Who can safely discontinue lifelong follow-up among patients with sporadic pheochromocytoma and paraganglioma?

Abstract

Current guidelines recommend at least 10 years of follow-up for all pheochromocytoma and paraganglioma (PPGL) patients and lifelong monitoring for high-risk individuals. Nonetheless, data identifying patients who may not require routine lifelong follow-up are scarce. Among 999 patients with PPGL, 703 who were non-metastatic, non-hereditary, and had undergone complete resection were included. Variables that significantly differed between the recurrence (n = 50) and non-recurrence groups over 10 years (n = 83) were identified, and cutoff values were determined using receiver-operating characteristic curve analysis. These very low-risk criteria were validated in an internal cohort and an external dataset from the National Institutes of Health. The non-recurrence group was older and had smaller pheochromocytomas (PCCs) than the recurrence group, with cutoffs of 37 years and 5.7 cm, respectively. The non-recurrence group had a higher percentage of patients with pheochromocytoma of the adrenal gland scaled score (PASS) <4 or grading system for adrenal pheochromocytoma and paraganglioma (GAPP) score <3 (p = 0.027). Age >40 years (hazard ratio [HR] [95% confidence intervals] of 0.36 [0.17-0.76]), PCC size <6 cm (HR = 0.43 [0.19-0.98]), and PASS <4 or GAPP score <3 (HR = 0.37 [0.16-0.89]) were associated with lower recurrence risk. None of the patients meeting all these criteria in the internal (n = 114) and external (n = 13) validation sets experienced recurrence. This study suggests that routine lifelong follow-up may be unnecessary for patients with sporadic PCC aged >40 years, size <6 cm, and PASS <4 or GAPP score <3.