Tomás-Simó Patricia, Sierra-Rivera Antonio, Checa-Ros Ana, Rodríguez-López Raquel, Galán-Serrano Antonio, D'Marco Luis
Nephrology Department, Consorcio Hospital General Universitario de Valencia, Valencia, Spain.
Genetics Section, Clinical Analysis Service, Consorcio Hospital General Universitario de Valencia, Valencia, Spain.
Nephrology (Carlton). 2025 Sep;30(9):e70127. doi: 10.1111/nep.70127.
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.
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。