The University of Texas Health Science Center at Houston, USA.
J Investig Med High Impact Case Rep. 2024 Jan-Dec;12:23247096241286370. doi: 10.1177/23247096241286370.
While renal cell carcinoma (RCC) is often linked to smoking, obesity, and hypertension, hereditary forms also account for about 3% of RCC cases. Notably, NCCN guidelines identify 7 major hereditary syndromes associated with an increased RCC risk. Inherited mutations in DNA repair genes, such as ATM, BRCA, and TP53, significantly increase the risk of various cancers. Biallelic pathogenic mutations in ATM cause Ataxia-Telangiectasia (A-T) syndrome, while heterozygous germline pathogenic ATM mutations, present in about 1% of the population, also elevate cancer risk. RCC has not traditionally been associated with germline pathogenic ATM mutations, only limited retrospective analyses have identified such mutations. This case report presents a 68-year-old woman with a germline pathogenic ATM mutation (c.8786+1 G>A) who developed high-risk clear cell RCC followed by an acquired somatic VHL mutation in RCC and a 3-cm serous cystadenoma, illustrating the double-hit phenomenon. Her brother, who shares the same germline pathogenic mutation, was diagnosed with pancreatic cancer and prostate cancer. This case highlights the potential use for enhanced screening protocols for RCC in patients who have germline pathogenic ATM mutations and the importance of research in targeted treatments for tumors driven by dual genetic mechanisms. Increased awareness and vigilant screening for RCC are crucial in managing hereditary cancer syndromes effectively.
虽然肾细胞癌 (RCC) 通常与吸烟、肥胖和高血压有关,但遗传性形式也占 RCC 病例的约 3%。值得注意的是,NCCN 指南确定了与 RCC 风险增加相关的 7 种主要遗传性综合征。DNA 修复基因(如 ATM、BRCA 和 TP53)的遗传突变显著增加了各种癌症的风险。ATM 中的双等位致病性突变导致共济失调毛细血管扩张症 (A-T) 综合征,而杂合性生殖系致病性 ATM 突变,约占人口的 1%,也会增加癌症风险。RCC 传统上与生殖系致病性 ATM 突变无关,只有有限的回顾性分析确定了这些突变。本病例报告介绍了一名 68 岁女性,携带生殖系致病性 ATM 突变 (c.8786+1 G>A),她患有高风险透明细胞 RCC,随后在 RCC 中发生获得性体细胞 VHL 突变和 3 厘米浆液性囊腺瘤,说明了双打击现象。她的兄弟也携带相同的生殖系致病性突变,被诊断患有胰腺癌和前列腺癌。该病例强调了在具有生殖系致病性 ATM 突变的患者中使用增强的 RCC 筛查方案的潜力,以及针对双基因机制驱动的肿瘤进行靶向治疗的重要性。提高对 RCC 的认识和警惕性筛查对于有效管理遗传性癌症综合征至关重要。