Pheochromocytoma: Current Concepts and Emerging Evidence

Min Jeong ParkSouth Korea Speaker Pheochromocytoma: Current Concepts and Emerging EvidencePheochromocytoma and paraganglioma: Current Concepts and Emerging Evidence Min Jeong Park1 1Department of Internal Medicine, College of Medicine, Korea University, Seoul, South Korea Pheochromocytoma and paragangliomas (PPGLs) are rare neuroendocrine tumors. Over the past decade, rapid progress in molecular genetics, diagnostic and follow-up strategies, and systemic therapy has reshaped the landscape of these tumors. In this lecture, I will summarize current concepts and emerging evidence with particular attention to evolving clinical phenotype and long-term management challenges. Recent evidence suggests that a subset of PPGL patients exhibit autonomous hypercortisolemia in addition to catecholamine excess. In such cases, our group discovered higher rates of metabolic comorbidities – including diabetes mellitus and hypertension – compared with PPGL without cortisol excess. It infers the need for evaluation of pre and postoperative adrenal cortical function and metabolic comorbidities in patients with PPGL. PPGL are now recognized as among the most heritable human tumors, with 30-40% harboring germline mutation across more than 20 susceptibility genes. Molecular profiling had led to classification into three major clusters: Pseudohypoxia, kinase signaling, and Wnt-pathway – which informs biochemical phenotype, imaging patterns, and metastatic behavior. Multi-omics analyses identify ATRX/TERT alterations, copy-number burden, and immune microenvironmental features as potential prognostic biomarkers. Surgery remains curative for localized disease with alpha-blockade based perioperative optimization. For metastatic or unresectable disease, tyrosine kinase inhibitors, radionuclide therapies, and immune checkpoint inhibitors form an evolving systemic therapy for PPGL. Prediction of recurrence is also a major unmet domain. Current guideline recommend more than 10 years of surveillance for all resected PPGL and lifelong follow-up for high-risk patients; however, criteria for discontinuing surveillance in low-risk patients after 10 years remain undefined. Our group investigated features of the very-low risk group, which may discontinue follow-up after free of recurrence for 10 years of follow-up. This lecture will integrate recent advances in PPGL biology and management with practical consideration for metabolic assessment and long-term surveillance, including new data that may guide personalized decision-making for folllow-up cessation in selected low-risk patients.