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黑斑息肉综合征:一种新的STK11基因变异的早期临床表现

Peutz-Jeghers syndrome: early clinical expression of a new STK11 gene variant.

作者信息

Brito Sara, Póvoas Marta, Dupont Juliette, Lopes Ana Isabel

机构信息

Gastroenterology Unit, Paediatric Department, Hospital Santa Maria, Lisbon, Portugal.

Genetics Service, Paediatric Department, University Hospital Santa Maria, Lisbon, Portugal.

出版信息

BMJ Case Rep. 2015 Oct 1;2015:bcr2015211345. doi: 10.1136/bcr-2015-211345.

Abstract

Genetic heterogeneity has been recognised in Peutz-Jeghers syndrome (PJS) (over 230 STK11 gene mutations reported). We report a rare PJS phenotype with early extensive gastrointestinal (GI) presentation and a new genetic variant. The case presented as haematochezia and mucocutaneous pigmentation (the patient was 3 years of age). Endoscopy showed several polyps throughout the stomach/colon (PJ-type hamartomas); the larger polyps were resected. Small bowel imaging detected multiple jejunum/ileum small polyps. During 8 years of follow-up of this asymptomatic patient, an increasing number of diffusely distributed polyps was observed and polypectomies were performed. Subsequently, the patient failed consultations; when the patient was 13 years of age, emergency surgery was required due to small bowel intussusception (ileal polyp). A STK11 gene study identified two missense variants in heterozygous (yet unknown significance but probably pathogenic): c.854T>A (exon 6) and c.446C>T* (exon 2) (*not previously reported). We report two STK11 gene variants (one not previously described) of yet undetermined causality in a paediatric patient presenting with extensive GI involvement at a very early age, with no family medical history. Structural and functional repercussion of the newly described variants should be further investigated.

摘要

黑斑息肉综合征(PJS)已被确认存在遗传异质性(已报道超过230种STK11基因突变)。我们报告了一例罕见的PJS表型,其具有早期广泛的胃肠道(GI)表现及一种新的基因变异。该病例表现为便血和黏膜皮肤色素沉着(患者3岁)。内镜检查显示胃/结肠有多个息肉(PJ型错构瘤);较大的息肉被切除。小肠成像检测到空肠/回肠有多个小息肉。在对该无症状患者进行8年的随访期间,观察到息肉数量不断增加且分布弥漫,并进行了息肉切除术。随后,患者失访;患者13岁时,因小肠套叠(回肠息肉)需要进行急诊手术。一项STK11基因研究发现了两个杂合的错义变异(意义未知但可能致病):c.854T>A(外显子6)和c.446C>T*(外显子2)(*此前未报道)。我们报告了一名儿科患者中两个因果关系尚未确定的STK11基因变异(其中一个此前未描述),该患者在很小的年龄就出现广泛的胃肠道受累,且无家族病史。新描述变异的结构和功能影响应进一步研究。

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本文引用的文献

1
Genetic and Clinical Analyses of Southern Chinese Children with Peutz-Jeghers Syndrome.
Genet Test Mol Biomarkers. 2015 Sep;19(9):528-31. doi: 10.1089/gtmb.2015.0109. Epub 2015 Jul 30.
3
Findings from the Peutz-Jeghers syndrome registry of uruguay.乌拉圭皮杰氏综合征登记处的研究结果。
PLoS One. 2013 Nov 19;8(11):e79639. doi: 10.1371/journal.pone.0079639. eCollection 2013.

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