新生儿盐耗损综合征:由 CYP11B2 的一个新剪接变体引起的醛固酮合成酶缺乏症。

IF 1.3 4区 医学 Q3 PEDIATRICS Archives De Pediatrie Pub Date : 2024-02-01 DOI:10.1016/j.arcped.2023.10.007
Rémy Gerard , Clément Sauvestre , Pascal Barat , Jérôme Harambat , Clément Janot , Delphine Mallet , Florence Roucher-Boulez , Lise Allard
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引用次数: 0

摘要

醛固酮合成酶缺乏症(ASD)是一种罕见的常染色体隐性遗传疾病,涉及孤立的醛固酮缺乏症,而其他肾上腺激素没有受到任何损害。这种疾病主要在新生儿期和婴儿期表现为盐耗损综合征,伴有呕吐和发育不良。由于 ASD 有可能危及生命,因此需要在适当的激素检查基础上进行仔细和早期诊断,以便采取适当的治疗措施:补液以及补充盐分和氟氢可的松。对 CYP11B2 基因的遗传分析在大多数情况下都能确诊 ASD。我们报告的病例中,一名新生儿具有典型的临床表现,同时伴有一些不常见的表型特征(多汗症、肝损伤)。此外,我们的患者还携带一种新的 CYP11B2 剪接变异,这是已报道的约 60 种致病或可能致病变异之外的又一种新变异。
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Neonatal salt wasting syndrome: Aldosterone synthase deficiency caused by a new splicing variant in CYP11B2

Aldosterone synthase deficiency (ASD) is a rare autosomal recessive disorder involving isolated aldosterone deficiency without any compromise of other adrenal hormones. This condition manifests mainly in the neonatal period and in infants as a salt wasting syndrome with vomiting and failure to thrive. Due to its potentially life-threatening effects, ASD requires a careful and early diagnosis based on appropriate hormonal investigations in order to initiate adequate management: rehydration as well as salt and fludrocortisone supplementation. Genetic analysis of the CYP11B2 gene will confirm ASD in most cases. We report the case of a newborn with a typical clinical presentation associated with some uncommon phenotypic features (hyperhidrosis, liver injury). Furthermore, our patient carries a new CYP11B2 splicing variant to be added to the approximately 60 pathogenic or likely pathogenic variants already reported.

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来源期刊
Archives De Pediatrie
Archives De Pediatrie 医学-小儿科
CiteScore
2.80
自引率
5.60%
发文量
106
审稿时长
24.1 weeks
期刊介绍: Archives de Pédiatrie publishes in English original Research papers, Review articles, Short communications, Practice guidelines, Editorials and Letters in all fields relevant to pediatrics. Eight issues of Archives de Pédiatrie are released annually, as well as supplementary and special editions to complete these regular issues. All manuscripts submitted to the journal are subjected to peer review by international experts, and must: Be written in excellent English, clear and easy to understand, precise and concise; Bring new, interesting, valid information - and improve clinical care or guide future research; Be solely the work of the author(s) stated; Not have been previously published elsewhere and not be under consideration by another journal; Be in accordance with the journal''s Guide for Authors'' instructions: manuscripts that fail to comply with these rules may be returned to the authors without being reviewed. Under no circumstances does the journal guarantee publication before the editorial board makes its final decision. Archives de Pédiatrie is the official publication of the French Society of Pediatrics.
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