伴有SMAD4嵌合变异的胃局限性青少年息肉病综合征1例,表现为缓慢进展的表型。

IF 0.8 Q4 GASTROENTEROLOGY & HEPATOLOGY
Hiroki Fukuya, Taisuke Sasaki, Eikichi Ihara, Daisuke Yoshimura, Ryota Okitsu, Akito Ohkubo, Yuki Matsushita, Hirofumi Hasuda, Yoshihisa Sakaguchi, Naohiko Harada
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引用次数: 0

摘要

青少年息肉病综合征(JPS)是一种罕见的常染色体显性遗传病,由母亲的致病性变异引起的十肢截瘫同源物4 (SMAD4)和骨形态发生蛋白受体1A型(BMPR1A)基因。它的特点是在胃肠道中出现多个错构瘤性息肉。我们报告一例73岁的日本男性散发性,胃局限性JPS携带SMAD4马赛克变体,其表现为缓慢进展的内窥镜病变。55岁时胃镜检查发现胃底有红肿粘膜。在接下来的18年中,患者发展为进行性胃息肉病,导致难治性贫血和低白蛋白血症。基因检测鉴定出一个SMAD4移码马赛克变异(c.1245_1248del (p.Asp415Glufs)),等位基因频率为23%。行全胃切除术并D1淋巴结切除术,证实JPS诊断,并确定为局部胃腺癌,无淋巴结转移。该病例突出了具有SMAD4马赛克变体的JPS的独特自然历史,这可能有助于缓慢进展的表型。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
A case of gastric-localized juvenile polyposis syndrome with SMAD4 mosaic variant presenting as a slowly progressive phenotype.

Juvenile polyposis syndrome (JPS) is a rare autosomal dominant disorder caused by pathogenic variants in the mothers against decapentaplegic homolog 4 (SMAD4) and bone morphogenetic protein receptor type 1A (BMPR1A) genes. It is characterized by the development of multiple hamartomatous polyps in the gastrointestinal tract. We report a case of a 73-year-old Japanese male with sporadic, gastric-localized JPS harboring a SMAD4 mosaic variant who presented with slowly progressive endoscopic lesions. Endoscopy at the age of 55 revealed erythematous and edematous mucosa in the gastric fundus. During the following 18 years, the patient developed progressive gastric polyposis, leading to refractory anemia and hypoalbuminemia. Genetic testing identified a SMAD4 frameshift mosaic variant (c.1245_1248del (p.Asp415Glufs)) with an allele frequency of 23%. A total gastrectomy with D1 lymphadenectomy was performed, confirming the JPS diagnosis and the identification of a localized gastric adenocarcinoma without lymph node metastasis. This case highlights the unique natural history of JPS with a SMAD4 mosaic variant, which potentially contributes to a slowly progressive phenotype.

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来源期刊
Clinical Journal of Gastroenterology
Clinical Journal of Gastroenterology GASTROENTEROLOGY & HEPATOLOGY-
CiteScore
2.00
自引率
0.00%
发文量
182
期刊介绍: The journal publishes Case Reports and Clinical Reviews on all aspects of the digestive tract, liver, biliary tract, and pancreas. Critical Case Reports that show originality or have educational implications for diagnosis and treatment are especially encouraged for submission. Personal reviews of clinical gastroenterology are also welcomed. The journal aims for quick publication of such critical Case Reports and Clinical Reviews.
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