{"title":"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。","authors":"Aslihan Pekmezci, Aydeniz Aydin Gumus, Ozge Polat Korkmaz","doi":"10.20945/2359-4292-2024-0491","DOIUrl":null,"url":null,"abstract":"<p><p>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.</p>","PeriodicalId":54303,"journal":{"name":"Archives of Endocrinology Metabolism","volume":"69 3","pages":"e240491"},"PeriodicalIF":1.6000,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Progeroid features in a patient with Malouf syndrome due to a rare LMNA variant: a case report and review of the literature.\",\"authors\":\"Aslihan Pekmezci, Aydeniz Aydin Gumus, Ozge Polat Korkmaz\",\"doi\":\"10.20945/2359-4292-2024-0491\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><p>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.</p>\",\"PeriodicalId\":54303,\"journal\":{\"name\":\"Archives of Endocrinology Metabolism\",\"volume\":\"69 3\",\"pages\":\"e240491\"},\"PeriodicalIF\":1.6000,\"publicationDate\":\"2025-07-24\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Archives of Endocrinology Metabolism\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.20945/2359-4292-2024-0491\",\"RegionNum\":4,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q4\",\"JCRName\":\"ENDOCRINOLOGY & METABOLISM\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Archives of Endocrinology Metabolism","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.20945/2359-4292-2024-0491","RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q4","JCRName":"ENDOCRINOLOGY & METABOLISM","Score":null,"Total":0}
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.
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.