一例罕见的LMNA变异引起的Malouf综合征患者的类早衰特征:一个病例报告和文献回顾。

IF 1.6 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM
Aslihan Pekmezci, Aydeniz Aydin Gumus, Ozge Polat Korkmaz
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引用次数: 0

摘要

层板病是一种罕见的遗传性疾病,影响各种器官和组织,包括皮肤、肌肉、脂肪组织、骨骼和心血管系统。LMNA基因是层压板病最常见的致病基因,其变异可导致多种临床表型,如类早衰综合征、脂肪营养不良症、肌肉营养不良症和心肌病。本文报告一例年轻女性患者,其表现为前驱糖尿病、继发性闭经和继发性骨质疏松症。一位28岁的女性以闭经和骨密度下降为主诉来到我们的诊所。她表现出明显的面部畸形和不发达的第二性征。实验室调查显示促性腺功能亢进、性腺功能减退、糖尿病前期和高脂血症。超声心动图显示明显的二尖瓣环形钙化。遗传分析显示在LMNA基因的外显子1上有一个新的变异。这个病例揭示了一种新的椎板病与Malouf综合征的临床特征重叠,同时也表现出额外的早衰性特征,代表了一种独特的椎板病。此外,与先前报道的这种基因型病例不同,它不对应于典型的与LMNA变异相关的类早衰综合征。此外,本病例报告附有相关文献的回顾。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Progeroid features in a patient with Malouf syndrome due to a rare LMNA variant: a case report and review of the literature.

Laminopathiesrepresent a rare group of genetic disorders affecting various organs and tissues, including the skin, muscles, adipose tissue, bone, and cardiovascular system. The LMNA gene, the most common pathogenic gene responsible for laminopathies, harbors variants that can lead to diverse clinical phenotypes, such as progeroid syndromes, lipodystrophies, muscular dystrophies, and cardiomyopathies. This report presents a case of a young female patient who presented with prediabetes, secondary amenorrhea, and secondary osteoporosis. A 28-year-old female presented to our clinic with complaints of amenorrhea and decreased bone mineral density. She exhibited pronounced facial abnormalities and underdeveloped secondary sexual characteristics. Laboratory investigations revealed hypergonadotropic hypogonadism, prediabetes and hyperlipidemia. Significant mitral annular calcification was revealed via echocardiography. Genetic analysis revealed a de novo variant in exon 1 of the LMNA gene. This case reveals a novel laminopathy overlapping with the clinical features of Malouf syndrome while also exhibiting additional progeroid features, representing a distinct laminopathy. Furthermore, unlike previously reported cases with this genotype, it does not correspond to a progeroid syndrome typically associated with LMNA variants. Additionally, this case report is accompanied by a review of the relevant literature.

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来源期刊
Archives of Endocrinology Metabolism
Archives of Endocrinology Metabolism Medicine-Endocrinology, Diabetes and Metabolism
CiteScore
2.90
自引率
5.90%
发文量
107
审稿时长
7 weeks
期刊介绍: The Archives of Endocrinology and Metabolism - AE&M – is the official journal of the Brazilian Society of Endocrinology and Metabolism - SBEM, which is affiliated with the Brazilian Medical Association. Edited since 1951, the AE&M aims at publishing articles on scientific themes in the basic translational and clinical area of Endocrinology and Metabolism. The printed version AE&M is published in 6 issues/year. The full electronic issue is open access in the SciELO - Scientific Electronic Library Online e at the AE&M site: www.aem-sbem.com. From volume 59 on, the name was changed to Archives of Endocrinology and Metabolism, and it became mandatory for manuscripts to be submitted in English for the online issue. However, for the printed issue it is still optional for the articles to be sent in English or Portuguese. The journal is published six times a year, with one issue every two months.
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