MEN1-related pancreatic NETs: identification of unmet clinical needs and future directives: CURRENT STATE-OF-THE-ART AND RESEARCH OPPORTUNITIES: MEN1-related pancreatic NETs: identification of unmet clinical needs and future directives
Pieterman, C R C; Sadowski, S M; Maxwell, J E; Katz, M H G; Lines, K E; Heaphy, C M; Tirosh, A; Blau, J E; Perrier, N D; Lewis, M A; Metzcar, J P; Halperin, D M; Thakker, R V; Valk, G D
(2020) Endocrine-Related Cancer, volume 27, issue 8, pp. T9 - T25
(Article)
Abstract
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
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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.
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Keywords: MEN1, multiple endocrine neoplasia type 1, pancreatic neuroendocrine tumor, conference proceeding, unmet clinical needs, research infrastructure, management, risk stratification, surveillance, treatment, Unmet clinical needs, Multiple endocrine neoplasia type 1, Management, Conference proceeding, Pancreatic neuroendocrine tumor, Treatment, Surveillance, Risk stratification, Research infrastructure, Endocrinology, Oncology, Cancer Research, Endocrinology, Diabetes and Metabolism, Journal Article
ISSN: 1351-0088
Publisher: Society for Endocrinology
Note: Funding Information: This paper is part of a thematic section on current knowledge and future research opportunities in hereditary endocrine tumours, as discussed at MEN2019: 16th 阀nternational Workshop on Multiple Endocrine Neoplasia, 27–29 March 2019, Houston, TX, USA. This meeting was sponsored by Funding Information: R V T is supported by the Wellcome Trust Senior 阀nvestigator Award (grant number 106995/Z/15/Z), National 阀nstitute for Health Research (N 阀HR) Oxford Biomedical Research Centre Programme and N 阀HR Senior 阀nvestigator Award (RVT) (grant number NF-S 阀-0514-10091). Publisher Copyright: © 2020 Society for Endocrinology Published by Bioscientifica Ltd. Printed in Great Britain. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.
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