Wallander Karin, Thonberg Håkan, Nilsson Daniel, Tham Emma
Department of Molecular Medicine and Surgery, Karolinska Institutet, Stockholm, Sweden.
Department of Clinical Genetics, Karolinska University Hospital, Stockholm, Sweden.
Hered Cancer Clin Pract. 2021 Oct 28;19(1):46. doi: 10.1186/s13053-021-00203-z.
Multiple primary cancers, defined as three or more primary tumours, are rare, and there are few genetic studies concerning them. There is a need for increased knowledge on the heritability of multiple primary cancers and genotype-phenotype correlations. We have performed whole-genome/exome sequencing (WGS/WES) in ten individuals with three or more primary tumours, with no previous findings on standard clinical genetic investigations. In one individual with a clinical diagnosis of MEN1, a likely pathogenic cryptic splice site variant was detected in the MEN1 gene. The variant (c.654C > A) is synonymous but we showed in a cDNA analysis that it affects splicing and leads to a frameshift, with the theoretical new amino acid sequence p.(Gly219Glufs*13). In one individual with metachronous colorectal cancers, ovarian cancer, endometrial cancer and chronic lymphocytic leukaemia, we found a likely pathogenic variant in the MLH1 gene (c.27G > A), and two risk factor variants in the genes CHEK2 and HOXB13. The MLH1 variant is synonymous but has previously been shown to be associated to constitutional low-grade hypermethylation of the MLH1 promoter, and segregates with disease in families with colorectal and endometrial cancer. No pathogenic single nucleotide or structural variants were detected in the remaining eight individuals in the study. The pathogenic variants found by WGS/WES were in genes already sequenced by Sanger sequencing and WES in the clinic, without any findings. We conclude that, in individuals with an unequivocal clinical diagnosis of a specific hereditary cancer syndrome, where standard clinical testing failed to detect a causative variant, re-analysis may lead to a diagnosis.
多原发性癌症定义为三个或更多原发性肿瘤,较为罕见,且关于它们的遗传学研究很少。需要增加对多原发性癌症遗传性以及基因型 - 表型相关性的了解。我们对10名患有三个或更多原发性肿瘤的个体进行了全基因组/外显子组测序(WGS/WES),这些个体在标准临床基因检测中之前未发现异常。在一名临床诊断为MEN1的个体中,在MEN1基因中检测到一个可能致病的隐蔽剪接位点变异。该变异(c.654C>A)是同义变异,但我们在cDNA分析中表明它影响剪接并导致移码,理论上新的氨基酸序列为p.(Gly219Glufs*13)。在一名患有异时性结直肠癌、卵巢癌、子宫内膜癌和慢性淋巴细胞白血病的个体中,我们在MLH1基因中发现了一个可能致病的变异(c.27G>A),以及在CHEK2和HOXB13基因中的两个风险因素变异。MLH1变异是同义变异,但先前已证明与MLH1启动子的体质性低级别高甲基化相关,并在结直肠癌和子宫内膜癌家族中与疾病共分离。在该研究的其余8名个体中未检测到致病的单核苷酸或结构变异。通过WGS/WES发现的致病变异存在于临床中已通过桑格测序和WES进行测序但未发现任何异常的基因中。我们得出结论,对于临床明确诊断为特定遗传性癌症综合征但标准临床检测未能检测到致病变异的个体,重新分析可能会得出诊断结果。