体 nf-PanNET 的非侵入性方法；(4) 基于基础研究进行化学预防临床试验；(5) 基于合理的生物学机制，为晚期疾病确定治疗靶点。
