三A综合征——贲门失弛缓症的罕见遗传原因。

IF 0.6 Q4 GASTROENTEROLOGY & HEPATOLOGY
ACG Case Reports Journal Pub Date : 2025-04-25 eCollection Date: 2025-05-01 DOI:10.14309/crj.0000000000001686
Shivangini Duggal, Monica Botros, Emerald Zaw, Swati Mahapatra, Jesus Guzman, Keith Garrison, Richard Mccallum
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引用次数: 0

摘要

aaa综合征(TAS)是一种罕见的常染色体隐性遗传疾病。它的特点是缺氧、失弛缓和肾上腺功能不全。它是由AAAS基因突变引起的,该基因突变破坏了ALADIN蛋白(alacrima - achalasia-肾上腺功能不全神经障碍蛋白),该蛋白在核胞质转运和细胞应激反应中起重要作用。与上述病例不同的是,大多数TAS患者在儿童早期出现各种症状,包括眼睛干涩、吞咽困难、反复感染,以及低血压、低血糖和色素沉着等肾上腺危机迹象。诊断可以通过基因检测来实现,揭示AAAS基因的突变。TAS的管理主要侧重于分别处理每一种情况,如对泪漏开具人工泪液,对肾上腺功能不全开具糖皮质激素替代治疗,对失弛缓症进行气动扩张或手术干预。早期诊断对于改善生活质量和尽量减少与该综合征有关的发病率和死亡率至关重要。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Triple A Syndrome-A Rare Hereditary Cause of Achalasia.

Triple A syndrome (TAS) is a rare disorder inherited in an autosomal recessive manner. It is characterized by the triad of alacrimia, achalasia, and adrenal insufficiency. It is caused by mutations in the AAAS gene (achalasia-addisonianism-alacrima syndrome), which disrupts the protein ALADIN (Alacrima-Achalasia-Adrenal insufficiency Neurologic disorder protein), which plays an important role in nucleocytoplasmic transport and cellular stress response. Unlike the presented cases, most patients with TAS present in early childhood with various symptoms including dry eyes, difficulty swallowing, and recurrent infections in addition to signs of adrenal crisis such as hypotension, hypoglycemia, and hyperpigmentation. Diagnosis can be achieved by genetic testing, revealing the mutations in the AAAS gene. Management of TAS mainly focuses on addressing each condition separately such as prescribing artificial tears for alacrimia, glucocorticoid replacement therapy for adrenal insufficiency, and performing pneumatic dilation or surgical intervention for achalasia. Early diagnosis is crucial for improving quality of life and minimizing the morbidity and mortality linked to the syndrome.

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来源期刊
ACG Case Reports Journal
ACG Case Reports Journal GASTROENTEROLOGY & HEPATOLOGY-
自引率
14.30%
发文量
170
审稿时长
12 weeks
期刊介绍: ACG Case Reports Journal is a peer-reviewed, open-access publication that provides GI and hepatology fellows, private practice clinicians, and other healthcare providers an opportunity to share interesting case reports with their peers and with leaders in the field. ACG Case Reports Journal publishes case reports, images, videos and letters to the editor in all topics of gastroenterology and hepatology, including: Biliary Colon Endoscopy Esophagus Functional Bowel Disorders Inflammatory Bowel Disease Liver Nutrition and Obesity Pancreas Pathology Pediatric Small Bowel Stomach.
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