Alamri Abdulrahman S, Mahmoud Hatim A, Abu Alnasr Abdulaziz A, Alahmadi Alaa K, Qari Yousef H
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.
Cureus. 2024 May 15;16(5):e60396. doi: 10.7759/cureus.60396. eCollection 2024 May.
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.
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。