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偶然发现的诊断:库欣综合征由分泌皮质醇的肾上腺腺瘤引起,作为1型多发性内分泌肿瘤的初始表现,病因是罕见的MEN1基因剪接位点突变。

Diagnosis by serendipity: Cushing syndrome attributable to cortisol-producing adrenal adenoma as the initial manifestation of multiple endocrine neoplasia type 1 due to a rare splicing site MEN1 gene mutation.

作者信息

Alzahrani Ali S, Al-Khaldi Nojoud, Shi Yufei, Al-Rijjal Roua A, Zou Minjing, Baitei Essa Y, Amin Tarek

机构信息

Department of Medicine, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia.

出版信息

Endocr Pract. 2008 Jul-Aug;14(5):595-602. doi: 10.4158/EP.14.5.595.

Abstract

OBJECTIVE

To report a case that highlights the potential for Cushing syndrome to be the first manifestation of multiple endocrine neoplasia type 1 (MEN 1) syndrome and to describe the rare underlying genetic mutation and the heterogeneous manifestations of the syndrome within the same family.

METHODS

We present a case report including biochemical and radiologic findings, review family data, and discuss the results of genetic analyses.

RESULTS

A 16-year-old girl who was not known to have any medical illness and had no known family history of MEN 1 syndrome presented with Cushing syndrome attributable to a cortisol-producing adrenal adenoma. During her evaluation, she was found to have primary hyperparathyroidism and a pituitary microprolactinoma. These findings raised the possibility of MEN 1 syndrome. She did not have clinical, biochemical, or radiologic evidence of islet cell pancreatic tumors. Family screening showed that her father had evidence of primary hyperparathyroidism, mild hyperprolactinemia, normal findings on magnetic resonance imaging of the pituitary, and a 1.2-cm nodule in the tail of the pancreas in conjunction with slight elevation of serum insulin and normal gastrin levels. The patient's 5 siblings had evidence of primary hyperparathyroidism, and 2 of them also had mild hyperprolactinemia. Genetic screening confirmed the presence of a MEN1 gene missense G to A mutation in the patient, her father, and her siblings at the splicing site of intron 6 (IVS6+1G>A). This mutation leads to frameshift and truncation of the MEN1 gene.

CONCLUSION

In MEN 1, Cushing syndrome is an extremely rare and usually late manifestation. Most cases are due to corticotropin-producing pituitary adenomas. Although Cushing syndrome generally develops years after the more typical manifestations of MEN 1 appear, it may be the primary manifestation of MEN 1 syndrome. There is considerable heterogeneity in the manifestations of MEN 1, even within a family having the same genetic mutation.

摘要

目的

报告1例以库欣综合征作为多发性内分泌腺瘤1型(MEN 1)综合征首发表现的病例,并描述该综合征罕见的潜在基因突变以及同一家族中该综合征的异质性表现。

方法

我们呈现1例病例报告,包括生化和影像学检查结果,回顾家族资料,并讨论基因分析结果。

结果

一名16岁女孩,此前无任何疾病史,也无已知的MEN 1综合征家族史,因分泌皮质醇的肾上腺腺瘤出现库欣综合征。在评估过程中,发现她患有原发性甲状旁腺功能亢进和垂体微泌乳素瘤。这些发现提示了MEN 1综合征的可能性。她没有胰岛细胞胰腺肿瘤的临床、生化或影像学证据。家族筛查显示,她的父亲有原发性甲状旁腺功能亢进的证据、轻度高泌乳素血症、垂体磁共振成像结果正常、胰腺尾部有一个1.2 cm的结节,同时血清胰岛素轻度升高,胃泌素水平正常。患者的5个兄弟姐妹有原发性甲状旁腺功能亢进的证据,其中2人也有轻度高泌乳素血症。基因筛查证实患者、其父亲和兄弟姐妹在第6内含子剪接位点(IVS6+1G>A)存在MEN1基因错义G到A突变。该突变导致MEN1基因移码和截短。

结论

在MEN 1中,库欣综合征是一种极其罕见且通常为晚期的表现。大多数病例是由于分泌促肾上腺皮质激素的垂体腺瘤所致。虽然库欣综合征通常在MEN 1更典型的表现出现数年之后才发生,但它可能是MEN 1综合征的首发表现。MEN 1的表现存在相当大的异质性,即使在具有相同基因突变的家族中也是如此。

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