Journal of Genetic Syndromes & Gene Therapy
Open Access

Abstract

Combined Biological Therapy is Effective to Control all Neuroendocrine Tumor Manifestations in a Patient with MEN1 Syndrome

Francesca Marciello, Carolina Di Somma, Silvia Savastano, Luigi Camera, Marco Volante, Mauro Papotti, Maria Luisa Brandi, Leo J Hofland, Annamaria Colao and Antongiulio Faggiano

Multiple endocrine neoplasia type 1 (MEN1) is a hereditary syndrome predisposing to the development of many endocrine tumors. Mainly pituitary, parathyroids and pancreas are involved although a proliferative state interests all neuroendocrine system. MEN1 pancreatic neuroendocrine tumors (pNET) are multiples and can secrete different hormones resulting in specific endocrine syndrome. The therapeutic approach is based in surgery which usually is followed by tumor relapse or persistence unless to be highly aggressive. Biotherapy with somatostatin analogs (SSAs) and dopamine agonists could be of great benefit to manage these patients without altering their life quality.

We report a case of a 36-year-old MEN1 man affected with multicentric PNETs associate with insulinoma and Zollinger-Ellison syndrome, pituitary prolactinoma and primary hyperparathyroidism. Therapy with symptomatic agents (proton pump inhibitors, diazoxide) as well as biotherapy (lanreotide, cabergoline) was started. At six month follow-up, symptomatic agents were stopped and disease control was only based on lanreotide plus cabergoline. This combined biotherapy was able to control endocrine syndromes and tumor growth, resulting in the possibility to perform a safer and selective surgical intervention on pNETs. Somatostatin and dopamine receptor expression has been evaluated and related to clinical response to biotherapy.

Therapy with lanreotide and cabergoline was able to control multiple secreting tumors in a patient with MEN1, due to high expression of specific receptors. The combined use of somatostatin analogues and dopamine agonists is suggested in patients with MEN1.