Raygada Margarita, Pasini Barbara, Stratakis Constantine A
Adv Otorhinolaryngol. 2011;70:99-106. doi: 10.1159/000322484. Epub 2011 Feb 24.
Paragangliomas (PGL) and pheochromocytomas (PCC) are rare, usually benign tumors that originate from the neuroendocrine tissue along the paravertebral axis. Up to 35% of these tumors may be hereditary; they are associated with germline mutations in genes encoding subunits of the succinate dehydrogenase (SDH) enzyme complex in the context of the familial PGL syndromes, PGL1, 3 and 4 caused by mutations in the SDHD,SDHC and SDHB genes, respectively. Another familial PGL syndrome, PGL2, is caused by mutations in SDHAF2/SDH5, which encodes for a molecule that is an accessory to the function of the SDH enzyme and its SDHA subunit. Less frequently, mutations in the genes responsible for Von Hippel Lindau disease (VHL), multiple endocrine neoplasia type 2 (MEN2), and neurofibromatosis type 1 (NF1) are also found in patients with hereditary PGL and PCC. Recently mutations were found in the SDHA subunit in a limited number of patients with PGL and/or PCC. The SDHB, SDHC and SDHD gene mutations (but not SDHA) can also be found in patients with PGL and/or PCC and gastrointestinal stromal tumors (GISTs), also known as the Carney-Stratakis syndrome; SDHB mutations, in particular, may also predispose to thyroid and renal cancer, and possibly other tumors. A new gene was recently found to predispose to PGL and/or PCC when mutated is TMEM127. In this text, we provide an overview of the genetics of PGLs and related conditions with an emphasis on genetic risk assessment, prevention, and prognosis.
副神经节瘤(PGL)和嗜铬细胞瘤(PCC)是罕见的、通常为良性的肿瘤,起源于椎旁轴沿线的神经内分泌组织。这些肿瘤中高达35%可能是遗传性的;在家族性PGL综合征(分别由SDHD、SDHC和SDHB基因突变引起的PGL1、3和4)的背景下,它们与编码琥珀酸脱氢酶(SDH)酶复合体亚基的基因突变相关。另一种家族性PGL综合征PGL2是由SDHAF2/SDH5基因突变引起的,该基因编码一种对SDH酶及其SDHA亚基功能起辅助作用的分子。在遗传性PGL和PCC患者中,也较少发现与冯·希佩尔-林道病(VHL)、2型多发性内分泌肿瘤(MEN2)和1型神经纤维瘤病(NF1)相关的基因突变。最近,在少数PGL和/或PCC患者中发现了SDHA亚基的突变。在PGL和/或PCC以及胃肠道间质瘤(GIST)患者中也可发现SDHB、SDHC和SDHD基因突变(但不包括SDHA),这也被称为卡尼-斯特拉塔基斯综合征;特别是SDHB突变,也可能易患甲状腺癌和肾癌,以及可能的其他肿瘤。最近发现一个新基因TMEM127,其突变时易患PGL和/或PCC。在本文中,我们概述了PGL及其相关疾病的遗传学,重点是遗传风险评估、预防和预后。