Suppr超能文献

伊朗一名 TRPM6 新变异患者的肠道低镁血症:病例报告及文献复习。

Intestinal hypomagnesemia in an Iranian patient with a novel TRPM6 variant: a case report and review of the literature.

机构信息

Pediatric Nephrology Research Center, Research Institute for Children's Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran.

Student Research Committee, Alborz University of Medical Sciences, Karaj, Iran.

出版信息

CEN Case Rep. 2023 Nov;12(4):413-418. doi: 10.1007/s13730-023-00785-8. Epub 2023 Mar 26.

Abstract

TRPM6 is predominantly expressed in the kidney and colon and encodes a protein containing an ion channel domain and a protein kinase domain. It is crucial for magnesium homeostasis and plays important roles in epithelial magnesium transport and the active magnesium absorption. In this study, we present a 70-day-old Iranian female patient from consanguineous parents with hypomagnesemia and secondary hypocalcemia. She presented with seizures 19 days after birth and refractory watery non-bloody diarrhea. She consequently had failure to thrive. Other features included hypotonia, wide anterior fontanel, ventriculomegaly, and pseudotumor cerebri following administration of nalidixic acid. She had severe hypomagnesemia and hypocalcemia which were treated with magnesium and calcium supplementation. Despite initial unstable response to supplemental magnesium, she eventually improved and the diarrhea discontinued. The patient was discharged by magnesium and calcium therapy. At the last follow-up at age 2.5 years, the patient remained well without any recurrence or complication. Genetic testing by whole-exome sequencing revealed a novel homozygous frameshift insertion-deletion (indel) variant in exon 26 of the TRPM6 gene, c.3693-3699del GCAAGAG ins CTGCTGTTGACATCTGCT, p.L1231Ffs*36. Segregation analysis revealed the TRPM6 heterozygous variant in both parents. Patients with biallelic TRPM6 pathogenic variants typically exhibit hypomagnesemia with secondary hypocalcemia and present with neurologic manifestations including seizures. In some patients, this is also complicated by chronic diarrhea and failure to thrive. Long-term complications are rare and most of the patients show a good prognosis with supplemental magnesium therapy.

摘要

TRPM6 主要在肾脏和结肠中表达,编码一种含有离子通道结构域和蛋白激酶结构域的蛋白质。它对镁稳态至关重要,在肠上皮细胞镁转运和主动镁吸收中发挥重要作用。本研究报道了一对近亲伊朗夫妇的一个 70 天大的女婴,表现为低镁血症和继发性低钙血症。她出生后 19 天出现癫痫发作,随后出现难治性水样非血性腹泻和生长迟缓。其他特征包括使用萘啶酸后出现低张力、宽前囟、脑积水和假性脑瘤。她有严重的低镁血症和低钙血症,通过补充镁和钙进行治疗。尽管最初对补充镁的反应不稳定,但她最终得到改善,腹泻停止。患者出院时接受镁和钙治疗。在 2.5 岁时的最后一次随访中,患者情况良好,无任何复发或并发症。全外显子组测序的基因检测显示 TRPM6 基因外显子 26 中存在一个新的纯合移框插入-缺失(indel)变异,c.3693-3699del GCAAGAG ins CTGCTGTTGACATCTGCT,p.L1231Ffs*36。家系分析显示父母均携带 TRPM6 杂合变异。具有双等位基因 TRPM6 致病性变异的患者通常表现为低镁血症伴继发性低钙血症,并出现包括癫痫发作在内的神经表现。在一些患者中,还伴有慢性腹泻和生长迟缓。长期并发症罕见,大多数患者通过补充镁治疗预后良好。

相似文献

引用本文的文献

本文引用的文献

2
Long-term Clinical Follow-up of Patients with Familial Hypomagnesemia with Secondary Hypocalcemia.家族性低镁血症伴低钙血症患者的长期临床随访。
J Clin Res Pediatr Endocrinol. 2021 Aug 23;13(3):300-307. doi: 10.4274/jcrpe.galenos.2021.2020.0192. Epub 2021 Feb 10.
8
Aldosterone and Ion Channels.醛固酮与离子通道
Vitam Horm. 2019;109:105-131. doi: 10.1016/bs.vh.2018.10.004. Epub 2018 Dec 1.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验