Targeted molecular medicine: advances in the treatment of metastatic phaeochromocytoma and paraganglioma
Mené sur 78 patients atteints d'un phéochromocytome ou d'un paragangliome de stade métastatique, cet essai multicentrique de phase II évalue l'efficacité, du point de vue de la survie sans progression à 12 mois, et la toxicité du sunitinib
WHO defines phaeochromocytomas and paragangliomas as neuroendocrine tumours arising from chromaffin cells of the adrenal medulla or extra-adrenal paraganglia, respectively. 1 Since catecholamine-secreting phaeochromocytomas and paragangliomas were first successfully resected 100 years ago, 2 , 3 major advances have occurred in the measurement of catecholamine and catecholamine metabolites, computed cross-sectional imaging, total body nuclear imaging, and laparoscopic approaches to tumour resection. In the early 2000s, many phaeochromocytoma and paraganglioma susceptibility genes were reported, underscoring the frequent hereditary nature of these rare endocrine neoplasms. 4 Concomitantly, metastatic phaeochromocytomas and paragangliomas have been increasingly recognised to be first detected up to 50 years postoperatively. 5 In 2017, WHO's classification of tumours of endocrine organs advised that all phaeochromocytomas and paragangliomas have malignant potential—a potential that is only confirmed when metastatic disease is documented. 1 In the 21st century, the primary diagnostic challenges for phaeochromocytoma and paraganglioma have become the early distinction between metastatic and non-metastatic forms and detecting pathogenic variants in the genes that predispose to the development of phaeochromocytomas and paragangliomas and distant metastases. On the management front, the main challenges remain the detection and treatment of metastatic phaeochromocytoma and paraganglioma, for which curative options are yet to be established.
The Lancet 2024