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与吉特曼综合征相关的SLC12A3新型致病基因型:一例报告

Novel Pathogenic Genotype in SLC12A3 Associated to Gitelman Syndrome: A Case Report.

作者信息

Tomás-Simó Patricia, Sierra-Rivera Antonio, Checa-Ros Ana, Rodríguez-López Raquel, Galán-Serrano Antonio, D'Marco Luis

机构信息

Nephrology Department, Consorcio Hospital General Universitario de Valencia, Valencia, Spain.

Genetics Section, Clinical Analysis Service, Consorcio Hospital General Universitario de Valencia, Valencia, Spain.

出版信息

Nephrology (Carlton). 2025 Sep;30(9):e70127. doi: 10.1111/nep.70127.

DOI:10.1111/nep.70127
PMID:40944618
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12433184/
Abstract

Gitelman syndrome (GS) is considered one of the most common hereditary renal tubular disorders, characterised by hypokalemia, hypomagnesemia, hypocalciuria, and metabolic alkalosis. The primary cause of this disorder resides in the SLC12A3 gene, which encodes the NaCl cotransporter in the distal convoluted tubule, and for which more than 500 mutations associated with GS have been described. We present the case of a 51-year-old female referred for evaluation of recurrent hypokalemia and hypomagnesemia, with no clinical symptoms. The blood test also revealed hypocalciuria and metabolic alkalosis. Oral supplementation with potassium and magnesium was prescribed. A next-generation sequencing (NGS) test was performed on her and her child (no other relatives alive), who was also asymptomatic with no obvious electrolytic abnormalities. Two mutations confirmed as pathogenic were found in the SLC12A3 gene (NM_000339.3) of the mother, c.704C>G and c.704C>T, as well as a new heterozygous variant in trans not reported before, c.704C>A p.(Thr235Lys), and identified as a variant of uncertain significance (VUS). This new VUS (c.704C>A p) was also present in the child, increasing evidence of its potential pathogenicity. The new SLC12A3 gene variant could represent a pathogenic mutation associated with GS. The use of NGS-based panel is recommended to cover the large genotypic variability associated with this disease, in an attempt to identify novel SLC12A3 gene variants of potential pathogenicity.

摘要

吉特曼综合征(GS)被认为是最常见的遗传性肾小管疾病之一,其特征为低钾血症、低镁血症、低钙尿症和代谢性碱中毒。这种疾病的主要病因在于SLC12A3基因,该基因编码远端曲小管中的氯化钠共转运体,目前已描述了500多种与GS相关的突变。我们报告了一例51岁女性病例,该患者因反复出现低钾血症和低镁血症前来评估,无临床症状。血液检查还显示有低钙尿症和代谢性碱中毒。给予口服补钾和补镁治疗。对她和她的孩子(无其他在世亲属)进行了下一代测序(NGS)检测,孩子也无症状,无明显电解质异常。在母亲的SLC12A3基因(NM_000339.3)中发现了两个被确认为致病的突变,即c.704C>G和c.704C>T,以及一个之前未报道过的反式杂合新变异体c.704C>A p.(Thr235Lys),被鉴定为意义未明的变异体(VUS)。这个新的VUS(c.704C>A p)也存在于孩子体内,增加了其潜在致病性的证据。新的SLC12A3基因变异体可能代表一种与GS相关的致病突变。建议使用基于NGS的检测板来涵盖与该疾病相关的巨大基因变异,以试图识别具有潜在致病性的新型SLC12A3基因变异体。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6e2a/12433184/5111ec86caa9/NEP-30-0-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6e2a/12433184/5111ec86caa9/NEP-30-0-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6e2a/12433184/5111ec86caa9/NEP-30-0-g001.jpg

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本文引用的文献

1
Functional evaluation of novel compound heterozygous variants in SLC12A3 of Gitelman syndrome.吉特曼综合征SLC12A3基因新型复合杂合变异的功能评估
Orphanet J Rare Dis. 2025 Feb 11;20(1):66. doi: 10.1186/s13023-025-03577-8.
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An unusual cause of hypokalaemia.
BMJ. 2024 Feb 8;384:e077724. doi: 10.1136/bmj-2023-077724.
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Long-Read Sequencing Identifies Novel Pathogenic Intronic Variants in Gitelman Syndrome.长读测序鉴定出 Gitelman 综合征中的新型致病性内含子变异。
J Am Soc Nephrol. 2023 Feb 1;34(2):333-345. doi: 10.1681/ASN.2022050627. Epub 2022 Nov 9.
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Endocr Connect. 2022 Jan 27;11(1):e210262. doi: 10.1530/EC-21-0262.
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Delayed growth and puberty in Gitelman syndrome.
Med Clin (Barc). 2021 Dec 24;157(12):588-589. doi: 10.1016/j.medcli.2021.01.025. Epub 2021 May 24.
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Clinical and Genetic Features in 31 Serial Chinese Children With Gitelman Syndrome.31例中国吉特曼综合征患儿的临床及遗传学特征
Front Pediatr. 2021 Apr 29;9:544925. doi: 10.3389/fped.2021.544925. eCollection 2021.
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Ventricular arrhythmia and tachycardia-induced cardiomyopathy in Gitelman syndrome, hypokalaemia is not the only culpable.吉特曼综合征中的室性心律失常和心动过速性心肌病,低钾血症并非唯一罪魁祸首。
BMJ Case Rep. 2019 Dec 15;12(12):e232086. doi: 10.1136/bcr-2019-232086.
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Novel heterozygous missense mutation of gene in Gitelman syndrome: A case report.吉特曼综合征中基因的新型杂合错义突变:一例报告。
World J Clin Cases. 2019 Jun 26;7(12):1522-1528. doi: 10.12998/wjcc.v7.i12.1522.
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Gitelman syndrome: consensus and guidance from a Kidney Disease: Improving Global Outcomes (KDIGO) Controversies Conference.Gitelman 综合征:改善全球肾脏病预后组织(KDIGO)争议会议的共识和指导意见。
Kidney Int. 2017 Jan;91(1):24-33. doi: 10.1016/j.kint.2016.09.046.
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Future considerations based on the information from Barrter's and Gitelman's syndromes.基于巴特综合征和吉特林综合征信息的未来考量。
Curr Opin Nephrol Hypertens. 2017 Jan;26(1):9-13. doi: 10.1097/MNH.0000000000000285.