Long‐term outcomes in patients with multiple endocrine neoplasia type 1 and pancreaticoduodenal neuroendocrine tumours

• Published in: Clinical endocrinology (Oxford) 2017-02, Vol.86 (2), p.199-206
• Main Authors: Donegan, D., Singh Ospina, N., Rodriguez‐Gutierrez, R., Al‐Hilli, Z., Thompson, G.B., Clarke, B.L., Young, W.F.
• Format: Article
• Language: English
• Subjects: Adult; Cohort Studies; Follow-Up Studies; Humans; Middle Aged; Multiple Endocrine Neoplasia Type 1 - complications; Multiple Endocrine Neoplasia Type 1 - mortality; Multiple Endocrine Neoplasia Type 1 - pathology; Neoplasm Metastasis; Neuroendocrine Tumors - complications; Neuroendocrine Tumors - mortality; Neuroendocrine Tumors - surgery; Pancreatic Neoplasms - chemistry; Pancreatic Neoplasms - mortality; Pancreatic Neoplasms - surgery; Retrospective Studies; Treatment Outcome; Young Adult
• ISSN: 0300-0664; 1365-2265
• DOI: 10.1111/cen.13264

Summary Background In patients with multiple endocrine neoplasia type 1 (MEN‐1), pancreaticoduodenal (PD) neuroendocrine tumours (NETs) are associated with early mortality, yet the best treatment strategy remains uncertain. Aim To assess patient important outcomes (mortality and metastasis) of PD‐NETs and predictors of outcomes in patients with MEN‐1. Methods Retrospective cohort of patients with MEN‐1 who attended the Mayo Clinic, Rochester, MN from 1997 to 2014. Results We identified 287 patients with MEN‐1; 199 (69%) patients had 217 PD‐NETs. Among those with a PD‐NETs, 129 (65%) had surgery of which 90 (70%) had their primary surgery performed at Mayo Clinic. The median postoperative follow‐up was 8 years during which 13 (14%) patients died. The mean (±standard deviation) age of death was 51 (±9) years. Tumour size, metastasis at surgery or tumour type were not predictive of mortality, but for every year older at surgery, the odds of metastasis increased by 6%. Surgery was not performed in 70 (35%) patients. Among those who were observed/medically managed without known metastatic disease, mean tumour growth was 0·02 cm/year (range, −0·13–0·4 cm/year). Four patients (7%) died at a median age of 77 (range, 51–89) years. Conclusion PD‐NETs are common in patients with MEN‐1 and are associated with early mortality even after surgical intervention. Active surveillance is a viable option in nonaggressive PD‐NETs, although definitive factors identifying such patients are lacking. Therefore, counselling regarding risks and benefits of current treatment options remains integral to the care of patients with MEN‐1.