锯齿状息肉病综合征患者和非锯齿状息肉病综合征患者的 RNF43 基因致病变异。

IF 1.8 4区 医学 Q3 GENETICS & HEREDITY
Heidi Hesselø Brinch, Anna Byrjalsen, Zuzana Lohse, Andreas Ørslev Rasmussen, John Gásdal Karstensen, Britta Schlott Kristiansen, Anne Marie Jelsig
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引用次数: 0

摘要

锯齿状息肉病综合征(SPS)的特征是结肠多发性和/或大面积锯齿状息肉,且罹患结直肠癌(CRC)的风险增加。其病因尚不清楚,但在一部分 SPS 患者中检测到了 RNF43 的单等位致病变体。然而,迄今为止,对携带 RNF43 致病变体 (PV) 的患者的渗透性和表型谱的描述还很少。我们介绍了来自四个无血缘关系家庭的八名患有或未患有锯齿状息肉的 RNF43 可能致病变体 (LPV) 患者,并将结果与现有文献进行了比较。这些患者因怀疑患有遗传性癌症而被转介到遗传咨询中心。作为常规遗传检查的一部分,他们接受了包括 RNF43 在内的定制 NGS 基因面板遗传检测。在四个家庭中检测到三个 LPV,一个多外显子缺失和两个无义变异。I 家系曾有过 CRC 和锯齿状息肉病史,而其他三个家系(II-IV)则没有 CRC 或锯齿状息肉病史。尽管这些家族中的一些人年龄相对较大,但对他们进行的结肠镜检查并未发现任何锯齿状息肉和/或 CRC。我们的研究结果表明,RNF43 相关疾病的渗透率比以前认为的要低得多,并提出了有关 RNF43 与疾病之间联系的问题。这些结果突显了 RNF43 阳性家族遗传咨询的复杂性,尤其是在无息肉病的家族中。要阐明 RNF43 在 SPS 和 CRC 风险中的作用,还需要进一步的研究。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Germline pathogenic variants in RNF43 in patients with and without serrated polyposis syndrome.

Serrated Polyposis Syndrome (SPS) is characterized by multiple and/or large serrated polyps in the colon and an increased risk of colorectal cancer (CRC). The etiology is largely unknown, but in a subset of patients with SPS, monoallelic pathogenic variants in RNF43 are detected. To date, however, the penetrance and phenotypic spectrum of patients carrying pathogenic variants (PV) in RNF43 are poorly described. We present eight patients both with and without serrated polyps from four unrelated families with likely pathogenic variants (LPV) in RNF43 and compare the results to current literature. The patients were referred to genetic counseling due to suspicion of hereditary cancer. They underwent genetic testing with custom NGS gene panels including RNF43 as part of a routine genetic work-up. Three LPVs, one multi-exon deletion and two nonsense variants, were detected in four families. Family I had a history of CRC and serrated polyps, but in the three other families (II‒IV) there was no history of CRC or serrated polyps. Colonoscopies in the probands of these families did not reveal any serrated polyps and/or CRC despite some of them being relatively old. Our findings suggest that the penetrance of RNF43-related disease is much lower than previously thought, and raise questions about the connection between RNF43 and disease. The results highlight the complexity of genetic counseling in RNF43 positive families- particularly in families without polyposis. Further research is needed to elucidate the role of RNF43 in the risk of SPS and CRC.

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来源期刊
Familial Cancer
Familial Cancer 医学-遗传学
CiteScore
4.10
自引率
4.50%
发文量
36
审稿时长
6-12 weeks
期刊介绍: In recent years clinical cancer genetics has become increasingly important. Several events, in particular the developments in DNA-based technology, have contributed to this evolution. Clinical cancer genetics has now matured to a medical discipline which is truly multidisciplinary in which clinical and molecular geneticists work together with clinical and medical oncologists as well as with psycho-social workers. Due to the multidisciplinary nature of clinical cancer genetics most papers are currently being published in a wide variety of journals on epidemiology, oncology and genetics. Familial Cancer provides a forum bringing these topics together focusing on the interests and needs of the clinician. The journal mainly concentrates on clinical cancer genetics. Most major areas in the field shall be included, such as epidemiology of familial cancer, molecular analysis and diagnosis, clinical expression, treatment and prevention, counselling and the health economics of familial cancer.
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