Department of Pathology, Molecular, and Cell-Based Medicine, Icahn School of Medicine at Mount Sinai, New York, NY, 10029, USA.
Department of Pathology and Laboratory Medicine, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Acta Neuropathol Commun. 2023 May 3;11(1):73. doi: 10.1186/s40478-023-01573-2.
Molecular characterization of gliomas has uncovered genomic signatures with significant impact on tumor diagnosis and prognostication. CDKN2A is a tumor suppressor gene involved in cell cycle control. Homozygous deletion of the CDKN2A/B locus has been implicated in both gliomagenesis and tumor progression through dysregulated cell proliferation. In histologically lower grade gliomas, CDKN2A homozygous deletion is associated with more aggressive clinical course and is a molecular marker of grade 4 status in the 2021 WHO diagnostic system. Despite its prognostic utility, molecular analysis for CDKN2A deletion remains time consuming, expensive, and is not widely available. This study assessed whether semi-quantitative immunohistochemistry for expression of p16, the protein product of CDKN2A, can serve as a sensitive and a specific marker for CDKN2A homozygous deletion in gliomas. P16 expression was quantified by immunohistochemistry in 100 gliomas, representing both IDH-wildtype and IDH-mutant tumors of all grades, using two independent pathologists' scores and QuPath digital pathology analysis. Molecular CDKN2A status was determined using next-generation DNA sequencing, with homozygous CDKN2A deletion detected in 48% of the tumor cohort. Classifying CDKN2A status based on p16 tumor cell expression (0-100%) demonstrated robust performance over a wide range of thresholds, with receiver operating characteristic curve area of 0.993 and 0.997 (blinded and unblinded pathologist p16 scores, respectively) and 0.969 (QuPath p16 score). Importantly, in tumors with pathologist-scored p16 equal to or less than 5%, the specificity for predicting CDKN2A homozygous deletion was 100%; and in tumors with p16 greater than 20%, specificity for excluding CDKN2A homozygous deletion was also 100%. Conversely, tumors with p16 scores of 6-20% represented gray zone with imperfect correlation to CDKN2A status. The findings indicate that p16 immunohistochemistry is a reliable surrogate marker of CDKN2A homozygous deletion in gliomas, with recommended p16 cutoff scores of ≤ 5% for confirming and > 20% for excluding biallelic CDKN2A loss.
胶质母细胞瘤的分子特征揭示了对肿瘤诊断和预后具有重要影响的基因组特征。CDKN2A 是一个参与细胞周期控制的肿瘤抑制基因。CDKN2A/B 基因座的纯合缺失与胶质瘤发生和肿瘤进展有关,通过细胞增殖失调。在组织学上低级别胶质瘤中,CDKN2A 纯合缺失与更具侵袭性的临床病程相关,并且是 2021 年 WHO 诊断系统中 4 级状态的分子标志物。尽管其具有预后效用,但 CDKN2A 缺失的分子分析仍然耗时、昂贵,并且不是广泛可用。本研究评估了 p16(CDKN2A 的蛋白产物)的半定量免疫组织化学表达是否可以作为胶质母细胞瘤中 CDKN2A 纯合缺失的敏感和特异性标志物。使用两位独立病理学家的评分和 QuPath 数字病理学分析,在 100 例胶质瘤中通过免疫组织化学对 p16 表达进行了定量,这些胶质瘤代表了所有分级的 IDH 野生型和 IDH 突变型肿瘤。使用下一代 DNA 测序确定了 CDKN2A 的分子状态,在肿瘤队列中检测到 48%的肿瘤存在 CDKN2A 纯合缺失。基于 p16 肿瘤细胞表达(0-100%)对 CDKN2A 状态进行分类,在广泛的阈值范围内表现出稳健的性能,盲法和非盲法病理学家 p16 评分的接收者操作特征曲线面积分别为 0.993 和 0.997,以及 QuPath p16 评分 0.969。重要的是,在病理学家评分的 p16 等于或小于 5%的肿瘤中,预测 CDKN2A 纯合缺失的特异性为 100%;而在 p16 大于 20%的肿瘤中,排除 CDKN2A 纯合缺失的特异性也为 100%。相反,p16 评分在 6-20%的肿瘤代表了与 CDKN2A 状态相关性不理想的灰色区域。研究结果表明,p16 免疫组化是胶质母细胞瘤中 CDKN2A 纯合缺失的可靠替代标志物,推荐的 p16 截断值为≤5%用于确认和>20%用于排除 CDKN2A 双等位基因缺失。