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阿尔斯特伦综合征:一名沙特女性2型糖尿病患者伴完全失明的具有挑战性的病例研究。

Alström Syndrome: A Challenging Case Study of a Female Saudi Patient With Type 2 Diabetes Mellitus and Complete Vision Loss.

作者信息

Alamri Abdulrahman S, Mahmoud Hatim A, Abu Alnasr Abdulaziz A, Alahmadi Alaa K, Qari Yousef H

机构信息

Diabetes and Endocrinology Department, Prince Mohammed Bin Abdulaziz National Guard Hospital, Madinah, SAU.

Department of Family Medicine, Prince Mohammed Bin Abdulaziz National Guard Hospital, Madinah, SAU.

出版信息

Cureus. 2024 May 15;16(5):e60396. doi: 10.7759/cureus.60396. eCollection 2024 May.

DOI:10.7759/cureus.60396
PMID:38883102
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11179030/
Abstract

Alström syndrome is a genetic disease that impacts numerous systems in the human body. The symptoms can vary and appear gradually. Childhood obesity, heart disease (cardiomyopathy), abnormalities in vision, and hearing issues are the main symptoms of this disorder in children. Diabetes mellitus, hepatic issues, and renal dysfunction can all occur over time. Genetic alterations in the ALMS1 gene are the cause of Alström syndrome. It has an autosomal recessive inheritance pattern. We address the case of a Saudi woman in her 20s. She had been initially referred for type 2 diabetes, intellectual disability since early childhood, metabolic acidosis, and micrognathia; however, she also exhibited blindness, chronic kidney disease (CKD), and hearing loss, all of which are indicative of Alström syndrome. DNA testing showed that she has a homozygous pathogenic variant in the ALMS gene. Autosomal recessive Alström syndrome has been confirmed as a genetic diagnosis. No other clinically significant variations were found that are associated with the mentioned phenotype. By reporting this mutation, we hope to learn more about the genotypic range of the disease, particularly in the Saudi population. As each member of the family underwent genetic testing, we established a stringent follow-up schedule for our patient and her family.

摘要

阿尔斯特伦综合征是一种影响人体多个系统的遗传性疾病。症状可能各不相同且逐渐显现。儿童肥胖、心脏病(心肌病)、视力异常和听力问题是儿童期这种疾病的主要症状。随着时间的推移,还可能出现糖尿病、肝脏问题和肾功能障碍。ALMS1基因的基因改变是阿尔斯特伦综合征的病因。它具有常染色体隐性遗传模式。我们讲述一位20多岁沙特女性的病例。她最初因2型糖尿病、自幼智力残疾、代谢性酸中毒和小颌畸形前来就诊;然而,她还表现出失明、慢性肾脏病(CKD)和听力丧失,所有这些都表明是阿尔斯特伦综合征。DNA检测显示她在ALMS基因中有一个纯合致病性变异。常染色体隐性阿尔斯特伦综合征已被确认为基因诊断。未发现与上述表型相关的其他具有临床意义的变异。通过报告这一突变,我们希望更多地了解该疾病的基因型范围,尤其是在沙特人群中。由于家族中的每个成员都接受了基因检测,我们为患者及其家人制定了严格的随访计划。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/db19/11179030/cd7f81f7aa36/cureus-0016-00000060396-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/db19/11179030/f925197fa284/cureus-0016-00000060396-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/db19/11179030/cd7f81f7aa36/cureus-0016-00000060396-i02.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/db19/11179030/f925197fa284/cureus-0016-00000060396-i01.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/db19/11179030/cd7f81f7aa36/cureus-0016-00000060396-i02.jpg

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本文引用的文献

1
A review of Alström syndrome: a rare monogenic ciliopathy.阿尔斯特伦综合征综述:一种罕见的单基因纤毛病。
Intractable Rare Dis Res. 2021 Nov;10(4):257-262. doi: 10.5582/irdr.2021.01113.
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Novel Mutations of the ALMS1 Gene in Patients with Alström Syndrome.Alström 综合征患者中 ALMS1 基因的新突变。
Intern Med. 2021 Dec 1;60(23):3721-3728. doi: 10.2169/internalmedicine.6467-20. Epub 2021 Jun 19.
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Liver Fibrosis and Steatosis in Alström Syndrome: A Genetic Model for Metabolic Syndrome.阿尔斯特伦综合征中的肝纤维化和脂肪变性:代谢综合征的遗传模型
Diagnostics (Basel). 2021 Apr 28;11(5):797. doi: 10.3390/diagnostics11050797.
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Consensus clinical management guidelines for Alström syndrome.《Alström 综合征的临床管理共识指南》。
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Ophthalmic features of cone-rod dystrophy caused by pathogenic variants in the ALMS1 gene.ALMS1 基因突变致锥-杆细胞营养不良的眼部特征。
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Characteristics of cardiomyopathy in Alström syndrome: Prospective single-center data on 38 patients.阿尔斯特伦综合征中心肌病的特征:38例患者的前瞻性单中心数据。
Mol Genet Metab. 2017 Aug;121(4):336-343. doi: 10.1016/j.ymgme.2017.05.017. Epub 2017 May 30.
8
Whole exome sequencing of a consanguineous family identifies the possible modifying effect of a globally rare AK5 allelic variant in celiac disease development among Saudi patients.对一个近亲结婚家庭进行的全外显子组测序发现,一种全球罕见的AK5等位基因变异可能对沙特患者乳糜泻的发展产生修饰作用。
PLoS One. 2017 May 15;12(5):e0176664. doi: 10.1371/journal.pone.0176664. eCollection 2017.
9
Alström syndrome: A novel mutation in Saudi girl with insulin-resistant diabetes.阿尔斯特伦综合征:一名患有胰岛素抵抗性糖尿病的沙特女孩的新突变。
Medicine (Baltimore). 2017 Mar;96(10):e6192. doi: 10.1097/MD.0000000000006192.
10
Novel Mutations in Two Saudi Patients with Congenital Retinal Dystrophy.两名患有先天性视网膜营养不良的沙特患者中的新突变
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