Université Paris Cité, PARCC, INSERM, Paris, France.
Département de Médecine Génomique des Tumeurs et des Cancers, Assistance Publique-Hôpitaux de Paris, Hôpital Européen Georges Pompidou, Paris, France.
Endocr Relat Cancer. 2023 Mar 8;30(4). doi: 10.1530/ERC-22-0373. Print 2023 Apr 1.
Paragangliomas (PGL) of the adrenal (also known as pheochromocytomas) or extra-adrenal neural crest-derived cells are highly heritable tumors, usually driven by single pathogenic variants that occur mutually exclusively in genes involved in multiple cellular processes, including the response to hypoxia, MAPK/ERK signaling, and WNT signaling. The discovery of driver mutations has led to active clinical surveillance with outcome implications in familial PGL. The spectrum of mutations continues to grow and reveal unique mechanisms of tumorigenesis that inform tumor biology and provide the rationale for targeted therapy. Here we review recent progress in the genetics and molecular pathogenesis of PGLs and discuss new prospects for advancing research with new disease models and ongoing clinical trials presented at the recent International Symposium of Pheochromocytomas and Paragangliomas (ISP2022) held in October 2022 in Prague.
嗜铬细胞瘤(PGL)起源于肾上腺(也称为嗜铬细胞瘤)或肾上腺外神经嵴细胞,是一种高度遗传性肿瘤,通常由单一的致病性变异驱动,这些变异在涉及多个细胞过程的基因中相互排斥出现,包括对缺氧、MAPK/ERK 信号和 WNT 信号的反应。驱动突变的发现导致了对家族性 PGL 具有预后意义的主动临床监测。突变谱不断扩大,并揭示了肿瘤发生的独特机制,为肿瘤生物学提供了信息,并为靶向治疗提供了依据。在这里,我们回顾了 PGL 的遗传学和分子发病机制的最新进展,并讨论了新的疾病模型和正在进行的临床试验的新前景,这些都在 2022 年 10 月于布拉格举行的国际嗜铬细胞瘤和副神经节瘤研讨会(ISP2022)上提出。