Abstract
Paragangliomas and pheochromytomas (PPGLs) exhibit variable malignancy, advanced/hormonally active/progressive need therapy. PRRT (Peptide Receptor Radionuclide Therapy) could be an option for these patients. To evaluate the effectiveness of PRRT (90Y DOTATATE), based on overall survival (OS) and progression-free survival (PFS), in patients with PPGLs, related to SDHx gene mutation, we conducted a prospective open-label, single-center, phase II study. Thirteen patients were observed, eight PGL1 and five PGL4, all with advanced, non-resectable tumors, and eight had metastases. All were treated with 90Y DOTATATE. Efficacy was based on OS and PFS, and radiological response was based on RECIST. Hormonal activity was evaluated using serum-fractionated free catecholamines. Eight subjects had a clinical response, three were stable, and two exhibited disease progression. Among four patients with hormonally-active PPGLs, three showed a reduction and one showed normalization. OS for all was 68.0 months; PFS was 35.0 months. OS in PGL4 = 25.0 vs. N.R. (not reached) in PGL1. PFS in PGL4 = 12.0 vs. N.R. in PGL1. A difference was seen in the OS and PFS in patients who did not respond clinically, compared to those who did, OS = 22.0 vs. N.R. PFS = 7.0 vs. N.R. A difference in the OS and PFS was noted in patients with liver and bone involvement compared to those without. PRRT is an effective therapy in selected population of patients with SDHx, in those with locally-advanced, non-resectable tumors. Furthermore, it is effective regardless of the secretory status.
Highlights
Paraganglioma (PGL) and pheochromocytoma (PCC), commonly called PPGLs, are rare, biologically and clinically heterogeneous neuroendocrine tumors
Since (TCA) cycle-related PPGLs show significantly high expression of somatostatin receptor (SSTR), the aim of this study was to evaluate the clinical effectiveness of peptide receptor radionuclide therapy (PRRT) using (90Y DOTA0, D-Phe1, Tyr3-octreotate), based on overall survival (OS) and progression-free survival (PFS) in 13 patients with PGL1 and PGL4 syndromes and unresectable or metastatic PPGLs treated in a single center
Overall 32 therapeutic sessions were performed in a group of 13 patients with succinate dehydrogenase enzyme complex (SDHx) mutations, with a mean cumulative administered activity of 8.3 GBq of 90Y per patient, mean 3.4 GBq per therapy session
Summary
Paraganglioma (PGL) and pheochromocytoma (PCC), commonly called PPGLs, are rare, biologically and clinically heterogeneous neuroendocrine tumors. Paragangliomas arise from the sympathetic/ parasympathetic chain ganglia. Pheochromocytomas arise from adrenal medulla chromaffin cells. The incidence of PPGL based on nearly 1500 patients who were diagnosed and histopathologically confirmed between 1995 and 2015 was 0.04–0.21 per 100,000 person-years [1]. PPGLs can be found within the parasympathetic and sympathetic autonomic nervous system from the skull base to pelvis. Sympathetic tumors often secrete catecholamines [2,3] and are usually associated with hypertension and other symptoms, typically including palpitations, sweating, pallor, headache, and anxiety, and life–threatening cardiac complications, such as hypertension crisis, different types of cardiomyopathies, life-threatening arrhythmias, end-organ damage, cardiogenic shock, etc. Sympathetic tumors often secrete catecholamines [2,3] and are usually associated with hypertension and other symptoms, typically including palpitations, sweating, pallor, headache, and anxiety, and life–threatening cardiac complications, such as hypertension crisis, different types of cardiomyopathies, life-threatening arrhythmias, end-organ damage, cardiogenic shock, etc. [3]
Sign-in/Register to view highlights & summary Sign-in/Register to access full text options 
Free) 
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a call
