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一例罕见的先天性尿崩症。

A Rare Case of Congenital Diabetes Insipidus.

机构信息

Nephrology Division, Department of Medicine, Jacobi Medical Center, Albert Einstein College of Medicine , Bronx, NY , USA.

University Medical Center of Princeton at Plainsboro, Rutgers Robert Wood Johnson Medical School , Plainsboro, NJ , USA.

出版信息

Front Med (Lausanne). 2015 Jul 7;2:43. doi: 10.3389/fmed.2015.00043. eCollection 2015.

DOI:10.3389/fmed.2015.00043
PMID:26217664
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC4493400/
Abstract

Congenital nephrogenic diabetes insipidus (NDI) is a conformation disease resulting from protein misfolding. Ninety percent of mutations result from the inactivating mutations of the arginine vasopressin receptor 2 (AVPR2) gene transmitted in an X-linked fashion, blocking the response to vasopressin, resulting in the inability to concentrate urine. Clinical features include polyuria, polydispsia, dehydration, and hypernatremia. They are generally more severely in affected males but present variably in females due to skewed inactivation of the X chromosome. We describe a case of a 40-year-old woman with a history of Type 2 diabetes mellitus, hyperlipidemia, and obesity, who presents with debilitating polyuria since the age of 5 with no clear diagnosis. Interestingly, her son was diagnosed with NDI. Genetic testing revealed that she was heterozygous for the Val88Met mutation in the AVPR2 gene while her son was hemizygous for the same. The patient has since been successfully treated with diuretics and a low solute diet. We highlight that although X-linked NDI patients are mostly males, it should be considered in symptomatic females to prevent delays in the diagnosis. Conformational diseases such as NDI are presently the subject of research using pharmacological chaperones to restore proper receptor membrane localization and function.

摘要

先天性肾性尿崩症(NDI)是一种由蛋白质错误折叠引起的构象疾病。90%的突变是由于 X 连锁方式失活的精氨酸加压素受体 2(AVPR2)基因突变引起的,阻断了对加压素的反应,导致无法浓缩尿液。临床特征包括多尿、多饮、脱水和高钠血症。它们在受影响的男性中通常更为严重,但由于 X 染色体的偏性失活,在女性中表现出不同的程度。我们描述了一例 40 岁女性的病例,她有 2 型糖尿病、高血脂和肥胖症病史,自 5 岁起出现严重的多尿,一直未明确诊断。有趣的是,她的儿子被诊断为 NDI。基因检测显示她是 AVPR2 基因中 Val88Met 突变的杂合子,而她的儿子是相同突变的半合子。此后,该患者成功地接受了利尿剂和低溶质饮食治疗。我们强调,尽管 X 连锁 NDI 患者主要是男性,但在有症状的女性中也应考虑到这种疾病,以避免延误诊断。构象疾病,如 NDI,目前是使用药理学伴侣来恢复适当的受体膜定位和功能的研究主题。

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A Novel Mutation in the Gene Causing Congenital Nephrogenic Diabetes Insipidus.导致先天性肾性尿崩症的基因中的一种新型突变。
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本文引用的文献

1
Characterization of AQP-2 gene mutation (R254Q) in a family with dominant nephrogenic DI.一个显性遗传性肾性尿崩症家系中AQP-2基因突变(R254Q)的特征分析
Pediatr Int. 2013 Feb;55(1):105-7. doi: 10.1111/j.1442-200X.2012.03614.x.
2
Hereditary nephrogenic diabetes insipidus in Japanese patients: analysis of 78 families and report of 22 new mutations in AVPR2 and AQP2.日本遗传性肾性尿崩症患者:AVPR2 和 AQP2 中 78 个家系的分析及 22 个新突变的报告。
Clin Exp Nephrol. 2013 Jun;17(3):338-44. doi: 10.1007/s10157-012-0726-z. Epub 2012 Nov 14.
3
AVPR2 variants and mutations in nephrogenic diabetes insipidus: review and missense mutation significance.肾性尿崩症中的精氨酸加压素受体2(AVPR2)变体与突变:综述及错义突变的意义
J Cell Physiol. 2008 Dec;217(3):605-17. doi: 10.1002/jcp.21552.
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X chromosome-inactivation patterns of 1,005 phenotypically unaffected females.1005名表型正常女性的X染色体失活模式
Am J Hum Genet. 2006 Sep;79(3):493-9. doi: 10.1086/507565. Epub 2006 Jul 27.
5
Pharmacologic chaperones as a potential treatment for X-linked nephrogenic diabetes insipidus.药物伴侣作为X连锁肾性尿崩症的一种潜在治疗方法。
J Am Soc Nephrol. 2006 Jan;17(1):232-43. doi: 10.1681/ASN.2005080854. Epub 2005 Nov 30.
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Molecular biology of hereditary diabetes insipidus.遗传性尿崩症的分子生物学
J Am Soc Nephrol. 2005 Oct;16(10):2836-46. doi: 10.1681/ASN.2005040371. Epub 2005 Aug 10.
7
Bladder function impairment in aquaporin-2 defective nephrogenic diabetes insipidus.水通道蛋白-2缺陷型肾性尿崩症中的膀胱功能损害
Nephrol Dial Transplant. 2004 Mar;19(3):608-13. doi: 10.1093/ndt/gfg574.
8
Treatment of congenital nephrogenic diabetes insipidus with hydrochlorothiazide and amiloride in an adult patient.成年患者先天性肾性尿崩症采用氢氯噻嗪和氨氯吡咪治疗。
Horm Res. 2004;61(2):63-7. doi: 10.1159/000075241. Epub 2003 Dec 1.
9
The ins and outs of aquaporin-2 trafficking.水通道蛋白-2转运的来龙去脉。
Am J Physiol Renal Physiol. 2003 May;284(5):F893-901. doi: 10.1152/ajprenal.00387.2002.
10
Three families with autosomal dominant nephrogenic diabetes insipidus caused by aquaporin-2 mutations in the C-terminus.三个因水通道蛋白-2 C末端突变导致常染色体显性遗传性肾源性尿崩症的家族。
Am J Hum Genet. 2001 Oct;69(4):738-48. doi: 10.1086/323643. Epub 2001 Aug 30.