학술논문
Initiating Pancreatic Neuroendocrine Tumor (pNET) Screening in Young MEN1 Patients: Results From the DutchMEN Study Group
Clinical Research Article
Clinical Research Article
Document Type
Clinical report
Author
Haneveld, Mirthe J. Klein; van Treijen, Mark J. C.; Pieterman, Carolina R. C.; Dekkers, Olaf M.; Ven, Annenienke van de; Herder, Wouter W. de; Zandee, Wouter T.; Drent, Madeleine L.; Bisschop, Peter H.; Havekes, Bas; Vriens, Menno R.; Stuart, Annemarie A. Verrijn; Valk, Gerlof D.; van Leeuwaarde, Rachel S.
Source
Journal of Clinical Endocrinology & Metabolism. December 2021, Vol. 106 Issue 12, p3515, 11 p.
Subject
Language
English
ISSN
0021-972X
Abstract
Multiple endocrine neoplasia type 1 (MEN1) is a rare tumor predisposition syndrome caused by an inactivating germline mutation in the MEN1 gene (1, 2). Its 3 main clinical manifestations include [...]
Context: Nonfunctioning pancreatic neuroendocrine tumors (NF-pNETs) are highly prevalent and constitute an important cause of mortality in patients with multiple endocrine neoplasia type 1 (MEN1). Still, the optimal age to initiate screening for pNETs is under debate. Objective: The aim of this work is to assess the age of occurrence of clinically relevant NF-pNETs in young MEN1 patients. Methods: Pancreatic imaging data of MEN1 patients were retrieved from the DutchMEN Study Group database. Interval-censored survival methods were used to describe age-related penetrance, compare survival curves, and develop a parametric model for estimating the risk of having clinically relevant NF-pNET at various ages. The primary objective was to assess age at occurrence of clinically relevant NF-pNET (size [greater than or equal to] 20 mm or rapid growth); secondary objectives were the age at occurrence of NF-pNET of any size and pNET-associated metastasized disease. Results: Five of 350 patients developed clinically relevant NF-pNETs before age 18 years, 2 of whom subsequently developed lymph node metastases. No differences in clinically relevant NF-pNET-free survival were found for sex, time frame, and type of MEN1 diagnosis or genotype. The estimated ages (median, 95% CI) at a 1%, 2.5%, and 5% risk of having developed a clinically relevant tumor are 9.5 (6.5-12.7), 13.5 (10.2-16.9), and 17.8 years (14.3-21.4), respectively. Conclusion: Analyses from this population-based cohort indicate that start of surveillance for NF-pNETs with pancreatic imaging at age 13 to 14 years is justified.The psychological and medical burden of screening at a young age should be considered. Key Words: multiple endocrine neoplasia type 1, pancreatic NET, age-related penetrance, surveillance
Context: Nonfunctioning pancreatic neuroendocrine tumors (NF-pNETs) are highly prevalent and constitute an important cause of mortality in patients with multiple endocrine neoplasia type 1 (MEN1). Still, the optimal age to initiate screening for pNETs is under debate. Objective: The aim of this work is to assess the age of occurrence of clinically relevant NF-pNETs in young MEN1 patients. Methods: Pancreatic imaging data of MEN1 patients were retrieved from the DutchMEN Study Group database. Interval-censored survival methods were used to describe age-related penetrance, compare survival curves, and develop a parametric model for estimating the risk of having clinically relevant NF-pNET at various ages. The primary objective was to assess age at occurrence of clinically relevant NF-pNET (size [greater than or equal to] 20 mm or rapid growth); secondary objectives were the age at occurrence of NF-pNET of any size and pNET-associated metastasized disease. Results: Five of 350 patients developed clinically relevant NF-pNETs before age 18 years, 2 of whom subsequently developed lymph node metastases. No differences in clinically relevant NF-pNET-free survival were found for sex, time frame, and type of MEN1 diagnosis or genotype. The estimated ages (median, 95% CI) at a 1%, 2.5%, and 5% risk of having developed a clinically relevant tumor are 9.5 (6.5-12.7), 13.5 (10.2-16.9), and 17.8 years (14.3-21.4), respectively. Conclusion: Analyses from this population-based cohort indicate that start of surveillance for NF-pNETs with pancreatic imaging at age 13 to 14 years is justified.The psychological and medical burden of screening at a young age should be considered. Key Words: multiple endocrine neoplasia type 1, pancreatic NET, age-related penetrance, surveillance