Onder Gizem, Unal Busra, Ozdemir Ozkan, Amanvermez Ufuk, Elmas Merve Acıkel, Gokbayrak Merve, Ugurtas Cansu, Cine Naci, Kalay İrem, Ozbek Ugur, Ng Ozden Hatirnaz, Agaoglu Nihat Bugra
Department of Molecular Biology and Biochemistry, Institute of Health Sciences, Acibadem University, Istanbul, Turkey.
Rare Diseases and Orphan Drugs Application and Research Center (ACURARE), Acibadem University, Istanbul, Turkey.
Mol Genet Genomics. 2025 Apr 24;300(1):45. doi: 10.1007/s00438-025-02250-w.
Advances in high-throughput sequencing have increased the detection of TP53 variations, many of which occur at low allelic fractions. Such variants may arise due to clonal hematopoiesis (CHIP) or constitutional mosaicism, complicating their clinical classification and management. Since guidelines recommend Li-Fraumeni syndrome (LFS)-like management for individuals carrying TP53 variations, accurately determining the origin of low variant allelic fraction (VAF) variants is essential for risk assessment and clinical decision-making. This study evaluates TP53 VAF in patients with suspected hereditary cancer predisposition, tested via multigene panels and emphasizes the importance of conducting a detailed investigation before making clinical decisions in patients with low-VAF. In retrospectively analyzed 1,520 cases, we identified 17 actionable TP53 variations in 16 cases (1%). All cases were female (mean cancer onset age of 45.9 years) and classified as attenuated LFS. Eleven of the variants had an allelic fraction of ≤ 20%. Patients over 60 years showed significantly lower VAF than those under 40 (p = 0.03). The TP53 variant was detected in only one ancillary sample, and her tumor sample was monoallelic, confirming the germline origin. For an accurate classification and successful management of cases with TP53 variations, defining the origin of variants, especially for low VAF, is imperative.
高通量测序技术的进步提高了TP53变异的检测率,其中许多变异以低等位基因分数出现。这些变异可能源于克隆性造血(CHIP)或体细胞镶嵌现象,使其临床分类和管理变得复杂。由于指南建议对携带TP53变异的个体采用类李-弗劳梅尼综合征(LFS)的管理方法,准确确定低变异等位基因分数(VAF)变异的起源对于风险评估和临床决策至关重要。本研究评估了通过多基因检测板检测的疑似遗传性癌症易感性患者的TP53 VAF,并强调在对低VAF患者做出临床决策之前进行详细调查的重要性。在对1520例病例进行回顾性分析时,我们在16例(1%)病例中鉴定出17个可采取行动的TP53变异。所有病例均为女性(平均癌症发病年龄45.9岁),并被归类为轻型LFS。其中11个变异的等位基因分数≤20%。60岁以上的患者VAF显著低于40岁以下的患者(p = 0.03)。TP53变异仅在一个辅助样本中检测到,其肿瘤样本为单等位基因,证实了种系起源。为了对TP53变异病例进行准确分类和成功管理,确定变异的起源,尤其是低VAF变异的起源,是必不可少的。