MEN1-related pancreatic NETs: identification of unmet clinical needs and future directives.

Autor: 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.
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Zdroj: Endocrine-Related Cancer; Aug2020, Vol. 27 Issue 8, p9-25, 17p
Abstrakt: 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 c linical 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. [ABSTRACT FROM AUTHOR]
Databáze: Complementary Index