Laboratory of Pathology.
Urologic Oncology Branch.
Am J Surg Pathol. 2021 Jan;45(1):137-142. doi: 10.1097/PAS.0000000000001611.
Renal cell carcinoma (RCC) is a heterogenous disease composed of several different cancer types characterized by distinct histologies and genetic alterations, including mutation of the Krebs cycle enzyme genes for fumarate hydratase and succinate dehydrogenase (SDH). This report describes a patient with multifocal renal tumors that presented with a novel, biphasic histologic morphology with one component consisting of small cells growing in a diffuse pattern occasionally forming glandular and cystic structures, reminiscent of type 1 papillary RCC, and the other component having larger cells with abundant eosinophilic and clear cytoplasm and appearing in a solid pattern of growth. Genetic analysis of multiple tumors showed that all had a somatic mutation of the IDH2 gene that created the known pathogenic, gain-of-function p.R172M alteration that results in abnormal accumulation of the oncometabolite 2-hydroxyglutarate (2-HG). Analysis of multiple tumors demonstrated highly elevated levels of 2-HG and a CpG island methylator phenotype that is characteristic of 2-HG-related inhibition of the Ten-eleven translocation (TET) family of DNA demethylases. In combination with fumarate hydratase-deficient and succinate dehydrogenase-deficient RCCs that have increased levels of the fumarate and succinate oncometabolites, respectively, the mutation of isocitrate dehydrogenase 2 represents the third Krebs cycle enzyme alteration to be associated with oncometabolite-induced RCC tumorigenesis. This study associates the discovery of a new histologic presentation of RCC with the first report of an IDH2 gain-of-function mutation in RCC.
肾细胞癌 (RCC) 是一种异质性疾病,由几种不同的癌症类型组成,其特征为明显的组织学和遗传改变,包括 Krebs 循环酶基因富马酸水合酶和琥珀酸脱氢酶 (SDH) 的突变。本报告描述了一位患有多灶性肾肿瘤的患者,其具有新颖的双相组织学形态,其中一个成分由小细胞组成,呈弥漫性生长,偶尔形成腺泡和囊状结构,类似于 1 型乳头状 RCC,另一个成分由大细胞组成,具有丰富的嗜酸性和透明细胞质,呈实性生长模式。对多个肿瘤的基因分析表明,所有肿瘤均存在 IDH2 基因的体细胞突变,该突变产生了已知的致病、功能获得性 p.R172M 改变,导致致癌代谢物 2-羟基戊二酸 (2-HG) 的异常积累。对多个肿瘤的分析表明,2-HG 水平高度升高,并且存在 CpG 岛甲基化表型,这是 2-HG 相关的 Ten-eleven 易位 (TET) 家族 DNA 去甲基酶抑制的特征。与分别具有增加的富马酸和琥珀酸盐致癌代谢物水平的琥珀酸水合酶缺陷型和琥珀酸脱氢酶缺陷型 RCC 相结合,异柠檬酸脱氢酶 2 的突变代表与致癌代谢物诱导的 RCC 肿瘤发生相关的第三个 Krebs 循环酶改变。本研究将 RCC 的新组织学表现与 RCC 中首次报道的 IDH2 功能获得性突变相关联。