Rahner Nils, Höefler Gerald, Högenauer Christoph, Lackner Caroline, Steinke Verena, Sengteller Marlies, Friedl Waltraut, Aretz Stefan, Propping Peter, Mangold Elisabeth, Walldorf Constanze
Institute of Human Genetics, University of Bonn, Bonn, Germany.
Am J Med Genet A. 2008 May 15;146A(10):1314-9. doi: 10.1002/ajmg.a.32210.
Lynch syndrome (hereditary non-polyposis colorectal cancer, HNPCC) is an autosomal dominant condition caused by heterozygous germline mutations in the DNA mismatch repair (MMR) genes MLH1, MSH2, MSH6, or PMS2. Rare cases have been reported of an inherited bi-allelic deficiency of MMR genes, associated with multiple café-au-lait spots, early onset CNS tumors, hematological malignancies, and early onset gastrointestinal neoplasia. We report on a patient with vitiligo in segments of the integument who developed systemic lupus erythematosus (SLE) at the age of 16, and four synchronous colorectal cancers at age 17 years. Examination of the colorectal cancer tissue showed high microsatellite instability (MSI-H) and an exclusive loss of expression of the MSH6 protein. Immunohistochemical analysis of normal colon tissue also showed loss of MSH6, pointing to a bi-allelic MSH6 mutation. Sequencing of the MSH6 gene showed the two germline mutations; c.1806_1809delAAAG;p.Glu604LeufsX5 and c.3226C > T;p.Arg1076Cys. We confirmed that the two mutations are on two different alleles by allele-specific PCR. To our knowledge, neither parent is clinically affected. They did not wish to be tested for the mutations identified in their daughter. These data suggest that bi-allelic mutations of one of the MMR genes should be considered in patients who develop early-onset multiple HNPCC-associated tumors and autoimmune disorders, even in absence of either hematological malignancies or brain tumors.
林奇综合征(遗传性非息肉病性结直肠癌,HNPCC)是一种常染色体显性疾病,由DNA错配修复(MMR)基因MLH1、MSH2、MSH6或PMS2中的杂合种系突变引起。已有罕见病例报道MMR基因的遗传性双等位基因缺陷,与多个咖啡牛奶斑、早发性中枢神经系统肿瘤、血液系统恶性肿瘤和早发性胃肠道肿瘤相关。我们报告了一名患者,其皮肤部分出现白癜风,16岁时患上系统性红斑狼疮(SLE),17岁时患了4处同步性结直肠癌。对结直肠癌组织的检查显示高微卫星不稳定性(MSI-H)以及MSH6蛋白表达的完全缺失。对正常结肠组织的免疫组化分析也显示MSH6缺失,提示双等位基因MSH6突变。MSH6基因测序显示了两个种系突变:c.1806_1809delAAAG;p.Glu604LeufsX5和c.3226C>T;p.Arg1076Cys。我们通过等位基因特异性PCR证实这两个突变位于两个不同的等位基因上。据我们所知,其父母在临床上均未受影响。他们不希望对其女儿中发现的突变进行检测。这些数据表明,对于患有早发性多种HNPCC相关肿瘤和自身免疫性疾病的患者,即使没有血液系统恶性肿瘤或脑肿瘤,也应考虑MMR基因之一的双等位基因突变。