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[家族性低钙血症性高钙血症:一种新的突变]

[Familial hypocalciuric hypercalcemia: a new mutation].

作者信息

Ubetagoyena Arrieta M, Castaño González L, Pérez de Nanclares Leal G, Arruebarrena Lizarraga D, Imaz Murguiondo M, Areses Trapote R

机构信息

Sección de Nefrología Pediátrica, Osakidetza, Hospital Universitario Donostia, San Sebastián, Spain.

出版信息

An Pediatr (Barc). 2011 Jan;74(1):47-50. doi: 10.1016/j.anpedi.2010.10.001. Epub 2010 Dec 24.

Abstract

Familial hypocalciuric hypercalcemia (FHH) is a cause of hypercalcemia with autosomal dominant pattern of inheritance and high penetrance. In most of the cases it can be shown to be due to an inactivating mutation on the gene coding for the calcium-sensing receptor (CaSR). Heterozygous cases usually do not present symptoms and they are diagnosed as an incidental finding. We report three affected children with an inactivating heterozygous mutation, p.Phe789del, in exon 7 of the calcium-sensing receptor gene (CASR gene), situated in chromosome 3q21 (Ensembl ENSG00000036828), which results in elevated serum calcium, normal o high level of parathyroid hormone (PTH) and reduced urinary excretion with hypocalciuria. It is very important to determine the difference between FHH and primary hyperparathyroidism. Therefore, in a mild to moderate PTH-dependent hypercalcemia we must perform a family study and determine the urinary excretion of calcium. The presence of any other affected family member or reduced urinary calcium excretion is enough to suspect FHH, and this should be confirmed by the mutational analysis of the CASR gene, in order to establish the correct diagnosis, differentiated from primary hyperparathyroidism, to avoid unnecessary investigations or operations.

摘要

家族性低钙血症性高钙血症(FHH)是一种常染色体显性遗传且具有高外显率的高钙血症病因。在大多数情况下,可证实其是由编码钙敏感受体(CaSR)的基因发生失活突变所致。杂合子病例通常无明显症状,多为偶然发现而得以诊断。我们报告了3名受影响儿童,其钙敏感受体基因(CASR基因)位于3号染色体q21(Ensembl ENSG00000036828)的第7外显子存在失活杂合突变p.Phe789del,该突变导致血清钙升高、甲状旁腺激素(PTH)水平正常或升高以及尿钙排泄减少伴低钙尿症。明确FHH与原发性甲状旁腺功能亢进之间的差异非常重要。因此,对于轻度至中度依赖PTH的高钙血症,我们必须进行家族研究并测定尿钙排泄情况。存在任何其他受影响的家庭成员或尿钙排泄减少足以怀疑FHH,应通过对CASR基因进行突变分析来确诊,以明确正确诊断,与原发性甲状旁腺功能亢进相鉴别,避免不必要的检查或手术。

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