一种新的ANO1基因变异与伪装成巨结肠疾病的肠道运动障碍综合征相关:一例报告。

Ahmed H Al Sharie, Balqis M Abu Mousa, Yazan O Al Zu'bi, Mohammad A Al Qudah, Saied A Jaradat, Ahmad Barakat, Eyad Altamimi
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引用次数: 0

摘要

ANO1 (ANO1)相关肠蠕动障碍综合征(OMIM: 620045)是一种极为罕见的疾病,医学文献中仅报道了2例。我们报告一个2个月大的男婴,因腹泻、呕吐和腹胀来到我们中心。常规检查没有明确的诊断。全外显子组测序显示一种新的纯合子无义ANO1致病变异(c.1273G>T),其蛋白突变为p.Glu425Ter,符合患者的表型。Sanger测序显示双亲中有相同的ANO1变异,呈杂合形式,证实了常染色体隐性遗传模式。患者经历了多次腹泻相关的代谢性酸中毒、脱水和严重的电解质失衡,需要重症监护病房监测。患者被保守管理,并在门诊定期随访。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

A Novel <i>ANO1</i> Gene Variant is Associated with Intestinal Dysmotility Syndrome Masquerading as Hirschsprung Disease: A Case Report.

A Novel ANO1 Gene Variant is Associated with Intestinal Dysmotility Syndrome Masquerading as Hirschsprung Disease: A Case Report.

Anoctamin 1 (ANO1)-related intestinal dysmotility syndrome (OMIM: 620045) is an extremely rare disorder with only 2 cases reported in the medical literature. We present the clinical scenario of a 2-month-old male infant that presented to our center with diarrhea, vomiting, and abdominal distension. Routine investigations did not yield a clear diagnosis. Whole-exome sequencing showed a novel homozygous nonsense ANO1 pathogenic variant (c.1273G>T) with a protein alternation of p.Glu425Ter that fits the patient's phenotype. Sanger sequencing revealed the same ANO1 variant in both parents in a heterozygous form confirming an autosomal recessive mode of inheritance. The patient experienced multiple bouts of diarrhea-related metabolic acidosis, dehydration, and severe electrolyte imbalances that required intensive care unit monitoring. The patient was managed conservatively and being followed regularly in an outpatient setting.

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