Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Department of Pathology, Massachusetts General Hospital, Boston, MA, USA.
Semin Diagn Pathol. 2024 Jan;41(1):8-19. doi: 10.1053/j.semdp.2023.09.001. Epub 2023 Sep 20.
Tuberous sclerosis complex (TSC) is an autosomal dominant genetic disease characterized by hamartomatous tumors involving multiple organs such as the brain, skin, heart, lung and kidney. TSC is caused by inactivating mutations in TSC1/TSC2, which encodes hamartin and tuberin, respectively, and forms a complex that regulates mechanistic target of rapamycin complex 1 (mTORC1), resulting in cell overgrowth and oncogenesis. Since a leading cause of morbidity and mortality in TSC relates to chronic kidney disease and the ability to preserve renal function, this review describes the important pathologic findings in TSC-associated renal neoplasms and their correlating sporadic counterparts. The most common renal tumor in TSC patients are AMLs, followed by a heterogeneous spectrum of renal epithelial tumors, which may provide clues to establishing a diagnosis of TSC.
结节性硬化症复合征(TSC)是一种常染色体显性遗传疾病,其特征为涉及脑、皮肤、心脏、肺和肾等多个器官的错构瘤。TSC 是由 TSC1/TSC2 的失活突变引起的,分别编码错构瘤蛋白和结节性硬化蛋白,形成一个复合物,调节雷帕霉素靶蛋白复合物 1(mTORC1),导致细胞过度生长和肿瘤发生。由于 TSC 患者发病率和死亡率的主要原因与慢性肾脏病和维持肾功能的能力有关,因此本综述描述了 TSC 相关肾肿瘤的重要病理发现及其相关散发性肿瘤。TSC 患者最常见的肾肿瘤是 AMLs,其次是一系列异质性的肾上皮肿瘤,这可能为建立 TSC 的诊断提供线索。