Pekmezci Aslihan, Gumus Aydeniz Aydin, Korkmaz Ozge Polat
Department of Endocrinology and Metabolism, Basaksehir Cam and Sakura City Hospital, University of Health Sciences, Istanbul, Turkey.
Department of Medical Genetics, Basaksehir Cam and Sakura City Hospital, University of Health Sciences, Istanbul, Turkey.
Arch Endocrinol Metab. 2025 Jul 24;69(3):e240491. doi: 10.20945/2359-4292-2024-0491.
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.
核纤层蛋白病是一类罕见的遗传性疾病,会影响包括皮肤、肌肉、脂肪组织、骨骼和心血管系统在内的各种器官和组织。LMNA基因是导致核纤层蛋白病最常见的致病基因,携带着一些可导致多种临床表型的变异,如早老症综合征、脂肪营养不良、肌肉萎缩症和心肌病。本报告介绍了一例年轻女性患者,该患者出现了糖尿病前期、继发性闭经和继发性骨质疏松症。一名28岁女性因闭经和骨密度降低前来我院就诊。她表现出明显的面部异常和第二性征发育不全。实验室检查发现高促性腺激素性性腺功能减退、糖尿病前期和高脂血症。超声心动图显示二尖瓣环有明显钙化。基因分析发现LMNA基因第1外显子有一个新发变异。该病例揭示了一种与马洛夫综合征临床特征重叠的新型核纤层蛋白病,同时还表现出其他早老症特征,代表了一种独特的核纤层蛋白病。此外,与先前报道的具有这种基因型的病例不同,它并不对应于通常与LMNA变异相关的早老症综合征。此外,本病例报告还附有相关文献综述。